– On track to commence Phase 2 study of Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease before year end
– Continued execution on advancement of lead development program evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) in ongoing global pivotal study (TransportNPC™)
GAINESVILLE, Fla.–(BUSINESS WIRE)–Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today reported its financial results for the second quarter 2022 and provided a business update.
“Our primary focus remains on advancing our clinical programs evaluating Trappsol® Cyclo™ for the treatment of NPC and Alzheimer’s disease. Of particular note over the past six months, we have spent dedicated time with the NPC patient advocacy organizations and have focused on building our presence in this community globally, which continues to be invaluable. We believe the engagement with the broader NPC community is integral to the continued successful execution of our pivotal program,” commented N. Scott Fine, CEO of Cyclo Therapeutics. “Additionally, we are making tremendous progress in gearing up for our Phase 2 Alzheimer’s disease study and are on track to enroll and dose patients before the end of this year. As we advance to the second half of the year, we are encouraged by the progress made thus far and look to continue driving value for all stakeholders in the near and long-term.”
Recent Highlights
- Presented at the 2022 China Nieman Pick Medical Exchange & Sixth Patient Association, a physician, researcher and patient focused event to discuss NPC and the latest research results for diagnosis and treatment;
- Attended the NNPDF-INPDA Conferences 2022 with Caroline Hastings, M.D., Key Opinion Leader in NPC and Global Principal Investigator for the ongoing TransportNPC™ study, to discuss Trappsol® Cyclo™ and its clinical development program for the treatment of NPC;
- Provided an overview of the Trappsol® Cyclo™ program for the treatment of NPC at the World Orphan Drug Congress USA 2022;
- Presented an update on the results from Phase 1 and Phase 1/2 studies and launch of the international Phase 3 pivotal TransportNPC™ trial at the 2022 NPC Patient and Family Conference hosted by the Australian NPC Disease Foundation;
- Continued engagement with investment community and industry partners at the BIO International Convention; and
- Continued progress toward commencement of Phase 2 study of Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease in 2022.
Trappsol® Cyclo™ Clinical Program Update
Trappsol® Cyclo™ is a proprietary formulation of hydroxypropyl beta cyclodextrin, used intravenously (IV) and currently in development for the treatment of NPC, a rare genetic disorder causing cholesterol accumulation in lysosomes of cells, organ dysfunction and premature death.
Niemann-Pick Disease Type C1 Development Program
The Company’s ongoing pivotal Phase 3 study, TransportNPC™, is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1. The Phase 3 study intends to enroll at least 93 pediatric (age 3 years and older) and adult patients with NPC1 in at least 23 study centers in 9 countries. Eligible patients will be randomized 2:1 to receive either Trappsol® Cyclo™ or a placebo. Randomization will not be constrained based on patient age, nor will patient enrollment be gated by patient age. The study duration is 96 weeks and includes an interim analysis at 48 weeks. Data seen to-date provide additional support for the capacity of Trappsol® Cyclo™ to stabilize disease progression with home-based intravenous infusions as well as for a favorable safety profile of more than two years in NPC.
The Company recently established a Global Steering Committee (GSC) to guide the pivotal Phase 3 global clinical development program of Trappsol® Cyclo™ for the treatment of NPC. As the Global Principal Investigator for the TransportNPC™ study, Caroline Hastings, MD serves as the senior scientific and clinical expert for the trial and chair of the GSC. Other members of the Companies GSC include leading experts and renowned Key Opinion Leaders.
Additionally, Cyclo Therapeutics received a positive opinion from the Paediatric Committee (PDCO) of the EMA and agreement on its Paediatric Investigation Plan (PIP) for Trappsol® Cyclo™. The PIP opinion from PDCO endorsed the clinical program to evaluate the safety, tolerability and efficacy of Trappsol® Cyclo™ in patients from 3 to less than 18 years of age with NPC in the randomized study, and in addition, to include a single-arm open-label sub-study of patients from birth to less than 3 years of age with NPC Type C1 irrespective of symptoms to evaluate safety and to obtain descriptive data on global disease severity and the response to Trappsol® Cyclo™. The sub-study in patients from birth to less than 3 years of age will only be conducted in the EU and countries following EMA guidelines.
For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.
Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.
Alzheimer’s Disease Asset
Cyclo Therapeutics is also planning to evaluate Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease, targeting the reduction of amyloid beta and tau. To assist in driving the development program forward, the Company recently appointed Cynthia A. Lemere, PhD, a renowned translational researcher focused on understanding, preventing, and treating Alzheimer’s disease, as the Senior Advisor for the Company’s Alzheimer’s Disease program. In December 2021, the Company received IND clearance from the U.S. FDA to advance its Phase 2 study of intravenous Trappsol® Cyclo™ for the treatment of early Alzheimer’s Disease. The Company is on track to commence this Phase 2 study and enroll patients this year.
