CAPR

EX-99.1

2 capr-20260813xex99d1.htm

EX-99.1

Exhibit 99.1

Capricor Therapeutics Reports Second Quarter 2026 Financial Results and Provides Corporate Update

●Deramiocel Biologics License Application (BLA) under active FDA review

●HOPE-3 Phase 3 results published in The Lancet; primary endpoint of upper limb function achieved at p=0.029

●Cash, cash equivalents and marketable securities of approximately $238 million as of June 30, 2026

●Conference call and webcast today at 4:30 p.m. ET

SAN DIEGO, Aug. 13, 2026 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced its financial results for the second quarter ended June 30, 2026, and provided a corporate update.

“Our priority is, and always has been, to get Deramiocel to the patients and families living with Duchenne who need it most,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “The Advisory Committee outcome was not the one we hoped for. The indication we requested in 2024 was the treatment of cardiomyopathy in DMD, and that is the question the Committee was asked to vote on, not the HOPE-3 primary endpoint. HOPE-3 was designed and powered to demonstrate efficacy in upper limb function; cardiac function was a key secondary endpoint, measured across all patients enrolled rather than only those with established cardiomyopathy. The full dataset has since been published in The Lancet following extensive and independent peer review, and we continue to believe there is a path to approval for Deramiocel.”

Dr. Marbán continued, “The most powerful part of the Advisory Committee was the open public hearing, where patients, families and clinicians described what this therapy has meant, or could mean, to them. That testimony is on the public record, and it is a reminder of how urgent the unmet need in Duchenne remains. We are continuing to work with the Agency on a path forward.”

Second Quarter 2026 and Recent Highlights

●Deramiocel BLA Under FDA Review: Capricor is continuing to work with the FDA on the review of its BLA. The Company plans to provide a regulatory update on its conference call today and will provide further updates as they become available.

●FDA Advisory Committee Outcome: On July 29, 2026, the Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against on whether available evidence provides substantial evidence of effectiveness of Deramiocel for the treatment of cardiomyopathy in patients with DMD. The Committee was not asked to vote on the HOPE-3 primary endpoint or on overall benefit-risk, and in a separate discussion of upper limb function its feedback was directionally supportive of the HOPE-3 clinical evidence. The recommendation is advisory and non-binding.

●HOPE-3 Results Published in The Lancet: The full HOPE-3 Phase 3 dataset was published in The Lancet in July following extensive and independent peer review. Deramiocel demonstrated a statistically significant slowing of upper limb disease progression as measured by PUL 2.0 (p=0.029), with supportive results across additional functional and cardiac measures, which are characterized as nominally significant under the applicable hierarchical testing procedures. In connection with the peer review, Capricor identified an issue with the statistical model in the clinical study report and reverted to the statistical analysis plan in place prior to unblinding. The only endpoint affected was left ventricular ejection fraction, which yields (p=0.09) and a 1.8 percentage point treatment difference, compared to (p=0.04) and a 2.4 percentage point difference reported as topline data. The pre-specified cardiomyopathy subgroup was unchanged at (p=0.02), nothing else changed in the data or its analysis, and the HOPE-3 primary endpoint was unaffected.

●FDA Bioresearch Monitoring Inspection: As part of the review process, the FDA conducted a BIMO inspection in July 2026 and issued a Form 483 citing one observation. The Company has submitted its responses and is currently awaiting feedback.

●Commercial and Manufacturing Readiness: The Company's GMP manufacturing facility in San Diego is operational and positioned to support an initial commercial launch, if Deramiocel is approved. The second-floor expansion is targeted for full validation and FDA inspection in 2027, as planned. Michael Maurer joined as Chief

Commercial Officer, bringing direct DMD and rare disease experience, and has been judiciously building out the launch organization. Capricor is advancing commercial readiness at a slower pace pending regulatory clarity.

●NS Pharma Dispute: The state court was scheduled to hear Capricor's motion for preliminary injunction on August 10, 2026, ahead of the PDUFA action date. Capricor withdrew the motion, without prejudice, having determined that resolving this contractual dispute in arbitration following the FDA's de

EX-99.1

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EX-99.1

Exhibit 99.1

Capricor Therapeutics Reports First Quarter 2026 Financial Results and Provides Corporate Update

●Deramiocel BLA under active FDA review; PDUFA target action date of August 22, 2026; labeling discussions expected to commence soon

●HOPE-3 Phase 3 trial met its primary endpoint (PUL v2.0; upper limb function) and all Type I error-controlled secondary endpoints

●GMP manufacturing facility fully operational; second-floor expansion well underway

●Chief Commercial Officer with direct DMD commercial experience expected to join the Company in the coming weeks

●Filed suit against Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. seeking rescission of U.S. distribution agreement and preliminary injunction; FDA review and PDUFA date unaffected

●Cash balance of approximately $279 million expected to support operations into Q4 2027

●Eligible for a Priority Review Voucher upon approval; PRV is transferable and monetizable, offering potential non-dilutive capital

●Conference call and webcast today at 4:30 p.m. ET

SAN DIEGO, May 12, 2026 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced its financial results for the first quarter ended March 31, 2026, and provided a corporate update.

