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Yahoo
3 days ago
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- Yahoo
Quince Therapeutics Exceeds 75% Enrollment in Pivotal Phase 3 NEAT Clinical Trial in Ataxia-Telangiectasia
SOUTH SAN FRANCISCO, Calif., June 03, 2025--(BUSINESS WIRE)--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, today announced that its pivotal Phase 3 NEAT (Neurological Effects of eDSP on Subjects with A-T; NCT06193200/IEDAT-04-2022) clinical trial in the rare neurodegenerative disease Ataxia-Telangiectasia (A-T) has reached an important milestone as enrollment now exceeds 75% with a total of 65 participants randomized in the study's targeted 86 participants in the six to nine year-old primary analysis population. Key Phase 3 NEAT clinical trial enrollment highlights to date include: A total of 76 participants have been enrolled, including 65 participants in the six to nine year-old primary analysis population and 11 participants aged 10 years or older. Study powering based on currently enrolled participants is approximately 80%. The rate of screening and randomization activities has materially increased with the recent activation of all study sites. A total of 108 NEAT participants have been screened to date with a low screen failure rate of 10%. All 37 NEAT participants to date have elected to transition to the NEAT open label extension (OLE) study (NCT06664853/IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study. Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2026. Quince was granted FDA Fast Track designation for the company's eDSP System for the treatment of patients with A-T based on the potential for eDSP to address a high unmet medical need. NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince's lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T. Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. About Ataxia-Telangiectasia A-T is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. There are currently no approved therapeutic treatments in any global market for A-T. About eDSP for A-T eDSP is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient's own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment. eDSP leverages Quince's proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient's own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince's AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. About Quince Therapeutics Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on the company and its latest news, visit and follow Quince on social media platforms LinkedIn, Facebook, X, and YouTube. Forward-looking Statements Statements in this news release contain "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the "safe harbor" created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as "believe," "may," "should," "expect," "anticipate," "plan," "believe," "estimated," "potential," "intend," "will," "can," "seek," or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including plans and the ability to enroll participants, impact of closing enrollment, conduct, and/or complete current and additional studies; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP; planned regulatory agency submissions, including NDAs, and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company's market opportunity. Forward-looking statements are based on Quince's current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled "Risk Factors" in the company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 13, 2025, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law. View source version on Contacts Media & Investor Contact: Stacy RoughanQuince Therapeutics, President, Corporate Communications & Investor Relationsir@ Sign in to access your portfolio
Business Wire
3 days ago
- Business
- Business Wire
Quince Therapeutics Exceeds 75% Enrollment in Pivotal Phase 3 NEAT Clinical Trial in Ataxia-Telangiectasia
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, today announced that its pivotal Phase 3 NEAT (N eurological E ffects of e DSP on Subjects with A - T; NCT06193200 / IEDAT-04-2022) clinical trial in the rare neurodegenerative disease Ataxia-Telangiectasia (A-T) has reached an important milestone as enrollment now exceeds 75% with a total of 65 participants randomized in the study's targeted 86 participants in the six to nine year-old primary analysis population. Key Phase 3 NEAT clinical trial enrollment highlights to date include: A total of 76 participants have been enrolled, including 65 participants in the six to nine year-old primary analysis population and 11 participants aged 10 years or older. Study powering based on currently enrolled participants is approximately 80%. The rate of screening and randomization activities has materially increased with the recent activation of all study sites. A total of 108 NEAT participants have been screened to date with a low screen failure rate of 10%. All 37 NEAT