This section highlights FDA-related milestones and regulatory updates for drugs developed by Moleculin Biotech (MBRX).
Over the past two years, Moleculin Biotech has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Annamycin, WP1066, Ara-C, and MB-106. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
Annamycin FDA Regulatory Timeline and Events
Annamycin is a drug developed by Moleculin Biotech for the following indication: Soft tissue sarcoma (STS) lung metastases.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Annamycin
- Announced Date:
- May 13, 2026
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced that it is approaching the first unblinding of data from its pivotal Phase 2B/3 "MIRACLE" trial evaluating Annamycin in combination with cytarabine for the treatment of subjects that have been relapsed or refractory to their primary line of treatment for acute myeloid leukemia (R/R AML).
AI Summary
Moleculin Biotech says it is nearing the first unblinding of data from its pivotal Phase 2B/3 MIRACLE trial of Annamycin plus cytarabine for patients with relapsed or refractory acute myeloid leukemia (R/R AML). Early blinded results from the first 45 patients show a complete remission rate of about 30%, which the company says is roughly 67% better than standard care. That first data unblinding is expected before June 30, 2026.
The trial is aimed at a high-need AML group, including patients with R/R Venetoclax. Recruitment for Part A is still moving toward 90 total patients, with 56 already enrolled and randomized. Moleculin says the continued lack of cardiotoxicity, along with the early signs of strong activity, could make Annamycin a major advance in AML treatment.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- March 23, 2026
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that the 45th subject has been enrolled in its pivotal Phase 2B/3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adult subjects with relapsed or refractory acute myeloid leukemia (R/R AML).
AI Summary
Moleculin Biotech announced that the 45th subject has been enrolled in its pivotal Phase 2B/3 MIRACLE trial testing Annamycin combined with cytarabine (AnnAraC) to treat adults with relapsed or refractory acute myeloid leukemia (R/R AML). The company described this enrollment milestone as progress toward completing the trial, which is designed to evaluate the combination’s safety and effectiveness in a difficult-to-treat patient population. Reaching 45 subjects helps sustain momentum in the study’s key development stage and supports ongoing data collection needed for future analysis.
For more details on the MIRACLE trial, see ClinicalTrials.gov using Identifier NCT06788756. Moleculin also continues development of other programs, including WP1066 (an immune/transcription modulator) and antimetabolite candidates such as WP1122, while advancing its oncology-focused pipeline.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- December 9, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., today provided an update on enrollment from its November report with an increase to 78% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 "MIRACLE" study of Annamycin in combination with cytarabine 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).
AI Summary
Moleculin Biotech reported that 78% of the target subjects for the first planned interim unblinding in its pivotal Phase 2B/3 MIRACLE study have now consented, up from 60% in November. The first unblinding threshold is 45 subjects, and the company expects to complete treatment of those first 45 in the first quarter of 2026. Consented participants are now enrolled across seven countries, and additional subjects continue to be identified by site investigators as recruitment and screening proceed.
The company said blinded response activity is tracking within expected ranges. The initial unblinding will include 30 subjects treated with Annamycin (two dose levels) plus cytarabine and 15 subjects on cytarabine plus placebo. Although the 45-subject readout isn’t powered for definitive statistics, Moleculin expects at least one Annamycin arm to outperform control. Recruitment for the full Part A (up to 90 patients) will continue while the first data are unblinded, with Part A expected to finish in the first half of 2026.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- November 12, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced it has entered into research and material transfer agreement with the University of North Carolina at Chapel Hill (UNC) for investigator-initiated preclinical research evaluating Annamycin for the treatment of pancreatic cancer.
AI Summary
Moleculin Biotech announced a research and material transfer agreement with the University of North Carolina at Chapel Hill to support investigator‑initiated preclinical studies testing Annamycin for pancreatic cancer. Under the deal Moleculin will supply Annamycin while William C. Zamboni, PharmD, PhD, at UNC’s Eshelman School of Pharmacy and Lineberger Cancer Center will lead the funded research.
The studies will test liposomal Annamycin (L‑Annamycin) and Free‑Annamycin combined with novel agents and delivery modalities to boost tumor uptake. UNC researchers will compare Annamycin formulations to Doxil and free doxorubicin in genetically engineered mouse models of pancreatic ductal adenocarcinoma (PDAC GEMMs).
