RCKT

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Second Quarter 2026 Financial Results and

Highlights Recent Progress

All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period; no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed

Rocket is actively engaging with the FDA to align on the path to rapidly completing the pivotal Phase 2 trial in Danon disease

Multiple clinical and regulatory milestones remain on track for the second half of 2026, including a broader Danon disease program update, a regulatory update for the planned RP-A601 pivotal study in PKP2-ACM and initial patient dosing with RP-A701 in BAG3-DCM

FDA approval of KRESLADI™ and subsequent monetization of the Priority Review Voucher generated $180 million in non-dilutive capital; focused preparations are underway for commercial availability and patient onboarding beginning in the fourth quarter of 2026

Cash, cash equivalents and investments of approximately $283.7 million; expected cash runway into the second quarter of 2028

CRANBURY, NJ – August 10, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing a sustainable pipeline of genetic medicines for rare disorders with high unmet need, today reported financial and recent operational results for the second quarter ended June 30, 2026.

“The second quarter of 2026 materially strengthened Rocket’s position, with the approval of KRESLADI demonstrating our ability to advance a genetic medicine through regulatory approval, the subsequent sale of the Priority Review Voucher generating $180 million in non-dilutive capital, and safety clearance for the first three Danon disease patients in our modified pivotal trial,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Together with the more focused operating structure we implemented in late 2025, these achievements provide a stronger financial and executional foundation for advancing our cardiovascular pipeline. Based on the positive safety observations in Danon disease, we are working with the FDA on the path to treating additional patients and completing the pivotal trial as quickly as possible, while progressing toward additional regulatory and clinical milestones across the remainder of our cardiac portfolio, including PKP2-ACM and BAG3-DCM.”

Recent Pipeline and Operational Updates

•

Positive Initial RP-A501 Safety Findings Support Regulatory Engagement on Next Steps in Danon Disease

o

Rocket previously disclosed that the initial three patients were safely dosed under the modified pivotal Phase 2 protocol for RP-A501 in Danon disease. All three patients have completed the protocol-defined safety observation period, with no thrombotic microangiopathy (TMA), capillary leak syndrome, or other significant safety concerns observed at the recalibrated dose with the refined immunomodulatory regimen.

o

The recalibrated Phase 2 dose of 3.8 × 10¹³ GC/kg was selected based on the characteristics of the current drug product, the underlying biology of Danon disease and the Phase 1 experience, with the objective of preserving the potential for meaningful clinical benefit while optimizing RP-A501’s benefit-risk profile.

o

Rocket is actively engaging with the FDA to seek alignment on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory pathway in the second half of 2026.

o

Rocket is on track to host an investor webinar in the second half of 2026 to provide a comprehensive Danon disease program update.

o

In parallel with the pivotal Phase 2 trial, Rocket’s global natural history study has enrolled more than 50 patients, including both males and females, building an increasingly robust longitudinal dataset to further characterize Danon disease.

•

Continued FDA Engagement and Phase 1 Enrollment for RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

o

Rocket continues to engage with the FDA regarding alignment on the design of a potential pivotal study of RP-A601 in PKP2-ACM with regulatory update expected in the second half of 2026. Previously reported Phase 1 findings demonstrated increased PKP2 protein expression and improved desmosomal localization, together with directional improvements in arrhythmia measures and right ventricular function.

o

The ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.

•

Phase 1 Trial Initiation Anticipated in 2026 for RP-A701 in BAG3-Associated Dilated Cardiomyopathy (BAG3-DCM)

o

Patient screening and enrollment has begun, with initial patient dosing anticipa

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports First Quarter 2026 Financial Results and

Highlights Recent Progress

Pivotal Phase 2 trial of RP-A501 for Danon disease progressing with dosing reinitiated

First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy

anticipated in mid-2026

KRESLADI™

granted FDA accelerated approval; Rare Pediatric Disease Priority Review Voucher

monetized for $180 million

Pro forma cash, cash equivalents and investments of approximately $322.6 million, including balance as of March 31, 2026, and non-dilutive proceeds from the PRV sale; expected operational runway into the second quarter of 2028

CRANBURY, NJ – May 7, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the first quarter ended March 31, 2026.

“During the first quarter, we continued advancing our cardiovascular gene therapy portfolio, including reinitiating dosing in the initial three-patient cohort of our pivotal Phase 2 study of RP-A501 for Danon disease, while also achieving the accelerated approval of KRESLADI for severe LAD-I,” said Gaurav Shah. “The subsequent $180 million non-dilutive monetization of our Rare Pediatric Disease Priority Review Voucher further strengthened our balance sheet and extended our expected cash runway into the second quarter of 2028. We remain focused on disciplined execution across our deep cardiovascular pipeline and anticipate providing an update on the Danon program in the second half of the year.”

Recent Pipeline and Operational Updates

•

Dosing the initial three-patient cohort for the Phase 2 study of RP-A501 in Danon disease is on track.

o

Rocket previously disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months.

o

Per agreement with FDA, three additional patients are being treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week inter-patient dosing interval and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with FDA regarding the completion of the Phase 2 pivotal study.

o

Following FDA alignment, Rocket anticipates providing a program update in the second half of 2026.

o

Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

•

Engagement with FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).

o

Rocket continues to engage with FDA on alignment for a potential pivotal Phase 2 trial design for RP-A601 in PKP2-ACM, while the ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.

•

Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM).

o

The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Dosing of the first patient is anticipated in mid-2026.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338.

•

FDA granted accelerated approval for KRESLADI™ (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I

(LAD-I).

o

In March 2026, FDA granted KRESLADI™ accelerated approval for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression.

o

On April 28, 2026, Rocket announced a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $180 million.

First Quarter 2026 Financial Results

•

Cash position. Cash, cash equivalents and investments as of March 31, 2026, were $144.4 million, excluding PRV monetization.

•

R&D expenses. Research and development expenses were $31.5 million for the three months ended March 31, 2026, compared to $35.9 million for the three months ended March 31, 2025. The decrease of $4.4 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $5.8 million, stock-based and other compensation and benefit expenses of $2.0 million due to decreased R&D headcount, partially offset by increa

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results and

Highlights Recent Progress

Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026

KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date

Dosing of first patient in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy anticipated in mid-2026

Cash, cash equivalents and investments of approximately $188.9M;

expected operational runway into the second quarter of 2027

CRANBURY, NJ – February 26, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the fourth quarter and year ended December 31, 2025.

“In 2025, we strengthened Rocket’s leadership in cardiovascular gene therapy, supported by more than five years of clinical experience in Danon disease and continued advancement across our PKP2-ACM and BAG3-DCM programs,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Our focus remains on rigorous clinical execution across three cardiovascular programs, including resuming the Phase 2 pivotal clinical trial in Danon disease, initiating the Phase 1 clinical trial in BAG3-DCM, and advancing alignment with the FDA on the pivotal study design for PKP2-ACM. As we move through 2026, Rocket is well positioned to advance three high-impact cardiovascular programs and thoughtfully expand our cardiac gene therapy pipeline.”

Recent Pipeline and Operational Updates

•

Dosing of additional patients for the Phase 2 study of RP-A501 for Danon disease anticipated in the first half of 2026.

o

In August 2025, Rocket disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months.

o

Per agreement with the FDA, three additional patients will be treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week interval between dosing and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with the FDA regarding the completion of the Phase 2 pivotal study.

o

Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

•

Engagement with the FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).

o

Rocket continues to work closely with the FDA to advance alignment on a potential pivotal Phase 2 trial design for RP-A601 in PKP2-ACM, while the ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.

•

Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM).

o

The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Rocket expects to dose the first BAG3-DCM patient mid-2026.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338.

•

FDA accepted the resubmission of the BLA for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

In October 2025, KRESLADITM received a Prescription Drug User Fee Act (PDUFA) target action date for March 28, 2026.

o

Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), with the approval of KRESLADI™.

Fourth Quarter and Full Year 2025 Financial Results

•

Cash position. Cash, cash equivalents and investments as of December 31, 2025, were $188.9 million.

•

R&D expenses. Research and development expenses were $142.0 million for the twelve months ended December 31, 2025, compared to $171.2 million for the twelve months ended December 31, 2024. The decrease of $29.2 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $10.8 million, professional fees of $7.0 million, lab supplies and office expenses of $4.4 million, stock-based and other compensation and benefit expenses of $3.7 million, and clinical trial expenses of $2.7 million. The reduction reflects disciplined resource allocation following the company’s recent organizational realignment.

