IRulya Therapeutics (QNCX) KOL event summary

Introduction and agenda

• Focus on LAM-001, an inhaled rapamycin (mTOR inhibitor) formulation, studied in PH-ILD, BOS post-lung transplant, and SAPH, with key data readouts expected between 2027 and 2028.

• Event structured into two sessions: PH-ILD and BOS, each followed by a Q&A.

• Presentations covered current therapies, unmet needs, and the role of mTOR inhibition in disease modification.

• Review of Phase 2a data for LAM-001 and ongoing clinical trial designs for PH-ILD and BOS.

KOL background and credentials

• Dr. Waxman: Director at Brigham and Women's Hospital, Harvard professor, lead PI on major PH studies, and lead investigator for LAM-001 PH-ILD trials.

• Dr. Yu: Director at MGH Cardiovascular Research Center, Harvard associate professor, expert in pulmonary hypertension and mTOR signaling.

• Dr. Hays: Medical Director at UCSF Advanced Lung Disease and Lung Transplant Program, principal investigator for BOS LAM-001 study.

Market insights and analysis

• PH-ILD affects approximately 200,000 patients in the US/EU; BOS post-lung transplant affects 30,000; SAPH affects 60,000.

• BOS is a leading cause of death post-lung transplant, with no FDA-approved therapies and high unmet need.

• PH-ILD and BOS care is concentrated in specialized centers, facilitating rapid uptake if approved.

• LAM-001 aims to address significant unmet needs in these serious, progressive pulmonary conditions.