Regulus Tumbles in Pre-market Trading- Regulus Pharmaceuticals (RGLS) shares have fallen almost 50% in pre-market trading today after the company was verbally notified by the FDA that its Investigational New Drug (IND) application for lead product candidate RG-101 for the treatment of hepatitis C virus (HCV) infection has been placed on clinical hold (prohibiting the recruitment of new patients to clinical studies) after the company reported a second serious adverse event (SAE) of jaundice. The SAE occurred in an HCV patient with end-stage renal disease on dialysis in its ongoing U.S.-based Phase 1 trial 117 days after receiving a single dose of RG-101. Regulus expects to receive a clinical hold letter within the next 30 days. It intends to work closely with agency to promptly investigate and resolve the issue.
In an article earlier today, I noted that it seems unlikely that serious adverse events were treatment related. The sell-off therefore could be an opportunity to enter RGLS.
GW Pharma’s Epidiolex Succeeds in Late-Stage Study- GW Pharmaceuticals (GWPH) shares rose more than 6% on Monday after the company announced positive top-line data from a Phase 3 clinical trial evaluating its lead product candidate, Orphan Drug-tagged Epidiolex (cannabidiol), for the treatment of Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy. The study achieved its primary endpoint of a statistically valid reduction in the monthly frequency of drop seizures over the 14-week treatment period compared to placebo (p=0.0135).
Medicine Company’s Carbavance Succeeds in Late-Stage Study- The Medicine Company’s (MDCO) announced that a Phase 3 clinical trial, TANGO 1, evaluating its investigational antibiotic, Fast Track-tagged CARBAVANCE (meropenem-vaborbactam), for the treatment of complicated urinary tract infections (cUTI) met both FDA and EMA pre-specified endpoints, showing an overall success rate of 98.4%, statistically significantly better than piperacillin-tazobactam. The study met the FDA's primary endpoint of overall success rate (98.4% for CARBAVANCE versus 94.0% for piperacillin-tazobactam) and the European Medicines Agency's primary endpoint of microbiological outcome of eradication versus baseline (66.7% for CARBAVANCE compared to 57.7% for piperacillin-tazobactam). Treatment-emergent adverse events (AEs) were similar: 39.0% for CARBAVANCE and 35.5% for piperacillin-tazobactam as were serious AEs: 4.0% and 4.4%, respectively. There were two deaths in each treatment group.
Puma Biotechnology Submits MAA for Neratinib- Puma Biotechnology (PBYI) announced that it has submitted ts Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for PB272 (Neratinib). The potential indication is for the extended adjuvant treatment of HER2-positive early stage breast cancer that has previously been treated with Trastuzumab (Herceptin)-based adjuvant therapy. The company has provided data from he ExteNET Phase III study which showed treatment with neratinib resulted in a 33% reduction of risk of invasive disease recurrence or death versus placebo (hazard ratio = 0.67, p = 0.009).
Teva Launches Generic Version of Imitrex- Teva Pharmaceutical Industries (TEVA) announced the launch of its generic equivalent of GlaxoSmithKline’s (GSK) Imitrex (sumatriptan succinate) in the U.S. for the treatment of acute migraine, with or without aura, and acute cluster headaches in adults. According to data from IMS, sumatriptan injection sales in the U.S. are ~$183M a year.
Meanwhile, Reuters has reported that Teva is close to divesting assets worth $600 million to Australia’s Mayne Pharma. TEVA has been divesting some of its businesses in order to get a nod from regulators for its $40 billion acquisition of Allergan’s (AGN) generic business.
Emergent BioSolutions Bags BARDA Contract- Emergent BioSolutions (EBS) announced the receipt of a task order from the Biomedical Advanced Research and Development Authority (BARDA) to develop and manufacture three GMP-compliant lots of Zika vaccine, which will be used in a Phase 1 clinical trial. BARDA will provide the base vaccine candidate while Emergent will conduct technology transfer of process materials and information, process and analytical method development, execute small-scale production runs and perform cGMP (current Good Manufacturing Practice) cell banking leading to cGMP manufacture of bulk drug substance and final product.
Ionis Pharmaceuticals Reports Encouraging Results for Volanesorsen- Ionis Pharmaceuticals (IONS) announced that a Phase 2 clinical trial evaluating volanesorsen (formerly ISIS-APOCIIIrx) in patients with high plasma triglycerides and type 2 diabetes showed encouraging results, including an improvement in insulin sensitivity. The data have been published in the June edition of the journal Diabetes Care (paper titled: "Antisense-Mediated Lowering of Plasma Apolipoprotein C-III by Volanesorsen Improves Dyslipidemia and Insulin Sensitivity in Type 2 Diabetes").
Horizon Pharma Considering Potential Stake Sale- Horizon Pharma (HZNP) has hired Bank of America to help in selling a significant equity stake to an investor, according to Reuters. Reuters, citing people familiar with the matter reported, the stake sale would boost HZNP’s balance sheet. Reuters noted that it is not clear how HZNP would use the proceeds from the sale.