participants to date have elected to transition to the NEAT open label extension (OLE) study (NCT06664853 / IEDAT-04-2022). Participants who complete the full treatment period, complete study assessments, and provide informed consent are eligible to transition to the OLE study. Assuming positive study results, the company plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2026. Quince was granted FDA Fast Track designation for the company's eDSP System for the treatment of patients with A-T based on the potential for eDSP to address a high unmet medical need. NEAT is an international, multicenter, randomized, double-blind, placebo-controlled clinical trial to evaluate the neurological effects of Quince's lead asset, eDSP (dexamethasone sodium phosphate [DSP] encapsulated in autologous red blood cells; previously referred to as EryDex) in patients with A-T. Participants are randomized (1:1) between eDSP or placebo and treatment consists of six infusions scheduled once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last efficacy visit in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS) compared to placebo. About Ataxia-Telangiectasia A-T is an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in the ATM gene, which is responsible for cell homeostatic and cell division functions including but not limited to double-stranded DNA repair. Typically, A-T is first diagnosed before the age of five as children begin to develop an altered gait and fall with greater frequency. Neurological symptoms worsen and patients with A-T frequently become wheelchair-bound by adolescence. Teenage years for patients with A-T are typically marked by repeated infections, pulmonary impairment, and malignancies. The median lifespan is approximately 25 to 30 years old with mortality due to infections and malignancy. Based on IQVIA Medical Claims (Dx), PharmetricsPlus (P+), and IQVIA Analytics information, there are approximately 4,600 diagnosed patients with A-T in the U.S. Quince estimates that there are approximately 5,000 patients with A-T in the U.K. and EU4 countries. There are currently no approved therapeutic treatments in any global market for A-T. About eDSP for A-T eDSP is comprised of dexamethasone sodium phosphate (DSP) encapsulated in a patient's own red blood cells (autologous erythrocytes). DSP is a corticosteroid well known for its anti-inflammatory properties as well as its dose-limiting toxicity due to adrenal suppression. The eDSP System is designed to provide the efficacy of corticosteroids and to reduce or eliminate the significant adverse effects that accompany chronic use of corticosteroid treatment. eDSP leverages Quince's proprietary Autologous Intracellular Drug Encapsulation, or AIDE, technology platform, which is a novel drug/device combination that uses an automated process designed to encapsulate a drug into the patient's own red blood cells. Red blood cells have several characteristics that make them a potentially effective vehicle for drug delivery, including potentially better tolerability, enhanced tissue distribution, reduced immunogenicity, and prolongation of circulating half-life. Quince's AIDE technology is designed to harness these benefits to allow for the chronic administration of drugs that have limitations due to toxicity, poor biodistribution, suboptimal pharmacokinetics, or immune response. About Quince Therapeutics Quince Therapeutics, Inc. (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases. For more information on the company and its latest news, visit and follow Quince on social media platforms LinkedIn, Facebook, X, and YouTube. Forward-looking Statements Statements in this news release contain 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the 'safe harbor' created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as 'believe,' 'may,' 'should,' 'expect,' 'anticipate,' 'plan,' 'believe,' 'estimated,' 'potential,' 'intend,' 'will,' 'can,' 'seek,' or other similar words. Examples of forward-looking statements include, among others, statements relating to the timing, success, and reporting of results of the clinical trials and related data, including plans and the ability to enroll participants, impact of closing enrollment, conduct, and/or complete current and additional studies; current and future clinical development of eDSP, including for the potential treatment of Ataxia-Telangiectasia (A-T), Duchenne muscular dystrophy (DMD), and other potential indications; the strategic development path for eDSP; planned regulatory agency submissions, including NDAs, and clinical trials and timeline, prospects, and milestone expectations; and the potential benefits of eDSP and the company's market opportunity. Forward-looking statements are based on Quince's current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled 'Risk Factors' in the company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 13, 2025, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.
