Larimar Therapeutics, Inc. (NASDAQ: LRMR) saw a 17.19% increase in after-hours trading on Monday, reaching $8.52, following a positive regulatory update. Investor excitement over recent advancements in the company's clinical trials is reflected in this notable boost.
- FDA Lifts Partial Hold On Nomlabofusp Program
- Promising Interim Data And Future Plans
FDA Lifts Partial Hold On Nomlabofusp Program
Larimar Therapeutics (LRMR) announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on its nomlabofusp (CTI-1601) clinical program. Nomlabofusp, a pioneering protein replacement therapy, is being developed to treat Friedreich's Ataxia (FA).
This therapeutic approach aims to address the underlying cause of FA by delivering frataxin directly to mitochondria. The FDA made its judgment after carefully examining the information from the recently finished four-week Phase 2 dosage exploration trial for Larimar, which was placebo-controlled.
Two cohorts participated in the trial; they were given dosages of 25 mg and 50 mg, respectively, and were dosed daily for the first 14 days, then every other day until day 28. Larimar was able to move forward with increasing the dosage in its current Open-Label Extension (OLE) trial to 50 mg as a result of this evaluation.
Promising Interim Data And Future Plans
The OLE study, which is currently evaluating the long-term safety and pharmacokinetics of nomlabofusp, will initially focus on 25 mg daily subcutaneous injections, either self-administered or administered by a caregiver. Larimar plans to escalate to a 50 mg dose after further characterizing frataxin pharmacodynamics (PD) at the 25 mg level. Should there be a need to escalate beyond 50 mg, additional data will be required for FDA review.
Nomlabofusp was generally well tolerated, with a predictable pharmacokinetic profile and dose-dependent increases in skin and buccal cell frataxin levels, according to the Phase 2 research. Notably, patients in the 50 mg cohort achieved significant increases in frataxin levels, with some reaching over 50% of the average levels observed in healthy individuals.
Interim data from the OLE study are anticipated in the fourth quarter of 2024, which will provide further insights into the long-term effects of nomlabofusp in patients with FA. This data is crucial for determining the future trajectory of the drug's development and potential FDA approval for broader use.
在监管机构公布积极的最新消息后，Larimar Therapeutics, Inc.（纳斯达克股票代码：LRMR）周一盘后交易上涨17.19％，达到8.52美元。投资者对公司临床试验最近进展的兴奋反映在这一显著增长中。
- 美国食品和药物管理局取消了对Nomlabofusp计划的部分搁置
- 前景光明的中期数据和未来计划
美国食品和药物管理局取消了对Nomlabofusp计划的部分搁置
Larimar Therapeutics（LRMR）宣布，美国食品药品监督管理局（FDA）已解除对其nomlabofusp（CTI-1601）临床计划的部分临床搁置。Nomlabofusp是一种开创性的蛋白质替代疗法，正在开发用于治疗弗里德赖希的共济失调（FA）。
这种治疗方法旨在通过将frataxin直接输送到线粒体来解决FA的根本原因。美国食品药品管理局在仔细研究了最近完成的为期四周的安慰剂对照的Larimar第二阶段剂量探索试验的信息后做出了判断。
两个队列参与了试验；他们分别服用了25 mg和50 mg的剂量，并在最初的14天内每天给药，然后每隔一天给药一次，直到第28天。由于这项评估，Larimar得以将其当前的开放标签延期（OLE）试验的剂量增加到50 mg。
前景光明的中期数据和未来计划
OLE研究目前正在评估nomlabofusp的长期安全性和药代动力学，最初将侧重于每天25毫克的皮下注射，可以自行给药，也可以由护理人员给药。在进一步表征弗拉他辛药效学（PD）的25毫克水平后，Larimar计划将剂量扩大到50毫克的剂量。如果需要上报超过50 mg，则需要其他数据才能进行FDA审查。
根据第二阶段研究，Nomlabofusp的耐受性总体良好，药代动力学特征可预测，皮肤和口腔细胞frataxin水平的剂量依赖性增加。值得注意的是，50 mg队列中的患者的frataxin水平显著增加，其中一些达到了健康个体平均水平的50％以上。
OLE研究的中期数据预计将在2024年第四季度公布，这将为nomlabofusp对FA患者的长期影响提供进一步的见解。这些数据对于确定该药物的未来发展轨迹以及美国食品药品管理局可能批准的更广泛用途至关重要。
