uniQure (NASDAQ: QURE) Announces Third Quarter Financial Results and Recent Progress

On November 5, 2024, leading gene therapy company uniQure N.V. issued a press release unveiling its financial results for the third quarter of 2024 and highlighting recent advancements across its operations. Among the key highlights shared in the press release, uniQure reported strong progress on multiple fronts, including developments in its pipeline of clinical gene therapy programs and organizational restructuring aimed at reducing operating expenses and enhancing shareholder value.

The company disclosed that a Type B meeting is scheduled with the FDA in the fourth quarter of 2024 to initiate talks regarding a potential expedited clinical development pathway for AMT-130, a gene therapy candidate for Huntington’s disease. Positive interim data from the Phase I/II trial of AMT-130 showcased significant slowing of Huntington’s disease progression and reductions in crucial biomarkers of neurodegeneration.

Apart from AMT-130, uniQure has initiated patient dosing in new Phase I/II studies for AMT-162 in SOD1-ALS and AMT-191 in Fabry disease. Additionally, the company enrolled the first patient in the observational stage of the Phase I/II study of AMT-260 in mesial temporal lobe epilepsy.

uniQure highlighted its efforts in capital preservation, including the completion of the sale of its Lexington manufacturing facility and an organizational restructuring anticipated to significantly reduce both operating expenses and annual cash burn. These strategic decisions have extended the company’s cash runway through the end of 2027 and are expected to drive operational efficiencies.

Financially, as of September 30, 2024, uniQure reported a cash position of approximately $435 million following the retirement of $50 million of debt, ensuring cash sufficiency to fund operations through the end of 2027.

Moving forward, uniQure emphasized its near-term focus on collaborating with the FDA to potentially secure an accelerated pathway for AMT-130 and advancing its clinical pipeline. The company aims to provide further updates on its progress in due course.

Investors can look forward to uniQure’s participation in upcoming events like the Guggenheim Healthcare Talks – Global Healthcare Conference on November 12th in Boston, MA, and the Stifel 2024 Healthcare Conference on November 18th in New York, NY.

In conclusion, uniQure’s recent financial results and operational updates underscore its commitment to pioneering gene therapy solutions for severe medical conditions, positioning the company at the forefront of genomic medicine innovation.

-END-

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read uniQure’s 8K filing here.

About uniQure

uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases. It offers HEMGENIX that has completed Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also develops AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease.