Windtree Therapeutics (WINT) Emerging Growth Conference77 summary

Lead drug candidate and mechanism of action

• Istaroxime is a first-in-class dual mechanism agent targeting both heart contraction and relaxation via SERCA2a activation.

• Only late-stage SERCA2a activating agent in development, with four positive phase II studies and over 300 patients treated.

• Acquired in 2019, Istaroxime received Fast Track designation from the FDA and is being positioned for cardiogenic shock.

• Demonstrates increased blood pressure and cardiac output without raising heart rate, and often decreases heart rate.

• Shows improvements in renal function and maintains kidney health in heart failure patients.

Strategic focus and market opportunity

• Cardiogenic shock selected as lead indication due to high unmet need and $1.25B global market in 2020, projected to reach $3B by 2039.

• Portfolio includes next-generation preclinical SERCA2a activators for acute and chronic heart failure.

• Oral dosing and outpatient use planned for future dual mechanism and pure SERCA2a activators.

• Licensing partner in Greater China to start phase III acute heart failure study at their expense.

• Actively seeking additional licensing and business development opportunities.

Clinical data and physician feedback

• Cardiogenic shock is a severe, high-mortality condition with urgent need for innovation.

• 99 out of 100 surveyed U.S. cardiologists see high need for new drugs; 84% likely to use Istaroxime, with many making it first-line.

• Istaroxime improves both systolic and diastolic cardiac function, decreases filling pressures, and preserves renal function.

• No increase in arrhythmias observed in over 300 patients; reduces pulmonary congestion.

• Mechanism involves sodium-potassium ATPase inhibition and SERCA2a activation, improving both contraction and relaxation.