Solid Biosciences Outlines Key Priorities for Advancing Diversified Neuromuscular and Cardiac Development Pipeline and Establishing Leadership in Precision Genetic Medicines
Solid Biosciences (NASDAQ: SLDB) provided updates on its neuromuscular and cardiac development pipeline. The company has dosed four patients in the INSPIRE DUCHENNE trial for SGT-003, with initial three-patient data expected in Q1 2025. The FDA cleared an IND for SGT-212, a dual-route administration gene therapy for Friedreich's ataxia, with first-in-human trials planned for 2H 2025.
The company plans to submit an IND for SGT-501 to treat CPVT in 1H 2025. Additionally, Solid is advancing SGT-601 for TNNT2 Thin Filament Cardiomyopathy with IND submission planned for 2H 2026. The company has also collaborated with Mayo Clinic for developing therapies for cardiac conditions.
Solid enters 2025 with approximately $148.9 million in cash and investments, expected to fund key priorities into 2026.
Solid Biosciences (NASDAQ: SLDB) ha fornito aggiornamenti sul suo pipeline di sviluppo neuromuscolare e cardiaco. L'azienda ha trattato quattro pazienti nello studio INSPIRE DUCHENNE per SGT-003, con i dati iniziali su tre pazienti attesi nel Q1 2025. La FDA ha approvato un IND per SGT-212, una terapia genica a somministrazione duale per l'atassia di Friedreich, con i primi trial sull'uomo pianificati per il 2° semestre del 2025.
L'azienda prevede di presentare un IND per SGT-501 per trattare la CPVT nel 1° semestre del 2025. Inoltre, Solid sta progredendo con SGT-601 per la cardiomiopatia a filamenti sottili TNNT2, con la presentazione dell'IND prevista per il 2° semestre del 2026. L'azienda ha anche collaborato con la Mayo Clinic per sviluppare terapie per condizioni cardiache.
Solid entra nel 2025 con circa $148,9 milioni in contante e investimenti, previsti per finanziare priorità chiave fino al 2026.
Solid Biosciences (NASDAQ: SLDB) proporcionó actualizaciones sobre su pipeline de desarrollo neuromuscular y cardíaco. La empresa ha tratado a cuatro pacientes en el estudio INSPIRE DUCHENNE para SGT-003, con los primeros datos de tres pacientes esperados para el primer trimestre de 2025. La FDA aprobó un IND para SGT-212, una terapia génica de administración dual para la ataxia de Friedreich, con ensayos en humanos previstos para el segundo semestre de 2025.
La empresa planea presentar un IND para SGT-501 para tratar la CPVT en el primer semestre de 2025. Además, Solid está avanzando con SGT-601 para la cardiomiopatía de filamento delgado TNNT2, con la presentación del IND programada para el segundo semestre de 2026. La compañía también ha colaborado con la Mayo Clinic para desarrollar terapias para condiciones cardíacas.
Solid entra en 2025 con aproximadamente $148.9 millones en efectivo e inversiones, que se espera financien prioridades clave hasta 2026.
솔리드 바이오사이언스(Solid Biosciences, NASDAQ: SLDB)는 근육신경 및 심장 개발 파이프라인에 대한 업데이트를 제공하였습니다. 회사는 SGT-003의 INSPIRE DUCHENNE 시험에서 4명의 환자에게 투약하였으며, 초기 3명 환자의 데이터는 2025년 1분기에 기대됩니다. FDA는 프리드리히 확산증에 대한 이중 경로 전달 유전자 요법인 SGT-212에 대한 IND를 승인하였으며, 인체 시험은 2025년 하반기에 계획되어 있습니다.
회사는 2025년 1분기에 CPVT 치료를 위한 SGT-501의 IND를 제출할 계획입니다. 또한, 솔리드는 TNNT2 얇은 필라멘트 심근병증을 위해 SGT-601을 진행하고 있으며, IND 제출은 2026년 하반기로 계획되어 있습니다. 회사는 또한 심장 질환 치료제를 개발하기 위해 메이요 클리닉과 협력하였습니다.
솔리드는 2025년을 약 $148.9 백만의 현금 및 투자 금액으로 시작하며, 이는 2026년까지 주요 우선 순위를 지원할 것으로 예상됩니다.
Solid Biosciences (NASDAQ: SLDB) a fourni des mises à jour sur son pipeline de développement neuromusculaire et cardiaque. L'entreprise a dosé quatre patients dans l'essai INSPIRE DUCHENNE pour SGT-003, avec des données initiales concernant trois patients attendues pour le premier trimestre 2025. La FDA a approuvé un IND pour SGT-212, une thérapie génique à administration double pour l'ataxie de Friedreich, avec des essais chez l'homme prévus pour le second semestre 2025.
L'entreprise prévoit de soumettre un IND pour SGT-501 pour traiter la CPVT au premier semestre 2025. De plus, Solid progresse avec SGT-601 pour la cardiomyopathie TNNT2 à filament mince, avec une soumission d'IND prévue pour le second semestre 2026. L'entreprise a également collaboré avec le Mayo Clinic pour développer des thérapies pour des conditions cardiaques.
