Therapeutic Focus
In order to maximize the impact of our investments in R&D and increase our chances of success in discovering and developing new medical therapies, we focus our expertise on specific therapeutic areas. In our R&D core areas of Oncology, Neurodegenerative Diseases and Rheumatology, we are exploiting synergies in disease mechanisms and biological pathways.
We employ the whole array of drug-like molecules at our disposal as potential therapeutic agents - from antibodies, fusion proteins, engineered hormones, nanobodies and protein binders to small molecules. We leverage advanced technology platforms such as gene cloning and X-ray crystallography in our daily work. Expertise in recombinant biotechnology and chemical synthesis of molecules allows us to combine the best of both worlds in transforming promising scientific concepts into innovative medicines. Our objective is to bring the best compound to the clinic as quickly as possible, which is why we aim for proof-of-concept with a confirmed mode of action in patients at an early stage.
In addition to the drug candidates now being evaluated in clinical trials, we have more than 70 pre-clinical research projects. During all phases of discovery, our work is supported by strong portfolio and business development. A network of alliances between Merck Serono and biotech companies, clinical research groups and academic institutions, fosters a constant flow of innovations that enriches our disease knowledge, compound libraries and technological capabilities directed towards finding the next generation of breakthrough therapies.
At Merck Serono, we have embraced the principle of the personalized approach in our oncology strategy early on. The principle centers on biomarker-guided drug development. Our goal is far-reaching: to identify the patients who will benefit most from a specific treatment in order to achieve the best possible medical outcome.
A second principle on which we focus in oncology is “Combination is key”: We believe that combination is key in oncology for the following aspects:
In order to optimize therapeutic success, Merck Serono combines approaches that target the following three areas of the disease process:
Targeting the tumor cell
The elimination of the tumor cell remains the essential goal for cancer therapies. Given that there are a variety of ways to achieve this goal, Merck Serono's oncology pipeline includes therapies with a wide range of expected actions against tumor cells, especially interference with proliferation and survival pathways, offering efficacy against many different tumor types.
Targeting the tumor environment
Tumor cells are vulnerable not only to the direct effects of cancer therapies, but also to strategies that render their environment hostile. Tumor cells secrete angiogenic factors into their environment that activate endothelial cells and promote capillary sprouting in order to establish new vasculature. Angiogenesis can be blocked by inhibiting the release of pro-angiogenic factors or by blocking integrin-mediated pathways, which play a fundamental role in endothelial cell proliferation. Blocking angiogenesis denies the tumor its principal means of creating a sustaining environment, and reduces the potential for metastases.
It is becoming increasingly clear that the tumor environment is an important contributor to the greatly reduced dependence on exogenous growth stimulation of tumor cells.
Targeting the immune system
Immunotherapies that enhance the ability of the patient's own immune system to fight cancer are another central area of research and development for Merck Serono Oncology.
A second principle on which we focus in oncology is “Combination is key”: We believe that combination is key in oncology for the following aspects:
- Develop products that target multiple aspects of the disease process and are suitable for use in combination with established standard therapies
- Combine innovative thinking with intelligent research and technical excellence: Merck Serono has brought together a dedicated group of individuals who can look beyond conventional horizons to explore further possibilities
- Combine insights, experience and skills: Merck Serono is committed to working in partnership with oncology experts, patients and healthcare providers
- The tumor cell directly
- The tumor’s local environment
- The patient’s immune system
The elimination of the tumor cell remains the essential goal for cancer therapies. Given that there are a variety of ways to achieve this goal, Merck Serono's oncology pipeline includes therapies with a wide range of expected actions against tumor cells, especially interference with proliferation and survival pathways, offering efficacy against many different tumor types.
Targeting the tumor environment
Tumor cells are vulnerable not only to the direct effects of cancer therapies, but also to strategies that render their environment hostile. Tumor cells secrete angiogenic factors into their environment that activate endothelial cells and promote capillary sprouting in order to establish new vasculature. Angiogenesis can be blocked by inhibiting the release of pro-angiogenic factors or by blocking integrin-mediated pathways, which play a fundamental role in endothelial cell proliferation. Blocking angiogenesis denies the tumor its principal means of creating a sustaining environment, and reduces the potential for metastases.
