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Yahoo
12-05-2025
- Business
- Yahoo
Moleculin Receives European Medicines Agency Approval to Expand Phase 3 MIRACLE Clinical Trial
Adds nine additional countries to the Company's ongoing pivotal Phase 3 trial; Authorization granted in all EU countries requested Enrollment and dosing underway in Phase 3 clinical trial (the 'MIRACLE' trial) evaluating Annamycin for the treatment of R/R AML; Interim data readout expected in the second half of 2025 Subjects targeted for the 'MIRACLE' trial include venetoclax regimen failures where outcomes with currently available therapies are considered dismal HOUSTON, May 12, 2025 (GLOBE NEWSWIRE) -- 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, today announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as 'Ara-C' and for which the combination of Annamycin and Ara-C is referred to as 'AnnAraC'). The study is for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML) and is approved in all nine countries submitted in the European Union (EU). This Phase 3 'MIRACLE' trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East. 'EMA approval of the MIRACLE trial protocol is a huge milestone for us. Although we're already seeing recruitment in our first non-EU country, we believe that this expansion into the EU really supercharges our recruitment potential,' said Walter Klemp, Chairman and CEO of Moleculin. 'Importantly, when combined with the sites we are opening in the US, this approval from the EMA, along with the individual country committee and/or ethics approvals, for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain positions us to remain on track with our expected enrollment and data milestones.' Mr. Klemp continued: 'Being accepted in all nine of the countries for which we submitted, we believe indicates the magnitude of the need for a better answer for R/R AML patients, especially venetoclax regimen failures where the outcomes from currently available therapies are considered dismal in published studies. While there are minor differences between the US and EU protocols with the FDA and EMA, respectively, we do not view these as a barrier to conducting the study and are working to harmonize the protocols as appropriate. We are grateful for the international collaboration and believe it underscores the significant unmet need in R/R AML and the potential of Annamycin to provide a much needed second line treatment option. We remain focused on driving enrollment and patient dosing and look forward to reporting initial data on the first 45 subjects in the second half of 2025.' 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 and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on and the reference identifier there is 2024-518359-47-00. Annamycin, also known by its non-proprietary name of 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 EMA. About Moleculin Biotech, Inc. Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company's lead program, Annamycin, is a next-generation highly efficacious and well tolerated anthracycline designed to avoid multidrug resistance mechanisms and to lack the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. The Company is initiating the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study remains subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents. Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin is also engaged in the development of a portfolio of antimetabolites, including WP1122 for the potential treatment of pathogenic viruses, as well as certain cancer indications. For more information about the Company, please visit and connect on X, LinkedIn and Facebook. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the timing of the release of the initial data on the first 45 subjects in the trial and the Company's ability to harmonize the US and EU protocols with the FDA and EMA, respectively. Moleculin will require significant additional financing, for which the Company has no commitments, in order to conduct its clinical trials as described in this press release, and the milestones described in this press release assume the Company's ability to secure such financing on a timely basis. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including 'believes,' 'estimates,' 'anticipates,' 'expects,' 'plans,' 'projects,' 'intends,' 'potential,' 'may,' 'could,' 'might,' 'will,' 'should,' 'approximately' or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. 'Risk Factors' in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. Investor Contact:JTC Team, LLCJenene Thomas(908) 824-0775MBRX@
Yahoo
25-03-2025
- Business
- Yahoo
Moleculin Biotech Inc (MBRX) Q4 2024 Earnings Call Highlights: Promising Clinical Trials and ...
Cash Balance: Approximately $13 million as of the end of the year, including $9 million raised in February 2025. Operating Expenses: Reduced by about $3 million in 2024 compared to 2023. Market Cap: $16.2 million with 14 million shares outstanding. Phase 2 Clinical Trial Results: 50% complete remission rate in second-line AML patients treated with Annamycin plus high-dose Cytarabine. Overall Survival: 11 months for second-line therapy patients in the Phase 2 trial. Progression-Free Survival: Median of 9 months in the Phase 2 trial. Phase 3 MIRACLE Trial: Designed to compare Annamycin plus high-dose Cytarabine against placebo plus high-dose Cytarabine. Interim Data Unblinding: Planned after 45 and between 75-90 subjects in the Phase 3 trial. Shares Trading Volume: Trailing one-year trading volume of 1.4 million shares per day. Warning! GuruFocus has detected 1 Warning Sign with MBRX. Release Date: March 24, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Moleculin Biotech Inc (NASDAQ:MBRX) has initiated the MIRACLE Phase 3 pivotal trial for Annamycin, targeting relapsed and refractory AML patients, with 25 sites selected globally. The company has regulatory and ethics approval in its first European country and expects more approvals in the second quarter. Annamycin has shown promising Phase 