Spinal Muscular Atrophy

Spinal muscular atrophy treatment Spinraza (nusinersen) became Biogen’s best-selling drug in Q1 2025. Image credit: Shutterstock/ Tada Images. Biogen has unveiled Q1 2025 revenues above estimates, spurred by strong sales of rare disease assets amid a sales dropoff from its multiple sclerosis business. Quarterly revenues for the neurological disease drugmaker were $2.43bn, above analyst group William Blair’s estimates of $2.3bn.  The Q1 sales performance represents a 6% increase compared to the same period in 2024, with Biogen ramping up commercial duties for existing drugs and buying assets during that time. One of those assets is Skyclarys, a drug picked up in a $7.3bn acquisition of Reata Pharmaceuticals in September 2023. The treatment for the neurodegenerative disorder Friedreich’s ataxia generated revenue of $124m in Q1, a value that had already taken into account the impact seen in US sales due to Medicare discount dynamics. Approved in the US, Europe, and UK, amongst others, Skyclarys has a high growth ceiling. GlobalData’s Pharma Intelligence Centre forecasts sales of $1.3bn by 2031. GlobalData is the parent company of Pharmaceutical Technology . Skyclarys is one of the drugs spurring a change of the guard in Biogen’s pipeline. The company is well known for its host of multiple sclerosis treatments, such as Vumerity (diroximel fumarate), Tysabri (natalizumab), and Avonex (interferon beta-1a). But, sales for these treatments have declined over the years as biosimilars entered the market. The company’s multiple sclerosis business shrank 11% in Q1 2025 compared to the same period last year. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence “Biogen is really, a tale of two companies in my view. There’s one company which has been a multiple sclerosis company, and that portfolio, as you all know, has been gradually declining,” said Biogen’s CEO Chris Vibacher in a Q1 earnings call. “But there’s a new Biogen emerging. And when I look at the rare disease business […] we actually have a commercial portfolio that we’re actively promoting that’s now gotten to be about 45% of our product revenue. And those products mostly have a very long runway to continue to grow,” Vibacher added. Alongside Skyclarys, Biogen’s rare disease business is headed by spinal muscular atrophy treatment Spinraza (nusinersen). Spinraza generated global Q1 2025 sales of $423.9m, up from $341.3m in Q1 2024. This increase, coupled with the decline of Tysabri, meant Spinraza was Biogen’s best-selling drug in Q1 2025. Leqembi’s “modest growth” Another key part of Biogen’s business model is its Alzheimer’s segment, now solely headed by Leqembi (lecanemab) after the company discontinued Aduhelm (aducanumab) at the start of 2024. Biogen splits revenue from Leqembi with co-developer Eisai equally, the latter reporting Q1 sales of $96m for the drug. The US, where Leqembi got a full approval in July 2023, accounted for $52m of those sales. Biogen has already secured key milestones for Leqembi this year. In January, the US Food and Drug Administration (FDA) approved maintenance dosing of Leqembi, a move expected to expand its market share. Then in April, the Leqembi was authorised for use in the European Union (EU). Biogen said the Q1 sales for Leqembi represented “continued sequential growth.” William Blair analyst Myles R. Minter said in a research note that: “Skyclarys had a strong quarter, but the commercial focus remains on Leqembi, which has seen only modest growth, in our view.” In the mergers and acquisitions space, Biogen failed a $469m takeover bid of Sage Therapeutics in January.

