Krystal Biotech Announces Update on Development Plans for Oncology Program KB707 and Prioritization of Inhaled KB707 for the Treatment of Non-Small Cell Lung Cancer
Krystal Biotech (NASDAQ: KRYS) announced a strategic update for its oncology program KB707, focusing on the inhaled delivery method for treating non-small cell lung cancer (NSCLC). The company has secured an FDA End of Phase 2 meeting in October to discuss potential registration pathways for inhaled KB707.
The decision follows promising clinical results presented at ASCO 2025, showing an objective response rate of 36% in heavily pre-treated NSCLC patients. The treatment demonstrated durability with median response and progression-free survival not yet reached. Importantly, inhaled KB707 showed a favorable safety profile with no Grade 4 or 5 adverse events.
As part of this prioritization, Krystal has paused enrollment in the OPAL-1 study for intratumoral KB707 while continuing the KYANITE-1 study for inhaled KB707 in lung tumors.
Krystal Biotech (NASDAQ: KRYS) ha comunicato un aggiornamento strategico sul programma oncologico KB707, puntando sulla somministrazione per via inalatoria per il trattamento del carcinoma polmonare non a piccole cellule (NSCLC). La societ脿 ha ottenuto un meeting con la FDA di fine Fase 2 previsto per ottobre, durante il quale discuter脿 possibili percorsi di registrazione per KB707 inalato.
La scelta segue risultati clinici promettenti presentati all'ASCO 2025, che hanno mostrato un tasso di risposta obiettiva del 36% in pazienti con NSCLC fortemente pretrattati. Il trattamento ha mostrato durata delle risposte, con mediana della risposta e della sopravvivenza libera da progressione non ancora raggiunte. Di rilievo, KB707 inalato ha evidenziato un profilo di sicurezza favorevole, senza eventi avversi di grado 4 o 5.
Come parte della riorganizzazione delle priorit脿, Krystal ha sospeso il reclutamento nello studio OPAL-1 per KB707 intratumorale, mantenendo invece attivo lo studio KYANITE-1 per KB707 inalato nei tumori polmonari.
Krystal Biotech (NASDAQ: KRYS) anunci贸 una actualizaci贸n estrat茅gica de su programa oncol贸gico KB707, centr谩ndose en la administraci贸n inhalada para el tratamiento del c谩ncer de pulm贸n no microc铆tico (NSCLC). La compa帽铆a ha conseguido una reuni贸n con la FDA de fin de Fase 2 en octubre para debatir posibles v铆as de registro para KB707 inhalado.
La decisi贸n sigue a resultados cl铆nicos prometedores presentados en ASCO 2025, que mostraron una tasa de respuesta objetiva del 36% en pacientes con NSCLC fuertemente pretratados. El tratamiento demostr贸 durabilidad, ya que la mediana de la duraci贸n de la respuesta y de la supervivencia libre de progresi贸n a煤n no se ha alcanzado. Importante: KB707 inhalado present贸 un perfil de seguridad favorable, sin eventos adversos de grado 4 o 5.
Como parte de esta priorizaci贸n, Krystal ha pausado el reclutamiento en el estudio OPAL-1 para KB707 intratumoral, mientras contin煤a el estudio KYANITE-1 para KB707 inhalado en tumores pulmonares.
Krystal Biotech (NASDAQ: KRYS)電� 膦呾枒頃� 頂勲攴鸽灗 KB707鞐� 雽�頃� 鞝勲灥鞝� 鞐呺嵃鞚错姼毳� 氚滍憸頃橂┌ 牍勳唽靹疙彫韽愳晹(NSCLC) 旃橂毳� 鞙勴暅 頋§瀰 韴棳 氚╈嫕鞐� 歆戩頃滊嫟瓿� 氚濏様鞀惦媹雼�. 須岇偓電� 頋§瀰順� KB707鞚� 鞀轨澑 瓴诫 臧�電レ劚鞚� 雲检潣頃橁赴 鞙勴暣 10鞗� FDA End of Phase 2 氙疙寘鞚� 頇曤炒頄堨姷雼堧嫟.
