Phase 2 Study Evaluating the Efficacy of the Combination of DKN-01 (DKK1 Inhibitor, Leap Therapeutics) and Pembrolizumab in the Treatment of Advanced or Recurrent Endometrial Cancer

To learn if the combination of DKN-01 and pembrolizumab can help to control advanced or recurrent endometrial cancer.

Start Date29 Aug 2023

Sponsor / Collaborator

The University of Texas MD Anderson Cancer Center

[+3]

NCT05480306

/ CompletedPhase 2

Randomized Phase 2 Study of DKN-01 Plus FOLFIRI/FOLFOX and Bevacizumab Versus FOLFIRI/FOLFOX and Bevacizumab as Second-line Treatment of Advanced Colorectal Cancer (DeFianCe)

This is a Phase 2 randomized, open-label, two-part, multicenter study with a safety run-in to evaluate efficacy and safety of DKN-01 plus FOLFIRI/FOLFOX and bevacizumab versus standard of care (SOC) [FOLFIRI/FOLFOX and bevacizumab] as second-line treatment of advanced CRC patients.

Start Date30 Aug 2022

Sponsor / Collaborator

Cypherpunk Technologies, Inc.

NCT04363801

/ TerminatedPhase 2

A Phase 2, Multicenter, Open-Label Study of DKN-01 in Combination With Tislelizumab ± Chemotherapy as First-Line or Second-Line Therapy in Adult Patients With Inoperable, Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma (DisTinGuish)

A Phase 2, Multicenter, Open-Label Study of DKN-01 in Combination with Tislelizumab ± Chemotherapy as First-Line or Second-Line Therapy in Adult Patients with Inoperable, Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Adenocarcinoma

Start Date29 Jul 2020

Sponsor / Collaborator

Cypherpunk Technologies, Inc.

[+1]

100 Clinical Results associated with Sirexatamab

100 Translational Medicine associated with Sirexatamab

100 Patents (Medical) associated with Sirexatamab

Literatures (Medical) associated with Sirexatamab

01 Jun 2025·Prostate cancer and prostatic diseases

A Phase 1/2 multicenter trial of DKN-01 as monotherapy or in combination with docetaxel for the treatment of metastatic castration-resistant prostate cancer (mCRPC)

Article

Author: Loomis, Cynthia ; Pachynski, Russell K ; Leis, Dayna ; Griglun, Sarah ; Newman, Walter ; Deng, Jiehui ; Chattopadhyay, Pratip ; Kagey, Michael H ; Ren, Qinghu ; Fedal, Ezeddin ; Waalkes, Erika ; Gargano, Gabrielle ; Febles, Victor Adorno ; Wise, David R ; Sirard, Cynthia A ; Troxel, Andrea B ; Baum, Jason ; Chiriboga, Luis ; Deng, Fang-Ming ; Yang, Nina ; Soamchand, Nadia ; Economides, Minas P ; Denmeade, Samuel R ; Aggarwal, Rahul R ; Selvaraj, Shanmugapriya ; Wong, Kwok-Kin ; Aranchiy, Viktoriya ; Balar, Arjun V ; Haas, Michael ; Puranik, Amrutesh ; Machado, Sabrina ; Melamed, Jonathan

BACKGROUND:

Dickkopf-related protein 1 (DKK1) is a Wingless-related integrate site (Wnt) signaling modulator that is upregulated in prostate cancers (PCa) with low androgen receptor expression. DKN-01, an IgG4 that neutralizes DKK1, delays PCa growth in pre-clinical DKK1-expressing models. These data provided the rationale for a clinical trial testing DKN-01 in patients with metastatic castration-resistant PCa (mCRPC).

METHODS:

This was an investigator-initiated parallel-arm phase 1/2 clinical trial testing DKN-01 alone (monotherapy) or in combination with docetaxel 75 mg/m² (combination) for men with mCRPC who progressed on ≥1 AR signaling inhibitors. DKK1 status was determined by RNA in-situ expression. The primary endpoint of the phase 1 dose escalation cohorts was the determination of the recommended phase 2 dose (RP2D). The primary endpoint of the phase 2 expansion cohorts was objective response rate by iRECIST criteria in patients treated with the combination.

RESULTS:

18 pts were enrolled into the study-10 patients in the monotherapy cohorts and 8 patients in the combination cohorts. No DLTs were observed and DKN-01 600 mg was determined as the RP2D. A best overall response of stable disease occurred in two out of seven (29%) evaluable patients in the monotherapy cohort. In the combination cohort, five out of seven (71%) evaluable patients had a partial response (PR). A median rPFS of 5.7 months was observed in the combination cohort. In the combination cohort, the median tumoral DKK1 expression H-score was 0.75 and the rPFS observed was similar between patients with DKK1 H-score ≥1 versus H-score = 0.

