-
Positive outcome from End of Phase 1B/2 Meeting with FDA
-
Company proceeding with a pivotal, adaptive Phase 3 clinical
trial (the "MIRACLE" trial) designed for possible accelerated
approval of Annamycin in combination with cytarabine for the
treatment of R/R AML
-
Webcast replay of the Company's recent MIRACLE trial update
available here
HOUSTON, Aug. 14,
2024 /PRNewswire/ -- Moleculin Biotech, Inc.,
(Nasdaq: MBRX) ("Moleculin" or the "Company"), a Phase 3 clinical
stage pharmaceutical company with a broad portfolio of drug
candidates targeting hard-to-treat tumors and viruses, today
reported its financial results for the quarter ended June 30, 2024.
![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. (PRNewsfoto/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. (PRNewsfoto/Moleculin Biotech, Inc.)](https://mma.prnewswire.com/media/959635/Moleculin_Biotech_Inc_Logo.jpg)
"I am extremely pleased with our recent clinical and regulatory
achievements. Of particular note, the recent positive outcome from
our end of phase 1B/2 clinical trial
(EOP1B/2) meeting with the US Food and Drug Administration (FDA)
combined with the encouraging Annamycin data demonstrated to date,
positions us well for the next phase of development for our AML
program," commented Walter Klemp,
Chairman and Chief Executive Officer of Moleculin. "We are grateful
to the FDA for their constructive feedback and I would like to
congratulate our entire clinical team for their operational
excellence in driving Annamycin forward as a potential treatment
for relapsed or refractory acute myeloid leukemia (R/R AML)
patients. We believe we are truly on the cusp of unlocking
high-value potential for all stakeholders, and most importantly
addressing a significant unmet need for R/R AML patients."
Recent Highlights
- Hosted webcast presentation to discuss the Company's previously
announced plans for its MIRACLE Phase 3 pivotal trial;
- Completed EOP1B/2 meeting with FDA and planning for pivotal,
adaptive Phase 3 clinical trial of Annamycin in combination with
cytarabine for the treatment of R/R AML;
- Reported additional positive efficacy findings from the
Company's Phase 1B/2 (MB-106)
clinical trial evaluating Annamycin in combination with Cytarabine
(also known as "Ara-C" and for which the combination of Annamycin
and Ara-C is referred to as AnnAraC) for the treatment of subjects
with acute myeloid leukemia (AML);
- Announced abstract has been accepted for poster presentation at
the EHA2024 Hybrid Congress being held in Madrid, Spain and virtually; and
- Recruitment began in an Investigator-initiated Phase 2 study
evaluating WP1066 in combination with radiation therapy for the
treatment of adults with glioblastoma (NU 21C06) in cooperation
with the Company.
AML Clinical Development Update
The Company recently announced the positive discussion in and
outcome of its End of Phase 1B/2
(EOP1B/2) meeting with the US Food and Drug Administration (FDA)
supporting the advancement of Annamycin in combination with
Cytarabine (also known as "Ara-C" and for which the combination of
Annamycin and Ara-C is referred to as "AnnAraC") to a Phase 3
pivotal trial for the treatment of AML patients who are refractory
to or relapsed after induction therapy (R/R AML). This Phase 3
"MIRACLE" trial (derived from Moleculin R/R
AML AnnAraC Clinical Evaluation) will be a
global trial, including sites in the US.
The EOP2 meeting was supported by second-line treatment results
from the Company's ongoing MB-106 clinical trial. As recently
reported on June 14, 2024, a total of
22 subjects (Lines 1st-7th) have been
enrolled (the Intent-to-Treat population, ITT) and have completed
efficacy evaluations with 9 subjects (41%) achieving a composite
complete remission (CRc or CR/CRi), consisting of 8 (36%) subjects
with complete remission (CR) and one subject with complete
remission with an incomplete recovery of peripheral blood counts
(CRi), following treatment with AnnAraC.
Of the 10 ITT subjects for whom AnnAraC was administered in the
2nd line setting, 5 achieved a CR (50%) and 6 achieved a
CRc (60%). Of the 14 subjects in the ITT evaluable population that
were 2nd line or 3rd line treatment, 6
achieved a CR (43%) and 7 achieved a CRc (50%). The mDOR for the 9
subjects who achieved a CRc is approximately 7 months and
climbing.
