- Solid clinical and operational execution demonstrated with advancement of Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) with commencement of pivotal study (TransportNPC™) and start of commercial-scale manufacturing

- Progression of Phase 2 study for Alzheimer’s Disease asset towards investigational new drug application (IND) filing remains on track for H2 2021

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 of 2021, and provided a business update.

“Our second quarter was marked by solid execution across multiple fronts. On the heels on positive topline results from our Phase 1/2 study in NPC and the recently announced positive long-term safety and efficacy from our Phase 1 open-label extension study, we have continued to build on that momentum and initiated our pivotal Phase 3 study in NPC as well as commenced commercial-scale manufacturing for Trappsol® Cyclo™. Additionally, with the positive feedback from the FDA supporting our strategy moving forward with our Alzheimer’s Disease program, we continue to advance towards multiple catalytic milestones ahead. I am incredibly pleased with our team and our progress and believe we are poised for an exciting remainder of the year and beyond,” commented N. Scott Fine, CEO of Cyclo Therapeutics.

Recent Highlights

  • Announced new positive safety and efficacy data from ongoing Phase 1 open-label extension study of Trappsol® Cyclo™ for the treatment of NPC;
  • Commenced the commercial-scale production of batches of Trappsol® Cyclo™ which will be used to support ongoing TransportNPC™ study in NPC1, the upcoming Phase 2 study in Alzheimer’s Disease, and the Company’s active compassionate use program;
  • Commenced site activation and patient enrollment for the TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of NPC1;
  • Appointed Lori McKenna Gorski as Global Head of Patient Advocacy. Ms. Gorski is a leading biotechnology executive with more than 20 years of experience specializing in patient advocacy for rare disease communities, including lysosomal disorders; and
  • Received feedback from the FDA supporting the Company’s development strategy to submit an IND application for a Phase 2 study of Trappsol® Cyclo™ in the treatment of early Alzheimer’s Disease, following a positive Type B interaction.

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 pivotal Phase 3 study 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.

The Company recently 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.

Additionally, Cyclo Therapeutics recently presented new long-term safety and efficacy data from its ongoing Phase 1 open-label extension study evaluating Trappsol® Cyclo™ for the treatment of NPC. The 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.

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. As part of a Type B interaction with the FDA, Cyclo Therapeutics received positive feedback supporting the Company’s development strategy to submit an IND application for a Phase 2 study of intravenous Trappsol® Cyclo™ in the treatment of early Alzheimer’s disease. The Company is on track to file its IND for a Phase 2 study of Alzheimer’s disease before year-end 2021.

Expected Upcoming Milestones

  • Target filing IND in H2 2021 for potential Phase 2 study evaluating Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease.

Summary of Financial Results for Second Quarter 2021

Net loss for the quarter ended June 30, 2021 was approximately $3.6 million. Research and development expenses increased 54% to $2.6 million for the three months ended June 30, 2021, from $1.7 million for the three months ended June 30, 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 2021 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 $11.4 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.

-- Financial Tables Follow--

CONSOLIDATED BALANCE SHEETS

 

 

June 30, 2021

 

December 31, 2020

 

 

(Unaudited)

 

 

 

ASSETS

CURRENT ASSETS

 

 

 

 

 

 

Cash and cash equivalents

 

$

11,463,098

 

 

$

12,846,113

 

Accounts receivable

 

 

182,431

 

 

 

71,017

 

Inventory, net

 

 

268,930

 

 

 

237,909

 

Current portion of mortgage note receivable

 

 

42,053

 

 

 

40,772

 

Prepaid insurance and services

 

 

172,191

 

 

 

126,474

 

Prepaid clinical expenses

 

 

1,991,866

 

 

 

727,952

 

Total current assets

 

 

14,120,569

 

 

 

14,050,237

 

 

 

 

 

 

 

 

 

 

FURNITURE AND EQUIPMENT, NET

 

 

67,997

 

 

 

53,910

 

 

 

 

 

 

 

 

 

 

RIGHT-TO-USE LEASE ASSET, NET

 

 

26,453

 

 

 

34,011

 

 

 

 

 

 

 

 

 

 

MORTGAGE NOTE RECEIVABLE, LESS CURRENT PORTION

 

 

28,356

 

 

 

49,806

 

 

 

 

 

 

 

 

 

 

TOTAL ASSETS

 

$

14,243,375

 

 

$

14,187,964

 

 

 

 

 

 

 

 

 

 

LIABILITIES AND STOCKHOLDERS’ EQUITY

 

 

 

 

 

 

 

