US biotech Samumed has some clinical data to back up its staggering $12bn valuation, coming from a mid-stage trial of its lead candidate for osteoarthritis (OA).
The San Diego company presented data at this week’s American College of Rheumatology (ACR) meeting showing that a single injection of SM04690 into the joints of people with OA of the knee improved pain, joint function and patient global scores.
Samumed came out of stealth mode two years ago with a portfolio of small-molecule drugs aimed at diseases associated with ageing, and has already picked up $650m in funding, most recently a whopping $438m from an equity release in August.
SM04690 is the lead programme in a diverse pipeline largely made up of drugs that target the Wnt pathway, a signalling mechanism that seems to regulate adult stem cells in the body. The company thinks that by modulating Wnt activity in selected tissues it can switch on natural repair mechanisms that tackle the root cause of disease, rather than just symptoms.
In the case of SM04690, a Wnt inhibitor, the drug is intended to help regenerate cartilage, slow down cartilage breakdown, and reduce inflammation in the joint.
The 24-week phase 2b trial in almost 700 patients was presented as a poster at the ACR meeting, and shows that SM04690 improved pain and joint function compared to placebo across a range of doses, and appeared to be safe and well-tolerated. Patient-reported outcomes data also favoured the active drug, and Samumed is now planning to talk to the FDA about a phase 3 programme for the drug it hopes will start early next year.
The biotech has already completed a phase 2a trial over 52 weeks which backed up SM04690’s proposed mechanism of action with data showing that it could reduce the medial joint space width (mJSW), which gives a measure of the amount of cartilage present in the knee joint. No significant differences were seen between placebo and the active drug at 24 weeks in the latest study, although the company says that is because that isn’t enough time for the regeneration to become visible.
Samumed will showcase another of its pipeline drugs this week at the Clinical Trials on Alzheimer’s Disease (CTAD) Congress, which started in Barcelona yesterday. Preclinical data on DYRK1A inhibitor SM07883, which the company says is a potential first-in-class drug for the treatment of Alzheimer’s, will be presented in a poster at the conference.