Pipeline and regulatory updates are in focus in the biotech sector. Among these, BridgeBio Pharma, Inc. BBIO gained on approval of cardiovascular drug while a few others faced setbacks.
Recap of the Week’s Most Important Stories:
BBIO Stock Up on Drug Approval
BridgeBio Pharma, Inc. announced that it has obtained FDA approval for its pipeline candidate acoramidis for the treatment of adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization. The candidate has been approved under the brand name Attruby. BBIO stock surged on the news.
The FDA approval is based on positive results from the phase III ATTRibute-CM study, in which Attruby significantly reduced death and cardiovascular-related hospitalization and improved quality of life. The approval came ahead of the targeted action date of Nov. 29.
Per BBIO, Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and demonstrate benefits on cardiovascular outcomes.
The drug was generally well-tolerated. The most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation.
SAVA Stock Plunges on Study Failure
Cassava Sciences SAVA recently reported that its lead pipeline drug, simufilam, failed to meet the primary endpoints in a late study for Alzheimer’s disease (AD).
Top-line results from the ReThink-ALZ study, which evaluated simufilam in patients with mild-to-moderate AD, showed that treatment with the drug failed to achieve a significant reduction in cognitive or functional decline in AD patients when compared to placebo over a 52-week period, as assessed by the ADAS-COG12 and ADCS-ADL scales. The study failed to meet any pre-specified secondary and exploratory biomarker endpoints.
Based on the above study results, Cassava also decided to discontinue the phase III ReFocus-ALZ study, which evaluated simufilam in AD patients over a 76-week period. It will also discontinue the open-label extension study on the drug. Shares of Cassava Sciences plunged on the news as simufilam is the only candidate in Cassava’s pipeline.
AMGN’s Obesity Data
Amgen AMGN announced 52-week top-line data from a phase II study on MariTide, its GLP-1 therapy being developed for obesity and related conditions. The data from the double-blind dose-ranging study showed that in people who were obese or overweight but without type II diabetes,
MariTide caused approximately 20% average weight loss over 52 weeks without reaching a weight loss plateau. In patients with type II diabetes who were obese or overweight, the weight loss reduction was approximately 17% at 52 weeks, with concurrent hemoglobin A1C (HbA1c) reduction of up to 2.2 percentage points. The weight loss in this patient group was also without a plateau, which means there is potential for weight loss beyond 52 weeks.
MariTide also demonstrated other cardiometabolic benefits like sustained improvements in blood pressure, triglycerides and inflammation markers like high-sensitivity C-reactive protein (hs-CRP). The candidate also did not cause any significant increase in free fatty acids.
In terms of safety and tolerability, the most common adverse events were mild, transient gastrointestinal issues, primarily nausea and vomiting, often associated with the first dose. The discontinuation rates due to adverse events were approximately 11%. MariTide also did not result in any significant change in bone mineral density.
Despite the encouraging data, Amgen stock declined, probably as the weight loss did not impress investors much.
APLT Stock Plunges on Setback
Applied Therapeutics APLT announced that the FDA has issued a Complete Response Letter (“CRL”) for the new drug application (NDA) seeking approval for lead candidate, govorestat, to treat classic galactosemia. The CRL stated that the FDA has reviewed the application and cannot approve the NDA in its current form due to deficiencies in the clinical study results submitted in support of the application.
Applied Therapeutics is evaluating the FDA's feedback. Shares of APLT plummeted following the disappointing news.
APLT’s govorestat (also known as AT-007) is a novel central nervous system penetrant ARI, which is currently being developed for three rare neurological diseases. The NDA for govorestat for the galactosemia indication was accepted by the FDA in late February under its Priority Review pathway.
Performance
The Nasdaq Biotechnology Index has gained 4.09% in the past four trading sessions and Moderna’s shares have gained 13.44%. In the past six months, shares of MRNA have lost 71.68%. (See the last biotech stock roundup here: Biotech Stock Roundup: SAGE, INCY Suffer Setbacks, JAZZ Wins Oncology Drug Approval & More)
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What's Next in Biotech?
Stay tuned for more pipeline updates.
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