List of Small Molecule Pharmaceuticals Companies in Australia - 29

Founded in 2003, Base Pharmaceuticals has been a beacon of innovation and quality in the healthcare industry. Our logo, "Reason for Being," symbolizes our dedication to providing life-saving and life-enhancing pharmaceutical solutions. We are committed to improving the quality of life for individuals and communities across North India.

Beroni Group is an international biotechnological company listed on the National Stock Exchange (Australia) and traded on the OTC markets in the U.S.A. with business presence in Australia, China, Japan and the USA. It currently has four core businesses – cell therapies, developing new anti-cancer drugs, e-commerce platform for pharmaceutical and healthcare products, and detection & diagnosis of infectious diseases. Beroni Group’s overall strategic goal is to become a world’s leading enterprise in the biotechnology, life sciences, and environmental science industries.

Bionomics is a global, clinical stage biopharmaceutical company focused on leveraging its ion channel and chemistry platform to develop novel and first-in-class ion channel modulators to treat patients suffering from central nervous system disorders and cancer.

Clarity Pharmaceuticals is a clinical stage radiopharmaceutical company developing next-generation theranostic (therapy and imaging) products. It was founded in 2010 and is based in Australia. The company focuses on addressing the growing need for the use of radiopharmaceutical therapies in the treatment of serious diseases.

ENA Respiratory is a clinical stage pharmaceutical company, developing INNA-051, a topical, broad spectrum antiviral immunomodulator for pre- and post-exposure prophylaxis of respiratory viral infections

Formulytica is a specialty contract research, development and manufacturing organisation (CRDMO) with extensive expertise in formulation, analytical method development, and manufacture for a wide variety of topical and injectable dermatology, pharmaceutical, cosmetic and agricultural products. The laboratory is attached to a FDA, TGA and APVMA registered manufacturing plant that allow us to deliver unmatched product development and chemistry, manufacture and control (CMC) services including clinical and stability registration batch preparation. Formulytica is staffed with research scientists with an average 20 years' experience in the pharmaceutical industry, each now making their skills and experience available to other companies to develop or register new products.

We are a global biotechnology company specialising in ingestible gel technologies for humans and animals, covering the pharmaceutical, healthcare, nutrition & sport sectors. Our proprietary technology has been designed to assist with the delivery of APIs (active pharmaceutical ingredients) or nutritional compounds via an easy to consume gel based formulation. We provide a unique gel based technology for clients who are looking to offer an alternative to pills, powders and capsules. If you are looking to innovate the way you can deliver improved health to your clients, please get in touch.

Founded in 1975, IDT Australia is a publicly listed Australian pharmaceutical manufacturing company, based in Boronia Victoria. Our experienced and awarded team of scientists and specialists provide a flexible and comprehensive service, focussed in our three strategic pillars: Active Pharmaceutical Ingredient synthesis design, scale up and cGMP manufacture including high potent molecules Specialty Orals novel and early stage design, formulation and cGMP manufacturing specifically targeting neuro disorders Advanced Technology, complex formulations for novel injectable drug development through to cGMP manufacture. This pillar includes RNA and Antibody Drug Conjugates. IDT Australia also has a fully equipped laboratory approved to TGA and FDA standards to develop, validate and operate GLP analytics for release, process validation and stability. IDT Australia has labelling and packaging capacity and capability and specialises in clinical trial labelling and packing.

Immutep is a globally active biotechnology company that is a leader in the development of LAG-3 related immunotherapeutic products for the treatment of cancer and autoimmune disease. Immutep is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximize value to shareholders.

Incannex is a clinical-stage pharmaceutical development company focused on discovering and developing life-changing medicines for patients struggling with serious, chronic diseases and limited treatment options. A leader in innovating proprietary cannabinoid pharmaceuticals and psychedelic-based therapies, we are developing medicines with the potential to alter the lives of patients suffering from obstructive sleep apnea (OSA), generalized anxiety disorder (GAD), and inflammatory disorders such as rheumatoid arthritis (RA). Working together in these relatively new fields with clinical, scientific, and drug development experts, we are efficiently innovating big solutions that are specifically designed and formulated to make a meaningful difference in patients' lives. Founded in 2015 and headquartered in Melbourne, Australia, we are expanding in the U.S. and worldwide to help more patients and their families. Incannex trades on the Nasdaq Global Market under the symbol IXHL.