Solid aborde 2025 avec environ $148,9 millions en liquidités et investissements, qui devraient financer des priorités clés jusqu'en 2026.
Solid Biosciences (NASDAQ: SLDB) hat Updates zu seinem Entwicklungsportfolio für neuromuskuläre und kardiale Therapien bereitgestellt. Das Unternehmen hat vier Patienten in der INSPIRE DUCHENNE-Studie für SGT-003 behandelt, wobei die ersten Daten von drei Patienten im ersten Quartal 2025 erwartet werden. Die FDA hat eine IND für SGT-212 genehmigt, eine duale Verabreichungsgentherapie für Friedreich-Ataxie, deren erste Tests am Menschen für das zweite Halbjahr 2025 geplant sind.
Das Unternehmen plant die Einreichung einer IND für SGT-501 zur Behandlung von CPVT im ersten Halbjahr 2025. Darüber hinaus entwickelt Solid SGT-601 für TNNT2 dünne Filamentkardiomyopathie, mit einer IND-Einreichung, die für das zweite Halbjahr 2026 vorgesehen ist. Das Unternehmen hat auch mit der Mayo Clinic zusammengearbeitet, um Therapien für Herzkrankheiten zu entwickeln.
Solid startet 2025 mit ungefähr $148,9 Millionen an Bargeld und Investments, die voraussichtlich Schlüsselprioritäten bis 2026 finanzieren werden.
- Strong cash position of $148.9 million, funding operations into 2026
- FDA IND clearance received for SGT-212 Friedreich's ataxia treatment
- Successful dosing of four patients in INSPIRE DUCHENNE trial with no SAEs observed
- Expansion of pipeline with multiple clinical-stage assets
- Strategic collaboration with Mayo Clinic for cardiac gene therapy development
- Initial trial data for SGT-003 not available until Q1 2025
- SGT-212 human trials won't begin until 2H 2025
- Multiple programs still in early development stages with uncertain outcomes
Insights
The company's financial position shows $148.9 million in cash and investments as of December 2024, providing runway into 2026. This strengthens their ability to execute multiple clinical programs without immediate capital needs. The diversified pipeline strategy across neuromuscular and cardiac diseases significantly reduces single-program risk.
The licensing of AAV-SLB101 to 15 academic labs and corporations represents a potential recurring revenue stream. The Mayo Clinic collaboration for six cardiac programs expands their intellectual property portfolio and market opportunities. The dual-administration approach for Friedreich's ataxia could create a first-mover advantage in this indication.
The clinical progress is noteworthy with four patients dosed in the INSPIRE DUCHENNE trial showing positive safety data. The upcoming Q1 2025 data readout from the first three patients will be crucial. The FDA IND clearance for SGT-212 in FA utilizing a novel dual route of administration (IDN and IV) represents a significant technical advancement in gene therapy delivery.
The expansion into cardiac indications with SGT-501 for CPVT and development of proprietary capsid libraries demonstrates technical depth. The focus on manufacturing excellence with improved full-to-empty capsid ratios could enhance therapy efficacy and reduce production costs.
With a market cap of
The immunomodulation studies for potential re-dosing in pre-treated patients could unlock a substantial market of previously treated patients. The geographic expansion with MHRA clearance in the UK demonstrates growing international presence. The development of multiple proprietary technologies creates additional partnership and licensing opportunities.
- Duchenne: Dosed four patients in INSPIRE DUCHENNE clinical trial; SGT-003 has been well tolerated in all patients with no SAEs observed; initial three patient data expected Q1 2025 -
- FA: First-in-human clinical study of SGT-212 utilizing a dual route of administration to treat Friedreich’s ataxia (FA) anticipated in 2H 2025 following recent FDA IND clearance -
- CPVT: Submission of IND for SGT-501 on track for 1H 2025; the submission will expand clinical-stage portfolio to three unique candidates in diverse indications with significant unmet need -
- Cash: Company enters 2025 with approximately
CHARLESTOWN, Mass., Jan. 15, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will provide a corporate update outlining the Company’s expanded clinical-stage pipeline and 2025 objectives as it completes its transformation into a multi-program leader in the development of precision genetic medicines. Bo Cumbo, President and CEO, and Gabriel Brooks, M.D., Chief Medical Officer, will provide the update at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 3:45 p.m. PT (6:45 p.m. ET).
“At the time I joined Solid Biosciences there was a singular focus on genetic treatments for Duchenne muscular dystrophy,” said Bo Cumbo, President and CEO of Solid Biosciences. “In the last two years, we have broadened the pipeline to include gene therapy treatments for other devastating genetic neuromuscular and cardiac diseases, entered the clinic with our next generation gene therapy for Duchenne, opened a second IND with an industry first dual route of administration gene therapy to treat Friedreich’s ataxia, and continued to make advancements in next-generation capsid technologies, novel promoters, immunomodulation techniques, and manufacturing excellence. We are committed to innovating and building a true ‘next generation’ genetic medicines company, while maintaining Solid’s historic focus on the needs of the patient.”
