Guard shares soar as new data show clinical benefit despite missing primary endpoint

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Guard Therapeutics is staging a remarkable recovery following a setback in its phase 2 clinical trial for kidney injury treatment. Despite the trial being halted early due to a primary endpoint failure, the Swedish biotech is boldly declaring this setback as “insignificant.” Instead, they are highlighting promising longer-term efficacy data that points to a bright future for their asset.

The phase 2 trial initially aimed to determine whether RMC-035, a genetically engineered protein variant known for organ protection, outperformed a placebo in preventing acute kidney injury (AKI) within 72 hours after heart surgery. An interim analysis of the primary endpoint data suggested that RMC-035 did not surpass the placebo in this aspect. Nevertheless, Guard continued to monitor the 177 participants for longer-term outcomes.

“We are very excited by the results of this phase 2 trial with RMC-035. Whereas the chosen primary (acute) endpoint AKI failed, it is important to note that AKI merely serves as a short-term prognostic indicator of clinically relevant outcomes, including long-term kidney function and Major Adverse Kidney Events (MAKE). The actual efficacy of RMC-035 assessed by these hard clinical outcomes is far above expectations and makes the chosen primary endpoint, AKI, irrelevant in this context. The study has clearly demonstrated the efficacy signals needed to advance the project towards a future pivotal study. With these results we are back on track, strengthened in our confidence to establish a new and unique treatment for the prevention of kidney injuries in heart surgery and proceed with the clinical development of RMC-035 as planned. We are eagerly awaiting further discussions with regulatory authorities, and we look forward to providing more information about the clinical pathway shortly” 

– Tobias Agervald, CEO at Guard Therapeutics

Now, Guard has disclosed the extended data on secondary endpoints. The company designed the trial in a way that any p-values < 0.1 would be statistically significant, and it achieved this on two of its 90-day secondary endpoints.

One notable finding was the improvement in eGFR change, a measure of kidney function, from baseline on Day 90, favouring RMC-035 over the placebo, with a p-value of 0.06. Additionally, Guard linked RMC-035 to a reduction in major adverse kidney events through Day 90. The treatment arm exhibited a rate of 6.7%, compared to the placebo group’s 15.9%, resulting in a p-value of 0.047.

“The top-line results from Guard Therapeutics’ phase 2 clinical trial AKITA with RMC-035 are very exciting, as they represent a potential breakthrough for the protection of kidney function in open-heart surgery. The consistency between improved kidney function and MAKE reduction in the stable phase after surgery is compelling because these outcome measures offer distinct perspectives on assessing the kidney-protective treatment effect. As the lead investigator of the AKITA trial, I am eager to see the compound’s further development progress and ultimate contribution to improving patient outcomes.” 

– Prof. Dr. Alexander Zarbock

However, it’s important to note that the primary endpoint faced a significant setback. The incidence of AKI in the RMC-035 group was 50.6%, higher than the 39.8% seen in the placebo group. Guard attributed this result, and possibly the primary endpoint’s failure, to a drug-induced increase in serum creatinine among RMC-035 recipients. As a result, the company argued against placing undue focus on the AKI data.

This unique perspective resonated positively with some investors, as Guard’s stock price tripled in response to the announcement, surging from 0.16 Swedish kronor ($0.01) to 0.48 Swedish kronor. However, it’s worth noting that the stock price remains below its pre-April levels when news of the primary endpoint failure initially emerged. This intriguing turn of events suggests that Guard Therapeutics is not giving up on its kidney injury treatment just yet, with longer-term data offering a glimmer of hope amid initial setbacks.

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