Bioblast Pharma

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Trial-related news archive

Trial-related News Archive

  • June 22, 2015
  • Bioblast Pharma
  • Trial-related news

Bioblast Announces Positive Preclinical Proof-Of-Concept Results Of Its Novel Read- through Platform in Spinal Muscular Atrophy (SMA)

TEL AVIV, Israel, June 22, 2015 – Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today positive preclinical in vitro and in vivo proof-of-concept study results for its Read-through drug candidate (BBrm02) for Spinal Muscular Atrophy (SMA). BBrm02 is a proprietary, intrathecal formulation of azithromycin (19th International SMA Researcher Meeting, Kansas City, MO).

In a series of cell and mouse studies BBrm02 was effective in creating high levels of full length SMN2 protein (Survival Motor Neuron 2) in the central nervous system resulting in a high degree of cellular and disease phenotypic rescues. Significant levels of full length SMN2 protein were detected following treatment with BBrm02 in several different patient cell lines representing different stages of disease severity. This SMN2 protein was tested for biological activity and was found to be highly functional.

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  • June 18, 2015
  • Bioblast Pharma
  • Trial-related news

Bioblast Pharma to Present Preclinical Proofs of Concept in Spinal Muscular Atrophy and Friedreich’s Ataxia at Two Leading Scientific Meetings in June

TEL AVIV, Israel, June 18, 2015 (GLOBE NEWSWIRE) — Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it will present pre-clinical proofs of concept for two of its therapeutic platforms during June.

At the 19th International SMA Researcher Meeting in Kansas City, MO (see details below), Bioblast will present its proof of concept for BBrm02 (intrathecally-administered azithromycin) for Spinal Muscular Atrophy, its lead drug candidate in its Read-through Platform.

At the United Mitochondrial Disease Foundation – Mitochondrial Medicine 2015 Meeting, in Herndon, VA (see details below), Bioblast will present its proof of concept for BB-FA (frataxin protein replacement) for Friedreich’s Ataxia, its lead drug candidate in its Mitochondrial Protein Replacement Platform (mPRT).

Details of the two meetings:

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  • June 15, 2015
  • Bioblast Pharma
  • Trial-related news

Bioblast Announces The Start Of A Phase 3 Study For Cabaletta In Oculopharyngeal Muscular Dystrophy (OPMD)

New Pivotal Study Reflects Positive Signals Reported From The Current HOPEMD Study,Which Will Be Closed Out At 25 patients

TEL AVIV, Israel, June 15, 2015– Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today the start of a Phase 3 pivotal study in the USA and Canada for Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy (OPMD).

Colin Foster, Bioblast’s President and CEO said, “We see positive signals in a consequential number of the 25 treated patients currently enrolled in our open label HOPEMD study, with respect to various muscle and other performance tests. These positive signals lead us to believe that the clinical study duration required to demonstrate efficacy may be considerably shorter than we initially predicted.

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