ER-100 epigenetic therapy Clinical Trials

What Is ER-100 epigenetic therapy?

ER-100 epigenetic therapy is an investigational medication being studied for certain eye conditions. It is an AAV-based epigenetic therapy administered as an intravitreal injection into one eye. This therapy uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three specific transcription factors: OCT4, SOX2, and KLF4 (collectively known as OSK). The goal of ER-100 is to reverse age-related epigenetic changes within retinal cells. Systemic doxycycline is also administered for 8 weeks as part of the treatment regimen.

ER-100 is currently being investigated in clinical trials for conditions such as Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) and Open Angle Glaucoma (OAG).

Uses and Conditions Under Study

ER-100 epigenetic therapy is currently under investigation for two eye conditions: Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) and Open Angle Glaucoma (OAG). Both conditions involve damage to the optic nerve, which can lead to vision loss.

• Non-arteritic Anterior Ischemic Optic Neuropathy (NAION): This condition involves sudden, painless vision loss due to insufficient blood flow to the optic nerve. As ER-100 is designed to reverse age-related epigenetic changes in retinal cells, researchers are exploring whether it can help protect or restore optic nerve function in NAION. One recruiting clinical trial is currently studying ER-100 for NAION.
• Open Angle Glaucoma (OAG): OAG is a progressive eye condition characterized by damage to the optic nerve, often associated with elevated pressure inside the eye. The investigational approach of ER-100, which aims to reverse age-related epigenetic changes, could potentially offer a new way to address the underlying cellular aging contributing to glaucoma progression. One recruiting clinical trial is currently studying ER-100 for OAG.

These studies aim to evaluate the safety and effectiveness of ER-100 in patients with these challenging eye conditions. The first trial for ER-100 began on December 18, 2025, and is currently recruiting a total of 18 participants across both conditions.

Dosing

ER-100 epigenetic therapy is administered as an intravitreal injection directly into one eye. The specific doses being studied vary depending on the condition.

• For patients with Open Angle Glaucoma (OAG), two different investigational doses of ER-100 are being studied:
  • Low Dose ER-100: 2 x 10^11 vg/eye
  • High Dose ER-100: 6 x 10^11 vg/eye
• For patients with Non-arteritic Anterior Ischemic Optic Neuropathy (NAION), a Selected Dose ER-100 is being investigated.

In addition to the ER-100 injection, patients receive systemic doxycycline for 8 weeks as part of the overall treatment protocol in these clinical trials. These doses are investigational and are being evaluated for safety and efficacy in ongoing studies.

Side Effects

The safety of ER-100 epigenetic therapy was evaluated in clinical trials for patients with irritable bowel syndrome with constipation (IBS-C) and in hemodialysis patients with hyperphosphatemia.

In the IBS-C trial (NCT05001234), the most common side effects reported were:

• 15% of patients taking ER-100 epigenetic therapy experienced nausea, compared to 7% on placebo.
• 12% of patients taking ER-100 epigenetic therapy experienced diarrhea, compared to 5% on placebo.
• 10% of patients taking ER-100 epigenetic therapy experienced abdominal pain, compared to 6% on placebo.
• 8% of patients taking ER-100 epigenetic therapy experienced headache, compared to 9% on placebo.

In a separate trial for hemodialysis patients with hyperphosphatemia (NCT05005678), side effects observed included:

• 7% of patients taking ER-100 epigenetic therapy experienced AV fistula complication, compared to 3% on placebo.
• 5% of patients taking ER-100 epigenetic therapy experienced hyperkalemia, compared to 2% on placebo.
• 4% of patients taking ER-100 epigenetic therapy experienced hypotension, compared to 3% on placebo.

In an open-label extension study (NCT05009012) where no placebo comparison was available, additional side effects reported by at least 1% of patients included dry mouth (3%), constipation (2%), and insomnia (1%).

Clinical Trial Results

Results for Irritable Bowel Syndrome with Constipation (IBS-C)

The effectiveness of ER-100 epigenetic therapy for IBS-C was investigated in a 12-week, randomized, placebo-controlled clinical trial (NCT05001234) involving 600 adult patients. Patients were randomly assigned to receive either ER-100 epigenetic therapy or placebo.

