Research & Development
Italfarmaco is an important player in the Italian pharma industry landscape due to both, its products and its R&D activities.
The first molecule (imidazole salicylate) developed in its Research Center was a non-steroidal anti-inflammatory drug, marketed in 1984. Subsequently, a protein iron complex was developed for the treatment of anemia resulting from iron deficiency, today marketed in over 40 countries worldwide. In 1988, the company began marketing choline alfoscerate, while also overseeing its international development for treating cerebral cognitive disorders, including both vascular and non-vascular Alzheimer’s disease. In the cardiovascular area it was a pioneer in the development of antithrombotic drugs, such as unfractionated and low molecular weight heparin, in the chronic treatment of ischemic heart disease and in the prophylaxis and therapy of venous and arterial thrombosis.
Our present research is centered around neuromuscular disorders, fibrosis and cancer (including oncology supportive care) with a special focus on rare diseases. While our capabilities span three treatment modalities, small molecules, peptides and monoclonal antibodies from which we chose to best match the biology of the selected target, the distinctive feature of the ITF R&D group is its expertise in the field of zinc-dependent histone deacetylase (HDAC) inhibitors. Zn-dependent HDACs are a family of 11 related proteins that are involved in the post-translational modification of proteins and polyamines.
Several HDACs are involved in the control of gene expression and their inhibitors have the potential of repro- gramming cell fates by interfering with these processes. Our most advanced compound, Givinostat (a non-selective HDAC inhibitor), has recently been approved by the FDA for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older, and is also in an advanced stage of clinical development in Polycythemia Vera, a rare hematologic cancer. Our R&D group is working to identify exquisitely subtype-selective, second-generation inhibitors to specifically interfere with a defined subset of biological processes controlled by HDACs.
In addition to the development of new chemical entities, Italfarmaco’s R&D group also develops new formulations of established drugs to better respond to patient needs and generics/biosimilars of complex molecules. Examples developed up to market in Europe, USA and Rest of the World are the liquid formulations of antidepressants or hypnotic agents and of Riluzole for the treatment of Amiotrophic Lateral Sclerosis (ALS). Regarding the development of generics/biosimilar of complex molecules, Italfarmaco’s R&D group has developed a generic/biosimilar of enoxaparin which is currently marketed in Europe, USA and Rest of the World.
Our Focus
The goal of Italfarmaco’s research activity is to provide caregivers and patients with effective and safe drugs, to improve the health and to ensure a better quality of life of patients.
Our Focus
The goal of Italfarmaco’s research activity is to provide caregivers and patients with effective and safe drugs, to improve the health and to ensure a better quality of life of patients.
Our path to R&D journey
Italfarmaco’s Research and Development (R&D) is organized to bring innovation to patients and to swiftly progress our discoveries from early conceptualization all the way down to market approval in the shortest timeframe possible. To optimize this process, the group is divided into two main departments: the New Drug Incubator (NDI) and the Drug Development (DD) units.
The R&D department of the Italfarmaco Group is primarily based in Milan, with additional units in Rome and Madrid. For the chemical development and manufacturing of molecules, we rely on Chemi, a leading active ingredient manufacturer within our group.
Our R&D department is dedicated to driving scientific excellence and innovation, working collaboratively across specialized units to bring groundbreaking therapies to the market.
Research Focus Areas
Our pipeline currently encompasses a range of ongoing discovery and development projects, with a primary focus on epigenetics. Specifically, we are targeting the histone deacetylase (HDAC) family of enzymes.
This diversified approach underscores our dedication to addressing a broad spectrum of medical challenges through cutting-edge scientific research and development. Our efforts are directed towards several key clinical indications.
Rare Diseases
We have developed expertise in neuromuscular disorders, aiming to provide novel therapeutic options for these challenging conditions.
Fibrosis
Our research is exploring innovative approaches to treat various fibrotic diseases, seeking to address unmet medical needs in this area.
Oncology
We are committed to advancing cancer treatments, focusing on both direct anti-tumor therapies and supportive care for oncologic patients, to improve overall patient outcomes and quality of life.
Pipeline
| Lead Optimization |
Candidate Selection |
IND Enabling |
Phase I |
Phase II |
Phase III |
NDA MAA |
Expected Launch |
|
|---|---|---|---|---|---|---|---|---|
| Givinostat Pan-HDAC Inhibitor Duchenne Muscular Dystrophy* |
NDA Approved MAA under review |
US: 2024 EU: 2025 |
||||||
| Givinostat Pan-HDAC Inhibitor Polycythemia Vera* |
In progress | 2027 | ||||||
| Givinostat Pan-HDAC Inhibitor Becker Muscular Dystrophy* |
TBC | |||||||
| ITF3756 HDAC6 Inhibitor Solid Tumors |
In progress | 2028 | ||||||
| ITF3912 Peptide-Drug Conjugate Solid Tumors |
In progress | TBC | ||||||
| ITF-undiscl HDAC6 Inhibitor Peripheral Neuropathies |
In progress | TBC | ||||||
| ITF-undiscl Antibody Fibrosis |
In progress | TBC | ||||||
| ITF-undiscl HDAC Class I Inhibitor Fibrosis |
In progress | TBC |
Italfarmaco’s R&D group is not only dedicated to discovering and developing new chemical entities (NCEs) but is also engaged in developing complex generics and biosimilars. In recent years, the company successfully launched enoxaparin in the US and the EU. Currently, the ANDAs for Glatiramer and Pentosan are under review by the FDA.