Not so crazy now: Biotech’s most close­ly watched uni­corn just pro­duced its first clear snap­shot of hu­man ef­fi­ca­cy da­ta

The ex­ec­u­tive team at Mod­er­na raised a cheer to­day af­ter pub­lish­ing their first ear­ly snap­shot of hu­man ef­fi­ca­cy da­ta that demon­strate their mes­sen­ger RNA tech works — at least on the first try.

Stéphane Ban­cel

The biotech test­ed their H10N8 flu vac­cine on a small group of 31 sub­jects, look­ing at their re­sponse in two dif­fer­ent mea­sures. All demon­strat­ed a suf­fi­cient im­mune re­sponse to fight off the virus in the first mea­sure, and all but 3 in the sec­ond, for a to­tal of 23 who re­ceived the vac­cine.  None of the 8 sub­jects who re­ceived a place­bo re­spond­ed.

Typ­i­cal­ly, ev­i­dence of Phase I suc­cess in a vac­cine study — which fol­lowed a num­ber of an­i­mal stud­ies — wouldn’t at­tract much at­ten­tion for your av­er­age biotech. But there’s noth­ing av­er­age about Mod­er­na. CEO Stéphane Ban­cel and his team have at­tract­ed $1.9 bil­lion to cre­ate a com­pa­ny that’s build­ing a pipeline around mes­sen­ger RNA, one of a hand­ful of play­ers look­ing to coax cells to pro­duce pro­teins to fight dis­ease. And that’s a mas­sive amount for a com­pa­ny that just showed hu­man proof of con­cept da­ta.

This vac­cine is not point­ed to­ward com­mer­cial­iza­tion, Ban­cel told me Thurs­day af­ter­noon. It’s a demon­stra­tion project. The goal was to start out with “low bi­ol­o­gy risk where we knew where the goal post was be­fore we start­ed the tri­al.”

Mod­er­na’s board has al­ready de­cid­ed not to seek an IPO this year, and Ban­cel adds that there’s no de­fin­i­tive plans set for 2018 ei­ther. What he would like to do, he says, is add new pieces to the pipeline — new ver­ti­cals to the three he’s al­ready out­lined — adding more da­ta on the po­ten­tial mR­NA has to cre­ate a new path­way on drug de­vel­op­ment for a wide ar­ray of dis­eases. Then, have some­thing sub­stan­tial that proves what they can do, he’ll set out to con­vince in­vestors.

The huge amount of cash raised to date and Mod­er­na’s plans to prove the prin­ci­ples be­hind its sci­ence — fo­cus­ing on vac­cines as the most like­ly ini­tial demon­stra­tion pro­gram that could prove that mR­NA can safe­ly work — has al­so trig­gered more than a lit­tle schaden­freude for a com­pa­ny that is of­ten billed as mys­te­ri­ous and lav­ish­ly fund­ed. STAT picked up on that, re­port­ing a bat­tery of most­ly anony­mous crit­i­cism about its tough work en­vi­ron­ment and lofty claims, lay­ing in­to the biotech at every turn.

It is still ear­ly days at Mod­er­na, which has to prove that it can de­vel­op mR­NA drugs for some of the more lu­cra­tive ther­a­pies than the fron­trun­ning vac­cines. That will take some time. But Mod­er­na’s been do­ing much more to out­line what pro­grams it has in the pipeline, and when it can dis­cuss more about the da­ta.

“I’m cau­tious­ly op­ti­mistic this could be a re­al­ly im­por­tant ad­di­tion to our vac­cine ar­se­nal,” Bar­ney Gra­ham, deputy di­rec­tor of the Vac­cine Re­search Cen­ter at the Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases, told the Wall Street Jour­nal. He added that RNA is un­sta­ble, leav­ing him ea­ger to see more ma­ture da­ta on how this works.

Ban­cel and his fel­low ex­ecs at Mod­er­na may have a long way to go, but they’re con­fi­dent that they took a big step along the jour­ney to­day. From now on, it’s all about clin­i­cal da­ta at Mod­er­na.

H10 mR­NA Im­muno­genic­i­ty in Hu­mans | Mol­e­c­u­lar Ther­a­py

Said Tal Zaks, Chief Med­ical Of­fi­cer of Mod­er­na:

The da­ta “pro­vide im­por­tant val­i­da­tion of our core mR­NA plat­form, as we con­tin­ue to ad­vance our de­vel­op­ment pipeline, tack­ling more com­plex vac­cines in­clud­ing per­son­al­ized can­cer vac­cines, and mov­ing our mR­NA ther­a­peu­tics in­to clin­i­cal de­vel­op­ment.”

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: 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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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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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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Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.

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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