Early but strong data suggest "we might be onto something" with self-injected APL-2 for the blood disease paroxysmal nocturnal hemoglobinuria (PNH), Apellis Pharmaceuticals Inc. CEO Cedric Francois told BioWorld Today, as the company disclosed positive interim results from two ongoing phase Ib open-label, dose-escalation trials.

With efficacy data thus far sans toxicity, "our dose escalation continues," Bluebird Bio Inc.'s chief medical officer, David Davidson, told BioWorld Today as onlookers cheered early success with the anti-BCMA chimeric antigen receptor (CAR) T-cell candidate BB2121 for relapsed/refractory multiple myeloma (MM), in development with Celgene Corp.

After toxicology problems led RNA interference (RNAi) specialist Arrowhead Pharmaceuticals Inc. to give up on its clinical pipeline that deploys the EX1 delivery method, worries turned to whether the subcutaneous platform might run into similar woes, though the firm's chief operating officer, Bruce Given, said that "we see bigger safety margins and it just doesn't look at all like the EX1 program."

In July, aldoxorubicin “seemed dead and buried,” Cytrx Corp.’s chief medical officer, Daniel Levitt, acknowledged to BioWorld Today, but sifting updated data from the phase III trial in soft-tissue sarcoma (STS) turned up enough promise that the company plans to file an NDA late next year.

Last week’s phase III blowup of Eli Lilly and Co.’s solanezumab (sola) in Alzheimer’s disease (AD) struck up a familiar dirge in the failure-strewn space, and set experts to debating – again, still – the amyloid beta hypothesis, as companies that have chosen to pursue other routes continued to laud their approaches and ever-ambitious big pharma snatched up another player in the space.

Privately held Arog Pharmaceuticals Inc. won orphan drug status in Europe for crenolanib to treat acute myeloid leukemia (AML) and soft-tissue sarcoma. An orally available benzimidazole, crenolanib is a type I kinase inhibitor that goes after the signaling of wild-type and mutant isoforms of class III receptor tyrosine kinases FLT3 and platelet-derived growth factor receptor (PDGFR) alpha/beta.

Acorda Therapeutics Inc.'s phase III fizzle in post-stroke walking difficulties (PSWD) of potassium channel blocker Ampyra (dalfampridine) didn't put much of a dent in the stock, but it made for the second development halt this year and put pressure on the rest of the pipeline, especially late-stage CVT-301, an inhaled levodopa for "off" episodes in Parkinson's disease.

As partner Mylan NV makes ready to launch in Canada the erectile dysfunction (ED) drug-device combo Vitaros (alprostadil cream) – approved there as well as certain countries in Europe, Latin America and the Middle East – Apricus Biosciences Inc. heard from the FDA what's necessary in the U.S. to resolve issues in the complete response letter (CRL) dating back to 2008.

Amesh Adalja, of the University of Pittsburgh School of Medicine, told BioWorld Today that, although too early to tell for sure about toxicity, it’s “an encouraging sign” that Nabriva Therapeutics AG has chosen to develop lefamulin, a new antibiotic in the mushroom-derived pleuromutilin class that has shown a clean profile in phase II trials against skin infections.