Latest news with #DELIVER
Yahoo
24-04-2025
- Business
- Yahoo
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy
- Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 - WALTHAM, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile (SV95C), an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe. 'Our recent long-term DELIVER trial results demonstrated clinically relevant and sustained functional improvement through 18 months, including as assessed by SV95C, which may support a strong rationale for regulatory approval in Europe,' said Doug Kerr, MD, PhD, chief medical officer of Dyne. 'We are pleased that the EC has granted orphan drug designation to DYNE-251, reinforcing our belief that our next-generation exon 51 skipping investigational therapy for DMD may be able to bring clinically meaningful functional improvement to those living with this devastating disease. With full enrollment of the registrational expansion cohort in the DELIVER trial complete, we look forward to sharing data from this cohort in late 2025 and the potential to move forward with our first regulatory submissions in early 2026.' The EC grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union (EU). Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and the potential for up to 10 years of market exclusivity in the EU if approved. DYNE-251 was also granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations in March 2023. Key Milestones for the DELIVER Trial Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping. Dyne has fully enrolled the registrational expansion cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025. Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026. About the DELIVER Trial DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit (NCT05524883) and (2023-510351-31-00). About DYNE-251 DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1), which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping. In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy (DMD) DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD, and currently approved therapies provide limited benefit. About Dyne Therapeutics Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. For more information, please visit and follow us on X, LinkedIn and Facebook. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne's strategy, future operations, prospects and plans, objectives of management, the therapeutic potential of DYNE-251, the anticipated timeline for reporting additional data from the DELIVER clinical trial, the availability of expedited approval pathways for DYNE-251, expectations regarding the timing and outcome of interactions with regulatory authorities, and expectations regarding the timing of submitting applications for U.S. Accelerated Approval and other regulatory approvals, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'objective,' 'ongoing,' 'plan,' 'predict,' 'project,' 'potential,' 'should,' or 'would,' or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne's ability to enroll patients in clinical trials; whether results from preclinical studies and data from clinical trials will be predictive of the final results of the clinical trials or other trials; whether data from clinical trials will support submission for regulatory approvals; uncertainties as to the FDA's and other regulatory authorities' interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and as to the regulatory approval process for Dyne's product candidates; whether Dyne's cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne's filings with the Securities and Exchange Commission (SEC), including the company's most recent Form 10-K and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne's views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne's views as of any date subsequent to the date of this press release. Contacts: InvestorsMia Tobiasir@ MediaStacy Nartkersnartker@ in to access your portfolio
Yahoo
24-04-2025
- Business
- Yahoo
Interim Report January-March 2025
GOTHENBURG, SE / / April 24, 2025 / XVIVO Perfusion AB (STO:XVIVO)(LSE:0RKL)(FRA:3XV) First quarter 2025 (Jan-Mar) Net sales increased to SEK 218.6 million (186.0), corresponding to growth of 18 percent in SEK and 16 percent in local currencies. Organic growth amounted to 14 percent in local currencies. The Business areas Thoracic and Abdominal delivered sales growth in local currencies: Thoracic 16 percent and Abdominal 28 percent. Services delivered negative growth of -6 percent. Total gross margin was 73 percent (73). The gross margin for the business areas amounted to: Thoracic 82 percent (81), Abdominal 63 percent (68) and Services 38 percent (36). Operating income (EBIT) increased to SEK 26.6 million (19.0). Adjusted EBIT increased by 50 percent to SEK 29.8 million (19.9). Operating income before depreciation and amortization (EBITDA) increased to SEK 43.0 million (36.0), corresponding to an EBITDA margin of 20 percent (19). Adjusted EBITDA increased to SEK 46.2 million (36.9), corresponding to an adjusted EBITDA margin of 21 percent (20). Net profit amounted to