Palatin Technologies, Inc. announced the FDA has confirmed acceptance of the protocols and endpoints relating to the signs and symptoms of its dry eye disease (DED)-targeted investigational candidate for the company's two remaining phase 3 clinical trials.
First up: Palatin.
The Cranbury, New Jersey-based biopharmaceutical company is developing first-in-class medicines based on molecules that regulate melanocortin receptor (MCr) system activity.
Its pipeline: Therapeutics for inflammation and autoimmune conditions, such as DED and retinal diseases.
- Note: Palatin received FDA approval in 2019 for Vyleesi (bremelanotide injection), the first-ever melanocortin-based commercial product for premenopausal women suffering from hypoactive sexual desire disorder (HSDD).
Why focus on regulating MCr activity?
The MCr system is involved in and impacts a number of biological functions, including inflammation, immune system responses, metabolism, steroid hormone production, and sexual function.
As such, regulating these MCrs is reported to have medically significant pharmacological effects, according to Palatin.
And how does this relate to ocular diseases?
Palatin noted that MCrs exist on the surface of various cells within the eye as well as in immune cells located within ocular tissue. Specifically: “These melanocortin receptors are a highly tractable target for melanocortin agonists to provide relief and healing for those living with ocular diseases,” the company stated.
Gotcha. Now discuss this DED candidate.
PL9643 is Palatin’s most advanced ocular therapeutic.
Administered via topical application, the formulation has previously demonstrated excellent ocular tolerability as well as statistically significant improvement in multiple signs and symptoms among moderate-to-severe DED patients.
Tell me more about its clinical data history.
A multicenter, randomized, double-masked, placebo-controlled phase 2 trial (NCT04268069) evaluating PL9643 failed to meet its primary endpoints and did not reach statistical significance in the overall enrolled patient population (measured at 12 weeks).
Read more about that December 2020 announcement.
And more recently?
In March 2024, the company released data from its pivotal phase 3 MELODY-1 trial (NCT05201170), which assessed the safety and efficacy of PL9643.
Those findings:
- The good: PL9643 was well tolerated and demonstrated clinically meaningful and statistically significant data for ocular pain (p < 0.025) and other system endpoints.
- Visual analog score reduction of > 10 points from baseline
- The bad: No statistical significance was reached for ocular pain (a primary endpoint) and secondary endpoints—as well as for all endpoints in the unadjusted planned analyses
- Note, all three primary endpoints included:
- Inferior corneal fluorescein staining
- Ocular discomfort
- Conjunctival sum lissamine green staining
- Note, all three primary endpoints included:
What does the company have to say about PL9643’s performance thus far?
Current prescription DED treatments have low treatment efficacy, slow onset of action, and poor tolerability, according to President and CEO Carl Spana, PhD.
He noted that PL9643’s early onset of efficacy for both DED signs and symptoms—plus its “excellent ocular safety and tolerability profile”—supports its potential “as a highly differentiated product with an opportunity to bring relief to millions of dry eye sufferers.”
Alrighty, now what’s this latest update?
Essentially: During a Type C meeting, Palatin and the FDA agreed on a regulatory path forward to support the company submitting a new drug application (NDA) for the DED candidate.
What this involves: Both protocols and signs / symptoms endpoints for the remaining phase 3 clinical program trials:
- MELODY-2
- MELODY-3
Note: Both trials are not yet listed by Clinical Trials.
What’s the plan for these trials?
Palatin plans to initiate participant enrollment in both MELODY-2 and MELODY-3 by Q4 2024.
- The target: To release topline data by “year-end calendar 2025.”
And the tentative timeline for that NDA?
Providing all goes well with the phase 3 trials—with recruitment goals being met and successful results—the company anticipates submitting its NDA by H1 2026.