Karolinska Development's portfolio company Aprea Therapeutics has received FDA Orphan Drug Designation and Fast Track Designation for APR-246
STOCKHOLM, April 16, 2019. Karolinska Development's portfolio company Aprea Therapeutics has from FDA received an Orphan Drug Designation for APR-246 for the treatment of patients with Myelodysplastic Syndromes (MDS) having a TP53 mutation. In addition, FDA has also granted Fast Track Designation to APR-246 for treatment of MDS.
Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, tax credits related to clinical trial expenses, and an exemption from FDA user fees.
The FDA's Fast Track program facilitates the development of drugs intended to treat serious conditions and that have the potential to address unmet medical needs. A drug program with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval.
Aprea's drug candidate APR-246 has been shown to reactivate mutant and inactivated p53 protein and has demonstrated pre-clinical anti-tumor activity in a wide variety of solid and hematological (blood) tumors, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to preclinical testing, a Phase I/II clinical program with APR-246 has been completed, demonstrating a favorable safety profile, biological activity and clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene. Based on the high overall response rate (ORR) and complete response rate (CR) in these studies, a Phase III study in p53-mutated, high-risk MDS patients has been initiated.
"The decision by FDA showcase the importance of the strong data that Apreas has been able to show for APR-246. This drug candidate has a huge potential in treating cancer patients that lack efficient treatments today. We are looking forward to continuously follow the development of APR-246 as Aprea work hard to bring the drug candidate to patients as soon as possible," comments Karolinska Development's CEO Viktor Drvota.
For further information, please contact:
Viktor Drvota, CEO, Karolinska Development AB
Phone: +46 73 982 52 02, e-mail: viktor.drvota@
TO THE EDITORS
About Karolinska Development AB
Karolinska Development AB (Nasdaq Stockholm: KDEV) is a Nordic life sciences investment company. The company focuses on identifying breakthrough medical innovations in the Nordic region that are developed by entrepreneurs and leadership teams. The Company invests in the creation and growth of companies that advance these assets into commercial products that are designed to make a difference to patients' lives while providing an attractive return on investment to shareholders.
Karolinska Development has access to world-class medical innovations at the Karolinska Institutet and other leading universities and research institutes in the Nordic region. The Company aims to build companies around scientists who are leaders in their fields, supported by experienced management teams and advisers, and co-funded by specialist international investors, to provide the greatest chance of success.
Karolinska Development has established a portfolio of ten companies targeting opportunities in innovative treatment for life-threatening or serious debilitating diseases.
The Company is led by an entrepreneurial team of investment professionals with a proven track record as company builders and with access to a strong global network.
For more information, please visit www.karolinskadevelopment.com