ALDX Catches Eye, RVNC Faces Busy Year Ahead, SRPT Plunges On DMD Trial Data
BEIJING (dpa-AFX) - Today s Daily Dose brings you news about Alnylam s transthyretin-mediated (ATTR) amyloidosis trial results; Sarepta s mixed results of investigational gene therapy SRP-9001 in patients with Duchenne muscular dystrophy; Aldeyra s dry eye disease trial results; and Revance s anticipated milestones.
Read on.
1. Aldeyra Catches Investor s Eye
Aldeyra Therapeutics Inc. s (ALDX) run-in cohort of phase III trial of Reproxalap in patients with dry eye disease has achieved statistical significance for ocular redness, an FDA-approvable sign, and clinical symptoms of ocular dryness and discomfort.
Dry eye disease is a common inflammatory disease affecting an estimated 34 million or more adults in the United States. The disease is characterized by insufficient moisture and lubrication in the anterior surface of the eye, leading to dryness, inflammation, pain, discomfort, irritation, diminished q
Mid-Day Market Update: Dow Falls 50 Points; Chimerix Shares Spike Higher
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Mid-Afternoon Market Update: Gold Dips 3 5%; Merus Shares Surge
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Author Bio
Keith began writing for the Fool in 2012 and focuses primarily on healthcare investing topics. His background includes serving in management and consulting for the healthcare technology, health insurance, medical device, and pharmacy benefits management industries.
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What happened
Shares of
Sarepta Therapeutics (NASDAQ:SRPT) were crashing 50.7% as of 11:11 a.m. EST on Friday. The huge decline came after the biotech announced top-line results late on Thursday from the first part of a clinical study evaluating experimental gene therapy SRP-9001 in treating Duchenne muscular dystrophy (DMD).
Sarepta reported that SRP-9001 met its primary biological endpoint of micro-dystrophin protein express. DMD is caused by a genetic mutation that results in lower or no production of dystrophin, which is important for muscles to function properly.
Date Time
Pfizer Doses First Participant in Phase 3 CIFFREO study
NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that the first participant has been dosed in the Phase 3 CIFFREO study, which will evaluate the efficacy and safety of investigational gene therapy candidate PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The CIFFREO trial is expected to enroll 99 ambulatory male patients, ages 4 through 7, across 55 clinical trial sites in 15 countries. The first patient was dosed at a site in Barcelona, Spain on December 29, 2020.
CIFFREO is a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled study. The primary endpoint of the study is the change from baseline in the North Star Ambulatory Assessment (NSAA) at one year. The NSAA is a 17-item test that measures gross motor function in boys with DMD. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the