2 data with a 50% complete remission rate in second-line AML patients, outperforming existing therapies. The MIRACLE trial includes multiple unblindings of data, providing stakeholders with visibility into the trial's progress. Moleculin Biotech Inc (NASDAQ:MBRX) has reduced operating expenses by $3 million in 2024 compared to 2023, demonstrating financial discipline. The US is expected to be one of the last countries to begin enrolling in the MIRACLE trial due to longer approval processes. The company faces a significant financial challenge, with the Phase 3 trial potentially costing upwards of $60 million to $70 million. Moleculin Biotech Inc (NASDAQ:MBRX) has a limited cash runway, with funds expected to last only until the third quarter of 2025. There is uncertainty regarding which Annamycin dosing regimen will be most effective, requiring further data analysis. The company acknowledges the complexity of achieving early approval, as it requires a very statistically significant result in Part A of the trial. Q: What efficacy is required to pick one Annamycin dose at 45 patients rather than waiting until 90 if it doesn't happen at 45? A: Walter Klemp, CEO: If Annamycin performs as well in Part A as it did in the Phase 2 trial, and HiDAC performs as expected, we might hit the statistical significance required to accelerate approval. However, HiDAC might underperform due to being limited to one cycle. Paul Waymack, Senior Chief Medical Officer, added that it's a multivariable equation, and while early termination is possible, it's not highly likely. Q: Towards the end of the trial, what was the thinking that went into cutting off about 10% of patients from Part D? A: Walter Klemp, CEO: The FDA suggested a different biostatistical scheme during protocol review, which allowed us to reduce the number of patients. Q: What is the STS lung met efficacy bogey you need to hit to proceed to a pivotal trial? A: Walter Klemp, CEO: We've already hit that. The STS patients treated were more challenged than expected, yet we achieved OS numbers typical of first-line patients. This has attracted interest from sarcoma experts, and a pivotal trial is being considered. Q: What are the overall costs of the trial? A: Jonathan Foster, CFO: The full patient load for the Phase 3 trial could cost $60-$70 million. Our cash burn for 2025 is $5 million per quarter, increasing to $7-$8 million in 2026 as we prepare for an NDA. Q: What are your thoughts on moving Annamycin to frontline AML treatment? A: Paul Waymack, Senior Chief Medical Officer: Once we document efficacy and file our NDA, we plan to explore multiple areas, including first-line therapy. Annamycin's lack of cardiotoxicity and resistance issues makes it suitable for first-line therapy, especially for unfit patients. Q: What was the rationale for choosing the 190 mg/m dose for the MIRACLE trial? A: Paul Waymack, Senior Chief Medical Officer: The 230 mg/m dose showed great results in the 106 study. The FDA's Project Optimus led us to include a lower 190 mg/m dose, which also showed efficacy. Both doses are expected to perform similarly. Q: Regarding the 190 mg/m dose, do you need to be superior to the 17%-18% CR rate observed in other trials to apply for early approval? A: Paul Waymack, Senior Chief Medical Officer: If one dose is clearly superior, the comparison would be against placebo. For early approval after Part A, the P-value would need to be less than 0.01, considering the totality of data. Q: Will you preserve the 190 mg/m dose in case of safety signals with the 230 mg/m dose? A: Walter Klemp, CEO: We have enough experience with Annamycin to be confident in its safety profile. However, if 230 mg/m becomes problematic, we could revert to 190 mg/m. By the end of Part A, we expect to have enough data to make an informed choice. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
13-02-2025
- Business
- Yahoo
Moleculin Biotech gains approval for enrolment in trial of AML therapy in Ukraine
US-based pharmaceutical company Moleculin Biotech has received approval from Ukraine's Ministry of Health to begin enrolment in its Phase III trial of annamycin with cytarabine for treating acute myeloid leukaemia (AML). The MIRACLE trial will take place at sites across Europe, the Middle East and the US, enrolling AML patients who are refractory to or relapsed post-induction therapy. It will follow an adaptive design, initially randomising the first 75-90 participants in Part A to receive high-dose cytarabine with either a placebo or one of two doses (190 mg/m² or 230 mg/m²) of Annamycin, as recommended by the Food and Drug Administration (FDA). Moleculin said that the amended protocol enables the unblinding of preliminary primary efficacy data and safety/tolerability outcomes for the three treatment arms at 45 participants. This early unblinding will involve 30 participants receiving the combination of annamycin and cytarabine, while the other 15 will only be given high-dose cytarabine. The first unblinding is anticipated in the second half of this year, with a second to follow early next year. Part B of the trial will involve nearly 244 additional participants and will aim to determine the optimum dose of annamycin based on safety, pharmacokinetics and efficacy. Moleculin chairman and CEO Walter Klemp said: 'We continue to make solid progress across our site initiation and enrolment efforts. 'Achieving this important milestone of receiving our first country's approval puts us another step closer to getting this study well underway and further bolsters our confidence that we're on track for unblinded preliminary data from the first 45 subjects in the second half of this year.' Based in Texas, Moleculin Biotech develops therapeutic candidates for a range of tumours and viruses that are considered difficult to treat. In May last year, the company began a Phase II trial of WP1066 with radiation therapy for glioblastoma. "Moleculin Biotech gains approval for enrolment in trial of AML therapy in Ukraine" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
