The Boardroom Series: Interview with Orphazyme's CMO Carlos Camozzi

Dr Carlos Camozzi is the new Chief Medical Officer at the Biotech Company Orphazyme, located in Denmark. Carlos has over 25 years in clinical research and development roles in pharma and biotech companies, including leading clinical operations in the rare disease area. Before joining Orphazyme, Dr Camozzi was the VP CMO of uniQure (ex AMT) in Netherlands, and prior to that, he was Medical Director and Head of Medical Affairs with Orphan Europe.

 The landscape for the Pharma industry has changed, both from a scientific and regulatory standpoint. Business models must change in order to stay relevant and deliver the best results for patients. In this Interview Carlos Camozzi argues that a future based on finding the next blockbuster will be difficult and that real advances can be made by shifting focus onto diagnostics and treatments for rare diseases.


Pharma IQ: What current projects are you concentrating on at Orphazyme?

 Carlos Camozzi: We have been looking at the pioneering of a very special protein, called Heat Shock Protein, and, based on that molecule; the therapies will become leading projects for the company. We are also tackling rare diseases, which will lead us into the development of orphan drugs. So, we are dedicated to that development, trying to change the different points already demonstrated in medicine, to improve the lives of those patients, and their families.



 Pharma IQ: In a recent interview, the CEO of Novartis said the big pharma business models need to be updated. What are your thoughts on this as the definition of the blockbuster changes?

 Carlos Camozzi: In the past blockbusters generated $1 billion per year for big pharma companies. This target has become more difficult to be achieved, because discovering new molecules that are going to be a breakthrough in treatment, is more complex, complicated and expensive, so the challenges are there.

 Those challenges are primarily located in research and development, trying to find real innovation but apparent improvement of molecules. It will demand a complex selection of the diseases intended to treat with the new product. There is a need for efficiency in development, improved regulatory pathways and more precise guidelines and rules to review the files clinical and scientific data. New and creative financial models should prove to be sustainable or adapted to different kinds of companies, different kinds of diseases and, at the same time, different kinds of treatments.

 Pharma IQ: In your opinion, what do pharma companies need to start doing in order to reverse the problem of antibiotic resistance?

 Carlos Camozzi:  I think there is a need for more extensive research. I believe that there is a need to put together all the stakeholders, and find solutions in these kinds of situations. That could be the example that you mention in antibacterial, but it could be repeated in many other drugs for other treatments.

 Pharma IQ: Carlos, What do you see as the biggest challenges in drug development right now? What has your experience been across uniQure and now in Orphazyme?

 Carlos Camozzi: I think that the latest experiences are showing that the main two challenges are around innovation and the need for changes or an update on the regulatory process. Innovation in research and development will create more extensive scientific understanding of the diseases and the pathological pathways that define the diseases. Therefore, the translation of the non-clinical information to be applied in clinical research and development is the key to effective and safe treatments.

 In the regulatory area, there are conditions where evolution and maturation is needed. At the same time, more dynamic interaction with knowledge centres is required in order to be able to move a significant step towards the newer scientific concepts.

 Pharma IQ: What regulations are coming forward, in 2013, where pharma industries and, indeed, biotech industries need to prepare themselves for?

 Carlos Camozzi:There are many changes that have been applied. One of the most important recently applied in Europe is related to pharmacovigilance.

 I have seen the development of orphan drugs evolving towards a dramatic change in the regulations, standards, and guidelines. At the same time, a remarkable change in regulations.

 In the near future, there is going to be an evolution of these regulations, which will generate a positive impact in research and development of innovative therapeutic options. We will see updated regulations which are quicker and in accordance with new discoveries and statements that are coming from clinical research together with non-clinical development.

 Pharma IQ: What changes do you predict in medicine in the next ten years?

 Carlos Camozzi: The trend is that patients are going to be treated in a more individual way. As the same disease is expressed differently from patient-to-patient, the response to treatment is going to be different from patient-to-patient. It’s going to be more of an individualised treatment, more tailor-made according to the profile of the patient. So the advance in diagnoses is the most important point here, and then this should move even quicker than today.

 This trend, especially in the area of rare diseases, may modify completely the traditional and conventional concept of the “blockbuster”.

 Another subject to be considered is certain diseases or clinical conditions that require combined therapeutic interventions, which mean more than one product, would be indicated for certain specific diseases.

 This scenario is relatively common in the oncological therapies, and it seems that doing the same kind of therapeutic approach would also apply to other diseases. Therefore, the cost of drugs may initially be impacted but the treatment efficiency will be better, and then I could say that the health cost of the individual treatment, the complete treatment, should be not much higher at the end.

 Pharma IQ: Carlos, you have over 25 years' experience in clinical research and development roles in big pharma and, indeed, more recently in biotech. Where do you enjoy working the most?

 Carlos Camozzi:Clinical research and development place the same kind of challenges to big and small pharma and biotech companies. I don't think that the challenges are going to be different. So, enjoyment comes from the possibility of making a significant or positive impact in the life of every individual patient that you may have in mind.

