DYN 最新10-Q变化

相对上期新增的文字 · 来源:10-Q · 2026-07-29

• from the FDA, the European Medicines Agency and the Japanese Ministry of Health, Labour and Welfare for the treatment of individuals with DMD, amenable to exon 51 skipping. Additionally, we are advancing four development candidates (DYNE-253, DYNE-245, DYNE-244 and DYNE-255) for the treatment of DMD…

• Z-rostudirsen is being evaluated in the long-term extension portion of the DELIVER trial, a global Phase 1/2 clinical trial designed to be registrational, and the FORZETTO trial, a global Phase 3 clinical trial designed to be confirmatory. We have aligned with the FDA on the FORZETTO trial design an…

• In May 2026, we initiated our Phase 3 FORZETTO clinical trial, a global, randomized, placebo-controlled, double-blind, confirmatory Phase 3 trial designed to assess the efficacy, safety, and tolerability of z-rostudirsen administered intravenously to ambulatory male participants with DMD amenable to…

• In July 2026, we announced that the FDA accepted for review our BLA for z-rostudirsen for the treatment of individuals with DMD amenable to exon 51 skipping. The FDA has granted the BLA priority review and assigned a Prescription Drug User Fee Act, or PDUFA, target action date of January 21, 2027. W…

• Z-basivarsen is being evaluated in the ACHIEVE trial, a global Phase 1/2 clinical trial designed to be registrational, and the HARMONIA trial, a global Phase 3 clinical trial designed to be confirmatory. We have aligned with the FDA on the HARMONIA trial design and protocol.

相对上期删除的文字 · 来源:10-Q · 2026-05-11

• Z-rostudirsen is currently being evaluated in the DELIVER trial, a global Phase 1/2 clinical trial which is designed to be registrational. Following a positive pre-BLA meeting with the FDA, we continue to plan to submit a biologics license application, or BLA, to the FDA for U.S. Accelerated Approva…

• Z-basivarsen is being evaluated in the ACHIEVE trial, a global Phase 1/2 clinical trial designed to be registrational, and the HARMONIA trial, a global Phase 3 clinical trial designed to be confirmatory, which was initiated in March 2026. We have aligned with the FDA on the HARMONIA trial design and…

• We continue to explore additional applications for our proprietary FORCE platform, along with further iterations. To that end, we have identified two compounds, which we refer to as Conjugate 1 and Conjugate 2, utilizing a Fab conjugated to

• microtubule associated protein tau (MAPT) siRNA designed to downregulate expression of all MAPT isoforms. Conjugate 1 utilizes our FORCE platform with the same Fab as our other programs. Conjugate 2 utilizes a modified form of the FORCE TfR1-binding Fab that has been further optimized for enhanced c…

• enter into such agreements or arrangements as and when needed, we may have to significantly delay, reduce or eliminate the development or future commercialization of one or more product candidates we may develop.

相对上期新增的文字 · 来源:10-Q · 2026-07-29

• controls and procedures as of June 30, 2026, our Chief Executive Officer and Chief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.

• There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that occurred during the three months ended June 30, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control ove…

相对上期删除的文字 · 来源:10-Q · 2026-05-11

• There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) that occurred during the three months ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control ov…

相对上期新增的文字 · 来源:10-Q · 2026-07-29

• unknown factors that may adversely affect our business. The size of our future net losses will depend, in part, on the rate of future growth of our expenses and our ability to generate revenue.

• We may never succeed in these activities and, even if we do, may never generate revenues that are significant enough to achieve profitability. Our product candidates are in varying stages of preclinical and clinical development, and we have not completed clinical development of any product candidate…

• As of June 30, 2026, we had cash, cash equivalents and marketable securities of $898.5 million. In addition, in connection with our July 2026 offering, we estimate we received net proceeds of approximately $405.0 million, after deducting underwriting discounts and commissions and offering expenses p…

• losses, or NOLs, or research and development tax credit carryforwards. As of December 31, 2025, we had federal NOL carryforwards of $981.6 million and state NOL carryforwards of $1.0 billion.

• In addition, if we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies governing clinical trials, our development plans may be impacted. For example, in December 2022, with the passage of FDORA, Congress required sponsors to develop and sub…

相对上期删除的文字 · 来源:10-Q · 2026-05-11

• We are only in the preliminary stages of most of these activities. We may never succeed in these activities and, even if we do, may never generate revenues that are significant enough to achieve profitability. Our product candidates are in

• varying stages of preclinical and clinical development, and we have not completed clinical development of any product candidate. Because of the numerous risks and uncertainties associated with product development, we are unable to accurately estimate or know the nature, timing or costs of the effort…

• As of March 31, 2026, we had cash, cash equivalents and marketable securities of $972.2 million.

• and eventual commercialization of our product candidates, which may never occur. We currently generate no revenue from sales of any product, and we may never be able to develop or commercialize a marketable product.

• In addition, if we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies governing clinical trials, our development plans may be impacted. For example, in December 2022, with the passage of FDORA, Congress required sponsors to develop and sub…

相对上期新增的文字 · 来源:10-Q · 2026-07-29

• None of our directors or officers (as defined in Rule 16a-1(f) under the Securities Exchange Act of 1934, as amended) adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement (as defined in Item 408(c) of Regulation S-K) during the quarterly period covered by …

相对上期删除的文字 · 来源:10-Q · 2026-05-11

• The following table describes, for the period covered by this Quarterly Report, each trading arrangement for the sale or purchase of Company securities adopted or terminated by our directors and officers that is a contract, instruction or written plan intended to satisfy the affirmative defense cond…

• * In addition to 10% of the number of shares acquired upon the vesting of restricted stock units after a sell-to-cover transaction to satisfy tax withholding obligations, which number cannot be determined at this time.