On November 13, 2025 Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, reported the presentation of new data from 14 studies across company-sponsored and company-funded externally led studies at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, to be held Dec. 6-9, 2025, in Orlando, Florida.

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Taiho Oncology will share findings from clinical trials in two oral presentations and evidence from real-world studies in two online abstracts.

The oral presentations will highlight results from the Phase 2 part of ASTX030-01, a randomized, open-label, crossover trial comparing oral combination of azacitidine and cedazuridine to subcutaneous azacitidine and the Phase 2 portion of the Phase 1/2 trial ASTX727-03 of low-dose versus standard-dose oral decitabine and cedazuridine in patients with lower-risk myelodysplastic syndromes (MDS).
The two online posters will present real-world data on U.S. adults with acute myeloid leukemia (AML), describing treatment patterns and outcomes with hypomethylating agent (HMA) plus venetoclax, and evaluating the time burden of initiating parenteral HMA-based therapy in routine practice.
Additionally, 10 presentations – including four oral presentations – led by independent investigators will feature data from Taiho-funded investigator-initiated or collaborative studies. These presentations will highlight:

Results from a UK multicenter Phase 2 randomized trial comparing ASTX727 with hydroxycarbamide or best supportive care in advanced MDS/ myeloproliferative neoplasm (MPN).
Data from a Phase 2 study evaluating the all-oral combination of ASTX727, venetoclax and revumenib in newly diagnosed AML patients with NPM1m, KMT2Ar or NUP98r AML mutations.
Results of a Phase 1/2 clinical trial of the combination of oral decitabine/cedazuridine with the venetoclax in patients with untreated high-risk MDS and chronic myeloid leukemia (CMML).
Safety and efficacy results from a Phase 1/2 trial evaluating an all-oral combination of ASTX727, venetoclax and gilteritinib in both relapsed refractory and newly diagnosed FLT3-mutated AML, high-risk MDS or MPN.
"As the leader of oral HMA development, we’re excited that these data will be featured in six oral presentations at the ASH (Free ASH Whitepaper) Annual Meeting," said Harold Keer, MD, PhD, Taiho Oncology Chief Medical Officer. "These data highlight our increasing understanding of novel oral regimens in hematology. We are invested in developing treatments that have the potential to improve flexibility and lower the treatment burden for patients, and collaborating with leading institutions, evidenced by our continued support of independent clinical research to advance cancer care. We look forward to participating in the essential scientific exchange that contributes to progress in hematology at the ASH (Free ASH Whitepaper) Annual Meeting."

Data being presented by Taiho Oncology and its partners at the 2025 ASH (Free ASH Whitepaper) Annual Meeting and Exposition include:

Company-Sponsored Studies

Oral Presentations

Title: A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia
Abstract Number: 491
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Session Date: Dec. 7, 2025
Session Time: 9:30 a.m. to 11a.m.
Presentation Time: 10:15 to 10:30 a.m.
Location: OCCC – Valencia Room W415D
Presenter: Guillermo Garcia-Manero, MD

Title: Low-dose oral decitabine and cedazuridine among patients with low-risk myelodysplastic syndromes
Abstract Number: 790
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Lower risk Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms
Session Date: Dec. 8, 2025
Session Time: 10:30 a.m. to 12 p.m.
Presentation Time: 11:15 to 11:30 a.m.
Location: OCCC – Valencia Room W415A
Presenter: Guillermo Garcia-Manero, MD

Online Abstracts

Title: Time burden of treatment with parenteral HMAs in combination with venetoclax among patients with newly diagnosed AML
Abstract Number: 6990
Lead Author: Amer Zeidan, MD

Title: Real-World Characteristics, Treatment Modifications, and Outcomes for AML Patients Receiving HMAs + Venetoclax (1L)
Abstract Number: 8176
Lead Author: Amer Zeidan, MD

