Neu­ro­crine ex­ecs shrug off a PhII set­back for Tourette syn­drome

Neu­ro­crine was forced to con­cede that its lead drug – al­ready on track at the FDA with an ap­pli­ca­tion for tar­dive dysk­i­ne­sia — just flunked a Phase II tri­al in Tourette syn­drome.

The VMAT2 in­hibitor im­proved symp­toms among the pa­tients tak­ing the drug, but failed to hit the pri­ma­ry end­point for the change-from-base­line in the Yale Glob­al Tic Sever­i­ty Scale at week 8. The biotech, though, is keep­ing the hard da­ta un­der wraps for now.

Don’t look for the com­pa­ny $NBIX to throw in the tow­el. Their state­ment im­plied that they’re go­ing to take what they learned from this set­back and ap­ply it again in an­oth­er study.

Their stock slid a bit more than 5% in af­ter-hours trad­ing, an ini­tial in­di­ca­tion that in­vestors didn’t see the set­back as cat­a­stroph­ic.

“At present, we are not dis­clos­ing spe­cif­ic de­tails of this study in or­der to avoid the po­ten­tial in­tro­duc­tion of as­sess­ment bias in the on­go­ing Phase II T-Force GREEN study of pe­di­atric Tourette pa­tients,” not­ed CEO Kevin Gor­man. “We look for­ward to the pe­di­atric study read­out next quar­ter and our sub­se­quent dis­cus­sion with the FDA on our plans for Phase III de­vel­op­ment of In­grez­za in Tourette syn­drome.”

A to­tal of 124 pa­tients were en­rolled in 32 cen­ters.

Just a few days ago the FDA an­nounced that they were scrap­ping plans for a pan­el re­view of the drug for tar­dive dysk­i­ne­sia, a move that sev­er­al an­a­lysts took as a pos­i­tive in­di­ca­tion of its re­cep­tion at the agency. The PDU­FA date for that in­di­ca­tion is set for April 11.

Chris O’Brien, the chief med­ical of­fi­cer of Neu­ro­crine, said:

Through this ini­tial place­bo-con­trolled study we gained valu­able in­sights in­to the con­duct of clin­i­cal tri­als in Tourette pa­tients, in­clud­ing pa­tient iden­ti­fi­ca­tion meth­ods and ap­pro­pri­ate study in­clu­sion/ex­clu­sion cri­te­ria. As we have done with our pre­vi­ous ear­ly Phase II stud­ies in both en­dometrio­sis and tar­dive dysk­i­ne­sia, we will ap­ply these new in­sights to fu­ture Tourette stud­ies.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

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Doug Williams, departing Codiak BioSciences CEO

Co­di­ak files for Chap­ter 11 bank­rupt­cy as most ex­ec­u­tives head for the ex­it

Codiak BioSciences has filed for Chapter 11 bankruptcy, spelling an end to the employment of most executives, including founder Doug Williams, as the biotech says it “expects to consummate a sale.”

The eight-year journey at Codiak is nearing an end with Williams; CFO Linda Bain; medical chief David Mauro; scientific head Sriram Sathyanarayanan; legal and compliance chief Yalonda Howze; and SVP of HR Nicole Barna all packing up their bags in the first few days of April. Chief technology officer Konstantin Konstantinov will stay.

Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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Max Colao, OncoVerity CEO

Tiny mul­ti­omics biotech se­cures for­mer J&J drug, new ex­ecs and new fi­nanc­ing

A new spinout from Belgium’s argenx seeks to give new life to a candidate once in-licensed to J&J.

OncoVerity announced Monday afternoon that it had in-licensed cusatuzumab from argenx. The biotech also appointed two new C-suite executives and secured a $30 million Series A round.

CEO Max Colao joins OncoVerity after working as Aurinia’s commercial chief, and stints at Alexion and Amgen. New CMO Clay Smith has been associate chief of the University of Colorado’s hematology division.

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Sharon Mates, Intra-Cellular Therapies CEO

In­tra-Cel­lu­lar Ther­a­pies gets a boost on PhI­II de­pres­sion drug da­ta

Biotech Intra-Cellular Therapies touting new data Tuesday for its drug designed to treat major depressive disorder.

The biotech shared results from its Study 403 investigating lumateperone, or Caplyta, as a monotherapy to treat major depressive episodes. The study looked at major depressive disorder (MDD) and bipolar depression with “mixed features.” The trial had three populations: participants with both MDD and bipolar depression, patients with only MDD and patients with only bipolar depression.