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03 June |  2020

Prilenia therapeutics raises $62.5m to fund late stage trials in HD and ALS

•  Forbion leads Series A financing round, accompanied by Morningside, Sectoral and Talisman
•  Funding to support two late-stage trials of pridopidine in Huntington Disease (HD) and Amyotrophic
   Lateral Sclerosis (ALS)
•  Former Chairman, Michael R. Hayden, MD PhD, appointed CEO of Prilenia

[Naarden, NL and Herzliya, IL, 03 June 2020] - Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, announces that is has raised $62.5M in a Series A financing round to launch the Company’s planned HD and ALS late-stage clinical trials.
The funding round was led by Forbion and included new investors, Morningside Venture Investments and Sectoral Asset Management. 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 since its foundation in September 2018 to $84.5M.

The proceeds 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 (S1R) agonist. It is shown to maintain functional capacity in early HD patients, as measured by the Total Functional Capacity (TFC) score. Furthermore, it was recently selected from an international competition of over 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 H2 2020.

Alongside the closing of the financing round, Michael R. Hayden, MD, PhD, has been appointed as CEO of Prilenia. He has been serving as Executive Chairman of Prilenia since the Company was founded. Michael is a world-renowned scientist in Huntington’s Disease research. He 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. Michael co-founded five biotechnology companies of which two realized successful exits and three became public, most recently 89bio (ETNB).

About Pridopidine

Prilenia’s lead asset is Pridopidine, a first-in-class drug candidate with an established safety profile and therapeutic potential in several neurodegenerative diseases affecting adults and children. The highly selective S1R agonist was acquired from Teva in 2018.

Pridopidine for Huntington Disease

HD is a fatal, inherited, neurodegenerative disorder. Every offspring of an HD patient has a 50% chance of inheriting the gene. Usually starting at around 40 years of age, HD patients suffer from movement disorder, progressive functional and cognitive decline, psychiatric disturbances and behavioral symptoms. Following diagnosis, functional, motor and cognitive functions decline steadily, ultimately leading to immobility, dementia and premature death.
Pridopidine has demonstrated maintenance of functional capacity in HD patients, as measured by Total Functional Capacity (TFC), in a clinical trial. This effect was most prominent in early stage HD patients (HD1 and HD2), who showed functional benefit from pridopidine 45 mg, taken twice a day.

There is extensive preclinical evidence that further supports pridopidine’s potential beneficial effect in HD. The therapeutic effect has been shown to be mediated exquisitely by the sigma-1 receptor (S1R) using multiple deletion and antagonist models.

Prilenia has an orphan drug designation for pridopidine for the treatment of HD in both the US and Europe.

Pridopidine for ALS

ALS is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently three FDA therapies approved specifically for treating ALS: riluzole, nuedexta and edaravone.

Compelling preclinical data supports the potential use of pridopidine as a therapeutic for ALS. In ALS SOD1G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specifically, pridopidine improves BDNF (brain-derived neurotrophic factor) and GDNF (glial cell line-derived neurotrophic factor) axonal transport, restores synaptic activity and neuro-muscular junction (NMJ) function, and increases neuronal survival. In vivo, pridopidine treatment of SOD1G93A mice reduces toxic protein aggregates and ameliorates muscle fiber wasting.

Previous clinical data also suggest that S1R is a promising target for ALS therapy, indicating that S1R activation may enhance bulbar and speech function in ALS patients. The sigma 1 receptor has been genetically validated for ALS, as patients with mutations in this gene develop ALS.

In addition to the HD and ALS programs, Pridopidine is also being studied for the treatment of Parkinson’s Disease Levodopa Induced Dyskinesia (PD-LID) and other conditions.

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