Rigel to Participate in Upcoming September Investor Conferences

On August 26, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported that Raul Rodriguez, the company’s president and chief executive officer, and Dean Schorno, the company’s chief financial officer, will participate in the following investor conferences in September (Press release, Rigel, AUG 26, 2025, View Source [SID1234655483]):

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Citi’s 2025 Biopharma Back to School Conference in Boston, MA
Rigel will participate in one-on-one meetings on Tuesday, September 2.

Cantor Global Healthcare Conference 2025 in New York, NY
Rigel will participate in one-on-one meetings and present a company overview on Wednesday, September 3, at 8:35 a.m. ET.

2025 Wells Fargo Healthcare Conference in Boston, MA
Rigel will participate in one-on-one meetings on Thursday, September 4.

H.C. Wainwright 27th Annual Global Investment Conference in New York, NY
Rigel will participate in one-on-one meetings and present a company overview on Tuesday, September 9, at 1:30 p.m. ET.
To access the live webcasts or archived recordings of the Cantor and H.C. Wainwright Conference presentations, visit the Investor Relations section of the company’s website at www.rigel.com. Please connect to Rigel’s website prior to the start of the live webcast to allow for any software downloads.

EMA approves ODD for OXC-101 in AML

On August 26, 2025 Oxcia reported its drug candidate OXC-101 received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (Press release, Oxcia, AUG 26, 2025, View Source;utm_medium=rss&utm_campaign=ema-approves-odd-for-oxc-101-in-aml [SID1234655482]). Now, the European Medicines Agency (EMA) has also granted OXC-101 ODD approval for European markets.

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This approval significantly strengthens Oxcia’s position, both from a strategic, regulatory and commercial standpoint, according to Oxcia’s CEO Ulrika Warpman Berglund:

-These dual approvals give us up to 10 years of market exclusivity in the EU and 7 years in the US, accelerated regulatory processes and significant cost savings. EMA’s approval also provides support and scientific advice from EMA’s expert committee (COMP) for orphan drugs which is a large advantage.

EMA is also evaluating whether the drug candidate can offer significant benefit compared to existing treatments. Obtaining ODD from both the FDA and EMA, two of the world’s largest drug regulatory authorities, strengthens scientific credibility and differentiation from competitors. It facilitates the work of raising additional venture capital and signing licensing agreements with pharmaceutical companies.

Briefly about OXC-101 in AML

Oxcia’s lead drug candidate OXC-101 is a so-called "first-in-class" mitotic MTH1 inhibitor. It has a dual mechanism of action that allows it to target a weakness of cancer cells – their high levels of oxidative stress and their propensity to develop DNA damage. In short, OXC-101 induces further oxidative stress and prevents the cancer cells from repairing the DNA damage.

Preclinical studies have shown that OXC-101 significantly reduces tumor growth and prolongs survival in models of acute myeloid leukemia (AML). In addition, there is support for further development from clinical results from a phase I study in patients with advanced hematological cancers. In addition to OXC-101 having potential as a monotherapy with better efficacy than cytarabine (which is one of the standard treatments for AML) in AML disease models, OXC-101 may also further improve the efficacy of treatment with various standard combinations (various chemotherapies).

Oxcia is currently conducting an expansion study, a combined phase I/II study, in a selected group of patients with relapsed/recurrent AML. The treatment is given in combination with one of the standard treatments (idarubicin). Oxcia has obtained a Vinnova grant for the expansion phase of the study. The aim of the study is to confirm the promising preliminary effects previously observed, and to lay the foundation for a pivotal phase II study that could form the basis for a regulatory accelerated approval. In several of the patients in the MAATEO study, clinical benefits have been observed with a partial response and some stable disease up to 5 months, which is a long time for this aggressive disease in this phase. Some improvement in quality of life has also been reported.

