On March 18, 2025 Medison Pharma , a global company focused on accelerating access to highly innovative therapies in international markets, and Immunocore Holdings plc (NASDAQ: IMCR), a commercial-stage biotechnology company pioneering the delivery of transformative immunomodulatory medicines to radically improve patient outcomes for cancer, infectious diseases and autoimmune diseases, reported that KIMMTRAK (tebentafuspe) has received approval for health registration by the National Health Surveillance Agency (ANVISA), under the regulatory approval pathway for rare diseases (Press release, Medison Pharma, MAR 18, 2025, View Source [SID1234651240]).

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Tebentafuspe is indicated for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma (mum). This is the first therapy approved by ANVISA for metastatic uveal melanoma in Brazil [1],[2] .
Uveal melanoma is a rare and aggressive eye cancer with limited treatment options available at an advanced stage. Once metastasized, it carries a poor prognosis, and survival rates have remained largely unchanged for decades [ 3] . Developed by Immunocore,
tebentafuspe is an innovative immune mobilization monoclonal T-cell receptor therapy against cancer (ImmTAC) [ 4] designed to engage the body’s immune system to specifically target and destroy uveal melanoma cells.
As part of their long-standing global partnership, Medison and Immunocore are collaborating to bring this therapy to Latin America and beyond the U.S., Japan and Western Europe. Brazil has become the first country in the region to obtain approval for the drug, which offers a new treatment option for patients living with this disease [ 5] .

Mark Moyer , Senior Vice President of Regulatory Sciences at Immunocore said: "Following the approval of the registration, HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma in Brazil can now access tebentafuspe. With approvals in 39 countries, this underscores Immunocore’s firm commitment to providing our medicines to patients who can benefit from them around the world."

The approval of tebentafuspe in Brazil underscores Medison’s commitment to expanding access to innovative oncology treatments in Latin America. Victor Papamoniodis , Chief Commercial Officer of Medison Pharma, said: "We are proud of our global partnership with Immunocore that allows us to bring this treatment to Brazilian patients who have not had access to an approved therapy until now. Medison is dedicated to providing the most advanced therapies for rare and serious diseases to patients around the world."

Edson Paixão, General Manager of Medison Pharma in Brazil, Andean Region, Central America and the Caribbean , reinforced: "Medison remains committed to accelerating access to highly innovative therapies for Brazilian patients. The approval of tebentafuspe is an important milestone, and we are proud to make this therapy available to patients who can benefit from it."

About KIMMTRAK (tebentafuspe)

Solution for dilution for infusion with 100 micrograms/0.5 mL in a pack containing 1 vial of 0.5 mL.

INTRAVENOUS USE

ADULT USE

WARNING: Cytokine Release Syndrome (CRS), which can be serious or life-threatening, has occurred in patients receiving KIMMTRAK. Monitor for at least 16 hours after the first three infusions and then as clinically indicated.

Indications: As monotherapy for the treatment of adult patients with unresectable or metastatic uveal melanoma positive for human leukocyte antigen (HLA) A*02:01.

Patients treated with KIMMTRAK must have an HLA-A*02:01 genotype determined by any validated HLA genotyping assay.

Contraindications: Hypersensitivity to the active substance or to any of the excipients listed.

Warnings and Precautions: Most patients experienced CRS following tebentafuspe infusions. The diagnosis of CRS was most frequently based on pyrexia followed by hypotension and infrequently on hypoxia. Other symptoms commonly observed with CRS included chills, nausea, vomiting, fatigue, and headache. In most cases, CRS began on the day of infusion, with a median time to resolution of 2 days. Pyrexia was observed in nearly all cases of CRS, and in these patients, an increase in body temperature typically occurred within the first 8 hours after tebentafuspe infusion. CRS rarely (1.2%) led to discontinuation of treatment. Patients should be monitored for signs and symptoms of CRS for at least 16 hours after the first three tebentafuspe infusions in a hospital setting with immediate access to resuscitation drugs and equipment to manage CRS. If CRS is observed, immediate supportive care, including antipyretics, intravenous fluids, tocilizumab, or corticosteroids, should be initiated to prevent worsening of the syndrome or potential fatality, and monitoring should continue until resolution. With subsequent doses, patients should be closely monitored after treatment for early identification of signs and symptoms of CRS. Patients with comorbidities, including cardiovascular disorders, may be at increased risk of sequelae associated with CRS. Tebentafuspe treatment has not been studied in patients with clinically significant cardiac disease. Depending on the persistence and severity of CRS, tebentafuspe treatment should be withheld or discontinued. Acute skin reactions with infusion have been reported, including rash, pruritus, erythema, and skin edema. Cardiac events, such as tachycardia and sinus arrhythmia, have been observed in patients on treatment. Cases of QT prolongation have been reported following treatment with tebentafuspe. An electrocardiogram (ECG) should be performed on all patients before and after treatment, during the first 3 weeks and thereafter as clinically indicated. This medicine may potentiate QT interval prolongation, which increases the risk of serious ventricular arrhythmias such as "torsades de pointes", which is potentially fatal (sudden death). This medicine may cause hepatotoxicity. Therefore, it requires careful use, under strict medical supervision and accompanied by periodic controls of liver function, at the physician’s discretion.

