For decades, the biopharma industry has struggled to develop "game-changing" therapies in heart failure, according to Tassos Gianakakos, CEO of Myokardia Inc. Read More

Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn., said it completed randomization of it phase II/III trial testing trigriluzole (previously known as BHV-4157) in 141 patients with spinocerebellar ataxia. The trial compares trigriluzole, a glutamate modulator, to placebo, measuring the change from baseline in patient scores on the Scale for the Assessment and Rating of Ataxia after eight weeks of treatment. Biohaven expects top-line data from the trial in the fourth quarter. Read More

Amicus Therapeutics Inc., of Cranbury, N.J., reported net revenue of $7.2 million, primarily from commercial sales of its Fabry disease therapy Galafold (migalastat), which launched in May 2016. Net loss for the quarter was $48.1 million, or 34 cents per share, compared to a net loss of $51.1 million, or 40 cents per share, for the second quarter 2016. The company ended the quarter with about $227.2 million in cash and cash equivalents. Its shares (NASDAQ:FOLD) gained 67 cents to close at $13.90. Read More

Zynerba Pharmaceuticals Inc. shares (NASDAQ:ZYNE) plunged more than 55 percent Monday to $6.67 on news that its lead candidate, the cannabidiol (CBD) gel ZYN-002, missed the primary endpoint of a phase II trial, failing to help adult epilepsy patients with focal seizures achieve a statistically significant reduction in seizures during the 12-week treatment period of the trial, called Star 1. Read More

Talk turned to the finer points of phase IIb data from the trial called ROYAL-1 – in particular, success with regard to high-sensitivity C-reactive protein (hsCRP) – after Wall Street put a dent in Gemphire Therapeutics Inc., despite the fact that gemcabene achieved its primary endpoint of cholesterol lowering in the study. Read More

HONG KONG – Glaxosmithkline plc confirmed it will be shutting down operations at its Neuroscience R&D Centre in Shanghai. The U.K.-based pharmaceutical giant will move key programs to the global R&D hub in the U.S. "following a portfolio review and prioritization," as outlined in a statement. Read More

HONG KONG – The launch of the South African Health Products Regulatory Authority (SAHPRA) will have long-term implications for South Africa's pharmaceutical industry, but it will take time for domestic drugmakers to see tangible benefits. Read More

Antriabio Inc., of Louisville, Colo., acquired the rights to Berkeley, Calif.-based Activesite Pharmaceuticals Inc.'s plasma kallikrein inhibitor (PKI) program so it can develop, file, manufacture, market and sell products for diabetic macular edema and other conditions. It will pay Activesite $750,000 up front and various milestone payments ranging from $1 million to $10 million. It could also provide Activesite with royalty payments of 2 percent of sales for any products that use the PKI program. Read More

Nuo Therapeutics Inc., of Gaithersburg, Md., cited "prevailing market conditions" in deciding to withdraw its registration for a public offering on Nasdaq. The company had filed to sell 2.1 million shares at a price of between $4.50 and $5.50 per share after executing a 1-for-2.5 reverse stock split. Prior to changing its name in 2014, the company was known as Cytomedix Inc. Read More

The FDA finalized 48 product-specific guidances on how to develop generic drugs that are therapeutically equivalent to certain reference-listed drugs. The guidances are intended to help sponsors identify the most appropriate methodology for developing generics and generating the evidence needed to support approval. Although the ultimate goal is to facilitate the availability of generics, some of the drugs referenced by the final guidances already have a number of approved generics. For instance, one of the guidances addresses diabetes drug acarbose (Precose, Bayer Healthcare Pharmaceuticals Inc.), which was first approved 20 years ago. Since 2008, the FDA has approved seven generic versions of the drug, according to the Orange Book. Read More