He continued: “With two clinical stage assets and a third IND submission anticipated in 1H 2025, Solid Biosciences stands on the threshold of realizing that vision: a platform poised to create transformative genetic medicines for patients and create value for our shareholders.”
Highlights from the presentation to be given at the J.P. Morgan Healthcare Conference include:
Neuromuscular Pipeline
SGT-003 for Duchenne muscular dystrophy (Duchenne)
- SGT-003 continues to be well tolerated in the first four patients dosed in the ongoing INSPIRE DUCHENNE clinical trial.
- Patient enrollment and dosing under the expanded INSPIRE DUCHENNE protocol is ongoing.
- Six clinical sites are activated in North America (five in the U.S., one in Canada).
- The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) cleared the INSPIRE DUCHENNE clinical trial application (CTA) with initial site activation expected in 2H 2025.
- Solid expects to report data from the first three patients dosed in Q1 2025.
SGT-212 for Friedreich’s ataxia (FA)
- As announced on January 7, 2025, the FDA has cleared the IND for SGT-212 for the treatment of cardiac and CNS manifestations of FA. SGT-212 is the first and only gene therapy to utilize a dual route of administration to treat FA.
- FA is a degenerative disease caused by insufficient levels of frataxin protein. SGT-212 is an AAV-based FA gene therapy candidate designed to deliver full-length frataxin via direct intradentate nuclei (IDN) infusion into the cerebellum to treat neurologic manifestations and systemic intravenous (IV) infusion to target cardiac and systemic manifestations.
- The Company expects to initiate a first-in-human, open-label, Phase 1b clinical trial of SGT-212 in 2H 2025. The study will enroll non-ambulatory and ambulatory adult patients living with FA across up to three cohorts and will evaluate the safety and tolerability of contemporaneous systemic and bilateral IDN administration of SGT-212.
Cardiac Pipeline
SGT-501 for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
- IND-enabling Good Laboratory Practice (GLP) toxicology studies in non-human primates (NHP) are underway, with the in-life portion of the six-month GLP toxicology study expected to be completed in Q1 2025.
- The Company anticipates submitting an IND for SGT-501 for the treatment of CPVT in 1H 2025.
SGT-601 for TNNT2 Thin Filament Cardiomyopathy
- Preclinical IND-enabling studies are underway and planned throughout 2025, with anticipated IND submission in 2H 2026.
Mayo Clinic Collaboration
- As announced on December 4, 2024, Solid entered into a collaboration with Mayo Clinic to develop an AAV gene therapy platform for the development of therapies to treat sudden cardiac death-predisposing genetic cardiomyopathies and channelopathies.
- Under the collaboration, Solid received an exclusive license to develop and commercialize six undisclosed cardiac gene therapy programs.
Platform Technologies – Capsids & Other
Solid is building an innovative library of enabling technologies, across:
Capsids & Promoters
- The Company is building multiple cardiac and neuromuscular capsid and promoter libraries with final capsid selection from the first library anticipated in Q4 2025.
- AAV-SLB101, Solid’s proprietary capsid used in SGT-003, has been licensed to 15 academic labs and corporations.
Immunomodulation
- Multiple immunomodulatory preclinical studies are underway to determine viability of potential future dosing with AAV-SLB101 in AAV-gene therapy pre-treated and seropositive individuals.
CMC Purity
- Solid’s manufacturing constructs continue to establish manufacturing excellence with further improvements in full-to-empty capsid ratios seen at research scales in early-stage cardiac programs.
43rd Annual J.P. Morgan Healthcare Conference Webcast
Mr. Cumbo and Dr. Brooks will present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 3:45 p.m. PT (6:45 p.m. ET).
A live webcast of the presentation will be available on the Events page of the Investors section of the Company website or by clicking here. A webcast replay will be archived for 30 days on the Events page.
Institutional investors interested in meeting with management during the conference may reach out to their J.P. Morgan representatives.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-212 for the treatment of Friedreich’s ataxia, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy, SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the Company; the ability to successfully achieve and execute on the company’s goals, priorities and achieve key clinical milestones; the Company’s pipeline of programs for neuromuscular and cardiac diseases, including its SGT-003 and SGT-212 programs and expectations for CTA filings, site activations, clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the ability to successfully develop other preclinical programs and its capsid libraries; the sufficiency of the Company’s cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance SGT-003, SGT-212, SGT-501, SGT-601, SGT-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne, Friedreich’s ataxia and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-212, SGT-501, SGT-601, SGT-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
Solid Biosciences Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
________________
1 Unaudited
FAQ
When will Solid Biosciences (SLDB) report initial data from the INSPIRE DUCHENNE trial?
How much cash does SLDB have as of December 2024?
When will SLDB begin human trials for SGT-212 in Friedreich's ataxia?
What is the timeline for SLDB's SGT-501 IND submission for CPVT?
How many clinical sites are active for SLDB's INSPIRE DUCHENNE trial?
