10-K: Phio Pharma's 2025 10-K: PH-762 Progress, Funding Secured

Phio Pharmaceuticals reports promising early clinical data for PH-762 and significant capital raises in 2025, extending its operational runway.

Summary

• Phio Pharmaceuticals Corp. is a clinical-stage biopharmaceutical company focused on immuno-oncology using its proprietary INTASYL RNAi gene silencing technology.
• The lead product candidate, PH-762, is being evaluated in a U.S. multi-center Phase 1b dose-escalating clinical trial for cutaneous squamous cell carcinoma (cSCC), melanoma, and Merkel cell carcinoma.
• The PH-762 trial was fully enrolled in November 2025 with a total of 22 patients.
• Revised data for cSCC patients showed an overall response rate of 65%, including 9 patients with complete response (100% clearance), 2 with major/near clear response (>90% clearance), and 2 with partial response (>50% clearance) among 20 cSCC patients.
• PH-762 was well tolerated in all enrolled patients, with no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects.
• Development of PH-894, an INTASYL compound targeting BRD4, has been deferred due to the reprioritization of advancing the PH-762 clinical trial.
• The company raised $20.6 million in net proceeds from multiple financings in 2025 (January, July, and November).
• Net loss increased to $8.7 million in 2025 from $7.2 million in 2024.
• Research and development expenses increased by 27% to $4.6 million in 2025, primarily due to PH-762 clinical study advancement.
• General and administrative expenses increased by 23% to $4.6 million in 2025.
• Cash and cash equivalents stood at $21.0 million as of December 31, 2025, up from $5.4 million at December 31, 2024.
• Management believes the company has sufficient cash to meet its current planned obligations for at least 12 months from the financial statements' issuance date (March 5, 2026).
• A 1-for-9 reverse stock split of outstanding Common Stock was completed on July 5, 2024.

Sentiment

Score: 7

Explanation: StockSavvy.ai views this as a moderately positive report, driven by promising early clinical data for PH-762 and successful capital raises, which provide a runway for continued development despite ongoing losses and the deferral of PH-894.

Positives

• PH-762 Phase 1b trial for cutaneous squamous cell carcinoma (cSCC) showed a 65% overall response rate, with 9 patients achieving complete response (100% clearance).
• PH-762 demonstrated a favorable safety profile in clinical trials, with no dose-limiting toxicities or clinically relevant treatment-emergent adverse effects.
• Successful capital raises in 2025 generated $20.6 million in net proceeds, significantly improving the cash and cash equivalents position to $21.0 million.
• Management projects sufficient cash and cash equivalents to meet planned obligations for at least 12 months.
• A comprehensive drug substance development services agreement was entered into with a U.S. manufacturer for cGMP manufacture of PH-762, supporting future clinical development.
• Preclinical studies indicate INTASYL compounds can enhance the activity of adoptive cell therapies and potentially increase addressable drug targets when combined with antibodies.

Negatives

• Net loss increased to $8.7 million in 2025 from $7.2 million in 2024.
• Research and development expenses increased by 27% and general and administrative expenses increased by 23% in 2025.
• The IND submission for PH-894 has been deferred due to the reprioritization of PH-762 clinical trials.
• The company has a history of recurring net losses and has not generated any product revenue, expecting to continue incurring losses for the foreseeable future.
• Future financing may involve the issuance of debt or equity, which could dilute current stockholders' ownership or impose restrictive covenants.
• The company's ability to use net operating loss carryforwards and other tax benefits may be limited due to past and potential future ownership changes.

Risks

• Dependence on the success of the unproven INTASYL technology and early-stage product candidates, which may never lead to approved and marketable products.
• Potential for significant delays or failure in clinical development, or inability to identify or discover additional product candidates.
• Disruptions at the FDA, including workforce reductions or inadequate funding, could prevent the FDA from performing normal functions, negatively impacting the business.
• Delays or difficulties in identifying and enrolling patients in clinical trials, leading to delays in generating clinical data and regulatory approvals.
• Topline or interim data from clinical trials may not accurately reflect or may materially differ from the complete results.
• Reliance on third parties for the manufacture of clinical supply for product candidates, posing risks of supply interruption, quality issues, or increased costs.
• Business and operations would suffer in the event of a cybersecurity incident, leading to potential regulatory scrutiny, litigation, operational interruptions, and reputational damage.
• Dependence on owned patents and licensed technologies; failure to maintain patents or loss of licensing rights would harm the ability to develop new products.
• Requirement for substantial additional funds to complete research and development activities, with no assurance that additional financing will be available on acceptable terms or at all.
• Future financing through the issuance of debt or equity may have an adverse effect on stockholders (e.g., dilution) or otherwise adversely affect the business.
• Inability to maintain compliance with the continued listing requirements of The Nasdaq Capital Market, potentially leading to delisting.
• Significant competition in the biotechnology and pharmaceutical industries from companies with greater resources and experience.
• Potential liabilities from clinical testing and future product liability claims, which may not be adequately covered by insurance.
• Uncertainty regarding the protection of intellectual property rights and potential claims of infringement by third parties.

