Alpha Tau Announces FDA Approval of IDE to Initiate Multi-Center Investigator-Initiated Study of Alpha DaRT® in Immunocompromised Patients with Recurrent cSCC
Alpha Tau Medical (NASDAQ: DRTS, DRTSW) announced FDA approval of an Investigational Device Exemption (IDE) for a multi-center study using Alpha DaRT® to treat recurrent cutaneous Squamous Cell Carcinoma (cSCC) in immunocompromised patients. The study, led by Winship Cancer Institute of Emory University, will enroll up to 28 U.S. patients across 8 institutions.
The trial's primary efficacy objective is the objective response rate (ORR), with secondary objectives including progression-free survival, overall survival, and local control. Immunocompromised patients face a significantly higher risk of cSCC, with organ transplant recipients having a 65-100 fold greater incidence compared to the general population.
This study addresses clinician requests to treat immunocompromised patients, who are ineligible for Alpha Tau's ongoing ReSTART pivotal trial for recurrent cSCC. The company aims to provide a new potential treatment alternative for this vulnerable population.
Alpha Tau Medical (NASDAQ: DRTS, DRTSW) ha annunciato l'approvazione da parte della FDA di un'Investigational Device Exemption (IDE) per uno studio multicentrico che utilizza Alpha DaRT® per trattare il carcinoma squamoso cutaneo ricorrente (cSCC) in pazienti immunocompromessi. Lo studio, guidato dall'Emory Winship Cancer Institute, accoglierà fino a 28 pazienti statunitensi in 8 istituzioni.
L'obiettivo principale di efficacia del trial è il tasso di risposta obiettivo (ORR), con obiettivi secondari che includono la sopravvivenza libera da progressione, la sopravvivenza globale e il controllo locale. I pazienti immunocompromessi si trovano ad affrontare un rischio significativamente più elevato di cSCC, con i riceventi di trapianto d'organo che hanno un'incidenza da 65 a 100 volte maggiore rispetto alla popolazione generale.
Questo studio risponde alle richieste dei clinici di trattare i pazienti immunocompromessi, che non sono idonei per la sperimentazione fondamentale ReSTART di Alpha Tau per il cSCC ricorrente. L'azienda mira a fornire una nuova potenziale alternativa terapeutica per questa popolazione vulnerabile.
Alpha Tau Medical (NASDAQ: DRTS, DRTSW) ha anunciado la aprobación por parte de la FDA de una Investigational Device Exemption (IDE) para un estudio multicéntrico utilizando Alpha DaRT® para tratar el carcinoma espinocelular cutáneo recurrente (cSCC) en pacientes inmunocomprometidos. El estudio, dirigido por el Emory Winship Cancer Institute, inscribirá hasta 28 pacientes en EE. UU. en 8 instituciones.
El objetivo primario de eficacia del ensayo es la tasa de respuesta objetiva (ORR), con objetivos secundarios que incluyen la supervivencia libre de progresión, la supervivencia general y el control local. Los pacientes inmunocomprometidos enfrentan un riesgo significativamente mayor de cSCC, con los receptores de trasplante de órganos teniendo una incidencia de 65 a 100 veces mayor en comparación con la población general.
Este estudio responde a las solicitudes de los clínicos para tratar a pacientes inmunocomprometidos, que no son elegibles para el ensayo pivotal ReSTART de Alpha Tau para el cSCC recurrente. La empresa busca proporcionar una nueva alternativa de tratamiento potencial para esta población vulnerable.
알파 타우 메디컬( NASDAQ: DRTS, DRTSW)은 면역이 저하된 환자의 재발성 피부 편평 세포 암종(cSCC)을 치료하기 위해 Alpha DaRT®를 사용하는 다기관 연구에 대한 FDA의 Investigational Device Exemption(IDE)을 승인받았다고 발표했습니다. 이 연구는 에모리 대학교의 윈십 암 연구소가 주도하며, 8개의 의료 기관에서 최대 28명의 미국 환자를 모집할 예정입니다.
이 시험의 주요 효능 목표는 객관적 반응률(ORR)이며, 보조 목표로는 무진행 생존율, 전체 생존율 및 국소 통제가 포함됩니다. 면역이 저하된 환자는 cSCC의 위험이 상당히 높으며, 장기 이식 수혜자의 경우 일반 인구에 비해 65배에서 100배 더 높은 발생률을 보입니다.
이 연구는 면역이 저하된 환자를 치료해 달라는 임상의의 요청에 응답하며, 이들은 Alpha Tau의 진행 중인 재발성 cSCC에 대한 ReSTART 주요 시험에 적합하지 않습니다. 회사는 이 취약한 집단을 위한 새로운 잠재적 치료 대안을 제공하는 것을 목표로 하고 있습니다.
Alpha Tau Medical (NASDAQ: DRTS, DRTSW) a annoncé l'approbation par la FDA d'une Investigational Device Exemption (IDE) pour une étude multicentrique utilisant Alpha DaRT® pour traiter le carcinome à cellules squameuses cutané récurrent (cSCC) chez des patients immunodéprimés. L'étude, dirigée par le Winship Cancer Institute de l'Université Emory, inscrira jusqu'à 28 patients américains dans 8 institutions.
