Rocket Pharmaceuticals: $26.4M nine-month net income
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Form Type
8-K
Rhea-AI Filing Summary
Rocket Pharmaceuticals, Inc. (RCKT) reported a third-quarter 2026 net loss of $49.234 million, or $0.43 per share, compared with a $50.332 million loss, or $0.45 per share, a year earlier. For the nine months, net income was $26.386 million, including a $178.190 million gain from sale of a PRV, versus a $180.585 million loss.
Cash, cash equivalents and investments totaled $279.4 million at September 30, 2026. Under its current operating plan, Rocket expects that balance to fund operations into the third quarter of 2028. Its Hercules credit facility provides up to $150 million; $35 million was received at closing. Additional borrowings could extend runway into 2029, subject to borrowing conditions, draw timing and amounts, and operating expenditures.
FDA alignment on RP-A501 establishes a 12-patient pivotal Phase 2 efficacy population; Rocket expects dosing to finish by mid-2027 and topline results in mid-2028. RP-A701 Phase 1 dosing began, and the European Commission granted it orphan designation on October 20, 2026. KRESLADI reached commercial readiness on October 1, 2026, with two Qualified Treatment Centers activated and ready to begin patient onboarding.
1 point · 0 major0 major · 0 points
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Positive
- Moderate pointNine-month net income was $26.386 million versus a $180.585 million loss.
Negative
- None.
Filing Explained
Further Hercules draws: thirty-five million at Rocket’s option; thirty million on a specified Danon clinical milestone; fifty million subject to Hercules investment committee approval.
The release spells out the efficacy success bar for the FDA-aligned RP-A501 pivotal Phase 2 trial: at the 12-month assessment, at least 7 of 12 patients must meet both co-primary endpoints. Those endpoints require myocardial LAMP2 expression at Grade 1 or higher and at least a
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, or exhibit attachments filed with this report.
Key Figures
Net loss: $49.234 million
Loss per share: $0.43 per share (basic and diluted)
Nine-month net income: $26.386 million
+5 more
8 metrics
Net loss
$49.234 million
Three months ended September 30, 2026
Loss per share
$0.43 per share (basic and diluted)
Three months ended September 30, 2026
Nine-month net income
$26.386 million
Nine months ended September 30, 2026; includes a $178.190 million gain from sale of a PRV
Gain from sale of PRV
$178.190 million
Nine months ended September 30, 2026
Research and development expenses
$31.172 million
Three months ended September 30, 2026
Cash, cash equivalents and investments
$279.4 million
As of September 30, 2026
Hercules credit facility
Up to $150 million
$35 million received at closing
Expected cash runway
Into the third quarter of 2028
Based on the current operating plan and the balance as of September 30, 2026
Key Terms
co-primary endpoints, accelerated approval pathway, orphan designation, myeloablative conditioning, +2 more
6 terms
co-primary endpoints medical
"established 12-month co-primary endpoints"
Co-primary endpoints are two or more key outcomes that a clinical study must meet simultaneously to be considered successful, like needing both improved symptoms and a marker of disease reduction. For investors, they matter because meeting multiple agreed goals strengthens the evidence that a treatment works and can influence regulatory approval, market access and commercial potential—similar to a product needing to pass both safety and performance checks before launch.
accelerated approval pathway regulatory
"intended to support a potential accelerated approval pathway"
The accelerated approval pathway is a process that allows new medicines to be approved more quickly based on early evidence that they may be effective, rather than waiting for full proof. This can help patients access promising treatments faster, but it also means ongoing studies are needed to confirm the benefits. For investors, it highlights potential faster market entry and earlier revenue opportunities, along with some uncertainty about long-term outcomes.
orphan designation regulatory
"granted orphan designation to RP-A701"
Orphan designation is a regulatory label granted to a drug or therapy intended to treat a rare disease, which typically affects a small number of patients. It matters to investors because it unlocks incentives—such as development cost reductions, tax credits, priority review and a period of market exclusivity—that improve the commercial and regulatory prospects of a product; think of it as a government-backed head start and safety net for a niche but potentially high-value medicine.
myeloablative conditioning medical
"administration of myeloablative conditioning prior to KRESLADI infusion"
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.
lentiviral vector-mediated gene transfer medical
"ex vivo lentiviral vector-mediated gene transfer"
LVV-mediated insertional oncogenesis medical
"LVV-mediated insertional oncogenesis may occur"
Earnings Snapshot
Net loss: $49.234 million · Guidance included
Third quarter 2026
Net loss
$49.234 million
Compared with a net loss of $50.332 million for the three months ended September 30, 2025.
Loss per share (basic and diluted)
$0.43 loss per share
Compared with $0.45 loss per share for the three months ended September 30, 2025.
Research and development expenses
$31.172 million
Compared with $34.068 million in the third quarter of 2025; decrease of $2.9 million.
