FDA Confirms Acceptability of Palatin's Remaining Phase 3 Pivotal Clinical Trials for PL9643 in Dry Eye Disease (DED)

Rhea-AI Summary

Palatin Technologies (NYSE American: PTN) has received FDA confirmation for the protocols and endpoints of its remaining Phase 3 pivotal clinical trials for PL9643 in Dry Eye Disease (DED). The trials, MELODY-2 and MELODY-3, are set to begin patient enrollment in Q4 2024, with topline results expected by year-end 2025. If successful, an NDA submission is anticipated in H1 2026.

The company's first Phase 3 study, MELODY-1, completed in February 2024, showed promising results with rapid onset of efficacy and statistical significance in multiple symptom and sign endpoints. PL9643 demonstrated an excellent safety and tolerability profile, positioning it as a potentially highly differentiated product in the DED market, which affects an estimated 38 million adults in the US and 719 million globally.

Positive

• FDA confirmed acceptability of protocols and endpoints for PL9643 Phase 3 trials in DED
• MELODY-1 Phase 3 study showed statistical significance in co-primary symptom endpoint and 7 of 11 secondary symptom endpoints
• PL9643 demonstrated rapid onset of efficacy at 2-week timepoint
• PL9643 showed excellent safety and tolerability profile
• Clear regulatory path established for PL9643 NDA submission in DED

Negative

• Patient enrollment for MELODY-2 and MELODY-3 not expected to start until Q4 2024
• Topline results for remaining Phase 3 trials not expected until Q4 2025
• NDA submission anticipated for H1 2026, indicating a lengthy timeline to potential market approval

Insights

Medical Research Analyst

The FDA's acceptance of Palatin's Phase 3 trial protocols for PL9643 in Dry Eye Disease (DED) is a significant milestone. This approval paves the way for the MELODY-2 and MELODY-3 trials, with patient enrollment expected in Q4 2024 and topline results by end of 2025. The rapid onset of efficacy demonstrated in MELODY-1, with statistical significance achieved at just 2 weeks for multiple endpoints, is particularly noteworthy.

PL9643's potential as a fast-acting, well-tolerated treatment could address major shortcomings in current DED therapies. With an estimated 38 million adults affected in the US alone, the market potential is substantial. However, investors should note that the 2026 NDA submission timeline means revenue generation is still years away and success in the upcoming trials is important for Palatin's future in this space.

Financial Analyst

While the FDA's acceptance is positive, Palatin's financial position needs scrutiny. The extended timeline to potential commercialization - with an NDA submission not expected until 2026 - means continued R&D expenses without immediate revenue prospects from this product. Investors should examine Palatin's cash runway and potential need for additional financing to support these trials.

However, the large addressable market for DED treatments presents a significant opportunity if PL9643 succeeds. With many current treatments considered inadequate, a superior product could capture substantial market share. The stock may see increased interest as trial milestones approach, but volatility is likely given the binary nature of clinical trial outcomes. Prudent investors should consider Palatin's entire pipeline and financial health when evaluating its long-term potential.

Market Research Analyst

The DED market is ripe for disruption, with current treatments often falling short in efficacy or tolerability. PL9643's potential for rapid onset and dual action on signs and symptoms could position it as a preferred option if approved. The global prevalence of 757 million adults with DED represents a massive market opportunity.

However, the competitive landscape is evolving. Investors should monitor other emerging therapies and how they compare to PL9643 in efficacy, safety and convenience. The increasing prevalence of DED, partly due to digital screen use, suggests a growing market. Yet, market penetration will depend on factors like pricing, reimbursement and marketing strategies. Palatin's ability to secure strong partnerships for commercialization could be important for maximizing PL9643's potential market impact.

• FDA & Palatin agree on clear regulatory path for PL9643 NDA submission in DED
• FDA confirms acceptability of protocols and endpoints for remaining MELODY-2 & MELODY-3 Phase 3 Pivotal Clinical Trials in DED
  • Currently anticipate patient enrollment to start in 4Q calendar year 2024
  • Topline results currently expected in 4Q calendar year 2025

CRANBURY, N.J., Aug. 28, 2024 /PRNewswire/ -- Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor system, today announced receipt of FDA correspondence confirming the acceptability of the protocols and endpoints for signs and symptoms of PL9643 Phase 3 pivotal clinical trials for dry eye disease (DED). The remaining Phase 3 clinical trial program consists of two Phase 3 studies, MELODY-2 and MELODY-3, and include sign and symptom endpoints in each study. 

"We are pleased to have concluded a positive Type C meeting with the FDA and agreement on the remaining two Phase 3 trial protocols, including the sign and symptom endpoints, for PL9643 for the treatment of patients with DED," said Carl Spana, Ph.D., President and Chief Executive Officer of Palatin. "Current prescription options for DED are generally regarded as inadequate by many eye care professionals and patients due to low treatment efficacy, slow onset of action and/or poor tolerability. PL9643, with its early onset of efficacy for both signs and symptoms, and its excellent ocular safety and tolerability profile, is positioned as a highly differentiated product with an opportunity to bring relief to millions of dry eye sufferers."

Patient enrollment in MELODY-2 and MELODY-3 are currently expected to begin in the fourth quarter of calendar year 2024, resulting in topline results available by year-end calendar 2025. If recruitment goals are met and the trial results are successful, an NDA submission in the first half of calendar year 2026 would be anticipated.

