Rusfertide acetate

iStock, Sean Pavone Beginning this week in Chicago, the American Association for Cancer Research’s annual conference will feature presentations that could have far-reaching implications for breast and blood cancers and more. Kicking off Friday and running through June 3 at McCormick Place Convention Center in Chicago, the 2025 annual meeting of the American Association for Cancer Research will include some of the most cutting-edge developments in cancer, which experts believe could have far-reaching implications across the industry and for medical practice. “As we head into the 2025 ASCO Annual Meeting, there are several key breast cancer trials that reflect major advancements in how we approach treatment,” Jason Mouabbi, assistant professor of Breast Medical Oncology at The University of Texas MD Anderson Cancer Center, told BioSpace in an email. Meanwhile, Lore Gruenbaum, chief scientific officer of The Leukemia & Lymphoma Society, is looking forward to a host of blood cancer-related presentations, including those that explore minimal residual disease (MRD) testing in multiple myeloma—a development she called “exciting.” MRD testing, Gruenbaum added, “will likely change clinical development strategies for novel agents and may provide significant time savings” for drugmakers. Besides breast and blood cancers, many of the late-breakers at ASCO 2025 are focused on melanoma, gastric, prostate and lung cancers. Breast and Blood Cancers Amid the variety of presentations at ASCO 2025, the buzz around breast and blood cancers stands out. One such attention-grabber is Takeda and Protagonist Therapeutics’ investigational hepcidin mimetic rusfertide, which the partners are developing for polycythemia vera, a rare but potentially deadly blood malignancy. “Rusfertide is a promising emerging treatment option,” Gruenbaum told BioSpace, noting that the molecule could provide an effective alternative for patients who do not adequately respond to standard therapies. In March, Takeda and Protagonist released topline data from their Phase III VERIFY study, which showed that rusfertide met its primary efficacy endpoint, eliciting treatment responses in 77% of patients versus 33% of placebo counterparts. Rusfertide likewise aced key secondary endpoints, including reducing phlebotomies, boosting hematocrit control and improving patient-reported outcomes. While Gruenbaum had not yet seen the ASCO abstract , “the data available so far indicate the potential for Rusfertide to significantly reduce the need for phlebotomies and improve hematocrit control in patients with polycythemia vera,” she said, adding that an improvement in quality of life data “could be very meaningful to patients.” VERIFY has been granted a plenary spot at the conference. In breast cancer, several presentations have drummed up considerable pre-conference interest. One of these is AstraZeneca’s Phase III SERENA-6 trial, which MD Anderson’s Mouabbi said is “one of the most anticipated studies at ASCO.” SERENA-6 combines the pharma’s oral SERD camizestrant with a CDK4/6 blocker to treat patients with HR-positive, HER2-negative advanced breast cancer with emergent ESR1 mutations. High-level outcomes posted in February showed the camizestrant regimen significantly improved progression-free survival in these patients versus standard-of-care aromatase inhibitors. AstraZeneca did not reveal specific data at the time—but will do so now at ASCO. “This trial is especially compelling because it’s the first to show a statistically significant improvement in progression-free survival in this setting, without requiring radiographic progression,” Mouabbi told BioSpace . “It’s a potential paradigm shift in how we manage endocrine resistance.” Like VERIFY, SERENA-6 is also a plenary presentation . AstraZeneca will also have additional Phase III data from DESTINY-Breast09, which will be “another standout” presentation, according to Mouabbi. The trial centers on the antibody-drug conjugate Enhertu—which AstraZeneca is developing in partnership with Daiichi Sankyo—testing it with or without Roche’s Perjeta as a first-line option in patients with HER2-positive metastatic breast cancer. Last month, the partners announced that Enhertu significantly improved progression-free survival in Destiny-Breast09, though again without providing specific data. “This is a significant development for HER2+ patients, offering a less chemotherapy-intensive option with robust efficacy,” Mouabbi said. Other notable breast cancer presentations, according to Mouabbi, include Merck and Gilead’s ASCENT-04/KEYNOTE-D19 , which combines the anti-TROP2 ADC Trodelvy with the mega-blockbuster Keytruda, and Pfizer and Arvinas’ VERITAC-2 trial, which in March returned mixed