Chemo­Cen­tryx gets new PDU­FA date af­ter ma­jor amend­ment; Nim­i­um launch­es with eye on obe­si­ty

Fol­low­ing talks with the FDA, Chemo­Cen­tryx on Mon­day filed an amend­ment to its ap­pli­ca­tion for ava­co­pan as a treat­ment of an­ti-neu­trophil cy­to­plas­mic au­toan­ti­body (AN­CA)-as­so­ci­at­ed vas­culi­tis, re­sult­ing in a new PDU­FA goal date of Oct. 7.

Thomas Schall

While the com­pa­ny did not ex­plain what was con­tained in the amend­ment fil­ing, its stock spiked by more than 10% on the news. How the agency will ul­ti­mate­ly de­cide on the drug re­mains a mys­tery. The agency’s Arthri­tis Ad­vi­so­ry Com­mit­tee in May vot­ed 9-9 on whether the ef­fi­ca­cy da­ta from a small Phase III tri­al sup­port ap­proval of ava­co­pan, 10-8 in fa­vor of the drug’s safe­ty pro­file, and 10-8 that the ben­e­fit-risk pro­file is ad­e­quate to sup­port ap­proval.

There was a con­tentious de­bate at the meet­ing over whether the Phase III tri­al was enough to sup­port the ap­proval as the FDA raised con­cerns about the sta­tis­ti­cal analy­ses of the da­ta in the tri­al and what ef­fect the use of glu­co­cor­ti­coids on top of cy­clophos­phamide or rit­ux­imab in both treat­ment arms had on the ef­fi­ca­cy of ava­co­pan.

“We ap­pre­ci­ate the op­por­tu­ni­ty to put ad­di­tion­al da­ta and in­for­ma­tion be­fore the Agency, in­for­ma­tion which we be­lieve ad­dress­es many of the is­sues raised at the Ad­vi­so­ry Com­mit­tee meet­ing,” Chemo­Cen­tryx CEO Thomas Schall said in a state­ment. — Zachary Bren­nan

Biotech up­start launch­es with an eye on ‘ex­cess caloric in­take’

A new biotech fo­cused on obe­si­ty has launched in Mon­tre­al.

The up­start is fo­cused on the work of Marc Pren­t­ki, the prin­ci­pal sci­en­tist at the Cen­tre de recherche du Cen­tre Hos­pi­tal­ier de l’Uni­ver­sité de Mon­tréal. He’s cred­it­ed with dis­cov­er­ing that “an en­zyme, glyc­erol-3-phos­phate phos­phatase (G3PP), plays a key role in sug­ar, fat and en­er­gy me­tab­o­lism.”

The com­pa­ny, dubbed Nim­i­um, has li­censed IP cov­er­ing agents that ac­ti­vate the hu­man G3PP en­zyme.

Philippe Walk­er, an As­traZeneca vet, is lead­ing the start­up. He notes: “From obe­si­ty to type 2 di­a­betes, and oth­er dis­eases such as chron­ic kid­ney dis­ease and non-al­co­holic fat­ty liv­er dis­ease, there is a glob­al epi­dem­ic re­lat­ed to the tox­ic ef­fects of over­nu­tri­tion. Through Nim­i­um, we aim to ad­dress this health cri­sis by de­vel­op­ing a new ther­a­peu­tic ap­proach that will re­duce the ef­fects of ex­cess caloric in­take, and that should pro­mote healthy ag­ing.” — John Car­roll

Mimetas and Roche part­ner for hu­man dis­ease mod­els

Roche has en­tered in­to a col­lab­o­ra­tion with Nether­lands-based Mimetas to de­vel­op hu­man dis­ease mod­els for in­flam­ma­to­ry bow­el dis­ease and he­pati­tis B virus in­fec­tions, the com­pa­nies said in a re­lease Tues­day.

Mimetas will de­vel­op tis­sue-based mod­els in OrganoPlate, its or­gan-on-chip plat­form that in­creas­es the pre­dictabil­i­ty of bio­mark­ers and re­duces an­i­mal use in test­ing. Roche, in re­turn, will get ac­cess to the tech­nol­o­gy, dis­ease mod­els and re­sults, as well as an op­tion to ex­clu­sive­ly li­cense mod­els and as­says for drug dis­cov­ery. Mimetas is el­i­gi­ble for both up­front and mile­stone pay­ments from Roche.

“The col­lab­o­ra­tion with Roche lever­ages our on­go­ing fo­cus on de­vel­op­ing pre­dic­tive, phe­no­typ­ic mod­els, pre­ced­ed by nu­mer­ous suc­cess­ful projects over the last eight years,” Mimetas CEO Jos Joore said in a press re­lease. “We will lever­age our dis­ease mod­el­ing ex­per­tise in our world-lead­ing OrganoPlate plat­form to gain nov­el in­sights in IBD and HBV.” — Josh Sul­li­van

So­sei en­lists In­ve­ni­AI in AI-pow­ered and GPCR-fo­cused drug dis­cov­ery col­lab­o­ra­tion

Miles Con­greve

So­sei Group is part­ner­ing with In­ve­ni­AI to de­vel­op new ther­a­peu­tic prod­uct con­cepts for im­mune dis­eases where an AI- and ML-based ap­proach can be used to gen­er­ate com­pelling ev­i­dence for the role of G-pro­tein cou­pled re­cep­tors (GPCRs) in rel­e­vant im­munomod­u­la­to­ry path­ways. The goal is to use these tar­gets as a ba­sis for struc­ture-based drug de­sign to gen­er­ate nov­el com­pounds that could im­prove re­spons­es to ex­ist­ing im­munother­a­pies.

“GPCRs rep­re­sent an im­por­tant class of phar­ma­ceu­ti­cal tar­gets, and it is es­ti­mat­ed that about 30% of known drugs have their mode of ac­tion through a rel­a­tive­ly small num­ber of GPCRs,” So­sei Hep­tares CSO Miles Con­greve said in a state­ment. “This leaves a sig­nif­i­cant num­ber of re­main­ing GPCR tar­gets that could be at­trac­tive for ther­a­peu­tic in­ter­ven­tion. In­ve­ni­AI’s Al­phaMeld plat­form will de­con­vo­lute the bi­ol­o­gy of dis­ease and in­dus­tri­al­ize tar­get dis­cov­ery by rapid­ly un­rav­el­ling con­nec­tions be­tween GPCR tar­gets and dis­eases with high un­met need.” — Zachary Bren­nan

Leo Phar­ma gets new da­ta part­ner af­ter April CRL

Look­ing to re­bound from an April CRL, Leo Phar­ma has en­list­ed a new part­ner to help it con­tin­ue pur­su­ing der­ma­tol­ogy-re­lat­ed dis­eases.

The Dan­ish biotech has en­list­ed Waltham, MA-based X-Chem in a part­ner­ship that will ap­ply the lat­ter’s plat­form to help iden­ti­fy po­ten­tial new tar­gets. Leo will re­tain the op­tion for ex­clu­sive glob­al rights for any med­i­cine that comes out of the agree­ment, as well as all rights to com­mer­cial­iza­tion.

Leo will be fur­ther re­spon­si­ble for all pre­clin­i­cal and clin­i­cal R&D. Fi­nan­cial terms of the deal were not dis­closed.

“X-Chem’s DEL plat­form tech­nol­o­gy is a pow­er­ful ap­proach giv­ing us in­creased ca­pa­bil­i­ties to feed our in­no­v­a­tive project port­fo­lio,” Leo head of ear­ly R&D Thorsten Thor­mann said in a state­ment. — Max Gel­man

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.