FDA’s OTAT in Agreement With 12-Month Reduction in Pain as Primary Endpoint for Chronic Low Back Pain Program
December 15 2021 - 7:09PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
announced that it has received feedback from the US Food & Drug
Administration’s (FDA) Office of Tissues and Advanced Therapies
(OTAT) on the Phase 3 program of rexlemestrocel-L in patients with
chronic low back pain (CLBP) due to degenerative disc disease (DDD)
refractory to available therapies, including opioids.
Mesoblast plans to conduct an additional US Phase 3 trial which
may support submissions for potential approval in both the US and
EU. The trial will include at least 20% of subjects from the EU to
support global submission plans. Following review of the completed
Phase 3 trial data, OTAT agreed with Mesoblast’s proposal for pain
reduction at 12 months as the primary endpoint of the next trial,
with functional improvement and reduction in opioid use as
secondary endpoints.
If this trial is successful and leads to EU regulatory approval,
Mesoblast will be eligible to receive payments up to US$112.5
million prior to product launch in the EU, from its partner in
Europe and Latin America, Grünenthal, inclusive of US$17.5 million
already received, if certain clinical and regulatory milestones are
satisfied and reimbursement targets are achieved. Cumulative
milestone payments could reach US$1 billion depending on the final
outcome of Phase 3 studies and patient adoption. Mesoblast will
also receive tiered double-digit royalties on product sales.
In the US, excessive use of opioids in this patient population,
with more than 50% of US opioid prescriptions being for the
treatment of CLBP,1-3 continues to be a major unmet medical need
and focus for healthcare policymakers, regulatory authorities, and
pharmaceutical companies. A key objective is to demonstrate
reduction in pain and opioid usage and position rexlemestrocel-L as
a potential opioid-sparing agent.
About Chronic Low Back Pain due to Degenerative Disc
Disease Chronic low back pain (CLBP) affects approximately
10-15% of the adult population, equivalent to more than 30 million
people in the United States and almost 40 million people across the
EU5.1 Degenerative disc disease (DDD) causing discogenic pain is
the most common etiology of CLBP in adults.6,7 Over 7 million
patients in each of the United States and E.U.5 are thought to
suffer from CLBP caused by degenerative disc disease,2,6,7 a
disease which involves inflammation and degeneration of the
intervertebral discs due to various factors including age, trauma
or genetic pre-disposition.
Back pain causes more disability than any other condition and
inflicts substantial direct and indirect costs on the healthcare
system2, including excessive use of opioids in this patient
population. There are few treatment options for patients with CLBP
who fail conservative therapy, including opioids, spinal injections
and surgery (e.g., spinal fusion or total disk arthroplasty).3 More
than 50% of US opioid prescriptions are for the treatment of
CLBP,1,4-5 despite the fact that opioids are associated with
serious and potentially life-threatening side effects and have not
demonstrated efficacy in the treatment of CLBP.5,8,9 In 2018, more
than 67,000 drug overdose deaths occurred in the United States10 of
which almost 47,000 (70%) were opioid related.
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 stromal
cell technology platforms. Remestemcel-L is being developed for
inflammatory diseases in children and adults including steroid
refractory acute graft versus host disease and moderate to severe
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
- Decision Resources: Chronic Pain Report 2015.
- Williams, J., NG, Nawi, Pelzter, K. (2015) Risk factors and
disability associated with low back pain in older adults in low-and
middle-income countries. Results from the WHO Study on global
ageing and adult health (SAGE). PloS One. 2015; 10(6):
e0127880
- Zigler J, et al. Comparison of lumbar total disc replacement
with surgical spinal fusion for the treatment of single-level
degenerative disc disease: a meta-analysis of 5-year outcomes from
randomized controlled trials. Glob Spine J. 2018;8(4):413–23
- Abdel Shaheed C, Maher CG, Williams KA, Day R, McLachlan AJ.
Efficacy, tolerability, and dose-dependent effects of opioid
analgesics for low back pain: a systematic review and
meta-analysis. JAMA Intern Med 2016;176(7):958–68
- Hudson TJ, Edlund MJ, Steffick DE, Tripathi SP, Sullivan MD.
Epidemiology of regular prescribed opioid use: results from a
national, population-based survey. J Pain Symptom Manage
2008;36(3):280–8
- DePalma MJ, et al. What Is the Source of Chronic Low Back Pain
and Does Age Play a Role? Pain Med. 2011; 12: 224–233
- Peng BG. Pathophysiology, diagnosis, and treatment of
discogenic low back pain. World J Orthop. 2013 April 18; 4(2):
42-52
- Chaparro LE, Furlan AD, Deshpande A, Mailis-Gagnon A, Atlas S,
Turk DC. Opioids compared to placebo or other treatments for
chronic low-back pain. Cochrane Database Syst Rev
2013(8):CD004959
- Chou R, Turner JA, Devine EB, Hansen RN, Sullivan SD, Blazina
I, et al. The effectiveness and risks of long-term opioid therapy
for chronic pain: a systematic review for a National Institutes of
Health Pathways to Prevention Workshop. Ann Intern Med 2015;162
(4):276–86
- Annual surveillance report of drug-related risks and outcomes
United States, 2019. Centers for Disease Control and
Prevention
Forward-Looking StatementsThis announcement
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
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multi-national clinical trials; Mesoblast’s ability to advance its
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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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to establish and maintain intellectual property on its product
candidates and Mesoblast’s ability to successfully defend these in
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able to establish and maintain for intellectual property rights
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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
forward-looking statements. We do not undertake any obligations to
publicly update or revise any forward-looking statements, whether
as a result of new information, future developments or
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Release authorized by the Chief Executive.
For more information, please contact:
Corporate Communications / Investors |
Media |
Paul Hughes |
Sumit Media |
T: +61 3 9639 6036 |
Grant Titmus |
E: investors@mesoblast.com |
T: +61 419 388 161 |
|
E: grant@sumitmedia.com.au |
|
|
|
Rubenstein |
|
Nadine Woloshin |
|
T: +1 917-699-9456 |
|
E: nwoloshin@rubenstein.com |
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