results for their investigational PROTAC degrader vepdegestrant. Bi – and Trispecifics Aside from specific presentations, experts are also looking forward to advancements in various treatment modalities. Such developments could give rise to new treatment opportunities for what Scott Kopetz, professor of Gastrointestinal Medical Oncology at The University of Texas MD Anderson Cancer Center, called “cold tumors”—those that are unlikely to elicit an immune response and are, in turn, less susceptible to immunotherapies. In particular, Kopetz has his eye on bispecific antibodies, which he said could usher in “a new era in VEGF-targeted therapies.” These emerging therapies “not only inhibit angiogenesis [the formation of new blood vessels] but also enhance immune responses,” he continued, noting that such a mechanism could lead to “promising efficacy and safety in advanced solid tumors.” As a result, bispecific antibodies could yield “potential breakthroughs” in the treatment of immunologically cold tumors, such as castration-resistant prostate and gallbladder cancers, Kopetz said. According to Gruenbaum, bispecifics, and now also trispecific antibody platforms, could likewise open up new treatment avenues for blood cancers. ASCO, she continued, will witness initial clinical data for two trispecifics, including Johnson & Johnson’s JNJ-5322 and Ichnos Glenmark Innovation’s ISB-2001, both of which are being developed for relapsed/refractory multiple myeloma. There is also now a growing number of bispecific therapies for blood cancers, some of which are inching toward regulatory approval, which they could secure “in the near future,” Gruenbaum said. Drug developers are also starting to move these agents into earlier lines of therapy, she added. For David Piwnica-Worms, chair of Cancer Systems Imaging at MD Anderson, ASCO 2025 will be an opportunity to learn about some of the latest developments in radiopharmaceuticals. “We’re really seeing two trends,” he told BioSpace in an email. The first deals with identifying biomarkers for cancers earlier in the disease cycle, while the second is focused on identifying novel isotopes. Both of these trends, Piwnica-Worms said, underscore that “this is an exciting time” for the radiopharma space, since “the field is rapidly expanding with new isotopes and new molecular targets.” China in Focus In a May 21 note to investors, analysts at Truist Securities made an astute observation: nearly a third of presentations at ASCO involve assets that came from Chinese companies. The considerable presence of China at the conference reflects “the country’s increase in R&D efforts, as per the Truist note. “China’s biotech innovation footprint has clearly expanded on the global stage.” Christiana Bardon, co-managing partner at biotech investment firm MPM BioImpact, made a similar observation. In an email to BioSpace , she pointed out that whereas Chinese companies “historically have focused on making ‘fast-follower drugs,” they are now taking an active leadership role in innovation and are advancing “disruptive, first-in-class medicines.” Bardon pointed to Summit Therapeutics’ and its Chinese partner Akeso’s PD-1/VEGF bispecific ivonescimab, “which may displace Keytruda’s dominant position in cancer immunotherapy.” According to Bardon, many Big Pharma players have been playing catch-up to Summit and Akeso in this space: “Since Summit Therapeutics in-licensed ivonescimab from Akeso, we have seen BioNTech, Merck, and Pfizer reach for additional PD(L)1 x VEGF assets from China.” Bardon sees no real problem with this, however. “Increasing innovation will ultimately benefit patients, regardless of where it originates,” she said. Paul Moore, chief scientific officer at Zymeworks, agreed, noting that China’s ascent as a worldwide leader in cancer innovation creates a “more competitive environment” across the oncology space, in turn pushing U.S. companies to be more “innovative and efficient.” Drugmakers, Moore offered, should become more “intentional” about their pipelines, look for opportunities to differentiate themselves and “prioritize thoughtful trial design.” Studies should pay particular consideration to the underlying biological mechanisms of diseases, while also taking advantage of companies’ respective “unique areas of expertise,” he added. Such an approach, Moore explained, is likely to result in drug candidates “with novel biological mechanisms and strong clinical potential.” Development efficiency will no longer be enough, Moore added. “Real progress will come from therapies...that offer something new and impactful for patients.” In all, he said that healthy competition between global leaders in biopharma innovation could lead to a “steel-sharpens-steel phenomenon.”