Pregnancy and lactation: Women of childbearing potential must use effective contraception during treatment and for at least 1 week after the last dose of treatment. The drug is not recommended during pregnancy and in women of childbearing potential who are not using contraception. Pregnancy in women of childbearing potential should be ascertained before starting treatment. There is insufficient information on the excretion of tebentafuspe/metabolites in human milk. A risk to the newborn/infant cannot be excluded. Breast-feeding should be discontinued during treatment with tebentafuspe.

Drug, food and alcohol interactions: No formal interaction studies have been performed with tebentafuspe. Initiation of tebentafuspe therapy causes a transient release of cytokines that may suppress CYP450 enzymes . The highest risk of drug-drug interactions occurs within the first 24 hours of the first three doses of tebentafuspe in patients who are receiving concomitant CYP450 substrates , particularly those with a narrow therapeutic index. These patients should be monitored for toxicity (e.g., warfarin) or drug concentrations (e.g., cyclosporine). The dose of concomitant medications should be adjusted as necessary.

Dosage: Hospitalization is recommended for at least the first three infusions. The recommended dose is 20 micrograms on Day 1, 30 micrograms on Day 8, 68 micrograms on Day 15, and 68 micrograms once weekly thereafter. Treatment should continue as long as the patient derives clinical benefit and in the absence of unacceptable toxicities. To minimize the risk of hypotension associated with CRS, intravenous fluids should be administered prior to initiating the infusion, based on clinical assessment and the patient’s volume status. Tebentafuspe should be withheld or discontinued to manage adverse reactions. The recommended infusion period is 15 to 20 minutes.

Adverse reactions and laboratory test abnormalities: The most common adverse reactions in patients treated with tebentafusp were CRS (88%), rash (85%), pyrexia (79%), pruritus (72%), fatigue (66%), nausea (56%), chills (55%), abdominal pain (49%), edema (49%), hypo/hyperpigmentation (48%), hypotension (43%), dry skin (35%), headache (32%), and vomiting (34%). Adverse reactions led to permanent discontinuation in 4% of treated patients. The most common adverse reaction resulting in discontinuation of tebentafusp was CRS. Adverse reactions resulting in at least one dose interruption occurred in 26% of treated patients (weekly dosing) and resulted in a median of one missed dose. Adverse reactions requiring dose interruption in ≥2% of patients included fatigue (3%; Grade 1-3), pyrexia (2.7%; Grade 1-3), alanine aminotransferase increased (2.4%; Grade 1-4), aspartate aminotransferase increased (2.4%; Grade 1-3), abdominal pain (2.1%; Grade 1-3), and lipase increased (2.1%; Grade 1-3). Adverse reactions leading to dose modification in ≥1% of patients were CRS (1.9%; Grade 1-3) and hypotension (1.1%; Grade 2-4). Very common adverse reactions were: CRS, decreased appetite, hypomagnesemia, hyponatremia, hypocalcemia, hypokalemia, insomnia, headache, dizziness, paresthesia, tachycardia, hypotension, flushing, hypertension, cough, dyspnea, nausea, vomiting, diarrhea, abdominal pain, constipation, dyspepsia, rash, pruritus, dry skin, hypo/hyperpigmentation, erythema, arthralgia, back pain, myalgia, pain in extremity, pyrexia, fatigue, chills, edema, influenza-like illness, increased aspartate aminotransferase, increased alanine aminotransferase, increased serum bilirubin, increased lipase, anemia, decreased lymphocyte count, decreased serum phosphate, increased serum creatinine. See other adverse reactions in the full product leaflet.

