Cytori Therapeutics Inc (CYTX - NASDAQ - $0.36 )
Target Price: $5.00
Two-Year Follow-Up Data From the Original Scleroderma Trial (SCLERADEC-I) Shows Continuing Benefit With P-Values
Jason McCarthy, Ph.D.
Cytori announced data that supports a sustained benefit at two-years in the 12-patient SCLERADEC-I trial, which forms the basis of the current pivotal STAR trial in the same indication (hand dysfunction associated with scleroderma).
Recall that the SCLERADEC-I trial is an investigator-initiated, open-label, 12-patient trial, led by Dr. Brigitte Granel and Dr. Guy Magalon from the Assistance Publique des Hôpitaux de Marseille. A detailed manuscript describing the data from baseline to 12-month follow-up was recently published in the journal Rheumatology. The recently announced data extended to 24 months.
The two-year follow-up data in the SCLERADEC-I trial showed sustained improvement in four key endpoints: (1) Cochin Hand Function Score (CHFS); (2) Scleroderma Health Assessment Questionnaire; (3) Raynaud’s Condition Score (which assesses the severity of Raynaud’s phenomenon); and (4) hand pain, as assessed by a standard visual analogue scale.
Average values at the two-year timeframe for all four of these endpoints were statistically significantly and improved over the baseline. These were similar to improvements observed after one year (see below). These data, along with other endpoints from the SCLERADEC-I trial, will be presented by Dr. Magalon at the 4th Systemic Sclerosis World Congress to be held in Lisbon, Portugal in February 2016.
The U.S. Phase III/pivotal STAR trial (n=80) and EU SCLERADEC-II trial have both initiated patient enrollment and treated the first patients in both sites.
Conclusion: We are encouraged by the strong results seen in the original trial showing a sustained benefit at two years. We see compelling value for patients and the potential for a premium pricing basis for the therapy.
The STAR trial. The trial will evaluate a single administration of ECCS-50 in patients with scleroderma of the hands. The primary endpoint is the Cochin Hand Function Score (a validated measure of hand function) to evaluate the safety and efficacy six months after a single dose of ECCS-50 versus placebo. Other key efficacy endpoints include the assessment of Raynaud's phenomenon and overall health-related quality of life. Follow-up will be 48 weeks after the trial. The STAR trial was developed based on the previously published trial, SCLERADEC-I, which was conducted in France. Results showed significant improvement in hand function, pain, and Raynaud's severity in patients with scleroderma affecting the hands. Cytori has supported the trial in the form of devices, consumables, and training.
The STAR trial was based on the strong data generated after one year from the SCLERADEC-I trial. That data showed a significant decrease from baseline of 51.3% (P < 0.001) for Cochin Hand Function Scale score, 63.2% (P < 0.001) for RP severity, and 46.8% (P = 0.001) for quality of life (Scleroderma Health Assessment Questionnaire). Significant improvements of finger oedema, skin sclerosis, motion and strength of the hands, and the vascular suppression score were also noted. The reduction in hand pain approached statistical significance (P = 0.052). The questionnaire revealed a benefit in daily activities, housework, and social activities.
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