WEST HOLLYWOOD, Calif.--(BUSINESS WIRE)--Beren Therapeutics, P.B.C. (Beren), a biotechnology company, today announced that its novel lead asset, BRN-002, was awarded an Innovation Passport under the United Kingdom’s Innovative Licensing and Access Pathway (ILAP), for the reversal of atherosclerosis in patients with Homozygous Familial Hypercholesterolemia (HoFH). The ILAP aims to accelerate the time to market for innovative medicines that address the needs of patients with life-threatening or seriously debilitating diseases.
HoFH is a rare genetic disease where patients have high levels of LDL cholesterol typically beginning in childhood causing plaque to build up in the arteries (atherosclerosis). The disease is currently managed with LDL-apheresis beginning as early as the age of seven and other LDL-lowering therapies. Despite the current therapies and treatments, the average patient life expectancy is between 30 and 40 years. It is estimated that HoFH affects as many as 1 in 250,000 people in the UK. Untreated, HoFH results in premature heart disease including heart attacks and aortic valve disease beginning in the early teen years and sometimes even earlier, resulting in a large impact on quality of life. BRN-002 is being developed to reverse the underlying cause of atherosclerosis, plaque in the arteries. Currently, there are no approved therapies or treatments that reverse atherosclerosis.
“The Innovation Passport provides Beren the opportunity to collaborate with MHRA and its partner agencies to develop a roadmap for development and regulatory milestones with the primary goal of achieving early patient access for the treatment of HoFH in the UK while ensuring patient safety and efficacy,” said Jason Camm, Beren CEO. “The inter-agency collaboration model inherent to the ILAP program is ideal for developing novel therapies that can broadly impact patients and public health. Programs like ILAP align with Beren’s founding vision to develop novel therapies hand in hand with the relevant stakeholders and to make the right decisions at the right time to optimize for speed to approval, and access and distribution once approved. We are excited to begin work under ILAP, and look forward to collaborating with other regulators, payors and governments to expedite development and identify and remove access barriers.”
“HoFH patients desperately need novel treatments to halt and reverse this debilitating disease, and the acceleration of this clinical program will hopefully allow a new medicine to be available sooner,” said Jules Payne, CEO of HEART UK. “Significant unmet needs remain and with this advancement, we are hopeful for a new treatment option that can truly alter the course of the disease for the children and patients living with HoFH.”
About ILAP and Innovation Passport
The Innovation Passport is awarded by the UK’s Innovation Licensing and Access Pathway Steering Group, which consists of representatives from MHRA, NICE, the Scottish Medicines Consortium (SMC), and the National Health Service (NHS) England. To enter ILAP and receive an Innovation Passport, an experimental drug needs to meet the following public health and/or patient-centric criteria: (1) the condition is life-threatening or seriously debilitating; (2) the program fulfills at least one of the following: innovative medicine, clinically significant new indication or, it is intended for a special population; and (3) the medicine has the potential to offer benefits to patients.
The ILAP aims to accelerate the time to market and facilitate patient access to medicines through the development of a target development profile (TDP) that outlines a unique product-specific roadmap for regulatory and development milestones. Other benefits provided by ILAP include the potential for a 150-day accelerated assessment of a Marketing Authorization Application (MAA) and rolling review. The ILAP also provides opportunities for frequent interactions with the review staff at the MHRA and its partner agencies to discuss the drug’s development and regulatory plans, as well as reimbursement.
About Beren Therapeutics, P.B.C.
Beren Therapeutics, P.B.C. is currently in stealth. More details will be available soon.