Company-Funded Externally Led Studies

Oral Presentations

Title: Phase II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE) in Newly Diagnosed AML
Abstract Number: 47
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens
Session Date: Dec. 6, 2025
Session Time: 9:30 to 11 a.m.
Presentation Time: 10:30 to 10:45 a.m.
Location: OCCC – Valencia Room W415A
Presenter: Wei-Ying Jen, BM BCh, FRCPath

Title: Oral decitabine/cedazuridine in combination with venetoclax in treatment-naïve high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia: Updates of a Phase 1/2 clinical trial
Abstract Number: 237
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes
Session Date: Dec. 6, 2025
Session Time: 2 to 3:30 p.m.
Presentation Time: 2:30 to 2:45 p.m.
Location: OCCC – Chapin Theater W415BC
Presenter: Alex Bataller, MD, PhD

Title: ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial
Abstract Number: 488
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML
Session Date: Dec. 7, 2025
Session Time: 9:30 to 11 a.m.
Presentation Time: 10:45 to 11 a.m.
Location: OCCC – Valencia Room W415D
Presenter: Daniel H. Wiseman, MB, ChB, PhD

Title: Phase I/II Study of Decitabine/Cedazuridine (ASXT727), Venetoclax, and Gilteritinib for Patients with FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
Abstract Number: 997
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens
Session Date: Dec. 8, 2025
Session Time: 4:30 to 6 p.m.
Presentation Time: 4:30 to 4:45 p.m.
Location: OCCC – Chapin Theater W320
Presenter: Roberta S. Azevedo, MD

Poster Presentations

Title: Translational Investigation of Tolinapant (ASTX660) in Acute Myeloid Leukemia Using Integrated Clinical, Bioinformatic, and Pharmacological Approaches
Abstract Number: 2552
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I
Session Date: Dec. 6, 2025
Session Time: 5:30 to 7:30 p.m.
Location: OCCC – West Halls B3-B4

Title: A Randomized Phase II Trial of ASTX727 and Venetoclax With or Without Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy (MM1OA-S03)
Abstract Number: 3466
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: Trial in progress: A Phase I Study Evaluating the Safety of cirtuvivint (CIRT) as Monotherapy and in Combination with ASTX727 in Patients with MDS and AML
Abstract Number: 3431
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: Trial in Progress – A Randomized Study of ASTX727 With or Without Ladademstat in Accelerated/Blast-Phase Myeloproliferative Neoplasms
Abstract Number: 3827
Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: A Comparison of Overall Survival and Quality of Life in MDS Patients Treated with Azacitidine vs Decitabine: A Propensity-Matched Registry Study
Abstract Number: 3862
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II
Session Date: Dec. 7, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

Title: A Multiarm Phase 1b Study of Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Plus Physician’s Choice of Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib in Acute Myeloid Leukemia
Abstract Number: 5228
Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster III
Session Date: Dec. 8, 2025
Session Time: 6 to 8 p.m.
Location: OCCC – West Halls B3-B4

(Press release, Taiho, NOV 13, 2025, View Source [SID1234659936])

On November 13, 2025 General Proximity, a biotechnology company discovering and developing the next generation of induced proximity medicines, reported a strategic multi-target collaboration with Daiichi Sankyo (TSE: 4568) through its Daiichi Sankyo Research Institute in Boston to apply General Proximity’s proprietary OmniTAC discovery platform to oncology programs of interest to Daiichi Sankyo.

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Induced proximity medicines enable the precise and selective modification of disease-driving proteins, creating therapeutic opportunities for traditionally undruggable targets. The approach uses small molecules to bring two proteins into close physical proximity and subsequently induce a desired therapeutic effect. The OmniTAC platform allows unbiased screening for effector proteins capable of modulating disease-relevant targets, offering a powerful and differentiated approach to drug discovery.

"Daiichi Sankyo is a leader in oncology research and development, and we are excited to work with such a strong collaborator," said Armand Cognetta, Founder & Chief Executive Officer of General Proximity. "This agreement represents an important validation of our novel induced proximity approach and highlights the potential of our OmniTAC discovery platform to deliver innovative solutions for patients."

