AMLX

Lead Candidate Avexatide (Post-Bariatric Hypoglycemia, PBH) Clinical Progress

• Avexatide is a first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy Designation for PBH, a chronic condition with no approved therapies. The pivotal Phase III Lucidity trial has completed all last participant visits, with top-line data expected in late August or early September 2026; the database is not yet locked, and management remains blinded to results.
• The Lucidity trial is a randomized, double-blind, placebo-controlled study of 90mg daily Avexatide in adults with PBH after Roux-en-Y gastric bypass surgery, with a primary endpoint of reduction in composite Level 2 and Level 3 hypoglycemic events through 16 weeks.
• The company is actively advancing NDA preparation in parallel with trial completion, and has completed most non-clinical and CMC sections of the filing, with the 16-week Lucidity data being the final major component needed.

Commercial Launch Readiness

• Management estimates 160,000 total prevalent cases of PBH in the U.S. across Roux-en-Y gastric bypass and sleeve gastrectomy, 120,000 of which are from Roux-en-Y gastric bypass (the population enrolled in Lucidity).
• A dedicated disease state education campaign, Uncover the Mystery of Post-Bariatric Hypoglycemia, and companion educational website UncoverPBH.com have been launched for healthcare providers (HCPs) and patients. A field medical affairs team is already active to drive scientific exchange with endocrinologists, and hiring for commercial, marketing, and market access teams is ongoing in preparation for a potential 2027 launch.
• In June 2026, CMS and CDC published a dedicated ICD-10 code for PBH, effective October 1, 2026, reflecting growing recognition of the condition within the medical community.

Broader Pipeline Progress

• For AMX35 in Wolfram syndrome, 96-week Phase II open-label data presented in spring 2026 continued to show stabilization or improvement in glycemic control and vision, consistent with earlier 24-week and 48-week results. The company is currently working on Phase III trial design for this indication, including evaluation of the accelerated approval pathway.
• For AMX114 in ALS, the Phase I Lumina multiple ascending dose trial is progressing through dose cohorts, with enrollment ongoing in Cohort 3, and a favorable safety profile observed to date.
• For AMX318, a long-acting GLP-1 receptor antagonist, IND-enabling studies are underway, with an IND filing targeted for 2027.
• AMLEX entered into a second research collaboration with Gubra in July 2026 to screen and develop peptide candidates for another rare endocrine disease with high unmet need, following the original collaboration that generated AMX318.

• Management confirmed top-line data from the Phase III Lucidity trial of Avexatide remains on track for release in late August or early September 2026.
• A potential NDA submission for Avexatide, if the trial is successful, and a commercial launch in 2027 remains the company's baseline expectation.
• Current cash and marketable securities ($250.8 million) are expected to provide a cash runway into 2028, enough to fund operations through all key upcoming milestones, including the Lucidity readout, potential FDA approval, and the planned 2027 launch of Avexatide.
• An IND filing for AMX318 is targeted for 2027.
• Management reaffirmed the 160,000 prevalent population estimate for PBH in the U.S.

AMLEX is a clinical-stage biopharma focused on rare endocrine and metabolic diseases, with all revenue tied to pipeline development activity to date. No separate product segment revenue is reported, as the company is still in clinical development with no approved commercial products. For Q2 2026, total operating expenses were $45.7 million, a 7% increase from Q2 2025. Research and development (R&D) expenses were $23.8 million, down from $27.2 million in Q2 2025: the decrease stemmed from lower spending on AMX35 for progressive supranuclear palsy, offset by increased clinical development spending for lead candidate Avexatide. Selling, general and administrative (SG&A) expenses were $21.9 million, up from $15.6 million in Q2 2025, driven by a one-time legal charge and investments in commercial readiness initiatives for Avexatide. Non-cash stock-based compensation was $8.3 million for the quarter, up from $7.4 million in Q2 2025. The company ended Q2 2026 with $250.8 million in cash and marketable securities, down from $279.8 million at the end of Q1 2026.

• Top-line results from the Lucidity trial are still pending, and actual efficacy and safety outcomes may differ from management's expectations based on earlier Phase II data. It is the first pivotal Phase III trial conducted in PBH, so the magnitude of placebo response is unknown, and the trial could fail to meet its primary endpoint.
• If the trial is successful, FDA could restrict the initial indication label to only PBH after Roux-en-Y gastric bypass surgery, rather than all PBH regardless of antecedent bariatric surgery, which would limit the initial commercial opportunity until additional clinical data is generated.
• All pipeline candidates are still in clinical or pre-clinical development, and there is risk that candidates will fail to demonstrate safety or efficacy, resulting in delayed or abandoned development and no commercial approval.
• Actual commercial uptake after launch could differ from management estimates, due to ongoing diagnosis and awareness gaps for PBH, even with ongoing educational efforts.

Q: What steps are left before the Lucidity database lock, and how is the Early Access Program (EAP) progressing? / A: The final last patient visit completed in mid-late July 2026. The only remaining steps are rigorous data cleaning and verification to ensure accuracy, which is ongoing as of the call. Top-line data remains on track for late August/early September. The EAP is in early rollout, initially open to patients who completed prior Avexatide trials or the Lucidity double-blind period who wish to continue access. Site activation for the separate EAP protocol is ongoing, and patient enthusiasm for participation is high.

Q: Will the Lucidity trial data support a broad PBH label covering all bariatric surgeries, and what is the realistic 5-year treated patient population? / A: Lucidity only enrolled patients with PBH after Roux-en-Y gastric bypass, but Phase IIb data included patients with PBH after other bariatric surgeries and showed consistent efficacy. Management will argue to FDA for a broad label based on shared PBH pathophysiology regardless of surgery. If FDA restricts the label to Roux-en-Y PBH, an additional small, low-burden confirmatory study would be required to expand labeling. At launch, the eligible population would still be 120,000 patients, a substantial commercial opportunity. The 160,000 total prevalent PBH estimate is confirmed; launch will focus on high-volume centers with known patients initially, and growth will come over time as awareness expands, with high endocrinologist intent to treat once an approved therapy is available.

Q: What assumptions were made around placebo response for the Lucidity trial, and how is the trial powered? / A: Prior Phase II data shows a ~30% treatment difference versus placebo in mean hypoglycemic event reduction. The trial was conservatively powered at 90% to detect a 35% treatment difference versus placebo, even assuming a 50% placebo effect. Management notes no prior trial has shown a 50% placebo effect, so the powering assumptions are highly conservative, and the primary endpoint of Level 2/3 hypoglycemic events is clinically meaningful to both physicians and patients.

Q: What is the current competitive landscape for PBH, and what is the development plan for long-acting AMX318? / A: Management states there are no other candidates that have demonstrated a clinical profile matching Avexatide for PBH, so Avexatide would be the first and only approved therapy if approved, with no meaningful near-term competition. AMX318, the long-acting GLP-1 antagonist from the Gubra collaboration, is still in IND-enabling studies, with all injectable formulation options (vials, auto-injectors, pens) on the table, and no technical formulation hurdles expected. The company intends to continue innovating in the PBH space after launching Avexatide.