Eton Pharmaceuticals Announces Licensing and Initiation of ET-900 Program Evaluating Mecasermin/IGF-1 for Treatment of Infantile and Epileptic Spasms

ET-900 would align with Eton’s planned entry into pediatric neurology, while the company retains commercial rights.

Rhea-AI Summary

Eton Pharmaceuticals (Nasdaq: ETON) licensed Baylor College of Medicine patent rights and launched ET-900 to evaluate mecasermin for infantile and epileptic spasms. Mecasermin is the active ingredient in INCRELEX, which Eton markets in the United States. Eton retains commercial rights to ET-900.

In an animal model, recombinant human insulin-like growth factor 1 (IGF-1) reduced daily spasm counts by approximately 50% and eliminated spasms in 20% of treated animals. A research Investigational New Drug application has been submitted for a University of Chicago-sponsored Phase 2 study in children with refractory epileptic spasms. The study is expected to enroll up to 20 patients ages 2–18 years across three sites, with initial enrollment and dosing targeted for Q1 2027. Mecasermin is not FDA-approved for these uses, and safety and efficacy for them have not been established.

Positive

• Minor pointET-900 licensing secures Baylor patent rights covering IGF-1 treatment of spasms; Eton retains commercial rights.
• Minor pointAnimal-model treatment reduced daily spasms approximately 50% and eliminated spasms in 20% of treated animals.
• Minor pointResearch IND submitted for an investigator-sponsored Phase 2 study evaluating mecasermin in children with refractory epileptic spasms.
• Minor point. Forward-looking: it has not happened yet and may not happen.Phase 2 study expects up to 20 patients, with initial enrollment and dosing targeted for Q1 2027.

Negative

• None.

Key Figures

Reduction in daily spasm counts: approximately 50% Animals with spasms eliminated: 20% Planned enrollment: up to 20 patients +4 more

Reduction in daily spasm counts

approximately 50%

Animal model; recombinant human IGF-1 treatment

Animals with spasms eliminated

20%

Animal model; treated animals

Planned enrollment

up to 20 patients

Investigator-sponsored Phase 2 study

Patient age range

2–18 years

Investigator-sponsored Phase 2 study

Injection frequency

twice daily

Initial treatment period

Initial treatment period

three to five weeks

Investigator-sponsored Phase 2 study

Targeted initial enrollment and dosing

Q1 2027

ET-900 investigator-sponsored Phase 2 study

Key Terms

igf-1, investigational new drug application, subcutaneous injections, electroencephalogram

4 terms

igf-1 medical

"Mecासermin is a recombinant form of IGF-1"

A naturally occurring protein hormone that helps cells grow, survive and repair, acting like a biological 'fertilizer' for tissues. Investors watch IGF-1 because drugs that raise, block, or mimic it can affect treatments for growth disorders, metabolic diseases and certain cancers, making it a common target in clinical trials; trial results, safety concerns or regulatory decisions tied to IGF-1 activity can materially change a healthcare company’s value.

investigational new drug application regulatory

"A research IND has been submitted for an investigator-sponsored Phase 2"

An investigational new drug application is a formal request made to regulatory authorities to begin testing a new medication in humans. It is a critical step in the drug development process, as approval indicates the drug has passed initial safety checks and can be studied further. For investors, this signals that a potential new treatment is progressing through its early testing stages, which can impact the company's future growth prospects.

subcutaneous injections medical

"Patients will receive twice-daily subcutaneous injections"

Subcutaneous injections are shots given into the thin layer of fat just beneath the skin, usually with a small needle—think of tucking medicine into a little pocket under your skin. For investors, the delivery route matters because subcutaneous products are often easier for patients to use outside hospitals, can reduce administration costs, affect safety and dosing schedules, and therefore influence a therapy’s market size, pricing, and regulatory prospects.

electroencephalogram medical

"resolution or improvement of associated electroencephalogram (“EEG”) abnormalities"

An electroencephalogram (EEG) is a medical test that records the brain’s electrical activity through sensors placed on the scalp, producing a visual trace much like an audio recording of brain waves. For investors, EEGs matter because they are standard clinical tools and trial endpoints for neurological drugs and devices, so EEG results can influence a company’s regulatory progress, market potential, and valuation by showing objective signs of treatment effect or diagnostic capability.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• ET-900 targets a potential new indication for mecasermin, the active ingredient in Eton’s INCRELEX^®
• Eton has licensed patent rights from Baylor College of Medicine covering the use of insulin-like growth factor1 (IGF-1) for infantile and epileptic spasms
• Research conducted at Baylor College of Medicine and Texas Children’s Hospital and funded by CURE Epilepsy and NIH, showed IGF-1 therapy led to reduction in spasms in the majority of treated animals
• An Investigational New Drug application (IND) has been submitted to the United States Food and Drug Administration (FDA) for an investigator-sponsored Phase 2 study to evaluate mecasermin/IGF-1 in children with refractory epileptic spasms, with first patient dosing planned in Q1 2027
• Eton retains commercial rights to ET-900 and the product candidate would align with the Company’s planned entry into the pediatric neurology therapeutic area
  
  

DEER PARK, Ill., Nov. 02, 2026 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc. (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced ET-900, a new development program evaluating mecasermin injection for the treatment of infantile and epileptic spasms. Mecasermin is a recombinant form of IGF-1 and the active ingredient in INCRELEX, which Eton markets in the United States. The Company has entered into a license agreement with Baylor College of Medicine covering patent rights related to the use of IGF-1 for the treatment of spasms.

