Summary of Rocket Pharmaceuticals FY Conference Call Company Overview - **Company**: Rocket Pharmaceuticals (NasdaqGM:RCKT) - **Industry**: Gene Therapy - **Focus**: Two platforms - AAV-based therapies for cardiovascular diseases and ex vivo Lenti-based therapies for hematology [2][3] Core Points and Arguments Clinical Programs - **Clinical Programs**: Three hematology programs (LAD-I, Fanconi anemia, pyruvate kinase deficiency) and three cardiovascular programs (Danon disease, PKP2, BAG3) [2] - **AAV Portfolio Focus**: Increased focus on AAV programs due to high unmet need and potential for value creation [2][3] Danon Disease Program - **Trial Resumption**: The hold on the Danon program was lifted rapidly; trial will restart in the first half of 2026 with a staggered dosing approach [5][6] - **Patient Safety**: Previous safety events led to the removal of complement inhibitors from the trial design [5][6] - **Regulatory Alignment**: Ongoing positive interactions with the FDA regarding trial design and patient count [12][13] PKP2 Program - **Trial Design Discussions**: Ongoing discussions with the FDA about trial design; emphasis on a robust design that demonstrates clear benefit-risk [19][20] - **Endpoints Consideration**: Potential for composite endpoints due to the heterogeneity of the disease [21][22] BAG3 Program - **Clinical Entry**: The BAG3 program is set to enter the clinic next year, focusing on dilated cardiomyopathy (DCM) with traditional endpoints [25][26] - **Predictability**: DCM is better characterized, allowing for more predictable outcomes [25] Lenti-based Therapies - **Focus Shift**: A strategic decision to focus on AAV programs while seeking partnerships for Lenti-based therapies like Fanconi anemia [30][31] - **PRV Eligibility**: All Lenti programs (LAD, Fanconi, PKD, Danon) are eligible for Priority Review Vouchers (PRV), providing potential non-dilutive cash sources [34] CMC and Regulatory Updates - **CMC Findings**: Previous CMC findings have been addressed, and a resubmission to the FDA has been made, with hopes for approval by the PDUFA date of March 28 [35] Other Important Insights - **Community Engagement**: Strong commitment to patient advocacy and community engagement, especially following safety events [16][17] - **Market Opportunity**: The company is preparing for a comprehensive update on trial progress and market opportunity once clarity is achieved [10][15] This summary encapsulates the key points discussed during the conference call, highlighting Rocket Pharmaceuticals' strategic focus, clinical program updates, regulatory interactions, and community engagement efforts.

Core Points - U.S. stock futures are lower, with Dow futures down approximately 200 points [1] - Helmerich and Payne Inc reported a fourth-quarter loss of 1 cent per share, with quarterly sales of $1.012 billion, exceeding analyst expectations of $973.678 million [1] - Helmerich and Payne shares fell 7% to $25.69 in pre-market trading [1] Company Movements - Alpha Technology Group Ltd shares decreased by 19% to $17.50 after a significant surge of 132% on Monday [4] - Invivyd Inc shares fell 13.4% to $2.46 following the announcement of a $125 million public offering [4] - Nice Ltd shares dropped 7.3% to $112.00 after a 9% decline on the previous day [4] - H World Group Ltd shares fell 6.7% to $41.95 despite reporting better-than-expected third-quarter results [4] - Fulcrum Therapeutics Inc shares decreased by 6.6% to $9.01 after a 12% gain on Monday [4] - Rocket Pharmaceuticals Inc shares declined 6% to $2.86 [4] - OBOOK Holdings Inc shares fell 5.6% to $7.53 [4] - Iperionx Ltd shares decreased by 5% to $33.00 [4] - Home Depot Inc shares slipped 2% to $351.01 after reporting worse-than-expected third-quarter adjusted EPS and lowering FY25 adjusted EPS outlook [4]

