On August 14, 2025 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, reported its 2025 Interim Results (Press release, Carsgen Therapeutics, AUG 14, 2025, View Source [SID1234655321]).

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Business Highlights

Cash and bank balances were around RMB1,261 million as of June 30, 2025. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.
During H1 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities. CARsgen has received a total of 111 confirmed orders from its commercialization partner Huadong Medicine.
The Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China has accepted the New Drug Application (NDA) for satri-cel.
The results of satri-cel confirmatory Phase II trial in China have been published in The Lancet and at the 2025 ASCO (Free ASCO Whitepaper) Annual Meeting.
Multiple allogeneic CAR-T products are in development, covering treatment areas such as hematologic malignancies, solid tumors, and autoimmune diseases.
CARsgen introduced Zhuhai SB Xinchuang to accelerate allogeneic CAR-T development in mainland China.
Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics, said, "In the first half of 2025, we made significant strides across technology innovation, product development, and commercialization. Zevor-cel sales surged, while satri-cel became the world’s first CAR-T targeting solid tumors to file an NDA. We are also advancing multiple allogeneic CAR-T therapies to enhance clinical outcomes and expand patient access."

Financial Highlights

CARsgen’s revenue was around RMB51 million for the six months ended June 30, 2025, mainly from zevor-cel, which was calculated on the basis of ex-works price, rather than end-of-market prices. Our revenue is recognized upon completion of ex-works delivery of products. Due to the inherent time cycle of CAR-T manufacturing, there is a discrepancy between the number of orders obtained from Huadong Medicine and number of ex-works deliveries. CARsgen’s gross profit was around RMB29 million for the six months ended June 30, 2025. In the commercialization stage, we are demonstrating a strong cost competitive advantage, which is mainly due to self-manufacture for plasmids and vectors with stable output and high yield per batch.

Cash and bank balances were around RMB1,261 million as of June 30, 2025, representing a decrease of around RMB218 million from around RMB1,479 million as of December 31, 2024. The decrease was mainly due to the payment of research and development expenses, administrative expenses and investment of capital expenditure. Cash and cash equivalents and deposits at the end of 2025 are expected to be not less than RMB1,100 million. We expect to have adequate cash into the 2028 excluding subsequent cash inflows.

Zevor-cel Demonstrates Rapid Sales Growth

Zevorcabtagene autoleucel (zevor-cel, R&D code: CT053) is an autologous fully human CAR T-cell product against B-cell maturation antigen (BCMA) approved by the NMPA of China for the treatment of adult patients with relapsed or refractory multiple myeloma (R/R MM) who have progressed after at least 3 prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent).

CARsgen entered into a collaboration agreement with Huadong Medicine (000963.SZ) for the commercialization of zevor-cel in mainland China. In terms of commercialization, Huadong Medicine has established a dedicated, professional, and comprehensive commercial team to promote the use of zevor-cel and has been utilizing China’s multi-layered insurance system to improve patient accessibility. During the first half of 2025, certification and regulatory filings for zevor-cel have been completed in more than 20 provinces or cities and we have received a total of 111 confirmed orders from Huadong Medicine. We anticipate that growth of sales revenue of zevor-cel will further accelerate with continuous marketing activities and broader insurance coverage.

Satri-cel NDA Accepted for Review in China

Satricabtagene autoleucel (satri-cel, R&D code: CT041) is an autologous humanized CAR T-cell product against Claudin18.2 (CLDN18.2). In June 2025, the CDE of NMPA of China has accepted the New Drug Application (NDA) for satri-cel for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction adenocarcinoma (G/GEJA) in patients who have failed at least two prior lines of therapy. Satri-cel is the first and only CAR T-cell product globally for which an NDA submitted for the treatment of solid tumors. Satri-cel was granted Priority Review in May 2025 and Breakthrough Therapy Designation (BTD) in March 2025 by the CDE.

The results of satri-cel confirmatory Phase II trial (NCT04581473) in China have been published in The Lancet and were orally presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Satri-cel demonstrated significant progression-free survival (PFS) improvement and a clinically meaningful overall survival (OS) benefit with a manageable safety profile, compared to standard therapy.

