CSL has moved quickly to spend some of the money freed up by recent cuts, securing an option to buy the phase 3-ready blood clotting specialist VarmX in return for $117 million upfront.
Last month, CSL revealed plans to lay off up to 15% of its workforce and change its mix of R&D spending on internal and external opportunities. CEO Paul McKenzie told investors at the time that the Australian company planned to reinvest about 50% of the savings, adding that “an acquisition or licensing agreement for a new asset” were among the ways the biopharma was considering spending the money.
Now, CSL has struck a deal with Netherlands-based VarmX. The agreement gives CSL an option to buy the company once it sees results from a phase 3 trial of VMX-C001, an engineered recombinant human coagulation factor X.
VarmX has designed VMX-C001 to be insensitive to inhibition by FXa direct oral anticoagulants such as Bristol Myers Squibb’s Eliquis, Daiichi Sankyo’s Savaysa and Johnson & Johnson’s Xarelto. By bypassing the effect of the drugs, VMX-C001 could enable normal blood clotting in patients who have taken the anticoagulants.
VarmX received FDA clearance in July to run a phase 3 trial in patients who need urgent surgery. At one time, the biotech was aiming to start a pivotal phase 2/3 program in early 2024.
The CSL deal secures funding for the phase 3 trial as well as for manufacturing and pre-launch activities. VarmX raised 32 million euros ($38 million) in a series B round in 2020 and followed up with 30 million euros ($44.8 million) in series B2 cash in 2023. Since then, the company has disclosed 15 million euros ($17.7 million) in follow-on equity funding.
CSL could provide an exit for VarmX’s investors. If CSL takes up its option to buy VarmX, investors could receive up to $388 million in payments up to the commercial launch of VMX-C001. With VarmX targeting a commercial launch in 2029, the deal also includes a further $1.7 billion in potential sales-based milestones.
Portola Pharmaceuticals won accelerated FDA approval for the anticoagulant reversal agent Andexxa in 2018 and was bought by Alexion for $1.4 billion two years later. AstraZeneca, which acquired the drug in its takeover of Alexion, received a complete response letter when it applied for full approval last year. The rejection followed an FDA advisory panel that questioned Andexxa’s safety profile.
Andexxa generated $219 million for AstraZeneca last year, but the Big Pharma recorded $504 million in impairment charges after stopping promotion of the drug. Perosphere Pharmaceuticals was developing a rival candidate, ciraparantag, but that program has suffered its own setback amid a series of takeovers. The sponsor terminated the most recent ciraparantag study on ClinicalTrials.gov in April.
The upshot is there remains an opportunity for a medicine that bypasses all direct oral anticoagulants, allowing physicians to use the drug in emergencies when a patient’s treatment history is unknown. The challenges faced by Andexxa point to the value of next-generation products also having a lower risk of thromboembolic events and a simpler dosing regimen than AstraZeneca’s product does.