Latest news with #OLE
Business News Wales
4 days ago
- Automotive
- Business News Wales
Transformation Work Continues on Rhymney Valley Railway Line
Transport for Wales (TfW) says it has made significant progress on its intensive eight-month programme to transform and electrify the Rhymney Valley railway line. The second six-week railway line closure will start on Saturday 19 July, with no rail services running between Caerphilly and Rhymney until 31 August. Since construction on the upper Rhymney line began in March 2025, teams have upgraded over 15 kilometres of railway track. In June the last of the 693 planned foundations from Caerphilly to Rhymney was completed. These foundations will support the Overhead Line Equipment (OLE) that will power the new electric trains. To date, 466 steel posts have been installed to hold the overhead wires in place, which is approximately 55% of the total number of posts that will be installed along the line. The previous six-week closure of the upper Rhymney line, which took place in April, allowed teams to work more efficiently, TfW said, with more access to the railway line to carry out these essential upgrades. In late 2024, teams began ground investigations on the Rhymney line. Initially, they faced a refusal rate of 50% for the remaining 333 foundations, meaning that half of the foundations could not be completed in one go. This would have led to costly return visits and more disruptions for residents, TfW said. However, increased access to the line during railway closures allowed teams to conduct foundation probing. This process checks the level of bedrock, which helps teams change their approach or design for the Overhead Line Equipment (OLE) system. Adjusting piling methods to suit the bedrock conditions has resulted in the refusal rate dropping from 50% to just 0.3%, with only 10 refusals out of the 333 foundations. The extended closures on the line have also allowed most of the noisy piling work to take place during the daytime. To allow teams to progress with their work to install OLE and electrify the Upper Rhymney line, the line between Caerphilly and Rhymney will close from Saturday 19 July to Sunday 31 August. Rail replacement services will be in operation, and the line will re-open to accommodate the Catfish And The Bottlemen concert in the Principality Stadium on Friday 01 August. TfW said it encouraged all passengers traveling on the Rhymney line to check before they travel. More information on Rhymney line closures, rail replacement services and where to find local bus stops can be found on TfW's website: Rhymney line transformation | Transport for Wales
Business Wire
10-07-2025
- Health
- Business Wire
Stoke Therapeutics and Biogen Announce Presentation of Data from Studies of Zorevunersen, an Investigational Medicine for Dravet syndrome, at the 16th European Paediatric Neurology Society (EPNS) Congress
BEDFORD, Mass., & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB) today announced the presentation of data from an analysis that informed the design of the Phase 3 EMPEROR study and evaluated the potential effects of the Phase 3 zorevunersen dosing regimen. The data are complementary to previously reported data from a broader cohort of patients treated with zorevunersen in the Phase 1/2a and open label extension (OLE) studies that showed improvements within the first 9 months and continuing improvements through an additional two years. The new analysis is best aligned with the timing and dosing regimen that will be evaluated in the pivotal Phase 3 EMPEROR study and showed improvements in multiple measures of cognition and behavior at Week 68. The results contrasted with findings from a natural history study in which patients with Dravet syndrome were treated with standard of care medicines. Zorevunersen is in development as a first-in-class potential disease modifying treatment for Dravet syndrome. Results were presented as part of the Epilepsy II session at the 16th European Paediatric Neurology Society (EPNS) Congress. "Dravet syndrome is a complex neurodevelopmental disorder that has significant impacts on patients and their families,' said Dr. Andreas Brunklaus, Consultant Paediatric Neurologist at the Royal Hospital for Children in Glasgow, Honorary Professor at the University of Glasgow, and a zorevunersen study investigator. 'Natural history data shows the limitations of treating this disease with anti-seizure medicines. The zorevunersen data give us early evidence that this new genetically-targeted approach could address the underlying cause of Dravet syndrome, resulting in additional seizure control and offer patients the opportunity to experience improvements in cognition and behavior." Previously presented data from the two Phase 1/2a and OLE studies showed substantial and durable reductions in major motor seizure frequency on top of a background of standard anti-seizure medicines and improvements in multiple measures of cognition and behavior through two years of treatment in the OLE studies. Data indicated responses may be better among patients who were treated with loading doses of 70mg followed by maintenance doses of 45mg. Zorevunersen was generally well-tolerated across these studies. "Effects on behavior and cognition are a key secondary endpoint in our Phase 3 EMPEROR study," said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. 