Ophthalmic diseases specialist Oculis Holding AG announced positive phase II data showing its serum-glucose corticoid kinase-2 activator OCS-05 protected nerve cells and improved vision in patients with acute optic neuritis. The placebo-controlled, randomized trial assessed the neuroprotective effect of OCS-05 in combination with standard of care steroid injections against steroids alone, with a benefit seen in preserving both ganglions and retinal cell nerve fibers. That led to a clinically meaningful improvement in visual function, with an average increase of 18 letters compared to baseline at three months in the test that is used to measure visual acuity in patients who suffer from poor contrast vision. The improvement in vision was maintained at the end of the study at six months. “This is big,” said Riad Sherif, Oculis CEO. “This is the first time we have shown neuroprotection. The aim now is to go full steam to bring this product to market,” he told an analyst meeting held to discuss the data.
US FDA issues first AI guidance for drug development
With artificial intelligence (AI) becoming more and more common in drug development since 2016, the U.S. FDA is now issuing its first draft guidance on that use. The Center for Drug Evaluation and Research (CDER) “has seen a significant increase in the number of drug application submissions using AI components over the past few years,” a CDER spokesperson told BioWorld. “These submissions traverse the drug product life cycle, which includes nonclinical, clinical, post-marketing, and manufacturing phases.” Focusing on AI technologies intended to support regulatory decisions about the safety, effectiveness or quality of drugs and biologics, the guidance released today provides a risk-based credibility assessment framework that can be used to establish and evaluate the credibility of an AI model for a particular context of use of a drug.
Competitors line runway in PH with preserved ejection fraction
An indication of rising interest among drug developers is pulmonary hypertension (PH) due to heart failure with preserved ejection fraction. The condition involves high blood pressure because of heart failure in which the amount of blood pumped out of the heart stays the same. People who end up with PH among this population number about 30-80%. Players in the space include Astrazeneca plc, Eli Lilly and Co., Merck & Co. Inc., Tectonic Therapeutic Inc., and Tenax Therapeutics Inc.
Taiwan’s Tanvex receives second CRL for Herceptin biosimilar
Tanvex Biopharma Inc. said its U.S. subsidiary, Tanvex Biopharma U.S.A. Inc., received an FDA complete response letter (CRL) on Jan. 3 for its TX-05 BLA, a biosimilar that references Roche AG’s Herceptin (trastuzumab). The CRL cites unnamed issues that need to be addressed by the downstream manufacturer of TX-05, which is a third-party service provider of Tanvex U.S. for its drug product. Other than the issues to be addressed by the downstream manufacturer, the FDA has not raised any approvability issues with respect to the drug substance made by Tanvex, the company said in a notice to the Taiwan Stock Exchange (TSX) on Jan. 6.
South Korea ups 2025 health agency budget, biomedical R&D funds
South Korea’s government continued to ramp up investment in its pharmaceutical and med-tech sector in 2025, including a 7.2% hike for the Ministry of Health and Welfare’s (MOHW) budget and a near 15% increase in funding to research and develop innovative biotechnologies. On Dec. 10, 2024, the MOHW announced that Korea’s National Assembly approved an increased budget of ₩125.49 trillion (US$85.42 billion) for the year (excluding childcare-related funding), indicating a 7.2% jump from last year’s budget of ₩117.44 trillion. At a policy meeting Dec. 20, the government met with a medical technology policy deliberating committee to approve a 2025 budget of ₩2.14 trillion to support major health and medical R&D projects across five ministries, including Korea’s MOHW; Ministry of Science and ICT (information and communication technology); Ministry of Trade, Industry and Energy; Ministry of Food and Drug Safety; and Korea Disease Control and Prevention Agency.
HHS floats much more stringent set of HIPAA security measures
Makers of digital health apps are not often subject to the provisions of the Health Insurance Portability and Accountability Act (HIPAA), but any such liabilities may soon become more onerous. The Department of Health and Human Services has released a draft update for HIPAA cybersecurity mandates, the final version of which is sure to be accompanied by much more vigorous enforcement.
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Amylyx, Ani, Astralbio, Beigene, Blossomhill, Capricor, Cyclacel, Eton, GSK, Halia, Hanx, Hutchmed, Iambic, Ibio, Innovent, Perspective, Portage, Regeneron, Septerna, Unity