KALA BIO Announces Completion of Enrollment in CHASE Clinical Trial Evaluating KPI-012 for the Treatment of Persistent Corneal Epithelial Defect (PCED)
KALA BIO (NASDAQ:KALA) has completed enrollment in its CHASE Phase 2b clinical trial for KPI-012, a treatment for persistent corneal epithelial defect (PCED). The trial enrolled 79 patients across 37 sites in the US and Latin America, evaluating two doses of KPI-012 ophthalmic solution versus vehicle control.
The study aims to assess the safety and efficacy of KPI-012, a human mesenchymal stem cell secretome (MCS-S), with topline results expected by Q3 2025. If successful, the CHASE trial could potentially serve as a pivotal trial for a Biologics License Application (BLA). The treatment targets an estimated 100,000 PCED patients in the US, addressing an unmet need as there are currently no FDA-approved products for all PCED etiologies.
[ "KPI-012 has received both Orphan Drug and Fast Track designations from FDA", "Trial could potentially serve as pivotal study for BLA submission", "Addresses large market opportunity of 100,000 PCED patients in US", "Successfully completed enrollment with 79 patients across 37 sites" ]KALA BIO (NASDAQ:KALA) ha completato l'arruolamento nel suo studio clinico di Fase 2b CHASE per KPI-012, un trattamento per il difetto persistente dell'epitelio corneale (PCED). Lo studio ha coinvolto 79 pazienti in 37 centri negli Stati Uniti e in America Latina, valutando due dosi della soluzione oftalmica KPI-012 rispetto al controllo con veicolo.
L'obiettivo dello studio è valutare la sicurezza e l'efficacia di KPI-012, un secretoma di cellule staminali mesenchimali umane (MCS-S), con risultati principali attesi entro il terzo trimestre del 2025. Se avrà successo, lo studio CHASE potrebbe rappresentare un trial pivotale per la presentazione di una domanda di autorizzazione per prodotti biologici (BLA). Il trattamento si rivolge a circa 100.000 pazienti con PCED negli Stati Uniti, rispondendo a un bisogno insoddisfatto dato che attualmente non esistono prodotti approvati dalla FDA per tutte le eziologie del PCED.
- KPI-012 ha ricevuto le designazioni di Farmaco Orfano e Fast Track dalla FDA
- Lo studio potrebbe fungere da trial pivotale per la presentazione della BLA
- Risponde a un'ampia opportunità di mercato con 100.000 pazienti PCED negli USA
- Completato con successo l'arruolamento di 79 pazienti in 37 centri
KALA BIO (NASDAQ:KALA) ha completado la inscripción en su ensayo clínico de Fase 2b CHASE para KPI-012, un tratamiento para el defecto persistente del epitelio corneal (PCED). El ensayo incluyó a 79 pacientes en 37 sitios en Estados Unidos y América Latina, evaluando dos dosis de la solución oftálmica KPI-012 frente al control con vehículo.
El estudio busca evaluar la seguridad y eficacia de KPI-012, un secretoma de células madre mesenquimales humanas (MCS-S), con resultados principales esperados para el tercer trimestre de 2025. Si tiene éxito, el ensayo CHASE podría servir como estudio pivotal para la solicitud de licencia de productos biológicos (BLA). El tratamiento está dirigido a unos 100,000 pacientes con PCED en Estados Unidos, cubriendo una necesidad no satisfecha ya que actualmente no existen productos aprobados por la FDA para todas las etiologías del PCED.
- KPI-012 ha recibido las designaciones de Medicamento Huérfano y Fast Track de la FDA
- El ensayo podría servir como estudio pivotal para la presentación de la BLA
- Atiende una gran oportunidad de mercado con 100,000 pacientes con PCED en EE.UU.
- Completó exitosamente la inscripción con 79 pacientes en 37 sitios
KALA BIO (NASDAQ:KALA)가 지속성 각막 상피 결손증(PCED) 치료제인 KPI-012의 CHASE 2b상 임상시험 등록을 완료했습니다. 이 임상시험은 미국 및 라틴 아메리카의 37개 기관에서 79명의 환자를 등록하여 KPI-012 안약 두 가지 용량과 대조군(플라시보)을 비교 평가했습니다.
본 연구는 인간 중간엽 줄기세포 시크리톰(MCS-S)인 KPI-012의 안전성과 유효성을 평가하는 것을 목표로 하며, 주요 결과는 2025년 3분기까지 발표될 예정입니다. 성공할 경우 CHASE 임상시험은 생물학적 제제 허가 신청(BLA)을 위한 중추적 임상시험이 될 수 있습니다. 이 치료제는 미국 내 약 100,000명의 PCED 환자를 대상으로 하며, 현재 FDA 승인을 받은 PCED 전 원인에 대한 치료제가 없어 미충족 의료 수요를 해결합니다.