•

G&A expenses. General and administrative expenses were $86.5 million for the twelve months ended December 31, 2025, compared to $102.0 million for the twelve months ended December 31, 2024. The decrease of $15.5 million in G&A expenses was primarily driven by decreases in c

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Third Quarter 2025 Financial Results and

Highlights Recent Progress

Pivotal Phase 2 trial of RP-A501 for Danon disease to resume in 1H 2026

KRESLADI™ for severe LAD-I on track for March 28, 2026 PDUFA date

Leadership updates with the appointments of Dr. Syed Rizvi, Chief Medical Officer, Christopher Stevens, Chief Operating Officer, and Sarbani Chaudhuri, Chief Commercial & Medical Affairs Officer

Cash, cash equivalents and investments of approximately $222.8M;

expected operational runway into the second quarter of 2027

CRANBURY, NJ – November 6, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the third quarter ending September 30, 2025.

“During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket’s AAV cardiovascular gene therapy portfolio,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “In under three months, we aligned with the FDA to resume the Phase 2 pivotal study of RP-A501 for Danon disease, an important milestone that underscores the FDA’s collaborative approach and recognition of the urgent need for innovative therapies in rare cardiovascular disorders. In parallel, we are advancing RP-A601 toward a pivotal Phase 2 study in PKP2-ACM and preparing RP-A701 for first-in-human evaluation in BAG3-DCM. Finally, the upcoming March PDUFA date for KRESLADI™ represents a significant near-term commercial milestone as we work to bring this potentially life-saving therapy to patients with severe LAD-I.”

Recent Pipeline and Operational Updates

•

Dosing of additional patients for the Phase 2 study of RP-A501 for Danon disease anticipated in the first half of 2026.

o

In August, Rocket disclosed that the U.S. Food and Drug Administration’s (FDA) lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months.

o

Per agreement with the FDA, three additional patients will be treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week interval between dosing and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with the FDA regarding the path forward for completion of the Phase 2 pivotal study.

o

Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

•

Engagement with the FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).

o

Rocket continues to collaborate with the FDA to align upon potential pivotal Phase 2 trial design to evaluate the efficacy and safety of RP-A601 for PKP2-ACM.

•

Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM).

o

The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with

BAG3-DCM.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT07137338.

•

FDA accepted the resubmission of the BLA for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

In October, KRESLADITM received a Prescription Drug User Fee Act (PDUFA) target action date for March 28, 2026.

o

Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher (PRV), with the approval of KRESLADI™.

•

Leadership updates with several key appointments to support Rocket’s transition toward late-stage development and anticipated commercialization.

o

In September 2025, Syed Rizvi, M.D., was named Chief Medical Officer, adding over 20 years of clinical strategy, development and commercialization experience and a proven track record in cell and gene therapy, guiding the development and commercialization of multiple cell therapies for cancer treatments, including three approved autologous CAR-T cell therapies. Dr. Rizvi most recently served as Chief Medical Officer at Poseida Therapeutics, where he advanced the allogeneic CAR-T and gene therapy pipeline through the Company’s acquisition by Roche in January 2025. Previously, Dr. Rizvi held global clinical leadership roles at Legend Biotech, Celgene, Novartis, Merck, Caribou Biosciences and Genta Inc.

o

Christopher Stevens, appointed Chief Operating Officer in July, adding more than two decades of global operations, product strategy, manufacturing, and supply chain leadership experience in the biopharmaceutical industry. Mr. Stevens previously served as Executive Vice President and Chief Patient Supply Officer at Spark Therapeutics, where he oversaw end-to-end gene t

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and

Highlights Recent Progress

Prioritizing development of RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM) to advance AAV cardiovascular gene therapy platform for sustained value creation

FDA RMAT designation awarded to RP-A601 for PKP2-ACM; Engaging with FDA on pivotal trial design following encouraging initial Phase 1 data at ASGCT

IND accepted and FDA Fast Track designation received for RP-A701 in BAG3-DCM program; Phase 1 trial start-up activities underway

Organizational restructuring expected to reduce headcount by approximately 30% and lower 12-month cash burn by nearly 25%

Chris Stevens appointed Chief Operating Officer

Cash, cash equivalents and investments of approximately $271.5M;

expected operational runway into the second quarter of 2027

CRANBURY, NJ – August 7, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the second quarter ending June 30, 2025.

“The second quarter of 2025 marked an important inflection point for Rocket as we refined our strategic focus around our AAV cardiovascular gene therapy platform and took multiple decisive steps to strengthen our financial foundation and thoughtfully adjust to market dynamics. We are fortifying our path to sustained value creation by leaning into programs with the highest value, conserving cash, and driving an efficient, agile organization,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “With regulatory alignment to resolve the clinical hold for RP-A501 in Danon disease in progress, momentum building for RP-A601 in PKP2-ACM as it advances toward a pivotal Phase 2 trial, and RP-A701 preparing to enter the clinic for the treatment of BAG3-DCM, Rocket is positioned as the leader in the development of gene therapies for inherited cardiomyopathies. While we are pausing additional investments in the FA and PKD programs, we are deeply grateful and committed to the patient communities and are exploring strategic alternatives to advance these programs externally. Finally, our recent reorganization ensures we are appropriately resourced to execute on our near-term milestones with nearly two years of capital.”

Recent Pipeline and Operational Updates

●

Investigation into the Serious Adverse Event (SAE) from the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing.

o

Rocket previously disclosed an SAE, related to clinical complications from capillary leak syndrome and unfortunately leading to the patient’s death. In response, Rocket paused dosing and initiated a root cause analysis, focusing on the recent addition of a C3 inhibitor to the pre-treatment regimen. On May 23, 2025, FDA placed the trial on clinical hold for further evaluation.

o

Rocket is actively working with FDA, independent safety monitors, and clinical experts to ensure patient safety and resume the trial.

o

While the clinical hold remains in place, the company cannot provide guidance on trial completion timing.

●

Actively engaging with FDA on the advancement of RP-A601 for PKP2 arrhythmogenic cardiomyopathy

(PKP2-ACM) in potential pivotal trial following initial positive Phase 1 data at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).

o

In July, Rocket received FDA Regeneration Medicine Advanced Therapy (RMAT) designation for RP-A601 for PKP2-ACM.

o

Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy outcomes of RP-A601 for PKP2-ACM were presented at ASGCT in May 2025.

o

Rocket is engaging with FDA on potential pivotal trial design to evaluate the efficacy and safety of RP-A601.

●

Phase 1 trial start-up activities are underway for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM).

o

In June, an Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-DCM, received clearance from FDA.

o

RP-A701 was recently granted FDA Fast Track designation, designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions that fill an unmet medical need. This enables increased communication with FDA, the potential for accelerated approval, and permits a rolling Biologics License Application (BLA) review.

o

The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with

BAG3-DCM.

o

BAG3-DCM is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premat

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports First Quarter 2025 Financial Results and

Highlights Recent Progress

Phase 2 pivotal study of RP-A501 for Danon disease is ongoing; Program update expected mid-year 2025

Initial data from the Phase 1 study of RP-A601 for PKP2-ACM anticipated May 2025

IND submission for BAG3-DCM program expected mid-year 2025

Sarbani Chaudhuri appointed Chief Commercial & Medical Affairs Officer

Cash, cash equivalents and investments of approximately $318.2M;

expected operational runway into the fourth quarter of 2026

CRANBURY, NJ – May 8, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the first quarter ending March 31, 2025.

"Rocket maintained strong momentum during the first quarter, and we look forward to sharing updates of our recent progress starting with initial data from the Phase 1 study of RP-A601 for PKP2-ACM this month, followed by an update on our Danon disease program in mid-year 2025. We also remain on track to submit an IND for BAG3-DCM. We continue to prioritize our current resources towards our AAV cardiovascular programs, which we see as holding the greatest potential to deliver transformative therapies to patients and provide the strongest value to shareholders.” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “As we continue into 2025, we remain steadfast in preserving resources and safeguarding financial health to keep on advancing our mission of seeking gene therapy cures for patients with devastating diseases, and as result have expanded our runway into the fourth quarter of 2026.”

