– Annamycin in
combination with Cytarabine (AnnAraC) achieves CRc rate of 60% (50%
CR) in 2nd line AML subjects
– AnnAraC has
potential to fill significant unmet need and expected to increase
2nd line AML CRs more than double over existing
2nd line therapies
– All 82 Annamycin
subjects (in multiple studies) continue to show no signs of
cardiotoxicity during study; Lower toxicity profile than
traditional intensive therapy
– Annamycin is
advancing towards pivotal AML study in 2024 and may qualify for an
accelerated approval pathway
– Company to host
conference call and webcast today, March
25th at 8:30 AM
ET
HOUSTON, March 25,
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, provided a preliminary
update on recent clinical activity and expected near term
milestones for its lead program MB-106 for the treatment of Acute
Myeloid Leukemia (AML) following its Fiscal Year 2023 filing with
the Securities and Exchange Commission. As previously announced,
the Company will host a conference call and live audio webcast,
today, March 25, 2024, at
8:30 AM ET (details below).
"We remain highly encouraged by the positive data seen to-date
in our Phase 1B/2 AnnAraC trial for
AML," commented Walter Klemp,
Chairman and Chief Executive Officer of Moleculin. "With a CRc rate
of 60% as 2nd line therapy and with 50% of those being
full CRs, AnnAraC has the potential to offer 2nd line
patients a viable alternative, regardless of prior treatments or
mutations. Importantly, we recently visited prominent key opinion
leaders in Europe and the U.S.
Without exception, they each believe there remains a significant
unmet need for 2nd line AML therapies, that our results
in 2nd line subjects are significant, and that, if
approved, they would use Annamycin in their practice. We are seeing
genuine enthusiasm for how Annamycin might change the
paradigm."
"Understanding the continuing unmet need in AML is key to
understanding Moleculin's opportunity. It is so key that we created
an informative animation of the patient journey to help investors
realize our potential in AML. In brief, even though
tremendous effort has been focused on targeted therapies in AML and
five new drugs have been approved for 2nd line use, they
are only expected to result in a positive outcome for about 7% of
the total AML population. We believe that leaves nearly 60% of AML
patients without a viable path to lasting remission. At the rate
indicated by our latest data, Annamycin could more than double the
number of 2nd line patients achieving a complete
response from all the currently approved targeted therapies
combined," added Mr. Klemp.
"Our growing body of data are now propelling our clinical and
regulatory strategies toward our next phase of development. We are
preparing for an End of Phase 2 meeting with FDA and believe
following this meeting we will be in a position to commence a
pivotal registration study as a 2nd line therapy in AML
before year end," concluded Mr. Klemp.
Ongoing AML Clinical Trial Overview
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.
During 2023, Moleculin commenced its MB-106 clinical trial with
AnnAraC for the treatment of AML in an all-comers trial, accepting
subjects without regard to the number of prior therapies in the
Phase 1 portion with a limit of two prior therapies in the Phase 2
portion. Nine clinical sites in Poland and Italy have been activated for the MB-106.
To date, 20 subjects have been enrolled in the full MB106 Phase
1B/2 study with two subjects not yet
evaluable. At the end of January
2024, the Company completed recruiting the desired number of
2nd line subjects and began preparation for an End of
Phase 2 (EoP2) meeting with the FDA. In addition, Moleculin
expanded the MB-106 study protocol to include 1st line
subjects to provide data to enable the designing of a potential
confirmatory Phase 3 post-approval study, however, do not expect
the addition of this cohort to delay the EoP2 meeting. The study
has recruited three 1st line subjects to date with one
CR and one not yet evaluable.
Moleculin's current planned pathway for approval for Annamycin
in combination with Cytarabine for the treatment of AML is as a
2nd line therapy. Therefore, the focus is primarily on
securing an accelerated approval pathway for the treatment of
2nd line subjects (those who were relapsed from or
refractory to a 1st line AML therapy, regardless as to
whether the subject was deemed "fit" or "unfit"). Moleculin has
begun recruiting 1st line subjects into this trial to
provide data for a possible future confirmatory Phase 3 clinical
trial in 1st line patients.
