Mesoblast Ltd ADR (MESO)

[Docstemcell2020]

I just halted my major krapp for the night. :)

[Sage7243]

Check Twitter. It's a 7% discount to current AU price. But, covered by investment firms.

[randychub]

100 million finance - not sure of the price

[JPetroInc]

--Mesoblast American Depositary Shares Halted Pending Release of News

BY MT Newswires

— 7:13 AM ET 05/11/2020

07:13 AM EDT, 05/11/2020 (MT Newswires) --

Price: 11.39, Change: -0.01, Percent Change: -0.09

MT Newswires does not provide investment advice. Unauthorized reproduction is strictly prohibited.

[Docstemcell2020]

Psti To the moon today!

[WinWithBricks]

Fresh, positive news frequently, moving forward. The slow steady progress just weeding out the weak hands. MESO is moving along well and quickly. This is going to skyrocket once reports of trials breaks.

[frugallife]

Never thought i would short a stock bet against a stock. But if i can invest in the OTC mostly fake company why not short one that is loosing interest. Back to 9 tomorrow.

[cjstocksup]

MESO should be a couple hundred dollar stock ahead. Just with last weeks news we should be well over $100.00 per share.

MESO Info - small trial (MSC) in London.

China 7 patients - https://t.co/ZhY9iYSiTK?amp=1


The stem cell therapy is currently being reviewed by the FDA for potential approval in the treatment of children with steroid-refractory acute graft-vs.-host disease (aGVHD). The company submitted the final module of a rolling BLA in January 2020. The FDA has set a PDUFA date of Sept. 30 for the product branded as Ryoncil. The clinical data submitted with the BLA showed a survival rate of 79% compared to an expected 30% survival rate in the pediatric phase III trial in aGVHD. “Up to now we’ve been focused on GVHD, so we’ll need to ramp up manufacturing,” the CEO said, adding that he is in active partnering discussions. MESO CHRONIC HEART FAILURE Phase 3 trial (DREAM) 566 randomized patients shall report top-line any time now. 1) an interim futility test which tested safety and efficacy of primary endpoints which was successfully passed. 2) the recent comments of the Data Monitoring Controller at the end of the trial, who is the only person who is un-blinded throughout. 3) Mesoblast having already achieved a major reduction in ischemic cardiac bleeding events in the independently sponsored LVAD trials. 4) the placebo adjusted Results of the phase Two MPC 150m dosage in showing significant improvement in systolic performance of the left ventricle CHRONIC LOWER BACK PAIN Phase 3 trial has just ended and awaiting results. In a Phase 2 trial of 100 patients, randomized placebo/controlled, a single injection of MSC's into the disc in patients with severe chronic disabling back pain resulted in substantial improvement in pain and improvement in function for up to two years in a significantly greater proportion of patients than in the placebo group. The degree of improvement in pain and improvement in function was of an order that was very similar to what one would expect if you underwent a surgical procedure to replace the disc and this is with a simple outpatient procedure and injection that takes no more than about 20 minutes. (Mid 2020)

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[randychub]

MESO Info - small trial (MSC) in London.

China 7 patients - https://t.co/ZhY9iYSiTK?amp=1


The stem cell therapy is currently being reviewed by the FDA for potential approval in the treatment of children with steroid-refractory acute graft-vs.-host disease (aGVHD). The company submitted the final module of a rolling BLA in January 2020. The FDA has set a PDUFA date of Sept. 30 for the product branded as Ryoncil. The clinical data submitted with the BLA showed a survival rate of 79% compared to an expected 30% survival rate in the pediatric phase III trial in aGVHD. “Up to now we’ve been focused on GVHD, so we’ll need to ramp up manufacturing,” the CEO said, adding that he is in active partnering discussions. MESO CHRONIC HEART FAILURE Phase 3 trial (DREAM) 566 randomized patients shall report top-line any time now. 1) an interim futility test which tested safety and efficacy of primary endpoints which was successfully passed. 2) the recent comments of the Data Monitoring Controller at the end of the trial, who is the only person who is un-blinded throughout. 3) Mesoblast having already achieved a major reduction in ischemic cardiac bleeding events in the independently sponsored LVAD trials. 4) the placebo adjusted Results of the phase Two MPC 150m dosage in showing significant improvement in systolic performance of the left ventricle CHRONIC LOWER BACK PAIN Phase 3 trial has just ended and awaiting results. In a Phase 2 trial of 100 patients, randomized placebo/controlled, a single injection of MSC's into the disc in patients with severe chronic disabling back pain resulted in substantial improvement in pain and improvement in function for up to two years in a significantly greater proportion of patients than in the placebo group. The degree of improvement in pain and improvement in function was of an order that was very similar to what one would expect if you underwent a surgical procedure to replace the disc and this is with a simple outpatient procedure and injection that takes no more than about 20 minutes. (Mid 2020)

[cjstocksup]

MESO - ARDs related MSC data

https://www.sciencedirect.com/science/article/pii/S2095809920300370


The study was not blinded and probably not randomized given the unusual ratio of treated (17) to control patients (44). All patients received anti-virals, most received antibiotics. A higher percentage of patients in the treated group received hepatic and renal support, despite similar baseline chemistry numbers in both groups. So, potential confounding variables. Treated patients did have a higher percentage with shock (dangerously low blood pressure) or treatment with extra-corporeal membrane oxygenation, markers for higher risk of mortality. Taking all that into consideration the results remain striking. Following treatment with MSC's patients survived at 3 times the rate of untreated patients (p=.006 indicating high statistical significance). Importantly, follow up on 4 patients at 5 years showed no evident adverse effects from treatment

[cjstocksup]

Read the board and other filings and DD. They have cash so no they should not. You never know but I doubt it highly.