Yahoo
4 days ago
- Business
- Yahoo
Pharvaris Presents Data Supporting Ongoing Clinical Development of Deucrictibant in Bradykinin-Mediated Angioedema
Deucrictibant data shows single-dose durability without symptom reoccurrence in the majority of HAE attacks treated First-ever bradykinin B2 receptor mechanism-on-mechanism prophylactic/on-demand data supports potential for deucrictibant portfolio Clinically validated biomarker assay has potential to eventually expand treatment opportunities of deucrictibant into additional forms of bradykinin-mediated angioedema Epidemiologic data and cognitive interviews further elucidate the unmet needs in bradykinin-mediated angioedema ZUG, Switzerland, June 02, 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 14th C1-Inhibitor Deficiency and Angioedema Workshop. 'Additional analyses of deucrictibant data demonstrate consistency in the clinical profile shown in both the prophylactic and on-demand treatment settings,' said Berndt Modig, Chief Executive Officer of Pharvaris. 'Deucrictibant's early-onset and durable treatment response in the on-demand setting, the maintenance of attack reduction for over a year and a half in the prophylactic setting, and the potential for deucrictibant to be used together in both the prophylactic and on-demand settings, if needed, provide additional evidence of deucrictibant's potential in the treatment of bradykinin-mediated angioedema. Pharvaris continues to diligently execute on the deucrictibant clinical program and is planning for two pivotal data readouts in the next 18 months.' Details of the presentations are outlined below:ProphylaxisLong-Term Safety and Efficacy of Oral Deucrictibant for Prophylaxis in Hereditary Angioedema: Data Snapshot Results of the CHAPTER-1 Open-Label Extension Study, an oral presentation by Emel Aygören-Pürsün, M.D. 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. Peng Lu, M.D., Ph.D., Chief Medical Officer of Pharvaris, stated, 'Deucrictibant remains the only drug in development for bradykinin-mediated angioedema that has the potential to both prevent attacks and treat them when they occur. The data from the ongoing study further bolsters the potential value proposition of deucrictibant as it provides initial evidence that a bradykinin B2 receptor antagonist can effectively manage a breakthrough attack during treatment with a B2 receptor antagonist, if it were to occur. We believe further confirming these post-hoc open-label findings in our ongoing CHAPTER-3 study would provide additional evidence on the potential of deucrictibant to help address unmet needs of people living with bradykinin-mediated angioedema.' Long-Term Prophylactic Treatment with Oral Deucrictibant Improves Health-Related Quality of Life and Disease Control in Participants with Hereditary Angioedema: CHAPTER-1 Open-Label Extension Study, a poster presentation 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 at week 62 of the OLE. All of the participants in the OLE reported well controlled HAE and a high level of satisfaction with treatment. Sustained Therapeutic Exposure with Once-Daily Oral Deucrictibant XR Tablet for Prophylaxis of Hereditary Angioedema Attacks: Results of a Pharmacokinetics Study in Healthy Volunteers, a poster presentation by Zhi-Yi Zhang, Ph.D. To confirm its potential for once-daily prophylactic treatment, a Phase 1 pharmacokinetic study was conducted to compare the profile of the XR formulation (40 mg) to the immediate-release (IR) formulation (2 x 20 mg in a single administration), which was shown to be efficacious and well tolerated in the proof-of-concept CHAPTER-1 prophylaxis study. Deucrictibant extended-release (XR) tablet was well tolerated with no adverse events. Deucrictibant XR's pharmacokinetic profile demonstrated sustained exposure for over 24 hours, supporting once-daily dosing, and showed, on average, approximately a four-fold higher mean plasma concentration than therapeutic threshold (EC85) at 24 hours, supporting its further investigation as a potential oral once-daily prophylactic therapy for bradykinin-mediated angioedema. 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 poster presentation by Andrea Zanichelli, M.D., Ph.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) 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. On-DemandDurability Of Response to a Single Dose of Oral Deucrictibant for On-Demand Treatment of Hereditary Angioedema Attacks, a poster presentation by Anna Valerieva, M.D., Ph.D. A post-hoc analysis of the placebo-controlled RAPIDe-1 trial and the RAPIDe-2 extension study assessed the durability of effects in HAE attacks treated with a single dose of deucrictibant. In both studies, the majority of attacks were treated with a single dose of deucrictibant. In RAPIDe-1, 95-100% of the attacks and, in RAPIDe-2, 98-100% of the attacks that achieved symptom relief and resolution had a durable response without symptom reoccurrence. Dr. Lu continued, 'The ideal on-demand treatment for people living with HAE should offer both rapid symptom relief and complete symptom resolution with a single dose; this can only be achieved if the response to treatment is sustained without attack symptom reoccurrence. We believe the recently presented data on durability of response could be compelling to multiple stakeholders in the HAE community, including those living with HAE, their prescribing physicians, and the payor community. We aim to further evaluate deucrictibant's ability to rapidly and completely address bradykinin-mediated angioedema attack symptoms in our ongoing Phase 3 RAPIDe-3 clinical study.' Long-Term Safety and Efficacy of Oral Deucrictibant for Treatment of Hereditary Angioedema Attacks: Results of the RAPIDe-2 Extension Study, an oral presentation by Marc A. Riedl, M.D., M.S. 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. The median time to onset of symptom relief was 1.1 hours, and 97.8% of attacks achieved onset of symptom relief in 12 hours. The median time to complete attack resolution was 10.6 hours, and 86.9% of attack achieved complete resolution at 24 hours. Eighty-nine percent 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 poster presentation by Ramón Lleonart, M.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 compared to 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 Biomarker Assay to Characterize Bradykinin-Mediated Angioedema in Prospective and Biobank Plasma Samples, an oral presentation 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, resulting in a qualified kinin assay that can be used to reliably characterize people with bradykinin-mediated angioedema and could become a key tool aiding identification, study, and management of bradykinin-mediated pathologies including angioedema. Further applications of this biomarker assay will be explored during Pharvaris' R&D call on June 4 (register here). Acquired Angioedema Due to C1-Inhibitor Deficiency: Patient Experience and Assessment of Patient-Reported Outcome Measures, a poster presentation 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. Epidemiology of Bradykinin-Mediated Angioedema in the European Population, a poster presentation by Emel Aygören-Pürsün, M.D. A systematic literature review was conducted to summarize epidemiologic data on bradykinin-mediated angioedema, including HAE due to C1 inhibitor deficiency (HAE-C1INH Type 1 and Type 2), HAE due to other mutations in people with normal C1 levels and function (HAE-nC1INH), and AAE-C1INH, in the European Union (EU) and United Kingdom (UK). The review of 14 peer-reviewed scientific articles allowed to estimate the prevalence of HAE-C1INH (Type 1/2) as ranging between 0.05-0.33/10,000 individuals, the prevalence of HAE-nC1INH ranging between <0.01-0.07/10,000 individuals, and the prevalence of AAE-C1INH ranging between 0.01-0.02/10,000 individuals in European countries. The presentation slides and 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 successfully remediate the material weaknesses in our internal control over financial reporting and 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. 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Yahoo
7 days ago
- Business
- Yahoo
Roche's Multiple Sclerosis Drug Shows Low Disease Activity Over Two Years
Roche Holdings AG (OTC:RHHBY) on Friday announced new 96-week data for fenebrutinib demonstrating that patients with relapsing multiple sclerosis (RMS) maintained no disability progression and low disease activity levels for up to two years. The latest results for this investigational Bruton's tyrosine kinase (BTK) inhibitor from the Phase II FENopta open-label extension (OLE) study were presented at the Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting. Ninety-nine patients entered the OLE and 93 remained in the OLE after 96 the OLE period, patients treated with fenebrutinib for up to 96 weeks had a low annualised relapse rate (ARR) of 0.06, and during this time there was no disability progression, as measured by the Expanded Disability Status Scale (EDSS). MRI scans showed that fenebrutinib treatment suppressed disease activity in the brain. At 96 weeks zero new T1 gadolinium-enhancing (T1-Gd+) lesions, which are markers of active inflammation, were detected. In the treatment group that switched from placebo to fenebrutinib in the OLE, the annualized rate of new or enlarging T2 lesions, which represent chronic disease burden, decreased from 6.72 at the end of the 12-week double-blind period to 0.34 by 96 weeks. The safety profile of fenebrutinib in the OLE was consistent with previously reported data, with no new safety concerns identified at 96 