A PHASE 1, RANDOMIZED, OPEN-LABEL, SINGLE DOSE STUDY TO INVESTIGATE THE PHARMACOKINETICS, PHARMACODYNAMICS, SAFETY AND TOLERABILITY OF VUPANORSEN ADMINISTERED SUBCUTANEOUSLY TO HEALTHY CHINESE ADULTS

This is a Phase 1, randomized, parallel-cohort, open-label study to characterize the pharmacokinetics, pharmacodynamics, safety and tolerability of vupanorsen following 80 mg and 160 mg single subcutaneous dose in healthy Chinese adults with elevated fasting triglyceride.

Start Date17 Jun 2021

Sponsor / Collaborator

Pfizer Inc.

CTR20210981 / CompletedPhase 1

一项在中国健康成人中评估VUPANORSEN单剂量皮下注射给药的药代动力学、药效学、安全性和耐受性的随机、开放、I 期临床研究

[Translation] A randomized, open-label, Phase I clinical study evaluating the pharmacokinetics, pharmacodynamics, safety, and tolerability of a single subcutaneous dose of VUPANORSEN in healthy Chinese adults

主要目的：在中国健康成人中评估Vupanorsen 80 mg和160 mg单剂量皮下注射给药后的药代动力学特征次要目的：在中国健康成人中评估Vupanorsen 80 mg和160 mg单剂量皮下注射给药后的安全性和耐受性。在中国健康成人中评估Vupanorsen 80 mg和160 mg单剂量皮下注射给药后的药效学。

[Translation]

Primary objective: To evaluate the pharmacokinetic characteristics of Vupanorsen 80 mg and 160 mg after single subcutaneous injection in healthy Chinese adults Secondary objective: To evaluate the safety and tolerability of Vupanorsen 80 mg and 160 mg after single subcutaneous injection in healthy Chinese adults. To evaluate the pharmacodynamics of Vupanorsen 80 mg and 160 mg after single subcutaneous injection in healthy Chinese adults.

Start Date17 Jun 2021

Sponsor / Collaborator

Pfizer Inc.

[+2]

NCT04516291 / CompletedPhase 2

A Phase 2b Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Dose-Ranging Study to Assess the Efficacy, Safety, and Tolerability of Vupanorsen (PF-07285557) in Statin-Treated Participants With Dyslipidemia

This is a multicenter, Phase 2b, double-blind, placebo-controlled, parallel group study to provide data on efficacy, safety, tolerability, and pharmacokinetics (PK) of PF-07285557 (hereafter, vupanorsen) administered subcutaneously (SC) at various doses and regimens in participants with dyslipidemia, defined in this study as participants with elevated non-HDL-C and TG who are receiving a stable dose of a statin.
This study is also known as TaRgeting ANGPTL3 with an aNtiSense oLigonucleotide in AdulTs with dyslipidEmia (TRANSLATE-TIMI 70).

Start Date28 Sep 2020

Sponsor / Collaborator

Pfizer Inc.

[+1]

100 Clinical Results associated with Vupanorsen

100 Translational Medicine associated with Vupanorsen

100 Patents (Medical) associated with Vupanorsen

Literatures (Medical) associated with Vupanorsen

01 Jun 2024·DRUGS IN R&D

A Randomized, Open-Label, Phase I, Single-Dose Study of Antisense Oligonucleotide, Vupanorsen, in Chinese Adults with Elevated Triglycerides

Article

Author: Terra, Steven G ; Bramson, Candace ; Han, Xiaoran ; Wu, Xiaojie ; Zhang, Chunye ; Wang, Jeffrey ; Yu, Jicheng ; Liu, Jing ; Ge, Beikang ; Kalluru, Hindu ; Mao, Xiaomeng

BACKGROUND:

Vupanorsen is a GalNAc₃-conjugated antisense oligonucleotide targeting angiopoietin-like 3 (ANGPTL3) mRNA shown to reduce atherogenic lipoproteins in individuals with dyslipidemia.

OBJECTIVES:

The aim of this study was to satisfy Chinese regulatory requirements and support ethnic sensitivity assessment by evaluating pharmacokinetics (PK), pharmacodynamics (PD), and safety of vupanorsen in healthy Chinese adults with elevated triglycerides (TG).

