Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Solid Biosciences (SLDB) has received Fast Track designation from the FDA for SGT-212, its dual-route gene therapy candidate for Friedreich's ataxia (FA). SGT-212 is designed to deliver the full-length frataxin gene through both intradentate nucleus and intravenous infusions to address neurologic, cardiac, and systemic manifestations of FA.
The Fast Track status, granted to products treating serious conditions with unmet medical needs, will facilitate more frequent FDA interactions and potential priority review eligibility. The company received FDA IND clearance on January 7th, 2025, and plans to initiate a Phase 1b trial in the second half of 2025. This first-in-human, open-label, multicenter study will evaluate safety and tolerability in both ambulatory and non-ambulatory FA patients, with a five-year follow-up period.
Solid Biosciences (SLDB) ha ricevuto la designazione Fast Track dalla FDA per SGT-212, il suo candidato alla terapia genica a doppio percorso per l'atrofia di Friedreich (FA). SGT-212 è progettato per offrire il gene della frataxina a lunghezza completa tramite infusione sia intradentata che endovenosa per affrontare le manifestazioni neurologiche, cardiache e sistemiche della FA.
Lo stato Fast Track, concesso a prodotti che trattano condizioni gravi con bisogni medici insoddisfatti, faciliterà interazioni più frequenti con la FDA e la potenziale idoneità per una revisione prioritaria. L'azienda ha ricevuto l'approvazione IND dalla FDA il 7 gennaio 2025 e prevede di avviare uno studio di Fase 1b nella seconda metà del 2025. Questo studio multicentrico, in aperto e sui primi esseri umani, valuterà la sicurezza e la tollerabilità in pazienti FA sia ambulatoriali che non ambulatoriali, con un periodo di follow-up di cinque anni.
Solid Biosciences (SLDB) ha recibido la designación de Fast Track de la FDA para SGT-212, su candidato de terapia génica de doble vía para la ataxia de Friedreich (FA). SGT-212 está diseñado para entregar el gen de frataxina de longitud completa a través de infusiones tanto intradentarias como intravenosas para abordar las manifestaciones neurológicas, cardíacas y sistémicas de la FA.
El estado de Fast Track, otorgado a productos que tratan condiciones graves con necesidades médicas insatisfechas, facilitará interacciones más frecuentes con la FDA y la posible elegibilidad para revisión prioritaria. La compañía recibió la autorización IND de la FDA el 7 de enero de 2025 y planea iniciar un ensayo de Fase 1b en la segunda mitad de 2025. Este estudio en humanos, abierto y multicéntrico, evaluará la seguridad y tolerabilidad en pacientes FA ambulatorios y no ambulatorios, con un período de seguimiento de cinco años.
Solid Biosciences (SLDB)는 SGT-212에 대해 FDA로부터 패스트트랙 지정을 받았습니다. SGT-212는 프리드리히 운동실조증(FA)을 위한 이중 경로 유전자 치료 후보로, FA의 신경학적, 심장 및 전신 증상을 해결하기 위해 내시경핵 및 정맥 주사를 통해 전체 길이의 프라탁신 유전자를 전달하도록 설계되었습니다.
심각한 조건의 치료로 unmet medical needs가 있는 제품에 부여되는 패스트트랙 상태는 FDA와의 상호작용을 더 자주 하도록 하고, 우선 심사를 받을 수 있는 자격을 제공합니다. 이 회사는 2025년 1월 7일 FDA IND 승인을 받았으며, 2025년 하반기에 1b상 시험을 시작할 계획입니다. 이 연구는 첫 번째 인간 연구로, 개방형 다기관 연구로서, FA 환자의 이동 가능 여부에 관계없이 안전성과 내약성을 평가하며, 5년의 추적 관찰 기간이 있습니다.
Solid Biosciences (SLDB) a reçu la désignation Fast Track de la FDA pour SGT-212, son candidat à la thérapie génique à double voie pour l'ataxie de Friedreich (FA). SGT-212 est conçu pour livrer le gène de la frataxine de longueur complète via des infusions intradentaires et intraveineuses afin de traiter les manifestations neurologiques, cardiaques et systématiques de la FA.
Le statut Fast Track, accordé aux produits traitant des conditions graves aux besoins médicaux non satisfaits, facilitera des interactions plus fréquentes avec la FDA et une éventuelle éligibilité à une révision prioritaire. L'entreprise a reçu l'autorisation IND de la FDA le 7 janvier 2025 et prévoit de lancer un essai de Phase 1b dans la seconde moitié de 2025. Cette première étude humaine, ouverte et multicentrique, évaluera la sécurité et la tolérabilité chez les patients FA ambulatoires et non ambulatoires, avec une période de suivi de cinq ans.
Solid Biosciences (SLDB) hat von der FDA die Fast-Track-Bezeichnung für SGT-212 erhalten, sein Dual-Route-Gen Therapie-Kandidat für die Friedreich-Ataxie (FA). SGT-212 ist so konzipiert, dass es das vollständige Frataxin-Gen sowohl durch intradentate Injektionen als auch intravenöse Infusionen liefert, um neurologische, kardiologische und systemische Manifestationen der FA zu behandeln.
Der Fast-Track-Status, der Produkten gewährt wird, die schwerwiegende Erkrankungen mit unerfüllten medizinischen Bedürfnissen behandeln, wird häufigere Interaktionen mit der FDA fördern und eventuell eine bevorzugte Überprüfung ermöglichen. Das Unternehmen erhielt am 7. Januar 2025 die IND-Zulassung von der FDA und plant, in der zweiten Jahreshälfte 2025 eine Phase-1b-Studie zu initiieren. Diese erste klinische Studie am Menschen wird die Sicherheit und Verträglichkeit bei ambulanten und nicht-ambulanten FA-Patienten bewerten, mit einer fünfjährigen Nachbeobachtungszeit.
