Press Release
GBT Reports First Quarter 2022 Financial Results
Achieved Oxbryta® (voxelotor) net revenues of
Strong new prescription growth driven by the recent launch for children ages 4 to less than 12 years and improving trends for patients ages 12 years and over
Reinitiated GBT601 Phase 1 trial in sickle cell disease (SCD) patients to study 150 mg daily dose; on track to initiate Phase 2/3 trial by mid-year
Conference call today at
“In the first quarter, we achieved two major milestones in our journey to expand patient access to Oxbryta,” said
“On the clinical front, we are enrolling patients in our two Phase 3 trials evaluating inclacumab for the potential reduction of the frequency of vaso-occlusive crises and related hospital readmissions. In addition, we expect to soon restart our GBT601 Phase 1 trial due to interest from the patients, which will also give us the opportunity to study an increased dose of 150 mg, and we plan to advance GBT601 into a Phase 2/3 trial by mid-year. We continue to be excited about GBT601, which we believe has the potential to improve clinical outcomes for people living with SCD, in a low-dose, once-daily pill,” added
Recent Business Progress
Commercial
- Recorded Oxbryta® (voxelotor) net sales of $55.2 million in the first quarter, an increase of 41% year over year. On a sequential basis, sales decreased 2%, driven primarily by distributors reducing inventory in the first quarter following purchases at the end of 2021 and a higher gross-to-net adjustment, partially offset by higher patient demand. The net number of patients taking Oxbryta increased compared to the prior quarter and has increased each quarter since launch in 2019.
- Achieved more than 1,200 new prescriptions for Oxbryta in the first quarter, including a strong launch in children ages 4 to less than 12 years, which began in early
January 2022 . GBT continues to believe that as the COVID-19 pandemic subsides, the number of new Oxbryta prescriptions will improve. GBT anticipates that eventually, in future periods, new prescriptions will surpass pre-COVID-19 levels. - Oxbryta continues to have broad payer coverage for patients ages 12 years and older, with more than 90% of covered lives having access through their healthcare plans. For patients ages 4 to less than 12 years, GBT continues to work towards securing Oxbryta reimbursement coverage and is on track to achieve its goal of broad payer coverage in this age group by mid-year.
- Launched Oxbryta in the
United Arab Emirates (UAE ) via GBT's distributor partnership with Biopharma-MEA. This is the first commercial launch of Oxbryta outside of theU.S. - Following the receipt of
European Commission (EC) marketing authorization inFebruary 2022 , GBT is actively working to commercially launch Oxbryta inGermany , which is anticipated by mid-May. In addition, GBT initiated reimbursement discussions inGermany ,France andEngland , and is educating healthcare providers acrossEurope on Oxbryta.
Clinical
- Enrollment is ongoing in two global, randomized, placebo-controlled, pivotal Phase 3 trials, collectively named THRIVE, evaluating the safety and efficacy of inclacumab, GBT’s P-selectin inhibitor, for the potential reduction of vaso-occlusive crisis (VOC) frequency and VOC-related hospital readmissions.
- In April, data from the Phase 2a HOPE-KIDS 1 study was published in Pediatric Blood & Cancer, an online peer-reviewed journal.
- GBT anticipates restarting in May the Phase 1 study of GBT021601 (GBT601), the company’s investigational next-generation sickle hemoglobin (HbS) polymerization inhibitor, to study a 150 mg daily dose, which is an increase over the 100 mg daily maintenance dose GBT previously studied. All six SCD patients that participated in the study in 2021 have expressed interest in participating in the restarted study. Upon completion, the company plans to submit this data for presentation at a medical meeting later this year. GBT is also planning to advance GBT601 into a Phase 2/3 trial, and anticipates initiating the Phase 2 portion by mid-2022.
- Results from the Oxbryta expanded access program for children (ages 4 to 11) with SCD will be presented in early May at the
American Society of Pediatric Hematology/Oncology Conference , reinforcing the efficacy and safety of treatment with Oxbryta in these patients. In addition, the majority of patients had improved scores as measured by the patient and clinical global impressions of change scale.
Corporate
- Anticipates receiving marketing authorization for Oxbryta in
Great Britain by mid-2022. There are approximately 15,0001 SCD patients inGreat Britain . - Strengthened the company’s research and development (R&D) team with the appointment of
R. Clark Brown , M.D., Ph.D., a world-renowned pediatric hematologist-oncologist and SCD expert, as vice president of integrated science and clinical site excellence, starting inJuly 2022 . In this new role,Dr. Brown will lead clinical research planning and implementation including initiatives to drive increased engagement at new and existing clinical trial sites. Previously,Dr. Brown served as the director of clinical research at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare ofAtlanta , the largest comprehensive SCD program in theU.S. - Published inaugural environmental, social and governance (ESG) report, demonstrating GBT’s progress with its ESG priorities, which include improving access to affordable care; investing in GBT employees and communities; increasing environmental sustainability; and upholding GBT’s ethics and values.
Financial Results for the First Quarter 2022
Total net product sales for the first quarter of 2022 were
Cost of sales for the three months ended
R&D expenses for the three months ended March 31, 2022, were $52.8 million, compared with $50.9 million for the same period in 2021. The increase in R&D expenses for the period was primarily due to an increase in external costs related to the GBT601 program, partially offset by a decrease in external costs related to Oxbryta. Total R&D non-cash stock compensation expense incurred for the three months ended
Sales, general, and administrative (SG&A) expenses for the three months ended March 31, 2022, were $74.5 million, compared with $59.0 million for the same period in 2021. The increase in SG&A expenses for the period was primarily attributable to increased professional and consulting services associated with the company’s expanded commercial and medical affairs operations for Oxbryta, including supporting the pediatric launch in the
Net loss for the three months ended March 31, 2022, was $81.4 million, compared with $74.9 million for the same period in 2021. Basic and diluted net loss per share for the three months ended March 31, 2022, was $1.26, compared with $1.21 for the same period in 2021.