Multiple Allogeneic CAR-T Product Candidates in Development

CARsgen has been advancing differentiated allogeneic CAR T-cell products utilizing the proprietary THANK-uCAR platform. CARsgen has recently developed the THANK-u PlusTM platform as an enhanced version of THANK-uCAR to address the potential impact of NKG2A expression levels on therapeutic efficacy of the allogeneic CAR T-cells.

CT0596 is an allogeneic BCMA-targeted CAR T-cell product utilizing THANK-u Plus platform for the treatment of R/R MM and R/R plasma cell leukemia (PCL). The investigator-initiated trials (IITs) are ongoing in China. Preliminary clinical data for CT0596 were released in May 2025 on CARsgen’s official website. Based on the preliminary safety and efficacy data, CT0596 demonstrated favorable tolerability and encouraging efficacy signals in R/R MM patients across all predefined dose levels, with CAR-T expansion observed.

In addition, there are several allogeneic CAR T-cell products under development:

KJ-C2219: Targeting CD19/CD20, for the treatment of hematologic malignancies and autoimmune diseases. An IIT for relapsed/refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) has been initiated at the end of 2024. A separate IIT for systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) has been initiated in the first half of 2025.
KJ-C2320: Targeting CD38, for the treatment of acute myeloid leukemia (AML). An IIT has been initiated at the end of 2024.
KJ-C2114: For the treatment of solid tumors.
KJ-C2526: Targeting NKG2DL, for the treatment of AML, other malignancies, and senescence.
On February 25, 2025, CARsgen has entered into the agreements (the "Agreements") with an investment fund (the "Investor") managed by Zhuhai Hengqin SB Xinchuang Equity Investment Management Enterprise (Limited Partnership), pursuant to which, among others, the Investor has agreed to subscribe to additional registered capital of UCARsgen Biotech Limited ("UCARsgen") at a cash consideration of RMB80,000,000, representing 8% stake of the enlarged registered capital of UCARsgen. Upon the completion of the capital increase, CARsgen’s share in UCARsgen will be diluted from 100% to 92%.

UCARsgen is a China-based new drug discovery biotechnology company focused on allogeneic CAR T-cell therapies for the treatment of hematologic malignancies. Under the Agreements, UCARsgen has secured the exclusive rights in mainland China for the research, development, manufacture, and commercialization of the following allogeneic CAR T-cell products from CARsgen: the BCMA-targeted allogeneic CAR T-cell therapy for the treatment of multiple myeloma and plasma cell leukemia and the CD19/CD20 dual-targeted allogeneic CAR T-cell therapy for the treatment of B-cell malignancies (excluding indications for the treatment of autoimmune diseases).

On August 14, 2025 Plus Therapeutics, Inc. (Nasdaq: PSTV) ("Plus" or the "Company"), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers, reported positive data from a retrospective analysis of the CNSide Cerebrospinal Fluid (CSF) Assay Platform at the 2025 Society for Neuro-Oncology (SNO)/American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) CNS Metastases Conference in Baltimore, Maryland (Press release, Plus Therapeutics, AUG 14, 2025, View Source [SID1234655320]).

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The presentation, titled "The Oncogenic Flip in Patients with Leptomeningeal Metastatic Disease (LMD): Longitudinal Detection in Cerebrospinal Fluid Tumor Cells (CSF-TCs) Reveals Implications for Differential Treatment of the LMD Tumor," was a retrospective, multi-center analysis of 613 CNSide assays ordered by 19 physicians from 5 institutions at 2 health systems for 218 individual patients. 74% of the patients were female and the cancers most analyzed were breast (n=105) and lung (n=65). The research was presented by Priya U. Kumthekar, M.D., Professor of Neurology and Medicine at Northwestern University.

Data Demonstrated:

CSF tumor cells detected in 67% (412/613) patients using CNSide;
66 patients underwent 2 or more CSF draws; 24 patients underwent 5 or more;
20% (13/66) of patients were found to have a flip in immunocytochemistry (ICC) detection; and
88% (58/66) of patients were found to have a flip in FISH probe detection.
"The CNSide CSF Assay Platform can be used to detect gene amplification on CSF tumor cells of patients with LM and, therefore, may provide therapeutic insights to specifically target the LM tumor," said Priya U. Kumthekar, M.D., "Further, longitudinal CSF tumor cell analysis using CNSide may provide insights to modify treatment of the LM tumor over time."

