Findings from Minzasolmin Proof-of-Concept ORCHESTRA Study Shape Next Steps in UCB Parkinson’s Research Program

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Brussels, Belgium – 16 December 2024 -- UCB today announced the ORCHESTRA proof-of-concept study of minzasolmin - an investigational, oral small molecule, alpha-synuclein misfolding inhibitor - developed in partnership with Novartis for early Parkinson’s disease, did not meet its primary and secondary clinical endpoints.

UCB has a portfolio of early phase pre-clinical and clinical programs evaluating multiple and distinct potential new treatment approaches in Parkinson’s disease. The misfolding and aggregation of alpha-synuclein is a key pathological driver of Parkinson’s disease2, therefore, in addition to minzasolmin, which focused on inhibiting intracellular alpha-synuclein misfolding, UCB is progressing UCB7583, currently under investigation for preventing extracellular alpha-synuclein spread. Going beyond alpha-synuclein pathology, UCB is progressing glovadalen (UCB0022), an orally available, brain-penetrant, small molecule, designed to enhance the potency of dopamine ‘when and where needed’ to activate the dopamine D1 receptor and thereby improve symptom control.3,4

Alistair Henry, Chief Scientific Officer, UCB commented: “Our team is grateful to the patients, families and healthcare professionals involved in the minzasolmin development program. With a strong and proven heritage in Parkinson’s disease, UCB continues its commitment to a science and patient driven strategy to address both its causes and symptoms. By leveraging the latest biological evidence, UCB’s strategy includes investigating mechanisms to inhibit the misfolding and propagation of alpha-synuclein - processes believed to underlie the spread of neurodegeneration. Alongside this, we are advancing research into innovative therapies for symptom control, recognizing the diverse needs of each patient throughout their disease trajectory.”

Following the evaluation of the ORCHESTRA study results, UCB has initiated measures to terminate the extension phase of this program. This decision is based on the absence of evidence of clinical benefit in both primary and secondary endpoints. Disease biomarker data, in which there was a preliminary signal, is still being analyzed.5 The safety profile of minzasolmin was consistent with previous knowledge and no new safety concern has been identified.5 The findings from this study have been submitted to an upcoming scientific meeting and will be submitted for publication in a peer-reviewed journal.

ORCHESTRA/PD0053 (NCT04658186) was a large Phase 2a, randomised, placebo-controlled, 18-month studyevaluating the efficacy, safety, tolerability and pharmacokinetics of oral minzasolmin in people living with early-stage Parkinson’s. The purpose of the study was to assess the safety and tolerability of minzasolmin and to demonstrate its superiority over placebo with regards to clinical symptoms of disease progression over 12 to 18 months in participants diagnosed with early-stage Parkinson’s disease. The primary endpoint was the progression in the Movement Disorder Society-Unified PD Rating Scale (MDS-UPDRS) Parts I–III sum score. More than 450 patients were enrolled in the study worldwide. The incidence of treatment emergent adverse events was comparable across all three treatment groups (180mg/day, 360mg/day, or placebo) in the study, and no new safety risks were identified.5

About UCBUCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will”, “continue” and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB’s efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB’s products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB’s data and systems.

Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future.

UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations.

Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction.

References:

  • https://www.ucb.com/innovation/clinical-studies/clinical-studies-index/UCB0599. Accessed December 2024.
  • Bendor JT, Logan TP, Edwards RH. The function of α-synuclein. Neuron. 2013 Sep 18;79(6):1044-66.
  • https://www.ucb.com/innovation/clinical-studies/clinical-studies-index/UCB7853. Accessed December 2024.
  • https://www.ucb.com/innovation/clinical-studies/clinical-studies-index/UCB0022. Accessed December 2024.
  • UCB Data on File. December 2024.

    • Source: UCB

    Posted : 2024-12-17 06:00

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