On August 14, 2024 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, reported its financial results for the quarter ended June 30, 2024 (Press release, Moleculin, AUG 14, 2024, View Source [SID1234645923]).

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"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 EHA (Free EHA Whitepaper)2024 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.