Jon
LightNews LightNews
banner
jonu235.bsky.social
Jon
@jonu235.bsky.social
43 followers 150 following 27 posts
$SRPT #Duchenne #Elevidys #GeneTherapy data which STATnews, Fierce Biotech, BiopharmaDIVE, and BioCentury REFUSE to report! https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-results-part-2-embark-study
Posts Replies Media Videos
jonu235.bsky.social
"Statistically significant differences were observed on all key endpoints; Significant functional benefits for 8 and 9-year-olds with Duchenne in Part 2 of the EMBARK study" #Elevidys delandistrogene moxeparvovec #Duchenne muscular dystrophy #GeneTherapy investorrelations.sarepta.com/news-release...
Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2 05/16/25 9:00 AM EDT Significant functional benefits for 8- and 9-year-olds with Duchenne in Part 2 of the EMBARK study, contributing to the evidence of stabilization or slowing of disease progression in later childhood when muscle weakness typically progresses Statistically significant differences were observed on all key endpoints including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort In the recent analysis of Part 2 of the EMBARK study, participants with Duchenne muscular dystrophy who had received a placebo in Part 1 and were aged 8 to 9 years (n=14) at crossover were included. At one year post ELEVIDYS treatment, there were between-group differences (least square means) on all key endpoints that were statistically significant, including 4.75 points (P=0.0026) on North Star Ambulatory Assessment (NSAA), 6.87 seconds in time-to-rise (TTR) from the floor (P=0.0010), and 4.76 seconds in 10-meter walk/run (10MWR) (P=0.0097) compared to a well-matched external control cohort. "The latest data from the EMBARK study highlighting motor function improvements in 8- and 9-year-old boys is encouraging and adds to the growing body of evidence supporting ELEVIDYS," said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics at the University of Arkansas for Medical Sciences and Arkansas Children’s Hospital. "What stands out is that these patients were treated at an age when motor decline is typically expected in those with Duchenne. Yet, those who received ELEVIDYS demonstrated statistically significant and clinically meaningful functional improvements compared to external controls." The results presented at ASGCT are from the ongoing analysis of results from Part 2 of EMBARK, which compared two-year outcomes from 63 participants against data from an external control group of untreated individuals with Duchenne. Results at two years post-treatment showed that individuals treated with ELEVIDYS had better outcomes in multiple motor function measures, compared to a well-matched external control group. Additionally, no new safety signals were observed in the EMBARK study over the two-year duration and, in a subset of patients (n=16), micro-dystrophin expression and sarcolemmal localization was sustained from Week 12 to Week 64. “This has been a significant year for our neuromuscular portfolio, with multiple, ongoing analyses and longer-term data on efficacy and safety presented for ELEVIDYS,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “Building on the topline EMBARK Part 2 data from earlier this year, we’re committed to sharing ongoing analyses as fast as possible. The one-year results of patients treated with ELEVIDYS at 8 to 9 years old provide evidence that those treated with gene therapy outperform those who don’t receive it at a critical point when more dramatic functional decline is expected.”
May 16, 2025 at 4:31 PM
jonu235.bsky.social
Protein sure is a hot topic these days! "Treatment with #ELEVIDYS (delandistrogene moxeparvovec) for #Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6)" 💪💪💪 #GeneTherapy investorrelations.sarepta.com/news-release...
