Rocket Pharmaceuticals, Inc. (RCKT) announced that it has completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease. Danon disease is a rare X-linked inherited disorder caused by mutations in the LAMP2 gene. Per the company, an estimated 15,000 to 30,000 patients suffer from Danon disease in the United States and Europe. Currently, the only available treatment for the disease is cardiac transplantati ...

Shares of Rocket Pharmaceuticals (RCKT) have plunged 32.9% in the past six months compared with the industry's decline of 3.4%. The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. In June 2024, the FDA issued a complete response letter (CRL) to Rocket's biologics license application (BLA) seeking approval for Kresladi to treat LAD-I. Per the CRL, the FDA sought "limited" add ...

Rocket Pharmaceuticals (RCKT) announced that the FDA issued a complete response letter (CRL) to its biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. Per the CRL, the FDA sought 'limited' additional information on the therapy's Chemistry Manufacturing and Controls (CMC), which is a part of the BLA submission. The goal of CMC is to ensure that every batc ...

Rocket Pharmaceuticals' experimental treatment for a rare immune disorder in children was delayed again by the Food and Drug Administration (FDA). Regulators sent a Complete Response Letter to the company, asking for more information on Kresladi. The FDA was initially expected to grant approval in March, but delayed it for three months to complete its review. Rocket Pharmaceuticals (RCKT) shares declined in intraday trading Friday after the biopharmaceutical firm announced that the Food and Drug Administrat ...

In the reported quarter, general and administrative (G&A) expenses rose 40% year over year to $22.1 million. The upside was driven by an increase in the company's commercial preparation expenses in anticipation of a potential FDA approval for Kresladi in severe LAD-I. A month has gone by since the last earnings report for Rocket Pharmaceuticals (RCKT) . Shares have lost about 4.8% in that time frame, underperforming the S&P 500. Will the recent negative trend continue leading up to its next earnings release ...

