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iStock, Natalya Kosarevich Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular dystrophy. BridgeBio Pharma’s small-molecule drug candidate significantly improved the levels of a key disease biomarker in a Phase 3 limb-girdle muscular dystrophy study, results that analysts say could pave the way for a U.S. launch early next year. With these findings, announced in a news release on Wednesday , BridgeBio continues “building on the potential of BBP-418 to rapidly improve biomarker levels and functional benefit” in patients with limb-girdle muscular dystrophy (LGMD) type 2I/R9 (LGMD2I/R9), Mizuho Securities told investors in a Wednesday note. The company is planning to application package for BBP-418 with the FDA in the first half of this year, according to BridgeBio’s release, with analysts anticipating a U.S. launch in late 2026 or early 2027. LGMD 2I/R9 is a monogenic and autosomal recessive subtype of LGMD that manifests as skeletal myopathy of the limbs, followed later by progression into the lung and heart muscles. The condition is caused by mutations in the FKRP gene, weakening its function. This leads to defective alpha dystroglycan (αDG), which under healthy conditions helps stabilize muscles. BridgeBio’s answer to LGMD 2I/R9 is BBP-418 , which works by bombarding the FKRP protein with its corresponding substrate, in effect driving increased function of αDG and improving muscle stability. In the Phase 3 FORTIFY trial, BridgeBio enrolled 81 patients who were given either BBP-418 or placebo. At three months, the investigational drug nearly doubled the biomarker αDG, which helps measure muscle stabilization in patients, the company reported in October 2025. Wednesday’s data build on these findings, showing a significant 1.8-times increase in αDG at three months, whereas placebo controls saw “approximately no change” in this biomarker, according to the release. BBP-418 was also able to sustain these “highly statistically significant increases” in αDG through 12 months, BridgeBio added. William Blair also expressed confidence in BridgeBio’s candidate. “While FORTIFY was designed to support an accelerated approval pathway using glycosylated αDG as a surrogate endpoint, based on a meeting BridgeBio held with the FDA, the agency has recommended orienting the NDA toward a traditional approval,” the analysts said in a Thursday note to investors. “We believe [this] is indicative of the strength and consistency of both biomarker and functional efficacy data favoring BBP-418 treatment,” they added. Neuroscience BridgeBio Pops on Late-Stage Limb-Girdle Muscular Dystrophy Data New interim data from a Phase III trial puts the company on track to FDA approval next year in an indication that not only lacks a disease-modifying treatment but suffered significant setbacks after a patient died in a clinical trial for Sarepta’s investigational gene therapy. October 27, 2025 · 2 min read · Dan Samorodnitsky Read more Mizuho also “observed that the favorable effects of BBP-418 vs placebo start early in the treatment”—as early as three months in. This is true even for clinical outcomes, such as the 100-meter timed test and pulmonary function. Taken together, FORTFY’s findings “look highly concordant across both efficacy biomarkers . . . and functional measures,” according to Jefferies, which sent out its own investor note on Wednesday. Consequently, the firm stated a 90%+ confidence rate in a full approval for BBP-418 by the end of 2026 or early 2027. Jefferies anticipates at least $600 million in peak sales, noting that this forecast is “quite conservative.”

As for the prospect of facing sooner-than-expected generic pricing pressure from Vyndaqel copycats in Europe, BridgeBio CEO Neil Kumar said that his company is counting on continued efforts to set Attruby apart from Pfizer's tafamidis franchise. Since BridgeBio scored FDA approval for cardiomyopathy drug Attruby 15 months ago, its launch has progressed smoothly as the treatment has proven a worthy competitor to Pfizer’s powerhouse Vyndamax franchise. All told, the BridgeBio drug's sales scaled upward each earnings period last year, reaching $146 million in the fourth quarter, representing a 35% sequential increase. Still, the cardiomyopathy drug's gains are tempered slightly by a recent move by BridgeBio's rival that rattled investors. Three weeks ago, Pfizer withdrew a patent in Europe that protects its compound tafamidis—known as Vyndaqel in Europe—opening the possibility of generic pricing pressure on Attruby by 2028, and BridgeBio's share price fell by 15%. In turn, BridgeBio used its earnings presentation Tuesday to give investors a dose of reassurance, laying out its expectations on generic tafamidis pressure in Europe and providing more evidence of Attruby's strong launch. While BridgeBio already reported its preliminary fourth quarter sales last month, the company this week filled in some of the blanks on Attruby's growing momentum, noting that 7,804 prescriptions had been written for its drug as of Feb. 20, 2026. Based on that stat, analysts from Mizuho Securities calculated that Attruby is attracting an average of 161 new patients per week, up from some some 143 in January.  “Management noted that this acceleration can be explained by a rapid patient identification, plus prescribers responding to Attruby’s data being put forth,” the Mizuho team wrote, adding that “continued acceleration” should “reinforce” Attruby’s profile. For the full year 2025, Attruby generated sales of $362 million, with 40% of that revenue collected in the fourth quarter. BridgeBio added that its $146 million sales for Attruby in the fourth quarter suggest that the treatment is attracting more than 25% of new patient starts in its cardiomyopathy indication.Analysts from Evercore ISI further highlighted the recent “prescribing momentum” of Attruby in a note to clients this week, adjusting their 2026 worldwide sales projection for the oral medication from $828 million to $1.02 billion. Evercore also increased its peak sales forecast for Attruby from $2.9 billion to $3.5 billion.  As for the prospect of facing sooner-than-expected generic pricing pressure from Vyndaqel copycats in Europe, BridgeBio CEO Neil Kumar said that his company is counting on continued efforts to set Attruby apart from Pfizer's tafamidis franchise.“We have a vastly superior enthalpic binding mode than does tafamidis, which in concert with superior binding kinetics continues to reinforce Attruby’s better stabilization profile,” Kumar said on an analyst call this week. “A recent bevy of literature further supports that increases in serum (transthyretin) are associated reliably with decreases in the relative risk of mortality.” “It is important to separate two things: the legal process around tafamidis and the fundamental strength of Attruby," he added. "Our confidence in Attruby is rooted in its clinical profile and market positioning, not a particular IP outcome,” said Kumar, who added that Pfizer’s withdrawal of its patent was “unexpected.” During the Q&A portion of his company's earnings call, Kumar cited other indications—including pulmonary arterial hypertension, statins and prostate cancer—where second-to-market drugs continued to grow even after the first-to-market product lost its exclusivity.  “We believe physicians are making decisions based on clinical performance, not simply price, and prescribing trends we are seeing are reflecting that,” Kumar said. “Even in a hypothetical scenario involving generic tafamidis, we do not believe a less efficacious product would undermine the role of a clinically differentiated therapy in a serious, progressive disease like ATTR cardiomyopathy.”

Today, a brief rundown of news from Novo Nordisk and Palvella Therapeutics, as well as updates from Gossamer Bio, Vanda Pharmaceuticals and Pfizer that you may have missed.Novo Nordisk on Tuesday announced plans to lower the U.S. list prices of its diabetes and obesity drugs by as much as 50% starting next year. According to Novo, Wegovy and Ozempics wholesale acquisition cost will be $675 per month effective Jan. 1, 2027 reductions of 50% and 35%, respectively, from their current prices. The cuts apply to all doses and also include Rybelsus, an oral medication Novo sells for diabetes. They dont affect the costs involved when people bypass insurance and pay for Novos drugs in cash. Ben FidlerShares of Palvella Therapeutics climbed by nearly 40% Tuesday on positive Phase 3 study results in people with vascular abnormalities known as microcystic lymphatic malformations. Palvella said treatment with its therapy, a type of topical rapamycin gel, hit all main and key secondary goals and will be submitted to U.S. regulators this year. The data could position Palvellas drug as the first approved for the condition, and easily tops most expectations, wrote Mizuho Securities analyst Graig Suvannavejh. The findings also suggest the therapy could be viable in treating other vascular malformations, leaving significant value still waiting to be unlocked, he added. Ben FidlerSan Diego-based drugmaker Gossamer Bio lost more than 80% of its market value Monday after disclosing its sole clinical program failed a late-stage study. Gossamer, in collaboration with Chiesi Group, has been developing an experimental medicine called seralutinib across a couple lung diseases. Fresh results from a study focused on pulmonary arterial hypertension showed that, statistically, the medicine was not significantly better than a placebo on a test that evaluates lung capacity and cardiovascular health. Faheem Hasnain, Gossamers CEO, noted that this measure was narrowly missed, and the overall data clearly demonstrate seralutinib is an active drug in patients with PAH. The company therefore believes further discussions with the Food and Drug Administration regarding a potential path forward are warranted. Jacob BellThe FDA late last week approved a new atypical antipsychotic from Vanda Pharmaceuticals. Now sold as Bysanti, the medication works by inhibiting certain receptor proteins that interact with neurotransmitters like serotonin, dopamine and adrenaline. In the body, Bysanti breaks down into a molecule known as iloperidone, which is also the active ingredient in Vandas already marketed therapy Fanapt. The FDA specifically approved the newer product for schizophrenia and for the acute treatment of manic or mixed episodes associated with bipolar I disorder. Vanda said it expects Bysanti to become commercially available sometime between July and the end of September. Jacob BellPfizer will pay as much as $495 million for partial ownership of an injectable obesity medicine developed by Hangzhou, China-based Sciwind Biosciences. The deal announced Wednesday hands Pfizer exclusive commercialization rights in China to the therapy, which is known as ecnoglutide and already approved in the country for Type 2 diabetes. An approval application for weight management has also been accepted by Chinas drug regulator. Pfizer has been using dealmaking to acquire obesity assets. Last year, it paid $10 billion to nab Metsera and inked a potentially $2 billion licensing deal with YaoPharma. Ben Fidler '