'Feedback from caregivers and clinicians, and analyses like this one, give us insight into which assessments have the greatest potential to demonstrate meaningful effects for patients within the year-long treatment period.' 'Most patients with Dravet syndrome continue to experience seizures despite treatment with the best available anti-seizure medicines, and there are currently no medications approved that address the underlying cognitive and behavioral aspects of the disease,' said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. 'We look forward to continuing to work together to advance zorevunersen as a potential first-in-class disease modifying medicine for Dravet syndrome.' Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) that is characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. There are no approved disease-modifying therapies for people living with Dravet syndrome. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the United States (~16,000), United Kingdom, EU-4 (Germany, France, Italy, Spain) and Japan. 1 Description of the Modeled Analysis A mixed-effects model for repeated measures (MMRM) analysis was used to evaluate the potential effects of the Phase 3 zorevunersen dosing regimen on patient cognition and behavior at Week 68. The model was developed using clinical data from patients in the Phase 1/2a ADMIRAL study and the LONGWING OLE study. An analysis of patients who received a total cumulative dose consistent with the Phase 3 EMPEROR regimen of two loading doses of 70mg followed by two maintenance doses of 45mg, showed improvements in cognition and behavior. The analysis was performed to inform the design of the Phase 3 EMPEROR study. Baseline covariates for patients followed in the BUTTERFLY natural history study were matched to the selected ADMIRAL patient population. Improvements in patients treated with zorevunersen contrasted with findings from BUTTERFLY. These data support the selection of five sub-domains of the Vineland-3 Adaptive Behavior Scales, including Receptive Communication, Expressive Communication, Interpersonal Relationships, Coping Skills, and Personal Skills now under evaluation as key secondary endpoints in the Phase 3 EMPEROR study. Details of the presentation are as follows: Title: Zorevunersen demonstrates potential as a disease modifying therapy in patients with Dravet syndrome through durable seizure reduction and improvements in cognition, behavior, and functioning with up to 24 months of maintenance dosing in open-label extension studies Presenter: Andreas Brunklaus, M.D., Consultant Paediatric Neurologist at the Royal Hospital for Children, Glasgow, Honorary Professor at the University of Glasgow Session: Epilepsy II Date/Time: Thursday, July 10, 11:51 AM CEST Location: Saal 5, International Congress Center München, Germany The presentation will be available for download on the Stoke Therapeutics website under the Investors & News tab. About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. 1 About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for Stoke's proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; and the design, timing and results of the Phase 3 EMPEROR study. Statements including words such as 'anticipate,' 'could,' 'expect,' 'plan,' 'will,' or 'may' and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stoke's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Stoke's collaborators were to breach or terminate their agreements, it would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading 'Risk Factors' in Stoke's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Biogen Safe Harbor This press release contains forward-looking statements, relating to: our strategy and plans; the potential of, and expectations for, our commercial business and pipeline programs; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, and efficacy of our and our collaboration partners' products and investigational therapies; and actions to improve the risk profile and productivity of R&D pipeline, collaborations, and business development activities. These forward-looking statements may be accompanied by such words as 'aim,' 'anticipate,' 'assume,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'guidance,' 'hope,' 'intend,' 'may,' 'objective,' 'outlook,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'prospect,' 'should,' 'target,' 'will,' 'would,' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including relating to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome, the design, timing and results of the Phase 3 EMPEROR study and the potential effects of the Phase 3 zorevunersen dosing regimen. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q , in each case including in the sections thereof captioned 'Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by .
