The U.S. Food and Drug Administration has cleared the way for a phase 2/3 clinical trial of NTX-1024, an investigational ophthalmic solution for the treatment of vernal keratoconjunctivitis (VKC), a rare and severe inflammatory eye disease. The clearance of the Investigational New Drug (IND) application, announced by NexEos Bio, marks a significant step forward in the development of a targeted therapy for this challenging condition.
VKC is an eosinophil-driven inflammatory disease of the conjunctiva and cornea, often affecting children and young adults. It can cause intense itching, redness, discharge, and potentially lead to irreversible ocular damage, including vision loss, if left untreated. Current treatment options often involve topical corticosteroids, which can have long-term side effects such as glaucoma, increased risk of eye infections, and corneal degeneration.
NTX-1024 is designed to neutralize the toxic effects of eosinophil cationic protein, a key mediator of inflammation in VKC. According to Ken Mandell, MD, PhD, vision health program lead at NexEos Bio, “To our knowledge, NTX-1024 is the first investigational drug to directly target the toxic eosinophil granule proteins that cause damage to the conjunctiva and cornea in VKC.” This novel mechanism of action offers the potential for a safer and more effective treatment option.
The planned phase 2/3 trial will be a randomized, double-masked, vehicle-controlled study to evaluate the safety and efficacy of NTX-1024 ophthalmic solution in patients with VKC. This trial builds upon promising results from an earlier, open-label investigator-initiated study conducted in Mexico. That study, involving 15 adults with mild to moderate VKC, demonstrated significant improvements in patient-reported symptoms, such as itching and quality of life, as well as reductions in clinical signs of inflammation, including redness and corneal staining. Importantly, NTX-1024 was found to be safe and well-tolerated in this initial study.
“Developing NTX-1024 as a safe, well-tolerated and rapidly acting alternative to existing therapies is an important step to relieving the burden of VKC disease,” Mandell stated. He further explained that NTX-1024 is “expected to be steroid sparing,” potentially minimizing the long-term risks associated with chronic corticosteroid use.
The FDA’s granting of Orphan Drug Designation to NTX-1024 in January recognizes the unmet medical need in VKC and provides incentives for the development of therapies for rare diseases. This designation underscores the importance of finding effective treatments for this debilitating condition.
VKC is characterized by an influx of eosinophils, a type of white blood cell, into the conjunctiva, and cornea. These eosinophils release toxic proteins that cause inflammation and damage to the ocular surface. NTX-1024 aims to interrupt this process by neutralizing these harmful proteins, thereby reducing inflammation and protecting the eye from further damage.
The upcoming phase 2/3 trial will be crucial in determining the efficacy and safety of NTX-1024 in a larger and more diverse patient population. If successful, this therapy could offer a much-needed new treatment option for individuals suffering from VKC, potentially improving their quality of life and preserving their vision. The trial is expected to begin later this year.
For patients currently managing VKC, it’s important to continue following the guidance of their ophthalmologist and adhering to prescribed treatment plans. While NTX-1024 holds promise, it is still an investigational therapy and not yet available for widespread use. Ongoing research and clinical trials are essential to advancing the understanding and treatment of this complex eye disease.
