MBX Biosciences Completes Enrollment in Phase 2 Avail™ Trial of MBX 2109 (Canvuparatide) for Hypoparathyroidism
MBX Biosciences (Nasdaq: MBX) has completed enrollment for its Phase 2 Avail™ trial of MBX 2109 (canvuparatide), surpassing the initial target of 48 patients with 64 enrollees. The trial evaluates a long-acting parathyroid hormone peptide prodrug for treating chronic hypoparathyroidism (HP).
The study is a randomized, multicenter, 12-week, double-blind, placebo-controlled trial assessing safety, pharmacokinetics, and efficacy. The primary endpoint focuses on patients' ability to discontinue active vitamin D and reduce calcium supplements while maintaining normal serum albumin-adjusted calcium levels. Secondary endpoints include safety, tolerability, pharmacodynamic activity, and quality of life measures.
Topline results are expected in Q3 2025. The company believes canvuparatide could transform HP treatment by providing more consistent therapeutic effects and improved patient convenience.
MBX Biosciences (Nasdaq: MBX) ha completato l'arruolamento per il suo trial di Fase 2 Avail™ di MBX 2109 (canvuparatide), superando l'obiettivo iniziale di 48 pazienti con 64 arruolati. Lo studio valuta un pro-farmaco peptidico dell'ormone paratiroideo a lunga durata d'azione per il trattamento dell'ipoparatiroidismo cronico (HP).
Lo studio è un trial randomizzato, multicentrico, di 12 settimane, in doppio cieco e controllato con placebo che valuta la sicurezza, la farmacocinetica e l'efficacia. Il endpoint primario si concentra sulla capacità dei pazienti di interrompere l'assunzione di vitamina D attiva e ridurre i supplementi di calcio mantenendo livelli normali di calcio sierico aggiustato per l'albumina. Gli endpoint secondari includono la sicurezza, la tollerabilità, l'attività farmacodinamica e le misure della qualità della vita.
I risultati preliminari sono attesi nel Q3 2025. L'azienda crede che il canvuparatide potrebbe trasformare il trattamento dell'HP fornendo effetti terapeutici più consistenti e una maggiore comodità per i pazienti.
MBX Biosciences (Nasdaq: MBX) ha completado la inscripción para su ensayo de Fase 2 Avail™ de MBX 2109 (canvuparatide), superando el objetivo inicial de 48 pacientes con 64 inscritos. El ensayo evalúa un pro-fármaco peptídico de hormona paratiroidea de acción prolongada para el tratamiento de la hipoparatiroidismo crónico (HP).
El estudio es un ensayo aleatorizado, multicéntrico, de 12 semanas, doble ciego y controlado con placebo que evalúa la seguridad, la farmacocinética y la eficacia. El punto final primario se centra en la capacidad de los pacientes para interrumpir la vitamina D activa y reducir los suplementos de calcio mientras mantienen niveles normales de calcio sérico ajustados por albúmina. Los puntos finales secundarios incluyen seguridad, tolerabilidad, actividad farmacodinámica y medidas de calidad de vida.
Se esperan resultados preliminares en Q3 2025. La empresa cree que el canvuparatide podría transformar el tratamiento del HP al proporcionar efectos terapéuticos más consistentes y mejorar la comodidad del paciente.
MBX 바이오사이언스 (Nasdaq: MBX)는 MBX 2109 (칸부파라타이드)의 2상 Avail™ 시험에 대한 등록을 완료했으며, 초기 목표인 48명의 환자를 초과하여 64명이 등록되었습니다. 이 시험은 만성 저부갑상선증(HP) 치료를 위한 장기 작용 부갑상선 호르몬 펩타이드 프로드럭을 평가합니다.
