Periodic Reporting for period 1 - TOPFIBRO (Targeting OGG1 in Idiopathic Pulmonary Fibrosis)
Reporting period: 2023-05-01 to 2024-04-30
Idiopathic Pulmonary Fibrosis (IPF) is a serious progressive lung disease that severely affects physical well-being and the condition develops when lung tissue becomes thick and stiff. It is characterized by a high degree of illness and mortality. Over time permanent scarring in the lung, called fibrosis, happens and this makes it difficult to breathe. Besides shortness of breath that becomes worse over time, additional symptoms can be a dry cough that is not improving, achy joints and muscles, feeling tired/weak and slowly losing weight. Common complications of IPF are pulmonary hypertension and respiratory failure. This happens when the lungs cannot deliver enough oxygen to the rest of the body, including the brain.
The underlying cause of IPF is unknown, but risk factors are for instance male sex, aging, smoking, environmental exposures (e.g. metal dust), infections (e.g. human herpes viruses, hepatitis C), gastroesophageal reflux disease, and a family history of IPF.
Today there is no cure for IPF. Average survival is only 3-5 years after diagnosis (Swedish Pulmonary Medicine Association 2019). There are two approved drugs on the market – pirfenidone (Esbriet) and nintedanib (Ofev). These treatments do not cure the disease or improve survival, only slow down the progression of IPF and help the lungs work better. They are limited by adverse effects and drug interactions. There are reports showing that > 40% of patients stop the treatment within the first year due to side-effects. Doctors are sometimes reluctant to initiate therapy due to the shortcomings of available therapy which in many markets are also very costly. Consequently, there is a large proportion of patients that receive suboptimal or no treatment.
Thus, the medical need for new effective and well tolerated treatments are huge.
Oxcia is developing a novel solution, OXC-201, based on completely new science published in the prestigious scientific journal Science 2018 by Professor Helleday, academic collaborators and Oxcia team. OXC-201 has the potential to improve and extend the lives of patients suffering from the currently incurable IPF. The goal of Oxcia’s research is to target the underlying inflammation and fibrosis in IPF to stop disease progression, protect and repair the lung tissue, improve lung function and halt the disease.
The underlying cause of IPF is unknown, but risk factors are for instance male sex, aging, smoking, environmental exposures (e.g. metal dust), infections (e.g. human herpes viruses, hepatitis C), gastroesophageal reflux disease, and a family history of IPF.
Today there is no cure for IPF. Average survival is only 3-5 years after diagnosis (Swedish Pulmonary Medicine Association 2019). There are two approved drugs on the market – pirfenidone (Esbriet) and nintedanib (Ofev). These treatments do not cure the disease or improve survival, only slow down the progression of IPF and help the lungs work better. They are limited by adverse effects and drug interactions. There are reports showing that > 40% of patients stop the treatment within the first year due to side-effects. Doctors are sometimes reluctant to initiate therapy due to the shortcomings of available therapy which in many markets are also very costly. Consequently, there is a large proportion of patients that receive suboptimal or no treatment.
Thus, the medical need for new effective and well tolerated treatments are huge.
Oxcia is developing a novel solution, OXC-201, based on completely new science published in the prestigious scientific journal Science 2018 by Professor Helleday, academic collaborators and Oxcia team. OXC-201 has the potential to improve and extend the lives of patients suffering from the currently incurable IPF. The goal of Oxcia’s research is to target the underlying inflammation and fibrosis in IPF to stop disease progression, protect and repair the lung tissue, improve lung function and halt the disease.
In the TOPFIBRO (Targeting OGG1 in Idiopathic Pulmonary Fibrosis) program supported by EIC Transition grant the overall aim is to complete the pre-clinical development of the drug candidate OXC-201 as well as prepare and initiate the first-in-human studies investigating safety of the novel IPF treatment in human healthy volunteers. Involvement of patient organizations, physicians and Key Opinion leaders is part of the plan to secure their input into the project development. Furthermore, Oxcia has frequent contacts with Pharma and investors to understand their requirements.
