Huntington Disease

SAN DIEGO, April 30, 2025 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the initiation of a Phase 3 registrational program to evaluate the efficacy, safety and tolerability of NBI-1117568, the company's investigational oral muscarinic M4 selective orthosteric agonist, as a potential treatment for schizophrenia. Positive top-line data for the Phase 2 clinical study in adults with schizophrenia were reported in August 2024. "There is a significant need for new and innovative medicines to treat schizophrenia, a disorder that impacts millions of people and their families," said Eiry W. Roberts, M.D., Chief Medical Officer, Neurocrine Biosciences. "With positive Phase 2 data in hand, we're excited to advance this investigational novel compound that works directly and selectively at the muscarinic M4 receptor." The Phase 3 study is a global double-blind, placebo-controlled trial evaluating NBI-1117568 in adults with a primary diagnosis of schizophrenia who are experiencing an acute exacerbation or relapse of symptoms. The study is expected to enroll approximately 280 patients. The primary endpoint of the study is a reduction from baseline in the Positive and Negative Syndrome Scale (PANSS). The key secondary endpoint is improvement in the Clinical Global Impression of Severity (CGI-S) scale. Neurocrine is initiating the Phase 3 study supported by positive top-line data from the Phase 2 clinical study, which met its primary endpoint for the once-daily 20 mg dose. The study found: A clinically meaningful and statistically significant reduction from baseline in the PANSS total score at Week 6 with a placebo-adjusted mean reduction of 7.5 points (p=0.011 and effect size of 0.61) and an 18.2-point reduction from baseline. A statistically significant improvement across several secondary endpoints, including the CGI-S scale, Marder Factor Score – Positive Symptom Change, and Marder Factor Score – Negative Symptom Change. NBI-1117568 was generally safe and well tolerated at all doses studied, with minimal gastrointestinal and cardiovascular adverse events. About NBI-1117568 NBI-1117568 is the first and only investigational oral muscarinic M4 selective orthosteric agonist in clinical development for the treatment of schizophrenia. There are five muscarinic acetylcholine receptors involved in neurotransmission. Muscarinic receptors are central to brain function and validated as drug targets in psychosis and cognitive disorders. As an M4 selective orthosteric agonist, NBI-1117568 offers the potential for a novel mechanism with an improved safety profile without the need for combination therapy to minimize off-target pharmacology-related side effects, while also not being dependent on the presence of acetylcholine for efficacy. About Neurocrine Biosciences' Muscarinic Portfolio In addition to NBI-1117568, Neurocrine has a broad portfolio of assets in clinical development that selectively target muscarinic receptors. The company's muscarinic agonist portfolio also includes NBI-1117567, NBI-1117569, and NBI-1117570, which the company acquired the rights to develop and commercialize from Nxera Pharma. Neurocrine also is developing NBI-1076986, an investigational, selective M4 antagonist that was discovered and is being developed internally at Neurocrine. About Schizophrenia Schizophrenia is a serious and complex syndrome with heterogeneous symptoms. The World Health Organization estimates that the disorder impacts approximately 24 million people worldwide. Annual associated costs for schizophrenia are estimated to be more than $150 billion in the United States. As one of the leading causes of disability worldwide, it often results in significant emotional and functional burden for those who experience symptoms, as well as their family and friends. This chronic and disabling mental health condition is thought to result from a complex interplay of genetic and environmental risk factors. Traditional treatment approaches for schizophrenia rely on the use of antipsychotic medications that can lead to considerable short- and long-term health impacts. About Neurocrine Biosciences, Inc. Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, classic congenital adrenal hyperplasia, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X, and Facebook. (*in collaboration with AbbVie) NEUROCRINE, the NEUROCRINE BIOSCIENCES Logo, and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements regarding the clinical results from, and our future development plans with respect to, NBI-1117568, as well as the therapeutic potential and clinical benefits or safety profile of NBI-1117568. Factors that could cause actual results to differ materially from those stated or implied in the forward-looking statements include, but are not limited to, the following: top-line data that we report may change following a more comprehensive review of the data related to the clinical study and such data may not accurately reflect the complete results of the clinical study; risks that clinical development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing, or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that regulatory submissions for our product candidates may not occur or be submitted in a timely manner; our future financial and operating performance; risks associated with our dependence on third parties for development, manufacturing, and commercialization activities for our products and product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products or product candidates; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that our products, and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with U.S. federal or state legislative or regulatory and/or policy efforts which may result in, among other things, an adverse impact on our revenues or potential revenue; risks associated with potential generic entrants for our products; and other risks described in the Company's periodic reports filed with the Securities and Exchange Commission, including without limitation the Company's annual report on Form 10-K for the year ended December 31, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than required by law. © 2025 Neurocrine Biosciences, Inc. All Rights Reserved. SOURCE Neurocrine Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

