Moleculin(US:MBRX)2025-11-13 21:16Clinical Trials and Drug Development - The company is conducting a pivotal Phase 3 trial for Annamycin in combination with Cytarabine for the treatment of relapsed/refractory acute myeloid leukemia (AML), with an interim unblinding expected after recruiting the first 45 subjects[85]. - As of November 4, 2025, 60% of the targeted 45 subjects have consented to participate in the MIRACLE trial, with treatment of the last subject expected in Q1 2026[98]. - Annamycin has been administered to 84 subjects across five clinical trials with no evidence of cardiotoxicity, including subjects treated above the FDA's lifetime maximum anthracycline limit of 550 mg/m2[93]. - The company has a total of fourteen clinical trials, with ongoing studies for Annamycin and other drug candidates, including a Phase 2B/3 trial for AML and a Phase 1B/2 trial for soft tissue sarcoma[96]. - The MIRACLE trial is designed as an adaptive study with a lead-in Part A to determine the optimum dose of Annamycin, followed by Part B for further evaluation[97]. - The company has received ten communications from the FDA regarding the MIRACLE trial, focusing on safety and monitoring, with no significant changes to the trial design or dosing of Annamycin[101]. - Annamycin is designed to be non-cardiotoxic and has shown no cardiotoxicity in clinical trials, which is a significant advantage over currently prescribed anthracyclines[89]. - The MIRACLE trial protocol was amended to allow dosing above the lifetime maximum allowable dose for anthracyclines, with FDA feedback reducing the size of Part B of the Phase 3 trial to 222 subjects, a 10% reduction[102]. - The European Medicines Agency approved the Clinical Trial Application for the MIRACLE trial in nine EU countries, contingent on presenting results of nonclinical GLP studies before initiating Part B[104]. - In the Phase 1B/2 clinical trial of AnnAraC for AML, 36% of subjects achieved complete remission, with a median durability of 10 months for those in remission[106]. - The FDA approved a single pediatric study plan for Annamycin in combination with Cytarabine for children with relapsed/refractory AML, expected to commence in the second half of 2027[108]. - The MB-107 trial for Annamycin in STS Lung Mets showed a median overall survival of 13.5 months, compared to 8-12 months for standard treatments[110]. - Annamycin is being evaluated for pancreatic cancer in a grant-funded research project at UNC, with a Phase 1B/2 study planned to start in 2026[113][115]. Financial Performance - Research and development (R&D) expenses decreased to $3.7 million in Q3 2025 from $4.9 million in Q3 2024, a reduction of approximately 24.5%[134]. - General and administrative expenses slightly decreased to $2.1 million in Q3 2025 from $2.2 million in Q3 2024, a decrease of about 4.6%[135]. - The net loss for Q3 2025 was $25.4 million, compared to a net loss of $7.0 million in Q3 2024, indicating a significant increase in losses[133]. - The company recorded a net gain of $1.6 million from the change in fair value of warrant liability in Q3 2025, compared to no gain in Q3 2024[136]. - Cash used in operating activities was $17.4 million for the nine months ended September 30, 2025, a decrease of $1.4 million from $18.8 million in the prior year[145]. - The company raised gross proceeds of $6.0 million from the exercise of Series E warrants in August 2025[146]. - The company reported a net increase in cash and cash equivalents of $2.4 million for the nine months ended September 30, 2025, compared to a decrease of $14.1 million in the prior year[144]. - The company must seek additional funds of approximately $7 million to support operations into the second quarter of 2026[152]. Intellectual Property and Collaborations - The Australian Patent Office granted a patent for preliposomal Annamycin lyophilizate, extending the patent term until June 2040[122]. - The World Health Organization approved "naxtarubicin" as the international non-proprietary name for Annamycin, facilitating its recognition in clinical documents[125]. - The US Patent and Trademark Office granted two additional patents covering methods of making liposomal Annamycin, extending the patent term until June 2040[127]. - The company received a Notice of Intent to Grant for a European patent for "Method of Reconstituting Liposomal Annamycin," enhancing global exclusivity with a base patent term extending until June 2040[128]. - The company has additional patent applications related to Annamycin pending in major jurisdictions worldwide[128]. - The company has established collaborations with the University of Texas MD Anderson Cancer Center for research and development of its drug candidates[84]. Regulatory and Compliance - The company received a subpoena from the SEC in March 2022 regarding information related to its COVID-19 drug candidate, with expenses of approximately $0.1 million and $0.2 million for the three and nine months ended September 30, 2024, respectively[153]. - There have been no material changes to the company's critical accounting policies and estimates from those disclosed in the Form 10-K for the year ended December 31, 2024[154]. - The company is classified as a smaller reporting company, and thus, quantitative and qualitative disclosures about market risks are not applicable[156]. Strategic Focus and Growth - The company plans to expand treated indications with Annamycin in 2026, indicating a focus on growth and market expansion[86]. - The company aims to attract and retain key personnel to effectively manage its business and support future growth[84].