METHODS:

In this phase I, parallel-cohort, open-label study, 18 Chinese adults with elevated fasting TG (≥ 90 mg/dL) were randomized 1:1 to receive a single subcutaneous dose of vupanorsen 80 mg or 160 mg. PK parameters, PD markers (including ANGPTL3, TG, non-high-density lipoprotein cholesterol [non-HDL-C]), and safety were assessed.

RESULTS:

Absorption of vupanorsen was rapid (median time to maximum concentration [T_max]: 2.0 h for both doses), followed by a multiphasic decline (mean terminal half-life 475.9 [80 mg] and 465.2 h [160 mg]). Exposure (area under curve [AUC] and maximum plasma concentration [C_max]) generally increased in a greater than dose-proportional manner from 80 mg to 160 mg. Time-dependent reductions in ANGPTL3 and lipid parameters were observed. Mean percentage change from baseline for the 80-mg and 160-mg doses, respectively, were - 59.7% and - 69.5% for ANGPTL3, - 41.9% and - 52.5% for TG, and - 23.2% and - 25.4% for non-HDL-C. No serious or severe adverse events (AEs), deaths, or discontinuations due to AEs were reported. Three participants experienced treatment-related AEs; all were mild and resolved by end of study.

CONCLUSIONS:

This study provided the first clinical vupanorsen data in China. In Chinese participants with elevated TG, PK and PD parameters were consistent with those reported previously in non-Chinese participants, including in Japanese individuals. No safety concerns were noted.

TRIAL REGISTRATION:

ClinicalTrials.gov identifier: NCT04916795.

01 Mar 2024·Journal of clinical lipidology

Hepatic fat changes with antisense oligonucleotide therapy targeting ANGPTL3

Article

Author: Jevne, Alexandra ; Bergmark, Brian A ; Zimerman, Andre ; Curto, Madelyn ; Marston, Nicholas A ; Terra, Steven G ; Ramos, Vesper ; Kuder, Julia F ; Ran, Xinhui ; Verma, Subodh ; Wojakowski, Wojtek ; Sabatine, Marc S ; Park, Jeong-Gun ; Murphy, Sabina A ; Bramson, Candace R ; Wiviott, Stephen D

BACKGROUND:

Angiopoietin-like protein 3 (ANGPTL3) is a novel therapeutic target for hyperlipidemia. Vupanorsen, an antisense oligonucleotide targeting ANGPTL3, reduced triglycerides up to 57% in a phase 2b trial, but caused dose-dependent increases in hepatic fat fraction (HFF).

OBJECTIVE:

To determine the degree of HFF progression with escalating doses of vupanorsen, differential HFF increases in key patient subgroups, and the correlation between changes in HFF and liver enzymes.

METHODS:

TRANSLATE-TIMI 70 was a randomized, placebo-controlled trial testing 7 dosing regimens of vupanorsen in 286 adults with hyperlipidemia. A total of 227 patients had HFF measured at baseline and 24 weeks and were included in this analysis.

RESULTS:

The median HFF at baseline was 8.5%. Vupanorsen led to dose-dependent relative increases in HFF of up to 76% at 24 weeks (p < 0.001), corresponding to an absolute increase of up to 7.0% at the highest dose (p < 0.001). Increases in HFF were numerically greater in patients who had elevated baseline HFF, body mass index, triglycerides, or diabetes. Vupanorsen also increased liver enzymes in a dose-dependent manner, and changes in HFF were moderately positively correlated with changes in aspartate transaminase (AST) (rho = 0.49, p < 0.001) and alanine transaminase (ALT) (rho = 0.50, p < 0.001).

CONCLUSION:

Vupanorsen, an inhibitor of ANGPTL3 protein synthesis, caused dose-dependent increases in HFF. Increases in HFF were only moderately correlated with elevations in AST and ALT, suggesting that liver enzymes are an imperfect indicator to detect increases in hepatic fat. These results highlight the need to monitor HFF in clinical trials of therapies targeting intracellular ANGPTL3 inhibition, especially those that are targeted to the liver.