General and administrative expenses
$19.793 million
Compared with $18.351 million in the third quarter of 2025; increase of $1.4 million.
Cash, cash equivalents and investments
$279.4 million
As of September 30, 2026.
Guidance
Based on its current operating plan, Rocket expects cash, cash equivalents and investments of $279.4 million as of September 30, 2026, to fund operations into the third quarter of 2028.
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What was Rocket Pharmaceuticals (RCKT)'s third-quarter 2026 net loss?
RCKT reported a net loss of $49.234 million, or $0.43 per share, for the three months ended September 30, 2026.
How much could Rocket draw under the Hercules credit facility, and what are the conditions?
The facility provides up to $150 million, with $35 million received at closing. Rocket may draw another $35 million at its option during specified periods, subject to the agreement's terms and conditions. An additional $30 million may become available upon achievement of a specified Danon clinical milestone, and $50 million is subject to Hercules' investment committee approval.
What criteria apply to Rocket's RP-A501 pivotal Phase 2 efficacy assessment?
At the 12-month assessment, at least seven of 12 patients must show both myocardial LAMP2 expression at Grade 1 or higher and a decrease of 10% or more from baseline in left ventricular mass index. The co-primary endpoints are intended to support a potential accelerated approval pathway.
AI-generated analysis. How Rhea-AI works. Not financial advice.
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): November 12, 2026
(Exact name of registrant as specified in its charter)
| | | |
| (State or other jurisdiction of incorporation) | (Commission File Number) | (IRS Employer Identification No.) |
| | | |
| (Address of principal executive offices) | | (Zip Code) |
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):
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
| | | |
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. ☐
Item 2.02.
Results of Operations and Financial Condition.
On November 12, 2026, Rocket Pharmaceuticals, Inc. (the “Company”) announced certain financial information for the quarter ended September 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.
Item 9.01.
Financial Statements and Exhibits.
(d)
Exhibits.
|
99.1
|
Press Release of Rocket Pharmaceuticals, Inc. dated November 12, 2026.
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
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.
|
|
Rocket Pharmaceuticals, Inc.
|
|
|
|
|
|
|
Date: November 12, 2026
|
By:
|
/s/ Martin Wilson
|
|
|
|
Martin Wilson
|
|
|
|
General Counsel and Chief Corporate Officer
|
Exhibit 99.1
Rocket Pharmaceuticals Reports Third Quarter 2026 Financial Results and
Highlights Recent Progress
FDA alignment establishes path to complete the pivotal Phase 2 trial of RP-A501 in Danon disease; dosing completion expected by mid-2027
October Danon disease investor webinar highlighted updated longer-term RP-A501 Phase 1 findings and encouraging initial safety observations in the first three patients treated under the modified Pivotal Phase 2 protocol
Dosing initiated in the Phase 1 trial of RP-A701 for BAG3-associated dilated cardiomyopathy
KRESLADI™ achieved commercial readiness on October 1, 2026; Qualified Treatment Centers activated and ready to begin commercial patient onboarding
Cash, cash equivalents and investments of approximately $279.4 million as of September 30, 2026, including the initial $35 million Hercules draw, are expected to fund planned operations into Q3 2028. Additional borrowings could extend runway into 2029, subject to borrowing conditions, draw timing and amounts, and operating expenditures.
CRANBURY, NJ – November 12, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and life-threatening diseases, with a strategic focus on inherited cardiovascular disorders, today reported financial results for the third quarter ended September 30, 2026, and provided recent business updates.
“With FDA alignment on the pivotal RP-A501 trial in Danon disease, our priority is to complete dosing by mid-2027,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “We are also advancing our broader cardiovascular portfolio, with dosing initiated in the Phase 1 trial of RP-A701 for BAG3-DCM and continued FDA engagement on a potential pivotal study of RP-A601 in PKP2-ACM. Commercial readiness for KRESLADI and activation of our first Qualified Treatment Centers bring us closer to treating eligible children. Our financial resources and the additional flexibility provided by the Hercules facility support these priorities as we work toward our next clinical and commercial milestones.”
Recent Pipeline and Operational Updates
•
FDA Alignment Establishes Path to Complete RP-A501 Pivotal Phase 2 Trial in Danon Disease
o
On September 15, 2026, Rocket announced FDA alignment on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 in Danon disease, including the recalibrated dose, 12-patient pivotal population and established 12-month co-primary endpoints. The first three patients treated under the modified protocol count toward the 12-patient pivotal efficacy population. At the 12-month assessment, at least seven of the 12 patients must show both myocardial LAMP2 expression at Grade 1 or higher and a decrease of 10% or more from baseline in left ventricular mass index to meet the pivotal efficacy success criterion. The co-primary endpoints are intended to support a potential accelerated approval pathway.
o
On October 6, Rocket hosted an investor webinar, highlighting key findings and development milestones for RP-A501, including updated longer-term Phase 1 follow-up and encouraging initial safety observations in the first three patients treated under the modified protocol. Rocket reviewed updated Danon disease epidemiology, with independent genotype- and phenotype-based analyses estimating 10,000-11,000 people living with Danon disease in the U.S., including approximately 4,000 males. Rocket has identified more than 900 Danon patients. Prevalence estimates may include undiagnosed or asymptomatic individuals and do not indicate treatment eligibility.
o
Rocket expects to complete pivotal trial dosing by mid-2027, with topline results anticipated in mid-2028.
