– MB-106 AML Phase 1B/2 trial (MB-106) preliminary data readout:
Reached 67% recruitment
– MB-106 preliminary results: Intent to Treat
CR rate of 40% plus a CR/ CRi rate of 47% (N=15); Dosed Per
Protocol CR rate of 46% plus a CR/CRi rate of 54% (N=13)
– First 1st line AML subject
treated and resulted in a CR in MB-106
– No CR/CRi's in MB-106 have relapsed to
date
– MB-107 STS Lung Mets Phase 1B/2 trial preliminary data readout: Phase
1B median extended overall survival
of 11 months for heavily pre-treated subjects (N=15); Phase 2 data
continues to develop
– 100% of Annamycin subjects in multiple
studies (N=62) continue to show no signs of cardiotoxicity during
study as evidenced by an independent expert's reports
– Company provides 2024 business outlook and
updates its upcoming milestones
HOUSTON, Jan. 24,
2024 /PRNewswire/ -- Moleculin Biotech,
Inc., (Nasdaq: MBRX) (Moleculin or the Company), a
clinical stage pharmaceutical company with a broad portfolio of
drug candidates targeting hard-to-treat tumors and viruses, today
announced 2023 year-end Annamycin clinical trials preliminary data
and 2024 expectations for multiple data readouts and a transition
to pivotal Phase 2B/3 clinical
studies. The Company also updated its upcoming milestones.
"Over the course of 2023, we delivered on our promise for a year
of important data from our Annamycin clinical development programs.
We are well-positioned to continue building upon our encouraging
growing body of preliminary clinical data and transition to pivotal
Phase 2B/3 clinical trials by
year-end 2024. We believe that Annamycin should be positioned as a
2nd line therapy for acute myeloid leukemia or AML, and
for the first time ever, should enable a clear majority of patients
to benefit from anthracyclines. Anthracyclines continue to
represent one of the most important treatments for AML and advanced
soft tissue sarcoma," commented Walter
Klemp, Chairman and Chief Executive Officer of
Moleculin.
Mr. Klemp continued, "Our progress is accelerating. Last month,
we reported strong results from our first 11 patients in our AML
clinical trial. Now, just weeks later our evaluable patients have
increased to 15 and the results are even stronger. Enrollment
continues to be robust, and we expect to announce the completion of
enrollment of 2nd line patients by the time we announce
financial results at the end of March, if not sooner. We believe
that data will present a clear picture of the approvability of
Annamycin and will support our discussions with the FDA for
developing an accelerated approval pathway as a 2nd line
therapy for relapsed and refractory AML, including a pivotal
approval clinical trial we expect to begin before the end of 2024.
With our recent financing in December
2023, we have extended our runway into the fourth quarter of
2024, as well. We thank our investors and collaborators for their
support in 2023 and we look forward to an exciting and prosperous
2024."
Preliminary 2023 Year-End Annamycin Clinical Trials
Data
AML
The Company is currently conducting its Phase 1B/2 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 AML as both first line therapy
and for subjects who are refractory to or relapsed after induction
therapy (MB-106). clinicaltrialsregister.eu: EudraCT 2020-005493-10
or clinicaltrials.gov: NCT05319587. The Company has also begun
recruiting 1st line subjects into this trial. The
Company continues to treat subjects in the Phase 2 portion of the
study. Below is a summary of the data to date on this trial which
is preliminary and subject to change.
- Among subjects who had an evaluable post treatment bone
marrow biopsy, or who dropped out due to an adverse event (AE),
there have been 6 complete responses (CR's) and 1 complete response
with incomplete recovery of platelets and/or neutrophils or 40% CR
and 47% CR/CRi's of the intent to treat (ITT) subjects (N=15) and
46% CR's and 54% CR/CRi's of the subjects treated (dosed with
Annamycin) (N=13). Two subjects experienced adverse events and were
not dosed per protocol with one having an allergic reaction to
Annamycin, the first the Company has seen in over 70 subjects dosed
in the Company's multiple Annamycin clinical trials; the second
adverse event was due to an allergic reaction to cytarabine. The
CR/CRi's have been spread across 3 different sites in two different
countries (Poland and Italy) and 7 out of 9 sites participating in
the study have recruited subjects to date.
- The Company has expanded the study protocol to include a cohort
of 1st line subject which should provide data to enable
the designing of a potential confirmatory Phase 3 post-approval
study, however it does not expect the addition of this cohort to
delay its End of Phase 2 (EOP2) Meeting with the FDA, which will
focus primarily on securing an accelerated approval pathway for the
treatment of 2nd line patients (those who were relapsed
from or refractory to a 1st line AML therapy). The first
1st line therapy AML subject for Annamycin was
recruited, treated, and upon evaluation it was determined that the
treatment resulted in a CR.
- The first CR subject was treated in February of 2023. The
Company has not been notified of any relapses to date, indicating
significant durability of the CRs being generated in this
study.
There continues to be no evidence of cardiotoxicity as reported
by an independent expert's reports following assessments of
ejection fractions, strain analyses, ECGs, and cardiac biomarkers
including Troponin-I and T cross all trials (N=62). Most subjects
have experienced myelosuppression, febrile neutropenia and/or
thrombocytopenia, which are common adverse events with other
anthracyclines.
- Currently, the median age of the 15 intent to treat subjects in
MB-106 is 69 years (range of age is 32 to 78 years) with a median
number of prior therapies for AML of 1 (range of 0 to 6).
- The Company has recruited 19 subjects to date with 2 subjects
withdrawing from the trial due to adverse events and 4 other
subjects not yet having a bone marrow aspirate
fully evaluable. The Company may recruit up to 28 subjects in
the Phase 1B/2 clinical trial. At the
Company's current rate of recruitment for the study, Moleculin
expects to complete recruitment and report topline data in 1H 2024,
if not by the end of the first quarter.
