Therapy Cell Crossing Babylon? – Mesoblast (NASDAQ: MESSAGE)


Trade Stem Cell Investment

In 1924, historical studies suggested that one type of pre-cell cells within the mesenchyme was developed to a wide variety of different cell cells (ISBN 978-1-4614-7696-2). Mesenchymal cells (MSCs) are one type of cellulite that has been a major player in researchers from the 1950s; a large working group has been to analyze how MSC is going to & # 39; make differences in different types of cells, which include bone, cartilage, muscles and fat. Find it PubMed on mesenchymal cells up more than 86,000 applications that give a little indication of just how much time and money is spent in this area. Today, MSC is available as a research entrant because different types of MSC can be purchased from ATCC and commercial merchants.

Clinical scientists have made a huge effort to bring MSCs into remedies for a variety of diseases since the early 2000s. For example, meta-analysis was published in 2011 of myocardial patients Oste bone cell bone extract. Experts identified 45 cell-proofing checks that could be suitable for their analysis. Twenty-nine of these 45 tests were included in the analysis; These 29 tests consisted of 1,830 patients with MSCs. Hearts are clearly interested in a cure based on cells to cure heart disease and have made an ongoing effort to date. MI is not the only body to use MSC; Signs where MSC is used include knee laboratory, tight blades, lukodystrophy, Hurler syndrome, Crohn fistula, dose repair, hematologic cancer, arthritis, arthritis disease …

After 30-40 years of major investment, technology is not now regarded as a "hot" field of research for young researchers and financial financial managers tend to be unbelievable. A paragraph from a wild Biotech article in 2013 brings up the status: "Once it's a major new pharmaceutical pharmacist, the search of cannon cells has declined and declined in recent years with some inspiration from Big Pharma players. Sanofi went out to work with $ 1.25 billion with Osiris, Genzyme costing $ 130 million ahead. But a number of smaller players are still enthusiastic about long-term success in the long run. In the last words of an Oppenheimer analyst MSCs were pulled through the mudmixed with it of making inconsistent, inconsistent tests, and badly marked marks. "

After many fatal beginnings, are the main vines and failures in the field that are the game cells really the complex thermal agent? The article gives a brief view of Mesoblast (NASDAQ: MESO) and allows the reader to reach their own decision on whether it is worth giving them more dedication to this Australian biology .

Mesenchymal Kells:

Mesenchymal cells are found in a variety of human cannons, including bone marrow, placenta, amniotic fluid and licensed devices. In addition to the ability to create different types of knitwear, MSC has a second main function. MSCs will produce biomolecules that will be used; carry forward machine repairs or change protection response. For example, in response to spoiled blood vessels, MSCs identify growth factors that promote the restoration of blood vessels; MSCs are extracting chemokines, growth features and engines that make it possible; Promoting survival, diversity and central repairs in the heart, brain and bone marrow. Ultimately, MSC can make an emotional effect through interaction with white blood cells.

Mesoblast cells: MPCan to MLCs

In 1991, Fred Hutchinson's scientists in Seattle created monoclonal antibodies to an STRO-1 antigen that linked to about 10% of bone bone MSC; These researchers decided that this antibody could be used to produce cells beforehand for MSCs. In September 2007, a team of French antibody researchers used to STRO-1 to divide MSC STRO-1 from all other MSC in bone marrow; They then evaluated anti-MSR anti-MSC anti-functioning operations compared to a combination of all MSC in bone marrow. The two graphs below show that the growing place in mononuclear cells (PBMCs) is a & # 39; left (left graph) and presence (correct graph) of a psychologist at different levels of equality of STRO-1 / Total MSC.

Int. J Lab Hematology, Nasef et al (2009) 31: 9-19

The gray bars represent the growth of blood mononuclear cells (PBMCs) without MSCs (advanced control). Blackbirds represent the growth of PBMCs in the presence of MSCs at a variety of variants of rich Stro-1 MSC cells (the highest ratio 0.3% generates 30% of MSC STC-1 cells ). The white bars represent the growth of PBMCs presenting a similar number of unnamed MSCs. This allowed researchers to compare the STC-1 MSC related tasks for handling each MSC that was cleared to the use of the traditional methodology. These searches concluded that (1) the STRO-1 signal is transported with primitive MSCs purely and (2) MSCs STRO-1 show a greater 1,000-fold obstruction of other MSC cells a & # 39; lymphocyte expansion

At the same time, the French team hosted the MSS STRO-1. MESO scientists were working on a manuscript that revealed the isolation and ownership of repositioning separately of MSCs that they believe are a & # 39; affecting different MSCs; that is, the type of single cell from which other MSCs are coming. MESO is named after their cell mesenchymal ro-cell cells (MPC). It was an essential technical step for MESO; in identifying advanced MSC and skill to successfully complete this work.