이 연구는 안전성, 약리학적 작용 및 효능을 평가하는 무작위 다기관 12주 이중 맹검 위약 대조 시험입니다. 주요 평가 지표는 환자가 활성 비타민 D를 중단하고 알부민 조정 칼슘 수치를 정상으로 유지하면서 칼슘 보충제를 줄일 수 있는 능력에 중점을 둡니다. 2차 평가 지표에는 안전성, 내약성, 약리학적 활동 및 삶의 질 측정이 포함됩니다.
최종 결과는 2025년 3분기에 예상됩니다. 회사는 칸부파라타이드가 HP 치료를 혁신하여 보다 일관된 치료 효과와 환자의 편의성을 향상시킬 수 있다고 믿고 있습니다.
MBX Biosciences (Nasdaq: MBX) a terminé le recrutement pour son essai de Phase 2 Avail™ de MBX 2109 (canvuparatide), dépassant l'objectif initial de 48 patients avec 64 participants. L'essai évalue un pro-médicament de peptide d'hormone parathyroïdienne à action prolongée pour le traitement de l'hypoparathyroïdie chronique (HP).
L'étude est un essai randomisé, multicentrique, de 12 semaines, en double aveugle et contrôlé par placebo, évaluant la sécurité, la pharmacocinétique et l'efficacité. Le point final principal se concentre sur la capacité des patients à interrompre la vitamine D active et à réduire les suppléments de calcium tout en maintenant des niveaux normaux de calcium sérique ajustés à l'albumine. Les points finaux secondaires incluent la sécurité, la tolérabilité, l'activité pharmacodynamique et les mesures de qualité de vie.
Les résultats préliminaires sont attendus au T3 2025. L'entreprise estime que le canvuparatide pourrait transformer le traitement de l'HP en offrant des effets thérapeutiques plus cohérents et une meilleure commodité pour les patients.
MBX Biosciences (Nasdaq: MBX) hat die Rekrutierung für seine Phase-2-Studie Avail™ von MBX 2109 (Canvuparatide) abgeschlossen und damit das ursprüngliche Ziel von 48 Patienten mit 64 Rekrutierten übertroffen. Die Studie bewertet ein langwirksames Prodrug eines parathyroiden Hormons zur Behandlung der chronischen Hypoparathyreoidismus (HP).
Die Studie ist eine randomisierte, multizentrische, 12-wöchige, doppelblinde, placebokontrollierte Studie, die Sicherheit, Pharmakokinetik und Wirksamkeit bewertet. Der primäre Endpunkt konzentriert sich auf die Fähigkeit der Patienten, aktive Vitamin D-Präparate abzusetzen und die Calciumergänzungen zu reduzieren, während sie normale serumalbumin-adjustierte Calciumwerte aufrechterhalten. Sekundäre Endpunkte umfassen Sicherheit, Verträglichkeit, pharmakodynamische Aktivität und Lebensqualitätsmessungen.
Die vorläufigen Ergebnisse werden im Q3 2025 erwartet. Das Unternehmen glaubt, dass Canvuparatide die Behandlung von HP revolutionieren könnte, indem es konsistentere therapeutische Effekte und eine verbesserte Patientenbequemlichkeit bietet.
- Exceeded enrollment target by 33% (64 vs 48 patients)
- On track for topline results in Q3 2025
- Potential to transform treatment landscape with long-acting therapy
- Results not expected until Q3 2025
- Still in early Phase 2 stage, far from potential commercialization
Insights
MBX Biosciences' announcement marks a significant clinical milestone with completed enrollment for their Phase 2 Avail™ trial of canvuparatide in hypoparathyroidism. Exceeding the target enrollment (64 vs. 48 patients) suggests strong investigator interest and potentially enhances the statistical power of the upcoming results.
The trial's primary endpoint—reduction in medication burden while maintaining normal calcium levels—addresses a key clinical need for hypoparathyroidism patients who currently require intensive daily management with calcium and vitamin D supplements. Secondary endpoints evaluating quality of life outcomes further align with patient-centric development strategies increasingly valued in rare disease markets.
