Moleculin(US:MBRX)2024-05-10 20:30Clinical Trial Results - Annamycin has shown a complete remission (CR) rate of 40% and an overall response rate (ORR) of 55% across all subjects in the Phase 1B/2 trial for AML, with a CR rate of 67% for first-line and 60% for second-line therapies [69]. - The median durability of complete remission (CRc) is approximately 4.9 months, with one death and one relapse reported out of ten CRs [73]. - Annamycin demonstrated no cardiotoxicity in subjects treated, even at doses exceeding the lifetime cumulative anthracycline dose associated with increased cardiomyopathy risk [74]. - The Phase 1B/2 trial for Annamycin in combination with Cytarabine is expected to support a Phase 2 registration-directed clinical trial for New Drug Approval (NDA) [71]. - The company has engaged an independent expert to assess the cardiovascular safety of Annamycin, with ongoing data collection from multiple trials [63]. Regulatory and Patent Information - Annamycin has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for the treatment of AML, enhancing its commercial exclusivity [78]. - The U.S. Patent and Trademark Office issued U.S. Patent number 11,951,118 for Annamycin, with coverage extending until June 2040, subject to regulatory approval timelines [76]. Financial Performance - Revenues for the three months ended March 31, 2024, were $0, consistent with the same period in 2023 [85]. - Research and development expenses decreased to $4.3 million in Q1 2024 from $5.7 million in Q1 2023, a reduction of approximately 24.6% [86]. - General and administrative expenses decreased to $2.4 million in Q1 2024 from $2.6 million in Q1 2023, a reduction of approximately 7.7% [87]. - The net loss for Q1 2024 was $4.97 million, compared to a net loss of $7.92 million in Q1 2023, representing a 37.4% improvement [85]. - Cash used in operating activities increased to $6.72 million in Q1 2024 from $6.02 million in Q1 2023, an increase of approximately 11.7% [91]. - The company recorded a net gain of $1.5 million from the change in fair value of warrant liability in Q1 2024, compared to a gain of $0.04 million in Q1 2023 [88]. - As of March 31, 2024, the company had $0.4 million in cash on hand in Australia, with no known liquidity limitations [90]. - The company expects existing cash and cash equivalents to fund operations into Q4 2024 without additional equity issuance [93]. - The company expensed approximately $0.1 million in general and administrative fees related to an SEC investigation in Q1 2024, down from $0.5 million in Q1 2023 [94]. - The company did not engage in any stock sales during Q1 2024, resulting in no cash provided by financing activities [92]. Research and Development Initiatives - The WP1066 portfolio includes three compounds that have successfully completed Phase 1 clinical trials, with ongoing discussions for further trials in glioblastomas and pediatric brain tumors [81]. - The company is exploring external funding avenues for the WP1122 portfolio, with a final clinical study report submitted in late October 2023 [83]. - Annamycin's unique lipid-based delivery technology aims to improve tissue distribution and reduce toxicity compared to traditional anthracyclines [64].