New translational data suggests potential
disease-modifying effects following treatment with pelabresib of
both first-line and ruxolitinib-relapsed/refractory patients
A comparative model suggests an improvement in
SVR35 and TSS50 in JAK inhibitor-naïve myelofibrosis patients
treated with pelabresib plus ruxolitinib over JAK inhibitors as
monotherapy
Additional presentations include positive
interim data from the MANIFEST Phase 2 trial and design of
MANIFEST-2, a global Phase 3, randomized, double-blind trial of
pelabresib in combination with ruxolitinib in treatment-naïve
patients
MorphoSys AG (FSE: MOR; NASDAQ: MOR) is presenting data from
multiple analyses of the ongoing MANIFEST study, an open-label
Phase 2 clinical trial of pelabresib, an investigational BET
inhibitor, in patients with myelofibrosis, a rare bone marrow
cancer for which only limited treatment options are available. The
latest findings suggest pelabresib may have disease-modifying
properties and confirm previous data supporting the potential of
pelabresib as a treatment for patients with myelofibrosis. The data
are being presented during oral and poster sessions at the European
Hematology Association 2022 (EHA 2022) Hybrid Congress being held
in Vienna.
“The standard for evaluating disease response in myelofibrosis
focuses on symptom relief rather than true disease modification,
which remains an unmet need for these patients,” said John
Mascarenhas, M.D., Director of the Adult Leukemia Program at The
Tisch Cancer Institute at Mount Sinai, New York. “The body of data
being presented at EHA 2022 – including new findings that
pelabresib may address cellular defects seen in myelofibrosis,
thereby getting at the root cause of the disease – with correlated
clinical improvements, suggests pelabresib may have the potential
to enhance the current standard of care in the first-line treatment
of myelofibrosis.”
A study that will be presented in an oral session on June 11
analyzed cells derived from blood of patients who enrolled in the
MANIFEST trial and from healthy volunteers. The findings indicate
that pelabresib alone or in combination with the JAK inhibitor
ruxolitinib may have the potential to improve the typical imbalance
in the two white blood cell populations, the myeloid and lymphoid
cells, and help restore normal blood cell development. These
improvements also correlated with decreases in spleen volume, a key
clinical measure of treatment success. Additionally, pelabresib
alone or in combination decreased pro-inflammatory and pro-fibrotic
signaling in monocytes, suggesting a potential attenuation of
disease processes.
“The latest findings from the MANIFEST trial at EHA 2022
highlight the potential of pelabresib to offer patients and their
physicians benefits over monotherapy with JAK inhibitors, if
approved,” said Malte Peters, M.D., MorphoSys Chief Research and
Development Officer. “The full complement of MANIFEST data being
presented this week suggests pelabresib may help improve outcomes
for patients with myelofibrosis and reaffirms our confidence in the
Phase 3 MANIFEST-2 study. We are committed to these patients, who
need better options in first-line treatment and beyond.”
A second oral presentation on June 11 highlights positive
interim data from the MANIFEST trial on the safety and efficacy of
pelabresib in combination with ruxolitinib in patients who were not
previously treated with a JAK inhibitor and in those with
suboptimal response to ruxolitinib. The findings show that the
combination was generally well tolerated and offered reductions in
spleen volume and symptom burden, with disease-modifying activity
as measured by reduced levels of pro-inflammatory cytokines and
improved bone marrow morphology. Over two-thirds (68%; n=57) of JAK
inhibitor-naïve patients treated with the combination achieved at
least a 35% reduction in spleen volume (SVR35) from baseline at
week 24. Notably, 80% of patients achieved SVR35 at any time on
study. Most patients also saw their symptoms reduced, with 56%
(n=46) achieving at least a 50% reduction in total symptom score
(TSS50) from baseline at week 24. No new safety signals were
identified in the study. The most common hematologic adverse events
were thrombocytopenia (12%, grade 3/4) and anemia (34%, grade 3/4).
Non-hematological events included dyspnea (5%, grade 3) and
respiratory tract infections (8%, grade 3/4).
In a poster presentation at EHA 2022, matching-adjusted indirect
comparisons were used to compare findings for the combination of
pelabresib plus ruxolitinib in treatment-naïve patients with
intermediate- or high-risk disease in one arm of the MANIFEST trial
with findings from historical clinical trials examining the use of
JAK inhibitor monotherapy in myelofibrosis. Adjusting for
cross-trial differences, the estimated response rate ratios favored
the pelabresib combination over ruxolitinib, fedratinib or
momelotinib monotherapy for SVR35 and for TSS50, suggesting
improved efficacy versus the JAK inhibitors alone.
A second poster presentation includes trial design information
for the Phase 3 MANIFEST-2 study. MANIFEST-2, which is currently
enrolling, will compare pelabresib in combination with ruxolitinib
versus placebo plus ruxolitinib in approximately 400 patients with
myelofibrosis who are naïve to JAK inhibitor therapy. MorphoSys is
expected to report topline data from the MANIFEST-2 trial in the
first half of 2024.
About Pelabresib
Pelabresib (CPI-0610) is an investigational selective small
molecule designed to promote anti-tumor activity by inhibiting the
function of bromodomain and extra-terminal domain (BET) proteins to
decrease the expression of abnormally expressed genes in cancer.
