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

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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.

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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.

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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.

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

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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

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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.

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

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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.

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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.

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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

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

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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.

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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

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

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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.

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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