Bellicum Announces Favorable Interim Event-Free Survival Data for Rivo-cel Treated Patients in Presentation at ASH 2018
Interim results suggest rivo-cel outcomes were comparable to a control study of transplants from matched unrelated donors, a requirement for product registration in
Company on track to report final results in 2019 followed by European regulatory submission
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
“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
A copy of this ASH 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
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.
About Bellicum Pharmaceuticals
Bellicum is a clinical stage biopharmaceutical company striving to deliver cures through controllable cell therapies. The Company’s next-generation product candidates are differentiated by powerful cell signaling technologies designed to produce more effective CAR-T, TCR and allogeneic T cell therapies. Its lead product candidate, rivo-celTM, is an allogeneic polyclonal T cell therapy that has shown promising clinical trial results in reducing leukemia relapse after a stem cell transplant. Bellicum’s lead GoCAR-T® candidate, BPX-601, is designed to be a more efficacious CAR-T cell product capable of overriding key immune inhibitory mechanisms. More information can be found at www.bellicum.com.
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research and development activities relating to rivo-celTM and rimiducid; the effectiveness of rivo-cel, its possible ranges of application and potential curative effects and safety in the treatment of diseases, including as compared to other treatment options and competitive therapies; the timing and success of our current and planned clinical trials, including the timing of receipt of data from such clinical trials and the timing of our reports of such data; the timing and success of regulatory filings; our ability to build a successful commercial organization in Europe; the presentation of our preclinical and clinical data at medical or scientific meetings and our cash uses and cash runway. Various factors may cause differences between Bellicum’s expectations and actual results as discussed in greater detail under the heading “Risk Factors” in Bellicum’s filings with the Securities and Exchange Commission, including without limitation our quarterly report on Form 10-Q for the three months ended September 30, 2018 and our annual report on Form 10-K for the year ended December 31, 2017. Any forward-looking statements that Bellicum makes in this press release speak only as of the date of this press release. Bellicum assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
Chad Rubin646-378-2947 email@example.com Media: Brad Miles917-570-7340 firstname.lastname@example.org -or- Amy Bonanno914-450-0349 email@example.com
Source: Bellicum Pharmaceuticals, Inc.