Global Blood Therapeutics Reports Recent Business Progress And Provides Third Quarter 2016 Financial Results

SOUTH SAN FRANCISCO, Calif., Nov. 09, 2016 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet need, today reported business progress and financial results for the third quarter ended September 30, 2016.

"We continue to make important progress across our GBT440 development programs and are particularly excited to have reached agreement with the FDA on key elements of our Phase 3 HOPE Study design for GBT440 in sickle cell disease (SCD)," said Ted W. Love, M.D., president and chief executive officer of GBT. "SCD is an urgent unmet medical need, and we believe the HOPE Study is uniquely designed to demonstrate the benefit of GBT440's mechanism of action. We look forward to initiating clinical trial sites for this Phase 3 study and beginning to screen patients by December."

Recent Business Progress

Sickle Cell Disease
  • Reached agreement with the U.S. Food and Drug Administration (FDA) on key elements of the pivotal program for GBT440 in SCD. The Phase 3 clinical trial, the HOPE ( Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polym Erization) Study, is a randomized, double-blind, placebo-controlled, multi-center pivotal trial. It is expected to enroll up to 400 SCD patients, age 12 and older who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year. The HOPE Study is expected to begin screening patients by December 2016.
  • Presented clinical data that continue to support the durability, safety and mechanism of action of GBT440 as a potentially disease-modifying therapy for SCD at the Academy for Sickle Cell and Thalassaemia Conference (ASCAT) in October. The data and results, which were previously presented at the European Hematology Association's 21 st Congress in June, included 90-day data from a cohort of patients in the ongoing Phase 1/2 GBT440-001 study who were taking 700 mg/day of GBT440 and 28-day data from three dosing cohorts of GBT440. The findings demonstrated that all SCD patients dosed with GBT440 have shown a positive hematologic response.
  • Joined the SCD Coalition, which is comprised of leading patient advocacy groups, researchers, clinicians, policymakers, industry and foundations, in a collective effort to improve SCD care, early diagnosis, treatment and research both in the United States and globally. During a press conference to launch the SCD Coalition, a call to action on SCD was announced, and the American Society of Hematology and several members of the SCD Coalition issued a new report, State of Sickle Cell Disease: 2016.
  • Announced publication of GBT440 preclinical data that were previously presented at the 57 th American Society of Hematology (ASH) Annual Meeting & Exposition in the online edition (doi: 10.1111/bjh.14264) of the British Journal of Haematology Review.
  • Announced publication of preclinical data highlighting the potential of GBT440 to reduce sickling and sickling-related complications in individuals with sickle cell trait in the online edition (Volume 8, No 3 - 2016) of Hematology Reports.
  • Received acceptance of three poster presentations at the upcoming 58 th ASH Annual Meeting & Exposition, which will be held December 3-6 in San Diego. The presentations will highlight data that support the ongoing GBT440 development program for SCD, including an overview of the Patient Reported Outcome (PRO) measurement tool that will be used in the Phase 3 HOPE Study.   

Idiopathic Pulmonary Fibrosis (IPF)
  • Announced publication of preclinical results that support the development of GBT440 for the potential treatment of hypoxemic pulmonary disorders, including IPF, in the online edition of Physiological Reports. The published study is the first to suggest that hemoglobin modifiers, such as GBT440, have the potential to increase oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues.

  • Announced the appointment of Tricia Suvari as chief legal officer. Ms. Suvari has approximately 20 years of experience in the life sciences industry and has served in senior roles at CV Therapeutics, Genentech and the non-profit Peninsula Open Space Trust.

Summary Financial Results for the Quarter Ended September 30, 2016

Cash and cash equivalents totaled $217.8 million at September 30, 2016, compared with $148.5 million at December 31, 2015.

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