February 13, 2025 - 🧬 [nGram] Today’s News Scoop: Genentech's Evrysdi Tablet Approved, NKGen's Fast Track, Alto's Trial Success

1. FDA approves Genentech’s Evrysdi tablet for spinal muscular atrophy

   • The FDA has approved Genentech's Evrysdi tablet for spinal muscular atrophy (SMA), making it the first non-invasive disease-modifying treatment for SMA.
   • The approval is based on a bioequivalence study showing the 5 mg tablet provides comparable exposure to the original oral solution.
   • The tablet is suitable for individuals aged 2 years or older, weighing more than 44 lbs (20 kgs), and offers a convenient treatment option.
   • Evrysdi is currently approved in over 100 countries and is being evaluated in multiple global clinical trials for SMA.
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2. NKGen Biotech receives U.S. FDA fast track designation for troculeucel for the treatment of moderate Alzheimer’s disease

   • NKGen Biotech's troculeucel receives FDA Fast Track designation for moderate Alzheimer's disease.
   • The designation accelerates the drug's development and review process, potentially leading to faster market access.
   • Troculeucel is an ex vivo expanded autologous NK cell therapy, currently in Phase 2a trials.
   • NKGen plans to share updated clinical data by the end of 2025.
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3. Alto neuroscience announces favorable outcome from interim analysis of ALTO-300 phase 2b major depressive disorder trial

   • Alto Neuroscience announced positive interim results for the Phase 2b trial of ALTO-300 for major depressive disorder.
   • The trial will continue with an additional 50 biomarker positive patients, aiming for a total of 200.
   • The study is double-blind and placebo-controlled, focusing on patients with an EEG biomarker signature.
   • Topline results are expected by mid-2026.
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4. Alpha Cognition secures additional U.S. patent for ZUNVEYL in Alzheimer's treatment

   • Alpha Cognition has been granted a new U.S. patent for ZUNVEYL, a treatment for mild to moderate Alzheimer's disease.
   • The patent covers the composition of tablet formulations for benzgalantamine, extending protection until 2044.
   • This milestone aligns with the planned U.S. launch of ZUNVEYL this quarter.
   • ZUNVEYL will be available by prescription in pharmacies nationwide in Q1 2025.
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5. Restore Vision doses first patient in phase I/II trial of RV-001 for retinitis pigmentosa

   • Restore Vision has initiated a Phase I/II clinical trial for RV-001, a gene therapy for retinitis pigmentosa (RP).
   • The trial is open-label and aims to assess safety, light-activation, and light-sensitivity in patients with advanced RP.
   • RV-001 uses adeno-associated virus vectors to deliver a gene encoding 'Chimeric Rhodopsin' to enhance light sensitivity in retinal interneurons.
   • The trial is supported by the Japan Agency for Medical Research and Development and academic grants from Keio University and Nagoya Institute of Technology.
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6. Newleos therapeutics secures $93.5 million in series A financing

   • Newleos Therapeutics raised $93.5 million in an oversubscribed Series A financing led by Goldman Sachs Alternatives.
   • The funds will support proof-of-concept clinical trials for their pipeline, in-licensed from Roche, targeting neuropsychiatric disorders.
   • Lead program NTX-1955, a GABAA-γ1 selective PAM, aims to treat anxiety disorders with fewer side effects.
   • Additional assets include NTX-1472, NTX-2001, and NTX-1511, targeting social anxiety, substance use disorders, and cognitive impairment, respectively.
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