Amylyx Pharmaceuticals (AMLX) 3rd Annual Guggenheim Healthcare Innovation Conference summary

LUCIDITY results

• Phase III LUCIDITY met its prespecified primary endpoint on Level 2/3 hypoglycemia rate (p=0.000003) and all secondary endpoints, with a favorable safety profile.

• Results were consistent across Level 2 and Level 3 events, and Level 2 measurements by finger stick and continuous glucose monitoring; findings aligned with prior trials.

• In 16-week data, no evidence of weight gain was observed; physicians cited efficacy, safety and tolerability as encouraging.

• Level 2 hypoglycemia below 54 mg/dL was described as a medical emergency; Level 3 events require independent rescue, and avexitide reduced both event types.

Regulatory and product plans

• NDA submission remains on track for year-end 2026; work began ahead of the Phase III readout, supported by prior trials and experienced regulatory, clinical and CMC teams.

• Manufacturing preparations are advanced, with no issues anticipated for submission; launch is planned for 2027, if approved.

• Intended commercial dose is one 90 mg injection; LUCIDITY used two consecutive 45 mg injections, while the open-label extension uses a single 90 mg injection.

• The submission will make the case that PBH after different gastric surgeries shares pathophysiology; if the label is limited to Roux-en-Y patients, that represents ~120,000 of an estimated 160,000 U.S. patients.

Commercial preparation and market

• Estimated U.S. PBH population is 160,000, with no FDA-approved treatment; ~90% of people with PBH describe themselves as disabled.

• Medical affairs and commercial leadership are in place; preparations include claims analyses, patient-journey research, payer work and disease education campaigns, set to ramp toward anticipated 2027 launch.

• Launch plans prioritize higher-volume sites and care teams, while digital tools can extend outreach to less concentrated locations; physicians expressed high intent to treat across levels of disease severity.

• A dedicated PBH ICD-10 code can help patients and sites track the condition; it is a tracking code, not a reimbursement code.

• Disease education campaign engagement has included reports of people receiving diagnoses; endocrinologists recognize the need for a treatment pathway.