pharmaphorum Daily Newsletter - 2nd Dec.

pharmaphorum Daily Newsletter - 2nd Dec.

This Monday, news breaks that 诺华 has paid $1bn upfront for an oral mRNA splicer for Huntington's disease from PTC Therapeutics.

Meanwhile, 百时美施贵宝 has become the latest pharma in the US to file a legal challenge to a block on a 340B drug rebate model opposed by HRSA and four years after buying US rights to GAIA's digital therapeutic to help people with depression, Swedish drugmaker Orexo AB publ has terminated the deal.

Elsewhere, faced with a cash crunch, Alligator Bioscience AB has said it plans to shed up to 70% of its workforce and Abingworth is reported to be raising $1.5bn for further risk-sharing, royalty-bearing development deals with large pharma groups.

In our longer reads today, dive into a Part Two feature on navigating the complexities of global market access for rare and complex conditions.

All this and more on pharmaphorum.com


News

Novartis pays $1bn upfront for Huntington's drug from PTC

Novartis has made a return to the Huntington's disease stage by licensing an oral mRNA splicer from PTC Therapeutics for $1 billion upfront.

BMS files legal challenge to HHS' 340B rebate decision

Bristol-Myers Squibb has become the latest pharma in the US to file a legal challenge to a block on a 340B drug rebate model opposed by HRSA.

Orexo exits depression DTx alliance with GAIA

Four years after buying US rights to GAIA's digital therapeutic to help people with depression, Swedish drugmaker Orexo has terminated the deal.

Alligator slashes workforce and pares down its pipeline

Faced with a cash crunch, Alligator Biosciences has said it plans to shed up to 70% of its workforce.

Abingworth 'will launch $1.5bn fund for co-developed drugs'

Abingworth is reported to be raising $1.5bn for further risk-sharing, royalty-bearing development deals with large pharma groups.


Views & Analysis

Part Two: Navigating the complexities of global market access for rare and complex conditions

The journey to global market access for innovative therapies is fraught with challenges that vary significantly across the world. Drug manufacturers must adeptly navigate ever-changing regulatory frameworks, cultural expectations, and commercial landscapes to maximise the potential of novel treatments.

Part One: Overcoming challenges and seizing opportunities in global market access for rare and complex conditions

The outlook for drug development to treat rare diseases looks encouraging, offering hope to the 300 million people globally facing the significant challenges of these often-overlooked conditions.

Solution Spotlight: Medscape Extend

Medscape Extend is a transformative platform designed to revolutionise how pharmaceutical advertisers communicate with verified healthcare professionals (HCPs). Utilising the advanced capabilities of PulsePoint technology, Medscape Extend enables precise identification and targeting of Medscape-verified HCPs offsite on non-endemic properties, ensuring that marketing messages reach them across their digital journey.

Designing personalised digital experiences that engage healthcare professionals

Healthcare professionals (HCPs) expect digital experiences to be as seamless and personalised as the apps and services they use in their daily lives. It’s essential that pharmaceutical organisations understand these expectations.

Putting the UK back on the map for clinical trials: NWEH partners with CPRD

The state of clinical trials in the UK has been a central topic of discussion for some years now, but keenly so since Brexit and following the COVID-19 pandemic. Since publication of Lord James O’Shaughnessy’s Independent Review however, it seems, indeed, that small steps are being taken in the right direction to making Britain a tour de force in the life sciences sector. And NWEH is one such company assisting in these endeavours.


Podcasts & Videos

CAR-T therapies: Faster, better, cheaper

In a new pharmaphorum podcast, editor-in-chief Jonah Comstock speaks with Phil Johnson, MD, president and CEO of Interius Biotherapeutics, a company working in the in vivo cell therapy space.

Health Innovators: Lori Fletcher and Amber Beimer, Advanced Clinical

Clinical research is full of challenges, from recruitment to data management to global resourcing. Working with a functional service provider (FSP) can be a great option for meeting these needs. But what exactly is an FSP, and what are the advantages to working with one? In this interview, brought to you by Advanced Clinical, Programme Directors Lori Fletcher and Amber Beimer speak with pharmaphorum editor-in-chief Jonah Comstock and clear up some misconceptions that FSPs are merely staffing agencies.

Looking to history to prepare for America’s uncertain future

In today’s podcast, web editor Nicole Raleigh is joined by Jesse Mendelsohn, senior vice president of Model N’s Center of Excellence, for a conversation that explores multiple aspects of the drug pricing debate in America – speaking before President-Elect Donald Trump confirmed his nomination of Robert F Kennedy Jr as US Health and Human Services Secretary.

Next gen wearables: Transforming remote patient care

In a new podcast, web editor Nicole Raleigh speaks with Dr Samuel Ewing, global head of pharma partnerships at Doccla, about the next generation of digital tools such as wearable medical devices, as well as remote patient monitoring and digital healthcare generally.

From neurosurgery to focused ultrasound: Not if, but when

In another pharmaphorum podcast, web editor Nicole Raleigh speaks with Dr Neal Kassell, chair and founder of the Focused Ultrasound Foundation and previously a neurosurgeon, who created the Foundation in 2006 to improve the lives of millions of people with serious medical disorders by accelerating the development and adoption of focused ultrasound.


Webinars & White Papers

The AI effect in pharma commercial strategy: From data to real-time insight & actions

Join us THIS WEDNESDAY 4th December @ 2pm GMT - 3pm CET - 9am EST, for an exclusive webinar, sponsored by Trueblue, to learn how life sciences organisations are leveraging the power of AI-driven strategic insights to revolutionise decision-making, enhance customer engagement, and gain a competitive edge.

Decoding physician prescribing behaviour: A data-driven journey

In this webinar, sponsored by PurpleLab, learn how to gain a competitive edge by understanding physician prescribing behaviour in target markets. Available now on demand.

Enabling site success: Key insights to reducing burden and increasing access

In a webinar sponsored by Syneos Health - with Atlas Clinical Research’s Chief Executive Officer Mark Scullion, and Batisha Anson, Global Head of Patient Diversity and Health Equity at Syneos Health - learn more about the site experience for clinical research sites. Now available on demand.

Global reach, personal touch: Mastering customer engagement at scale Discover how to balance personalised engagement with regulatory compliance across global pharma markets in a webinar presented by EVERSANA INTOUCH. Available on demand now.

Beyond clinical: Boosting recruitment and retention with SDOH data In a Socially Determined sponsored webinar, discover how industry leaders are harnessing non-clinical data to enhance recruitment, retention, and broader evidence generation. Now available on demand.


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