Juno’s lead CAR-T slammed with a clin­i­cal hold in the last lap ahead of FDA fil­ing

One of Juno’s crit­i­cal stud­ies for its lead CAR-T ther­a­py has just been in­def­i­nite­ly side­lined fol­low­ing the un­ex­plained death of two pa­tients from neu­ro­tox­i­c­i­ty, de­rail­ing the com­pa­ny’s plans to seek mar­ket­ing ap­proval in time for a 2017 launch.

In a nasty sur­prise for share­hold­ers, Juno an­nounced Thurs­day af­ter­noon that the FDA had placed a clin­i­cal hold on its ROCK­ET study, a Phase II clin­i­cal tri­al of JCAR015 in adult pa­tients with re­lapsed or re­frac­to­ry B cell acute lym­phoblas­tic leukemia. That hob­bles the com­pa­ny just as it en­tered the last lap in a tight race with Kite Phar­ma.

The stock $JUNO im­me­di­ate­ly plunged 30%.

In a call with an­a­lysts, Juno CEO Hans Bish­op said that the com­pa­ny no longer ex­pects to get the drug on the mar­ket in 2017. That dead­line has now been pushed to 2018. He al­so said that an­oth­er pa­tient had died of neu­ro­tox­i­c­i­ty in May, but the study con­tin­ued af­ter dis­cus­sions with the FDA. All the pa­tients who died were young.

The biotech in­stant­ly point­ed to the re­cent ad­di­tion of flu­dara­bine as part of the pre-con­di­tion­ing reg­i­men pa­tients un­der­go as a like­ly cause of the deaths. And they have al­ready pro­posed drop­ping that.

“Flu­dara­bine in some ways acts like a dose am­pli­fi­er,” says Bish­op, ex­plain­ing the com­pa­ny’s the­o­ry of what went wrong and trig­gered these cas­es of cere­bral ede­ma.

The FDA has asked for: “A re­vised pa­tient in­formed con­sent form, a re­vised in­ves­ti­ga­tor brochure, a re­vised tri­al pro­to­col, and a copy of the pre­sen­ta­tion made to the agency yes­ter­day.”

All oth­er stud­ies are con­tin­u­ing. And a sub­mis­sion is al­ready ex­pect­ed to be sub­mit­ted this week, un­der­scor­ing the com­pa­ny’s be­lief that it can get the hold lift­ed quick­ly.

There are ab­solute­ly no guar­an­tees, how­ev­er, that that can be done. Zaf­gen has been wait­ing for months for reg­u­la­tors to ad­dress their clin­i­cal hold. And reg­u­la­tors are known for tak­ing their time in re­solv­ing these is­sues.

That’s a huge is­sue for Juno, which was telling re­porters just weeks ago that it was con­fi­dent of fil­ing for an ac­cel­er­at­ed ap­proval in time to get their drug on the mar­ket next year —  rac­ing with Kite to be the first on the mar­ket. It may al­so af­fect oth­er pro­grams as well, if Juno is right. Re­cent re­search has in­di­cat­ed that adding flu­dara­bine im­proves CAR-T cell ex­pan­sion and per­sis­tence, pro­vid­ing what was thought to be an added edge in treat­ing can­cer.

Arch man­ag­ing part­ner and Juno di­rec­tor Robert Nelsen took to Twit­ter to de­fend the com­pa­ny.

 

Juno cap­tured pub­lic at­ten­tion with its pi­o­neer­ing work on CAR-Ts, tak­ing pa­tient cells and adapt­ing them with a chimeric anti­gen re­cep­tor to make them in­to can­cer cell at­tack ve­hi­cles. Ear­ly on in­ves­ti­ga­tors had to ad­just their stud­ies to counter the chance that pa­tients would be hit with a cy­tokine storm as the drug was ad­min­is­tered. This is the first time that neu­ro­tox­i­c­i­ty has halt­ed a CAR-T study.

And where does this leave Kite? Clear­ly in the lead.

The biotech put out a re­lease late Thurs­day as­sert­ing that it com­plet­ed en­rolling pa­tients for its on­go­ing Phase II piv­otal study of KTE-C19, leav­ing it on track for a sub­mis­sion to the FDA by the end of the year.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Sean Nolan and RA Session II

Less than 3 months af­ter launch, the AveX­is crew’s Taysha rais­es $95M Se­ries B. Is an IPO next?