Under the terms of the collaboration, General Proximity will leverage OmniTAC to discover and characterize novel effector-target pairs with the potential to generate first-in-class therapeutic candidates.

In parallel with this collaboration, General Proximity will continue to expand the reach of its OmniTAC platform by leveraging the precise control of proximity to address undruggable targets across multiple disease areas. In addition to oncology, the company is advancing programs in cardiometabolic disease, neurodegeneration, and longevity, underscoring its commitment to translating breakthrough science into transformative therapies for patients worldwide.

(Press release, General Proximity, NOV 13, 2025, https://www.prnewswire.com/news-releases/general-proximity-announces-multi-target-collaboration-with-daiichi-sankyo-to-discover-and-advance-novel-oncology-therapeutics-using-omnitac-platform-302612832.html [SID1234659935])

On November 13, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported the pricing of its previously announced underwritten public offering of 8,543,794 shares of its common stock at an offering price to the public of $7.25 per share, and in lieu of common stock to certain investors, pre-funded warrants to purchase up to an aggregate of 3,870,000 shares of its common stock at an offering price to the public of $7.2499 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.0001 per share exercise price for each such pre-funded warrant.

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The gross proceeds from the offering, before deducting underwriting discounts and commissions and other estimated offering expenses payable by Verastem Oncology, are expected to be approximately $90 million. In addition, Verastem Oncology has granted the underwriters a 30-day option to purchase up to an additional 1,862,069 shares of its common stock on the same terms and conditions. The offering is expected to close on or about November 17, 2025 subject to satisfaction of customary closing conditions. All of the securities to be sold in the offering are to be sold by Verastem Oncology.

Jefferies, Guggenheim Securities and Cantor are acting as joint book-running managers for the offering. H.C. Wainwright & Co. is acting as lead manager for the offering.

Verastem Oncology intends to use the net proceeds from the public offering to fund commercial activities for AVMAPKI FAKZYNJA CO-PACK that has been approved by the U.S. Food and Drug Administration to treat KRAS-mutated recurrent Low-Grade Serous Ovarian Cancer, continued clinical research and development of product candidates including the VS-7375 oral KRAS G12D (ON/OFF) inhibitor program, and for working capital and other general corporate purposes.

A shelf registration statement on Form S-3 relating to the public offering of the securities described above was declared effective by the Securities and Exchange Commission (SEC) on November 20, 2023. The offering is being made by means of a written prospectus and prospectus supplement that form a part of the registration statement. Before you invest, you should read the preliminary prospectus supplement relating to and describing the terms of the public offering, the accompanying base prospectus, and the related registration statement and other documents that Verastem Oncology has filed with the SEC for more complete information about Verastem Oncology and the offering. An electronic copy of the preliminary prospectus supplement and accompanying prospectus relating to the offering is free and can be found by visiting EDGAR on the SEC website at www.sec.gov. An electronic copy of the final prospectus supplement and accompanying prospectus relating to the offering will be available on the SEC website at www.sec.gov or may be obtained, when available, by contacting Jefferies LLC, by mail at Attn: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, or by telephone at (212) 518-9544, or by email at [email protected]; or Cantor Fitzgerald & Co., Attention: Equity Capital Markets, 110 E. 59th Street, 6th Floor, New York, NY 10022, or by email at [email protected].

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

(Press release, Verastem, NOV 13, 2025, View Source [SID1234659934])

On November 13, 2025 RenovoRx, Inc. (Nasdaq: RNXT) ("RenovoRx" or the "Company"), a life sciences company developing innovative targeted oncology therapies and commercializing RenovoCath, a novel, FDA-cleared drug-delivery device, reported its financial results and provided a business update to shareholders for the third quarter ended September 30, 2025.

