Solid Biosciences Reports Fourth Quarter and Fiscal Year 2018 Financial Results and Provides Business Update
“In our first year as a public company, we have significantly advanced our innovative science for Duchenne muscular dystrophy, made meaningful progress towards commercial scale manufacturing and continued to add talent and capabilities to our team to prepare to bring differentiated therapies to patients,” said
“We are encouraged to continue advancing SGT-001 in the clinic,” said
- Today Solid announced that it has completed the necessary steps to escalate the dose of SGT-001 to 2E14 vg/kg in a second cohort of patients, which include agreement from the IGNITE DMD clinical trial Data Safety Monitoring Board (DSMB) and
University of Florida Institutional Review Board(IRB). Solid will continue to enroll children in the study, which is a randomized, controlled, open-label, single-ascending dose Phase I/II clinical trial to assess the safety and efficacy of SGT-001 (an investigational AAV-mediated microdystrophin gene transfer) for the treatment of Duchenne muscular dystrophy (DMD), and intends to resume dosing adolescents in the future. Solid anticipates providing additional data on IGNITE DMD later this year.
- In February, Solid announced preliminary findings based on three-month biopsy data from the first three patients dosed with 5E13 vg/kg of SGT-001, the initial dose outlined in the IGNITE DMD protocol. In one patient, microdystrophin was detected via western blot below the five percent level of quantification of the assay and in approximately 10 percent of fibers via immunofluorescence. There were also signs of co-localization of neuronal nitric oxide synthase (nNOS) and beta-sarcoglycan associated with microdystrophin expression. In the second and third patients, microdystrophin was detected via immunofluorescence at very low levels, but it was undetectable via western blot. The safety profile of SGT-001 remains unchanged.
- In February, Solid’s Scientific Advisory Board Chair Dr.
Jeffery Chamberlainand other researchers at the University of Washingtonpublished preclinical data highlighting the unique attributes of the SGT-001 transgene in the journal MolecularTherapy. The paper, called Development of Novel Micro-dystrophins With Enhanced Functionality, supports the potential of the SGT-001 transgene to protect against contraction-induced injury and recruit key proteins of the dystrophin glycoprotein complex, including nNOS.
- In January, Solid presented additional information about its scalable manufacturing process at the 37th Annual
J.P. Morgan Healthcare Conference, noting that the Company has successfully produced multiple batches at 250 liters in suspension, each of which can dose multiple patients, at good manufacturing practice-compliant facilities.
- In January, the Company appointed
Lynette Herschaas Chief Legal Officer. Ms. Herscha brings to Solid more than 20 years of legal experience in the technology and biopharma industry. Prior to joining Solid, she served as General Counsel, Secretary and a member of the Executive Team at Concert Pharmaceuticals, Inc.Before joining Concert Pharmaceuticals, Ms. Herscha held various senior legal positions at Momenta Pharmaceuticals, Inc.and Phase Forward, Inc.She began her career at the law offices of Fullbright & Jaworski.
Research and development expenses for the fourth quarter of 2018 were
General and administrative expenses for the fourth quarter of 2018 were
Net loss for the fourth quarter of 2018 was
Management is scheduled to present at the
A live video webcast will be available at http://arminvestorday.com/webcast/ and will also be published there shortly after the event.
Solid’s lead candidate, SGT-001, is a novel adeno-associated viral (AAV) vector-mediated gene transfer under investigation for its ability to address the underlying genetic cause of Duchenne muscular dystrophy (DMD), 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 transgene, 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). SGT-001 utilizes AAV9, which has an affinity for muscle and is currently being evaluated in multiple clinical programs in other indications. Data from Solid’s preclinical program suggest that SGT-001 has the potential to slow or stop the progression of DMD, 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, and the potential of SGT-001, the sufficiency of our cash, cash equivalents and investments to fund our operation 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 obtain and maintain necessary approvals from the
Consolidated Statements of Operations
(unaudited, in thousands, except share and per share data)
|Year Ended December 31,|
|Research and development||57,965||39,905||20,116|
|General and administrative||17,722||14,952||5,460|
|Total operating expenses||75,687||54,857||25,576|
|Loss from operations||(75,687||)||(54,857||)||(25,576||)|
|Other income (expense):|
|Revaluation of preferred unit tranche rights||-||459||1,163|
|Total other income (expense), net||889||1,679||1,803|
|Net loss attributable to non-controlling interest||-||(1,060||)||(2,234||)|
|Net loss attributable to Solid Biosciences Inc.||$||(74,798||)||$||(52,118||)||$||(21,539||)|
|Decretion (accretion) of preferred units to redemption value||-||(959||)||4,309|
|Redemption of preferred units||-||15,685||-|
|Redemption of redeemable interest from non-controlling interest in Solid GT||-||(1,925||)||-|
|Net loss attributable to common stockholders||$||(74,798||)||$||(39,317||)||$||(17,230||)|
|Net loss per share attributable to common stockholders, basic and diluted||$||(2.25||)||$||(2.88||)||$||(10.14||)|
|Weighted average shares of common stock outstanding, basic and diluted||33,262,597||13,649,485||1,698,904|
Consolidated Balance Sheets
(unaudited, in thousands, except share and per share data)
|Cash and cash equivalents||$||86,366||$||52,080|
|Prepaid expenses and other current assets||6,175||1,499|
|Total current assets||128,639||70,658|
|Property and equipment, net||10,422||2,429|
|Other non-current assets||209||-|
|Deferred offering costs||-||3,106|
|Liabilities, Preferred Units and Stockholders' / Members' Equity / (Deficit)|
|Current portion of capital lease obligations||173||-|
|Other current liabilities||382||233|
|Total current liabilities||12,481||11,271|
|Capital lease obligations, net of current portion||859||-|
|Other non-current liabilities||1,074||-|
|Series 2 Senior Preferred Units||-||55,002|
|Series 1 Senior Preferred Units||-||25,000|
|Junior Preferred Units||-||44,177|
|Stockholders' / Members' Equity / (Deficit)|
|Series A, B, C and D Common Units||-||65,014|
|Additional paid-in capital||324,209||-|
|Accumulated other comprehensive loss||(5||)||(13||)|
|Total stockholders' / members' equity (deficit)||125,183||(59,257||)|
|Total liabilities, preferred units and stockholders' / members' equity||$||139,597||$||76,193|
Source: Solid Biosciences Inc.