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Last update 27 Jun 2026

ZT-01

Last update 27 Jun 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

ZT 01, ZT-01

Target

SSTR2

Action

antagonists

Mechanism

SSTR2 antagonists(Somatostatin receptor 2 antagonists)

Therapeutic Areas

Immune System DiseasesEndocrinology and Metabolic Disease

Active Indication

Diabetes Mellitus, Type 1Hypoglycemia

Inactive Indication

Diabetes Mellitus, Type 2

Originator Organization

York University

Tulane University Hospital & Clinic

University of Toronto

Active Organization

Zucara Therapeutics, Inc.

Inactive Organization

New York University

License Organization

Zucara Therapeutics, Inc.

Sanofi

Drug Highest PhasePhase 2

First Approval Date-

Regulation-

Clinical Trials associated with ZT-01

NCT05762107

/ RecruitingPhase 2

A Phase 2a Randomized, Double-blind, Placebo-controlled, Multiple-dose, Crossover Study of the Effect of ZT-01 on Frequency of Nocturnal Hypoglycemia in Type 1 Diabetes Mellitus

The goal of this clinical trial is to learn about the effect of the study drug (ZT-01) on low blood sugar (hypoglycemia) in adults with type 1 diabetes (T1D) who have been having low blood sugars ("hypos") at night. ZT-01 increases the amount of a hormone called glucagon during low blood sugar, and this may help prevent the occurrence of hypos. The main questions this trial aims to answer are whether ZT-01 lowers the number of hypos happening at night, and what its effects are on blood sugar levels. The safety of ZT-01 will also be measured.
Participants will be asked to wear a study-provided continuous glucose monitor (CGM) during two 4-week periods when they will self-inject the study drug before bed. They will get ZT-01 at one of three dose levels during one period, and placebo (which looks like the study drug but doesn't contain the active ingredient) during the other. Neither the participant nor the study site will know what they are receiving during each treatment period or see data from the CGM. The participant will continue to use their usual methods of measuring blood sugar (including their personal CGM) and giving insulin during the study. The participant will be asked to complete a short diary each evening, and will be asked to upload the CGM data to a study phone every day.
If a participant uses their own CGM and is willing to share information on how often they have low blood sugar with the study site at the first visit to see if they meet study entry requirements, they will have 6 study visits, 2 study phone calls, and be in the study for about 16 weeks. If they don't use CGM or don't want to share their information, then they will be asked to wear a study CGM for an extra 4 weeks to find out how many low blood sugars they have, and will have an extra visit.
Study participants will be asked to give blood and urine for testing to see whether they meet the requirements to enter the study, and at the start and end of each treatment period to see if the study treatment has any effects. They will also have their blood pressure and temperature taken at each study visit, and have an ECG at 4 visits to measure the electrical activity of their heart.
Some participants will be asked to also take part in a sub-study where their blood level of ZT-01 and glucagon is measured, after the first and last dose. They will be asked to stay at the study site overnight for each set of measurements (4 in total).

Start Date28 Jul 2023

Sponsor / Collaborator

Zucara Therapeutics, Inc.

NCT05007977

/ TerminatedPhase 1

Pharmacodynamic Evaluation of ZT-01 in a Stepped Hypoglycemic Clamp Model in Subjects With Type 1 Diabetes Mellitus