07 Nov 2023·Circulation

ANGPTL3 Deficiency and Risk of Hepatic Steatosis

Article

Author: Nicolucci, Antonio ; Burks, Kendall H. ; Bini, Simone ; Catalano, Carlo ; Maranghi, Marianna ; D’Erasmo, Laura ; Arca, Marcello ; Zheng, Jie ; Kang, Chul Joo ; Labbadia, Giancarlo ; Stitziel, Nathan O. ; Ceci, Fabrizio ; Davidson, Nicholas O. ; Fraum, Tyler J. ; Minicocci, Ilenia ; Neufeld, Thomas ; Di Martino, Michele ; Pigna, Giovanni ; Lucisano, Giuseppe ; Di Costanzo, Alessia ; Fierro, Davide ; Montali, Anna

BACKGROUND::

ANGPTL3 (angiopoietin-like 3) is a therapeutic target for reducing plasma levels of triglycerides and low-density lipoprotein cholesterol. A recent trial with vupanorsen, an antisense oligonucleotide targeting hepatic production of ANGPTL3, reported a dose-dependent increase in hepatic fat. It is unclear whether this adverse effect is due to an on-target effect of inhibiting hepatic ANGPTL3.

METHODS::

We recruited participants with ANGPTL3 deficiency related to ANGPTL3 loss-of-function (LoF) mutations, along with wild-type (WT) participants from 2 previously characterized cohorts located in Campodimele, Italy, and St. Louis, MO. Magnetic resonance spectroscopy and magnetic resonance proton density fat fraction were performed to measure hepatic fat fraction and the distribution of extrahepatic fat. To estimate the causal relationship between ANGPTL3 and hepatic fat, we generated a genetic instrument of plasma ANGPTL3 levels as a surrogate for hepatic protein synthesis and performed Mendelian randomization analyses with hepatic fat in the UK Biobank study.

RESULTS::

We recruited participants with complete (n=6) or partial (n=32) ANGPTL3 deficiency related to ANGPTL3 LoF mutations, as well as WT participants (n=92) without LoF mutations. Participants with ANGPTL3 deficiency exhibited significantly lower total cholesterol (complete deficiency, 78.5 mg/dL; partial deficiency, 172 mg/dL; WT, 188 mg/dL; P <0.05 for both deficiency groups compared with WT), along with plasma triglycerides (complete deficiency, 26 mg/dL; partial deficiency, 79 mg/dL; WT, 88 mg/dL; P <0.05 for both deficiency groups compared with WT) without any significant difference in hepatic fat (complete deficiency, 9.8%; partial deficiency, 10.1%; WT, 9.9%; P >0.05 for both deficiency groups compared with WT) or severity of hepatic steatosis as assessed by magnetic resonance imaging. In addition, ANGPTL3 deficiency did not alter the distribution of extrahepatic fat. Results from Mendelian randomization analyses in 36 703 participants from the UK Biobank demonstrated that genetically determined ANGPTL3 plasma protein levels were causally associated with low-density lipoprotein cholesterol ( P =1.7×10 -17 ) and triglycerides ( P =3.2×10 -18 ) but not with hepatic fat ( P =0.22).

CONCLUSIONS::

ANGPTL3 deficiency related to LoF mutations in ANGPTL3 , as well as genetically determined reduction of plasma ANGPTL3 levels, is not associated with hepatic steatosis. Therapeutic approaches to inhibit ANGPTL3 production in hepatocytes are not necessarily expected to result in the increased risk for hepatic steatosis that was observed with vupanorsen.

