On May 27, 2025 Theriva Biologics (NYSE American: TOVX), ("Theriva" or the "Company"), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported the upcoming presentation of final clinical outcomes and safety data from the investigator sponsored Phase 1 clinical study conducted at Sant Joan de Déu Barcelona Children’s Hospital evaluating the safety and tolerability of two intravitreal injections of VCN-01 (zabilugene almadenorepvec) in patients with intraocular retinoblastoma that was refractory to systemic, intra-arterial, or intravitreal chemotherapy, and for whom enucleation was the only recommended treatment (NCT03284268) (Press release, Theriva Biologics, MAY 27, 2025, View Source [SID1234653408]). These data will be featured in a poster at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago, IL from May 30-June 03, 2025.

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Details on Poster number 161 (abstract number 10046) can be found below.

Presenting Author: Dr. Jaume Català-Mora, Pediatric Ophthalmologist, Sant Joan de Déu-Barcelona Children’s Hospital
Title: A Phase I dose-escalation study to assess the oncolytic virus VCN-01 safety and efficacy in refractory retinoblastoma patients.
Poster Session: Pediatric Oncology
Date & Time: Saturday May 31st, 2025, at 0900-1200 US CDT
Location: online at the conference portal and in person at Hall A, McCormick Place, Chicago, IL
"We are very pleased that the important work by our collaborators at Sant Joan de Déu-Barcelona Children’s Hospital is being presented at the premier international oncology conference" said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "We previously reported that the Study Monitoring Committee determined the trial results to be positive. This poster presentation provides the investigators with the opportunity to discuss the detailed results on VCN-01 safety and long-term efficacy in this population with a broader clinical oncology audience and obtain feedback that may assist in refining our clinical strategy for VCN-01 in this underserved pediatric cancer population."

Guillermo Chantada, world-recognized expert in retinoblastoma and one of the investigators in the trial indicated "The oncolytic virus VCN-01 is a promising new player for the treatment of retinoblastoma. In our study, it has shown a tolerable toxicity profile and encouraging response in refractory vitreous seeds which are still the major cause of conservative therapy failure. Through its mechanism of action, we found that VCN-01 specifically targets the tumor cells and by being a non-chemotherapeutic or radiotherapeutic agent, it is of additional interest in this population with higher risk of treatment-induced malignancies".

Theriva senior management will also attend the ASCO (Free ASCO Whitepaper) conference and participate in an off-site meeting on Monday June2nd, 2025 to review the recently reported topline results of the VIRAGE Phase 2b clinical trial in first-line metastatic pancreatic ductal adenocarcinoma (PDAC) with investigators and garner additional insights relevant to the design and execution of a Phase 3 clinical trial in this indication.

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 140 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov. VCN-01 has Orphan Drug designation from the EMA and both Orphan Drug designation and Fast Track designation from the FDA for the treatment of pancreatic cancer. VCN-01 also has Orphan Drug designation and Rare Pediatric Diseases designation from the FDA for the treatment of retinoblastoma.

About Retinoblastoma

Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 – 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In Europe, retinoblastoma has an estimated incidence rate of 1 per 13,844 live births (14.1 per million children under the age of 5) with approximately 300 children diagnosed per year (Stacey et al. 2021). Preserving life and preventing the loss of an eye, blindness, and other serious effects of treatment that reduce the patient’s life span or the quality of life remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries.

On May 27, 2025 Biogen Inc. (Nasdaq: BIIB) and City Therapeutics, Inc., a privately held biopharmaceutical company leading the future of RNA interference (RNAi)-based medicine, reported a strategic collaboration to develop select novel RNAi therapies (Press release, Biogen, MAY 27, 2025, View Source [SID1234653407]).

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Through the collaboration, City Therapeutics will leverage its next-generation RNAi engineering technologies to develop an RNAi trigger molecule combined with proprietary drug delivery technology from Biogen. The collaboration will initially focus on a single target that mediates key central nervous system diseases, utilizing tissue enhanced delivery technologies with the aim of allowing for systemic administration of medicines. Biogen will be responsible for IND-enabling studies and global clinical development along with any regulatory submissions and all activities related to commercialization.

