MBX Biosciences Announces Once-Weekly Canvuparatide Achieved Primary Endpoint in Phase 2 Trial with 63% Responder Rate at 12 Weeks; 79% Responder Rate at 6 Months in Open-Label Extension
MBX Biosciences (Nasdaq: MBX) announced significant positive results from its Phase 2 Avail™ trial of once-weekly canvuparatide for chronic hypoparathyroidism (HP). The trial achieved its primary endpoint with a 63% responder rate at 12 weeks compared to 31% for placebo (p=0.042), and demonstrated a 79% responder rate at 6 months in the open-label extension (OLE).
Key highlights include 100% patient retention through the 12-week study, with 94% entering the OLE. The drug showed positive effects on bone and kidney biomarkers, with a 48% reduction in urine calcium in treated patients. The treatment was well-tolerated with no serious adverse events or discontinuations. Based on these results, MBX plans to initiate a Phase 3 trial in 2026.
MBX Biosciences (Nasdaq: MBX) ha annunciato risultati positivi significativi dal suo studio di fase 2 Avail™ su canvuparatide somministrato una volta alla settimana per l'ipoparatiroidismo cronico (HP). Lo studio ha raggiunto l'obiettivo primario con un 63% tasso di risposta a 12 settimane rispetto al 31% del placebo (p=0,042), e ha mostrato un 79% tasso di risposta dopo 6 mesi nell'estensione in aperto (OLE).
Tra i punti chiave vi è 100% di mantenimento dei pazienti fino alle 12 settimane dello studio, con il 94% che è entrato nell'OLE. Il farmaco ha mostrato effetti positivi sui biomarcatori ossei e renali, con una riduzione del 48% del calcio nelle urine nei pazienti trattati. Il trattamento è stato ben tollerato, senza eventi avversi gravi né interruzioni. In base a questi risultati, MBX prevede di avviare un trial di fase 3 nel 2026.
MBX Biosciences (Nasdaq: MBX) anunció resultados positivos significativos de su ensayo de fase 2 Avail™ de canvuparatide administrado una vez a la semana para el hipoparatiroidismo crónico (HP). El ensayo alcanzó su objetivo primario con una tasa de respuesta del 63% a las 12 semanas frente al 31% de placebo (p=0.042), y demostró una tasa de respuesta del 79% a los 6 meses en la extensión abierta (OLE).
Entre los puntos clave se incluye retención del 100% de los pacientes hasta las 12 semanas del estudio, con un 94% que ingresó en la OLE. El fármaco mostró efectos positivos en biomarcadores óseos y renales, con una reducción del 48% de la calcemia urinaria en pacientes tratados. El tratamiento fue bien tolerado sin eventos adversos graves ni descontinuaciones. Con base en estos resultados, MBX planea iniciar un ensayo de fase 3 en 2026.
MBX Biosciences (Nasdaq: MBX)가 만성 부갑상샘 기능저하증(HP)을 위한 주 1회 투여 카누파라타이드(canvuparatide)의 2상 Avail™ 임상에서 상당한 긍정적 결과를 발표했습니다. 이 연구는 12주 차에 63% 응답률을 31%의 위약 대비 primary 엔드포인트를 달성했고, 오픈 라벨 확장(OLE)에서 6개월 시점 79% 응답률을 보여주었습니다.
주요 하이라이트로는 연구 12주 차까지 환자 유지율 100%이며, 그 중 94%가 OLE에 진입했습니다. 약물은 뼈 및 신장 바이오마커에 긍정적 영향을 보였고, 치료군에서 소변 칼슘 48% 감소를 나타냈습니다. 치료는 중대한 이상반응 없이 잘 견뎌졌으며 중단도 없었습니다. 이러한 결과를 바탕으로 MBX는 2026년 3상 시험을 시작할 계획입니다.
MBX Biosciences (Nasdaq: MBX) a annoncé des résultats positifs et significatifs de son essai de phase 2 Avail™ sur le canvuparatide administré une fois par semaine pour l’hypoparathyroïdie chronique (HP). L’essai a atteint son objectif primaire avec un taux de répondeurs de 63% à 12 semaines contre 31% pour le placebo (p=0,042), et a démontré un taux de répondeurs de 79% à 6 mois dans l’extension en open-label (OLE).
