A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Intravenous Prasinezumab in Participants With Early-Stage Parkinson's Disease

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of prasinezumab compared with placebo in participants with early-stage Parkinson's disease (PD) on stable symptomatic monotherapy with levodopa.

Start Date31 Dec 2025

Sponsor / Collaborator

Roche Holding AG

[+1]

NCT07055087

/ Not yet recruitingPhase 2IIT

A Randomized, Double-blind, Placebo-controlled, 104-week Proof-of-concept Study to Evaluate the Efficacy of Intravenous Prasinezumab in Participants With Parkinson's Disease Carrying a Severe Mutation in the GBA Gene

This is a proof-of-concept trial to investigate the efficacy of prasinezumab to slow or prevent cognitive decline in people with Parkinson's disease carrying a severe mutation in the GBA (glucocerebrosidase) gene. The duration of the intervention per patient will be 104 weeks with monthly infusions. The investigators plan to enroll 120 participants (60 participants per treatment arm). This study will be conducted across Europe in the following countries: France, Germany, Italy, Luxembourg, Spain, Sweden, UK.

Start Date01 Dec 2025

Sponsor / Collaborator-

NCT04777331

/ Active, not recruitingPhase 2

A Phase IIB, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Intravenous Prasinezumab in Participants With Early Parkinson's Disease

This is a multicenter, randomized, double-blind, placebo-controlled study that will evaluate the efficacy and safety of intravenous (IV) prasinezumab versus placebo in participants with Early Parkinson's Disease (PD) who are on stable symptomatic PD medication.

Start Date05 May 2021

Sponsor / Collaborator

Hoffmann-La Roche, Inc.

[+1]

100 Clinical Results associated with Prasinezumab

100 Translational Medicine associated with Prasinezumab

100 Patents (Medical) associated with Prasinezumab

Literatures (Medical) associated with Prasinezumab

01 Apr 2025·MOVEMENT DISORDERS

Prasinezumab: A Bayesian Perspective on Its Efficacy

Article

Author: Costa, Tommaso ; Russo, Mirella ; Calisi, Dario ; Sensi, Stefano L.

01 Mar 2025·PARKINSONISM & RELATED DISORDERS

A Phase 2b, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of intravenous prasinezumab in early-stage Parkinson's disease (PADOVA): Rationale, design, and baseline data

Article

Author: Kustermann, Thomas ; Rutten-Jacobs, Loes ; Respondek, Gesine ; Svoboda, Hanno ; Nikolcheva, Tania ; Postuma, Ronald B ; Shariati, Nima ; Pagano, Gennaro ; Seppi, Klaus ; Trundell, Dylan ; Monnet, Annabelle ; Simuni, Tanya ; Bonni, Azad ; Marek, Kenneth ; Doody, Rachelle ; Taylor, Kirsten I ; Stocchi, Fabrizio ; Pavese, Nicola ; Ricci, Benedicte ; Pross, Nathalie ; Fontoura, Paulo

INTRODUCTION:

Prasinezumab was shown to potentially delay motor progression in individuals with early-stage Parkinson's disease (PD) who were either treatment-naïve or on monoamine oxidase type B inhibitor (MAO-Bi) therapy in the PASADENA study. We report the rationale, design, and baseline patient characteristics of the PADOVA study, designed to evaluate prasinezumab in an early-stage PD population receiving standard-of-care (SOC) symptomatic medications.

METHODS:

PADOVA (NCT04777331) is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group study, in which individuals with early-stage PD on SOC stable symptomatic monotherapy (levodopa or MAO-Bi) receive intravenous prasinezumab 1500 mg every 4 weeks. The primary endpoint is time to confirmed motor progression, defined as ≥5 points increase from baseline on the Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III in practically defined OFF-medication state.

RESULTS:

586 participants were enrolled between May 5th, 2021 and March 22nd, 2023. At baseline, 74.2 % and 25.8 % of participants were receiving levodopa and MAO-Bi, respectively. Mean age was 64.2 years and 63.5 % were male. Mean time from diagnosis was 18.6 months, 85 % of participants were in Hoehn & Yahr (H&Y) Stage 2, and mean MDS-UPDRS Part III score was 24.5. Compared with the PASADENA population, PADOVA participants were older (∼5 years), with longer disease duration (∼8 months), and slightly more advanced based on H&Y stage (10 % more in Stage 2) and MDS-UPDRS Part III (∼3 points more).

CONCLUSIONS:

PADOVA has successfully recruited an early-stage PD population to test the effect of prasinezumab when added to background SOC.

01 Jan 2025·JOURNAL OF NEUROLOGY

An update on immune-based alpha-synuclein trials in Parkinson’s disease

Review

Author: Barker, Roger A ; Alfaidi, Maha ; Kuan, Wei-Li

Abstract:

Parkinson’s disease (PD) is a prevalent, chronic neurodegenerative disorder characterised by the progressive loss of dopaminergic neurons in the substantia nigra and other brain regions. The aggregation of alpha-synuclein (α-syn) into Lewy bodies and neurites is a key pathological feature associated with PD and its progression. Many therapeutic studies aim to target these aggregated forms of α-syn to potentially slow down or stop disease progression in PD. This review provides a comprehensive analysis of recent clinical trials involving vaccines and monoclonal antibodies targeting α-syn. Specifically, UB-312, AFFITOPE PD01A, PD03A and ACI-7104.056 are designed to provoke an immune response against α-syn (active immunisation), while Prasinezumab and Cinpanemab, MEDI1341 and Lu AF82422 focus on directly targeting α-syn aggregates (passive immunisation). Despite some promising results, challenges such as variable efficacy and trial discontinuations persist. Future research must address these challenges to advance disease-modifying therapies for PD around this therapeutic target.

