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Mesoblast Starts Rolling BLA Submission for Remestemcel-L

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Mesoblast Limited (MESO - Free Report) announced that it has initiated the rolling submission of a biologics license application (BLA) to the FDA for its lead product candidate, remestemcel-L. The investigational therapy is being developed for treating children with steroid-refractory acute graft as compared to host disease (aGVHD), a life-threatening complication of an allogeneic bone marrow transplant.

Remestemcel-L is being investigated in a phase III study, which evaluated the candidate in 55 children, majority of whom had severe forms of aGVHD. Data from the study showed that treatment with remestemcel-L can lead to 69% chances of six-month survival for the subjects. The program met the primary endpoint of increased overall response compared with the protocol-defined historical control rate of 45% at day 28.

The rolling submission came in after the FDA’s Fast Track designation, which was granted to remestemcel-L for treating aGVHD pertaining to the given patient population. Upon completion and acceptance of this BLA filling, Mesoblast plans to request for a priority review to the FDA.

Shares of Mesoblast have rallied 23.3% so far this year, outperforming the industry’s increase of 0.8%.

 

Per the press release, approximately 30,000 allogeneic bone marrow transplants are conducted across the world annually, primarily for patients diagnosed with blood cancer. Of this number, 50% subjects develop aGVHD. If approved, Mesoblast plans to make this therapy available at the earliest to address this life-threatening disease for the given patient population.

We would like remind investors that a few companies are already working to introduce medicines for treating this devastating disease.  Notably, Incyte Corp’s (INCY - Free Report) lead drug, Jakafi (ruxolitinib), is approved for treating the rare blood cancers of MF and PV.

Last week, the FDA lent a nod to Incyte’s supplemental new drug application (sNDA) for Jakafi with regard to the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients aged 12 years or above, who had showed an inadequate response to corticosteroids. This is the third indication, for which the drug has been given a go-ahead in the United States.

According to Incyte, this is the first and the only FDA-approved treatment for the aforementioned indication.      

Zacks Rank & Stocks to Consider

Mesoblast currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Acorda Therapeutics, Inc. (ACOR - Free Report) and Repligen Corp. (RGEN - Free Report) , both sporting a Zacks Rank #1 (Strong Buy).You can see the complete list of today’s Zacks #1 Rank stocks here.

Acorda’s loss per share estimates have been narrowed 6.5% for 2019 and 6.9% for 2020 over the past 60 days.

Repligen’s earnings estimates have been revised 13.3% upward for 2019 and 12% for 2020 over the past 60 days. The stock has surged 30.6% year to date.

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