Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA announces new initiative for rare diseases
The FDA has unveiled a new Rare Disease Framework Initiative designed to “reimagine the development of drugs and biologics for rare diseases,” according to the press release. Recognizing that only five percent of rare diseases having an FDA-approved treatment, the announcement describes how the new initiative seeks to include patients’ voices, build shared resources on rare disease drug development and review, and champion the use of cutting-edge science, among other commitments. As outlined in the new white paper accompanying the framework, “The goal is a regulatory framework where fit-for-purpose evidentiary expectations are the norm for all rare disease applications; the FDA will work closely with sponsors and patients to support innovative approaches to generating evidence that will meet the FDA’s rigorous statutory standard of safety and effectiveness.” – Allison Whitten
Early results show Arrowhead’s Alzheimer’s drug decreases tau in the brain
On Wednesday, Arrowhead Pharmaceuticals announced positive interim results from their Phase 1/2a study of their RNAi drug, ARO-MAPT. According to the press release, the drug led to an average decrease of 73 percent in CSF total tau on day 105 in healthy volunteers — a result that could usher in a new era of targeting tau. The drug works by silencing gene expression of the MAPT gene that produces the tau protein. The current results also show that the company’s Targeted RNAi Molecule (TRiMTM) delivery platform was able to successfully deliver the therapy across the blood-brain barrier, which could prove useful for a variety of neurological disorders. According to the statement, the next steps for ARO-MAPT will be to complete dosing in healthy volunteers and continue enrollment in their early Alzheimer’s disease cohorts, with plans to eventually test the therapy in MAPT-associated frontotemporal dementia and Progressive Supranuclear Palsy. “These early clinical data are exciting because they demonstrate that ARO-MAPT can achieve deep and durable reductions of tau expression in the brain following a simple subcutaneous administration, representing an important step toward developing a potential treatment for Alzheimer’s disease and other primary tauopathies,” said Christopher Anzalone, President and CEO of Arrowhead, in the press release. – Allison Whitten
Positive results for celiac disease in Phase 1b trial
First Tracks Biotherapeutics, which focuses on advancing antibody therapeutics for autoimmune and inflammatory diseases, announced encouraging results for ANB033 to treat celiac disease. The drug is a CD122 antagonist that inhibits both IL-15 and IL-2 on immune cells. Compared to placebo, the treatment led to statistically significant changes in the villus height-to-crypt depth ratio, intraepithelial lymphocyte (IEL) density, and symptom improvements on a celiac disease scale — suggestive of protective effects against intestinal damage during a gluten challenge test. “We look forward to continuing to work with regulators, clinicians and the patient community as we advance ANB033 into late-stage development in 2027,” said Daniel Faga, President and CEO of First Tracks in the press release. The results add to what is setting up to be a competitive landscape for celiac disease, with recent positive Phase 2 results announced from Teva Pharmaceuticals and argenx in September. – Allison Whitten
BioNTech’s mRNA cancer therapy misses first goal in head and neck trial
BioNTech announced that its Phase 2/3 AHEAD-MERIT trial missed its first dual primary endpoint, with BNT113 plus Merck’s Keytruda (pembrolizumab) failing to significantly improve progression-free survival over Keytruda alone in first-line, HPV16-positive head and neck squamous cell carcinoma. The trial will continue to its second endpoint, overall survival, and a significant survival benefit would still count as a positive outcome under the pre-specified design. The miss follows Moderna and Merck’s Phase 3 melanoma win, but the two programs differ in ways that matter. BNT113 encodes shared HPV16 viral proteins, while Moderna’s therapy is built around each patient’s own tumor mutations, and melanoma carries an unusually heavy mutational load that offers more neoantigens to target. Whether that success carries over to tumors with fewer mutations remains an open biological question. Choosing the right antigen is only the first hurdle.The vaccine must also generate effective T cells, get them into the tumor, and keep them active in an immunosuppressive microenvironment. Taken together, these readouts suggest the field’s next test does not necessarily give them the best chance to. – Andrea Corona
GSK moves to axe over 1,000 jobs in Belgium site
GSK said it could cut as many as 1,217 jobs in Belgium, the first concrete sign that CEO Luke Miels’ three-year plan to save 1.9 billion pounds (2.5 billion dollars) is moving into action. The proposed cuts would affect some of the company’s more than 8,000 employees at sites in Rixensart, Wavre and Gembloux, mainly in research, manufacturing support and administrative roles, with factory workers spared. GSK said it will simplify those operations through more digital tools and automation, and will remain Belgium’s largest biopharmaceutical employer while continuing to produce most of its vaccines there. The company also said the changes will help its factories better match demand for some established vaccines, following last month’s announcement that it will close its Dresden, Germany vaccine plant next year amid falling demand for egg-based flu shots. In Belgium, GSK will keep working with universities and research centers but focus R&D resources on high-priority projects, including its mRNA flu vaccine. Miels launched the savings effort in July to speed up drug development and reassure investors as GSK prepares for the patent cliff on its blockbuster HIV drug dolutegravir. – Andrea Corona
