Solid Bio Adds Friedreich Ataxia to Pipeline With Aavanti Acquisition


Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.

Solid Biosciences has acquired AavantiBio, a company whose lead program, AVB-202, is a gene therapy candidate in preclinical studies for Friedreich’s ataxia.1

The combined company will now focus on advancing Solid’s preclinical gene therapy program SGT-003 for the potential treatment of Duchenne muscular dystrophy (DMD) as well as AVB-202 under the leadership of AavantiBio’s current chief executive officer (CEO), Bo Cumbo. Aavanti’sadditional programs in development include AVB-401 for BAG3-mediated dilated cardiomyopathy and undisclosed programs for other cardiac indications.

“I created Solid with my wife, Annie, and our co-founders nearly 10 years ago, to bring meaningful treatment options to patients and families who, like ours, live with the devastating consequences of DMD,” Ilan Ganot, president, chief executive officer and co-founder, Solid Biosciences, said in a statement.1 “This acquisition provides exciting opportunities to bring our potentially best-in-class Duchenne gene transfer candidate, SGT-003, to patients and to expand our portfolio with innovative gene therapies designed to address significant unmet need in additional, adjacent rare disease indications. Bo Cumbo will assume the role of president and CEO and is a seasoned biotech executive with extensive expertise in bringing products through development and commercialization. I look forward to helping Bo with the leadership transition following the acquisition, and I am grateful to the investors who continue to support Solid’s critical efforts.”

Solid Biosciences also announced that it has entered into a securities purchase agreement with investors for a $75 million private placement expected to close concurrently with the merger. With the funding, thecombined company is expected to have approximately $215 million to be able to fund it into 2025 and advance the gene therapy programs.

WATCH NOW: Craig M. McDonald, MD, on Developing Gene Therapy for Different Populations of DMD

“I am excited for the opportunity to lead Solid Biosciences and to help build a leading genetic medicine company with a focus on neuromuscular and cardiac rare diseases,” said Bo Cumbo, president and chief executive officer, AavantiBio, said in a statement.1 “The Solid team has done incredible work in advancing Duchenne gene therapy, and its commitment to the Duchenne community aligns with AavantiBio’s patient-centric mission of bringing new therapies that can positively improve the quality of life of rare disease patients and their families. I also look forward to advancing our library of next generation cardiac and skeletal muscle capsids to extend our opportunities to bring treatment options to more people in need.”

Solid previously announced in a company update that it would be pausing development on SGT-001, its previous lead candidate for DMD, in favor of SGT-003, its next-generation candidate.2 The company stated that it would be switching to a scaled transient transfection-based manufacturing process of SGT-001 and that its phase 1/2 IGNITE-DMD trial (NCT03368742) would continue dosing in mid-2023. Solid also plans to be able to submit an investigational new drug application for SGT-003 in mid-2023.

Prior to the reprioritization, Solid had presented positive 2-year data from the IGNITE-DMD study at the 2022 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, held March 13-16, in Nashville, Tennessee.3 These data, from the first 3 patients treated in the high-dose cohort (2E14vg/kg), were in line with previous data reported from the 12- and 18-month time points.

Participants exhibited improved motor function, with a mean 16-meter improvement (range, -12 to 39; mean difference from natural history, +100.6) in 6-Minute Walk Test from baseline and a –1.7 change (range, -3 to –1; mean difference from natural history, +4.3) in North Star Ambulatory Assessment from baseline. They also showed improvements from baseline differing from natural history in pulmonary function with a mean 9.2% improvement in forced vital capacity (range, -1.4 to 29.0; mean difference from natural history, +19.2) and a 6.5% improvement in peak expiratory flow (range, -5.8 to 14.8; mean difference from natural history, +16.5). Patient-reported outcomes also improved and the therapy was well-tolerated.

While Solid’s progress in the DMD space seems to have stalled for the time being, other competitors such as Sarepta, Dystrogen, and CRISPR Therapeutics, are racing ahead with their respective DMD programs in recent months. CRIPSR received IND clearance for its CRISPR therapeutic CRD-TMH-001 in August 2022, and in September, Dystrogen reported positive data on DT-DEC01 while Sarepta filed a biologic license application for SRP-9001 (delandistrogene moxeparvovec).

1. Solid Biosciences announces acquisition of AavantiBio and concurrent $75 million private placement. News release. Solid Biosciences. September 30, 2022.
2. Solid Biosciences provides second quarter 2022 business update and financial results. News release. Solid Biosciences. August 11, 2022.
3. Dreghici R, Redican S, Lawrence J, et al. Ignite DMD phase I/II study of SGT-001 microdystrophin gene therapy for DMD: 2-year outcomes update. Presented at MDA Clinical and Scientific Conference; March 13-16. Poster 46.
4. Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic. News release. Cure Rare Disease. August 10, 2022.
5. Sarepta Therapeutics submits biologics license application for SRP-9001 for the treatment of ambulant patients with Duchenne muscular dystrophy. News release. Sarepta Therapeutics. Septemebr 29, 2022.
6. Dystrogen Therapeutics investigational chimeric cell therapy DT-DEC01 for Duchenne muscular dystrophy demonstrates clinically significant functional and biomarker improvements. News release. Dystrogen Therapeutics, Corp. September 26, 2022.
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