Krystal Biotech Announces Early Evidence of Monotherapy Activity in Heavily Pre-Treated Patients with Advanced Non-Small Cell Lung Cancer
Krystal Biotech (NASDAQ: KRYS) reported promising preliminary results from its KYANITE-1 study evaluating inhaled KB707 in lung tumor patients. The study showed significant efficacy in non-small cell lung cancer (NSCLC) patients, with a 27% objective response rate (ORR) and 73% disease control rate (DCR).
The trial involved 37 patients, including 17 with advanced NSCLC. Among 11 evaluable NSCLC patients, who had received median 4 prior therapy lines, KB707 demonstrated notable results with 7 patients still on treatment. In lung lesions specifically, the ORR was 36% with three partial responses and one complete response.
Based on these positive results, Krystal Biotech has expanded the study to include combination therapy cohorts with anti-PD-1 therapy and chemotherapy. The treatment has been generally well-tolerated with mostly mild to moderate adverse events.
Krystal Biotech (NASDAQ: KRYS) ha riportato risultati preliminari promettenti dal suo studio KYANITE-1 che valuta l'KB707 inalato nei pazienti con tumore polmonare. Lo studio ha dimostrato una significativa efficacia nei pazienti con cancro polmonare non a piccole cellule (NSCLC), con un 27% di tasso di risposta obiettivo (ORR) e un 73% di tasso di controllo della malattia (DCR).
Il trial ha coinvolto 37 pazienti, di cui 17 con NSCLC avanzato. Tra 11 pazienti NSCLC valutabili, che avevano ricevuto in media 4 linee di terapia precedenti, KB707 ha mostrato risultati notevoli con 7 pazienti ancora in trattamento. In particolare, nelle lesioni polmonari, l'ORR era del 36% con tre risposte parziali e una risposta completa.
Basandosi su questi risultati positivi, Krystal Biotech ha ampliato lo studio per includere coorti di terapia combinata con terapia anti-PD-1 e chemioterapia. Il trattamento è stato generalmente ben tollerato, con eventi avversi per lo più lievi o moderati.
Krystal Biotech (NASDAQ: KRYS) reportó resultados preliminares prometedores de su estudio KYANITE-1 que evalúa el KB707 inhalado en pacientes con tumor de pulmón. El estudio mostró una eficacia significativa en pacientes con cáncer de pulmón no microcítico (NSCLC), con un 27% de tasa de respuesta objetiva (ORR) y un 73% de tasa de control de la enfermedad (DCR).
El ensayo involucró a 37 pacientes, incluidos 17 con NSCLC avanzado. Entre 11 pacientes evaluables de NSCLC, que habían recibido una media de 4 líneas de terapia previas, KB707 demostró resultados notables con 7 pacientes aún en tratamiento. En lesiones pulmonares específicamente, la ORR fue del 36% con tres respuestas parciales y una respuesta completa.
Con base en estos resultados positivos, Krystal Biotech ha ampliado el estudio para incluir cohortes de terapia combinada con terapia anti-PD-1 y quimioterapia. El tratamiento ha sido generalmente bien tolerado, con eventos adversos mayormente leves a moderados.
크리스탈 바이오테크 (NASDAQ: KRYS)는 폐 종양 환자에게 흡입형 KB707를 평가하는 KYANITE-1 연구에서 유망한 초기 결과를 발표했습니다. 이 연구는 비소세포 폐암(NSCLC) 환자에서 27%의 객관적 반응률(ORR)과 73%의 질병 통제율(DCR)을 보였으며, 상당한 효능을 나타냈습니다.
이 시험에는 37명의 환자가 참여하였으며, 그 중 17명은 고급 NSCLC 환자였습니다. 중간 4회 치료를 받은 11명의 평가 가능한 NSCLC 환자 중, KB707는 7명을 치료 중인 상태로 유지하며 주목할 만한 결과를 보여주었습니다. 폐 병변에 대해서는 ORR이 36%로, 3명의 부분 반응과 1명의 완전 반응이 있었습니다.
이러한 긍정적인 결과를 바탕으로 크리스탈 바이오테크는 항-PD-1 치료 및 화학요법과의 병용 치료 집단을 포함하도록 연구를 확장하였습니다. 치료는 일반적으로 잘 견뎌졌으며, 대부분 경미하거나 중간 정도의 부작용이 발생했습니다.
