Daniel O'Day speaks during a meeting with President Donald Trump in the Oval Office of the White House (AP Images)

FDA cheers on Gilead­'s remde­sivir with a snap emer­gency OK, giv­ing bio­phar­ma a shot at re­demp­tion

The FDA didn’t leave much time to as­sess the pos­i­tive na­ture of the first cut of da­ta from a con­trolled study of remde­sivir in se­vere­ly af­flict­ed coro­n­avirus cas­es. On Fri­day the agency stamped their ap­proval on Gilead’s drug, which NI­AID di­rec­tor An­tho­ny Fau­ci has al­ready de­ter­mined should be­come the new stan­dard of care in treat­ing hos­pi­tal­ized pa­tients fight­ing off Covid-19.

Then on Sun­day, Gilead CEO Dan O’Day took to “Face the Na­tion” to promise that the first 1.5 mil­lion dos­es of this drug — enough to treat any­where from 100,000 to 200,000 pa­tients — would be rushed, free of charge, to US cities where it is need­ed the most.

“(W)e in­tend to get that to pa­tients in the ear­ly part of this next week, be­gin­ning to work with the gov­ern­ment, which will de­ter­mine which cities are most vul­ner­a­ble and- and where the pa­tients are that need this med­i­cine.”

While Wall Street an­a­lysts have been dis­ap­point­ed by O’Day’s lack of clar­i­ty on what it plans to charge for this drug, the com­pa­ny finds it­self in a unique po­si­tion to re­frame Amer­i­cans’ at­ti­tude to­ward drug­mak­ers. Be­fore the pan­dem­ic, phar­ma ex­ecs were held in low­er es­teem than law­mak­ers, rest­ing at the bot­tom of the totem pole in terms of pop­u­lar­i­ty. Now the com­pa­ny that shocked pay­ers in­to ac­tion on high hep C drug prices is in the dri­ver’s seat on Covid-19. If they pass muster on their price now, Gilead and the Roche vet who now helms the com­pa­ny will set the pace for the en­tire in­dus­try and go a long way for a pub­lic reap­praisal of the in­dus­try.

Any mis­han­dling of this, how­ev­er, could be a dis­as­ter.

Gilead has raised geopo­lit­i­cal con­cerns by hand­ing the ini­tial drug sup­ply over to the US gov­ern­ment. The whole world is scram­bling for ther­a­peu­tic in­ter­ven­tions, and giv­ing every­thing to Amer­i­cans won’t sit well abroad.

Af­ter re­cal­i­brat­ing the pri­ma­ry end­point, re­searchers de­ter­mined that the drug cut the av­er­age time to re­cov­ery to 11 days in the drug arm, com­pared to 15 in the con­trol group. The drug arm mor­tal­i­ty rate hit 8%, beat­ing out the con­trol group at 11%, but failed to come in at a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment on the first cut of the da­ta.

The ap­proval comes just 2 months af­ter the out­break tru­ly be­gan to rat­tle the US, and 3 months af­ter the Wuhan out­break trig­gered a pan­dem­ic that has killed peo­ple and shat­tered economies around the globe, sick­en­ing mil­lions. The FDA’s ac­tion fol­lows an emer­gency use au­tho­riza­tion for hy­drox­y­chloro­quine and chloro­quine, malar­ia meds that have al­so raised safe­ty alarms on their side ef­fects.

Stephen Hahn

As FDA com­mis­sion­er Stephen Hahn ob­served, the time it took to ramp up the tri­al and gain emer­gency au­tho­riza­tion stretched over 90 days — vir­tu­al­ly light speed in drug R&D and reg­u­la­to­ry terms. The FDA has made it clear that reg­u­la­tors will move first and ask com­plex ques­tions lat­er. And in this case, there’s still much left to learn about remde­sivir, in­clud­ing the best dose and its im­pact on dif­fer­ent pa­tient groups.

An­oth­er mys­tery is how Gilead plans to make mon­ey on the drug — and how much. An­a­lysts grilled CEO O’Day on that score on Thurs­day evening, dur­ing their Q1 call, but to no avail. All that has yet to play out, as Gilead of­fers the first 1.5 mil­lion dos­es for free.

O’Day’s em­pha­sis on re­spon­si­bil­i­ty first and prof­its sec­ond, though, has some dis­count­ing any up­side for the com­pa­ny.

Evan Seiger­man at Cred­it Su­isse was one of the first an­a­lysts to make a com­ment, not­ing:

We do not ex­pect RDV to be a sig­nif­i­cant com. biz for GILD & do not mod­el any sales.

Gilead’s stock, which has surged dra­mat­i­cal­ly from the start of the year, was down close to 5% by the clos­ing bell on Fri­day.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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