The purpose of this study is to determine whether ZT-01 increases the glucagon response to hypoglycemia in type 1 diabetes (T1D). ZT-01 is a specific somatostatin (SST) type 2 receptor antagonist hypothesized to increase the counterregulatory glucagon release during hypoglycemia, which is impaired in diabetes, and may thus reduce the occurrence of hypoglycemia.
Approximately 25 participants with well-controlled type 1 diabetes will be recruited to a crossover study and be randomized to the order in which they receive a single administration of placebo, low dose and high dose ZT-01 during three euglycemic-hypoglycemic clamps 2 to 6 weeks apart, with a follow-up visit approximately 1 week after the final clamp.
Participants will stay overnight in the clinic before each clamp, and their plasma glucose (PG) will be kept at euglycemic levels with IV insulin and dextrose as needed. The following morning, the clamp will begin and the subject's PG will be kept at 5.5 mmol/L (euglycemia) with variable IV insulin (and dextrose as needed). Study treatment will be administered during this euglycemic period, and then insulin will be increased so that PG falls to a plateau of mild hypoglycemia, and then increased further to clinically significant hypoglycemia. Release of glucagon and other counterregulatory hormones will be measured, and a symptom score will be completed, during euglycemia before and following study treatment, and during each level of hypoglycemia. Following the clamp, the participant's blood glucose will be returned to normal levels prior to leaving the clinic.

Start Date08 Jul 2021

Sponsor / Collaborator

Zucara Therapeutics, Inc.

100 Clinical Results associated with ZT-01

100 Translational Medicine associated with ZT-01

100 Patents (Medical) associated with ZT-01

Literatures (Medical) associated with ZT-01

01 Dec 2025·IMMUNOLOGIC RESEARCH

From immunosuppression to immune reprogramming: is triptolide a potential “immune reset” agent in autoimmune diseases?

Review

Author: Yu, Dehui ; Lin, Hu

Current biologics and small-molecule inhibitors for autoimmune diseases often provide symptomatic relief but fail to restore immune tolerance, necessitating lifelong treatment with associated risks. Triptolide, a natural compound from Tripterygium wilfordii Hook F, exhibits a unique capacity for immune reprogramming, simultaneously suppressing pathogenic immunity while enhancing regulatory functions, positioning it as a potential 'immune reset' agent. However, its clinical translation is plagued by a narrow therapeutic window due to mechanism-based toxicity, creating a critical challenge of decoupling efficacy from toxicity. This review moves beyond a descriptive cataloguing of triptolide derivatives to provide a critical appraisal of the field's progress in achieving this decoupling. We systematically evaluate the most promising candidates (e.g., LLDT-8, Minnelide, ZT01), not only examining their mechanisms but also analyzing why most stall in early development. By integrating mechanistic insights with clinical progress data, we dissect the structural determinants of toxicity and efficacy and propose a concrete future roadmap focused on rational drug design (e.g., novel targets like TAK1), targeted delivery systems, and biomarker-driven precision medicine to advance safe and effective triptolide-based therapies to the clinic.

01 Jun 2023·Diabetes, obesity & metabolism

Effects of somatostatin receptor type 2 antagonism during insulin‐induced hypoglycaemia in male rats with prediabetes

Article

Author: Atherley, Sara ; Liggins, Richard ; D'Souza, Ninoschka C. ; Hoffman, Emily G. ; Aiken, Julian ; Riddell, Michael C.

Abstract:

Aims:

To examine if glucagon counterregulatory defects exist in a rat model of prediabetes (pre‐T2D) and to assess if a selective somatostatin receptor 2 antagonist (SSTR2a), ZT‐01, enhances the glucagon response to insulin‐induced hypoglycaemia.

Materials and methods:

Hyperglycaemia was induced in 8‐ to 9‐week‐old male, Sprague‐Dawley rats via 7 weeks of high‐fat diet followed by a single, low‐dose intraperitoneal injection of streptozotocin (30 mg/kg). After 2 weeks of basal insulin therapy (0‐4 U/d insulin glargine, administered subcutaneously [SC]) to facilitate partial glycaemic recovery and a pre‐T2D phenotype, n = 17 pre‐T2D and n = 10 normal chow‐fed control rats underwent the first of two hypoglycaemic treatment‐crossover experiments, separated by a 1‐week washout period. On each experimental day, SSTR2a (3 mg/kg ZT‐01, SC) or vehicle was administered 1 hour prior to insulin‐induced hypoglycaemia (insulin aspart, 6 U/kg, SC).

