Avalyn Pharma (AVLN) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Strategy and delivery

• Inhaled pirfenidone and nintedanib aim to deliver more than 10-fold lower doses than oral therapy, improve lung exposure and reduce systemic exposure and tolerability burdens.

• Better tolerability is intended to help patients remain on treatment longer and enable combination therapy, which management sees as a potential shift in pulmonary fibrosis care.

• The small handheld nebulizer delivers a gentle mist with normal tidal breathing; it is 510(k)-cleared, patent-protected, licensed and exclusive for the molecules.

• Bronchoalveolar lavage comparisons showed better lung exposure for both inhaled programs; an in silico model presented at ERS indicated alveolar deposition even in severe IPF lungs.

Clinical programs

• AP01 historical data showed lung-function stabilization trends across cohorts and in long-term extensions; five-year IPF data had responder bias, while some patients were seven years from diagnosis and stable.

• Imaging analysis found less change in fibrosis at the selected AP01 high dose; fibrosis quantification correlated with lung-function change.

• The phase IIb MIST study of AP01 in PPF enrolled 398 patients, above its planned 375, ahead of schedule; patients may receive background nintedanib or immunosuppressants and are stratified accordingly.

• AP02 is a 12-week inhaled nintedanib dose-ranging study; management expects the duration to distinguish doses, select a dose for phase III and move the program forward quickly.

• AP02 is being conducted outside the U.S., including Canada, Europe, Australia, New Zealand, Argentina and Turkey.

Market and combination strategy

• Management sees potential additive effects from pirfenidone and nintedanib, with clinical data from both phase II programs expected to inform positioning and combination plans.

• MIST includes patients on background oral nintedanib and immunosuppressants, providing a real-world assessment of inhaled pirfenidone alongside existing therapies.

• Management cited ~300,000 diagnosed U.S. patients and a $42bn market estimate using $150,000 annual therapy pricing; OFEV was described as a $4bn global product.

• AP03 is the initial combination strategy; over the next five to seven years, management plans to consider additional mechanisms as the field evolves.

• Potential future indications include chronic lung allograft dysfunction and interstitial lung abnormalities, which management identified as areas of unmet need.