Amylyx Pharmaceuticals details 55% hypoglycemia reduction in Phase 3
New controlled event rates expand the avexitide readout, while the company still targets a year-end FDA application.
Adrian Kessler
A pivotal trial met its main endpoint with 55 per cent fewer low-blood-sugar events against placebo as Amylyx Pharmaceuticals, Inc. NASDAQAMLX released detailed avexitide results on Saturday. The company still targets an FDA application by the end of 2026 and a potential launch in 2027, if approved.
The Nov. 14 release adds arm-level event rates and secondary statistics to results first announced Aug. 18. Amylyx described the primary endpoint as FDA-agreed in that earlier announcement.
Amylyx is a Massachusetts drug developer focused on endocrine and neurodegenerative diseases.
Shares closed at US$30.29 on Nasdaq on Nov. 13, according to Google Finance's 4:00 p.m. EST timestamp for NASDAQAMLX. That quotation precedes Saturday's release and does not measure the response to the detailed findings.
Severe Event Rates Fell Against Placebo
LUCIDITY randomized 78 adults with post-bariatric hypoglycemia following Roux-en-Y gastric bypass, assigning 47 to avexitide and 31 to placebo. Avexitide was given as a daily injection under the skin at a dose of 90 milligrams. The blinded period was completed by 45 avexitide recipients and 28 placebo recipients.
Both groups received dietary management throughout the 16-week blinded comparison. The primary endpoint combined glucose-meter-confirmed Level 2 episodes with Level 3 episodes assessed by a blinded adjudication committee.
The adjusted event rate was 0.55 per week on avexitide, compared with 1.24 on placebo. The reduction was statistically significant, with p=0.0000034.
The adjudicated Level 3 event rate fell 47 per cent against placebo, with p=0.0016. Reductions also reached statistical significance for Level 2 events recorded by glucose meters and continuous monitors.
All prespecified secondary event-rate endpoints met the trial's multiplicity controls, which account for testing several outcomes. The placebo-controlled design measures benefit against a concurrent comparison group receiving placebo and the same dietary management.
Adverse events were generally mild to moderate and mainly gastrointestinal or injection-site reactions; there were no deaths or treatment-related serious adverse events during the blinded period. Investigators observed no clinically meaningful changes in body weight or fasting plasma glucose.
An FDA Application Remains Planned For Year-End
Avexitide blocks the GLP-1 receptor, aiming to reduce the exaggerated insulin response that drives low blood sugar in this condition. Amylyx says there are currently no FDA-approved therapies for post-bariatric hypoglycemia.
"We're moving with urgency toward an NDA submission by year end and continue to prepare for a potential commercial launch of avexitide in 2027, if approved," co-chief executives Justin Klee and Joshua Cohen said in Saturday's release.
The study's open-label extension remains ongoing, alongside an expanded-access programme for eligible adults. The extension provides longer follow-up beyond the blinded comparison and runs for 32 weeks.
Shares of Rezolute, Inc. NASDAQRZLT closed at US$3.87 on Nasdaq on Nov. 12, according to Google Finance's 4:00 p.m. EST timestamp. Its earlier quotation also predates Amylyx's detailed release.
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The broader State Street SPDR S&P Biotech ETF NYSEARCAXBI stood at US$151.20 at Google Finance's Nov. 12, 8:00 p.m. EST timestamp. These historical snapshots provide price context rather than a comparison of trading reactions.
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Amylyx's next scheduled discussion is an investor webcast on Nov. 15 at 11:45 a.m. EST, covering the LUCIDITY results and questions. The next regulatory milestone is the planned year-end application, which would put the evidence before the FDA for review.
