Proteostasis’ PTI-428 Receives FDA’s Breakthrough Therapy Designation for Cystic Fibrosis (CF)

 Proteostasis’ PTI-428 Receives FDA’s Breakthrough Therapy Designation for Cystic Fibrosis (CF)

Proteostasis’ PTI-428 Receives FDA’s Breakthrough Therapy Designation for Cystic Fibrosis (CF)

Shots:

  • The FDA’s BT designation is based on P-II study results assessing PTI-428 (50 mg, qd) vs PBO in 24 CF with F508del mutation on background treatment with Orkambi for 28 days
  • The P-II study results: improvement in forced expiratory volume in 1 second (ppFEV1) 5.2%; increase in nasal mucosal CFTR protein
  • Proteostasis’ PTI-428 is a part of triple combination therapy including PTI-808 (potentiator) & PTI-801 (corrector) involved in increasing the level of CFTR protein to treat CF patients who are homozygous for the F508del mutation in the CFTR gene

Click here to read full press release/ article | Ref: Globe NewsWire | Image: Contract Pharma

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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