In collaboration with Dr. Tom Erkers and Nona Struyf, Scilifelab/Karolinska Institute, so-called precision medicine studies are being carried out on bone marrow samples from patients in the MAATEO study. These studies aim to identify the patients with the best response. The method may be used to stratify patients in future studies.

Marker Therapeutics Provides Update on Phase 1 APOLLO Study Highlighting Encouraging Overall Response Rates in Relapsed Lymphoma

On August 26, 2025 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications, reported an update on the progress and clinical observations from the Phase 1 APOLLO study (Press release, Marker Therapeutics, AUG 26, 2025, View Source [SID1234655481]).

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The Company’s Phase 1 APOLLO study (clinicaltrials.gov identifier: NCT05798897) is a multicenter, open-label clinical study investigating MT-601, a Multi-Antigen Recognizing (MAR)-T cell product, in patients with lymphoma who have relapsed after anti-CD19 CAR-T cell therapy or for whom anti-CD19 CAR-T cell therapy is not an option. Today, Marker is reporting an update on the safety and efficacy data from the dose escalation portion of the study showing a favorable safety profile across all evaluated doses (dose range 100×106-400×106 cells) and a 66% objective response rate in patients with NHL, with 50% demonstrating complete response.

A total of 24 B-cell lymphoma patients have been treated with MT-601 across 7 U.S. clinical sites, including 15 patients with Non-Hodgkin Lymphoma (NHL) and 9 patients with Hodgkin Lymphoma (HL). At the time of the data cutoff (June 2025), 12 NHL and 9 HL patients have been assessed. Patients with NHL and HL received doses ranging from 100×106-400×106 cells and showed objective responses and a favorable safety profile.

"We are very excited and encouraged by the progress of the study," said Juan Vera, M.D., President and CEO of Marker Therapeutics. "The safety and preliminary efficacy data from our Phase 1 APOLLO study underscore the potential of MT-601 in heavily pre-treated patients with lymphoma, who have relapsed after multiple lines of therapy, including CAR-T cells and bispecific antibodies. While CAR-T cells have gained acceptance in the treatment of lymphoma, with approximately 8,000 patients treated globally in 2024, 40-60% of such patients have disease progression within the first year of treatment. We believe that MT-601 could address this critical unmet need and offer new hope to patients who have exhausted multiple lines of treatment, including CAR-T cell therapy."

Efficacy and Duration of Response

The 12 NHL patients received doses ranging from 100×106-200×106 cells and had undergone multiple lines of therapy (median of 5 prior lines of therapy), including anti-CD19 CAR-T cells (n=9) and bispecific antibodies (n=4); dual exposed patients (n=4). 8 out of 12 NHL patients had objective responses (66%), with 6 patients demonstrating complete response (CR) as best response (50%). Durable responses were observed (range 3-24 months) with 5 patients showing continued response ≥6 months, including 3 patients with ≥12 months durability.

HL patients received doses ranging from 200×106-400×106 cells and had undergone a median of 8 prior lines of therapy. Seven out of 9 HL patients had objective responses (78%), with 1 patient demonstrating CR (11%) highlighting the versatility of MT-601 across multiple histologies.

Safety Profile

The dose escalation portion of the study tested doses ranging from 100×106-400×106 cells in patients with B-cell lymphoma. To date, no DLTs have been reported at the highest dose (400×106 cells). Infusion of MT-601 was well tolerated in all study participants, with no observation of ICANS and two reported Grade 1 cytokine release syndrome (CRS) events (fever; no treatment was required). Patients were treated with or without lymphodepleting chemotherapy prior to receiving infusions of MT-601. No change in DLTs or ICANS was observed between patients treated with and without lymphodepletion. Data collected from the 24 patients treated demonstrated a robust safety profile with no reported serious adverse events reinforcing the benefit of MT-601.