Many of the known risk factors for Alzheimer’s disease are associated with cholesterol metabolism. Cholesterol imbalance in Alzheimer’s patients is well known, and significant research exists, suggesting these imbalances are responsible for amyloid beta (Aβ) and tau accumulation. Furthermore, neurons, because of their high metabolic demands, experience an increased level of oxidative stress. Oxidative stress has also been linked to abnormal cholesterol accumulation and processing.
Pipeline Expansion Opportunities
In March 2022, the Company entered into a research and collaboration agreement with the University of the Witwatersrand, Johannesburg (Wits) represented by Wits Commercial Enterprise (Pty) Ltd, a wholly owned subsidiary of the University whose mandate is to protect and manage the University’s intellectual property. Under the terms of the collaboration agreement, Wits and Cyclo Therapeutics plan to develop and coordinate projects or activities to further scientific advancement of the Company’s proprietary platform technology, Trappsol® Cyclo™.
The Collaboration consists of cooperative agreements in specific, undisclosed research and development, technology development and commercialization, and training projects, or any other activity agreed upon by Cyclo Therapeutics and Wits. Due to intellectual property development and for competitive reasons, Cyclo Therapeutics is not disclosing details related to the exploration of therapeutic areas or indications at this time.
Summary of Financial Results for Second Quarter 2022
Net loss for the quarter ended June 30, 2022 was approximately $3.5 million. Research and development expenses decreased 29% to approximately $1.9 million for the three months ended June 30, 2022, from $2.6 million for the three months ended June 30, 2021. The decrease in research and development expense in the more recent period was related to the timing of startup costs in our clinical programs in the prior year period. The Company expects research and development to further increase in 2022 as we continue to seek regulatory approval for the use of Trappsol® Cyclo™ in the treatment of NPC and Alzheimer’s disease.
The Company ended the quarter with approximately $7.5 million of cash.
About Cyclo Therapeutics
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering from disease. The Company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of four formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, (www.ClinicalTrials.gov, NCT02939547, NCT02912793, NCT03893071 and NCT04860960). The Company is planning an early phase clinical trial using Trappsol® Cyclo™ intravenously in Alzheimer’s Disease based on encouraging data from an Expanded Access program for late-onset Alzheimer’s Disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com.
Safe Harbor Statement
This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities, including, without limitation, statements regarding the satisfaction of closing conditions relating to the offering and the anticipated use of proceeds from the offering. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company’s future performance include the company’s ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company’s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.
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CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES CONSOLIDATED BALANCE SHEETS |
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|
|
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|
|
|
June 30, |
December 31, |
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|
|
|
(unaudited) |
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ASSETS |
|||||||
|
|
|
|
|||||
|
CURRENT ASSETS |
|
|
|||||
|
Cash and cash equivalents |
$ |
7,453,391 |
|
$ |
16,612,711 |
|
|
|
Accounts receivable, net |
|
409,932 |
|
|
493,113 |
|
|
|
Inventory, net |
|
260,613 |
|
|
227,437 |
|
|
|
Current portion of mortgage note receivable |
|
28,762 |
|
|
45,977 |
|
|
|
Prepaid insurance and services |
|
186,217 |
|
|
42,246 |
|
|
|
Prepaid clinical expenses |
|
3,438,121 |
|
|
2,014,851 |
|
|
|
Total current assets |
|
11,777,036 |
|
|
19,436,335 |
|
|
|
|
|
|
|
||||
|
FURNITURE AND EQUIPMENT, NET |
|
54,701 |
|
|
59,583 |
|
|
|
|
|
|
|
|
|||
|
