Two and a half years ago APLT had just completed a phase 3 adult study and were expecting an NDA for their lead candidate, AT-007, in Galactosemia by Q3 2021. However, things took a different turn over the next 30 months.
During this period, I noticed three recurring trends at APLT. First, they had regular positive trial updates for AT-007. Second, there were frequent delays in the NDA submission. And third, they resorted to multiple dilutive stock offerings. These factors significantly impacted the company’s stock performance.
One major development occurred in September 2021 when the company decided to delay the NDA submission. They wanted to gather additional data to optimize dosing based on weight rather than age for AT-007. Despite consistently positive results in clinical trials, the FDA required a clinical outcomes study, which led to the delay.
In October, the company shared positive data from a pediatric study, showing that AT-007 substantially reduced plasma galactitol by about 40%, a statistically significant improvement compared to placebo.
In January of the following year, the company faced disappointment when the FDA changed their stance on accelerated approval and mandated a clinical outcomes study. As a result, the NDA filing had to wait until data from the phase 3 ACTION-Galactosemia Kids trial was available in Q1 2022.
Unfortunately, in April, the 18-month data from the trial revealed that AT-007 did not meet the primary endpoint with statistical significance. While the company highlighted some positive aspects of the trial, it struggled to meet the FDA’s requirements for strong and unquestionable data.
Moreover, APLT’s financial condition has become concerning. The company has had to resort to several rounds of fundraising through dilutive stock offerings, which negatively impacted the stock’s performance.
As for the disease being targeted, Galactosemia, it is a rare genetic disorder with a small patient population, but the market potential is still significant due to limited treatment options.
Overall, APLT’s future seems uncertain, given the lack of solid data and poor financial condition. The risks associated with the company include the need for additional funding and the possibility of regulatory challenges due to inadequate data. While some may see potential in the stock, I prefer to avoid uncertain gambles and, for now, I am opting not to invest in APLT.
