TLX101-Px (Pixlumi®) MAA Accepted in Europe

On April 30, 2026 Telix Pharmaceuticals Limited (ASX: TLX, NASDAQ: TLX, "Telix") reported that the marketing authorization application (MAA) filed in Europe for TLX101-Px (O-(2-[18F]fluoroethyl)-L-tyrosine, 18F-FET), its glioma (brain cancer) imaging candidate[1], has been validated and accepted for review.

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The application, covering commercially significant European markets[2], has now moved into a 210-day active assessment phase[3]. Telix is seeking to expand patient access to advanced brain imaging through a broad clinical label, reflective of current clinical practice guidelines[4]. Assuming a positive outcome from the application at Day 210, national marketing authorizations are expected to follow shortly after.

In Europe, there is currently no generally available commercial product for PET[5] imaging of glioma with 18F-FET ("FET-PET"), resulting in an acute and immediate need for a consistent, high-quality product [6]. Through this MAA, Telix aims to expand patient access to advanced imaging that can distinguish progressive or recurrent glioma from treatment-related changes in both adults and children, with potential for additional future indications. TLX101-Px is also being developed as a patient selection and response assessment tool for Telix’s glioblastoma therapy candidate TLX101-Tx (iodofalan 131I), which has been granted orphan drug designation in Europe and the U.S. The Phase 3 IPAX-BrIGHT[7] trial of TLX101-Tx in patients with recurrent glioblastoma has commenced patient dosing internationally[8] and is launching in multiple European countries.

Sied Kebir, MD, Head of Clinical Neuro-Oncology, University Hospital Essen, said: "In our day-to-day practice, one of the hardest questions we face is whether a change on conventional imaging reflects tumor progression or a treatment-related effect. PET imaging with ¹⁸F-FET can be used to help resolve this dilemma. The acceptance of this application is a welcome step toward broader, standardized patient access across Europe, and more timely and accurate decision-making."

Raphaël Ortiz, Chief Executive Officer, Telix International, commented, "The acceptance of our European MAA represents a significant regulatory milestone for Telix and for TLX101‑Px. It supports a critical unmet need for widely accessible glioma imaging for both diagnostic evaluation and therapeutic decision‑making. Subject to regulatory approval, we are preparing to bring this powerful precision medicine product to market in both Europe and the United States, where our new drug application has recently been accepted[9]."

About glioma in Europe

In Europe, approximately 67,500 brain and central nervous system tumors are diagnosed every year[10], with gliomas accounting for approximately 30% of these, and up to 80% of all malignant brain tumors[11]. There is a critical unmet need to improve the diagnosis and management of gliomas, which are the most common primary brain tumors of the central nervous system, particularly in the post-treatment setting4. Conventional MRI imaging techniques have several limitations, including a lack of biological specificity, dependency on blood-brain barrier disruption, and an inherent inability to differentiate between tumor progression or treatment-related causes. This can yield inconclusive results and delay time-sensitive treatment decisions[12]. With low survival rates and the need to make rapid decisions, precision imaging is paramount6. Subject to regulatory approval, TLX101-Px has the potential to address this need, enabling patients in Europe and worldwide to receive greater clarity in their diagnosis and treatment decision making.

About TLX101-Px

TLX101-Px (O-(2-[18F]fluoroethyl)-L-tyrosine) is Telix’s PET imaging candidate for the characterization of glioma. TLX101-Px targets membrane transport proteins known as L-type amino acid transporters 1 and 2 (LAT1 and LAT2). This enables TLX101-Px to be potentially utilized as a patient selection and response assessment tool for TLX101-Tx (iodofalan 131I), Telix’s LAT1-targeting glioblastoma (GBM) therapy candidate, currently under investigation in Telix’s IPAX-2[13] and IPAX-BrIGHT studies. TLX101-Px and TLX101-Tx have not received marketing authorizations in any jurisdiction. In relevant European markets, the proposed brand name for TLX101-Px is "Pixlumi". Brand name and commercial launch are subject to final regulatory approval.

(Press release, Telix Pharmaceuticals, APR 30, 2026, View Source [SID1234664980])

Krystal Biotech to Present at Upcoming Scientific Conferences

On April 30, 2026 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) reported that the Company will be presenting on multiple programs at upcoming scientific conferences being held in May and June.

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Presentation details are outlined below.

