On February 11, 2025 CSL reported its results Presentation for the Half-year ended 31 December 2024 (Presentation, CSL, FEB 11, 2025, View Source [SID1234652128]).

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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.

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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]).

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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 10, 2025 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported the following update on FLAMINGO-01 open label HLA data (Press release, Greenwich LifeSciences, FEB 10, 2025, View Source [SID1234650131]).

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Analysis of the open label data from FLAMINGO-01 has commenced and has been conducted in a manner that maintains the study blind. A preliminary review of FLAMINGO-01 HLA data in both the HLA-A*02 treated and placebo arms and the third open label arm with all other HLA types, shows that approximately 46% of all screened patients have at least one HLA-A*02 allele from either parent.

The main purpose of the open label arm is to investigate the safety and efficacy of GLSI-100 vaccination in patients who do not have an HLA-A*02 allele. It is possible that the open label arm may be large enough to draw immune response and efficacy conclusions. As discussed below, the HLA type can be analyzed by race and ethnicity in those patients who self-reported such information.

CEO Snehal Patel commented, "The review of open label data and the ability to look at multiple patient populations in the Phase III trial will be much greater than was possible in the Phase IIb trial. The HLA-A*02 prevalence of 46% in all screened patients meets our expectations of 40-50% prevalence and supports our sample size estimates for the trial and the interim analysis. We are also interested in studying the 8% of patients who have received HLA-A*02 alleles from both parents, as the mechanism of action in these double HLA-A*02 patients could lead to greater immune response and efficacy."

Mr. Patel further added, "There may be other subgroups of HLA types that can be analyzed in addition to the main arms. Approximately 92% of the patients that are in the HLA-A*02 arms have a second HLA-A type from the other parent that is not HLA-A*02 and could be any of 6 or more other prevalent HLA-A types. We can compare these HLA combinations against each other for immune response and clinical outcome, which could allow for subgroup analysis of HLA-A combinations. The prevalence of various HLA-A types by race or ethnicity may also help to inform the Company in its initial commercial development strategy by suggesting those markets where the most efficacious HLA patient populations may reside."

Background on FLAMINGO-01 Trial Design and HLA Testing

The design of the Phase III trial can be seen here. The trial is a prospective, randomized, double-blinded, multi-center study. The patient population is defined by major screening criteria and is stratified to balance the patient population between the treated and placebo arms of the trial.

As currently designed, approximately 500 patients with the HLA-A*02 allele will be randomized to receive GLSI-100 (GP2 and GM-CSF) or placebo control in the first two pivotal arms of the trial with a planned interim analysis.

In addition, patients without the HLA-A*02 allele will be enrolled in the third open label arm where all patients will receive GLSI-100 and where all endpoints will be open label. This non-HLA-A*02 arm was recently expanded from 100 to 250 patients based on recommendation of the steering committee and review/approval by the FDA and EMA.

HLA-A*02 blinded arms: A patient has 2 HLA-A genes, one from each parent, thus a single HLA-A*02 patient has received the HLA-A*02 allele from one parent. A double HLA-A*02 patient has received the HLA-A*02 allele from both parents. Both single and double HLA-A*02 patients are enrolled in the HLA-A*02 treated and placebo arms, which are blinded. Those patients who have a single HLA-A*02 allele will also have a second HLA-A gene of any other type.
Double HLA-A*02 Potential Mechanism of Action: Theoretically, a double HLA-A*02 patient may have double the amount of HLA-A*02-GP2 complex presented to the immune system to create cancer killing T-cells during the GLSI-100 vaccinations, and as a cancer cell recurs, the HER2 positive recurring cancer cells may have double the amounts HLA-A*02-GP2 complex for the trained T-cells to target and kill. It may be interesting to investigate immune or clinical response differences between single and double HLA-A*02 patients.
Open Label non-HLA-A*02 Third Arm: If a patient has no HLA-A*02 alleles, they will have 2 different or identical non-HLA-A*02 alleles. These non-HLA-A*02 patients are enrolled in the open label arm, where the immune or clinical response can be assessed as a group or by each HLA-A type, including double HLA-A types, providing the number of patients is sufficiently high to draw conclusions or trends.
Additional Information: A central laboratory in the US is sequencing the DNA of patients to determine both HLA-A allele types. The technology is available to sequence the HLA-B and HLA-C alleles, in addition to the HLA-A allele, to further assess other HLA types that may associate with GP2 to create a positive therapeutic effect. GP2 prediction binding algorithms may suggest that some HLA-B or HLA-C alleles may associate similarly to or stronger than HLA-A*02.
Phase IIb Clinical Trial Results

A variety of HLA types are predicted to associate with GP2 based on binding algorithms, and such binding can be tested in preclinical experiments. However, HLA-A*02 is the most common HLA type, thus it was studied first, and all patients in the Phase IIb trial had at least one HLA-A*02 allele. The HLA data collected did not identify if a patient was double HLA-A*02, nor were any other non-HLA-A*02 alleles identified.

Preliminary Review of FLAMINGO-01 HLA Data

Estimates of HLA prevalence by race are available in literature. As there are many sources and population studies to reference, a general consensus is that HLA-A*02 is prevalent in about 40-50% of the Caucasian population, which is the majority of the population in the US and Europe where the study is being conducted. To assess the prevalence of various HLA-A alleles by race, we have been collecting race and ethnicity data on all patients screened. We have summarized the preliminary data available to date in a blinded manner and have observed the results below. It is important to note that this preliminary summary may not reflect results at the end of the study.

Across all screened patients, HLA-A*02 prevalence is about 46%. The double HLA-A*02 prevalence, in patients who have received HLA-A*02 alleles from both parents, is about 8%. Because there are 2 HLA-A genes, one from each parent, the total of all prevalence percentages exceeds 100% and is less than 200% because of double HLA-A types. The HLA-A*03, HLA-A*24, and HLA-A*01 prevalences are about 20-25% for each allele. The HLA-A*11, HLA-A*68, HLA-A*29, and HLA-A*30 prevalences are about 9-12% for each allele.
In those screened patients who self-report as White, at least single or double HLA-A*02 genes are prevalent in approximately 50% of the patients. The double HLA-A*02 alleles are prevalent in 10% of these patients screened. The next most prevalent HLA-A types in the White populations are HLA-A*01 (29%), HLA-A*03 (21%), HLA-A*24 (19%), HLA-A*68 (10%), HLA-A*29 (13%), and HLA-A*11 (9%).
In those screened patients who self-report as Hispanic or Latino, at least single or double HLA-A*02 alleles are prevalent in approximately 50% of the patients. The double HLA-A*02 genes are prevalent in 7% of these patients screened. The next most prevalent HLA-A types in the Hispanic or Latino populations are HLA-A*01 (20%), HLA-A*24 (22%), HLA-A*68 (22%), HLA-A*30 (18%), HLA-A*29 (13%), and HLA-A*11 (13%).
In those screened patients who self-report as Black or African-American, at least single or double HLA-A*02 alleles are prevalent in approximately 40% of the patients. The next most prevalent HLA-A types in the Black or African-American populations are HLA-A*68 (33%), HLA-A*03 (27%), HLA-A*30 (27%), HLA-A*24 (13%), HLA-A*29 (13%), and HLA-A*23 (13%).
In those screened patients who self-report as Asian, at least single or double HLA-A*02 alleles are prevalent in approximately 17% of the patients. The other prevalent HLA-A types are HLA-A*24 (42%), HLA-A*33 (42%), HLA-A*11 (25%), and HLA-A*03 (25%).
The above preliminary Flamingo-01 open label data on HLA-A alleles by race and ethnicity is similar to the data available in literature. If any of the non-HLA-A*02 alleles have a strong association to GP2, it may be interesting to study the immune response and efficacy of GLSI-100 in patients with one allele of that type and one allele that is HLA-A*02 in addition to in patients with the double HLA-A*02 alleles.

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US clinical sites from university-based hospitals and cooperative networks with plans to expand into Europe and to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients will be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types will be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On February 10, 2025 iBio, Inc. (NYSEA:IBIO), reported financial results for the second quarter ended Dec. 31, 2024, and provided a corporate update on its progress (Press release, iBioPharma, FEB 10, 2025, View Source [SID1234650132]).

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"In our second fiscal quarter we further strengthened our leadership with key Board appointments, reinforcing our commitment to innovation and execution as we work to develop next-generation therapeutics," said CEO and Chief Scientific Officer Dr. Martin Brenner, Ph.D. "Following more recent developments, we also want to highlight the significant strides we have made in advancing our preclinical pipeline with the in-licensing of potentially best-in-class IBIO-600, the notable discovery of a novel Activin E antibody, and the launch of a bispecific antibody program targeting myostatin/activin A. We are excited by the momentum we have built through these results and remain focused on leveraging our AI-driven platform as we aim to transform the treatment landscape for patients with cardiometabolic diseases and obesity, offering hope for more effective, targeted therapies addressing the underlying causes of these conditions while improving overall metabolic health and quality of life."

Fiscal Second Quarter 2025 & Recent Corporate Updates:

Discovered a novel antibody targeting activin E in collaboration with AstralBio, leveraging iBio’s Machine-Learning Antibody Engine to overcome significant technical challenges, demonstrating the platform’s ability to engineer innovative therapeutics potentially for cardiometabolic disease and obesity.

Expanded iBio’s cardiometabolic and obesity program with IBIO-600, the long-acting anti-myostatin antibody in-licensed from AstralBio in January. IBIO-600 was discovered by AstralBio through the use of iBio’s Machine-Learning Antibody Engine and was designed for subcutaneous administration with the potential for an extended half-life.

Initiated a bispecific antibody program targeting myostatin/activin A to promote weight loss, muscle preservation, and prevent weight regain with plans for clinical investigation in obesity and cardiometabolic disorders in 2026. The program leverages iBio’s Machine-Learning Antibody Engine as well as the technology of IBIO-600.
Strengthened the Board of Directors with the appointments of biotech industry veterans David Arkowitz and António Parada on November 25, 2024.
In January we further extended our cash runway with the closing of a private placement offering with members of our Board of Directors and Officers, underscoring their confidence and support in our strategy to advance as a clinical-stage biotech.

Fiscal Second Quarter 2025 Financial Results:

Revenue of $0.2 million was reported for services provided to a collaborative partner during the quarter ended Dec. 31, 2024.

R&D and G&A expenses for the second quarter of fiscal 2025 totaled approximately $4.6 million as compared to $4.5 million in the same period of fiscal year 2024, an increase of approximately 3%. This slight increase is a result of additional spending on consumables supplies and research related activities offset by lower G&A personnel related costs, consulting fees and outside services spending. Net loss from continuing operations for the second quarter ended Dec. 31, 2024, was approximately $4.4 million, or $0.48 per share, compared to a net loss of approximately $4.5 million, or $2.42 per share, in the same period of fiscal 2024.

Cash, cash equivalents and restricted cash as of Dec. 31, 2024, was approximately $7.2 million, inclusive of $0.2 million of restricted cash.