LRMR — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-08-04

• In June 2026, following minutes from a multidisciplinary Type B pre-BLA meeting and FDA review of the nomlabofusp briefing package, we announced continued agreement with the FDA on key elements of a potential BLA submission including:

• confirmed that the existing data package appears capable of supporting submission and review of a BLA seeking accelerated approval based on data from the OL study; approval will be a matter of review;

• reaffirmed its willingness to consider FXN as a novel surrogate endpoint and confirmed that our exposure-response analysis linking nomlabofusp exposure to clinical outcomes may support the BLA submission;

• stated that the prospectively collected gene expression and lipid biomarker data may provide an opportunity to further characterize the biological activity of nomlabofusp beyond FXN tissue concentrations;

• In June 2026, we submitted the first module of our rolling BLA submission and expect that the remaining modules will be submitted in the second half of 2026.

Text removed vs the prior filing · source: 10-Q · 2026-05-14

• We have scheduled a Type B meeting with the FDA later in the second quarter of 2026. We plan to provide topline study data from our OL study in the second quarter of 2026 and we plan to initiate a rolling BLA seeking accelerated approval with the submission of nonclinical and clinical modules in Jun…

• For our global confirmatory Phase 3 study, we are planning sites in the U.S., E.U., U.K., Canada and Australia. We have obtained feedback from both the FDA and the EMA on the study protocol. We plan to initiate dosing of the first patient in mid-2026.

• We have funded our operations to date primarily with proceeds from sales of common stock, proceeds from the sale of prefunded warrants for the purchase of common stock, the acquisition in 2020 of cash, cash equivalents, marketable securities, and restricted cash upon the merger with Zafgen, Inc. (“Z…

• In May 2024, we entered into a Sales Agreement (“2024 ATM Agreement”) with Guggenheim Securities, LLC in connection with the establishment of an “at-the-market” offering program providing for the sale of up to an aggregate of $100 million of shares of our common stock from time to time. To date, we …

• In July 2025, we completed an underwritten public offering in which we issued and sold 21,562,500 shares of our common stock, including the exercise in full of the underwriters' option to purchase additional shares, at a public offering price of $3.20 per share. We received net proceeds of approxima…

Text added vs the prior filing · source: 10-Q · 2026-08-04

• We initiated our rolling BLA submission to the FDA for marketing approval of nomlabofusp using the accelerated approval pathway and expect to submit the remaining modules in the second half of 2026; however, there can be no assurance that we will submit the remaining modules on our expected timeline…

• We submitted the first module of our BLA to the FDA in June 2026 to request marketing approval of nomlabofusp. We expect to submit the remaining modules in the second half of 2026. However, our anticipated submission timeline is based on our current development, manufacturing and regulatory plans an…

• We also rely on third parties for non-clinical, clinical and manufacturing activities to support the conduct of our trials and our BLA submission. As part of the FDA review process, we are subject to inspections by FDA, as are each of our third-party sites, vendors and suppliers including our clinic…