"This collaboration underscores Biogen’s new strategic research approach of balancing our differentiated internal capabilities with external investments in cutting-edge science. With this effort, we are further expanding the modalities in our R&D toolbox to potentially reach our targets of interest more precisely by adding an RNAi-based approach," said Jane Grogan, Ph.D., Head of Research at Biogen. "We are excited to collaborate with City Therapeutics and their world-class scientists on key programs, as well as to invest in their company as part of this innovative effort to develop new approaches to treating disease."

"Partnering with Biogen represents a meaningful milestone in our mission to expand the therapeutic reach of RNAi, as we pioneer the next generation of RNAi technology for breakthrough medicines," said Andy Orth, Chief Executive Officer of City Therapeutics. "By combining our novel RNAi platform with Biogen’s industry-leading capabilities in global drug development, we aim to accelerate the advancement of therapies for serious diseases. We look forward to demonstrating the potential of our RNAi platform in addressing this area of significant unmet need."

Under the terms of the agreement, City Therapeutics will receive a total of $46 million in payments including a $16 million upfront payment and an investment of $30 million in exchange for a City Therapeutics convertible note, representing a minority equity interest in the company if converted. The upfront payment will be recorded by Biogen as an Acquired In-Process Research and Development expense in the second quarter of 2025. Should the initial program achieve certain development and commercial milestones, City Therapeutics is eligible to receive up to approximately $1 billion in potential milestone payments plus tiered royalties in the high single-digit to low double-digit range based on net sales. Biogen will have the option to select one additional target in the collaboration, subject to an additional payment and availability of the target.

On May 27, 2025 BeiGene, Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company that will change its name to BeOne Medicines Ltd., reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion recommending approval of TEVIMBRA (tislelizumab), in combination with gemcitabine and cisplatin, for the first-line treatment of adult patients with recurrent, not amenable to curative surgery or radiotherapy, or metastatic NPC (Press release, BeiGene, MAY 27, 2025, View Source [SID1234653406]).

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"Chemotherapy has been the standard treatment for metastatic NPC; however, distant metastasis continues to be a significant cause of treatment failure and death, making new treatment options essential," said Prof. Lisa Licitra, Chief of Head and Neck Cancer Medical Oncology Department, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. "Results from the RATIONALE-309 study support the robust clinical benefit of TEVIMBRA plus chemotherapy, which has the potential to significantly reduce the risk of disease progression or death for patients with recurrent or metastatic NPC."

The extension of indication for NPC is based on results from BeiGene’s RATIONALE-309 (NCT03924986), a randomized, double-blind, placebo-controlled, multicenter, Phase 3 study to evaluate the efficacy and safety of TEVIMBRA, in combination with gemcitabine plus cisplatin, as first-line treatment in adult patients with recurrent or metastatic NPC. At the time of the prespecified interim analysis, the study, which randomized 263 treatment-naïve patients, met its primary endpoint, significantly prolonging progression-free survival (PFS) in the intent-to-treat (ITT) population (HR 0.52 [95% CI:0.38, 0.73] p<0.0001). The median PFS in the TEVIMBRA with chemotherapy arm was 9.2 months compared to 7.4 months in the placebo with chemotherapy arm. An updated analysis showed consistent efficacy results with the interim analysis. The median overall survival (OS) was 45.3 months for TEVIMBRA plus chemotherapy versus 31.8 months for placebo plus chemotherapy. TEVIMBRA plus chemotherapy was generally well tolerated, with no new safety signals identified.

"Today’s announcement marks a second positive CHMP opinion for TEVIMBRA in 2025, signifying the potential to expand into yet another disease area in the European Union and to support even more patients living with cancer," said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeiGene. "As the foundation of our solid tumor portfolio, we are encouraged by TEVIMBRA’s momentum in achieving more than 100 regulatory approvals for a range of cancer indications across the world, including major markets such as the US, the EU, China, and Japan, demonstrating the strength of evidence across a range of indications."

