On February 20, 2025 OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage biotechnology company advancing immunotherapies and targeted drug conjugates for cancer treatment, reported that it received a Notice of Allowance from the United States Patent & Trademark Office (USPTO) that a patent will be issued covering the manufacturing methods required for the OST-HER2 commercial product (Press release, OS Therapies, FEB 20, 2025, View Source [SID1234650434]). The USPTO granted a Patent Term Adjustment of 572 days, providing market exclusivity for the OST-HER2 commercial drug product into 2040. OS Therapies is preparing to initiate discussions with the United States Food & Drug Administration (FDA) following the successful treatment phase of its Phase 2b clinical trial in the prevention of recurrent, resected, lung metastatic osteosarcoma with a view towards submitting a Biologics Licensing Application (BLA) and gaining conditional or accelerated FDA approval in 2025. The Company recently completed a $7.1 million financing in January 2025 and has sufficient capital into mid-2026.

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OST-HER2 has already received rare pediatric disease (RPDD), fast-track (FTD) and orphan drug (ODD) designations from the US FDA for osteosarcoma. The Company intends to (1) focus on gaining FDA BLA approval for OST-HER2 in osteosarcoma in late 2025, (2) sell the Priority Review Voucher (PRV) it would receive from the FDA BLA approval prior to the September 30, 2026 PRV deadline to a larger pharmaceutical company at prevailing market prices (most recent PRV sale transaction was $150 million), (3) commercialize OST-HER2 in osteosarcoma and (4) then expand the clinical development of OST-HER2 into breast cancer and other larger solid tumor indications for additional revenue potential.

OST-HER2 has successfully completed a Phase 1 trial in adult patients with HER2 overexpressing cancers, primarily breast cancer patients. Preclinical breast cancer results with OST-HER2 showed:

78% reduction in tumor size (3mm for OST-HER2 treated vs. 14mm for control arm) in FVB/N HER2 transgenic mouse model of breast cancer treatment at day 75
33% prevention of breast cancer in OST-HER2 treated mice vs. 0% prevention of breast cancer in FVB/N HER2 transgenic model of breast cancer prevention at week 50
20% reduction of tumor size for OST-HER2 plus HER2-targeted antibody vs. HER2-targeted antibody alone Tg tumor regression model of breast cancer at day 42
65% reduction cellular concentration of metastatic cells for OST-HER2-treated mice compared with controls in brain metastasis model of primary breast cancer
The osteosarcoma treatment market was estimated at $1.2 billion in 2022 according to Data Bridge Market Research. The Company believes the market opportunity for OST-HER2 in the prevention of lung metastases in osteosarcoma is over $500 million. The breast cancer treatment market was estimated at $29.2 billion in 2023 and expected to grow to $53.7 billion by 2030 according to Grandview Research. The Company believes the market opportunity for OST-HER2 in the treatment of breast cancer exceeds $1 billion.

On February 20, 2025 Oxford Vacmedix (OVM) reported the first patient being treated with new extended dosing of OVM-200, at the prestigious Sarah Cannon Research Institute in London (Press release, Oxford Vacmedix, FEB 20, 2025, View Source;utm_medium=rss&utm_campaign=first-patient-ovm-200-cancer-vaccine-extended-dosing [SID1234650417]). The extended dosing protocol was first suggested by the clinical investigators in the trial, following the excellent safety record seen in Phase 1a. The new regime will allow up to 11 vaccinations of OVM-200 over a six-month period and has been approved by the UK Medicines and Healthcare products Regulatory Agency (MHRA). OVM-200 is a new therapeutic cancer vaccine developed using OVM’s novel recombinant overlapping peptide (ROP) platform. It targets survivin, a protein overexpressed by cancer cells, which prevents them being attacked by the body’s immune system.

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The Phase I trial of OVM-200 is focused on safety and on establishing an immune response in patients with three tumour types – non small cell lung cancer (NSCLC), prostate cancer and ovarian cancer. It is being run at four sites in the UK including the Sarah Cannon Institute and University College Hospital (UCH) in London, the Churchill hospital of the Oxford University Hospitals Foundation Trust (OUHFT) and the Christie NHS Foundation Trust in Manchester. The first part of the trial, Phase 1a, has been completed and has shown both excellent safety and a strong immune response. The Chief Investigator for the trial is Professor Martin Forster, based at UCH. This trial is both the first time OVM-200 has been used in people and also the first time any ROP-based vaccine has been tested in the clinic.

William Finch, CEO of Oxford Vacmedix, said:

The ROP technology has been developed from an initial concept in the laboratory to now being tested as a treatment for critically ill patients. We see the potential benefits of a vaccination approach both in stimulating the body’s immune system to attack the cancer and also, in future trials, enhancing the efficacy of other immune oncology agents. This Phase I trial is a first step towards having effective cancer vaccines.

