February 6, 2025 - 🧬 [nGram] Today’s News Scoop: Annovis Phase 3, Recursion Phase 2, Ultragenyx Gene Therapy

1. Annovis begins pivotal phase 3 study of buntanetap for early Alzheimer's disease

   • Annovis Bio has initiated a pivotal Phase 3 trial for buntanetap in early Alzheimer's disease.
   • The study is a randomized, placebo-controlled, double-blind trial lasting 18 months.
   • Primary outcomes include cognitive and functional assessments using ADAS-Cog13 and ADCS-iADL scales.
   • The trial aims to enroll over 750 participants across approximately 100 sites in the U.S.
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2. Recursion presents phase 2 data for REC-994 in CCM at international stroke conference

   • REC-994 met the primary endpoint of safety and tolerability in CCM patients with no treatment-related discontinuations or Grade 3 adverse events.
   • Promising signals observed in MRI-based lesion volume reduction and functional outcomes with REC-994 400 mg compared to placebo.
   • In brainstem cavernomas, REC-994 400 mg showed decreases in lesion volume and improvements in mRS scores.
   • Next steps include regulatory discussions and an ongoing long-term extension study.
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3. Ultragenyx announces new data on UX111 gene therapy for Sanfilippo syndrome type A

   • UX111 gene therapy showed significant improvement in cognitive and communication functions in children with Sanfilippo syndrome type A.
   • The therapy led to a 65% reduction in cerebrospinal fluid heparan sulfate levels, indicating a positive biological response.
   • The treatment was well-tolerated, with mild to moderate liver enzyme elevations as the most common side effect.
   • Data from the study supports a Biologics License Application submitted to the FDA, with a decision expected in the second half of 2025.
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4. Viralgen and CTNNB1 Foundation partner to manufacture a new gene therapy for CTNNB1 Syndrome

   • Viralgen and the CTNNB1 Foundation are collaborating to develop an AAV investigational gene therapy for CTNNB1 Syndrome.
   • The therapy aims to restore gene function, potentially improving motor and cognitive development in affected children.
   • The partnership leverages Viralgen's AAV manufacturing expertise and the CTNNB1 Foundation's research commitment.
   • Clinical trials for the gene therapy are expected to begin next year, with potential patient benefits by mid-2025.
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5. New England Journal of Medicine publishes results of global, multicenter eNRGy study evaluating zenocutuzumab-zbco in NRG1+ cancer

   • The eNRGy trial is a global, multicenter, single-arm, Phase 2 study involving 204 patients with NRG1 gene fusions.
   • Zenocutuzumab-zbco (BIZENGRI) showed promising overall response rates and durability in patients with advanced pancreatic adenocarcinoma and NSCLC.
   • The FDA granted accelerated approval for BIZENGRI based on the trial's results, with continued approval contingent on confirmatory trials.
   • Partner Therapeutics holds exclusive rights to develop and commercialize zenocutuzumab-zbco in the U.S. and is working on its commercial distribution.
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