On May 15, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA"), a Phase 3 immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported financial results for the Company’s first quarter ended March 31, 2025, and provided a corporate update (Press release, TuHURA Biosciences, MAY 15, 2025, View Source [SID1234653183]).

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"TuHURA has had an impressive start to 2025, and we continue to execute our corporate strategy as we drive towards TuHURA’s four major clinical data readouts anticipated over the next 24 months. We recently initiated our Phase 1b/2a study of IFx-Hu2.0 in combination with pembrolizumab in checkpoint-naïve MCCUP patients. Evaluating IFx-Hu2.0 in MCCUP patients is an important component of our overall strategy for the program, as the Phase 1b/2a trial will include patients without skin lesions who present with metastatic deep-seated tumors in the liver, lungs or retroperitoneum (abdomen), who are not eligible to participate in our Phase 3 accelerated approval trial due to the trial’s primary lesion enrollment criteria. Approximately 30% of MCC patients have unknown primary lesions, and demonstrating safety and efficacy in the MCCUP patient population would allow us the opportunity to provide IFx-Hu2.0 to more patients with MCC," said James Bianco, M.D., President and Chief Executive Officer of TuHURA Biosciences. "We are also moving towards initiating our Phase 3 accelerated approval trial of IFx-Hu2.0 in first-line advanced and metastatic MCC and anticipate the lifting of the manufacturing-related partial clinical hold in the coming weeks. Our Phase 3 trial, for which we have an SPA agreement with the FDA, will be a single randomized placebo-controlled trial of IFx-Hu2.0 as adjunctive therapy with Keytruda (pembrolizumab) as a first-line therapy for advanced and metastatic Merkel cell carcinoma (MCC), and, if positive, may satisfy the requirement for a post-approval confirmatory trial, saving time, money and risk associated with a second trial."

"In addition to our IFx-Hu2.0 drug candidate, we continue to assemble an exciting late-stage pipeline through our pending acquisition of Kineta, Inc. and its VISTA inhibitor antibody, KVA12123. We are targeting to close the acquisition later this quarter subject to financing and other conditions and advance KVA12123 into a Phase 2 trial in relapsed or refractory NPM1-mutated AML where VISTA expression on leukemic blasts is believed to be responsible for how leukemic cells escape immune recognition contributing to poor responses to therapy and high rates of relapse," stated Dr. Bianco. "We are also developing tumor microenvironment modulators in the form of bi-specific immune modulating Antibody Peptide Conjugates (APCs) and Antibody Drug Conjugates (ADCs) targeting myeloid-derived suppressor cells (MDSCs). We plan to present data at a scientific conference this year on the discovery of high-expression delta opioid receptor (DOR) and the effect on MDSCs and M2 macrophages in the tumor environment. We believe that our DOR program has the potential to reprogram the function of MDSCs and M2 macrophages within the tumor microenvironment, which could have broad implications," concluded Dr. Bianco.

Corporate Highlights

Initiation of Phase 1b/2a Study of IFx-Hu2.0 as Adjunctive Therapy to Keytruda in 1L MCCUP. In May 2025, TuHURA announced that it initiated its Phase 1b/2a trial of IFx-Hu2.0 as an adjunctive therapy to pembrolizumab in MCCUP. This Phase 1b/2a trial is investigating safety and feasibility of IFx-Hu2.0 when administered via interventional radiology to patients with deep-seated tumors who would not be eligible for the Phase 3 trial.
Presentation of Two Posters at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. In April 2025, TuHURA presented a poster at the AACR (Free AACR Whitepaper) Annual Meeting outlining a greater than 90% VISTA receptor occupancy following treatment with Kineta’s VISTA inhibitor mAb as monotherapy and in combination with pembrolizumab in advanced solid tumors. Additionally, the Markowitz Lab at Moffitt Cancer Center presented a poster of IFx-Hu2.0, demonstrating increased T Cell and B Cell production in peripheral blood relative to tumor tissue following adjunctive administration of IFx-Hu2.0 in combination with pembrolizumab.
Appointment of Dr. Bertrand Le Bourdonnec as Head of Drug Discovery. In April 2025, TuHURA announced the appointment of Dr. Le Bourdonnec as the Company’s Executive Vice President, Head of Drug Discovery, Early Development and Program Management. Dr. Le Bourdonnec has extensive experience in the biology and molecular pharmacology of the DOR, TuHURA’s core target on MDSCs for the Company’s emerging suite of ADC and APC development candidates.
Appointment of Dr. Craig L. Tendler to Board of Directors. In March 2025, TuHURA announced the appointment of industry veteran Dr. Craig Tendler to its Board of Directors. Dr. Tendler brings to TuHURA decades of experience in cancer therapeutic development, and most recently served as Johnson & Johnson’s Vice President, Oncology Clinical Development. During his tenure at J&J, Dr. Tendler oversaw 30 major drug approvals, and led development planning from proof of concept through registration and life-cycle management, for J&J’s treatments in hematological malignancies and more prevalent solid tumor diseases.
Upcoming Anticipated Milestones by Program