MESO can now expand significantly on MPCs as long as they are. maintain the ability to achieve important biomolecules related to health and machine repair. This allows them to deliver a canned product with explanatory product criteria; use to use power certification. This offers a huge benefit over self-regulating cell cells that are made from the patient's cells. MESO has targeted mesenchymal adult cell cells (MLCs) that are present around the blood vessels in devices where they can respond effectively to different signs of timber damage. MLCs of a mixture of biomolecules associated with a number of repair devices related to the maintenance and repair of machinery.

MESO list their Sàr 1 (priority) materials in a corporate backdrop on their website. The items listed below are made by different cultural traditions, based on different creatures, delivered by different administrative routes and various data regulations.



Management Level


Class 3 advanced heart faults

Class 4 advanced heart faults*

Step 3

Step 2B


A funny backdrop

Step 3



DN / Type 2 Diabetes

Step 2

Step 2


GvHD Summary

Stage 3 / BLA movement?

Japan is agreed

* Trial designed and managed by NIH

This is an incredible list of marks. After this time, money and talent that has been thrown at this technology, is there any reason to believe that this list will be # 39; creating income?

Overview of Three of MESO's clinical programs:

GvHD Summary: Avisol Capital Partners wrote a recent MESO remedy for GvHD and so I would not talk about the clinical results for this test or on; market while he was & # 39; cover in detail. This product (MSC-100-IV) has been marketed in Japan and MESO has recently completed a successful test in the US. MESO said that 69% have a total duration of 180 days and they are in a position; plan to meet FDA and want to record the continued BLA capability.

Ultimate heart failure: A continuous MESO Level III test (NCT02032004) is a dual, random, controlled, controlled and MPC (Rexlemestrocel-L) safety in 600 patients with continuous heart disease. The result of this test should be a great part of corporate valuation guidance over the next 12-18 months.

The statement is about 500,000 new cases of heart failure. Fifty percent of cases on the recital will die within 5 years of confirmation; 75% will die within 8 years. The patient's healthcare system costs around $ 17.8 billion annually on these patients. More than 1.3 million patients are classified as a Class III coronary heart failure (CHF) using New York Heart (NYHA) Association, 250,000 CHF Class IV CHF, and 50,000 patients with CHF level-level . These patients get an increase in serious death and death.

Stage III of a blind page (1: 1) test is carried out in many research sites in the USA and Canada. Adult infantry with a state-of-the-art middle-class society New Heart Heart Class II / III Association with continuous heart failure with sistolic cluster dysfunction potentially potentially a partner. The main level of its program efficiency is; in comparison with variable variables that are associated with non-fatal heart failure (HF-MACE) in a medium-class CHF patient receiving the second MPC-150-IM catheter intake, into the heartbeat that is left by a skeleton rule. MESSO recently related relations with China to Tasly for this sign.

What's in HF-MACE? MACE stands for bad friendship events that include friendship deaths, non-mortal microbial casualties, non-routine declining or regional outlook intervention. HF-MACE MACE is not the need for a hospital due to heart failure.

There is a scientific controversy associated with this program. SA Alpha Exposure has written 3 negative articles about MESO, and was last published in September 2016. The article that MESO of data mining expresses the result of this level 2 test. This is not an unreasonable complaint as Stephen Farris and April Stempien-Otero published at the Washington Medical University manuscript in a magazine Circulation research which examined the results of the Phase II study of MESO. This manuscript was published in the same journal that published the results of the Stage II analysis of MESO. This is the same as a formal science pissing game. The fines from their fine include:

  • although comparison between HF-MACE between each group and the control face with exams was corrected, correction was not used to use a complex comparison. … so it should be careful to use this data as a major justification of efficacy. In addition, as explained specifically in the study, the completion of the HF-MACE end was created in a post-hoc mannerSouth Westerly
  • … the team did not get any invasion control over a vehicle, PBS, or MSC cells. … It is possible to decide that cells would enter into the weakness that is missing; promoting myocardium and as a consequence of biological impact that may be appropriate. … But, apparently because safety was in place, the FDA has agreed to Stage III review to decide what to do; The impact of the translucent injection is.

