HOUSTON, Aug. 16, 2019 /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,
today announced its financial results for the second quarter ended
June 30, 2019. Additionally, the
Company announced potential upcoming milestones and recent
corporate developments.
Management Discussion
Walter Klemp, chairman and chief
executive officer of Moleculin, said, "The second quarter of 2019
exhibited significant progress in the various research initiatives
and clinical trials that are underway on our drug candidates.
During the quarter, we announced three important research
developments that we believe can have meaningful impacts in
successfully attacking certain rare and difficult cancers."
"We recently announced an important discovery for the treatment
of glioblastoma - one of the most common, and aggressive, types of
malignant brain tumor among adults. In animal models, researchers
at MD Anderson have combined our lead STAT3 inhibitor - WP1066 -
with radiation therapy, the combination of which appears to have
developed an immunological memory in immune-competent mice that
enabled them to prevent regrowth of the tumor after these tumor
cells were reintroduced. The result was the development of
long-term survivors, leading to an increase in overall survival in
these models. The median survival time with glioblastoma is 15 to
16 months in people who get surgery, chemotherapy, and radiation
treatment. This is an important breakthrough that could have a
profound impact in extending the lives of people afflicted with
glioblastoma in the years to come."
"The second important research development during the quarter,"
Mr. Klemp continued, "was the announcement of additional positive
safety and efficacy data from our ongoing Phase 1/2 study - in
Poland - of Annamycin for the
treatment of acute myeloid leukemia, and consequently the
advancement to our third cohort of patients to be treated. This
third cohort of patients will be treated at a dose level of 180
mg/m2. The previous two cohorts were treated at lower
levels - 120 mg/m2 and 150 mg/m2,
respectively. We believe one of the most important unique
attributes of Annamycin is its lack of cardiotoxicity, since all
currently approved anthracyclines are significantly cardiotoxic
(potential to damage the heart). Importantly, we have seen no
cardiotoxicity in any of the patients treated to date both in the
US and in Europe. We believe this
is an important pathway that may increase the opportunity for
leukemia patients to qualify for potentially life-saving bone
marrow transplants at a much higher rate than traditionally has
been the case."
"Our third important development during the quarter was the
announcement that our ongoing sponsored research at The
University of Texas MD Anderson Cancer
Center resulted in the discovery that Annamycin, our lead drug
candidate for the treatment of acute myeloid leukemia, has
demonstrated - in animal models - an ability to significantly
improve survival in triple negative breast cancer that has
metastasized to the lungs. Annamycin has previously demonstrated a
high uptake into the lungs in animal models. Triple-negative breast
cancer is considered to be more aggressive and have a poorer
prognosis than other types of breast cancer, mainly because there
are fewer targeted medicines that treat triple-negative breast
cancer. Studies have shown that triple-negative breast cancer is
more likely to spread beyond the breast and more likely to recur
after treatment. With the demonstrated increased uptake into the
lungs in animal models, we are excited about the possibilities for
Annamycin in an expanding list of indications."
Mr. Klemp concluded, "From a strategic standpoint, we are highly
focused on developing 'multiple shots on goal' for the treatment of
rare and difficult cancers. We are pleased with the progress being
achieved in our research initiatives and clinical trials. We remain
on track to deliver data from our ongoing clinical trials through
the balance of the year."
Jonathan Foster, executive vice
president and chief financial officer of Moleculin, stated, "From a
financial standpoint we are in a strong position. We finished the
second quarter with a solid balance sheet with cash of
approximately $18.7 million and no
debt. The strength of our balance sheet provides us the ability to
fund our ongoing research programs and clinical trials. We believe
our existing cash and cash equivalents will be sufficient to fund
our planned operations well into 2020 without the issuance of
additional equity for cash. We are focused on being capital
efficient through our important developmental process."
