A study of tolododekin alfa (also known as ANK-101) administered prior to surgery in kidney transplant participants that also have high-risk cutaneous squamous cell carcinoma (CSCC).

Start Date15 Dec 2025

Sponsor / Collaborator

Ankyra Therapeutics, Inc.

NCT07027514

/ RecruitingPhase 1

A Phase 1b, Two-Part Study of Tolododekin Alfa (ANK-101) in Combination With an Anti-PD-1/PD-L1 Antibody in Participants With Advanced Non-Small Cell Lung Cancer

A study of tolododekin alfa (also known as ANK-101) administered in combination with an anti-programmed cell death protein 1 (PD-1)/programmed cell death ligand 1 (PD-L1) antibody in participants with advanced or metastatic non-small cell lung cancer (NSCLC). Cohort A will enroll participants who have progressed on prior standard of care treatment with an anti-PD-1/PD-L1 antibody and a platinum-based chemotherapy regimen. Cohort B will enroll participants who are treatment-naïve for locally advanced or metastatic NSCLC.

Start Date22 Oct 2025

Sponsor / Collaborator

Ankyra Therapeutics, Inc.

NCT06171750

/ RecruitingPhase 1

A Phase I Open-Label, Dose Escalation Study of the Safety and Tolerability of Tolododekin Alfa (ANK-101) in Advanced Solid Tumors

This is a Phase 1, multicenter, open-label dose escalation study to determine the safety and tolerability of intratumoral (IT) injection of tolododekin alfa (ANK-101) in participants with advanced solid tumors who have progressed during or after receiving standard of care (SOC) therapy or who will not benefit from such therapy. The study will be conducted in three parts; in Part 1, participants with superficial lesions will receive ANK-101 as a single agent; in Part 2, participants with visceral lesions will receive ANK-101 as a single agent; and in Part 3, participants with cutaneous squamous cell carcinoma (CSCC) will receive ANK-101 in combination with cemiplimab.

Start Date19 Jan 2024

Sponsor / Collaborator

Ankyra Therapeutics, Inc.

100 Clinical Results associated with Tolododekin Alfa

100 Translational Medicine associated with Tolododekin Alfa

100 Patents (Medical) associated with Tolododekin Alfa

Literatures (Medical) associated with Tolododekin Alfa

02 Jul 2025·MOLECULAR CANCER THERAPEUTICS

A Pharmacokinetic and Pharmacodynamic Model of an IL-12 Anchored-Drug Conjugate for the Treatment of Solid Tumors

Article

Author: Battula, Sailaja ; Hodson, David ; Tighe, Robert ; Kaufman, Howard L. ; Mistry, Hitesh B. ; Schmidt, Michael M. ; Chassagnole, Christophe

Abstract:

IL-12 mediates innate and adaptive immune responses and has demonstrated therapeutic antitumor activity, but clinical development has been hindered by a narrow therapeutic window. We generated a novel IL-12–anchored drug conjugate by physiochemical linking of murine IL-12 to aluminum hydroxide (alum). The complex was designed to utilize alum as a scaffolding for durable retention of IL-12 within the tumor microenvironment as a strategy to increase the therapeutic window. To better define the systemic pharmacokinetic (PK) profile of the anchored IL-12 (mANK-101), a model-based assessment tool was developed to describe the systemic PK profile and downstream signaling factors following intratumoral injection of mANK-101. When compared with nonanchored IL-12, mANK-101 exhibited a distinct PK profile. Specifically, mANK-101 treatment was associated with a significant ninefold increase in the systemic terminal volume of distribution (Vd). Furthermore, linear mixed-effects models provided evidence that CD8+ T-cell infiltration and increased serum IFN-γ levels were correlated with tumor regression after a single dose of mANK-101. In addition, PK/pharmacodynamic modeling confirmed a link between systemic IL-12 and serum IFN-γ. The model also suggests that the anchored IL-12 drug conjugate is expected to prolong the absorption half-life (115 hours vs. 8 hours for the unanchored drug) with durable local retention and limited systemic absorption. In addition, serum IFN-γ may be a surrogate marker for drug activity. The PK modeling predictions may also contribute to determining the optimal clinical dose and schedule of mANK-101 and other anchored drug conjugates in patients with solid tumors.

