Moleculin Abstract Accepted for Poster Presentation at the Acute Leukemias XIX (ISALXIX) International Symposium

On March 3, 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 tumors and viruses, reported its abstract has been accepted for poster presentation at the Acute Leukemias XIX (ISALXIX) International Symposium being held March 16-19, 2025 in Munich, Germany (Press release, Moleculin, MAR 3, 2025, View Source [SID1234650844]).

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Details for the poster presentation are as follows:

Abstract: P002
Session: Clinical Studies in AML and ALL
Title: Liposomal Annamycin in Combination with Cytarabine for Treatment of Acute Myeloid Leukemia (AML)
Presenting Author: Cristina Papayannidis, MD, PhD, Adjunct professor, Department of Medical and Surgical Sciences, University of Bologna, Bologna Italy

For more information about the ISALXIX International Symposium, visit the event website.

Laekna Announces IND Approval of LAE120 (a Novel USP1 Inhibitor) for Treatment of Advanced Solid Tumors by FDA

On March 3, 2025 Laekna (2105.HK) reported that the U.S. Food and Drug Administration (FDA) has approved the IND for LAE120, an internally discovered USP1 inhibitor, for the treatment of advanced solid tumors (Press release, Laekna Therapeutics, MAR 3, 2025, View Source [SID1234650861]).

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LAE120 is a novel, allosteric and highly potent USP1 inhibitor, displaying monotherapy potency and combination activity with PARP inhibitor in HRD (homologous recombination deficiency) cancers. It has a unique chemical structure differentiated from all the other disclosed USP1 inhibitors and is expected to induce a different conformational change in USP1. LAE120 shows robust tumor inhibitory activity across various xenograft models such as MDA-MB-436 and K562 as a single agent and exhibits synergistic effect in combination with PARP inhibitors. It also demonstrates good therapeutic windows in GLP long-term toxicology study. Laekna is actively exploring partnerships to accelerate the clinical development of LAE120.

"Leveraging our deep know-how and extensive expertise in drug discovery, Laekna has developed a distinctive portfolio of innovative drug candidates through the close collaboration of our Med Chem, Biology and AIDD (AI-driven Drug Discovery) teams, continuously advancing preclinical drug candidates into clinical stage. Laekna has also significantly accelerated the progress of drug discovery by utilizing cutting-edge artificial intelligence tools," said Dr. Justin Gu, Chief Scientific Officer of Laekna. "We look forward to bringing novel drugs to patients as swiftly as possible," he added.

Advancing Diversified Pipelines

Laekna is actively advancing preclinical drug candidates. In the fourth quarter of 2024, another internally discovered anti-tumor drug candidate, LAE118, a potentially best-in-class, mutant-selective PI3Kαinhibitor, has advanced to IND-enabling study. PI3Kα mutations are prevalent in patients with breast, colorectal, lung, endometrial, and numerous other cancers. However, the first-generation drugs targeting PI3Kα inhibit the wild-type and mutant PI3Kα with equal potency, which raises concerns of tolerability and therapeutic efficacy.

As a novel allosteric inhibitor, LAE118 demonstrates excellent potency and selectivity towards various PI3Kα mutants. With superior anti-cancer efficacy and tolerability than other current PI3Kα inhibitors, LAE118 is potentially the best-in-class pan-mutant-selective PI3Kα inhibitor. Laekna has presented the preclinical characterization of LAE118 at the San Antonio Breast Cancer Symposium (SABCS) in December 2024. LAE118 is in IND-enabling studies and IND is expected to be filed in the fourth quarter of 2025.

Strategic Partnerships to Accelerate Globalization

"We will continue to advance and expand our product portfolio in the therapeutic areas where we have accumulated tremendous experience and extensive know-how," said Dr. Chris Lu, Chief Executive Officer of Laekna. "In November 2024, the Group has entered into a clinical collaboration agreement with Lilly (NYSE:LLY) to support and accelerate global clinical development of LAE102 for the treatment of obesity. We plan to pursue strategic partnerships with global leading pharmaceutical companies to accelerate clinical development and commercialization of our drug candidate assets. We keep advancing and expanding our pipeline and are committed to bringing life-changing medicines to more people around the world," he added.

