bocidelpar (MA-0211) / Astellas - LARVOL DELTA

Pharmacokinetics of Bocidelpar, ASP0367, in Renal and Hepatic Impairment: Results From Two Phase 1 Studies. (PubMed, Clin Transl Sci) - P1 | "Overall, bocidelpar was well tolerated and had an acceptable safety profile. Severe renal impairment had a minimal effect on bocidelpar pharmacokinetics, while moderate hepatic impairment resulted in increased bocidelpar concentration and exposure compared with matched controls."

Journal • P1 data • PK/PD data • Hepatology • Renal Disease

Evaluation of CD1a immunostaining in the diagnosis of cutaneous leishmaniasis caused by Leishmania donovani in Sri Lanka. (PubMed, Parasitology) - "Our findings show that CD1a clone MTB1 successfully stains amastigotes of L. donovani species and can be used as a supplementary diagnostic tool in detecting CL, especially when LD bodies are low in number. This method could be validated to detect other forms of leishmaniasis caused by L. donovani in Indian and sub-Saharan regions."

Journal • Dermatology • Dermatopathology • Infectious Disease • CD1a

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1 | N=8 | Terminated | Sponsor: Astellas Pharma Inc | Phase classification: P1b ➔ P1

Phase classification • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

MOUNTAINSIDE: A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (clinicaltrials.gov) - P2 | N=34 | Terminated | Sponsor: Astellas Pharma Inc | Phase classification: P2/3 ➔ P2 | N=195 ➔ 34 | Trial completion date: Nov 2025 ➔ May 2024 | Active, not recruiting ➔ Terminated | Trial primary completion date: May 2025 ➔ Apr 2024; Due to failure to meet the pre-specified criteria for efficacy

Enrollment change • Phase classification • Trial completion date • Trial primary completion date • Trial termination • Myositis

Disseminated Mycobacterium Tuberculosis Post Lung Transplantation (ISHLT 2024) - "Induction immunosuppression (ISN) included basiliximab, mycophenolate (MMF) & solumedrol followed by maintenance ISN of tacrolimus (FK,10-12 ng/ml), MMF, & prednisone...Prednisone dose was 20 mg a day while on rifabutin...Rapid onset & dissemination of MTB 1 month after the transplant makes our case unique.Donor was a young female, who moved from Mexico...This brings up the inquiry of whether & how to screen the donors for LTBI. The limitation would be both TST & IGRA might not be feasible due to time constraints and AFB cultures on BAL if sent, will be finalized after organ is transplanted."

Cough • Hematological Disorders • Infectious Disease • Leukopenia • Neutropenia • Pulmonary Disease • Respiratory Diseases • Transplantation • Tuberculosis

A Study to Evaluate ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction (clinicaltrials.gov) - P1 | N=31 | Terminated | Sponsor: Astellas Pharma Global Development, Inc. | Phase classification: P1b ➔ P1

Phase classification

MOUNTAINSIDE: A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (clinicaltrials.gov) - P2/3 | N=195 | Active, not recruiting | Sponsor: Astellas Pharma Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Myositis

MOUNTAINSIDE: A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (clinicaltrials.gov) - P2/3 | N=195 | Recruiting | Sponsor: Astellas Pharma Inc | Trial completion date: May 2024 ➔ Nov 2025 | Trial primary completion date: Oct 2023 ➔ May 2025

Trial completion date • Trial primary completion date • Myositis

A Study of ASP0367 in People With Kidneys That do Not Work Well and in Healthy People (clinicaltrials.gov) - P1 | N=13 | Completed | Sponsor: Astellas Pharma Global Development, Inc. | Recruiting ➔ Completed | N=36 ➔ 13 | Trial completion date: Mar 2023 ➔ Nov 2022 | Trial primary completion date: Mar 2023 ➔ Nov 2022

Enrollment change • Trial completion • Trial completion date • Trial primary completion date • Renal Disease

A Study to Evaluate ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction (clinicaltrials.gov) - P1b | N=31 | Terminated | Sponsor: Astellas Pharma Global Development, Inc. | Trial completion date: May 2023 ➔ Dec 2022 | Recruiting ➔ Terminated | Trial primary completion date: May 2023 ➔ Dec 2022; Lack of Pharmacological effect

Trial completion date • Trial primary completion date • Trial termination

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1b | N=8 | Terminated | Sponsor: Astellas Pharma Inc | Trial completion date: Mar 2023 ➔ Sep 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Mar 2023 ➔ Sep 2022; Operational Reasons

Trial completion date • Trial primary completion date • Trial termination • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

Immunodiagnostic potential of CD1a marker to detect cutaneous leishmaniasis caused by Leishmania donovani (ASTMH 2022) - "CD1a clone MTB1 successfully stains leishmania amastigotes of L.donovani strain and can be used as a reliable diagnostic tool in patients whose parasite counts are extremely low & whose diagnostic confirmation got failed using other conventional methods. This tool may come in handy for hospital settings where SSS sampling is fairly poor due to the lack of expertized personnel."

