On November 2, 2026 Ractigen Therapeutics, a leading clinical-stage biopharmaceutical company developing innovative small activating RNA (saRNA) therapeutics, reported the official initiation of its multi-center Phase II clinical trial (Study RAG010201) of RAG-01 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) in China, marked by a clinical site initiation meeting held at Fudan University Shanghai Cancer Center (FUSCC).

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Designed as a randomized, controlled, multi-center Phase II study, the RAG010201 trial evaluates the efficacy, safety, tolerability, and pharmacokinetics (PK) of intravesical RAG-01 as a monotherapy and in combination with the chemotherapeutic agent mitomycin C (MMC) in patients with intermediate- or high-risk NMIBC.

The multi-center Phase II trial is led by Leading Principal Investigator Professor Ding-Wei Ye, a renowned authority in urologic oncology at FUSCC and Chief Expert of the Urologic Oncology Multidisciplinary Team (MDT). A total of 11 premier medical institutions across China are participating in the study, including:

Fudan University Shanghai Cancer Center (Prof. Ding-Wei Ye)
Peking University First Hospital (Prof. Xue-Song Li)
Ruijin Hospital affiliated to Shanghai Jiao Tong University School of Medicine (Prof. Dan-Feng Xu)
The Second Hospital of Tianjin Medical University (Prof. Hai-Long Hu)
Hunan Cancer Hospital (Prof. Gong-Qian Zeng)
Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School (Prof. Hong-Qian Guo)
Union Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology (Prof. Xiao-Ping Zhang)
Qilu Hospital of Shandong University (Prof. Ben-Kang Shi)
Fujian Medical University Union Hospital (Prof. Shao-Xing Zhu)
The First Affiliated Hospital of Xiamen University (Prof. Jin-Chun Xing)
Sun Yat-sen Memorial Hospital of Sun Yat-sen University (Prof. Jian Huang)
To address the distinct clinical challenges across risk stratifications in intermediate- and high-risk NMIBC, the RAG010201 study incorporates two tailored cohorts:

Cohort A (High-Risk NMIBC): Patients with high-risk NMIBC face a substantial risk of tumor recurrence and progression to muscle-invasive disease, creating an urgent need for more potent bladder-sparing regimens. Cohort A will assess RAG-01 monotherapy versus RAG-01 in combination with MMC. Following a safety run-in phase to establish the recommended dosing regimen, the study will proceed to an open-label, 1:1 randomized controlled phase with the 24-month event-free survival (EFS) rate as the primary endpoint, evaluating both the standalone activity of RAG-01 and its synergistic potential with intravesical chemotherapy.
Cohort B (Intermediate-Risk NMIBC): For intermediate-risk patients, clinical management prioritizes preventing recurrence and extending recurrence-free survival while minimizing treatment-related toxicity. Cohort B is designed as a single-arm study evaluating intravesical RAG-01 monotherapy, with the 24-month recurrence-free survival (RFS) rate serving as the primary endpoint.
RAG-01 is a first-in-class saRNA therapeutic designed to selectively target and upregulate p21, a key tumor suppressor gene that regulates cell cycle arrest and cellular senescence, thereby inhibiting the abnormal proliferation of bladder cancer cells. Delivered locally via intravesical instillation using Ractigen’s proprietary LiCO delivery system, RAG-01 has already demonstrated a favorable safety profile, clear dose-dependent target engagement, and encouraging durable clinical responses in its Phase I clinical trial in Australia. Preclinical study results demonstrated that RAG-01 in combination with MMC exerted significant synergistic inhibitory effects on bladder cancer cell proliferation.

Professor Ding-Wei Ye, Leading Principal Investigator of the study at Fudan University Shanghai Cancer Center, commented: "Following transurethral resection of bladder tumor (TURBT), patients with NMIBC continue to face daunting rates of tumor recurrence and progression. In clinical practice, persistent Bacillus Calmette-Guérin (BCG) shortages, BCG intolerance or failure, and the suboptimal durability of conventional intravesical chemotherapies highlight a critical unmet need for therapies with novel mechanisms of action. By leveraging cutting-edge RNA activation (RNAa) technology to selectively upregulate the p21 tumor suppressor gene, RAG-01 introduces an entirely new therapeutic paradigm for localized bladder cancer treatment. We look forward to collaborating with top urology centers nationwide to advance this rigorously designed Phase II study and evaluate the potential of RAG-01, both alone and in combination, to reduce recurrence, delay disease progression, and preserve patients’ bladders."