Their objections are correct and valid but they certify that the FDA is in a position; Let this science continue. For investors, there is no question that there is enough scientific measure in the MESO Stage II inspection instead which has a clinical capacity and a way of showing the same? I was informed that a group of engineers would be able to. provide the same data set and that the objectives would be considered independent if they did not participate in the MESO action course.

The test did not meet the stated efficiencies in the patient population surveyed using the registration used (n = 60: three data groups of 20 with 5 in each group used as a control). However, the test gave a satisfactory level for the next review to determine effectiveness; It also gave an overview of the patient's population that could benefit from cure. The development of the advanced development is the ultimate skill in the shortest time to use the smallest facilities; Scientists who sing to tune are different. Applied science and development requires at least some skill sets.

The authors of the II level investigation included a section they were named Post-test HF-MACE in which they said there was a degree of level of events time-to-first HF-MACE 33% for their disciplinary body, 20% for 25 million MPC, 40% for 75 million MPC, and 0% for its & # 39; 150 million MPC group. … the 150 million MPC groups were quite different for HFMACE from all other organizations. I was not missed by the fact that this study was done post hoc.

Follow company on going to & # 39; Patients continued in this lawsuit and gave an update in Jan 2018. The graphs are below; Showing 36 months for patients in the 150M bilingual group against placebo. The graph on the left shows% of all patients in each group free of charge from HF-MACE; the graph on the right is a sub-populated survey that's a; Significantly influenced and entered into the test with sistolic measurement on the left (LVESV)> 100ml at bottom line. There is a division between the medicine and placebo group; increase over time. That is, the statistical power of a positive positive position has risen over time. Her first review was used in & # 39; Increasing this test should be determined in my mind; however, the company had a penalty in the scientific court of public opinion.

Attracted from JPMorgan 36th Conf Healthcare. – slide 29

Main post-hoc study: 11 exercises with LVESV> 100ml in the organism were 11 HF-MACE events totally or recycled over 36 months compared to 0 in a & # 39; group managed 150M MPC. Probably defined by its company's capability criteria (LVESV> 100ml) related to clinical benefits; This may be important to patients and be the basis for valid patent applications. It would be impossible to identify its & # 39; ability for relationship to cut and respond LVESV 100ml at the beginning of the test; in fact, this relationship is not yet proven. However, MESO's judgment in court design is a reasonable and apparent indication.

It was also said that the company has modified the terminals for this test. The reader can make their own decision about this application by reviewing the following link: changes. The connection offers side-by-comparison of the first protocol and the current protocol. Be aware that this was a Teva (NYSE: TEVA) that was the responsibility of this lawsuit and that's why you see their name.

In April 2017, MESO published that the MPC-150-IM did not have a safety lawyer at the Independent Data Research Committee of the test and it formally recommended that the test should be held as expected. His company also stated that they were " meet the interim analysis to determine the efficiency validity in the & # 39; first 270 patients.

A company has still been convicted of safety data and the effectiveness of the test as well as the numerical results of this interim inspection and so the ICMC does not; Understand how robust the effectiveness data is. If the data had been very strong the ICMC could stop its lawsuit to allow all patients to cure but the results were not very useful for this activity.

Ventricular Clì Aid Tools: Stage 2b analysis and design by NIH was selected as a late showcase at the 2018 American Science Society's Science Sessions in Chicago early (November 11). This study was funded by NIH and the Canadian Health Research Institutions, and led by the Cardiothoracic Ceremic Tests Network that was supported by NIH.

MESO MPC-150-IM was used in an earlier safety test (EU data for GI erosion shown below) for the treatment of a heart failure patient that was sent organized by an audio visual support device (LVAD). If proofing results are independently validated (a) they can influence the thinking of academic craftsmen in the role of cell cells in this patient population and (b) It's better to succeed in other features.

MPC-150-IM is used for a terminal patient with LVADs known as RMAT. 50,000 new patients have been diagnosed with heart failure in the US every year and their 1 year mortality is more than 50%. A small proportion of this population is worthy of LVADs but there is also a major disease and dies 1 to 20-30%. The US market for MPC-150-IM is estimated to have an endowment core heart vacuum with LVADs at $ 500M without any other product development. The I testification showed that it reduced hospitalized and embedded indigenous heartwork as considered by moving from LVAD. Success in this test can affect how institutional investors can see the stock.

