ALZ002 / AnnJi Pharma, Fortress - LARVOL DELTA

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients (clinicaltrials.gov) - P1/2 | N=25 | Completed | Sponsor: AnnJi Pharmaceutical Co., Ltd. | Active, not recruiting ➔ Completed

Trial completion • Muscular Atrophy • AR

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients (clinicaltrials.gov) - P1/2 | N=24 | Active, not recruiting | Sponsor: AnnJi Pharmaceutical Co., Ltd. | Recruiting ➔ Active, not recruiting

Enrollment closed • Muscular Atrophy • AR

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: AnnJi Pharmaceutical Co., Ltd. | Trial completion date: Dec 2023 ➔ Apr 2024 | Trial primary completion date: Sep 2023 ➔ Mar 2024

Trial completion date • Trial primary completion date • Muscular Atrophy • AR

Avenue Therapeutics Announces First Patient Dosed in Phase 1b/2a Clinical Trial of AJ201 for the Treatment of Spinal and Bulbar Muscular Atrophy (Kennedy's Disease) (GlobeNewswire) - "Avenue Therapeutics, Inc...today announced that the first patient has been dosed in the Phase 1b/2a clinical trial of AJ201 for the treatment of spinal and bulbar muscular atrophy ('SBMA'), also known as Kennedy's Disease. A recent study used genetic analysis to estimate disease prevalence of 1:6,887 males.1 AJ201 is currently the lead drug candidate in the clinic for SBMA and enrollment in the trial is expected to be complete by the end of 2023 or early 2024 with potential topline data in 2024....The primary endpoint of the study is to assess safety and tolerability of AJ201 in subjects with clinically and genetically defined SBMA."

P1/2 data • Trial status • Genetic Disorders • Muscular Atrophy

Avenue Therapeutics Enters into a Transformational License Agreement with AnnJi Pharmaceutical to Develop and Commercialize AJ201, a First-in-Class Clinical Asset for the Treatment of Spinal and Bulbar Muscular Atrophy (GlobeNewswire) - "Avenue Therapeutics...announced that it has entered into an exclusive license agreement with AnnJi Pharmaceutical Co., Ltd. ('AnnJi), a Taiwanese clinical-stage drug company, for AJ201, a first-in-class clinical asset currently in a Phase 1b/2a study in the U.S. for the treatment of spinal and bulbar muscular atrophy ('SBMA'), also known as Kennedy's Disease."

Licensing / partnership • CNS Disorders • Genetic Disorders • Muscular Atrophy

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients (clinicaltrials.gov) - P1/2 | N=24 | Recruiting | Sponsor: AnnJi Pharmaceutical Co., Ltd. | Not yet recruiting ➔ Recruiting | Initiation date: Nov 2022 ➔ Feb 2023

Enrollment open • Trial initiation date • Muscular Atrophy • AR

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: AnnJi Pharmaceutical Co., Ltd.

New P1/2 trial • Muscular Atrophy • AR

In Vitro Efficacy and Molecular Mechanism of Curcumin Analog in Pathological Regulation of Spinocerebellar Ataxia Type 3. (PubMed, Antioxidants (Basel)) - "Unlike other nuclear factor erythroid-2-related factor 2 (Nrf2) activators, the mechanism of action of curcumin analog, ASC-JM17 (JM17), in regulating oxidative homeostasis remains unknown...Presently, we compared actions of JM17 with those of known Nrf2 activators, omaveloxolone (RTA-408) and dimethyl fumarate (DMF), using human neuroblastoma SK-N-SH cells with stable transfection of full-length ataxin-3 protein with 78 CAG repeats (MJD78) to clarify the resulting pathological mechanism by assaying mitochondrial function, mutant ataxin-3 protein toxicity, and oxidative stress...It showed that activation of Nrf2 in response to ROS generated in mitochondria could play an import role in the benefit of JM17. This study presents the diversified regulation of JM17 in a pathological process and helped develop more effective therapeutic strategies for SCA3."

Journal • Preclinical • Ataxia • CNS Disorders • Movement Disorders • Neuroblastoma • Oncology • Solid Tumor • ATXN3 • SOD2

Diketo-Ketoenol Tautomers in Curcuminoids: Synthesis, Separation of Tautomers, and Kinetic and Structural Studies. (PubMed, J Org Chem) - "Additionally, a pure ketoenol tautomeric form of the active pharmaceutical ingredient (API) ASC-JM17 has been unequivocally determined by X-ray crystallography. Two different polymorphs of this API have been microscopically identified in the X-ray sample and manually separated, and a solid-state NMR study of the two polymorphs has also been performed. This work reports on the slow kinetics of diketo-ketoenol tautomerization in particular solvents that allow the separation and full characterization of both curcuminoids' tautomers."

Journal

A Study to Investigate the Safety, Tolerability, Pharmacokinetics of ALZ002 DS in Healthy Volunteers (clinicaltrials.gov) - P1; N=72; Completed; Sponsor: Allianz Pharmascience Limited; Recruiting ➔ Completed

Trial completion

A Study to Investigate the Safety, Tolerability, Pharmacokinetics of ALZ002 DS in Healthy Volunteers (clinicaltrials.gov) - P1; N=72; Recruiting; Sponsor: Allianz Pharmascience Limited; Trial completion date: Jan 2021 ➔ Aug 2021; Trial primary completion date: Dec 2020 ➔ May 2021

Clinical • Trial completion date • Trial primary completion date