Tib­so­vo clears an­oth­er hur­dle for Servi­er, but can it make Agios' old drug prof­itable?

When Eu­ro­pean reg­u­la­tors saw the da­ta Agios used to win US ap­proval for their AML drug Tib­so­vo, they sent the more than decade-old biotech back to the draw­ing board. A sin­gle, sin­gle-armed tri­al was not go­ing to cut it.

On Mon­day, though, the drug’s new own­ers an­nounced it had cleared a more rig­or­ous study. In a ran­dom­ized, Phase III tri­al of cer­tain new­ly di­ag­nosed pa­tients, those who re­ceived a com­bi­na­tion of Tib­so­vo and chemother­a­py lived longer than those who re­ceived a com­bi­na­tion of place­bo and chemother­a­py. Those pa­tients al­so had high­er re­sponse rates and com­plete re­mis­sion rates.

The re­sults ac­tu­al­ly sur­passed the goal­posts Agios ex­ec­u­tives had set when they de­signed the tri­al in 2017. Al­though over­all sur­vival was a sec­ondary end­point, the pri­ma­ry end­point was sim­ply event-free sur­vival: the time un­til a treat­ment fail­ure, re­lapse, or death. The tri­al hit that end­point too.

The re­sults are a win for the 8% of AML pa­tients with an IDH1 mu­ta­tion — the sub­set el­i­gi­ble for Tib­so­vo, a tar­get­ed ther­a­py — who con­tin­ue to face a grim prog­no­sis and few op­tions out­side of chemo. They of­fer the first ev­i­dence that Tib­so­vo can not on­ly work by it­self but al­so in com­bi­na­tion with chemother­a­py.

The da­ta are al­so a boon for Servi­er, the French phar­ma that spent near­ly $2 bil­lion last year to ac­quire Tib­so­vo and the rest of Agios’ can­cer pipeline. In part be­cause so few pa­tients have AML with IDH1 mu­ta­tion, the biotech had strug­gled to turn its sci­en­tif­ic suc­cess in­to a com­mer­cial suc­cess, col­lect­ing on­ly about $30 mil­lion per quar­ter from the drug be­fore the sale.

Servi­er was bet­ting both that the ex­per­i­men­tal mol­e­cules Agios was putting through tri­als held promise and that its own ex­ten­sive com­mer­cial ex­pe­ri­ence would al­low them to boost Tib­so­vo bet­ter than the small­er biotech could. The new re­sults should al­low the com­pa­ny to mar­ket the drug more wide­ly in the US and al­low the French phar­ma to file again for ap­proval in Eu­rope.

Servi­er did not break down de­tailed re­sults from the study but said it would do so at a fu­ture med­ical con­fer­ence. If the com­pa­ny us­es the da­ta to get ap­proval in Eu­rope, it would like­ly be in a slight­ly dif­fer­ent in­di­ca­tion than they had orig­i­nal­ly ap­plied for: new­ly di­ag­nosed AML pa­tients with an IDH1 mu­ta­tion, rather than IDH1-pos­i­tive AML pa­tients who have al­ready re­ceived treat­ments.

Tib­so­vo, though, may not be alone on the mar­ket for long. For­ma Ther­a­peu­tics re­leased da­ta last year from their own tar­get­ed IDH1-pos­i­tive AML treat­ment that looked re­mark­ably sim­i­lar to the da­ta Agios used to gain ap­proval. A For­ma ap­proval would give pa­tients a sec­ond op­tion but, for Servi­er, put an­oth­er ob­sta­cle to mak­ing Tib­so­vo prof­itable.

“Tib­so­vo monother­a­py has been in­stru­men­tal in trans­form­ing out­comes for adult pa­tients with new­ly di­ag­nosed or re­lapsed re­frac­to­ry AML har­bor­ing an IDH1 mu­ta­tion,” Servi­er VP of clin­i­cal de­vel­op­ment Su­san Pandya said in a state­ment. “These promis­ing re­sults from the AG­ILE study sup­port the added ben­e­fit of in­hibit­ing the mu­tant IDH1 en­zyme in com­bi­na­tion with stan­dard chemother­a­py in the new­ly di­ag­nosed in­ten­sive chemother­a­py in­el­i­gi­ble set­ting.”

So­cial: HJBC / Shut­ter­stock

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.

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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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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.

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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.

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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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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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