News (Medical) associated with Prasinezumab

04 Aug 2025

·ir.prothena.com

Prothena Reports Second Quarter 2025 Financial Results and Business Highlights

Net cash used in operating and investing activities was $46.4 million in the second quarter and net cash used in operating and investing activities was $99.8 million for the first six months of 2025; quarter-end cash and restricted cash position was $372.3 million Prothena expects to convene an Extraordinary General Meeting by year-end 2025 to propose that shareholders approve a reduction of share capital to create distributable reserves in order to support a potential share redemption program if deemed appropriate Roche to advance prasinezumab, a potential first-in-class anti-alpha-synuclein antibody targeting a known biological driver of Parkinson’s disease progression, into Phase 3 development for early-stage Parkinson's disease by end of 2025 Initial data from Phase 1 ASCENT clinical trials evaluating PRX012, a potential single-injection once-monthly subcutaneous treatment for millions of patients with presymptomatic or early symptomatic Alzheimer’s disease, expected in August 2025 Potential to earn up to $105 million in aggregate clinical milestone payments in 2026 related to the advancement of coramitug for ATTR amyloidosis with cardiomyopathy by Novo Nordisk and PRX019 for neurodegenerative diseases by Bristol Myers Squibb DUBLIN--(BUSINESS WIRE)-- Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical biotechnology company with a robust pipeline of investigational therapeutics built on protein dysregulation expertise, today reported financial results for the second quarter and first six months of 2025 and provided business highlights. “We are excited that our partner Roche is advancing prasinezumab into Phase 3 development in early-stage Parkinson’s disease with initiation expected by the end of 2025. Prasinezumab could be the first disease-modifying treatment for a condition that affects 10 million people worldwide. Later this month we plan to share initial data from the Phase 1 ASCENT clinical trials of our wholly-owned PRX012 program in Alzheimer’s disease,” said Gene Kinney, Ph.D., President and Chief Executive Officer, Prothena. “We are also looking forward to data from Novo Nordisk’s Phase 2 clinical trial evaluating coramitug for ATTR amyloidosis with cardiomyopathy expected in the second half of 2025. Bristol Myers Squibb is conducting a Phase 2 TargetTau-1 clinical trial evaluating BMS-986446 in Alzheimer’s disease with completion expected in 2027 and a Phase 1 clinical trial evaluating BMS-986446 in a potential subcutaneous formulation with completion expected in the second half of 2025. In addition, we are conducting a Phase 1 clinical trial for PRX019 in collaboration with Bristol Myers Squibb with expected completion in 2026. Finally, there is a potential for us to earn up to $105 million in aggregate clinical milestone payments if Novo Nordisk advances coramitug and Bristol Myers Squibb decides to advance PRX019.” Second Quarter, Recent Business Highlights and Upcoming Milestones Neurodegenerative Diseases Portfolio Parkinson’s Disease Prasinezumab, a potential first-in-class antibody for the treatment of Parkinson’s disease that is designed to target a key epitope within the C-terminus of alpha-synuclein and is the focus of a worldwide collaboration with Roche. Partner Roche to advance prasinezumab into Phase 3 development for early-stage Parkinson's disease with initiation expected by the end of 2025 Prasinezumab is being investigated in ongoing open label extensions (OLEs) of the Phase 2 PASADENA and Phase 2b PADOVA clinical trials; both clinical trials are being conducted by our partner Roche Roche has stated that prasinezumab has peak sales potential greater than $3 billion (unadjusted) and could be the first disease-modifying treatment for a condition that affects 10 million people worldwide Alzheimer’s Disease PRX012, a wholly-owned potential best-in-class, single-injection once-monthly antibody delivered subcutaneously for the treatment of presymptomatic or early symptomatic Alzheimer’s disease that targets a key epitope at the N-terminus of amyloid beta (Aβ) with high binding potency. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for PRX012 for the treatment of Alzheimer’s disease. Prothena expects initial data in August from the Phase 1 ASCENT clinical trials Following the data from the Phase 1 ASCENT clinical trials, Prothena expects to advance PRX012 through non-dilutive and capital efficient structures BMS-986446 (formerly PRX005), a potential best-in-class antibody for the treatment of Alzheimer’s disease that specifically targets a key epitope within the microtubule binding region (MTBR) of tau, a protein implicated in the causal pathophysiology of Alzheimer’s disease. Bristol Myers Squibb is conducting the Phase 2 TargetTau-1 clinical trial in approximately 310 patients with early Alzheimer’s disease; primary completion expected in 2027 ( NCT06268886) Bristol Myers Squibb is also conducting a Phase 1 open-label single-dose clinical trial to assess a subcutaneous administration; primary completion expected in 2H 2025 ( NCT06955741) Bristol Myers Squibb is responsible for all communication, development, manufacturing, and commercialization PRX123, a wholly-owned potential first-in-class dual Aβ/tau vaccine designed for the treatment and prevention of Alzheimer’s disease, is a dual-target vaccine targeting key epitopes within the N-terminus of Aβ and MTBR-tau designed to promote amyloid clearance and block the transmission of pathogenic tau. The FDA cleared the investigational new drug (IND) application and granted Fast Track designation for PRX123 for the treatment of Alzheimer’s disease. Prothena expects to advance PRX123 through non-dilutive and capital efficient structures Neurodegenerative Diseases PRX019, a potential treatment of neurodegenerative diseases in development in collaboration with Bristol Myers Squibb. Bristol Myers Squibb obtained the exclusive global license for PRX019 in 2024 Prothena is conducting a Phase 1 first-in-human clinical trial to evaluate the safety, tolerability, immunogenicity, and pharmacokinetics of single