Future Outlook

The company targets an FDA submission in Q2 2026 to propose and seek guidance for next steps in PH-762 clinical study design. Safety data through an extended follow-up period for PH-762 is also expected to be reported in Q2 2026. The company expects to incur significant operating losses for the foreseeable future and will need to raise additional capital until profitability is achieved, which may never occur.

Management Comments

• "We are committed to discovering and developing innovative cancer treatments for patients by creating new pathways toward a cancer-free future."
• "We believe that our INTASYL technology provides the following benefits including, but not limited to: Ability to target a broad range of cell types and tissues; Ability to target both intracellular and extracellular protein targets; Efficient uptake by target cells, avoiding the need for assisted delivery; Ability to target multiple genes in one drug product; Promising clinical efficacy and pathology results in cutaneous squamous cell carcinoma (cSCC) with local administration; Favorable clinical safety profile; and Readily manufactured under current good manufacturing practices."
• "We believe these data further support the potential for PH-762 to provide a strong local immune response without the dose immune-related adverse effects seen with systemic antibody therapy."
• "These preclinical data indicate that PH-894 can reprogram T cells and other cells in the tumor microenvironment to provide enhanced immunotherapeutic activity."
• "Based on current cash flow projections, we believe we have sufficient cash and cash equivalents, including net proceeds from our January 2025 Offerings, July 2025 Financing and November 2025 Financing, to meet our current planned obligations for at least 12 months from the date these financial statements are issued."

Industry Context

StockSavvy.ai notes that Phio Pharmaceuticals operates in the highly competitive and rapidly evolving immuno-oncology field, where many larger pharmaceutical and biotechnology companies possess greater financial and human capital resources. The focus on RNA interference (RNAi) and self-delivering siRNA technology (INTASYL) represents a novel approach to drug development, particularly for "undruggable" targets, which could differentiate Phio if successful. However, the early stage of development for its product candidates, especially with the deferral of PH-894, highlights the significant R&D risks inherent in this sector.

Comparison to Industry Standards

• The 65% overall response rate for PH-762 in cSCC, including 100% complete responses in 9 patients, is a promising early-stage clinical result, especially given the favorable safety profile. This compares favorably to some early-stage immunotherapy trials, but direct comparisons require more mature data and larger patient cohorts.
• The deferral of PH-894's IND submission is a strategic decision to prioritize PH-762, a common practice for smaller biotechs with limited resources, but it means delaying the advancement of another potentially valuable asset.
• The company's reliance on third-party manufacturing and CROs is standard for clinical-stage biopharmaceutical companies without in-house capabilities, but it introduces supply chain and quality control risks.

Management Changes

Corporate Governance

Legal Proceedings

• The company is not currently a party to any actual or threatened material legal proceedings.

Related Party Transactions

• Intracoastal Capital LLC, a beneficial owner of more than 5% of outstanding shares, participated in the July 2024, January 2025, July 2025, and November 2025 financings, acquiring common stock and warrants.
• Heights Capital Management, Inc. (investment manager to CVI Investments, Inc., a beneficial owner of more than 5% of outstanding shares) participated in the December 2024, January 2025, and November 2025 financings, acquiring common stock and warrants.

Stakeholder Impact

• Shareholders: Experienced dilution from multiple equity financings in 2025, with potential for further dilution. The stock price has been volatile. However, promising early clinical data for PH-762 and extended cash runway offer potential for future value creation.
• Employees: A small core management team, with support for development and cybersecurity training. Stock-based compensation is a component of executive and employee incentives.
• Customers (future patients): The company's focus on developing innovative cancer treatments using INTASYL technology offers potential new therapeutic options if product candidates are successfully developed and commercialized.
• Suppliers/Creditors: The company relies on third-party contract manufacturing organizations and contract research organizations for R&D and clinical supply, indicating ongoing relationships with these partners.

Next Steps

• FDA submission targeted for Q2 2026 to propose and seek guidance for next steps in PH-762 clinical study design.
• Safety data through an extended follow-up period for PH-762 expected to be reported in Q2 2026.
• Conduct a non-clinical toxicology study for PH-762, required by the FDA prior to commencing a human clinical trial for registration purposes.
• Continue to rely on contract manufacturing organizations for product candidates.
• Ongoing evaluation of in-licensed intellectual property and the field for new in-licensing opportunities.
• Assess the need to conduct an ownership change analysis annually to determine limitations on net operating loss or tax credit carryforwards.

Key Dates

Recommendation

The company shows promising early clinical data for PH-762 and has secured significant funding to extend its operational runway. However, it remains a clinical-stage biopharmaceutical company with a history of losses, no product revenue, and substantial future funding requirements. The deferral of PH-894 also indicates a focused but narrowed pipeline in the near term. The positive clinical results and improved liquidity are encouraging, but the inherent risks of drug development and potential for further dilution warrant a cautious "hold" stance for seasoned investors, awaiting further clinical milestones and a clearer path to commercialization.

Keywords

Biopharmaceutical, Immuno-oncology, RNAi, INTASYL, PH-762, PH-894, Clinical Trial, Cancer Treatment, Gene Silencing, Nasdaq, SEC Filing, 10-K, Oncology, Biotechnology, Drug Development, Cybersecurity, Intellectual Property, Capital Raise