Siddhartha Mukherjee (Evan Agostini/Invision/AP)

With lessons learned from Vor, Sid­dhartha Mukher­jee takes a dif­fer­ent route to tar­get sol­id tu­mors with start­up Myeloid

Sid­dhartha Mukher­jee is a busy man these days.

The Pulitzer Prize-win­ning au­thor, Co­lum­bia pro­fes­sor, on­col­o­gist, hema­tol­o­gist and lead­ing can­cer re­searcher in 2016 launched Vor, a Cam­bridge, MA-based start­up fo­cused on a new can­cer treat­ment in which cell sur­face anti­gens such as CD33 are re­moved from hematopoi­et­ic stem cells. They can then un­leash a CAR-T ther­a­py — made from T cells in a pa­tient’s blood and ge­net­i­cal­ly en­gi­neered to hunt for the anti­gens — to treat can­cer us­ing the body’s own im­mune sys­tem with­out at­tack­ing healthy cells that would’ve shared those anti­gens.

Now, Mukher­jee is adding a new start­up to his plate, still fo­cused on treat­ing can­cer us­ing the im­mune sys­tem. But this time, his fo­cus is on types of can­cers that have demon­strat­ed they can’t be cured by the CAR-T cell tech­nol­o­gy he pri­or­i­tizes at Vor.

This start­up, al­so based in Cam­bridge, is called Myeloid Ther­a­peu­tics, named for the spe­cif­ic cells which play a crit­i­cal role in a new ther­a­py found­ed by Mukher­jee and renowned bio­chemist Ronald Vale. The two are backed by over $50 mil­lion in Se­ries A fund­ing from around the bio­phar­ma in­dus­try, led by New­path Part­ners out of Boston.

Mukher­jee told End­points News in an in­ter­view that us­ing myeloid cells to tar­get can­cers — most­ly those in sol­id tu­mors — has the po­ten­tial to meet an “enor­mous” un­met med­ical need.

“Most sol­id can­cers such as ovar­i­an can­cer or col­orec­tal can­cer or esophageal can­cer for what­ev­er rea­son are not amenable to CAR-T cell ther­a­py or to so-called check­point in­hibitor ther­a­pies, which ac­ti­vat­ed T cells against these can­cers,” he said. “We now know a lot about why, and that’s be­cause when you look at these can­cers, even af­ter you treat them with these (ther­a­pies), you find that they form a kind of shell and re­sis­tance shell around the can­cer … which ex­clude T cells from en­ter­ing the tu­mor.”

It’s al­most like the shell on the out­side of a Star Wars bat­tle­ship, Mukher­jee said.

Ronald Vale

This is where myeloid cells come in­to play. Un­like T cells, myeloid cells are ac­tu­al­ly “in­trin­si­cal­ly de­signed” to pen­e­trate sol­id can­cer tu­mors, he said, as if they have a free pass to in­vade the mass and sur­vey what’s hap­pen­ing in­side of it.

Mukher­jee and Vale dis­cov­ered that they could ge­net­i­cal­ly en­gi­neer re­cep­tors in the myeloid cells to not on­ly pen­e­trate a tu­mor, but to rec­og­nize the tu­mor and be ac­ti­vat­ed by the tu­mor it­self. In oth­er words, the tech­nol­o­gy bridges the gap be­tween the hu­man body’s in­nate im­mune sys­tem, which can gen­er­al­ly scan the body for virus­es and the like, and the im­mune sys­tem’s ca­pac­i­ty to rec­og­nize and lat­er at­tack can­cer cells.

Thus be­came Myeloid There­apeu­tics.

“Those were the two cru­cial in­sights that drove this com­pa­ny and the ther­a­pies that this com­pa­ny is pro­duc­ing,” Mukher­jee said. “The fact that you can take the in­nate im­mune sys­tem, which is not de­signed to rec­og­nize one par­tic­u­lar virus or mi­crobe or can­cer, but en­gi­neer the im­mune sys­tem to rec­og­nize can­cer and be­come ac­ti­vat­ed by the can­cer … We’re en­abling the most an­cient part of the im­mune sys­tem to be­come can­cer-spe­cif­ic and can­cer-ac­ti­vat­ed.”

Daniel Getts

Myeloid will ded­i­cate much of the ini­tial $50 mil­lion fund­ing to­ward ini­ti­at­ing clin­i­cal tri­als for its two main pro­grams, which tar­get T cell lym­phoma, glioblas­toma and oth­er sol­id tu­mors. The team will al­so con­tin­ue to de­sign and ad­vance a broad pipeline of tar­get­ed myeloid cell ther­a­pies, in­clud­ing primed myeloid cells, myeloid mul­ti-spe­cif­ic en­gagers and oth­er de­vel­op­ment can­di­dates cre­at­ed with Myeloid’s nov­el mR­NA de­liv­ery tech­nolo­gies.

The com­pa­ny ex­pects to en­ter the clin­ic with its two lead pro­grams in glioblas­toma and T cell lym­phoma in 2021.

Myeloid CEO Daniel Getts told End­points that through­out his ca­reer in the biotech in­dus­try, he’s been in­volved with oth­er ven­tures that looked at the use of T cells in fight­ing can­cer­ous tu­mors. But be­fore now, it’s seemed like the tech­nolo­gies and can­cer reme­dies avail­able were dis­joint­ed, he said. Where Myeloid hopes to be dif­fer­ent is that it com­bines the con­cepts of those past ther­a­pies in­to a sin­gle ther­a­peu­tic through the myeloid-cell mech­a­nism.

“I think the pow­er of what we have is un­sur­passed right now. And, you know, we’re ben­e­fi­cia­ries,” Getts said. “We’re stand­ing on the shoul­ders of a lot of these oth­er gene en­gi­neer­ing com­pa­nies be­cause they’re show­ing the world what’s pos­si­ble.”

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.

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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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.

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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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

Rajesh Devraj, Rectify Pharmaceuticals CEO

At­las backs a long­time Ver­tex em­ploy­ee’s quest to bring CF suc­cess to nu­mer­ous oth­er dis­eases

One of Vertex’s longest-tenured employees believes he can take the biotech’s biggest medical and scientific accomplishments and use it to develop treatments for more than just cystic fibrosis.

Three years ago, Jonathan Moore, a scientist and then executive at Vertex from 1990 to 2018, founded a company to develop treatments for diseases that, like CF, are caused by mutations in a “super family” of proteins known as ABC transporters.

Yao-Chang Xu, Abbisko Therapeutics founder and CEO

Qim­ing-backed Ab­bisko makes $200M+ Hong Kong de­but, as a SPAC and Agenus spin­out al­so price on Nas­daq

Three new entities priced their public debuts late Thursday and early Friday, including a SPAC, a traditional Nasdaq IPO and a Chinese biotech joining the Hong Kong Index.

Shanghai-based Abbisko Therapeutics raised the most money of the triumvirate, garnering $226 million in its Hong Kong debut and pricing at HK$12.46, or roughly $1.60 in US dollars. The blank check company followed up with a $150 million raise, while MiNK Therapeutics priced on Nasdaq at $12 per share and a $40 million raise.