Amylon Therapeutics announces seed investment round, focus on CNS disorders

 

Key updates

  • Amylon Therapeutics B.V. is a spin-off company from ProQR Therapeutics N.V., a NASDAQ listed biopharmaceutical company based in Leiden, the Netherlands.
  • Amylon announces the closure of its first investment round.
  • Amylon will use these funds to further advance the development of AT-010, a drug candidate targeting Katwijk’s disease.

 

LEIDEN, the Netherlands, September 12, 2017 – Amylon Therapeutics B.V., a spin-off company of ProQR Therapeutics, today announced the closure of its first investment round. The seed round was provided by a consortium of private and institutional investors. ProQR becomes a majority shareholder. Amylon Therapeutics targets Central Nervous System (CNS) disorders through an ultra-genetics approach, focusing on rare genetic disorders that can serve as a possible gateway to global indications.

 

Amylon will use the investment funds to continue research of therapies for CNS disorders, with a primary focus on its lead program AT-010, a drug candidate targeting Katwijk’s disease. Katwijk’s disease is a rare genetic beta amyloid-related disorder that causes the early onset of hemorrhagic (bleeding) strokes during mid-adulthood. Future beta amyloid related targets could include cerebral amyloid angiopathy (CAA). CAA is a frequent occurring disease and the primary cause of hemorrhagic (bleeding) stroke. Currently there is no treatment for these diseases.

 

Amylon is a spin-off company from ProQR Therapeutics N.V., a NASDAQ listed biopharmaceutical company based in Leiden, the Netherlands. Amylon is led by founder Thomas de Vlaam, a science entrepreneur who invented and led the program at ProQR Therapeutics as therapeutic area leader for CNS indications. “I am incredibly excited to announce that we have closed our first investment round. It is a great compliment and affirmation that we are heading in the right direction with our company and program,” said De Vlaam, Chief Executive Officer of Amylon.  “We are grateful to have been able to incubate Amylon under the wings of ProQR. The access to knowledge and the opportunity to work with such an accomplished group of people has allowed us to mature to a point that we are confident in moving forward independently.”

 

”The unmet need of patients living with beta amyloid related disorders is very significant and to date no disease modifying treatments are available,” said Thomas Wisniewski, 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.”


Amylon also announces that Dinko Valerio, founder and former CEO of Crucell and chairman of the supervisory board at ProQR, will join Amylon as a member of the supervisory board. “Targeting the brain has historically been a difficult challenge. The recent breakthroughs in technology however have dramatically increased our chances to tackle these devastating disorders,” said Valerio. ”We believe that the time to move into CNS is now, and that belief is the foundation of Amylon. I am very pleased to be given the chance to help start Amylon and support Thomas with this exciting venture.”


The scientific advisory board at Amylon will consist of the following members:

  • Philip Scheltens MD, Professor of Neurology and Director of Alzheimer Center at the VU University Medical Center Amsterdam.
  • Thomas Wisniewski MD, Director of the Alzheimer’s Disease Research Center NYC, Professor of Neurology, Pathology and Psychiatry at the New York University School of Medicine.
  • Gerard Platenburg, co-founder and Chief Innovation Officer at ProQR.

 

About Amylon

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 Therapeutics, Amylon enjoys a wealth of knowledge and experience through its shareholders and network.

About AT-010

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 Beta-amyloid peptide.

About ProQR

ProQR Therapeutics is dedicated to changing lives through 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. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

About CAA

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.

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