Gbola Amusa of Chardan Capital said although the AAV-based gene therapy (GT) of Uniqure NV (NASDAQ: QURE has better timing, Spark Therapeutics Inc (NASDAQ: ONCE)has better efficacy in hemophilia B patients.
Amusa gave the above comments after both companies presented their data at The American Society of Hematology (ASH) meeting on Saturday and Sunday.
Conference Reveals
“High-dose cohort data may not return uniQure to a competitive position in hemB GT,” Amusa wrote in a note.
UniQure may have a first-mover advantage in hemophilia B AAV GT space, but Amusa noted that the “combination of modest FIX production and a liver enzymes profile that must be further elucidated raise significant concerns for the long-term potential for AMT-060 in the hemophilia space.”
On the other hand, Spark Therapeutics showed better efficacy in hemophilia B gene therapy, but risks abound. The analyst continues to be impressed by the FIX activity of 12–65 percent achieved so far.
Analyst's Take
One key risk Amusa cited was the occurrence of two independent immune responses against the Spark100 capsid, which fundamentally undermines the hypothesis (lowering viral dose can evade immune activation against AAV GT).
“We thus wonder how such immune evaluation will be performed in pivotal trials or in the real world,” Amusa highlighted.
He posited whether the immune monitoring should be limited to the first 12 weeks, which is believed to be the key period for immune response against AAV. He also questioned whether future use requires rapid steroid treatment upon liver enzyme elevations to maintain AAV function.
Amusa has a Buy rating on uniQure and a Neutral rating on Spark Therapeutics.
At last check after a significant early morning drop, uniQure shares were up 3.42 percent at $5.87. Spark shares were up 1.52 percent.