Pegcetacoplan

Biogen\'s \"open innovation model\" aims to push the company toward more partnerships, licensing and venture investments.\n From persevering with a tricky Alzheimer’s program to securing a late-stage CD38-directed antibody and posting encouraging lupus data, Biogen has remained in the news recently—and that’s no accident. The neurology- and immunology-focused biopharma is in the middle of transitioning to what leaders have dubbed “the new Biogen.” This strategy involves embracing partnerships, investment and an entrepreneur’s approach in order to replenish the company’s pipeline and propel it into the future.In the last couple of years, Biogen has brought in new leadership to head up the company’s R&D and venture arms. The aim is to rebuild Biogen\'s long-term pipeline, as Head of Immunology Research Nick Wilson, Ph.D., told Fierce, in a “fundamental difference in how we\'re approaching our research engine.”From academic partnerships to licensing deals and acquisitions, Biogen\'s leaders have described a shift in the company’s philosophy away from an inward-focused mindset and toward an “open innovation model” that prioritizes external investment, partnerships and licensing.“We\'ll use all the levers at our disposal,” said Wilson, who joined Biogen in January 2025 after leading immunology teams at Bristol Myers Squibb and Gilead. “It’s a deliberate effort we\'ve put in play over the last year that is integrated to build the pipeline of the future.”Biogen’s foundations rest upon a historic multiple sclerosis (MS) franchise, with drugs like Tecfidera, Tysabri and Vumerity establishing the company as a leader in the indication. But pressure from competitors has led to years of sales declines.Biogen CEO Chris Viehbacher made the distinction between the MS side of the company and other indications with more promise, describing Biogen as a “tale of two companies” in an earnings call last year. By May 2025, the transition was already taking place, with 45% of Biogen’s revenue coming from medicines outside its MS franchise.In its 2025 full-year earnings presentation, Biogen celebrated its revenue turnaround. For the first time since 2019, the company posted a year-over-year increase, when revenue bumped up 2% to $9.9 billion. The company noted that its growth products, which included the company’s spinal muscular atrophy (SMA) drug Spinraza and its Eisai-partnered Alzheimer’s treatment Leqembi, generated about $3.3 billion in revenue last year, up 19% from the year prior. But with Spinraza’s patent set to expire in 2030 and Leqembi’s in 2032, Biogen is looking outward to reload its pipeline. In 2024, Viehbacher announced a push to bring in more partnered assets after the company’s $7.3 billion buyout of Reata, which resulted in Biogen obtaining Skyclarys, the first FDA-approved prescription medication for Friedreich’s ataxia (FA) in adults and adolescents. In his interview with Fierce, Wilson pointed to Biogen’s $1.15 billion HI-Bio acquisition in 2024, with immunology products expected to reach the market in 2027 and 2030, as another example of the model in practice. He also noted Biogen’s $70 million upfront payment for a Vanqua Bio immunology asset last fall as a recent move that reflects the philosophical change. “The open innovation model is something we\'ve been talking about, but now we\'re actually putting it into action,” Wilson said. “And there\'s more that we\'ve got cooking there as well.”For both its R&D and venture arms, Biogen is looking to grow while reducing risk. Balancing acquisitions of early-stage assets with candidates that have a shorter path to the market through licensing, partnerships and acquisitions is a goal of the company’s new investment arm.Chelsea Johnson, Ph.D., joined the company to lead the New Ventures team in October 2025 after several years as a principal at biotech-focused Atlas Venture. “Biogen is building out this venture\'s effort to complement our open innovation strategy, where we\'re balancing our internal research with both BD opportunities and investing opportunities,” Johnson told Fierce during the same interview. “We will look to gain more exposure to the exciting science innovation that\'s happening in the biotech ecosystem.”The venture team will mostly stay close to Biogen’s strengths, focusing on immunology, neurology, rare disease and renal targets. But Johnson said there is also an opportunity to invest in new spaces.Because the New Ventures arm was launched relatively recently, Biogen told Fierce it is still in the process of evaluating investment opportunities. However, the team has plans to lay down some money later this year.“We can take a lot of risk off the table early on because we can drive early to proof of mechanism, proof of biology, and potentially early proof of concept opportunities,” Wilson said. “So that diversifies some of the risk in the overall Biogen pipeline.” Biogen’s equity investments will allow the company to secure a stake in a biotech’s entire portfolio while it considers a more serious partnership. Last year, Biogen made an offer to buy neurology-focused Sage Therapeutics for $442 million after being an equity partner for years. In that case, the offer was ultimately unsuccessful.“We view equity investing as the lead up where we can invest in earlier-stage programs or programs with risk that still needs to be discharged to hopefully set those companies up for success to be partnering with us in the future,” Johnson explained to Fierce.She emphasized that the investment arm is different from the average venture fund, as it is integrated across everything from business development to R&D at Biogen, with moves made in conjunction with the internal pipeline’s strengths and weaknesses.“Our primary goal is to invest in companies that we can partner with in the future and bring those assets into our internal pipeline,” Johnson added.As Biogen’s Head of Neurology Research, Ross Jeggo, Ph.D., puts it, the open innovation model allows Biogen to use its resources more strategically. “It gives you more agility, because you can turn that resource up or down, which allows you to take some of that expertise from outside and bring it in,” Jeggo told Fierce.The new model isn’t all about growth; it\'s also about right-sizing the company’s investment. Jeggo said Biogen has made some tough decisions on the way to refocusing the pipeline.