ADARx Pharmaceuticals — Third Quarter 2026 Earnings Summary

Financial Performance:

• Reported collaboration revenue of $2.2 million for the quarter ended September 30, 2026, an increase from $1.6 million in the same period in 2025.
• Research and development expenses were $26.7 million for the quarter, up from $19.4 million in the prior year period, driven by increases in external R&D expenses and personnel-related costs.
• General and administrative expenses were $7.7 million for the quarter, compared to $5.1 million in the prior year period, due to higher personnel costs, professional fees, and stock-based compensation.
• Net loss for the quarter was $28.1 million, compared to a net loss of $17.9 million in the same period in 2025.
• As of September 30, 2026, the Company held cash, cash equivalents, and short-term investments of $901.0 million, excluding $62.3 million in net proceeds received on October 1, 2026.
• Total assets increased to $929.1 million as of September 30, 2026, from $507.7 million as of December 31, 2025.
• Total liabilities were $354.7 million as of September 30, 2026, compared to $352.3 million as of December 31, 2025.
• Stockholders' equity turned positive to $574.4 million as of September 30, 2026, from a deficit of $202.3 million as of December 31, 2025.
• Accumulated deficit increased to $(297.2) million as of September 30, 2026, from $(220.7) million as of December 31, 2025.
• Net loss per share (basic and diluted) was $(1.74) for the quarter ended September 30, 2026, compared to $(1.85) in the same period in 2025.

Guidance and Future Outlook:

• The Company expects its existing cash, cash equivalents, short-term investments, and recent IPO proceeds to fund projected operating expenses and capital requirements into 2030.
• Anticipated key milestones include:
  • Agazisiran Phase 2 data for IgAN (mid-2027 for 9-month UPCR reduction; 2H 2027 for 1-year eGFR stabilization), C3G (2H 2027), and PNH (2H 2027).
  • Onvuzosiran Phase 3 STOP-HAE topline data by year-end 2027.
  • ADX-626 Phase 2a/b initiation in secondary stroke prevention in 2027.
  • ADX-077 Phase 1 initiation in obesity in 2027.
  • ADX-199 Phase 1 initiation by year-end 2027 or early 2028.
• Management intends to explore additional dosing regimens in Phase 2 IgAN and PNH clinical trials.

Business Segments and Product Lines:

• Agazisiran: Advanced three Phase 2 clinical trials for complement-mediated diseases in renal diseases (IgA nephropathy, C3G, IC-MPGN), paroxysmal nocturnal hemoglobinuria (PNH), and geographic atrophy (GA) secondary to AMD.
• Onvuzosiran: Received Fast Track designation from the FDA in August 2026 for prophylaxis to prevent attacks of hereditary angioedema (HAE); currently evaluated in the Phase 3 STOP-HAE trial.
• ADX-626: Phase 2 planning underway for secondary stroke prevention (SSP) and exploratory studies in stroke prevention for atrial fibrillation (AFib); higher doses will be evaluated in the planned Phase 2 trial after determining not to evaluate higher doses in the ongoing Phase 1 trial beyond the 400 mg cohort.
• ADX-077 and ADX-199: Advanced preclinical candidates targeting obesity and Alzheimer's disease (APP), respectively, with Phase 1 initiation planned for 2027.
• Collaboration: Revenue recognized under the Collaboration and License Option Agreement with AbbVie based on performance of research and development services.

Market and Competitive Landscape:

• The Company positions its differentiated liver-targeted programs and targeted delivery to extrahepatic tissues to expand the range of diseases addressed by siRNA therapeutics.
• ADARx views its pipeline as a "pipeline-in-a-product" opportunity, particularly regarding agazisiran.

Risks and Challenges:

• Forward-looking statements highlight risks related to the timing, progress, and results of preclinical studies and clinical trials.
• Risks include the ability to obtain regulatory clearances and approvals, reliance on third parties, and capital needs.
• Actual results may differ materially from expectations due to substantial risks and uncertainties.

Management Commentary and Tone:

• Zhen Li, Ph.D., President and CEO, stated the Company has made significant progress across its pipeline, bolstered by the successful IPO and private placement.
• Management expressed confidence in the resources provided by the financings to advance three clinical-stage programs in parallel, bring two advanced preclinical candidates into the clinic, and support additional IND filings.
• The Company sees "tremendous opportunity" to expand the range of diseases siRNA therapeutics can address as the field evolves.

Other Key Points:

• Completed an initial public offering (IPO) and concurrent private placement generating aggregate gross proceeds of approximately $602.5 million.
• The IPO consisted of 30,187,500 shares at $17.00 per share, including 3,937,500 shares issued upon the underwriters' exercise of the over-allotment option; gross proceeds from the IPO were $513.2 million.
• The concurrent private placement with AbbVie involved 5,255,542 shares, generating gross proceeds of $89.3 million.
• Common stock began trading on the Nasdaq Global Select Market under the ticker symbol "ADRX" on September 25, 2026.
• Expanded leadership team with the appointment of Ying Huang, Ph.D. (current K2 Therapeutics CEO) to the Board of Directors and Robert Ackles as Chief People Officer.
• All convertible preferred stock (Series A, B, B-1, and C) was retired as of September 30, 2026, with zero shares issued and outstanding.
• Common stock issued and outstanding increased to 107,255,963 as of September 30, 2026, from 9,659,572 as of December 31, 2025.
• Deferred revenue decreased slightly to $326.4 million total (current and non-current) as of September 30, 2026, from $331.6 million as of December 31, 2025.