- 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
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210817005231/en/
Investor Contact: JTC Team, LLC Jenene Thomas (833)
475-8247 CYTH@jtcir.com
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