MALIGNANT PLEURAL EFFUSION (MPE)
Rare disease
Malignant Pleural Effusion (MPE) is the second most common cause of pleural exudate, a complication that involves the build-up of fluid containing cancer cells between the membranes that line the lungs. MPE occurs in 15% of all cancer patients and accounts for greater than 125,000 admissions per year in the United States, with costs at around $5 billion for inpatient charges alone. Diagnosis of MPE usually indicates an advanced or metastatic stage of the disease and portends a poor prognosis and limits the patient’s quality of life (QOL). The average life expectancy of patients with MPE is 3-12 months and is dependent on the underlying patient comorbidities.
Patients frequently experience debilitating dyspnea and other symptoms which severely compromise their QOL. Management of MPE is challenging and is mainly focused on the relief of symptoms and improving the patient’s QOL. Palliation of symptoms has been the goal for management of these effusions while keeping the patient’s hospital stay to a minimum. Palliative care includes the use of a thoracostomy (chest) tube for drainage followed by instillation of sclerosing agents in the pleural space.
Significant unmet need
MPE is a lethal disease with a low survival rate. There is an incidence of over 95,000 new patients. Lung cancer, breast cancer and lymphoma contribute to the majority of these effusions. Management of MPE is challenging and is mainly focused on the relief of symptoms and improving the patient’s QOL.
The treatment therapy for MPE needs to be tailored to individual patients taking into account their preferences, life expectancy, affordability and the resources available. Treatment options for the management of MPE include surgery, chemotherapy, radiation and multimodality treatment that includes surgery. Surgery is only possible with disease confined to the pleural space, so many patients are not eligible for this option. The current standard of care in chemotherapy is intravenous treatment, which has dose limiting toxicities that limit efficacy due to systemic toxicity.
Dyspnea is the most common symptom which occurs in almost all MPE patients. Even small effusions can cause significant dyspnea and patients experience relief of symptoms with drainage as low as 200 – 300 cc of pleural fluid, due to the deflation of the thoracic cage and associated improvement in respiratory muscle function.
The primary treatment involves thoracoscopic pleurodesis (TP) with talc slurry or chemotherapy agents. Palliative treatment includes indwelling thoracostomy tube for more frequent pleurodesis or drainage for relief of symptoms.
LiPax-PE
LIPAC is developing a new Liposomal Enhanced IntraThoracic Paclitaxel (LEITP) for the treatment of MPE utilizing the LiPax technology. Preclinical studies with LiPax-PE have demonstrated high potency of LiPax against existing chemotherapy treatment options. Our LiPax-PE program leverages the preclinical studies performed under the NMIBC program. In addition, the low survivability of MPE patients enables an efficient clinical end point of survival rate combined with the use of an accelerated approval pathway. LiPax-PE will leverage a potential early approval with a post approval Phase 3 study strategy.