Recent Pipeline and Operational Updates

•

Phase 2 pivotal study of RP-A501 for Danon disease is ongoing.

o

Program update anticipated in mid-year 2025 and a clinical data readout expected in mid-year 2026. Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

o

In March, the largest longitudinal natural history study of Danon disease to date was published in the Journal of the American Heart Association (JAHA), revealing key insights into the distinct cardiac patterns of Danon disease patients, showing earlier, more severe heart issues in male patients, while also noting that many females develop progressive cardiomyopathy and heart failure in adolescence or early adulthood.

•

Initial data from the low dose cohort of the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM) expected in May 2025.

o

Initial data from the Phase 1 study will be presented at an upcoming medical/scientific conference.

o

Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket’s pipeline of disclosed programs. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412.

•

Progressed BAG3-associated dilated cardiomyopathy preclinical program.

o

Submission of the IND is anticipated mid-year 2025.

•

Regulatory progression of RP-L102 investigational gene therapy for Fanconi Anemia (FA).

o

Initiated rolling Biologics License Application (BLA) for RP-L102, anticipating final module submission late 2025/early 2026.

o

Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority (EMA) is ongoing.

•

U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

In May, clinical outcomes data from the nine severe LAD-I patients treated in the global Phase I/II study of KRESLADITM was published in the New England Journal of Medicine (NEJM), highlighting the transformational effect of KRESLADITM towards the treatment of severe LAD-I and sustained durability with all patients remaining symptom-free at two years post-treatment without the need of an allogeneic HSCT.

o

Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe

LAD-I.

o

The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research. Submission of complete BLA to resolve Complete Response Letter anticipated in 2025.

•

In April, Rocket appointed Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer.

o

With over 20 years of experience in the biopharma industry, Chaudhuri is a seasoned executive with a proven track record of building, transforming, and steering companies towards delivering transformative patient outcomes.

o

Chaudhuri most re

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and

Highlights Recent Progress

Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025

New England Journal of Medicine publication of Phase 1 study of RP-A501 to treat patients with Danon disease

Long-term data from the Phase 1 trial of RP-A501 for Danon disease presented at AHA on safety and meaningful efficacy

Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Initial data expected in the first half of 2025

Regulatory reviews ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia)

Cash, cash equivalents and investments of approximately $372.3M;

expected operational runway into the third quarter of 2026

CRANBURY, NJ – Feb. 27, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the fourth quarter and year ended December 31, 2024.

"In 2024, we made strong progress in advancing our gene therapy pipeline, underscored by the New England Journal of Medicine publication of the Phase 1 study of RP-A501 for Danon disease and long-term data presented at AHA showing its safety and meaningful efficacy up to five years. Our momentum continues as we progress with the Phase 2 pivotal trial of RP-A501 and the Phase 1 trial of RP-A601 for PKP2-ACM, and we remain on track to submit the IND for BAG3-DCM in the first half of 2025,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “Looking ahead to 2025, we will maintain our focus and resources on advancing our AAV cardiovascular programs while seeking to realize value in our full pipeline in a thoughful manner, so we deliver the greatest value to our patients and shareholders.”

Recent Pipeline and Operational Updates

•

Dosing in the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing.

o

Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

o

Program update anticipated in the first half of 2025.

•

Long-term data from the Phase 1 study of RP-A501 for Danon disease published in The New England Journal of Medicine and new data presented at the American Heart Association’s 2024 Late-Breaking Science sessions.

o

RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10%

reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years).

o

Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months.

o

All evaluable patients had reductions in NYHA heart failure (from Class II to Class I; no longer displaying symptoms of heart failure),

improvements in KCCQ (median 27-point increase), and substantial improvements in troponin (median reduction 84%) and BNP (median reduction 57%) observed 24-54 months after treatment.

•

Progressed the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).

o

Completed enrollment of all patients in the low dose cohort, and initial data from the Phase 1 study is expected in the first half of 2025.

o

Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket’s pipeline of disclosed programs.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412.

•

Progressed BAG3-associated dilated cardiomyopathy preclinical program.

o

Nonclinical, IND-enabling studies are ongoing.

o

Submission of the IND is anticipated in the first half of 2025.

•

Progressed RP-L102 investigational gene therapy for Fanconi Anemia (FA).

o

Initiated rolling Biologics License Application (BLA) for RP-L102 and submission of the final module is anticipated in 2025.

o

Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is ongoing.

•

U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe

LAD-I.

o

The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research.

o

Submission of complete BLA to resolve Complete Response Letter anticipated in 2025.

•

Raised net proceeds of $182.5M through public of

EX-99.1

2 ef20038212_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and

Highlights Recent Progress

Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of

RP-A501 for Danon disease

Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association’s 2024 Late-Breaking Scientific Sessions

Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025

Rolling BLA submission initiated for RP-L102 for Fanconi Anemia

Progress on FDA-approval of KRESLADI for severe LAD-I; Approval anticipated in 2025

Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors

Cash, cash equivalents and investments of approximately $235.7M;

expected operational runway into 2026

CRANBURY, NJ – Nov. 7, 2024 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the third quarter ending September 30, 2024.

“Rocket made meaningful progress during the third quarter, notably with the completion of enrollment in the RP-A501 program for Danon disease, low dose cohort enrollment completion in the RP-A601 program for PKP2-ACM, and appointment of seasoned pharmaceutical executive, Mikael Dolsten to our Board of Directors,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “As we continue to pursue our mission of seeking gene therapy cures for patients with rare and devastating diseases, we remain focused on expediently advancing our deep pipeline of cardiovascular and hematology programs.”

Recent Pipeline and Operational Updates

•

Continued advancement of Phase 2 pivotal study of RP-A501 for Danon Disease.

o

In September, Rocket announced completion of enrollment in the Phase 2 pivotal study of RP-A501 to treat Danon Disease.

o

Dosing in the Phase 2 pivotal study is ongoing.

o

Updated data from the Phase 1 study to be presented at the American Heart Association’s 2024 Late-Breaking Science sessions on November 18.

o

Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

•

Progressed the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).

o

Completed patient enrollment in the low dose cohort.

o

Preliminary data from the Phase 1 study is expected in the first half of 2025.

o

Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe, representing the largest market opportunity in Rocket’s pipeline of disclosed programs.

o

Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412.

•

Progressed BAG3-associated dilated cardiomyopathy preclinical program.

o

Nonclinical, IND-enabling studies are ongoing.

o

Submission of the IND is anticipated in the first half of 2025.

•

Progressed RP-L102 investigational gene therapy for Fanconi Anemia (FA).

o

Initiated rolling Biologics License Application (BLA) for RP-L102.

o

Secured an ICD-10 code from the Centers for Medicare and Medicaid Services to document patients with FA.

o

Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is underway.

•

U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I.

o

The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research.

o

Approval of KRESLADI anticipated in 2025.

•

Initiated global Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency.

o

Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06422351.

•

Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors.

o

Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. He currently serves as the Chief Scientific Officer, President, Research & Development at Pfizer Inc., focused on advancing gene therapies, small-molecule medicines, biotherapeutics, and vaccines.

Third Quarter Financial Results

•

Cash position. Cash, cash equivalents and investments as of September 30, 2024, were $235.7 million.

•

R&D expenses. Research and development expenses were $133.9 million for the nine months ended September 30, 2024, compared to $144.6 million for the nine months ended September 30, 2023. The decrease of $10.7 million in R&D expenses was prima

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and

Highlights Recent Progress

Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2 arrhythmogenic cardiomyopathy

Working toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch

Presented long-term KRESLADITM follow-up data from the global Phase 1/2 study for severe LAD-I, results from the global Phase 1/2 study of RP-L102 for Fanconi Anemia, and data from the Phase 1 study of RP-L301 for PKD at ASGCT in May

Cash, cash equivalents and investments of approximately $278.8M; expected operational runway into 2026

CRANBURY, NJ – Aug. 5, 2024 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending June 30, 2024.

“Over the quarter, Rocket has been advancing its clinical pipeline as we progressed our RP-A501 and RP-A601 cardiac programs, targeting Danon disease and PKP2-ACM, and continued to actively enroll patients,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “At ASGCT, we shared follow-up data from across our hematology portfolio including 4-year follow-up data for KRESLADI to treat patients with severe LAD-I, demonstrating a 100% survival rate. In parallel, we have been preparing for the anticipated FDA approval of KRESLADI.”

Recent Pipeline and Operational Updates

•

Continued advancement of Phase 2 pivotal study of RP-A501 for Danon Disease.

o

Enrollment in the Phase 2 pivotal study of RP-A501 to treat Danon Disease is actively progressing. Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.

•

Granted orphan medicinal product designation from the European Commission (EC) for RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).

o

In May, Rocket announced orphan medicinal product designation from the EC for RP-A601 for the treatment of PKP2-ACM. Enrollment in the Phase 1 study is ongoing. Details of the study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.

o

Orphan medicinal product designation by the EC is available to novel therapeutics that prevent or treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 persons in the European Union (EU). The designation qualifies for financial and regulatory benefits including protocol assistance from the European Medicines Agency during clinical development, access to centralized marketing authorization, and a 10-year period of marketing exclusivity after product approval.

•

U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information underway for KRESLADITM for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

o

In June, Rocket announced that the FDA requested limited additional CMC information to complete its review of KRESLADITM (marnetegragene autotemcel; marne-cel) to treat severe LAD-I. The Company is working with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research to support the approval of KRESLADITM.

o

Long-term KRESLADITM follow-up data from the global Phase 1/2 study were presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting. Data demonstrated survival of 100% in the absence of allogeneic hematopoietic stem cell transplantation from 18 to 45 months with a well-tolerated safety profile in all nine patients with severe

LAD-I.

•

Progressed Fanconi Anemia (FA) program through regulatory and clinical milestones.

o

Regulatory filings and review for RP-L102 for the treatment of FA are on track with health authorities in the U.S. and Europe.

o

Results from the global Phase 1/2 study of RP-L102 were presented at the ASGCT 27th Annual Meeting. Previously disclosed data demonstrated genetic and phenotypic correction combined with hematologic stabilization extending to 42 months with polyclonal integration patterns.

•

Furthered Pyruvate Kinase Deficiency (PKD) program through clinical milestones.

o

Updated data from the Phase 1 study of RP-L301 for PKD were presented at the ASGCT 27th Annual Meeting. Sustained and clinically meaningful hemoglobin improvement and well-tolerated safety profile were observed in PKD patients up to 36 months after RP-L301 treatment.

o

The global Phase 2 pivotal study of RP-L301 for PKD has been initiated. Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06422351.

•

Progressing BAG3-associated dilated cardiomyopathy preclinical program.

o

Nonclinical, IND-enabling studies are o

EX-99.1

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EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Advanced RP-L102 for Fanconi Anemia towards regulatory reviews; EMA accepted MAA for review and BLA submission anticipated in the first half of 2024

Continued preparations to launch LV portfolio beginning with KRESLADI™ (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024

Appointed Aaron Ondrey as Chief Financial Officer, bringing seasoned leadership experience in commercial-stage financial management, strategic planning, and capital allocation

Cash, cash equivalents and investments of approximately $330.3M; expected operational runway into 2026

CRANBURY, NJ – May 6, 2024 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024.

“Rocket has had a strong start to 2024. This quarter has been marked by meaningful progress across all our clinical programs, most notably with the EMA’s review of RP-L102 for Fanconi Anemia, commercial preparations for the potential approval of KRESLADI for severe LAD-I, and ongoing enrollment in the Phase 2, pivotal study of RP-A501 for Danon Disease and the Phase 1 study of RP-A601 for PKP2-ACM,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “As we build on the advancements made this quarter, we remain focused on execution for patients with rare and devastating diseases with limited treatment options.”

Recent Pipeline and Operational Updates

•

Advanced Fanconi Anemia (FA) program through key regulatory milestones.

o

In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA. MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.

o

The Company remains on track to submit the Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2024.

•

KRESLADI™ on track for June 30, 2024, PDUFA date.

o

Rocket continues to ramp up enabling activities to support the launch of its lentiviral (LV) vector portfolio beginning with KRESLADI™ for severe Leukocyte Adhesion Deficiency-I (LAD-I). Qualified Treatment Center initiation, disease education, payer engagement, and field team build-out are all underway.

•

Bolstered finance, investor relations, and corporate communications expertise of company leadership to support evolution towards commercial stage.

o

Rocket appointed Aaron Ondrey as Chief Financial Officer (CFO). Mr. Ondrey brings over 20 years of experience leading commercial-stage financial management, strategic planning, and capital allocation. Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

o

In addition, Meg Dodge was appointed Vice President to lead Investor Relations & Corporate Communications. Ms. Dodge is experienced with engaging stakeholders across investors, media, and other communities in the biotech and financial sectors. Prior to joining Rocket, Ms. Dodge was Head of Investor Relations and Corporate Communications at Krystal Biotech.

•

Celebrated annual Rare Disease Day with multi-faceted awareness campaign.

o

On February 29, 2024, Rocket hosted its annual Rare Disease Day recognition program highlighting the theme, “Leap into Action for Rare.” Several hundred attendees gathered at the Liberty Science Center and virtually to hear inspirational stories from the community. Rocket also continued its Light Up for Rare initiative in collaboration with global partners to light up buildings and landmarks in Rare Disease Day colors, including the Empire State Building.

o

Rocket remains highly committed to supporting the rare disease community through patient-focused events, education and advancing science to bring potential treatments to patients with unmet needs.

•

Milestones in 2024 are on track across its pipeline of gene therapies for rare and devastating diseases.

o

Rocket continues to advance three disclosed programs from its adeno-associated virus (AAV) cardiovascular portfolio, including:

■

Phase 2 pivotal study of RP-A501 for Danon Disease,

■

Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and

■

IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM)

o

In its late-stage LV portfolio, Rocket is working towards initiation of the Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

•

Data from Rocket’s LV hematology portfolio to be presented at the American Society of Gene and Cell Therapy (ASGCT) 27th Annu

EX-99.1

2 ef20022436_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Progress

Advancing leading pipeline of six disclosed programs across AAV cardiovascular and LV hematology portfolios; all milestones remain on track for 2024

Expanding commercial capabilities to support launch of LV portfolio beginning with KRESLADITM (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024

Cash, cash equivalents and investments of approximately $407.5M; expected operational runway into 2026

CRANBURY, N.J. – Feb. 26, 2024 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2023.

“I am pleased with the strong results Rocket delivered in 2023, as we closed another successful year of progress across all six disclosed gene therapy programs spanning our AAV cardiovascular and LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “We are well poised to make further strides in 2024, notably expanding our commercial and operational capabilities to support the anticipated launch of KRESLADI™ (marnetegragene autotemcel) for severe LAD-I, representing Rocket’s first step in making our gene therapies available to patients who need them most. At the same time, we are laser focused on regulatory filings in Fanconi Anemia and continuing to advance our clinical programs in Danon Disease, PKP2-ACM and PKD in the year ahead. As we close the quarter, Rocket continues to solidify our industry leadership in gene therapy across multiple therapeutic areas and platforms as we seek to meet the needs of patients living with rare and devastating diseases.”

Recent Pipeline and Operational Updates

•

Milestones remain on track for 2024 across pipeline of adeno-associated virus (AAV) cardiovascular and lentiviral (LV) vector hematology portfolios. The Marketing Authorisation Application (MAA) and Biologics License Application (BLA) for Fanconi Anemia remain on track for filing with the European Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA), respectively, in the first half of 2024. Rocket is also progressing its Phase 2 pivotal study of RP-A501 for Danon Disease, Phase 2 pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD), Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy (ACM), and IND-enabling studies for BAG3-associated dilated cardiomyopathy (DCM).

•

KRESLADITM for severe Leukocyte Adhesion Deficiency-I (LAD-I) on track for PDUFA date of June 30, 2024. Based

on the positive top-line efficacy and safety data from the global Phase 1/2 study and high unmet need in patients with severe LAD-I, the FDA accepted the BLA and granted Priority Review for KRESLADITM. On February 13, 2024, Rocket announced the FDA extended the Priority Review period by three months, to June 30, 2024, to allow additional time to review clarifying Chemistry, Manufacturing, and Controls (CMC) information submitted by Rocket in response to FDA information requests. The FDA further confirmed that an advisory committee meeting is not needed. In support of its first product launch, Rocket continues to expand its commercial and operational infrastructure, including Qualified Treatment Center initiation, channel strategy, disease education, and payer engagement.

•

Published data from preclinical study of RP-A601 in PKP2-ACM. “AAV-Mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans” was published in Circulation: Genomic and Precision Medicine. Results from the study demonstrated the potential of RP-A601 as a gene therapy for patients with PKP2-ACM.

•

Bolstered commercial and operational expertise of Board of Directors with appointment of R. Keith Woods. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain. He recently served as Chief Operating Officer of argenx, where he led its global commercial organization, including marketing, market access, medical affairs, program management, and supply chain, during the company’s successful transition to commercial stage. Mr. Woods launched Vyvgart, a treatment for a rare autoimmune condition that causes muscle weakness, generating more than $1 billion in sales.

