PledPharma applies for an Orphan Drug Designation (ODD) in the US for Aladote

Stockholm, December 24, 2018. PledPharma AB (publ) announces that the company has filed an application for an Orphan Drug Designation (ODD) to the US FDA for the drug candidate Aladote^®, developed to reduce liver injury caused by paracetamol overdose.

The current standard of care for paracetamol poisoning (NAC) is effective if the patient seeks medical care within 8 hours of ingestion. Patients arriving later to the hospital, and for those with  high paracetamol levels, there is a need for a more effective treatment. Aladote^® is a first-in-class drug candidate developed to prevent the development of acute liver injury caused by paracetamol overdose.

Aladote^® is considered by PledPharma to fulfil the requirements for Orphan drug status in the US, i.e. less than 200,000 patients per year are affected from paracetamol overdose and the scientific rational as well as the clinical results from the recently completed proof-of-principle study indicate that Aladote^® has a potential in reducing liver injury for these patients. The FDA is expected to respond to the application in the first half of 2019.

"The reason for an application is the benefits of an orphan drug status. If granted, we are likely to acquire in total less development costs and a shorter development lead time.Furthermore, we receive additional dedicated FDA support during the drug development and get seven years of market exclusivity following a market approval," says Nicklas Westerholm, CEO, Pled Pharma AB.

For further information, please contact:
Nicklas Westerholm, CEO
Phone +46 (0)73 354 20 62
nicklas.westerholm@pledpharma.se

Yilmaz Mahshid, CFO
Phone +46 (0)72 231 68 00
yilmaz.mahshid@pledpharma.se

About Aladote^®
Aladote^® is a "first-in-class” drug candidate with the potential to prevent the development of acute liver injury caused by paracetamol overdose. Aladote® has shown good efficacy in relevant preclinical models, even in the time-window when N-acetylcysteine ??(NAC) treatment is no longer is effective. A proof of principle study in patients with paracetamol poisoning has been successfully completed.
Paracetamol is the most used drug in the world for the treatment of fever and pain, but also one of the most overdosed drugs – intentional or unintentional. Paracetamol overdose is also one of the most common method in intentional suicide attempts. When excessive amounts of paracetamol are broken down in the liver, the harmful metabolite NAPQI is formed, which can cause acute liver failure. The current standard of care for paracetamol poisoning (NAC) is effective if the patient seeks medical care within 8 hours of ingestion. However, NAC is substantially less effective if started more than 8 hours after overdose.
About the Aladote^® proof of principle-studie
The primary objective of the trial was to evaluate the safety and tolerability of Aladote^® in combination with N-acetylcysteine (NAC). NAC is the current standard of care for the treatment of paracetamol poisoning. In addition, several biomarkers of liver damage were measured. In total, 24 patients were recruited to three different dose cohorts with eight patients per cohort. In each cohort, six patients were treated with the combination of Aladote® and NAC and two were treated with NAC alone as control. The study results established the safety and tolerability of the combination of Aladote® and NAC. Further, the results shows that Aladote^® may reduce liver injury in this patient population. This is based on the measurement of the pre-defined exploratory biomarkers, Keratin-18 (K18) and microRNA-122 (miR-122) in patients treated with Aladote® and NAC compared to NAC alone. There was no difference in alanine transaminase (ALT) activity across the treatment groups.

About PledPharma
PledPharma develops new drugs that protect the body against oxidative stress – a potentially debilitating and sometimes life-threatening condition that can be caused by chemotherapy treatment and following acetaminophen (paracetamol) overdose. The company's most advanced project PledOx^® is being developed to reduce nerve damage associated with chemotherapy. A phase IIb study has been conducted and serves as the basis for the initiated global phase III program. The drug candidate Aladote^® is being developed to reduce the risk of acute liver failure associated with acetaminophen poisoning. A proof of principle study has been successfully completed and will serve as the basis for the continued development. PledPharma (STO: PLED) is listed on Nasdaq First North. Erik Penser Bank is the company’s Certified Adviser (tel +46 8 463 80 00). For more information, see http://www.pledpharma.se/