CONCLUSION:

DKN-01 600 mg was well tolerated. DKK1 blockade has modest anti-tumor activity as a monotherapy for mCRPC. Anti-tumor activity was observed in the combination cohorts, but the response duration was limited. DKK1 expression in the majority of mCRPC is low and did not clearly correlate with anti-tumor activity of DKN-01 plus docetaxel.

20 Jan 2025·JOURNAL OF CLINICAL ONCOLOGY

DKN-01 in Combination With Tislelizumab and Chemotherapy as First-Line Therapy in Advanced Gastric or Gastroesophageal Junction Adenocarcinoma: DisTinGuish

Article

Author: Stilian, Melissa C. ; Thoma, Mathis ; Ajani, Jaffer A. ; Scott, Aaron James ; Chung, Vincent ; Chiu, Vi K. ; Kagey, Michael H. ; Uronis, Hope Elizabeth ; Tejani, Mohamedtaki Abdulaziz ; Baum, Jason ; Sirard, Cynthia A. ; Sonbol, Mohamad Bassam ; Wainberg, Zev A. ; Iqbal, Syma ; Altura, Rachel A. ; Zhang, Ying ; Klempner, Samuel J.

PURPOSE:

The outcomes of anti–PD-1 agents plus fluoropyrimidine/platinum in frontline advanced gastroesophageal adenocarcinomas (aGEAs) remain poor. We investigated the safety, tolerability, and activity of fluoropyrimidine/oxaliplatin and tislelizumab with the DKK1-neutralizing antibody DKN-01 in aGEAs in a phase IIa open-label study.

PATIENTS AND METHODS:

Patients had untreated human epidermal growth factor receptor 2–negative aGEAs, RECIST v1.1 measurable disease, Eastern Cooperative Oncology Group (ECOG) performance status 0-1, and adequate organ function. Patients received intravenous DKN-01 300 mg once every 2 weeks, tislelizumab 200 mg once every 3 weeks, oxaliplatin 130 mg/m 2 once every 3 weeks, and capecitabine 1,000 mg/m 2 twice daily on days 1-15 of each 21-day cycle. The primary end point was safety and tolerability. Key secondary end points included objective response rate (ORR) by RECISTv1.1, progression-free survival (PFS), and overall survival (OS).

RESULTS:

Between September 18, 2020, and April 8, 2021, 25 patients were enrolled. All patients who received at least one dose of DKN-01 were included in the safety analysis. Most patients had gastroesophageal junction tumors, median age was 61 years, 76% were male, and 55% were ECOG of 0. All patients reported at least one treatment-emergent adverse event. The ORR was 73% (95% CI, 49.8 to 89.3), with a disease control rate of 95%. The ORR was 90% (95% CI, 55.5 to 99.7) in the DKK1-high tumor patients and 67% (95% CI, 29.9 to 92.5) in the DKK1-low tumor patients. The median PFS was 11.3 months (95% CI, 5.8 to 12.0) and the 12-month PFS rate was 33%. The median OS was 19.5 months (95% CI, 15.2 to 24.4) with a 12-month OS rate of 76% and an 18-month OS rate of 55%.

CONCLUSION:

DKN-01 can be safely combined with frontline fluoropyrimidine/oxaliplatin and tislelizumab and demonstrates encouraging activity independent of PD-L1 expression levels. A randomized phase II trial is ongoing (ClinicalTrials.gov identifier: NCT04363801 ).

01 Jan 2023·Liver International

Article

Author: Tsokkou, Panagiota ; Musialik, Kamila I. ; Horcas‐Lopez, Marta ; Newman, Walter ; Kagey, Michael H. ; Boulter, Luke ; Waddell, Scott H. ; Gournopanos, Konstantinos ; MacMaster, Stephanie ; Soong, Daniel Y. H. ; Ng, Minn‐E ; Cambridge, William A. ; Wilson, David H. ; Jarman, Edward J. ; Pollard, Jeffrey W.

Abstract:

Background and aims:

Dickkopf‐1 (DKK1) is associated with poor prognosis in intrahepatic cholangiocarcinoma (iCCA), but the mechanisms behind this are unclear. Here, we show that DKK1 plays an immune regulatory role in vivo and inhibition reduces tumour growth.

Methods:

Various in vivo GEMM mouse models and patient samples were utilized to assess the effects of tumour specific DKK1 overexpression in iCCA. DKK1 ‐driven changes to the tumour immune microenvironment were characterized by immunostaining and gene expression analysis. DKK1 overexpressing and damage‐induced models of iCCA were used to demonstrate the therapeutic efficacy of DKK1 inhibition in these contexts using the anti‐DKK1 therapeutic, DKN‐01.

Results:

DKK1 overexpression in mouse models of iCCA drives an increase in chemokine and cytokine signalling, the recruitment of regulatory macrophages, and promotes the formation of a tolerogenic niche with higher numbers of regulatory T cells. We show a similar association of DKK1 with FOXP3 and regulatory T cells in patient tissue and gene expression data, demonstrating these effects are relevant to human iCCA. Finally, we demonstrate that inhibition of DKK1 with the monoclonal antibody mDKN‐01 is effective at reducing tumour burden in two distinct mouse models of the disease.

