RARE Daily

Alkeus Raises $150 Million to Advance Treatment for Stargardt Disease to Market

June 6, 2023

Rare Daily Staff

Alkeus Pharmaceuticals said it has raised a $150 million in a series B financing to support the registration and launch of its experimental therapy gildeuretinol to treat the rare, genetic eye condition Stargardt disease and named former Vertex Pharmaceuticals found Joshua Boger as its executive chairman.

Bain Capital Life Sciences led the financing round with additional participation by TCGX, Wellington Management, and Sofinnova Investments.

“The proceeds from this $150 million financing will allow Alkeus to expand our team and drive gildeuretinol towards NDA submission and FDA approval as rapidly as possible,” said Leonide Saad, CEO, president, and co-founder of Alkeus.

Stargardt disease is a leading cause of blindness in children and young adults, affecting more than 30,000 people in the United States and more than 150,000 worldwide. Patients with Stargardt disease are typically born with normal vision, but mutations in the ABCA4 gene lead to accelerated dimerization — or clumping—of vitamin A in the eye, causing damage to the retina and subsequent progressive vision loss, starting as early as five years of age.

Gildeuretinol is a chemically altered form of vitamin A with the potential to slow or halt the progression of vision loss in Stargardt patients without disrupting the visual cycle. The U.S. Food and Drug Administration has granted gildeuretinol Breakthrough Therapy designation and Orphan Drug designation. Alkeus plans to apply for FDA approval of gildeuretinol in 2024.

Phase 2 clinical data has shown a statistically and clinically meaningful slowing of retinal damage in Stargardt patients.

The newly appointed Boger said that the phase 2 data was “positive and compelling” across multiple stages of the disease, including a showing of statistically- and clinically-significant slowing of disease in patients with advanced disease. Additional data, he noted, showed a halting of disease in patients, including children.

“We are now poised to make this transformative medicine a reality for all Stargardt patients,” he said.

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