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

Kunwoo Lee was a graduate student at UC-Berkeley when gene editing pioneer Jennifer Doudna — who happened to work in the same building where he studied — published a paper on CRISPR/Cas9. So he did what any aspiring bioengineer would do: He ran to her lab, and grabbed a postdoc there.

“We started really thinking about the future coming (for) gene therapy and gene editing,” he said.

Lee’s research with Doudna led him to co-found a small San Francisco-based biotech called GenEdit in 2016, the same year he graduated. After five quiet years, the team is now unveiling a $26 million Series A round with support from some big names like Eli Lilly to fund their work on one of the most pressing challenges in gene therapy: what Lee calls the “delivery problem.”

When Thomas Braun was starting out as a young professor at Germany’s University of Würzburg in 1997, he decided to try his hand at a new field: heart regeneration, a sci-fi-esque premise that could offer a way to treat patients recovering from a heart attack. He thought it would take a few years before they got results.

“We were,” he acknowledges now, “rather naïve.”

But on Thursday, after two and a half decades of fitful starts and abandoned leads, Braun and a team of researchers at the Max Planck Institute showed that they could reprogram heart cells in mice and get the animals to regenerate cardiac tissue after a heart attack. The breakthrough, published in Science, adds new evidence that it will eventually be possible to help patients recover muscle lost in heart attacks and gives another boon to anti-aging researchers who want to one day apply these rejuvenation techniques across much of the body.

In an attempt to get its house of accelerated approvals in order, the FDA is holding its second adcomm of 2021 to review cancer drugs that won accelerated approvals but failed to confirm clinical benefit in subsequent trials or have taken a long time to read those data out.

On Dec. 2, the FDA’s Oncologic Drugs Advisory Committee will review two accelerated approvals from Secura Bio’s Farydak (panobinostat), a third-line multiple myeloma drug, and Acrotech Biopharma’s Marqibo, as a third-line drug for adult patients with Philadelphia chromosome negative acute lymphoblastic leukemia. Both drugs have been marketed for more than five years under their accelerated approvals but have recorded negligible sales in their respective indications in recent years.

Oxford Biomedica has landed some new cash.

The British gene and cell therapy biotech announced Wednesday it has secured an investment “just over” £50 million from the Serum Institute of India’s investment arm. Funds are expected to help Oxford Biomedica build out its 84,000-square-foot manufacturing facility and bring online several independent suites by mid-2023.

“Serum Institute of India has played a big part in the fight against COVID-19, as have we, and we look forward to a strong and collaborative relationship,” CEO John Dawson said in a statement. “This investment will allow us to expand capacity at Oxbox at a time when our business development pipeline has never looked stronger.”