Latest news with #Moleculin
Business Upturn
6 days ago
- Business
- Business Upturn
Moleculin Biotech, Inc. Unveils Promising Preclinical Data of Annamycin in Liver Cancer Treatment
Presented data highlights the potential of Annamycin for treating broad range of human cancers, including high-need oncology indications Annamycin currently in late-stage clinical development in combination with cytarabine for the treatment of AML; Anticipated preliminary data readout in 2H 2025 HOUSTON, Aug. 06, 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 tumors and viruses, today 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. This is believed to be the result of targeted accumulation in the liver and other organs. These findings were highlighted in a poster titled ' Liposomal Annamycin (L-ANN) Efficacy Against Primary and Metastatic Liver Cancers , ' presented by Dr. Waldemar Priebe, Lead Author and Chairman of the Scientific Advisory Board at Moleculin at the recently held Shelby-Lavine Pancreatic Cancer Symposium at MD Anderson Cancer Center. Key Highlights Targeted Accumulation in Organs: The preclinical studies confirmed that Annamycin exhibits distinct organotropic properties, leading to significantly higher concentrations in the liver, spleen, lungs, and pancreas when compared to doxorubicin (DOX). This targeted accumulation is critical for effectively treating liver-localized tumors. The preclinical studies confirmed that Annamycin exhibits distinct organotropic properties, leading to significantly higher concentrations in the liver, spleen, lungs, and pancreas when compared to doxorubicin (DOX). This targeted accumulation is critical for effectively treating liver-localized tumors. Efficacy in Orthotopic Hepatocellular Carcinoma (HCC) Models: Annamycin demonstrated excellent anti-tumor activity in orthotopic HCC models (Hepa 1-6 Luc), showing a marked reduction in tumor progression and improved survival rates in treated animals compared to vehicle controls. Annamycin demonstrated excellent anti-tumor activity in orthotopic HCC models (Hepa 1-6 Luc), showing a marked reduction in tumor progression and improved survival rates in treated animals compared to vehicle controls. Potent Impact on Colorectal Liver Metastasis: In an experimental liver metastatic model of colorectal carcinoma (CT26 Luc), Annamycin significantly inhibited metastatic growth and extended survival, highlighting its potential for addressing liver metastases in colon cancer that affects close to 50% of patients. In an experimental liver metastatic model of colorectal carcinoma (CT26 Luc), Annamycin significantly inhibited metastatic growth and extended survival, highlighting its potential for addressing liver metastases in colon cancer that affects close to 50% of patients. Promising Results in Pancreatic Cancer Liver Metastasis: Annamycin also showed compelling efficacy in liver-implanted human pancreatic ductal adenocarcinoma (MIA PaCa-2), leading to inhibition of tumor growth, suggesting its potential role in managing advanced pancreatic cancer with liver involvement. Annamycin also showed compelling efficacy in liver-implanted human pancreatic ductal adenocarcinoma (MIA PaCa-2), leading to inhibition of tumor growth, suggesting its potential role in managing advanced pancreatic cancer with liver involvement. Favorable Safety Profile: Consistent with previous preclinical and clinical findings, Annamycin continued to show low or no cardiotoxicity, a significant advantage over traditional anthracyclines like doxorubicin, which are often limited by dose-dependent cardiac side effects. This safety profile was previously observed in clinical trials, where 32 out of 42 subjects reviewed received more than the FDA-established lifetime maximum allowable level of anthracycline without evidence of cardiotoxicity. 'We are incredibly encouraged by these preclinical results, which further validate Annamycin's potential as a powerful and differentiated therapeutic agent for liver cancers,' said Dr. Priebe. 