Rocket Pharmaceuticals (NASDAQ: RCKT) wins KRESLADI nod, sells $180M PRV
Filing Impact
Filing Sentiment
Form Type
8-K
Rhea-AI Filing Summary
Rocket Pharmaceuticals, Inc. reported first quarter 2026 results and highlighted major pipeline and financing milestones. The company received FDA accelerated approval for KRESLADI for severe LAD-I and monetized a Rare Pediatric Disease Priority Review Voucher for $180 million.
Pro forma cash, cash equivalents and investments were approximately $322.6 million, providing an expected operational runway into the second quarter of 2028. For the three months ended March 31, 2026, Rocket recorded a net loss of $47.6 million compared with $61.3 million a year earlier, with total operating expenses declining to $48.5 million from $64.4 million. Cash, cash equivalents and investments were $144.4 million as of March 31, 2026.
Positive
- FDA accelerated approval of KRESLADI for severe LAD-I adds Rocket’s first approved gene therapy product and validates its platform in an ultra-rare pediatric immunodeficiency.
- $180 million PRV monetization from the KRESLADI Rare Pediatric Disease Priority Review Voucher provides substantial non-dilutive capital to fund ongoing development.
- Extended cash runway into Q2 2028 with pro forma cash of approximately $322.6 million reduces near-term financing risk while the cardiovascular pipeline advances.
- Narrower quarterly net loss, improving from $61.3 million to $47.6 million, reflects lower operating expenses while maintaining multiple late-stage and early-stage gene therapy programs.
Negative
- None.
Insights
Accelerated approval and PRV sale strengthen Rocket’s cash runway while losses narrow.
Rocket Pharmaceuticals achieved FDA accelerated approval for KRESLADI, its gene therapy for severe LAD-I, and sold the related Rare Pediatric Disease Priority Review Voucher for $180 million. Management cites pro forma cash of $322.6 million and an expected operational runway into Q2 2028.
First quarter 2026 net loss improved to $47.6 million from $61.3 million, driven by lower research and development and general and administrative expenses. This suggests some cost discipline as the company advances its cardiovascular gene therapy portfolio, including a pivotal Phase 2 trial of RP-A501 for Danon disease.
The combination of a newly commercial product, significant non-dilutive funding from the PRV sale, and extended runway reduces near-term financing pressure. Future disclosures around KRESLADI uptake and updates on the Danon program in the second half of the year will help clarify the trajectory of both revenue and development risk.
8-K Event Classification
2 items: 2.02, 9.01
2 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, and exhibit attachments filed with this report.
Key Figures
Net loss Q1 2026: $47,594
Net loss Q1 2025: $61,334
Total operating expenses Q1 2026: $48,511
+5 more
8 metrics
Net loss Q1 2026
$47,594
Three months ended March 31, 2026
Net loss Q1 2025
$61,334
Three months ended March 31, 2025
Total operating expenses Q1 2026
$48,511
Research and development plus general and administrative
Net loss per share Q1 2026
$0.42
Basic and diluted for the quarter ended March 31, 2026
Cash, cash equivalents and investments
$144,379
As of March 31, 2026
Total assets
$285,407
As of March 31, 2026
PRV sale proceeds
$180,000,000
Monetization of KRESLADI Rare Pediatric Disease PRV
Pro forma cash and investments
$322,600,000
Including PRV proceeds and March 31, 2026 balance
Key Terms
accelerated approval, Rare Pediatric Disease Priority Review Voucher, myeloablative conditioning, veno-occlusive disease, +2 more
6 terms
accelerated approval regulatory
"KRESLADI™ granted FDA accelerated approval; Rare Pediatric Disease Priority Review Voucher monetized"
Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.
Rare Pediatric Disease Priority Review Voucher regulatory
"Rare Pediatric Disease Priority Review Voucher monetized for $180 million"
A rare pediatric disease priority review voucher is a transferable regulatory benefit awarded to a company that wins approval for a drug treating a serious but uncommon childhood illness. It works like a “fast-pass” with regulators: the holder can use it to get an accelerated review of a future drug application or sell the voucher to another company, often for a large sum. Investors care because it can speed time to market or generate immediate cash, boosting potential returns and lowering risk on other programs.
myeloablative conditioning medical
"KRESLADI is administered as a one-time intravenous infusion following myeloablative conditioning."
