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.

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Overview
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.