Inebilizumab-cdon

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: