ELOX — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-05

• We are a clinical-stage biopharmaceutical company developing novel, small-molecule product candidates designed to modulate the ribosome and promote readthrough of premature stop codons induced by nonsense mutations (“NMs”) to enable the production of full-length proteins. Targeting ribosome subunits…

• Between January 2023 and April 2024, we conducted a proof-of-concept Phase 2a open-label trial in the United Kingdom in three patients with autosomal recessive Alport Syndrome (“AS”) and a NM in the COL4A4 gene. The primary endpoint of this study was to assess the number of participants with adverse…

• Our pipeline also includes a preclinical program evaluating exaluren for the treatment of autosomal dominant polycystic kidney disease (“ADPKD”) in patients that have NMs (“nmADPKD”). ADPKD is the most common monogenic kidney disease based on genetic diagnosis, affecting approximately 160,000 to 200…

• We have also exclusively licensed ZKN-013, an oral ribosome modulating agent (“RMA”) with structural similarity to azithromycin that induces PTC readthrough to Almirall. In March 2024, we entered into an exclusive global rights agreement with Almirall (the “Almirall License Agreement”) for Almirall …

• indications. Through the Almirall License Agreement, Almirall is developing ZKN-013 for the treatment of recessive dystrophic epidermolysis bullosa (“RDEB”) and junctional epidermolysis bullosa (“JEB”) with NMs. RDEB and JEB are rare skin diseases characterized by mutations in the Collagen VII (RDEB…

Text removed vs the prior filing · source: 10-Q · 2023-11-13

• We are a clinical-stage biopharmaceutical company engaged in the science of ribosome modulation, leveraging both our innovative TURBO-ZM™ chemistry technology platform and our library of novel aminoglycosides to develop novel oral small molecule Ribosome Modulating Agents (“RMAs”) and Eukaryotic Rib…

• We have multiple programs in our pipeline including a clinical program for the treatment of Alport syndrome with ELX-02 (subcutaneous delivery), preclinical programs in investigational new drug ("IND") enabling stages in Recessive Dystrophic (“RDEB”) and Junctional Epidermolysis Bullosa (“JEB”) and …

• In March 2022, we announced our decision to advance ELX-02 delivered subcutaneously for the restoration of Collagen IV protein for the treatment of nonsense mutation Alport syndrome patients. This is a genetic disorder that occurs in 1 in 50,000 newborns in the United States with an estimated preval…

• In November 2022, we began a Phase 2 monotherapy clinical trial for ELX-02 for the potential treatment of ELX-02 in nonsense mutation Alport syndrome. This Phase 2 trial included Alport syndrome patients with nonsense mutations in the COL4 gene. Three patients were dosed with ELX-02 for two months w…

• All three patients treated were 19 years or younger and had Autosomal Recessive Alport syndrome with a nonsense mutation on one allele and a pathogenic missense mutation on the second allele with complete loss of function. The loss of collagen 4 functional protein was confirmed with immunofluorescen…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• The Company identified the following material weakness in its internal control over financial reporting during the year ended December 31, 2025, which had not been remediated as of March 31, 2026:

• The material weakness resulted from inconsistent execution of certain financial reporting controls, primarily within the financial close and reconciliation processes. Specifically, controls related to timely preparation and review of account reconciliations and period-end review procedures were not …

• Notwithstanding the material weakness, management believes the unaudited condensed consolidated financial statements in this report present fairly, in all material respects, our financial position, results of operations, and cash flows in conformity with U.S. GAAP.

• Management is fully committed to addressing the material weakness in our internal controls and has implemented several key initiatives to strengthen them. During the quarter ended March 31, 2026, management continued to execute on its remediation plan. Remediation activities during the quarter inclu…

•  Increasing accounting and financial reporting resources through the addition of qualified personnel and the continued use of external advisors;

Text removed vs the prior filing · source: 10-Q · 2023-11-13

• There were no changes in our internal controls over financial reporting (as defined in Rules 13a-15(f) and 15(d)-15(f) under the Exchange Act) during the quarter ended September 30, 2023 that have materially affected, or are reasonably likely to materially affect, our internal control over financial…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• As a smaller reporting company, we are not required to provide the information required by this item. Please see the “Risk Factors” section in Part I, Item 1A in our Annual Report on Form 10-K for the year ended December 31, 2025.

Text removed vs the prior filing · source: 10-Q · 2023-11-13

• Investing in our common stock involves a high degree of risk. You should carefully consider the risks and uncertainties described below, together with all of the other information in this Quarterly Report on Form 10-Q, before you decide to purchase our common stock. If any of the possible adverse ev…

• Risks Related to Our Financial Position and Need for Additional Capital

• Our recurring losses from operations raise substantial doubt regarding our ability to continue as a going concern.

• For the nine months ended September 30, 2023, our net losses were $14.2 million and as of September 30, 2023, we had an accumulated deficit of $288.6 million. We anticipate operating losses to continue for the foreseeable future due to, among other things, costs related to research, development of o…

• We are exploring various sources of funding such as strategic collaborations and the issuance of equity to fund our operations. If we raise additional funds through strategic collaborations and alliances, which may include existing collaboration partners, we may have to relinquish valuable rights to…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• During the three months ended March 31, 2026, no director or “officer” (as defined in Rule 16a-1(f) under the Exchange Act) of the Company adopted or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” as each term is defined in Item 408(a) of Regulation S-K.

Text removed vs the prior filing · source: 10-Q · 2023-11-13

• On November 10, 2023, the Company entered into the Third Amendment (the “Third Amendment”) to the Hercules Term Loan Agreement which provides for a temporary reduction in the minimum qualified cash balance amount to $2.25 million for the period from November 15, 2023 through December 15, 2023, unles…

• The foregoing description of the Third Amendment is qualified in its entirety by reference to the full text of the Third Amendment, a copy of which is filed as Exhibit 10.5 to this Quarterly Report on Form 10-Q and is incorporated herein by reference.

• During the three months ended September 30, 2023, no director or “officer” (as defined in Rule 16a-1(f) under the Exchange Act) of the Company adopted or terminated a “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” as each term is defined in Item 408(a) of Regulation S-K.