On May 14, 2025 Theriva Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Theriva Biologics, MAY 14, 2025, View Source [SID1234653088]).

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"We have started 2025 with outstanding clinical progress," said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. "The VIRAGE Phase 2b clinical trial of VCN-01 (zabilugene almadenorepvec) with gemcitabine/nab-paclitaxel in newly diagnosed metastatic pancreatic cancer patients achieved its primary survival and safety endpoints, highlighting the potential therapeutic benefits of our oncolytic virus platform. We are working to scale up manufacturing and finalize the design of a Phase 3 trial of VCN-01 with gemcitabine/nab-paclitaxel which if successful, may allow us to deliver this innovative treatment option to patients suffering this fatal disease."

Recent Highlights and Anticipated Milestones

VCN-01

Metastatic Pancreatic Ductal Adenocarcinoma (mPDAC):

As recently announced, mPDAC patients treated with VCN-01 (zabilugene almadenorepvec) plus gemcitabine/nab-paclitaxel standard-of-care (SoC) chemotherapy had increased overall survival (OS), progression free survival (PFS), and duration of response (DOR) compared to patients treated with gemcitabine/nab-paclitaxel SoC.
VCN-01 was well-tolerated, with transient and reversible adverse events (AEs).
The increase in OS was greater for patients who received 2 doses of VCN-01 and 4 or more cycles of gemcitabine/nab-paclitaxel compared with patients who received 4 or more cycles of gemcitabine/nab-paclitaxel SoC alone, suggesting that the second dose of VCN-01 (administered 3 months after the first dose) provides a meaningful additional benefit in this treatment subgroup.
Theriva had hosted a virtual event featuring feature eminent pancreatic cancer clinician/researchers to review and discuss the data from the VIRAGE trial of VCN-01. To access the replay of the event, click HERE.
The Company is currently working to scale up manufacturing of VCN-01 and finalizing the design of a potential Phase 3 confirmatory trial for VCN-01 in mPDAC.
SYN-004

Allogeneic hematopoietic cell transplant (HCT):

As recently announced, data from a Phase 1b/2a trial investigating SYN-004 (ribaxamase) in allogeneic hematopoietic cell transplant (HCT) recipients for the prevention of acute graft-versus-host-disease (aGVHD) was presented at the Congress of the European Society of Clinical Microbiology and Infectious Diseases (ESCMID Global) in April.
Corporate Updates

As recently announced, Theriva closed a public offering of 6,818,180 shares of common stock (or pre-funded warrants in lieu thereof) and warrants to purchase up to 6,818,180 shares of common stock at a combined offering price of $1.10 per share and accompanying warrant (the "Offering"). The Company received aggregate gross proceeds of approximately $7.5 million, before deducting placement agent fees and other offering expenses. The warrants have an exercise price of $1.10 per share, are exercisable immediately and expire five years from the issuance date.
The Company intends to use the net proceeds from the Offering primarily for working capital and general corporate purposes, including for research and development, and manufacturing scale-up of VCN-01 for a potential Phase 3 clinical trial.
First Quarter Ended March 31, 2025 Financial Results

General and administrative expenses decreased to $1.4 million for the three months ended March 31, 2025, from $1.9 million for the three months ended March 31, 2024. This decrease of 25% is primarily comprised of the decrease in salary costs, travel, lower director and officer insurance, and a decrease in fair value of the contingent consideration adjustment. The charge related to stock-based compensation expense was $54,000 for the three months ended March 31, 2025, compared to $101,000 for the three months ended March 31, 2024.

Research and development expenses decreased to $3.0 million for the three months ended March 31, 2025, from approximately $3.5 million for the three months ended March 31, 2024. This decrease of 14% is primarily the result of lower indirect cost related to decreased VCN-01 manufacturing costs and lower clinical trial expenses related to our Phase 1b/2a clinical trial of SYN-004 (ribaxamase) in allogeneic HCT recipients, offset by slightly higher clinical trial expenses related to our VIRAGE Phase 2b clinical trial of VCN-01 in PDAC and higher patent expenses related to SYN-020. We anticipate research and development expense to increase as we complete our VIRAGE Phase 2b clinical trial of VCN-01 and plan for our Phase 3 clinical trial of VCN-01 in PDAC, advance our VCN-01 program in retinoblastoma, expand GMP scale-up manufacturing activities for VCN-01, and continue supporting our other preclinical and discovery initiatives. The charge related to stock-based compensation expense was $46,000 for the three months ended March 31, 2025, compared to $58,000 related to stock-based compensation expense for the three months ended March 31, 2024.