In its EOP1B/2 meeting, the Company obtained valuable input from
the FDA and having resolved a number of key issues, believes that
it has significantly de-risked the pathway to a potential approval.
The MIRACLE study, subject to appropriate future filings with and
potential additional feedback from the FDA and their foreign
equivalents, is expected to initially utilize an adaptive design
whereby the first 75 subjects will be randomized to receive high
dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of
Annamycin, or 230 mg/m2 of Annamycin. At that point, the trial will
be unblinded to select the optimum dose for Annamycin. For the
second half of the trial, approximately 120 additional subjects
will be randomized to receive either HiDAC plus placebo or HiDAC
plus the optimum dose of Annamycin. The selection of the optimum
dose will be based not only on the absence of dose limiting
toxicities but also on the overall balance of safety,
pharmacokinetics and efficacy, consistent with the FDA's new
Project Optimus initiative.
Expected Milestones for Annamycin AML Development
Program
- 2H 2024 – Begin contracting with MIRACLE trial sites
- Q1 2025 – First subject treated in MIRACLE trial
- Q4 2025 – Recruitment update (n=40)
- Mid 2026 – Interim data (n=75) unblinded and Optimum Dose set
for MIRACLE trial
- 2026 – Begin enrollment of 3rd line subjects in
MIRACLE2
- 2027 – Enrollment ends in 2nd line subjects
- 2028 – Final Data for 2nd line subjects in
MIRACLE
- 2H 2028 – Begin submission of a new drug application (NDA) the
treatment of R/R AML for accelerated approval on primary endpoint
of CR from MIRACLE
Annamycin currently has Fast Track Status and Orphan Drug
Designation from the FDA for the treatment of relapsed or
refractory acute myeloid leukemia, in addition to Orphan Drug
Designation for the treatment of soft tissue sarcoma. Furthermore,
Annamycin has Orphan Drug Designation for the treatment of relapsed
or refractory acute myeloid leukemia from the European Medicines
Agency (EMA). For more information about the ongoing MB-106 Phase
1B/2 trial, visit
clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or
clinicaltrials.gov and reference NCT05319587.
Summary of Financial Results for the Second Quarter
2024
Research and development (R&D) expense was $4.1 million and $3.9
million for the three months ended June 30, 2024 and 2023, respectively. The
increase of $0.2 million is mainly
related to sponsored research costs.
General and administrative expense was $2.1 million and $2.5
million for the three months ended June 30, 2024 and 2023, respectively. The
decrease of $0.4 million is mainly
related to a decrease in regulatory and legal fees.
As of June 30, 2024, the Company
had cash and cash equivalents of $10.8
million and believes that the existing cash and cash
equivalents as of June 30, 2024, will
be sufficient to fund our planned operations into the fourth
quarter of 2024. An S-1 was recently filed with the Securities and
Exchange Commission indicating our intentions to raise additional
cash via the issuance of equity in the amount of $12 million. We believe that our existing cash
and cash equivalents as of June 30,
2024, along with the cash expected from this raise will be
sufficient to fund our planned operations into the second
quarter of 2025. The amount of this raise may increase or
decrease. There is no guarantee that the Company will be successful
in such a raise.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical stage
pharmaceutical company advancing a pipeline of therapeutic
candidates addressing hard-to-treat tumors and viruses. The
Company's lead program, Annamycin, is a next-generation
anthracycline designed to avoid multidrug resistance mechanisms and
to eliminate the cardiotoxicity common with currently prescribed
anthracyclines. Annamycin is currently in development for the
treatment of relapsed or refractory acute myeloid leukemia (AML)
and soft tissue sarcoma (STS) lung metastases.
The Company is initiating the MIRACLE (Moleculin
R/R AML AnnAraC Clinical Evaluation)
Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating
Annamycin in combination with cytarabine, together referred to as
AnnAraC, for the treatment of relapsed or refractory acute myeloid
leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the
Company believes it has substantially de-risked the development
pathway towards a potential approval for Annamycin for the
treatment of AML. This study is subject to appropriate future
filings with potential additional feedback from the FDA and their
foreign equivalents.
