Melt Pharmaceuticals Announces Dosing of Last Patient in Pivotal Phase 3 Study of Its Lead Product Candidate, MELT-300, for Needle- and Opioid-Free Sedation in Patients Undergoing Cataract Surgery

Rhea-AI Summary

Melt Pharmaceuticals has announced the completion of patient dosing in its pivotal Phase 3 study for MELT-300, a non-IV, non-opioid tablet for procedural sedation during cataract surgery. The study, conducted at 13 clinical sites with over 525 patients, compares MELT-300 to sublingual midazolam and placebo. Topline results are expected by the end of 2024.

The FDA has agreed to a Special Protocol Assessment (SPA) for the MELT-300 Phase 3 study, establishing a binding agreement on key elements for a future marketing application. Additionally, a cardiac safety study showed MELT-300 does not alter normal heart rhythm.

MELT-300 combines fixed doses of midazolam (3mg) and ketamine (50mg) in a sublingual tablet using Catalent's Zydis® delivery technology. Previous Phase 2 results showed MELT-300 was statistically superior for procedural sedation compared to placebo, midazolam, and ketamine individually.

Positive

• Completion of patient dosing in pivotal Phase 3 study for MELT-300
• FDA agreement on Special Protocol Assessment (SPA) for MELT-300 Phase 3 study
• Cardiac safety study showed MELT-300 does not alter normal heart rhythm
• Phase 2 results demonstrated statistical superiority of MELT-300 for procedural sedation
• Potential market of over 5 million annual cataract surgery patients in the U.S.
• Possibility for label expansion to over 100 million annual procedures in various medical fields

Negative

• Topline results of Phase 3 study not yet available
• Final FDA approval for MELT-300 still pending

Insights

Medical Research Analyst

The completion of patient dosing in Melt Pharmaceuticals' pivotal Phase 3 study for MELT-300 marks a significant milestone in the development of needle- and opioid-free sedation for cataract surgery. Key points to consider:

• The study enrolled over 525 patients across 13 clinical sites, demonstrating a robust sample size.
• The FDA's agreement on a Special Protocol Assessment (SPA) provides regulatory clarity and confidence in the study design.
• Topline results are expected in Q4 2024, which could be a catalyst for the stock if positive.
• MELT-300 showed statistical superiority in Phase 2 trials compared to placebo, midazolam and ketamine alone.
• The potential market is substantial, with an estimated 5 million annual cataract surgeries in the U.S.

If successful, MELT-300 could revolutionize procedural sedation, potentially expanding to over 100 million annual procedures across various medical fields. This represents a significant market opportunity for Melt Pharmaceuticals and its parent company, Harrow Health.

Financial Analyst

The advancement of MELT-300 to late-stage clinical trials represents a significant value driver for Harrow Health (HROW), which owns approximately 44% of Melt Pharmaceuticals. Key financial implications include:

• Reduced development risk with FDA's SPA agreement, potentially increasing the asset's value.
• Near-term catalyst with topline results expected in Q4 2024, which could impact HROW's stock price.
• Large addressable market of 5 million annual cataract surgeries in the U.S., with potential expansion to 100 million procedures across other fields.
• Possible reduction in healthcare costs by eliminating the need for IV sedation and associated complications.

If MELT-300 succeeds, it could significantly boost HROW's valuation, considering the company's substantial ownership stake. Investors should monitor the upcoming Phase 3 results closely, as positive outcomes could lead to a re-rating of HROW's stock, reflecting the increased probability of regulatory approval and commercial success.

Topline Readout Expected in Q4 2024

FDA Agrees to a MELT-300 Phase 3 Special Protocol Assessment

NASHVILLE, Tenn.--(BUSINESS WIRE)-- Melt Pharmaceuticals, Inc. (“Melt”), a clinical-stage pharmaceutical company developing novel approaches for procedural sedation, today announced the dosing of the last patient in its pivotal Phase 3 study evaluating the safety and efficacy of its lead product candidate, MELT‑300, a non-IV, non-opioid tablet for procedural sedation during cataract surgery.

MELT-300 combines fixed doses of midazolam (3mg) and ketamine (50mg) in one tablet that is administered sublingually using Catalent’s proprietary Zydis® delivery technology to dissolve and absorb the active ingredients across the sublingual mucosa rapidly. The MELT-300 Phase 3 clinical trial is a randomized, double-blind, three-arm study comparing, at a 4:1:1 ratio, MELT-300, sublingual midazolam, and sublingual placebo, respectively, for procedural sedation. The study was conducted at 13 clinical sites and enrolled over 525 patients. The topline readout from this study is expected before the end of 2024.