[shurtha2000]

any chance this does an offering Monday morning or this week

[jdlamont]

I bought MESO Thursday. Figured results were good ahead of time after reading PR news fro FLDM.
What a gift.

[Golden Cross]

Not true but believe what you want...LOL

[cjstocksup]

Charts are completely irrelevant here they have never had any big drug news.

[cjstocksup]

Look at he market caps between MESO and MRNA. We should be well over $100.00 to $150.00 in comparison. I hope they gap it up huge tomorrow again.

[Golden Cross]

$MESO Daily chart...

[Golden Cross]

Huge move Friday...Looking good!

[ClayTrader]

* * $MESO Video Chart 04-24-2020 * *

Link to Video - click here to watch the technical chart video

[Volcano]

If anybody looking for next 500% runner in a week

[crudeoil24]

Huge news > Mesoblast Reports 10 Out Of 12 Patients With Ventilator-Dependent Coronavirus Infection Survived Following 2 Intravenous Infusions Of Co.'s Allogeneic Mesenchymal Stem Cell Product Candidate Remestemcel-L

[crudeoil24]

Going GANGBUSTERS

[crudeoil24]

17.17 > circuit breaker halt.

[crudeoil24]

Go time!

[balamidas]

MRNA 15 B Market cap and MESO will be at 1.6 B at open !


way to go

could GO as high as MRNA with such results !

[crudeoil24]

GILD money setting up camp here > MESO

[TheFinalCD]

WOW thats GIGANTIC NEWS, considering GILD's fail

Mesoblast Reports 10 Out Of 12 Patients With Ventilator-Dependent Coronavirus Infection Survived Following 2 Intravenous Infusions Of Co.'s Allogeneic Mesenchymal Stem Cell Product Candidate Remestemcel-L
6:04 am ET April 24, 2020 (Benzinga) Print
Mesoblast Limited (NASDAQ:MESO, ASX:MSB)) today announced 83% survival in ventilator-dependent COVID-19 patients (10/12) with moderate/severe acute respiratory distress syndrome (ARDS) treated during the period March-April 2020 with two intravenous infusions of Mesoblast’s allogeneic mesenchymal stem cell product candidate remestemcel-L within the first five days. 75% (9/12) have successfully come off ventilator support at a median of 10 days. At this time, seven have been discharged from the hospital. Patients received a variety of experimental agents prior to remestemcel-L. All patients were treated under an emergency Investigational New Drug (IND) application or expanded access protocol at New York City’s Mt Sinai hospital.

In contrast, only 9% (38/445) of ventilator-dependent COVID-19 patients at a major referral hospital network in New York City were able to come off ventilator support when treated with standard of care during March/April 2020.1 Moreover, there was 88% mortality with only 12% survival (38/320) among ventilator-dependent COVID-19 patients at a second major referral hospital network in New York City during the same period.2 These poor outcomes are consistent with earlier published data from China where mortality rates of over 80% were reported in patients with COVID-19 and moderate/severe ARDS.3

Mesoblast Chief Executive Dr Silviu Itescu stated: “The remarkable clinical outcomes in these critically ill patients continue to underscore the potential benefits of remestemcel-L as an anti-inflammatory agent in cytokine release syndromes associated with high mortality, including acute graft versus host disease and COVID-19 ARDS. We intend to rapidly complete the randomized, placebo-controlled Phase 2/3 trial in COVID-19 ARDS patients to rigorously confirm that remestemcel-L improves survival in these critically ill patients.”

Mesoblast Chief Medical Officer Dr Fred Grossman said: “There is a significant need to improve the dismal survival outcomes in COVID-19 patients who progress to ARDS and require ventilators. We have implemented robust statistical analyses in our Phase 2/3 trial as recommended by the US Food and Drug Administration (FDA) in order to maximize our ability to evaluate whether remestemcel-L provides a survival benefit in moderate/severe COVID-19 ARDS.”

References
1 Petrilli CM et al. Factors associated with hospitalization and critical illness among 4,103 patients with COVID-19 disease in New York City. MedRxiv 2020 doi: https://www.medrxiv.org/content/10.1101/2020.04.08.20057794v1.full.pdf

2 Richardson S et al. Presenting characteristics, comorbidities, and outcomes among 5700 patients hospitalized with COVID-19 in the New York City area. JAMA 2020. doi:10.1001/jama.2020.6775

3 Liu Y et al. Clinical features and progression of acute respiratory distress syndrome in coronavirus disease 2019. MedRxiv 2020; https://www.medrxiv.org/content/10.1101/2020.04.08.20057794v1.full.pdf

© 2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
© 2020 InvestorsHub.com, Inc.

[crudeoil24]

Mesoblast Reports 10 Out Of 12 Patients With Ventilator-Dependent Coronavirus Infection Survived Following 2 Intravenous Infusions Of Co.'s Allogeneic Mesenchymal Stem Cell Product Candidate Remestemcel-L
6:04 am ET April 24, 2020 (Benzinga) Print
Mesoblast Limited (NASDAQ:MESO, ASX:MSB)) today announced 83% survival in ventilator-dependent COVID-19 patients (10/12) with moderate/severe acute respiratory distress syndrome (ARDS) treated during the period March-April 2020 with two intravenous infusions of Mesoblast’s allogeneic mesenchymal stem cell product candidate remestemcel-L within the first five days. 75% (9/12) have successfully come off ventilator support at a median of 10 days. At this time, seven have been discharged from the hospital. Patients received a variety of experimental agents prior to remestemcel-L. All patients were treated under an emergency Investigational New Drug (IND) application or expanded access protocol at New York City’s Mt Sinai hospital.