"Kicking off our Phase II trial at Fudan University Shanghai Cancer Center alongside Professor Ding-Wei Ye and 10 other top-tier clinical centers across China is a major milestone for the RAG-01 program," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "As a former urologist myself, addressing the unmet needs of bladder cancer patients has been a driving force behind translating RNA activation technology from bench to bedside. Building on the encouraging durability and dose-response signals observed in our Phase I study, we are confident that the world-class surgical quality and rigorous clinical execution of our participating Chinese centers will enable us to fully unlock the therapeutic potential of RAG-01."

About RAG-01

RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNA activation (RNAa). Traditionally considered "undruggable" by conventional small molecules or antibodies, p21 presents a unique opportunity for saRNA-based targeted gene activation. Delivered through intravesical instillation using Ractigen’s proprietary LiCO delivery technology, RAG-01 has demonstrated encouraging preliminary efficacy, a favorable safety profile, and clear target engagement in its Phase I clinical trial conducted in Australia. RAG-01 previously received IND clearance from the U.S. Food and Drug Administration (FDA), was granted Fast Track Designation (FTD) by the FDA in 2024, and received Phase II IND approval from China’s NMPA Center for Drug Evaluation (CDE).

About NMIBC

Non-muscle invasive bladder cancer (NMIBC) is a common malignancy confined to the mucosal and submucosal lining of the bladder, accounting for approximately 75% of all newly diagnosed bladder cancer cases. The standard first-line treatment for intermediate- and high-risk NMIBC is transurethral resection of the bladder tumor (TURBT) followed by intravesical Bacillus Calmette-Guérin (BCG) immunotherapy or intravesical chemotherapy. However, a significant proportion of patients experience frequent recurrence, disease progression, or become unresponsive to BCG, often leaving radical cystectomy (complete removal of the bladder) as the only remaining option and highlighting the critical need for novel bladder-sparing therapies.

About RNAa

RNA activation (RNAa) is a clinically validated platform technology discovered and developed by Dr. Long-Cheng Li and his team. It utilizes small activating RNAs (saRNAs) to target gene regulatory domains at the transcriptional level, activating endogenous gene expression and restoring therapeutic protein levels. This transformative technology holds vast potential for developing first-in-class therapeutics across oncology, genetic disorders, and chronic diseases, particularly for targets where traditional pharmacological approaches fall short.

(Press release, Ractigen Therapeutics, NOV 2, 2026, View Source [SID1234674049])

On November 2, 2026 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that clinical and preclinical research from its neuro-oncology portfolio will be presented at the upcoming 2026 Society for Neuro-Oncology (SNO) Annual Meeting, taking place November 12-15 in Philadelphia.

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Key highlights include:

The confirmatory Phase 3 ACTION trial evaluating Modeyso (dordaviprone), formerly ONC201, continues to advance. In the trial, patients with newly diagnosed H3 K27M-mutant diffuse glioma who have completed standard frontline radiotherapy are randomized 1:1:1 to receive placebo, once-weekly dordaviprone, or twice-weekly dordaviprone on two consecutive days. The primary efficacy endpoint is overall survival.
A Phase 2 clinical trial for JZP3507 (formerly ONC206) has been initiated in recurrent meningioma, the most common form of primary central nervous system cancer in adults. JZP3507 is a dual targeted investigational therapy that is an agonist of the mitochondrial protease ClpP and an antagonist of the G protein-coupled receptor DRD2.
An oral presentation for JZP3507 will show new preclinical findings characterizing the anti-cancer activity in H3 G34-mutant glioma. JZP3507 demonstrated nanomolar potency in cell lines and patient-derived cells, as well as a ClpP-dependent mechanism underlying its anti-cancer effects.
"The data being presented at SNO demonstrates Jazz’s expanding commitment to scientific innovation and our progress to develop new therapies for patients facing rare and highly aggressive brain cancers," said Josh Allen, Ph.D., chief scientific officer, oncology, Jazz Pharmaceuticals. "Building on the landmark approval of Modeyso, we are advancing the Phase 3 ACTION trial, with an interim analysis in the first half of 2027, to potentially bring this therapy to patients earlier in the disease course."