American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting
Poster Presentation

Title: Evaluation of KB409 and KB410, two HSV-1-based gene therapy vectors for the treatment of primary ciliary dyskinesia (PCD)
Presenter: Bruce Nmezi, PhD
Date and Time: May 12, 2026 from 5:00PM to 6:30PM ET

American Thoracic Society (ATS) International Conference 2026
Oral Presentation

Title: Interim results of the CORAL-1 trial of KB407 for the treatment of cystic fibrosis
Presenter: Jorge Lascano, MD, Professor of Medicine, Associate Director of the Adult Cystic Fibrosis Program, and Director of the Cystic Fibrosis Therapeutics Development Center at the University of Florida
Date and Time: May 20, 2026 at 10:03AM ET

2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting
Poster Presentation

Title: Inhaled delivery of KB707, a novel HSV-based immunotherapy, in combination with pembrolizumab in advanced non-small cell lung cancer: a phase 1/2 study
Presenter: Wen Wee Ma, MBBS, Enterprise Vice Chair for Research and Director of the Novel Cancer Therapeutics Center at Cleveland Clinic Cancer Institute
Date and Time: May 31, 2026 from 9:00AM to 12:00PM CT

Additional presentation details will be available to conference attendees. Following completion of each conference, presentation slides or posters, as applicable, will also be available to view online on the Investor section of the Company’s website.

(Press release, Krystal Biotech, APR 30, 2026, View Source [SID1234664979])

Rakovina Therapeutics Inc. Announces 2025 Financial Results and Provides Corporate Update

On April 30, 2026 Rakovina Therapeutics Inc. ("Rakovina" or the "Company") (TSX-V: RKV) (FSE: 7J0), a biopharmaceutical company advancing next-generation cancer therapies through artificial intelligence (AI)-powered drug discovery, reported its financial results for the fourth quarter and fiscal year ended December 31, 2025, and provided a corporate update.

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As Rakovina enters 2026, the Company is advancing its three primary drug discovery programs toward key value-creating milestones. For kt-5000, lead optimization continues with Variational AI, with lead candidate selection and IND-enabling studies targeted for the second half of 2026. For kt-3283, in vivo ADME and efficacy testing of the LNP formulation is underway with NanoPalm. For kt-2000, a second round of AI output and in vitro testing is advancing. Across all programs, the Company intends to progress pharmaceutical partnership discussions as preclinical data matures.

"Completing the first phase of our restructuring marks an important inflection point for Rakovina," said Kim Oishi, CEO of Rakovina. "Through the recent debenture restructuring, new financing, and the strengthening of our board and executive team, we are well on our way to repositioning the Company for success. As we continue to put our financial house in order, we are sharpening our focus on execution — advancing our DDR inhibitor pipeline, expanding our strategic partnerships, and unlocking the full potential of our world-class AI-driven drug discovery engine to deliver value for our shareholders."

2025 Highlights and Recent Developments

Leadership and Board

On January 27, 2026, Rakovina appointed Kim Oishi as Chief Executive Officer. Mr. Oishi brings over 25 years of capital markets experience, including as founder of Grand Rock Capital Inc. and partner at First Growth Equity Partners Inc.
On January 27, 2026, Rakovina welcomed Frank Holler to its Board of Directors. Mr. Holler has been directly involved in the founding and development of a number of Canada’s leading biopharma companies, including ID Biomedical Corp., Angiotech Pharmaceuticals Inc. and Xenon Pharmaceuticals Inc.
On May 5, 2025, Rakovina appointed Dr. David Kideckel, PhD, MBA as Chief Financial Officer. Dr. Kideckel brings over 20 years of life sciences and capital markets experience, with prior senior operational and finance positions at both public and private companies, including at Johnson & Johnson, Alexion Pharmaceuticals (acquired by AstraZeneca), and ATB Cormark Capital Markets.
On April 29, 2025, Rakovina appointed Yevgeniy Meshcherekov and Dr. David Kideckel to the Board, with Mr. Meshcherekov assuming Chair of the Audit Committee.
Financing and Capital Structure

On March 5, 2026, Rakovina closed a non-brokered private placement of convertible debenture units for gross proceeds of $1,000,000 and concurrently restructured $1,587,131 in existing debentures through a combination of share settlements (3,265,585 shares at $0.12) and replacement debentures, significantly strengthening the Company’s balance sheet.
On July 15, 2025, Rakovina common shares became eligible for electronic clearing and settlement through the Depository Trust Company (DTC), broadening access for U.S. investors.
On June 24, 2025, Rakovina completed a 10-to-1 share consolidation of its issued and outstanding common shares.
On June 6, 2025, Rakovina closed a non-brokered private placement of equity units for gross proceeds of $3,555,150, concurrent with a $1,350,000 convertible debenture financing.
On January 30, 2025, Rakovina listed its common shares on the Frankfurt Stock Exchange (FSE) under the ticker symbol "7J0", broadening access to European investors.
AI Platform Partnerships

