San Francisco biotechnology Tenaya Therapeutics has shared “encouraging” data from its first patients in the MyPEAK-1 clinical trial of TN-201, despite some concerns over elevations in liver enzymes.
The US clinical-stage biotechnology company’s heart disease treatment TN-201 is being developed for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy (HCM), among a cohort of three patients. Results found that TN-201 was generally well tolerated with biomarkers of cardiac muscle strain and injury remaining largely stable.
However, during the study (NCT05836259) all three patients experienced isolated elevations in liver enzymes associated with TN-201 treatment. The company said these were not associated with other signs or symptoms of liver damage and were well managed with the administration of corticosteroids. One patient did experience an asymptomatic enzyme elevation that was designated as a serious adverse event (SAE).
Following the announcement yesterday (17 December) the company’s stock value dropped again from $2.93 per share, down to $1.42 after the announcement. It has been a rough year for the company financially, after starting out the year at a stock value of $2.09 per share, the company saw a promising rise in March, up to a height of $6.80 per share after the company shared pre-clinical data of TN-201, before dropping to its current level. Also yesterday, the company’s senior vice president Chihiro Saito announced she would be stepping down from her position.
Milind Desai, investigator for the MyPeak-1 Phase Ib/II clinical trial, said: “The initial patients enrolled in the MyPEAK-1 Phase Ib/II clinical study are like many we see in our clinic: relatively young adults whose HCM is keeping them from having an adequate quality of life, including being able to perform activities of daily living and whose disease is progressing despite treatment interventions, putting them at significant risk of dire complications.
“The goal of gene therapy is to halt or even reverse the steady decline in MYBPC3-associated HCM by addressing the underlying genetic cause of disease. Initial data from this first-in-human clinical trial of TN-201 demonstrate tolerability and early evidence of protein expression support additional investigation to build on these findings.”
The company said that whilst there were some other AEs, they were primarily mild, transient or reversible. Additionally, patients one and two have successfully tapered off immunosuppressives and all three patients remain in the still ongoing study.
Elsewhere in the field of HCM, Edgewise Therapeutics has announced positive top-line findings from two clinical trials where its EDG-7500 showed benefit as a potential treatment for obstructive HCM. Meanwhile, Bristol Myers Squibb (BMS) has unveiled new long-term follow-up results from the EXPLORER-LTE cohort of the MAVA-long-term extension (LTE) clinical trial of CAMZYOS.