SEK -12.4 million (22.8), impacted by currency effects in cash and cash equivalents of SEK -22.5 million (8.3). Earnings per share amounted to SEK -0.39 (0.72). Cash flow from operating activities was SEK -15.3 million (1.6). The quarter was impacted by increased working capital tied up due to higher sales, as well as the annual payout of employee-related liabilities. Total cash flow amounted to SEK -77.2 million (-43.3), primarily impacted by investments in R&D projects of SEK -40.1 million. Significant events in the quarter Rapid FDA approval of the IDE application for the DELIVER study using Liver Assist FDA approval for continued use of XVIVO's heart technology through the PRESERVE CAP study For further information, please contact: Christoffer Rosenblad, CEO, +46 73 519 21 59, e-mail: Kristoffer Nordström, CFO, +46 73 519 21 64, e-mail: Conference Call Christoffer Rosenblad, CEO and Kristoffer Nordström, CFO, will present the report in a conference call today at 2.00 PM CET / 8.00 AM EST. For registration to the conference call, see information in previous press release: About Us Founded in 1998, XVIVO is the only medical technology company dedicated to extending the life of all major organs - so transplant teams around the world can save more lives. Our solutions allow leading clinicians and researchers to push the boundaries of transplantation medicine. XVIVO is headquartered in Gothenburg, Sweden, and has offices and research sites on two continents. The company is listed on Nasdaq Stockholm under the ticker symbol XVIVO. More information can be found on the website This information is information that XVIVO Perfusion AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact persons set out above, at 2025-04-24 07:30 CEST. Attachments XVIVO Interim Report Q1 2025 SOURCE: XVIVO Perfusion AB View the original press release on ACCESS Newswire Sign in to access your portfolio
Yahoo
10-04-2025
- Health
- Yahoo
AAN 2025: Lundbeck highlights sustained efficacy of Vyepti for migraine prevention
On 9 April, at the American Academy of Neurology (AAN) 2025 annual meeting, three posters highlighting the sustained long-term efficacy and real-world impact of Lundbeck's anti-calcitonin gene-related peptide (CGRP) monoclonal antibody (mAb) Vyepti (eptinezumab) for migraine prevention were presented. Vyepti was approved by the FDA for the prevention of migraine in adults in 2020. As the last of the anti-CGRP mAbs to enter the migraine market, Vyepti has seen moderate uptake since its approval, with the majority of key opinion leaders (KOLs) previously interviewed by GlobalData indicating that one of the subcutaneously administered anti-CGRP mAbs would be their first choice, highlighting the intravenous administration of Vyepti as a logistical challenge. The posters show that early response to Vyepti can be predictive of sustained treatment response, as well as highlighting meaningful benefits of Vyepti treatment. Post hoc analysis of the long-term, open-label Phase III PREVAIL (NCT02985398) study evaluating two-year safety and patient-reported outcomes with Vyepti in adults with chronic migraine was presented in a poster at AAN 2025. The analysis showed that the majority of the participants who achieved 50% or greater headache response or 75% or greater headache response in the first 12 weeks after receiving their first dose of Vyepti were able to maintain that level of response up to Week 84. Sustained treatment response was further demonstrated in another poster presenting post hoc analysis of data from the randomized, placebo-controlled Phase III DELIVER (NCT04418765) trial. In this study, patients had either chronic migraine or high-frequency episodic migraine, and they had failed to respond to two to four prior migraine preventive treatments. Up to 83% of patients who responded to Vyepti treatment within the first one to 24 weeks of the trial (i.e., having received the first two doses of Vyepti) were able to maintain that response over the 72-week trial. These analyses highlight that early treatment response with Vyepti may be able to predict long-term sustained treatment response. A third poster focused on patient-reported impact of Vyepti treatment in adults with chronic migraine. It presented a post hoc analysis of the impact of a 75% or greater increase in good days/month in patients treated with Vyepti from a real-world, observational, US-based study. Approximately two-thirds of the patients in the study reported a 75% or greater increase in good days/month, and this was correlated with higher satisfaction regarding the effects of Vyepti on migraine symptom severity, frequency, and duration; daily activities, such as being able to participate in social/family life, being productive, and ability to plan; and overall wellbeing when compared with patient with less than 75% increase in good days/month. Prior to treatment, the prevalence of brain fog was similar across the treatment response subgroups; however, following Vyepti treatment, 76.6% of the patients with a 75% or greater increase in good days/month reported moderate-to-complete