In­tel­liphar­ma­ceu­tics shares crushed as FDA ex­perts snub pain drug; Mon­cef Slaoui joins Mod­er­na board

→ Af­ter get­ting chas­tised by reg­u­la­tors in an in­ter­nal FDA re­view of Toron­to-based In­tel­liphar­ma­ceu­tics$IP­CI new pain drug Rex­ista, a pan­el of out­side ex­perts joined in, vot­ing to rec­om­mend a re­jec­tion of the ther­a­py. Billed as a new ver­sion of oxy with new fea­tures which the biotech claimed would de­ter abuse, the pan­elists vot­ed 22 to 1 against an OK. And all of them agreed that the com­pa­ny nev­er of­fered the da­ta that would be need­ed to gain an abuse-de­ter­rent note on the la­bel, if it were to be ap­proved. The com­pa­ny’s shares cratered, drop­ping 45%.

→ Rad­nor, PA-based Com­plexa has raised a big $62 mil­lion round de­signed to fu­el a pair of mid-stage tri­als for its lead ther­a­py, CXA-10. The drug will be test­ed in fo­cal seg­men­tal glomeru­loscle­ro­sis (FS­GS), an or­phan dis­ease af­fect­ing the kid­ney, and pul­monary ar­te­r­i­al hy­per­ten­sion (PAH), a rare pul­monary dis­ease. New En­ter­prise As­so­ci­ates and Pfiz­er Ven­ture In­vest­ments led the round with help from Ed­mond de Roth­schild In­vest­ment Part­ners, HBM Health­care In­vest­ments and ex­ist­ing in­vestors in­clud­ing JAF­CO, which led the Com­pa­ny’s Se­ries B fi­nanc­ing in 2014.

→ Fre­mont, CA-based Quark Phar­ma­ceu­ti­cals is re­port­ing that their Phase II study for the siR­NA drug QPI-1002 came through with pos­i­tive da­ta for re­duc­ing the rate of acute kid­ney in­jury fol­low­ing car­diac surgery. The study en­rolled 341 sub­jects. “Quark is de­vel­op­ing QPI-1002 in a va­ri­ety of in­di­ca­tions as­so­ci­at­ed with is­chemia-reper­fu­sion re­nal in­jury,” stat­ed Shai Er­lich, CMO and pres­i­dent of Quark’s US op­er­a­tions. “We are ex­cit­ed about the QRK-209 study re­sults which re­in­force the pos­i­tive ef­fect of QPI-1002 as pre­vi­ous­ly re­port­ed in a ran­dom­ized, dou­ble-blind, place­bo-con­trolled mul­ti­cen­ter Phase 2 study (QRK-006b; NCT00802347) where QPI-1002 was shown to re­duce the in­ci­dence and sever­i­ty of de­layed graft func­tion (DGF) in pa­tients un­der­go­ing re­nal trans­plan­ta­tion.”

→ Mon­cef Slaoui, the for­mer R&D and vac­cines chief at Glax­o­SmithK­line, is join­ing the board at the mR­NA com­pa­ny Mod­er­na. Slaoui is an en­thu­si­as­tic cham­pi­on of pi­o­neer­ing drug R&D. And he says he’s look­ing “for­ward to work­ing with the team to nav­i­gate this un­chart­ed ter­ri­to­ry, and sup­port the strate­gic plan­ning al­ready un­der­way as Mod­er­na works to de­liv­er mR­NA med­i­cines and vac­cines to ad­dress se­ri­ous un­met med­ical needs in the U.S. and glob­al­ly.”

Sarep­ta was stunned by the re­jec­tion of Vyondys 53. Now it's stun­ning every­one with a sur­prise ac­cel­er­at­ed ap­proval

Sarepta has a friend in the FDA after all. Four months after the agency determined that it would be wrong to give Sarepta an accelerated approval for their Duchenne MD drug golodirsen, regulators have executed a stunning about face and offered the biotech a quick green light in any case.

It was the agency that first put out the news late Thursday, announcing that Duchenne MD patients with a mutation amenable to exon 53 skipping will now have their first targeted treatment: Vyondys 53, or golodirsen. Having secured the OK via a dispute resolution mechanism, the biotech said the new drug has been priced on par with their only other marketed drug, Exondys 51 — which for an average patient costs about $300,000 per year, but since pricing is based on weight, that sticker price can even cross $1 million.

Sarepta shares $SRPT surged 23% after-market to $124.

