Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome

Ultragenyx Pharmaceutical (NASDAQ:RARE) has announced it has partnered with GeneTx Biotherapeutics to develop GeneTx’s GTX-102 drug to treat Angelman syndrome.

As quoted in the press release:

GTX-102 is currently in late preclinical development with an investigational new drug (IND) application expected to be filed with the U.S. Food and Drug Administration (FDA) in the first half of 2020.

“Angelman syndrome is a devastating neurological condition with no treatment options that represents a very significant unmet medical need,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “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.”

“GeneTx was formed by the Foundation for Angelman Syndrome Therapeutics (FAST), the largest patient organization in the Angelman community. Donations to FAST, from family and friends, supported the preclinical research on this therapeutic strategy,” stated Paula Evans, Chief Executive Officer of GeneTx. “It is entirely fitting that GeneTx would partner with a company that not only has proven expertise in the development and commercialization of rare disease therapeutics, but truly puts the patient first in each and every step of the treatment process.”

Under the terms of the agreement, Ultragenyx will make an upfront payment of $20 million for an exclusive option to acquire GeneTx. This option may be exercised any time prior to 30 days following FDA acceptance of the IND for GTX-102. Ultragenyx may extend the option period by paying an option extension payment of $25 million. Ultragenyx may exercise this extended option any time until the earlier of 30 months from the first dosing of a patient in a planned Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.

Click here to read the full press release.