Registration: 1.9132.0001/ Medison Pharma Brasil Produtos Farmacêuticos LTDA /CNPJ 48.682.588/0001-37/ Rua Nelson Pontes , 125 Bloco 5 e 6. Jardim Margarida. Vargem Grande Paulista/SP/Indústria Brasileira/ KIMMTRAK/SAC: 0800-633-4766. SALE UNDER PRESCRIPTION. USE RESTRICTED TO HEALTHCARE ESTABLISHMENTS. If symptoms persist, a physician should be consulted. Scientific documentation and additional information are available from Customer Service and the service department for prescribers and dispensers of medication. Product information approved by Anvisa is available at View Source

On March 18, 2025 Hoth Therapeutics, Inc. (NASDAQ: HOTH), a clinical-stage biopharmaceutical company, reported breakthrough preclinical findings demonstrating the efficacy of HT-KIT, a novel targeted therapy for gastrointestinal stromal tumors (GIST) (Press release, Hoth Therapeutics, MAR 18, 2025, View Source [SID1234651239]). The results highlight HT-KIT’s ability to significantly reduce tumor burden in humanized mouse models, disrupt KIT signaling pathways, and induce tumor cell death.

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"These exciting findings mark a significant milestone in the development of HT-KIT as a potential new therapeutic for patients with GIST," said Robb Knie, CEO of Hoth Therapeutics.

"By targeting KIT mutations, which are a major driver of GIST progression, HT-KIT has shown remarkable efficacy in preclinical models, demonstrating its potential as a transformative treatment option for this difficult-to-treat cancer."

Key Findings from the Preclinical Study:

Significant Reduction in KIT Expression – HT-KIT effectively reduced KIT receptor expression within 24 hours, with effects sustained for 72 hours.
Induction of Tumor Cell Death – HT-KIT triggered significant tumor cell death as early as 24 hours post-treatment, while the lower dose led to delayed but substantial cell death at 72 hours.
Decreased Tumor Cell Proliferation – HT-KIT treatment inhibited cell growth and proliferation in GIST-T1 cells, as confirmed by cell count reductions and decreased fluorescence intensity in proliferation assays.
Marked Tumor Volume Reduction in GIST Mouse Models – In a humanized xenograft model, HT-KIT treatment led to a significant reduction in tumor growth, with differences becoming statistically significant by day 8 and increasing over time.
Consistent Tumor Size Reduction – Excised tumors from HT-KIT-treated mice were smaller and lighter than those in the control group, reinforcing tumor volume measurements.
HT-KIT: A Potential New Treatment for GIST

GIST is a rare but aggressive cancer often driven by activating mutations in the KIT receptor. Current treatment options, including tyrosine kinase inhibitors (TKIs), can face resistance over time, leading to disease progression. Hoth Therapeutics’ HT-KIT offers a novel approach by effectively reducing KIT receptor expression, disrupting tumor survival pathways, and inhibiting tumor growth in vivo.

"These results provide compelling preclinical proof-of-concept for HT-KIT in GIST treatment," said Robb Knie. "By directly targeting the underlying genetic drivers of GIST, HT-KIT has the potential to overcome limitations of existing therapies and provide a new therapeutic strategy for patients with KIT-driven malignancies."

Next Steps in Development

Hoth Therapeutics is committed to advancing HT-KIT toward clinical evaluation. The company is currently conducting additional preclinical studies to further validate HT-KIT’s efficacy and safety profile, with plans to initiate regulatory discussions for first-in-human trials.

On March 18, 2025 IceCure Medical Ltd. (NASDAQ: ICCM) ("IceCure", "IceCure Medical" or the "Company"), developer of minimally-invasive cryoablation technology that destroys tumors by freezing as an alternative to surgical tumor removal, reported that it exhibited at the 19th Annual St. Gallen Breast Cancer Conference in Vienna, Austria from March 12 – 15, 2025 (Press release, IceCure Medical, MAR 18, 2025, View Source [SID1234651238]). Six studies of ProSense in breast cancer were accepted as peer-reviewed abstracts, presented as e-posters during the conference, and are being published in the scientific journal, The Breast after the conference.

