GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

SARASOTA, FL and NOVATO, CA, USA I January 15, 2020 I GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

"The FDA’s clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community,” said Paula Evans, Chief Executive Officer at GeneTx.  “Assessing an investigational therapy that aims to address the underlying cause of this devastating disorder has the potential to meaningfully alter the clinical course of patients’ lives.”

“Based on preclinical data, we believe that GTX-102 offers tremendous promise and may one day provide patients with a potentially transformative therapeutic option,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We look forward to continuing our partnership with GeneTx to address this devastating disorder for which there are currently no approved treatments.”

“The FDA cleared the IND which allows GTX-102 to enter the clinic,” said Scott Stromatt, MD, Chief Medical Officer, GeneTx.  “GeneTx’s Phase 1/2 study, named KIK-AS (Knockdown of UBE3A-antisense in Kids with AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020. The goal of this multiple dose, dose escalating, open-label study is to examine the safety, tolerability, and pharmacokinetics of GTX-102 in pediatric patients with Angelman syndrome. On February 15, 2020 we will open a website with more information on the KIK-AS study and provide a toll free number for parents and caregivers to obtain information on enrolling into the clinical study.”

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

About GTX-102

GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

About GeneTx Biotherapeutics

GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

About Ultragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

SOURCE: Ultragenyx Pharmaceutical

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