The out-hoc survey of the 30-patient safety test reduced hospitalized and enhanced indigenous heart failure, for example. At 30 days 30% of the body had a dead control against 0% of a medical group. GI bleeding and post-surgical post-linked hospital with an EU are important to this surgery. Their descendants below show their & # 39; The group that was handled (red) was later displayed for its 1st hospital due to GI cleaning. The FDA has given MESO guidance that there is a reduction in major GI collisions and hospitals related to clinical significance (a high non-accomplished need) which could be a key regulatory component.

Taken from MESO 6K recorded on Nov 07 2018

Update on Ventricular Support Support Tools: MESO declared the results for the LVAD heart respiration found on the 11thth November. Demonstration to meet the & # 39; primary pre-specific primary point. Key issues are listed below

  • This test was funded by NHLBI and NINDS of the NIH and Canadian Institutes for Health Research and the design and implementation of examinations was carried out outside MESO;
  • The surgeons designed by the test decided to determine the main point of "temporary delivery from LVAD supporters";
  • FDA named RMAT for this patient number based on the data in the pilot survey and indicated the following in two separate FDA meetings held in December 2017 (agreed by RMAT) and June 2018 (type B mtg):
    • Unconnected Gogging and / or epistaxis which is clinically responsible issues for patients with LVADs and represents an unmet need;
    • The LVAD's temporary blockage is the bio-signal and is not the result of a clinical meaning.
  • The degree of GI / Epistaxis bleeding was different (p <0.001) between control (15.9 incidents / 100 months of patient; n = 53) and a medicine group (3.8 incidents / 100 months of patient; n = 106).
  • There was no difference in dying at 6 or 12 months between the two groups.
  • As well as being absent non-celestial there were no differences in SAEs between the two groups.

Taken from the MESO corporate display: November 13, 2018 with the title Delivering LVAD Webcast Results

The company expects to meet the FDA and ask if the data from this Stage II test is sufficient to apply BLA for MPC-150-IM in a patient with a terminal failure at a final stage forwarded by a left-handed left-handed device for reduction in GG vaccinations episodes and associated hospital.

Rescue Reusato Arthritis (UK): MESO is targeting a number of UK patients who do not have a disability; Respond to TNF barriers or can not obstruct TNF restrictions that are about 1/3 of the 3 million patients in the US. The UK has been well-researched by the condition of illness and FDA uses American College of Rheumatology Criteria (ACR-N) to assess remedies. Drugs need to show at least 20% (improvement) in ACR-N for a patient to have a clerical clinical development, such as a cure success. For example, the Gilead / Galapagos drug has recently reported that 80% of patients showed at least 20% in signs and UK marks at week 16 against 33% for placebo.

The two-test results in a UK MESO test for patients who fail at least 1 biological agent are shown below. The 1-dimensional red line of 1M cells has a 2M cellular 2-inch purple line. Placebo is black. At 2M cells there is a steady improvement of about 48% seen in week 12 which is maintained for week 39. The average values ​​for 1M medicine group showing steady progress on week 39, which is very interesting and praises some of the things that are going on. The Post Office The two medical groups are statistically important against the venue. This is clearly a huge market opportunity for its company.

Taken from the Corporate Website Broadcasting Exhibition February 16, 2017

Intellectual Property (IP): Is there a moss?

The company reports that they have about 800 patents and patent applications across all jurisdictions. Google Patent Search for Patterns given to Mesoblast offers 76 specific records. Thirty of these recordings were thirteen before the creation of the Mesoblast in 2004 as six were recorded as early as 1990. Much of MESO's IP was received from Osiris Therapeutics (OSIR) in & # 39; Getting their step-by-step industry? including the product of GVHD MESO. OSIR portfolio portfolio was 110 entries, including 48 in the United States, 21 in Europe and 9 in Japan. It was early entry; in Osiris in game cells and had been approved by JCR Pharmaceuticals in August 2003; JCR Pharmaceuticals is currently marketing GVHD MESO products in Japan.

In 2003, Osiris collaborated with Boston Scientific for the clinical use of MSCs in bone marrow replacement as a support for poor sheep cases, heart repairs after a mycircardic chemistry, heart failure and respiratory removal after meniscctomy. And I can understand how a museum could provide background information in ceilidh cures to the ability of a company to protect materials based on ideas that were investigated clinically for more than 20 years . Analysis of this large petent portal is further than the scope of this article and the ability / knowledge of this author; However, some of the most recent may be useful to review to spread IP registration.

