Clin­i­cal un­cer­tain­ty, price push­es UK's NICE to spurn Akcea's Waylivra

Echo­ing some of the con­cerns the FDA un­der­scored be­fore hand­ing its re­jec­tion, the UK’s cost-ef­fec­tive­ness watch­dog NICE has al­so for­sak­en Akcea’s drug, volane­sors­en.

On Fri­day, NICE is­sued a draft rec­om­men­da­tion re­ject­ing the fa­mil­ial chy­lomi­cron­aemia syn­drome (FCS) drug, cit­ing a myr­i­ad of rea­sons in­clud­ing un­cer­tain­ty un­der­pin­ning its clin­i­cal ev­i­dence as well as price. The drug is en­gi­neered to work by di­min­ish­ing the pro­duc­tion of ApoC-III, a pro­tein that reg­u­lates plas­ma triglyc­erides. FCS is caused by in­suf­fi­cient or im­paired func­tion of an en­zyme re­spon­si­ble for break­ing down triglyc­erides. It af­fects be­tween 55 and 110 peo­ple in Eng­land.

The drug, brand­ed Waylivra, was grant­ed con­di­tion­al ap­proval in Eu­rope last May. The FDA re­ject­ed the drug in Au­gust 2018, de­spite a pos­i­tive 12-8 rec­om­men­da­tion by an ex­pert ad­vi­so­ry pan­el. Ex­perts with­in and out­side the agency did, how­ev­er, spot­light the drug’s link with sud­den and un­ex­pect­ed drops in platelet counts as well as Akcea’s de­ci­sion to switch the dos­ing reg­i­men and platelet mon­i­tor­ing strat­e­gy, which were nev­er test­ed in clin­i­cal tri­als.

NICE, in its re­buff, al­so took is­sue with the dif­fer­ent dose. In ad­di­tion, the in­sti­tute high­light­ed the un­cer­tain­ty of the drug’s long term ef­fect, ac­knowl­edg­ing that it does show a short term ben­e­fit. NICE al­so said as­pects of the com­pa­ny’s eco­nom­ic mod­el­ing were “high­ly un­cer­tain,” and that the drug’s cost-ef­fec­tive­ness es­ti­mates are much high­er than what it con­sid­ers ac­cept­able.

The list price of volane­sors­en is £11,394 per sin­gle-use 285 mg sy­ringe. It is giv­en as a once-week­ly self-ad­min­is­tered in­jec­tion (de­creas­ing to once-fort­night­ly af­ter 3-months).

The NICE de­ci­sion is open for pub­lic con­sul­ta­tion un­til 24 Jan­u­ary 2020.

Akcea ex­pressed its dis­ap­point­ment at NICE’s de­ci­sion but pledged to keep work­ing with the in­sti­tute to en­sure Waylivra is en­dorsed.

The com­pa­ny, along with sis­ter drug­mak­er Io­n­is, de­vel­ops an­ti­sense drugs that are en­gi­neered to in­ter­rupt the pro­duc­tion of dis­ease-caus­ing pro­teins by tar­get­ing the spe­cif­ic cor­re­spond­ing mes­sen­ger RNA (mR­NA) that en­codes that pro­tein, there­by ma­nip­u­lat­ing pro­tein pro­duc­tion.

In an un­ex­pect­ed twist in Sep­tem­ber, a tri­fec­ta of se­nior ex­ec­u­tives run­ning Akcea — CEO Paula Soteropou­los, pres­i­dent Sarah Boyce and COO Jef­frey Gold­berg — left the Boston drug­mak­er in one fell swoop.

The board of the Io­n­is ma­jor­i­ty-owned spin­off swooped in to place Damien McDe­vitt — the chief busi­ness of­fi­cer of the Cal­i­for­nia com­pa­ny — in the in­ter­im chief spot. He will now be joined by for­mer GSK ex­ec­u­tive Alex Howarth who will take on the role of COO.

When the un­cer­e­mo­ni­ous de­par­tures were an­nounced ear­li­er this year, no ex­pla­na­tion was ini­tial­ly pro­vid­ed by the com­pa­ny. Even­tu­al­ly, it emerged that the ex­its were un­re­lat­ed to the launch­es of Akcea’s two drugs, Tegse­di and Waylivra, but were in fact dri­ven by the need to go shop­ping for deals to ex­pand its ar­se­nal of drugs-in-de­vel­op­ment as the next big tri­al read­outs are ex­pect­ed by 2021.

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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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.

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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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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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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Mer­ck wins a third FDA nod for an­tibi­ot­ic; Mereo tack­les TIG­IT with $70M raise in hand

Merck — one of the last big pharma bastions in the beleaguered field of antibiotic drug development — on Friday said the FDA had signed off on using its combination drug, Recarbrio, with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia. The drug could come handy for use in hospitalized patients who are afflicted with Covid-19, who carry a higher risk of contracting secondary bacterial infections. Once SARS-CoV-2, the virus behind Covid-19, infects the airways, it engages the immune system, giving other pathogens free rein to pillage and plunder as they please — the issue is particularly pertinent in patients on ventilators, which in any case are breeding grounds for infectious bacteria.