FDA Grants ODD to AP303 for Treatment of Patients With Autosomal Dominant Polycystic Kidney Disease


The ODD comes after AP303 presented meaningful improvements in renal survival in an ADPKD and the completion of the first study that evaluated healthy human participants.

Human kidneys -- Image credit: Crystal light | stock.adobe.com

Image credit: Crystal light | stock.adobe.com

About the Trial

Trial Name: A Safety, Tolerability, and Pharmacokinetics Study of AP303 in Healthy Subjects

ClinicalTrials.gov ID: NCT05503693

Sponsor: Alebund Pty Ltd

Completion Date: July 7, 2023

The FDA has granted an orphan drug designation (ODD) to AP303 (Alebund Pharmaceuticals) for the treatment of patients with autosomal dominant polycystic kidney disease (ADPKD). The novel drug demonstrated meaningful improvement of renal survival in an ADPKD mice model and completed its first in-human study in healthy participants.1

ADPKD is the most common monogenic kidney disease and is 1 of the leading causes of end-stage kidney disease in adults worldwide. The condition is portrayed with the development and expansion of cysts that are present throughout the kidney parenchyma. Further, the condition is caused by mutations in the PDK1 or PKD2 genes (78% and 15% of disease pedigrees, respectively) and treatment recommendation for those with ADPKD includes antihypertensive treatment, dietary restrictions, and pharmacotherapy, though this is only applicable to a small population of patients. Currently, more treatment options that are broadly applicable and have fewer adverse effects (AEs) are needed to slow the decline of kidney function and improve kidney outcomes for these patients.1

The ODD comes after a single-center, randomized, double-blind, placebo-controlled trial which evaluated the safety, tolerability, and pharmacokinetics of AP303 in healthy adult participants. Participants were aged 18 to 55 years and had body mass indexes between 18 to 32 kg/m2, and female participants were not pregnant and used 2 forms of birth control. The study consisted of 2 parts: part A, which was a single ascending dose phase that enrolled 4 cohorts of healthy participants; and part B, which was a multiple ascending dose phase that enrolled 3 cohorts of healthy participants. In 1 of the 4 cohorts in part A, participants were asked to fast to investigate the potential effects of AP303 and food.2

Patients received treatment with oral AP303 as low as 50 μg and increased as high as 600 μg, with the placebo comparator groups receiving the same dosage amounts. The primary end points of the trial included incidence and severity of AEs, multiple dose safety outcome measures, and pharmacokinetic characteristics following both single and multiple doses, and secondary end points included single dose and food effect safety outcome measures.2

The FDA grants ODDs to support the development and evaluation of potential new medicines that are intended to treat, diagnose, or prevent rare diseases and disorders. The ODD demonstrates the need for new treatment options of patients living with ADPDK as well as the potential AP303 has in treating this population.1

"We are very excited that AP303 has been granted ODD by the FDA,” said Gavin Xia, co-founder, chairman, and CEO of Alebund, in a press release. "It is an important milestone for Alebund as well as our efforts to address the significant unmet medical needs with ADPKD. We look forward to advancing AP303 to improve future treatment options for patients in need.”1

1. PR Newswire. Alebund's Innovative Investigational Drug AP303 Receives FDA Orphan Drug Designation (ODD) for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). News release. March 29, 2024. Accessed March 29, 2024. https://prnmedia.prnewswire.com/news-releases/alebunds-innovative-investigational-drug-ap303-receives-fda-orphan-drug-designation-odd-for-the-treatment-of-autosomal-dominant-polycystic-kidney-disease-adpkd-302101896.html
2. A Safety, Tolerability, and Pharmacokinetics Study of AP303 in Healthy Subjects.ClinicalTrials.gov identifier: NCT05503693. Updated July 17, 2023. Accessed March 29, 2024. https://www.clinicaltrials.gov/study/NCT05503693
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