Can-Fite Innovative Publication: Namodenoson in Liver Cancer Treatment Alongside with Cardiac, Liver, and Neurological Benefits

On March 3, 2025 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company developing a pipeline of proprietary small molecule drugs targeting oncological and inflammatory diseases, reported that the European Society of Medicine Journal published an innovative article titled: "The Neuro- Cardio- and Hepato- Protective Effects of Namodenoson are Mediated by Adiponectin" (Press release, Can-Fite BioPharma, MAR 3, 2025, View Source [SID1234650826]). The study is the result of a collaboration between Can-Fite scientists and leading hepatologists from the Department of Gastroenterology and Liver Diseases at Soroka University Medical Center, Beer Sheva, Israel.

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The article presents compelling preclinical and clinical data demonstrating Namodenoson’s potent anti-ischemic, anti-inflammatory, anti-fibrotic, and anti-toxicity effects across multiple body tissues including the liver, central nervous system, and cardiovascular system. The study highlights Namodenoson’s ability to increase adiponectin levels, a key cytokine known to drive multi-organ protective effects. Importantly, the manuscript underscores Namodenoson’s dual role as both an anti-cancer therapy and a protective agent for normal tissues, setting it apart from conventional chemotherapy and other oncology treatments with significant toxicity.

Dr. Pnina Fishman, CSO & Chairperson of Can-Fite BioPharma, commented: "The data published in this journal further supports Namodenoson’s unique profile as an anti-cancer drug that simultaneously safeguards healthy body systems. This critical distinction positions Namodenoson as a potential breakthrough therapy, unlike traditional chemotherapy, which is often associated with severe toxicity.

Namodenoson is currently being evaluated in LiverationTM, a pivotal Phase III study for advanced liver cancer that has been approved by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The drug has an Orphan Drug status with both FDA and EMA and a Fast Track status with the FDA.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is currently being evaluated in a pivotal Phase III trial for advanced liver cancer, a Phase IIb trial for the treatment of Metabolic Dysfunction-associated Steatohepatitis (MASH), and in a Phase IIa study in pancreatic cancer. A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential expression may be one of the important factors that accounts for the excellent safety profile of the drug.

Imfinzi-based perioperative regimen recommended for approval in the EU by CHMP for resectable non-small cell lung cancer

On March 3, 2025 AstraZeneca reported that Imfinzi (durvalumab) in combination with chemotherapy has been recommended for approval in the European Union (EU) for the treatment of adults with resectable non-small cell lung cancer (NSCLC) at high risk of recurrence and no epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements (Press release, AstraZeneca, MAR 3, 2025, View Source [SID1234650846]). In this regimen, patients are treated with Imfinzi in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery.

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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from the pivotal AEGEAN trial, which were published in The New England Journal of Medicine.

Results from a planned interim analysis of event-free survival (EFS) showed a statistically significant and clinically meaningful 32% reduction in the risk of recurrence, progression events or death versus neoadjuvant chemotherapy alone in patients treated with the Imfinzi-based perioperative regimen (32% data maturity; EFS hazard ratio [HR] 0.68; 95% confidence interval [CI] 0.53-0.88; p=0.003902). In a final analysis of pathologic complete response (pCR), treatment with Imfinzi plus neoadjuvant chemotherapy before surgery resulted in a pCR rate of 17.2% versus 4.3% for patients treated with neoadjuvant chemotherapy alone (difference in pCR 13.0%; 95% CI 8.7-17.6).

Additionally, interim overall survival (OS) results presented at the 2024 World Conference on Lung Cancer showed a favourable trend with the Imfinzi-based perioperative regimen (35% data maturity; median OS: not reached [NR] versus 53.2 months; HR=0.89; 95% CI 0.70-1.14). The OS data were not tested for statistical significance at this interim analysis and will continue to be assessed as a key secondary endpoint at final analysis.

