Puma wins big FDA OK for ner­a­tinib af­ter run­ning the gamut with in­vestors

Short in­vestors loved to hate Puma Biotech­nol­o­gy, tak­ing the com­pa­ny $PBYI to task for the side ef­fects that ham­pered its lead can­cer drug ner­a­tinib. But the FDA had no trou­ble sanc­tion­ing the prod­uct for breast can­cer, ap­prov­ing it to­day for use in pre­vent­ing re­cur­rence of the dis­ease af­ter the biotech gained a lop­sided vote in its fa­vor among out­side agency ex­perts.

The FDA ap­proved ner­a­tinib — to be sold as Ner­l­ynx — for pre­vent­ing the re­turn of breast can­cer af­ter ther­a­py in­clud­ing trastuzum­ab. And the em­pha­sis in the OK was on adding op­tions for doc­tors and pa­tients.

“HER2-pos­i­tive breast can­cers are ag­gres­sive tu­mors and can spread to oth­er parts of the body, mak­ing ad­ju­vant ther­a­py an im­por­tant part of the treat­ment plan,” said Richard Paz­dur, M.D., di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence and act­ing di­rec­tor of the Of­fice of Hema­tol­ogy and On­col­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “Now, these pa­tients have an op­tion af­ter ini­tial treat­ment that may help keep the can­cer from com­ing back.”

There are some caveats in the la­bel. Con­cerned about the high rate of Grade 2 and Grade 3 di­ar­rhea in the clin­i­cal stud­ies, reg­u­la­tors are telling physi­cians to stop us­ing the drug if their pa­tient ex­pe­ri­ences Grade 4 di­ar­rhea or Grade 2 af­ter a max­i­mum dose re­duc­tion. But Michael Schmidt at Leerink sees the plus­es as well in a broad la­bel:

The prod­uct la­bel looks broad, not re­strict­ing use to spe­cif­ic pa­tient pop­u­la­tions (e.g., hor­mone-re­cep­tor [HR] pos­i­tive pa­tients) and there is no black box warn­ing. The pre­scrib­ing in­for­ma­tion con­tains fair­ly de­tailed in­for­ma­tion about the use of an­tidiar­rheal pro­phy­lax­is mea­sures, pro­vid­ing spe­cif­ic in­for­ma­tion on how to man­age the drug’s main side ef­fect.

Puma says it isn’t re­leas­ing the price yet and has no time­line on that. Its stock price still had some up­side left af­ter the OK, though, with shares surg­ing 7.5% on a com­pa­ny with a $3.2 bil­lion mar­ket cap. Now that the ap­proval has come through, look for lots more chat­ter about Puma as a po­ten­tial takeover tar­get — even though M&A has large­ly been MIA this year in biotech.

Alan Auer­bach

The FDA ex­perts raised a va­ri­ety of con­cerns in their re­view of ner­a­tinib, mak­ing some points that could hin­der the drug’s mar­ket po­ten­tial now that Puma CEO Alan Auer­bach has a green light to sell it. Crit­i­cal to its suc­cess is a bet­ter un­der­stand­ing of its prop­er role in treat­ing breast can­cer, and the lim­its of its ef­fec­tive­ness.

To get here, Puma had to over­come reg­u­la­tors’ ob­jec­tions in the spring of 2016 to fil­ing the drug with the da­ta that were avail­able, cit­ing a prob­lem­at­ic ap­proach to de­ci­pher­ing the re­sults. The shorts were al­so fix­at­ed on a high rate of grade 3 di­ar­rhea in the tri­als, but ex­perts hard­ly paid any at­ten­tion to that, hap­py to trade off the po­ten­tial for man­age­able side ef­fects for a chance of adding a new ar­row to their quiver of ther­a­pies.

“There’s no ex­cla­ma­tion point af­ter my ‘yes’,” said An­drew Sei­d­man, an on­col­o­gist at Memo­r­i­al Sloan Ket­ter­ing af­ter the FDA vote. “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.

Puma’s po­si­tion has been height­ened af­ter an­a­lysts fa­vor­ably com­pared ner­a­tinib’s 34% re­duc­tion in the risk of re­cur­rence or death against a mar­gin­al im­prove­ment seen in a Roche com­bi­na­tion study of Per­je­ta and Her­ceptin. It takes a leap to do these kinds of cross-tri­al analy­ses that dri­ve drug de­vel­op­ers a tad crazy now and then, but Wall Street an­a­lysts were quick to give Puma a thumbs up, par­tic­u­lar­ly af­ter it had just hand­i­ly won the FDA pan­el vote.

Eval­u­atePhar­ma pegged 2022 sales at $1.25 bil­lion, mak­ing it one of the top 15 drugs in the in­dus­try pipeline at the be­gin­ning of this year.

George Yancopoulos (Regeneron)

Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

On a roll, Mer­ck blazes through a new seg­ment of the bio­mark­er trail

Merck has notched an approval for using Keytruda to treat a biomarker-based subset of first-line colorectal cancer patients with unresectable or metastatic tumors, as the pharma giant continues to find new niches for its blockbuster PD-1 star.

The OK is significant in a number of ways. Not only does it build on an accelerated approval for all tumors characterized as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR); it also marks the first single treatment for colorectal cancer that doesn’t contain chemotherapy.

Pfiz­er shares surge on pos­i­tive im­pact of their mR­NA Covid-19 vac­cine — part­nered with BioN­Tech — in an ear­ly-stage study

Pfizer and their partners at the mRNA specialist BioNTech have published the first glimpse of biomarker data from an early-stage study spotlighting the “robust immunogenicity” triggered by their Covid-19 vaccine, which is one of the leaders in the race to vanquish the global pandemic.

Researchers selected 45 healthy volunteers 18-55 years of age for the study. They were randomized to receive 2 doses, separated by 21 days, of 10 µg, 30 µg, or 100 µg of BNT162b1, “a lipid nanoparticle-formulated, nucleoside-modified, mRNA vaccine that encodes trimerized SARS-CoV-2 spike glycoprotein RBD.” Their responses were compared against the effect of a natural, presumably protective defense offered by a regular infection.

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Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi preps an­oth­er round of lay­offs as new ex­ecs look to slim down the glob­al phar­ma in an on­go­ing re­or­ga­ni­za­tion — re­port

Sanofi is reportedly once again sharpening up the budget axe as the pharma giant prepares to chop more jobs.

Reuters reports this morning that Sanofi has its sights set on cutting 1,680 jobs in Europe, where the unions have been combating rounds of cutbacks at the Paris-based player. Sanofi itself hasn’t said anything about these cuts, as the company looks to discuss a 3-year plan with staff representatives.

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Covid-19 roundup: Vac­cines will need to beat place­bo by 50% to qual­i­fy for FDA OK; UK tri­al drops Kale­tra

The FDA will set the bar for approving a Covid-19 vaccine at 50% efficacy, the Wall Street Journal reported, meaning any successful candidate will have to reduce the risk of coronavirus disease by at least half compared to placebo.

That requirement is part of guidance that the agency is set to release later today, laying out detailed criteria for vaccine developers — some of whom are eyeing an OK by the end of the year, in line with expectations at Operation Warp Speed.

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