Press Release
GBT Reports Recent Business Progress and Fourth Quarter and Full Year 2019 Financial Results
Obtained FDA approval of Oxbryta® (voxelotor), the first treatment that specifically targets the root cause of sickle cell disease (SCD), three months ahead of schedule
Made Oxbryta available to adults and children 12 years of age and older with SCD within days of FDA approval
Maintained strong balance sheet with
Conference Call today at
“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
Recent Business Progress
Commercial
Received U.S. Food and Drug Administration (FDA) approval of Oxbryta for the treatment of SCD in adults and children 12 years of age and older. The accelerated approval came three months before the Prescription Drug User Fee Act (PDUFA) target action date ofFebruary 26, 2020 .- On the day of FDA approval, launched GBT Source™, a comprehensive Oxbryta support program for patients and physicians. GBT Source provides a wide range of practical, educational and financial support customized to each patient’s needs and eligibility. GBT Source also provides educational and support resources for physician practices and streamlined administrative processes for payers.
- Made Oxbryta available to patients and physicians through GBT’s specialty pharmacy partner network within days after approval.
- Began accepting patient enrollments into GBT Source and facilitating reimbursement coverage, primarily through medical exceptions, within days after approval.
- Completed the build-out and launch training of the company’s commercial organization.
- On track to complete most payer meetings to discuss Oxbryta’s approved label and price during the first half of 2020, to meet the company’s goal of obtaining broad coverage by the end of the year.
Clinical
- Initiated the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of Oxbryta to decrease the risk of stroke in children 2 to 15 years of age.
- Initiated a dose-optimization study designed to explore the potential safety and tolerability of Oxbryta at doses higher than 1500 mg.
- Presented eight abstracts at the 61st
American Society of Hematology (ASH) Annual Meeting & Exposition. Key abstracts included:- Three post-hoc analyses of the Phase 3 HOPE Study that provide greater insight into the safety and efficacy of Oxbryta for treating SCD:
- Oxbryta safely raised hemoglobin without evidence of viscosity-related complications. Rates of vaso-occlusive crises (VOCs) were inversely related to attained hemoglobin levels, and there was no evidence of an increased risk of VOC within 28 days of discontinuing treatment with Oxbryta as compared to placebo.
- Oxbryta-treated patients with >1 g/dL increase in hemoglobin showed the greatest reduction in markers of hemolysis, consistent with the mechanism of inhibition of sickle hemoglobin polymerization.
- Hemoglobin increases were comparable in Oxbryta-treated patients regardless of concomitant hydroxyurea use, providing reassurance that the increases seen with Oxbryta were not due to increased hydroxyurea compliance during the study.
- A longitudinal analysis of children treated with hydroxyurea showed that a therapeutic rise in hemoglobin levels was significantly associated with a reduction in TCD levels.
- An analysis demonstrating the economic burden of end organ damage among
U.S. patients with SCD, with predicted mean annual costs as high as$286,000 for patients in the first year after a stroke.
- Three post-hoc analyses of the Phase 3 HOPE Study that provide greater insight into the safety and efficacy of Oxbryta for treating SCD:
Corporate
- Entered into a collaboration with Syros Pharmaceuticals, Inc., to discover, develop and commercialize novel therapies for SCD and beta thalassemia. Syros will use its leading gene control platform to identify therapeutic targets and discover small-molecule drugs that potentially induce fetal hemoglobin. GBT has the option to obtain an exclusive worldwide license to develop, manufacture and commercialize therapies resulting from the collaboration.
- Entered into a
$150 million loan agreement with funds managed byPharmakon Advisors LP , and drew down on the first$75 million of the non-dilutive loan. The proceeds will be used to advance the discovery and development of potential novel treatments for SCD and other grievous blood-based conditions without diverting financial resources from the launch of Oxbryta. GBT believes that the proceeds from this loan, in conjunction with existing cash and investments, have the potential to provide the necessary runway for the company to achieve positive cash flow while enabling the continued advancement of clinical development programs and other earlier-stage product candidates. - Strengthened the company’s leadership team with the appointment of
Steven Immergut as senior vice president, head of corporate communications.
