Latest news with #DMD
Time of India
an hour ago
- General
- Time of India
Cal HC bats for more centres of excellence for rare diseases in state
Kolkata: Persons suffering from rare diseases should be treated as a "class of themselves", the Calcutta High Court observed while suggesting the state increase the monthly stipend from Rs 1,000 to Rs 5,000. The division bench of Chief Justice TS Sivagnanam and Justice Chaitali Chatterjee (Das) has now requested Additional Solicitor General (ASG) Ashoke Kumar Chakraborti to examine the policy by the central govt on the establishment of more than one centre of excellence for rare diseases in a state. There is only one centre of excellence for rare diseases in the eastern region — IPGMER. The division bench was hearing a PIL filed by parents of children suffering from muscular dystrophy, a rare disease. On the court's inquiry, the state informed that they pay a monthly stipend of Rs 1,000 to people suffering from rare diseases, the same as the amount paid to persons with disabilities. "...Assist the court as to what directions can be issued so that another centre of excellence can be established in Bengal since the court is informed that not only patients from Bengal come here but also from the neighbouring states" the CJ told ASG. "So far as persons suffering from rare diseases are concerned, they cannot be equated to a person who is normally classified as a person with disability. Though the person suffering from rare diseases is undoubtedly a person of rare disability, the disability is more severe as it is a degenerative disease," the bench observed. The court suggested that the tablet "Deflazecort," which is the approved therapy for DMD, be given to the patients to cover the dosage for a minimum of two months. If the tablets dispensed are for a lesser period, then each time the patient needs to travel to the hospital, which is a herculean task for persons suffering from rare diseases. One of the parents of the patients in court, who happens to be an employee of the High Court, stated that his son recently became very sick, and since no ICU bed was available, he admitted his son to a private hospital. He was kept in the ICU for 10 days, and the cost incurred was very high and unmanageable. The state was directed to submit a report on June 27 on the establishment of three ICU units in the police hospital, which will provide exclusive care to persons suffering from rare diseases, apart from the facility available in the SSKM hospital and the other hospitals situated in and around the police hospital. "This is so required because high-speed oxygen is required for the patients, and in the event there is no bed available in the SSKM hospital main campus, then the patient will be put to irreparable hardship," CJ stated. The state was also directed to take expeditious decisions and ensure that the five mechanised wheelchairs are procured and supplied to the hospital no later than June 13. The court also asked the state whether a subsidy can be given for the purchase of a mechanised wheelchair.
Yahoo
5 days ago
- Business
- Yahoo
PepGen price target lowered to $1 from $3 at BofA
BofA lowered the firm's price target on PepGen (PEPG) to $1 from $3 and keeps an Underperform rating on the shares after the company announced it will be discontinuing development of Duchenne muscular dystrophy programs following 'disappointing' 10mg/kg data for PGN-EDO51. PepGen will now focus developmental efforts on its DM1 program, which the firm thinks 'still needs significant de-risking,' the analyst tells investors. The firm updated its model for Q1 results, cash and share count and removed value for DMD from its model based on the discontinuation. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See Insiders' Hot Stocks on TipRanks >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO Sign in to access your portfolio
Yahoo
5 days ago
- Business
- Yahoo
PepGen to discontinue DMD programs after PGN-EDO51 did not achieve target
PepGen (PEPG) announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy patients amenable to exon 51 skipping, the company will focus on advancing its 'promising' myotonic dystrophy type 1 program currently in Phase 2 clinical development. The company is voluntarily discontinuing development of PGN-EDO51 and intends to wind down all DMD-related research and development activities. In the 10 mg/kg cohort of the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26%; however, total dystrophin only increased to 0.59% of normal levels. The safety profile of PGN-EDO51 continued to be generally favorable and all treatment-related adverse events were mild in nature. No serious adverse events were reported in the study. 'We are disappointed by the dystrophin results observed in the 10 mg/kg dose cohort in CONNECT1, as it was our hope that we could improve upon existing therapies for patients in a more profound way,' said James McArthur, PhD, President and CEO of PepGen. 'As we wind down our DMD program, we would like to thank the patients, families, caregivers, investigators and study staff for their support and participation in this research. I also want to acknowledge our team's hard work and commitment to advancing new potential treatments for DMD patients.' PepGen continues to expect to report data from its FREEDOM-DM1 15 mg/kg cohort in patients with DM1 during the second half of 2025. FREEDOM is a Phase 1 single ascending dose, randomized, placebo-controlled clinical trial, with endpoints including safety, 28-day splicing correction and functional benefit measures. The company also continues to expect to report data from the 5 mg/kg cohort of its FREEDOM2-DM1 study in DM1 patients in the first quarter of 2026. FREEDOM2 is a Phase 2 multiple ascending dose, randomized, placebo-controlled clinical trial, with endpoints following four doses that include safety, splicing correction and functional benefit measures. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on PEPG: Disclaimer & DisclosureReport an Issue PepGen price target lowered to $8 from $14 at H.C. Wainwright PepGen Inc.'s Strategic Shift Amidst DMD Program Discontinuation and Competitive Challenges PepGen Inc. Discontinues DMD Programs After Trial Results PepGen appoints Kasra Kasraian as CTO PepGen Inc.'s Promising Advances in DMD and DM1 Programs Justify Buy Rating 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
