Two years af­ter Newron $NWRN came up with a mixed set of Phase IIa da­ta for its an­tipsy­chot­ic eve­namide, the FDA is halt­ing their move in­to piv­otal work while the biotech ex­plores the im­pli­ca­tions of some new safe­ty con­cerns re­gard­ing the drug.

Ravi Anand Newron

Those con­cerns in­volve sig­nals spot­ted in a new­ly com­plet­ed rat study — which were not ex­plained — as well as “CNS events at high­er dos­es in dogs, and the po­ten­tial im­pli­ca­tion of these find­ings for pa­tients.” Reg­u­la­tors now want to see some new an­i­mal and hu­man stud­ies done to ad­dress the safe­ty is­sue be­fore they launch Phase II/III.

Back when I last cov­ered the drug in ear­ly 2017, the CMO was quite con­fi­dent that Newron was on to some­thing. The mean change for the Pos­i­tive and Neg­a­tive Syn­drome Scale (PANSS) score over­all was -5.1 for the add-on com­pared to -3.7 for the place­bo, which Newron chief med­ical of­fi­cer Ravi Anand de­scribed as “very close” to sta­tis­ti­cal sig­nif­i­cance. If you just looked at the pos­i­tive side of the scale, he added, the re­sults were sta­tis­ti­cal­ly sig­nif­i­cant.

That’s bor­der­line, but the Ital­ian biotech clear­ly felt it was worth pur­su­ing.

Anand now is in con­tact with the FDA to work out the next round of safe­ty work to clear up the is­sues, with the com­pa­ny “con­fi­dent that they can be ad­dressed sat­is­fac­to­ri­ly.”

Its shares were knocked down 12% Tues­day morn­ing.

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin and UBS in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.

Vertex has clinched a priority review for the all-important cystic fibrosis triple that will blaze the trail for treating a large group of patients unhelped by its current drugs.

FDA regulators have set a PDUFA date of March 19, 2020, just a year after the Boston biotech posted positive Phase III results showing that people with two F508del mutations experienced statistically significant improvements in lung function after a 4-week regimen of VX-445, tezacaftor and ivacaftor. After reviewing 24-week data among patients with one F508del mutation and one minimal function mutation — and thoroughly comparing the VX-445 triple with another combo featuring VX-659 on scores like safety, drug-drug interactions, and photosensitivity — Vertex ultimately went with VX-445.