Presentations at SNO 2026 include:

Presentation Title

Authors

Presentation Details

ACTION trial update: A randomized phase 3 study of dordaviprone (ONC201) in patients with newly diagnosed H3 K27M-mutant diffuse glioma

Arrillaga-Romany I*, Chang JH, Pellerino A, Brazil L, Cappellano A, McBain C, Lombardi G, Cardoso A, Dahlrot RH, Carceller F, Gironés Sarrió R, Pavon-Mengual M, Bhatia A, Allen J, Melemed A, Mayo C, Tarapore R, Wen P, Cloughesy T

Type: Poster presentation

Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center

Date: Friday, November 13, 7:30-9:30 p.m. ET

Number: TIP-11

A phase 2 study to investigate the efficacy and safety of JZP3507 (formerly ONC206) in adults with recurrent grade 2 or 3 meningioma

Ramage S*, Arrillaga-Romany I, Barbaro M, Gatson N, Johnson M, Kurz S, Mendez J, Odia Y, Sharma A, Umemura Y, Wen P, Mayo C, Shonka N

Type: Poster presentation

Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center

Date: Friday, November 13, 7:30-9:30 p.m. ET

Number: TIP-12

Allosteric ClpP agonist ONC206 alters mitochondrial metabolism to elicit anti-cancer efficacy in H3 G34-mutant glioma

Prabhu V*, Lee A, Maranto C, Foster S, Allen J

Type: Oral presentation

Date: Saturday, November 14, 2:30-3:30pm ET

Location: 120 ABC, First Floor, Pennsylvania Convention Center

Number: EXTH-15

EGFR signaling drives mesenchymal cell-state transition and therapeutic vulnerability in H3K27M-mutant Diffuse Midline Glioma

Ji S, Doherty R, Pearson A*, Wink K, Lau B, Murthy B, Mota M, Natarajan S, de la Nava D, Sajjakulnukit P, Adam T, Niculcea M, Vo K, Bavani S, Yadavalli S, Cruz A, Li F, Knowles T, Phillips J, Vo T, Liu I, Kumar P, McThenia S, Mazewski C, Pytel N, Bowers D, Green A, Lyssiotis C, Qin T, Solomon D, Beck A, Nuechterlein N, Agnihotri S, Toll S, Dun M, Venneti S, Mueller S, Nazarian J, Waszak S, Koschmann C

Type: Pediatric Oral Abstracts Session I

Date: Saturday, November 14, 3:45-4:45pm ET

Location: 120 ABC, First Floor, Pennsylvania Convention Center

Number: CSIG-09

Spatial Architecture of H3 K27M-mutant Diffuse Midline Glioma Reveals Prognostic Niches in Patients from the PNOC022 Clinical Trial

Savary C*, Vo T, Jackson E, Persson M, Dubois O, Douglas A, McEwen H, McLachlan T, Kline C, Franson A, Valvi S, van der Lugt J, Nazarian J, Waszak S, Koschmann C, Gupta N, Mueller S, Dun M

Type: CNS Rare Tumor Oral Abstracts Session I

Date: Friday, November 13, 2:30-3:30pm ET

Location: 119 AB, First Floor, Pennsylvania Convention Center

Number: BIOM-72

Leveraging the immunomodulatory effects of dordaviprone to induce adaptive immune surveillance in diffuse midline glioma

Persson M*, Savary C, Jackson E, Duchatel R, Vo T, Dubois O, Loughnan O, Findlay I, Douglas A, Bramberger L, de la Nava D, McEwen H, Beitaki T, Mannan A, Jayaraman R, Thomas B, Rodriguez de la Fuente L, Colino-Sanguino Y, Valdes Mora F, Faridi P, Holst J, Mayall J, Kline C, Franson A, Venneti V, Hondermarck H, Horvat J, Pearson A, Ji S, Nazarian J, Hulleman E, Mueller S, Vitanza N, Koschmann C, Alonso M, Carvalheiro T, van der Lugt J, Dun M