Results:

Glucagon counterregulation was marginally reduced with the induction of pre‐T2D. Treatment with SSTR2a raised peak plasma glucagon levels and glucagon area under the curve before and after insulin overdose in both and pre‐T2D rats. Blood glucose concentration was elevated by 30 minutes after SSTR2a treatment in pre‐T2D rats, and hypoglycaemia onset (≤3.9 mmol/L) was delayed by 15 ± 12 minutes compared with vehicle ( P < 0.001), despite similar glucose nadirs in the two treatment groups (1.4 ± 0.3 mmol/L). SSTR2a treatment had no effect on blood glucose levels in the control group or on the hypoglycaemia‐induced decline in plasma C‐peptide levels in either group.

Conclusions:

Treatment with an SSTR2a increases glucagon responsiveness and delays the onset of insulin‐induced hypoglycaemia in this rat model of pre‐T2D where only a modest deficiency in glucagon counterregulation exists.

01 May 2021·International immunopharmacologyQ2 · MEDICINE

Exploring a novel triptolide derivative possess anti-colitis effect via regulating T cell differentiation

Q2 · MEDICINE

Article

Author: Zhou, Yu ; Chen, Chengjuan ; Sun, Tao ; Wu, Lei ; Zhang, Tiantai ; Zhang, Dongming ; Zang, Yingda ; Fu, Junmin ; Shao, Shuai ; Shi, Gaona ; Zhu, Guoyuan

Inflammatory bowel disease (IBD) is generally characterized by chronic inflammatory disorders of the gastrointestinal tract that are known as ulcerative colitis (UC) or Crohn's disease (CD). Although the underlying mechanism of action of IBD is unclear and because of the lack of satisfactory treatment, increasing evidence has indicated that pro-inflammatory cytokines that activate JAK-STAT signaling pathway regulate the differentiation of naïve T cells towards T helper (Th)1 and Th17 cell subsets and contribute to the development of IBD. ZT01 is a newly obtained triptolide derivative with strong anti-inflammatory effects and low toxicity. In this study, we evaluated the effects of ZT01 on DSS-induced colitis and investigated the underlying mechanism of action involved. Mice with DSS-induced acute or chronic colitis were used to assess the efficacy of ZT01 treatment, and T cells were cultured to analyze the differentiation of Th1 and Th17 cell by flow cytometry. In addition, intestinal epithelial barrier function, macrophage polarization, activation of the JAK-STAT signaling pathway, and the expression of cytokines and transcription factors were measured to assess the possible mechanisms of ZT01. We found that ZT01 had an obviously beneficial effect on DSS-induced colitis by improving the symptoms of bloody diarrhea, weight loss, and a shortened colon, thereby preserving the epithelial barrier function in the mouse colon. Furthermore, ZT01 significantly inhibited T cell differentiation into Th1 and/or Th17 cell subsets and macrophage polarization towards into an inflammatory phenotype via regulating the JAK-STAT signaling pathway. Thus, our findings suggested that ZT01 might be a potential pharmaceutical candidate that deserves to be further investigated as a treatment for IBD patients.

