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SOUTH SAN FRANCISCO, Calif., Aug. 23, 2018 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) announced today that it has entered into an exclusive worldwide licensing agreement with F. Hoffmann-La Roche Ltd. for the development and commercialization of inclacumab, a novel fully human monoclonal antibody against P-selectin. GBT plans to develop inclacumab as a treatment for vaso-occlusive crises (VOC) in patients with sickle cell disease (SCD).
Under the terms of the agreement, GBT will be responsible for all development, manufacturing, and commercialization of inclacumab worldwide. Roche will receive an upfront payment of $2.0 million from GBT and is eligible to receive up to approximately $125 million in development and commercialization milestone payments for the sickle cell disease indication. Additionally, Roche is eligible to receive tiered royalties based on net revenues for inclacumab.
“We have been working diligently to diversify our product pipeline through both internal research and external business development efforts and are excited to have entered into this agreement for inclacumab,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We are committed to developing transformative treatments for the SCD community and becoming the preeminent SCD company. Inclacumab is an ideal complement to voxelotor, our lead investigational oral, once-daily therapy, in Phase 3 clinical development for SCD. Like voxelotor, inclacumab has a strong scientific rationale and has the potential to provide significant clinical benefit for SCD patients.”
Roche was previously developing inclacumab for patients with coronary artery disease. The pharmacokinetic, safety, and tolerability profile of inclacumab are well characterized based upon Roche’s prior clinical studies, which enrolled more than 500 patients. Roche discontinued the inclacumab program following Phase 2 clinical trials.
P-selectin inhibition is a clinically validated target in SCD, known to reduce the incidence of VOCs. GBT plans to develop inclacumab for this indication and intends to leverage the safety data produced from Roche’s prior clinical studies as we proceed with our development in SCD. GBT has already begun the process of technology transfer from Roche to a contract manufacturing organization, and anticipates submitting an Investigational New Drug application to the U.S. Food and Drug Administration for inclacumab in 2021.
Inclacumab is a novel, fully human monoclonal antibody designed to bind to and selectively inhibit P-selectin, an adhesion molecule found on endothelial cells and platelets that contributes to the cell-cell interactions that are involved in the pathogenesis of VOC.
About Sickle Cell Disease
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). In its deoxygenated state, HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which causes hemolytic anemia (the destruction of RBCs) that can eventually lead to multi-organ damage and early death. This sickling process also causes blockage in capillaries and small blood vessels, and adhesion molecules, such as P-selectin play an important role in this process. Beginning in childhood, SCD patients suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities.
About Vaso-Occlusive Crisis
Vaso-occlusive crisis (VOC) is the most common clinical manifestation of SCD. It occurs when the microcirculation is obstructed by sickled RBCs, causing ischemic injury to the organ supplied and resultant pain. Approximately half of individuals with SCD experience VOC. Pain crises begin suddenly and may last several hours to several days. The pain can affect any body part but often involves the abdomen, bones, joints and soft tissue.1 VOC result in a decrease in quality of life2 and an increase in the risk of death.3
GBT is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. GBT is developing its lead product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
GBT Forward-Looking Statements
Statements we make 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. We intend these forward-looking statements, including statements regarding the therapeutic potential and safety profile of inclacumab, the therapeutic potential and safety profile of voxelotor, our plans to develop inclacumab as a treatment for VOCs in patients with SCD and leverage the safety data from Roche’s prior clinical studies, the potential upfront, milestone and royalty payments, our commitment to developing transformative treatments for the SCD community and becoming the preeminent SCD company, our belief that inclacumab is an ideal complement to voxelotor, our anticipated submission of an IND to the FDA for inclacumab in 2021, our ability to implement and complete our clinical development plans for voxelotor and inclacumab, our ability to generate and report data from our ongoing and potential future studies of voxelotor and inclacumab, regulatory review and actions relating to voxelotor and inclacumab, and the timing of these events, 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 are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We 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 our control including, without limitation, the risks that our clinical and preclinical development activities may be delayed or terminated for a variety of reasons, that results of clinical trials may be subject to differing interpretations, that regulatory authorities may disagree with our clinical development plans or require additional studies or data to support further clinical investigation of our product candidates, that drug-related adverse events may be observed in clinical development, and that 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 our Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and our Quarterly Report on Form 10-Q for the quarter ended June 30, 2018, as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Myesha Lacy (investors)
Julie Normart (media)
W2O pure for GBT
1 https://emedicine.medscape.com/article/205926-clinical . Accessed June 5, 2018.
2 van Tuijn CF, van Beers EJ, Schnog JJ, Biemond BJ. Pain rate and social circumstances rather than cumulative organ damage determine the quality of life in adults with sickle cell disease. Am J Hematol. 2010;85:532-535
3 Platt OS, Brambilla DJ, Rosse WF, et al. Mortality in sickle cell disease — life expectancy and risk factors for early death. N Engl J Med .1994;330:1639-1644.