Invion is a clinical-stage life-sciences company that is leading the global clinical development of the Photosoft™ technology for the treatment of cancers. Invion has been appointed exclusive distributor and licensee in Australia and New Zealand of Photosoft™. The appointment has been made by technology licensor, The Cho Group, a Hong Kong based group that has funded and successfully commercialised a number of unique and advanced technologies. Via an R&D services agreement between the two entities, the research and clinical trials of Photosoft™ are funded by The Cho Group. Invion has an alliance with leading Australian medical research institute, Hudson Institute of Medical Research, for the Photosoft™ research program.

Island Pharmaceuticals is a drug research and repurposing company, focused on developing preventative or therapeutic drugs for viral infections. We have a lead asset – ISLA-101 - that was initially developed by our wholly owned subsidiary, Isla Pharmaceuticals. ISLA-101 is a drug with a well-established safety profile, being repurposed for the prevention and treatment of dengue fever and other mosquito (or vector) borne diseases.

Kazia Therapeutics Limited, an oncology-focused biotechnology company, develops anti-cancer drugs. Its lead development candidate is GDC-0084, a small molecule, brain-penetrant inhibitor of the PI3K/Akt/mTor pathway, which is developed as a potential therapy for glioblastoma. The company is also developing Cantrixil, which is being developed for the treatment of ovarian cancer and is in hospitals across Australia and the United States under an Investigational New Drug application. Kazia Therapeutics Limited has collaboration agreements with St Jude Childrens Hospital; Dana-Farber Cancer Institute; Alliance for Clinical Trials in Oncology; Memorial Sloan Kettering Cancer Centre; and University of Newcastle. Kazia Therapeutics Limited has collaboration with Dana-Farber Cancer Institute to investigate the use of Kazia’s investigational new drug, paxalisib, in primary central nervous system lymphoma; and Kintara Therapeutics, Inc. for the activation of paxalisib and VAL-083. The company was formerly known as Novogen Limited and changed its name to Kazia Therapeutics Limited in November 2017. Kazia Therapeutics Limited was founded in 1994 and is based in Sydney, Australia.

Kinoxis Therapeutics Pty Ltd is a private, Australian-based, clinical-stage biotechnology company developing first-in-class therapeutics to address the escalating demand for safe and effective treatments for behavioural and psychological symptoms in neuropsychiatric disorders. Kinoxis’ lead candidate, KNX100, is being developed for the treatment of behavioural and psychological symptoms in neuropsychiatric disorders, including agitation and aggression in dementia, and several substance use disorders. KNX100 has a novel mechanism of action, interacting with disease relevant pharmacological, molecular, and neural targets, to modify the dysregulated brain systems linked with specific behavioural and psychological symptoms in neuropsychiatric disorders. The compound has an extensive pre-clinical data package consisting of multiple cross-species animal efficacy models conducted across several independent research organisations. Kinoxis has completed a Phase 1 study conducted under a US IND and have demonstrated KNX100 to be safe and tolerable in healthy volunteers. Currently, KNX100 is being investigated in the CARES-X Phase 2 clinical trial, a multi-site study in Australia focused exploring its safety, tolerability, and efficacy as a treatment for agitation in dementia. Kinoxis is also advancing clinical programs targeting stimulant, opioid, and alcohol use disorders. Notably, the alcohol use disorder program is being developed in collaboration with the National Institute on Alcohol Abuse and Alcoholism (NIAAA), while the opioid use disorder program has received significant funding from the US National Institute of Health/National Institute on Drug Abuse (NIH/NIDA). In addition to the KNX100 program, Kinoxis have a strategic partnership and licensing agreement with Boehringer Ingelheim to develop first-in-class oxytocin receptor targeting precision psychiatry treatments to improve the quality of life of people living with neuropsychiatric disorders.

Kira Biotech is an emerging Australian biotechnology company developing novel immunomodulatory compounds for the treatment of immune system disorders. Its lead candidate, KB312, is a first-in-class, selective, immune-cell depleting monoclonal antibody which targets activated immune cells and aims to restore homeostasis through induction of immune tolerance. Kira Biotech has attracted venture capital funding and is progressing KB312 through preclinical development and phase 1 clinical trials with a team of drug development experts led by well-known US-based rheumatologist and immunologist, Dr Dan Baker.

