• Early data from a Phase 1 first-in-human trial of AIC468, a second-generation antisense oligonucleotide (ASO), showed promising pharmacokinetic (PK) properties in healthy volunteers.
  • AIC468 has demonstrated a safe and tolerable profile in all patients assessed thus far.
  • Preclinical results combined with human PK data suggest that effective kidney concentrations can be reached with weekly or less frequent doses within the studied range.

Wuppertal, Germany, August 5, 2025 – Aicuris today shared initial clinical findings from its ongoing Phase 1 first-in-human trial of AIC468. This novel antiviral antisense oligonucleotide is being developed to treat BK virus (BKV) infections in kidney transplant recipients. The interim data, presented at the World Transplant Congress in San Francisco on August 4, 2025, detail the PK characteristics of AIC468 from the Phase 1 clinical trial involving healthy volunteers.

“BK virus infections pose a significant and ongoing risk for immunocompromised patients, potentially leading to renal damage or kidney loss after transplantation. The PK results and safety data obtained to date are highly encouraging, offering valuable insights for the continued development of this unique program,” stated Cynthia Wat, MD, CMO of Aicuris. “Our objective is to deliver effective therapeutic solutions for immunocompromised kidney transplant recipients, and these robust PK and safety data represent a crucial step towards achieving this goal.”

Data from the first-in-human trial, encompassing 72 healthy volunteers across six single ascending dose (SAD) subcutaneous cohorts, one intravenous dose level, and two multiple ascending dose (MAD) subcutaneous cohorts, were presented at the World Transplant Congress. AIC468 exhibited safety and tolerability across all cohorts examined to date.

The product candidate displayed excellent bioavailability (82%), rapid absorption, and distribution to peripheral tissues. A favorable half-life, combined with preclinical data, indicates that effective kidney concentrations can be achieved with weekly or less frequent dosing within the investigated dose range. Furthermore, renal clearance of AIC468 accounted for less than 2% of total clearance, indicating a negligible route of elimination. Tissue uptake and subsequent metabolism appear to be the primary route of clearance, supporting the observed favorable safety profile. The trial is advancing as planned in the third MAD cohort.

“Aicuris maintains its momentum by executing our clinical trial strategy and achieving the first positive data set for our third clinical program targeting immunocompromised patients,” added Larry Edwards, CEO of Aicuris. “We anticipate that data from the Phase 1 program in the second half of 2025 will enable us to rapidly advance AIC468 into a Phase 2a proof-of-mechanism trial in H1 2026, demonstrating its value for patients.”

The randomized, double-blind, placebo-controlled first-in-human trial is designed to assess the safety, tolerability, and pharmacokinetics of AIC468 in healthy volunteers. SAD cohorts involved a total of 56 healthy volunteers who received either AIC468 or a placebo control across six subcutaneous (25 mg to 600 mg) and one intravenous dose level (200 mg). In the MAD cohorts, an additional 24 subjects received five repeated doses of AIC468 (130 mg, 230mg, and 330 mg). Dosing has been successfully completed in all SAD and the first two MAD cohorts. The trial is progressing as scheduled in the third MAD cohort, with data expected later this year.

About BKV
BKV is a common polyomavirus that infects most people early in life, usually without causing symptoms. However, in immunocompromised individuals, such as organ transplant recipients, BKV can reactivate, leading to serious health complications. In kidney transplant patients, BKV reactivation can lead to BK virus-associated nephropathy (BKVAN), affecting up to 10% of recipients and potentially causing graft loss. Current treatment involves reducing immunosuppressive therapy, which increases the risk of graft rejection. Despite its prevalence, there is currently no approved antiviral treatment specifically for BKV.

About AIC468
AIC468 is an antisense oligonucleotide therapy being developed to treat BK virus reactivation in kidney transplant patients, a significant health risk for this population. The candidate works by blocking viral replication within infected cells by inhibiting the splicing of the pre-mRNA that encodes the virus’s large T-antigen. This novel approach has already shown 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 addresses the unmet needs of the growing population of immunocompromised individuals who require precise therapies to effectively treat infections. Our flagship product, PREVYMIS®, marketed by our partner MSD, prevents CMV in a specific group of transplant recipients. Our pivotal Phase 3 candidate, pritelivir, is intended to treat refractory HSV infections in a broad population of patients with weakened immune systems. For immunocompromised people, a manageable infection can become life-threatening. Aicuris, with its expertise and expanding pipeline, is committed to providing therapeutic solutions for them now and in the future.

Contact:
Aicuris Anti-infective Cures AG
info@aicuris.com

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