08 Dec 2023·JCI insight

Intratumoral aluminum hydroxide–anchored IL-12 drives potent antitumor activity by remodeling the tumor microenvironment

Article

Author: Kaufman, Howard L ; Battula, Sailaja ; Schmidt, Michael M ; Wittrup, K Dane ; Papastoitsis, Gregory

IL-12 is a potent cytokine that can promote innate and adaptive anticancer immunity, but its clinical development has been limited by toxicity when delivered systemically. Intratumoral (i.t.) administration can expand the therapeutic window of IL-12 and other cytokines but is in turn limited by rapid drug clearance from the tumor, which reduces efficacy, necessitates frequent administration, and increases systemic accumulation. To address these limitations, we developed an anchored IL-12 designated ANK-101, composed of an engineered IL-12 variant that forms a stable complex with the FDA-approved vaccine adjuvant aluminum hydroxide (Alhydrogel). Following i.t. administration of murine ANK-101 (mANK-101) in early intervention syngeneic mouse tumors, the complex formed a depot that was locally retained for weeks as measured by IVIS or SPECT/CT imaging, while unanchored protein injected i.t. was cleared within hours. One or 2 i.t. injections of mANK-101 induced single-agent antitumor activity across a diverse range of syngeneic tumors, including models resistant to checkpoint blockade at doses where unanchored IL-12 had no efficacy. Local treatment with mANK-101 further induced regressions of noninjected lesions, especially when combined with systemic checkpoint blockade. Antitumor activity was associated with remodeling of the tumor microenvironment, including prolonged IFN-γ and chemokine expression, recruitment and activation of T and NK cells, M1 myeloid cell skewing, and increased antigen processing and presentation. Subcutaneous administration of ANK-101 in cynomolgus macaques was well tolerated. Together, these data demonstrate that ANK-101 has an enhanced efficacy and safety profile and warrants future clinical development.

Nature Communications

Interleukin-12 anchored drug conjugate (tolododekin alfa) in patients with advanced solid tumors: first-in-human Phase 1 trial

Article

Author: Battula, Sailaja ; Wehrenberg-Klee, Eric ; Iodice, Gail ; Tighe, Robert ; Park, Jong Chul ; Curti, Brendan ; Butler, Marcus ; Elassal, Joseph ; Kirkwood, John M ; Kaufman, Howard L

Anchored immunotherapy is a novel approach for retaining drugs within the tumour microenvironment. Tolododekin alfa is a first-in-class anchored Interleukin 12 (IL-12) linked to aluminum hydroxide. Safety and biological activity were evaluated in a Phase 1 clinical trial in patients with accessible advanced solid tumours in Part 1 of a 3-part trial. The primary objectives are safety and tolerability as well as determination of the recommended dose for expansion (RDE). Secondary objectives include pharmacokinetics (PK), pharmacodynamics, preliminary antitumour activity, and immunogenicity. Exploratory analyses include serum and tissue biomarkers. Fifteen patients were enrolled at escalating doses of tolododekin alfa given by injection every 3 weeks. There were no dose-limiting toxicities or treatment-related serious adverse events. PK/Pharmacodynamic measurements demonstrate retention of drug in the tumour. Biological activity demonstrates increased CD8⁺ T cells, Programmed Cell Death Ligand 1 (PD-L1) expression, and prolonged pro-inflammatory gene expression. Nine patients (60%) achieved stable disease with one partial response. At a median follow-up of 5.2 months, the median duration of stable disease was 5.3 months (range 3.6-7.6 months). Median progression-free survival (PFS) was not estimable (NE) (95% CI, 2.57 months - NE). These findings support continued clinical development of tolododekin alfa. ClinicalTrials.gov registration: NCT06171750.

News (Medical) associated with Tolododekin Alfa

17 Nov 2025

·www.businesswire.com

Ankyra Therapeutics Announces First Patient Dosed in Phase 1b Trial Evaluating First-in-Class Anchored Immunotherapy in Combination with Immune Checkpoint Blockade in Patients with Lung Cancer

