From artificial intelligence and digital therapeutics to the latest in oncological research and RNA therapeutics, from developments in the CNS field to what’s what in customer experience and pharma marketing, many of the events that pharmaphorum has covered in 2023 presented a broad spectrum of insights and telling suggestion of future trends to come. Here are some snippets of the most memorable conversations from Pharma Integrates, BIO-Europe Spring, NEXT Pharma, and RNA Therapeutics.
The ‘Big C’ proved to be a key talking point during Pharma Integrates London at the close of 2022. In a panel moderated by Linda Summerton, head of scientific affairs and oncology at Transcript, Jane Robertson, chief medical officer at Redx Pharma, reported noticeable progress in cervical, breast, and prostate cancer screening, but noted that more collaboration with academia and industry partners was needed to better identify high-risk patients and those who need a specific, directed screening programme.
Renata Crome, trustee, non-executive director, and pharmaceutical development advisor at PTEN research foundation, noted that the ‘needle has moved’, but not always in the right direction. Indeed, a lot more is now known about the effects on mental health and anxiety in the cancer care pathway, including the trauma of multiple biopsies.
However, Emma Kinloch, founder of Salivary Gland Cancer UK (SGA UK), caveated that if a patient has a cancer with no identifiable risk factors and is asymptomatic, that patient is unlikely to go into the system and has a low risk of overtreatment. Rather, the problems around access to screening and systemic inequalities need to be addressed.
Personalised medicine was a point not to be overlooked, of course, the crucial question being whether it works in the real world. Crome stated categorically that it’s not on the agenda in poorer countries, while in richer countries personalised medicine isn’t necessarily effective due to genetic variations between ethnic groups. Nonetheless, she also said that the UK is perfectly set to conduct such studies, due to its fantastic diversity of population, of ethnic groups, and cancer centres of excellence. However, there could still be more done with the models in preclinical trials.
Although 2023 has progressed a long way in the field of AI, with conversations on developments frequently scattered with utterance of ‘ChatGPT’, at the turn of the new year, conference attendees were still in need of convincing.
Steve Garden, CEO of PrecisionLife Ltd, noted the technology’s use in precision medicine analytics, novel target identification, and trial design, describing it as a blend of hype and hope and happening. Raminderpal Singh, CEO and co-founder of Incubate Bio, agreed, mentioning its role in Alzheimer’s targets, also. However, what Singh also highlighted was the buzzword nature of it. In fact, there’s nothing wrong with linear, with calculators, but the ‘junk in, junk out’ method of statistics, data crunching, and data cleaning, he said, seem old fashioned in comparison to the excitement around AI.
An important factor to bear in mind, though, Fordham said, were the semantics, the translation layer between the language a patient uses, for example, and the language of medical literature: a tummy ache becoming abdominal in a paper. There is a risk of overcomplication, despite being seen as a silver bullet; the risk of hype, with a lot of variables in the mix.
For Garden, the transformational quality of AI comes down to biomarkers, and the ability to distinguish between groups of patients, while for Fordham and Harwich it was clear data cleaning had become tarnished with the concept of a dusty veneer of history. This, Singh said, was down to the hype surrounding AI and similar technologies – and the salaries that go with jobs in that sector.
While you might have caught our initial coverage of BIO-Europe Spring this year, there was so much more to the event, including conversations on dealmaking, the obesity marketplace, and the future of CNS.
In a panel moderated by Susanne Weissbaecker, partner at Ernst & Young, Bristol Myers Squibb’s SVP of business development in innovative technologies, Konstantina Katcheves, noted that – despite the macroeconomic situation and geopolitical volatility at the start of the year – strategy to partner for innovative medicines remained unchanged.
That said, Bradley Hardiman, head of Europe in search & evaluation, business development at Astellas Pharma Europe, said a key quality was flexibility, to which MSD’s executive director of transactions, Karen McGurk, added collaboration, given that the right relationship that fits the rationale supports a partner through difficult times.
In short, “communication, communication, communication,” as Hardiman put it, takes companies “back to the thesis: the value to patients.”
Melanie Senior, healthcare writer and analyst at Nature Biotech, Evaluate, IN VIVO, moderated a conversation with Alessandro Toniolo, CEO of Resalis Therapeutics, and Josua Jordi, CEO of EraCal Therapeutics, on trends in the obesity marketplace.
In the US, six times more people are obese than are fighting any type of cancer, and the health risks are multiple: from diabetes and heart disease, to depression, orthopaedic conditions, and even cancer. The American Medical Association didn’t recognise obesity as a disease until around 2013, and there wasn’t an effective pharmacological treatment for obesity until the middle of the pandemic – then, Novo Nordisk launched Wegovy; next, Eli Lilly & Co were in regulatory submission for Mounjaro.
With questions remaining over whether these therapies have to be taken lifelong and the true value of investment, and with the regulatory pathway in obesity particularly difficult, it’s worth remembering the chequered history of this area of research, including appetite suppressants and the safety issues therein. But what crept up in discussions was the possibility that bodyweight – once thought to have a natural set weight – can be ‘reset’.
The central nervous system (CNS) remained a hot topic going into 2023 and a panel discussion moderated by Christian Jung, partner at The Dementia Discovery Fund – SV Health Investors, explored the latest developments in CNS and neurodegeneration and how neuroscience is currently experiencing a renaissance.
Céline Bouquet, executive director global business development in neuroscience, ophthalmology, and rare diseases at Roche, predicted the continuing development of biomarkers for diseases such as Alzheimer’s, but that endpoints for Huntington’s, for example, are still being sought. It is, though, a community effort and ‘long and deep investment’ is required.
Florian Muellershausen, managing director at Novartis Venture Fund, noted that there are already a lot of promising pipeline programmes, but that more should be trying novel avenues, rather than aggregates – including the biological risks implicated if there is a known path forward.
Meanwhile, Rob Pinnock, director of external innovation at Biogen Idec Ltd, had been advocating for a risk containment strategy for some time. Looking at the treatment of neurological disorders only 30 years ago, back when Multiple Sclerosis (MS) was essentially untreatable – now, with the therapies available, things can actually be achieved in CNS, he said.
Nicki Thompson, CEO of Amphista Therapeutics, cautioned a step back to assess the approaches used in the space, with the route of administration still a challenge and the enormous scope of the resurgent interest in small molecules, with the ability to get to the cells that need to be targeted. However, she queried when a bifunctional molecule gets so small that it becomes a glue.
Flying out to the breathtaking coastline of Croatia isn’t a particularly onerous request when tasked with it, all things considered – though the wet and windy weather perhaps begged to differ – so off pharmaphorum duly went to the Adriatic. And NEXT Pharma didn’t disappoint.
Opening the event was a simple question: who ignites technology and makes that fancy gadget shine? The answer is us, human beings. Pretty much everything we do leads to engagement, internal and external. With literal fire sparklers to commence the show, some opening remarks from Dario Safaric, NEXT Pharma summit chairman, had been crafted by ChatGPT.
But there is a sense of digital fatigue and what matters now, perhaps more than ever, is customer experience (CX). A sense of momentum to continued transformation is necessary and it was with that sentiment that proceedings began.
For Stefani Fruchtman Klaskow, director of healthcare at Google, to seek and find information is a basic human need. Having been with Google and life science organisations for over 16 years, one thing she believes to be fundamentally true is that information is the most important determinant of health: it empowers patients, and allows them to make informed decisions about their health.
However, there is an information gap that needs to be bridged and Google realises that its products have an important role to play in that process. After all, everyone should have access to useful, valid health resources, and 75% of people head online when they first have a question about their health.
In an increasingly digital world, the next phase in health communication, Klaskow said, is video. With people only remembering about half of what they’re told in an appointment, and 17.4 minutes being the average length of such consultations, understanding and recall are fundamental components when it comes to patient adherence. Indeed, there has been a 38% increase in queries for health-related content on YouTube recently.
YouTube Health is built on three fundamental pillars: principles, partnerships, and platform. To give one example, when a condition like depression is searched for, a shelf of related content from accredited institutions and sources is shown, and it is reliable due to the accreditation.
AI is the third big shift, she said. First there was the internet, then mobile, and all will be done similarly with AI.
While Plato and Hippocrates, the father of modern medicine, might have believed in the ‘wandering womb’, that it moves sideways and up and down, and while the Ancient Greeks applied scents to the vagina in the belief that it would put the womb back in place, it is shocking that such misunderstandings went centuries without being corrected. In fact, as a Nature-published study showed, women are diagnosed years later than men for the same diseases.
As Camilla Harder Hartvig, EVP and chief commercial officer at Theramex, explained during her presentation, an enormous issue that is underfunded and underserved is that creating a world that has healthier and more empowered women – if it needed to be said – means beneficial gains for families and workplaces. Or, as Hartvig put it: you can’t have a thriving pharmaceutical industry without thriving women who continue to work in the industry.