Type: Rapid Orals Session VI A

Poster Session

Date: Friday, November 13, 11:30am-12:30pm ET

Friday, November 13, 7:30-9:30pm ET

Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center

Number: TMIC-98

Exploiting Tumor Stress and Innate Immunity: ONC201 Enhances NK Cell Therapy for Pediatric Diffuse Midline Gliomas

Cheng D, Zhang P, Basar R, Shanley M, Singh A, Swaminathan J, Shrestha R, Siddaway R, Vadivel AA, Sharma A, Yang Y, Anjier A, Maegawa S, Feng S, Gordon N, Daw N, Gorlick R, Tarapore R, Prabhu V, Allen J, Koul D, Hawkins C, Zaky W, Wintermark M, Rezvani K, Gopalakrishnan V*

Type: Poster Session

Date: Friday, November 13, 7:30-9:30pm ET

Location: Exhibit Hall D & E, Second Floor, Pennsylvania Convention Center

Number: EXTH-26

In vitro and in vivo evaluation of CT-179 in combination with ONC201 and ONC206 in patient-derived models of diffuse midline glioma

Mizoguchi S, Yu J, Kritzer B, Laternser S, Stein G, Kesari S, Packer P, Nazarian J, Yadavilli S*

Type: Poster Session

Date: Friday, November 13, 7:30-9:30pm ET

Location: Exhibit Hall D & E, Second Floor, Philadelphia Convention Center

Number: EXTH-33

* Presenting author

About Modeyso (dordaviprone)
Modeyso (dordaviprone) is approved by FDA for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy.1 Modeyso is an orally administered small molecule given once weekly. Modeyso is a protease activator of the mitochondrial ClpP and also inhibits DRD2. In vitro, dordaviprone activates the integrated stress response, induces apoptosis, and alters mitochondrial metabolism, leading to restored histone H3 K27 trimethylation in H3 K27M-mutant diffuse glioma.1

Modeyso received accelerated approval based on overall response rate and duration of response derived from a pre-specified integrated efficacy analysis of 50 adult and pediatric patients with recurrent H3 K27M-mutant diffuse midline glioma enrolled across five open-label clinical studies (ONC006, ONC013, ONC014, ONC016, and ONC018). Full approval for this indication is contingent upon us satisfying post-marketing requirements, including the verification and description of clinical benefit in the Phase 3 ACTION confirmatory trial and other applicable post-marketing commitments. The FDA granted dordaviprone Rare Pediatric Disease designation and Fast-Track designation, and dordaviprone received Orphan Drug Designation in the United States, Europe and Australia. Modeyso was developed by Chimerix prior to its acquisition by Jazz Pharmaceuticals in April 2025.

Modeyso (dordaviprone) is approved in the United States and Japan. It is not approved anywhere else in the world.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Hypersensitivity
MODEYSO can cause severe hypersensitivity reactions.

In the pooled safety population, Grade 3 hypersensitivity reactions occurred in 0.3% of patients receiving MODEYSO. Signs and symptoms of hypersensitivity may include rash, hives, fever, low blood pressure, wheezing, or swelling of the face or throat.

Inform patients about the signs and symptoms of hypersensitivity reactions and instruct them to seek immediate medical attention if symptoms occur.

If clinically significant hypersensitivity or anaphylaxis occur, immediately interrupt MODEYSO and initiate appropriate medical treatment and supportive care. Based on the severity of the adverse reaction, temporarily interrupt or permanently discontinue MODEYSO.

QTc Interval Prolongation
MODEYSO causes concentration-dependent QTc interval prolongation, which can increase the risk for ventricular tachyarrhythmias (e.g. torsades de pointes) or sudden death.

In patients who received MODEYSO and underwent at least one post baseline ECG, QTcF increase of >60 msec compared to baseline and QTcF >500 msec occurred in 6% and 1.2% of patients, respectively.

Monitor ECGs and electrolytes prior to initiation and periodically during treatment, as clinically indicated. Increase the frequency of monitoring in patients with congenital long QT syndrome, existing QTc prolongation, a history of ventricular arrhythmias, electrolyte abnormalities, heart failure, or who are taking strong or moderate CYP3A4 inhibitors.

Avoid concomitant use with other agents known to prolong the QT interval. If concomitant use cannot be avoided, increase the frequency of monitoring and separate administration of MODEYSO and QT-prolonging product.

