Clean Phase 3 win in post-bariatric hypoglycemia positions avexitide for NDA filing; approval converts a no-revenue rebuilding story into a commercial franchise
- avexitide FiledPost-Bariatric Hypoglycemia · GLP-1 receptor antagonist
- avexitide FiledCongenital Hyperinsulinism · GLP-1 receptor antagonist
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Ryoncil's $115M launch year funds the rexlemestrocel-L CLBP Phase 3 bet; the readout determines whether Mesoblast is a commercial platform or a one-product story
- Revascor FiledEnd-Stage Ischemic Heart Failure With Reduced Ejection Fraction · mesenchymal stem cell therapy
- Rexlemestrocel-L FiledEnd-Stage Heart Failure With Reduced Ejection Fraction · mesenchymal stem cell therapy
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REDEMPLO approved in five markets; sHTG label expansion with pancreatitis claim vs. approved olezarsen is next value inflection
- REDEMPLO FiledSevere Hypertriglyceridemia · APOC3 siRNA
- plozasiran Phase 3Severe Hypertriglyceridemia · RNAi therapeutic / siRNA
- olpasiran Phase 3Atherosclerotic Cardiovascular Disease · RNAi therapeutic / siRNA
- zodasiran Phase 3Homozygous Familial Hypercholesterolemia · RNAi therapeutic / siRNA
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NDA filed on a missed primary endpoint; FDA acceptance and then approvability of marginal Phase 3 data are the two sequential tests
- Seralutinib FiledPulmonary Arterial Hypertension · PDGFR/FGFR/CSF1R kinase inhibitor
- Seralutinib Phase 3Pulmonary Hypertension Associated With Interstitial Lung Disease · PDGFR/FGFR/CSF1R kinase inhibitor
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vCLAS pivotal data support the filed PMA, but the strategic review makes financing or a transaction the immediate survival test
- vCLAS Ventricular Ablation System FiledVentricular Tachycardia · Ultra-Low Temperature Cryoablation (ULTA) catheter ablation
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Ionis has three approved drugs and two fresh Phase 3 wins, but the hepatitis B functional-cure PDUFA is the single event that rerates the stock
- Eplontersen Phase 3Transthyretin Amyloid Cardiomyopathy · TTR antisense oligonucleotide
- Olezarsen Phase 3Familial Chylomicronemia Syndrome · APOC3 antisense oligonucleotide
- Pelacarsen Phase 3Lp(A)-Driven Cardiovascular Disease · Lp(a) antisense oligonucleotide
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MYQORZO approved in US, EU, and UK with Phase 3 wins in two HCM subtypes; commercial ramp and BMS patent litigation are the tests
- aficamten Phase 3Non-Obstructive Hypertrophic Cardiomyopathy · cardiac myosin inhibitor
- omecamtiv mecarbil Phase 3Heart Failure With Reduced Ejection Fraction · cardiac myosin activator
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Dual GLP-1/glucagon agonist with BTD in MASH and Phase 2 AUD win, but approval hinges on 2029 Phase 3 biopsy readout
- pemvidutide Phase 3Metabolic Dysfunction-Associated Steatohepatitis · balanced glucagon/GLP-1 dual receptor agonist
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Four late-stage programs approaching simultaneous approval decisions; encaleret's May 2027 PDUFA with clean Phase 3 data is the first test of whether BridgeBio can convert its pipeline into a multi-franchise rare-disease company
- Acoramidis Phase 3Transthyretin Amyloid Cardiomyopathy · transthyretin stabilizer
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Endoscopic duodenal procedure seeks to solve GLP-1 rebound obesity; pivotal data and financing both arrive before early 2027
- Revita Phase 3Obesity · Duodenal mucosal resurfacing device
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IKT-001 enters Phase 3 in PAH fully funded to readout, but efficacy in humans is unproven; enrollment pace and Part A PVR signal are the first tests
- IKT-001 Phase 3Pulmonary Arterial Hypertension · c-Abl/PDGFR kinase inhibitor
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NATiV3 Phase 3 readout is the single event that determines whether Inventiva's pan-PPAR bet in MASH justifies its reconstructed balance sheet
- lanifibranor Phase 3Metabolic Dysfunction-Associated Steatohepatitis · pan-PPAR agonist
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Profitable pericarditis franchise funds KPL-387's Phase 3 pivotal trial; approval hinges on randomized withdrawal recurrence data
- KPL-387 Phase 3Recurrent Pericarditis · IL-1 inhibitor
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CARDAMYST approved and launching, but $28M quarterly burn against H2 2027 runway forces commercial ramp to outpace the clock
- etripamil Phase 3Atrial Fibrillation With Rapid Ventricular Rate · calcium channel blocker
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EU approval of Ubeslo and Evlarco via Menarini; PREVAIL CVOT interim now determines whether obicetrapib reaches blockbuster CV outcomes labeling
- Obicetrapib Phase 3Atherosclerotic Cardiovascular Disease · CETP inhibitor
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FDA independently reviewing non-primary CGM data after Phase 3 miss; upLIFT's 7/8 interim response rate adds a second pathway but neither is confirmed
- ersodetug Phase 3Congenital Hyperinsulinism · anti-insulin receptor monoclonal antibody
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First-ever approved dermatomyositis therapy just launched; commercial uptake now determines whether a multi-indication TYK2/JAK1 platform compounds or stalls
- Mosliciguat Phase 3Pulmonary Hypertension · sGC activator
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Divesiran's 88% Phase 2 response rate in polycythemia vera positions Silence for Phase 3, but ~$72M cash and no partner fund the pivotal trial
- zerlasiran Phase 3Cardiovascular Disease · Lp(a)-targeting siRNA
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Oral GLP-1 agonist with Phase 2 efficacy rivaling injectables enters Phase 3, but the oral obesity field is crowding fast
- aleniglipron Phase 3Obesity Or Overweight · GLP-1 receptor agonist
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LEVEL Phase 3 miss forces enriched-population retry; FDA Type C meeting will determine whether a subgroup rescue is registrationally viable
- TNX-103 Phase 3Pulmonary Hypertension With Heart Failure With Preserved Ejection Fraction · calcium sensitizer and phosphodiesterase inhibitor
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Subcutaneous VK2735 Phase 3 fully enrolled in obesity; registrational data will confirm or deny the bull case against entrenched GLP-1 rivals
- VK2735 Phase 3Obesity · dual GLP-1 and GIP receptor agonist
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Cadisegliatin's CATT1 Phase 3 readout will determine whether a BTD-designated insulin adjunct can redefine T1D management
- cadisegliatin Phase 3Type 1 Diabetes · Glucokinase activator
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