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.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine -- so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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No­var­tis says Kym­ri­ah reach­es pri­ma­ry end­point in new PhII, al­though num­bers still to come

The race to develop CAR-T therapies has died down since Novartis’ Kymriah and Gilead’s Yescarta first crossed the finish line, though Tecartus also recently received approval. But the companies continue to expand their drugs’ applications, with Novartis preparing to conclude a new Phase II.

Interim data announced by the Swiss pharma show that Kymriah met its primary endpoint of complete response rate in treating patients with relapsed or refractory follicular lymphoma, the second-most common form of non-Hodgkin lymphoma. Based on preliminary trial findings, Kymriah had received RMAT designation from the FDA in April for r/r follicular lymphoma.

Ab­b­Vie shrugs off $134M cash deals, quit­ting a neu­ro R&D pact with Voy­ager Ther­a­peu­tics on vec­tor­ized an­ti­body treat­ments

It’s the end of the road for Voyager Therapeutics’ collaboration with AbbVie on tau and alpha-synuclein vectorized antibody development.

In two deals spanning the last two years, AbbVie dropped more than $134 million upfront for Voyager’s preclinical R&D of vectorized antibody treatments for diseases like Alzheimer’s and Parkinson’s. But Voyager says AbbVie is walking away now, without offering an explanation for why.

Mer­ck scoops up a PhII J&J dis­card in a bar­gain-base­ment deal. And this time they’re shoot­ing at NASH

When J&J turned to South Korea’s Hanmi for a GLP-1/glucagon dual receptor agonist obesity drug, the pharma giant paid $105 million in a cash upfront for the licensing rights and plotted a big clinical trial program to test it. A year ago, like a few of Hanmi’s big partners, J&J reviewed their trial data and walked away, handing it back.

Now Merck is stepping up to grab it for their NASH pipeline — and they got it a lot cheaper than J&J.

Igor Splawski (CureVac)

Cure­Vac nabs a top No­var­tis sci­en­tist for CSO slot as mR­NA vac­cines seize the spot­light

One of the key players in the race to develop a new mRNA vaccine to fight Covid-19 has reshuffled the top spots in the executive suite. And they’re bringing in a Novartis vet out of Harvard to spearhead their work on mRNA.

CureVac, which just filed for an IPO that’s still taking shape, has formally handed Franz-Werner Haas the CEO title, after giving it to him on an interim basis. And the still rather stealthy German biotech largely owned by billionaire Dietmar Hopp has recruited Igor Splawski as its chief scientific officer.

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