Stifel - PT $86
Heme B Dosing Completed; Huntington's Dosing Delayed ButPatients Screened/Identified
We are reiterating our Buy rating on QURE - the company announced this morning that AMT-061 dosing has been completed in the Heme B study, keeping top-line data on track for late 2020. The company is taking the necessary precautions as it relates to patient follow-up within the coronavirus pandemic; it noted that it is following FDA guidelines. In the meantime, somewhat unsurprisingly, the dosing of the first two patients in the Huntington's trial (AMT-130) is delayed - recall, this medicine is given via surgical delivery, and in the midst of a pandemic, patient appointments have been pushed back. Fortunately, QURE has been able to screen/identify patients that meet its criteria, which is encouraging given initial challenges that were associated with narrow enrollment criteria.
We are reiterating our Buy rating on QURE - the company announced this morning that AMT-061 dosing has been completed in the Heme B study, keeping top-line data on track for late 2020. The company is taking the necessary precautions as it relates to patient follow-up within the coronavirus pandemic; it noted that it is following FDA guidelines. In the meantime, somewhat unsurprisingly, the dosing of the first two patients in the Huntington's trial (AMT-130) is delayed - recall, this medicine is given via surgical delivery, and in the midst of a pandemic, patient appointments have been pushed back. Fortunately, QURE has been able to screen/identify patients that meet its criteria, which is encouraging given initial challenges that were associated with narrow enrollment criteria.
Key Points
QURE successfully enrolled the first two patients into its ph1/2 AMT-130 HD trial, though the ongoing COVID-19 situation has led to a delay in dosing. Recall that AMT-130 is administered via targeted surgical delivery that in preclinical models demonstrated mHTT knockdown both within deep brain structures and the cortex. Initial enrollment was challenging due to specific criteria (preserved brain structure volume + certain level of disease severity), so it's encouraging that after working with the FDA to modify enrollment restrictions that the company has been able to identify two qualifying patients. That said, dosing now has been delayed at the clinical center. We are unsure how this will impact timelines, though it would seem that they could potentially be on track -- QURE previously guided to a safety update later this year and biomarker/imaging data in 2021. While we don't know how the pandemic will shake out, we hope that these timelines aren't aggressive and prove meetable.
uniQure also provided an update from its HOPE-B open-label ph3 trial of AMT-061 in hemophilia B; the final patient has been dosed (n=54) and QURE reiterated expectations for top-line data by year-end 2020 and BLA submission in 2021. HOPE-B had targeted enrollment of ~50 hemophilia B patients with severe and moderately severe disease; we're pleased to see dosing of the AAV5-based AMT-061 finished, particularly in today's uncertain clinical trial environment as it relates to SARS-CoV-2. While there's still some uncertainty/challenges lingering, as uniQure needs to be able to complete follow-up visits with patients (HOPE-B's primary endpoint is F9 levels at t=26 weeks, along with secondary measures including ABR and use of F9 replacement therapy), one would hope that it's no more challenging for this study (than the average biotech trial) given that treatment is one-and-done. Moving forward, uniQure plans to work within the FDA's recent draft guidance (link here) around conducting clinical trials during the pandemic.