This study investigates the safety and tolerability of drug IkT-148009 in untreated Parkinson's disease volunteers (30 to 80 years old). It also looks at the pharmacokinetics of IkT-148009 in the body and evaluates the effect of IkT-148009 on motor and non-motor features of the disease. This 12 week study is designed to be 3:1 randomized across 3 doses of IkT-148009 or placebo. Each participant will self-administer one of 3 doses or placebo of IkT-148009 once daily (QD) with food for 12 weeks. For more information, visit our website: www.the201trial.com

Start Date15 May 2023

Sponsor / Collaborator

Inhibikase Therapeutics, Inc.

NCT04350177 / CompletedPhase 1

A Phase I, Randomized Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Study to Determine the Safety, Tolerability and Pharmacokinetics (PK) of IkT-148009 in Older Adult and Elderly Healthy Volunteers With Extension Into Parkinson's Patients

This study investigates the safety and tolerability of drug IkT-148009 in healthy elderly volunteers (55 to 70 years old). It also looks at the movement of IkT-148009 in the body. This first-in-human study is designed in 3 parts. In Part A, healthy participants will take a single, oral dose of IkT-148009 or placebo. Part A participants will be at the study site for approximately 4 days. In Part B, healthy participants will take an oral dose of IkT-148009 once a day for 7 days. Part B participants will be at the study site for approximately 12 days. In Part C, Parkinson's patients will take an oral dose of IkT-148009 once a day for 7 days. Part C participants will be at the study site for approximately 12 days.

Start Date16 Feb 2021

Sponsor / Collaborator

Inhibikase Therapeutics, Inc.

100 Clinical Results associated with Risvodetinib

100 Translational Medicine associated with Risvodetinib

100 Patents (Medical) associated with Risvodetinib

Literatures (Medical) associated with Risvodetinib

05 Mar 2024·Journal of Parkinson's disease

A Phase I, Randomized, SAD, MAD, and PK Study of Risvodetinib in Older Adults and Parkinson’s Disease

Article

Author: Ellenbogen, Aaron ; Pellecchia, Jacqueline ; Kruger, Sydney ; Olanow, C Warren ; Rodriguez, Carlos ; Blob, Lawrence ; Walaker, Shannon ; Safirstein, Beth ; Samara, Emil ; Hassman, Howard ; Ereshefsky, Larry ; Meyer, Christopher ; McGarry, Andrew ; Klint, Carl ; Werner, Milton H

Background: Pre-clinical studies suggest that c-Abl activation may play an important role in the etiology of Parkinson’s disease, making c-Abl an important target to evaluate for potential disease-modification. Objective: To assess safety, tolerability, and pharmacokinetics of the c-Abl inhibitor risvodetinib (IkT-148009) in healthy subjects and participants with Parkinson’s disease. Methods: Part 1 (single ascending dose (SAD)) and Part 2 (7-day multiple ascending dose (MAD)) studies were in healthy volunteers. Participants were randomized 3 : 1 across 9 SAD doses and 3 MAD doses of risvodetinib (IkT-148009) or placebo. Part 3 was a MAD study conducted at two doses in 14 participants with mild-to-moderate PD (MAD-PD). Primary outcome measures were safety, tolerability and pharmacokinetics. Exploratory outcomes in PD participants included clinical measures of PD state, GI function, and cerebrospinal fluid (CSF) concentration. Results: 108 patients were treated with no dropouts. The SAD tested doses ranging from 12.5 to 325 mg, while the MAD tested 25 to 200 mg and MAD-PD tested 50 to 100 mg in Parkinson’s participants. All active doses had a favorable safety profile with no clinically meaningful adverse events. Single dose pharmacokinetics were approximately linear between 12.5 mg and 200 mg for both Cmax and AUC0 - inf without distinction between healthy volunteers and participants with PD. Exposures at each dose were high relative to other drugs in the same kinase inhibitor class. Conclusions: Risvodetinib (IkT-148009) was well tolerated, had a favorable safety and pharmacology profile over 7-day dosing, did not induce serious adverse events and did not appear to induce deleterious side-effects in participants administered anti-PD medications.