 The potential therapeutic benefit of the drugs in development today is the most motivating factor, at least for me, and the challenges in small companies are related to the limited human and financial resources. This reality obliges you to be extremely efficient to use those resources in a very productive way, to comply with regulatory requirements more precisely. It demands more creative and innovative clinical development plans and design before clinical trials start. Therefore, frequent interaction and consultation with health authorities from the very beginning are mandatory.

 Pharma IQ; Carlos, what are you hoping to achieve at Orphazyme over the next, say, five years?

 Carlos Camozzi:Improvement in diagnosis of rare diseases and collaborating with the groups of experts. The second is to be closer to the needs of the patients in order to achieve a more interactive and closer feedback from those groups, I mean patient associations. If I can achieve that and bring in useful, effective and safe therapeutic options, for patients who could die 24 hours after the diagnosis of the disease, I will be very satisfied.

 Pharma IQ:  It's such an amazing job, to be able to say you have the possibility of doing that, Carlos. What challenges lie ahead around orphan diseases and drug development for you right now?

 Carlos Camozzi:Trying to develop orphan drugs for rare diseases, the challenges are directly linked to the rarity of the diseases. You have very few patients, and there is no complete knowledge and understanding of the disease. Therefore, challenges are the size of the population for the clinical trials design, complexity of statistical analyses and regulatory requirements. So, there is a need to change that.

 There is a significant lack of scientific and clinical knowledge about diseases, especially around the diseases that we are talking about, and this is negatively impacting the selection of validated endpoints. That is the main challenge today for clinical development and regulatory purposes.

 Pharma IQ: So what advances are being made around Orphan Drugs and what are you most proud of in your career?

 Carlos Camozzi:I think that there were significant advances in regulations and in local health authorities. That, in fact, facilitated the improvement and the processes of research and development of different therapeutic options for rare diseases and clinical development in the paediatric population. These two were the main advances.

 The genetic profiling of patients facilitated the appropriate selection of those to be included in clinical trials. Patient associations are actively participating with the researchers, the industry, the regulators and the lawmakers, and there is a clear picture now in Europe and United States.

 We also have an increased number of diagnostic tools that are going to facilitate the identification of the patient. I am feeling proud of all of them, but from my personal point of view, from a career point of view, I was able to participate in different developments and different processes just to bring some few products into the market for the treatment of the paediatric population for very rare and life-threatening conditions.

 Pharma IQ: Carlos, what exciting changes do you anticipate in clinical research between now, say, 2020?

 Carlos Camozzi: As I mentioned before, there is going to be a more precise identification of the disease, and then we will bring more efficient treatment for each one of the patients.

 The second point is that we are going to see a myriad of different possibilities for treatment in the next seven to ten years. We know that with our different advanced therapies, this is a revolution, but these are not going to show all the solutions for the treatment of the diseases, especially in the rare diseases area. But there are other molecules, other proteins and so on, which the investment models and the grants for research to better understanding is going to be the real revolution within the next ten years.

 Pharma IQ: What are the biggest challenges around capital and funding in the biotech industry?

 Carlos Camozzi:I think that funding new scientific ideas is not easy and it's placing a lot of challenges on those who are deciding where to fund or invest. I think that reliable research results and development plans are necessary to capture the interest of venture capitalists. Research grants or a partnership with other companies are other financial options.  So advancement in the research and development programmes with encouraging meaningful data will differentiate companies to invest in.

 Pharma IQ: Can you perhaps just give us a personal reflection as to the main hurdles you face in your day-to-day role?

 Carlos Camozzi:I feel that every minute that I miss not thinking the right way; I am risking the life of a patient. So, for me, it's extremely important that all the projects that we are trying to design are very thoughtful ones and not time-wasting.

 Time is something extremely important. This is why I would prefer to have the certainty, before entering into a clinical development, that we will bring some solution to the patients that are going to be accepted by regulators and those that are creating the funding, and are going to bring certainty about the potential results. I cannot anticipate the results, but I try to. So time, is the most important thing and the certainty that I am going to meet the solutions for those patients that are in life-threatening conditions, especially the paediatric population.


Dr. Carlos R. Camozzi is the Chief Medical Officer of Orphazyme ApS, in Copenhagen, Denmark, a company with the high expertise in heat shock protein research and moved by the passion to bring outstanding solutions to the most challenging rare diseases.

Dr. Camozzi has more than 25 years experience in the bio-pharmaceutical industry, with 10 years dedicated to research and development of orphan therapeutic options for rare diseases and for the paediatric population. He played key role that led to the approval of the first gene therapy in western world for uniQure BV (Amsterdam, Netherlands) in 2012, and approvals of orphan products both in Europe (EMA) and in USA (FDA) for Orphan Europe (Paris, France). He is a physician specialised in Paediatrics, Neonathology and Clinical Pharmacology. He was member of the Division of Molecular Pathology at Basel University in Switzerland. He also held roles of increasing responsibility in Roche, Lederle and Mepha.

Interview conducted by Pharma IQ's Niamh Madigan