On December 10, 2025 NEC Bio Therapeutics reported results from the Phase I basket clinical trial of an orally administered cancer vaccine, NECVAX-NEO1, used in combination with checkpoint inhibitors (CPIs) for treating patients with solid tumors. The findings are being presented in a poster at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress in London, United Kingdom, from December 10 to 12, 2025.

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NECVAX-NEO1 is a personalized bacteria-based oral DNA therapeutic vaccine, developed using AI prediction of the most immunogenic patient-specific neoepitopes. This vaccine is designed to activate a patient’s immune system, prompting a T-cell response that can precisely target and eliminate tumor cells based on an individual’s unique neoantigens.

In the phase I study, 6 patients with melanoma, renal cell cancer, or head and neck cancer, who have been on CPI treatment for at least three months, were treated with NECVAX-NEO1. The safety run-in phase showed no treatment-related toxicities, allowing a dose increase. Clinically, 83% of the patients achieved stable disease at the end of 24 weeks of treatment, which was followed by a 12 week follow-up period. In all patients immunogenic neoepitopes used in the vaccine, were detected, as demonstrated by ELISPOT analysis.

Regarding the results of the clinical study, Dr. Heinz Lubenau, CEO of NEC Bio Therapeutics, commented, "Our clinical Phase I data are demonstrating promising immune responses in treated cancer patients. The translational biomarker data are in line with the clinical data so that we are presenting a consistent data set. The progress is encouraging as we advance two additional clinical studies in 3 European countries in both early- and later-stage cancer settings. We look forward to further evaluating NECVAX-NEO1 as a potential treatment option for cancer patients with hard-to-treat tumors."

Motoo Nishihara, Corporate Executive Vice President and CTO of NEC Corporation, further commented, "We are proud to present the progress of the NECVAX-NEO1 trial, which demonstrates safety as well as signs of immunogenicity and early efficacy. NECVAX-NEO1 is the first oral cancer vaccine asset to be clinically developed by NEC. The results of this trial are a testament to our proprietary AI predictive software that supports immunological and clinical readouts. This development aligns closely with NEC’s broader mission to deliver global healthcare solutions using state-of-the-art technologies developed in-house."

Details of the poster are below:

Poster title: NECVAX-NEO1, a bacteria-based personalized neoepitope vaccine combined with PD-1/PD-L1 checkpoint inhibition in a phase I, open-label, multicenter study: safety, immunogenicity and early efficacy signals. NCT05354323

Authors: D. Vaitiekus, E. Juozaityte, L. Puzauskienė, S. Tulyte-Kirzova, L. Gatijatullin, M. Platten, I. Poschke, I.Hülsmeyer, A. Kuhn, A. Aranguren, H. Lubenau, R. Stratford, T. Clancy, H. Fontenelle, B. Simovski, Y. Yamashita, C. Chaput, A. Meiser, V. Urbonas

Poster Number: 258P

(Press release, NEC, DEC 10, 2025, View Source [SID1234661347])

On December 10, 2025 Pfizer Inc. (NYSE: PFE) reported detailed results from the Phase 3 HER2CLIMB-05 trial of the tyrosine kinase inhibitor TUKYSA (tucatinib) as part of an investigational first-line maintenance treatment combination, following chemotherapy-based induction, in patients with human epidermal growth factor receptor 2-positive (HER2+) metastatic breast cancer (MBC). The primary endpoint analysis showed a 35.9% reduction in the risk of disease progression or death among patients treated with TUKYSA, trastuzumab, and pertuzumab compared to those treated with placebo, trastuzumab, and pertuzumab, as assessed by the investigator (hazard ratio [HR] of 0.641, 95% confidence interval (CI): 0.514-0.799; 2-sided p<0.0001). These findings were published today in the Journal of Clinical Oncology, shared in an oral presentation at the 48th San Antonio Breast Cancer Symposium (SABCS), and highlighted in the SABCS official press program.