The application titles that start the year after receipt include the following 7 bids; Starting: (1) Enhanced cellulose coordination, (2) Illicit medicine, (3) Improving heart failure failure, (4) Method for heart failure treatment, (5) Stone culture mode for mesenchymal cell cells, (6) Energy measurement and (7) Methods and mechanisms for separating cells from micro-transport. These are the patent large patch types such as, even new active agents with unique chemical structures. However, if MESO recognizes a new unintentional element (creation, method of use, …) which provides a resource, they have a reasonable opportunity to succeed in obtaining an additional IP.

Subsequent 3 above (US-2017333482-A1), for example, a & # 39; cover; The biggest target market that MESO continues. In this application, the engineers say they have identified the number of subjects with a heart disease that responds to cure with properly prepared MSCs, even patients with a lower left-end volume (LVESV) of 75 mL is not larger. And what cells are right? Application number 6 (CA-2984987-A1) may give an opinion: Method for proofing the potential of mesenchymal or cell-cell fill warnings; including … to determine the level of TGF.beta.1 that was distributed to the media into the medium of culture, containing a measure of at least 2800 pg per 1 million cells of TGF.beta-1 a & # 39; showing biological activity or medicinal efficacyPost-Appeals These applications are quite different from writing business claims that the traditional medicine industry is dependent on. Are these applications patental? The answer needs reading of the arts before and analyzed but with so much failure in the field it is not clear to me why we define the correct population and that the right features cell in a US patent where these elements are not identified in the art advance. Indeed, an argument can make a powerful visibility if the conditions that MESO have previously faced the art. MESO's engineers may have explanations and definitions of the signal / method that explains its resource provision and this can provide a clear basis for patent.

Undoubtedly, I looked at an application 2 above in relation to defenses. The first application in this application is: Method to expand and / or to; influencing anti-inflammatory cells in a subject … co-writing that includes untranslated genetic obesity cells with genetic unchanged cells can say angiopoietin-1 (Ang1) in a measure of at least 0.1 μg / 10E6 cells. If the arts are not in advance of identifying this relationship and that it is In line with resources, then they should be able to make an application.

It would be interesting to listen to two patent candidates to discuss how well they are; Applications in these patterns and approximate how much & # 39; s they reduce the likelihood of other companies that are experiencing; invest in competitors based on MSC. Fortunately, we can monitor the results of such a debate in the MESO-TIG license agreement that was completed in December 2017. A recent Canadian company of cellular companies in TiGenix is ​​a Canadian company. Thog TiGenix CX601 (Alofisel) airson a bhith a &#39;làimhseachadh fístilean iom-fhillte ann an galar Crohn. Is e Alofisel a &#39;chiad leigheas ceum-cealla gus beachdan dheimhinneach fhaighinn bho CHMP. Chaidh a &#39;chùmhnant ceadachd eadar MESO agus TIG fhoillseachadh ann am brath naidheachd co-roinnte a thug a-mach na buadhan a chaidh a thoirt do gach companaidh. Fhuair MESO $ 24M ann am pàigheadh ​​agus dìlseachdan aon-dhidseatach air reic lìn Alofisel. Fhuair TIG ruigsinneachd shònraichte do chuid de na h-àiteachan [MESO] pàtranan gus taic a thoirt do mhalairteachadh cruinneil de thionndaidh ceall-cheum mesenchymal a chaidh a thoirt a-steach le adipose Cx601 airson leigheas ionadail fistulaeS an Iar- Tha e coltach rium gur e an aon cho-dhùnadh reusanta a tharraing gu bheil luach aig IP MESO oir cha do rinn MESO tasgadh dìreach ann an leasachadh Alofisel.

Chan eil coltas ann gum bi companaidh sam bith a &#39;toirt dùbhlan do na stuthan MESO a tha dùbhlanach gus am bi iad soirbheachail ann an co-dhiù aon chomharra. Ma thachras sin, chan eil mi a &#39;faicinn carson a bhiodh pharma mòr a&#39; tasgadh ann am prògram leasachaidh gus stuthan stèidhichte air MSC a leasachadh. Bhiodh an dealbhadh airson tasgadh den leithid a &#39;gabhail a-steach saorsa a bhith a&#39; cleachdadh beachd bho luchd-cùraim. Dh&#39;fheumadh an luchd-tagraidh beachd a thoirt seachad nach biodh toradh sam bith mar sin a &#39;briseadh aon de na tagraidhean anns na pàtranan a th&#39; aig MESO. Carson a ghluaiseas an cunnart seo; is e an cluiche as sàbhailte dìreach an companaidh a cheannach.