Study Design and Dose Expansion

The Phase 1 APOLLO study is composed of a dose escalation phase, followed by a dose expansion phase. The dose escalation cohort evaluated the safety and optimum dose level of MT-601 with doses ranging from 100×106-400×106 cells. On June 17, the Safety Review Committee (SRC) cleared the pre-specified maximum dose (400×106 cells) for the dose expansion portion of the trial. The dose expansion will enroll patients with DLBCL who have relapsed after anti-CD19 CAR-T cells or who are ineligible for CAR-T therapy.

"The observed outcomes in NHL demonstrate that certain patients can achieve clinically meaningful responses with MT-601 even at lower doses of 100×106 or 200×106 cells," commented Dr. Vera. "We look forward to advancing the study to the dose expansion phase, where we will investigate MT-601 at the maximum dose of 400×106 cells in patients with relapsed DLBCL to potentially build upon these promising results."

Dr. Vera continued, "The encouraging efficacy in patients with NHL was achieved at doses ranging from 100×106-200×106 cells. We believe that investigating MT-601 at the maximum dose of 400×106 cells in the dose expansion cohort could have the potential to further improve the clinical efficacy and durability we are currently observing in patients with NHL. It is particularly encouraging that even at the highest dose level no DLTs were reported in the dose escalation phase. We will continue to closely monitor the safety and efficacy of MT-601 in treated patients and anticipate to provide another data update in the first half of 2026."Webcast Details

Marker will host a live, online-only webcast today at 8:30 am E.D.T. to discuss the clinical results and provide a corporate update. To attend the live event, please use this link to register. During the event, attendees will have the opportunity to submit written questions via the webcast platform’s Q&A feature. After the event, a recording will be made available for replay on the Company’s IR website under Events & Presentations for approximately 30 days.

About MT-601

The Company’s lead product, MT-601, is a multi-antigen recognizing (MAR) T cell product that utilizes a non-genetically modified approach that specifically targets six different tumor antigens upregulated in lymphoma cells (Survivin, PRAME, WT-1, NY-ESO-1, SSX-2, MAGEA-4). Marker is currently investigating MT-601 in the Company-sponsored Phase 1 APOLLO trial (clinicaltrials.gov identifier: NCT05798897) for the treatment of patients with lymphoma who have relapsed after or are not candidates for anti-CD19 CAR-T cell therapies.

About APOLLO

The APOLLO trial (clinicaltrials.gov Identifier: NCT05798897) is a Phase 1, multicenter, open-label study designed to evaluate the safety and efficacy of MT-601 in participants with relapsed or refractory lymphoma who have either failed anti-CD19 chimeric antigen receptor (CAR) T cell therapy or are not candidates for anti-CD19 CAR-T cell therapy. The primary objective of this exploratory Phase 1 clinical trial is to evaluate the optimum dose, safety, and preliminary efficacy of MT-601 in participants with various lymphoma subtypes. The APOLLO study is supported by the National Cancer Institute of the National Institutes of Health under Award Number R44CA291521.

About MAR-T cells

The multi-antigen recognizing (MAR) T cell platform (formerly known as multiTAA-specific T cells) is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s/donor’s blood capable of recognizing a broad range of tumor antigens. Unlike other T cell therapies, MAR-T cells allow the recognition of hundreds of different epitopes within up to six tumor-specific antigens, thereby reducing the possibility of tumor escape. Since MAR-T cells are not genetically engineered, Marker believes that its product candidates will be easier and less expensive to manufacture, with an improved safety profile compared to current engineered T cell approaches and may provide patients with meaningful clinical benefits.

