Gilead boasts of pos­i­tive remde­sivir da­ta on mor­tal­i­ty — but their analy­sis pro­vokes the skep­tics

Gilead is surg­ing again off da­ta that sug­gest its an­tivi­ral remde­sivir might im­prove sur­vival.

The new da­ta come from an analy­sis Gilead con­duct­ed com­par­ing the death rate and re­cov­ery time of pa­tients in one of its remde­sivir tri­als to a group of 800 pa­tients “with sim­i­lar base­line char­ac­ter­is­tics and dis­ease sever­i­ty” who re­ceived on­ly stan­dard-of-care around the same time. The re­sult, they said, sug­gest­ed that pa­tients who re­ceived remde­sivir had a 62% bet­ter chance at sur­viv­ing than those who did not.

Ret­ro­spec­tive looks are more sus­pect than place­bo-con­trolled tri­als, so the da­ta are far from con­clu­sive. But it does add new ev­i­dence to the ques­tion of whether remde­sivir can im­prove mor­tal­i­ty. The NIH tri­al that led the FDA to au­tho­rize remde­sivir for use in Covid-19 pa­tients on­ly showed that remde­sivir could help pa­tients re­cov­er faster. Few­er pa­tients on remde­sivir died in that tri­al than on stan­dard-of-care — 8% vs 11% — but the re­sults were not sig­nif­i­cant.

For­mer FDA chief sci­en­tist Lu­ciana Bo­rio said that da­ta from ob­ser­va­tion­al stud­ies are dif­fi­cult to in­ter­pret, but that the bulk of ev­i­dence points to remde­sivir help­ing pa­tients sur­vive Covid-19. “It is very like­ly that Remde­sivir does in­deed af­ford pa­tients a mor­tal­i­ty ben­e­fit,” she said in an email. “The gold stan­dard RCT con­duct­ed by the NIH demon­strat­ing the clin­i­cal ben­e­fit of Remde­sivir in im­prov­ing time to re­cov­er wasn’t pow­ered to de­tect a dif­fer­ence in mor­tal­i­ty. It doesn’t mean that there wasn’t one”

In a tweet, for­mer FDA chief Scott Got­tlieb called the re­sults “en­cour­ag­ing” but urged that it need­ed to be con­firmed. The com­pa­ny ac­knowl­edged in their press re­lease that a prospec­tive study was need­ed.

Gilead was up 2% pre-mar­ket on the news, from $74.71 to $76.18.

Oth­er an­a­lysts and ex­perts, though, were more skep­ti­cal and in some cas­es en­tire­ly dis­mis­sive. Pe­ter Bach, the di­rec­tor for the cen­ter for health pol­i­cy and out­comes at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter, said on Twit­ter that com­par­ing tri­al par­tic­i­pants to re­al-world pa­tients on­ly works when doc­tors have a good un­der­stand­ing of the dis­ease, some­thing that isn’t the case with Covid-19.

Baird an­a­lyst Bri­an Sko­r­ney com­pared the study to work from Di­di­er Raoult, the French physi­cian who pub­lished in Feb­ru­ary one of the first tri­als on hy­drox­y­chloro­quine in Covid-19. That study, de­spite gin­ning up sig­nif­i­cant in­ter­est in the drug, was seen as rife with is­sues, in­clud­ing in “los­ing” 6 of the tri­al’s 26 pa­tients “in fol­low-up,” 4 of whom died.

In the study, 312 pa­tients from Gilead’s open-la­bel SIM­PLE tri­al were com­pared to 818 re­al-world pa­tients who had sim­i­lar char­ac­ter­is­tics. Pa­tients who re­ceived remde­sivir had a 7.6% chance of dy­ing, com­pared to 12.6% for those who re­ceived stan­dard-of-care. That trans­lates to 62% im­proved odds of sur­vival — or slight­ly more than dou­ble the 30% im­proved odds the NIH tri­al’s raw num­bers sug­gest­ed.

Sun­Trust an­a­lyst Robyn Kar­nauskas said the sur­vival rates in each tri­al were “com­pa­ra­ble,” while ac­knowl­edg­ing the study was “not an ap­ples-to-ap­ples com­par­i­son.”

The remde­sivir pa­tients in the analy­sis al­so had a 74.4% chance of re­cov­er­ing by day 14, ver­sus 59.9% for stan­dard-of-care.

Not every tri­al, though, has found remde­sivir has a ben­e­fit. On the same day as the NIH read­out, The Lancet pub­lished a study from Chi­na that found remde­sivir im­proved nei­ther mor­tal­i­ty nor oth­er pa­tient met­rics. That study, though, was end­ed ear­ly af­ter the first out­break in Chi­na end­ed.

For hos­pi­tals that can get their hands on it, remde­sivir so far has been one of on­ly two proven treat­ments for Covid-19, along with a gener­ic steroid called dex­am­etha­sone. More an­tivi­rals and neu­tral­iz­ing an­ti­bod­ies, though, are now be­ing stud­ied, hav­ing be­come a grow­ing fo­cus of the Trump ad­min­is­tra­tion. If more ther­a­pies prove ef­fec­tive, the new Gilead da­ta could help physi­cians choose the best op­tion.

“While not as vig­or­ous as a ran­dom­ized con­trolled tri­al, this analy­sis im­por­tant­ly draws from a re­al-world set­ting and serves as an im­por­tant ad­junct to clin­i­cal tri­al da­ta, adding to our col­lec­tive un­der­stand­ing of this virus and re­flect­ing the ex­tra­or­di­nary pace of the on­go­ing pan­dem­ic,” in­ves­ti­ga­tor Su­san Olen­der of Co­lum­bia Uni­ver­si­ty Irv­ing Med­ical Cen­ter said in a state­ment.

So­cial: AP Im­ages

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2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

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Philip Astley-Sparke, Replimune CEO

Replimune looks to rope in $225M on the back of melanoma da­ta

The Massachusetts-based, oncolytic virus biotech Replimune is feeling bullish now that it has lifted the cover on data for its lead product.

Replimune said Thursday it looks to nab about $225 million from a public offering after giving a snapshot of some initial data from its IGNYTE clinical study earlier this week. The trial is investigating RP1 in combination with Opdivo, for patients with melanoma and who did not have a response when being treated with a PD-1.