RIGHT-TO-USE LEASE ASSET, NET |
|
10,078 |
|
|
17,636 |
|
|
|
|
|
|
|
||||
|
MORTGAGE NOTE RECEIVABLE, LESS CURRENT PORTION |
|
– |
|
|
7,279 |
|
|
|
|
|
|
|
||||
|
TOTAL ASSETS |
$ |
11,841,815 |
|
$ |
19,520,833 |
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|
|
|
|
|
|
|
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LIABILITIES AND STOCKHOLDERS’ EQUITY |
|||||||
|
|
|
|
|
|
|||
|
CURRENT LIABILITIES |
|
|
|
|
|||
|
Current portion of lease liability |
$ |
9,805 |
|
$ |
19,245 |
|
|
|
Current portion of note payable |
|
– |
|
|
133,712 |
|
|
|
Accounts payable and accrued expenses |
|
2,091,968 |
|
|
3,677,979 |
|
|
|
Total current liabilities |
|
2,101,773 |
|
|
3,830,936 |
|
|
|
|
|
|
|
|
|||
|
LONG-TERM LIABILITIES |
|
|
|
|
|||
|
Long-term note payable, less current portion |
|
– |
|
|
18,034 |
|
|
|
|
|
|
|
|
|||
|
Commitments and contingencies |
|
|
|
|
|||
|
|
|
|
|
|
|||
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STOCKHOLDERS’ EQUITY |
|
|
|
|
|||
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Common stock, par value $.0001 per share, 20,000,000 shares authorized, 8,439,435 and 8,403,869 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively |
|
844 |
|
|
841 |
|
|
|
Preferred stock, par value $.0001 per share, 5,000,000 shares authorized, 0 issued and outstanding |
|
– |
|
|
– |
|
|
|
Additional paid-in capital |
|
64,311,270 |
|
|
64,019,513 |
|
|
|
Accumulated deficit |
|
(54,572,072 |
) |
(48,348,491 |
) |
||
|
Total stockholders’ equity |
|
9,740,042 |
|
|
15,671,863 |
|
|
|
|
|
|
|
||||
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TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY |
$ |
11,841,815 |
|
$ |
19,520,833 |
|
|
|
CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) |
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|
|
Three Months Ended |
|
Six Months Ended |
||||||||||||
|
|
June 30, |
|
June 30, |
||||||||||||
|
|
2022 |
|
2021 |
|
2022 |
|
2021 |
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|
|
|
|
|
|||||||||||
|
REVENUES |
|
|
|
|
|||||||||||
|
Product sales |
$ |
541,886 |
|
$ |
238,590 |
|
$ |
736,790 |
|
$ |
596,723 |
|
|||
|
|
|
|
|
|
|
|
|
|
|||||||
|
EXPENSES |
|
|
|
|
|
|
|
|
|||||||
|
Personnel |
|
970,759 |
|
|
542,192 |
|
|
2,187,664 |
|
|
1,101,516 |
|
|||
|
Cost of products sold (exclusive of |
|
73,667 |
|
|
30,158 |
|
|
90,131 |
|
|
64,754 |
|
|||
|
Research and development |
|
1,874,383 |
|
|
2,643,544 |
|
|
2,958,435 |
|
|
5,901,659 |
|
|||
|
Repairs and maintenance |
|
3,675 |
|
|
1,647 |
|
|
7,998 |
|
|
3,313 |
|
|||
|
Professional fees |
|
649,842 |
|
|
373,618 |
|
|
1,061,897 |
|
|
596,489 |
|
|||
|
Office and other |
|
291,611 |
|
|
241,739 |
|
|
585,787 |
|
|
555,513 |
|
|||
|
Board of Director fees and costs |
|
124,131 |
|
|
– |
|
|
216,256 |
|
|
– |
|
|||
|
Depreciation |
|
4,741 |
|
|
4,863 |
|
|
9,482 |
|
|
8,413 |
|
|||
|
Freight and shipping |
|
5,101 |
|
|
1,281 |
|
|
9,621 |
|
|
2,794 |
|
|||
|
Total operating expenses |
|
3,997,910 |
|
|
3,839,042 |
|
|
7,127,271 |
|
|
8,234,451 |
|
|||
|
|
|
|
|
|
|
|
|
||||||||
|
LOSS FROM OPERATIONS |
|
(3,456,024 |
) |
|
(3,600,452 |
) |
|
(6,390,481 |
) |
|
(7,637,728 |
) |
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|
|
|
|
|
|
|
|
|
||||||||
|
OTHER INCOME |
|
|
|
|
|
|
|
||||||||
|
Investment and other income |
|
4,034 |
|
|
3,054 |
|
|
8,376 |
|
|
3,715 |
|
|||
|
Gain on forgiveness of PPP loan |
|
– |
|
|
– |
|
|
158,524 |
|
|
– |
|
|||
|
Total other income |
|
4,034 |
|
|
3,054 |
|
|
166,900 |
|
|
3,715 |
|
|||
|
|
|
|
|
|
|
|
|
|
|||||||
|
LOSS BEFORE INCOME TAXES |
|
(3,451,990 |
) |
|
(3,597,398 |
) |
|
(6,223,581 |
) |
|
(7,634,013 |
) |
|||
|
|
|
|
|
|
|
|
|
||||||||
|
PROVISION FOR INCOME TAXES |
|
– |
|
|
– |
|
|
– |
|
|
– |
|
|||
|
|
|
|
|
|
|
|
|
||||||||
|
NET LOSS |
$ |
(3,451,990 |
) |
$ |
(3,597,398 |
) |
$ |
(6,223,581 |
) |
$ |
(7,634,013 |
) |
|||
|
|
|
|
|
|
|
|
|
||||||||
|
BASIC AND DILUTED NET LOSS PER |
$ |
(0.41 |
) |
$ |
(0.56 |
) |
$ |
(0.74 |
) |
$ |
(1.31 |
) |
|||
|
|
|
|
|
|
|
|
|
|
|||||||
|
WEIGHTED AVERAGE NUMBER OF COMMON |
|
8,424,003 |
|
|
6,368,025 |
|
|
8,417,901 |
|
|
5,842,100 |
|
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Contacts
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
CYTH@jtcir.com