Cloudbreak Pharma announces positive clinical results for vascularized pinguecula therapy

Cloudbreak Pharma Inc., a clinical-stage ophthalmology company based in Irvine, California, reported that its investigational eye drop therapy, CBT-004, achieved positive topline results in a Phase 2 clinical trial targeting vascularized pinguecula—a common but difficult-to-treat ocular surface condition. The announcement was made via a press release from the company and supported by commentary from trial investigators and independent ophthalmology experts.

What is vascularized pinguecula and why does it matter?

Vascularized pinguecula is a prevalent, non-cancerous conjunctival growth that becomes problematic when abnormal blood vessels and inflammation develop, leading to visible redness (hyperemia), irritation, and ocular discomfort. Affecting millions of Americans, the condition has historically lacked FDA-approved, targeted therapies.

Current treatments often rely on off-label corticosteroids, which carry risks of elevated intraocular pressure and long-term safety concerns. In more severe cases, surgical excision is considered, though this option is invasive and not ideal for broader patient populations. With no standard of care specifically approved for vascularized pinguecula, ophthalmologists and patients alike have sought safer and more effective alternatives.

What happened in Cloudbreak Pharma’s Phase 2 study?

The multicenter, randomized, double-masked, vehicle-controlled Phase 2 study enrolled 88 adult patients diagnosed with vascularized pinguecula and its associated redness and discomfort. Participants were assigned to receive one of two concentrations of CBT-004 or a placebo-like vehicle control. The primary goal of the study was to measure changes in conjunctival hyperemia at Day 28, using standardized digital imaging analyzed by an independent reading center.

According to Cloudbreak Pharma, both concentrations of CBT-004 significantly outperformed the vehicle in reducing redness by Day 28. Key improvements were observed as early as Day 7, with the higher concentration delivering the most pronounced early results.

What symptom improvements did patients report?

Patients treated with CBT-004 experienced statistically significant relief across five commonly reported symptoms associated with vascularized pinguecula. These included burning or stinging sensations, itching, the feeling of a foreign body in the eye, general discomfort, and pain. Compared to those who received the vehicle control, individuals in both CBT-004 treatment arms reported consistently better outcomes. These improvements were measured using validated patient-reported outcome scales and were observed throughout the 28-day treatment period, reinforcing the therapy’s potential to address both visible redness and subjective discomfort.

How safe is CBT-004?

Safety remains a cornerstone in the evaluation of ophthalmic therapies, particularly for conditions that may require long-term or repeated treatment. According to Cloudbreak Pharma, CBT-004 demonstrated a strong safety profile throughout the Phase 2 trial. No treatment-related adverse events were reported among participants. The majority of side effects observed were mild to moderate in intensity, and no clinically meaningful changes were detected in either visual acuity or intraocular pressure—two areas of concern commonly associated with corticosteroid use. These findings reinforce CBT-004’s position as a non-steroidal, preservative-free option with a favorable risk-benefit profile.

What do experts say about CBT-004?

Prominent investigators involved in the trial have offered strong endorsements for the drug’s potential. “There is a significant unmet need for patients suffering from symptomatic pinguecula, as current therapies are largely off-label and may carry safety concerns with long-term use,” said Dr. John Hovanesian, Clinical Professor of Ophthalmology and recognized expert in anterior segment disease.

“The results from this trial are exciting, as they demonstrate that a targeted, non-steroidal therapy can meaningfully improve both the clinical signs and symptoms that impact patients’ quality of life.”

Echoing that sentiment, Dr. Sherif El-Harazi, Medical Director at Global Research Management and principal investigator in the study, remarked: “I was impressed by the consistency and magnitude of improvement in both objective redness and patient-reported discomfort with CBT-004. The safety profile was excellent, and I believe this therapy could represent a meaningful advance for our patients with vascularized pinguecula.”

What’s next for CBT-004?

While Cloudbreak Pharma has not yet provided a detailed timeline for next steps, the successful completion of this Phase 2 trial positions CBT-004 to potentially move into Phase 3 development, pending regulatory feedback. Further studies would aim to validate the efficacy and safety findings in a larger, more diverse patient population.

The company has indicated that it remains committed to addressing underserved ocular surface conditions through the development of targeted, preservative-free therapies.

How does this fit into broader ophthalmology trends?

The announcement comes amid growing industry focus on ocular surface diseases, where inflammation and chronic irritation are often under-addressed. New entrants in the field—particularly those developing non-steroidal and preservative-free solutions—are gaining attention from both clinicians and investors.

In recent years, companies like Aldeyra Therapeutics and Oyster Point Pharma (acquired by Viatris) have targeted similar inflammatory ocular diseases with novel mechanisms of action. Cloudbreak Pharma’s success with CBT-004 adds to the momentum and could place it in a favorable position for future partnerships or acquisitions, should Phase 3 results mirror current findings.

Could CBT-004 reshape treatment standards for vascularized pinguecula?

As vascularized pinguecula becomes increasingly recognized for its quality-of-life burden and lack of safe, targeted therapies, the emergence of CBT-004 marks a potentially pivotal advancement in ophthalmic care. For years, clinicians have had to rely on corticosteroids, lubricating drops, or even surgical excision—none of which offer an ideal combination of long-term safety, symptom relief, and efficacy. The Phase 2 results from Cloudbreak Pharma suggest that CBT-004 could alter that dynamic by introducing a well-tolerated, non-steroidal, and preservative-free therapy specifically designed to reduce both objective signs of inflammation and the subjective discomfort experienced by patients.

Should future trials confirm these findings in larger and more diverse patient populations, CBT-004 could establish a new clinical standard for treating this underdiagnosed and undertreated condition. Beyond pinguecula, it may also open pathways for Cloudbreak to explore its use in related inflammatory ocular conditions, where similar pathophysiological mechanisms exist. The therapeutic approach aligns with the broader shift in ophthalmology toward targeted, surface-friendly treatments that minimize systemic exposure and long-term side effects.

In a space where innovation has lagged behind other specialties, CBT-004 offers a rare combination of early efficacy signals, tolerability, and strategic potential. With Phase 3 trials likely on the horizon, stakeholders across the ophthalmology ecosystem—including physicians, regulatory bodies, and potential commercial partners—will be closely watching how this novel compound performs as it advances through the clinical pipeline.