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In HER2CLIMB-05, the median progression-free survival (PFS) was 24.9 months (95% CI: 21.3-not reached) in the TUKYSA arm and 16.3 months (95% CI:12.6-18.7) in the placebo arm, representing an extension in median PFS of 8.6 months. A PFS benefit was observed across all prespecified patient subgroups, including de novo or recurrent diagnosis, hormone receptor (HR)-positive or HR-negative disease, and with or without the presence or history of brain metastases at baseline. The key secondary endpoint of overall survival was not mature at the time of the analysis (20% of the required events have occurred to date) but showed a numerical trend for improvement with TUKYSA.

"Most patients with HER2-positive metastatic breast cancer face disease progression within two years of starting first-line treatment, often requiring a transition to chemotherapy," said Erika Hamilton, M.D., principal investigator of HER2CLIMB-05 and Director of Breast Cancer Research for Sarah Cannon Research Institute (SCRI). "These results demonstrate that adding tucatinib to first-line maintenance therapy extends the time patients live without their disease progressing, while maintaining a manageable safety profile, suggesting a promising new potential approach that could advance the current standard of care for HER2-positive disease."

TUKYSA in combination with trastuzumab and pertuzumab demonstrated a safety profile generally consistent with the established safety profiles of each individual therapy, except for a higher rate of asymptomatic Grade ≥3 liver transaminases, which were typically manageable and reversible with TUKYSA dose modifications and/or discontinuations. The most common adverse events observed in the TUKYSA combination arm were diarrhea, hepatic events, and nausea.

"TUKYSA has become a trusted standard of care for patients with later-line HER2-positive metastatic breast cancer, and the results from HER2CLIMB-05 support its potential use as part of a chemotherapy-free, front-line maintenance strategy," said Jeff Legos, Chief Oncology Officer, Pfizer. "At Pfizer, we are committed to advancing treatment options that meaningfully improve the lives of people with metastatic breast cancer, and we are proud to share these promising results for patients and their families."

TUKYSA is not currently approved for first-line treatment. The results from HER2CLIMB-05 will be discussed with regulatory authorities. Since its initial approval in 2020, TUKYSA in combination with trastuzumab and capecitabine has become a standard of care for HER2+ MBC patients in the third-line setting. TUKYSA is currently approved in more than 50 countries; in the United States, TUKYSA is approved for use in combination with trastuzumab and capecitabine for adult patients with advanced unresectable or metastatic HER2+ breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting.

About HER2-Positive Metastatic Breast Cancer (MBC)
HER2 is overexpressed in up to 15-20% of breast cancers and is associated with poor prognosis, with an estimated five-year survival rate for HER2+ MBC of 41-47%, depending on HR status.1-3 First-line standard-of-care maintenance treatment has remained unchanged since 2012, and the majority of patients with HER2+ MBC face disease progression within two years of initiating therapy.4 Until recently, there have been limited advancements for these patients.

About the HER2CLIMB-05 Trial
HER2CLIMB-05 is a randomized, double blind, placebo-controlled, pivotal Phase 3 study evaluating the efficacy and safety of TUKYSA (tucatinib) compared to placebo, both in combination with trastuzumab and pertuzumab, as maintenance therapy for patients with HER2+ MBC following induction therapy in the first-line setting.

Trial participants who completed induction therapy of trastuzumab, pertuzumab, and a taxane, with no evidence of progression were randomized to receive TUKYSA in combination with trastuzumab plus pertuzumab (n=326), or placebo in combination with trastuzumab plus pertuzumab (n=328). The primary endpoint is progression-free survival (PFS) as assessed by the investigator. Key secondary endpoints include overall survival.

About TUKYSA (tucatinib)
TUKYSA (tucatinib) is an orally administered tyrosine kinase inhibitor of HER2. TUKYSA is approved in combination with trastuzumab and capecitabine to treat adults with HER2-positive advanced unresectable or metastatic breast cancer, including patients with brain metastases who have received one or more prior anti-HER2 breast cancer treatments in the metastatic setting.

The full U.S. Prescribing Information for TUKYSA can be found here.

