AMLX — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-08-06

• The safety profile of AMX0035 in HELIOS data at Week 96, Week 48, and Week 24 were generally consistent with prior safety data from the studies of AMX0035. Nearly all AEs were mild or moderate, and there were no serious AEs related to AMX0035 treatment. We continue to work with the FDA on a Phase 3 …

• In addition, we are committed to supporting medically and scientifically sound research, including externally-sponsored research conducted with an institution or organization. Breakthrough T1D has provided funding to University of Washington and Amsterdam University Medical Center for a trial invest…

• In June 2026, we presented Cohort 1 biomarker data at the Annual Meeting of the European Network to Cure ALS. AMX0114 showed no drug-related serious adverse events, or SAEs, and no serious nervous system disorder adverse events, or AEs, in Cohort 1 (lowest dose level 12.5 mg). These safety findings …

• We entered into a second research collaboration with Gubra in July 2026 to identify potential development candidates for a rare endocrine disease of high unmet need.

• Research and development expenses were $23.8 million for the three months ended June 30, 2026, compared to $27.2 million for the three months ended June 30, 2025. The decrease was primarily due to a $7.1 million decrease in expenses related to AMX0035 for the treatment of PSP after its discontinuati…

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

• The safety profile of AMX0035 in HELIOS data at Week 48 and Week 24 were consistent with prior safety data from the studies of AMX0035. All AEs were mild or moderate, and there were no serious AEs related to AMX0035 treatment. We anticipate presenting longer-term Week 96 data from HELIOS at an upcom…

• In December 2025, we presented initial safety and tolerability data from Cohort 1 (n=12) of LUMINA demonstrating AMX0114 was generally well-tolerated, with no treatment-related serious AEs. Cohort 1 biomarker data from LUMINA is expected to be presented at the 2026 European Network to Cure ALS (ENCA…

• through a research collaboration with Gubra A/S, a company specializing in peptide-based drug discovery and preclinical contract research services. IND-enabling studies for AMX0318 are underway with an IND targeted for 2027.

• Comparison of the three months ended March 31, 2026 and 2025

• Research and development expenses were $27.6 million for the three months ended March 31, 2026, compared to $22.1 million for the three months ended March 31, 2025. The increase was primarily due to a $4.6 million increase in expenses related to the pivotal Phase 3 LUCIDITY clinical trial in PBH and…

Text added vs the prior filing · source: 10-Q · 2026-08-06

• In addition to the Shih Complaint, two derivative complaints were filed in the U.S. District Court for the District of Massachusetts on October 2, 2024, Jones v. Cohen, et al., 1:24-cv-12527, and on July 2, 2025, Hassine v. Cohen, et al., 1:25-CV-11879 (together, the Massachusetts Derivative Complai…

• On July 16, 2025, the parties to both Massachusetts Derivative Complaints moved the Court to consolidate the two actions and stay the consolidated action, which the Court approved on July 22, 2025. On June 12, 2026, following the lifting of the stay, plaintiffs filed an amended complaint. On July 14…

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

• In addition to the Shih Complaint, on October 2, 2024, a derivative complaint was filed in the U.S. District Court for the District of Massachusetts against certain current and former director and officer defendants, or the Individual Defendants, naming the Company as a nominal defendant (Jones v. C…

• On July 2, 2025, a second derivative complaint was filed in the Court against certain current and former directors and officer defendants, naming the Company as nominal defendant (Hassine v. Cohen, et al., 1:25-CV-11879, or the Hassine Derivative Complaint and, together with the Jones Derivative Com…

• We intend to defend against the Derivative Complaints vigorously. At this time, an estimate of the impact, if any, of the claims made in the Derivative Complaints cannot be made.

Text added vs the prior filing · source: 10-Q · 2026-08-06

• Our future success depends significantly on our ability to successfully develop, and obtain regulatory approvals for and commercialize, avexitide in PBH and AMX0035 in indications other than ALS, including Wolfram syndrome. Avexitide has been

• evaluated in five Phase 1 and Phase 2 clinical trials for PBH and has also been studied in congenital HI. In February 2025, we activated the first sites for the pivotal Phase 3 LUCIDITY clinical trial for avexitide in PBH and in April 2025, we announced that the first participant had been dosed. Rec…

• Similar requirements may be imposed on us by the European Medicines Agency, or EMA, in the EU and comparable regulatory authorities in other jurisdictions where we intend to seek regulatory approval. For any fixed-dose combination products we may develop, we may be required to produce clinical data …

• If the FDA or other comparable foreign regulatory authorities require us to conduct one or more clinical trials to support such a demonstration, such as a factorial study, the design, duration and scope of such clinical trials will be decided upon after further discussions with those agencies and ot…

• We, and any future collaborators, are not permitted to commercialize, market, promote or sell any product candidate in the U.S. or elsewhere without obtaining regulatory approval from the FDA and other comparable foreign regulatory authorities. Regulatory

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

• Adverse developments affecting the financial services industry, such as actual events or concerns involving liquidity, defaults or non-performance by financial institutions or transactional counterparties, could adversely affect our current and projected business operations and our financial conditi…

• Events involving limited liquidity, defaults, non-performance or other adverse developments that affect financial institutions, transactional counterparties or other companies in the financial services industry or the financial services industry generally, or concerns or rumors about any events of t…

• liquidity problems. Although we do not currently have investments with any financial institution that has experienced such events, if any financial institution with which we have a relationship were to be placed into receivership, we may be unable to access such funds. In addition, if any parties wi…

• Inflation and fluctuations in interest rates have led to a decline in the trading value of previously issued government securities with interest rates below current market interest rates. Although the U.S. Department of Treasury, Federal Deposit Insurance Corporation, or FDIC, and Federal Reserve Bo…

• Although we assess our banking relationships as we believe necessary or appropriate, our access to funding sources in amounts adequate to finance or capitalize our current and projected future business operations could be significantly impaired by factors that affect us, the financial institutions w…

Text added vs the prior filing · source: 10-Q · 2026-08-06

• Gina M. Mazzariello, our Chief Legal Officer and General Counsel, adopted a new Rule 10b5-1 trading plan on April 9, 2026, which is scheduled to expire on December 31, 2026. The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 55,000;

• James Frates, our Chief Financial Officer, adopted a new Rule 10b5-1 trading plan on April 10, 2026, which is scheduled to expire on January 31, 2027. The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 40,000;

• George Milne, a member of our board of directors, adopted a new Rule 10b5-1 trading plan on April 28, 2026, which is scheduled to expire on December 31, 2026. The aggregate number of shares of our common stock authorized to be sold under this new arrangement is 100,000;

• Joshua Cohen, our Co-Chief Executive Officer and a member of our board of directors, adopted a new Rule 10b5-1 trading arrangement on May 11, 2026, which is scheduled to expire on December 31, 2026. The aggregate number of shares of our common stock authorized to be sold under this new arrangement i…

• Justin Klee, our Co-Chief Executive Officer and a member of our board of directors, adopted a new Rule 10b5-1 trading arrangement on May 11, 2026, which is scheduled to expire on December 31, 2026. The aggregate number of shares of our common stock authorized to be sold under this new arrangement is…