The FDA has granted Orphan Drug Designation (ODD) to Cellusion’s CLS001, an investigational regenerative compound for the treatment of bullous keratopathy.
Let’s start with the company.
The Tokyo, Japan-based Cellusion Inc. was founded in 2015 as a regenerative medicine startup company intent on solving the global shortage of corneal transplant donors.
The company has developed a novel mass culture system for corneal endothelial (CE) cells produced from induced pluripotent stem cells (iPSCs) as part of its mission to deliver novel therapies with cellular technology.
Now talk about CLS001.
CLS001 is an iPSC-derived corneal endothelial (CE) therapy designed as an alternate solution for longstanding corneal transplantation problems by injecting CE cells from iPSCs.
The therapy injects CE cells from iPSCs via a syringe, and would require a patient to remain face-down for 3 hours following treatment.
Any clinical data on it yet?
In progress…
Back in March 2023, the company announced that the first patient had received a transplant of CLS001 in its first-in-human (FIH) study of patients with regrafted cases of bullous keratopathy.
Tell me about it.
The exploratory, single-arm, FIH clinical study (jRCTa031210199) is examining the safety and efficacy of CLS001 transplanted in three patients (ages 45-85) with regrafted bullous keratopathy.
Inclusion criteria included a central CE cell density of > 500 cells/mm2 measured by specular or confocal microscope, or undetectable.
And what’s being measured?
Primary outcomes include:
- Systemic safety endpoints
- All adverse events, systemic systems included
- Ophthalmologic safety endpoints
- Adverse events caused by iPSC-derived CE substitute cells:
- Unintended cell proliferation
- Increased intraocular pressure (IOP)
- Rejection reaction
- Adverse events from transplant surgery and procedures:
- Infectious disease
- Cataract progression
- Expulsive hemorrhage
- Adverse events caused by iPSC-derived CE substitute cells:
Secondary outcomes include efficacy endpoints such as :
- Visual acuity
- Corneal thickness
So what’s the plan for this therapy?
Cellusion has partnered with New Jersey-based Minaris Regenerative Medicine, LLC, a global contract and development manufacturing organization, after announcing a letter of intent (LOI) in October 2022.
Minaris will perform process optimization as part of the LOI to meet requirements for the FDA. If approved, they will manufacture CLS001 as a corneal endothelial cell regenerative therapy in the United States.
Gotcha. And about this designation … what’s the significance?
With no FDA-approved treatment for bullous keratopathy on the market, CLS001 has the potential to fill the unmet need for treating blindness due to corneal diseases via—corneal transplants—not just in the U.S., but across the globe.
Next steps?
With the FIH trial ongoing, there’s no word on a data readout yet. However, the company is also planning to initiate another trial in Japan that will be followed by global studies within the U.S. and European Union.