IFx-Hu2.0

Q2 2025: TuHURA anticipates the FDA’s complete response letter lifting the partial clinical hold relating to completion of certain CMC requirements.
Q2 2025: Initiation of Phase 3 accelerated approval trial in first line Merkel cell carcinoma
VISTA Inhibiting Monoclonal Antibody

Q2 2025: TuHURA expects to close its acquisition of Kineta, Inc. and Kineta’s VISTA inhibiting mAb.
Q3 2025: Initiation of Phase 2 trial of VISTA inhibiting mAb in combination with a menin inhibitor for the treatment of NPM1-mutated AML.
ADC and APC Development Candidates

TuHURA continues to advance its bi-specific, bi-functional immune modulating ADCs and APCs that target the DOR on MDSCs, inhibiting their immune suppressing effects in the tumor microenvironment while localizing a checkpoint inhibitor like the VISTA inhibiting antibody.
In 2025, TuHURA anticipates presenting non-clinical data at relevant medical meetings.
Financial Results for the Three Months Ended March 31, 2025

Research and development expenses were $4.6 million and $3.6 million for the three months ended March 31, 2025, and 2024, respectively.

General and administrative expenses were $2.4 million and $1.0 million for the three months ended March 31, 2025, and 2024, respectively.

As of March 31, 2025, TuHURA’s total shares outstanding was approximately 43.7 million.

As previously announced, on December 11, 2024, TuHURA entered into a definitive agreement with Kineta, Inc. (OTC Pink: KANT) in which, as amended, TuHURA agreed to acquire Kineta, including the rights to Kineta’s novel KVA12123 antibody, for a combination of cash and shares of TuHURA common stock via a merger transaction upon the terms and conditions described in TuHURA’s Form 8-Ks filed on December 12, 2024 and May 7, 2025. The merger is currently targeted to close in Q2 2025 pending the satisfaction of certain financing and other closing conditions.

On May 15, 2025 TriSalus Life Sciences, Inc. (Nasdaq: TLSI) (the "Company"), an oncology company integrating novel delivery technology with standard of care therapies, and its investigational immunotherapeutic to transform treatment for patients with solid tumors, reported financial results for the quarter ended March 31, 2025, and provides an operational update and revised financial guidance (Press release, TriSalus Life Sciences, MAY 15, 2025, View Source [SID1234653201]).

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"Our strong first quarter performance reflects the growing impact of our PEDD platform across a variety of solid tumor indications," said Mary Szela, President and CEO of TriSalus. "With the decision to partner Nelitolimod development following completion of the Phase 1 trials, and the expansion of our TriNav portfolio for a wide range of new applications, we are better positioned than ever to execute our long-term strategy.

"Following our recent private placement, we have made the strategic decision to further invest in our sales force expansion and clinical registries in new applications of our technology. We believe this growth-oriented strategy balances financial discipline with bold investments designed to drive long-term value creation. Accordingly, we’ve updated our 2025 guidance to reflect these strategic priorities. We are confirming our guidance of at least 50% revenue growth; however, due to increased investment, we no longer expect to be adjusted EBITDA-positive or cash flow-positive in 2025."

First Quarter 2025 Highlights

Generated $9.2 million in net sales, a 42% increase year-over-year, and sequential growth of 11% over the fourth quarter 2024.
Continued strong commercial momentum with expanded use of TriNav in liver embolization and entry into new clinical settings.
Presented compelling real-world health economics and outcomes research at SIO 2025.
Announced HCPCS code C8004 from CMS, expanding reimbursement coverage for mapping procedures using TriNav.
Launched TriNav LV and TriGuide, expanding PEDD technology to larger vessels and broader clinical utility.
Subsequent to the first quarter, the Company raised $22.0 million in gross proceeds via private placement to support continued growth.
Reached agreement with 55% of preferred shareholders to simplify capital structure and eliminate the reset provision with full preferred share conversion by mid July 2025.
Updated 2025 Guidance

TriSalus is confirming its revenue guidance of at least 50% growth due to continued commercial momentum and increasing market adoption of the TriNav platform.