improvement in brain fog, compared with 42.3% of patients with less than 75% increase in good days/month. These results highlight the meaningful benefits that patients experience following Vyepti treatment and will help Lundbeck with its focus on 'raising the bar around preventive treatment expectations in migraine.' Historically, migraine prevention has been managed with a wide variety of drug classes, such as beta-adrenergic receptor blockers, calcium antagonists, antidepressants, and anti-epileptics. However, many of these oral preventive treatments have been associated with poor side-effect profiles, and KOLs previously interviewed by GlobalData reported that low efficacy of oral preventive treatment was common among patients, meaning that patients often cycle through the different drug classes of oral preventive treatments. The first preventive therapies developed specifically for the treatment of migraine were the anti-CGRP mAbs, followed by the oral gepants; however, they are typically prescribed as second- or third-line options for many patients due to reimbursement restrictions requiring failure with the oral preventives before they can be prescribed. In the US, a 2024 statement was published by the American Headache Society recommending that the anti-CGRP mAbs and gepants should be used as first-line preventive options due to their superior efficacy and tolerability compared with the oral preventive options. Long-term sustained efficacy data and demonstration of meaningful benefit for patients, such as was presented for Vyepti at AAN 2025, are likely to further cement the case for first-line adoption of the anti-CGRP mAbs; however, given the comparative costs of the oral preventives and the mAbs, whether this recommendation will be implemented by insurance companies remains to be seen. Vyepti faces significant competition from three other anti-CGRP mAbs: Amgen's Aimovig (erenumab), Eli Lilly's Emgality (galcanezumab), and Teva's Ajovy (fremanezumab). Additional competition also comes from two orally administered gepants: Pfizer's Nurtec (rimegepant) and AbbVie's Qulipta (atogepant). KOLs agreed that the CGRP mAbs had similar efficacy profiles, with treatment choice often based on side effects, dosing schedule, or patient preference. As the only intravenously administered mAb, Vyepti may be at a disadvantage among patients who prefer to self-administer one of the other mAbs, and therefore highlighting the long-term efficacy, particularly with a focus on some of the patient-reported meaningful benefits, will be crucial in helping Vyepti to remain competitive in the migraine prevention market. "AAN 2025: Lundbeck highlights sustained efficacy of Vyepti for migraine prevention" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
17-03-2025
- Business
- Yahoo
Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months
- Continued favorable safety profile for DYNE-251 - - DELIVER Registrational Expansion Cohort is fully enrolled; data from this cohort planned for late 2025 - - Potential for Biologics License Application submission for U.S. accelerated approval in early 2026 - WALTHAM, Mass., March 16, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg/kg Q4W (approximate PMO dose). The DELIVER trial is evaluating DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, and updated results from the trial are being presented this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. 'With Dyne-251, we have the opportunity to deliver a durable and redosable therapy demonstrating clinically meaningful and sustained functional improvement in DMD. The consistency of these new data across multiple endpoints and timepoints underscores the potential of DYNE-251 to meaningfully address the significant unmet need in Duchenne despite available therapies,' said John Cox, president and chief executive officer of Dyne. 'We are rapidly advancing DYNE-251 toward a readout later this year with the potential to submit for U.S. accelerated approval in early 2026 based on a well-established regulatory pathway leveraging dystrophin expression as a surrogate endpoint. If approved, we believe there is an opportunity for rapid adoption by physicians and currently treated patients, as well as those naïve to therapy.' 'I am very encouraged by these new, long-term data for DYNE-251 in the exon 51 skip amenable population and the promise of sustained functional improvement which has continued to elude the DMD community,' said Pat Furlong, founder and president of Parent Project Muscular Dystrophy. 'The investment and innovation in DMD are delivering, and this is a prime example of how the accelerated approval pathway may swiftly enable a new generation of therapies that address unmet and urgent medical needs.' "The amount of expression of near-full length dystrophin induced by DYNE-251 has not been previously seen with exon 51 skipping agents and is associated with evidence for clinical efficacy,' said Kevin Flanigan M.D., Director, Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital. 