Rare Disease Drug for Blindness Towards FDA Approval
Alkeus Pharmaceuticals Secures $150 Million in Series B Funding to Advance Innovative Treatment for Stargardt Disease ????️???? In a significant development, Alkeus Pharmaceuticals has successfully raised $150 million through a Series B funding round. The company aims to utilize this investment to further develop gildeuretinol, a precision medicine candidate designed to combat Stargardt disease.
This genetic disorder, which currently lacks a cure, causes blindness in children and young adults. The recent announcement, made on Monday, revealed that the funding will be primarily used to propel gildeuretinol through the FDA approval process and prepare for the New Drug Application (NDA) submission.
While specific expansion plans were not disclosed, Alkeus intends to leverage the funding to grow its operations and make significant strides in the field. Gildeuretinol, which has obtained breakthrough therapy designation and orphan drug designation, is expected to undergo NDA filing next year. These designations signify the drug candidate’s potential to address a critical unmet medical need.
Alkeus Pharmaceuticals secured breakthrough therapy designation for gildeuretinol back in 2021, highlighting its promising therapeutic capabilities. Bain led the Series B funding round, with notable contributions from TCGX, Wellington Management, and Sofinnova Investments, further validating the potential of Alkeus Pharmaceuticals and its innovative approach to treating Stargardt disease. Research suggests that Stargardt disease affects approximately 1 in 8,000 to 10,000 individuals.
The disease initially presents with normal vision, but mutations in the ABCA4 gene result in the clumping of vitamin A in the eye. This damages a portion of the retina, leading to progressive vision loss. Alkeus aims to address this challenge with gildeuretinol, which is a modified form of vitamin A.
By replacing three hydrogen atoms with deuterium, the drug candidate minimizes the clumping tendency, potentially slowing down the progression of the disease. In a Phase II clinical trial involving 50 patients with Stargardt disease, Alkeus Pharmaceuticals achieved a significant milestone. The trial successfully met the primary endpoint for efficacy, with gildeuretinol demonstrating a remarkable 21% reduction in the growth of atrophic lesions. These compelling results were supported by a statistically significant p-value of p<0.001. Additionally, Alkeus Pharmaceuticals announced the return of Joshua Boger, co-founder and former CEO of Vertex, as the executive chairman. Boger, an accomplished veteran in the biotech industry, previously served as Alkeus’ chairman from 2012 to 2016.
His return signifies a renewed commitment to the company’s mission and signals a period of accelerated growth. In a recent LinkedIn post, Boger expressed his enthusiasm for the company’s future, stating, “I’ll help build a company: Alkeus was literally two employees earlier this year. We’ve just about doubled twice already and are poised to double three or four more times in the coming months.
Just snagged $150M to get the job done. This all seemed more important to do than anything else I could be doing. I’ve got one more in me.” While other biotech companies, including Nanoscope Therapeutics and Ascidian, are also exploring treatments for Stargardt disease, Alkeus Pharmaceuticals remains at the forefront of innovation. In addition to Stargardt disease, the company is actively investigating the potential of gildeuretinol in addressing geographic atrophy secondary to dry age-related macular degeneration (AMD).
Rare Disease Drug
