AML digital therapeutic lands Breakthrough Device Designation from FDA

Digital therapies developer has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Device Designation for its BNT200 device. BNT200 is a prescription-only digital therapeutic intended to treat anxiety and depressive symptoms in adults with acute myeloid leukemia (AML) who are hospitalized for high-intensity induction chemotherapy.

AML is a rare variety of bone marrow and blood cancer; it reportedly accounts for about 1% of all cancers. An estimated 20,240 new cases were reported in 2021, with about 11,400 deaths.

“For the thousands of patients diagnosed with AML each year, managing and coping with disease symptoms can be especially difficult​,” said Areej El-Jawahri, Massachusetts General Hospital and principal investigator of the team leading the study evaluating the device. “The advancement of innovative therapeutic options for the treatment of the associated distress that often follows a diagnosis is promising for patients and addresses an unmet need.”​

Michelle Rubin-Onur, Ph.D., regulatory lead with Blue Note Therapeutics took a few moments to discuss the therapy with Outsourcing-Pharma, how it works, and what the designation might mean for AML patients.

OSP: Please tell us a bit about AML, and how anxiety and depressive symptoms impact them.​

MROP: AML is a type of bone marrow and blood cancer that often presents suddenly and progresses rapidly. Without treatment, the median survival is six weeks; therefore, immediate, intensive treatment, including extensive hospital stays and isolation, may be required.

The physical and psychological burden of AML, including the setting, treatment regimen, and side effects, is taxing and patients frequently report a significant disruption in life as they often require inpatient treatments and/or hospitalization. This makes patients living with AML especially susceptible to anxiety, depression, and other types of distress related to cancer from diagnosis to treatment, and into survivorship. 

OSP: Could you please tell us a bit about how Blue Note created BNT200? How does this device work?​

MRO: BNT200 is based on an existing, cancer distress intervention developed by leading experts at Massachusetts General Hospital to treat the anxiety and depressive symptoms created by the unique psychological stressors specific to adult patients with AML undergoing high-intensity induction chemotherapy (HIC). Blue Note is translating this proven face-to-face intervention into a self-directed digital format, developed as a software as a medical device (SaMD).

This first-of-its-kind digital therapy is intended to be used in the inpatient setting during the four to six weeks associated with HIC treatment. The content can be accessed on-demand via a mobile device and is synchronized with the HIC treatment regimen to blunt the unique psychological stressors endured by this subset of patients with AML. It is, therefore, not applicable to patients with AML undergoing outpatient treatment.

Similar to prescription medicines, BNT200 must demonstrate safety and efficacy in randomized clinical trials prior to approval by the FDA; our application for Breakthrough Device Designation was supported by positive usability and preliminary efficacy findings.

OSP: What does the breakthrough designation mean for the future of the device?​

MRO: With this designation status, the FDA is committing to prioritize the review and approval process for BNT200. This is especially exciting as it holds the promise of expediting our ability to alleviate anxiety and depression in AML patients hospitalized for chemotherapy. BNT200 is also a first-of-its-kind in digital therapeutics to be used in acute cancer, and in the inpatient setting, reaching a subset of patients that are especially susceptible to cancer-related distress. 

OSP: Does Blue Note have any other projects in the works, interesting collaborations going on, or other news you’d like to share?​

MRO: Blue Note continues to license best-in-class interventions from around the world that can be translated into a digital format to serve more patients, more conveniently, and with a more personalized touch. We continue to deliver - not as a company per se - but as a collaborative group with leading cancer centers, researchers, and an increasing cadre of people living with cancer who help us get it right and pay it forward to those who will someday be diagnosed.

This is Blue Note’s second Breakthrough Device Designation received by the FDA which reinforces our commitment to ease the burden of cancer, and to do so as quickly as possible. The first Breakthrough Designation was given to BNT001 in June 2021.

As well documented from clinical studies, patients living with cancer face distress, such as anxiety and depression, following a diagnosis and treatment. However, there are limitations of current face-to-face interventions in treating these symptoms. Recognizing this unmet need, Blue Note committed to the development of BNT001, translating a validated cancer distress intervention into a digital, self-directed format.

Timing for the development of BNT001 coincided with the emergence of the COVID-19 pandemic, which for many living with cancer further compounded their symptoms of distress and disrupted care. Through the FDA public health emergency (PHE), Blue Note was, fortunately, able to reach patients who may benefit from BNT001 quicker.

Blue Note has a pipeline of four product candidates, singularly dedicated to addressing symptoms of cancer yet diversified to work along the care continuum - from diagnosis through to survivorship. We are conducting several registrational clinical trials and studies to demonstrate the ability of our digital therapies to help people living with cancer cope with cancer-related distress.