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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
_____________________
Form 8-K
_____________________
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event Reported): October 24, 2016
Global Blood Therapeutics, Inc.
Delaware
(State or Other Jurisdiction of
Incorporation)
(Exact Name of Registrant as Specified in Charter)
001-37539
27-4825712
(Commission File Number)
(I.R.S. Employer Identification Number)
400 East Jamie Court, Suite 101, South San Francisco, CA 94080
(Address of Principal Executive Offices) (Zip Code)
(650) 741-7700
(Registrant's telephone number, including area code)
Not applicable
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant
under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Item 5.02. Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory
Arrangements of Certain Officers.
On the conference call, the Company announced that its Chief Medical Officer, Dr. Eleanor Ramos, has retired from her position,
effective October 24, 2016.
Item 8.01. Other Events.
On October 24, 2016, Global Blood Therapeutics, Inc. (the “Company”) issued a press release titled, “Global Blood Therapeutics
Announces Pivotal Study for GBT440 in Sickle Cell Disease With Primary Hemoglobin Endpoint” (the “Press Release”). A copy of
the Press Release is filed herewith as Exhibit 99.1 and is incorporated herein by reference.
After issuing the Press Release, on October 24, 2016 the Company hosted a conference call to provide a program update, at which
time additional updates were provided, including those described in this Form 8-K. On the conference call, the Company estimated
that the external costs for the HOPE study are expected to be approximately $40 million over a multi-year period. Accordingly, the
Company disclosed that based upon its current operating plan, the Company expects its current cash and cash equivalents to extend
into the fiscal second quarter of 2018. The Company expects to provide operating expense guidance for its fiscal year 2017 at the
time of its annual filing on Form 10K.
In addition, on the conference call, the Company announced that it has decided to discontinue its research and development activities
related to GBT18713, an oral kallikrein inhibitor for the prevention of hereditary angioedema attacks.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
Exhibit No.
99.1
Description
Press Release, dated October
24, 2016.
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its
behalf by the undersigned hereunto duly authorized.
Global Blood Therapeutics, Inc.
Date: October 24, 2016
By:
/s/ Jeffrey Farrow
Jeffrey Farrow
Chief Financial Officer
(Principal Financial Officer)
EXHIBIT INDEX
Exhibit No.
Description
99.1
Press Release, dated October
24, 2016.
EXHIBIT 99.1
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 1:30 p.m. PT/ 4:30 p.m. ET
SOUTH SAN FRANCISCO, Calif., Oct. 24, 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 announced that it has reached agreement with the U. S. Food and Drug Administration (FDA)
regarding the design of its pivotal trial for GBT440 in adults and adolescents with sickle cell disease (SCD).
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 FDA. We believe that this
agreed-upon study design underscores our common goal to address the urgent unmet medical need for new disease-modifying
preventative therapies for SCD and the importance of innovative regulatory strategies to bring new therapies to market to treat these
patients,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Solidification of the path forward for our pivotal
study is an important milestone and we believe the HOPE Study design has a strong grounding in GBT440’s mechanism of action,
including its potential to fundamentally modify the course of the disease by inhibiting sickle hemoglobin polymerization.”
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 FDA have focused on a pathway to full approval based on the HOPE
Study, by meeting the primary and at least one key secondary endpoint.
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 Wally Smith, M.D., Florence Neal Cooper Smith Professor of Sickle Cell Disease
Director, a comprehensive sickle cell program at Virginia Commonwealth University. “By utilizing the PRO, the innovative design of
the HOPE Study should allow measurement of the true burden of SCD painful crises and other symptoms.”
Conference Call with Management
Management will host a conference call to provide a program update today at 1:30 p.m. PT/ 4:30 p.m. ET. To participate in the
conference call, please dial (844) 471-0808 (domestic) or (480) 696-7309 (international) and refer to conference ID 3206614. Live
audio of the conference call will be simultaneously webcast and will be available under the Investors and Media section of the
company’s website at www.globalbloodtx.com.
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.
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, in recognition of the critical need for new treatments. GBT440 is currently being evaluated in an ongoing Phase
1/2 clinical study. This randomized, placebo-controlled, double-blind, single and multiple ascending dose study is evaluating the
safety, tolerability, pharmacokinetics and pharmacodynamics of GBT440 in both healthy subjects and adults with SCD. Additionally,
in adolescents (age 12 to 17) with SCD, an open-label, single and multiple dose study, evaluating the safety, tolerability,
pharmacokinetics and exploratory treatment effect of GBT440 is ongoing.
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.
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. In its lead clinical program,
GBT is developing GBT440 as an oral, once-daily therapy for sickle cell disease (SCD) and is currently evaluating GBT440 in a
Phase 1/2 study in both healthy subjects and adults with SCD and a Phase 2a study in adolescents with SCD. GBT is also investigating
GBT440 for the treatment of hypoxemic pulmonary disorders in an ongoing Phase 2a study in patients with idiopathic pulmonary
fibrosis. To learn more, please visit: www.globalbloodtx.com.
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 December 31, 2015 and in our Quarterly Report on Form 10-Q for the quarter
ended June 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 Edwards (investors)
Global Blood Therapeutics
650-351-4730
[email protected]
Julie Normart (media)
BrewLife
415-946-1087
[email protected]