Two Rare Disease Drug Developers Raise Combined $416 Million in IPOs

Rare Daily Staff

Rare disease drug developers Design Therapeutics and Edgewise Therapeutics completed initial public offerings that raised a combined $416 million to advance their programs.

Design Therapeutics, a preclinical biotech developing a platform of gene targeted chimera (GeneTAC) small molecules for the treatment of serious degenerative disorders caused by inherited nucleotide repeat expansions, went public one year after launching. The company priced 12 million shares of its common stock at $20 per share to raise $240 million less underwriting discounts and commissions. In addition, Design has granted the underwriters a 30-day option to purchase up to an additional 1.8 million shares of its common stock at the IPO price. Shares will trade on the Nasdaq Global Select Market under the ticker symbol “DSGN.”

The funds will be used to advance lead therapeutic candidate into clinical development for the treatment of Friedreich’s ataxia, and support discovery programs for multiple other degenerative diseases, including fragile X syndrome and myotonic dystrophy.

Nucleotide repeat disorders are a set of genetic disorders caused by repeat expansion in certain genes. Depending on where it is located, the repeat may cause a change in the regulation of gene expression or produce a toxic gene product, all of which are recognized as significant contributors to degenerative diseases.

Friedreich’s ataxia, affects the nervous system resulting in progressive and life-altering movement problems and shortened life expectancy. Patients with Friedreich’s ataxia have an expanded GAA repeat in the first intron of the FXN gene, which blocks transcription and limits production of the frataxin protein. Design’s technology unblocks transcription, thereby restoring the natural production and function of frataxin.

Edgewise Therapeutics, which is developing orally bioavailable, small molecule therapies for rare muscle disorders, raised $176 million through an initial public offering of 11 million shares of its common stock at $16.00 per share. In addition, Edgewise has granted the underwriters a 30-day option to purchase up to 1.65 million additional shares of its common stock at the IPO price less underwriting discounts and commissions.

The shares will trade on the Nasdaq Global Select Market under the ticker symbol “EWTX.”

Proceeds from the IPO will be used to advance clinical development of Edgewise’s lead drug candidate, EDG-5506, for Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), as well as the company’s other rare disease programs.

Edgewise aims to modulate processes in skeletal muscle by create therapeutic agents that will reduce muscle damage, normalize muscle function, decrease mortality and profoundly benefit patients’ quality of life. The company’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue, initially focused on addressing rare neuromuscular and cardiac diseases.

Lead candidate EDG-5506, currently in phase 1 studies, is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD by selectively limiting injurious hyper-contraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It can be used as a single agent therapy but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development.

These two IPOs bring the total raised in 2021 by eight rare disease therapeutics IPOs to $1.6 billion.