Af­ter NIH tri­al, EMA be­gins rolling re­view for remde­sivir, set­ting up po­ten­tial au­tho­riza­tions in US and Eu­rope

A day af­ter the NIH re­leased pos­i­tive re­sults from their tri­al test­ing remde­sivir in Covid-19, the EMA has an­nounced the start of a rolling re­view process for the drug. With an FDA de­ci­sion ex­pect­ed to ar­rive in the com­ing days, the news sets the stage for the Gilead an­tivi­ral to be­come the stan­dard-of-care for the dead­ly virus in both the US and Eu­rope.

Yes­ter­day morn­ing, Gilead is­sued a press re­lease that the NIH would soon is­sue pos­i­tive da­ta on a much-an­tic­i­pat­ed tri­al test­ing their an­tivi­ral remde­sivir against a place­bo in Covid-19 pa­tients. The news prompt­ed im­me­di­ate spec­u­la­tion of whether an emer­gency use au­tho­riza­tion (EUA) — a clear­ance from the FDA that by­pass­es the lengthy ap­proval process — would fol­low. The agency had con­tro­ver­sial­ly is­sued an EUA for the an­ti­malar­i­als hy­drox­y­chloro­quine and chloro­quine in March, a de­ci­sion that was tak­en with­out the sup­port of a ran­dom­ized con­trolled tri­al.

“Remde­sivir would ap­pear to meet or ex­ceed stan­dard for au­tho­riza­tion un­der EUA, es­pe­cial­ly rel­a­tive to oth­er EUAs is­sued,” for­mer FDA chief Scott Got­tlieb said on Twit­ter an hour af­ter Gilead’s press re­lease.

Af­ter the da­ta came out — show­ing a 31% im­prove­ment in re­cov­ery time and a nu­mer­i­cal, but not sta­tis­ti­cal­ly sig­nif­i­cant, im­prove­ment in mor­tal­i­ty for pa­tients tak­ing the drug — The New York Times re­port­ed that the FDA could au­tho­rize the drug that day. In a state­ment to End­points News, the FDA didn’t com­ment on the re­port but said the agency has “been en­gaged in sus­tained and on­go­ing dis­cus­sions with Gilead Sci­ences re­gard­ing mak­ing remde­sivir avail­able to pa­tients as quick­ly as pos­si­ble, as ap­pro­pri­ate.”

An­tho­ny Fau­ci, di­rec­tor of the Na­tion­al In­sti­tute of Al­ler­gy and In­fec­tious Dis­eases, in an Oval Of­fice press con­fer­ence, said that the drug should be­come the “new stan­dard of care” for all Covid-19 clin­i­cal tri­als.

The EMA, how­ev­er, has tak­en a hard­er stance on emer­gency au­tho­riza­tions for Covid-19. Four days af­ter the FDA is­sued its let­ter al­low­ing the use of an­ti­malar­i­als, the EMA is­sued a con­trast­ing state­ment. They cau­tioned physi­cians against pre­scrib­ing the drug out­side their ap­proved use or spe­cif­ic na­tion­al pro­to­cols, cit­ing both po­ten­tial side ef­fects and not­ing that, “ef­fi­ca­cy in treat­ing COVID-19 is yet to be shown in stud­ies. (Sub­se­quent da­ta on the drug have yet to clear the pic­ture, al­though large ran­dom­ized con­trol stud­ies are un­der­way.)

The agency said they be­gan the rolling re­view process for remde­sivir in re­sponse to the da­ta from the NIH tri­al, which was the largest ran­dom­ized, place­bo-con­trolled study to read­out on a po­ten­tial Covid-19 drug and the first to show ef­fi­ca­cy. They said they will al­so con­sid­er oth­er da­ta around the drug, in­clud­ing a Chi­nese study pub­lished in The Lancet yes­ter­day that showed no sig­nif­i­cant ef­fi­ca­cy for the drug but in a far small­er pa­tient pop­u­la­tion.

The agency said they could not of­fer a time­line for when a de­ci­sion would be made, al­though the process would be faster than the stan­dard ap­proval process for new drugs.

An emer­gency use au­tho­riza­tion would po­ten­tial­ly al­low for more pa­tients to ac­cess the drug. To date, the an­tivi­ral, orig­i­nal­ly de­vel­oped for he­pati­tis C,  has on­ly been avail­able to pa­tients in clin­i­cal tri­als or un­der spe­cif­ic com­pas­sion­ate use or ex­pand­ed ac­cess pro­to­cols.

In an open let­ter yes­ter­day, Gilead CEO Daniel O’Day said the com­pa­ny has been ramp­ing up sup­ply of the drug since Jan­u­ary and now have about 1.5 mil­lion dos­es on hand. That would be enough for 140,000 cours­es of 10-day treat­ment or 280,000 cours­es of 5-day treat­ments — a Gilead study al­so re­leased yes­ter­day sug­gest­ed both of­fered sim­i­lar ef­fi­ca­cy.

“On the sup­ply side, we are work­ing to build a glob­al con­sor­tium of phar­ma­ceu­ti­cal and chem­i­cal man­u­fac­tur­ers to ex­pand glob­al ca­pac­i­ty and pro­duc­tion,” he said. “In the event of reg­u­la­to­ry ac­tion, we are in dis­cus­sions with var­i­ous groups about how we might bring remde­sivir to the de­vel­op­ing world.”

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

So­cial im­age: An­tho­ny Fau­ci and Deb­o­rah Birx, AP Im­ages

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.