Corporate Strategy and Financial Overview - Rocket Pharmaceuticals reported a strategic corporate reorganization aimed at maximizing near-term value and extending operational runway, which includes a 30% workforce reduction and a projected 25% decrease in 12-month operating expenses [123]. - The company has raised approximately $1.2 billion from investors since inception to fund its operations and R&D activities [188]. - The company has not generated any revenue from product sales to date and does not expect to do so in the near future [189]. - The company incurred restructuring expenses of approximately $3.3 million in June 2025 due to workforce reduction [200]. - The net loss for the three months ended September 30, 2025, was $50.3 million, a decrease of $16.4 million from a net loss of $66.7 million in 2024 [206]. - The company incurred net losses of $180.6 million for the nine months ended September 30, 2025, and $258.7 million for the year ended December 31, 2024, with an accumulated deficit of $1.40 billion as of September 30, 2025 [219]. - Cash used in operating activities was $155.2 million for the nine months ended September 30, 2025, compared to $162.8 million for the same period in 2024 [221]. - The company had $222.8 million in cash, cash equivalents, and investments as of September 30, 2025, down from $372.3 million as of December 31, 2024 [230]. - The company is dependent on generating cash from operating activities or raising additional capital to finance operations, which may lead to stockholder dilution [220]. - The company has experienced negative cash flows from operations each year since inception, indicating ongoing financial challenges [219]. Research and Development Focus - The company is focusing on advancing its AAV cardiovascular gene therapy platform and responding to the CRL for KRESLADI™, targeting an exceptionally small patient population [123]. - The strategic focus is on developing potentially curative first-in-class gene therapies for rare diseases with significant unmet medical needs [122]. - The company aims to submit BLAs for certain clinical programs and expand its gene therapy platform to additional indications compatible with its technologies [129]. - RP-A501 holds multiple designations including FDA RMAT, Fast Track, Rare Pediatric, and Orphan Drug in the U.S., along with ATMP and PRIME designations in the EU [134]. - The company has global commercialization and development rights to its product candidates under royalty-bearing license agreements [132]. - The company is no longer allocating resources towards regulatory filings for RP-L102 and is exploring external partnerships for its development [179]. - Significant additional R&D efforts are required for drug candidates currently under development, necessitating substantial capital and compliance capabilities [218]. Clinical Trials and Results - The Phase 1 clinical trial of RP-A501 for Danon disease involved seven patients, with a high-dose cohort showing a patient who underwent heart transplant due to advanced disease [135]. - Long-term safety and efficacy results from the Phase 1 study of RP-A501 indicated that all evaluable patients demonstrated LAMP2 protein expression at 12 months, sustained up to 60 months, and a reduction of left ventricular mass index by ≥10% at 12 months [139]. - The Phase 2 pivotal trial of RP-A501 for Danon disease (DD) has enrolled 12 patients, including a pediatric safety run-in of 2 patients, with a dose level of 6.7 x 10^13 GC/kg [147]. - The co-primary endpoints for the trial include a median reduction of 24% in left ventricular mass index (LVMI) and improvements in LAMP2 protein expression [151]. - Cardiac biomarkers showed a median reduction of 84% in cardiac troponin I and 57% in BNP [151]. - The FDA has authorized the resumption of the Phase 2 study with a recalibrated dose of 3.8 x 10^13 GC/kg after a temporary clinical hold due to serious adverse events [155]. - The ongoing Phase 1 study of RP-A601 has completed enrollment in the U.S. and is assessing safety and preliminary efficacy in high-risk patients [161]. - Preliminary data from the Phase 1 study of RP-A601 indicated a 110% and 398% increase in PKP2 protein expression in patients with low baseline levels [163]. - The Phase 1/2 trial of RP-L201 for severe LAD-I treated nine patients, showing 100% survival without the need for allogeneic transplant, with significant reductions in infections and restoration of wound healing capabilities [170]. - The FDA accepted a BLA filing for RP-L201 with priority review, initially set for March 31, 2024, but extended to June 30, 2024, due to additional information requests [171]. Manufacturing and Operational Capacity - The company operates a ~100,000 sq. ft. U.S.-based in-house AAV cGMP manufacturing facility in Cranbury, New Jersey, to support clinical and future commercial endeavors [129]. - The company operates a 103,720 square foot cGMP manufacturing facility in Cranbury, New Jersey, supporting clinical development and potential future commercialization [187]. - The company has successfully produced multiple commercial-grade Danon AAV cGMP batches since 2022, improving product quality for the Phase 2 study [152]. Market and Competitive Landscape - The cardiovascular gene therapy programs target major genetically defined causes of hypertrophic, arrhythmogenic, and dilated cardiomyopathies, impacting over 100,000 patients in the U.S. and EU [129]. - BAG3 mutations are estimated to cause 2.3% to 6.7% of dilated cardiomyopathy (DCM) cases in the U.S., Europe, and Japan, with a prevalence of approximately 30,000 individuals in the U.S. [164]. - The company operates in a highly competitive environment with rapid technological changes, impacting the viability of its product development efforts [218].