Initial Data Shows 100% Disease Control in 5 Out of 5 Patients With Recurrent Glioblastoma With Two Patients in Near Complete Response Treated With ImmunityBio’s ANKTIVA®, NK Cell Therapy Plus Optune Gio® Device

On August 26, 2025 ImmunityBio, Inc. (NASDAQ: IBRX), a leading immunotherapy company, reported early findings from the first five recurrent glioblastoma patients treated with its investigational immune-boosting regimen including ANKTIVA (nogapendekin alfa inbakicept-pmln) in this pilot study (NCT06061809) (Press release, ImmunityBio, AUG 26, 2025, View Source [SID1234655480]). All five patients achieved 100% disease control with the regimen that combines ANKTIVA, an IL-15 agonist being studied for its ability to enhance natural killer (NK) cell activity, NK cell therapy (PD-L1 t-haNK), and Optune Gio Tumor Treating Fields. Of the 5 patients treated to date, 3 responded of which 2 at near complete response and the remaining 2 having stable disease to date. This finding of 5 out of 5 (100%) disease control in 2nd line recurrent glioblastoma receiving a chemotherapy free immunotherapy with Optune immune stimulating device is highly encouraging.

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We believe these early results of this combination immune-stimulating therapy are notable, given both the fact that GBM is a common form of brain tumor, as well as one that is exceptionally difficult to treat successfully with currently approved therapies. GBM has a single digit five-year survival rate for patients aged 45 and over. While the initial treatments in this trial involve a small cohort, the results are sufficiently encouraging for the company to plan a Phase 2 trial in second-line glioblastoma to further evaluate the potential for this combination treatment.

"GBM is a devastating type of brain cancer for which there are currently no durable treatment options, which is why this study has such important potential," said Dr. Simon Khagi, Medical Director of Neuro-Oncology at the Hoag Family Cancer Institute, and the Principal Investigator for this study. "In my years of treating patients with glioblastoma I have never experienced these near complete responses as well as the rapidity of the response as seen in these patients to date," he further stated. "There has been little advance in therapy for decades for glioblastoma. This chemotherapy free, immune-stimulating combination approach with ANKTIVA is highly promising and may represent a fundamental advance in therapy in patients with tumors of the brain."

"Although they are early, these results are very encouraging, given the high risk and low survival rates associated with GBM," added Dr. Patrick Soon-Shiong, Founder, Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio. "There is compelling evidence that ANKTIVA’s mechanism of proliferating NK and T cells plays an important role in treating patients with cancer Independent of tumor type. By activating the immune system the goal of providing durable responses is at hand. We believe these preliminary results in patients with GBM, whose lymphocyte counts (NK and T cells) are low as a consequence of radiation and chemotherapy after first-line treatment, can be rescued following ANKTIVA and NK cell therapy, and warrant the rapid expansion of this study in recurrent glioblastoma."

To learn more about this glioblastoma trial visit clinicaltrials.gov/study/NCT06061809 and cssifm.org

ANKTIVA, which is approved by the U.S. Food and Drug Administration with Bacillus Calmette-Guérin (BCG) for the treatment of patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, is being evaluated alone and with other agents in multiple studies for non-small cell lung cancer, non-Hodgkin lymphoma, Lynch syndrome (hereditary colon cancer), ovarian cancer and Human Papillomavirus (HPV) associated tumors. ANKTIVA is also being studied in Human Immunodeficiency Virus (HIV) and lymphopenia.

To learn more about ImmunityBio’s clinical trials, visit immunitybio.com/find-a-trial/ and cssifm.org.

Optune Gio is a registered trademark of Novocure GmbH.

About ANKTIVA (nogapendekin alfa inbakicept-pmln)

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response. A key component in the company’s BioShield platform, ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and driving the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones.

IMPORTANT SAFETY INFORMATION

INDICATION AND USAGE: ANKTIVA is an interleukin-15 (IL-15) receptor agonist indicated with Bacillus Calmette-Guérin (BCG) for the treatment of adult patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

WARNINGS AND PRECAUTIONS: Risk of Metastatic Bladder Cancer with Delayed Cystectomy. Delaying cystectomy can lead to the development of muscle-invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after a second induction course of ANKTIVA with BCG, reconsider cystectomy.

DOSAGE AND ADMINISTRATION: For Intravesical Use Only. Do not administer by subcutaneous or intravenous routes.

Please see the complete Prescribing Information for ANKTIVA at Anktiva.com.