Preclinical work suggests Annamycin concentrates in the pancreas and targets topoisomerase II, a factor tied to poorer survival in pancreatic cancer. The collaboration aims to define Annamycin’s potential role in pancreatic cancer and guide future investigator‑initiated studies.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- October 29, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that the Australian Patent Office (IP Australia) has granted Patent No. 2024203598 titled, "PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE," with claims covering certain preliposomal Annamycin lyophilizates with improved stability and high purity, with a base patent term currently extending until June 2040, subject to extension to account for time required to fulfill requirements for regulatory approval.
AI Summary
Moleculin Biotech, Inc. announced that IP Australia granted Patent No. 2024203598 titled “PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE.” The patent covers specific preliposomal Annamycin lyophilizates that offer improved stability and high purity. The base term runs until June 2040 and may be extended to account for time spent on regulatory approval requirements.
Walter Klemp, Chairman and CEO of Moleculin, said this new patent strengthens the company’s global patent protection and competitive position in oncology. He noted that adding the Australian patent demonstrates the team’s dedication and belief in Annamycin’s potential to treat hard-to-treat tumors. Annamycin is a next-generation anthracycline designed to avoid heart damage.
This patent is part of a growing global family that includes expected Canadian protection as well as already issued U.S. and European patents. These additions expand Moleculin’s intellectual property portfolio worldwide.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- October 23, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced it is working with Atlantic Health on an investigator-initiated Phase 1B/2 single-arm study evaluating Annamycin for third-line ("3L") treatment of advanced pancreatic cancer.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- September 9, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc announced updates to its active site status and recruitment for 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") 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).
AI Summary
Moleculin Biotech announced updates on its pivotal Phase 2B/3 “MIRACLE” trial of Annamycin plus cytarabine (AnnAraC) in relapsed or refractory adult AML. The study is multi-center, randomized, double-blind, placebo-controlled, and uses an adaptive design to measure safety and efficacy against standard therapy.
This month, the trial expanded beyond Ukraine to Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US, adding eight new sites for a total of 20 active centers. So far 13 subjects have been recruited, with 10 dosed. The company expects to enroll at least 20 subjects by month’s end.
Looking ahead, Moleculin aims to enroll the 45th patient in Q4 2025, which will allow an early data unblinding for preliminary efficacy and safety insights. The company plans to expand beyond 30 sites by year-end to support completing Part A in early 2026 and move toward Part B of the study.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- August 6, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced the presentation of encouraging preclinical data for its lead drug candidate, Annamycin, also known by its non-proprietary name of naxtarubicin, which demonstrated significant efficacy against various primary and metastatic liver cancers, including hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases.
AI Summary
Moleculin Biotech, Inc. announced promising preclinical results for its lead drug candidate, Annamycin (naxtarubicin). The data, presented at the Shelby-Lavine Pancreatic Cancer Symposium, showed strong activity against several primary and metastatic liver cancers, including hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases.
In lab models, Annamycin built up selectively in the liver, spleen, lungs, and pancreas—outperforming doxorubicin by reaching higher concentrations in these organs. In orthotopic HCC models, it reduced tumor growth and extended survival. In colorectal liver metastasis models, it halted metastatic spread and improved survival rates. It also blocked tumor growth in PDAC liver metastases, highlighting its potential to address aggressive forms of liver cancer.
The compound maintained a favorable safety profile, showing little or no cardiotoxicity compared to standard anthracyclines. These findings support further development of Annamycin as a targeted therapy for hard-to-treat liver tumors and unmet oncology needs.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- June 18, 2025
- Target Action Date:
- H2 2025
- Estimated Target Date Range:
- July 1, 2025 - December 31, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc announced that Enrollment and dosing are ongoing in pivotal, adaptive design Phase 3 MIRACLE trial of Annamycin in adult patients with R/R AML; Initial data readout is on track for the second half of 2025
AI Summary
Moleculin Biotech, Inc. has announced progress in its pivotal Phase 3 MIRACLE trial for Annamycin. This adaptive design study focuses on treating adult patients with relapsed or refractory acute myeloid leukemia (R/R AML). Enrollment and dosing of patients in the trial are currently ongoing across multiple international sites, including locations in the United States, Europe, and the Middle East.