- KPI-012는 FDA로부터 희귀의약품 및 패스트트랙 지정 받음
- 임상시험은 BLA 제출을 위한 중추 임상시험이 될 가능성 있음
- 미국 내 100,000명의 PCED 환자라는 큰 시장 기회 대응
- 37개 기관에서 79명의 환자 등록 성공적으로 완료
KALA BIO (NASDAQ:KALA) a terminé le recrutement de son essai clinique de phase 2b CHASE pour KPI-012, un traitement du défaut épithélial cornéen persistant (PCED). L'essai a inclus 79 patients répartis sur 37 sites aux États-Unis et en Amérique latine, évaluant deux doses de la solution ophtalmique KPI-012 contre un contrôle par véhicule.
L'étude vise à évaluer la sécurité et l'efficacité de KPI-012, un secretome de cellules souches mésenchymateuses humaines (MCS-S), avec des résultats principaux attendus d'ici le troisième trimestre 2025. En cas de succès, l'essai CHASE pourrait servir d'étude pivot pour une demande d'autorisation de mise sur le marché biologique (BLA). Le traitement cible environ 100 000 patients atteints de PCED aux États-Unis, répondant à un besoin médical non satisfait car aucun produit n'est actuellement approuvé par la FDA pour toutes les étiologies du PCED.
- KPI-012 a obtenu les désignations de médicament orphelin et de procédure accélérée (Fast Track) de la FDA
- L'essai pourrait servir d'étude pivot pour la soumission de la BLA
- Répond à une large opportunité de marché avec 100 000 patients PCED aux États-Unis
- Recrutement réussi avec 79 patients sur 37 sites
KALA BIO (NASDAQ:KALA) hat die Einschreibung in seine CHASE Phase 2b-Studie für KPI-012, eine Behandlung für persistierende Hornhautepitheldefekte (PCED), abgeschlossen. Die Studie rekrutierte 79 Patienten an 37 Standorten in den USA und Lateinamerika und verglich zwei Dosierungen der KPI-012 Augenlösung mit einem Vehikel-Kontrollpräparat.
Ziel der Studie ist es, die Sicherheit und Wirksamkeit von KPI-012, einem humanen mesenchymalen Stammzellsekretom (MCS-S), zu bewerten, wobei Topline-Ergebnisse für das dritte Quartal 2025 erwartet werden. Bei Erfolg könnte die CHASE-Studie als entscheidende Studie für die Einreichung eines Biologics License Application (BLA) dienen. Die Behandlung richtet sich an schätzungsweise 100.000 PCED-Patienten in den USA und deckt einen ungedeckten medizinischen Bedarf ab, da derzeit keine von der FDA zugelassenen Produkte für alle PCED-Ätiologien existieren.
- KPI-012 hat sowohl Orphan Drug- als auch Fast Track-Designationen von der FDA erhalten
- Studie könnte als entscheidende Studie für die BLA-Einreichung dienen
- Adressiert eine große Marktchance von 100.000 PCED-Patienten in den USA
- Erfolgreich die Einschreibung von 79 Patienten an 37 Standorten abgeschlossen
- None.
- Results are pending and success is not guaranteed
- Still requires regulatory discussions and approval process
Insights
KALA's KPI-012 PCED trial completes enrollment; topline results in Q3 2025 could support BLA submission for an untreated eye condition.
KALA BIO has reached a significant clinical milestone with the completion of enrollment in their CHASE Phase 2b trial for KPI-012, a human mesenchymal stem cell secretome (MSC-S) therapy targeting persistent corneal epithelial defect (PCED). The trial successfully randomized 79 patients across 37 sites in the US and Latin America, testing two doses against a vehicle control.
This trial carries substantial strategic importance as KALA believes it could potentially serve as a pivotal trial to support a Biologics License Application (BLA), pending positive results expected by Q3 2025. The FDA has already granted both Orphan Drug and Fast Track designations for KPI-012, recognizing the therapy's potential to address a serious condition with limited treatment options.
The study design is methodologically sound - randomized, double-masked, and vehicle-controlled - with the primary endpoint being complete healing of PCED as measured by corneal fluorescein staining photographs analyzed by an independent reading center. This objective endpoint strengthens the trial's scientific rigor.
What makes KPI-012 particularly noteworthy is its potential to treat all underlying etiologies of PCED, which affects an estimated 100,000 people in the United States. Currently, there are no FDA-approved products with a broad indication covering all PCED etiologies, creating a significant market opportunity if the therapy proves successful.
