Fierce Biotech: Roivant and Plexcera Launch Rare Disease Focused Spinout

July 22, 2016

Roivant Sciences and Plexcera Therapeutics have today launched a new biotech company with a singular focus on the ultrarare condition known as Farber disease, as Roivant continues with its spinoff strategy.

The new company, which is to be called Enzyvant Sciences, is currently finishing preclinical studies in recombinant human acid ceramidase (rhAC)–an enzyme replacement therapy for patients with Farber disease.

Farber disease is a very rare inherited metabolic disorder that happens as the result of either the lack or malfunction of a particular enzyme needed to break down complex chemical compounds in the structures within cells known as lysosomes.

The condition usually has three symptoms: painful and progressively deformed joints, nodules under the skin and progressive hoarseness. Current treatments are fairly limited, with corticosteroid drugs typically being used.

Enzyvant, however, is hoping to use rhAC to help combat this disease using the tech which was originally developed in the lab of Professor Edward Schuchman at the Icahn School of Medicine at Mount Sinai. In a statement, the biotechs said these showed “promising results” in a mouse model of Farber disease, and was in fact initially licensed to Plexcera before the creation of Enzyvant.

The goal is to now bring this into the clinic, which will get help from Plexcera–which itself has only been around since 2013, also formed with the goal of helping create a new rhAC–while also tapping Roivant for its development and financial support.

Roivant has been around a bit longer, working on a number of therapeutic areas such as rare diseases, oncology and neurology–and has a history of tie-ups, having formed partnerships with Takeda, Eisai and GlaxoSmithKline ($GSK), to name a few.

It’s also not new to the spinout game, having successfully launched Axovant Sciences ($AXON) (formerly Roivant Neurosciences) a few years ago, and helped with its huge $315 million IPO, masterminded by the company’s young former hedge fund manager Vivek Ramaswamy. Another spinout, called Myovant and created in conjunction with Takeda, is also in the mix for the company.

Enzyme replacement therapies have for a few years now been used on a number of other rare diseases, including Sanofi’s ($SNY) Fabry disease drug Fabrazyme (originally developed by its Genzyme unit) and Cerezyme for Gaucher disease.

“I am very excited about the future of rhAC. Similar to other enzyme replacement therapies, rhAC has the potential to be a transformative therapy for patients afflicted with Farber disease, a population with clear unmet medical needs,” said Professor Schuchman, who will continue to work on the tech.

– check out the release


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