Reg­u­la­tors or­der AB Sci­ence to halt tri­al work on PhI­II ALS drug, de­mand­ing proof it's cleaned up

Just weeks af­ter lay­ing out claims on pos­i­tive Phase III da­ta for their ALS drug ma­sitinib, French reg­u­la­to­ry au­thor­i­ties are drop­ping the ham­mer on AB Sci­ence, or­der­ing a halt to clin­i­cal re­search work on the drug un­til the biotech can prove it’s com­pli­ant with rules gov­ern­ing drug study con­duct.

In a re­lease out ear­ly Fri­day, the biotech said that the Agence Na­tionale de la Sécu­rité des Médica­ments is de­mand­ing an ex­ter­nal au­dit to clear their work on tri­al pro­ce­dures. AB Sci­ence says that the ANSM ze­roed in on a trou­bled study —AB06006 — in­volv­ing ma­sitinib in treat­ing mas­to­cy­to­sis. But those prob­lems, the biotech kicked back, all oc­curred be­tween 2009 and 2015, be­fore they put a new qual­i­ty con­trol sys­tem in place.

The biotech — a pub­licly trad­ed com­pa­ny in France — added that the sus­pen­sion of all tri­al work on the drug is on­ly tem­po­rary and they plan to get it all re­solved ex­pe­di­tious­ly, com­plet­ing the au­dit “in com­ing months.”

In ad­di­tion, AB’s lengthy state­ment not­ed that the sus­pen­sion of tri­al work is lim­it­ed to France. It won’t af­fect their ALS plans, they add, be­cause their late-stage tri­al oc­curred out­side of France and the au­dit will be com­plet­ed soon in any case.

The 16-year-old biotech, though, run by co-founder and CEO Alain Moussy, has a cred­i­bil­i­ty is­sue. Over the years AB Sci­ence has been known to put out cheery re­leas­es on da­ta and prospects on­ly to be forced to walk it all back. The EMA re­ject­ed its mar­ket­ing ap­pli­ca­tions on ma­sitinib for ad­vanced in­op­er­a­ble pan­cre­at­ic can­cer and gas­troin­testi­nal stro­mal tu­mors in 2014 and 2013, ham­mer­ing the com­pa­ny on poor tri­al de­sign, safe­ty as well as man­u­fac­tur­ing short­com­ings.

The tim­ing of the halt couldn’t have come at a worse mo­ment. In March re­searchers claimed the first ever suc­cess­ful Phase III study in ALS, with plans to de­tail the da­ta at an up­com­ing sci­en­tif­ic con­fer­ence. The drug was filed at the EMA last fall for ALS.

Just days ago Mit­subishi Tan­abe won the first new drug ap­proval at the FDA in more than 20 years, for Rad­i­ca­va, and on­ly af­ter they asked the com­pa­ny to file it for a mar­ket­ing OK. The last new ther­a­py, Ri­lutek (rilu­zole) from Sanofi, ar­rived in the US in the mid-90s, un­der­scor­ing just how dif­fi­cult this dis­ease has been to tack­le in the clin­ic.

AB Sci­ence’s trou­ble could work to the ad­van­tage of Cy­to­ki­net­ics, which is work­ing on a late-stage ALS drug called tirasem­tiv.

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

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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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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.

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.

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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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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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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.

News brief­ing: FDA re­quests new tri­al for Reata's Friedre­ich's atax­ia pro­gram; J&J's Trem­fya picks up ex­pand­ed la­bel in Eu­rope

Three months after Reata Pharmaceuticals suggested its Friedreich’s ataxia program omaveloxolone could be delayed, the company revealed that is indeed going to be the case.

Reata $RETA shares took a nosedive Wednesday after the biotech revealed that the FDA said supplemental data for its pivotal trial did not strengthen the case for approval. As a result, the drug is likely to need another study before the FDA takes up the case.