- 82% (41 of 50) of patients
achieved complete remission or complete remission with incomplete
blood count recovery in interim analysis of Novartis study
(ELIANA)
- ELIANA, the first global CAR T
cell trial, will be the basis of a Biologics Licensing Application
(BLA) to the FDA in early 2017
- Novartis pivotal data from
ELIANA is supported by CTL019 US multicenter trial (ENSIGN) as well
as earlier single site trial in r/r pediatric and young adult
patients with B-cell ALL
- The University of Pennsylvania
(Penn) will present findings evaluating overall response to CTL019
therapy among r/r DLBCL patients with poor prognosis
Basel, December 4, 2016
- Findings from a Novartis clinical trial
(ELIANA) evaluating efficacy and safety of CTL019, an
investigational chimeric antigen receptor T cell (CAR T) therapy,
in relapsed/refractory (r/r) pediatric and young adult patients
with B-cell acute lymphoblastic leukemia (ALL) will be presented
today during an oral session at the 58th American Society of
Hematology (ASH) annual meeting (Abstract #221, December 3,
4:00-5:30 p.m.). The global Phase II study found that 82% (41 of
50) of infused patients achieved complete remission or complete
remission with incomplete blood count recovery at three months post
CTL019 infusion. For all patients with complete remission, no
minimal residual disease was detected. In addition, the estimated
relapse-free rate among responders was 60% (95% CI: 36, 78) six
months after infusion with CTL019.[1] The results set the
stage for filing CTL019 with the US Food and Drug Administration
(FDA) in early 2017 for pediatric and young adult patients with r/r
B-cell ALL.
ELIANA is the first pediatric global CAR T cell
registration trial with study enrollment having occurred across 25
centers in the US, EU, Canada, Australia and Japan. Forty-eight
percent of patients in ELIANA experienced grade 3 or 4 cytokine
release syndrome (CRS), a known complication of the investigational
therapy that may occur when the engineered cells become activated
in the patient's body. CRS was managed on a global scale using
prior site education with implementation of the CRS treatment
algorithm. There were no deaths due to CRS. Fifteen percent of
patients experienced grade 3 neurological and psychiatric events
including encephalopathy and delirium, with no grade 4 events
seen.[1]
"These global multicenter trial data build on
earlier encouraging research conducted at a single trial site, and
advance the case for CTL019 as a potential treatment for children
and young adults with relapsed or refractory B-cell ALL," said lead
investigator Stephan Grupp, MD, PhD, the Yetta Deitch Novotny
Professor of Pediatrics at the Perelman School of Medicine at the
University of Pennsylvania (Penn), and Director of the Cancer
Immunotherapy Frontier Program at the Children's Hospital of
Philadelphia (CHOP).
In addition to filing CTL019 for approval with the
FDA in early 2017, Novartis plans to file with the European
Medicines Agency (EMA) later in 2017. The investigational therapy
received PRIME (PRIority MEdicines) designation from the EMA
earlier this year.
"This first-of-its-kind trial represents exciting
progress toward our goal of helping children and young adults with
relapsed or refractory B-cell ALL, a patient population with an
urgent need for new treatment options," said Bruno Strigini, CEO,
Novartis Oncology. "We are committed to advancing CTL019 and look
forward to working closely with the FDA and EMA in the coming
months."
Dr. Shannon Maude from CHOP will give a poster
presentation highlighting data from ENSIGN, the first US
multicenter Phase II trial for CTL019 in pediatric and young adults
with B-cell ALL (Abstract #2801, December 4, 6:00-8:00 p.m.).[2] A
separate poster presentation will also highlight an ongoing Phase
IIa study led by Penn which investigated the efficacy and safety of
CTL019 in poor prognostic groups of relapsed or refractory diffuse
large B-cell lymphoma (DLBCL) patients (Abstract #3026, December 4,
6:00-8:00 p.m.).[3]
Because CTL019 is an investigational therapy, the
safety and efficacy profile has not yet been established. Access to
investigational therapies is available only through carefully
controlled and monitored clinical trials. These trials are designed
to better understand the potential benefits and risks of the
therapy. Because of uncertainty of clinical trials, there is no
guarantee that CTL019 will ever be commercially available anywhere
in the world.