Cosgaisean Toraidh Dh&#39;fhaodadh a bhith na Dhiùltach:

Na stuthan a dh&#39;fhaodadh a bhith aig MESO a bhith a &#39;faighinn buannachdan thairis air stuthan cealla-chealla fèin-riaghlaidh; ge-tà, cha bhi stuthan MESO a &#39;dol a-steach gu saor. Tha MLCan iomallach bho smior cnàimh air a thoirt bho inbhich fallain; tha gach neach-tabhartais co-ionann ri aon bhàr maighstir cealla. An uairsin thèid bancaichean mòra cealla a leudachadh gus an teirpeach stèidhichte air cealla a dhèanamh; faodaidh gach leudachadh mìltean de dhòighean a thoirt a rèir an toraidh. Tha an toradh an uairsin air a chrùthadh, air a phacaigeadh agus air a ghleidheadh. Thèid bathar a chur gu luchd-sgaoilidh no dìreach gu ionadan leigheis ann an luchd-cròidh far a bheil e a &#39;fuireach anns a&#39; chlaidheamh gus an tèid a dhìteadh airson cleachdadh euslainteach.


Bha airgead pro-forma air 30 Ògmhios 2018, US $ 116.8 millean, a &#39;gabhail a-steach airgead co-chothromachaidh de $ 37.8 millean; $ 40.0 millean bho Tasly Pharmaceutical Group (tasgadh com-pàirteachais ro-innleachdail le Tasly) agus US $ 39.0 millean bho NewQuest Capital Management. Is e roinn seilbh ro-innleachdail de Quintiles a th &#39;ann an NovaQuest, aig a bheil eòlas domhainn anns gach raon de leasachadh clionaigeach oir is iad sin na CRO as motha san t-saoghal anns an t-saoghal airson còrr air deich bliadhna; dh&#39;iarr an t-ùghdar air Quintiles mu 15 bliadhna air ais airson grunn ghnìomhan eadar-dhealaichte agus faodaidh e dearbhadh a dhèanamh air an ìre àrd de eòlas agus de eòlas a dh&#39;fhaodas iad a tharraing anns gach raon de leasachadh clionaigeach. San Iuchar, chaidh MESO gu goireas maoineachaidh $ 50.0 millean le NovaQuest a bha gu bhith air a chleachdadh airson leasachadh leantainneach agus malairteachadh remestemcel-L (MSC-100-IV) airson clann le galar gèidhe a tha an-aghaidh teasairginn stroideach (AGVHD). Nuair a dhùin e, tharraing Mesoblast $ 30.0 millean agus thug e $ 10.0 millean ann an earrannan àbhaisteach. Mus tèid a choileanadh sa Iuchar 2026, chan eil an iasaid ach ri phàigheadh ​​air ais bho reic lìn de remestemcel-L. Thèid pàigheadh ​​riadh a chur dheth gus an tèid a &#39;chiad reic à-ghnìomhach de remestemcel-L a dhèanamh às an Àisia. Tha an ionmhas seo fo smachd an àrd chreideasair a tha Hercules. Tha an maoin Hercules $ 75.0 millean na ghoireas creideis ceithir-bliadhna neo-fheumail; chaidh a &#39;chiad earrann de $ 35.0 millean a tharraing air dùnadh. Faodar $ 15.0 millean a bharrachd a tharraing air no roimhe Q4 CY2018, agus faodar $ 25.0 millean eile a tharraing air no roimhe Q3 CY2019, oir thèid clachan mìle a choileanadh.

Tha airgead mar thoradh air sgàth gu bheil MESO air airgead mòr a chosg gu h-eachdraidheil; is e sin, tha iad air a bhith na chompanaidh leasachaidh airson còrr is 15 bliadhna gun teachd-a-steach ciallach. Bha an ìre losgaidh ràitheil mu dheireadh timcheall air $ 20 millean. Leis gu bheil MESO air a liostadh anns na SA an-uiridh chan eil a h-uile bliadhna aca rim faotainn airson ath-sgrùdadh ach ma dh &#39;fheumadh iad WAG a ghabhail aig na cosgaisean iomlan aca, chuirinn iad aig $ 1.5 billean; Chan eil an Dr Itescu, an fhrith-uidheamachd air cùlaibh a &#39;chompanaidh, gainnead comais, lèirsinn agus dràibheadh; tha a chomas a bhith a &#39;cumail a&#39; maoineachadh a &#39;chompanaidh teicneolais lèirsinneach seo airson 15 bliadhna an aghaidh droch dhuilgheadas cho mòr, ma tha e nas motha, na choileanadh na teicneòlasan.

