中风的演变格局：关键管线和临床试验见解

The Evolving Landscapeof Stroke: Key Pipeline IQVIA Pipeline Link/Trial Link — December 2025 DR. HARI KRISHNA MYLAPPAGARI,Senior Insights Associate, IQVIA Pipeline Link/Trial LinkSHIKHA YADAV,Insights Associate, IQVIA Pipeline Link/Trial Link Table of contents Introduction1Treatment options for stroke3Stroke pipeline insights3Novel therapies in early stage5Stroke clinical trial landscape6Conclusions7References7About the authors8 Introduction Stroke, also referred to as brain attack orcerebrovascular accident, is a medical condition statistics, in 2019 there were 12.2 million new casesof stroke, 6.6 million deaths and 143 million years of life lost prematurely or lived with disability.4Accordingto the US Centers for Disease Control and Prevention or emergency caused by blockage of blood flow toor sudden bleeding in the brain. This interruption deprives brain tissue, the body’s command center,of the nutrients and oxygen essential for normal function.1Stroke remains the second leading causeof death and the third leading cause of death anddisability combined among non-communicabledisorders.2The signs and symptoms of stroke can Despite decades of research, Genentech andBoehringer Ingelheim’s alteplase (Activase) was theonly FDA-approved tissue plasminogen activator (tPA)indicated for acute ischemic stroke (AIS) until March include weakness or numbness in the face, arm, This article provides an update on the currenttreatment options for stroke, outlining the latest Emerging therapeutic strategies for stroke increasingly focus on novel Treatment options for stroke Stroke pipeline insights OVERVIEW OF PHASE-WISE DISTRIBUTION, Immediate intervention is critical in strokemanagement, yet only about 0.5% of eligible patientsreceive thrombolysis due to the narrow 3 to 4.5htreatment window. Stroke pharmacotherapy includesreperfusion therapy, neuroprotective agents, and As of November 2025, IQVIA Pipeline Link andTrial Link listed 77 unique programs in developmentfor stroke. Figure 1 illustrates distribution of productsby the highest phase considering the indication in a The pipeline is led by small molecules (51%) followedby mesenchymal stem cell therapies (10%) (asillustrated in Figure 2), driven by their proven efficacy, Current treatments have limited windows and donot repair brain damage, highlighting the need Current treatments have limited windows, highlighting the need for newtherapies to extend treatment window and improve recovery. factors (F11A, F10A, F2R) for anticoagulation, DLG4(PSD-95) and GRIN2B for mitigating excitotoxicity,free radical pathways for reducing oxidative stress,and SERPINF2, VWF and MPO that addressthrombolysis, thrombosis and inflammation. medicine, with stem cell therapies gaining momentumfor their dual capability in neuroprotection andregeneration. At the same time, monoclonal Emerging therapeutic strategies for stroke increasinglyfocus on novel molecular targets implicated in SELECTED LATE-STAGE PROGRAMS IN DEVELOPMENT to placebo. Healios is planning to file a conditional andtime-limited approval application based on secondaryendpoints with statistically significant results in Japan. Healios’ Invimestrocel (MultiStem; HLCM051) isthe most advanced program, expected to come tomarket in Q1 2026. Invimestrocel is a multipotent Yiling Pharmaceutical’s XY03-EA (autophagy inhibitor)and Neurodawn’s Y-3 (GABRA2 agonist; MPO, DLG4and NOS1 inhibitor) have shown promising preclinical immunosuppressant drugs and is expected to providea therapeutic benefit if given within 36h of stroke. ThePhase II/III (TREASURE; NCT02961504) and Phase III(MASTERS-2; NCT03545607) trials did not meet theprimary endpoint of proportion of patients achieving GNT Pharma’s nelonemdaz, Corxel’s lemuplamorand Diamedica’s rinvecalinase alfa (DM199) showedpromising Phase II data in the late treatment Strong industry shift toward precision medicine, with stem cell therapiesgaining momentum for their dual capability in neuroprotection Novel therapies in early stage Advances in understanding stroke pathophysiology have improved acute treatments, but long-term recoveryremains limited. The risk of brain hemorrhage associated with thrombolytic agents has hindered the development Scp776 Scp776, an insulin-like growth factor 1 fusion protein, developed by Silver Creek Pharmaceuticals, is currentlyin Phase II development for AIS. It works by protecting neurons from excitotoxicity, hypoglycemic damage,oxidative stress, inflammation, and mitigating apoptosis. In the ARPEGGIO (NCT05585606) Phase II trial,Scp776 improved NIH Stroke Scale (NIHSS) by 2.26 points compared to placebo (p = 0.066) and had a 15% ODATROLTIDE Odatroltide (LT3001), a peptide-small molecule drug conjugate, developed by Lumosa Therapeutics and ShanghaiPharmaceutical holding, is in Phase II development. It is a dual functional drug that consist of a peptide whichinduces reperfusion, restoring occluded blood flow, and a small molecu