Larimar Therapeutics' Friedreich's Ataxia Investigational Drug Differentiated From Biogen's Marketed Drug
Larimar Therapeutics' Friedreich's Ataxia Investigational Drug Differentiated From Biogen's Marketed Drug
On Monday, Larimar Therapeutics Inc (NASDAQ:LRMR) announced that the FDA removed the previous partial clinical hold on -nomlabofusp (CTI-1601) clinical program for Friedreich's Ataxia (FA).
週一，Larimar Therapeutics Inc（納斯達克股票代碼：LRMR）宣佈，美國食品藥品管理局取消了先前對弗裏德賴希共濟失調（FA）的-nomlabofusp（CTI-1601）臨床計劃的部分臨床擱置。
Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria.
Nomlabofusp是一種新的蛋白質替代療法，旨在通過向線粒體輸送弗拉他辛來解決FA的根本原因。
The FDA removed the partial clinical hold after reviewing data from the company's recently completed Phase 2 dose exploration study.
美國食品藥品管理局在審查了該公司最近完成的第二階段劑量探索研究的數據後，取消了部分臨床擱置。
The review included data from the 25 mg and 50 mg cohorts in patients who received daily dosing of nomlabofusp for 14 days, followed by every other day dosing until day 28.
該審查包括來自25毫克和50毫克隊列的數據，這些患者每天服用nomlabofusp，持續14天，然後每隔一天給藥一次，直到第28天。
In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period.
在第二階段劑量探索研究中，nomlabofusp在爲期四周的治療期內總體耐受性良好。
Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells.
Nomlabofusp具有可預測的藥代動力學特徵，並表現出皮膚和口腔細胞中frataxin水平的劑量依賴性增加。
All patients with quantifiable levels at baseline and Day 14 in the 50 mg cohort achieved frataxin levels in skin cells over 33% of the average level observed in healthy volunteers at Day 14, and 3 patients achieved levels greater than 50% of the average healthy volunteer level.
在50 mg隊列中，所有在基線和第14天具有可量化水平的患者的皮膚細胞中frataxin水平均超過健康志願者在第14天觀察到的平均水平的33％，有3名患者的水平超過平均健康志願者水平的50％。
The long-term safety and tolerability, pharmacokinetics, and frataxin levels in peripheral tissues following nomlabofusp are currently being evaluated in the ongoing OLE study.
正在進行的OLE研究目前正在評估nomlabofusp後外周組織中的長期安全性和耐受性、藥代動力學和弗拉他辛水平。
The OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp.
OLE研究最初將評估每天皮下注射25毫克的nomlabofusp。
Larimar plans to dose escalate to 50 mg in the OLE study following additional characterization of frataxin PD at the 25 mg dose.
Larimar計劃在OLE研究中將劑量增加到50 mg，此前對25mg劑量的frataxin PD進行了進一步的表徵。
William Blair writes that the 50 mg dose can potentially increase FXN levels beyond the 5%-10% increase over baseline KOLs have suggested as a therapeutic threshold.
威廉·布萊爾寫道，50毫克的劑量有可能使FXN水平超過KOL所建議的治療閾值比基線增加5％-10％。
If necessary, dose escalation above 50 mg would require the submission of additional data for FDA review to support the increased dose.
如有必要，劑量增加到50 mg以上將需要提交額外數據以供FDA審查，以支持增加劑量。
Interim data from the OLE study is expected in the fourth quarter of 2024.
OLE研究的中期數據預計將在2024年第四季度公佈。
The analyst sees nomlabofusp as the leading therapy to boost FXN expression, which is differentiated from Biogen Inc's (NASDAQ:BIIB) Skyclarys and could potentially be used adjunctively pending safety data. William Blair reiterates the Outperform rating on Larimar.
該分析師將nomlabofusp視爲增強FXN表達的主要療法，FXN與百健公司（納斯達克股票代碼：BIIB）的Skyclarys不同，在獲得安全數據之前，有可能作爲輔助用途。威廉·布萊爾重申了對拉里瑪的跑贏大盤評級。
Price Action: LRMR shares are up 10.66% at $8.05 at last check Tuesday.
價格走勢：在週二的最後一次檢查中，LRMR股價上漲10.66％，至8.05美元。
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