Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers
Keros Therapeutics (NASDAQ: KROS) has announced positive initial topline results from its Phase 1 clinical trial of KER-065 in healthy volunteers. The trial successfully met its key objectives for safety, tolerability, and pharmacological measures through the multiple ascending dose treatment period.
Key findings as of February 6, 2025, showed that KER-065 was generally well-tolerated with no serious adverse events or dose-limiting toxicities. The drug demonstrated evidence of activin inhibition through:
- Increases in bone formation biomarkers and decreases in bone resorption markers
- Positive changes in fat mobilization biomarkers
- Improvements in body composition, including increased bone mineral density and muscle mass
Based on these results, Keros plans to engage with regulatory authorities in Q3 2025 and aims to initiate a Phase 2 clinical trial for Duchenne muscular dystrophy (DMD) patients in Q1 2026.
Keros Therapeutics (NASDAQ: KROS) ha annunciato risultati iniziali positivi dal suo studio clinico di Fase 1 su KER-065 in volontari sani. Lo studio ha raggiunto con successo i suoi obiettivi chiave per sicurezza, tollerabilità e misure farmacologiche durante il periodo di trattamento con dosi crescenti multiple.
I risultati chiave al 6 febbraio 2025 hanno mostrato che KER-065 è stato generalmente ben tollerato, senza eventi avversi gravi o tossicità limitanti la dose. Il farmaco ha dimostrato evidenza di inibizione dell'activina attraverso:
- Aumenti nei biomarcatori di formazione ossea e diminuzioni nei marcatori di riassorbimento osseo
- Cambiamenti positivi nei biomarcatori di mobilizzazione dei grassi
- Miglioramenti nella composizione corporea, inclusi l'aumento della densità minerale ossea e della massa muscolare
Basandosi su questi risultati, Keros prevede di interagire con le autorità regolatorie nel terzo trimestre del 2025 e mira a iniziare un studio clinico di Fase 2 per pazienti affetti da distrofia muscolare di Duchenne (DMD) nel primo trimestre del 2026.
Keros Therapeutics (NASDAQ: KROS) ha anunciado resultados iniciales positivos de su ensayo clínico de Fase 1 de KER-065 en voluntarios sanos. El ensayo cumplió con éxito sus objetivos clave en cuanto a seguridad, tolerabilidad y medidas farmacológicas durante el período de tratamiento con dosis ascendentes múltiples.
Los hallazgos clave a partir del 6 de febrero de 2025 mostraron que KER-065 fue generalmente bien tolerado, sin eventos adversos graves ni toxicidades limitantes de dosis. El fármaco mostró evidencia de inhibición de activina a través de:
- Aumentos en biomarcadores de formación ósea y disminuciones en biomarcadores de reabsorción ósea
- Cambios positivos en biomarcadores de movilización de grasas
- Mejoras en la composición corporal, incluyendo aumento de la densidad mineral ósea y masa muscular
Con base en estos resultados, Keros planea interactuar con las autoridades regulatorias en el tercer trimestre de 2025 y tiene como objetivo iniciar un ensayo clínico de Fase 2 para pacientes con distrofia muscular de Duchenne (DMD) en el primer trimestre de 2026.
케로스 테라퓨틱스 (NASDAQ: KROS)는 건강한 자원봉사자를 대상으로 한 KER-065의 1상 임상 시험에서 긍정적인 초기 결과를 발표했습니다. 이 시험은 여러 단계의 용량 증가 치료 기간 동안 안전성, 내약성 및 약리적 측정에 대한 주요 목표를 성공적으로 달성했습니다.
2025년 2월 6일 기준 주요 발견에 따르면 KER-065는 일반적으로 잘 견디며 심각한 부작용이나 용량 제한 독성이 없었습니다. 이 약물은 다음을 통해 액티빈 억제의 증거를 보여주었습니다:
- 골 형성 바이오마커의 증가 및 골 흡수 바이오마커의 감소
- 지방 동원 바이오마커의 긍정적인 변화
- 골밀도 및 근육량 증가를 포함한 신체 구성의 개선
이 결과를 바탕으로 케로스는 2025년 3분기에 규제 당국과 소통할 계획이며, 2026년 1분기에 뒤셴 근육병(DMD) 환자를 위한 2상 임상 시험을 시작하는 것을 목표로 하고 있습니다.
