SOUTH SAN FRANCISCO - Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported recent business progress and financial results for the fourth quarter and year ended December 31, 2019.

'2019 was a momentous year for both the sickle cell community and GBT as we saw the FDA approval of Oxbryta, the first medicine that directly inhibits sickle hemoglobin polymerization, the root cause of this devastating, lifelong genetic disease, three months ahead of schedule,' said Ted W. Love, M.D., president and chief executive officer of GBT. 'With the early investment we made in building out our commercial infrastructure, we were able to make Oxbryta available to patients and physicians within days of its approval. Patient access to this needed therapy is a top priority for us. We will continue to work closely with payers throughout 2020 to meet our goal of obtaining broad coverage by the end of the year. Most importantly, we are encouraged by the positive feedback we have received to date from physicians and patients who have started Oxbryta therapy. We are optimistic about achieving our vision of making sickle cell disease a well-managed chronic condition by establishing Oxbryta as a standard of care, expanding its approved label and availability around the world, and continuing to research new pathways and develop innovative new therapies.'

About Sickle Cell Disease

Sickle cell disease (SCD) affects an estimated 100,000 people in the United States and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.2 Due to a genetic mutation, people 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.2-4 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.4-6

About Oxbryta (voxelotor) tablets

Oxbryta (voxelotor), previously called GBT440, is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD. On November 25, 2019, Oxbryta received U.S. Food and Drug Administration (FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.7 As a condition of accelerated approval, GBT will continue to study voxelotor in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of Oxbryta to decrease stroke risk in children 2 to 15 years of age.

In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.

About Global Blood Therapeutics

Global 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 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. In addition, GBT's drug discovery teams are working on new targets to develop the next generation of treatments for SCD.

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, goals and vision, the significance of 2019 for the sickle cell community and GBT, the safety, efficacy and mechanism of action of Oxbryta and other product characteristics, the availability, use, commercialization and commercial and medical potential of Oxbryta, the need for Oxbryta and other SCD treatments, making SCD a well-managed condition, establishing Oxbryta as a standard of care, expanding its approved label and availability around the world, and researching new pathways and developing new therapies, working and meeting with payers and obtaining reimbursement for Oxbryta, transforming the treatment and care of SCD and establishing GBT as a leader in addressing blood disorders, ongoing studies of Oxbryta and related protocols, activities and expectations, GBT's collaboration with Syros and related rights, obligations, activities and expectations, the Pharmakon loan, including its significance, the use of its proceeds, the impact on GBT's use of its financial resources, and the availability of additional funds under the loan, GBT's financial position, outlook and expectations, including its financial runway and potential to achieve positive cash flow while continuing research and development, and advancing GBT's pipeline and discovering, developing and delivering innovative 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, compliance with the funding and other obligations under the Pharmakon loan, the timing and progress of GBT's and Syros' research and development activities under their collaboration, the amount and timing of resources devoted by each of such parties to activities under the collaboration, the risks that GBT has only recently established its commercialization capabilities and may not be able to successfully commercialize Oxbryta, risks associated with GBT's dependence on third parties for development, manufacture 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, and data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval, along with those risks set forth in GBT's Annual Report on Form 10-K for the fiscal year ended December 31, 2018, and in GBT's most recent Quarterly Report on Form 10-Q 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.

Contact:

Steven Immergut

Tel: 650-410-3258

(C) 2020 Electronic News Publishing, source ENP Newswire