The company expects the initial data readout from this trial to be available in the second half of 2025. This data will help assess the treatment’s safety and effectiveness in managing R/R AML, potentially paving the way for advancements in therapy options for this hard-to-treat form of cancer.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- June 18, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc announced it has received a written response from the Office of Oncologic Diseases - Pediatric Oncology, of the U.S. Food and Drug Administration (FDA) regarding the Company's Initial Pediatric Study Plan (iPSP), which was submitted after a June 2024 end-of-phase 1/2 meeting.
AI Summary
Moleculin Biotech, Inc. announced that it has received a written response from the FDA’s Office of Oncologic Diseases – Pediatric Oncology regarding its Initial Pediatric Study Plan (iPSP), which was submitted after a June 2024 end-of-phase 1/2 meeting. The FDA agreed to a single pediatric study evaluating Annamycin, in combination with Cytarabine, as a second line therapy for pediatric patients with relapsed/refractory acute myeloid leukemia. Importantly, the FDA recommended including patients as young as 6 months old, which is younger than Moleculin had initially proposed. The agency clarified that similar drug exposure and safety profiles compared to adult data could allow for efficacy extrapolation. Moleculin is now updating its iPSP to incorporate this feedback and expects to submit the revised plan later this quarter, with plans to begin the pediatric trial in the second half of 2027.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- April 29, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that an abstract and poster presentation regarding the Company's next-generation anthracycline, Annamycin, was presented at the American Association for Cancer Research (AACR) Annual Meeting 2025, on April 28, 2025, at the McCormick Place Convention Center in Chicago, IL.
AI Summary
Moleculin Biotech, Inc. announced that it presented an abstract and poster on its next-generation anthracycline, Annamycin, at the AACR Annual Meeting 2025. The presentation took place on April 28, 2025, at the McCormick Place Convention Center in Chicago, IL. The research highlighted Annamycin’s potential to work synergistically with a variety of FDA-approved anticancer drugs. Initial studies, carried out both in laboratory (in vitro) and animal (in vivo) models, suggest that combining Annamycin with standard anticancer therapies might significantly enhance treatment outcomes. These findings point to a possibility for broader clinical applications, potentially extending the use of Annamycin in treating not only blood cancers but also solid tumors such as sarcoma and pancreatic cancer. The Company's work at the AACR Annual Meeting reinforces its commitment to developing innovative cancer therapies and exploring effective drug combinations for challenging cancers.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- January 27, 2025
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced that Walter Klemp, Chairman and Chief Executive Officer of Moleculin participated in a Virtual Investor "What This Means" segment.
AI Summary
Moleculin Biotech, Inc. announced that its Chairman and CEO, Walter Klemp, recently took part in a Virtual Investor “What This Means” segment. In this online session, Klemp provided an update on the company's latest progress, focusing on the Phase 3 pivotal “MIRACLE” trial. This trial tests Annamycin in combination with Cytarabine to treat patients with acute myeloid leukemia (AML) who have not responded to or relapsed after initial therapy.
Klemp discussed how the global trial, which is being conducted across the US, Europe, and the Middle East, marks an important step forward for the company’s drug development efforts. He also explained what the recent advancements in the AML field could mean for Moleculin’s ongoing program with Annamycin, highlighting the significance of the trial’s progress for patients in need of new treatment options.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- December 11, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the online publication of its abstract titled, "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML," as part of the ASH Annual Meeting held December 7-10, 2024, in San Diego, CA.
