A clinical study published in the British Journal of Cancer shows encouraging antitumor activity and a good safety profile of the compound LAM561 (hydroxyoleic acid, sodium) against brain tumors (gliomas and glioblastomas, the most aggressive and common tumors) and other types. of cancer, after completing a Phase I/II clinical study.
This investigational drug is being developed by the Spanish biotechnology company Laminar Pharmaceuticals, together with some UiB research teams, and is currently in Phase IIB/III of clinical development. If the results of the interim analysis show significant clinical benefit, conditional marketing approval could be obtained in Europe for newly diagnosed glioblastoma patients during 2024.
The Phase I/II clinical study was an open-label, nonrandomized trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and antitumor activity of daily oral treatment with LAM561 in 54 patients with glioma and other advanced solid tumors. In the study, a promising response was observed in patients with glioma and other high-grade tumors, who had no therapeutic alternatives after being treated unsuccessfully with alternative antitumor therapies.
Of the 21 high-grade glioma patients treated throughout dose escalation and expansion, eight (38.1%) had a clinical benefit (partial response or disease stabilization) according to RANO consensus medical criteria ( Response Assessment in Neuro-Oncology), with clinical benefit lasting at least 6 months in five patients, with one patient showing an exceptional response lasting more than 2.5 years.
Good security profile
Data from this clinical study demonstrate that LAM561 is generally well tolerated, which is unusual for traditional antitumor drugs, which tend to have high toxicity. LAM561 presents a combination of antitumor effects and a good safety profile, a promising combination in cancer chemotherapy.
The preliminary results are encouraging and, together with the additional positive data expected from the ongoing comparative Phase IIb/III study in the near future, LAM561 could become an important therapeutic alternative for these patients, whose life expectancy is just one year from the time of diagnosis. “In the study it can be seen that this product shows clinical activity against different types of cancer, and almost 40 percent of patients with glioma or glioblastoma obtained a therapeutic response in our study,” explains Professor Pablo Escribá, CEO of Laminar Pharma.
Phase III clinical study
Escribá believes that “in the image of the evolution of the tumor of a patient from the study published in the British Journal of Cancer, you can see how, in this patient who was taking LAM561 as monotherapy (without added chemotherapy treatment), the tumor brain is significantly reduced (93%) over several treatment cycles.
This investigational medication is not currently available to patients outside of clinical trials. Although these first results in patients are encouraging, we must wait until 2024 to obtain preliminary comparative results against placebo and thus request conditional marketing authorization from the EMA. In order to accelerate the process, Laminar Pharmaceuticals has already received orphan drug designation (drug for rare diseases with significant unmet therapeutic needs) in the European Union (by the EMA) and in the United States (by the FDA).
Furthermore, in the US, LAM561 has obtained the Fast-Track designation for evaluation in the treatment of glioblastoma, being the first Spanish drug to achieve this important milestone for an oncological drug, which aims to advance and facilitate the evaluation and development of promising treatments. LAM561 is currently being evaluated in a study
pivotal clinical trial (Phase IIb/III) and a first analysis of the results will be carried out in early 2024 by an independent committee. If the results prove to be clinically significant, this would allow a marketing authorization to be applied for.
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