Global Blood Therapeutics Announces Enrollment of First Patient in Phase 3 HOPE Study in Sickle Cell Disease

Global Blood Therapeutics Announces Enrollment of First Patient in Phase 3 HOPE Study in Sickle Cell Disease

SOUTH SAN FRANCISCO, Calif. – January 17, 2017 – Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced enrollment of the first patient in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a pivotal Phase 3 clinical trial of GBT440 in people with sickle cell disease (SCD). The HOPE Study will be conducted at leading SCD sites globally and is expected to enroll up to 400 adults and adolescents with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year.

“Enrolling the first patient in our pivotal Phase 3 HOPE Study is an important milestone for GBT as we continue to work to bring GBT440 to the SCD community,” said Ted W. Love, M.D., president and chief executive officer of GBT. “The innovative HOPE Study trial design has strong grounding in the mechanism of action of GBT440 and is designed to assess the efficacy of GBT440 in producing meaningful clinical improvements in anemia and daily symptoms. GBT440 has the potential to fundamentally modify the course of this devastating disease by inhibiting sickle hemoglobin polymerization, the fundamental cause of SCD pathophysiology.”

HOPE Study Design

The randomized, double-blind, placebo-controlled, multi-national trial will enroll patients age 12 and older with SCD who have had at least one episode of 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 selected from 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 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 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 U.S. Food and Drug Administration (FDA) has granted GBT440 both fast track and orphan drug designations and the European Commission (EC) has designated GBT440 as an orphan medicinal product, in each case for the treatment of patients with SCD. In addition to the Phase 3 HOPE Study and the ongoing Phase 1/2 GBT440-001 trial, GBT440 is being evaluated in an open-label, single and multiple dose study in adolescents (age 12 to 17) with SCD, to assess the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440.

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 (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which can cause blockage in 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.

Forward-Looking Statements

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: www.globalbloodtx.com.

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 clinical trials of GBT440 for sickle cell disease, including our ability to enroll patients in, conduct and complete 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 December 31, 2015, and in our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2016, June 30, 2016 and September 30, 2016, 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) GBT
650-351-4730 investor@globalbloodtx.com

Julie Normart (media) BrewLife
415-946-1087 media@globalbloodtx.com

Global Blood Therapeutics (GBT), Inc. Community Q & A Document

Global Blood Therapeutics (GBT), Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat blood-based disorders with significant unmet need. Our most advanced program, GBT440, is in development as a once-daily therapy taken by mouth for sickle cell disease.

At GBT, we are committed to advancing the science and transforming the treatment of sickle cell disease. We maintain an open dialogue with the sickle cell community to strengthen our understanding of the needs of the community and share the latest information on GBT’s sickle cell research, including our ongoing clinical trials.

Today January 18, 2017, we were pleased to announce via a press release, the enrollment of the first patient in the GBT HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a pivotal Phase 3 clinical study of our investigational drug candidate, GBT440, in people living with sickle cell disease.

The HOPE study has an innovative design that will allow GBT to utilize a primary endpoint that is a clinically meaningful measure of sickle cell disease, and a novel patient reported outcome (PRO) tool that will allow us to capture patients’ daily experience of pain, fatigue and other symptoms of sickle cell disease. We are deeply grateful to the patients, families, healthcare providers, and advocacy organizations that contributed their insights during the design of our clinical program and collaborated with us in the development of the PRO tool, which is designed to measure the true burden of painful crises and other sickle cell symptoms.

As we move forward with our efforts, we are committed to working closely with you, our patient advocacy community partners, to increase awareness of GBT440 and the HOPE study.

Following is a brief list of Questions & Answers to assist you, or others in your organization, address questions or concerns that your constituents may have regarding GBT440 and the HOPE study. You can also direct the community to the GBT HOPE Clinical Research Program website gbtsicklecellstudy.com that will be launched later in January. The website will have general information about the HOPE study, a pre-screener to help patients (or caregivers) determine if they may be eligible for the study or other sickle cell related studies GBT is conducting over time, and contact information of study sites in the United States.