Myeloablative conditioning is an intensive medical treatment that uses high-dose chemotherapy and sometimes radiation to destroy a patient’s bone marrow before a stem cell or bone marrow transplant. Investors should care because it is central to the risk, cost, and potential market size of transplant therapies and related drugs: like clearing a garden before replanting, it creates the conditions for a new immune system but also raises safety, hospitalization, and regulatory considerations.
veno-occlusive disease medical
"Veno-occlusive disease has occurred with KRESLADI treatment."
Veno-occlusive disease is a serious liver condition in which tiny blood channels inside the liver become blocked, causing liver swelling, fluid buildup, and impaired liver function. It most often appears after high-dose chemotherapy or stem-cell transplants and can be life-threatening. Investors care because VOD can drive clinical trial failures, safety warnings, treatment costs, regulatory delays, and potential liability exposure, all of which can sharply affect a biotech or hospital company's valuation.
LVV-mediated insertional oncogenesis medical
"Lentiviral vector (LVV)-mediated insertional oncogenesis may occur after treatment with KRESLADI."
neutrophil engraftment failure medical
"Neutrophil engraftment failure may occur after treatment with KRESLADI."
Neutrophil engraftment failure occurs when transplanted stem cells do not begin producing neutrophils, the white blood cells that fight infection, after a bone marrow or stem cell transplant. Like a newly planted tree that fails to take root and grow leaves, this failure raises the risk of serious infections, extended hospital stays, additional treatments, and higher costs—factors that can affect clinical success, regulatory reviews, and a therapy’s commercial prospects.
Earnings Snapshot
Net loss: $47,594 · Guidance included
Q1 2026
Net loss
$47,594
Net loss per share (basic and diluted)
$0.42
Total operating expenses
$48,511
Cash, cash equivalents and investments
$144,379
Guidance
Pro forma cash, cash equivalents and investments of approximately $322.6 million are expected to provide operational runway into the second quarter of 2028.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 7, 2026
(Exact name of registrant as specified in its charter)
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Registrant’s telephone number, including area code: (609 )
659-8001
Not applicable
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the
following provisions (see General Instruction A.2):
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
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Securities registered pursuant to Section 12(b) of the Act:
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Trading
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Name of each exchange on which
registered
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this
chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new
or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
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Item 2.02.
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Results of Operations and Financial Condition.
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On May 7, 2026, Rocket Pharmaceuticals, Inc. (the “Company”) announced certain financial information for the quarter ended March 31, 2026. A copy of
the press release is being furnished as Exhibit 99.1 to this Current Report on Form 8-K.
The information in this Item 2.02 and Exhibit 99.1 attached hereto is intended to be furnished and shall not be deemed “filed” for purposes of Section 18
of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act, except as
expressly set forth by specific reference in such filing.
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Item 9.01.
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Financial Statements and Exhibits.
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(d) Exhibits.
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99.1
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Press Release of Rocket Pharmaceuticals, Inc. dated May 7, 2026.
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its
behalf by the undersigned hereunto duly authorized.
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Rocket Pharmaceuticals, Inc.
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Date: May 7, 2026
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By:
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/s/ Martin Wilson
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Martin Wilson
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General Counsel and Chief Corporate Officer
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Exhibit 99.1
Rocket Pharmaceuticals Reports First Quarter 2026 Financial Results and
Highlights Recent Progress
Pivotal Phase 2 trial of RP-A501 for Danon disease progressing with dosing reinitiated
First patient dosing in Phase 1 study of RP-A701 for BAG3-related dilated cardiomyopathy
anticipated in mid-2026
KRESLADI™
granted FDA accelerated approval; Rare Pediatric Disease Priority Review Voucher
monetized for $180 million
Pro forma cash, cash equivalents and investments of approximately $322.6 million, including balance as of March 31, 2026, and non-dilutive proceeds
from the PRV sale; expected operational runway into the second quarter of 2028
CRANBURY, NJ – May 7, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing a
sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the first quarter ended March 31, 2026.