The pooled safety data in this extension of indication included more than 3,900 patients who received TEVIMBRA as either monotherapy (n=1,952) or in combination with chemotherapy (n=1,950) at the approved dosing regimen. The most common Grade 3 or 4 adverse reactions (≥ 2%) for TEVIMBRA given in combination with chemotherapy were neutropenia, anemia, thrombocytopenia, hyponatremia, hypokalemia, fatigue, pneumonia, lymphopenia, rash, decreased appetite, increased aspartate aminotransferase, and increased alanine aminotransferase.

TEVIMBRA is currently approved in the EU as a first-line treatment for eligible patients with gastric or gastroesophageal junction (G/GEJ) adenocarcinoma, as a first-line treatment for unresectable esophageal squamous cell carcinoma (ESCC), as a second-line treatment in ESCC after prior platinum-based chemotherapy, as a first-line treatment for extensive-stage small cell lung cancer (ES-SCLC) in combination with chemotherapy, and for three non-small lung cancer (NSCLC) indications covering both the first- and second-line settings.

About Nasopharyngeal Cancer (NPC)

Nasopharyngeal cancer is a type of head and neck cancer that starts in the nasopharynx,1 the upper throat passage that connects the nose to the lungs.2 NPC is often diagnosed at advanced stages due to its deep anatomical location and mild early symptoms, making early detection challenging.3 Globally, NPC accounts for approximately 133,000 new cancer cases and 80,000 deaths per year and exhibits a unique geographical pattern, with its prevalence notably concentrated in Asia.4 While the overall 5-year survival rate for NPC is approximately 63%, in advanced disease the survival rate decreases to 49%.5

About TEVIMBRA (Tislelizumab)

TEVIMBRA is a uniquely designed humanized immunoglobulin G4 (IgG4) anti-programmed cell death protein 1(PD-1) monoclonal antibody with high affinity and binding specificity against PD-1. It is designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors.

TEVIMBRA is the foundational asset of BeiGene’s solid tumor portfolio and has shown potential across multiple tumor types and disease settings. The global TEVIMBRA clinical development program includes almost 14,000 patients enrolled to date in 35 countries and regions across 70 trials, including 21 registration-enabling studies. TEVIMBRA is approved in 46 countries, and more than 1.5 million patients have been treated globally.

Important Safety Information

The current European Summary of Product Characteristics (SmPC) for TEVIMBRA is available from the European Medicines Agency.

This information is intended for a global audience. Product indications vary by region.

On May 27, 2025 Volastra Therapeutics, a clinical-stage cancer biotechnology company, reported the appointment of David P. Southwell as Chief Executive Officer (Press release, Volastra Therapeutics, MAY 27, 2025, View Source [SID1234653405]). Mr. Southwell brings over 30 years of C-level executive biotechnology experience to Volastra as the company continues to advance its pipeline, which is led by two novel clinical stage KIF18A inhibitors.

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"We are delighted to welcome David to Volastra," said Sandi Peterson, Chair of Volastra’s Board of Directors. "David’s deep biotech leadership experience and proven track record make him the ideal CEO to lead Volastra through this exciting period of growth."

"Volastra has pioneered novel therapeutic approaches in the emerging field of chromosomal instability, which is central to the progression of many cancers," said David Southwell. "With exciting early clinical data and a world-class team of management and investors, Volastra is well-positioned to be a leader in oncologic therapies."

Mr. Southwell has built a strong foundation of financial, operational and strategic expertise throughout his career leading innovative biotechnology and pharmaceutical companies. He most recently served as President and Chief Executive Officer of TScan Therapeutics, an immune-oncology company advancing T-cell receptor-based therapies. Prior to that, he was President, Chief Executive Officer, and Board member of Inotek Pharmaceuticals, guiding the company through its merger with Rocket Pharmaceuticals in early 2018. Mr. Southwell also held senior executive roles as CFO at Human Genome Sciences through its acquisition by GlaxoSmithKline, and at Sepracor Inc., where he served as Executive Vice President and CFO for over a decade. He has served on the Boards of several public and private life sciences companies and currently serves on the Boards of PTC Therapeutics and Rocket Pharmaceuticals.