Conclusion:

DKK1 promotes tumour immune evasion in iCCA through the recruitment of immune suppressive macrophages. Targeting DKK1 with a neutralizing antibody is effective at reducing tumour growth in vivo. As such, DKK1 targeted and immune modulatory therapies may be an effective strategy in iCCA patients with high DKK1 tumour expression or tolerogenic immune phenotypes.

News (Medical) associated with Sirexatamab

20 Oct 2025

·www.prnewswire.com

Leap Therapeutics Presents Final Data from DeFianCe Study at ESMO 2025

Sirexatamab demonstrated statistically significant improvement in PFS and OS in DKK1-high population Increasing DKK1 levels further improved PFS, OS, and ORR advantage for the Sirexatamab Arm Leap to continue supporting development of sirexatamab in DKK1-high CRC patients CAMBRIDGE, Mass., Oct. 20, 2025 /PRNewswire/ -- Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, today announced final results from Part B of the DeFianCe study (NCT05480306), a Phase 2 study of sirexatamab (DKN-01), an anti-DKK1 monoclonal antibody, in combination with bevacizumab and chemotherapy (Sirexatamab Arm) compared to bevacizumab and chemotherapy (Control Arm) in patients with microsatellite stable (MSS) colorectal cancer (CRC) who have received one prior systemic therapy for advanced disease. The final clinical results were presented on behalf of the DeFianCe study investigators by Zev Wainberg, MD, Professor of Medicine and Co-Director of the GI Oncology Program at UCLA in a Mini Oral session at the European Society for Medical Oncology (ESMO) Congress 2025 in Berlin, Germany. "Circulating DKK1 is a negative prognostic factor and elevated in patients with advanced, metastatic CRC. The data presented at ESMO demonstrate that sirexatamab, which binds to and removes free DKK1, has significant potential to provide a survival benefit for CRC patients who have high DKK1 levels and who are likely to have poor outcomes receiving the current standard of care alone," said Dr. Wainberg. "Sirexatamab has the potential to be a valuable addition to the CRC treatment paradigm as a targeted therapeutic for patients with high DKK1 and should move forward to be evaluated in a biomarker-focused registrational trial." The DeFianCe study was a two part, open-label, multi-country study. Part A of the DeFianCe study enrolled 33 patients, including a significant number of patients who had early progression on first-line therapy, previous exposure to bevacizumab, tumors with RAS mutations, or liver and lung metastases. The study expanded into a 188 patient Part B randomized controlled trial. The primary objective of the study was progression-free survival PFS. Secondary objectives included objective response rate (ORR), duration of response, and overall survival (OS). A key pre-defined exploratory population was those patients who had high levels of circulating DKK1, as measured by a biomarker assay. Key Part B DeFianCe Study Findings: Across the DKK1-high (upper median) patients (n=88): ORR was 38.0% in the Sirexatamab Arm compared to 23.7% ORR in the Control Arm. mPFS was 9.03 months in the Sirexatamab Arm compared to 7.06 months in the Control Arm, Hazard Ratio (HR) 0.61, p-value = 0.0255. mOS was not reached in the Sirexatamab Arm compared to 14.39 months in the Control Arm, HR 0.42, p-value = 0.0118. Across the DKK1-high (upper quartile) patients (n=44): ORR was 44.0% in the Sirexatamab Arm compared to 15.8% ORR in the Control Arm. mPFS was 9.36 months in the Sirexatamab Arm compared to 5.88 months in the Control Arm, HR 0.46, p-value = 0.0168. mOS was not reached in the Sirexatamab Arm compared to 9.66 months in the Control Arm, HR 0.17, p-value < 0.001. In the full intent-to-treat population (n=188): ORR was 35.1% in the Sirexatamab Arm compared to 26.6% ORR in the Control Arm. mPFS was 9.2 months in the Sirexatamab Arm compared to 8.3 months in the Control Arm, HR 0.84, p-value = 0.1712. Event-free rate favors Sirexatamab Arm beginning at month 9 (53 vs 47%) with further separation at month 12 (34 vs 23%). Sirexatamab, in combination with chemotherapy and bevacizumab, was safe and well tolerated Overall treatment-emergent adverse effects (TEAE) profile was similar between the Sirextamab and Control Arms, suggesting sirexatamab did not impact the safety profile when combined with the standard of care. "The DeFianCe study results demonstrate the significant potential of sirexatamab in patients with advanced CRC. Patients with this aggressive cancer, particularly those with high DKK1 levels, have poor overall survival outcomes and few promising second-line or later options," said Douglas E. Onsi, President and Chief Executive Officer of Leap. "Sirexatamab has repeatedly demonstrated its potential as a novel, first-in-class antibody targeting DKK1 that provides deep and durable benefit for patients in desperate need of new therapies. With support from a recently completed financing, Leap plans to engage with regulatory authorities over the registrational path for sirexatamab in CRC and to optimize the DKK1 biomarker diagnostic test that could be used to identify these CRC patients with poor prognosis." About Leap Therapeutics Leap Therapeutics (Nasdaq: LPTX) is focused on developing targeted and immuno-oncology therapeutics. Leap's pipeline includes sirexatamab (DKN-01), a humanized monoclonal antibody targeting the Dickkopf-1 (DKK1) protein, and FL-501, a humanized monoclonal antibody targeting the growth and differentiation factor 15 (GDF-15) protein. Leap initiated a digital asset treasury strategy following a $58.88 million private placement led by Winklevoss Capital, aiming to build long-term shareholder value and continue the development of sirexatamab and FL-501. For more information about Leap Therapeutics, visit or view our public filings with the SEC that are available via EDGAR at or via . FORWARD-LOOKING STATEMENTS This press release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements generally can be identified by the use of words such as "anticipate," "expect," "plan," "could," "may," "will," "believe," "estimate," "forecast," "goal," "project," and other words of similar meaning. These forward-looking statements, including statements regarding the potential safety, efficacy, and regulatory and clinical progress of sirexatamab; regulatory feedback that Leap may receive from U.S. Food and Drug Administration (FDA) or equivalent foreign regulatory agency or from site institutional review boards; the intended use of proceeds from the recent private placement offering; the assets to be held by the Company; the expected future market, price and liquidity of the digital assets the Company acquires; the macro and political conditions surrounding digital assets; and the Company's plan for value creation and performance. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, any regulatory feedback that Leap may receive from FDA or equivalent foreign regulatory agency or from site institutional review boards; failure to realize the anticipated benefits of the digital asset treasury strategy; changes in business, market, financial, political and regulatory conditions; risks relating to the Company's operations and business, including the highly volatile nature of the price of cryptocurrencies; the risk that the price of the Company's common stock may be highly correlated to the price of the digital assets that it holds; risks related to increased competition in the industries in which the Company does and will operate; risks relating to significant legal, commercial, regulatory, and technical uncertainty regarding pharmaceutical development and digital assets generally; and risks relating to the treatment of crypto assets for U.S. and foreign tax purposes. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Leap may not actually achieve the forecasts disclosed in such forward-looking statements, and you should not place undue reliance on such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Leap's most recent Annual Report on Form 10-K filed with the SEC, as well as discussions of potential risks, uncertainties, and other important factors in its subsequent filings with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither Leap, nor any of its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing Leap's views as of any date subsequent to the date hereof. CONTACT: Douglas E. Onsi President & Chief Executive Officer Leap Therapeutics, Inc. 617-714-0360 [email protected] Matthew DeYoung Investor Relations Argot Partners 212-600-1902 [email protected] SOURCE Leap Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultPhase 2ASCOImmunotherapy