weeks. During the OLE, one patient experienced asymptomatic alanine aminotransferase elevation at OLE week 4, after 16 weeks on treatment, which resolved with treatment discontinuation. Three Phase 3 trials are ongoing, including the FENhance 1 and 2 trials in RMS and the FENtrepid trial in primary progressive multiple sclerosis (PPMS). The first data from these studies are expected at the end of 2025. In April, Roche announced it would invest $50 billion in the U.S. over the next five years. These investments further strengthen Roche's already significant U.S. footprint, which includes 13 manufacturing and 15 R&D sites across the Pharmaceutical and Diagnostics Divisions. The investment is expected to create over 12,000 new jobs, including nearly 6,500 construction jobs and 1,000 jobs at new and expanded facilities. Price Action: RHHBY stock is trading higher by 1.83% to $40.25 at last check Friday. Read Next:Photo by OleksSH via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? This article Roche's Multiple Sclerosis Drug Shows Low Disease Activity Over Two Years originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved. Error 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
Yahoo
7 days ago
- Business
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Genentech's Fenebrutinib Maintains Near-Complete Suppression of Disease Activity and Disability Progression for up to Two Years in People With Relapsing Multiple Sclerosis
- Patients on fenebrutinib had low relapse rates with data showing no active brain lesions or disability progression after nearly two years of treatment - - Phase III studies for fenebrutinib in relapsing and primary progressive multiple sclerosis are expected to start reading out at year end - SOUTH SAN FRANCISCO, Calif., May 30, 2025--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new, 96-week data for fenebrutinib demonstrating that patients with relapsing multiple sclerosis (RMS) maintained no disability progression and low levels of disease activity for up to two years. The latest results for this investigational Bruton's tyrosine kinase (BTK) inhibitor from the Phase II FENopta open-label extension (OLE) study were presented at the Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting in Phoenix, Arizona. "These data show that patients treated with fenebrutinib experienced an annualized relapse rate equal to one relapse every 17 years and no observed disability progression up to two years,'' said Levi Garraway, M.D., Ph.D., Genentech's chief medical officer and head of Global Product Development. "Fenebrutinib is potent, highly selective and the only reversible BTK inhibitor currently in Phase III trials for multiple sclerosis, and we look forward to seeing the first of those results later this year." Ninety-nine patients entered the OLE and 93 remained in the OLE after 96 weeks. During the OLE period, patients treated with fenebrutinib for up to 96 weeks had a low annualized relapse rate (ARR) of 0.06, and during this time there was no disability progression, as measured by the Expanded Disability Status Scale (EDSS). MRI scans showed that fenebrutinib treatment suppressed disease activity in the brain. At 96 weeks zero new T1 gadolinium-enhancing (T1-Gd+) lesions, which are markers of active inflammation, were detected. In the treatment group that switched from placebo to fenebrutinib in the OLE, the annualized rate of new or enlarging T2 lesions, which represent chronic disease burden, decreased from 6.72 at the end of the 12 week double-blind period to 0.34 by 96 weeks. The safety profile of fenebrutinib in the OLE was consistent with previously reported data, with no new safety concerns identified at 96 weeks. The most common adverse events (AEs) in ≥5% of patients were COVID-19 (10%), urinary tract infection (10%), pharyngitis (6%) and respiratory tract infection (5%). Serious AEs occurred in two patients (2%). During the OLE, one patient experienced asymptomatic alanine aminotransferase elevation at OLE week 4, after 16 weeks on treatment, which resolved with treatment discontinuation. Three Phase III clinical trials are ongoing, including the FENhance 1 and 2 trials in RMS and the FENtrepid trial in primary progressive multiple sclerosis (PPMS). The first data from these studies, which will characterize the effects of fenebrutinib on disease progression across the multiple sclerosis spectrum, are expected at the end of 2025. About fenebrutinib Fenebrutinib is an investigational oral, reversible and non-covalent Bruton's tyrosine kinase (BTK) inhibitor that blocks the function of BTK. BTK, also known as tyrosine-protein kinase BTK, is an enzyme that regulates B-cell development and activation and is also involved in the activation of innate immune system myeloid lineage cells, such as macrophages and