相关临床试验
15
0 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
已完成
11
73.3%
招募中
2
13.3%
终止
1
6.7%
撤回
1
6.7%
暂无批准数据
- A new multisource analysis estimates Phelan-McDermid syndrome affects approximately 1 in 7,300 people, dramatically exceeding prior prevalence estimates. - Researchers analyzed genetic testing data from nearly 180,000 individuals with autism across ten independent sources, including GeneDx, Labcorp, and the SPARK study. - The findings suggest more than 45,000 people in the United States may be living with the condition, with a large gap between known and estimated cases. - Multiple clinical trials are underway for PMS, making expanded genetic testing access critical for connecting undiagnosed patients to emerging precision therapies.
- Neuren Pharmaceuticals has dosed the first patient in its landmark Koala Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome, marking the first-ever Phase 3 study for this rare neurodevelopmental disorder. - Published Phase 2 data showed statistically significant improvements across 10 of 14 efficacy assessments, including communication, behavior, and quality of life measures in 18 children with PMS. - The Phase 3 trial has received regulatory approval from both the FDA and Health Canada, with plans to enroll approximately 160 children aged 3-12 years across multiple sites. - NNZ-2591 has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations by the FDA, highlighting the urgent unmet medical need for PMS treatments.
- Neuren Pharmaceuticals received FDA Fast Track designation for NNZ-2591 to treat Phelan-McDermid syndrome and Angelman syndrome, potentially accelerating development and review timelines. - The company has initiated its Phase 3 "Koala" clinical trial evaluating NNZ-2591 in children aged three to 12 years with Phelan-McDermid syndrome. - Neuren shares surged 8.8% following the announcement and have gained 71% over the past 12 months, with the stock up 79% year-to-date.
- Neuren Pharmaceuticals achieved record quarterly royalties of A$16.4 million from DAYBUE (trofinetide), driven by Acadia Pharmaceuticals' US$101.1 million in third-quarter sales for the Rett syndrome therapy. - The number of patients receiving DAYBUE shipments surpassed 1,000 for the first time, with 74% of new prescriptions now written by community-based physicians rather than specialists. - Neuren's second drug candidate NNZ-2591 has initiated its first-ever Phase 3 trial for Phelan-McDermid syndrome, marking a significant milestone for this rare neurodevelopmental disorder with no approved treatments. - The company's strong financial position includes $300 million in cash and investments, supporting accelerated development of multiple neurodevelopmental disorder programs.
- Neuren Pharmaceuticals has initiated the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome, marking the first-ever Phase 3 trial for this severe neurodevelopmental disorder. - The randomized, double-blind, placebo-controlled trial will assess 13 weeks of treatment in approximately 160 children aged 3 to 12 with PMS, with all participants eligible for a 12-month open-label extension. - Phelan-McDermid syndrome affects an estimated 1 in 8,000 to 1 in 15,000 people and currently has no approved treatments available. - The trial program is fully funded by Neuren's existing cash reserves, following FDA alignment on the Phase 3 trial design including primary efficacy endpoints.
- Neuren Pharmaceuticals received US patent approval for NNZ-2591 to treat Pitt Hopkins syndrome, with patent protection extending to April 2040. - Phase 2 clinical trial results showed 9 out of 11 children demonstrated clinician-assessed improvements in communication, social interaction, cognition and motor abilities. - The FDA has granted both Orphan Drug and Fast Track designations for NNZ-2591 in PTHS, while no approved treatments currently exist for this debilitating condition. - Neuren also secured Rare Pediatric Disease Designation for NNZ-2591 in Phelan-McDermid syndrome, potentially qualifying for a priority review voucher.
- Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug. - In the Phase 1/2 HALOS study, 97% of participants receiving medium or high doses of ION582 showed improvements in communication, cognition, and motor function, with favorable safety profiles. - The race to develop treatments for Angelman syndrome is intensifying, with Ultragenyx's GTX-102 already in Phase 3 trials and Neuren Pharmaceuticals advancing an oral synthetic peptide, highlighting significant progress in addressing this rare genetic disorder.
- Neuren Pharmaceuticals receives FDA Fast Track designation for their investigational treatment targeting Pitt Hopkins syndrome, a rare neurodevelopmental disorder affecting 1 in 34,000-41,000 people. - The Fast Track status aims to expedite the development and review process for this potential first-ever approved treatment for Pitt Hopkins syndrome. - Company shares responded positively to the announcement, rising 5.4% to $13.77, marking a significant development in addressing this serious unmet medical need.
- Immutep's IMP-761, a LAG-3 agonist, demonstrates a favorable safety profile in Phase I trials, potentially addressing autoimmune diseases like rheumatoid arthritis. - Percheron Therapeutics anticipates Phase 2b trial results for ATL-1102 in Duchenne muscular dystrophy, a genetic condition affecting muscle protein production. - PYC Therapeutics reports positive pre-clinical data for PYC-002, targeting the underlying cause of Phelan-McDermid syndrome, with human trials expected in 2026.
- Several ASX-listed biotech companies are anticipating crucial Phase III clinical trial results in 2025, which could significantly impact their market value and future prospects. - Dimerix expects results from its ACTION3 Phase III trial of DMX-200 for focal segmental glomerulosclerosis (FSGS) by mid-2025, a rare kidney condition with no approved treatments. - Opthea anticipates Phase III trial results for sozinibercept (OPT-302) in combination with aflibercept or ranibizumab for wet age-related macular degeneration (wet-AMD) in 2025. - Recce Pharmaceuticals is set to commence a Phase III trial in Indonesia for RECCE 327 as a topical gel to treat diabetic foot infections (DFI) in early 2025.