IMPORTANT TUKYSA (tucatinib) SAFETY INFORMATION FROM THE U.S. PRESCRIBING INFORMATION

Warning and Precautions:

Diarrhea: TUKYSA can cause severe diarrhea including dehydration, hypotension, acute kidney injury, and death. If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Based on the severity of the diarrhea, interrupt dose, then dose reduce or permanently discontinue TUKYSA.
In HER2CLIMB, 81% of patients who received TUKYSA experienced diarrhea, including 0.5% with Grade 4 diarrhea and 12% with Grade 3 diarrhea. The median time to onset of the first episode of diarrhea was 12 days and the median time to resolution was 8 days. Diarrhea led to dose reductions of TUKYSA in 6% of patients and discontinuation of TUKYSA in 1% of patients. Prophylactic use of antidiarrheal treatment was not required on HER2CLIMB.
Hepatotoxicity: TUKYSA can cause severe hepatotoxicity. Monitor ALT, AST, and bilirubin prior to starting TUKYSA, every 3 weeks during treatment, and as clinically indicated. Based on the severity of hepatotoxicity, interrupt dose, then dose reduce or permanently discontinue TUKYSA.
In HER2CLIMB, 8% of patients who received TUKYSA had an ALT increase > 5 × ULN, 6% had an AST increase > 5 × ULN, and 1.5% had a bilirubin increase > 3 × ULN (Grade ≥3). Hepatotoxicity led to dose reduction of TUKYSA in 8% of patients and discontinuation of TUKYSA in 1.5% of patients.
Embryo-fetal Toxicity: TUKYSA can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with TUKYSA and for 1 week after the last dose.
Adverse Reactions:
In HER2CLIMB, serious adverse reactions occurred in 26% of patients who received TUKYSA. Serious adverse reactions in ≥ 2% of patients who received TUKYSA were diarrhea (4%), vomiting (2.5%), nausea (2%), abdominal pain (2%), and seizure (2%). Fatal adverse reactions occurred in 2% of patients who received TUKYSA including sudden death, sepsis, dehydration, and cardiogenic shock. Adverse reactions leading to treatment discontinuation occurred in 6% of patients who received TUKYSA. Adverse reactions leading to treatment discontinuation of TUKYSA in ≥1% of patients were hepatotoxicity (1.5%) and diarrhea (1%). Adverse reactions leading to dose reduction occurred in 21% of patients who received TUKYSA. Adverse reactions leading to dose reduction of TUKYSA in ≥2% of patients were hepatotoxicity (8%) and diarrhea (6%). The most common adverse reactions in patients who received TUKYSA (≥20%) were diarrhea, palmar-plantar erythrodysesthesia, nausea, hepatotoxicity, vomiting, stomatitis, decreased appetite, anemia, and rash.

Laboratory Abnormalities:
In HER2CLIMB, Grade ≥3 laboratory abnormalities reported in ≥5% of patients who received TUKYSA were decreased phosphate, increased ALT, decreased potassium, and increased AST. The mean increase in serum creatinine was 32% within the first 21 days of treatment with TUKYSA. The serum creatinine increases persisted throughout treatment and were reversible upon treatment completion. Consider alternative markers of renal function if persistent elevations in serum creatinine are observed.

(Press release, Pfizer, DEC 10, 2025, View Source [SID1234661363])

On December 10, 2025 NovaBridge Biosciences (Nasdaq: NBP) (NovaBridge or the Company) a global biotechnology platform company committed to accelerating access to innovative medicines, reported the presentation of new data from the expanded 3mg/kg every 6 week (Q6W) Phase 1 dosing study for ragistomig, a bispecific 4-1BB X PD-L1 antibody, in a poster at the European Society for Medical Oncology – Immuno-Oncology Congress 2025 (ESMO-IO 2025) by co-developer ABL Bio. The poster (Poster 257P), presented by Gerald Falchook, MD, Director of the Sarah Cannon Research Institute (SCRI) at HealthONE Denver, showed the new Q6W schedule demonstrated consistent monotherapy antitumor activity with improved immunological endpoints and tolerability.

