Luspatercept-AAMT

Pictured: FDA sign at its office in Washington, DC/iStock, JHVEPhoto Geron Corporation announced Friday the FDA has approved Rytelo (imetelstat) for patients with lower- to intermediate-risk myelodysplastic syndromes, a group of blood cancers caused by blood cells that are poorly formed or don’t function properly. Rytelo’s approval sent Geron’s stock surging more than 30% in Friday morning trading. Rytelo is approved for myelodysplastic syndromes (MDS) patients with transfusion-dependent anemia who do not respond or are ineligible for the standard-of-care treatment, erythropoiesis-stimulating agents (ESA). In addition to being first-in-its class, the treatment is also Geron’s first approved drug after 34 years in business. “For patients with lower-risk MDS and anemia who are transfusion dependent, we have very few options today and often cycle through available therapies, making the approval of Rytelo potentially practice changing for us,” Rami Komrokji, vice chair of the Malignant Hematology Department at Moffitt Cancer Center and an investigator in the pivotal IMerge trial, said in a statement. MDS is a group of blood cancers in which blood cells in the bone marrow do not mature or become healthy blood cells. In lower-risk MDS, it often progresses to require intense management of its key symptoms like anemia and can eventually cause organ dysfunction and heart complications. In the Phase III IMerge trial, patients on Rytelo had significantly higher rates of red blood cell transfusion independence (RBC-TI) over placebo for at least 24 weeks—28% in the treatment arm versus 3% on placebo. In these responders, RBC-TI was sustained for a median of 1.5 years. Administered every four weeks intravenously, Geron described the most common side effects of the drug as “manageable and short-lived" with Grade 3/4 adverse reactions of neutropenia (72%) and thrombocytopenia (65%) lasting a median of less than two weeks. In August 2023, Bristol Myers Squibb’s Reblozyl snagged a label expansion to position it as a first line treatment for anemia in patients with low-risk MDS with no prior ESA treatment. BMS’ molecule is an erythroid maturation agent that drives the production of red blood cells, minimizing the need for transfusion. When pitted against ESA treatments in a Phase III trial, 58.5% of patients on Reblozyl achieved RBC-TI that lasted at least 12 weeks, versus 31.2% for ESA-treated patients. GlobalData analysts project Reblozyl sales to reach $3.2 billion by 2029, while BMS has set its sights on $4 billion-plus aspirations for the drug. Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

Today, several brief updates from BioPharma Dive Reporter Gwendolyn Wu, who spent this week attending BIOs annual meeting in San Diego, as well as notes on Geron, AbbVie and Deerfield Management.Food and Drug Administration Commissioner Robert Califf, speaking on a Wednesday panel, told BIO president John Crowley that he did not want to see the pharmaceutical industry “completely dependent“ on China, an issue thats been in focus as Congress considers legislation targeting certain Chinese companies. “There could be a catastrophic thing like an earthquake or a geopolitical war, so you want to have a distributed supply chain that is resilient in case something goes wrong,“ Califf said. Gwendolyn WuIn the same conversation, Califf described himself as a radical advocate for the GLP-1 drugs the FDA has approved for obesity. But, true to form, he said more should be done in preventive care. “I do have sort of nightmares that my great grandchildren are going to read about a country called America where, on average, people are gaining a pound per year because of their eating, and instead of fixing that, this country decided to invent a class of drugs that cost $20,000 a year to reverse basic eating habits,“ he said. Gwendolyn WuGeron, a 34-year-old biotechnology company, won its first drug approval Thursday when the Food and Drug Administration cleared Rytelo for myelodysplastic syndromes, or MDS. The FDA OK came after clinical setbacks for Geron and positions the therapy to challenge Bristol Myers Squibbs Reblozyl in treating people with lower-risk MDS and anemia. Notably, the FDA didnt put a warning for hematological side effects on the drugs label as analysts had suspected. That could boost buyout interest in Geron, as well as in Rytelos launch, Stifel analyst Stephen Willey wrote in a research note. Shares climbed by more than 20% Friday. Ben FidlerShares of Shionogi fell sharply in trading on the Tokyo stock exchange Friday, possibly due to an announcement that the company is considering a new development strategy for an obesity drug its developing. In an R&D presentationFriday, the company revealed preliminary data from Phase 2 testing showing treatment with its drug led to less than 5% weight loss among study participants. As a result, Shionogi may develop it in combination with other therapies.Ned PagliaruloAbbVie spent $10 billion last year to acquire ImmunoGen, drawn in large part to the potential of the antibody-drug conjugates the biotechnology company had developed. One of those, an ovarian cancer treatment called Elahere, has now succeeded in another study, AbbVie said Thursday. The drug shrank tumors in half of treated participants, who had platinum-sensitive ovarian cancer.Full data will be presented at an upcoming medical meeting. Ned PagliaruloFrank Nestle, the former chief scientific officer and head of research at Sanofi, is starting a new career at the biotech company creator Deerfield Management. Nestle, who had joined Sanofi in 2016, oversaw the French drugmaker's rise in immunology as it built a pipeline beyond its top-selling Dupixent.In addition to serving as CEO of Deerfield Discovery and Development, Nestle will join the firm's therapeutics team as a partner. Gwendolyn Wu '