Approval Probability
TA Base Rate
Adjusted LOA
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CTX340 · 1 trial · 1 indication
To evaluate the safety and tolerability of a single ascending dose of CTX340 in participants with hypertension to determine the recommended Phase 2 dose (RP2D).
To evaluate the pharmacodynamics (PD) effect of CTX340 at the recommended Phase 2 dose (RP2D)
| Arm | Type | Description |
|---|---|---|
| Phase 1: CTX340 | EXPERIMENTAL | Phase 1 is An Open-Label, Ascending Dose Design. |
| Phase 2: Recommended Phase 2 Dose CTX340 | EXPERIMENTAL | Phase 2: Recommended Phase 2 Dose CTX340. |
| Phase 2: Placebo | EXPERIMENTAL | Phase 2: Placebo. |
| Name | Type | Description |
|---|---|---|
| CTX340 | DRUG | CTX340 is an in vivo gene editing therapy designed to utilize clustered regularly interspaced short palindromic repeats-CRISPR-associated protein 9 (CRISPR-Cas9) to target and disrupt human angiotensinogen (AGT) gene in liver. |
| Placebo | DRUG | One of the arms in Phase 2 will be placebo. |
Key Inclusion Criteria: 1. Age: ≥18 and ≤75 years. 2. Body mass index ≤40 kg/m2. 3. 24-hour mean ambulatory blood pressure monitoring (ABPM) systolic blood pressure (SBP) measurement of ≥130 mm Hg but ≤160 mm Hg despite treatment 4. Be on treatment with ≥4 antihypertensive therapies at effective do...
Top 7 of 14 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Eli Lilly and Company | LLY | 3 | PHASE3 | Orforglipron |
| Alnylam Pharmaceuticals, Inc | ALNY | 2 | PHASE3 | Zilebesiran, ALN-AGT01 RVR |
| Mineralys Therapeutics, Inc. | MLYS | 1 | PHASE3 | lorundrostat |
| United Therapeutics Corporation | UTHR | 1 | PHASE3 | Ralinepag |
| Novartis AG Sponsored ADR | NVS | 2 | PHASE2 | QCZ484 |
| Vanda Pharmaceuticals Inc. | VNDA | 1 | PHASE2 | iloperidone |
| Opus Genetics, Inc. | IRD | 1 | PHASE1 | OPGx-BEST1 |
CTX340 is an investigational small molecule being developed for the treatment of hypertension. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is being evaluated in a clinical trial for its safety, tolerability, and efficacy in participants with hypertension.
CTX340 targets the AGT gene, which encodes angiotensinogen, a key component of the renin-angiotensin system involved in blood pressure regulation. By targeting this gene, CTX340 aims to address hypertension at a molecular level. The drug is designed as a small molecule therapeutic for this cardiovascular condition.
CTX340 is being developed by CRISPR Therapeutics AG, a biopharmaceutical company publicly traded under the ticker symbol CRSP. The company is conducting a Phase 1 clinical trial to evaluate the drug's safety, tolerability, and efficacy in patients with hypertension.
CTX340 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory bodies. The ongoing Phase 1 trial is currently recruiting participants to assess the drug's safety, tolerability, and efficacy in individuals with hypertension.
CTX340 is being evaluated in a Phase 1 clinical trial registered as NCT07758634, titled "A Trial Evaluating Safety, Tolerability and Efficacy of CTX340 in Participants With Hypertension." The trial is recruiting 69 participants in the United States and Australia, with an estimated enrollment of 69 individuals aged 18 years and older.