09 Oct 2025

·www.prnewswire.com

Leap Therapeutics Announces Closing of $58.88 Million Private Placement in Cash Led by Winklevoss Capital to Initiate a Digital Asset Treasury Strategy

CAMBRIDGE, Mass., Oct. 9, 2025 /PRNewswire/ -- Leap Therapeutics (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, today announced that it has closed its previously announced private placement led by Winklevoss Capital raising $58,888,888 in cash to initiate a digital asset treasury strategy. At the closing of the transaction, Leap issued (i) 15,212,311 shares of common stock (the "Shares"), (ii) pre-funded warrants (the "Pre-Funded Warrants") to purchase up to an aggregate of 80,768,504 shares of common stock, and (ii) warrants to purchase an additional 71,985,605 shares of common stock at an exercise price of $0.5335 per share (the "Warrants" and together with the Shares and the Pre-Funded Warrants, the "Units"). Winklevoss Capital will provide both capital and strategic support to help Leap structure and execute its digital asset treasury strategy. Leveraging the expertise of Winklevoss Capital, Leap aims to build long-term shareholder value through participation in digital assets. A portion of the capital will be used to continue development of Leap's therapeutic programs, including FL-501 and sirexatamab, which has recently completed a randomized controlled Phase 2 trial in patients with colorectal cancer that is being presented in a Mini Oral Session at the European Society for Medical Oncology (ESMO) Congress 2025 on October 19 in Berlin, Germany. Pursuant to a lead investor agreement, Leap has increased the size of its Board of Directors to 12 members, and Winklevoss Capital will have the right to nominate two individuals to Leap's Board of Directors, one of whom shall also be the chairperson of the Board. Leap expects to release additional updates regarding its treasury activities in the near-term. Parcrest acted as the placement agent in connection with the financing. Morgan, Lewis & Bockius LLP acted as legal advisor to Leap, and Cooley LLP acted as legal advisor to Winklevoss Capital. The offer and sale of the foregoing securities is being made in a private placement in reliance on an exemption from the registration requirement of the Securities Act of 1933, as amended (the "Securities Act"), pursuant to Section 4(a)(2) of the Securities Act and/or Regulation D promulgated thereunder, and applicable state securities laws. Accordingly, the securities offered in the private placement may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirement of the Securities Act and such applicable state securities laws. Concurrently with the execution of the securities purchase agreements, the Company and the investors entered into a registration rights agreement pursuant to which the Company has agreed to file a registration statement with the Securities and Exchange Commission (the "SEC") registering the resale by the purchasers participating in the offering of the Shares and the shares of common stock underlying the Pre-Funded Warrants and the Warrants. Any offering of the Company's common stock under the resale registration statement will only be made by means of a prospectus. This press release shall not constitute an offer to sell or a 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. The private placement was conducted in accordance with applicable Nasdaq rules and was priced to satisfy the "Minimum Price" requirement (as defined in the Nasdaq rules). About Leap Therapeutics Leap Therapeutics (Nasdaq: LPTX) is focused on developing targeted and immuno-oncology therapeutics. Leap's pipeline includes sirexatamab (DKN-01), a humanized monoclonal antibody targeting the Dickkopf-1 (DKK1) protein and FL-501, a humanized monoclonal antibody targeting the growth and differentiation factor 15 (GDF-15) protein. Leap is initiating a digital asset treasury strategy following a $58.88 million investment led by Winklevoss Capital. For more information about Leap Therapeutics, visit or view our public filings with the SEC that are available via EDGAR at or via . About Winklevoss Capital Winklevoss Capital is an investment firm founded in 2012 by Cameron and Tyler Winklevoss that invests in frontier technologies. Forward Looking Statements This press release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements generally can be identified by the use of words such as "anticipate," "expect," "plan," "could," "may," "will," "believe," "estimate," "forecast," "goal," "project," and other words of similar meaning. These forward-looking statements address various matters including statements relating to the anticipated benefits and timing of the completion of the proposed offering and related transactions, the intended use of proceeds from the offering, the assets to be held by the Company, the expected future market, price and liquidity of the digital assets the Company acquires, the macro and political conditions surrounding digital assets, the Company's plan for value creation and strategic advantages, market size and growth opportunities, regulatory conditions, competitive position and the interest of other corporations in similar business strategies, technological and market trends, future financial condition and performance and the expected financial impacts of the proposed transactions described herein. Each forward-looking statement contained in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others, the risk that the proposed transactions described herein may not be completed in a timely manner or at all; failure to realize the anticipated benefits of the transactions and the proposed digital asset treasury strategy; changes in business, market, financial, political and regulatory conditions; risks relating to the Company's operations and business, including the highly volatile nature of the price of cryptocurrencies; the risk that the price of the Company's common stock may be highly correlated to the price of the digital assets that it holds; risks related to increased competition in the industries in which the Company does and will operate; risks relating to significant legal, commercial, regulatory, and technical uncertainty regarding digital assets generally; risks relating to the treatment of crypto assets for U.S. and foreign tax purposes, as well as those risks and uncertainties identified in the Company's filings with the Securities and Exchange Commission. The forward-looking statements in this press release speak only as of the date of this document, and the Company undertakes no obligation to update or revise any of these statements. Contacts Douglas E. Onsi President & Chief Executive Officer Leap Therapeutics, Inc. 617-714-0360 [email protected] Matthew DeYoung Investor Relations Argot Partners 212-600-1902 [email protected] SOURCE Leap Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2