News (Medical) associated with ZT-01

02 Oct 2025

·www.mobihealthnews.com

QuantHealth, Sanofi Ventures team up to advance clinical trial simulations

AI-enabled clinical trial simulation company QuantHealth announced a strategic investment from Sanofi Ventures , the venture capital division of biopharmaceutical company Sanofi , bringing its total funds raised to $30 million. Cris De Luca, partner at Sanofi Ventures, will join QuantHealth's board of directors as an observer. WHAT IT DOES QuantHealth's AI-powered clinical simulator predicts how an individual in a clinical trial will respond to treatment. The aim is to assist pharmaceutical companies in improving their clinical trial design. According to QuantHealth, its trial design teams virtually test thousands of clinical hypotheses, assessing the impact of various inclusion/exclusion criteria, treatment, and control arms and endpoints. The platform also allows the trial design team to modify any protocol parameter and assess its clinical, operational and commercial impact. QuantHealth's AI-based indication selection module predicts the efficacy and safety of drugs across various medical conditions and diseases. The company will use the funds to expand its patient-level simulations and digital twin technologies in the drug development space. "Sanofi is at the forefront of digital transformation in pharma and this relationship will help us scale our impact and bring more predictive, AI-driven approaches to clinical development," Orr Inbar, CEO and cofounder of QuantHealth, said in a statement. Sanofi Venture's De Luca said QuantHealth has the ability to change how clinical trials are designed and optimized. "Their approach to leveraging digital twins and real-world data is advancing the next generation of R&D, and I look forward to supporting the team," De Luca said in a statement. MARKET SNAPSHOT In May, QuantHealth unveiled its large real-world drug model, saying it can process and utilize data from more than 100 million patients and billions of therapeutic data points in a single simulation. That same month, Viz.ai partnered with Regeneron and Sanofi to deploy and study an AI-enabled workflow tool for the treatment of COPD . Through the partnership, Sanofi and Regeneron supported the company's investigation of its Viz COPD Module, which employs AI and electronic health record data, using natural language processing to detect and manage high-risk COPD patients. In 2024, OMNY Health , which helps hospitals monetize operational data, launched an AI-powered platform for health technology companies to leverage its data on more than 75 million patients . QuantHealth added OMNY's data to its existing AI technology, which is trained on a dataset of 350 million patients. The collaboration helped QuantHealth's pharmaceutical partners accelerate drug development through simulated clinical trials and predict clinical trial outcomes, patient responses and drug efficacy. That same year QuantHealth added additional funding to its Series A round , bringing its total Series A raise to $17 million. Accenture Ventures, a CRO firm and additional investors contributed to the additional Series A investment. In 2023, QuantHealth scored $15 million in a Series A funding round co-led by Pitango HealthTech and Bertelsmann Investments, bringing its total raise to $20 million. The company said it used the funds to develop its product, grow its platform, expand its offerings commercially and evaluate use cases beyond clinical trials, including early research and development and regulatory support. Zucara Therapeutics announced that Sanofi made a strategic investment in the company as part of its $20 million Series B financing. Sanofi received an exclusive right of first negotiation. Proceeds from the financing funded Zucara's Phase 2a trial of the effect of ZT-01 on nocturnal hypoglycemia events in Type 1 diabetes ("T1D") mellitus ("ZONE") and the nonclinical activities to support a once-weekly version of ZT-01.

Phase 2

01 May 2025

·www.prnewswire.com

Zucara Therapeutics Announces Second and Final Closing of US$25 Million Series B Financing

