On February 11, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies, reported encouraging new clinical data from the ongoing Phase 1 investigator-sponsored trial of INB-100, an allogeneic gamma-delta T cell therapy designed to help patients with complex leukemias, including AML (Press release, In8bio, FEB 11, 2025, View Source [SID1234650171]). INB-100, given following hematopoietic stem cell transplantation (HSCT), is demonstrating the potential to achieve durable long-term remissions and improved survival. The data will be presented at the 2025 Tandem Meetings | Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, hosted in Honolulu, HI.

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!

Highlights

100% of AML patients remain relapse-free
All treated AML patients in both the original and expansion cohorts through January 17, 2025 remain in complete remission. The original cohort of AML patients has reached a median CR of 23.3 months, with several patients in remission for over three years. The median duration across all treated AML patients (N=9) is 20.1 months.
Trial displays improved survival outcomes vs. standard treatment
When compared with real-world historical data, INB-100 demonstrated significantly higher survival rates:
INB-100:
All patients – 90.9% PFS and OS of 100% at one-year; and
AML patients – 100% PFS and OS of 100% at one-year.
Historical controls in AML:
Center for International Blood and Marrow Transplant Research (CIBMTR) demonstrate a PFS of 67.8% and OS of 74.7% at one-year; and
Kansas University Cancer Center (KUCC) PFS of 57.4% and OS of 66.7% at one-year.
Results demonstrate activity even with older, high-risk patients receiving reduced intensity conditioning (RIC)
Relapse is the most significant challenge leading to mortality for patients undergoing HSCT.
Many of the patients enrolled in the study were older (median age = 68), had complex, high-risk disease or had failed multiple prior therapies, including CAR-T treatments, yet they achieved durable, long-term remission with manageable side effects.
Therapy appears to be well-tolerated without significantly impacting patient Quality of Life
No cytokine release syndrome (CRS) or neurotoxicity; (ICANs)
Tolerable graft-versus-host disease (GvHD) in-line with historical data that is managed with steroids; and
Limited, mild infections.
Dr. Joseph P. McGuirk, Schutte-Speas Professor of Hematology-Oncology, Division Director, Hematologic Malignancies and Cellular Therapeutics Medical Director, Blood and Marrow Transplant, The University of Kansas Cancer Center, commented, "These data suggest that the addition of allogeneic INB-100 gamma-delta T cells appears to have the potential to support durable relapse-free remissions in high-risk leukemia patients, with 100% of treated AML patients remaining in remission after a median follow-up of almost two years post-transplant. Typically, patients receiving reduced-intensity conditioning face substantial risks of relapse within a year, and those who relapse are often left with very few treatment options. INB-100 is not only helping patients avoid this common relapse timeline but is doing so while helping to preserve their quality of life. These results are truly exciting. We are seeing something we rarely encounter in high-risk leukemia patients: sustained, durable remissions with minimal side effects to date. These continued results of INB-100, with the manageable toxicity profile, suggest it could become an attractive cellular therapy with the potential to extend survival in this difficult-to-treat patient population."

William Ho, Chief Executive Officer and co-founder of IN8bio, added, "We’re incredibly pleased with our efforts to continue to deliver consistent, long-term remission results with INB-100. It’s rare to see 100% relapse-free survival in high-risk AML patients, especially over a prolonged period. For patients who may not have had a clear path forward in the past, INB-100 is providing hope, extending survival, and demonstrating the potential to change the standard-of-care. What makes this even more exciting is the safety profile we’ve observed. Gamma-delta T cells are showing that they can do the job of fighting residual cancer cells without causing significant side effects like CRS or neurotoxicity—issues that often plague other cell therapies. As we continue to enroll patients and expand the trial network, we are working diligently to lay the groundwork for the future regulatory pathway towards a potential registrational trial. The IN8bio team is working hard to de-risk the future path to approval and to bring this innovative therapy towards broader patient access. We are committed to providing further updates later this year as we build momentum toward this goal."

Conference Call Details

IN8bio will host a conference call and webcast today, Tuesday, February 11, 2025, at 8:30 am ET. The webcast can be accessed by clicking this link and can also be accessed on the Events & Presentations page of the Company’s website. To participate in the live call, please register using this link. It is recommended that participants register at least 5 minutes in advance of the call. Once registered, participants will be informed of the dial-in number and will be provided a unique PIN.

For more information about the study – including detailed findings, conclusions and next steps – please visit the Company’s poster being presented at the American Society of Transplantation and Cellular Therapy conference: View Source

On February 11, 2025 Ellipses Pharma Limited ("Ellipses"), a global drug development company focused on accelerating the development of cancer treatments through an innovative drug development model, reported it is to develop a next-generation immuno-oncology drug which could address the needs of cancer patients who do not respond to existing checkpoint inhibitors (Press release, Ellipses Pharma, FEB 11, 2025, View Source [SID1234650187]).

