Harmony Biosciences Presents New Analysis Of Clinical Impact For WAKIX® (pitolisant) At The SLEEP 2021 Annual Meeting

Rhea-AI Summary

Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) announced a new analysis of WAKIX® (pitolisant) presented at SLEEP 2021, emphasizing its clinical effectiveness for excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The analysis revealed a number needed to treat (NNT) of 5 for EDS at week 8 in the HARMONY 1 trial and an NNT of 3 for EDS and cataplexy at week 7 in the HARMONY CTP trial. Harmony's Chief Medical Officer highlighted the importance of these findings for treating patients with narcolepsy.

Positive

• WAKIX demonstrated a NNT of 5 for EDS with 67.7% of patients responding, significantly higher than the placebo at 43.3%.
• In the HARMONY CTP trial, WAKIX achieved an NNT of 3 with responses of 68.6% for EDS and 66.7% for cataplexy.

Negative

• WAKIX is contraindicated in patients with severe hepatic impairment, restricting its use.
• Potential risks include QT prolongation, which limits its use in patients with known conditions affecting heart rhythm.

PLYMOUTH MEETING, Pa., June 10, 2021 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced results of a new analysis evaluating number needed to treat (NNT) and effect sizes for treatment with WAKIX^® (pitolisant) that demonstrates the magnitude of its clinical effectiveness for excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. The poster was presented at the 35^th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2021," held virtually June 10-13.

"Excessive daytime sleepiness or impaired wakefulness can often result in brain fog, fatigue, changes in mood and other symptoms that have a large physical, emotional and psychological impact on a patient's life," said Harmony's Chief Medical Officer, Jeffrey Dayno, M.D. "We're encouraged to see our data supporting the clinical utility of WAKIX and its value as an effective and safe treatment option to help this patient population." 

Assessment of the Clinical Benefits of Pitolisant on Excessive Daytime Sleepiness and Cataplexy in Adults With Narcolepsy

This poster presents results from a new analysis that evaluated the clinical impact of pitolisant in adults with narcolepsy using NNT and effect sizes as response to treatment metrics based on data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials. NNT and effect size are important metrics as they measure the number of patients that need to be treated to achieve a specific outcome for one person and the magnitude of drug–placebo difference on outcome measures, respectively.

At week 8 in the HARMONY 1 trial (pitolisant, n=31; placebo, n=30), pitolisant had a NNT of 5 with 67.7% of patients treated with pitolisant achieving treatment response for EDS compared to placebo (43.3%) and effect size was 0.61 based on change in Epworth Sleepiness Scale scores compared to baseline.

At week 7 in the HARMONY CTP trial (pitolisant, n=54, placebo, n=51), pitolisant had a NNT of 3 with 68.6% and 66.7% of patients treated with pitolisant achieving treatment response for EDS and for cataplexy respectively compared to placebo (34% and 25.5%). Effect size was 0.86 based on changes in the Epworth Sleepiness Scale score and weekly rate of cataplexy compared to baseline.

About WAKIX^® (pitolisant) Tablets
WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy. WAKIX has been commercially available in the U.S. since Q4 2019. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States. 

Important Safety Information
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment. WAKIX is extensively metabolized by the liver and there is a significant increase in WAKIX exposure in patients with moderate impairment.

WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use of WAKIX in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is not recommended in patients with end-stage renal disease (ESRD).

In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at ≥2% and more frequently than in patients treated with placebo included headache, upper respiratory infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.

Please see the Full Prescribing Information for WAKIX for more information.

About Narcolepsy  
Narcolepsy is a rare, chronic, debilitating neurological disorder of sleep-wake state instability that impacts approximately 165,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation, which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed. 

About Harmony Biosciences 
Harmony Biosciences is a pharmaceutical company headquartered in Plymouth Meeting, PA. The Company was established by Paragon Biosciences, LLC, with a vision to provide novel treatment options for people living with rare neurological disorders who have unmet medical needs. For more information on Harmony, please visit the company's website: www.harmonybiosciences.com.

Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our product WAKIX. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: our commercialization efforts and strategy for WAKIX; the rate and degree of market acceptance and clinical utility of WAKIX, pitolisant in additional indications, if approved, and any other product candidates we may develop or acquire, if approved; our research and development plans, including our plans to explore the therapeutic potential of pitolisant in additional indications; our ongoing and planned clinical trials; our ability to expand the scope of our license agreement with Bioprojet; the availability of favorable insurance coverage and reimbursement for WAKIX; the impact of the COVID-19 pandemic; the timing of and our ability to obtain regulatory approvals for pitolisant for other indications as well as any other product candidates; our estimates regarding expenses, future revenue, capital requirements and needs for additional financing; our ability to identify additional products or product candidates with significant commercial potential that are consistent with our commercial objectives; our commercialization, marketing and manufacturing capabilities and strategy; significant competition in our industry; our intellectual property position; loss or retirement of key members of management; failure to successfully execute our growth strategy, including any delays in our planned future growth; our failure to maintain effective internal controls; the impact of government laws and regulations; volatility and fluctuations in the price of our common stock; and the significant costs and required management time as a result of operating as a public company; the fact that the price of Harmony's common stock may be volatile and fluctuate substantially; significant costs and required management time as a result of operating as a public company. These and other important factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K filed with the Securities and Exchange Commission (the "SEC") on March 25, 2021, and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

Harmony Biosciences Media Contact: 

Nancy Leone 
215-891-6046 
nleone@harmonybiosciences.com 

Harmony Biosciences Investor Contact: 

Lisa Caperelli 
484-539-9736 
lcaperelli@harmonybiosciences.com 

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SOURCE Harmony Biosciences

FAQ

What were the results of the WAKIX analysis presented by Harmony Biosciences at SLEEP 2021?

The analysis showed a NNT of 5 for excessive daytime sleepiness (EDS) and a NNT of 3 for EDS and cataplexy, highlighting the clinical effectiveness of WAKIX.

What is the significance of the NNT findings for WAKIX in narcolepsy patients?

A lower NNT indicates that fewer patients need treatment to achieve a specific clinical outcome, demonstrating WAKIX's potential effectiveness in treating narcolepsy symptoms.

When was WAKIX approved and what conditions does it treat?

WAKIX was approved by the FDA in Q4 2019 for treating excessive daytime sleepiness and cataplexy in adult patients with narcolepsy.

What safety concerns are associated with WAKIX?

WAKIX has contraindications for patients with severe hepatic impairment and carries risks like QT prolongation, which necessitates careful patient monitoring.

How is WAKIX expected to impact Harmony Biosciences' market position?

The positive clinical data supports WAKIX's value proposition in the market, potentially enhancing Harmony's competitive position in the treatment of narcolepsy.