Continued progress in lead development program
evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1)
in ongoing pivotal study (TransportNPC™)
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—
CYCLO THERAPEUTICS, INC. AND
SUBSIDIARIES
CONSOLIDATED BALANCE
SHEETS
September 30, 2021
December 31, 2020
(Unaudited)
ASSETS
CURRENT ASSETS
Cash and cash equivalents
$
8,441,297
$
12,846,113
Accounts receivable
354,864
71,017
Inventory, net
253,797
237,909
Current portion of mortgage note
receivable
45,070
40,772
Prepaid insurance and services
92,483
126,474
Prepaid clinical expenses
1,804,664
727,952
Total current assets
10,992,175
14,050,237
FURNITURE AND EQUIPMENT, NET
63,790
53,910
RIGHT-TO-USE LEASE ASSET, NET
22,044
34,011
MORTGAGE NOTE RECEIVABLE, LESS CURRENT
PORTION
18,520
49,806
TOTAL ASSETS
$
11,096,529
$
14,187,964
LIABILITIES AND STOCKHOLDERS’
EQUITY
CURRENT LIABILITIES
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
Total current liabilities
3,419,587
3,672,553
LONG-TERM LIABILITIES
Long-term lease liability, less current
portion
4,978
18,434
Long-term note payable, less current
portion
44,910
44,495
Total long-term liabilities
49,888
62,929
STOCKHOLDERS' EQUITY
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
646
477
Preferred stock, par value $.0001 per
share, 5,000,000 shares authorized
-
-
Additional paid-in capital
53,182,513
44,513,841
Accumulated deficit
(45,556,105
)
(34,061,836
)
Total stockholders' equity
7,627,054
10,452,482
TOTAL LIABILITIES AND STOCKHOLDERS'
EQUITY
$
11,096,529
$
14,187,964
CYCLO THERAPEUTICS, INC. AND
SUBSIDIARIES
CONSOLIDATED STATEMENTS OF
OPERATIONS
(Unaudited)
Three Months Ended
Nine Months Ended
September 30,
September 30,
2021
2020
2021
2020
REVENUES
Product sales
$
403,918
$
222,462
$
1,000,641
$
757,790
EXPENSES
Personnel
1,668,010
424,823
2,769,526
1,328,156
Cost of products sold (exclusive of direct
and indirect overhead and handling costs)
22,671
11,578
87,425
50,958
Research and development
1,837,720
1,086,753
7,739,379
4,859,794
Repairs and maintenance
2,425
1,408
5,738
4,521
Professional fees
450,490
72,319
1,046,979
435,282
Office and other
244,343
48,202
799,856
306,387
Board of Director fees and costs
30,708
9,718
30,708
38,434
Depreciation
4,207
3,117
12,620
9,353
Freight and shipping
2,360
543
5,154
3,575
Bad debt expense
-
-
-
1,272
Total operating expenses
4,262,934
1,658,461
12,497,385
7,037,732
LOSS FROM OPERATIONS
(3,859,016
)
(1,435,999
)
(11,496,744
)
(6,279,942
)
OTHER INCOME (EXPENSE)
Investment and other income (expense)
(1,240
)
390
2,475
17,949
LOSS BEFORE INCOME TAXES
(3,860,256
)
(1,435,609
)
(11,494,269
)
(6,261,993
)
PROVISION FOR INCOME TAXES
-
-
-
-
NET LOSS
$
(3,860,256
)
$
(1,435,609
)
$
(11,494,269
)
$
(6,261,993
)
BASIC AND DILUTED NET LOSS PER COMMON
SHARE
$
(0.60
)
$
(0.94
)
$
(1.90
)
$
(4.34
)
WEIGHTED AVERAGE NUMBER OF COMMON
SHARES OUTSTANDING
6,447,803
1,519,457
6,040,524
1,443,109
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211115006216/en/
JTC Team, LLC Jenene Thomas (833) 475-8247 CYTH@jtcir.com
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