No­var­tis builds its case for a new sick­le cell drug, eye­ing a de­layed FDA fil­ing in ‘19

Re­searchers at No­var­tis have tak­en an­oth­er crack at the da­ta they col­lect­ed in a Phase II study of their sick­le cell dis­ease drug crizan­l­izum­ab, build­ing their case ahead of a loom­ing reg­u­la­to­ry fil­ing.

Check­ing on the rate of dis­ease-re­lat­ed pain crises among pa­tients, No­var­tis scored their drug with 35.8% of the pa­tients on drug avoid­ing a cri­sis dur­ing the pre­vi­ous year. That’s more than twice the 16.9% rate tracked in the place­bo arm.

All of the pa­tients in the SUS­TAIN study had ex­pe­ri­enced at least 2 episodes of what’s called a va­so-oc­clu­sive cri­sis in the year be­fore they joined the study, putting them at high risk for more.

Ab­dul­lah Kut­lar

“The un­pre­dictable, in­tense painful crises that pa­tients with sick­le cell dis­ease ex­pe­ri­ence are the hall­mark of the dis­ease and the pri­ma­ry cause of hos­pi­tal­iza­tions in this pa­tient pop­u­la­tion,” said Ab­dul­lah Kut­lar, the pri­ma­ry au­thor of the SUS­TAIN analy­sis. “I am en­cour­aged that re­sults from this post hoc analy­sis of SUS­TAIN study da­ta found that crizan­l­izum­ab could sub­stan­tial­ly de­lay or pre­vent these crises, which al­so may mean less or­gan dam­age in the long run.”

These pain crises oc­cur when sick­le-shaped cells block the flow of blood in small blood ves­sels. And it’s the most ob­vi­ous symp­tom of the dis­ease. No­var­tis picked up the drug in 2016 in their ac­qui­si­tion of Se­lexys Phar­ma­ceu­ti­cals in a deal val­ued at up to $665 mil­lion.

Out­lin­ing the re­sponse in the high-dose arm 2 years ago, in­ves­ti­ga­tors re­port­ed in the New Eng­land Jour­nal of Med­i­cine the me­di­an rate of crises per year was “1.63 with high-dose crizan­l­izum­ab ver­sus 2.98 with place­bo (in­di­cat­ing a 45.3% low­er rate with high-dose crizan­l­izum­ab, P=0.01). The me­di­an time to the first cri­sis was sig­nif­i­cant­ly longer with high-dose crizan­l­izum­ab than with place­bo (4.07 vs. 1.38 months, P=0.001), as was the me­di­an time to the sec­ond cri­sis (10.32 vs. 5.09 months, P=0.02). The me­di­an rate of un­com­pli­cat­ed crises per year was 1.08 with high-dose crizan­l­izum­ab, as com­pared with 2.91 with place­bo (in­di­cat­ing a 62.9% low­er rate with high-dose crizan­l­izum­ab, P=0.02).”

The low dose did not de­liv­er a sta­tis­ti­cal­ly sig­nif­i­cant re­sponse.

No­var­tis says now that it’s look­ing at a 2019 FDA fil­ing, which rep­re­sents a de­lay from the 2018 time­line ex­ecs had pro­vid­ed ear­li­er af­ter the ini­tial read­out ar­rived.

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.

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.

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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