18 Jan 2023·Science translational medicineQ1 · MEDICINE

The c-Abl inhibitor IkT-148009 suppresses neurodegeneration in mouse models of heritable and sporadic Parkinson’s disease

Q1 · MEDICINE

Article

Author: Rush, Roger ; Marino Lee, Shirley ; Dawson, Valina L. ; Kim, Erica ; Nguyen, Richard ; Dang, Brianna ; Werner, Milton H. ; Wang, Hu ; Kelly, Terence ; Sloan, Nicholas ; Dawson, Ted M. ; Bisen, Shivani ; Brahmachari, Saurav ; Lee, Hyeun Woo ; Karuppagounder, Senthilkumar S. ; Yamashita, Yoko ; Sigmon, Alexander ; Watkins, Leslie ; Kumar, Manoj

Parkinson’s disease (PD) is the second most prevalent neurodegenerative disease of the central nervous system, with an estimated 5,000,000 cases worldwide. PD pathology is characterized by the accumulation of misfolded α-synuclein, which is thought to play a critical role in the pathogenesis of the disease. Animal models of PD suggest that activation of Abelson tyrosine kinase (c-Abl) plays an essential role in the initiation and progression of α-synuclein pathology and initiates processes leading to degeneration of dopaminergic and nondopaminergic neurons. Given the potential role of c-Abl in PD, a c-Abl inhibitor library was developed to identify orally bioavailable c-Abl inhibitors capable of crossing the blood-brain barrier based on predefined characteristics, leading to the discovery of IkT-148009. IkT-148009, a brain-penetrant c-Abl inhibitor with a favorable toxicology profile, was analyzed for therapeutic potential in animal models of slowly progressive, α-synuclein–dependent PD. In mouse models of both inherited and sporadic PD, IkT-148009 suppressed c-Abl activation to baseline and substantially protected dopaminergic neurons from degeneration when administered therapeutically by once daily oral gavage beginning 4 weeks after disease initiation. Recovery of motor function in PD mice occurred within 8 weeks of initiating treatment concomitantly with a reduction in α-synuclein pathology in the mouse brain. These findings suggest that IkT-148009 may have potential as a disease-modifying therapy in PD.

News (Medical) associated with Risvodetinib

18 Jun 2024

·www.globenewswire.com

Inhibikase Therapeutics Completes Enrollment of the Phase 2 ‘201’ Trial Evaluating Risvodetinib in Untreated Parkinson’s Disease

June 17, 2024 -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (“Inhibikase” or “Company”), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease, Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today announced the company has completed enrollment in the Phase 2 ‘201’ trial evaluating the safety and tolerability of risvodetinib (“risvo”), a potent selective c-Abl inhibitor, for treatment in untreated Parkinson’s patients. The company expects to report trial results in the fourth quarter of 2024. "The completion of enrollment for the 201 Trial in untreated Parkinson’s disease represents a major milestone for Inhibikase,” said Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. “This achievement reflects the hard work of our team, the success of our proprietary and innovative patient identification and referral service and the commitment of our 32 U.S. study sites to evaluating potential disease-modifying solutions for patients with this debilitating disease. With enrollment in the study now complete, we look forward to reporting trial results in the fourth quarter, and the discussion with the FDA on our plans for pivotal Phase 3 trials by the end of the year.” The 201 Trial is a 12-week, randomized, double-blind, multi-center, placebo-controlled clinical trial evaluating three risvo doses in patients with untreated Parkinson’s disease to assess safety, tolerability and efficacy of risvo in untreated Parkinson’s disease. The trial has enrolled all 120 participants across 32 sites across the United States, and expects to randomize 126 patients total so as not to exclude already screened participants from participating in the trial. As of June 17, 2024, 69 participants have completed the 12-week dosing period. To-date, there have been 32 mild and 5 moderate adverse events observed that may be related to risvo treatment. Four people withdrew from the trial without completing 12 weeks of treatment. Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics for Parkinson's disease and related disorders. Inhibikase's multi-therapeutic pipeline has a primary focus on neurodegeneration and its lead program risvodetinib, an Abelson Tyrosine Kinase (c-Abl) inhibitor, targets the treatment of Parkinson's disease inside and outside the brain as well as other diseases that arise from Abelson Tyrosine Kinases. Its multi-therapeutic pipeline is pursuing Parkinson's-related disorders of the brain and GI tract, orphan indications related to Parkinson's disease such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors such as IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified several follow-on compounds to risvodetinib that could potentially be applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with offices in Lexington, Massachusetts. The content above comes from the network. if any infringement, please contact us to modify.

Phase 2

17 Jun 2024

·www.biospace.com

Inhibikase Therapeutics Completes Enrollment of the Phase 2 ‘201’ Trial Evaluating Risvodetinib in Untreated Parkinson’s DiseaseCompany expects to report trial results in 4Q24

