- Initial results from the First in Human (FIH) Phase 1 trial evaluating AIC468, a 2nd generation antisense oligonucleotide (ASO), in healthy volunteers demonstrated favorable pharmacokinetic (PK) characteristics
- AIC468 was safe and well tolerated in all evaluated patients to date
- Preclinical and human PK results suggest that efficacious kidney concentrations can be achieved by weekly or less frequent dosing within the investigated dose range
Wuppertal, Germany, August 5, 2025 – Aicuris Anti-infective Cures AG today announced initial clinical data from its ongoing first-in-human Phase 1 trial with AIC468. The novel antiviral antisense oligonucleotide is in development for the treatment of BK virus (BKV) infections in kidney transplant recipients. The interim results, presented at the World Transplant Congress in San Francisco on August 4, 2025, provide an overview of PK characteristics of AIC468 from the Phase 1 clinical trial in healthy volunteers.
“BK virus infections represent a serious and persistent threat for immunocompromised patients with a high risk of renal damage or kidney loss after transplantation. The PK results and safety data to date are very encouraging and provide us with valuable insights for the continued development of this differentiated program,” said Cynthia Wat, MD, CMO of Aicuris. “Our goal is to achieve tangible therapeutic solutions for immunocompromised patients with kidney transplants, and these strong PK and safety data are an important step for us in achieving this goal.”
FIH trial data from 72 healthy volunteer subjects, six single ascending dose (SAD) subcutaneous cohorts, one intravenous dose level and two multiple ascending dose (MAD) subcutaneous cohorts were presented at World Transplant Congress. AIC468 was safe and well tolerated across all cohorts investigated to date.
The product candidate achieved excellent bioavailability (82%), rapid absorption, and distribution to peripheral tissues. A favorable half-life together with pre-clinical data, suggests that efficacious kidney concentrations can be attained by weekly or less frequent dosing regimens within the investigated dose range. Additionally, renal clearance for AIC468 accounted for less than 2% of the total clearance, representing a negligible route of elimination. Tissue uptake and subsequent metabolism appear to be the primary route of clearance, supporting the observed benign safety profile. The trial is progressing as planned in the third MAD cohort.
“Aicuris continues to build momentum by executing on our clinical trial strategy and achieving the first positive data set for our third clinical program for immunocompromised patients,” added Larry Edwards, CEO of Aicuris. “In the second half of 2025 we expect data from the Phase 1 program to allow us to rapidly advance AIC468 into a Phase 2a proof-of-mechanism trial in H1 2026 to demonstrate its value for patients.”
The randomized, double-blind, placebo-controlled first-in-human trial (2023-510074-13-00) is designed to evaluate the safety, tolerability and pharmacokinetics of AIC468 in healthy volunteers. SAD cohorts tested AIC468 or a placebo control in a total of 56 healthy volunteers across six subcutaneous (25 mg to 600 mg) and one intravenous dose level (200 mg). In the MAD cohorts an additional 24 subjects receive five repeated doses of AIC468 (130 mg, 230mg and 330 mg). Dosing in all SAD and the first two MAD cohorts have been successfully completed. The trial is progressing as planned in the third MAD cohort with data expected later this year.
About BKV
BKV is a ubiquitous polyomavirus that infects most people in early childhood, typically without symptoms. In immunocompromised individuals, such as organ transplant recipients, BKV can reactivate, leading to serious health issues. In kidney transplant patients, BKV reactivation can cause BK virus-associated nephropathy (BKVAN), affecting up to 10% of recipients and potentially resulting in graft loss. Current management involves reducing immunosuppressive therapy, which increases the risk of graft rejection. Despite its prevalence, there is no approved antiviral treatment specifically for BKV.
About AIC468
AIC468 is an antisense oligonucleotide therapy designed to treat BK virus reactivation in kidney transplant patients which can pose a significant health risk for these patients. The candidate blocks viral replication within infected cells by inhibiting splicing of the pre-mRNA that encodes for the virus’ large T-antigen. This innovative approach has already demonstrated potent antiviral activity along with a favorable pharmacokinetic and safety profile in preclinical studies and is currently being evaluated in a Phase 1 clinical trial.
About Aicuris
Aicuris is meeting the needs of the growing population of immunocompromised people who require precise therapies to effectively treat infection. Our flagship product, PREVYMIS®, marketed by our partner MSD, prevents CMV in a defined group of transplant recipients. Our pivotal Phase 3 candidate, pritelivir, aims to address refractory HSV infections in a broad population of patients with weakened immune systems. For immunocompromised people, an otherwise manageable infection can mean life or death. Aicuris, with its expertise and growing pipeline, is committed to providing therapeutic solutions for them now and in the future.
Contact:
Aicuris Anti-infective Cures AG
info@aicuris.com
Trophic Communications
Dr. Stephanie May and Dr. Charlotte Spitz
Phone: +49 171 3512733
Email: aicuris@trophic.eu
