Breaking News
Get Actionable Insights with InvestingPro+: Start 7 Day FREE Trial Register here
Investing Pro 0
Ad-Free Version. Upgrade your Investing.com experience. Save up to 40% More details

Applied Therapeutics Tanks as Drug Candidate for Rare Disease Faces Delays

Stock Markets Jan 03, 2022 08:14AM ET
Saved. See Saved Items.
This article has already been saved in your Saved Items
 

By Dhirendra Tripathi

Investing.com – Applied Therapeutics stock (NASDAQ:APLT) sank 25% in Monday’s pre-market trading after receiving bad news from the FDA for the company’s lead drug candidate.

The company was earlier expecting an accelerated approval for the drug AT-007, which aims to treat galactosemia, a rare genetic metabolic disease, from the Food and Drug Administration, but the regulator has now indicated that clinical outcomes data will likely be required for it, according to a company release. The company has thus decided to hold on submitting a new drug application for AT-007 for treatment of galactosemia pending additional discussions with the FDA.

The first assessment of the clinical outcome will be completed in the first quarter of 2022, and then every 6 months thereafter until the study reaches statistical significance, the release said.

The company is developing AT-007 for treatment of galactosemia, a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior and motor skills.

AT-007 is currently being studied in a phase-3 clinical outcomes trial (ACTION-Galactosemia Kids) in 2-17-year-olds suffering from galactosemia. There are approximately 3,000 patients with galactosemia in the US and 80 new births per year.

Applied Therapeutics Tanks as Drug Candidate for Rare Disease Faces Delays
 

Related Articles

Add a Comment

Comment Guidelines

We encourage you to use comments to engage with other users, share your perspective and ask questions of authors and each other. However, in order to maintain the high level of discourse we’ve all come to value and expect, please keep the following criteria in mind:  

  •            Enrich the conversation, don’t trash it.

  •           Stay focused and on track. Only post material that’s relevant to the topic being discussed. 

  •           Be respectful. Even negative opinions can be framed positively and diplomatically. Avoid profanity, slander or personal attacks directed at an author or another user. Racism, sexism and other forms of discrimination will not be tolerated.

  • Use standard writing style. Include punctuation and upper and lower cases. Comments that are written in all caps and contain excessive use of symbols will be removed.
  • NOTE: Spam and/or promotional messages and comments containing links will be removed. Phone numbers, email addresses, links to personal or business websites, Skype/Telegram/WhatsApp etc. addresses (including links to groups) will also be removed; self-promotional material or business-related solicitations or PR (ie, contact me for signals/advice etc.), and/or any other comment that contains personal contact specifcs or advertising will be removed as well. In addition, any of the above-mentioned violations may result in suspension of your account.
  • Doxxing. We do not allow any sharing of private or personal contact or other information about any individual or organization. This will result in immediate suspension of the commentor and his or her account.
  • Don’t monopolize the conversation. We appreciate passion and conviction, but we also strongly believe in giving everyone a chance to air their point of view. Therefore, in addition to civil interaction, we expect commenters to offer their opinions succinctly and thoughtfully, but not so repeatedly that others are annoyed or offended. If we receive complaints about individuals who take over a thread or forum, we reserve the right to ban them from the site, without recourse.
  • Only English comments will be allowed.

Perpetrators of spam or abuse will be deleted from the site and prohibited from future registration at Investing.com’s discretion.

Write your thoughts here
 
Are you sure you want to delete this chart?
 
Post
Post also to:
 
Replace the attached chart with a new chart ?
1000
Your ability to comment is currently suspended due to negative user reports. Your status will be reviewed by our moderators.
Please wait a minute before you try to comment again.
Thanks for your comment. Please note that all comments are pending until approved by our moderators. It may therefore take some time before it appears on our website.
 
Are you sure you want to delete this chart?
 
Post
 
Replace the attached chart with a new chart ?
1000
Your ability to comment is currently suspended due to negative user reports. Your status will be reviewed by our moderators.
Please wait a minute before you try to comment again.
Add Chart to Comment
Confirm Block

Are you sure you want to block %USER_NAME%?

By doing so, you and %USER_NAME% will not be able to see any of each other's Investing.com's posts.

%USER_NAME% was successfully added to your Block List

Since you’ve just unblocked this person, you must wait 48 hours before renewing the block.

Report this comment

I feel that this comment is:

Comment flagged

Thank You!

Your report has been sent to our moderators for review
Continue with Google
or
Sign up with Email