Rocket Pharmaceuticals (RCKT) turns Q2 profit on $178M voucher gain and boosts cash
Rhea-AI Filing Summary
Rocket Pharmaceuticals, Inc. reported a sharp improvement in results for the quarter ended June 30, 2026, driven by U.S. FDA approval of KRESLADI™ and monetization of the associated Priority Review Voucher. Second-quarter net income was $123.2 million, compared with a net loss of $68.9 million a year earlier, helped by a $178.2 million gain from the sale of the voucher. Operating expenses declined to $46.9 million from $71.1 million, reflecting lower research and development and general and administrative costs following a prior restructuring.
For the first six months of 2026, Rocket reported net income of $75.6 million versus a net loss of $130.3 million in the prior-year period. Cash, cash equivalents and investments increased to $283.7 million as of June 30, 2026, from $188.9 million at year-end 2025, and the company expects its cash runway to extend into the second quarter of 2028. Rocket highlighted completion of safety observation for the first three Danon disease patients in its modified pivotal Phase 2 trial, continued FDA interactions on the path to rapidly completing this trial, and multiple planned second-half 2026 milestones across its cardiovascular gene therapy programs, including PKP2-ACM and BAG3-DCM, alongside commercial preparations for KRESLADI.
Positive
- Net income of $123.2 million in Q2 2026 versus a net loss of $68.9 million in Q2 2025, driven largely by the gain on sale of the Priority Review Voucher.
- Gain of $178.2 million from sale of the Priority Review Voucher provided substantial non-operating income and strengthened the company’s financial position.
- Cash, cash equivalents and investments of $283.7 million at June 30, 2026, up from $188.9 million at December 31, 2025, with an expected cash runway into the second quarter of 2028.
- Total operating expenses fell to $46.9 million in Q2 2026 from $71.1 million in Q2 2025, reflecting reduced R&D and G&A spending after prior restructuring.
- FDA approval of KRESLADI™ and commercial preparations for launch in the fourth quarter of 2026 add a commercial product to complement the pipeline.
Negative
- Loss from operations remained $46.9 million in Q2 2026, indicating underlying operating activities are still unprofitable despite the one-time voucher sale gain.
Insights
Analyzing...
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 income Q2 2026: $123,214
Net loss Q2 2025: $68,919
Gain from sale of PRV: $178,190
+5 more
8 metrics
Net income Q2 2026
$123,214
Three months ended June 30, 2026 net income
Net loss Q2 2025
$68,919
Three months ended June 30, 2025 net loss
Gain from sale of PRV
$178,190
Gain from sale of Priority Review Voucher in Q2 2026
Total operating expenses Q2 2026
$46,922
Three months ended June 30, 2026 total operating expenses
Cash, cash equivalents and investments
$283,747
Balance at June 30, 2026
Cash, cash equivalents and investments
$188,929
Balance at December 31, 2025
Total stockholders’ equity
$368,830
Balance at June 30, 2026
Weighted-average shares basic Q2 2026
112,844,880
Basic weighted-average common shares outstanding Q2 2026
Key Terms
Priority Review Voucher, pivotal Phase 2 trial, neutrophil engraftment failure, LVV-mediated insertional oncogenesis, +2 more
6 terms
Priority Review Voucher regulatory
"sale of the Priority Review Voucher generating $180 million in non-dilutive capital"
A priority review voucher is a transferable regulatory incentive that lets a company move a future drug or device application to the front of the review line, shortening the review period by several months. For investors it matters because the voucher can speed up market access for a high-value product or be sold to other companies for significant cash, acting like a tradable fast-pass that can accelerate revenue or create immediate financial upside.
pivotal Phase 2 trial medical
"modified pivotal Phase 2 protocol completed the protocol-defined safety observation period"
A pivotal phase 2 trial is a mid-stage clinical study designed to provide strong, convincing evidence about a drug’s safety and effectiveness that regulators can use to decide on further approvals or larger trials. For investors, it matters because positive results can sharply increase the likelihood of regulatory success and future sales—similar to a product prototype winning a major usability test that clears the way for full production and investment.
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.
LVV-mediated insertional oncogenesis medical
"Lentiviral vector (LVV)-mediated insertional oncogenesis may occur after treatment with KRESLADI."