5 days ago
- Business
- Yahoo
PepGen Inc. (PEPG) Halts DMD Program After Trial Miss; Shifts Focus to DM1
Boston-based biotech PepGen Inc. (NASDAQ:PEPG) announced today it will discontinue all Duchenne muscular dystrophy (DMD) research after its lead candidate, PGN-EDO51, failed to achieve target dystrophin levels in the CONNECT1-EDO51 trial. Despite being well tolerated at 10 mg/kg, PGN-EDO51 only increased dystrophin to 0.59% of normal levels, falling short of expectations. All treatment-related adverse events were mild, and no serious events were reported. A healthcare professional in a meeting with a patient discussing care options using digital technology. 'We are disappointed by the results and will wind down our DMD program,' said CEO James McArthur, PhD, thanking patients and staff for their support. PepGen Inc. (NASDAQ:PEPG) will now focus on its promising myotonic dystrophy type 1 (DM1) program, PGN-EDODM1, which has shown robust target engagement and a favorable safety profile. PepGen Inc. (NASDAQ:PEPG) expects data from its Phase 1 FREEDOM-DM1 trial's 15 mg/kg cohort in the second half of 2025, and from the Phase 2 FREEDOM2-DM1 5 mg/kg cohort in early 2026. PGN-EDODM1, which has Orphan Drug and Fast Track status, targets the genetic root of DM1, a rare, life-shortening disorder with no approved treatments. While we acknowledge the potential of PEPG to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than PEPG and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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
5 days ago
- Business
- Yahoo
Intellia stock dips following safety event in Phase III gene therapy trial
Intellia Therapeutics' stock has fallen nearly 23% following an announcement that a patient in its Phase III trial for a gene therapy targeting transthyretin amyloid (ATTR) cardiomyopathy suffered a serious adverse event (AE). In a filing by the Securities and Exchange Commission (SEC), the company reported that one patient experienced Grade 4 liver transaminase elevations after treatment with nexiguran ziclumeran (nex-z) in the Phase III MAGNITUDE trial (NCT06128629). According to Intellia, the event is resolving without the need for hospitalisation or medical intervention. Upon the announcement, Intellia's stock, listed on the Nasdaq exchange, dropped by 22.87% from $9.66 a share at market close on 28 May to $7.45 at market close on 29 May. Intellia emphasised that this is a single event from more than 200 patients dosed with nex-z in the MAGNITUDE study. Other AEs have been similar to those seen in the Phase I study, including infusion-related reactions and asymptomatic liver transaminase elevations. The SEC filing added that Intellia said it will continue to monitor these events as the study progresses. The trial has so far enrolled 365 patients out of an expected total enrollment of approximately 765 patients, which Intellia says is on track. The company aims to complete enrollment by early 2027. Intellia is also investigating nex-z in the Phase III MAGNITUDE-2 study (NCT06672237), which is enrolling patients with hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Dosing has started, with investigators seeking data on whether the therapy will lead to a reduction in serum TTR. Intellia will be waiting for data from MAGNITUDE-2 before filing a biologics licence application (BLA) to the US Food and Drug Administration (FDA) in 2028, with hopes for a 2029 commercial launch. It has been a challenging time for gene therapy development, with several severe AEs and, in some instances, fatalities seeming to plague investors' views on the therapeutic approach. On 27 May, Rocket Pharmaceuticals announced that a patient died in its Phase II trial for a Danon disease gene therapy after suffering a serious AE. In March 2025, Sarepta and Roche reported the death of a teenage patient dosed with its AAV gene therapy for Duchenne muscular dystrophy (DMD), Elevidys (delandistrogene moxeparvovec-rokl), causing agencies to pause ongoing clinical studies. This pause has since been lifted. Last year, a fatality in Pfizer's Phase II DMD gene therapy trial, as well as low efficacy in a Phase III study, caused the big pharma to drop the development of the AAV candidate, named fordadistrogene movaparvovec. Earlier this month, the FDA shared that American haematologist oncologist Vinay Prasad will lead its Center for Biologics Evaluation and Research (CBER), the division responsible for regulating gene therapies and vaccines. Prasad has openly criticised Elevidys, primarily about the clinical evidence and the FDA's decision to approve the therapy, which raises questions about his overall opinion of the gene therapy approach. "Intellia stock dips following safety event in Phase III gene therapy trial" 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. 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
