Unpartnered assets: on the shelf or hidden treasure?

A look at biopharma’s most highly valued yet unpartnered assets features Karuna, Ventyx and regular bridesmaid Ascendis.

Getting a novel drug to market is the endgame for many in biopharma but moving through the clinic without attracting the interest of a larger developer can be considered a slur. Figuring out whether these unencumbered projects are rare gems or fools’ gold is a major challenge, for both investors and pipeline-hungry partners.

Enter Evaluate Vantage’s latest list of biopharma’s mostly highly valued but unpartnered assets, featuring a few names that have cropped up in years past: Karuna, Ascendis and Madrigal. A run of big pharma bolt-on deals this year suggests that it is premature to consider their respective assets forever on the shelf, while the analysis throws up plenty of new entries to mull over.

The table below is based on the net present value of these assets, as computed by Evaluate Omnium from sellside consensus forecasts. For inclusion, an asset had to be wholly owned, outside of clinical trial collaborations or small, regional tie-ups, and its developer unlikely to want to launch alone.

Bullish sellside forecasts are not always echoed by investors, of course. Developers in the list below whose market caps are dwarfed by the asset NPV should probably be considered the most speculative. ALX Oncology and evorpacept, for example, which continues to attract some very optimistic numbers from certain equity analysts despite a problematic development history and broader concerns about the potential of CD47 blockade.  

Not that investors always get it right, of course, as some formerly hotly tipped candidates demonstrate. Kodiak’s KSI-301, Allakos’s lirentelimab and Bridgebio’s acoramadis have all disappointed since featuring in our previous run at this analysis, in November 2021.

A selection of biotech's biggest unpartnered assets
Project Company Status  Mechanism Today's NPV ($bn) NPV as % of share price
KarXT Karuna Therapeutics Ph3 M1/M4 muscarinic agonist + muscarinic receptor antagonist 8.29 97%
Aficamten Cytokinetics Ph3 Cardiac myosin inhibitor 4.15 115%
VTX958 Ventyx Biosciences Ph2 Tyk2 inhibitor 3.65 180%
TransCon PTH Ascendis Pharma Filed Parathyroid hormone regulator 3.35 66%
GTX-102 (UTPH) Ultragenyx Pharmaceutical Ph2 UBE3A-AS antisense 3.30 95%
Rusfertide (PTG-300) Protagonist Therapeutics Ph3 Hepcidin antagonist 3.16 210%
Resmetirom (MGL-3196) Madrigal Pharmaceuticals Ph3 Thyroid hormone receptor beta agonist 3.01 59%
AMT-130 Uniqure Ph2 Huntington's gene therapy 2.70 122%
PRX012 Prothena Ph1 Beta amyloid MAb 2.50 65%
Evorpacept ALX Oncology Ph2 CD47 inhibitor + SIRP alpha inhibitor 2.41 673%
Source: Evaluate Omnium.

Karuna, Madrigal and Ascendis have long been tipped as takeover targets yet remain solo stories; they have featured in this analysis as far back as 2018 for Madrigal and 2020 for Ascendis. It is worth remembering that past articles in this series have identified projects that went on to be sold for billions: Turning Point, Global Blood and Spark.

Many biopharma watchers also reckon that Cytokinetics’ cardiomyopathy contender aficamten will attract partnering attention, although the developer is approaching a crucial trial readout later this year, from a study called Sequoia. Still, this week’s buyout of Chinook by Novartis, also ahead of a big phase 3 readout, shows that clinical validation is not always a necessity.

The $1.6bn NPV of Chinook’s lead asset, atrasentan, put that project outside of the top 10 above. Notably, Iveric’s geographic atrophy project Zimura would have made the cut with a $3.2bn NPV, ahead of that biotech’s $5.9bn buyout by Astellas.

More to prove?

Others projects on the list arguably have more to prove, their sellside champions notwithstanding. Ultragenyx's Angelman’s project GTX-102, an antisense oligonucleotide, is due to yield early data later this year. Some analysts have pencilled in a late 2026 launch, which feels ambitious.

Ventyx has also promised mid-stage data by year end with its Tyk2 inhibitor, VTX958; whether the company can attract a partner as successfully as Nimbus did for its similar project is the big question here.

Protagonist has already found a partner in J&J for its IL-23 antagonist, and if sellside hopes are to be believed rusfertide, its wholly-owned hepcidin mimetic being developed in polycythaemia vera, also presents a compelling opportunity. The asset’s phase 3 trial, Verify, is expected to be fully enrolled by the end of the year and yield data later in 2024.

Uniqure is due to release data on its Huntington’s gene therapy AMT-130 in the second quarter so news should emerge any day now. As well as safety and biomarker measures, investors probably need to see some preservation of function to support punchy sellside numbers.

Prothena has also shown itself to be a canny dealmaker over the years, licensing its amyloidosis project PRX004 to Novo Nordisk in 2021; an anti-Tau Mab, PRX005, to Celgene in 2018; and prasinezumab, a Parkinson’s disease asset, to Roche back in 2013. Perhaps some are betting that it can repeat the trick with the Alzheimer’s Mab PRX012 – the company currently boasts a $4bn valuation.  

Previous iterations of this analysis show that not all of these assets will end up in the hands of a partner. And this is not always important, as another regular name in this analysis shows. Argenx had long featured thanks to Vyvgart, a drug that it launched independently and that continues to beat sales expectations. 

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