Prometic granted orphan drug designation by FDA for Inter-Alpha-Inhibitor-Proteins for treatment of necrotizing enterocolitis (NEC)

February 15, 2018 Frederic Dumais
  • Additional orphan drug designation added to growing portfolio of unique plasma-derived therapeutics
  • NEC is the most common acquired gastrointestinal disease in premature neonates and one of the leading causes of death in neonatal intensive care units
  • The economic cost of NEC is high, accounting for approximately 19% of neonatal expenditures and an estimated cost of $5 billion per year for hospitalizations in the USA

LAVAL, QUEBEC, CANADA – February 15, 2018 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“Prometic”) announced today that an orphan drug designation status has been granted to its Inter-Alpha-Inhibitor-Proteins (“IaIp”) for the treatment of necrotizing enterocolitis (“NEC”) by the US Food and Drug Administration (“FDA”).

IaIp are endogenous proteins that control excessive inflammatory responses to toxins, infectious organisms, and tissue and organ damage. An inverse correlation between IaIp levels in plasma and disease severity / mortality has been demonstrated in humans with sepsisIn a gold-standard animal model proven to emulate NEC in humans, the supplementation of IaIp significantly increased the survival rate.

“We know that IaIp are rapidly consumed and cleared from circulation during severe sepsis cases, as evidenced by the significantly lower plasma levels of IaIp in newborns suffering from NEC. There is also strong scientific evidence indicating that IaIp play a key role in modulating systemic inflammation”, said Dr. John Moran, Prometic’s Chief Medical Officer. “We believe that systemic IaIp administration to replenish these decreased levels could therefore reduce the severity of inflammation and resulting tissue injury experienced by NEC patients”.

Pierre Laurin, Prometic’s President and Chief Executive Officer, commented: “IaIp are just another example of rare proteins that could be made accessible to address unmet medical needs through the use of our proprietary plasma purification platform. The sequential addition of these rare proteins is key to our strategy of building a unique portfolio of innovative therapeutics targeting unmet medical needs while generating substantial revenue levels with each liter of plasma purified”.

NEC is the most common acquired gastrointestinal disease diagnosed in premature neonates and is one of the leading causes of death in neonatal intensive care units. The economic cost of NEC is high, accounting for approximately 19% of neonatal expenditures and an estimated $5 billion per year for hospitalizations in the United States alone. If the disease is able to be treated without surgery, the average cost of hospitalization has been estimated at around $73 700, with a length of stay exceeding 22 days more than that for other premature infants. However, if surgical care is required, there is an average additional cost of $186 200, and infants require a length of stay 60 days longer than other premature infants.

Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.

About Inter-Alpha-Inhibitor-Proteins (IaIp)

Inter-alpha inhibitor proteins (IaIp) are serine proteases inhibitors that modulate endogenous protease activity. Severe sepsis results in reduced IαIp with the loss of protease inhibitory activity. Treatment with IαIp is protective in numerous preclinical sepsis models

About Necrotizing Enterocolitis 

Necrotizing enterocolitis (NEC) is a devastating inflammatory bowel condition that affects predominantly premature infants. NEC can ultimately destroy the wall of the bowel (intestine) and lead to perforation of the intestine and spillage of stool into the infant’s abdomen, which can result in an overwhelming infection and death. The cause of NEC is not well understood but appears to involve bacteria, injury to the bowel lining, inadequate oxygen supply to the bowel, and an abnormal immune response. Overall, NEC affects an estimated 8,000 – 12,000 live births each year in the USA. The disease has been reported to affect about 11 percent of very low birthweight infants born before 29 weeks of age. Mortality rates are high and range from about 15% to 30%.

About Prometic Life Sciences Inc. 

Prometic Life Sciences Inc. (www.prometic.com) is a biopharmaceutical corporation with two drug discovery platforms focusing on unmet medical needs in the field of fibrosis and orphan diseases. The first platform, small molecule therapeutics, stems from the discovery of two receptors GPR40/GPR84 acting as dual master-switches which are at the core of the healing process as opposed to fibrosis. The second platform, plasma-derived therapeutics, leverages Prometic’s vast experience in bioseparation technologies to address unmet medical needs with therapeutic proteins not currently commercially available, such as Ryplazim™ (plasminogen human). Prometic is also leveraging the second platform higher recovery yield advantage to develop some more established plasma-derived therapeutics with significant growth in demand such as Intravenous Immunoglobulin (IVIG) and provides access to its proprietary bioseparation technologies to enable pharmaceutical companies in their production of non-competing biopharmaceuticals. Globally recognized as a bioseparations expert, the Corporation derives revenue from this activity through sales of affinity chromatography media which contributes to offset the costs of its own R&D investments. Headquartered in Laval (Canada), Prometic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and commercial activities in the U.S., Canada, Europe and Asia.

Forward Looking Statements 

This press release contains forward-looking statements about Prometic’s objectives, strategies and businesses that involve risks and uncertainties. These statements are “forward-looking” because they are based on our current expectations about the markets we operate in and on various estimates and assumptions. Actual events or results may differ materially from those anticipated in these forward-looking statements if known or unknown risks affect our business, or if our estimates or assumptions turn out to be inaccurate. Such risks and assumptions include, but are not limited to, Prometic’s ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the ability of Prometic to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. You will find a more detailed assessment of the risks that could cause actual events or results to materially differ from our current expectations in Prometic’s Annual Information Form for the year ended December 31, 2016, under the heading “Risk and Uncertainties related to Prometic’s business”. As a result, we cannot guarantee that any forward-looking statement will materialize. We assume no obligation to update any forward-looking statement even if new information becomes available, as a result of future events or for any other reason, unless required by applicable securities laws and regulations. All amounts are in Canadian dollars unless indicated otherwise.

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