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.

On May 15, 2025 I-Mab (NASDAQ: IMAB) (the "Company"), a U.S.-based, global biotech company, focused on the development of precision immuno-oncology agents for the treatment of cancer, reported financial results for the three months ended March 31, 2025, and highlighted recent pipeline progress and business updates (Press release, I-Mab Biopharma, MAY 15, 2025, View Source [SID1234653169]).

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"2025 is off to a strong start for I-Mab. Designation of givastomig as our lead program has enabled us to unlock significant value for the Company by considerably accelerating our Phase 1b program, as we work to improve the care of patients with gastric cancers, which impact more than 250,000 people globally," said Sean Fu, PhD, Chief Executive Officer of I-Mab. "Driven by study momentum and investigator interest, we have completed patient enrollment in the first of two Phase 1b dose expansion cohorts ahead of schedule. We expect to share data on both cohorts in 1H 2026. We believe the combination of significant progress in our givastomig program, substantial cash balance, streamlined operations, and new U.S.-based business model positions I-Mab to deliver for both patients and our investors."

Pipeline Overview and Anticipated Upcoming Milestones

Upcoming anticipated milestones for givastomig (CLDN18.2 x 4-1BB bispecific), prioritized to be I-Mab’s lead program in January 2025:

•
July 2025: Presentation of new givastomig dose escalation combination data on U.S. patients at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) ("ESMO") Gastrointestinal ("GI") Cancers Congress 2025, being held July 2-5 in Barcelona, Spain
Details of the ESMO (Free ESMO Whitepaper) GI Mini Oral Presentation:

Title: Preliminary Safety and Efficacy of Givastomig, a Novel Claudin 18.2/4-1BB Bispecific Antibody, in Combination with Nivolumab and mFOLFOX in Metastatic Gastroesophageal Carcinoma (mGEC)

Speaker: Samuel J. Klempner, MD, Associate Professor of Medicine, Massachusetts General Hospital

Presentation Number: 388MO

Date and Time: Wednesday, July 2nd at 16:50 CEST (10:50am EST)

•
1H 2026: Presentation of data from givastomig dose expansion cohorts (n=40)
Enrollment in the ongoing dose expansion study for givastomig is progressing ahead of schedule. In addition, the Company anticipates updates in 2026 for two programs being developed with its partners: uliledlimab (monoclonal antibody targeting CD73); and ragistomig (PD-L1 x 4-1BB bispecific).

First Quarter 2025 Financial Results – In connection with the divestiture of its Greater China assets and business operations, I-Mab’s first quarter 2024 amounts have been recast to conform to the discontinued operations presentation. Additionally, certain non-recurring costs occurred during the first quarter of 2024 that impact quarter-over-quarter comparisons.

Cash Position

As of March 31, 2025, the Company had cash and cash equivalents, and short-term investments of $168.6 million. The Company’s current cash position is expected to fund the givastomig Phase 1b study through anticipated dose expansion data readouts and further development initiatives into 2027.

Shares Outstanding

As of March 31, 2025, the Company had 187,818,796 ordinary shares issued and outstanding, representing the equivalent of 81,660,346 ADSs, assuming the conversion of all ordinary shares into ADSs.

Research and Development Expenses

Research and development expenses were $0.8 million for the three months ended March 31, 2025, compared to $6.1 million for the three months ended March 31, 2024. The decrease was primarily driven by reimbursements recognized under an existing collaboration agreement and lower contract research organization costs during the three months ended March 31, 2025.

Administrative Expenses

Administrative expenses were $4.5 million for the three months ended March 31, 2025, compared to $2.4 million for the three months ended March 31, 2024. Employee share-based compensation expenses during the three months ended March 31, 2024 were $4.8 million lower, primarily driven by forfeitures in connection with the divestiture of the Greater China assets and business operations. Additionally, legal expenses were $2.5 million lower during the three months ended March 31, 2025.

Interest Income

Interest income was $1.9 million for the three months ended March 31, 2025, compared to $0.7 million for the three months ended March 31, 2024. The increase was primarily attributable to higher interest rates earned on cash balances in 2025.

Other Income (Expenses), Net

Other income (expenses), net were $0.2 million for the three months ended March 31, 2025, compared to $(0.6) million for the three months ended March 31, 2024.

Equity in Loss of Affiliates

Equity in loss of affiliates was $1.0 million for the three months ended March 31, 2024 due to recognition of the employee stock ownership plan expenses from the Company’s unconsolidated investee as a result of the divestiture of the Greater China assets and business operations. There was no equity in loss of affiliates for the three months ended March 31, 2025.

Net Loss from Continuing Operations

Net loss from continuing operations was $(3.2) million for the three months ended March 31, 2025, compared to $(9.4) million for the three months ended March 31, 2024. Net loss from continuing operations per share attributable to ordinary shareholders was $(0.02) for the three months ended March 31, 2025, compared to $(0.05) for the three months ended March 31, 2024.

