Solid Biosciences(SLDB)

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SLDB Stock Up as FDA Clears IND for SGT-212 in Friedreich's Ataxia
ZACKS· 2025-01-08 16:26
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]
Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
GlobeNewswire News Room· 2025-01-07 21:15
- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich’s ataxia - - Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease - - Phase 1b clinical trial initiation expected in 2H 2025 - - Company to hold a conference call tomorrow, January 8, 2025, at 8:30 AM ET - CHARLESTOWN, Mass., Jan. 07, 2025 (GLOBE NEWSWI ...
Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich's Ataxia
Newsfilter· 2025-01-07 21:15
- SGT-212 is the only full-length frataxin replacement gene therapy candidate targeting the CNS and cardiac manifestations of Friedreich's ataxia - - Dual route of administration enables direct delivery of AAV-based gene therapy to the cerebellum and heart to potentially address the most significant symptoms of the disease - - Phase 1b clinical trial initiation expected in 2H 2025 - - Company to hold a conference call tomorrow, January 8, 2025, at 8:30 AM ET - CHARLESTOWN, Mass., Jan. 07, 2025 (GLOBE NEWSWI ...
Solid Biosciences Added to the Nasdaq Biotechnology Index
Globenewswire· 2024-12-23 21:05
CHARLESTOWN, Mass., Dec. 23, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it was added to the Nasdaq Biotechnology Index® (Nasdaq: NBI) effective prior to the market open on Monday, December 23, 2024. The Nasdaq Biotechnology Index is designed to track the performance of a set of securities listed on The Nasdaq Stock Market® (Nasdaq®) that ...
Solid Biosciences Positioned For Next-Gen Duchenne Muscular Dystrophy Treatment Success: Analyst
Benzinga· 2024-12-13 18:52
Core Viewpoint - Wedbush initiated coverage on Solid Biosciences, Inc., a gene therapy company focused on treating Duchenne muscular dystrophy (DMD) [1] Company Overview - Solid Biosciences is developing SGT-003, the leading program in its pipeline, with plans to advance additional projects toward clinical trials in 2025 [2] - The therapy utilizes AAV-SLB101 capsid and a specialized cargo (nNOS binding domain) to potentially enhance dystrophin expression compared to earlier treatments [2][3] Market Potential - The analyst notes that while first-generation gene therapies for DMD have opened new treatment possibilities, there is still significant room for improvement [3] - Solid Biosciences' innovative approach and novel capsid technology could improve microdystrophin delivery [3] Milestones and Projections - The initial Phase 1/2 INSPIRE DUCHENNE trial results are expected in the first quarter of 2025, marking a significant near-term milestone for the company [4] - The approval of Sarepta Therapeutics Inc's Elevidys is seen as a key milestone, but the market still requires newer treatments with better results [4] Financial Outlook - Solid Biosciences' stock is currently trading around $5, with approximately $4 per share in cash providing some downside protection [4] - The company ended Q3 2024 with about $171.1 million in cash and equivalents, with a cash runway anticipated into 2026 [6] - Assuming SGT-003 follows a timeline similar to Sarepta's Elevidys, even a one-year delay would lower the price target to $11 per share, still above the current trading price of $4.83 [5][7] Analyst Rating - The analyst sees strong potential in Solid Biosciences and has initiated coverage with an Outperform rating [5]
Solid Biosciences to Participate at Upcoming Investor Conferences
GlobeNewswire News Room· 2024-11-26 13:00
CHARLESTOWN, Mass., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced participation in the following investor conferences: Citi’s 2024 Global Healthcare Conference – Miami, FLBo Cumbo, President and CEO, will participate in a panel discussion titled “Neuromuscular Disorders” on Wednesday, December 4, 2024, at 1:00 p.m. ET. Piper Sandler 36th ...
Solid Biosciences to Present at the Jefferies London Healthcare Conference
GlobeNewswire News Room· 2024-11-14 13:00
Core Viewpoint - Solid Biosciences Inc. is actively engaging in the life sciences sector, focusing on developing precision genetic medicines for neuromuscular and cardiac diseases, with a presentation scheduled at the Jefferies London Healthcare Conference [1]. Company Overview - Solid Biosciences is dedicated to advancing a portfolio of gene therapy candidates, including SGT-003 for Duchenne muscular dystrophy, SGT-501 for catecholaminergic polymorphic ventricular tachycardia, and AVB-401 for BAG3-mediated dilated cardiomyopathy, among others [3]. - The company aims to improve the daily lives of patients affected by rare neuromuscular and cardiac diseases, emphasizing a patient-focused approach and collaboration among experts in various fields [3]. Event Details - The presentation at the Jefferies London Healthcare Conference will take place on November 20, 2024, at 1:30 p.m. GMT (8:30 a.m. ET) [1]. - A live webcast of the presentation will be available on the company's website, with a replay archived for 30 days [2].
Solid Biosciences(SLDB) - 2024 Q3 - Quarterly Report
2024-11-06 21:02
UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 Securities registered pursuant to Section 12(b) of the Act: Title of each class Trading Symbol Name of exchange on which registered Common Stock, $0.001 par value per share SLDB The Nasdaq Global Select Market FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIE ...
Solid Biosciences Reports Second Quarter 2024 Financial Results and Provides Business Updates
GlobeNewswire News Room· 2024-08-13 20:08
– Patients dosed in Phase 1/2 INSPIRE DUCHENNE trial of SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne); Dosing was well tolerated in all patients with initial data expected Q4 2024 – – Solid plans to expand patient dosing with additional clinical trial sites in the U.S., Canada and Europe and plans accelerated production of multiple GMP batches of SGT-003 to support trial expansion – – Targeting the submission of 3-4 INDs by the end of 2026, strategically selecting cardiac and neuromusc ...
Solid Biosciences Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)
Newsfilter· 2024-07-01 20:19
CHARLESTOWN, Mass., July 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company"), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced a grant of 11,847 restricted stock units ("RSUs") to one newly hired employee. Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treat ...