Patients were randomized to receive 100 mg PTI-428 or placebo for seven days in addition to their background Orkambi therapy.
Preliminary data confirmed the PTI-428 PK profile to be comparable to that achieved in healthy volunteers and showed a lack of impact on Orkambi exposure levels.
With advances in medication and management, many patients with CF live well into their 30s, 40s and sometimes beyond.
Proteostasis Therapeutics (NYSE: PTI) announces preliminary data from the Multiple Ascending Dose cohort of its Phase 1 trial designed to evaluate the safety and pharmacokinetics of PTI-428 in cystic fibrosis (CF) subjects.
People with this mutation represent the largest population of those with CF, a rare, life-threatening disease.
But Ashley did stabilize and when she was taken off sedation one of the first people she saw was Ryan. A few hours later another life-changing event happened.
Her family and fiancé were told Ashley was a match for a pair of donor lungs, and she was finally going to get her transplant after being listed for more than two years.
Her boyfriend, Ryan Denkenberger, 20 — the proverbial "friend of a friend" she met on Facebook — was at her side, watching a ventilator do her breathing as she lay on the bed, sedated. It was nothing fancy, he says, just dinner and a movie. a rock, whose mother raised him right.” He would help her carry her oxygen, and when she couldn’t make it up to the second-story of her home, Ryan would put her on his back and climb the stairs piggyback style.
Ryan was a young man in love, but he didn’t know if Ashley was going to survive. “I wanted her to know just how much I loved her, no matter what,” says Ryan, who works in the energy industry. “I thought it couldn’t get any better than that.” But it could, and did.