“During the first quarter, we continued advancing our cardiovascular gene therapy portfolio, including reinitiating dosing in the initial three-patient cohort of our
pivotal Phase 2 study of RP-A501 for Danon disease, while also achieving the accelerated approval of KRESLADI for severe LAD-I,” said Gaurav Shah. “The subsequent $180 million non-dilutive monetization of our Rare Pediatric Disease Priority Review
Voucher further strengthened our balance sheet and extended our expected cash runway into the second quarter of 2028. We remain focused on disciplined execution across our deep cardiovascular pipeline and anticipate providing an update on the Danon
program in the second half of the year.”
Recent Pipeline and Operational Updates
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Dosing the initial three-patient cohort for the Phase 2 study of RP-A501 in Danon disease is on track.
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Rocket previously disclosed that the U.S. Food and Drug Administration’s (FDA)
lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease in under three months.
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Per agreement with FDA, three additional patients are being treated at a recalibrated dose of 3.8 × 10¹³ GC/kg with a minimum four-week inter-patient dosing
interval and a modified immunomodulatory regimen. Following the treatment of these three patients, Rocket will align with FDA regarding the completion of the Phase 2
pivotal study.
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Following FDA alignment, Rocket anticipates providing a program update in the second half of 2026.
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Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under
NCT identifier NCT06092034.
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Engagement with FDA is ongoing regarding RP-A601 for PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM).
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Rocket continues to engage with FDA on alignment for a potential pivotal Phase 2 trial design for RP-A601 in PKP2-ACM, while the ongoing Phase 1 study remains
open and actively enrolling to further characterize biological activity across a broader range of disease severity.
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Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT
identifier NCT05885412.
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Phase 1 trial start-up activities are ongoing for RP-A701 in BAG3-associated dilated cardiomyopathy (BAG3-DCM).
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The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary
efficacy of RP-A701 in adults with BAG3-DCM. Dosing of the first patient is anticipated in mid-2026.
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Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT
identifier NCT07137338.
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FDA granted accelerated approval for KRESLADI™ (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
(LAD-I).
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In March 2026, FDA granted KRESLADI™ accelerated approval for the treatment of
pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte
antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant. This indication is approved under accelerated approval based on increase in neutrophil CD18 and CD11a surface expression.
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On April 28, 2026, Rocket announced a definitive agreement to sell its Rare
Pediatric Disease Priority Review Voucher (PRV) for $180 million.
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First Quarter 2026 Financial Results
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Cash position. Cash, cash equivalents and investments as of March
31, 2026, were $144.4 million, excluding PRV monetization.
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R&D expenses. Research and development expenses were $31.5
million for the three months ended March 31, 2026, compared to $35.9 million for the three months ended March 31, 2025. The decrease of $4.4 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $5.8 million, stock-based and
other compensation and benefit expenses of $2.0 million due to decreased R&D headcount, partially offset by increases in clinical trial expenses of $2.8 million and professional fees of $0.6 million. The reduction reflects
disciplined resource allocation following the company’s recent organizational realignment.
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G&A expenses. General and administrative expenses were $17.1
million for the three months ended March 31, 2026, compared to $28.4 million for the three months ended March 31, 2025. The decrease of $11.3 million in G&A expenses was primarily driven by decreases in legal expenses of $5.7 million
due to a legal settlement in 2025, decrease in commercial preparation-related expenses of $4.6 million due to lower headcount and lower spending on commercial launch, and stock-based and other compensation and benefit expenses of $2.8
million due to decreased G&A headcount, partially offset by an increase in milestone related expenses of $2.4 million.
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Net loss. Net loss was $47.6 million or $0.42 per share (basic and
diluted) for the three months ended March 31, 2026, compared to $61.3 million or $0.56 (basic and diluted) for the three months ended March 31, 2025.
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Shares outstanding. 109,123,671 shares of common stock were
outstanding as of March 31, 2026.
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Financial Guidance
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Cash position. As of March 31, 2026, Rocket had cash, cash equivalents
and investments of $144.4 million. Rocket expects such resources, together with proceeds from the sale of the Priority Review Voucher, to be sufficient to fund its operations into the second quarter of 2028.
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About KRESLADITM
KRESLADI™ (marnetegragene autotemcel) is an autologous hematopoietic stem cell-based gene therapy designed to address the underlying genetic cause of severe leukocyte
adhesion deficiency-I (LAD-I), an ultra-rare, life-threatening pediatric immunodeficiency. The therapy utilizes ex vivo lentiviral vector-mediated gene transfer to introduce a functional copy of the ITGB2 gene into a patient’s hematopoietic stem cells, enabling expression of CD18 and restoration of leukocyte adhesion and migration.
KRESLADI is administered as a one-time intravenous infusion following myeloablative conditioning. In clinical studies, treatment with KRESLADI resulted in increased
neutrophil CD18 and CD11a surface expression, supporting the biological activity of the therapy and forming the basis for accelerated approval. Continued approval may be contingent upon verification and description of clinical benefit through
ongoing clinical follow-up and additional post-marketing data collection.
Severe LAD-I is characterized by recurrent, serious bacterial and fungal infections beginning in early infancy and is associated with high early-childhood mortality
without effective treatment.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Serious Infections
Serious infections have occurred with KRESLADI administration. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion.
Serious infections have occurred with KRESLADI administration. Increased susceptibility to infections may occur due to administration of myeloablative conditioning prior to KRESLADI infusion.
Monitor patients for signs and symptoms of infection before and after KRESLADI infusion and treat appropriately. Administer prophylactic antimicrobials
according to institutional guidelines.
Avoid administration of KRESLADI in patients with active bloodstream infections or other serious, untreated infections.
Any blood products required after KRESLADI infusion should be irradiated.
Veno-Occlusive Disease
Veno-occlusive disease has occurred with KRESLADI treatment. Increased susceptibility to veno-occlusive disease may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of veno-occlusive disease including assessment of liver function tests during the first month following KRESLADI infusion.
Veno-occlusive disease has occurred with KRESLADI treatment. Increased susceptibility to veno-occlusive disease may occur due to administration of myeloablative conditioning prior to KRESLADI infusion. Monitor patients for signs and symptoms of veno-occlusive disease including assessment of liver function tests during the first month following KRESLADI infusion.
Neutrophil Engraftment Failure
Neutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 500 cells/microliter obtained on different days by Day 43 after infusion of KRESLADI. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with KRESLADI, provide rescue treatment with the back-up collection of CD34+ cells.
Neutrophil engraftment failure may occur after treatment with KRESLADI. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 500 cells/microliter obtained on different days by Day 43 after infusion of KRESLADI. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with KRESLADI, provide rescue treatment with the back-up collection of CD34+ cells.
Delayed Platelet Engraftment
Delayed platelet engraftment may occur after treatment with KRESLADI. Monitor platelet counts and bleeding until platelet engraftment and platelet
recovery are achieved.
LVV-Mediated Insertional Oncogenesis
Lentiviral vector (LVV)-mediated insertional oncogenesis may occur after treatment with KRESLADI. Hematologic malignancy is a lifelong risk and patients
treated with KRESLADI may develop hematologic malignancy at any time following treatment.
Monitor for hematologic malignancies clinically, and with a complete blood count (with differential) at least annually and integration site analysis as
warranted for at least 15 years after treatment with KRESLADI and as clinically indicated. If malignancy is detected in any patient who received KRESLADI, contact Rocket Pharmaceuticals, Inc. at 1-800-982-2410 for reporting and to obtain
instructions on collection of samples for testing.
Hypersensitivity Reactions
Hypersensitivity reactions including anaphylaxis may occur with the infusion of KRESLADI. The dimethyl sulfoxide (DMSO) in KRESLADI may cause hypersensitivity reactions which may occur in patients with and without prior exposure to DMSO.
Hypersensitivity reactions including anaphylaxis may occur with the infusion of KRESLADI. The dimethyl sulfoxide (DMSO) in KRESLADI may cause hypersensitivity reactions which may occur in patients with and without prior exposure to DMSO.
Monitor patients for signs and symptoms of hypersensitivity reactions during and after KRESLADI infusion. If a hypersensitivity reaction occurs, pause
infusion if ongoing and manage according to clinical practice.
