AM-Pharma Completes Enrollment in Phase 2 Study of Ilofotase Alfa for Prevention of Cardiac Surgery-Associated Renal Damage

Utrecht, The Netherlands, October 2, 2025 – AM-Pharma B.V. today announced that it has successfully completed enrollment of patients in its ongoing Phase 2 clinical trial evaluating ilofotase alfa for the prevention of cardiac surgery-associated renal damage (CSA-RD). CSA-RD, a common cause of Acute Kidney Injury (AKI), is a serious complication of cardiac surgery occurring in up to 40% of patients. AKI following surgery is associated with both short- and long-term impairment of kidney function, an increased risk of requiring renal replacement therapy, sometimes lifelong, and higher mortality rates. Ilofotase alfa is the company’s proprietary therapeutic candidate that has consistently demonstrated safety, tolerability and reno-protective effects, including a reduction in Major Adverse Kidney Events (MAKE), across global clinical trials involving more than 1,000 patients diagnosed with AKI.

“CSA-RD represents a serious post-surgical complication, often leading to prolonged hospitalization, increased complications such as the need for renal replacement therapy and a higher long-term risk of chronic kidney disease, end stage renal disease, and mortality,” said Peter Pickkers, MD, PhD, Principal Investigator in the Phase 2 study and Professor of Experimental Intensive Care Medicine at Radboud University Nijmegen Medical Centre, The Netherlands. “With no pharmacological therapies currently available, there is a clear need for novel approaches. This trial is designed to confirm and expand upon the reno-protective effects observed with ilofotase alfa in prior studies, with the goal of developing a meaningful treatment option for patients at risk of kidney injury following cardiac surgery.”

The study (NCT06168799) is a multicenter, multinational, randomized, double-blind, placebo-controlled trial that randomized 244 patients at high risk for renal damage following open-heart surgery. Patients received two intravenous (IV) doses, one before and one after surgery, of either 128 mg ilofotase alfa or placebo, and are being followed for 60 days to assess efficacy, safety and pharmacokinetics. The primary endpoint is serum creatinine ratio (sCrR), a sensitive measure of kidney function changes that closely correlates with Major Adverse Kidney Events at 60 days (MAKE60). MAKE60 is an FDA-registrable endpoint, which would serve as the primary endpoint in the expected pivotal Phase 3 trial for ilofotase alfa. Topline data, including data on MAKE60 measured in this study as a secondary endpoint, are expected in early 2026, and the full dataset will be presented thereafter at a scientific conference.

“Reaching this enrollment milestone is a key inflection point in the clinical development of ilofotase alfa, bringing us closer to addressing a significant unmet need in hospital care,” said Juliane Bernholz, PhD, Chief Executive Officer of AM-Pharma. “We are grateful to our dedicated clinical partners and the patients for their vital support and trust. With recruitment now complete, we are focused on the data that will inform our next steps to realize the potential for ilofotase alfa to become the first approved therapy to prevent CSA-RD.”

About ilofotase alfa

Ilofotase alfa is a recombinant alkaline phosphatase, engineered from two human isoforms of the enzyme, which has demonstrated to be highly active and stable in multiple clinical trials. The recombinant enzyme acts by dephosphorylating and detoxifying damage-associated molecular patterns (DAMPs) and pathogen-associated molecular patterns (PAMPs)—including lipopolysaccharide (LPS), ATP, ADP, and other extracellular substrates—that drive acute inflammation, coagulation, and microvascular ischemia in the kidney following sepsis or ischemia-induced injury.

Dephosphorylation of ATP by ilofotase alfa provides dual reno-protective benefits: clearance of pro-inflammatory ATP and generation of adenosine, which activates the tissue-protective adenosine A2a receptor pathway. Through this mechanism, ilofotase alfa has consistently demonstrated safety, tolerability, and reno-protective effects, including a reduction in Major Adverse Kidney Events (MAKE), across clinical studies in patients with sepsis associated-acute kidney injury (SA-AKI), with an even greater effect observed in patients with pre-existing chronic kidney disease (CKD)¹.

AM-Pharma is developing ilofotase alfa as two distinct therapeutic products: an intravenous (IV) formulation for AKI and a highly concentrated subcutaneous (SC) formulation designed for chronic dosing as enzyme replacement therapy in hypophosphatasia (HPP). A Phase 1b study in adult HPP patients, supported by recent preclinical findings, has reinforced the therapeutic potential of the SC formulation by demonstrating dose-dependent, clinically meaningful effects on key biomarkers (PPi, PLP, and PEA) and showing benefits beyond bone health, including improvements in muscle metabolism and stamina. These effects are particularly relevant for adults with HPP, who often suffer from fatigue and mobility limitations.

About cardiac surgery-associated renal damage (CSA-RD)

CSA-RD is a clinical complication that arises from acute kidney injury (AKI) following open heart surgery performed with the use of a cardiopulmonary bypass (CPB) pump. Apart from sepsis, this type of surgery is the most frequent cause of AKI. CSA-AKI occurs in about 1 in 3 patients undergoing this surgical procedure.² There is currently no pharmacological therapy to prevent AKI, and the kidney injury can result in long-term renal impairment as well as the need for renal replacement therapy (RRT) such as dialysis. Notably, mortality can be up to 50% in CSA-AKI patients who require RRT post-operatively.

About AM-Pharma

AM-Pharma strives to develop medicines for patients confronted with severe medical conditions. Our proprietary asset, ilofotase alfa, is being developed for the treatment of patients with acute kidney injury (AKI) and has been granted FDA fast-track status. We also develop ilofotase alfa in the severe rare disease HPP where ilofotase alfa has orphan drug status in the US and EU. With approximately 1,000 subjects evaluated to date in clinical trials, ilofotase alfa has a proven safety profile and a demonstrated kidney benefit. We are a dedicated team driven to bring treatment options to severely ill patients, their families, and acute care professionals. Find out more about us online at: www.am-pharma.com.

Contacts

Investors:
Juliane Bernholz, Chief Executive Officer
j.bernholz@am-pharma.com

Media:
Trophic Communications
Eva Mulder
Phone: +31 6 52 33 15 79
am-pharma@trophic.eu

1 Pickkers, P et al., Intensive Care Med., 2024
2 Andújar A et al., Front. Nephrol., 2023

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