Positive Topline Results Available on TPIP for PAH

During clinical development, treatments are sometimes first evaluated in healthy volunteers. This is the route that Insmed Incorporated (“Insmed”) took with treprostinil palmitil inhalation powder (TPIP). Now, following positive topline results, Insmed is looking forward to moving forward the TPIP clinical development program for patients with pulmonary arterial hypertension (PAH). Interested in hearing the Insmed conference call discussing these results? Listen here.

Treprostinil Palmitil Inhalation Powder (TPIP)

TPIP is a dry powder which was fully developed by Insmed. Altogether, it is a treprostinil palmitil formulation. The treatment is administered via an inhalation device. While study data showed the treatment’s safety and efficacy, TPIP is an investigational product and thus not yet approved for use.

During the Phase 1 clinical trial, healthy volunteers received either a single dose daily or multiple doses over a 7-day period. In the single dose cohort, participants received either 112.5 µg, 225 µg, or 450 µg. Later, participants received either 675 µg or a placebo. Later, in the multiple dose cohorts, patients received either 225 µg once daily vs. a placebo, or 112.5 µg for 4 days following by 225 µg for 3 days.

Altogether, the drug was safe and well-tolerated, though some side effects did occur. The most common included nausea, headache and dizziness, and a cough. No adverse reactions were severe or warranted additional treatment. TPIP has a longer half-life than available treatment options, potentially offering a more sustained response.

Moving forward, Insmed hopes to run multiple studies to evaluate TPIP for patients with PAH. Outside of PAH, TPIP is being explored as a potential treatment for idiopathic pulmonary fibrosis (IPF) and interstitial lung disease.

Pulmonary Arterial Hypertension (PAH)

In many cases, BMPR2 gene mutations cause pulmonary arterial hypertension (PAH), a progressive and chronic form of high blood pressure. However, HIV infection, sickle cell disease, liver disease, and other conditions may also cause PAH. In around 15-20% of cases, PAH is inherited. No matter how someone develops PAH, the condition causes the progressive thickening or hardening of pulmonary arteries. When these arteries become thickened, narrowed, or blocked, the heart must work harder to force blood through and receive oxygen. This causes the heart to weaken and less oxygenated blood to reach the rest of the body. Females are 2x more likely to have PAH than males. This condition usually affects those between ages 30 and 60. Symptoms include:

  • Chest pain and/or pressure
  • Fatigue
  • Dizziness
  • Swelling in the lower extremities
  • Shortness of breath that worsens during exertion
  • Heart palpitations
  • Cyanosis (bluish tint to the lips and skin)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email