Amylyx Pharmaceuticals (AMLX) investor relations material

Clinical development and trial design

• Lead asset AVEXITIDE is approaching pivotal phase III data readout, expected in the third quarter, with prior trials showing strong efficacy in reducing hypoglycemia in PBH patients.

• Phase III trial design closely mirrors earlier phases, with consistent inclusion criteria and endpoints, and a longer 16-week duration compared to previous 4-week studies.

• The study is powered to detect a 35% effect size with 90% power, using conservative statistical assumptions, and includes expert U.S. endocrinology centers.

• Placebo effect is expected to be minimal due to the severity of PBH and lack of effective existing treatments, with prior studies showing little to no placebo response.

• Expanded access program is underway, initially enrolling prior trial participants, with plans to expand to more patients before launch.

Market opportunity and commercialization plans

• Estimated 160,000 PBH patients in the U.S., based on bariatric surgery cohorts and claims data, with an ICD-10 code for PBH expected in October.

• Initial commercial focus will be on high-expertise centers, with ongoing education efforts for both physicians and patients to expand market reach.

• Market is expected to grow year over year, with potential expansion into other post-surgical hypoglycemia populations and ex-U.S. geographies.

• Adherence to daily subcutaneous injection is anticipated to be high, as patients are already accustomed to frequent glucose monitoring.

• Cash runway extends into 2028, supporting launch and early commercialization without resource constraints.

Regulatory and operational strategy

• NDA submission preparations are well advanced, aiming for rapid filing post-data to enable a 2027 launch, leveraging Breakthrough Therapy designation for expedited review.

• Regulatory interactions have been routine, with a placebo-controlled study and established endpoints supporting registration.

• No significant CMC or operational hurdles anticipated post-readout; submission process is streamlined.