Amylyx Pharmaceuticals (NasdaqGS:AMLX) FY Conference Transcript

Summary of Amylyx Pharmaceuticals FY Conference Call Company Overview - Company: Amylyx Pharmaceuticals (NasdaqGS:AMLX) - Focus: Development of innovative therapies, particularly in the area of post-bariatric hypoglycemia (PBH) and other rare diseases Key Points on Avexetide and PBH - Avexetide: A competitive inhibitor of the GLP-1 receptor currently in a phase three pivotal study for PBH, following five successful prior studies that supported FDA Breakthrough Therapy designation [1][2] - Market Size: Approximately 160,000 people in the U.S. are estimated to have PBH, a number expected to grow due to the increasing prevalence of bariatric surgeries [3][4] - Bariatric Surgery: About 270,000 bariatric surgeries are performed annually in the U.S., with a significant portion of patients potentially developing PBH [7][10] - Awareness and Diagnosis: PBH has historically been under-recognized, but awareness is increasing, with presentations at medical conferences and discussions about creating an ICD-10 code for PBH [5][16] Clinical Development and Pipeline - AMX0035: In clinical development for Wolfram syndrome, with positive phase two results and phase three design pending FDA alignment [2][25] - Calpain-2 Antisense Oligonucleotide: Currently in phase one study for ALS, with safety and tolerability data expected later this year [2][25] Market Dynamics and Adoption Drivers - Patient Journey: Diagnosis of PBH can take one to three years post-bariatric surgery, with a significant number of patients actively managed at specialized centers [12][13] - Educational Efforts: There is a need for increased education among general endocrinologists and surgeons to recognize PBH symptoms and refer patients appropriately [13][15] - Launch Expectations: Anticipation of a bolus effect of initial patients seeking treatment upon approval, followed by a typical S-shaped launch curve [14][15] Phase Three Study Design - Study Duration: The phase three study has a treatment period of 16 weeks, increased from 4 weeks in phase two, with no evidence of tachyphylaxis expected [18][19] - Outcome Measurement: Primary outcomes will measure hypoglycemic events using the same methods as in phase two, ensuring consistency [19][20] Future Opportunities - Potential for Other Indications: The mechanism of Avexetide may have applications in other conditions related to persistent hypoglycemia, such as congenital hyperinsulinism and complications from upper GI surgeries [23][24] - Market Size for Wolfram Syndrome: Estimated at about 3,000 people in the U.S., with a larger population outside the U.S. [25] Conclusion - Financial Position: Recent financing provides a cash runway into 2028, supporting ongoing clinical programs and market entry strategies [2] - Outlook: The company is optimistic about its pipeline and the potential for Avexetide to address an unmet medical need in the PBH market [2][26]