'The ability of Annamycin to concentrate effectively in the liver, combined with its demonstrated efficacy across multiple aggressive liver cancer models and its favorable cardiotoxicity profile, positions it as a highly promising candidate for patients facing these devastating diseases. We believe these data provide a strong foundation for advancing Annamycin in clinical development for these indications, offering new hope where treatment options are often limited.' Walter Klemp, Chairman and CEO of Moleculin, added, 'We continue to be strongly encouraged by the potential of Annamycin. Beyond our preliminary programs in AML and soft tissue sarcoma lung metastases (STS lung mets), we continue to advance and develop Annamycin through multiple investigator-initiated studies to further unlock its potential as a treatment option for many other types of cancers. We believe this preclinical data highlights that potential and provides additional validation of its unique pharmacological profile and importantly showcases the opportunity for its use across a wide range of cancers.' Moleculin's novel drug candidate is being positioned to become the first ever non-cardiotoxic anthracycline to be approved and is currently being developed for the treatment of AML and STS lung mets. For more information, please visit 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 has begun 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 whether the preclinical results described above are able to be reproduced in clinical trials. 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) 824-0775 [email protected]
Yahoo
11-06-2025
- Business
- Yahoo
Moleculin Participates in Virtual Investor 'What This Means' Segment
Globally recognized Oncology Key Opinion Leader, Brian Andrew Van Tine, MD, PhD, discusses the positive topline results from the U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of Soft Tissue Sarcoma Lung Metastases (MB-107) Watch the 'What This Means" segment here HOUSTON, June 11, 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 tumors and viruses, today announced that it participated in a Virtual Investor 'What This Means' segment. For the segment, Brian Andrew Van Tine, MD, PhD, Professor of Medicine, Washington University School of Medicine, discussed the Company's recently announced positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of Soft Tissue Sarcoma Lung Metastases ('STS lung mets') (MB-107). The MB-107 trial was a multi-center, open-label, single-arm monotherapy study that in Phase 1B determined the Maximum Tolerable Dose and Recommended Phase 2 Dose ('MTD', 'RP2D' respectively) and safety of Annamycin and in Phase 2 explored the efficacy of Annamycin as a single agent for the treatment of subjects with STS lung mets for which chemotherapy was considered appropriate. For more information about the MB-107 trial visit and reference identifier NCT04887298. The Virtual Investor 'What This Means' segment featuring Moleculin is now available here. 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 Company has begun 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 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 StatementsSome 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 reconcile 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@
Associated Press
04-06-2025
- Business
- Associated Press
Moleculin Reports Positive Topline Efficacy Results from U.S. Phase 1B/2 Clinical Trial Evaluating Annamycin for the Treatment of Soft Tissue Sarcoma Lung Metastases (MB-107)
Annamycin delivering better performance 7thline than would be expected even in 2ndline for monotherapy Responders (Stable Disease or Partial Response) after 2 cycles of Annamycin showed improvement in OS and Progression Free Survival ('PFS') Clinical Benefit Rate ('CBR') was 59.4% (n=32), comprised of 18 subjects with stable disease and 1 subject with a partial response Potential to address 13,500 new incidents of STS each year and market opportunity expected to grow to $2.6B by 20301 On-demand video webcast with members of the Moleculin management team and internationally renowned Key Opinion Leaders available Thursday, June 5th HOUSTON, June 04, 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 viruses, today reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ('STS lung mets') (MB-107). The MB-107 trial was a multi-center, open-label, single-arm monotherapy study that in Phase 1B determined the Maximum Tolerable Dose and Recommended Phase 2 Dose ('MTD', 'RP2D' respectively) and safety of Annamycin and in Phase 2 explored the efficacy of Annamycin as a single agent for the treatment of subjects with STS lung mets for which chemotherapy was considered appropriate. For more information about the MB-107 trial visit and reference identifier NCT04887298. 'These positive topline results from MB-107 are incredibly encouraging. The impact Annamycin demonstrated on median overall survival, particularly with patients who received multiple prior chemotherapy regimens, exceeded expectations. Additionally, the improvement seen with PFS after two doses represents a real potential for Annamycin to provide a meaningful treatment option for the treatment of STS lung mets,' commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. 