Agios to Present Clinical Data from Ongoing AG-120 Phase 1 Trial in Advanced Solid Tumors at AACR-NCI-EORTC

Investor Lunch and Webcast on Sunday, November 8, 2015

CAMBRIDGE, Mass., Oct. 26, 2015 -- Agios Pharmaceuticals (AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, today announced that the first results from the Phase 1 study of AG-120 in patients with IDH1-mutant positive advanced solid tumors will be presented in an oral presentation and featured in the press program at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics taking place November 5-9, 2015, in Boston.

BioBlast Announces Positive Interim Results From HOPEMD Phase 2 Clinical Study of Cabaletta(R) in Oculopharyngeal Muscular Dystrophy (OPMD)

Conference Call and Webcast, 8:30 a.m. Eastern Time

NEW HAVEN, Conn. and TEL AVIV, Israel, Oct. 27, 2015  -- BioBlast Pharma Ltd., (ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced positive interim results from a Phase 2 open label clinical study of its lead compound, Cabaletta^(R) (IV trehalose), in 25 patients with oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by severe swallowing difficulties (dysphagia) leading to malnutrition, dehydration, and aspiration of food into the lungs, as well as more generalized, progressive muscle weakness. Aspiration pneumonia and severe emaciation are frequently the cause of death.

Revive Therapeutics Announces US FDA Grants Orphan Drug Designation for Bucillamine for the Treatment of Cystinuria

TORONTO, ONTARIO--(Oct. 26, 2015) - Revive Therapeutics Ltd. ("Revive" or the "Company") (TSX VENTURE:RVV) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (US FDA) has granted orphan designation status for the use of the drug Bucillamine for the treatment of cystinuria. Orphan drug designation is granted to therapeutics treating rare diseases affecting less than 200,000 people in the U.S. The orphan drug designation qualifies Revive for various incentives such as a seven-year period of marketing exclusivity in the U.S., the potential for expedited drug development, and opportunities for drug grants and assistance in clinical research study design from the U.S. FDA.