Aeglea BioTherapeutics Updates Phase I/II Trial Data

Aeglea Biotherapeutics, headquartered in Austin, Texas, announced new repeat dose data from its Phase I/II trial of AEB1102 (pegzilarginase) in patients with Arginase 1 Deficiency. It also reported fourth-quarter and year-end financial results.

Arginase 1 Deficiency is a rare inherited disorder caused by complete or partial lack of the enzyme arginase, one of the six enzymes involved in the breakdown and removal of nitrogen from the body. It allows for excessive accumulation of nitrogen in the form of ammonia in the blood. Children who are untreated may have seizures, spasticity, short stature and intellectual disability.

Pegzilarginase degrades arginine and is derived from human Arginase 1. Aeglea claims their version has a potential advantage over microbial enzymes because it is less likely to stimulate an immune response. The results were presented at the Society for Inherited Metabolic Disorders Annual Meeting in San Diego, California.

"We have seen encouraging results after repeated doses of pegzilarginase in patients with Arginase 1 Deficiency," said Anthony Quinn, Aeglea’s interim chief executive officer, in a statement. "Along with marked and sustained reductions in plasma arginine levels, we are seeing consistent reductions in the levels of other guanidine compounds beyond what appears to be achieved through current approaches to disease management. This is important given the potential contribution of guanidine compounds to the progressive hyperargininemia-related neurological abnormalities seen in this patient population. In addition to our developing understanding of the effects of lowering arginine levels in patients with Arginase 1 Deficiency, we have a good understanding of the safety profile in a larger population that includes patients from our cancer trials."

The compound is also being evaluated in a Phase I/II trial in uveal and cutaneous melanoma patients. Topline data from that study is expected in the fourth quarter of this year.

The company wrapped the year with, as of December 31, 2017, $50.3 million in cash. The company believes it has enough financial runway to make it through the third quarter of 2019.

The company had grant revenues of $1.5 million in the fourth quarter, compared to $1.2 million in the same quarter of the previous year. This is the result of a $19.8 million grant from the Cancer Prevention and Research Institute of Texas (CPRIT).

Research and development expenses were $5.8 million for the fourth quarter, compared to $4.7 million from the same quarter in 2016. The increase was mostly due to an increase of clinical activity for pegzilarginase. Yearly R&D expenses were $22.8 million for the year, compared to $18.1 million in 2016. The increase was related to expanded manufacturing, regulatory, research, and clinical development activities.

Net loss for the quarter was $6.5 million, compared to $5.5 million in the same period the previous year. Net loss for 2017 was $27.2 million, compared to $21.7 million in 2016.

The company’s losses are not unexpected in a development-stage company. Simply Wall Street wrote last week, "According to the industry analysts covering Aeglea, breakeven is near. They expect the company to post a final loss in 2021, before turning a profit of $8.33 million (US) in 2022. Aeglea is therefore projected to breakeven around four years from today. What rate will Aeglea have to grow year-on-year in order to breakeven on this date? Using a line of best fit, I calculated an average annual growth rate of 5.66 percent, which is fair. Should the business grow at a faster rate, it will become profitable at an earlier date than expected."

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