Laminar Pharma obtains FDA Fast Track designation for LAM561 for the treatment of glioblastoma
Laminar Pharma has been granted by the US FDA with a Fast-Track designation for the investigation of LAM561 for the treatment of patients with glioblastoma. Fast track is a mechanism designed to facilitate the development and expedite the review of drugs to treat serious conditions and which demonstrate the potential to address an unmet medical need. If there are approved therapies available, a Fast Track drug must show some advantage over available therapy. The purpose is to get important new drugs to the patient earlier. Therefore, Fast Track addresses a broad range of serious conditions.
The European Grant shows the scientific potential of the Technology Platform (Melitherapy®) developed by Laminar, which has led to the discovery, research and development of a few drugs currently in human trials. All the milestones and recognitions obtained so far rewards approximately 2 decades of research, in which the discovery of LAM561 and its antitumor effects were published by a research group from the University of the Balearic Islands and transferred to the local biotech company Laminar Pharmaceuticals.
One hundred thousand people die every year of Glioblastoma, a fast-progressing brain cancer that even with the current aggressive treatments only has a median survival of 1 year and a 2% survival after 5 years. Doctor Adrian G McNicholl, Chief of Clinical Operations, explains; “Patients diagnosed with this disease suddenly need to face low life expectancy; fast deterioration of personal autonomy and quality of life; a severe impact on their families (psychological and economic); and having to undergo invasive surgery followed by an aggressive combination of radiotherapy and chemotherapy that will only extend their life by some weeks”.
In this context, LAM561 has been investigated in 2 completed and 2 ongoing clinical trials and the FDA has considered it meets the requirements to receive designation as a Fast Track development program for the investigation of LAM561 for the treatment of patients with glioblastoma. In the words of Doctor Pablo Escriba, CEO of Laminar Pharma “This is an important milestone for Laminar Pharma because it shows that an independent review by FDA experts agrees that the nonclinical and clinical data on our product demonstrates sufficient potential to address an unmet medical need to warrant fast track designation for investigation in the treatment of this devastating cancer”.
Laminar Pharma, especially the US branch directed by Doctor Victoria Lladó, is proud to obtain this difficult landmark, as she presents “Fast-track is no easy endeavour, and most drugs, even some effective ones are unable to obtain it: the number of Fast Track designations by the FDA is relatively low, just 35”. And she adds “during the period 2020 to June 2022 no fast-track designation has been granted for a medicine to treat glioblastoma. In fact, the current standard-of-care treatment has not changed during the last 20 years. A plausible treatment against glioblastoma has been the desired aim of several big pharmaceutical companies and relevant research groups worldwide.
Doctor Richard Taylor, Head of Medical and Regulatory Affairs for Laminar Pharma echoed the view that this latest regulatory recognition by the FDA, added to the Orphan Drug and Orphan Product designations previously obtained in the US and in Europe, are strategic achievements which provide the opportunity for enhanced engagement and development support from the FDA and European Medicines Agency (EMA) to help accelerate/facilitate the LAM-561 development program.
The activity of LAM561 on tumors is explained by Doctor Paula Fernandez, Scientific chair “our active principle Idroxioleic acid is a fatty acid that targets tumour cell membranes changing their biophysical properties from a pathological profile to one similar to healthy cells. These modifications have an impact on cancer cell signaling as a whole, stopping cancer growth and finally leading to cancer cell death”.Clinical trials have shown that idroxioleic acid is a generally well-tolerated drug with a good safety profile that has in some instances been taken over a prolonged period both as a stand-alone and in combination with chemotherapy and radiotherapy. LAM561 has been developed as a patient-friendly oral suspension, and more recently a patient-friendly sachet presentation, that can be easily administered at home. For this reason, after a request by and in collaboration with the Dana Farber Foundation (MA, USA) and Hackensack Hospital (NJ, USA), Laminar is currently conducting a Phase I/IIA clinical study to evaluate the safety, tolerability and clinical effects of LAM561 in children with advanced brain tumors. In the words of Doctor Derek Hanson (Director of Pediatric Neuro-oncology at Hackensack Meridian Health Joseph M. Sanzari Children´s Hospital, and Independent Principal Investigator of the trial): “If LAM561 meets the expectations, and nothing indicates otherwise, this new approach to anti-tumor treatment could be a game changer for both children and adult cancer patients”.
*Research and development of this drug have received public funds from the European Commission, the Government of Spain, the Government of Illes Balears, and from the Spanish Association against Cancer (AECC)