Park­er In­sti­tute backs an up­start biotech look­ing to de­vel­op a new, con­vert­ible CAR-T

The bil­lion­aire-backed Park­er In­sti­tute for Can­cer Im­munother­a­py is in­vest­ing and part­ner­ing with a biotech their re­searchers be­lieve has the po­ten­tial to de­vel­op a new-mod­el CAR-T that can be bet­ter guid­ed down nar­row path­ways and equipped with a con­ve­nient on/off switch to help avoid any fa­tal ac­ci­dents.

Jef­frey Blue­stone

Park­er CEO Jef­frey Blue­stone isn’t giv­ing away any num­bers right now, but he says the in­sti­tute’s eq­ui­ty in­vest­ment in Xyphos Bio­sciences “isn’t triv­ial.” The pri­ma­ry pur­pose of the in­vest­ment — and the re­search al­liance that plugs Xyphos in­to their net­work of promi­nent in­ves­ti­ga­tors — is to ac­cel­er­ate the work and pre­vent it from get­ting stuck in the R&D mud.

And I’m just get­ting start­ed with the car metaphors, so buck­le up.

What at­tract­ed Park­er to the Bay Area im­munother­a­py play­er?

Gen­er­al­ly speak­ing, it’s their work on con­vert­ible CAR-Ts, re­volv­ing around NKG2D re­cep­tors on sev­er­al cell types, in­clud­ing T cells and NK killer cells that have fas­ci­nat­ed the field. 

Us­ing their pro­tein en­gi­neer­ing tech, the com­pa­ny craft­ed the nat­ur­al NKG2D re­cep­tor to be in­ac­tive un­til it’s turned on by their bis­pe­cif­ic an­ti­body. One end of the bis­pe­cif­ic binds to the re­cep­tors on the con­vert­ible CAR-T, with the oth­er end de­signed to bind to the tar­get­ed ma­lig­nant cell. Their col­li­sion is in­tend­ed to crush the can­cer cell.

There are sev­er­al ad­van­tages to the ap­proach, Blue­stone tells me. The safe­ty as­pect is en­hanced by the “reg­u­lat­ed sys­tem” Xyphos is us­ing. “You’re con­trol­ling to T cells: You can stop en­gag­ing the tar­get” — and that off switch can be hit in the event of a sud­den cy­tokine storm that can en­gulf a pa­tient, a rou­tine threat for the pro­to­type CAR-Ts on the mar­ket.

These bis­pecifics can al­so tar­get a cell se­quen­tial­ly, shift­ing tar­gets when a pa­tient starts to de­vel­op re­sis­tance, com­mon­ly lead­ing to a re­lapse. And while the work is still pre­clin­i­cal, Park­er’s in­vest­ment is in­tend­ed to push the team to­ward the clin­ic.

If it pans out, says Blue­stone, they’ll have a best-in-class ther­a­py able to go af­ter a va­ri­ety of sol­id tu­mors.

Park­er has done a num­ber of re­search al­liances with bio­phar­mas like Tes­sa, but steered on­ly a few eq­ui­ty in­vest­ments like this. They’ve backed the on­col­o­gy imag­ing out­fit Imag­inAb and Tmu­ni­ty, the start­up from Penn’s Carl June and Oz Azam, and now Xyphos. There are al­so a few in­vest­ments they’ve made which they’re still play­ing close to the vest.

They’re in for the long haul, with some near-term des­ti­na­tions in mind.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

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UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

Stephen Hahn, AP

The FDA un­veils a new reg­u­la­to­ry frame­work to speed along gene ther­a­pies, re­ward­ing the lead­ing play­ers

Bioregnum Opinion Column by John Carroll

The emphasis at the FDA over the past 5 years or so has been on assisting drug developers as much as they can to speed up regulatory reviews and push more drugs into the market. And they are now crafting a final set of regulations aimed at flagging through a whole new generation of gene therapies in clinical testing at a rapid clip.

In a set of 6 prospective guidances posted on the FDA web site Tuesday morning, FDA commissioner Stephen Hahn committed the agency to staying flexible in handing out designations that are critical to gaining early approvals for drugs that claim to be once-and-done but don’t have anything close to the data needed to prove it.

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Mike Bloomberg (AP IMAGES)

Mike Bloomberg joins a grow­ing cho­rus of De­mo­c­ra­t­ic pres­i­den­tial can­di­dates threat­en­ing to go af­ter drug patents

As the mayor of New York City, Mike Bloomberg had a few modest ideas about lowering prescription drug prices in the Big Apple that gained little traction. Now on the campaign trail on a faint hope of clinching the Democratic presidential nomination, the billionaire has some bigger plans — including one that would alter the patent system central to the biopharma business.

In a barebones drug pricing plan posted on Monday, Bloomberg came out blasting President Donald Trump for failing to deliver his promise to lower drug prices, and then making misleading claims about them. The price of over 3,000 drugs still increased at a rate five times higher than inflation in the first six months of 2019, he wrote.