HOUSTON, March 24, 2021
/PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX)
(Moleculin or the Company), a clinical stage pharmaceutical company
with a broad portfolio of drug candidates targeting highly
resistant tumors and viruses, today announced its financial results
for the year ended December 31, 2020
and provided a business update.
Recent Milestones and Accomplishments:
Corporate Strategy and Events
Raised gross proceeds of approximately $81 million through registered equity offering
and ATM program in 1Q21 providing runway into 2025 based on
the Company's current R&D spending levels. The Company may
expand its R&D expenditures to take advantage of new
opportunities within its broad pipeline and/or increase the speed
of its clinical trials. At a minimum however, the Company intends
for current cash levels to support an operating runway through at
least 2023.
Next Generation Anthracycline - Annamycin
- Received $1.5 million grant to
fund a Phase 1b/2 clinical trial for
the treatment of soft tissue sarcoma ("STS") lung metastases in
Europe
- Received FDA IND and ODD for Annamycin against STS; plan to
begin a Phase 1b/2 clinical trial in
the US for patients after receiving first-line therapy for STS that
has metastasized to the lungs
- Reached a 2nd dose limiting toxicity (DLT) in March 2021 and plan to establish a maximum
tolerable dose (MTD) in our European trial for Annamycin against
acute myeloid leukemia (AML); plan to pursue Phase 2 once the
recommended Phase 2 dose (RP2D) is established
- Presented animal data at American Society for Hematology
showing Annamycin's synergistic activity against AML when used in
combination with the Ara-C; based on this data we plan a potential
Phase 1/2 trial with Annamycin in combination with Ara-C on AML in
2021
Immune/Transcription Modulators - WP1066 Portfolio
- Reported positive interim results in Emory
University pediatric brain tumor Phase 1 clinical trial;
DIPG patient showed an apparent response in first cohort
- Advanced WP1066 for GBM in adults to fourth and final cohort in
dose escalation trial at MD Anderson; notified physician sponsoring
trial is leaving MD Anderson; pursuing IND transfer and
continuation of research
- Received "Rare Pediatric Disease" designation from FDA for
WP1066; entitles Moleculin to receive a transferrable Priority
Review Voucher upon New Drug Approval for any one of three
different brain tumor indications
Infectious Disease and Metabolism/Glycosylation Inhibitors-
WP1122, WP1096 and WP1097 Portfolio
- Multiple positive pre-clinical in-vitro studies on WP122 in its
potential ability to address COVID-19
- Positive in vitro results demonstrating the antiviral activity
of WP1096 and WP1097 in a range of infectious diseases including:
SARS-CoV-2, HIV, Zika and Dengue Fever
- Working to initiate a Phase 1a/1b
clinical trial in COVID-19 or a physician-sponsored clinical trial
for a cancer indication, or both in 2021
Anticipated 2021 Milestones
- Potential for 8 clinical trials in 2021, including 3 to be
conducted by Moleculin and 5 primarily externally funded and
conducted trials; External funding will be relied upon to the
extent it is available
Management Discussion
"We are extremely pleased by the progress we made over the past
year despite headwinds from the global COVID-19 pandemic. 2020
proved to be a pivotal year for the Company as we progressed our
clinical trials and expanded our product pipeline. Although we are
still in the early months of 2021, we are excited to see this
momentum build, as we have raised approximately $81 million in
the first quarter, which will enable us to further pursue our
pipeline with expanded pre-clinical and clinical activities,
through at least 2023," commented Walter
Klemp, Chairman and CEO of Moleculin.
"We continue to see tremendous progress and promise across our
three primary drug candidates, which have accounted for five Phase
1 clinical trials either completed or under way to date. Our lead
candidate Annamycin, our "Next Generation Anthracycline" designed
to avoid multidrug resistance mechanisms, received an independent
assessment last year, which confirmed the absence of cardiotoxicity
in patients treated in both our US and European open label and
single arm Phase 1/2 clinical trials for acute myeloid leukemia. We
were pleased to conclude our US Phase 1/2 clinical trial of
Annamycin in AML, and following discussions with the FDA, will
focus on establishing a recommended Phase 2 Dose and generating
requested safety and efficacy data within our European trial in
Poland. In our European trial, we
are currently treating patients in the 5th cohort at 240
mg/m2. Dose limiting toxicities relating to liver function
have now been noted at this level sufficient to establish an upper
limit of dosing. We are planning to amend the protocol for
this trial to allow exploration of an intermediate dose level
between the 210 mg/m2 dose in the fourth cohort and the
current 240 mg/m2 dose level, in order to establish the
maximum tolerated dosage (MTD) and Recommended Phase 2 dose (RP2D),
which may be the same. As soon as the RP2D is established, we
intend to begin a Phase 2 expansion phase to assess the efficacy of
Annamycin as a single agent. In addition, as a result of our
preclinical research showing potential synergistic effect from
combining Annamycin with Ara-C (a drug commonly used as a single
agent and in combination chemotherapy for AML), we also intend to
begin the Phase 1 portion of an AML trial using Annamycin in
combination with Ara-C.