- T1D Fund Joins Financing Syndicate - - Proceeds to Fund Remainder of Phase 2a 'ZONE' Trial and Nonclinical Development of a Once-Weekly Version of ZT-01 - TORONTO, May 1, 2025 /PRNewswire/ - Zucara Therapeutics Inc., ("Zucara" or the "Company") a diabetes life sciences company developing ZT-01, the first once-daily therapeutic to prevent hypoglycemia (low blood glucose levels) in people with diabetes, today announced that it has completed the second and final closing of its US$25 million Series B Financing (the "Financing"). The T1D Fund: A Breakthrough T1D Venture and other new investors invested a combined US$5 million to complete the Financing. As previously announced, strategic investor, Sanofi, and existing investor, The Perceptive Xontogeny Venture Fund ("PXV Fund I"), invested a combined US$20 million as part of the Financing's first closing. "We are thrilled to close our Series B financing, which will enable us to complete our Phase 2a clinical trial and the nonclinical activities to support a once-weekly version of ZT-01," said Michael Midmer, CEO of Zucara. "As one of the largest disease-focused impact investing funds in the world, the T1D Fund focuses on funding therapies that have significant potential for clinical impact in Type 1 diabetes, so we are delighted to have their support." T1D Fund Managing Director, Sylvia Tobé, commented, "Our investment builds on funding that our parent organization, Breakthrough T1D, previously granted to Zucara to support the discovery and clinical development of ZT-01. Having followed Zucara for some time, we are excited to support the further advancement of this promising candidate that has the potential to address a common and potentially life-threatening complication of Type 1 diabetes." In connection to the Financing, Dr. Tobé will join Zucara as a Board Observer. Dr. Tobé is an experienced biotech industry professional with over a decade of experience in the sector. Prior to joining the T1D Fund, she was a Principal on the investment team at Omega Funds, where she focused on early-stage investments and company creation in various therapeutic areas. She also previously served at publicly traded Ra Pharmaceuticals, a peptide drug discovery company, where she provided scientific leadership for multiple discovery programs within its internal pipeline and in collaboration with Merck. Dr. Tobé is a Board Director at DiogenX and a Board Observer at various biotech companies across the auto-immune and regenerative spaces. Proceeds from the Financing are expected to fund the remainder of Zucara's ongoing Phase 2a trial of the effect of ZT-01 On Nocturnal hypoglycemia Events in Type 1 diabetes ("T1D") mellitus ("ZONE"), and the nonclinical development of a once-weekly version of ZT-01. About The T1D Fund The T1D Fund is an impact investment fund focused on accelerating life-changing solutions to treat, prevent, and ultimately cure type 1 diabetes (T1D). Launched in December 2016, the T1D Fund is a unique, evergreen construct that is the first scaled, mission-driven venture fund established to catalyze the development of T1D cure-oriented therapies through equity investments. By partnering with additional sources of capital, including venture capital, corporations, and foundations, the T1D Fund is able to attract and effectively deploy capital towards disease-modifying therapies and potential cures for T1D. Managed by a dedicated team of investment professionals and supported by its foundational partners - including Breakthrough T1D (formerly JDRF) and the Helmsley Charitable Trust, the leading global organizations funding T1D research - the T1D Fund continues to pursue innovative approaches that have the curative potential to make a significant impact for people living with T1D. For more information, visit t1dfund.org or follow the T1D Fund on LinkedIn. About ZT-01 ZT-01 is designed to prevent potentially dangerous low blood glucose by restoring the body's ability to counterregulate hypoglycemia. In people without diabetes, α-cells secrete glucagon that signals the body to release its own glucose stores to prevent or reverse hypoglycemia. However, in people with insulin-dependent diabetes – including those with T1D and insulin-dependent Type 2 diabetes – evidence suggests that elevated secretion of pancreatic somatostatin suppresses glucagon release from α-cells. Zucara has demonstrated that, in people with T1D, the glucagon response can be increased with ZT-01, a first-in-class SST receptor 2 antagonist. About Zucara Therapeutics Inc. Zucara Therapeutics is developing ZT-01, a first-in-class, once-daily therapeutic to prevent hypoglycemia in people with T1D and insulin-dependent Type 2 diabetes. ZT-01 is designed to inhibit somatostatin, a pancreatic hormone that impairs the glucagon response to hypoglycemia in people with these conditions. ZT-01 is designed to restore glucagon secretion to prevent hypoglycemia, which could dramatically change diabetes disease management and improve both patient health and quality of life. For more information, visit . SOURCE Zucara 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

12 Nov 2024

·www.mobihealthnews.com

Optic, Vindur Tx partner to develop effective cancer treatments and more digital health news

