The Scleroderma Research Foundation (SRF) Launches Conquest Trial Platform to Address Critical Issues in Clinical Development and Enable Advances in Scleroderma Therapeutics

• Sanofi is inaugural partner

• Initial focus on Interstitial Lung Disease Secondary to Scleroderma

• SRF will submit and hold the IND as the trial Sponsor

SAN FRANCISCO--(BUSINESS WIRE)--The Scleroderma Research Foundation (SRF), the nation’s largest non-profit funder of scleroderma research, today announced that Sanofi will contribute the first experimental agent to CONQUEST, a highly innovative clinical trial platform created by the SRF. The platform is designed to rapidly advance promising treatments for scleroderma and to identify those agents that should progress from Phase 2b to Phase 3 clinical trials.

Using a model first created over a decade ago to accelerate oncology drug development, the SRF platform is the first of its kind in rare autoimmune disease. In its initial iteration, CONQUEST will focus on Interstitial Lung Disease secondary to Scleroderma (SSc-ILD). In the future, the CONQUEST platform will be expanded to address other manifestations of scleroderma.

This groundbreaking effort, conceived and led by the SRF, will enroll patients across more than 130 centers in more than 22 countries. CONQUEST offers advantages to all stakeholders -- patients, physician, and drug innovators -- and provides for a seamless transition to a global Phase 3 trial, investigation of a drug’s activity in a wide range of genetic backgrounds, as well as rapid patient enrollment. By assembling a global network of high-performing centers that are dedicated to treating scleroderma, the SRF and its pharmaceutical partners expect to make enduring contributions to the scleroderma community by creating an ecosystem designed to enable success in new drug development.

Scleroderma is a systemic autoimmune disease that affects approximately 100,000 patients in the US with high morbidity and mortality rates and for which there are limited treatment options. The disease presents multiple challenges for patients with a spectrum of symptoms that prominently include vascular disease and internal organ fibrosis.

Luke Evnin, Ph.D., Chairman of the Scleroderma Research Foundation, commented, “Our commitment to innovation in scleroderma spans basic, translational, and clinical research. A decade ago, we began to plan how we could adapt some of the impressive clinical development innovations in cancer to scleroderma.

Recent advances in understanding drivers of autoimmunity and fibrosis have generated a rich pipeline of promising therapeutics across autoimmunity and specifically, for scleroderma. However, among other issues, the breadth and depth of opportunities poses clinical development challenges in a rare disease setting such as scleroderma, and CONQUEST arrives at the right moment to address those challenges.”

Dr. Evnin added, “As with our all of our research efforts, the incubation and assembly of CONQUEST was underwritten by generous SRF donors. With essential contributions from expert clinicians other as well as our potential pharmaceutical partners, the platform is ready. We are confident that this initiative will pay huge dividends for the global scleroderma community -- from patients, to clinicians, to our pharmaceutical partners.”

CONQUEST uses a master protocol that provides a regulatory framework to study multiple potential treatments for scleroderma, and like other platform trials, this provides multiple efficiencies not available in traditional clinical trial protocols, including:

• New agents may enter and leave the platform without halting patient enrollment or resubmitting the entire clinical trial protocol for regulatory review.
• The trial infrastructure is built once but remains open with substantial efficiencies for each cycle and enabling knowledge gained to improve the platform over time.
• A common control group is enrolled that serves multiple experimental arms.
• In all, the platform provides a win-win for all stakeholders as compared to conventional trials - smaller sample sizes, fewer patients on placebo, reduced costs, faster timelines to data, better inferences for regulators and the ability to share the accumulated learnings across the entire scleroderma research community.

Dinesh Khanna, MD, CONQUEST Principal Investigator and the Frederick G.L. Huetwell Professor of Medicine and Director, University of Michigan Scleroderma Center noted, “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. CONQUEST is a huge step forward and I am honored to lead a truly global effort that will unite the scleroderma community to efficiently test novel agents and speed the introduction of highly effective therapies to our needy patients.”

Toby Maher, MD, CONQUEST Co-Principal Investigator and the Professor of Medicine and Director of Interstitial Lung Disease at Keck School of Medicine, the Professor of Interstitial Lung Disease at Imperial College London and the British Lung Foundation Professor of Respiratory Medicine added, “CONQUEST has brought together innovative pharmaceutical partners, together with leading scleroderma experts from rheumatology and leading ILD experts from pulmonology to develop a platform that will efficiently test novel medicines for SSc-ILD patients. Leveraging the efficiencies of CONQUEST, we anticipate that the rate of innovation in scleroderma will dramatically change.”

The CONQUEST Therapy Evaluation Committee, comprised of expert scleroderma clinicians and scientists, selected from a range of possible treatments for the inaugural run of the CONQUEST platform. The SRF anticipates that treatments will be added to the CONQUEST Platform Trial and is in active discussions with academic and industry partners for the next round of CONQUEST. Nominations of therapies for inclusion in the platform trial will continue on an ongoing, rolling basis.

The first iteration of CONQUEST is designed to include two innovative drugs including one from Sanofi. CONQUEST represents the first time this drug will be evaluated in scleroderma patients, but it has been evaluated in related diseases in early-stage clinical trials. The initial run of CONQUEST is expected to enroll approximately 400 patients and trial initiation is targeted for Q4 2023. The primary endpoint as measured at 52 weeks is forced vital capacity (FVC), the total volume of air that can be exhaled during a maximal forced expiration effort.

About the Scleroderma Research Foundation

The Scleroderma Research Foundation (SRF), a 501(c)(3) organization, was established in 1987 with a mission to fund and facilitate the most promising, highest quality research aimed at new treatments and, ultimately, a cure for scleroderma. Led by a Scientific Advisory Board comprised of some of the most highly regarded scientists in the nation, the SRF’s research program actively seeks out the leading scientific minds from disparate fields including autoimmunity, immunology, genetics, and fibrosis to join the scleroderma research community. In addition to its core research program, the SRF has also led the formation of two other large-scale projects aimed at accelerating scleroderma research:

• The CONQUER Registry, the first nationwide longitudinal registry for scleroderma patients, and
• The GRASP Project, which examines scleroderma in the African American community.

The SRF is also dedicated to educating people living with scleroderma and their caregivers as they learn about how to best manage the challenges of the disease.

Media:
Stephanie Blank, FGS Global
stephanie.blank@fgsglobal.com
917-593-2907