Yahoo
16-06-2025
- Business
- Yahoo
Pharvaris Presents Data Highlighting the Potential for Deucrictibant to Prevent and Treat Bradykinin-Mediated Angioedema Attacks at the EAACI Congress
ZUG, Switzerland, June 16, 2025 (GLOBE NEWSWIRE) -- Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated diseases such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced a summary of data that were presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025. 'Pharvaris embraced the opportunity to engage in scientific exchange with the HAE thought leader community during EAACI as we presented data supporting the differentiated profile of deucrictibant for the prophylactic and on-demand treatment of bradykinin-mediated angioedema attacks,' said Berndt Modig, Chief Executive Officer of Pharvaris. 'Building on our R&D call from last week, we shared data demonstrating the potential for deucrictibant to address the unmet needs of people living with bradykinin-mediated angioedema beyond HAE-1/2. Deucrictibant showed sustained attack reduction and improved quality of life measures in the randomized portion of the CHAPTER-1 study, which was maintained in the open-label extension study, as well as early-onset symptom relief and complete symptom resolution in a single dose in most attacks in our ongoing RAPIDe-2 on-demand long-term extension study. Finally, RAPIDe-3 is the first and only phase 3 on-demand study that will explore 'end-of-progression' as a new pre-specified endpoint, which is particularly meaningful for people living with HAE. Together with the outcomes from other study endpoints, we will be able to assess the full impact of deucrictibant on an HAE attack from start to end.' Details of the presentations are outlined below:ProphylaxisLong-Term Safety and Efficacy of Oral Deucrictibant for Prophylaxis in Hereditary Angioedema: Results of the CHAPTER-1 Open-Label Extension Study, a poster presentation by Emel Aygören-Pürsün, M.D. First-ever bradykinin B2 receptor antagonism mechanism-on-mechanism prophylactic/on-demand data supports potential for deucrictibant portfolio. The ongoing Phase 2 CHAPTER-1 open-label extension (OLE) study provides further evidence on the long-term safety and efficacy of oral deucrictibant for prevention of HAE attacks. The attack rate has remained low, irrespective of baseline attack rate, for over a year and a half in OLE participants. When evaluating mechanism-on-mechanism responses, the response to icatibant for on-demand treatment of breakthrough attacks appeared to be maintained when used for breakthrough attacks during prophylactic treatment with deucrictibant. Long-Term Prophylactic Treatment with Oral Deucrictibant Improves Disease Control and Health-Related Quality of Life in Participants with Hereditary Angioedema in the CHAPTER-1 Open-Label Extension Study, a flash talk by Markus Magerl, M.D. The impact of deucrictibant treatment on health-related quality of life (HRQoL), disease control, and treatment satisfaction during the ongoing CHAPTER-1 OLE was evaluated. All of the participants who received deucrictibant reported clinically meaningful improvements in HRQoL at the end of the randomized portion of the trial, which was maintained up to the latest timepoint assessed at the time of data cutoff (week 62) of the OLE. All of the participants in the OLE reported well controlled HAE and a high level of satisfaction with treatment. CHAPTER-3 Phase 3 Trial Design: Efficacy and Safety of the Oral Bradykinin B2 Receptor Antagonist Deucrictibant Extended-Release Tablet for Prophylaxis of Hereditary Angioedema Attacks, a flash talk by William Lumry, M.D. CHAPTER-3 is an ongoing, global, Phase 3 study designed to evaluate the efficacy and safety of once-daily, oral deucrictibant (40 mg/day) extended release (XR) tablet for prophylaxis of attacks in adolescents and adults with HAE. Results from the Phase 2 CHAPTER-1 study support the CHAPTER-3 study design. Health-Related Quality of Life and Clinical Characteristics in People Living with Hereditary Angioedema Prescribed Long Term Prophylaxis Alone and On-Demand Treatment Alone, an oral presentation by Laurence Bouillet, M.D., Ph.D. A real-world cross-sectional survey was conducted to assess the relationship between treatment and outcomes of patients with HAE type 1/2 prescribed LTP or ODT alone in a real-world setting. 162 physicians reported data for 601 patients from Europe and the United States, collected via the Adelphi HAE Wave II Disease Specific Programme™ (DSP). Of the 601 patients, 41% were taking LTP, and 59% were taking ODT alone. Study results showed that patients with HAE prescribed LTP in the last 12 months experienced more mild attacks than moderate or severe attacks and had significantly better health related quality of life at the time of the survey compared with those prescribed ODT alone. Analysis suggests that both LTP and ODT play important roles in HAE management and corroborates international guidelines that recommend patients with HAE on LTP must always have ODT available at all times. On-DemandLong-Term Safety and Efficacy of Oral Deucrictibant for Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study, a thematic poster session by Henriette Farkas, M.D., Ph.D., Following the closure of Part A of RAPIDe-2, a Phase 2/3 study of deucrictibant for the on-demand treatment of HAE attacks, an analysis of 465 attacks from 19 participants, including 14 upper airway attacks from seven participants, was conducted. The final results from Part A of the RAPIDe-2 extension are consistent with the Phase 2 RAPIDe-1 randomized study. Deucrictibant continued to be well tolerated across all doses with no treatment-related treatment-emergent adverse events reported. The median time to onset of symptom relief was 1.1 hours, and 97.8% of attacks achieved onset of symptom relief by 12 hours. The median time to complete attack resolution was 10.6 hours, and 86.9% of attack achieved complete resolution at 24 hours. Deucrictibant data shows single-dose durability without symptom reoccurrence in most HAE attacks treated. 89.2% of the attacks that achieved symptom resolution at 24 hours were treated with a single dose of deucrictibant. Safety and Efficacy of Oral Deucrictibant for Treatment of Upper Airway and Laryngeal Hereditary Angioedema Attacks: Results from the RAPIDe-2 Extension Study, a flash talk by Anna Valerieva, M.D., Ph.D. The final data from Part A of the RAPIDe-2 study showed that safety and efficacy outcomes of treatment with deucrictibant IR were consistent for both HAE attacks affecting the upper airways, including laryngeal attacks, and HAE attacks occurring in other locations. Deucrictibant was generally well tolerated with no treatment-related treatment-emergent adverse events reported across upper airway and non-upper airway attacks. Fourteen upper airway attacks were treated by 7 participants; the median time to onset of symptom relief, as measured by Patient Global Impression of Change (PGI-C) of 'a little better', was 1.4 hours (n=14) for upper airway attacks and 1.1 hours for non-upper airway attacks (n=451). Endpoint measurements taken throughout the span of an entire attack until and including complete resolution were similar for both upper airway and non-upper airway attacks. Importantly, 92.9% of the upper airway attacks were treated with a single dose of deucrictibant. Expansion Beyond HAEClinical Validation of a Novel Kinin Biomarker Assay for Characterization of Bradykinin-Mediated Pathologies in U.S. Subjects with Hereditary Angioedema, a flash talk by Evangelia Pardali, Ph.D. Assays for an early and accurate diagnosis of bradykinin-mediated angioedema are lacking. Cold activation of plasma from people living with HAE resulted in increased levels of bradykinin compared to cold-activated plasma of healthy volunteers. The qualified kinin assay can be used to reliably characterize a bradykinin signature in people with recurrent angioedema and could become a key tool aiding identification, study, and management of bradykinin-mediated pathologies including bradykinin-mediated angioedema. As presented at the 14th C1-Inhibitor Deficiency and Angioedema Workshop, the performance of the assay does not depend on availability of 'fresh' plasma samples and the assay can also be applied in biobank samples for identification of people with bradykinin-mediated angioedema. Development of a Conceptual Model Supporting a Clinical Outcome Assessment Strategy for Acquired Angioedema due to C1 Inhibitor Deficiency, a thematic poster session by Andrea Zanichelli, M.D., Ph.D. There are currently no approved therapies for the treatment of AAE-C1INH attacks, nor patient-reported outcome measures validated in AAE-C1INH. Concept elicitation and cognitive interviews were performed to develop a conceptual model of AAE-C1INH that could reveal important disease concepts supporting a clinical outcome assessment strategy, as well as evaluating the comprehension and interpretation of PGI-C, PGI-Severity (PGI-S), patient global assessment of change (PGA-C), and PGA-Status (PGA-S), and explore perceptions of meaningful change using these measures. One hundred percent of participants considered PGI-C 'better' to be a meaningful change four hours post-treatment. Epidemiologic data and cognitive interviews further elucidate the unmet needs in bradykinin-mediated angioedema. The posters are available on the Investors section of the Pharvaris website at: About DeucrictibantDeucrictibant is a novel, potent, orally bioavailable small-molecule bradykinin B2 receptor antagonist currently in clinical development. Deucrictibant is being investigated for its potential to prevent the occurrence of bradykinin-mediated angioedema attacks and to treat the manifestations of attacks if/when they occur by inhibiting bradykinin signaling through the bradykinin B2 receptor. Pharvaris is developing two formulations of deucrictibant for oral