Professor Martin Reck, Head of the Department of Thoracic Oncology at the Lung Clinic Grosshansdorf, Germany, member of the AEGEAN Steering Committee and investigator in the trial, said: "Adding perioperative durvalumab to neoadjuvant chemotherapy significantly improved outcomes for patients with resectable non-small cell lung cancer, who experience high rates of recurrence and poor outcomes. Today’s recommendation marks an important step towards patients and their clinicians in Europe gaining access to an innovative treatment that should become a backbone combination approach in this curative-intent setting."

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "This recommendation highlights the potential of Imfinzi to address an unmet need for patients with resectable lung cancer who need new treatment options that increase the time they live without recurrence or progression. AEGEAN underscores our commitment to transforming care in the early stages of lung cancer where there is the greatest potential for cure."

Each year in Europe, there are more than 450,000 people diagnosed with lung cancer.1 Around 25-30% of all patients with NSCLC, the most common form of lung cancer, are diagnosed early enough to have surgery with curative intent.2-3 However, the majority of patients with resectable disease will develop recurrence and only 36-46% of patients with Stage II disease will survive for five years.4-5 This decreases to 24% for patients with Stage IIIA disease and 9% for patients with Stage IIIB disease, reflecting a high unmet medical need.4

Imfinzi was generally well tolerated, and no new safety signals were observed in the neoadjuvant and adjuvant settings. Further, adding Imfinzi to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not compromise patients’ ability to complete surgery versus chemotherapy alone.

Imfinzi is approved in the US and several other countries in this setting based on the AEGEAN results. Regulatory applications are also currently under review in China, Japan and additional countries.

Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy (CRT) based on the PACIFIC Phase III trial.

Notes

Lung cancer
Lung cancer is the leading cause of cancer death among both men and women, accounting for about one-fifth of all cancer deaths.6-7 Lung cancer is broadly split into NSCLC and small cell lung cancer (SCLC), with 80-85% of patients diagnosed with NSCLC.8-9 An estimated 28,000 people are treated for resectable NSCLC across the five major European countries each year.10

Early-stage lung cancer diagnoses are often only made when the cancer is found on imaging for an unrelated condition.11-12 The majority of patients with resectable disease eventually develop recurrence despite complete tumour resection and adjuvant chemotherapy.5

AEGEAN
AEGEAN is a randomised, double-blind, multi-centre, placebo-controlled global Phase III trial evaluating Imfinzi as perioperative treatment for patients with resectable Stage IIA-IIIB (Eighth Edition AJCC Cancer Staging Manual) NSCLC, irrespective of PD-L1 expression. Perioperative therapy includes treatment before and after surgery, also known as neoadjuvant/adjuvant therapy. In the trial, 802 patients were randomised to receive a 1500mg fixed dose of Imfinzi plus chemotherapy or placebo plus chemotherapy every three weeks for four cycles prior to surgery, followed by Imfinzi or placebo every four weeks (for up to 12 cycles) after surgery. Patients with known EGFR or ALK genomic tumour aberrations were excluded from the primary efficacy analyses.

In the AEGEAN trial, the primary endpoints were pCR, defined as no viable tumour in the resection specimen (including lymph nodes) following neoadjuvant therapy, and EFS, defined as the time from randomisation to an event like tumour recurrence, progression precluding definitive surgery, or death. Key secondary endpoints were major pathologic response, defined as residual viable tumour of less than or equal to 10% in the resected primary tumour following neoadjuvant therapy, disease-free survival, OS, safety and quality of life. The final pathologic response analyses were performed after all patients had the opportunity for surgery and pathology assessment per the trial protocol. The trial enrolled participants from 264 centres in more than 25 countries including in the US, Canada, Europe, South America and Asia.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its indications in resectable, early-stage (IIa-IIIB) NSCLC and unresectable, Stage III NSCLC, Imfinzi is also approved for use in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy (etoposide and either carboplatin or cisplatin) for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy (gemcitabine plus cisplatin) in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). Imfinzi is also approved as a monotherapy in unresectable HCC in Japan and the EU.

