Calliditas Therapeutics AB (NASDAQ: CALT) (NASDAQ Stockholm:CALTX) today showcased impressive interim data from the Phase 2 trial of its lead product candidate, setanaxib, in patients with the serious and commonly diagnosed squamous cell carcinoma of the head and neck (SCCHN). Analyses revealed promising and encouraging early clinical progression-free survival (PFS) results, further confirming setanaxib’s proposed anti-fibrotic mode of action.
This interim study involved a data set of 20 patients with recurrent or metastatic SCCHN, of which 16 had evaluable tumor size and PFS related results. Moreover, 12 of these had tumor biopsies before and after the treatment, which were subject to transcriptomic analysis and evaluated pathology markers including SMA, Foxp3 regulatory T cells and PD-L1 CPS.
However, caution is strongly advised as the small sample size and heterogeneity of the patient population could limit the accuracy of the inferences derived from this analysis.
The transcriptomic analysis demonstrated that the two most prominent pathways affected by the treatment were related to fibrosis, confirming the postulated mechanism of action concerning the control of myofibroblastic fibroblasts and the associated clinical studies.
The identified pathways included the Idiopathic Pulmonary Fibrosis Signaling Pathway and the Hepatic Fibrosis/Hepatic Stellate Cell Activation Pathway.
Pathology analysis revealed that tumors of patients who received setanaxib treatment had heightened immunological activity, which was observed through increases in Foxp3 and PDL-1 CPS markers.
Although baseline SMA levels were not balanced between the groups, and samples were limited in size, the observed changes in the tumors still showed promise for setanaxib’s potential to reduce SMA levels.
The data from the 16 evaluable patients in the study reveal promising results. Of these, 7 showed progression-free survival with either stable disease or partial response, with 6 out of the 7 patients being in the setanaxib arm and 1 in the placebo arm.
What’s more impressive, is that 6 of the 7 patients were still on the study drug at the data read out, with the longest period of time on drug being reported to be a remarkable 21 weeks, all attributed to a patient in the setanaxib arm.
With exciting clinical and transcriptomic results, our trial is well on the way to success. We are encouraged by the data which suggests a beneficial impact on two fibrosis-related signalling pathways, backing up our pipeline programmes.
Our full trial will read out on tumour size and progression-free survival next year, and we look forward to unlocking the potential of setanaxib in disease areas where there are limited treatment options. It is an exciting prospect for us and we can’t wait to see the results.
We are delighted to have seen such positive results from our interim analysis in a patient population requiring more treatment options. Our dedicated team of investigators at test sites around the world are working together with us to put the finishing touches on the study and we look forward to the results. As Chief Medical Officer, Richard Philipson, commented, “We are pleased with these encouraging interim data.”
In an exciting breakthrough for cancer treatment, a randomized, placebo-controlled, double-blind, proof-of-concept Phase 2 study is currently underway to ascertain the effect of setanaxib 800 mg twice daily combined with pembrolizumab 200 mg IV, administered every 3 weeks, on 50 patients with moderate or high CAF-density tumors.
This standard treatment regimen for SCCHN requires biopsies to be taken prior to randomization and again after at least 9 weeks of treatment, and the trial hopes to conclude with final data collection in 2024.
Calliditas Therapeutics is a commercial stage biopharma company revolutionizing the field of novel treatments for orphan diseases, with an initial focus on renal and hepatic diseases. Their lead product, TARPEYO®/Kinpeygo®, has been granted both accelerated approval by the FDA and conditional marketing authorization by the European Commission, and is currently being commercialized throughout the EU.
Additionally, they have two other clinical trials underway for primary biliary cholangitis and head and neck cancer. Their common shares are listed on Nasdaq Stockholm (ticker: CALTX) and their American Depositary Shares are listed on the Nasdaq Global Select Market (ticker: CALT), exemplifying their commitment to making an impact on the orphan healthcare field.