News (Medical) associated with Vupanorsen

24 Nov 2021

·www.biospace.com

Pfizer-licensed Cardiovascular Drug Reduces Triglycerides in Phase II

Dan Kitwood/Getty Images A cardiovascular drug Pfizer licensed from Akcea Therapeutics in 2019 hit the mark in a Phase IIb dose-ranging study in patients with elevated non-HDL-C and triglycerides. Vupanorsen is an investigational antisense therapy that is being developed for cardiovascular risk reduction and severe hypertriglyceridemia, an abnormal triglyceride concentration in the blood. This morning, Ionis Pharmaceuticals, which acquired Akcea last year, announced that the dose-ranging Phase IIb study met the primary endpoint. According to the announcement, patients who were treated with vupanorsen achieved a statistically significant reduction in non-HDL-C at all doses tested at 24 weeks compared to placebo. Additionally, Ionis announced that patients who received the experimental medication achieved statistically significant reductions in triglycerides and angiopoietin-like 3 (ANGPTL3) at all dose levels at 24 weeks compared to placebo. Vupanorsen is designed to reduce the production of ANGPTL3, a key regulator of triglyceride and cholesterol metabolism that occurs in the liver. Pfizer licensed vupanorsen in October 2019 with a $250 million upfront payment. At the time Pfizer struck the licensing deal, Mikael Dolsten, chief scientific officer and president of Worldwide Research & Development and Medical at Pfizer, said the asset will complement the company’s clinical mid-stage internal medicine pipeline. The pharma giant took over the development of vupanorsen following positive Phase II data for hypertriglyceridemia that was reported by Akcea. The Phase IIb study TaRgeting ANGPTL3 with an aNtiSense oLigonucleotide in AdulTs with dyslipidEmia (TRANSLATE-TIMI 70) included 286 patients ages 40 and older who have dyslipidemia. Patients received a range of dose levels over a period of 24 weeks. The primary endpoint was a percent change from baseline in non-HDL-C at week 24. Sotirios "Sam" Tsimikas, vice president of global cardiovascular development and cardiovascular franchise lead at Ionis, expressed his excitement at the topline results of the Phase IIb study. He said the companies were pleased to see statistically significant reductions in non-HDL-cholesterol, which was the primary endpoint, as well as achieving the secondary endpoint of reductions of triglycerides at all doses tested. “The topline results of the Phase IIb study also showed that vupanorsen dose-dependently lowered its target, angiopoietin-like 3. Pfizer is continuing to review the findings to determine next steps regarding future development,” Tsimikas said in a statement. He added that the company is looking forward to seeing the full data set from the study that will be presented at a medical conference in 2022. The most common adverse events recorded were injection site reactions. Those most often happened in the highest vupanorsen dose group. The most common laboratory abnormalities reported were increases in liver enzymes, alanine aminotransferase and aspartate aminotransferase. Those were also primarily seen at the higher doses with no severe adverse events associated to the treatment.

Oligonucleotide

24 Nov 2021

·www.biospace.com

Ionis announces that Pfizer reports topline results from Phase 2b clinical study of vupanorsen