07 Oct 2025

·www.prnewswire.com

Clinical Breakthroughs Converge as Immunotherapy Market Surges Past $136 Billion

USA News Group News Commentary Issued on behalf of GT Biopharma, Inc. VANCOUVER, BC, Oct. 7, 2025 /PRNewswire/ -- USA News Group News Commentary – Major pharmaceutical companies are unveiling breakthrough cancer data at the European Society for Medical Oncology Congress taking place October 17-21 in Berlin[1], while the global cancer immunotherapy market reached $137.70 billion in 2024 and analysts project explosive growth toward $261.74 billion by 2033[2]. The timing reflects a critical inflection point as HHS doubled federal funding for its Childhood Cancer Data Initiative from $50 million to $100 million, while researchers discovered how cancer uses a hidden hormone to shield itself from immune attack[3]. Against this backdrop of scientific momentum and institutional capital deployment, clinical-stage immunotherapy developers including GT Biopharma, Inc. (NASDAQ: GTBP), Leap Therapeutics, Inc. (NASDAQ: LPTX), IO Biotech (NASDAQ: IOBT), Celcuity Inc. (NASDAQ: CELC), and MAIA Biotechnology, Inc. (NYSE-American: MAIA). With eight new oncology approvals issued from July through September[4] and regulatory agencies accelerating approval pathways through breakthrough therapy designations, institutional forecasters at Precedence Research project the market will expand at a 10.65% CAGR through 2034[5] as long-term survival data validates immunotherapy durability. The convergence of rising disease burden, proven efficacy across cancer types, and expanded federal research support creates favorable conditions for early-positioned companies with differentiated mechanisms advancing through clinical milestones before broader institutional recognition materializes. GT Biopharma, Inc. (NASDAQ: GTBP) is a clinical-stage immunotherapy company making significant progress in its fight against difficult-to-treat cancers. The San Francisco-based biotech has been advancing its lead drug candidate, GTB-3650, through a Phase 1 clinical trial targeting blood cancers that have stopped responding to other treatments. In August, the company successfully moved into Cohort 3 after formal safety reviews of the first two patient groups showed no safety or tolerability problems. The trial had treated five patients by mid-August, with encouraging early signals of immune system activation. "We are pleased with the enrollment momentum in our Phase 1 clinical trial evaluating GTB-3650 in cancer patients, which continues to advance on schedule," said Michael Breen, Executive Chairman and Chief Executive Officer of GT Biopharma. "Moving into the third dose cohort after a successful safety review and encouraging early evidence of immunological activity, mark important steps forward in the development of GTB-3650. We look forward to sharing more data later this year to reinforce the ability of our TriKE constructs to activate endogenous NK cells, and the potential for broader utility with other targets to treat solid tumors (GTB-5550) and autoimmune indications (GTB-7550)." The Phase 1 trial is testing GTB-3650 in patients with relapsed or refractory CD33-expressing blood cancers, including acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). These are patients whose cancers have come back or never responded to standard therapies. The drug works by activating the body's own natural killer cells to attack cancer cells. Patients receive the treatment through continuous infusions in two-week cycles, alternating two weeks on and two weeks off, for up to four months based on how well they're responding. What makes the early data particularly interesting is the biomarker evidence. Multiple blood tests from the first four patients showed measurable increases in natural killer cell activity and expansion. This suggests the drug is doing exactly what it was designed to do—wake up the immune system and direct it against cancer. GT Biopharma expects to release more detailed Phase 1 results later this year after completing additional dose cohorts. Beyond blood cancers, GT Biopharma has a second drug candidate moving toward the clinic. GTB-5550 targets a protein called B7H3 that appears in many different types of solid tumors, including breast, lung, ovarian, head and neck, pancreatic, bladder, and prostate cancers. The company expects to submit its application to start human testing of GTB-5550 during the fourth quarter of this year. Unlike many cancer immunotherapies that require lengthy hospital infusions, GTB-5550 is being developed as a simple injection that patients could potentially give themselves at home, similar to insulin shots. Both drug candidates are built on GT Biopharma's proprietary TriKE platform, which uses specialized antibody fragments originally discovered in camels and llamas. These molecules are smaller and more stable than traditional antibodies, allowing them to work more effectively. GT Biopharma holds an exclusive worldwide license from the University of Minnesota to develop and commercialize therapies using this technology. As of June 30, 2025, GT Biopharmareported cash and cash equivalents of approximately $5.3 million, which management expected would fund operations into the first quarter of 2026. CONTINUED… Read this and more news for GT Biopharma, Inc. at: Leap Therapeutics, Inc. (NASDAQ: LPTX) are set to present final clinical results from Part B of the DeFianCe study at the European Society for Medical Oncology (ESMO) Congress 2025, taking place October 17-21 in Berlin, Germany. The Phase 2 study evaluated sirexatamab (DKN-01), an anti-DKK1 monoclonal antibody, in combination with bevacizumab and chemotherapy compared to