Key findings
- ClinicalTrials.gov lists 237 1 interventional studies of the siRNA, antisense and oral lipid drugs in this track; 90 2 are active and 103 3 are Phase 3, a high share that reflects large outcome programmes.
- 128 4 started between January 2023 and September 2026: 90 5 with a US site and 35 6 with a China site.
- Oral PCSK9 inhibitors (enlicitide, AZD0780) appear in 45 7 of the new studies and inclisiran in 32 8.
- Lp(a)-lowering drugs appear in 14 9 new studies, most of them large Phase 3 programmes.
Momentum: lumpy, because the trials are big
Trial starts went from 6 10 in 2020 to 28 11 in 2021, 22 12 in 2023 and a peak of 51 13 in 2024, then 30 14 in 2025. The pattern follows the launch of a few large programmes rather than a broad wave of new entrants: each Phase 3 programme registers several studies at once.
The United States hosts most studies, with 35 15 of the 2024 starts. China's participation is small but rising, from 4 16 studies in 2021 to 12 17 in 2025. Chinese joint ventures, such as Arrowhead's partner Visirna, now run their own trials.
Cardiometabolic RNA and oral lipid trial starts peaked in 2024
Interventional cardiometabolic RNA and next-generation lipid drug studies by start year; a study with sites in both countries counts in both lines
View the underlying data
| Series | Period | Type | Value |
|---|---|---|---|
| All studies | 2025 | Actual | 30 |
| All studies | 2024 | Actual | 51 |
| All studies | 2023 | Actual | 22 |
| All studies | 2022 | Actual | 32 |
| All studies | 2021 | Actual | 28 |
| All studies | 2020 | Actual | 6 |
| With a US site | 2025 | Actual | 26 |
| With a US site | 2024 | Actual | 35 |
| With a US site | 2023 | Actual | 13 |
| With a US site | 2022 | Actual | 25 |
| With a US site | 2021 | Actual | 22 |
| With a US site | 2020 | Actual | 6 |
| With a China site | 2025 | Actual | 12 |
| With a China site | 2024 | Actual | 12 |
| With a China site | 2023 | Actual | 7 |
| With a China site | 2022 | Actual | 3 |
| With a China site | 2021 | Actual | 4 |
| With a China site | 2020 | Actual | 0 |
Live registry counts retrieved 2026-09-27. Studies registered only outside ClinicalTrials.gov, including many China-only trials, are not counted.
Programmes: pills and twice-yearly injections
LDL cholesterol. Merck's oral PCSK9 inhibitor enlicitide and AstraZeneca's AZD0780 together appear in 45 7 studies started since 2023. Novartis's twice-yearly siRNA inclisiran appears in 32 8. NewAmsterdam's oral CETP inhibitor obicetrapib appears in 13 28.
Lp(a). Amgen's olpasiran, Lilly's lepodisiran and oral muvalaplin, Silence's zerlasiran and Novartis and Ionis's antisense pelacarsen together appear in 14 9 new studies. These are few in number but each is a large outcome trial.
Triglycerides. APOC3 drugs, Ionis's olezarsen and Arrowhead's plozasiran, appear in 7 29 new studies, and ANGPTL3 drugs in 4 30.
Transthyretin. TTR silencers used in amyloid cardiomyopathy and polyneuropathy (vutrisiran, eplontersen, nucresiran) appear in 6 31.
Oral PCSK9 inhibitors and inclisiran lead new trials
Interventional studies started since January 2023, by asset or class; a study can count in more than one bar
View the underlying data
| Category | Source | Type | Value |
|---|---|---|---|
| Oral PCSK9 inhibitors (enlicitide, AZD0780) | ClinicalTrials.gov | Actual | 45 |
| Inclisiran (PCSK9 siRNA) | ClinicalTrials.gov | Actual | 32 |
| Lp(a) lowering (olpasiran, lepodisiran, zerlasiran, pelacarsen, muvalaplin) | ClinicalTrials.gov | Actual | 14 |
| Obicetrapib (CETP) | ClinicalTrials.gov | Actual | 13 |
| APOC3 (olezarsen, plozasiran) | ClinicalTrials.gov | Actual | 7 |
| TTR silencers (vutrisiran, patisiran, eplontersen, nucresiran) | ClinicalTrials.gov | Actual | 6 |
| ANGPTL3 (zodasiran, solbinsiran) | ClinicalTrials.gov | Actual | 4 |
Live registry counts retrieved 2026-09-27. Name and code matching can miss studies registered under other identifiers.
Approvals
- Redemplo (plozasiran, APOC3 siRNA), approved 18 November 2025 (NDA 219947)
- Tryngolza (olezarsen, APOC3 antisense), 19 December 2024 (NDA 218614)
- Wainua (eplontersen, TTR), 21 December 2023 (NDA 217388)
- Amvuttra (vutrisiran, TTR), 13 June 2022 (NDA 215515)
- Leqvio (inclisiran, PCSK9), 22 December 2021 (NDA 214012)
The first APOC3 approvals are in familial chylomicronaemia syndrome, a rare disease. The larger prize is severe hypertriglyceridaemia and cardiovascular risk reduction, which needs further trials.