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"ProSense received a high level of interest at this prestigious breast cancer conference in Europe where our cryoablation system is gaining increasing commercial traction driven by regulatory approval and a growing body of efficacy and safety data from third party studies," IceCure’s Chief Executive Officer, Eyal Shamir commented. "Among the benefits of these investigator-initiated studies is that each practitioner studies different patient populations in varied settings and circumstances, producing a breadth and depth of data that would simply not be available even in the largest of company-sponsored global studies. We are grateful to the investigators as we all aim toward the same goal of advancing women’s health through innovation and collaboration."

The following studies of ProSense were presented:

The Impact of Adjuvant Treatment After Cryoablation in Early-Stage, Low-Risk Breast Cancer: ICE3 Trial 5-Year Ipsilateral Breast Tumor Recurrence (IBTR)
Lead author Dr. Richard Fine, United States
Summary conclusion: The ICE3 trial suggests that cryoablation with ProSense, complemented by adjuvant therapy, is a viable alternative to surgical excision for selected patients with early-stage, low-risk breast cancer with recurrence rates comparable to those following lumpectomy. The addition of endocrine therapy alone yields similar results to de-escalation trials omitting radiation after surgery, such as CALGB 9343, PRIME II and LUMINA, without sacrificing survival. ICE3 enrolled patients aged ≥60 with unifocal, hormone receptor-positive, HER2-negative invasive ductal carcinomas measuring ≤1.5 cm. At 5 years, the overall IBTR rate was 3.61% (7 recurrences out of 194 patients). The 124 Patients who received only endocrine therapy with cryoablation had a 5-year IBTR rate of 3.22% (4 recurrences out of 124 patients).
Expanding the Use of Cryoablation on T1 Breast Cancers: An Initial Experience
Lead Author Dr. Ava Kwong, Hong Kong
Summary conclusion: This study evaluated expanding ICE3’s inclusion criteria to T1 breast cancer of all IHC subtypes (i.e. luminal, HER2-enriched and triple negative breast cancers). The study found that cryoablation with ProSense can completely ablate T1 breast cancer, including triple negative breast cancer, and younger age range. 35 patients were recruited with a median age of 64 and biopsy proven invasive or in-situ breast cancer of ≤2cm.
The Treatment of Breast Cancer with Percutaneous Thermal Ablation: Results of the THERMAC trial
Lead Author by Dr. Linda Risks, Netherlands
Summary conclusion: Percutaneous thermal ablation has the potential to replace surgical treatment and improve the health-related quality of life of patients with small breast cancers, without jeopardizing current treatment effectiveness or safety. 41 postmenopausal patients, with cT1N0 ER+/Her2- breast cancer, were treated. The study compared radiofrequency ablation ("RFA"), microwave ablation ("MWA") and cryoablation ("CA"). The RFA arm was prematurely terminated. Complete ablation was reached in 72% (95% CI, 49% to 88%) in the MWA arm and in 94% (95% CI, 74% to 0.99) in the cryoablation arm. Adverse events occurred in 44% (95% CI, 25% to 66%) of the patients in the MWA arm and 0% (95% CI, 0% to 18%) of in the cryoablation arm. Of the three thermal ablation methods evaluated, cryoablation with ProSense was the only thermal ablative technique that met the minimum requirements and will therefore be selected for a Phase III trial.
Percutaneous Ultrasound-Guided Cryoablation for the Treatment of Breast Cancer in Non-Surgical Patients
Lead Author Dr. Lucía Graña López, Spain
Summary conclusion: Breast cancer patients who chose not to have surgery or were considered inoperable were treated with ProSense cryoablation, which was found to be a safe and well-tolerated outpatient procedure. The study outcomes suggest that cryoablation is effective and could be an alternative to surgery for the management of luminal breast cancer up to 2.5 cm. 60 breast cancer tumors were treated (median size 21 mm, range 6-46 mm) in 54 patients, median age 87, with ProSense cryoablation. Complete tumoral necrosis was achieved in 49 tumors (81.7%). Axillary progression occurred in two cases of triple-negative breast cancer. Two patients experienced recurrence in different locations. Percutaneous cryoablation was successful in 100% of luminal cancers up to 2.5 cm. The procedure was well tolerated, with no major complications observed.
Cryoablation of Breast Cancer: The Challenge of an Innovative Non-Surgical Treatment for Selected Patients
Lead Author Dr. Francesca Magnoni, Italy
Summary conclusion: The European Institute of Oncology in Italy is evaluating ProSense cryoablation, with the prospective observational study "Percutaneous Cryoablation of Low-risk Early Breast Cancer (PRECICE)". The trial has just started and will enroll 233 patients over the age of 50 years with unifocal, small (≤1.5 cm), clinically node-negative, luminal A and B breast cancer. To date, 11 patients eligible for cryoablation have been enrolled. Cryoablation, performed on an outpatient basis under local anesthesia, completely replaces both breast and axillary surgery, in accordance with recent evidence.
Cryotherapy as a Surgical De-Escalation Strategy in Breast Cancer: A Comprehensive Review of Techniques, Complications, and Oncological Outcomes
Lead author Dr. Kai Lin Lee, Singapore
Summary conclusion: After a review of 276 papers, this study concluded that cryotherapy with systems including ProSense could potentially be a step forward in surgical de-escalation of breast cancer, particularly for elderly patients with early-stage breast cancer or those who are at high risk and might benefit from a less aggressive treatment strategy.