bevacizumab and chemotherapy alone in patients with advanced microsatellite stable colorectal cancer who received one prior systemic therapy for advanced disease. The final results will be presented in a Mini Oral Session on October 19, 2025 at 4:05 p.m. CEST, with the presentation providing detailed efficacy and safety data from the second-line treatment cohort. Leap Therapeutics' pipeline also includes FL-501, a humanized monoclonal antibody targeting the growth and differentiation factor 15 protein, as the company continues developing targeted and immuno-oncology therapeutics for solid tumor indications. IO Biotech (NASDAQ: IOBT) announced that the U.S. Food and Drug Administration has recommended against submitting a Biologics License Application based on data from the IOB-013 clinical trial, in which Cylembio plus pembrolizumab improved progression free survival but narrowly missed statistical significance. The company is implementing a restructuring that includes an approximate 50 percent reduction in full-time employees and expects to incur a non-recurring charge of between $1.0 million and $1.5 million in the third quarter of 2025. "We had a productive meeting with FDA; while this is not the outcome we had hoped for, we respect FDA's feedback and remain confident in the therapeutic potential of Cylembio," said Mai-Britt Zocca, PhD, CEO of IO Biotech. "We look forward to continuing the dialogue with FDA to align on the design for a potential new registrational study. Additionally, we plan to discuss the data from our IOB-013 study with European regulators and determine a path to submission in the EU." The company plans to design a new registrational study for Cylembio for the treatment of first-line patients with advanced melanoma and currently has capital to run its operations into the first quarter of 2026. IO Biotech is pursuing a pathway to regulatory approval while working to complete ongoing studies of its immune-modulatory, off-the-shelf therapeutic cancer vaccine candidate. Celcuity Inc. (NASDAQ: CELC) will present detailed efficacy and safety data from the PIK3CA wild-type cohort of the pivotal Phase 3 VIKTORIA-1 trial at the European Society of Medical Oncology Congress 2025 in Berlin, Germany. The late breaking abstract has been selected for an oral presentation on October 18, 2025 at 10:15 a.m. CEST in a Proffered Paper Session, with the presentation covering results from gedatolisib plus fulvestrant with or without palbociclib versus fulvestrant in patients with HR+/HER2-/PIK3CA wildtype advanced breast cancer. The VIKTORIA-1 trial has completed enrollment and reported topline data for the PIK3CA wild-type cohort, while the company continues enrolling patients for the PIK3CA mutant cohort. Celcuity is also conducting its VIKTORIA-2 Phase 3 trial evaluating gedatolisib plus a CDK4/6 inhibitor and fulvestrant as first-line treatment for HR+/HER2- advanced breast cancer, along with a Phase 1/2 trial combining gedatolisib with darolutamide in metastatic castration resistant prostate cancer patients. MAIA Biotechnology, Inc. (NYSE American: MAIA) has been awarded a $2.3 million grant by the National Institutes of Health for the expansion of its THIO-101 Phase 2 clinical trial evaluating ateganosine as a third-line treatment for patients with advanced non-small cell lung cancer. The grant, to be distributed over three years from 2025-2027, will support expenses related to enrollment of U.S. patients who are resistant to chemotherapy and immunotherapy, with Parts A and B of THIO-101 demonstrating median overall survival of 17.8 months for 22 patients in third-line treatment compared to 5 to 6 months for standard-of-care chemotherapy treatments. "We are thrilled to receive this prestigious NIH grant for the expansion of our Phase 2 trial," said Vlad Vitoc, M.D., CEO of MAIA Biotechnology. "It's a great honor to have the support of the National Institutes of Health as we seek to further validate the efficacy of our lead agent ateganosine and its potential to be a breakthrough treatment within the vastly underserved NSCLC market. With the clearance of FDA Investigational New Drug (IND) for THIO-101 in 2023, we can begin enrolling U.S. patients in the expansion phase of the trial immediately." The first patient in the expansion of the trial was dosed in July 2025 in Taiwan, with the NIH grant enabling MAIA to access a substantially larger patient pool across multiple continents. Ateganosine is a first-in-class investigational telomere-targeting agent that induces telomerase-dependent telomeric DNA modification and activates both innate and adaptive immune responses, currently being developed as a second or later line of treatment for NSCLC patients who have progressed beyond standard-of-care checkpoint inhibitor regimens. Article Sources: CONTACT: USA NEWS GROUP [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. This article is being distributed by USA News Group on behalf of Market IQ Media Group Inc. ("MIQ"). MIQ has been paid a fee for GT Biopharma, Inc. advertising and digital media from Creative Digital Media Group ("CDMG"). There may be 3rd parties who may have shares of GT Biopharma, Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ/BAY does not own any shares of GT Biopharma, Inc. but reserve the right to buy and sell, and will buy and sell shares of GT Biopharma, Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved on behalf of GT Biopharma, Inc. by CDMG; this is a paid advertisement, we currently own shares of GT Biopharma, Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment. SOURCES: 1. 2. 3. 4. 5. Logo - WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultImmunotherapyPhase 3Phase 2Phase 1

