Express . . . Treatment of rare immune diseases, precision anti-cancer therapy Phase 2 data positive.

▎ today, blueprint medicine announced that its oral kit and pdapritinib, an oral inhibitor of PDGFR α, have significantly improved the comprehensive symptoms of patients with systemic mastocytosis (SM) in the phase II clinical trial of Pioner in the treatment of systemic mastocytosis (SM).SM is a rare disease caused by uncontrolled proliferation and activation of mast cells.it has several forms, including inert SM, invasive SM and advanced SM.mast cells are immune cells that produce histamine and other factors that mediate inflammation and allergic reactions.in patients with SM, the release of these factors at high levels can lead to different degrees of symptoms, including pain, nausea, rash, fever, fatigue, etc.in severe cases, multiple organ systems of SM patients will appear debilitating symptoms, which may be life-threatening.the D816V mutation of kit protein is the main driving factor of almost all SM diseases.at present, there is no approved therapy targeting this mutation.avaparinib is a type 1 inhibitor targeting kinase activated conformation, which can inhibit a variety of protein kinases carrying kit and PDGFRA gene mutations.blueprint medicines initially developed avaparinib for the treatment of advanced gastrointestinal stromal tumors (GIST) and advanced SM.in June 2017, avaparinib was approved by FDA as a breakthrough therapy for patients with unresectable or metastatic GIST carrying PDGFR α d842v mutation.previously, the FDA also granted avapritinib orphan drug qualification for the treatment of gist and mastocytosis.cornerstone pharmaceutical has entered into a partnership with blueprint medicine to acquire the right to develop avapritinib in Greater China.} compared with placebo, avaparinib significantly improved the ism-saf total symptom score (image source: reference [2]). Pianeer is a randomized, double-blind, phase 2 clinical trial with a placebo-controlled group to evaluate the efficacy and safety of avaparinib in the treatment of patients with indolent SM.the trial consists of three stages: dose determination phase, registration trial phase and long-term treatment phase.the data from this trial showed that after 16 weeks of treatment, the total symptom score of ism-saf in the treatment group decreased by about 30%, while that in the placebo group decreased by only 3%. The levels of serum tryptase, bone marrow mast cells and kit D816V allele load were also significantly decreased in the25 mg dose group. in addition, avaparinib treatment resulted in a sustained decrease in ism-saf skin, gastrointestinal, and nervous system symptoms by 37%, 25%, and 26%, respectively. the quality of life score improved by 34% on average in patients with placebo, which was 7% higher than that at baseline. "avaparinib is specifically designed to inhibit the D816V mutant of kit to provide transformative clinical benefits to patients," said Andy Boral, MD, chief medical officer, blueprint medicine. "These placebo-controlled data are the first to show consistent clinical improvements in clinical outcomes and multiple disease indicators such as quality of life. these results give us the confidence to treat the root cause of the disease and bring more clinical benefits to patients with SM. "References: [1] blueprint medicines announcements Part 1 results from pioneer trial showing broad activity of avaprinib across measures of mast cell burden, clinical outcomes and quality of life in indolent systemic mastocytosis, retrieved March 16, 2020, from blueprint medicines aaaaaI conference call, retrieved March 16, Note: This article aims to introduce the progress of medical and health research, not to recommend treatment options. if you need guidance on treatment plan, please go to a regular hospital.