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Rocket Pharma Q2 net income $123.2M on PRV sale; cash $283.7M, runway to Q2 2028
Net income $123.2M ($1.09 basic EPS) vs net loss $68.9M ($0.62 loss) in Q2 2025.
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Rocket Pharma reports positive safety data from first 3 patients in Phase 2 Danon disease trial
Initial three patients treated safely with RP-A501 at recalibrated dose 3.8×10^13 GC/kg; no TMA or capillary leak syndrome observed.
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Rocket Pharmaceuticals closes sale of rare pediatric disease PRV for $180M
Closed sale of Rare Pediatric Disease PRV to large pharma for gross proceeds of $180M.
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Rocket Pharma Q1 net loss $47.6M; KRESLADI gets FDA accelerated approval; PRV sold for $180M
Net loss of $47.6M ($0.42/sh) narrowed from $61.3M ($0.56/sh) YoY.
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Rocket Pharmaceuticals sells Rare Pediatric Disease PRV for $180M, extending cash runway to Q2 2028
Enters definitive agreement to sell PRV for $180M in cash; closing subject to HSR antitrust waiting period.
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FDA approves Rocket's gene therapy KRESLADI for severe LAD-I; Rare Pediatric PRV granted
KRESLADI (marnetegragene autotemcel) approved for pediatric severe LAD-I with no HLA-matched sibling donor.
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Rocket Pharma FY2025 net loss $223.1M; cash $188.9M; KRESLADI PDUFA March 28, 2026
Net loss $223.1M ($2.01/shr) vs $258.7M ($2.73/shr) in FY2024; R&D spend down 17% to $142.0M.
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Rocket Pharma Q3 net loss $50.3M; KRESLADI PDUFA March 2026; Danon trial to resume 1H 2026
Q3 net loss $50.3M ($0.45/share), down from $66.7M ($0.71) YoY; R&D expenses $34.1M vs $42.3M.
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Rocket Pharma: FDA accepts BLA resubmission for KRESLADI gene therapy; PDUFA March 28, 2026
FDA accepted BLA resubmission for KRESLADI (marne-cel) for severe LAD-I; PDUFA target date March 28, 2026.
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Rocket Pharma withdraws BLA for RP-L102 (Fanconi anemia gene therapy) as part of corporate prioritization
Voluntarily withdrew BLA for RP-L102 (mozafancogene autotemcel) from FDA on October 3, 2025.
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Rocket Pharma finalizes Dr. Patel separation; consulting through Dec 2026 with $582K retainer
Dr. Kinnari Patel receives $482,040 lump-sum severance (10 months salary) and $30,045 healthcare allowance.
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Rocket Pharmaceuticals: FDA lifts clinical hold on Phase 2 trial of RP-A501 for Danon disease
FDA lifted clinical hold in under 3 months; trial resumes with recalibrated dose 3.8x10^13 GC/kg for 3 patients with 4-week intervals.
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Rocket Pharmaceuticals: President Patel to depart, 30% workforce cut, Q2 net loss $68.9M
Dr. Kinnari Patel to leave as President and Head of R&D effective Dec 31, 2025; steps down Aug 8.
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Rocket Pharma restructures, cuts headcount 30%, delays FA/PKD programs; cash runway to Q2 2027
Headcount reduction of ~30% and 12-month cash burn reduction of ~25% as part of corporate reorganization.
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Rocket Pharmaceuticals cuts workforce 30%, extends cash runway into Q2 2027
Reduction in workforce of ~30%; expected to cut 12-month operating expenses by ~25%.
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FDA grants RMAT designation to Rocket Pharma's RP-A601 gene therapy for PKP2-ACM
FDA granted RMAT designation for RP-A601 (AAV-based gene therapy) for PKP2-arrhythmogenic cardiomyopathy.
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Rocket Pharma announces FDA clinical hold on Phase 2 Danon gene therapy trial after patient death
Patient in Phase 2 trial of RP-A501 experienced SAE (capillary leak syndrome) and later died due to acute systemic infection.
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Rocket Pharma reports positive Phase 1 data for RP-A601 in PKP2-ACM
RP-A601 well-tolerated; no dose-limiting toxicities in 3 patients with up to 12 months follow-up.
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Rocket Pharma Q1 net loss $61.3M; cash $318.2M; pipeline on track
Net loss of $61.3M ($0.56/share) vs $62.1M in Q1 2024; R&D expenses down to $35.9M.
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Rocket Pharma FY2024 net loss $258.7M ($2.73/sh); cash $372.3M; RP-A501 Phase 2 ongoing
Net loss $258.7M ($2.73/sh) for FY2024 vs $245.6M ($2.92/sh) in FY2023.
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Rocket Pharmaceuticals raises ~$182.8M in public offering and private placement
Public offering of 15.18M shares at $12.50/sh, including full exercise of underwriters' option; net proceeds ~$178.1M.
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Rocket Pharma reports positive long-term Phase 1 Danon Disease data; PKD program enrollment delayed to 2025
All evaluable Danon disease patients showed cardiac LAMP2 protein expression at 12 months, sustained up to 60 months, and ≥10% LV mass index reduction sustained up to 54 months.
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Rocket Pharma Q3 2024 net loss $198.4M; cash $235.7M; pipeline advances
Net loss $198.4M ($2.11/share) for nine months ended Sept 30, 2024, vs $185.9M ($2.30/share) prior year.
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Rocket Pharma H1 2024 net loss $131.7M; KRESLADI review ongoing, cash into 2026
Net loss of $131.7M ($1.40/sh) for H1 2024 vs $124.0M ($1.55/sh) for H1 2023.
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Rocket Pharma receives FDA Complete Response Letter for KRESLADI BLA; limited CMC info requested
FDA issued Complete Response Letter for KRESLADI (marne-cel) BLA; requested limited additional CMC information.
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Rocket Pharma increases authorized shares to 180M after shareholder approval
Authorized common shares increased from 120M to 180M (50% increase); amendment effective June 20, 2024.
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Rocket Pharma Q1 net loss $62.1M; cash $330.3M; EMA accepted MAA for FA, PDUFA date June 30 for LAD-I
Net loss of $62.1M ($0.66/sh) vs $58.3M ($0.73/sh) in Q1 2023; G&A rose to $22.1M on commercial prep.
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Rocket Pharma names Aaron Ondrey CFO; expands leadership team for commercial transition
Aaron Ondrey appointed CFO, effective March 25, 2024; previously CFO of Mirati Therapeutics.
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Rocket Pharma Reports Q4 2023 Net Loss of $59.7M; PDUFA Date for KRESLADI Set for June 30, 2024
Cash, cash equivalents and investments stood at $407.5M as of Dec 31, 2023, providing runway into 2026.
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FDA extends Rocket Pharma's KRESLADI BLA review to June 30, 2024; no advisory committee needed
FDA extended Priority Review by 3 months to June 30, 2024 to review clarifying CMC information submitted in response to FDA requests.
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FDA accepts BLA and grants Priority Review for Rocket's RP-L201 gene therapy for severe LAD-I; PDUFA March 31, 2024
PDUFA target action date set for March 31, 2024.
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Rocket Pharmaceuticals raises ~$188.9M in public offering of common stock and pre-funded warrants
Offered 7,812,500 shares at $16.00/share and pre-funded warrants for 3,126,955 shares at $15.99 each.
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Rocket Pharma reaches FDA alignment on Phase 2 pivotal trial for RP-A501 in Danon Disease
FDA agreed to single-arm, open-label trial with 12 patients, natural history comparator, and co-primary endpoint of LAMP2 expression and LV mass at 12 months for accelerated approval.