100 Deals associated with Sirexatamab

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Indication	Highest Phase	Country/Location	Organization	Date
Metastatic Colorectal Carcinoma	Phase 2	United States	-	22 Dec 2023
Metastatic Colorectal Carcinoma	Phase 2	Germany	-	22 Dec 2023
Metastatic Colorectal Carcinoma	Phase 2	South Korea	-	22 Dec 2023
Adenocarcinoma of large intestine	Phase 2	United States	Cypherpunk Technologies, Inc.	30 Aug 2022
Adenocarcinoma of large intestine	Phase 2	Germany	Cypherpunk Technologies, Inc.	30 Aug 2022
Adenocarcinoma of large intestine	Phase 2	South Korea	Cypherpunk Technologies, Inc.	30 Aug 2022
Advanced Colorectal Adenocarcinoma	Phase 2	United States	Cypherpunk Technologies, Inc.	30 Aug 2022
Advanced Colorectal Adenocarcinoma	Phase 2	Germany	Cypherpunk Technologies, Inc.	30 Aug 2022
Advanced Colorectal Adenocarcinoma	Phase 2	South Korea	Cypherpunk Technologies, Inc.	30 Aug 2022
Gastroesophageal junction adenocarcinoma	Phase 2	United States	BeOne Medicines Ltd.	29 Jul 2020