Dr Anja Williams Principal Investigator at the Sarah Cannon Research Institute UK, added:

It is a privilege to work with Oxford Vacmedix on this innovative vaccine programme for patients with lung, prostate, and ovarian cancer. We are very pleased with the results to date and strongly believe that vaccine treatments will play a major role in future cancer treatments. Extending the dosing will maximise the potential benefits of the vaccine.

On February 20, 2025 Incyte (Nasdaq:INCY) and Genesis Therapeutics, Inc. reported that the companies have entered into a strategic collaboration focused on the research, discovery and development of novel small molecule medicines, with an initial focus on collaboration targets selected by Incyte (Press release, Incyte, FEB 20, 2025, View Source [SID1234650435]).

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Genesis is pioneering generative and predictive artificial intelligence (AI) technologies to help create therapeutics for challenging targets. Utilizing Genesis’ proprietary AI platform, GEMS (Genesis Exploration of Molecular Space), the partnership will pursue the discovery and optimization of small molecule compounds for the collaboration targets. Incyte is granted exclusive rights for potential clinical development and commercialization of collaboration products.

"As a leader in pharmaceutical innovation, Incyte is continually seeking new technologies that can transform how new medicines are discovered and developed," said Pablo J. Cagnoni, M.D., President and Head of Research and Development at Incyte. "Partnering with Genesis Therapeutics presents a unique opportunity to leverage their AI technologies to accelerate the discovery of breakthrough small molecules for high-impact targets in our pipeline."

"AI has the potential to redefine how we discover small molecule medicines, and our team is at the forefront of this revolution," said Evan Feinberg, Ph.D., Founder and Chief Executive Officer of Genesis. "We are pleased to establish this world-class partnership to combine our GEMS AI platform with Incyte’s deep expertise and track record in drug discovery and development, with the shared goal of advancing critical treatments for patients with severe diseases."

Terms of the Agreement

Under the terms of the agreement, Genesis will receive an upfront payment of $30 million. Genesis and Incyte have agreed to collaborate on two initial targets, and Incyte will have the option to nominate an additional target for a predetermined fee. If all milestones are achieved, Genesis is eligible to receive up to $295 million in development, regulatory and commercial milestone payments per target. Genesis is also eligible to receive tiered royalties on sales of any collaboration products, once approved.

On February 20, 2025 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that its executives will participate in fireside chats at the following investor conferences (Press release, PTC Therapeutics, FEB 20, 2025, View Source [SID1234650418]):

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TD Cowen 45th Annual Health Care 2025 Conference
Tuesday, March 4 at 11:10 a.m. ET

Barclays 27th Annual Global Healthcare Conference
Tuesday, March 11 at 12:30 p.m. ET

The presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On February 20, 2025 Signet Therapeutics, a clinical-stage biotech company leveraging organoid- and AI-driven cancer drug discovery, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for SIGX1094, the world’s first potential targeted therapy for diffuse gastric cancer (DGC) (Press release, Signet Therapeutics, FEB 20, 2025, View Source [SID1234650436]). This designation is designed to accelerate the development and regulatory review of therapies addressing serious diseases with unmet medical needs, bringing promising new treatments to patients more quickly. In November 2024, SIGX1094 was also granted Orphan Drug Designation (ODD) from the FDA.

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"Receiving Fast Track Designation for SIGX1094 underscores the FDA’s recognition of both the serious, life-threatening nature of diffuse gastric cancer and the potential of SIGX1094 to address this critical medical need," said Dr. Haisheng Zhang, founder and CEO of Signet Therapeutics. "This milestone highlights our commitment to rapidly advancing innovative cancer treatments through our organoid and AI platform, bringing us closer to offering a breakthrough solution for patients with diffuse gastric cancer."

SIGX1094 is an innovative targeted therapy developed using Signet’s proprietary organoid + AI drug discovery platform. Based on a novel therapeutic target independently identified by Signet, SIGX1094 was discovered and optimized in collaboration with XtalPi (2228.HK), a leading drug discovery platform integrating quantum physics, AI, and robotics. As the world’s first drug developed through the integration of organoid models and AI, SIGX1094 represents a major advance in the treatment of DGC, a highly aggressive cancer with no approved targeted therapies. The drug is currently being evaluated in a Phase I clinical trial at Beijing Cancer Hospital in China.

With Fast Track Designation, Signet Therapeutics will benefit from more frequent interactions with the FDA throughout the drug development process, early regulatory guidance, and potential eligibility for accelerated approval, priority review, and rolling review—all aimed at expediting SIGX1094’s path to market and addressing the urgent need for effective DGC treatments. SIGX1094 received Orphan Drug Designation (ODD) from the U.S. FDA in November 2024. The FDA grants ODD to therapies for rare diseases affecting fewer than 200,000 people in the U.S.

Signet Therapeutics is at the forefront of integrating organoid models with AI to revolutionize drug discovery. By combining AI-driven molecular screening with its proprietary organoid platform, Signet accelerates the identification, evaluation, and optimization of novel therapeutics. This innovative approach significantly enhances drug development efficiency and clinical success rates, representing a paradigm shift in the convergence of AI and biotechnology for precision oncology.