Novartis announces CTL019 data published in NEJM demonstrating efficacy in certain patients with acute lymphoblastic leukemia (ALL)
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Novartis announces CTL019 data published in NEJM demonstrating efficacy in
certain patients with acute lymphoblastic leukemia (ALL)
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* Preliminary study results show 27 of 30 pediatric and adult patients with
relapsed/refractory (r/r) ALL (90%) experienced complete remissions with
personalized cell therapy, CTL019[1]
* Largest published cohort to date for CTL019, which served as the basis for
recent Breakthrough Therapy designation from the FDA[2]
* Sustained remissions of up to two years in r/r ALL patients with six-month
event-free survival of 67% and overall survival of 78%[1]
* Novartis and Penn have exclusive global collaboration to research, develop
and commercialize CAR T cell therapies for the investigational treatment of
cancers
Basel, Switzerland, October 15, 2014 - Novartis and the University of
Pennsylvania's Perelman School of Medicine (Penn) today announced preliminary
results from two pilot clinical trials published in The New England Journal of
Medicine (NEJM) evaluating the efficacy and safety of CTL019 in patients with
relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The studies,
conducted by Penn, demonstrated that 27 of 30 pediatric and adult patients, or
90%, experienced complete remissions with the investigational chimeric antigen
receptor (CAR) therapy CTL019[1].
"These interim results, which supported the recent FDA Breakthrough Therapy
designation, reinforce the potential CTL019 has as a life-saving therapy for
patients with relapsed/refractory ALL," said Usman Azam, Global Head, Cell &
Gene Therapies Unit, Novartis Pharmaceuticals. "These studies are another
promising development in CTL019's history. With each new CTL019 milestone, we
are one step closer to potentially offering these seriously ill patients an
additional treatment option."
These data build on earlier research findings and are part of two pilot clinical
studies that demonstrated sustained remissions of up to two years in pediatric
and adult patients with r/r ALL. Median follow-up was just over six months, with
event-free survival of 67% and overall survival of 78%. Probability of six-month
CTL019 persistence was 68% and CTL019-modified T cells were detectable in the
blood by flow cytometry for up to 11 months. Sustained remissions were seen in
15 patients and were associated with CAR T cell persistence and B cell
aplasia[1]. Updated results have been submitted for presentation at a medical
congress taking place later in 2014.
"We are excited by these results, which indicated how effective CTL019 may be in
fighting ALL, a leading cause of childhood cancer deaths," said lead
investigator Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of
Pediatrics in the Perelman School of Medicine at the University of Pennsylvania
and director of Translational Research in the Center for Childhood Cancer
Research at the Children's Hospital of Philadelphia (CHOP), where 25 pediatric
patients were treated in the study cohort[1]. "This represents the largest
experience to date of CD19-CAR T cells and demonstrates the ability of this
approach to achieve sustained complete responses in a patient population with
few other treatment options. We are especially hopeful for those patients who
remain in remission for 1-2 years without further therapy."
In July 2014, the FDA designated CTL019 as a Breakthrough Therapy under the Penn
IND, which is intended to expedite the development and review of drugs that
treat serious or life-threatening conditions if the therapy has demonstrated
substantial improvement over an available therapy on at least one clinically
significant endpoint[3].
Novartis holds the worldwide rights to CARs developed through the collaboration
with Penn for all cancer indications, including the lead program, CTL019.
About These Studies
Twenty-five patients enrolled in the pediatric pilot trial at CHOP and 5
patients enrolled in the adult pilot trial at Penn from April 2012 to February
2014. The patients were infused with autologous T cells transduced with a CD19-
directed CAR (CTL019) lentiviral vector at doses of 0.7-20.6x10(6) CTL019
cells/kg. The study found that 27 of 30 pediatric and adult patients with r/r
ALL (90%) experienced complete remissions, including two blinatumomab-refractory
patients and 15 with prior stem cell transplant[1].
Of the 27 patients who achieved a complete remission, five went off-study for
alternate therapy, three of whom proceeded to allogeneic SCT in remission.
