News:

Apellis Reports Positive Results from Phase 3 Study Comparing Pegcetacoplan (APL-2) to Eculizumab in PNH Patients

January 16, 2020 Apellis Pharmaceuticals, a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, announced positive results from the Phase 3 PEGASUS study evaluating pegcetacoplan (APL-2) in adults with PNH. Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16. The safety profile of pegcetacoplan was comparable with eculizumab in this study. Click here to read the company’s full press release.

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Akari Therapeutics Announces Positive Interim Data from Phase 3 Study of Nomacopan in PNH Patients

January 16, 2020 Akari Therapeutics, a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases, announced positive data from Phase 3 of their CAPSTONE study in complement inhibitor naïve, transfusion-dependent PNH patients. The interim data on the first eight patients, who were all transfusion dependent at entry to the CAPSTONE study, show that all four patients randomized to nomacopan were transfusion independent for the first six months of treatment, while all four patients on standard of care (blood transfusion with or without anticoagulation) remained transfusion dependent.    Click here to read the company’s full press release.

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New Phase 3 C3 inhibitor clinical trial begins

Apellis Pharmaceuticals Inc., a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, has initiated its PEGASUS Phase 3 clinical trial assessing the safety and efficacy of APL-2, a novel inhibitor of complement factor C3, in patients with PNH compared to eculizumab.

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The PNH Guide has been updated!

The Guide to Living Well with PNH has been updated to include information on the latest news in PNH research, navigating the complexities of accessing treatment through clinical trials, traveling with PNH, and more! Please click here to read more and to download or order your copy today.

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Positive Results for PNH Study

Alexion Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of life-transforming therapies for patients with devastating and rare diseases, recently announced positive results from a phase 3 study of their second generation of Soliris® (ALXN1210), for the treatment of PNH. The press release announcing these results can be read here.

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Positive Interim Results for PNH Study

Ra Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases, recently announced interim results from their ongoing, global Phase 2 study evaluating RA101495 for the treatment of PNH. The press release announcing these results can be read here.

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Ontario’s New OHIP+ Program

This post is for all patients living in Ontario who might be affected by the new OHIP+: Children and Youth Pharmacare Program. Beginning January 1, 2018, all children and youth 24 years of age or younger in Ontario will be able to get their prescription medications for free by simply showing their Ontario health card number and a prescription. Coverage will be automatic, with no upfront costs.

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New Research Page

The Canadian Association of PNH Patients (PNHCA) is thrilled to announce the creation of a new page on the PNHCA website called “What’s New in Research.” For the last 20 years, there has only been one Health Canada-approved treatment for PNH patients, but that may change in the coming years. There is a significant amount of activity underway to discover and develop new treatments for PNH, and right now, there are 10 organizations currently conducting clinical trials around the world.

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Rare Blood Disorders Supplement

Rare Blood Disorders Supplement Barry Katsof, president of the Canadian Association of PNH Patients, recently contributed to a rare blood disorders supplement that ran in print in Maclean’s Magazine, as well as online at PersonalHealthNews.ca. The article discusses Barry’s journey with PNH, the challenges he faced at diagnosis and how advancements in treatment options are helping patients with rare blood disorders live their lives to the fullest. To read the full article, click here.

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Drug Approval and Funding Process in Canada

Drug Approval and Funding Process in Canada If you think that deciphering how prescription medicines are approved and funded in Canada is akin to learning a new language, you’re right. It incorporates a combination of multiple processes and variations on those processes, and is complicated and complex. Just figuring out all the acronyms is enough to boggle the mind. If you’re looking for a simple snapshot of the process, we’ve got the solution for you. The following infographic will help you navigate a drug’s journey – from “bench to bedside. To download an expanded version of the infographic, click here.

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