Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
IONIS-PKKRx
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
IONIS-PKKRx is a second-generation, 2′-O-(2-methoxyethyl)-modified chimeric antisense oligonucleotide designed to selectively bind and reduce hepatic prekallikrein (PKK) mRNA, thereby lowering plasma PKK protein levels. By inhibiting the production of PKK in the liver, it reduces bradykinin generation capacity—a key mediator in hereditary angioedema (HAE) and other diseases involving contact system activation. The drug has demonstrated dose-dependent reductions of plasma PKK in animal models and healthy human volunteers with an acceptable safety profile. It was developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) for the prevention and treatment of HAE and has also been studied for migraine[1][2][3][5][7].
- Other names
- PKK
02
Targets
PKK (Plasma prekallikrein (KLKB1))
Beyond the preview
Go deeper on IONIS-PKKRx.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on IONIS-PKKRx.