While we are pleased by our continued development of Annamycin
in AML, we are also excited by the encouraging results observed in
Annamycin's ability to treat lung metastases. Sponsored research
has demonstrated that Annamycin is capable of accumulating in the
lungs in animal models at concentration levels up to 34-fold higher
than doxorubicin, the current standard of care chemotherapy for a
range of lung metastases. This research has also shown that
Annamycin has activity in several different lung metastases,
including sarcoma, colorectal cancer and triple negative breast
cancer. Most recently, we announced that Annamycin demonstrated a
potentially significant therapeutic benefit against metastatic
osteosarcoma in a preclinical animal study. In this preclinical
study, computerized tomography scans showed that animals treated
with Annamycin exhibited significant suppression of tumor growth.
Further, not a single death was observed in the treated animals,
whereas significant tumor burden contributed to the rapid death of
90% of untreated animals. As of day 130 in the trial, the
survival rate for animals treated with Annamycin was 100%, compared
with only 10% for untreated animals. We have received both
Investigational New Drug ("IND") status, and Orphan Drug
Designation ("ODD") for Annamycin, allowing us to begin a Phase
1b/2 clinical trial in the US for
patients with soft tissue sarcoma (STS) that has metastasized to
the lungs after first-line therapy for their disease. To manage the
upcoming Phase 1/2 Study in the US, we selected Catalyst Clinical
Research as our contract research organization. Our efforts in
progressing Annamycin in lung metastasis have also paved the way
for a second European trial in 2021, as our license partner
recently received a $1.5 million
grant from Agencja Badań Medycznych in Poland to fund a Phase 1b/2 clinical trial of Annamycin for the
treatment of soft tissue sarcoma lung metastases in Europe.
We also continued to drive the clinical development of WP1066,
the lead molecule in Moleculin's portfolio of immune stimulators
and modulators of transcription. WP1066 is currently in two US
physician-sponsored Phase 1/2 clinical trials, one at MD Anderson
for the treatment of glioblastoma ("GBM") in adults and the second
at Emory University for the treatment
of pediatric brain tumors. In our Phase 1 clinical trial of WP1066
for the treatment of brain tumors in children being at conducted at
the Aflac Cancer & Blood Disorders Center at Children's
Healthcare of Atlanta, the first
cohort of patients was treated with no adverse events related to
treatment and the trial has progressed full enrollment of the
second cohort at a dose level of 6mg/kg. Notably, within the first
cohort, one patient with diffuse intrinsic pontine glioma ("DIPG")
showed an apparent response to the treatment with both clinical
improvement and radiologic reduction of tumor size; we are
particularly encouraged by this apparent response as approximately
200 clinical trials have been conducted in DIPG, and no drug to
date has been able to show significant activity in this
disease.
In our trial at MD Anderson, WP1066 is in the fourth and final
cohort in the dose escalation phase. We were notified during the
first quarter of 2021 that the physician sponsoring this trial is
leaving MD Anderson. Although we cannot be assured that this trial
will continue at MD Anderson after her departure, we have requested
that MD Anderson have the IND for this trial transferred into our
name to help ensure the potential continuation of this important
research. While we are making arrangements to pursue this research
in additional physician-sponsored trials, we expect that continued
research on WP1066 in adult GBM will be temporarily delayed in
2021.
In addition to WP1066, we see meaningful opportunity in WP1220,
which is an analog of WP1066, in treating cutaneous T-cell lymphoma
("CTCL"). The US market for CTCL had estimated sales of
$40 million in 2020 and consisted of
technologies that are as much as 40 years old. The data from our
WP1220 Proof of Concept Trial for the treatment of CTCL, while
limited in patient size, was promising; WP1220 demonstrated an
objective response rate of 45%, with no adverse events and 55%
stable disease, resulting in 100% clinical benefit. Given the
tremendous market opportunity and these strong early indications of
efficacy, we plan to seek a collaborative partner to support a
Phase 2 clinical study of WP1220 in CTCL in 2021.
