After experiencing a setback in its phase 3 study for sepsis-associated acute kidney injury (SA-AKI), Dutch biotech company AM-Pharma is charting a new course for its lead program. The trial, focused on ilofotase alfa, was discontinued in October 2022 due to a lack of efficacy in reducing all-cause deaths at the 28-day mark. SA-AKI is a severe and intricate consequence of sepsis, where the body’s immune response turns against itself, leading to high mortality rates and unfavorable outcomes.
Despite the primary study failure, a glimmer of hope emerged from a pre-specified secondary analysis involving all 649 patients. This analysis revealed a reduction in major adverse kidney events by day 90 among patients treated with ilofotase alfa, prompting optimism within the company.
However, AM-Pharma acknowledges that change is necessary. In response, the company has announced a workforce reduction, though specific numbers were not disclosed. As per its LinkedIn page, the company currently employs 33 individuals.
Additionally, the long-standing CEO, Erik van den Berg, is passing the torch to Juliane Bernholz, Ph.D., who previously held the position of chief operating officer. Van den Berg, who has led the company since 2011, will transition to the company’s supervisory board and continue as a strategic advisor.
“With the updated development strategy and funding in place for the CSA-RD Phase 2 study and awaiting imminent results from the Phase 1b in HPP, I am excited to pass on the baton to Juliane after serving 12 years as AM-Pharma’s CEO. Juliane’s expertise in clinical and corporate development, together with her experience as AM-Pharma’s COO over the past four years, will be extremely useful as the company pursues its development strategy. I am grateful to all at AM-Pharma and the clinical site staff who have contributed to the development of ilofotase alfa from initial studies to the current clinical stage of the program.”
– Erik van den Berg, Supervisory Board member and former Chief Executive Officer of AM-Pharma
AM-Pharma is shifting its focus to two indications for ilofotase alfa. Leading the way is the investigation of cardiac surgery-associated renal damage in a phase 2 study, which will enroll 150 patients at risk of kidney damage based on pre-operative kidney function, scheduled for complex open-heart surgery.
Simultaneously, a phase 1b study is underway, exploring ilofotase alfa as an enzyme replacement therapy for patients with the rare bone metabolism disorder hypophosphatasia. Initial clinical results from this study are anticipated before the end of the year.
AM-Pharma boasts support from notable pharmaceutical giants, including Pfizer, Takeda, and AbbVie, among others like Nestle, Cowen Healthcare Investments, and Forbion. Pfizer’s involvement is particularly noteworthy, as the company invested $87.5 million in 2015 for a minority stake in AM-Pharma, with an option to acquire the company for $512.5 million upon favorable phase 2 results. However, Pfizer opted not to proceed after reviewing the data in 2018.
The revised clinical development strategy has garnered support from existing investors, who have provided additional funding, although specific financial details have not been disclosed. This strategic pivot reflects AM-Pharma’s determination to navigate the challenging terrain of drug development and make a positive impact on patients’ lives.