'I look forward to the opportunity to present new functional data and updated safety data at the 2025 MDA Clinical & Scientific Conference.' This updated assessment of the DELIVER trial evaluating DYNE-251 includes new functional data out to 12 months from 6 patients enrolled in the 20 mg/kg Q4W cohort, and 18-month functional data from 6 patients in the 10 mg/kg Q4W cohort (these participants began transitioning to the 20 mg/kg Q4W regimen after month 6). In addition, updated safety data as of February 7, 2025, continue to demonstrate a favorable safety profile for DYNE-251. Key findings from the DELIVER Phase 1/2 trial presentation include: Function: Meaningful and sustained improvements from baseline in multiple functional endpoints were observed in both the 20 mg/kg (selected registrational dose) and 10 mg/kg1 DYNE-251 Q4W cohorts, through 12 and 18 months, respectively. Functional assessments included Stride Velocity 95th Centile (SV95C), North Star Ambulatory Assessment (NSAA), 10-Meter Walk/Run Time (10-MWR), and Time to Rise from Floor. Starting at the 6-month timepoint, the SV95C change from baseline observed in both the 10 mg/kg and 20 mg/kg cohorts of DELIVER exceeded the published proposed minimal clinically important difference (MCID). SV95C is a digital objective outcome measure of ambulatory performance in patients' normal daily environment and is accepted as a primary endpoint for DMD clinical trials in Europe. Dystrophin expression: As previously reported, DYNE-251 demonstrated unprecedented near-full length dystrophin expression as measured by Western blot for patients with DMD who are amenable to exon 51 skipping. At the 6-month time point, patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content). Dyne has confirmed that the U.S. Food and Drug Administration precedent for using dystrophin as a surrogate biomarker for accelerated approval remains available. Safety and tolerability: DYNE-251 has demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. Since the prior update provided as of November 21, 2024, the safety profile remains unchanged, and no new treatment-related serious adverse events have been observed.2 970 doses of study drug have been administered to date over a period of 77.1 patient-years of follow-up with some patients followed for up to ~2.5 years. 546 doses of study drug at the 20 mg/kg dose level have been administered to date.3 Key Milestones for the DELIVER Trial Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping. Dyne has fully enrolled the Registrational Expansion Cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025. Dyne anticipates a potential Biologics License Application (BLA) submission for U.S. accelerated approval in early 2026. Dyne Presentations at the 2025 MDA Clinical & Scientific ConferenceThe new assessment of the DELIVER trial is being presented in an oral presentation 'Safety and Efficacy from the Ongoing Phase 1/2 DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping' on Wednesday, March 19, at 8:30-8:45 a.m. CT by Kevin Flanigan, M.D., Director, Center for Gene Therapy, Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, Ohio and Principal Investigator for the DELIVER Trial. A poster with the same title will be available starting at 6:00 p.m. CT Sunday, March 16, 2025, through Tuesday, March 18, 2025, in the conference exhibit hall. Both the oral presentation and poster are now available in the Scientific Publications & Presentations section of Dyne's website, along with several other posters and presentations including an encore presentation of the most recent positive results for DYNE-101 from the Phase 1/2 ACHIEVE trial in myotonic dystrophy type 1 (DM1). About the DELIVER Trial DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit (NCT05524883) and (2023-510351-31-00). About DYNE-251 DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping. In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy (DMD) DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD, and currently approved therapies provide limited benefit. About Dyne Therapeutics Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. For more information, please visit and follow us on X, LinkedIn and Facebook. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne's strategy, future operations, prospects and plans, objectives of management, the potential of DYNE-251, the anticipated timelines for reporting additional data from the DELIVER clinical trial, including the registrational cohort, the availability of accelerated approval pathways for DYNE-251 and expectations regarding the timing of submitting applications for U.S. accelerated approval, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'objective,' 'ongoing,' 'plan,' 'predict,' 'project,' 'potential,' 'should,' or 'would,' or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne's ability to enroll patients in clinical trials; whether results from preclinical studies and data from clinical trials will be predictive of the final results of the clinical trials or other trials; whether data from clinical trials will support submission for regulatory approvals; uncertainties