Children's gospel! Two new SMA drugs have made positive progress June 19, 2018 Source: WuXi PharmaTech Spinal muscular atrophy (SMA) is a serious genetic disorder that can lead to a worsening of muscle function in patients and leading to death. According to statistics, SMA is one of the most deadly genetic diseases, but our treatment options are still very limited. Fortunately, two recent clinical trials have shown us hope for more new drugs to go on the market. ▲SMA affects a large number of children (Source: Pixabay) The first study came from the research therapy reldesemtiv of Cytokinetics. This is the next generation of fast skeletal muscle troponin activator, which slows the release of calcium ions from the troponin complex in fast skeletal muscle fibers, thereby enhancing skeletal muscle contraction. In a phase 2 clinical trial, it achieved primary and secondary clinical goals. The test used two doses of reldesemtiv (150 mg or 450 mg). Studies have shown that the patient's six-minute walking distance is improved and the degree of improvement is dose-related. In the 450 mg group, the walking distance of the patients increased by 35.63 m (4 weeks, p = 0.0037) and 24.89 m (8 weeks, p = 0.0584), respectively, compared with placebo. In addition, the patient's maximum respiratory pressure has also increased. “We are pleased to see that the treatment of reldesemtiv has the potential to bring clinical benefits to adolescents and adult SMA patients. This is supported by the dose-dependent six-minute walking distance and maximum respiratory pressure data compared with placebo,†Dr. Fady I. Malik, Executive Vice President of Research and Development at Cytokinetics, said: "Considering that there is no efficacy platform and no limit on the safety or tolerability of the dose, we believe that these data support our assessment in future clinical trials. Higher doses of reldesemtiv." In addition, PTC Therapeutics also published an interim data from a clinical trial of the research therapy risdiplam (RG7916). This new drug regulates the splicing of SMN2 RNA and produces functional transcription. Previously, it has been demonstrated that this new drug can cross the blood-brain barrier by oral administration and can be systematically delivered to organs with lower levels of SMN protein. In a publicly labelled clinical trial, the researchers obtained the results of the first partial dose escalation trial. They were pleased to find that on Day 182, 90% of children achieved a score of more than 4 points in the CHOP-INTEND score (evaluating the patient's motor development level) compared to baseline. The second part of the trial is ongoing. “We are pleased to see that the protein production has increased by as much as 6.5 times, and it is good to see that increased protein levels translate into clinical benefits,†said Dr. Stuart W. Peltz, CEO of PTC Therapeutics: “The survival rate Data and CHOP-INTEND scores have great potential. In general, children with Category 1 SMA do not experience functional motor improvement. We look forward to sharing further results from these projects at future medical conferences." We look forward to more innovative SMA therapies to come out and benefit patients! Reference materials: [1] Cytokinetics Announces Data From Phase 2 Clinical Study of Reldesemtiv in Patients With Spinal Muscular Atrophy Presented at the 2018 Annual Cure SMA Conference [2] Updated Preliminary Data from SMA FIREFISH Program in Type 1 Babies Presented at the CureSMA Conference
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