FDA Ad­Com rec­om­mends GSK’s an­ti-BC­MA drug for ap­proval 12-0 de­spite sear­ing in-house re­view — does it mat­ter?

The FDA may have ripped Glax­o­SmithK­line for the safe­ty of its an­ti-BC­MA drug, but that didn’t stop an ad­vi­so­ry com­mit­tee from vot­ing 12-0 to rec­om­mend ap­proval for the mul­ti­ple myelo­ma treat­ment.

The de­ci­sion is in line with the com­mit­tee’s long-run­ning pref­er­ence to ap­prove can­cer drugs that show enough ef­fi­ca­cy and not over­whelm­ing safe­ty con­cerns, with the goal of giv­ing on­col­o­gists as many tools to work with as pos­si­ble as they treat in­di­vid­ual pa­tients. Yet while it sig­naled a strong like­li­hood of FDA ap­proval — and boost­ed GSK’s stock by a nifty 1.6% — the vote still leaves open the ques­tion of how use­ful the drug will ul­ti­mate­ly be and, ac­cord­ing­ly, how well it will sell for a com­pa­ny try­ing to claw back in­to the can­cer ther­a­peu­tics busi­ness.

The case for the ther­a­py, an an­ti­body drug con­ju­gate called be­lan­tam­ab, is sim­ple: It had a 31% re­sponse rate in mul­ti­ple myelo­ma pa­tients who had failed mul­ti­ple oth­er lines of ther­a­pies, in­clud­ing the J&J block­buster Darza­lex.

The case against the drug is al­so sim­ple: The FDA con­clud­ed that GSK, run­ning sin­gle-arm tri­als, failed to prove it helps pa­tients live longer.  And the down­sides were sig­nif­i­cant: Near­ly a fifth of pa­tients in one tri­al saw their vi­sion de­cline to lev­els at which they would not legal­ly be al­lowed to dri­ve in one state. One pa­tient on the ap­pli­ca­tion dose and two pa­tients on the high­er dose be­came legal­ly blind. “[I]t is not clear that the ben­e­fits out­weigh the risk of oc­u­lar tox­i­c­i­ty,” the FDA con­clud­ed in its re­view last week.

All that may not be enough to dra­mat­i­cal­ly cur­tail the drug’s com­mer­cial po­ten­tial if it weren’t for the deep well of ex­per­i­men­tal ther­a­pies that have shown far bet­ter re­sults in the clin­ic and are now near­ing ap­proval.

GSK will like­ly have to com­pete with the an­ti-BC­MA CAR-T ther­a­py from J&J-part­nered Leg­end Biotech that re­cent­ly showed a 100% re­sponse rate and a 69% com­plete re­sponse rate in pa­tients who had un­der­gone a me­di­an of five lines of ther­a­py. Blue­bird and Bris­tol My­ers Squibb have faced em­bar­rass­ing de­lays in bring­ing their so-called “ide-cel” CAR-T ther­a­py to mar­ket, but their da­ta are pret­ty clear: a 71% re­sponse rate and a 31% com­plete re­sponse rate in Phase II.

Then there’s the bis­pecifics from Re­gen­eron, Am­gen and Bris­tol My­ers, which are fur­ther be­hind but have been greet­ed with promise. In a dose-es­ca­la­tion study un­veiled at ASH in De­cem­ber, Re­gen­eron’s REGN5458 in­duced re­spons­es in 4 of 7 pa­tients who had al­ready re­ceived 7 lines of ther­a­py.

Be­lan­tam­ab was meant to be a cen­tral part of GSK’s re­turn to can­cer un­der CEO Em­ma Walm­s­ley, and it may soon get an ap­proval. How much it will mean for pa­tients or for a drug com­pa­ny look­ing to re-ce­ment its name re­mains un­clear.

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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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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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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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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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.