Li­cens­ing As­traZeneca castoff, Bio­haven preps PhI­II ef­fort in new neu­rode­gen­er­a­tive in­di­ca­tion

Bio­haven $BHVN has added an­oth­er drug off of As­traZeneca’s shelf to its neu­rol­o­gy pipeline.

Com­ing with a pack­age of da­ta from four Phase I and three Phase IIa stud­ies, AZD3241 in­hibits myeloper­ox­i­dase, or MPO, a key dri­ver of in­flam­ma­to­ry process that’s ob­served in high lev­els in a range of brain dis­or­ders.

The biotech plans to test the drug — now re­named BHV3241 — in a Phase III tri­al as a treat­ment of mul­ti­ple sys­tem at­ro­phy, hav­ing seen pre­lim­i­nary re­sults from a small Phase IIa tri­al that showed a no­table bio­mark­er re­sponse and “nu­mer­i­cal im­prove­ments” on the Uni­fied MSA Rat­ing Scale af­ter 12 weeks of treat­ment.

Vlad Coric

That, ac­cord­ing to Bio­haven, makes the case for a po­ten­tial first-in-class treat­ment for the rare neu­rode­gen­er­a­tive dis­ease that of­ten kills pa­tients af­ter five to sev­en years of tremors, slow move­ment, mus­cle rigid­i­ty and atax­ia.

“These da­ta, cou­pled with a fa­vor­able safe­ty pro­file and ev­i­dence that BHV3241 re­duced in­tra­cel­lu­lar ag­gre­gates of al­pha-synu­cle­in, sup­pressed mi­croglial ac­ti­va­tion and res­cued neu­rode­gen­er­a­tion in MSA an­i­mal mod­els, sup­port our be­lief that BHV3241 has po­ten­tial as a dis­ease-mod­i­fy­ing treat­ment in Mul­ti­ple Sys­tem At­ro­phy,” said pro­fes­sor Wern­er Poewe at Aus­tria’s Med­ical Uni­ver­si­ty Inns­bruck in a state­ment.

Wern­er Poewe

As­traZeneca is gain­ing some shares in Bio­haven in ad­di­tion to an un­spec­i­fied up­front cash pay­ment for the as­set, as well as oth­er mile­stones and up to dou­ble-dig­it roy­al­ties should the drug make it to the mar­ket. It has al­so promised not to pur­sue any of the oth­er MPO in­hibitors in its pipeline for neu­ro­log­ic dis­eases for five years as Bio­haven ex­plores oth­er po­ten­tial in­di­ca­tions for BHV3241.

This is the sec­ond as­set the UK phar­ma gi­ant has hand­ed off to the Yale spin­out, fol­low­ing a $210 mil­lion deal that in­volved an NM­DA an­tag­o­nist.

The deal adds to Bio­haven’s late-stage pipeline, which is cur­rent­ly dom­i­nat­ed by rimegepant, the CGRP mi­graine ther­a­py it in-li­censed from Bris­tol-My­ers Squibb. Ex­ecs are count­ing on rimegepant and tri­grilu­zole — a re­for­mu­la­tion of an old ALS drug called rilu­zole that’s al­so be­ing stud­ied for OCD and atax­ia — for near-term FDA ap­pli­ca­tions, putting their ini­tial fo­cus on de­pres­sion and anx­i­ety on the back­burn­er for now.

“The in-li­cens­ing of BHV3241 ex­pands Bio­haven’s port­fo­lio of in­no­v­a­tive, late-stage prod­uct can­di­dates for the treat­ment of neu­ro­log­ic and psy­chi­atric dis­ease in­di­ca­tions, “ said CEO and Yale pro­fes­sor Vlad Coric.

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.