Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
New 3D brain atlas bridges anatomy and imaging to map white matter connections
Researchers at the University of Trento have created BraDiPho, a photogrammetry-based 3D atlas that integrates ex vivo brain dissections with in vivo MRI data to more accurately map white matter connections. Published in Nature Communications, the tool combines neuroscience, artificial intelligence, and neuroanatomy to generate high-resolution, navigable models that bridge anatomical and radiological views of the brain. The open-access platform, available at bradipho.eu, provides neurosurgeons and researchers with a validated reference for studying, teaching, and planning interventions involving the brain’s structural pathways. – Andrea Corona
Roche bets on Manifold’s platform to engineer next-generation shuttles to the brain
By paying $55 million upfront, Roche will apply Manifold Bio’s artificial intelligence (AI)-guided direct-to-vivo platform to develop shuttles to carry their therapies across the blood-brain barrier. The deal could be worth up to $2 billion for Manifold depending on whether milestones are hit. As part of the collaboration, Manifold will lead initial activities to identify and develop the shuttles for Roche’s products, while Roche would then take the reins on preclinical, clinical, and commercialization efforts. “Today we’ve built the world’s first AI system for the design of tissue-targeted biologics. With the resulting data, we are already building the world’s first virtual organism model. The collaboration with Roche validates our vision that the future of biomedicine lies in data-driven, generative design directly informed by living biology,” said Pierce Ogden, cofounder and Chief Technology Officer of Manifold Bio in the press release. – Allison Whitten
Researchers reveal deadly virus strategy that could change antiviral design
Researchers in Australia have uncovered how viruses like rabies manage to hijack and control human cells despite carrying only a handful of genes — a finding that could open the door to new antivirals and vaccines. The study, led by scientists at Monash University and the University of Melbourne and published in Nature Communications, shows that a key rabies virus protein, known as P protein, can change its shape and bind to RNA inside human cells. This flexibility allows the virus to infiltrate multiple membraneless organelles within the cell and disrupt critical systems, including immune defenses and protein production. This shape-shifting mechanism helps to explain how small RNA viruses can be so lethal despite their simplicity. Because similar strategies are likely used by dangerous viruses such as Nipah and Ebola, targeting this newly uncovered mechanism could lead to broad-spectrum antiviral approaches. – Bree Foster
FDA approves first treatment for ultra-rare mitochondrial disorder
Patients with one of the world’s rarest mitochondrial disorders now have an FDA-approved treatment for the first time. UCB’s Kygevvi, a combination of doxecitine and doxribtimine, is now approved for use in adults and children whose symptoms begin at or before age 12, marking the first therapy for thymidine kinase 2 deficiency (TK2d). The ultra-rare disease, which affects fewer than two in a million people, stems from a genetic defect that prevents cells from producing and repairing mitochondrial DNA, leading to progressive muscle weakness and potentially life-threatening respiratory failure. Kygevvi works by delivering pyrimidine nucleosides to skeletal muscle mitochondria to help rebuild DNA. Approval was supported by a Phase 2 trial, two retrospective chart reviews, and an expanded access program, which together showed the therapy reduced the risk of death by around 86 percent compared to untreated patients. Common side effects included diarrhea, vomiting, and abdominal pain. The company expects Kygevvi to be available in the US early next year, with European regulatory review ongoing. – Bree Foster
FDA rejects Biohaven’s experimental treatment for rare neurodegenerative disease
The FDA has issued a Complete Response Letter for Biohaven’s New Drug Application for its investigational therapy targeting spinocerebellar ataxia (SCA), a hereditary neurodegenerative disorder with no approved treatments. The company’s submission included data showing a 50 to 70 percent slowing of disease progression and more than a 50 percent reduction in fall risk among treated patients. However, the FDA cited concerns about potential bias and confounding factors common to externally controlled studies. The therapy had received Orphan, Fast Track, and Priority Review designations. Following the rejection, Biohaven said it will meet with the FDA to discuss next steps and will shift focus to its late-stage programs, including extracellular degraders for IgA nephropathy and Graves’ disease, the Kv7 ion channel activator opakalim, and the myostatin-activin inhibitor taldefgrobep alfa, while reducing annual R&D spending by about 60 percent. – Andrea Corona
Blackstone to fund Merck’s antibody-drug conjugate
On Tuesday, Merck announced an agreement to receive $700 million from Blackstone Life Sciences to help develop their antibody-drug conjugate, sacituzumab tirumotecan (sac-TMT). Sac-TMT targets trophoblast cell-surface antigen 2 (TROP2), a protein that is overexpressed in a variety of cancers like pancreatic, ovarian, and prostate. Merck’s asset was developed by Kelun-Biotech, and it’s currently in 15 global Phase 3 trials. Per the agreement, Blackstone’s nonrefundable $700 million will go towards anticipated costs in 2026, and the company is eligible to receive low-to-mid single-digit royalties on net sales upon FDA approval of sac-TMT as a first-line therapeutic for triple-negative-breast cancer. Antibody-drug conjugates continue to be one of the most promising and profitable segments in oncology, with the global market valued at about $11 billion in 2023 and expected to surpass $24 billion by 2030 as demand and investment surge. For Blackstone, the agreement builds on their growing portfolio of investments in biotech and startup company drugs, including a recent payout from their 2020 investment in Alnylam Pharmaceuticals. – Allison Whitten
