risvodetinib (IkT-148009) / Inhibikase, Clintrex, ABLi Therapeutics - LARVOL DELTA

ABLi Therapeutics Announces Details of the End of Phase 2 Meeting with FDA for Evaluation of Risvodetinib as a Treatment for Parkinson’s Disease (GlobeNewswire) - "ABLi Therapeutics...completed an End of Phase 2 (EOP2) meeting with the Food and Drug Administration (FDA) to discuss outcomes of the 201 Trial in untreated Parkinson’s disease and to review plans for the Phase 3 program...The meeting’s primary purpose was to discuss endpoints to be used in a Phase 3 program and to define what tangential clinical and pre-clinical experiments may be necessary for a potential filing of a New Drug Application (NDA.) The Company presented data showing that the 201 Trial reached nominal significance for the Movement Disorder Society revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and for the Schwab & England Activities of Daily Life (SEADL) scale in a 12 week treatment, which were consistent with the FDA’s preference for evaluating patient quality of life as a primary assessment of clinical benefit."

FDA event • New P3 trial • Trial status • Parkinson's Disease

ABLi Therapeutics Launches with a Focus on Developing Treatments for Neurodegenerative Diseases that Arise from Activation of c-Abl Kinases (GlobeNewswire) - "ABLi Therapeutics...launched today with the mission of developing its lead product candidate, Risvodetinib (ABLi-148009), as a potential disease-modifying therapy for Parkinson’s and Parkinson’s-related diseases and a portfolio of second-generation product candidates."

Pipeline update • Parkinson's Disease

A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY OF RISVODETINIB, A SELECTIVE C-ABL INHIBITOR, IN UNTREATED PARKINSON'S DISEASE (ADPD 2025) - P2 | "Conclusions Formal Results of the study will be presented at the meeting. To date, IkT-148009 appears to be safe and well tolerated"

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease • ABL1 • ABL2

IkT-148009-201: A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson's Disease (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: ABLi Therapeutics, Inc. | Trial completion date: Jan 2025 ➔ Jun 2025

Trial completion date • CNS Disorders • Movement Disorders • Parkinson's Disease

Parkinson’s Disease New Drug Development Fails in Series… Inhibikase Discontinues ’Risvodetinib’ and Focuses on PAH [Gogole trnaslation] (The Bio) - P2 | N=120 | NCT05424276 | Sposnor: Inhibikase Therapeutics, Inc. | "On the 30th (local time), American pharmaceutical company Inhibikase Therapeutics...announced that it will discontinue the development of 'risvodetinib (development code name IkT-148009),' a new drug candidate for Parkinson's disease in phase 2 clinical trials. Instead, Inhibikase plans to focus on its other major development program, 'IkT-001Pro (development code name)...risbodetinib met the primary efficacy objectives in terms of safety and tolerability in the phase 2 clinical trial, but lacked the primary functional improvement effect. Inhibicase plans to present additional analysis results at a future academic conference...The clinical results showed that lisbodetinib showed a similar frequency and severity of side effects compared to the placebo group after 95% of participants completed the 12-week treatment, indicating no issues related to safety and tolerability."

Discontinued • P2 data • Parkinson's Disease • Pulmonary Arterial Hypertension

A PK Study of IkT-148009 in Older and Elderly Healthy Subjects (clinicaltrials.gov) - P1 | N=8 | Completed | Sponsor: Inhibikase Therapeutics, Inc.

New P1 trial

A Phase I 7-Day Dosing Study of IkT-148009. (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Inhibikase Therapeutics, Inc.

New P1 trial

The 201 Trial in Untreated Parkinson's Disease (MDS Congress 2024) - "Data from a 12 week double-blinded, placebo controlled trial will be presented providing a first look into whether a treatment validated in models of heritable and sporadic PD can impact disease in human Parkinson's."

