Press Release
GBT Reports Recent Business Progress and Second Quarter 2020 Financial Results
Achieved Oxbryta® (voxelotor) net revenues of
Announced plans to seek approval of Oxbryta for younger pediatric patients in
Conference call today at
“In the second quarter of 2020, we continued to make important progress on GBT’s vision of being a leading biopharmaceutical company and the leader in sickle cell disease. Despite the impact of COVID-19, we continued to successfully execute the launch of Oxbryta and make significant progress on new prescriptions, new prescribers, and payer coverage. During the quarter, we were pleased to see increased use of virtual engagements and telemedicine by healthcare professionals and continued growth of Oxbryta prescriptions. In addition, we made significant progress in our efforts to expand the approved use of Oxbryta for more patients in
“Last quarter was also marked by a magnification of health disparities driven by COVID-19, which is having an outsized impact on African Americans, and a renewed focus on racial injustice sparked by deaths due to police brutality. The sickle cell disease community lives and breathes these issues, and GBT is working to address them, as evidenced by our cohort of ACCEL grant recipients. We are committed to enacting real and lasting change for communities of color, especially when it comes to ensuring patients have access to the care they need and deserve,” added
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) net sales of
$31.5 million in the second quarter of 2020. - Despite the impact of COVID-19, recorded approximately 1,000 new prescriptions of Oxbryta during the second quarter.
- The decrease in new prescriptions stabilized following an initial impact of COVID-19 at the end of the first quarter. The stabilization was primarily due to the increased use of telemedicine by healthcare providers and increased comfort with virtual engagements with GBT field teams. When the pandemic subsides, GBT expects that, over time, the number of new prescriptions will improve from current levels and surpass pre-COVID-19 levels.
- Conducted a review of patient charts, claims, and lab data that showed Oxbryta is being prescribed to a broad range of patients irrespective of hemoglobin (Hb) level, history of vaso-occlusive crisis (VOC), and current therapy for sickle cell disease (SCD). Almost half of patients started therapy with baseline Hb greater than 8 g/dL, and more than half take Oxbryta in combination with another SCD therapy.
- Continued to secure Oxbryta reimbursement coverage, with 53% of lives covered by payers either through published policies or medical exceptions. GBT has secured fee-for-service Medicaid coverage in 42 states, including all 17 priority states where most SCD patients live. GBT believes it is on track to meet its goal of obtaining broad coverage by the end of the year.
Clinical
- Collaborated with clinical trial sites to implement appropriate protocols that would, over time, enable the resumption of patient enrollment and participation in GBT-sponsored clinical trials. The company believes it remains on track with the long-term timelines for its clinical trials.
- Presented four abstracts at the virtual edition of the 25th Annual
European Hematology Association Congress :- A retrospective analysis of data from the landmark STOP 2 study linking higher Hb levels to lower transcranial Doppler flow velocity, a predictor of stroke risk in children with SCD.
- Three encore presentations of the pivotal Phase 3 HOPE Study that reinforced key attributes of Oxbryta, including that the lowest incidence of VOCs occurred in patients who achieved the highest Hb levels; that biomarkers of hemolysis decrease with increasing dose; and that the treatment effect seen with Oxbryta was independent of hydroxyurea use.
Corporate
- Announced plans to expand the potential use of Oxbryta for the treatment of SCD in children as young as 4 years old. GBT plans to submit a New Drug Application, which will include a new age-appropriate formulation, to the
U.S. Food and Drug Administration by mid-2021. - Announced plans to seek full marketing authorization in
Europe for Oxbryta to treat hemolytic anemia in SCD patients ages 12 years and older. GBT plans to submit a Marketing Authorization Application to theEuropean Medicines Agency by mid-2021. - Awarded
$250,000 in grants to five nonprofit organizations to advance access to care for people living with SCD through the company’s Access to Excellent Care for Sickle Cell Patients (ACCEL) program. The ACCEL program was established by GBT in 2019 to help fund programs that have the potential to deliver high-quality healthcare to people living with SCD.
