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Phio Pharmaceuticals Announces Pathology Results for Four of Five Patients in Fourth Cohort

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Phio Pharmaceuticals (NASDAQ: PHIO) has announced pathology results for four out of five patients in the fourth cohort of its Phase 1b clinical trial for PH-762. The trial evaluates the intratumoral use of PH-762 in various skin cancers. Key results include a complete pathologic response in one cutaneous squamous cell carcinoma (cSCC) patient and a partial response in a Stage 4 metastatic Merkel cell patient.

Across all four cohorts, 15 patients have been treated with no dose-limiting toxicities or clinically relevant adverse effects. Among 13 cSCC patients, five achieved complete response, one near-complete response, one partial response, and six non-responses. The company is now enrolling what is expected to be the final cohort in the Phase 1b study.

Phio Pharmaceuticals (NASDAQ: PHIO) ha annunciato i risultati patologici di quattro pazienti su cinque nel quarto gruppo della sua sperimentazione clinica di fase 1b per PH-762. Lo studio valuta l'uso intratumorale di PH-762 in diversi tipi di tumori cutanei. I risultati principali includono una risposta patologica completa in un paziente con carcinoma squamoso cutaneo (cSCC) e una risposta parziale in un paziente con carcinoma a cellule di Merkel metastatico di stadio 4.

Nel complesso, in tutti e quattro i gruppi, sono stati trattati 15 pazienti senza tossicità dose-limitante o effetti avversi clinicamente rilevanti. Tra 13 pazienti con cSCC, cinque hanno ottenuto una risposta completa, uno una risposta quasi completa, uno una risposta parziale e sei nessuna risposta. L'azienda sta ora reclutando quello che si prevede sarà l'ultimo gruppo nello studio di fase 1b.

Phio Pharmaceuticals (NASDAQ: PHIO) ha anunciado los resultados patológicos de cuatro de cinco pacientes en la cuarta cohorte de su ensayo clínico de fase 1b para PH-762. El estudio evalúa el uso intratumoral de PH-762 en varios tipos de cáncer de piel. Los resultados clave incluyen una respuesta patológica completa en un paciente con carcinoma cutáneo de células escamosas (cSCC) y una respuesta parcial en un paciente con carcinoma de células de Merkel metastásico en estadio 4.

En las cuatro cohortes, se han tratado 15 pacientes sin toxicidades limitantes de dosis ni efectos adversos clínicamente relevantes. De 13 pacientes con cSCC, cinco lograron respuesta completa, uno casi completa, uno respuesta parcial y seis no respondieron. La compañía está actualmente inscribiendo lo que se espera sea la cohorte final del estudio de fase 1b.

Phio Pharmaceuticals (NASDAQ: PHIO)� PH-762� 4번째 코호트에 속한 5� � 4명의 병리 결과� 발표했습니다. � 임상 1b� 시험은 다양� 피부암에 대� PH-762� 종양 � 투여� 평가합니�. 주요 결과로는 피부 편평세포�(cSCC) 환자 1명에� 완전 병리학적 반응4� 전이� 머클 세포� 환자 1명에� 부� 반응� 있었습니�.

� � 코호� 전체에서 15명의 환자가 치료� 받았으며, 용량 제한 독성이나 임상적으� 의미 있는 부작용은 없었습니�. 13명의 cSCC 환자 � 5명은 완전 반응� 보였�, 1명은 거의 완전 반응, 1명은 부� 반응, 6명은 반응� 없었습니�. 회사� 현재 1b� 연구� 마지� 코호트로 예상되는 환자 모집� 진행 중입니다.

Phio Pharmaceuticals (NASDAQ : PHIO) a annoncé les résultats pathologiques de quatre patients sur cinq dans la quatrième cohorte de son essai clinique de phase 1b pour PH-762. L’étude évalue l’utilisation intratumorale de PH-762 dans divers cancers de la peau. Les résultats clés incluent une réponse pathologique complète chez un patient atteint d’un carcinome épidermoïde cutané (cSCC) et une réponse partielle chez un patient atteint d’un carcinome à cellules de Merkel métastatique de stade 4.

Au total, 15 patients ont été traités dans les quatre cohortes, sans toxicité limitant la dose ni effets indésirables cliniquement significatifs. Parmi 13 patients atteints de cSCC, cinq ont obtenu une réponse complète, un une réponse quasi complète, un une réponse partielle et six aucune réponse. L’entreprise recrute désormais ce qui devrait être la dernière cohorte de l’étude de phase 1b.