Anticipated Milestones
Anticipated
Milestones
|
Potential Time
Frame
|
Next Generation
Anthracycline - Annamycin
|
|
Clinical Trials -
Complete cohort of 150 mg/m2 - prior trial recommended
Phase II dose (RP2D)
|
Accomplished and ongoing
2019
|
Announcement of initial clinical data for Annamycin
trial
|
Accomplished and ongoing
2019
|
Announcement of RP2D for Poland Trial
|
2020
|
Poland clinical trial (MB-105) begins Phase II
|
2020
|
Approach FDA on U.S. trial (MB-104) on dose expansion using
Poland trial data
|
2020 (Fast Track
Approved)
|
Announce additional
clinical research in lung and metastatic lung cancers
|
2020
|
Immune/Transcription Modulators - WP1066
Family
|
|
Clinical Trials WP
1066 - Announcement of initial clinical data from WP1066 clinician
sponsored trial
|
2019
|
Phase 1 surgical cohort begins
|
2020
|
Transfer clinician sponsored trial WP1066 IND to
Moleculin
|
Second Half of
2019
|
Emory Physician Led Pediatric Medulloblastoma Trial
begins
|
Second Half of
2019
|
Announcement of
further benefits of our sponsored research agreement with MD
Anderson
|
Accomplished and ongoing
2019
|
Clinical Trial WP1220
- Announce filing and approval of CTA for WP1220 for the treatment
of cutaneous T-cell lymphoma (CTCL)
|
Accomplished
|
Assess WP1220 initial patient data
|
Q4-2019
|
IND for WP1732
submitted
|
2020
|
Dose first patient in
Phase I trial for WP1732
|
2020
|
Announce further
preclinical research results on WP1066 family
|
Accomplished and ongoing
2019
|
Metabolism/Glycosylation Inhibitors
|
|
Begin preclinical
work on WP1122
|
Accomplished
|
Complete formulation
and begin manufacture of drug for trial
|
First Half of
2020
|
File IND for
WP1122
|
2020
|
General
Clinical
|
|
Announce a fourth
approved clinical trial
|
Accomplished
|
Announce a fifth
approved clinical trial
|
2019
|
Second Quarter Highlights and Recent Corporate
Developments
Moleculin Announces Breakthrough Discovery: WP1066
Potentially Capable of Immune Reprogramming in Glioblastoma Animal
Models - Data to be presented at the Inaugural
Conference on Brain Metastases, August
16-17, 2019 - August 6,
2019, the Company announced that a paper entitled
"Immunological Reprogramming in the CNS Tumor Microenvironment and
Therapeutic Efficacy of Radiotherapy with STAT3 Blockade" will be
presented at the Inaugural Conference on Brain Metastases, in
New York City, August 16-17, 2019. Dr. Martina Ott, of MD Anderson Cancer Center, will
be presenting the findings of the research she conducted in
collaboration with Dr. Amy
Heimberger (the Principle Investigator of the current
investigator-initiated clinical of WP1066 for brain tumors) in
combining WP1066 with radiation therapy in glioblastoma animal
models.
One of the findings of her research that is especially
encouraging is that immune-competent mice treated with both
radiation and WP1066 developed an immunological memory that enabled
them to prevent regrowth of the tumor after these tumor cells were
reintroduced. The result was the development of long-term
survivors, leading to an increase in overall survival in these
models.
This study was also particularly interesting because it showed
the most robust immunological responses were located in the CNS
(Central Nervous System) tumor microenvironment rather than
peripheral non-tumor tissue. Importantly, the study indicated
that the combination of STAT3 inhibition with whole brain
radiotherapy had the capacity to enhance the therapeutic effect
against established tumors based on immunological competence.
Moleculin Announces Annamycin in Acute Myeloid Leukemia in
Poland Advances to 3rd Cohort
- July 18, 2019, the Company
announced additional positive interim safety and efficacy data from
its ongoing open label, single arm Phase 1/2 study of Annamycin in
Poland. Three patients were
treated at a dose level of 150 mg/m2 with no
drug-related adverse events, including no signs of cardiotoxicity.
The results for all 3 patients were reviewed by the Drug Safety
Review Committee, which determined that the trial could progress to
the next higher dose level of 180 mg/m2. To date in
Poland, one patient experienced
grade 2 mucositis (which resolved to grade 1 within 2 days) and no
other adverse events related to Annamycin have been reported. One
patient has completed treatment in the 120 mg/m2
(second) cohort in the Company's parallel US clinical trial (the US
trial started at a lower initial dose of 100 mg/m2). The
Company continues to see no evidence of cardiotoxicity in any of
the patients treated thus far in its clinical trials.
Moleculin Files for New Patents for Annamycin After Receiving
FDA Approval of Fast Track Designation - July 10, 2019, the Company announced it has filed
new patents covering the production and reconstitution of
Annamycin, which is currently in two clinical trials for the
treatment of relapsed or refractory acute myeloid leukemia (AML).
Annamycin has Orphan Drug Designation in the US for the treatment
of AML and the Company recently announced promising preclinical
data showing the potential for Annamycin to become an important
treatment for lung metastases. If these patent applications are
approved, this will potentially give the Company 20 years of patent
protection for Annamycin.
