Rocket Pharma secures FDA agreement on Danon trial
Rhea-AI Filing Summary
Rocket Pharmaceuticals, Inc. (RCKT) reported that the U.S. Food and Drug Administration agreed on key elements for the global pivotal Phase 2 trial of gene therapy RP-A501 in Danon disease, including a 12-patient pivotal efficacy population and use of a recalibrated dose.
The FDA reviewed clinical data from the first three male patients treated under a modified protocol at 3.8 × 10¹³ GC/kg, noting no clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome, and confirmed continued enrollment and dosing at this dose. These first three patients count toward the 12-patient pivotal population, leaving nine to enroll, and Rocket expects to complete dosing of the remaining patients by mid-2027. The primary efficacy assessment will occur at 12 months using co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index, which are intended to support a potential accelerated approval pathway.
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8-K Event Classification
2 items: 7.01, 9.01
2 items
Item 7.01
Regulation FD Disclosure
Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Key Figures
Pivotal efficacy population: 12 patients
Recalibrated RP-A501 dose: 3.8 × 10¹³ GC/kg
Patients already treated under modified protocol: 3 patients
+3 more
6 metrics
Pivotal efficacy population
12 patients
Total male patients to be treated with RP-A501 at the recalibrated dose in the pivotal Phase 2 trial
Recalibrated RP-A501 dose
3.8 × 10¹³ GC/kg
Genome copies per kilogram used in the pivotal study with commercial-grade product
Patients already treated under modified protocol
3 patients
Initial patients at the recalibrated dose who count toward the 12-patient pivotal population
Remaining patients to enroll
9 patients
Additional male patients to be enrolled and treated to complete the pivotal population
Primary efficacy assessment timing
12 months
Timepoint for evaluating co-primary endpoints after dosing in the pivotal trial
Investor webinar date and time
October 6, 2026 at 4:30 p.m. ET
Scheduled virtual investor webinar on the Danon disease program
Key Terms
accelerated approval, co-primary endpoints, thrombotic microangiopathy, capillary leak syndrome, +2 more
6 terms
accelerated approval regulatory
"These endpoints are intended to support a potential accelerated approval pathway"
Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.
co-primary endpoints medical
"established 12-month co-primary endpoints intended to support potential accelerated approval"
Co-primary endpoints are two or more key outcomes that a clinical study must meet simultaneously to be considered successful, like needing both improved symptoms and a marker of disease reduction. For investors, they matter because meeting multiple agreed goals strengthens the evidence that a treatment works and can influence regulatory approval, market access and commercial potential—similar to a product needing to pass both safety and performance checks before launch.
thrombotic microangiopathy medical
"No clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome"
Thrombotic microangiopathy is a medical condition where tiny blood clots form inside the smallest blood vessels, blocking flow and damaging organs such as the kidneys and brain. Investors should care because TMA can be a serious safety signal in clinical trials or post-market reports, trigger regulatory action or product recalls, and create liability or revenue risk for companies developing or selling related therapies—think of it as microscopic plumbing clogs that can shut down vital systems.
capillary leak syndrome medical
"No clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome"
A rare medical condition in which fluids and proteins rapidly escape from small blood vessels into surrounding tissues, causing sudden swelling, low blood pressure and reduced blood flow to organs — like a pipe developing leaks so the contents pool where they shouldn’t. Investors pay attention because it can appear as a serious side effect of drugs or treatments, affecting clinical trial outcomes, regulatory decisions, product labeling, sales prospects and legal risk.
immunomodulatory regimen medical
"an optimized immunomodulatory regimen comprised of rituximab, sirolimus and corticosteroids"
vector biodistribution medical
"demonstrated comparable vector biodistribution"
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What FDA alignment did RCKT announce for the RP-A501 Danon disease trial?
Rocket announced FDA alignment on continued enrollment and dosing and key elements of the pivotal Phase 2 RP-A501 trial in Danon disease, including a 12-patient pivotal population, a recalibrated dose, and established 12‑month co-primary endpoints intended to support a potential accelerated approval pathway.
How many patients will be included in Rocket Pharmaceuticals’ pivotal Phase 2 RP-A501 trial?
The pivotal efficacy population will include 12 male patients treated with RP-A501 at the recalibrated dose of 3.8 × 10¹³ GC/kg. The first three patients already treated under the modified protocol count toward this total, leaving nine additional patients to be enrolled and treated.
What safety findings did the FDA review for RP-A501 in the RCKT trial?
The FDA reviewed clinical data from the first three patients treated under the modified RP-A501 protocol. All three completed at least four weeks of follow-up and were discharged after observation, with no clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome reported.
What are the co-primary endpoints for Rocket Pharmaceuticals’ RP-A501 pivotal trial?
The 12‑month primary assessment will use co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index. These co-primary endpoints are intended to support a potential accelerated approval pathway for RP-A501.
When does Rocket Pharmaceuticals expect to complete dosing in the RP-A501 pivotal trial?
Rocket expects to complete dosing of the remaining nine patients by mid‑2027. The 12‑month primary efficacy assessment will then be conducted using the established co-primary endpoints related to myocardial LAMP2 protein expression and left ventricular mass index.
What additional update did RCKT schedule on its Danon disease program?
Rocket plans a virtual investor webinar providing a comprehensive Danon disease program update on October 6, 2026, at 4:30 p.m. ET. Webcast access details will be available in the Investors section of the company’s website.
AI-generated analysis. How Rhea-AI works. Not financial advice.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 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): September 15, 2026
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this
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| Item 7.01. |
Regulation FD Disclosure.
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On September 15, 2026, Rocket Pharmaceuticals, Inc. (the “Company”) issued a press release announcing alignment with the U.S. Food and Drug
Administration on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease, including the recalibrated dose, 12-patient pivotal population and established 12-month co-primary
endpoints.
