Appendix 4C Quarterly Activity Report
April 28 2022 - 8:35PM
Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in
allogeneic cellular medicines for inflammatory diseases, today
provided an activity report for the third quarter ended March 31,
2022.
Financial highlights
- Net cash usage for operating
activities in the quarter was reduced by 40%, or US$10.3 million,
to US$15.5 million compared with US$25.8 million in the comparative
quarter last year
- Net cash usage for operating
activities, excluding inventory for the planned remestemcel-L
product launch, was reduced by 50% to US$11.2 million from US$22.2
million in the comparative quarter
- Cash on hand at the end of the
quarter was US$76.8 million, with up to an additional US$40 million
available to be drawn down from existing financing facilities
subject to certain milestones
- Revenues in the quarter were US$2.1
million, including US$1.9 million from TEMCELL® HS Inj.1 royalties
on sales for SR-aGvHD in Japan, an increase of 5% on the
comparative quarter last year
New appointments to the Board and
Management:
- Philip R. Krause, M.D. joined the
Board of Directors in March. Dr. Krause was for the past decade
Deputy Director, Office of Vaccines Research and Review (OVRR) at
the United States Food and Drug Administration’s (FDA) Center for
Biologics Evaluation and Research (CBER). Dr. Krause is currently
Chair of the World Health Organization COVID Vaccines Research
Expert Group, and most recently he shared responsibility for
regulatory authorizations of COVID-19 vaccines in the US. Dr.
Krause’s deep insights and knowledge of regulatory processes will
be invaluable to Mesoblast as it prepares its resubmission of the
Biologics License Application (BLA) to the FDA for remestemcel-L in
the treatment of children with steroid-refractory acute graft
versus host disease (SR-aGVHD)
- Eric Rose, M.D. was appointed as the
Company’s Chief Medical Officer (CMO), having been a non-executive
director of Mesoblast since 2013. Previously Chairman of Surgery at
Columbia University’s School of Medicine, Dr. Rose brings to his
new role an extensive record of excellence in clinical development
and successful interactions at the highest levels with key
regulatory, industry and government stakeholders including the
United States FDA, the National Institutes of Health (NIH), and the
Biomedical Advanced Research and Development Authority (BARDA)
Key updates on remestemcel-L:
BLA resubmission to the FDA for the treatment of children with
SR-aGVHD
- Detailed data requirements and
documentation being finalized for resubmission of the Biologics
License Application (BLA) to the FDA for remestemcel-L in the
treatment of children with SR-aGVHD
- Mesoblast has generated substantial
new data, as previously discussed, that it believes establish the
relevance of the proposed potency assay measuring remestemcel-L’s
in vitro anti-inflammatory and immunomodulatory activity to the in
vivo clinical effect of the product in the Phase 3 trial in
children with SR-aGVHD, including survival and biomarkers of in
vivo activity
- Mesoblast will provide these new
data to FDA and address all chemistry, manufacturing and controls
(CMC) outstanding items as required for the planned BLA
resubmission. If the resubmission is accepted, CBER will consider
the adequacy of the clinical data in the context of the related CMC
issues noted above
Inflammatory Bowel Disease
- The results of the first cohort of
patients from the randomized, controlled study of remestemcel-L by
direct endoscopic delivery to areas of inflammation in patients
with medically refractory ulcerative colitis or Crohn’s colitis
were presented at the 17th Congress of European Crohn’s and Colitis
Organisation (ECCO) by the trial’s lead investigator Dr. Amy L.
Lightner, Associate Professor of Surgery in the Department of Colon
and Rectal Surgery at Cleveland Clinic
- Results from an interim analysis of
the first twelve patient cohort were published in the Journal of
Crohn's and Colitis.2,3 A single local delivery of remestemcel-L by
colonoscopy resulted in rapid mucosal healing, improved clinical
and endoscopic scores as early as two weeks following
remestemcel-L, and a high incidence of disease remission by six
weeks. Dr. Lightner is scheduled to present results from additional
patients at the world’s premier meeting for physicians, researchers
and industry professionals in the fields of gastroenterology,
hepatology, endoscopy and gastrointestinal surgery, Digestive
Disease Week® (DDW) next month
COVID-19 acute respiratory distress syndrome (ARDS)
- Clinical outcomes from the pilot
study of remestemcel-L in 11 ventilator-dependent COVID-19 patients
with ARDS treated under emergency investigational new drug (eIND)
applications at Mt Sinai Hospital in New York, was published in the
peer-reviewed journal Cytotherapy this month. The manuscript4
titled ‘Mesenchymal Stromal Cell Therapy for Acute Respiratory
Distress Syndrome due to COVID-19’ provides the pilot experience
with remestemcel-L in 11 ventilator-dependent patients (10/11 were
< 65 years) in the intensive care unit (ICU) with
moderate/severe ARDS, 82% of whom were successfully discharged from
the ICU. The authors noted that there are currently no approved
therapies for COVID-19 ARDS and concluded that the case series
demonstrated remestemcel-L infusions to treat moderate to severe
COVID-19 ARDS were safe, well-tolerated, and resulted in improved
clinical outcomes including liberation from mechanical ventilation
and discharge from the ICU and/or hospital. These data formed the
