Bioblast Pharma

Hope should not be rare

Pipeline updates archive

Pipeline Updates Archive

  • June 25, 2015
  • Bioblast Pharma
  • Pipeline updates

Bioblast Pharma Receives Orphan Drug Designation From the European Commission for Cabaletta for the Treatment of Oculopharyngeal Muscular Dystrophy

TEL AVIV, Israel, June 25, 2015 (GLOBE NEWSWIRE) — Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage orphan disease-focused biotechnology company, announced today that it has been granted Orphan Drug Designation by the European Commission for Cabaletta for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD).

Bioblast is starting a double-blind, placebo-controlled Phase 3 pivotal study across multiple clinical centers in the US and Canada in OPMD. The company recently announced positive clinical signals observed in its ongoing HOPEMD study.

Cabaletta is a chemical chaperone that has been shown to prevent pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation, including cell and animal models of Spinocerebellar Ataxia and other PolyA/PolyQ diseases, in addition to OPMD.

Continue reading

  • April 9, 2015
  • Bioblast Pharma
  • Pipeline updates

Bioblast Pharma Announces Granting of Fast Track Designation by the FDA for Cabaletta in Oculopharyngeal Muscular Dystrophy (OPMD)

Bioblast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company announced today that the United States Food and Drug Administration (FDA) has granted Fast Track designation to Cabaletta for the treatment of patients with Oculopharyngeal Muscular Dystrophy (OPMD). 

Cabaletta is being developed to treat OPMD, a rare and debilitating muscular dystrophy for which there is no cure or approved therapy. Bioblast is currently conducting a Phase 2/3 clinical study for OPMD and recently announced the granting of an IND, enabling the opening of a US clinical center in addition to the two ongoing clinical sites in Israel and Canada.

Continue reading

  • February 24, 2015
  • Bioblast Pharma
  • Pipeline updates

Bioblast Receives Notice of Allowance From USPTO Covering Cabaletta for the Treatment of OPMD

Bioblast Pharma Ltd (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition and method for the treatment of oculopharyngeal muscular dystrophy (OPMD) with Cabaletta. The Company has demonstrated efficacy in preclinical cell and animal models with Cabaletta, its lead therapeutic program and it is currently the subject of human clinical trials pursuing two indications.

Continue reading

Pages