Calliditas announces positive TRANSFORM Phase 2b topline data in primary biliary cholangitis
Calliditas Therapeutics AB (NASDAQ: CALT) announced positive topline results from its Phase 2b TRANSFORM trial evaluating setanaxib in patients with primary biliary cholangitis (PBC) and elevated liver stiffness. The study met its primary endpoint, showing statistically significant improvement in Alkaline Phosphatase (ALP) for both tested doses versus placebo. Key findings include:
- 19% ALP improvement in the 1600mg arm
- 14% ALP improvement in the 1200mg arm
- Positive trends on liver stiffness at 24 weeks
- Generally well-tolerated treatment
- Efficacy demonstrated in patients already on multiple medications
The company is conducting additional trials with setanaxib, including a Phase 2 trial in idiopathic pulmonary fibrosis (IPF) and a Phase 2 proof of concept trial in Alport syndrome.
Calliditas Therapeutics AB (NASDAQ: CALT) ha annunciato risultati positivi preliminari dal suo trial di Fase 2b TRANSFORM che valuta il setanaxib in pazienti con colangite biliare primaria (PBC) e rigidità epatica elevata. Lo studio ha raggiunto il suo obiettivo primario, mostrando un significativo miglioramento statistico nella fosfatasi alcalina (ALP) per entrambe le dosi testate rispetto al placebo. I risultati chiave includono:
- Miglioramento del 19% dell'ALP nel gruppo da 1600mg
- Miglioramento del 14% dell'ALP nel gruppo da 1200mg
- Tendenze positive sulla rigidità epatica a 24 settimane
- Trattamento generalmente ben tollerato
- Efficacia dimostrata in pazienti già in trattamento con più farmaci
La società sta conducendo trial aggiuntivi con il setanaxib, inclusi un trial di Fase 2 in fibrosi polmonare idiopatica (IPF) e un trial di Fase 2 di prova di concetto nella sindrome di Alport.
Calliditas Therapeutics AB (NASDAQ: CALT) anunció resultados preliminares positivos de su ensayo TRANSFORM de Fase 2b que evalúa el setanaxib en pacientes con colangitis biliar primaria (PBC) y rigidez hepática elevada. El estudio alcanzó su objetivo primario, mostrando una mejora estadísticamente significativa en la fosfatasa alcalina (ALP) para ambas dosis probadas en comparación con el placebo. Los hallazgos clave incluyen:
- Mejora del 19% en el ALP en el grupo de 1600mg
- Mejora del 14% en el ALP en el grupo de 1200mg
- Tendencias positivas en la rigidez hepática a las 24 semanas
- Tratamiento generalmente bien tolerado
- Eficacia demostrado en pacientes ya en múltiples medicamentos
La compañía está llevando a cabo ensayos adicionales con setanaxib, incluyendo un ensayo de Fase 2 en fibrosis pulmonar idiopática (IPF) y un ensayo de concepto de Fase 2 en la síndorme de Alport.
Calliditas Therapeutics AB (NASDAQ: CALT)는 원발성 담즙성 담관염 (PBC) 환자와 간 경직이 증가한 환자를 대상으로 한 2b상 TRANSFORM 임상 시험에서 세타나시빕의 긍정적인 초기 결과를 발표했습니다. 연구는 주요 목표를 달성하였으며, 알칼리성 인산가수분해효소 (ALP)의 통계적으로 유의미한 개선을 보여주었습니다. 주요 발견 사항은 다음과 같습니다:
- 1600mg 투여군에서 ALP 19% 개선
- 1200mg 투여군에서 ALP 14% 개선
- 24주 시점에서 간 경직에 긍정적인 경향
- 일반적으로 잘 견디는 치료
- 다수의 약물에 이미 투여 중인 환자에서 효능 입증
회사는 세타나시빕으로 추가 임상 시험을 수행하고 있으며, 여기에는 특발성 폐섬유증 (IPF)에서의 2상 시험과 알포트 증후군에서의 개념 증명 시험이 포함됩니다.