Moleculin Announces Additional Positive Interim Results in
First Cohort of Phase 1/2 Clinical Studies of Annamycin in Acute
Myeloid Leukemia in Europe - 2
of 3 patients qualify to proceed to a potentially curative bone
marrow transplant; trial advances to next higher dose
level - May 7, 2019, the
Company announced additional positive interim safety and efficacy
data from its ongoing open label, single arm Phase 1/2 study of
Annamycin in Poland. After
receiving a single starting dose of 120 mg/m2 in the
first cohort of the dose escalation phase of the trial, 2 of 3
patients treated responded sufficiently to qualify for a
potentially curative bone marrow transplant. The results for all 3
patients were reviewed by the Safety Review Committee, which
determined that no drug-related adverse events were observed that
would prevent advancing the trial to the next higher dose level of
150 mg/m2. To date in the European trial, one patient
experienced grade 2 mucositis (which resolved to grade 1 within 2
days) and no other adverse events related to Annamycin have been
reported. No additional patient data have been developed in the
Company's parallel US clinical trial, which is currently recruiting
its second cohort to be given a dose level of 120 mg/m2
(the U.S. trial started at a lower initial dose of 100
mg/m2).
Moleculin Announces $15.0
Million Registered Direct Offering - April 23,2019, the Company announced that it has
entered into definitive agreements with institutional investors to
purchase an aggregate of 9,375,000 units at a public offering price
of $1.60 per unit in a registered
direct offering, which offering was closed on April 25, 2019. Each unit is comprised of one
share of common stock and 0.5 of a warrant to purchase one share of
common stock. Each warrant has an exercise price of $1.75 per share and is exercisable immediately.
The warrants will expire five years from the date of issuance. The
gross proceeds of the offering were approximately $15.0 million, prior to deducting the placement
agent fees and other estimated offering expenses.
Moleculin Receives FDA Approval of Fast Track Designation for
Annamycin - April 18, 2019,
the Company announced that the FDA has approved its request for
Fast Track Designation for its drug, Annamycin, for the treatment
of relapsed or refractory AML.
A drug that receives Fast Track designation is eligible for some
or all of the following:
- More frequent meetings with FDA to discuss the drug's
development plan and ensure collection of appropriate data needed
to support drug approval;
- More frequent written communication from FDA about such things
as the design of the proposed clinical trials and use of
biomarkers;
- Eligibility for Accelerated Approval and Priority Review, if
relevant criteria are met;
- Rolling Review, which means that a drug company can submit
completed sections of its Biologic License Application (BLA) or New
Drug Application (NDA) for review by FDA, rather than waiting until
every section of the NDA is completed before the entire application
can be reviewed. BLA or NDA review usually does not begin until the
drug company has submitted the entire application to the FDA.
Moleculin Announces Significant Discovery in Lung Cancer
Models - Annamycin Found to be Active Against Metastases
to the Lungs in Pre-Clinical Testing - April 17, 2019, the Company announced that its
ongoing sponsored research at The University
of Texas MD Anderson Cancer Center has now demonstrated that
Annamycin is able to significantly improve survival in an
aggressive form of triple negative breast cancer metastasized to
the lungs in animal models. The Company believes its success in
increasing the survival rate in mice with this tumor model in
combination with the previously observed high uptake of Annamycin
by the lungs is a promising indication that supports additional
clinical research in lung and metastatic lung cancers.
Moleculin Announces Agreement with Emory
University to Conduct Pediatric Brain Tumor Trial -
April 11, 2019, the Company announced
it has entered into an agreement with Emory
University to conduct a Phase 1 clinical trial of WP1066 in
children with recurrent or refractory malignant brain tumors. The
study will be conducted at the Aflac Cancer & Blood Disorders
Center at Children's Healthcare of Atlanta.
Moleculin Announces Preclinical Pancreatic Cancer Data
Presented at American Association for Cancer Research Annual
Meeting - April 3, 2019, the
Company announced that preclinical data supporting activity of its
STAT3-inhibiting Immune/Transcription Modulators was presented by
Dr. Waldemar Priebe, Founder and
Chair of the Scientific Advisory Board of Moleculin, Inc. at the
2019 Annual Meeting of the American Association for Cancer Research
in Atlanta, GA.