AI Summary
Moleculin Biotech, Inc. recently announced the online publication of its abstract titled "Annamycin, a non-cardiotoxic anthracycline, demonstrates unique organotropism and activity against Ara-C and Venetoclax resistant AML" as part of the ASH Annual Meeting held December 7–10, 2024, in San Diego, CA. The study focuses on preclinical data showing that Annamycin can effectively target acute myeloid leukemia (AML) cells that have become resistant to standard treatments such as Cytarabine (Ara-C) and Venetoclax. Researchers highlighted that Annamycin not only works synergistically with Ara-C but also avoids the cardiotoxic side effects common to other anthracyclines, making it a promising option for patients with relapsed or refractory AML. These encouraging results support ongoing clinical evaluations and further development of Annamycin as a potential treatment for hard-to-treat leukemia patients.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- September 23, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc., announced the recent presentation of positive in vivo efficacy data of Annamycin in orthotopic and experimental lung metastatic models of sarcoma.
AI Summary
Moleculin Biotech, Inc. recently showcased promising results for its next-generation anthracycline, Annamycin, at the IASLC 2024 World Conference on Lung Cancer. The new data highlights that Annamycin significantly inhibits tumor growth and extends survival in orthotopic lung cancer models, as well as in experimental lung metastatic models of sarcoma. When compared to the commonly used anthracycline doxorubicin, Annamycin achieved 10- to 30-fold higher concentration levels in lung tissues, suggesting it has superior organotropic properties. In addition, Annamycin was confirmed to be 100% non-cardiotoxic, which is a critical benefit given the heart risks associated with other similar drugs. These encouraging results strengthen the potential of Annamycin as a treatment option for both primary and metastatic lung cancers, supporting further clinical evaluation in hard-to-treat cancers.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- August 1, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the positive discussion in and outcome of its End of Phase 1B/2 (EOP1B/2) meeting with the US Food and Drug Administration (FDA) supporting the advancement 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") to a Phase 3 pivotal trial for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).,
AI Summary
Moleculin Biotech announced a positive outcome from its End of Phase 1B/2 meeting with the FDA. In this meeting, the agency supported moving forward with a pivotal Phase 3 trial for Annamycin when used together with Cytarabine (often called Ara-C, and in combination, “AnnAraC”). The trial, named MIRACLE, will focus on patients with relapsed or refractory acute myeloid leukemia (R/R AML) who did not respond well to initial treatment.
The FDA’s feedback included using an adaptive design that relies on achieving a complete remission by day 30 as the primary endpoint. This approach aims to support a potential accelerated approval. Additionally, the trial will be global, including sites in the United States, and will allow dosing above the currently approved lifetime maximum for anthracyclines. Moleculin is optimistic that the trial will generate key data needed for regulatory approval.
Read Announcement- Drug:
- Annamycin
- Announced Date:
- July 10, 2024
- Indication:
- Soft tissue sarcoma (STS) lung metastases
Announcement
Moleculin Biotech, Inc. announced the completion of its End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its Phase 1B/2 clinical trial evaluating 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) for the treatment of subjects with AML as both first line therapy and for subjects who are refractory to or relapsed after induction therapy (MB-106). ,
AI Summary
Moleculin Biotech, Inc. announced that it has completed its End of Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for its Phase 1B/2 clinical trial. The trial evaluates the combination of Annamycin and Cytarabine—referred to as AnnAraC—for treating patients with acute myeloid leukemia (AML). The study targets both first line therapy patients as well as those who did not respond or relapsed after induction therapy under the MB-106 program.
The meeting with the FDA focused on evaluating the safety and efficacy data from the ongoing trial and outlining next steps for clinical development. The company expects to receive the official FDA meeting minutes by the end of Q3 2024, with further details to be reported before the end of August. Moleculin expressed gratitude for the productive discussions and is optimistic about advancing its AML treatment program.
Read Announcement
WP1066 FDA Regulatory Timeline and Events
WP1066 is a drug developed by Moleculin Biotech for the following indication: For the Treatment of Glioblastoma (NU 21C06).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- WP1066
- Announced Date:
- December 17, 2025
- Indication:
- For the Treatment of Glioblastoma (NU 21C06)
Announcement
Moleculin Biotech, Inc announced positive results from the Emory University physician-sponsored Phase 1 clinical trial conducted at the Aflac Cancer and Blood Disorders Center at Children's Healthcare of Atlanta led by pediatric oncologist, Tobey MacDonald, MD, who discovered STAT3 is critical to certain childhood brain tumors and currently serves as Professor of Pediatrics and Director of the Pediatric Neuro-Oncology Program.
