Esperion Tumbles on Update for Lead Product Candidate- Esperion Therapeutics (ESPR) shares plunged more than 26% in after-hours trading on Tuesday after the company provided an update on the clinical development and regulatory path for lead product candidate bempedoic acid, its candidate for lowering "bad" cholesterol in patients intolerant of statins. Investors have been mainly disappointed with the news that the FDA has refused to clarify a regulatory pathway for the LDL-C indication citing potential changes in their view of LDL-C lowering as a surrogate endpoint and the possibility of a shift in the future standard-of-care for statin-intolerant patients with elevated LDL-C. ESPR noted that if lowering LDL-C is no longer a surrogate endpoint for approval, it will still submit a New Drug Application (NDA) for a CV disease risk reduction claim by 2022. This means a significant delay in potential approval in the U.S. However, there some positive news as well. ESPR said that a global pivotal Phase 3 trial to evaluate the safety and efficacy is expected to start in the fourth quarter. A cardiovascular outcomes study is also expected to commence in the fourth quarter. The company believes that the study timelines will support a MAA in Europe in 2019 for patients with elevated LDL-C.
Intra-Cellular Commences Phase 3 Study of Lead Product Candidate- Intra-Cellular Therapeutics (ITCI) announced hat it has initiated a Phase 3 clinical trial evaluating its lead product candidate ITI-007 for the treatment of agitation in patients with dementia, including Alzheimer's disease (AD). The primary endpoint is the treatment effect compared to placebo as measured by a 37-item scale called Cohen-Mansfield Agitation Inventory - Community version (CMAI-C) which measures the ability of a drug to reduce the overall frequency of agitation symptoms, including aggressive behaviors.
BioLineRx Files Regulatory Submissions to Initiate Phase 2a Trial for BL-8040- BioLineRx (BLRX) announced that it has filed regulatory submissions required to initiate a Phase 2a trial assessing BL-8040, in combination with Merck's (MRK) KEYTRUDA (pembrolizumab), for the treatment of pancreatic cancer. The study, called COMBAT, is expected to commence in Q3. The study is designed to evaluate the clinical response, safety and tolerability of the combination as well as multiple pharmacodynamic parameters, including the ability to improve infiltration of T cells into the tumor and their reactivity in patients with metastatic pancreatic adenocarcinoma.
Cempra Submits Solithromycin MAA- Cempra (CEMP) announced that it has completed submission of its marketing authorization application (MAA) for solithromycin to the European Medicines Agency (EMA) for the treatment of community-acquired bacterial pneumonia (CABP). The MAA is for the intravenous and oral capsule formulations. The submission in Europe follows the completed submissions of two New Drug Applications (NDAs) in the U.S. several months ago.
FDA to Review Egalet’s ARYMO ER NDA on August 4- Egalet (EGLT) announced that a joint meeting between the FDA's Anesthetic and Analgesic Drug Products Advisory Committee and the Drug Safety and Risk Management Advisory Committee will take place on August 4 to review its New Drug Application (NDA) seeking approval of ARYMO ER (morphine sulfate) extended-release tablets, an abuse-deterrent formulation of morphine, for the long-term management of pain. The FDA's action date (PDUFA) is October 14.
Pfizer’s Bococizumab Succeeds in Late Stage Study- Pfizer (PFE) announced successful outcomes in two additional Phase 3 clinical trials assessing its PCSK9 inhibitor bococizumab in patients with high cholesterol. The studies, SPIRE-HR and SPIRE-FH, are the third and fourth of six SPIRE lipid-lowering studies to be completed and demonstrate positive results. SPIRE-HR evaluated bococizumab in 711 adults with primary hyperlipidemia or mixed dyslipidemia at high or very high risk of cardiovascular (CV) events who were receiving a maximally tolerated dose of statin therapy. The primary endpoint was the percent change in LDL-C from baseline to week 12 versus placebo. SPIRE-FH evaluated bococizumab in 370 adults with hereditary high cholesterol, heterozygous familial hypercholesterolemia (FeFH), at high or very risk of CV events who were receiving a maximally tolerated dose of statin therapy. The primary endpoint was the same.
Marinus Reports Top-Line Data from Phase 2 Exploratory Study for Lead Product Candidate- Marinus Pharma (MRNS) announced top-line data from a Phase 2 exploratory study evaluating its lead product candidate, ganaxolone, for the treatment of anxiety and attention in children with Fragile X syndrome (FXS) showed some positive effects despite failing to achieve the primary endpoint. However, MRNS plans to advance clinical development for the indication. The company believes that the results strongly support the rationale for advancing clinical development.
Coherus BioSciences Reports Encouraging Data for CHS-131- Coherus BioSciences (CHRS) announced that a Phase 2b clinical trial evaluating its CHS-131 n treatment-naive relapsing remitting multiple sclerosis (RRMS) patients has produced encouraging results. Treated patients showed a 50% decrease in the incidence of new contrast-enhancing (CE) lesions over six months compared to placebo. The primary endpoint of the study was the reduction in the cumulative number of (CE) lesions from baseline to week 24 (determined by MRI) versus placebo. Treated patients experienced a statistically valid reduction in CE lesions that was dose dependent. The 3 mg cohort showed a 46.3% reduction (p=0.01).