- Study met its primary endpoint, achieving statistically significant reduction in non-high density lipoprotein cholesterol (non-HDL-C) compared to placebo at all doses tested - Key secondary endpoints were met including statistically significant reductions in triglycerides and angiopoietin-like-3 (ANGPTL3) at all doses tested - Pfizer is continuing to review the findings to determine next steps regarding future development [24-November-2021] CARLSBAD, Calif., Nov. 24, 2021 /PRNewswire/ -- Ionis Pharmaceuticals, Inc., (NASDAQ: IONS) today announced that Pfizer has provided an update on the Phase 2b study of vupanorsen, formerly IONIS-ANGPTL3-LRx. Vupanorsen is an investigational antisense therapy being developed for indications in cardiovascular (CV) risk reduction and severe hypertriglyceridemia (SHTG). In the dose-ranging study in subjects with elevated non-HDL-C and triglycerides (TG), the study met its primary endpoint, achieving a statistically significant reduction in non-HDL-C at all doses tested at 24 weeks, compared to placebo. In addition, subjects treated with vupanorsen achieved statistically significant reductions in TG and ANGPTL3 at all dose levels at 24 weeks, compared to placebo. "We were pleased to see statistically significant reductions in the primary endpoint, non-HDL-cholesterol, and in the secondary endpoint of triglycerides at all doses tested. The topline results of the Phase 2b study also showed that vupanorsen dose-dependently lowered its target, angiopoietin-like 3. Pfizer is continuing to review the findings to determine next steps regarding future development. We look forward to the full data set being presented at a medical meeting next year," said Sotirios "Sam" Tsimikas, M.D., vice president of global cardiovascular development and cardiovascular franchise lead at Ionis. The most common adverse events were injection site reactions, which occurred most often in the highest vupanorsen dose group. The most common laboratory abnormalities were increases in liver enzymes, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) and were seen primarily at the higher doses. There were no Hy's Law cases in vupanorsen-treated subjects, and no meaningful changes in bilirubin. Certain doses of vupanorsen were associated with increases from baseline in hepatic fat fraction, measured by magnetic resonance imaging proton density fat fraction at Week 24, compared to placebo. No subject had a confirmed platelet count abnormality or a confirmed reduction in the estimated glomerular filtration rate. There were no serious adverse events (SAEs) related to treatment. The incidence of SAEs was similar between active and placebo groups. The global multicenter, double-blind, placebo-controlled, dose-ranging Phase 2b study TaRgeting ANGPTL3 with an aNtiSense oLigonucleotide in AdulTs with dyslipidEmia (TRANSLATE-TIMI 70) enrolled 286 participants (≥ 40 years old) with dyslipidemia, defined in this study as participants with elevated non-HDL-C (≥ 100 mg/dL) and TG (150-500 mg/dL), who are receiving a stable dose of a statin. Participants received either 80 mg, 120 mg or 160 mg every 4 weeks, or 60 mg, 80 mg, 120 mg or 160 mg every two weeks via subcutaneous injection. The study was designed to assess the efficacy, safety, tolerability and pharmacokinetics of vupanorsen, and the primary endpoint was percent change from baseline in non-HDL-C at week 24. About vupanorsen Vupanorsen is an investigational antisense therapy discovered by Ionis and being developed by Pfizer for potential indications in CV risk reduction and SHTG. Vupanorsen is designed to reduce the production of ANGPTL3 protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. This antisense therapy was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology. The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of LDL-C and TG, and a decreased risk of diabetes and CV disease.i In a Phase 1 study, subjects treated with vupanorsen achieved robust, dose-dependent reductions in ANGPTL3, TG, LDL-C, non-HDL-C and total cholesterol with a favorable safety and tolerability profile.ii In a Phase 2a study, vupanorsen met the primary endpoint of significant reductions in TG levels and multiple secondary endpoints compared to placebo, with a favorable safety and tolerability profile.iii In November 2019, Pfizer licensed vupanorsen from Ionis in a worldwide exclusive agreement. About Ionis Pharmaceuticals, Inc. For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing the standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry leading neurological and cardiometabolic franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming one of the most successful biotechnology companies. To learn more about Ionis visit or follow us on Twitter @ionispharma. Ionis' Forward-looking Statement This press release includes forward-looking statements regarding Ionis' business, and the therapeutic and commercial potential of vupanorsen and Ionis' technologies and products in development. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including those related to the impact COVID-19 could have on our business, and including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2020, and the most recent Form 10-Q quarterly filing, which are on the SEC. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. References i JAMA Cardiol. 2018 Oct 1;3(10):957-966. ii N Engl J Med. 2017 Jul 20;377(3):222-232. iii Eur Heart J. 2020 Oct 21;41(40):3936-3945. Company Codes: NASDAQ-NMS:IONS

