Solid Biosciences (reserved Nasdaq symbol: SLDB) set terms for its proposed initial public offering in its amended S-1 filing today. The company, which is developing a gene therapy treatment for Duchenne muscular dystrophy (DMD), set a range of $16 to $18 for its IPO of 5,890,000 shares, or a raise of about $100 million at the midpoint – a post-money valuation of about $548 million before the green shoe. This relatively small transaction has attracted a high profile roster of banks, led by JP Morgan, Goldman and Leerink. The deal is expected to price next week, according to Renaissance Capital.
DMD is a genetic muscle-wasting disease predominantly affecting boys, with symptoms that usually manifest between three and five years of age. DMD is a progressive, irreversible and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 10,000 to 15,000 cases in the US. DMD is caused by mutations in the dystrophin gene, which result in the absence or near-absence of dystrophin protein, which normally works to strengthen muscle fibers and protect them from daily wear and tear. Without functioning dystrophin and certain associated proteins, muscles suffer excessive damage from normal daily activities and are unable to regenerate, leading to the build-up of fibrotic and fat tissue.
The company’s lead product candidate, SGT-001, is a gene therapy that is being developed to restore functional dystrophin protein expression. SGT-001 has been granted Rare Pediatric Disease Designation in the US and Orphan Drug Designations in both the US and EU. SGT-001 is currently being evaluated in a Phase I/II clinical trial.
Pipeline
Source: Solid Biosciences
SGT-001 is a gene transfer candidate designed to address the underlying genetic cause of DMD by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. The transgene is delivered via an adeno-associated virus (AAV) vector, which also contains a muscle-specific promoter. Our vector is a modified version of an AAV, a naturally occurring, non-pathogenic virus selected for its ability to efficiently enter skeletal, diaphragm and cardiac muscle tissues. The vector will carry a synthetic dystrophin transgene construct, called microdystrophin, that retains the most critical components of the full-size dystrophin gene yet is small enough to fit within AAV packaging constraints.
The current Phase I/II clinical study, called IGNITE DMD, is designed to evaluate SGT-001 in ambulatory and non-ambulatory 4 to 17-year-old males with DMD. tient reported outcomes and quality of life measures, among other endpoints. The study will enroll approximately 16 to 32 patients with DMD, who will be randomly assigned to either an active treatment group or a delayed-treatment control group. Initially, adolescents aged 12 to 17 years will receive treatment, and at a later stage of the study, children aged four to 11 years will be dosed. Efficacy will be assessed by comparing microdystrophin protein expression in muscle biopsy before treatment and 12 months after treatment for each patient. Participants in the control group who continue to meet inclusion criteria and not meet exclusion criteria will receive active treatment after 12 months.
“Solid” is the English translation of Eytani, the Hebrew name of Ilan and Annie Ganot’s son, who was diagnosed with the disease in 2012. Mr. Ganot, who co-founded Solid Biosciences in 2013, was formerly a banker at JP Morgan, Nomura and Lehman. In 2015, the company began exclusively licensing the elements of the construct for SGT-001 and other elements of its program from the University of Michigan, the University of Missouri and the University of Washington.
The company has previously raised private capital from Bain Capital Life Sciences, Biogen, JP Morgan (via JPMC Strategic Investments II), Perceptive Advisors and RA Capital, along with several additional corporate and private investors. In addition, three leading U.K.-based DMD charities provided initial seed equity funding for the company’s gene transfer program in return for equity in our company.
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