In contrast, only 9% (38/445) of ventilator-dependent COVID-19 patients at a major referral hospital network in New York City were able to come off ventilator support when treated with standard of care during March/April 2020.1 Moreover, there was 88% mortality with only 12% survival (38/320) among ventilator-dependent COVID-19 patients at a second major referral hospital network in New York City during the same period.2 These poor outcomes are consistent with earlier published data from China where mortality rates of over 80% were reported in patients with COVID-19 and moderate/severe ARDS.3

Mesoblast Chief Executive Dr Silviu Itescu stated: “The remarkable clinical outcomes in these critically ill patients continue to underscore the potential benefits of remestemcel-L as an anti-inflammatory agent in cytokine release syndromes associated with high mortality, including acute graft versus host disease and COVID-19 ARDS. We intend to rapidly complete the randomized, placebo-controlled Phase 2/3 trial in COVID-19 ARDS patients to rigorously confirm that remestemcel-L improves survival in these critically ill patients.”

Mesoblast Chief Medical Officer Dr Fred Grossman said: “There is a significant need to improve the dismal survival outcomes in COVID-19 patients who progress to ARDS and require ventilators. We have implemented robust statistical analyses in our Phase 2/3 trial as recommended by the US Food and Drug Administration (FDA) in order to maximize our ability to evaluate whether remestemcel-L provides a survival benefit in moderate/severe COVID-19 ARDS.”

References
1 Petrilli CM et al. Factors associated with hospitalization and critical illness among 4,103 patients with COVID-19 disease in New York City. MedRxiv 2020 doi: https://www.medrxiv.org/content/10.1101/2020.04.08.20057794v1.full.pdf

2 Richardson S et al. Presenting characteristics, comorbidities, and outcomes among 5700 patients hospitalized with COVID-19 in the New York City area. JAMA 2020. doi:10.1001/jama.2020.6775

3 Liu Y et al. Clinical features and progression of acute respiratory distress syndrome in coronavirus disease 2019. MedRxiv 2020; https://www.medrxiv.org/content/10.1101/2020.04.08.20057794v1.full.pdf

© 2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

[jdlamont]

looks like both doing the exact same thing and both have clearance by FDA for compassionate use.
CTSO probably works a lot faster, but you need their machine vs. an I.V. infusion

[Docstemcell2020]

Psti

[jdlamont]

BIG NEWS BIG NEWS

Uncovered MSC study showing efficacy positive outcomes in Chinese study in COVID 19 patients
Came across it on FLDM PR dated April 2, 2020. check it out.

[edwardport]

recent news look promising for MESO

MESOBLAST PARTNERS WITH THE CARDIOTHORACIC SURGICAL TRIALS NETWORK ESTABLISHED BY THE U.S. NATIONAL INSTITUTES OF HEALTH’S NATIONAL HEART, LUNG AND BLOOD INSTITUTE TO CONDUCT RANDOMIZED CONTROLLED TRIAL OF REMESTEMCEL-L FOR PATIENTS WITH ACUTE RESPIRATORY DISTRESS SYNDROME DUE TO COVID-19

[biosectinvestor]

Mesoblast Submits Clinical Efficacy And Safety Data To FDA In Rolling Biologics License Application For Remestemcel-L

https://finance.yahoo.com/news/mesoblast-submits-clinical-efficacy-safety-110010878.html

[biosectinvestor]

MESO is looking good today... and is off to a good start for the year.

[Fenix1357]

The fact that I can not find any published papers on the phase two trial for back pain worry me, imply there were some flaws in the study which kept them from publishing the paper or being accepted by scientific journals. They have no p values published either

[Fenix1357]

"This Phase 3 clinical trial will use a primary endpoint that comprises both pain relief and improved function, consisting of a 50% reduction in lower back pain as measured by Visual Analog Score and a 15-point improvement in Oswestry Disability Index, with no additional interventions."

This 50% reduction in pain seems very ambitious. I wish they had kept it to a meaningful reduction in pain, like all other pain trials which means a 20% reduction in pain as measured by VAS.

I wonder what their phase two results and p value were.

[XenaLives]

Mesoblast Appoints Leading Pharmaceutical Industry Executive as Chief Medical Officer
Date : 08/12/2019 @ 6:00AM
Source : GlobeNewswire Inc.
Stock : Mesoblast Limited (MESO)
Quote : 5.12 0.0 (0.00%) @ 4:00AM

Mesoblast Appoints Leading Pharmaceutical Industry Executive as Chief Medical Officer

Today : Tuesday 13 August 2019

Mesoblast Limited (Nasdaq: MESO; ASX: MSB), global leader in cellular medicines for inflammatory diseases, is pleased to announce the appointment of Dr Fred Grossman as Chief Medical Officer (CMO), based in New York. Dr Grossman brings a wealth of commercial experience gained from numerous leadership roles at global pharmaceutical companies. The appointment aligns closely with the Company’s commercial objectives for its lead products.

Dr Grossman has over 20 years of industry experience, and has held key leadership positions at major global pharmaceutical companies, including Eli Lilly, Johnson & Johnson (J&J), Bristol Myers Squibb (BMS), Sunovion, and Glenmark. During his career, he has managed global clinical development, pharmacovigilance, medical affairs and clinical operations for innovative product development, as well as United States Food and Drug Administration (FDA) approvals and post-market support for numerous blockbuster, specialty and generic products. Dr Grossman has led and built teams in the United States, Europe and Japan with responsibility for global medical affairs, global clinical development, health economics & outcomes research (HEOR) and global drug safety.

