Etranacogene dezaparvovec

Hands passing money/Taylor Tieden for BioSpace When Orchard Therapeutics announced the $4.25 million price tag in March for its newly approved rare disease gene therapy Lenmeldy, it became the most expensive on the market. But since 2019, the FDA has approved five others that cost more than $2 million. Some insurers, even when legally required, are not having it when it comes to paying out the cost of a treatment. A November 2023 ProPublica investigation found that insurance provider Priority Health quietly nixed coverage of gene therapies as a cancer treatment, even though under Michigan state law the payer was required to cover clinically effective cancer drugs. The gene therapy in question, CAR-T, was the last hope for patient Forrest VanPatten to recover from an aggressive case of lymphoma. According to ProPublica, his CAR-T treatment was estimated to cost $475,000. VanPatten died while waiting for the results of his third request for coverage. Globally, many healthcare systems are choosing not to cover gene therapies at all. Even though every health regulatory body reviews the same scientific and trial information, Canada and countries across Europe have completely different gene therapy coverage models than the U.S., according to a December 2021 study in Health Policy. “The primary underlying reasons for access restrictions in all regions appear to be scientific uncertainty at the time of approval, limited evidence, and the high cost of these therapies,” the study authors wrote. But even with the high price tag and what some call questionable evidence that a treatment will provide a lifetime benefit, private American payers are already shelling out for gene therapy treatments, said Graig Suvannavejh, the managing director of biopharma and biotech at Mizuho Securities. However, debate remains over what conditions warrant six-figure prices and how payers can recoup that money if a gene therapy fails. “This will continue to be an evolving dynamic over the next 10 to 20 years,” Suvannavejh said. How Do Insurers Pay Biopharma Firms for Gene Therapy? At this juncture, both public and private payers can pay for gene therapies either upfront or in annual installments over a handful of years. Both types of agreements can, and largely do, build in a caveat that if a patient does not see a significant benefit over time, the payer can receive a rebate of part of the gene therapy's cost. These are known as outcome- or value-based agreements. “Gene therapy [means] that you’re given it one time [and] you’re stuck with it, good and bad,” Suvannavejh told Biospace. “The issue then is if it is meant to be given as a one-time treatment, are you actually getting the benefits for the duration of your life?” Suvannavejh said that other factors, such as existing therapeutic options beyond gene therapy and the disease’s fatality rate, are part of the coverage discussion. If a condition has a high fatality rate and few other options, there may be greater willingness to agree to a high price tag. Right now, he added, gene therapy payments tend to be upfront in a lump sum. Bob Lojewski, senior vice president and general manager of CSL Behring North America, said that private payers have been largely receptive to the $3.5 million price tag of the company’s HEMGENIX, a gene therapy treatment for hemophilia B. Currently, payers are covering “the vast majority of the U.S. population” who may need HEMGENIX, he said. “To date, every patient referred for HEMGENIX has been covered through insurance, and we expect access for eligible people interested in this one-time treatment will continue,” he said. Lojewski added that the CSL team has seen an “accelerating rate” of patients referred for treatment in the past six months, potentially due to the agreements in place to cover the therapy. He did not say whether insurers were paying by lump sum or in installments. Internal CSL figures estimate that between annual bleed rates and required therapies to address hemophilia B, a patient with the disease can cost an insurer $20 million over the course of their life. That puts the expenditure of $3.5 million for a gene therapy to reverse or cure the condition into perspective. When that is extrapolated out, HEMGENIX could save the U.S. healthcare ecosystem tens of billions of dollars over the next 20 years, Lojewski said. However, saving all of that money in the long run comes with tens of millions of dollars in upfront costs to private companies or the federal government, while it may take decades to realize the cost savings from that investment. According to the Alliance for Regenerative Medicine, an analysis by NEWDIGS at Tufts University estimates that gene therapy expenditure in the U.S. will reach $7.5 billion by 2030. In context, that’s approximately 0.1% of national projected healthcare spending for that year. Public Payers Negotiate Costs for Gene Therapies Medicare and Medicaid both cover many patients affected by conditions addressed by new gene therapies. Medicaid covers two-thirds of patients with sickle cell disease, which bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics' Casgevy, listed at $3.1 and $2.2 million respectively, are designed to treat. Furthermore, Medicaid provides healthcare coverage for over half of children with spinal muscular atrophy, some cases of which can be treated with Novartis’ $2.1 million Zolgensma treatment. But public payers may be negotiating discounts on those list prices down year over year, while it’s unclear whether private payers are negotiating regularly for treatments to be significantly less than their list prices. Take Zolgensma. With a $2.1 million listing price, public insurers paid $1.89 million per claim in 2019, and by 2022, that figure dropped to $1.36 million, nearly a 40% discount from its list price, according to the Centers for Medicare & Medicaid Services (CMS). In 2022, CMS spent roughly $87.25 million on some 63 Zolgensma treatments. Like private insurers, public payers are setting up rebate programs in case these therapies do not deliver on their promises, although this strategy isn’t commonly used just yet. “Supplemental rebates only accounted for 9% of all Medicaid drug prices in fiscal year 2022,” an Alliance for Regenerative Medicine representative told Biospace. Karen Fischer is a freelance science writer based in New Mexico. Reach her at kfischerwrites.com.