Les points clés incluent une Rétention de 100% des patients jusqu’à 12 semaines et 94% entrant dans l’OLE. Le médicament a montré des effets positifs sur les biomarqueurs osseux et rénaux, avec une réduction de 48% du calcium urinaire chez les patients traités. Le traitement a été bien toléré sans événements indésirables graves ni abandons. Sur la base de ces résultats, MBX prévoit de lancer un essai de phase 3 en 2026.
MBX Biosciences (Nasdaq: MBX) kündigte signifikante positive Ergebnisse aus ihrer Phase-2-Avail™-Studie mit einmal wöchentlich verabreichtem Canvuparatide zur chronischen Hypoparathyreoidismus (HP) an. Die Studie erreichte den primären Endpunkt mit einer 63%-Respondenzrate nach 12 Wochen im Vergleich zu 31% Placebo (p=0,042) und zeigte eine Respondenzrate von 79% nach 6 Monaten in der Open-Label-Erweiterung (OLE).
Zu den wichtigen Highlights gehört eine 100%ige Patientenbindung bis zur 12-Wochen-Studie, wobei 94% in die OLE wechselten. Das Medikament zeigte positive Effekte auf Knochen- und Nieren-Biomarker, mit einer 48%-igen Reduktion des Urin-Calciums bei behandelten Patienten. Die Behandlung wurde gut toleriert, ohne schwerwiegende unerwünschte Ereignisse oder Abbrüche. Basierend auf diesen Ergebnissen plant MBX, eine Phase-3-Studie im Jahr 2026 zu initiieren.
MBX Biosciences (Nasdaq: MBX) أعلنت عن نتائج إيجابية مهمة من تجربتها من المرحلة 2 Avail™ لعقار كانفوپراتيد Canvuparatide المستخدم مرة في الأسبوع لعلاج نقص الغدة جار درقية مزمن (HP). حققت الدراسة نقطة النهاية الأساسية بمعدل استجابة 63% عند 12 أسبوعاً مقارنة بـ 31% في الدواء الوهمي (p=0.042)، وأظهرت معدل استجابة 79% عند 6 أشهر في التمديد ذو الملصق المفتوح (OLE).
ومن النقاط الرئيسية الاحتفاظ بالمرضى بنسبة 100% حتى الدراسة لمدة 12 أسبوعاً، مع دخول 94% إلى التمديد. أظهر الدواء آثاراً إيجابية على مؤشرات العظام والكلى، مع انخفاض 48% في كالسيوم البول لدى المرضى المعالجين. كان العلاج متحملاً جيداً دون وقوع أحداث ضارة خطيرة أو انسحاب. بناءً على هذه النتائج، تخطط MBX لبدء تجربة المرحلة 3 في عام 2026.
MBX Biosciences（纳斯达克股票代码：MBX）宣布其阶段2 Avail™ 临床研究中每周一次给药的卡诺帕拉替迪（canvuparatide）用于慢性甲状旁腺功能减退症（HP）的显著积极结果。研究在12周时达到主要终点，63% 的应答率相比安慰剂的 31%（p=0.042），在开放标签扩展（OLE）中6个月时的应答率为 79%。
关键亮点包括在12周内达到 100% 的患者保留率，其中 94% 进入 OLE。药物对骨骼和肾脏生物标志物显示出积极作用，治疗组的 尿钙下降 48%。治疗耐受良好，未出现严重不良事件或中止。基于这些结果，MBX 计划在 2026 年启动 III 期试验。
- None.
- 19% injection site reactions reported vs 13% in placebo group
Insights
MBX's once-weekly canvuparatide showed strong efficacy in hypoparathyroidism with 63% responder rate at 12 weeks, advancing to Phase 3.
The Phase 2 Avail™ trial results for MBX Biosciences' once-weekly canvuparatide represent a significant clinical milestone in hypoparathyroidism treatment. The data shows 63% of treated patients achieved the primary endpoint at 12 weeks versus 31% for placebo (p=0.042), with response rates further improving to 79% at 6 months in the open-label extension.
