Servier to Present Extended Follow-Up Results from the Phase 3 INDIGO Trial Showing Durable and Sustained Treatment Benefits of VORANIGO® (vorasidenib) at ASCO 2026

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May 31, 2026, 17:36 ET

Latest long-term analysis, including more than three years of follow-up data of patients with Grade 2 IDH-mutant glioma treated with VORANIGO^® (vorasidenib), will be presented in a rapid oral session on May 31 at 4:36 p.m. CDT
Results show median progression-free survival among patients treated with VORANIGO was 44.1 months
Findings from an exploratory analysis from the INDIGO study evaluating the impact of VORANIGO on seizure rates and quality of life will be shared in a separate poster presentation

BOSTON, May 31, 2026 /PRNewswire/ -- Servier today announced updated efficacy and safety results from more than three years of follow-up in the Phase 3 INDIGO trial evaluating VORANIGO^® (vorasidenib) versus placebo in patients with Grade 2 mutant isocitrate dehydrogenase 1 or 2 (IDH1/2) glioma following surgical intervention and who are not in immediate need of chemoradiotherapy. These data, which will be presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting on May 31 at 4:36 p.m. CDT in Chicago, strengthen the previous findings from the INDIGO pivotal trial and demonstrate the clinical benefits of VORANIGO continue to improve over time.

The extended analysis includes 21.3 months of additional unblinded data collected between the March 7, 2023, trial unblinding and the January 17, 2025, data cutoff. As of data cutoff, all 163 patients enrolled in the placebo arm discontinued treatment and 144 crossed over to the VORANIGO treatment arm. Median follow-up was 41.6 months. These results build upon the placebo-controlled, double-blind data previously published in The Lancet Oncology.

"The extended Phase 3 INDIGO trial analysis significantly improves our understanding of the clinical benefits of VORANIGO," said Islam Hassan, M.D., MSc., Global Head of Development-Neuro-Oncology at Servier. "With more than three years of follow-up data, these results validate the durable and sustained benefits of long-term treatment with VORANIGO and demonstrate a steady improvement in patient responses over time. We are proud to share these results and remain committed to expanding our understanding of the long-term impact of VORANIGO for the glioma community."

A global, randomized, double-blinded, Phase 3 trial of vorasidenib vs placebo in patients with grade 2 glioma with an IDH1/2 mutation (INDIGO): updated efficacy and safety – Sunday, May 31, 4:36 - 4:42 p.m. CDT

Key findings, which were presented by Timothy Cloughesy, M.D., David Geffen School of Medicine, Department of Neurology, University of California, Los Angeles, an investigator for the INDIGO trial, include:

Median progression-free survival (PFS) per blinded independent review committee (BIRC) was 44.1 months (95% CI, 27.7-not estimable [NE]) in patients treated with VORANIGO. PFS was the primary endpoint of the trial.
The median time to next intervention (TTNI) per BIRC for patients treated with VORANIGO was NE (95% CI, 52-NE). The number of next-intervention events remained low among VORANIGO patients (23.8%), indicating that VORANIGO is effectively delaying the need for subsequent treatment. TTNI was a key secondary endpoint of the trial.
Patients treated with VORANIGO experienced an objective response rate (ORR) per BIRC of 20.8% (95% CI, 15%-27.8%). ORR improved with longer follow-up, reflecting durable and gradual responses with VORANIGO. An additional 72.6% of patients experienced stable disease.
Median duration of treatment among VORANIGO patients was 38.3 months (95% CI, 19.98-43.76).
A 72% reduction in the rate of on-treatment seizures was observed in patients treated with VORANIGO.
The safety profile of VORANIGO was tolerable with mainly low-grade adverse events (AEs) and consistent with previously reported data. The most commonly reported Grade ≥3 or worse treatment-emergent adverse events (TEAEs) were increased alanine aminotransferase (10.8%), increased aspartate aminotransferase (4.8%), seizures (4.8%), increased gamma-glutamyltransferase (3%) and syncope (1.8%). No new safety signals were detected and fewer than 5% of patients discontinued treatment due to an AE. There were no treatment-related deaths.

"Historically, many patients with Grade 2 IDH-mutant gliomas faced a poor long-term prognosis and managed their disease with a 'watch and wait' strategy due to the limited availability of targeted treatment options," said Dr. Timothy Cloughesy. "The latest results from the Phase 3 INDIGO trial—the largest dataset in IDH-mutant glioma to date—provide valuable evidence on how long-term IDH inhibition can delay disease progression and extend the time to next intervention."