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.
hematopoietic stem cell-based gene therapy medical
"is an autologous hematopoietic stem cell-based gene therapy designed to address the underlying genetic cause"
Earnings Snapshot
Net income: $123,214 · Guidance included
quarter ended June 30, 2026
Net income
$123,214
compared with net loss of $68,919 in the prior-year quarter
Total operating expenses
$46,922
down from $71,149 in the prior-year quarter
Six-month net income (loss)
$75,620
compared with net loss of $130,253 in the prior-year period
Cash, cash equivalents and investments
$283,747
up from $188,929 at December 31, 2025
Guidance
The company expects its cash runway to extend into the second quarter of 2028.
AI-generated analysis. How Rhea-AI works. Not financial advice.
FAQ
How did Rocket Pharmaceuticals (RCKT) perform financially in Q2 2026?
Rocket reported net income of $123.2 million for Q2 2026, compared with a net loss of $68.9 million in Q2 2025. The improvement was mainly due to a $178.2 million gain from selling a Priority Review Voucher and lower operating expenses.
What is Rocket Pharmaceuticals’ (RCKT) current cash position and runway?
As of June 30, 2026, Rocket held $283.7 million in cash, cash equivalents and investments, up from $188.9 million at year-end 2025. The company expects this to provide a cash runway into the second quarter of 2028.
How did operating expenses change for Rocket Pharmaceuticals (RCKT) in Q2 2026?
Total operating expenses were $46.9 million in Q2 2026, down from $71.1 million in Q2 2025. Both research and development and general and administrative costs declined, reflecting a more focused operating structure after a 2025 restructuring.
What drove Rocket Pharmaceuticals’ (RCKT) profit in the first half of 2026?
For the first six months of 2026, Rocket generated net income of $75.6 million versus a $130.3 million loss a year earlier. A key contributor was the $178.2 million gain from selling a Priority Review Voucher tied to KRESLADI’s approval.
What clinical progress did Rocket Pharmaceuticals (RCKT) report in Danon disease?
All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period. Rocket reported no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns in these patients.
What are Rocket Pharmaceuticals’ (RCKT) key upcoming milestones in 2026?
For the second half of 2026, Rocket expects a broader Danon disease program update, a regulatory update for the planned RP-A601 pivotal study in PKP2-ACM, and initial patient dosing with RP-A701 in BAG3-DCM, along with KRESLADI commercial launch preparations.
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): August 10, 2026
(Exact name of registrant as specified in its charter)
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Registrant’s telephone number, including area code: (609 )
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(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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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
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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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Results of Operations and Financial Condition.
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On August 10, 2026, Rocket Pharmaceuticals, Inc. (the “Company”) announced certain financial information for the quarter ended June 30, 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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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 August 10, 2026.
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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: August 10, 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 Second Quarter 2026 Financial Results and
Highlights Recent Progress
All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period; no thrombotic
microangiopathy, capillary leak syndrome, or other significant safety concerns observed
Rocket is actively engaging with the FDA to align on the path to rapidly completing the pivotal Phase 2 trial in Danon disease
Multiple clinical and regulatory milestones remain on track for the second half of 2026, including a broader Danon disease program update, a regulatory update for the
planned RP-A601 pivotal study in PKP2-ACM and initial patient dosing with RP-A701 in BAG3-DCM
FDA approval of KRESLADI™ and subsequent monetization of the Priority Review Voucher generated $180 million in non-dilutive capital; focused preparations are underway
for commercial availability and patient onboarding beginning in the fourth quarter of 2026
Cash, cash equivalents and investments of approximately $283.7 million; expected cash runway into the second quarter of 2028
CRANBURY, NJ – August 10, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing a sustainable pipeline of genetic medicines for rare disorders
with high unmet need, today reported financial and recent operational results for the second quarter ended June 30, 2026.
“The second quarter of 2026 materially strengthened Rocket’s position, with the approval of KRESLADI demonstrating our ability to advance a genetic medicine through
regulatory approval, the subsequent sale of the Priority Review Voucher generating $180 million in non-dilutive capital, and safety clearance for the first three Danon disease patients in our modified pivotal trial,” said Gaurav Shah, M.D., Chief
Executive Officer of Rocket Pharmaceuticals. “Together with the more focused operating structure we implemented in late 2025, these achievements provide a stronger financial and executional foundation for advancing our cardiovascular pipeline. Based
on the positive safety observations in Danon disease, we are working with the FDA on the path to treating additional patients and completing the pivotal trial as quickly as possible, while progressing toward additional regulatory and clinical
milestones across the remainder of our cardiac portfolio, including PKP2-ACM and BAG3-DCM.”