Sarepta Therapeutics Shares New Protein Expression and Safety Results from ENDEAVOR in Participants 2 Years Old at Time of Treatment - Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) - Safety profile consistent with prior studies of ELEVIDYS and real-world experience CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 16, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Treatment with ELEVIDYS in the ENDEAVOR participants in cohort 6 who were 2 years old at the time of treatment (n=6), demonstrated mean expression of 93.87% of normal, as measured by western blot, and 79.9 percent dystrophin positive fibers (PDPF), as measured by immunofluorescence. The results were seen in biopsies taken 12 weeks after treatment. Safety in cohort 6 was consistent with clinical and real-world experience with ELEVIDYS. The most common adverse events were nausea and vomiting. Elevated liver enzymes were seen in two patients and resolved with steroid administration. “The strength of the biomarker results that we are seeing in younger patients is extremely encouraging and we have a meeting with U.S. FDA next month to discuss expanding the ELEVIDYS label to include younger patients,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research and development, Sarepta Therapeutics. “In addition to positive expression results, the safety profile in these patients is consistent with what we’ve seen in prior studies and in patients who have been prescribed treatment.” Sarepta had previously shared expression and safety from Cohort 4 of ENDEAVOR, in which participants were 3 years old at the time of treatment. In biopsies taken 12 weeks after treatment with ELEVIDYS, participants in Cohort 4 (n=7) demonstrated mean protein levels of 99.64 percent, as measured by western blot. More than 25 patients under the age of 4 have now been treated in our clinical studies.
May 16, 2025 at 3:57 PM
jonu235.bsky.social
Chugai Receives Regulatory Approval for #Elevidys (delandistrogene moxeparvovec) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan 💪💚https://www.chugai-pharm.co.jp/english/news/detail/20250513181500_1160.html
Approval Information Product Name: ELEVIDYS® Intravenous Infusion Generic Name: delandistrogene moxeparvovec Contraindication and Prohibition (partial excerpt): Patients do not have a deletion of any portion or the entirety of exon 8 and/or exon 9 in the dystrophin gene Efficacy or Effects: Duchenne muscular dystrophy Indicated for patients who meet all of the following criteria: Patients who are negative for anti-AAVrh74 antibodies Ambulatory patients Patients aged 3 years or older and younger than 8 years Approval Conditions and Time Limit: [Approval Conditions] During the period until the reapplication for marketing approval of this product after conditional and time-limited approval, post-marketing approval condition evaluation shall be conducted through clinical trials aimed at confirming the long-term efficacy and safety of this product, as well as post-marketing surveillance targeting all cases in which this product is used. Necessary measures shall be taken, including dissemination of proper use guidelines developed in cooperation with relevant academic societies, to ensure that physicians with sufficient knowledge and experience in Duchenne muscular dystrophy use this product in accordance with the "Efficacy or Effects" and "Dosage and Administration" after thoroughly acquiring knowledge of the clinical trial results and adverse events of this product, at medical institutions with established systems for treating Duchenne muscular dystrophy. Necessary measures shall be taken, including dissemination of the usage regulations, to ensure that this product is used in compliance with the Type 1 Use regulations approved under the "Act on the Conservation and Sustainable Use of Biological Diversity through Regulations on the Use of Living Modified Organisms (Act No. 97 of 2003)." [Time Limit] 3 years [Reference] Chugai Files Elevidys (SRP-9001) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan (Press release by Chugai issued on August 14, 2024) https://www.chugai-pharm.co.jp/english/news/detail/20240814150000_1091.html Roche Announces Outcomes for EMBARK Study of Elevidys in Duchenne Muscular Dystrophy (Press release by Chugai issued on October 31, 2023)
May 13, 2025 at 5:07 PM
jonu235.bsky.social
apnews.com/article/fda-... @apnews.com
Nearly half the FDA’s $7 billion budget comes from fees collected from drug, device and tobacco companies. The agency uses the money to hire thousands of staffers to quickly and efficiently review new products. For example, about 70% of the FDA’s drug program is financed by user-fee agreements, which must be reauthorized by Congress every five years. But the agreements stipulate that if FDA’s federal funding falls below set levels, companies are no longer required to pay and, in some cases, can claw back their money. The threshold requirements are designed to ensure Congress continues funding FDA, rather than relying entirely on the private sector.
April 10, 2025 at 4:52 PM
jonu235.bsky.social
Sarepta Therapeutics @sarepta.com Provides (safety) Update on #ELEVIDYS (delandistrogene moxeparvovec) #Duchenne muscular dystrophy #GeneTherapy #RareDisease investorrelations.sarepta.com/news-release...
