uniQure(US:QURE)2023-05-09 11:20Financial Performance - Total revenues for Q1 2023 were $5.325 million, a significant increase from $1.792 million in Q1 2022, primarily driven by contract manufacturing revenues of $4.937 million[17]. - The net loss for Q1 2023 was $77.227 million, compared to a net loss of $46.678 million in Q1 2022, indicating a worsening financial performance year-over-year[17]. - Cash used in operating activities for Q1 2023 was $78.302 million, significantly higher than $25.711 million in Q1 2022[22]. - The company reported a basic and diluted net loss per ordinary share of $1.63 for Q1 2023, compared to $1.00 for Q1 2022[17]. - The accumulated deficit as of March 31, 2023, was $659.2 million, up from $581.9 million as of December 31, 2022[85]. - The company reported a net loss of $77.2 million in Q1 2023, compared to a net loss of $46.7 million in Q1 2022, representing a 65.2% increase in losses[132]. Cash and Assets - Cash and cash equivalents decreased to $153.851 million as of March 31, 2023, down from $228.012 million at the end of 2022[14]. - Total assets decreased to $631.004 million as of March 31, 2023, from $704.964 million at the end of 2022[14]. - Total shareholders' equity fell to $412.771 million as of March 31, 2023, down from $476.009 million at the end of 2022[14]. - The company had cash and cash equivalents of $153.851 million as of March 31, 2023, down from $228.012 million as of December 31, 2022[42]. - As of March 31, 2023, cash and cash equivalents totaled $318.5 million, with expectations to fund operations into 2025[125]. Research and Development - Research and development expenses rose to $60.809 million in Q1 2023, compared to $45.003 million in Q1 2022, reflecting increased investment in product development[17]. - The company anticipates increased expenses related to advancing AMT-130 into phase III clinical studies and other gene therapy programs[86]. - The company incurred $4.3 million and $2.6 million for the preclinical development of temporal lobe epilepsy in Q1 2023 and Q1 2022, respectively, reflecting a 65.4% increase[110]. - The total research and development expenses for Q1 2023 were $18.7 million, compared to $16.2 million in Q1 2022, marking a 15.4% increase[115]. - The company spent $1.9 million on preclinical programs in Q1 2023, up from $1.3 million in Q1 2022, indicating a 46.2% increase[112]. Clinical Trials and Product Development - The company is conducting a Phase I/II clinical trial for AMT-130, its gene therapy candidate for Huntington's disease, with 26 patients enrolled[79]. - AMT-130 showed a 53.8% decline in mutant Huntingtin protein levels in cerebral spinal fluid at 12 months of follow-up in treated patients[82]. - The company initiated a GLP toxicology study for AMT-260 in the third quarter of 2022, targeting refractory temporal lobe epilepsy[83]. - The company has a pipeline of innovative gene therapies, including candidates for Huntington's disease and ALS, with HEMGENIX® approved for commercialization in the U.S. and Europe[75]. Revenue Sources - Contract manufacturing revenues recognized for HEMGENIX® amounted to $4.9 million in the three months ended March 31, 2023, compared to nil in the same period in 2022[104]. - Collaboration revenues decreased to $0.4 million in the three months ended March 31, 2023, from $1.4 million in the same period in 2022[103]. Expenses and Liabilities - Selling, general and administrative expenses rose to $17.8 million in Q1 2023 from $11.0 million in Q1 2022, a significant increase of 61.8%[117]. - Interest expense associated with the 2021 Restated Facility was $3.6 million for the three months ended March 31, 2023, compared to $2.4 million during the same period in 2022[54]. - The company had total liabilities of $37.222 million as of March 31, 2023, which included $36.894 million in contingent consideration[42]. Regulatory and Compliance Risks - The company faces significant risks related to FDA compliance and manufacturing regulations, which could materially harm its business if not adhered to[190]. - The complexity of gene therapies may lead to production difficulties, impacting the ability to meet regulatory requirements and commercial prospects[194]. - Regulatory approval processes for gene therapies are expensive and lengthy, with potential for significant delays or rejection of applications[200]. - The company has encountered risks related to the compliance of its manufacturing facility with government regulations, which could materially harm its business[158]. Future Outlook - Future capital requirements will depend on various factors, including milestone payments from CSL Behring and the costs of clinical trials for AMT-130[145]. - The company has potential milestone payments of up to $43.5 million related to the Corlieve acquisition, with additional payments of $174.0 million tied to Phase III development[128].