HOUSTON--(BUSINESS WIRE)--Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, today announced that the European Commission (EC) has granted orphan drug designations for both its lead T-cell therapy product candidate BPX-501 for treatment in hematopoietic stem cell transplantation (HSCT), and for activator agent rimiducid for the treatment of Graft vs. Host Disease (GvHD). Earlier this year, BPX-501 and rimiducid received orphan drug status from the U.S. Food and Drug Administration (FDA) as a combination therapy.
Bellicum also announced that it has met with regulatory authorities in Europe to discuss the potential approval pathway for BPX-501 and rimiducid for the treatment of immunodeficiency and GvHD following a haploidentical HSCT in pediatric patients with leukemias, lymphomas and rare inherited blood diseases who do not have a matched donor. These discussions have resulted in initial agreement regarding the Company’s development plans, subject to further refinement in a formal Protocol Assistance process that is available for orphan drug products.
Based on regulatory discussions, Bellicum believes that data from the European arm of its BP-004 trial, with a six-month follow-up time and expanded to enroll additional patients, could form the basis of Marketing Authorization Applications for BPX-501 and rimiducid. The EMA’s Committee for Medicinal Products for Human Use (CHMP) has agreed that review and approval under “exceptional circumstances” may be suitable, recognizing that a randomized trial may not be feasible in the pediatric setting. In place of a randomized trial, the Company intends to collect data from a concurrent observational study of allogeneic HSCT outcomes in the pediatric setting.
“We are pleased with the progress we have made toward defining an expedient pathway to approval of BPX-501 and of rimiducid for pediatric transplant patients in Europe,” said Tom Farrell, President and CEO of Bellicum. “We are now initiating discussions with the FDA, and expect to be able to provide additional guidance on the approval pathways during the fourth quarter.”
About EU Orphan Drug Designation
Orphan drug designation from the EC provides regulatory and financial incentives for companies to develop and market therapies that treat life-threatening or very serious conditions that affect no more than five in 10,000 people in the European Union (EU), and where no treatment is currently approved. In addition to a 10-year period of marketing exclusivity in the EU upon product approval, orphan drug designation provides fee waivers, protocol assistance, and marketing authorization under the centralized procedure granting approval in all EU countries.
About Exceptional Circumstances
The European Medicines Agency (EMA) can grant early market authorization to orphan drug products under exceptional circumstances. Exceptional circumstances can be granted for medicines that treat very rare diseases, or where controlled studies are impractical or not consistent with accepted principles of medical ethics.
About BPX-501 and the BP-004 Trial
BPX-501 is an adjunct T-cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe® safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. This enables physicians to more safely perform stem cell transplants by adding back BPX-501 engineered T cells to speed immune reconstitution and provide control over viral infections, without unacceptable risk of uncontrollable GvHD.
BP-004, a Phase 1/2 open-label dose escalation trial in pediatric patients with malignant and nonmalignant diseases, is evaluating whether BPX-501 T cells from a haploidentical donor, administered following a T-depleted HSCT, are safe and can enhance immune reconstitution. The trial is being conducted in European and U.S. pediatric centers. Interim results from the lead clinical site in Italy, presented at the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation in April, are available in the Events & Presentations section of bellicum.com.
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), and CAR T and TCR cell therapies. 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. Bellicum may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," “designed,” "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" 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: our research and development activities relating to rimiducid or CaspaCIDe; the effectiveness of rimiducid or CaspaCIDe, their possible range of application and potential curative effects and safety in the treatment of diseases; the timing and success of our clinical trials, including the rate and progress of enrollment in our BP-004 clinical trial or any observational studies; the timing of regulatory filings for BPX-501 and for rimiducid; the availability of early market authorization for BPX-501; our research and development activities relating to BPX-501; and the potential applications and effectiveness of our product candidate BPX-501, including as compared to other treatment options and competitive therapies. 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 annual report on Form 10-K for the year ended December 31, 2015. 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.