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ankyra Therapeutics, a clinical-stage biotechnology company pioneering anchored drug conjugate technology for cancer and other diseases, today announced that the first patient has been dosed in its ANK-101-004 clinical trial (NCT07027514). This study will evaluate the combination of Ankyra’s tolododekin alfa (ANK-101), an anchored IL-12 drug conjugate with the anti-PD1 agent, cetrelimab, in patients who have progressed after initial treatment of metastatic, non-mutated non-small cell lung cancer (NSCLC). In addition, the study will evaluate tolododekin alfa in combination with standard of care immune checkpoint blockade in first-line treatment of patients with metastatic, non-mutated NSCLC and a tumor proportion score (TPS) of ≥ 50%. “The potential use of anchored IL-12 to drive better responses without adding appreciable toxicity could be an important advance for patients.” “While there has been considerable progress in the treatment of lung cancer many patients do not respond to current approaches”, said Thomas Marron, MD, PhD, Director of the Early Phase Trials Unit at the Tisch Cancer Institute at Mount Sinai and Chief Medical Officer of OCCAM Immune and the principal investigator of the study. “The potential use of anchored IL-12 to drive better responses without adding appreciable toxicity could be an important advance for patients.” “Tolododekin alfa has already demonstrated objective responses as a single agent in patients with anti-PD-1-refractory cancers in phase 1 trials”, stated Howard L. Kaufman, MD. CEO at Ankyra Therapeutics, “and now we have an opportunity to test tolododekin in patients with advanced lung cancer.” Ankyra is working with OCCAM Immune at Mount Sinai in support of Ankyra the LANTERN clinical trial, which is evaluating ANK-101 in combination with PD-1 blockade in patients with first- and second-line NSCLC. Through state-of-the-art immune profiling, OCCAM Immune aims to generate a comprehensive immune atlas to correlate immune modulation with therapeutic efficacy and disease progression, advance biomarker discovery and deepen mechanistic insights into Ankyra’s novel therapeutic approach. The ANK-101-004 (LANTERN) trial will be conducted at several institutions, including, Icahn School of Medicine at Mount Sinai, Roswell Park Cancer Institute, Mayo Clinic, Moffitt Cancer Center, The University of Chicago, Community Health Network, The University of Miami Sylvester Comprehensive Cancer Center, Karmanos Cancer Institute, FirstHealth of the Carolinas, and OSF Saint Francis Medical Center with additional sites expected as well. About Tolododekin alfa (ANK-101) Tolododekin alfa (ANK-101) is an anchored drug conjugate composed of interleukin-12 (IL-12) linked to aluminum hydroxide. ANK-101 enables local delivery of functional IL-12 to the tumor microenvironment where it remains biologically active for several weeks with transient exposure to the systemic circulation, thereby avoiding systemic toxicity. Treatment with ANK-101 in animal models has been associated with immune activation and rapid tumor regression. ANK-101 is being evaluated for the treatment of advanced solid tumors alone and in combination with anti-PD-1 agents. The first-in-human clinical trial of ANK-101 (NCT06171750) consists of monotherapy dose escalation, dose expansion in combination with cemiplimab, and dose optimization cohorts. The ANK-101-004 clinical trial (NCT07027514) will focus on non-mutated metastatic non-small cell lung cancer. About Ankyra Therapeutics Ankyra Therapeutics is a biotechnology company that has developed a highly differentiated technology platform that expands the therapeutic window of therapeutic drugs by forming a stable depot after local administration leading to prolonged immune activation and potent local and systemic immunity with reduced systemic toxicity. Ankyra is headquartered in Cambridge, Massachusetts. For more information, please visit www.ankyratx.com.

ImmunotherapyPhase 1

05 Nov 2024

·www.businesswire.com

Ankyra Therapeutics Announces First Visceral Tumor Patient Dosed in Phase 1 ANCHOR Clinical Trial of ANK-101, an Anchored Interleukin-12 (IL-12)