Tha MESO an dùil a bhith a &#39;ceadachadh an cuid làimhseachadh fàilligeadh cridhe (MPC-150-IM) agus leigheas RA (MPC-300-IV) agus an Dr Itescu air a ràdh gu bheil a&#39; chompanaidh ann an còmhraidhean le luchd-ceadachd a dh&#39;fhaodadh a bhith ann. Chan eil mi a &#39;creidsinn gum bi teachd-a-steach bho remestemcel-L (MSC-100-IV) airson clann le Galair Graft versus Host Hostal (aGVHD) aodach teasairginn steroid (aGVHD) a&#39; dèanamh fiachan nan cosgaisean ann an 2018 no 2019. Tha e gu tur comasach gum bi a &#39;chompanaidh a&#39; stiùireadh co-dhiù aon ionmhas a bharrachd ann an 2019.

Sealladh an ùghdair:

Tha luachadh a &#39;MESO san àm ri teachd an crochadh gu mòr air toradh an deuchainn Dream HF-1 (NCT02032004) agus an comas a bhith a&#39; ceadachadh comharradh falamh cridhe sna SA agus san Roinn Eòrpa (cead Tasly ann an Sìona). Dh&#39;fhoillsich Philip Taylor pìos ann am Bith-eòlas Biotasach air a &#39;bhuil a tha a&#39; sealltainn cho cudromach &#39;sa tha an deuchainn seo airson MESO. Mar thoradh air na toraidhean deuchainnean seo, chaidh mi air ais agus ath-leughadh an làmh-sgrìobhainn le Stephen Farris agus an Giblean Stempien-Otero a rinn càineadh air na toraidhean foillsichte bho dheuchainn deuchainn MESO aig ìre II. A-rithist, lorg mi na co-dhùnaidhean fìor èifeachdach. Chan eil dàta sònraichte AE bho sgrùdadh mearachd cridhe phase II air fhoillseachadh le Perin air a ghabhail a-steach anns na toraidhean agus tha an cumhachd ro ìseal gus co-dhùnaidhean brìoghmhor a tharraing ann an tachartas sam bith.

Tha aon rud a &#39;coimhead soilleir, chan e iongnadh a th&#39; ann an Rexlemestrocel-L druga a leigeas leotha euslainteach cridhe dannsa draoidheil mar bhràithrean Nicholas; ge-tà, tha eadar-dhealachaidhean eadar-dhealaichte a &#39;toirt taic do na co-dhùnaidhean gu bheil lùghdachadh ann an lasadh siostamach gu ìre gu leòr gus buannachd chudromach a thoirt seachad do chinn-uidhe sònraichte a tha FDA air aontachadh. Tha Biocardia cuideachd air toradh adhartach fhoillseachadh le bhith a &#39;cleachdadh cheallan-ceal ann an sgrùdadh glè bheag ann an euslaintich a&#39; fàilligeadh cridhe ischemic. Cuideachd, tha Atersys air dàta dearbhach a shealltainn a &#39;cleachdadh ceallan-ceal ann an fulangaichean stròc.

Let&#39;s look at the consequences of this current trial as opposed to the headlines. MESO just hit a prespecified FDA approved endpoint (GI bleeds/hospitalizations) in a $500M-market that has no other treatment. That market can be accessed directly by the company without a partner since there are a limited number of locations that implant LVADs. This market alone could make MESO cash-flow positive. Will FDA consider approval based on the outcomes from this trial? This will be a very interesting example of how FDA interprets RMAT; I think the chances of an acceptance based on this trial would have been strongly improved if there had been a difference in the survival data.

If the agency does not allow MESO a limited label based on data from this trial, it will take another 18 months and likely further equity dilution to complete a second trial for this indication. In the interim, the initial read on Dream trial should have been announced.

Disclosure: I am/we are long MESO.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: This article does not constitute investment advice. The author strongly advises readers to consult their financial advisor prior to interested in biotechnology stocks as they are fraught with risk.

Source link