Company Finalizing Response to FDA Regarding Clinical Hold on SGT-001 Phase I/II Clinical Trial.
Solid Biosciences Inc. (NASDAQ:SLDB) today reported financial results for the first quarter ended March 31, 2018 and provided a business update.
“The first quarter of 2018 marked Solid’s entry into public markets with the completion of our initial public offering, which we believe helps position us to achieve our mission to provide meaningful new therapies to patients with Duchenne muscular dystrophy,” said Ilan Ganot, Chief Executive Officer of Solid Biosciences. “We are pleased with the resolution of the manufacturing-related partial clinical hold on the high dose of our lead gene transfer candidate, SGT-001, in our Phase I/II clinical trial. Working with the FDA to resolve the full clinical hold on the clinical trial is currently our top priority.”
Recent Developments
- Completed its initial public offering, in which the Company issued and sold 8,984,375 shares of common stock, including 1,171,875 shares of common stock pursuant to the underwriters’ overallotment, at a public offering price of $16.00 per share. The Company received total net proceeds of approximately $129.1 million from the offering after discounts, commissions and offering expenses.
- Announced the receipt of a full clinical hold letter from the U.S. Food and Drug Administration (FDA) relating to IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational gene therapy, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). The clinical hold is in response to an unexpected Serious Adverse Event (SAE) reported in the first patient dosed with SGT-001. In its clinical hold letter, the FDA requested additional information, including an assessment of the underlying etiology of the event, the patient’s clinical status and laboratory parameters, and any additional measures to address patient safety. Solid is finalizing its response to the FDA letter.
- Announced that the earlier partial clinical hold on IGNITE DMD related to manufacturing processes for the high dose of SGT-001 had been resolved. To lift the hold, Solid submitted additional data to the FDA demonstrating that its current manufacturing process and product attributes for SGT-001 could support the high-dose group, enabling the use of a single lot for dose administration and limiting the number of vials of product required to treat each patient.
- Continued to build the Company’s gene therapy pipeline through an expanded collaboration with Synpromics Ltd to further evaluate next generation promoters and an exclusive option agreement with Lonza to explore the use of a novel capsid library developed at Massachusetts Eye and Ear and for a potential license.
- Expanded its footprint in Cambridge, Massachusetts with the opening of its new corporate headquarters and 9,500 sq. ft. of custom-built research and process development laboratory space in Kendall Square.
Anticipated Milestones
- Work with the FDA to resolve the full clinical hold on IGNITE DMD and resume the clinical trial as soon as possible.
- Present two platform presentations and five posters at the 21st Annual Meeting of the American Association of Gene and Cell Therapy (ASGCT) in May in Chicago. These presentations include data from several studies that expand upon the preclinical safety and efficacy of SGT-001 for the treatment of DMD, as well as studies supporting the Company’s growing next generation gene therapy portfolio.
Financial Highlights
Solid Biosciences reported a net loss of $15.9 million for the first quarter of 2018 as compared to $13.9 million for the first quarter of 2017. The increase in net loss for the year was due to increased research and development expenses, as well as investments in the Company’s infrastructure.
Research and development expenses for the first quarter of 2018 were $11.9 million as compared to $8.7 million for the prior year period. The increase in research and development expenses was primarily driven by increased compensation, headcount and facility costs, as well as increased costs related to the clinical development and manufacturing activities for SGT-001, which were offset by a reduction in the preclinical costs associated with SGT-001.
General and administrative expenses were $4.0 million for the first quarter of 2018 as compared to $5.4 million for the prior year period. The decrease in general and administrative expenses was primarily due to a decrease in equity-based compensation offset by an increase in salary and benefit related costs due to the increase in employee related expenses, as well as an increase in other corporate costs.
Solid ended the first quarter of 2018 with $182.4 million in cash, cash equivalents and available-for-sale securities as compared to $69.1 million as of December 31, 2017. The increase was primarily the result of the completion of the Company’s initial public offering on January 30, 2018.
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