Additionally, the Company is developing 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 and
other cancers. Moleculin is also engaged in the development of a
portfolio of antimetabolites, including WP1122 for the potential
treatment of viruses, as well as certain cancer
indications.
For more information about the Company, please visit
www.moleculin.com and connect on X, LinkedIn and Facebook.
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 Company's forecasted cash
burn rate (including its estimate of cash sufficient to meet its
projected operating requirements) and the achievement of the
expected milestones set forth above. 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 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.
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
MBRX@jtcir.com
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|
|
|
Moleculin Biotech,
Inc.
|
|
|
|
|
|
|
|
|
Unaudited Condensed
Consolidated Balance Sheets
|
|
|
|
|
|
|
(in
thousands)
|
|
|
|
|
|
June 30,
2024
|
|
December 31,
2023
|
Current
assets:
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
|
|
|
|
$
10,845
|
|
$
23,550
|
Prepaid expenses and
other current assets
|
|
|
|
|
|
2,886
|
|
2,723
|
Total current
assets
|
|
|
|
|
|
13,731
|
|
26,273
|
Furniture and
equipment, net
|
|
|
|
|
|
221
|
|
272
|
Intangible
assets
|
|
|
|
|
|
11,148
|
|
11,148
|
Operating lease
right-of-use asset
|
|
|
|
|
|
475
|
|
524
|
Total assets
|
|
|
|
|
|
$
25,575
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|
$
38,217
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|
|
|
|
|
|
|
|
|
Current
liabilities:
|
|
|
|
|
|
|
|
|
Accounts payable and
accrued expenses and other current liabilities
|
|
|
|
|
|
$
5,746
|
|
$
6,815
|
Total current
liabilities
|
|
|
|
|
|
5,746
|
|
6,815
|
Operating lease
liability - long-term, net of current portion
|
|
|
|
|
|
420
|
|
474
|
Warrant liability -
long term
|
|
|
|
|
|
1,704
|
|
4,855
|
Total liabilities
|
|
|
|
|
|
7,870
|
|
12,144
|
Total stockholders'
equity
|
|
|
|
|
|
17,705
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|
26,073
|
Total liabilities and
stockholders' equity
|
|
|
|
|
|
$
25,575
|
|
$
38,217
|
|
|
|
|
|
|
|
|
|
Unaudited Condensed
Consolidated Statements of Operations
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|
|
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|
|
|
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|
Three Months Ended
June 30,
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Six Months Ended
June 30,
|
(in thousands,
except share and per share amounts)
|
|
2024
|
|
2023
|
|
2024
|
|
2023
|
Revenues
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|
$
-
|
|
$
-
|
|
$
-
|
|
$
-
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
4,090
|
|
3,888
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|
8,342
|
|
9,576
|
General and
administrative and depreciation and amortization
|
|
2,095
|
|
2,523
|
|
4,520
|
|
5,190
|
Total operating
expenses
|
|
6,185
|
|
6,411
|
|
12,862
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|
14,766
|
Loss from
operations
|
|
(6,185)
|
|
(6,411)
|
|
(12,862)
|
|
(14,766)
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Other
income:
|
|
|
|
|
|
|
|
|
Gain from change in
fair value of warrant liability
|
|
1,696
|
|
36
|
|
3,151
|
|
75
|
Other income,
net
|
|
11
|
|
9
|
|
22
|
|
17
|
Interest income,
net
|
|
159
|
|
390
|
|
400
|
|
783
|
Net
loss
|
|
(4,319)
|
|
(5,976)
|
|
(9,289)
|
|
(13,891)
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|
|
|
|
|
|
|
|
|
Net loss per common
share - basic and diluted
|
|
$
(1.70)
|
|
$
(3.02)
|
|
$
(3.71)
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|
$
(7.13)
|
Weighted average common
shares outstanding - basic and diluted
|
|
2,543,244
|
|
1,979,258
|
|
2,504,709
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|
1,948,135
|
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SOURCE Moleculin Biotech, Inc.