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"We continue to make strong progress both in our commercial and clinical activities," said Shaun Bagai, CEO of RenovoRx. "On the commercial side, we are encouraged by the continued RenovoCath clinical adoption we are seeing in the field. Year-to-date revenue, through the end of the third quarter, was approximately $900,000, reflecting the growing clinical need and market demand for targeted drug-delivery solutions. Since organically launching commercial sales less than a year ago, we have expanded from five centers at the start of 2025 to 14 leading cancer centers now approved to purchase RenovoCath, including several that have already made repeat orders."

"With the recent hiring of Philip Stocton as Senior Director of Sales & Market Development and two regional sales managers, we are building the foundation for sustained growth while maintaining a lean operating structure and prudent capital deployment philosophy," continued Mr. Bagai. "Our team remains laser-focused on strategic, data-driven expansion and is encouraged by the increasing physician-to-physician advocacy for our TAMP (Trans-Arterial Micro-Perfusion) platform as we continue to position RenovoRx for long-term success. Our mission continues with the goal of integrating an approach to therapeutic drug-delivery into the standard of care and ultimately improving patient outcomes by offering a more targeted and tolerable treatment path."

"On the clinical side, we continue to advance our Phase III TIGeR-PaC trial towards enrollment completion in early 2026 and final data readout in 2027, and we’ve successfully launched a new post-marketing registry study from which we expect to garner real-world evidence on the use of the RenovoCath device. We look forward to continuing our progress in the Phase III trial and post-market registry study as we seek to drive value for our shareholders," concluded Mr. Bagai.

Commercialization Update

RenovoRx continued to execute on its RenovoCath commercialization strategy in the third quarter of 2025, demonstrating progress as year-to-date revenue grew to approximately $900,000. Although small revenue fluctuations can be expected quarter-by-quarter at this early stage of commercialization, the positive trends of expanding approved cancer center customers and increasing clinical adoption are very promising.

As of November 7, 2025, RenovoRx has expanded from five cancer center customers approved to purchase RenovoCath at the start of the year to 14 customers. These include additional high-volume National Cancer Institute (NCI)-designated cancer centers and community hospitals. The growth in patient procedures using RenovoCath at five active centers has led to an increase in repeat purchase orders, underscoring both physician satisfaction and growing demand. In addition to these 14 centers now approved to purchase RenovoCath, the Company has delivered product quotes to 10 additional leading centers across the nation, bringing the total to 24 centers who have formally requested quotes for RenovoCath. In addition, RenovoRx has engaged with dozens of physicians, both at various medical conferences and within their institutions, who have expressed commercial interest in utilizing RenovoCath for their patients.

Since organically launching commercial sales less than a year ago without a dedicated sales and marketing team, RenovoRx has achieved early commercial traction that reflects the strong clinical need for targeted drug-delivery solutions designed to reduce systemic toxicity and improve quality of life. The Company’s small but growing team has established a diverse domestic geographic network of academic institutions, NCI-designated cancer centers, and high-volume community hospitals, both successfully implementing and interested in utilizing RenovoCath-enabled TAMP technology.

RenovoRx continues to collect valuable market insights, including sales cycle trends, activation timelines, customer preferences, and other commercial data, as the Company seeks to grow its customer base, fulfill repeat RenovoCath orders, and build a strong foundation for long-term commercial growth. These insights from the first year of commercialization will inform the Company’s strategy and execution plans heading into 2026.

To strengthen and expand the Company’s commercial efforts, in August 2025, RenovoRx announced the hiring of Philip Stocton as Senior Director of Sales and Market Development. Mr. Stocton brings more than 25 years of MedTech leadership, including a decade focused on interventional oncology. His expertise has been invaluable as the Company continues to broaden its footprint across the U.S., while maintaining a lean operating structure. In alignment with the existing budget and to respond to growing demand, the Company has added two regional sales managers and plans to add a marketing director to drive additional physician engagement by the end of the year.

RenovoRx continues to estimate that the initial total addressable market (TAM) for RenovoCath as a stand-alone device represents an approximately $400 million peak annual U.S. sales opportunity, with long-term, several-billion-dollar potential as the platform expands into additional solid tumor indications.

