Disc Medicine周一表示，其针对一种因阳光导致极端疼痛的罕见病的实验性药片在晚期试验中达到了所有主要和次要目标，导致其股价在盘前交易中飙升65%。
Disc Medicine said on Monday its experimental pill for a rare disease that causes extreme pain from sunlight met all of its main and secondary goals in a late-stage trial, sending its shares surging 65% in premarket trading.
公司正在测试比托珀林作为治疗红细胞生成性原卟啉症（EPP）的药物，这是一种使患者对阳光极度敏感的血液疾病。该病会导致一种称为原卟啉IX（PPIX）的物质在血液中积聚，当患者暴露在阳光下时会引发疼痛的皮肤反应。此结果是在今年早些时候美国FDA因未能明确建立患者原卟啉水平降低与其对阳光耐受性之间的关联而拒绝批准该药物之后得出的。
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公司表示，共有183名年龄在12岁及以上的患者服用比托珀林后，其血液中的PPIX水平比安慰剂组低了41.4%。比托珀林总体耐受性良好，未发现新的安全问题。Disc Medicine表示，通过待在室内和穿戴防护衣物来避免阳光仍是管理该病的主要方式。目前美国仅批准的EPP治疗方法是一种称为Scenesse的皮下激素植入剂。
The company was testing bitopertin as a treatment for erythropoietic protoporphyria (EPP), a blood disorder that makes patients extremely sensitive to sunlight The condition causes a substance called protoporphyrin IX (PPIX) to build up in the blood, which causes painful skin reactions when patients are exposed to sunlight The results come after the US FDA declined to approve the drug earlier this year, citing a lack of a clear link between the reduction in patients' protoporphyrin levels and their tolerance to sunlight A total of 183 patients aged 12 and older who took bitopertin had 41.4% less PPIX in their blood compared with those on placebo, the company said Bitopertin was generally well tolerated with no new safety concerns, Disc Medicine said Avoiding sunlight, by staying indoors and wearing protective clothing, remains the main way to manage the disease The only US-approved treatment for EPP is an under-the-skin hormone implant known as Scenesse