What it means for founders and R&D teams
- This is a big-company game at the late stage. Almost all studies in this track are industry-sponsored, and 103 3 are Phase 3. Cardiovascular outcome trials enrol very large numbers of patients and are beyond any start-up's budget.
- Early-stage opportunities are in delivery and new liver targets. Longer-acting siRNA, delivery to tissues beyond the liver and new targets such as AGT for hypertension are where small companies can create value, then partner.
- Watch Lp(a) outcome results. The first outcome trials were scheduled to read out from 2026. A positive result would open a very large market. A negative one would reset the whole class.
- One-time editing is the next step. Lilly's acquisition of Verve Therapeutics, completed in July 2025 (Lilly), shows that big pharma is also betting on one-time gene editing of PCSK9 and other lipid genes.
Methodology and limitations
Every trial count is the totalCount of one ClinicalTrials.gov API v2 query, retrieved on 26 September 2026. Each numbered citation opens a record whose source link re-runs the query. The track includes interventional studies naming any of the listed siRNA, antisense or oral lipid drugs, from inclisiran to nucresiran. It is defined by drug rather than by disease, so it also covers transthyretin amyloidosis.
Counts are small and dominated by a few large programmes. Site-country counts overlap and China-only registrations are missing. Approval dates come from Drugs@FDA. Acquisitions and trial timelines are based on company announcements.
Approved, Phase 3 and Phase 2 drugs
The named drugs in this report by their furthest stage: US approval from Drugs@FDA, phase from ClinicalTrials.gov. Sort any column or download CSV; each date and trial number links to its source.
| Drug | Stage | First FDA approval | Latest trial at that stage | Trial start | Trial |
|---|---|---|---|---|---|
| Enlicitide (oral PCSK9) | Approved | 15 Jul 2026 Lipfendra · NDA 220848 | NCT07216482 Phase 3 · Active, not recruiting | 11 Nov 2025 | A Clinical Study of Enlicitide in Participants With High Cholesterol (MK-0616-037) |
| Eplontersen (TTR) | Approved | 21 Dec 2023 Wainua · NDA 217388 | NCT06194825 Phase 3 · Active, not recruiting | 1 Dec 2023 | EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy |
| Inclisiran (PCSK9 siRNA) | Approved | 22 Dec 2021 Leqvio · NDA 214012 | NCT07102628 Phase 3 · Recruiting | 3 Oct 2025 | Evaluation of Efficacy and Safety of Early in Hospital Initiation of Inclisiran Treatment in Patients With Acute Coronary Syndromes |
| Lerodalcibep (PCSK9) | Approved | 12 Dec 2025 Lerochol · BLA 761427 | NCT07102511 Phase 3 · Enrolling by invitation | 20 Jul 2026 | Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children 6 to 17 Years, With Heterozygous FH |
| Olezarsen (APOC3) | Approved | 19 Dec 2024 Tryngolza (autoinjector) · NDA 218614 | NCT07727538 Phase 3 · Recruiting | 31 Jul 2026 | A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants |
| Patisiran (TTR) | Approved | 10 Aug 2018 Onpattro · NDA 210922 | NCT03997383 Phase 3 · Completed | 4 Sep 2019 | APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy) |
| Plozasiran (APOC3) | Approved | 18 Nov 2025 Redemplo · NDA 219947 | NCT06880770 Phase 3 · Recruiting | 24 Apr 2025 | Study of Plozasiran in Adults With Severe Hypertriglyceridemia at Risk of Acute Pancreatitis |
| Vutrisiran (TTR) | Approved | 13 Jun 2022 Amvuttra · NDA 215515 | NCT06679946 Phase 3 · Enrolling by invitation | 3 Dec 2024 | A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy |
| AZD0780 (oral PCSK9) | Phase 3 | Not checked: development code, no approved name | NCT07000136 Phase 3 · Active, not recruiting | 10 Jun 2025 | A Phase III Study to Assess the Effect of AZD0780 on LDL-C in Patients With HeFH |
| Lepodisiran (Lp(a)) | Phase 3 | Not FDA approved | NCT07613294 Phase 3 · Recruiting | 1 Jun 2026 | A Study to See if Lepodisiran Can Reduce Plaque in Coronary Arteries of Adults With Elevated Lp(a) Who Have Had Heart Events or Are at High Risk |
| Muvalaplin (oral Lp(a)) | Phase 3 | Not FDA approved | NCT07157774 Phase 3 · Recruiting | 2 Sep 2025 | Assessing the Impact of Muvalaplin on Major Cardiovascular Events in Adults With Elevated Lipoprotein(a) |
| Nucresiran (TTR) | Phase 3 | Not FDA approved | NCT07223203 Phase 3 · Recruiting | 3 Feb 2026 | TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy |
| Obicetrapib (CETP) | Phase 3 | Not FDA approved | NCT07614958 Phase 3 · Recruiting | 6 May 2026 | Phase 3 Clinical Study Evaluating Efficacy, Safety, and Tolerability of Obicetrapib Vs Bempedoic Acid to Treat Dyslipidemia in Adults With Primary Non-familial Hypercholesterolemia or Mixed Dyslipidemia at High to Very High Cardiovascular Risk, During a Treatment Period of Approximately 84 Days |
