No­var­tis pitch­es two new CAR-T ap­pli­ca­tions to EMA; J&J re­ports pos­i­tive da­ta for lu­pus pro­gram

→ Basel-based No­var­tis $NVS has pushed along a dou­ble-bar­reled ap­pli­ca­tion for its lead­ing CAR-T drug Kym­ri­ah (CTL019). The phar­ma gi­ant has ap­plied to the EMA for ap­provals in B-cell acute lym­phoblas­tic leukemia as well as dif­fuse large B-cell lym­phoma, which will like­ly put it head-to-head with ri­val Gilead’s Yescar­ta. “Since the his­toric FDA ap­proval of Kym­ri­ah, for­mer­ly CTL019, we have launched, man­u­fac­tured and sup­plied this high­ly in­di­vid­u­al­ized im­muno­cel­lu­ar ther­a­py in a com­mer­cial set­ting and the sub­mis­sion to the EMA is a ma­jor step to­ward our goal of de­liv­er­ing it to more crit­i­cal­ly ill can­cer pa­tients around the world,” said Vas Narasimhan, who’s in the process of step­ping up to the CEO post.

→ Bris­bane, CA-based Aim­mune $AIMT CEO Stephen Dil­ly says he plans to step down by the end of the year so he can spend more time with his fam­i­ly. The biotech plans to mount a search for a new CEO. Dil­ly not­ed: “My de­ci­sion to re­tire is based sole­ly on my per­son­al de­sire to have more time for my fam­i­ly, es­pe­cial­ly my el­dest son, who has spe­cial needs. We are an­nounc­ing this now in or­der to fa­cil­i­tate an or­der­ly ex­ec­u­tive search and tran­si­tion pe­ri­od, and I re­main com­plete­ly com­mit­ted to con­tin­u­ing to lead Aim­mune through the ex­cit­ing events ahead. We are look­ing for­ward to the com­ple­tion of our piv­otal Phase 3 PAL­ISADE tri­al around year-end and shar­ing top-line da­ta in the first quar­ter of 2018.”

→  Trans­late Bio CEO Ron Re­naud has re­cruit­ed Agios vet Ann Bar­bi­er for the CMO po­si­tion. Bar­bi­er had been vice pres­i­dent of clin­i­cal de­vel­op­ment, rare ge­net­ic dis­eases at Agios, a trend­set­ter in the Cam­bridge, MA biotech hub. Ear­li­er, she had worked at Shire for 7 years. “With near-term clin­i­cal pro­grams in cys­tic fi­bro­sis and OTC de­fi­cien­cy, Trans­late Bio is ap­proach­ing a crit­i­cal and ex­cit­ing time,” said Bar­bi­er.

VBL Ther­a­peu­tics $VBLT bagged a $15 mil­lion up­front pay­ment from NanoCar­ri­er Co. in ex­change for ex­clu­sive rights to VBL-111 in Japan.

J&J $JNJ has had some set­backs on the R&D front re­cent­ly, but over the week­end it pro­duced some im­pres­sive re­sults for their block­buster Ste­lara — al­ready ap­proved for pso­ri­a­sis and Crohn’s — in lu­pus. The drug helped 60% of the group on the drug arm in a re­cent study, com­pared to on­ly 31% for the place­bo-plus stan­dard of care. Reuters notes that the on­ly drug ap­proved for lu­pus in the last 50 years has been GSK’s Benlysta.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.