Interrupt or reduce the dose of MODEYSO in patients who develop QT prolongation; permanently discontinue in patients with signs of life-threatening arrhythmias.

Embryo-Fetal Toxicity
MODEYSO can cause fetal harm when administered to a pregnant woman.

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 MODEYSO and for 1 month after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with MODEYSO and for 1 month after the last dose.

ADVERSE REACTIONS
Serious adverse reactions occurred in 33% of the 376 patients who received MODEYSO. Serious adverse reactions in >2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%), and muscular weakness (2.1%). Fatal adverse reactions occurred in 1% of patients who received MODEYSO, including cardiac arrest (0.5%), intracranial hemorrhage (0.3%), and encephalopathy (0.3%).

The most common adverse reactions (≥20%) reported in clinical trials with MODEYSO were fatigue (34%), headache (32%), vomiting (24%), nausea (24%), and musculoskeletal pain (20%). The most common (≥2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (7%), decreased calcium (2.7%), and increased alanine aminotransferase (2.4%).

DRUG INTERACTIONS
Strong and Moderate CYP3A4 Inhibitors
Avoid concomitant use of MODEYSO with strong and moderate CYP3A4 inhibitors. If concomitant use cannot be avoided, reduce the MODEYSO dose as recommended and monitor for toxicity.

Strong and Moderate CYP3A4 Inducers
Avoid concomitant use of strong and moderate CYP3A4 inducers with MODEYSO.

USE IN SPECIFIC POPULATIONS
Lactation
There are no data on the presence of MODEYSO in human milk because of the potential for serious adverse reactions from MODEYSO in breastfed children, advise women not to breastfeed during treatment with MODEYSO and for 1 week after the last dose.

Pediatric Use
The safety and effectiveness of MODEYSO have not been established in patients less than 1 year of age. Dosing has not been established for patients weighing less than 22 pounds (10 kg).

Please refer to the full Prescribing Information, including both Patient Information and Instructions for Use, for complete safety and administration information.

The full U.S. Prescribing Information for MODEYSO is available at: View Source

(Press release, Jazz Pharmaceuticals, NOV 2, 2026, View Source [SID1234674048])

On November 2, 2026 Bold Therapeutics, a clinical-stage biopharmaceutical company developing novel metallotherapeutics, reported it will present at BIO-Europe in Cologne, Germany, which is taking place from November 9-11.

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Bold Therapeutics addresses high unmet medical needs through innovative mechanisms of action that transform oncology treatment. The company’s lead asset, BOLD-100, is a first-in-class ruthenium-based small molecule in a global randomized Phase 2 clinical trial for patients with second-line metastatic colorectal cancer (mCRC). The study evaluates the efficacy, safety, and tolerability of two BOLD-100 dose levels (500 mg/m² and 625 mg/m²) combined with FOLFOX-based standard of care (SOC) versus FOLFOX-based SOC alone.

The company will share key efficacy and tolerability findings from an interim analysis during its BIO-Europe presentation. "Bold Therapeutics has achieved significant corporate and clinical milestones that advance BOLD-100 toward registrational trials," said Jim Pankovich, EVP of Bold Therapeutics. "Results from this interim analysis build on earlier positive Phase 2 findings in gastrointestinal cancers, including biliary tract, gastric, and colorectal cancers. This progress brings BOLD-100 closer to delivering novel treatment options to underserved patient populations."

Presentation Details:

Date: November 9, 2026 (Day 1)

Time: 1:15PM – 1:30PM

Room: Theater A

(Press release, Bold Therapeutics, NOV 2, 2026, View Source [SID1234674047])

On November 2, 2026 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported its financial results for the third quarter ended September 30, 2026, and provided a business update.

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"We delivered another quarter of strong commercial results, generating $83 million in total revenue to Syndax. Revuforj net revenue grew to $61 million, driven by a 20% increase in new patients and an increasing number of patients on therapy in the post-transplant setting. We continue to have the dominant share of new patients across all age groups starting a menin inhibitor and the vast majority of revenue for the class, underscoring our position as the market leader. Importantly, a growing number of both R/R KMT2Ar and NPM1m patients are proceeding to a stem cell transplant after receiving Revuforj and then resuming therapy afterward, a dynamic that positions the business for long-term growth as the average duration of therapy extends," said Michael A. Metzger, Chief Executive Officer.