"Capricor enters this pivotal moment with important regulatory and clinical momentum as we work toward potential approval of Deramiocel for the treatment of Duchenne muscular dystrophy," said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. "With the FDA review of our BLA underway and a PDUFA target action date of August 22, 2026, our highest priority is execution: working closely with the Agency, preparing for potential launch, and continuing to build the capabilities of a commercial-stage company. We remain focused on ensuring that Deramiocel reaches every eligible patient as quickly as possible, which is why we took necessary legal action against NS Pharma to remove a structural barrier to patient access created by a flawed pricing arrangement and to address our distributor's failure to fulfill its commercial obligations."

“Our thesis is straightforward: a potential first-in-class approval in a defined rare disease population, proprietary in-house manufacturing, a growing pipeline, and a leadership team with the conviction and the capital to execute,” said Dr. Marbán. “We are building something that will create meaningful and durable value for patients, for this company, and for the shareholders who have believed in this mission.”

First Quarter 2026 and Recent Highlights

●Deramiocel BLA Under FDA Review: Capricor's Biologics License Application (BLA) seeking approval of Deramiocel for the treatment of DMD is currently under review by the U.S. Food and Drug Administration. The FDA accepted the Company's Class 2 resubmission as complete, resuming its full review of the BLA, with a PDUFA target action date of August 22, 2026. There has been a significant number of information requests from FDA to Capricor, all of which the Company has been able to address. The Company looks forward to continuing an active dialogue with the FDA and expects labeling discussions to commence soon.

●Additional HOPE-3 Late-Breaking Data Presented at MDA, AAN, and ASGCT (2026): HOPE-3 data were selected as one of only four late-breaking oral presentations at the 2026 MDA Clinical & Scientific Conference. Data were also presented at the 2026 AAN Annual Meeting and the 2026 ASGCT Annual Meeting. Cardiac MRI analyses showed a significant reduction in myocardial fibrosis by late gadolinium enhancement (LGE) versus placebo, a clinically meaningful finding given that fibrosis is cumulative and irreversible. The Duchenne Video Assessment (DVA) "eat 10 bites" measure, a home-based, caregiver-captured assessment of upper limb function and daily living activities, demonstrated statistically significant improvement versus placebo, directly correlated with patient independence and quality of life. The full HOPE-3 dataset has been submitted for publication to a peer-reviewed journal.

●Commercial Launch Preparations Underway: The Company's GMP manufacturing facility in San Diego successfully completed an FDA Pre-License Inspection, with all Form 483 observations addressed. The facility is

operational and positioned to support initial commercial launch. Second-floor expansion adding additional cleanrooms is well underway, scaling to approximately 2,000–2,500 patients per year, roughly 10,000 doses annually, at full capacity, with full facility validation and FDA approval targeted for the first half of 2027. The Company will begin stockpiling commercial doses once guidance on the label is received. Planning is underway across all critical launch functions, including patient support, market access, reimbursement planning, medical affairs and physician education.

CAPRICOR THERAPEUTICS, INC._March 12, 2026 0001133869false00011338692026-03-122026-03-12 ​ ​

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) ​ March 12, 2026

​

CAPRICOR THERAPEUTICS, INC.

(Exact name of Registrant as Specified in its Charter) ​ ​ ​ ​ ​ ​

Delaware ​ ​ ​ 001-34058 ​ ​ ​ 88-0363465

(State or other jurisdiction of incorporation) ​ (Commission File Number) ​ (I.R.S. Employer Identification No.)

​ ​ ​ ​

10865 Road to the Cure, Suite 150, San Diego, California (Address of principal executive offices) ​ ​ ​ 92121 (Zip Code)

​ (858) 727-1755 (Registrant’s telephone number, including area code) ​ 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 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). ​ 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. ◻ ​ Securities registered pursuant to Section 12(b) of the Act: ​ ​ ​

Title of Each Class ​ ​ ​ Trading Symbol(s) ​ ​ ​ Name of Each Exchange on Which Registered

Common Stock, par value $0.001 per share

CAPR

The Nasdaq Capital Market

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Item 2.02 Results of Operations and Financial Condition.

​ On March 12, 2026, Capricor Therapeutics, Inc., a Delaware corporation (the “Company”), issued a press release announcing its financial results for the quarter and full year ended December 31, 2025. A copy of the press release is being furnished herewith as Exhibit 99.1 to this Current Report on Form 8-K. ​ The information under Item 2.02 of this Current Report on Form 8-K and Exhibit 99.1 attached hereto is being furnished and shall not be deemed to be filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be incorporated by reference into any of the Company’s filings under the Exchange Act, unless expressly set forth as being incorporated by reference into such filing. ​ Item 9.01Financial Statements and Exhibits. ​ (d) Exhibits ​

99.1 ​ Press Release, titled “Capricor Therapeutics Reports Fourth Quarter and Full Year 2025 Results and Provides Corporate Update”, dated March 12, 2026.

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104 ​ Cover Page Interactive Data File (formatted as inline XBRL).

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SIGNATURES

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

CAPRICOR THERAPEUTICS, INC.

Date: March 12, 2026 By: /s/ Linda Marbán, Ph.D.

Linda Marbán, Ph.D.

Chief Executive Officer

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