Batoclimab

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:

Switching to IMVT-1402 extends the head starts Immunovant has already ceded to its rivals. \n A phase 3 trial of Immunovant’s FcRn inhibitor batoclimab has hit its primary endpoint. But, having made a second-generation molecule its lead candidate last year, the biotech isn’t currently planning to file for batoclimab approval in myasthenia gravis or chronic inflammatory demyelinating polyneuropathy (CIDP).Immunovant switched its focus from batoclimab to IMVT-1402, another FcRn inhibitor, last year. Talking at the J.P. Morgan Healthcare Conference in January, CEO Pete Salzmann, M.D., said Immunovant’s “base case” was that it would opt against commercializing batoclimab. Neither the phase 3 myasthenia gravis data nor phase 2b data in CIDP have persuaded the biotech to veer away from this strategy.The myasthenia gravis trial linked low and high doses of batoclimab to  4.7- and 5.6-point improvements, respectively, on the MG-ADL symptom and activity scale. Scores improved by 3.6 points in the placebo cohort. The gap between the treatment and control groups caused the trial to hit its primary endpoint.Efficacy correlated to IgG reduction, with Immunovant seeing declines of 64% and 74%, respectively, on the low and high doses. The biotech compared the results to data on argenx’s Vyvgart and Johnson & Johnson’s nipocalimab. Immunovant’s rivals linked their FcRn inhibitors to similar levels of IgG reductions and MG-ADL improvements as the low dose of batoclimab. Despite claiming the high dose “breaks the therapeutic ceiling,” Immunovant isn’t planning to bring the drug candidate to market in myasthenia gravis. The decision reflects evidence batoclimab lowers albumin and, in doing so, affects cholesterol. Believing it has mitigated that problem with IMVT-1402, the biotech presented the batoclimab data as evidence that its next-generation candidate will beat the competition.“What we\'re clearly gonna be able to do better, based on this dataset, is get deeper faster and hold those responses for longer,” Matt Gline, CEO of majority Immunovant shareholder Roivant, said on a call with investors to discuss the data. “That\'s the impact of the deeper IgG suppression that we deliver.”Switching to IMVT-1402 extends the head starts Immunovant has already ceded to its rivals. Argenx won FDA approval for Vyvgart in 2021 and launched a subcutaneous version in 2023. Immunovant is yet to start a phase 3 trial of IMVT-1402 in myasthenia gravis.The biotech confirmed IMVT-1402 is its lead candidate in CIDP after getting a look at phase 2b data on batoclimab in the indication. Immunovant linked the molecule to improvements on multiple CIDP scales, with 84% of people who had at least a 70% drop in IgG meeting its response criteria. But, like in myasthenia gravis, the biotech has decided to take IMVT-1402 forward in the indication. Immunovant has left the door open for batoclimab in thyroid eye disease. The biotech plans to make a final decision on whether to seek approval for its first-generation FcRn inhibitor in the indication after seeing phase 3 data.Investors reacted negatively to the news, sending Immunovant’s stock down 10% to below $17 before the market opened. However, some analysts were more upbeat. Asking a question on Immunovant’s call, Piper Sandler’s Yasmeen Rahimi, Ph.D., said: “The most important thing that you proved, and that\'s incredible, is the debate on whether deeper matters. I think this data today, this morning, really put that to rest.”