On April 29, 2015 Agios Pharmaceuticals reported that it plans to advance into clinical development AG-881, a small molecule that has shown in preclinical studies to fully penetrate the blood brain barrier and inhibit isocitrate dehydrogenase-1 (IDH1) and IDH2 mutant cancer models, in collaboration with its cancer metabolism partner Celgene Corporation (Press release, Agios Pharmaceuticals, APR 29, 2015, View Source [SID:1234503204]). The companies have entered into a new joint worldwide development and profit share collaboration for AG-881 and plan to initiate clinical development of AG-881 in the second quarter of 2015. AG-881 will be the third IDH mutant inhibitor discovered by Agios to enter clinical development.

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"The addition of our third IDH mutant inhibitor to our growing pipeline is an exciting milestone for Agios and underscores our goals to lead the scientific understanding of cancer metabolism and help as many patients as possible with an IDH mutant positive cancer," said David Schenkein, M.D., chief executive officer of Agios. "AG-221 and AG-120 remain our lead medicines in clinical development and are advancing rapidly. We believe the addition of AG-881, given its unique profile, provides added flexibility to our portfolio of IDH inhibitors. Based on our preclinical findings, it has the potential to support our ongoing development effort to provide treatment options to patients with glioma, and it represents a possible second-generation molecule for both AG-221 and AG-120 in IDH mutant tumors. We look forward to generating data for AG-881 to inform our future development plans."

Under the terms of the new AG-881 collaboration, Agios will receive an initial payment of $10 million in the second quarter of 2015 and is eligible to receive regulatory milestone payments of up to $70 million. Agios and Celgene will jointly collaborate on the worldwide development program for AG-881, sharing development costs 50/50 worldwide. The two companies have agreed to share any worldwide profits 50/50, with Celgene booking worldwide commercial sales. Agios would lead commercialization in the U.S. with both companies sharing equally in field-based commercial activities, and Celgene would lead commercialization ex-U.S. with Agios providing one third of field-based commercial activities in the major E.U. markets.

Summary of Agios and Celgene Collaboration on IDH Mutant Inhibitors

Agios and Celgene entered a global, strategic collaboration in April 2010 and, to date, three potential new distinct investigational medicines have emerged – the IDH2 mutant inhibitor, AG-221; the IDH1 mutant inhibitor, AG-120; and the pan-IDH mutant inhibitor, AG-881, which as described above is now part of a new collaboration between the companies. These three investigational medicines aim to improve the treatment outcomes for patients whose cancers carry these IDH mutations, including difficult to treat acute myelogenous leukemia and glioma, a type of aggressive brain tumor with poor prognosis. Each of these investigational medicines carries different financial terms and rights under the collaboration, including:

AG-221: Celgene has worldwide development and commercialization rights for AG-221. Agios is eligible for up to $120 million in milestone payments and royalties on any net sales.
AG-120: Agios retains U.S. development and commercialization rights, while Celgene has development and commercialization rights outside the U.S. Agios is eligible to receive royalties on any net sales outside the U.S. and up to $120 million in milestone payments. Celgene is eligible to receive royalties on any net sales in the U.S.
AG-881: Joint worldwide development and 50/50 profit share collaboration. Agios is eligible to receive regulatory milestone payments up to $70 million.

On April 28, 2015 Bio-Path Holdings reported that they have received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its lead compound Liposomal Grb-2 for the treatment of acute myeloid leukemia (AML) (Filing, 8-K, Bio-Path Holdings, APR 28, 2015, View Source [SID:1234503201]). Orphan drug status provides Bio-Path with seven years of exclusivity after receiving formal marketing approval, as well as additional development incentives. The FDA grants this designation to certain drugs that are targeting diseases affecting fewer than 200,000 people in the United States.

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"This designation from the FDA demonstrates the unmet need for an effective therapy for patients suffering from AML," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "It also marks a key regulatory milestone for Bio-Path and will be valuable as we continue to progress Liposomal Grb-2 through clinical trials and toward potential commercialization."

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Liposomal Grb-2 is currently in a safety segment of a Phase 2 trial in combination with Ara-C, for acute myeloid leukemia; and is being evaluated for chronic myelogenous leukemia.

Pre-clinical development is underway for triple negative and inflammatory breast cancers.

About Acute Myeloid Leukemia

AML is a fast-growing cancer of the blood and bone marrow where the bone marrow makes many cancerous cells called leukemic blasts. Normal blasts develop into white blood cells that fight infection. In AML, the leukemic blasts do not develop properly and cannot fight infections. These leukemic blasts grow quickly and crowd out the bone marrow, preventing it from making the normal red blood cells, white blood cells, and platelets that the body needs.
Nearly 15,000 people in the United States are diagnosed with AML each year.

About Growth Receptor Bound protein-2 (Grb-2)

The adaptor protein Growth Receptor Bound protein-2 (Grb-2) is essential to cancer cell signaling because it is utilized by oncogenic tyrosine kinases to induce cancer progression. Suppressing the function or expression of Grb-2 should interrupt its vital signaling function and have a therapeutic application in cancer. BP-1001 is a neutral-charge, liposome-incorporated antisense drug substance designed to inhibit Grb-2 expression.