Alkeus Pharmaceuticals announced recently that it has raised a $150 million Series B financing to support the registration and launch of gildeuretinol (ALK-001), which is a potential disease-modifying, precision medicine for the treatment of Stargardt disease (a leading genetic cause of blindness in children and young adults). This funding round was led by Bain Capital Life Sciences with additional participation by TCGX, Wellington Management and Sofinnova Investments. Plus Alkeus also announced that Joshua Boger, Ph.D., a biotech industry pioneer and the founder of Vertex Pharmaceuticals, has joined Alkeus as Executive Chairman.
Stargardt disease is a leading cause of blindness in children and young adults, affecting more than 30,000 people in the U.S. 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 the first and only medicine in clinical development to address the underlying mechanism of Stargardt disease by substantially reducing vitamin A dimerization in the eye, without any impact on normal vision. Phase 2 clinical data has shown a statistically and clinically meaningful slowing of retinal damage in Stargardt patients. The U.S. FDA has granted Breakthrough Therapy Designation and Orphan Drug Designation to gildeuretinol (ALK-001). Alkeus plans to submit an NDA for approval of gildeuretinol in 2024.
KEY QUOTES:
“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. I am thrilled to work hand in hand with Joshua as Executive Chairman as we build a patient-focused company. Joshua’s deep experience leading the successful development and commercialization of transformative medicines will be of tremendous value to Alkeus during this period of organizational growth, in preparation for the launch of gildeuretinol.”
– Leonide Saad, Ph.D., CEO, President, and Co-Founder of Alkeus
“Stargardt is a progressive, debilitating disease that leads inevitably to devastating vision loss. The genetic cause is well characterized, but to date there is no effective treatment. Leonide and the Alkeus team have brought gildeuretinol from the lab all the way to positive and compelling clinical data across multiple stages of the disease, including a Phase 2 placebo-controlled trial in patients with advanced disease, where we have reported a statistically- and clinically-significant slowing of disease. Additional clinical data show halting of disease in patients, including children.”
“We are now poised to make this transformative medicine a reality for all Stargardt patients.”
– Joshua Boger, Ph.D., Executive Chairman of Alkeus