While we remain focused on improving adjusted EBITDA performance, we are intentionally investing in strategic priorities that position us for long-term growth. In particular, we are deploying capital to accelerate the development of new clinical applications for our core technology and to expand our commercial organization. These investments are designed to broaden our addressable market and drive sustained value creation. As a result, we do not expect to be adjusted EBITDA positive or cash flow positive in 2025.

Financial Results for Q1 2025

Revenue, all from the sale of the TriNav system, was $9.2 million for the three months ended March 31, 2025, an increase of 42% compared to the same period in 2024 and 11% sequential growth. Revenue growth was driven primarily by increased selling resources and increased market share.

Gross margins were 84% for the three months ended March 31, 2025, compared to 85% for the same period in 2024. The current year decline was caused by decreased production due to clean room expansion.

Operating losses were $7.3 million for the three months ended March 31, 2025, compared to losses of $11.7 million for the same period in 2024. Current year reductions in operating losses are due to increased sales and reduced research and development expenses associated with the ramp-down of clinical trial spending.

Net losses available to common stockholders were $10.4 million for the three months ended March 31, 2025, compared to losses of $13.2 million for the same period in 2024. Net losses in 2025 include non-cash related losses on change in fair value of various derivatives of $1.7 million for the three months ended March 31, 2025, compared to losses of $1.5 million for the same period in 2024. The basic and diluted loss per share for the three months ended March 31, 2025, was $0.39, compared to $0.60 for the same period in 2024.

The non-GAAP measure of adjusted EBITDA is shown for the first time and reconciled in the table below as the Company believes it is an important measure of performance. Adjusted EBITDA losses were $5.5 million for the three months ended March 31, 2025, compared to losses of $10.4 million for the same period in 2024. Current year reductions in adjusted EBITDA losses are due to increased sales, reduced research and development expenses and increased stock compensation in 2025.

On March 31, 2025, cash and cash equivalents totaled $13.0 million. The Company subsequently raised $22.0 million in gross proceeds via a private placement in the second quarter. The Company believes that these proceeds provide sufficient cash runway throughout 2025 and expects to be adjusted EBITDA positive in the first half of 2026.

Conference Call & Webcast

The Company will host a conference call and webcast today at 8:00 AM eastern time to discuss its financial results for the quarter ended March 31, 2025. Parties interested in participating by phone should register using this online form. After registering for the webcast, dial-in details will be provided in an auto-generated e-mail containing a link to the conference phone number along with a personal pin. The event will also be webcast live on the investor relations section of TriSalus’ website. A replay will also be available on the website following the event.

On May 15, 2025 HCW Biologics Inc. (the "Company" or "HCW Biologics") (NASDAQ: HCWB), a clinical-stage biopharmaceutical company focused on discovering and developing novel immunotherapies to lengthen health span by disrupting the link between inflammation and age-related diseases, reported financial results and recent business highlights for its first quarter ended March 31, 2025 (Press release, HCW Biologics, MAY 15, 2025, View Source [SID1234653168]).

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On May 15, 2025, the Company closed an equity offering with gross proceeds of $5.0 million with a single institutional investor. Dr. Hing Wong, Founder and CEO, stated, "We are pleased to have completed a successful $5.0 million equity offering in a difficult market without using a highly structured deal. This funding will be used to open clinical sites for our Phase 1 clinical trial to evaluate HCW9302 in an autoimmune disorder." Dr. Wong continued, "Our new round of financing will provide funding for critical studies to complete the research package for our business development campaign to identify a licensing partner to commercialize our Immune-Cell Engagers, including T-Cell Engagers, which we created using our TRBC drug discovery and development platform."

Dr. Wong explained the Company’s financing strategy, stating, "We have over 50 compounds that we have created and own. We are continually assessing our portfolio of molecules to identify strong candidates to develop and commercialize through licensing or other business development transactions. We plan to ramp up our business development efforts in the second half of 2025. One of our compounds that is ready to commercialize is HCW9206, a clinical-stage molecule. It is a promising revolutionary reagent to replace anti-CD3/anti-CD28/IL-2-based approaches to streamline and lower the costs of CAR-T manufacturing. Equally important, we believe that HCW9206 can improve the functional activities and persistence of CAR-Ts following adoptive transfer, a goal that has not been achieved for the last decade."