L'objectif principal du procès en termes d'efficacité est le taux de réponse objectif (ORR), avec des objectifs secondaires comprenant la survie sans progression, la survie globale et le contrôle local. Les patients immunodéprimés font face à un risque significativement plus élevé de cSCC, les receveurs de greffes d'organes ayant une incidence 65 à 100 fois supérieure à celle de la population générale.
Cette étude répond aux demandes des cliniciens de traiter les patients immunodéprimés, qui ne sont pas éligibles pour l'essai pivotal ReSTART d'Alpha Tau pour le cSCC récurrent. L'entreprise vise à fournir une nouvelle alternative thérapeutique potentielle pour cette population vulnérable.
Alpha Tau Medical (NASDAQ: DRTS, DRTSW) hat die FDA-Zulassung für eine Investigational Device Exemption (IDE) für eine multizentrische Studie zur Behandlung von wiederkehrendem kutanem Plattenepithelkarzinom (cSCC) bei immungeschwächten Patienten bekanntgegeben. Die Studie, die vom Winship Cancer Institute der Emory University geleitet wird, wird bis zu 28 US-Patienten an 8 Einrichtungen einschreiben.
Das primäre Wirksamkeitsziel der Studie ist die objektive Ansprechrate (ORR), während sekundäre Ziele die progressionsfreie Überlebenszeit, das Gesamtüberleben und die lokale Kontrolle umfassen. Immungeschwächte Patienten haben ein erheblich höheres Risiko für cSCC, wobei Organtransplantatempfänger eine 65- bis 100-fache höhere Inzidenz im Vergleich zur allgemeinen Bevölkerung aufweisen.
Diese Studie reagiert auf die Anfragen von Klinikern, immungeschwächte Patienten zu behandeln, die für die laufende ReSTART-Hauptstudie von Alpha Tau für wiederkehrendes cSCC nicht in Frage kommen. Das Unternehmen hat sich zum Ziel gesetzt, dieser schutzbedürftigen Gruppe eine neue potenzielle Behandlungsalternative anzubieten.
- FDA approval of IDE for multi-center study in immunocompromised patients
- Expansion of potential patient population for Alpha DaRT® treatment
- Addressing unmet medical need in high-risk patient group
- Collaboration with prestigious Winship Cancer Institute of Emory University
- patient enrollment (up to 28 patients) in the study
- Study results and potential FDA approval for this indication still pending
Insights
The FDA's approval of an IDE for Alpha Tau's Alpha DaRT® therapy in immunocompromised patients with recurrent cSCC is a significant development. This trial targets a important gap in treatment options for a highly vulnerable population. Key points:
- The study will enroll up to 28 patients across 8 U.S. institutions, focusing on those with weakened immune systems.
- Primary efficacy objective is the objective response rate (ORR), with secondary objectives including progression-free survival and overall survival.
- Immunocompromised patients have a
65-100% higher incidence of cSCC compared to the general population. - Immunosuppression is associated with a 2.32-times increased risk of disease-specific death in cSCC patients.
This trial could potentially provide a valuable treatment alternative for a patient group with options, potentially improving outcomes in a high-risk population.
The initiation of this trial for Alpha DaRT® in immunocompromised patients with recurrent cSCC is a promising step. As an oncologist, I see several important implications:
- Current treatment options for immunocompromised patients with recurrent cSCC are , making this trial particularly significant.
- The Alpha DaRT® therapy, using targeted alpha radiation, could potentially offer a less invasive alternative to surgery or systemic treatments.
- The focus on objective response rate (ORR) as the primary efficacy measure aligns with standard oncology practice for evaluating new therapies.
- The inclusion of progression-free survival and overall survival as secondary endpoints will provide important long-term efficacy data.
If successful, this trial could fill a critical treatment gap for a patient population that often faces challenges with traditional cancer therapies due to their compromised immune status.
From a market perspective, Alpha Tau's expansion into treating immunocompromised patients with recurrent cSCC presents significant potential:
- Addressable market expansion: With
50% or more of Caucasian transplant recipients developing cutaneous carcinomas, this represents a substantial patient population. - Unmet medical need: The higher risk and worse outcomes for immunocompromised patients highlight a clear market opportunity for effective treatments.
- Competitive advantage: Success in this niche could differentiate Alpha Tau in the oncology market, potentially leading to increased adoption and market share.
- Regulatory progress: FDA approval of the IDE demonstrates regulatory traction, which is important for investor confidence.
While it's early-stage, positive results from this trial could significantly enhance Alpha Tau's market position and valuation potential in the cancer therapy space.
-Trial to focus on particularly vulnerable subset of patients, following strong clinician interest in use of the Alpha DaRT in this patient population -
JERUSALEM, Sept. 20, 2024 (GLOBE NEWSWIRE) -- Alpha Tau Medical Ltd. ("Alpha Tau", or the “Company”) (NASDAQ: DRTS, DRTSW), the developer of the innovative alpha-radiation cancer therapy Alpha DaRT®, announced today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) application to initiate a multi-center study for the treatment of recurrent cutaneous Squamous Cell Carcinoma (cSCC) in immunocompromised patients using the Alpha DaRT.
