Top sto­ries of the week: Thumbs up/Thumbs down

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Cel­gene taps its new biotech co-star


This was the per­fect week to an­swer a ques­tion I’ve been think­ing about re­gard­ing Cel­gene. The Big Biotech, which has spe­cial­ized in lin­ing up rich deals for star col­lab­o­ra­tors, has set­tled on Jounce as its lat­est pick for up-and-com­ing biotech with the most po­ten­tial. Cel­gene has been stitch­ing to­geth­er a whole pipeline of im­muno-on­col­o­gy drugs through its BD team, head­ed by George Golumbes­ki. Our chart on all its pacts, and the $3.2 bil­lion it spent on deals over the past two years, proved to be the most pop­u­lar part of the week. Those deals al­so il­lus­trate how in­dus­try part­ner­ships dri­ve new pro­grams. Im­por­tant point: It’s not over at Cel­gene.

What were they think­ing at Zaf­gen?


There aren’t many biotechs which han­dle set­backs well. Zaf­gen, though, has be­come a case study in clue­less­ness. Af­ter in­sist­ing for months that they had com­pelling piv­otal da­ta and an ef­fec­tive plan to get its obe­si­ty drug back on track af­ter the death of two pa­tients, Zaf­gen had to ac­knowl­edge this week that the drug is dead. But on­ly af­ter tout­ing pre­clin­i­cal da­ta for their new, un­known lead drug. By the end of this week, the com­pa­ny’s shares are trad­ing at half cash, not what you would call a ring­ing en­dorse­ment for the new plan or the way they han­dled them­selves in the cri­sis. There’s no ques­tion about the com­pa­ny’s sin­cer­i­ty—these are smart, ex­pe­ri­enced and com­mit­ted play­ers who lost their way–but as an­a­lysts turn their backs on Zaf­gen, that may not mat­ter much any­way. This is a teach­able mo­ment in cri­sis PR. Don’t waste it.

  The Scan­gos lega­cy is­sue


George Scan­gos ar­rived on the scene at Bio­gen six years ago as its sav­ior. He leaves as the com­pa­ny looks for a new mes­si­ah. But what a ride. He and his new team pushed Tec­fidera through, and made it a block­buster in the MS mar­ket. In the mid­dle of a biotech boom, Bio­gen could boast ma­jor league sta­tus along­side Gilead and Cel­gene. Then the sales weak­ened, pipeline set­backs tar­nished their im­age, an over­haul was in or­der and the com­pa­ny is now in line for a new makeover, it might even be ac­quired. Maybe Scan­gos stayed a year too long and trust­ed in home run swings too much. But no one can take away the win­ning years.

The bio­phar­ma mi­gra­tion to the hubs con­tin­ues


We’ve been gath­er­ing some fresh ev­i­dence this week on just how pop­u­lar the two big biotech hubs are in the U.S. Mer­ck has been prep­ping a big move in­to a new cam­pus in the Bay Area as more re­searchers are be­ing pulled in­to Boston/Cam­bridge. And Ger­many’s Mer­ck KGaA un­veiled its blue­print for a new cam­pus in Burling­ton, MA. Big bio­phar­ma R&D loves the hubs, as com­pa­nies look to put them­selves in the main­stream of all the best sci­ence. It doesn’t hurt that these two big hubs al­ready host most of the start­up com­pa­nies in biotech.

More un­cer­tain­ty in a new round of re­or­ga­ni­za­tions


But of course there’s a down side to every big trend. The lat­est re­or­ga­ni­za­tions com­ing hand in hand with down­siz­ing some op­er­a­tions, as Mer­ck made clear. As­traZeneca has al­so been culling its U.S. ranks in Mary­land and Mass­a­chu­setts. The biggest re­search groups be­gan to over­haul R&D about 7 or 8 years ago now. Mer­ck, Pfiz­er, As­traZeneca, Sanofi, Roche and oth­ers all took a big swing at their or­ga­ni­za­tions, look­ing to find some re­al ef­fi­cien­cies from the multi­bil­lion dol­lar op­er­a­tions com­mit­ted to drug de­vel­op­ment. These lat­est re­or­ga­ni­za­tions un­der­score the fact that any­one look­ing for job se­cu­ri­ty might want to find an­oth­er line of work, or a dif­fer­ent place to do it. If it’s not about down­siz­ing, there’s al­ways a re­jig in core fo­cus ar­eas. Still, few in­dus­tries of­fer the kind of short-term op­por­tu­ni­ties as bio­phar­ma R&D; ask any se­r­i­al en­tre­pre­neur in the field.

Brex­it blues


Per­haps it was an un­in­tend­ed con­se­quence, but the gov­ern­ment reshuf­fle fol­low­ing the Brex­it vote in the UK cost the in­dus­try its ded­i­cat­ed min­is­ter for the life sci­ences. George Free­man heads to big­ger and bet­ter things at a time the in­dus­try faces a cri­sis of con­fi­dence it can ill af­ford. Leav­ing the EU means wav­ing off EU re­search cash and tal­ent from the con­ti­nent, some­thing the Gold­en Tri­an­gle can miss bad­ly as it looks to es­tab­lish their life sci rep­u­ta­tion. On the oth­er hand, the UK con­tin­gent of biotech has a scrap­py un­der­dog side to it that will see it through to bet­ter days. Stay calm and car­ry on. It’s worked be­fore.

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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