Quince Therapeutics Announces Phase 2a Data for LAM-001
Quince Therapeutics announced Phase 2a data evaluating LAM-001, an inhaled formulation of rapamycin, in patients with pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease, presented at the American Thoracic Society conference in Orlando. The company recently acquired LAM-001 through its previously announced acquisition of Orphai Therapeutics, Inc., a clinical-stage biotechnology company developing LAM-001 to treat rare pulmonary diseases. The Phase 2a study was a 24-week, open-label trial conducted across four clinical sites evaluating LAM-001 as an add-on therapy to standard of care in 10 adult patients with PAH and PH-ILD who remained symptomatic despite background therapy. Primary endpoints included change from baseline in peak oxygen uptake at Week 24, safety and tolerability. Secondary endpoints included pulmonary vascular resistance, six-minute walk distance and change in functional class, with change in N-terminal pro-B-type natriuretic peptide assessed as an exploratory endpoint. Treatment with LAM-001 was associated with improvement or stabilization from baseline across multiple clinically relevant measures, including 6MWD, PVR, NT-proBNP and forced vital capacity, supporting potential benefit across exercise capacity, pulmonary hemodynamics, cardiac stress and lung function. Improvements in 6MWD, a clinically meaningful endpoint commonly used in pulmonary hypertension clinical trials and regulatory submissions, were observed alongside favorable changes in hemodynamic and biomarker measures. All evaluable patients also transitioned from Functional Class III to Functional Class II by Week 24. In addition, two patients transitioned to Functional Class I at 16- and 52-week evaluations. LAM-001 was generally well tolerated in patients receiving background standard-of-care therapy. A total of 6 patients were evaluable at the 24-week endpoint, of which 4 were PH-ILD patients. All 4 PH-ILD patients were receiving stable doses of treprostinil therapy before and throughout the study.