The clinical study, which is an investigator-initiated study led by the Winship Cancer Institute of Emory University in Atlanta, has been approved to enroll up to 28 U.S. patients at up to 8 institutions in the U.S., and will focus on patients with recurrent cSCC who have a weakened immune system due to any primary or secondary immunodeficiencies, excluding diabetes. The primary efficacy objective of the study is the objective response rate (ORR) to the treatment, as measured by best overall response. Secondary efficacy objectives include progression-free survival, overall survival and local control up to twelve months after treatment, and the safety objective is the measurement of any related adverse events.
A 2015 article in Journal of Clinical Medicine noted that non-melanoma skin cancers represent a major cause of morbidity for patients after organ transplantation, and cSCC is the most common skin cancer seen in this population, with a 65–100 fold greater incidence in organ transplant recipients compared to the general population. For example, a 2003 article in the New England Journal of Medicine cited a number of sources indicating that
In addition, a 2019 article in JAMA Otolaryngology – Head & Neck Surgery found that immunosuppression is independently associated with a worse outcome in cSCC, with a 2.32-times increased risk of disease-specific death, after adjusting for assorted demographic factors.
"As we continue to progress in our ReSTART multi-center pivotal trial for recurrent cutaneous SCC, a number of investigators asked about the ability to treat immunocompromised patients, who are ineligible for the ReSTART trial," commented Alpha Tau CEO Uzi Sofer. "Emory University is an important partner of ours and we are proud to work with them in initiating a trial for this population. Given the continued requests we receive from clinicians to help them treat immunocompromised patients, we are confident that a successful clinical trial can help deliver an important new potential alternative for these patients."
Zachary Buchwald, MD, PhD, an Assistant Professor of Radiation Oncology at Winship Cancer Institute of Emory University and Principal Investigator of the trial, noted, “We are thrilled to be able to initiate this trial and pioneer the use of the Alpha DaRT in immunocompromised patients. As we continue to see the promise of the Alpha DaRT through our participation in the ReSTART trial, it is obvious to us that we need to use this treatment elsewhere, particularly in populations at risk such as this.”
Dr. Robert B. Den, Alpha Tau Chief Medical Officer, added, "This trial targets a particularly vulnerable population for whom treatment options are limited, which affords the ability to provide even more potential value to these patients. We are looking forward to treating patients in this trial soon."
About Alpha DaRT®
Alpha DaRT (Diffusing Alpha-emitters Radiation Therapy) is designed to enable highly potent and conformal alpha-irradiation of solid tumors by intratumoral delivery of radium-224 impregnated sources. When the radium decays, its short-lived daughters are released from the sources and disperse while emitting high-energy alpha particles with the goal of destroying the tumor. Since the alpha-emitting atoms diffuse only a short distance, Alpha DaRT aims to mainly affect the tumor, and to spare the healthy tissue around it.
About Alpha Tau Medical Ltd.
Founded in 2016, Alpha Tau is an Israeli oncology therapeutics company that focuses on research, development, and potential commercialization of the Alpha DaRT for the treatment of solid tumors. The technology was initially developed by Prof. Itzhak Kelson and Prof. Yona Keisari from Tel Aviv University.
Forward-Looking Statements
This press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. When used herein, words including "anticipate," "being," "will," "plan," "may," "continue," and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Alpha Tau's current expectations and various assumptions. Alpha Tau believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Alpha Tau may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation: (i) Alpha Tau's ability to receive regulatory approval for its Alpha DaRT technology or any future products or product candidates; (ii) Alpha Tau's limited operating history; (iii) Alpha Tau's incurrence of significant losses to date; (iv) Alpha Tau's need for additional funding and ability to raise capital when needed; (v) Alpha Tau's limited experience in medical device discovery and development; (vi) Alpha Tau's dependence on the success and commercialization of the Alpha DaRT technology; (vii) the failure of preliminary data from Alpha Tau's clinical studies to predict final study results; (viii) failure of Alpha Tau's early clinical studies or preclinical studies to predict future clinical studies; (ix) Alpha Tau's ability to enroll patients in its clinical trials; (x) undesirable side effects caused by Alpha Tau's Alpha DaRT technology or any future products or product candidates; (xi) Alpha Tau's exposure to patent infringement lawsuits; (xii) Alpha Tau's ability to comply with the extensive regulations applicable to it; (xiii) the ability to meet Nasdaq's listing standards; (xiv) costs related to being a public company; (xv) changes in applicable laws or regulations; and the other important factors discussed under the caption "Risk Factors" in Alpha Tau's annual report filed on form 20-F with the SEC on March 7, 2024, and other filings that Alpha Tau may make with the United States Securities and Exchange Commission. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While Alpha Tau may elect to update such forward-looking statements at some point in the future, except as required by law, it disclaims any obligation to do so, even if subsequent events cause its views to change. These forward-looking statements should not be relied upon as representing Alpha Tau's views as of any date subsequent to the date of this press release.
Investor Relations Contact:
IR@alphatau.com
FAQ
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