Everything's Bigger in Texas: Aeglea Quickly Growing into Leading Austin Biotech
September 13, 2016
By Mark Terry, BioSpace.com Breaking News Staff
In three short years, Austin, Texas-based Aeglea BioTherapeutics has gone from startup to 30 employees, with three ongoing clinical trials and a fourth expected to begin in 2017.
aeglea biotherapeutics rings the @Nasdaq Opening Bell! pic.twitter.com/zsb2hSYYGH
— Nasdaq (@NASDAQ) July 19, 2016
David Lowe, co-founder, president and chief executive officer of Aeglea, sat down with BioSpace to discuss the company’s progress and future in an exclusive interview.
Aeglea, founded December 2013, was based on the work of University of Texas at Austin professor George Georgiou. “We are the leader in developing and creating engineered human enzymes,” Lowe told BioSpace. “Our products are designed to degrade or decrease specific amino acids in patients’ blood as replacement therapies for genetic rare diseases, and as a means of targeting abnormal tumor metabolism for cancer treatment.”
It is this two-pronged opportunity—replacement therapies and treatment of amino acid-dependent cancers—that gives Aeglea a particular boost. There are several diseases that are caused by a deficiency of specific amino acids, for example, Arginase I Deficiency. A number of cancers are also dependent on specific amino acids to survive.
Aeglea’s lead pipeline compound is AEB1102, which is currently in three Phase I clinical trials for Arginase I Deficiency, in solid tumors, as well as in hematological cancers.
Various arginine-dependent cancers include liver, melanoma, ovarian, pancreatic, and uveal melanoma.
On August 29, Aeglea dosed its first patient with AEB1102 to treat acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). “We are rapidly executing on our strategy for AEB1102 in oncology, having now dosed our first patient with AML/MDS refractory to hypomethylating agents,” Sandra Rojas-Caro, the company’s chief medical officer, said in a statement. “Current treatment options for patients who have these forms of hematological malignancies are limited. AEB1102 may have the potential to offer a significant clinical benefit for these patients in need.”
AEB1102, Lowe said, “degrades the amino acid arginine in blood that we’re developing to treat two extremes of arginine metabolism. In the extreme instance, is enzyme replacement therapy. In this particular genetic disease, there’s too much arginine in the patient’s blood, and if they are missing an enzyme that degrades arginine. This results in an accumulation of nitrogen in the blood in the form of ammonia. So if the patient has toxic levels of arginine in their blood, we want to reduce that down to the normal range.”
Children who are untreated can develop seizures, spasticity, short stature and intellectual disability. It is quite rare and occurs in about 1 in 300,000 to 1 million births, according to the National Organization for Rare Disorders (NORD).
Current treatments include dialysis, intravenous and oral drugs known as nitrogen scavengers (Ammonul, Bupheny, Ravicti), and various anti-seizure medications.
On the other extreme of arginine metabolism is cancer. “We want to target amino acids specific to tumors that are addicted to arginine, so we’re taking the normal level of arginine in the blood down to a level the tumor can’t tolerate, but normal tissues otherwise can. This allows us to target a tumor growth pathway that otherwise can’t be blocked with typical antibody-based therapeutics,” Lowe said.
Aeglea went public in April. “We were very pleased to have very strong support from our inside syndicate, as well as attracting some very strong new investors. I think we’re well positioned to continue to fund the company going forward as we instill value incrementally, and by delivering on our critical milestones in the three clinical trials we currently have ongoing,” Lowe said.
Its IPO raised about $50 million. Prior to the IPO, Aeglea had raised $56 million in two equity rounds from six investors. Investors have included Lilly Ventures, Novartis Venture Fund, New Ground Ventures, KBI Biopharma, UT Horizon Fund and Venrock.
In addition to AEB1102, Aeglea has three other compounds in its pipeline, AEB3103, for cysteine/cysteine amino acid metabolism; AEB2109 for methionine metabolism; and AEB4104 for homocysteine metabolism. AEB3101 and 2109 are being developed for oncology indications. AEB4104 is being studied for classical homocystinuria, a rare inherited genetic disorders that affects about 1 in 344,000 individuals worldwide, and about 1 in 65,000 in Ireland.
Lowe noted that the company is growing and currently employs 30 people. A quick look at the company’s careers page on its website shows three current positions for clinical project management director, project manager/senior project manager, and associate director/director, patient advocacy.
“We’re adding talent. I don’t use the word headcount, it’s so impersonal. You bring people into the organization because of the talent they have. As a result, they’re really contributing meaningfully to the long-range goals of the organization, so I prefer to use the word talent. We have plans to continue growing and we need to build up the organization in several areas to effectively prosecute the development of AEB1102, as well as in the other cancer areas,” Lowe said.
For a small company, Aeglea has a lot going on, with three ongoing trials and a planned start on another, as well as the development of three other pipeline products. “We have a lot of opportunity with AEB1102 and the emerging pipeline as we unlock the value there. With the shorter path to market with the rare genetic disease, the potential for a nicely balanced business model with long-term value in rare diseases and cancer, we’re looking at a portfolio that really captures that concept,” Lowe said.
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