Other income was $93,000 for the three months ended March 31, 2025 compared to other income of $227,000 for the three months ended March 31, 2024. Other income for the three months ended March 31, 2025 is primarily comprised of interest income of $96,000 and an exchange loss of $3,000. Other income for the three months ended March 31, 2024 is primarily comprised of interest income of $228,000 and exchange loss of $1,000.

Cash and cash equivalents totaled $10 million as of March 31, 2025, compared to $11.6 million as of December 31, 2024. Subsequent to closing of the public offering on May 8 2025, the Company’s cash balance was $14.1 million.

About Pancreatic Ductal Adenocarcinoma

Cancer of the pancreas consists of two main histological types: cancer that arises from the ductal (exocrine) cells of the pancreas or, much less often, cancers may arise from the endocrine compartment of the pancreas. Pancreatic ductal adenocarcinoma ("PDAC") accounts for more than 90% of all pancreatic tumors. It can be located either in the head of the pancreas or in the body/tail. Pancreatic cancer usually metastasizes to the liver and peritoneum. Other less common metastatic sites are the lungs, brain, kidney and bone. In its early stages, pancreatic cancer does not typically result in any characteristic symptoms, so in most cases it is diagnosed in its late stages (locally advanced non-metastatic or metastatic disease) when surgical resection and possibly curative treatment is not possible. It is generally assumed that only 10% of cases are resectable at presentation, whereas 30-40% of patients are diagnosed at local advanced/unresectable stage and 50-60% present with distant metastases.

About VIRAGE

VIRAGE was a two-arm, Phase 2b open-label, randomized, controlled, multicenter clinical trial in patients with histologically confirmed, newly-diagnosed metastatic PDAC. Patients were enrolled at 5 sites in the U.S. and 9 sites in Spain. In both the control and VCN-01 (zabilugene almadenorepvec) treatment arms, patients received gemcitabine/nab-paclitaxel standard-of-care chemotherapy in repeated 28-day cycles until disease progression. In the VCN-01 treatment arm only, patients were also administered intravenous VCN-01 seven-days prior to starting the first and fourth cycles of gemcitabine/nab-paclitaxel treatment (study days 1 and ~92 respectively). Primary endpoints for the trial include overall survival and VCN-01 safety/tolerability. Additional endpoints include progression free survival, duration of response, and measures of VCN-01 biodistribution, replication, and immune response. More information about the trial is available on Clinicaltrials.gov (NCT05673811), through the Spanish Clinical Trials Registry and European Union Drug Regulating Authorities Clinical Trials Database (EudraCT Number: 2022-000897-24).

About VCN-01

VCN-01 (zabilugene almadenorepvec) is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to 142 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov.

On May 14, 2025 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported the online publication of three abstracts submitted to the European Hematology Association (EHA) (Free EHA Whitepaper) Congress, to be held June 12-15, 2025, Milan, Italy (Press release, Autolus, MAY 14, 2025, View Source [SID1234653087]). Autolus will have two oral and one poster presentation, which includes updated follow up from the FELIX study of obecabtagene autoleucel (obe-cel) in adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL).

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Oral S113:
Title: Can CAR T-cell therapy be a definitive treatment for adult r/r B-ALL without transplant? Long-term findings and predictors of sustained remission for obecabtagene autoleucel
Session Name: s447 Immunotherapy and CAR-T cells for ALL
Session room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Jae H Park, MD

Summary: Obe-cel persistence, low disease burden at lymphodepletion and obe-cel use in earlier lines were independent factors associated with better outcomes and longer survival in adult pts with r/r B-ALL. At the current follow-up comprising patients studied for ≥3 years, 40% of responders are in ongoing remission without subsequent stem cell therapy or other new therapies, suggesting the potential of obe-cel as a definitive treatment for adult r/r B-ALL. An updated analysis with additional follow-up is underway and will be presented.

Oral S114:
Title: Efficacy and Safety Outcomes of Obecabtagene Autoleucel (obe-cel) Stratified by Age in Patients with r/r B-ALL
Session Name: Immunotherapy and CAR-T cells for ALL
Session Room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Bijal D. Shah, MD

Summary: Obe-cel treatment was associated with deep and durable remissions resulting in favorable overall remission rate, event free survival, and overall survival with low incidence of Grade ≥3 CRS and ICANS in both age groups (<55 years and ≥55 years). These findings indicate that obe-cel is effective and has a positive benefit and risk profile regardless of patient age, including in older adults with R/R B-ALL, despite few patients receiving consolidative stem cell therapy.

