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百靈佳殷格翰 nerandomilast 在關鍵的第三期 FIBRONEER™-IPF 臨床試驗中達成主要療效指標

殷格翰, 德國,
  • FIBRONEER-IPF™ 的試驗數據顯示，臨床試驗化合物(investigational compound) nerandomilast 已達到了其主要療效指標，意即與安慰劑相比，第 52 周的用力肺活量 [mL] 改變與 基準相比具有絕對性的變化。
  • FIBRONEER™-IPF 是截至目前為止針對特發性肺纖維化 (IPF) 領域進行最大規模的臨床試驗。收案患者遍及逾 30 個國家、330 多個試驗中心。
  • 此試驗的完整療效與安全性數據將於 2025 年上半年公佈
  • 百靈佳殷格翰將向美國食品藥物管理局 (FDA) 與其他全球衛生主管機關提交 nerandomilast 用於治療特發性肺纖維化 (IPF) 的新藥申請
IPF study

百靈佳殷格翰今日宣布，FIBRONEER™-IPF 臨床試驗達成其主要療效指標，意即與安慰劑相比，第 52 周的用力肺活量 [mL] 改變與基準相比具有絕對性的變化。用力肺活量 (Forced Vital Capacity [mL] , FVC) 為肺功能之衡量指標。

根據這些研究結果，百靈佳殷格翰將向美國食品藥物管理局 (FDA) 與其他全球衛生主管機關提交nerandomilast 用於治療特發性肺纖維化 (IPF) 的新藥申請。美國食品藥物管理局 (FDA) 於 2022 年已授予其IPF 突破性療法認定。

「這是十年來首次達成主要療效指標的特發性肺纖維化 (IPF) 第三期臨床試驗」百靈佳殷格翰全球治療領域負責人 Ioannis Sapountzis 表示。「今天的公告代表著我們在這項疾病研究的悠久歷史上邁出下一步。特發性肺纖維化 (IPF) 對患者仍有許多未被滿足的需求，我們仍不斷地推動我們的研究活動，以開發更多針對這種最常見間質性肺病之一的治療選擇」

Nerandomilast 是一種口服的研究性磷酸二酯酶 4B (PDE4B) 抑制劑，目前尚未獲得使用批准，因此其安全性和療效尚未確立。它正作為 FIBRONEER™ 全球計劃的一部分研究，該計劃包括兩項第三期研究—針對 IPF 患者的 FIBRONEER™-IPF4 和針對進行性間質性肺病 (PPF) 患者的 FIBRONEER™-ILD5。

 

關於 FIBRONEER™-IPF (NCT05321069)4

一項雙盲、隨機、安慰劑對照的臨床試驗，評估 nerandomilast 在用於治療特發性肺纖維化 (IPF) 患者中至少 52 週的療效和安全性。

主要療效指標: 第52週基線用力肺活量 (FVC)（mL）改變與基準相比的絕對變化。

關鍵次要療效指標:

  • 複合指標構成要素的首次發生時間：在試驗期間首次急性 IPF 惡化；首次因呼吸原因住院；或死亡（以先發生者為準）。

受試者隨機分配至三個組別內。兩組受試者每日服用不同劑量之 nerandomilast 藥片兩次。

安慰劑組的受試者每日服用安慰劑藥片兩次。安慰劑藥片看起來像 nerandomilast，但未含有任何藥效

此試驗已在超過 30 個國家進行，且隨機分配了 1177 位患者

 

關於 FIBRONEER™ 臨床試驗計畫4,5

FIBRONEER™計畫包含兩項第三期隨機分配、雙盲、安慰劑對照試驗— FIBRONEER™-IPF (NCT05321069) and FIBRONEER™-ILD (NCT05321082) —以研究 nerandomilast 在IPF患者與PPF患者中至少52週的療效、安全性與耐受度

參與 FIBRONEERTM-IPF 試驗的患者每日兩次接受口服 nerandomilast，其劑量為 9 毫克 (mg)、 18 毫克(mg) 或安慰劑，並持續服用至少 52 週。每日兩次 18 毫克(mg)的 nerandomilast 劑量得到了第二期研究結果的支持。額外的 每日兩次9 毫克的 nerandomilast 劑量被添加進來，以評估較低劑量的效益-風險概況，並提供進一步的劑量反應和暴露反應數據。

在這兩項試驗中，主要療效指標是第52週基線用力肺活量 (FVC) (ml) 改變與基準相比的絕對變化。關鍵次要療效指標是複合指標構成要素的首次發生時間：在試驗期間首次急性 IPF/PPF 惡化、首次因呼吸原因住院或死亡（以先發生者為準）。

 

