Axsome Therapeutics Begins Phase III Study on AXS-14 for Fibromyalgia

Axsome Therapeutics AXSM announced the initiation of the phase III study called FORWARD with the dosing of the first patient, evaluating AXS-14 (esreboxetine) for the management of fibromyalgia.

AXS-14 is an oral, investigational drug, being developed to treat fibromyalgia and help reduce chronic pain and related symptoms by increasing norepinephrine levels in the brain.

FORWARD is a phase III double-blind, placebo-controlled withdrawal study in patients with fibromyalgia. Participants achieving a treatment response during the 12-week open-label period will be randomized in a 1:1 ratio to continue AXS-14 at 8 mg once daily or switch to placebo for up to 12 weeks or until loss of therapeutic response. The study’s primary endpoint is to assess the time from randomization to loss of therapeutic response.

Over the past year, AXSM’s shares have risen 89.4% compared with the industry’s 18.7% rise.

Axsome submitted a new drug application (NDA) seeking FDA approval of AXS-14 for fibromyalgia in May 2025. The NDA filing was delayed from the previous filing timeline of the first quarter of 2024.

In June 2025, the company received a Refusal to File (“RTF”) letter from the FDA related to its NDA for AXS-14. The regulatory authority found one of the two placebo-controlled studies in the submission to be inadequate and not well-controlled due to its 8-week primary endpoint and flexible dosing design, which were deemed insufficient to demonstrate efficacy in fibromyalgia. To address this regulatory feedback, Axsome initiated the phase III FORWARD study.

Fibromyalgia is a chronic, CNS-mediated neurological pain disorder affecting approximately 17 million people in the United States. It is characterized by widespread pain accompanied by fatigue, sleep disturbances, mood and cognitive impairment, sensory hypersensitivity, headaches, and tingling sensations.

Beyond AXS-14, Axsome’s CNS pipeline includes AXS-12, currently being developed in three separate studies (CONCERT, SYMPHONY, and ENCORE) for the treatment of narcolepsy. Treatment with AXS-12 demonstrated statistically significant efficacy versus placebo in all three studies. Based on the data from these studies, Axsome plans to submit an NDA for AXS-12 for cataplexy in patients with narcolepsy to the FDA in late January 2026.

Axsome Therapeutics’ Zacks Rank & Stocks to Consider

AXSM currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are MannKind MNKD, Keros Therapeutics KROS, and Amicus Therapeutics FOLD, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for MannKind’s 2026 earnings per share have increased from 7 cents to 9 cents. Shares of MNKD have declined 7.2% over the past year.

MannKind’s earnings beat estimates in two quarters, missed in one and were in line in the remaining quarter, with the average surprise being 33.33%.

Over the past 60 days, 2026 loss per share estimates for Keros Therapeutics have narrowed from $3.56 to $3.36. KROS shares have risen 77.3% over the past year.

Keros Therapeutics’ earnings beat estimates in three of the trailing four quarters and missed in the remaining quarter, with the average surprise being 9098.63%.

Over the past 60 days, estimates for Amicus Therapeutics’ 2026 earnings per share have declined from 67 cents to 65 cents. Shares of FOLD have increased 52% over the past year.

Amicus Therapeutics’ earnings beat estimates in one quarter and missed in the remaining three trailing quarters with the negative average earnings surprise being 20.21%.

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