Stealth BioTherapeutics Corp (NASDAQ:MITO) Q4 2019 Earnings Conference Call - Final Transcript

Apr 01, 2020 • 08:30 am ET

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Stealth BioTherapeutics Corp (NASDAQ:MITO) Q4 2019 Earnings Conference Call - Final Transcript

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Presentation
Executive
Reenie McCarthy

to stand, and a multi-domain Responder Index.

In early March, we announced that the FDA had granted us rare pediatric disease designation for Barth, for which we may be entitled to a rare pediatric voucher, should we receive approval in conjunction with an expedited review.

More recently, we met with the FDA to discuss the natural history comparative control efficacy study and the TAZPOWER open label extension, cardiac surrogate endpoint and functional endpoint data. Our meeting was productive and informative relative to our regulatory path forward, and we look forward to providing further details once we've received the meeting minutes.

We are actively exploring pipeline expansion opportunities in other rare metabolic cardiomyopathies. These include Duchenne and Becker's muscular dystrophies, Friedreich's ataxia, all of which entail heart failure as the leading cause of premature death. We've met with leading advocacy groups and we plan to convene a Cardiology Advisory Board to assess endpoints and trial design. We hope to meet with FDA to discuss this initiative towards the end of this year.

Moving onto our ophthalmic pipeline, we continue to enroll patients for our Phase 2b clinical trial, which is ReCLAIM-2b, which is evaluating the effect of Elamipretide in patients with dry AMD with geographic atrophy. We have Fast Track status on that program from the FDA.

The primary endpoint of the trial is agreed with the FDA as low luminance visual acuity, which is low light or night vision, particularly compromised in patients with geographic atrophy. We're also evaluating secondary endpoints, including other visual function endpoint. The rate of progression of GA by optical coherence tomography and fundus autofluorescence, as well as visual function and quality of life questionnaires.

Since our AMD trial is primarily being conducted in elderly patient population, we are experiencing some enrolment delay as a result of the COVID-19 pandemic. We are currently over 2/3 enrolled and we had anticipated completing enrolment during the first half of this year. In light of the impact of physical distancing restrictions on clinics and patients, to be conservative, we're adjusting our estimate of enrollment completion for the second half of this year. We will continue to monitor the situation closely, and we will make any necessary updates, should this change in a meaningful way.

We submitted our Phase 3 protocol for Elamipretide in Leber's hereditary optic neuropathy at the end of 2019. And we look forward to receiving comments from the FDA on that protocol. Consistent with prior guidance, we do not intend to initiate this protocol until 2021, due to financing constraints.

In January, we advanced our next generation product candidate, SBT-272, which has improved CNS penetration and is being evaluated for the treatment of ALS and rare neurodegenerative diseases into a Phase 1 trial to evaluate safety and tolerability, pharmacokinetics and appropriate dose range. The Phase 1 trial is a double-blind placebo controlled single ascending dose study, enrolling up to 40 healthy subjects across multiple cohorts. SBT-272 is being administered orally in this study.

We still expect