J&J does Alzheimer's gene ther­a­py deal, along with 14 oth­er new col­lab­o­ra­tions

John­son & John­son has inked a re­search deal with Uni­ver­si­ty of Penn­syl­va­nia to work on gene ther­a­py for Alzheimer’s. The part­ner­ship lever­ages the uni­ver­si­ty’s ade­no-as­so­ci­at­ed virus­es and J&J’s an­ti-Alzheimer’s an­ti­bod­ies in hopes to pen­e­trate the blood brain bar­ri­er.

The part­ner­ship in­volves the phar­ma gi­ant’s in­no­va­tion-fo­cused arm John­son & John­son In­no­va­tion and Janssen Phar­ma­ceu­ti­cals. J&J hopes to use the uni­ver­si­ty’s AAV vi­ral de­liv­ery to trig­ger the ex­pres­sion of ther­a­peu­tic an­ti­bod­ies to the brain – an ef­fort that would change the sta­tus quo. Cur­rent­ly, the blood brain bar­ri­er lim­its the use of an­ti­bod­ies to treat brain dis­eases.

Janssen will have ex­clu­sive glob­al rights to com­mer­cial­ize prod­ucts un­der this new agree­ment.

Paul Stof­fels, J&J

The new re­search deal was one of 15 new col­lab­o­ra­tions J&J an­nounced as part of their reg­u­lar op­er­a­tional up­dates. John­son & John­son In­no­va­tion is no stranger to deals like this, as it’s penned more than 350 such part­ner­ships since its in­cep­tion in 2012.

“By ad­vanc­ing trans­for­ma­tive health­care in­no­va­tions to­geth­er with en­tre­pre­neurs, aca­d­e­m­ic cen­ters and in­sti­tu­tions, we are one step clos­er to ad­dress­ing many press­ing glob­al health­care chal­lenges,” said Paul Stof­fels, ex­ec­u­tive vice pres­i­dent and CSO of John­son & John­son, in a state­ment.

Here’s a sum­ma­ry of oth­er no­table ther­a­peu­tic re­search deals an­nounced in­volv­ing var­i­ous J&J arms:

→ A mul­ti-year re­search col­lab­o­ra­tion with Bea­con Dis­cov­ery, a G-Pro­tein Cou­pled Re­cep­tor (GCPR) drug dis­cov­ery in­cu­ba­tor launched by Belviq-mak­er Are­na Phar­ma­ceu­ti­cals in 2016. The col­lab­o­ra­tion will be to dis­cov­er and de­vel­op next-gen­er­a­tion ther­a­peu­tics to treat obe­si­ty and oth­er meta­bol­ic dis­eases.

→ A col­lab­o­ra­tion with a Boston mi­cro­bio­me ther­a­peu­tics com­pa­ny Holo­bio­me Cor­po­ra­tion to treat dis­eases of the cen­tral and en­teric ner­vous sys­tems. The col­lab­o­ra­tion will ex­am­ine a con­sor­tium of bac­te­ria that could be used to cre­ate a dif­fer­en­ti­at­ed pro­bi­ot­ic or over-the-counter of­fer­ing that ad­dress­es sleep­less­ness.

→ A col­lab­o­ra­tion with San Diego’s Der­mala to de­vel­op mi­cro­bio­me-de­rived treat­ments for skin con­di­tions. Der­mala’s tech­nol­o­gy har­ness­es the ben­e­fi­cial func­tion of good skin bac­te­ria to elim­i­nate the bad bac­te­ria and bal­ance the mi­cro­bio­me.

→ A col­lab­o­ra­tion with Monash Uni­ver­si­ty aimed at fur­ther ex­plor­ing the un­der­ly­ing trig­gers of pso­ri­a­sis to dis­cov­er and de­vel­op po­ten­tial new treat­ments that pre­vent fu­ture oc­cur­rences of the dis­ease.

→ A col­lab­o­ra­tion with the Tai­wan In­dus­tri­al Tech­nol­o­gy Re­search In­sti­tute to set up co-fund­ing agree­ments for projects in lung can­cer, chron­ic ob­struc­tive pul­monary dis­ease, di­a­betes, eye health and dig­i­tal health ini­tia­tives.

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive PhI­II for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma. 

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.