| Olpasiran (Lp(a)) | Phase 3 | Not FDA approved | NCT07293260 Phase 3 · Recruiting | 23 Mar 2026 | Olpasiran Trials of Cardiovascular Events and Lipoprotein(a) Reduction - Coronary Computed Tomography Angiography Trial |
| Pelacarsen (Lp(a)) | Phase 3 | Not FDA approved | NCT07625306 Phase 3 · Not yet recruiting | 31 Aug 2026 | Lp(a) Lowering Study of Pelacarsen (TQJ230) in Patients in the US With Elevated Lp(a) and Recent ACS (STEMI/NSTEMI) - Lp(a)FRONTIERS PEARL |
| Zilebesiran (angiotensinogen) | Phase 3 | Not FDA approved | NCT07181109 Phase 3 · Recruiting | 22 Sep 2025 | Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease |
| Zodasiran (ANGPTL3) | Phase 3 | Not FDA approved | NCT07473843 Phase 3 · Not yet recruiting | Oct 2026 | Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia |
| Solbinsiran (ANGPTL3) | Phase 2 | Not FDA approved | NCT07269210 Phase 2 · Recruiting | 8 Dec 2025 | A Study of Solbinsiran (LY3561774) in Participants With Severe Hypertriglyceridemia |
| Zerlasiran (Lp(a)) | Phase 2 | Not FDA approved | NCT05537571 Phase 2 · Completed | 13 Dec 2022 | Evaluate SLN360 in Participants With Elevated Lipoprotein(a) at High Risk of Atherosclerotic Cardiovascular Disease Events |
How this report is checked
Every figure is a count from ClinicalTrials.gov, the US National Library of Medicine's trial registry. Each links to a StatOrigin record that keeps the exact query, so anyone can run it again; how the counts are made and what they leave out is set out under Methodology and limitations above.
The development-stage table
Checked on 4 October 2026. A drug's US approval is the original approval of its first new drug application or biologics licence application in Drugs@FDA, read through the FDA's openFDA service, with the FDA approval letter where Drugs@FDA links one. Cell and gene therapies are licensed by a different FDA centre (CBER) and are not in Drugs@FDA; an approval for one of them is shown only where it was checked by hand against the FDA's product page. Approvals outside the United States — by the European Medicines Agency or China's NMPA, for example — are not shown. The phase is the highest phase among the drug's interventional ClinicalTrials.gov records. The trial shown is the most recently started one at that phase whose title names the drug, or, where none does, the most recently started one that lists it.
Writing and review
Written by StatOrigin editorial with AI assistance. Each count is checked automatically against the registry's own response before it is published. No named clinical reviewer has checked this report yet. Report an error through the corrections page.
Not medical advice
This report describes research activity and regulatory status. It is not medical advice and does not say whether any medicine is safe or effective for any person. Trial activity is not evidence that a drug works. Decisions about treatment belong with a qualified health professional; for what a medicine is approved to treat, read its current FDA label.
Data behind this report
The industry hubs and indicator series these figures come from. Each page carries the full table, every source and the records this report does not quote.
Industry hubs
Indicator series
- Cardiometabolic RNA and next-generation lipid drug trial starts 21
- Active cardiometabolic RNA and next-generation lipid drug trials 1
- Phase 3 cardiometabolic RNA and next-generation lipid drug trials 1
- Registered interventional cardiometabolic RNA and next-generation lipid drug trials 1
- Trial starts since 2023: ANGPTL3 (zodasiran, solbinsiran) 1
- Trial starts since 2023: APOC3 (olezarsen, plozasiran) 1
- Trial starts since 2023: Inclisiran (PCSK9 siRNA) 1
- Trial starts since 2023: Lp(a) lowering (olpasiran, lepodisiran, zerlasiran, pelacarsen, muvalaplin) 1
- Trial starts since 2023: Obicetrapib (CETP) 1
- Trial starts since 2023: Oral PCSK9 inhibitors (enlicitide, AZD0780) 1
- Trial starts since 2023: TTR silencers (vutrisiran, patisiran, eplontersen, nucresiran) 1
Sources
Every figure in this report is a published StatOrigin record. The table lists the 31 cited statistics. Sort any column or download CSV. Open a record for APA, MLA, Chicago or BibTeX.
Cite this report
StatOrigin. (2026). Cardiovascular RNA drugs and oral lipid lowering: Lp(a), APOC3 and oral PCSK9. https://statorigin.org/reports/cardiovascular-rna-lipid-drug-pipeline-2026
Prefer citing the underlying statistic when you only need one figure. Published 26 September 2026 · updated 27 September 2026. Data licence: CC BY 4.0.