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Bio Blast Pharma (ORPN)

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  1. I Love Melbourne 19 juni 2016 13:18
    Bioblast Pharma Ltd. (Nasdaq: ORPN) announced that it plans to initiate a Phase 2b trial with trehalose IV solution in OPMD during the second half of 2016, and has commenced a scientific and business assessment to determine the suitability of using trehalose IV solution in other orphan diseases.

    The company intends to enroll more than 70 patients in the Phase 2b clinical trial at up to 15 sites in the United States and Canada. This double-blind, placebo-controlled trial follows from the HOPEMD open label Phase 2a study in which trehalose IV solution was observed to be safe and well tolerated, with no drug-related serious adverse events. Additionally, improvements versus baseline were observed in multiple efficacy endpoints related to dysphagia and muscle strength and function.

    “Given the interest in the scientific and medical community with regard to alternative therapeutic uses of trehalose, we have decided to concentrate our internal development efforts exclusively on this drug candidate, which will enable us to leverage our extensive knowledge and expertise from our R&D and manufacturing efforts,” stated Fredric Price, Bioblast’s Executive Chairman. “In the event that we determine to investigate other diseases for which trehalose IV solution may provide positive disease modifying outcomes, we anticipate that we will be able to enter into additional Phase 2 programs rapidly and with little or no incremental preclinical expenses. We anticipate providing shareholders with more information on this expanded trehalose IV solution development program later this year. With this change in mind, we have chosen to postpone work on the mitochondrial replacement and read-through platforms."

    “Consequently, with our focus on trehalose IV solution, we have made the decision to streamline company expenditures and downsize the management team, with the result being, among other things, that up to 8 employees have left or will be leaving Bioblast, including the Chief Commercial Officer, Chief Corporate Development Officer, and Chief Financial Officer. Additionally, Colin Foster, Bioblast’s President and CEO, will be leaving his operational position but will continue as a member of the Board of Directors,” continued Mr. Price. “I will remain in my role as Executive Chairman and will lead the Company’s day-to-day operational efforts starting July 1, 2016. Commitments for the payment of severance and related benefits in connection with the personnel adjustments of approximately $1.7 million are expected to be substantially paid out during the third quarter of 2016. The savings from this streamlining are expected to be realized beginning in 2017.”

    Bioblast ended the first quarter of 2016 with $21.6 million in cash and equivalents.

    “I would like to express my gratitude to those members of our Bioblast team affected by this downsizing,” said Mr. Foster. “These employees have worked with dedication and passion and we are grateful and proud of the work they have done. I am confident that the core remaining operational team, under Fred’s leadership has the skills necessary to drive the trehalose programs forward.”

    “I am gratified that Colin will remain on the Board of Directors and want to thank him for his extraordinary service to the Company in an operational capacity,” said Mr. Price. “His advice and counsel are invaluable to the Board and me, and I look forward to working with him closely as we move the Company forward.”

    www.streetinsider.com/Corporate+News/... (9-6-2016)
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