Keros Therapeutics (NASDAQ: KROS) a annoncé des résultats préliminaires positifs de son essai clinique de Phase 1 sur KER-065 chez des volontaires sains. L'essai a réussi à atteindre ses objectifs clés en matière de sécurité, de tolérance et de mesures pharmacologiques pendant la période de traitement à doses croissantes multiples.
Les résultats clés au 6 février 2025 ont montré que KER-065 était généralement bien toléré, sans événements indésirables graves ni toxicités limitantes de dose. Le médicament a montré des preuves d'inhibition de l'activine par le biais de :
- Augmentations des biomarqueurs de formation osseuse et diminutions des biomarqueurs de résorption osseuse
- Changements positifs dans les biomarqueurs de mobilisation des graisses
- Améliorations de la composition corporelle, y compris une augmentation de la densité minérale osseuse et de la masse musculaire
Sur la base de ces résultats, Keros prévoit d'engager un dialogue avec les autorités réglementaires au troisième trimestre 2025 et vise à initier un essai clinique de Phase 2 pour les patients atteints de dystrophie musculaire de Duchenne (DMD) au premier trimestre 2026.
Keros Therapeutics (NASDAQ: KROS) hat positive erste Ergebnisse aus seiner Phase-1-Studie zu KER-065 bei gesunden Probanden bekannt gegeben. Die Studie hat erfolgreich ihre Hauptziele in Bezug auf Sicherheit, Verträglichkeit und pharmakologische Messungen während der Phase der mehrfachen aufsteigenden Dosis erreicht.
Wichtige Ergebnisse zum 6. Februar 2025 zeigten, dass KER-065 im Allgemeinen gut vertragen wurde, ohne schwerwiegende Nebenwirkungen oder dosislimitierende Toxizitäten. Das Medikament zeigte Hinweise auf eine Hemmung von Activin durch:
- Erhöhungen von Biomarkern für die Knochenbildung und Abnahmen von Biomarkern für den Knochenabbau
- Positive Veränderungen bei Biomarkern für die Fettmobilisierung
- Verbesserungen der Körperzusammensetzung, einschließlich einer erhöhten Knochendichte und Muskelmasse
Basierend auf diesen Ergebnissen plant Keros, im dritten Quartal 2025 mit den Regulierungsbehörden in Kontakt zu treten und beabsichtigt, im ersten Quartal 2026 eine Phase-2-Studie für Patienten mit Duchenne-Muskeldystrophie (DMD) zu beginnen.
- Phase 1 trial met all key safety and efficacy objectives
- No serious adverse events or dose-limiting toxicities reported
- Demonstrated positive biomarker changes indicating drug effectiveness
- Clear development timeline with Phase 2 trial planned for Q1 2026
- Extended timeline to Phase 2 initiation (Q1 2026)
- Regulatory approval required before advancing to Phase 2
- Early-stage development with no efficacy data in actual DMD patients yet
Insights
The topline results from Keros Therapeutics' Phase 1 trial for KER-065 represent a promising initial clinical milestone for their DMD program. The trial demonstrated a clean safety profile with no serious adverse events or dose-limiting toxicities - the primary goal of any Phase 1 study.
More noteworthy is the evidence of target engagement across multiple tissues. The biomarker data showing increases in bone formation markers (BSAP) with concurrent decreases in bone resorption markers (CTX) indicates the drug is having the desired biological effects on bone metabolism. Similarly, the adiponectin increases coupled with leptin decreases suggest metabolic impact through the intended pathway.
The body composition changes are particularly compelling for DMD applications - increases in bone mineral density and muscle mass align perfectly with addressing the progressive muscle wasting and bone fragility that characterize Duchenne. These pharmacodynamic signals in healthy volunteers provide a rational basis for therapeutic potential in DMD patients.
This aligns with the scientific understanding of activin's role in muscle pathology. Activin inhibition has shown promise in addressing both the primary muscle degeneration and secondary complications of DMD in preclinical models. The biomarker and composition data suggest KER-065 successfully inhibits this pathway in humans.
