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Hello and welcome back to the new Endpoints Weekly. It was another busy week in the biopharma world and we’ve got all of the headlines for you below. We also had an exclusive from pharma reporter Max Bayer this week, on how a meeting of CDC vaccine advisors has been rescheduled for the middle of April. The meeting was previously scheduled for late February but was postponed amid a broader pause on panels across federal health agencies. Keep it locked in at Endpoints for biopharma news, exclusives, analysis and more. — Max Gelman A 16-year-old boy died from liver failure after receiving Sarepta’s gene therapy for Duchenne muscular dystrophy Elevidys, the company said . Testing showed the patient had a recent cytomegalovirus infection (CMV), which the treating physician said was “a possible contributing factor,” according to Sarepta. Sarepta said this is the first death possibly related to Elevidys. More than 800 patients have received the gene therapy in trials and post-approval. Acute liver injury is a known side effect of the therapy, which was granted broad approval by the FDA last year. No deaths were reported in Sarepta’s pivotal studies of Elevidys. The company said it will “continue to gather and analyze information” related to the event. Sarepta reported the death to relevant health authorities, and said it “intends to update the prescribing information to appropriately represent this event.” Duchenne muscular dystrophy is an inherited disease that causes progressive muscle degeneration and weakness. It occurs primarily in young boys. 🤔 One of Roivant’s portfolio companies, Immunovant reported Phase 3 data for a rare disease drug called batoclimab this week, saying it succeeded in its primary goal. But rather than take batoclimab to market, Immunovant will instead shift its focus toward a program farther behind in the pipeline, IMVT-1402. Researchers were testing the drug in an autoimmune disorder called myasthenia gravis, which destroys the connection between nerves and muscles. Roivant CEO Matt Gline said although he was pleased with the outcome, batoclimab’s data were not stronger than already approved drugs — most notably Vyvgart, a multibillion-dollar product from argenx. In particular, the high dose only achieved a two-point reduction over placebo on a symptom measurement scale. “Our view is, why fight that fight when we can fight the better fight in a couple of years, when we can launch ‘1402,” Gline told Endpoints this week. And Roivant won’t treat this like another TL1A scenario, with Gline saying definitively that the company is not actively looking to out-license batoclimab or house it in another subsidiary. Roivant took that route with its anti-TL1A molecule a few years ago, buying it on the cheap from Pfizer, shepherding it through a mid-stage trial and then selling it to Roche for $5 billion-plus. However, Gline did not rule out a deal entirely, if an appealing one were to come along. The outcome also raises the stakes for IMVT-1402’s eventual readout, as Immunovant believes the program can produce better efficacy results than batoclimab while also improving on safety measurements. By the time IMVT-1402 reaches its pivotal readouts, there may be other approved drugs beyond Vyvgart: Amgen and Johnson & Johnson both recorded Phase 3 wins in myasthenia gravis last year for Uplizna and nipocalimab, respectively. ✅ Alnylam received approval for its RNAi drug Amvuttra in a heart disease known as transthyretin amyloid cardiomyopathy, or ATTR-CM, this week. It comes with a broad label that executives hope to use as an advantage over its competitors Pfizer and BridgeBio. Alnylam is pricing the drug in ATTR-CM at about $476,000 per year, which is the same price used for its other approved indication, hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy. The company has never been profitable, but that could change next year. Alnylam has been gearing up to launch Amvuttra in ATTR-CM for some time, getting into a growing market that analysts expect could reach $10 billion-plus by 2030. Alnylam lost $278 million last year and the CEO, Yvonne Greenstreet, believes the company can make enough in new Amvuttra sales to put the company “on the path” to profitability by the start of 2026. But many observers are skeptical that such goals will be achieved immediately. Analysts who spoke to Endpoints described a tough road for Alnylam ahead, especially for those individuals who haven’t had their disease treated yet. Both of the Pfizer and BridgeBio drugs are taken orally while Amvuttra needs to be injected by a physician. Ultimately, how patients receive treatment may come down to their individual circumstances. Alnylam is hoping its broader label will be one of the biggest keys to profitability . 💰 AstraZeneca has inked a deal to acquire a cell therapy startup for $425 million upfront. The company, called EsoBiotec, is working on an immune-shielded lentiviral vector dubbed ESO-T01 that’s designed to reprogram T lymphocytes into BCMA CAR-T cells within the patient’s body. AstraZeneca has promised up to $575 million more if certain development and regulatory milestones are met. EsoBiotec caught AstraZeneca’s attention at the JP Morgan Healthcare Conference in January, where it presented data that AstraZeneca’s head of oncology and hematology R&D Susan Galbraith said were “really quite impressive.” ESO-T01 entered a China-based trial in multiple myeloma patients in January, and data are expected to read out in the second half of this year. That isn’t the only deal AstraZeneca made this week. The British pharma also struck two R&D deals with Harbour BioMed and Syneron Bio, each of which are potentially worth more than $3.4 billion. Roche also inked a billion-dollar deal this week when it formed a multiyear partnership with Oxford Biotherapeutics. Roche agreed to put down $36 million upfront to work with OBT on finding new targets for antibody-based cancer treatments. OBT stands to make more than $1 billion in milestones and royalties, the companies announced. OBT says its discovery platform, called OGAP-Verify, is more sensitive than immunohistochemistry-based technology and can find targets that might otherwise be missed. And Sanofi said it would pay $600 million upfront for an early-stage cancer and autoimmune disease candidate from Dren Bio. The B cell-depleting antibody is in two Phase 1 trials: one in relapsed/refractory B cell non-Hodgkin lymphoma and another in a range of autoimmune conditions. Sanofi has promised up to $1.3 billion in development and launch milestones. 🧬Just over a year into his role as CEO of Illumina, Jacob Thaysen detailed his plans to expand into multiomics, a field that involves decoding proteins, RNA and other elements of biology. DNA is still core to Illumina’s business (as evidenced by a coiled staircase that mimics a DNA strand at its San Diego headquarters). But Thaysen said biology is “much more complicated.” Many diseases involve intricate biological interactions that DNA alone can’t fully explain. Illumina has faced a series of challenges in recent months, including competition from other companies in the sequencing space, threats to NIH funding that many of Illumina’s academic customers rely on, and China’s ban on imports of Illumina’s sequencers. But Thaysen said he plans to face those challenges head-on. “You can sit on the sideline and eat the popcorn, or you can go in and be a part of the solution,” he said. You can read more about Thaysen’s plans for Illumina’s transformation in Jared Whitlock’s feature. DON’T MISS:

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED