Bris­tol My­ers Squibb and blue­bird bio re­turn to FDA; Take­da gets a break­through nod

More than two months af­ter the FDA slammed Bris­tol My­ers Squibb and blue­bird bio with a suprise refuse-to-file on their  “ide-cell” CAR-T ther­a­py, the pair of de­vel­op­ers have re­sub­mit­ted their ap­pli­ca­tion.

The new ap­pli­ca­tion comes just in time to keep the con­tin­gent val­ue rights from the Cel­gene deal alive. The “ide-cell” was one of the ther­a­pies Bris­tol My­ers picked up in the mon­ster ac­qui­si­tion and one of three ther­a­pies that have to be ap­proved by ear­ly next year to gain every Cel­gene share­hold­er $9. Mizuho an­a­lyst Sal­im Syed es­ti­mat­ed that Bris­tol My­ers would have to re­sub­mit by Ju­ly 31 — to­mor­row — to get an FDA de­ci­sion on time.

The FDA ini­tial­ly spurned the ap­pli­ca­tion over CMC is­sues. Such is­sues have dogged blue­bird for years, de­lay­ing ther­a­pies that have oth­er­wise looked promis­ing in the clin­ic, in­clud­ing their gene ther­a­py for sick­le cell ane­mia.

Take­da gets a break­through des­ig­na­tion in MDS

Take­da R&D chief Andy Plump has been ea­ger to bur­nish the com­pa­ny’s top drug prospects in the wake of the Shire ac­qui­si­tion and re­or­ga­ni­za­tion. And now he’s get­ting some help from the FDA.

The agency has stamped their “break­through ther­a­py des­ig­na­tion” on pevonedi­s­tat for high­er-risk myelodys­plas­tic syn­dromes, or HR-MDS. This is a NEDD8-ac­ti­vat­ing en­zyme in­hibitor and if it works as planned, will be the first drug for HR-MDS to come along in quite some time.

The agency was won over by Take­da’s Phase II da­ta on the drug.

“High­er-risk MDS is as­so­ci­at­ed with poor prog­no­sis, di­min­ished qual­i­ty of life and a high­er chance of trans­for­ma­tion to acute myeloid leukemia, an­oth­er ag­gres­sive can­cer. The com­bi­na­tion of pevonedi­s­tat and azac­i­ti­dine is a promis­ing ther­a­peu­tic ap­proach with the po­ten­tial to be the first nov­el treat­ment ad­vance­ment for high­er-risk MDS in more than 10 years,” said Christo­pher Arendt, who heads Take­da’s On­col­o­gy Ther­a­peu­tic Area Unit.

An­oth­er pri­or­i­ty re­view for an Ebo­la an­ti­body 

A day af­ter Re­gen­eron an­nounced a near­ly $350 mil­lion con­tract from BAR­DA to sup­ply the gov­ern­ment with their Ebo­la an­ti­body cock­tail, Ridge­back Bio­ther­a­peu­tics an­nounced that the FDA had ac­cept­ed an ap­pli­ca­tion for ap­proval for their Ebo­la an­ti­body and giv­en it pri­or­i­ty re­view.

Ridge­back’s an­ti­body — known as mAb114, an­su­vimab or sim­ply “the NIH an­ti­body” — and Re­gen­eron’s an­ti­body cock­tail were the two drugs that proved ef­fec­tive in last year’s NIH ebo­la tri­al, beat­ing out the ZMapp an­ti­body and the Gilead an­tivi­ral remde­sivir. Un­like Re­gen­eron, Ridge­back did not de­vel­op the an­ti­body but rather li­censed it from the NIH, which had de­vel­oped it in part­ner­ship with the In­sti­tute for Bio­med­ical Re­search in Switzer­land.

mAb114 re­ceived FDA break­through des­ig­na­tion last year. Ridge­back did not dis­close a PDU­FA date. Re­gen­eron is ex­pect­ing a de­ci­sion on their an­ti­body by Oc­to­ber 25.

Geno­cea an­nounces “pos­i­tive” can­cer vac­cine re­sults, stock falls

Neoanti­gen biotech Geno­cea an­nounced what it called “pos­i­tive pre­lim­i­nary re­sults” from its lead can­cer vac­cine, but in­vestors were ev­i­dent­ly unim­pressed, drop­ping the com­pa­ny’s stock $GN­CA 34%, from $4.97 to $3.28.

The com­pa­ny re­port­ed that three of five pa­tients had a RE­CIST re­sponse — an im­prove­ment in a score for sol­id tu­mors — in their Phase I/IIa study. Pa­tients were giv­en check­point in­hibitors plus the neoanti­gen vac­cine. Geno­cea said they saw an “ac­cel­er­a­tion of shrink­age be­yond that” seen in check­point in­hibitors alone, but the com­pa­ny did not in­clude a con­trol group for the tri­al, so it’s dif­fi­cult to draw ma­jor con­clu­sions.

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.

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.

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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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.

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.

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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