administration: an extended-release tablet to enable sustained absorption and efficacy as prophylactic treatment, and an immediate-release capsule to enable rapid onset of activity for on-demand treatment. Deucrictibant has been granted orphan drug designation for the treatment of bradykinin-mediated angioedema by the U.S. Food and Drug Administration and orphan designation by the European Commission. About PharvarisPharvaris is a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to potentially address all types of bradykinin-mediated angioedema. Pharvaris intends to provide injectable-like efficacy™ and placebo-like tolerability with the convenience of oral therapies to prevent and treat bradykinin-mediated angioedema attacks. With positive data in both Phase 2 prophylaxis and on-demand studies in HAE, Pharvaris is currently evaluating the efficacy and safety of deucrictibant in a pivotal Phase 3 study for the prevention of HAE attacks (CHAPTER-3) and a pivotal Phase 3 study for the on-demand treatment of HAE attacks (RAPIDe-3). For more information, visit Forward Looking StatementsThis press release contains certain forward-looking statements that involve substantial risks and uncertainties. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements relating to our future plans, studies and trials, and any statements containing the words 'believe,' 'anticipate,' 'expect,' 'estimate,' 'may,' 'could,' 'should,' 'would,' 'will,' 'intend' and similar expressions. These forward-looking statements are based on management's current expectations, are neither promises nor guarantees, and involve known and unknown risks, uncertainties and other important factors that may cause Pharvaris' actual results, performance or achievements to be materially different from its expectations expressed or implied by the forward-looking statements. Such risks include but are not limited to the following: uncertainty in the outcome of our interactions with regulatory authorities, including the FDA; the expected timing, progress, or success of our clinical development programs, especially for deucrictibant immediate-release capsules and deucrictibant extended-release tablets, which are in late-stage global clinical trials; our ability to replicate the efficacy and safety demonstrated in the RAPIDe-1, RAPIDe-2, and CHAPTER-1 Phase 2 and Phase 3 studies in ongoing and future nonclinical studies and clinical trials; risks arising from epidemic diseases, which may adversely impact our business, nonclinical studies, and clinical trials; our ability to potentially use deucrictibant for alternative purposes, for example to treat C1-INH deficiency (AAE-C1INH); the outcome and timing of regulatory approvals; the value of our ordinary shares; the timing, costs and other limitations involved in obtaining regulatory approval for our product candidates, or any other product candidate that we may develop in the future; our ability to establish commercial capabilities or enter into agreements with third parties to market, sell, and distribute our product candidates; our ability to compete in the pharmaceutical industry, including with respect to existing therapies, emerging potentially competitive therapies and with competitive generic products; our ability to market, commercialize and achieve market acceptance for our product candidates; our ability to produce sufficient amounts of drug product candidates for commercialization; our ability to raise capital when needed and on acceptable terms; regulatory developments in the United States, the European Union and other jurisdictions; our ability to protect our intellectual property and know-how and operate our business without infringing the intellectual property rights or regulatory exclusivity of others; our ability to manage negative consequences from changes in applicable laws and regulations, including tax laws (including the Biosecure Act), our ability to maintain an effective system of internal control over financial reporting; changes and uncertainty in general market conditions; disruptions at the FDA and other agencies; political conditions, such as the current war between Russia and Ukraine; economic conditions, including continuing inflation concerns; and the other factors described under the headings 'Cautionary Statement Regarding Forward-Looking Statements' and 'Item 3. Key Information—D. Risk Factors' in our Annual Report on Form 20-F and other periodic filings with the U.S. Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. While Pharvaris may elect to update such forward-looking statements at some point in the future, Pharvaris disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing Pharvaris' views as of any date subsequent to the date of this press release. CONTACT: Contact Maggie Beller Executive Director, Head of Corporate and Investor Communications in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
16-06-2025
- Health
- Business Wire