microglia. Preclinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible BTK inhibitor currently in Phase III trials for multiple sclerosis. Fenebrutinib has been shown to be 130 times more selective for BTK vs. other kinases. Fenebrutinib is a dual inhibitor of both B-cell and microglia activation. This dual inhibition may be able to reduce both multiple sclerosis disease activity and disability progression, thereby potentially addressing the key unmet medical need of disability progression in people living with multiple sclerosis. The fenebrutinib Phase III program includes two identical trials in relapsing multiple sclerosis (RMS) (FENhance 1 & 2) with active comparator teriflunomide and the only trial in primary progressive multiple sclerosis (PPMS) (FENtrepid) in which a BTK inhibitor is being evaluated against Ocrevus. To date, more than 2,700 patients and healthy volunteers have been treated with fenebrutinib in Phase I, II and III clinical programs across multiple diseases, including multiple sclerosis and other autoimmune disorders. About the FENopta study The FENopta study was a global Phase II, randomized, double-blind, placebo-controlled 12-week study to investigate the efficacy, safety and pharmacokinetics of fenebrutinib in 109 adults aged 18-55 years with relapsing multiple sclerosis (RMS). The primary endpoint was the total number of new T1 gadolinium-enhancing (T1-Gd+) lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks. Secondary endpoints included the number of new or enlarging T2-weighted lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks, and the proportion of patients free from any new T1-Gd+ lesions and new or enlarging T2-weighted lesions as measured by MRI scans of the brain at 4, 8 and 12 weeks. The goal of the FENopta study was to characterize the effect of fenebrutinib on MRI and soluble biomarkers of disease activity and progression, and it included an optional substudy to measure cerebrospinal fluid fenebrutinib levels and biomarkers of neuronal injury. Data from the 12-week study showed that fenebrutinib is central nervous system (CNS) penetrant (crosses the blood-brain barrier) and has the potential to impact mechanisms underlying chronic progressive disease biology in multiple sclerosis patients. Fenebrutinib significantly reduced new T1-Gd+ lesions and new/enlarging T2 lesions compared to placebo. The safety profile of fenebrutinib was consistent with previous and ongoing fenebrutinib clinical trials and there were no new safety concerns identified. Patients who completed the FENopta study were given the option to take part in an open-label extension (OLE) study, in which all patients receive fenebrutinib up to 192 weeks. About multiple sclerosis Multiple sclerosis (MS) is a chronic disease that affects more than 2.9 million people worldwide. MS occurs when the immune system abnormally attacks the insulation and support around nerve cells (myelin sheath) in the central nervous system (brain, spinal cord and optic nerves), causing inflammation and consequent damage. This damage can cause a wide range of symptoms, including weakness, fatigue and difficulty seeing, and may eventually lead to disability. Most people with MS experience their first symptom between 20 and 40 years of age, making the disease the leading cause of acquired non-traumatic disability in younger adults. People with all forms of MS experience disease progression – permanent loss of nerve cells in the central nervous system – from the beginning of their disease even if their symptoms aren't apparent or don't appear to be getting worse. Delays in diagnosis and treatment can negatively impact people with MS, in terms of their physical and mental health, and contribute to the negative financial impact on the individual and society. An important goal of treating MS is to slow, stop and ideally prevent progression as early as possible. Relapsing-remitting MS (RRMS) is the most common form of the disease and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. Approximately 85% of people with MS are initially diagnosed with RRMS. The majority of people who are diagnosed with RRMS will eventually transition to secondary progressive MS (SPMS), in which they experience steadily worsening disability over time. Relapsing forms of MS (RMS) include people with RRMS and people with SPMS who continue to experience relapses. Primary progressive MS (PPMS) is a debilitating form of the disease marked by steadily worsening symptoms but typically without distinct relapses or periods of remission. Approximately 15% of people with MS are diagnosed with the primary progressive form of the disease. Until the FDA approval of Ocrevus intravenous (IV) infusion, there had been no FDA-approved treatments for PPMS and Ocrevus IV and Ocrevus Zunovo are the only approved treatments for PPMS. About Genentech in neuroscience Neuroscience is a major focus of research and development at Genentech and Roche. 