- Annamycin in combination with Cytarabine (AnnAraC) achieves
CRc rate of 62% (54% CR) in 1st and 2nd line
AML subjects (N=13)
- Full MB-106 trial reaches 20 subjects with CRc of 45%
in 1st – 7th line, with median durability at 4.9 months and
climbing
- Interim data supports advancement to development of Phase 2
registration-directed clinical trial (MB-108) to further provide
data for efficacy to support an eventual application for New Drug
Approval (NDA)
- Virtual AML Clinical Day featuring internationally
renowned clinician, Martin S.
Tallman, MD, today, May
7th at 11:00 AM ET
- Register Here
HOUSTON, May 7, 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 the
presentation of positive interim data for Annamycin from its
ongoing acute myeloid leukemia (AML) clinical development program.
As previously announced, the Company will host a virtual AML
Clinical Day, today, May 7, 2024, at
11:00 AM ET (details below) to
discuss these results.
"Adding the latest two subjects in MB-106 AML trial with AnnAraC
brings the composite complete remission (CRc) rate up from 37% to
45% for all of the 20 subjects with prior therapies ranging from
none to six. This adds to our growing body of positive data which
continues to provide a clear line of sight for our clinical and
regulatory strategies towards registration for Annamycin in AML,"
commented Walter Klemp, Chairman and
Chief Executive Officer of Moleculin. "Focusing on 2nd
line subjects, a CRc rate of 60% 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. While we believe that the full trial results are
impressive, we continue to believe that our results in
2nd line subjects, where there is a significant unmet
need, are exceptional and believe the CRc rate demonstrated by
AnnAraC in 2nd line patients substantially exceeds the
performance reported by any drug currently approved for use in
2nd line AML."
"We continue to progress our preparations 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 early next year," added
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
clinical trial.
To date, 20 subjects have been enrolled in the full MB-106 Phase
1B/2 study. The median durability of
remission (DoR) is 4.9 months and climbing. DoR is defined as the
time from initiation of treatment to the point of disease relapse
or death. 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 for the added indication
of 1st line subjects, however, the Company does not
expect the addition of this cohort to delay the EoP2 meeting. The
study has recruited three 1st line subjects to date with
two achieving CRs.
Moleculin's current planned pathway for initial NDA approval
indication 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 of whether the subject was deemed "fit" or "unfit" for
intensive chemotherapy). Moleculin continues to recruit
1st line subjects into MB-106 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 is shown
below. These subjects had 0-6 prior therapies. The preliminary data
for MB-106 demonstrate a CRc rate of 45% and an overall remission
rate (ORR) of 55% for all subjects, regardless of the number of
prior treatments (N=20). Segmenting the MB-106 subject population
for 1st line (N=3) and, most notably, 2nd line (N=10) therapies in
the trial, yields a CRc rate of 67% and 60% and an ORR of 67% and
70%, both respectively.
Median durability for MB-106 is 4.9 months and climbing with one
death (suspected to be related to pneumonia) and one relapse to
date out of the 9 CRcs. The first subject with a CRc (and who has
yet to relapse) was treated in February
2023. 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 updated 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 initial 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). All data from MB-106 are interim,
preliminary, and subject to change until a clinical trial report is
published.
Table 1 - Summary of Annamycin Remissions
in MB-106 as of May 2, 2024
Study MB-106
Combination Therapy – Phase 1B/2 with
Ara-C + Annamycin "5+3"
|
All Lines (Range
1-7)
|
1st
Line
|
2nd
Line
|
1st and
2nd Line Combined
|
All
Subjects
|
|
|
|
|
Recruited and
Evaluable
|
20
|
3
|
10
|
13
|
Subjects Evaluable Not
Dosed Per Protocol
|
2
|
0
|
1
|
1
|
Median Prior
Therapies
|
1
|
0
|
1
|
1
|
Median Age - Years
(Range)
|
69 (19-78)
|
49 (19-69)
|
71 (53 - 78)
|
67 (19-78)
|
Complete Remissions
(CR)
|
8
|
2
|
5
|
7
|
CR with incomplete
recovery (CRi)
|
1
|
0
|
1
|
1
|
Total Composite
Complete Remission (CRc)
|
9
|
2
|
6
|
8
|
Complete Remission (CR)
Rate
|
40 %
|
67 %
|
50 %
|
54 %
|
Complete Remission
Composite (CRc) Rate
|
45 %
|
67 %
|
60 %
|
62 %
|
Partial Remissions
(PRs)
|
2
|
0
|
1
|
1
|
Overall Response Rate
(CRc's + PRs) or ORR
|
55 %
|
67 %
|
70 %
|
69 %
|
CRc Relapsed or Death
to Date
|
2
|
0
|
2
|
2
|
BMT To Date (in
CR's)
|
2
|
0
|
1
|
1
|
See Note 1
below
|
|
|
|
|
Notes for Table 1: Data from MB-106 is for Intent To
Treat (ITT) subjects and is preliminary and subject to change.
Expected Milestones for Annamycin AML Development
Program
- H1 2024: Complete MB-106 Phase 1B/2 clinical trial.
- H1 2024: MB-106 End of Phase 2 Meeting with FDA.
- H1 2025: Initiate pivotal trial.
- H2 2026 Conclude pivotal trial.
- 2027: Potential NDA submission.
Virtual AML Clinical Day Details
As previously announced, Moleculin will host a virtual AML
Clinical Day today, Tuesday, May 7,
2024 at 11:00 AM ET. For the
event, Walter Klemp, Chairman and
Chief Executive Officer, and Dr. J. Paul
Waymack, Senior Chief Medical Officer of Moleculin will be
joined by key opinion leader, Dr. Martin
Tallman, an internationally renowned clinical investigator
whose discoveries have fueled the progress of leukemia-targeting
therapies, most recently with the Robert H. Lurie Comprehensive
Cancer Center of Northwestern
University. Dr. Tallman is a member of our Science Advisory
Board.
A live video webcast of the event will be available on the
Events page under the Investors section of the Company's website
(moleculin.com). A webcast replay will be available two hours
following the live event and will be accessible 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. All interim and
preliminary data discussed above is subject to change.
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 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 timing of the commencement
of a pivotal registration study of Annamycin as a
2nd line therapy in AML in the first half of 2025.
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
SHDOCS:220926192.6
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