- Mesoblast (MSB) is gearing up for what could be a very successful two months as it gets closer in approving lead drug
- The company is expecting a meeting with the U.S. Food and Drug Administration (FDA) about its drug, remestemcel-L, for paediatric patients with steroid-refractory acute graft versus host disease
- This meeting comes after the FDA proposed Mesoblast to hold another trial on the effectiveness of remestemcel-L on the disease
- For its COVID-19 treatment, Mesoblast is expecting to receive the first 135 patients data from its phase three trial in November
- It will send this data to the independent Data Safety Monitoring Board, who will decide to continue the trial or close early
- Mesoblast has started its trial for Crohn’s Disease with 48 patients at the Cleveland Clinic to undergo the trial
- The company is expected to receive results from its chronic heart failure and lower back pain trials by the end of this year
- Mesoblast is up 1.29 on the market today and is trading for $3.14 per share
Mesoblast (MSB) is gearing up for what could be a very successful two months as it gets closer in approving its lead drug.
The business has given an update on its current trials and has released its September quarter results.
Children with steroid-refractory acute graft versus host disease
In August, the company received the green tick from the Oncologic Drugs Advisory Committee (ODAC) to support the efficacy of remestemcel-L in paediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).
Despite this, in September, the U.S. Food and Drug Administration (FDA) recommended that Mesoblast conduct at least one additional randomised, controlled study in adults and children to provide further evidence of the effectiveness of remestemcel-L for ST-aGVHD.
The health-care stock believes that its treatment meets the criteria for the approval as there is currently no approved treatments for this life-threatening condition.
Mesoblast has requested a meeting with the FDA about the treatment, which is expected to occur in November.
The business has been testing its remestemcel-L for COVID-19 patients with moderate to severe acute respiratory distress syndrome (ARDS).
COVID-19 cases have surged in the United States and globally, deaths from the disease have increased with ARDS in ventilator dependent patients.
“It is now evident that in both adults and children COVID-19 causes severe inflammation of other organ systems in addition to the lungs, including the heart, brain and kidneys,” the company posted on Thursday.
“The immunomodulatory mechanism of action of remestemcel-L may be beneficial in the treatment of ARDS as well as in involvement of other organ systems,” the company announced.
Under the companies trial, nine out of 12 COVID-19 ARDS patients who received two doses of remestemcel-L at New York hospital, Mt Sinai, were successfully discharged in around 10 days.
On top of this, two COVID-19 infected children with multisystem inflammatory syndrome who received remestemcel-L for severe heart failure were fully recovered and discharged within 30 hours of the second dose.
To confirm this data, Mesoblast is undertaking a phase three trial for 300 ventilator-dependent adults with moderate or severe COVID-19 ARDS. Trial enrolment has surpassed 50 per cent of the total 300 patients across more than 20 hospitals.
The first 135 patients will complete 30 days of follow up during October after which the independent Data Safety Monitoring Board (DSMB) will analyse the data.
DSMB is expected to inform Mesoblast in early November on where the trial should proceed or stop early.
Crohn’s disease is a type of inflammatory bowel disease (IBD) and it is estimated that more than three million people (1.3 per cent) in the United Staes have IBD.
More than 33,000 new cases of Crohn’s disease and 38,000 new cases of ulcerative colitis are diagnosed every year. Up to 80 per cent of patients with Crohn need surgical treatment, which can have a devastating impact on the quality of life.
Mesoblast has commenced a study to confirm the potential for remestemcel-L to induce luminal healing and early remission in a wider spectrum of diseases that are associated with severe inflammation of the gut.
The investigator study is taking place at Cleveland Clinic for 48 people who have medically refractory Crohn’s disease and ulcerative colitis.
Chronic heart failure
In the United States, more than 6.5 million people have chronic heart failure, with more than 1.3 million patients in advance stages of the disease.
The company is testing its product Revascor to reduce or reverse the severe inflammatory process in the damaged heart of these patients, thereby preventing or delaying mortality.
Phase three trial results are expected to be released in the fourth quarter of 2020.
Chronic Lower Back Pain
There is currently a need for a sale, efficacious and durable treatment in patients with chronic low back pain due to severely inflamed degenerative disc disease.
Degenerative disc disease affects 3.2 million patients in the United States and around 4 million in the EU5 (France, Germany, Italy, Spain, U.K.).
Results from the phase three trial for chronic lower back pain is expected by the end of the year. Additionally, Mesoblast and its partner Grünenthal GmbH are collaborating for a confirmatory phase three trial in Europe.
First quarter results
The ASX-200-lister has finished the quarter with US$108.1 million (around A$153 million) cash on hand. Over the next 12 months, Mesoblast will have access to an additional US$67.5 million (nearly A$96 million) through existing financing facilities and strategic partnerships.
Research and development costs were US$9.9 million (around A$14 million), which is an increase from last year’s US$7.8 million (approx. A$11 million). This increase was due to recruitment activities for the phase three trial for remestemcel-L in COVID-19 patients.
Product and manufacturing costs were also up from last year at US$5.2 million (around A$7.3 million), compared to US$3.5 million (nearly A$5 million) due to the increased capacity requirements for the COVID-19 trial.
“We believe the immunomodulatory properties of remestemcel-L position this potential therapy at the forefront of treatment for severe and life threatening inflammatory conditions, including COVID-19 acute respiratory distress syndrome and steroid-refractory acute graft versus host disease,” Mesoblast CEO Dr Silviu Itescu said.
“We are pursuing an accelerated approval pathway for remestemcel-L in the treatment of children with SR-aGVHD, and a parallel approval pathway for COVID-19 ARDS if the randomized controlled phase three trial is positive,” he added.
Mesoblast is up 1.29 on the market today and is trading for $3.14 per share at 1:44 pm AEDT.