Mr. Metzger continued, "We look forward to sharing new Revuforj combination data in the fourth quarter that reinforce our confidence that our ongoing pivotal frontline trials could transform the treatment paradigm for newly diagnosed AML. We remain well positioned to be the first to deliver a menin inhibitor in the frontline, unlocking a multi-billion-dollar opportunity. With growing revenue from two medicines, a robust pipeline generating multiple blockbuster opportunities, world-class R&D capabilities, and a strong balance sheet, Syndax is poised to deliver long-term value."

Recent Business Highlights and Anticipated Milestones

Revuforj (revumenib)

Achieved $60.7 million in Revuforj net revenue in the third quarter of 2026, a 90% increase over the third quarter of 2025 and an 11% increase over the second quarter of 2026. New patients and total prescriptions increased by approximately 20% and 13%, respectively, in the third quarter of 2026 compared to the second quarter of 2026. The double-digit growth in net revenue, new patients, and total prescriptions reflects robust uptake in both Revuforj indications and a growing number of relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) and NPM1-mutated (NPM1m) patients proceeding to hematopoietic stem cell transplant (HSCT) and then resuming therapy in the post-transplant setting.

Claims data indicate that approximately 50% of R/R KMT2Ar patients proceed to HSCT after receiving Revuforj, of which about 60% have resumed therapy post-transplant. Notably, the data also indicate that approximately 40% of R/R NPM1m patients proceed to HSCT after receiving Revuforj, of which about 50% have resumed therapy post-transplant.

In October 2026, Revuforj received the Best Product for Rare/Orphan Diseases award from The Galien Foundation, one of the world’s most prestigious awards for biomedical research and innovation. Additionally, Revuforj was named as one of TIME’s Best Inventions of 2026 in the Medical and Healthcare category.

The Company expects to have a major presence at upcoming medical meetings, with the presentation in the fourth quarter of 2026 of new/updated revumenib data including:

Frontline data from the BEAT AML trial of revumenib in combination with venetoclax/azacitidine in NPM1m and KMT2Ar acute myeloid leukemia (AML)

Frontline data from the SAVE trial of the all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax in NPM1m, KMT2Ar, or NUP98-rearranged (NUP98r) AML

Frontline data from a Phase 1 trial of revumenib in combination with intensive chemotherapy in NPM1m, KMT2Ar, or NUP98r AML

Post-transplant maintenance data

Real-world evidence from multiple centers

Data from INTERCEPT, a Phase 1 trial evaluating revumenib as pre-emptive therapy for measurable residual disease (MRD) or early relapse among patients with NPM1m or KMT2Ar AML

The Company expects to publish safety and efficacy data from R/R NUP98r acute leukemia patients treated with revumenib in the fourth quarter of 2026.

Published preclinical revumenib data showing that menin is a novel dependency in proliferative megakaryocytes, major drivers of myelofibrosis (MF), in Cancer Cell in July 2026. These data provide the basis for the Company’s plans to develop SNDX-62122, an internally developed, next-generation menin inhibitor, for MF.

Multiple clinical trials evaluating revumenib across the acute leukemia treatment continuum are ongoing, such as:

EVOLVE-2: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed NPM1m (primary efficacy analysis population) and KMT2Ar AML patients who are unfit for intensive chemotherapy. The trial is being conducted in collaboration with the HOVON network, a leading cooperative clinical trial group with extensive experience studying novel therapies for hematologic malignancies.

REVEAL-ND: A pivotal, Phase 3, randomized, double-blind, placebo-controlled trial of revumenib in combination with intensive chemotherapy in newly diagnosed NPM1m AML patients.

SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with newly diagnosed and R/R AML or mixed-lineage acute leukemia harboring either NPM1m, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center.

Intensive chemotherapy: Two ongoing Phase 1 trials evaluating the combination of revumenib with intensive chemotherapy (7+3) in newly diagnosed NPM1m or KMT2Ar acute leukemia patients.

BEAT AML: A Phase 1b trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed older adults (≥60 years) with NPM1m or KMT2Ar AML. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial.