'Looking ahead, we believe these results strongly support further evaluations of Annamycin for the treatment of STS lung mets and we look forward to exploring opportunities to potentially bring this important treatment option to patients.' Topline Efficacy Results Summary Annamycin 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. On-Demand Webcast Details An on-demand Virtual STS Lung Mets KOL Webcast to discuss the MB-107 data will be available for investors, analysts, and other interested parties on Thursday, June 5, 2025 beginning at 8:30 AM ET. For the event, Walter Klemp, Chairman and Chief Executive Officer, and Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin will be joined by Key Opinion Leaders: Mohamad Cherry, MD, Medical Director of Hematology at Atlantic Health System; Prof. Bernd Kasper, Sarcoma Unit of the Mannheim Cancer Center (MCC) at the Mannheim University Medical Center, University of Heidelberg; and Sant P. Chawla, MD, Director, Sarcoma Oncology Center, Director, Cancer Center of Southern California. Interested participants and investors may access the on-demand video webcast on the Events page of the Investors section of the Moleculin website, The webcast will be accessible for 90 days. About STS Lung Mets Soft tissue sarcoma is a type of cancer that originates in the soft tissues of the body, including muscles, tendons, fat, blood vessels and nerves. Lung metastases are a common occurrence in STS, and can impact overall survival. According to The American Cancer Society approximately 13,500 individuals, both adults and children, will be diagnosed with soft tissue sarcoma in 20252. Soft tissue sarcomas account for 1% of all adult cancers, 7% of cancers in children up to age 15, and 3% of cancers in children under the age of 14. The Soft Tissue Sarcoma Market was valued at USD 1.58 Billion in 2024, and is expected to reach USD 2.57 Billion by 2030, rising at a CAGR of 8.43%. OS for standard of care for metastatic STS has been estimated at 8-12 months and certain experimental targeted and cytotoxic therapies (as monotherapies or some in combination) have yielded OS of 13.4 months as second line therapy.3 MB-107 Study Design In Phase 2, Annamycin was administered as an intravenous (IV) infusion over 2 hours on Day 1, followed by 20 days off (1 cycle = 21 days). Subjects visit the study site every 21 days (±3 days) at which time safety monitoring – including for adverse events (AEs), as well as a physical examination, laboratory evaluations (clinical chemistry, complete blood count), vital signs, weight measurements, Eastern Cooperative Oncology Group (ECOG) performance status, and electrocardiograms (ECGs) – is performed, followed by an IV infusion of study drug. Cardiac function is followed by echocardiogram (ECHO) scans at screening, at the end of the first two cycles and then following every other cycle thereafter, at the End of Treatment visit, and if feasible, during follow up at 6 months (±1 month) and 1 year (±1 month) after study drug discontinuation. As long as the Investigator considers that the benefits of treatment with Annamycin continue to outweigh the risks, treatment will continue every 21 days until tumor progression is observed or unacceptable toxicity occurs. Tumor response is monitored every 6 weeks (±1 week) from Cycle 1 Day 1 during treatment, at the End of Treatment visit, and then every 3 months (±1 month) until disease progression using RECIST 1.1 criteria. Those subjects who leave the study after a maximum response is achieved and who do not start another therapy will be followed every 3 months (±1 month) for progression-free survival (PFS). If a subject receives further therapy after discontinuing from the study, they will be followed only for overall survival (OS) and if feasible, follow-up ECHO scans at 6 months (±1 month) and 1 year (±1 month) were conducted after study drug discontinuation. 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 has begun 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 reconcile 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, LLC Jenene Thomas (908) 824-0775 [email protected] 1 2 American Cancer Society. Cancer Facts & Figures 2025. Atlanta: American Cancer Society; 2025. Available at 3 Comandone A, et al; 'Salvage Therapy in Advanced Adult Soft Tissue Sarcoma: A Systematic Review and Meta-Analysis of Randomized Trials'; The Oncologist 2017;22:1518–1527
