Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
it has entered into an agreement with Sanofi S.A. to exclusively
in-license worldwide rights to two early-stage research programs in
sickle cell disease (SCD): one that pursues a novel anti-sickling
mechanism and another that leverages a new approach to reduce
inflammation and oxidative stress. These mechanisms are distinct
and potentially complementary to that of Oxbryta® (voxelotor)
tablets, a novel hemoglobin S polymerization inhibitor approved in
the United States for the treatment of SCD in patients ages 12
years and older. The programs, from Sanofi’s Bioverativ subsidiary,
supplement GBT’s existing pipeline and support the company’s
strategy to address SCD from multiple approaches.
“We envision a future in which sickle cell disease is a
well-managed condition with the potential for a functional cure in
the form of patient-friendly oral therapies. As we work toward this
vision and our goal to transform the treatment and care of people
living with this devastating disease, we are advancing our robust
internal research programs with disease-modifying potential while
continually exploring partnership opportunities across a variety of
mechanisms,” said Jung E. Choi, chief business and strategy officer
of GBT. “These novel discovery programs represent promising
approaches that we believe may have the potential to lead to
meaningful improvements for patients.”
Under the terms of the agreement, GBT will conduct all research,
development, regulatory and commercialization activities worldwide.
Sanofi will receive an upfront payment and is entitled to payments
up to approximately $353 million upon achievement of development,
regulatory and commercial milestones and single-digit tiered
royalties on worldwide net sales.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United
States,1 an estimated 52,000 people in Europe,2 and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa.1 It also affects
people of Hispanic, South Asian, Southern European and Middle
Eastern ancestry.1 SCD is a lifelong inherited rare blood
disorder that impacts hemoglobin, a protein carried by red blood
cells that delivers oxygen to tissues and organs throughout the
body.3 Due to a genetic mutation, individuals with SCD form
abnormal hemoglobin known as sickle hemoglobin. Through a process
called hemoglobin polymerization, red blood cells become sickled –
deoxygenated, crescent-shaped and rigid.3-5 The sickling
process causes hemolytic anemia (low hemoglobin due to red blood
cell destruction) and blockages in capillaries and small blood
vessels, which impede the flow of blood and oxygen throughout the
body. The diminished oxygen delivery to tissues and organs can lead
to life-threatening complications, including stroke and
irreversible organ damage.4-7
About Global Blood TherapeuticsGlobal
Blood Therapeutics (GBT) is a biopharmaceutical company
dedicated to the discovery, development and delivery of
life-changing treatments that provide hope to underserved patient
communities. Founded in 2011, GBT is delivering on its goal to
transform the treatment and care of sickle cell disease (SCD), a
lifelong, devastating inherited blood disorder. The company has
introduced Oxbryta® (voxelotor), the first FDA-approved
treatment that directly inhibits sickle hemoglobin polymerization,
the root cause of red blood cell sickling in SCD. GBT is also
advancing its pipeline program in SCD with inclacumab, a P-selectin
inhibitor in development to address pain crises associated with the
disease, and GBT021601 (GBT601), the company’s next-generation
hemoglobin S polymerization inhibitor. In addition, GBT’s drug
discovery teams are working on new targets to develop the next wave
of treatments for SCD. To learn more, please
visit https://gbt.com and follow the company on
Twitter @GBT_news.
Forward-Looking Statements Certain statements
in this press release are forward-looking within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements containing the words “will,” “anticipates,” “plans,”
“believes,” “forecast,” “estimates,” “expects” and “intends,” or
similar expressions. These forward-looking statements are based on
GBT’s current expectations and actual results could differ
materially. Statements 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. GBT intends these forward-looking statements, including
statements regarding GBT’s priorities, dedication, commitment,
focus, goals, mission and vision; the Sanofi agreement, including
rights, obligations and potential activities, results and payments
thereunder; the research programs under the Sanofi agreement,
including their mechanism of action and potential to complement
Oxbryta, supplement GBT’s pipeline, support GBT’s strategy, and
lead to improvements for patients; exploring partnership
opportunities; safety, efficacy and mechanism of action of Oxbryta
and other product characteristics; significance of reducing
hemolysis and raising hemoglobin; commercialization, delivery,
availability, use and commercial and medical potential of Oxbryta;
ongoing and planned studies and related protocols, activities and
expectations; altering the treatment, course and care of SCD and
mitigating related complications; potential and advancement of
GBT’s pipeline, including inclacumab and other product candidates;
and working on new targets and discovering, developing and
delivering treatments, 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
GBT makes this statement for purposes of complying with those safe
harbor provisions. These forward-looking statements reflect GBT’s
current views about its plans, intentions, expectations, strategies
and prospects, which are based on the information currently
available to the company and on assumptions the company has made.
GBT 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 GBT’s control, including, without
limitation, risks and uncertainties relating to the COVID-19
pandemic, including the extent and duration of the impact on GBT’s
business, including commercialization activities, regulatory
efforts, research and development, corporate development activities
and operating results, which will depend on future developments
that are highly uncertain and cannot be accurately predicted, such
as the ultimate duration of the pandemic, travel restrictions,
quarantines, social distancing and business closure requirements in
the U.S. and in other countries, and the effectiveness of
actions taken globally to contain and treat the disease; the risks
that GBT is continuing to establish its commercialization
capabilities and may not be able to successfully commercialize
Oxbryta; risks associated with GBT’s dependence on third parties
for development, manufacture, distribution and commercialization
activities related to Oxbryta; government and third-party payor
actions, including those relating to reimbursement and pricing;
risks and uncertainties relating to competitive products and other
changes that may limit demand for Oxbryta; the risks regulatory
authorities may require additional studies or data to support
continued commercialization of Oxbryta; the risks that drug-related
adverse events may be observed during commercialization or clinical
development; data and results may not meet regulatory requirements
or otherwise be sufficient for further development, regulatory
review or approval; compliance with obligations under the Pharmakon
loan; and the timing and progress of activities under GBT’s
research collaborations; along with those risks set forth in GBT’s
Annual Report on Form 10-K for the fiscal year ended December
31, 2020, filed with the U.S. Securities and Exchange
Commission, as well as discussions of potential risks,
uncertainties and other important factors in GBT’s subsequent
filings with the U.S. Securities and Exchange Commission.
Except as required by law, GBT assumes no obligation to update
publicly any forward-looking statements, whether as a result of new
information, future events or otherwise.
References
- Centers for Disease Control and Prevention website. Sickle
Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- European Medicines
Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed August 5, 2019.
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev.
2014;15(1):17-23.
Contact:Steven
Immergut (media)650.410.3258simmergut@gbt.com
Courtney
Roberts (investors)650.351.7881croberts@gbt.com
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