Apellis

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We are writing to introduce ourselves and to share some exciting cold agglutinin disease (CAD) news. 

Apellis is a development stage, global biopharmaceutical company that was established 10 years ago to combine courageous science, creativity, and compassion to deliver life-changing medicines for a broad range of serious diseases. Our entire team has a commitment to patients, their caregivers, and communities. We are sincere when we say that we care deeply.

Our programs are focused on targeting the complement cascade, a part of the immune system that eliminates damaged or diseased cells in the body as one of its functions. Specifically, pegcetacoplan (APL-2) targets C3, a protein that plays a very important role in the early stage of the complement cascade. Uncontrolled complement activation is thought to drive the onset of many diseases. Pegcetacoplan is an investigational drug currently being studied in Phase 3 clinical trials to evaluate its potential to treat a range of complement-driven diseases, such as paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder, and geographic atrophy (GA), an advanced form of age-related macular degeneration of the eye (AMD).

Complement also plays an important role in driving the anemia associated with CAD. In CAD, red blood cells (RBCs) are heavily coated with antibodies that lead to the body’s immune system destroying healthy cells. This destruction of healthy cells by the immune system results from activation of the complement cascade.  

In 2019, Apellis presented the preliminary results of our Phase 2 PLAUDIT Study evaluating pegcetacoplan in patients with CAD and autoimmune hemolytic anemia (AIHA). Last week, we shared that we have entered a new collaboration with Swedish Orphan Biovitrum AB (publ) (“Sobi”), a global leader in hematology and rare diseases, for pegcetacoplan. Apellis and Sobi are committed to advancing global development of pegcetacoplan. This collaboration agreement strengthens our commitment to the CAD community as it allows us to accelerate the advancement of pegcetacoplan with the goal of bringing our targeted C3 therapy to individuals living with CAD as quickly as possible. 

Along with the collaboration, it was announced that Sobi plans to initiate a Phase 3 clinical trial of pegcetacoplan for CAD. As planning advances on this pivotal trial, we want to make sure we share information about the program in a timely manner. Our communication today is the first step in keeping that commitment. 

We will partner closely with the CAD community to listen and learn from you and keep you up to date on the details of this program as they become available. 

If you have any questions, please reach out to patientadvocacy@apellis.com.  

Thank you for your interest and your support.

Sincerely,

The Apellis and Sobi Team