關於 nerandomilast

Nerandomilast (BI 1015550) 是一種研究性口服優先磷酸二酯酶 4B (PDE4B) 抑制劑，正研究其作為特發性肺纖維化 (IPF) 和漸進性肺纖維化 (PPF) 潛在療法的可能性。此化合物是一種研究性藥物，尚未獲得使用批准。其療效和安全性尚未確立。

Nerandomilast 於 2022 年 2 月獲得美國食品藥物管理局(FDA) 授予的特發性肺纖維化 (IPF) 突破性療法認定。

Nerandomilast 的療效、安全性和耐受度在一項針對 IPF 患者（n=147）的第二期隨機、雙盲、安慰劑對照試驗中進行了研究。主要療效指標是在 12 週治療期內基線用力肺活量 (FVC)（肺功能測量指標）的變化

 

關於特發性肺纖維化 (IPF) 與漸進性肺纖維化 (PPF)

特發性肺纖維化 (IPF) 是較常見的進行性纖維化間質性肺病 (ILD) 之一。IPF 的症狀包括活動時呼吸困難、乾咳和持續性咳嗽、胸部不適、疲勞和虛弱。儘管被認為是“罕見”的疾病，但 IPF 影響了全球約 300 萬人。該疾病主要影響 50 歲以上的患者，且男性患者多於女性。

除了特發性肺纖維化 ( IPF) 之外，某些特定類型的纖維化間質性肺病 (ILD) 患者可能會發展出一種稱為漸進性肺纖維化  (PPF) 的漸進性表現型。在特發性肺纖維化 ( IPF) 以外的間質性肺病 (ILD) 中，漸進性肺纖維化的定義是呼吸症狀惡化、疾病進展的生理證據和疾病進展的放射學影像證據

 

關於百靈佳殷格翰

百靈佳殷格翰是一家全球領先的生物製藥企業，包含人用處方用藥與動物保健兩大業務。作為產業中研發投入最高的公司之一，百靈佳殷格翰致力於研究突破性療法，解決巨大而未被滿足的醫療需求。百靈佳殷格翰自 1885 年成立以來一直是一家獨立的家族企業，始終著眼長遠發展，並將永續發展嵌入價值鏈。公司在全球有超過 5.3 萬名員工，服務逾 130 個市場，致力於打造一個更健康、更永續、更公平的未來。更多詳情，請訪問：www.boehringer-ingelheim.com/tw

「台灣百靈佳殷格翰股份有限公司」與「台灣百靈佳殷格翰動物事業股份有限公司」是百靈佳殷格翰在台子公司，成立於 1975 年，迄今已逾 49 年，目前在台灣北、中、南共設有3個辦公室，員工近 350 人。人類處方用藥及動物保健是公司的核心業務，人類處方用藥持續在呼吸道疾病、癌症、糖尿病、心血管疾病、中風、巴金森病、纖維化疾病等領域幫助更多病患；動物保健方面，我們持續在內外寄生蟲預防、心臟疾病、慢性腎病與疼痛治療上協助伴侶動物與其照顧者，更也在許多疾病上協助經濟動物產業的畜場主人，如豬隻環狀病毒及藍耳病、家禽馬立克病及新城雞病…等。 

 

Boehringer’s nerandomilast meets primary endpoint in pivotal phase-III FIBRONEER™-IPF study

 

  • Topline data from FIBRONEER™-IPF show that the investigational compound nerandomilast met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] at week 52 versus placebo. 
  • The FIBRONEER™-IPF trial is the largest trial in idiopathic pulmonary fibrosis (IPF) conducted to date. Patients were recruited in over 330 sites and in over 30 countries.1,5
  • Full efficacy and safety data from the trial will be presented in the first half of 2025.
  • Boehringer Ingelheim will submit a new drug application for nerandomilast for the treatment of IPF to the US FDA and other Health Authorities worldwide.

 

Boehringer Ingelheim announced today that the FIBRONEER™-IPF trial met its primary endpoint, which was the absolute change from baseline in Forced Vital Capacity [mL] (FVC) at week 52 versus placebo. FVC is a measure of lung function.1

Based on these results, Boehringer Ingelheim will submit the new drug application for nerandomilast for the treatment of IPF to the US Food & Drug Administration (FDA) and other Health Authorities worldwide. The FDA granted Breakthrough Therapy Designation in IPF in 2022.2

“This is the first IPF phase-III-trial in a decade to meet its primary endpoint9,” said Ioannis Sapountzis, Head of Global Therapeutic Areas at Boehringer Ingelheim.  “Today’s announcement represents the next step in our long history in the research of this disease. IPF has a high unmet need for patients, and we are continuously fostering our research activities to develop more options for one of the most common interstitial lung diseases.”

Nerandomilast is an oral, investigational phosphodiesterase 4B (PDE4B) inhibitor and has not been approved for use, therefore safety and efficacy have not been established.3 It is being investigated as part of the FIBRONEER™ global program, which includes two Phase III studies —FIBRONEER™-IPF4 in patients with IPF and FIBRONEER™-ILD5 in people living with Progressive Pulmonary Fibrosis (PPF). 

