BioMarin Submits Long-Term Efficacy Data for Cerliponase Alfa- BioMarin Pharmaceuticals (BMRN) announced that it has submitted ong-term efficacy data supporting the use of cerliponase alfa in children with CLN2 disease, a form of Batten disease, a rare inherited disorder in which the body's cells lose their ability to dispose of wastes. The majority of affected children are no longer able to walk and talk by age six. The submission was made as per a request from the FDA. he agency designated the submission as a major amendment so its action date (PDUFA) has been extended three months to April 27, 2017. An advisory committee meeting will also be held, although the specific date has not been set.
Retrophin Announces Positive Results from DUET Study- Retrophin (RTRX) shares closed more than 27% higher on Wednesday after the company announced positive results in a Phase 2 clinical trial, DUET, assessing its sparsentan (RE-021) for the treatment of focal segmental glomerulosclerosis, a rare kidney disorder that leads to end-stage renal disease. The study met its primary endpoint of a statistically valid reduction (more than two-fold) in proteinuria (protein in urine) compared to irbesartan [Sanofi's (NYSE:SNY) Avapro] after an eight-week treatment period. Specifically, patients treated with 200, 400 and 800 mg/day of sparsentan (n=64) showed a mean reduction of proteinuria from baseline of 44.8% after eight weeks of treatment compared to 18% for those (n=32) receiving 300 mg/day of irbesartan (p=0.006). The 400 mg and 800 mg sparsentan cohorts also showed superiority to 300 mg of irbesartan, 47.4% vs. 19.0% (p=0.011).
Agios Rallies as Celgene Files NDA for AG-221- Agios Pharmaceuticals (AGIO) shares rose more than 25% on Wednesday after the company announced that its collaboration partner Celgene (CELG) plans to file a New Drug Application (NDA) with the FDA by year end seeking approval of Agios' AG-221 for the treatment of patients with advanced blood cancers who have a mutation in a protein called isocitrate dehydrogenase-2 (IDH2). Celgene will be discussing its plans for the NDA today at the Citi 11th Annual Biotech Conference in Boston. The data supporting the NDA was generated in a Phase 1/2 study in patients with IDH2 mutation-positive acute myeloid leukemia (AML). AG-221 (enasidenib) inhibits IDH2.
Evoke Pharma Provides Update on EVK-001- Evoke Pharma (EVOK) shares surged on Wednesday after the company announced results from its pre-NDA meeting with the FDA regarding lead product candidate Gimoti (EVK-001)(metoclopramide nasal spray) for the relief of symptoms related to acute/recurrent diabetic gastroparesis in adult women. Based on the agency's response to the information package and what was discussed during the meeting, the company believes it has the information it needs to complete the sections in a manner acceptable to the FDA.
CytRx Completes Enrollment in Mid-Stage Study of Lead Product Candidate- CytRx (CYTR) announced that it has completed enrollment of 132 patients in its Phase 2b clinical trial assessing lead product candidate aldoxorubicin in treatment-experienced patients with small cell lung cancer (SCLC). The primary endpoint is progression-free survival (PFS) at Month 24. Overall survival is a secondary endpoint. According to ClinicalTrials.gov, the estimated study completion date is July 2017, but this needs revising since the completion should be September 2018 based on two-year PFS data.
Raptor Pharmaceuticals Announces Data for Inhaled Levofloxacin- Raptor Pharmaceuticals (RPTP) announced that a meta-analysis of all randomized clinical trials of inhaled antibiotics in cystic fibrosis (CF) patients showed QUINSAIR (aerosolized form of levofloxacin) had similar efficacy to three other inhalable antibiotics in treating lung infections involving the bacterium Pseudomonas aeruginosa, the primary cause of progressive lung function decline in CF sufferers. The data were presented today at the European Respiratory Society International Congress in London. QUINSAR, acquired from Tripex Pharmaceuticals in August 2015, was approved in Europe in March 2015 and in Canada three months later. It is not yet available in the U.S.
Capricor Completes Enrollment in Early Stage Study of Lead Product Candidate- Capricor Therapeutics (CAPR) announced that it has completed enrollment target of 25 patients n its Phase 1/2 study, HOPE-Duchenne, assessing lead product candidate CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). The primary endpoint of the trial is the safety and tolerability of CAP-1002 72 hours post infusion. Secondary endpoints include a cardiac structural composite measured by MRI, a functional composite of mobility measurements and a quality-of-life composite. According to ClinicalTrials.gov, the estimated final data collection date for the primary endpoint is August.