The data builds upon previously announced results, "CSF Tumor Cell (CSF-TC) Detection, Quantification and Biomarker Assessment Helps in Clinical Management of Breast Cancer and Non-Small Cell Lung Cancer Patients Having Leptomeningeal Disease," from the prospective FORESEE study, which was also presented by Dr. Kumthekar, principal investigator. The study met key primary and secondary endpoints and showed that the CNSide CSF Assay platform influenced clinical management decisions in over 90% of LM cases. Further the CNSide CSF Assay demonstrated 2.8 times the diagnostic sensitivity versus standard CSF cytology.

About CNSide Diagnostic, LLC
CNSide Diagnostics, LLC is a wholly owned subsidiary of Plus Therapeutics, Inc. that develops and commercializes proprietary laboratory-developed tests, such as CNSide, designed to identify tumor cells that have metastasized to the central nervous system in patients with carcinomas and melanomas. The CNSide CSF Assay Platform enables quantitative analysis and molecular characterization of tumor cells and circulating tumor DNA in the cerebrospinal fluid that inform and improve the management of patients with leptomeningeal metastases.

About Leptomeningeal Metastases
Leptomeningeal metastases (LM) are a rare but severe complication of advanced cancer, affecting the fluid-lined structures of the central nervous system. LM occurs in approximately 5% of patients with metastatic cancer, but the actual incidence may be higher as it can be difficult to diagnose. Postmortem studies show the frequency of LM to be around 20% or more, highlighting healthcare providers’ need for more sensitive diagnostic options.

On August 14, 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 second quarter ended June 30, 2025 (Press release, Xilio Therapeutics, AUG 14, 2025, View Source [SID1234655319]).

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"During the second quarter, we continued to make strong progress across our pipeline of novel tumor-activated immuno-oncology therapies, and with our recent financing, we believe that we are well-positioned to execute on our strategic goals," said René Russo, Pharm.D., president and chief executive officer of Xilio. "We believe our masked T cell engager molecules have the potential to be best-in-class, and we anticipate nominating our first development candidates for our wholly owned programs later this year. In addition, we are very encouraged by the clinical progress for XTX301, our tumor-activated IL-12 advancing in partnership with Gilead, and we look forward to providing a program update in the near-term. We also recently reported updated Phase 2 combination data for vilastobart at ASCO (Free ASCO Whitepaper) and believe these data not only highlight the significant opportunity for vilastobart across a range of I-O combinations and tumor types, but also provide further clinical validation for our proprietary masking technology and approach."

Pipeline and Business Updates

Vilastobart: tumor-activated, Fc-enhanced, high affinity binding anti-CTLA-4

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). 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 metastatic microsatellite stable (MSS) colorectal cancer (CRC).

● In May 2025, Xilio announced promising updated Phase 2 data for vilastobart in combination with atezolizumab in patients with metastatic MSS CRC at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. These data demonstrated a preliminary 26% objective response rate in heavily pre-treated metastatic MSS CRC patients without liver metastases, including deep and durable responses accompanied by substantial decreases in tumor biomarkers and improvements in clinical symptoms, as well as a differentiated and well-tolerated safety profile with a low incidence of colitis and other immune-related adverse events, which have historically limited the potential for anti-CTLA-4 therapies. For more information, read the press release here. Xilio anticipates reporting additional data from the Phase 2 trial in the first half of 2026.

● Based on the encouraging clinical data to date, Xilio believes vilastobart has the potential to be an I-O combination agent of choice and be combined with a range of existing and next-generation agents. Xilio continues to engage strategic partners on potential opportunities to accelerate and expand further development for vilastobart.

XTX301: tumor-activated IL-12

XTX301 is an investigational tumor-activated IL-12 designed to potently stimulate anti-tumor immunity and reprogram the TME of poorly immunogenic "cold" tumors towards an inflamed or "hot" state. In March 2024, Xilio entered into an exclusive license agreement with Gilead Sciences, Inc. (Gilead) related to Xilio’s tumor-activated IL-12 program, including XTX301. Xilio is evaluating XTX301 as a monotherapy in an ongoing Phase 1 clinical trial in patients with advanced solid tumors.