AACR Abstract:
https://www.moleculin.com/inhibition-of-stat3-in-pancreatic-ductal-adenocarcinoma-and-immunotherapeutic-implications/
The presentation included data resulting from preclinical
evaluation in pancreatic cancer models of STAT3 inhibitors WP1066
and WP1732, both discovered at The University
of Texas MD Anderson Cancer Center and licensed by
Moleculin. WP1066 is an orally bioavailable drug with significant
brain uptake that is currently in Phase 1 clinical studies in
patients with brain tumors. Complementary to WP1066, we believe
STAT3 inhibitor WP1732 may be suitable for IV administration and
demonstrates high uptake by the pancreas without uptake to the
brain.
Financial Results for the Second Quarter ended June 30, 2019
Research and Development Expense. Research and
development ("R&D") expense was $2.1
million and $4.2 million for
the three months ended June 30, 2019
and 2018, respectively. The decrease of $2.1
million is mainly related to costs incurred in 2018 of
producing additional drug product for the Company's Annamycin
clinical trials.
General and Administrative Expense. General and
administrative expense was $1.5
million and $1.2 million for
the three months ended June 30, 2019
and 2018, respectively. The increase of $0.3
million was mainly attributable to an increase in G&A
related payroll costs.
Gain from Change in Fair Value of Warrant
Liability. We recorded a net gain of $2.4 million in the second quarter of 2019 as
compared to a net gain of $0.3
million in the second quarter of 2018, for the change in
fair value on revaluation of our warrant liability associated with
our warrants issued in conjunction with our stock offerings in
April 2019, March 2019, June
2018, February 2018, and
February 2017. We are required to
revalue certain warrants at the time of each warrant exercise and
at the end of each reporting period and reflect in the statement of
operations a gain or loss from the change in fair value of the
warrant in the period in which the change occurred. We calculated
the fair value of the warrants outstanding using the Black-Scholes
model. A gain results principally from a decline in our share price
during the period and a loss results principally from an increase
in our share price. During the quarter, our stock price
fluctuated greatly.
Liquidity and Capital Resources
As of June 30, 2019, the Company
had cash and cash equivalents of $18.7
million. In April 2019, the
Company received gross proceeds of approximately $16.6 million, as a result of a completed public
offering and the exercise of various warrants from past public
offerings. This brings the Company's total net cash raised through
its financing efforts year to date to $20.8
million.
Cash used in operations was $9.2
million for the six months ended June
30, 2019. This $2.9 million
increase over the prior year of $6.3
million was mainly due to preparing for clinical trials, an
increase in R&D payroll costs, an increase in paid
sponsored research and related expenses, and an increase in
license fees. These are all a reflection of the ongoing clinical
and pre-clinical activity and the associated increase in G&A
support for our three core drug technologies.
The Company believes that its existing cash and cash equivalents
as of June 30, 2019, will be
sufficient to fund planned operations into the second quarter of
2020, without the issuance of additional equity for cash. Such
issuances should extend the funding of the Company's planned
operations significantly beyond the second quarter of 2020. Such
plans are subject to the Company's stock price, market conditions,
changes in planned expenses depending on clinical enrollment
progress, the use of drug product or a combination thereof.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical-stage pharmaceutical
company focused on the treatment of highly resistant cancers.
Moleculin has three core technologies, all of which are based on
discoveries made at M.D. Anderson Cancer Center by Dr. Waldemar Priebe and his team. The Company's
clinical-stage drugs are Annamycin, a Next Generation Anthracycline
being studied for the treatment of relapsed or refractory acute
myeloid leukemia, or AML, and WP1066, an Immune/Transcription
Modulator targeting brain tumors, pancreatic cancer and AML. The
Company is also engaged in preclinical development of additional
drug candidates, including additional Immune/Transcription
Modulators, as well as Metabolism/Glycosylation Inhibitors.
Moleculin's Next Generation Anthracycline, Annamycin, we believe,
is unlike any currently approved anthracyclines, as it is designed
to avoid multidrug resistance mechanisms with little to no
cardiotoxicity. Annamycin has preliminary clinical data suggesting
its potential to become the first successful therapy suitable for
the majority of relapsed or refractory AML patients and is
currently in two Phase I/II clinical trials. WP1066 is one of
several Immune/Transcription Modulators capable of stimulating
immune response to tumors by inhibiting the errant activity of
Regulatory T-Cells (TRegs) while also inhibiting key oncogenic
transcription factors, including p-STAT3, c-Myc and HIF-1.
These transcription factors are widely sought targets that may also
play a role in the inability of immune checkpoint inhibitors to
affect more resistant tumors. Moleculin is also developing new
prodrugs to exploit the potential uses of inhibitors of glycolysis.