Anti-Retroviral Use
Anti-retroviral medications may interfere with manufacturing of KRESLADI. If a patient requires anti-retrovirals for HIV prophylaxis, mobilization and apheresis of CD34+ cells for KRESLADI manufacturing should be delayed until HIV infection is adequately ruled out. Patients should not take anti-retroviral medications for at least one month prior to mobilization, or for the expected duration required for the elimination of the anti-retroviral medications, and until all cycles of apheresis are completed.
Anti-retroviral medications may interfere with manufacturing of KRESLADI. If a patient requires anti-retrovirals for HIV prophylaxis, mobilization and apheresis of CD34+ cells for KRESLADI manufacturing should be delayed until HIV infection is adequately ruled out. Patients should not take anti-retroviral medications for at least one month prior to mobilization, or for the expected duration required for the elimination of the anti-retroviral medications, and until all cycles of apheresis are completed.
Interference with Serology Testing
Patients who have received KRESLADI are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to LVV provirus insertion resulting in a false-positive test for HIV. Therefore, patients who have received KRESLADI should not be screened for HIV infection using a PCR-based assay.
Patients who have received KRESLADI are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to LVV provirus insertion resulting in a false-positive test for HIV. Therefore, patients who have received KRESLADI should not be screened for HIV infection using a PCR-based assay.
Blood, Organ, Tissue, and Cell Donation
Patients treated with KRESLADI should not donate blood, organs, tissues, or cells for transplantation at any time in the future.
Patients treated with KRESLADI should not donate blood, organs, tissues, or cells for transplantation at any time in the future.
ADVERSE REACTIONS
The most common non-laboratory adverse reactions (≥ 30% of patients) include: mucositis, upper respiratory tract infection, viral infection, febrile
neutropenia, skin lesion, nausea/vomiting, rash/dermatitis, pyrexia, device related infection, and skin infection.
The most common laboratory adverse reactions (≥ 30% of patients) include: hemoglobin decreased, platelet count decreased, neutrophil count decreased,
leukocyte count decreased, aspartate aminotransferase increased, and alanine aminotransferase increased.
For additional safety information, refer to the full Prescribing Information.
DRUG INTERACTIONS
No formal drug interaction studies have been performed. KRESLADI is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug
transporters.
Vaccines
The safety and effectiveness of immunization with live viral vaccines during or following KRESLADI treatment has not been studied. Vaccination is not recommended during the 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following treatment with KRESLADI. Where feasible, administer childhood vaccinations prior to myeloablative conditioning for KRESLADI.
The safety and effectiveness of immunization with live viral vaccines during or following KRESLADI treatment has not been studied. Vaccination is not recommended during the 6 weeks preceding the start of myeloablative conditioning, and until hematological recovery following treatment with KRESLADI. Where feasible, administer childhood vaccinations prior to myeloablative conditioning for KRESLADI.
Anti-retroviral Medications
Patients should not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration for elimination of the medications, and until all cycles of apheresis are completed. Anti-retroviral medications may interfere with manufacturing of KRESLADI.
Patients should not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization and for the expected duration for elimination of the medications, and until all cycles of apheresis are completed. Anti-retroviral medications may interfere with manufacturing of KRESLADI.
REFERENCE TO FULL PRESCRIBING INFORMATION
Please see full Prescribing
Information for KRESLADI.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated biotechnology company advancing gene therapies for rare and devastating cardiovascular diseases, with
additional programs in hematology and immunology. Rocket’s cardiovascular pipeline includes three clinical stage programs that each target one of the major inherited cardiomyopathy subtypes: hypertrophic, arrhythmogenic, and dilated
cardiomyopathies. Together these conditions represent more than 100,000 patients in the U.S. and EU. The Company’s platform is supported by proprietary AAV manufacturing capabilities, multi-year efficacy and safety data in cardiac gene therapy, and
experience treating several cardiac patients across late-stage AAV programs. For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.