On January 8, 2026, Rakovina expanded its collaboration with Variational AI and its Enki generative AI platform for continued lead optimization of the kt-5000 dual ATR-mTOR inhibitor program.
On August 12, 2025, Rakovina and NanoPalm Ltd. (Riyadh, Saudi Arabia) announced a non-binding Letter of Intent to form a joint venture to co-develop AI-discovered oncology therapies, beginning with kt-3283 delivered via NanoPalm’s patterned lipid nanoparticle (pLNP) system designed using its EnsaliX AI platform.
Scientific and Clinical Milestones

In April 2026, Rakovina presented two posters at the 2026 AACR (Free AACR Whitepaper) Annual Meeting in San Diego. For kt-5000, a prototype lead candidate demonstrated in vivo tumour growth inhibition comparable to ceralasertib in an LNCaP prostate model, with significantly improved tolerability and no signs of hematological toxicity. For kt-3283 LNP, successful characterization of the EnsaliX-designed formulation confirmed uniform particle size, stable colloidal behavior, and surface features predicted to enhance cellular uptake.
On November 24, 2025, Rakovina presented two posters at the Society for Neuro-Oncology (SNO) Annual Meeting, reporting CNS penetrance, metabolic stability, and in vivo tolerability for lead candidates in both the kt-5000 and kt-2000 programs.
On November 18, 2025, Rakovina announced that President & CSO Prof. Mads Daugaard was invited to present and participate as a panelist at the 9th Annual DNA Damage Response (DDR) Inhibitors Summit in January 2026.
On October 27, 2025, Rakovina presented pre-clinical data at the AACR (Free AACR Whitepaper)-NCI-EORTC International Conference confirming potent ATR inhibition and CNS penetration for the kt-5000 series – a milestone differentiator in the DDR inhibitor space.
On August 26, 2025, Rakovina announced that President & CSO Prof. Mads Daugaard was invited to speak at the 13th Tuscany Retreat on Cancer Research & Apoptosis (August 23-30), highlighting Rakovina’s DDR-targeted drug discovery and development accomplishments.
On April 28-29, 2025, Rakovina presented pre-clinical data on the kt-2000 and kt-5000 programs at the 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
The Company’s preclinical kt-3283 program continues to demonstrate superior cytotoxicity versus FDA-approved olaparib and vorinostat across multiple tumor types, as reported in a peer-reviewed publication in Clinical Cancer Research.
Pipeline Progress

On July 23, 2025, Rakovina announced that its kt-5000 program had identified potent ATR inhibitor hits during early-stage screening.
On March 12, 2025, Rakovina received the first synthesized batch of AI-generated ATR inhibitor compounds from its Variational AI collaboration.
On January 13, 2025, Rakovina achieved a shortlist of AI-generated CNS-penetrant ATR-targeting molecules, completing the initial AI-driven candidate generation phase for kt-5000.
On January 6, 2025, Rakovina received initial synthesized AI-generated PARP inhibitor compounds for in vitro and in vivo validation in the kt-2000 program.
Summary Financial Results for the Fourth Quarter and Year Ended December 31, 2025

All dollar amounts reflected in Canadian dollars unless otherwise stated.

At December 31, 2025, the Company had a working capital deficit of approximately $2,149,223 and cash and cash equivalents of $298,758. For the three- and twelve-months ended December 31, 2025, the Company reported a net loss of $1,893,159 and $8,680,576, respectively. Research and development expenses were $828,931 and $4,603,002; general and administrative expenses were $972,872 and $3,684,750 for the three- and twelve-months ended December 31, 2025, respectively. Total operating expenses for the three- and twelve-months ended December 31, 2025 were $1,801,803 and $8,287,752, respectively.

Subsequent to year-end, the Company strengthened its balance sheet through approximately $1.0 million in new convertible debenture financing and the restructuring of $1,587,131 in existing debentures, as further described in the Company’s audited financial statements and MD&A for the year ended December 31, 2025, filed on SEDAR+.

