Merck KGaA, Darmstadt, Germany, Announces Positive Phase III Results for Arpraziquantel as Part of its Schistosomiasis Elimination Program
- Primary efficacy endpoint of clinical cure was met with a favorable safety profile
- Arpraziquantel is a potential new treatment option for the estimated 50 million preschool-aged children with schistosomiasis
- Merck KGaA, Darmstadt, Germany, intends to submit regulatory file to the European Medicines Agency (EMA)
Merck KGaA, Darmstadt, Germany, a leading science and technology company, and the Pediatric Praziquantel Consortium today announced that the pivotal Phase III trial for arpraziquantel, a potential new treatment option for the neglected tropical disease schistosomiasis, has met the primary endpoint of clinical cure in children 3 months to 6 years of age. Arpraziquantel is tailored to the needs of the estimated 50 million preschool-aged children suffering from schistosomiasis, who currently have no treatment option, and forms part of an ongoing effort to eliminate this devastating parasitic disease.
“With this milestone, we continue our commitment to eliminating schistosomiasis and ensuring all people affected by this neglected tropical disease have access to a life-saving therapy. Together with our Consortium partners, we are steadfast in our vision to bring new hope to the world’s most vulnerable populations,” said Peter Guenter, Member of the Executive Board of Merck KGaA, Darmstadt, Germany, and CEO Healthcare.
Through its Schistosomiasis Elimination Program, Merck KGaA, Darmstadt, Germany, addresses the key requirements of the WHO’s 2021-2030 Roadmap for Neglected Tropical Diseases and contributes to the United Nations’ Sustainable Development Goals, especially Good Health and Well-Being (Goal 3) and Partnerships (Goal 17), in line with the company’s sustainability strategy.
Clinical Phase III shows high efficacy across all age groups
In the completed Phase III trial, children aged 3 months to 6 years infected with S. mansoni or S. haematobium were enrolled in different age groups and treated with a single dose of arpraziquantel. High efficacy was observed with cure rates close to or above 90% for S. mansoni (at a dose of 50 mg/kg) and S. haematobium (at a dose of 60 mg/kg). The primary endpoint of clinical cure, defined as no parasite eggs in the stool (S. mansoni) 17 to 21 days after treatment or urine (S. haematobium) 17 to 21 days and additionally 35 to 40 days after treatment, met the pre-specified success criteria. Arpraziquantel treatment at both doses demonstrated favorable safety, tolerability and improved palatability among preschool-aged children. No new potential risks or safety concerns were identified.
Derived from praziquantel, arpraziquantel is an orally dispersible tablet developed by the Pediatric Praziquantel Consortium, a public-private partnership led by Merck KGaA, Darmstadt, Germany, through its Global Health Institute, with the aim to close the current treatment gap for an estimated 50 million preschool-aged children with schistosomiasis.
Conducted by the Université Félix Houphouet-Boigny in Côte d’Ivoire and the Kenya Medical Research Institute in Kenya, the Phase III trial was supported by the Global Health Innovative Technology (GHIT) Fund and the European & Developing Countries Clinical Trials Partnership (EDCTP).
Michael Makanga, Executive Director, EDCTP said: “With the completion of the Phase III trial, the Pediatric Praziquantel Consortium demonstrates that balanced North-South collaboration with complementary expertise, bidirectional knowledge sharing, and mutual trust, is a key success factor to develop and deliver safe and affordable treatments for neglected tropical diseases, such as schistosomiasis.”
Kio Yamabe, Acting CEO of the GHIT Fund said: “Having partnered with the Pediatric Praziquantel Consortium since 2013, we believe that international collaborations like this are key to addressing the burden of major infectious diseases in the developing world. The successful joint development of arpraziquantel by Consortium partners, Astellas, Merck KGaA, Darmstadt, Germany, and Farmanguinhos embodies our unwavering commitment to drive Japanese innovation and technology through global partnerships.”
Merck KGaA, Darmstadt, Germany, on behalf of the Consortium, intends to apply for a scientific opinion by EMA under the EU-M4all procedure for high-priority medicines for human use intended for markets outside the European Union. A positive opinion by EMA would facilitate the inclusion of arpraziquantel in the World Health Organization (WHO) list of prequalified medicinal products as well as regulatory approvals in endemic countries. With continued support from the GHIT Fund and EDCTP, the delivery of arpraziquantel is being prepared through the Consortium’s access program, ADOPT. In addition to Farmanguinhos in Brazil, Merck KGaA, Darmstadt, Germany, has also partnered with Universal in Kenya for extensive local production capacities for endemic African countries.
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