Ultragenyx Buys GeneTx to Take Angelman ASO the Distance
19 Jul 2022
OligonucleotideAcquisitionCollaborate
Ultragenyx is exercising its exclusive option to acquire partner GeneTx Biotherapeutics. The deal will see the Novato, California-based company surrender $75 million in upfront payments to GeneTx, along with future milestones and royalties.
The buyout comes on the heels of promising Phase I/II results from GTX-102, an antisense oligonucleotide candidate being developed to treat Angelman syndrome (AS), a rare neurogenetic disease. Interim data from a total of 11 children with a genetically confirmed diagnosis showed that GTX-102 induced meaningful clinical improvements in Angelman symptoms without causing side effects that were too severe.
In the Phase I/II trial, treatment efficacy was measured using the AS change and severity scales, both of which require clinicians to conduct assessments across five domains: sleep, behavior, communication and fine and gross motor skills. At 128 days after GTX-102 dosing, nine of the 11 evaluable children demonstrated marked improvements in at least three of five of these domains, and in their overall scores. At the time of the interim findings, none of the treated patients had developed serious adverse events.
“We are seeing encouraging early clinical activity across multiple domains and multiple measurements without any evidence of drug-related safety issues. The therapeutic effect should be further enhanced by starting at higher monthly loading doses that have already been shown to be tolerated,” Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx, said in a statement.
“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,” he added.
Affecting up to 1 in 12,000 people worldwide, Angelman syndrome is caused by a loss-of-function mutation on the allele of the UBE3A gene passed down from the mother. This peculiar maternal mode of disease inheritance is due to the natural silencing of the father’s UBE3A allele through an antisense transcript. Patients with AS develop more slowly, have communication, balance and motor issues and suffer from debilitating seizures. Often, patients will need continuous care for their entire lives and are unable to live independently. There are currently no approved treatments for the disease.
A FAST Development Friendship
After funding Dindot’s laboratory at the Texas A&M University for over five years, the Foundation for Angelman Syndrome Therapeutics (FAST) founded GeneTx in 2017 to focus solely on developing a treatment for Angelman. At the core of this spinoff firm was GTX-102, an ASO designed to target and bind the antisense transcript that deactivates the paternal UBE3A allele. This, in theory, would leave one functional copy of the UBE3A gene to ease AS disease activity.
Ultragenyx entered the picture in 2019, when it partnered with GeneTx to nurture GTX-102 and see it through to approval. Yesterday’s acquisition is the culmination not only of this partnership but also of FAST’s years-long quest to fulfill a huge unmet need for AS patients.
"What happened today is why FAST was founded, why we started GeneTx, and why we donate and fundraise: To discover and accelerate promising therapies for AS,” John Schlueter, chairperson of the board of FAST, said in a statement e-mailed to BioSpace. "Our approach, from funding frontline discoveries through preclinical studies to forging strategic partnerships in the biotechnology industry, aims to quickly advance translational research so that families living with AS move closer to potentially transformative treatments."
For more details,please visit the original website
The content of the article does not represent any opinions of Synapse and its affiliated companies. If there is any copyright infringement or error, please contact us, and we will deal with it within 24 hours.
Organizations
Indications
Targets
Drugs
Hot reports
Get started for free today!
Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.
Start your data trial now!
Synapse data is also accessible to external entities via APIs or data packages. Leverages most recent intelligence information, enabling fullest potential.