Stealth Biotherapeutics Corp (NASDAQ: MITO) has joined an alliance for life science companies focusing on rare diseases treatments discovery, development, and delivery to Rare Disease Company Coalition patients.
Coalition to advocate for better prescription drugs pricing laws
Reenie McCarthy, Stealth CEO, said, “We are thrilled to be a part of the first-of-its-kind Rare Disease Company Coalition, which is focused on supporting rare disease patients through the development of innovative therapeutics. We look forward to working with the other inspiring companies to foster greater understanding of the unique challenges and circumstances associated with the development of rare disease treatments.”
The coalition will work with policymakers to advocate for important pharmaceutical and healthcare laws and regulations currently being debated, such as prescription drugs pricing, and emphasize the negative effects that blanket legislation can have on rare disease therapy innovation.
The coalition’s mission as an education and an advocacy-focused group of enterprises is to educate lawmakers about the distinctive challenges and opportunities of rare disease drug research, development, and production for tiny populations in order to keep important innovation going. To achieve this, the coalition will push for long-term, consistent, sustainable, and equitable government policies allowing life science companies to continue providing rare disease patients with access to approved treatments.
Stealth developing treatment for dysfunctional mitochondria-related rare diseases
Stealth is developing a treatment for rare genetic diseases characterized by dysfunctional mitochondria. The company believes its flagship drug candidate, elamipretide, can potentially treat rare metabolic cardiomyopathies such as Duchene muscular dystrophy, Barth and Friedreich’s Ataxia, as well as other rare mitochondrial dysfunction diseases that entail nuclear DNA mutations. Similarly, the company is evaluating a second-generation product candidate, SBT-272, in clinical-stage trials and SBT-550 to treat various rare neurological indications after encouraging pre-clinical data.