Press Release
Global Blood Therapeutics Announces New Details on its Novel Patient Reported Outcomes (PRO) Tool to be used in Phase 3 HOPE Study
-- PRO designed to establish clinical benefit –
-- First time a PRO to be used as a secondary clinical endpoint in a SCD registrational study --
--Company to Host Webcast Today at
“Previous SCD studies have focused on vaso-occlusive crisis (VOC) defined as a painful crisis requiring hospital or emergency room utilization as the traditional clinical endpoint. However, studies have shown that VOC is not a full measure of the burden of SCD daily symptoms because many episodes of pain are managed at home rather than in a doctor’s office, hospital or emergency room and are thus under-reported. VOC is the tip of the iceberg,” said
PRO development used qualitative and quantitative analyses and was undertaken in collaboration with SCD patient groups and external experts, and with active engagement with the Clinical Outcomes Assessment group of the
“We are excited to have developed, in conjunction with the SCD community, the first PRO for SCD suitable for use as a clinical endpoint that could support potential
Also presented at ASH yesterday were data from the ongoing Phase 1/2 GBT440-001 study that further support the safety and efficacy profile of GBT440 as a potentially disease-modifying therapy for sickle cell disease (SCD) and results showing how GBT440 is metabolized in healthy subjects.
Investor Event Webcast Details
Today,
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 dramatically modify the course of SCD.
In recognition of the critical need for new SCD treatments, the
Additionally, GBT440 will be evaluated in the pivotal Phase 3 HOPE Study. This randomized, double-blind, placebo-controlled, multi-national trial 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 study will be conducted in two parts: Part A will compare GBT440 administered at doses of 900 or 1,500 mg per day vs. placebo in up to 150 patients treated for at least 12 weeks, and Part B will 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 PRO instrument. The primary efficacy endpoint of the HOPE Study is the proportion of patients who achieve a greater than 1 g/dL increase in hemoglobin at 24 weeks of treatment compared with baseline. Key secondary efficacy endpoints include the effect of GBT440 on SCD symptom exacerbation (as measured by the HOPE PRO instrument), overall SCD symptoms, traditionally defined VOCs, hospitalizations and red blood cell transfusions.
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 (
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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, and our ability to generate data from, our ongoing Phase 1/2 clinical trial of GBT440 and our ability to begin and generate data from our HOPE Study, and regarding the use of the PRO in our HOPE Study, 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
Contact Information:Myesha Lacy (investors)Global Blood Therapeutics 650-351-4730 investor@globalbloodtx.comJulie Normart (media) BrewLife 415-946-1087 media@globalbloodtx.com