Purespring on nephrology & gene therapy in kidney disease

*This episode of the pharmaphorum podcast is brought to you in association with Klick Health* When it comes to digital innovation and AI, consideration of using and implementing such responsibly and ethically ever more becomes forefront. But how can pharma lead the way in ethical AI and trust-driven innovation? In a new pharmaphorum podcast, Erik Janssen, head of digital care transformation at UCB, joins web editor Nicole Raleigh for a conversation on responsible digital innovation and ethical AI. Janssen discusses how pharma can lead in responsible experimentation by focusing digital innovation on enhancing (not replacing) human care. He also speaks about co‑creating solutions with clinicians and patients, as well as the role of evidence‑based technology across the innovation framework. You can listen to episode 274 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from. *This episode of the pharmaphorum podcast is brought to you in association with Klick Health*

Public demand for prevention is surging, with 33.6 million people now using the NHS App, yet, NHS leaders say the digital infrastructure required to deliver prevention at scale isn’t in place. In a new episode of the podcast, pharmaphorum spoke with James Davis, a healthcare expert at PA Consulting, for a conversation on the digital foundations needed to unlock a prevention first NHS. Davis also discusses the initiatives with the biggest near term impact, what NHS leaders are worried about, and issues of trust and inequality. You can listen to episode 273 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

For a variety of reasons, early stage clinical trials have been moving out of the United States more and more over the past few years, as companies pursue savings and lighter regulatory burden in places like China and Australia. The industry has long urged the US government to take some action to make it more attractive to bring these trials back to the States. That's the broad idea behind Operation TRIALBLAZER, a campaign launched this summer by HHS and the FDA. On today's podcast, host Jonah Comstock is joined by Dr Harpreet Singh, currently chief medical officer at Precision for Medicine. That's her first private sector job after nearly nine years at the FDA, so she's able to bring both an insider and outsider perspective in discussing this initiative. In addition to sharing some details about her time at the FDA and how the organisation has changed, Dr Singh discusses the situation in clinical research that's led to the US' leadership in clinical trials slipping away, what's in the Trialblazer plans, and whether she thinks it will be enough to turn the tide. They also talk a little bit about radiotherapeutics regulation and discuss why having clinical trials in the United States is important not just for the economy, but also for patients who are missing out on opportunities for cutting-edge care. Tune in for a fascinating primer on a major new US government clinical trials initiative.

Chronic liver disease affects around 1.5 billion people globally. As understanding of liver disease improves, outcomes are increasingly determined by how early patients are identified, not just what therapies are available. In a new pharmaphorum podcast, web editor Nicole Raleigh speaks with Lee Lehman Becker, Roche’s global head of healthcare insights at Roche Information Solutions (RIS), for a discussion of the liver disease epidemic that is being diagnosed too late and driving a growing health system crisis globally. The conversation also explores scalable diagnostic innovation and the role of this in addressing health system burden – metabolic liver disease projected to affect some 1.8 billion people by 2050, with global costs expected to exceed $2.3 trillion. You can listen to episode 271 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

The drug price negotiation provisions in the Inflation Reduction Act were designed to continue evolving with each passing year. As CMS prepares for 2028 and 2029, the agency has put out new draft guidance codifying and, in some cases, modifying the fine print of the controversial law. They’ve also recently issued additional draft guidance on how they’ll handle the imminent introduction of Part B drugs into the programme. Hogan Lovells partner Alice Valder Curran returned to join pharmaphorum editor-in-chief Jonah Comstock to elucidate some of the finer points of this latest communication from CMS – and to urge the industry to take advantage of the public comment periods associated with it. Curran and Comstock discuss small, but impactful, changes in CMS’s policies around deemed biologics, vaccines, and the special provisions that protect small biotechs. And Curran gives a rundown of what’s in – as well as what’s not in – the new MFP effectuation guidance, laying out how the differences in how Part D and Part B drugs are paid for leave some big question marks hanging over the programme. Tune in for an easy-to-understand deep dive into some complicated subject matter, and for a more comprehensive rundown don’t forget to check out Hogan Lovells’ policy briefs on the new draft rules and the MFP effectuation guidance.

SYNGAP1-related disorder negatively impacts synaptic signalling during critical periods of brain development, leading to a range of cognitive, epileptic, behavioural, and motor symptoms. In a new episode of the pharmaphorum podcast, web editor Nicole Raleigh spoke with Kelly Gold, CFO of CAMP4 Therapeutics, about antisense oligonucleotides (or ASOs) - medicines designed to selectively restore natural protein production in diseases caused by gene under expression. Gold discusses the space and CAMP4’s CMP-002 study, targeting the regRNA associated SYNGAP1 gene. You can listen to episode 269 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

The M&A market has been rebounding in the US, driven by factors like patent cliffs and improved balance sheets – but the question is whether this will play out in Europe and how the wave could differ. In a new pharmaphorum podcast, web editor Nicole Raleigh speaks with Andrea Carta Mantiglia, partner and lead of the Healthcare & Life Sciences Focus Team at Italian law firm BonelliErede, about whether the current US biotech M&A surge will indeed extend into Europe. The conversation touches upon the appetite for cross-border dealmaking, the growing role of private equity in European pharma and medtech, generational exits among family-owned pharma groups in Italy, and the role of digitalisation in all this. You can listen to episode 268 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

Purespring Therapeutics is a precision nephrology company pioneering first-in-class, targeted genetic therapies designed to preserve kidney function. In a new pharmaphorum podcast, web editor Nicole Raleigh speaks with Haseeb Ahmad, CEO of Purespring, who discusses transforming the treatment of kidney disease, an area of enormous unmet need, with more than 840 million people worldwide living with chronic kidney disease. Indeed, Purespring’s lead programme is the podocyte-targeted gene therapy PS-002, which is for IgA nephropathy (or IgAN). The conversation also touches upon the evolution of the gene therapy landscape more generally, and where next with this type of innovation in the nephrology space. You can listen to episode 267 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

Europe's biotech sector is facing an innovation cliff, with fewer clinical starts, tighter capital, and increasing development complexity. In a new pharmaphorum podcast, web editor Nicole Raleigh caught up with Paul Bridges, president, consulting, at Parexel, and Charlotte Moser, Parexel’s chief medical officer, following BIO-Europe Spring in Lisbon, Portugal. The conversation touches upon regulatory strategy and evolving policy shifts, trial design and execution – and the importance of patient centricity – as well as AI in drug development and medical leadership in drug development today. You can listen to episode 266 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.

Cutaneous T-cell lymphoma (or CTCL) is a rare form of non-Hodgkin lymphoma that begins in T lymphocytes, a kind of white blood cell that plays a crucial role in the immune system. And Citius Oncology is focused on developing and commercialising a targeted immune therapy for the initial indication of the treatment of persistent or recurrent CTCL. In a new pharmaphorum podcast, web editor Nicole Raleigh spoke with Leonard Mazur, CEO and Chairman of Citius Pharmaceuticals about the launch of its first FDA-approved drug, Lymphir, for CTCL. Mazur discusses the landscape of unmet need in this space, as well as Citius’ advancement of clinical trials in other areas without approved treatments and the future horizon for such oncological innovations. You can listen to episode 265 of the pharmaphorum podcast in the player below, download the episode to your computer, or find it – and subscribe to the rest of the series – on Apple Podcasts, Spotify, Overcast, Pocket Casts, Podbean, and pretty much wherever else you download your other podcasts from.