Therapeutic ProgramsAileron is developing a broad and growing pipeline of Stapled Peptide drugs across a range of therapeutic areas, including cancer, infectious disease, metabolic and endocrine conditions, and inflammation. In early 2013, Aileron will begin clinical evaluation of its wholly owned and internally-discovered lead Stapled Peptide drug candidate, ALRN-5281, a proprietary, long-acting growth-hormone-releasing hormone agonist for treating orphan endocrine disorders, including adult growth hormone deficiency and HIV lipodystrophy, as well as broader patient populations involving a wide variety of metabolic/endocrine diseases. The ALRN-5281 Phase 1 study will mark the first-ever Stapled Peptide clinical trial. Aileron is also advancing multiple Stapled Peptide drug candidates in collaboration with Roche, including its lead oncology program, a highly potent and specific dual inhibitor of MDM2 and MDMX for p53-dependent cancers. Aileron’s Stapled Peptide technology enables a unique approach that locks native peptides into their biologically active shape, and imparts stability to achieve best-in-class pharmacokinetic (PK) parameters. In particular, the Stapled Peptide technology can create a prolonged “square-wave” PK profile that stays in the therapeutic range, with minimal crossover into unacceptable safety or inadequate efficacy drug levels. By applying Stapled Peptide technology to appropriate targets, Aileron can create optimized medicines with the potential for enhanced safety, efficacy, and patient convenience and compliance.
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