Prometic Announces Positive Long Term Clinical Data on Ryplazym™ in Plasminogen Congenital Deficiency and Provides Regulatory Update
- Category: Proteins and Peptides
- Published on Tuesday, 11 July 2017 14:58
- Hits: 1926
- Ryplazym™ (Plasminogen IV) long-term treatment shown to prevent recurrence of lesions at 48 weeks
- Ryplazym™ (Plasminogen IV) maintains the same safety, tolerability profile without any serious adverse events at 48 weeks
- FDA inspection of RyplazymTM manufacturing facility, as part of ongoing BLA evaluation, currently scheduled for summer 2017
- 48-week data to form basis of regulatory filing with Health Canada in 4Q 2017
LAVAL, Canada I July 11, 2017 I Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) ("Prometic" or the "Corporation") today announced new long-term clinical data from its pivotal Phase 2/3 trial of Ryplazym™ (Plasminogen IV) in patients with congenital plasminogen deficiency. The data demonstrates that in 10 patients treated with RyplazymTM for a total of 48 weeks, there was no recurrence of lesions and no safety or tolerability issues observed related to this longer-term dosing.
Prometic has previously reported data from this pivotal Phase 2/3 trial, which showed that RyplazymTM treatment consistently replaced and maintained the plasminogen concentration at an appropriate level and that it resolved all lesions in all 10 patients treated for 12 weeks. These data fulfilled the clinical information required for the Biologics License Application (BLA) filing with the US Food and Drug Administration (FDA) for the Accelerated Regulatory Pathway Approval. Under the same pivotal Phase 2/3 protocol, these 10 patients have been treated for an additional 36 weeks, for a total drug exposure period of 48 weeks. In addition to the dossier filed with the FDA, the 48-week clinical efficacy data will form the basis for the upcoming regulatory filing with Health Canada in the fourth quarter of 2017.
"This pivotal Phase 2/3 trial of RyplazymTM has successfully met all primary and secondary endpoints, and we are pleased to announce these follow-up results which confirm the durability of the positive clinical efficacy and safety profile out to 48 weeks", stated John Moran, M.D., Chief Medical Officer of Prometic. "The 48-week clinical data will be submitted as a supplement to our BLA filing, after RyplazymTM receives its expected accelerated approval in the fourth quarter of 2017."
Pursuant to the BLA process, the FDA's inspection of Prometic's RyplazymTM manufacturing facility is scheduled to take place this summer. Also as part of the rolling BLA process, the Corporation is responding diligently to all FDA requests which to date are not related to underlying clinical data for RyplazymTM and will provide further updates on the exact expected timeline of regulatory approval as soon as available.
The RyplazymTM Phase 2/3 trial is still underway, and the dataset for all 15 patients that will have been treated over a period of 48 weeks is expected to be an acceptable demonstration of the efficacy of plasminogen therapy in patients with plasminogen congenital deficiency for full licensure, without the need for any additional clinical trials.
Prometic is also in discussions with the European Medicines Agency (EMA) with regards to the clinical information which would be required to secure regulatory approval of RyplazymTM in Europe and expects to be able to provide an update thereon in the fall of 2017.
Finally, Prometic has provided further data to the FDA to support its claims that the serious and life-threatening clinical manifestations associated with congenital plasminogen deficiency occurs predominantly in pediatric patients, as required to qualify for a rare pediatric disease designation.
Ryplazym Phase 2/3 Clinical Data Presented at ISTH Congress in Berlin
Clinical data for the 10 patients with 12 weeks of plasminogen treatment were featured in a poster session yesterday, July 10, at the International Society on Thrombosis and Hemostasis (ISTH) Congress, currently taking place in Berlin. To view a copy of the poster, refer to http://prometic.com/wp-content/uploads/2017/07/ISTH-plasminogen-phase-2-3-poster-final-submitted-19-Jun.pdf
Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.
About Plasminogen Deficiency
The most common condition associated with plasminogen deficiency is ligneous conjunctivitis, which is characterized by thick, woody (ligneous) growths on the conjunctiva of the eye, and if left untreated, can lead to corneal damage and blindness. Ligneous growths tend to recur after surgical excision, thereby requiring multiple surgeries.
While ligneous conjunctivitis is the best characterized lesion of plasminogen deficiency, hypoplasminogenemia is a multi-systemic disease that can also affect the ears, sinuses, tracheobronchial tree, genitourinary tract, and gingiva. Tracheobronchial lesions including hyperviscous secretions can result in respiratory failure. Hydrocephalus has also been reported in children with severe hypoplasminogenemia, thought to be related to the deposition of fibrin in the cerebral ventricles.
About Prometic Life Sciences Inc.
Prometic Life Sciences Inc. (www.prometic.com) is a long established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. Prometic is active in developing its own novel small-molecule therapeutic products targeting unmet medical needs in the field of fibrosis, cancer and autoimmune diseases/inflammation. A number of plasma-derived and small molecule products are under development for orphan drug indications. Prometic also offers its state of the art technologies for large-scale purification of biologics, drug development, proteomics and the elimination of pathogens to a growing base of industry leaders and uses its own affinity technology that provides for highly efficient extraction and purification of therapeutic proteins from human plasma in order to develop best-in-class therapeutics and orphan drugs. 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, Russia, Australia and Asia.
SOURCE: Prometic Life Sciences