Research programme: proteostasis regulators - Proteostasis Therapeutics

Drug Profile

Research programme: proteostasis regulators - Proteostasis Therapeutics

Alternative Names: PTI 801 - Proteostasis Therapeutics; PTI 808; PTI NC 733; PTI-130; PTI-C1811; PTI-P271; PTI801

Latest Information Update: 21 Oct 2016

Price : $50

At a glance

  • Originator Proteostasis Therapeutics
  • Developer Biogen; Cystic Fibrosis Foundation Therapeutics; Harvard Medical School; Proteostasis Therapeutics
  • Class Small molecules
  • Mechanism of Action Alpha-synuclein inhibitors; Cystic fibrosis transmembrane conductance regulator stimulants; Protein folding modulators; Protein modulators; USP14 protein inhibitors
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Chronic obstructive pulmonary disease; Cystic fibrosis; Neurodegenerative disorders
  • Research Inflammation; Metabolic disorders; Parkinson's disease

Most Recent Events

  • 11 Aug 2016 Proteostasis Therapeutics announces intention to submit IND applications for PTI 801 and PTI 808 to US FDA in December 2016
  • 27 Jul 2016 Preclinical trials in Cystic fibrosis in USA (PO)
  • 13 May 2016 Preclinical trials in Chronic obstructive pulmonary disease in USA (PO)
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