ascending and multiple doses in healthy adults with completion expected in 2026 Potential to earn a clinical milestone in 2026 should Bristol Myers Squibb decide to further develop PRX019 Rare Peripheral Amyloid Disease ATTR Amyloidosis Coramitug (formerly PRX004), a potential first-in-class amyloid depleter antibody for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) designed to deplete the pathogenic, non-native forms of the transthyretin (TTR) protein, is being developed by Novo Nordisk as part of its up to $1.2 billion acquisition of Prothena’s ATTR amyloidosis business and pipeline. Phase 2 clinical trial in 105 patients has completed with results expected in 2H 2025 ( NCT05442047) Coramitug is being evaluated in an ongoing open label extension trial ( NCT06260709) for participants who completed the Phase 2 trial Potential to earn a clinical milestone in 2026 when prespecified enrollment criteria are met in a Phase 2b or Phase 3 clinical trial by Novo Nordisk Second Quarter and First Six Months of 2025 Financial Results For the second quarter and first six months of 2025, Prothena reported net loss of $125.8 million and $186.0 million, respectively, which includes restructuring charges of $32.6 million associated with the discontinuation of the birtamimab program and the reduction in workforce announced in June 2025, and a $44.9 million non-cash income tax expense to book a full valuation allowance against its federal deferred tax asset as compared to a net income of $66.9 million for the second quarter of 2024 and a net loss of $5.4 million for the first six months of 2024, respectively. Net loss per share was $2.34 and $3.45 for the second quarter and first six months of 2025 respectively, as compared to a net income per share on a diluted basis of $1.22 for the second quarter of 2024 and net loss per share of $0.10 the first six months of 2024. Prothena reported total revenue of $4.4 million and $7.2 million for the second quarter and first six months of 2025, respectively, as compared to total revenue of $132.0 million and $132.1 million for the second quarter and first six months of 2024. Total revenue for the second quarter and first six months of 2025 was primarily from collaboration revenue from Bristol Myers Squibb related to the partial performance of our PRX019 Phase 1 clinical trial obligation. Total revenue for the second quarter and first six months of 2024, was primarily from collaboration revenue from Bristol Myers Squibb. Research and development (R&D) expenses totaled $40.5 million and $91.3 million for the second quarter and first six months of 2025, respectively, as compared to $57.5 million and $121.6 million for the second quarter and first six months of 2024, respectively. The decrease in R&D expenses for the second quarter and first six months of 2025 compared to the same periods in the prior year was primarily due to lower clinical trial expenses, lower manufacturing and lower personnel expenses. R&D expenses included non-cash share-based compensation expense of $4.7 million and $9.5 million for the second quarter and first six months of 2025, respectively, as compared to $5.6 million and $11.1 million for the second quarter and first six months of 2024, respectively. General and administrative (G&A) expenses totaled $15.9 million and $33.5 million for the second quarter and first six months of 2025, respectively, as compared to $16.1 million and $33.6 million for the second quarter and first six months of 2024, respectively. G&A expenses included non-cash share-based compensation expense of $5.7 million and $11.8 million for the second quarter and first six months of 2025, respectively, as compared to $6.4 million and $13.3 million for the second quarter and first six months of 2024, respectively. Restructuring costs totaled $32.6 million for the second quarter and first six months of 2025, as compared to nil for the second quarter and first six months of 2024. Restructuring charges incurred for the second quarter and first six months of 2025 primarily consist of employee termination benefits in connection with the reduction in workforce announced in June 2025 and contract termination costs primarily associated with exit fees relating to third-party manufacturers that were contracted for commercial supplies of birtamimab. Employee termination benefits include severance costs, employee-related benefits, and non-cash share-based compensation expense related to the acceleration of stock options. Restructuring costs included non-cash share-based compensation expense of $2.1 million for the second quarter and first six months of 2025, as compared to nil for the second quarter and first six months of 2024. Total non-cash share-based compensation expense was $12.4 million and $23.4 million for the second quarter and first six months of 2025 respectively, as compared to $12.0 million and $24.4 million for the second quarter and first six months of 2024, respectively. As of June 30, 2025, Prothena had $372.3 million in cash, cash equivalents and restricted cash, and no debt. As of July 25, 2025, Prothena had approximately 53.8 million ordinary shares outstanding. 2025 Financial Guidance The Company continues to expect its full year net cash used in operating and investing activities to be $170 to $178 million and to end the year with approximately $298 million (midpoint) in cash, cash equivalents, and restricted cash. The estimated full year 2025 net cash used from operating and investing activities is primarily driven by an estimated net loss of $240 to $248 million, which includes an estimated $36 million of non-cash share-based compensation expense and a $44.9 million non-cash income tax expense to book a full valuation allowance against its federal deferred tax assets. Share Redemption Program Prothena expects to convene an Extraordinary General Meeting (“EGM”) of shareholders by year-end 2025 to vote on a proposal to approve a reduction in Prothena’s share capital to create distributable reserves, subject to confirmation by the Irish High Court, to support a potential share redemption program. The Board of Directors intends to seek shareholder approval of such a proposal to create flexibility to potentially