Myopathy

·MeSH: Myofibrillar Myopathy

66 drugs in development·35 companies·1 clinical trial

Drug Pipeline by Phase

Approved(5)

Drug	Company
Amvuttra	Royalty Pharma plc (RPRX)
AMVUTTRA	ALNYLAM PHARMACEUTICALS, INC. (ALNY)
attruby	BridgeBio Pharma, Inc. (BBIO)
myqorzo	CYTOKINETICS INC (CYTK)
supplemental new drug	ALNYLAM PHARMACEUTICALS, INC. (ALNY)

Phase 3(24)

Drug	Company
Aficamten	CYTOKINETICS INC (CYTK)
aficamten (5 mg	CYTOKINETICS INC (CYTK)
alxn2060	ALEXION PHARMACEUTICALS, INC. (ALXN)
alxn2220	Hepion Pharmaceuticals, Inc. (CTRVP)
alxn2350	ALEXION PHARMACEUTICALS, INC. (ALXN)
AOC 1086	Avidity Biosciences, Inc. (RNAM)
arry-797	ARRAY BIOPHARMA INC (ARRY)
AT-001 (caficrestat)	Applied Therapeutics, Inc. (APLT)
azd4063	ASTRAZENECA PLC (AZN)
ck-3773274	CYTOKINETICS INC (CYTK)
duchenne muscular dystrophy"	CAPRICOR THERAPEUTICS, INC. (CAPR)
eplontersen	ASTRAZENECA PLC (AZN)
eplontersen	IONIS PHARMACEUTICALS INC (IONS)
Metoprolol succinate	CYTOKINETICS INC (CYTK)
metoprolol succinate (50 mg	CYTOKINETICS INC (CYTK)
nnc6019-0001	Novo Nordisk A/S (NVO)
NNC6019-0001	PROTHENA CORP PUBLIC LTD CO (PRTA)
Nucresiran	ALNYLAM PHARMACEUTICALS, INC. (ALNY)
Patisiran	ALNYLAM PHARMACEUTICALS, INC. (ALNY)
SGT-601	Solid Biosciences Inc. (SLDB)
SGT-701	Solid Biosciences Inc. (SLDB)
Trientine	UNITED THERAPEUTICS Corp (UTHR)
vclas cryoablation system	Adagio Medical Holdings Inc (ADGM)
Vutrisiran	ALNYLAM PHARMACEUTICALS, INC. (ALNY)

Phase 2(9)

Drug	Company
alxn2220	ASTRAZENECA PLC (AZN)
danicamtiv	MyoKardia, Inc. (MYOK)
Dyscorban®	CUMBERLAND PHARMACEUTICALS INC (CPIX)
ifetroban	CUMBERLAND PHARMACEUTICALS INC (CPIX)
myk-224	BRISTOL MYERS SQUIBB CO (BMY)
myk-491	MyoKardia, Inc. (MYOK)
Ninerafaxstat 200mg MR	ARS Pharmaceuticals, Inc. (SPRY)
NNC6019	PROTHENA CORP PUBLIC LTD CO (PRTA)
Vasculan	CUMBERLAND PHARMACEUTICALS INC (CPIX)

Phase 1(7)

Drug	Company
4D-310	4D Molecular Therapeutics, Inc. (FDMT)
coramitug	Novo Nordisk A/S (NVO)
EDG-7500	Edgewise Therapeutics, Inc. (EWTX)
ion826	BICYCLE THERAPEUTICS PLC (BCYC)
NTLA-2001	Intellia Therapeutics, Inc. (NTLA)
rese-cel (resecabtagene autoleucel)	Cabaletta Bio, Inc. (CABA)
rp-a601	Rocket Pharmaceuticals Inc. (RCKT)

Preclinical(7)

Drug	Company
act-1	MyoKardia, Inc. (MYOK)
AVB-401	Solid Biosciences Inc. (SLDB)
AVB-501	Solid Biosciences Inc. (SLDB)
AVB-601	Solid Biosciences Inc. (SLDB)
mavodelpar	OnKure Therapeutics, Inc. (OKUR)
myk-224	MyoKardia, Inc. (MYOK)
ren-001	Renovacor, Inc. (RCOR)

Unknown(2)

Drug	Company
approved hypertrophic cardiomyopathy drug treatments	BRISTOL MYERS SQUIBB CO (BMY)
laromestrocel	Longeveron Inc. (LGVN)

Early Phase 1(1)

Drug	Company
Sotagliflozin	ENANTA PHARMACEUTICALS INC (ENTA)

Phase 1/Phase 2(7)

Drug	Company
asp2957	Sio Gene Therapies Inc. (SIOX)
lx2020	Lexeo Therapeutics, Inc. (LXEO)
lx2021	Lexeo Therapeutics, Inc. (LXEO)
lx2022	Lexeo Therapeutics, Inc. (LXEO)
tn-201	Tenaya Therapeutics, Inc. (TNYA)
tn-401	Tenaya Therapeutics, Inc. (TNYA)
ux016	Ultragenyx Pharmaceutical Inc. (RARE)

Phase 2/Phase 3(1)

Drug	Company
caba-201 following preconditioning with fludarabine	Cabaletta Bio, Inc. (CABA)

Phase 4(3)

Drug	Company
Mavacamten	BRISTOL MYERS SQUIBB CO (BMY)
MavacamtenRights	BRISTOL MYERS SQUIBB CO (BMY)
non-mavacamten symptomatic ohcm therapy	BRISTOL MYERS SQUIBB CO (BMY)

BioSniper tracks 66 drugs in development for Myopathy across 35 companies.

There are 1 clinical trial targeting Myopathy tracked by BioSniper.

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