Duchenne Muscular Dystrophy New Drug Phase 2 Positive Results
February 28, 2018 Source: WuXi PharmaTech
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];Summit Therapeutics recently announced more positive results from the clinical trial of PhaseOut DMD. The trial is a multicenter, open-label, phase 2 clinical trial designed to evaluate the efficacy and safety of ezutromid in patients with Duchenne muscular dystrophy (DMD). Ezutromid is a utrophin protein regulator. Further analysis of the 24-week interim data showed a statistically significant decrease in the transverse relaxation time T2 inversion spectrum (MRS-T2) measured by magnetic resonance in the treated patients.
DMD is a progressive muscular atrophy, which mainly affects men, because the gene causing the disease exists on the X chromosome, and the male has only one X chromosome, so there is only one DMD allele. If the gene is defective, it may be affected. disease. According to statistics, there are about 50,000 male children and adolescents in developed countries suffering from the disease. The DMD gene encodes a dystrophin protein, which is mainly found in skeletal muscle and cardiomyocytes, and is also expressed in nerve cells, which is required for muscle cells to perform healthy functions. DMD is currently incurable and the average life expectancy of patients is only around 26 years old. For these patients, extending life expectancy is the most urgent medical need.
Summit's ezutromid is an oral small molecule drug that is a regulator of utrophin protein. The Utrophin protein is functionally and structurally similar to the dystrophin protein. Preclinical studies have shown that sustained expression of utrophin has a positive effect on muscle performance. Summit believes that regardless of the mutation in the dystrophin protein gene, if the patient's utrophin protein is regulated, it may slow or even stop the development of DMD. Moreover, utrophin regulation has the potential to complement other DMD therapies. The US FDA and the European Medicines Agency (EMA) have granted ezutromid orphan drug status.
The new results announced this time came from the PhaseOut DMD Phase 2 clinical trial, which recruited 40 patients in the United States and the United Kingdom (two of whom withdrew for reasons unrelated to ezutromid), aged 5-10 between. The trial lasted for 48 weeks, after which the patient was selected to continue taking ezutromid for an extended period. All patients underwent muscle biopsy of the biceps at baseline, with 24 patients undergoing a second biopsy 24 weeks after dosing and the remaining 16 patients undergoing a second biopsy 48 weeks after dosing. The aim of this trial was to validate the effect of ezutromid on the regulation of utrophin by measuring utrophin protein, muscle fiber regeneration and muscle fat infiltration in muscle biopsy samples. The primary endpoint of the trial was the change in magnetic resonance spectroscopy parameters associated with the leg muscles compared to baseline. Secondary endpoints included assessment of utrophin and muscle damage by tissue biopsy. Exploratory endpoints include a six-minute walking distance, a North Star Ambulatory Assessment, and patient report results.
The 24-week interim data reported in January 2018 showed that biomarker developmental myosin levels were measured by muscle tissue biopsy and a significant and meaningful reduction in muscle damage was observed. PhaseOut DMD is still in progress and is expected to announce the top line results for the 48th week in the third quarter of this year.
The new analysis released this time showed that the decrease in MRS-T2 measured in PhaseOut DMD was consistent with the expected effect of ezutromid stabilizing myofiber membrane to reduce muscle fiber damage and inflammation. Previous studies have shown a reduction in MRS-T2 in patients with DMD treated with steroids. However, because all patients participating in PhaseOut DMD are receiving a stable steroid regimen, the reduction in MRS-T2 observed in the trial is beyond the effects of steroids. The new MRS data showed that patients treated with ezutromid (n=38) had a statistically significant decrease in the T2 relaxation time of the soleus muscle (soleus, calf muscle) compared to baseline, with an average fall time of -0.861 ms (baseline). 31.850 milliseconds to 24 weeks 30.989 milliseconds, 95% CI, -1.440, -0.281). The soleus muscle is one of the most reliable leg muscles in monitoring disease progression in DMD patients, and an increase in T2 relaxation time of this muscle is associated with loss of muscle function. Published data show that the increase in T2 relaxation time as the disease progresses is due to inflammation caused by the cycle of muscle damage and repair. In patients treated with ezutromid, an average of -0.470 milliseconds of MRS-T2 was observed in the lateral femoral muscles (vastus lateralis, thigh muscles) (baseline 32.265 milliseconds to 24 weeks 31.795 milliseconds, 95% CI, -1.158, 0.218) .
â–² Dr. H Lee Sweeney, Director of the Institute of Muscle Research at the University of Florida and Director of Imaging DMD (Source: University of Florida Official Website)
"MRS-T2 is a technology that can objectively monitor the progression of DMD disease because it accurately quantifies muscle damage and inflammation. In DMD patients, MRS-T2 values ​​usually increase over time," University of Florida Muscle Study Dr. H Lee Sweeney, Director of the Myology Institute at the University of Florida and Director of Imaging DMD, commented: "The reduction in MRS-T2 observed in PhaseOut DMD is encouraging, suggesting that ezutromid has a positive effect on muscle health. The data may indicate that the severity of the disease in these patients is decreasing and shows that ezutromid has the potential to affect the disease. I look forward to seeing more results from the PhaseOut DMD trial."
â–² Dr. David Roblin, Chief Medical Officer and R&D President of Summit (Source: Summit Therapeutics Official Website)
“A 24-week mid-term analysis of PhaseOut DMD shows ezutromid's encouraging activity, and these MRS-T2 results show a positive impact of treatment on downstream muscle health,†added Dr. David Roblin, Chief Medical Officer and R&D President of Summit. "This result, combined with evidence that ezutromid regulates utrophin protein and significantly reduces muscle damage, further demonstrates that ezutromid has the potential to be a therapeutic benefit for all genetically modified DMDs."
Reference materials:
[1] Summit announces new analysis showing ezutromid significant reduced muscle inflammation in Phase 2 clinical trial in DMD
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