Rocket Pharmaceuticals (RCKT) investor relations material

Executive summary

• Achieved FDA accelerated approval for KRESLADI™ in March 2026 for severe LAD-I, marking the first commercial product launch and receipt of a $180M Priority Review Voucher (PRV) subsequently sold in Q2 2026.

• Monetized a Priority Review Voucher for $180 million, significantly strengthening the cash position.

• Advanced pivotal Phase 2 trial for RP-A501 in Danon disease after FDA clinical hold was lifted, with dosing resumed under a modified protocol and no new significant safety concerns as of August 2026.

• Multiple clinical and regulatory milestones remain on track for the second half of 2026, including updates on Danon disease, PKP2-ACM, and BAG3-DCM programs.

• Continued development of cardiovascular genetic medicines portfolio, including RP-A601 (PKP2-ACM) and RP-A701 (BAG3-DCM), and commercial launch preparations for KRESLADI™.

Financial highlights

• Net income of $123.2M for Q2 2026, compared to a net loss of $68.9M in Q2 2025, driven by the $178.2M gain from the PRV sale.

• Six-month net income of $75.6M, reversing a $130.3M loss in the prior year period.

• R&D expenses decreased to $29.5M in Q2 2026 (down $13.2M YoY) and $61.0M for the first half (down $17.6M YoY), reflecting portfolio prioritization and restructuring.

• G&A expenses fell to $17.4M in Q2 2026 (down $7.6M YoY) and $34.5M for the first half (down $19.0M YoY), mainly due to lower headcount and commercial launch spending.

• Cash, cash equivalents, and investments totaled $283.7M as of June 30, 2026, sufficient to fund operations into Q2 2028.

Outlook and guidance

• KRESLADI™ commercial rollout expected to be phased, with no material product revenue anticipated in the near term due to the ultra-rare patient population.

• Focus remains on advancing AAV-based cardiovascular programs and leveraging the strengthened balance sheet for long-term growth.

• Management expects continued operating losses as R&D investment persists, but current liquidity is projected to support operations into Q2 2028.

• Regulatory and clinical updates for Danon disease, PKP2-ACM, and BAG3-DCM anticipated in the second half of 2026.

• Commercial availability and patient onboarding for KRESLADI expected to begin in Q4 2026.