While we continue to drive the further development of our drugs
that are showing meaningful activity in cancer indications, we
believe our WP1122 portfolio holds tremendous opportunity for
creating long-term shareholder value in the area of infectious
disease. In 2020, WP1122 demonstrated its unique mechanism of
action and in-vitro activity in numerous preclinical studies and
independent research. We believe the preclinical work conducted and
under way for WP1122 will support an IND application or its
equivalent in other countries for either cancer-related or
virus-related clinical trials (or both) during the first half of
2021. Although our initial preclinical focus for the WP1122 program
was to help provide a treatment for the growing COVID-19 pandemic,
we discovered that two other molecules within our portfolio of
antimetabolites displayed significant in vitro antiviral activity
against SARS-CoV-2 and other hard to treat viruses. Independent
laboratory testing of our drug candidates, WP1096 and WP1097, not
only showed significant antiviral activity against SARS-CoV-2, but
also showed greater potential against HIV, Zika, and Dengue
Fever. We caution that the above data is preclinical and there
is no assurance that we will see similar results in our planned
clinical trials."
Mr. Klemp concluded, "Our strategy since founding Moleculin has
been to deliver long term shareholder value through our 'multiple
shots on goal strategy'. Following our recent capital raise in the
first quarter of 2021, we are now optimally positioned to deliver
on this strategy, with cash runway through at least 2023, and the
potential to see 8 clinical trials this year on our drug
candidates."
Financial Results for the Year Ended December 31, 2020
Research and development ("R&D") expense was $12.8 million and $11.0
million for the years ended December
31, 2020 and 2019, respectively. The increase in R&D of
$1.8 million was primarily driven by
increased clinical trial activity (3 drugs in 4 clinical trials in
2019, versus 3 drugs in 5 clinical trials in 2020), increased costs
related to sponsored research agreements, costs related to
manufacturing of additional drug product, and two additional
employees in R&D headcount.
General and administrative ("G&A") expense was $6.8 million and $6.3
million for the years ended December
31, 2020 and 2019, respectively. The increase in G&A of
$0.5 million was mainly attributable
to increased payroll related costs for an additional finance staff,
increased stock-based compensation expense, and increased costs for
officer's liability insurance being partially offset by reduced
travel expenses due to the COVID-19 pandemic.
Net loss for the year ended December 31,
2020 was $17.4 million, which
included non-cash gains of $2.3
million on warrants in 2020 as compared to $4.1 million in the prior year and approximately
$1.7 million of stock-based
compensation expense in 2020 as compared to $1.5 million in 2019.
Liquidity and Capital Resources
We believe that our cash resources as of December 31, 2020, along with the additional
funding received subsequent to year-end, will be sufficient to meet
our projected operating requirements, based on our current use of
cash, through at least the year 2023. Such projections are subject
to changes in our internally funded preclinical and clinical
activities, including unplanned preclinical and clinical
activity.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company focused on the development of a broad portfolio of oncology
drug candidates for the treatment of highly resistant tumors and
viruses. The Company's clinical stage drugs are: Annamycin, a Next
Generation Anthracycline, designed to avoid multidrug resistance
mechanisms with little to no cardiotoxicity being studied for the
treatment of relapsed or refractory acute myeloid leukemia, more
commonly referred to as AML, WP1066, an Immune/Transcription
Modulator capable of inhibiting p-STAT3 and other oncogenic
transcription factors while also stimulating a natural immune
response, targeting brain tumors, pancreatic cancer and hematologic
malignancies, and WP1220, an analog to WP1066, for the topical
treatment of cutaneous T-cell lymphoma. Moleculin is also engaged
in preclinical development of additional drug candidates, including
other Immune/Transcription Modulators, as well as WP1122 and
related compounds capable of Metabolism/Glycosylation
Inhibition.
For more information about the Company, please visit
http://www.moleculin.com.
Forward-Looking Statements
Some of the statements in this release are forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933, Section 21E of the Securities Exchange Act of 1934 and the
Private Securities Litigation Reform Act of 1995, which involve
risks and uncertainties. Forward-looking statements in this press
release include, without limitation, our expected cash runway;
establishing a recommended Phase 2 Dose for Annamycin in
2021; the timing of the commencement and progress of the
clinical trials conducted by Moleculin and by third parties; the
ability of Moleculin to find a collaborative partner to support a
Phase 2 clinical study of WP1220 in CTCL in 2021; and the ability
to file for W1122 an IND application or its equivalent for either
cancer-related or virus-related clinical trials in the first half
of 2021. Although Moleculin believes that the expectations
reflected in such forward-looking statements are reasonable as of
the date made, expectations may prove to have been materially
different from the results expressed or implied by such
forward-looking statements. Moleculin Biotech has attempted to
identify forward-looking statements by terminology including
''believes,'' ''estimates,'' ''anticipates,'' ''expects,''
''plans,'' ''projects,'' ''intends,'' ''potential,'' ''may,''
''could,'' ''might,'' ''will,'' ''should,'' ''approximately'' or
other words that convey uncertainty of future events or outcomes to
identify these forward-looking statements. These statements are
only predictions and involve known and unknown risks,
uncertainties, and other factors, including those discussed under
Item 1A. "Risk Factors" in our most recently filed Form 10-K filed
with the Securities and Exchange Commission ("SEC") and updated
from time to time in our Form 10-Q filings and in our other public
filings with the SEC. Any forward-looking statements contained
in this release speak only as of its date. We undertake no
obligation to update any forward-looking statements contained in
this release to reflect events or circumstances occurring after its
date or to reflect the occurrence of unanticipated events.
