Paul Cassar, PhD

Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted and highly selective small molecules to restore cellular metabolic quality control, thereby addressing the dysregulated growth and hyperactive signaling found in certain rare genetic and age-related diseases. Our lead development candidate, AV078, is a first-in-class CNS-penetrant selective mTORC1 inhibitor in Phase 2 development for refractory epilepsy associated with tuberous sclerosis complex (TSC), a rare genetic disorder driven by hyperactive mTORC1 signaling. Beyond AV078, we’ve developed a proprietary library of small molecules, which are potent and selective mTORC1 and CD38 inhibitors that have the potential to precisely target the underlying biology of multiple rare and age-related disease.

Oligon™ is an emerging biotechnology company developing a novel class of multimodal RNA therapeutics that can simultaneously silence numerous disease targets with a single RNA molecule to improve outcomes and combat emerging drug resistance. Our proprietary SeekR™ platform is used to digitally engineer and rapidly produce self-delivering therapeutics which target specific tissues with unprecedented precision and accuracy. We are focused on building a pipeline of products in oncology, yet our multimodal RNA platform can be harnessed to transform outcomes for any complex multigenic disease.

Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The companys approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. Glycomine’s lead drug candidate, GLM101, is a novel substrate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), a rare disease representing a critical unmet medical need.

Thryv Therapeutics Inc. (previously LQT Therapeutics Inc.) is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to treat genetic and drug-induced Long QT Syndromes, atrial fibrillation, and resistant cancers with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit www.thryvtrx.com.

Find Therapeutics is clinical-stage biopharmaceutical company dedicated to the development of next generation therapies to treat inflammatory autoimmune diseases. The company was launched in 2020 and is supported with investments from CTI Life Sciences, adMare BioInnovations, Domain Therapeutics and Investissment Québec and an exclusive license from SATT-Conectus and the University of Strasbourg on a technology and related know-how.

Amolyt Pharma is building on its team’s established expertise to deliver life-changing treatments to patients suffering from rare endocrine and related diseases. Amolyt Pharma’s portfolio includes eneboparatide (AZP-3601), a long-acting PTH1 receptor agonist as a potential treatment for hypoparathyroidism, and AZP-3813, a peptide growth hormone receptor antagonist for the potential treatment of acromegaly. Amolyt Pharma aims to further expand and develop its portfolio by leveraging its global network in the field of endocrinology and with support from a strong syndicate of international investors.

A newly established NewCo developing therapeutics targeting a novel class of tumour-specific antigen.

Aeovian Pharmaceuticals is a clinical-stage biopharmaceutical company developing targeted and highly selective small molecules to restore cellular metabolic quality control, thereby addressing the dysregulated growth and hyperactive signaling found in certain rare genetic and age-related diseases. Our lead development candidate, AV078, is a first-in-class CNS-penetrant selective mTORC1 inhibitor in Phase 2 development for refractory epilepsy associated with tuberous sclerosis complex (TSC), a rare genetic disorder driven by hyperactive mTORC1 signaling. Beyond AV078, we’ve developed a proprietary library of small molecules, which are potent and selective mTORC1 and CD38 inhibitors that have the potential to precisely target the underlying biology of multiple rare and age-related disease.

Oligon™ is an emerging biotechnology company developing a novel class of multimodal RNA therapeutics that can simultaneously silence numerous disease targets with a single RNA molecule to improve outcomes and combat emerging drug resistance. Our proprietary SeekR™ platform is used to digitally engineer and rapidly produce self-delivering therapeutics which target specific tissues with unprecedented precision and accuracy. We are focused on building a pipeline of products in oncology, yet our multimodal RNA platform can be harnessed to transform outcomes for any complex multigenic disease.

Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The companys approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. Glycomine’s lead drug candidate, GLM101, is a novel substrate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), a rare disease representing a critical unmet medical need.

Ability Pharmaceuticals is a clinical-stage biopharmaceutical company committed to develop first-in-class therapies that address unmet medical needs in the oncology space. AbilityPharmas is currently conducting, with ABTL0812, the most advanced asset, an international multi-center, double-blind, placebo-controlled phase 2b clinical trial in patients with metastatic pancreatic cancer to investigate ABTL0812 as first-lin therapy in combination with FOLFIRINOX chemotherapy. The study includes reference centers in Spain, France, United States and Israel. ABTL0812 has finished with very positive results a phase 2a clinical trial as first-line therapy in endometrial cancer and in squamous NSCLC in combination with paclitaxel and carboplatin. The study included leading institutions in Spain and France (Vall d’Hebron Institut d’Oncologia – VHIO, Institut Gustave Roussy, Centre Léon Bérard). ABTL0812 is the first cancer drug killing tumor cells through potent cytotoxic autophagy via inhibition of the Akt/mTOR axis, and the induction of robust endoplasmic reticular stress (ER Stress). Its unique mechanism of action was partially published at Clinical Cancer Research in December 2015 and fully characterized at Autophagy journal in May 2020. The compound has also immunomodulatory effects, turning "cold" immunogenic tumors into "hot" immunogenic. The compound is active as standalone treatment, it has synergy with chemotherapy, and it has synergy with anti PD1 therapies. The product has potential in several cancer types as first-line and maintenance therapy including lung, endometrial, pancreatic, breast, head and neck cancer, glioblastoma, cholangiocarcinoma and the pediatric cancer neuroblastoma. ABTL0812 has been granted Orphan Drug Designation for pancreatic cancer, neuroblastoma, and biliary tract cancer, by the US FDA and the European EMA. Development and marketing rights for Greater China granted to SciClone Pharmaceuticals, Inc.

Thryv Therapeutics Inc. (previously LQT Therapeutics Inc.) is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to treat genetic and drug-induced Long QT Syndromes, atrial fibrillation, and resistant cancers with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit www.thryvtrx.com.