RARE Daily

Vico Therapeutics Raises $31 Million to Advance Therapies for Rare CNS Diseases

July 29, 2020

Rare Daily Staff

Dutch biotech Vico Therapeutics said it raised $31 million (€27 million) in a series A financing round to advance the development of RNA modulating therapies for rare neurological disorders.

Life Science Partners and Kurma Partners co-led the financing, with participation by Pontifax, Droia Genetic Disease, Polaris Partners, Pureos Bioventures, and Idinvest Partners.

Vico will use this funding to further advance its late preclinical stage Antisense OligoNucleotides (AON) lead platform for the development of therapies for different forms of Spinocerebellar Ataxia (SCA) and Huntington disease (HD) into first-in-human clinical trials in late 2021. Its early discovery RNA editing platform is directed towards RETT syndrome.

“We see tremendous potential to advance the field and apply the breadth of our antisense oligonucleotide (AON) expertise to address severe neurological disorders. We are looking forward to accelerating the development of our platform technologies around AON technology and RNA-modulation/editing to bring best-in-class therapies to patients,” said Luc Dochez, founder and chairman of Vico Therapeutics.

SCAs are a group of rare hereditary diseases of the nervous system caused by degeneration of the cerebellum and often other connected brain regions that lead to progressive lack of muscle control and coordination that is caused by degeneration of the cerebellum and often other connected brain regions. More than 30 types of SCAs have been identified with disease onset typically occurring in early/mid adulthood. Ultimately, the disease progresses to total disability resulting in death over the course of 10-20 years after first symptoms.

Huntington’s disease (HD) is a rare inherited neurodegenerative disorder for which there is currently no effective therapeutic. The disease has a wide variation in onset age, with an average age at onset of mid adulthood. This neurodegenerative disorder has a progressive and fatal course characterized by movement disorders, cognitive impairment, dementia and psychiatric manifestations including depression and psychosis. Prevalence is an estimated 4 to 10 per 100,000 population.

Rett syndrome is a neurodevelopmental disorder affecting brain development resulting in severe mental and physical disability. There is no known treatment for Rett syndrome. It is estimated that approximately 1 in 10,000 – 15,000 girls each year are affected and is only rarely seen in boys.

Vico’s lead asset is an experimental AON therapy targeting expanded (CAG) trinucleotide repeats that translate into abnormally long and toxic polyglutamine (polyQ) stretches in proteins that cause neurodegenerative polyQ disorders like spinocerebellar ataxia type 1 and type 3 and Huntington’s disease. 

Vico’s therapy has been demonstrated to preferentially reduce the levels of mutant polyQ compared to wildtype proteins. This was confirmed in several established mouse models for SCA1, SCA3 and Huntington disease, with a long-term and widespread distribution of the compound throughout the central nervous system. Based on these encouraging data combined with the broad applicability of the lead compound to multiple different brain disorders with high unmet medical need, Vico is advancing this program towards a first-in-human trial expected in late 2021.

“A major strength of Vico’s approach is the broad applicability to different polyQ diseases and the selectivity for mutant proteins,” said Martijn Kleijwegt, managing partner at LSP, lead investor, and board member.

Photo: Luc Dochez, founder and chairman of Vico Therapeutics

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