AI Summary
Moleculin Biotech announced positive results from an Emory University physician‑sponsored Phase 1 trial of WP1066 at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta. The study was led by pediatric oncologist Tobey MacDonald, MD, who discovered that STAT3 is critical to certain childhood brain tumors and who serves as Professor of Pediatrics and Director of the Pediatric Neuro‑Oncology Program. WP1066 is an immune/transcription modulator designed to block oncogenic factors like phosphorylated STAT3 and to stimulate anti‑tumor immune responses.
In the trial, 10 children received WP1066 twice daily for 14 days to find a maximum feasible dose; three additional children received compassionate use treatment. Results showed no significant toxicity, suppression of STAT3 activity, clear anti‑tumor immune changes, and a partial response in a diffuse intrinsic pontine glioma (DIPG) patient. The findings were published in Journal of Clinical Investigation Insight and support moving toward a Phase 2 trial.Read Announcement
- Drug:
- WP1066
- Announced Date:
- September 9, 2024
- Indication:
- For the Treatment of Glioblastoma (NU 21C06)
Announcement
Moleculin Biotech, Inc., announced the enrollment and treatment of patients in an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06).
AI Summary
Moleculin Biotech, Inc. announced that patients with glioblastoma are now being enrolled and treated in an Investigator-initiated Phase 2 study at Northwestern University. This trial is testing WP1066 in combination with radiation therapy to treat adults with newly diagnosed glioblastoma, a highly aggressive brain tumor. The study, known as NU 21C06, is supported by funding from the NIH and BrainUp®, a non-profit focused on brain cancer awareness.
The trial aims to improve progression-free survival and includes tumor microenvironment analyses. Encouraging preclinical data showed that combining WP1066 with radiation not only increased survival rates in animal models but also triggered anti-tumor immune responses. Researchers are hopeful that this approach could address the unmet needs in glioblastoma treatment by providing a novel therapeutic option for patients with this challenging disease.
Read Announcement- Drug:
- WP1066
- Announced Date:
- May 15, 2024
- Indication:
- Ependymoma
Announcement
Moleculin Biotech, Inc., announced the commencement of an Investigator-initiated Phase 2 study evaluating WP1066 in combination with radiation therapy for the treatment of adults with glioblastoma (NU 21C06) in cooperation with the Company.
AI Summary
Moleculin Biotech, Inc. has announced the start of an important clinical study that could change the treatment options for adults with glioblastoma, a severe form of brain cancer. The company is initiating a Phase 2 trial—named NU 21C06—that will combine its drug candidate WP1066 with radiation therapy. This study is carried out through investigator initiative in cooperation with the company, aiming to explore and evaluate whether this combination can offer improved outcomes compared to current treatment methods.
The trial’s main focus is to determine the safety and effectiveness of using WP1066 alongside radiation therapy. Researchers are hopeful that this new approach could provide benefits to patients by enhancing the overall treatment strategy against glioblastoma. The results from this study are eagerly anticipated as they could pave the way for future advancements in cancer therapy.
Read Announcement
Ara-C FDA Regulatory Timeline and Events
Ara-C is a drug developed by Moleculin Biotech for the following indication: For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Ara-C
- Announced Date:
- October 30, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced it will present at the 14th Annual Acute Leukemia Meeting being held October 30-31, 2025 at the MD Anderson Cancer Center Spain Foundation in Madrid, Spain.
AI Summary
Moleculin Biotech, Inc. announced it will present at the 14th Annual Acute Leukemia Meeting on October 30-31, 2025, at the MD Anderson Cancer Center Spain Foundation in Madrid, Spain. This global scientific gathering brings experts together to discuss new therapies for acute leukemia.
The company’s presentation is titled “L-Annamycin – Non-Cardiotoxic Anthracycline; MIRACLE Pivotal AML Study.” It will take place during a lunch session on Thursday, October 30th, at 2:35 PM CEST. Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin, will deliver the talk.
Dr. Waymack will review the ongoing pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin combined with cytarabine (AnnAraC) for adult patients with relapsed or refractory acute myeloid leukemia (AML). Known as the “MIRACLE” trial, it includes clinical sites in the US, Europe and the Middle East.
