Scleroderma clinical trial initiative snags Sanofi as first partner to accelerate research

Sanofi will be the inaugural partner for the Scleroderma Research Foundation’s new clinical trial initiative to advance treatments for the rare autoimmune disorder of the skin and organs.

The non-profit organization announced the launch of CONQUEST with one of Sanofi’s therapies in a Tuesday press release. The new clinical trial platform will help accelerate the development of therapies for scleroderma by identifying agents that are ready to progress from phase 2b to phase 3.

While the model has been used before to advance oncology trials, this will be the first initiative of its kind for autoimmune disorders, the SRF said.

“A decade ago, we began to plan how we could adapt some of the impressive clinical development innovations in cancer to scleroderma,” said Luke Evnin, Ph.D., chairman of the SRF. “Recent advances in understanding drivers of autoimmunity and fibrosis have generated a rich pipeline of promising therapeutics across autoimmunity and specifically, for scleroderma.”

But while having new options to test is great news, it does represent a challenge identifying the right therapies, especially in a rare disease like scleroderma, Evnin added. “CONQUEST arrives at the right moment to address those challenges.”

CONQUEST will initially focus on interstitial lung disease secondary to scleroderma but eventually expand into all manifestations of the disorder. Patients with scleroderma experience skin tightening and thickening that can affect the joints, muscles, heart, kidneys and other vital organs.

The first run will include 400 patients testing two drugs, including the offering from Sanofi, and is expected to get going in the fourth quarter. The trial will measure forced vital capacity, a measuring of the total volume of air that can be exhaled during a maximal forced expiration effort, as the primary endpoint.

Patients will be enrolled at more than 130 centers in over 22 countries. The goal is to develop a network of centers treating scleroderma to facilitate success in developing new treatments for the disease.

The master protocol will allow for new agents to enter and leave testing without halting patient enrollment or having to resubmit the entire trial protocol for regulatory review. The trial infrastructure will remain open, meaning the cycle can continue efficiently. A common control group will be used across the arms of the trial to serve multiple experimental arms.

“I have been involved in numerous recent clinical trials for our scleroderma patients. It was clear to me that a radical re-working of how we approach clinical trials in our rare disease community was required if our patients were going to realize the tremendous benefits that patients suffering from other autoimmune diseases have recently experienced,” said Dinesh Khanna, M.D., principal investigator for CONQUEST and the Frederick G.L. Huetwell Professor of Medicine and director at the University of Michigan Scleroderma Center.

Overall, the platform means fewer patients on placebo, smaller sample sizes, faster timelines to data and the ability to share learnings across those researching scleroderma.

While Sanofi is the first company to contribute a therapy to the effort, the SRF is in “active discussions” with academic and industry partners to find more options for the next round.