Additional performance-based milestones of up to
Xenpozyme is the only product developed and approved for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency ('ASMD'), also known as Niemann-Pick disease, in pediatric and adult patients. Xenpozyme was approved in
Xenpozyme is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin. In individuals with ASMD, the insufficient amount of the ASM enzyme means sphingomyelin is poorly metabolized, potentially leading to lifelong accumulation of sphingomyelin and damage to multiple organs.
Xenpozyme is a product that provides ASMD patients and their families with a disease-modifying therapy where none previously existed. The drug represents a transformational shift in therapies available to patients who suffer from ASMD' said
The transaction entitles DRI to royalties equal to approximately one percent of worldwide net sales of Xenpozyme. DRI is entitled to receive semi-annual royalty payments in respect of net sales of Xenpozyme commencing from the transaction date on a two-quarter lag from the respective half-year period. For sales made in the first and second quarters of a year, DRI expects to receive its royalty payment in the fourth quarter of that year. For sales made in the third and fourth quarters of the year, DRI expects to receive its royalty payment in the second quarter of the following year. DRI expects to receive royalties on net sales of Xenpozyme for approximately 15 years.
About Xenpozyme (olipudase alfa)
Xenpozyme addresses a significant unmet need in ASMD patients. It is an intravenously infused recombinant human acid sphingomyelinase enzyme intended to directly replace ASM expression in patients with ASMD, thereby improving clinical manifestations of the disease.
About Acid Sphingomyelinase Deficiency
ASMD is a rare, progressive, and potentially life-threatening lysosomal storage disorder. ASMD is a disease spectrum and patients are characterized by neurological (Type A) and non-neurological (Type B) manifestations. The estimated prevalence of ASMD is widely variable and is approximately 2,000 patients worldwide. Patients with ASMD Type B have varying degrees of non-neurologic symptoms such as hepatosplenomegaly, atherogenic lipid profile, interstitial lung disease with progressive impairment of pulmonary function and hematologic abnormalities including cytopenias. Other common clinical manifestations include liver dysfunction, heart disease, skeletal abnormalities, and growth delays. Some patients with ASMD who survive beyond early childhood have intermediate phenotypes (ASMD Type A/B) characterized by combinations of non-neurologic and mild to severe neurologic symptoms. Common symptoms include shortness of breath, joint or limb pain, abdominal pain, bleeding, and bruising. The disease often leads to chronic fatigue, limited physical or social activity and difficulties in performing daily activities or work. Many patients die before or in early adulthood, often from pneumonia, respiratory failure, or liver failure. Available treatments are limited to symptom management and supportive care.
About
Caution concerning forward-looking statements
This news release may contain forward-looking information within the meaning of applicable securities legislation. Forward-looking information generally can be identified by the use of forward-looking words such as 'expect', 'continue', 'anticipate', 'intend', 'aim', 'plan', 'believe', 'budget', 'estimate', 'forecast', 'foresee', 'close to', 'target' or negative versions thereof and similar expressions. Some of the specific forward-looking information in this news release may include, among other things, statements that we expect to receive royalties on net sales of Xenpozyme for approximately 15 years. Forward-looking information is based on a number of assumptions and is subject to a number of risks and uncertainties, many of which are beyond the Trust's control that could cause actual results to differ materially from those that are disclosed in or implied by such forward-looking information. These risks and uncertainties include, but are not limited to, those that are disclosed in the Trust's most recent annual information form. The forward-looking information in this news release is based on our assumptions regarding the performance of our royalty interest in Xenpozyme, including with respect to worldwide sales. All forward-looking information in this news release speaks as of the date of this news release. The Trust does not undertake to update any such forward-looking information whether as a result of new information, future events or otherwise except as required by law. Additional information about these assumptions and risks and uncertainties is contained in the Trust's filings with securities regulators, including its latest annual information form and Management's Discussion and Analysis. These filings are also available at the Trust's website at drihealthcaretrust.com.
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