Business Highlights

Business Development Transactions

•
On May 13, 2025, the Company delivered its technology report to WY Biotech in accordance with the terms of the WY Biotech exclusive worldwide licensing agreement to use and apply HCW11-006 for in vivo applications. HCW11-006 is a preclinical drug built with our second-generation discovery and development platform — the TRBC platform. WY Biotech has 30-days due diligence to study this report, after which the Company expects to recognize revenue for a $7.0 million upfront licensing fee.

Financing Transactions

•
On May 15, 2025, the Company completed a $5.0 million offering of an aggregate of 671,140 units at a purchase price of $7.45 per unit priced at-the-market under Nasdaq rules. Each unit consisted of one share of Common Stock or one Pre-Funded Warrants to purchase one share of Common Stock, with two warrants, each of which can be exercised for one share of Common Stock for $7.45 per share. In addition, the Company entered into a privately negotiated agreement with the holder of certain existing outstanding warrants to purchase up to 167,925 shares of common stock (the "Existing Warrants") to reduce the exercise price of such Existing Warrants from $41.20 per share to $7.45 per share.
•
On May 13, 2025, the Company received a compliance letter from the Nasdaq Panel to confirm that the Company has regained compliance with the bid price requirement in Listing Rule 5550(a)(2), the public float requirement in Listing Rule 5550(a)(4), and the market value of publicly held shares requirement in Listing Rule 5550(a)(5), as required by the Nasdaq Panel decision of April 8, 2025, as amended. The Company remains subject to the remaining terms of the Panel’s April 8, 2025, decision to provide an extension to the compliance period for other Listing Rules. The Company must be in compliance with all Listing Rules by June 16, 2025.

Clinical Development Results

•
On January 28, 2025, the Company received clearance of its IND from the FDA to initiate a first-in-human Phase 1 dose escalation clinical trial to evaluate one of its lead drug candidates, HCW9302, in patients with moderate-to-severe alopecia areata, a common autoimmune disease in humans that currently has no curative FDA approved treatments. This will be a multi-site, Company-sponsored trial that is expected to be initiated in the third quarter of 2025.
•
The Company is launching the commercialization its clinical-stage molecule, HCW9206. Positive results of studies presented by the Company’s research collaborator Dr. Harris Goldstein’s laboratory at the Albert Einstein College of Medicine, Bronx, New York, at the 2025 Annual Meeting of American Association of Immunologists (AAI 2025), Honolulu, HI. The results of these studies represent an alternative novel strategy for CAR-T cell production with the advantage of generating a large population of CAR-Ts with a stem cell-like memory T cell phenotype, which should enhance the persistence of CAR-Ts in patients. We believe that this strategy will likely improve long-term survival of disease-specific CAR-Ts following adoptive transfer and enable sustained suppression of malignancies, chronic infections and autoimmune diseases, and lower the cost of CAR-T manufacturing. Also, we believe that it provides the Company with an in-road opportunity to participate in the development of "in-vivo CAR-T manufacturing technology," a highly promising emergent field.