 

 

CURRENT LIABILITIES

 

 

 

 

 

 

 

 

Current portion of lease liability

 

$

18,014

 

 

$

17,483

 

Current portion of note payable

 

 

86,884

 

 

 

114,029

 

Accounts payable and accrued expenses

 

 

2,930,267

 

 

 

3,541,041

 

Total current liabilities

 

 

3,035,165

 

 

 

3,672,553

 

 

 

 

 

 

 

 

 

 

LONG-TERM LEASE LIABILITY

 

 

 

 

 

 

 

 

Long-term lease liability, less current portion

 

 

10,392

 

 

 

18,434

 

Long-term note payable, less current portion

 

 

71,640

 

 

 

44,495

 

Total long-term liabilities

 

 

82,032

 

 

 

62,929

 

 

 

 

 

 

 

 

 

 

STOCKHOLDERS' EQUITY

 

 

 

 

 

 

 

 

Common stock, par value $.0001 per share, 20,000,000 shares authorized, 6,437,881 and 4,770,761 shares issued and outstanding, at June 30, 2021 and December 31, 2020, respectively

 

 

644

 

 

 

477

 

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

 

 

-

-

 

Additional paid-in capital

 

 

52,821,383

 

 

 

44,513,841

 

Accumulated deficit

 

 

(41,695,849

)

 

 

(34,061,836

)

Total stockholders' equity

 

 

11,126,178

 

 

10,452,482

 

 

 

 

 

 

 

 

 

 

TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY

 

$

14,243,375

 

 

$

14,187,964

 

CONSOLIDATED STATEMENTS OF OPERATIONS

(Unaudited)

 

 

Three Months Ended

 

Six Months Ended

 

 

June 30,

 

June 30,

 

 

 

2021

 

2020

 

2021

 

2020

 

 

 

 

 

 

 

 

 

 

 

 

 

REVENUES

 

 

 

 

 

 

 

 

 

 

 

 

Product sales

 

$

238,590

 

 

$

209,594

 

 

$

596,723

 

 

$

535,328

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

EXPENSES

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Personnel

 

 

542,192

 

 

 

433,628

 

 

 

1,101,516

 

 

 

903,333

 

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

 

 

30,158

 

 

 

12,947

 

 

 

64,754

 

 

 

39,380

 

Research and development

 

 

2,643,544

 

 

 

1,713,435

 

 

 

5,901,659

 

 

 

3,773,041

 

Repairs and maintenance

 

 

1,647

 

 

 

1,311

 

 

 

3,313

 

 

 

3,113

 

Professional fees

 

 

373,618

 

 

 

143,427

 

 

 

596,489

 

 

 

362,963

 

Office and other

 

 

241,739

 

 

 

79,823

 

 

 

555,513

 

 

 

258,185

 

Board of Director fees and costs

 

 

-

 

 

 

21,367

 

 

 

-

 

 

 

28,716

 

Depreciation

 

 

4,863

 

 

 

3,118

 

 

 

8,413

 

 

 

6,236

 

Freight and shipping

 

 

1,281

 

 

 

1,169

 

 

 

2,794

 

 

 

3,032

 

Bad debt expense

 

 

-

 

 

 

1,272

 

 

 

-

 

 

 

1,272

 

Total operating expenses

 

 

3,839,042

 

 

2,411,497

 

 

8,234,451

 

 

 

5,379,271

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

LOSS FROM OPERATIONS

 

 

(3,600,452

)

 

 

(2,201,903

)

 

 

(7,637,728

)

 

 

(4,843,943

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

OTHER INCOME

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Investment and other income

 

 

3,054

 

 

 

9,511

 

 

 

3,715

 

 

 

17,559

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

LOSS BEFORE INCOME TAXES

 

 

(3,597,398

)

 

 

(2,192,392

)

 

 

(7,634,013

)

 

 

(4,826,384

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

PROVISION FOR INCOME TAXES

 

 

-

 

 

 

-

 

 

 

-

 

 

 

-

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

NET LOSS

 

$

(3,597,398

)

 

$

(2,192,392

)

 

$

(7,634,013

)

 

$

(4,826,384

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

BASIC AND DILUTED NET LOSS PER COMMON SHARE

 

$

(.56

)

 

$

(1.61

)

 

$

(1.31

)

 

$

(3.74

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

WEIGHTED AVERAGE NUMBER OF COMMON SHARES OUTSTANDING

 

 

6,368,025

 

 

 

1,366,761

 

 

 

5,842,100

 

 

 

1,291,205

 

 

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

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