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NAARDEN, Netherlands & WALTHAM, Mass.--( BUSINESS WIRE )-- Prilenia Therapeutics B.V. , a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, announces the acceptance of its European Marketing Authorisation Application (MAA) for pridopidine (45 mg orally twice daily) for the treatment of adults with Huntington’s disease (HD) by the European Medicines Agency (EMA). Pridopidine is a highly selective and potent sigma-1 receptor (S1R) agonist with an established safety profile. If approved, pridopidine could be the first treatment for Huntington’s disease with the ability to impact progression. The MAA is based on the totality of evidence for safety and efficacy from pridopidine’s extensive development program. “HD is a genetic, rare, fatal neurodegenerative disease that remains without any approved therapeutic options capable of impacting the progression of the disease. All we have to give patients right now is a small handful of options that may offer some degree of symptom control for HD-related chorea and behavioral complications,” said Dr. Ralf Reilmann, Board Certified Neurologist and Founding Director of the George-Huntington-Institute in Muenster, Germany . “Patients deserve better than to slowly and inexorably decline from this devastating disease, and this could be the biggest advance in therapy in years. Pridopidine could, for the first time, provide an option that may slow down decline in several functionally relevant disease domains and thus potentially offer patients and their families an extension to the quality time they have together. Since its safety profile appears favorable and it is taken orally, pridopidine can be easily made available to patients . ” “We have studied pridopidine extensively and have a clear understanding of its efficacy and safety. Pridopidine has delivered consistent efficacy benefits across multiple key measures of HD and has demonstrated a placebo-like safety profile in a large safety database,” said Dr. Michael R. Hayden, CEO of Prilenia . “We have submitted a compelling suite of evidence and pridopidine presents the opportunity for a much-needed paradigm shift in HD therapy.” “People with HD are acutely aware of their need for new and better treatment options, especially those that can slow down decline,” said Astri Arnesen, President of the European Huntington's Association . “Complex and rare diseases, like HD, are highly challenging and require forward-thinking, and we applaud the progress regulators are making in trying to support the urgent needs of people with HD. If we can come out of the other end of this process with a new therapeutic approach, offering the potential to slow down disease progression, we will have taken a huge step forward for the whole community.” MAA review typically takes 12-14 months but can take longer. In parallel, Prilenia continues its dialogue with the FDA on a potential path forward for pridopidine in the U.S. The Company will consider regulatory submissions for additional countries and regions following the regulatory review process in Europe. About Pridopidine Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability profile similar to placebo in clinical studies to date. The current safety dataset includes approximately 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, regulating several key processes that are commonly impaired in HD and other neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways, including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. About Huntington’s disease Huntington’s disease (HD) is a rare inherited (genetic) neurodegenerative disorder caused by a mutation in the huntingtin gene. Each child of a parent with HD has a 50 percent chance of developing the disease. HD causes neurons in the brain to degenerate and lose their ability to communicate with each other, resulting in functional, motor, cognitive and behavioral symptoms. HD affects about 100,000 people around the world with an additional 300,000 people at risk of developing HD. It is usually diagnosed between the ages of 30 and 50, although HD can also occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day to day life and take care of themselves. Currently, there is no treatment to address the progression of HD. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S. For more information, visit www.prilenia.com and follow us on LinkedIn or X (Twitter) . © 2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at www.prilenia.com .

Pridopidine is one of the most advanced investigational new drugs in HD and ALS and has shown consistent treatment benefits across independent measures important to patients and families -- Data to be presented at the American Academy of Neurology Annual Meeting 2024: HD: Oral presentation: The Impact of Antidopaminergic Medication on Longitudinal Clinical Progression in Huntington Disease -- Poster presentation: Analyses of the Phase 3 Trial of Pridopidine’s Outcome On Function in Huntington Disease (PROOF-HD) Demonstrate Efficacy in Participants Without Antidopaminergic Medication -- ALS: Oral presentation: Pridopidine For the Treatment of ALS – Results From the Phase 2 HEALEY ALS Platform Trial NAARDEN, Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)-- Prilenia Therapeutics B.V., a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, today announced the presentation of the latest research from the pridopidine Huntington disease (HD) and amyotrophic lateral sclerosis (ALS) programs at the American Academy of Neurology (AAN) Annual Congress, in Denver, Colorado, April 13-18. “Pridopidine is now in pre-registration phase in HD, with a first submission planned Mid 2024i, and is also set to commence a global Phase 3 study in ALS later this year. We are making significant strides forward and this is reflected by presentation of the data at one of the most important medical congresses in the field of Neurology,” said Jina Swarz, MD. Ph.D., the recently appointed Chief Medical Officer of Prilenia. “Pridopidine is one of the most advanced investigational new drugs in HD and ALS and has shown consistent treatment benefits across independent measures that are important to patients and families. The presented data advances knowledge