Cash, cash equivalents, and marketable securities totaled $662.1 million at March 31, 2022, compared with $734.8 million at December 31, 2021.
Conference Call Details
GBT will host a conference call today, Wednesday, May 4, 2022, at 4:30 p.m. ET to discuss the financial results for the first quarter 2022 and provide a business update. To participate in the conference call, please dial 877-407-3982 (domestic) or +1 201-493-6780 (international). A live audio webcast of the conference call can be accessed on GBT’s website at www.gbt.com in the Investors section. An archived audio webcast will be available for one month following the event.
About Sickle Cell Disease
Sickle cell disease (SCD) affects more than 100,000 people in
About Oxbryta® (voxelotor) Tablets and Tablets for Oral Suspension
Oxbryta (voxelotor) 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, Oxbryta inhibits sickle hemoglobin polymerization and the resultant sickling and destruction of red blood cells leading to hemolysis and hemolytic anemia, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD.
In
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 received the prestigious 2021
Oxbryta has been granted Priority Medicines (PRIME) designation from the
Please click here for Important Safety Information and full Prescribing Information including Patient Information for Oxbryta in the
About
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, starting with sickle cell disease (SCD). Founded in 2011, GBT is delivering on its goal to transform the treatment and care of SCD, a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta® (voxelotor), the first FDA-approved medicine that directly inhibits sickle hemoglobin (HbS) 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 Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next generation HbS polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of potential treatments for SCD. To learn more, please visit www.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, commitment, dedication, focus, goals, mission, vision, and positioning; the safety, efficacy, and mechanism of action of Oxbryta, and other product characteristics; the commercialization, awareness, delivery, availability, use, and commercial and medical potential of Oxbryta, including the use, significance and potential of related initiatives; expanding patient access to Oxbryta; potential launch of Oxbryta in
References
NHS England . https://www.england.nhs.uk/wp-content/uploads/2018/08/Specialised-services-for-haemoglobinopathy-care-all-ages.pdf. Last accessedApril 2022 .Centers for Disease Control and Prevention website. Sickle Cell Disease Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html. Accessed February 23, 2022.European Medicines Agency . https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125 AccessedFebruary 23, 2022 .- Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed February 23, 2022.
- National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Rees DC, et al.
Lancet . 2010;376(9757):2018-2031. - Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- Oxbryta (voxelotor) tablets and tablets for oral suspension prescribing information. South San Francisco, Calif. Global Blood Therapeutics, Inc.; December 2021.
Condensed Consolidated Statements of Operations | ||||||||
(Unaudited) | ||||||||
(In thousands, except share and per share amounts) | ||||||||
Three Months Ended |
||||||||
2022 | 2021 | |||||||
Product sales, net | $ | 55,160 | $ | 39,043 | ||||
Costs and operating expenses: | ||||||||
Cost of sales | 1,055 | 584 | ||||||
Research and development | 52,833 | 50,857 | ||||||
Selling, general and administrative | 74,533 | 58,966 | ||||||
Total costs and operating expenses | 128,421 | 110,407 | ||||||
Loss from operations | (73,261 | ) | (71,364 | ) | ||||
Other income (expense): | ||||||||
Interest income (expense), net | (7,705 | ) | (3,360 | ) | ||||
Other expenses, net | (286 | ) | (83 | ) | ||||
Total other expense, net | (7,991 | ) | (3,443 | ) | ||||
Loss before income taxes | (81,252 | ) | (74,807 | ) | ||||
Provision for income taxes | 171 | 123 | ||||||
Net loss | (81,423 | ) | (74,930 | ) | ||||
Other comprehensive expense: | ||||||||
Net unrealized loss on marketable securities, net of tax | (1,915 | ) | (181 | ) | ||||
Cumulative translation adjustment | (147 | ) | — | |||||
Comprehensive loss | $ | (83,485 | ) | $ | (75,111 | ) | ||
Basic and diluted net loss per common share | $ | (1.26 | ) | $ | (1.21 | ) | ||
Weighted-average number of shares used in computing basic and diluted net loss per common share | 64,842,086 | 62,101,070 |
Condensed Consolidated Balance Sheets | ||||||
(In thousands) | ||||||
Assets | (Unaudited) | |||||
Current assets: | ||||||
Cash and cash equivalents | $ | 391,803 | $ | 684,717 | ||
Short-term marketable securities | 161,614 | — | ||||
Other current assets | 122,231 | 117,253 | ||||
Total current assets | 675,648 | 801,970 | ||||
Long-term marketable securities | 108,667 | 50,057 | ||||
Property and equipment, net | 33,598 | 34,918 | ||||
Operating lease right-of-use assets | 47,271 | 48,015 | ||||
Other assets, noncurrent | 4,359 | 4,248 | ||||
Total assets | $ | 869,543 | $ | 939,208 | ||
Liabilities and Stockholders’ Equity | ||||||
Current liabilities | 79,305 | 87,998 | ||||
Long-term debt, net | 246,738 | 246,352 | ||||
Convertible debt, net | 334,451 | 334,089 | ||||
Other liabilities, noncurrent | 75,047 | 74,359 | ||||
Total liabilities | 735,541 | 742,798 | ||||
Total stockholders’ equity | 134,002 | 196,410 | ||||
Total liabilities and stockholders’ equity | $ | 869,543 | $ | 939,208 |
Contact Information:
Steven Immergut (media)
+1 650-410-3258
simmergut@gbt.com
Courtney Roberts (investors)
+1 650-351-7881
croberts@gbt.com

Source: Global Blood Therapeutics, Inc.