On May 13, 2024 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, reported its financial results for the quarter ended March 31, 2024 (Press release, Moleculin, MAY 13, 2024, View Source [SID1234643150]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results at 8:30 AM ET on Monday, May 13, 2024. (details below).
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"We recently hosted a virtual AML Clinical Day featuring renowned KOLs, Dr. Martin Tallman and Dr. Michael Andreeff. It was great to hear their support of and thoughts on Annamycin’s recent developments in AML. You can hear that discussion here. Combined with the encouraging Annamycin data demonstrated to date and the positive feedback from the KOLs, we continue to believe that our results in 2nd line subjects fill a significant unmet need. We believe this is supported by the exceptional CRc rate demonstrated by AnnAraC in 2nd line patients, which substantially exceeds the performance reported by any drug currently approved in the U.S. for use in 2nd line AML. We believe we are well-positioned for a highly productive End of Phase 2 meeting with FDA to discuss our planned pivotal study of Annamycin for AML," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.
Recent Highlights
Bolstered the patent estate with issuance of U.S. patents. We recently received an Issue Notification from the United States Patent and Trademark Office (USPTO) for U.S. Patent number 11,980,634 titled, "Method of Reconstituting Liposomal Annamycin." Earlier, we received an Issue Notification titled, "Preparation of Preliposomal Annamycin Lyophilizate." Both provide claims to compositions that contain Annamycin with a base patent term extending until mid-2040.
Hosted a virtual AML clinical day highlighting positive interim data for Annamycin from its ongoing acute myeloid leukemia (AML) clinical development program.
Full MB-106 trial reaches 20 subjects with CRc of 45% in 1st – 7th line, with median durability at 4.9 months and climbing.
1st and 2nd line patients reached a CRc rate of 62% and a CR rate of 54%.
The 2nd line performance from the combination of Annamycin with Ara-C (AnnAraC) exceeds the approval data for all currently approved 2nd line drugs.
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).
Announced the formation of its Annamycin Scientific Advisory Board and inaugural appointment of Dr. Martin Tallman.
Granted Orphan Drug Designation of Annamycin for the treatment of AML from the European Medicines Agency (EMA), adding to US Orphan Drug and Fast Track designations.
Presented positive data demonstrating high anti-cancer activity of Annamycin and non-cardiotoxic properties at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
Ongoing AML Clinical Trial Summary
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.
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.
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 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.
H2 2024: Feedback from the FDA EOP2 Meeting.
H1 2025: Initiate pivotal trial.
H2 2026: Conclude pivotal trial.
2027: Potential NDA submission.
Summary of Financial Results for the First Quarter 2024
Research and development (R&D) expense was $4.3 million and $5.7 million for the three months ended March 31, 2024 and 2023, respectively. The decrease of $1.4 million is mainly related to the clinical trials activity levels.
General and administrative expense was $2.4 million and $2.6 million for the three months ended March 31, 2024 and 2023, respectively. The decrease of $0.2 million is mainly related to a decrease in regulatory and legal fees.
As of March 31, 2024, the Company had cash and cash equivalents of $16.8 million and believes that this cash is sufficient to meet its projected operating requirements, which includes the Company’s current Phase 1B/2 clinical programs and preparations for future clinical trials, into the fourth quarter of 2024.
Conference Call and Webcast
Moleculin management will host its quarterly conference call and webcast for investors, analysts, and other interested parties Monday, May 13, 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 audio 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.