HRMY Stock Down as Fragile X Syndrome Study Fails to Meet Goal

Shares of Harmony Biosciences Holdings, Inc. HRMY lost 16.56% on Sept. 24 after the company announced the failure of the late-stage registrational RECONNECT study on pipeline candidate ZYN002 in Fragile X syndrome (FXS).

RECONNECT Study was a randomized, double-blind, placebo-controlled, multiple-center study to assess the efficacy and safety of ZYN002.

ZYN002 is a pharmaceutically manufactured cannabidiol administered as a transdermal gel to patients with FXS aged 3 to under 30 years.

FXS is a rare genetic disorder that is the leading known cause behind inherited intellectual disability and autism spectrum disorder.

The study did not meet the primary endpoint of improvement in social avoidance, primarily due to a higher-than-expected placebo response rate.

The failure of a late-stage study disappointed investors.

ZYN002 was added to HRMY’s pipeline through the acquisition of Zynerba Pharmaceuticals in 2023.

Harmony Biosciences has lost 22.2% year to date against the industry’s gain of 5.3%.

HRMY Has Other Arrows in Its Arsenal

Harmony Biosciences’ lead drug, Wakix (pitolisant), is a first-in-class therapy with a novel mechanism of action designed to enhance histamine signaling in the brain by binding to H3 receptors. Revenues from the drug totaled $200.5 million in the second quarter.

HRMY plans to initiate phase III registrational trials evaluating a high dose of pitolisant in both narcolepsy and idiopathic hypersomnia in the fourth quarter of 2025.

Data from a pivotal bioequivalence study on pitolisant gastro-resistant is anticipated in the fourth quarter.

HRMY broadened its pipeline into rare epilepsy with the acquisition of Epygenix Therapeutics, Inc.

The acquisition added EPX-100, a serotonin (5HT-2) receptor agonist, and EPX-200, a selective 5HT2C agonist, to its pipeline. EPX-100 is in phase III registrational trials for Dravet Syndrome and Lennox-Gastaut Syndrome. EPX-200 is in the pre-IND phase for developmental and epileptic encephalopathies.

HRMY Zacks Rank and Stocks to Consider

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

Some better-ranked stocks in the biotech sector are Halozyme Therapeutics HALO and ANI Pharmaceuticals, Inc. ANIP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Halozyme Therapeutics

The Zacks Consensus Estimate for 2025 EPS has increased 74 cents to $6.18 in the past 90 days. Shares of Halozyme have surged 58.2% year to date.

HALO’s earnings beat estimates in each of the trailing four quarters, the average surprise being 19.74%.

ANI Pharmaceuticals

ANIP’s shares have gained 71.9% so far this year. Earnings estimates for 2025 have risen 82 cents in the past 60 days. ANIP’s earnings beat estimates in each of the trailing four quarters, the average surprise being 22.66%.

 

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