First Quarter 2025 Financial Results

•
Revenues: Revenues for the first quarters ended March 31, 2024 and 2025 were $1.1 million and $5,065, respectively. Revenues in both periods were derived exclusively from the sale of licensed molecules to the Company’s licensee, Wugen. The licensed molecules are one of the components used by Wugen in manufacturing their immunotherapeutic products.
•
Research and development (R&D) expenses: R&D expenses for the first quarters ended March 31, 2024 and 2025 were $2.1 million and $1.5 million, respectively, a decrease of $644,573, or 30%. R&D expenses were comparatively lower in the reporting period of the first quarter of 2025 primarily due to a decline in manufacturing and material and preclinical expenses, but all expense categories were comparatively lower in the first quarter of 2025 when compared to the first quarter of 2024.
•
General and administrative (G&A) expenses: G&A expenses for the first quarters ended March 31, 2024 and 2025 were $1.6 million and $2.2 million, respectively, an increase of $661,505, or 42%. The increase was primarily due to the waiver of performance bonuses of $293,159 in the aggregate in the first quarter of 2024, which were earned by officers of the Company in prior periods. Expenses increased by $273,059 as a result of accretion of a fixed bonus that will be due if the Secured Notes are repaid on the Maturity Date. Subsequent to the end of the first quarter of 2025, $6.6 million of the outstanding principal for these Secured Notes elected to convert to equity and a portion of the Company’s shares in Wugen common stock. Other increases in expenses were related to insurance costs and professional services, such as auditing services as well as tax and accounting advisors.
•
Legal expenses: Legal expenses, net represent the legal fees that the Company incurred for an Arbitration. On July 13, 2024, the parties entered into a Settlement Agreement and General Release, and the Arbitration and related Complaint were dismissed on December 24, 2024. In the first quarter of 2024, while the Company was preparing for the hearings which took place in May 2024, the Company incurred $4.4 million of legal fees. In January 2025, the Company received a $2.0 million insurance reimbursement that was paid directly to the law firm involved in representing Dr. Hing C. Wong, the Company’s Founder and Chief Executive Officer, in the Arbitration. The Company is engaged in discussions with the law firms involved with this matter to arrange a reasonable payment plan with respect to those legal fees.
•
Net loss: Net loss for the first quarters ended March 31, 2024 and 2025 was $7.5 million and $2.2 million, respectively.

Financial Guidance

As of March 31, 2025, the Company believes that substantial doubt exists regarding its ability to continue as a going concern for at least 12 months from the issuance date of the audited financial statements, without additional funding or financial support. We considered future elements of our financing plan that were probable and likely to be implemented within the next year to determine if financing activities currently underway are sufficient to mitigate the substantial doubt in our going concern analysis. We have had some early success in completing key elements of our multi-step financing plan, however, we cannot be assured that we will continue to have success with all of the elements of our plan.

On May 15, 2025 Merus N.V. (Nasdaq: MRUS) ("Merus", "the Company", "we", or "our"), an oncology company developing innovative, full-length multispecific antibodies and antibody drug conjugates (Biclonics, Triclonics and ADClonics), reported publication on the mechanism of action of petosemtamab, a bispecific antibody targeting Epidermal Growth Factor Receptor (EGFR) and Leucine-Rich G (LGR5), in the scientific journal "Cancers", a MDPI publication (Press release, Merus, MAY 15, 2025, View Source [SID1234653186]).

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"LGR5+ stem-cell-like cells within a tumor, are frequently reported to be a central contributor to cancer growth, treatment resistance, and recurrence in CRC and other solid tumors," said Cecile Geuijen, Ph.D., Senior Vice President and Chief Scientific Officer. "This publication outlining the scientific rationale for petosemtamab, a bispecific targeting EGFR and LGR5, underpins the strong previously reported phase 2 interim clinical data in both 1L PD-L1+ r/m HNSCC in combination with pembrolizumab and as a single-agent in 2L+ r/m HNSCC."

Petosemtamab employs three distinct mechanisms of action: EGFR ligand blocking, EGFR receptor internalization and degradation in LGR5+ cells, and Fc-mediated activation of the innate immune system by antibody-dependent cellular phagocytosis (ADCP) and enhanced antibody-dependent cellular cytotoxicity (ADCC).

Petosemtamab has demonstrated substantial clinical activity in recurrent/metastatic head and neck squamous cell carcinoma (r/m HNSCC) as reported in our 2024 press releases, Merus’ Petosemtamab in Combination with Pembrolizumab Interim Data Demonstrates Robust Response Rate and Favorable Safety Profile in 1L r/m HNSCC (May 28, 2024) and Merus’ Petosemtamab Monotherapy Interim Data Continues to Demonstrate Clinically Meaningful Activity in 2L+ r/m HNSCC (Dec. 7, 2024). Phase 3 trials are ongoing in both first-line (1L) programmed PD-L1+ (CPS≥1) and second/third-line (2/3L) r/m HNSCC with both trials expected to be substantially enrolled by year end.

A phase 2 trial evaluating petosemtamab in combination with standard chemotherapy in 1L and 2L metastatic colon cancer (mCRC), and as monotherapy in heavily pretreated (3L+) mCRC, is enrolling. We expect to provide initial clinical data for petosemtamab in mCRC in second half of 2025.

The publication is available on the Publications page of our website.

