RARE Daily

Prilenia Therapeutics Raises $62.5 Million to Fund Late Stage Trials in HD and ALS

June 3, 2020

Rare Daily Staff

Prilenia Therapeutics has raised $62.5 million in a series A financing round to launch its planned late stage clinical trials in Huntington’s disease and amyotrophic lateral sclerosis.

Huntington’s disease (HD) causes uncontrolled movements, emotional problems, and loss of cognitive ability. If a parent has the gene, a child has a one in two chance of inheriting the disease. Adult-onset Huntington’s disease, the most common form of this disorder, usually appears in a person’s thirties or forties. Individuals with the adult-onset form of Huntington disease usually live about 15 to 20 years after signs and symptoms begin. A less common juvenile form of the disease begins in childhood or adolescence. It also involves movement problems and mental and emotional changes.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare progressive neurodegenerative disease impacting nerve cells in the brain and spinal cord. ALS breaks down nerve cells, reducing muscle function and causing loss of muscle control. ALS can be traced to mutations in more than 25 different genes and is often caused by a combination of multiple sub-forms of the condition. Average life expectancy for people with ALS is just three years.

Prilenia’s funding round was led by Forbion and included new investors, Morningside Venture Investments and Sectoral Asset Management, with participation by existing investors Talisman Capital Partners and Genworks 2. also participated in the round. The series A financing brings the total capital invested in Prilenia Therapeutics to $84.5 million since its founding in September 2018 in Israel with the purpose of improving the lives of patients and their families by developing treatments for neurodegenerative and neurodevelopmental disorders.

Proceeds from the current financing will fund two late-stage trials, which could lead to the registration of pridopidine for the treatment of HD and ALS. Pridopidine is a highly selective sigma-1 receptor agonist. It is shown to maintain functional capacity in early HD patients, as measured by the Total Functional Capacity score. It was recently selected from an international competition of more than 30 potential therapeutics for inclusion in the first ever ALS platform trial, led by the Healey Center for ALS at Massachusetts General Hospital. The trials—phase 3 trial in HD and the platform trial in ALS—are expected to commence in the second half of 2020.

Alongside the closing of the financing round, the company appointed Michael Hayden CEO of Prilenia. He has been serving as executive chairman of Prilenia since founding. Haydon, a Huntington’s disease researcher, is the former president of global R&D and chief scientific officer at Teva Pharmaceuticals, where he led the development of 35 new products towards approval in several major markets, predominantly in CNS. He also co-founded five biotechnology companies of which two realized successful exits and three became public.

“With this funing, we will be able to complete two critical clinical trials in disease areas with significant unmet need,” said Hayden. “We believe we have the opportunity to bring real hope to HD and ALS patients and their families and we are keen to accelerate our progress.”

As part of this series A round, a new Dutch company, Prilenia Therapeutics BV has been formed that will hold Israeli subsidiary company Prilenia Neurotherapeutics Ltd.

Photo: Michael Hayden CEO of Prilenia


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