Days af­ter In­ter­cept re­jec­tion, Akero surges on ‘un­prece­dent­ed‘ NASH da­ta

A year and a half af­ter scor­ing a $70 mil­lion Se­ries B and a top Gilead ex­ec­u­tive as CEO, Akero Ther­a­peu­tics has an­nounced new da­ta on their NASH drug. And with the field still reel­ing from a sur­prise FDA re­jec­tion this week, the news was enough to send their stock surg­ing.

Akero had al­ready said in March that its lead drug had beat­en place­bo in its Phase II tri­al, re­duc­ing liv­er fat by 14% in the high­est dose group com­pared to 0.3% in place­bo, ac­cord­ing to MRI scans. But al­though NASH is an obe­si­ty-re­lat­ed con­di­tion and re­sults from fat­ty buildup in the liv­er, the re­al im­me­di­ate ques­tion for any ther­a­py is whether it can re­solve the fi­bro­sis and in­flam­ma­tion that re­sults from that buildup. Those da­ta re­quire biop­sy­ing the pa­tients, a longer and more in­va­sive process that was fur­ther com­pli­cat­ed by a pan­dem­ic.

Now the biop­sies are in: Of the 40 pa­tients in the tri­al who re­spond­ed to treat­ment — an old Am­gen drug known as efrux­ifer­min — and were biop­sied, 48% saw at least a one-stage im­prove­ment in fi­bro­sis with­out any wors­en­ing on NAS, a score that mea­sures in­flam­ma­tion and oth­er bi­o­log­i­cal mark­ers of dis­ease. Ad­di­tion­al­ly, 48% of pa­tients saw their NASH re­solve with­out fi­bro­sis wors­en­ing. Near­ly a third saw a 2-stage im­prove­ment in fi­bro­sis with­out NAS wors­en­ing. There was al­so a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in weight loss for the high treat­ment arm.

Now what pre­cise­ly does all those da­ta mean?

In­vestors ap­peared to take it as a clear pos­i­tive, par­tic­u­lar­ly af­ter the FDA re­ject­ed what would have been the first specif­i­cal­ly ap­proved NASH drug on Mon­day, and its de­vel­op­er, In­ter­cept, saw its stock crash. Akero’s stock was up near­ly 40% pre-mar­ket Wednes­day, from $24.92 to $34.42 per share.

An­a­lysts, too, were bull­ish. Peg­ging a tar­get price at $48, Jef­feries’ Michael Yee called it “best-in-class NASH da­ta so far,” with “the best fi­bro­sis re­sults and strong ben­e­fits across meta­bol­ic and di­a­betes com­po­nents.” Ever­core ISI’s Josh Schim­mer said it was “an un­prece­dent­ed ef­fect on fi­bro­sis im­prove­ment and NASH res­o­lu­tion.” He pegged the stock at $80.

The tone from the com­pa­ny was sim­i­lar­ly up­beat. An­drew Cheng, the ex-Gilead ex­ec­u­tive who came over as CEO, said the da­ta “ex­ceed­ed our ex­pec­ta­tions.”

Still, the cen­tral ques­tion raised by In­ter­cept’s sur­prise re­jec­tion re­mains unan­swered: What, pre­cise­ly, does the FDA con­sid­er to be a good bar for ef­fec­tive­ness in NASH?

In lieu of stronger but nec­es­sar­i­ly longer term goals such as sur­vival or the num­ber of pa­tients who reach cir­rho­sis, com­pa­nies, with FDA guid­ance, have so far fo­cused on two end­points: im­prov­ing fi­bro­sis with­out NASH wors­en­ing or re­solv­ing NASH with­out fi­bro­sis wors­en­ing.

In­ter­cept was the first com­pa­ny to hit on one of those in a Phase III tri­al, al­though they missed on the oth­er. Their re­jec­tion means that just hit­ting one end­point won’t be enough.

Ad­di­tion­al­ly, to­day’s da­ta are on­ly a sec­ondary analy­sis of re­spon­ders, and don’t prove a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in those end­points com­pared to place­bo. Akero will need a larg­er piv­otal tri­al for that, one that could be the next big study for the field.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

In search of elu­sive NASH break­through, Pfiz­er spot­lights com­bo ap­proach

Pfizer’s second crack at steering a NASH candidate through a battered field seems to be going better than the first.

The pharma giant has scored the FDA’s fast track designation for an experimental combination therapy as a treatment for NASH with liver fibrosis. The combo consists of ervogastat, a diacylglycerol O-acyltransferase 2 inhibitor (DGAT2i), and clesacostat, an acetyl-CoA carboxylase inhibitor (ACCi).

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

AS­CO ab­stracts mint some ear­ly win­ners, but Io­vance, Spring­Works get hit by mas­sive loss­es

Before cancer-focused biotechs start their trek to the first in-person American Society of Clinical Oncology (ASCO) annual conference since the pandemic began, investors have taken a good look at the teasers for the data scheduled to be presented — and started placing bets.

With its power to confer overnight fame, ASCO is a stage where impressive or surprise debut performances can go a long way. On the other hand, disappointing details could be punishing.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Levi Garraway, Roche CMO (Genentech)

Roche's CD20xCD3 does­n't beat Gen­mab at ORR, but sets bar for CR da­ta on lym­phoma drug

On its way to potentially becoming the first to market with a CD20xCD3 bispecific for aggressive lymphoma, Big Pharma’s largest R&D spender has some more data to pad its case with an FDA filing slated for later this year.

Roche dropped some more details from a Phase II expansion study of its fixed-duration glofitamab, to be presented at next week’s ASCO annual meeting, in patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). The patients had received a median of three prior therapies.

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