 

About FIBRONEER™-IPF (NCT05321069)4

A Double Blind, Randomized, Placebo-controlled Trial Evaluating the Efficacy and Safety of nerandomilast Over at Least 52 Weeks in Patients With Idiopathic Pulmonary Fibrosis (IPF).

Primary endpoint: Absolute change from baseline in Forced Vital Capacity (FVC) (mL) at Week 52. 

Key secondary endpoint: 

  • Time to the first occurrence of any of the components of the composite endpoint: time to first acute IPF exacerbation; first hospitalization for respiratory cause; or death (whichever occurs first) over the duration of the trial.

Participants are put into 3 groups randomly, which means by chance. Participants in 2 groups take different doses of nerandomilast as tablets twice a day. Participants in the placebo group take placebo tablets twice a day. Placebo tablets look like nerandomilast tablets but do not contain any medicine.

The trial has been conducted in more than 30 countries,1 and randomized 1177 patients. 

 

About the FIBRONEER™ clinical program4,5

The FIBRONEER™ program includes two Phase III randomized, double-blind, placebo-controlled trials — FIBRONEER™-IPF (NCT05321069) and FIBRONEER™-ILD (NCT05321082) — to investigate the efficacy, safety and tolerability of nerandomilast over at least 52 weeks in patients with IPF and in patients with PPF. 

Patients participating in the FIBRONEER™-IPF trial were treated with either oral nerandomilast at twice-daily doses of 9 mg or 18 mg, or placebo, over at least 52 weeks.1 The 18 mg twice-daily dose of nerandomilast is supported by the results from the Phase II study.1 An additional 9 mg twice-daily dose of nerandomilast was added to evaluate the benefit-risk profile at a lower dose, as well as to provide further dose-response and exposure-response data.1

In both trials, the primary endpoint is the absolute change from baseline in FVC at week 52. The key secondary endpoint is the time to the first occurrence of any of the components of the composite endpoint: time to first acute IPF/PPF exacerbation, first hospitalization for respiratory cause, or death (whichever occurs first) over the duration of the trials.4,5

 

About nerandomilast

Nerandomilast (BI 1015550) is an investigational oral, preferential inhibitor of phosphodiesterase 4B (PDE4B) that is being studied as a potential treatment for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF).2 This compound is an investigational agent and has not been approved for use. The efficacy and safety of this investigational compound has not been established.

Nerandomilast was granted FDA Breakthrough Therapy Designation for the treatment of idiopathic pulmonary fibrosis (IPF) in February 2022.2 

The efficacy, safety, and tolerability of nerandomilast was studied in a Phase II randomized, double-blind, placebo-controlled trial of patients with IPF (n=147). The primary endpoint was a change from baseline in FVC (a measure of lung function) over a 12-week treatment period.3

 

About IPF and PPF

IPF is one of the more common progressive fibrosing interstitial lung diseases (ILD). Symptoms of IPF include breathlessness during activity, a dry and persistent cough, chest discomfort, fatigue and weakness. Although considered “rare,” IPF affects approximately 3 million people worldwide.6,7 The disease primarily affects patients over the age of 50 and affects more men than women.6 

In addition to IPF, patients with certain types of fibrosing ILD may develop a progressive phenotype known as progressive pulmonary fibrosis (PPF). In ILDs other than IPF, progressive pulmonary fibrosis is defined by worsening respiratory symptoms, physiological evidence of disease progression and radiological evidence of disease progression.8 

 

About Boehringer Ingelheim

Boehringer Ingelheim is a biopharmaceutical company active in both human and animal health. As one of the industry’s top investors in Research and Development, the company focuses on developing innovative therapies in areas of high unmet medical need. Independent since its foundation in 1885, Boehringer takes a long-term perspective, embedding sustainability along the entire value chain. More than 53,500 employees serve over 130 markets to build a healthier, more sustainable, and equitable tomorrow.  

 

References:
  1. Richeldi L, et al. (2023) Design of a phase III, double-blind, randomised, placebo-controlled trial of BI 1015550 in patients with idiopathic pulmonary fibrosis (FIBRONEER-IPF). In: BMJ Open Respir Res. 2023; 10(1): e001563.
  2. Boehringer Ingelheim (2022) FDA Grants BI 1015550 Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis. Accessed August 2024. Available at: https://www.boehringer-ingelheim.com/us/human-health/lung-diseases/pulmonary-fibrosis/fda-grants-bi-1015550-breakthrough-therapy.
  3. Richeldi L, et al. (2022) Trial of a Preferential Phosphodiesterase 4B Inhibitor for Idiopathic Pulmonary Fibrosis. In: N Engl J Med 2022;386:2178-2187.