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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Scott Struthers, Crinetics CEO

Cri­net­ics spins out ra­dio­phar­ma ef­forts in­to a new com­pa­ny, high­light­ing the grow­ing field­'s al­lure

Largely known for its nonpeptide small molecule research, Crinetics has been keeping its radiopharma work comparatively under wraps. But that changed Monday afternoon as the California biotech spun out a new company focused solely on the burgeoning field.

Crinetics launched Radionetics after the closing bell Monday, the company announced, seeding the new entity with $30 million raised from 5AM Ventures and Frazier Healthcare Partners. Radionetics will start with its own radiopharma-centric platform and a pipeline of 10 programs aimed at solid tumors.

Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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