Agios Pharmaceuticals, Inc. (Nasdaq:AGIO), a leader in the fields of cancer metabolism and rare genetic disorders of metabolism, announced the initiation of a Phase 1/2 multicenter study of AG-221 in patients with advanced solid tumors, including gliomas, as well as angioimmunoblastic T-cell lymphoma (AITL) that carry an isocitrate dehydrogenase-2 (IDH2) mutation. The study will enroll patients who have recurred or progressed following standard therapy or have not responded to prior standard therapy. This is the second trial to be initiated in patients with cancer as part of AG-221's clinical development program, which includes the ongoing Phase 1 trial with four expansion cohorts in patients with hematologic malignancies.

Preclinical evidence shows that mutant IDH2 enzyme, the target of AG-221, produces 2-hydroxyglutarate (2HG), which blocks the normal maturation of progenitor cells. In solid tumor cells expressing the IDH2 mutation, AG-221 inhibited the production of 2HG, which has the potential to affect differentiation and cell proliferation in patients with solid tumors. In addition, AG-221 has demonstrated an acceptable safety profile and evidence of antitumor activity in the ongoing Phase I trial of AG-221 in patients with advanced hematologic malignancies that carry an IDH2 mutation.

"Evaluating AG-221 in patients with advanced solid tumors is an important next step in our efforts to understand the potential of this investigational medicine to treat a broad range of cancers with the IDH2 mutation," said Chris Bowden M.D., chief medical officer at Agios. "The safety, pharmacokinetics, clinical activity, and effect on the biomarker 2HG we have observed from the different dose levels studied in the Phase 1 trial for advanced hematologic malignancies give us insights into the potential to fight cancer in patients with advanced solid tumors. AG-221 will only be evaluated in prospectively defined patients whose cancers carry an IDH2 mutation, and who we believe have the greatest potential to benefit from treatment."

Pluristem Therapeutics Inc. (NasdaqCM: PSTI), a leading developer of placenta-based cell therapy products, announced it has completed development of its second major product line, and can now begin manufacturing the cells on a large-scale at its state-of-the art facility in order to meet demand for anticipated studies in a range of hematologic conditions.

This second cell product candidate, called PLX-RAD, was created using Pluristem’s proprietary three dimensional cell expansion technology platform. The Company’s first product, PLX-PAD, is already in clinical trials for the treatment of peripheral artery disease, muscle injury and pulmonary arterial hypertension.

Pluristem’s development plan for the PLX-RAD cells considers numerous potential clinical indications such as: 1) enhancement of engraftment of transplanted hematopoietic stem cells for the treatment of  bone marrow deficiency, which can result from immune system disorders, genetic diseases, and treatment of leukemia and other blood cancers; 2) treatment of bone marrow deficiency in patients who have undergone chemotherapy; 3) treatment of acute radiation syndrome (ARS) in conjunction with the U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases.

“We’ve just completed a two-year development cycle for our PLX-RAD cells, and have also developed new manufacturing equipment, methods and know-how. We believe that our state-of-the-art technology platform can be used to create additional cell products from the placenta, tailored to potentially deliver targeted treatments for a variety of new indications,” stated Zami Aberman, Chairman and CEO of Pluristem.

“Our technology platform, robust manufacturing capabilities and broad IP portfolio open the door for potential institutional and commercial partners, and we’re pleased with the level of interest we have received in our technology platform. Pluristem is in a unique position to be a leader in the cell therapy industry,” Aberman concluded.

Data from a study of PLX-RAD cells conducted at Hadassah Medical Center were published in the peer-reviewed journal, PLOS ONE.