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​In our November Town hall we brought together Elizabeth Hoffman, EB mom and fierce EB advocate, and Victoria Prieto Filice, passionate and dedicated biotechnologist and program manager at Eliksa Therapeutics to discuss significant advancements and updates in EB-related ocular care.

Elizabeth Hoffman’s Journey with Demah’s Eye Care 

Elizabeth shared her daughter Demah’s courageous battle with junctional EB, focusing on her severe and persistent corneal abrasions. Demah first began experiencing corneal abrasions in 2022. Doctors advised Elizabeth to use lubricating eye drops and humidifiers to help Demah. Unfortunately, in January 2023, Demah began getting many more abrasions that would last longer and longer, sometimes leaving her unable to see for days at a time, severely impacting her quality of life. 

"I felt a tremendous amount of guilt that she was experiencing this, and I just felt like we had to do something more." - Elizabeth Hoffman
Unsatisfied and determined, Elizabeth refused to accept this as her daughter’s reality. Their turning point came in November 2023, when they discovered Eliksa Therapeutics, a company working on innovative therapies for EB-related corneal abrasions. Armen Karamanian, CEO of Eliksa Therapeutics suggested Elizabeth look into using Amniotic Membrane (AM) Eye Drops. AM is rich in healing proteins like laminin, essential for patients with junctional EB. These drops are not available in the US, but they are in Italy, so the Hoffman’s decided to travel to Venice Italy to get access to the drops to treat Demah. 

Throughout this process, Elizabeth also learned about Autologous Serum Eye Drops. These are made from a patient’s own blood (or a parent’s blood) and use plasma to promote healing and prevent corneal abrasions.

Using a combination of both the serum and the AM eye drops has greatly improved Demah’s condition.  

“She has been abrasion free for months now…This year, it wasn’t even a thought. She could have her party, she could have her cake, she could SEE her cake, she could open up her presents.”  - Elizabeth Hoffman

​Eliksa Update with Victoria Prieto

Our next speaker, Victoria Prieto, provided a comprehensive update on Eliksa Therapeutics’ work, including their pioneering clinical trial for ELK 003, an amniotic fluid-derived biologic agent aimed at healing corneal abrasions in EB patients.

EBRP is proud to have invested $850,000.00 in Eliksa Theraputics in 2022 and see the incredible progress that's happened over the last two years. 

Eliksa's Journey: Pioneering Solutions for EB Ocular Challenges:

ELK 003: Science Simplified

• Derived from amniotic fluid (not membrane).
• Collected during routine C-sections from screened donors.
• Amniotic Fluid contains high concentrations of proteins critical to healing:
  • Collagen VII: Essential for skin and eye health (deficient in EB patients).
  • Laminin 332: Supports cell adhesion and wound repair.
  • Other key components: extracellular matrix proteins, hyaluronic acid, and growth factors.

Preclinical Experiment: Mouse Model

• Model: Collagen VII hypomorphic mice (EB-like genetic modification).
• Experiment: 
  • Corneal abrasions were induced in each eye of the mouse and then each eye had a treatment. Left eye had a placebo while the right eye had the treatment (ELK 003). 
  • ​Drops applied for 11 days, 4x per day 
• Experiment Results:
  • Placebo eyes took until day 8 to reach the same healing level.
  • ​ELK-treated eyes surpassed 90% healed by day 3.
• Demonstrated ELK 003's ability to accelerate healing time by more than 50%.

Clinical Trial in Chile

• Design: Two phases.
  • Observational Phase (4–6 months): Baseline established while participants continue usual treatment.
  • Treatment Phase (6 months): Daily application of ELK 003.
• Participants:
  • ​Goal: 15 (currently 9).
  • EB Subtypes: Junctional EB and Recessive Dystrophic EB.
• ​Timeline:
  • Expect to have the first cohort data from the Chile trial by summer 2025.
  • ​US trial expected to launch in summer 2026 (randomized, placebo-controlled).

Clinical Trial in U.S.

• Will need to seek FDA approval in the US so this will likely come with different requirements and regulations than the trial in Chile. 
• ​Design of the US trial will be finalized once we get Data from the Trial in Chile and are able to use those insights. 

Are you interested in getting involved with Eliksa Theraputics?

• Join Eliksa's Patient Advisory Group (PAG) to:
  • Participate in focus groups and surveys.
  • Receive trial updates and advocate for EB community needs.​

Join Eliksa's PAG Here!