• Updated longer-term analysis, including up to four years of follow-up data in patients with relapsed or refractory BRAF-altered pediatric low-grade glioma treated with OJEMDA^®(tovorafenib), will be presented as an oral presentation on November 14 at 3:45 p.m. EST
• Data expand on clinically meaningful results from earlier analyses of FIREFLY-1 pivotal trial

BOSTON, Nov. 14, 2026 /PRNewswire/ -- Servier today announced updated four-year results from the Phase 2 FIREFLY-1 study of OJEMDA^® (tovorafenib) in patients aged six months to 25 years with relapsed or refractory BRAF-altered pediatric low-grade glioma (pLGG). The data are being presented as an oral presentation at the 31st Society for Neuro-Oncology (SNO) Annual Meeting on November 14 at 3:45 p.m. EST in Philadelphia.

The four-year FIREFLY-1 analysis includes exploratory outcomes across the treatment and post-treatment observation periods, with a median study duration of 52.5 months in the registrational arm as of the June 5, 2026, data cutoff. Patients received OJEMDA orally once weekly in 28-day cycles for 26 cycles, after which they could continue treatment or enter the optional post-treatment observation period. The analysis assessed exploratory endpoints of time to next treatment and treatment-free interval to further characterize outcomes following treatment discontinuation.

"With four years of follow-up from FIREFLY-1, we are building a more complete picture of OJEMDA's long-term outcomes, which will help physicians and families make critical treatment decisions," said Elly Barry, MD, Vice President of Clinical Development, Servier Pharmaceuticals. "We are proud to share these results, which, combined with the consistent safety profile, reinforce OJEMDA's role as a meaningful treatment option. We remain committed to evaluating the effects of OJEMDA over time and continuing to support children, adolescents and young adults with pLGG."

Updated 4-year results from the phase 2 FIREFLY-1 study: Deep responses and prolonged time off therapy with tovorafenib in relapsed/refractory pediatric low-grade glioma – Saturday, November 14, 3:45 – 3:52 p.m. EST

Key findings from the exploratory analysis to be presented by Daniel Landi, MD, Duke University and Duke Cancer Institute, an investigator on the FIREFLY-1 trial, include:

• Among the 76 efficacy-evaluable patients, median time to next treatment was 50.9 months (95% CI 36.7-not estimable [NE]).
  • The median time to next treatment in the three-year analysis, which was presented at SNO 2025, was 42.6 months.
• Among the 41 patients who entered the optional post-treatment observation period:
  • 76% of patients (n=31) remained treatment-free for at least 12 months, and 54% of patients (n=22) remained treatment-free for at least 24 months. The median treatment-free interval was not reached.
  • 54% of patients (n=22) experienced tumor rebound off treatment and seven patients were retreated.
    • 27% of patients (n=11) were retreated with OJEMDA. At last scan, nine of those patients had a smaller tumor than prior to retreatment.
    • Five of 13 patients with BRAF V600E mutations experienced tumor rebound and four were retreated.
• The safety profile of OJEMDA remained consistent with the established profile. In the safety analysis set (n=137), the most common Grade ≥3 treatment-related adverse events (TRAEs) observed in ≥5% of patients included reduced growth velocity (36%), anemia (14%), increased CPK (11%), maculopapular rash (8%), and increased ALT (5%). There were no treatment-related deaths and no additional discontinuations due to TRAEs since the three-year data cutoff.
  • In the growth analysis set (n=90), 88% of patients (n=79) demonstrated growth recovery, irrespective of the degree of on-treatment growth suppression.

"For children and families living with pediatric low-grade glioma, time spent off treatment is meaningful," said Daniel Landi, MD, Duke University and Duke Cancer Institute. "These longer-term FIREFLY-1 findings provide additional information about patient outcomes, including treatment duration and time off therapy, which may help inform discussions between physicians and families about treatment decisions."

Media Contact 
Darby Malkin: darby.malkin@servier.com 

About OJEMDA^®
OJEMDA^® (tovorafenib) is a Type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. OJEMDA is indicated for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Tovorafenib was granted Breakthrough Therapy and Rare Pediatric Disease designations by the FDA for the treatment of patients with pLGG harboring an activating RAF alteration, and it was evaluated by the FDA under priority review. Tovorafenib has also received Orphan Drug designation from the FDA for the treatment of malignant glioma and from the European Commission for the treatment of glioma.

Please see full prescribing information including important safety information about OJEMDA at www.OJEMDA.com.

About Pediatric Low-Grade Glioma
Pediatric low-grade gliomas (pLGG) are the most common brain tumor with an estimated US incidence of 1,100 and Europe incidence of 700 children per year who are eligible for front-line systemic therapy.^1,2 BRAF is the gene most commonly altered in pLGG, which includes two primary types of BRAF alterations – a BRAF gene fusion and BRAF point mutation. These BRAF alterations account for >50% of pLGG cases worldwide and prior to the introduction of OJEMDA, there were no approved treatments for people with pLGG driven by BRAF fusions.^1,3

Pediatric low-grade gliomas can be chronic and relentless, with patients suffering profound side effects from both the tumor and the treatment, which may include chemotherapy and radiation. These side effects can impact their life over the long term, and may include motor deficiencies, vision loss, hormone deficiency and alterations in growth and development. Most children with pLGG will survive their cancer, but for the majority of those in whom a complete surgical resection is not possible, these tumors tend to recur frequently throughout childhood, necessitating multiple treatments. The cumulative toxicity of numerous therapies, along with the damage caused by multiple episodes of tumor progression, take a significant toll on the children and their families.

