No­vo Nordisk qui­et­ly nabs a rare 'break­through' sta­tus in NASH for its cash cow

Ear­li­er this month, the small French biotech In­ven­ti­va tout­ed them­selves as hav­ing won the first FDA break­through des­ig­na­tion in NASH since In­ter­cept grabbed the first one back in 2015, be­fore the dis­ease had be­come one of biotech’s hottest ar­eas. Un­be­knownst to them, though, a much larg­er Eu­ro­pean ri­val had al­ready land­ed the sta­tus months pri­or.

No­vo Nordisk, the Dan­ish meta­bol­ic gi­ant, dis­closed in their Q3 re­port Fri­day that back in Au­gust, the FDA gave them break­through sta­tus for semaglu­tide, their block­buster di­a­betes drug, in NASH, or non-al­co­holic steato­hep­ati­tis. The des­ig­na­tion sets No­vo up as a lead­ing con­tender in a new wave of com­pa­nies de­vel­op­ing treat­ments for a silent and wide­spread dis­ease that, de­spite a se­ries of set­backs, is still viewed by many as a huge po­ten­tial mar­ket.

As with In­ven­ti­va, the sta­tus came on the heels of pos­i­tive re­sults in a Phase II tri­al. Back in their Q1 — some com­pa­nies have a habit of bury­ing neg­a­tive news deep in SEC fil­ings, No­vo seems to have a habit of bury­ing good news — the Big Phar­ma re­vealed da­ta from a 230-per­son study, show­ing that those who re­ceived any of 3 dos­es of the drug were sig­nif­i­cant­ly more like­ly than a place­bo arm to see their NASH re­solve with­out their fi­bro­sis wors­en­ing. On the high dose, 33 out of 56 pa­tients achieved res­o­lu­tion, com­pared to 10 out of 58 on place­bo.

No­vo said to­day they plan to launch a Phase III tri­al next year. It’s a stage where oth­er big NASH drugs have gone to die, al­though not all had shown clear re­sults in Phase II. Gilead didn’t run a place­bo-con­trolled Phase II study pri­or to their Phase III stum­bles, and Gen­fit’s drug had failed a Phase II be­fore it failed its Phase III.

At the mo­ment, it’s not clear what bar No­vo — or In­ven­ti­va or NGM — would have to clear in a Phase III tri­al to gain ap­proval. Re­ly­ing on their read­ing of guid­ance from the FDA, In­ter­cept filed for ap­proval af­ter a Phase III in which they showed the drug could im­prove fi­bro­sis but didn’t show it could re­solve NASH. The FDA, how­ev­er, re­ject­ed the pitch.

A NASH ap­proval would rep­re­sent an­oth­er ex­pan­sion on a GLP-1 drug that has al­ready reaped bil­lions for No­vo Nordisk, tak­ing in $1.64 bil­lion last year in sales. The com­pa­ny is al­so plan­ning to ex­pend a pri­or­i­ty re­view vouch­er on an ap­pli­ca­tion in obe­si­ty.

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

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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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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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

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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FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

Bax­ter con­tin­ues on-shoring push with $50M In­di­ana ex­pan­sion

It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing.

Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN.

Eu­ro­pean Union aims to es­tab­lish patent workaround in case of emer­gen­cies while try­ing to strength­en its own IP

The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says.

Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.