Regulatory Approvals and Clinical Trials - The FDA accepted the Biologics License Application for RP-L201 for severe LAD-I, with regulatory filings for FA anticipated in 2024[102]. - The company has initiated a Phase 1 study for Plakophilin-2 Arrhythmogenic Cardiomyopathy after receiving FDA clearance[104]. - The global Phase 2 pivotal trial for RP-A501 has been aligned with the FDA, targeting 12 patients with a dose level of 6.7 x 10^13 GC/kg[125]. - The company received regenerative medicine advanced therapy designation from the FDA and priority medicines designation from the EMA for RP-A501 in 2023[127]. - A BLA filing for RP-L201 was accepted by the FDA with priority review in October 2023, with an initial Prescription Drug User Fee Act date of March 31, 2024[154]. - The Phase 1/2 trial of RP-L201 showed 100% overall survival at 12 months post-infusion for all nine LAD-I patients, with significant reductions in hospitalizations and infections[152]. Financial Performance and Funding - The company has sold 4.2 million shares under the at-the-market offering program for gross proceeds of $65.8 million, resulting in net proceeds of $63.8 million[104]. - The company raised approximately $1.0 billion from investors through equity and convertible debt financing from inception through March 31, 2024[165]. - The company has not generated any revenue from product sales to date and does not expect to do so in the near future[166]. - The net loss for the three months ended March 31, 2024, was $62.1 million, compared to a net loss of $58.3 million for the same period in 2023[180]. - Total operating expenses increased by $5.2 million to $67.4 million for the three months ended March 31, 2024, compared to $62.2 million in the same period of 2023[180]. - Cash used in operating activities was $56.9 million for the three months ended March 31, 2024, compared to $57.6 million for the same period in 2023[188]. - The company had an accumulated deficit of $1.02 billion as of March 31, 2024[186]. - The company expects R&D expenses to increase for the foreseeable future as it continues to invest in product candidates[172]. Research and Development - RP-A501 is in clinical trials for Danon Disease, with an estimated prevalence of 15,000 to 30,000 patients in the U.S. and EU[111]. - In the Phase 1 trial of RP-A501, a low-dose cohort showed a 98% reduction in high sensitivity troponin I and an 83% reduction in BNP after 36 months[119]. - The company has produced 2 cGMP RP-A501 batches with superior specifications compared to Phase I material, potentially optimizing the safety profile[121]. - The company is focusing on a low dose of 6.7e13 gc/kg for RP-A501 to mitigate safety concerns observed in higher doses[115]. - The NYHA Class assessment indicated that patients in the low-dose cohort exhibited improvements, with some achieving Class I status[116]. - RP-A501 showed durable treatment activity with improvements in biomarkers and NYHA class in pediatric and adult patients over follow-up periods of 6 to 36 months[124]. - The ongoing Phase 2 study of RP-L102 has treated 14 patients, achieving phenotypic correction in at least 6 of 10 evaluable patients with ≥12 months of follow-up[145]. - The primary endpoint for the RP-L102 study is resistance to mitomycin-C in bone marrow stem cells, with a threshold of 10% for marketing application support[144]. - As of April 17, 2023, RP-L102 demonstrated sustained genetic correction in 8 of 12 evaluable patients and comprehensive phenotypic correction in 7 of 12 evaluable patients with ≥12 months of follow-up[147]. - The safety profile of RP-L102 remains highly favorable, with no signs of bone marrow dysplasia or insertional mutagenesis observed[147]. - RP-L301 has shown robust and sustained efficacy in adult patients, including hemoglobin normalization and transfusion independence, with no serious adverse events reported[158]. - The company is initiating a 10-patient, single-arm Phase 2 pivotal trial for RP-L301, with a primary endpoint of ≥1.5 point hemoglobin improvement at 12 months[161]. - The manufacturing facility in Cranbury, New Jersey, has been scaled up to produce AAV drug products for the Phase 2 pivotal study[162]. - The BAG3-DCM program aims to address a patient population of approximately 30,000 individuals in the U.S. with no current therapies targeting the underlying cause[135]. - The company completed the acquisition of Renovacor, enhancing its gene therapy capabilities for BAG3-DCM[137]. - The prevalence of PKP2-ACM is estimated at 50,000 patients in the U.S. and EU, highlighting a significant unmet medical need[130]. - The company has initiated a multi-center Phase 1 study for RP-A601, targeting PKP2-ACM patients with a starting dose of 8 x 10^13 GC/kg[133]. Cash and Investments - The company had $330.3 million in cash, cash equivalents, and investments as of March 31, 2024, which is expected to fund operations into 2026[186]. - Cash provided by investing activities was $35.0 million for the three months ended March 31, 2024, primarily from proceeds of $101.0 million from the maturities of investments[191]. - As of March 31, 2024, the company’s cash, cash equivalents, and marketable securities are primarily invested in U.S. treasury securities and corporate bonds, classified as available-for-sale securities[196]. - If market interest rates increase by 100 basis points, the net fair value of the company's interest-sensitive marketable securities would hypothetically decline by $1.3 million[197]. - The company maintains significant amounts of cash and marketable securities exceeding federally insured limits, which poses potential risks of loss due to financial institution instability[197]. Internal Controls and Legal Matters - The company's management evaluated the effectiveness of disclosure controls and procedures as of March 31, 2024, concluding they were effective at a reasonable assurance level[199]. - There were no changes in internal control over financial reporting during the reporting period that materially affected the company's internal controls[202]. - The company does not believe it is party to any legal claims that would materially adversely affect its business, although litigation can still impact due to costs and resource diversion[204]. Expenses - R&D expenses decreased by $1.1 million to $45.2 million for the three months ended March 31, 2024, compared to $46.4 million in the same period of 2023[181]. - General and administrative expenses increased by $6.3 million to $22.1 million for the three months ended March 31, 2024, compared to $15.8 million in the same period of 2023[182]. - Other income increased by $1.5 million to $5.3 million for the three months ended March 31, 2024, compared to $3.9 million in the same period of 2023[183].

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 Of icer, bringing seasoned leadership experience in commercial-s ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR □ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Its Charter) (Registrant's Telephone Number, including Ar ...

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; exp ...

Form 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Commission File Number: 001-36829 Rocket Pharmaceuticals, Inc. (Exact name of registrant as specified in its charter) (State or other jurisdicti ...