Promis­ing or in­con­clu­sive? Gilead fields PhII GS-4997 da­ta, trig­ger­ing more push­back on NASH claims

Gilead post­ed a de­tailed snap­shot of Phase II ef­fi­ca­cy da­ta for GS-4997, now known as selon­sert­ib, for NASH and liv­er fi­bro­sis, trig­ger­ing a mixed re­sponse of en­thu­si­asm and some clear skep­ti­cism from the an­a­lysts watch­ing this late-stage drug, some of whom got their first crack in when Gilead out­lined the top line da­ta from this study in Oc­to­ber.

The num­bers were de­tailed for selon­sert­ib alone, in com­bi­na­tion with sim­tuzum­ab or in sim­tuzum­ab alone. And in­ves­ti­ga­tors had a mixed bag of re­sults un­der­scor­ing drug ac­tiv­i­ty, rang­ing from the 43% of pa­tients tak­ing selon­sert­ib see­ing an im­prove­ment in fi­bro­sis com­pared to the 20% in the sim­tuzum­ab arm to the 20% in the selon­sert­ib arm ex­pe­ri­enc­ing a min­i­mum 15% re­duc­tion in liv­er stiff­ness to the 32% in the com­bo group and none tak­ing sim­tuzum­ab alone.

Phase 2 Da­ta for Selon­sert­ib in Non­al­co­holic Steato­hep­ati­tis (NASH) Pre­sent­ed at The Liv­er Meet­ing® 2016.

 

Gilead $GILD ac­knowl­edged ear­li­er this month that sim­tuzum­ab has been a bust in the clin­ic, prompt­ing its R&D team to drop it com­plete­ly. It al­so has GS-9674 in ear­ly-stage stud­ies for NASH and re­cent­ly picked up an­oth­er pro­gram from Nim­bus. And NASH re­mains a key fea­ture in the pipeline as Gilead looks to over­come the hang­over from its hep C bash, where rev­enue is now de­clin­ing steadi­ly.

Bri­an Sko­r­ney, Baird

Even last month, though, an­a­lysts like Bri­an Sko­r­ney at Baird were shak­ing their head over what Gilead had to of­fer:

“The num­bers are too small, the du­ra­tion of treat­ment is too short and the da­ta is com­pli­cat­ed by the in­clu­sion of an­oth­er drug in­stead of place­bo.”

Af­ter its stun­ning suc­cess with hep C, Gilead’s R&D strat­e­gy in gen­er­al has come un­der fre­quent at­tack as skep­ti­cism grows about its abil­i­ty to over­come its grow­ing prob­lems on the rev­enue side of the busi­ness.

Ro­hit Loom­ba, the lead study au­thor and di­rec­tor of the NAFLD Re­search Cen­ter, had this to say in a state­ment:

“Af­ter on­ly 24 weeks of ther­a­py, selon­sert­ib ex­hib­it­ed promis­ing an­ti-fi­brot­ic ac­tiv­i­ty in this study, which was the first known mul­ti-cen­ter NASH clin­i­cal tri­al to use cen­tral­ly-as­sessed MRE, MRI-PDFF, in ad­di­tion to liv­er biop­sy as end­points. Based on these da­ta, selon­sert­ib rep­re­sents an im­por­tant in­ves­ti­ga­tion­al drug can­di­date for fur­ther clin­i­cal tri­als in pa­tients with NASH and sig­nif­i­cant fi­bro­sis.”

Ge­of­frey Porges, Leerink

Well, maybe, re­sponds Leerink’s Ge­of­frey Porges, who notes that this is the study that prompt­ed Gilead to go big in Phase III:

“In their most re­cent earn­ings con­fer­ence call the com­pa­ny dis­closed that the prod­uct has failed in de­vel­op­ment in oth­er in­di­ca­tions such as di­a­bet­ic nephropa­thy, and par­al­lel pro­grams in these liv­er dis­eases, in­clud­ing sim­tuzum­ab, have al­so been dis­con­tin­ued. How­ev­er, man­age­ment’s re­ac­tion to GS4997 was very pos­i­tive and sug­gest­ed im­pres­sive phase II re­sults. Our re­ac­tion to the phase II re­sults is that they do show en­cour­ag­ing signs of dis­ease ac­tiv­i­ty, but at this stage re­gard the ev­i­dence of ben­e­fit, and safe­ty and tol­er­a­bil­i­ty, as in­con­clu­sive. The ev­i­dence of ef­fect is per­sua­sive on some end­points, but un­clear on oth­ers, and the NASH field is like­ly to re­quire con­tin­ued re­search to re­fine tri­al end­points and to es­tab­lish some mean­ing­ful cor­re­la­tion be­tween those end­points and clin­i­cal ben­e­fit for pa­tients. GS4997 al­so ap­pears to have some tol­er­a­bil­i­ty li­a­bil­i­ties, which could add to the drug’s lim­i­ta­tions in both de­vel­op­ment and com­mer­cial­iza­tion.”

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 153,900+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting: