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Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.
Modulating amyloid proteins is a validated target in systemic amyloidosis. Therapies recently approved in ATTR amyloidosis have proven that reducing or stabilizing the pre-cursor protein leads to slowing disease progression. In AL amyloidosis, off-label plasma cell targeting agents including chemotherapy and Autologous Stem Cell Transplant, which both reduce the precursor protein, have also been shown to improve clinical outcomes. Reducing or stabilizing the pre-cursor protein in AL and ATTR amyloidosis results in the reduction of formation of new amyloid, but does not directly address already deposited toxic amyloid.
There is a substantial unmet need to improve the diagnosis of amyloidosis in patients. The majority of systemic amyloidosis patients see more than four specialists and take more than 18 months to get an accurate diagnosis, with 25% of patients taking more than five years to get a correct diagnosis. Unfortunately, due to this prolonged process, a significant number of patients die without a diagnosis. Of the patients that do receive a diagnosis, there are a number of diagnostic procedures required with clear challenges in getting an early and accurate diagnosis. A misdiagnosis can be deadly and delayed diagnosis leads to poorer patient outcomes.
A prompt diagnosis is urgently needed, but for most patients, it takes over 18 months from first symptoms to confirmed diagnosis. For 25% of patients, it takes more than five years.
A significant portion of the long diagnostic timeframes is owed to an initial delay in suspicion of amyloidosis. Patients present with non-specific and seemingly unrelated cluster of symptoms such as fatigue, dyspnea, edema or GI issues. Most patients see more than four specialists before reaching a diagnosis.
The Attralus PET/CT imaging agent AT-01 has
the potential to provide a rapid and accurate diagnosis of
systemic amyloidosis.
Even after amyloid is suspected, the diagnostic pathway can be arduous and require numerous tests, including an invasive biopsy for most patients. Current diagnostic tests only reveal an incomplete picture of the patient’s disease, often with amyloid
only confirmed in one organ.
AT-01 has the potential to comprehensively measure amyloid deposition in organs throughout the whole body, to differentiate between subtypes of amyloidosis, and to monitor disease progression and treatment response.
Attralus is developing novel pan-amyloid targeting agents to directly bind and remove toxic amyloid fibrils from organs and tissues throughout the body, with the potential to reverse disease pathology – going beyond current treatments that reduce the formation of amyloid fibrils and slow disease progression but do no remove amyloid from the body.
When available, current treatment options for AL and ATTR do not directly address already deposited toxic amyloid in organs and tissues. These treatments have limited impact of later stage patients, which represent ~50% of the population.
Attralus’s therapeutics target motifs that are universally and ubiquitously presented on all amyloid fibrils. This pan-amyloid binding profile allows Attralus’s therapeutics to target multiple types of amyloid fibrils including AL, ATTR, and ALECT2.
While there are treatments in two types of
amyloidosis (ATTR & AL), there remains a very high
unmet need with poor median survival & quality of life.
Not only do we have the potential to develop therapeutics to treat patients with any form of systemic amyloidosis, we also can potentially treat those with late-stage disease for whom current therapies have not shown significant impact.
