Juno gets some bad­ly need­ed re­lief af­ter a glimpse of up­beat ear­ly da­ta on JCAR017

Anx­ious to turn the page on a dead­ly se­ries of cere­bral ede­ma cas­es, Juno Ther­a­peu­tics to­day turned to an up­beat set of pre­lim­i­nary da­ta for JCAR017, which may soon emerge as its top drug in the pipeline.

Twelve of 20 pa­tients who could be eval­u­at­ed for ef­fi­ca­cy in the study of CD19-pos­i­tive cas­es of non-Hodgkin lym­phoma demon­strat­ed a com­plete re­sponse to the CAR-T ther­a­py fol­low­ing treat­ment at dose lev­el 1 (5×107 cells). There was some ev­i­dence of dura­bil­i­ty, with 42% of the pa­tients (8/19) with dif­fuse large B-cell lym­phoma treat­ed more than three months be­fore the da­ta cut­off ex­pe­ri­enc­ing an on­go­ing re­sponse. And there were no cas­es of se­vere cy­tokine re­lease syn­drome with grade 3-4 neu­ro­tox­i­c­i­ty seen in three in­di­vid­u­als among 22 in the dose lev­el 1 group who were evalu­able for safe­ty.

Juno has re­peat­ed­ly been ham­mered by set­backs on JCAR015, a lead CAR-T that has been linked to 5 pa­tient deaths. The biotech was forced to re­sume a clin­i­cal hold re­cent­ly af­ter two new deaths from brain swelling stunned in­vestors.

Juno had re­as­sured the FDA that it could elim­i­nate cas­es of cere­bral ede­mas by elim­i­nat­ing flu­dara­bine, a con­di­tion­ing agent used along with cy­clophos­phamide in a com­bo de­signed to help pa­tients re­spond fa­vor­ably to the cell ther­a­py, de­signed to at­tack can­cer cells.

The tur­moil at Juno has thrown the biotech well off a track that once promised to seek an ap­proval in 2017. JCAR015’s ap­proval was pushed back to 2018, and now a num­ber of ob­servers are wait­ing to see if Juno sim­ply drops JCAR015 and turns its at­ten­tion to JCAR017 as its best shot at a best-in-class con­tender.

No­var­tis and Kite, mean­while, are both plan­ning on Q1 ap­pli­ca­tions for their lead­ing ef­forts, putting them well ahead of Juno on the pac­ing.

Martin Landray, Protas CEO (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Those big bil­lion-dol­lar PhI­II stud­ies? Mar­tin Lan­dray says they can be done for a tiny frac­tion of the cost

Martin Landray knows what controversy in clinical drug development feels like, from first-hand experience.

Landray was the chief architect of RECOVERY, a study that pitted a variety of drugs against Covid-19. And he offered some landmark data that would help push dexamethasone out into broader use as a cheap treatment, while helping ice hydroxy’s reputation as a clear misfire.

“Lots of people told us we shouldn’t use it,” Landray says about dexamethasone and Covid-19. “It was dangerous. We shouldn’t even do a trial. They also cared about hydroxychloroquine and lots of people said we shouldn’t do a trial because it must be used. I’ve got the letters from both sets of people.”

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IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

James Dentzer, Curis CEO

FDA lifts par­tial hold on Curis' lym­phoma study — shares spike

Four months after the FDA put two clinical trials from Curis on clinical hold, the FDA is now apparently content with how the biotech will change up managing one of the studies.

The Massachusetts oncology biotech put out word early Thursday that the federal regulator lifted a partial clinical hold of the company’s Phase I/II study of emavusertib in lymphoma, following a new data package that the biotech recently submitted to the agency. Shares of the biotech $CRIS, hovering just above penny stock territory, shot up more than 55% in early trading before settling at close to a 30% share price boost.

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Geoffrey Porges, new Schrödinger CFO

Long­time an­a­lyst Ge­of­frey Porges de­parts SVB to lead fi­nances at a drug dis­cov­ery shop

Geoffrey Porges has ended his two-decade run as a biotech analyst, as the former SVB Securities vice chair began as CFO of Schrödinger on Thursday.

The long-running analyst, who previously headed up vaccines marketing at Merck before the turn of the millennium, will lead the financial operations of the 700-employee company as Schrödinger broadens its focus from a drug discovery partner to also building out an in-house pipeline, with clinical trial No. 1 set to begin next quarter.

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Astel­las' hot flash­es drug will get speedy re­view at FDA; US opts out of Val­ne­va vac­cine

The FDA will decide on Astellas’ menopausal symptom drug by Feb. 22 of next year, as the Japanese pharma disclosed it had paid about $97 million to get a priority review voucher to speed up the review.

Astellas said the agency has accepted the pharma’s application for fezolinetant for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. VMS includes hot flashes and/or night sweats. The company said as many as 80% of women in the US experience those symptoms during or after the menopausal transition.

Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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