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Pinnacle raises $89M for its oral peptide drugs & Beam reports positive Phase 1/2 trial of its base editing therapy

Good Morning! The FDA just reminded rare disease biotechs that flexibility isn’t dead yet.

What happened: Denali won accelerated approval for Avlayah, the first biologic designed to cross into the brain and treat the neurological effects of Hunter syndrome, a rare genetic disorder affecting about 500 people in the U.S. The decision lands after months of FDA reversals, delays, and public criticism over how the agency has handled rare disease therapies.

Why it matters: Hunter syndrome is a life-threatening disorder, usually affecting boys, in which the body lacks an enzyme needed to break down certain sugar molecules. Those molecules accumulate and damage the skeleton, heart, brain and other organs. Existing treatments have not been designed to address neurological symptoms by entering the central nervous system.

Bottom line: Avlayah isn’t just a win for Denali and the patients, but also for other rare disease biotechs fighting for approval.

— Joachim E.

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SNAPSHOT

Pinnacle Medicines raises $89M to accelerate oral peptide drugs

Plaid | Blender 3D

Pinnacle Medicines secured an $89 million Series B, bringing total funding to $134 million, to advance its oral peptide drug pipeline into early clinical trials.

Why it matters: Oral alternatives to injectable peptide drugs, like GLP-1 therapies, could dramatically improve patient convenience, adherence and market adoption, making this a high-stakes race in biotech.

Backstory: Founded in 2024, Pinnacle focuses on developing orally delivered peptide therapeutics, traditionally limited by poor absorption. The company integrates AI and physics-based simulations to design more effective compounds.

Big picture: The push for oral peptide drugs is intensifying across pharma, with major players investing billions to replace injections with pills, especially in obesity, diabetes and chronic disease markets. Among the players in this game is Novo Nordisk, which recently launched an oral Wegovy and Merck & Co. (MSD), which licensed an oral peptide platform from Cyprumed for $493M.

Zoom in: The round was backed by OrbiMed, LAV, Foresite Capital, RA Capital, Logos Capital and others across the U.S. and China.

What’s next: Funds will support preclinical programs through early human trials and expand discovery efforts. The initial targets for this funding round are immunology and cardiometabolic diseases.

SNIPPETS

What’s happening in biotech today?

🧬 Editing win: Beam Therapeutics reported positive updated data from a Phase 1/2 trial of its base editing therapy, BEAM-302, for alpha-1 antitrypsin deficiency (AATD), a rare liver and lung disease caused by a genetic mutation that produces misfolded proteins. In 29 patients across multiple dose groups, the treatment restored production of functional AAT protein above protective levels and reduced harmful misfolded protein by over 80%. While two patients showed elevated liver enzymes, no treatment was required. Beam plans to advance a 60 mg dose into further trials and pursue accelerated FDA approval, positioning the therapy as a potentially transformative approach in a competitive field.

🚪NK exit: Innate Pharma has discontinued development of its NK cell engager IPH6501 in a phase 1/2 trial for B-cell non-Hodgkin lymphoma as part of a broader strategy to prioritize higher-value pipeline assets. The decision follows earlier signals that the drug was not among the company’s top priorities, alongside workforce reductions aimed at focusing resources. Innate will instead emphasize candidates such as IPH4502, an antibody-drug conjugate showing early anti-tumor activity, as well as late-stage programs including lacutamab for Sézary syndrome and monalizumab, partnered with AstraZeneca, for lung cancer. The company also noted shifting dynamics in its Sanofi collaboration, with some NK cell engager assets deprioritized or returned.

🧓 Aging play: Pulmatrix has agreed to a reverse merger with private biotech Eos SENOLYTIX following the collapse of its prior deal with Cullgen, shifting its focus to Eos’s lead candidate, PTC-2105, which targets age-related muscle loss and metabolic health. The combined company, to trade as “EOSX,” will be primarily owned by Eos investors (94%), with Pulmatrix shareholders holding about 6%, and is expected to close in mid-2026 alongside $19 million in new financing. Eos’s approach centers on mitochondrial-targeted therapies designed to improve muscle mass, reduce fat, and address aging-related diseases, positioning PTC-2105 as a potential alternative or complement to GLP-1 treatments.

🤝 Licensing deal: Korean biotech Alteogen has signed an exclusive licensing deal with Biogen worth up to $579 million to develop and commercialize subcutaneous formulations of two biologic drugs using its Hybrozyme-based ALT-B4 technology. The agreement includes $20 million upfront, $10 million tied to development of a second product, and up to $549 million in milestone payments, along with royalties on future sales. Biogen also has the option to expand the collaboration to a third product.

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