return capital to shareholders via a share redemption program through open market purchases or other permissible means. Any such program would be subject to the discretion of the Board of Directors and Prothena’s then-current financial condition. About Prothena Prothena Corporation plc is a late-stage clinical biotechnology company with expertise in protein dysregulation and a pipeline of investigational therapeutics with the potential to change the course of devastating neurodegenerative and rare peripheral amyloid diseases. Fueled by its deep scientific expertise built over decades of research, Prothena is advancing a pipeline of therapeutic candidates for a number of indications and novel targets for which its ability to integrate scientific insights around neurological dysfunction and the biology of misfolded proteins can be leveraged. Prothena’s pipeline includes both wholly-owned and partnered programs being developed for the potential treatment of diseases including ATTR amyloidosis with cardiomyopathy, Alzheimer’s disease, Parkinson’s disease and a number of other neurodegenerative diseases. For more information, please visit the Company’s website at www.prothena.com and follow the Company on X (formerly Twitter) @ProthenaCorp. Forward-Looking Statements This press release contains forward-looking statements. These statements relate to, among other things, the sufficiency of our cash position to fund advancement of a broad pipeline and completion of our ongoing clinical trials; the continued advancement of our discovery, preclinical, and clinical pipeline, and expected milestones in 2025, 2026, 2027, and beyond; the treatment potential, designs, proposed mechanisms of action, and potential administration of PRX012, BMS-986446/PRX005, PRX123, prasinezumab, PRX019, and coramitug/PRX004; plans for ongoing and future clinical trials of PRX012, BMS-986446/PRX005, PRX123, prasinezumab, PRX019, and coramitug/PRX004, including Roche’s expected advancement of prasinezumab into Phase 3 development for early-stage Parkinson's disease with initiation expected by the end of 2025; the expected timing of reporting data from clinical trials, including initial data from our ongoing Phase 1 clinical trials evaluating PRX012 in August 2025; potential to advance our PRX012 and PRX123 programs through non-dilutive and capital efficient structures; timing of and amounts we may receive under our collaborations with Novo Nordisk and Bristol Myers Squibb; our anticipated net cash burn from operating and investing activities for 2025 and expected cash balance at the end of 2025; our expectation to convene an EGM by year-end 2025; the potential and ability to return capital to shareholders via a share redemption program or other permissible means if shareholders approve a reduction in share capital to create distributable reserves; and our estimated net loss and non-cash share-based compensation expense for 2025. These statements are based on estimates, projections and assumptions that may prove not to be accurate, and actual results could differ materially from those anticipated due to known and unknown risks, uncertainties and other factors, including but not limited to uncertainties related to the completion of operational and financial closing procedures, audit adjustments and other developments that may arise that would require adjustments to the preliminary financial results included in this press release, as well as those described in the “Risk Factors” sections of our Quarterly Report on Form 10-Q to be filed with the Securities and Exchange Commission (SEC) on August 4, 2025, and discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the SEC. We undertake no obligation to update publicly any forward-looking statements contained in this press release as a result of new information, future events, or changes in our expectations. PROTHENA CORPORATION PLC CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (unaudited - amounts in thousands except per share data) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Collaboration revenue 4,420 132,014 $ 7,198 $ 132,014 Revenue from license and intellectual property — — 50 50 Total revenue 4,420 132,014 7,248 132,064 Operating expenses: Research and development 40,517 57,510 91,328 121,624 General and administrative 15,910 16,127 33,508 33,591 Restructuring costs 32,609 — 32,609 — Total operating expenses 89,036 73,637 157,445 155,215 Income (loss) from operations (84,616 ) 58,377 (150,197 ) (23,151 ) Other income, net 3,651 6,470 7,859 13,558 Income (loss) before income taxes (80,965 ) 64,847 (142,338 ) (9,593 ) Provision for (benefit from) income taxes 44,802 (2,039 ) 43,624 (4,240 ) Net Income (loss) $ (125,767 ) $ 66,886 $ (185,962 ) $ (5,353 ) Basic net income (loss) per ordinary share $ (2.34 ) $ 1.24 $ (3.45 ) $ (0.10 ) Diluted net income (loss) per ordinary share $ (2.34 ) $ 1.22 $ (3.45 ) $ (0.10 ) Shares used to compute basic net income (loss) per share 53,827 53,767 53,827 53,740 Shares used to compute diluted net income (loss) per share 53,827 55,043 53,827 53,740 PROTHENA CORPORATION PLC CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited - amounts in thousands) June 30, December 31, 2025 2024 Assets Cash and cash equivalents $ 371,435 $ 471,388 Prepaid expenses and other current assets 14,040 14,024 Total current assets 385,475 485,412 Property and equipment, net 2,690 3,081 Operating lease right-of-use assets 9,563 10,708 Restricted cash, non-current 860 860 Other non-current assets 478 47,047 Total non-current assets 13,591 61,696 Total assets $ 399,066 $ 547,108 Liabilities and Shareholders’ Equity Accrued research and development 10,929 13,428 Deferred revenue, current 5,100 8,850 Restructuring liability 30,330 — Lease liability, current 2,853 2,610 Other current liabilities 18,595 23,613 Total current liabilities 67,807 48,501 Deferred revenue, non-current — 3,448 Lease liability, non-current 6,928 8,233 Total non-current liabilities 6,928 11,681 Total liabilities 74,735 60,182 Total shareholders’ equity 324,331 486,926 Total liabilities and shareholders’ equity $ 399,066 $ 547,108 Mark Johnson, CFA, Vice President, Investor Relations 650-417-1974, mark.johnson@prothena.com

