Bluebird suspends gene therapy trials; Cortexyme tripped up by FDA hold; Guardant Health launches surveillance blood test; Enhertu scores approval

Posted on Feb 18, 2021 by Delveinsight

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Bluebird suspends gene therapy trials after two sickle cell patients develop cancer

Bluebird bio has stopped two clinical trials of its sickle cell disease gene therapy after participants developed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). During the pause, bluebird will assess whether the BB305 lentiviral vector is linked to AML and MDS cases. 

The AML case involves a patient who was received LentiGlobin gene therapy for sickle cell disease in a phase 1/2 clinical trial more than five years ago. It is currently unclear whether the development of AML is linked to the BB305 lentiviral vector used in bluebird’s gene therapy. 

Bluebird also lately realized a case of MDS in a participant in the phase 1/2 clinical trial. The patient is the second recipient of gene therapy to develop bone marrow disease. Bluebird concluded the 2018 case was an outcome of the chemotherapy the patient received to prepare them to receive the gene therapy. That patient had a classic MDS mutation and no vector in the blast cells.

Bluebird CEO Nick Leschly put forward various potential explanations for the new cases of AML and MDS. Spontaneous genetic mutations unrelated to the gene therapy could account for the cases, as could preconditioning chemotherapy or other aspects of the patients’ conditions and treatment. However, bluebird cannot currently rule out a causal role for BB305. 

Cortexyme tripped up by FDA hold over liver toxicity

Pfizer, Verily-backed Alzheimer’s biotech Cortexyme, has hit a snag. The FDA has slammed a partial clinical hold on its key Alzheimer’s drug atuzaginstat (called COR388), hitting specifically the open-label extension part of its ongoing phase 2/3 study, known as the GAIN Trial.

Under the hold, the company will not be able to enroll new patients in the open-label extension segment of the test, and lately, enrolled participants will be discontinued as per the biotech announcement. 

The 643 patients in the fully enrolled double-blind, placebo-controlled randomized phase of the GAIN trial will, nevertheless, continue to receive the study drug at their assigned dose, with top-line results anticipated by the fourth quarter.

The partial clinical hold was commenced following the review of hepatic adverse events in the atuzaginstat trial by the FDA, as confirmed by Cortexyme. It further included that these were reversible and without any known long-term severe effects for the participants.

Casey Lynch, Cortexyme’s chief executive, co-founder, said in a statement that the highest priority of Cortexyme is the safety of study participants. The ongoing double-blind GAIN Trial in mild to moderate Alzheimer’s disease will proffer a critical evaluation of efficacy and safety in treating this devastating disease.

Guardant Health launches surveillance blood test for detection of colorectal cancer recurrence

Guardant Health has launched a simple blood test to detect patients after undergoing surgery for colorectal cancer to detect any early signs that tumor cells remain or to catch the disease before it returns.

The Guardant Reveal test, hoped to turn around results in one week, is the company’s first liquid biopsy to help manage early-stage cancer treatment, and the company contemplates making it available for other cancer types in the future.

Guardant CEO Helmy Eltoukhy said that colorectal cancer is the second leading cause of cancer death in the United States and around 10% to 30% of early-stage patients recur. Hence oncologists must have better tools to manage these patients by swiftly detecting those high-risk patients who may benefit from adjuvant therapy after surgery and identifying recurrence earlier as part of regular surveillance.

The test separates tumor DNA fragments floating in the bloodstream and can help identify recurring cancers months earlier than the current standard imaging procedures and blood tests for carcinoembryonic antigen levels as per Guardant.

By sequencing and matching the DNA found to the tumor and assessing the genetic material’s larger structure for the revealing signs of cancer, the Guardant Reveal test can offer a sensitivity of 91%, the company said.

The diagnostic also lets patients be screened multiple times in the months following surgery, compared to invasive tissue removal and biopsy procedure.

AstraZeneca, Daiichi Sankyo’s Enhertu scores approval in the United Kingdom 

AstraZeneca (AZ) and Daiichi Sankyo’s antibody-drug conjugate (ADC) Enhertu has scored a conditional marketing authorization in the U.K. for individual breast cancer patients.

The conditional marketing authorization covers Enhertu (trastuzumab deruxtecan) as a monotherapy for treating adults with unresectable or metastatic HER2-positive breast cancer who have got two or more prior anti-HER2 based regimens.

The U.K. authorization is based on results from the Phase II DESTINY-Breast01 trial, in which patients treated with Enhertu showed a confirmed objective response rate (ORR) of 61.4%, as well as a 6.5% complete response rate and a 54.9% partial response rate.

Moreover, after a median follow-up of 20.5 months, the median duration of response (DoR) was 20.8 months.

Arun Krishna, head of oncology, AstraZeneca U.K., said in a statement that the DESTINY-Breast01 trial showed a duration of response not previously observed in patients after progression on first- and second-line treatment.

He added that Enhertu is an important new treatment option for patients at this stage of care and will shift clinical discussions towards a focus on targeted treatment. This is the first new cancer medicine to be authorized by the MHRA in 2021, and its focus now is on securing access for NHS patients as quickly as possible.

The U.K.’s National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) is recently appraising Enhertu, with NHS access decisions anticipated later in 2021.

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