On Wednesday, Goldman Sachs maintained its Neutral rating on shares of uniQure BV (NASDAQ:QURE), with a steady price target of $20.00. The firm's stance comes as uniQure announced a regulatory advancement for its gene therapy, AMT-130, for Huntington's disease.
The stock has shown remarkable momentum, with InvestingPro data revealing a 143% surge in the past week alone, reaching $15.30 from its previous $7.29. The therapy has received guidance from the FDA on an accelerated approval pathway following a Type B meeting.
The development is significant as it allows the company to use data from ongoing Phase 1/2 studies and natural history as an external control. The FDA has reviewed two-year data from 24 patients, which will be used alongside the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as a clinical endpoint.
The FDA's review and the agreed-upon accelerated pathway have increased the likelihood of AMT-130's success, according to the firm. The clarity on the regulatory path provided by the FDA negates the need for additional trials. This development has caught analysts' attention, with InvestingPro showing six analysts revising their earnings estimates upward for the upcoming period.
The stock currently trades above its Fair Value, reflecting market optimism about these developments. uniQure is scheduled to discuss the statistical analysis plan and Chemistry, Manufacturing, and Controls (CMC) requirements with the FDA in the first half of 2025. The manufacturing process for AMT-130 is considered to be de-risked, as it is highly comparable to Hemgenix and takes place in the same facility.
In mid-2025, uniQure is expected to release follow-up data, including three-year outcomes from 21 patients. Success is anticipated to mirror the trends seen in previous updates, such as dose-dependent slowing of disease progression measured by the cUHDRS and stable cerebrospinal fluid neurofilament light chain levels.
The company's financial position shows mixed signals. While the current ratio of 6.51 indicates strong short-term liquidity, InvestingPro data suggests the company is quickly burning through cash. With a market capitalization of $733.58 million, the company operates with a moderate debt level and maintains liquid assets that exceed short-term obligations.
The firm also noted a recent licensing deal within the sector, which involves Novartis (SIX:NOVN) and PTC Therapeutics (NASDAQ:PTCT), acknowledging the competitive landscape. In addition to AMT-130, uniQure's growth potential is being monitored through its early-stage gene therapy pipeline.
Expectations are set for initial Phase 1/2 data from AMT-191 in Fabry disease, AMT-162 in SOD1 ALS, and AMT-260 in refractory mesial temporal lobe epilepsy, which are all slated for release in 2025.
In other recent news, uniQure N.V. has made significant strides with its gene therapy product, AMT-130, targeted for Huntington's Disease treatment. The company has reached an agreement with the U.S. Food and Drug Administration (FDA) for an accelerated approval submission of AMT-130. Raymond (NS:RYMD) James recently upgraded uniQure's stock from Outperform to Strong Buy following these developments.
Stifel, a financial services company, also expressed a positive outlook for uniQure, indicating the FDA alignment as a significant win for the biotechnology firm. Furthermore, uniQure has initiated a Phase I/II clinical trial for its investigational treatment AMT-162, aimed at addressing ALS caused by SOD1 mutations.
These recent developments reflect uniQure's progress in gene therapy, particularly with AMT-130 for Huntington's disease and AMT-162 for ALS. Analysts from firms such as Raymond James, H.C. Wainwright, and Stifel have maintained a positive rating on the company, indicating a favorable view of these recent developments.
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