Phio Pharmaceuticals (NASDAQ: PHIO) hat die pathologischen Ergebnisse von vier von fünf Patienten der vierten Kohorte seiner Phase-1b-Studie für PH-762 bekanntgegeben. Die Studie bewertet die intratumorale Anwendung von PH-762 bei verschiedenen Hautkrebsarten. Zu den wichtigsten Ergebnissen gehören eine vollständige pathologische Remission bei einem Patienten mit kutanem Plattenepithelkarzinom (cSCC) und eine partielle Remission bei einem Patienten mit metastasiertem Merkelzellkarzinom im Stadium 4.

In allen vier Kohorten wurden 15 Patienten behandelt, ohne dosislimitierende Toxizitäten oder klinisch relevante Nebenwirkungen. Von 13 cSCC-Patienten erreichten fünf eine vollständige Remission, einer eine nahezu vollständige Remission, einer eine partielle Remission und sechs keine Anspreche. Das Unternehmen rekrutiert nun die voraussichtlich letzte Kohorte der Phase-1b-Studie.

Positive
  • Complete pathologic response (100% clearance) achieved in one cSCC patient in fourth cohort
  • No dose-limiting toxicities or clinically relevant adverse effects reported across all cohorts
  • Strong overall response rate with 5 complete responses and 2 near-complete/partial responses out of 13 cSCC patients
  • PH-762 well tolerated across all dose escalation cohorts with no disease progression
Negative
  • Two cSCC patients in fourth cohort showed non-response (<50% tumor clearance)
  • Melanoma patient was a non-responder (<50% clearance)
  • Six out of 13 cSCC patients showed pathologic non-response

Insights

Phio reports mixed but promising Phase 1b results for PH-762, with complete response in one cSCC patient and partial response in Stage 4 Merkel cell patient.

Phio Pharmaceuticals has announced pathology results from the fourth cohort of its Phase 1b trial evaluating PH-762, an siRNA therapeutic using their INTASYL® technology. The results show one complete pathologic response (100% tumor clearance) in a cutaneous squamous cell carcinoma (cSCC) patient and one partial response (>50% clearance) in a patient with metastatic Merkel cell carcinoma. Two cSCC patients showed non-responses (<50% tumor clearance), with one patient's results still pending.

The cumulative data across all cohorts now includes 5 complete responses, 1 near-complete response (>90% clearance), and 2 partial responses among the 15 treated patients. Importantly, no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects were reported, and no disease progression was observed in any patient.

These results are particularly significant because they demonstrate potential efficacy in multiple skin cancer types. The complete response rate of approximately 38% (5/13) in cSCC patients suggests meaningful clinical activity. The partial response in the Stage 4 metastatic Merkel cell patient is especially noteworthy, as metastatic disease is typically more difficult to treat than localized tumors.

The company is now enrolling what they expect to be the final cohort in this study. If safety and efficacy continue to be demonstrated, PH-762 could potentially offer a non-surgical alternative for skin cancer patients, addressing a significant unmet need in this growing market.

Complete Pathologic Response in patient with cutaneous squamous cell carcinoma

Partial Response in Stage 4 metastatic Merkel cell patient

King of Prussia, Pennsylvania--(Newsfile Corp. - July 25, 2025) - Phio Pharmaceuticals Corp. (NASDAQ: PHIO) is a clinical-stage siRNA biopharmaceutical company developing therapeutics using its proprietary INTASYL® gene silencing technology to eliminate cancer.

Phio announced today that pathologic results are available for four of the five patients treated in the fourth cohort. A complete pathologic response (100% tumor clearance) has been reported for one patient with cutaneous squamous cell carcinoma (cSCC). One patient with metastatic Merkel cell carcinoma was reported as a partial response (> 50% clearance). Two patients with cSCC were reported as having a pathologic non-response (<50% tumor clearance). Pathologic results for the remaining patient with cSCC are still pending.

Phio's ongoing Phase 1b dose escalation clinical trial (NCT 06014086) is designed to evaluate the safety and tolerability of neoadjuvant use of intratumoral PH-762 in Stages 1, 2 and 4 cSCC, Stage 4 melanoma, and Stage 4 Merkel cell carcinoma. To date, a total of 15 patients with cutaneous carcinomas have been treated across the four cohorts. There were no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects in the patients receiving intratumoral PH-762 in this trial. Moreover, PH-762 has been well tolerated in all enrolled patients in each escalating dose cohort. No patients exhibited clinical progression of disease.

