On December 1, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported late interim results from its BP-004 European registration trial, suggesting that adding rivo-celTM (rivogenlecleucel, formerly BPX-501) to stem cell transplants in patients who lack a matched donor may deliver comparable outcomes to matched unrelated donor (MUD) transplants (Press release, Bellicum Pharmaceuticals, DEC 1, 2018, View Source [SID1234531767]). The data were reviewed in a presentation today at the 60th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2018) by Mattia Algeri, M.D., Cell and Gene Therapy Unit, Ospedale Bambino Gesù, Rome, Lazio, Italy.

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The interim data show a 90.9 percent event-free survival (EFS) rate at 180 days for patients treated with rivo-cel (n=166) after a stem cell transplant from a haploidentical, or partially matched, donor. These results appear comparable to an interim evaluation of patients who received a MUD transplant in the concurrently-run observational study (n=91), demonstrating an 87.7 percent EFS rate at 180 days. The Company expects to report final comparison results from both study populations and file for approval in Europe in 2019. Demonstrating non-inferiority to MUD transplantation is the primary study objective required for rivo-cel product registration in Europe.

"While a stem cell transplant is an established, often curative treatment option for children with cancers and hereditary blood diseases, the majority of patients are unable to find an HLA-matched related donor, which is associated with the best outcomes," commented Dr. Algeri. "While matched unrelated donor transplants are an important option, only half of patients can identify a match. Further, it can take up to several months to do so, a delay that is problematic for many transplant patients. These data suggest that rivo-cel treatment may enable haploidentical stem cell transplant with overall outcomes similar to MUD, making it an attractive alternative for many of these patients."

Study Design and Highlights

The BP-004 trial is a prospective, multicenter, European Phase 1/2 trial in pediatric patients with malignant or nonmalignant disorders. Patients received rivo-cel within a few weeks following a haplo-transplant. No post-transplant Graft versus Host Disease (GvHD) prophylaxis was used. Patients who developed visceral GvHD or were refractory to standard of care treatments were eligible to receive rimiducid. The primary endpoint was EFS at day 180 (events included transplant-related mortality or non-relapse mortality for malignant patients, severe GvHD, and life-threatening infections).

Rivo-cel was well-tolerated, with few patients experiencing non-GvHD adverse events considered to be possibly or probably related to rivo-cel. Overall GvHD rates were low, with few cases of high-grade acute GvHD or chronic GvHD. The majority of patients with visceral GvHD or who were refractory to standard of care treatment for GvHD responded to rimiducid.

The observational comparator trial (C/CP-004) was designed to collect both prospective and retrospective data from pediatric patients with either malignant or nonmalignant disease who underwent a matched unrelated donor transplant during the same time period and at the same treatment centers where the BP-004 rivo-cel study was conducted.

"These interim data increase our confidence that rivo-cel may be an approvable new treatment option for transplant patients with leukemias, lymphomas and genetic blood diseases in Europe upon final data read-out in 2019," commented Rick Fair, Bellicum President & CEO. "If we continue to demonstrate that rivo-cel enables patients without a matched donor to achieve outcomes comparable to a MUD transplant, that would be very clinically meaningful and would fulfill a requirement for European approval. We look forward to reporting final six-month results for all patients in 2019. We also plan to continue to follow these patients to evaluate the impact of rivo-cel in preventing relapse and extending overall survival, particularly in patients with leukemia."

A copy of this ASH (Free ASH Whitepaper) presentation will be made available in the Abstracts & Presentations section of the Company’s website. The Company expects to report topline final results in early 2019 followed by publication of a comprehensive data set.

Analyst and Investor Luncheon Event and Webcast
Bellicum will host a live and webcast analyst and investor luncheon event on December 3, 2018 at 12:00 p.m. – 1:30 p.m. PST in San Diego, CA. Featured speakers include Dr. Alice Bertaina, Associate Professor of Pediatrics (Stem Cell Transplantation), Lucile Packard Children’s Hospital at the Stanford School of Medicine, as well as Bellicum senior management. A webcast replay of the event will be available in the News & Events section of the Bellicum website, and available for at least two weeks following the event.

About Rivo-cel (BPX-501)
Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s antiviral benefits may also reduce morbidity and mortality in patients susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and genetic blood disease patients following a haploidentical stem cell transplant.