Windtree Therapeutics Updates on Istaroxime Development and Upcoming Trial Data

Windtree Therapeutics, Inc. (NasdaqCM: WINT), a biotechnology firm advancing therapies for critical conditions, has provided updates on the clinical and business development of its innovative therapy, istaroxime. This first-in-class therapy enhances both systolic and diastolic heart functions and has shown promise in improving blood pressure and renal function without increasing cardiac arrhythmias. The drug has demonstrated positive outcomes in three Phase 2 trials with patients suffering from acute heart failure (AHF) and early cardiogenic shock.

Windtree is nearing the completion of enrollment for its Phase 2 SEISMiC Extension Study, which targets patients in SCAI Stage B early cardiogenic shock. This Extension Study, involving up to 30 patients, aims to optimize dosing and further characterize the effects of SERCA2a, an important aspect for planning Phase 3 trials. It builds on the original SEISMiC study by extending the dosing duration from 24 to 60 hours. Patients will receive one of two istaroxime dosing regimens or a placebo. The study seeks to determine the most effective dosing regimen and collect detailed heart function data.

Parallelly, Windtree is initiating a study for the more severe SCAI Stage C cardiogenic shock patients. This phase will review data from up to 20 patients in a double-blinded, placebo-controlled environment where istaroxime will be added to the current standard care involving inotropes or vasopressors. Key measurements will include systolic blood pressure changes, progression to more severe stages of shock, and overall patient outcomes, such as length of hospital stay and survival rates.

In addition, Windtree announced a licensing agreement with Lee’s Pharmaceutical (HK) Limited in January 2024. This agreement grants Lee’s the rights to develop and commercialize istaroxime in Greater China. The deal also includes Windtree’s preclinical SERCA2a activators and rostafuroxin, a candidate for treating hypertension. The agreement could potentially yield Windtree up to $138 million in future milestones, plus additional royalties, with Lee’s bearing all development and commercialization costs in the licensed regions. Windtree plans to provide an update on Lee’s progress later in the year.

Craig Fraser, CEO and Chairman of Windtree, expressed satisfaction with the progress and emphasized the critical unmet needs in the patient populations being studied. He highlighted the advantageous partnership with Lee’s, which allows Windtree to leverage additional resources for its global cardiogenic shock program.

Istaroxime works through a dual mechanism: it increases myocardial contractility by inhibiting Na+/K+- ATPase and facilitates myocardial relaxation through the activation of the SERCA2a calcium pump. This dual action contributes to its effectiveness in improving cardiac function and blood pressure without increasing heart rate or causing arrhythmias, as demonstrated in multiple Phase 2 studies.

Windtree Therapeutics continues to advance its pipeline of innovative therapies for critical diseases, focusing on both early and late-stage products. In addition to istaroxime, their portfolio includes preclinical SERCA2a activators and precision aPKCi inhibitors for oncology applications. The company also pursues a licensing business model, with existing out-licenses supporting its clinical development efforts.

Craig Fraser concluded by noting the company's eagerness to report the topline results of the SEISMiC Extension Study and, with adequate resources, to continue advancing the Phase 3 readiness and the SCAI Stage C study.