Press Release
GBT Reports Recent Business Progress and Third Quarter 2020 Financial Results
Achieved Oxbryta® (voxelotor) net revenues of $36.9 million with more than 1,000 new patient prescriptions in the third quarter and net revenues of
New Oxbryta data and new research on GBT’s pipeline to be presented at the 2020
Conference call today at
“Our team continued to successfully execute on the launch of Oxbryta during the third quarter, expanding access and adoption of this first-in-class treatment that directly targets the underlying cause of sickle cell disease (SCD). We delivered an increase in new prescriptions despite a significant increase in COVID-19 infections in the
“During the quarter, we took another step toward making Oxbryta available globally with a distribution agreement aimed at providing access to the large number of sickle cell patients in the
Recent Business Progress
Commercial
- Achieved Oxbryta (voxelotor) net sales of
$36.9 million and$82.5 million in the three and nine months endedSeptember 30, 2020 , respectively. - Recorded more than 1,000 new prescriptions of Oxbryta in the quarter, despite a dramatic increase in COVID-19 cases in the
U.S. during the third quarter. - The growth in new prescriptions from the second quarter reflects ongoing increases in the use of telemedicine by healthcare providers, virtual engagements with GBT field teams and in-person visits in some geographies. New prescriptions were higher in August and September, with some weeks nearing pre-pandemic levels. When the pandemic subsides, GBT expects that, over time, the number of new prescriptions will further improve and surpass pre-pandemic levels.
- Secured broad Oxbryta reimbursement coverage one quarter ahead of expectations, with 90% of lives covered by payers either through published policies or verified patient adjudication by the end of the third quarter. GBT has secured fee-for-service Medicaid coverage in 44 states, including all 17 priority states where most SCD patients live.
Clinical
- Received acceptance of nine abstracts on GBT’s SCD programs to be presented at the 62nd
American Society of Hematology (ASH) Annual Meeting & Exposition, which will be held onDecember 5-8 . The presentations will include the 72-week analysis of the Phase 3 HOPE Study, real-world experience with Oxbryta, preclinical data highlighting the promise of the company’s SCD pipeline, including inclacumab and the company’s next generation hemoglobin S polymerization inhibitor. - Announced that the company will host a virtual Analyst & Investor Day on
December 7 to review data being presented at the 2020 ASH Annual Meeting. - Presented two abstracts that provide greater insight into the safety and efficacy of Oxbryta at the 15th Annual
Scientific Conference on Sickle Cell and Thalassemia (ASCAT) and 1stEHA European Sickle Cell Conference .
Corporate
- Announced an exclusive agreement with Biopharma-Middle East and
Africa (Biopharma-MEA) to distribute Oxbryta inBahrain ,Kuwait ,Oman ,Qatar ,Saudi Arabia , and theUnited Arab Emirates , collectively known as theGulf Cooperation Council (GCC) region. There are estimated to be more than 100,000 people age 12 years and older in this region living with SCD.1 - Hosted the 9th Annual
SCD Therapeutics Conference , which highlighted recent advances and future trends in the treatment of SCD, and the impact of COVID-19 on this vulnerable patient population. - Strengthened the company’s leadership team with the appointment of
Rajiv Patni , M.D., as chief medical officer.Dr. Patni joined GBT from Portola Pharmaceuticals, where he was the chief medical officer. He brings 20 years of biopharmaceutical product development experience, including 16 programs and eight regulatory approvals, across the cardiology, diabetology, dermato-oncology, hepatology, neurology, and hematology therapeutic areas.
- Received the 2020 Rare Impact Award® for Industry Innovation for Oxbryta from the
National Organization for Rare Disorders (NORD); in addition, Oxbryta was selected as Breakthrough Drug of the Year by the 2020 National Xconomy Awards.
Financial Results for the Third Quarter 2020
Total net product sales for the third quarter of 2020 was
Cost of sales for the three months ended
Research and development (R&D) expenses for the three months ended September 30, 2020, were $40.2 million compared with $39.1 million for the same period in 2019. The increase in R&D expense was primarily due to increased external costs related to GBT’s inclacumab program and preclinical research activities related to the collaboration with Syros Pharmaceuticals, Inc., which were partially offset by decreased manufacturing costs for Oxbryta. Following FDA approval of Oxbryta in
Sales, general, and administrative (SG&A) expenses for the three months ended September 30, 2020, were $54.5 million compared with $29.7 million for the same period in 2019. The increase in SG&A expenses for this comparative period 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 company’s commercial operations and launch of Oxbryta. Total SG&A non-cash stock compensation expense incurred in the three months ended September 30, 2020, was $14.9 million compared with $7.3 million for the same period in 2019.
Net loss for the three months ended September 30, 2020, was $59.9 million compared with $64.5 million for the same period in 2019. Basic and diluted net loss per share for the three months ended September 30, 2020, was $0.97 compared with $1.07 for the same period in 2019. The company expects its operating costs to increase in subsequent quarters due to costs associated with expanding commercialization activities as well as costs associated with the advancement of its clinical pipeline.
