Aldeyra Therapeutics Inc.'s (ALDX) randomized vehicle-controlled clinical trial of topical dermatologic NS2 in patients with Sjögren-Larsson Syndrome has demonstrated superiority of NS2 over the vehicle in achieving clinically meaningful improvement.
Patients with Sjögren-Larsson Syndrome, or SLS, a rare inborn error of aldehyde metabolism, suffer from ichthyosis, a debilitating dermatologic disease characterized by dry, thickened, scaly skin.
The trial results suggest that NS2, when applied topically to the skin, has the potential to help patients suffering from SLS by lowering toxic aldehyde levels and preventing the dermal dysfunction that causes ichthyosis.
ALDX closed Tuesday's trading at $6.00, up 2.04%.
ARCA biopharma Inc. (ABIO) expects the outcome of the DSMB's interim analysis of the company's phase 2b/3 clinical trial of Gencaro in atrial fibrillation, dubbed GENETIC-AF trial, in the second quarter of 2017.
The company expects to enroll at least 150 patients in the trial by the end of 2016.
The company's experimental drug Gencaro was issued a Complete Response Letter by the FDA in 2009. The regulatory agency asked the company to conduct additional clinical efficacy trial, additional clinical pharmacology studies and additional non-clinical studies related to Gencaro.
ABIO closed Tuesday's trading at $3.05, down 0.65%.
Catalyst Pharmaceuticals Inc. (CPRX) awaits a busy 2017 with a couple of catalysts lined up for the year.
The company expects top-line data from two phase III trials of Firdapse for Lambert-Eaton myasthenic syndrome, or LEMS, in the second half of 2017.
A phase III trial of Firdapse for Congenital Myasthenic Syndromes is underway - with top-line data anticipated in the second half of 2017.
A small phase III investigator-sponsored study of Firdapse for MuSK antibody positive Myasthenia Gravis is ongoing, and top-line data is anticipated in early 2017.
CPRX closed Tuesday's trading 2.29% higher at $0.78.
Coherus BioSciences Inc. (CHRS) has submitted the biologics license application for CHS-1701, a biosimilar candidate to Amgen Inc.'s (AMGN) Neulasta, to the FDA under the 351(k) pathway.
Neulasta was approved by the FDA in January 2002 and by the European Medicines Agency in August 2002 to decrease the risk of infection associated with treatment for cancer. Neulasta had worldwide sales $4.72 billion in 2015 compared to $4.59 billion in 2014.
CHRS closed Tuesday's trading at $29.70, down 1.13%.
Eagle Pharmaceuticals (EGRX) rose over 14% on Tuesday after being told by the FDA that no additional human safety and efficacy data is required for the submission of the company's New Drug Application for Ryanodex for the treatment of exertional heat stroke.
Eagle completed its clinical study in EHS patients during the Hajj pilgrimage in Saudi Arabia last September.
EGRX closed Tuesday's trading at $55.43, up 14.62%.
Marinus Pharmaceuticals Inc. (MRNS) expects results from its exploratory phase II clinical trial of Ganaxolone in females with PCDH19 pediatric epilepsy this quarter.
The company is also exploring the use of an intravenous formulation of Ganaxolone for use in Status Epilepticus. Enrollment and dosing is continuing in a phase 1 clinical trial designed to evaluate the safety, pharmacokinetics, and pharmacodynamics of ganaxolone IV in healthy volunteers.
Upon a successful completion of the phase 1 study, Marinus plans to evaluate Ganaxolone IV in patients with SE. Observations of SE patient response to Ganaxolone are expected to be available first half 2017.
MRNS closed Tuesday's trading at $1.45, down 2.03%.
Voyager Therapeutics Inc. (VYGR) has initiated the third Cohort in its ongoing Phase 1b open-label trial of VY-AADC01 in patients with advanced Parkinson's disease.
The company is on track to deliver six-month data on safety, motor function and biomarkers from patients in Cohort 1 and 2, as well as preliminary data from some patients in Cohort 3 by the end of this year.
VYGR closed Tuesday's trading at $16.21, down 0.31%.
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