Cyclo Therapeutics reports promising NPC1 treatment findings

Published 07/02/2025, 14:10
Cyclo Therapeutics reports promising NPC1 treatment findings

GAINESVILLE, Fla. - Cyclo Therapeutics, Inc. (NASDAQ:CYTH), a clinical-stage biotechnology firm with a market capitalization of $22.15 million, announced positive data from a sub-study of its pivotal Phase 3 TransportNPC™ trial, evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1). The findings were presented at the 21st Annual WORLDSymposium™ in San Diego, California. According to InvestingPro analysis, the company maintains impressive gross profit margins of 91.24%, though it’s currently in a phase of rapid cash consumption typical for clinical-stage biotech companies.

The sub-study focused on patients from newborns to three years old, with results showing 87% of participants at 24 weeks and 86% at 48 weeks demonstrating stabilization or improvement on the Clinical Global Impression – Change (CGI-C) Scale. The TransportNPC™ study is considered the most comprehensive of its kind concerning patient size, global reach, duration, and clinical outcomes for NPC1 treatment. The stock has shown resilience with a 30.51% year-to-date return, despite broader market volatility, demonstrated by its negative beta of -0.49.

NPC1 is a rare genetic disorder characterized by the abnormal accumulation of cholesterol in cells, leading to organ dysfunction and early death. The sub-study aims to assess Trappsol® Cyclo™, particularly in young patients, as early intervention may have a preventative effect on symptom development.

The adverse effect profile of the treatment aligns with previous studies, with most reported adverse effects being mild or moderate. Only one mild adverse event was considered possibly related to the study drug, and no serious adverse events were deemed related to it.

Cyclo Therapeutics has completed enrollment for the TransportNPC™ study in May 2024 and expects to release top-line data from a 48-week interim analysis in the first half of 2025. If the interim data indicates statistical significance, the company plans to submit marketing applications based on the 48-week data. InvestingPro subscribers can access 8 additional key insights about CYTH’s financial health, valuation metrics, and growth prospects, helping investors make more informed decisions about this emerging biotech company.

The company’s CEO, N. Scott Fine, expressed optimism about the data and its implications for addressing NPC1. The ongoing TransportNPC™ study is a randomized, double-blind, placebo-controlled trial designed to assess the efficacy and safety of Trappsol® Cyclo™.

Cyclo Therapeutics has been granted Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC1 in the U.S. and EU, along with Fast Track and Rare Pediatric Disease Designations in the U.S.

This report is based on a press release statement from Cyclo Therapeutics.

In other recent news, biopharmaceutical firm Cyclo Therapeutics has made several key financial moves. The company extended its merger agreement with Rafael Holdings, pushing the completion deadline from February 15, 2025, to March 31, 2025. The amendment gives the Securities and Exchange Commission (SEC) more time to approve the registration statement filed by Rafael. As part of the merger, Cyclo Therapeutics also revised its Note Purchase Agreement with Rafael, issuing a $2 million convertible promissory note. This note is part of a series of financial arrangements between the two companies since June 2024, totaling $16 million.

Cyclo Therapeutics also faced a potential delisting from the Nasdaq due to non-compliance with certain listing rules. The company did not conduct its annual shareholder meeting within twelve months following the end of its fiscal year. To regain compliance, Cyclo Therapeutics is required to submit a plan by February 24, 2025.

In addition to these developments, Cyclo Therapeutics secured a $3 million convertible promissory note with Rafael Holdings. The note, due on February 15, 2025, carries an interest rate of 5% per annum. The company plans to use the proceeds from the note for working capital and general corporate purposes. These recent developments underline the ongoing relationship between Cyclo Therapeutics and Rafael Holdings as they navigate towards the completion of their merger.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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