Puma wins a big round in fight for ner­a­tinib OK as ex­perts of­fer a cru­cial thumbs up

Puma Biotech­nol­o­gy $PBYI won a cru­cial vote in fa­vor of its ex­per­i­men­tal breast can­cer drug ner­a­tinib, with a ma­jor­i­ty of out­side ex­perts of­fer­ing the FDA a thumbs-up vote in fa­vor of ap­proval as an ex­tend­ed ad­ju­vant treat­ment.

On the key ques­tion of whether the risk/ben­e­fit pro­file of ner­a­tinib war­rant­ed an ap­proval, the pan­el vot­ed twelve to four in fa­vor of the drug. But the sup­port came with plen­ty of caveats about the nar­row ben­e­fit the da­ta demon­strat­ed and ques­tions about the drug’s of­ten harsh im­pact on pa­tients. Sev­er­al pan­el mem­bers called on the FDA to nar­row the ap­proval to a small­er set of pa­tients most like­ly to ben­e­fit.

“There’s no ex­cla­ma­tion point af­ter my ‘yes’,” said An­drew Sei­d­man, an on­col­o­gist at Memo­r­i­al Sloan Ket­ter­ing. “It’s just a yes.” He added that he was hap­py that the drug looks durable and the sta­tis­ti­cal analy­ses ap­plied to the da­ta giv­en the sig­nif­i­cant changes to the study de­sign. “I do think that physi­cians will se­lect pa­tients very se­lec­tive­ly for us­ing this,” he not­ed, echo­ing a con­cern that the ap­proval the com­pa­ny was seek­ing was too broad giv­en the da­ta avail­able.

If it makes it to the mar­ket, those com­ments may come back to haunt Puma, to some ex­tent. But to­day, Puma ex­ecs had plen­ty to cel­e­brate. Its shares im­me­di­ate­ly shot up more than 25% af­ter trad­ing re­sumed in the ear­ly af­ter­noon, adding to the big spike that oc­curred when the FDA’s writ­ten re­view hit on Mon­day.

Puma has been at the cen­ter of a rov­ing street fight be­tween in­vestors go­ing long and short on the stock. But there were none of the fire­works pre­dict­ed dur­ing the re­view on Wednes­day.

The bears’ ar­gu­ment that the drug trig­gered such se­ri­ous ad­verse events that it would be spurned by the FDA ran in­to the ex­perts’ sup­port for the mar­gin­al ben­e­fit the drug demon­strat­ed, im­prov­ing dis­ease-free sur­vival in HER2 pos­i­tive pa­tients by 2.3%. About 94.2% of pa­tients in the ner­a­tinib arm were alive with­out their dis­ease pro­gress­ing at two years, com­pared with 91.9% in the con­trol arm.

One of the biggest is­sues high­light­ed in the FDA’s writ­ten re­view of the drug was that agency of­fi­cials had ad­vised Puma not to file based on the da­ta from their piv­otal tri­al. That didn’t seem to weigh heav­i­ly on the pan­el, though Puma won’t know for sure if it’s out of the woods un­til the FDA pro­vides a for­mal de­ci­sion.

Pan­el votes are not bind­ing.

There were se­ri­ous ad­verse events in the study, pri­mar­i­ly re­flect­ed in high lev­els of se­ri­ous, though not life threat­en­ing, cas­es of di­ar­rhea. The agency’s re­view, though, did not raise ma­jor red flags for Puma.

“Most tox­i­c­i­ties are non­se­ri­ous and re­versible,” said the FDA’s Aman­da Walk­er, “but the true mag­ni­tude of ben­e­fit re­mains un­cer­tain.”

The ben­e­fit was un­cer­tain be­cause of the changes Puma made to the reg­is­tra­tional study af­ter tak­ing over from Pfiz­er. In­ves­ti­ga­tors for Puma went back to re­con­sent pa­tients for a longer pe­ri­od of re­view and did that with 75% of the pa­tients, but da­ta were still miss­ing on 754 pa­tients.

But why would a physi­cian choose this drug com­pared to what’s al­ready avail­able, asked one of the pan­elists.

Puma con­sul­tant Jose Bisel­ga not­ed the “rel­a­tive risk re­duc­tion of 34% com­pared to what is avail­able cur­rent­ly.” Would I want the op­tion to use it? “To this I would an­swer ‘yes.’ I would go a long way to get this done.”

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.