Cel­gene's AML drug clears late-stage test

Cel­gene is gear­ing up for reg­u­la­to­ry sub­mis­sions af­ter a piv­otal acute myeloid leukemia (AML) study showed its drug helped pa­tients live longer.

The drug­mak­er — which is in the process of be­ing swal­lowed by Bris­tol-My­ers Squibb $BMY — test­ed its ex­per­i­men­tal drug, CC-486, in new­ly-di­ag­nosed pa­tients with AML who ful­ly or par­tial­ly ben­e­fit­ed from chemother­a­py.

Jay Back­strom

In the 472-pa­tient QUAZAR AML-001 tri­al, pa­tients on sup­port­ive care were ei­ther giv­en CC-486 or a place­bo once dai­ly for two weeks. Main­te­nance treat­ment with CC-486 con­ferred a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in over­all sur­vival — meet­ing the main goal — as well as the end­point of re­lapse-free sur­vival, Cel­gene $CELG said on Fri­day.

“AML re­mains a dead­ly blood can­cer where most pa­tients are not cur­able and less than 30% of pa­tients sur­vive five years,” Cel­gene’s chief med­ical of­fi­cer Jay Back­strom said in a state­ment. “The CC-486 QUAZAR AML-001 study is the first phase 3 tri­al to demon­strate that the ad­di­tion of main­te­nance ther­a­py has the po­ten­tial to ex­tend over­all sur­vival in a broad pop­u­la­tion of pa­tients with new­ly di­ag­nosed AML who have achieved re­mis­sion with in­duc­tion chemother­a­py.”

De­tailed da­ta on CC-486 — which is al­so be­ing test­ed in oth­er forms of can­cer — are ex­pect­ed at a fu­ture med­ical con­fer­ence, and the com­pa­ny is plan­ning reg­u­la­to­ry sub­mis­sions next year.

AML is a form of can­cer in which the bone mar­row pro­duces ab­nor­mal myeloblasts (a type of white blood cell), red blood cells, or platelets. About 21,450 cas­es of AML will be di­ag­nosed in 2019, and 10,920 will suc­cumb to the dis­ease, es­ti­mates the Amer­i­can Can­cer So­ci­ety.

Last No­vem­ber, Japan’s Astel­las se­cured FDA ap­proval for its AML drug, gilter­i­tinib, for use in pa­tients that car­ry a mu­ta­tion that ac­counts for about a third of all AML cas­es. In Ju­ly 2018, Agios al­so scored FDA ap­proval for its tar­get­ed AML drug, Tib­so­vo, for pa­tients that car­ry a par­tic­u­lar mu­ta­tion.

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.”

Endpoints Premium

Premium subscription required

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

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

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.

James Sabry, Roche global head of pharma partnering

Roche, Genen­tech plunk down $60M up­front to part­ner with Chi­nese phar­ma on PRO­TAC-based prostate can­cer drug

Roche and Genentech are always on the hunt for deals, and on Thursday they found their newest partner.

The pair will team up with the Chinese pharma company Jemincare to push forward a new program for prostate cancer, the companies announced. Roche is ponying up $60 million upfront to get its hands on the candidate and promising up to $590 million in biobucks, plus royalties, down the line.

In return, Genentech will get a worldwide license to develop the program, known as JMKX002992, and bring it to market.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

Andrew Hopkins, Exscientia CEO

Ex­sci­en­tia ter­mi­nates Bay­er pact half a year ear­ly, col­lect­ing small por­tion of €240M promised

Bayer and Exscientia are winding down their three-year collaboration, leaving the big German pharma to take the AI-designed compounds born out of the pact further.

London-based Exscientia revealed in its Q2 update that the partners have “mutually agreed to end” their collaboration, which kicked off in early 2020, after recently achieving a drug discovery milestone. In an SEC filing, Exscientia said it terminated the pact on May 30, about six months early.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 148,400+ biopharma pros reading Endpoints daily — and it's free.