UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a block­buster rare dis­ease gi­ant in its sights in Alex­ion’s Soliris, small biotech Apel­lis has rea­son to think its com­peti­tor is wor­thy of the spot­light. Now, with the FDA on its side, Apel­lis will get its chance to be the David to Alex­ion’s Go­liath.

The FDA on Fri­day ap­proved Em­paveli (pegc­eta­coplan), a C3 com­ple­ment in­hibitor the biotech thinks can prove a wor­thy chal­lenger to Alex­ion’s C5 in­hibitors Soliris and fol­low-up drug Ul­tomiris in rare dis­ease parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

In piv­otal Phase III da­ta on which the agency based its re­view, Em­paveli best­ed Soliris in a head-to-head test in terms of he­mo­glo­bin pro­duc­tion with a rel­a­tive in­crease of 3.9 g/dL (p<0.0001), and some fa­vor­able trend lines in sec­ondary end­points such as trans­fu­sions and fa­tigue.

Em­paveli will come with the same black box warn­ing la­bel as Soliris, re­strict­ing use un­der a risk eval­u­a­tion and mit­i­ga­tion strat­e­gy for meningo­coc­cal in­fec­tions. The ther­a­py was ap­proved for pa­tients re­gard­less of pri­or ther­a­pies, mean­ing its use won’t be re­strict­ed to for­mer C5 pa­tients alone.

On a call with an­a­lysts Mon­day, Apel­lis said Em­paveli would come with an av­er­age an­nu­al whole­sale ac­qui­si­tion cost of $458,000. That’s “at par­i­ty” with Ul­tomiris and a dis­count on Soliris, the com­pa­ny said.

Apel­lis thinks that a big bump in he­mo­glo­bin lev­els could help it im­me­di­ate­ly cap­ture about a third of the ex­ist­ing PNH pa­tients who have a low and falling he­mo­glo­bin count that is cur­rent­ly un­der­served by the C5 drugs, CEO Cedric Fran­cois told End­points News pri­or to the ap­proval.

“If the Phase III study did one thing it is mak­ing it very clear that by con­trol­ling ex­travas­cu­lar he­mol­y­sis, you can el­e­vate the stan­dard of care in these pa­tients,” Fran­cois said.

The key to Apel­lis’ pitch is C3, which it be­lieves can of­fer a longer-last­ing so­lu­tion for he­mo­glo­bin pro­duc­tion as C5 of­ten doesn’t boost lev­els for more than a few days, the team said. With an ap­proval in hand, Apel­lis said it plans to tar­get those 500 or so US pa­tients with the low­est he­mo­glo­bin counts but thinks Em­paveli could have a mean­ing­ful up­take across the en­tire PNH spec­trum.

The com­pa­ny has al­ready built up a sales team that has been do­ing some of the ground­work on physi­cian out­reach in the lead-up to ap­proval. It’s a small group of PNH spe­cial­ists, and Apel­lis thinks it’s found a work­able, hy­brid dig­i­tal/in-per­son ed­u­ca­tion cam­paign to tell doc­tors about the drug’s ben­e­fits.

But beat­ing Alex­ion — and soon to be As­traZeneca — won’t be an easy task by any means. Alex­ion’s en­tire fran­chise was built around build­ing Soliris in­to a block­buster, and the com­pa­ny has been busy amid a gung-ho switch­ing cam­paign to fol­low-up drug Ul­tomiris.

As­traZeneca an­nounced its in­tent to buy Alex­ion late last year for a cool $39 bil­lion. The rare dis­ease spe­cial­ist will im­port its ex­pan­sive port­fo­lio of C5 drugs, which sport in­di­ca­tions be­yond PNH.

This sto­ry will be up­dat­ed.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, 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.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

FDA plans new stud­ies on ac­cel­er­at­ed ap­proval dis­clo­sures in bio­phar­ma ads

When people read biopharma companies’ websites about new drugs approved via the FDA’s accelerated pathway, like Biogen’s new Alzheimer’s drug, do they understand that these drugs may only be reasonably likely to predict clinical benefit and still require confirmatory studies?

That’s what the FDA’s Office of Prescription Drug Promotion wants to firm up as an agency analysis of direct-to-consumer websites for accelerated approval drugs previously found that only 21% of the disclosures used language directly from the label.

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An ex­pen­sive watch, shell com­pa­nies and fake in­voic­es: How two Is­raeli traders tapped in­to a $100M glob­al biotech in­sid­er trad­ing ring

It appears that we have reached the end of the saga about the global insider trading ring that collectively reaped $100 million from placing “timely, profitable” trades in biotech stocks like Ariad, Pharmacyclics and Receptos.

Tomer Feingold and Dov Malnik — Israeli traders living in Switzerland — were the last out of eight to be charged as the SEC unraveled the scheme, which ran from 2013 through 2017. Together, according to a statement in March, the pair had pocketed more than $4 million.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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