Impact of vorasidenib vs placebo on seizure rates and quality of life: exploratory analysis from Phase 3 INDIGO study – Monday, June 1, 1:30 - 4:30 p.m. CDT

Findings from an exploratory analysis of the INDIGO study evaluating the impact of VORANIGO on seizure rates and quality of life will be shared in a separate presentation. As of data cut off in January 2025, results show:

The rate of seizures per person per year in the VORANIGO arm (15.9 seizures per person-year [95% CI, 9.1-27.7]; p=0.0002) demonstrated the sustained, positive, long-term effect of VORANIGO on seizure control.
The effects of VORANIGO were more pronounced among people with oligodendrogliomas (6.9 seizures per person-year [95% CI, 2.7-17.7]; p<0.0001) than those with astrocytoma (17.5 seizures per person-year [95% CI, 9.4-32.3]; p=0.6448).
VORANIGO had an acceptable safety profile with no new safety signals during extended follow-up.

Media Contact
Darby Malkin: [email protected]

About Servier in Oncology
Servier is a global leader in oncology, governed by a non-profit foundation. Servier approaches innovation with a long-term vision, free of influence from fiduciary responsibilities.

Servier invests close to 20% of revenue from brand-name medicines in R&D each year. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes we can serve more people by helping the right patients find the right treatment, at the right time.

Servier takes a One Innovation Engine approach to R&D and is actively seeking alliances, partnerships and acquisitions at various stages of the portfolio.

For more information about working with Servier to bring the promise of tomorrow to the patients it serves, visit Servier.com and Servier.us.

About the INDIGO Phase 3 Trial (NCT04164901)¹
INDIGO, the pivotal Phase 3 clinical trial, was a registration-enabling Phase 3 global, randomized, double-blind placebo-controlled study of vorasidenib in patients with residual or recurrent Grade 2 glioma with an isocitrate dehydrogenase 1/2 (IDH1/2) mutation who have undergone surgery as their only treatment.

As of March 7, 2023, 331 patients were randomized globally to receive VORANIGO 40 mg daily (n=168) or placebo (n=163) continuously in 28-day cycles. Of the 331 patients, 172 had oligodendroglioma (88 VORANIGO; 84 placebo) and 159 patients had astrocytoma (80 VORANIGO; 79 placebo).

About Glioma²
Gliomas are tumors that arise from glial or precursor cells within the central nervous system (CNS). The 2021 World Health Organization (WHO) classification recognizes four general groups of gliomas, one of which is adult-type diffuse gliomas. These diffuse gliomas are the most common primary malignant brain tumors in adults. The pathogenesis and prognosis of these tumors are tightly linked to mutations (or lack thereof) in the metabolic enzyme isocitrate dehydrogenase (IDH), and molecular testing is required for proper diagnosis. As of 2021, adult-type diffuse gliomas are sub-divided into only three categories:

Astrocytoma, IDH-mutant (CNS WHO grades 2-4)
Oligodendroglioma, IDH-mutant and1p19q-codeleted (CNS WHO grades 2-3)
Glioblastoma, IDH-wildtype (CNS WHO grade 4)

VORANIGO IMPORTANT SAFETY INFORMATION

What is VORANIGO?

VORANIGO (40 mg tablets) is a prescription medicine used to treat adults and children 12 years of age and older with certain types of brain tumors called astrocytoma or oligodendroglioma with an isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, following surgery. Your healthcare provider will perform a test to make sure that VORANIGO is right for you. It is not known if VORANIGO is safe and effective in children under 12 years of age.

What are the possible side effects of VORANIGO?

VORANIGO may cause serious side effects, including:

Liver problems. Changes in liver function blood tests may happen during treatment with VORANIGO and can be serious. Your healthcare provider will do blood tests to check your liver function before and during treatment with VORANIGO. Tell your healthcare provider right away if you develop any of the following signs and symptoms of liver problems:
- yellowing of your skin or the white part of your eyes (jaundice)
- dark tea-colored urine 
- loss of appetite 
- pain on the upper right side of your stomach area 
- feeling very tired or weak

The most common side effects of VORANIGO include:

increased liver enzyme levels in the blood
lack of energy, tiredness
headache
COVID-19
muscle aches or stiffness
diarrhea
nausea
seizure

Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with VORANIGO if you have certain side effects.