•
Continued Advancement of RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
o
Rocket continues to advance RP-A601 for PKP2-ACM through its ongoing Phase 1 study and engagement with the FDA regarding the next stage of clinical development. Previously reported Phase 1 findings showed increased PKP2 protein expression and improved desmosomal localization, together with directional improvements in arrhythmia measures and right ventricular function. The Company will provide further clinical and regulatory updates as the program progresses.
o
The ongoing Phase 1 study remains open and actively enrolling to further characterize biological activity across a broader range of disease severity.
o
Details of the Phase 1 study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.
•
Dosing Initiated in Phase 1 Trial of RP-A701 for BAG3-Associated Dilated Cardiomyopathy (BAG3-DCM); EMA Orphan Designation Granted
o
Rocket has initiated dosing in the Phase 1 trial of RP-A701 for BAG3-DCM, marking an additional clinical milestone for Rocket’s cardiovascular gene therapy portfolio.
o
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.
o
On October 20, 2026, the European Commission granted orphan designation to RP-A701 for the treatment of BAG3-DCM.
•
KRESLADI™ (marnetegragene autotemcel) Achieves Commercial Readiness; Qualified Treatment Centers Activated
o
Rocket achieved commercial readiness for KRESLADI™ on October 1, 2026. Two Qualified Treatment Centers have been activated and ready to begin commercial patient onboarding, and Rocket's external manufacturing network is in place.
•
Strategic Credit Facility Provides Access to Up to $150 Million to Support Cardiovascular Pipeline
o
On October 6, 2026, Rocket announced a credit facility agreement with Hercules Capital, Inc. for up to $150 million to support execution of the pivotal Phase 2 trial of RP-A501 in Danon disease and continued advancement of its broader cardiovascular pipeline. Rocket received $35 million at closing and may draw an additional $35 million at its option during specified periods, subject to the agreement’s terms and conditions. An additional $30 million may become available upon achievement of a specified Danon clinical milestone, and an additional $50 million is subject to Hercules’ investment committee approval.
Third Quarter 2026 Financial Results
•
Cash position. Cash, cash equivalents and investments as of September 30, 2026, were $279.4 million.
•
R&D expenses. Research and development expenses were $31.2 million for the three months ended September 30, 2026, compared to $34.1 million for the three months ended September 30, 2025. The decrease of $2.9 million in R&D expenses was primarily driven by reductions in stock-based and other compensation and benefits expense of $3.5 million due to decreased R&D headcount, decreases in manufacturing and development and direct material costs of $1.9 million and depreciation expenses of $1.2 million due to decreased asset base. These decreases were partially offset by increases in expenses for laboratory-related expenses of $1.4 million, professional fees of $1.4 million, and clinical trial expenses of $0.6 million.
•
G&A expenses. General and administrative expenses were $19.8 million for the three months ended September 30, 2026, compared to $18.4 million for the three months ended September 30, 2025. The increase of $1.4 million in G&A expenses was primarily driven by increases in post marketing commitment expenses of $3.4 million and commercial preparation related expenses of $0.6 million. These increases were partially offset by decreases in stock-based and other compensation and benefit expenses of $2.2 million due to decreased G&A headcount and legal expenses of $0.6 million.
•
Other Income. Other Income was $1.8 million for the three months ended September 30, 2026, compared to $1.9 million for the three months ended September 30, 2025. The decrease in other income was primarily driven by a decline in accretion of discount on investments, net, of $0.4 million partially offset by an increase in interest income of $0.3 million.
•
Income Taxes. Income taxes were $0.1 million for the three months ended September 30, 2026, compared to $0.0 million for the three months ended September 30, 2025, because of a change in estimated income taxes payable during the three months ended September 30, 2026.
•
Net income. Net loss was $49.2 million or $0.43 per share (basic and diluted) for the three months ended September 30, 2026, compared to $50.3 million or $0.45 (basic and diluted loss per share) for the three months ended September 30, 2025.
•
Shares outstanding. 111,386,536 shares of common stock were outstanding as of September 30, 2026.