Upcoming Investor Conferences

•

Cowen’s 44th Annual Health Care Conference: March 5, 2024

•

Leerink Partners Global Biopharma Conference: March 12, 2024

•

Needham 23rd Annual Virtual Healthcare Conference: April 9, 2024

Fourth Quarter and Full Year 2023 Financial Results

•

Cash position. Cash, cash equivalents a

EX-99.1

2 brhc10048939_ex99-1.htm

EXHIBIT 99.1

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results

Advanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results for Danon Disease, Fanconi Anemia, LAD-I and PKD

Expanded leadership position in AAV cardiac gene therapy, with significant opportunities for value creation across Danon Disease, PKP2-ACM and BAG3-DCM programs

Established in-house cGMP manufacturing for AAV, poised to support Danon pivotal trial in Q2’23 and subsequent commercialization

Cash, cash equivalents and investments of approximately $400M; expected operational runway through 2024

CRANBURY, N.J. – Feb. 27, 2023 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare  disorders with high unmet need, today reported financial and operational results for the fourth quarter and year ended December 31, 2022.

“Rocket closed 2022 with positive results across four clinical gene therapy programs spanning both our AAV cardiology and LV hematology therapeutic areas in an unprecedented fashion for a gene therapy company, validating our approach to strong science and R&D, carefully selected assets and smart execution. We are thrilled to begin 2023 with the FDA recently granting RMAT designation to RP-A501 for Danon Disease, validating the strength of our results and underscoring RP-A501's potential as a transformative therapy for Danon patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.

Dr. Shah continued, “At the same time, we expanded our leadership position in AAV cardiac gene therapy with the recent unveiling of compelling preclinical proof of concept for RP-A601 for the treatment of PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) and addition of the BAG3-associated dilated cardiomyopathy (DCM) asset following the acquisition of Renovacor. Taken together, these two diseases along with Danon Disease affect more than 100,000 patients in the U.S. and EU. Further, our LV hematology portfolio delivered stellar results as we now advance towards our first regulatory filings for Leukocyte Adhesion Deficiency (LAD-I) in the second quarter of 2023 and Fanconi Anemia (FA) in the fourth quarter of 2023, and subsequent commercialization.”

“I am also pleased that we brought in additional funds of $197.7 million in 2022. We begin this year in a strong financial position to execute on near and long-term milestones, anticipating that our cash runway of approximately $400 million will fund operations through 2024,” said Dr. Shah. “I am grateful to our growing Rocket team of multi-disciplinary experts, scientific collaborators and the critical voices of the patient community who were essential to this progress and continue to help advance our mission of seeking gene therapy cures. I look forward to building off the successes of 2022 and continuing our progress in 2023.”

Key Pipeline and Operational Updates

•

Announced positive clinical data from Phase 1 trial of RP-A501 for Danon Disease. The Company provided an update at the 41st Annual J.P. Morgan Healthcare Conference that continued to demonstrate RP-A501 was generally well tolerated with evidence of restored expression of the deficient LAMP2 protein and durable improvement or stabilization of clinical parameters in the adult and pediatric Danon Disease patients treated in the Phase 1 study. The Company previously presented positive data from the study at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. The FDA recently granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501 that will provide the benefits of added intensive FDA guidance and expedited review through the program’s development. The initiation of the Phase 2 pivotal trial is on track for the second quarter of 2023. As previously disclosed, the Company anticipates pursuing a single arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.

•

Successfully completed two in-house AAV production runs at state-of-the-art,

Current Good Manufacturing Practice (cGMP) manufacturing and R&D facility in Cranbury, N.J. In-house production runs of AAV drug product for the planned Phase 2 pivotal study in Danon Disease resulted in high-quality drug substance enabling an approximately threefold increase in the number of patients treatable per batch, a significantly improved full versus empty particle ratio, and promising product comparability data generated to date compared to the Phase 1 material manufactured externally.

•

Announced positive top-line data from Phase 2 pivotal trial of RP-L201 for

Leukocyte Adhesion Deficiency-I (LAD-I). Positive top-line data presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) and at the 64th American Soci

EX-99.1

2 brhc10043730_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Third Quarter 2022 Financial Results and Highlights Recent Progress

— Positive updates from Phase 1 clinical trial in Danon Disease showed RP-A501 was well tolerated with evidence of durable treatment effect and disease improvement for both pediatric patients and adult patients with up to nine and 36 months of follow-up, respectively—

— Phase 2 pivotal study design and endpoint selection for Danon Disease on track with FDA feedback anticipated in Q4 2022; previously disclosed data to be presented at AHA 2022—

— LAD-I and Fanconi Anemia (FA) gene therapy programs remain on track, regulatory filings for LAD-I and FA anticipated 1H 2023 and 2H 2023, respectively; clinical data across LAD-I, FA and PKD lentiviral (LV) programs to be presented at the 64th ASH Annual Meeting—

— Announced acquisition of Renovacor extending Rocket’s leadership and capabilities in adeno-associated virus (AAV)-based cardiac gene therapy; expected to close by Q1 2023—

— Enrollment completed in Phase 1 trial of RP-L301 for PKD; Phase 1 update now anticipated 1H 2023 and Phase 2 pivotal trial initiation expected in 2023—

— Cash, cash equivalents and investments of $306.5M; with additional net proceeds of $108.2M from recent equity offering, expected operational runway now extends into 2H 2024, pending close of Renovacor acquisition—

CRANBURY, N.J. – November 3, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending September 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

“Rocket had a remarkable third quarter highlighted by significant progress in our Phase 1 clinical trial for Danon Disease, with positive early findings demonstrating RP-A501 was generally well tolerated with evidence of positive treatment effect and improvement of the natural course of the disease in pediatric patients with up to nine months follow-up. Additionally, we observed continued, durable robust activity in adults with up to 36 months follow-up,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “As previously announced, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal study. We are now laser focused on advancing Phase 2 pivotal study design and endpoint selection with FDA feedback expected this quarter.”

Dr. Shah continued, “In the third quarter, we also announced the expected acquisition of Renovacor which aims to further solidify our leadership in AAV-based cardiac gene therapy. By combining Rocket’s clinical, regulatory, and chemistry, manufacturing and control expertise with Renovacor’s compelling preclinical research in BAG3-associated dilated cardiomyopathy and early assets, we look forward to advancing precision gene therapies even further to address the significant unmet need of patients with genetically driven cardiac diseases.”

“Based on the strong results presented across our Leukocyte Adhesion Deficiency-I (LAD-I) and Fanconi Anemia (FA) lentiviral gene therapy programs to date, we remain on track towards our first regulatory filings in 2023 – LAD-I in the first half and FA in the second half – and expect to present data on both programs at ASH in December,” said Dr. Shah. “Regarding our Pyruvate Kinase Deficiency (PKD) program, we also expect to share updated data from both adult patients at ASH. Both pediatric patients in the study have been enrolled. We now anticipate a Phase 1 update in the first half of 2023 and initiation of the Phase 2 pivotal trial in 2023.”

Dr. Shah continued, “Finally, our exceptional third quarter progress was capped off by an extension of our cash runway into the second half of 2024 following an equity raise with net proceeds of $108.2 million. Taken together, I am very proud of our continued progress at Rocket and look forward to the final push towards another successful year in our pursuit of gene therapy cures for patients and their loved ones facing these devastating, life-threatening genetic diseases.”

Key Pipeline and Operational Updates

•

Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. Data presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022 included updated safety and efficacy data from patients in the pediatric and adult cohorts. Results showed RP-A501 was generally well tolerated with evidence of treatment effect and improvement of Danon Disease, including for both pediatric patients with up to nine months of follow-up and four adult patients with up to 36 months of f

EX-99.1

2 brhc10040558_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Second Quarter 2022 Financial Results and Highlights Recent Progress

— Reported positive safety data at ASGCT from pediatric cohort of Phase 1 Danon Disease study that demonstrated RP-A501 was well-tolerated; efficacy update across pediatric as well as adult cohorts on track for late Q3 2022—

— Announced top-line data at ASGCT from pivotal Phase 2 trial for severe LAD-I that showed RP-L201 was well-tolerated with 100% overall survival at one year; regulatory filings anticipated in first half of 2023 —

— Primary endpoint met for Fanconi Anemia pivotal Phase 2 trial, FDA dialogue initiated for BLA planning; continued data readouts for Fanconi Anemia and Pyruvate Kinase Deficiency programs expected in Q4 2022 —

— Achieved Current Good Manufacturing Practice (cGMP) readiness milestone for state-of-the-art AAV manufacturing facility in Cranbury, N.J.; continuing on the path toward commercialization —

— Appointed proven gene therapy technical operations expert and manufacturing leader Mayo Pujols as first Chief Technical Officer —

— Cash, cash equivalents and investments of $321.4M; expected operational runway into first half of 2024 —

CRANBURY, N.J. – August 8, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today reports financial results for the quarter ending June 30, 2022, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.

