BridgeBio Pharma, Inc. (BBIO) has secured FDA approval for Attruby (acoramidis) for treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM). This approval, based on the Phase III ATTRibute-CM study, highlights Attruby's ability to significantly reduce cardiovascular death and cardiovascular-related hospitalization, while also improving patient quality of life. The approval came ahead of the FDA's target action date of November 29.
Attruby's Clinical Impact
According to BridgeBio, Attruby stands out as the first and only approved product with a label indicating near-complete stabilization of TTR (transthyretin). This stabilization preserves TTR's native function as a transport protein for thyroxine and vitamin A, leading to improved cardiovascular outcomes. The drug was generally well-tolerated, with mild side effects such as diarrhea and abdominal pain that did not require discontinuation of treatment.
Cassava Sciences' Simufilam Disappointment
In contrast, Cassava Sciences (SAVA) reported that its lead drug candidate, simufilam, failed to meet primary endpoints in a late-stage study for Alzheimer’s disease (AD). The ReThink-ALZ study, evaluating simufilam in patients with mild-to-moderate AD, showed no significant reduction in cognitive or functional decline compared to placebo over 52 weeks, as measured by ADAS-COG12 and ADCS-ADL scales. Consequently, Cassava has discontinued the Phase III ReFocus-ALZ study and the open-label extension study for simufilam.
Amgen's MariTide Shows Promise in Obesity Treatment
Amgen (AMGN) announced positive 52-week top-line data from a Phase II study of MariTide, a GLP-1 therapy for obesity and related conditions. The double-blind, dose-ranging study demonstrated approximately 20% average weight loss over 52 weeks in obese or overweight individuals without type II diabetes. In patients with type II diabetes who were obese or overweight, MariTide resulted in approximately 17% weight loss and a reduction in HbA1c by up to 2.2 percentage points. These weight loss effects did not plateau, suggesting potential for continued weight loss beyond 52 weeks.
MariTide also exhibited other cardiometabolic benefits, including improvements in blood pressure, triglycerides, and inflammation markers like high-sensitivity C-reactive protein (hs-CRP). The most common adverse events were mild, transient gastrointestinal issues, with discontinuation rates due to adverse events around 11%.
Applied Therapeutics' Govorestat Faces Regulatory Hurdle
Applied Therapeutics (APLT) received a Complete Response Letter (CRL) from the FDA for its New Drug Application (NDA) for govorestat, a treatment for classic galactosemia. The FDA cited deficiencies in the clinical study results as the reason for not approving the NDA in its current form. Applied Therapeutics is currently evaluating the FDA's feedback. Govorestat, also known as AT-007, is a central nervous system penetrant ARI being developed for three rare neurological diseases. The FDA had accepted the NDA for govorestat under Priority Review in late February.
