The Scleroderma Research Foundation Announces the Launch of the CONQUEST Trial Platform and IND Clearance

• FDA clears IND to launch CONQUEST trial platform with initial focus on Interstitial Lung Disease Secondary to Scleroderma (SSc-ILD)
  
  
• SCLERODERMA RESEARCH FOUNDATION (SRF) holds the IND as trial Sponsor
  
  
• Sanofi and Boehringer Ingelheim share the platform; patient enrollment to begin early in 2024

SAN FRANCISCO, Dec. 19, 2023 /PRNewswire/ -- The Scleroderma Research Foundation (SRF), the nation's largest non-profit funder of scleroderma research, today announced that the FDA has cleared its Investigational New Drug application (IND) to launch the CONQUEST clinical trial platform and begin enrolling patients. Sanofi and Boehringer Ingelheim will each contribute an experimental agent to CONQUEST, and as such they are the first pharma partners to commit to this protocol designed to efficiently advance new scleroderma treatments through clinical development. The first two nominated investigational drugs will be evaluated for safety and efficacy in scleroderma patients burdened by interstitial lung disease (SSc-ILD). The Sanofi agent is amlitelimab, a non-depleting monoclonal antibody that binds to OX40-Ligand; the Boehringer Ingelheim agent is BI -1015550, an oral PDE-4B inhibitor. Both investigational agents have shown preliminary evidence of efficacy in prior clinical trials for non-scleroderma indications in modifying disease processes.

Luke Evnin, Ph.D., Chairman of the Scleroderma Research Foundation, commented, "Both Sanofi and Boehringer Ingelheim have a rich history of innovation in the immunologic and fibrotic disease arena, and they are bringing their considerable expertise to CONQUEST. With these two partners, CONQUEST is well positioned to ensure that the platform will animate the SRF vision of building a global community tuned to rapid and efficient clinical development of promising novel therapeutic agents for the high unmet needs of scleroderma patients. I am proud and excited to see two promising experimental agents for SSc-ILD advancing, and I have high hopes that these agents will prove safe and efficacious in our patients." 

Boehringer Ingelheim's BI - 1015550 is an orally-administered phosphodiesterase 4B (PDE4B) inhibitor that is being investigated as a potential treatment for idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). Preclinical studies have shown that BI 1015550 has anti-inflammatory and antifibrotic properties. 

The FIBRONEER™ global program includes two Phase III studies – FIBRONEER-IPF (NCT05321069) in patients with IPF and FIBRONEER-ILD (NCT05321082) in people living with other progressive fibrosing interstitial lung diseases (ILDs) and was initiated in 2022. The FDA granted BI - 1015550 Breakthrough Therapy Designation for the treatment of IPF in February 2022 and Orphan Drug Designation for BI -1015550 in IPF in October 2022.

According to Ioannis Sapountzis, Global Head of Therapeutic Areas, Boehringer Ingelheim, "As a global innovation leader, we remain steadfast in our commitment for people living with scleroderma. Mounting our agents into CONQUEST is a critical step to help bring forward this next generation of treatment to those in need as quickly as possible."

Sanofi's amlitelimab is a fully human non-depleting monoclonal antibody that binds to OX40-ligand, a key immune regulator, and has the potential to be a first-in-class treatment for a range of immune-mediated diseases and inflammatory disorders, including moderate-to-severe atopic dermatitis, asthma and hidradenitis suppurativa.  By targeting OX40-ligand, amlitelimab aims to restore balance between pro-inflammatory and regulatory T cells.

According to Naimish Patel, M.D., Head of Global Development, Immunology and Inflammation, Research & Development, Sanofi, "We look forward to initiating the amlitelimab development program in scleroderma, an underserved condition with high morbidity and mortality rates and limited treatment options. With our knowledge about the underlying biology of the disease and amlitelimab's unique mechanism of action, we hypothesize that amlitelimab could have benefits on ILD and dermal fibrosis endpoints of scleroderma. To date, amlitelimab's potential has been encouraging and we look forward to further advancing this asset as part of our commitment to delivering best-in-class treatments that improve the lives of people living with chronic inflammatory diseases." 

Amlitelimab is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

The CONQUEST platform will be open to adding additional investigational agents starting in 2025. One of the key features of the platform is that new drugs can be added at any time with minimal regulatory overhead and an opportunity to take maximum benefit of the infrastructure of the platform. In addition, the anonymized patient-by-patient data from the placebo arm will be owned by the SRF and become an invaluable resource for the entire community. 

About CONQUEST

Using a model first created over a decade ago to accelerate oncology drug development, the SRF platform clinical trial is the first of its kind in autoimmune diseases. In its initial iteration, CONQUEST will focus on Interstitial Lung Disease secondary to Scleroderma (SSc-ILD) which manifests in patients as lung fibrosis. In the future, the CONQUEST platform will be expanded to address other manifestations of scleroderma. The ground-breaking effort, conceived and led by the SRF, will enroll patients across more than 130 centers in more than 22 countries. CONQUEST allows multiple therapies to be evaluated under a common trial infrastructure with a common control arm.

CONQUEST will initially enroll approximately 400 patients but with the advantages of the platform design, only approximately 134 patients will be randomized to the placebo arm (to receive standard-of-care).  Accordingly, the design is very patient-centric since patients generally enroll in a trial to get access to novel medicines.  However, the trial retains high statistical power for analysis of efficacy endpoints since the data from the placebo patients from all concurrently enrolled sub-protocols (e.g. Sanofi and Boehringer Ingelheim) will be pooled for efficacy analysis.

This efficiency is not available in traditional trials and is one of the features that underscore the uniqueness of CONQUEST.  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. Sanofi and Boehringer Ingelheim are the inaugural partners for CONQUEST, which was first announced on August 1, 2023.

About Scleroderma

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.

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.

About Boehringer Ingelheim

Boehringer Ingelheim is working on breakthrough therapies that transform lives, today and for generations to come. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. More than 52,000 employees serve over 130 markets in the two business areas, Human Pharma and Animal Health. Learn more at www.boehringer-ingelheim.com.  For more information about the FIBRONEER^™ program, please visit ClinicalTrials.gov.

BI -1015550 is an investigational therapy, which has not been approved for any proposed indication by any regulatory authority including the U.S. Food and Drug Administration (FDA). Boehringer Ingelheim is currently investigating its efficacy and safety profile. 

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SOURCE Scleroderma Research Foundation