After the first year, Oxcia has obtained additional new data supporting the potential of OXC-201 as a novel IPF therapy. In disease models, OXC-201 has shown significant reduction in inflammation- and fibrotic markers important for the IPF development, suggesting that this drug candidate can hinder the progress of the disease. This has been validated in human IPF lung tissue, in so called ex vivo studies, where lung tissue from IPF patients is treated with the drug candidate. OXC-201 is shown to be more effective compared to standard of care, supporting the ground-breaking potential of this new treatment. Furthermore, preliminary data demonstrates improvement of the lung function with OXC-201 treatment in the disease models, something that will be further studied in the next coming months. The initial pre-clinical safety assessments have also been initiated. So far, OXC-201 seems to be well tolerated.
After the first year, Oxcia has obtained additional new data supporting the potential of OXC-201 as a novel IPF therapy. In disease models, OXC-201 has shown significant reduction in inflammation- and fibrotic markers important for the IPF development, suggesting that this drug candidate can hinder the progress of the disease. This has been validated in human IPF lung tissue, in so called ex vivo studies, where lung tissue from IPF patients is treated with the drug candidate. OXC-201 is shown to be more effective compared to standard of care, supporting the ground-breaking potential of this new treatment. Furthermore, preliminary data demonstrates improvement of the lung function with OXC-201 treatment in the disease models, something that will be further studied in the next coming months. The initial pre-clinical safety assessments have also been initiated. So far, OXC-201 seems to be well tolerated.
OXC-201 has the potential to radically change and grow the IPF market. It has potential to halt the disease, improve lung function, improve survival and be safe and tolerable. It has potential to protect lung tissue and repair lung tissue, thereby possibly turning IPF to a chronic disease rather than a deadly disease.
The pre-clinical work in WP1 has during the first 12 months, delivered data that confirms the promising profile of OXC-201. Patent applications of the new chemical entity have been approved in Japan, China and US - countries with high number of IPF patients. EU is pending and we don’t anticipate any issues with the EU patent.
The IPF market has grown, there is a lot of untreated patients due to the limitations of current treatments and the interest from big pharma and biotech is currently very high with a lot of acquisition and licensing activities and high deal values.
The market research done in the program has confirmed that the two approved therapies for IPF have severe limitations and that the unmet medical need is enormous. Costs are also very high.
OXC-201 has potential to provide important economic and societal benefits.
The foremost effect of TOPFIBRO will be accelerating the development of an urgently needed treatment for IPF. IPF is considered a terminal diagnosis and low survival rate of 3-5 years post diagnosis. Shortness of breath and severe cough, further decrease quality of life and overall capabilities of the patient. OXC-201 would be the first potentially curative therapy for IPF, thereby significantly improving quality and length of life compared with current standard of care treatment. There will also be other positive impacts following the project, for patients in the selected additional indications and regarding employment when Oxcia significantly gears up resource wise for further clinical development.
Oxcia's goal is to demonstrate health economic value with OXC-201. Today the cost burden is very high both for Health payers and patients, who are today not always able to afford treatment. As OXC-201 significantly improves lung function and survival and has very good tolerability, quality of life will increase and mean higher Quality Associated Life Years (QALYs) compared to current standard of care.
The pre-clinical work in WP1 has during the first 12 months, delivered data that confirms the promising profile of OXC-201. Patent applications of the new chemical entity have been approved in Japan, China and US - countries with high number of IPF patients. EU is pending and we don’t anticipate any issues with the EU patent.
The IPF market has grown, there is a lot of untreated patients due to the limitations of current treatments and the interest from big pharma and biotech is currently very high with a lot of acquisition and licensing activities and high deal values.
The market research done in the program has confirmed that the two approved therapies for IPF have severe limitations and that the unmet medical need is enormous. Costs are also very high.
OXC-201 has potential to provide important economic and societal benefits.
The foremost effect of TOPFIBRO will be accelerating the development of an urgently needed treatment for IPF. IPF is considered a terminal diagnosis and low survival rate of 3-5 years post diagnosis. Shortness of breath and severe cough, further decrease quality of life and overall capabilities of the patient. OXC-201 would be the first potentially curative therapy for IPF, thereby significantly improving quality and length of life compared with current standard of care treatment. There will also be other positive impacts following the project, for patients in the selected additional indications and regarding employment when Oxcia significantly gears up resource wise for further clinical development.
Oxcia's goal is to demonstrate health economic value with OXC-201. Today the cost burden is very high both for Health payers and patients, who are today not always able to afford treatment. As OXC-201 significantly improves lung function and survival and has very good tolerability, quality of life will increase and mean higher Quality Associated Life Years (QALYs) compared to current standard of care.