Rocket Pharmaceuticals — Third Quarter 2026 Earnings Summary

Financial Performance:

• Net loss was $49.2 million ($0.43 per share, basic and diluted) for the three months ended September 30, 2026, compared to a net loss of $50.3 million ($0.45 per share) for the same period in 2025.
• Research and development expenses were $31.2 million for the quarter ended September 30, 2026, a decrease from $34.1 million in the same period of 2025, driven by reductions in stock-based compensation, manufacturing costs, and depreciation, partially offset by increases in laboratory, professional, and clinical trial expenses.
• General and administrative expenses were $19.8 million for the quarter ended September 30, 2026, an increase from $18.4 million in the prior year period, primarily due to higher post-marketing commitment and commercial preparation expenses, partially offset by lower stock-based compensation and legal expenses.
• Other income was $1.8 million for the quarter ended September 30, 2026, down from $1.9 million in the prior year period, driven by a decline in accretion of discount on investments.
• Income taxes were $0.1 million for the quarter ended September 30, 2026, compared to $0.0 million in the prior year period.
• Cash, cash equivalents, and investments totaled $279.4 million as of September 30, 2026, compared to $188.9 million as of December 31, 2025.
• Total assets were $419.5 million as of September 30, 2026, compared to $330.4 million as of December 31, 2025.
• Total liabilities were $92.8 million as of September 30, 2026, compared to $53.2 million as of December 31, 2025.
• Total stockholders' equity was $326.7 million as of September 30, 2026, compared to $277.2 million as of December 31, 2025.
• 111,386,536 shares of common stock were outstanding as of September 30, 2026.

Guidance and Future Outlook:

• Rocket expects its current cash position of $279.4 million to fund operations into the third quarter of 2028.
• Additional borrowings under the Hercules credit facility could extend the cash runway into 2029, subject to borrowing conditions, draw timing, amounts, and operating expenditures.
• The company expects to complete dosing for the pivotal Phase 2 trial of RP-A501 in Danon disease by mid-2027, with topline results anticipated in mid-2028.

Business Segments and Product Lines:

• RP-A501 (Danon Disease): FDA alignment was achieved on September 15, 2026, for the pivotal Phase 2 trial, including a recalibrated dose and 12-patient population. The first three patients treated under the modified protocol count toward the pivotal efficacy population. The trial aims to support an accelerated approval pathway with co-primary endpoints of myocardial LAMP2 expression and left ventricular mass index reduction.
• RP-A601 (PKP2-ACM): The Phase 1 study remains open and actively enrolling to characterize biological activity. Previous findings showed increased PKP2 protein expression and improvements in arrhythmia measures and right ventricular function.
• RP-A701 (BAG3-DCM): Dosing was initiated in the Phase 1 trial for BAG3-associated dilated cardiomyopathy. The European Commission granted orphan designation for RP-A701 on October 20, 2026.
• KRESLADI™: Achieved commercial readiness on October 1, 2026. Two Qualified Treatment Centers have been activated to begin commercial patient onboarding, and the external manufacturing network is in place.

Market and Competitive Landscape:

• Independent analyses estimate 10,000-11,000 people living with Danon disease in the U.S., including approximately 4,000 males. Rocket has identified more than 900 Danon patients.
• The company focuses on inherited cardiovascular disorders, with a portfolio of three clinical-stage gene therapy programs targeting hypertrophic, arrhythmogenic, and dilated cardiomyopathies.

Risks and Challenges:

• Clinical Trial Risks: Outcomes depend on patient enrollment, dosing completion, and clinical results; unexpected adverse events or safety findings could impact development.
• Regulatory Risks: Approval and timing depend on regulatory interactions, submissions, and review outcomes. Continued approval of KRESLADI may be contingent on verification of clinical benefit through post-marketing data.
• Manufacturing and Supply: Risks include manufacturing, product comparability, quality, and supply considerations, as well as dependence on third parties for development and commercialization.
• Financial Risks: Future capital requirements, ability to obtain additional funding, and satisfaction of credit facility covenants and debt service obligations.
• Safety Risks: KRESLADI carries risks of serious infections, veno-occlusive disease, neutrophil and platelet engraftment failure, lentiviral vector-mediated insertional oncogenesis, and hypersensitivity reactions.
• Intellectual Property: Risks related to obtaining, maintaining, and enforcing intellectual property protection and defending against infringement claims.

Management Commentary and Tone:

• CEO Gaurav Shah stated that with FDA alignment on the RP-A501 trial, the priority is to complete dosing by mid-2027.
• Management highlighted the advancement of the broader cardiovascular portfolio, including RP-A701 dosing and FDA engagement on RP-A601.
• The tone emphasized that commercial readiness for KRESLADI and the activation of treatment centers bring the company closer to treating eligible children.
• Management noted that financial resources and the Hercules facility support these priorities as the company works toward the next clinical and commercial milestones.

Other Key Points:

• Credit Facility: On October 6, 2026, Rocket announced a credit facility agreement with Hercules Capital, Inc. for up to $150 million. The company received an initial $35 million draw and may draw an additional $35 million at its option. An additional $30 million is available upon achieving a specified Danon clinical milestone, and $50 million is subject to Hercules' investment committee approval.
• Investor Webinar: An investor webinar on October 6, 2026, highlighted updated longer-term Phase 1 findings for RP-A501 and encouraging initial safety observations in the first three patients treated under the modified pivotal protocol.
• Nine Months 2026 Performance: For the nine months ended September 30, 2026, the company reported a net income of $26.4 million ($0.23 per share), compared to a net loss of $180.6 million ($1.63 per share) in the prior year period, primarily due to a $178.2 million gain from the sale of PRV.