Aeglea BioTherapeutics issued an update on Tuesday for its ongoing Phase 1/2 trial of pegzilarginase in patients with a rare genetic disease, Arginase 1 Deficiency (ARG1-D).
While some companies wait until clinical trials are finished with dosing and collecting data to begin releasing information, those that release interim data can give investors a better handle on how the trial is going.
On Tuesday (September 4), Aeglea BioTherapeutics (NASDAQ:AGLE) issued an update for its ongoing Phase 1/2 trial of pegzilarginase in patients with a rare genetic disease, Arginase 1 Deficiency (ARG1-D).
The trial released positive interim clinical data and surpassed its enrollment target with 15 patients.
Clinical data was presented at the 2018 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium place in Athens, Greece . Additional data will be presented at the American Society of Human Genetics conference in October.
“Given the challenges of lowering arginine levels with current approaches, it is very encouraging to see the marked reductions in plasma arginine in our patient following treatment with pegzilarginase,” George Diaz, chief of division of medical genetics at the Icahn School of Medicine at Mount Sinai, said at the symposium presentation. “We are seeing evidence of an impact on important disease manifestations with better walking, improved posture, and enhanced alertness.”
Other results from the trial include the drug was well-tolerated, all patients saw improvements at eight weeks of repeat dosing, and there were no marked or sustained increases in anti-drug antibodies titers. All of this data combined means the development of pegzilarginase will continue.
Investors can look forward to future milestones for the study in Q4 2018 when the company plans to release the pivotal study design of ARG1-D. The company anticipates the trial will start in the first half of 2019.
ARG1-D is a debilitating urea cycle disorder with no current effective therapy. This urea cycle disorder is caused by a deficiency of a key arginine metabolizing enzyme which leads to hyperargininemia-related neurological problems and early mortality, among other conditions.
Aeglea believes there are 600 potential patients with the disease; it plans to enter the addressable markets if the drug is successful.
However, Anthony Quinn, the company’s CEO, said he feels very encouraged it has already found 140 patients with the disease in the US and believes the market potential could be higher. According to Quinn, some patients may have been wrongly diagnosed, which widens the potential span of the market.
Pegzilarginase comes into play as an enzyme replacement therapy in patients to reduce the elevated blood arginine levels, which the trial’s early data has shown to perform.
This is the Aeglea’s lead investigational therapy which represents one of five ongoing trials. Pegzilarginase is also in development for uveal and cutaneous melanoma and small cell lung cancer.
As with these trials, the company designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer.
As of market close on Tuesday, Aeglea’s share price decreased 15.46 percent to US$9.24.
TipRanks shows, Wells Fargo analyst Jim Birchenough issued a note to investors on Tuesday, downgrading the company’s stock to a “Hold.”
Still, interested investors can follow the company’s news for its upcoming milestones, including the pivotal trial design in Q4 2018 and commencement of the trial by the first half of 2019.
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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.