GAINESVILLE, Fla. - Cyclo Therapeutics, Inc. (NASDAQ:CYTH), a clinical-stage biotechnology company, has shared preliminary data from its ongoing TransportNPC™ study and substudy, which are focused on treating Niemann-Pick Disease Type C1 (NPC1). The findings were presented at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2024, held from September 3-6 in Porto, Portugal.
The TransportNPC™ study, completed in May 2024, is a global Phase 3 trial involving 104 patients and is notable for its breadth in patient size, duration, and clinical outcomes for NPC1. The substudy, part of the Pediatric Investigational Plan, targets the disease's visceral aspects in patients from birth to three years old.
Cyclo Therapeutics' CEO, N. Scott Fine, expressed optimism about the trial's progress and the potential for interim data readout expected in the first half of 2025. Professor Caroline Hastings, the Global Principal Investigator, highlighted the preliminary safety data and the treatment's potential impact on NPC symptoms.
The TransportNPC™ study's primary endpoint will compare changes in the 5D-NPC-CSS, which measures ambulation, fine motor skills, speech, swallowing, and cognition, between the investigational drug Trappsol® Cyclo™ and a placebo. As of June 30, 2024, the average participant had been in the study for 44 weeks, with six patients transitioning to open-label Trappsol® Cyclo™ due to disease progression.
Safety findings from the study indicate that most of the 625 reported adverse events were mild and related to the underlying NPC disease. The substudy involving the youngest patients also showed a safety profile consistent with previous studies, with no serious adverse events linked to the treatment.
The company plans to submit marketing applications for approval based on the 48-week interim data if it meets statistical significance. The substudy aims to provide insights into early intervention and prevention of disease progression, with results anticipated as the enrolled patients complete their respective treatment durations.
This article is based on a press release statement from Cyclo Therapeutics. The company is also conducting a Phase 2b clinical trial for early Alzheimer's disease using Trappsol® Cyclo™ intravenously, following promising data from an Expanded Access program. Cyclo Therapeutics is committed to developing treatments for rare and fatal genetic diseases, with several clinical trials currently underway.
In other recent news, Cyclo Therapeutics and Rafael Holdings have announced a definitive merger agreement, aiming to advance the development of Trappsol Cyclo for the treatment of Niemann-Pick disease type C1. The merger is expected to finalize in late 2024, pending shareholder approval. Cyclo Therapeutics shareholders are set to receive Class B common stock of Rafael Holdings, with the exchange ratio valuing Cyclo Therapeutics shares at $0.95 each.
Additionally, Cyclo Therapeutics has secured a $2 million convertible promissory note deal with Rafael Holdings, to be used for working capital and corporate purposes. The company also received approval from the European Patent Office for its Alzheimer's disease treatment method, set to take effect in 2024.
In the wake of these developments, Maxim (NASDAQ:MXIM) Group downgraded Cyclo Therapeutics stock from Buy to Hold, while Ascendiant Capital reaffirmed its Buy rating but reduced its price target. H.C. Wainwright also downgraded the stock to Neutral, aligning with the acquisition terms. These recent developments reflect the evolving landscape for Cyclo Therapeutics as it navigates its merger with Rafael Holdings and continues its research and development efforts.
InvestingPro Insights
As Cyclo Therapeutics (NASDAQ:CYTH) continues to make strides in its clinical trials, the financial health and market sentiment surrounding the company also play a crucial role in evaluating its prospects. According to recent metrics from InvestingPro, Cyclo Therapeutics has a market capitalization of $20.39 million, reflecting the company's valuation in the market. Despite facing challenges, the company boasts an impressive gross profit margin of 91.56% for the last twelve months as of Q2 2024, underscoring its ability to retain a significant portion of revenue after accounting for the cost of goods sold.
InvestingPro Tips suggest that while the stock price has experienced significant declines over the past week, month, and three months, with a total return of -7.7%, -32.96%, and -44.05% respectively, analysts predict the company will be profitable this year. This could indicate a potential turnaround for investors who have weathered recent volatility. Moreover, the stock is currently trading near its 52-week low, which could present a buying opportunity for those who believe in the company's long-term potential, especially as its net income is expected to grow.
For investors seeking more in-depth analysis, there are an additional 16 InvestingPro Tips available at https://www.investing.com/pro/CYTH, offering valuable insights into Cyclo Therapeutics' performance and market position. These tips provide a comprehensive look at various aspects of the company's financials, from cash flow concerns to market trends, helping investors make informed decisions.
As Cyclo Therapeutics advances its clinical programs and moves closer to potential marketing applications, keeping an eye on both the scientific progress and the financial metrics will be key for stakeholders following the company's journey.
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