uniQure shares hold Buy rating on promising trial data By Investing.com

On Tuesday, uniQure BV (NASDAQ:) sustained its Buy rating by an analyst at TD Cowen, following the release of additional interim data from its Phase I/II trials. The data demonstrated that uniQure’s AMT-130, a gene therapy for Huntington’s disease, showed a reduction in disease progression at 24 months when compared to an external natural history control.

The findings indicated a statistically significant reduction in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) for the high dose group and notable results for the low dose group.

The high dose group saw a decrease of 0.2 in the cUHDRS with a p-value of 0.007, suggesting a strong statistical significance. The low dose group experienced a reduction of 0.7, though with a p-value of 0.21, indicating less statistical significance.

The control group’s cUHDRS score decreased by 1.0. Furthermore, the data showed significant statistics on Neurofilament light chain (NfL) at 24 months, a biomarker associated with neuronal damage and neurodegeneration.

The analyst’s commentary highlighted the importance of these results and the company’s plan to engage with the FDA in a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting during the second half of the year. The meeting aims to discuss expedited pathways for AMT-130’s development and regulatory approval.

uniQure’s continued progress in its clinical trials and the potential to expedite AMT-130’s pathway through regulatory discussions are pivotal for the company’s trajectory. The maintained Buy rating reflects confidence in uniQure’s direction and the therapeutic potential of its gene therapy candidate.

In other recent news, uniQure N.V. revealed promising interim data from Phase I/II trials of its potential treatment for Huntington’s disease, AMT-130. The trials indicate a significant slowing in disease progression and a reduction in a neurodegeneration marker in patients’ cerebrospinal fluid.

This has led to discussions with the FDA about potentially expedited clinical development. In related news, Genezen has acquired uniQure’s commercial gene therapy operations in Lexington, MA, a strategic move to enhance its global gene therapy development capabilities.

In other developments, uniQure’s shareholders approved an expansion of the company’s share incentive plan, reappointed board members, and adopted the 2023 statutory annual accounts.

Stifel, an investment firm, maintained a Buy rating on uniQure following the FDA’s granting of an RMAT designation to AMT-130. This designation could potentially expedite the development and approval process for the gene therapy candidate.

Lastly, Paula Soteropoulos, a non-executive director of uniQure, has decided not to stand for re-election at the next annual meeting. Her departure is not due to any disagreements with uniQure’s management, operations, or practices. The board has not yet announced a successor. These are all recent developments in the ongoing evolution of uniQure N.V.

InvestingPro Insights

In light of uniQure’s (NASDAQ:QURE) recent clinical trial developments, the InvestingPro data presents a nuanced picture of the company’s financial health. With a market capitalization of $309.02 million and a striking revenue decline of -82.73% in the last twelve months as of Q1 2024, investors may exercise caution.

Despite a quarterly revenue growth of 59.34% in Q1 2024, uniQure’s gross profit margin remains deeply negative at -952.89%, underscoring challenges in profitability. Moreover, the company’s stock performance has been underwhelming, with a one-month price total return of -29.08% and a significant year-to-date price total return drop of -44.17%.

InvestingPro Tips suggest that analysts are not expecting uniQure to be profitable this year, and the company has been quickly burning through cash. Still, it is worth noting that uniQure’s liquid assets exceed its short-term obligations, which might provide some financial flexibility in the near term. The stock’s RSI indicates it is in oversold territory, which could be of interest to value investors looking for entry points.

For those seeking a deeper dive into uniQure’s financials and future outlook, InvestingPro offers additional insights and tips. Use coupon code PRONEWS24 to get up to 10% off a yearly Pro and a yearly or biyearly Pro+ subscription, and discover the 10 additional tips listed on InvestingPro that could inform your investment strategy.

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