Abeona Therapeutics Inc(NASDAQ: ABEO) stock surged over 11.1% this morning as analysts from RBC Capital started coverage on the stock with an “Outperform” rating. The stock delivered an outstanding rally this year and generated over 299.5% returns this year to date. The analysts issued a price target of $23, which is a strong upside to the current 19.40 price (As of 1:14PM EDT on October 10th, 2017; Source: Google finance).
The group recently reported a positive one-year data from Cohort 1 of the ongoing ABO-102 Phase 1/2 trial for Sanfilippo syndrome Type A (MPS IIIA). The results show major reductions in biopotency and biophysical measures, preservation of deep brain architecture, and stabilization across multiple neurocognitive assessments reported in comparison to untreated control subjects. The group’s ongoing gene therapy trial for ABO-102 (AAV-SGSH) is using a single intravenous injection for treating subjects with Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.
The results support the group’s intravenous delivery approach for patients with a lysosomal storage disease. The group is aiming to speeden their enrollment with their active global sites in Spain and Australia while reporting further clinical data from the ABO-102 global MPS IIIA trial later this year.
As Per the design of the clinical trial, subjects got a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Results show that ABO-102 reached target tissues throughout the body, including in the central nervous system.
Meanwhile, the group made a strategic manufacturing commitments with Brammer Bio for commercial translation. The group appointed Phillip B. Maples, as Vice President of Therapeutic Development and Quality Management
The group has a Cash position of $58.3 million as of June, 2017 while Net cash used in operating activities was $10.8 million in the six months ended June 30, 2017 as compared to $5.6 million in the same period in 2016. Their Revenues improved to $217 thousand for the second quarter of 2017, from $214 thousand in the second quarter of 2016.