Global Blood Therapeutics Inc GBT, a clinical-stage biopharmaceutical company that focuses on treating blood-based disorders, released results from multiple data presentations during the 59th American Society of Hematology Annual Meeting & Exposition.
The Analyst
Morgan Stanley's Matthew Harrison maintains an Overweight on Global Blood's stock with an unchanged $55 price target.
The Thesis
Global Blood's interim data for its Phase 2a trial of the sickle cell disease therapy GBT440 in adolescents was consistent with prior adult results and demonstrates efficacy on top of hydroxyurea and utility of the PRO, Harrison said in a Sunday note. (See Harrison's track record here.)
Among the 11 patients with available efficacy data through 16 weeks, six of them achieved increased hemoglobin of greater than 1 g/dL and the median hemoglobin change was 1.1 g/dL, according to Morgan Stanley. There was also a reduction in daily symptoms among 10 of the 12 patients as measured by the total symptom score, or TSS. In fact, five of the 12 patients saw their TSS decline to zero as measured by the PRO.
After meeting with the company's management team, Harrison said the executives were "clearly encouraged" by the data. In addition, the company plans to update investors on the validation of the PRO and provide a quantitative measure of clinical utility based on the PRO.
"Investors seemed focused on the potential correlation of the PRO with hemoglobin response, but management cautioned that in such a small dataset any views were just anecdotal," the analyst said. "Investors also focused on other factors which could influence the PRO, such as pain medication. Management suggested randomization would mitigate this concern."
Price Action
Shares of Global Blood were trading lower by 10.28 percent at $41 Monday morning.
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Photo by Dr. Graham Beards/Wikimedia.
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