Can Keros Therapeutics Rinvatercept Stand Out in the DMD Market?

Keros Therapeutics KROS 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.

Rinvatercept, 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.

Keros Therapeutics is advancing rinvatercept 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 August 2025, it announced that the FDA granted orphan drug designation for KER-065 for the treatment of DMD.

KROS plans to begin a phase II trial in patients with DMD in the first quarter of 2026. Last year, the company reported initial top-line results from a phase I study of KER-065 in healthy volunteers.

KROS is also developing for the treatment of amyotrophic lateral sclerosis (ALS). The company plans to engage with regulatory authorities in the second half of 2026 to discuss the design of a phase II study evaluating rinvatercept in patients with ALS.

Keros Therapeutics was previously advancing cibotercept for pulmonary arterial hypertension, but in August 2025, it discontinued the program and redirected resources toward rinvatercept, which currently appears to offer more compelling potential.

KROS’ Competition in the DMD Space

DMD is 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 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 four to five 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 temporarily paused U.S. shipments after reports of fatal liver injury but resumed distribution for ambulatory patients after the FDA indicated the pause could be lifted.

PTC Therapeutics, Inc. PTCT 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 with continued treatment allowed if patients 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.

KROS’ Price Performance, Valuation & Estimates

Shares of KROS have gained 2.9% in the past year compared with the industry’s rise of 15.9%.

Going by the price/book ratio, KROS is quite inexpensive. Its shares currently trade at 0.51x tangible book value, lower than the industry’s average of 3.76X and its mean of 3.91X.

The Zacks Consensus Estimate for 2026 loss per share is unchanged at $3.36 in the past seven days and is pegged at $4.34 for 2027.

KROS currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.




 

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This article originally published on Zacks Investment Research (zacks.com).

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