- Lead development program evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) in ongoing pivotal study (TransportNPC™) continues to progress

- Company advancing Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease into Phase 2 study

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 full year 2021 and provided a business update.

“Over the course of the past year, we have made significant progress across our clinical programs. We are pleased by the continued advancement of our ongoing pivotal Phase 3 study evaluating Trappsol® Cyclo™ as a potential treatment for NPC, TransportNPC™. With the added expertise in our Global Steering Committee, to help guide this important study, we are encouraged and remain focused on driving it to completion with the potential to provide the first U.S. FDA approved therapy for NPC,” commented N. Scott Fine, CEO of Cyclo Therapeutics. “Additionally, with the clearance of our IND from the FDA, we are working towards launching our Phase 2 study for our Alzheimer’s Disease asset. 2022 is poised to be an exciting year as we execute on the milestones ahead and continue to build momentum.”

Recent Highlights

  • Announced the formation of Global Steering Committee (GSC) comprised of leading experts to advise on the global Phase 3 clinical development program for Trappsol® Cyclo™ in NPC;
  • Appointed Caroline Hastings, M.D. as Global Principal Investigator for ongoing TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of NPC;
  • Received clearance from the U.S. Food and Drug Administration (FDA) of its investigational new drug (IND) application to advance its Phase 2 Study of Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease;
  • Named preeminent neuroscientist and world-renowned researcher, Cynthia A. Lemere, PhD, as Senior Advisor for advancement of Alzheimer’s Disease asset.
  • Closed a $11.7 million underwritten public offering.

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

In June 2021, the Company commenced the TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of NPC1, a rare, progressive and fatal genetic disorder. Initial sites are in the U.S.

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 announced the formation of 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 will also chair 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. 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 expects to commence this Phase 2 study and enroll patients in 2022.

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.

Summary of Financial Results for Full Year 2021

Net loss for the year ended December 31, 2021 was approximately $14.3 million. Research and development expenses increased 50% to $9.2 million for the year ended December 31, 2021, from $6.1 million for the year ended December 31, 2020. The increase in research and development expense is due to increased activity in the Company’s international clinical program and U.S. clinical trials. The Company expects research and development costs 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 year with approximately $16.6 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.

CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED BALANCE SHEETS

 

 

December 31,

 

2021

 

2020

ASSETS

 

 

 

 

 

CURRENT ASSETS

 

 

 

 

Cash and cash equivalents

 

$

16,612,711

 

$

12,846,113

Accounts receivable, net

 

 

493,113

 

 

71,017

Inventory, net

 

 

227,437

 

 

237,909

Current portion of mortgage note receivable

 

 

45,977

 

 

40,772

Prepaid insurance and services

 

 

42,246

 

 

126,474

Prepaid clinical expenses

 

 

2,014,851

 

 

727,952

Total current assets

 

 

19,436,335

 

 

14,050,237

 

 

 

 

 

 

FURNITURE AND EQUIPMENT, NET

 

 

59,583

 

 

53,910

 

 

 

 

 

 

 

RIGHT-TO-USE LEASE ASSET, NET

 

 

17,636

 

 

34,011

 

 

 

 

 

 

MORTGAGE NOTE RECEIVABLE, LESS CURRENT PORTION

 

 

7,279

 

 

49,806

 

 

 

 

 

 

TOTAL ASSETS

 

$

19,520,833

 

$

14,187,964

 

 

 

 

 

 

 

LIABILITIES AND STOCKHOLDERS' EQUITY

 

 

 

 

 

 

 

CURRENT LIABILITIES

 

 

 

 

 

 

Current portion of lease liability

 

$

19,245

 

$

17,483

Current portion of note payable

 

 

133,712

 

 

114,029

Accounts payable and accrued expenses

 

 

3,677,979

 

 

3,541,041

Total current liabilities

 

 

3,830,936

 

 

3,672,553

 

 

 

 

 

 

 

LONG-TERM LIABILITIES

 

 

 

 

 

 

Long-term lease liability, less current portion

 

 

-

 

 

18,434

Long-term note payable, less current portion

 

 

18,034

 

 

44,495

Total long-term liabilities

 

 

18,034

 

 

62,929

 

 

 

 

 

 

 

STOCKHOLDERS' EQUITY

 

 

 

 

 

 

Common stock, par value $.0001 per share, 20,000,000 and 10,000,000

shares authorized, 8,403,869 and 4,770,761 shares issued and

outstanding on December 31, 2021 and 2020, respectively

 

 

841

 

 

477

Preferred stock, par value $.0001 per share, 5,000,000 shares authorized,

 

 

 

 

0 outstanding

-

-

Additional paid-in capital

 

 

64,019,513

 

 

44,513,841

Accumulated deficit

 

 

(48,348,491)

 

(34,061,836)

Total stockholders' equity

 

 

15,671,863

 

 

10,452,482

 

 

 

 

 

 

TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY

 

$

19,520,833

 

$

14,187,964

CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES

CONSOLIDATED STATEMENTS OF OPERATIONS

 

 

 

Year Ended December 31,

 

 

2021

 

2020

 

 

 

 

 

REVENUES

 

 

 

 

Product sales

 

$

1,585,756

 

 

$

903,376

 

 

 

 

 

 

 

EXPENSES

 

 

 

 

 

Personnel

 

 

3,838,477

 

 

 

2,561,710

 

Cost of products sold (exclusive of direct and indirect overhead and handling costs)

 

 

156,080

 

 

 

66,398

 

Research and development

 

 

9,153,780

 

 

 

6,096,445

 

Repairs and maintenance

 

 

10,708

 

 

 

7,188

 

Professional fees

 

 

1,474,911

 

 

 

536,876

 

Office and other

 

 

1,061,937

 

 

 

507,474

 

Board of Directors fees and costs

 

 

122,833

 

 

 

69,649

 

Depreciation

 

 

16,827

 

 

 

12,763

 

Freight and shipping

 

 

18,336

 

 

 

5,006

 

Bad debt expense

 

 

21,927

 

 

 

1,272

 

Total operating expenses

 

 

15,875,816

 

 

 

9,864,781

 

 

 

 

 

 

 

LOSS FROM OPERATIONS

 

 

(14,290,060

)

 

 

(8,961,405

)

 

 

 

 

 

 

 

OTHER INCOME

 

 

 

 

 

 

Investment and other income

 

 

3,405

 

 

 

19,802

 

 

 

 

 

 

 

LOSS BEFORE INCOME TAXES

 

 

(14,286,655

)

 

 

(8,941,603

)

 

 

 

 

 

 

PROVISION FOR INCOME TAXES

 

 

-

 

 

 

-

 

 

 

 

 

 

 

NET LOSS

 

$

(14,286,655

)

 

$

(8,941,603

)

 

 

 

 

 

 

BASIC AND DILUTED NET LOSS PER COMMON SHARE

 

$

(2.24

)

 

$

(5.60

)

 

 

 

 

 

 

WEIGHTED AVERAGE NUMBER OF COMMON SHARES OUTSTANDING

 

 

6,370,073

 

 

 

1,599,223

 

 

JTC Team, LLC Jenene Thomas (833) 475-8247 CYTH@jtcir.com

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