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UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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Paul Biondi (File photo)

Paul Biondi's track record at Bris­tol-My­ers cov­ered bil­lions in deals of every shape and size. Here's the com­plete break­down

Paul Biondi was never afraid to bet big during his stint as business development chief at Bristol-Myers Squibb. And while the gambles didn’t all pay out, by any means, his roster of pacts illustrates the broad ambitions the pharma giant has had over the last 5 years — capped by the $74 billion Celgene buyout.

On Thursday, we learned that Biondi had exited the company. And Chris Dokomajilar at DealForma came up with the complete breakdown on every buyout, licensing pact and product purchase Bristol-Myers forged during his tenure in charge of the BD team at one of the busiest companies in biopharma.

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This image shows a lab technician measuring the zone of inhibition during an antibiotic sensitivity test, 1972. The zone of inhibition is measured and compared to a standard in order to determine if an antibiotic is effective in treating the bacterial infection. (Gilda Jones/CDC via Getty Images)

Bio­phar­ma has aban­doned an­tibi­ot­ic de­vel­op­ment. Here’s why we did, too.

Timing is Everything
When we launched Octagon Therapeutics in late 2017, I was convinced that the time was right for a new antibiotic discovery venture. The company was founded on impressive academic pedigree and the management team had known each other for years. Our first program was based on a compelling approach to targeting central metabolism in the most dangerous bacterial pathogens. We had already shown a high level of efficacy in animal infection models and knew our drug was safe in humans.

Shehnaaz Suli­man dives back in­to Alzheimer's at Alec­tor; Pyx­is re­cruits Spring­Works founder Lara Sul­li­van as CEO

Amid Shehnaaz Suliman’s lengthy resume it could be easy to miss her stint leading early-stage Alzheimer’s R&D at Genentech, where she oversaw a program for the ill-fated crenezumab and initiated one of the first prevention studies around the devastating neurodegenerative disease. But it is this experience that she — after thinking long and hard about her next career move over the past months — will be leaning heavily on as the first president and COO of Alector.

Paul Biondi (File photo)

Bris­tol-My­er­s' strat­e­gy, BD chief Paul Bion­di ex­it­ed the com­pa­ny — just ahead of the $74B Cel­gene deal close

Paul Biondi, who orchestrated billions of dollars in deals for Bristol-Myers Squibb over the 5 years he’s run their business development team, has exited the company. Biondi left last month, according to a company spokesperson, in pursuit of another — unspecified — external opportunity.

After 17 years with Bristol-Myers Squibb, Paul Biondi, Head of Strategy and Business Development, decided to leave the company to pursue an external opportunity. The company wishes him well in his new endeavors. Bristol-Myers Squibb  is actively searching for Paul’s successor, and will make an announcement, as appropriate.

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PhII fail­ure in rare neu­rode­gen­er­a­tive dis­ease? No mat­ter, Bio­gen will mo­tor on in Alzheimer's

Biogen’s fierce focus on disorders of the brain has hit another roadblock.

On Friday, the US drugmaker — which recently resurrected its amyloid-targeting Alzheimer’s drug, aducanumab — said its anti-tau drug, gosuranemab, failed a mid-stage study in patients with progressive supranuclear palsy (PSP), a rare brain disorder that results from deterioration of brain cells that control movement and thought.

A USP­TO le­gal ad­vis­er is off con­tro­ver­sial Gilead HIV case af­ter ac­tivists al­lege tweets show bias

Last week, a top legal adviser in the US Patent and Trademark Office working on the high-profile Gilead HIV PrEP case tweeted at Sen Bernie Sanders (I-VT) “What proof????” and then at activists “Do facts even matter to you?”

Now, STAT reports, she’s off of the case.

Activists in the coalition PrEP4All filed a petition to the USPTO on December 9 asking longtime senior legal advisor Mary Till be removed from the Gilead case, saying her tweets showed a bias toward Gilead. PrEP4All requested earlier this month the agency reject Gilead’s three-year patent extension for TAF (tenofovir alafenamide), a component of one of the HIV prevention regimens often referred to as PrEP. They allege the pharma giant delayed developing the drug in order to “game” the system and hold off generics.

What's next for Sarep­ta? A third DMD ap­proval, an­a­lysts pre­dict

What Sarepta wants, Sarepta usually gets.

In dramatic fashion on Thursday, the approval of Vyondys 53 — to treat a subset of Duchenne muscular dystrophy (DMD) patients — was unveiled, four months after the FDA’s initial rejection. With two drugs now approved on the basis of ~1% expression of dystrophin — the missing protein that causes DMD — Sarepta is in a prime position to take its third DMD drug to the regulator, analysts said, predicting healthier odds of success.

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