Paracrine Announces Publication of STAR Trial, Including Improvement in Hand Function and Quality of Life in Patients With Diffuse Cutaneous Scleroderma

FDA-approved STAR II trial designed and powered based on these findings

SAN DIEGO--(BUSINESS WIRE)--Paracrine, Inc., announced today the online publication (ahead of print) of the findings from the STAR I trial in Arthritis & Rheumatology Journal, the official journal of the American College of Rheumatology. The manuscript by Khanna et al. is entitled “Adipose-Derived Regenerative Cell Transplantation in Systemic Sclerosis: Scleroderma Treatment with Celution Processed Adipose Derived Regenerative Cells - a Randomized Clinical Trial” (https://onlinelibrary.wiley.com/doi/epdf/10.1002/art.42133).

The STAR I trial was a randomized placebo-controlled trial that evaluated the improvement in hand function in patients with either diffuse cutaneous scleroderma (dcSSc) or limited cutaneous scleroderma (lcSSc) with hand dysfunction who were treated with autologous Adipose-Derived Regenerative Cell (ADRC) Therapy. The primary endpoint of this study was change in hand function at 24 and 48 weeks, assessed using a validated patient reported outcome measure, the Cochin Hand Function Scale (CHFS).

Additional endpoints included changes in Health Assessment Questionnaire Disability Index (HAQ-DI) and the assessment of general health related quality of life (EQ-5D). The study design included a pre-specified subgroup analyses of patients with either dcSSc or lcSSc.

“The initial STAR I trial, designed and conducted by our predecessor, was an important feasibility study that yielded valuable actionable information on optimal patient selection and endpoints,” noted Dr. Alex Milstein, Chief Clinical Officer and EVP of Clinical Affairs for Paracrine. “I would like to express our gratitude to the patients, investigators, and clinical trial staff who made this trial possible.”

While the STAR I trial did not meet its primary endpoint, it yielded a number of important findings that have helped instruct the design and endpoint selection for the STAR II trial—an appropriately powered, FDA-approved, phase III pivotal trial in patients with dcSSc:

• Trends of efficacy demonstrating improvement in CHFS were observed in dcSSc patients at 24 weeks (p=0.111) and 48 weeks (p=0.069)
• ADRC-treated patients with dcSSc reported Improvement in the Health Assessment Questionnaire Disability Index (HAQ-DI) at 48 weeks (p=0.044)
• Majority of patients with dcSSc (52%) reported improvement greater than the minimal clinically important difference for both CHFS and HAQ-DI compared with 16% in the placebo group (nominal p=0.016, post-hoc analysis)
• Majority of patients with dcSSc (63%) exhibited improvement in HAQ-DI at 48 weeks that was greater than the established Minimal Clinically Important Difference, compared to only 26% in the placebo-treated arm (nominal p value = 0.019, post-hoc analysis)
• ADRC-treated patients with dcSSc reported improvement in Health-Related Quality of Life Survey (EQ-5D 3L) at 48 weeks (p=0.005)
• Beneficial effects in dcSSc patients were maintained over 48 weeks
• The procedure and injections were well tolerated, and there were no new safety concerns

“The consistency and durability of clinically meaningful improvement in patients with diffuse cutaneous scleroderma in this trial supports our hypothesis that a single treatment with ADRCs is safe and feasible, improves the ability of patients to perform daily activities, and improves their quality of life,” commented Dr. Dinesh Khanna, Professor of Rheumatology at the University of Michigan, and Co-Principal Investigator for both the STAR I and the STAR II Trials.

“Despite the significance of hand dysfunction in patients with systemic scleroderma, there are few treatments with demonstrated effectiveness that specifically address this problem,” continued Dr. Khanna. “Based on these highly consistent and promising findings in patients with diffuse cutaneous scleroderma, we are planning to initiate the STAR II Trial by this summer to conclusively determine these benefits for this otherwise medically unaddressed condition.”

In January 2022, the FDA granted an Investigational Device Exemption (IDE) approval for Paracrine to conduct a pivotal trial in patients with diffuse cutaneous scleroderma titled Scleroderma Treatment with Celution® Processed Adipose Derived Regenerative Cells: A Randomized, Double-Blind, Placebo-Controlled Study—the “STAR II trial.” The primary endpoint will be the change in Cochin Hand Function Scale at 26 weeks.