Calliditas Therapeutics AB (NASDAQ: CALT) a annoncé des résultats préliminaires positifs issus de son essai TRANSFORM de phase 2b évaluant le setanaxib chez des patients présentant une cholangiite biliaire primitive (PBC) et une rigidité hépatique élevée. L'étude a atteint son objectif principal, montrant une amélioration statistiquement significative de la phosphatase alcaline (ALP) pour les deux doses testées par rapport au placebo. Les principales conclusions comprennent :
- Amélioration de 19 % de l'ALP dans le bras de 1600 mg
- Amélioration de 14 % de l'ALP dans le bras de 1200 mg
- Tendances positives sur la rigidité hépatique à 24 semaines
- Traitement généralement bien toléré
- Efficacité démontrée chez des patients déjà sous plusieurs médicaments
La société mène d'autres essais avec le setanaxib, y compris un essai de phase 2 dans la fibrose pulmonaire idiopathique (IPF) et un essai de preuve de concept de phase 2 dans le syndrome d'Alport.
Calliditas Therapeutics AB (NASDAQ: CALT) kündigte positive vorläufige Ergebnisse aus seiner Phase 2b TRANSFORM-Studie an, die Setanaxib bei Patienten mit primärer biliärer Cholangitis (PBC) und erhöhter Lebersteifheit bewertet. Die Studie hat ihr primäres Ziel erreicht und zeigte eine statistisch signifikante Verbesserung der alkalischen Phosphatase (ALP) für beide getesteten Dosen im Vergleich zu Placebo. Zu den wichtigsten Ergebnissen gehören:
- 19% ALP-Verbesserung in der 1600mg-Gruppe
- 14% ALP-Verbesserung in der 1200mg-Gruppe
- Positive Trends bei der Lebersteifheit nach 24 Wochen
- Generell gut verträgliche Behandlung
- Wirksamkeit bei Patienten, die bereits mehrere Medikamente einnehmen
Das Unternehmen führt weitere Studien mit Setanaxib durch, einschließlich einer Phase-2-Studie bei idiopathischer Lungenfibrose (IPF) und einer Phase-2- Machbarkeitsstudie bei Alport-Syndrom.
- Phase 2b TRANSFORM trial met its primary endpoint with statistically significant ALP improvement
- 19% ALP improvement in the 1600mg arm and 14% in the 1200mg arm
- Positive trends observed on liver stiffness at 24 weeks
- Efficacy demonstrated in patients already on multiple medications
- Additional ongoing trials for setanaxib in IPF and Alport syndrome
- Higher frequency of treatment emergent adverse events leading to study discontinuation in active treatment groups compared to placebo
Insights
The positive Phase 2b TRANSFORM trial results for setanaxib in primary biliary cholangitis (PBC) patients are highly significant for Calliditas Therapeutics. As a Medical Research Analyst, I find several key points noteworthy:
- The trial met its primary endpoint with statistically significant improvements in Alkaline Phosphatase (ALP) levels:
19% reduction in the 1600mg arm and14% in the 1200mg arm compared to placebo. - Importantly, over
40% of trial participants were on dual therapy and13% on triple therapy, suggesting setanaxib provides incremental benefits beyond current standard of care. - Positive trends in liver stiffness reduction were observed, although specific data wasn't provided.
- The safety profile appears generally favorable, with similar overall adverse event rates between treatment and placebo groups.
These results are particularly impressive given the challenging patient population and relatively small sample size of 76 patients. The data supports setanaxib's potential as a first-in-class NOX inhibitor for PBC treatment, especially for patients with inadequate response to current therapies.
However, it's important to note that this is a Phase 2b trial and larger Phase 3 studies will be necessary to confirm efficacy and safety. The higher discontinuation rate in the treatment group also warrants further investigation in future trials.
From a financial perspective, these positive Phase 2b results for setanaxib in PBC are a significant milestone for Calliditas Therapeutics. Here's why this news is particularly impactful:
- Market Potential: PBC is a rare disease with treatment options. A novel therapy showing efficacy could capture substantial market share.
- Competitive Advantage: As a first-in-class NOX inhibitor, setanaxib could differentiate Calliditas in the PBC market, potentially commanding premium pricing.