in the field, providing important learnings for both clinical practice and for the design of our programs going forward. This advance is evident with novel findings from studies showing benefit with pridopidine in those with HD who are not taking anti-dopaminergic medication (ADMs) and a positive impact on speech and prolonged survival with pridopidine treatment in ALS.” Two oral and one poster presentations will be made at AAN 2024: Oral Presentations: Analyses of the Phase 3 Trial of Pridopidine’s Outcome On Function in Huntington Disease (PROOF-HD) Demonstrates Efficacy in Participants Without Antidopaminergic Medication Session name - S30: Movement Disorders: Clinical Trials in Movement Disorders; Program Number: S30.002. Presented by: Michael R Hayden, Founder and CEO, Prilenia Therapeutics; Wed, April 17, at 1:12 PM (MT) Pridopidine for the Treatment of ALS – Results from the Phase 2 HEALEY ALS Platform Trial Session name - S5: ALS and CMT: New Therapeutic Approaches; Program Number: S5.003. Presented by: Jeremy M Shefner, Professor, Department of Neurology, Barrow Neurological Institute; Sun, April 14, at 3:54 PM (MT) Poster Presentation: Analyses of the Phase 3 Trial of Pridopidine’s Outcome On Function in Huntington Disease (PROOF-HD) Demonstrate Efficacy in Participants Without Antidopaminergic Medication Session name - P6: Movement Disorders: Huntington's and Other Choreas 1; Program Number: P6.003. Presented by: Randal Hand, Director of Neuroscience, Prilenia Therapeutics; Tue, April 16, from 8:00-9:00a (MT) About Pridopidine Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability pro to placebo in clinical studies to date. The safety dataset includes over 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S. For more information, visit and follow us on LinkedIn and X (formerly known as Twitter). ©2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at . i First submission planned for the EU, with other regulatory submissions to follow

-- Expansion of leadership team aligned with planned first global regulatory submission for approval for pridopidine for Huntington’s disease and preparation for commercialization -- NAARDEN, Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)-- Prilenia Therapeutics B.V., a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, today announced the appointment of Dr. Jina Swartz, M.D. Ph.D., as its first Chief Medical Officer (CMO). In this role, Jina will lead all clinical development and medical affairs activities at Prilenia. This press release features multimedia. View the full release here: Jina Swartz, M.D. Ph.D., Chief Medical Officer at Prilenia (Photo: Business Wire) Jina has more than 30 years’ experience in both academic neurology practice and in global drug development at leading pharmaceutical companies and joins Prilenia from Exciva, a neurodegeneration-focused biotech, where she was Chief Medical Officer. Previously, Jina served as Therapeutic Area Head of Neuroscience in Global Clinical Drug Development at Merck and Co. Renowned within both research and industry circles, Jina is active in several important neurology and neurodegeneration associations including the European Brain Council and the European Federation of Neurological Associations. She also was recently elected to the Fellowship of the Academy of Medical Sciences for her expertise in neurodegenerative disorders and neuropsychiatric drug development. “Jina joins Prilenia during an important phase of the Company’s growth. We recently announced plans to submit pridopidine for regulatory review in Europe for Huntington’s disease (HD) and to commence a pivotal, global Phase 3 study in amyotrophic lateral sclerosis (ALS). Jina, in our newly created CMO role, will be a key leader as we advance these important initiatives,” said Dr. Michael R. Hayden, CEO of Prilenia. “Jina’s deep expertise as a thought leader, clinician treating neurodegenerative diseases and leader in global drug development is perfectly matched to our goal of changing the therapeutic landscape in HD and ALS.” “This is such an exciting time to be joining Prilenia and to help advance the Company’s mission to develop new therapeutic options for people living with neurodegenerative diseases like HD and ALS,” said Jina Swartz, M.D. Ph.D., Chief Medical Officer of Prilenia. “We have a remarkable opportunity, with pridopidine, to positively impact the lives of those living with these devastating diseases where progression has proven difficult to slow thus far. I believe that Prilenia is on the cusp of changing that paradigm.” Jina completed her Doctorate at the University of Cambridge, where her thesis focused on the exploration of molecular mechanisms of neurodegeneration in Huntington's disease and trinucleotide disorders. About Pridopidine Pridopidine (45 mg twice daily) is an oral, highly selective and potent investigational S1R agonist that has exhibited a safety and tolerability pro to placebo in clinical studies to date. The safety dataset includes approximately 1,700 patients who have been on pridopidine for up to seven years. The S1R protein is highly expressed in the brain and spinal cord, where it regulates several key processes that are commonly impaired in various neurodegenerative diseases. Activation of the S1R by pridopidine stimulates multiple cellular protective pathways including enhancing autophagy, axonal transport, mitochondrial energy production and respiration, and restores calcium homeostasis. These effects are essential to neuronal function and survival, and lead to neuroprotective effects. Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of HD. About Prilenia Prilenia is a clinical stage biotechnology company founded in 2018 focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders. The initial focus of the company has been on HD and ALS. Prilenia is backed by a group of well-respected investors including: Forbion, Morningside, Sands Capital, SV Health Investors, Sectoral Asset Management, Talisman, Amplitude Ventures and the ALS Investment Fund. The Company is based in the Netherlands, Israel and Massachusetts in the U.S. For more information, visit and follow us on LinkedIn and Twitter. ©2024 Prilenia Therapeutics B.V. For a copy of this release, visit Prilenia’s website at .