#ASH17: The BC­MA-tar­get­ed CAR-T bb2121 from blue­bird and Cel­gene takes a star turn in the spot­light

A few days ago, Cel­gene $CELG launched the piv­otal Phase II/III study that will dic­tate the fu­ture of the BC­MA-tar­get­ing bb2121, the CAR-T in-li­censed from blue­bird bio $BLUE. And if they come close to the da­ta that blue­bird has on of­fer at ASH this week­end, this drug will go on to blaze a big trail in the mar­ket as the de­vel­op­ers move on to the next big fo­cus in the field of en­gi­neered T cells.

In­ves­ti­ga­tors in the Phase Ib study out­lined a 94% over­all re­sponse rate for bb2121, with a jaw-drop­ping 56% com­plete re­sponse rate among a small group of 18 pa­tients in heav­i­ly pre­treat­ed ac­tive dose co­horts. Three of those 10 CRs, though, were un­con­firmed at the time of the pre­sen­ta­tion in At­lanta.

The me­di­an pro­gres­sion-free re­sponse rate had yet to be reached. But the 6- and 9-month PFS marks were passed with 81% and 71% rates.

Nick Leschly

“This is what we all dream about,” says blue­bird CEO Nick Leschly, “this kind of da­ta for pa­tients that are in a re­al­ly, re­al­ly tough po­si­tion.”

Blue­bird’s stock jumped 24% in pre-mar­ket trad­ing Mon­day, with Cel­gene {$CELG) up close to 5%.

To be sure there was plen­ty of tox­i­c­i­ty on the dose rang­ing study, with an ad­vanced pa­tient with a pun­ish­ing tu­mor bur­den ex­pe­ri­enc­ing grade 4 neu­ro­tox­i­c­i­ty with cere­bral ede­ma and sub­arach­noid he­m­or­rhage af­ter get­ting the high dose: 450 x 106 CAR+ T cells. And there were a num­ber of cas­es of cy­tokine re­lease syn­drome, a hall­mark of these first gen­er­a­tion CAR-Ts go­ing through the clin­ic.

Cel­gene will now take 150 to 300 mil­lion cell dos­es in­to the land­mark study with about 80 pa­tients, look­ing to see large­ly if it can repli­cate any­thing sim­i­lar. If they are suc­cess­ful, the part­ners can look for­ward to a block­buster mar­ket for one of the most wide­ly an­tic­i­pat­ed late-stage stud­ies in on­col­o­gy.

David David­son

The da­ta un­der­score the po­ten­tial of BC­MA as a tar­get in the field, which is see­ing the first pi­o­neers hit the mar­ket by hit­ting CD19 with ma­jor ef­fect. The da­ta will cer­tain­ly bright­en the stage for blue­bird and Cel­gene, the clear lead­ers in the field, as a bevy of ri­vals fol­low up in hopes of catch­ing or leapfrog­ging the fron­trun­ners.

Juno, mak­ing a CAR-T come­back with JCAR017, just bagged a gam­ma sec­re­tase in­hibitor from Eli Lil­ly for its BM­CA ther­a­py, adding IP agree­ments with the Fred Hutch and On­co­Track­er on their com­bo ap­proach.

But blue­bird isn’t sit­ting still. It’s al­so snap­ping up new tech to keep its cut­ting edge, like the CAR-en­gi­neered gam­ma delta T cell pact it struck with Scot­land’s TC Bio­Pharm a few days ago.

“We’ve got all cylin­ders fir­ing,” says David David­son, the blue­bird chief med­ical of­fi­cer.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,400+ biopharma pros reading Endpoints daily — and it's free.

ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.