● Xilio recently completed enrollment in Phase 1A monotherapy dose escalation and evaluation of those patients is ongoing. In addition, Xilio continues to enroll patients in Phase 1B monotherapy dose expansion of the ongoing Phase 1 clinical trial of XTX301.

XTX501: masked PD-1/IL-2 bispecific

XTX501 is a novel, tumor-activated bispecific PD-1/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.

● Xilio is continuing to advance XTX501 in investigational new drug (IND)-enabling studies and 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 the tumor-associated antigens for PSMA, CLDN18.2 and STEAP1 and an additional program in collaboration with 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 T cell activation.

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

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

Corporate Updates

● In June 2025, Xilio closed a follow-on public offering of prefunded warrants and accompanying common stock warrants and received initial gross proceeds of approximately $50.0 million before deducting underwriting discounts and commissions and offering expenses. In connection with the offering, Xilio issued Series B and Series C common stock warrants and will receive up to $100.0 million of additional gross proceeds by the second half of 2026 if all of those warrants are exercised in cash at their initial exercise price of $0.75 per warrant. The financing was co-led by new investors Coastlands Capital and Frazier Life Sciences and included participation from Gilead Sciences, Inc., Logos Capital, Samsara BioCapital and other new and existing institutional investors. For more information, read the press release here.

● In June 2025, Xilio announced the appointment of Akintunde (Tunde) Bello, Ph.D., to the company’s board of directors. For more information, read the press release here.

Second Quarter 2025 Financial Results

● Cash Position: Cash and cash equivalents were $121.6 million as of June 30, 2025, compared to $55.3 million as of December 31, 2024. In the second quarter of 2025, Xilio received $47.0 million in net proceeds from its June 2025 follow-on public offering after deducting underwriting discounts and commissions and offering expenses payable by the company.

● Collaboration and License Revenue: Collaboration and license revenue was $8.1 million for the quarter ended June 30, 2025, compared to $2.4 million for the quarter ended June 30, 2024. Collaboration and license revenue for the quarter ended June 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 June 30, 2024 consisted of revenue recognized in connection with Xilio’s collaboration with Gilead.

● Research & Development (R&D) Expenses: R&D expenses were $15.3 million for the quarter ended June 30, 2025, compared to $11.2 million for the quarter ended June 30, 2024. The increase was primarily driven by increased clinical development activities related to vilastobart, increased costs related to early-stage programs and indirect research and development and increased personnel-related costs, which were partially offset by decreased costs related to clinical development activities for XTX202, a tumor-activated IL-2, as a result of discontinuing further investment in that program. For the quarter ended June 30, 2025, R&D expenses also included manufacturing activities related to IND-enabling studies and preclinical development activities for XTX501, which were not separately tracked for the quarter ended June 30, 2024 as the company had not yet begun performing IND-enabling studies. For the quarter ended June 30, 2024, R&D expenses also included a $1.0 million development milestone under the CTLA-4 monoclonal antibody license agreement with WuXi Biologics (Hong Kong) Limited for which there was no comparable cost for the quarter ended June 30, 2025.

● General & Administrative (G&A) Expenses: G&A expenses were $7.1 million for the quarter ended June 30, 2025, compared to $5.8 million for the quarter ended June 30, 2024. The increase was primarily driven by an increase in professional and consulting fees, including legal fees and other professional costs, and an increase in personnel-related costs, which were partially offset by a decrease in costs related to directors’ and officers’ liability insurance.

● Net Loss: Net loss was $15.8 million for the quarter ended June 30, 2025, compared to $13.9 million for the quarter ended June 30, 2024.

Financial Guidance

Based on its current operating plans, Xilio anticipates that its cash and cash equivalents as of June 30, 2025 will be sufficient to enable it to fund its operating expenses and capital expenditure requirements through the end of the third quarter of 2026.

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 and tolerability of the combination in Phase 1C dose escalation in patients with advanced solid tumors and the safety and efficacy of the combination in Phase 2 in patients with metastatic microsatellite stable colorectal cancer with and without liver metastases. Please refer to NCT04896697 on www.clinicaltrials.gov for additional details.