Financial Results for the Second Quarter 2020
Total net product sales for the second quarter of 2020 was
Cost of sales for the three months ended
Research and development (R&D) expenses for the three months ended June 30, 2020, were $34.1 million compared with $36.0 million for the same period in 2019. The decrease in R&D expenses for this comparative period was primarily attributable to a decrease in manufacturing costs for Oxbryta. Following FDA approval of Oxbryta in
Sales, general, and administrative (SG&A) expenses for the three months ended June 30, 2020, were $49.1 million compared with $24.8 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 June 30, 2020, was $13.1 million compared with $6.2 million for the same period in 2019.
Net loss for the three months ended June 30, 2020, was $52.8 million compared with $57.3 million for the same period in 2019. Basic and diluted net loss per share for the three months ended June 30, 2020, was $0.86 compared with $1.01 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 $574.2 million at June 30, 2020, compared with $695.0 million at December 31, 2019.
Conference Call Details
GBT will host a conference call today, Wednesday, August 5, 2020, at 4:30 p.m. ET to provide a general business update and discuss the financial results for the second quarter 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 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.8 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.
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 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 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; use of Oxbryta, including new prescriptions and related expectations; payer coverage for Oxbryta; ongoing and planned studies of Oxbryta and related protocols, activities, and expectations, including with respect to resumption of enrollment and participation in clinical trials and the impact on related timelines; 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
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.
- 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 June 30, | Six Months Ended June 30, | |||||||||||
2020 | 2019 | 2020 | 2019 | |||||||||
(Unaudited) | (Unaudited) | (Unaudited) | (Unaudited) | |||||||||
Product sales, net | $ | 31,501 | $ | — | $ | 45,619 | $ | — | ||||
Costs and operating expenses: | ||||||||||||
Cost of sales | 377 | — | 512 | — | ||||||||
Research and development | 34,085 | 36,010 | 73,858 | 70,476 | ||||||||
Selling, general and administrative | 49,075 | 24,794 | 96,736 | 42,849 | ||||||||
Total costs and operating expenses | 83,537 | 60,804 | 171,106 | 113,325 | ||||||||
Loss from operations | (52,036 | ) | (60,804 | ) | (125,487 | ) | (113,325 | ) | ||||
Other income (expense): | ||||||||||||
Interest income | 1,514 | 3,706 | 4,370 | 7,537 | ||||||||
Interest expense | (2,282 | ) | (160 | ) | (4,596 | ) | (341 | ) | ||||
Other expenses, net | (36 | ) | (63 | ) | (153 | ) | (115 | ) | ||||
Total other income, net | (804 | ) | 3,483 | (379 | ) | 7,081 | ||||||
Net loss | $ | (52,840 | ) | $ | (57,321 | ) | $ | (125,866 | ) | $ | (106,244 | ) |
Basic and diluted net loss per common share | $ | (0.86 | ) | $ | (1.01 | ) | $ | (2.06 | ) | $ | (1.88 | ) |
Weighted-average number of shares used in computing basic and diluted net loss per common share |
61,116,707 | 56,539,760 | 60,952,269 | 56,386,560 | ||||||||
Condensed Consolidated Balance Sheets
(In thousands)
Assets | (Unaudited) | ||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 384,716 | $ | 302,237 | |||
Short-term marketable securities | 156,014 | 307,732 | |||||
Other current assets | 53,303 | 18,028 | |||||
Total current assets | 594,033 | 627,997 | |||||
Property and equipment, net | 39,668 | 27,113 | |||||
Long-term marketable securities | 33,479 | 85,030 | |||||
Operating lease right-of-use assets | 51,580 | 52,775 | |||||
Other assets | 2,907 | 3,184 | |||||
Total assets | $ | 721,667 |
$ | 796,099 | |||
Liabilities and Stockholders’ Equity | |||||||
Current liabilities | $ | 69,402 | $ | 71,453 | |||
Long-term debt | 73,775 | 73,559 | |||||
Operating lease liabilities, noncurrent | 81,903 | 72,359 | |||||
Other noncurrent liabilities | 771 | 34 | |||||
Total liabilities | 225,851 | 217,405 | |||||
Total stockholders’ equity | 495,816 | 578,694 | |||||
Total liabilities and stockholders’ equity | $ | 721,667 | $ | 796,099 |
Contact Information:
650-410-3258
simmergut@gbt.com
650-741-7730
syao@gbt.com
Source: Global Blood Therapeutics, Inc.