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.
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 quality of life, and in severe cases, permanent vision impairment.
The trial, dubbed TRANQUILITY, enrolled 23 patients in the run-in cohort – 12 patients were randomized to receive 0.25% Reproxalap ophthalmic solution and 11 patients were randomized to receive vehicle ophthalmic solution.
The main cohort of TRANQUILITY is expected to begin enrollment in February 2021, following completion of the analysis of tear levels of pro-inflammatory reactive aldehyde species (RASP), and finalization of trial design.
ALDX closed Thursday’s trading at $8.47, up 26.42%.
2. Alnylam’s HELIOS-A Study Shines
Alnylam Pharmaceuticals Inc.’s (ALNY) phase III study of Vutrisiran for the treatment of transthyretin-mediated (ATTR) amyloidosis has yielded encouraging results.
Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.
The phase III study, dubbed HELIOS-A, met primary and all secondary endpoints at 9 months, with statistically significant improvements in progression of neuropathy, quality of life (QOL), and gait speed, relative to placebo. Vutrisiran also showed improvements in the 9-month exploratory cardiac endpoint of NT-proBNP compared to placebo.
Based on the positive results, the company plans to seek FDA approval for Vutrisiran in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan. In the EU, the company will be filing for authorization only after obtaining the results of the 18-month analysis – expected in late 2021.
ALNY closed Thursday’s trading at $139.40, up 9.91%.
3. Revance Faces a Busy Year Ahead
Revance Therapeutics Inc. (RVNC) has a couple of catalysts to watch out for this year.
The company’s DaxibotulinumtoxinA, proposed for the treatment of Glabellar Lines, is under FDA review, with a decision now expected this year. The original decision date was November 25, 2020, but it was deferred due to the FDA’s inability to conduct a required inspection of the company’s Northern California manufacturing facility as a result of COVID-19 pandemic travel restrictions.
The topline results from a phase II study of DaxibotulinumtoxinA for Injection in Muscle Movement Disorders, dubbed JUNIPER, are expected in first quarter 2021. Results from a Phase 3, long-term safety study of DaxibotulinumtoxinA for cervical dystonia, known as ASPEN-OLS, are expected in the second half 2021.
RVNC closed Thursday’s trading at $29.59, up 8.79%.
4. Sarepta Plunges on Duchenne Muscular Dystrophy Trial Data
Shares of Sarepta Therapeutics Inc. (SRPT) plunged 50% in after-hours Thursday, following top-line results from Part 1 of a phase II trial of investigational gene therapy SRP-9001 in patients with Duchenne muscular dystrophy, dubbed Study 102.
According to the company, the study met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in SRP-9001-treated participants versus placebo. However, the study did not achieve statistical significance on the primary functional endpoint of improvement in NSAA total score compared to placebo at 48 weeks post-treatment.
The NSAA or North Star Ambulatory Assessment is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne. It is used to monitor the progression of the disease and treatment effects which makes it suitable as an endpoint in clinical trials for Duchenne.
In the Study 102, all 41 participants have completed their Part 1, 48-week assessment and have entered the Part 2 crossover phase.
SRPT closed Thursday’s trading at $168.95, up 1.50%. In after-hours, the stock fell 50.01% to $84.45.
5. Stocks that Gained/Lost the Most
SOS Limited (SOS) closed Thursday’s trading at $3.07, up 78.49%.
Arcturus Therapeutics Holdings Inc. (ARCT) closed at $66.97, up 35.65%.
Oragenics Inc. (OGEN) closed at $0.84, up 35.48%.
Addex Therapeutics Ltd (ADXN) closed at $10.51, 21.57%.
ViewRay Inc. (VRAY) closed at $6.09, down 15.93%.
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