Poster PF378:
Title: Predicting Hematotoxicity Risk and Outcomes in Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-All): Should Hematotox Models be CAR Specific rather than Disease Specific
Session Title: Poster Session 1
Session date and time: Friday, June 13; 18:30 – 19:30 CEST.
Presenting Author: Claire Roddie, MD

Summary: Although both the CAR-Hematotox (CAR-HT) model, and the ALL-Hematotox (ALL-HT) model show potential, ALL-HT appears to improve risk stratification and may be a better predictor of response, survival and safety outcomes in adult patients with r/r B-ALL treated with obe-cel, than CAR-HT. Taken together with other published reports, our data suggest that the strength of HT-model predictions may be CAR specific. Further analyses are needed.

On May 14, 2025 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company developing a potentially first-in-class and best-in-class WEE1 inhibitor for patients with ovarian cancer and other tumor types, reported financial results for the first quarter 2025 and highlighted recent operational progress (Press release, Zentalis Pharmaceuticals, MAY 14, 2025, View Source [SID1234653082]).

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"We continued advancement of azenosertib and made solid progress against our strategic goals in the first quarter. The initiation of DENALI Part 2 moves us into a new chapter as a late-stage development company focusing on a significant patient population in platinum-resistant ovarian cancer with limited choices." said Julie Eastland, Chief Executive Officer of Zentalis. "Our team remains focused on the enrollment and execution of DENALI. Zentalis is financially and organizationally well-positioned to continue advancing azenosertib as a potential treatment option for PROC patients."

Program Highlights

•First patient dosed in Part 2 of the Phase 2 DENALI clinical trial.
◦In April 2025, the Company announced that the first patient had been dosed in Part 2a of the Phase 2 DENALI clinical trial (NCT05128825) of azenosertib in patients with Cyclin E1+ platinum-resistant ovarian cancer (PROC).
◦Part 2a is designed to confirm the primary dose-of-interest with a target enrollment of approximately 30 patients at each of two dose levels: 400mg QD 5:2 and 300mg QD 5:2 (intermittent daily dosing with a five days on, two days off dosing schedule).
◦The Company expects to disclose topline data from DENALI Part 2 (Part 2a and Part 2b) by year end 2026. DENALI Part 2, if successful, has the potential to support an accelerated approval, subject to FDA review.

•Azenosertib clinical data demonstrated clinically meaningful response rates and a consistent therapeutic profile.
◦Previously disclosed clinical data from Part 1b of the DENALI study showed clinically meaningful results in patients with Cyclin E1+ PROC. As of the January 13, 2025 data cutoff, patients who were response-evaluable (n=43) had an objective response rate (ORR) of 34.9% and a median duration of response (mDOR) of 6.3 months. The mDOR is subject to change as there were patients with ongoing responses as of the cutoff date.
◦Across multiple monotherapy studies of azenosertib with significant sample sizes, as of the December 2, 2024 data cutoff, azenosertib demonstrated meaningful antitumor activity (ORR >30% at 400mg QD 5:2) and a manageable safety profile in Cyclin E1+ PROC patients.

Upcoming Events

•Zentalis plans to participate in the following scientific and medical conference:
◦American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting – May 30 to June 3 in Chicago, IL
◦NRG Oncology Summer Meeting – July 24 to 26 in Washington, D.C.

•Zentalis plans to participate and present in the following investor events:
◦H.C. Wainwright 3rd Annual BioConnect Investor Conference – May 20 in New York, NY
◦TD Cowen’s 6th Annual Oncology Innovation Summit – May 27 Virtual
◦Jefferies Healthcare Conference – June 4 in New York, NY

First Quarter 2025 Financial Results

•Cash, Cash Equivalents and Marketable Securities Position: As of March 31, 2025, the Company had cash, cash equivalents and marketable securities of $332.5 million, which includes $12.2 million representing the March 31, 2025 fair value of Immunome common stock received by the Company from the sale of its ROR1 antibody-drug conjugate (ADC) product candidate and ADC platform to Immunome in October 2024. The Company believes that its existing cash, cash equivalents and marketable securities as of March 31, 2025 will be sufficient to fund its operating expenses and capital expenditure requirements into late 2027.