“We had a few too many projects in different areas that weren\'t moving forward with the right speed, so we have refocused the pipeline,” Jeggo said. “The refocus is set on the strategic areas where we see we have the highest chance of success.”In late May, Biogen ended development of a Parkinson’s disease candidate after a phase 2b trial missed its primary endpoint. By that point, Biogen had already paid Denali $400 million in 2020 to partner on the program.Jeggo said Biogen’s refocus in neurology is centered around amyotrophic lateral sclerosis and neuromuscular conditions. Alzheimer’s remains important as well, as Biogen and Ionis Pharmaceuticals have committed to registrational development of their tau-targeting candidate despite some disappointing data. In May, Biogen shared that the Celia study evaluating diranersen, a tau-targeting antisense oligonucleotide, missed its primary endpoint. But Biogen argued that the trial was the first evidence from a randomized phase 2 study of a tau-directed therapy with biomarker impact and cognitive benefit in early Alzheimer’s. The reductions in tau pathology and cognitive benefits led Guggenheim Securities analysts to hail the data as the “moment of truth for tau.”In addition to April’s deal with TJ Biopharma to expand the rights to the Greater China region for a phase 3-stage CD38-directed antibody, this year has also seen Biogen acquire Apellis Pharmaceuticals for about $5.6 billion in March, granting the biopharma access to the approved medicines Syfovre for the eye condition geographic atrophy and Empaveli for two rare kidney diseases.The deals show how Biogen aims to balance long-term opportunity with more immediate impact. “That\'s only going to be achieved through tapping into our open innovation model,” Jeggo said. “We couldn\'t have done that without [our] existing internal pipeline.”“The enablement from the executive committee and working with Chelsea and her team to place some of these bets in our ecosystem is going to be the way we can sustain the pipeline growth,” he added.Jeggo also acknowledged the venture team’s ability to strategically deploy limited funds. “As a greedy research unit head, it gives us optionality,” he said.“We\'ve got a limited bandwidth of resources from a budget perspective,” he pointed out. “If we don\'t have the expertise or the budget to execute something, it gives us an optionality that\'s very appealing.”For Wilson, the new philosophy is all about equipping the company to continue its turnaround. “This open innovation model gives us agility to use a fungible resource in a good way,” he said. “You can kind of tap into things when timing is right, but if you need to pull back on things, you can also do that without major structural changes within the company,” Wilson added. “It’s that entrepreneurial spirit.”

Sobi's presentations highlight research across multiple rare diseases in haematology including PNH, ITP, HLH and myelofibrosis. STOCKHOLM, June 11, 2026 /PRNewswire/ -- Sobi® (STO: SOBI) today announced that new clinical and real-world data will be presented at the 2026 Congress of the European Hematology Association (EHA2026), taking place June 11–14 in Stockholm, Sweden. The presentations include data across paroxysmal nocturnal haemoglobinuria (PNH), immune thrombocytopenia (ITP), primary haemophagocytic lymphohistiocytosis (HLH), and myelofibrosis. The data include real-world evidence and post hoc analyses for Aspaveli® (pegcetacoplan), Doptelet® (avatrombopag), Gamifant® (emapalumab), and Vonjo® (pacritinib). "As EHA comes to Stockholm this year, we are pleased to present new clinical and real-world evidence spanning multiple rare diseases," said Lydia Abad-Franch, MD, Head of R&D and Medical Affairs and Chief Medical Officer at Sobi. "These presentations reflect the breadth of our ongoing research across PNH, ITP, HLH and myelofibrosis, and continue to build understanding of treatment outcomes in areas of high unmet need." About Aspaveli®/Empaveli® (pegcetacoplan) Aspaveli/Empaveli (pegcetacoplan) is a targeted C3 and C3b therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Aspaveli/Empaveli is approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the United States, European Union, and other countries globally. It is the first treatment for C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients 12 years of age and older approved in the United States, European Union, and other countries globally. About the Sobi® and Apellis (now part of Biogen) collaboration Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialisation rights for systemic pegcetacoplan. Apellis has exclusive U.S. commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy. About Doptelet® (avatrombopag) Doptelet® is an orally administered thrombopoietin receptor agonist (TPO-RA) that increases platelet count. It is approved in the United States, European Union and Japan for thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, and for ITP in adults according to local labeling. In the United States, Doptelet® is also approved for pediatric patients 1 year and older with persistent or chronic ITP who have had an insufficient response to a previous treatment. About Gamifant (emapalumab) Gamifant is the only approved anti-interferon gamma (IFNy) monoclonal antibody. Gamifant works by binding to and neutralising interferon gamma (IFNy). When interferon gamma (IFNy) is secreted in an uncontrolled manner, hyperinflammation occurs within the body. Gamifant is indicated for administration through intravenous infusion over one hour. Gamifant is approved in the US for the treatment of adult and paediatric patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Gamifant is also approved in the US for the treatment of adult and paediatric patients with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still's disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS . About Vonjo (pacritinib) Vonjo is a kinase inhibitor that is indicated in the US for the treatment of adults with intermediate or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis with a platelet count below 50 × 109/L. This indication is approved under accelerated approval based on spleen volume reduction. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Sobi® Sobi is a global biopharma company unlocking the potential of breakthrough innovations, transforming everyday life for people living with rare diseases. Sobi has approximately 2 000 employees across Europe, North America, the Middle East, Asia and Australia. In 2025, revenue amounted to SEK 28 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. Visiting address Norra Stationsgatan 93A, Stockholm +46 8 697 20 00 [email protected] This information was brought to you by Cision . The following files are available for download: 21% more press release views with Request a Demo