Only 4% of R&D in biopharma investment is dedicated to women’s health issues specifically, despite women representing 51% of the global population. Indeed, out of 37 total prescription drugs FDA approved in 2022, only two were for specific female health conditions.
The ancient view that women are only worth making babies needs to be discarded, Hartvig insisted. An estimated 13 million women in the UK are perimenopausal or menopausal: that’s a third of the workforce, which includes 38% who have thought about quitting work due to their symptoms.
You’ll likely have caught our earlier coverage of the first day of the RNA Therapeutics conference in London, an SAE Media Group event, but on the second day of the fascinating and 14th annual RNA Therapeutics conference pharmaphorum got to delve deep beneath the surface of matters, into the very science of what makes the industry we cover tick.
Covadonga Paneda, chief development officer at Altamira Therapeutics, presented on OligoPhore and SemaPhore for extrahepatic delivery of therapeutic RNA, the company’s nanoparticles formed from a peptide and RNA, developed by Samuel Wickline at Washington University. Chemically modified and non-modified RNAs have been experimented with to tackle the challenges of instability, in often degraded models. This particular peptide is derived from melittin, which comes from bee venom, so has the drawback of exhibiting a toxicity – a toxicity which Wickline spent 10 years modifying and reducing.
Meanwhile, Virginia Castilla-Llorente, associate director at Evox Therapeutics Ltd., explored extracellular vesicles as delivery agents for RNA therapeutics, looking at exosomes as a novel therapeutic modality. A disruptive approach with differentiating attributes, essentially, exosomes are delivery and communication agents. A safe and potent modality for precision delivery of complex payloads, DeliverEX is Evox’ platform for creating next generation genetic medicines, including next-gen AAV gene therapy, rapid-acting genome editing delivery, and differentiated RNA therapeutics, in conjunction with Takeda and Eli Lilly.
Also mentioning the scalability and proprietary exosome manufacturing processes, Castilla-Lorente said that it is a question of loading the drugs, including endogenous drug loading and exogenous drug loading. But an in vivo study in mice found that exosomes outcompete LNPs as a more efficient way of delivery. Ongoing research continues, however, into the stability of the mRNA – indeed, Christian Plank, chief technology officer at Ethris GmbH, later discussed the exploration of mRNA therapeutics, in his case, in the treatment of lung diseases.
In a panel led by Professor Ekkebard Leberer, life sciences consultant at Elbicon, he noted that the growth goal is to make RNA an important third modality in addition to small molecules and modalities. Heinrich Haas, VP of formulation and drug delivery at BioNTech, posited that it’s a question of doing the right thing at the right time with technology, while Christian Plank of Ethris GmbH asked what comes after vaccines. Furthermore, in the mRNA field and in artificial intelligence, how much is yet undiscovered?
Mark Cunningham head of operations at Nucleoic Acid Therapy Accelerator (NATA), stated that, if a naked RNA is placed in the body, it doesn’t speak to the tissue: it has to be conjugated to the target ligand. Indeed, it is targeting, rather than delivery, that will release quite a bit of the pressure on nucleoic acid therapies, he said. The other challenge is manufacturing.
Shalini Andersson, VP of oligonucleotide discovery at AstraZeneca noted that there are remaining gaps in the knowledge on RNA biology that need to be bridged, and efforts have to be put into academia in this respect. Certainly, she said, there will be targets that can’t be modulated with anything but RNA, and that is an exciting area to look towards. There will be lots of ups and downs on the journey with mRNAs, siRNAs, the separation of oligonucleotides, and the like. Plank caveated that mRNA will not be a cure for everything.
Nicole Datson, chief development officer at Vico Therapeutics, later focused on novel RNA-modulating therapies for the treatment of genetic neurological diseases, looking at the therapeutic benefits and challenges of antisense oligonucleotides (ASOs) for CNS. She explained that specific challenges remain for ASO therapeutics in CNS: biodistribution (uptake in deeper brain regions, targeting specific brain cell types, conjugating to specific cell-type specific ligands), less invasive dosing (devices, BBB passage allowing systemic dosing, e.g., LNPs), and establishing more appropriate clinical trial designs (earlier treatment, long trials, dosing regimens).
Nicole Raleigh, Web Editor
Nicole Raleigh is pharmaphorum’s web editor. Transitioning to the healthcare sector in the last few years, she is an experienced media and communications professional who has worked in print and digital for over 18 years.