uniQure N.V.: A Pathway To Approval

Now is the time at uniQure N.V.

This will be a “best ideas only” bio substack providing actionable, highly digestible and high conviction value to investors focused on quality not quantity. Disclaimer: Nothing in this article (or substack) is considered investment advice and is solely the opinion of the author.

LONG
Ticker: QURE
Duration: +12 months
Target: +$55
Likely Outcome: Buyout
Value Driver: The Designation Golden Trifecta for AMT-130 (AA, Orphan and Breakthrough Designations)

uniQure N.V. (QURE) is one of the more compelling names in bio-land, primarily driven by the significant potential of its Huntington's disease (HD) program, which appears to be materially de-risked following constructive interactions with regulatory authorities.

The company is advancing towards key clinical milestones for its HD program. Three-year follow-up data from the Phase I/II study is anticipated in mid-2025 (June/July or Q3 ). uniQure has expressed confidence in its ability to file a Biologics License Application (BLA) shortly after this data release, with BLA-readiness activities already underway. The BLA submission package will have data from 27, 24, 8, and 1 patients with 2, 3, 4, and 5 years of follow-up, respectively, though the primary analysis will focus on the three-year population.

Huntington's Disease Program: Path to Approval

uniQure's therapeutic candidate for HD, AMT-130, is designed as a one-time administered gene therapy. Its mechanism of action involves targeting the disease-causing exon 1 of the huntingtin gene, aiming to lower the production of the mutant huntingtin protein. The therapy is delivered directly to the brain via intrastriatal administration (via Clearpoint Neuro, a company which I happen to be a fan of), which, while somewhat invasive, is intended to ensure that the therapeutic agent reaches the striatum, a key brain region affected early and severely in HD. This targeted approach, addressing the genetic root cause of the disease, represents a promising strategy for modifying its progression which is the key factor that matters.