Bellicum Pharmaceuticals Announces Rivo-cel™ Achieves Primary Endpoint in Pediatric Registrational Trial

HOUSTON, TX - Jul 8, 2019 - Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, today announced that the primary endpoint of event free survival at 180 days in its BP-004 European registration trial for rivo-cel (rivogenlecleucel) has been achieved. Data from this trial will form the basis of an anticipated submission of European Marketing Authorisation Applications (MAAs) for rivo-cel and rimiducid in support of potential regulatory approval.
These clinical results indicate that adding rivo-cel to stem cell transplants in patients lacking a matched donor delivered comparable outcomes to HLA-matched unrelated donor (MUD) transplants. These data suggest that, if approved, rivo-cel could become an attractive treatment option for the 20-25% of stem cell transplant patients who lack an HLA-matched donor. In addition, rivo-cel may also benefit patients who urgently need a curative transplant and are at risk from waiting to search for an HLA-matched unrelated donor, which can take several months.
In the final analysis, Event Free Survival (EFS) at 180 days of 90.9% (95% CI: 84.8, 94.6) in the BP-004 study was non-inferior to EFS at 180 days of 89.9% (95% CI: 82.4, 94.3) from the comparative C/CP-004 study of patients who received a MUD HLA 10/10 transplant. A 10/10 matched transplant represents a highly compatible match based on a standardized assessment of the histocompatibility of 10 surface protein antigens on white blood cells. Further, key secondary endpoints, tested in a hierarchical fashion, including transplant-related mortality, graft-versus-host-disease (GvHD), relapse-free survival, and disease-free survival also demonstrated non-inferiority. Finally, clinical outcomes in patients given rimiducid because of visceral GvHD or refractoriness to standard treatment were positive and consistent with previously presented data. Management is planning to present additional results from the study, including longer term follow-up data, at an appropriate future medical meeting.
“These impressive results confirm the clinical value rivo-cel may deliver to patients undergoing HSCT from a haploidentical, or partially matched donor,” said Principal Investigator Franco Locatelli, M.D., Ph.D., Director of the Department of Hematology and Oncology and Cell/Gene Therapy at Ospedale Pediatrico Bambino Gesù in Rome, Italy. “Historically, outcomes of stem cell transplantation were much better when patients received a graft from an HLA-matched donor. I believe the results reported today establish the addition of rivo-cel to a haploidentical HSCT as a potential new standard of care for pediatric patients with high-risk hematologic malignancies and non-malignant disorders who need stem cell transplantation but do not have a readily available HLA-matched donor.” 
“These final results support our belief that rivo-cel, if approved, may be a transformational new treatment option for pediatric patients with leukemias, lymphomas and genetic blood diseases,” said Rick Fair, President & CEO of Bellicum Pharmaceuticals. “Adding rivo-cel to haploidentical stem cell transplantation improved the curative outcomes of these procedures, and may enable them to become more broadly available to patients without access to an HLA-matched donor.”
In light of this positive pivotal trial outcome and planned MAA filings in Europe, Bellicum is actively seeking a strategic partner for rivo-cel in anticipation of a potential commercial launch.
Study Design

The BP-004 trial (clinicaltrials.gov identifier NCT02065869) was designed as a prospective, multicenter, European Phase 1/2 trial that enrolled pediatric patients with malignant or nonmalignant disorders. Patients received rivo-cel within a few weeks following a haplo-transplant and 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 composite primary endpoint was EFS events at day 180 and included transplant-related mortality for non-malignant patients or non-relapse mortality for malignant patients, severe GvHD, and life-threatening infections.

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 (MUD) HLA 10/10 transplant during the same time period and at the similar treatment centers where the BP-004 rivo-cel study was conducted.

For the primary endpoint, the EFS rate in patients treated with rivo-cel at 180 days in the BP-004 trial was 90.9% (95% CI: 84.8, 94.6). The EFS rate in patients treated with hematopoietic stem cell transplant (HSCT) with a MUD in the observational study (C/CP-004) at 180 days was 89.9% (95% CI: 82.4, 94.3). The difference in EFS rates at 180 days was estimated to be 1.0% (95% CI: -6.4, 8.4). This estimate was within the pre-specified non-inferiority margin of 10%, indicating statistical non-inferiority for treatment with rivo-cel was achieved.

About Rivo-cel (BPX-501)
Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce the rate of 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 and allogeneic T cell therapies. 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. Bellicum’s rivo-cel product candidate is an allogeneic polyclonal T cell therapy that has shown promising clinical trial results in reducing leukemia relapse after a stem cell transplant. More information can be found at www.bellicum.com.
Forward-Looking Statement
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Bellicum may, in some cases, use terms such as “potential,” “continue,” “designed,” “expects,” “plans,” “intends,” “may,” “will,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the timing of the regulatory filings for rivo-cel and rimiducid in the EU, the possible ranges of application and potential safety and curative effects of rivo-cel and rimiducid in the treatment of diseases, including as compared to other treatment options and competitive therapies, and the potential for rivo-cel availability to increase use of haploidentical HSCT or otherwise impact the standard of care for patients.  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 March 31, 2019 and our annual report on Form 10-K for the year ended December 31, 2018. 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.
Source: Bellicum Pharmaceuticals
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