dosing strategy for a subsequent randomized controlled trial in 222
patients with moderate-severe COVID ARDS. Mesoblast continues to be
in discussions with its collaborators to begin a trial to confirm
the observed outcomes in patients < 65, with a potential pathway
to seek Emergency Use Authorization
Key updates on rexlemestrocel-L:
Chronic Heart Failure
- Dr. Eugene Braunwald who has often
been called the father of modern cardiology and the most frequently
cited author in cardiology,5 this month published an article in
European Heart Journal titled Cardiac cell therapy: a call for
action.6 The paper highlighted next generation mesenchymal stromal
(bone marrow-derived) cells as attractive candidates for cardiac
cell therapy (CCT). He specifically highlighted the clinical
outcomes observed in Mesoblast’s DREAM-HF Phase 3 trial and pointed
out the company’s commercial leadership globally in the field of
CCT for heart failure
- Mesoblast received feedback last
quarter from FDA confirming that reduction in major adverse
cardiovascular events (MACE) of cardiovascular mortality or
irreversible morbidity (non-fatal heart attack or stroke) is an
acceptable clinically meaningful endpoint for determining the
treatment benefit of rexlemestrocel-L for patients with chronic
heart failure and low ejection fraction (HFrEF). Subsequently
provided FDA with top-line results of new analyses in pre-specified
high-risk groups in the DREAM-HF Phase 3 trial of rexlemestrocel-L
in HFrEF which showed that the greatest treatment benefit is in
patients with diabetes and/or myocardial ischemia (72% of total
treated population), a target population at very high risk for
mortality and irreversible morbidity due to micro- and
macro-vascular disease despite receiving optimal standard of care
therapies 7
- Mesoblast expects to receive
guidance from FDA on a potential pathway to approval following
detailed review of the outcomes identified in high-risk HFrEF
patients with diabetes and/or myocardial ischemia
Chronic Low Back Pain
- Presented 36-month follow-up results
from the 404-patient Phase 3 trial of rexlemestrocel-L (MPC-06-ID)
in patients with chronic low back pain (CLBP) associated with
degenerative disc disease (DDD) which showed durable reduction in
back pain lasting at least three years from a single intra-discal
injection of rexlemestrocel-L+hyaluronic acid (HA) carrier
- Mesoblast received feedback last
quarter from FDA on the Phase 3 program for CLBP and plans to
conduct an additional US Phase 3 trial which may support
submissions for potential approval in both the US and EU. Following
review of the completed Phase 3 trial data, FDA 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
Other:
Salary payments to full-time Executive Directors were US$289,088
and fees to Non-Executive Directors were US$207,296, detailed in
Item 6 of the Appendix 4C cash flow report for the quarter.8
A copy of the Appendix 4C – Quarterly Cash Flow Report for the
third quarter FY2022 is available on the investor page of the
company’s website www.mesoblast.com.
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
- TEMCELL® HS Inj. is a registered trademark of JCR
Pharmaceuticals Co. Ltd.
- Lightner A., et al. A Phase IB/IIA study of remestemcel-L, an
allogeneic bone marrow derived mesenchymal stem cell product, for
the treatment of medically refractory Crohn’s colitis: A
preliminary analysis. Journal of Crohn’s and Colitis, Volume 16,
Issue Supplement_1, January 2022, Pages i412–i413,
https://doi.org/10.1093/ecco-jcc/jjab232.555
- Lightner A., et al. A Phase IB/IIA study of remestemcel-L, an
allogeneic bone marrow derived mesenchymal stem cell product, for
the treatment of medically refractory ulcerative colitis: An
interim analysis. Journal of Crohn’s and Colitis, Volume 16, Issue
Supplement_1, January 2022, Pages i398–i399,
https://doi.org/10.1093/ecco-jcc/jjab232.534
- Whittaker Brown S., et al. Mesenchymal Stromal Cell Therapy for
Acute Respiratory Distress Syndrome due to COVID-19. Cytotherapy,
April 2022, https://doi.org/10.1016/j.jcyt.2022.03.006
- Dunlay SM., et al. Circulation. 2019;140:e294–e324
- Neill US. Conversations with Giants in Medicine – A
conversation with Eugene Braunwald. J Clin Invest. 2013;123(1):1-2.
https://doi.org/10.1172/JCI67778
- Braunwald E. Cardiac cell therapy: a call for action. European
Heart Journal (2022) 00, 1–2,
https://doi.org/10.1093/eurheartj/ehac188
- As required by ASX listing rule 4.7 and reported in Item 6 of
the Appendix 4C, reported are the aggregated total payments to
related parties being Executive Directors and Non-Executive
Directors
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 (including BLA resubmission), 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 maintain established strategic
collaborations; Mesoblast’s ability to establish and maintain
intellectual property on its product candidates and Mesoblast’s
ability to successfully defend these in cases of alleged
infringement; the scope of protection Mesoblast is able to
establish and maintain for intellectual property rights covering
its product candidates and technology; estimates of Mesoblast’s
expenses, future revenues, capital requirements and its 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
from those which may be expressed or implied by such statements,
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
otherwise.
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 |
|
Tali Mackay |
|
E: tmackay@rubenstein.com |
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