Tale Of Two Ionis-Partnered Neurology-Focused Early Stage Assets-Biogen Discontinues One, Decides Not To Exercise Licensing Option For Another
Tale Of Two Ionis-Partnered Neurology-Focused Early Stage Assets-Biogen Discontinues One, Decides Not To Exercise Licensing Option For Another
Thursday, Ionis Pharmaceuticals Inc (NASDAQ:IONS) and Biogen Inc (NASDAQ:BIIB) decided to terminate the development of BIIB105 (ION541), an investigational antisense oligonucleotide (ASO) for amyotrophic lateral sclerosis (ALS) based on topline results from the Phase 1/2 ALSpire study.
週四，愛奧尼斯製藥公司（納斯達克股票代碼：IONS）和百健公司（納斯達克股票代碼：BIIB）根據AlSpire第1/2期研究的主要結果，決定終止用於肌萎縮性側索硬化（ALS）的研究性反義寡核苷酸（ASO）BIIB105（ION541）的開發。
BIIB105 was designed to reduce the expression of ataxin-2 (ATXN2) protein and demonstrated statistically significant cerebrospinal fluid (CSF) ATXN2 protein reductions in the study.
BIIB105 旨在減少共濟失調毒素-2（ATXN2）蛋白的表達，在研究中顯示出具有統計學意義的腦脊液（CSF）ATXN2 蛋白降低。
Related: Rare Disease Focused Ionis Pharmaceuticals Gears Up for FDA Submission After Donidalorsen Outperforms Placebo In Swollen Vessels Study.
相關： 在Donidalorsen的血管腫脹研究中表現優於安慰劑之後，專注於罕見病的愛奧尼斯製藥公司正準備提交美國食品藥品管理局的申請。
However, over the 6-month placebo-controlled period, treatment with BIIB105 did not result in a reduction in levels of plasma neurofilament light chain (NfL), a marker of neurodegeneration and neuronal damage.
但是，在 6 個月的安慰劑對照期內，使用 BIIB105 治療並未導致血漿神經絲輕鏈 (nFl) 水平降低，血漿神經絲輕鏈 (nFL) 是神經變性和神經元損傷的標誌。
Additionally, BIIB105 did not demonstrate an impact on clinical outcome measures of function, breathing and strength.
此外，BIIB105 未顯示出對功能、呼吸和力量等臨床結果指標的影響。
"While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process," said Stephanie Fradette, Head of the Neuromuscular Development Unit at Biogen.
Biogen神經肌肉發育部負責人斯蒂芬妮·弗雷德特說：“儘管 BIIB105 降低了 ATXN2 蛋白，但它並沒有減少神經絲，這使我們相信 BIIB105 沒有減緩疾病進程。”
Longer-term biomarker and efficacy data from the open-label extension were similar to those seen during the 6-month placebo-controlled treatment period, with sustained reductions in ATXN2 but no impact on NfL or clinical outcome measures over 40+ weeks of follow-up.
開放標籤延期的長期生物標誌物和療效數據與6個月的安慰劑對照治療期內的數據相似，ATXN2 持續下降，但在40多周的隨訪中對nFl或臨床結果指標沒有影響。
No evidence of benefit was observed in any subgroup evaluated, including those participants with a Poly-CAG expansion in the ATXN2 gene.
在評估的任何亞組中均未觀察到有益的證據，包括那些在亞組中擴張了Poly-CAG的參與者 ATXN2 基因。
Data analysis from the study is ongoing. The companies will present the BIIB105 Phase 1/2 data at the upcoming European Network to Cure ALS meeting in June.
該研究的數據分析正在進行中。兩家公司將在即將於6月舉行的歐洲慢性側索硬化症治療網絡會議上公佈 BIIB105 第1/2階段的數據。
Concurrently, Ionis Pharmaceuticals reported topline data from the HALOS Phase 1/2a open-label study of ION582 in Angelman syndrome.
同時，愛奧尼斯製藥公司報告了安格爾曼綜合徵中 ION582 的 HALOS 1/2a 期開放標籤研究的頭條數據。
ION582 was safe and well tolerated in the study.
在研究中，ION582 是安全的，耐受性良好。
ION582 demonstrated consistent improvements across multiple functional domains in Angelman syndrome patients, with improvements observed in key areas of functioning, including cognition, communication, and motor function.
ION582 顯示 Angelman 綜合徵患者的多個功能領域持續改善，包括認知、溝通和運動功能在內的關鍵功能領域均有改善。
Ionis also announced that it will independently advance ION582 as Biogen has elected not to exercise its option to license ION582.
愛奧尼斯還宣佈，它將獨立推進 ION582，因爲百健已選擇不行使許可 ION582 的選擇權。
Ionis plans to review the ION582 Phase 1/2a results with regulatory authorities to align on the design of the pivotal program.
愛奧尼斯計劃與監管機構一起審查 ION582 1/2a 階段的結果，以協調關鍵計劃的設計。
Price Action: IONS shares are down 5.19% at $37.17, and BIIB shares are down 2.12% at $230.33 at last check Thursday.
價格走勢：週四最後一次查看時，IONS股價下跌5.19％，至37.17美元，BIIB股價下跌2.12％，至230.33美元。
Illustration of Phrama lab worker created with MidJourney
使用 MidJourney 創作的 Phrama 實驗室工作人員的插圖