Additionally, Melt today announced that it had reached an agreement with the U.S. Food and Drug Administration (“FDA”) on a Special Protocol Assessment (“SPA”) for the MELT-300 Phase 3 study. FDA agreed the study would “adequately address the objectives necessary to support a regulatory submission.” The SPA agreement establishes a binding agreement on key elements to support a future marketing application. Further, based on its review of results from a recently completed cardiac safety study evaluating the potential risk for a serious drug-induced irregular heart rhythm, the FDA agreed that MELT-300 did not alter normal heart rhythm. A final decision regarding marketing approval will be based on the FDA’s review of the full MELT-300 submission package.

Melt previously announced its MELT-300 Phase 2 clinical trial results in patients undergoing cataract surgery, which compared MELT-300 against (i) sublingual placebo alone, (ii) sublingual midazolam, and (iii) sublingual ketamine in over 300 patients. MELT-300 was statistically superior for procedural sedation compared to all individual comparator arms: (i) sublingual placebo (P<0.0001), (ii) sublingual midazolam (P=0.0129), and (iii) sublingual ketamine (P=0.0096).

In commenting on the announcement, Dr. Larry Dillaha, Chief Executive Officer of Melt, said, “Completing the enrollment in our Phase 3 study and receiving the FDA’s agreement on our SPA are key milestones in the development of MELT-300 as we pursue our mission to provide needle- and opioid-free procedural sedation. We are extremely grateful to the investigators and patients who participated in this pivotal study and are excited to learn and share the findings of the Phase 3 study in the coming weeks.”

Dr. Dillaha added, “To our knowledge, this is the first constructive attempt to advance the standard of care of procedural sedation for cataract surgery patients, affecting the more than 5 million patients estimated to undergo this procedure annually in the U.S. in the coming years. Additionally, we believe success in the MELT-300 Phase 3 study should fuel further development and label expansion, with the potential of allowing MELT-300 to be utilized for procedural sedation in over 100 million annual procedures in areas such as dermatology, plastics, dental, gastrointestinal, and emergency rooms.”

About Melt Pharmaceuticals

Melt Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company focused on developing proprietary non-IV, non-opioid, sedation, and analgesia therapeutics for human medical procedures in the hospital, outpatient, and in-office settings. Melt intends to seek regulatory approval through the FDA’s 505(b)(2) regulatory pathway for its proprietary, patented small-molecule product candidates, where possible. Melt’s core intellectual property is the subject of multiple granted patents in North America, Europe, Asia, and the Middle East. Melt Pharmaceuticals, Inc. is a former subsidiary of Harrow, Inc. (Nasdaq: HROW) and was carved out as a separately managed business in 2019. To learn more about Melt, please visit their website, www.meltpharma.com.

Forward-Looking Statements

This press release contains forward-looking statements, which are all statements other than those statements of historical facts. No representation or warranty is made as to such statements, all of which involve substantial risks and uncertainties. Actual results could vary materially.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241010858192/en/

Investor Contact:

Larry Dillaha, M.D.

Chief Executive Officer

ldillaha@meltpharma.com

Media Contact:

Deb Holliday

Holliday Communications, Inc.

deb@hollidaycommunications.net

412-877-4519

Source: Harrow, Inc.

FAQ

What is MELT-300 and what is it used for?

MELT-300 is a non-IV, non-opioid tablet developed by Melt Pharmaceuticals for needle- and opioid-free procedural sedation, initially targeting patients undergoing cataract surgery.

When are the topline results of MELT-300's Phase 3 study expected?

The topline results of MELT-300's Phase 3 study are expected to be released before the end of 2024.

What is the significance of the FDA's Special Protocol Assessment (SPA) for MELT-300?

The FDA's SPA agreement establishes a binding agreement on key elements to support a future marketing application for MELT-300, indicating that the study design adequately addresses objectives necessary for regulatory submission.

How many patients were enrolled in MELT-300's Phase 3 study?

The Phase 3 study for MELT-300 enrolled over 525 patients across 13 clinical sites.

What were the results of MELT-300's cardiac safety study?

The cardiac safety study showed that MELT-300 did not alter normal heart rhythm, addressing potential concerns about drug-induced irregular heart rhythms.