- Received FDA Fast Track designation for SGT-212
- Only dual-route gene therapy for FA with FDA IND clearance
- Potential eligibility for priority review
- Phase 1b trial initiation planned for H2 2025
- Early-stage development (Phase 1b) indicates long pathway to potential commercialization
- Extended five-year follow-up period required for trial participants
Insights
The FDA Fast Track designation for SGT-212 represents a significant regulatory milestone for Solid Biosciences' gene therapy program. The dual-route administration approach (IDN and IV) is technically innovative and addresses a important gap in current FA treatment options. The full-length frataxin gene delivery mechanism targets the root cause of the disease rather than just managing symptoms.
The regulatory pathway acceleration could potentially reduce development timelines by 2-3 years. Previous FA gene therapy programs have faced challenges in achieving sufficient protein expression across different tissue types - SGT-212's dual administration strategy may overcome this limitation. The planned Phase 1b trial's 5-year follow-up period indicates a robust safety monitoring approach, which is important for novel gene therapies.
For investors, this positions SLDB favorably in the $2.5 billion FA treatment market. Being the only dual-route gene therapy with both IND clearance and Fast Track status creates a significant competitive advantage. The H2 2025 trial initiation timeline allows for adequate preparation while maintaining momentum.
The therapeutic approach of SGT-212 is scientifically compelling. Friedreich's ataxia, affecting approximately 1 in 50,000 people, has been notoriously difficult to treat due to its multi-system impact. The dual administration strategy is particularly noteworthy - intradentate nucleus delivery targets central nervous system manifestations, while intravenous administration addresses cardiac and peripheral symptoms.
Clinical development risks are partially mitigated by the comprehensive approach to frataxin restoration. The inclusion of both ambulatory and non-ambulatory patients in the Phase 1b trial is strategically important, as it will provide data across disease stages. The five-year follow-up period will generate important long-term safety and efficacy data, essential for gene therapy approval.
In simple terms, think of this therapy as a two-pronged approach: one route directly targets the brain's control center for movement and balance, while the other spreads throughout the body via bloodstream to help heart and other affected organs. This comprehensive strategy could potentially stop disease progression at any stage - a game-changing prospect for FA patients.
- Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation -
CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company’s, AAV-based gene therapy candidate for the treatment of Friedreich’s ataxia (FA). SGT-212 will deliver the full-length frataxin gene via dual routes of administration incorporating intradentate nucleus (IDN) and intravenous (IV) infusions, designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA.
Fast Track designation is granted to products that are developed to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. This designation is intended to facilitate development and expedite review of qualifying drugs. SGT-212 will benefit from this designation by having more frequent interactions with the FDA and the potential to be eligible for priority review.
Bo Cumbo, President and CEO commented: “SGT-212 is the only FA therapy in development that is designed to address frataxin deficiency, the underlying cause of FA, and all manifestations of this devastating disease, and in doing so, hopefully halt the full spectrum of symptom progression regardless of where the patient is in their journey with this terrible disease. We believe Fast Track designation may enable us to more rapidly develop SGT-212 and bring hope to those living with FA who need and deserve better treatment options.”
FDA IND clearance for SGT-212 was announced January 7th, 2025. The planned Phase 1b trial will be a first-in-human, open-label, multicenter study to evaluate the safety and tolerability of contemporaneous systemic IV and bilateral IDN administration of SGT-212 in adult non-ambulatory and ambulatory patients with FA. Dosing is expected to initiate in the second half of 2025 and participants in the trial will be followed for five years after receiving SGT-212.
“We are grateful for the FDA’s recognition that the needs of the FA community remain underserved, and that SGT-212 has the potential to bring meaningful change to their lives,” said Jessie Hanrahan, Ph.D., Chief Regulatory Officer at Solid Biosciences. “We look forward to working closely with the Agency to discuss the most effective and expeditious development pathway for SGT-212 to pursue future marketing authorization.”
About Fast Track Designation
The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.
About SGT-212
SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (FXN) via a dual route of administration: intradentate nucleus (IDN) infusion, using an FDA-approved, stereotactic, precision MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic FXN levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. Targeted delivery to the dentate nuclei will be confirmed in real time via gadolinium, an MRI-enhancing contrast agent. Restoration of FXN levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address neurologic, cardiac and systemic manifestations of the disease.
About Friedreich’s Ataxia (FA)
FA is an inherited, life-threatening, degenerative multisystem disease caused by defects in the frataxin gene that disrupt production of the frataxin protein, a mitochondrial iron-binding protein involved in essential cellular processes, including energy production. FA is known to cause progressive nervous system damage, movement problems, and cardiac dysfunction, with cardiac complications identified as the primary cause of death. FA impacts approximately 5,000 people in the United States and 15,000 in Europe. There are currently no treatments that provide a cure or halt disease progression.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated dilated cardiomyopathy, and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare 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-212 and SGT-003 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 regulatory approval; the benefits and impact of Fast Track designation; 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-212, SGT-003, 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 and designations 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-212, SGT-003, 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 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
FAQ
What is the significance of FDA Fast Track designation for SLDB's SGT-212?
When will SLDB begin Phase 1b trials for SGT-212?
How is SLDB's SGT-212 different from other Friedreich's ataxia treatments?
What is the duration of SLDB's Phase 1b trial follow-up for SGT-212?
Which patient groups will be included in SLDB's SGT-212 Phase 1b trial?
