ROCKET PHARMACEUTICALS, INC. (RCKT)·Q2 2025 Earnings Summary

Executive Summary

• Q2 2025 GAAP net loss was $68.9M with GAAP EPS of -$0.62 (basic and diluted), modestly improved year over year versus -$69.6M and -$0.74 in Q2 2024; operating expense reductions began to flow through R&D and G&A lines .
• Cash, cash equivalents and investments were $271.5M; management extended operational runway guidance to the second quarter of 2027, aided by a ~30% headcount reduction and ~25% 12‑month OpEx reduction plan .
• Strategic focus sharpened on AAV cardiovascular programs: RP‑A601 received FDA RMAT designation and is being advanced toward a potential pivotal trial; RP‑A701 IND cleared and Fast Track designated with Phase 1 start‑up underway .
• RP‑A501 Phase 2 pivotal study (Danon disease) remains under clinical hold following an SAE and patient death; the company cannot provide guidance on trial completion timing, and paused incremental investment in FA and PKD programs while exploring strategic alternatives .

What Went Well and What Went Wrong

What Went Well

• “The second quarter of 2025 marked an important inflection point… 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.” — Gaurav Shah, M.D., CEO .
• FDA granted RMAT designation for RP‑A601 (PKP2‑ACM); engagement underway on potential pivotal trial design following encouraging initial Phase 1 data presented at ASGCT in May 2025 .
• RP‑A701 (BAG3‑DCM) received IND clearance and Fast Track designation; Phase 1 start‑up underway to evaluate safety, biological activity and preliminary efficacy in adults with BAG3‑DCM .

What Went Wrong

• RP‑A501 (Danon disease) pivotal trial placed on FDA clinical hold after an SAE related to capillary leak syndrome leading to a patient’s death; dosing paused and root‑cause analysis initiated with regulators and safety monitors; no timeline guidance on trial completion .
• Portfolio deprioritization: Rocket paused additional investments in FA (RP‑L102) and PKD (RP‑L301), and indicated FA approval is no longer anticipated in 2026, reflecting capital allocation constraints and regulatory prioritization .
• Restructuring charges of ~$3.5M were incurred in 1H 2025; while cost actions support longer runway, they reflect near‑term operational headwinds and transition costs .

Financial Results

Income Statement (Quarterly comparison: oldest → newest)

Notes: Company did not report product revenue or margin metrics in these releases; no segment revenue disclosed .

Balance Sheet Snapshot (Period‑end)

KPIs (Operational)

• Shares outstanding: 106.754M at Mar 31, 2025; 107.884M at Jun 30, 2025 .

Versus Estimates

• S&P Global Wall Street consensus for Q2 2025 EPS and revenue was unavailable via our S&P data source at the time of analysis; therefore estimate comparisons are not included (unavailable via S&P Global) [GetEstimates].

Guidance Changes

Earnings Call Themes & Trends

Note: A Q2 2025 earnings call transcript was not available in our document corpus; themes derived from company press releases and 8‑K filings.

Management Commentary

• “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.” — Gaurav Shah, M.D., CEO .
• On RP‑A601 (PKP2‑ACM): “Initial Phase 1 data demonstrating encouraging safety and preliminary efficacy… RMAT designation… engaging with FDA on potential pivotal trial design.” .
• On RP‑A701 (BAG3‑DCM): “IND received clearance… Fast Track designation… enables increased communication with FDA, potential for accelerated approval, and rolling BLA review.” .
• On RP‑A501 (Danon): “Investigation into SAE… trial on clinical hold… actively working with FDA… cannot provide guidance on trial completion timing.” .
• Strategic focus and cost actions: Organizational restructuring to align with AAV CV prioritization, ~30% workforce reduction and ~25% 12‑month OpEx reduction .

Q&A Highlights

• A full Q2 2025 earnings call transcript was not available in our document set; no direct Q&A content to summarize or clarify. Company disclosures on the clinical hold, cost actions, and pipeline progress are captured in the Q2 8‑K and press releases .

Estimates Context

• S&P Global Wall Street consensus estimates for Q2 2025 EPS and revenue were unavailable at the time of analysis; as such, formal comparisons versus S&P consensus cannot be provided and estimate‑driven “beat/miss” designations are not included (unavailable via S&P Global) [GetEstimates].
• Given lack of reported product revenue, the company did not provide margin metrics in the Q2 press release; investor focus should remain on cash runway, cost trajectory, and regulatory milestones .

Key Takeaways for Investors

• The clinical hold on RP‑A501 is the principal overhang; re‑initiation timing remains uncertain and will likely be the key stock driver near term .
• The pivot to AAV cardiovascular with RMAT for RP‑A601 and an IND‑cleared, Fast Track RP‑A701 adds regulatory momentum that can offset hold‑related concerns if pivotal designs advance smoothly .
• Cost actions are material: ~30% headcount reduction and ~25% 12‑month OpEx reduction extend runway to Q2 2027, providing execution time for pivotal planning and early‑stage clinical progress .
• KRESLADI (LAD‑I) BLA resubmission is expected before year‑end; acceptance and review progress could add a non‑cardiac catalyst path and potential PRV value option if ultimately approved .
• Management’s tone emphasizes disciplined capital allocation and focus on higher‑value programs; near‑term updates from FDA engagements (RP‑A601) and trial start‑up (RP‑A701) are important milestones to watch .
• With product revenue absent and S&P consensus unavailable, traders should center on regulatory outcomes and cost trajectory rather than earnings “beats/misses,” as narrative inflections will be driven by FDA interactions and clinical events .
• Leadership updates (COO appointment) and organizational changes suggest an operational posture geared to manufacturing, supply chain and clinical execution as programs mature toward pivotal stages .