About XTX301

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. In March 2024, Xilio entered into an exclusive license agreement with Gilead Sciences, Inc. for Xilio’s tumor-activated IL-12 program, including XTX301. Xilio is evaluating the safety and tolerability of XTX301 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. Please refer to NCT05684965 on www.clinicaltrials.gov for additional details.

On August 14, 2025 Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, reported financial results for the second quarter ended June 30, 2025, and highlighted recent corporate progress (Press release, Tyra Biosciences, AUG 14, 2025, View Source [SID1234655318]).

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"We see significant opportunity to transform the treatment of bladder cancer and skeletal dysplasia by precisely targeting FGFR3. With BEACH301 open for enrollment and SURF302 advancing in intermediate-risk non-muscle invasive bladder cancer, we’re building a franchise with dabogratinib around the power of FGFR3 selectivity and sensitivity," said Todd Harris, CEO of TYRA. "Backed by a strong balance sheet, we’re well positioned to deliver meaningful Phase 2 readouts in SURF302 and BEACH301.

Second Quarter 2025 and Recent Corporate Highlights

Dabogratinib (TYRA-300)

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Dabogratinib (formerly TYRA-300) is an oral investigational FGFR3-selective inhibitor being developed for the treatment of IR NMIBC and ACH.
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Dosed first patient in Phase 2 NMIBC Study – SURF302. SURF302 is an open-label Phase 2 clinical study evaluating the efficacy and safety of dabogratinib in participants with FGFR3-altered low-grade, IR NMIBC. Participants will be randomized initially to treatment with dabogratinib at 50 mg once-daily (QD) (Cohort 1) or treatment with dabogratinib at 60 mg QD (Cohort 2). The primary endpoint is complete response (CR) rate at three months. Secondary endpoints include time to recurrence, median duration of response, recurrence free survival, progression free survival, safety and tolerability.
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Advanced Phase 2 ACH Study – BEACH301. BEACH301 is a Phase 2, multicenter, open-label, dose-escalation/dose-expansion study evaluating dabogratinib in children ages 3 to 10 with achondroplasia with open growth plates. The study will enroll children who are treatment-naïve (Cohort 1) and those who have received prior growth-accelerating therapy (Cohort 2) at multiple sites across the globe. Each of these cohorts is expected to enroll up to 10 participants per dose level (0.125, 0.25, 0.375, 0.50 mg/kg) for up to 12 months. The study is now enrolling a safety sentinel cohort of up to 3 participants per dose level in children ages 5 to 10.
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Phase 1/2 mUC Study – SURF301. Dabogratinib continued to be evaluated in Part B of SURF301 (NCT05544552) at potentially therapeutic QD doses in preparation for potential future Phase 2 studies.
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Presented preclinical results at ENDO 2025. The late-breaking poster that was presented summarized the results of dabogratinib across three genetic contexts: wild-type mice, the Fgfr3Y367C/+ mouse model of ACH, and the Fgfr3N534K/+ mouse model of hypochondroplasia. Among other things, dabogratinib significantly improved the size and shape of the skull and foramen magnum in Fgfr3Y367C/+ mice. These studies demonstrate that dabogratinib led to a significant increase in bone growth in two independent FGFR3-driven preclinical models, as well as in wild-type mice, and provide further support to broaden the development of dabogratinib into skeletal dysplasias beyond ACH.

TYRA-430

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Advanced Phase 1 SURF431 Study. TYRA-430 is an oral, investigational FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers. Patient dosing is ongoing in SURF431, a Phase 1, multicenter, open-label, first-in-human study of TYRA-430 in advanced hepatocellular carcinoma (HCC) and other solid tumors with activating FGF/FGFR pathway aberrations. We believe TYRA-430 has the potential to address a significant unmet need in HCC, where there are no approved biomarker-driven, targeted therapies.

TYRA-200

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Advanced Phase 1 SURF201 Study. TYRA-200 is an FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The SURF201 study continues to enroll and dose adults with unresectable locally advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating FGFR2 gene alterations.

SNÅP Platform and Pipeline

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TYRA continued to advance its in-house precision medicine discovery engine, SNÅP, used to develop therapies in targeted oncology and genetically defined conditions.