Phase 1Phase 2VaccineFinancial StatementLicense out/in

28 Jul 2025

·www.prnewswire.com

Alpha-synuclein Inhibitors Market to Witness Strong CAGR Through 2034, Driven by Advances in Neurodegenerative Therapies | DelveInsight

No alpha-synuclein inhibitors are approved, underscoring a major need for therapies that slow or halt Parkinson's progression. Developing safe, brain-penetrant drugs that target toxic alpha-synuclein while preserving normal function is vital for early intervention and better outcomes. Late-stage candidates like Buntanetap (Annovis Biopharm), Prasinezumab (Roche/Prothena), and Amlenetug (Lundbeck) highlight growing innovation and competition in the alpha-synuclein inhibitors market. LAS VEGAS, July 28, 2025 /PRNewswire/ -- DelveInsight's Alpha-synuclein Inhibitors Market Size, Target Population, Competitive Landscape & Market Forecast report includes a comprehensive understanding of current treatment practices, addressable patient population, which includes top indications such as Parkinson's disease, Alzheimer's disease, Lewy body dementia, Multiple system atrophy, and others. The selected indications are based on approved therapies and ongoing pipeline activity. The report also provides insights into the emerging alpha-synuclein inhibitors, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM. Key Takeaways from the Alpha-synuclein Inhibitors Market Report As per DelveInsight's analysis, the total market size of alpha-synuclein inhibitors in the 7MM is expected to surge significantly by 2034. The report provides the total potential number of patients in the indications, such as Parkinson's disease, Alzheimer's disease, Lewy body dementia, Multiple system atrophy, and others. Leading alpha-synuclein inhibitors companies, such as Annovis Biopharm, Lundbeck, Roche, Prothena, and others, are developing novel alpha-synuclein inhibitors that can be available in the alpha-synuclein inhibitors market in the coming years. Some of the key alpha-synuclein inhibitors in the pipeline include Buntanetap, Amlenetug, Prasinezumab, and others. In March 2025, Annovis Bio participated in AD/PD 2025, scheduled from April 1–5 in Vienna. During the conference, the company Annovis gave two presentations, showcasing findings from its Phase III Parkinson's disease trial and data on buntanetap in ApoE4 carriers from its Phase II/III Alzheimer's disease study. In February 2025, the FDA granted Fast Track Designation (FTD) to amlenetug for the treatment of MSA. In January 2025, Annovis Bio secured a significant milestone with the issuance of a US patent protecting its novel methods for treating and preventing acute brain and nerve injuries through buntanetap. Discover which indication is expected to grab the major alpha-synuclein inhibitors market share @ Alpha-synuclein Inhibitors Market Report Alpha-synuclein Inhibitors Market Dynamics The alpha-synuclein inhibitors market is driven by the growing prevalence of neurodegenerative disorders such as Parkinson's disease, Lewy body dementia, and multiple system atrophy, where alpha-synuclein aggregation plays a critical pathogenic role. With the global burden of Parkinson's disease expected to double by 2040, there is a pressing demand for disease-modifying therapies that can go beyond symptomatic relief. Current treatment options, such as dopaminergic drugs, primarily address motor symptoms but fail to halt disease progression. This unmet medical need is pushing pharmaceutical and biotechnology companies to focus on alpha-synuclein inhibitors, which target the underlying pathology by preventing protein misfolding, aggregation, and the subsequent neurodegenerative cascade. Significant research and development investments are shaping the competitive landscape of the alpha-synuclein inhibitors market. Several leading pharmaceutical players are actively pursuing monoclonal antibodies and small molecule inhibitors targeting alpha-synuclein. Clinical trials have advanced the understanding of the therapeutic potential of targeting alpha-synuclein, though some setbacks have been observed due to limited clinical efficacy in late-stage trials. Nonetheless, advancements in biomarker discovery and imaging technologies for early diagnosis and monitoring disease progression are expected to enhance the success rate of ongoing and future clinical programs. Market dynamics are also influenced by regulatory and reimbursement factors. Given the high cost and complexity of neurodegenerative disease drug development, regulatory agencies like the FDA and EMA are encouraging accelerated pathways and breakthrough therapy designations for promising candidates. However, high development costs, combined with stringent clinical efficacy requirements, pose challenges for new entrants and smaller biotech firms. Moreover, reimbursement policies in major markets will play a critical role in determining the commercial success of these therapies once approved. Collaborations and strategic partnerships are increasingly shaping the alpha-synuclein inhibitors landscape. Big pharmaceutical companies are entering licensing agreements, mergers, or co-development partnerships with biotech firms to expand their neurodegenerative pipelines. For instance, Roche and Prothena's collaboration on prasinezumab reflects the industry trend of leveraging external innovation to accelerate drug development. Furthermore, increasing venture capital funding and public-private initiatives are supporting the growth of innovative startups working on novel alpha-synuclein-targeting approaches, such as antisense oligonucleotides and gene therapy-based strategies. Looking ahead, the alpha-synuclein inhibitors market is poised for steady growth. The next decade is expected to witness significant milestones in the approval and commercialization of disease-modifying therapies, which could revolutionize the treatment paradigm for Parkinson's and related disorders. Alpha-synuclein Inhibitors Treatment Market Alpha-synuclein inhibitors are therapeutic agents developed to prevent the aggregation of alpha-synuclein, a protein closely linked to neurodegenerative disorders such as Parkinson's disease, Lewy body dementia, and multiple system atrophy. These inhibitors function by blocking protein misfolding, reducing toxic buildup, and promoting clearance pathways. By targeting alpha-synuclein pathology, they aim to slow the progression of these diseases, offering promising advancements in neuroprotection and symptom relief. Although no alpha-synuclein inhibitors have yet received regulatory approval, the FDA has issued a 'Letter of Support' encouraging the use of the synuclein-based biomarker, αSyn-SAA, in clinical trials for Parkinson's disease and related disorders. This recognition underscores the biomarker's potential to improve disease monitoring, streamline drug development, and refine treatment strategies. With no approved therapies in this category, several companies, including Annovis Bio, Roche/Prothena, and Lundbeck, are actively progressing their pipelines to develop innovative candidates. These initiatives aim to enhance therapeutic approaches, improve drug effectiveness, and address the significant unmet needs in neurodegenerative conditions such as Parkinson's, Lewy body dementia, and MSA. Learn more about the alpha-synuclein inhibitors @ Alpha-synuclein Inhibitors Analysis Key Emerging Alpha-synuclein Inhibitors and Companies Emerging alpha-synuclein inhibitors in the pipeline, including buntanetap (Annovis Biopharm), prasinezumab (Roche/Prothena), amlenetug (Lundbeck), and others, are showing promise for expanding treatment options. Buntanetap, formerly known as Posiphen or ANVS401, works against neurodegeneration by preventing the build-up of toxic proteins such as amyloid beta, tau, alpha-synuclein, and TDP43. This action helps boost synaptic function, improve axonal transport, and reduce neuroinflammation—key processes required to maintain healthy neurons. The accumulation of these proteins disrupts neural pathways, leading to neuronal damage and cell death, which underlie diseases like Alzheimer's and Parkinson's. By targeting these mechanisms, buntanetap aims to reverse neurodegeneration, restore brain function, and significantly enhance patients' quality of life. In March 2025, Annovis Bio participated in the AD/PD 2025 conference (April 1–5, Vienna), where it delivered two presentations highlighting data from its Phase III Parkinson's disease trial and findings on buntanetap in ApoE4 carriers from its Phase II/III Alzheimer's disease study. Earlier, in July 2024, Annovis announced results from its Phase III Parkinson's disease trial (NCT05357989). Among patients diagnosed with Parkinson's for over three years, buntanetap demonstrated significant improvements in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) scores, Parts II, III, II+III, and Total, compared with placebo and baseline. Prasinezumab, an experimental monoclonal antibody, is designed to selectively bind aggregated alpha-synuclein (α-syn) and reduce its neurotoxic effects. By targeting α-syn accumulation and its cell-to-cell spread, prasinezumab has the potential to slow disease progression. Targeting α-syn aggregates is supported by extensive research as a viable therapeutic strategy for Parkinson's disease. At the AD/PD 2025 conference, the company presented results from the Phase IIb PADOVA study. While the drug showed potential in slowing motor symptom progression, it did not achieve statistical significance in the primary analysis, and further evaluations are ongoing. Amlenetug, a human monoclonal antibody, is designed to bind all major extracellular forms of α-syn, preventing their uptake and halting aggregation seeding. Its active Fc region may promote immune-mediated clearance of α-syn/antibody complexes through microglial uptake, thereby reducing pathological deposits. Developed by Lundbeck in collaboration with Genmab A/S, amlenetug is part of ongoing efforts to develop disease-modifying therapies for neurodegenerative conditions. In February 2025, the FDA granted Fast Track Designation (FTD) for amlenetug in treating multiple system atrophy (MSA). It previously received Orphan Drug Designation (ODD) from the FDA in April 2024, SAKIGAKE designation from Japan's Ministry of Health, Labour and Welfare in March 2023, and ODD from the European Medicines Agency (EMA) in May 2021. The anticipated launch of these emerging therapies are poised to transform the alpha-synuclein inhibitors market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the alpha-synuclein inhibitors market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about alpha-synuclein inhibitors clinical trials, visit @ Alpha-synuclein Inhibitors Treatment Alpha-synuclein Inhibitors Overview Alpha-synuclein is a highly soluble and intrinsically disordered protein that accumulates in Lewy bodies and Lewy neurites, playing a key role in the development of Parkinson's disease and other synucleinopathies. While predominantly located in the brain, smaller amounts are also present in the heart, muscles, and various tissues. Within neurons, α-Syn is concentrated in presynaptic terminals, where it regulates synaptic vesicle dynamics. Studies indicate that it may restrict vesicle mobility, thereby influencing synaptic recycling and neurotransmitter release. Another view suggests that α-Syn interacts with VAMP2 (synaptobrevin) to stabilize SNARE complexes, with recent research linking these interactions to synaptic function. Moreover, α-Syn may modulate dopamine release, affecting both voluntary and involuntary motor control. Synucleinopathies are marked by the accumulation of α-Syn amyloid deposits in the brain, driving neurodegeneration. Preventing α-Syn aggregation remains challenging due to its inherently disordered structure and the complex, unpredictable nature of fibrillogenesis. Nevertheless, targeting α-Syn is considered a promising therapeutic strategy, offering potential avenues for the treatment of Parkinson's disease and related conditions. Alpha-synuclein Inhibitors Epidemiology Segmentation The alpha-synuclein inhibitors market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Cases in Selected Indications for Alpha-synuclein Inhibitors Total Eligible Patient Pool in Selected Indications for Alpha-synuclein Inhibitors Total Treated Cases in Selected Indications for Alpha-synuclein Inhibitors Scope of the Alpha-synuclein Inhibitors Market Report Alpha-synuclein Inhibitors Therapeutic Assessment: Alpha-synuclein Inhibitors current marketed and emerging therapies Alpha-synuclein Inhibitors Market Dynamics: Conjoint Analysis of Emerging Alpha-synuclein Inhibitors Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Alpha-synuclein Inhibitors Market Access and Reimbursement Discover more about alpha-synuclein inhibitors in development @ Alpha-synuclein Inhibitors Clinical Trials Table of Contents Related Reports Parkinson's Disease Market Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease companies, including UCB Biopharma SRL, Novartis, Annovis Bio, Supernus Pharmaceuticals, Inc., Britannia Pharmaceutical, Pharma Two B, Mitsubishi Tanabe Pharma (NeuroDerm), AbbVie, Cerevel Therapeutics, LLC, among others. Alzheimer's Disease Market Alzheimer's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Alzheimer's disease companies including AB Science, Alzheon Inc., AriBio Co., Ltd., AgeneBio, Inc., Anavex Life Sciences Corp., Annovis Bio, Inc., Cerecin, BioVie, Cassava Sciences, Novo Nordisk, Eli Lilly, Neurim Pharmaceuticals, Suven Life Sciences, Bristol-Myers Squibb, Karuna Therapeutics, T3D Therapeutics, Inc., Lexeo Therapeutics, Axsome Therapeutics, Inc., Araclon Biotech S.L., Eisai Co., Ltd., TauRx Therapeutics, TrueBinding, Inc., AC Immune SA, Johnson & Johnson, Longeveron Inc., Vaccinex Inc., IGC Pharma LLC, among others. Multiple System Atrophy Market Multiple System Atrophy Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MSA companies including H Lundbeck A/S, Brain Neurotherapy Bio, Inc., Asklepios BioPharmaceutical, Inc., Theravance Biopharma, Ono Pharmaceutical Co. Ltd, Ionis Pharmaceuticals, Inc., Biogen, Alterity Therapeutics, Teva Pharmaceutical, MODAG GmbH, among others. Lewy Body Dementia Market Lewy Body Dementia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Lewy body dementia companies including EIP Pharma Inc., Cognition Therapeutics, Eisai Inc., Sun Pharma Advanced Research Company Limited, Georgetown University, Eli Lilly and Company, CuraSen Therapeutics, Inc., Athira Pharma, Aptinyx Inc., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultFast TrackPhase 3Orphan DrugPhase 2

25 Jul 2025

·www.fiercebiotech.com

\'We will crack gene therapy\': Elevidys fallout doesn\'t dent Roche\'s hopes for modality

Roche Pharma CEO Teresa Graham spoke to Fierce Biotech on the sidelines of the company\'s first-half earnings event.\n The uncertainty around the future of Elevidys hasn’t destroyed Roche’s belief in the potential of gene therapies, the Swiss company’s head of pharma has told Fierce Biotech, as the pharma continues to expand its research reach outside of its traditional oncology focus.Roche and its partner Sarepta Therapeutics both paused trials of Elevidys back in April following the death of a 16-year-old patient in the U.S. Since then, Roche—which markets the drug outside of the U.S.—has stopped all ex-U.S. distribution of the drug in the commercial setting for non-ambulatory patients, while the FDA requested that Sarepta halt Elevidys shipments in the U.S. Earlier this week, Roche paused shipments in all patients in countries where Elevidys’ approvals referenced the FDA.But Roche Pharma CEO Teresa Graham denied Fierce’s suggestion that the issues around Elevidys have altered how the company views gene therapy as a modality.“It doesn’t, actually,” Graham told Fierce in an interview at Roche’s headquarters in Basel, Switzerland on Thursday.“The reality is that gene therapy will be an important modality in the treatment of human health,” Graham continued. “There is a lot of promise in gene therapy. I think what the entire industry is learning is that, scientifically, it\'s just a little harder than we might have thought.” Even before the latest difficulties with Elevidys, Roche had announced a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. That overhaul closely followed Roche’s decision to end work on a hemophilia A gene therapy candidate, which was a focus of the Spark acquisition.Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved hemophilia B treatment Beqvez. Bluebird bio, a gene therapy pioneer once valued at $10 billion at its peak, was sold for just $49 million upfront to private equity firms.But Roche still sees potential in the modality, according to Graham.“I am firmly convicted that over time we will crack gene therapy and we will be able to deliver really meaningful treatments to patients,” she said.Graham spoke to Fierce on the sidelines of the company’s first-half earnings event, where Roche CEO Thomas Schinecker reminded journalists that 760 boys have successfully received Elevidys in the ambulatory setting.“I\'m really hopeful that we can find a path forward for this medicine,” Graham said in the interview on Thursday. “We clearly need to talk to regulators; we clearly need to have some good conversations about the safety—but we do firmly believe in the risk-benefit of this product.”Not all regulators sound ready to be convinced, with the European Medicines Agency (EMA) announcing Friday morning that it won\'t recommend Elevidys for approval in the ambulatory setting. Roche said in a statement that it “plans to continue to work with the EMA to explore a potential path forward.” Mixed results from emerging pipeline Another drug that Roche is keeping faith in is the Parkinson’s disease prospect prasinezumab. The future of the Prothena Biosciences–partnered asset looked in doubt after the anti-alpha-synuclein antibody failed a phase 2 study in December 2024.But Roche is giving prasinezumab a second chance, with plans to launch a phase 3 trial by the end of the year off the back of an analysis of data from the failed study. Specifically, the pharma pointed to longer-term follow-up data from an open-label portion of the study suggesting a clinical benefit on top of symptomatic treatment in early-stage Parkinson’s.“Given the dramatic unmet need in Parkinson\'s, it does feel like it would be a miss not to explore this,” Graham explained.“There\'s only really been minor improvements in treatments for these patients over the last 50 years, and, in every conversation I\'ve had with [key opinion leaders], they have really encouraged us to take this forward,” she said.“There\'s a little bit of a finger feel that there\'s something there, and we\'re only really ever going to be able to pull the signal out in a phase 3,” Graham added. Roche had less faith in CT-173, an early-stage obesity drug picked up as part of its $2.7 billion acquisition of Carmot Therapeutics in 2023. The Swiss pharma had previously touted the long-acting PYY analog’s potential to drive weight loss past the GLP-1 plateau, but Graham announced at the earnings event that Roche would no longer take CT-173 into human trials after assessing its “developability and competitiveness.”Despite this early-stage setback, Graham still feels that combinations offer Roche a path to carving out its own corner of the increasingly crowded obesity space.“Obesity is inherent in something like 200 other diseases,” she said. “So the ability to actually look at where we believe our portfolio in combination with other drugs can really help address some of these comorbidities is one of the more exciting pieces.”“This is an incredibly big market,” Graham added. “It is very diverse. There are a lot of scientific pathways to explore here.”One of those pathways is amylin, a hormone involved in the regulation of food intake. In March, Roche paid Zealand Pharma $1.6 billion for the long-acting amylin analog petrelintide. Graham sees the drug’s potential growing as the obesity market segments among patients “looking for maybe different depths of weight loss or different tolerability profiles.”Meanwhile, Roche has yet to make a final call on whether to take the GLP-1/GIP receptor agonist CT-388—another Carmot asset—into a phase 3 obesity trial, but Graham said the company is “right on the cusp” of taking that program forward.“We plan to make those decisions later this year, and, once we get rolling here, I think you\'re really going to see us flesh out our [obesity] strategy,” she said. When it comes to bringing new drugs into Roche’s pipeline, Graham was tight-lipped on which of the company’s five core focus areas—neurology, oncology/hematology, immunology, ophthalmology and CVRM (cardiovascular renal metabolic)—the company is prioritizing for M&A.“Clearly, within our five therapeutic areas, there are definitely places where we would like to bring in additional assets,” she told Fierce. “As Thomas [Schinecker] said, we look at an unbelievably large number of deals every year, and are constantly on the hunt, not only for things that will fit that strategy but things that are truly transformational.”With such a multifaceted pipeline, what are the readouts Graham is most excited about?“It\'s like trying to choose your favorite child,” she said, before name-checking the recent “super exciting” phase 2 results for the BTK inhibitor fenebrutinib in relapsing multiple sclerosis (MS). “It could be a really huge step forward for MS patients,” she explained.There’s also the “high-risk, high-reward bet” on the Parkinson’s drug prasinezumab, said Graham, who ended by referencing the IgG1 monoclonal antibody afimkibart.Along with CT-388 and the Alzheimer’s disease prospect trontinemab, afimkibart was one of the assets Roche fast-tracked as part of its reallocation of R&D resources.“I\'m an immunology girl at heart—it’s where I started my career,” Graham explained. “There\'s so much opportunity in the immunological space and so many diseases that we could potentially tackle with afimki.”