VORANIGO may affect fertility in females and males, which may affect the ability to have children. Talk to your healthcare provider if this is a concern for you.

These are not all of the possible side effects of VORANIGO.

Before taking VORANIGO, tell your healthcare provider about all of your medical conditions, including if you:

have liver problems
have kidney problems or are on dialysis
smoke tobacco
are pregnant or plan to become pregnant. VORANIGO can harm your unborn baby

Females who are able to become pregnant:

Your healthcare provider will do a pregnancy test before you start treatment with VORANIGO
You should use effective nonhormonal birth control during treatment with VORANIGO and for 3 months after the last dose. VORANIGO may affect how hormonal contraceptives (birth control) work and cause them to not work well. Talk to your healthcare provider about birth control methods that may be right for you during treatment with VORANIGO
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with VORANIGO

Males with female partners who are able to become pregnant:

You should use effective birth control during treatment with VORANIGO and for 3 months after the last dose
Tell your healthcare provider right away if your partner becomes pregnant or thinks she may be pregnant during your treatment with VORANIGO

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if VORANIGO passes into breast milk. Do not breastfeed during treatment with VORANIGO and for 2 months after the last dose.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VORANIGO may affect the way other medicines work, and other medicines may affect how VORANIGO works.

Please click here for full prescribing information.

Disclosures
This release contains general information about the Servier Group and its entities (hereinafter "Servier and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however, Servier and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect.

Servier and its Affiliates are not acting as an advisor to the recipient of this information, and the ultimate decision to proceed with any transaction rests solely with the recipient of this information. Therefore, prior to entering into any proposed transaction, the recipient of this information should determine, without reliance upon Servier or its Affiliates, the economic risks and merits, as well as the legal, tax, and accounting characterizations and consequences, of the transaction and that it is able to assume these risks.

This statement also contains forward-looking statements that are subject to varying levels of uncertainty and risk. Investigational new drugs and indications are subject to further scientific and medical review and regulatory approval. They are not approved for use by the FDA.

Any reliance placed on this document is done entirely at the risk of the person placing such reliance. The information contained in this document is neither an offer to sell nor the solicitation of an offer to enter into a transaction.

The content of this document is a summary only, is not complete, and does not include all material information about Servier and its Affiliates, including potential conflicts of interest.

To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates disclaim all representations, warranties, conditions and guarantees, whether express, implied, statutory or of other kind, nor does it accept any duty to any person, in connection with this document. Without prejudice to the generality of the foregoing, Servier and its Affiliates do not warrant or represent that the information or opinions contained in this document is accurate or complete.

To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates shall not be liable for any loss, damage or expense whatsoever, whether direct or indirect, howsoever arising, whether in contract, tort (including negligence), strict liability or otherwise, for direct, indirect, incidental, consequential, punitive or special damages arising out of or in connection with this document, including (without limitation) any course of action taken on the basis of the same. The estimates, strategies, and views expressed in this document are based upon past or current data and information and are subject to change without notice.

References
1. Mellinghoff, I. K., van den Bent, M. J., Blumenthal, D. T., Touat, M., Peters, K. B., Clarke, J., Mendez, J., Yust-Katz, S., Welsh, L., Mason, W. P., Ducray, F., Umemura, Y., Nabors, B., Holdhoff, M., Hottinger, A. F., Arakawa, Y., Sepulveda, J. M., Wick, W., Soffietti, R., … Cloughesy, T. F. (2023). Vorasidenib in idh1- or IDH2-mutant low-grade glioma. New England Journal of Medicine, 389(7), 589–601. https://doi.org/10.1056/nejmoa2304194

2. Louis DN, Perry A, Wesseling P, Brat DJ, Cree IA, Figarella-Branger D, Hawkins C, Ng HK, Pfister SM, Reifenberger G, Soffietti R, von Deimling A, Ellison DW. The 2021 WHO Classification of Tumors of the Central Nervous System: a summary. Neuro Oncol. 2021 Aug 2;23(8):1231-1251. doi: 10.1093/neuonc/noab106. PMID: 34185076; PMCID: PMC8328013.

SOURCE Servier Pharmaceuticals