Second Quarter 2025 Financial Results

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Cash, Cash Equivalents and Short-Term Investments. As of June 30, 2025, TYRA had cash, cash equivalents, and marketable securities of $296.3 million. TYRA’s current cash, cash equivalents and marketable securities are expected to allow TYRA to execute on its plans through at least 2027.
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Research and Development (R&D) Expenses. R&D expenses for the three months ended June 30, 2025 were $24.3 million compared to $18.0 million for the same period in 2024. The increase was associated with start-up and enrollment activities for BEACH301, SURF302 and SURF431, as well as increased CMC and personnel-related costs, including non-cash stock-based compensation.
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General and Administrative (G&A) Expenses. G&A expenses for the three months ended June 30, 2025 were $7.1 million compared to $5.5 million for the same period in 2024. The increase was primarily driven by higher personnel-related costs, including non-cash stock-based compensation.
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Net Loss. Second quarter net loss was $28.1 million compared to $18.7 million for the same period in 2024.

Upcoming Clinical Milestones for Dabogratinib

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BEACH301: dose first child with achondroplasia – 3Q 2025
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SURF302: topline initial three-month complete response data – 1H 2026

About Dabogratinib (formerly TYRA-300)

Dabogratinib is TYRA’s lead precision medicine candidate stemming from its in-house SNÅP platform. Dabogratinib is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasia that has demonstrated interim clinical proof-of-concept results in metastatic urothelial cancer (mUC). The current planned clinical development for dabogratinib includes Phase 2 clinical trials for IR NMIBC (SURF302) and pediatric achondroplasia (BEACH301), and potentially mUC.

Please visit the Patients page of our website for more information on our clinical trials.

About TYRA-430

TYRA-430 is an oral, investigational FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers. The Phase 1 study (SURF431) is a multicenter, open-label, first-in-human study of TYRA-430 and is currently enrolling and dosing adults with advanced HCC and other solid tumors with activating FGF/FGFR pathway aberrations (NCT06915753).

About TYRA-200

TYRA-200 is an oral, investigational, FGFR1/2/3 inhibitor with potency against activating FGFR2 gene alterations and resistance mutations. The Phase 1 clinical study of TYRA-200, SURF201 (NCT06160752), is a multi-center, open label study designed to evaluate the maximum tolerated dose and the recommended Phase 2 dose of TYRA-200, as well as to evaluate the preliminary antitumor activity of TYRA-200. SURF201 is currently enrolling and dosing adults with advanced/metastatic intrahepatic cholangiocarcinoma and other advanced solid tumors with activating alterations in FGFR2.

On August 14, 2025 TuHURA Biosciences, Inc. (NASDAQ: HURA) ("TuHURA" or the "Company"), a Phase 3 immuno-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported financial results for the Company’s second quarter ended June 30, 2025, and provided a corporate update (Press release, TuHURA Biosciences, AUG 14, 2025, View Source [SID1234655317]).

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"TuHURA had a strong first-half of the year with the initiation of its Phase 3 accelerated approval trial of IFx-2.0 as an adjunctive therapy to pembrolizumab as a first line treatment for patients with advanced or metastatic MCC. Conducted under an SPA Agreement with the FDA, the Phase 3 trial is a single randomized placebo-controlled trial that, if successful, has the potential to both meet and satisfy the requirements for both accelerated and full approval without the need to conduct a post-accelerated approval confirmatory trial. This potentially translates to a meaningful time and cost savings to TuHURA," stated James Bianco, M.D., President and Chief Executive Officer of TuHURA. "In addition to our accelerated approval Phase 3 trial of IFx-2.0, we also initiated a Phase 1b/2a trial employing interventional radiologic administration of IFx-2.0 as an adjunctive therapy to pembrolizumab in first-line treatment of checkpoint-naïve patients with MCC of unknown primary origin (MCCUP). This trial will enroll newly diagnosed patients who present metastatic, deep-seated tumors in the liver, lungs, or retroperitoneum, without accessible cutaneous, subdermal or nodal lesions. Patients with MCCUP represent approximately a thirty percent (30%) of all newly diagnosed advanced or metastatic MCC cases and can meaningfully augment IFx-2.0’s commercial market opportunity."

