Shares of OS Therapies Inc. (NYSE-A:OSTX) jumped more than 10% in morning trading after the company reported positive phase IIb data, showing OST-HER2, an immunotherapy approach comprising the HER2/neu antigen expressed by an attenuated Listeria monocytogenes vector, hit the primary endpoint of 12-month event-free survival (EFS), preventing recurrence in fully resected, lung metastatic osteosarcoma patients. Data also showed strong trends in overall survival, with all patients who achieved the EFS endpoint remaining alive as of data cutoff. With those results in hand, OS plans to meet with the U.S. FDA regarding an accelerated approval filing for OST-HER2, which has rare pediatric disease designation for osteosarcoma.

Medipost grows US, Canada base; plans US phase III of Cartistem

As more Asia biotechs turn to regenerative medicine to address disorders without a cure, Medipost Inc. is continuing global expansion with Cartistem, its allogeneic human umbilical cord blood-derived mesenchymal stem cell product that gained clearance in South Korea in 2012 to treat knee osteoarthritis. “Stem cells can be used in a variety of clinical indications,” Medipost CEO Edward Ahn said during an interview with BioWorld. “These stem cells have been used not only for knee osteoarthritis, but also for Alzheimer’s, some lung diseases and diabetic neuropathy as well.” Founded in June 2000, Seongnam-si, Korea-headquartered Medipost originally launched under the name of Celltree as a domestic umbilical cord blood bank. Medipost raised $12.5 million in its IPO in July 2005 on the Kosdaq market, becoming one of the first listed Korean bioventures to focus on cell therapy.

Biotech Showcase: GLP-1s and the search beyond obesity

Can the market justify the hundreds of GLP-1 developers that are working to eventually reach the market? When the dust settles, Minji Kim, of Cross Border Partners, told attendees at the Biotech Showcase in San Francisco, a few leading companies will end up dominating the field. Those companies that remain are the ones now looking for novel disease targets instead of concentrating on obesity, she added. Fellow panel member Ashley Zehnder, of Fauna Bio, added that big pharma are developing their GLP-1s using molecular phenotyping to expand their reach, much like breast cancer treatments were developed in the past 10 years. Biotech Showcase is running concurrently with the J.P. Morgan Healthcare Conference in San Francisco.

JPM: CBER’s Marks ‘reassured’ as FDA to refine warnings on CAR Ts

The U.S. FDA is to temper the alert it put out in November 2023 pointing to a potential risk of CAR T therapies causing de novo malignancies. “There was this issue of possible safety concerns with T cell lymphomas, with these CAR T cells. I think this year, we are feeling reassured in this regard,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), told the Alliance for Regenerative Medicine briefing at the J. P. Morgan Healthcare Conference on Jan. 13. “You can take it, that in the not-too-distant future, you’ll be hearing from the agency, as we refine the warnings that we made last year in this area,” Marks said. The Nov. 2023 safety alert was issued after genetic sequencing of a few cases of secondary cancer showed transgenes from the engineered chimeric antigen receptor occurred in the malignancy.

OIG raises concerns about accelerated approval deviations

The U.S. FDA needs to strengthen the guardrails along the accelerated approval pathway to ensure its “appropriate and consistent use,” the Health and Human Services Office of Inspector General (OIG) said in a report released yesterday. In its review of a sampling of 24 drugs granted accelerated approval, the OIG was critical of how the agency used the path in approving three of those drugs – Aduhelm (adacanumab, Biogen Inc.), Exondys 51 (eteplirsen, Sarepta Therapeutics Inc.) and Makena (hydroxyprogesterone caproate, Covis Pharma BV). The FDA’s process for approving those three drugs “deviated from the other 21 approvals in our sample in ways that raised concerns,” OIG said.

TREM2 remains popular despite Alector hitch, varied parties in play

Triggering receptor expressed on myeloid cells 2 (TREM2), an immune receptor that plays a role in inflammation and phagocytosis, continues to charm developers despite setbacks. Vigil Neuroscience Inc. has drawn more of Wall Street’s attention since the deal signed in December by Muna Therapeutics ApS, of Copenhagen, with London-based GSK plc. Among setbacks for the TREM2 approach: South San Francisco-based Alector Inc. reported, also in December, that AL-002, a humanized monoclonal antibody that binds to the target, fell short of its endpoint in a phase II trial in Alzheimer’s disease (AD). Specifically, the compound failed to meet the primary endpoint of slowing of AD clinical progression as measured by the Clinical Dementia Rating Sum of Boxes.

The BioWorld Insider podcast: a year of interviews and insights

The BioWorld Insider podcast is a monthly dive into the business and science of drug development. Conversations with CEOs and BioWorld analysts in the past 12 months have included a wide range of chats with experts who are shaping the future. Subjects included quarterly reviews for M&As and financings, the slow but steady growth of funding and partnering for therapies to treat women, the impact of artificial intelligence and quantum computing on drug development, rare disease drug development, the costs of delays in clinical trials, and an overview of the one of the year’s biggest conferences, the annual American Society of Clinical Oncology gathering in Chicago.

2024 BioWorld Year in Review

BioWorld’s staff recaps the therapeutic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as biopharma forges ahead in 2025.

2024 BioWorld MedTech Year in Review

BioWorld MedTech’s staff recaps the med-tech and diagnostic trends and breakthroughs of 2024, the economic highs and lows facing the industry throughout the past year and the regulatory actions that are likely to have implications as med tech forges ahead in 2025.

2024 BioWorld Science Year in Review

Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health.

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Acadia, Ashvattha, Astrazeneca, Bayer, Bioarctic, Biogen, Biohaven, Biomarin, Bio-Thera Solutions, Corvus, Curevo, Daiichi Sankyo, Eisai, Galderma, Hutchmed, Immuneering, Inhibrx, Mereo, Metagenomi, Neomorph, Nimmune, Nrx, Ocugen, Oncozenge, Oric, Phio, Provectus, Quoin, Santhera, Serina, Sling, Soligenix, Sunshine, Talphera, Umoja, Vaxart, Verastem, Visirose, Vistagen, Vivoryon