Krystal Biotech (NASDAQ: KRYS) a rapporté des résultats préliminaires prometteurs de son étude KYANITE-1 évaluant le KB707 inhalé chez des patients atteints de tumeurs pulmonaires. L'étude a montré une efficacité significative chez les patients atteints de cancer du poumon non à petites cellules (NSCLC), avec un 27% de taux de réponse objective (ORR) et un 73% de taux de contrôle de la maladie (DCR).
L'essai a impliqué 37 patients, dont 17 atteints de NSCLC avancé. Parmi 11 patients NSCLC évaluables, qui avaient reçu en médiane 4 lignes de thérapie précédentes, le KB707 a démontré des résultats notables avec 7 patients encore sous traitement. En ce qui concerne les lésions pulmonaires, l'ORR était de 36% avec trois réponses partielles et une réponse complète.
Sur la base de ces résultats positifs, Krystal Biotech a élargi l'étude pour inclure des cohortes de thérapie combinée avec thérapie anti-PD-1 et chimiothérapie. Le traitement a généralement été bien toléré, avec des événements indésirables majoritairement légers à modérés.
Krystal Biotech (NASDAQ: KRYS) hat vielversprechende vorläufige Ergebnisse aus seiner KYANITE-1-Studie zur Bewertung des inhalativen KB707 bei Patienten mit Lungenkrebs berichtet. Die Studie zeigte eine signifikante Wirksamkeit bei Patienten mit nicht-kleinzelligem Lungenkarzinom (NSCLC), mit einer 27%igen objektiven Ansprechrate (ORR) und einer 73%igen Krankheitskontrollrate (DCR).
Die Studie umfasste 37 Patienten, darunter 17 mit fortgeschrittenem NSCLC. Bei 11 auswertbaren NSCLC-Patienten, die im Median 4 vorherige Therapielinien erhalten hatten, zeigte KB707 bemerkenswerte Ergebnisse, wobei 7 Patienten weiterhin behandelt wurden. Bei Lungenläsionen betrug die ORR 36% mit drei partiellen Antworten und einer kompletten Antwort.
Basierend auf diesen positiven Ergebnissen hat Krystal Biotech die Studie erweitert, um Kombinationstherapien mit Anti-PD-1-Therapie und Chemotherapie einzubeziehen. Die Behandlung wurde insgesamt gut vertragen, mit überwiegend milden bis moderaten unerwünschten Ereignissen.
- 27% objective response rate (ORR) and 73% disease control rate (DCR) in NSCLC patients
- 36% ORR in lung lesions with three partial responses and one complete response
- 7 out of 11 evaluable patients remain on treatment
- Treatment well-tolerated with mostly mild to moderate adverse events
- Study expansion to include combination therapy cohorts
- Small patient sample size (11 evaluable NSCLC patients)
- Early-stage data with duration of treatment (10.3 to 33.3 weeks)
Insights
The preliminary results from the KYANITE-1 study demonstrate significant promise for KB707 in treating advanced NSCLC. The
The novel inhaled delivery approach maximizes local efficacy while minimizing systemic exposure, potentially offering a better therapeutic window than traditional systemic treatments. The planned combination studies with anti-PD-1 therapy could further enhance efficacy, as cytokine therapy often shows synergistic effects with checkpoint inhibition.
This positive early clinical data represents a significant value driver for Krystal Biotech, potentially expanding their addressable market into oncology. NSCLC represents a massive market opportunity, with global NSCLC therapeutics valued at over
The successful demonstration of repeat HSV-1 based gene delivery to lungs also validates the broader platform technology, which could have implications for the company's other respiratory programs. The expansion into combination therapy cohorts suggests confidence in the initial data and could accelerate the path to potential commercialization if successful.
Preliminary clinical data in post-anti-PD-1 NSCLC patients demonstrated a
Monotherapy activity with inhaled KB707 provides further evidence of successful repeat administration of HSV-1 based inhaled lung gene delivery and builds on recent clinical data update for CF and AAT deficiency respiratory disease programs
PITTSBURGH, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today initial clinical results from its ongoing KYANITE-1 study evaluating inhaled KB707 in patients with solid tumors of the lung. KB707 administered via inhalation demonstrated early evidence of monotherapy activity that was most pronounced in patients with advanced non-small cell lung cancer (NSCLC), where an objective response rate (ORR) of
“The inhaled local delivery of cytokine that maximizes efficacy and limits systemic toxicity is truly innovative,” said Wen Wee Ma, MBBS, Vice Chair of Research and Director of the Novel Cancer Therapeutics Center at Cleveland Clinic. “To see potential benefit as a monotherapy in NSCLC patients who have progressed after standard of care treatments is very encouraging and provides much needed hope.”
