GBT Reports Recent Business Progress and Fourth Quarter and Full Year 2020 Financial Results – GlobeNewswire

Achieved Oxbryta (voxelotor) net revenues of $41.3 million in the fourth quarter and$123.8 million for full year 2020

Further established leadership in sickle cell disease (SCD) with data presentations on pipeline programs inclacumab and GBT021601 (GBT601)

Conference call today at 4:30 p.m. ET

SOUTH SAN FRANCISCO, Calif., Feb. 24, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported recent business progress and financial results for the fourth quarter and year ended December 31, 2020.

In 2020, GBT adapted to the challenging conditions of the global COVID-19 pandemic to safely and successfully execute the launch of Oxbryta. It is a testament to our teams dedication and the need for innovative therapies for sickle cell disease that we ended the year with thousands of patients on Oxbryta, an established base of prescribers, broad payer coverage, and a growing body of real-world evidence demonstrating statistically significant decreases in VOCs and transfusions and improved overall health-status. In addition, we announced robust progress for our pipeline with plans to advance inclacumab and GBT601 in 2021. We believe both these programs have the potential to become best-in-class SCD therapies, further expanding our leadership in SCD, said Ted W. Love, M.D., president and CEO of GBT.

We continue to be confident in the long-term potential of Oxbryta and are well positioned to build on our strong fundamentals to enhance our commercialization efforts in the U.S. and accelerate adoption. Furthermore, we continue to make important progress on our goal to provide access to Oxbryta to more SCD patients. We will also continue to support the SCD community and efforts to improve access to care, which has become even more important because of the impact of the COVID-19 pandemic that has dramatically magnified long-standing health inequities, added Dr. Love.

Recent Business Progress

Commercial

Clinical

Corporate

Financial Results for the Fourth Quarter and Year-End 2020Total net product sales for the fourth quarter of 2020 was $41.3 million, resulting from sales of Oxbryta, compared with $2.1 million for the same period in 2019. Net product sales resulted from sales of Oxbryta, which was launched in the United States in December 2019. Total net product sales for the full year 2020 was $123.8 million, compared with $2.1 million for the same period in 2019.

Cost of sales for the three months ended December 31, 2020, was $1.0 million compared with $48,000 for the same period in 2019. Total cost of sales for the full year 2020 was $2.0 million, compared with $48,000 for the same period in 2019. Manufacturing costs incurred prior to FDA approval of Oxbryta in November 2019 were previously recorded as research and development expense in the companys consolidated statement of operations. GBT expects that the cost of Oxbryta sales as a percentage of revenue will increase in future periods as product manufactured prior to FDA approval, and therefore fully expensed, is utilized.

Research and development (R&D) expenses for the three months endedDecember 31, 2020, were$41.1 millioncompared with$65.0 millionfor the same period in 2019. R&D expense in the fourth quarter 2019 included a $20 million upfront expense incurred in December 2019 related to the companys Syros collaboration agreement. Excluding this expense, the decrease in R&D expense compared to the prior year was primarily due to lower expense as a result of capitalization of Oxbryta manufacturing costs to inventory, which was partially offset by higher pre-clinical development activities under the Syros collaboration and other clinical programs. Following FDA approval of Oxbryta in November 2019, GBT capitalizes manufacturing of Oxbryta to inventory. R&D expenses for the year endedDecember 31, 2020, were$155.1 millioncompared with$174.6 millionfor the same period in 2019. The year-over-year decrease was primarily due to a $33.9 million decrease in Oxbryta related manufacturing costs and medical affairs costs that were previously expensed to R&D and a $1.7 million decrease in other preclinical programs. The decrease was partially offset by a $16.3 million increase in external costs associated with inclacumab and driven by the manufacturing activities. Total R&D non-cash stock compensation expense incurred for the three months ended December 31, 2020, was $3.7 million compared with $5.3 million for the same period in 2019. Total R&D non-cash stock compensation expense incurred for the year ended December 31, 2020, was $18.1 million compared with $19.1 million for the same period in 2019.

Selling, general and administrative (SG&A) expenses for the three months endedDecember 31, 2020, were$58.6 millioncompared with$44.6 millionfor the same period in 2019. SG&A expenses for the year endedDecember 31, 2020, were$210.9 millioncompared with$117.1 millionfor the same period in 2019. The increase in SG&A expenses for both comparative periods was primarily attributable to increased employee-related costs, including non-cash stock compensation expense, and increased professional and consulting services associated with the build-out of the companys commercial operations and launch of Oxbryta. Total SG&A non-cash stock compensation expense incurred in the three months endedDecember 31, 2020, was$14.3 million compared with$7.5 millionfor the same period in 2019. Total SG&A non-cash stock compensation expense incurred in the year endedDecember 31, 2020, was$53.4 million compared with$26.5 millionfor the same period in 2019.

A non-cash gain on the companys lease modification for the year ended December 31, 2020, was $1.0 million compared with $8.3 million for the same period in 2019. This is a non-recurring item related to the move to the companys new headquarters in 2020 and related termination of its previous lease in 2019.