Financial Performance - Rocket Pharmaceuticals reported a net loss of $50.3 million, or $0.45 per share, for Q3 2025, compared to a net loss of $66.7 million, or $0.71 per share, for Q3 2024[9]. - Total operating expenses for Q3 2025 were $52.2 million, down from $69.4 million in Q3 2024[15]. - The company incurred approximately $3.3 million in restructuring expenses in 2025[8]. Research and Development - Research and development expenses decreased to $34.1 million in Q3 2025 from $42.3 million in Q3 2024, reflecting a reduction of $8.2 million[9]. - The Phase 2 trial of RP-A501 for Danon disease is set to resume in the first half of 2026, with three additional patients to be treated at a recalibrated dose[4]. Administrative Expenses - General and administrative expenses were $18.4 million for Q3 2025, down from $27.1 million in Q3 2024, a decrease of $8.7 million[9]. Cash and Assets - Cash, cash equivalents, and investments totaled approximately $222.8 million as of September 30, 2025, expected to fund operations into Q2 2027[9]. - Rocket's total assets were $368.0 million as of September 30, 2025, compared to $527.7 million at the end of 2024[15]. Regulatory Updates - The FDA has set a PDUFA date of March 28, 2026, for KRESLADI™ for the treatment of severe leukocyte adhesion deficiency-I (LAD-I)[4]. Leadership Changes - Rocket Pharmaceuticals appointed Dr. Syed Rizvi as Chief Medical Officer, bringing over 20 years of experience in cell and gene therapy[5].

Group 1 - Rocket Pharmaceuticals Inc. is recognized as one of the best NASDAQ stocks under $5, with the recent FDA acceptance of the resubmission of the Biologics License Application for KRESLADI [1][2] - KRESLADI is an investigational gene therapy aimed at treating severe Leukocyte Adhesion Deficiency-I, a rare genetic immune disorder, supported by positive clinical data from a global Phase 1/2 study [2][3] - The Phase 1/2 study reported a 100% overall survival rate at 12 months post-infusion, with all primary and secondary endpoints met, and no serious treatment-related adverse events [3] Group 2 - Rocket Pharmaceuticals operates as a late-stage biotechnology company focused on developing gene therapies for rare and devastating diseases in the US [4]

Core Viewpoint - Bragar Eagel & Squire, P.C. is investigating potential claims against Rocket Pharmaceuticals, Inc. due to allegations of fiduciary duty breaches by the board of directors, following a class action complaint related to misleading statements about the company's clinical trial safety [2][8]. Group 1: Legal Investigation - The investigation is focused on long-term stockholders of Rocket Pharmaceuticals who held securities between February 27, 2025, and May 26, 2025 [2]. - The class action complaint was filed on June 11, 2025, concerning the company's failure to disclose critical information about the safety of its RP-A501 clinical trial [2][8]. Group 2: Clinical Trial Issues - The complaint alleges that Rocket provided overly positive statements while concealing material adverse facts regarding the safety of RP-A501, including the risk of Serious Adverse Events (SAEs) [8]. - On May 27, 2025, Rocket announced that the FDA placed a clinical hold on the RP-A501 Phase 2 pivotal study after a patient suffered a SAE, which included death, following undisclosed amendments to the trial protocol [8]. Group 3: Stock Price Impact - Following the announcement of the clinical hold, Rocket's stock price plummeted from $6.27 per share on May 23, 2025, to $2.33 per share on May 27, 2025, marking a decline of approximately 37% in just one trading day [8].