The companies plan to commence a Phase III trial of the therapy for ALS. Credit: murat photographer/Shutterstock. Prilenia Therapeutics has signed a partnership and licence agreement with Ferrer to jointly develop and commercialise pridopidine in Europe and other chosen markets. The partnership could potentially bring a new treatment option to individuals with Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS). Prilenia will receive an upfront payment of almost €80m ($91m) and could earn up to €45m ($51.1m) in near-term development, commercial and regulatory milestones. The deal’s total value could reach €500m in upfront and milestone payments, with Prilenia also set to receive tiered double-digit royalties on net sales. Administered orally, pridopidine acts as a sigma-1 receptor (S1R) agonist, tailored to regulate neuroprotective mechanisms that are found impaired in ALS and HD. In the therapy’s HD development programme, it has shown benefits across various aspects of the disease, including cognition, function and motor skills, with these effects sustained for up to a two-year period. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Prilenia filed a marketing authorisation application (MAA) with the European Medicines Agency (EMA) for the approval of the therapy in treating HD. The EMA has accepted the application for review, and an opinion from the Committee for Medicinal Products for Human Use is anticipated in the second half of this year. Discussions are ongoing with the US Food and Drug Administration regarding the next steps for the therapy’s development in HD in the US. For ALS treatment, both companies are planning to commence a Phase III trial of the therapy, aiming to confirm outcomes from the Phase II HEALEY ALS platform trial. Prilenia Therapeutics CEO Dr Michael Hayden stated: “We are proud to partner with Ferrer as we advance our shared mission to bring transformative therapies to people living with neurodegenerative diseases around the world. “By combining our unique strengths and shared commitment to these patient communities, we believe that this partnership has the potential to accelerate the delivery of pridopidine to the thousands of people who are waiting for a new treatment option as well as broaden its impact through additional indications in the future.”

Prilenia Therapeutics can garner as much as about 500 million euros (around $568 million) in upfront and total milestone payments, plus royalties.\n Dutch biotech Prilenia Therapeutics has entered a licensing pact with Spanish pharmaceutical company Ferrer to push the biotech\'s sole asset through clinical development and beyond in Europe.Under the terms of the deal, Prilenia and Ferrer will jointly develop and potentially commercialize neurodegenerative disease candidate pridopidine in Europe and other undisclosed select markets, the companies said in an April 28 release. Prilenia will retain full rights to the oral sigma-1 receptor agonist in North America, Japan and the Asia-Pacific region.Ferrer is paying Prilenia 80 million euros upfront, with up to 45 million euros available in near-term development, regulatory and commercial milestones. In total, Prilenia can garner as much as 500 million euros (around $568 million) in upfront and milestone payments, according to the release, plus royalties on net sales.The focus of the partnership will be Huntington\'s disease and amyotrophic lateral sclerosis (ALS), with the partners planning to launch a phase 3 ALS trial in early 2026, a Ferrer spokesperson told Fierce Biotech in an email. Pridopidine previously failed to improve ALS disease severity in a phase 2 test, but the company said at the time that “positive trends” warranted further studies of the drug candidate. A post hoc analysis of the data found that some patients who were rapidly declining in speech, handwriting and other daily functions had “substantially less decline” when given pridopidine compared to those who received placebo, Prilenia said.In addition to Huntington\'s and ALS, Ferrer has first rights to develop pridopidine in other potential indications, the company\'s spokesperson said. Prilenia has already submitted a marketing authorization application to the European Medicines Agency (EMA) for the use of pridopidine in Huntington’s disease, according to the release, and the biotech is in discussions with the FDA about next steps for Huntington\'s in the U.S. A decision from the EMA\'s Committee for Medicinal Products for Human Use is expected in the second half of 2025, the Ferrer spokesperson said.“Ferrer continues to grow their already significant presence throughout Europe and key international markets with particular focus on innovative products for rare diseases,” Prilenia CEO Michael Hayden, Ph.D., said in the release. “We believe that this partnership has the potential to accelerate the delivery of pridopidine to the thousands of people who are waiting for a new treatment option as well as broaden its impact through additional indications in the future.”In addition to Huntington’s and ALS, Prilenia is also testing pridopidine in vanishing white matter disease, an inherited genetic disorder, and exploring the asset’s potential in other neurodegenerative diseases, according to the biotech’s website.Ferrer’s R&D work focuses on neurological and pulmonary diseases, and the Spanish pharma has bought into ALS assets in the past. In March 2024, the firm pledged more than $121.7 million in upfront and potential milestone payments for the ex-U.S. rights to Verge Genomics’ ALS candidate, which is currently in a phase 1b proof-of-concept trial.