At J&J and Eli Lilly, Dr Grossman was responsible for multiple New Drug Applications (NDAs) to the FDA. At BMS, he was Global Head of the Medical Affairs organization, with focus on immunology and cardiovascular diseases. As Head of Clinical Development, Medical Affairs & Drug Safety at 6,000-person global pharma Sunovion, a subsidiary of Japan’s Sumitomo Dainippon Pharma Co., Dr Grossman had responsibility across teams in the United States, Europe and Japan and successfully launched a blockbuster product in the United States market. More recently, he was CMO at Glenmark, an India-based global pharma with 12,000 people, where he oversaw a portfolio spanning biologics, branded drugs, and generics, including in immunology and immuno-oncology.

Dr Grossman said: “I am very excited to have the opportunity of working at Mesoblast, a very innovative company with a mature pipeline of cellular medicines that have the potential to address some of the most severe diseases affecting people worldwide. I look forward to utilizing the experience gained from many product launches to ensure that Mesoblast successfully transitions to a commercial organization with the planned launch of its first product in the United States.”

Mesoblast Chief Executive Dr Silviu Itescu thanked outgoing CMO Dr Donna Skerrett for her valuable contributions during the Company’s development stages. Dr Skerrett will continue in an advisory role.

Dr Itescu welcomed Dr Grossman to the Company, stating: “Fred brings to Mesoblast extensive industry experience which is extremely valuable to the strategic and execution phases of our commercial transition as we prepare for our first FDA Biologics License Application (BLA) and commercial product launch in the United States market. His strong track record of building high-performing teams and setting corporate culture make him ideally suited to lead our late-stage product development strategies and commercial execution.”

About Mesoblast
Mesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary technology platform to establish a broad portfolio of late-stage product candidates with three product candidates in Phase 3 trials – acute graft versus host disease, chronic heart failure and chronic low back pain due to degenerative disc disease. Through a proprietary process, Mesoblast selects rare mesenchymal lineage precursor and stem cells from the bone marrow of healthy adults and creates master cell banks, which can be industrially expanded to produce thousands of doses from each donor without the need for tissue matching. Mesoblast has facilities in Melbourne, New York, Singapore and Texas and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). www.mesoblast.com

[XenaLives]

Circulation Research Special Article Highlights Potential Of Mesoblast Cell Therapy In Treatment Of Advanced Heart Failure

Mesoblast Limited (Nasdaq: MESO; ASX: MSB) today announced that premier cardiovascular journal Circulation Research has published a Special Article highlighting the important potential clinical benefits of Mesoblast’s allogeneic mesenchymal precursor cell (MPC) technology platform as immunotherapy in patients with advanced chronic heart failure.
The Special Article highlighted that cardiac inflammation drives heart failure progression, and concluded that based on preclinical and Phase 2 clinical data there is a biologic rationale for the use of Mesoblast’s MPCs in targeting this inflammatory process in order to improve heart failure outcomes.

The manuscript, titled ‘Phase 3 DREAM-HF Trial of Mesenchymal Precursor Cells in Chronic Heart Failure; A Review of Biological Plausibility and Implementation of Flexible Clinical Trial Design,’ was requested by the journal’s Editor-in-Chief. Its authors include the Phase 3 trial’s co-principal investigators, Dr Emerson Perin, Texas Heart Institute, and Dr Barry Greenberg, University of California, San Diego Healthcare System. The article can be accessed at https://doi.org/10.1161/CIRCRESAHA.119.314951.

The ongoing, placebo-controlled double-blind Phase 3 trial of Mesoblast’s heart failure cellular medicine Revascor, comprising 150 million MPCs, is evaluating the immunotherapy for reduction of heart failure-related hospitalizations and terminal cardiac events in patients with advanced heart failure. The events-driven trial completed enrollment of 566 patients in February 2019, and was previously successful in a pre-specified interim futility analysis of the primary efficacy endpoint in the first 270 patients.

Revascor is also being evaluated to prevent mucosal bleeding in end-stage chronic heart failure patients with a Left Ventricular Assist Device (LVAD) and recently received Orphan Drug Designation from the United States Food and Drug Administration (FDA) for this indication. Mesoblast is in discussions with the FDA regarding a potential approval pathway under the product’s existing Regenerative Medicine Advanced Therapy (RMAT) designation for this life-threatening condition.

https://www.globenewswire.com/news-release/2019/07/25/1887815/0/en/Circulation-Research-Special-Article-Highlights-Potential-Of-Mesoblast-Cell-Therapy-In-Treatment-Of-Advanced-Heart-Failure.html

[gesundheit]

Gesundheit : Meso gets orphand drug approval from FDA in USA .

https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=661518

[XenaLives]

Any biotech knowledgable folk willing to discuss this stock?

My cursory overview says this may be cutting edge stuff but I don't have the background to pontificate.

[XenaLives]

FDA Agrees to Rolling Review of Mesoblast’s Biologics License Application for Its Cell Therapy in Children With Steroid-Refractory Acute Graft Versus Host Disease
Email Print Friendly Share
April 16, 2019 06:00 ET | Source: Mesoblast Limited
NEW YORK and MELBOURNE, Australia, April 16, 2019 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) global leader in cellular medicines for inflammatory diseases, announced today that the United States Food and Drug Administration (FDA) has agreed that Mesoblast can submit on a rolling basis a Biologics License Application (BLA) for its allogeneic cellular medicine remestemcel-L in children with steroid-refractory acute Graft Versus Host Disease (SR-aGVHD).

