John Reed (Endpoints JPM19, Jeff Rumans)

An­a­lysts poured cold wa­ter, but FDA gives a fast-pass for Sanofi's new Pompe dis­ease drug

An­a­lysts didn’t love the re­sults from Sanofi’s piv­otal Pompe dis­ease study, but the FDA has ev­i­dent­ly seen enough promise to give it an ex­pe­dit­ed look.

On Wednes­day, the French gi­ant an­nounced that the agency has giv­en pri­or­i­ty re­view for their Pompe dis­ease drug aval­glu­cosi­dase al­fa, hand­ing them a PDU­FA da­ta for May 18 and a chance to get a fur­ther head start on Am­i­cus and the ri­val drug now in Phase III.

Sanofi has had a toe hold in Pompe dis­ease since it bought out Gen­zyme in 2011. The Cam­bridge biotech de­vel­oped My­ozyme and Lu­mizyme, the first and so far on­ly treat­ments for the rare lyso­so­mal dis­or­der. Caused by a miss­ing or dys­func­tion­al sug­ar-pro­cess­ing en­zyme known as GAA, the dis­ease leads to a buildup of sug­ars in cells through­out the body, of­ten caus­ing ir­re­versible mus­cle dam­age.

My­ozyme and Lu­mizyme pro­vides an ar­ti­fi­cial copy of the en­zyme, slow­ing but not stop­ping dam­age.

Sanofi de­signed the new en­zyme to more eas­i­ly squeeze in­side cells, in the­o­ry al­low­ing it to process more sug­ar and fur­ther slow dam­age.

In a 100-pa­tient Phase III study, though, the new en­zyme failed to prove it was su­pe­ri­or to the orig­i­nal drug in im­prov­ing res­pi­ra­to­ry func­tion. It came close, with a P val­ue of .06, and still man­aged to meet the pri­ma­ry end­point of non-in­fe­ri­or­i­ty. But with oth­er op­tions on the hori­zon, the re­sults left an­a­lysts unim­pressed.

“To­day’s an­nounce­ment was in line with our ex­pec­ta­tions that…. Ph.3 da­ta would be un­der­whelm­ing,” SVB Leerink’s Joseph Schwartz wrote in June, when the re­sults came out. Am­i­cus, he said, would be “like­ly the best-in-class next-gen­er­a­tion” en­zyme re­place­ment drug.

An­a­lysts pro­ject­ed the drug would be ap­proved but that it would not be­come a huge mon­ey­mak­er. Cowen’s Steve Scala pro­ject­ed a €100 mil­lion in rev­enue in 2025, com­pared to €1.25 bil­lion for Lu­mizyme.

Of course, much of their skep­ti­cism comes from the of­ten nec­es­sary but crude art of cross-tri­al com­par­isons, with Schwartz com­par­ing open-la­bel Am­i­cus re­sults to Sanofi’s place­bo-con­trolled da­ta. Am­i­cus will get a chance to prove that, un­like Sanofi, they can achieve su­pe­ri­or­i­ty over Lu­mizyme when they re­lease their Phase III tri­al re­sults.

Those are ex­pect­ed to come in the first quar­ter of the year. John Reed and the R&D team over at Sanofi will like­ly be watch­ing close­ly.

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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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)

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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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.