CAMBRIDGE, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today reported financial results for the third quarter ended September 30, 2020 and provided a business update.

“With the FDA’s lifting of the clinical hold on the IGNITE DMD trial, we are well underway in completing the activities necessary to resume dosing, which we expect will occur in the first quarter of 2021,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences. “This important event and establishing a strategic collaboration with Ultragenyx to develop additional gene therapies for Duchenne mark important progress toward our goal of improving the lives of patients living with Duchenne. We are also increasing production of SGT-001 using our improved manufacturing process in support of dosing additional patients in 2021. Additionally, we strengthened our balance sheet with additional capital from the Ultragenyx collaboration and our recent at-the-market, or ATM, equity financing, both of which will support our planned clinical advancement of SGT-001.”

Recent Developments

• In October 2020, Solid announced that the U.S. Food and Drug Administration (FDA) lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. Solid expects to resume dosing in the clinical trial in the first quarter of 2021.
  • Solid implemented and shared with the FDA manufacturing process changes that remove the majority of empty viral capsids. The improved process is averaging approximately 90% full capsids, allowing target dosing to be achieved with fewer viral particles.
  • Solid submitted data from a new, quantitative, in vitro microdystrophin expression assay that demonstrates comparability between SGT-001 manufactured by the two processes.
  • Solid is reducing the maximum weight of the next two patients dosed to 18 kg. This reduction, in conjunction with the delivery of fewer viral particles as a result of the company’s manufacturing process improvements, will reduce patients’ total viral load while continuing dosing at the 2E14 vg/kg dose.
  • Solid has amended the IGNITE DMD clinical protocol to include the prophylactic use of both anti-complement inhibitor eculizumab and C1 esterase inhibitor, and an increase in prednisone dose in the first month post dosing.
  • Solid also provided the FDA with updated functional efficacy data (including 6-Minute Walk Test and North Star Ambulatory Assessment data) for all patients dosed to date in IGNITE DMD.
• In October 2020, Solid and Ultragenyx Pharmaceutical (Ultragenyx) announced a strategic collaboration to develop and commercialize new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform for adeno-associated virus (AAV) vectors using AAV8 variants. The goal of the collaboration is to expand the pipeline of potential gene therapies for patients living with Duchenne.
  • Ultragenyx made a $40 million investment in Solid at a 33% premium.
  • Ultragenyx has also agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.
  • Solid retains full rights to SGT-001 as well as the opportunity to establish additional partnerships around SGT-001 or the Company’s proprietary and differentiated microdystrophin construct outside of AAV8 variants.
• In October 2020, Solid announced that it sold shares of its common stock pursuant to a sales agreement dated March 13, 2019, between the Company and Jefferies LLC that resulted in gross proceeds of $23.9 million (ATM Sale).

Financial Highlights

Research and development expenses for the third quarter of 2020 were $16.0 million, compared to $22.8 million for the third quarter of 2019. Research and development expenses for the first nine months of 2020 were $49.2 million, compared to $67.7 million for the first nine months of 2019. The decrease was primarily attributable to a reduction in personnel and facility related expenses as a result of the restructuring that occurred in January 2020, as well as lower manufacturing costs and a decrease in costs related to other product candidates as the Company focuses on advancing SGT-001.

General and administrative expenses for the third quarter of 2020 were $5.2 million, compared to $6.9 million for the third quarter of 2019. General and administrative expenses for the first nine months of 2020 were $16.0 million, compared to $19.3 million for the first nine months of 2019. The decrease was primarily attributable to decreased personnel costs and corporate expenses partially due to the restructuring that occurred in January 2020.

Net loss for the third quarter of 2020 was $21.2 million, compared to $29.3 million for the third quarter of 2019. Net loss for the first nine months of 2020 was $66.9 million, compared to $85.4 million for the first nine months of 2019.

Solid had $24.8 million in cash and cash equivalents as of September 30, 2020. The Company expects that its cash and cash equivalents, combined with proceeds of $40 million from the issuance and sale of shares of common stock to Ultragenyx and the net proceeds of $23.2 million from the ATM Sale will enable Solid to fund its operating expenses into the second half of 2021.

About SGT-001

Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase nNOS. Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

About Solid Biosciences
Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive – work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001. For more information, please visit www.solidbio.com.