“Rocket continues on an excellent trajectory following a strong and highly productive quarter that featured positive results across all four clinical programs, concrete steps taken toward manufacturing readiness and leadership, and filing preparedness for our first two gene therapies,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “Positive safety data from our Phase 1 study for Danon Disease demonstrated RP-A501 was well-tolerated in both pediatric patients. We now look forward to presenting early efficacy data from the pediatric cohort with three to six months of follow-up in late Q3. If early signals of efficacy and ongoing tolerability in the pediatric cohort are demonstrated along with evidence of longer-term safety and efficacy in the adult cohort, we expect to begin Phase 2 pivotal trial planning activities in Q4, including FDA alignment on study design and endpoints.”

Dr. Shah continued, “In parallel, we reached an understanding with the FDA on chemistry, manufacturing and controls (CMC) requirements to start AAV cGMP manufacturing at our in-house facility as well as potency assay plans for a Phase 2 pivotal trial in Danon Disease. To further strengthen our manufacturing and commercial capabilities, we appointed Mayo Pujols, one of the most seasoned cell and gene therapy technical operations and manufacturing leaders in the industry, as our Chief Technical Officer.”

“This quarter, we also shared positive top-line data from our pivotal Phase 2 trial for severe LAD-I showing that RP-L201 was well-tolerated and associated with 100% overall survival at one year,” said Dr. Shah. “We have initiated work towards regulatory filings planned for the first half of 2023. In addition, based on achievement of the primary endpoint in our pivotal Phase 2 study for Fanconi Anemia, we have initiated FDA dialogue around BLA planning activities."

“These steps deliver on our effort to best leverage our strong cash position to create value as we embark on transitioning from a clinical to a commercial-stage company. Importantly, we continue to maintain a healthy operational cash runway into the first half of 2024,” concluded Dr. Shah. “Taken together, our positive data updates and steady progress this quarter continue to motivate us to push the boundaries of science and deliver on our mission to seek gene therapy cures for patients and families facing devastating, life-threatening diseases.”

Key Pipeline and Operational Updates

•

Danon, FA, LAD-I and PKD trials remain on track. All 2022 milestones remain on track, including pediatric efficacy data readout from the Phase 1 Danon Disease trial in late Q3, updated results for FA and preliminary Phase 1 data readout for PKD in Q4. The originally planned Q3 topline readout for FA was achieved earlier than anticipated in Q2 when the trial met its primary endpoint.

•

Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. Data

presented at the 2022 Annual Meeting of the ASGCT included new initial safety data from the low-dose (6.7 x 1013GC/kg; n=2) pediatric cohort as of April 30, 2022, cut-off date. Results demonstrated RP-A501 was well-tolerated in both patients. The patients were observed to have normal-range platelets, diminished complement activation and no complement-r

EX-99.1

2 brhc10034431_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial and Operational Results

—

Positive Phase 1 data from Danon Disease gene therapy trial demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four adult patients with long-term follow up; pediatric cohort data expected Q3 2022 —

—

Reported engraftment in all nine severe LAD-I patients treated in pivotal Phase 2 trial and CD18 expression ranging from 26% to 87% of normal; top-line data expected Q2 2022—

—

Demonstrated evidence of engraftment in six Fanconi Anemia patients with at least 12-months of follow-up with MMC resistance between 16% and 63% at a minimum of one timepoint in pivotal Phase 2 trial; top-line data expected Q3 2022 —

— Cash position of $388.7M; runway expected into 2H’23 —

CRANBURY, N.J. – Feb. 24, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial and operational results for the fourth quarter and year ended December 31, 2021.

“2021 was a very productive year marked by clinical data supporting potential durable cures in three devastating bone marrow-derived diseases, and for the first time in history by the demonstration of potential durable clinical benefit in a cardiac disease using a gene therapy approach,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “The stage is set for 2022 to be an important year for Rocket in which we anticipate progression of the Danon Disease program toward a registrational trial, as well as top-line data from our pivotal LAD-I and Fanconi Anemia clinical trials that move us toward registration. I am growing increasingly confident about our unique gene therapy approach at Rocket focused on addressing specific rare diseases using highly targeted, enabled technologies with a proven track record. Additionally, we anticipate in-house AAV cGMP manufacturing readiness in the second quarter of this year, as we rapidly scale our capabilities and work towards commercial readiness.”

Dr. Shah continued, “I am extremely proud of the positive momentum witnessed in 2021 and our steadfast focus on superior execution. Notably, we announced positive data from our Phase 1 trial in Danon Disease showing clinical, functional and biomarker improvements at one year or beyond, and we initiated treatment in the pediatric patient cohort at the low-dose, with pediatric data anticipated in the third quarter of 2022. In our pivotal Phase 2 trials in LAD-I and Fanconi Anemia, the favorable safety and efficacy observed are very encouraging as we approach top-line data readouts. We were also pleased with the progress of our PKD program demonstrating sustained normal-range hemoglobin through 12-months post-treatment in two adult patients in the Phase 1 trial. As we continue accelerating our current first wave of clinical programs, we are also poised to grow our Wave 2 pipeline based on our core R&D strategy from this year onward. We are excited by the momentum and look forward to continuing on our mission of seeking gene therapy cures for young patients and their families facing such devastating diseases.”

Key Pipeline and Operational Updates

•

Announced positive clinical data from ongoing Phase 1 trial of RP-A501 for Danon Disease. In November 2021, the Company reported updated data from the Phase 1 trial evaluating a single intravenous infusion of RP-A501 for the treatment of Danon Disease, including interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts. Results demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up. RP-A501 was generally well tolerated at the low-dose. Data from the pediatric patient cohort of the Phase 1 trial are expected in the third quarter of 2022 and Phase 2 trial activities are expected to begin in the fourth quarter of 2022.

•

Announced positive clinical data from ongoing pivotal Phase 2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). The Company provided an update in its presentation at the 40th Annual J.P. Morgan Healthcare Conference, indicating engraftment in all nine severe LAD-I patients who have received RP-L201 in the pivotal Phase 2 trial; all patients had at least three months of follow-up and demonstrated CD18 expression ranging from 26% to 87% of normal. The most recently treated patient has engrafted with neutrophil CD18 expression of 61% at three months. The Company previously presented preliminary data from eight of nine severe LAD-I patients who received RP-L201 treatment at the 63rd American Society of Hematology (ASH) Annual Meeting. Top-line data from the pivotal Phase 2 trial are expected in the second quarter of 2022.

EX-99.1

2 brhc10030396_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Third Quarter 2021 Financial Results and Highlights Recent Progress

— Treatment initiated in pediatric patient cohort in Danon Disease trial of RP-A501—

— Treatment completed in nine of nine patients in LAD-1 Phase 1/2 trial; favorable safety profile and initial clinical benefit reported in all seven patients with at least 3 months of follow up —

— Clinical updates anticipated in Danon Disease at AHA 2021 Scientific Sessions and in FA, LAD-I and PKD at 63rd ASH Annual Meeting —

— Ending Balance Sheet with $421.5 Million in Cash; Cash Runway Expected into 2H’23 —

CRANBURY, NJ – November 3, 2021 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter ending September 30, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones.

“We are excited about the strong progress we made in the third quarter as we initiated treatment in our trial of RP-A501 for Danon Disease in the low-dose (6.7e13 vg/kg) pediatric patient cohort,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “I am proud of our team’s collaboration with the FDA, through which we were able to swiftly resolve the clinical hold on the Danon trial and resume this highly important work on behalf of Danon patients. We are gearing up for the remainder of 2021 and look forward to reporting a comprehensive clinical update on our Phase I trial in Danon Disease this month at the AHA Scientific Sessions as well as key updates on all five of our first-in-class gene therapy programs throughout the fourth quarter.”

