As CEO bounces back from Covid-19, Apel­lis says FDA meet­ing clears path to file for an OK on Soliris ri­val

Af­ter hold­ing up the com­pa­ny more than 4 months af­ter re­port­ing pos­i­tive head-to-head da­ta against the block­buster drug Soliris, Apel­lis $APLS says it’s cleared things up with the FDA and is ready to file for an ap­proval with their ri­val PNH drug pegc­eta­coplan.

The Waltham, MA-based biotech — whose CEO, Cedric Fran­cois, has re­cov­ered from a bout with Covid-19 — put out the word Thurs­day morn­ing that ex­ecs had a “suc­cess­ful out­come of the pre-NDA meet­ing” with the agency and they’re ready to press ahead with their fil­ing in the sec­ond half of this year.

The un­ex­pect­ed de­lay marred Apel­lis’ pos­i­tive read­out back in ear­ly Jan­u­ary, as re­searchers tracked an “im­prove­ment in ad­just­ed means of 3.8 g/dL of he­mo­glo­bin at week 16 (p<0.0001). At week 16, pegc­eta­coplan-treat­ed pa­tients (n=41) had an ad­just­ed mean he­mo­glo­bin in­crease of 2.4 g/dL from a base­line of 8.7 g/dL, com­pared to eculizum­ab-treat­ed pa­tients (n=39) who had a change of -1.5 g/dL from a base­line of 8.7 g/dL.”

The hitch, though, was spelled out in an SEC fil­ing. The agency told them that:

(F)or the ap­proval of a new treat­ment for PNH, he­mo­glo­bin sta­bi­liza­tion in con­junc­tion with change in trans­fu­sion de­pen­dence con­sti­tute ac­cept­ed clin­i­cal ben­e­fit, but that a rise in he­mo­glo­bin lev­els may not trans­late to clin­i­cal ben­e­fit in pa­tients who en­tered the tri­al with high he­mo­glo­bin lev­els, such as per­mit­ted by the in­clu­sion cri­te­ria of the PE­GA­SUS tri­al, and who do not re­quire trans­fu­sions.

Apel­lis didn’t spell out the agency’s new po­si­tion, but ev­i­dent­ly no longer be­lieves the FDA may have trou­ble with its ap­pli­ca­tion.

Phil Nadeau at Cowen, mean­while, says he’s ex­pect­ing da­ta from a study in­volv­ing treat­ment-naive pa­tients in the ear­ly part of next year af­ter Covid-19 de­layed some of the com­pa­ny’s time­lines on de­vel­op­ment. He not­ed this morn­ing:

(T)here had re­mained a chance that FDA would want ad­di­tion­al da­ta be­yond PE­GA­SUS and that the NDA fil­ing would have to wait un­til re­sults from the sec­ond Phase III tri­al in treat­ment-naive PNH pa­tients in ear­ly 2021. With this morn­ing’s an­nounce­ment, that pos­si­bil­i­ty has been re­moved.

Nadeau is ex­pect­ing an ap­proval with peak sales ahead of $400 mil­lion a year.

As we re­port­ed a cou­ple of months ago, Fran­cois post­ed that he was one of the first to be in­fect­ed by coro­n­avirus dur­ing the first wave. A spokesper­son for the com­pa­ny tells me:

Cedric has been ful­ly re­cov­ered for some time now with a high lev­el of an­ti­bod­ies, and is ex­cit­ed for the fu­ture of Apel­lis and to see all the col­lab­o­ra­tion be­tween biotechs and health­care com­pa­nies dur­ing this pan­dem­ic.

Fangliang Zhang, AP Images

UP­DAT­ED: Leg­end fetch­es $424 mil­lion, emerges as biggest win­ner yet in pan­dem­ic IPO boom as shares soar

Amid a flurry of splashy pandemic IPOs, a J&J-partnered Chinese biotech has emerged with one of the largest public raises in biotech history.

Legend Biotech, the Nanjing-based CAR-T developer, has raised $424 million on NASDAQ. The biotech had originally filed for a still-hefty $350 million, based on a range of $18-$20, but managed to fetch $23 per share, allowing them to well-eclipse the massive raises from companies like Allogene, Juno, Galapagos, though they’ll still fall a few dollars short of Moderna’s record-setting $600 million raise from 2018.

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As it hap­pened: A bid­ding war for an an­tibi­ot­ic mak­er in a mar­ket that has rav­aged its peers

In a bewildering twist to the long-suffering market for antibiotics — there has actually been a bidding war for an antibiotic company: Tetraphase.

It all started back in March, when the maker of Xerava (an FDA approved therapy for complicated intra-abdominal infections) said it had received an offer from AcelRx for an all-stock deal valued at $14.4 million.

The offer was well-timed. Xerava was approved in 2018, four years after Tetraphase posted its first batch of pivotal trial data, and sales were nowhere near where they needed to be in order for the company to keep its head above water.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Bris­tol My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Drug man­u­fac­tur­ing gi­ant Lon­za taps Roche/phar­ma ‘rein­ven­tion’ vet as its new CEO

Lonza chairman Albert Baehny took his time headhunting a new CEO for the company, making it absolutely clear he wanted a Big Pharma or biotech CEO with a good long track record in the business for the top spot. In the end, he went with the gold standard, turning to Roche’s ranks to recruit Pierre-Alain Ruffieux for the job.

Ruffieux, a member of the pharma leadership team at Roche, spent close to 5 years at the company. But like a small army of manufacturing execs, he gained much of his experience at the other Big Pharma in Basel, remaining at Novartis for 12 years before expanding his horizons.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Covid-19 roundup: Ab­b­Vie jumps in­to Covid-19 an­ti­body hunt; As­traZeneca shoots for 2B dos­es of Ox­ford vac­cine — with $750M from CEPI, Gavi

Another Big Pharma is entering the Covid-19 antibody hunt.

AbbVie has announced a collaboration with the Netherlands’ Utrecht University and Erasmus Medical Center and the Chinese-Dutch biotech Harbour Biomed to develop a neutralizing antibody that can treat Covid-19. The antibody, called 47D11, was discovered by AbbVie’s three partners, and AbbVie will support early preclinical work, while preparing for later preclinical and clinical development. Researchers described the antibody in Nature Communications last month.