Clinical Trials

Date: 2017-12-11

Type of information: Presentation of results at a congress

phase: 1b

Announcement: presentation of results at the 59th American Society of Hematology (ASH) Annual Meeting in Orlando

Company: Caelum Biosciences (USA - NY)

Product: CAEL-101 (mAb 11-1F4)

Action mechanism:

• monoclonal antibody. CAEL-101 is a chimeric fibril-reactive monoclonal antibody (mAb) that has completed a Phase 1a/1b clinical trial at Columbia University for the treatment of patients with amyloid light chain (“AL”) amyloidosis. While current treatment with chemotherapy is aimed at reducing production of the amyloid-forming light-chain protein, CAEL-101 attempts to reduce and / or eliminate the amyloid deposits.

Disease: amyloid light chain amyloidosis

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

• The Phase 1a/1b study is examining the tolerance, safety, pharmacokinetics and possible clinical benefit of CAEL-101, a chimeric fibril-reactive monoclonal antibody, in patients with AL amyloidosis. CAEL-101 is administered to patients through a single intravenous infusion in the Phase 1a, and one weekly infusion for four weeks during the Phase 1b. The first patient received the starting dose and, if tolerated, the following patients received progressively higher doses. When trial investigators reached the maximum tolerated dose without toxicity, investigators enrolled another six patients to receive the same dose. (NCT02245867)

Latest news:

• • On December 11, 2017, Caelum Biosciences, a Fortress Biotech company focused on developing treatments for rare and life-threatening diseases, announced full Phase 1a/1b clinical data demonstrating CAEL-101’s (mAb 11-1F4) ability to bind to light-chain amyloid fibrils and achieve early and clinically efficacious organ responses in patients with relapsed and refractory amyloid light chain amyloidosis. The data were presented by Columbia University on December 10th in an oral session at the 59th American Society of Hematology Annual Meeting.
• Key Efficacy and Safety Findings: Twenty-seven patients were treated with CAEL-101 in this open-label, dose-escalation trial. In the Phase 1a trial, CAEL-101 was administered to eight patients via a single IV infusion at week one. In the Phase 1b trial, CAEL-101 was administered to 19 patients via one weekly IV infusion for four weeks. Trial investigators at Columbia determined the study achieved its primary objective of establishing maximum tolerated dose of up to 500mg/m2 of CAEL-101.
• Trial investigators presented organ response rates in the Phase 1a and the Phase 1b, with 63 percent (14 of 24) overall organ response rate, 67 percent (8 of 12) overall cardiac response rate and 50 percent (5 of 10) overall renal response rate.1 Early organ response was demonstrated in a high-mortality population (21 days median time to cardiac response in Phase 1b; 28 days median time to renal response in Phase 1b2).
• Trial investigators found that CAEL-101 achieved and demonstrated organ response at multiple points in time throughout the duration of treatment; all patients showed an organ response or were stable, and no patients showed organ progression. Organ response independent of a chemotherapy-free light chain response was demonstrated. No drug-related grade 4 or 5 adverse events or dose-limiting toxicities were seen in the trial. There was no mortality during the study. The investigators followed patients beyond the study and reported an overall survival rate of 93 percent (median follow-up period of 18.6 months). Michael Spector, President and Chief Executive Officer of Caelum said that “according to investigators, CAEL-101 has improved organ response independent of the free light chain response of chemotherapy. In addition, an enlarged liver returned to normal in a patient who had a hematologic response eight months after their organ response. We believe these data underscore CAEL-101’s potential to be a best-in-class treatment for AL amyloidosis, and provide clear signals that support advancement into a Phase 2b/3 trial in the second half of 2018.” • On May 4, 2017, Fortress Biotech announced that study sponsor Columbia University has dosed the final patient in the Phase 1b clinical trial of CAEL-101, in development by Fortress subsidiary Caelum Biosciences, for the treatment of amyloid light chain (“AL”) amyloidosis. Caelum expects to readout preliminary Phase 1b data mid-2017, and full data by the end of the year. Interim Phase 1a/1b data presented by Columbia at the American Society of Hematology’s (ASH) 58th Annual Meeting demonstrated that CAEL-101 is safe and well-tolerated with no drug-related adverse events or dose-limiting toxicity up to 500mg/m2. A single infusion of CAEL-101, or one weekly infusion for four weeks, provided early and sustained organ response in cardiac, renal, gastrointestinal, skin and soft tissue. Interim clinical efficacy data showed that CAEL-101 promoted amyloid resolution in 67 percent of patients (63 percent in Phase 1a; 70 percent in Phase 1b).

Is general: Yes