Fifteen patients remain in remission with a median follow-up of seven months.
Sustained remissions were achieved up to two years with six-month event-free
survival 67% (95% CI, 51% to 88%) and overall survival 78% (95% CI, 65% to
95%). The probability of six-month CTL019 persistence was 68% (95% CI, 50 to
92%) and relapse-free B cell aplasia was 73% (95% CI, 57 to 97%). CTL019-
modified T cells were detectable in the blood by flow cytometry for up to 11
months, and CTL019 sequences remained detectable by quantitative PCR (Q-PCR) in
patients with sustained remissions for up to two years[1].
All patients experienced cytokine release syndrome (CRS). Of the 30 patients,
74% (n=22) experienced mild to moderate CRS. Severe CRS, seen in 27% of patients
(n=8), was associated with higher disease burden and effectively treated with
the IL-6 receptor antibody tocilizumab. Several patients experienced neurologic
toxicities, which fully resolved without further intervention or apparent long-
term implications[1].
About CTL019
CTL019 uses CAR technology to reprogram a patient's own T cells to "hunt" cancer
cells that express specific proteins, called CD19. After they have been
reprogrammed, the T cells (now called CTL019) are re-introduced into the
patient's blood; they proliferate and bind to the targeted CD19+ cancer cells
and destroy them.
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.
About Acute Lymphoblastic Leukemia
Acute lymphoblastic leukemia (ALL) is the most common cancer diagnosed in
children, representing approximately 25% of cancer diagnoses among children
younger than 15 years, according to data published in 2013[4]. It can also occur
in adults. ALL is a type of cancer in which the bone marrow makes too many
abnormal white blood cells (lymphocytes). ALL usually gets worse quickly if it
is not treated and can be fatal within a few months; therefore, it is critical
for patients to start treatment soon after diagnosis. Patients with relapsed ALL
experience ALL cells returning in the marrow and a decrease in normal blood
cells following their remission. Patients with refractory ALL still have
leukemia cells in their bone marrow following treatment[5].
Disclaimer
The foregoing release contains forward-looking statements that can be identified
by words such as "Breakthrough Therapy," "investigational," "potential,"
"promising," "potentially," "submitted for presentation," "may," "hopeful,"
"will," 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 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, cost-saving generic pharmaceuticals, preventive vaccines, over-the-
counter and animal health products. Novartis is the only global company with
leading positions in these areas. In 2013, the Group achieved net sales of USD
57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9
billion (USD 9.6 billion excluding impairment and amortization charges).
Novartis Group companies employ approximately 135,000 full-time-equivalent
associates and sell products in more than 150 countries around the world. For
more information, please visit http://www.novartis.com.
Novartis is on Twitter. Sign up to follow (at)Novartis at
http://twitter.com/novartis.
References
[1] Maude S et al. Chimeric Antigen Receptor T Cells for Sustained Remissions in
Leukemia. N Engl J Med. 2014; 371:1507-17.
[2] Novartis Press Release. "Novartis personalized cell therapy CTL019 receives
FDA Breakthrough Therapy designation." Available at:
http://www.novartis.com/newsroom/media-releases/en/2014/1816270.shtml. Accessed
September 2014.
[3] US Food and Drug Administration. Guidance for Industry Expedited Programs
for Serious Conditions - Drugs and Biologics Frequently Asked Questions:
Breakthrough Therapies. Available at:
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guida
nces/UCM358301.pdf. Accessed September 2014.
[4] Howlader, N., Noone, A. M., Krapcho, M, et al. SEER Cancer Statistics
Review, 1975-2010. National Cancer Institute, April 2013; Section 28.9 (12).
http://www.seer.cancer.gov/csr/1975_2010/results_merged/sect_28_childhood_cancer
.pdf. Accessed June 2014.
[5] Apostolidou, Effrosyni, et al. Treatment of Acute Lymphoblastic Leukaemia.
Drugs 2007; 67 (15): 2153-2171.
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