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, stroke, Alzheimer's disease, Huntington's disease, Parkinson's disease, Duchenne muscular dystrophy and autism spectrum disorder. 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 Access Solutions Access Solutions is part of Genentech's commitment to helping people access the Genentech medicines they are prescribed, regardless of their ability to pay. The team of in-house specialists at Access Solutions is dedicated to helping people navigate the access and reimbursement process and to providing assistance to eligible patients in the United States who are uninsured or cannot afford the out-of-pocket costs for their medicine. To date, the team has helped more than 2 million patients access the medicines they need. Please contact Access Solutions (866) 4ACCESS/(866) 422-2377 or visit for more information. 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 Indications and Important Safety Information What are Ocrevus and Ocrevus Zunovo? Ocrevus and Ocrevus Zunovo are prescription medicines used to treat: Relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults Primary progressive MS, in adults It is not known if Ocrevus and Ocrevus Zunovo are safe and effective in children. Who should not receive Ocrevus or Ocrevus Zunovo? Do not receive Ocrevus or Ocrevus Zunovo if you: have an active hepatitis B virus (HBV) infection. have had a life-threatening administration reaction to ocrelizumab. have had a life-threatening allergic reaction to ocrelizumab, hyaluronidase, or any of the ingredients of Ocrevus Zunovo. Tell your healthcare provider if you have had an allergic reaction to Ocrevus or Ocrevus Zunovo or any of their ingredients in the past. What is the most important information I should know about Ocrevus and Ocrevus Zunovo? Ocrevus and Ocrevus Zunovo can cause serious side effects, including: Infusion reactions (Ocrevus): Infusion reactions are a common side effect of Ocrevus, which can be serious and may require you to be hospitalized. You will be monitored during your infusion and for at least 1 hour after each infusion of Ocrevus for signs and symptoms of an infusion reaction. Injection reactions (Ocrevus Zunovo): Injection reactions are a common side effect of Ocrevus Zunovo, which can be serious and may require you to be hospitalized. You will be monitored for signs and symptoms of an injection reaction when you receive Ocrevus Zunovo. This will happen during all injections for at least 1 hour after your first injection, and for at least 15 minutes after all injections following the first injection. Tell your healthcare provider or nurse if you get any of these symptoms: itchy skin trouble breathing nausea shortness of breath rash throat irritation or pain headache fatigue hives feeling faint swelling of the throat fast heartbeat tiredness fever dizziness coughing or wheezing redness on your face (flushing) Additionally, for Ocrevus Zunovo: injection site pain swelling redness These infusion and injection reactions can happen during or up to 24 hours after administration. It is important that you call your healthcare provider right away if you get any of the signs or symptoms listed above after each infusion or injection. Infection: Infections are a common side effect. Ocrevus and Ocrevus Zunovo increase your risk of getting upper respiratory tract infections, lower respiratory tract infections, skin infections, and herpes infections. Serious infections can happen with Ocrevus and Ocrevus Zunovo, which can be life-threatening or cause death. Tell your healthcare provider if you have an infection or have any of the following signs of infection including fever, chills, or a cough that does not go away, or painful urination. Signs of herpes infection include: cold sores, shingles, genital sores, skin rash, pain, and itching. Signs of more serious herpes infection include: changes in vision, eye redness or eye pain, severe or persistent headache, stiff neck, and confusion. Signs of infection can happen during treatment or after you have received your last dose of Ocrevus or Ocrevus Zunovo. Tell your healthcare provider right away if you have an infection. Your healthcare provider should delay your treatment with Ocrevus or Ocrevus Zunovo until your infection is gone. Hepatitis B virus (HBV) reactivation: Before starting treatment with ocrelizumab, your healthcare provider will do blood tests to check for hepatitis B viral infection. If you have ever had hepatitis B virus infection, the hepatitis B virus may become active again during or after treatment with Ocrevus or Ocrevus Zunovo. Hepatitis B virus becoming active again (called reactivation) may cause serious liver problems including