DNLI — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-07

• •our ability to successfully market, manufacture, and commercialize AVLAYAH and any other products for which we obtain marketing approval;

• •our ability to successfully conduct in-house manufacturing;

• We currently have one approved product. Our commercial product, AVLAYAHTM (tividenofusp alfa-eknm) received U.S. Food and Drug Administration ("FDA") approval on March 24, 2026 and is approved for the treatment of neurologic manifestations in patients with Hunter syndrome or mucopolysaccharidoses II…

• The approval of AVLAYAH is based on the reduction of a key disease biomarker, cerebrospinal fluid heparan sulfate (CSF HS), as a surrogate endpoint reasonably likely to predict clinical benefit in the treatment of neurologic manifestations of Hunter syndrome. In a Phase 1/2 clinical trial, AVLAYAH d…

• In connection with the approval of AVLAYAH, the FDA granted us a Rare Pediatric Disease Priority Review Voucher ("PRV"). This voucher may be used to obtain priority review for a future marketing application or transferred to another sponsor.

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• •our plans and ability to establish sales, marketing, manufacturing, and distribution infrastructure to commercialize any product candidates for which we obtain approval;

• •our plans and ability to develop our own manufacturing facilities;

• •Tividenofusp alfa (DNL310, ETV:IDS), composed of iduronate-2-sulfatase ("IDS") fused to TV, is designed to deliver IDS into cells and tissues throughout the body, including the brain by crossing the BBB, with the goal of addressing the behavioral, cognitive, and physical manifestations of MPS II (H…

• •DNL126 (ETV:SGSH), composed of N-sulfoglucosamine sulfohydrolase ("SGSH") fused to TV, is designed to deliver SGSH into cells and tissues throughout the body, including the brain by crossing the BBB, with the goal of treating MPS IIIA (Sanfilippo syndrome);

• •TAK-594/DNL593 (PTV:PGRN), composed of PGRN fused to TV, is designed to restore PGRN levels in the brain with the goal of treating frontotemporal dementia ("FTD") associated with a mutation in the granulin ("GRN") gene and is being developed in collaboration with Takeda;

Text added vs the prior filing · source: 10-Q · 2026-05-07

• Conclusions Regarding the Effectiveness of Disclosure Controls and Procedures

• As of March 31, 2026, management, with the participation of our Chief Executive Officer and Chief Operating and Financial Officer, has performed an evaluation of the effectiveness of the design and operation of our disclosure controls and procedures as defined in Rules 13a-15(e) and 15d-15(e) of the…

• Any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objective and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on this evalu…

• Our management, with the participation of our Chief Executive Officer and Chief Operating and Financial Officer, has evaluated any changes in our internal control over financial reporting during the quarter ended March 31, 2026, to identify any change that has materially affected, or is reasonably l…

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• Our management has performed an evaluation of the effectiveness of the design and operation of our disclosure controls and procedures as defined in Rules 13a-15(e) and 15d-15(e) of the Exchange Act. Our disclosure controls and procedures are designed to ensure that information required to be disclos…

• Any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving the desired control objective and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on this evalu…

• There was no change in our internal control over financial reporting identified in connection with the evaluation required by Rule 13a-15(d) and 15d-15(d) of the Exchange Act that occurred during the quarter ended September 30, 2025 that has materially affected, or is reasonably likely to materially…

Text added vs the prior filing · source: 10-Q · 2026-05-07

• •We are a commercial-stage biopharmaceutical company in the initial stages of commercializing our product AVLAYAH, which may make it difficult for investors to evaluate our business and prospects.

• •To the extent that AVLAYAH is not commercially successful, our business, financial condition and results of operations would be materially and adversely affected and the price of our common stock would decline.

• •We may require substantial additional financing to achieve our goals, and a failure to obtain this necessary capital if or when needed on acceptable terms, or at all, could harm our business.

• •We may expend our limited resources on programs that do not yield a successful product candidate and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.

• Risks Related to the Discovery, Development and Commercialization of Our Product and Product Candidates

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• •We are in the clinical stages of drug development and have a limited operating history and no products approved for commercial sale, which may make it difficult to evaluate our current business and predict our future success and viability.

• •Drug development is a highly uncertain undertaking. We have never generated any revenue from product sales, and may never do so.

• •Due to the significant resources required for the development of our programs, and depending on our ability to access capital, we must prioritize development of certain product candidates.

• Risks Related to the Discovery, Development and Commercialization of Our Product Candidates

• •We are heavily dependent on the successful development of our TV technology and the programs currently in our pipeline, which are in the preclinical and clinical development stages.

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• On November 4, 2025, the Company's Board of Directors ("Board") increased the size of the Board from eight members to nine members and appointed Tim Van Hauwermeiren to serve as a Class I director, with a term expiring at the Company’s 2027 annual meeting of the stockholders.

• In accordance with the Company’s amended outside director compensation policy (the “Policy”), Mr. Van Hauwermeiren will receive annual cash compensation of $50,000 for his services as a member of the Board, payable quarterly in arrears on a pro-rata basis, and on November 4, 2025, Mr. Van Hauwermeir…

• Mr. Van Hauwermeiren will be eligible for equity awards on the same terms as other continuing non-employee members of the Board. The Policy provides that on the date of each annual meeting of stockholders, each non-employee director who has been a director for six months or more on the date of the a…

• Mr. Van Hauwermeiren also executed the Company’s standard form of indemnification agreement, a copy of which has been filed as Exhibit 10.1 to the Company’s Amendment No. 2 to the Registration Statement on Form S-1 (File No. 333-221522) filed with the Securities and Exchange Commission on December 7…

• There is no arrangement or understanding between Mr. Van Hauwermeiren and any other persons pursuant to which Mr. Van Hauwermeiren was elected as a director. In addition, Mr. Van Hauwermeiren is not a party to any transaction, or series of transactions, required to be disclosed pursuant to Item 404(…