Amylyx Pharmaceuticals' Investigational Drug Shows Improved Pancreatic Function In Patients With Inherited Disorder

Wednesday, Amylyx Pharmaceuticals Inc AMLX released interim data from the ongoing Phase 2 HELIOS trial of AMX0035 for Wolfram syndrome.

Wolfram syndrome is an inherited condition that typically includes childhood-onset insulin-dependent diabetes mellitus and progressive optic atrophy.

The interim data from eight participants who have completed 24 weeks of treatment demonstrated that AMX0035 had a clinically meaningful effect on key outcomes measuring the progression of diabetes, visual decline, and overall disease burden.

In this interim analysis of eight participants treated with AMX0035, increases were observed on average in the primary outcome of total C-peptide response (C-peptide AUC change from baseline), including in the 90-minute response at Week 24 (+15.6 ng*min/mL). 

In Wolfram syndrome, progressive decline would have been expected on this measure. 

Additionally, seven out of eight participants demonstrated at least a 30-minute shorter time to peak C-peptide response. 

In Wolfram syndrome, a progressive increase in time to peak C-peptide response, indicating slower pancreatic response and reduced total C-peptide response, would have been expected.

The following includes additional key data from the interim analysis:

• Hemoglobin A1C (HbA1c) was reduced by 0.26% on average after 24 weeks of AMX0035 treatment, with six out of eight participants showing improvement in their HbA1c. Many studies have associated reduced HbA1c with better clinical outcomes.
•  On average, visual acuity improved +0.05 -LogMAR, with five out of eight participants demonstrating some improvement in vision. 
•  Of those who improved, one participant changed from legally blind to legally sighted. Optical coherence tomography outcomes have not yet been assessed and will be included in the final data analysis from HELIOS.
•  All participants showed disease stability or improvement at Week 24. 

The safety profile of AMX0035 in HELIOS was consistent with prior safety data. 

Last week, Amylyx Pharmaceuticals shared a restructuring plan related to removing Relyvrio/Albrioza for amyotrophic lateral sclerosis in the U.S. and Canada due to disappointing Phase 3 trial data. 

The company will reduce its workforce by approximately 70% to focus resources on key clinical and preclinical programs and extend the expected cash runway into 2026.

Price Action: AMLX shares are down 6.74% at $2.49 on the last check Wednesday.