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.

Precision CMO Alan List

ASH: An­oth­er 'off-the-shelf' cell ther­a­py leader shows dura­bil­i­ty is­sues, rais­ing re­newed con­cerns about emerg­ing field

The next generation of cell therapies have focused in large part on the development of allogeneic — better known as “off-the-shelf — drugs that can cut manufacturing times and hopefully evade a patient’s immune system. One of the early players in that race has new data at #ASH21 that show deep responses but will also raise fresh concerns about these therapies’ durability.

Precision Biosciences’ PBCAR0191, a CD19-directed allogeneic CAR-T cell therapy, posted a complete response rate of 59% in 22 heavily pretreated patients with various forms of relapsed or refractory non-Hodgkin’s lymphoma and acute lymphocytic leukemia, six of whom had previously received an autologous CAR-T before dosing, the biotech said.

End­points hon­ors 20 women su­per­charg­ing drug R&D; Omi­cron is a 'stress test' for the next pan­dem­ic; Roche and Genen­tech strike an AI pact with Re­cur­sion; 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.

The bulk of this week’s report is brought to you by Endpoints editors Nicole DeFeudis and Max Gelman, who are covering for me as I take a few days off after the big Women in Biopharma R&D event. We are really proud of both the special report and the live panel, which featured some great stories from trailblazing leaders and insights on gender diversity in biotech. Do check them out below if you haven’t had a chance.

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Op­ti­miz­ing Oral Drug De­liv­ery us­ing Zy­dis® Oral­ly Dis­in­te­grat­ing Tablet Tech­nol­o­gy to Ad­dress Pa­tient Chal­lenges

KEY POINTS

Patients prefer oral dosing, but swallowing tablets can be a challenge for many patients.
The Zydis® orally disintegrating tablet (ODT) platform addresses challenges associated with oral dosing, expanding benefits for patients and options for healthcare providers.
A strong growth trajectory is expected for ODTs given therapeutic innovation and continued technology development.

Many patients prefer conventional tablets for the administration of medications, but some geriatric and pediatric patients and those with altered mental status and physical impairments find swallowing tablets to be difficult. Orally disintegrating tablets (ODTs), which dissolve completely without chewing or sucking, offer a patient-friendly dosage form for the administration of small-molecule drugs, peptides and proteins. With the potential for multiple sites of drug absorption, often faster onset action for the active pharmaceutical ingredient (API), and potentially greater bioavailability, ODTs are an attractive option for drug developers considering first-to-market formulations or product line extensions of existing drugs with compatible API. In this report, we look at how innovation in the industry-leading Zydis ODT platform is expanding oral formulation options and bringing benefits to patients.

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Helen Heslop, Center for Cell and Gene Therapy director

ASH: Tes­sa Ther­a­peu­tic­s' next-gen cell ther­a­py clears four pa­tients' tu­mors in ear­ly study

Tessa Therapeutics is doing CAR-T a bit differently. After reading out some positive — albeit early — results back in May showing their virus-specific T cells (VSTs) achieved three partial responses in patients with CD30-positive lymphomas, the company now says the fuller picture looks even brighter.

TT11x, Tessa’s “off the shelf” CD30.CAR-modified Epstein-Barr virus-specific T-cell (EBVST) therapy, achieved a 77.8% overall response rate (7 of 9 patients) in a Phase I trial, the Singapore-based company announced at this year’s ASH conference. What’s more, four of those seven patients saw a complete response, Tessa said.

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Christi Shaw, Kite CEO

ASH: Gilead­'s Kite un­veils more da­ta on Yescar­ta's win in sec­ond-line lym­phoma pa­tients, set­ting up bat­tle for SOC

As a spate of researchers work diligently on next-gen CAR-T cell therapies, the big players in the current generation of those drugs are still angling for more market share. Getting to patients earlier is now the game plan, and Gilead’s Kite has uncorked some impressive data backing up its case in lymphoma.

Kite’s Yescarta (axicabtagene ciloleucel) cut the risk of disease progression, death or the need for additional therapy by a little more than 60% compared with standard of care in second-line patients with relapsed or refractory large B cell lymphoma, according to full data from the Phase III ZUMA-7 study revealed Saturday at #ASH21.

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ASH: With Yescar­ta lead­ing in ear­li­er-line lym­phoma, Bris­tol My­ers keeps the heat on with full da­ta for Breyanzi

Kicking off this weekend’s #ASH21, Gilead’s Kite and Bristol Myers Squibb have released competing data for their current-gen CAR-T drugs in second-line B cell lymphoma patients. It’s a heated contest to move these drugs into earlier lines of therapy, and Bristol Myers thinks these fuller data will keep the pressure on.

Bristol Myers’ Breyanzi (lisocabtagene maraleucel) cut the risk of disease progression, death and other events by 65% over standard of care in second-line relapsed or refractory LBCL patients, according to data from the Phase III TRANSFORM study presented Saturday.

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Stéphane Bancel, Moderna CEO (Endpoints JPM20/Jeff Rumans)

Mod­er­na's flu da­ta of­fer a clear les­son: mR­NA is­n't mag­ic

Last fall, as their Covid-19 vaccine crossed the finish line, Moderna unveiled plans to take its newly proven mRNA platform and use it to effectively change how the world blocks humanity’s most persistent viral foes.

In addition to their pre-existing vaccine programs, executives announced new ones for flu, where vaccines have chronically underperformed, and HIV, which has eluded every inoculation effort over nearly 40 years. In flu, the other mRNA vaccine companies — BioNTech (with Pfizer), Translate Bio (under Sanofi), and CureVac (with GSK) — all had similar ambitions, hoping to make shots that were as high as 80% effective.

House Speaker Nancy Pelosi (Jacquelyn Martin/AP Images)

Over­sight Com­mit­tee un­leash­es scathing drug pric­ing re­port as Sen­ate preps to vote on al­low­ing Medicare ne­go­ti­a­tions

Back in January 2019, the late House Oversight Committee chair Elijah Cummings kicked off a nearly 3-year-long drug pricing investigation that culminated today in a major new report detailing how prices for vital drugs have risen substantially since their launch, while calling on the Senate to pass a bill that will allow Medicare to negotiate some prices.

The committee’s investigation focused on 12 of the most expensive drugs for Medicare, showing massive price spikes that have accumulated over the years and made some drugs, like insulin, entirely unaffordable for some, to the point where some diabetics have had to ration their life-saving insulin, and some have died.

Andreas and Thomas Strüngmann (via Agreus Group)

Bil­lion­aire BioN­Tech back­ers have been kick­ing the tires at No­var­tis' big gener­ics arm — re­port

While the ultimate fate of Novartis’ big generics arm Sandoz may still be up in the air, there’s no doubt it’s in play as a potential buyout target.

Overnight, Reuters picked up on a report out of Germany that EQT and the billionaire Strüngmann brothers — enjoying a huge windfall from the overnight success of BioNTech’s mRNA Covid vaccine — are kicking the tires at Sandoz. And Novartis CEO Vas Narasimhan confirmed they’ve seen some M&A interest, even if no hard offers are on the table.

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