Lateral Pharma Pty Ltd Is a privately-owned Melbourne AU-based biotechnology company that commenced operations in May 2015 to repurpose LAT8881, a Phase 2 ready orally-available compound with extensive toxicology and human safety data, into new high potential pain and respiratory indications. Lateral aims to demonstrate efficacy in a human clinical Phase 2 proof-of-concept study using LAT8881 to treat neuropathic pain within the next 12 months. Lateral Pharma has discovered a new class of naturally-derived peptide motifs and their novel target family of proteins that restore normal tissue function in mammals under conditions of damage and cellular stress. Small synthetic peptides derived from growth hormone and at least three other proteins are active in animal models of neuropathic pain, osteoarthritis, obesity, COPD, severe influenza A and recently in initial data in SARS-CoV-2. Lateral has filed multiple patents protecting its intellectual property Lateral is focussed on developing its drugs for the treatment of Neuropathic pain but subject to additional funding will also explore development in respiratory diseases such as Influenza, SARS-CoV-2, COPD, Steroid resistant asthma, and Post-COVID Syndrome (Long-COVID).

LBT Innovations is a medical technology company that utilizes artificial intelligence to deliver automation in healthcare through a patent-protected machine learning and intelligent imaging platform.

OccuRx is a biotechnology company that specializes in developing innovative precision therapies for the treatment of inflammatory and fibrotic diseases.

Percheron Therapeutics Limited (ASX: PER) is an international biotechnology company focused on the development of novel therapies for rare diseases. The company's lead program is ATL1102, an antisense oligonucleotide targeting the CD49d receptor. ATL1102 is currently the subject of an ongoing international phase IIb clinical trial for the treatment of non-ambulant patients with Duchenne Muscular Dystrophy (DMD), for which data is expected in 2H CY2024. The drug has previously reported promising results from an exploratory phase IIa study in the same population and has been awarded orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the US FDA. For more information, please contact info@PercheronTx.com.

Prescient Therapeutics (ASX: PTX) is a clinical stage oncology company developing personalised medicine approaches to cancer, including targeted and cellular therapies. Cell Therapies OmniCAR: is a universal immune receptor platform enabling controllable cell activity and multi- antigen targeting with a single cell product. OmniCAR’s modular CAR system decouples antigen recognition from the cell signalling domain. It is the first universal immune receptor allowing post- translational covalent loading of binders to immune cells. OmniCAR is based on technology licensed from Penn and Oxford University; and other assets. Prescient is developing OmniCAR-T cells programs for next-generation CAR-T therapies for AML; Her2+ solid tumours, including breast, ovarian and gastric cancers; and GBM. CellPryme: Prescient's novel, ready-for-the-clinic, CellPryme-M technology enhances adoptive cell therapy performance by shifting T and NK cells towards a central memory phenotype, improving persistence, and increasing the ability to find and penetrate tumours. CellPryme-M is a 24-hour, non-disruptive process during cell manufacturing. Targeted Therapies PTX-100 is a first in class compound with the ability to block an important cancer growth enzyme known as geranylgeranyl transferase-1 (GGT-1). It disrupts oncogenic Ras pathways by inhibiting the activation of Rho, Rac and Ral circuits in cancer cells, leading to apoptosis (death) of cancer cells. PTX-100 is now in a Phase 1b expansion cohort study in T cell lymphomas, where it has shown encouraging efficacy signals and safety. PTX-200 is a novel PH domain inhibitor that inhibits an important tumour survival pathway known as Akt, which plays a key role in the development of many cancers, including breast and ovarian cancer, as well as leukemia. Unlike other drug candidates that target Akt inhibition, PTX-200 has a novel mechanism of action that specifically inhibits Akt without non-specific kinase inhibition effects.

Prota Therapeutics is an Australian biotech company established in 2016, dedicated to developing and commercializing cutting-edge oral immunotherapy treatments for food allergies. They have achieved significant quality of life improvements and clinical remission of peanut allergy in pediatric patients, with a 51 percent peanut allergy remission rate. The company has also secured a $21 million financing led by SPRIM Global Investments.

Psylo is a drug development company focused on next generation psychedelics; our goal is to make broadly accessible medicines as improved treatments to currently available SSRI medications, using psychedelic molecules as the starting point. We are building a pipeline of novel molecules with the ultimate goal of taking leading candidates through to clinical trials.

Radiopharm Theranostics is developing a world-class platform of radiopharmaceutical and nuclear medicine products for both diagnostic and therapeutic applications. The company is focused on innovating the way we see and treat cancer, with a particular emphasis on HER-2 cancers. Radiopharm Theranostics has a strong team of executives and board members, and is actively engaged in investor relations and corporate governance. The company has also launched a joint venture with MD Anderson to focus on novel radiopharmaceuticals.

