On September 7, 2023 Daiichi Sankyo (TSE: 4568) reported that the first patient has been dosed in a first-in-human phase 1/2 trial evaluating DS-3939 in patients with several types of advanced solid tumors including non-small cell lung, breast, urothelial, ovarian, biliary tract, and pancreatic cancer (Press release, Daiichi Sankyo, SEP 7, 2023, View Source [SID1234634989]).

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DS-3939 is a specifically engineered potential first-in-class tumor-associated mucin-1 (TA-MUC1) directed antibody drug conjugate (ADC) designed using Daiichi Sankyo’s proprietary DXd ADC technology. TA-MUC1 is a tumor-specific transmembrane glycoprotein and is overexpressed in most human epithelial cancers, making it a promising target for cancer therapy.1,2 Currently, there are no TA-MUC1 directed therapies approved for any type of cancer.

"With DS-3939, we are pairing our unique DXd antibody drug conjugate technology with a TA-MUC1 antibody in order to evaluate this novel treatment strategy for patients with several types of advanced cancer," said Mark Rutstein, MD, Global Head, Oncology Clinical Development, Daiichi Sankyo.

"The initiation of this trial is a significant milestone as DS-3939 is an important new addition to our growing DXd ADC portfolio, which now consists of six ADCs in clinical development, and represents our ongoing commitment to create new standards of care for patients with cancer."

About the Phase 1/2 Trial

The two-part, multicenter, open-label, first-in-human phase 1/2 trial will assess the safety and efficacy of DS-3939 in patients with locally advanced, metastatic or unresectable solid tumors not amenable to standard of care treatment for each tumor type.

The first part of the trial (dose escalation) will assess the safety and tolerability of increasing doses of DS-3939 to determine the maximum tolerated dose and/or the recommended doses for expansion (RDEs) in patients with locally advanced, metastatic, or unresectable solid tumors.

The second part of the trial (dose expansion) will consist of multiple expansion cohorts to continue to assess the safety and efficacy of DS-3939. The trial will evaluate safety endpoints including dose-limiting toxicities and adverse events, and efficacy endpoints including overall response rate, disease control rate, duration of response, time to response, progression-free survival and overall survival. Pharmacokinetic and biomarker endpoints also will be assessed.

The trial is expected to enroll patients across multiple sites globally, including Asia and North America. For more information, please visit ClinicalTrials.gov.

About TA-MUC1

TA-MUC1 is a tumor-specific transmembrane glycoprotein with aberrant glycosylation due to changes of the expression patterns of some sialyltransferases.1 Based on the overexpression of TA-MUC1 in most human epithelial cancers, it is an attractive target for cancer therapy.2 Currently, there are no TA-MUC1 directed therapies approved for any type of cancer.

About DS-3939

DS-3939 is an investigational potential first-in-class TA-MUC1 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, DS-3939 is comprised of a humanized anti-TA-MUC1 antibody licensed from Glycotope GmbH, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

On September 7, 2023 Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, reported poster presentations of data from IMerge, the Company’s Phase 3 clinical trial evaluating its first-in-class investigational telomerase inhibitor imetelstat vs. placebo in patients with lower risk myelodysplastic syndromes (MDS) at the eleventh annual Society of Hematologic Oncology Annual Meeting (SOHO) held in Houston, Texas and virtually (Press release, Geron, SEP 7, 2023, View Source [SID1234634988]).

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"For imetelstat-treated patients in the IMerge Phase 3 trial, the durability of transfusion independence, substantial increases in hemoglobin, robust treatment effect across subgroups and improvement in fatigue, along with a manageable safety profile, represent, we believe, an unparalleled clinical benefit in lower risk MDS," said Faye Feller, M.D., Executive Vice President, Chief Medical Officer of Geron. "It was particularly meaningful for these data to be presented at SOHO, where there was a broad array of professionals who touch the lives of patients with lower risk MDS."

The following posters presented at SOHO reflected data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) annual meeting in June 2023:

MDS-572: Continuous Transfusion Independence with Imetelstat in Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Neoplasms Relapsed/Refractory/Ineligible for Erythropoiesis-Stimulating Agents in IMerge Phase III
MDS-604: Improvement of Patient-Reported Fatigue in IMerge Phase III Trial of Imetelstat vs Placebo in Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Neoplasms Relapsed/Refractory/Ineligible for Erythropoiesis-Stimulating Agents
MDS-605: Disease Modifying Activity of Imetelstat in Patients with Heavily Transfused Non-Del(5q) Lower-Risk Myelodysplastic Neoplasms Relapsed/Refractory/Ineligible for Erythropoiesis-Stimulating Agents in IMerge Phase III
The posters are available under the Publications section of Geron’s website: View Source

