Prescient’s (ASX: PTX) PTX-100 bags Orphan Drug Designation

Be the First to Comment Read
Sponsored

Prescient’s (ASX: PTX) PTX-100 bags Orphan Drug Designation

Follow us on Google News:
 Prescient’s (ASX: PTX) PTX-100 bags Orphan Drug Designation
Image source: © Jhvephotos | Megapixl.com

Highlights

  • PTX-100 receives Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of peripheral T-cell lymphomas (PTCL)
  • PTCL is a blood cancer with unmet clinical need
  • One of the benefits of Orphan Drug Designation is that it provides Prescient a guaranteed exclusive market of seven years.
  • It is believed that PTX-100 is the world’s only GGT-1 (a key cancer growth enzyme) inhibitor under clinical development.
  • Expansion cohort in current trial on track to fully recruit this year under the leadership of globally-renowned lymphoma expert, Professor H. Miles Prince, AM.

 

Prescient Therapeutics (ASX: PTX) has crossed a crucial milestone in its journey towards developing personalised therapies against cancer. PTX-100, one of its targeted therapies, has received Orphan Drug Designation from the Office of Orphan Products Development at the US Food and Drug Administration (FDA) for treating peripheral T-cell lymphomas (PTCL), a blood cancer with high unmet clinical need.

Prescient will benefit from the incentives provided by Orphan Drug Designation, supercharging the development of PTX-100. Following the news, shares of PTX soared by 12.5%, and were trading at AU$0.18 (as of 10:47:03 AEST, 15 July 2022) on the ASX.

Prescient is a clinical-stage oncology company having a strong pipeline of personalised cancer therapies. It consists of CAR-T and targeted therapies for fighting different cancer types.

What is Orphan Drug Designation?

Orphan Drug Designation is a special status provided to drugs intended for rare diseases affecting less than 200K people in the US. It provides incentives for drug development in less common diseases and includes:

  • Secured market exclusivity of seven years from granting of regulatory approval
  • A waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, which is worth US$3.1 million in 2022

First in class compound

PTX-100, currently under Phase 1b expansion cohort study in T cell lymphomas, can potentially block geranylgeranyl transferase-1 (GGT-1). GGT-1 is a crucial enzyme involved in cancer growth, and it is believed that PTX-100 is the only GGT-1 inhibitor in the globe under clinical development.

Image Source: © 2022 Kalkine Media®

PTCL is a serious unmet medical need. Currently, therapies for this disease show a high occurrence of serious toxicities, low response rate and short duration of responses. It has a poor survival rate following relapse and has not considerably improved in the last 20 years.

Image source: © 2022 Kalkine Media®

In clinical trials to date, PTX-100 has demonstrated a promising safety profile with inspiring efficacy signals, including one PTCL patient, who displayed a strong response and is still on therapy after 28 months.

Image source: © 2022 Kalkine Media®

 

To know more about Prescient Therapeutics Limited, click here. Also, to stay updated with PTX company activities and announcements, please update your details on their investor centre.

 

Disclaimer

Featured Articles

We use cookies to ensure that we give you the best experience on our website. If you continue to use this site we will assume that you are happy with it. OK