4D Molecular Therapeutics (FDMT) Morgan Stanley 24th Annual Global Healthcare Conference summary

Platform and pipeline overview

• Next-generation gene therapy platform uses directed evolution to create optimized vectors, improving safety, efficacy, and cost for large-market indications.

• Lead asset 4D-150 targets neovascular retinal diseases, starting with wet AMD, expanding to diabetic macular edema (DME) and diabetic retinopathy.

• Pipeline includes additional programs in retina and lung indications.

Clinical development and trial design

• 4D-150 aims to dramatically extend treatment durability for wet AMD, potentially reducing or eliminating the need for frequent injections.

• Phase I/II PRISM program showed robust dose response and consistent safety across severe and newly diagnosed wet AMD patients.

• Phase III 4FRONT program enrolls treatment-naive and recently diagnosed patients, with stringent criteria to optimize efficacy and safety endpoints.

• Over 525 patients randomized in each 4FRONT study, increasing statistical power for global and U.S. endpoints.

Commercial partnerships and financials

• Otsuka holds Asia-Pacific commercial rights for 4D-150, representing 10-15% of global value; $110M+ upfront payment provided non-dilutive funding.

• Partnership enabled acceleration of DME program and global phase III trial, with regulatory alignment from FDA and EMA.

• $431 million in cash as of June 30, 2024, with runway into the second half of 2028.