On May 15, 2025 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported preliminary data from the Phase 1 clinical trial of RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) (Press release, Rocket Pharmaceuticals, MAY 15, 2025, View Source [SID1234653202]). RP-A601 showed a well-tolerated safety profile with no dose-limiting toxicities, increased PKP2 protein expression, and preliminary indications of improvement or stabilization in arrhythmia burden, heart function, and quality of life in all three patients followed for up to 12 months. Results were presented today as a late-breaking oral presentation at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) and will be discussed on a company webinar today at 4:30 p.m. ET.

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"Preliminary data from the Phase 1 study of RP-A601 for PKP2-ACM are highly encouraging, signaling potential clinical benefit along with a generally well-tolerated safety profile," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "These initial results represent the second gene therapy from our AAV cardiovascular portfolio to show positive clinical data, propelling us one step closer towards our mission of delivering potentially curative treatments to patients with rare and devastating heart conditions."

The preliminary safety and efficacy of RP-A601 were evaluated in a single-arm, open-label, multi-center Phase 1 study in three patients with PKP2-ACM who received a dose of 8×1013 GC/kg. Initial data from the Phase 1 study (safety cut-off May 6, 2025; efficacy cut-off April 2025) showed that RP-A601 was generally well-tolerated with no dose-limiting toxicities observed in all patients followed for up to 12 months. Most treatment emergent adverse events were mild/moderate in severity and self-limited with only one patient experiencing severe adverse events which resolved without clinical sequelae within two months post-treatment, believed to be associated with the immunomodulatory regimen.

Cardiac biopsies showed RP-A601 increased PKP2 protein expression in all three patients. In the patients with low baseline PKP2 expression (N=2), improvements in PKP2 protein expression relative to total cell protein were approximately 110% and 398%, respectively, from baseline to six months follow-up. In addition, RP-A601 increased protein expression and promoted desmosomal localization of PKP2, Desmocollin-2, and Cadherin-2 in all three patients.

Preliminary indications of improvement or stabilization were observed in arrhythmia burden, heart function, and quality of life. Patients in the Phase 1 trial also demonstrated:

Improvements/stabilization in right ventricular (RV) function.
All patients showed normal RV systolic function at most recent follow-up.
Improvements in quality-of-life as assessed through the Kansas City Cardiomyopathy Questionnaire (KCCQ) and New York Heart Association (NYHA) Class.
Clinical improvement in KCCQ-12 score of 34-41 points (≥5 point increases considered clinically meaningful in adults) and improved NYHA class (from Class II at baseline to Class I; NYHA Class I reflects the absence of clinical signs of heart failure) in both patients followed beyond six months.
Decreased/stabilized ventricular ectopy (premature ventricular contractions [PVC], non-sustained ventricular tachycardia [NSVT]) on rhythm monitoring.
Patients experienced a 9% to 63% reduction in PVCs from baseline evaluated six to 12 months post-treatment.
The only patient who had baseline NSVTs saw a decrease from five to zero NSVT episodes per 24-hour period at six months post-treatment.
Decreased/stabilized T-wave inversions on electrocardiogram (ECG).
One patient saw a reduction in ECG leads with T-wave inversions (precordial and inferior ECG) from six to two at six months post-treatment.
Investor Webcast Information
Company management will host a webinar today, May 15, 2025, at 4:30 p.m. ET. To join the investor webinar, please register at View Source The webcast is available under "Events" in the Investors section of the Company’s website at: View Source The webcast replay will be available on the Rocket website upon completion of the event.

About RP-A601
RP-A601 is an investigational gene therapy for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 consists of a recombinant adeno-associated serotype rh74 capsid containing a functional version of the human PKP2 transgene (AAVrh74.PKP2) which is administered as a single intravenous (IV) infusion. RP-A601 is being investigated as a one-time, potentially curative gene therapy treatment that may improve survival and quality of life for patients affected by PKP2-ACM. Rocket holds Fast Track designation in the U.S. and Orphan Drug designation in the U.S. and Europe for the program.

About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)
PKP2-ACM is an inherited heart disease caused by mutations in the PKP2 gene and characterized by life-threatening ventricular arrhythmias, cardiac structural abnormalities, and sudden cardiac death. PKP2-ACM affects approximately 50,000 adults and children in the U.S. and Europe. Patients living with PKP2-ACM have an urgent unmet medical need, as current medical, implantable cardioverter defibrillator (ICD), and ablation therapies do not consistently prevent disease progression or arrhythmia recurrence, are associated with significant morbidity including inappropriate shocks and device and procedure-related complications, and do not address the underlying pathophysiology or genetic mutation.