Executive Summary

Q3 2025 delivered a narrower net loss and lower operating expenses year over year: net loss improved to $50.3M (vs. $66.7M in Q3’24) and diluted EPS to $(0.45) (vs. $(0.71) in Q3’24), reflecting disciplined cost control following the mid‑year reorganization .
Cash, cash equivalents and investments declined sequentially to $222.8M (from $271.5M in Q2), but management reaffirmed liquidity with an operational runway into Q2 2027; 108.2M common shares were outstanding at quarter‑end .
FDA lifted the clinical hold on RP‑A501 (Danon disease) and authorized resumption at a recalibrated dose; three additional patients are expected to be dosed in 1H 2026. FDA accepted the KRESLADI BLA resubmission with a March 28, 2026 PDUFA date—key 6–12 month regulatory catalysts .
Organization aligned to late‑stage development with leadership additions (CMO, COO, Chief Commercial & Medical Affairs); restructuring charges in 2025 totaled ~$3.3M, supporting lower R&D and G&A trends .
Wall Street consensus (S&P Global) for Q3 2025 revenue/EPS was unavailable; comparisons to estimates are therefore not presented (S&P Global data unavailable).

What Went Well and What Went Wrong

What Went Well

Rapid regulatory progress: “In under three months, we aligned with the FDA to resume the Phase 2 pivotal study of RP‑A501 for Danon disease,” with a revised dose and immunomodulatory plan—setting up 1H 2026 dosing and a clearer path forward .
Additional near‑term catalyst: “KRESLADI™ … received a PDUFA target action date for March 28, 2026,” with Rocket eligible for a Rare Pediatric Disease PRV upon approval .
Financial discipline taking hold: R&D fell to $34.1M (from $42.3M y/y) and G&A to $18.4M (from $27.1M y/y) as cost actions and prioritization reduced expenses; net loss improved to $50.3M (from $66.7M y/y) .

What Went Wrong

Cash draw sequentially: cash/investments declined to $222.8M (from $271.5M in Q2), reflecting continued burn in a pre‑revenue profile despite cost cuts .
Program risk overhang: Following two SAEs (including one death) in May within the RP‑A501 Phase 2, FDA imposed (now lifted) a clinical hold—highlighting safety vigilance and the need to validate the recalibrated dose and immunomodulation .
Litigation overhang: consolidated securities class actions and a derivative suit related to RP‑A501 disclosures remain at early stages; outcomes uncertain and could consume management bandwidth .

Financial Results

Metric	Q3 2024	Q2 2025	Q3 2025
Revenue ($M)	$0.0	—	$0.0
R&D Expense ($M)	$42.3	$42.7	$34.1
G&A Expense ($M)	$27.1	$25.0	$18.4
Restructuring ($M)	$0.0	$3.5	$(0.2) (credit)
Total OpEx ($M)	$69.4	$71.1	$52.2
Net Loss ($M)	$(66.7)	$(68.9)	$(50.3)
Diluted EPS ($)	$(0.71)	$(0.62)	$(0.45)

KPIs and Liquidity

KPI	Q1 2025	Q2 2025	Q3 2025
Cash, Cash Equivalents & Investments ($M)	$318.2	$271.5	$222.8
Shares Outstanding (Period End) (M)	106.75	107.88	108.21
Runway (Mgmt Guidance)	Into Q4 2026	Into Q2 2027	Into Q2 2027

Notes: Company reports one operating segment (no segment revenue/margin breakdown available) . No non‑GAAP metrics were provided in the Q3 release/10‑Q .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Cash runway	Corporate	Into Q2 2027 (Q2’25)	Into Q2 2027 (Q3’25)	Maintained
RP‑A501 (Danon) pivotal Phase 2	Next dosing	No timing while on hold (Q2’25)	Dose 3 additional pts at 3.8×10^13 GC/kg in 1H 2026	Raised visibility (timelines set)
KRESLADI (LAD‑I) BLA	FDA status	Resubmission anticipated before year‑end 2025 (Q2’25)	BLA resubmission accepted; PDUFA 3/28/26	Advanced (date set)
RP‑A701 (BAG3‑DCM)	Phase 1 start‑up	IND accepted; Fast Track; start‑up underway (Q2’25)	Start‑up ongoing (Q3’25)	Maintained
Restructuring charges	FY 2025	~$3.5M in 1H’25	~$3.3M total in 2025	Final estimate updated

Earnings Call Themes & Trends

Transcript not available in our corpus for Q3 2025; themes below reflect company filings/press releases.

