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PTX-022 (novel, high-strength rapamycin topical formulation, optimized for dermal targeting)

Palvella is currently entering a Phase 2/3 clinical program with lead candidate PTX-022, a topical formulation comprising high-strength rapamycin developed to treat pachyonychia congenita (PC).

PTX-022 is the first Palvella product to leverage the company’s proprietary QTORIN™ technology. QTORIN™ was designed and developed by a global research team led by Dr. Braham Shroot, an internationally regarded scientist and senior executive in the biotech industry with more than 30 years of experience in dermatology and topical formulation development. Dr. Shroot has led the development of numerous novel topical therapies which have achieved regulatory approvals and successful commercialization in multiple geographies.

PTX-022 has multiple issued method-of-use patents in the US broadly covering the use of rapamycin in PC through 2032.


Scientific Rationale

The potential for rapamycin in PC was originally discovered by leading US and EU scientists in the field of pachyonychia congenita in 2006 who elucidated a potential direct mechanism of action of rapamycin on the mutant keratin genes driving the pachyonychia congenita disease process.

Further therapeutic effects of rapamycin in PC are likely derived from rapamycin’s known anti-angiogenic properties, which is important given the documented hyper-neovascularization in PC.

Topical and oral formulations of rapamycin have previously achieved proof of concept in small human studies that have demonstrated rapid improvement in ambulation and improved quality of life in patients with PC.

PTX-022 was developed to deliver clinically meaningful concentrations of rapamycin through the targeted skin layers of the epidermis and dermis while minimizing systemic absorption. PTX-022 will be GMP compliant, providing a consistent quality, purity and concentration of rapamycin in a temperature-stable formulation.


Regulatory Designations

PTX-022 received FDA orphan designation in 2013 and was subsequently granted orphan designation by the European Medicines Agency in 2017.

The FDA also awarded the program an FDA Orphan Products Clinical Trials Grant based on its scientific and technical merit.

The goal of the Orphan Products Clinical Trials Grants Program is to encourage clinical development of products for use in rare diseases or conditions. Orphan products clinical trials grants are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases/conditions. The Orphan Products Clinical Trials Grants Program has been used to bring more than 60 products to marketing approval. (Source: FDA)


Corporate Development

Palvella Therapeutics is actively seeking additional in-licensing and acquisition opportunities to expand our pipeline of novel therapies for rare diseases. For more information, please contact the company at info@palvellatx.com.