Post-transplant maintenance: A Phase 1 trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance in patients with KMT2Ar or NPM1m acute leukemia. The trial is being conducted by investigators from the City of Hope Medical Center.

Break Through Cancer: A Phase 2 trial studying whether the combination of revumenib and venetoclax can eliminate MRD in patients with AML and extend progression-free survival. The trial is being conducted by Break Through Cancer, a collaboration between leading U.S. cancer research centers.

INTERCEPT: A Phase 1 trial evaluating the use of novel therapies, including revumenib, to target MRD and early relapse in AML. The trial is being conducted by the Australasian Leukaemia and Lymphoma Group as part of the INTERCEPT AML master clinical trial.

The Company expects the RAVEN trial to initiate in the fourth quarter of 2026. RAVEN is a Phase 2 collaborative trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed KMT2Ar patients who would be considered eligible, or fit, for intensive chemotherapy.

The Company expects the MenTain Phase 2 trial to initiate in the first quarter of 2027. MenTain will be the first randomized, placebo-controlled trial specifically focused on evaluating revumenib as post-transplant maintenance.

Niktimvo (axatilimab-csfr)

Achieved $67.4 million in Niktimvo net revenue in the third quarter of 2026, a 47% increase over the third quarter of 2025 and a 12% increase over the second quarter of 2026. Syndax and Incyte are co-commercializing Niktimvo. Syndax records 50% of the Niktimvo net commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. Syndax’s share of the Niktimvo product contribution, reported as collaboration revenue, was $22.6 million in the third quarter of 2026.

In October 2026, data supporting the safety and feasibility of reduced-frequency dosing of axatilimab in patients with recurrent or refractory chronic graft-versus-host disease (GVHD) were published in Blood Advances. Data from the pivotal AGAVE-201 trial show that transition from axatilimab 0.3 mg/kg every two weeks to 0.6 mg/kg every four weeks was feasible and well tolerated. Patients had a prolonged duration of therapy after the switch (median of 21 months), and 84% remained on treatment as of the data cutoff, suggesting ongoing clinical benefit.

Two trials evaluating axatilimab in patients with newly diagnosed chronic GVHD are ongoing, including a Phase 2 trial in combination with ruxolitinib and a Phase 3 trial in combination with corticosteroids.

The Company expects topline data from the Phase 2 trial of axatilimab in combination with ruxolitinib to be presented at a medical meeting in the first quarter of 2027.

The Company expects topline data from the Phase 3 trial of axatilimab in combination with corticosteroids in early 2028.

In October 2026, the Company announced that the MAXPIRe Phase 2 trial of axatilimab in idiopathic pulmonary fibrosis (IPF) did not meet its primary endpoint. The safety and tolerability profile observed in MAXPIRe was consistent with previous clinical studies of axatilimab, with no new safety signals identified. Based on the results observed, Syndax does not plan to move forward with further development of axatilimab in IPF. The results from the MAXPIRe trial do not affect ongoing trials of axatilimab in other potential indications.

Pipeline Assets

SNDX-4321

In July 2026, the Company announced the expansion of its pipeline with SNDX-4321, a mutant-selective, CNS-penetrant, allosteric EGFR inhibitor. SNDX-4321 is in development for non-small cell lung cancer (NSCLC) patient populations with significant unmet needs, such as those with L858R mutations, CNS metastases, atypical activating mutations, or acquired resistance to current therapies. In contrast to ATP-site directed third- and fourth-generation EGFR inhibitors, SNDX-4321 is a novel allosteric inhibitor which binds at a pocket adjacent to the ATP site that is only accessible in the presence of L858R and certain other EGFR mutations.

The Company expects to submit an investigational new drug (IND) application for SNDX-4321 by the end of 2026 and to initiate a Phase 1 trial in EGFRm NSCLC in 2027.

SNDX-62122

In July 2026, the Company announced the selection of SNDX-62122, a next-generation menin inhibitor, for development in MF. SNDX-62122 is the first candidate from a library of internally developed, wholly owned next-generation menin inhibitors that the Company intends to advance into new areas.

The Company expects to submit an IND and initiate a Phase 1 trial of SNDX-62122 in MF in the fourth quarter of 2027. The development of SNDX-62122 will be informed by a Phase 1/2 proof-of-principle trial of revumenib in MF that is expected to initiate in the fourth quarter of 2026 with initial clinical data expected in the second half of 2027.

