Bellicum Announces Interim Results Showing Rimiducid Controlled GvHD in Patients Treated with Rivo-cel Following a Stem Cell ...
December 01 2018 - 2:00PM
Experience to date in clinical study shows high clinical
response rates for patients with visceral GvHD or GvHD that was
refractory to standard of care treatment
Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in
developing novel, controllable cellular immunotherapies for cancers
and orphan inherited blood disorders, today announced interim
results from two multicenter trials of patients treated with
rivo-celTM (rivogenlecleucel, formerly BPX-501) following a
haploidentical stem cell transplant showing that rimiducid
administration led to resolution of most cases of advanced Graft
versus Host Disease (GvHD) or those who were refractory to standard
of care treatment. The data were reviewed in a presentation today
at the 60th Annual Meeting of the American Society of Hematology
(ASH 2018) by Principal Investigator Reem Elfeky, M.D., UCL Great
Ormond Street Hospital, London.
“These compelling data demonstrate the activity
of rimiducid in combination with the CaspaCIDe® safety switch in
the treatment of advanced or refractory GvHD based on the totality
of our experience to date in pediatric patients who received
rivo-cel,” said Rick Fair, Bellicum’s President & CEO. “The
combination of rivo-cel and rimiducid may represent an exciting new
way to more safely utilize allogeneic T cells to both clear
residual cancer cells and improve control over infection following
a stem cell transplant. These data also represent the strongest
clinical validation to date of our industry-leading molecular
switch technology.”
Study Design and Results
Patients in E.U. and U.S. multicenter trials
were treated with rivo-cel (donor T cells genetically modified with
the CaspaCIDe safety switch) following an αβT-cell receptor and
B-cell depleted haploidentical stem cell transplant. Patients had
malignant or nonmalignant disorders. No post-transplant
pharmacologic GvHD prophylaxis was used.
Patients who developed visceral GvHD or were
refractory to standard of care treatment were eligible to receive
one dose of rimiducid (protocol later adjusted to allow for up to
three doses of rimiducid). Of the 238 patients evaluable for GvHD,
54 patients (22.7%) developed acute GvHD (29 Grade II-IV; 7 Grade
III-IV). 10 patients (5.6%) developed mild-to-severe chronic GvHD;
8 patients developed moderate-to-severe chronic GvHD. Results are
summarized as follows:
- 24 patients met the rimiducid efficacy-evaluable population
definition
- The best overall clinical response (CR/PR) within 7 days post
rimiducid was an overall clinical response rate of 70% (16
responders)
- A complete response (CR) or partial response (PR) to rimiducid
was observed in 9 and 7 patients, respectively
- Median time to initial response was 1 day (1-4 days)
- 4 patients who achieved a partial or non-evaluable response
within the first 7 days following rimiducid administration went on
to achieve a complete response within 30 days following rimiducid
administration
- Nine patients (42.9%) received a second dose of rimiducid
- The majority of patients were in partial response or no
response at time of second dose of rimiducid
- 2 patients in partial response at time of second dose of
rimidicid went on to achieve a complete response
- The median duration of standard GvHD treatment prior to
rimiducid administration was 68 days
Differential Depletion of Activated T
Cells
In a separate poster session, researchers
presented data showing highly activated T cells express higher
levels of iC9, making them more sensitive to rimiducid. This
results in selectively depleting more GvHD-causing rivo-cel cells
while sparing less alloreactive rivo-cel cells to continue to
provide immunity and graft versus leukemia effect (GvL).
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 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.
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.
Forward-Looking StatementThis
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,
rimiducid, CaspaCIDe®, the effectiveness of rivo-cel and rimiducid,
their 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.
Source: Bellicum Pharmaceuticals
Investors:
Chad Rubin
646-378-2947
crubin@soleburytrout.com
Media:
Brad Miles
917-570-7340
bmiles@soleburytrout.com
-or-
Amy Bonanno
914-450-0349
abonanno@soleburytrout.com
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