RARE - Ultragenyx to acquire GeneTx after promising data on Angelman syndrome treatment

• Ultragenyx Pharmaceutical ( NASDAQ: RARE ) on Monday said it had exercised its option to acquire GeneTx Biotherapeutics for a $75M upfront payment following promising interim data from a phase 1/2 study of the privately-held biotech company's investigational drug GTX-102.
• GeneTx is developing GTX-102 in partnership with RARE for the treatment of Angelman syndrome, which is a rare genetic disorder caused due to a loss of function of the maternally inherited UBE3A gene. Symptoms include delayed development, speech and balance problems and intellectual disability.
• Interim data from an early-to-mid stage trial of GTX-102 in 14 kids who have a genetically confirmed diagnosis of UBE3A deletion showed a meaningful improvement in clinical disease and an acceptable safety profile.
• "These data combined with the excellent science of Dr. Scott Dindot, the inventor of GTX-102, have given us the confidence to exercise our option to acquire GeneTx at an earlier timepoint so that we can take the lead on advancing GTX-102 into late-stage development for Angelman syndrome," said RARE CEO Emil Kakkis.
• Ultragenyx ( RARE ) stock earlier closed -5.8% at $61.02.

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