RenovoRx recently launched a commercial website for clinicians and staff to learn more about the RenovoCath device, as well for patients to find treatment cancer centers, which can be accessed here: View Source

Clinical Research and Scientific Programs Update

In the third quarter of 2025, RenovoRx continued advancing its ongoing Phase III TIGeR-PaC clinical trial evaluating the novel drug-device combination oncology product candidate (intra-arterial gemcitabine delivered via RenovoCath, known as IAG). The trial remains on track, with enrollment expected to be completed in early 2026 and final data anticipated in 2027. Enabled by the RenovoCath device, TAMP is designed to deliver therapeutic agents across the arterial wall near the tumor site to bathe the target tumor, potentially optimizing local drug concentration while minimizing systemic exposure and related toxicities versus the standard of care, systemic intravenous therapy. TIGeR-PaC remains the cornerstone of RenovoRx’s clinical development program, validating its mechanism of action and safety profile through rigorous, long-term evaluation.

RenovoRx strengthened its Scientific Advisory Board during the third quarter with the additions of renowned surgeon and pancreatic cancer expert, Dr. Timothy Donahue, Director of the Agi Hirshberg Center for Pancreatic Diseases, Chief of the Division of Surgical Oncology at the David Geffen School of Medicine, and Garry Shandling Chair in Pancreatic Surgery at UCLA, and Dr. Thierry de Baère, Head of Interventional Radiology at the Gustave Roussy Cancer Centre and University Paris-Saclay in France.

During the third quarter, RenovoRx advanced its broader clinical programs. The Company’s post-marketing registry study continues to progress, generating real-world data on the safety and effectiveness of RenovoCath in patients with solid tumors. In September, the first registry-eligible patient procedure was initiated at the University of Vermont Cancer Center, with Baptist Health Miami Cancer Institute and the University of Pittsburgh Medical Center joining as additional study sites.

The Company also continues to support investigator-initiated trials in borderline resectable and oligometastatic pancreatic cancer. These capital-efficient studies are designed to be cost-neutral while providing meaningful data that may further broaden the application for the TAMP therapy platform.

Financial Highlights for the Third Quarter Ended September 30, 2025

Revenue: RenovoRx reported third quarter revenues of approximately $266,000, driven by both new customer orders and repeat purchases of the RenovoCath device. The quarter ended on September 30, 2025 marked the Company’s third full quarter of revenue generation from RenovoCath sales.

Cash Position: As of September 30, 2025, the Company had $10.0 million in cash and cash equivalents. Based on its current operating plan, RenovoRx believes this cash is sufficient to fund ongoing commercialization efforts for RenovoCath and the completion of enrollment in its Phase III TIGeR-PaC clinical trial in early 2026.

R&D Expenses: Research and development expenses were $1.7 million for the quarter ended September 30, 2025, remaining relatively unchanged from the same period in the prior year, reflecting continued investment in the TIGeR-PaC trial as well as support for investigator-initiated and registry studies.

SG&A Expenses: Selling, general, and administrative expenses were approximately $1.7 million for the quarter ended September 30, 2025, compared to $1.2 million for the same period in the prior year.

Net Loss: Net loss was $2.9 million for the quarter ended September 30, 2025, compared to a net loss of $2.5 million for the quarter ended September 30, 2024.

Shares Outstanding: As of November 7, 2025, common shares outstanding totaled approximately 36.6 million.

Conference Call Details

Event: RenovoRx Third Quarter 2025 Financial Results and Business Highlights Conference Call
Date: Thursday, November 13, 2025
Time: 4:30 p.m. ET
Live Call: 1-877-407-4018 (U.S. Toll Free) or 1-201-689-8471 (International)
Webcast: View Source

For interested individuals unable to join the conference call, a dial-in replay of the call will be available until November 27, 2025, and can be accessed by dialing 1-844-512-2921 (U.S. Toll Free) or 1-412-317-6671 (International) and entering replay pin number: 13756201.