Dr. Shah continued, “We are equally excited about the progress of our LAD-I program, where we completed treatment for all nine patients in the RP-L201 Phase 1/2 clinical trial and presented positive interim data updates on the initial seven patients at the ESGCT Congress in October. Based on the data presented from these seven patients, RP-L201 continues to demonstrate a favorable safety profile and preliminary clinical benefit in patients with severe LAD-I. We will share additional clinical data from the LAD-I trial at the 63rd ASH Annual Meeting in December, where we will also report clinical updates on our Fanconi Anemia and PKD programs. I am proud of the Rocket team’s unwavering dedication to developing and bringing life-changing curative therapies to patients with rare diseases.”

Key Pipeline and Operational Updates

Danon Disease:

•

Initiated pediatric patient treatment in Phase 1 trial of RP-A501 for the treatment of Danon Disease. Rocket has resumed patient enrollment and initiated treatment in the low-dose (6.7e13 vg/kg) pediatric patient cohort. A comprehensive clinical update is anticipated at the American Heart Association (AHA) Scientific Sessions 2021 being held virtually November 13-15, 2021.

•

Presented previously disclosed data from ongoing RP-A501 Phase 1 trial in Danon Disease at HFSA. The

late-breaking oral presentation at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021 overviewed data from the low-dose (6.7e13 vg/kg) adult cohort which demonstrated RP-A501 was well tolerated and showed progressive and durable clinical benefit.

Leukocyte Adhesion Deficiency-I (LAD-I):

•

Presented positive interim data updates from RP-L201 LAD-I trial at ESGCT. The oral

presentation included data from the initial seven patients with severe LAD-I who were treated with RP-L201 in the Phase 1/2 trial. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related

serious adverse events. Preliminary efficacy was evident in all seven patients, including two patients with at least 12 months of follow-up. All seven patients demonstrated durable neutrophil CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent with reversal of the severe LAD-I phenotype. Peripheral blood vector copy number (VCN) levels have been stable and in the 0.5 – 2.5 copy per genome range. No patients have had LAD-I related infections requiring hospitalization subsequent to hematopoietic reconstitution post RP-L201. Additional clinical data are anticipated at the 63rd American Society of Hematology (ASH) Annual Meeting being held December 11-14, 2021.

Fanconi Anemia (FA):

•

Presented previously disclosed clinical data from RP-L102 Fanconi Anemia (FA) program at ESGCT. The

oral presentation included data for nine pediatric patients treated with RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate, in the ongoing clinical trials. RP-L102 demonstrated a highly favorable safety profile with all subjects being treated without conditioning and with no sign of dysplasia or other concerning features. RP-L102 showed evidence of pre

EX-99.1

2 brhc10027803_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Second Quarter 2021 Financial Results and Highlights Recent Progress

—  Working with FDA on Changes to Clinical Trial Protocol in Danon Disease Trial; Rocket Anticipates Trial Will Resume in 3Q—

—  Increasing and Durable Benefit Observed in Low Dose Cohort (6.7e13 dose) in Danon Disease; Removing Higher Dose Cohort (1.1e14 dose) From Future Dosing Plans; Full Clinical Update Expected in 4Q –

—  Positive Results from FA, LAD-I, PKD Trials Presented at 24th ASGCT Annual Meeting Show Preliminary Evidence of Activity and Favorable Tolerability —

—  Clinical Updates in FA, LAD-I, PKD and IMO Also Expected in 4Q —

— Ending Balance Sheet with $426.8 Million in Cash; Cash Runway Expected into 2H'23 —

— Conference Call to Be Held at 4:30 p.m. ET Today—

CRANBURY, NJ – Aug. 9, 2021 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter ending June 30, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones.

“We are grateful to the FDA for its support and for working with us on our Danon program toward resuming our trial, which we believe will occur in the third quarter,” said Gaurav Shah, M.D., chief executive officer of Rocket Pharma. “Further, we have observed durable clinical benefit in the low dose adult cohort, which we believe is supportive of its potential as a viable Phase 2 dose. As of July 2021, we see improvement in two of three low dose patients in NYHA class. In these two patients, we also observed substantial improvement of a key marker of heart failure, BNP, which decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other as well as improvement in cardiac output by 35 percent in one patient and 62 percent in the other as measured by invasive hemodynamics. The third patient has demonstrated stabilization of NYHA class and BNP. Given the positive benefit/risk profile in the low dose, and additionally to mitigate safety concerns observed at the higher dose, in agreement with FDA we will no longer treat Danon patients with the higher dose (1.1e14). With our full focus on the low dose, we look forward to progressing our trial on behalf of Danon patients devastated by this disease.”

Dr. Shah continued, “We also presented positive data across three of our lentiviral-based gene therapy programs at ASGCT in May, which we believe support the growing potential of these programs to treat Fanconi Anemia (FA), LAD-1 and PKD patients. Based on these results, we continue our momentum toward advancing these programs. In the case of our FA program, two recent publications reinforce the natural history, clinical design and methods being utilized in our Phase 1/2 FA trial of RP-L102. We look forward to providing updates on all five of our programs in the fourth quarter of 2021.”

Dr. Shah added, “Finally, and importantly, we are deeply saddened that the first patient dosed in our Phase 1 IMO trial, has passed way from pulmonary hemorrhage related to thrombocytopenia following conditioning therapy and also related to underlying osteopetrosis. This event was considered likely not related to RP-L401 gene therapy. Consistent with the trial protocol, enrollment has been temporarily paused pending a comprehensive evaluation in collaboration with the Independent Data Monitoring Committee, which will include a review of the conditioning regimen and other potential safety measures to mitigate the impact of underlying disease on treatment. This outcome underscores the need to find cures for this devastating disease and has furthered our commitment and dedication to patients and our mission to develop curative gene therapies for rare disease.”

Key Pipeline and Operational Updates

Danon Disease:

•

Progressed toward agreement with FDA on changes to the Phase 1 clinical trial protocol in Danon Disease. Rocket anticipates trial may resume this quarter. The U.S. Food and Drug Administration (FDA) had previously requested Rocket pause patient dosing in the Phase 1 clinical trial of RP-A501 and modify the protocol and other supporting documents with revised guidelines for patient selection and management. No new drug-related safety events were observed in the low- or higher-dose adult cohorts; the previously disclosed SAE of thrombotic microangiopathy, which has since resolved, was reclassified as a SUSAR. All follow-up study activities continue. Longer-term results from the low (6.7e13) and higher dose (1.1e14) adult cohorts will be reported in the fourth quarter. In agreement with FDA, Rocket will no longer continue dosing patients at the higher dose (1.1e14).

Fanconi Anemia (FA):

•

Presented positive clinical updates from RP-L102 Fanconi Anemia (FA)

program at ASGCT. Preliminary resu

EX-99.1

2 brhc10024381_ex99-1.htm

EXHIBIT 99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports First Quarter 2021 Financial Results and Highlights Recent Progress

—RP-A501 Phase 1 Trial for the Treatment of Danon Disease Placed on Clinical Hold To Address FDA Request For Additional Risk Mitigation Methods in Protocol; No New Safety Events Observed; Anticipate Delay in Enrollment by One Quarter —

—Updated Low Dose Results in RP-A501 Treatment for Danon Disease Demonstrate Stabilization or Improvements in Key Clinical Outcomes Including 6MWT and NYHA Class; Full Data to be presented in 4Q—

— Reported Hemoglobin Increases of Nearly 2x from Baseline Sustained Up to 6-Months Post RP-L301 Treatment in PKD —

— Announced Sustained Efficacy, Durability and Safety Up to 18-Months Post RP-L201 Treatment in LAD-I —

— Advanced Fifth Gene Therapy Program in Infantile Malignant Osteopetrosis into Patient Dosing —

—Updated Results from FA, LAD-I, PKD Trials to be Presented at 24th ASGCT Virtual Annual Meeting —

— Redemption of 2022 Convertible Notes Strengthens Balance Sheet; $466.4 Million in Cash Solidifies Runway into 2H 2023—

— Webcast and Conference Call Regarding Danon Program to be Held at 4:30 PM EDT Today —

CRANBURY, NJ – May 10, 2021 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the quarter ending March 31, 2021 and updates on the Company's key pipeline developments, business operations, and upcoming milestones.

