Today, a brief rundown of news involving Madrigal Pharmaceuticals and Viridian Therapeutics, as well as updates from Apellis Pharmaceuticals, PTC Therapeutics and Arrowhead Pharmaceuticals that you may have missed.

今天，简要概述一下涉及百灵佳制药和维里迪恩治疗公司以及 Apellis 制药、PTC 治疗和箭头制药的新闻更新，您可能错过了这些信息。

Seeking to expand treatment of the liver disease MASH,

寻求扩大对肝病MASH的治疗，

Madrigal Pharmaceuticals

麦德里加尔制药公司

is

是

paying $120 million

支付1.2亿美元

to Hong Kong-based drugmaker

总部位于香港的制药商

CSPC Pharmaceutical Group

石药集团

for global rights to an oral GLP-1 drug in preclinical development. The deal offers CSPC potentially $2 billion in additional payments if certain development, regulatory and commercial milestones are achieved. Madrigal executives said they plan on combining the CSPC drug, called

对于一款处于临床前开发阶段的口服GLP-1药物的全球权利。如果达成特定的开发、监管和商业里程碑，该协议可能为石药集团带来额外20亿美元的付款。马德里加尔制药的高管表示，他们计划将石药集团的这款药物（称为

SYH2086

SYH2086

, with the company’s MASH drug

，该公司的MASH药物

Rezdiffra

Rezdiffra

in a once-daily pill. They envision treatment could combine the benefits of weight loss from a GLP-1 pill with the antifibrotic and fat reducing qualities of Rezdiffra to give people with MASH additional disease control. —

在每日一次的药丸中，他们设想治疗可以结合GLP-1药丸的减重效果与Rezdiffra的抗纤维化和减脂特性，为MASH患者提供更多的疾病控制。——

Jonathan Gardner

乔纳森·加德纳

Kissei Pharmaceutical

岐阜制药

is paying $70 million upfront and offering up to $315 million in milestone payments to

预先支付7000万美元，并提供高达3.15亿美元的里程碑付款给

Viridian Therapeutics

翡翠治疗学

for Japanese rights

关于日本版权

to two antibody drugs for thyroid eye disease. Viridian has already announced

对两种用于治疗甲状腺眼病的抗体药物。Viridian已经宣布了

positive Phase 3 study data

三期阳性研究数据

for the most advanced drug in the deal, called veligrotug. The second, called VRDN-003, is in ongoing Phase 3 testing. The company hopes to compete with Amgen’s marketed drug for thyroid eye disease, Tepezza, by offering a

在此次交易中，最先进的药物名为veligrotug。第二种药物名为VRDN-003，正在进行第三阶段测试。该公司希望通过提供一种药物与安进公司已上市的甲状腺眼病药物Tepezza竞争，

more convenient dosing regimen

更方便的给药方案

.—

.—

Jonathan Gardner

乔纳森·加德纳

The

The

Food and Drug Administration

食品药品监督管理局

on Monday approved broader use of

周一批准了更广泛的使用

Apellis Pharmaceuticals

Apellis制药公司

’ rare disease drug Empaveli, clearing it for the kidney diseases C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis in people 12 years of age or older. In testing, treatment with Empaveli reduced urine protein levels by significantly more than placebo and stabilized kidney function.

罕见病药物Empaveli获批用于治疗12岁及以上患者的C3肾小球病或原发性免疫复合物膜增生性肾小球肾炎。在试验中，使用Empaveli治疗显著降低了尿蛋白水平，效果远优于安慰剂，并稳定了肾功能。

Earlier this year, .

今年早些时候，。

Novartis

诺华

won FDA approval

获得FDA批准

of its drug

其药物的

Fabhalta

法布哈塔

for use in adults with C3G. —

用于治疗成人C3G。—

Ned Pagliarulo

内德·帕利亚鲁洛

People with the rare disease phenylketonuria, or PKU, have a new treatment option after the FDA on Monday

患有罕见疾病苯丙酮尿症（PKU）的患者在周一FDA批准后有了新的治疗选择。

approved the drug

批准了该药物

Sephience

赛菲恩斯

from

来自

PTC Therapeutics

PTC治疗公司

. In a statement, the company highlighted what it described as a broad label that includes all disease subtypes in people who are 1 month of age or older. News of the approval sent shares in PTC higher by nearly 10% this week. The company is also awaiting agency decisions on drugs for

公司在一份声明中强调了它所谓的广泛标签，包括所有一个月或以上的疾病亚型。批准的消息使PTC的股价本周上涨了近10%。该公司还在等待机构对其药物的决定。

Friedrich's ataxia and Duchenne muscular dystrophy. —

弗里德赖希共济失调和杜氏肌营养不良。——

Ned Pagliarulo

内德·帕利亚鲁洛

Arrowhead Pharmaceuticals

箭头制药公司

said Monday

周一表示

that it had earned a $100 million milestone payment from

它已经赚得了1亿美元的里程碑付款

Sarepta Therapeutics

萨雷普塔治疗公司

under their research alliance, after meeting a patient enrollment target in a Phase 1/2 trial of its treatment for myotonic muscular dystrophy. Such fees are part of the normal course of business in biotech, but payment by Sarepta had suddenly become in doubt after the

在他们达成研究联盟后，其治疗肌强直性肌营养不良的1/2期试验达到了患者招募目标。这类费用是生物技术行业正常业务的一部分，但在

company’s standoff with the FDA

公司与FDA的对峙

over whether it could continue selling its gene therapy

关于是否可以继续销售其基因疗法

Elevidys

艾勒维迪斯

. The Duchenne treatment is vital to Sarepta’s financial future, so the prospect of it being removed from market sparked concerns Sarepta might reach a point when it would no longer be able to meet its obligations. That risk appears to have receded for now, after the FDA

杜氏治疗对Sarepta的财务未来至关重要，因此它可能被撤出市场的前景引发了人们对该公司的担忧，认为它可能会达到无法再履行义务的地步。在FDA介入后，这一风险目前似乎已经有所缓解。

relented Tuesday

周二让步了

. Arrowhead expects to receive the $100 million within 60 days. —

Arrowhead预计将在60天内收到1亿美元。——

Ned Pagliarulo

内德·帕利亚鲁洛