It was a busy week in the biotech sector with important regulatory and pipeline updates. Among these, Cassava Sciences, Inc SAVA nosedived following the failure of a late-stage study, while Alnylam Pharmaceuticals ALNY gained following FDA nod for label expansion of its key drug.
Recap of the Week’s Most Important Stories:
SAVA Stock Plunges on Study Data
Cassava Sciences stock crashed after the company announced that the late-stage study REFOCUS-ALZ on lead pipeline candidate, simufilam, in mild-to-moderate Alzheimer’s disease (AD) did not show a significant reduction in co-primary endpoints of cognitive or functional decline versus placebo in patients with mild-to-moderate Alzheimer’s disease.
Top-line data indicate that the phase III study REFOCUS-ALZ did not meet any of the prespecified co-primary, secondary and exploratory biomarker endpoints.
In November 2024, SAVA reported that the candidate failed to meet the primary endpoints in the late-stage RETHINK-ALZ study, which evaluated simufilam in patients with mild-to-moderate AD.
Consequently, Cassava announced plans to discontinue the REFOCUS-ALZ study and its intention to report top-line data from that trial, including the complete 52-week dataset and a large portion of the 76-week data. Simufilam continued to demonstrate a favorable overall safety profile.
SAVA will discontinue all efforts to develop simufilam for AD and expects to phase out the program by the end of the second quarter.
GSK Obtains FDA Nod for Antibiotic
GSK plc GSK obtained FDA approval for its oral antibiotic gepotidacin for treating uncomplicated urinary tract infections (uUTIs) in female adults and pediatric patients (12 years and above). This approval for Blujepa marks the introduction of a new class of oral antibiotics for uUTIs in nearly 30 years.
Per GSK, the drug targets a different part of the bacteria than most existing antibiotics, making it effective against resistant strains. Additionally, the risk of bacterial resistance is low, as mutations would need to occur in both enzymes that Blujepa targets.
The drug has been designed to treat uUTIs caused by susceptible microorganisms like Escherichia coli, Klebsiella pneumoniae, Citrobacter freundii complex, Staphylococcus saprophyticus and Enterococcus faecalis.
The FDA approval is supported by data from two late-stage studies, EAGLE-2 and EAGLE-3, which showed that Blujepa is at least as effective as nitrofurantoin, a leading standard-of-care (SOC) therapy for uUTIs. Besides uUTI, GSK is also evaluating the safety and efficacy of Blujepa to ceftriaxone plus azithromycin in patients with uncomplicated urogenital gonorrhoea caused by Neisseria gonorrhoeae.
GSK currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
ALNY Stock Up on Drug Label Expansion
Alnylam Pharmaceuticals ALNY obtained FDA approval for the label expansion of its lead drug, Amvuttra (vutrisiran), for treating the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
ALNY’s Amvuttra, an RNAi therapeutic, was previously approved in the United States for treating adult patients with polyneuropathy associated with transthyretin-mediated amyloidosis (hATTR-PN). It is also marketed in the EU for treating hATTR-PN in adult patients with stage 1 or stage 2 polyneuropathy.
The FDA’s latest nod makes Amvuttra the first and only therapeutic approved in the United States for both ATTR-CM and hATTR-PN in adults, marking a milestone. The approval was based on positive results from the phase III HELIOS-B study of the drug, which met all primary and secondary endpoints across both the overall and monotherapy populations, each with statistical significance.
OPTN Surges on Acquisition Deal
OptiNose OPTN stock skyrocketed after the company announced that it was set to be acquired by Paratek Pharmaceuticals, a private biotech. This deal will add OPTN’s approved product, Xhance (fluticasone propionate), to Paratek Pharmaceuticals’ commercial portfolio.
Xhance is an innovative drug-device therapy for chronic rhinosinusitis (CRS), with or without nasal polyps. The acquisition enables OptiNose to expand Xhance’s market reach, addressing long-standing challenges in adoption.
Per the terms of the agreement, all outstanding shares of OptiNose will be acquired by Paratek for $9 per share in cash, with the potential for an additional $5 per share in contingent value rights, contingent upon Xhance achieving certain revenue milestones. This brings the total deal value to up to $330 million.
The upfront cash payment of $9 per share represents a 50% premium over OptiNose’s closing share price on March 19, 2025. The significant premium impressed investors. The transaction is expected to be completed in mid-2025, subject to the fulfillment of certain customary and regulatory closing conditions.
Performance
The Nasdaq Biotechnology Index has lost 0.97% in the past five trading sessions and REGN’s shares have lost 4.88%. In the past six months, shares of MRNA have plunged 47.64%. (See the last biotech stock roundup here: Biotech Stock Roundup: Patient Death Hits SRPT, INCY Study Data & More)
Zacks Investment Research
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.
Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report
