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.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Bris­tol My­ers Squibb fi­nal­ly gets in the front­line NSCLC game dom­i­nat­ed by Mer­ck, adding a sec­ond Op­di­vo/Yer­voy-based op­tion

Bristol Myers Squibb may be trailing Merck and Roche in the checkpoint race to treat frontline cases of non-small cell lung cancer, but as it does, it makes sure to bring its best feet forward.

Just days after scoring a landmark NSCLC approval for Opdivo and Yervoy alone for PD-L1 positive patients, the company said the FDA has also OK’d using the two agents with a limited course of chemo regardless of the biomarker status.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.