On May 14, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported its financial results for the first quarter 2025 ended March 31, 2025 (Press release, Moleculin, MAY 14, 2025, View Source [SID1234653068]). As previously announced, the Company will host a conference call and live audio webcast to discuss the operational and financial results today, May 14, 2025 at 8:30 AM ET.
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"We are pleased with the continued progress of our pivotal, adaptive Phase 3 MIRACLE trial and remain encouraged by the Annamycin data demonstrated to date. In particular, with the sites opening in the US, the recent approval from the EMA, and the individual country committee and/or ethics approvals we have received for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain positions us to continue building momentum and remain on track with our expected enrollment and data milestones," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.
Mr. Klemp continued, "In addition to the progress with our AML program, we are seeing advancements across our pipeline. We continue to be encouraged by the MB-107 trial data demonstrated by Annamycin for the treatment of STS lung mets and expect to report final data readouts from that trial before the end of June. Additionally, investigator-initiated clinical and preclinical work continues on WP1066, our STAT3 inhibitor."
Recent Highlights
Received European Medicines Agency (EMA) approval for its Clinical Trial Application (CTA) to conduct Phase 3 MIRACLE clinical trial in all nine countries submitted in the European Union (EU);
Announced the International Nonproprietary Names (INN) Expert Committee of the World Health Organization approved "naxtarubicin" for the non-proprietary names of the Company’s next-generation anthracycline in development, Annamycin;
Bolstered Annamycin intellectual property portfolio with granting of two new U.S. patents: U.S. patent number 12,257,261 titled, "Preparation of Preliposomal Annamycin Lyophilizate" and U.S. patent 12,257,262 titled "Method of Reconstituting Liposomal Annamycin";
Presented new pre-clinical data for Annamycin demonstrating market expansion potential including treatment for pancreatic cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025; and
Commenced patient dosing in its ongoing pivotal, adaptive design Phase 3 MIRACLE trial.
Clinical Development Update
Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML)
The Company is currently evaluating Annamycin (naxtarubicin) in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") in 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 global, including sites in the US, Europe and the Middle East. As of the end of April 2025, 38 sites have been selected in all of the regions targeted, with 5 sites in the US.
The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.
The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding will yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) and HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.
The clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved protocol.
For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.
Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on clinicaltrials.eu, and the reference identifier there is 2024-518359-47-00.
Expected Milestones for Annamycin AML Development Program
1Q – 3Q 2025 – Update on MIRACLE trial site selection/approvals by countries
2025 – Recruitment update for MIRACLE trial
2H 2025 – Data readout (n=45) unblinded efficacy/safety review
2H 2025 – 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update
1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial
2027 – Begin enrollment of 3rd line subjects in MIRACLE2
2027 – Enrollment ends in 2nd line subjects
2027 – Begin enrollment in pediatric AML trial
2028 – Primary efficacy data for 2nd line subjects in MIRACLE
2028 – Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE
2028 – Primary efficacy data for 2nd line subjects
2028 – Rolling NDA submission begins
Soft Tissue Sarcoma (STS) Lung Metastases
As previously announced, the Company completed enrollment in the Phase 2 portion of its U.S. Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases. Subjects who had stable disease at the time of study discontinuation were followed for progression free response and overall survival. The clinical study report is finalized but has not yet been filed. The Company remains positive about the data and is expected to release the data by the end of June.
Expected Milestones for Annamycin STS Lung Mets Development Program
1H 2025 – Final MB-107 data readout
2025 – Identify next phase of development / pivotal IIT (investigator-initiated-trial) program
Annamycin (naxtarubicin) 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).
WP1066 & Brain Tumors
With regard to the Company’s WP1066 oral formula, the Company has an externally funded phase 1B/2 in combination with radiation treating glioblastoma (GBM), a form of brain cancer, at Northwestern University (Northwestern) that is actively recruiting. This is an investigator-initiated trial where Moleculin’s main cost is supplying drug product. To date Northwestern has recruited 7 subjects. No data has been released. Also, the Company has signed an agreement with Emory University enabling Emory to study various WP1066 IV formulations in preclinical studies with the goal of selecting the best molecule to move into a clinical setting towards, most likely, brain cancers such as GBM. Study drug was delivered in April 2025 to Emory with results from such studies expected in the second half of 2025.
Summary of Financial Results for the First Quarter 2025
Research and development (R&D) expense was $3.4 million and $4.3 million for the three months ended March 31, 2025 and 2024, respectively. The decrease of $0.9 million is mainly related to the clinical trials activity levels.
General and administrative expense was $2.5 million and $2.4 million for the three months ended March 31, 2025 and 2024, respectively. The increase of $0.1 million is mainly related to a slight overall increase in regulatory and legal fees.
As of March 31, 2025, the Company had cash and cash equivalents of $7.7 million and believes that the cash on hand is sufficient to fund planned operations into the third quarter of 2025.
Conference Call and Webcast
Moleculin management will host a conference call and live audio webcast to discuss the operational and financial results today, Wednesday, May 14, 2025 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.