At Prometic Life Sciences we are committed to addressing unmet medical needs in rare diseases such as Congenital Plasminogen Deficiency (PLGD). PLGD is a very rare genetic disease that occurs when the level of plasminogen in the body is absent or very low.
Plasminogen is initially released from the liver and converted to the active enzyme plasmin by tissue plasminogen activator (tPA) and urokinase type plasminogen activator (uPA). The principal role of plasmin is to dissolve fibrin blot clots, however it also plays an important role in wound healing, cell migration, tissue remodelling, angiogensis and embryogensis.
Patients with this condition tend to form fibrous deposits on mucus membranes throughout the body. Ligneous conjunctivitis occurs when such growth arise in and around the eye lids and is one of the most prevalent manifestations. Severe complications can manifest if these lesions are left untreated. These may include loss of hearing, loss of sight and dentition, recurrent pneumonia, airway obstruction, urethral obstruction and even death. Existing medical and surgical treatment options are limited and have not been consistently effective. Fibrous lesions frequently reoccur shortly after surgical removal.
The first therapeutic in Prometic's development pipeline is a concentrated plasminogen replacement therapy for congenital plasminogen deficiency. This product can help control and reduce fibrous lesions and may be administered over an extensive period of time to prevent new growths from occuring. Physicians will monitor pharmacokinetic levels and patient response and may choose to escalate the dose for optimal efficacy. Treatment at all doses is well tolerated and safe with improvement observed across multiple organs.
Plasminogen replacement therapy offers new hope for patients with this rare disease.