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Is KROS' KER-065 the Next Breakthrough in DMD Space?
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Key Takeaways
Keros Therapeutics is developing KER-065, an inhibitor for treating Duchenne muscular dystrophy.
KROS reported phase I top-line data in March 2025 and received FDA orphan drug designation in August 2025.
Keros plans to start a phase II DMD trial in Q1 2026 after halting cibotercept to focus on KER-065.
Keros Therapeutics (KROS - Free Report) is focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta, or TGF-ß, family of proteins.
KER-065, KROS’ lead pipeline candidate, is engineered to selectively bind and inhibit TGF-β ligands, including myostatin (GDF8) and activin A, which are key negative regulators of muscle and bone mass and strength. By blocking these pathways, the company believes KER-065 has the potential to promote skeletal muscle regeneration, increase muscle size and strength, reduce body fat and muscle fibrosis, and enhance overall bone strength.
The company is advancing KER-065 for the treatment of neuromuscular disorders, initially targeting Duchenne muscular dystrophy (DMD).
According to Keros, glucocorticoids — the current standard of care for DMD — are associated with significant long-term side effects, including muscle catabolism, increased fat accumulation and accelerated bone loss.
In March 2025, Keros reported initial top-line results from a phase I study of KER-065 in healthy volunteers. In August 2025, it announced that the FDA granted orphan drug designation for KER-065 for the treatment of DMD.
The company plans to begin a phase II trial in patients with DMD in the first quarter of 2026 and explore additional indications where KER-065’s mechanism of action is believed to have a strong potential for clinical success.
Keros also plans to pursue additional indications where KER-065’s mechanism of action has a high likelihood of clinical success.
The company was previously advancing cibotercept for pulmonary arterial hypertension, but in August 2025, it discontinued the program and redirected resources toward its lead asset, KER-065, which currently appears to offer more compelling potential.
KROS’ Competition in the DMD Space
DMD is itself a particularly competitive and complex therapeutic area. At present, many DMD patients are treated with corticosteroids to manage the inflammatory component of the disease.
Sarepta Therapeutics (SRPT - Free Report) is a formidable player in this space with a strong DMD franchise that includes exon-skipping therapies, such as Exondys 51, Vyondys 53 and Amondys 45. In June 2023, Sarepta obtained accelerated approval from the FDA for Elevidys, an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD with a confirmed mutation in the DMD gene.
Thereafter, in June 2024, the FDA granted full approval to Elevidys for the treatment of ambulatory individuals aged four years and older, and accelerated approval for the treatment of non-ambulatory individuals aged four years and older.
In July 2025, Sarepta announced its decision to voluntarily and temporarily pause all shipments of Elevidys for patients with DMD in the United States. Nonetheless, a month later, Sarepta announced that the FDA notified the company that it may lift its voluntary pause on shipments of Elevidys for ambulatory patients with DMD, and Sarepta resumed shipping to such patients immediately.
PTC Therapeutics, Inc., (PTCT - Free Report) currently markets Emflaza (deflazacort) for the treatment of DMD.
Emflaza is approved in the United States for the treatment of DMD in patients two years and older.
PTCT’s other DMD drug, Translarna, has marketing authorization in Russia for the treatment of nonsense mutation DMD (nmDMD) in patients aged two years and older, and in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become nonambulatory, as well as in various other countries.
PTC Therapeutics held a conditional marketing authorization for Translarna in the European Economic Area (EEA), subject to annual EMA review. Despite submitting additional clinical data and requests to convert to full approval and renew the authorization, the EMA’s CHMP issued repeated negative opinions. In March 2025, the European Commission adopted the CHMP’s decision not to renew Translarna’s authorization for nmDMD.
Our Take on KROS
Keros has made notable progress with its lead DMD candidate. Any positive clinical or regulatory updates related to KER-065 could serve as a meaningful catalyst and provide a significant upside driver for KROS shares.
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Is KROS' KER-065 the Next Breakthrough in DMD Space?
Key Takeaways
Keros Therapeutics (KROS - Free Report) is focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta, or TGF-ß, family of proteins.
KER-065, KROS’ lead pipeline candidate, is engineered to selectively bind and inhibit TGF-β ligands, including myostatin (GDF8) and activin A, which are key negative regulators of muscle and bone mass and strength. By blocking these pathways, the company believes KER-065 has the potential to promote skeletal muscle regeneration, increase muscle size and strength, reduce body fat and muscle fibrosis, and enhance overall bone strength.
The company is advancing KER-065 for the treatment of neuromuscular disorders, initially targeting Duchenne muscular dystrophy (DMD).
According to Keros, glucocorticoids — the current standard of care for DMD — are associated with significant long-term side effects, including muscle catabolism, increased fat accumulation and accelerated bone loss.
In March 2025, Keros reported initial top-line results from a phase I study of KER-065 in healthy volunteers. In August 2025, it announced that the FDA granted orphan drug designation for KER-065 for the treatment of DMD.
The company plans to begin a phase II trial in patients with DMD in the first quarter of 2026 and explore additional indications where KER-065’s mechanism of action is believed to have a strong potential for clinical success.
Keros also plans to pursue additional indications where KER-065’s mechanism of action has a high likelihood of clinical success.
The company was previously advancing cibotercept for pulmonary arterial hypertension, but in August 2025, it discontinued the program and redirected resources toward its lead asset, KER-065, which currently appears to offer more compelling potential.
KROS’ Competition in the DMD Space
DMD is itself a particularly competitive and complex therapeutic area. At present, many DMD patients are treated with corticosteroids to manage the inflammatory component of the disease.
Sarepta Therapeutics (SRPT - Free Report) is a formidable player in this space with a strong DMD franchise that includes exon-skipping therapies, such as Exondys 51, Vyondys 53 and Amondys 45. In June 2023, Sarepta obtained accelerated approval from the FDA for Elevidys, an adeno-associated virus-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD with a confirmed mutation in the DMD gene.
Thereafter, in June 2024, the FDA granted full approval to Elevidys for the treatment of ambulatory individuals aged four years and older, and accelerated approval for the treatment of non-ambulatory individuals aged four years and older.
In July 2025, Sarepta announced its decision to voluntarily and temporarily pause all shipments of Elevidys for patients with DMD in the United States. Nonetheless, a month later, Sarepta announced that the FDA notified the company that it may lift its voluntary pause on shipments of Elevidys for ambulatory patients with DMD, and Sarepta resumed shipping to such patients immediately.
PTC Therapeutics, Inc., (PTCT - Free Report) currently markets Emflaza (deflazacort) for the treatment of DMD.
Emflaza is approved in the United States for the treatment of DMD in patients two years and older.
PTCT’s other DMD drug, Translarna, has marketing authorization in Russia for the treatment of nonsense mutation DMD (nmDMD) in patients aged two years and older, and in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become nonambulatory, as well as in various other countries.
PTC Therapeutics held a conditional marketing authorization for Translarna in the European Economic Area (EEA), subject to annual EMA review. Despite submitting additional clinical data and requests to convert to full approval and renew the authorization, the EMA’s CHMP issued repeated negative opinions. In March 2025, the European Commission adopted the CHMP’s decision not to renew Translarna’s authorization for nmDMD.
Our Take on KROS
Keros has made notable progress with its lead DMD candidate. Any positive clinical or regulatory updates related to KER-065 could serve as a meaningful catalyst and provide a significant upside driver for KROS shares.