The presentation will highlight the trial’s design, patient enrollment and expected milestones. For more information about the meeting and session details, please visit the event website.
Read Announcement- Drug:
- Ara-C
- Announced Date:
- September 8, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced that it has enrolled the first two subjects, and treated one, in the European Union (EU) in 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") 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).
AI Summary
Moleculin Biotech announced that its first European site in Spain has enrolled two subjects and treated one in its pivotal Phase 2B/3 “MIRACLE” trial of Annamycin combined with cytarabine (AnnAraC) for adults with relapsed or refractory acute myeloid leukemia. The global, randomized, double-blind, placebo-controlled study is also recruiting in the U.S., Ukraine, Georgia, and Romania.
The adaptive Part A will randomize 75–90 patients to receive high-dose cytarabine with placebo or one of two Annamycin doses. The trial allows early unblinding of complete remission and safety data after 45 subjects. Moleculin aims to reach this milestone by the end of 2025.
Part B will add about 220 more patients to confirm the optimal AnnAraC dose and support global approval. Initial data are expected in the second half of 2025.
Read Announcement- Drug:
- Ara-C
- Announced Date:
- July 9, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia 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") 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).
AI Summary
Moleculin Biotech, Inc. announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in Georgia has approved its Clinical Trial Application (CTA) for its pivotal Phase 2B/3 trial. This study will test Annamycin in combination with cytarabine (known as AnnAraC) to treat adult patients with acute myeloid leukemia (AML) who have not responded to or have relapsed after initial therapy. The trial is designed as a multi-center, randomized, double-blind, placebo-controlled study with an adaptive design to assess the drug’s safety and effectiveness. Patient dosing in Georgia is expected to start by the end of August, and additional sites in Europe and the US are scheduled to join later, with over 30 sites planned for Part A. The study supports the search for better treatment options for R/R AML patients in need of new therapies.
Read Announcement- Drug:
- Ara-C
- Announced Date:
- May 12, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., 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").
AI Summary
Moleculin Biotech, Inc. announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA) to run a pivotal Phase 2B/3 study using its combination treatment, AnnAraC, which pairs Annamycin with cytarabine (Ara‐C). This multi-center, randomized, double-blind, placebo-controlled trial is designed with an adaptive structure, aimed at treating adult patients with relapsed or refractory acute myeloid leukemia (R/R AML). The EMA approval covers nine European Union countries—Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain. This milestone approval enhances Moleculin’s recruitment and expands its global study efforts. The study is expected to help provide a much-needed treatment option for R/R AML patients, especially for those who have not responded to existing therapies, including recent venetoclax regimen failures.
Read Announcement - Drug:
- Ara-C
- Announced Date:
- April 1, 2025
- Indication:
- For the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
Announcement
Moleculin Biotech, Inc., announced the first patient has been dosed in its Phase 3 pivotal trial evaluating 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") for the treatment of AML patients who are refractory to or relapsed after induction therapy (R/R AML).
AI Summary
Moleculin Biotech, Inc. announced that the first patient has been dosed in its Phase 3 pivotal trial for relapsed or refractory acute myeloid leukemia (AML). The trial is evaluating Annamycin in combination with Cytarabine—also known as Ara-C—a regimen referred to as AnnAraC. This global study, called the MIRACLE trial, is taking place in the United States, Europe, and the Middle East, and it uses an adaptive trial design. Early unblinding of safety and efficacy data is expected from the initial 45 subjects in the second half of 2025, with further data planned for release in the first half of 2026. Moleculin’s dosing design, which includes different dose levels of Annamycin alongside a standard high dose of Cytarabine, aims to provide a promising new treatment option for AML patients who do not respond to or relapse after induction therapy.
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MB-106 FDA Regulatory Timeline and Events
MB-106 is a drug developed by Moleculin Biotech for the following indication: Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- MB-106
- Announced Date:
- August 27, 2025
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., announced the completion of its Phase 1B/2 (MB-106) clinical trial evaluating 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) for the treatment of subjects with acute myeloid leukemia (AML).
