Université Laval

WEST CHESTER, PA, USA I December 09, 2024 I Molecular Targeting Technologies, Inc. (MTTI) published preclinical study results for their proprietary 225 Ac-EBTATE against SSTR2 NET cancers online in the European Journal of Nuclear Medicine. (“Long acting 225 Ac-EBTATE is highly efficacious against somatostatin receptor-2-positive neuroendocrine tumors” ( https://rdcu.be/d2lCG ). Professor Humphrey Fonge of the Université de Laval and lead author commented, “At just 40% of the administered dose reported for 225 Ac-DOTATATE, 225 Ac-EBTATE produces enhanced anti-tumor efficacy in Small Cell lung cancer (SCLC) and Pan-neuroendocrine tumor (NET) models as shown with complete tumor remissions. At a therapeutic dose of 2x 34 kBq, 225 Ac-EBTATE showed general safety for 28 days after blood biochemistry analysis, CBC, and histopathological examination of major organs and tissues. We demonstrated that 225 Ac-EBTATE was effective against human small-cell lung cancer (SCLC) with 80% complete remission and 100% survival in preclinical models.” MTTI has licensed commercial use rights for the patented Evans blue (EB) technology from the National Institute of Biomedical Imaging and Bioengineering (“ NIBIB ”), part of the National Institutes of Health (“ NIH ”). This platform is the basis for the best-in-class, new generation, collection of targeted radiopharmaceuticals (TRP). EB binds to serum albumin, extending in vivo circulatory half-life and tumor residence time, enhancing up to 30-fold uptake at a tumor and enabling good efficacy with significantly lower radiopharmaceutical activity and fewer dosing cycles vs. the current standard of care. Our 3-year follow up of 177 Lu-EBTATE in patients* (N=30) with metastatic neuroendocrine tumors, demonstrated good safety with no nephro- or hepatoxicity and 86% disease control rate using only 40% of the administered dose of 177 Lu-DOTATATE. Dr. Jean-Mathieu Beauregard, MD, Associate Professor of Department of Radiology of Université Laval, said, “I am encouraged to see the positive results of 225 Ac-EBTATE in treating small cell lung cancer and Pan-neuroendocrine tumor (NET) in preclinical studies. I look forward to working with MTTI and Professor Fonge to translate this into the clinic.” Dr. Chris Pak, President & CEO of MTTI commented “ 225 Ac-EBTATE effectiveness was encouraging, paralleling the clinical superiority of 177 Lu-EBTATE. We look forward to collaborating with Professors Beauregard and Fonge to drive 225 Ac-EBTATE into clinical trials in 2025.” Molecular Targeting Technologies, Inc. (MTTI) . Molecular Targeting Technologies, Inc. is a private, clinical stage biotech developing targeted radiopharmaceuticals (TRP) for rare cancers. MTTI is committed to building value by translating innovative TRP to improve human health. For more information: www.mtarget.com . *Jiang Y, Liu Q, Wang G, et al. Safety and efficacy of peptide receptor radionuclide therapy with 177 Lu-DOTA-EB-TATE in patients with metastatic neuroendocrine tumors. Theranostics. 2022;12(15):6437-6445. SOURCE: Molecular Targeting Technologies Inc