Oligonucleotide

10 Jun 2021

·www.globenewswire.com

Lipodystrophy Pipeline | A Clinical Trials Analysis Report

Los Angeles, USA, June 09, 2021 (GLOBE NEWSWIRE) -- Lipodystrophy Pipeline | A Clinical Trials Analysis Report | DelveInsight The companies with their Lipodystrophy drug candidates in the most advanced stage (Phase III stage) include Ionis Pharmaceuticals, soon drifting the Lipodystrophy Pipeline landscape. DelveInsight's "Lipodystrophy Pipeline Insight" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Lipodystrophy pipeline landscapes. It comprises Lipodystrophy pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Lipodystrophy therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Lipodystrophy pipeline products. Some of the key takeaways from the Lipodystrophy Pipeline Report Get an overview of pipeline landscape @ Lipodystrophy Clinical Trials Analysis Lipodystrophy is a medical problem where there is an abnormal distribution of fat in the body. The condition is also characterised by a lack of circulating leptin, which may lead to osteosclerosis. Currently, there is no specific treatment, which will permanently replace adipose tissue. The Lipodystrophy treatment is focused on managing metabolic abnormalities to prevent complications and cosmetic appearance. Lipodystrophy Emerging Drugs IONIS-APOCIII-LRx, formerly known as AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to prohibit the production of apoC-III for patients who are at risk of condition due to increased triglyceride levels. ApoC-III is a protein produced in the liver that maintains triglyceride metabolism in the blood. The drug is currently being investigated in the Phase III stage of development to treat patients with Hyperlipoproteinaemia and Lipodystrophy. The U.S. FDA and European Medicines Agency have also granted the drug with the Orphan drug designation. Vupanorsen is an investigational antisense therapy founded by Ionis Pharmaceuticals and co-developed by Ionis and its subsidiary Akcea Therapeutics. Vupanorsen has been created using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform to prohibit the production of angiopoietin-like 3 (ANGPTL3) protein, which a vital regulator of triglyceride and cholesterol metabolism in the liver. Pfizer has started a Phase 2b study of vupanorsen in statin-treated patients with elevated non-high-density lipoprotein cholesterol (non-HDL-C) and triglycerides (TGs). REGN4461 is a leptin receptor (LEPR) agonist for lipodystrophy and obesity which is being developed by Regeneron Pharmaceuticals. It is currently in the Phase II stage of development. It is administered intravenously or subcutaneously. CGT2 was licensed from Lipigon Pharmaceuticals 2019; CombiGene expanded its scope to include metabolic diseases. The project's initial goal is to develop a gene therapy treatment for partial lipodystrophy, a very rare disease that today completely lacks adequate treatment. CGT2 is currently in the Preclinical stage of development. For further information, refer to the detailed report @ Lipodystrophy Pipeline Therapeutics Scope of Lipodystrophy Pipeline Drug Insight Key Questions regarding Current Lipodystrophy Treatment Landscape and Emerging Therapies Answered in the Pipeline Report Table of Contents Get a customised pipeline report @ Lipodystrophy Drugs Pipeline Report Related Reports DelveInsight's Lipodystrophy - Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and a depth understanding of historical and forecasted epidemiology. DelveInsight' s HIV-associated Lipodystrophy - Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and a depth understanding of historical and forecasted epidemiology. Lipodystrophy Market Research Report deliver an in-depth understanding of the Lipodystrophy Market Size, Share, Trends, Pipeline Therapies, Key Companies, and Epidemiology Forecast in the 7MM. DelveInsight's Lipodystrophy - Epidemiology Forecast 2030 report delivers an in-depth understanding of the disease, historical, and forecasted epidemiology of Lipodystrophy in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. DelveInsight's Liposarcoma Market Insights, Epidemiology and Market Forecast - 2030 report provides a depth understanding of historical and forecasted epidemiology. DelveInsight's Necrobiosis Lipoidica (NL) - Market Insights, Epidemiology and Market Forecast - 2030 report provides a detailed overview of the disease and a depth understanding of historical and forecasted epidemiology. About DelveInsightDelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also provides Healthcare Consulting services comprising credible market analysis that will help accelerate the business growth and overcome challenges with a practical approach.

