EDI048 / Novartis - LARVOL DELTA

A first-in-human phase 1 clinical study in healthy volunteers to assess the safety, tolerability and pharmacokinetics of EDI048, a novel promising agent to treat enteric cryptosporidiosis. (PubMed, J Infect Dis) - "EDI048 had a tolerable safety profile at all doses tested, up to 400 mg orally administered BID for 5.5 days. Pharmacokinetic analysis showed minimal systemic exposure of EDI048 in healthy adults. These results support further clinical development for cryptosporidiosis."

First-in-human • Journal • P1 data • PK/PD data • Gastrointestinal Disorder

CRYPTONITE: Study Testing the Efficacy, Safety, and Tolerability of EDI048 in Cryptosporidium Infection Model in Healthy Adults (clinicaltrials.gov) - P2 | N=80 | Recruiting | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Infectious Disease

CRYPTONITE: Study Testing the Efficacy, Safety, and Tolerability of EDI048 in Cryptosporidium Infection Model in Healthy Adults (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

New P2 trial • Infectious Disease

First in Human Study to Assess the Safety, Tolerability and Pharmacokinetics of EDI048 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=110 | Completed | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Completed | N=80 ➔ 110

Enrollment change • Trial completion

Cryptosporidium PI(4)K inhibitor EDI048 is a gut-restricted parasiticidal agent to treat paediatric enteric cryptosporidiosis. (PubMed, Nat Microbiol) - "Safety and pharmacological studies demonstrated predictable metabolism and low systemic exposure of EDI048, providing a substantial safety margin required for a paediatric indication. EDI048 is a promising clinical candidate for the treatment of life-threatening paediatric cryptosporidiosis."

Journal • Gastrointestinal Disorder • Infectious Disease • Pediatrics

First in Human Study to Assess the Safety, Tolerability and Pharmacokinetics of EDI048 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=80 | Recruiting | Sponsor: Novartis Pharmaceuticals | Active, not recruiting ➔ Recruiting | N=54 ➔ 80

Enrollment change • Enrollment open

First in Human Study to Assess the Safety, Tolerability and Pharmacokinetics of EDI048 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=54 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Nov 2023 ➔ Nov 2024 | Trial primary completion date: Nov 2023 ➔ Nov 2024

Trial completion date • Trial primary completion date

Discovery of EDI048, a first-in-class oral soft-drug Cp PI(4)Ki for the treatment of pediatric cryptosporidiosis (ACS-Fall 2023) - "The only approved medication for cryptosporidiosis, nitazoxanide, relies on host immunity system and is weakly efficacious in the most vulnerable populations, e.g. malnourished children. This "soft-drug" approach provides high levels of epithelial exposure while minimizing the risks inherent to human kinase inhibition. Herein, we will discuss the compound design and in vitro data used to optimize EDI048 for sufficient exposure in the intestine and low systemic exposure, pre-clinical efficacy in mice and calves to support compound selection, and pre-clinical safety and dose-projection analyses on EDI048."

Clinical • Pediatrics

First in Human Study to Assess the Safety, Tolerability and Pharmacokinetics of EDI048 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=54 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Recruiting ➔ Active, not recruiting | N=80 ➔ 54 | Trial completion date: Mar 2023 ➔ Nov 2023 | Trial primary completion date: Mar 2023 ➔ Nov 2023

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date

First in Human Study to Assess the Safety, Tolerability and Pharmacokinetics of EDI048 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=80 | Recruiting | Sponsor: Novartis Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial completion date: Nov 2022 ➔ Mar 2023 | Trial primary completion date: Nov 2022 ➔ Mar 2023

Enrollment open • Trial completion date • Trial primary completion date