Bellicum Announces BPX-501 European Regulatory Updates
Company receives orphan drug designations in the
Bellicum plans to pursue EU approval based on ongoing BP-004 clinical trial under “exceptional circumstances” provision
Bellicum also announced that it has met with regulatory authorities in
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
“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
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
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
About Bellicum
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.
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
"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
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Source:
Investors:
Bellicum Pharmaceuticals, Inc.
Alan Musso,
832-384-1116
CFO
amusso@bellicum.com
or
Media:
BMC
Communications
Brad Miles, 646-513-3125
bmiles@bmccommunications.com