OCKHAM'S DISEASE FOCUS AND COMMERCIAL PROSPECTS

Why invest in Ockham Biotech?

Ockham Biotech Ltd is currently seeking partners for the commercialisation of inhaled heparin in COPD, bronchiectasis, asthma, cystic fibrosis and other diseases characterised by airway obstruction with mucus

Reducing the burden of treatment and meeting a clinical need

The reasons for administering mucolytics to patients with obstructive airways disease were summarised by Bruce Rubin, Wake Forest University, School of Medicine in Lancet, 359, June 2002:

"If you clear out all this garbage from the airways, you may have a better opportunity to treat the inflammation and provide relief to the patient"

Cystic fibrosis and non-cystic fibrosis bronchiectasis

Bronchiectasis is the UK's third most common chronic lung condition affecting one in 200 adults, yet remains largely unknown. It is complex, often under-diagnosed and misdiagnosed, and currently has no licensed treatments available. The prevalence of bronchiectasis increases with age – it affects over 1% of men and women aged over 70 years. Symptoms include cough, excess sputum production, dyspnea and episodes of exacerbation. It can be related to cystic fibrosis (CF), but is also found outside this indication (non-CF bronchiectasis (NCFBE)).

CF has traditionally been defined as the most common life-threatening inherited disorder of children in Caucasian populations, with an incidence of 1/2500 live births. However, recent advances in treatment have significantly improved the outlook for CF patients. There are close to 40,000 children and adults living with CF in the United States (and an estimated 105,000 people have been diagnosed with CF across 94 countries). Approximately 1,000 new cases of CF are diagnosed each year.

CF and NCFBE are characterised by cycles of infection, inflammation and mucus retention in the airways that, over time, lead to loss of lung function. A treatment that addresses all aspects of the disease will therefore be more effective and easier for patients to use than multiple individual treatments in isolation, which may have only modest effects on clinical outcomes.

In patients with CF, CFTR modulator therapy (e.g. Kaftrio) is available and highly effective for some, but not all, patients. However, even this therapy will not reverse established damage to the airways (bronchiectasis), which predisposes people with CF to infection and further airways inflammation. This perpetuates the cycle of airway damage.

In addition, with the emerging threat of microbes resistant to antibiotics, new anti-infection strategies are needed. There are no treatments licensed in Europe or USA for NCFBE. Therefore, there remains a need for more effective antimicrobial, anti-inflammatory and mucus-thinning drugs in CF and NCFBE. In addition, the treatment burden and the complexity of treatment is high in both patient groups contributing to reduced adherence to treatment.

Our development candidate, OCK4, has an excellent safety profile and multiple relevant activities. It is believed to reduce excessive mucus secretion and improve mucus clearance through its anti-inflammatory effects as well as direct effects that thin mucus. Anti-microbial effects are thought to be through mechanisms that avoid bacterial resistance to the treatment. Inhaled OCK4 simultaneously targets multiple components in the cycles of infection and inflammation that drive disease progression in CF and NCFBE.

We expect it will reduce the burden of treatment, while meeting a clinical need in diseases where there is substantial unmet clinical need, regardless of the cause of bronchiectasis and in both CF patients responsive to CFTR therapy as well as those that are non-responsive.

Chronic Obstructive Pulmonary Disease (COPD)

Beyond CF and NCFBE, COPD is also potentially treatable with our approach; this condition is predominantly caused by cigarette smoking and inhalation of environmental factors, including wood smoke from cooking over open fires, and is believed to affect up to 10% of the world population. It is expected that COPD will be the fourth leading cause of death worldwide by 2030. The COPD market was valued at $12.6 billion in 2023 and is expected to experience consistent growth over the next ten years.

There is a high level of unmet need for drugs that can reduce COPD exacerbation rates, slow or halt the progression of COPD and improve lung function, exercise capacity and the quality of life in COPD patients. There are no specific treatments for COPD. The development of novel inhaled therapies based on the multiple pharmacological properties of OCK4, both alone and in combination with other drugs, is expected to provide new treatment options for COPD patients worldwide, and will play a major role in the expansion of the COPD market in the coming years.