Akari Therapeutics, Plc (Nasdaq: AKTX) has made exciting progress in its development of nomacopan, an advanced therapy for treating hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA). The company is on track to open enrollment for its registrational Phase 3 study in pediatric HSCT-TMA this year, with data expected to be supportive of a potential regulatory filing and approval.
Akari is also actively planning to launch a Phase 3 double-blind placebo-controlled clinical trial of nomacopan in adult HSCT-TMA, with enrollment set to begin in 2024. With these proactive steps, Akari is on the forefront of developing cutting-edge treatments for autoimmune and inflammatory diseases.
At Akari, we are passionately striving to bring an approved treatment to those suffering from a devastating condition. Our goal is to positively shape the standard of care for both children and adults facing this difficult condition, and we are committed to making a meaningful impact.
Hematopoietic stem cell transplants (HSCTs) are used to treat more than 22,000 people each year in the United States, but unfortunately, 36% of these recipients experience serious complications in the form of transplant-related thrombotic microangiopathy (HSCT-TMA). Despite its prevalence, there is no approved treatment for HSCT-TMA and those with severe cases have a mortality rate of 80%.
Notably, approximately 300 to 400 of these cases are children, with ten times the number of adult patients experiencing HSCT-TMA. Such statistics are a tragic reminder of the importance of continued research and the development of treatments that can help those affected by this life-threatening condition.
Recently published consensus guidelines by an international panel of experts are providing a platform for earlier diagnosis of transplant-associated thrombotic microangiopathies (TMAs), a condition associated with significant mortality.
By harmonising diagnostic criteria, these guidelines are aiding the development of the Akari HSCT-TMA Phase 3 clinical trials design – allowing earlier and more accurate diagnosis of high-risk patients. This is a significant advancement in providing life-saving care to those affected.
Proper screening and early diagnosis of Transplant-Associated Microangiopathy (TMA) can be a lifesaver, potentially preventing the irreversible and fatal multi-organ dysfunction that may otherwise ensue. This critical step is widely recognized by clinicians and researchers alike, according to Sonata Jodele, M.D. of Cincinnati Children’s Hospital Medical Center.
Akari is pushing the boundaries of medical science with their Phase 3 open-label, single-arm study of nomacopan in pediatric HSCT-TMA, followed by their imminent plans to move into a Phase 3 double-blind, placebo-controlled study of nomacopan in adult HSCT-TMA in 2023 and 2024.
Their breakthrough progress in this research is made possible thanks to Akari’s PK/PD data (in adults) and the USFDA’s agreement to use PK/PD modelling and predictions about pediatric dosing. This milestone is set to open up a new world of medical care and possibilities in the treatment of HSCT-TMA.
Harmonizing adult and pediatric studies, ensuring that their study designs, such as inclusion criteria, are comparable will bring great clarity to regulators when reviewing applications for approval. Furthermore, having unified clinical trial data will be a great advantage to future researchers. This is why John Neylan, M.D., Chief Medical Officer of Akari, stresses the importance of harmonization.
The FDA has awarded a range of designations to nomacopan, facilitating its potential use in pediatric HSCT-TMA. Not only does the Rare Pediatric Disease Designation qualify sponsors for Priority Review Vouchers, but its recent positive opinion from the EMA for orphan drug designation is also likely to pave the way for its insertion in hematopoietic stem cell transplantations. The prospects for nomacopan look promising indeed!
About Akari Therapeutics
Akari Therapeutics, Plc (Nasdaq: AKTX) is a biotechnology game-changer revolutionizing the way autoimmune and inflammatory diseases are treated. The company’s lead asset, investigational nomacopan, is a revolutionary new bispecific recombinant inhibitor with the potential to control both complement C5 activation and leukotriene B4 (LTB4) activity.
Akari is currently conducting a Phase 3 clinical trial on nomacopan as a new treatment for severe pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA) and has been awarded several key designations from the FDA including Orphan Drug and Rare Pediatric Disease.
Additionally, the company is trialing nomacopan for the treatment of adult HSCT-TMA and researching the effects of long-acting PAS-nomacopan for geographic atrophy (GA). Akari is pioneering the future of autoimmune and inflammatory disease treatment.