Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Memory neurons split by protein output in first single-cell translation map of the brain
Scientists at Scripps Research and University of California, San Diego have developed a method to directly measure protein production in individual brain cells, uncovering unexpected differences among memory-related neurons that may reshape understanding of neurological disease. In a study published in Nature, researchers used a technique called Ribo-STAMP to map translation across nearly 20,000 single cells in the mouse hippocampus. They found that CA3 pyramidal neurons produce proteins at significantly higher rates than neighboring CA1 neurons, despite their shared roles in memory circuits. The study also showed that specific mRNA isoforms influence how much protein is made and revealed that neurons can shift between high and low “translation states,” potentially reflecting differences in activity. The findings provide a new framework for studying conditions such as autism spectrum disorder and fragile X syndrome, where disruptions in protein production have been implicated. — Andrea Corona
Researchers develop blood test to predict Alzheimer’s years in advance
Researchers at Washington University School of Medicine in St. Louis have developed a blood test that can predict when someone is likely to develop symptoms of Alzheimer’s disease, potentially years in advance. This could provide opportunities to prevent or delay the onset of Alzheimer’s, for instance, using existing anti-seizure medication. The study, published in Nature Medicine, showed that levels of a protein called p-tau217 in plasma can estimate the age at which individuals may begin experiencing cognitive decline, with a margin of error of three to four years. Using data from more than 600 older adults in two long-term Alzheimer’s research programs, the team found that higher p-tau217 levels correlated with the buildup of amyloid and tau proteins in the brain, key hallmarks of the disease. The researchers said the models could make clinical trials of preventive therapies more efficient and, eventually, help doctors guide patients on strategies to slow or prevent symptoms. “Our work shows the feasibility of using blood tests, which are substantially cheaper and more accessible than brain imaging or spinal fluid tests, for predicting the onset of Alzheimer’s symptoms,” said senior author Suzanne Schindler in the press release. The team has shared all code for their predictive models and developed a web-based tool for researchers, with plans to refine the approach using additional biomarkers in the future. – Bree Foster
FDA rejects Disc’s rare disease drug despite priority voucher
The FDA has rejected Disc Medicine’s rare disease candidate bitopertin, a treatment for erythropoietic protoporphyria (EPP), despite the compound achieving its primary endpoint in late-stage trials. The agency raised concerns about the drug’s reliance on a surrogate biomarker, protoporphyrin IX (PPIX), rather than direct clinical outcomes such as improved tolerance to sunlight, according to the press release. Bitopertin had been reviewed under the FDA’s new Commissioner’s National Priority Voucher program, designed to expedite approval for drugs addressing critical unmet needs, and under an accelerated review timeline. Disc believes that the decision is addressable through the ongoing Phase 3 Apollo trial, which is testing sun exposure outcomes and is expected to complete enrollment in March with topline data by the end of 2026. The company plans to refile for approval once those results are available. However, Disc developed this late-stage trial protocol with the FDA in 2024 under the Biden administration, so there is uncertainty over whether the new FDA leadership will accept the same design. – Bree Foster
Compass Pathways psilocybin therapy shows rapid and durable benefit in depression
Compass Pathways reported strong positive results from two Phase 3 trials of its psilocybin-based therapy COMP360 for treatment-resistant depression (TRD), highlighting both rapid onset and durable effects. In the COMP006 trial, two 25mg doses given three weeks apart produced a highly statistically significant 3.8-point reduction on the Montgomery-Åsberg Depression Rating Scale (MADRS) compared with a 1mg control, while COMP005 data showed that participants maintained meaningful improvement for at least 26 weeks after just one or two doses. The therapy was generally well-tolerated, with most adverse events mild or moderate and resolving within a day. Executives emphasized that durability of effect, a key point for the FDA, strengthens Compass’ case for a rolling NDA submission expected in the fourth quarter. COMP360’s profile is highly differentiated from Johnson & Johnson’s Spravato, which requires frequent dosing, and could mark a significant advance for the roughly four million Americans living with TRD. – Bree Foster
FDA reverses course on Moderna’s flu vaccine
The FDA has agreed to review Moderna’s mRNA-based flu vaccine, mRNA-1010, after previously issuing a refusal-to-file letter citing concerns about the Phase 3 trial comparator. Following a Type A meeting, the agency reversed its decision and set an August 5 action date. Moderna is seeking traditional approval for adults 50 to 64 and accelerated approval for those 65 and older, along with a commitment to conduct a post-marketing study in older adults; if cleared, the shot could be available for the 2026–27 flu season. The initial rejection, signed by FDA vaccine chief Vinay Prasad, drew industry backlash, with analysts suggesting the unusually rapid turnaround for the meeting may have influenced the reversal. The decision comes amid heightened scrutiny of mRNA vaccines under Department of Health and Human Services (HHS) leadership. – Andrea Corona
CDC vaccine advisory panel postpones February meeting
The CDC’s Advisory Committee on Immunization Practices (ACIP) has postponed its February meeting to an unspecified date next month amid ongoing turbulence in US vaccine policy. The session, which was expected to focus on COVID-19 and other mRNA vaccines, comes at a time of heightened scrutiny following HHS Secretary Robert F. Kennedy Jr.’s overhaul of the panel last year and broader shifts in federal immunization guidance. ACIP recommendations play a central role in shaping CDC policy and insurance coverage decisions, though final approval rests with CDC leadership, currently under interim direction by NIH Director Jay Bhattacharya. The delay also follows legal challenges from the American Academy of Pediatrics over recent panel staffing changes and revisions to the childhood immunization schedule, as well as reports that required disclosure deadlines to hold the meeting were missed. – Andrea Corona
