Continued progress in lead development program evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) in ongoing 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 third quarter of 2021, and provided a business update.
“We have executed on multiple fronts over the course of the third quarter. Our pivotal Phase 3 study evaluating Trappsol® Cyclo™ as a potential treatment for Niemann-Pick Disease, TransportNPC™, is now underway and we are pleased with the progress made thus far. Additionally, we’ve bolstered the leadership and expertise of our team with the appointments of our Chief Medical Officer and a Senior Advisor for the Alzheimer’s disease program, both of whom are world renowned, and we expect will add significant value as we look to advance Trappsol® Cyclo™ through the clinic and potentially to market,” commented N. Scott Fine, CEO of Cyclo Therapeutics. “Lastly, we continue to advance towards launching a Phase 2 study for our Alzheimer’s disease asset. We continue to execute on all fronts and are well-positioned for what we believe will be an exciting remainder of the year and beyond.”
Recent Highlights
- Bolstered executive leadership team with appointment of Lise Lund Kjems, MD, PhD as Chief Medical Officer;
- Named preeminent neuroscientist and world-renowned researcher, Cynthia A. Lemere, PhD, as Senior Advisor for advancement of Alzheimer’s Disease program; and
- Announced new positive safety and efficacy data from ongoing Phase 1 open-label extension study of Trappsol® Cyclo™ for the treatment of NPC.
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 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.
Summary of Financial Results for Third Quarter 2021
Net loss for the quarter ended September 30, 2021 was approximately $3.8 million. Research and development expenses increased 69% to $1.8 million for the three months ended September 30, 2021, from $1.1 million for the three months ended September 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 $8.4 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—
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CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES CONSOLIDATED BALANCE SHEETS |
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September 30, |
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December 31, |
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(Unaudited) |
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ASSETS |
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CURRENT ASSETS |
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Cash and cash equivalents |
$ |
8,441,297 |
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$ |
12,846,113 |
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Accounts receivable |
|
354,864 |
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|
|
71,017 |
|
|
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Inventory, net |
|
253,797 |
|
|
|
237,909 |
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|
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Current portion of mortgage note receivable |
|
45,070 |
|
|
|
40,772 |
|
|
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Prepaid insurance and services |
|
92,483 |
|
|
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126,474 |
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|
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Prepaid clinical expenses |
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1,804,664 |
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|
|
727,952 |
|
|
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Total current assets |
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10,992,175 |
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14,050,237 |
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FURNITURE AND EQUIPMENT, NET |
|
63,790 |
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|
|
53,910 |
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RIGHT-TO-USE LEASE ASSET, NET |
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22,044 |
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|
34,011 |
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MORTGAGE NOTE RECEIVABLE, LESS CURRENT PORTION |
|
18,520 |
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49,806 |
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TOTAL ASSETS |
$ |
11,096,529 |
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$ |
14,187,964 |
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LIABILITIES AND STOCKHOLDERS’ EQUITY |
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CURRENT LIABILITIES |
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|
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Current portion of lease liability |
$ |
18,882 |
|
|
$ |
17,483 |
|
|
|
Current portion of note payable |
|
106,836 |
|
|
|
114,029 |
|
|
|
Accounts payable and accrued expenses |
|
3,293,869 |
|
|
|
3,541,041 |
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|
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Total current liabilities |
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3,419,587 |
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3,672,553 |
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LONG-TERM LIABILITIES |
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Long-term lease liability, less current portion |
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4,978 |
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|
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18,434 |
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|
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Long-term note payable, less current portion |
|
44,910 |
|
|
|
44,495 |
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|
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Total long-term liabilities |
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49,888 |
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|
|
62,929 |
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STOCKHOLDERS’ EQUITY |
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Common stock, par value $.0001 per share, 20,000,000 shares authorized, 6,453,869 and 4,770,761 shares issued and outstanding, at September 30,2021 and December 31, 2020, respectively |
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646 |
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|
477 |
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Preferred stock, par value $.0001 per share, 5,000,000 shares authorized |
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– |
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– |
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|
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Additional paid-in capital |
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53,182,513 |
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|
|
44,513,841 |
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Accumulated deficit |
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(45,556,105 |
) |
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(34,061,836 |
) |
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Total stockholders’ equity |
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7,627,054 |
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10,452,482 |
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TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY |
$ |
11,096,529 |
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|
$ |
14,187,964 |
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CYCLO THERAPEUTICS, INC. AND SUBSIDIARIES CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) |
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Three Months Ended |
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Nine Months Ended |
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September 30, |
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September 30, |
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2021 |
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2020 |
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2021 |
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2020 |
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REVENUES |
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Product sales |
$ |
403,918 |
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$ |
222,462 |
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$ |
1,000,641 |
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$ |
757,790 |
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EXPENSES |
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Personnel |
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1,668,010 |
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|
424,823 |
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|
2,769,526 |
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|
1,328,156 |
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Cost of products sold (exclusive of direct and indirect overhead and handling costs) |
|
22,671 |
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|
11,578 |
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|
87,425 |
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|
50,958 |
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Research and development |
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1,837,720 |
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|
1,086,753 |
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|
7,739,379 |
|
|
4,859,794 |
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Repairs and maintenance |
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2,425 |
|
|
1,408 |
|
|
5,738 |
|
|
4,521 |
|
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Professional fees |
|
450,490 |
|
|
72,319 |
|
|
1,046,979 |
|
|
435,282 |
|
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Office and other |
|
244,343 |
|
|
48,202 |
|
|
799,856 |
|
|
306,387 |
|
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Board of Director fees and costs |
|
30,708 |
|
|
9,718 |
|
|
30,708 |
|
|
38,434 |
|
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Depreciation |
|
4,207 |
|
|
3,117 |
|
|
12,620 |
|
|
9,353 |
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Freight and shipping |
|
2,360 |
|
|
543 |
|
|
5,154 |
|
|
3,575 |
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Bad debt expense |
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– |
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– |
|
|
– |
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1,272 |
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Total operating expenses |
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4,262,934 |
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1,658,461 |
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12,497,385 |
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|
7,037,732 |
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LOSS FROM OPERATIONS |
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(3,859,016 |
) |
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(1,435,999 |
) |
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(11,496,744 |
) |
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(6,279,942 |
) |
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OTHER INCOME (EXPENSE) |
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Investment and other income (expense) |
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(1,240 |
) |
|
390 |
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|
2,475 |
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|
17,949 |
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LOSS BEFORE INCOME TAXES |
|
(3,860,256 |
) |
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(1,435,609 |
) |
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(11,494,269 |
) |
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(6,261,993 |
) |
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PROVISION FOR INCOME TAXES |
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– |
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– |
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– |
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– |
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NET LOSS |
$ |
(3,860,256 |
) |
$ |
(1,435,609 |
) |
$ |
(11,494,269 |
) |
$ |
(6,261,993 |
) |
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BASIC AND DILUTED NET LOSS PER COMMON SHARE |
$ |
(0.60 |
) |
$ |
(0.94 |
) |
$ |
(1.90 |
) |
$ |
(4.34 |
) |
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WEIGHTED AVERAGE NUMBER OF COMMON SHARES OUTSTANDING |
|
6,447,803 |
|
|
1,519,457 |
|
|
6,040,524 |
|
|
1,443,109 |
|
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Contacts
JTC Team, LLC
Jenene Thomas
(833) 475-8247
CYTH@jtcir.com