“I am pleased that we began 2021 with sustained momentum across clinical, regulatory and manufacturing activities as reflected in our exciting PKD and LAD-I results, advancement of the pipeline toward regulatory submissions, and continued progress of our GMP manufacturing facility,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket. “New data readouts and designations from regulatory agencies continue to validate and reinforce the transformative potential of our gene therapies for patients suffering from rare diseases with no current drug treatments. We continued to strengthen our world class operations and capabilities as we added to our leadership team and fortified our balance sheet.”

Dr. Shah continued, “We are encouraged by the positive low dose RP-A501 Phase 1 trial results in Danon Disease disclosed today. We anticipate a one quarter delay in enrollment to address FDA requests for risk mitigation methods in our protocol. We have successfully treated five patients in the low- and high-dose adult cohorts. Today, we announce that new longer-term low-dose data demonstrate durable expression and ongoing improvements in biomarkers, and evidence of a positive risk/benefit profile out to 18-months. In the two patients followed to 18-months, we see improvement in 6MWT in one patient and stabilization/improvement the other, and improvement in NYHA Class from II to I in one patient with stabilization in the other. These updated results increase our confidence in the low dose as a potentially viable dose for patients with Danon Disease. Safety is our top priority as we progress our gene therapy trials. We are diligently working with the agency prior to initiating our low-dose pediatric cohort, which we believe has high potential as a Phase 2 dose given the durable results noted in adult patients. “

Dr. Shah continued, “The balance of the year is shaping up to be a productive period, starting next week with updates from our FA, LAD-I, and PKD programs at ASGCT, followed by initial IMO data in the third quarter of 2021. We are focused on strong clinical and operational execution as we continue our development of total cures at the genetic level.”

Key Pipeline and Operational Updates

•

RP-A501 Danon Disease program paused for additional risk mitigation. No

new drug-related safety events have been observed in the low- or high-dose adult cohorts of the Phase 1 trial. The U.S. Food and Drug Administration (FDA) has requested the Company to pause patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management. All follow-up study activities will continue and no additional data are requested. Rocket is continuing its dialogue with the agency to ensure safety measures are updated and harmonized adequately and anticipates additional patient treatment by 3Q2021.

•

Low Dose (6.7x1013) RP-A501 Treatment Demonstrates Durable Expression and Improvements in Biomarkers. New Data Include:

o

Patient 1002: 78% IHC (revised from previous 67% due to improved technique), BNP continues to decrease to 200 (baseline 943)

o

Functional outcomes

◾

Patient 1001: NYHA Class stable (II), 6-minute walk test (MWT) improved at 18-months

◾

Patient 1002: NYHA Class Improved (II to I), 6MWT stable to modestly improved at 18 months

◾

Patient 1005: NYHA Class Stable (II), 6MWT pending at 12

EX-99.1

3 d454697dex991.htm

EX-99.1

EX-99.1

Exhibit 99.1

Rocket Pharmaceuticals Reports Full Year 2017 Financial Results and Operational Highlights

– Enters 2018 as a Public Company with Solid Financial Position Following Successful Completion of Merger with Inotek Pharmaceuticals and Subsequent Follow-on Offering –

– Multiple Milestones Expected in 2018, with Additional Data in Fanconi Anemia, Disclosure of AAV Program, and Several Programs Advancing Toward the Clinic –

NEW YORK—March 6, 2018—Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today reported financial results and operational highlights for the full year ended December 31, 2017.

“2017 was an important year for Rocket. We advanced our pipeline of gene therapy programs focused on life-threatening and devastating pediatric diseases, and we implemented strategic initiatives that have strengthened our organization,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “We believe Rocket’s multi-platform development approach uniquely positions us to aggressively pursue a broad range of best-in-class indications, enabling first-mover advantage.”

“With the successful closing of our merger with Inotek Pharmaceuticals and recent public offering in January, we enter 2018 with a solid financial position to accomplish our key clinical and operational goals,” continued Dr. Shah. “In addition to more clinical data on our lead program in Fanconi Anemia (FA), we plan to announce the disease indication for our adeno-associated viral vector (AAV) gene therapy program in the second half of this year. We also expect to file Investigational Medicinal Product Dossier (IMPD) applications for our Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) programs, as well as an Investigational New Drug (IND) application for our AAV program over the next 12 months. With multiple programs moving forward in parallel, we believe we have the key elements in place to build a transformative, multi-platform gene therapy company delivering sustained, long-term growth for our shareholders.”

Recent Pipeline and Corporate Updates

•

Promising preliminary patient data reported for RP-L102, a Phase 1/2 lentiviral vector (LVV)-based gene therapy for FA. At the European Society of Gene and Cell Therapy’s Annual Meeting in October 2017, Rocket’s collaboration partners at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain presented interim results from the ongoing Phase 1/2 trial of RP-L102 in FA. Data presented in FA patients, without the use of conditioning, showed promising in vivo engraftment and stabilization of blood counts despite declining counts in the months to years preceding therapy.

•

Studies of FA gene therapy product, RP-L101, utilizing new gene modified cell product manufacturing process underway. At the American Society of Hematology Annual Meeting in December 2017, Rocket’s collaboration partners at the Fred Hutchinson Cancer Research Center presented data from a pilot clinical trial assessing the feasibility of a novel lineage depletion protocol for hematopoietic stem cell (HSC) enrichment and subsequent transduction with the LVV-based gene therapy, RP-L101. Collection, lentiviral transduction and re-infusion of gene

modified cells was shown to effectively produce long-term engraftment in mice. Patient treatment with this novel protocol is underway.

•

Three additional LVV-based gene therapy pipeline programs in rare bone marrow disorders advance towards the clinic. LAD-I and PKD programs continue to move forward in preclinical IND-enabling studies. An additional program targeting Infantile Malignant Osteopetrosis (IMO) is also in preclinical development. Similar to FA, these diseases can lead to serious complications and have very limited long-term safe and effective treatment options.

•

Rocket commenced trading on the NASDAQ Global Market under the symbol “RCKT”, following the successful closing of its with merger with Inotek Pharmaceuticals Corporation (Inotek) in January. In conjunction with the completion of the merger, the Company announced its executive management team will be led by Dr. Shah and will consist of: Jonathan Schwartz, M.D., Chief Medical Officer, and Kinnari Patel, Pharm.D., MBA, Chief Operating Officer.

•

Rocket completed an oversubscribed underwritten public offering of 6,325,000 shares of its common stock in January 2018. Net proceeds to Rocket from the offering were approximately $78.8 million after deducting underwriting discounts and commissions.

Anticipated Milestones

•

Updated FA patient data (2Q18)

•

Preclinical data and disclosure of the AAV-based gene therapy program (2H18)

•

Target rolling IMPD filing for the LAD-I program (4Q18)

•

Target IND filing for the AAV-based program (next 12 months)

•

Target rolling IMPD filing for the PKD program (next 12 months)

Full Y

8-K 1 d434045d8k.htm 8-K

8-K

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT

REPORT

Pursuant to Section 13 or 15(d)

of The Securities Exchange Act of 1934

Date of Report (Date of Earliest Event Reported): August 3, 2017

Inotek Pharmaceuticals Corporation

(Exact name of registrant as specified in its charter)

DELAWARE

001-36829

04-3475813

(State or other jurisdiction

of incorporation)

(Commission

File Number)

(I.R.S. Employer Identification No.)

91 Hartwell Avenue

Lexington, MA

02421

(Address of principal executive offices)

(Zip Code)

Registrant’s telephone number, including area code (781) 676-2100

Not Applicable

(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 or Rule 12b-2 of the Securities Exchange Act of 1934.

Emerging growth company     ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.     ☒

Item 2.02 Results of Operations and Financial Condition

On August 3, 2017, Inotek Pharmaceuticals Corporation announced its financial results for the quarter ended June 30, 2017. A copy of the press release is being furnished as Exhibit 99.1 to this Report on Form 8-K.

The information in this Report on Form 8-K and Exhibit 99.1 attached hereto is intended to be furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act, except as expressly set forth by specific reference in such filing.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.

Description

99.1

Press release issued by Inotek Pharmaceuticals Corporation on August 3, 2017, furnished herewith.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

Date: August 3, 2017

INOTEK PHARMACEUTICALS CORPORATION

By:

/s/ Dale Ritter

Dale Ritter

Vice President —Finance

EXHIBIT INDEX

Exhibit No.

Description

99.1

Press release issued by Inotek Pharmaceuticals Corporation on August 3, 2017, furnished herewith.