On October 23, 2025 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, reported 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.
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
Pancreatic cancer continues to present significant treatment challenges, being the cancer with the highest mortality rate globally and the seventh leading cause of cancer death. However, recent preclinical studies indicate that Annamycin may target critical factors associated with this disease, showcasing its potential as a novel therapeutic option.
Walter Klemp, Chairman and CEO of Moleculin, commented, "We are excited to work with Atlantic Health to advance the development of Annamycin for the treatment of pancreatic cancer. Their interest in funding and conducting this study stems from the compelling preclinical data presented at several American Association for Cancer Research (AACR) (Free AACR Whitepaper) conferences showing a high level of activity by Annamycin against pancreatic cancer and associated liver metastases in preclinical studies. We believe this may be related to Annamycin’s demonstrated high affinity for and ability to concentrate in the pancreas, especially considering the well-documented challenge of achieving adequate drug uptake to the pancreas with existing therapies. In addition, recent published data1 now reveal that the upregulation of topoisomerase II, the primary target of Annamycin, is highly correlated with poor survival in pancreatic cancer patients, so we have a highly validated target with pancreatic cancer. Coupled with Annamycin’s demonstrated lack of cardiotoxicity, which could enable, for the first time ever, the use of an anthracycline in chronic or maintenance therapies, we believe pancreatic cancer could be an important additional opportunity for Annamycin. Such maintenance therapy demonstrated promising results in a recently concluded clinical study with Annamycin for the treatment of soft tissue sarcoma metastasized to the lungs (MB-107)."
"This study further bolsters our efforts to advance and develop Annamycin through multiple investigator-initiated studies to unlock its potential as a treatment option for many other types of cancers," Mr. Klemp continued. "We continue to be encouraged by the preliminary preclinical data seen to date and believe Annamycin is a highly promising candidate for patients where there remains significant unmet need, such as pancreatic cancer."
"Pancreatic cancer remains one of the most devastating and difficult-to-treat malignancies, with limited options once patients progress beyond first- and second-line therapy," commented Dr. Angela Alistar, MD, Atlantic Health, the study’s principal investigator. "In particular, for the treatment options in the third-line setting, where survival rates are poor, no approved standard of care exists. We are committed to driving innovation in this space and based on the consistent safety and efficacy data, both clinically and preclinically, seen with Annamycin across a broad range of cancers, we believe this represents a great opportunity to address this high unmet need."
In connection with the planned study, Moleculin will supply Annamycin and be responsible for submitting and maintaining an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA), and Dr. Alistar of Atlantic Health will be responsible for conducting the Phase 1B/2 study. Moleculin estimates the additional cost to run this trial from 2026 into 2030 (for long-term follow-up) will be approximately $1 million, mainly for drug supply and outside laboratory testing.
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 AML, in addition to Orphan Drug Designation for the treatment of STS lung mets. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory AML from the EMA.
Moleculin is currently conducting 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). 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.
For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.
(Press release, Moleculin, OCT 23, 2025, View Source [SID1234656950])