David Schenkein, Agios CEO

Cel­gene and Agios didn’t have to wait un­til the PDU­FA date at the end of Au­gust to get a speedy FDA de­ci­sion on enasi­denib (AG-221), their new ther­a­py for acute myeloid leukemia. The FDA cut to the chase and ap­proved it to­day.

Cel­gene $CELG re­worked its deal with Agios $AGIO last year, but kept its hands on its rights to AG-221 in pa­tients with ad­vanced hema­to­log­ic ma­lig­nan­cies with an IDH2 mu­ta­tion. In turn, Agios stayed ra­zor fo­cused on grab­bing the ear­li­est pos­si­ble ap­proval, hus­tling Phase I/II da­ta straight to reg­u­la­tors for an ac­cel­er­at­ed re­view.

The drug will now be sold as Id­hi­fa, con­cen­trat­ing on about 9% to 13% of the AML mar­ket.

“The use of Id­hi­fa was as­so­ci­at­ed with a com­plete re­mis­sion in some pa­tients and a re­duc­tion in the need for both red cell and platelet trans­fu­sions,” not­ed the FDA’s Richard Paz­dur in herald­ing the OK to­day. The drug will be used with the Re­al­Time IDH2 As­say, which is used to de­tect spe­cif­ic mu­ta­tions in the IDH2 gene in pa­tients with AML.

It won’t come cheap. From Cel­gene:

The ap­proval of ID­HI­FA rep­re­sents a new, tar­get­ed ther­a­py for a sub­type of AML with an ex­treme­ly rare ge­net­ic mu­ta­tion. The 8-19% of AML pa­tients that have an IDH2 mu­ta­tion rep­re­sent about 1,200 to 1,500 in­di­vid­u­als in the U.S. These pa­tients are liv­ing with dis­ease that has a poor prog­no­sis and no stan­dard of care and no oth­er cur­rent­ly ap­proved op­tions un­til to­day. Pair­ing this ther­a­py with an ap­proved com­pan­ion di­ag­nos­tic al­so sig­nif­i­cant­ly im­proves iden­ti­fi­ca­tion of el­i­gi­ble pa­tients who could ben­e­fit from this new­ly ap­proved treat­ment.

The month­ly whole­sale ac­qui­si­tion cost of ID­HI­FA is $24,872. In the piv­otal study, the me­di­an time on ther­a­py for pa­tients was 4.3 months

Cel­gene first al­lied it­self with Agios back in 2010, part­ner­ing on its work in can­cer me­tab­o­lism. That deal led Cel­gene to grab world­wide rights to AG-221 in 2014. Cel­gene hand­ed back rights to AG-120, an IDH1 in­hibitor, but al­so an­ted up an­oth­er $200 mil­lion to grab op­tions on a slate of ex­per­i­men­tal drugs in the biotech’s pipeline.

The re­la­tion­ship paid off nice­ly for Cel­gene. The jour­ney from first-in-hu­man test­ing to a reg­u­la­to­ry fil­ing took just three years, an in­cred­i­bly fast speed in R&D. And Agios plans to do that again with oth­er drugs in its pipeline. Leerink’s says it’s a big step for both com­pa­nies, which will be fol­lowed close­ly dur­ing the mar­ket launch.

This is the first drug to emerge from Cel­gene’s col­lab­o­ra­tion with Agios, and from Agios’ re­search plat­form in can­cer me­tab­o­lism, and al­so the first drug to be ap­proved from Cel­gene’s net­worked re­search arrange­ments. It pro­vides im­por­tant val­i­da­tion for both plat­form and strat­e­gy, and in­vestors will close­ly mon­i­tor the drug’s com­mer­cial per­for­mance to as­sess how well these ad­vances trans­late in­to com­mer­cial suc­cess. Cel­gene is the pri­ma­ry com­mer­cial­iz­ing par­ty in the US, al­though Agios has opt­ed in to co-com­mer­cial­ize the prod­uct in the US. Cel­gene has sug­gest­ed that they will add ap­prox­i­mate­ly 30 head­count to sup­port the prod­uct’s com­mer­cial­iza­tion on the US. Cel­gene es­ti­mates that there is a tar­get­ed pa­tient pop­u­la­tion of 1,200-1,500 pa­tients in the US and ex­pects the du­ra­tion of treat­ment to ap­prox­i­mate the 4 months ob­served in the phase I/II tri­al.

Leerink’s Michael Schmidt al­so took a step back and sized up the im­pli­ca­tions for the biotech, now that it owns the IDH1 ther­a­py. It all adds up to a siz­able op­por­tu­ni­ty, he notes:

In our view, FDA-ap­proval of Id­hi­fa al­so re­duces reg­u­la­to­ry risk for ivosi­denib in AML, the lat­ter be­ing pro­pri­etary to AGIO and hence rep­re­sent­ing a more sig­nif­i­cant val­ue dri­ver for the com­pa­ny. AGIO’s IDH1 & 2 in­hibitors ad­dress a ~$270M and ~$370M glob­al mar­ket op­por­tu­ni­ty in re­lapsed/re­frac­to­ry AML, re­spec­tive­ly, based on our up­dat­ed es­ti­mates; po­ten­tial front-line use in com­bi­na­tion with ap­proved or in­ves­ti­ga­tion­al agents re­flects a sig­nif­i­cant­ly larg­er $1Bn+ com­mer­cial op­por­tu­ni­ty based on our re­cent due dili­gence should on­go­ing and planned front-line clin­i­cal tri­als suc­ceed.

CEO David Schenkein is fond of re­call­ing Agios start­ed out with lit­tle more than a blank sheet of pa­per. Since then, the pages have been filled with high-pro­file part­ner­ships, an IPO for its po­ten­tial cut­ting-edge work and now its first new drug ap­proval. It’s a wa­ter­shed mo­ment for this biotech.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

A California judge will allow a plaintiff in a state court case to introduce expert testimony connecting a potential carcinogen in former blockbuster medicine Zantac to cancer.

The order was handed down on Thursday from state judge Evelio Grillo, who is now allowing both parties to introduce expert testimony in an upcoming trial after what’s known as a Sargon hearing, where a judge determines the admissibility of expert witnesses and expert opinions.