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05480306 (DeFianCe, ESMO2025)	Phase 2	Microsatellite Instability cancer Second line MSS \| BRAF wildtype	188	Sirexatamab (DKN-01) + FOLFIRI/FOLFOX + bevacizumab	egieyyqicd(isfzvhophl) = szutkxcxsf bqcushgbnk (jafkafgpxm ) View more	Positive	17 Oct 2025
				FOLFIRI/FOLFOX + bevacizumab	egieyyqicd(isfzvhophl) = hlnugqqcvu bqcushgbnk (jafkafgpxm ) View more
NCT04363801 (DisTinGuish, Pubmed) Manual	Phase 2	Gastroesophageal junction adenocarcinoma Second line DKK1 Expression	52	DKN-01+tislelizumab	zilsgxktoh(roqgxgvhky) = ftsixdaypx wczrwokiik (appxkbeups ) Met View more	Positive	10 Jul 2025
NCT05480306 (DeFianCe, Company_Website) Manual	Phase 2	Colorectal Cancer Second line DKK1 Expression \| Microsatellite Stable (MSS)	188	Sirexatamab + bevacizumab + chemotherapy (high DKK1 levels)	ritoohatwj(ofdisrblbi) = dexmnptarx srmydrlezx (tfwnyhxyiw ) View more	Positive	26 Mar 2025
				bevacizumab + chemotherapy (high DKK1 levels)	ritoohatwj(ofdisrblbi) = aqbcwgtpaw srmydrlezx (tfwnyhxyiw ) View more
NCT04363801 (DisTinGuish, PRNewswire) Manual	Phase 2	Gastrooesophageal junction cancer \| Advanced gastric carcinoma First line HER2 Negative	170	Sirexatamab + tislelizumab + chemotherapy	yxveoyyacz(abnpvofvpw) = puxfgvodhw oknectmasg (sxvdawpmud ) View more	Positive	28 Jan 2025
				Tislelizumab + chemotherapy	yxveoyyacz(abnpvofvpw) = kbbgqqceuw oknectmasg (sxvdawpmud ) View more
NCT05480306 (DeFianCe, PRNewswire) Manual	Phase 2	Microsatellite Stable Colorectal Carcinoma Second line Microsatellite Stable (MSS)	188	Sirexatamab + bevacizumab + chemotherapy	pqjbamfupk(boqjgtvrue) = qsfotxvxue sbjjaqjoth (duwoprarzd ) View more	Positive	28 Jan 2025
				bevacizumab + chemotherapy	pqjbamfupk(boqjgtvrue) = oobdrojeod sbjjaqjoth (duwoprarzd ) View more
NCT04363801 (DisTinGuish, Pubmed \| J Clin Oncol)	Phase 2	Advanced Gastroesophageal Junction Adenocarcinoma First line DKK1-high tumor \| DKK1-low tumor	25	DKN-01	kvrpuzmjgl(tobmkjakom) = datrfqpsgq vjmkgalyey (ptwkndkwhy, 49.8 - 89.3) View more	Positive	21 Oct 2024
NCT05480306 (DeFianCe, NEWS) Manual	Phase 2	Microsatellite Stable Colorectal Carcinoma	33	DKN-01	nokkixjjdr(ghnecjhued) = xphhdozgyi euatmvszve (wyjfneehlm ) View more	Positive	03 Jun 2024
				DKN-01 (左结肠肿瘤)	nokkixjjdr(ghnecjhued) = qdnslynpnf euatmvszve (wyjfneehlm ) View more
NCT04057365 (CTgov)	Phase 2	Advanced Bile Duct Carcinoma	15	Nivolumab+DKN-01 (DKN 01 and Nivolumab Safety Run in)	pwndguxmxy = ehxcxhuxvo emnhskhtob (wtfxacihhn, ushlfapmcq - rkwfjicfnf) View more	-	10 Apr 2024
				Nivolumab+DKN-01 (DKN 01 and Nivolumab)	lwvxnbjsfj(tznkohzbgi) = jyjwzefdov hoeqkvzkem (kumovmibxg, hhucsmuyhf - gwnazgagwr) View more
NCT05480306 (DeFianCe, ASCO_GI2024) Manual	Phase 2	Microsatellite Stable Colorectal Carcinoma Second line Microsatellite Stable (MSS)	33	DKN-01 + FOLFIRI/FOLFOX + bevacizumab	yafgifbjjs(fidmwjkwez) = vzixghqbwd uiuyhfnwck (elyzohnqim ) View more	Positive	18 Jan 2024
NCT03837353 (CTgov)	Phase 1/2	Advanced Prostate Carcinoma	18	DKN-01+Docetaxel (Cohort 1A.1 (DKN-01 300mg, Docetaxel 75 mg/m2))	wbbeshwwtw(zkntfvstyb) = fbtmmskvsc mmvewekkcc (koenoabmdm, 0) View more	-	18 Dec 2023
				DKN-01+Docetaxel (Cohort 1A.2 (DKN-01 600mg, Docetaxel 75 mg/m2))	wbbeshwwtw(zkntfvstyb) = vizahbalhz mmvewekkcc (koenoabmdm, 0) View more

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