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Mallinckrodt To Acquire InfaCare Pharmaceutical Corporation And Stannsoporfin, Its Proprietary Therapy In Late-Stage Development For Treatment Of Newborns At Risk For Developing Severe Jaundice

STAINES-UPON-THAMES, United Kingdom, Aug. 4, 2017 /PR Newswire/ -- Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, and InfaCare Pharmaceutical Corporation today announced that they have entered into an agreement under which Mallinckrodt will acquire InfaCare, a privately held specialty pharmaceutical company focused on development and commercialization of proprietary pharmaceuticals for neonatal and pediatric patient populations. InfaCare's developmental product stannsoporfin, a heme oxygenase inhibitor, is under investigation for its potential to reduce the production of bilirubin, the elevation of which can contribute to serious consequences in infants.

In July 2016 InfaCare and the U.S. Food and Drug Administration (FDA) reached agreement that a New Drug Application (NDA) could be filed for stannsoporfin using the totality of the drug's data package, including:

  • a positive Phase 2(b) trial as its pivotal study, and
  • data from a second positive Phase 2(b) trial, with
  • no additional studies required pre-approval.

This allowance reflects the medical need in infants at risk of developing severe jaundice. There are also challenges in conducting controlled trials in this fragile population.

In December 2016 the FDA also granted stannsoporfin its Fast Track designation, a process designed to facilitate development and expedite the review of drugs to treat serious conditions and fill an unmet medical need1. Fast Track status allows for a "rolling" NDA data submission that has recently begun, and approval is anticipated in the first half of 2018. Post-approval commitments required by the FDA would include conducting trials in pre-term infants less than 35 weeks gestational age as part of the pediatric requirements. If approved, the drug will have substantial durability both as a new chemical entity2 and through its intellectual property which is valid until 20323.

"Severe hyperbilirubinemia can result in serious complications in infants, including brain damage and, rarely, death," said Steven Romano, MD, Chief Scientific Officer and Executive Vice President of Mallinckrodt. "We look forward to bringing this much-needed treatment option to babies at greatest risk for the consequences of this condition."

"We believe stannsoporfin has the potential to help thousands of infants whose severe jaundice is unresolved by current treatments," said Dan Burns, President and Chief Executive Officer, InfaCare. "We're also excited by the additional development capability and commercial reach that can be gained by becoming part of Mallinckrodt. Together I'm confident we can successfully bring this important treatment to market."

Understanding Severe Jaundice (Severe Hyperbilirubinemia) Jaundice, or hyperbilirubinemia, is a common clinical condition seen in both term and pre-term newborns. Though lower levels of bilirubin can be benign, some newborns are at greater risk as a result of hemolysis (accelerated breakdown of red blood cells) contributing to the potential of reaching severe bilirubin levels. If a baby develops severe jaundice, there is a risk of bilirubin passing into the brain, a syndrome called acute bilirubin encephalopathy – which can be a serious condition. Prompt treatment may prevent significant lasting damage. But persistent, high levels of bilirubin in the brain can progress to kernicterus, a rare condition associated with severe and permanent brain damage. Symptoms include poor feeding, shrill cry, muscle rigidity, markedly arched back with a backwards hyperextension of the neck, seizures, and stupor or coma. Complications may also include hearing loss and death4,5. American Academy of Pediatrics guidelines recommend all newborns be assessed for the risk of hyperbilirubinemia prior to discharge from the hospital6.

Stannsoporfin Reduces Potential for Developing Severe Jaundice through Novel Method of Action Current therapies focus on removing excess bilirubin from the infant's system. Preliminary data from controlled clinical studies show that stannsoporfin's novel method of action demonstrated a robust effect in inhibiting bilirubin production. The drug has also been shown to have a good safety profile when compared to placebo, and has convenient administration through a single intramuscular injection. If approved, this proprietary therapy is expected to be the first and only pharmacologic treatment indicated for treatment of newborns at risk for developing severe infantile jaundice in the U.S.

"We believe stannsoporfin has the potential to significantly alter the treatment paradigm for infants with this condition which, if unchecked, can have devastating impact to the patient," said Mark Trudeau, Chief Executive Officer and President of Mallinckrodt. "The addition of this highly durable, unique developmental asset to our growing hospital business is an excellent example of Mallinckrodt's investment strategy."

InfaCare's founder and Chairman, Robert Vukovich, added, "A number of years ago we recognized the potential medical importance of stannsoporfin and the role it would play in treating severe neonatal jaundice and the potential serious neurological consequences of elevated bilirubin levels in newborns. Stannsoporfin is a breakthrough therapy and I am delighted to see that, with this transaction, the dream we have had to successfully develop this drug is coming to fruition, and we look forward to making it available for clinical use."

Global Severe Jaundice Market and Existing Treatment Paradigm In the U.S., the total number of term births is estimated at 3.7 million per year7,8 and, of those...