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"We are very pleased by the impressive ragistomig Phase 1 dose expansion data presented today at ESMO (Free ESMO Whitepaper)-IO, indicating that the prior Q2W schedule demonstrated meaningful clinical activity, and that the new Q6W dosing interval provides comparable efficacy with a more favorable safety profile. The study achieved its objective by extending the therapeutic window and supports the advancement of ragistomig into combination studies. We are particularly encouraged by the improved safety profile, with only 5% of patients experiencing ≥ Grade 3 liver function elevation, while maintaining comparable immune-mediated activity. These observations, combined with comparable confirmed responses and durable immune engagement, underpins our optimism that ragistomig has the potential to make an important contribution to more effective treatment outcomes for patients with relapsed/refractory solid tumor cancers," said Phillip Dennis, MD, PhD, Chief Medical Officer of NovaBridge.

"Ragistomig was designed to overcome resistance in patients who relapsed after treatment with checkpoint inhibitors, a multi-billion dollar pillar of cancer treatment. The ragistomig data presented at ESMO (Free ESMO Whitepaper)-IO build on the promising results presented at ASCO (Free ASCO Whitepaper) 2024. This program aligns well with our strategy to partner with innovators around the world to advance transformative and potentially breakthrough therapies. We are enthusiastic to collaborate with our partner, ABL Bio, to initiate combination studies," said Sean Fu, PhD, Chief Executive Officer of NovaBridge.

ESMO-IO Meeting information:

•
Title: Phase 1 Clinical Trial of Ragistomig (ABL503/TJ-L14B: PD-L1 × 4-1BB bispecific antibody) Q6W Dosing Balances Favorable Safety and Sustained Efficacy Through Extended Immunologic Memory and Reinvigoration of CD8+ T Cells
•
Abstract #688/Poster 257P
•
Date and Time: Wednesday, December 10th at 5:15 PM GMT
A copy of the poster will be available here after the session. To review an overview of the Phase 1 dose escalation data, click here.

Ragistomig Phase 1 Monotherapy Q6W Data (per October 22, 2025 data cut-off):

•
Ragistomig demonstrated comparable anti-tumor efficacy for the Q6W dose schedule compared to the Q2W regimen (58.8% disease control rate (DCR) at Q6W compared to 64.3% at Q2W)
•
Ragistomig exhibited a favorable and improved safety profile, with 1/20 Grade ≥3 liver function test elevations (LFT), no treatment discontinuations due to treatment emergent adverse events (TEAEs) and no reported cytokine release syndrome (CRS)
•
Overall immune cell activity was consistent between the Q6W and Q2W dosing. Immune cell pharmacodynamics with the Q6W dosing demonstrated expansion of effector memory and CD8+ T cells, with attenuated Treg expansion, indicating durable immune engagement
•
The data suggest evaluation of the ongoing 5 mg/kg Q6W dosing cohort and evaluation of ragistomig in future combination studies
Patient Characteristics:

•
20 heavily pre-treated subjects received 3 mg/kg Q6W ragistomig. In this group, 100% were previously treated with immuno-oncology therapies and 70% had previously received ≥3 lines of systemic treatment and exhausted all available standard treatment options
•
Dosing is underway in 10 patients who are receiving 5 mg/kg Q6W ragistomig
Efficacy Results, based on 17 evaluable patients receiving 3 mg/kg Q6W v. 14 evaluable patients with 3 mg/kg Q2W

ABL503 monotherapy
efficacy profiile

3 mgkg Q6W
(N=17)

3 mg/kg Q2W
(N=14)

Objective Response Rate, n (%)

2 (11.8%)

4 (28.6%)

Disease Control Rate, n (%)

10 (58.8%)

9 (64.3%)

Complete response

0 (0%)

1 (7.1%)

Partial response

2 (11.8%)

3 (21.4%)

Stable disease

8 (47.1%)

5 (35.7%)

Progressive disease

7 (41.2%)

5 (35.7%)