The cumulative pathologic response in 13 patients with cSCC include five complete response (100% clearance), one patient with a near complete response (>90% clearance) and one with a partial response (>50% clearance) and six patients with a pathologic non-response (< 50% clearance).

The one Merkel cell carcinoma patient with stage 4 metastatic disease had a partial response (>50% clearance). The melanoma patient was a non-responder (<50% clearance).

Phio is now enrolling what is expected to be the 5th and final cohort in the Phase 1b study.

"The positive safety and efficacy outcomes through the fourth cohort continue to indicate that PH-762 may present a viable non-surgical alternative in this large and continually expanding skin cancer market," said Robert Bitterman, CEO and Chairman of Phio Pharmaceuticals.

About Phio Pharmaceuticals Corp.

Phio Pharmaceuticals Corp. (NASDAQ: PHIO) is a clinical-stage siRNA biopharmaceutical company advancing its INTASYL® gene silencing technology focused on immuno-oncology therapeutics. Phio's INTASYL compounds are designed to enhance the body's immune cells to more effectively kill cancer cells. Phio's lead clinical program is an INTASYL compound, PH-762, that silences the PD-1 gene implicated in various forms of skin cancer. The on-going Phase 1b trial (NCT# 06014086) is evaluating PH-762 for the treatment of cutaneous squamous cell carcinoma, melanoma and Merkel cell carcinoma. PH-762 is a potential non-surgical treatment for skin cancers.

For additional information, visit the Company's website, .

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as "intends," "believes," "anticipates," "indicates," "plans," "expects," "suggests," "may," "would," "should," "potential," "designed to," "will," "ongoing," "estimate," "forecast," "target," "predict," "could" and similar references, although not all forward-looking statements contain these words. Examples of forward-looking statements contained in this press release include, among others, the possibility that our INTASYL® siRNA gene silencing technology will make the body's immune cells more effective in killing cancer cells and statements regarding our commercial and clinical strategy, development plans and timelines and other future events.  

These statements are based only on our current beliefs, expectations and assumptions and are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control.  Our actual results may differ materially from those indicated in the forward-looking statements as a result of a number of important factors, including, but not limited to, the impact to our business and operations by inflationary pressures, rising interest rates, recession fears, the development of our product candidates, results from our preclinical and clinical activities, our ability to execute on business strategies, our ability to develop our product candidates with collaboration partners, and the success of any such collaborations, the timeline and duration for advancing our product candidates into clinical development, the timing or likelihood of regulatory filings and approvals, the success of our efforts to commercialize our product candidates if approved, our ability to manufacture and supply our product candidates for clinical activities, and for commercial use if approved, the scope of protection we are able to establish and maintain for intellectual property rights covering our technology platform, our ability to obtain future financing, market and other conditions and those identified in our Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q under the caption "Risk Factors" and in other filings the Company periodically makes with the SEC. Readers are urged to review these risk factors and to not act in reliance on any forward-looking statements, as actual results may differ from those contemplated by our forward-looking statements. Phio does not undertake to update forward-looking statements to reflect a change in its views, events or circumstances that occur after the date of this release, except as required by law.  

Contact:
Phio Pharmaceuticals Corp. 
Jennifer Phillips: [email protected]  
Corporate Affairs  

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FAQ

What are the latest Phase 1b trial results for Phio Pharmaceuticals (NASDAQ: PHIO)?

In the fourth cohort, one cSCC patient achieved complete response (100% clearance), one Merkel cell patient showed partial response (>50% clearance), and two cSCC patients were non-responders (<50% clearance).

How many patients have been treated with PH-762 in Phio's clinical trial?

A total of 15 patients with cutaneous carcinomas have been treated across four cohorts, with no dose-limiting toxicities or clinically relevant adverse effects reported.

What is the overall response rate for cSCC patients in Phio's PH-762 trial?

Among 13 cSCC patients, 5 achieved complete response (100% clearance), 1 had near-complete response (>90% clearance), 1 showed partial response (>50% clearance), and 6 were non-responders.

What are the safety results of PH-762 in Phio's clinical trial?

PH-762 has been well-tolerated across all dose escalation cohorts with no dose-limiting toxicities, no clinically relevant treatment-emergent adverse effects, and no disease progression.

What is the next step for Phio Pharmaceuticals' PH-762 trial?

Phio is currently enrolling patients in what is expected to be the 5th and final cohort of the Phase 1b study.
Phio Pharmaceuticals Corp

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