Press Release
Global Blood Therapeutics Announces Pivotal Study for GBT440 in Sickle Cell Disease with Primary Hemoglobin Endpoint
HOPE – A Phase 3 Clinical Trial to Initiate in December
Company to Host Conference Call and Webcast Today at
The Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study will be conducted at leading SCD sites globally and will enroll adults and adolescents with SCD 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 with top-line data anticipated in the first half of 2019.
“We are extremely pleased with our interactions and outcome of our pre-Phase 3 process with the
HOPE Study Design
The HOPE Study is a randomized, double-blind, placebo-controlled, multi-national, Phase 3 trial, which will enroll up to 400 patients age 12 and older with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year.
The HOPE Study will be conducted in two-parts:
Part A - Compare two dose levels of GBT440 – 900 mg and 1500 mg versus placebo. Part A will include up to 150 patients.
Part B - To include 250 patients randomized to placebo or a dose of GBT440 based on Part A.
The main objectives of Part A are to select the optimal dose, define the final secondary endpoints for Part B and qualify the Patient Reported Outcome (PRO) instrument.
The primary efficacy endpoint of the HOPE Study will be the proportion of patients who achieve a >1 g/dL increase in hemoglobin at 24 weeks of treatment vs baseline. Our discussions with the
Key secondary efficacy endpoints will include the effect of GBT440 on SCD symptom exacerbation, which will be measured by our PRO instrument, in addition to overall SCD symptoms as compared to placebo. The PRO is administered on a hand-held electronic device, and is designed to capture the full range of daily SCD symptoms. We will also assess traditionally defined VOCs as well as hospitalizations and red blood cell transfusions as secondary endpoints.
“Previous SCD studies have generally focused on VOC, defined as a painful crisis requiring hospital or emergency room utilization. But we know that patients have 4-5 times more frequent painful crises, with or without utilization. As a result, the burden of painful crises is dramatically under-reported,” said
Conference Call with Management
Management will host a conference call to provide a program update today at
The webcast will be archived under the investors and media section of the company’s website and will be available for replay for at least one month after the conference call.
About GBT440 in Sickle Cell Disease
GBT440 is being developed as an oral, once-daily therapy for patients with sickle cell disease (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 be capable of modifying the progression of SCD.
About Sickle Cell Disease (SCD)
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS. In its deoxygenated state, HbS has a propensity to polymerize, or bind together forming long, rigid rods within a red blood cell (
About
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, our ability to implement our clinical development plans for GBT440, the timing of the initiation of, and availability of top-line data from, our HOPE Study, our ability to enroll patients in the study, and the scope and number of endpoints required to be met to support regulatory approval, 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 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 or regulatory approval of our product candidates, and that drug-related or other 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
Contact Information:Myesha Edwards (investors)Global Blood Therapeutics 650-351-4730 investor@globalbloodtx.comJulie Normart (media) BrewLife 415-946-1087 media@globalbloodtx.com