as to the FDA's and other regulatory authorities' interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and as to the regulatory approval process for Dyne's product candidates; whether Dyne's cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne's filings with the Securities and Exchange Commission (SEC), including the Company's most recent Form 10-K and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne's views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne's views as of any date subsequent to the date of this press release. During the OLE period, all participants in 10 mg/kg cohort were dose escalated to 20 mg/kg Q4W regimen. DYNE-251 safety data as of February 7, 2025. As of February 21, 2025. Contacts: InvestorsMia Tobiasir@ Nartkersnartker@ in to access your portfolio
Yahoo
16-03-2025
- Business
- Yahoo
Dyne Therapeutics Announces New Long-Term Clinical Data from Phase 1/2 DELIVER Trial of DYNE-251 in Duchenne Muscular Dystrophy Demonstrating Unprecedented and Sustained Functional Improvement Through 18 Months
- Continued favorable safety profile for DYNE-251 - - DELIVER Registrational Expansion Cohort is fully enrolled; data from this cohort planned for late 2025 - - Potential for Biologics License Application submission for U.S. accelerated approval in early 2026 - WALTHAM, Mass., March 16, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating unprecedented and sustained functional improvement at the selected registrational dose of 20 mg/kg Q4W (approximate PMO dose). The DELIVER trial is evaluating DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping, and updated results from the trial are being presented this week at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. 'With Dyne-251, we have the opportunity to deliver a durable and redosable therapy demonstrating clinically meaningful and sustained functional improvement in DMD. The consistency of these new data across multiple endpoints and timepoints underscores the potential of DYNE-251 to meaningfully address the significant unmet need in Duchenne despite available therapies,' said John Cox, president and chief executive officer of Dyne. 'We are rapidly advancing DYNE-251 toward a readout later this year with the potential to submit for U.S. accelerated approval in early 2026 based on a well-established regulatory pathway leveraging dystrophin expression as a surrogate endpoint. If approved, we believe there is an opportunity for rapid adoption by physicians and currently treated patients, as well as those naïve to therapy.' 'I am very encouraged by these new, long-term data for DYNE-251 in the exon 51 skip amenable population and the promise of sustained functional improvement which has continued to elude the DMD community,' said Pat Furlong, founder and president of Parent Project Muscular Dystrophy. 'The investment and innovation in DMD are delivering, and this is a prime example of how the accelerated approval pathway may swiftly enable a new generation of therapies that address unmet and urgent medical needs.' "The amount of expression of near-full length dystrophin induced by DYNE-251 has not been previously seen with exon 51 skipping agents and is associated with evidence for clinical efficacy,' said Kevin Flanigan M.D., Director, Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital. 'I look forward to the opportunity to present new functional data and updated safety data at the 2025 MDA Clinical & Scientific Conference.' This updated assessment of the DELIVER trial evaluating DYNE-251 includes new functional data out to 12 months from 6 patients enrolled in the 20 mg/kg Q4W cohort, and 18-month functional data from 6 patients in the 10 mg/kg Q4W cohort (these participants began transitioning to the 20 mg/kg Q4W regimen after month 6). In addition, updated safety data as of February 7, 2025, continue to demonstrate a favorable safety profile for DYNE-251. Key findings from the DELIVER Phase 1/2 trial presentation include: Function: Meaningful and sustained improvements from baseline in multiple functional endpoints were observed in both the 20 mg/kg (selected registrational dose) and 10 mg/kg1 DYNE-251 Q4W cohorts, through 12 and 18 months, respectively. Functional assessments included Stride Velocity 95th Centile (SV95C), North Star Ambulatory Assessment (NSAA), 10-Meter Walk/Run Time (10-MWR), and Time to Rise from Floor. Starting at the 6-month timepoint, the SV95C change from baseline observed in both the 10 mg/kg and 20 mg/kg cohorts of DELIVER exceeded the published proposed minimal clinically important difference (MCID). SV95C is a digital objective outcome measure of ambulatory performance in patients' normal daily environment and is accepted as a primary endpoint for DMD clinical trials in Europe. Dystrophin expression: As previously reported, DYNE-251 demonstrated unprecedented near-full length dystrophin expression as measured by Western blot for patients with DMD who are amenable to exon 51 skipping. At the 6-month time point, patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 8.72% of normal (adjusted for muscle content). Dyne has confirmed that the U.S. Food and Drug Administration precedent for using dystrophin as a surrogate biomarker for accelerated approval remains available. Safety and tolerability: DYNE-251 has demonstrated a favorable safety profile based on 54 participants enrolled in the DELIVER trial. Since the prior update provided as of November 21, 2024, the safety profile remains unchanged, and no new treatment-related serious adverse events have been observed.2 970 doses of study drug have been administered to date over a period of 77.1 patient-years of follow-up with some patients followed for up to ~2.5 years. 