Keros Therapeutics (NASDAQ:KROS) CEO Jasbir Seehra outlined the company’s development priorities and partnership plans during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences conference, highlighting plans to initiate a Duchenne muscular dystrophy (DMD) trial for rinvatercept (KER-065) and to engage regulators on a potential phase II program in amyotrophic lateral sclerosis (ALS) later this year.
Focus on TGF-β superfamily signaling
Seehra said Keros is a clinical-stage biopharmaceutical company focused on developing therapies that modulate dysfunctional or imbalanced signaling within the TGF-β superfamily, which the company believes contributes to disease pathophysiology. The company’s pipeline includes its lead program, rinvatercept (KER-065), for neuromuscular diseases and partnered program elritercept for anemia and thrombocytopenia associated with hematologic disorders. Seehra also referenced a broader set of preclinical assets across neuromuscular and neurodegenerative diseases, rare bone and fibrotic diseases, and potential applications in obesity and fertility.
Rinvatercept: mechanism and differentiation
He also emphasized a design feature intended to reduce binding to BMP9, which he said was associated with vascular and bleeding events seen with earlier molecules based on wild-type ActRIIB-Fc. According to Seehra, those bleeding-related events (including telangiectasias) were not observed with KER-065 in the company’s phase I experience.
DMD opportunity and phase I readouts
In DMD, Seehra characterized the disease as severe, progressive, and ultimately fatal, driven by lack of dystrophin protein that results in muscle weakness and degeneration. He said there are no cures and outlined limitations of current approaches, including glucocorticoids (short-lived benefit and associated muscle and bone loss), exon-skipping therapies (amenable to under ~40% of patients and “minimal benefit to date,” per his comments), gene therapy (benefit described as limited with eventual decline), and FDA-approved HDAC inhibitors (benefit through reduced fibrosis but with safety signals).
Seehra presented preclinical and early clinical observations suggesting rinvatercept could address multiple aspects of DMD, including muscle, bone, and fat parameters, and potentially mitigate negative consequences of glucocorticoids. He also said the company has observed effects relevant to combination strategies: enhanced expression of truncated dystrophin when used with exon-skipping phosphorodiamidate morpholino oligomers (PMOs), and increased expression of utrophin (a dystrophin homolog) in the absence of exon skipping.
On phase I results, Seehra said KER-065 was generally well tolerated in healthy volunteers across single ascending doses (1 mg/kg to 5 mg/kg) and multiple ascending doses (1.25 mg/kg and 2 mg/kg for three months). He reported no dose-limiting toxicities or serious adverse events. He noted one participant experienced a “very high” transient creatine kinase increase following vigorous exercise prior to arriving at the study site. Injection site reactions were observed, consistent with subcutaneous administration, and there was an asymptomatic and reversible increase in hemoglobin that he said could be managed with titration.
Seehra also highlighted phase I pharmacology signals described by the company, including increased lean mass and thigh muscle volume, improvements in bone mineral density and related bone biomarkers (including increased bone-specific alkaline phosphatase and reduced bone resorption markers), and changes in metabolic biomarkers (increased adiponectin and decreased leptin) alongside reductions in whole-body and visceral fat mass.
ALS rationale and planned regulatory interactions
Seehra said Keros is also developing rinvatercept in ALS, describing the disease as a progressive neurodegenerative condition marked by loss of motor neurons and neuromuscular junctions that leads to muscle weakness and paralysis. He cited an estimate of roughly 30,000 ALS patients living in the U.S. and said most cases are sporadic.
He outlined a therapeutic hypothesis focused on skeletal muscle: by promoting muscle regeneration, he said, rinvatercept could help compensate for loss of denervated muscle fibers by strengthening still-innervated fibers, potentially preserving function and quality of life. As supportive context, he referenced phase III data for apitegromab in spinal muscular atrophy (SMA) that he said showed functional gains, which Keros believes supports the concept of preserving muscle function in the setting of neural degeneration. He also cited preclinical data in an ALS mouse model, describing preservation of grip strength and muscle force compared with vehicle.
Looking ahead, Seehra said the company plans to engage with regulators in the second half of the year on the design of a phase II clinical trial evaluating rinvatercept in ALS. He also said Keros is working to start the DMD trial “in this quarter,” characterizing the year as one focused on execution and noting the company has not yet provided timing guidance for data.
Elritercept partnership with Takeda and timing expectations
Seehra reviewed Keros’ partnership with Takeda for elritercept, an investigational therapy being developed for anemia and thrombocytopenia in myelodysplastic syndrome (MDS) and myelofibrosis. He said Keros entered a global license agreement with Takeda in December 2024 (excluding mainland China, Hong Kong, and Macau), which included a $200 million upfront payment and eligibility for more than $1.1 billion in development and commercial milestones, plus tiered royalties ranging from low double digits to high teens. He added that as of January 2025, Takeda became fully responsible for clinical development, manufacturing, and commercialization in its territory.
On program timing, Seehra said Keros has not disclosed milestone details but noted development milestones are associated with the start of phase III trials. He said the phase III RENEW trial is currently running and that Takeda is committed to starting a frontline treatment trial in Takeda’s 2025 fiscal year, which ends March 31. He suggested that, based on the timelines of similar trials, enrollment could take roughly two to three years with a 12-month endpoint, which he said would imply commercialization “towards the end of this decade into early next decade.”
On the company’s financial position, Seehra said Keros reported $383 million in cash as of its third-quarter earnings report, and that the company expected runway into the first half of 2028, including anticipated expenses for both phase II trials and advancing preclinical assets into the clinic.
About Keros Therapeutics (NASDAQ:KROS)
Keros Therapeutics, Inc (NASDAQ: KROS) is a clinical-stage biopharmaceutical company dedicated to discovering and developing novel therapies for disorders of erythropoiesis and iron regulation. The company’s research centers on modulating the transforming growth factor-beta (TGF-β) superfamily to rebalance hematopoiesis and improve red blood cell production. By targeting key signaling pathways involved in anemia, Keros aims to provide new treatment options for patients with myelodysplastic syndromes, beta-thalassemia and other chronic anemias with significant unmet need.
The company’s lead product candidate, KER-050, is an engineered activin receptor ligand trap designed to restore effective erythropoiesis and reduce transfusion dependence in patients with anemia associated with myelodysplastic syndromes and primary myelofibrosis.