BOSTON and ATLANTA, June 17, 2024 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (“Inhibikase” or “Company”), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease, Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today announced the company has completed enrollment in the Phase 2 ‘201’ trial evaluating the safety and tolerability of risvodetinib (“risvo”), a potent selective c-Abl inhibitor, for treatment in untreated Parkinson’s patients. The company expects to report trial results in the fourth quarter of 2024. "The completion of enrollment for the 201 Trial in untreated Parkinson’s disease represents a major milestone for Inhibikase,” said Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. “This achievement reflects the hard work of our team, the success of our proprietary and innovative patient identification and referral service and the commitment of our 32 U.S. study sites to evaluating potential disease-modifying solutions for patients with this debilitating disease. With enrollment in the study now complete, we look forward to reporting trial results in the fourth quarter, and the discussion with the FDA on our plans for pivotal Phase 3 trials by the end of the year.” The 201 Trial is a 12-week, randomized, double-blind, multi-center, placebo-controlled clinical trial evaluating three risvo doses in patients with untreated Parkinson’s disease to assess safety, tolerability and efficacy of risvo in untreated Parkinson’s disease. The trial has enrolled all 120 participants across 32 sites across the United States, and expects to randomize 126 patients total so as not to exclude already screened participants from participating in the trial. As of June 17, 2024, 69 participants have completed the 12-week dosing period. To-date, there have been 32 mild and 5 moderate adverse events observed that may be related to risvo treatment. Four people withdrew from the trial without completing 12 weeks of treatment. About Inhibikase ( ) Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics for Parkinson's disease and related disorders. Inhibikase's multi-therapeutic pipeline has a primary focus on neurodegeneration and its lead program risvodetinib, an Abelson Tyrosine Kinase (c-Abl) inhibitor, targets the treatment of Parkinson's disease inside and outside the brain as well as other diseases that arise from Abelson Tyrosine Kinases. Its multi-therapeutic pipeline is pursuing Parkinson's-related disorders of the brain and GI tract, orphan indications related to Parkinson's disease such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors such as IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified several follow-on compounds to risvodetinib that could potentially be applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with offices in Lexington, Massachusetts. Social Media Disclaimer Investors and others should note that the Company announces material financial information to investors using its investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use X, Facebook, LinkedIn and YouTube as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as "believes," "expects," "may," "will," "should," "anticipates," "plans," or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Inhibikase's current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase's actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to enroll and complete the 201 Trial evaluating risvodetinib in untreated Parkinson’s disease, to successfully apply for and obtain FDA approval for IkT-001Pro in blood and stomach cancers or other indications, to successfully conduct clinical trials that are statistically significant and whether results from our animal studies may be replicated in humans, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contacts: Company Contact: Milton H. Werner, PhD President & CEO 678-392-3419 info@inhibikase.com Investor Relations: Alex Lobo Stern Investor Relations, Inc. alex.lobo@sternir.com

Phase 2

05 Jun 2024

·www.biospace.com

Inhibikase Therapeutics Announces Expansion to its Therapeutic Pipeline and Updates its Research and Development Programs

