Ver­tex's rapid-fire PhI­II pro­gram hits a road­block at the FDA. Will reg­u­la­tors force ri­val Gala­pa­gos to slow down too?

Ver­tex’s R&D team doesn’t make many mis­takes. So dur­ing yes­ter­day’s Q1 call, a group of an­a­lysts were quick to pick up on a rare stum­ble that just might make it more vul­ner­a­ble to a ri­val like Gala­pa­gos.

Ge­of­frey Porges, Leerink

While not­ing plans for launch­ing a new Phase III ef­fort with a com­bi­na­tion drug us­ing VX-445 with Symdeko, fol­low­ing up as planned, in­ves­ti­ga­tors $VRTX al­so con­ced­ed that the FDA sees its deuter­at­ed ver­sion of Ka­ly­de­co — VX-561 — as a nov­el agent that needs some ad­di­tion­al dose-rang­ing tri­al work be­fore reg­u­la­tors will al­low a move in­to Phase III.

Ver­tex bought ‘561 from Con­cert a lit­tle more than a year ago for $160 mil­lion in cash and an­oth­er $90 mil­lion in mile­stones, tak­ing over the Phase II tri­al.

Ge­of­frey Porges at Leerink sees a de­lay on VX-561 of at least a year, forc­ing the shift of fo­cus to the VX-445 com­bo now as a kind of Plan B.

This means that the com­pa­ny is now run­ning two rough­ly equiv­a­lent hors­es in the same ex­pen­sive race, and while the risk mit­i­ga­tion from this strat­e­gy is laud­able, it does seem more ex­pen­sive, in terms of tri­al costs, time and rev­enue fore­gone, than is nec­es­sary. Ver­tex may still de­vel­op a Vx561 con­tain­ing once-dai­ly triple com­bi­na­tion, but such a prod­uct might not come to mar­ket now un­til 2022 or 2023. The FDA’s stance does pro­vide some re­as­sur­ance about Ver­tex’s com­pet­i­tive po­si­tion – the FDA is clear­ly sig­nal­ing that there are no short-cuts (at least in the US) to tra­di­tion­al good drug de­vel­op­ment prac­tices in this in­di­ca­tion.

That “com­pet­i­tive po­si­tion” he’s re­fer­ring to deals with Gala­pa­gos $GLPG, which launched an ex­plorato­ry study called FAL­CON for its triple ear­li­er this week. And what the FDA finds good for Ver­tex, they’ll like­ly find good for Gala­pa­gos, where the dose-rang­ing stan­dards may al­so like­ly to slow things down. Notes Michael Yee from Jef­feries:

Thus since GLPG has all new NCE’s they will need to do lots of dose-find­ing work re­quir­ing lots more Phase IIs than the “quick” stud­ies they’re do­ing now…so while GLPG might have “da­ta” for FAL­CON study H2-18 and more da­ta in 2019 — these are sim­ple stud­ies and will need thor­ough work per FDA read-through.

Bri­an Sko­r­ney

Baird’s Bri­an Sko­r­ney al­so took a shot at Gala­pa­gos.

(W)e be­lieve it shows clear reg­u­la­to­ry prece­dent for po­ten­tial com­peti­tors who think they can move a com­bi­na­tion of three new chem­i­cal en­ti­ties in­to late stage de­vel­op­ment with­out demon­strat­ing the clin­i­cal at­trib­ut­es and li­a­bil­i­ties of each com­po­nent (we’re look­ing at you Gala­pa­gos).

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Pascal Soriot, AstraZeneca CEO (AP Images)

Pas­cal So­ri­ot cash­es in As­traZeneca’s chips on Mod­er­na for $1.2B cash in­jec­tion

While still working to prove its own Covid-19 vaccine, AstraZeneca has reportedly capitalized on the success of another.

The company has sold off its 7.7% stake in Moderna and turned it into $1.2 billion in cash, according to the Times, beefing up the reserves just as Pascal Soriot is wrapping up his $39 billion acquisition of Alexion and its rare disease pipeline.

AstraZeneca’s stock sale follows a similar move by Merck in December. But like its pharma brethren, the British giant is keeping its R&D collaborations with Moderna.

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Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

UP­DAT­ED: Feds clear the road for J&J to start de­liv­er­ing mil­lions of dos­es of their Covid-19 vac­cine — but frets linger about run­ner-up sta­tus

All the pieces needed to trigger a third wave of Covid-19 vaccine supply to start washing over the US fell neatly into place over the weekend.

After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days.

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