Bioblast Pharma

Hope should not be rare

Pipeline updates archive

Pipeline Updates Archive

  • June 9, 2016
  • Bioblast Pharma
  • Pipeline updates

Bioblast Pharma Updates Corporate Plans

Trehalose Phase 2b Trial in Oculopharyngeal Muscular Dystrophy (OPMD) 
On Track to Commence During the Second Half of 2016 

Focus On Trehalose As a Potential Platform

Corporate Restructuring and Management Changes:
Fredric Price, Executive Chairman, will lead the
Company’s day-to-day operational efforts

NEW HAVEN, Conn., June 09, 2016 (GLOBE NEWSWIRE) -- Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, today 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.

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  • December 9, 2015
  • Bioblast Pharma
  • Pipeline updates

Bioblast Pharma Provides Research and Development Update

NEW HAVEN, Conn., Dec. 9, 2015 (GLOBE NEWSWIRE) — Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage biotechnology company developing meaningful therapies for patients with rare and ultra-rare genetic diseases, today provided an update on its research and development programs. Recently, the Company announced positive, open-label, interim HOPEMD Phase 2 clinical data for Cabaletta in oculopharyngeal muscular dystrophy (OPMD), in which proof-of-concept efficacy signals were observed in patients early in treatment. Based on the positive data from the HOPEMD study, the Company intends to conduct a double-blind, placebo-controlled, Phase 2b study to augment the existing data package for Cabaletta and to further guide the design of a future pivotal Phase 3 study.

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  • September 14, 2015
  • Bioblast Pharma
  • Pipeline updates

Bioblast Granted Patent From USPTO For Cabaletta For The Treatment Of Spinocerebellar Ataxia (SCA)

TEL AVIV, Israel, September 14, 2015 – Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it was granted a patent from the United States Patent and Trademark Office (patent no. 9,125,924) for Cabaletta (IV trehalose) entitled “Compositions and Methods for Treating Spinocerebellar Ataxia”.

Cabaletta is in Phase 2 clinical development for the treatment of Spinocerebellar Ataxia Type 3 (also known as SCA3 or Machado Joseph disease). The Company anticipates that it will start pivotal Phase 3 studies subject to ongoing regulatory discussions in each of Spinocerebellar Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy in 2015.

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