Summary of MB-106 Data
A summary table of the MB-106 preliminary results, which are
subject to change, is shown below. The total CRs in both Phases to
date in MB-106 represent a complete response composite (CRc) rate
of 39% in all currently evaluable, intent to treat subjects (n= 18)
with two additional subjects recruited not yet evaluable. These
subjects had 0-6 prior therapies. This is comprised of a CR rate of
33% and CRi of 6%. Durability data are developing with one CRc having relapsed to date. The first CR
subject was treated in February 2023
and remains durable after over a year and continues in remission.
Durability of CRs is confirmed by repeat bone marrow aspirates
(BMAs). The median age of all subjects recruited is 69, ranging
from 19 to 78. Median durability will be established as the trial
data mature. This trial may enroll up to 28 subjects,
however, having already recruited the desired number of
2nd line subjects to support an EoP2 meeting with the
FDA, the Company may elect to complete this trial with fewer than
28 subjects.
The trial continues recruitment for treatment as 1st
line and 3rd line therapy. Since Moleculin intends to
position AnnAraC for approval as a 2nd line therapy, the
Company believes that the most important data from this trial are
the results in 2nd line subjects (excluding subjects who
are either 1st line or 3rd line and beyond).
When stratified for that population (n=10), the CRc rate is 60%,
being comprised of a CR rate of 50% and a CRi of 10%.
Table 1 - Summary of Annamycin Responses
in MB-106 as of March 19, 2024
Study
|
Study MB-106
Combination Therapy–
Phase 1B/2 All Lines
(Range 1-7)
|
Study MB-
106Combination
Therapy– Phase 1B/2 As
1st Line
|
Study MB-
106Combination
Therapy– Phase 1B/2 As
2nd Line
Only
|
All
Subjects
|
|
|
|
Recruited
|
20
|
3
|
10
|
Subjects Not Yet
Evaluable
|
2
|
1
|
0
|
Subjects Evaluable
To Date
|
18
|
2
|
10
|
Subjects Evaluable
Not
Dosed Per Protocol
|
2
|
0
|
1
|
Median Prior
Therapies
|
1
|
0
|
1
|
Median Age -
Years
(Range)
|
69 (19-78)
|
49 (19-69)
|
71 (53 - 78)
|
Complete
Responses
(CR)
|
6
|
1
|
5
|
CR with
incomplete
recovery (CRi)
|
1
|
0
|
1
|
Total Complete
Response(s) (CRc)
|
7
|
1
|
6
|
Complete Response
(CR)
Rate
|
33 %
|
50 %
|
50 %
|
Complete
Response
Composite (CRc) Rate
|
39 %
|
50 %
|
60 %
|
Partial Responses
(PRs)
|
2
|
0
|
1
|
CRc Relapsed To
Date
|
1
|
0
|
1
|
BMT To
Date
|
1
|
0
|
1
|
Durability of
CR
|
Developing
|
Developing
|
Developing
|
|
(1,2,3,4)
|
(2,3,4)
|
(2,3,4)
|
Notes for Table 1: 1) The 19th subject, being
treated as a 3rd line therapy, had two BMAs tested
where they have been inconclusive, and we are awaiting further
testing. This subject will move the CRc in the "MB-106 Phase
1B/2 All Lines" column to either 42%
or 37% (n=19). The 20th subject is a first-line therapy
subject and has just begun treatment; 2) Data from MB-106 is for
intent to Treat subjects; 3) Data from MB-106 is
preliminary and subject to change; and 4) Durability is
developing.
Expected 2024 MB-106 Milestones
- Complete MB-106 Phase 1B/2
clinical trial.
- Present topline data from MB-106 clinical trial.
- Report outcome of MB-106 End of Phase 2 Meeting.
- Initiate pivotal program.
Conference Call and Webcast
Moleculin management will host its quarterly conference call and
webcast for investors, analysts, and other interested parties
Monday, March 25, 2024, at
8:30 AM ET.
Interested participants and investors may access the conference
call by dialing (877) 407-0832 (domestic) or (201) 689-8433
(international) and referencing the Moleculin Biotech Conference
Call. The live webcast will be accessible on the
Events page of the Investors section of the Moleculin
website, moleculin.com, and will be archived for 90 days.
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 relapsed or refractory 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.