Safety Data, based on 20 evaluable patients receiving 3 mg/kg Q6W v. 15 evaluable patients with 3 mg/kg Q2W

o
An improved safety profile was observed with the 3 mg/kg Q6W regimen
o
The 3 mg/kg Q6W regimen was identified as an optimal potential regimen for combination strategies
o
1/20 subjects (5%) experienced ≥Grade 3 LFT elevation at Q6W dosing v. 40% at Q2W dosing
o
TEAEs ≥Grade 3 were 50% at the Q6W (10/20) v. 66.7% (10/15) at 3 mg/kg Q2W. In the Q6W dose, these TEAEs, including LFT elevations, decreased platelet count, anemia and decreased neutrophil count, were recovered within 3-14 days (with or without treatment interventions), with no discontinuations
o
No cases of CRS were reported with either dosing schedule
Immunology Data:

o
Effector and memory CD8+ T cell subsets were increased, with comparable fold-changes between dosing groups, suggesting that immune mediated pharmacodynamic activity may contribute to efficacy
o
CTLA+ Treg frequencies remained near baseline in the Q6W group, suggesting a more favorable effector-to-regulatory balance
About Ragistomig

Ragistomig (also known as ABL503) is a differentiated novel bispecific that integrates a single-chain, Fc-silent PD-L1 segment as a tumor engager and 4-1BB segment as a conditional T cell activator. It was developed using ABL Bio’s "Grabody-T" bispecific antibody platform technology to overcome resistance to PD-(L)1 inhibition and stimulate 4-1BB activation only in the presence of PD-L1 expressing tumor cells, to minimize the risk of off-tumor toxicity. Preclinical studies demonstrated that the bispecific antibody showed better anti-tumor activity than its single-agent components. A Phase 1 dose expansion study (NCT04762641) is currently being conducted in the U.S. and South Korea. The study was designed with a primary endpoint of defining the dose-limiting toxicity and adverse event profile of ragistomig, as well as to observe the objective response rate, pharmacokinetic and immunogenicity profiles and other secondary endpoints.

Ragistomig (also known as ABL503) is being jointly developed with ABL Bio

ASCO 2024: the 2024 American Society for Clinical Oncology Annual Meeting; Q6W: every six weeks

(Press release, NovaBridge Biosciences, DEC 10, 2025, View Source [SID1234661348])

On December 10, 2025 NeoGenomics, Inc. (NASDAQ: NEO), a leading provider of oncology diagnostic solutions that enable precision medicine, reported that data utilizing its RaDaR 1.0 assay for the detection of molecular residual disease (MRD) will be presented at the 2025 San Antonio Breast Cancer Symposium (SABCS), taking place Dec. 9–12, 2025, at the Henry B. Gonzalez Convention Center in San Antonio, Texas.

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NeoGenomics will present new data from the SURVIVE HERoes Phase III trial and the CLEVER study, both of which used RaDaR 1.0 circulating tumor DNA (ctDNA) testing to evaluate molecular residual disease and recurrence risk. These findings reinforce the growing role of tumor-informed ctDNA approaches in early breast cancer research and recurrence monitoring.

Presentation details

The first study, titled "5-year outcomes and ctDNA findings in the CLEVER trial targeting disseminated dormant tumor cells," investigates long-term recurrence biology in patients with high-risk breast cancer and shows that positive ctDNA frequently precedes clinical recurrence. Five-year follow-up data demonstrate that RaDaR-detected ctDNA was present in most patients with disseminated tumor cells, often months before relapse. These findings reinforce the potential role that sensitive ctDNA testing can play in monitoring molecular residual disease during periods of ongoing long-term risk. Investigators from the University of Pennsylvania will present on Thursday, Dec. 11, 2025, 7:00 AM–8:30 AM CST. [PD5-02]

The SURVIVE Phase III randomized case-control trial, presented as "Reevaluating Follow-Up in Early Breast Cancer, guided by Liquid Biopsy: the SURVIVE Study (NCT05658172)," investigates whether the use of the RaDaR assay in liquid-biopsy guided follow-up may enable earlier detection of recurrence and improve overall survival. Investigators from the University Hospital Ulm, Germany, presenting on behalf of recruiting centers across Germany, will share the current status on Friday, Dec. 12, 2025, 12:30 PM–2:00 PM CST. [PS5-08-13]

