Durable Three-Year Outcomes In Degenerative Disc Disease After a Single Injection of Mesoblast’s Cell Therapy
15 March 2017 - 9:00PM
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today announced 36-month
results from the randomized, placebo-controlled 100-patient Phase 2
trial of its proprietary allogeneic Mesenchymal Precursor
Cells (MPCs) in patients with chronic low back pain
(CLBP) due to intervertebral disc degeneration. A
single intra-discal injection of 6 million MPCs resulted in
meaningful improvements in both pain and function that were durable
for at least 36 months.
“The sustained benefits on pain and function over
three years seen with a single injection of Mesoblast’s cell
therapy have the potential to transform the treatment paradigm
for chronic low back pain due to disc degeneration,” said trial
investigator Dr Hyun Bae, Professor of Surgery and Director of
Education at the Cedars Sinai Spine Center, and Director of the
Spine Institute in Los Angeles, CA. “Instead
of replacing or fusing the disc, there is mounting compelling
evidence that we can use this regenerative medicine to heal the
disc. We are fast approaching this inflection point in the
treatment of low back pain, which is particularly important in
view of the epidemic of opioid abuse."
The durable outcomes seen from a single MPC
injection in patients with degenerative disc disease who have
failed conservative measures are consistent with an overarching
mechanism of action that may also be evident in treatment of other
chronic diseases where a single MPC dose has resulted in
sustained benefits, including advanced chronic heart failure
and biological-resistant rheumatoid arthritis. In each of
these diseases, MPCs are thought to be activated by signals in the
damaged tissues to release factors that both inhibit damaging
inflammation and induce a pro-reparative state.
The Phase 2 trial compared a single intra-discal
injection of 6 million or 18 million MPCs against two placebo arms,
saline or hyaluronic acid, using a pre-specified Per Protocol (PP)
population analysis. The primary endpoint composite was the
same as is being used in the ongoing Phase 3 trial, a 50% reduction
in the Visual Analog Scale (VAS) pain score and a 15-point
reduction in the Oswestry disability index (ODI), with no
additional intervention, at both 12 and 24 months.
In line with United States Food and Drug
Administration (FDA) guidance for the ongoing Phase 3 trial, the
24-month primary endpoint composite was additionally analyzed
using an intent to treat (ITT) population. The 36-month
analysis aimed to determine the proportion of patients who
maintained treatment success beyond the 24-month primary
evaluation.
Key trial results were:
- the primary endpoint composite over 24 months was achieved by
41% of patients who received 6 million MPCs, 35% of the 18
million MPC group, 18% of the hyaluronic acid group, and 13% of the
saline group, using the pre-specified PP population analysis-
pain responder criteria (50% pain reduction with no
additional intervention at both 12 and 24 months) was achieved by
52% of the 6 million MPC group compared with 13% of the saline
group (p<0.05)- functional responder criteria (15-point
reduction in ODI and no additional intervention at both 12 and 24
months) was achieved by 48% of the 6 million MPC group compared
with 13% of the saline group (p<0.05)
- similar results were seen for the primary endpoint composite
over 24 months using the ITT analysis, with 38% of the 6 million
MPC group achieving this outcome compared with 10% of the saline
group (p<0.05)- 82% of the 6 million MPC group who
achieved the primary endpoint composite over 24 months maintained
treatment success using this composite endpoint at 36 months-
86% of the 6 million MPC group who successfully met
the pain responder criteria (50% pain reduction with no additional
intervention at both 12 and 24 months) remained pain responders
through 36 months- 92% of the 6 million MPC group
who met the functional responder criteria (15-point reduction
in ODI and no additional intervention at both 12 and 24 months)
remained functional responders through 36 months
- there were no significant differences in measurements of
safety between cell-treated patients and controls over 36
months
The 36-month Phase 2 trial results support
the ongoing 360-patient Phase 3 trial of Mesoblast's product
candidate MPC-06-ID for CLBP by reinforcing the rationale for
MPC dose selection, use of saline control, and the trial's primary
endpoint composite over 24 months. If similar
clinical durability is seen in the Phase 3 program, it is
anticipated such data will translate into meaningful health
economic benefits including increased productivity that may support
attractive product reimbursement.
In December 2016, Mesoblast and Mallinckrodt
Pharmaceuticals entered into an agreement to exclusively negotiate
a commercial and development partnership for MPC-06-ID in the
treatment of chronic low back pain due to disc degeneration.
About Chronic Low Back Pain (CLBP) Caused
By Degenerative Disc Disease
Over 33 million1 patients in the U.S. alone suffer
from CLBP with approximately 22%2 caused by degenerative disc
disease. Total costs of low back pain in the U.S. are
estimated to be between US$100 billion and US$200 billion annually
with two thirds of these costs attributed to patients’ decreased
wages and productivity.
All therapies for progressive, severe and
debilitating pain due to degenerating intervertebral discs treat
the symptoms of the disease, but are not disease-modifying and thus
do not address the underlying cause of the disease.
Limited treatment options exist for patients who
have failed conservative treatment (e.g. physical therapy,
anti-inflammatory agents or analgesics) or other measures including
opioids and epidural steroid injections. When disc
degeneration has progressed to a point that pain and loss of
function can no longer be managed by conservative means, major
invasive surgery is the only remaining option. Even with
surgical intervention such as spinal fusion or artificial disc
replacement, over 50% of patients are considered treatment
failures.
Mesoblast’s MPC-06-ID Phase 3 program targets a
patient population with significant unmet need who have exhausted
conservative treatment options. MPC-06-ID is being developed
to alleviate pain and improve function, either before the use of
opioids and/or epidural steroid injections or after failure of
these approaches, in order to prevent invasive and costly surgical
interventions.
About MesoblastMesoblast Limited
(Nasdaq:MESO) (ASX:MSB) is a global leader in developing innovative
cell-based medicines. The Company has leveraged its
proprietary technology platform, which is based on specialized
cells known as mesenchymal lineage adult stem cells, to establish a
broad portfolio of late-stage product candidates. Mesoblast’s
allogeneic, ‘off-the-shelf’ cell product candidates target advanced
stages of diseases with high, unmet medical needs including
cardiovascular conditions, orthopedic disorders, immunologic and
inflammatory disorders and oncologic/hematologic conditions.
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1 Decision Resource 2015 Chronic Pain Report2
https://academic.oup.com/painmedicine/article-lookup/doi/10.1111/pme.12809
For further information, please contact:
Julie Meldrum
Corporate Communications
T: +61 3 9639 6036
E: julie.meldrum@mesoblast.com
Schond Greenway
Investor Relations
T: +1 212 880 2060
E: schond.greenway@mesoblast.com
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