Sarepta Therapeutics Provides Update on ELEVIDYS 04/04/25 8:44 AM EDT CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 4, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne muscular dystrophy. Following the safety update on acute liver failure that was issued on March 18, European Union (EU) reference member country authorities requested that the independent data monitoring committee (DMC) meet to review the adverse event. While the analysis is being finalized, recruitment and dosing in certain clinical studies of ELEVIDYS are temporarily halted. The independent DMC met on April 3 and concurred that based on the totality of evidence, the overall benefit-risk profile remains favorable to continue dosing in the paused clinical trials without changes to the study protocols. At the request of EU regulators, Sarepta and Roche will submit this information in a response to the temporary halt within a week. Evaluation of the submission and the subsequent decision for lifting the temporary halt will follow the EU regulatory process. The clinical studies affected by the temporary halt are Study SRP-9001-302 (ENVOL), Study SRP-9001-303 (ENVISION) and Study SRP-9001-104. Monitoring and data collection for already-enrolled participants continues, and we do not anticipate a material impact on the timeline for these studies.
April 4, 2025 at 7:21 PM
jonu235.bsky.social
"And so far, the results have been eye-opening." 💪 M Health Fairview Masonic Children's Hospital @wcconews.bsky.social @sarepta.com #Elevidys delandistrogene moxeparvovec #Duchenne muscular dystrophy #GeneTherapy #RareDisease cbsnews.com/minnesota/ne...
And so far, the results have been eye-opening. In three months they've watched their son get stronger. He's able to go up and down the stairs on his own. And if Colton Belluzzo has his way, he'll be playing baseball in no time. "Just his energy levels are higher. He can do things. He's playing a lot more," said Dan Belluzzo. "It's amazing, it's amazing that we were able to make this happen for our child," said Morgan Belluzzo.
March 22, 2025 at 12:58 AM
jonu235.bsky.social
AJMC: Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD 💪 'Study results support the role of functional dystrophin and suggest that #Elevidys stabilizes or slows #Duchenne muscular dystrophy progression.' @mda.org @sarepta.com #GeneTherapy www.ajmc.com/view/delandi...
March 21, 2025 at 6:14 PM
jonu235.bsky.social
$SRPT 2025 Muscular Dystrophy Association MDA @mda.org Clinical and Scientific Conference - @sarepta.com Supporting Materials #Duchenne muscular dystrophy #LGMD Limb Girdle Muscular Dystrophy #RareDisease investorrelations.sarepta.com/events/event...
March 17, 2025 at 3:05 AM
jonu235.bsky.social
$SRPT Expert (Craig McDonald, MD @ucdavishealth.bsky.social ): Pharmacists Play Crucial Role in Safe, Effective Elevidys Treatment in DMD @pharmacytimes.bsky.social
#Duchenne muscular dystrophy #Elevidys delandistrogene moxeparvovec #GeneTherapy #RareDisease pharmacytimes.com/view/expert-...
Pharmacy Times: Is there anything else that you want to add? McDonald: I would just like to say that we're excited about the conduct of future clinical trials. Many of those trials, the Elevidys trials in the non-ambulatory population, are being conducted at European sites. We're now commercially treating non-ambulatory patients based on accelerated approval criteria that allow us to treat patients if we obtain insurance authorization to try to get the patients to the drug treatment for these non-ambulatory patients sooner. We're really looking forward to getting more safety and efficacy data on the older, non-ambulatory patients with DMD in terms of preservation of arm function, upper limb function, and also preservation of breathing function and heart function.
March 16, 2025 at 8:07 PM
jonu235.bsky.social
not quite the snip I wanted.. better.. #CaseOfTheMondays
March 10, 2025 at 5:31 PM
jonu235.bsky.social
Let's see.. President Elmo's rockets.. 💥 President Elmo's social media site.. 💥 President Elmo's car company.. 💥 Wonder if he'll go four for four on running America.. 💥? You know those billionaires.. overachievers..
March 10, 2025 at 5:24 PM