•Research and Development Expenses: Research and development expenses for the three months ended March 31, 2025 were $27.2 million, compared to $49.6 million for the three months ended March 31, 2024. The decrease of $22.4 million was primarily due to decreases of $10.7 million for clinical expenses, $4.8 million for drug manufacturing, $3.5 million for lab services and $5.1 million related to consulting and personnel expenses including non-cash stock-based compensation. These decreases were partially offset by a one-time impairment charge of $1.2 million related to the disposal of research and development equipment and an increase of $0.5 million of allocated overhead.

•General and Administrative Expenses: General and administrative expenses for the three months ended March 31, 2025 were $10.6 million, compared to $15.7 million during the three months ended March 31, 2024. This decrease of $5.1 million was primarily attributable to non-cash stock-based compensation.

•Operating Expenses: Total operating expenses were $45.6 million for the three months ended March 31, 2025, compared to $65.3 million for the three months ended March 31, 2024. Total operating expenses for the first quarter of 2025 include the non-recurring restructuring expenses of $7.8 million associated with the strategic restructuring announced in January 2025.

About Azenosertib

Azenosertib is a novel, selective, and orally bioavailable inhibitor of WEE1 currently being evaluated as a monotherapy and combination clinical studies in ovarian cancer and additional tumor types. WEE1 acts as a master regulator of the G1-S and G2-M cell cycle checkpoints, through negative regulation of both CDK1 and CDK2, to prevent replication of cells with damaged DNA. By inhibiting WEE1, azenosertib enables cell cycle progression, despite high levels of DNA damage, thereby resulting in the accumulation of DNA damage and leading to mitotic catastrophe and cancer cell death.

About DENALI Clinical Trial

DENALI is a multi-part Phase 2 clinical trial studying azenosertib in platinum-resistant ovarian cancer (PROC) patients. Part 1b enrolled patients with PROC regardless of Cyclin E1 protein expression, all treated at 400mg 5:2(intermittent daily dosing with a five days on, two days off dosing schedule). Interim results from Part 1b were presented at the Society of Gynecologic Oncology (SGO) 2025 Annual Meeting. Part 2 is ongoing and is enrolling PROC patients with Cyclin E1 protein overexpression based on Zentalis’ proprietary immunohistochemistry cutoff. Part 2 includes Part 2a, a dose confirmation portion evaluating two doses, 300mg 5:2 and 400mg 5:2, and Part 2b, a portion designed to complete enrollment at the selected dose. Part 2, in total, is designed for accelerated approval, pending study outcome and discussions with the U.S. Food and Drug Administration.

On May 14, 2025 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immuno-oncology technologies addressing difficult to treat cancers, reported its financial results for the quarter ended March 31, 2025 (Press release, Xenetic Biosciences, MAY 14, 2025, View Source [SID1234653081]).

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Recent Highlights

Announced that its collaboration partner, PeriNess Ltd., entered into a Clinical Study Agreement to advance development of DNase platform for the treatment of relapsed/refractory osteosarcoma and Ewing sarcoma; and

Continued pursuit of other strategic collaborations to advance the Company’s technology.

"We remain focused on engaging with our strategic partners to participate in a series of exploratory studies to evaluate our systemic DNase I in combination with immunotherapy, chemotherapy, and radiotherapy in various oncology indications where there remains significant unmet need to advance our development programs forward. These partnerships allow us to advance our technology toward the clinic while utilizing our resources efficiently and minimizing our internal investment. Additionally, this development strategy opens up valuable opportunities to continue expanding our growing body of positive preclinical data that supports the use of DNase I across several cancer indications," commented James Parslow, Interim Chief Executive Officer and Chief Financial Officer of Xenetic.

Xenetic continues to advance its DNase-based technology towards Phase 1 clinical development for the treatment of pancreatic carcinoma and other locally advanced or metastatic solid tumors. Preliminary preclinical studies evaluating the combinations of DNase I with chemotherapy and DNase I with immuno-therapies in colorectal cancer models as well as CAR-T therapy have been completed.

Additionally, as previously announced in December 2024, Xenetic entered into a Clinical Trial Services Agreement with PeriNess, under which PeriNess will lead in the regulatory approval, operational execution and management of potential exploratory, investigator initiated studies of recombinant DNase as an adjunctive treatment in patients with pancreatic carcinoma and other locally advanced or metastatic solid tumors receiving chemotherapy and immunotherapy in Israeli medical centers.

