MedPharm was selected by Palvella to support the development of QTORIN™ rapamycin, with MedPharm’s scientists working closely alongside Palvella’s research and development team and leadership to advance the formulation strategy from initial concept through clinical development and preparation for commercial manufacturing, including:

• Collaborating with Palvella to advance the formulation strategy for rapamycin, an inherently
  challenging molecule due to its large molecular weight, poor solubility, chemical instability, and
  complex physicochemical properties.
• Supporting the development of a dermal-targeted, room temperature stable anhydrous gel with a
  high drug load, designed to locally inhibit the mammalian target of rapamycin (mTOR) pathway
  within affected skin tissue across multiple mTOR-driven diseases while minimizing systemic
  exposure.
• Conducting an intensive, multi-year formulation development program, involving complex
  physicochemical optimization to achieve the performance characteristics required by Palvella,
  including applying MedPharm’s specialized expertise to conduct in vitro skin permeation testing
  (IVPT) and in vitro release testing (IVRT) to refine the formulations for release and targeted delivery to
  the site of action.
• Incorporating patient-informed insights into formulation design, including hosting a patient with a
  rare skin disease at MedPharm’s UK laboratories to inform key product attributes.
• Supporting scale-up and manufacturing readiness, including enabling production of clinical trial
  supply and preparation for anticipated near-term commercial-scale manufacturing.

Palvella is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapies to treat patients suffering from serious, rare skin diseases and vascular malformations for which there are no U.S. FDA-approved therapies. Palvella’s QTORIN™ rapamycin has received Breakthrough Therapy, Orphan Drug, and Fast Track designations from the FDA for the treatment of microcystic LMs, as well as an FDA Orphan Products Development grant. QTORIN™ rapamycin is also under development for cutaneous venous malformations and clinically significant angiokeratomas.

“I am delighted that MedPharm has been able to contribute to Palvella reaching this important milestone,” said Bill Humphries, Chief Executive Officer of MedPharm. “As global leaders in the development of semisolid drug formulations, MedPharm looks forward to supporting Palvella with their vision to become the leading rare disease biopharma company serving patients with serious, rare skin diseases and vascular malformations.”

MedPharm has also been selected by Palvella to advance the development of QTORIN™ pitavastatin for porokeratosis, a serious, rare genetic skin disease characterized by chronic, often pruritic lesions and an increased risk of progression to skin cancer. There are currently no FDA-approved therapies for porokeratosis. If approved, QTORIN™ pitavastatin could become the first therapy specifically targeted to treat this condition, addressing a significant unmet medical need for patients.

From its facilities in Durham, North Carolina and Guildford in the United Kingdom, MedPharm provides a full suite of services for creams, gels, ointments, liquids, lotions, and pastes. With over two decades of experience, the company is able to develop and optimize semi-solid dosage forms to ensure maximized drug delivery and stability, enhanced patient adherence, and regulatory compliance.

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About MedPharm

MedPharm is an end-to-end contract service provider of topical and transepithelial products supporting early-phase research, formulation development and in vitro testing services in addition to both clinical and commercial manufacturing. With its 2024 merger with Tergus Pharma, MedPharm enhanced its capabilities, transforming the business into a truly differentiated CDMO of scale with superior end-to-end capabilities from drug development and formulation to large batch commercial manufacturing and distribution. For more than 25 years, MedPharm has specialized in reducing risk through its unique, industry-leading in vitro testing and research biology models and is a global leader in product development for dermal/transdermal, lung and nasal, mucosal membrane, ungual, otic, and ophthalmic delivery. MedPharm has locations in the United Kingdom and the United States, including a new 110,000 sq. ft. state-of-the-art manufacturing facility in Durham, North Carolina. For more information, visit medpharm.com or follow us on LinkedIn.

QTORIN™ rapamycin and QTORIN™ pitavastatin are for investigational use only and neither has been approved by the FDA or by any other regulatory agency for any indication.

Media Contacts:

Sadie Smith, Marketing and Events Coordinator
E: sadie.smith@medpharm.com

Chris Halling, Director of Communications, Orientation Marketing
T: +44 (0)7580 041073
E: chris.halling@orientation.agency