Recent Pipeline and Operational Updates
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Positive Initial RP-A501 Safety Findings Support Regulatory Engagement on Next Steps in Danon Disease
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Rocket previously disclosed that the initial three patients were safely dosed under the modified pivotal Phase 2 protocol for RP-A501 in Danon disease. All three patients have completed the
protocol-defined safety observation period, with no thrombotic microangiopathy (TMA), capillary leak syndrome, or other significant safety concerns observed at the recalibrated dose with the refined immunomodulatory regimen.
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The recalibrated Phase 2 dose of 3.8 × 10¹³ GC/kg was selected based on the characteristics of the current drug product, the underlying biology of Danon disease and the Phase 1 experience, with the
objective of preserving the potential for meaningful clinical benefit while optimizing RP-A501’s benefit-risk profile.
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Rocket is actively engaging with the FDA to seek alignment on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory
pathway in the second half of 2026.
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Rocket is on track to host an investor webinar in the second half of 2026 to provide a comprehensive Danon disease program update.
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In parallel with the pivotal Phase 2 trial, Rocket’s global natural history study has enrolled more than 50 patients, including both males and females, building an increasingly robust longitudinal
dataset to further characterize Danon disease.
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Continued FDA Engagement and Phase 1 Enrollment for RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
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Rocket continues to engage with the FDA regarding alignment on the design of a potential pivotal study of RP-A601 in PKP2-ACM with regulatory update expected in the second half of 2026. Previously
reported Phase 1 findings demonstrated increased PKP2 protein expression and improved desmosomal localization, together with directional improvements in arrhythmia measures and right ventricular function.
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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 Initiation Anticipated in 2026 for RP-A701 in BAG3-Associated Dilated Cardiomyopathy (BAG3-DCM)
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Patient screening and enrollment has begun, with initial patient dosing anticipated in the second half of 2026.
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The multicenter, dose-escalation study is designed to evaluate the safety, biological activity and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Details of the Phase 1 study can be found
at www.ClinicalTrials.gov under NCT identifier NCT07137338.
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Focused Commercialization Strategy Underway for KRESLADI™ (marnetegragene autotemcel) Following FDA Approval
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Following the announcement of the FDA’s accelerated approval of KRESLADI™ for severe leukocyte adhesion deficiency-I (LAD-I), Rocket is implementing a focused commercialization strategy
calibrated to the ultra-rare patient population and concentrated treatment landscape.
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The strategy is centered on a limited network of specialized Qualified Treatment Centers and an appropriately scaled commercial infrastructure, supporting patient access while maintaining disciplined
investment.
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KRESLADI represents Rocket’s first FDA-approved product and demonstrates the Company’s ability to advance a complex genetic medicine through clinical development, CMC, regulatory review, and
commercial readiness. Rocket anticipates commercial availability and the start of patient onboarding in the fourth quarter of 2026.
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On June 12, 2026, Rocket announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $180 million. This substantial non-dilutive
capital strengthened Rocket’s financial position and supports the continued advancement of its prioritized cardiovascular pipeline.
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Second Quarter 2026 Financial Results
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Cash position. Cash, cash equivalents and investments as of
June 30, 2026, were $283.7 million. The cash position reflects the proceeds from the sale of the Rare Pediatric Disease Priority Review Voucher.
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R&D expenses. Research and development expenses were
$29.5 million for the three months ended June 30, 2026, compared to $42.7 million for the three months ended June 30, 2025. The decrease of $13.2 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $7.5 million and stock-based and other compensation and benefit
expenses of $3.6 million due to decreased R&D headcount, depreciation expenses of $1.0 million due to decreased asset base, and clinical trial expenses of $1.0 million.
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G&A expenses. General and administrative expenses were
$17.4 million for the three months ended June 30, 2026, compared to $25.0 million for the three months ended June 30, 2025. The decrease of $7.6 million in G&A expenses was primarily driven by decreases in commercial preparation-related
expenses of $4.8 million due to lower headcount and lower spending on commercial preparation, stock-based and other compensation and benefit expenses of $1.9 million due to decreased G&A headcount, and legal expenses of $1.4 million.
