Maxim Group Maintains Bullish View on Abeona Therapeutics (ABEO) After Safety Hurdle Cleared
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Maxim Group analyst Jason McCarthy reiterated a Buy rating and $14 price target on Abeona Therapeutics (NASDAQ: ABEO) after the company announced that the DSMB (Data Safety Monitoring Board) has reviewed the safety data from the first three children with Sanfilippo Syndrome (Type A) treated with ABO-102 gene therapy in the phase I/II study and cleared the study to continue dose escalation.
- The second cohort (N=6-9) will use a dose 2X (1X1013 vg/kg) that used in the first cohort. In preclinical studies, the activity of the replaced enzyme delivered via gene therapy was greater with increasing dose.
- In addition, recall that ABO-102 (and ABO-101) uses the same vector to deliver genes as AveXis' AVX-101 (developed in the same lab at Nationwide Children's Hospital), which has demonstrated positive data in infants with spinal muscular atrophy.
- However, AveXis is using a dose 10X higher than what Abeona is using for Cohort 2 in Sanfilippo and has shown a positive safety profile, thus there may be more room for dose escalation. ABO-102 has already demonstrated positive data at the lower dose.
- Conclusion. Progress in gene therapy trials, particularly for ultra-orphan indications like Sanfilippo Syndrome, is measured patient by patient and data point by data point. The question is: where is proof of concept? Is it N=1? Or N="not that many"? ABO-102 has demonstrated positive early data in three patients (safety and early efficacy), and the study should be enrolling patients in the higher dose cohort soon. More data are expected this fall.
Shares of Abeona Therapeutics closed at $6.01 yesterday.
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