Mr. Southwell succeeds Charles Hugh-Jones, who has led Volastra Therapeutics from its inception as CEO. The Board greatly appreciates Charles’ leadership, vision and substantial contributions to the Company’s success over the past 5 years. He will continue as an advisor to the Company.

Separately, Volastra will be presenting dose escalation data from its first-in-human VLS-1488 trial in an oral presentation on June 2nd at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.

On May 27, 2025 Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and Alzheimer’s disease (AD) through the inhibition of Semaphorin 4D (SEMA4D), reported that it will present new data characterizing the unique mechanism of pepinemab to enhance immune responses to checkpoint therapies in the neoadjuvant setting that are associated with improved pathologic response in patients with head and neck cancer (Press release, Vaccinex, MAY 27, 2025, View Source [SID1234653404]). Lead investigator and collaborator, Conor Steuer, MD from Winship Cancer Center at Emory University, will present results at the 2025 Annual Meeting of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago on June 1, 2025.

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ASCO Conference Information:

Date: Sunday, June 1, 2025

Presentation title: Neoadjuvant biomarker trial of pepinemab to enhance nivolumab or ipilimumab activity in resectable head and neck cancer. Abstract 103.

Time: 9:45-11:15 AM CST/10:45 AM – 12:15 PM EST.

Session Title: Clinical Science Symposium – Turning "Cold" Tumors "Hot"

Previously reported data from our collaboration with Emory University suggest a crucial role of pepinemab treatment in melanoma patients to facilitate immune cell interactions within highly organized and robust centers of immunity, called tertiary lymphoid structures, or TLS. By blocking the SEMA4D inhibitory signal to Dendritic Cells (DC), pepinemab allows productive, coordinated interactions between SEMA4D+ T cells, key effector cells capable of eradicating tumors, and DC, regulatory cells that promote immune cell interactions within TLS so as to amplify mature T cell responses.

New data presented at ASCO (Free ASCO Whitepaper) will characterize the mechanisms of neoadjuvant treatment with pepinemab in patients with resectable head and neck cancer (HNSCC). Standard of care for these patients often involves toxic chemotherapy and/or radiotherapy, in addition to surgery, which can significantly impact their quality of life. Peri-operative treatment with immunotherapy has recently reported promising and potentially practice-changing results. Certain HNSCC with hot beds of immune cells organized into TLS have been shown to correlate with clinical benefit and positive response to immune checkpoint therapy. However, many HNSCC are considered immunologically "cold" tumors, due to exclusion of immune cells from tumor and/or high levels of immune suppressor cells, making them resistant to immune checkpoint therapy.

Pepinemab has potential to be a major advance for HNSCC patients with cold and resistant disease, with capacity of a well-tolerated and effective treatment that can induce formation and harness the power of TLS to optimize the clinical benefit of immunotherapy. Our data demonstrate that the addition of pepinemab to neoadjuvant immune checkpoint treatments did not compound toxicities, yet it enhanced TLS maturity that correlated with improved pathologic response. Collectively, these results highlight the potential of pepinemab to turn immunologically cold tumors, such as HPV-negative head and neck cancer, into hot immune centers by inducing robust and mature TLS.

About Pepinemab
Pepinemab is a humanized IgG4 monoclonal antibody designed to block SEMA4D, which can otherwise bind to plexin-B1 receptors to trigger collapse of the actin cytoskeleton and lead to loss of homeostatic functions of dendritic cells in immune tissue and of astrocytes and other glial cells in the brain. Pepinemab appears to be well-tolerated with a favorable safety profile in multiple clinical trials in different cancer and neurological indications.