If you have additional questions, please contact Eydith Comenencia Ortiz, Associate Director of Community Engagement & Partnership, at patientadvocacy@globalbloodtx.com.

 

GBT HOPE Study Q & A

What is GBT440?

GBT440 is a potential therapy for patients with sickle cell disease that is designed to attack sickle hemoglobin, the cause of the disease. GBT440 works by helping hemoglobin, the molecules inside red blood cells, hold onto more oxygen as the red blood cells travel through the body. This may help keep red blood cells in their normal shape and stop sickling.

In clinical studies to date, GBT440 has been well-tolerated and has reduced the damage to red blood cells caused by sickle cell disease. A large, ongoing global study (Phase 3 clinical trial), called the GBT HOPE study, will evaluate the safety and efficacy of GBT440 in adolescents and adults with sickle cell disease.

What is the GBT HOPE Study?

The GBT HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) study is a global clinical research study that will evaluate whether GBT440 will be effective in reducing anemia, and severity of daily symptoms due to sickle cell disease in adolescents and adults with sickle cell disease. It will also study the effects of different doses of GBT440 to determine the best dose to give to adolescents and adults with sickle cell disease. The study will utilize an increase in hemoglobin as a primary measure to evaluate the effectiveness of GBT440, and an electronic patient diary that is designed to capture patients’ daily experience and overall severity of symptoms due to their sickle cell disease.

The HOPE study is a randomized placebo-controlled clinical study, which means that participants will be assigned at random (like flipping a coin) to receive either the investigational medication, GBT440, or placebo. Placebo is a “dummy pill” that looks like the investigational medication but does not contain any drug.

After participants complete the study, they may be able to take GBT440 in an extension of the HOPE study, regardless of whether they received GBT440 or placebo during the study.

Who can participate in the HOPE Study?

The HOPE study will include up to 400 adolescents and adults living with sickle cell disease (ages 12 to 65) that have had at least one sickle cell crisis or vaso-occlusive crisis in the previous year.

Potential HOPE study participants need to go through a screening process to determine if they meet all the requirements for participation. Study doctors and staff will evaluate potential participants and explain other requirements for enrollment. If you or a loved one is interested in participating in the GBT HOPE study, go to gbtsicklecellstudy.com or talk to your doctor.

How do I know if there is a clinical study site near me?

The HOPE study is a global clinical trial that will include sites throughout the United States. There will also be study sites in Canada, and in some countries in Europe, the Middle East, and Africa. GBT is committed to working with sickle cell patient advocacy and community based organizations to provide information about clinical trial sites opening in your area. To find information about study sites in your area you can go to gbtsicklecellstudy.com talk to your doctor, or contact your local community-based organization(s).

podcast-icon-4753 CIRM & VetStem

WEBINAR – Creating a New SCD Clinic: A Case Study. Register Now: Jan. 25

Sickle Cell Data Collection

The California Rare Disease Surveillance Program invites you to attend a webinar, Creating a New SCD Clinic: A Case Study, to find out how a few dedicated professionals created a new county clinic in South Los Angeles, uniquely qualified to serve the population that needed its services the most. 

Doctors, public health and patient advocates will describe what it takes to create and maintain a new SCD clinic on Wednesday, January 25, 2017, from 10-11 am PST (1-2 pm EST).

 

Register here: 

https://attendee.gotowebinar.com/register/5503505776450805764

The agenda will include a brief update on California’s Sickle Cell Data Collection Program (the host of the webinar).

This webinar will be the sixth in a series of quarterly webinars featuring experts in sickle cell disease and updates on this project. Previous webinars are available at http://CASickleCell.org/CAData. This work is funded by the CDC Foundation, Pfizer Inc., and Biogen Inc., and is a partnership with CDC’s Division of Blood Disorders