Safe­ty scare cleared and ear­ly da­ta in hand, Ul­tragenyx dish­es out $75M to buy rare dis­ease part­ner

It had its ups and downs, but Ul­tragenyx’s three-year-old part­ner­ship with an An­gel­man syn­drome-fo­cused biotech has cul­mi­nat­ed in a buy­out.

With $75 mil­lion up­front, the rare dis­ease spe­cial­ist is ex­er­cis­ing the op­tion it lined up back in 2019 to ac­quire GeneTx on the back of what it called en­cour­ag­ing in­ter­im da­ta from a Phase I/II dose es­ca­la­tion study show­ing “ear­ly clin­i­cal ac­tiv­i­ty across mul­ti­ple do­mains and mul­ti­ple mea­sure­ments with­out any ev­i­dence of drug-re­lat­ed safe­ty is­sues.” The com­pa­nies al­so got cleared to start test­ing high­er dos­es of GTX-102 in search of an op­ti­mal dos­ing reg­i­men — which Ul­tragenyx be­lieves could lead to even bet­ter ef­fects.

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