Moleculin(MBRX) - 2025 Q2 - Quarterly Results

Q2 2025 Financial Results and Corporate Highlights The company highlights significant Q2 2025 progress in its pivotal MIRACLE trial and positive Annamycin data Overview Moleculin advanced its pivotal Phase 2B/3 MIRACLE trial for AML and reported positive data for Annamycin in soft tissue sarcoma - The company is actively recruiting for its Phase 2B/3 MIRACLE trial, with sites now open in the US and EU[1] - Moleculin expects to have over 20 additional clinical sites in the EU and US recruiting for the MIRACLE trial by the end of Q3 2025[1][5] - The initial pivotal data readout from Part A of the MIRACLE trial is targeted for before the end of 2025[1][2] - The company reported compelling topline data from its Phase 1B/2 trial of Annamycin for soft tissue sarcoma lung metastases[2] Clinical Development Update The company details advancements in its key clinical programs, including Annamycin for AML and STS, and WP1066 for brain tumors Annamycin Program The Annamycin program is advancing with its pivotal MIRACLE trial, positive STS data, and strengthened intellectual property - Annamycin has received Fast Track and Orphan Drug Designations from the FDA for AML, and Orphan Drug Designation for soft tissue sarcoma[12] - The company bolstered its Annamycin intellectual property with a notice of intent to grant a new European patent[5] - Engaged industry veteran Adriano Treve to explore strategic partnerships related to Annamycin[5] Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) - MIRACLE Trial The global pivotal MIRACLE trial for R/R AML is expanding sites, with key data readouts expected before year-end 2025 - The MIRACLE trial is a global, pivotal Phase 2B/3 study evaluating Annamycin in combination with Cytarabine (AnnAraC) for R/R AML[3][4] - Trial Progress: Four sites are actively screening subjects in Ukraine, Georgia, Spain, and the US, with over 20 additional sites expected by the end of Q3 2025[5] - The FDA provided positive feedback on a pediatric study plan for Annamycin in children with R/R AML, with the trial set to start in 2027[5] Key MIRACLE Trial Milestones | Milestone | Expected Timeline | | :--- | :--- | | First patients treated in US and EU | Q3 2025 | | Data readout (n=45) unblinded review | Q4 2025 | | Interim data (n=~75-90) & Optimum Dose set | 1H 2026 | | Begin enrollment of 3rd line subjects | 2027 | | Begin Rolling NDA Submission for R/R AML | 2028 | Soft Tissue Sarcoma (STS) Lung Metastases Positive topline results from the Phase 1B/2 trial showed a favorable median overall survival for Annamycin in STS lung metastases - The Phase 1B/2 trial (MB-107) demonstrated a median overall survival (OS) of 13.5 months for subjects who were on a median 7th line of therapy[11] - This OS of 13.5 months compares favorably to the 8-12 months for standard of care treatments in advanced STS as a 2nd line therapy[11] - The company expects to identify the next phase of development or a pivotal investigator-initiated trial program in Q4 2025[12] WP1066 & Brain Tumors The WP1066 program is advancing through an investigator-initiated trial for glioblastoma and a preclinical collaboration for IV formulations - An externally funded Phase 1B/2 trial of oral WP1066 in combination with radiation for glioblastoma (GBM) is actively recruiting at Northwestern University[13] - A preclinical study agreement is in place with Emory University to evaluate various WP1066 IV formulations, with results expected in H2 2025[13] Q2 2025 Financial Results The company reports its second-quarter financial performance, including operating expenses, cash position, and consolidated statements Financial Performance Summary The company reported lower R&D expenses and ended Q2 2025 with $7.6 million in cash, sufficient to fund operations into Q4 2025 Q2 2025 Operating Expenses (in millions) | Expense Category | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Research & Development | $3.6 | $4.1 | -$0.5 | | General & Administrative | $2.1 | $2.1 | $0.0 | - The company held $7.6 million in cash and cash equivalents as of June 30, 2025[15] - Current cash reserves are expected to fund planned operations into the fourth quarter of 2025[15] Condensed Consolidated Financial Statements Unaudited statements show increased liabilities and a net loss of $7.6 million for the quarter ending June 30, 2025 Condensed Consolidated Balance Sheet (in thousands) | Account | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $7,557 | $4,278 | | Total current assets | $9,076 | $5,194 | | Total assets | $21,593 | $16,925 | | Total current liabilities | $7,913 | $5,359 | | Total liabilities | $28,758 | $10,946 | | Total stockholders' equity (deficit) | $(7,165) | $5,979 | Condensed Consolidated Statement of Operations (in thousands, except per share data) | Account | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Research and development | $3,600 | $4,090 | | General and administrative | $2,120 | $2,095 | | Loss from operations | $(5,720) | $(6,185) | | Net loss | $(7,640) | $(4,319) | | Net loss per common share | $(0.49) | $(1.70) | Corporate Information An overview of the company's focus as a clinical-stage entity developing treatments for hard-to-treat cancers and viruses Company Overview Moleculin is a Phase 3 company developing treatments for difficult cancers, led by its next-generation anthracycline, Annamycin - Moleculin is a Phase 3 clinical-stage company with a pipeline targeting difficult cancers and viruses[16] - Lead program Annamycin is a next-generation anthracycline designed to avoid cardiotoxicity and multidrug resistance[16] - Other pipeline assets include WP1066, an Immune/Transcription Modulator for brain tumors, and WP1122, an antimetabolite for viruses and cancers[19]