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Ankyra Therapeutics, a clinical stage biotechnology company pioneering anchored therapy to treat cancer, today announced the first patient dosed in a visceral tumor in Part 2 of their dose escalation Phase 1 study. The ANCHOR study is a first-in-human Phase 1 study being conducted in two parts. Part 1 is enrolling patients with superficially accessible tumors and is expected to complete enrollment by December 2024. Part 2 is now open to patients with solid tumors located in deep viscera accessed by interventional radiologic or endoscopic procedures for injection. Referred to as “anchored therapy” the company has identified a method for prolonged drug retention within the tumor microenvironment. The anchored strategy drives local anti-tumor activity, similar to antibody drug conjugates, while limiting systemic toxicity. The company’s lead asset, ANK-101, is an interleukin-12 (IL-12) physically anchored to aluminum hydroxide. ANK-101 is locally delivered and retained in the tumor microenvironment for several weeks where it mediates recruitment and activation of effector immune cells. In multiple preclinical models, a murine adapted ANK-101 demonstrated increased immune cell infiltration and activation with significant therapeutic activity across multiple tumor types. In addition, a canine version of ANK-101 has been evaluated in an exploratory phase I study in dogs with advanced melanoma. Human ANK-101 is now in Phase 1 clinical trials for solid tumors in the U.S. and Canada. “The first patient dosed in the Part 2 of our Phase 1 study advances our lead asset into visceral solid tumors and marks a key milestone in our mission to bring novel medicines to patients with cancer,” said Joe Elassal, M.D. MBA. Part 2 of the ANCHOR study will assess the safety of ANK-101 and determine the recommended dose for expansion in patients with viscerally injected solid tumors. Secondary objectives include evaluation of pharmacokinetics, immunogenicity, and preliminary clinical activity of the medicine. Enrollment in Part 1 of the ANCHOR trial is ongoing. “I have been impressed with ANK-101 in Part 1 of the Phase 1 study and expansion of the trial to include visceral lesions will enable us to deliver ANK-101 to a broader group of cancer patients,” said Jong Chul Park, MD, Assistant Professor at Harvard Medical School and attending physician at Mass General Cancer Center. He added, “we look forward to advancing this part of the trial and exploring the safety and therapeutic potential of ANK-101 for patients with more advanced disease.“ About ANK-101 ANK-101 is an anchored drug complex composed of human interleukin-12 (IL-12) linked to aluminum hydroxide. ANK-101 enables local delivery of functional IL-12 to the tumor microenvironment where it remains biologically active for several weeks but does not diffuse into the systemic circulation, thereby avoiding systemic toxicity. Treatment with ANK-101 in animal models has been associated with recruitment and retention of CD8+ T cells, NK cells and M1 macrophages activating innate and adaptive anti-tumor immunity. ANK-101 is being evaluated for the treatment of advanced solid tumors alone and in combination with anti-PD-1 agents. Phase 1 first-in-human, open-label clinical trial of ANK-101 as a monotherapy (NCT:06171750) consists of dose escalation portions in both superficial and visceral lesions that will evaluate the safety and tolerability of ANK-101, followed by dose expansion cohorts. About Ankyra Therapeutics Ankyra Therapeutics is a biotechnology company pioneering anchored therapy to transform cancer treatment. The company’s platform is fueling a pipeline of novel therapeutics, including cytokine therapies, designed to anchor to the tumor microenvironment for sustained local delivery and retention at higher concentrations, while minimizing systemic exposure and on-target/off-tumor effects. Ankyra’s lead program ANK-101, IL-12 anchored to aluminum hydroxide, has potential to treat a broad range of cancers. For more information, please visit www.ankyratx.com .

Phase 1

23 Sep 2024

·www.businesswire.com

Ankyra Announces Appointment of Dr. Julia Butchko and Ms. Tara Withington to the Board of Directors

