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.

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.