A question-and-answer session will occur at the end of the call, and a link to the recording of this presentation will be available on RenovoRx’s Investor Relations website after the event.

(Press release, Renovorx, NOV 13, 2025, View Source [SID1234659930])

On November 13, 2025 Xilio Therapeutics, Inc. (Nasdaq: XLO), a clinical-stage biotechnology company discovering and developing tumor-activated immuno-oncology therapies for people living with cancer, reported pipeline progress and business updates and reported financial results for the third quarter ended September 30, 2025.

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"As we advance our robust pipeline of innovative masked immunotherapies, we continue to provide additional validation of our proprietary masking technology and ability to deliver differentiated molecules across a wide range of targets and design formats," said René Russo, Pharm.D., president and chief executive officer of Xilio. "At SITC (Free SITC Whitepaper), we presented compelling data across our clinical and preclinical programs, including data supporting the best-in-class potential of our masked T cell engager programs to meaningfully widen the therapeutic window relative to non-masked T cell engagers as well as our unique ability to incorporate co-stimulation to substantially improve the durability of T cell response."

Dr. Russo added, "For our clinical-stage programs, we continue to be encouraged by the promising data for both vilastobart and efarindodekin alfa, which have each shown differentiated clinical efficacy and safety for patients with high unmet need. In particular, new data for vilastobart leveraging plasma TMB as a predictive biomarker showed a 40% response rate in patients with MSS mCRC without liver metastases, supporting the significant opportunity for vilastobart as a combination therapy. As we look ahead to 2026, we are focused on execution across our clinical programs, while rapidly advancing XTX501, our bispecific PD-1/IL-2, toward a planned IND submission in mid 2026 and our masked T cell engager programs into IND-enabling studies."

Pipeline and Business Updates

Vilastobart: tumor-activated, Fc-enhanced anti-CTLA-4

Vilastobart is currently being evaluated in combination with atezolizumab (Tecentriq) in Phase 1C combination dose escalation in patients with advanced solid tumors and in a Phase 2 clinical trial in patients with microsatellite stable (MSS) metastatic colorectal cancer (mCRC).

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In November 2025, Xilio announced new late-breaking data from its ongoing Phase 2 clinical trial at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 40th Annual Meeting. These data demonstrated a 40% objective response rate (ORR) in heavily pre-treated, plasma tumor mutational burden (TMB) high patients (≥10 mutations/Mb) with MSS mCRC without liver metastases and showed a statistically significant correlation (p=0.05) between plasma TMB status and response. Approximately 55% of patients with MSS mCRC are estimated to have high plasma TMB, representing a meaningful patient population with high unmet need. For more information, read the press release here, and watch a replay of the webcast with leading colorectal cancer experts here.

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In November 2025, Xilio announced additional new Phase 2 data presented at SITC (Free SITC Whitepaper), which demonstrated the potential for circulating tumor DNA (ctDNA) as an early predictive biomarker for response to treatment with vilastobart in combination with atezolizumab in patients with MSS mCRC. For more information, read the press release here.

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Based on the promising clinical activity and safety profile demonstrated by vilastobart as a combination therapy, including in patients who had high plasma TMB, Xilio is actively seeking a partner to develop vilastobart in combination with PD-(L)1 or PD1-VEGF in MSS CRC and other tumor types.

Efarindodekin Alfa: tumor-activated IL-12

Xilio is evaluating efarindodekin alfa as a monotherapy in an ongoing Phase 1/2 clinical trial in patients with advanced solid tumors.

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In November 2025, Xilio presented Phase 1 monotherapy dose escalation data for efarindodekin alfa at SITC (Free SITC Whitepaper) demonstrating promising monotherapy anti-tumor activity in patients with advanced solid tumors as well as a generally well-tolerated safety profile at doses over 100-fold greater than the maximum tolerated dose of recombinant human IL-12. For more information, read the press release here.

