SAN FRANCISCO – Calling "unconscionable" the way some Alzheimer's disease (AD) trials have gone about enrollment and pointing to the misguided approach as a reason many experiments failed, University of Oxford neuroscience professor Simon Lovestone told a packed room at the BIO International Convention that studies have tested patients when "it's already too late."

But for outcomes in "a patient or two," Alexion Pharmaceuticals Inc.'s top-line results from the trial called Regain with Soliris (eculizumab) in refractory generalized myasthenia gravis (gMG) "would have been very different," CEO David Hallal said, calling the data package available so far "compelling and certainly supportive of the potential efficacy."

After having its abstract on a phase II trial in triple-negative breast cancer (TNBC) named one of the "best" slated for roll-out at the American Society of Clinical Oncology (ASCO) meeting that had just begun in Chicago, Immunomedics Inc. was informed that the presentation was being dropped, apparently because officials believed the dataset had been made public earlier, violating the premier cancer meeting's embargo policy.

CEO Stanley Erck told BioWorld Today that Novavax Inc. is shifting focus to generate "much more broadly neutralizing antibodies which should – we have to demonstrate this in humans – make for a better flu vaccine," as the firm sets aside its virus-like particle (VLP)-based seasonal influenza candidate in favor of pursuing a nanoparticle-based prospect.

Pfizer Inc.'s $5.2 billion takeover of Anacor Pharmaceuticals Inc. to grab crisaborole for atopic dermatitis (AD, or eczema) didn't knock anyone out of their chairs but triggered some chin-scratching speculation about whether the merger-and-acquisition (M&A) waters have begun to roil again.

With its $1.5 billion buyout of Celator Pharmaceuticals Inc. to take ownership of Vyxeos for high-risk (secondary) acute myeloid leukemia (AML), Jazz Pharmaceuticals plc is not done exploring the blood cancer space, said CEO Bruce Cozadd.

After presenting full results from the phase III trial with Portola Pharmaceuticals Inc.’s oral betrixaban for prophylaxis of venous thromboembolism (VTE) in France, study steering committee leader Michael Gibson told BioWorld Today that the company is “beginning to chip away at the chip of the iceberg with respect to the data and will continue to work closely with [the FDA] to explore all their questions.” South San Francisco-based Portola plans to submit a new drug application to the FDA in the second half of this year, and pursue approval in Europe as well.

Positive phase IIb data with MIN-101 in schizophrenia paves the way to pivotal testing and gives patients hope for their negative symptoms, Minerva Neurosciences Inc. spokesman William Boni told BioWorld Today. “I don’t want to use any words like ‘breakthrough,’” he said, because there needs to be more testing done, “but there is no effective treatment for negative symptoms at this point.”

Eight months after its $38 million series E round brought aboard new strategic and crossover investors, Selecta Biosciences Inc. made manifest what the move often signals, filing for an IPO that would raise as much as $75 million.

With a pivotal phase II/III trial to start "in the next several weeks," Oxigene Inc. CEO William Schwieterman told BioWorld Today that the just-published results on CA4P (fosbretabulin) paired with approved cancer therapy Avastin (bevacizumab, Roche AG) in recurrent ovarian cancer "are potentially huge for the field" of vascular-targeted therapy in solid tumors. "While [CA4P] is old, we have a new plan and a new story," he said.