Epilepsy is one of the most common neurological conditions ​ in the world, with around 50 million people with the condition. There are a variety of approved medications for epilepsy and pain. but many patients cannot benefit from the drugs or have severe side effects such as depressive mood, psychosis and anxiety, according to Axonis’ website ​. This means more approaches are needed to help these patients. Axonis was launched in 2020 based on research from the U.S. centers Boston Children's Hospital and Harvard in addition to Canada’s Université Laval. The startup’s lead candidate is designed to enhance the effectiveness of Potassium-chloride co-transporter 2 (KCC2), a protein involved in nerve cell inhibitory transmission. By doing so, it can make nerve cells function normally and help patients who previously didn’t respond to treatments or suffer side effects. The program is due to reach clinical testing later this year. The round, which was co-led by Cormorant Asset Management and venBio Partners, will be used to take Axonis’ lead programme through clinical proof-of-concept in patients with epilepsy and pain. It will also let the company work on separate pipeline programmes treating psychiatric and neurodevelopmental disorders. "Axonis' vision is to advance a pipeline of oral KCC2 therapeutics to restore functional inhibition in the CNS to treat neurological disorders without disabling side effects," said Axonis CEO Joanna Stanicka in a public release ​. The epilepsy treatment market ​ was worth around $10.15 billion in 2022 and is projected to grow to around $15.35 billion by 2030, with an annual growth rate of 5.1%. This growth is spurred by increasing approvals of new treatments in addition to improved diagnosis approaches. Some of the current drugs in this market include UCB’s Briviact and Pfizer’s Lyrica. googletag.cmd.push(function () { googletag.display('text-ad1'); }); There are other efforts ​ in development to target KCC2 such as directly activating the protein with small molecules, enhancing its expression in nerve cells with RNA or the gene editing tool CRISPR. Many startups fighting epilepsy are in the medtech space, such as Epi-Minder, an Australian firm that bagged $16 million in a bridge financing round in 2022 to fund the development of its patient monitoring device. Earlier this year, the U.S. company Neurava closed a seed round of more than $2 million to kick off the development of wearable devices to save the lives of epilepsy patients.

Epilepsy is one of the most common neurological conditions ​ in the world, with around 50 million people with the condition. There are a variety of approved medications for epilepsy and pain. but many patients cannot benefit from the drugs or have severe side effects such as depressive mood, psychosis and anxiety, according to Axonis’ website ​. This means more approaches are needed to help these patients. Axonis was launched in 2020 based on research from the U.S. centers Boston Children's Hospital and Harvard in addition to Canada’s Université Laval. The startup’s lead candidate is designed to enhance the effectiveness of Potassium-chloride co-transporter 2 (KCC2), a protein involved in nerve cell inhibitory transmission. By doing so, it can make nerve cells function normally and help patients who previously didn’t respond to treatments or suffer side effects. The company’s lead program is due to reach clinical testing later this year, with phase 1 results due in early 2025. The round, which was co-led by Cormorant Asset Management and venBio Partners, will be used to take Axonis’ lead programme through clinical proof-of-concept in patients with epilepsy and pain. It will also let the company work on separate pipeline programmes treating psychiatric and neurodevelopmental disorders. “KCC2 is the major central nervous system (CNS)-specific chloride transporter. Unlike ion channels and other targets which are activated by ligands, KCC2 transporter biology is more complex which makes drug discovery difficult,” Axonis CEO Joanna Stanicka told Outsourcing Pharma, adding that the company’s drug discovery technology is backed by high expertise in this protein. googletag.cmd.push(function () { googletag.display('text-ad1'); }); The epilepsy treatment market ​ was worth around $10.15 billion in 2022 and is projected to grow to around $15.35 billion by 2030, with an annual growth rate of 5.1%. This growth is spurred by increasing approvals of new treatments in addition to improved diagnosis approaches. Some of the current drugs in this market include UCB’s Briviact and Pfizer’s Lyrica. There are other efforts ​ in development to target KCC2 such as directly activating the protein with small molecules, enhancing its expression in nerve cells with RNA or the gene editing tool CRISPR. Many startups fighting epilepsy are in the medtech space, such as Epi-Minder, an Australian firm that bagged $16 million in a bridge financing round in 2022 to fund the development of its patient monitoring device. Earlier this year, the U.S. company Neurava closed a seed round of more than $2 million to kick off the development of wearable devices to save the lives of epilepsy patients. “The progress in the precision neuroscience field has enabled major clinical successes in recent years, which has led to multi-billion dollar M&A deals for CNS companies, as well as hugely successful IPOs and private financings recently,” Stanicka said.