The planned advancement to Phase 2 represents standard clinical development progression following these positive findings. However, investors should recognize that while these results are encouraging, Phase 2 will provide the first efficacy data in actual DMD patients - the true test of therapeutic potential.
Keros' Phase 1 results for KER-065 represent a positive development for their clinical pipeline, potentially expanding their addressable market into neuromuscular disorders. The clean safety profile and pharmacodynamic signals provide the company with multiple strategic options for their development program.
DMD represents a significant commercial opportunity despite being a rare disease. Current therapies have substantial limitations - many are mutation-specific and address only certain aspects of the disease. KER-065's mechanism targeting activin signaling could potentially offer broader applicability across DMD patient populations regardless of genetic mutation.
The company's statement about a "strong financial foundation" suggests adequate runway to advance this program alongside their other pipeline assets. This reduces near-term financing risk that often impacts clinical-stage biotech valuations. Their planned regulatory engagement in Q3 2025 followed by potential Phase 2 initiation in Q1 2026 provides clear development milestones for investors to monitor.
From a valuation perspective, successful Phase 1 completion de-risks the program slightly, though the most significant value inflection points remain ahead with efficacy data in DMD patients. The therapeutic potential beyond DMD into "other neuromuscular indications" hints at pipeline expansion opportunities that could enhance the long-term value proposition.
Investors should note that while these results support continued development, KER-065 remains years from potential commercialization. The actual commercial value will depend on efficacy data, regulatory approval, and positioning against both existing therapies and others in development for DMD.
- Trial achieved key objectives for safety, tolerability, pharmacokinetics and pharmacodynamics
- Clinical data from this trial, together with preclinical data, support the therapeutic potential of KER-065 for broad impact in Duchenne muscular dystrophy (“DMD”) and other neuromuscular indications
- Keros plans on advancing KER-065 into a Phase 2 clinical trial in DMD, subject to positive regulatory interaction
- Keros to host a conference call and webcast today, March 31, 2025, at 8:00 a.m. ET
LEXINGTON, Mass., March 31, 2025 (GLOBE NEWSWIRE) -- Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company 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 (“TGF-ß”) family of proteins, today announced initial topline results from the Phase 1 clinical trial of KER-065 in healthy volunteers. Topline results from this ongoing trial are through the multiple ascending dose (“MAD”) treatment period (Day 85).
“We are pleased to report topline results that met the key objectives of the Phase 1 clinical trial and provided important insights to inform the development of KER-065 for patients with DMD,” said Jasbir S. Seehra, Ph.D., Chair and Chief Executive Officer. “Considering the limitations of currently available therapies, the need for additional treatments in DMD remains high, and KER-065 has the potential to address multiple aspects of the disease, including across important tissues and underlying genetic deficiencies. Our strong financial foundation enables us to continue advancing our promising pipeline of novel therapeutics, and we look forward to engaging with regulators towards the aim of moving KER-065 to a Phase 2 clinical trial in the first quarter of 2026.”
Key findings of this trial as of a February 6, 2025 data cut-off date include the following:
- KER-065 was generally well-tolerated, with no major safety signals observed to date
- No serious adverse events or dose-limiting toxicities reported
- Evidence for activin inhibition across tissues of interest, as KER-065 elicited:
- Increases in bone specific alkaline phosphate (BSAP), a biomarker of bone formation, and decreases in C-Terminal Telopeptide (CTX), a biomarker of bone resorption
- Increases in adiponectin, a biomarker of fat mobilization, and decreases in leptin, a biomarker of fat mass
- Changes in body composition, as demonstrated by increases in bone mineral density and muscle mass and decreases in fat mass, which in totality were consistent with activin inhibition
“We observed evidence of activin inhibition based on multiple biomarkers and body composition data. These data, coupled with preclinical and mechanistic insights on the pivotal role of the activin pathway in neuromuscular pathobiology, demonstrate the exciting therapeutic potential of KER-065 in DMD and other neuromuscular disorders,” said Yung H. Chyung, M.D., Chief Medical Officer.
