Sid Mukherjee (Photo by Brian Ach/Getty Images for The New Yorker)

J&J, Sid Mukher­jee's Vor team up to pair bis­pecifics with en­gi­neered stem cells for blood can­cer

J&J is one of many Big Phar­ma drug­mak­ers chas­ing the gold­en goose in on­col­o­gy bis­pecifics, with some ear­ly pos­i­tive re­sults in lung can­cer to show for it. Now, look­ing to break through in blood can­cer, J&J is pair­ing up with a pre­co­cious stem cell play­er to part­ner with its pipeline.

J&J’s Janssen unit has tapped Vor Bio­phar­ma, the brain­child of Co­lum­bia on­col­o­gist and best­selling au­thor Sid Mukher­jee, to pair the drug gi­ant’s bis­pe­cif­ic an­ti­bod­ies with Vor’s en­gi­neered stem cells in a mar­riage the com­pa­nies hope will change the game in treat­ing acute myeloid leukemia.

De­tails on the deal were scarce with no fi­nan­cial terms dis­closed and lit­tle in­for­ma­tion on fu­ture mile­stones, but the com­pa­nies did note they would keep all rights and own­er­ship of their re­spec­tive pro­grams and plat­forms.

It’s the lat­est in a string of deals for Vor, which is work­ing on “in­vis­i­ble” stem cell trans­plants the biotech thinks will al­low ad­vanced cell ther­a­pies like CAR-T to more ef­fec­tive­ly at­tack blood can­cer.

Last month, the biotech signed a mul­ti­year deal with Pitts­burgh-based Abound Bio to ac­cess its plat­form of sin­gle- and mul­ti-tar­get CAR-Ts to com­bine with Vor’s pipeline. The biotechs will ini­tial­ly tar­get acute myeloid leukemia and think that a mul­ti-tar­get­ed CAR-T paired with an HSC trans­plant could be a path to bet­ter treat­ing hard-to-hit can­cer.

Vor’s lead pro­gram is VOR33, an HSC the biotech is study­ing so­lo and as a pair­ing with CD33 tar­get­ing CAR-T can­di­date VCAR33, which it in-li­censed from the NIH, in IND en­abling stud­ies in AML.

J&J, for its part, is one of many Big Phar­ma play­ers look­ing for a leg up in the bis­pe­cif­ic space amid a gold rush for next-gen on­col­o­gy drugs. Its lead bis­pe­cif­ic, an EGFR/MET an­ti­body dubbed ami­van­tam­ab, demon­strat­ed some ear­ly ef­fi­ca­cy in lung can­cer, whet­ting in­vestors’ ap­petites over the drug­mak­er’s hopes there.

At this year’s AS­CO, J&J rolled out ear­ly da­ta show­ing ami­van­tam­ab and small-mol­e­cule TKI drug laz­er­tinib post­ed a me­di­an du­ra­tion of re­sponse of 9.6 months in pa­tients with non-small cell lung can­cer with ex­on 19 dele­tion or L858R mu­ta­tion that hadn’t pre­vi­ous­ly un­der­gone chemo but pre­vi­ous­ly failed on As­traZeneca’s Tagris­so.

It’s a mea­ger up­date for J&J’s com­bo af­ter the drugs post­ed a 100% com­plete re­sponse — and whet­ted in­vestors’ ap­petites — at last year’s ES­MO for EGFR-mu­tat­ed NSCLC pa­tients who were treat­ment-naïve. In the re­lapsed set­ting, a 45-pa­tient co­hort hit a 36% con­firmed re­sponse rate with 1 com­plete re­sponse and 15 par­tial.

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

Laurent Fischer, Adverum CEO

Ad­verum faces murky fu­ture af­ter re­view turns up deep­er safe­ty is­sues for gene ther­a­py

Three months after revealing that a patient lost significant vision in one eye after receiving its experimental gene therapy, Adverum announced it found the safety issues were more widespread: Five of 12 patients who received a high dose of the therapy saw “similar clinically-relevant events.”

Three required surgery on their treated eye. And all 12 are being recommended “aggressive immunomodulatory treatments” to prevent further injury.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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John Houston, Arvinas CEO

Pfiz­er bets $1B cash on the orig­i­nal pro­tein de­graders as tech­nol­o­gy nears prime time

As one of the largest multinational corporations in the world, Pfizer has its tendrils in everything. The Big Pharma has potentially hundreds of billions of dollars to play with, and when it decides to go big, it can go as big as it wants.

And did Pfizer ever on Thursday.

Returning to one of its partners in protein degradation, Pfizer is teaming up again with Arvinas to advance and vastly expand a program for breast cancer. As part of the deal, Pfizer is handing over $1 billion immediately — $650 million in upfront cash and $350 million in an equity investment — and promising up to another $1.4 billion in regulatory and commercial milestones.

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