PTC Therapeutics announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, has recommended approval of expanding the indication of Translarna to include ambulatory children aged two to five years with nonsense mutation Duchenne muscular dystrophy, or nmDMD. This broadens the use beyond the current indication which is for ambulatory patients who are over five years of age. This recommendation is based on the CHMP’s review of PTC’s Study 030 trial results which the CHMP concluded demonstrates a positive benefit-risk ratio in this population. In addition to the label expansion, the CHMP has also recommended the renewal of the current marketing authorization of Translarna. PTC’s focus on early patient identification and market readiness have been intensified in anticipation of the CHMP recommendation and the launch of Translarna for patients as young as 2 years of age is planned to start immediately at the time of EC ratification.
Friday, June 1, 2018
Jazz remains ‘top idea’: RBC
RBC analyst Randall Stanicky reiterated an Outperform rating and $194 price target on Jazz Pharmaceuticals after hosting a dinner with Chairman and CEO Bruce Cozadd. In a research note to investors, Stanicky said he left the dinner more confident in targeted business development with a view that a deal is likely in 2018. Overall, the analyst said Jazz remains a top pick and sees “lots” of room to run for the stock.
Immunomedics to start Phase 2 of cancer med trial
Immunomedics announced that the company plans to initiate a Phase 2 pivotal TROPHY U-01 study of sacituzumab govitecan, the company’s lead investigational ADC, as a single agent in patients with locally advanced or metastatic urothelial cancer who have relapsed after a platinum-based regimen and/or immune checkpoint inhibitor therapy. The Phase 2 pivotal study, which is expected to be activated next week, will be a single arm, international, multicenter study that will enroll approximately 100 mUC patients who have received prior platinum-based and CPI treatment. The primary endpoint will be overall response rate, with duration of response, progression-free survival, and overall survival serving as secondary endpoints. Response assessments will be in accordance with RECIST 1.1 and all patients will be centrally reviewed. In addition, the study will also enroll an additional and separate cohort of patients who are cisplatin ineligible and have received prior CPI treatment to evaluate safety and efficacy in an earlier treatment setting.
Viking follows Madrigal NASH lead higher
Shares of the clinical-stage biotech Viking Therapeutics (NASDAQ:VKTX) soared following positive mid-stage trial results for Madrigal Pharmaceuticals‘ (NASDAQ:MDGL) nonalcoholic steatohepatitis (NASH) candidate MGL-3196.
The reason? Viking’s experimental NASH drug, VK2809, reportedly has a similar mechanism of action as MGL-3196. Investors are apparently bidding up Viking’s shares on the belief that VK2809’s mid-stage results will ultimately mirror those of MGL-3196.
However, shares in Intercept Pharmaceuticals (NASDAQ:ICPT), which sports the most advanced NASH candidate, Ocaliva, are down 10.3% at the time of writing. While Ocaliva is well ahead of both MGL-3196 and VK2809 in terms of clinical development, the drug does have some serious safety issues that could negatively impact its commercial uptake if approved.
NASH is widely expected to be the next multibillion-dollar drug market, thanks to the rising rates of obesity worldwide and the fact that there are no approved therapies for this condition. Intercept, Madrigal, and Viking are thus all hoping to beat out far larger competitors, like Allergan, Pfizer, and Gilead Sciences, in this emerging space and gain an all-important first-mover advantage.
Viking’s mid-stage NASH study is set to wrap up next month, according to ClinicalTrials.gov. A top-line data release should thus occur within the next four to six weeks. So, in light of this upcoming clinical catalyst, Viking’s stock appears primed to continue its upward trajectory, even after today’s monstrous move higher.
The long and short of it is that VK2809 should garner significant interest from multiple big pharmas currently vying for a piece of this enormous NASH market. As proof, Madrigal has already bubbled to the top of the buyout-rumor mill after MGL-3196’s breakout success due to the tremendous demand for these drugs. Put simply, it might be a good idea to grab at least a few shares of this promising biotech ahead of this upcoming clinical catalyst.
Thursday, May 31, 2018
Amgen, University of Texas MD Anderson Cancer Center in T-cell collaboration
Amgen and The University of Texas MD Anderson Cancer Center announced two multi-year collaboration agreements aimed at accelerating development of a variety of Amgen’s early-stage oncology therapies for patients with leukemia, myelodysplastic syndromes, multiple myeloma, small-cell lung cancer, and other non-lung cancers with small-cell histologies. The agreements combine Amgen therapies nearing clinical development or those that have already begun the process with MD Anderson’s translational medicine capabilities. The collaborations will focus on Amgen’s bispecific T cell engager BiTE , chimeric antigen receptor CAR T cell and small molecule programsThe five-year collaboration will begin with Phase 1 clinical studies for BiTE antibody constructs and CAR T cell therapies for multiple myeloma and small cell lung cancer. The second agreement spans four years and will study BiTE antibody constructs, CAR T and small molecule treatments in leukemia and myelodysplastic syndromes. The collaboration includes multi-institutional pre-clinical and clinical trials, some of which will be led by MD Anderson, which may offer the potential for identifying new biomarkers.
Regeneron’s investigational Ebola treatment shipping to Congo
Regeneron Pharmaceuticals’ investigational therapy that combines three fully-human monoclonal antibodies, REGN-EB3, is being shipped to the Democratic Republic of the Congo for use in the current Ebola virus infection outbreak. REGN-EB3 is one of several investigational therapeutics evaluated for use by a panel of independent scientific experts convened by the World Health Organization and selected for inclusion in a potential WHO-coordinated clinical trial. A new publication from the Journal of Infectious Diseases describes development of REGN-EB3 and efficacy results at different doses in animal models. In three preclinical studies, REGN-EB3 was seen to treat advanced Ebola virus disease and prevent mortality of infected non-human primates. Regeneron worked with the WHO and FDA to develop an Expanded Access Program protocol to support use of this treatment in the most recent outbreak in the Congo. Regeneron has secured various regulatory approvals, country approvals and import licenses and has hundreds of doses of REGN-EB3 ready to ship beyond the initial shipment currently in process. REGN-EB3 has received orphan drug designation from both the FDA and European Medicines Agency. It is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority.
Reata gets Euro orphan drug tag for kidney med candidate
Reata Pharmaceuticals announced that the European Commission, or EC, has granted orphan drug designation, based on the positive opinion from the Committee for Orphan Medicinal Products, or COMP, of the European Medicines Agency, or EMA, for bardoxolone methyl for the treatment of Alport syndrome. Alport syndrome is a rare, genetic form of chronic kidney disease, or CKD, caused by mutations in genes encoding type IV collagen, which is a major structural component of the glomerular basement membrane in the kidney. A majority of patients with Alport syndrome develop CKD and many progress to end-stage renal disease. In the Phase 2 portion of the Phase 2/3 CARDINAL study, bardoxolone significantly increased patients’ estimated glomerular filtration rate after 36 weeks of treatment
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