GSK/Tesaro PARP in­hibitor Ze­ju­la wins an­oth­er speedy re­view

Months af­ter part­ner Zai Lab $ZLAB scored an ac­cel­er­at­ed re­view for PARP ther­a­py Ze­ju­la in Chi­na, GSK has se­cured pri­or­i­ty US re­view for the drug, known chem­i­cal­ly as ni­ra­parib, to ex­pand the use of the drug in pa­tients with ad­vanced ovar­i­an can­cer.

GSK $GSK, which shelled out just over $5 bil­lion to swal­low Ze­ju­la-de­vel­op­er Tesaro, said the FDA is ex­pect­ed to make its de­ci­sion by Oc­to­ber 24.

The drug was sub­mit­ted for re­view in pa­tients with ad­vanced ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer pa­tients with or with­out BR­CA mu­ta­tions who have been treat­ed with three or more pri­or chemother­a­py reg­i­mens based on da­ta from the QUADRA tri­al, which showed 13 (28%) of 47 pa­tients in the pri­ma­ry ef­fi­ca­cy pop­u­la­tion achieved an over­all re­sponse — the best re­sponse record­ed from the start of the treat­ment un­til the dis­ease pro­gress­es — as per RE­CIST (Re­sponse Eval­u­a­tion Cri­te­ria in Sol­id Tu­mors) method­ol­o­gy.

Mary Lynne Hed­ley

“The re­sults of the QUADRA study demon­strate that ZE­JU­LA is ac­tive as a late-line treat­ment for pa­tients be­yond those with BR­CA mu­ta­tions,” said Tesaro’s COO Mary Lynne Hed­ley in a state­ment.

Ovar­i­an can­cer is the most com­mon cause of gy­nae­co­log­i­cal can­cer death in the Unit­ed States. Al­though most pa­tients with ad­vanced ovar­i­an can­cer re­spond to ini­tial ther­a­py, 70% will re­lapse and ul­ti­mate­ly suc­cumb to their dis­ease. Late-line treat­ment op­tions for pa­tients with ovar­i­an can­cer are few, with the pro­por­tion of pa­tients achiev­ing an over­all re­sponse typ­i­cal­ly less than 10%, and me­di­an over­all sur­vival af­ter third-line ther­a­py of 5–9 months, re­searchers not­ed in Lancet On­col­o­gy.

The drug, which com­petes against As­traZeneca’s $AZN Lyn­parza as well as Clo­vis’ $CLVS Rubra­ca, is al­ready ap­proved as a first­line main­te­nance treat­ment of women with re­cur­rent ovar­i­an, fal­lop­i­an tube, or pri­ma­ry peri­toneal can­cer when their can­cer comes back.

The once-dai­ly Ze­ju­la is cur­rent­ly be­ing eval­u­at­ed in three piv­otal tri­als, in­clud­ing a late-stage tri­al in pa­tients with first-line ovar­i­an can­cer (the PRI­MA tri­al), a Phase III tri­al for the treat­ment of pa­tients with germline BR­CA-mu­tat­ed, metasta­t­ic breast can­cer (the BRA­VO tri­al), and a reg­is­tra­tional Phase II treat­ment tri­al in pa­tients with ovar­i­an can­cer (the QUADRA tri­al). Part­ner J&J $JNJ is test­ing the use of the drug in prostate can­cer. Mean­while, a num­ber of com­bi­na­tion tri­als are al­so un­der­way, in­clud­ing test­ing the use of Ze­ju­la and Mer­ck’s $MRK key­stone check­point in­hibitor Keytru­da in breast and ovar­i­an can­cer.

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. 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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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.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.

Ver­tex deal for Scot­land — no deal for Eng­land

Cystic fibrosis (CF) drug maker Vertex Pharmaceuticals — which is still locked in negotiation with NHS England to endorse the use of its medicines — has successfully negotiated a deal with Scottish authorities.

A month ago, the Scottish Medicines Consortium spurned two of the company’s medicines — Orkambi and Symkevi — citing uncertainty over their long-term efficacy in relation to their cost.

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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