Rocket Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as
assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor
provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance
on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of
those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s cash runway and financial position, Rocket’s planned use of proceeds from the monetization of the KRESLADI™ PRV, Rocket’s expectations of
our ability to obtain additional funding to conduct our planned research and development efforts, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory
interactions and planned submissions, Rocket’s plans for the advancement of its cardiovascular AAV programs and KRESLADI™, including its planned pivotal trials, and the safety, effectiveness and timing of related pre-clinical studies and clinical
trials, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates and Rocket’s ability to expand its pipeline to target additional indications that are
compatible with its gene therapy technologies. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those
indicated by these forward-looking statements as a result of various important factors, including, without limitation, the results of Rocket’s ongoing and planned clinical trials, Rocket’s dependence on third parties for development, manufacture,
marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s
ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint
ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring, including extending our cash runway,
Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled "Risk
Factors" in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2025, filed February 26, 2026 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue
reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events
or otherwise.
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Three Months Ended March 31,
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2026
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2025
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Operating expenses:
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Research and development
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$
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31,454
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$
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35,942
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General and administrative
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17,057
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28,446
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Total operating expenses
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48,511
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64,388
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Loss from operations
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(48,511
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)
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(64,388
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)
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Interest expense
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(473
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(472
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)
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Interest and other income, net
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161
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1,336
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Accretion of discount on investments, net
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1,229
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2,190
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Net loss
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$
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(47,594
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)
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$
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(61,334
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)
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Net loss per share - basic and diluted
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$
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(0.42
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)
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$
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(0.56
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)
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Weighted-average common shares outstanding - basic and diluted
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112,134,059
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110,093,461
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||||||
|
March 31, 2026
|
December 31, 2025
|
|||||||
|
Cash, cash equivalents, and investments
|
$
|
144,379
|
$
|
188,929
|
||||
|
Total assets
|
285,407
|
330,449
|
||||||
|
Total liabilities
|
47,368
|
53,228
|
||||||
|
Total stockholders' equity
|
238,039
|
277,221
|
||||||
Investors
Meg Dodge
mdodge@rocketpharma.com
Media
Kevin Giordano
media@rocketpharma.com
FAQ
What were Rocket Pharmaceuticals (RCKT) key financial results for Q1 2026?
Rocket reported a net loss of $47.6 million for Q1 2026, improving from $61.3 million a year earlier. Total operating expenses declined to $48.5 million from $64.4 million, and cash, cash equivalents and investments were $144.4 million as of March 31, 2026.
How much cash runway does Rocket Pharmaceuticals (RCKT) expect after Q1 2026?
Rocket cites pro forma cash, cash equivalents and investments of about $322.6 million, including Q1 balances and PRV sale proceeds. Management expects this to fund operations into the second quarter of 2028, supporting ongoing clinical trials and commercialization efforts.
What is KRESLADI and what milestone did Rocket Pharmaceuticals (RCKT) achieve?
KRESLADI is an autologous gene therapy for severe LAD-I, using lentiviral-modified hematopoietic stem cells to restore leukocyte adhesion. Rocket received FDA accelerated approval, with continued approval contingent on verification of clinical benefit through ongoing follow-up and additional post-marketing data.
How did Rocket Pharmaceuticals (RCKT) use its Rare Pediatric Disease Priority Review Voucher?
Rocket monetized its KRESLADI-related Rare Pediatric Disease Priority Review Voucher for $180 million in non-dilutive proceeds. This transaction contributed to pro forma cash of $322.6 million and helped extend the company’s expected operational runway into the second quarter of 2028.
What are the main risks and side effects associated with KRESLADI?
Key risks highlighted include serious infections, veno-occlusive disease, neutrophil and platelet engraftment failure, and potential LVV-mediated insertional oncogenesis. Common adverse reactions include mucositis, infections, febrile neutropenia, rashes, fever, and cytopenias. Long-term monitoring for hematologic malignancies is recommended.
What pipeline progress did Rocket Pharmaceuticals (RCKT) report alongside Q1 2026 results?
Rocket noted reinitiated dosing in the initial cohort of its pivotal Phase 2 RP-A501 trial for Danon disease. The company also plans first patient dosing in a Phase 1 RP-A701 study for BAG3-related dilated cardiomyopathy around mid-2026 and expects a Danon program update in the second half of the year.