Solid Biosciences Provides Third Quarter 2021 Business Update and Financial Results
- Continued focus on advancing SGT-001 includes additional patient dosings in IGNITE DMD expected to begin in Q4 2021 -
- Continued progress with SGT-003; Solid’s next-generation Duchenne gene therapy program demonstrates enhanced muscle tropism and microdystrophin expression -
- Company ends Q3 with approximately
“Solid made progress across all its strategic priorities during the third quarter. We expect to dose additional patients in IGNITE DMD beginning in the fourth quarter of 2021. We also shared meaningful clinical data across a variety of endpoints from the ongoing IGNITE DMD clinical trial of SGT-001, which continue to guide our understanding of the potentially differentiated benefit that SGT-001 may offer to patients. In addition, we advanced our Duchenne pipeline program, SGT-003, including selecting a manufacturing approach that will allow us to quickly move into human proof of concept in early 2023,” said
Solid expects to dose additional patients in IGNITE DMD, beginning in the fourth quarter of 2021. In addition, the company expects to report 90-day biopsy data from the first three patients dosed with SGT-001 manufactured with its improved process as well as long-term expression and functional data in early 2022. Concurrent with activities to support continued patient dosing in IGNITE DMD, Solid is also engaged in a variety of activities to support continued advancement of SGT-001, including scaling its improved manufacturing process.
R&D Pipeline Update
SGT-003, Solid’s next-generation adeno-associated virus (AAV) capsid pipeline program, continued its progress during the third quarter and continues to demonstrate enhanced muscle tropism and microdystrophin expression in preclinical studies. Biodistribution data show increased vector genomes in muscle and heart as well as decreased vector genomes in liver with SGT-003 compared to AAV9 in various in vitro and in vivo models. This biodistribution profile has the potential to increase efficiency and specifically target muscle cells, which could potentially allow for a reduced total viral load.
As announced last month, Solid has entered into an agreement with Forge Biologics to advance the development and manufacturing of SGT-003 using a transient transfection process. Solid is working closely with Forge to advance manufacturing of SGT-003 and is looking toward a target IND filing in early 2023.
Solid also continued its progress with Ultragenyx on the companies’ collaboration that leverages Solid’s proprietary nNOS-containing microdystrophin construct with an AAV8-like capsid within Ultragenyx’s HeLa producer cell line manufacturing approach. A program update is expected before the end of 2021.
Recent Company Developments
Solid, in collaboration with REGENXBIO formally launched the
Collaboration revenue for the third quarter of 2021 was
Research and development expenses for the third quarter of 2021 were
General and administrative expenses for the third quarter of 2021 were
Net loss for the third quarter of 2021 was
Solid had approximately
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 clinical 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.
SGT-003, Solid's next-generation gene therapy candidate for the treatment of Duchenne, utilizes a rationally designed AAV-based vector to deliver the proprietary and differentiated microdystrophin construct that is also incorporated into SGT-001. SGT-003 has demonstrated improved biodistribution compared with AAV9 in various in vitro and in vivo models, with increased delivery to and expression in skeletal and heart muscle and reduced tropism for liver cells. Solid is targeting an IND filing in early 2023.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the ability of the Company to continue dosing patients in the IGNITE DMD trial, the Company’s plans to represent data from IGNITE DMD, the implication of interim clinical data, the safety or potential treatment benefits of SGT-001 in patients with DMD, the Company’s regulatory plans, the Company’s SGT-003 program, including the Company’s expectation for filing an IND, timelines, 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 continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approvals from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll additional patients in IGNITE DMD and on the timeline expected; the Company’s dosing strategy; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; whether the interim data referenced in this release will be predicative of the final results of the trial or will demonstrate a safe or effective treatment benefit of SGT-001; whether the methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters will yield meaningful statistical results; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, SGT-003 and other product 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
Source: Solid Biosciences Inc.