Dermatology • Dermatopathology • Infectious Disease • CD1a

Immunodiagnostic potential of CD1a marker to detect cutaneous leishmaniasis caused by Leishmania donovani (ASTMH 2022) - "CD1a clone MTB1 successfully stains leishmania amastigotes of L.donovani strain and can be used as a reliable diagnostic tool in patients whose parasite counts are extremely low & whose diagnostic confirmation got failed using other conventional methods. This tool may come in handy for hospital settings where SSS sampling is fairly poor due to the lack of expertized personnel."

Dermatology • Dermatopathology • Infectious Disease • CD1a

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1b | N=8 | Active, not recruiting | Sponsor: Astellas Pharma Inc | Recruiting ➔ Active, not recruiting | N=18 ➔ 8

Enrollment change • Enrollment closed • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

A Study to Learn How [14C]ASP0367 is Processed by the Body in Healthy Men (clinicaltrials.gov) - P1 | N=9 | Completed | Sponsor: Astellas Pharma Global Development, Inc. | Recruiting ➔ Completed

Trial completion

A Study to Evaluate ASP0367 in Participants With Reduced Maximum Oxygen Uptake Due to Poor Systemic Oxygen Extraction (clinicaltrials.gov) - P1b | N=40 | Recruiting | Sponsor: Astellas Pharma Global Development, Inc. | Trial completion date: Jul 2022 ➔ May 2023 | Trial primary completion date: Jul 2022 ➔ May 2023

Trial completion date • Trial primary completion date

A Study to Learn How [14C]ASP0367 is Processed by the Body in Healthy Men (clinicaltrials.gov) - P1 | N=7 | Recruiting | Sponsor: Astellas Pharma Global Development, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open

A Study to Evaluate ASP0367 in Participants With Mild/Moderate Hepatic Impairment Compared to Participants With Normal Hepatic Function (clinicaltrials.gov) - P1 | N=25 | Completed | Sponsor: Astellas Pharma Global Development, Inc. | Recruiting ➔ Completed

Trial completion • Hepatology

A Study to Evaluate an ASP0367 Solution in Healthy Male Participants (clinicaltrials.gov) - P1 | N=7 | Not yet recruiting | Sponsor: Astellas Pharma Global Development, Inc.

New P1 trial

MOUNTAINSIDE: A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (clinicaltrials.gov) - P2/3; N=195; Recruiting; Sponsor: Astellas Pharma Inc; N=149 ➔ 195

Clinical • Enrollment change • Myositis

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P1b; N=18; Recruiting; Sponsor: Astellas Pharma Inc; Trial completion date: Mar 2022 ➔ Mar 2023; Trial primary completion date: Mar 2022 ➔ Mar 2023

Clinical • Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • Pediatrics

A Study of ASP0367 in People With Kidneys That do Not Work Well and in Healthy People (clinicaltrials.gov) - P1; N=36; Recruiting; Sponsor: Astellas Pharma Global Development, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Renal Disease

A Study of ASP0367 in People With Kidneys That do Not Work Well and in Healthy People (clinicaltrials.gov) - P1; N=36; Not yet recruiting; Sponsor: Astellas Pharma Global Development, Inc.

Clinical • New P1 trial • Renal Disease

Single- and multiple-dose safety, tolerability, pharmacokinetic, and pharmacodynamic profiles of ASP0367, or bocidelpar sulfate, a novel modulator of peroxisome proliferator-activated receptor delta in healthy adults: Results from a phase 1 study. (PubMed, Muscle Nerve) - "ASP0367, or bocidelpar sulfate, was well tolerated; rapid absorption, roughly dose-proportional bioavailability, and effects on PPARδ target genes were demonstrated in healthy adult participants."

Clinical • Journal • P1 data • PK/PD data • Duchenne Muscular Dystrophy • Genetic Disorders • Metabolic Disorders • Muscular Dystrophy • Myositis

A Study to Evaluate ASP0367 in Participants With Mild/Moderate Hepatic Impairment Compared to Participants With Normal Hepatic Function (clinicaltrials.gov) - P1; N=32; Recruiting; Sponsor: Astellas Pharma Global Development, Inc.; Not yet recruiting ➔ Recruiting

Enrollment open • Hepatology