Drug intelligence / Profile preview

ACP-001

Development stage
Phase 2
Lead developer
Ascendis Pharma
Modality
Cell Therapies, Small Molecules
Administration
Subcutaneous, Intravenous
01

Overview

ACP-001 refers to at least two distinct investigational therapies in clinical development, each by different companies and for different indications: 1. **Ascendis Pharma's ACP-001** is a proprietary once-weekly human growth hormone (hGH) prodrug designed to treat growth hormone deficiency in children and adults. It releases unmodified hGH in a controlled manner, aiming to improve patient compliance compared to daily injections. The drug has demonstrated safety and efficacy in Phase 1 studies, with ongoing Phase 2 trials evaluating its pharmacokinetics, pharmacodynamics, safety, and tolerability[7][9]. 2. **XNK Therapeutics' ACP-001** is an autologous natural killer (NK) cell therapy developed for the treatment of multiple myeloma as consolidation after high-dose autologous stem cell transplantation. This cell therapy leverages immunologic cytotoxicity via NK cells to target cancer cells. Early-phase clinical trials have shown promising safety results and measurable responses in patients[3][4]. Because both drugs share the code name "ACP-001" but are unrelated products from different developers with distinct mechanisms of action and indications, all relevant details are provided below.

02

Targets

GHR (Growth hormone receptor)SLAMF7 (Signaling lymphocytic activation molecule family member 7)

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