We recognize the work of Allievex Corporation, and the company's dedication to treating children with #SanfilippoSyndrome type B. Recent successes with biomarkers in #MPS have been encouraging. Despite the pragmatism of Dr. Patrizia Cavazzoni, the Director of CDER, in reconsidering initial decisions, small companies lack clarity for accelerated approval and more importantly so do patients. We are profoundly sad for the families and patients impacted by the loss of this program. #RareDisease #Allievex
It is with very heavy hearts that we share this update from Allievex Corporation. Cure Sanfilippo Foundation has worked alongside the Allievex team and patient families to share the real world impact of AX 250 with FDA regulators over the years. This spring we were so encouraged by FDA’s agreement to use spinal fluid heparan sulfate levels as a surrogate biomarker. Sadly, in the business lifecycle of this drug development program, time was not on our side. Our community will continue to press forward to support the development of treatments for those who are losing access to this treatment and all individuals affected by Sanfilippo type B. #CureSanfilippo - - - - - - - - - - - - - - - Letter from Allievex on July 24, 2024 Dear Parents, Families, Children, Caregivers and Clinicians, As you are aware, Allievex terminated its business operations in October 2023, to include all clinical studies treating children with Sanfilippo syndrome type B with our enzyme replacement therapy, tralesinidase alfa (AX 250). At the same time, we laid off all seven employees as we sought a buyer for our program. During this time, the former employees of Allievex worked tirelessly to ensure that we were able to initiate investigator sponsored treatments of some children who were previously receiving treatment in our clinical program. However, these treatments are dependent on the remaining drug product supply, which is very limited and will be exhausted before the end of this year. Yesterday, Allievex entered an Assignment for the Benefit of Creditors under the supervision of the Chancery Court of Delaware. This is ultimately a formal liquidation of our assets to pay our creditors. Since 2019, the lack of alignment with the U.S. Food and Drug Administration on a development pathway towards an Accelerated Approval left the company with very little options to raise the necessary capital or to be acquired. On March 15, 2024, former Allievex employees met with the FDA, and the Center for Drug Evaluation and Research completely changed course and encouraged Allievex to file its Biologics License Application for consideration under Accelerated Approval Pathway. I credit the pragmatism of Dr. Patrizia Cavazzoni, the Director of CDER, in this change at FDA. While this late breaking good news was not enough to allow us to solve our business situation, we do take great pride in our advocacy at FDA and your commitment to our clinical trials. Together, we have now enabled multiple other sponsored programs in development in MPS disorders to move rapidly forward to potential Accelerated Approval for diseases like MPS I, MPS II and MPS III. You have seen these recent announcements from companies like Denali, Ultragenyx and RegenxBio. See comments for the remainder of the letter's contents.