ProQR Therapeutics recently spun out Amylon Therapeutics, a privately held company focused on the development of therapies for central nervous system (CNS) disorders. The CNS field holds much promise, despite some research challenges.

Amylon will specifically focus on developing therapeutics for beta amyloid-related disorders. The spinoff will continue research and work on the development of therapies for CNS disorders.

According to Daniel de Boer, chief executive officer of ProQR, since its inception in 2012 the company has built a group of focused business units with the intention of developing additional programs. Each of these business units has a specific focus, with the ultimate goal of making programs available to as many individuals as possible. The recent spinoff of Amylon successfully adheres to this company strategy. To date, ProQR has dedicated teams of specialists focusing on cystic fibrosis, eye diseases, skin diseases and CNS diseases. 

“Through spinning out Amylon Therapeutics, we position the beta amyloid program for optimal success with a dedicated team and the funding to develop treatments for patients suffering from these devastating brain diseases,” said de Boer.

Thomas De Vlaam, who holds a bachelor’s degree in international medicine and global health, will lead Amylon as chief executive officer. De Vlaam joined ProQR in 2015 to develop the CNS activities with the goal of spinning it out into a new company in the future.

“The unmet need of patients living with beta amyloid-related disorders is very significant.  To date no disease modifying treatments are available,” said Thomas Wisniewski, M.D., director of the Alzheimer’s Disease Research Center in New York. “The technology that Amylon is developing for these patients is built on a solid scientific rationale and holds great promise for patients.”

Wisniewski will serve on the scientific advisory board at Amylon. Philip Scheltens, M.D., Ph.D., professor of Neurology and director of the Alzheimer Centre at the VU University Medical Center Amsterdam, and Gerard Platenburg, co-founder and chief innovation officer of ProQR, will join him.

ProQR Therapeutics has focused on the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Amylon is exclusively using its technology for beta amyloid-related disorders to initially target a rare disease called Hereditary cerebral hemorrhage with amyloidosis of the Dutch type (HCHWA-D).

 HCHWA-D is the genetic subtype of cerebral amyloid angiopathy (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. After onset of HCHWA-D, the quality of life diminishes quickly and severely. There is currently no intervention available or in development to battle the cause and/or symptoms of HCHWA-D. 

Amylon cites potential expansion into more prevalent indications such as CAA and Alzheimer’s disease for the future.

CAA is the leading cause of hemorrhagic 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.

Research into neurodegenerative diseases, such as Alzheimer’s disease and Parkinson’s disease, for example, presents a particular set of challenges and can be disappointing, according to experts in the field.

“Medicine has made great advancements relating to many areas of the body, but the area that has not seen as much progress is CNS,” said Christopher U. Missling, Ph.D., president and chief executive officer of Anavex Life Sciences Corp.  “This is often the case when the brain is involved because it is so complex.”

However, CNS research has experienced a shift in strategy, from that of a micro-management to more of a macro-management perspective, borrowed from the field of oncology. One new approach involves not blocking receptors, as is usually done, but rather activating a protein with small molecules to assist with CNS diseases.

“The preliminary data is promising with this new approach, although more statistics and data are necessary to confirm,” said Dr. Missling.

At a recent CNS Diseases Summit, sponsored by Global Technology Community, the state of the industry relating to CNS research was discussed at great length. One board member, Iva Toudjarska, a biopharma strategist and business development expert with Halloran Consulting Group, participated on a panel specific to venture capital and public market perspectives in CNS.

According to data provided by the Halloran Consulting Group, neurodegenerative diseases have the highest failure rates in drug development due, at least in part, to complex disease etiology, difficulty with early disease detection and challenging clinical end points and surrogate biomarkers.

“After analyzing publicly available information about completed deals in neurodegenerative diseases, we have observed several trends, including significant increases in deal volume, size and stage of development since 2014,” Toudjarska said.

Despite the industry facing significant challenges with research, recent developments suggest promise and progression for the CNS industry as a whole. Experts agree this is an encouraging trend for those in the field, patients and their caregivers. 

This article was reprinted from Volume 21, Issue 38, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »