Plozasiran

iStock, ismagilov With the second quarter earnings period underway, BioSpace examines five biotechs with meaningful catalysts that analysts and investors are watching. Biotechs are heading into the second quarter earnings period ready to pressure their Big Pharma peers with new drug launches. More than ever, smaller biotechs are choosing to go at it alone to market their drugs, rather than partner up or sell. This has created a dynamic environment among these upstart companies, with plenty of eyes watching to see if they succeed. From Agios Pharmaceuticals to Ultragenyx, BioSpace takes a look at five biotechs with major catalysts to report on over the next few weeks. ezstandalone.cmd.push(function () { ezstandalone.showAds(129); }); Earnings Q2 earnings arrive as pharma picks up steam, buoyed by dealmaking, policy tailwinds Biopharma is entering its second-quarter earnings season riding high on a wave of massive deals and venture capital flow, plus a clearing of regulatory and policy overhangs. What can industry watchers expect to hear on the upcoming investor calls? July 15, 2026 · 6 min read · Tristan Manalac Read more Agios Pharmaceuticals Q2 stock performance: 35% Earnings date: July 30 After reporting the failure of a Phase 2 study for tebapivat in sickle cell disease, Agios’ management will have to show confidence in the biotech’s remaining pipeline when reporting second quarter earnings. Last week, Agios announced that the next-generation PK activator would be discontinued after failing to differentiate from the company’s own mitapivat or Novo Nordisk’s etavopivat. Analysts hadn’t given tebapivat a lot of credit heading into the readout, but the candidate served as a “narrative enricher for longer-term franchise durability,” according to Truist Securities. The failure of the candidate puts more “concentrated risk” on mitapivat, just as the drug class gets more competition from Novo Nordisk. Mitapivat is already approved as Pyrukynd for pyruvate kinase (PK) deficiency and Aqvesme for alpha- or beta-thalassemia. In the second quarter presentation, Truist will be watching for details on the thalassemia launch and the drug’s potential in sickle cell disease with tebapivat out of the running. The drug is under FDA review via the accelerated pathway for the indication with a decision date set for November 1. Arrowhead Pharmaceuticals FY3Q stock performance: 567% Earnings date: August 4 Arrowhead Pharmaceuticals has already begun its fiscal fourth quarter of 2026 on strong footing, with the announcement last week that two Phase 3 studies of plozasiran in severe hypertriglyceridemia (sHTG) were successful . The results sent the company’s shares up 20% that day to $91.48, while rival Ionis Pharmaceuticals dipped. Analysts had been anticipating the readout in the leadup to fiscal third quarter earnings. Beyond the outstanding results of those sHTG trials, analysts are watching for a second half readout for Arrowhead’s obesity programs , ARO-INHBE and ARO-ALK7. ARO-INHBE has already shown a doubling of the weight loss achieved by Eli Lilly’s tirzepatide in an interim readout from a small group of patients in two Phase 1/2a trials. “ARWR is ahead of peers across both the INHBE+ALK7 programs, w/ multiple paths forward depending on data,” Jefferies wrote in an earnings preview. Arrowhead is also expecting data for the Alzheimer’s disease therapy ARO-MAPT in the third quarter, which could help validate the company’s blood-brain barrier delivery tech. On the commercial side, Arrowhead celebrated its first ever approval late last year, when plozasiran was approved to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). As of the company’s last earnings call, the drug was “trending better” than Ionis’ rival product, according to Jefferies. The firm is expecting sales of about $6 million for the quarter. C-suite Caught In Sarepta ‘Downdraft,’ Arrowhead Becomes RNAi Knight in Shining Armor Sarepta’s troubles had nothing to do with Arrowhead’s assets, and yet both companies have seen their stock prices decline this past month. BioSpace caught up with Arrowhead’s Chris Anzalone to talk about the biotech’s role as an RNAi pipeline savior. August 12, 2025 · 7 min read · Annalee Armstrong Read more BridgeBio Q2 stock performance: 125% Earnings date: TBD BridgeBio is on track to deliver a strong second quarter report, as Attruby continues to steal market share in the crowded transthyretin amyloid cardiomyopathy (ATTR-CM) space, according to Jefferies. The drug is expected to make sales of $215 million for the quarter thanks to increases in front-line uptake, the firm predicted in a July 19 note to clients. This growth is expected to continue quarter-over-quarter going forward, which will in turn build investor confidence in BridgeBio’s next three planned launches in rare disease, Jefferies said. The company is working toward FDA approvals for BBP-418 in limb girdle muscular dystrophy type 2, infigratinib in alternating hemiplegia of childhood and encaleret in autosomal dominant hypocalcemia type 1. Infigratinib was approved in 2021 for cholangiocarcinoma and is marketed as Truseltiq. Each of these could mean $1 billion opportunities for BridgeBio, Jefferies noted, while Attruby is on track to deliver $3–$4 billion in peak sales in ATTR-CM. The drug faces competition from Pfizer’s Vyndaqel and Vyndamax, Alnylam’s Amvuttra and Ionis/AstraZeneca’s eplotersen in the space, but fears that BridgeBio will lose significant market share are subsiding as the market becomes more mature, according to Jefferies. Ionis Pharmaceuticals Q2 performance: 190% Earnings date: July 29 Ionis is well ahead in the marketplace with Tryngolza, which now has two approved indications in familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG) but is still facing immense pressure from Arrowhead. Analysts are expecting Ionis’ overall revenue to decline by more than half