546 doses of study drug at the 20 mg/kg dose level have been administered to date.3 Key Milestones for the DELIVER Trial Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping. Dyne has fully enrolled the Registrational Expansion Cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025. Dyne anticipates a potential Biologics License Application (BLA) submission for U.S. accelerated approval in early 2026. Dyne Presentations at the 2025 MDA Clinical & Scientific ConferenceThe new assessment of the DELIVER trial is being presented in an oral presentation 'Safety and Efficacy from the Ongoing Phase 1/2 DELIVER Trial of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping' on Wednesday, March 19, at 8:30-8:45 a.m. CT by Kevin Flanigan, M.D., Director, Center for Gene Therapy, Abigail Wexner Research Institute of Nationwide Children's Hospital in Columbus, Ohio and Principal Investigator for the DELIVER Trial. A poster with the same title will be available starting at 6:00 p.m. CT Sunday, March 16, 2025, through Tuesday, March 18, 2025, in the conference exhibit hall. Both the oral presentation and poster are now available in the Scientific Publications & Presentations section of Dyne's website, along with several other posters and presentations including an encore presentation of the most recent positive results for DYNE-101 from the Phase 1/2 ACHIEVE trial in myotonic dystrophy type 1 (DM1). About the DELIVER Trial DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit (NCT05524883) and (2023-510351-31-00). About DYNE-251 DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping. In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. About Duchenne Muscular Dystrophy (DMD) DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD, and currently approved therapies provide limited benefit. About Dyne Therapeutics Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. For more information, please visit and follow us on X, LinkedIn and Facebook. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, including statements regarding Dyne's strategy, future operations, prospects and plans, objectives of management, the potential of DYNE-251, the anticipated timelines for reporting additional data from the DELIVER clinical trial, including the registrational cohort, the availability of accelerated approval pathways for DYNE-251 and expectations regarding the timing of submitting applications for U.S. accelerated approval, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. The words 'anticipate,' 'believe,' 'continue,' 'could,' 'estimate,' 'expect,' 'intend,' 'may,' 'might,' 'objective,' 'ongoing,' 'plan,' 'predict,' 'project,' 'potential,' 'should,' or 'would,' or the negative of these terms, or other comparable terminology are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Dyne may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the initiation and completion of preclinical studies and clinical trials; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; the timing of and Dyne's ability to enroll patients in clinical trials; whether results from preclinical studies and data from clinical trials will be predictive of the final results of the clinical trials or other trials; whether data from clinical trials will support submission for regulatory approvals; uncertainties as to the FDA's and other regulatory authorities' interpretation of the data from Dyne's clinical trials and acceptance of Dyne's clinical programs and as to the regulatory approval process for Dyne's product candidates; whether Dyne's cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; as well as the risks and uncertainties identified in Dyne's filings with the Securities and Exchange Commission (SEC), including the Company's most recent Form 10-K and in subsequent filings Dyne may make with the SEC. In addition, the forward-looking statements included in this press release represent Dyne's views as of the date of this press release. Dyne anticipates that subsequent events and developments will cause its views to change. However, while Dyne may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Dyne's views as of any date subsequent to the date of this press release. During the OLE period, all participants in 10 mg/kg cohort were dose escalated to 20 mg/kg Q4W regimen. DYNE-251 safety data as of February 7, 2025. As of February 21, 2025. Contacts: InvestorsMia Tobiasir@ Nartkersnartker@ in to access your portfolio
