Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Another OX40 therapy drops out of the race
As part of their Q3 financial results, AbCellera announced they do not plan to continue developing their investigational antibody drug blocking the OX40 receptor for atopic dermatitis and other autoimmune conditions. This news came despite meeting all endpoints in the Phase 1 trial. The announcement follows similar decisions from companies developing OX40 inhibitor drugs for skin conditions, with Kyowa Kirin and Amgen announcing their decision to discontinue their anti-OX40 monoclonal antibody in March, and Sanofi who announced in July that they decided not to submit their monoclonal antibody for global regulatory reviews. Both times, the pharma companies cited safety concerns in their statements. OX40 drugs have also seen major setbacks in treating cancer, where the therapies are being used not as OX40 blockers but instead as OX40 activators to stimulate the immune system to attack tumors. This pursuit saw early high-profile drops from AstraZeneca, GSK, Genentech, and Bristol Myers Squibb. Yet, Inhibrx Biosciences designed a second-generation version and recently reported the first positive Phase 2 results when given alongside Keytruda in head and neck cancer, currently acting as the last remaining hope for the drug modality. – Allison Whitten
HHS expands funding for psychedelic mental health research
The US Department of Health and Human Services (HHS) announced up to $4.9 million over five years through the Advanced Research Projects Agency for Health (ARPA-H) to prepare ibogaine for clinical testing in post-traumatic stress disorder (PTSD) and other serious mental illnesses. A team led by Mark Cuban Cost Plus Drug Company will build the regulatory, chemical, and manufacturing foundation for a domestically made, single-molecule ibogaine hydrochloride product. Separately, Beond will generate clinical data on safety, dosing, efficacy, and durability. The funding covers Investigational New Drug (IND) preparation, clinical-grade drug supply, and manufacturing processes. Human trials will require separate authorization. All awardees will open-source their data. ARPA-H expects to make further awards focused on substance use disorders, including opioid use disorder. Ibogaine carries serious cardiac risks, including potentially life-threatening heart rhythm problems. HHS said future studies will need cardiac monitoring and independent safety oversight. Separately, the National Institute of Mental Health (NIMH) issued a $20 million funding opportunity for a research network on rapid-acting psychotropics such as ketamine and psilocybin. – Andrea Corona
Deceased donor bone marrow used to treat aplastic anemia
Franciscan Health and Ossium Health have reported what they describe as the first bone marrow transplant for a non-malignant disease using marrow recovered from a deceased organ donor. The procedure was performed in April in a 40-year-old woman with severe aplastic anemia whose disease had progressed despite multiple treatments. Six months after receiving the transplant, the patient had achieved durable engraftment and hematologic recovery, becoming independent of blood and platelet transfusions with full donor chimerism and no reported graft-versus-host disease. For non-malignant conditions including severe aplastic anemia, sickle cell disease, beta thalassemia, and other inherited or acquired bone marrow failure disorders, bone marrow is generally preferred over peripheral blood stem cells because it contains fewer T cells and may reduce the risk of chronic graft-versus-host disease. However, living donors more commonly provide peripheral blood stem cells because collection is less invasive. The case suggests that recovering and banking bone marrow from deceased organ donors could expand access to a graft source that is often in limited supply, although further transplants will be needed to establish the broader clinical potential of the approach. – Bree Foster
Compass submits psilocybin treatment for FDA approval
Compass Pathways has completed its New Drug Application submission to the FDA for COMP360, a synthetic formulation of psilocybin being developed for treatment-resistant depression. The application is supported by two Phase 3 trials and a Phase 2b study involving more than 1,000 participants, all of which met their primary endpoints. If approved, COMP360 could become the first classic psychedelic medicine authorized by the FDA, potentially opening a new treatment option for patients whose depression has not responded adequately to at least two antidepressant therapies. The therapy has previously received FDA Breakthrough Therapy designation, and Compass is preparing for a potential US launch in the first half of 2027, pending regulatory approval. An approval could have implications beyond COMP360, providing the first major regulatory precedent for a classic psychedelic medicine as a growing pipeline of next-generation psychedelic and non-hallucinogenic compounds moves through clinical development. – Bree Foster
BioNTech acquires FDA priority review voucher for $205 million
On November 10th, BioNTech announced they would acquire a priority review voucher from the Cambridge-based company Scholar Rock. The voucher drops the review time from about ten months down to six. Scholar Rock had received it on September 11th as a reward for developing a rare pediatric disease drug after their spinal muscular atrophy (SMA) drug was approved. It will cost BioNTech $205 million to obtain the voucher, and the company did not disclose which asset they will use it for. Since priority review vouchers were first awarded in 2009, they have always been sellable and transferrable, offering a way for small biotechs to raise capital and quickly add it back into their R&D pipeline. To date, the largest sale went for $350 million when AbbVie purchased a priority review voucher from United Therapeutics in 2015. – Allison Whitten
Pfizer taps Blackstone for up to $1.5B to fund obesity drug
Pfizer will receive up to $1.5 billion from Blackstone through 2028 to help develop berobenatide, an investigational GLP-1 receptor agonist being studied as a once-monthly obesity treatment. Under the co-funding deal, Blackstone covers part of the development costs and earns returns tied to regulatory and sales milestones, while Pfizer keeps control of the program. Pfizer got berobenatide through its roughly $10 billion acquisition of Metsera, which it won in a bidding war with Novo Nordisk. With Eli Lilly and Novo Nordisk leading the market, newer drugs are trying to stand out on convenience, and less frequent dosing is one way to do that. Deals like this let drugmakers split the cost and risk of large Phase 3 programs with outside investors. Whether berobenatide succeeds will still come down to its Phase 3 results. – Andrea Corona