Mesoblast will submit each module of the BLA to the FDA on a rolling basis as it is completed. The rolling process will provide opportunity for ongoing and frequent communication, and during this process the Company expects it will be able to adequately address any substantial matters raised by the FDA.

Mesoblast has previously received Fast Track designation from the FDA for remestemcel-L in SR-aGVHD and is eligible for priority review once the BLA filing is completed and accepted by the FDA. Mesoblast expects to submit the first module shortly.

About Steroid-refractory Acute Graft Versus Host Disease
SR-aGVHD is a life-threatening complication of a bone marrow transplant in patients primarily being treated for blood cancers. There are more than 30,000 allogeneic bone marrow transplants performed globally, with a total of over 20,000 in the United States and the EU5 annually. Approximately 20% occur in children. Currently, there are no approved products for SR-aGVHD in children outside Japan, where Mesoblast licensee JCR Pharmaceuticals markets TEMCELL®1 HS Inj. for both children and adults with aGVHD.

About Mesoblast
Mesoblast Limited (Nasdaq:MESO; ASX:MSB) has leveraged its proprietary immunomodulatory cellular technology platform to establish a broad portfolio of late-stage allogeneic (off-the-shelf) product candidates with three product candidates in Phase 3 trials – acute graft versus host disease, chronic heart failure and chronic low back pain due to degenerative disc disease. Mesoblast has developed proprietary cell manufacturing processes, including use of immunoselection, master cell banks, industrialized culture expansion, potency assays, and stringent lot release criteria, to enable large-scale production of cellular therapies that can be used with off-the-shelf logistics from a single donor to thousands of unrelated recipients. Mesoblast has facilities in Melbourne, New York, Singapore and Texas and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). www.mesoblast.com

1. TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.

Forward-Looking Statements
This 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 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 timing, progress and results of Mesoblast’s preclinical and clinical studies; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; 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.

For further information, please contact:

Julie Meldrum Schond Greenway
Corporate Communications Investor Relations
T: [phone] T: [phone]
E: [e-mail] E: [e-mail]

https://www.globenewswire.com/news-release/2019/04/16/1804538/0/en/FDA-Agrees-to-Rolling-Review-of-Mesoblast-s-Biologics-License-Application-for-Its-Cell-Therapy-in-Children-With-Steroid-Refractory-Acute-Graft-Versus-Host-Disease.html

[XenaLives]

Age related inflammation causes general havoc..

CHRONIC LOW BACK PAIN DUE TO DISC DEGENERATION
MPC-06-ID is a Phase 3 product candidate for the treatment of chronic low back pain caused by disc degeneration (CLBP). It is being developed for patients who have exhausted conservative treatment options, may have failed epidural steroid injections and have no further treatment option other than invasive and costly surgical interventions.

Disease Indication and Patient Population

Over four million patients in the United States alone suffer from CLBP1,2,3,4 , which is caused by damage to the disc as a result of aging, genetics, and injuries. This compromises the disc’s capacity to act as a fluid-filled cushion between vertebrae and to provide anatomical stability. Damage also causes an inflammatory response with ingrowth of nerves that result in chronic pain. This combination results in CLBP and functional disability.

When disc degeneration has progressed to a point that pain and loss of function can no longer be managed by conservative means e.g. medication and physiotherapy, major invasive surgery such as spinal fusion is the only remaining option.

Existing therapies treat the symptoms of the disease, but are not disease-modifying and thus do not address the underlying disease cause. As a result, we believe that the most significant unmet need is a therapy that can not only improve the patient’s pain and function, but has the ability to reverse, halt or slow disease progression.

By treating the cause of CLBP, we believe MPC-06-ID could fill an unmet treatment gap for a large population of patients.

MPC-06-ID

MPC-06-ID is a tier 1 product candidate which consists of a unit dose of 6 million mesenchymal precursor cells (MPCs). It is injected by syringe directly into a targeted damaged disc in an outpatient procedure.

Mechanism of Action

Extensive preclinical studies have established that MPCs have anti-inflammatory effects and secrete multiple paracrine factors that stimulate new proteoglycan and collagen synthesis by chondrocytes in vitro and by resident cells in the nucleus and annulus in vivo. MPCs have also been shown to produce anti-inflammation factors. Together these effects offer the potential to strengthen the load bearing function of the disc by increasing its water content, improving disc anatomy and stability, while also reducing inflammation and pain.

Clinical Trials

Ongoing Phase Trial 3

A Phase 3 clinical program has completed enrollment of 404 patients.

This Phase 3 clinical trial will use a primary endpoint that comprises both pain relief and improved function, consisting of a 50% reduction in lower back pain as measured by Visual Analog Score and a 15-point improvement in Oswestry Disability Index, with no additional interventions.

Find out more about this Phase 3 trial.

Phase 2 Trial

MPC-06-ID was evaluated in a randomized, placebo and active controlled Phase 2 trial conducted in 100 patients with more than six months of CLBP due to disc degeneration.

In March 2017, 36-month results were released and showed that a single intra-discal injection of 6 million MPCs resulted in meaningful improvements in both pain and function that were durable for at least three years. Previously, 24-month results were presented at the 24th Annual Scientific Meeting of the Spine Intervention Society in July 2016, and received the 2016 Best Basic Science Abstract award at the New Orleans meeting.

_________________________________________________

1Andersson GBJ. Epidemiological features of chronic low-back pain. Lancet 1999; 354: 581-85.

