LEO Pharma Funds PellePharm’s Phase 3 Trial of Promising Gorlin Syndrome Drug

According to an article published by CheckOrphan, LEO Pharma and PellePharm have announced a new multi-million dollar partnership together with the aim of advancing research for rare skin diseases that have no current cure, like Gorlin Syndrome or High Frequency Basal Cell Carcinoma.

What is Gorlin Syndrome?

Gorlin Syndrome is a rare condition affecting about 1 in 31,000 people.

Those with the condition are much more likely to develop basal cell carcinoma tumors, which are the most common form of skin cancer. Those afflicted typically detect their first basal cell carcinomas during their teenage and young adult years.

Some patients might not develop any tumors at all. Others will develop thousands.

The current approved treatment course is surgery, which is often regular. The condition lasts a patient’s lifetime – and some will have as many as 30 surgeries a year starting in their teens.

PellePharm and LEO Pharma Partner Up

PellePharm is a California-based biotechology company developing a promising topical treatment for Gorlin Syndrome that is preparing to undergo phase 3 clinical trials.

Under the terms of the new agreement, LEO Pharma has committed an initial $70 million in financing and R&D support for PellePharm’s phase 3 trial. The agreement also included an option for LEO to aquire all PellePharm shares outright for another $690 million and merger consideration.

The global phase 3 trials will likely begin in early 2019, according to PellePharm president and CEO Sanuj Ravindran.

Pending the success of the new trial and potential merger, Ravindran said his company would be eager to work more with LEO to address other rare skin diseases.

Patidegib to Undergo Phase 3 Trials

PellePharm’s hopes are riding on a topical gel comprised of a 2% formulation of patidegib. The gel treatment showed promising results in a phase 2 clinical study, and aims to provide the relief of basal cell carcinomas by blocking the disease in its main highway, the hedgehog signaling pathway.

Yes, you read that right. Essentially, the hedgehog signaling pathway is a communication system used by the body to give newborn cells instructions on how to differentiate themselves (i.e. it tells a new cell whether to become a brain cell or a liver cell).

However, clinical trials are lengthy affairs, and there is no guarantee that patidegib will pass its phase 3 clinical study. Regardless of how the research will turn out, it’s encouraging to see private entities taking the initiative in researching treatments for currently incurable diseases.


Have you ever been a part of a clinical trial? Do pharmaceutical mergers help or hamper the development of new drugs? Share your thoughts with the Patient Worthy Community!

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