- For the patients who continued on trehalose treatment (the blue line in the following chart), the cold water drinking test time remained relatively stable across the treatment period
- For the patients who were not on treatment (the red line in the following chart), the cold water drinking test time worsened
Warren Wasiewski, MD, Bioblast's Chief Medical Officer said, "We recognize that these results are in a small number of patients and that while there were treatment and non-treatment arms, this was not a double-blind, placebo-controlled study. Nevertheless, we are encouraged by the fact that the improvement found first in the HOPEMD study is sustainable over an extended period of time and that the difference between patients who continued on treatment and those who were randomized to be withdrawn from treatment in this extension study has provided us with valuable information for our upcoming Phase 2b double-blind, placebo-controlled trial, which is planned to enroll more than 70 patients with OPMD."Based on the results in the Extension study (as well as from the earlier HOPEMD study), the primary efficacy measures in the Phase 2b trial will be the cold water drinking test and dysphagia related quality of life questionnaires (SWAL-QOL and Sydney Swallowing Questionnaire). Other tests, including those related to muscle function and power and weight, will be evaluated as secondary endpoints. More information from the Extension trial There were three main objectives of this study:
- To determine the long-term effect of trehalose on disease progression as assessed by the changes in the disease markers as well as in the patients' swallowing quality of life.
- To assess which efficacy measures were appropriate for consideration as endpoints in the upcoming Phase 2b trial. The efficacy measures included change from baseline in cold water drinking test time, video fluoroscopy, the total score and total symptom score on the patient reported swallowing quality of life questionnaire (SWAL-QOL), functional muscle testing, muscle strength testing by handheld dynamometer, histologic changes on percutaneous muscle biopsy, and weight.
- To assess safety parameters in the upcoming Phase 2b trial, including the frequency, severity, and duration of adverse events (AEs), as well as clinically significant laboratory abnormalities after administration of trehalose. The results from the Extension study indicated that:
- Trehalose was generally safe and well tolerated.
- There were no clinically significant changes in safety labs.
- There was one serious adverse event (SAE) unrelated to drug treatment.
- There were no infusion reactions or adverse events leading to discontinuation.
About Bioblast Pharma Ltd.Bioblast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases with a lead drug candidate, trehalose 90mg/mL solution, in Phase 2 development. Bioblast was founded in 2012 and is traded on the NASDAQ under the symbol "ORPN". For more information, please visit our website, www.bioblastpharma.com, the content of which is not incorporated herein by reference. Forward Looking Statements This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward looking statements when we discuss our plans to conduct a Phase 2b clinical study, and the design and timing of such study. In addition, historic results of scientific research and clinical and preclinical trials do not guarantee that the conclusions of future research or trials would not suggest different conclusions or those historic results referred to in this press release would not be interpreted differently in light of additional research and clinical and preclinical trial results. Because such statements deal with future events and are based on Bioblast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Bioblast Pharma could differ materially from those described in or implied by the statements in this press release, including those discussed under the heading "Risk Factors" in Bioblast Pharma's Annual Report on Form 20-F filed with the Securities and Exchange Commission ("SEC") on March 29, 2016, and in any subsequent filings with the SEC. Except as otherwise required by law, Bioblast Pharma disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.
INVESTOR CONTACT: Matthew P. DuffyManaging DirectorLifeSci Advisors, LLCTelephone: 212-915-0685