This progression represents important pipeline validation for MBX, a clinical-stage company whose market valuation depends heavily on successful advancement of key assets. Canvuparatide's long-acting mechanism could potentially differentiate it from existing therapies that require frequent administration. With topline results expected in Q3 2025, investors now have a clear catalyst timeline to monitor for this program.
The development of canvuparatide addresses a substantial unmet need in hypoparathyroidism management. Current standard of care—high-dose calcium and active vitamin D supplements—treats symptoms without addressing the underlying hormone deficiency, often leading to suboptimal outcomes and quality of life impacts for patients.
MBX's approach with a long-acting PTH peptide prodrug is mechanistically sound, aiming to more closely mimic natural hormone patterns. The trial's primary endpoint (discontinuation of active vitamin D with reduced calcium supplementation while maintaining normal calcium levels) represents a clinically meaningful goal that would significantly improve disease management if achieved.
The inclusion of pharmacodynamic markers (urine calcium, serum phosphorus, vitamin D metabolites, and bone biomarkers) will provide important insights into how comprehensively canvuparatide addresses the complex mineral metabolism disruptions in hypoparathyroidism. Additionally, the focus on patient-reported outcomes acknowledges the substantial burden of this chronic condition on daily functioning and well-being—aspects often overlooked in purely biochemical assessments.
AvailTM trial exceeded enrollment target; topline results expected in Q3 2025
CARMEL, Ind., March 03, 2025 (GLOBE NEWSWIRE) -- MBX Biosciences, Inc. (Nasdaq: MBX), a clinical-stage biopharmaceutical company focused on the discovery and development of novel precision peptide therapies for the treatment of endocrine and metabolic disorders, today announced the completion of enrollment in its Phase 2 Avail™ trial of MBX 2109 (canvuparatide), the Company’s parathyroid hormone (PTH) peptide prodrug. Canvuparatide is designed to be long-acting and is in development for the treatment of chronic hypoparathyroidism (HP).
“Completion of enrollment in our Phase 2 trial in HP marks a significant milestone for MBX and brings us closer to our goal of delivering a long-acting PTH therapy designed to treat the underlying disease pathophysiology,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “We believe canvuparatide could transform the HP treatment landscape, potentially providing a more consistent therapeutic effect, while offering improved patient convenience and minimizing symptoms associated with large fluctuations in calcium. We look forward to reporting top-line data from the Phase 2 Avail™ trial in the third quarter of 2025.”
The Phase 2 Avail™ trial (NCT06465108) is a randomized, multicenter, 12-week, double-blind, placebo-controlled study evaluating the safety, pharmacokinetics, and efficacy of canvuparatide in adults with HP. The study enrolled 64 patients, exceeding the original target of 48. The primary endpoint of the Phase 2 clinical trial is the proportion of patients who can discontinue active vitamin D and reduce calcium supplements to less than or equal to 600 mg per day after 12 weeks of treatment while maintaining normal serum albumin-adjusted calcium levels (8.2-10.6 mg/dL). Secondary endpoints include safety and tolerability of canvuparatide and characterization of its pharmacokinetics and pharmacodynamic activity (including urine calcium, serum phosphorus, 1,25 dihydroxyvitamin D and bone biomarkers) and the impact on quality of life using patient-reported outcome tools.
Top-line results from the Phase 2 Avail trial are expected in the third quarter of 2025. More information on the trial can be found at www.clinicaltrials.gov, identifier NCT06465108.
About Hypoparathyroidism (HP)
HP is a rare endocrine disease caused by a deficiency of PTH released by the parathyroid glands that results in decreased calcium levels in the blood leading to hypocalcemia. Hypocalcemia can result in a variety of acute symptoms, such as muscle cramping or spasm, tingling, and neurological symptoms such as depression, confusion and cognitive impairment. More serious complications can occur, including seizures and cardiac arrhythmia. As a result, HP can interfere with daily activities, negatively impacting the quality of life for patients and we estimate that HP affects approximately 120,000 people in the United States and more than 250,000 in the United States and Europe. The most common cause for HP, in approximately
About Canvuparatide (MBX 2109)
Canvuparatide is a parathyroid hormone peptide prodrug that is designed as a potential long-acting hormone replacement therapy for the treatment of HP. Leveraging the company’s proprietary Precision Endocrine Peptide™ (PEP™) platform technology, canvuparatide was designed to provide convenient, once-weekly administration and a continuous, infusion-like PTH exposure with lower daily peak-to-trough ratios than observed with daily PTH dosing regimens. Canvuparatide received orphan drug designation from the U.S. Food and Drug Administration for the treatment of HP.