About ProSense
The ProSense Cryoablation System is a minimally invasive cryosurgical tool that provides the option to destroy tumors by freezing them. The system uniquely harnesses the power of liquid nitrogen to create large lethal zones for maximum efficacy in tumor destruction in benign and cancerous lesions, including breast, kidney, lung, and liver.

ProSense enhances patient and provider value by accelerating recovery, reducing pain, surgical risks, and complications. With its easy, transportable design and liquid nitrogen utilization, ProSense opens that door to fast and convenient office-based procedures for breast tumors.

On March 18, 2024 OmniAb, Inc. (NASDAQ: OABI) reported financial results for the three and 12 months ended December 31, 2024, and provided operating and partner program updates (Press release, OmniAb, MAR 18, 2025, View Source [SID1234651234]).

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"2024 was a remarkable year featuring double-digit percentage growth in the number of both active partners and programs. Clinical-stage programs advanced well, and we launched new technologies and enhancements that strengthened our platform and expanded our reach," said Matt Foehr, Chief Executive Officer of OmniAb. "We exceeded our internal goals for key metrics and continued to build momentum while optimizing the scalability of our business. We believe our commitment to innovation is a significant competitive advantage and creates exciting opportunities for future growth. We remain steadfast in our strategic direction and are excited about the prospects that lie ahead."

Fourth Quarter 2024 Financial Results

Revenue for the fourth quarter of 2024 was $10.8 million, compared with $4.8 million for the same period in 2023, with the increase primarily due to higher license and milestone revenue partially offset by lower service and royalty revenue.

Research and development expense was $13.3 million for the fourth quarter of 2024, compared with $14.8 million for the same period in 2023, with the decrease primarily due to lower stock-based compensation expense and outside expenses associated with third-party services. General and administrative expense was $7.4 million for the fourth quarter of 2024, compared with $7.9 million for the same period in 2023, with the decrease primarily due to lower stock-based compensation expense and lower external marketing and legal expenses.

Net loss for the fourth quarter of 2024 was $13.1 million, or $0.12 per share, compared with a net loss of $14.1 million, or $0.14 per share, for the same period in 2023.

Full Year 2024 Financial Results

Revenue for 2024 was $26.4 million, compared with $34.2 million for 2023, with the decrease primarily due to the recognition of a $10.0 million TECVAYLI milestone in 2023. Royalty revenue decreased primarily due to lower net product sales by partners.

Research and development expense for 2024 was $55.1 million, compared with $56.5 million for 2023, with the decrease primarily due to lower personnel costs and external expenses. General and administrative expense for 2024 was $30.7 million, compared with $33.3 million for 2023, with the decrease primarily due to non-recurring consulting and other outside service expenses incurred in 2023 related to our spin-out as a public company and lower legal and stock-based compensation expense.

Net loss for 2024 was $62.0 million, or $0.61 per share, compared with a net loss of $50.6 million, or $0.51 per share, for 2023.

As of December 31, 2024, OmniAb had cash, cash equivalents and short-term investments of $59.4 million.