What makes these results particularly impressive is the zero contribution from rescue therapy in responders and the 100% completion rate in the 12-week trial period, with 94% of patients choosing to continue treatment in the extension study. This exceptional retention suggests strong patient satisfaction with the treatment experience.
The drug demonstrated the expected physiological effects by improving bone turnover markers (BSAP, CTx, P1NP) and reducing urinary calcium by 48% in patients with elevated levels at baseline. The safety profile appears favorable with no treatment-related serious adverse events or discontinuations.
The once-weekly administration represents a potential paradigm shift for hypoparathyroidism management, which currently relies on complex daily regimens of calcium supplements and vitamin D. For patients struggling with unpredictable calcium fluctuations and the burden of multiple daily treatments, this simplified approach with continuous PTH-like exposure could significantly improve disease management and quality of life.
With Phase 3 preparations underway for 2026 and the strong foundation of these results, canvuparatide is positioning itself as a potential best-in-class therapy for this challenging endocrine disorder.
Statistically significant responder rate achieved at 12 weeks with zero contribution from rescue therapy (PRN) and further improvement sustained in open-label extension (OLE)
Positive findings in bone and kidney biomarkers
All patients completed the 12-week AvailTM trial and
Once-weekly canvuparatide was generally well tolerated, with no treatment-related serious adverse events or discontinuations during the 12-week trial
Preparation underway to initiate Phase 3 trial in 2026
Company to host conference call at 8:00 am ET today
CARMEL, Ind., Sept. 22, 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 once-weekly canvuparatide achieved the primary endpoint with statistical significance at Week 12 in its Phase 2 Avail™ trial, and demonstrated positive 6-month results from the OLE, in adult patients with chronic hypoparathyroidism (HP). All patients (n=64) completed the 12-week study, and
In the 12-week randomized portion of the trial,
Based on these positive results, MBX is preparing to initiate a Phase 3 clinical trial of once-weekly canvuparatide in 2026.
“The results from the Avail trial are encouraging. A once-weekly therapy could simplify administration and help address important unmet medical needs of patients with hypoparathyroidism,” said Mishaela Rubin, MD, MS, Professor of Medicine at Columbia University Vagelos College of Physicians and Surgeons and an investigator in the Avail clinical trial. “Hypoparathyroidism poses a substantial burden to patients, who often face complex treatment regimens and unpredictable swings in calcium levels that can lead to serious complications. The 12-week and 6-month data provide promising early evidence that this investigational therapy may offer a potential option for long-term management, pending further study.”
“We are very pleased with the clinically meaningful and statistically significant topline results from our once-weekly canvuparatide Phase 2 trial. These data reinforce our conviction that canvuparatide could become a potential best-in-class treatment for hypoparathyroidism and demonstrate the value of our novel Precision Endocrine Peptide platform technology,” said Kent Hawryluk, President and Chief Executive Officer of MBX Biosciences. “We believe the totality of the data support a once-weekly product profile with continuous infusion-like PTH exposure. These 12-week and 6-month results represent the potential for a meaningful improvement over current treatment options for HP patients and provide a strong foundation for further development. We look forward to sharing additional once-weekly canvuparatide clinical data at an upcoming medical meeting as we prepare for initiation of our Phase 3 trial.”
Phase 2 Avail Topline Results
12-week and 6-month Responder Rates:
- At 12 Weeks: The primary composite endpoint (maintaining albumin-adjusted serum calcium levels in the normal range (8.2–10.6 mg/dL) and independence from conventional therapy (active vitamin D and >600 mg/day of calcium supplements)) was achieved in
63% of canvuparatide-treated patients (30/48) compared with31% in placebo-treated patients (5/16) (p=0.042) at Week 12 - At 6 Months: In the OLE,
79% of patients (44/56 evaluable) who received treatment achieved responder status at 6 months, including patients initially randomized to placebo
Select Secondary and Exploratory Endpoints
- Pharmacokinetics: Pharmacokinetic (PK) findings were consistent with the Phase 1 results, supporting a once-weekly dosing schedule
- Bone Activity: Bone turnover and formation markers (BSAP, CTx and P1NP) increased over 12 weeks compared to placebo, consistent with enhanced bone remodeling
- Kidney Activity: In patients with elevated urine calcium at screening that normalized at Week 12, mean urine calcium was reduced by
48% in patients treated with once-weekly canvuparatide compared with33% on placebo
Safety Summary
- All doses of canvuparatide were generally well-tolerated with no discontinuations related to canvuparatide
- Most treatment emergent adverse events were categorized as mild or moderate
- No SAEs related to canvuparatide were reported
- Injection site reactions (ISRs):
19% in the pooled treatment group versus13% in placebo - No deaths were reported
“We are thrilled with these trial results,” said Patty Keating, Executive Director of the HypoPARAthyroidism Association. “For so many in our community, life with hypoparathyroidism means living with constant symptoms and the limitations of daily supplements. A once-weekly treatment option that maintains stable calcium control while reducing the day-to-day burden would be a major step forward.”
