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Syros Pharma Teams Up with Global Blood Therapeutics to Fight Sickle Cell Disease


Syros Pharmaceuticals' shares rose 20% today after the firm reported that it entered into a collaboration agreement with Global Blood Therapeutics for development of therapies for sickle cell disease and beta thalassemia that could result in payments to Syros as high as $315 million over three years.

This morning, gene control platform and biopharmaceutical development firm Syros Pharmaceuticals Inc. (SYRS:NASDAQ) and clinical-stage biopharmaceutical company Global Blood Therapeutics Inc. (GBT:NASDAQ) announced that they have entered into a collaboration to discover, develop and commercialize novel therapies for sickle cell disease (SCD) and beta thalassemia.

The firms stated that the collaboration will combine GBT's therapeutic area leadership with the power of Syros' leading gene control platform. Syros and GBT indicated that the agreement was established in order to find new medicines to identify therapeutic targets and discover drugs to induce the production of fetal hemoglobin, which they advised is known to exert protective effects on the red blood cells of patients with SCD and beta thalassemia and mitigate the clinical manifestation of these diseases.

According to the agreement terms, GBT will be required to pay $20 million upfront to Syros and fund up to $40 million in preclinical research for at least three years. Under the terms of the agreement, GBT will receive an option to obtain an exclusive worldwide license to develop, manufacture and commercialize products resulting from the collaboration. The companies mentioned that if GBT were to exercise its exclusive worldwide license option, Syros could receive a total of up to $315 million under that scenario, if all the other conditions and milestones of the development and commercialization agreement are met.

Nancy Simonian, M.D., CEO of Syros Pharmaceuticals, commented, "We believe it is possible to provide a functional cure for patients with sickle cell disease or beta thalassemia by switching on the gamma globin gene with an oral medicine...Partnering with GBT, an established leader in sickle cell disease with proven research, development, manufacturing and commercialization capabilities, allows us to expand and accelerate our program, exploring multiple approaches in parallel with the aim of bringing much-needed new therapies to market for patients with sickle cell disease and beta thalassemia as quickly as possible."

Global Blood Therapeutics' President and CEO Ted W. Love, M.D., commented, "The discovery and development of novel therapeutic approaches to treat sickle cell disease has been a driving force for GBT since we were founded...We believe that Syros' approach to inducing fetal hemoglobin is one of the most promising ways to identify the next generation of therapies to treat sickle cell disease and beta thalassemia at a fundamental level upstream of serious complications such as organ damage, organ failure and early death. We will continue to seek the best scientific approaches to transform the treatment of these devastating lifelong diseases."

Syros Pharmaceuticals is headquartered in Cambridge, Mass., and states that it is "redefining the power of small molecules to control the expression of genes" and that by using the firm's unique abilities it aims to develop medicines that provide a profound benefit for patients with diseases that have previously eluded other genomics-based approaches. The company's pipeline includes SY-1425 currently in a Phase 2 trial for treatment in a genomically defined subset of acute myeloid leukemia patients, and SY-5609, a highly selective and potent oral CDK7 inhibitor in investigational new drug application-enabling studies in cancer. The firm indicated that it also has several other preclinical and discovery programs in oncology and monogenic diseases, including sickle cell disease.

Global Blood Therapeutics has a market cap of approximately $4.7 billion and is based in South San Francisco, Calif. The company describes itself as a clinical-stage biopharmaceutical company "determined to discover, develop and deliver innovative and life-changing treatments that provide hope to underserved patient communities." The firm is developing two therapies for the potential treatment of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder, including its late-stage oral, once-daily product candidate Oxbryta (voxelotor). The firm is also advancing its inclacumab drug, a p-selectin inhibitor in development to address pain crises associated with the disease.

Syros Pharmaceuticals started the day with a market cap of $196.1 million with 42.44 million shares outstanding and a short interest of around 4.4%. SYRS shares opened higher today at $4.88 (+$0.26, +5.63%) compared to yesterday's $4.62 closing price. The stock has traded between $4.82 and $5.97 per share today and presently is trading at $5.56 (+$0.94, +20.35%).


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