Summary of Financial Results for First Quarter 2025

Net loss for the quarter ended March 31, 2025 was approximately $0.9 million. Revenue increased by approximately $0.1 million, or 16.1%, to approximately $0.6 million during the three months ended March 31, 2025 from approximately $0.5 million in the comparable quarter in 2024. Total operating costs and expenses for the three months ended March 31, 2025 decreased by approximately $244,000, or 13.7%, to approximately $1.5 million from approximately $1.8 million in the comparable quarter in 2024. The decrease was primarily due to a decrease in personnel costs and share-based expense related to the departures of the Company’s former Chief Executive Officer and Chief Scientific Officer during the second quarter of 2024.

The Company ended the quarter with approximately $5.2 million of cash.

On May 14, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, reported that multiple abstracts showcasing clinical data for Revuforj (revumenib) and Niktimvo (axatilimab-csfr) were accepted for presentation at the 30th European Hematology Association (EHA) (Free EHA Whitepaper) Annual Congress Meeting being held June 12-15, 2025, in Milan, Italy (Press release, Syndax, MAY 14, 2025, View Source [SID1234653080]).

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"The data being presented at EHA (Free EHA Whitepaper) showcase the potential for revumenib and axatilimab to transform the treatment paradigm for patients with acute leukemia and chronic GVHD, respectively," said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. "We and our clinical collaborators are particularly pleased to share updated data from the BEAT AML frontline trial of revumenib in combination with venetoclax and azacitidine in mNPM1 and KMT2Ar AML. We anticipate that the oral presentation of these encouraging results will drive even greater physician interest in the ongoing EVOLVE-2 frontline global registration trial of this same combination."

Key data being presented at EHA (Free EHA Whitepaper) 2025 include:

An oral presentation reporting updated results from the Phase 1 BEAT AML trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mutant NPM1 (mNPM1) or KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) patients aged 60 years or older.
A poster presentation reporting data from the 77 patients with relapsed or refractory (R/R) mNPM1 AML who met the efficacy evaluable criteria in the Phase 2 cohort of the AUGMENT-101 trial of revumenib. The poster will expand upon the topline data that was reported from this population in December 2024.
A poster presentation reporting outcomes in patients with R/R NUP98r acute leukemias in the Phase 1 portion of the AUGMENT-101 trial.
A poster presentation reporting the dynamics of overall and organ-specific responses in the AGAVE-201 trial of axatilimab in chronic graft-versus-host disease (GVHD).
The accepted abstracts listed below are now available online at the EHA (Free EHA Whitepaper) conference website. Copies of the oral and poster presentations will be made available in the ‘Publications & Meetings Presentations’ section of the Syndax website after the presentations occur.

Presentations During EHA (Free EHA Whitepaper) 2025 (all times in CEST):

Revumenib

Abstract Titles Presentation Details
Azacitidine, Venetoclax, and Revumenib for Newly Diagnosed Older Adults with Acute Myeloid Leukemia (AML) and NPM1 mutation or KMT2A rearrangement: Updated Results from the Beat AML Consortium Oral presentation
Abstract #: S138
Thursday, June 12
Session 5:00-6:15 pm
Patients With Relapsed or Refractory (R/R) Nucleophosmin 1–Mutated (NPM1m) Acute Myeloid Leukemia (AML): Updated Results From the Phase 2 AUGMENT-101 Study Poster presentation (Session 2)
Abstract #: PS1467
Saturday, June 14 at 6:30-7:30 pm
Updated Results and Longer Follow-Up From the AUGMENT-101 Phase 2 Study of Revumenib in All Patients With Relapsed or Refractory (R/R) KMT2Ar Acute Leukemia Poster presentation (Session 2)
Abstract #: PS1473
Saturday, June 14 at 6:30-7:30 pm
Encore presentation
Revumenib Activity in Patients with NUP98r Acute Leukemia: Results from the AUGMENT-101 Phase 1 Study Poster presentation (Session 2)
Abstract #: PS1501
Saturday, June 14 at 6:30-7:30 pm
Trial in Progress: A Phase 1 Study of Revumenib + Intensive Chemotherapy in Patients With Newly Diagnosed Acute Myeloid Leukemia Harboring Genetic Alterations in KMT2A, NPM1, or NUP98 (SNDX-5613-0708) Publication only*
Abstract: PB2576
Treatment Patterns in Patients with Relapsed or Refractory NPM1 Mutated (NPM1m) Acute Myeloid Leukemia (AML) in the United States (US): Real-world Data Analysis Publication only*
Abstract: PB2513
* Abstracts will be published in the online Abstract Book, a supplement of HemaSphere (EHA’s official journal), EHA (Free EHA Whitepaper) Library and Congress platform.

