— Obtained rare pediatric disease and orphan drug designation for Duchenne myopathy
Administration of dystrophy (Duchenne) gene therapy candidate SGT-003 to patients in phase 1/2 study
Expected in Q2 2024 —
— The Company has entered into a non-exclusive license agreement for the use of proprietary muscle.
Targeted AAV-SLB101 capsid —
— The company has approximately $206.1 million in cash and investments through the end of the first quarter of 2024.
Solids forecasts cash runway through 2026 —
CHARLESTOWN, Mass., May 15, 2024 (Globe Newswire) — Solid Biosciences, Inc. (NASDAQ: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, announced today that reported financial results for the first quarter ended March 31. , 2024, provided business updates.
“In the first quarter of 2024, we continue to focus on execution across our pipeline and remain on track to begin dosing patients in the SGT-003 study in the second quarter of 2024. “We expect to provide top-line information on safety and microdystrophin expression with functional benefit by the end of the year,” said Bo Kunbo, President and CEO of Solid Biosciences. “Our strong cash position of $206.1 million, supported by the $108.9 million financing completed in January 2024, is supported by our anticipated runway by 2026 and the SGT for Duchenne muscular dystrophy. We will provide both the resources to execute across our portfolio, including the clinical implementation of -003, advancing SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT) toward an IND filing, neuromuscular and cardiac We continue to advance the remainder of our diverse pipeline of gene therapy candidates.”
Company latest information
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Solid plans to begin a Phase 1/2 study of SGT-003 in pediatric patients with Duchenne disease in the second quarter of 2024. Initial safety, microdystrophin expression and functional benefit data from the first three to four patients enrolled in the trial are expected by the fourth quarter of 2024.
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Solid plans to submit an investigational new drug application (IND) for SGT-501, its first cardiac gene therapy candidate for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), in the first quarter of 2025 .
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Solid continues to advance the rest of its therapeutic portfolio with preclinical studies with BAG3, mouse studies with TNNT2, and preclinical studies with RBM20. Platforms that enable work in both capsid (NHP, mouse and pig studies) and his CMC process development work across the portfolio is ongoing.
Recent company highlights
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On April 1, 2024, Solid announced that the FDA has granted rare pediatric disease designation to its Duchenne gene therapy candidate SGT-003. This designation encourages the development of new drugs for the prevention and treatment of rare childhood diseases. If the new Biologics License Application (BLA) for SGT-003 is approved, we may be eligible to receive a priority review voucher. This voucher may be redeemed, sold or transferred for priority consideration of a subsequent marketing application for another product.
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On March 7, 2024, Solid signed a non-exclusive license agreement for the use of its proprietary muscle-targeted AAV-SLB101 capsid in the development of RNAi therapies to treat FSHD (facioscapulohumeral muscular dystrophy). It was announced that the agreement had been concluded. The Company aims to broadly license AAV-SLB101 to both companies and academic institutions pursuing treatments for rare diseases.
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On January 16, 2024, Solid announced that the FDA granted orphan drug designation to SGT-003. This designation provides certain benefits, including certain financial incentives to support clinical development, and could result in up to seven years of market exclusivity in the United States, subject to regulatory approval.
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On January 8, 2024, Solid announced a $108.9 million private placement with new and existing investors. The Company intends to use the net proceeds of the Private Placement, approximately $103.7 million, to fund its ongoing pipeline development programs, business development activities, working capital and other general corporate purposes.
Financial Highlights for Q1 2024
There was no collaboration revenue in the first quarter of 2024 or 2023.
Research and development expenses for the three months ended March 31, 2024 were $18.9 million and research and development expenses for the three months ended March 31, 2023 were $24.6 million. The $5.8 million decrease in research and development expenses was primarily due to a $7.2 million decrease. SGT-003 manufacturing and research-related costs increased and SGT-001 costs decreased by $2.3 million, primarily related to the decision to prioritize SGT-003, and SGT-501 manufacturing and partially offset by an increase in research-related costs of $3.8 million.
General and administrative expenses for the three months ended March 31, 2024 were $8.0 million and general and administrative expenses for the three months ended March 31, 2023 were $7.4 million. The $600,000 increase primarily relates to a $400,000 increase in attorney fees. Corporate and business development costs increased by $200,000.
The net loss for the first quarter of 2024 was $24.3 million and the net loss for the first quarter of 2023 was $30.1 million. The decrease in net loss was due to lower research and development expenses and higher yields on cash, cash equivalents and available assets. – Securities for sale.
Solids’ cash, cash equivalents and other securities were $206.1 million at March 31, 2024, compared to $123.6 million at December 31, 2023. . The sale of securities as of March 31, 2024 will enable us to fund key strategic priorities through 2026.
About solid state bioscience
Solid Biosciences is developing gene therapies such as SGT-003 for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), and AVB-401 for the treatment of cancer drugs. A life sciences company focused on advancing its portfolio of candidates. Additional assets for the treatment of BAG3-mediated dilated cardiomyopathy, and for the treatment of fatal heart disease. Solid brings together experts in science, technology, disease management and care to advance a diverse pipeline across rare neuromuscular and cardiac diseases. Patient-focused and founded by those directly affected, Solid’s mission is to improve the daily lives of patients living with these devastating diseases. Learn more about. 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 the Company’s future expectations, plans and prospects. Ability to successfully achieve and execute on company priorities and achieve key clinical milestones. The company’s SGT-003 program includes expectations for initiation of dosing and availability of clinical trial data. Company expectations for his IND submission for SGT-501. Solid plans to license AAV-SLB101 broadly to both businesses and academic institutions. Planned use of proceeds from private placement in January 2024. Potential Benefits of Pediatric Rare Disease Designation and Orphan Drug Designation. the Company’s financing options and the sufficiency of the Company’s cash, cash equivalents and available-for-sale securities to fund its operations; and “anticipate,” “believe,” “continue,” “may,” “estimate,” “expect,” “intend,” “may,” “plan,” and “possible.” Other statements containing words such as “probable” or “predict”. Expressions such as “project”, “should”, “target”, “will”, “working on”, etc. Forward-looking statements are based on management’s current expectations about future events, which could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. is subject to a number of risks and uncertainties. These risks and uncertainties include, but are not limited to, risks related to Solid’s ability to recognize the anticipated benefits of his acquisition of AavantiBio; the Company’s ability to advance SGT-003, SGT-501, AVB-401 and other preclinical programs and capsid libraries as expected or at all; obtaining and maintaining necessary approvals from the FDA and other regulatory authorities; Replicating the positive results found in preclinical studies of the company’s product candidates in clinical trials. obtain, maintain or protect intellectual property rights related to our product candidates; compete successfully with other companies seeking to develop Duchenne therapy and other neuromuscular and cardiac treatments and gene therapies; Manage expenses. and the schedule that requires significant additional capital to continue the development of SGT-003, SGT-501, AVB-401, and our other candidates, achieve our other business objectives, and continue as a going concern. I will procure it. Please refer to the “Risk Factors” section and the description of potential risks for a discussion of other risks and uncertainties and other important factors that could cause the Company’s actual results to differ from those contained in the forward-looking statements. please refer to. , uncertainties and other important factors are described in the company’s most recent filings with the Securities and Exchange Commission. Additionally, the forward-looking statements contained in this press release represent our views as of the date of this press release and should not be relied upon as representing our views as of any date subsequent to this press release. there is no. We expect subsequent events and developments to cause our views to change. However, although we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so.
Solid Bioscience Investor Contact:
investor@solidbio.com
Media contact:
glen silver
FINN Partner
glenn.silver@finnpartners.com