Cash, cash equivalents, and marketable securities totaled $535.2 million on
Conference Call Details
GBT will host a conference call today, Thursday, November 5, 2020, at 4:30 p.m. ET to provide a general business update and discuss the financial results for the third quarter 2020. To participate in the conference call, please dial 877-252-3031 (domestic) or 312-281-1210 (international). A live audio webcast of the conference call can be accessed on GBT’s website at www.gbt.com under 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 an estimated 100,000 people in
About Oxbryta® (voxelotor) tablets
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. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, 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.9 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 Oxbryta in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated Oxbryta as an orphan medicinal product for the treatment of patients with SCD.
GBT plans to seek regulatory approvals to expand the potential use of Oxbryta in the United States for the treatment of SCD in children age 4 to 11 years and to treat hemolytic anemia in SCD in people age 12 years and older in Europe.
Important Safety Information
Oxbryta 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 two 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 to Global Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Full Prescribing Information for Oxbryta is available at Oxbryta.com.
About
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, and vision; the safety, efficacy, and mechanism of action of Oxbryta, and other product characteristics; the commercialization, delivery, availability, and commercial and medical potential of Oxbryta; increases in use of telemedicine, virtual engagements and in-person visits; use of Oxbryta, including new prescriptions and prescribers and related expectations; payer coverage for Oxbryta; ongoing and planned studies of Oxbryta and related protocols, activities, and expectations; GBT’s financial position, outlook, guidance, and expectations; the COVID-19 pandemic and related expectations; the potential expansion of the approved use of Oxbryta for more patients in the U.S. and potential approval of Oxbryta to treat patients in
References
- Data on file.
- 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.
- Oxbryta (voxelotor) tablets prescribing information.
South San Francisco, Calif. Global Blood Therapeutics, Inc. ;November 2019 .
Condensed Consolidated Statements of Operations
(In thousands, except share and per share amounts)
Three Months Ended September 30, |
Nine Months Ended September 30, |
|||||||||||
2020 | 2019 | 2020 | 2019 | |||||||||
(Unaudited) | (Unaudited) | (Unaudited) | (Unaudited) | |||||||||
Product sales, net | $ | 36,889 | $ | — | $ | 82,508 | $ | — | ||||
Costs and operating expenses: | ||||||||||||
Cost of sales | 513 | — | 1,025 | — | ||||||||
Research and development | 40,196 | 39,088 | 114,054 | 109,564 | ||||||||
Selling, general and administrative | 54,491 | 29,654 | 151,227 | 72,503 | ||||||||
Total costs and operating expenses | 95,200 | 68,742 | 266,306 | 182,067 | ||||||||
Loss from operations | (58,311 | ) | (68,742 | ) | (183,798 | ) | (182,067 | ) | ||||
Other income (expense): | ||||||||||||
Interest income | 881 | 4,372 | 5,251 | 11,909 | ||||||||
Interest expense | (2,291 | ) | (146 | ) | (6,887 | ) | (487 | ) | ||||
Other expenses, net | (160 | ) | (31 | ) | (313 | ) | (146 | ) | ||||
Total other income (expense), net | (1,570 | ) | 4,195 | (1,949 | ) | 11,276 | ||||||
Net loss | $ | (59,881 | ) | $ | (64,547 | ) | $ | (185,747 | ) | $ | (170,791 | ) |
Basic and diluted net loss per common share | $ | (0.97 | ) | $ | (1.07 | ) | $ | (3.04 | ) | $ | (2.96 | ) |
Weighted-average number of shares used in computing basic and diluted net loss per common share | 61,573,877 | 60,098,093 | 61,160,984 | 57,637,318 | ||||||||
Condensed Consolidated Balance Sheets
(In thousands)
September 30, 2020 | |||||||||
Assets | (Unaudited) | ||||||||
Current assets: | |||||||||
Cash and cash equivalents | $ | 418,402 | $ | 302,237 | |||||
Short-term marketable securities | 113,523 | 307,732 | |||||||
Other current assets | 62,739 | 18,028 | |||||||
Total current assets | 594,664 | 627,997 | |||||||
Property and equipment, net | 38,697 | 27,113 | |||||||
Long-term marketable securities | 3,275 | 85,030 | |||||||
Operating lease right-of-use assets | 51,337 | 52,775 | |||||||
Other assets | 3,291 | 3,184 | |||||||
Total assets | $ | 691,264 |
$ | 796,099 |
|||||
Liabilities and Stockholders’ Equity | |||||||||
Current liabilities | $ | 77,946 | $ | 71,453 | |||||
Long-term debt | 73,880 | 73,559 | |||||||
Operating lease liabilities, noncurrent | 80,439 | 72,359 | |||||||
Other noncurrent liabilities | 1,344 | 34 | |||||||
Total liabilities | 233,609 | 217,405 | |||||||
Total stockholders’ equity | 457,655 | 578,694 | |||||||
Total liabilities and stockholders’ equity | $ | 691,264 | $ | 796,099 | |||||
Contact:
Steven Immergut
650-410-3258
simmergut@gbt.com
Source: Global Blood Therapeutics, Inc.