Date: 2016-05-17
Type of information: Licensing agreement
Compound: AG-120
Company: Celgene (USA - NJ) Agios Pharmaceuticals (USA - MA)
Therapeutic area: Cancer - Oncology
Type agreement: licensing
Action mechanism: enzyme inhibitor/isocitrate dehydrogenase inhibitor. AG-120 is a first-in-class, orally available, selective, potent inhibitor of the mutated IDH1 (Isocitrate dehydrogenase 1) protein, and is a highly targeted investigational medicine for the treatment of patients with cancers that harbor an IDH1 mutation. Isocitrate dehydrogenase (IDH) 1 and 2 are metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies, including acute myelogenous leukemia (AML) and glioma, a type of aggressive brain tumor with poor prognosis. Normally, IDH enzymes help to break down nutrients and generate energy for cells. When mutated, IDH creates a molecule that alters the cells’ genetic programming, and instead of maturing, the cells remain primitive and proliferate quickly.
Disease: cancers harboring an IDH1 mutation
Details: * On January 12, 2015, Agios Pharmaceuticals, a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, announced that its collaboration partner Celgene Corporation has agreed that it will exercise its option to obtain an exclusive license outside the United States for AG-120, a first-in-class, oral, potent inhibitor of the mutant IDH1 protein under the terms of the 2010 collaboration agreement. This would be the second IDH mutant inhibitor to be licensed by Celgene in less than a year. AG-120 is currently being evaluated in two Phase 1 dose escalation trials, one in advanced hematologic malignancies and the other in advanced solid tumors. Both trials are evaluating AG-120 in patients whose cancer harbors an IDH1 mutation. The first data from the AG-120 program were presented at the EORTC-NCI-AACR Symposium on November 19, 2014 in advanced hematologic malignancies. Agios expects to report the first data from the Phase 1 advanced solid tumor trial at a medical conference in 2015. Celgene's exercise of the option is subject to receipt of any required regulatory approvals including any applicable clearance under the Hart-Scott-Rodino Act.
Financial terms: Agios and Celgene entered into a global strategic collaboration in April 2010 to develop new therapeutics targeting cancer metabolism. By exercising its exclusive option under the terms of the agreement, Celgene would lead development and commercialization outside the United States for AG-120, and Agios and Celgene would equally fund the global development costs of AG-120 that are not specific to any particular region or country. Celgene would be responsible for development and commercialization costs specific to countries outside the United States, and Agios would be responsible for development and commercialization costs specific to the United States.Celgene would be eligible to receive royalties on any net sales in the U.S. Agios would be eligible to receive royalties on any net sales outside the U.S. and up to $120 million in payments on achievement of certain milestones.
Latest news: * On May 17, 2016, Agios Pharmaceuticals and Celgene announced that they have modified certain rights from their 2010 collaboration. First, Agios, which previously held U.S. rights for AG-120, gained global development and commercialization rights to the program from Celgene. As of August 15, 2016 , neither party will have financial or other obligations to each other related to AG-120. There are no other changes to the existing IDH partnership between Agios and Celgene. Second, the companies agreed that rights to two cancer metabolism programs discovered under the 2010 Agreement, including a program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers, will advance under the structure of the new research collaboration outlined below. Following the expiration of the discovery phase of the 2010 Agreement on April 14, 2016 , all other cancer metabolism programs discovered at Agios will remain wholly owned by Agios.
Agios now has full global development and commercial rights for AG-120, a first-in-class, oral, potent inhibitor of mutant isocitrate dehydrogenase 1 (IDH1). Agios is studying AG-120 in AML in multiple clinical trials, including as a single agent in the relapsed/refractory setting as well as in combination with standard chemotherapy regimens in the frontline setting. Additionally, Agios plans to initiate pivotal trials in AML and is exploring the use of AG-120 in several solid tumors, including cholangiocarcinoma and glioma.