Recce Pharmaceuticals Ltd (ASX: RCE, FSE:R9Q) is pioneering the development and commercialisation of a new class of synthetic antibiotics with broad spectrum activity designed to address the urgent global health problem of antibiotic resistant superbugs. Its patented lead candidate known as RECCE® 327 has been developed for the treatment of blood infections and sepsis derived from E. coli and S. aureus bacteria – including their superbug forms. Pre-clinical testing in laboratories and animal models, in Australia and overseas has demonstrated positive results to date. Recce Pharmaceuticals has manufacturing facilities in Australia and clinical research partners in the USA. Recce Pharmaceuticals has validated an automated process to manufacture its lead compound ahead of first-in-human clinical trials.

We are a biopharmaceutical research and production company, based on the Sunshine Coast, Queensland. Established in 2012, we research, develop and produce high-quality, innovative, medical grade biopharmaceuticals based on the latest scientific research. Servatus has two drug research platforms: Engineered proteins for the treatment of autoimmune diseases, and microbial biotherapeutics for the treatment of inflammatory conditions and bacterial infections. Our protein research focuses on improving specific characteristics of existing therapeutic proteins. We carefully select proteins with clear clinical utility, and through rational redesign of key features, we improve bioavailbility and biological potency. We then rapidly clone and express the proteins in amounts suitable for clinical use, thus providing faster, more effective treatment of chronic auto immune diseases, such as rheumatoid arthritis and psoriasis, with fewer undesirable side effects. In parallel with our protein work, we also develop live microbial biotherapeutic treatments for microbiome-associated conditions, including but not limited to inflammatory bowel disease (IBD), arthritis, atopic dermatitis, and gastrointestinal infections (for example, C. diff and H.pylori infections). We utilise bacterial strains that have not been widely employed previously, but have demonstrated bioactive properties in the latest scientific research. These bacteria have been shown to inhibit pathogenic bacterial growth and infection, modulate immune responses, and regulate inflammatory signals. Bacterial candidates are developed to act directly at specific sites to treat specific ailments and diseases. Servatus currently holds patents for numerous microbial biotherapeutics and engineered proteins for autoimmune disease treatments Servatus Ltd derives its name from the patron saint St Servatus, a 4th century churchman and diplomat, who was invoked as a saint for his healing powers for a range of ailments. #biotech

In collaboration with RMIT University, Snoretox is developing the world’s first injectable solution to treat obstructive sleep apnoea and other conditions involving low muscle tone. Our game-changing technology is based on molecules of tetanus toxin, modified to ensure they work around the tetanus vaccine. Just as Botox uses tiny amounts of botulinum toxin to relax muscles, our prototype therapeutic uses minute amounts of safe modified tetanus toxin to achieve the opposite effect – muscle toning.

SYNkinase's mission is to provide life-science researchers with access to the world's most up-to-date library of kinase inhibitors, and other small molecule inhibitors of important therapeutic proteins, for drug-discovery research use. With guidance from our founders, that include kinase biology scientists and expert medicinal and synthetic chemists, we have created a library of potent, pure and research-relevant inhibitors directed against specific protein serine/threonine kinases (PS/TKs), protein tyrosine kinases (PTK & RTK), as well as lipid kinases. There are more than 150 Kinase targeted drugs in clinical trials or development at present. Twenty four small molecule inhibitors have been FDA-approved including mTOR, B-Raf, MEK, EGFR, JAK, VEGFR and PI3K inhibitors. SYNkinase has many of these compounds available for sale as well as a large range of inhibitors described in the literature such as GPCR and epigenetic inhibitors. Importantly, as we are a primary source manufacturer we can maintain a fastidious focus on quality, as well as being first in the market with the most important and topical compounds. SYNkinase offers its inhibitor collections in several formats, including pre-formatted and custom arrays.

Telix is a biopharmaceutical company focused on the development and commercialisation of therapeutic and diagnostic ('theranostic') radiopharmaceuticals. Telix is headquartered in Melbourne, Australia with commercial operations in the United States, Europe (Belgium and Switzerland) and Japan. Telix is developing a portfolio of radiopharmaceutical products that aims to address significant unmet medical need in oncology and rare diseases. Telix is listed on the Australian Securities Exchange (ASX: TLX).

TGR BioSciences is an Australian biotechnology company, specializing in the development and manufacture of immunoassays and immunoassay technologies. TGR's CaptSure technology underpins its immunoassay products and is available for license.