Novartis is starting the year on a strong note as several of its core medicines beat Wall Street’s sales projections, prompting the drugmaker to shift its full-year guidance to a higher bar. In the first three months of 2025, the company’s net sales increased by 15% to $13.2 billion, beating Wall Street’s consensus of around $13 billion, the Swiss pharma said in a Tuesday release . With that, Novartis now expects net sales in 2025 to grow by high-single digits, up from a previous range spanning the mid- to high-single digits. This means Novartis could reach up to $55 billion in sales this year, Jefferies analysts said in a same-day note. Novartis’ Tuesday update follows its positive full-year 2024 earnings, which it described as one of the strongest in its history after its yearslong rejig as a pure-play pharma company. It achieved $50.3 billion in sales last year. As for its clinical pipeline, the company axed two programs: a chondrogenic asset called LRX712 and a radiopharmaceutical candidate dubbed GIZ943. Novartis dropped LRX712 after it showed “insufficient effect on knee cartilage” in a Phase 2 trial of patients with osteoarthritis, a company spokesperson told Endpoints News in an email. It nixed GIZ943, which was in development for ovarian and lung cancer, based on a Phase 1 risk-benefit assessment, the spokesperson said. The first half of 2025 should be especially strong, CFO Harry Kirsch said in a media call. Novartis expects generic competition for its heart failure drug Entresto, cancer medicine Tasigna and blood disorder treatment Promacta from midyear onwards. Products that performed better than the Street expected in Q1 included asthma drug Xolair ($456 million), spinal muscular atrophy therapy Zolgensma ($327 million) and Still’s disease treatment Ilaris ($419 million), according to Jefferies analysts. Meanwhile, Kisqali sales in the quarter grew 56% to $956 million, reflecting its position as “the CDK4/6 inhibitor of choice across metastatic and early breast cancer,” CEO Vas Narasimhan said on the call. And sales of cholesterol-lowering medicine Leqvio increased 72% to $257 million as it remains on track to reach blockbuster status this year. The company also highlighted that it won three FDA approvals in Q1, which should bolster its performance this year. In March, its radioligand drug Pluvicto was greenlit for certain prostate cancer patients who haven’t received chemotherapy. Pluvicto reached blockbuster status for the first time last year. Also in March, Fabhalta secured its third indication for an ultra-rare kidney disease called C3 glomerulopathy. And earlier this month, Vanrafia was approved for a rare kidney disease called IgA nephropathy.

iStock, Alona Horkova Biohaven will use the money to bankroll commercial preparations for the spinocerebellar ataxia drug candidate troriluzole, which is currently under FDA review with a decision expected in the third quarter. Biohaven has secured a potential $600 million non-dilutive infusion from private investment firm Oberland Capital, which will help fund its clinical and commercialization efforts, the company announced on Monday. Oberland will hand over $250 million once the deal closes, which Biohaven expects to occur “on or before April 30, 2025.” An additional $150 million tranche will be paid at the biopharma’s option and only after its spinocerebellar ataxia (SCA) drug troriluzole secures FDA approval, plus other specified conditions. The final $200 million balance will come once the parties reach a mutual agreement on certain “permitted strategic acquisitions and related costs and expenses.” In return for its investment, Oberland will be able to receive a regulatory milestone payment—which Biohaven can make quarterly through December 31, 2030—plus tiered single-digit royalties on net sales of troriluzole for up to 10 years. According to Biohaven, Monday’s funding arrangement will help it prepare for the commercial roll-out of troriluzole, in anticipation of an FDA approval. The drug, an investigational modulator of the glutamate neurotransmitter, is currently undergoing regulatory review for SCA, with a decision expected in the third quarter . Troriluzole’s prospects seem mixed. In September 2024, topline pivotal data showed that the drug candidate could slow patients’ rate of decline by 50% to 70% versus untreated counterparts. Troriluzole also delayed SCA progression by 1.5 years to 2.2 years. On the other hand, in August 2023, the FDA refused to even review troriluzole’s drug application, flagging that a Phase III trial used to support that application had failed to meet its primary endpoint. Oberland’s funding will also support Biohaven’s efforts across its developmental portfolio—a capital infusion that will help the company regain its footing in the clinic after a series of stumbles. In March, Biohaven reported that its potassium channel agonist BHV-7000 failed a Phase II/III trial in bipolar mania, unable to elicit significant improvements in manic symptoms in more than 250 patients with bipolar I disorder. In November last year, the myostatin and activin receptor blocker taldefgrobep alfa likewise flopped in the Phase III RESILIENT SMA study, failing to significantly boost motor function versus placebo in patients with spinal muscular atrophy. A few months earlier, in May 2024, Biohaven reported that its protein degrader BHV-1300 could reduce levels of autoantibody IgG in a Phase I healthy-volunteer trial. Investors were unimpressed by these data, however, sending the company crashing some 12% in its aftermath. At the time, analysts at William Blair said Biohaven’s findings fall “below the high bar” that many investors were hoping for.