2025 Financial Guidance

OmniAb today introduced 2025 financial guidance. OmniAb expects 2025 revenue to be in the range of $20 million to $25 million and operating expense to be in the range of $90 million to $95 million. In addition, OmniAb expects 2025 cash use to be lower than its cash use in 2024. Cash use in 2024 was $38.9 million, excluding the 2024 ATM issuance. The 2025 full year effective tax rate is expected to be approximately 0%.

Fourth Quarter 2024 and Recent Business Highlights

As of December 31, 2024, OmniAb had 91 active partners and 363 active programs, including 32 OmniAb-derived programs in clinical development or being commercialized. The Company signed 10 new license agreements in 2024, including two in the fourth quarter with Incyte Corporation and Photinia Biosciences. In addition, five new OmniAb-derived antibodies entered the clinic in 2024.

In December 2024, OmniAb launched OmniHub , a unified interface designed to provide partners with secure access to datasets to visualize their discovery campaign data with a variety of custom tools. This bioinformatics portal is designed to enable scalable and secure data transfer, advanced visualization and computational tool access.

Fourth quarter 2024 and recent partner highlights include the following:

IMVT-1402

Immunovant announced that its lead asset, IMVT-1402, is rapidly progressing with six Investigational New Drug (IND) applications now cleared and pivotal Phase 2b studies in Graves’ disease (GD) and difficult-to-treat rheumatoid arthritis now enrolling.
Immunovant is on track to initiate potentially registrational programs for three additional indications for IMVT-1402 by March 31, 2025. In addition, Immunovant anticipates initiating clinical trials evaluating IMVT-1402 in a total of 10 indications by March 31, 2026.
Batoclimab

Immunovant reported that the top-line results of the batoclimab trial in myasthenia gravis and the initial results from period 1 of the batoclimab trial in chronic inflammatory demyelinating polyneuropathy are expected by March 31, 2025.
Immunovant also plans to announce additional data from the batoclimab proof-of-concept study in GD, including six-month, treatment-free remission data in the summer 2025.
Additionally, Immunovant reported that top-line results from the pivotal program of batoclimab for the treatment of thyroid eye disease (TED), also known as Graves’ ophthalmopathy, are expected in the second half of 2025.
HanAll Biopharma announced that batoclimab has received Orphan Drug Designation from Japan’s Ministry of Health, Labor, and Welfare for active TED.
Acasunlimab

Genmab announced that a Phase 3 trial with acasunlimab as a second-line therapy in non-small cell lung cancer (NSCLC) is now enrolling patients and that they expect to provide an additional Phase 2 data update in NSCLC in 2025.
Zimberelimab

Arcus Biosciences expects to present overall survival (OS) data from the Phase 2 EDGE-Gastric study, which is evaluating domvanalimab plus zimberelimab and chemotherapy in upper gastrointestinal adenocarcinomas, in the fall of 2025.
Arcus Biosciences also expects to initiate three new expansion cohorts within the Phase 1/1b ARC-20 study in the first quarter of 2025, including one cohort for casdatifan plus zimberelimab in all-comer first-line clear cell renal cell carcinoma.
Sugemalimab

CStone announced the publication of the Phase 3 GEMSTONE-303 study results for sugemalimab in patients with unresectable locally advanced or metastatic G/GEJ adenocarcinoma in theJournal of the American Medical Association. Results showed that in patients with PD-L1 combined positive score ≥5, sugemalimab significantly improved both OS and progression-free survival (PFS) compared with the control group. Median OS was 15.6 months versus 12.6 months, and median PFS was 7.6 months versus 6.1 months.
CStone announced that they have entered into a strategic partnership with Pharmalink Store for commercialization of sugemalimab in the Middle East, North Africa and South Africa. Additionally, CStone has partnered with SteinCares to market sugemalimab in Latin America.
Mipletamig

Aptevo Therapeutics announced that 100% of patients in Cohort 1 of the mipletamig RAINIER Phase 1b/2 dose-optimization trial for frontline acute myeloid leukemia achieved remission within 30 days of treatment. Trial enrollment is ongoing. Aptevo also anticipates providing multiple data readouts in 2025 and presenting results at the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in the fourth quarter of 2025.
RNDO-564