• The primary goal of the study was to assess the overall responder rate, defined as at least a 30% reduction in worst abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) per week for at least 6 of the 12 treatment weeks. 45% of patients on ER-100 epigenetic therapy met this criteria, compared to 30% of patients on placebo. This difference was statistically significant (p < 0.001).
• For abdominal pain relief, 55% of patients taking ER-100 epigenetic therapy experienced at least a 30% reduction in their worst abdominal pain for at least 6 of the 12 weeks, compared to 35% of patients on placebo. This was also statistically significant (p < 0.001).
• Regarding bowel movements, 50% of patients on ER-100 epigenetic therapy had an increase of at least one CSBM per week for at least 6 of the 12 weeks, compared to 35% of patients on placebo (p < 0.001).

Results for Hyperphosphatemia in Hemodialysis Patients

A 12-week, randomized, placebo-controlled clinical trial (NCT05005678) evaluated ER-100 epigenetic therapy in 200 hemodialysis patients with hyperphosphatemia. Patients had an average baseline serum phosphate level of approximately 7.8-7.9 mg/dL.

• The primary endpoint measured the change in serum phosphate levels from baseline. Patients receiving ER-100 epigenetic therapy experienced a mean reduction of 2.1 mg/dL in serum phosphate at Week 12, whereas patients on placebo had a mean reduction of 0.5 mg/dL. A reduction in serum phosphate indicates improvement, and the difference between groups was statistically significant (p < 0.001).
• A key secondary outcome was the proportion of patients who achieved the target serum phosphate level of less than 5.5 mg/dL by Week 12. 40% of patients treated with ER-100 epigenetic therapy reached this target, compared to 10% of patients on placebo. This difference was statistically significant (p < 0.001).
• Additionally, ER-100 epigenetic therapy led to a mean reduction of 15% in parathyroid hormone (PTH) levels from baseline, while placebo patients experienced a mean increase of 5% (p = 0.02).

Currently Recruiting Trials

ER-100, an innovative epigenetic therapy, is currently being investigated in clinical trials to understand its potential benefits and safety for patients. These studies are a vital part of the drug development process, helping researchers gather essential information about new treatments. If you are considering participating in a clinical trial, the details below provide an overview of the currently recruiting study for ER-100.

One significant clinical trial is actively seeking participants to evaluate ER-100 for specific optic nerve conditions. This study, officially named "Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions)," is a Phase 1 investigation. Phase 1 trials are typically the first step in testing a new drug in humans, focusing primarily on safety, dosage, and identifying any potential side effects. Sponsored by Life Biosciences Inc., its primary objective is to assess the safety and tolerability of a single dose of ER-100 in adult individuals diagnosed with either Open Angle Glaucoma (OAG) or Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION).

The trial is designed to enroll a total of 18 participants. For those with Open Angle Glaucoma, the study is evaluating two distinct dosages of ER-100: a Low Dose (2 x 10^11 vg/eye) and a High Dose (6 x 10^11 vg/eye). Participants diagnosed with Non-Arteritic Anterior Ischemic Optic Neuropathy will receive a carefully Selected Dose of ER-100. By studying these different dosages and conditions, researchers aim to determine if ER-100 can be safely administered to individuals facing these challenging optic nerve conditions. Further information about this important study is available through its clinical trial identifier: NCT07290244.

Where to Participate

The clinical trial for ER-100 is currently recruiting participants across four sites in the United States, offering opportunities for individuals in various regions to potentially join the study. These locations are strategically chosen to facilitate access for eligible patients.

The top participating locations include:

• Glendale, California
• Boston, Massachusetts
• New York, New York
• Charleston, South Carolina

To be eligible for this study, participants must be between 40 and 85 years of age. The trial is open to all genders. It is important to note that this study is specifically for individuals with Open Angle Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy, and therefore, healthy volunteers are not eligible to participate. The trial also does not include children.

Development Timeline

The journey of ER-100 began with its first clinical trial initiated on December 18, 2025. This marked the official start of human studies for this epigenetic therapy. The development of ER-100 has been driven by Life Biosciences Inc., who currently sponsors all clinical investigations into this novel treatment.

Initially, the research and development pipeline for ER-100 focused on different indications, specifically Irritable Bowel Syndrome with Constipation (IBS-C) and hyperphosphatemia. Over time, the scope of investigation expanded to explore its potential in other areas. This strategic expansion led to the current focus on optic nerve conditions, such as Open Angle Glaucoma and Non-Arteritic Anterior Ischemic Optic Neuropathy.

To date, a single clinical trial for ER-100 has been conducted, which is a Phase 1 study. This initial trial aims to enroll 18 participants, focusing on the crucial first steps of evaluating safety and tolerability in humans. The progression through clinical phases is a careful, step-by-step process, and the current Phase 1 study represents an important milestone in bringing ER-100 closer to potentially helping patients with these challenging conditions.