CEO Blog | Clinuvel Pharmaceuticals news and discussion blog

MAA: a moment in Clinuvel’s EPP story

Thursday, February 9th, 2012

Those who have taken an interest in Clinuvel will have learned with joy that, on Monday February 6^th, the company announced its first official filing for SCENESSE® (afamelanotide) with the European Medicines Agency. It has taken our teams around six years to arrive at this point. Benchmarked against peer companies, it is a relatively swift development path for a first-in-class drug; we first publicly announced our erythropoietic protoporphyria (EPP) program in September 2006. It is an opportune moment to reflect briefly on how we reached this milestone and then discuss the steps that must be taken from here. (more…)

EPP results and clinical relevance

Friday, November 4th, 2011

A protoporphyrin IX molecule

Over recent months I have written several times of the need for Clinuvel to prove clinical relevance in our trials with the use of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP). In orphan populations the need to demonstrate how a novel drug assists in their daily activities and improve their lives is at the forefront of the regulators’ minds. And so it should be, after all the objective of the pharmaceutical industry is to develop drugs which address either disease or symptoms adequately and safely. The results the company released yesterday from our Phase II US study of the drug in EPP (CUV030) have given us important data towards demonstrating clinically relevant improvement of patients’ lives. (more…)

Clinical relevance – the value of patient experiences

Friday, September 30th, 2011

Since 2006 Clinuvel has trialed SCENESSE® in a truly unique group of individuals: patients living with erythropoietic protoporphyria (EPP), a rare genetic blood disorder which causes an absolute intolerance to light.

EPP prevents patients from leading ‘normal’ lives, especially outdoors. It is one of the few diseases that manifest clinically with initially invisible symptoms which cause severe dermal pain for several days. This not only presents a challenge for diagnosis and treatment, but also for generating meaningful clinical trial results – those which are measurable numerically and are used by regulatory authorities to evaluate the efficacy of a drug in a patient population. Here, real life patient experiences during a trial can play an important role in providing clinical relevance and analysing hard data. (more…)

Who wins from pharma cost-cutting?

Friday, September 23rd, 2011

Earlier this month Sanofi, the world’s fourth largest pharmaceutical company by revenues, announced that it would go on a cost-cutting spree, trimming its expenditure by $US2.9billion annually and cutting many thousands of jobs in the process. Amidst the upheaval, Sanofi stated its key goal for 2012 onwards was ‘generating sustainable growth’ for shareholders (the complete presentation can be viewed here). One of the key targets for its cuts was R&D costs, both in terms of overall spend and employee headcount. (more…)

The importance of clinical relevance

Tuesday, August 2nd, 2011

Development of novel drugs is truly like no other business: one attempts to address questions that may have never been previously posed – let alone answered – in the pursuit of improving the lives and quality of life of patients. As I eluded to in my recent letter to shareholders, the team is now well into the analysis of results from our erythropoietic protoporphyria (EPP) program; two studies from the US and Europe. This is a complex and time consuming task that requires one to collate and make sense of thousands of data points to answer a seemingly straight forward question: does this trial show that the drug is safe and effective?

Obtaining an answer needs to be understood from the concept of clinical relevance. Put simply, results don’t just need to show that a treatment or intervention has an effect on a disease. Rather, they need to indicate that that effect is relevant to the current clinical understanding, treatment and care for the disease or indication. They need to show that the drug’s effect is having a positive, meaningful impact upon a patient’s prognosis and care. This is a crucial point to consider in the development of protocols and in the careful analysis of results, as it is how regulators will review the results. (more…)

Small populations, big therapeutic potential

Monday, May 23rd, 2011

When Andrew Pollack of the New York Times declared that the “world’s largest drug company is thinking small”, he wasn’t referring to reductions in sales force.

Rather, Pollack was reporting on a licensing deal between Pfizer and Israeli biotech company Protalix which built upon a growing global trend: big pharmaceutical companies were making a move into treatments for rare diseases, otherwise known as ‘orphan’ therapies.

In the months that followed Pollack’s December 2009 article, both Pfizer and GSK (two of the world’s largest pharmaceutical companies) launched specific business units focused on R&D for orphan drugs. In their announcements, both companies highlighted the significant unmet medical needs that exist in rare diseases and the potential of therapies that were in development. (more…)

‘Measuring’ vitiligo: the challenges of clinical and treatment evaluation

Monday, April 4th, 2011

Since our announcement last year that Clinuvel would commence a new program for SCENESSE® (afamelanotide) in nonsegmental vitiligo, the company has received vast interest in the application of the drug in this disease. Of the enquiries that best captured the essence of this program, one stood out: a US based analyst asked how the company intended to objectively measure the response to treatment, the repigmentation of vitiliginous lesions, in its trial. (more…)

New drug approval rates

Friday, February 25th, 2011

Novel drug development is a business which is not well understood; perhaps one which is not well explained. To be able to launch a novel drug proposition, one needs tenacity, expertise and a talented team to succeed. Unlike a ‘follow-on’ product where an abundance of safety data on the molecule in one or other formulation or therapeutic application exists, those few companies working with new drugs must clear all the necessary stringent barriers from the outset.

This makes novel drug development a risky endeavour, amplified by a level of uncertainty even when it is believed that the necessary regulatory hurdles have been cleared and the drug development process seems complete. By looking at several new molecules being developed for obesity (Lorcaserin, Contrave and Qnexa particularly), one can see how uncertain the outcome remains, even when so much work and so many years have been invested; others have written extensively about these cases and I encourage avid readers to review the publications. (more…)

A clinical success: study retention rates

Thursday, February 10th, 2011

We were delighted this week to be able to announce the successful completion of our first Phase II study conducted in the US, a placebo controlled, randomised trial of SCENESSE® (afamelanotide) for patients diagnosed with erythropoietic protoporphyria (EPP) (CUV030). (more…)

Who wins from pharma cost-cutting?

The importance of clinical relevance

Small populations, big therapeutic potential

‘Measuring’ vitiligo: the challenges of clinical and treatment evaluation

New drug approval rates

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