About Paracrine

Paracrine, Inc. is an emerging biotechnology company developing the world’s first device-based pleiotropic cell therapy platform to treat debilitating chronic conditions. Paracrine’s cell therapy platform bears the promise of cost-effectively addressing common underlying pathology of debilitating chronic diseases due to its multi-modal mechanisms of action. The company is advancing several late-stage IDE clinical trials in the U.S. based on robust prior data.

About Celution® System

The Celution® System is a proprietary medical device designed to process human adipose tissue to extract, isolate, and concentrate a mixture of Adipose Derived Regenerative Cells (ADRCs) for subsequent reimplantation in the same patient.

About STAR I Trial

The STAR trial was a prospective, randomized, double-blind, placebo-controlled, multi-center device trial (RCT) to assess safety and efficacy of Celution® System processed ADRCs delivered by subcutaneous injection for the treatment of impaired hand function due to systemic scleroderma.

The trial enrolled 88 patients, 40 randomized to the placebo arm and 48 to the ADRC arm. The trial pre-specified subsets of limited and diffuse scleroderma patients, with the diffuse scleroderma subset comprised of 51 patients, of which 32 were enrolled in the ADRC-treated group and 19 in the control arm. The majority of subjects (85%) were women, the mean age was 53 years, and the average duration of disease was 13 years.

About STAR II Trial

STAR II is a Phase III Pivotal Trial designed to provide a robust data set on the safety and efficacy of Adipose Derived Regenerative Cells (ADRCs) in the treatment of patients with hand dysfunction due to diffuse cutaneous scleroderma.

The trial is designed as a prospective, randomized (1:1), placebo-controlled, double-blind, multi-center study. The primary endpoint is the change in Cochin Hand Function Scale (CHFS) at 26 weeks. CHFS is a validated patient-reported outcome for the assessment of hand function in patients with scleroderma.

About Cochin Hand Function Scale (CHFS)

The Cochin Hand Function Scale (CHFS) is a validated patient-reported outcome for assessment of hand function in systemic scleroderma. The instrument assesses five domains rated from 0 (no problems performing the task) to 5 (impossible to perform) with a total score range from 0 to 90. The survey consists of 18 questions that indicate the ability to perform activities of daily living, including in the kitchen, dressing, bathroom, office, and miscellaneous. A lower score indicates a greater ability to complete the task. Diffuse Cutaneous Scleroderma patients with a CHFS > 20 are eligible for the STAR II trial.

About Scleroderma

Scleroderma is a rare and chronic connective tissue disease generally classified as an autoimmune rheumatic disorder. The word “Scleroderma” is derived from two Greek words: “sclera,” which means hard, and “derma,” which means skin, as hardening of the skin is one of the most visible manifestations of the disease.

Diffuse cutaneous scleroderma (also known as systemic sclerosis) is a debilitating chronic disease affecting predominantly women and leading to significant disability, including hand dysfunction.

An estimated 200,000 Americans have scleroderma, about 80,000 of whom have the systemic form of the disease, known as systemic sclerosis (SSc). SSc is further sub-classified as diffuse cutaneous or limited cutaneous SSc. An estimated 30,000 patients have diffuse cutaneous SSc, the more severe disease with significant hand dysfunction.

Systemic Sclerosis contributes to hand impairment through inflammatory arthritis or inflammation of the joints, joint contractures, Raynaud’s Phenomenon (RP, skin discoloration resulting from narrowing of the blood vessels in response to cold, emotional upset, or stress), digital ulcers, puffy hands, skin fibrosis over the fingers and hands, and calcinosis (calcium deposits in the soft tissues of the hand). These manifestations contribute to difficulty with occupational and daily living activities, impacting quality of life. There are currently no effective treatments available for hand impairment.

Note: The Celution® System and ADRCs are not currently approved in the U.S.

Cautionary Statement Regarding Forward-Looking Statements

This press release includes forward-looking statements regarding events, trends and business prospects, which may affect our future operating results and financial position. Such statements, including, but not limited to, those regarding our ability to initiate and execute the pivotal STAR II Trial, are forward looking statements. Such statements are subject to risks and uncertainties that could cause our actual results and financial position to differ materially. Some of these risks include clinical and regulatory uncertainties, the challenges inherent in convincing physicians and patients to adopt the new technology, dependence on third party performance, performance and acceptance of our products, and other risks and uncertainties. Paracrine assumes no responsibility to update or revise any forward-looking statements contained in this press release to reflect events, trends or circumstances after the date of this press release.

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