Corporate Updates

On November 2, 2026, the Company announced Keith Goldan’s decision to retire from his role as Chief Financial Officer and the appointment of Aaron A. Ondrey as Chief Financial Officer, effective November 9, 2026. Mr. Ondrey brings to Syndax more than 25 years of financial and operational leadership experience across the biotechnology industry, with deep expertise in financial management, corporate strategy, commercialization, and mergers and acquisitions.

Third Quarter 2026 Financial Results

As of September 30, 2026, Syndax had cash, cash equivalents, and short- and long-term investments of $551.1 million and 90.0 million common shares outstanding.

Total revenue for the third quarter of 2026 was $83.3 million, which consisted of $60.7 million in Revuforj net revenue and $22.6 million in Niktimvo collaboration revenue. The Niktimvo collaboration revenue is derived from the $67.4 million in Niktimvo net revenue that was previously reported by the Company’s partner Incyte for the third quarter 2026. Syndax records 50% of the Niktimvo net commercial profit, defined as net revenue (recorded by Incyte) minus the cost of sales and commercial expenses.

Third quarter 2026 research and development expenses increased to $67.5 million from $56.3 million for the comparable prior year period. The year-over-year change was primarily the result of increased expenses associated with frontline trials evaluating revumenib in combination with standard-of-care agents in the treatment of AML.

Third quarter 2026 selling, general and administrative expenses decreased to $43.7 million from $44.9 million for the comparable prior year period. The year-over-year change was primarily the result of lower commercial expenses due to launch costs incurred in 2025 for Revuforj and Niktimvo that were not incurred in the same period in 2026.

For the three months ended September 30, 2026, Syndax reported a net loss attributable to common stockholders of $49.4 million, or $0.55 per share, compared to a net loss attributable to common stockholders of $60.7 million, or $0.70 per share, for the comparable prior year period.

Financial Guidance

For the full year of 2026, the Company continues to expect total research and development plus selling, general and administrative expenses to be approximately $400 million, excluding the impact of $50 million in estimated non-cash stock compensation expense.

Syndax expects that its cash, cash equivalents and short- and long-term investments, combined with its anticipated product revenue, collaboration revenue and interest income, will enable the Company to reach profitability.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, November 2, 2026.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website. Alternatively, the conference call may be accessed through the following:

Conference ID: Syndax3Q26
Domestic Dial-in Number: 800-590-8290
International Dial-in Number: 240-690-8800
Live webcast: View Source

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.

About Revuforj (revumenib)

Revuforj (revumenib) is the first and only menin inhibitor that is FDA approved for the treatment of adult and pediatric patients one year and older with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test.

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

About Niktimvo (axatilimab-csfr)

Niktimvo (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).

In 2016, Syndax licensed exclusive worldwide rights to develop and commercialize axatilimab from UCB. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD, including a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774).

(Press release, Syndax, NOV 2, 2026, View Source [SID1234674046])

On November 2, 2026 BioArctic AB (publ) (Nasdaq Stockholm: BIOA B) reported that Novartis Pharma AG has notified BioArctic that it has decided to terminate the Option, Collaboration and License Agreement entered into in August 2025 to explore a potential new treatment combining BioArctic’s proprietary BrainTransporter technology with an undisclosed Novartis proprietary antibody.

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The termination is the result of a Novartis decision to discontinue the program, unrelated to BioArctic’s BrainTransporter technology.

BioArctic will retain the full USD 30 million upfront payment and, as a result of the termination, will recognize the remaining unrecognized portion as revenue in 2026.

BioArctic’s BrainTransporter technology facilitates the passage of drugs into the brain using the transferrin receptor (TfR). It can be used in a multitude of different therapy areas for delivery of biologics and other modalities to the brain. To date, BioArctic has entered into five collaboration agreements with partners using the BrainTransporter technology.

This is information that BioArctic AB (publ) is obliged to disclose pursuant to the EU Market Abuse Regulation. The information was released for public disclosure, through the agency of the contact person below, on November 2, 2026, at 9:30 pm CET.

(Press release, BioArctic Neuroscience, NOV 2, 2026, View Source [SID1234674045])