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Some patients with aplastic anemia treated with CK0801 achieved durable transfusion independence lasting over 3 years.
The FDA has granted orphan drug designation (ODD) to CK0801, an allogeneic T regulatory (Treg) cell therapy developed by Cellenkos Inc for the treatment of aplastic anemia.1 The ODD allows for expedited development of treatments for rare diseases;2 with an incidence of 1 to 2.3 per million in the US and a prevalence of 5000 patients, CK0801 is poised to transform treatment of patients with this hematologic condition.1
Aplastic anemia can be difficult to treat in patients who have failed previous lines of treatment. | Image Credit: © Biggy - stock.adobe.com
"Receiving ODD for CK0801 in aplastic anemia underscores the importance of bringing novel, transformative treatment options to patients suffering from this rare disease," Simrit Parmar, MD, founder of Cellenkos, said in the news release. "We aim to deliver a transformative therapeutic that can reduce the burden of blood and platelet transfusions in patients with aplastic anemia who have failed to respond to standard-of-care treatment."1
In a phase 1 dose-escalation trial (NCT03773393), CK0801 showed indications of efficacy in patients with bone marrow failure syndromes—including aplastic anemia (n=4), myelofibrosis (n=4), and hypoplastic myelodysplasia (n=1)—who had suboptimal responses to prior therapy. The treatment was administered in an outpatient setting, with starting dose levels of 1×106 (n=3), 3×106 (n=3), and 10×106 (n=3) cells per kg of ideal body weight, according to the investigators.3,4
The FDA designates certain drugs or biological products as orphan products if they are intended to diagnose, prevent, or treat conditions affecting a small patient population. Receiving orphan drug designation provides sponsors with several benefits, such as eligibility for tax credits related to clinical trial costs, a waiver of FDA application fees, and the possibility of exclusive marketing rights for up to 7 years following product approval.2
Importantly, there were no infusion reactions, grade 3 or 4 severe adverse events (AEs), and no dose-limiting toxicity in any of the 9 patients. Investigators measured responses at 12 months; by this end point, CK0801 was found to have induced objective, partial responses in 3 of the 4 patients with aplastic anemia, and in 3 of the 4 patients with myelofibrosis. Transfusion independence was achieved in 3 of 4 patients who were previously transfusion-dependent prior to CK0801. In a critical development, there were no observed increases in infections, no transformations to leukemia, and no deaths, though the investigators noted that the duration of observation was limited.4
Patients with aplastic anemia, a rare and serious condition, have bone marrow that fails to produce sufficient blood cells, which can cause severe complications, including fatigue, infection, and bleeding. The condition can develop at different speeds and levels of severity, making it difficult to monitor and diagnose. Although blood and bone marrow transplants or transfusions represent potentially curative treatment options, these can be burdensome for patients, necessitating research into novel modes of treatment such as CK0801, which could improve outcomes following prior therapies.1,5
Pharmacists play an important role in managing patients with aplastic anemia while coordinating with treatment providers and clinicians in a possible scenario of CK0801 being approved for this condition. Patients often require frequent visits to their primary care provider for monitoring and blood screening; pharmacists, with their front-facing role on a patient’s care team, can assist in counseling them on what to expect with their condition. They can also aid in recommending pertinent medicines to ameliorate some of the AEs of aplastic anemia.
In the news release, Cellenkos officials said that CK0801 was advancing towards the initiation of a registration trial that was intended to support its future regulatory approval for the treatment of transfusion-dependent aplastic anemia. The availability of a non-immunosuppressive treatment for those unresponsive to other therapies could represent a shift in the anemia space, with possible applications in other anemias and hematologic conditions.
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