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).

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

President Donald Trump and FDA Commissioner Stephen Hahn (AP Images)

FDA is­sues fi­nal rule al­low­ing im­por­ta­tion of drugs from Cana­da — but al­so keeps the pow­er to re­voke it

Just over a month away from the presidential election, the FDA has issued a final regulation fulfilling President Trump’s promise to let states import certain prescription drugs from Canada.

On Thursday, Trump told a crowd in North Carolina that the new rule goes into effect “today.” But the published regulation states that it won’t take effect for 60 days. And even then, it could be a while before cheaper drugs make it across the border.

The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

President Trump walks past HHS secretary Alex Azar (Getty Images)

Azar falls in line un­der Trump again. Ex­perts say he's re­in­forc­ing a dark sig­nal sent to the FDA

In the latest incident where Alex Azar has steadfastly taken the side of President Donald Trump over that of the FDA, the HHS secretary was noncommittal this morning when asked if he supports the attempt by his subordinates at the FDA to strengthen guidelines for a vaccine EUA.

Appearing on NBC’s Today Show, the HHS secretary muddied the waters, stating that the guidance that matters is the one that is “actually already out there.”