Imfinzi is also approved in combination with chemotherapy (carboplatin and paclitaxel) followed by Imfinzi monotherapy in primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR) in the US. In the EU, Imfinzi plus chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient (pMMR) advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi alone is approved for patients with dMMR disease. In Japan, Imfinzi plus chemotherapy followed by Imfinzi monotherapy has also been approved as 1st-line treatment in primary advanced or recurrent endometrial cancer, and Imfinzi plus chemotherapy followed by Imfinzi and Lynparza has been approved for patients with pMMR disease.

Since the first approval in May 2017, more than 374,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with SCLC, NSCLC, bladder cancer, breast cancer, several gastrointestinal and gynaecologic cancers, and other solid tumours.

SOTIO Unveils Promising Preclinical Data on SOT109, a Next-Gen ADC Targeting Cadherin-17 (CDH17) for Colorectal Cancer

On March 3, 2025 SOTIO Biotech, a clinical-stage biopharmaceutical company owned by PPF Group, reported the first preclinical data on SOT109, a novel antibody-drug conjugate (ADC) targeting cadherin-17 (CDH17) (Press release, SOTIO, MAR 3, 2025, View Source [SID1234650862]). The data highlight SOT109’s promising activity and tolerability profile across multiple disease models, reinforcing its potential to become a best-in-class therapeutic for the treatment of colorectal cancer and other gastrointestinal (GI) cancers. The findings were presented at the 15th World ADC London 2025, taking place from March 3 to 6 at Novotel London West, London, UK.

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"We are excited to share groundbreaking data on SOT109, an innovative ADC designed to treat colorectal cancer and other GI malignancies," said Martin Steegmaier, Ph.D., chief scientific officer of SOTIO. "CDH17 has long been recognized as a highly compelling therapeutic target, yet previous non-ADC and ADC approaches have faced significant obstacles. Our cutting-edge design overcomes these challenges, utilizing state-of-the-art linker-payload technology and an optimized antibody backbone to enhance efficacy and tumor selectivity. Preclinical data indicate that our candidate not only delivers potent tumor cell killing but does so with an excellent safety profile and high therapeutic index. We believe this represents a major advancement in ADC development for GI cancers, and we look forward to filing an Investigational New Drug (IND) application for this program in Q2 2026."

CDH17 is a highly promising target homogenously overexpressed in more than 90% of colorectal cancers (CRC) and abundantly expressed in other GI cancers, including gastric, pancreatic, and esophageal cancers. In contrast, its expression in normal adult tissues is largely restricted to the GI tract, reducing the risk of off-target toxicity.

As CDH17 is an abundantly expressed and rapidly internalizing transmembrane protein on GI cancer cells, CDH17 is ideally suited for an ADC-mediated drug delivery approach. SOT109 is a fully human and highly specific ADC with exceptional binding and internalization properties against CDH17. SOT109 utilizes Synaffix’s clinically validated ADC platform, incorporating the SYNtecan E proprietary linker-payload system that contains an exatecan payload (DAR=4). For the treatment of CRC, SOT109’s exatecan payload is expected to demonstrate superior activity, reduced susceptibility to resistance, and an enhanced bystander effect due to its high cell permeability, setting it apart from other ADC payloads utilized to date.

In preclinical studies presented at World ADC London, SOT109 exhibited strong anti-tumor activity across multiple mouse xenograft models, including complete responses. It demonstrated favorable tolerability in an exploratory mouse study at doses up to 150 mg/kg. Furthermore, data from a subsequent toxicity study in non-human primates (NHPs) revealed that SOT109 displays a favorable therapeutic index underscoring its potential as a transformative therapy for GI cancers.