ROCKET PHARMACEUTICALS, INC. (RCKT)·Q1 2025 Earnings Summary

Executive Summary

• Q1 2025 showed disciplined cost control (R&D down year over year) and a modestly improved net loss per share, while management reiterated focus on AAV cardiovascular programs and extended cash runway guidance into Q4 2026; subsequent May update extended runway into 2027 via spending reductions .
• Preliminary RP-A601 Phase 1 data in May indicated tolerability with no dose-limiting toxicities and signs of clinical benefit across protein expression, arrhythmia burden, and quality of life, reinforcing the AAV thesis .
• A serious adverse event in the RP-A501 Danon Phase 2 trial resulted in a patient death and an FDA clinical hold, raising near-term regulatory and safety risk; dosing paused and timing for trial completion is now uncertain .
• Rolling BLA for RP-L102 (Fanconi Anemia) continued, and FDA review of additional CMC information for KRESLADI (severe LAD-I) remains ongoing, preserving potential hematology catalysts .
• Wall Street consensus (S&P Global) for Q1 2025 EPS and revenue was unavailable, limiting direct beat/miss comparisons; investors should anchor on cash, expenses, and pipeline milestones for near-term stock reaction drivers [GetEstimates: Q1 2025 returned no data].

What Went Well and What Went Wrong

What Went Well

• Management emphasized continued momentum and resource prioritization toward AAV cardiovascular programs: “We continue to prioritize our current resources towards our AAV cardiovascular programs… and as result have expanded our runway into the fourth quarter of 2026.” .
• RP-A601 initial Phase 1 data presented at ASGCT: RP-A601 was generally well-tolerated with no dose-limiting toxicities, increased PKP2 protein expression, and improvements/stabilization in arrhythmia burden and quality-of-life metrics; no further dose escalation planned .
• Regulatory progress: Rolling BLA initiated for RP-L102 with final module submission targeted late 2025/early 2026; EMA review ongoing; KRESLADI CMC review continues with a complete BLA to address CRL anticipated in 2025 .

What Went Wrong

• Danon (RP-A501) Phase 2 adverse event: capillary leak syndrome and patient death led to voluntary dosing pause followed by FDA clinical hold; trial resumption timing uncertain, increasing program risk .
• G&A rose year over year on legal and commercial preparation costs, pressuring operating spend: G&A was $28.4M in Q1 2025 vs $22.1M in Q1 2024 (drivers: +$4.0M legal, +$1.5M commercial prep, +$0.7M stock comp) .
• Cash declined quarter over quarter, reflecting ongoing investment: $318.2M at March 31, 2025 vs $372.3M at December 31, 2024, before subsequent spending reduction push to extend runway .

Financial Results

P&L and Balance Sheet (Quarterly)

Operating Expense Detail

Notes:

• Q1 YoY R&D decreased by $9.3M driven by lower manufacturing/development and materials, lab/office expense, compensation (headcount), and professional fees .
• Q1 YoY G&A increased on commercial preparation (+$1.5M), legal (+$4.0M), and stock comp (+$0.7M) .
• No non-GAAP metrics were disclosed in the documents .

EPS vs Estimates

S&P Global estimates data for Q1 2025 EPS and revenue were unavailable, so beat/miss vs consensus cannot be assessed [GetEstimates: Q1 2025 returned no data].

Guidance Changes

No numerical guidance was provided for revenue, margins, OpEx beyond qualitative drivers, OI&E, tax rate, or dividends in the quarter’s materials .

Earnings Call Themes & Trends

Management Commentary

• “We continue to prioritize our current resources towards our AAV cardiovascular programs… and as result have expanded our runway into the fourth quarter of 2026.” — Gaurav Shah, M.D., CEO .
• “Preliminary data from the Phase 1 study of RP-A601 for PKP2-ACM are highly encouraging, signaling potential clinical benefit along with a generally well-tolerated safety profile.” — Gaurav Shah, M.D., CEO .
• “We are heartbroken by this loss and are fully committed to our mission to develop gene therapies that address the underlying cause of devastating diseases like Danon.” — Gaurav Shah, M.D., CEO (RP-A501 update) .

Q&A Highlights

• An earnings call transcript for Q1 2025 was not available in the document catalog. Company hosted an investor webinar on May 15 to discuss RP-A601 preliminary data, and a May 27 call addressed the RP-A501 SAE and clinical hold; key themes centered on safety management, regulatory dialogue, and runway extension via spending reductions .
• Management highlighted no dose-limiting toxicities in RP-A601 and patient-level biomarker/clinical improvements; clarified that no further dose escalation is planned .
• For RP-A501, the company described the SAE context (novel immune suppression agent, complement mitigation) and ongoing root cause analysis with FDA and DSMC; guidance on Phase 2 timing is withdrawn pending resolution .

Estimates Context

• S&P Global Wall Street consensus for Q1 2025 EPS and revenue was unavailable; direct beat/miss cannot be determined [GetEstimates: Q1 2025 returned no data].
• Given the RP-A501 clinical hold and extended runway, we expect sell-side models to adjust timelines for Danon pivotal completion and potential commercialization, lower near-term R&D for AAV programs as spending is reduced, and extend cash runway assumptions into 2027, aligning with company commentary .

Key Takeaways for Investors

• RP-A601 Phase 1 data is a positive proof point for AAV cardiovascular strategy, with tolerability and multi-domain clinical signals; watch subsequent cohort and durability updates for confirmation .
• RP-A501 Danon safety event and FDA clinical hold are material overhangs; stock likely trades on resolution path, root-cause findings, and clarity on re-initiation/timing .
• Cash of $318.2M and extended runway into Q4 2026 (then into 2027) provide operational flexibility despite pipeline headwinds; near-term spend reductions help de-risk financing needs .
• R&D reductions year over year reflect focused resource allocation; however, higher G&A driven by commercial and legal costs offsets some savings—monitor expense trajectory through mid-2025 .
• Hematology portfolio remains a source of potential non-dilutive value creation (PRV sales upon approvals); RP-L102 rolling BLA and KRESLADI CMC path are key 2025 regulatory watch items .
• With consensus estimates unavailable for Q1 2025, trading setup hinges on event-driven catalysts: RP-A601 follow-ups, RP-A501 hold resolution, and regulatory updates; incremental disclosures can shift sentiment quickly .
• Near-term positioning: bias to event-driven strategy—consider hedging around RP-A501 regulatory milestones while maintaining optionality for RP-A601 upside if data mature positively .