Dive Brief:

简报：

An experimental drug from Belite Bio

Belite Bio公司的实验药物

succeeded in a Phase 3 trial

在三期试验中取得成功

in the most common form of Stargardt disease, positioning the company to seek regulatory approval next year of what could be the first marketed medicine for the condition.

在最常见的斯塔加特病形式中，使公司有望在明年寻求监管批准，这可能是该疾病首个上市的药物。

According to Belite, treatment with its drug, known as tinlarebant, was associated with a roughly 36% reduction in the growth rate of retinal lesions compared to a placebo over the course of two years, meeting the trial’s main goal. Both study groups had a minimal overall change in visual acuity, but Belite said that finding was “consistent” with historical data..

根据Belite的说法，其药物tinlarebant的治疗在两年内使视网膜病灶的增长率相比安慰剂减少了大约36%，达到了试验的主要目标。两个研究组的视力总体变化都很小，但Belite表示这一结果与历史数据“一致”。

Belite said tinlarebant was “well tolerated,” with only four patients stopping treatment due to adverse events. The most common eye side effects related to treatment were a type of color vision deficiency and issues seeing at night or adjusting to a dark environment. The majority of those cases were mild, and most resolved during the trial, the company said..

贝利特表示，丁拉瑞班“耐受性良好”，只有四名患者因不良事件停止治疗。与治疗相关最常见的眼部副作用是色觉缺陷和夜间视物困难或适应黑暗环境的问题。公司称，这些病例大多数为轻度，且在试验期间大多得到解决。

Dive Insight:

深入洞察：

Though it raised only $36 million in an initial public offering three years ago, Belite has quietly been

虽然Belite在三年前的首次公开募股中仅筹集了3600万美元，但它一直在悄然发展。

one of the biopharmaceutical sector’s top performers

生物制药领域表现最好的公司之一

ever since,

自从，

according to BioPharma Dive data

根据BioPharma Dive的数据

. Company shares, which debuted at $6 apiece, opened Monday trading at nearly $140. Belite is currently valued at around $5 billion.

公司股票每股最初以6美元的价格上市，周一开盘时已接近140美元。Belite目前的估值约为50亿美元。

The reason is tinlarebant, an oral drug with the chance to become the first marketed medicine for Stargardt. In Stargardt, a genetic defect changes how the body processes the vitamin A used to make retinal cells. That defect causes yellowish clumps to form in the eyes, destroying cells and triggering progressive vision loss.

原因是tinlarebant，这是一种口服药物，有望成为首个上市的斯塔加特病治疗药物。在斯塔加特病中，一种基因缺陷改变了身体处理用于制造视网膜细胞的维生素A的方式。这种缺陷导致眼睛中形成黄色斑块，破坏细胞并引发进行性视力丧失。

There are no available treatments for the roughly 50,000 people in the U.S. Belite estimates to have the disease..

美国大约有 50,000 人患有此病，Belite 估计目前尚无可用的治疗方法。

Tinlarebant is designed to reduce the accumulation of toxic vitamin A byproducts in the eye. The drug was first studied at Columbia University over a decade ago, and then put up for bid in a National Institutes of Health program in 2016. Belite’s CEO and chairman, Tom Lin, licensed it and formed Belite around the program two years later.

Tinlarebant旨在减少眼部有毒维生素A副产物的积累。该药物十多年前在哥伦比亚大学首次研究，然后在2016年被纳入美国国立卫生研究院项目进行招标。Belite的首席执行官兼董事长Tom Lin获得其许可，并在两年后围绕该项目成立了Belite。

Belite has since brought tinlarebant into late-stage testing in Stargardt as well as geographic atrophy, a more common type of vision loss..

贝利特随后将丁拉瑞班带入了Stargardt病以及更常见的视力丧失类型——地理性萎缩的晚期测试。

Belite’s value had already doubled this year in anticipation of the results in Stargardt. The company also received a

贝利特今年在斯塔加特结果的预期下价值已经翻倍。该公司还收到了

positive recommendation

正面建议

from trial monitors that the 104-patient study continue without modifications, and noted that China’s drugs regulator had begun reviewing an application based on interim results showing statistical significance on the study’s main measure. The likelihood of success was “fairly high” given those updates, Leerink Partners analyst Marc Goodman wrote last month.

来自试验监查员的信息显示，这项涉及104名患者的研究将继续进行，无需修改。此外，中国药品监管机构已开始根据中期结果审查一项申请，这些结果在研究的主要指标上显示出统计学意义。Leerink Partners分析师马克·古德曼上月写道，鉴于这些进展，成功的可能性“相当高”。

Goodman has forecast anywhere from $1.4 billion to $3.6 billion in peak annual sales, depending on the drug’s price..

古德曼预测，根据药品的价格，年销售额峰值可能在14亿美元到36亿美元之间。

With the positive results, Belite intends to discuss an approval application with regulatory authorities in the U.S. and elsewhere in first half of 2026. It could face competition soon, however.

有了积极的结果，Belite 打算在 2026 年上半年与美国及其他地区的监管机构讨论审批申请。然而，它可能很快面临竞争。

Alkeus Pharmaceuticals

阿尔凯乌斯制药公司

, a privately held company, is preparing a submission for a new drug that’s similarly designed to reduce the buildup of harmful vitamin A byproducts in the eye.

，一家私人公司，正在准备提交一种新药，该药物同样旨在减少眼睛中有害的维生素A副产物的积累。

Ocugen

欧库根

,

，

AAVantgarde

AAV前卫

,

，

Splice Bio

剪接生物

and

和

others

其他

are advancing genetic medicines for the condition, too.

也在推进针对该疾病的基因药物。

Stargardt experts believe gene therapy will eventually “be the preferred treatment approach” for the condition, wrote Mizuho Securities’ Graig Suvannavejh. But for now, the lack of other available therapies should mean “enthusiastic market uptake” for a drug proven to slow disease progression, he wrote in November..

斯塔加特病专家认为基因疗法最终会“成为该疾病的首选治疗方法”，Mizuho Securities的Graig Suvannavejh写道。但就目前而言，缺乏其他可用的疗法应该意味着“市场对一种被证明可以减缓疾病进展的药物的热情接纳”，他在十一月写道。

Belite shares climbed by double digits before reversing course and dipping a few percentage points early Monday.

贝尔特的股价在周一早盘前曾一度上涨了两位数，但随后逆转走势，下跌了几个百分点。