SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia

Company Update: Solid Biosciences Inc (SLDB) - The FDA has cleared the investigational new drug (IND) application for SGT-212, a novel AAV-based gene therapy candidate targeting Friedreich's ataxia (FA) [1] - SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the cardiac manifestations of FA [1] - The therapy aims to address the neurologic and systemic clinical manifestations of FA, covering the full spectrum of disease progression [2] - Shares of Solid Biosciences surged 17.4% in after-hours trading on Jan 7 and continued to rise in pre-market trading on Jan 8 [2] - Over the past year, SLDB shares have declined 51.4%, significantly underperforming the industry's 15.7% decline [2] Clinical Development of SGT-212 - The IND is supported by robust preclinical data on SGT-212 [3] - A phase Ib study for SGT-212 is planned to initiate in the second half of 2025 [3] - The study will evaluate the safety and tolerability of systemic intravenous infusion and direct intradentate nuclei (IDN) infusion in both non-ambulatory and ambulatory adult FA patients [3] - The dual route of administration enables direct delivery of SGT-212 to the heart and cerebellum, potentially addressing the most significant symptoms of FA [4] - SGT-212 represents a differentiated approach to treating the underlying disease [4] Competitive Landscape in FA Market - FA is a rare disease, with limited companies developing treatments [7] - Biogen (BIIB) markets Skyclarys, the first FDA-approved treatment for FA, approved in 2023 in the US and February 2024 in Europe [7] - Skyclarys was added to Biogen's portfolio through the acquisition of Reata Pharmaceuticals in September 2023 [7] - Biogen reports strong progress in the US and EU launch of Skyclarys, with increasing global demand [8] - Larimar Therapeutics (LRMR) is developing nomlabofusp (CTI-1601) as a potential FA treatment, with plans to file a biologics license application in the second half of 2025 [8] - Nomlabofusp has the potential to be the first frataxin protein replacement therapy for FA patients [9] Stock Performance and Industry Comparison - Solid Biosciences currently holds a Zacks Rank 3 (Hold) [10] - Puma Biotechnology (PBYI), a better-ranked stock in the biotech sector, holds a Zacks Rank 1 (Strong Buy) [10] - PBYI's 2025 earnings per share estimates have increased from 42 cents to 54 cents over the past 60 days [10] - PBYI shares have declined 21.7% over the past year [10] - Puma Biotechnology has beaten earnings estimates in three of the trailing four quarters, with an average surprise of 32.78% [11]