FDA Grants Regenerative Medicine Advanced Therapy (Rmat) Designation for Rexlemestrocel-L in Chronic Low Back Pain
February 08 2023 - 6:09PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced that the United States Food and Drug Administration’s
(FDA) Office of Tissues and Advanced Therapies (OTAT) has granted
Regenerative Medicine Advanced Therapy (RMAT) designation for
rexlemestrocel-L in the treatment of chronic low back pain (CLBP)
associated with disc degeneration, in combination with hyaluronic
acid (HA) as delivery agent for injection into the lumbar disc.
RMAT designations aim to expedite the
development of regenerative medicine therapies intended to treat,
modify, reverse, or cure a serious or life-threatening disease or
condition where preliminary clinical evidence indicates that the
drug has the potential to address unmet medical needs for the
disease or condition. An RMAT designation for rexlemestrocel-L
provides all the benefits of Breakthrough and Fast Track
designations, including rolling review and eligibility for priority
review on filing of a Biologics License Application (BLA).
There is a significant need for a safe,
effective, and durable opioid-sparing treatment in patients with
CLBP associated with degenerative disc disease. Data from the
completed 404-patient randomized, blinded placebo-controlled Phase
3 trial of rexlemestrocel-L combined with HA formed the basis for
FDA’s determination that Mesoblast’s allogeneic cell therapy has
the potential to address unmet medical needs for patients suffering
from CLBP due to disc degeneration. Results from the trial showed
that:
- A single injection
of rexlemestrocel-L+HA into the lumbar disc resulted in significant
reduction in pain compared with saline control at 12 and 24 months
across all subjects (n=404).
- The reduction in
low back pain observed in all subjects was substantially enhanced
in the pre-specified population with CLBP of shorter duration than
the median of 68 months for the study (n=194).
- Significant
improvement in function and quality of life measures were observed
in subjects with a shorter duration of CLBP at baseline.
- Pain reduction
through 36 months was seen in the subset of patients using opioids
at baseline (n=168) with the rexlemestrocel-L+HA group having
substantially greater reduction at all time points compared with
saline controls.
- Among patients on
opioids at baseline, despite instructions to maintain existing
therapies throughout the trial, at 36 months 28% who received
rexlemestrocel-L + HA were not taking an opioid compared with 8% of
saline treated controls.
“We are pleased to receive RMAT designation for
our cellular therapy to treat CLBP due to disc degeneration” said
Mesoblast Chief Executive Silviu Itescu. “We look forward to
working closely with FDA to efficiently generate the additional
data needed to support marketing approval of rexlemestrocel-L for
the treatment of this serious and debilitating condition.”
About Chronic Low Back PainCLBP
is a serious condition with an annual prevalence of low back pain
in the general US adult population of 10-30% and a lifetime
prevalence in US adults as high as 65-80%.1 Overall, low back pain
is the fifth most common reason for visiting a US doctor.2 In the
last 3 months alone, approximately 25% of adults in the USA
experienced low back pain for at least 24 hours.3 In the first 3
months, recovery is observed in 33% of patients, but 1 year after
onset, 65% still report pain.4 CLBP is one of the leading causes of
disability in the United States, and it is associated with impaired
quality of life, severe limitations in ability to perform
activities of daily living, reduced ability to work, and negative
impacts on mental health. In fact, back pain is the leading cause
of disability in Americans under 45 years old with more than 26
million Americans between the ages of 20-64 experiencing frequent
back pain.5 Back pain is listed as a cause of limitations in
activities of daily living by 15% to 31% of those persons with
limitations.6 When considering both death and disability,
musculoskeletal conditions such as CLBP are the second greatest
cause of disability and have the fourth greatest impact on the
overall health of the world population.7
About Mesoblast Mesoblast is a
world leader in developing allogeneic (off-the-shelf) cellular
medicines for the treatment of severe and life-threatening
inflammatory conditions. The Company has leveraged its proprietary
mesenchymal lineage cell therapy technology platform to establish a
broad portfolio of late-stage product candidates which respond to
severe inflammation by releasing anti-inflammatory factors that
counter and modulate multiple effector arms of the immune system,
resulting in significant reduction of the damaging inflammatory
process.
Mesoblast has a strong and extensive global
intellectual property portfolio with protection extending through
to at least 2041 in all major markets. The Company’s proprietary
manufacturing processes yield industrial-scale, cryopreserved,
off-the-shelf, cellular medicines. These cell therapies, with
defined pharmaceutical release criteria, are planned to be readily
available to patients worldwide.
