Mr. Ken d'Entremont reports

FDA APPROVES MEDEXUS'S SUPPLEMENTAL BIOLOGICS LICENSE APPLICATION FOR IXINITY TO TREAT HEMOPHILIA B IN PEDIATRIC PATIENTS

The U.S. Food and Drug Administration (FDA) recently approved Medexus Pharmaceuticals Inc.'s supplemental biologics licence application (sBLA) for Ixinity (coagulation factor IX (recombinant)) for the on-demand, prophylactic and perioperative treatment of pediatric patients under 12 years of age with hemophilia B. Ixinity, an intravenous recombinant factor IX therapeutic, is now approved for use in all patients with hemophilia B, a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood.

"We are pleased to secure approval for Ixinity for use in pediatric patients, a population that comprises approximately one-third of individuals with hemophilia B in the United States," said Ken d'Entremont, chief executive officer of Medexus. "The newly expanded indication makes Ixinity a viable factor IX option for children living with hemophilia B. We hope the greater availability of Ixinity will help ease the burden on children and families who face the challenge of managing hemophilia B and allow children living with this lifelong condition to enjoy more active lives."

"Ixinity effectively prevented and controlled bleeding episodes in the phase 3/4 pediatric study," commented Prof. Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, one of the study's principal investigators. "The study supported the efficacy and safety of Ixinity in pediatric patients, and the pharmacokinetics and safety profile were consistent with those observed in adults, although dose adjustment may be needed in pediatric patients. The results thus provide further validation of the clinical utility of Ixinity as a treatment for all people living with hemophilia B."

The sBLA and expanded indication for Ixinity are based on results from a phase 3/4 study that evaluated the pharmacokinetics (PK), safety and efficacy of Ixinity as a prophylactic treatment in previously treated pediatric patients under 12 years of age with severe or moderately severe hemophilia B. The study demonstrated that prophylaxis with Ixinity was associated with low annualized bleeding rates, effective control of bleeding episodes, consistent PK and a consistent safety profile. For more information about the study's methods, results and conclusions, see Medexus's Aug. 17, 2023, press release, available on the investors -- news and events section of Medexus's corporate website.

About Ixinity

Ixinity (coagulation factor IX (recombinant)) lyophilized powder for solution for intravenous injection is a coagulation factor IX (recombinant) indicated in adults and children with hemophilia B for on-demand treatment and control of bleeding episodes, for perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes.

The most common adverse reaction observed in greater than 2 per cent of patients in clinical trials was headache.

Ixinity is approved by the FDA for sale and use in the United States only and is not intended for export outside the United States. Medexus makes no representation that Ixinity is appropriate for or authorized for sale to or use by persons who are not located in the United States.

About hemophilia B

Hemophilia B is a genetic bleeding disorder caused by a deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles and soft tissues, resulting in joint damage, reduction in mobility and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor.

About Medexus Pharmaceuticals Inc.

Medexus is a leading specialty pharmaceutical company with a strong North American commercial platform and a growing portfolio of innovative and rare disease treatment solutions. Medexus's current focus is on the therapeutic areas of oncology, hematology, rheumatology, autoimmune diseases, allergy and dermatology.

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