On the cusp of PhI, Angelman-focused biotech scores potential buyout deal from Ultragenyx
Ultragenyx has found a new rare disease to devote itself to, roping in a young upstart as it begins a long trek to a new treatment for Angelman syndrome.
Their new partners at GeneTx is working toward an IND for GTX-102, an antisense oligonucleotide designed to inhibit the UBE3A-AS DNA strand and thereby turn on an allele in neurons that produces a key protein missing in patients with Angelman.
The collaboration — and potential acquisition — would pit Ultragenyx against Jeremy Levin’s team at Ovid, which has showcased some controversial Phase II data last year for its extrasynaptic GABAA receptor agonist. Angelman is also among the slate of neurological targets that Biogen and Ionis are tackling in an antisense deal.
Once the FDA accepts the drug for review, Ultragenyx will have 30 days to exercise an exclusive option to buy the company out — an option worth $20 million in the agreement. The duo has penciled in a potential extension, which would call for another $25 million payment.
“Angelman syndrome is a devastating neurological condition with no treatment options that represents a very significant unmet medical need,” said Ultragenyx CEO Emil Kakkis. “GTX-102 is a novel and promising potential therapy specific to the disease mechanism in these patients, and we are pleased that GeneTx chose to partner with us based on our track-record of rapid, innovative development in rare diseases.”
In addition, GeneTx has a unique connection to the patient community that made it appealing: The Foundation for Angelman Syndrome Therapeutics, the nonprofit group that launched GeneTx last February, is the largest patient organization for the rare neurodevelopmental disorder, Ultragenyx CBO Thomas Kassberg told BioCentury.
FAST is also the majority owner of GeneTx while Texas A&M University System owns a minority share. Paula Evans, FAST chairperson, GeneTx CEO and an Angelman mom, said it is fitting to shift from donations to a pact with an established industry player.
The two sides will collaborate on both the IND and management of the Phase I/II study. For now, GeneTx continues to fund all development activities and take charge of regulatory and scientific activities, while Ultragenyx offers strategic and clinical guidance.
Tile image: The Evans family, FAST