ROCKET PHARMACEUTICALS, INC. (RCKT)·Q4 2024 Earnings Summary

Executive Summary

• Operational runway strengthened: cash, cash equivalents and investments were $372.3M, with resources expected to fund operations into Q3 2026; company raised net proceeds of $182.5M in December via an upsized equity offering .
• Quarterly P&L improved sequentially: Q4 net loss per share was $(0.62) vs $(0.71) in Q3 2024 and $(0.64) in Q4 2023; total operating expenses fell to $62.7M from $69.4M in Q3 .
• Clinical/regulatory momentum: Phase 2 RP-A501 dosing ongoing with program update expected in H1 2025; initial Phase 1 RP-A601 data targeted for H1 2025; RP-L102 rolling BLA advancing; FDA review work continues for KRESLADI to resolve CRL in 2025 .
• Scientific validation: NEJM publication and AHA long-term data showed RP-A501 safety and meaningful efficacy across multiple clinical parameters, reinforcing the Danon thesis as a near-term narrative driver .

What Went Well and What Went Wrong

What Went Well

• NEJM and AHA data on RP-A501 demonstrated sustained LAMP2 expression, ≥10% LV mass index reduction at 12 months (up to five years), and improvements in NYHA class, KCCQ (+27 median), troponin (−84% median) and BNP (−57% median), supporting durable efficacy in Danon disease .
• Balance sheet and runway improved: year-end cash/investments of $372.3M and operational runway guided into Q3 2026; December public offering delivered $182.5M net proceeds to fund AAV cGMP batches and pipeline development .
• Management focus and execution: CEO emphasized prioritizing AAV cardiovascular programs while seeking value across the broader pipeline, maintaining BAG3-DCM IND timeline (H1 2025) and Phase 1 RP-A601 low-dose cohort completion .

What Went Wrong

• Continued losses and opex intensity: Q4 net loss of $(60.3)M and annual net loss of $(258.7)M; G&A for FY 2024 rose to $102.0M (from $73.3M) on commercial preparedness and legal costs, highlighting ongoing burn ahead of potential approvals .
• Regulatory overhang persists: FDA requested limited additional CMC information for KRESLADI; company plans a complete BLA submission in 2025 to resolve the CRL, extending timelines for the LAD‑I program .
• Equity dilution risk: December equity financing increased the share count, with 106.45M shares outstanding at year-end; while strengthening liquidity, it raises dilution concerns until commercial inflection .

Financial Results

P&L and EPS comparison

Balance Sheet snapshot

Segment revenue breakdown: Not applicable; company does not report commercial revenues in these materials .

KPIs and Operating Metrics:

• Year-end shares outstanding: 106,453,818 .
• FY 2024 R&D: $171.2M; FY 2024 G&A: $102.0M; FY 2024 net loss: $(258.7)M .
• Cash runway guided into Q3 2026 .

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• “In 2024, we made strong progress in advancing our gene therapy pipeline... we will maintain our focus and resources on advancing our AAV cardiovascular programs while seeking to realize value in our full pipeline...” — Gaurav Shah, M.D., CEO (Q4 release) .
• “Rocket made meaningful progress during the third quarter... completion of enrollment in the RP-A501 program... low dose cohort enrollment completion in the RP-A601 program...” — Gaurav Shah, M.D., CEO (Q3 release) .
• “Data presented today at AHA and published in The New England Journal of Medicine represents a critical milestone... demonstrating for the first time that AAV conferred long-term efficacy in a cardiac indication.” — Gaurav Shah, M.D., CEO (AHA/NEJM press release) .

Q&A Highlights

• No earnings call transcript was available in the document set for Q4 2024; management hosted an investor webcast on Nov 18, 2024 to discuss AHA/NEJM RP-A501 data, emphasizing long-term efficacy signals and program momentum .

Estimates Context

• Wall Street consensus (S&P Global) for Q4 2024 EPS and revenue was not available during this session; estimate-based comparisons are omitted. If needed, we can refresh S&P Global consensus on request and update beat/miss assessments accordingly.

Key Takeaways for Investors

• Liquidity and runway improved: year-end cash/investments of $372.3M and runway into Q3 2026 after $182.5M net equity proceeds; positions Rocket to fund AAV cGMP and multiple clinical catalysts through 2026 .
• Near-term catalysts in H1 2025: RP-A501 Phase 2 program update and initial RP-A601 Phase 1 data; BAG3-DCM IND submission targeted; these are likely stock drivers given Danon and PKP2-ACM market potential .
• Regulatory clarity: RP-L102 rolling BLA advancing (final module 2025); KRESLADI path refined with complete BLA submission planned in 2025 to resolve CRL; timelines suggest potential 2025 outcomes .
• Scientific validation reduces clinical risk: NEJM/AHA data show durable efficacy/safety for RP-A501 across clinically relevant endpoints, bolstering confidence in the Danon program’s pivotal trajectory .
• Expense discipline visible in Q4: sequential opex reduction and lower R&D vs prior year (driven by manufacturing/development cost declines) support burn moderation heading into key readouts .
• Dilution trade-off accepted for runway: December offering increased share count but materially strengthened cash; focus shifts to delivering clinical/regulatory milestones to offset dilution via value creation .
• Monitoring items: resolution timing for KRESLADI CRL, RP-A501 Phase 2 update scope and endpoints, RP-A601 safety/efficacy signals in low-dose cohort, and any additional financing ahead of commercial transitions .