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!

Ellipses has agreed to in-license global rights to ‘GENA-104’, a first-in-class immuno-oncology monoclonal antibody that targets CNTN4, a recently discovered checkpoint protein that is highly expressed in a wide range of tumour types. ‘GENA-104’ was first discovered by Genome & Company ("Genome"), a South Korea-based biotechnology company focusing on discovery and development of novel cancer target antibodies and antibody-drug conjugates (ADCs). Under the agreement with Genome, Ellipses will take responsibility for all future clinical development of GENA-104 under the identifier EP0089.

In October 2024, key research findings were published in the prestigious journal Science Immunology, demonstrating the potential of EP0089 to target CNTN4 as a novel immuno-oncology treatment strategy.

A Phase 1 IND for EP0089 was approved by the South Korean Ministry of Food and Drug Safety in January 2024. Ellipses expects to initiate a Phase 1 clinical trial in South Korea during 2025 with expansion of involvement to the US and Europe following the relevant regulatory approvals.

Professor Sir Chris Evans, OBE, Executive Chair of Ellipses, commented: "We are delighted to be taking forward this very exciting drug candidate through the clinical trial process. The outstanding potential of EP0089 fits well with our strategy of identifying the very best drug candidates, developing them at pace and getting them to patients as fast as possible."

Professor Tobias Arkenau, Global Head of Drug Development and Chief Medical Officer of Ellipses commented: "We are very excited to in-license EP0089, the first biological agent to be added to our pipeline. Targeting CNTN4 is a new approach that blocks the CNTN4-APP checkpoint interaction on T cells, promoting tumour cell killing. This approach could be particularly of interest across a range of cancers which respond poorly to conventional checkpoint inhibitors."

Y.S. (Yoo Seok) Hong, CEO of Genome & Company, commented: " GENA-104’s excellent potential as a novel target immuno-oncology agent has already been confirmed through preclinical studies, and with the IND approval for a phase 1 trial in Korea, we look forward to Ellipses Pharma utilising its strong financial resources and extensive experience and capabilities in oncology drug development to take forward GENA-104 as a novel immuno-oncology agent."

On February 11, 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 it has received first country regulatory approval in Europe to begin recruiting for its Phase 3 pivotal trial protocol 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) (MB-108) (Press release, Moleculin, FEB 11, 2025, https://moleculin.com/moleculin-receives-first-country-approval-in-europe-to-begin-recruiting-for-the-miracle-phase-3-r-r-acute-myeloid-leukemia-aml-pivotal-trial/ [SID1234650172]). In Ukraine the final necessary regulatory approvals from the Ministry of Health were received last week. This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) will be a global trial, including sites in the US, Europe and the Middle East.

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!

"We continue to make solid progress across our site initiation and enrollment efforts. Achieving this important milestone of receiving our first country’s approval puts us another step closer to getting this study well underway and further bolsters our confidence that we’re on track for unblinded preliminary data from the first 45 subjects in the second half of this year. Our team remains committed to operational execution and face-to-face meetings with investigators, over 30 investigators in 11 countries of which I personally have met," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Building on this momentum, we are focused on getting more of the initial 25 sites we have selected to date on board and enrolling for the study."

The MIRACLE study, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). This early unblinding will yield 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and 15 subjects with just HiDAC. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

For Part B of the trial, approximately 244 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. This increase from 240 to 244 subjects represents the statistical "cost" of the additional interim unblinding.

For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756.

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 European Medicines Agency (EMA).

On February 11, 2025 Oqory, Inc., a private biopharmaceutical company dedicated to developing next-generation antibody drug conjugates (ADCs) for the treatment of cancer, reported that Michael King, Chief Executive Officer, will present data at Oppenheimer’s 35th Annual Healthcare Life Sciences Conference (Press release, Oqory, FEB 11, 2025, View Source [SID1234650188]).

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!

Details are as follows:

Date: Wednesday, February 12, 2025

Time: 12:40 PM ET

Webcast link for conference participants:
View Source

On February 11, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported that it will webcast management participation as follows (Press release, Regeneron, FEB 11, 2025, View Source [SID1234650173]):

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!

TD Cowen 45th Annual Health Care Conference at 11:50 a.m. ET on Tuesday, March 4, 2025
Leerink Partners 2025 Global Healthcare Conference at 8:00 a.m. ET on Tuesday, March 11, 2025

The sessions may be accessed from the "Investors & Media" page of Regeneron’s website at View Source Replays and transcripts of the webcasts will be archived on the Company’s website for at least 30 days.