Celgene (CELG - Get Report) , which owns the rights to the Agios drug, known as AG-221, under an existing partnership, will be in charge of filing the new drug application for the treatment of patients with relapsed or refractory acute myeloid leukemia, or AML, caused by a mutated protein known as IDH2.
Agios disclosed the AG-221 approval submission plan in an 8-K filing to the Securities and Exchange Commission on Wednesday morning. Celgene plans to elaborate on its AG-221 plans later today during a presentation at a health care investor conference, the Agios 8-K said.
Until Wednesday, neither Celgene nor Agios had provided investors with a timeline for an AG-221 FDA submission. The faster-than-expected filing -- expected to be completed by the end of 2016 -- means AG-221 could be approved and on the market in the middle of 2017.
The new drug application will be based on data from an ongoing phase I/II study of AG-122 in patients diagnosed with various stages of IDH2-mutant AML, Agios said.
Some data from this study have already been announced and presented at scientific meetings, but an expansion to enroll additional AML patients was completed in the spring. Results from the expansion cohorts of the study have not yet been announced.
AG-221, also known as enasidenib, is a pill designed to block a mutated metabolic enzyme known as IDH2. Normally, cells use IDH2 to help break down nutrients and generate energy. When mutated, IDH2 alters the genetic programming of cells, preventing them from maturing and causing them to grow uncontrollably.
Patients with AML have bone marrow crowded with undifferentiated, rapidly dividing blast cells. AG-221 blocks the mutated IDH2 enzyme and allows the leukemic cells in the bone marrow to mature into normal blood cells.
Agios is developing a second drug known as AG-120, which works by blocking a related mutated enzyme known as IDH1. Agios owns AG-120 outright and intends to explore a similar expedited FDA approval filing that could be completed in 2017, the company said.
Agios shares closed Tuesday at $37.32. The stock has lost 43% of its value this year. In premarket trading, it is rising by 10%.