“These spasms can have devastating and lifelong consequences for children and their families, and there remains a significant and urgent need for new therapeutic approaches due to high rates of resistance to current therapies,” said Sean Brynjelsen, Chief Executive Officer of Eton Pharmaceuticals. “The Baylor research provides a compelling scientific rationale for evaluating IGF-1 therapy for use with this condition. With an IND now submitted, we are excited to help translate this research into meaningful clinical advancement.”

Research led by Dr. John W. Swann, Ph.D., Distinguished Emeritus Professor of Pediatrics and Neuroscience at Baylor College of Medicine, and funded by CURE Epilepsy has identified impaired IGF-1 signaling as a potential contributor to the development of epileptic spasms. In an animal model, treatment with recombinant human IGF-1 reduced daily spasm counts by approximately 50% and eliminated spasms in 20% of treated animals. These preclinical findings provide a scientific rationale for evaluating mecasermin in children with refractory epileptic spasms.

“The planned clinical trial is also warranted because studies of spasms patients with structural brain abnormalities indicate that IGF-1 levels are reduced in their brains. Consequently, we hope that mecasermin/IGF-1 will serve as a replacement therapy to restore normal brain activity,” added Dr. Swann.  

“CURE Epilepsy is thrilled to see this long-standing line of research from Dr. Swann’s lab move into human clinical trials. Dr. Swann was part of our Infantile Spasms Initiative, which began in 2012, and he and his collaborators have continued to fight to move their basic science discoveries toward clinical relevance for patients and families. Pediatric refractory epileptic spasms are catastrophic; these children urgently need new and better options, and we are grateful to Eton Pharmaceuticals for sponsoring this important next step towards a potential new treatment,” said Beth Dean, CEO of CURE Epilepsy.

A research IND has been submitted for an investigator-sponsored Phase 2 to evaluate mecasermin for efficacy, safety, and tolerability in children with refractory epileptic spasms. The study will be sponsored by the University of Chicago and is expected to enroll up to 20 patients ages 2–18 years across three sites, University of Chicago, Baylor College of Medicine, and UCLA, whose spasms have not responded to, or who have been unable to tolerate, hormonal therapy and vigabatrin. Patients will receive twice-daily subcutaneous injections during an initial three- to five-week treatment period. An additional treatment course may be available to certain patients based on clinical response. The study will assess spasm resolution, changes in daily spasm frequency, and resolution or improvement of associated electroencephalogram (“EEG”) abnormalities. Additional assessments will evaluate safety, tolerability, and adaptive functioning. Mecasermin is not FDA-approved for the treatment of infantile or epileptic spasms, and its safety and efficacy for these uses have not been established. Initial enrollment and dosing are targeted for the first quarter of 2027.

About Infantile and Epileptic Spasms

Infantile epileptic spasms syndrome is a rare, severe form of epilepsy that typically begins between the ages of 1 and 24 months. According to the American Academy of Pediatrics, approximately 2,500 children are diagnosed with infantile spasms in the United States each year. When spasms persist beyond the age of 2 they are referred to as epileptic spasms.

These seizures, known as epileptic spasms, frequently occur in clusters and may be accompanied by hypsarhythmia, a highly abnormal pattern of brain activity detected on an EEG. Spasms can persist beyond age two into later childhood. Prompt diagnosis and treatment are critical because persistent spasms may adversely affect neurological development.

Treatment resistance remains a significant challenge. A peer-reviewed study published in Epilepsy & Behavior reports that seizures in approximately half of children with infantile spasms fail to improve with initial treatment attempts. Current therapies are not effective for every patient and can be associated with significant limitations. For children whose spasms persist despite treatment, these challenges underscore an urgent need for new therapeutic options.

About Eton Pharmaceuticals

Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has twelve rare disease products: KHINDIVI^® (hydrocortisone), INCRELEX^® (mecasermin), ALKINDI SPRINKLE^® (hydrocortisone), DESMODA^™ (desmopressin acetate), GALZIN^® (zinc acetate), HEMANGEOL^® (propranolol hydrochloride), PKU GOLIKE^®, IMPAVIDO^® (miltefosine), LIBERVANT^® (diazepam), Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: AMGLIDIA^® (glyburide oral suspension), ASN-001, ET-700, ET-800, ET-900, and ZENEO^® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com.

Investor Relations:
Lisa M. Wilson, In-Site Communications, Inc.
T: 212-452-2793
E: lwilson@insitecony.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did the animal research supporting Eton’s ET-900 program show?

Recombinant human IGF-1 reduced daily spasm counts by approximately 50% and eliminated spasms in 20% of treated animals. These are preclinical findings; mecasermin’s safety and efficacy for infantile or epileptic spasms have not been established.

Who is eligible for Eton’s ET-900 Phase 2 study?

The study is expected to enroll children ages 2–18 years whose spasms have not responded to, or who have been unable to tolerate, hormonal therapy and vigabatrin. The three study sites are the University of Chicago, Baylor College of Medicine and UCLA.

How will mecasermin be given and assessed in the ET-900 Phase 2 study?

Patients will receive twice-daily injections under the skin during an initial three- to five-week treatment period. Certain patients may receive an additional course based on clinical response. Assessments include spasm resolution, daily spasm frequency, abnormalities in electrical brain activity, safety, tolerability and adaptive functioning.