Eli Lilly is increasing its ache drug pipeline with the acquisition of SiteOne Therapeutics, a startup whose lead program addresses the identical goal as a Vertex Prescribed drugs product whose approval earlier this 12 months ushered in a brand new class of non-opioid ache drugs.
Beneath deal phrases introduced Tuesday, Lilly has agreed to pay as much as $1 billion to purchase South San Francisco-based SiteOne. The pharma big didn’t present an extra breakdown of the monetary particulars of the deal, apart from to say the sum is inclusive of an upfront fee in addition to regulatory and business milestones. SiteOne’s drug candidate, STC-004, is on monitor to start Section 2 testing.
SiteOne develops medicine that concentrate on sodium channels, that are discovered primarily within the peripheral nervous system. This method is meant to keep away from the dependancy dangers posed by opioid ache medicine that hit receptors within the central nervous sytem. The function that the sodium channels NaV1.7 and Nav1.8 play within the transmission of ache indicators has been identified for many years. The problem for drug analysis has been figuring out small molecules able to blocking these channels with out additionally hitting different sodium channels and inflicting antagonistic results.
SiteOne has expertise that identifies small molecules able to selectively hitting the pain-transmitting sodium channels and modulating them. The title SiteOne refers to a binding web site on NaV1.7 that allows the startup’s medicine to be selective to that concentrate on, SiteOne scientific co-founder and CEO John Mulcahy instructed MedCity Information in a December 2024 interview. SiteOne’s STC-004 is an oral small molecule designed to focus on NaV1.8.
In February, SiteOne reported Section 1 outcomes exhibiting STC-004 was quickly absorbed with a half-life that helps once-daily dosing. The drug was effectively tolerated throughout all doses whereas will increase within the ache threshold of members point out the molecule engaged its goal, the corporate mentioned. Primarily based on these outcomes, the startup had deliberate to advance the drug to Section 2 testing in acute and continual peripheral ache within the second half of this 12 months. When the acquisition closes, that research will turn out to be the duty of Lilly.
“The worldwide burden of continual ache continues to extend, and an efficient non-opioid remedy stays elusive,” Mark Mintun, Lilly group vice chairman neuroscience analysis and growth, mentioned in a ready assertion. “Lilly is keen to proceed the event of STC-004 with the excellent SiteOne workforce as a part of our efforts to advance novel, addiction-free ache therapies.”
Lilly’s pipeline lists three packages in Section 2 testing for varied kinds of ache. Probably the most superior of them is mazisotine, a small molecule agonist of SSTR4 designed to modulate somatostatin signaling, regulating irritation and ache. A Section 2 take a look at that enrolled greater than 400 members with diabetic peripheral neuropathy is predicted to be full this July, in line with an investor presentation that accompanied Lilly’s report of first quarter 2025 monetary outcomes.
The opposite mid-stage packages are LY3848575, a monoclonal antibody designed to bind to epiregulin, stopping that protein from binding to its receptors as a possible remedy for continual ache, and LY3857210, a small molecule inhibitor of P2X7, an ion channel that prompts neuronal and non-neuronal cells in peripheral and central nervous system ache pathways. In Section 1 , Lilly is testing a small molecule AT2R antagonist and 6 further drug candidates for targets that stay undisclosed.
SiteOne, initially shaped in 2010, was based mostly on Mulcahy’s Ph.D. analysis at Stanford finding out toxins that concentrate on sodium channels; the startup has expertise licensed from the college. Amgen was an early collaborator and investor within the startup, although an alliance on NaV1.7 medicine ended when the pharma big exited neuroscience R&D. Late final 12 months, Novo Holdings led a $100 million Sequence C spherical of financing, which SiteOne was planning to make use of to finance medical growth of its lead program.
Beneath a collaboration with Vertex began in 2022, SiteOne is growing NaV1.7 inhibitors for ache; further particulars haven’t been disclosed. Vertex’s Journavx is the product of the pharma firm’s inner NaV1.8 analysis. This twice-daily capsule was awarded FDA approval in January as a remedy for moderate-to-severe acute ache in adults, making it the primary in a brand new class of ache medicine. Journavx has additionally reached Section 3 testing in diabetic peripheral neuropathy and Section 2 testing in painful lumbosacral radiculopathy.
Photograph: Craig F. Walker/The Boston Globe, by way of Getty Photos