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In September 2025, Xilio announced the selection of an initial recommended phase 2 dose (RP2D) and schedule for efarindodekin alfa and initiated patient dosing in the Phase 2 portion of the clinical trial. In connection with the initiation of Phase 2, Xilio achieved a development milestone of $17.5 million under its license agreement with Gilead Sciences, Inc. (Gilead). For more information, read the press release here.

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Xilio has completed enrollment in the Phase 1A monotherapy dose escalation and Phase 1B monotherapy dose expansion portions of the ongoing Phase 1/2 clinical trial, and evaluation of those patients is ongoing.

XTX501: bispecific PD-1 / masked IL-2

XTX501 is a novel, bispecific PD-1 / masked IL-2 designed to selectively stimulate PD-1 positive, antigen-experienced T cells and enhance their function. XTX501 incorporates masking designed to overcome IL-2 receptor-mediated clearance and peripheral activity. In preclinical studies, XTX501 demonstrated robust monotherapy activity (including in settings insensitive to PD-1) and tumor-selective pharmacodynamics consistent with its intended mechanism of action.

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XTX501 is currently advancing through investigational new drug (IND)-enabling studies, and Xilio plans to submit an IND application for XTX501 in the middle of 2026.

Masked T Cell Engager Programs

Xilio is leveraging its proprietary, clinically validated tumor-activation platform to advance multiple preclinical programs for masked T cell engagers, including wholly owned programs targeting tumor-associated antigens for PSMA (prostate cancer), CLDN18.2 (gastric, pancreatic, esophageal and lung cancers) and STEAP1 (prostate, colorectal and lung cancers), as well as an additional program in collaboration with AbbVie Group Holdings Limited (AbbVie).

Xilio’s masked T cell engager programs include bispecific molecules designed using its advanced tumor-activated cell engager (ATACR) format, which consists of a T cell engager with a masked CD3 targeting domain, and tri-specific molecules designed using its selective effector-enhanced cell engager (SEECR) format. The SEECR format builds upon the ATACR format by adding co-stimulatory signaling designed to further enhance potency and durability of T cell activation.

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In November 2025, Xilio presented new preclinical data at SITC (Free SITC Whitepaper) highlighting the potential for the company’s masking technology to significantly expand the therapeutic window for T cell engagers and overcome the challenges associated with current, systemically active non-masked T cell engagers. Xilio’s masked T cell engager molecules demonstrated potent anti-tumor activity with evidence of reduced systemic toxicity in murine models, supporting its broad applicability and potential best-in-class profile across a diverse range of targets, and the incorporation of co-stimulatory signaling in Xilio’s proprietary SEECR format enhanced durability of anti-tumor activity compared with T cell engager molecules that lacked co-stimulation. For more information, read the press release here.

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In the third quarter of 2025, Xilio nominated a development candidate for its PSMA program (ATACR format).

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Xilio anticipates nominating development candidates for its CLDN18.2 program (ATACR format) in the fourth quarter of 2025 and for its STEAP1 program (SEECR format) in the first half of 2026.

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Xilio anticipates advancing at least two of these programs into IND-enabling studies and submitting IND applications for those programs in 2027.

Third Quarter 2025 Financial Results

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Cash Position: Cash and cash equivalents were $103.8 million as of September 30, 2025, compared to $55.3 million as of December 31, 2024. The increase was primarily driven by $52.0 million in total upfront payments under the collaboration, license and option agreement and stock purchase agreement entered into in February 2025 with AbbVie and $47.0 million in net proceeds received from Xilio’s June 2025 follow-on public offering, partially offset by cash used for operating activities.