KYANITE-1 is an ongoing, open-label, multicenter, dose escalation and expansion study evaluating inhaled KB707 for the treatment of solid tumors of the lung. Treatments of either 108 PFU or 109 PFU of KB707 were evaluated in dose escalation, following which 109 PFU was selected for dose expansion. Frequency of KB707 administration has been consistent throughout dose escalation and expansion, with patients receiving KB707 via inhalation once weekly for the first three weeks, then once every three weeks. Trial objectives include evaluation of safety, tolerability, and tumor response measured using RECIST v1.1 criteria. Additional details of the KYANITE-1 study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.
The first patient in KYANITE-1 was dosed on April 17, 2024. A total of 37 patients were enrolled and received at least one dose of inhaled KB707, including 17 patients with a diagnosis of advanced NSCLC. All patients had malignant lesions in the lung at baseline.
Inhaled KB707 has been safe and generally well tolerated to date in this diverse, heavily pre-treated patient population with advanced disease, and amenable to administration in an outpatient setting. Treatment-emergent adverse events have been predictable and consistent with both the underlying disease and known adverse event profiles of interleukin-2 and interleukin-12. The majority of treatment-related adverse events have been mild to moderate in severity and transient, with no Grade 4 or 5 adverse events observed.
Clinical activity observed to date in the KYANITE-1 study has shown the most therapeutic benefit in patients with advanced NSCLC. As of the data cut-off, 11 NSCLC patients were evaluable for response with at least one radiographic scan and RECIST v1.1 evaluation. Patients included in the analysis were heavily pre-treated with 4 median lines of prior therapy and all had received at least one line of prior immunotherapy. In this NSCLC patient analysis cohort, an ORR of
In addition to preliminary evidence of abscopal effect and treatment benefit outside of the lung, treatment responses in lesions of the lung were especially notable. Among the same 11 evaluable NSCLC patients, the ORR in target lung lesions specifically was
“Signals of monotherapy activity with inhaled KB707, although early, are an exciting milestone for our program and highlight the significant potential of our vectorized cytokine approach in the treatment of difficult cancers,” said Suma Krishnan, President of Research and Development of Krystal Biotech. “These data add to a rapidly growing clinical dataset, generated across multiple programs and patient populations, demonstrating that our HSV-1 platform can safely and repeatedly deliver functional genetic material to the lung and impact the course of disease. We are excited about the implications for our platform and the prospect of delivering meaningful clinical benefit to patients suffering from rare and serious lung diseases.”
Based on positive initial results in monotherapy, the Company has amended the KYANITE-1 protocol to add two cohorts evaluating inhaled KB707 for the treatment of advanced NSCLC in combination with either anti-programmed cell death protein 1 (PD-1) therapy or anti-PD-1 therapy and chemotherapy. No patients have been enrolled in the combination expansion cohorts to date.
The Company expects to disclose detailed and updated results of KYANITE-1 at future scientific conference(s).
About IL-2, IL-12, and KB707
IL-2 and IL-12 are secreted cytokines with complementary functions promoting cell-mediated immunity in humans. Both IL-2 and IL-12 have been shown to elicit anti-tumor immune responses in preclinical or clinical models and have been extensively studied for their potential in cancer immunotherapy. Despite promising signs of efficacy, it has proven difficult to effectively harness IL-2 and IL-12 for therapeutic benefit, as systemic administration is often poorly tolerated, and their inherently short half-lives necessitate high dose levels and extremely frequent dose intervals. KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 directly to a patient’s tumor(s) and promote systemic immune-mediated tumor clearance. KB707 targets solid tumors that are accessible via intratumoral injection or inhalation.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK® is the Company’s first commercial product, the first-ever redosable gene therapy, and the first medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter).
Forward-Looking Statements
Any statements in this press release about future expectations, plans, and prospects for Krystal Biotech, Inc., including statements about the significant potential of the Company’s vectorized cytokine approach in the treatment of difficult cancers; the Company’s HSV-1 platform and its ability to safely and repeatedly deliver functional genetic material to the lung and impact the course of disease; the Company’s expectation that it will disclose detailed and updated results of KYANITE-1 at future scientific conference(s); 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 within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including uncertainties inherent in the initiation and conduct of clinical trials and such other important factors as are set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.
CONTACT
Investors and Media:
Stéphane Paquette, PhD
Krystal Biotech
spaquette@krystalbio.com
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