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.   

WHAT IS OJEMDA^® (tovorafenib)?
OJEMDA is a prescription medicine used to treat a type of brain tumor (cancer) called pediatric low-grade glioma (pLGG) in people 6 months of age and older whose tumor has come back after previous treatment or did not respond to previous treatment, and that has a certain type of abnormal BRAF gene. A healthcare provider will perform a test to confirm that OJEMDA is right for the patient.

OJEMDA was approved based on how many patients' tumors responded to treatment and how long those responses lasted. Continued approval may depend on confirmation of clinical benefit in a confirmatory study. It is not known if OJEMDA is safe and effective in children younger than 6 months of age.

IMPORTANT SAFETY INFORMATION

OJEMDA may cause serious side effects, including:

• Bleeding problems (hemorrhage). Bleeding is common during treatment with OJEMDA and can also be serious. Patients should contact their healthcare provider or get medical help right away if they have any signs of bleeding, such as headache, dizziness or weakness, coughing up or vomiting blood (or vomit that looks like coffee grounds), or red or black stools that look like tar.

• Skin reactions, including sensitivity to sunlight (photosensitivity). OJEMDA can cause skin reactions that may become severe. Patients should tell their healthcare provider about new or worsening skin reactions, such as rash, acne, bumps, blisters, or peeling, redness, or irritation. Patients should limit time in sunlight and use sun protection, such as sunscreen, sunglasses, and protective clothing, during treatment.

• Liver problems. OJEMDA can cause liver problems. A healthcare provider will do blood tests to check the liver before and during treatment. Patients should tell their healthcare provider right away about yellowing of the skin or the whites of the eyes, dark or brown (tea-colored) urine, nausea or vomiting, loss of appetite, feeling tired, bruising, bleeding, or pain in the upper right stomach area.

• Slowed growth in children. OJEMDA can slow growth in children. A child's growth will be checked routinely during treatment with OJEMDA.

• Harm to an unborn baby. OJEMDA can harm an unborn baby. A healthcare provider will check whether a patient is pregnant before starting OJEMDA. Females who are able to become pregnant should use effective nonhormonal birth control during treatment and for 28 days after the last dose, because OJEMDA can cause some hormonal birth control to not work as well. Males with female partners who are able to become pregnant should use effective birth control during treatment and for 2 weeks after the last dose. Patients should tell their healthcare provider right away if they become pregnant or think they might be pregnant during treatment.

Before receiving OJEMDA, patients should tell their healthcare provider:

• about all of their medical conditions, including if they have bleeding, skin, or liver problems; or are pregnant, plan to become pregnant, or are breastfeeding.

• about all the medicines they take, including prescription and over-the-counter medicines, vitamins, herbal supplements, and any hormonal birth control. OJEMDA may affect how other medicines work, and other medicines may affect how OJEMDA works.

Patients should not breastfeed during treatment with OJEMDA and for 2 weeks after the last dose.

OJEMDA may cause fertility problems in males and females, which may affect the ability to have children.

The most common side effects of OJEMDA include rash, hair color changes, feeling tired, viral infection, vomiting, headache, bleeding, fever, dry skin, constipation, nausea, acne, and upper respiratory tract infection.

The most common severe abnormal blood test results include decreased phosphate, decreased red and white blood cells, increased creatine phosphokinase, increased liver function tests, decreased albumin, decreased potassium, and decreased sodium.

These are not all of the possible side effects of OJEMDA. Talk to a healthcare provider for medical advice about side effects.

Patients are encouraged to report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or to Day One Biopharmaceuticals at 1-877-204-2820.

Please see Patient Information and Instructions for Use.

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. Ryall S, et al. Acta Neuropathol Commun. 2020;8(1):30.
2. Estimates of annual incidence and prevalence for addressable patient population in E.U. 4 + U.K. are based on Ipsen calculations from publicly available data (Eurostat, <25yo population; Global Burden of Disease 2019; Desandes et al. Incidence and survival of children with central nervous system primitive tumors in the French National Registry of Childhood Solid Tumors. Neuro Oncol. 2014 Jul;16(7):975-83. doi: 10.1093/neuonc/not309; Qaddoumi et al. Outcome and prognostic features in pediatric gliomas: a review of 6212 cases from the Surveillance, Epidemiology, and End Results database. Cancer. 2009 Dec 15;115(24):5761-70. doi: 10.1002/cncr.24663)
3. Traunwieser T, et al. Neurooncol Adv. 2020;2(1):vdaa094.

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