We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Stealth (MITO) Files NDA for Ultra-Rare Disease Candidate
Read MoreHide Full Article
Stealth BioTherapeutics Corp announced that it has submitted a new drug application (“NDA”) to the FDA, seeking approval for its lead pipeline candidate, elamipretide, for the treatment of Barth syndrome, an ultra-rare genetic condition characterized by a weakened heart. Currently, there is no treatment approved for the given indication.
The NDA was based on data from the phase III SPIBA-001 retrospective natural history control study, which compared data from the open-label portion of the phase II/III TAZPOWER study to matched natural history controls.
The SPIBA-001 study met the primary and most secondary endpoints by showing elamipretide-mediated improvements in assessments of exercise tolerance, strength and cardiac function.
Data from the TAZPOWER study also demonstrated improvements in several surrogate and intermediate clinical endpoints that may likely predict the clinical benefits for patients suffering this debilitating disease. This data might likely support an accelerated approval pathway for elamipretide.
Shares of Stealth BioTherapeutics have plunged 20.4% so far this year against the industry’s growth of 1.2%.
Image Source: Zacks Investment Research
We remind investors that the FDA has already granted a rare pediatric designation, fast track designation and an orphan drug designation to elamipretide for treating Barth syndrome. The European Medicines Agency has also granted an orphan drug designation to elamipretide for the given indication.
Apart from Barth syndrome, elamipretide is also being evaluated in mid- to late-stage studies for treating several other diseases like Geographic atrophy, mitochondrial diseases entailing nuclear DNA mutations, Duchenne muscular dystrophy and Friedreich's ataxia.
Stealth BioTherapeutics has no approved product in its portfolio at the moment. Therefore, pipeline development remains a key focus for the company. If successfully developed and upon potential approval, elamipretide will be a boost to the company’s growth prospects, as it will be able to generate revenues.
Image: Bigstock
Stealth (MITO) Files NDA for Ultra-Rare Disease Candidate
Stealth BioTherapeutics Corp announced that it has submitted a new drug application (“NDA”) to the FDA, seeking approval for its lead pipeline candidate, elamipretide, for the treatment of Barth syndrome, an ultra-rare genetic condition characterized by a weakened heart. Currently, there is no treatment approved for the given indication.
The NDA was based on data from the phase III SPIBA-001 retrospective natural history control study, which compared data from the open-label portion of the phase II/III TAZPOWER study to matched natural history controls.
The SPIBA-001 study met the primary and most secondary endpoints by showing elamipretide-mediated improvements in assessments of exercise tolerance, strength and cardiac function.
Data from the TAZPOWER study also demonstrated improvements in several surrogate and intermediate clinical endpoints that may likely predict the clinical benefits for patients suffering this debilitating disease. This data might likely support an accelerated approval pathway for elamipretide.
Shares of Stealth BioTherapeutics have plunged 20.4% so far this year against the industry’s growth of 1.2%.
Image Source: Zacks Investment Research
We remind investors that the FDA has already granted a rare pediatric designation, fast track designation and an orphan drug designation to elamipretide for treating Barth syndrome. The European Medicines Agency has also granted an orphan drug designation to elamipretide for the given indication.
Apart from Barth syndrome, elamipretide is also being evaluated in mid- to late-stage studies for treating several other diseases like Geographic atrophy, mitochondrial diseases entailing nuclear DNA mutations, Duchenne muscular dystrophy and Friedreich's ataxia.
Stealth BioTherapeutics has no approved product in its portfolio at the moment. Therefore, pipeline development remains a key focus for the company. If successfully developed and upon potential approval, elamipretide will be a boost to the company’s growth prospects, as it will be able to generate revenues.
Zacks Rank & Stocks to Consider
Stealth BioTherapeutics currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the biotech sector include Spero Therapeutics, Inc. (SPRO - Free Report) , Corvus Pharmaceuticals, Inc. (CRVS - Free Report) and Vertex Pharmaceuticals Incorporated (VRTX - Free Report) , all carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Spero Therapeutics’ loss per share estimates have narrowed 9.1% for 2021 and 12.4% for 2022, over the past 60 days.
Corvus Pharmaceuticals’ loss per share estimates have narrowed 24.4% for 2021 and 21.4% for 2022, over the past 60 days.
Vertex’s earnings estimates have been revised 10.2% upward for 2021 and 6.4% upward for 2022 over the past 60 days.