Progress and Pitfalls
Whatever size their starting assets, these biotechs are all reaching for the stars. "We expect to be a leader in MASH for many years to come," asserted Madrigal CEO Bill Sibold during JP Morgan. Madrigal is benchmarking Rezdiffra’s launch against the top ten specialty launches of the last decade (though won’t say which ones). "We want to make history as one of the best," says Huntsman.
It is early days for these and several other biotechs launching into 2025’s markets. They are still only reaching a small proportion of their target populations. Launches can run out of steam as the needs of an initial cohort of on-side specialists and patients are met. The US healthcare system faces multiple uncertainties resulting from the new Trump presidency and his swathe of (sometimes controversial) healthcare-related appointees. And even supposed tailwinds, like great data analytics, can backfire. "Too much data can paralyze an organization," warns Outten.
Launches can run out of steam as the needs of an initial cohort of on-side specialists and patients are met.
Maintaining a dual focus on both R&D and commercial could prove tricky for some biotechs, especially for those with busy pipelines. Investors’ goalposts move once revenues start, and pressure shifts from reaching R&D milestones to meeting sales targets and, ultimately, breaking even.
Huntsman claims that Madrigal isn’t yet facing that pressure to reach breakeven. "Investors first want to see what we can do in terms of launch success," she says. "We’re starting to convince some that we can do it."
Potential pharma buyers will be watching closely how these biotechs fare through 2025 and beyond. Meanwhile, the wealthiest biotech company creators – VCs like ARCH Venture Partners or RA Capital – claim to have product-launch in mind from day one, not least since a biotech equipped to succeed independently has even greater leverage with strategic acquirors.
Market and M&A dynamics, as much as commercial skills, will determine whether those that look most likely to turn into the next Vertex or Gilead actually have the chance to do so.
MAKING IT HAPPEN – LAUNCHING A CAR-T CELL THERAPY
Contract manufacturing has expanded amid growing demand, multiplying modalities and Big Pharma outsourcing. That allows most go-it-alone biotechs to avoid significant production outlay.
But it doesn’t help them all.
Autolus’ CAR-T cell therapy Autaczyl (obecabtagene autoleucel), approved in November 2024 for some forms of acute lymphoblastic leukemia, requires bespoke manufacturing that no CDMO could provide. Autolus’ launch preparations (and its registrational trials) therefore involved building an entire commercial manufacturing capability, says CEO Christian Itin.
The nature of CAR-T cell therapy –which involves collecting patients’ immune cells, genetically engineering them ex-vivo, and re-infusing the finished product - means that manufacturing and commercialization are uniquely tightly connected, making it "much harder to partner," says Itin. And the workforce required to operate this process "doesn’t exist in the labor market," he adds. "You have to train them."
The huge capital outlay – and relatively paltry sales from such products so far – helps explain why many investors have gone cool on cell and gene therapies. (Autolus got a $250m investment from Blackstone in 2021). Itin points to the slow-but-steady ramp-up of Amgen’s ALL immunotherapy Blincyto (blinatumomab), launched a decade ago and now selling close to $1 billion despite complex administration (he was previously CEO of Blincyto originator Micromet, sold to Amgen in 2012).
Potential pharma buyers will be watching closely how these biotechs fare through 2025 and beyond.