Digital biology company Optic is collaborating with early-stage oncology therapeutics venture, Vindur Tx, to use Optics proprietary generative AI platform to find novel highly selective kinase inhibitors and develop more effective cancer treatments. Vindur Tx agreed to pay Optic as much as $17.5 million for its proprietary technology to search for new ways of addressing what the company called previously "undruggable" targets in difficult-to-treat cancers. The strategy could lead to new and more effective treatments. Optic's AI platform BIOPTIC accelerates the drug discovery process by utilizing an ultra-high-performance exclusive AI screening platform for testing billions of compounds in minutes. The process helps explore the previously unreachable vast chemical space to search for novel drug candidates faster. "Working with BIOPTIC has been a revelation," Vindur Tx cofounder Deniz Kural said in a statement. "They delivered highly selective ligands in record time, significantly accelerating our research. We're thrilled with the results and the potential impact on our projects." Diabetes life sciences company, Zucara Therapeutics, announced that pharma giant Sanofi has made a strategic investment in the company as part of its $20 million Series B financing. Sanofi will also receive an exclusive right of first negotiation. The Perceptive Xontogeny Venture Fund and other investors participated in the round which could increase to $25 million. Proceeds from the financing will fund Zucara's ongoing Phase 2a trial of the effect of ZT-01 On Nocturnal hypoglycemia Events in Type 1 diabetes ("T1D") mellitus ("ZONE") and the nonclinical activities to support a once-weekly version of ZT-01. "Having led Zucara's US$21 million Series A financing in early 2020, we are proud to continue to support the company in advancing the development of ZT-01, especially alongside strategic partner and world-leading healthcare company, Sanofi," Chris Garabedian, portfolio manager at Venture for Perceptive Advisors, said in a statement. "By preventing hypoglycemia in people using insulin therapy, ZT-01 represents a promising approach to improving health outcomes of people living with diabetes." MintNeuro, a company involved in low-power semiconductor technology for minimally-invasive neural implant applications, announced it raised £1 million ($1.27 million) in a funding round led by Empirical Ventures. Parkwalk Advisors (through the Imperial College Enterprise Fund), Imperial College London, Plug & Play, Excellis Holding and several investors participated in the round. MintNeuro uses semiconductor technology to make neural implants smaller, more efficient and scalable. With over £14 million ($17.84 million) in underpinning R&D grant funding to date, the MintNeuro team used semiconductor manufacturing to permit cost-effective, mass-producible neural devices. "Our semiconductor-based approach is fundamentally reshaping what neural implants can achieve," Timothy Constandinou, CTO and cofounder of MintNeuro, said in a statement. "By offering a customisable, compact solution that integrates seamlessly into medical devices, we're empowering a new era of neural interfaces that are not only more powerful but also less invasive. This marks a transformative shift in neurological care."

Phase 2

100 Deals associated with ZT-01

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Diabetes Mellitus, Type 1	Phase 2	United States	Zucara Therapeutics, Inc.	28 Jul 2023
Diabetes Mellitus, Type 1	Phase 2	Canada	Zucara Therapeutics, Inc.	28 Jul 2023
Hypoglycemia	Phase 2	United States	Zucara Therapeutics, Inc.	28 Jul 2023
Hypoglycemia	Phase 2	Canada	Zucara Therapeutics, Inc.	28 Jul 2023
Diabetes Mellitus, Type 2	Preclinical	Canada	New York University	02 Nov 2020
Diabetes Mellitus, Type 2	Preclinical	Canada	Zucara Therapeutics, Inc.	02 Nov 2020

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05007977 (Pubmed \| Diabetologia)	Phase 1	Diabetes Mellitus, Type 1	24	3 mg ZT-01	qudkyzwers(cjsgnfnfki) = cwvbhlokjs znjqwrqohu (xpxqieddgr, 2.3) View more	Positive	21 May 2026
				20 mg ZT-01	qudkyzwers(cjsgnfnfki) = zwjukpouhb znjqwrqohu (xpxqieddgr, 2.4) View more
NCT05007977 (OP 11, EASD2023)	Phase 1	Diabetes Mellitus, Type 1	23	ZT-01 3 mg	dpyqunhiwv(esinxbdzqp) = No SAEs were reported aoyjdceuwu (gvygokoqjk ) View more	Positive	03 Oct 2023
				ZT-01 20 mg

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ZT-01

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