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Enodia snaps up competitor's pipeline for $127M & Ultragenyx AAV-based gene therapy strikes a win in phase 3

AuthorAuthor

Joachim Eeckhout & Eduardo Longoria
March 13, 2026

Good morning! Small molecules are having a rare-disease moment. Evaluate’s latest orphan drugs report found that small molecules now make up 45% of the 20 most valuable orphan drugs in development, well ahead of monoclonal antibodies at 20%. The poster child is Revolution Medicines’ daraxonrasib, a pan-RAS inhibitor now projected to reach $4 billion in 2032 sales.

Why it matters: That’s notable because only three of today’s top-selling orphan drugs are small molecules, making their heavy presence in the next wave of pipeline value look less like business as usual and more like a real resurgence. In other words, a modality that has been underrepresented among current orphan blockbusters is suddenly over-indexing in future winners.

Bottom line: Small molecules may have been discounted prematurely, especially now that more advanced chemistry and AI are expanding what they can do.

— Joachim E.

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SNAPSHOT

Enodia strikes up to $127M deal with Kezar to acquire competing protein-degradation pipeline

Two middle age business workers smiling happy and confident. Working together with smile on face hand giving high five at the office

Paris-based Enodia Therapeutics has acquired preclinical assets from Kezar Life Sciences’ Sec61-focused drug discovery program in a deal worth up to $127 million to strengthen its targeted protein degradation platform.

Why it matters: Protein degradation technologies are emerging as a promising strategy for treating diseases that have historically been difficult to target. Enodia’s approach could open new treatment possibilities in inflammation, immunology, and oncology by stopping harmful proteins before they fully form.

Backstory: Kezar previously advanced a Sec61 inhibitor, KZR-261, into clinical testing but halted the phase 1 trial in 2024 to focus on its immunoproteasome inhibitor program. Enodia, founded in Paris, recently raised €20.7 million in seed funding to develop small molecules targeting the Sec61 translocon, which is the gateway that allows newly formed proteins to enter the endoplasmic reticulum for completion.

Big picture: Most protein degradation technologies work after disease proteins are already produced inside cells. Enodia is pursuing an earlier intervention point, blocking problematic proteins during their production, which could offer a more efficient way to control disease pathways.

Zoom in: Under the agreement, Kezar receives $1 million upfront and could earn up to $127 million in development, regulatory, and commercial milestones, along with tiered royalties. Enodia will integrate more than a decade of Kezar’s research into its platform, which selectively targets the Sec61 translocon.

Yes, but: Enodia doesn’t intend to revive Kezar´s discontinued drug KZR-261. Instead, the company plans to leverage the acquired research to develop more selective Sec61 inhibitors that target pathogenic proteins involved in specific diseases.

What’s next: The newly acquired assets are expected to accelerate Enodia’s pipeline development and help the company advance programs toward IND-enabling studies and eventual clinical trials.

SNIPPETS

What’s happening in biotech today?

🔄 Comeback bid: Ultragenyx reported that its AAV-based gene therapy DTX301 met one of the co-primary endpoints in a phase 3 trial for ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder that prevents the body from properly clearing ammonia. After 36 weeks, treated patients showed 18% lower ammonia levels than placebo, a statistically significant improvement. The trial, which enrolled 37 patients, will next assess a second primary endpoint at 64 weeks, measuring how many patients can reduce or discontinue standard disease management. Results are expected in early 2027. Safety findings were consistent with prior studies, though liver inflammation occurred in one treated patient. Positive results could help Ultragenyx recover from recent regulatory setbacks, clinical failures, layoffs, and a class-action lawsuit.

🛡️ Neuron shield: Biogen presented updated early-stage data for salanersen, an antisense oligonucleotide drug being developed as a potential successor to Spinraza for spinal muscular atrophy (SMA), showing it slowed neurodegeneration and improved motor function in patients previously treated with Novartis’ gene therapy Zolgensma. In a study of 24 children aged six months to 12 years, the drug reduced neurofilament light chain levels by 75%, a marker of neurodegeneration, and half of the participants reached new motor function milestones, with all showing improvement on at least one endpoint after at least a year of follow-up. Biogen also outlined plans for three late-stage trials across different SMA populations, as the company positions salanersen, potentially dosed once yearly, to strengthen its SMA franchise amid competition from Zolgensma and Roche’s Evrysdi.

🧪 Trial win: BridgeBio Pharma reported statistically significant phase 3 results for its drug BBP-418 in the Fortify trial for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), a rare genetic disease that causes progressive muscle weakness and currently has no approved treatments. The study met both primary and secondary interim endpoints, with nearly 60% of treated patients reducing creatine kinase (CK) levels to within twice the normal limit and over 38% reaching normal CK levels, alongside improvements in functional outcomes compared with placebo. Analysts described the data as impressive and potentially disease-modifying, noting a favorable safety profile. BridgeBio plans to file for full FDA approval, which could enable a U.S. launch by the end of 2026, targeting a market the company estimates could exceed $1 billion in the U.S. and EU.

 💪 Muscle boost: Regenxbio reported updated phase 1/2 data showing a clean safety profile and functional improvements for its Duchenne muscular dystrophy (DMD) gene therapy RGX-202 as it approaches pivotal trial results expected early in the second quarter. Among 13 patients, no serious adverse events or liver toxicity, an issue seen with Sarepta’s Elevidys, were reported up to 24 months after treatment, and liver biomarkers remained within normal limits. Functional outcomes, including North Star Ambulatory Assessment scores and time-to-stand tests, suggested patients performed better than expected disease progression. All patients showed microdystrophin expression above the 10% threshold at Week 12. Regenxbio plans to seek an FDA meeting in mid-2026 to discuss accelerated approval.

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