CNS Disorders • Parkinson's Disease • ABL1

IkT-148009-201: A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson's Disease (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: Inhibikase Therapeutics, Inc. | Trial primary completion date: Jun 2024 ➔ Sep 2024

Trial primary completion date • CNS Disorders • Movement Disorders • Parkinson's Disease

Inhibikase Therapeutics Reports Second Quarter Financial Results and Highlights Recent Period Activity (GlobeNewswire) - "'We completed enrollment of The 201 Trial evaluating risvo in untreated Parkinson’s disease, and anticipate the last patient exiting the study in September, 2024. We anticipate reporting topline data in November, 2024.'"

P2 data • Trial status • CNS Disorders • Parkinson's Disease

Inhibikase Therapeutics Completes Enrollment of the Phase 2 ‘201’ Trial Evaluating Risvodetinib in Untreated Parkinson’s Disease (GlobeNewswire) - "Inhibikase Therapeutics...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 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."

Enrollment closed • P2 data • Parkinson's Disease

Inhibikase Therapeutics Announces Expansion to its Therapeutic Pipeline and Updates its Research and Development Programs (GlobeNewswire) - "Inhibikase Therapeutics, Inc...today announced expansion of its therapeutic pipeline and multiple updates to its Research and Development programs....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."

FDA event • Trial status • CNS Disorders • Parkinson's Disease

A Randomized, Double-Blind, Placebo-Controlled Trial of IkT-148009 in Untreated Parkinson's Disease (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: Inhibikase Therapeutics, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Movement Disorders • Parkinson's Disease

Emerging Novel Therapies in Development for PD (NeurologyLive) - "Kremens gave thoughts on the use of several investigational continuous agents, including IPX203, risvodetinib, and P2B001."

Video

The 201 Trial in Untreated Parkinson's Disease (AAN 2024) - "Data from a 12 week double-blinded, placebo controlled trial will be presented providing a first look into whether a treatment validated in models of heritable and sporadic PD can impact disease in human Parkinson's."

Cardiovascular • CNS Disorders • Movement Disorders • Parkinson's Disease • ABL1

Inhibikase Therapeutics to Provide Trial Update for Risvodetinib at the 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (GlobeNewswire) - "Inhibikase Therapeutics, Inc...today announced that Dr. Milton Werner, President and Chief Executive Officer of Inhibikase Therapeutics, will present an update on the of the 201 Trial evaluating risvodetinib ('risvo') at the 2024 AD/PD Meeting being held March 5-9, 2024 in Lisbon, Portugal."

P2 data • CNS Disorders • Parkinson's Disease

THE 201 TRIAL IN UNTREATED PARKINSON'S DISEASE (ADPD 2024) - "Data from a 12 week double-blinded, placebo controlled trial will be presented providing a first look into whether a treatment validated in models of heritbale and sporadic PD can impact disease in human Parkinson's."

Cardiovascular • CNS Disorders • Movement Disorders • Parkinson's Disease

Inhibikase Therapeutics Announces Publication Highlighting Results from its Phase 1 Studies with Risvodetinib (GlobeNewswire) - P1 | N=108 | "In Phase 1 studies, risvo was shown to have a favorable safety profile that was well tolerated by older or elderly healthy subjects and by patients with Parkinson’s disease. As we look ahead, we are rapidly advancing our 201 Trial in untreated PD patients and look forward to reporting topline results, possibly as early as the second half of 2024 depending on the enrollment date of the last trial participant....Only 11 of the observed adverse events were deemed possibly treatment-related, with none of clinical significance."

P1 data • CNS Disorders • Parkinson's Disease

A Phase I, Randomized, SAD, MAD, and PK Study of Risvodetinib in Older Adults and Parkinson's Disease. (PubMed, J Parkinsons Dis) - "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."