- Company positioning multiple assets for late-stage development across its therapeutic pipeline – BOSTON and ATLANTA, June 05, 2024 (GLOBE NEWSWIRE) --Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease ("PD"), Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today announced expansion of its therapeutic pipeline and multiple updates to its Research and Development programs. “At Inhibikase we have let fundamental scientific discoveries drive identification of new product candidates that could transform the lives of patients. We first began clinical development of our product candidates in 2021. In 2025, we anticipate having multiple late-stage ready assets across our therapeutic pipeline,” commented Dr. Milton H. Werner, President and Chief Executive Officer of Inhibikase. “The 201 Trial in untreated Parkinson’s disease has reached 94% enrollment and we anticipate enrolling the last patient in mid-June. In parallel, we are seeking grant funding for the 202 trial in Multiple System Atrophy through The National Institute of Neurological Diseases and Stroke (NINDS). Finally, following our pre-IND meeting with the U.S. Food and Drug Administration (FDA) in April, 2024, we have made the decision to redirect our efforts with IkT-001Pro into cardiopulmonary disease, opening a new therapeutic area for the Company. Taken together, we believe we are on track to advance our clinical assets into late-stage trials in the coming year.” Upcoming Milestones and Strategic Updates: Complete The 201 Trial in untreated Parkinson’s disease. The Company anticipates that the last patient will complete the 12-week treatment period before the close of the third quarter of 2024. The Company expects to report biomarker and outcome data to support the Company’s pursuit of an End of Phase 2 and Phase 3 protocol discussion with the FDA by the end of 2024. Expansion into cardiopulmonary disease. Following the Company’s pre-IND meeting with the FDA, the Company will submit its IND application to the FDA for IkT-001Pro as a treatment for Pulmonary Arterial Hypertension (PAH) early in the third quarter of 2024, opening a new therapeutic area for the Company. The active ingredient in IkT-001Pro, imatinib, has previously been shown to be disease-modifying for PAH. The Company believes that 001Pro could have a more favorable safety and tolerability pro to imatinib for this indication. If approved, IkT-001Pro could be a branded product with all the value drivers of a novel treatment for indication of high unmet need. The IND for 001Pro in PAH represents the seventh the Company has filed since 2019. Scaling manufacturing of IkT-001Pro. Following the Company’s pre-NDA meeting with the FDA in January, 2024, the Company is scaling its process development efforts for IkT-001Pro to support late-stage clinical development and NDA batch requirements. These activities include development of new dosage forms to differentiate 001Pro tablets from generic imatinib mesylate in alignment with FDA feedback. Seeking support for the 202 Trial in Multiple System Atrophy through the Other Transaction Authority of NINDS. The National Institute of Neurological Diseases and Stroke (NINDS) is initiating a new funding mechanism for clinical development in neuroscience beginning June, 2024. Through this new program, termed the Other Transaction Authority (OTA), the Company is seeking to support its Phase 2 ‘202 Trial’ in MSA trial using a dedicated U.S. trial network set-up by the Institute. Discontinuing antiviral development for Progressive Multifocal Leukoencephalopathy (PML): As part of the Company’s strategy to focus on late-stage clinical assets in neurodegeneration, cancer and cardiopulmonary disease, Inhibikase will discontinue development of treatments for PML. About Inhibikase ( ) Inhibikase Therapeutics, Inc. (Nasdaq: IKT) is a clinical-stage pharmaceutical company developing therapeutics for Parkinson's disease and related disorders. Inhibikase's multi-therapeutic pipeline has a primary focus on neurodegeneration and its lead program Risvodetinib, an Abelson Tyrosine Kinase (c-Abl) inhibitor, targets the treatment of Parkinson's disease inside and outside the brain as well as other diseases that arise from Abelson Tyrosine Kinases. Its multi-therapeutic pipeline is pursuing Parkinson's-related disorders of the brain and GI tract, orphan indications related to Parkinson's disease such as Multiple System Atrophy, and drug delivery technologies for kinase inhibitors such as IkT-001Pro, a prodrug of the anticancer agent imatinib mesylate that the Company believes will provide a better patient experience with fewer on-dosing side-effects. The Company's RAMP™ medicinal chemistry program has identified several follow-on compounds to Risvodetinib that could potentially be applied to other cognitive and motor function diseases of the brain. Inhibikase is headquartered in Atlanta, Georgia with offices in Lexington, Massachusetts. Social Media Disclaimer Investors and others should note that the Company announces material financial information to investors using its investor relations website, press releases, SEC filings and public conference calls and webcasts. The Company intends to also use X, Facebook, LinkedIn and YouTube as a means of disclosing information about the Company, its services and other matters and for complying with its disclosure obligations under Regulation FD. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking terminology such as "believes," "expects," "may," "will," "should," "anticipates," "plans," or similar expressions or the negative of these terms and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Inhibikase's current expectations and assumptions. Such statements are subject to certain risks and uncertainties, which could cause Inhibikase's actual results to differ materially from those anticipated by the forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include our ability to enroll and complete the 201 Trial evaluating Risvodetinib in untreated Parkinson’s disease, to successfully apply for and obtain FDA approval for IkT-001Pro in blood and stomach cancers or other indications, to successfully conduct clinical trials that are statistically significant and whether results from our animal studies may be replicated in humans, as well as such other factors that are included in our periodic reports on Form 10-K and Form 10-Q that we the U.S. Securities and Exchange Commission. Any forward-looking statement in this release speaks only as of the date of this release. Inhibikase undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws. Contacts: Company Contact: Milton H. Werner, PhD President & CEO 678-392-3419 info@inhibikase.com Investor Relations: Alex Lobo Precision AQ alex.lobo@precisionaq.com

NDAINDPhase 2

100 Deals associated with Risvodetinib

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Constipation	Phase 2	-	Inhibikase Therapeutics, Inc.	23 May 2022
Deglutition Disorders	Phase 2	-	Inhibikase Therapeutics, Inc.	23 May 2022
Parkinson Disease	Phase 2	US	Inhibikase Therapeutics, Inc.	23 May 2022
Multiple System Atrophy	Phase 1	US	Inhibikase Therapeutics, Inc.	-

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04350177 (Pubmed) Manual	Phase 1	Parkinson Disease	108	Risvodetinib	fkquwxfnyh(tshrynpikn) = All active doses had a favorable safety profile with no clinically meaningful adverse events. erljqdwisc (auhimwkmcf ) View more	Positive	13 Jan 2024
MDS2023	Not Applicable	Parkinson Disease	-	IkT-148009	bbzbcvydly(txzovilsqh) = feiziaosdz bfllpagynj (qiaygazoxk ) View more	-	27 Aug 2023
NCT04350177 (MDS2022) Manual	Phase 1	Parkinson Disease	88	IkT-148009	zeeijdijhk(ysfytofstm) = The safety and tolerability profile of IkT-148009 in healthy subjects was consistent with chronic toxicology studies in rat and monkey. PD patiewts with mild to moderate disease (Hoehn & Yahr < 3.0) displayed a similar profile of safety and tolerability. bykgiexrjz (ytoyzymtsv )	Positive	18 Sep 2022

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