STS Lung Mets
On September 21, 2023, Moleculin
announced the completion of enrollment in the Phase 2 portion of
its U.S. Phase 1B/2 clinical trial
evaluating Annamycin as monotherapy for the treatment of soft
tissue sarcoma lung metastases (MB-107) clinicaltrials.gov:
NCT04887298. All subjects had pulmonary metastasis from soft tissue
sarcoma (STS Lung Mets or STS) and at least one prior therapy.
There was no limit on how many prior therapies a subject could have
prior to entering this study. Most subjects were heavily treated
with other therapies prior to entering our trial with our treatment
representing the 4th median therapy for all subjects in
the Phase 1B and Phase 2 portion of
the trial (range of 2 to 12). Below is a summary of the data to
date on this trial which is preliminary and subject to change.
- The Company previously announced the preliminary data from the
Phase 1B portion of the study showing
median progression free survival (PFS) for 67% of the Phase
1B subjects of over 2 months (N=15)
and median overall survival (OS) of 11.3 months. Not including the
one subject that withdrew from OS follow-up the median OS increased
to 13 months.
- Additionally, the Company noted that Phase 1B subjects with less than or equal to 2 prior
treatments and treated at the recommended Phase 2 dose or lower
demonstrated a higher percentage of subjects with PFS of 2
months or greater at 78% (N=9).
- Of the 17 subjects in the Phase 2 portion of the study, all
treatments have ceased, and subjects are being followed
for PFS and OS. A majority of subjects remain alive at this
time. The Company believes that the data will be similar to or
better than the Phase 1B portion of
the study and will announce the results once the study has
substantively concluded.
2024 Expected Upcoming Milestones
The Company announces the updated expected upcoming milestones
for 2024.
Annamycin
- AML
- 1H 2024: In-depth data review and presentation of topline data
on MB-106 clinical trial.
- 1H 2024: MB-106 End of Phase 2 (EOP2) Meeting with the FDA
and/or its European equivalent.
- Mid 2024: Identify next phase of development/pivotal
program.
- Late 2024 into 1H 2025: Initiate pivotal program.
- STS Lung Mets
- 2H 2024: Final MB-107 data readout.
- 2024: Identify next phase of development/pivotal program.
- Late 2024 into 1H 2025: Initiate 1st line STS study
with an investigator-initiated trial.
Other Drug Candidates
Moleculin is in ongoing discussions with multiple academic
institutions in separate programs evaluating WP1066 for the
treatment of glioblastomas and/or pediatric brain tumors. The
Company expects to finalize agreements with Northwestern University for an
investigator-initiated and funded trial in early 2024
(clinicaltrials.gov: NCT05879250).
Expected Upcoming Milestones
- Report topline results from investigator-initiated Phase 1
study in pediatric brain tumors.
- Announce results of formulation efforts for intravenous
delivery.
- Expect the commencement of an externally funded
investigator-initiated adult clinical trial to be announced in 1H
2024. Seeking investigator-initiated pediatric cancer clinical
trial in 2H 2024.
WP1122 was developed as a prodrug of 2 deoxy-D-Glucose (2-DG) to
provide a more favorable pharmacological profile and was found to
have greater potency than 2-DG monotherapy in preclinical models
where tumor cells require higher glycolytic activity than normal
cells. WP1122 has also been shown to have a greater antiviral
effect than 2-DG against SARS-CoV-2 in MRC-5 cells in culture. The
improved pharmacokinetic and pharmacodynamic (PK/PD) profile of
WP1122 compared to 2-DG was noted in mice following oral dosing at
equimolar (i.e., equivalent levels of 2-DG) doses. WP1122
successfully completed a Phase 1 clinical trial, which established
a recommended safe dose for future potential Phase 1B or Phase 2 clinical trials. The WP1122
Portfolio includes numerous analogs, including WP1096, which has
demonstrated the potential for broad antiviral capabilities in a
wide range of in vitro models including multiple
arenaviruses, filoviruses, Zika virus, and HIV. The Company looks
forward to the potential for additional externally funded research
to confirm such activity.
Expected Upcoming Milestones
- Report preliminary findings of National Institutes of Health
(NIH) funded animal testing of WP1096 in the Tacaribe
Arenavirus.
- Identify investigators interested in initiating a clinical
trial to study the safety, pharmacokinetics, and efficacy of oral
WP1122 in adult patients with GBM.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a clinical stage pharmaceutical
company with a growing pipeline, including Phase 2 clinical
programs, for hard-to-treat tumors and viruses. The Company's lead
program, Annamycin is a next-generation anthracycline designed to
avoid multidrug resistance mechanisms with little to no
cardiotoxicity. Annamycin is currently in development for the
treatment of acute myeloid leukemia (AML) and soft tissue sarcoma
(STS) lung metastases.
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, and WP1220, an analog to WP1066, for the topical
treatment of cutaneous T-cell lymphoma. Moleculin is also engaged
in the development of a portfolio of antimetabolites, including
WP1122 for the potential treatment of COVID-19 and other viruses,
as well as cancer indications including brain tumors, pancreatic
and other cancers.
For more information about the Company, please
visit www.moleculin.com and connect on Twitter,
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 Expected Upcoming
Milestones set forth above, the pace of enrollment
in Moleculin's clinical trials, the timing of Moleculin's
ability to report topline data from its studies, the timing of the
commencement of investigator-sponsored and/or externally funded
clinical trials which are outside the control of Moleculin, and
whether the results of Moleculin's preclinical animal models can be
replicated in human trials. 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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SOURCE Moleculin Biotech, Inc.