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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 Epitopea, a transatlantic cancer immunotherapy company, and Institute for Research in Immunology and Cancer (IRIC) of the Université de Montréal, a leading Canadian research institution renowned for scientific innovation and technology transfer, reported a scientific publication in the leading medical journal Nature Cancer (Press release, Epitopea, MAY 27, 2025, View Source [SID1234653392]). This paper demonstrates that Epitopea’s CryptoMapTM platform can identify shared, nonmutated, aberrantly-expressed tumor-specific antigens (CryptigensTM) as highly abundant, actionable targets for immunotherapy in melanoma and non-small cell lung cancer (NSCLC). The identification of CryptigensTM with potential clinical utility distinguishes Epitopea’s approach from many other immunotherapy companies that have focused on the identification of mutated tumor antigens (mTAs).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the paper, produced with international academic collaborators at Canada’s McGill University, University of Liege in Belgium, and Switzerland’s University of Lausanne, CryptoMapTM identified 589 non-redundant tumor antigens (TAs) in cutaneous melanoma and NSCLC. Significantly, only 1% of the actionable TAs were derived from mutated sequences or mTAs. Of the 99% of TAs remaining, approximately 37% (n=220) of TAs identified were CryptigensTM. These CryptigensTM are immunogenic, shared among tumor samples, and could contribute to immune checkpoint blockade responses, supporting their utility in immune targeting across the tumor landscape.

"These data from this latest publication from our collaborators at UdeM further validate the potential benefit of Epitopea’s CryptoMapTM platform and our transformative approach to treating cancer," commented Epitopea’s CEO, Alan C. Rigby. "We believe our CryptigensTM offer significant competitive advantages over current treatment approaches that have focused solely on mTAs. As illustrated, our approach has the potential to stimulate the immune system to precisely recognize and destroy cancer cells more rapidly and effectively, which we believe will translate into durable patient responses in these indications."

"We have long been researching immune-based approaches that could transform the lives of cancer patients through the discovery of accessible and effective immunotherapies for difficult-to-treat tumors. This latest research further strengthens our understanding of the target space amenable for the development of cancer immunotherapy treatments. In particular, the fact that only 1% of tumor antigens are derived from mutated sequences highlights the potential value of exploring unmutated sequences across the cancer landscape. Collectively, the findings in our collaborative manuscript challenge a dogmatic belief that mTAs are the dominant actionable targets for cancer immunotherapy," said Dr. Claude Perreault, Principal Investigator at UdeM’s Institute for Research in Immunology and Cancer, corresponding author of the paper, and a co-founder of Epitopea.

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 TCG Labs Soleil, a venture firm integrating dedicated capital with an in-house biotech R&D hub, and EpimAb Biotherapeutics, Inc. ("EpimAb") reported that Juri Biosciences, Inc. ("Juri"), a portfolio company of TCG Labs Soleil, has entered into a worldwide licensing agreement with EpimAb, a clinical-stage biopharmaceutical company specializing in the discovery and development of multi-specific antibodies for diseases with high unmet need (Press release, EpimAb Biotherapeutics, MAY 27, 2025, View Source [SID1234654051]). The agreement grants Juri exclusive global rights to a development-ready T-cell engager targeting kallikrein-related peptidase 2 (KLK2) and CD3 for the treatment of metastatic prostate cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Under the terms of the agreement, EpimAb is eligible to receive up to $210 million, including an upfront payment and milestone payments tied to development, regulatory, and commercial events, plus tiered royalties. Further financial details were not disclosed.

Juri is one of several portfolio companies formed by TCG Labs Soleil. Dr. Charles Sawyersi, the inaugural Director of the Human Oncology and Pathogenesis Program at Memorial Sloan Kettering Cancer Center, will serve as a strategic scientific advisor, working closely with the TCG Labs Soleil team to support the program and help guide the advancement of KLK2-directed therapies. A leading expert in metastatic prostate cancer and translational oncology, Dr. Sawyers co-discovered enzalutamide, one of the most widely used therapies for advanced prostate cancer today.

"This agreement with EpimAb is a clear demonstration of our venture-biotech model at work. We’ve acquired an exciting external innovation and are deploying our dedicated capital, scientific leadership and operational infrastructure to move it rapidly into the clinic," said Jin-Long Chen, Ph.D., Managing Partner of TCG Labs and Chief Executive Officer of TCG Labs Soleil. "Prostate cancer remains a leading cause of cancer-related death in men, and KLK2 is a highly prostate-specific antigen with the potential to enable more precise, targeted therapy. We look forward to advancing this program through our portfolio company, Juri Biosciences."

Chengbin Wu, Ph.D., Founder and Chief Executive Officer of EpimAb Biotherapeutics, commented, "We are pleased to have identified a partner for our KLK2 program in Juri Biosciences, backed by the experienced team of TCG Labs Soleil. By partnering in the solid tumor space, EpimAb further validated the broad potential of our TCE platform beyond immunology and hematology."

On May 27, 2025 BetaGlue Therapeutics S.p.A. ("BetaGlue"), a clinical-stage oncology company developing an innovative radiotherapy platform for the targeted and personalised treatment of unresectable solid tumours, called YntraDose, reported that an abstract was published by the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) entitled: "Preclinical study of safety and efficacy in vivo of a yttrium-90 resin microspheres glue formulation in a large animal model of pancreatic cancer."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The abstract describes the use of YntraDose to treat orthotopic tumors in an advanced in vivo porcine model called ‘oncopig’. Five animals were treated and followed-up over 30 days, the findings were:

No adverse events were reported throughout the study period;
YntraDose implantation and its associated radiotherapeutic payload were confined to the site of administration as demonstrated by radiological PET/CT assessment; and
At 30days visual necropsy assessment not only revealed evidence of tumor necrosis but also indicated tumor debulking.
Dr Colin Story, CEO, BetaGlue Therapeutics commented: "This study utilised a cutting edge advanced large animal model of pancreatic cancer where procedures were performed by world-renowned Interventional Radiologist and key opinion leader Professor Govindarajan Narayanan. BetaGlue is focused on bringing YntraDose to patients that present with Locally-Advanced Pancreatic Cancer which by definition is inoperable. We are very grateful to our collaborators Prof. Narayanan, Dr Geschwind and Ms Felicia Bealo for their contribution to this work."

Professor Govindarajan Narayanan, Chief of Interventional Oncology for the Miami Cancer Institute and lead author of the abstract commented: "Locally-Advanced Pancreatic Cancer (LAPC) accounts for around 30% of all Pancreatic Cancers worldwide. This study provides preliminary evidence of the utility for YntraDose as a local treatment option for LAPC with the potential to down-stage patients and enabling surgical resection to take place."

(Press release, BetaGlue Therapeutics, MAY 27, 2025, View Source [SID1234661239])