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).
周一，Larimar Therapeutics Inc（纳斯达克股票代码：LRMR）宣布，美国食品药品管理局取消了先前对弗里德赖希共济失调（FA）的-nomlabofusp（CTI-1601）临床计划的部分临床搁置。
Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria.
Nomlabofusp是一种新的蛋白质替代疗法，旨在通过向线粒体输送弗拉他辛来解决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.
美国食品药品管理局在审查了该公司最近完成的第二阶段剂量探索研究的数据后，取消了部分临床搁置。
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.
该审查包括来自25毫克和50毫克队列的数据，这些患者每天服用nomlabofusp，持续14天，然后每隔一天给药一次，直到第28天。
In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period.
在第二阶段剂量探索研究中，nomlabofusp在为期四周的治疗期内总体耐受性良好。
Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells.
Nomlabofusp具有可预测的药代动力学特征，并表现出皮肤和口腔细胞中frataxin水平的剂量依赖性增加。
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队列中，所有在基线和第14天具有可量化水平的患者的皮肤细胞中frataxin水平均超过健康志愿者在第14天观察到的平均水平的33％，有3名患者的水平超过平均健康志愿者水平的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.
正在进行的OLE研究目前正在评估nomlabofusp后外周组织中的长期安全性和耐受性、药代动力学和弗拉他辛水平。
The OLE study will initially evaluate daily subcutaneous injections of 25 mg of nomlabofusp.
OLE研究最初将评估每天皮下注射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计划在OLE研究中将剂量增加到50 mg，此前对25mg剂量的frataxin PD进行了进一步的表征。
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·布莱尔写道，50毫克的剂量有可能使FXN水平超过KOL所建议的治疗阈值比基线增加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，剂量增加到50 mg以上将需要提交额外数据以供FDA审查，以支持增加剂量。
Interim data from the OLE study is expected in the fourth quarter of 2024.
OLE研究的中期数据预计将在2024年第四季度公布。
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.
该分析师将nomlabofusp视为增强FXN表达的主要疗法，FXN与百健公司（纳斯达克股票代码：BIIB）的Skyclarys不同，在获得安全数据之前，有可能作为辅助用途。威廉·布莱尔重申了对拉里玛的跑赢大盘评级。
Price Action: LRMR shares are up 10.66% at $8.05 at last check Tuesday.
价格走势：在周二的最后一次检查中，LRMR股价上涨10.66％，至8.05美元。
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