Genentech to Advance Prasinezumab Into Phase III Development for Early-Stage Parkinson's Disease
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today its decision to proceed with Phase III development of prasinezumab, an investigational anti-alpha-synuclein antibody, in early-stage Parkinson's disease. This decision is informed by data from the Phase IIb PADOVA study and ongoing open-label extensions (OLEs) of PADOVA and Phase II PASADENA studies. "We are encouraged by the efficacy signals observed across the two Phase II trials and their open-label extensions, combined with the favorable safety and tolerability profile of prasinezumab," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "We also recognize the substantial need for new treatment options, and the totality of data suggest that prasinezumab may have the potential to become the first disease-modifying treatment for people with Parkinson's disease." Multiple endpoints from the PADOVA and OLE studies suggest a potential clinical benefit of prasinezumab when added to effective symptomatic treatment in early-stage Parkinson's disease. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression, although missed statistical significance. Positive trends towards reduced motor progression at 104 weeks (two years) were observed; these effects appear to be sustained over longer treatment periods based on additional OLE data. The PADOVA study also provided the first biomarker evidence of prasinezumab impacting the underlying disease biology. The PASADENA and PADOVA OLE studies, which are evaluating the long-term safety and efficacy of prasinezumab in over 750 people with early-stage Parkinson's disease, are ongoing. About prasinezumab Prasinezumab is an investigational monoclonal antibody designed to bind aggregated alpha-synuclein and thereby reduce neuronal toxicity. By reducing the build-up of alpha-synuclein protein in the brain, prasinezumab can potentially prevent further accumulation and spreading between cells, which may slow progression of the disease. Data from the Phase IIb PADOVA study suggest the possible clinical benefit of prasinezumab on top of effective symptomatic treatment in early-stage Parkinson's disease. PADOVA investigated prasinezumab in 586 people with early-stage Parkinson's disease, treated for a minimum of 18 months while on stable symptomatic treatment. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression with a HR=0.84 [0.69-1.01], although the study missed statistical significance (p=0.0657). In a pre-specified analysis, the effect of prasinezumab was more pronounced in the population treated with levodopa (75% of participants), HR=0.79 [0.63-0.99], p=0.0431 (nominal). Consistent positive trends across multiple secondary and exploratory endpoints were also observed. Trends towards reduced motor progression at 104 weeks (two years) were observed, showing 30-40% relative reduction versus placebo across the overall and levodopa-treated populations. Prasinezumab continues to be well tolerated and no new safety signals were observed in the study. The safety database for prasinezumab consists of data from more than 900 Parkinson's disease study participants that have been treated with the investigational medicine, of which more than 750 remain in open label treatment with over 500 treated for 1.5-5 years. Roche/Genentech entered into a Licensing, Development, and Commercialization agreement with Prothena in December 2013 to develop and commercialize monoclonal antibodies targeting aggregated alpha-synuclein, such as prasinezumab, for the treatment of Parkinson's disease. About Parkinson's disease Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disease characterized by the gradual loss of neurons that make dopamine and other nerve cells, and the development of motor and non-motor symptoms that may appear years before diagnosis. Today, Parkinson's disease affects over 10 million people worldwide. The prevalence of Parkinson's disease is increasing, and it has become one of the fastest-growing neurological disorders. Currently, symptomatic treatments that effectively alleviate motor symptoms are available today, having a significant impact on people's quality of life; however, no available symptomatic therapies slow down or stop the clinical progression of Parkinson's disease and the effects wear off over time as the disease progresses. Genentech and Roche are evaluating multiple approaches to slow down disease progression and potentially prevent Parkinson's disease that involve targeting underlying disease processes such as aggregated α-syn production, lysosomal dysfunction and neuroinflammation. About Genentech in Neuroscience Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer's disease, Huntington's disease, Parkinson's disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit
Business Wire
16-06-2025
- Business
- Business Wire
Prothena's Partner Roche to Advance Prasinezumab into Phase III Development for Early-Stage Parkinson's Disease