- Pipeline Validation: Success in PBC validates Calliditas' NOX platform, potentially increasing investor confidence in other pipeline candidates using the same technology.
- Near-term Catalysts: With additional readouts expected in Q4 2024/Q1 2025 for IPF and 2025 for Alport syndrome, Calliditas has multiple value-driving events on the horizon.
However, investors should consider that:
- Significant R&D expenses will likely continue as setanaxib progresses to Phase 3 trials.
- Commercialization is still years away, assuming successful completion of the clinical development program.
- As a small-cap biotech, Calliditas may need additional financing to fund late-stage development and potential commercialization efforts.
Overall, while risks remain, this news significantly de-risks Calliditas' lead program and could attract increased investor interest in the near term.
As a Market Research Analyst, I see several key implications of Calliditas' positive Phase 2b results for setanaxib in PBC:
- Unmet Medical Need: PBC affects approximately 130,000 people in the US and has treatment options. A new therapy showing efficacy could rapidly gain market share.
- Competitive Landscape: Current treatments include ursodeoxycholic acid (UDCA) and Ocaliva (obeticholic acid). Setanaxib's ability to show benefits in patients already on these therapies is a significant competitive advantage.
- Market Expansion: By potentially addressing patients with inadequate response to current therapies, setanaxib could expand the treatable PBC patient population.
- Pricing Potential: As a novel, first-in-class therapy for a rare disease, setanaxib could command premium pricing if approved.
- Future Indications: Success in PBC could pave the way for setanaxib's development in other fibrotic diseases, expanding its market potential.
However, challenges remain:
- Regulatory hurdles and the need for larger Phase 3 trials before potential approval.
- Educating physicians on a new mechanism of action (NOX inhibition) in PBC treatment.
- Potential competition from other novel therapies in development for PBC.
Overall, these results position Calliditas favorably in the PBC market, with potential for significant market penetration if further development is successful.
The TRANSFORM trial is a double-blind, randomized, placebo-controlled Phase 2b study investigating the effect of setanaxib 800 mg AM + 400 mg PM, ("1200 mg arm") and 800 mg BID ("1600 mg arm") over 24 weeks of treatment. The basis for the analysis consisted of a dataset of 76 patients with primary biliary cholangitis (PBC) and elevated liver stiffness.
The treatment groups were relatively well-balanced with no clinically relevant differences between the groups observed at baseline. The result is particularly encouraging as over
"It is very encouraging to see a statistically significant treatment effect in this hard-to-treat population which is already on multiple medications in this relatively small study," said Professor Dave Jones OBE; Director, NHIP Academy; Director, Newcastle Centre for Rare Disease; Professor of Liver Immunology, Newcastle University; and Honorary Consultant Hepatologist, Newcastle upon Tyne Hospitals.
"These positive data provide further clinical evidence of the potential of setanaxib in multiple rare diseases, and we are very pleased that we now have additional positive clinical evidence in support of our unique, first in class NOX platform. We also look forward to the read out of the investigator led study in IPF as well as the ongoing study in Alport syndrome in due course," said CEO Renée Aguiar-Lucander.
"I am delighted that we have seen statistically significant and clinically meaningful improvements in ALP with encouraging trends in other outcomes in this population of patients with PBC. I'd like to extend my thanks to investigators, clinical trial site staff, and most importantly patients, who have all contributed to this important study," said CMO Richard Philipson.
The company is conducting additional clinical trials with setanaxib and is expecting the investigator led Phase 2 trial in IPF (idiopathic pulmonary fibrosis) to provide top line data in Q4 2024 / Q1, 2025. There is also an ongoing Phase 2 proof of concept trial in Alport syndrome, which is expected to deliver top line data in 2025.
For further information, please contact:
Åsa Hillsten, Head of IR & Sustainability, Calliditas
Tel.: +46 76 403 35 43, Email: asa.hillsten@calliditas.com
The information was sent for publication, through the agency of the contact persons set out above, on July 26, 2024 at 08:00 a.m. CET.
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The following files are available for download:
Press release TRANSFORM_ENG |
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SOURCE Calliditas Therapeutics
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