Stealth BioTherapeutics Shares Drop 12% After FDA Refusal to File Letter
October 20 2021 - 01:11PM
Dow Jones News
By Chris Wack
Stealth BioTherapeutics Corp. shares were down 12% to $1.22
after the company said it has received a refusal to file letter
from the U.S. Food and Drug Administration regarding its new drug
application for elamipretide, a mitochondria-targeted therapy for
the treatment of Barth syndrome.
The biotechnology company said the FDA determined, upon its
preliminary review, that the NDA wasn't sufficiently complete to
permit a substantive review.
Volume for the stock was 768,000 shares at 12:35 p.m. ET,
compared with its 65-day average volume of 295,000 shares. The
stock hit its 52-week low of $1.06 on April 21.
In the letter, the FDA stated that the NDA doesn't contain an
adequate and well-controlled trial that provides evidence of
effectiveness, noting that the SPIBA-201 Phase 2 clinical trial of
elamipretide for the treatment of Barth syndrome was negative
during the randomized, double-blind portion of the study and that
the FDA doesn't consider the open label extension of the SPIBA-201
trial to be adequate and well-controlled.
The FDA said it was committed to work with Stealth on a pathway
by which a beneficial effect of elamipretide might be demonstrated
within the small patient population.
The letter didn't explain why the company's positive Phase 3
trial comparing SPIBA-201 results with a retrospective natural
history control and the primary basis for the NDA submission
wouldn't be considered an adequate and well-controlled trial.
Stealth is evaluating the appropriate next steps and anticipates
providing an update in early November.
Elamipretide was previously granted rare pediatric designation,
fast-track designation and orphan drug designation by the FDA, as
well as orphan drug designation by the European Medicines Agency,
for the treatment of Barth syndrome.
Write to Chris Wack at chris.wack@wsj.com
(END) Dow Jones Newswires
October 20, 2021 12:56 ET (16:56 GMT)
Copyright (c) 2021 Dow Jones & Company, Inc.
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