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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of face plants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.

Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.

The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.