TrialsFor those who have no interest in doing a lot of reading, here is what you need to know about the two trials:
STEM CELL THERAPEUTICS CORP.
Disease Indications
Acute Myeloid Leukemia (AML)
AML is the most common type of acute leukemia in adults, with approximately 13,000 new
cases diagnosed each year in the United States. The majority of AML patients receive induction
chemotherapy. In patients <60 years of age, remission rates of up to 75% can be achieved, and
patients with good-risk or standard-risk cytogenetics will typically receive post-remission
therapy with high dose cytarabine. However, relapses are common, and the majority of patients
will die from their disease. AML in elderly patients (>60 years of age) is notoriously difficult to
treat, with five-year survival rates reportedly of less than 10%. There is ample evidence that
AML is sustained by CSCs (known as leukemic stem cells), and the failure to eradicate these
CSCs using conventional chemotherapy is thought to be responsible for disease relapse.
Interstitial Cystitis (IC)
Interstitial cystitis is a chronic bladder disease characterized by pain, urinary urgency/frequency
and nocturia. Once considered a relatively rare condition, IC is now believed to affect up to eight
million women and two million men in the US alone. Current therapies are restricted to two
FDA-approved agents and a repertoire of older off-label drugs which provide modest relief at
best. Patients continue to suffer from debilitating symptoms that impact their physical and
emotional health. Although the etiology of IC has not been fully elucidated, there are strong data
indicating that increased bladder permeability may be an important contributing factor.
Competition
Acute Myeloid Leukemia (AML)
While there is significant clinical activity in AML, there are a limited number of agents in
development targeting AML leukemic stem cells. These are largely focused on cell surface
antigens such as CD123 and CD44, and thus are distinct from tigecycline, which targets the
mitochondrial ribosome. TTI-621 is in direct competition with a CD47 blocking antibody, which
is currently in preclinical development. Although both agents target the same pathway, they are
structurally distinct entities with unique properties.
Interstitial Cystitis (IC)
Despite the inadequacies of current IC therapies, there is a paucity of new treatments in the drug
development pipeline, with only a handful of agents currently in clinical testing. Much of the
work continues to be focused on the development of analgesics or bladder coating agents. These
approaches are palliative in nature, and do not address the proposed underlying cause of disease.
In contrast, TTI-1612 targets the root cause of IC: dysfunction of the bladder urothelium caused
by low levels of HB-EGF.