Rocket Pharmaceuticals | Danon Disease - Highly Fatal Cardiomyopathy in kids | Phase 2 pivotal trial for acc approval completed enrollment | Expect completion late next year. Data on epidemiology earlier 25. Single arm trial, comparison to natural history. Outcomes are 12 months for LVMI and LAMP2 (biomarkers) |
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Capricor Therapeutics | Duchenne Muscular Dystrophy - Progressive disease in young kids | Filing for Full approval for DMD - Cardiomyopathy | Expect to file early next year and approval late next year. The original trial failed, but the FDA came to them after seeing OLE data. |
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Denali Therapeutics | Hunter syndrome - Progressive disease in children | Phase ½ study complete | Plans to file for AA in early 2025. Approval based on surrogate. Heparin sulfate is well established. High patient advocacy |
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Soleno | Prader Willi syndrome - No approved therapies. People have uncontrollable urge to eat | Near registrational | PDUFA Date March 27 |
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Stealth medicine | Bard Syndrome - No approved therapies, Kids | PDUFA Jan 29 | Advisory committee voted for approval after trial failed |
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Lexeo Therapeutics | Fridrich’s ataxia Cardiomyopathy - Major cause of death, usually late middle age | Phase 1 in progress | Plans to initialize trial 2025. 10% LVMI reduction, FXN protein expression via IHC |
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Regulus Therapeutics | ADPKD - Progressive disease requiring transplant (usually middle age) | Phase 1 trial completing, Phase 2 starting next year after alignment | Meeting with FDA to determine path forward in Q4. Potential for AA based on cyst volume which was not allowed for Tolvaptan (other approved drug) |
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Disc Medicine | Erythropoeitic protoporphyria (patients can’t be in the sun at all) | Completed Phase 2 trial | Meet with FDA Q1 and update after. Positive on PPIX benefit but not secondary endpoint of time in sunlight. Use PPIX as biomarker for accelerated approval |
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Uniqure | Huntington’s disease - progressive neurological deficits and death in middle age | Phase 2 complete | Potential AA based on small trial compared to natural history |
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Edgewise therapeutics | Becker Muscular Dystrophy - progressive muscle dysfunction in middle age | Phase 2 trial complete | Trends in NSAA, positive CK benefit. Unclear. Plans to go to the FDA |
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Ovid Therapeutics/Takeda | Dravet Syndrome | Missed primary endpoint | Takeda plans to engage the FDA. |
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Larimar Therapeutics | Friedrich's Ataxia | Filing for approval late 2025 | Concerns over safety, approval based on Surrogate endpoint. |
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