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Cell-penetrating peptides (CPPs), are small peptides that facilitate cytosolic access and, thus, transport of therapeutic macromolecules to intracellular sites when conjugated to cargo proteins. As with all new delivery platforms, clinical development of CPP-containing therapeutics has faced considerable challenges. RTP004 is a novel, 35–amino acid, bi-CPP-containing excipient that binds noncovalently with its cargo (botulinum toxin type A) rather than conjugated as a fusion protein. An RTP004-containing neurotoxin formulation, daxibotulinumtoxinA-lanm for injection (DAXI), has recently been approved by the FDA. The formulation and pharmacological characteristics of RTP004 and the efficacy and safety of the RTP004-neurotoxin formulation are discussed. RTP004 is a highly positively charged lysine- and arginine-rich structure that provides formulation stability, preventing self-aggregation of the cargo protein and adsorption to container surfaces. The presence of RTP004 in the formulation also appears to increase presynaptic binding of the neurotoxin, reduces post-injection diffusion, and thus facilitates an increase in the cleavage of the intracellular substrate for the botulinum toxin, likely through enhanced cellular uptake. The RTP004-neurotoxin formulation is the first CPP-containing product approved for clinical use. The potential for RTP004 to facilitate other therapeutic cargo molecules requires further research.

A technology evaluation of the atypical use of a CPP-containing peptide in the formulation and performance of a clinical botulinum toxin product