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Other Income. Other Income was $178.8 million for the three
months ended June 30, 2026, compared to $2.2 million for the three months ended June 30, 2025. The increase in other income was primarily driven by the sale of the Rare Pediatric Disease Priority Review Voucher for net $178.2 million. The
increase was partially offset by a decline in accretion of discount on investments, net, of $1.5 million due to lower investment balance and interest rates year over year.
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Income Taxes. Income taxes were $8.6 million for the three
months ended June 30, 2026, compared to $0.0 million for the three months ended June 30, 2025, as a result of generating net income in the three months ended June 30, 2026.
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Net income. Net income was $123.2 million or $1.09 of basic
income per share and $1.08 of diluted income per share for the three months ended June 30, 2026, compared to a net loss of $68.9 million or $0.62 (basic and diluted loss per share) for the three months ended June 30, 2025.
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Shares outstanding. 109,539,424 shares of common stock were
outstanding as of June 30, 2026.
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Financial Guidance
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Cash position. As of June 30, 2026, Rocket had cash, cash
equivalents and investments of $283.7 million. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of June 30, 2026, to
fund 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.
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.
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.
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.
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.
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.
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.
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.
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.
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 commercial-stage biotechnology company developing genetic medicines for rare and devastating diseases, with a strategic
focus on inherited cardiovascular disorders and additional programs in hematology and immunology. Rocket's cardiovascular portfolio includes three clinical-stage gene therapy programs targeting hypertrophic, arrhythmogenic, and dilated
cardiomyopathies, together representing one of the broadest pipelines focused on inherited heart disease. The Company's integrated platform combines proprietary AAV manufacturing capabilities, extensive clinical experience in cardiac gene therapy,
and long-term safety and efficacy data supporting advancement across its cardiovascular portfolio.
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 safety and effectiveness of RP-A501 in its pivotal Phase 2 trial under the modified protocol, the Company's ability to align with the FDA on the path to
treating additional Danon disease patients, 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 June 30,
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Six Months Ended June 30,
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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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29,497
|
$
|
42,658
|
$
|
60,951
|
$
|
78,600
|
||||||||
|
General and administrative
|
17,425
|
25,020
|
34,482
|
53,466
|
||||||||||||
|
Restructuring
|
-
|
3,471
|
-
|
3,471
|
||||||||||||
|
Total operating expenses
|
46,922
|
71,149
|
95,433
|
135,537
|
||||||||||||
|
Loss from operations
|
(46,922
|
)
|
(71,149
|
)
|
(95,433
|
)
|
(135,537
|
)
|
||||||||
|
Gain from sale of PRV
|
178,190
|
-
|
178,190
|
-
|
||||||||||||
|
Interest expense
|
(473
|
)
|
(473
|
)
|
(946
|
)
|
(945
|
)
|
||||||||
|
Interest and other income, net
|
347
|
483
|
508
|
1,819
|
||||||||||||
|
Accretion of discount on investments, net
|
693
|
2,220
|
1,922
|
4,410
|
||||||||||||
|
Earnings (losses) before Income Taxes
|
131,835
|
(68,919
|
)
|
84,241
|
(130,253
|
)
|
||||||||||
|
Provision for Income Taxes
|
(8,621
|
)
|
-
|
(8,621
|
)
|
-
|
||||||||||
|
Net income (loss)
|
$
|
123,214
|
$
|
(68,919
|
)
|
$
|
75,620
|
$
|
(130,253
|
)
|
||||||
|
Net income (loss) per share - basic
|
$
|
1.09
|
$
|
(0.62
|
)
|
$
|
0.67
|
$
|
(1.18
|
)
|
||||||
|
Net income (loss) per share - diluted
|
$
|
1.08
|
$
|
(0.62
|
)
|
$
|
0.66
|
$
|
(1.18
|
)
|
||||||
|
Weighted-average common shares outstanding - basic
|
112,844,880
|
111,019,647
|
112,491,433
|
110,559,113
|
||||||||||||
|
Weighted-average common shares outstanding - diluted
|
114,513,964
|
111,019,647
|
114,329,763
|
110,559,113
|
||||||||||||
|
June 30, 2026
|
December 31, 2025
|
|||||||
|
Cash, cash equivalents, and investments
|
$
|
283,747
|
$
|
188,929
|
||||
|
Total assets
|
423,098
|
330,449
|
||||||
|
Total liabilities
|
54,268
|
53,228
|
||||||
|
Total stockholders' equity
|
368,830
|
277,221
|
||||||
Investors & Media
Meg Dodge
mdodge@rocketpharma.com