$\'We will crack gene therapy\': Elevidys fallout doesn\'t dent Roche\'s hopes for modality$

AcquisitionPhase 2Phase 3

100 Deals associated with Prasinezumab

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KEGG	Wiki	ATC	Drug Bank
D11420	-	-	DB14788

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Parkinson Disease	Phase 3	-	Roche Holding AG	31 Dec 2025

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04777331 (PADOVA, Company_Website) Manual	Phase 2	Parkinson Disease	586	Prasinezumab	nkksluqkfq(irxoiskhcs): HR = 0.84 (95% CI, 0.69 - 1.01), P-Value = 0.0657 Not Met	Negative	18 Dec 2024
				Placebo
NCT03100149 (PASADENA, AAN2023)	Phase 2	Parkinson Disease alpha-synuclein	316	Prasinezumab 1500 mg	ljhhnljdbd(gpqhoyenne) = fnmntwgxcr fzfgltagnv (hyrqsavsps ) View more	-	25 Apr 2023
				Placebo+Prasinezumab 1500 mg	ljhhnljdbd(gpqhoyenne) = wgnipefbtg fzfgltagnv (hyrqsavsps ) View more
NCT03100149 (PASADENA, MDS2022)	Phase 2	Parkinson Disease α-synuclein	-	Prasinezumab 1500 mg	bfrtoghqqd(bnocynchew) = guisawtbuc ozugegxsfs (cxjcqknzbf ) View more	-	15 Sep 2022
				Prasinezumab 4500 mg	bfrtoghqqd(bnocynchew) = bhqwrymozr ozugegxsfs (cxjcqknzbf ) View more
NCT03100149 (PASADENA, Pubmed \| N Engl J Med)	Phase 2	Parkinson Disease α-synuclein	316	Prasinezumab 1500 mg	myxjrzmzyc(srrjatyrfh) = infusion reactions occurred in 19.0% and 34.0%, respectively cupwgthmop (bgmhtnnwla ) View more	Negative	04 Aug 2022
				Prasinezumab 4500 mg
NCT03100149 (PASADENA, CTgov)	Phase 2	Parkinson Disease	316	Placebo (Part 1: Placebo)	xprmjucnoi(xrqfocxsbm) = tafdujsnpe xzfyglbvaw (yxlkvbrxdz, 1.221) View more	-	08 Feb 2021
				RO7046015 (Part 1: RO7046015 Low Dose)	xprmjucnoi(xrqfocxsbm) = viknyjlanf xzfyglbvaw (yxlkvbrxdz, 1.225) View more
NCT03100149 (PASADENA, Pubmed \| Front Neurol)	Phase 2	Parkinson Disease	316	Prasinezumab 1,500 mg	xihevtznyo(oepbiirkop) = ebsvdsgvsa oyibjhthuc (wstttamrog )	-	01 Jan 2021
				Prasinezumab 4,500 mg	xihevtznyo(oepbiirkop) = xegolfmbtz oyibjhthuc (wstttamrog )
NCT02157714 (Pubmed \| JAMA Neurol) Manual	Phase 1	Parkinson Disease	80	PRX002	mnsupnxlti(aqhlhmmdqq) = pfgkcgojqn tfksclkuhc (ckwzxhnldt ) View more	Positive	01 Oct 2018
				Placebo	mnsupnxlti(aqhlhmmdqq) = lozbctaccx tfksclkuhc (ckwzxhnldt )
EAN2017	Phase 1	Parkinson Disease alpha-synuclein	80	PRX002 0.3 mg/kg	lqkvdhovuu(covztzajxw) = TEAEs experienced by â¥5% of patients and >placebo were constipation glmpvyxnws (rdgwpozutj ) View more	Positive	13 Jul 2017
				PRX002 1 mg/kg

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