RCKT

Shares of Rocket Pharmaceuticals RCKT were down on Friday after the company announced weaker-than-expected first-quarter 2026 earnings. RCKT incurred a loss of 42 cents per share in the first quarter of 2026, wider than the Zacks Consensus Estimate of a loss of 41 cents. In the year-ago quarter, the company had reported a loss of 56 cents per share. Rocket Pharmaceuticals did not record any revenues in the first quarter. Year to date, shares of Rocket Pharmaceuticals have risen 3.4% against the industry’s decline of 1.7%. Image Source: Zacks Investment Research In the reported quarter, general and administrative expenses declined by around 39.8% year over year to $17.1 million, owing to lower legal expenses and other expenses. Research and development expenses were $31.5 million, down 12.2% from the year-ago quarter’s figure. The decrease reflects more disciplined spending and resource management after the company’s recent organizational restructuring. As of March 31, 2026, Rocket Pharmaceuticals had cash, cash equivalents and investments of $144.4 million compared with $188.9 million as of Dec. 31, 2025. Management expects this cash balance, along with proceeds from the sale of the Priority Review Voucher (PRV) announced last month, to fund operations into the second quarter of 2028. In March 2026, the FDA granted accelerated approval to RCKT’s gene therapy Kresladi (marnetegragene autotemcel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), an ultra-rare genetic disorder. Following the nod, Kresladi became the first gene therapy to be approved by the FDA for treating children with severe LAD-I due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. With the FDA approval for Kresladi, the company received a Rare Pediatric Disease PRV, which is an incentive given by the FDA to encourage the development of drugs and biologics for rare and serious diseases. Last month, RCKT entered into a definitive agreement to sell its PRV for $180 million. The PRV monetization provides non-dilutive capital to support the company’s cardiovascular pipeline. Last August, the FDA lifted the clinical hold on the pivotal phase II study evaluating RCKT’s investigational gene therapy candidate, RP-A501, for treating patients with Danon disease. RP-A501 is the most...

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, N.J., May 07, 2026--(BUSINESS WIRE)--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 Dosing the initial three-patient cohort for the Phase 2 study of RP-A501 in Danon disease is on track. 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. 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. Following FDA alignment, Rocket anticipates providing a program update in the second half of 2026. Details of th...

CRANBURY, N.J. (AP) — CRANBURY, N.J. (AP) — Rocket Pharmaceuticals Inc. (RCKT) on Thursday reported a loss of $47.6 million in its first quarter. The Cranbury, New Jersey-based company said it had a loss of 42 cents per share. The results fell short of Wall Street expectations. The average estimate of seven analysts surveyed by Zacks Investment Research was for a loss of 41 cents per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on RCKT at https://www.zacks.com/ap/RCKT

CRANBURY, N.J. (AP) — CRANBURY, N.J. (AP) — Rocket Pharmaceuticals Inc. (RCKT) on Thursday reported a loss of $42.5 million in its fourth quarter. On a per-share basis, the Cranbury, New Jersey-based company said it had a loss of 38 cents. The results topped Wall Street expectations. The average estimate of six analysts surveyed by Zacks Investment Research was for a loss of 42 cents per share. For the year, the company reported a loss of $223.1 million, or $2.01 per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on RCKT at https://www.zacks.com/ap/RCKT

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, N.J., February 26, 2026--(BUSINESS WIRE)--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. 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. 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. 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 cardiomyop...

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 Leadership updates with the appointments of Dr. Syed Rizvi, Chief Medical Officer, Christopher Stevens, Chief Operating Officer, and Sarbani Chaudhuri, Chief Commercial & Medical Affairs Officer Cash, cash equivalents and investments of approximately $222.8M; expected operational runway into the second quarter of 2027 CRANBURY, N.J., November 06, 2025--(BUSINESS WIRE)--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 third quarter ending September 30, 2025. "During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket’s AAV cardiovascular gene therapy portfolio," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "In under three months, we aligned with the FDA to resume the Phase 2 pivotal study of RP-A501 for Danon disease, an important milestone that underscores the FDA’s collaborative approach and recognition of the urgent need for innovative therapies in rare cardiovascular disorders. In parallel, we are advancing RP-A601 toward a pivotal Phase 2 study in PKP2-ACM and preparing RP-A701 for first-in-human evaluation in BAG3-DCM. Finally, the upcoming March PDUFA date for KRESLADI™ represents a significant near-term commercial milestone as we work to bring this potentially life-saving therapy to patients with severe LAD-I." 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. In August, 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. 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 path forward for completion of the Phase 2 pivotal study. Details of t...

CRANBURY, N.J. (AP) — CRANBURY, N.J. (AP) — Rocket Pharmaceuticals Inc. (RCKT) on Thursday reported a loss of $50.3 million in its third quarter. The Cranbury, New Jersey-based company said it had a loss of 45 cents per share. The results exceeded Wall Street expectations. The average estimate of seven analysts surveyed by Zacks Investment Research was for a loss of 48 cents per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on RCKT at https://www.zacks.com/ap/RCKT

Prioritizing development of RP-A501 (Danon disease), RP-A601 (PKP2-ACM), and RP-A701 (BAG3-DCM) to advance AAV cardiovascular gene therapy platform for sustained value creation FDA RMAT designation awarded to RP-A601 for PKP2-ACM; Engaging with FDA on pivotal trial design following encouraging initial Phase 1 data at ASGCT IND accepted and FDA Fast Track designation received for RP-A701 in BAG3-DCM program; Phase 1 trial start-up activities underway Organizational restructuring expected to reduce headcount by approximately 30% and lower 12-month cash burn by nearly 25% Chris Stevens appointed Chief Operating Officer Cash, cash equivalents and investments of approximately $271.5M; expected operational runway into the second quarter of 2027 CRANBURY, N.J., August 07, 2025--(BUSINESS WIRE)--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 second quarter ending June 30, 2025. "The second quarter of 2025 marked an important inflection point for Rocket as we refined our strategic focus around our AAV cardiovascular gene therapy platform and took multiple decisive steps to strengthen our financial foundation and thoughtfully adjust to market dynamics. We are fortifying our path to sustained value creation by leaning into programs with the highest value, conserving cash, and driving an efficient, agile organization," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. "With regulatory alignment to resolve the clinical hold for RP-A501 in Danon disease in progress, momentum building for RP-A601 in PKP2-ACM as it advances toward a pivotal Phase 2 trial, and RP-A701 preparing to enter the clinic for the treatment of BAG3-DCM, Rocket is positioned as the leader in the development of gene therapies for inherited cardiomyopathies. While we are pausing additional investments in the FA and PKD programs, we are deeply grateful and committed to the patient communities and are exploring strategic alternatives to advance these programs externally. Finally, our recent reorganization ensures we are appropriately resourced to execute on our near-term milestones with nearly two years of capital." Recent Pipeline and Operational Updates Investigation into...

CRANBURY, N.J. (AP) — CRANBURY, N.J. (AP) — Rocket Pharmaceuticals Inc. (RCKT) on Thursday reported a loss of $68.9 million in its second quarter. The Cranbury, New Jersey-based company said it had a loss of 62 cents per share. Losses, adjusted for restructuring costs, were 59 cents per share. The results fell short of Wall Street expectations. The average estimate of eight analysts surveyed by Zacks Investment Research was for a loss of 57 cents per share. _____ This story was generated by Automated Insights (http://automatedinsights.com/ap) using data from Zacks Investment Research. Access a Zacks stock report on RCKT at https://www.zacks.com/ap/RCKT