when the company reports earnings this week, as the price of the drug declines significantly to match the entry into a more common sHTG indication. Guggenheim analysts have revenue estimates above consensus at $209 million. This would mark a year-over-year decline of about 54% from last year, when revenue was $452 million . The slightly optimistic take as compared to consensus stems from higher than expected estimates for R&D collaboration revenue, since Recordati paid a $30 million upfront fee for a partnership with Ionis at the end of June. With the price reduction, Guggenheim has lowered its sales expectations to $2.9 billion as compared to $3.2 billion prior. But the analysts are optimistic that the dip for Tryngolza, which is now approved for FCS and sHTG, will be short lived as the sHTG label highlighted the therapy’s ability to reduce the risk of acute pancreatitis. Recent data also showed its ability to normalize hepatic fat. Moving forward, analysts will be focused on three upcoming Phase 3 readouts, including the Lp(a) HORIZON trial of pelacarsen, which is testing the drug for reducing lipoprotein(a). Rare diseases Ionis Paves Its Own Path as Initial Tryngolza Launch Defies Expectations Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026. February 18, 2026 · 3 min read · Annalee Armstrong Read more Ultragenyx 2Q performance: -1.33% Earnings date: TBD Ultragenyx has been through the wringer this year, witha high pro for a bone disease drug and an FDA rejection for a rare disease gene therapy. Heading into the third quarter, analysts are keenly watching for any details on a Phase 3 readout for apazunersen in Angelman syndrome, which could help turn the ship around. Jefferies anticipates peak worldwide sales of about $1.87 billion for the drug in 2036 if ultimately approved. Ultragenyx executives have had to make some tough decisions following the events of 2025, including implementing an expense reduction plan and layoffs . They also sold off two priority review vouchers that were awarded with the recent approvals of rare disease therapies Mepsevii and Crysvita. The biotech is awaiting two key regulatory decisions. Pariglasgene brecaparvovec, an AAV8 gene therapy, is under review for glycogen storage disease type 1a with an FDA action date of August 23. And rebisufligene etisparvovec, an AAV9 gene therapy, was accepted for priority review by the FDA with a decision date of September 19. Jefferies believes that both launches could be successful because there is unmet need in both indications. As for sales, Jefferies predicts Crysvita to come in between $500 and $520 million while Dojolvi could take home $100–110 million.

Less than a month after Ionis Pharmaceuticals scored an FDA expansion for Tryngolza to become the first drug to specifically treat severe hypertriglyceridemia (sHTG), Arrowhead Pharmaceuticals has countered with head-turning data that could set the company up for a matching nod. Testing Redemplo (plozasiran) in approximately 750 patients with sHTG, Arrowhead aced two phase 3 trials, achieving all primary and secondary prespecified endpoints. In both studies, when compared to placebo, Redemplo provided a 78% reduction in the pancreatitis rate, which was statistically significant. Pancreatitis attacks can land patients in the hospital for extended stays and be life-threatening. In the SHASTA-3 and SHASTA-4 studies, patients on Redemplo also had median triglyceride (TG) reductions of 79% and 81% at month 12 versus a 27% rate for those on placebo. Additionally, among a subset of patients at the highest risk—defined as those with TGs above 880 mg/dL and a prior medical history of acute pancreatitis—there was a 100% reduction in pancreatitis, as there were zero events. Arrowhead said it plans to file for FDA approval of Redemplo to treat sHTG by the end of this year, with submissions in other regions to follow. While Ionis has a first-to-market advantage, Arrowhead has an edge in convenience as Redemplo is injected every three months as opposed to monthly for Tryngolza. In a release, Arrowhead CEO Christopher Anzalone, Ph.D., pointed to other advantages as well. “We continue to see plozasiran data as best in class with respect to safety, activity, efficacy, and convenience,” Anzalone said. “These compelling Phase 3 data in a broad sHTG study population that closely resembles today’s diverse patient landscape demonstrate plozasiran’s potential to dramatically change the way people are treated.” Full data from the trials will be presented at the European Society of Cardiology Congress in Munich on Sunday, Aug. 30, with the company planning to conduct a webcast and conference call discussing the results the following day. The FDA originally approved Redemplo in November of last year to treat the genetic disorder familial chylomicronemia syndrome (FCS). Ionis scored its FDA nod for Tryngolza in the indication in December of 2024. While there are only about 3,000 people in the United States with FCS, sHTG brings a much larger patient population. The disorder affects roughly 3 million in the U.S., though last month, Ionis CEO Brett Monia said that the company is focusing on the high-risk group, which includes roughly 500,000 who have TG levels above 880 mg/dL. Adding spice to the rivalry of the two California companies are lawsuits they filed against each other last year. Ionis alleged that Arrowhead copied its patented mRNA technology to develop Redemplo, asking for an unspecified amount of monetary damages. Arrowhead argued in its lawsuit that the Ionis patent at the heart of the dispute was “invalid and not infringed by Arrowhead’s planned commercialization” of Redemplo. While Tryngolza and Redemplo are both designed to lower the body’s production of the protein APOC-3, they use a different mechanism of action. Tryngolza is an antisense oligonucleotide (ASO), while Redemplo is a small interfering RNA (siRNA) compound.