Financial Guidance
•
Cash position. As of September 30, 2026, Rocket had cash, cash equivalents and investments of $279.4 million. Based on its current operating plan, Rocket expects its cash, cash equivalents and investments as of September 30, 2026, to fund operations into the third quarter of 2028.
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 life-threatening 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 adeno-associated virus (AAV) manufacturing capabilities and extensive clinical experience in cardiac gene therapy.
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 actual results to differ materially from those expressed or implied by such forward-looking statements. Rocket makes such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other applicable federal securities laws. All statements other than statements of historical fact contained in this release are forward-looking statements. These statements may be identified by words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may,” “potential” and similar expressions, or their negatives. These forward-looking statements include, but are not limited to, statements concerning Rocket’s plans to advance its genetic medicines portfolio; the enrollment, dosing, completion and anticipated results of ongoing and planned clinical trials; the timing and outcome of regulatory interactions and potential approval pathways; estimated prevalence of Danon disease, patient identification efforts and the size of addressable patient populations; KRESLADI commercialization and patient access; expected cash runway and the sufficiency of financial resources; the availability of additional borrowings under the Hercules credit facility and satisfaction of applicable borrowing conditions; and Rocket’s development strategy and priorities.
Although Rocket believes that the expectations reflected in these forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially as a result of various important factors, including, without limitation: the results, timing and costs of Rocket’s ongoing and planned clinical trials; its ability to identify, enroll and dose eligible patients and successfully complete clinical studies; unexpected adverse events or other safety findings; the timing and outcome of regulatory interactions, submissions and review; manufacturing, product comparability, quality and supply considerations; Rocket’s dependence on third parties for development, manufacturing, commercialization and other activities; its ability to successfully commercialize its products; its future capital requirements and ability to obtain additional funding; its ability to satisfy conditions for additional borrowings, comply with credit facility covenants and meet debt service obligations; changes in its operating plan, development priorities, expenses or cash requirements; its ability to obtain, maintain and enforce intellectual property protection and defend against third-party infringement claims; and competing therapies and technological developments. Additional risks are described under “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission on February 26, 2026, and in its subsequent SEC filings, including its Quarterly Reports on Form 10-Q.
Accordingly, readers 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, except as required by law.
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2026 | 2025 | 2026 | 2025 | |||||||||||||
|
Operating expenses:
|
||||||||||||||||
|
Research and development
|
$ | 31,172 | $ | 34,068 | $ | 92,123 | $ | 112,668 | ||||||||
|
General and administrative
|
19,793 | 18,351 | 54,275 | 71,817 | ||||||||||||
|
Restructuring
|
- | (172 | ) | - | 3,299 | |||||||||||
|
Total operating expenses
|
50,965 | 52,247 | 146,398 | 187,784 | ||||||||||||
|
Loss from operations
|
(50,965 | ) | (52,247 | ) | (146,398 | ) | (187,784 | ) | ||||||||
|
Gain from sale of PRV
|
- | - | 178,190 | - | ||||||||||||
|
Interest expense
|
(473 | ) | (473 | ) | (1,419 | ) | (1,418 | ) | ||||||||
|
Interest and other income, net
|
1,014 | 709 | 1,522 | 2,528 | ||||||||||||
|
Accretion of discount on investments, net
|
1,244 | 1,679 | 3,166 | 6,089 | ||||||||||||
|
(Loss) income before Income Taxes
|
(49,180 | ) | (50,332 | ) | 35,061 | (180,585 | ) | |||||||||
|
Provision for Income Taxes
|
(54 | ) | - | (8,675 | ) | - | ||||||||||
|
Net (loss) income
|
$ | (49,234 | ) | $ | (50,332 | ) | $ | 26,386 | $ | (180,585 | ) | |||||
|
Net (loss) income per share - basic
|
$ | (0.43 | ) | $ | (0.45 | ) | $ | 0.23 | $ | (1.63 | ) | |||||
|
Net (loss) income per share - diluted
|
$ | (0.43 | ) | $ | (0.45 | ) | $ | 0.23 | $ | (1.63 | ) | |||||
|
Weighted-average common shares outstanding - basic
|
113,647,401 | 111,571,136 | 112,880,990 | 110,900,161 | ||||||||||||
|
Weighted-average common shares outstanding - diluted
|
113,647,401 | 111,571,136 | 114,706,711 | 110,900,161 | ||||||||||||
|
| September 30, 2026 | December 31, 2025 | ||||||
|
Cash, cash equivalents, and investments
|
$ | 279,445 | $ | 188,929 | ||||
|
Total assets
|
419,504 | 330,449 | ||||||
|
Total liabilities
|
92,780 | 53,228 | ||||||
|
Total stockholders' equity
|
326,724 | 277,221 | ||||||
Investors & Media
Meg Dodge
mdodge@rocketpharma.com
Brooke Schuster
bschuster@rocketpharma.com