As the amyotrophic lateral sclerosis (ALS) community hailed work published last week identifying a new gene linked to the disease, Cytokinetics Inc. and Tokyo-based Astellas Pharma Inc. strengthened their existing skeletal muscle activator deal with a focus on ALS, including an option to Astellas for rights to tirasemtiv, currently being tested in the phase III VITALITY-ALS study that could lead to approval applications in the U.S. and Europe.

With reimbursement issues in the rearview mirror and efforts from an amped-up sales force finally starting to yield momentum after a slower-than-expected launch for hyperphosphatemia drug Auryxia (ferric citrate), Keryx Biopharmaceuticals Inc.'s second-quarter earnings should have been a resounding message of reassurance.

As the amyotrophic lateral sclerosis (ALS) community hailed work published this week identifying a new gene linked to the disease, Cytokinetics Inc. and Astellas Pharma Inc. strengthened their existing skeletal muscle activator deal with a focus on ALS, including an option to Astellas for rights to tirasemtiv, currently being tested in the phase III VITALITY-ALS study that could lead to approval applications in the U.S. and Europe.

Positive data early next year from its pivotal study of M207, a zolmitriptan-coated microneedle patch for treating migraine, could go a long way to helping Zosano Pharma Corp. regain investor confidence after losing both its big pharma partnerships last year and watching shares plummet to a fraction of its January 2015 IPO price.

In another promising sign for the gene therapy space, shares of Audentes Therapeutics Inc. received an encouraging welcome on Wall Street Wednesday after the San Francisco-based firm priced a $75 million IPO, offering 5 million shares at $15 each, the midpoint of its proposed range.

The drug that had so far mounted a strong defense for Roche Holdings AG's CD20-targeting franchise against the encroachment of biosimilars stumbled in a late-stage trial reported over the weekend.

With the bulk of Galena Biopharma Inc.'s value riding on cancer vaccine Neuvax (nelipepimut-S), the firm's shares predictably plunged to a new 52-week low Wednesday as an independent data monitoring committee (IDMC) recommended the PRESENT phase III study in breast cancer be stopped for futility following a planned interim analysis.

Long-awaited data from a confirmatory phase III study aimed at convincing regulators of the efficacy of twice-rejected Kiacta (eprodisate) in AA amyloidosis, ended up dealing a crushing blow to Bellus Health Inc., with top-line results Monday showing the five-year, event-driven trial missed the primary endpoint of slowing renal function decline in patients with the orphan disease.

As investors await top-line phase III data later this summer for AM-101, its lead program in tinnitus, Auris Medical Holding AG continued advancing its other late-stage candidate, AM-111, launching as planned a second phase III study in hearing loss. Both indications represent potential first-mover opportunities for the Swiss-based biopharma.