Modular biopharma
As Big Pharma slim down and biotechs build up, the boundaries between “pharma” and “biotech” continue to melt away.
The biopharma world begins to look more modular as assets and franchises are bought, sold or swapped amid shifting policy and pricing dynamics, evolving sales channels, fast technological innovation and emergent geographies.
Biopharma firms of all sizes may have commercial arms and must adapt to more connected, price-sensitive users whose direct influence on drugmakers will likely grow as mainstream insurer channels continue to tighten. In this world, small and nimble players with a unique suite of solutions and fresher, more consumer-focused mindsets will have as much chance (or more) of winning customer recognition as the legacy pharma brands.
And the most valuable products may not necessarily be the most technologically novel.
It’s notable that the latest start-up from biotech veterans John Maraganore, founder of Alnylam, and Clive Meanwell, formerly of The Medicines Company, plans to develop a non-novel medicine to sell direct to consumers. Corsera Health, which emerged from stealth in August 2025, has its eyes on a single-shot PCSK9- and angiotensinogen-targeted RNAi drug to prevent cardiovascular disease, possibly accompanied by an AI-powered predictive tool. PCSK9 is the target of Novartis’ Leqvio (incliseran), licensed from Alnylam, and several clinical programs are chasing angiotensinogen (including one at Alnylam).
The innovation at Corsera isn’t the target or modality, but in how the medicine is packaged and delivered. Convenience and access have replaced novel science. “We have to exchange a set of norms in the industry from low-volume, high-margin products to relatively high-volume, low-margin but very, very effective” products, Meanwell told Endpoints News.
A decade or so ago, drug re-formulation novel delivery were widely viewed as poor cousins to ‘true’ scientific innovation. Yet as a growing current of new, highly complex drug modalities hits up against change-resistant, inadequately funded health systems, more attention is shifting to improving upon what’s already out there. “We are all reverting, a bit, to deal with more of the known and less of the new technology,” said the partner at one venture capital firm in September 2025, months after raising a $1billion-plus new fund.
Other current examples of highly valued re-formulations include Merck & Co.’s subcutaneous version of the cancer drug blockbuster Keytruda, whose faster administration may better suit patients and clinics, but also extends the drug’s patent life. It is among the top ten most valuable pipeline products by net present value, slated for FDA approval September 23, 2025. (See Evaluate World Preview report). Johnson & Johnson launched a similar subcutaneous version of blood cancer blockbuster Darzalex in 2020.
Smaller-scale reformulations are also attracting investors. The PAH project that sent Insmed’s stock soaring earlier this year, TPIP, is an inhaled pro-drug of treprostinil, sold since 2009 by United Therapeutics as Tyvaso. TPIP’s advantage is once daily administration, versus multiple times a day for Tyvaso (also sold since 2022 in dry-powder-inhalation form). Evaluate forecasts 2032 TPIP sales at over $1 billion.
In August 2025, PureTech Health’s deupirfenidone, a deuterated form of InterMune/Roche’s now-generic idiopathic pulmonary fibrosis drug Esbriet (pirfenidone), was packaged into new biotech Celea Therapeutics. It’s seeking external funding for Phase 3 trials of the molecule, whose pharmacokinetics appear to enable better tolerability and efficacy.
Target- and mechanism-based innovation won’t go away. But these examples, across both large and small biopharma, show a change in thinking brought on by the financial squeeze on payers, the China-accelerated urgency to achieve greater R&D efficiency and a more prominent role for patients.
Many questions arise from these shifts across the biopharma landscape. Might investors’ appetite for later-stage assets lead to long-term damage across the seed and start-up eco-system? Could China become the main source of clinical programs across Western pharma pipelines? (A recent Morgan Stanley report forecasts that, by 2040, nearly 35% of FDA approved drugs will come from China.) Will some Big Pharma investors start to shift their attention toward more focused franchises on offer across a growing cohort of commercial stage biotechs? Which types of company will win the battle for consumers’ hearts and healthcare dollars – and will those winners be biopharma, or their masters from another sector?
These questions – and more - challenge today’s biopharma firms. They’re also opportunities, however. It is quite possible that the biotech financing winter generates a more resilient biotech sector, and that slimmed down pharmaceutical firms become more agile and responsive. Chinese competition could raise global R&D efficiency and benefit patients globally. Direct-to-consumer sales may widen drug access and lead to more transparent pricing across conventional channels.
Such scenarios would likely draw investors to both the “bio” and the “pharma” ends of biopharma.
It is quite possible that the biotech financing winter generates a more resilient biotech sector.