The old AveXis team is moving quickly in Dallas.

Three months ago, they launched Taysha with $30 million in Series A funding and a pipeline of gene therapies out of UT Southwestern. Now, they’ve announced an oversubscribed $95 million Series B. And the biotech is declining all interview requests on the news, the kind of broad silence that can indicate an IPO is in the pipeline.

Biotechs, including those relatively fresh off launch, have been going public at a frenzy since the pandemic began. Investors have showed a willingness to put upwards of $200 million to companies that have yet to bring a drug into the clinic. Still, if Taysha were to go public in the near future, it would be perhaps the shortest path from launch to IPO in recent biotech memory.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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RA, No­var­tis back Gen­tiBio's seed round, plans to launch de­vel­op­ment of En­gTreg ther­a­pies

Boston, MA-based startup GentiBio landed a $20 million seed fund from three investors to dive into engineered regulatory T cell (EngTreg) development.

Marquee investors OrbiMed, Novartis Venture Fund and RA Capital Management have backed GentiBio’s mission to develop EngTregs for the treatment of autoimmune, alloimmune, autoinflammatory, and allergic diseases. Unlike other companies studying treatments using a patient’s own Tregs, GentiBio plans to make use of CD4+ immune cells, found in the blood.

Paul Laikind, ViaCyte CEO

Stem cell play­er Vi­a­Cyte ex­pands col­lab­o­ra­tion with Gore to de­vel­op sub­cu­ta­neous di­a­betes treat­ment

Longtime stem cell player ViaCyte has teamed up with a materials science company in an effort to solve immunosuppression challenges and advance its type 1 diabetes treatments.

Expanding on an existing collaboration, ViaCyte and W.L. Gore have agreed to combine the biotech’s PEC-Encap candidate with a Gore-produced membrane in what they hope will eliminate the need for immunosuppressive drugs. Such treatments have created foreign body responses in the past, and stamping these reactions out is the main goal, ViaCyte CEO Paul Laikind said.

My­ovant lands a fresh $200M loan as FDA mar­ket­ing de­ci­sion looms; Amarin goes it alone in Eu­rope

Myovant is getting ready to roll out its commercial operations to back relugolix, now under FDA review for prostate cancer.

The startup has added a fresh $200 million in support from Sumitomo Dainippon Pharma, which controls a majority of the stock $MYOV. Sumitomo is handing the cash over as a loan, bringing its total to $600 million. Myovant — which is gearing up for a showdown with AbbVie — has also filed an NDA to sell relugolix for uterine fibroids and recently posted positive late-stage data for endometriosis.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Mod­er­na CEO Stéphane Ban­cel out­lines a prospec­tive moth­er­lode of Covid-19 vac­cine rev­enue — will a back­lash fol­low?

Moderna shows no sign of slowing down, or turning charitable when it comes to pricing supplies of its Covid-19 vaccine.

One of the leaders in the Phase III race to get a Covid-19 vaccine across the finish line in record time, Moderna says it’s on track to complete enrollment in one of the most avidly watched studies in the world next month. And the biotech has already banked some $400 million in deposits for vaccine supply as it works through negotiations with countries around the world — as CEO Stéphane Bancel sets out to hire a commercial team.

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Covid-19 roundup: J&J and BAR­DA agree to $1 bil­lion for 100 mil­lion dos­es; Plas­ma re­duces mor­tal­i­ty by 50% — re­ports

J&J has become the latest vaccine developer to agree to supply BARDA with doses of their Covid-19 vaccine, signing an agreement that will give the government 100 million doses in exchange for $1 billion in funding.

The agreement, similar to those signed by Novavax, Sanofi and AstraZeneca-Oxford, provides funding not only for individual doses but to help J&J ramp up manufacturing. Pfizer, by contrast, received $1.95 billion for the doses alone. Still, if one looked at each agreement as purchase amounts, J&J’s deal would be $10 per dose, slotting in between Novavax’s $16 per dose and AstraZeneca’s $4 per dose.

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