Amylon Therapeutics and Alcyone Lifesciences enter into partnership on drug-device combo to treat HCHWA-D
Cambridge, Massachusetts, April 5, 2018
Amylon Therapeutics, a biotechnology company using an ultra-genetics approach towards neurological disorders, and Alcyone Lifesciences, a leader in precision advanced central nervous system (CNS) delivery systems, announce that they have entered into a collaboration partnership agreement. The companies will join forces to combine Amylon’s lead program AT-010 and Alcyone’s proprietary PulsarTM Advanced Intrathecal Precision Delivery Platform Technology, using their combined expertise to develop a drug-device combination.
Amylon and Alcyone will collaborate to investigate the potential of developing AT-010 and the PulsarTM platform into a drug-device combination that will allow AT-010 to reach the brains of patients. “We are incredibly excited to announce our partnership with Alcyone”, said Thomas de Vlaam, founder and CEO of Amylon. “The blood-brain-barrier normally prevents most therapeutic compounds from entering into the brain and reaching the affected areas. I believe that combining our knowledge of therapeutic development with Alcyone’s expertise on delivery is a great example of how the CNS field is evolving to get therapies to patients in the fastest way possible.”
Amylon’s lead program AT-010 is designed to prevent the onset of brain bleeds in the orphan disease HCHWA-D. Through an ultra-genetics approach, Amylon focuses on a rare genetic CNS disorder that could pave the way for going after CAA – the world’s largest cause of cerebral haemorrhage. AT-010 is a first-in-class RNA-based oligonucleotide that works by preventing the accumulation of toxic protein by acting at the RNA level.
Alcyone’s proprietary PulsarTM advanced precision delivery platform is an auto-intrathecal injector that utilizes cerebrospinal fluid dynamics to deliver therapeutics to anatomical areas in the CNS that are difficult to target. This platform has been demonstrated to increase the concentration of the therapeutic to CNS targets of interest consistently and reliably. The platform is designed to for easy deployment in the clinical setting and allows for ease of use with custom infusion algorithms for treating CNS diseases.
“This collaboration is based on foundational values that Alcyone and Amylon share”, said PJ Anand, CEO of Alcyone Lifesciences. “To develop life changing therapies based on highest quality of science and focus on patients. We look forward to making significant contribution to Amylon’s AT-010 program.”
Amylon Therapeutics targets CNS disorders through an ultra-genetics approach, focusing on rare genetic disorders that can serve as a possible gateway to more frequently occurring indications. The anti-amyloid technology of Amylon allows a focus on different disorders, with a primary focus on HCHWA-D and CAA. Amylon is led by founder Thomas de Vlaam, complemented by a scientific board of experienced scientists and biotech veterans. As a spin-off from the NASDAQ traded ProQR (Nasdaq: PRQR) Therapeutics, Amylon enjoys a wealth of knowledge and experience through its shareholders and network.
About Alcyone Lifesciences, Inc.
Alcyone Lifesciences, based in Massachusetts, is a privately-held company focused on transforming disease modifying therapies for rare, orphan and debilitating neurological disorders through an advanced precision delivery platform. For more information, please visit www.alcyonels.com.
AT-010 is a first-in-class RNA-based oligonucleotide that induces splicing modulation in the mature Amyloid Precursor Protein mRNA resulting in a protein that lacks the aggregation prone C-terminal segment of the Beta-amyloid peptide.
Cerebral Amyloid Angiopathy (CAA) is the leading cause of hemorrhagic (bleeding) stroke in the elderly. The disease is caused by the aggregation and deposition of the toxic peptide Amyloid Beta in the blood vessels in the brain. These vascular amyloid deposits ultimately cause irreparable damage to the vessel walls and lead to hemorrhagic stroke. There are currently no effective prevention or treatment strategies for CAA.
About Katwijk’s disease/HCHWA-D
Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch type (HCHWA-D), more commonly known as Katwijk’s disease, is the genetic subtype of CAA, in which a point mutation leads to the accelerated onset of disease. The condition is characterized by a progressive loss of intellectual function (dementia), stroke, and other neurological problems starting in mid-adulthood. Most affected individuals die within a decade after signs and symptoms first appear, and after the onset the quality of life diminishes quickly and severely. There is currently no intervention available nor in development to battle the cause and/or symptoms of HCHWA-D.