Harmony Biosciences Holdings, Inc. Stock Price, News & Analysis

Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) presented a comprehensive pipeline update at its Investor Day event, showcasing new data and program updates. Key highlights include:

1. Orexin-2 agonist program: BP1.15205 demonstrated greater potency compared to other orexin-2 agonists.

2. Pitolisant in Idiopathic Hypersomnia: Long-Term Extension study showed sustained efficacy.

3. Pitolisant-HD program: Preliminary data confirmed safety up to five times the highest labeled dose of WAKIX.

4. Fragile X syndrome program: Phase 3 RECONNECT study on track for topline data in mid-2025.

5. EPX-100 development program: Favorable risk/benefit profile for Dravet syndrome and Lennox-Gastaut syndrome.

Harmony aims to deliver one or more new product or indication launches each year over the next five years, potentially generating over $3 billion in annual revenue.

Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) will host its first Investor Day on October 1, 2024, at 8:30 a.m. ET in New York City. The event will showcase the company's robust, late-stage pipeline to investors and research analysts. Key highlights include:

• New data on pitolisant for idiopathic hypersomnia (IH)
• Updates on pitolisant-HD development program
• New data on BP1.15205, a potential best-in-class orexin-2 agonist
• Scientific rationale for ZYN002 and EPX100 in Phase 3 trials
• Market potential for late-stage pipeline assets

The company's portfolio now includes three orphan/rare CNS franchises, each with potential peak sales of $1B - $2B. Investors can attend in person or virtually by registering through Harmony's Investor Relations.

Harmony Biosciences Holdings (Nasdaq: HRMY) has announced its participation in two upcoming investor conferences. The company's management team will be present at:

1. The Wells Fargo 2024 Healthcare Conference in Boston, with a fireside chat scheduled for Thursday, September 5, 2024, at 9:30 a.m. ET.

2. The 2024 Cantor Global Healthcare Conference in New York City, featuring a fireside chat on Tuesday, September 17, 2024, at 3:05 p.m. ET.

Investors and interested parties can access webcasts of both fireside chats through the investor page of Harmony's website at https://ir.harmonybiosciences.com/.

Harmony Biosciences (HRMY) reported strong Q2 2024 financial results, with WAKIX® (pitolisant) net revenue of $172.8 million, representing 29% year-over-year growth. The company is advancing its pitolisant high-dose program with an expected PDUFA date in 2028, potentially extending the franchise beyond 2040. Key highlights include:

- WAKIX patent upheld by USPTO
- FDA approval for WAKIX in pediatric narcolepsy
- On track for sNDA submission for idiopathic hypersomnia in Q4 2024
- Average number of WAKIX patients increased to ~6,550
- Reiterates 2024 net product revenue guidance of $700-$720 million

The company reported GAAP net income of $11.6 million and non-GAAP adjusted net income of $60.6 million for Q2 2024.

Harmony Biosciences Holdings (Nasdaq: HRMY) has announced that it will release its second quarter 2024 financial results on Tuesday, August 6, 2024, before the U.S. financial markets open. The company will host a conference call and webcast at 8:30 a.m. ET on the same day to discuss the results. Interested parties can participate in the call by dialing (800) 225-9448 (domestic) or (203) 518-9708 (international) and using the passcode HRMYQ224. It is advised to dial in at least 10 minutes before the call starts. A live and replay webcast of the call will be available on the investor page of Harmony Biosciences' website.

On June 25, 2024, Harmony Biosciences announced that the FDA denied a Citizen Petition filed by a short seller, which challenged the safety and efficacy of WAKIX (pitolisant) for treating excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The FDA confirmed WAKIX's favorable benefit-risk profile, rejecting the petition's requests, including the drug's withdrawal and transition to a Compassionate Use Program. Furthermore, the FDA had recently approved a supplemental New Drug Application (sNDA) for WAKIX, extending its use to pediatric patients aged 6 and older with narcolepsy. Harmony Biosciences plans to launch at least one new product or indication annually over the next five years and aims to extend the pitolisant franchise beyond 2040 with next-generation formulations.

Harmony Biosciences (Nasdaq: HRMY) has received FDA approval for its drug WAKIX® (pitolisant) to treat excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy. This approval follows a Phase 3 study by Bioprojet, which also led to European Medicines Agency approval for the same use last year. While WAKIX will now be available for pediatric EDS, FDA has issued a complete response for its use in treating cataplexy in this age group. WAKIX, first approved in 2019 for adult narcolepsy, is the only FDA-approved non-scheduled treatment for this condition. The company plans to discuss further pathways for cataplexy treatment in pediatric patients with the FDA.

Harmony Biosciences (Nasdaq: HRMY) has reported positive data from its Phase 2 study on pitolisant for treating excessive daytime sleepiness (EDS) and fatigue in myotonic dystrophy type 1 (DM1) patients. The study showed significant improvement in EDS and fatigue symptoms with higher doses of pitolisant compared to placebo. The results encourage further development of pitolisant using the Next-Generation 2 (NG2) formulation for a pivotal Phase 3 study. The study included a randomized, double-blind, placebo-controlled design with a total of 25 participants. Both primary and secondary efficacy endpoints indicated greater mean improvements for pitolisant over placebo, and adverse event rates were similar across groups. Pitolisant, marketed as WAKIX® in the U.S., is currently FDA approved for narcolepsy-related EDS and cataplexy but remains investigational for DM1.

Harmony Biosciences announced its participation in the Goldman Sachs 45th Annual Global Healthcare Conference.

The event will take place on June 10, 2024, in Miami Beach, FL.

The management team will engage in a fireside chat at 9:20 a.m. ET.

Investors can access a webcast of the session on Harmony's investor relations page.