2Freburger et al. The Rising Prevalence of Chronic Low Back Pain. Arch Intern Med 2009; 169 (3): 251-258

3Malanga G et al. Epidemiology In: Cole & Herring eds. The Low Back Pain Handbook: A Guide for the Practicing Clinician. 2nd ed. Philadelphia, Pa.: Hanley and Belfus, 2003: 1-7

4DePalma MJ et al. What is the source of chronic low back pain and does age play a role? Pain Med 2011; 12:224-233

https://www.mesoblast.com/product-candidates/spine-orthopedic-disorders/chronic-discogenic-low-back-pain

[XenaLives]

Agreed, the FDA is broken. I hope we get someone as effective as Gottleib to head the FDA. Perhaps Woodcock can get the job done.

[XenaLives]

The FDA system is way too slow, we need more efficient genetic analysis when a drug works in some patients and not in others and testing in the patients most likely to succed.

CHRONIC HEART FAILURE
MPC-150-IM is a Phase 3 product candidate being developed as a treatment for both advanced and end-stage chronic heart failure (CHF).

Disease Indication and Patient Population

CHF is characterized by an enlarged heart and insufficient blood flow to the organs and extremities of the body. The condition, which affects 2% of the adult population of the United States, is progressive and can be caused by many factors that put an excess demand on the heart muscle such as high blood pressure, faulty valves, infections or congenital heart problems. CHF prevalence is expected to grow 46% by 2030, affecting more than 8 million Americans1.

CHF is classified in relation to the severity of the symptoms experienced by the patient. The most commonly used classification system was established by the New York Heart Association (NYHA) and ranges from Class I (mild) to Class IV or end stage (severe).

Patients with advanced or Class II/III heart failure disease continue to represent the greatest unmet medical need despite recent advances in new therapies.

MPC-150-IM

MPC-150-IM is a tier 1 product candidate which consists of 150 million mesenchymal precursor cells (MPCs) administered by direct injection into the heart muscle in patients suffering from CHF and progressive loss of heart function.

Mechanism of Action

MPCs release a range of factors when triggered by specific receptor-ligand interactions within damaged tissue. Based on preclinical data, it is believed that these factors induce functional cardiac recovery by simultaneous activation of multiple pathways, including induction of endogenous vascular network formation, reduction in harmful inflammation, reduction in cardiac scarring and fibrosis, and regeneration of heart muscle through activation of tissue precursors.

Clinical Trials

Ongoing Phase 3 Trial

Approximately 600 patients are being enrolled in a placebo-controlled Phase 3 trial to evaluate a single dose of MPC-150-IM in Class II/III CHF patients across multiple sites in North America. Patients with advanced heart failure are expected to constitute the majority of the patients enrolled in this clinical trial program.

The objectives of this Phase 3 events-driven trial are to evaluate the ability of MPC-150-IM to reduce the primary endpoint of recurrent non-fatal heart failure-related major adverse cardiac events (HF-MACE) in patients with left ventricular dysfunction, as well as delay or prevent disease progression to end-stage HF and terminal cardiac events, defined as death, left ventricular assist device (LVAD) implantation, or cardiac transplant.

In April 2017, the trial achieved a successful pre-specified interim futility analysis of the efficacy endpoint in the first 270 patients and an independent data monitoring committee formally recommended the continuation of the trial.

Additional information about this trial is available at: https://clinicaltrials.gov/ct2/show/NCT02032004 and at http://www.dreamheartfailure.com.

Phase 2 Trial

A Phase 2 dose-ranging study in patients with Class II/III (New York Heart Association) heart failure has been completed. Click here for clinical trial results.

End-Stage Heart Failure

MPC-150-IM is also under clinical investigation for the treatment of end-stage heart failure.

In the United States, there are approximately 250,000–300,000 patients annually who suffer from advanced systolic heart failure (NYHA Class IIIb–IV) who despite optimal medical therapy (excluding mechanical assist devices) have a one-year mortality >25% and exceeding 50% in class IV patients.1 The only options to increase survival in these patients are the use of cardiac assist devices or heart transplants. Due to the decline in organ donations and limited availability of healthy donor hearts, the treatment of CHF with mechanical circulatory support devices such as LVADs is gaining momentum, with 4,500–5,500 assist devices implanted annually in the United States.2,3,4 However, rehospitalization is frequent in patients with an LVAD ranging from 2.1–2.7 times per year. The majority of patients rehospitalized for non-device related causes are as a result of GI bleeding (34%-44%) and infections (36%-44%).5,6

Clinical Trials

Phase 2 Trial

Results of a 159-patient randomized placebo-controlled Phase 2 trial, sponsored and conducted by United States National Institutes of Health (NIH), evaluating MPC-150-IM in the treatment of end-stage heart failure patients implanted with a left ventricular assist device (LVAD) were presented at the 2018 American Heart Association Scientific Sessions.

Key outcomes:

The trial succeeded in achieving the clinically meaningful outcome of reduction in gastrointestinal (GI) bleeding and related hospitalizations
Results confirmed the previous pilot trial, which also demonstrated significant reduction in GI bleeding and related hospitalizations in MPC-150-IM treated LVAD patients
Pilot trial results formed the basis for the FDA Regenerative Medicine Advanced Therapy (RMAT) designation granted in December 2017, which aims to expedite the development of regenerative medicine therapies intended for the treatment of serious diseases and life-threatening conditions
Mesoblast intends to meet with the FDA to provide full study data and discuss the pathway to potential Biologics License Application (BLA) filing using reduction in GI bleeding and related hospitalizations as an approvable regulatory endpoint
While the trial did not meet the overall primary endpoint of temporary weaning, MPC-150-IM treatment did significantly improve weaning in the 44% of patients with chronic ischemic heart failure
LVAD patients with ischemic heart failure closely resemble the majority of patients enrolled in the ongoing Phase 3 trial of approximately 600 patients with moderate/ advanced heart failure.
Pilot trial results

The results of the pilot trial were presented at the American Heart Association Scientific Sessions 2013 and published in the journal Circulation in June 2014. Click here more information on the trial results.