鞚措矆 瓴办爼鞚� ASCO 2025鞐愳劀 氚滍憸霅� 鞙犽頃� 鞛勳儊 瓴瓣臣毳� 霐半ジ 瓴冹溂搿�, 雼ろ殞 旃橂毳� 氚涭潃 NSCLC 頇橃瀽鞐愳劀 臧濌磤鞝� 氚橃潙毳� 36%鞚� 氤挫榾鞀惦媹雼�. 旃橂 須臣電� 歆�靻嶌劚鞚� 氤挫榾鞙茧┌ 氚橃潙 欷戩暀臧掙臣 氍挫頄夓儩臁搓赴臧� 欷戩暀臧掛潃 鞎勳 霃勲嫭頃橃 鞎婌晿鞀惦媹雼�. 韸鬼瀳 頋§瀰順� KB707電� 4旮� 霕愲姅 5旮� 鞚挫儊 欷戨寑頃� 鞚挫儊氚橃潙鞚� 鞐嗢柎 鞎堨爠靹� 頂勲韺岇澕鞚� 鞏戫樃頄堨姷雼堧嫟.
鞖办劆靾滌渼 臁办爼鞚� 鞚柬櫂鞙茧 Krystal鞚� 膦呾枒雮� 韴棳 KB707鞚� OPAL-1 鞐瓣惮 霌彪鞚� 鞚检嫓 欷戨嫧頄堨溂氅�, 韽� 膦呾枒 雽�靸� 頋§瀰順� KB707鞚� KYANITE-1 鞐瓣惮電� 瓿勳啀 歆勴枆頃╇媹雼�.
Krystal Biotech (NASDAQ: KRYS) a annonc茅 une mise 脿 jour strat茅gique de son programme oncologique KB707, en privil茅giant l'administration par inhalation pour le traitement du cancer du poumon non 脿 petites cellules (NSCLC). La soci茅t茅 a obtenu une r茅union FDA End of Phase 2 en octobre pour discuter des voies d'enregistrement possibles pour KB707 inhal茅.
Cette d茅cision fait suite 脿 des r茅sultats cliniques prometteurs pr茅sent茅s 脿 l'ASCO 2025, montrant un taux de r茅ponse objective de 36% chez des patients NSCLC fortement pr茅trait茅s. Le traitement a montr茅 une bonne durabilit茅, la m茅diane de la r茅ponse et de la survie sans progression n'ayant pas encore 茅t茅 atteintes. Fait notable : KB707 inhal茅 a pr茅sent茅 un profil de s茅curit茅 favorable, sans 茅v茅nements ind茅sirables de grade 4 ou 5.
Dans le cadre de cette priorisation, Krystal a suspendu l'inclusion dans l'茅tude OPAL-1 pour KB707 intratumoral, tout en poursuivant l'茅tude KYANITE-1 pour KB707 inhal茅 dans les tumeurs pulmonaires.
Krystal Biotech (NASDAQ: KRYS) gab ein strategisches Update zu seinem Onkologieprogramm KB707 bekannt und fokussiert dabei die inhalative Applikationsform zur Behandlung von
Die Entscheidung folgt auf vielversprechende klinische Daten, die auf der ASCO 2025 pr盲sentiert wurden und eine objektive Ansprechrate von 36% bei stark vorbehandelten NSCLC-Patienten zeigten. Die Behandlung zeigte eine anhaltende Wirksamkeit: Medianwerte f眉r Ansprechdauer und progressionsfreies 脺berleben sind noch nicht erreicht. Wichtig ist, dass inhalatives KB707 ein g眉nstiges Sicherheitsprofil ohne Grad-4- oder 5-Nebenwirkungen aufwies.
Im Zuge der Priorisierung hat Krystal die Rekrutierung der OPAL-1-Studie f眉r intratumorales KB707 ausgesetzt, w盲hrend die KYANITE-1-Studie f眉r inhalatives KB707 bei Lungentumoren fortgesetzt wird.
- Strong efficacy with 36% objective response rate in heavily pre-treated NSCLC patients
- Favorable safety profile with no Grade 4 or 5 adverse events reported
- FDA granted End of Phase 2 meeting to discuss registration pathways
- Treatment shows potential for outpatient administration
- Paused enrollment in OPAL-1 study for intratumoral KB707
- Median duration of response and progression free survival data not yet mature
Insights
Krystal's inhaled KB707 shows promising 36% response rate in heavily pre-treated lung cancer patients, advancing toward potential FDA registration.
