BOSTON – December 18, 2017 – Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead candidate, elamipretide, for the treatment of Leber’s hereditary optic neuropathy (LHON).
“We are pleased with the FDA’s decision to grant Fast Track designation to elamipretide for the potential treatment of LHON,” Stealth BioTherapeutics Chief Executive Officer Reenie McCarthy said. “This designation, a first for our ophthalmology program, is evidence of the serious need for new therapies for those suffering from this devastating rare mitochondrial disease.”
LHON is an inherited mitochondrial disease that causes loss of central vision due to damage to neurons in the retina, called retinal ganglion cells, as a result of dysfunctional mitochondria. The disease, which affects approximately 35,000 people worldwide, can lead to legal blindness.
In June 2016, Stealth announced the initiation of ReSIGHT, a Phase 2, prospective, double-masked, vehicle-controlled clinical study to evaluate the safety, tolerability and efficacy of topical eye drop delivery of elamipretide in people with LHON. Top-line results from the study are expected by mid-2018.
Elamipretide has also been granted Fast Track designation for the treatment of primary mitochondrial myopathy (PMM) and Barth syndrome, two rare primary mitochondrial diseases.
The FDA’s Fast Track program facilitates the development and review of drugs to treat serious conditions with unmet medical needs. The designation provides the opportunity for more frequent meetings with the FDA over the course of development and allows a drug company to submit individual sections of its New Drug Application (NDA) for review as they are ready, rather than waiting until all sections of the NDA are complete. The designation also increases the likelihood of eligibility for priority review and accelerated approval if relevant criteria are met.
For additional information on the ReSIGHT study or elamipretide, please refer to Stealth’s website.
About Leber’s Hereditary Optic Neuropathy
Affecting approximately 35,000 people worldwide, LHON causes neuropathy of the optic nerve and retinal ganglion cells in the back of the eye and can lead to legal blindness. Mitochondrial dysfunction is a key factor in genetic optic neuropathies such as LHON, characterized by loss of visual function resulting from impaired cellular energetics. LHON primarily affects young men between the ages of 18 and 30. Vision problems, such as blurring or clouding of vision, may begin in one eye or both eyes at the same time. Over time, vision worsens in both eyes and often leads to severe and permanent legal blindness.
We are a privately held clinical-stage biotechnology company focused on the development of therapeutics for diseases involving mitochondrial dysfunction. We believe there is a strong rationale for our lead product candidate, elamipretide, in indications in these diseases based on encouraging preclinical and early clinical data. We are investigating elamipretide in three primary mitochondrial diseases – PMM, Barth syndrome and LHON – as well as in heart failure and dry age-related macular degeneration. We received Orphan Drug Designation of elamipretide for the treatment of PMM from the FDA in September 2017. We are developing our second product candidate, SBT-20, for neurodegenerative diseases. To learn more information about us and our pipeline, visit www.stealthbt.com.
Kate Contreras, 617-520-7088
Beth DelGiacco, 212-362-1200