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1Gustafsson F, Rogers JG. Left ventricular assist device therapy in advanced heart failure: patient selection and outcomes. European Journal of Heart Failure 2017;19:595-602

2United Network for Organ Sharing

3Agency for Healthcare Research and Quality – Healthcare Cost and Utilization Project – Claims Analysis ICD- 37.6

4Data on file

5Chatterjee A, Feldmann C, Hanke JS (2018) The momentum of HeartMate 3: a novel active magnetically levitated centrifugal left ventricular assist device (LVAD). J Thorac Dis 10 (Suppl 15): S1790-S1793

6Mehra, MR Salerno C, Cleveland JC (2018) Health care resources use and cost implications in the MOMENTUM 3 long-term outcome study: a randomized controlled trial of a magnetically levitated cardiac pump in advanced heart failure

https://www.mesoblast.com/product-candidates/cardiovascular-diseases/congestive-heart-failure

[XenaLives]

MPC-150-IM is our Phase 3 product candidate under investigation for the treatment of moderate to advanced chronic congestive heart failure (CHF) currently enrolling approximately 600 patients across North America.

We completed a Phase 2 trial in patients with Class II/III (New York Heart Association) CHF in 2013. Click here for clinical trial results.

The United States National Institute of Health has completed enrollment of a 159-patient Phase 2b trial using MPC-150-IM in patients with end-stage CHF requiring mechanical circulatory support.

Patients with advanced and end-stage heart failure represent a serious unmet medical need.

Learn more

MPC-06-ID is our Phase 3 product candidate under investigation for the treatment of chronic low back pain (CLBP) due to disc degeneration.

The primary objective of our Phase 2 study was to evaluate the safety of MPCs in CLBP. Secondary objectives were to evaluate efficacy parameters such as radiographic, low back pain, function/disability, medication usage, work status and quality of life improvement measures. Click here for the clinical trial results.

A Phase 3 clinical trial has completed enrollment of 404 patients.

Most current treatments for CLBP focus on pain relief rather than addressing the underlying degenerative nature of the disease. We believe MPC-06-ID has the potential to fill an unmet treatment gap for this large population of patients.

Learn more

MSC-100-IV is our Phase 3 intravenously-delivered product candidate, which is being developed for the treatment of acute Graft Versus Host Disease (aGVHD) following allogeneic bone marrow transplantation.

An open label Phase 3 trial for MSC-100-IV has completed enrollment of 55 children with steroid-refractory aGVHD in the United States.

This Phase 3 trial successfully met its primary endpoint of Day 28 Overall Response (OR) to remestemcel-L treatment, with 69% of patients achieving this endpoint compared to the protocol-defined historical control rate of 45% (p=0.0003). In patients who had a positive OR to treatment with remestemcel-L at Day 28, survival was 87% at Day 100. At Day 180, survival in these patients was 79% (p=0.001 by Kaplan-Meier survival estimates compared to non-responders). Overall Day 180 survival for the entire remestemcel-L treated group was 69%. Historical survival rates in patients with Grade C/D disease and failure to respond to steroids have been only 10-30%.

Mesoblast’s goal is for remestemcel-L to be the first commercially manufactured allogeneic cellular medicine available in the United States.

Mesoblast’s licensee in Japan for aGVHD, JCR Pharmaceuticals Co. Ltd., is marketing its its MSC-based product for children and adults with aGVHD. TEMCELL® HS Inj. was the first allogeneic cell-based product to receive full approval in Japan.

Learn more

MPC-300-IV is an intravenously-delivered immunomodulatory product candidate for the treatment of chronic inflammatory conditions.

Randomized, placebo-controlled Phase 2 trials of MPC-300-IV have been completed in patients with biologic-refractory rheumatoid arthritis and diabetic nephropathy. Both trials reported promising results in these diseases with high, unmet medical needs.

Learn more

A Phase 2 trial in patients with diabetic nephropathy was completed in 2015. The results were presented at the late-breaking scientific sessions of the 75th annual meeting of the American Diabetes Association in June 2015. Click here for results from this Phase 2 trial.

Learn more

https://www.mesoblast.com/product-candidates/lead-product-candidates

[XenaLives]

Anyone following this?

We have applied our proprietary cell technology platform, which is based on mesenchymal lineage adult stem cells (MLCs), to build what we believe is the most advanced cell-based product portfolio in the industry. This technology has the potential to address multiple conditions with significant unmet medical needs.

Understanding and capitalizing on the mechanisms of action of our proprietary MLCs has enabled us to develop MLC-based products that target specific disease states. These mechanisms of action demonstrated in preclinical models include induction of tissue repair, new blood vessel network formation, and immunomodulation.

Our ‘off-the-shelf’ products are allogenic - meaning stem cells from a single donor can be used different recipients without the need for matching.

The Japanese Government has approved TEMCELL® HS. Inj., a product based on our proprietary mesenchymal lineage adult stem cell technology licensed to JCR Pharmaceuticals Co. Ltd for the treatment of graft versus host disease. TEMCELL is the first allogeneic cell-based product to receive full regulatory approval in Japan.

