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IONIS TMPRSS6-Lrx · 1 trial · 1 indication
The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
| Arm | Type | Description |
|---|---|---|
| IONIS TMPRSS6-Lrx | EXPERIMENTAL | Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously |
| Placebo | PLACEBO_COMPARATOR | Saline .9% |
| Name | Type | Description |
|---|---|---|
| IONIS TMPRSS6-Lrx | DRUG | Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously |
| Placebo | OTHER | Saline .9% |
Inclusion Criteria: * Must have given written informed consent and be able to comply with all study requirements * Healthy males or females aged 18-65 inclusive at the time of Informed Consent * Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal * Male...
Top 6 of 8 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat, AG-348 |
| Bristol-Myers Squibb Company | BMY | 6 | PHASE3 | Luspatercept |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Etavopivat |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
IONIS TMPRSS6-Lrx is an investigational RNA therapy developed by Ionis Pharmaceuticals, Inc. (ticker IONS) for thalassemia, a group of inherited blood disorders. It is designed to be given by subcutaneous injection and has been studied in a Phase 1 clinical trial in healthy volunteers.
IONIS TMPRSS6-Lrx is being developed as a treatment for thalassemia, an inherited blood disorder. It is an RNA therapy administered subcutaneously. The program has completed a Phase 1 study in healthy volunteers, and no later-stage trials in patients with thalassemia have been reported.
IONIS TMPRSS6-Lrx is made by Ionis Pharmaceuticals, Inc., which trades under the ticker symbol IONS. Ionis is the developer of the asset, which is an RNA therapy being studied for thalassemia.
IONIS TMPRSS6-Lrx is in Phase 1 development. Its only reported clinical trial, NCT03165864, was a Phase 1 study that has been completed. The drug is investigational and has not been approved for any indication.
IONIS TMPRSS6-Lrx has been evaluated in one clinical trial, NCT03165864, titled "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers." The Phase 1 study enrolled 36 healthy volunteers in Australia, was randomized, double-blind, and placebo-controlled, and is now completed.
Yes. ISIS 702843 is an alternative name for IONIS TMPRSS6-Lrx, reflecting Ionis Pharmaceuticals' earlier corporate naming convention. The Phase 1 trial NCT03165864 was conducted under the ISIS 702843 designation, and both names refer to the same investigational RNA therapy for thalassemia.