AI Summary
Moleculin Biotech, Inc. announced the completion of its Phase 1B/2 (MB-106) clinical trial evaluating Annamycin combined with Cytarabine (AnnAraC) in patients with acute myeloid leukemia (AML).
The study enrolled 22 subjects across first to seventh treatment lines. Database lock is expected by the end of September, and a final clinical study report is projected for Q1 2026. Updated overall survival (OS) results show median OS of 15 months for patients achieving complete remission (n=8), 12 months in the second-line evaluable group (n=9), and 9 months in the intent-to-treat population (n=22). These outcomes exceed typical relapsed AML survival of 4–6 months.
The trial recorded a 36% complete remission rate overall and 50% in second-line patients. Median remission durability was 10 months, with no cardiotoxicity observed.
Moleculin continues to use these findings to inform its pivotal MIRACLE Phase 3 trial design.
Read Announcement- Drug:
- MB-106
- Announced Date:
- June 17, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Mustang Bio, Inc. announced that updated data from the ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy, show a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia ("WM"), a rare form of blood cancer.
AI Summary
Mustang Bio, Inc. announced encouraging new data from its ongoing Phase 1/2 clinical trial of MB-106, a CD20-targeted, autologous CAR T-cell therapy. The treatment showed a favorable safety and efficacy profile in patients with Waldenstrom macroglobulinemia (WM), a rare blood cancer. In this small study, 90% of the patients responded to treatment, with three achieving complete responses, including one patient who has remained in complete remission for 31 months. The patients, all of whom had been heavily pretreated and refractory to BTK inhibitors, exhibited durable responses. Only one patient required additional anti-WM treatment after MB-106, and the therapy was feasible for outpatient administration. These promising findings highlight MB-106’s potential as a treatment option for WM, a field where no FDA-approved CAR T-cell treatments currently exist.
Read Announcement- Drug:
- MB-106
- Announced Date:
- June 14, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., reported additional efficacy findings from the Company's ongoing Phase 1B/2 (MB-106) clinical trial evaluating 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) for the treatment of subjects with acute myeloid leukemia (AML).
AI Summary
Moleculin Biotech, Inc. has released new efficacy findings from its ongoing Phase 1B/2 (MB-106) trial testing Annamycin combined with Cytarabine (AnnAraC) for treating acute myeloid leukemia (AML). In the study, 22 subjects were enrolled and 20 completed efficacy evaluations. Of these patients, 45% achieved a composite complete remission (CRc), including both complete remission (CR) and CR with incomplete blood count recovery (CRi). Notably, 89% of the CRc group had poor prognosis cytogenetics or mutations. In second line treatment, AnnAraC achieved an estimated median overall survival of about 6 months, with a similarly rising median durability of response for CRc patients. Importantly, the trial demonstrated no cardiotoxicity, and the combination was generally well tolerated, supporting its potential as a promising treatment option for AML patients.
Read Announcement- Drug:
- MB-106
- Announced Date:
- May 16, 2024
- Indication:
- Relapsed or Refractory B-cell non-Hodgkin lymphomas (NHL) and Chronic Lymphocytic Leukemia (CLL)
Announcement
Moleculin Biotech, Inc., announced its abstract has been accepted for poster presentation at the EHA2024 Hybrid Congress being held June 13-16, 2024 in Madrid, Spain and virtually.
AI Summary
Moleculin Biotech, Inc. recently announced that its abstract has been accepted for a poster presentation at the EHA2024 Hybrid Congress. The event will take place from June 13-16, 2024 in Madrid, Spain, and will also be accessible virtually. The poster, titled “LIPOSOMAL ANNAMYCIN (L-ANN) IN COMBINATION WITH CYTARABINE FOR TREATMENT OF PATIENTS WITH ACUTE MYELOID LEUKAEMIA (AML) REFACTORY TO OR RELAPSED (R/R) AFTER INDUCTION THERAPY (MB-106 STUDY),” will be presented by Dr. Wolfram C. M. Dempke, who serves as Moleculin’s European Chief Medical Officer.
This presentation highlights the Company’s efforts in developing new treatment approaches for challenging blood cancers. Attendees at the congress will have the opportunity to learn about Moleculin’s latest research, which may pave the way for improved therapies for patients with acute myeloid leukemia.
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