Axatilimab

Abstract Titles Presentation Details
The Effects of Prior Lines of Therapy on Clinical Outcomes for Patients With Chronic Graft-Versus-Host Disease Receiving Axatilimab: A Post Hoc Analysis of AGAVE-201 Poster presentation (Session 2)
Abstract #: PS2029
Saturday, June 14 at 6:30-7:30 pm
Encore presentation
Dynamics of Overall and Organ-Specific Responses to Axatilimab in Chronic Graft-Versus-Host Disease: Analysis From the AGAVE-201 Study Poster presentation (Session 1)
Abstract #: PF1035
Friday, June 13 at 6:30-7:30 pm
Encore presentation
Trial in Progress: A Randomized, Open-Label, Phase 3 Study of Axatilimab Versus Best Available Therapy in Patients With Chronic Graft-Versus-Host Disease After ≥2 Prior Lines of Systemic Therapy Poster presentation (Session 1)
Abstract #: PF1090
Friday, June 13 at 6:30-7:30 pm
Correlations of Clinician-Reported Responses With Other Response Measures in Patients With Chronic Graft-Versus-Host Disease: An Analysis From the AGAVE-201 Trial Poster presentation (Session 1)
Abstract #: PF1041
Friday, June 13 at 6:30-7:30 pm
Encore presentation

About Revuforj (revumenib)

Revuforj (revumenib) is an oral, first-in-class, selective 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 in adult and pediatric patients one year and older.

Revumenib is in development for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial in this population with revumenib as a monotherapy were recently published and the Company submitted a supplemental NDA for revumenib in R/R mNPM1 AML in April 2025. Additionally, multiple trials of revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia are ongoing or planned across the treatment landscape, including in newly diagnosed patients.

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 – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

Niktimvo is a trademark of Incyte.

All other trademarks are the property of their respective owners.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, and/or hypotension. In clinical trials, DS occurred in 39 (29%) of 135 patients treated with Revuforj. DS was Grade 3 or 4 in 13% of patients and fatal in one. The median time to onset was 10 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10-mg IV every 12 hours in adults or dexamethasone 0.25-mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc interval prolongation: In the clinical trials, QTc interval prolongation was reported as an adverse reaction in 39 (29%) of 135 patients treated with Revuforj. QTc interval prolongation was Grade 3 in 12% of patients. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 8%, and the increase from baseline QTcF was greater than 60 msec in 18%. Revuforj dose reduction was required for 5% of patients due to QTc interval prolongation. QTc prolongation occurred in 16% of the 31 patients less than 17 years old, 33% of the 88 patients 17 years to less than 65 years old, and in 50% of the 16 patients 65 years or older.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.
Embryo-fetal toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 4 (3%) patients who received Revuforj, including 2 with differentiation syndrome, 1 with hemorrhage, and 1 with sudden death.

Serious adverse reactions were reported in 99 (73%) patients. The most frequent serious adverse reactions (≥5%) were infection (24%), febrile neutropenia (19%), bacterial infection (17%), differentiation syndrome (12%), hemorrhage (9%), and thrombosis (5%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were hemorrhage (53%), nausea (51%), phosphate increased (50%), musculoskeletal pain (42%), infection (41%), aspartate aminotransferase increased (37%), febrile neutropenia (35%), alanine aminotransferase increased (33%), parathyroid hormone intact increased (33%), bacterial infection (31%), diarrhea (30%), differentiation syndrome (29%), electrocardiogram QT prolonged (29%), phosphate decreased (25%), triglycerides increased (25%), potassium decreased (24%), decreased appetite (24%), constipation (23%), edema (23%), viral infection (23%), fatigue (22%), and alkaline phosphatase increased (21%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

Strong CYP3A4 inhibitors: reduce Revuforj dose
Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec.
SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNING.

Niktimvo (axatilimab-csfr)

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS
Infusion-Related Reactions
Niktimvo (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.

Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.

Embryo-Fetal Toxicity
Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.

ADVERSE REACTIONS
Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in >2 patients included infection (pathogen unspecified) (14%), viral infection (14%) and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain, and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea.

Clinically relevant adverse reactions in <10% of patients who received Niktimvo included:

Eye disorders: periorbital edema
Skin and subcutaneous skin disorders: pruritus
Vascular disorders: hypertension
Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions
Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS
Lactation
Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential
Pregnancy Testing
Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception
Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION
Dosage Modifications for Adverse Reactions
Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

Please see the full Prescribing Information for Niktimvo.