Selected Financial Information As at December 31, 2025 ($)
Cash & cash equivalents 298,758
Working capital (deficit) (2,149,223)
Intangible assets 3,439,896
Total Assets 4,240,407
Total liabilities 4,100,682
Deficit (23,678,505)
Total equity 139,725
Three months ended Dec 31, 2025 ($) Three months ended Dec 31, 2024 ($) Year ended Dec 31, 2025 ($) Year ended Dec 31, 2024 ($)
Expenses
Research and development 828,931 744,533 4,603,002 2,341,600
General and administrative 972,872 650,268 3,684,750 1,446,451
Net loss before other items 1,801,803 1,394,801 8,287,752 3,788,051
Other items
Interest income (773) – (797) (5,819)
Interest expense 146,338 45,793 274,596 182,177
Accretion expense (37,860) 23,291 132,100 86,363
Loss (gain) on settlement of debt – 18,815 – 18,815
Loss (gain) on modification of convertible debt (18,623) – (18,623) –
Foreign exchange loss 2,275 1,288 5,548 3,031
Total other expense (income) 91,357 89,187 392,824 284,567
Net loss and comprehensive loss (1,893,159) (1,483,988) (8,680,576) (4,072,618)
Rakovina Therapeutics’ financial statements as filed with SEDAR+ can be accessed from the Company’s website at: View Source

(Press release, Rakovina Therapeutics, APR 30, 2026, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-inc-announces-2025-financial-results-and-provides-corporate-update [SID1234664977])

Syndax Reports First Quarter 2026 Financial Results and Provides Business Update

On April 30, 2026 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported its financial results for the first quarter ended March 31, 2026, and provided a business update.

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"We delivered over $100 million in combined Revuforj and Niktimvo net sales in the first quarter, highlighting strong demand for our medicines and advancing the company towards profitability. Revuforj net revenue totaled $49 million, underscoring our leadership in menin inhibition and strong adoption in both R/R NPM1m AML and KMT2Ar acute leukemia. Notably, recent analysis indicates that Revuforj is enabling nearly half of KMT2A patients to receive a stem cell transplant, providing the best chance for durable remission and positioning the franchise for long-term growth as an increasing number of patients return to therapy post-transplant," said Michael A. Metzger, Chief Executive Officer. "We are poised for continued commercial growth with robust prescriber bases, excellent payer coverage, and multiple evolving treatment patterns that should extend the average duration of treatment for both medicines."

Mr. Metzger continued, "We are nearing multiple important catalysts this year, including new Revuforj data which will further highlight its best-in-class profile and topline data from Phase 2 trials of Niktimvo in frontline chronic GVHD and IPF. As we look ahead, we are focused on unlocking the multi-billion-dollar opportunities for our medicines and are well-positioned to be first to frontline AML with a menin inhibitor, with strong global site initiation and patient enrollment underway in our pivotal trials."

Recent Business Highlights and Anticipated Milestones

Revuforj (revumenib)

Achieved $48.9 million in Revuforj net revenue in the first quarter of 2026, representing a 144% increase over the first quarter of 2025 and an 11% increase over the fourth quarter of 2025, driven primarily by increasing uptake in relapsed or refractory (R/R) NPM1 mutated (NPM1m) acute myeloid leukemia (AML). Total prescriptions increased by approximately 160% compared to the first quarter of 2025 and approximately 13% compared to the fourth quarter of 2025. Notably, recent analysis indicates that nearly half of R/R KMT2A translocated patients are proceeding to a hematopoietic stem cell transplant (HSCT) after receiving Revuforj, a significant increase from prior estimates of 33%. The Company expects this growing transplant rate to extend the average treatment duration as an increasing number of patients return to therapy after transplant.
The Company expects the presentation of new revumenib data from multiple ongoing studies at major medical meetings throughout 2026.

New/updated data expected in the second quarter of 2026:

Findings from a multicenter real-world study.

Post-HSCT maintenance data from multiple trials and centers.

R/R NUP98r acute leukemia data from patients treated in the AUGMENT-101 trial or via an expanded access program.

R/R data from the SAVE trial of revumenib in combination with venetoclax and decitabine/cedazuridine in NPM1m, KMT2Ar, and NUP98r acute leukemia.

Frontline data from the Phase 1 trial of revumenib in combination with intensive chemotherapy in NPM1m, KMT2Ar, or NUP98r AML.
New/updated data expected in the second half of 2026:

Frontline data from the BEAT AML trial of revumenib in combination with venetoclax/azacitidine in NPM1m and KMT2Ar AML.