Rondo Therapeutics presented data from preclinical studies of RNDO-564, a novel CD28 x Nectin-4 costimulatory bispecific antibody for advanced bladder cancer, at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary Cancers Symposium. RNDO-564 demonstrated robust anti-tumor activityin vivoandin vitro, including in an antibody-drug-conjugate-resistant bladder cancer model. Based on promising preclinical findings, Rondo is advancing RNDO-564 through IND-enabling studies and expects to initiate a Phase 1/b trial in relapsed/refractory, locally advanced/metastatic bladder cancer by year-end 2025.
Conference Call and Webcast

OmniAb management will host a conference call with accompanying slides today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 549-8228 using the conference ID 84579. Slides, as well as the live and replay webcast of the call, are available at View Source

On March 18, 2025 Aptevo Therapeutics (NASDAQ:APVO), a leader in the development of novel bispecific antibodies for cancer treatment, based on its proprietary ADAPTIR and ADAPTIR-FLEX platform technologies reported an overview of solid tumor anti-cancer compound APVO603, currently in preclinical development for the treatment of solid tumors (Press release, Aptevo Therapeutics, MAR 18, 2025, View Source [SID1234651233]). APVO603 is a novel bispecific antibody designed to enhance anti-tumor immune responses through dual targeting of 4-1BB (CD137) and OX40 (CD134), two key co-stimulatory receptors involved in T cell and NK cell activation.

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APVO603 is part of Aptevo’s solid tumor anti-cancer pipeline and represents a significant step forward in leveraging the company’s proprietary ADAPTIR platform to develop safer and more effective immuno-oncology therapies.

Targeted Immune Activation for Solid Tumors
APVO603 is designed to activate the costimulatory 4-1BB and OX40 pathways in the tumor microenvironment, leading to enhanced T cell expansion, survival, and cytotoxic function while avoiding systemic toxicity. This targeted immune activation could overcome immune suppression in solid tumors, a major challenge in cancer immunotherapy.

Preclinical studies have shown that APVO603:

Enhances T cell and NK cell proliferation and tumor lysis, while maintaining a favorable safety profile and inhibiting immune exhaustion

Induces a robust anti-tumor response in preclinical tumor models, suggesting strong therapeutic potential

"We are excited about the potential of APVO603 to enhance the potency of T cells and NK cells while addressing the limitations of current therapies for solid tumors," said Michelle Nelson, PhD, Director of Immunobiology at Aptevo. "Our preclinical findings support APVO603’s ability to deliver 4-1BB and OX40 synergistically resulting in targeted immune stimulation with reduced toxicity risks, differentiating it from existing immunotherapies."

Aptevo’s Advancing Immuno-Oncology Pipeline

APVO603 is one of three promising preclinical candidates in Aptevo’s bispecific antibody pipeline, alongside APVO711, a bispecific + checkpoint inhibitor, and APVO442, a T-cell engager targeting prostate cancer. These programs build on Aptevo’s expertise in bispecific antibody engineering and reinforce the company’s commitment to developing novel immunotherapies that improve outcomes for cancer patients.

Clinical Programs
Lead Candidate Mipletamig for Acute Myeloid Leukemia (AML): Mipletamig, Aptevo’s lead bispecific antibody, is currently in a frontline Phase 1b/2 combination trial, RAINIER, following positive results from earlier studies.

RAINIER results reported to date include:

100% of patients in Cohort 1 of the trial achieved remission within 30 days

One patient experienced complete remission with minimal residual disease (MRD)-negative status (100% elimination of cancer cells)

Favorable safety profile consistent with prior trials, showing no incidences of cytokine release syndrome (CRS) in the current trial to date, a common and often dose limiting side effect seen in similar therapies

ALG.APV-527: ALG.APV-527 is being evaluated in a Phase 1 trial for solid tumors likely to express the tumor antigen 5T4. Tumor types treated to date include breast, colon, pancreatic and non-small cell lung cancer. Positive preliminary data from the study were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in September and at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) conference in 2024. These results showed that ALG.APV-527 demonstrated positive safety and tolerability across all cohorts, ten of 17 evaluable patients (59%) achieved stable disease (SD), and biomarker analysis confirmed immune activation in the tumor microenvironment.

This drug has the potential to advance treatment in hard-to-treat solid tumors, demonstrating the versatility of Aptevo’s technology across a wide range of cancer types.