Yahoo
04-06-2025
- Business
- Yahoo
Moleculin Reports Positive Topline Efficacy Results from U.S. Phase 1B/2 Clinical Trial Evaluating Annamycin for the Treatment of Soft Tissue Sarcoma Lung Metastases (MB-107)
Annamycin delivering better performance 7th line than would be expected even in 2nd line for monotherapy Responders (Stable Disease or Partial Response) after 2 cycles of Annamycin showed improvement in OS and Progression Free Survival ('PFS') Clinical Benefit Rate ('CBR') was 59.4% (n=32), comprised of 18 subjects with stable disease and 1 subject with a partial response Potential to address 13,500 new incidents of STS each year and market opportunity expected to grow to $2.6B by 20301 On-demand video webcast with members of the Moleculin management team and internationally renowned Key Opinion Leaders available Thursday, June 5th HOUSTON, June 04, 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 viruses, today reported positive topline efficacy results from its completed U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ('STS lung mets') (MB-107). The MB-107 trial was a multi-center, open-label, single-arm monotherapy study that in Phase 1B determined the Maximum Tolerable Dose and Recommended Phase 2 Dose ('MTD', 'RP2D' respectively) and safety of Annamycin and in Phase 2 explored the efficacy of Annamycin as a single agent for the treatment of subjects with STS lung mets for which chemotherapy was considered appropriate. For more information about the MB-107 trial visit and reference identifier NCT04887298. 'These positive topline results from MB-107 are incredibly encouraging. The impact Annamycin demonstrated on median overall survival, particularly with patients who received multiple prior chemotherapy regimens, exceeded expectations. Additionally, the improvement seen with PFS after two doses represents a real potential for Annamycin to provide a meaningful treatment option for the treatment of STS lung mets,' commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. 'Looking ahead, we believe these results strongly support further evaluations of Annamycin for the treatment of STS lung mets and we look forward to exploring opportunities to potentially bring this important treatment option to patients.' Topline Efficacy Results Summary Clinical Benefit Rate ('CBR') was 59.4% (n=32), comprised of 18 subjects with stable disease and 1 subject with a partial response (no subjects achieved Complete Response or CR) Progression Free Survival ('PFS') & Overall Survival ('OS'): Dose and regimen optimized subjects demonstrated PFS of ~4 months and OS of ~20 months Overall (N=36) Median PFS was 63 days, with a 95% Confidence Interval ('CI') between 43 and 105 days Median OS was 411 days, with a 95% CI between 241 and 583 days In Phase 2 (N=17) at 330 mg/m2: Median PFS was 105 days and OS for all-comers (median 6 prior therapies) of 13.5 months exceeded typical results for 2nd line monotherapies (8-12 months)1 OS/PFS was higher (19.9 months/127 days) for subjects with fewer prior therapies (≤2) (n=7) and receiving doses of Annamycin ≤ 330 mg/m2 (the RP2D) Results suggest overall disease control was better at 330 mg/m2, but once combined with Phase 1B data OS and PFS was the same as overall Subjects achieving a Partial Response ('PR') or Stable Disease ('SD') with ≥2 cycles also experienced higher OS/PFS (19.7 months/122 days), demonstrating that achieving CBR from Annamycin resulted in better outcomes No cardiotoxicity demonstrated in all subject data as noted by an independent Expert Annamycin 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. On-Demand Webcast Details An on-demand Virtual STS Lung Mets KOL Webcast to discuss the MB-107 data will be available for investors, analysts, and other interested parties on Thursday, June 5, 2025 beginning at 8:30 AM ET. For the event, Walter Klemp, Chairman and Chief Executive Officer, and Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin will be joined by Key Opinion Leaders: Mohamad Cherry, MD, Medical Director of Hematology at Atlantic Health System; Prof. Bernd Kasper, Sarcoma Unit of the Mannheim Cancer Center (MCC) at the Mannheim University Medical Center, University of Heidelberg; and Sant P. Chawla, MD, Director, Sarcoma Oncology Center, Director, Cancer Center of Southern California. Interested participants and investors may access the on-demand video webcast on the Events page of the Investors section of the Moleculin website, The webcast will be accessible for 90 days. About STS Lung Mets Soft tissue sarcoma is a type of cancer that