R/R data from the Phase 1 trial of revumenib in combination with gilteritinib in AML patients with a FLT3 mutation and a KMT2A translocation, NPM1m, or any other mutation associated with HOX-MEIS1 overexpression.
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 (MPAL) 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 1 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 after HSCT 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 measurable residual disease (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 second half 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.
Niktimvo (axatilimab-csfr)

Achieved $55.1 million in Niktimvo net revenue in the first quarter of 2026, representing significant growth compared to the $13.6 million in net revenue generated in the first quarter of 2025 from the first two months of the launch. 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 $15.9 million in the first quarter of 2026.
Presented data from nine axatilimab abstracts, including one oral presentation, at the Tandem Meetings (Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR) in February 2026. The data presented included a comprehensive analysis of axatilimab in patients with chronic graft-versus-host disease (GVHD)-related bronchiolitis obliterans syndrome (BOS) in two clinical studies. The results show clinical and symptom responses across a spectrum of lung involvement.
Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing, including:

A Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥ 12 years of age with newly diagnosed chronic GVHD. Topline data is now anticipated in the fourth quarter of 2026.
A pivotal Phase 3, randomized, double-blind, placebo-controlled, multicenter trial of axatilimab in combination with corticosteroids in patients ≥ 12 years of age with newly diagnosed chronic GVHD. Topline data is anticipated in early 2028.
Completed enrollment in MAXPIRe, a Phase 2, 26-week randomized, double-blinded, placebo-controlled trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF) in the first quarter of 2026. The Company expects to report topline data in the fourth quarter of 2026.
First Quarter 2026 Financial Results

As of March 31, 2026, Syndax had cash, cash equivalents, and short-term investments of $352.1 million and 88.8 million common shares and prefunded warrants outstanding.

Total revenue for the first quarter of 2026 was $64.9 million, which consisted of $48.9 million in Revuforj net revenue and $15.9 million in Niktimvo collaboration revenue. The Niktimvo collaboration revenue is derived from the $55.1 million in Niktimvo net revenue that was previously reported by the Company’s partner Incyte for the first 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.

First quarter 2026 research and development expenses decreased to $58.8 million from $61.6 million for the comparable prior year period. The year-over-year decrease was primarily due to a decrease in Niktimvo related development milestone expense recognized in the first quarter of 2025, offset by an increase in Revuforj related clinical trial and personnel expenses.

First quarter 2026 selling, general and administrative expenses decreased to $37.6 million from $41.0 million for the comparable prior year period. The year-over-year decrease was primarily due to a decrease in commercial-related expenses due to launch costs incurred in the first quarter of 2025 for Revuforj and Niktimvo that were not incurred in the same period in 2026 offset by a decrease in personnel expenses related to higher accrued compensation costs in 2025 for the achievement of corporate objectives.

For the three months ended March 31, 2026, Syndax reported a net loss attributable to common stockholders of $42.7 million, or $0.48 per share, compared to a net loss attributable to common stockholders of $84.8 million, or $0.98 per share, for the comparable prior year period.

Financial Guidance

For the full year of 2026, the Company expects 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 operating expense base will remain stable over the next couple of years. As a result, Syndax expects that its cash, cash equivalents and short-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, April 30, 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: Syndax1Q26
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 an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients one year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.

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.

Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.

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). Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

(Press release, Syndax, APR 30, 2026, View Source [SID1234664974])

OXC-101 for AML accepted to be presented at EHA

On April 30, 2026 Oxcia AB reported that the abstract "Interim results of the phase I/II study investigating karonudib in patients with refractory hematological malignancies paired with ex-vivo precision screen drug sensitivity screening" has been selected by the European Hematological Association (EHA) (Free EHA Whitepaper) Scientific Program Committee for a poster presentation during the EHA (Free EHA Whitepaper)2026 Congress. The congress takes place in Stockholm this year, June 11-14th. Stefan Deneberg, Principal Investigator of the clinical study and Austin Smith, Oxcia’s Chief Medical Officer, will present the interim results.

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The overall aim of the clinical study is to build on early encouraging signals of clinical activity and generate the data required to support a pivotal Phase II program in Acute Myeloid Leukemia. The on-going expansion group in the OXC-101 and idarubicin combination has expanded to Denmark and Serbia to facilitate timely recruitment.

OXC-101 (karonudib) has, as previously communicated, been granted ODD status by both the EMA and the FDA for Acute Myeloid Leukemia.

(Press release, Oxcia, APR 30, 2026, View Source;utm_medium=rss&utm_campaign=oxc-101-for-aml-accepted-to-be-presented-at-eha [SID1234664973])