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.”

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Af­ter the field left her be­hind, a sci­en­tist-turned-in­vestor gets her first R&D job; Nous­com finds new lead­er­ship for its can­cer vac­cines

Before she boarded the plane, Cristina Ghenoiu spent most school day afternoons at Bucharest’s National Museum of Natural History, studying endangered animals Romanian scientists had brought back from around the country, or the world. The communist government sponsored a wide range of programs for kids so both parents could work. Her sister danced; Ghenoiu fell in love with biology.

And she was good, at least good enough to win several national awards and then, at 15, a spot as Romania’s representative to an international school in Canada that accepted about one person per country.

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Brian Stuglik (file photo)

Turn­ing fo­cus to clin­i­cal work, Ve­rastem ax­es 31 jobs, scales back can­cer drug pro­mo­tion af­ter dis­ap­point­ing sales

Months after taking the helm at Verastem Oncology, Brian Stuglik has a plan to take the biotech in a “new strategic direction” — but not before some layoffs.

Left out of an upbeat press release spelling out its clinical plans, and buried below news of a $100 million private placement in an SEC filing, is a planned restructuring that will claim 31 jobs. Alongside some other cost-saving measures, Verastem expects to cut expenses down by $70 million to $80 million per year.

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Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Sage con­firms sus­pen­sion of 2 de­pres­sion tri­als af­ter PhI­II flop; Es­pe­ri­on fol­lows up maid­en ap­proval with com­bo OK

→ In the wake of a flop in the crucial Phase III MOUNTAIN study, Sage Therapeutics confirmed in its quarterly update that it’s suspended enrollment in two other pivotal trials for the oral depression drug SAGE-217 (or zuranolone) as it awaits guidance from the FDA. While REDWOOD (measuring relapse) and RAINFOREST (for patients with both major depressive disorder and insomnia) are on hold pending amendments, though, the open-label SHORELINE has completed enrollment. CEO Jeff Jonas remained tight-lipped about what specific tweaks they are considering for the program, reiterating only there have been issues with compliance and room for a higher dose.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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