Gene TherapyOrphan Drug

100 Deals associated with Vupanorsen

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hyperlipoproteinemias	Phase 2	US	Pfizer Inc.	28 Sep 2020
Hyperlipoproteinemias	Phase 2	CA	Pfizer Inc.	28 Sep 2020
Hyperlipoproteinemias	Phase 2	PL	Pfizer Inc.	28 Sep 2020
Lipodystrophy, Familial Partial	Phase 2	US	Ionis Pharmaceuticals, Inc.	15 Jun 2018
Homozygous familial hypercholesterolemia	Phase 2	CA	Ionis Pharmaceuticals, Inc.	12 Apr 2018
Diabetes Mellitus, Type 2	Phase 2	US	Ionis Pharmaceuticals, Inc.	21 Dec 2017
Diabetes Mellitus, Type 2	Phase 2	CA	Ionis Pharmaceuticals, Inc.	21 Dec 2017
Familial chylomicronaemia syndrome	Phase 2	CA	Ionis Pharmaceuticals, Inc.	21 Dec 2017
Familial Hyperchylomicronemia Syndrome	Phase 2	CA	Ionis Pharmaceuticals, Inc.	21 Dec 2017
Hypertriglyceridemia	Phase 2	US	Ionis Pharmaceuticals, Inc.	21 Dec 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04916795 (Pubmed) Manual	Phase 1	ANGPTL3	18	Vupanorsen 80 mg	mozcbvbtzf(noexehdeqv) = Three participants experienced treatment-related AEs; all were mild and resolved by end of study datugztywl (wybxscweqd ) View more	Positive	01 Jul 2024
				Vupanorsen 160 mg
NCT04916795 (Phase I, CTgov)	Phase 1	-	18	Vupanorsen (Vupanorsen 80 mg)	jqsyovnnyw(ltunlcudeh) = mizuwtheiw dqqmyjekmp (wdoewesxdc, wcbexttacy - zyjxritnvs) View more	-	12 Mar 2024
				Vupanorsen (Vupanorsen 160 mg)	jqsyovnnyw(ltunlcudeh) = yfdoyewkbs dqqmyjekmp (wdoewesxdc, buguanoiap - xskpkafsdg) View more
NCT04459767 (Pubmed)	Phase 1	-	4	Vupanorsen 80mg	mbpjqhfods(rewhlkowmc) = qjaxbsdypm emeostrtbi (hbratrjiou )	-	31 Mar 2023
				Vupanorsen 160mg	mbpjqhfods(rewhlkowmc) = wllyzbejmf emeostrtbi (hbratrjiou )
NCT04516291 (CTgov)	Phase 2	Hyperlipoproteinemias	286	Placebo	bqmjcvwazn(aepbrccpmx) = lmrkaxqccx ldzfrmugnq (anvwoetunr, xeuebkocxj - rduwfhqawn) View more	-	04 Oct 2022
NCT03514420 (Pubmed \| Lipids Health Dis) Manual	Phase 2	Lipodystrophy, Familial Partial	4	vupanorsen	xpdtgbbwzh(juezgdoksq) = xcrbivvlmj yjrnkjcxsg (bcghheldhy ) View more	Positive	05 Dec 2021
NCT03514420 (CTgov)	Phase 2	Lipodystrophy, Familial Partial	4	AKCEA-ANGPTL3-LRx	uaqascjaoz(sqwgchddob) = oazdtbrccs jrzuqunkne (rjzfnuguci, qsovtjbctw - zrilgiumej) View more	-	16 Feb 2021
NCT03371355 (AKCEA-ANGPTL3-L(Rx), CTgov)	Phase 2	Hypertriglyceridemia \| Diabetes Mellitus, Type 2 \| Nonalcoholic fatty liver	105	Placebo (Pooled Placebo)	arelcfjmcu(bbcqduujhz) = egjcugbwck kdawdgoggc (fyxulgzppa, bwrhzevwgj - etrmahumco) View more	-	01 Feb 2021
				ISIS 703802 40 mg (Cohort B: ISIS 703802, 40 mg Q4W)	arelcfjmcu(bbcqduujhz) = flgmkasott kdawdgoggc (fyxulgzppa, rxhkkjdicu - gmfzxcckyu) View more
NCT03360747 (CTgov)	Phase 2	Familial Hyperchylomicronemia Syndrome \| Familial chylomicronaemia syndrome	3	AKCEA-ANGPTL3-LRx	exgfmtrafe(baspbhcfbx) = ipkdcvqxvv idbsfuxilx (lzlnhruxis, zeatalcdgz - jyjmwasxpv) View more	-	07 Jan 2021
NCT03371355 (AKCEA-ANGPTL3-L(Rx), PRNewswire) Manual	Phase 2	Hypertriglyceridemia	105	Vupanorsen	wzlcnchzoo(qqxqllvegb) = hwppkcskwu zjdontwhkr (fuzjejlcha )	Positive	29 Aug 2020
				Placebo	-

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