Topic	Previous Mentions (Q1’25, Q2’25)	Current Period (Q3’25)	Trend
RP‑A501 (Danon) regulatory path	Q1: Phase 2 ongoing; mid‑year update expected . Q2: clinical hold; safety review; no timeline .	Hold lifted; dosing to resume at recalibrated dose; 1H 2026 for next 3 patients .	Improving clarity (risk managed; path re‑set)
KRESLADI (LAD‑I) BLA	Q1: FDA requested limited CMC info; resubmission in 2025 . Q2: resubmission anticipated before YE25 .	BLA resubmission accepted; PDUFA 3/28/26 .	Milestone advanced (date secured)
RP‑A601 (PKP2‑ACM)	Q1: initial Phase 1 data timing . Q2: RMAT; FDA engagement on pivotal design .	Ongoing FDA engagement on pivotal design .	Steady progress toward pivotal
RP‑A701 (BAG3‑DCM)	Q1: IND submission planned . Q2: IND cleared; Fast Track; Phase 1 start‑up .	Phase 1 start‑up ongoing .	Steady execution
Cost discipline/runway	Q1: runway into Q4’26 . Q2: reorg; ~30% headcount cut; runway into Q2’27 .	Runway into Q2’27 reaffirmed; OpEx down y/y .	Durable cost control
Legal/regulatory risk	—	Securities class actions initiated (subsequently consolidated).	Cases consolidated; derivative action filed in Oct .

Management Commentary

“During the third quarter, we maintained disciplined execution and sharpened our strategic focus on Rocket’s AAV cardiovascular gene therapy portfolio. In under three months, we aligned with the FDA to resume the Phase 2 pivotal study of RP‑A501 for Danon disease…” — Gaurav Shah, M.D., CEO .
“KRESLADI™ … received a PDUFA target action date for March 28, 2026.” .
On the RP‑A501 protocol updates: FDA authorized resumption with a recalibrated 3.8×10^13 GC/kg dose for three sequential patients and an immunomodulatory regimen closer to Phase 1 pediatrics (discontinuing prophylactic C3 inhibitor; lower threshold for reactive C5 inhibitor) .

Q&A Highlights

No Q3 2025 earnings call transcript was available in our dataset; guidance clarifications and thematic color are inferred from the 8‑K/press release and 10‑Q disclosures .

Estimates Context

S&P Global consensus for Q3 2025 revenue and EPS was unavailable at the time of analysis. As a pre‑revenue biotech, reported revenue is $0; EPS and OpEx trends versus year‑over‑year and sequential periods are shown above. Values retrieved from S&P Global.

Key Takeaways for Investors

The quarter de‑risked RP‑A501 near‑term: clinical hold lifted, dosing strategy revised, and a 1H 2026 dosing cadence established—watch for safety/biomarker read‑through from the next three patients as a potential stock catalyst .
KRESLADI BLA resubmission acceptance and a firm 3/28/26 PDUFA date provide a defined regulatory path and an option on PRV monetization (post‑approval), bolstering medium‑term funding flexibility .
Cost actions are showing up in P&L: R&D and G&A down y/y and net loss improved—sustained discipline will be key until catalysts translate into value inflections or partnerships .
Cash runway into Q2 2027 is intact, but sequential cash draw underscores the importance of milestone pacing (RP‑A501 pivotal progress, RP‑A601 pivotal design, RP‑A701 Phase 1 start‑up, KRESLADI review) .
Legal overhang (securities/derivative actions) adds uncertainty; monitor court filings and potential disclosures for risk to management focus/costs .
Medium‑term thesis centers on execution in inherited cardiomyopathies (Danon, PKP2‑ACM, BAG3‑DCM) and optionality from KRESLADI; externalization of FA/PKD could preserve upside with reduced cash burden .

Appendix: Additional Data

Selected quarterly financial statements (from 8‑K/10‑Q) confirm zero revenue, lower OpEx y/y, and improved EPS/net loss in Q3 2025 . Cash bridge shows $318.2M (Q1) → $271.5M (Q2) → $222.8M (Q3) with runway extended via cost actions .

ROCKET PHARMACEUTICALS, INC. (RCKT)·Q3 2025 Earnings Summary