In the fourth quarter of 2025, Xilio received the $17.5 million development milestone payment under its license agreement with Gilead.
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Collaboration and License Revenue: Collaboration and license revenue was $19.1 million for the quarter ended September 30, 2025, compared to $2.3 million for the quarter ended September 30, 2024. Collaboration and license revenue for the quarter ended September 30, 2025 consisted of revenue recognized in connection with Xilio’s collaborations with AbbVie and Gilead, and collaboration and license revenue for the quarter ended September 30, 2024 consisted of revenue recognized in connection with Xilio’s collaboration with Gilead.

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Research & Development (R&D) Expenses: R&D expenses were $14.3 million for the quarter ended September 30, 2025, compared to $10.8 million for the quarter ended September 30, 2024. The increase was primarily driven by increased clinical development activities related to efarindodekin alfa, manufacturing activities related to IND-enabling studies and preclinical development activities for XTX501, increased costs related to early-stage programs and indirect research and development and increased personnel-related costs.

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General & Administrative (G&A) Expenses: G&A expenses were $6.7 million for the quarter ended September 30, 2025, compared to $6.3 million for the quarter ended September 30, 2024. The increase was primarily driven by an increase in professional and consulting fees, including legal fees and other professional costs, which were partially offset by a decrease in costs related to directors’ and officers’ liability insurance.

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Net Loss: Net loss was $16.3 million for the quarter ended September 30, 2025, compared to $14.0 million for the quarter ended September 30, 2024.

Financial Guidance

Based on its current operating plans, Xilio anticipates that its cash and cash equivalents as of September 30, 2025, together with the $17.5 million development milestone received under its license agreement with Gilead, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2027.

About Vilastobart

Vilastobart is an investigational tumor-activated, Fc-enhanced, high affinity binding anti-CTLA-4 monoclonal antibody designed to block CTLA-4 and deplete regulatory T cells when activated in the tumor microenvironment (TME). In 2023, Xilio entered into a co-funded clinical trial collaboration with Roche to evaluate vilastobart in combination with atezolizumab (Tecentriq) in a multi-center, open-label Phase 1/2 clinical trial. Xilio is currently evaluating the safety of the combination in Phase 1C dose escalation in patients with advanced solid tumors and the efficacy and safety of the combination in Phase 2 in patients with microsatellite stable (MSS) metastatic colorectal cancer (mCRC) with and without liver metastases. Please refer to NCT04896697 on www.clinicaltrials.gov for additional details.

About Efarindodekin Alfa

Efarindodekin alfa (XTX301) is an investigational tumor-activated IL-12 designed to potently stimulate anti-tumor immunity and reprogram the tumor microenvironment (TME) of poorly immunogenic "cold" tumors towards an inflamed or "hot" state. Xilio is currently evaluating the safety and tolerability of efarindodekin alfa as a monotherapy in patients with advanced solid tumors in the Phase 1 portion of a first-in-human, multi-center, open-label Phase 1/2 clinical trial and the safety and efficacy of efarindodekin alfa as a monotherapy in the Phase 2 portion in patients with advanced solid tumors. The Phase 2 portion of the trial is anticipated to enroll approximately 40 patients in specific tumor types at multiple sites in the United States. Please refer to NCT05684965 on www.clinicaltrials.gov for additional details.

In March 2024, Xilio entered into an exclusive global license agreement with Gilead to develop and commercialize efarindodekin alfa and specified other molecules directed to IL-12.Xilio is responsible for conducting clinical development for efarindodekin alfa through the initial Phase 2 portion of the ongoing Phase 1/2 clinical trial. Following the delivery by Xilio of a specified clinical data package for efarindodekin alfa related to the Phase 1/2 clinical trial, Gilead can elect to transition responsibilities for the development and commercialization of efarindodekin alfa to Gilead, subject to the terms of the license agreement and payment by Gilead of a $75.0 million transition fee. If Gilead exercises its option for efarindodekin alfa, Xilio will be eligible to receive up to $500.0 million in specified development, regulatory and sales-based milestones and will be eligible to receive tiered royalties ranging from high single digits to mid-teens on annual global net product sales.

(Press release, Xilio Therapeutics, NOV 13, 2025, View Source [SID1234659919])