Journal • CNS Disorders • Movement Disorders • Parkinson's Disease • ABL1

Inhibikase Therapeutics Highlights Unblinded Functional Analysis from the 201 Trial of Risvodetinib in Untreated Parkinson’s Disease and Provides Update on Ongoing Enrollment (GlobeNewswire) - P2 | N=120 | NCT05424276 | Sponsor: Inhibikase Therapeutics, Inc. | "Inhibikase Therapeutics, Inc...today highlighted an analysis of eleven unblinded participants from the 201 Trial evaluating risvodetinib (IkT-148009)....At the End of Study timepoint, participants administered the 200 mg dose had a combined Part 2 and Part 3 score that was lower by an average of -8.7 points. By contrast, the combined placebo score increased by an average of +1.7 points, a -10.4 point spread between actively treated versus placebo participants. Patients administered 50 or 100 mg experienced an average change of +1.7 and -1.3 points, respectively, for the combined score....The S&E scale was reduced for the 200 mg group by an average of -3.3 points relative to baseline, while the placebos had an average score increase of +3.3 points, a 6.6 point spread between the actively treated participants and the placebos...while the 100 mg dose was on average -5.0 points lower relative to baseline."

P2 data • CNS Disorders • Parkinson's Disease

Inhibikase Therapeutics Highlights Unblinded Functional Analysis from the 201 Trial of Risvodetinib in Untreated Parkinson’s Disease and Provides Update on Ongoing Enrollment (GlobeNewswire) - "The Company also provided an update on the enrollment progress and recruitment tools being used in the ongoing 201 Trial....The 201 Trial is a 12-week double-blinded study across three doses plus a placebo group, 1:1:1:1 randomized. The trial is planned to extend every enrolled participant into a 12-month extension study without placebo, but this extension study is not yet implemented. Enrollment is progressing, with 27 of 34 planned sites consenting, screening and enrolling participants. 37 people are either undergoing informed consent, being screened or have been enrolled in the trial, and three participants have completed the full 12-week regimen."

Enrollment status • Trial status • CNS Disorders • Parkinson's Disease

Inhibikase Therapeutics Receives FDA Orphan Drug Designation for Risvodetinib for the Treatment of Multiple System Atrophy (GlobeNewswire) - "Inhibikase Therapeutics, Inc...today announced that risvodetinib (IkT-148009) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Multiple System Atrophy (MSA)."

Orphan drug • Multiple System Atrophy

Analysis of the therapeutic potential of c-Abl inhibition in Multiple System Atrophy (MDS Congress 2023) - "As of this writing, ongoing experiments have suggested a possible benefit for c-Abl inhibition as an interventional therapy for MSA. Detailed results will clarify the role of c-Abl activation in the disease process and the potential benefit of c-Abl inhibition as a disease-modifying therapy in the model organism."

CNS Disorders • Multiple System Atrophy • ABL1

Analysis of the therapeutic potential of IkT-148009 in Parkinson’s disease (MDS Congress 2023) - "In model studies, IkT-148009 drove functional recovery concomitant with clearance of the underlying alpha-synuclein pathology, consistent with the mechanism of disease initiation and progression recently described (2). Clinically, IkT-148009 was shown to be relatively safe and induced no serious or clinically significant adverse events across a wide dosing range. These outcomes warrant the continued evaluation of IkT-148009 as a potential disease-modifying therapy for PD."

CNS Disorders • Parkinson's Disease • ABL1

Inhibikase Therapeutics Initiates Development of Second Generation c-Abl inhibitors and names IkT-148009 as risvodetinib - "Inhibikase Therapeutics, Inc...is evaluating new molecules with multiple mechanisms of action for c-Abl inhibition. Along with the development of new molecules, IkT-148009 has now been granted the non-proprietary name of risvodetinib (or 'risvo')....'In addition, we are evaluating early stage molecules from other companies that inhibit c-Abl without binding to the enzyme active site. Such novel inhibitors, alone or in combination with active site inhibitors like IkT-148009, may be an improved approach to suppress neurodegeneration arising from c-Abl activation inside and outside of the brain.'"

Clinical • Preclinical • CNS Disorders • Parkinson's Disease