Oxcia's O2-DDR platform forms the foundation
Two of the building blocks for life are DNA and oxygen and these are key players in Oxcias O2-DDR technology platform. According to the company, the platform has the potential to cure diseases and extend life by carefully controlling oxidative stress, oxidative DNA damage and DNA damage response in cells.
Damage to the cell's DNA is an underlying cause of several serious diseases, such as cancer, pulmonary fibrosis and psoriasis – disease areas that Oxcia focuses on in the development of new treatment methods. The company's platform is versatile and different therapeutic effects can be achieved depending on which proteins are targeted and how they are modified. The O2-DDR platform enables the application of oxidative stress to cancer cells to kill them, as well as blocking oxidative stress in inflammatory conditions to prevent the body from overreacting and causing tissue damage.
The company's lead drug candidate, OXC-101, is a first-in-class mitotic MTH1 inhibitor that exploits the Achilles heel of cancer cells – high oxidative stress and DNA damage. OXC-101 has a dual mechanism of action that both stops cell division, which increases oxidative stress, and makes it more difficult for cancer cells to repair DNA damage.
Orphan Drug Designation gives OXC-101 several advantages
In February, OXC-101 was granted Orphan Drug Designation (ODD) status by FDA for the treatment of AML. This means that OXC-101 receives several benefits, such as regulatory support, tax breaks, cost deductions and market exclusivity for up to 7 years in the US.
“We are very pleased with the FDA’s decision to grant OXC-101 ODD designation for AML. This is an important milestone and underscores the great medical need for new medicines and the unique approach of OXC-101,” says Oxcia’s CEO. Ulrika Warpman Berglund.
The company also plans to submit an application for ODD to European Medicines Agency (EMA).
Potential for OXC-101 in AML
The fact that OXC-101 has received ODD for AML underlines the great medical need for new treatment options for this aggressive blood cancer. AML is the most common type of leukemia in adults, accounting for approximately 80 percent of cases. At the same time, the disease is relatively uncommon compared to other cancers, which makes ODD status possible for new drugs in development.
AML has a high mortality rate, with a five-year survival rate of only approximately 20 percent in some patient groups. Oxcia sees potential to improve the prognosis for these patients with the drug candidate OXC-101 based on both preclinical and clinical data. Studies in preclinical AML models show that OXC-101 significantly prolongs survival and reduces tumor growth. In addition, the candidate has shown promising Phase I clinical data for the treatment of advanced hematological cancers.
Focus on expansion study
Oxcia is currently conducting a Phase I/II study in patients with relapsed or refractory AML, combining OXC-101 with idarubicin, one of the standard treatments for the disease.
The clinical trial is an expansion study with the aim of confirming the preliminary effect previously observed in AML and laying the foundation for the continued clinical development of the drug candidate.
Adding clinical trial centers
So far, the phase I/II study in AML has been conducted in Sweden, but to increase patient recruitment, Oxcia will also include Rigshospitalet in Copenhagen and, among others, several testing sites in Serbia.
The study is partly financed by a grant of SEK 3 million from Swelife and Medtech4HealthIn addition, the company plans to conduct a capital raising during Q2/Q3 to finance the continued development of both OXC-101 and the company's other drug candidate OXC-201 for the treatment of idiopathic pulmonary fibrosis.
CEO talks about development status and plans going forward
To find out more about the company's development, we contacted the CEO Ulrika Warpman Berglund.
Ulrika, can you give us an update on how the company's Phase I/II study with OXC-101 in patients with AML is progressing?
– The study is proceeding as planned. An initial review with an independent safety data group shows that the combination of OXC-101 and idarubicin is well tolerated. It is also exciting that Oxcia has been contacted by MD Anderson, one of the largest cancer institutions in the US, after OXC-101 received ODD status. They expressed interest in the project and requested further information.
– The fact that OXC-101 has received orphan drug status demonstrates the project's level of innovation, which in turn may attract more attention – something that also became clear when MD Anderson contacted us.
What will be the next step once the study is completed?
– Following the study, the goal is to initiate a randomized phase II study to create a basis for accelerated regulatory approval. We are also investigating the possibilities of including the USA in the next step and are intensifying the work of seeking partners/licensees.
Can you tell us more about the need for new treatment options for AML and how OXC-101 can help improve treatment options?
– Current treatment consists primarily of chemotherapy and mutation-specific therapy for a few subpopulations. A large proportion relapses. Chemotherapy has a broad effect but has problems with tolerability, not least for older patients, and the development of resistance. Mutation-specific therapy (for example, for FLT3 or IDH1/2 mutations) is only applicable to the patient group that has that particular mutation. For the majority of mutations, there is no treatment.
– More effective and tolerable alternatives are clearly needed. OXC-101 has a unique mechanism of action with the potential to treat a large proportion of patients, overcome resistance and improve patients' quality of life and longevity. That's what we are working towards!