The SURVIVE HERoes Phase III trial, presented as "The SURVIVE HERoes study NCT06643585: Targeting molecular relapse in breast cancer," is an ongoing therapeutic intervention arm of the SURVIVE trial, which evaluates an emerging strategy for treating patients at the point of molecular relapse, when ctDNA is detectable when using the RaDaR assay despite no radiographic evidence of disease. The study examines whether earlier intervention in HER2-positive or HER2-low early breast cancer can improve long-term outcomes. Positive study results could help establish a new, molecularly guided, individualized surveillance and treatment approach. Investigators from the University Hospital Ulm will present on Friday, Dec.12, 2025, 12:30 PM–2:00 PM CST. [PS5-07-28]

"The findings presented at this year’s SABCS conference demonstrate how RaDaR-detected ctDNA can provide invaluable and actionable information to care teams as they monitor patients following their initial breast care treatment," said Tony Zook, Chief Executive Officer. "These studies represent an important step in building the evidence needed to integrate MRD insights into the routine oncology care that community oncologists and their patients deserve."

(Press release, NeoGenomics Laboratories, DEC 10, 2025, View Source [SID1234661364])

On December 9, 2025 Novartis reported positive results from VAYHIT2, a Phase III trial evaluating ianalumab plus eltrombopag in patients with primary immune thrombocytopenia (ITP) previously treated with corticosteroids1-3. Ianalumab (9 mg/kg) plus eltrombopag extended ITP disease control by 45%, based on the primary endpoint of time to treatment failure (TTF), which assesses how long patients maintain safe platelet levels during and after the treatment period1,2. The median time to treatment failure for patients receiving ianalumab plus eltrombopag was 2.8 times longer than those on placebo plus eltrombopag (13.0 months vs. 4.7 months)1,2.

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Detailed data will be presented during the Late-Breaking Abstract Session at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition (ASH) (Free ASH Whitepaper) and simultaneously published in The New England Journal of Medicine1,2.

"Treatments for ITP have historically focused on raising platelet counts, often requiring chronic therapy to control ITP. This means many patients remain on treatment long-term, facing persistent disease burden and symptoms like fatigue," said Hanny Al-Samkari, M.D., Peggy S. Blitz Endowed Chair in Hematology/Oncology, Mass General Brigham, and Associate Professor of Medicine, Harvard Medical School. "The VAYHIT2 trial results are encouraging, as they demonstrated improved disease control even while patients spend time off treatment, pointing toward possible progress for people living with ITP."

Patients receiving ianalumab (9 mg/kg) plus eltrombopag also achieved a significantly higher rate of sustained platelet count improvement at six months versus placebo plus eltrombopag (62% vs. 39%), meeting the key secondary endpoint1,2. Fatigue improvement, as measured by PROMIS Fatigue, showed a mean reduction of 7.7 points with ianalumab plus eltrombopag versus 3.6 points with placebo plus eltrombopag1,2.

"B cells drive the autoimmune response that leads to platelet destruction and increased bleeding risk in ITP. The novel dual mechanism of action of ianalumab aims to deplete B cells while blocking their survival signals," said Mark Rutstein, M.D., Global Head, Oncology Development, Novartis. "Guided by our decades-long experience advancing ITP care, the VAYHIT2 findings underscore the potential of ianalumab to deliver durable control with a short course of four once-monthly doses, offering patients the possibility of achieving disease stability without ongoing treatment."

Two doses of ianalumab were assessed in VAYHIT2 with ianalumab 9 mg/kg demonstrating statistically significant improvements across both the primary and key secondary endpoints, and ianalumab 3 mg/kg demonstrating statistically significant improvements in the primary endpoint and numerical improvements in the key secondary endpoint1-3.