Core Insights - Rocket Pharmaceuticals (RCKT) has had its resubmitted biologics license application (BLA) for gene therapy Kresladi accepted by the FDA, aiming to treat severe leukocyte adhesion deficiency-I (LAD-I) [1][7] - The FDA's decision on the BLA is anticipated by March 28, 2026, with the potential approval marking RCKT's first commercial product [1][10] - Following the announcement, RCKT's shares increased by 23.1% [1] Company Performance - Year-to-date, RCKT's shares have decreased by 66.1%, contrasting with a 7.7% increase in the industry [4] Clinical Data - The BLA is supported by positive results from a global phase I/II study, demonstrating 100% overall survival for LAD-I patients at 12 months post-infusion, meeting all primary and secondary endpoints [2] - Kresladi treatment was well tolerated, with no serious adverse effects reported [2] Regulatory Incentives - If approved, RCKT will qualify for a Rare Pediatric Disease Priority Review Voucher (PRV), which can expedite the approval of another drug or be sold to a larger pharmaceutical company [3] Background on LAD-I - LAD-I is a rare genetic disorder caused by a defect in the ITGB2 gene, leading to severe immune system issues and recurrent infections [9]

Core Insights - The FDA has accepted Rocket Pharmaceuticals' resubmission of the Biologics License Application for Kresladi, a gene therapy for severe Leukocyte Adhesion Deficiency-I [1][2] - The PDUFA date for Kresladi is set for March 28, 2026 [2] - The BLA is supported by positive clinical data showing 100% overall survival at 12 months post-infusion in a global Phase 1/2 study [3][4] Clinical Data - All primary and secondary endpoints were met in the clinical study, with Kresladi being well tolerated and no serious adverse events reported [4] - Significant reductions in the incidence of infections were observed compared to pre-treatment levels, along with improvements in skin lesions and wound healing [4] Market Outlook - Analyst Sami Corwin from William Blair views the acceptance positively, anticipating a potential launch in Q3 2026 and modeling peak sales of $294 million by 2031 [5] - The company plans to focus more on its late-stage AAV pipeline, which may lead to modest commercialization efforts for Kresladi [6] Stock Performance - Following the news, Rocket Pharmaceuticals' stock increased by 18.01%, reaching $4.09 [6]

Group 1: U.S. Stock Market Overview - U.S. stocks experienced a decline, with the Dow Jones index dropping approximately 300 points on Tuesday [1] Group 2: Domino's Pizza, Inc. Performance - Domino's Pizza reported third-quarter earnings per share of $4.08, surpassing the analyst consensus estimate of $3.96 [1] - The company's quarterly sales reached $1.147 billion, reflecting a year-over-year increase of 6.2%, exceeding the expected $1.137 billion [1] - Following the positive financial results, Domino's shares increased by 4.1% to $424.95 on Tuesday [1] Group 3: Notable Stock Gains - Astria Therapeutics, Inc. shares surged 40.6% to $11.91 after BioCryst announced plans to acquire the company [4] - Critical Metals Corp. shares rose 22.9% to $28.62 amid U.S.-China trade tensions and JPMorgan's $1.5 trillion initiative [4] - Trilogy Metals Inc. gained 20.6% to $7.93 [4] - American Resources Corporation jumped 17.5% to $5.85, participating in additional October conferences [4] - Navitas Semiconductor Corporation rose 16.3% to $11.60 after providing a progress update on its power devices [4] - Telefonaktiebolaget LM Ericsson gained 16% to $9.47, reporting third-quarter EPS and sales above estimates [4] - Rocket Pharmaceuticals, Inc. increased by 15.5% to $4.0100 after FDA acceptance of its Biologics License Application resubmission [4] - Orla Mining Ltd. surged 11.2% to $12.74, reporting strong gold production from Musselwhite [4] - Albertsons Companies, Inc. rose 9.7% to $18.58 after better-than-expected second-quarter results and a $750 million share repurchase agreement [4] - Polaris Inc. gained 9.2% to $66.98, announcing the divestment of its majority stake in Indian Motorcycle [4] - Spyre Therapeutics, Inc. rose 11.2% to $21.04, pricing a $275 million public offering of common stock [4]

Core Insights - Rocket Pharmaceuticals (NASDAQ:RCKT) saw an approximate 11% increase in premarket trading following the announcement that the FDA accepted its resubmitted marketing application for the gene therapy candidate Kresladi [4] - The FDA had previously rejected the company's biologics license application in June 2024 [4]