Roth Capital Affirms Bioblast Pharma (ORPN) at 'Buy'; Sees Trehalose PK Data as 'Meaningful'
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Roth Capital reaffirms its Buy rating and $15 price target on Bioblast Pharma Ltd. (Nasdaq: ORPN) after the company announced results of a double-blind, placebo-controlled pharmacokinetic (PK) study of intravenous (IV) trehalose 90mg/mL in healthy volunteers.
Analyst Joseph Pantginis commented today,
We believe these data are important and meaningful as it establishes a MTD that is twice the dosage of trehalose used in the HOPEMD and Extension studies which suggested that trehalose had a positive effect on dysphagia and that discontinuation of the therapy results in an increase in swallowing difficulty. A Phase IIb study is planned to start in 1Q17 with the goal of having the cold water drinking test and dysphagia related quality of life questionnaires as the primary endpoints. Muscle function, power and weight assessments will be included in the secondary endpoints.
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