Genmab Receives FDA Breakthrough Therapy Designation for Rinatabart Sesutecan (Rina-S®) in Advanced Endometrial Cancer (EC)

On August 26, 2025 Genmab A/S (Nasdaq: GMAB) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to rinatabart sesutecan (Rina-S), an investigational folate receptor alpha (FRα)-directed, TOPO1-inhibitor antibody-drug conjugate (ADC), for the treatment of adult patients with recurrent or progressive endometrial cancer (EC) who have disease progression on or following prior treatment with a platinum-containing regimen and a PD-(L)1 therapy (Press release, Genmab, AUG 26, 2025, View Source [SID1234655479]). BTD aims to expedite the development and review of investigational medicines by the U.S. FDA for serious or life-threatening diseases in cases where preliminary clinical evidence shows that a therapy may provide substantial improvements over available therapies.

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The designation was supported by previously published results from the endometrial cancer monotherapy dose expansion B2 cohort of the multi-part, Phase 1/2 RAINFOL-01 trial (NCT05579366), evaluating the safety and efficacy of Rina-S in solid tumors. In the B2 cohort, 64 patients with heavily pretreated advanced or recurrent EC whose disease had progressed on or after an anti-PD-(L)1 and platinum-based chemotherapy were enrolled and treated with Rina-S. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

"This Breakthrough Therapy Designation underscores the future potential of Rina-S as a treatment option for women diagnosed with advanced endometrial cancer, who face a poor prognosis after progressing on standard of care treatment," said Judith Klimovsky, M.D., Executive Vice President and Chief Development Officer of Genmab. "Rina-S reinforces Genmab’s determination to advance wholly owned antibody medicines in areas long overdue for innovation and our commitment to driving a strong clinical development program to help redefine what’s possible to treat gynecologic cancers."

Rina-S is advancing through late-stage development supported by a growing portfolio of clinical trials, including the ongoing Phase 1/2 RAINFOL-01 trial, the ongoing Phase 3 RAINFOL-02 trial (NCT06619236) in ovarian cancer, and several planned trials to evaluate Rina-S as a potential treatment option for a variety of tumor types, including a Phase 3 trial in endometrial cancer.

About the RAINFOLTM -01 Trial
RAINFOL-01 (NCT05579366) is an open-label, multicenter Phase 1/2 study, designed to evaluate the safety and efficacy of rinatabart sesutecan (Rina-S) Q3W at various doses in solid tumors that are known to express FRα. The study consists of multiple parts including Part A dose escalation; Part B tumor-specific monotherapy dose-expansion cohorts; Part C platinum-resistant ovarian cancer (PROC) cohort; Part D combination therapy cohorts; Part F a monotherapy endometrial cancer (EC) cohort; and Part K monotherapy QTc cohort in high-grade ovarian cancer.

About Endometrial Cancer
Endometrial cancer (EC) starts in the lining of the uterus, known as the endometriumii and ranks as the second most prevalent gynecologic cancer globally, with increasing incidence and mortality ratesiii,iv. Patients with advanced or recurrent EC have a relatively poor prognosis and treatment options are limited for those patients who have progressed following treatment with chemotherapy and immune checkpoint inhibitor. FRα is overexpressed on multiple tumors, including EC, making it a promising therapeutic target. Anti-tumor activity with Rina-S was observed across a broad range of FRα expression, and there are currently no approved FRα-directed therapies approved for the treatment of endometrial cancer.

About Rinatabart Sesutecan (Rina-S; GEN1184)
Rinatabart sesutecan (Rina-S; GEN1184) is an investigational ADC. It is composed of a novel human monoclonal antibody directed at folate receptor α (FRα), a novel hydrophilic protease-cleavable linker, and exatecan, a topoisomerase I inhibitor payload. The clinical trial program for Rina-S continues to expand including ovarian, endometrial and other cancers of unmet need.

The safety and efficacy of rinatabart sesutecan has not been established. Please visit www.clinicaltrials.gov for more information.