Takeda joins Pentavere to advance new HAE, rare disorder treatments
Collaboration has resulted in one of largest HAE-nl-C1INH patient datasets
A research collaboration between Pentavere Research Group and Takeda Canada has led to the creation of one of the largest cohort datasets of patients with an ultra-rare form of hereditary angioedema (HAE) where C1 inhibitor (C1-INH) protein levels are normal.
The public-private partnership seeks to “revolutionize” diagnosis and care for all rare disease patients in Canada by driving real-world evidence, but the focus is to address critical data gaps on the prevalence and clinical outcomes of this rare form of HAE, sometimes called HAE-nl-C1INH, for which there are no approved treatments.
A dearth of real-world data on these patients poses a significant challenge to developing new treatments, according to Takeda Canada, which has teamed up with Toronto-based Pentavere, a Healwell AI subsidiary and artificial intelligence (AI) digital health data company, and top HAE specialists across Canada with the aim of supporting research in the gaps in treatment guidelines and approved HAE-nl-C1INH therapies.
“We are pleased to be partnering with Pentavere as this study is an ongoing example of our steadfast commitment to working with leading data science companies to drive innovation and assist patients in their care journey,” Jefferson Tea, PhD, vice president of medical and scientific affairs at Takeda, said in a press release. “By harnessing the power of AI and data analytics, and collaborating with experts in these fields, we aim to advance the understanding and management of rare diseases like HAE, supporting patients and their families in their care journey, and ultimately improving patient outcomes.”
The hope is that the comprehensive dataset will be pivotal in closing the real-world data gap on the burden of disease and clinical outcomes for this patient population.
Using AI toward HAE care
Using advanced data science techniques, Pentavere analyzed the data as part of Takeda’s ongoing noninterventional, retrospective, observational PROSPECT study (NCT05578417), which is evaluating the treatment and outcomes of teens and adults in Canada with HAE-nl-C1INH and non-histaminergic angioedema with normal C1 inhibitor.
The analysis sought to glean actionable insights to inform treatment strategies and improve patient outcomes. A manuscript and abstract on the study data are being prepared and will be published.
Pentavere unlocks a better understanding of patient needs by employing its proprietary AI system DARWEN, which can harness electronic medical record data, and uses advanced large language models to assess clinical notes that may be difficult to access.
“When the number of patients is small, the use of AI and data analytics to understand the whole of their patient journey to inform diagnosis and best management is a game changer that could prove as impactful as decoding the human genome,” said Durhane Wong-Rieger, PhD, CEO of the Canadian Organization for Rare Disorders (CORD). “We are extremely grateful to Pentavere, HEALWELL, and Takeda for investing in this initiative and for including CORD as a partner to drive to a vision where this is available for all patients.”
HAE is mainly caused by SERPING1 gene mutations that impair the production or activity of C1-INH, which normally impedes proteins that promote bradykinin, a signaling molecule that causes blood vessels to widen and become more permeable. Producing too much bradykinin can lead to the swelling attacks that mark HAE by causing fluid from blood vessels to leak and pool in nearby tissues.
In rare cases, people can have a form of HAE associated with mutations in other genes and where C1-INH’s levels and activity are normal. The number of cases of HAE-nl-C1INH in the U.S. was estimated to be between 1,230 and 1,331 from May 2019 and April 2020, according to a 2023 survey of U.S. physicians.
The collaboration builds on Pentavere winning the Takeda Canada Innovation Challenge in 2022 to advance rare disease diagnosis and care using new AI technology.
“We are incredibly proud of our ongoing partnership with Takeda and how it has grown and evolved since winning the Takeda Canada Innovation Challenge to accelerate rare disease diagnosis and care,” said Aaron Leibtag, Pentavere’s CEO. “This collaboration not only underscores the significance of Pentavere’s technology, but also highlights the transformative impact data science can have in healthcare. Together, we are breaking new ground in real-world evidence analysis for rare diseases, reaffirming our commitment to driving innovation and improving patient outcomes.”
About the Author