About MBX Biosciences
MBX Biosciences is a biopharmaceutical company focused on the discovery and development of novel precision peptide therapies based on its proprietary PEP™ platform, for the treatment of endocrine and metabolic disorders. The Company is advancing a pipeline of novel candidates for endocrine and metabolic disorders with clinically validated targets, established endpoints for regulatory approval, significant unmet medical needs and large potential market opportunities. The Company’s pipeline includes its lead product candidate canvuparatide, in Phase 2 development for the treatment of chronic hypoparathyroidism (HP); MBX 1416, in Phase 1 development for the treatment of post-bariatric hypoglycemia (PBH); and an obesity portfolio that includes MBX 4291, as well as multiple discovery and pre-clinical candidates in development for the treatment of obesity. The Company is based in Carmel, Indiana. To learn more, please visit the Company website at www.mbxbio.com and follow it on LinkedIn.
About MBX’s Proprietary Precision Endocrine Peptide (PEP™) Platform
MBX was founded by global leaders with a transformative approach to peptide drug design and development. Leveraging this expertise, the Company designed its proprietary Precision Endocrine Peptide™ (PEP™) platform to overcome the key limitations of unmodified and modified peptide therapies and to improve clinical outcomes and simplify disease management for patients. PEPs are selectively engineered to have optimized pharmaceutical properties, including extended time-action profiles and consistent drug concentrations with low peak-to-trough concentration ratios, consistent exposure to target tissues, and less frequent dosing.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements include, but are not limited to, express or implied statements regarding: MBX Biosciences’ expectations regarding the Phase 2 Avail™ trial of MBX 2109 (canvuparatide), including the timing of top-line results and statements relating to canvuparatide’s clinical profile, including the potential to be a once-weekly PTH prodrug for the treatment of HP and the Company’s belief that canvuparatide could transform the HP treatment landscape, potentially providing a more consistent therapeutic effect, while offering improved patient convenience and minimizing symptoms associated with large fluctuations in calcium.
Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could negatively affect MBX Biosciences’ business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to the Company’s research and development activities; uncertainties relating to preclinical and clinical development activities; the risk that preliminary results may not be indicative of later results; the Company’s dependence on third parties to conduct clinical trials; MBX Biosciences’ ability to attract, integrate and retain key personnel; risks related to regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; as well as other risks described in “Risk Factors,” in MBX Biosciences’ Registration Statement on Form S-1 filed with the Securities and Exchange Commission (SEC), most recent Quarterly Report on Form 10-Q, as well as subsequent filings with the SEC. MBX Biosciences expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and claims the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
MBX uses and intends to continue to use its Investor Relations website as a means of disclosing material nonpublic information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Company’s Investor Relations website, in addition to following the Company’s press releases, SEC filings, public conference calls, presentations, and webcasts.
Media Contact:
Katie Beach Oltsik
Inizio Evoke Comms
katie.beach@inizioevoke.com
(937) 232-4889
Investor Contact:
Jim DeNike
MBX Biosciences
jdenike@mbxbio.com
FAQ
What are the primary endpoints of MBX's Phase 2 Avail trial for canvuparatide?
How many patients were enrolled in MBX Biosciences' Phase 2 Avail trial?
When will MBX Biosciences release the topline results for the Phase 2 canvuparatide trial?
What potential benefits could canvuparatide offer for hypoparathyroidism patients?
