GSK aban­dons Ion­is' FDA-ready in­ot­ersen as new CEO sweeps out rare dis­ease ef­forts

Just three months af­ter Io­n­is spelled out pos­i­tive Phase III ef­fi­ca­cy da­ta on its top prospect in­ot­ersen, Glax­o­SmithK­line is sweep­ing their al­liance on the drug out the back door, along with rights to an­oth­er treat­ment they have rights to.

Em­ma Walm­s­ley, GSK CEO

It’s a stun­ning 11th hour re­ver­sal for Io­n­is $IONS, which says it now plans to push for an FDA ap­proval of in­ot­ersen on its own this fall as it thinks through whether it will com­mer­cial­ize or co-com­mer­cial­ize in the US on its own.

Ac­cord­ing to Io­n­is, GSK ex­ecs de­cid­ed to aban­don in­ot­ersen, de­signed to treat rare cas­es of TTR amy­loi­do­sis (AT­TR), with an ini­tial fo­cus on polyneu­ropa­thy due to hered­i­tary TTR amy­loi­do­sis (hAT­TR-PN). That drug is a close­ly-watched ri­val to Al­ny­lam’s lead drug patisir­an. Al­so out: IO­N­IS-FB-LRx, now at the thresh­old of Phase II for dry age-re­lat­ed mac­u­lar de­gen­er­a­tion, as GSK turns its back on rare dis­eases as part of its top-to-bot­tom R&D re­vamp un­der new CEO Em­ma Walm­s­ley.

“Our goals for in­ot­ersen are to max­i­mize its com­mer­cial suc­cess and op­ti­mize our com­mer­cial par­tic­i­pa­tion. To achieve these goals, we are ac­tive­ly con­sid­er­ing form­ing a com­mer­cial sub­sidiary to com­mer­cial­ize or co-com­mer­cial­ize in­ot­ersen in North Amer­i­ca, as well as oth­er op­tions. Our re­cent ex­pe­ri­ence build­ing a com­mer­cial sub­sidiary has pre­pared us for this op­por­tu­ni­ty. We have sub­stan­tial in­ter­est from po­ten­tial part­ners and are in dis­cus­sions with sev­er­al par­ties. We be­lieve that, to­geth­er with the right com­mer­cial part­ner, we can max­i­mize the com­mer­cial suc­cess of the drug world­wide,” said Sarah Boyce, chief busi­ness of­fi­cer of Io­n­is Phar­ma­ceu­ti­cals.

Sarah Boyce, Io­n­is CBO

Fil­ing for ap­proval and get­ting it are two dif­fer­ent things, though. Io­n­is’ late-stage ef­fort has al­so been flagged for se­ri­ous safe­ty con­cerns which could al­so ham­per its quest for an OK, as well as pos­si­bly sign­ing up a new com­mer­cial­iza­tion part­ner. Paul Mat­teis at Leerink swift­ly not­ed:

Nor­mal­ly in such a sit­u­a­tion like this in­vestors might wor­ry that there’s some not-yet seen-el­e­ment of the full da­ta that may be prob­lem­at­ic or dis­con­cert­ing. To this point — giv­en the well doc­u­ment­ed safe­ty is­sues as­so­ci­at­ed with In­ot­ersen (i.e., throm­bo­cy­tope­nia), GSK’s de­ci­sion may in­crease in­vestor scruti­ny on TTR­rx’s safe­ty pro­file as it is de­pict­ed in ad­di­tion­al full da­ta pre­sen­ta­tions this Fall (Amer­i­can Neu­ro­log­i­cal As­so­ci­a­tion, Oct. 15-17, oth­ers). Our mod­el cur­rent­ly as­sumes a 75%/25% AL­NY (MP) ver­sus IONS split, and while we are en­cour­aged that IONS’s plans for a YE17 MAA fil­ing are still on track, we are a bit con­cerned that GSK’s de­ci­sion may al­so put IONS be­hind the curve with re­spect to launch pre­pared­ness.

Stan­ley Crooke, Io­n­is CEO

Two oth­er al­liances will con­tin­ue. GSK is cur­rent­ly in Phase II with IO­N­IS-HB­VRx and IO­N­IS-HBV-LRx.

CEO Stan­ley Crooke not­ed that Io­n­is is now “ac­cel­er­at­ing the ex­pan­sion of our TTR pro­gram for pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis and the de­vel­op­ment of our LI­CA fol­low-on drug. Our ex­pe­ri­ence in the com­plet­ed Phase 3 NEU­RO-TTR study pro­vides im­por­tant in­for­ma­tion to aid in de­sign of a study in pa­tients with car­diomy­opa­thy due to TTR amy­loi­do­sis. We have al­ready iden­ti­fied a more po­tent and con­ve­nient LI­CA fol­low-on and we ex­pect de­vel­op­ment of the LI­CA drug to al­so pro­ceed rapid­ly.”

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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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.

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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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.

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.