The completion of enrollment keeps KALA on track with their clinical development timeline, with topline results expected within the next quarter. If positive, these results could accelerate the regulatory pathway through the Fast Track designation, potentially bringing this therapy to patients with limited treatment options more quickly.
-- Topline results from the CHASE trial expected by end of Q3 2025 --
-- Potential for CHASE to serve as a pivotal trial in support of a Biologics License Application (BLA) submission, contingent on positive results --
ARLINGTON, Mass., July 09, 2025 (GLOBE NEWSWIRE) -- KALA BIO, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye, today announced the completion of patient enrollment in the CHASE (Corneal Healing After SEcretome therapy) Phase 2b clinical trial evaluating KPI-012, a human mesenchymal stem cell secretome (MCS-S), for the treatment of persistent corneal epithelial defect (PCED).
The CHASE Phase 2b trial is a multicenter, randomized, double-masked, vehicle-controlled, parallel-group study to evaluate the safety and efficacy of two doses of KPI-012 ophthalmic solution (3 U/mL and 1 U/mL) versus vehicle dosed topically QID for 56 days. The CHASE trial randomized 79 patients across 37 sites in the United States and Latin America with verified PCEDs at baseline that will be eligible for inclusion in the primary efficacy analysis. The primary endpoint is complete healing of PCED as measured by corneal fluorescein staining photographs analyzed by a masked central reading center.
“The completion of enrollment in the CHASE trial is a significant milestone in our pursuit to develop and deliver a treatment that addresses the high unmet need for patients with PCED,” said Kim Brazzell, Ph.D., Head of R&D and Chief Medical Officer of KALA BIO. “There are currently no U.S. Food and Drug Administration (FDA)-approved products with a broad indication covering all underlying etiologies of PCED. Given its potential to treat all underlying etiologies of PCED, we believe KPI-012 could be a significant advance for the treatment of the estimated 100,000 people with PCED in the United States.”
KALA expects to report topline data in the third quarter of 2025. Contingent on positive results and subject to discussion with regulatory authorities, the Company believes the CHASE trial could potentially serve as a pivotal trial required to support a Biologics License Application (BLA) to FDA. KPI-012 has received both Orphan Drug and Fast Track designations from the FDA for the treatment of PCED.
About KALA BIO, Inc.
KALA is a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye. KALA’s biologics-based investigational therapies utilize KALA’s proprietary mesenchymal stem cell secretome (MSC-S) platform. KALA’s lead product candidate, KPI-012, is a human MSC-S, which contains numerous human-derived biofactors, such as growth factors, protease inhibitors, matrix proteins and neurotrophic factors that can potentially correct the impaired corneal healing that is an underlying etiology of multiple severe ocular diseases. KPI-012 is currently in clinical development for the treatment of persistent corneal epithelial defect (PCED), a rare disease of impaired corneal healing, for which it has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration. KALA is also targeting the potential development of KPI-012 for the treatment of Limbal Stem Cell Deficiency and other rare corneal diseases that threaten vision and has initiated preclinical studies to evaluate the potential utility of its MSC-S platform for retinal degenerative diseases, such as Retinitis Pigmentosa and Stargardt Disease. For more information on KALA, please visit www.kalarx.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties. Any statements in this press release about KALA’s future expectations, plans and prospects, including but not limited to statements about KALA’s expectations with respect to potential advantages of KPI-012 and its MSC-S platform; the clinical utility of KPI-012 for PCED; anticipated timelines to report topline data for the CHASE Phase 2b clinical trial of KPI-012; KALA’s belief that the CHASE Phase 2b trial could serve as the first of two pivotal trials required to support the submission of a BLA to the FDA; KALA’s plans to pursue research and development of KPI-012 and its MSC-S platform for other indications; and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions constitute forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: market conditions; uncertainties regarding availability and timing of data from clinical trials; whether results of early clinical trials or trials in different disease indications will be indicative of the results of ongoing or future trials; whether results of the Phase 1b clinical trial of KPI-012 will be indicative of results for any future clinical trials and studies of KPI-012, including the CHASE trial; whether interim data from a clinical trial will be predictive of the results of the trial; uncertainties associated with regulatory review of clinical trials and applications for marketing approvals; and other factors discussed in the “Risk Factors” section of KALA’s Annual Report on Form 10-K, most recently filed Quarterly Report on Form 10-Q and other filings KALA makes with the Securities and Exchange Commission. These forward-looking statements represent KALA’s views as of the date of this press release and should not be relied upon as representing KALA’s views as of any date subsequent to the date hereof. KALA does not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Investor Contact:
Taylor Steiner
taylor.steiner@precisionaq.com
212-362-1200
FAQ
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