Global Blood Therapeutics Announces New Data Supporting Ongoing Clinical Development of GBT440 in Adolescents with Sickle Cell Disease

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Global Blood Therapeutics Announces New Data Supporting Ongoing Clinical Development of GBT440 in Adolescents with Sickle Cell Disease

SOUTH SAN FRANCISCO, Calif., June 23, 2017 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced the presentation of preliminary results from the single-dose adolescent (age 12 to 17) cohort of the ongoing HOPE-KIDS 1 Study, a Phase 2a open-label study of GBT440 in adolescents with sickle cell disease (SCD). The results, which demonstrated that the pharmacokinetics of GBT440 are similar in adolescents and adults, were presented at the 22nd Congress of the European Hematology Association (EHA) in Madrid.

“Because sickle cell disease is a lifelong inherited condition that begins manifestation in childhood, it is critical that adolescent and pediatric patients are included in ongoing research efforts. We believe GBT440 could be particularly impactful in these young patients before much of the irreversible damage associated with SCD occurs,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Based on these encouraging pharmacokinetic and safety results, we are expanding our HOPE-KIDS 1 trial to evaluate GBT440 administered at doses of 900 mg and 1,500 mg per day, consistent with the doses currently being administered in the Phase 3 HOPE Study. These data will also be used to support enrolling adolescent patients in the HOPE Study and to inform future GBT440 dose selections for evaluation in a younger pediatric population of children age 6 to 11.”

Abstract #P620: The Pharmacokinetics (PK) of GBT440 Are Similar in Adolescents and Adults with Sickle Cell Disease (SCD)
HOPE-KIDS 1 (GBT440-007) is an open-label, single- and multiple-dose study that is evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440 in adolescents age 12 to 17 years with SCD. Results presented at EHA showed that, in adolescents who received a single oral dose of 600 mg of GBT440:

  • The pharmacokinetics and half-life of GBT440 were similar in adolescents and adults, with results supporting once-daily dosing and a high specificity for hemoglobin.
  • GBT440 was well tolerated, with no serious or severe adverse events related to study drug observed.

About Sickle Cell Disease (SCD)
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). In its deoxygenated state, HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which can cause blockage in capillaries and small blood vessels. Beginning in childhood, SCD patients suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities. This blocked blood flow, combined with hemolytic anemia (the destruction of RBCs), can eventually lead to multi-organ damage and early death.

About GBT440 in Sickle Cell Disease
GBT440 is being developed as an oral, once-daily therapy for patients with SCD. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes GBT440 blocks polymerization and the resultant sickling of red blood cells. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT believes that GBT440 may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted GBT440 both Fast Track and Orphan Drug designations for the treatment of patients with SCD, and the European Commission (EC) has designated GBT440 as an orphan medicinal product for the treatment of patients with SCD. GBT is currently evaluating GBT440 in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical trial in patients age 12 and older with SCD. Additionally, GBT440 is being studied in the ongoing Phase 1/2 GBT440-001 trial and in the ongoing HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in adolescents (age 12 to 17) with SCD designed to assess the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440.

About Global Blood Therapeutics
Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. GBT is developing its lead product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease. GBT is also investigating GBT440 for the treatment of hypoxemic pulmonary disorders in two ongoing Phase 2a studies in patients with idiopathic pulmonary fibrosis. To learn more, please visit and follow the company on Twitter: @GBT_News.

Forward-Looking Statements 
Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. We intend these forward-looking statements, including statements regarding the therapeutic potential and safety profile of GBT440 including in particular patient populations, our ability to implement our enrollment and other clinical development plans for GBT440 in both SCD and hypoxemic pulmonary disorders, our ability to generate and report data from our ongoing studies of GBT440, and the timing of these events, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, the risks that our clinical and preclinical development activities may be delayed or terminated for a variety of reasons, that regulatory authorities may disagree with our clinical development plans or require additional studies or data to support further clinical investigation of our product candidates, and that drug-related adverse events may be observed in later stages of clinical development, along with those risks set forth in our Annual Report on Form 10-K for the fiscal year ended December 31, 2016 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact Information:
Myesha Lacy (investors)

Julie Normart (media)
Pure Communications

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