- Rare disease drugmaker Ultragenyx Pharmaceutical has acquired an experimental gene therapy for Sanfilippo syndrome Type A, licensing rights to the treatment from struggling developer Abeona Therapeutics in an agreement announced Tuesday.
- Under the deal, Abeona will receive royalties of up to 10% on net sales of the gene therapy, which has progressed through Phase 1/2 testing that's meant to support an approval application to the Food and Drug Administration. While Abeona won't receive any upfront payment, it could get up to $30 million from Ultragenyx if certain sales milestones are achieved.
- Abeona said interim results from its study showed treatment with its gene therapy helped preserve cognitive development, reduce liver volume and lower levels of large sugar molecules that accumulate in the brains and organs of people with Sanfilippo syndrome. Updated data was presented Tuesday at the American Society of Gene and Cell Therapy's annual meeting.
Ultragenyx, which sells three medicines for rare diseases, has built a pipeline of experimental gene therapies, four of which are in clinical testing. But the company believes Abeona's could become its first to reach market in the U.S.
"The Sanfilippo community has been waiting too long for a first treatment and we believe we can help accelerate this program," said Emil Kakkis, Ultragenyx's CEO, in a Tuesday statement on the deal.
Last year, Abeona said it had reached agreement with the FDA on the primary goal of its principal study for the treatment, called ABO-102 or UX111. That bodes well for a clear path to the regulator with data from that trial, although Ultragenyx did not specify any timeline for when it hopes to approach the agency with an approval application.
Ultragenyx is acquiring Abeona's treatment for relatively cheap, paying nothing upfront and promising limited royalties and milestone payments on any commercial sales that follow.
The deal follows a recent reprioritization of research and spending at Abeona, which in March discontinued a similar gene therapy and said it was searching for a partner on ABO-102. Shares in the biotech company have fallen nearly 90% over the past year and now trade below $1, risking a delisting from the Nasdaq stock exchange. (Abeona plans to maintain its position on the market through a reverse stock split and has called a shareholder meeting to approve the plan next month.)
"Offloading the MPS programs, in addition to ceasing the build-out of additional AAV facilities, should significantly reduce our cash burn and allow Abeona to fund operations over the next 12 months," said Abeona CEO Vish Seshadri on a conference call Tuesday.
Sanfilippo syndrome describes a cluster of conditions caused by mutations in four genes that produce enzymes involved in breaking down sugar molecules called glycosaminoglycans, or mucopolysaccharides (MPS). Without sufficient quantities of the right enzymes, people with Sanfilippo syndrome are unable to clear the sugar molecules, which accumulate within cells, particularly the lysosome.
The buildup leads to progressive cell damage, neurodevelopmental and physical decline. People with Sanfilippo syndrome Type A have a median life expectancy of 15 years.
ABO-102 is designed to remedy the toxic accumulation of glycosaminoglycans by delivering into the central nervous system and other organs a functional copy of the relevant gene, enabling the missing enzyme to be produced.