Moleculin Q1 2024 Financial Results and Corporate Update

Moleculin Biotech, Inc., a clinical-stage pharmaceutical firm, has announced promising interim data from its ongoing study involving Annamycin in combination with Cytarabine (referred to as AnnAraC) for treating acute myeloid leukemia (AML). The preliminary results revealed a complete response with incomplete hematologic recovery (CRc) rate of 62%, including a 54% complete response (CR) in first and second-line AML subjects. The MB-106 trial has enrolled 20 subjects to date.

The company will hold a conference call and webcast to discuss its quarterly financial results and recent achievements. The call is scheduled for Monday, May 13, 2024, at 8:30 AM ET.

Walter Klemp, Chairman and CEO of Moleculin, emphasized the significance of the positive Annamycin data in addressing unmet medical needs in second-line AML patients. The combination therapy with Cytarabine has shown impressive results, surpassing the efficacy of currently approved second-line treatments in the U.S. Klemp expressed optimism about the forthcoming End of Phase 2 meeting with the FDA to discuss their pivotal study plans for Annamycin in AML treatment.

Recent highlights for Moleculin include the issuance of new U.S. patents, advancing their intellectual property portfolio. They received U.S. Patent number 11,980,634, titled "Method of Reconstituting Liposomal Annamycin," and another patent titled "Preparation of Preliposomal Annamycin Lyophilizate." These patents extend the base term of protection for their compositions until mid-2040.

Moleculin also hosted a virtual AML Clinical Day, where key opinion leaders (KOLs) like Dr. Martin Tallman and Dr. Michael Andreeff shared their perspectives on Annamycin's progress in AML treatment. The company aims to build on its positive interim results as it advances towards a Phase 2 registration-directed clinical trial (MB-108).

Moreover, Moleculin announced the formation of its Annamycin Scientific Advisory Board with Dr. Martin Tallman as the inaugural appointee. The European Medicines Agency (EMA) has granted Orphan Drug Designation to Annamycin for AML treatment, adding to its existing U.S. Orphan Drug and Fast Track designations.

At the American Association for Cancer Research (AACR) Annual Meeting, the company presented data showcasing Annamycin's potent anti-cancer activity and non-cardiotoxic properties.

The ongoing Phase 1B/2 clinical trial (MB-106) is evaluating Annamycin combined with Cytarabine in AML patients, both as a first-line therapy and for those refractory or relapsed after induction therapy. Preliminary data indicates a CRc rate of 45% and an overall remission rate (ORR) of 55% among all participants, regardless of previous treatments. Specifically, first-line therapies showed a CRc rate of 67% and second-line therapies showed a CRc rate of 60%.

Upcoming milestones for the Annamycin AML development program include completing the MB-106 trial and holding an End of Phase 2 meeting with the FDA in the first half of 2024, initiating a pivotal trial in the first half of 2025, and potentially concluding the pivotal trial and submitting a New Drug Application (NDA) by 2027.

Financially, Moleculin reported research and development expenses of $4.3 million for the first quarter of 2024, down from $5.7 million in the same period in 2023. General and administrative expenses were also slightly lower at $2.4 million. As of March 31, 2024, the company held $16.8 million in cash and cash equivalents, sufficient to fund its operations into the fourth quarter of 2024.

Moleculin Biotech continues to develop its pipeline, which includes treatments for various hard-to-treat tumors and viruses. Their lead program, Annamycin, aims to overcome multidrug resistance and eliminate the cardiotoxicity associated with current anthracyclines. Other programs include WP1066 for brain tumors and pancreatic cancer, and WP1220 for cutaneous T-cell lymphoma. Additionally, WP1122 is in development for treating viruses and certain cancers.