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Ankyra is pleased to announce the appointment of Dr. Julia G. Butchko and Ms. Tara Withington to the Board of Directors effective September 25, 2024. They will both serve as independent board members and their appointment brings the total board composition to seven directors. “We are delighted to welcome Dr. Butchko and Ms. Withington to the Board”, stated Tillman Gerngross, PhD, Chairman of the Ankyra Board of Directors. “These accomplished individuals bring a fresh perspective to Ankyra at a critical time in the company’s growth.” Howard L. Kaufman, MD, President and Chief Executive Officer at Ankyra added “I am especially enthusiastic about working with Dr. Butchko and Ms. Withington since they bring considerable strengths in research and development as well as representing the patient perspective as we pursue our mission of delivering new drugs to patients with cancer.” Julia G. Butchko, Ph.D. brings 25 years of industry experience and most recently served as Chief Development Officer at Immunovant (NASDAQ: IMVT), a biopharmaceutical company focused on developing therapies for patients with autoimmune disease. Dr. Butchko joined Immunovant in October 2019 and has co-led Research and Development to advance batoclimab (IMVT-1401) from early development to multiple, global registration trials and IMVT-1402 from candidate identification through registration trial planning. Prior to Immunovant, Dr. Butchko held various leadership roles at Eli Lilly and Company where she most recently served as the Chief of Staff, Strategy and Operations for Lilly’s Immunology and Neuroscience businesses and Vice President, Lilly Oncology Portfolio Management and Clinical Development. Dr. Butchko earned a B.S. in Chemistry from the State University of New York at Fredonia, a Ph.D. in Chemistry from Pennsylvania State University, and a Business Certificate from Indiana University, Kelly School of Business. Dr. Butchko stated that “I am delighted to be joining Ankyra’s Board of Directors at this exciting time. This year will be an impactful one for the Company as the ANK-101 Phase 1 program expands, and the Company advances discovery and early development efforts. I believe Ankyra’s anchoring technology holds a breadth of potential to improve the treatment for cancer patients and look forward to working with the senior leadership team as the Company continues to grow.” Tara Withington has over 40 years’ executive experience serving the nonprofit sector. Currently she is a principal and Vice President with Executive Director, Incorporated (EDI), an association management company that manages more than 40 scientific organizations and professional boards. She takes special interest in improving organizational excellence, including board development and infrastructure improvement. She also has a keen ability to forecast influences impacting associations and offers innovative ideas and an entrepreneurial mindset to position organizations to take advantage of opportunities and identify challenges allowing organizations to pivot for optimal outcomes. She has previously served as executive director or consulting partner for over 20 organizational boards. Ms. Withington served as a member of the Board of Directors for the AMC Institute and is a member in good standing in the American Society of Association Executives (ASAE), where she currently serves as a member of the Foresight Advisory Task Force. In 2001, the Society for Immunotherapy of Cancer (SITC) joined EDI and Ms. Withington began her tenure as the Executive Director and principal staff member. Tara has been instrumental in positioning the society as a universally respected organization known for its dedication to advancing the science and application of cancer immunotherapy both in the US and abroad. Ms. Withington holds a degree in Business Management as well as the Certified Association Executive (CAE) designation. In 2020 she received the Society for Immunotherapy of Cancer inaugural Lifetime Achievement Award. She currently serves as the Executive Director Emeritus of SITC Ms. Withington states “I am especially pleased to be joining the Board at Ankyra at this exciting time in their development.” She added that immunotherapy “offers a chance for durable responses and better patient experience, and I share the Ankyra team’s vision of providing new drugs for patients with cancer while ensuring broad access to the drug for patients who may benefit.” Dr. Howard L. Kaufman, President and CEO at Ankyra added “I am delighted to welcome Dr. Butchko and Ms. Withington to the Ankyra Board of Directors. They bring valuable expertise to the company and will add diversity and new perspectives on our goal of advancing the research and development of our anchoring platform and our commitment to doing so in a patient-focused manner.” Ankyra’s lead asset, ANK-101, is an anchored IL-12 that entered phase I clinical trials earlier this year and now over 10 patients with advanced solid cancers have been treated at multiple study sites in the United States and Canada (NCT:06171750). About Ankyra Therapeutics Ankyra Therapeutics is a biotechnology company that has developed a highly differentiated technology platform that expands the therapeutic window of cytokine drugs by forming a stable depot in the tumor after local administration leading to prolonged immune activation and potent local and systemic immunity with reduced systemic toxicity. Ankyra was founded in 2019 and is headquartered in Cambridge, Massachusetts. For more information, please visit www.ankyratx.com

Executive ChangePhase 1

100 Deals associated with Tolododekin Alfa

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Solid tumor	Phase 1	-	Ankyra Therapeutics, Inc.	15 Dec 2025
Advanced Lung Non-Small Cell Carcinoma	Phase 1	United States	Ankyra Therapeutics, Inc.	22 Oct 2025
Advanced Malignant Solid Neoplasm	Phase 1	United States	Ankyra Therapeutics, Inc.	19 Jan 2024
Advanced Malignant Solid Neoplasm	Phase 1	Canada	Ankyra Therapeutics, Inc.	19 Jan 2024
Cutaneous Squamous Cell Carcinoma	Phase 1	United States	Ankyra Therapeutics, Inc.	19 Jan 2024
Cutaneous Squamous Cell Carcinoma	Phase 1	Canada	Ankyra Therapeutics, Inc.	19 Jan 2024
metastatic non-small cell lung cancer	Phase 1	United States	Ankyra Therapeutics, Inc.	19 Jan 2024
metastatic non-small cell lung cancer	Phase 1	Canada	Ankyra Therapeutics, Inc.	19 Jan 2024
Metastatic Solid Tumor	Phase 1	United States	Ankyra Therapeutics, Inc.	19 Jan 2024
Metastatic Solid Tumor	Phase 1	Canada	Ankyra Therapeutics, Inc.	19 Jan 2024

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06171750 (SITC2025)	Phase 1	Solid tumor NKT cells	15	ANK-101 (solid tumors)	foyjlubqrr(qyizpkyhxq) = uwrreogzoe kxzfkmjgku (gwfacdmctc )	Positive	05 Nov 2025
NCT06171750 (Pubmed \| Nat Commun)	Phase 1	Advanced Malignant Solid Neoplasm	15	Tolododekin alfa	ulqxrcezhz(zsaabcjvax) = bsogduzofj dkaapamhgb (ankwnkrjnx, 3.6 - 7.6) View more	Positive	29 Sep 2025

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