Solid Biosciences Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update
– Biopsy results from the third patient dosed at 2E14 vg/kg in the SGT-001 IGNITE DMD clinical trial provide further support for continued development –
– Solid continues to make progress to address the IGNITE DMD clinical hold and advance the next steps for the SGT-001 program –
- Today, Solid announced biomarker data from the third patient dosed in the 2E14 vg/kg dose cohort of IGNITE DMD, the company’s Phase I/II clinical trial of SGT-001, including three-month biopsy data. Using immunofluorescence assays, 50%-70% of the muscle fibers were determined to express SGT-001 microdystrophin. Immunofluorescence also showed stabilization and co-localization of nNOS and beta-sarcoglycan with SGT-001 microdystrophin. Inclusion of the dystrophin nNOS coding region in SGT-001 may result in unique activity, potentially providing important functional benefits such as diminished muscle fatigue and protection against ischemic muscle damage. Using western blot, expression was 8% of normal control samples. The levels of serum creatine kinase, a highly variable biochemical marker of muscle damage, declined from baseline.
January 2020, Solid announced changes to its organizational structure to create a leaner company focused on advancing SGT-001. The corporate changes implemented reduce the company’s planned corporate expenses and extend the expected cash runway.
December 2019, Solid announced biomarker data from two patients dosed in the 2E14 vg/kg dose cohort of IGNITE DMD. The data showed expression of SGT-001 microdystrophin and nNOS function that provides evidence SGT-001 could provide therapeutic benefit for patients with Duchenne.
November 2019, Solid reported that the U.S. Food and Drug Administration(FDA) placed IGNITE DMD on clinical hold following a serious adverse event in the sixth patient dosed. In December 2019, the company announced that the adverse event had fully resolved, and that the patient had resumed his normal activities. For all patients dosed in IGNITE DMD, any clinical or laboratory abnormalities observed following SGT-001 administration have fully resolved.
Research and development expenses for the fourth quarter of 2019 were
General and administrative expenses for the fourth quarter of 2019 were
Net loss for the fourth quarter of 2019 was
Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy under investigation for its ability to address the underlying genetic cause of Duchenne muscular dystrophy (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.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our expectations regarding the IGNITE DMD clinical trial, the safety or potential efficacy of SGT-001, the sufficiency of our cash, cash equivalents and investments to fund our operations and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” 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 Solid’s ability to satisfactorily respond to requests from the FDA for further information and data regarding IGNITE DMD; successfully resolve the clinical hold with regard to IGNITE DMD; obtain and maintain necessary approvals from the FDA and other regulatory authorities and investigational review boards at clinical trial sites; enroll patients in its clinical trials; continue to advance SGT-001 in clinical trials; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully 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 to achieve its business objectives. 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
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|Consolidated Statements of Operations|
|(unaudited, in thousands, except share and per share data)|
|Research and development||94,737||57,965||39,905|
|General and administrative||24,581||17,722||14,952|
|Total operating expenses||119,318||75,687||54,857|
|Loss from operations||(119,318||)||(75,687||)||(54,857||)|
|Other income (expense):|
|Revaluation of preferred unit tranche right||-||-||459|
|Total other income (expense), net||2,095||889||1,679|
|Net loss attributable to non-controlling interest||-||-||(1,060||)|
|Net loss attributable to
|Accretion of preferred units to redemption value||-||-||(959||)|
|Redemption of preferred units||-||-||15,685|
|Redemption of redeemable interest from non-controlling interest in Solid GT||-||-||(1,925||)|
|Net loss attributable to common stockholders||$||(117,223||)||$||(74,798||)||$||(39,317||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(2.91||)||$||(2.25||)||$||(2.88||)|
|Weighted average shares of common stock outstanding, basic and diluted||40,289,290||33,262,597||13,649,485|
|Consolidated Balance Sheets|
|(unaudited, in thousands, except share and per share data)|
|Cash and cash equivalents||$||76,043||$||86,366|
|Prepaid expenses and other current assets||2,778||6,175|
|Total current assets||86,302||128,639|
|Operating lease, right of use assets||4,988||-|
|Property and equipment, net||11,645||10,422|
|Other non-current assets||209||209|
|Liabilities and Stockholders' Equity|
|Operating lease liabilities||1,736||-|
|Finance lease liabilities||186||173|
|Other current liabilities||52||382|
|Total current liabilities||18,276||12,481|
|Operating lease liabilities, excluding current portion||4,414||-|
|Finance lease liabilities, excluding current portion||733||859|
|Other non-current liabilities||-||1,074|
|Additional paid-in capital||396,278||324,209|
|Accumulated other comprehensive gain (loss)||1||(5||)|
|Total stockholders' equity||80,048||125,183|
|Total liabilities and stockholders' equity||$||103,471||$||139,597|
Source: Solid Biosciences Inc.