Trial data in hand, Ultragenyx buys Angelman partner GeneTx

July 19, 2022

Confident in the encouraging interim data from a small phase 1/2 study in pediatric patients, Ultragenyx has exercised its option to acquire development partner, GeneTx.

Interim results from the open-label, dose-escalating study of GTX-102 — an antisense oligonucleotide for the treatment of Angelman syndrome — demonstrated meaningful improvement in clinical disease and an acceptable safety profile in the 9 patients from the U.K./Canada arm and 2 patients from the U.S. arm. Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. Individuals with Angelman syndrome have developmental delay, communications issues, balance issues, motor impairment and debilitating seizures.

In light of the promising data, California-based Ultragenyx said it has closed on the acquisition GeneTx for an upfront payment of $75 million plus future milestone and royalty payments.

The partnership dates back to an Aug. 2019 development agreement. At the time, GTX-102 was in late preclinical development with an IND application expected to be filed in the first half of 2020. Ultragenyx made an upfront payment of $20 million for the exclusive option to acquire GeneTx, with the choice to extend the option period by paying an extension payment of $25 million.

Now, according to Ultragenyx's CEO, “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.”