Researchers at the University of Chicago have demonstrated that by transplanting mice with skin cells expressing a cocaine-metabolizing enzyme, they were able to protect the animals from cocaine overdoses, and prevent drug-seeking behavior and relapse in addicted mice. Read More
Regen Biopharma Inc., of San Diego, said its researchers have identified a series of small-molecule drugs that inhibit NR2F6 and also activate human immune cells ex vivo. Evidence provided by studies suggest that NR2F6 represses the body's immune response against tumors, and inhibiting NR2F6 may lead to enhanced immune response against cancerous tumor cells, the company said, and – although preliminary – the findings are encouraging, since the antagonists are behaving as Regen hoped and expected. Read More
Acadia Pharmaceuticals Inc., of San Diego, said the FDA completed a postmarketing review and reaffirmed the risk-benefit profile of Parkinson's disease psychosis drug Nuplazid (pimavanserin). The agency found no new or unexpected safety risks associated with the drug in its analysis, which was conducted following reports of a number of deaths and other serious adverse events in patients taking the drug. Read More
Titan Pharmaceuticals Inc., of South San Francisco, said it priced an underwritten public offering with expected total gross proceeds of $9.5 million. The company's lead product is Probuphine, a long-acting formulation of buprenorphine for the long-term maintenance treatment of opioid dependence. Read More
HAMBURG, Germany – U.K. firm Cellesce Ltd., based in Cardiff, Wales, will be collaborating during the next 12 months with the U.K. National Physical Laboratory to document the company's full range of colorectal cancer organoids, which could contribute to the development of an international imaging standard for organoid analysis. Read More
PERTH, Australia – Australian stem cell therapy company Mesoblast Ltd. is on its way to filing a BLA for its allogeneic mesenchymal precursor cell therapy, after it showed strong survival rates in children with acute steroid-refractory graft-vs.-host disease (aGVHD) at six months. Read More
Much attention has been devoted to how the gut influences the brain, traditionally via secreted hormones and, more recently, via the microbiome. Now, it turns out that during all that time, a direct neuronal connection using the classical neurotransmitter glutamate from the intestines to brain has been overlooked. Read More
With Blueprint Medicines Corp. due to start screening patients soon for the phase II Pathfinder trial that will test avapritinib in systemic mastocytosis (SM), investors are weighing the chances of several players in the disease and mulling how success might most rightly be judged. Read More
DUBLIN – Spark Therapeutics Inc. is set to gain a European approval for Luxturna (voretigene neparvovec-rzyl), its gene therapy for treating retinal dystrophy arising from biallelic RPE65 mutations, following a positive vote Friday from the EMA's Committee on Human Medicinal Products (CHMP). Formal approval from the European Commission should follow in the coming months, which would trigger a $25 million milestone payment from Novartis AG and the start of what could be a tortuous series of reimbursement negotiations with the national health authorities of individual European Union member states. Read More
Shares of Gtx Inc. (NASDAQ:GTXI) dropped 92.3 percent to $1.80 on Friday after a phase II trial found two doses of its sole clinical candidate, a selective androgen receptor modulator (SARM), failed to separate from placebo in helping postmenopausal women reduce stress urinary incontinence (SUI). The candidate, enobosarm, has been evaluated in 27 trials to date. Barring a compelling finding in the full data, the program will likely be wound down, Executive Chairman Rob Wills told BioWorld. The Gtx team will now turn its attention to identifying a lead from its preclinical selective androgen receptor degrader (SARD) program, he said. Read More