Keros plans on engaging with regulatory authorities, starting in the third quarter of 2025. Subject to the outcome of these regulatory interactions, Keros expects to initiate a Phase 2 clinical trial of KER-065 in patients with DMD in the first quarter of 2026.
Conference Call and Webcast Information
Keros will host a conference call and webcast today, March 31, 2025, at 8:00 a.m. Eastern time. The conference call will be webcast live at: https://event.webcasts.com/starthere.jsp?ei=1713593&tp_key=5723864d86. The live teleconference may be accessed by dialing (877) 407-0309 (domestic) or (201) 389-0853 (international). An archived version of the call will be available in the investors section of the Keros website at http://ir.kerostx.com for 90 days following the conclusion of the call.
About the KER-065 Phase 1 Clinical Trial
The KER-065 Phase 1 clinical trial is a randomized, double-blind, placebo-controlled, two-part dose escalation (single ascending dose and MAD) trial in healthy volunteers. The primary objectives of this trial were to assess safety, tolerability and pharmacokinetics of KER-065. Exploratory endpoints include assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis.
About KER-065
KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from activin receptor type IIA and activin receptor type IIB that is fused to the portion of the human antibody known as the Fc domain. KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors, to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength. We are developing KER-065 for the treatment of neuromuscular diseases, with an initial focus on DMD.
About Duchenne Muscular Dystrophy (DMD)
DMD is the most common form of muscular dystrophy and results in muscle degeneration and premature death. DMD results from the lack of functional dystrophin protein that helps promote myofiber stability, caused by a gene mutation. The lack of dystrophin, an important structural component of muscle cells, causes muscle cells to have increased susceptibility to damage and to progressively die. Additionally, the absence of dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and the replacement of muscle with fibrotic and fatty tissue. The replacement of muscle fibers with fatty and fibrotic tissue leads to progressive loss of muscle strength and function leading to immobility and respiratory and cardiac complications. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. The National Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
About Keros Therapeutics, Inc.
Keros is a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the TGF-ß family of proteins. Keros is a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including blood, bone, skeletal muscle, adipose and heart tissue. By leveraging this understanding, Keros has discovered and is developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients. One of Keros’ product candidates, cibotercept (KER-012), is being developed for the treatment of pulmonary arterial hypertension and for the treatment of cardiovascular disorders. Keros’ second product candidate, KER-065, is being developed for the treatment of neuromuscular diseases. Keros’ most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndrome and in patients with myelofibrosis.
Cautionary Note Regarding Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Words such as “anticipates,” “believes,” “continue,” “expects,” “enable,” “potential” and “will” or similar expressions are intended to identify forward-looking statements. Examples of these forward-looking statements include statements concerning: Keros’ expectations regarding its strategy and timing of its Phase 2 clinical trial for KER-065, including its plans to engage with regulatory authorities; Keros’ ability to advance its pipeline of novel therapeutics; and the potential therapeutic benefits of KER-065. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others: Keros’ limited operating history and historical losses; Keros’ ability to raise additional funding to complete the development and any commercialization of its product candidates; Keros’ dependence on the success of its product candidates, cibotercept, KER-065 and elritercept; that Keros may be delayed in initiating, enrolling or completing any clinical trials; competition from third parties that are developing products for similar uses; Keros’ ability to obtain, maintain and protect its intellectual property; and Keros’ dependence on third parties in connection with manufacturing, clinical trials and preclinical studies.
These and other risks are described more fully in Keros’ filings with the Securities and Exchange Commission (“SEC”), including the “Risk Factors” section of the Company’s Annual Report on Form 10-K, filed with the SEC on February 26, 2025, and its other documents subsequently filed with or furnished to the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Except to the extent required by law, Keros undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Investor Contact:
Justin Frantz
jfrantz@kerostx.com
617-221-6042
FAQ
What were the key results of KROS's Phase 1 trial for KER-065?
When will KROS begin Phase 2 trials for KER-065 in DMD patients?
What biomarkers were affected by KER-065 in the Phase 1 trial?
How does KER-065 potentially benefit Duchenne muscular dystrophy patients?
