聚焦2025JPM 解锁医药研发新机会.docx

智慧芽

聚焦2025J.P.Morgan解锁医药研发新机会

智慧芽生物医药2025年01月

AlnylamAMGEN

012025JPM主要数据回顾

02重点创新药数据梳理

03具有立项价值的热门药物

04智慧芽数据库加速医药情报收集

05JPM总结和2025展望

山BristolMyersSquibb°山NOVARTIS

Daiichi-Sankyo

PassionforInnovation.CompassionforPatients!“

3

OAlnylam2025Goals

onpa驾nt)raGIMLAARrlO

CombinedNetProductRevenueGuidance

$2,050M-$2,250M

2025

VUTRISIRAN

ATTRAmyloidosis

U.S.FDAApproval

PDUFAdateMarch23,2025

AdditionalGlobalApprovals(Japan,EU)

Q2,Q3

NUCRESIRAN*(ALN-TTRsc04)

ATTRAmyloidosis

InitiatePhase3StudyinATTR-CM

H1

ZILEBESIRAN*

Hypertension

KARDIA-3Phase2Results

H2

InitiatePhase3CVOT

H2

MIVELSIRAN*

CerebralAmyloidAngiopathy

andAlzheimersDisease

InterimPhase1PartBDatainEOAD

H2

InitiatePhase2StudyinAD

H2

ALN-6400*

BleedingDisorders

InitiatePhase2Study

H2

ADDITIONALPROGRAMS

File≥4NewINDs

2025

KEYPARTNER-LED

PROGRAMMILESTONES

FITUSIRAN*(Sanofi)

Hemophilia

U.S.FDAApproval

PDUFAdateMarch28,2025

ELEBSIRAN*(Vir)

ChronicHBV/HDV

InitiatePhase3studyinHDV

H1

Phase2HBVFunctionalCureResults

Q2

CEMDISIRAN*(Regeneron)

Complement-MediatedDiseases

Phase3MGResults

H2

*NotapprovedforanyindicationandconclusionsregardingthesafetyoreffectivenessofthesedrugshavenotbeenestablishedEOAD=EarlyOnsetAlzheimersDisease;MG=MyastheniaGravis

oAlnylam:NucresiranOffersPotentialforBest-in-ClassProfileContinuedInnovationtoSecureFranchiseLeadership

智慧芽

MeanSerumTTRKnockdownEncouragingPhase

MeanSerumTTRKnockdown

EncouragingPhase1Data1

Time(days)

--Placebo(N=12)-0-5mg(N=6)-25mg(N=6)-0-100mg(N=6)

--300mg(N=6)-0-600mg(N=6)--900mg(N=6)

EmergingProfile

√95%TTRKnockdown

√Biannualorannualdosingtooptimizepatientexperience

√Encouragingsafetyprofiletodate

Phase

Phase3studyinitiationinATTR-CMexp