Net loss for the three months endedDecember 31, 2020, was$61.8 millioncompared with$96.0 millionfor the same period in 2019. Basic and diluted net loss per share for the three months endedDecember 31, 2020, was$1.00compared with$1.59for the same period in 2019. Net loss for the year ended December 31, 2020, was $247.6 million compared with $266.8 million for the same period in 2019. Basic and diluted net loss per share for the year ended December 31, 2020, was $4.04 compared with $4.57 for the same period in 2019. The company expects its operating costs to increase during 2021 due to costs associated with expanding commercialization activities related to Oxbryta and the advancement of its clinical pipeline.

Cash, cash equivalents, and marketable securities totaled$560.9 millionatDecember 31, 2020, compared with$695.0 millionatDecember 31, 2019.

Conference Call DetailsGBT will host a conference call today,Wednesday, February 24, 2021, at4:30 p.m. ET to provide a general business update and discuss the financial results for the fourth quarter and full year 2020. To participate in the conference call, please dial 877-407-3982 (domestic) or 201-493-6780 (international). A live audio webcast of the conference call can be accessed on GBTs website at http://www.gbt.com under the Investors section. An archived audio webcast will be available for one month following the event.

About Sickle Cell DiseaseSickle cell disease (SCD) affects an estimated 100,000 people inthe United States,1an estimated 52,000 people inEurope,2and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1It also affects people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.1SCD 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.3Due 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.3-5The 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 Oxbryta(voxelotor) tabletsOxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobins 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, which are primary pathologies faced by every single person living with SCD. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that Oxbryta has the potential to modify the course of SCD. OnNovember 25, 2019, Oxbryta receivedU.S. Food and Drug Administration(FDA) accelerated approval for the treatment of SCD in adults and children 12 years of age and older.8As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy 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. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from theEuropean Medicines Agency(EMA), and theEuropean Commission(EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.

The EMA has accepted for review GBTs MAA seeking full marketing authorization of Oxbryta in the European Union to treat hemolytic anemia in SCD patients ages 12 years and older. GBT also plans to seek regulatory approval to expand the potential use of Oxbryta inthe United Statesfor the treatment of SCD in children as young as 4 years old.

Important Safety InformationOxbryta should not be taken if the patient has had an allergic reaction to voxelotor or any of the ingredients in Oxbryta. See the end of the patient leaflet for a list of the ingredients in Oxbryta. Oxbryta can cause serious side effects, including serious allergic reactions. Patients should tell their healthcare provider or get emergency medical help right away if they get rash, hives, shortness of breath, or swelling of the face.

Patients receiving exchange transfusions should talk to their healthcare provider about possible difficulties with the interpretation of certain blood tests when taking Oxbryta.

The most common side effects of Oxbryta include headache, diarrhea, stomach (abdominal) pain, nausea, tiredness, rash, and fever. These are not all the possible side effects of Oxbryta.

Before taking Oxbryta, patients should tell their healthcare provider about all medical conditions, including if they have liver problems; if they are pregnant or plan to become pregnant as it is not known if Oxbryta can harm an unborn baby; or if they are breastfeeding or plan to breastfeed as it is not known if Oxbryta can pass into breastmilk or if it can harm a baby. Patients should not breastfeed during treatment with Oxbryta and for at least 2 weeks after the last dose.

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Some medicines may affect how Oxbryta works. Oxbryta may also affect how other medicines work.

Patients are advised to call their doctor for medical advice about side effects. Side effects can be reported to FDA at 1-800-FDA-1088. Side effects can also be reported toGlobal Blood Therapeuticsat 1-833-428-4968 (1-833-GBT-4YOU).

Full Prescribing Information for Oxbryta is available atOxbryta.com.

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 companys next generation hemoglobin S polymerization inhibitor. In addition, GBTs drug discovery teams are working on new targets to develop the next wave of treatments for SCD. To learn more, please visitwww.gbt.comand 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 GBTs 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 GBTs priorities, commitment, dedication, focus, goals, and vision; the safety, efficacy, and mechanism of action of Oxbryta, and other product characteristics; the commercialization, delivery, availability, use, adoption, and commercial and medical potential of Oxbryta; building on fundamentals and enhancing commercialization efforts; use of telemedicine and provider and patient interaction; payer coverage for Oxbryta; ongoing and planned studies and related protocols, activities, and expectations; GBTs financial position, outlook, guidance, and expectations; the COVID-19 pandemic and related expectations; the expanded access protocol and early access program for Oxbryta, including the potential availability, use and impact; providing access to Oxbryta to more patients; regulatory submissions, review and approval to potentially expand the approved use of Oxbryta for more patients in the U.S. and to treat patients in Europe; supporting the SCD community and impacting the treatment, care and course of SCD; expanding GBTs leadership in SCD; the potential and advancement of GBTs pipeline, including inclacumab and other product candidates; 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 GBTs 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 GBTs control including, without limitation, risks and uncertainties relating to the COVID-19 pandemic, including the extent and duration of the impact on GBTs 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 GBTs dependence on third parties for development, manufacture, distribution and commercialization activities related to Oxbryta; government and third-party payer 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 GBTs and Syros research and development activities under their collaboration; along with those risks set forth in GBTs Annual Report on Form 10-K for the fiscal year ended December 31, 2019, and in GBTs 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 GBTs 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

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Contact:Steven Immergut(media)650.410.3258simmergut@gbt.com

Courtney Roberts(investors)650.351.7881croberts@gbt.com

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GBT Reports Recent Business Progress and Fourth Quarter and Full Year 2020 Financial Results - GlobeNewswire