We expect to have the first industrially manufactured allogeneic mesenchymal lineage cell-based product launched in the United States. The Phase 3 trial using our product candidate MSC-100-IV in children with steroid-refractory acute graft versus host disease successfully met the primary endpoint of Day 28 overall response in February 2018. Based on interactions with the FDA, Mesoblast believes that successful results from the completed Phase 3 trial through Day 100, together with Day 180 safety and quality of life parameters in these patients, may provide sufficient clinical evidence for filing for MSC-100-IV in the United States under an accelerated approval pathway.

Two other lead product candidates, MPC-150-IM and MPC-06-ID, are in late-stage development in Phase 3 programs targeting, respectively, advanced chronic heart failure and chronic low back pain due to degenerative disc disease. These are major diseases with high unmet medical needs.

Additionally, we have a strong emerging pipeline of product candidates targeting a number of inflammatory/immune-mediated conditions.

https://www.mesoblast.com/product-candidates/product-candidates-overview

[Murph1953]

FDA
https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm596554.htm?utm_campaign=02132018_Statement_FY19%20Budget&utm_medium=email&utm_source=Eloqua

[Murph1953]

January 18, 2018

Researchers create first stem cells using CRISPR genome activation

"This is a new way to make induced pluripotent stem cells that is fundamentally different from how they've been created before,"

"Pluripotent stem cells can be turned into virtually any cell type in the body. As a result, they are a key therapeutic resource for currently incurable conditions, such as heart failure, Parkinson's disease, and blindness.


https://www.sciencedaily.com/releases/2018/01/180118162449.htm
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Harvard researchers genetically ‘edit’ human blood stem cells

"HIV specifically targets T cells, enters via a gene receptor called CCR5 that serves as a doorway into the cells

"...Using the CRISPR/Cas gene-editing technology, the Cowan and Rossi teams knocked the CCR5 receptor out of blood stem cells..
https://hsci.harvard.edu/news/harvard-researchers-genetically-%E2%80%98edit%E2%80%99-human-blood-stem-cells
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Protein Switch For Functional Insulin-producing Cells Found

"For more than a decade, scientists across the globe sought to replace failing pancreatic beta cells linked to immune destruction in children (type 1 diabetes) or obesity-associated diabetes in adults (type 2 diabetes). Although cells made in a dish were able to produce insulin, they were sluggish or simply unable to respond to glucose....

"Start With Induced Pluripotent Cells

https://reliawire.com/protein-switch-insulin-cells/?utm_source=TrendMD&utm_medium=cpc&utm_campaign=Reliawire_TrendMD_1
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Discovery may make it easier to develop life-saving stem cells

".. just now figuring out how adult somatic cells such as skin cells can be turned into embryonic stem cells.

"Japanese researchers found that by introducing four other genes into cells, stem cells could be created without the use of a human egg. These cells are called induced pluripotent stem cells, or iPSCs.

"This is important because the iPSCs are derived directly from adult tissue and can be a perfect genetic match for a patient,"

https://www.sciencedaily.com/releases/2014/07/140717180534.htm
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Skin cells transformed into functioning liver cells in mouse study
https://www.sciencedaily.com/releases/2014/02/140223131703.htm
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FDA Approved Cellular and Gene Therapy Products
https://www.fda.gov/biologicsbloodvaccines/cellulargenetherapyproducts/approvedproducts/default.htm

[Murph1953]

Expanded Access to Investigational Medical Devices/drugs

"There may be circumstances under which a health care provider may wish to use an unapproved device to save the life of a patient or to help a patient suffering from a serious disease or condition for which there no other alternative therapy exists. Learn more about IDEs and the expanded access requirements for medical devices.
back to top

"In some cases, patients may have to pay for using the investigational drug and/or for medical care associated with the use of the investigational drug. In others, pharmaceutical companies may elect not to charge.

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm#Expanded_Access_to_Investigational_Medical_Devices
https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm
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We provide information and access to treatment options within Clinical Trials & Early Access Programs
"myTomorrows"

https://mytomorrows.com/en/?utm_campaign=AW_EMEAU_ENG_Brand_Desktop_Hygiene_22.01.18_BR01WWENGV2_BMM&utm_source=google&utm_medium=cpc&utm_term=%2Bcompassionate%20%2Buse&utm_content=EMEAU_ENG_HY_TextAd5V4&utm_id=787118616&gclid=EAIaIQobChMIrNn7icic2QIViz2BCh2mVAPqEAAYASAAEgLib_D_BwE
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FDA Simplifies Compassionate Use Application and Drug Costs
https://www.medscape.com/viewarticle/864310
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Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs
http://www.nejm.org/doi/full/10.1056/NEJMhle1409465
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Working Group on Compassionate Use and Pre-Approval Access
https://med.nyu.edu/pophealth/divisions/medical-ethics/compassionate-use
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Compassionate Drug Use - American Cancer Society (speaking CAR-T cancer therapy)
" Some drug companies will supply the drug for free, but others charge patients. Most insurance companies will not pay for investigational drugs. There may also be ...
https://www.cancer.org/treatment/treatments-and-side-effects/clinical-trials/compassionate-drug-use.html
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An overview of Compassionate Use Programs in the European Union member states
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5116859/

[Murph1953]

Apparently Some stem cell applications don't have to go thru Phases 1, 2 and 3, although their production standards are controlled/monitored by the gov't.

I went to their presentation last week. It was amazing "before and after".

For these kinds of uses, isn't this de facto approval?

testimonial/video (in Atlanta)

https://www.vitalityhccs.com/

Others around the country, like in Manhattan

http://regenmedicalpc.com/about/

Nothing on the market for stroke/CLI/heart = Phases 1,,2, 3