liver failure or death. Your healthcare provider will monitor you if you are at risk for hepatitis B virus reactivation during treatment and after you stop receiving Ocrevus or Ocrevus Zunovo. Weakened immune system: Ocrevus or Ocrevus Zunovo taken before or after other medicines that weaken the immune system could increase your risk of getting infections. Progressive Multifocal Leukoencephalopathy (PML): PML is a rare brain infection that usually leads to death or severe disability and has been reported with ocrelizumab. Symptoms of PML get worse over days to weeks. It is important that you call your healthcare provider right away if you have any new or worsening neurologic signs or symptoms that have lasted several days, including problems with: thinking eyesight strength balance weakness on 1 side of your body using your arms or legs Decreased immunoglobulins: Ocrevus and Ocrevus Zunovo may cause a decrease in some types of immunoglobulins. Your healthcare provider will do blood tests to check your blood immunoglobulin levels. Before receiving Ocrevus or Ocrevus Zunovo, tell your healthcare provider about all of your medical conditions, including if you: have or think you have an infection. See "What is the most important information I should know about Ocrevus and Ocrevus Zunovo?" have ever taken, take, or plan to take medicines that affect your immune system, or other treatments for MS. These medicines could increase your risk of getting an infection. have ever had hepatitis B or are a carrier of the hepatitis B virus. have a history of inflammatory bowel disease or colitis. have had a recent vaccination or are scheduled to receive any vaccinations. You should receive any required 'live' or 'live-attenuated' vaccines at least 4 weeks before you start treatment with Ocrevus or Ocrevus Zunovo. You should not receive 'live' or 'live-attenuated' vaccines while you are being treated with Ocrevus or Ocrevus Zunovo and until your healthcare provider tells you that your immune system is no longer weakened. When possible, you should receive any 'non-live' vaccines at least 2 weeks before you start treatment with Ocrevus or Ocrevus Zunovo. If you would like to receive any non-live (inactivated) vaccines, including the seasonal flu vaccine, while you are being treated with Ocrevus or Ocrevus Zunovo, talk to your healthcare provider. If you have a baby and you received Ocrevus or Ocrevus Zunovo during your pregnancy, it is important to tell your baby's healthcare provider about receiving Ocrevus or Ocrevus Zunovo so they can decide when your baby should be vaccinated. are pregnant, think that you might be pregnant, or plan to become pregnant. It is not known if Ocrevus and Ocrevus Zunovo will harm your unborn baby. You should use birth control (contraception) during treatment with Ocrevus and Ocrevus Zunovo and for 6 months after your last dose of Ocrevus or Ocrevus Zunovo. Talk with your healthcare provider about what birth control method is right for you during this time. Tell your healthcare provider if you become pregnant while receiving Ocrevus or Ocrevus Zunovo. are breastfeeding or plan to breastfeed. It is not known if Ocrevus and Ocrevus Zunovo pass into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take Ocrevus or Ocrevus Zunovo. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. What are the possible side effects of Ocrevus and Ocrevus Zunovo? Ocrevus and Ocrevus Zunovo may cause serious side effects, including: Risk of cancers (malignancies) including breast cancer: Follow your healthcare provider's instructions about standard screening guidelines for breast cancer. Inflammation of the colon, or colitis: Tell your healthcare provider if you have any symptoms of colitis, such as: Diarrhea (loose stools) or more frequent bowel movements than usual Stools that are black, tarry, sticky or have blood or mucus Severe stomach-area (abdomen) pain or tenderness The most common side effects of Ocrevus Zunovo include: Injection reactions Respiratory tract infections Skin infections These are not all the possible side effects of Ocrevus and Ocrevus Zunovo. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088. You may also report side effects to Genentech at (888) 835-2555. For more information, go to or call 1-844-627-3887. Please see additional Important Safety Information throughout and click here for full Prescribing Information and Medication Guide for Ocrevus. Please see additional Important Safety Information throughout and click here for full Prescribing Information and Medication Guide for Ocrevus Zunovo. View source version on Contacts Media Contact:Michelle McCourt(650) 467-6800Advocacy Contact:Lily Rose Atherton(202) 713-0083Investor Contacts:Loren Kalm(650) 225-3217Bruno Eschli+4161 6875284 Error 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
