RARE Daily

Applied Therapeutics Raises $100 Million as FDA Grants Priority Review to Galactosemia Treatment

February 28, 2024

Rare Daily Staff

As the U.S. Food and Drug Administration accepted its New Drug Application for govorestat for the treatment of classic galactosemia with Priority Review, Applied Therapeutics entered into a securities purchase agreement for a private placement of $100 million of equity.

The company is selling 12.3 million shares of its common stock at a purchase price of $7.00 per share and 2 million pre-funded warrants to purchase common stock at $6.999, which is equal to the purchase price per share of common stock less the $0.001 per share exercise price of each pre-funded warrant, in a private placement for gross proceeds of $100 million, before deducting placement agent commissions and other offering expenses.

Proceeds will be used to fund commercial activities for govorestat and to further develop other pipeline candidates, and for working capital and general corporate purposes.

Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency in women. There are approximately 3,000 patients with galactosemia in the U.S. and approximately 4,000 patients with galactosemia in the EU. Newborn screening for galactosemia is mandatory in the U.S. and most EU countries, leading to rapid identification of affected patients.

Govorestat is an investigational, novel aldose reductase inhibitor (ARI) being developed for the treatment of several rare diseases. It is a potent and selective compound, which crosses the blood brain barrier into the central nervous system. The NDA filing of govorestat is supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile. The submission package included clinical outcomes data from the phase 3 registrational ACTION-Galactosemia Kids study in children aged 2-17 with galactosemia, the phase 1/2 ACTION-Galactosemia study in adult patients with galactosemia, and preclinical data.

“The FDA’s acceptance of the NDA for govorestat for the treatment of galactosemia represents a critical milestone for Applied Therapeutics and more importantly, for patients with galactosemia and their families. The Agency’s decision to grant Priority Review for this NDA underscores the urgent unmet medical need as there are currently no treatment options for this devastating disease,” said Shoshana Shendelman, founder and CEO of Applied Therapeutics.

If approved, govorestat would be the first medication indicated for the treatment of galactosemia and would be Applied Therapeutics’ first commercial product. The FDA grants Priority Review to applications for potential medicines that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition. Govorestat for the treatment of galactosemia has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) and Orphan Drug, Pediatric Rare Disease, and Fast Track designations from the FDA. The company has also submitted a Marketing Authorization Application for govorestat for the treatment of classic galactosemia to the EMA, which was validated in December 2023 and is under review by the EMA’s Committee for Medicinal Products for Human Use with a decision by the EMA expected in the fourth quarter of 2024.

The FDA is expected to act on the application for approval of Govorestat by August 28, 2024. The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application. Govorestat will qualify for a Priority Review Voucher upon approval.

Photo: Shoshana Shendelman, founder and CEO of Applied Therapeutics

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