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.”

In the study, pa­tients on Baven­cio lived, on av­er­age, 7.1 months longer than those giv­en best sup­port­ive care, rep­re­sent­ing a 31% re­duc­tion in the risk of death.

“With me­di­an over­all sur­vival of more than 21 months mea­sured from ran­dom­iza­tion, the longest over­all sur­vival in a Phase III tri­al in ad­vanced urothe­lial car­ci­no­ma, the JAVELIN Blad­der 100 reg­i­men with avelum­ab as a first-line switch main­te­nance treat­ment has the po­ten­tial to be­come a new stan­dard of care based on its proven abil­i­ty to re­in­force the ben­e­fit (re­sponse or sta­ble dis­ease) of in­duc­tion chemother­a­py and ex­tend the lives of pa­tients with this dev­as­tat­ing dis­ease,” Gri­vas said in a state­ment.

Al­though chemother­a­py tends to in­duce high re­sponse rates, most pa­tients ul­ti­mate­ly ex­pe­ri­ence dis­ease pro­gres­sion with­in nine months, the com­pa­nies not­ed. Among those whose can­cer has al­ready metas­ta­sized at di­ag­no­sis, on­ly 5% would live longer than five years.

It’s now up to the com­mer­cial and med­ical af­fairs teams at Pfiz­er and Mer­ck KGaA to re­al­ize that fun­da­men­tal shift in stan­dard of care they en­vi­sion.

The drug was al­ready sanc­tioned, un­der ac­cel­er­at­ed ap­proval, for use in pa­tients fol­low­ing dis­ease pro­gres­sion — based on tu­mor re­sponse and du­ra­tion of re­sponse back in 2017.

The new OK — one of the few bright spots in Baven­cio’s check­ered de­vel­op­ment his­to­ry — al­so high­lights the sort of speed de­vel­op­ers can ex­pect un­der the FDA’s re­al-time on­col­o­gy re­view (RTOR) pi­lot pro­gram. The part­ners com­plet­ed their sBLA sub­mis­sion in ear­ly April, al­though the three-month re­view is still rel­a­tive­ly long com­pared to what Mer­ck saw with Keytru­da in a cer­tain sub­type of col­orec­tal can­cer.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Vi­da Ven­tures co-leads Dyne's $115M megaround for next-gen oli­go ther­a­pies aimed square­ly at mus­cles

Dyne Therapeutics started out last April with a modest $50 million to mine targeted muscle disease therapies from its in-house conjugate technology. The biotech has now convinced more investors that it’s got gems on its hands, closing $115 million in fresh financing to push its next-gen oligonucleotide drugs into the clinic.

Vida Ventures and Surveyor Capital led the round, joined by a group of other new backers including Wellington Management Company, Logos Capital and Franklin Templeton.

Eli Lil­ly teams with Pieris on HER2+ tu­mors; Op­di­vo + Yer­voy best chemo in mesothe­lioma

Despite the FDA putting a partial clinical hold on its lead program only a few weeks ago, Boston-based Pieris Pharmaceuticals is plowing forward with a new collaboration.

Pieris will work with Eli Lilly to further advance studies on PRS-343, a 4-1BB/HER2 bispecific for HER2-positive tumors, in combination with the latter’s ramucirumab and paclitaxel for the second-line treatment of patients with HER2-positive gastric cancer in a single-arm, Phase II study.

In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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