Applied Therapeutics Reports AT-007 Single-Ascending Dose Data from Healthy Volunteer Portion of Phase 1/2 ACTION- Galactosemia Study
The results show that AT-007 was well tolerated, with no drug-related adverse events or dose-limiting toxicities reported. The SAD study treated 4 cohorts of 8 subjects each and explored doses from 0.5mg/kg to 20mg/kg. In addition to safety, AT-007 demonstrated a linear pharmacokinetic (PK) profile, favorable exposure, and half-life consistent with once-daily dosing.
“We are encouraged by the favorable safety profile in healthy volunteers, and we look forward to advancing AT-007 through the MAD study in healthy volunteers and to the Phase 2 portion of the study in patients with Galactosemia,” said
AT-007 will advance in parallel to a Phase 1b Multiple Ascending Dose (MAD) study in healthy volunteers (up to 7 consecutive days of treatment), and a Phase 2 study in adults with Galactosemia. Galactosemia patients will be eligible for treatment up to 28 days total (single dose followed by 27 consecutive days of dosing). In addition to safety and PK, the study will determine the ability of AT-007 to suppress toxic accumulation of galactitol in Galactosemia patients.
“AT-007 marks our second program to move through the clinic, and we are excited to see additional data in support of our technology and development strategy,” said
Galactosemia is a rare metabolic disease that affects how the body processes a simple sugar called galactose, and for which there is no known cure or approved treatment available. Galactose is found in foods, but the human body also naturally produces galactose on its own, so dietary restriction can’t prevent complications of disease. It is estimated that the U.S. Galactosemia population is approximately 2,800 patients, based on newborn screening data identifying 2,500 infants through 2014, and the estimated birth rate of 80 patients per year. High levels of galactose circulating in the blood and tissues of Galactosemia patients enables Aldose Reductase to convert galactose to a toxic metabolite, called galactitol, which causes long-term complications ranging from CNS dysfunction to cataracts.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in Phase 1/2 development for treatment of Galactosemia. AT-007 has been studied in in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications.
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding strategy, future operations, prospects, plans and objectives of management, including words such as "may," "will," "expect," "anticipate," "plan," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, statements regarding the (i) our cash runway and acceleration of our clinical development plan, (ii) the likelihood data will support future development of our product candidates, (iii) qualification for exemptions resulting from the receipt of orphan drug designation and (iii) the expected timing of the initiation of our clinical trials. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the uncertainties inherent in the initiation, execution and completion of clinical trials, in the timing of availability of trial data, in the results of the clinical trials, in the actions of regulatory agencies, in the commercialization and acceptance of new therapies. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the
(212) 600-1902 or
Galactosemia Patients/ Families:
If you are a patient or family member interested in receiving information regarding participation in the Phase 1/2 clinical trial, please email: firstname.lastname@example.org.
Source: Applied Therapeutics