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.

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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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

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Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.