The Company's lead Metabolism/Glycosylation Inhibitor compound,
WP1122, provides an opportunity to cut off the fuel supply of
tumors by taking advantage of their overdependence on glucose as
compared with healthy cells. New research also points to the
potential for the glucose decoy (2-DG) within WP1122 to be capable
of enhancing the usefulness of checkpoint inhibitors.
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, the ability of Moleculin to
successfully recruit sufficient patients to complete its current
clinical trials; the ability of Moleculin to file an IND for
WP1732; the ability of Moleculin's drug candidates to show safety
and efficacy in patients; and the ability of Moleculin to receive
patent protection for Annamycin. Although Moleculin Biotech
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.
---Financial tables on the following
page---
|
|
|
|
|
|
|
|
|
Moleculin Biotech,
Inc.
|
|
|
|
|
Unaudited
Condensed Consolidated Balance Sheets
(in
thousands)
|
|
June 30,
2019
|
|
December 31,
2018
|
Current
Assets:
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
|
|
|
|
$
|
18,695
|
|
|
$
|
7,134
|
|
Prepaid expenses and
other
|
|
|
|
|
|
1,507
|
|
|
840
|
|
Total current
assets
|
|
|
|
|
|
20,202
|
|
|
7,974
|
|
Furniture and
equipment, net
|
|
|
|
|
|
401
|
|
|
463
|
|
Intangible
assets
|
|
|
|
|
|
11,148
|
|
|
11,148
|
|
Operating lease
right-of-use asset
|
|
|
|
|
|
103
|
|
|
—
|
|
Total Assets
|
|
|
|
|
|
$
|
31,854
|
|
|
$
|
19,585
|
|
|
|
|
|
|
|
|
|
|
Current
Liabilities:
|
|
|
|
|
|
|
|
|
Accounts payable,
accrued expenses and other current liabilities
|
|
|
|
|
|
$
|
2,647
|
|
|
$
|
3,698
|
|
Warrant liability -
current
|
|
|
|
|
|
6,944
|
|
|
180
|
|
Total current liabilities
|
|
|
|
|
|
9,591
|
|
|
3,878
|
|
Operating lease
liability - long-term, net of current portion
|
|
|
|
|
|
171
|
|
|
—
|
|
Deferred rent -
long-term
|
|
|
|
|
|
—
|
|
|
107
|
|
Warrant liability -
long-term
|
|
|
|
|
|
—
|
|
|
1,328
|
|
Total Liabilities
|
|
|
|
|
|
9,762
|
|
|
5,313
|
|
Total Stockholders'
Equity
|
|
|
|
|
|
22,092
|
|
|
14,272
|
|
Total Liabilities and
Stockholders' Equity
|
|
|
|
|
|
$
|
31,854
|
|
|
$
|
19,585
|
|
|
|
|
|
|
|
|
|
|
Unaudited
Condensed Consolidated Statements of Operations
(in thousands, expect shares and per share amounts)
|
|
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
Revenues
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
—
|
|
|
$
|
—
|
|
Operating
Expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
2,099
|
|
|
4,231
|
|
|
5,031
|
|
|
5,469
|
|
General and
Administrative and depreciation and amortization
|
|
1,533
|
|
|
1,227
|
|
|
3,172
|
|
|
2,626
|
|
Total operating
expenses
|
|
3,632
|
|
|
5,458
|
|
|
8,203
|
|
|
8,095
|
|
Loss from
operations
|
|
(3,632)
|
|
|
(5,458)
|
|
|
(8,203)
|
|
|
(8,095)
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
Gain from change in
fair value of warrant liability
|
|
2,407
|
|
|
331
|
|
|
2,936
|
|
|
1,040
|
|
Other
expense
|
|
—
|
|
|
(1)
|
|
|
—
|
|
|
(1)
|
|
Interest income,
net
|
|
4
|
|
|
3
|
|
|
5
|
|
|
4
|
|
Net loss
|
|
$
|
(1,221)
|
|
|
$
|
(5,125)
|
|
|
$
|
(5,262)
|
|
|
$
|
(7,052)
|
|
Loss per common share
- basic and diluted
|
|
$
|
(0.03)
|
|
|
$
|
(0.20)
|
|
|
$
|
(0.15)
|
|
|
$
|
(0.29)
|
|
Weighted average
common shares outstanding - basic and diluted
|
|
42,393,031
|
|
|
25,888,931
|
|
|
35,765,790
|
|
|
24,617,372
|
|
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SOURCE Moleculin Biotech, Inc.