DUBLIN--(BUSINESS WIRE)--Prothena Corporation plc (NASDAQ:PRTA) today announced partner Roche will advance prasinezumab, an investigational anti-alpha-synuclein antibody, into Phase III development in early-stage Parkinson's disease. This decision is informed by data from the Phase IIb PADOVA study and ongoing open-label extensions (OLE) from both PADOVA and the Phase II PASADENA study. 'As pioneers in developing the first anti-alpha-synuclein targeting antibody, we are excited to see Roche advancing prasinezumab into Phase III development, with the potential to deliver the first disease-modifying treatment option to the millions of individuals living with Parkinson's disease and their families,' stated Gene Kinney, Ph.D., President and Chief Executive Officer, Prothena. Multiple endpoints from the PADOVA and OLE studies suggest a potential clinical benefit of prasinezumab when added to effective symptomatic treatment in early-stage Parkinson's disease. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression, although missed statistical significance. Positive trends towards reduced motor progression at 104 weeks (two years) were observed; these effects appear to be sustained over longer treatment periods based on additional OLE data. The PADOVA study also provided the first biomarker evidence of prasinezumab impacting the underlying disease biology. The PASADENA and PADOVA OLE studies, which are evaluating the long-term safety and efficacy of prasinezumab in over 750 people with early-stage Parkinson's disease, are ongoing. About Prasinezumab Prasinezumab is an investigational monoclonal antibody designed to bind aggregated alpha-synuclein and thereby reduce neuronal toxicity. By reducing the build-up of alpha-synuclein protein in the brain, prasinezumab can potentially prevent further accumulation and spreading between cells, which may slow progression of the disease. Data from the Phase IIb PADOVA study suggest the possible clinical benefit of prasinezumab on top of effective symptomatic treatment in early-stage Parkinson's disease. PADOVA investigated prasinezumab in 586 people with early-stage Parkinson's disease, treated for a minimum of 18 months while on stable symptomatic treatment. Prasinezumab showed potential clinical efficacy in the primary endpoint of time to confirmed motor progression with a HR=0.84 [0.69-1.01], although the study missed statistical significance (p=0.0657). In a pre-specified analysis, the effect of prasinezumab was more pronounced in the population treated with levodopa (75% of participants), HR=0.79 [0.63-0.99], p=0.0431 (nominal). Consistent positive trends across multiple secondary and exploratory endpoints were also observed. Trends towards reduced motor progression at 104 weeks (two years) were observed, showing 30-40% relative reduction versus placebo across the overall and levodopa-treated populations. Prasinezumab continues to be well tolerated and no new safety signals were observed in the study. The safety database for prasinezumab consists of data from more than 900 Parkinson's disease study participants that have been treated with the investigational medicine, of which more than 750 remain in open label treatment with over 500 treated for 1.5-5 years. In December 2013, Prothena and Roche entered into a worldwide collaboration to develop and commercialize antibodies that target alpha-synuclein, including prasinezumab. Roche has sole responsibility for developing and commercializing prasinezumab and has agreed to pay Prothena up to double-digit teen royalties on net sales. To date, Prothena has earned $135 million with up to $620 million in additional milestone payments that include regulatory and sales milestones. In addition, Prothena has an option to co-promote prasinezumab in the U.S. About Parkinson's disease Parkinson's disease is a chronic, progressive and debilitating neurodegenerative disease characterized by the gradual loss of neurons that make dopamine and other nerve cells. Today, Parkinson's disease affects over 10 million people worldwide. The prevalence of Parkinson's disease is increasing, and it has become one of the fastest-growing neurological disorders. Currently, symptomatic treatments that effectively alleviate motor symptoms are available. However, no therapies slow down or stop the clinical progression of Parkinson's disease. About Prothena Prothena Corporation plc is a clinical-stage 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 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 treatment potential, design, and proposed mechanism of action prasinezumab; plans for ongoing and future clinical trials of prasinezumab; and amounts we might receive under our collaboration with Roche. 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 those described in the 'Risk Factors' sections of our Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 8, 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.