A copy of the press release is included as Exhibit 99.1 hereto and is incorporated herein by reference.
The information under this Item 7.01, including Exhibit 99.1, shall not be deemed “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, and shall not be deemed to be incorporated by reference into the filings of the Company under the Securities Act or the Exchange Act, except as shall be
expressly set forth by specific reference in such filing.
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Financial Statements and Exhibits.
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Exhibits.
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99.1
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Press Release of Rocket Pharmaceuticals, Inc. dated September 15, 2026.
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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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.
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Rocket Pharmaceuticals, Inc.
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Date: September 15, 2026
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By:
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/s/ Martin Wilson
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Martin Wilson
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General Counsel and Chief Corporate Officer
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Exhibit 99.1
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Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete
Pivotal Phase 2 Trial of RP-A501 in Danon Disease
FDA confirms pivotal efficacy population of 12 patients, including the first three treated under the modified protocol
FDA review of initial safety data supports continued enrollment and dosing under modified protocol
FDA confirms established 12-month co-primary endpoints intended to support
potential accelerated approval
Comprehensive Danon disease program webinar for
October 6, 2026, at 4:30 p.m. ET
CRANBURY, N.J. – September 15, 2026 – Rocket Pharmaceuticals, Inc.
(NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and life-threatening diseases, focused on inherited cardiovascular disorders, today announced alignment with the U.S. Food and Drug
Administration (FDA) on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease, including the recalibrated dose, 12-patient pivotal population and established 12-month
co-primary endpoints.
The clinical data reviewed by FDA included results from the first three patients treated under the modified protocol. All three completed at least four weeks of follow-up
and had been discharged following protocol-specified observation. No clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome had been observed. Following its review of the available safety data, the FDA confirmed
continued enrollment and dosing under the modified protocol at the recalibrated dose.
The FDA also confirmed that the pivotal study population will include 12 male patients treated with RP-A501 at the recalibrated dose of 3.8 × 10¹³ genome copies per
kilogram (GC/kg) using commercial-grade product. Importantly, the first three patients treated at the recalibrated dose under the modified protocol count toward the 12-patient pivotal population, leaving nine additional patients to be enrolled and
treated. Rocket expects to complete dosing of the remaining patients by mid-2027.
The primary assessment will occur at 12 months using the previously established co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from
baseline in left ventricular mass index. These endpoints are intended to support a potential accelerated approval pathway.
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“FDA’s confirmation of the pivotal efficacy framework marks an important milestone for
RP-A501 and the Danon disease community,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “With the first three patients counting toward the 12-patient pivotal population and the established co-primary endpoints
preserved, we now have a clear and actionable path to complete the pivotal study. The initial clinical experience at the recalibrated dose and supportive product-bridging data further reinforce the path forward.”
The modified protocol incorporates a recalibrated RP-A501 dose and an optimized immunomodulatory regimen comprised of rituximab, sirolimus and corticosteroids, together
with enhanced eligibility criteria, safety monitoring and risk-mitigation measures. The recalibrated dose was selected based on analytical characterization of the Phase 2 commercial-grade product, including its full-particle content, and is
supported by nonclinical bridging data.
Nonclinical bridging data further support the pharmacologic activity of the recalibrated dose. In a Danon disease mouse model, administration of Phase 2 material at 3.8 ×
10¹³ GC/kg resulted in cardiac LAMP2B protein expression comparable to that observed at a higher dose and demonstrated comparable vector biodistribution.
Rocket plans to provide a comprehensive update on the Danon disease program during a virtual investor webinar on Tuesday, October 6, 2026, at 4:30 p.m. ET. Additional
information, including webcast access details, will be available in the Investors section of the Company’s website.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated commercial-stage biotechnology company developing genetic medicines for rare and
life-threatening diseases, with a strategic focus on inherited cardiovascular disorders and additional programs in hematology and immunology. Rocket's cardiovascular portfolio includes three clinical-stage gene therapy programs targeting
hypertrophic, arrhythmogenic, and dilated cardiomyopathies, together representing one of the broadest pipelines focused on inherited heart disease. The Company’s integrated platform combines proprietary adeno-associated virus (AAV) manufacturing
capabilities, extensive clinical experience in cardiac gene therapy, and long-term safety and efficacy data supporting advancement across its cardiovascular portfolio.
For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.
Rocket Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as
assumptions that, if they do not materialize or prove incorrect, could cause results to differ materially from those expressed or implied by such forward-looking statements. Rocket makes such forward-looking statements pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical fact contained in this release are forward-looking statements.
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These forward-looking statements include, but are not limited to, statements concerning the safety and potential effectiveness of RP-A501; the anticipated enrollment and
dosing of additional patients in the Phase 2 trial; the expected timing for completing dosing and conducting the 12-month primary efficacy assessment; the potential for the trial’s co-primary endpoints to support an accelerated approval pathway;
future regulatory interactions and submissions; and the timing and content of future clinical data updates.
Although Rocket believes that the expectations reflected in these forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may
differ materially as a result of various important factors, including the results of Rocket’s ongoing and planned clinical trials; Rocket’s ability to enroll and retain a sufficient number of patients and successfully complete clinical studies; the
timing and outcome of regulatory interactions; manufacturing and product-supply considerations; unexpected safety events; and other risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the
year ended December 31, 2025, filed with the Securities and Exchange Commission on February 26, 2026, and in subsequent filings with the SEC, including Rocket’s Quarterly Reports on Form 10-Q.
Accordingly, readers should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no
obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Investors & Media
Meg Dodge
mdodge@rocketpharma.com
Brooke Schuster
bschuster@rocketpharma.com