~ On track to initiate FDA interaction regarding AMT-130 in second quarter of 2024 and provide a clinical update from the Phase I/II trials in mid-2024 ~ ~ Clinical trial initiation for Fabry disease on track to begin in second quarter of 2024, followed by refractory mesial temporal lobe epilepsy and SOD1-ALS in third quarter of 2024 ~ ~ Comprehensive review of operations and options to reduce expenses underway and expected to be completed in mid-2024 ~ LEXINGTON, Mass. and AMSTERDAM, May 07, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2024 and highlighted recent progress across its business. “In the first quarter, we made solid progress across three key business priorities – clarifying the road ahead for AMT-130 in Huntington’s disease, initiating three new Phase I/II clinical trials, and prudently conserving our capital,” stated Matt Kapusta, chief executive officer of uniQure. “We are on track to initiate FDA interactions on AMT-130 this quarter and look forward to presenting longer-term follow-up data from our Phase I/II trials mid-year, which will include up to three years of patient follow-up on 29 treated patients. We have also made significant progress towards the initiation of new Phase I/II studies. We have obtained IRB approvals for initial sites, which are poised for imminent activation, and have also identified prospective patients. Enrollment for the Fabry disease study is on track to begin in the second quarter of 2024, with the SOD1-ALS and MTLE studies expected to begin in third quarter.” “In addition, we are actively engaged in a comprehensive review of uniQure’s operations and options to reduce expenses and increase shareholder value,” he added. “We believe deeply in the significant value of our assets, as well as the need to achieve a focused and sustainable level of investment in order to maximize this potential. We expect to complete this evaluation mid-2024.” Recent Updates Advancing AMT-130 for the treatment of Huntington’s disease The Company is on track to initiate interactions with the U.S. Food and Drug Administration in the second quarter of 2024 with the goal of defining the future clinical and regulatory pathway for AMT-130. These interactions, which are expected to continue throughout the year, are expected to include discussions of the interim data from the ongoing Phase I/II clinical trials and the potential to leverage natural history comparators alongside our long-term Phase I/II clinical data. The Company expects to provide an update on its regulatory plans as part of the next clinical update on AMT-130 mid-year and have greater clarity regarding a potential approval pathway for AMT-130 before the end of the year.In mid-2024, the Company expects to provide an update from its ongoing Phase I/II clinical trials of AMT-130. The data will include up to three years of follow-up data on 29 treated patients in both the low- and higher-dose cohorts, of which 21 patients will have a minimum of two years of follow-up.Patient dosing is ongoing in a third cohort of up to 12 patients to further evaluate both doses of AMT-130 in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. Enrollment in this third cohort is expected to be completed in the second half of 2024. Initiating new Phase I/II clinical studies AMT-191 for the treatment of Fabry disease - Patient enrollment in a Phase I/IIa clinical trial is expected to begin in the second quarter of 2024. The Phase I/IIa clinical trial will be a U.S.-based, multi-center, open-label trial consisting of two cohorts enrolling up to six adult male patients each. The study is designed to evaluate safety, tolerability, and early signs of efficacy. AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) – Patient enrollment in a Phase I/II clinical trial is expected to begin in the third quarter of 2024. The Phase I/II clinical trial will be a U.S.-based, multi-center, open-label trial consisting of three cohorts with up to four patients each receiving a one-time intrathecal infusion with immunosuppression. The study is designed to evaluate safety, tolerability, and early signs of efficacy. AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (MTLE) – Patient enrollment in a Phase I/IIa clinical trial is expected to begin in third quarter of 2024. The Phase I/II will be a U.S.-based consisting of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data. Upcoming Investor Events RBC Capital Markets 2024 Global Healthcare Conference, May 15th – New York, NY Financial Highlights Cash position: As of March 31, 2024, the Company held cash and cash equivalents and investment securities of $555.7 million, compared to $617.9 million as of December 31, 2023. The Company expects cash, cash equivalents and investment securities will fund operations into the second quarter of 2027. Revenues: Revenue for the three months ended March 31, 2024 was $8.5 million, compared to $5.3 million in the same period in 2023. The net increase of $3.2 million in revenue resulted from an increase of $2.9 million from collaboration revenue and an increase of $1.2 million in license revenue partially offset by a decrease of $0.9 million from contract manufacturing of HEMGENIX® for CSL. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were $9.1 million for the three months ended March 31, 2024, compared to $2.4 million for the same period in 2023. The increase primarily relates to expensing costs previously capitalized as inventory. R&D expenses: Research and development expenses were $40.7 million for the three months ended March 31, 2024, compared to $60.8 million during the same period in 2023. The $20.1 million decrease was primarily related to an $8.1 million decrease in external spend for the ALS SOD1 program, as prior year costs included a $10.0 million one-time cost for the acquisition of the program from Apic Bio. Additionally, there was a $7.0 million decrease in preclinical and other program spend, a $2.0 million decrease in costs incurred related to preclinical supplies and a $2.7 million decrease in employee related expenses. SG&A expenses: Selling, general and administrative expenses were $13.9 million for the three months ended March 31, 2024, compared to $17.8 million during the same period in 2023. The $3.9 million decrease was primarily related to a $1.7 million decrease in professional and intellectual property fees, as well as a reduction of expenses for information technology. Other non-operating items, net: Other non-operating items, net was an expense of $10.7 million for the three months ended March 31, 2024, compared to $4.3 million for the same period in 2023. The $6.5 million increase in other non-operating items, net was primarily related to an increase in non-cash interest expense of $12.5 million related to the royalty agreement that the Company entered into in May 2023, which partially was offset by an increase of $4.8 million in interest income earned on investment securities and cash on hand. Net loss:The net loss for the three months ended March 31, 2024, was $65.6 million, or $1.36 basic and diluted loss per ordinary share, compared to $77.2 million net loss for the same period in 2023, or $1.63 basic and diluted loss per ordinary share. About uniQure uniQure’s mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene therapy for hemophilia B – a historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are now leveraging our modular and validated technology and manufacturing platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," “establish,” "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," “seek,” "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second quarter of 2027; the Company’s ongoing review of its operations and options to reduce expenses, the Company’s expectations that such review will lead to a strategy that, if effectively executed, will reduce operating expenses and increase shareholder value, and the expected timing of the completion of such review; the Company’s plans to announce additional follow-up data from its ongoing U.S. and European Phase I/II clinical studies of AMT-130; the Company’s plans to initiate interactions with the FDA regarding the further development of AMT-130, the timing of such interactions and expectations regarding regulatory clarity from such interactions; the Company’s plans regarding the third cohort in its AMT-130 clinical trial and the timing of enrollment for such cohort; and the Company’s plans to initiate patient enrollment for AMT-191 in the second quarter of 2024, and to initiate patient enrollment for AMT-260 and AMT-162 in the third quarter of 2024. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; the Company’s ability to fund its operations and to raise additional capital as needed; and the impact of global economic uncertainty, rising inflation, rising interest rates or market disruptions on its business. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024 and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future. uniQure Contacts: FOR INVESTORS: FOR MEDIA: Maria E. CantorDirect: 339-970-7536Mobile: 617-680-9452m.cantor@uniQure.comChiara RussoDirect: 617-306-9137Mobile: 617-306-9137c.russo@uniQure.comTom MaloneDirect: 339-970-7558Mobile:339-223-8541t.malone@uniQure.com uniQure N.V.UNAUDITED CONSOLIDATED BALANCE SHEETS March 31, December 31, 2024 2023 (in thousands, except share and per share amounts)Current assets Cash and cash equivalents$243,062 $241,360Current investment securities 312,621 376,532Accounts receivable and contract asset 10,717 4,193Inventories 7,672 12,024Prepaid expenses 18,839 15,089Other current assets and receivables 3,092 2,655Total current assets 596,003 651,853Non-current assets Property, plant and equipment, net 44,554 46,548Operating lease right-of-use assets 27,695 28,789Intangible assets, net 59,111 60,481Goodwill 25,795 26,379Deferred tax assets, net 11,594 12,276Other non-current assets 5,298 5,363Total non-current assets 174,047 179,836Total assets$ 770,050 $ 831,689Current liabilities Accounts payable$5,231 $6,586Accrued expenses and other current liabilities 22,658 30,534Current portion of contingent consideration 27,587 28,211Current portion of operating lease liabilities 7,997 8,344Total current liabilities 63,473 73,675Non-current liabilities Long-term debt 102,120 101,749Liability from royalty financing agreement 405,398 394,241Operating lease liabilities, net of current portion 26,983 28,316Contingent consideration, net of current portion 14,625 14,795Deferred tax liability, net 7,376 7,543Other non-current liabilities 3,321 3,700Total non-current liabilities 559,823 550,344Total liabilities 623,296 624,019Shareholders' equity Total shareholders' equity 146,754 207,670Total liabilities and shareholders' equity$ 770,050 $ 831,689 uniQure N.V.UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS Three months ended March 31, 2024 2023 (in thousands, except share and per share amounts)Total revenues$ 8,485 $ 5,325 Operating expenses: Cost of license revenues (150) — Cost of contract manufacturing revenues (9,076) (2,435)Research and development expenses (40,692) (60,809)Selling, general and administrative expenses (13,937) (17,848)Total operating expenses (63,855) (81,092)Other income 1,376 1,811 Other expense (234) (216)Loss from operations (54,228) (74,172)Non-operating items, net (10,734) (4,262)Loss before income tax (expense) / benefit$ (64,962) $ (78,434)Income tax (expense) / benefit (656) 1,207 Net loss$ (65,618) $ (77,227) Basic and diluted net loss per ordinary share$(1.36) $(1.63)Weighted average shares used in computing basic and diluted net loss per ordinary share 48,384,510 47,436,335