Net Loss from Discontinued Operations

On April 2, 2024, the Company closed the China divestiture announced on February 7, 2024 (the "Transaction"). In accordance with ASC 205-20, the Company determined that the Transaction represented a strategic shift that had a major effect on the business and therefore, met the criteria for classification as discontinued operations. As a result, the Company recognized a loss from discontinued operations of $6.9 million for the three months ended March 31, 2024.

Net Loss

Net loss was $(3.2) million for the three months ended March 31, 2025, compared to $(16.3) million for the three months ended March 31, 2024. Net loss per share attributable to ordinary shareholders was $(0.02) for the three months ended March 31, 2025, compared to $(0.09) for the three months ended March 31, 2024.

About Givastomig

Givastomig (TJ033721 / ABL111) is a bispecific antibody targeting Claudin 18.2 ("CLDN18.2")-positive tumor cells. It conditionally activates T cells through the 4-1BB signaling pathway in the tumor microenvironment where CLDN18.2 is expressed. Givastomig is being developed for first line ("1L") metastatic gastric cancers, with further potential in other solid tumors. In Phase 1 trials, givastomig has shown promising anti-tumor activity attributable to a potential synergistic effect of proximal interaction between CLDN18.2 on tumor cells and 4-1BB on T cells in the tumor microenvironment, while minimizing toxicities commonly seen with other 4-1BB agents.

The ongoing Phase 1b study is evaluating givastomig for the treatment of gastric cancer in the 1L setting in combination with standard of care, nivolumab (an anti-PD-1 checkpoint inhibitor) plus chemotherapy, in dose escalation and dose expansion cohorts. Dose escalation is complete, and enrollment in the first dose expansion cohort (n=20) finished ahead of schedule. Enrollment continues to progress ahead of schedule in the second dose expansion cohort (n=20). The study builds on positive Phase 1 monotherapy data.

Givastomig is being jointly developed through a global partnership with ABL Bio, in which I-Mab is the lead party and shares worldwide rights, excluding Greater China and South Korea, equally with ABL Bio.

On May 15, 2025 Agenus Inc. ("Agenus" or the Company") (NASDAQ: AGEN), a leader in immuno-oncology, reported new data from its ongoing Phase 1 trial evaluating botensilimab and balstilimab (BOT/BAL) in patients with microsatellite stable (MSS) metastatic colorectal cancer (mCRC) at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Gastrointestinal Cancers Congress in Barcelona, Spain (Press release, Agenus, MAY 15, 2025, View Source [SID1234653187]). A poster presentation will feature updated findings from an expanded cohort of 123 patients, incorporating additional participants and extended follow-up to further assess clinical activity of the combination, including durability of response and overall survival (NCT03860272).

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Presentation Details:

Presentation: Botensilimab plus balstilimab in an expanded cohort of 123 patients with metastatic microsatellite stable colorectal cancer and no active liver metastases

Presenting Author: Dr. Benjamin Schlechter, Dana Farber Cancer Institute

Poster Number: 8P

Session Date and Time: 7/4/2025, 3:30 PM – 4:30 PM CEST (9:30 AM – 10:30 AM EDT)

About Botensilimab (BOT)

Botensilimab is a human Fc enhanced CTLA-4 blocking antibody designed to boost both innate and adaptive anti-tumor immune responses. Its novel design leverages mechanisms of action to extend immunotherapy benefits to "cold" tumors which generally respond poorly to standard of care or are refractory to conventional PD-1/CTLA-4 therapies and investigational therapies. Botensilimab augments immune responses across a wide range of tumor types by priming and activating T cells, downregulating intratumoral regulatory T cells, activating myeloid cells and inducing long-term memory responses.

Approximately 1,100 patients have been treated with botensilimab in phase 1 and phase 2 clinical trials. Botensilimab alone, or in combination with Agenus’ investigational PD-1 antibody, balstilimab, has shown clinical responses across nine metastatic, late-line cancers. For more information about botensilimab trials, visit www.clinicaltrials.gov.

About Balstilimab (BAL)

Balstilimab is a novel, fully human monoclonal immunoglobulin G4 (IgG4) designed to block PD-1 (programmed cell death protein 1) from interacting with its ligands PD-L1 and PD-L2. It has been evaluated in >900 patients to date and has demonstrated clinical activity and a favorable tolerability profile in several tumor types.

On May 15, 2025 BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), reported that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor (Press release, BrainChild Bio, MAY 15, 2025, View Source [SID1234653203]).

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The use of a regenerative medicine, specifically a CAR T-cell therapy, offers the potential to overcome barriers for other drug modalities to be effective in addressing DIPG, including the precarious location of the DIPG tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains intact during tumor progression. BrainChild Bio has designed BCB-276 to be administered by locoregional delivery of targeted CAR T-cells directly into the cerebrospinal fluid, permitting infused CAR T-cells to directly access the tumor bed, using an indwelling reservoir-catheter device.

This approach to administering an autologous B7-H3 CAR T-cell therapy has been successfully implemented and resulted in the promising overall survival benefit in patients with brain tumors observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio’s academic partner, Seattle Children’s Research Institute, and recently published in Nature Medicine.

"We are very pleased to now also receive RMAT designation, less than one month after being granted Breakthrough Therapy designation from FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG. Receiving designations from two independent reviews within FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG," stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. "Our team is keenly focused on initiating the pivotal Phase 2 trial by the end of this year and look forward to continuing to work with the FDA on an accelerated path forward to bring potential new CAR-T treatments for CNS brain tumors in children and adults."

FDA grants RMAT designation to investigational regenerative medicine therapies, including cell therapies, that are aimed at treating serious or life-threatening diseases have shown preliminary clinical evidence that the drug has the potential to address unmet medical needs for the disease. Investigational medicines with RMAT are provided intensive interactions with the FDA during the product candidate’s development process in addition to being eligible for rolling submission and priority review of the marketing application.

"It’s gratifying to see another important benchmark reached in our work to combat pediatric brain cancer," said Dr. Jeff Sperring, Chief Executive Officer at Seattle Children’s. "Our research is the foundation of progress to bring potential therapies to kids as fast as we can – and we’re excited about the possibilities afforded by this designation."

BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024.

About Diffuse Intrinsic Pontine Glioma (DIPG) and Application of CAR T-cell Therapies

Diffuse intrinsic pontine glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard-of-care treatment remains limited to palliative focal radiation therapy which results in a median overall survival of only about 11 months from diagnosis.1 Barriers to effective therapies for DIPG include the precarious location of the tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains relatively intact during tumor progression preventing therapies from gaining access to the cancer.

The barriers to effective therapies for DIPG can be effectively overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potential for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permitting infused CAR T-cells to directly access the tumor bed. This also allows for repetitive infusions of CAR T-cells to replenish the tumor bed, offering the potential for more durable and sustained efficacy. Additionally, with the blood brain barrier intact, this therapeutic approach can also minimize any on-target, off-tumor toxicities resulting from systemic exposure of CAR T-cells.

On May 15, 2025 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that an abstract regarding IPH6501, its ANKET targeting CD20 B cells currently developed in relapsed and/or refractory Non-Hodgkin Lymphoma, has been selected for the European Hematology Association (EHA) (Free EHA Whitepaper) Congress 2025, taking place June 12-15 in Milan, Italy (Press release, Innate Pharma, MAY 15, 2025, View Source [SID1234653170]).

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Abstract details
Antitumor characterization of IPH6501, a novel il2v-armed tetraspecific NK cell engager targeting CD20 B cells, in DLBCL and FL patient samples, and in preclinical combination with R-CHOP
•Abstract Code: PS2004
•Session: Poster session 2
•Session Date/Time: Saturday, June 14, 2025, 18:30 – 19:30 CEST
•More information can be found on the EHA (Free EHA Whitepaper) website.

In addition, an abstract related to SAR’514/IPH6401 (developed by Sanofi) was accepted for online publication.

The BCMA NK Cell Engager SAR’514 Induces Macrophage-Mediated Phagocytosis which is improved by combination with Evorpacept, a CD47 Blocker, in Multiple Myeloma
•Abstract Code: PB2850

About ANKET
ANKET (Antibody-based NK cell Engager Therapeutics) is Innate’s proprietary platform for developing next-generation, multi-specific natural killer (NK) cell engagers to treat certain types of cancer. This versatile, fit-for-purpose technology is creating an entirely new class of molecules to induce synthetic immunity against cancer.

About IPH6501
IPH6501 is the first Antibody-based NK cell Engager Therapeutic to co-engage activating receptors on NK cells (NKp46 and CD16), IL-2R (but not the alpha subunit) through a variant of human IL-2, and a tumor antigen (CD20) via a single molecule, hence providing proliferation and activation signals targeted to NK cells and promoting their cytotoxic activity against CD20 expressing malignant cells.

IPH6501 has shown better anti-tumor efficacy than approved benchmark antibodies in preclinical tumor models (Demaria, EHA (Free EHA Whitepaper) 2023, Carrette, SITC (Free SITC Whitepaper) 2024, Demaria et al, Science Immunology 2024).

IPH6501 is currently being evaluated in a Phase 1/2 multicenter trial (NCT06088654), investigating the safety and tolerability of IPH6501 in patients with relapsed and/or refractory CD20-expressing B-cell Non-Hodgkin’s Lymphoma.