Disclaimer
The foregoing release contains forward-looking statements that can
be identified by words such as "will," "investigational," "build
on," "encouraging," "advance the case," "potential," "exciting,"
"goal," "committed," "look forward," "yet," or similar terms, or by
express or implied discussions regarding potential marketing
approvals for CTL019, or regarding potential future revenues from
CTL019. You should not place undue reliance on these statements.
Such forward-looking statements are based on the current beliefs
and expectations of management regarding future events, and are
subject to significant known and unknown risks and uncertainties.
Should one or more of these risks or uncertainties materialize, or
should underlying assumptions prove incorrect, actual results may
vary materially from those set forth in the forward-looking
statements. There can be no guarantee that CTL019 will be submitted
or approved for sale in any market, or at any particular time. Nor
can there be any guarantee that CTL019 will be commercially
successful in the future. In particular, management's expectations
regarding CTL019 could be affected by, among other things, the
uncertainties inherent in research and development, including
unexpected clinical trial results and additional analysis of
existing clinical data; unexpected regulatory actions or delays or
government regulation generally; the company's ability to obtain or
maintain proprietary intellectual property protection; general
economic and industry conditions; global trends toward health care
cost containment, including ongoing pricing pressures; unexpected
safety, quality or manufacturing issues, and other risks and
factors referred to in Novartis AG's current Form 20-F on file
with the US Securities and Exchange Commission. Novartis is
providing the information in this press release as of this date and
does not undertake any obligation to update any forward-looking
statements contained in this press release as a result of new
information, future events or otherwise.
About Novartis
Novartis provides innovative healthcare solutions that address the
evolving needs of patients and societies. Headquartered in Basel,
Switzerland, Novartis offers a diversified portfolio to best meet
these needs: innovative medicines, eye care and cost-saving generic
pharmaceuticals. Novartis is the only global company with leading
positions in these areas. In 2015, the Group achieved net sales of
USD 49.4 billion, while R&D throughout the Group amounted to
approximately USD 8.9 billion (USD 8.7 billion excluding impairment
and amortization charges). Novartis Group companies employ
approximately 118,000 full-time-equivalent associates. Novartis
products are available in approximately 180 countries around the
world. For more information, please visit
http://www.novartis.com.
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References
[1] Grupp, Stephen A. et al. Analysis of a Global Registration
Trial of the Efficacy and Safety of CTL019 in Pediatric and Young
Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL).
Session 614, Saturday, December 3, 5:00 p.m. PST. 58th American
Society of Hematology Annual Meeting and Exposition: Abstract
221.
[2] Maude, Shannon L. et al. Efficacy and Safety of CTL019 in the
First US Phase II Multicenter Trial in Pediatric
Relapsed/Refractory Acute Lymphoblastic Leukemia: Results of an
Interim Analysis. Session 614, Sunday, December 4, 6:00 - 8:00 p.m.
PST. 58th American
Society of Hematology Annual Meeting and Exposition: Abstract
2801.
[3] Schuster, Stephen J. et al. Treatment with Chimeric Antigen
Receptor Modified T Cells Directed Against CD19 (CTL019) Results in
Durable Remissions in Patients with Relapsed or Refractory Diffuse
Large B Cell Lymphomas of Germinal Center and Non-Germinal Center
Origin, "Double Hit" Diffuse Large B Cell Lymphomas, and
Transformed Follicular to Diffuse Large B Cell Lymphomas. Session
626, Sunday, December 4, 6:00 - 8:00 pm PST. 58th
American Society of Hematology Annual Meeting and Exposition:
Abstract 3026.
# # #
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