Krystal Biotech is strategically prioritizing its inhaled KB707 immunotherapy program for non-small cell lung cancer (NSCLC) following encouraging early efficacy data. The therapy has demonstrated an
The company has secured an End of Phase 2 meeting with the FDA in October to discuss potential registration pathways, signaling accelerated development. The safety profile appears favorable with no Grade 4 or 5 adverse events reported, and the therapy can be administered in outpatient settings鈥攁 significant advantage for cancer patients.
What's particularly notable is the mechanism of action. By delivering IL-2 and IL-12 directly to lung tumors via inhalation, KB707 potentially achieves higher local concentration of these powerful immune stimulators while minimizing systemic toxicity鈥攁 common limitation of systemic immunotherapies. This localized approach could represent a meaningful advance in the treatment paradigm for NSCLC patients who have limited options after failing standard therapies.
The company's decision to pause enrollment in the OPAL-1 study (intratumoral delivery) to focus resources on the inhaled formulation indicates strong internal confidence in the inhaled approach and suggests a strategic prioritization of their pipeline based on clinical evidence rather than spreading resources across multiple delivery methods.
PITTSBURGH, Aug. 21, 2025 (GLOBE NEWSWIRE) -- (the 鈥淐ompany鈥�) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced an update on development plans for KB707, the Company鈥檚 redosable immunotherapy designed to drive sustained, localized expression of interleukin-2 (IL-2) and interleukin-12 (IL-12) in the tumor microenvironment and promote systemic immune-mediated tumor clearance following inhaled or intratumoral delivery to solid tumors.
Based on promising early evidence of efficacy in the treatment of non-small cell lung cancer (NSCLC), the Company was granted an End of Phase 2 meeting with the United States Food and Drug Administration (FDA) in October to discuss potential development pathways to support the registration of inhaled KB707.
鈥淭he acceleration of our work on inhaled KB707 is a reflection of both the clear and acute unmet need that exists for new treatments of NSCLC and the promising efficacy profile we have observed to date with inhaled KB707,鈥� said Suma Krishnan, President of Research and Development of聽Krystal Biotech. 鈥淲e look forward to meeting with the FDA and bringing another urgently needed therapeutic option to patients.鈥�
Evidence supporting the prioritization of inhaled KB707, including monotherapy activity and durable responses in heavily pre-treated patients with NSCLC, was by the Company at the 2025聽American Society of Clinical Oncology Annual Meeting聽earlier this year. As of data cut-off of April 15, 2025, the objective response rate in this heavily pre-treated NSCLC patient cohort was
Enrollment continues in the Company鈥檚 KYANITE-1 study, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating inhaled KB707 in patients with locally advanced or metastatic solid tumors of the lung. Details of the study can be found at under NCT identifier NCT06228326.
With the prioritization of inhaled KB707, the Company has paused enrollment in OPAL-1, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancies. Patients on OPAL-1 continue to be followed and based on safety and efficacy results from OPAL-1, the Company may adjust development plans for intratumoral KB707. Details of the study can be found at under NCT identifier NCT05970497.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK庐, the Company鈥檚 first commercial product, is the first-ever redosable gene therapy, and the first genetic medicine approved in the United States, Europe, and Japan 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 , and follow @KrystalBiotech on and (formerly Twitter).
Forward-Looking Statements
This press release contains 鈥渇orward looking鈥� statements within the meaning of the Private Securities Litigation Reform Act of 1995 based on the Company鈥檚 current expectations and beliefs regarding its KB707 program. All statements other than historical facts are or may be deemed to be forward-looking statements and involve known and unknown risks, uncertainties, and assumptions that could cause actual results to differ materially from those indicated by such forward-looking statements as a result of various important factors set forth under the caption 鈥淩isk Factors鈥� in the Company鈥檚 annual and quarterly reports on file with the U.S. Securities and Exchange Commission. The Company provides this information as of the date of this release and assumes no obligation to update any forward-looking statements.
CONTACT
Investors and Media:
St茅phane Paquette, PhD
Krystal Biotech
[email protected]
FAQ
What are the clinical results of Krystal Biotech's KB707 for NSCLC treatment?
Why did KRYS prioritize the inhaled version of KB707 over intratumoral delivery?
What is the current status of Krystal Biotech's KYANITE-1 study?
What happened to Krystal Biotech's OPAL-1 trial for KB707?
How does Krystal Biotech's KB707 work in cancer treatment?
