Pharma Friday – May 8, 2026

An Endocrine News roundup of the week’s pharmaceutical news, breakthroughs, and general information. *

Viridian Therapeutics Sees Positive Topline Results from Elegrobart Phase 3 REVEAL‑2 Clinical Trial in Chronic Thyroid Eye Disease

On May 5, Viridian Therapeutics, Inc., announced positive topline data from the REVEAL‑2 phase 3 clinical trial of elegrobart in patients with chronic thyroid eye disease (TED). Elegrobart is a subcutaneously delivered, half‑life‑extended monoclonal antibody targeting the insulin‑like growth factor‑1 receptor (IGF‑1R). REVEAL‑2 evaluated two dosing regimens, every four weeks (Q4W) and every eight weeks (Q8W), compared with placebo.

Viridian Therapeutics, Inc., is a biotechnology company focused on discovering, developing, and commercializing potentially best-in-class medicines for autoimmune and rare diseases.

“We are excited by today’s positive REVEAL 2 results and view these data as a major step forward for the chronic TED patient population. Given the IV-like proptosis response and our plans to launch with an at-home autoinjector, we believe elegrobart can meaningfully attract chronic patients to seek treatment. Elegrobart’s unmatched simplicity and convenience could uniquely drive expansion of the large and underserved chronic TED market,” said Steve Mahoney, president and chief executive officer of Viridian Therapeutics. “With our anticipated launch of veligrotug, which is a short IV infusion course, and two positive phase 3 REVEAL pivotal clinical trials supporting both Q4 weekly and Q8 weekly subcutaneous dosing for elegrobart, our portfolio has the potential to offer anti-IGF-1R efficacy and safety in convenient treatment regimens for TED patients with active or chronic disease.”

“Chronic TED remains a challenging condition. Many patients have been living with this disease for years or decades and would benefit from an effective and convenient treatment option,” said John Mandeville, MD, PhD, an oculoplastic surgeon at Ophthalmic Consultants of Boston and who is also a clinical associate at the Massachusetts General Hospital. “These REVEAL 2 results demonstrate the potential for elegrobart to provide meaningful improvement in the signs and symptoms of TED in as few as three doses. What’s more, a simple autoinjector that patients can use at home could be an attractive option for many patients living with chronic disease.”

Elegrobart REVEAL‑2 Phase 3 Topline Results

REVEAL‑2 assessed the efficacy and safety of subcutaneous Q4W or Q8W elegrobart versus placebo in patients with chronic TED. The clinical trial enrolled 204 patients, randomized 1:1:1 to elegrobart Q4W (n = 70), elegrobart Q8W (n = 68), and placebo (n = 66).

REVEAL‑2 Efficacy REVEAL-2 met its primary endpoint for both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) with high statistical significance (p < 0.0001). In addition, REVEAL-2 met all its proptosis key secondary endpoints in the Q4W and Q8W treatment arms with high statistical significance, and the Q4W treatment arm showed a statistically significant diplopia responder rate at week 24. Efficacy was generally consistent regardless of baseline Clinical Activity Score (CAS).

Amylyx Pharmaceuticals Expands Access Program for Adults with Post-Bariatric Hypoglycemia

On May 5, Amylyx Pharmaceuticals, Inc.  announced the launch of a U.S. Expanded Access Program (EAP) for up to 250 adults with post-bariatric hypoglycemia (PBH) to provide treatment access to avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist.

The EAP allows U.S. physicians to request avexitide for adults with PBH following Roux-en-Y gastric bypass (RYGB) surgery who have a serious unmet medical need, are unable to participate in an ongoing clinical trial, have exhausted available management options, and meet all other eligibility criteria. Initial eligible patients include individuals who have completed the pivotal Phase 3 LUCIDITY clinical trial and participants in a prior trial of avexitide in PBH following RYGB surgery.

“Listening to the PBH community is central to our work, and this dialogue directly informed our approach to the U.S. Expanded Access Program for avexitide,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “The experiences shared by those living with PBH underscore the profound unmet medical need they face every day. This program reflects our commitment to providing a potential option for eligible individuals as we continue to advance avexitide through clinical development.”

Avexitide is an investigational drug and has not been approved by the U.S. Food and Drug Administration (FDA) for any indication. Avexitide is being evaluated in the pivotal Phase 3 LUCIDITY clinical trial, a 16-week, multicenter, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of avexitide in adults with PBH following RYGB surgery. Participants who complete the 16-week double-blind period are eligible to enter a 32-week open-label extension period. The trial has enrolled 78 participants, with topline data readout anticipated in Q3 2026. If approved, commercial launch of avexitide is anticipated in 2027.

Physician Inquiry and Patient Eligibility

Individuals with PBH who are interested in learning more about potential access to avexitide through the EAP should speak with their treating physician or care team to determine whether they may be eligible. Access to avexitide through the EAP is limited, may change over time, and participation is not guaranteed. Additional information about the EAP is available at amylyx.com/global-access-policy.

About Avexitide

Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). In PBH, an exaggerated GLP-1 response leads to excessive insulin secretion, resulting in recurrent hypoglycemic events. Avexitide is a competitive GLP-1 receptor antagonist designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose levels. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events.

About Post-Bariatric Hypoglycemia (PBH)

PBH is a chronic metabolic condition that is estimated to affect approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery, sleeve gastrectomy and Roux-en-Y gastric bypass (approximately 160,000 people in the U.S.). PBH is thought to be driven by an exaggerated glucagon-like peptide-1 (GLP-1) response, primarily in response to food intake, leading to persistent, recurrent, and often debilitating rapid drops in blood glucose, known as hypoglycemia. The American Diabetes Association (ADA) recognizes hypoglycemia as a potential medical emergency because low blood glucose levels can compromise the body’s ability to maintain essential physiologic processes. In addition, hypoglycemia in the context of PBH may manifest as neuroglycopenia – an inadequate supply of glucose to the brain – which can cause confusion, cognitive dysfunction, loss of consciousness, and seizures. PBH can be associated with substantial disability, compromising safety, disrupting independent living, and affecting nutritional status and overall quality of life. Despite the substantial burden, there are currently no FDA-approved therapies for PBH.

About the LUCIDITY Trial

LUCIDITY (NCT06747468) is a 78-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following RYGB surgery. The Phase 3 trial is being conducted at 21 sites in the U.S. Participants were randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, a 16-week double-blind treatment period, and an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY is to evaluate the FDA-agreed upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated.

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