as various commentators in this publication have reported on many occasions, the pharma industry is evolving – forced to change its ways due to internal factors, such as patent expirations of big-selling drugs, and external conditions, including global economic woes and advancing technologies.
although few have pronounced big pharma dead, the blockbuster era that revolutionised healthcare and became many a company's main focus does appear to be drawing to a close, with larger firms now turning to partnerships and alternative methods to develop a more holistic approach to healthcare.
it's a climate that has also allowed smaller companies to come through, making greater use scientific advancements and high-level expertise to specialise in medicines that are increasingly more targeted and able to fill unmet medical needs.
someone with one of the best views of this evolving approach to developing and delivering medicines is elmar schnee, the former ceo of merck serono who is now ceo of the much smaller swiss-based start-up cardiorentis.
it's a company with great potential, according to schnee, who has high hopes for its lead drug candidate ularitide – an investigational product that is in competition with novartis' serelaxin to be the first approved medicine specifically available for the treatment of acute heart failure.
“i think that after my job at merck, i was looking at what to do next and i thought some variety could be nice,” he said, explaining his decision to join cardiorentis.
“when you run a €6bn organisation you have politics, you have a huge cascade of information, and that's the reason i joined cardiorentis, because i believe in the project and it's actually very fun not having a lot of people serving you.”
elmar schnee career highlights
working in this new setting has allowed schnee a more hands-on approach as ceo (he drew up the company's cro contract himself), and it's a method that can lead to improved efficiency.
“the pharma industry has to change and the big organisations we have are run pretty inefficiently,” he says.
“and i've learned already so many things being very close to the clinical development, because that's our primary focus and i'm involved in those discussions.
“as a ceo of a big organisation, you are not going into the trial and you are not part of the discussion about how you run a trial – about the logistics, about what you want from the cro. and here i think we have the chance to do it differently.”
of course, working in a smaller company means fewer available resources, but this lack of financial backing and staff numbers can help a firm focus its mind, according to schnee.
“we have a key project which we are working on, and every fibre of everyone is focused on this. it makes you very effective, and it makes you focus on the most important thing to bring a project to fruition.”
and all focus at the moment is on the development of ularitide, a human endogenous natriuretic peptide that is currently in phase iii development to treat acute heart failure (ahf).
my hopes are that we can deliver the first evidence-based treatment for acute heart failure, because there is nothing
ahf is a common, and potentially fatal, condition that is defined as a rapid change in the signs and symptoms of heart failure, which include problems with breathing and the build-up of fluid accumulation in the patient's lung.
most cases involve people who have been previously diagnosed with heart failure, and can be triggered a specific event, such as a change in a person's treatment or uncontrolled hypertension.
however, despite its common occurrence, there is still no specific drug available as a treatment in europe, with current standard therapy including diuretics to reduce fluid retention and vasodilators to widen the blood vessels and reduce blood pressure.
cardiorentis is in line to be the first company to provide one, however, if ularitide continues to demonstrate the positive results it has achieved in earlier clinical settings.
“my hopes are that we can deliver the first evidence-based treatment for acute heart failure, because there is nothing,” says schnee.
this lack of treatment is especially surprising considering the death rate for people who experience acute heart failure is greater than that of all cancers, apart from lung and pancreatic, according to schnee.
“in cancer, we have huge discussions in the paper, in the journals, in the media, and the pharma industry is putting a lot of money towards work in this area. but here, we have something that can be far worse if you are affected by it, and there are no treatments.”
the ageing population in europe means it's a problem that's only going to increase, says schnee, although the healthcare sector is gradually waking up to the need for improved treatment.
“in heart failure, there is now a big change on the way,” he comments. “because of the serelaxin trial [by novartis], you saw for the first time that you might do something, if the findings by novartis are robust.
“i think that physicians are also clear now that time is of the essence. up to now, the patients came in and they weren't treated immediately, but now it seems that time is crucial, we have to go in fast.”
this extra knowledge presents its own challenges, however, with schnee commenting that healthcare systems must adapt to ensure patients are treated within six hours in the ularitide trial in order for medicines to be most effective.
another area where changes need to be made is in regulation, says schnee, noting that “regulators put their demands too high” for drug approvals.
everything is driven by money, and there are fewer companies able to commit to clinical trials
he cites the request from regulators for a mortality-focused endpoint in clinical trials, meaning up to 10,000 patients would be needed for a trial – an impossible task for a company the size of cardiorentis.
the company did manage to successfully negotiate a revised endpoint of a reduction in symptoms, meaning trials involving about 2,000 patients, but schnee asserted the situation needs to change.
“i think there needs to be real dialogue between the regulators and between the healthcare community. everything is driven by money, and there are fewer companies able to commit to clinical trials”.
“we have to do something, because on the one hand you want cheaper drugs, but on the other hand, every year the cost of research goes up, so we have to have dialogue.”
overcoming such differences remains a challenge though, according to schnee.
“i especially think the incentive of the regulator is wrong,” he says. “one of their main tasks is to protect the population from side effects and you could say the easiest way to do that is not to approve any drugs.
“the risk shyness is just incredibly high, and we have to find the balance where we can allow drugs to come earlier to the market – maybe with smaller trials, with more stratified products.
“sometimes, regrettably, we will have some patients lost because we didn't do a multi-thousand people trial where we might have found a side effect that occurs in one in ten thousand people. but, overall, as a society as whole, i think these are discussions that we have to act on.”
as for where cardiorentis fits into this debate, it may not have the resources to effectively change behaviour on a large scale, but it still can play its part.
“what we can do in our particular area is we can be a small example of how one can do it differently,” says schnee.
“and then if bigger companies follow, we can create an effect. not only will we have a new drug, but we can have more drugs brought faster to people.”
commenting on his own company's future, schnee says there are several options that lie ahead if all goes well in phase iii development for ularitide.
these include an actelion-type model, with the company marketing the product itself. cardiorentis could also outlicense the project to a larger pharma firm, and then continue to develop drugs in other indications. alternatively, there could be an outright sale of cardiorentis to a larger company.
schnee has a firm belief in the future of his company, however, and if ularitide is successful, there is only more to come.
“i'm convinced that if we want to, and if the [ularitide] study is positive, we have enough ideas and access to molecules to build a sustainable business that can bring new products to the market.”