IROS, Prilenia and International Huntington’s Disease Association collaborate on first-ever Huntington’s disease (HD) trial in region

11 Jul, 2024:

IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial for Huntington’s disease (HD) in the UAE and MENA region, using Prilenia’s investigational drug, pridopidine.

In the presence of His Excellency Mansoor Ibrahim Al Mansouri, Chairman of the Department of Health – Abu Dhabi (DoH) and Dr. Fahed Al Marzooqi, Deputy Group Chief Operating Officer of M42, the agreement was signed at the DoH stand during the BIO International Convention in San Diego by Islam ElTantawy, General Manager at IROS, Dr. Michael Hayden, CEO of Prilenia and Svein Olaf Olsen, President of the IHA.

Aligning with Abu Dhabi’s healthcare aspirations for genetic rare disease care, the three organizations collaborating on the clinical study under consideration, aim to raise awareness about HD and to help bring innovative treatments to support patients and their families. This effort is part of Project NADER (Needs Assessment and Therapeutics Development for Rare Diseases), backed by DoH, which focuses on assessing needs and developing treatments for rare diseases.

HD affects up to 14 per 100,000 people worldwide, causing abnormal movements, psychiatric issues and severe cognitive decline. Caused by a mutation in the huntingtin gene it leads to the degeneration of neurons in the brain. As it advances, people gradually lose the ability to maintain employment, manage finances, and carry out essential daily activities, including self-care or simple household tasks. HD is often underreported and misdiagnosed, particularly in the MENA region, making the trial vital for raising awareness and improving patient outcomes.

Dr. Asma Ibrahim Al Mannaei, Executive Director, Research and Innovation Center at the DoH, said: “In reflection of our commitment to address global healthcare challenges and accelerate innovative solutions, the Department of Health – Abu Dhabi is proud to witness the launch of the region’s first clinical trial on Huntington’s disease. Abu Dhabi continues to spearhead life science initiatives as part of its priorities to improve rare disease awareness and management, thus improving healthcare outcomes for patients in the Emirate and beyond.”

Dr. Fahed Al Marzooqi, Deputy Group Chief Operating Officer of M42, said, “This collaboration brings hope to the Huntington’s disease community. There is a crucial need for more research and data on Huntington’s disease in the UAE and MENA as well as a need to ensure its populations are properly represented in clinical trials. At M42, we are committed to delivering specialized care and forming the right partnerships to improve healthcare delivery. The planned trial would represent a significant step towards shedding light on the ‘hidden disease’ and raise awareness at the national and global level.”

Svein Olaf Olsen, President of the IHA, said, “This is the first time a clinical trial in Huntington’s Disease is planned to take place in the region; this collaboration with IROS and Prilenia will aid our understanding of the disease. Unfortunately, many people with HD are regularly misdiagnosed with Parkinson’s or a mental illness, leading to significant frustrations and a decreased quality of life for both patients and their families. By focusing on Huntington’s disease, we are taking an important step toward raising awareness of brain diseases while exploring opportunities to delay their progression and improve the quality of life for those with genetic disorders. Through this trial, we may begin to transform hope into reality for brain disease patients. This is a game-changer for the whole UAE/MENA region.”

Islam ElTantawy, General Manager at IROS, said, “Finding and enrolling the right patients is critical to the success of any clinical trial, but it’s an especially significant hurdle when it comes to rare diseases like Huntington’s. Our partnership with Prilenia Therapeutics and the International Huntington’s Disease Association where we can leverage its deep connections within the Huntington’s community, will ensure we effectively reach and recruit the people who need these treatments the most.”

“Pridopidine is a selective and potent sigma–1–receptor agonist with neuroprotective properties. In clinical studies, it demonstrated consistent treatment benefits across independent measures that are important to patients and families. These measures include day-to-day function, cognition and motor and clinical progression in people living with HD. Our hope is that this collaboration may help in the process of bringing this innovative new treatment to those people with HD in the UAE and MENA region who are in need of new options,” said Dr. Michael R. Hayden, CEO of Prilenia.

The DoH led a high-profile Abu Dhabi delegation, headed by His Excellency Mansoor Ibrahim Al Mansoori, Chairman of the DoH, to the United States from May 30 to June 6 to showcase the emirate’s partnership opportunities and explore collaboration with leading organizations in research and development, manufacturing and innovation. The delegation began its mission in Philadelphia, where they met with existing and new partners to foster collaboration with leading educational research institutions, governmental bodies and health-tech giants. The transnational mission concluded in San Diego, coinciding with Abu Dhabi’s participation at the BIO International Convention 2024, highlighting the emirate’s growth and development in the biotechnology industry. The event included in-depth discussions, shared insights and expertise as well as explored collaborations in health-tech, life science and innovation.