Dr. Bianco continued, "In addition to our trial initiations in MCC and MCCUP, we recently bolstered our development pipeline with the acquisition of Kineta and their novel VISTA inhibiting antibody, TBS-2025. The acquisition provides for synergies across both TuHURA’s therapeutic focus as well as TuHURA’s antibody peptide or drug candidate (APC, ADC) technologies as we continue to assemble a diversified, late-stage immuno-oncology pipeline. We plan to advance TBS-2025 into a randomized Phase 2 trial in patients with relapsed or refractory NPM1-mutated AML planned for the second half of this year to determine if the addition of TBS-2025 to a menin inhibitor can improve the results seen in patients receiving a menin inhibitor."

Corporate Highlights

Inclusion of TuHURA Biosciences in the Russell 3000 and Russell 2000 Indexes. In June 2025, TuHURA announced its addition to the Russell 3000 Index, with automatic inclusion in the Russell 2000 Index, as a part of the 2025 Russell annual reconstitution. The Company’s addition was effective as of market close on June 27, 2025.
Completion of Kineta, Inc. Acquisition and Kineta’s VISTA Inhibiting mAb. In June 2025, TuHURA announced the closing of its acquisition of Kineta, and Kineta’s novel VISTA inhibiting mAb, now referred to as "TBS-2025." TuHURA plans to initiate a Phase 2 randomized trial of TBS-2025 in combination with a menin inhibitor for the treatment of relapsed or refractory NPM1-mutated AML, compared to a menin inhibitor alone, targeted for the second half of 2025.
Initiation of Phase 3 Accelerated Approval Trial of IFx-2.0 as Adjunctive Therapy to Keytruda in 1L MCC. In June 2025, TuHURA announced that it had initiated its Phase 3 accelerated approval trial of IFx-2.0 as an adjunctive therapy to pembrolizumab in MCC. Conducted under an SPA agreement with the U.S. FDA, TuHURA is investigating the effectiveness of IFx-2.0 as an adjunctive therapy to Keytruda compared to Keytruda plus placebo in first line treatment in advanced or metastatic MCC.
Completed $12.5 Million Equity Financing Transaction and Received an Additional $3.0 Million in Warrant Exercise Proceeds. In June 2025, TuHURA announced that it had entered into a definitive securities purchase agreement for the issuance and sale in a private placement of an aggregate of $12.5 million shares of its common stock. In addition to the offering, the Company secured $3.0 million in additional cash proceeds from the previously disclosed February 2025 cash exercise of approximately 1.0 million warrants to purchase shares of the Company common stock.
Upcoming Targeted Milestones by Program

IFx-2.0

Year-End 2025: TuHURA anticipates providing an update on enrollment progress in its Phase 3 accelerated approval trial of IFx-2.0 as an adjunctive therapy to pembrolizumab in first line MCC
Q1 2026: Anticipated topline results from Phase 1b/2a clinical trial of IFx-2.0 as an adjunctive therapy to pembrolizumab in first line treatment for MCC of unknown primary origin (MCCUP)
2H 2026: Anticipated topline results from Phase 3 accelerated approval trial
TBS-2025

2H 2025: Planned initiation of Phase 2 trial of VISTA inhibiting mAb in combination with a menin inhibitor for the treatment of relapsed or refractory NPM1-mutated AML
APC and ADC Development Candidates

TuHURA continues to advance its bi-specific, bi-functional immune modulating ADCs and APCs that target the Delta Opioid Receptor (DOR) on MDSCs, inhibiting their immune suppressing effects in the tumor microenvironment while localizing a checkpoint inhibitor like TBS-2025
In 2025, TuHURA anticipates presenting non-clinical data at relevant medical meetings
Financial Results for the Three Months and Six Months Ended June 30, 2025

Research and development expenses were $4.9 million and $2.8 million for the three months ended June 30, 2025, and 2024, respectively.

Net cash outflows from operating activities were ($10.9) million and ($8.9) million for the six months ended June 30, 2025, and 2024, respectively.

As of June 30, 2025, TuHURA’s total shares outstanding was approximately 49.9 million.