Uniqure Stock Price, News & Analysis

uniQure N.V. (NASDAQ: QURE) announced the granting of equity awards to 31 employees to incentivize their employment, as per Nasdaq Listing Rule 5635(c)(4). The awards include 63,300 restricted share units (RSUs) and options for 57,500 ordinary shares, with an exercise price of $20.06, reflecting the closing price on the grant date of February 23, 2023. Options will vest over four years, with one-quarter vesting after the first year and the remainder in quarterly installments. RSUs will vest over three years. This initiative is part of uniQure's strategy to attract talent as it advances its gene therapy pipeline targeting severe diseases.

uniQure N.V. (NASDAQ: QURE) announced its participation in various investor and scientific conferences in March 2023. Key events include the Cowen Annual Healthcare Conference from March 6-8 in Boston, where CEO Matt Kapusta will engage in one-on-one meetings and a panel discussion.

Other conferences include the Bioprocessing Summit Europe on March 15 in Barcelona, the Huntington's Disease Youth Organization Congress from March 17-19 in Glasgow, and the MDA Conference from March 19-22 in Austin. The company aims to showcase its advancements in gene therapy, particularly for severe medical conditions like hemophilia B.

uniQure N.V. (NASDAQ: QURE) announced significant milestones, including U.S. and European marketing approvals for HEMGENIX®, the first gene therapy for hemophilia B. The company is advancing AMT-130 for Huntington’s disease, with updates expected in Q2 2023. Plans for new clinical trials for AMT-260 (temporal lobe epilepsy) and AMT-162 (SOD1 ALS) are set for the second half of 2023. Financial results show a revenue decline to $106.5 million in 2022, down from $524 million in 2021, with a net loss of $126.8 million. The company holds $392.8 million in cash, expected to fund operations into 2025, contingent on milestone payments for HEMGENIX®.

uniQure (NASDAQ: QURE) announced significant results from the pivotal HOPE-B clinical study of its gene therapy HEMGENIX^® for hemophilia B, published in the New England Journal of Medicine. The study demonstrated HEMGENIX^® significantly reduced the annualized bleeding rate (ABR) by 71% for spontaneous bleeds and 78% for joint bleeds. After treatment, 96.3% of patients discontinued factor IX prophylaxis. The therapy maintains elevated factor IX levels over 18 months without serious adverse events related to treatment reported. HEMGENIX^® is approved in the U.S. and EU, representing a transformative development for hemophilia B patients.

CSL announced pivotal HOPE-B trial results for HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy approved for adult hemophilia B patients, showing significant long-term efficacy and safety. Published in NEJM, the trial demonstrated a 71% reduction in spontaneous bleeding episodes and a 78% reduction in joint bleeding episodes. Post-treatment, 96.3% of patients discontinued factor IX prophylaxis after 18 months, with infusions dropping from 72.5 to 2.5 annually. HEMGENIX® is now approved in the US and EU, showcasing CSL's commitment to innovative treatments for rare diseases.

uniQure N.V. (NASDAQ: QURE) announced that its partner CSL has received conditional marketing authorization from the European Commission for HEMGENIX® (etranacogene dezaparvovec), the first gene therapy for severe and moderately severe hemophilia B in the European Union. This approval is significant as it marks a major milestone in genomic medicine, allowing eligible patients to produce their own factor IX, thereby reducing annual bleeding rates and eliminating the need for routine factor IX infusions. The HOPE-B clinical trial showed a 64% reduction in adjusted annualized bleed rates, with 96% of patients discontinuing prophylaxis. uniQure stands to receive significant milestone payments and royalties as part of its agreement with CSL.

The European Commission has granted conditional marketing authorization for HEMGENIX® (etranacogene dezaparvovec), marking it as the first one-time gene therapy for treating severe hemophilia B in adults. The therapy showed a 64% reduction in the adjusted annualized bleed rate in patients during clinical trials. Following the pivotal HOPE-B trial, 96% of patients discontinued routine Factor IX prophylaxis, with a 97% reduction in Factor IX consumption. The approval reflects CSL's commitment to innovation and improving the quality of life for hemophilia B patients.