New Products

Updated: Celgene, Agios win a landmark FDA OK for new AML drug Idhifa

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.

 


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RAPS Regulatory Convergence 2017