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.”

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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Jean-Christophe-Hyvert, Lonza

Lon­za look­ing to build on 'd­if­fer­en­ti­at­ed ad­van­tage' in Covid-19, CD­MO mar­ket­place in 2021

It’s not new for Lonza, the Swiss CDMO nearing its quasquicentennial anniversary, to be in the upper echelon of the biotech manufacturing industry.

But 2020 — as it was for many CDMOs — was a special year even by Lonza’s standards. The company inked a deal to produce 1 billion worldwide doses of Moderna’s Covid-19 vaccine and tapped pharma vet Pierre-Alain Ruffieux to lead its operations, moves which have allowed Lonza to make a myriad of other deals that will continue to ramp up its global production capacity.

News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.