Aaron Kantoff (Medicxi)

A Medicxi-backed start­up looks to tack­le treat­ment-re­sis­tant blood can­cers, and it's go­ing af­ter AML first

In the hard­est-to-treat blood can­cers, re­sis­tance to ther­a­py is an ex­is­ten­tial prob­lem for drug de­vel­op­ers look­ing to per­ma­nent­ly stave off tu­mors. A small biotech is chas­ing an emerg­ing path­way to stop tu­mors’ abil­i­ty to re­sist treat­ment, and its work has caught the eye of a cou­ple of big-name in­vestors.

Kurome Ther­a­peu­tics snared a $15 mil­lion Se­ries A round it will use to iden­ti­fy and de­vel­op a lead pro­gram from its plat­form look­ing at dual in­hibitors of the IRAK1/4 sig­nal­ing path­way and FLT3 pro­tein re­cep­tors on heme blasts to crack treat­ment-re­sis­tant tu­mors, the biotech said Thurs­day.

Daniel Star­czynows­ki

The com­pa­ny’s work is based on re­search from Daniel Star­czynows­ki, a co-leader of the hema­to­log­ic ma­lig­nan­cies pro­gram at Cincin­nati Chil­dren’s Can­cer and Blood Dis­eases In­sti­tute, and one of the lead­ing re­searchers work­ing on IRAK, Kurome said. That path­way has been im­pli­cat­ed in reg­u­lat­ing tu­mor re­sis­tance, and Kurome thinks its dual ap­proach could have an ef­fect on a range of blood can­cers as well as some sol­id tu­mors with dys­reg­u­lat­ed IRAK 1/4.

Kurome ex­plic­it­ly called out acute myeloid leukemia as a pri­ma­ry tar­get for IRAK 1/4 ther­a­pies, with Curis the fur­thest along with IRAK4 in­hibitor CA-4948 in AML and myelodys­plas­tic syn­dromes. Curis un­veiled ex­pand­ed Phase I da­ta in May show­ing CA-4948 re­duced the num­ber of im­ma­ture blood cells, called blasts, in the bone mar­row for eight of nine evalu­able pa­tients. The drug post­ed four ob­jec­tive re­spons­es, in­clud­ing one com­plete re­sponse, one com­plete re­mis­sion with min­i­mal resid­ual dis­ease and two bone mar­row com­plete re­spons­es.

Kurome launched last year with seed fund­ing from Cin­cyTech based on Star­czynows­ki’s re­search at Cincin­nati Chil­dren’s Hos­pi­tal Med­ical Cen­ter in col­lab­o­ra­tion with NCATS. Ear­ly pre­clin­i­cal da­ta showed some promise for the biotech’s dual in­hibitor plat­form in shut­ting down adap­tive re­sis­tance, which caught the eye of Medicxi and Affin­i­ty As­set Ad­vi­sors, which co-led the round.

Ac­cord­ing to Medicxi ven­ture part­ner Aaron Kantoff, the ven­ture firm was at­tract­ed to the con­cept by Curis’ win­ning ear­ly-stage da­ta as well as Star­czynows­ki’s name recog­ni­tion as a top ex­pert in the space. With the right sci­ence and guid­ance, Kantoff said, Kurome could have an AML win­ner like Genen­tech and Ab­b­Vie’s Ven­clex­ta wait­ing in the wings.

“To be blunt, the space has evolved very quick­ly where we al­ready know FLT3 bi­ol­o­gy is well un­der­stood, so blend­ing that bi­ol­o­gy with the emerg­ing val­i­da­tion of IRAK1/4 … seemed like a good idea to gath­er syn­er­gy in a mul­ti­fac­to­r­i­al dis­ease like AML,” Kantoff said.

As part of Medicxi’s lat­est fund­ing round, Kantoff will join the com­pa­ny’s board and help steer it ahead to a lead pro­gram, which the biotech ex­pects to re­veal by the end of the year. Daniel Heller, gen­er­al part­ner and chief in­vest­ment of­fi­cer at Affin­i­ty, will al­so sit on the board.

With a lead pro­gram tapped, Kurome ex­pects to en­ter IND en­abling stud­ies by ear­ly 2022, Kantoff said. That means the team of just four will look to ex­pand pri­or to that hap­pen­ing, but Kantoff was mum on ex­act­ly what that ex­pan­sion would look like or when to ex­pect it.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

The IPO 4-1-1: Four fil­ings, a pric­ing and a with­draw­al head­line this week's Nas­daq ac­tion as raise ap­proach­es $7.5B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another week, another horde of biotechs is doing the Nasdaq dance.

This week saw four companies file their SEC paperwork ahead of expected debuts, another hit Nasdaq on Friday and a sixth formally withdrew its bid to go public. Aerovate Therapeutics, Ocean Biomedical and Acumen Pharmaceuticals all penciled in initial raises of $100 million, while Dermata Therapeutics is estimating a modest $18 million raise.

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Keiichi Fukuda, Heartseed CEO

Fresh off $598M deal with No­vo Nordisk, a Japan­ese stem cell com­pa­ny is on its way to the clin­ic with a dif­fer­ent ap­proach to treat­ing heart fail­ure

A common approach to treating heart failure with induced pluripotent stem cells involves grafting sheets of cells onto the surface of the heart to improve vascularization and blood flow. It’s the easiest method of transplantation — but you run the risk of not making an electrical connection with the heart and the cells not synchronizing with the patient’s heart muscle.

So what if you could inject spherical clusters of heart cells directly into the heart muscle wall? For Heartseed, that’s now the $37 million question.

Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

An­a­lysts hound Bio­gen with ques­tions over Aduhelm's $56K price point as news of Alzheimer's OK rip­ples through glob­al mar­kets

It’s been nearly 24 hours since the FDA handed down its controversial decision to approve Biogen’s Aduhelm drug for Alzheimer’s disease, and reactions from around the industry are pouring in as analysts try to size up the ramifications of Monday’s news.

Biogen hosted an investor call Tuesday morning, providing a forum for a number of topics that have been top of mind. The call came as the news of Aduhelm’s approval in the US rippled through global markets, while $BIIB stock soared to around $380 apiece (up from $286 on Friday), as Biogen’s partners at Eisai seek new approvals in Asia.

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Stuart Schreiber (Maria Nemchuk, Broad Institute)

Po­laris, Ab­b­Vie, Bay­er back Stu­art Schreiber's hunt to turn can­cer treat­ments in­to cures

What happens to cancer cells that don’t die after powerful treatment? Scientists have long known that in all but a handful of cases, they don’t vanish even if the patients appear cancer-free — instead lurking somewhere, unreachable and undetectable and preparing, like a vanquished movie villain at the start of a sequel, for a return.

Research over the last decade began to suggest there was something different about these survivor cells. It wasn’t just that they might mutate. They reprogrammed themselves, like an operating system sealing itself off after too many incorrect passwords, and entered a protective state that other cells in the body have been known to enter when threatened.

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