It is becoming increasingly clear that the tumor environment is an important contributor to the greatly reduced dependence on exogenous growth stimulation of tumor cells.
Targeting the immune system
Immunotherapies that enhance the ability of the patient's own immune system to fight cancer are another central area of research and development for Merck Serono Oncology.
At Merck Serono, our strength in immunomodulation and neurobiology research gives us a strong position for the development of disease-modifying therapies and improved symptom treatment. Studies are in progress to evaluate our multiple sclerosis therapy Rebif® (interferon beta-1a) in related indications such as clinically isolated syndrome (CIS) – intermittent neurological “attacks” that resemble those of multiple sclerosis. Our expertise in recombinant biotechnology has allowed us to develop an improved formulation of Rebif® that reduces injection-site reactions. Dedicated teams are also working on a long-acting form of interferon beta-1a.
In our pre-clinical pipeline, we are collaborating with the company Bionomics on compounds targeting the potassium ion channel Kv1.3, a key modulator of the immune system and a target found on human immune cells associated with nerve cell damage in patients with multiple sclerosis. Inhibitors of Kv1.3 have been shown to inhibit the proliferation of these immune cells.
In our pre-clinical pipeline, we are collaborating with the company Bionomics on compounds targeting the potassium ion channel Kv1.3, a key modulator of the immune system and a target found on human immune cells associated with nerve cell damage in patients with multiple sclerosis. Inhibitors of Kv1.3 have been shown to inhibit the proliferation of these immune cells.
Merck Serono's strength in biotherapeutics and immune-based therapies opens up possibilities for novel therapeutic approaches in Rheumatology, which focuses on joints, connective tissues and certain autoimmune diseases. Our research is centered on discovering drugs that modulate key pathogenic mechanisms in autoimmune and inflammatory diseases. We combine approaches that target the B-cell biology, T-cell biology, cytokines and chemokines pathways and tissue biology/repair mechanisms.
Innovative projects in our R&D pipeline in this therapeutic area include FGF 18, a recombinant protein that increases cartilage cell proliferation and production of extra-cellular matrix components - leading to repair and reconstruction of cartilaginous tissue. FGF 18 is being studied in both osteoarthritis (OA) and cartilage injury repair (CIR). Another innovative drug candidate is the soluble fusion protein atacicept, which is being studied in the autoimmune disease systemic lupus erythematosus (SLE). Other diseases under study in this therapeutic area include rheumatoid arthritis, lupus nephritis and Sjögren's syndrome.
Innovative projects in our R&D pipeline in this therapeutic area include FGF 18, a recombinant protein that increases cartilage cell proliferation and production of extra-cellular matrix components - leading to repair and reconstruction of cartilaginous tissue. FGF 18 is being studied in both osteoarthritis (OA) and cartilage injury repair (CIR). Another innovative drug candidate is the soluble fusion protein atacicept, which is being studied in the autoimmune disease systemic lupus erythematosus (SLE). Other diseases under study in this therapeutic area include rheumatoid arthritis, lupus nephritis and Sjögren's syndrome.
Merck Serono has a history as a pioneer in fertility treatments. Our first injectable human gonadotropin (Pergonal®) was approved in Italy in 1950. The launch of this reproductive hormone in the mid-1960s in the rest of Europe and in the 1970s in the United States made the treatment of fertility problems due to ovulatory dysfunction possible for the first time on a large scale. In the 1990s, we were at the forefront of recombinant biotechnology to produce purer formulations of reproductive hormones.
Today, Merck Serono continues to be a world leader committed to innovation in fertility. The objective of our research and development work is to help couples with conception problems at every stage of the reproductive cycle - from follicular development to early pregnancy - to realize their dream of having a child.
Today, Merck Serono continues to be a world leader committed to innovation in fertility. The objective of our research and development work is to help couples with conception problems at every stage of the reproductive cycle - from follicular development to early pregnancy - to realize their dream of having a child.
In Endocrinology, Merck Serono pursues focused development to respond to significant unmet medical needs in growth hormone disorders and metabolic indications. For example, we are currently conducting clinical studies on a long-acting form of recombinant growth hormone, which would require fewer injections for patients. Our approach to stratified medicine is also being applied to this therapeutic area – the PREDICT study is focused on finding biomarkers to determine precise dosing in pediatric indications for growth hormone therapy.