Ianalumab 9 mg/kg + eltrombopag (N=50) Ianalumab 3 mg/kg + eltrombopag (N=51) Placebo + eltrombopag (N=51)
Primary endpoint: Time to treatment failure (TTF) 13.0 months
(HR 0.55; 95% CI: 0.32, 0.92; p=0.021a) Not estimable
(HR 0.58; 95% CI: 0.34, 0.98; p=0.023a) 4.7 months
Key secondary endpoint: Stable response at 6 months (SR6) 62.0%
(p=0.023a) 56.9%
(p=0.035a) 39.2%
a. Required p-value for statistical significance is one-sided <0.025

Ianalumab was well tolerated with no new safety signals, and the side effect profile was consistent with previous studies1,2. Adverse events were comparable between the ianalumab and placebo arms, with the most common AEs for ianalumab plus eltrombopag being headache (14% with 9 mg/kg, 10% with 3 mg/kg vs. 8% with placebo) and infusion-related reactions (14% with 9 mg/kg, 8% with 3 mg/kg vs. 8% with placebo)1,2. Neutropenia* occurred more frequently in the ianalumab groups (16% with 9 mg/kg, 12% with 3 mg/kg) compared to placebo (2%) with most cases resolving without requiring treatment or dose interruption1,2. No on-treatment adverse event led to permanent discontinuation of therapy1,2.

VAYHIT2 marks the third positive Phase III trial with ianalumab, following two positive trials in adults with active Sjögren’s disease1,4. Novartis plans to submit the data from VAYHIT2 along with results from the ongoing first-line ITP trial, VAYHIT1, in 2027. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency5,6.

*An adverse event of special interest encompassing several terms related to low levels of neutrophils, neutrophil precursors and leukocytes

About ianalumab
Ianalumab (VAY736) is a novel fully human monoclonal antibody being investigated for its potential to treat various B cell-driven autoimmune diseases, including Sjögren’s disease, immune thrombocytopenia (ITP), systemic lupus erythematosus (SLE), lupus nephritis (LN), warm autoimmune hemolytic anemia (wAIHA) and diffuse cutaneous systemic sclerosis (dcSSc)3,7-13. Its mechanism of action targets B cells in two ways, namely combining B cell depletion via antibody-dependent cellular toxicity (ADCC) and interruption of BAFF-R mediated signals of B cell function and survival8. In clinical trials, ianalumab showed promising efficacy and a favorable safety profile in Sjögren’s disease, systemic lupus erythematosus, and immune thrombocytopenia4,14-16. Ianalumab originates from an early collaboration with MorphoSys AG, a company which Novartis later acquired in 202417.

About primary immune thrombocytopenia
Primary immune thrombocytopenia (ITP) is a rare, autoimmune disorder in which the immune system mistakenly targets and destroys platelets, the cells essential for blood clotting18. This can lead to symptoms such as prolonged bleeding, easy bruising and chronic fatigue, which can significantly impact daily life18,19.

Despite available treatments, many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control20. Current options often focus on maintaining safe platelet levels and preventing bleeding complications and may require ongoing use20,21. The burden of chronic treatment and unpredictability of relapses can significantly impact quality of life19,22. There is a need for therapies that offer durable response while reducing the burden of long-term treatment23.

About VAYHIT2
VAYHIT2 (NCT05653219) is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of two different doses of ianalumab versus placebo, in addition to eltrombopag, in adults with primary immune thrombocytopenia (ITP) (platelet count <30 G/L) who failed previous first-line treatment with corticosteroids3. Alongside eltrombopag, patients were randomized 1:1:1 to receive four once-monthly intravenous infusions of ianalumab at 3 mg/kg, ianalumab at 9 mg/kg or placebo3. The primary endpoint was time to treatment failure, which is defined as the time from randomization until either: a platelet count of less than 30 G/L later than 8 weeks from randomization; the need for rescue therapy later than 8 weeks from randomization; initiation of a new ITP treatment at any time; ineligibility or inability to taper/discontinue eltrombopag; or death3. The key secondary endpoint is the percentage of patients with a stable platelet count response at Month 63. Other secondary endpoints include measures of depth and duration of platelet response as well as patient-reported outcomes that measure quality of life and fatigue, among other endpoints.

(Press release, Novartis, DEC 9, 2025, View Source [SID1234661317])