Stealth BioTherapeutics Corp
Q4 2020 Earnings Call Transcript

Published: 6 Apr 2021

[Operator:]

Greetings. Welcome to the Stealth BioTherapeutics Fiscal Year 2020 Financial Results and recent Business Highlights. At this all participants will be in listen-only mode. A brief question-and-answer session will follow the formal presentation. Please note this conference is being recorded. At this time, I’ll turn the conference over to Henry Hess, Chief Legal Counsel. Mr. Hess, you may begin.

[Henry Hess:]

Good morning. I'd like to remind listeners that management will be making forward-looking statements on today's call, including, for example, the company's expected timeline and plans for development of Elamipretide and other pipeline programs, regulatory interactions and financial position and cash runway.

[Reenie McCarthy:]

Thank you, Henry. And thanks to those on the line for joining us today. With me and Henry on the line are Jim Carr, our Chief Clinical Development Officer; Rob Weiskopf, our Chief Financial Officer; and Brian Blakey, our Chief Commercial Officer. So during 2020, our team rallied from very challenging circumstances coming into the year to define a new path forward in rare cardiomyopathies. We continue to execute strongly on our dry AMD trial through the COVID-19 shutdown. And we made significant progress toward advancing our rare neurology pipeline. We expect 2021 to be another strong execution year, ahead of several key milestones expected in 2022. We have made progress in our efforts to develop an IVT formulation for dry AMD with a goal of informing Phase 3 formulation decisions commensurate with our Phase 2 data readout right around this time next year. In our rare cardiomyopathy franchise, as you can see from our press release, we've had a number of very recent interactions, including just last week with senior officials at both the office and division level of FDA, who have been very engaged in evaluating a path to registration in Barth. This may lead to a delay in our NDA submission if we agree to generate additional data, including by utilizing the existing open-label patients as FDA has suggested. We're evaluating various options along these lines, and we're continuing to seek engagement with the agency. As previously announced, we have rare pediatric designation for Barth. For other rare cardiomyopathy indications are Friedreich's trial we’ll start in the next several months. And we plan to engage with FDA regarding our Duchenne trial later this year. For our neurology platform, we plan to initiate Phase 3 trials in the prespecified subgroup of patients who responded in our mitochondrial myopathy program. We are also continuing to generate preclinical data with SBT-272, and other rare neurology indications, which we expect would be diseases affecting the central nervous system such as ALS. And now -