Can tiny Mus­tang Bio com­pete on the CAR-T front with gi­ants like No­var­tis and Gilead? Man­ny Litch­man aims to find out

Now that No­var­tis and Gilead have picked up the first two ap­provals for CAR-T drugs, you can ex­pect to see ri­vals com­ing along who will probe for any weak­ness­es in their ap­proach, look­ing to com­pete on the next wave of per­son­al­ized cell ther­a­pies head­ed through the clin­ic.

The small crew at Mus­tang Bio — $MBIO, up 11% to­day — has some big am­bi­tions on that score, and to­day they’re adding some man­u­fac­tur­ing ca­pac­i­ty that the com­pa­ny be­lieves will be cen­tral to its abil­i­ty to even­tu­al­ly com­pete with these two gi­ants.

Man­ny Litch­man

Mus­tang has inked a lease deal with the UMass Med­i­cine Sci­ence Park in Worces­ter, MA, where they ex­pect to be­gin pro­duc­tion of their CAR-Ts next year. And they’ll be aim­ing to make these cell ther­a­pies for ear­ly-stage stud­ies for glioblas­toma and acute myeloid leukemia and a grow­ing sta­ble of pre­clin­i­cal ef­forts.

Look­ing for com­pet­i­tive ad­van­tages at this stage of the game will re­ly quite a lot on a “more stream­lined, more cost ef­fec­tive man­u­fac­tur­ing ap­proach,” CEO Man­ny Litch­man tells me. “Every­thing re­al­ly has cost in mind,” he adds, siz­ing up the first two ther­a­pies that come with hefty six-fig­ure price tags.

“Look in the black box of man­u­fac­tur­ing,” he says, and you can see “dozens of pa­ra­me­ters that can vary.”

There’s more reengi­neer­ing work to be done to make CAR-Ts bet­ter at com­bat­ting can­cers like glioblas­toma and AML, he adds. In­ves­ti­ga­tors, for ex­am­ple, have been bal­anc­ing the im­pact of quick ver­sus slow­er ac­tion of the ther­a­peu­tic — slow­er ac­tion ap­pears to be more durable — along with a mul­ti­tude of com­bi­na­tion ap­proach­es that need to be ex­plored.

There’s enough po­ten­tial in these new ap­proach­es to of­fer an open­ing for a com­pa­ny like Mus­tang to step in and ex­ploit new op­por­tu­ni­ties, he be­lieves.

Litch­man left the helm at Arv­inas, a pro­tein degra­da­tion biotech spun out of the lab of Yale’s Craig Crews, to take the lunge at CAR-T. It’s a field he knows some­thing about. As for­mer head of on­col­o­gy BD at No­var­tis, Litch­man was present at the cre­ation of the Penn/No­var­tis deal that set the phar­ma gi­ant down the path to­ward an his­toric CAR-T ap­proval. He was pro­gram head of CTL019 for awhile. And he’s fol­low­ing in much the same path that the pi­o­neers — along with Kite and Juno — did, let­ting the sci­en­tif­ic founders do the ear­ly-stage re­search work that will be used to set up the fast-paced piv­otal de­vel­op­ment pro­grams to come.

Stephen For­man

In Mus­tang’s case, that in­volves Stephen For­man’s lab­o­ra­to­ry at City of Hope Na­tion­al Med­ical Cen­ter and top re­searchers at the Fred Hutchin­son Can­cer Re­search Cen­ter, where Oliv­er Press and Bri­an Till have been build­ing a T cell ther­a­py which ex­press­es a CD20-spe­cif­ic chimeric anti­gen re­cep­tor. That work has ex­pand­ed Mus­tang’s pipeline to 6 clin­i­cal and pre­clin­i­cal ef­forts.

The Cal­i­for­nia In­sti­tute for Re­gen­er­a­tive Med­i­cine, rest­less­ly look­ing to make a clin­i­cal mark af­ter years in­vest­ing in labs, just days ago pro­vid­ed a $12.8 mil­lion grant to the City of Hope for the Phase I glioblas­toma study. And that comes on top of a $94.5 mil­lion raise in Feb­ru­ary from a pri­vate place­ment arranged by its par­ent com­pa­ny Fortress, which is build­ing a port­fo­lio of biotechs.

Try­ing to leapfrog in­to a clin­i­cal ri­val­ry in a com­plex are­na like this will cost much, much more than that. And Litch­man tells me he has plans to raise some­where be­tween $60 mil­lion and $100 mil­lion more next year.

Right now, Mus­tang has 5 full timers, a tiny boat­load of staffers com­pared to the jug­ger­nauts crewed by No­var­tis and Gilead’s Kite. Juno al­so has a much larg­er op­er­a­tion look­ing to make a come­back af­ter their lead ther­a­py was de­stroyed by its lethal tox­i­c­i­ty. But Litch­man plans to up that to about 20 over the next year, while the in­de­pen­dent sci­en­tists con­tin­ue to do the heavy lift­ing in the ear­ly stud­ies.

This is one race that Litch­man says is still very much just be­gin­ning. And it won’t be dom­i­nat­ed by a hand­ful of lead­ers.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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