Pelabresib is being investigated as a treatment for myelofibrosis
and has not yet been evaluated or approved by any regulatory
authorities.
About Myelofibrosis
Myelofibrosis is a type of chronic leukemia that causes
extensive scarring in the bone marrow, which disrupts the body’s
normal production of healthy blood cells. The result is a reduction
in red blood cells, which can cause weakness and fatigue, and in
platelets, which increases the risk of bleeding due to deficient
clotting. Myelofibrosis often causes an enlarged spleen. It is most
often diagnosed in people older than 50 and can occur on its own
(called primary myelofibrosis) or because of another bone marrow
disorder.
About MANIFEST
MANIFEST (NCT02158858) is an open-label Phase 2 clinical trial
of pelabresib in patients with myelofibrosis.
The MANIFEST trial is evaluating pelabresib in combination with
ruxolitinib in JAK-inhibitor-naïve myelofibrosis patients (Arm 3),
with a primary endpoint of the proportion of patients with a ≥35%
spleen volume reduction from baseline (SVR35) after 24 weeks of
treatment. The trial is also evaluating pelabresib either as a
monotherapy in patients who are resistant to, intolerant of, or
ineligible for ruxolitinib and no longer on the drug (Arm 1) or as
add-on therapy in combination with ruxolitinib in patients with a
suboptimal response to ruxolitinib or myelofibrosis progression
(Arm 2). Patients in Arms 1 and 2 are being stratified based on
transfusion-dependent (TD) status. The primary endpoint for the
patients in cohorts 1A and 2A, who were TD at baseline, is
conversion to transfusion independence for 12 consecutive weeks.
The primary endpoint for patients in cohorts 1B and 2B, who were
not TD at baseline, is the proportion of patients with a ≥35%
spleen volume reduction from baseline after 24 weeks of
treatment.
Constellation Pharmaceuticals, Inc., a MorphoSys company, is the
MANIFEST trial sponsor.
About MANIFEST-2
MANIFEST-2 (NCT04603495) is a global, double-blind, randomized
Phase 3 clinical trial with pelabresib in combination with
ruxolitinib versus placebo plus ruxolitinib in JAK inhibitor-naïve
patients with myelofibrosis. The primary endpoint of the study is a
35% or greater reduction in spleen volume (SVR35) from baseline at
24 weeks. A key secondary endpoint of the study is a 50% or greater
improvement in total symptom score (TSS50) from baseline at 24
weeks.
Constellation Pharmaceuticals, Inc., a MorphoSys company, is the
MANIFEST-2 trial sponsor.
About MorphoSys:
At MorphoSys, we are driven by our mission: More life for people
with cancer. As a global commercial-stage biopharmaceutical
company, we use groundbreaking science and technologies to
discover, develop, and deliver innovative cancer medicines to
patients. MorphoSys is headquartered in Planegg, Germany, and has
its U.S. operations anchored in Boston, Massachusetts. To learn
more, visit us at www.morphosys.com and follow us on Twitter and
LinkedIn.
Forward Looking Statements
This communication contains certain forward-looking statements
concerning the MorphoSys group of companies. The forward-looking
statements contained herein represent the judgment of MorphoSys as
of the date of this release and involve known and unknown risks and
uncertainties, which might cause the actual results, financial
condition and liquidity, performance or achievements of MorphoSys,
or industry results, to be materially different from any historic
or future results, financial conditions and liquidity, performance
or achievements expressed or implied by such forward-looking
statements. In addition, even if MorphoSys' results, performance,
financial condition and liquidity, and the development of the
industry in which it operates are consistent with such
forward-looking statements, they may not be predictive of results
or developments in future periods. Among the factors that may
result in differences are that MorphoSys' expectations may be
incorrect, the inherent uncertainties associated with competitive
developments, clinical trial and product development activities and
regulatory approval requirements, MorphoSys' reliance on
collaborations with third parties, estimating the commercial
potential of its development programs and other risks indicated in
the risk factors included in MorphoSys' Annual Report on Form 20-F
and other filings with the U.S. Securities and Exchange Commission.
Given these uncertainties, the reader is advised not to place any
undue reliance on such forward-looking statements. These
forward-looking statements speak only as of the date of publication
of this document. MorphoSys expressly disclaims any obligation to
update any such forward-looking statements in this document to
reflect any change in its expectations with regard thereto or any
change in events, conditions or circumstances on which any such
statement is based or that may affect the likelihood that actual
results will differ from those set forth in the forward-looking
statements, unless specifically required by law or regulation.
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version on businesswire.com: https://www.businesswire.com/news/home/20220610005021/en/
Media Contacts: Thomas Biegi Vice President Tel.: +49
(0)89 / 89927 26079 thomas.biegi@morphosys.com Eamonn Nolan
Director Tel: +1 617-548-9271 eamonn.nolan@morphosys.com
Investor Contacts: Dr. Julia Neugebauer Senior Director Tel:
+49 (0)89 / 899 27 179 julia.neugebauer@morphosys.com Myles
Clouston Senior Director Tel: +1 857-772-0240
myles.clouston@morphosys.com
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