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The 16% selloff yesterday in uniQure shares after the FDA placed a clinical hold on its Phase III trial of AAV5-based gene therapy EtranaDez in hemophilia B creates a good entry point, Chardan analyst Gbola Amusa tells investors in a research note. The clinical hold relates to a "possibly related" serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient treated with EtranaDez in October 2019, the analyst points out. Amusa adds that uniQure's deal with CSL Behring has no opt-out based on the clinical hold, the patient was "extremely high risk," and that the company's guidance for a Biologics License Application filing is unchanged for the second half of 2021. Amusa keeps a Buy rating on uniQure with an $85 price target.

After uniQure announced its Phase 3 HOPE-B trial for EtranaDez was placed on clinical hold by the FDA after a safety report of a preliminary diagnosis of hepatocellular carcinoma, or HCC, in one patient, Jefferies analyst Suji Jeong noted that no other cases of liver cancer have been reported in uniQure's clinical and animal studies, or in other AAV gene therapy trials. Given those facts, and the fact that the patient already carried multiple risk factors for liver cancer, Jeong views it as unlikely that EtranaDez increases HCC risk in hemophilia B population, the analyst tells investors. Joeng maintains a Buy rating on uniQure with a $62 price target.

RBC Capital analyst Luca Issi lowered the firm's price target on uniQure to $53 from $61 and keeps an Outperform rating on the shares after EtranaDez was put on clinical hold due to a single case of suspected hepatocellular carcinoma. Issi believes this tilts the risk/benefit profile less favorably and increases regulatory risk, though thinks the CSL deal is unlikely at risk. The analyst is bullish on the Huntington's program going into 2021.

H.C. Wainwright analyst Patrick Trucchio lowered the firm's price target on uniQure to $80 from $85 and reiterates a Buy rating on the shares after the company announced that its hemophilia B program has been placed on clinical hold by the FDA. The analyst notes that patient dosing in each of uniQure's three hemophilia B gene therapy trials is complete so the existing timelines for HOPE-B top-line data in the second half of 2021 and a potential Biologics License Application submission remain intact.

Stifel analyst Paul Matteis acknowledges that uniQure's announcement of AMT-061 clinical hold is "obviously negative," but notes that at this stage "it's simply less clear what the actual implications will be." The analyst believes "a lot is at stake," including the regulatory prospects of AMT-061, AMT-061 commercial potential, and cash flows via the CSL deal. While the stock deserves to be down some, post-deal close, uniQure should have almost $700M in cash to fund Huntington's and the rest of its pipeline, he contends. Matteis has a Buy rating on the shares.

H.C. Wainwright analyst Patrick Trucchio raised the firm's price target on uniQure to $85 from $80 and reiterates a Buy rating on the shares. EtranaDez appears well positioned to lead in hemophilia B, Trucchio tells investors in a research note.

Jefferies analyst Suji Jeong raised the firm's price target on uniQure to $62 from $55 and keeps a Buy rating on the shares after the company shared "positive" data at the ASH meeting for EtranaDez in hemophilia B, which the analyst said support durable efficacy and safety. The Phase 3 topline data supports estimated U.S. EU launches of EtranaDez in 2022/2023, said Jeong.