Ham­mered hard by a sur­pris­ing FDA re­jec­tion, Io­n­is spin­out Akcea of­fers an ex­pla­na­tion and then chops 1-in-10 staffers

Akcea re­port­ed in an SEC fil­ing Thurs­day af­ter­noon that it is chop­ping 10% of its work­force as a re­sult of the FDA’s sur­prise re­jec­tion of volane­sors­en, their drug for reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome. The pink slips were hand­ed out the same day at the biotech, which is run by CEO Paula Soteropou­los.

The com­pa­ny al­so of­fered its first ac­knowl­edg­ment of what went wrong, and how it hopes to deal with the cri­sis.

 

The com­pa­ny, a spin­out from Io­n­is $IONS — which still has a con­trol­ling in­ter­est in Akcea — had been ex­tra­or­di­nar­i­ly tight lipped about its af­flic­tions, ini­tial­ly un­will­ing to spell out any rea­sons for the re­jec­tion. A ma­jor­i­ty of FDA ex­perts on the pan­el re­view ac­tu­al­ly vot­ed in fa­vor of mar­ket­ing ap­proval. But in a sur­prise turn­around the agency it­self is­sued a com­plete re­sponse let­ter 10 days ago.

Fol­low­ing up with the com­pa­ny Thurs­day night, a spokesper­son for Akcea said that they have out­lined what went wrong. In a note to End­points News, she not­ed:

Re­gard­ing the CRL, we said in our pub­licly we­b­cast pre­sen­ta­tion at Wells Far­go yes­ter­day that the is­sues raised are con­sis­tent with what was dis­cussed at the Ad­Comm and are re­lat­ed to da­ta on man­ag­ing platelet lev­els and dos­ing.  We hope to ad­dress this with da­ta from our on­go­ing Open La­bel Ex­ten­sion study and/or Ear­ly Ac­cess Pro­gram, but we will not know the spe­cif­ic path for­ward or if this will be a suf­fi­cient ap­proach un­til we meet with the FDA. We hope to do that as soon as pos­si­ble. FCS is a de­bil­i­tat­ing dis­ease with no ther­a­peu­tic op­tions. We con­tin­ue to be­lieve WAYLIVRA demon­strates a pos­i­tive ben­e­fit/risk op­tion for peo­ple liv­ing with the dai­ly risk and bur­den of FCS.

This is where I’ll note that FDA com­mis­sion­er Scott Got­tlieb once ad­vo­cat­ed for dis­clos­ing those CRLs, on­ly to re­nege af­ter be­ing vot­ed in as FDA com­mis­sion­er last year. Had he fol­lowed through, there wouldn’t be any ques­tions about Akcea’s po­si­tion. None of that, though, has dam­aged his pop­u­lar­i­ty with the in­dus­try, which has cheered the agency’s re­spon­sive­ness in speed­ing up re­views.

In an in­ter­nal re­view put out ahead of the pan­el re­view, though, the FDA re­view­ers spelled out the prob­lems with the ap­pli­ca­tion.

The agency re­view­ers spot­light­ed a gen­er­al agree­ment that the drug has a clear­ly pos­i­tive ef­fect on reg­u­lat­ing plas­ma triglyc­eride for pa­tients with rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome. That’s a sur­ro­gate end­point for the dis­ease. But is the ben­e­fit re­al­ly worth the risk of bleed­ing, af­ter the drug was linked to sud­den and un­ex­pect­ed drops in platelet counts?

Ev­i­dent­ly not.

Akcea has a mar­ket cap of $2.3 bil­lion, which it will need to ride out the storm. The com­pa­ny had 100 staffers at the end of the year, but was swelling fast in an­tic­i­pa­tion of an ap­proval for this and one oth­er drug. Be­fore the re­jec­tion, Akcea said it had 270 em­ploy­ees. Fri­day morn­ing, the com­pa­ny was small­er.

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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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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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.

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.

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.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

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.