The Company will present additional data from the Phase 2 trial and OLE at an upcoming major medical meeting.
Conference Call
The Company will host a conference call and webcast today at 8 am ET to discuss the results from the Avail™ Phase 2 trial. Those who would like to participate may access the live webcast here or dial 1-877-407-0779 (US) or 1-201-389-0914 (international). The live and archived webcast of the call and slide presentation will be available in the Investors section of the Company’s website at https://investors.mbxbio.com/news-events/events.
About the Avail™ Trial
The Avail™ Phase 2 trial (NCT06465108) is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, pharmacokinetics, and efficacy of canvuparatide in patients with hypoparathyroidism. The study randomized 64 patients into four treatment arms: canvuparatide 400ug, 600ug, 800ug administered by subcutaneous once-weekly injection, and a placebo arm. The 12-week treatment period includes a four-week fixed dose period followed by an 8-week titration period during which canvuparatide dosing may be adjusted every two weeks in 200ug increments. The primary endpoint for efficacy is normalization of albumin adjusted serum calcium while independent from active vitamin D and calcium supplements (<600 mg/day) at Week 12. Secondary endpoints include safety and tolerability; pharmacokinetic profile; urine calcium, serum phosphorus, 1,25 dihydroxyvitamin D, and bone biomarkers. Following the 12-week treatment period, 60 patients (
About Hypoparathyroidism (HP)
HP is a rare endocrine disease caused by a deficiency of parathyroid hormone (PTH) released by the parathyroid glands that results in decreased calcium levels in the blood, leading to hypocalcemia. Hypocalcemia can cause a variety of 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. HP can interfere with daily activities, negatively impacting the quality of life for patients. We estimate that HP affects more than 250,000 individuals in the U.S. and Europe. The current standard of care for HP does not address the underlying cause of the disease, PTH deficiency, and consists primarily of high doses of oral calcium and active vitamin D supplements.
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 canvuparatide (MBX 2109) for the treatment of chronic hypoparathyroidism (HP) in Phase 2 development; imapextide (1416) for the treatment of post-bariatric hypoglycemia (PBH) in Phase 2 development; and an obesity portfolio that includes MBX 4291 in Phase 1 development, as well as multiple discovery and pre-clinical obesity candidates. The Company is based in Carmel, Indiana. To learn more, please visit the Company website at https://mbxbio.com/ and follow it on LinkedIn.
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: trial results from MBX’s Phase 2 trial of canvuparatide, including topline results; statements related to the potential for canvuparatide to be a once-weekly PTH replacement therapy; expectations regarding future clinical evaluation of canvuparatide; and statements relating to canvuparatide having a favorable safety profile.
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; MBX Biosciences’ ability to execute on its strategy including obtaining the requisite regulatory approvals on the expected timeline, if at all; uncertainties relating to preclinical and clinical development activities; the Company’s dependence on third parties to conduct clinical trials, manufacture its product candidates and develop and commercialize its product candidates, if approved; MBX Biosciences’ ability to attract, integrate and retain key personnel; risks related to the Company’s financial condition and need for substantial additional funds in order to complete development activities and commercialize a product candidate, if approved; risks related to regulatory developments and approval processes of the U.S. Food and Drug Administration and comparable foreign regulatory authorities; risks related to establishing and maintaining MBX Biosciences’ intellectual property protections; and risks related to the competitive landscape for MBX Biosciences’ product candidates; as well as other risks described in “Risk Factors,” in MBX Biosciences’ Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC, 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:
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Inizio Evoke Comms
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