Am­i­cus chief John Crow­ley is snubbed at the FDA on his lat­est pitch for quick drug re­view. He'll be back, but an­a­lyst sees big de­lay

John Crow­ley

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Am­i­cus CEO John Crow­ley’s lat­est pitch to put a new drug on a path­way to ac­cel­er­at­ed ap­proval has run in­to a hur­dle at the FDA. Crow­ley ac­knowl­edged in a state­ment out ear­ly Mon­day that a meet­ing with reg­u­la­tors failed to gen­er­ate much en­thu­si­asm for a quick re­view of AT-GAA (ATB200/AT2221) based on what they’ve seen so far in a Phase I/II tri­al.

But Crow­ley $FOLD, who re­cent­ly over­came the odds in win­ning a re­ver­sal of the FDA’s de­ci­sion to ini­tial­ly re­ject Galafold, isn’t about to stop now. The com­pa­ny says it plans to keep press­ing, just as it did ear­li­er this sum­mer af­ter the EMA al­so held up a stop sign to a con­di­tion­al ap­proval.

Am­i­cus Ther­a­peu­tics in­tends to con­tin­ue to gen­er­ate da­ta to sup­port fur­ther dis­cus­sions on a po­ten­tial path­way for ac­cel­er­at­ed cp­proval with the FDA in 2019, in­clud­ing:

  • Da­ta from up to 10 ad­di­tion­al ERT-switch pa­tients in a new Co­hort 4 as part of the on­go­ing Phase 1/2 study (da­ta ex­pect­ed in 2019)
  • Pre­sen­ta­tion of longer-term clin­i­cal da­ta out to 18-months for the 19 orig­i­nal Phase 1/2 pa­tients (da­ta ex­pect­ed in 2H 2018)
  • Com­ple­tion of a ret­ro­spec­tive nat­ur­al his­to­ry study in ap­prox­i­mate­ly 100 ERT-treat­ed Pompe pa­tients (da­ta ex­pect­ed in 2H 2018).

That’s the same game plan Am­i­cus out­lined when the EMA ear­li­er closed the door on their quest to gain a con­di­tion­al ap­proval in Eu­rope.

There have been wan­ing ex­pec­ta­tions that the FDA would come through here on lit­tle more than bio­mark­er da­ta. But with the new Trump ad­min­is­tra­tion — rep­re­sent­ed by FDA com­mis­sion­er Scott Got­tlieb — cel­e­brat­ing fast new OKs while ham­mer­ing any prospec­tive price in­creas­es, hope lin­gered. And Crow­ley isn’t known for tak­ing no as an an­swer.

Leerink’s Joseph Schwartz, who led the sell side cheer­lead­ers, has been keep­ing the flame burn­ing. He not­ed re­cent­ly:

Galafold ap­proval could re­flect a more flex­i­ble FDA that is will­ing to ac­cel­er­ate the reg­u­la­to­ry process in the US.

Not this time. Or at least, not yet. And Schwartz this morn­ing says the snub will like­ly lead to a lengthy de­lay for this drug ahead of any ap­proval.

We had pre­vi­ous­ly as­signed 50% prob­a­bil­i­ty of a 2019 mkt. en­try but we are now push­ing out the com­mer­cial launch to 2022E, with a 75% PoS.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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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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As­traZeneca trum­pets the 'mo­men­tous' 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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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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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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Ab­b­Vie wins an ap­proval in uter­ine fi­broid-as­so­ci­at­ed heavy bleed­ing. Are ri­vals My­ovant and Ob­sE­va far be­hind?

Women expel on average about 2 to 3 tablespoons of blood during their time of the month. But with uterine fibroids, heavy bleeding is typical — a third of a cup or more. Drugmakers have been working on oral therapies to try and stem the flow, and as expected, AbbVie and their partners at Neurocrine Biosciences are the first to make it across the finish line.

Known chemically as elagolix, the drug is already approved as a treatment for endometriosis under the brand name Orilissa. It targets the GnRH receptor to decrease the production of estrogen and progesterone.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

Roger Perlmutter, Merck R&D chief (YouTube)

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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