Mesoblast is developing product candidates for
distinct indications based on its remestemcel-L and
rexlemestrocel-L allogeneic stromal cell technology platforms.
Remestemcel-L is being developed for inflammatory diseases in
children and adults including steroid refractory acute graft versus
host disease, biologic-resistant inflammatory bowel disease, and
acute respiratory distress syndrome. Rexlemestrocel-L is in
development for advanced chronic heart failure and chronic low back
pain. Two products have been commercialized in Japan and Europe by
Mesoblast’s licensees, and the Company has established commercial
partnerships in Europe and China for certain Phase 3 assets.
Mesoblast has locations in Australia, the United
States and Singapore and is listed on the Australian Securities
Exchange (MSB) and on the Nasdaq (MESO). For more information,
please see www.mesoblast.com, LinkedIn: Mesoblast Limited and
Twitter: @Mesoblast
References / Footnotes
- Urits I, Burshtein A, Sharma M, et
al. Low Back Pain, a Comprehensive Review: Pathophysiology,
Diagnosis, and Treatment. Current Pain and Headache Reports.
2019;23(3):1-10. doi:10.1007/s11916-019-0757-1.
- Atlas SJ, Deyo RA. Evaluating and
managing acute low back pain in the primary care setting. J Gen
Intern Med, Springer. 2001;16: 120–31.
- Petering RC, Webb C. Treatment
options for low back pain in athletes. Sports Health, SAGE
Publications. 2011;3:550–5.
- Itz CJ, Geurts JW, Kleef M,
Nelemans P. Clinical course of non‐specific low back pain: A
systematic review of prospective cohort studies set in primary
care. European Journal of Pain. 2012;17(1):5-15.
doi:10.1002/j.1532-2149.2012.00170.x.
- American Academy of Pain Medicine -
Get the Facts on Pain. The American Academy of Pain Medicine.
http://www.painmed.org/patientcenter/facts-on-pain/ Accessed on
June 28, 2017.
- United States Bone and Joint
Initiative: The Burden of Musculoskeletal Diseases in the United
States (BMUS), Third Edition, 2014. Rosemont, IL. Available at
http://www.boneandjointburden.org. Accessed on August 23,
2017.
- United States Bone and Joint
Initiative: The Burden of Musculoskeletal Diseases in the United
States (BMUS), Third Edition, 2014. Rosemont, IL. Available at
http://www.boneandjointburden.org. Accessed on August 23,
2017.
Forward-Looking StatementsThis
press release includes forward-looking statements that relate to
future events or our future financial performance and involve known
and unknown risks, uncertainties and other factors that may cause
our actual results, levels of activity, performance or achievements
to differ materially from any future results, levels of activity,
performance or achievements expressed or implied by these
forward-looking statements. We make such forward-looking statements
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
Forward-looking statements should not be read as a guarantee of
future performance or results, and actual results may differ from
the results anticipated in these forward-looking statements, and
the differences may be material and adverse. Forward-looking
statements include, but are not limited to, statements about: the
initiation, timing, progress and results of Mesoblast’s preclinical
and clinical studies, and Mesoblast’s research and development
programs; Mesoblast’s ability to advance product candidates into,
enroll and successfully complete, clinical studies, including
multi-national clinical trials; Mesoblast’s ability to advance its
manufacturing capabilities; the timing or likelihood of regulatory
filings and approvals, manufacturing activities and product
marketing activities, if any; the commercialization of Mesoblast’s
product candidates, if approved; regulatory or public perceptions
and market acceptance surrounding the use of stem-cell based
therapies; the potential for Mesoblast’s product candidates, if any
are approved, to be withdrawn from the market due to patient
adverse events or deaths; the potential benefits of strategic
collaboration agreements and Mesoblast’s ability to enter into and
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needs for additional financing; Mesoblast’s financial performance;
developments relating to Mesoblast’s competitors and industry; and
the pricing and reimbursement of Mesoblast’s product candidates, if
approved. You should read this press release together with our risk
factors, in our most recently filed reports with the SEC or on our
website. Uncertainties and risks that may cause Mesoblast’s actual
results, performance or achievements to be materially different
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and accordingly, you should not place undue reliance on these
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as a result of new information, future developments or
otherwise.
Release authorized by the Chief Executive.
For more information, please contact:
Corporate Communications / Investors |
Media |
Paul Hughes |
BlueDot Media |
T: +61 3 9639 6036 |
Steve Dabkowski |
E: investors@mesoblast.com |
T: +61 419 880 486 |
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E: steve@bluedot.net.au |
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Rubenstein |
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Tali Mackay |
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E: tmackay@rubenstein.com |
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