Contacts
James Salierno
/ Carol Ruth
The Ruth Group
973-255-8361 / 917-859-0214
jsalierno@theruthgroup.com
cruth@theruthgroup.com
-- Financial Tables Follow--
Moleculin Biotech,
Inc.
|
|
|
|
|
|
|
|
|
Unaudited
Condensed Consolidated Balance Sheets
|
|
|
|
|
|
|
|
(in
thousands)
|
|
December
31, 2020
|
|
|
December
31, 2019
|
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
15,173
|
|
|
$
|
10,735
|
|
Prepaid expenses and
other current assets
|
|
|
2,025
|
|
|
|
2,749
|
|
Total current
assets
|
|
|
17,198
|
|
|
|
13,484
|
|
Furniture and
equipment, net
|
|
|
483
|
|
|
|
316
|
|
Intangible
assets
|
|
|
11,148
|
|
|
|
11,148
|
|
Operating lease
right-of-use asset
|
|
|
202
|
|
|
|
287
|
|
Total
assets
|
|
$
|
29,031
|
|
|
$
|
25,235
|
|
|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
Accounts payable and
accrued expenses and other current liabilities
|
|
$
|
2,920
|
|
|
$
|
3,570
|
|
Total current
liabilities
|
|
|
2,920
|
|
|
|
3,570
|
|
Operating lease
liability - long-term, net of current portion
|
|
|
159
|
|
|
|
276
|
|
Warrant liability -
long term
|
|
|
8,192
|
|
|
|
5,818
|
|
Total
liabilities
|
|
|
11,271
|
|
|
|
9,664
|
|
Total stockholders'
equity
|
|
|
17,760
|
|
|
|
15,571
|
|
Total liabilities and
stockholders' equity
|
|
$
|
29,031
|
|
|
$
|
25,235
|
|
Unaudited
Condensed Consolidated Statements of Operations
|
|
|
|
|
|
|
|
|
|
|
|
Year Ended
December 31,
|
|
(in thousands,
except share and per share amounts)
|
|
|
2020
|
|
|
2019
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
|
|
|
|
|
|
|
|
12,757
|
|
|
|
11,013
|
|
General and
administrative and depreciation
|
|
|
|
|
|
|
|
|
|
|
6,985
|
|
|
|
6,511
|
|
Total operating
expenses
|
|
|
|
|
|
|
|
|
|
|
19,742
|
|
|
|
17,524
|
|
Loss from
operations
|
|
|
|
|
|
|
|
|
|
|
(19,742)
|
|
|
|
(17,524)
|
|
Other
income:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Gain from change in
fair value of warrant liability
|
|
|
|
|
|
|
|
|
|
|
2,346
|
|
|
|
4,062
|
|
Other income,
net
|
|
|
|
|
|
|
|
|
|
|
28
|
|
|
|
15
|
|
Interest income,
net
|
|
|
|
|
|
|
|
|
|
|
13
|
|
|
|
13
|
|
Net loss before
taxes
|
|
|
|
|
|
|
|
|
|
$
|
(17,355)
|
|
|
$
|
(13,434)
|
|
Income tax
benefit
|
|
|
|
|
|
|
|
|
|
|
—
|
|
|
|
229
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
$
|
(17,355)
|
|
|
$
|
(13,205)
|
|
Net loss per common
share - basic and diluted
|
|
|
|
|
|
|
|
|
|
$
|
(1.76)
|
|
|
$
|
(1.95)
|
|
Weighted average
common shares outstanding - basic and diluted
|
|
|
|
|
|
|
|
|
|
|
9,845,685
|
|
|
|
6,786,901
|
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/moleculin-biotech-inc-reports-financial-results-for-the-year-ended-december-31-2020-301254611.html
SOURCE Moleculin Biotech, Inc.