originates in the soft tissues of the body, including muscles, tendons, fat, blood vessels and nerves. Lung metastases are a common occurrence in STS, and can impact overall survival. According to The American Cancer Society approximately 13,500 individuals, both adults and children, will be diagnosed with soft tissue sarcoma in 20252. Soft tissue sarcomas account for 1% of all adult cancers, 7% of cancers in children up to age 15, and 3% of cancers in children under the age of 14. The Soft Tissue Sarcoma Market was valued at USD 1.58 Billion in 2024, and is expected to reach USD 2.57 Billion by 2030, rising at a CAGR of 8.43%. OS for standard of care for metastatic STS has been estimated at 8-12 months and certain experimental targeted and cytotoxic therapies (as monotherapies or some in combination) have yielded OS of 13.4 months as second line therapy.3 MB-107 Study Design In Phase 2, Annamycin was administered as an intravenous (IV) infusion over 2 hours on Day 1, followed by 20 days off (1 cycle = 21 days). Subjects visit the study site every 21 days (±3 days) at which time safety monitoring – including for adverse events (AEs), as well as a physical examination, laboratory evaluations (clinical chemistry, complete blood count), vital signs, weight measurements, Eastern Cooperative Oncology Group (ECOG) performance status, and electrocardiograms (ECGs) – is performed, followed by an IV infusion of study drug. Cardiac function is followed by echocardiogram (ECHO) scans at screening, at the end of the first two cycles and then following every other cycle thereafter, at the End of Treatment visit, and if feasible, during follow up at 6 months (±1 month) and 1 year (±1 month) after study drug discontinuation. As long as the Investigator considers that the benefits of treatment with Annamycin continue to outweigh the risks, treatment will continue every 21 days until tumor progression is observed or unacceptable toxicity occurs. Tumor response is monitored every 6 weeks (±1 week) from Cycle 1 Day 1 during treatment, at the End of Treatment visit, and then every 3 months (±1 month) until disease progression using RECIST 1.1 criteria. Those subjects who leave the study after a maximum response is achieved and who do not start another therapy will be followed every 3 months (±1 month) for progression-free survival (PFS). If a subject receives further therapy after discontinuing from the study, they will be followed only for overall survival (OS) and if feasible, follow-up ECHO scans at 6 months (±1 month) and 1 year (±1 month) were conducted after study drug discontinuation. 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 Company has begun 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 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 StatementsSome 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 reconcile 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@ 1 2 American Cancer Society. Cancer Facts & Figures 2025. Atlanta: American Cancer Society; 2025. Available at 3 Comandone A, et al; 'Salvage Therapy in Advanced Adult Soft Tissue Sarcoma: A Systematic Review and Meta-Analysis of Randomized Trials'; The Oncologist 2017;22:1518–1527Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
11-04-2025
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Expert says Summit's HARMONi-2 study could beat expectations, says Truist
The Survival read-out on the Summit Therapeutics (SMMT)' HARMONi-2 study is one of the most anticipated biotech datapoints this year, and Truist tells investors in a research note that its biostatistics expert consultation gives the firm confidence that this study could beat expectations, and may even show statistical significance. The firm believes that the consensus 'home-run scenario' will drive up Summit shares materially if it plays out. Truist has a Buy rating and $35 price target on the shares. Discover outperforming stocks and invest smarter with Top Smart Score Stocks. Filter, analyze, and streamline your search for investment opportunities using Tipranks' Stock Screener. Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on SMMT: Disclaimer & DisclosureReport an Issue Optimistic Buy Rating for Summit Therapeutics Driven by Promising HARMONi-2 Trial and Favorable Regulatory Pathway in China Promising Potential of Ivonescimab in Oncology: A Buy Recommendation for Summit Therapeutics Moleculin, CNS, Global-e, Tenet, Summit: Trending by Analysts Summit Therapeutics upgraded to Buy from Neutral at Citi FedEx downgraded, Adobe upgraded: Wall Street's top analyst calls