Financial Results for the Fourth Quarter and Year-End 2019
Total product sales, net for the fourth quarter of 2019 was
Cost of sales for the three months ended
Research and development (R&D) expenses for the three months ended December 31, 2019, were $65.0 million compared with $36.8 million for the same period in 2018. The increase in R&D expenses for this comparative period was primarily attributable to increased costs related to the company’s SCD program for Oxbryta, increased employee-related costs, including non-cash stock compensation expense, increased costs related to preclinical research and manufacturing activities for inclacumab, and a
Sales, general and administrative (SG&A) expenses for the three months ended December 31, 2019, were $44.6 million compared with $15.3 million for the same period in 2018. SG&A expenses for the year ended
A non-cash gain on the company’s lease modification for the three months ended
Net loss for the three months ended December 31, 2019, was $96.0 million compared with $49.2 million for the same period in 2018. Basic and diluted net loss per share for the three months ended December 31, 2019, was $1.59 compared with $0.93 for the same period in 2018. Net loss for the year ended
Cash, cash equivalents and marketable securities totaled $695.0 million at December 31, 2019, compared with $591.8 million at December 31, 2018.
Conference Call Details
GBT will host a conference call and webcast today, Wednesday, February 26, 2020, at 4:30 p.m. ET (
About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in
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
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
Indication
Oxbryta is a prescription medicine used for the treatment of sickle cell disease in adults and children 12 years of age and older. It is not known if Oxbryta is safe and effective in children below 12 years of age.
This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
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
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, 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
References
Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. AccessedJune 3, 2019 .- National Heart, Lung, and
Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. AccessedAugust 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 |
Year Ended |
||||||||||||||
2019 | 2018 | 2019 | 2018 | ||||||||||||
(Unaudited) | (Unaudited) | (Unaudited) | |||||||||||||
Product sales, net | $ | 2,108 | $ | — | $ | 2,108 | $ | — | |||||||
Costs and operating expenses: | |||||||||||||||
Cost of sales | 48 | — | 48 | — | |||||||||||
Research and development | 64,990 | 36,765 | 174,556 | 131,307 | |||||||||||
Selling, general and administrative | 44,585 | 15,319 | 117,088 | 51,435 | |||||||||||
Gain on lease modification | (8,301 | ) | — | (8,301 | ) | — | |||||||||
Total costs and operating expenses | 101,322 | 52,084 | 283,391 | 182,742 | |||||||||||
Loss from operations | (99,214 | ) | (52,084 | ) | (281,283 | ) | (182,742 | ) | |||||||
Other income (expense): | |||||||||||||||
Interest income, net | 3,275 | 2,850 | 14,697 | 8,618 | |||||||||||
Other income (expenses), net | (36 | ) | 33 | (180 | ) | (69 | ) | ||||||||
Total other income, net | 3,239 | 2,883 | 14,517 | 8,549 | |||||||||||
Net loss | $ | (95,975 | ) | $ | (49,201 | ) | $ | (266,766 | ) | $ | (174,193 | ) | |||
Basic and diluted net loss per common share | $ | (1.59 | ) | $ | (0.93 | ) | $ | (4.57 | ) | $ | (3.41 | ) | |||
Weighted-average number of shares used in computing basic and diluted net loss per common share | 60,352,124 | 52,972,225 | 58,321,612 | 51,150,728 |
Condensed Consolidated Balance Sheets | ||||||||
(In thousands) | ||||||||
Assets | (Unaudited) | |||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 302,237 | $ | 275,357 | ||||
Short-term marketable securities | 307,732 | 202,177 | ||||||
Other current assets | 18,028 | 8,246 | ||||||
Total current assets | 627,997 | 485,780 | ||||||
Property and equipment, net | 27,113 | 14,981 | ||||||
Long-term marketable securities | 85,030 | 114,281 | ||||||
Operating lease right-of-use assets | 52,775 | — | ||||||
Other assets | 3,184 | 2,601 | ||||||
Total assets | $ | 796,099 | $ | 617,643 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities | $ | 71,453 | $ | 33,773 | ||||
Long-term debt | 73,559 | — | ||||||
Operating lease liabilities, noncurrent | 72,359 | — | ||||||
Other noncurrent liabilities | 34 | 11,071 | ||||||
Total liabilities | 217,405 | 44,844 | ||||||
Total stockholders’ equity | 578,694 | 572,799 | ||||||
Total liabilities and stockholders’ equity | $ | 796,099 | $ | 617,643 | ||||
Contact Information:
650-410-3258
simmergut@gbt.com
650-741-7730
syao@gbt.com
Source: Global Blood Therapeutics, Inc.