Puma Biotech­nol­o­gy shares tank af­ter CHMP looks ready to hit a red light on ner­a­tinib

Six months af­ter Puma Biotech­nol­o­gy won an FDA OK for its can­cer ther­a­py ner­a­tinib, the EMA looks like its prepar­ing to throw up a big road­block in­to Eu­rope over con­cerns its reg­u­la­tors have about the drug. And the news im­me­di­ate­ly trig­gered an­oth­er sharp turn for the biotech’s stock $PBYI, which plunged 27% in af­ter-mar­ket trad­ing.

An­a­lysts have been fret­ting over the Eu­ro­pean de­ci­sion for months now, not­ing that a sci­en­tif­ic ad­vi­so­ry group meet­ing called by the EMA sug­gest­ed that Puma faced some broad chal­lenges that ex­tend­ed be­yond the ev­i­dence of se­vere di­ar­rhea linked to the drug.

Alan Auer­bach, Puma CEO

To­day Puma an­nounced that the CHMP no­ti­fied the com­pa­ny of a neg­a­tive trend vote, which loose­ly trans­lat­ed means that they are high­ly un­like­ly to get a green light in Eu­rope any­time soon to sell ner­a­tinib for the ex­tend­ed ad­ju­vant treat­ment of ear­ly stage HER2-pos­i­tive breast can­cer.

The com­pa­ny’s state­ment not­ed:

CHMP in­di­cat­ed that, in its opin­ion, the ben­e­fit risk as­sess­ment is neg­a­tive as the study re­sults are based on ev­i­dence from a sin­gle piv­otal tri­al and the 2- and 5-year in­va­sive dis­ease free sur­vival (iDFS) ben­e­fits ob­served to-date may lack suf­fi­cient clin­i­cal rel­e­vance.

That will like­ly make Puma’s jour­ney in search of an ap­proval in Eu­rope much, much longer, while it’s free to sell the drug in the US.

Leerink’s Michael Schmidt not­ed:

CHMP’s opin­ion was based on the re­sults from both the Phase III Ex­teNET tri­al in ex­tend­ed ad­ju­vant ear­ly stage HER2-pos­i­tive breast can­cer and the Phase II CON­TROL tri­al in ex­tend­ed ad­ju­vant ear­ly stage HER2-pos­i­tive breast can­cer ac­cord­ing to PBYI. In con­trast to that, the US FDA On­co­log­ic Drugs Ad­vi­so­ry Com­mit­tee (ODAC) vot­ed 12:4 in fa­vor of FDA ap­proval in­di­cat­ing that the risk-ben­e­fit pro­file of Ner­l­ynx was fa­vor­able. The FDA sub­se­quent­ly ap­proved Ner­l­ynx with a broad la­bel in the US which is not lim­it­ed to cer­tain pa­tient sub-pop­u­la­tions.

The de­bate over grade 3 di­ar­rhea seen in clin­i­cal tri­als ex­cit­ed con­sid­er­able short in­ter­est in the stock, but the FDA’s ad­vis­ers bare­ly hes­i­tat­ed in urg­ing an ap­proval, with a track record that has long fa­vored adding new op­tions for on­col­o­gists and patents when­ev­er pos­si­ble.

Puma’s shares — al­ways volatile in the back-and-forth de­bate over the drug’s side ef­fects and its ef­fi­ca­cy — dived 27% in­stant­ly.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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Judge al­lows ex­pert tes­ti­mo­ny in GSK tri­al al­leg­ing Zan­tac link to can­cer

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.