Vertex Founder Praised ‘Perfect Drug’ Candidate Sold for $1.2 Billion

A biotech startup developing a drug for an inherited eye disease has been sold in a deal worth approximately $1.2 billion.

Tarsus Pharmaceuticals, a developer of treatments for ophthalmic and dermatologic diseases, announced that it will acquire Alkeus Pharmaceuticals for a total of $800 million, according to BioPharma Dive on the 6th (local time). The acquisition is intended to secure gildeuretinol, Alkeus’ investigational treatment candidate for Stargardt disease.

Under the agreement, Tarsus will make upfront payments to Alkeus of $270 million in cash and $180 million in Tarsus stock. It will then pay up to an additional $350 million in milestones tied to achievements including gildeuretinol’s regulatory approval and first commercial sale. Alkeus shareholders will also receive separate sales royalties.

Gildeuretinol is attracting attention because it targets Stargardt disease, for which there is currently no treatment. Stargardt disease is a rare disorder that causes vision loss when a genetic defect disrupts the metabolism of vitamin A needed by retinal cells. Gildeuretinol is a modified form of vitamin A that works by blocking the chemical reaction that causes vision damage.

The drug’s potential has also been underscored by praise from a prominent figure. Josh Boger, founder of U.S. biotech company Vertex Pharmaceuticals, served as chairman of Alkeus’ board and previously described gildeuretinol as “the most perfect drug I’ve ever seen, if it succeeds.”

Positive data have also emerged in clinical trials. In one placebo-controlled study, the annual growth rate of lesions that cause vision loss was reduced by 29.5% in the gildeuretinol group. Another study found that the likelihood of losing vision in dark conditions was significantly lower than in the placebo group. More than 400 people have received the drug to date, with some continuing treatment for up to 7 years.

“Gildeuretinol has the potential to change the treatment paradigm for Stargardt disease,” said Tarsus CEO Bobby Azamian. A phase 3 trial is currently underway in approximately 230 patients, with results expected in 2029.