Research & Development
“The greatest enemy of knowledge is not ignorance; it is the illusion of knowledge.”
Stephen Hawking
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.
Research and Innovation in Therapies for Rare and Complex Diseases
Our present research is centered around neuromuscular disorders, fibrosis and cancer (including oncology supportive care) with a special focus on rare diseases. Our R&D group works with small molecules, peptides, and monoclonal antibodies to best suit each target’s biology, but our key strength is our expertise in 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 and the EMA 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.
Enhancing Therapeutic Options: New Formulations and Complex Biosimilars
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 generics/biosimilars of enoxaparin and glatiramer.
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* |
US: Launched 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 Antibody Fibrosis |
In progress | TBC | ||||||
| ITF-undiscl HDAC6 Inhibitor Peripheral Neuropathies |
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. Glatiramer has been approved by FDA, while Pentosan is still under review.
Drug Discovery: Innovative Approaches
At the heart of our discovery engine is the New Drug Incubator (NDI), a multidisciplinary group led by our Chief Scientific Officer (CSO), focused on the discovery and development of New Chemical Entities (NCEs) up to the stage of preclinical candidate (PCC) nomination, i.e., readiness for IND-enabling studies.
For hit identification, lead optimization, and candidate selection, we leverage a broad range of complementary methodologies, including:
Fragment-based screening, conducted in collaboration with external partners. This approach allows us to identify novel chemical matter by analyzing the interaction of small molecular fragments with the target, which are then combined and optimized to achieve the desired pharmacological profile.
Structure-based drug design, also in partnership with external collaborators. This technique enables detailed analysis of the target structure and its interactions with candidate molecules, guiding the rational design of potent and selective compounds.
AI-driven computational drug discovery with Iktos: we use their Makya™ platform for generative molecule design and Spaya™ for retrosynthetic analysis. These tools rapidly generate novel, synthesizable compounds tailored to targets, accelerating discovery timelines.
We are also investing significantly in internal innovation:
The integration of ITForge, our platform designed to unify and manage all in-silico activities, ensures streamlined workflows and enhanced collaboration. ITForge is our end-to-end, AI-driven pipeline developed to support hit-to-lead workflows through generative chemistry and machine learning. ITForge integrates and optimizes multiple open-source frameworks, using reinforcement learning to generate molecules optimized for drug-likeness and other physical-chemical properties. The virtual candidates are subsequently ranked by progressively accurate filtering and scoring methods and selected for synthesis. ITForge provides a flexible, scalable, and open-source solution for AI-driven drug design, integrating generative modeling, multi-objective optimization, and rigorous post-processing to prioritize high-quality candidate molecules for experimental validation.
Recent investments in hardware and software infrastructure, with dedicated NVIDIA GPUs (4x H100, 2x L40S) and a data science software framework to support high-throughput screening, in-silico modeling, and data integration.
In partnership with Istituto Ronzoni , we are acquiring a Bruker 800 MHz NMR spectrometer—the first of its kind in Italy’s private sector and the first ever installed in Northern Italy. This cutting-edge instrument will substantially strengthen our structural biology and compound characterization capabilities, enabling deeper exploration of complex molecular structures and the interactions between proteins and potential drug candidates. Jointly managed, the NMR platform will support Italfarmaco’s internal discovery programs and be available to qualified research partners across Northern Italy, fostering collaboration and accelerating scientific progress. This strategic investment underscores Italfarmaco’s strong commitment to scientific excellence and innovation, while expanding our capabilities in structural biology, molecular analysis, and early-stage drug discovery.