Palatin successfully completed MELODY-1, its first Phase 3 study, in February 2024. The co-primary symptom endpoint of pain met statistical significance (P<0.025) and 7 of 11 secondary symptom endpoints met statistical significance (P<0.05), at the 12-week treatment period. Data from MELODY-1 demonstrated a rapid onset of efficacy and multiple symptom endpoints, including the co-primary pain endpoint, which met statistical significance (P<0.05) at the 2-week timepoint and continued to improve over the 12-week treatment period. Also, at the 2-week timepoint, multiple sign endpoints, including all 4 fluorescein staining endpoints, met statistical significance (P<0.05). Importantly, PL9643 treatment demonstrated an excellent safety and tolerability profile.

About Dry Eye Disease (DED)
Dry eye disease is a common inflammatory disease that, left untreated, can become extremely painful and lead to permanent damage to the cornea and vision. DED affects the cornea and conjunctiva of the eye resulting in irritation, redness, pain, and blurred vision. The disease is characterized by insufficient moisture and lubrication in the anterior surface of the eye, leading to dryness, inflammation, pain, discomfort, irritation, diminished quality of life, and in severe cases, permanent vision impairment. Existing therapy for DED is generally regarded as inadequate by many physicians and patients, and often requires months to demonstrate activity.

Dry eye disease is one of the most common ocular surface disorders, affecting an estimated 38 million adults in the United States and an estimated 719 million more adults globally. ¹While once considered a disease of aging populations, modern advancements, such as prolonged digital screen time, have contributed to a significant rise in DED across age and gender. ²Many existing prescription options for dry eye disease are generally regarded by many eye care professionals and patients as inadequate due to low treatment efficacy, slow onset of action and/or poor tolerability.³

About Melanocortin Receptor Agonists and Inflammation
The melanocortin receptor ("MCR") system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors, MCR1 through MCR5. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects. Many tissues and immune cells located in the eye (and other places, like the gut and kidney) express melanocortin receptors, empowering our opportunity to directly activate natural pathways to resolve disease inflammation.

About Palatin
Palatin is a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor systems, with targeted, receptor-specific product candidates for the treatment of diseases with significant unmet medical need and commercial potential. Palatin's strategy is to develop products and then form marketing collaborations with industry leaders to maximize their commercial potential. For additional information regarding Palatin, please visit Palatin's website at www.Palatin.com and follow Palatin on Twitter at @PalatinTech.

Forward-looking Statements
Statements in this press release that are not historical facts, including statements about future expectations of Palatin Technologies, Inc., such as statements about Palatin products in development, clinical trial results, potential actions by regulatory agencies including the FDA, regulatory plans, development programs, proposed indications for product candidates, and market potential for product candidates are "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and as that term is defined in the Private Securities Litigation Reform Act of 1995. Palatin intends that such forward-looking statements be subject to the safe harbors created thereby. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause Palatin's actual results to be materially different from its historical results or from any results expressed or implied by such forward-looking statements. Palatin's actual results may differ materially from those discussed in the forward-looking statements for reasons including, but not limited to, results of clinical trials, regulatory actions by the FDA and other regulatory and the need for regulatory approvals, Palatin's ability to fund development of its technology and establish and successfully complete clinical trials, the length of time and cost required to complete clinical trials and submit applications for regulatory approvals, products developed by competing pharmaceutical, biopharmaceutical and biotechnology companies, commercial acceptance of Palatin's products, and other factors discussed in Palatin's periodic filings with the Securities and Exchange Commission. Palatin is not responsible for updating events that occur after the date of this press release.

References

1. Market Scope 2023 Dry Eye Product Market Review; does not include OTC artificial tears and other Rx anti-inflammatory and tear stimulants.
2. The Relationship Between Dry Eye Disease and Digital Screen Use - PMC (nih.gov)
3. Improved Dry Eye Drugs for 2022 and Beyond; https://www.aao.org/eye-health/tips-prevention/new-dry-eye-treatments-ocular-surface-disease

Palatin Technologies^® is a registered trademark of Palatin Technologies, Inc.

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SOURCE Palatin Technologies, Inc.

FAQ

What is the current status of Palatin's PL9643 clinical trials for Dry Eye Disease?

Palatin has received FDA confirmation for the protocols and endpoints of its remaining Phase 3 pivotal clinical trials (MELODY-2 and MELODY-3) for PL9643 in Dry Eye Disease. Patient enrollment is expected to begin in Q4 2024, with topline results anticipated by year-end 2025.

What were the results of Palatin's MELODY-1 Phase 3 study for PL9643 in DED?

MELODY-1, completed in February 2024, showed statistical significance in the co-primary symptom endpoint of pain and 7 of 11 secondary symptom endpoints. The study demonstrated rapid onset of efficacy at the 2-week timepoint and an excellent safety and tolerability profile for PL9643.

When does Palatin (PTN) expect to submit an NDA for PL9643 in Dry Eye Disease?

If recruitment goals are met and trial results are successful, Palatin anticipates submitting an NDA for PL9643 in Dry Eye Disease in the first half of calendar year 2026.

How many adults are affected by Dry Eye Disease according to Palatin's press release?

According to the press release, Dry Eye Disease affects an estimated 38 million adults in the United States and an estimated 719 million more adults globally.