As previously reported, in the IMerge Phase 3 clinical trial, the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (p<0.001), with median TI duration approaching one year for imetelstat 8-week TI responders. Mean hemoglobin levels in imetelstat-treated patients increased significantly (p<0.001) over time compared to placebo patients. Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category. Patient-reported outcomes (PRO) data reported a sustained meaningful improvement in fatigue for imetelstat-treated patients vs. placebo. Consistent with prior imetelstat clinical experience, the most common serious adverse events were primarily short-lived, manageable cytopenias. Treatment with imetelstat vs. placebo led to greater reduction in variant allele frequency (VAF) in multiple genes associated with lower risk MDS, which correlated with clinical endpoints of TI response, longer TI duration and increase in hemoglobin levels, suggesting the potential of imetelstat to modify the disease.

Based on results from the IMerge Phase 3 clinical trial, Geron submitted a New Drug Application for imetelstat in lower risk MDS that was accepted for review by the FDA and assigned a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024.

About IMerge Phase 3

The Phase 3 portion of the IMerge Phase 2/3 study is a double-blind, 2:1 randomized, placebo-controlled clinical trial to evaluate imetelstat in patients with IPSS Low or Intermediate-1 risk (lower risk) transfusion dependent MDS who were relapsed after, refractory to, or ineligible for, erythropoiesis stimulating agent (ESA) treatment, had not received prior treatment with either a HMA or lenalidomide and were non-del(5q). To be eligible for IMerge Phase 3, patients were required to be transfusion dependent, defined as requiring at least four units of packed red blood cells (RBCs), over an eight-week period during the 16 weeks prior to entry into the trial. The primary efficacy endpoint of IMerge Phase 3 is the rate of red blood cell transfusion independence (RBC-TI) lasting at least eight weeks, defined as the proportion of patients without any RBC transfusion for at least eight consecutive weeks since entry to the trial (8-week TI). Key secondary endpoints include the rate of RBC-TI lasting at least 24 weeks (24-week TI), the duration of TI and the rate of hematologic improvement erythroid (HI-E), which is defined under 2006 IWG criteria as a rise in hemoglobin of at least 1.5 g/dL above the pretreatment level for at least eight weeks or a reduction of at least four units of RBC transfusions over eight weeks compared with the prior RBC transfusion burden. A total of 178 patients were enrolled in IMerge Phase 3 across North America, Europe, Middle East and Asia.

About Imetelstat

Imetelstat is a novel, first-in-class investigational telomerase inhibitor exclusively owned by Geron and being developed in hematologic malignancies. Imetelstat has been granted Fast Track designation by the U.S. Food and Drug Administration for both the treatment of adult patients with transfusion dependent anemia due to Low or Intermediate-1 risk MDS that is not associated with del(5q) who are refractory or resistant to an erythropoiesis stimulating agent, and for adult patients with Intermediate-2 or High-risk myelofibrosis (MF) whose disease has relapsed after or is refractory to janus associated kinase (JAK) inhibitor treatment. Imetelstat is currently not approved by any regulatory authority.

On September 7, 2023 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its Chief Financial Officer, Ryan Confer, will be providing an overview of the Company’s gene therapies for cancer and diabetes at the upcoming H.C. Wainwright 25th Annual Global Investment Conference (Press release, Genprex, SEP 7, 2023, View Source [SID1234634987]).

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Event: H.C. Wainwright 25th Annual Global Investment Conference

Conference Dates: Sep. 11 – 13, 2023

Presentation Date: Wednesday, Sep. 13, 2023

Presentation Time: 3:00 p.m. ET

Presenter: Ryan Confer, Genprex’s Chief Financial Officer

Presentation link: https://bit.ly/3sFJE6U

A recording of this presentation will be available for replay on Genprex’s website for a period of time.

Mr. Confer will deliver an overview of the Company’s pioneering gene therapies for cancer and diabetes and will be available for in-person one-on-one meetings with investors through the conference platform.

For those interested in meeting Genprex management during the conference, please request a meeting through the conference portals or reach out to Investor Relations at [email protected].

On September 7, 2023 Eagle pharmaceuticals presented its corporate presentation (Presentation, Eagle Pharmaceuticals, SEP 7, 2023, View Source [SID1234634986]).

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On September 7, 2023 Can-Fite Biopharma presented its corporate presentation (Presentation, Can-Fite BioPharma, SEP 7, 2023, View Source [SID1234634985]).

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