54.227.97.219
dgid:
enl:
npi:0
-Advertisement-
-Advertisement-
AASLD
Autoimmune
Conference Roundup
SPLIT

Part 1 Results from PIONEER Trial in Indolent Systemic Mastocytosis Announced

Posted on March 16, 2020

Blueprint Medicines, a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced updated results from the Phase 2 PIONEER trial of avapritinib in patients with indolent systemic mastocytosis (SM) showing significant clinical improvements versus placebo.

In Part 1 of the PIONEER trial, patients treated with avapritinib showed a statistically significant mean decline of approximately 30 percent in total symptom score (TSS) at 16 weeks, as measured by the Indolent SM Symptom Assessment Form (ISM-SAF), and reductions in symptom scores have deepened over time. In addition, patients treated with avapritinib achieved consistent improvements across objective measures of mast cell burden and patient-reported quality of life. Avapritinib was well-tolerated with no patients discontinuing treatment due to adverse events (AEs). Based on the full Part 1 data, 25 mg once daily (QD) has been selected as the recommended Part 2 dose (RP2D).

Results from this data presentation will be available on an American Academy of Allergy, Asthma & Immunology (AAAAI) virtual forum, which was established following the cancellation of the 2020 AAAAI Annual Meeting.

Read the full press release here

Source: Blueprint Medicines Corporation
-Advertisement-
Related Articles
FDA Grants DSG3-CAART Orphan Drug Designation for the Treatment of Pemphigus Vulgaris
May 31, 2021
Data from Trial Evaluating Eculizumab for the Treatment of Neuromyelitis Optica Spectrum Disorder to be Presented at AAN Annual Meeting
May 31, 2020
AAAI Cancels Annual Meeting
Mar 14, 2020
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-
-Advertisement-