Strongbridge Biopharma plc Announces Publication of Long-term Efficacy and Safety Results for KEVEYIS® (dichlorphenamide) for the Treatment of Primary Periodic Paralysis in Muscle & Nerve

Rhea-AI Summary

Strongbridge Biopharma plc (Nasdaq: SBBP) announced that post hoc analyses from a year-long study on KEVEYIS (dichlorphenamide) confirm its long-term safety and effectiveness for treating Primary Periodic Paralysis (PPP). Results published in Muscle & Nerve highlight maintained efficacy over 61 weeks, with reduced attack rates among users. Adverse events, primarily paresthesia and cognitive impairment, were lower in frequency during the study's final 52 weeks. These findings support KEVEYIS as a key treatment option for this rare neuromuscular disorder.

Positive

• Post hoc analyses confirm long-term safety and effectiveness of KEVEYIS.
• Decreased median weekly attacks reported among continuous KEVEYIS users.
• No new safety signals noted during the final 52 weeks of the study.
• Adverse events reported at lower frequencies in the latter part of the study.

Negative

• None.

~ Study Confirms Long-Term Treatment with KEVEYIS is Safe and Effective for Chronic Use ~

~ Analyses Provide Potentially Useful Information to Help Guide Clinician Patient Counseling & Management When Starting Treatment with KEVEYIS ~

DUBLIN, Ireland and TREVOSE, Pa., July 13, 2021 (GLOBE NEWSWIRE) -- Strongbridge Biopharma plc, (Nasdaq: SBBP), a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs, today announced that post hoc analyses from a one year open-label study evaluating daily use of KEVEYIS® (dichlorphenamide) for the treatment of Primary Periodic Paralysis (PPP) following a nine-week randomized, controlled study were published in the peer-reviewed journal, Muscle & Nerve. The results confirmed that long-term treatment with KEVEYIS is safe and effective for chronic use.

“The published results from the post hoc analyses of the HYPHOP study extend the positive findings from the original KEVEYIS® (dichlorphenamide) HYPHOP study by demonstrating that efficacy was maintained over the entire 61-week study with no evidence of waning over time,” said Fredric Cohen, M.D., chief medical officer of Strongbridge Biopharma. “Moreover, there was evidence of further reduction in median weekly attacks among continuous KEVEYIS users to nearly none by the end of the study, and patients who were switched from placebo to open-label KEVEYIS after week nine had reduced attack rates that were similar in frequency at the end of the study to those who had been continuously treated with KEVEYIS over the entire study. Collectively, these results reinforce the utility of KEVEYIS as an effective option in treating PPP, a life-long rare, hereditary skeletal muscle disorder that leads to debilitating attacks of muscle weakness.”

The open label extension results of HYPHOP add to previously reported results from the short-term randomized, double-blind, placebo-controlled phase of the study, with hyperkalemic and hypokalemic PPP sub-studies, which demonstrated the effectiveness of KEVEYIS to prevent attacks of muscle weakness.

Adverse event analyses indicated no new safety signals during the final 52 weeks of the study versus the first nine weeks, with the common adverse events of paresthesia and cognition impairment, reported at lower frequencies during the extension as compared with the first 9 weeks. Clinicians can advise patients starting KEVEYIS that most patients who had one of these events initially reported it within the first month of treatment. Temporary dose reduction appeared to be a reasonable approach to manage bothersome paresthesia or cognition-related AEs, as patients with these AEs who were managed with dosage reduction often had subsequent symptom resolution.

The manuscript, entitled “Long-term efficacy and safety of dichlorphenamide for treatment of primary periodic paralysis,” can be accessed here.

About KEVEYIS
KEVEYIS^® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants. In clinical studies, the most common side effects of KEVEYIS were a numbness or tingling, difficulty thinking and paying attention, changes in taste, and confusion. These are not all of the possible side effects that you may experience with KEVEYIS. Talk to your doctor if you have any symptoms that bother you or do not go away. You are encouraged to report side effects to Strongbridge Biopharma at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch. For additional KEVEYIS important safety information and the full prescribing information visit www.keveyis.com.

About Strongbridge Biopharma
Strongbridge Biopharma is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Strongbridge’s rare endocrine franchise includes RECORLEV^® (levoketoconazole), an adrenal steroidogenesis inhibitor with a New Drug Application that is currently under review by the FDA for the treatment of endogenous Cushing’s syndrome, and veldoreotide extended release, a pre-clinical next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both RECORLEV and veldoreotide have received orphan drug designation from the FDA and the European Medicines Agency. The company’s rare neuromuscular franchise includes KEVEYIS^® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis. KEVEYIS has orphan drug exclusivity in the United States.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the federal securities laws. The words “anticipate,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will,” “would,” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. All statements, other than statements of historical facts, contained in this press release, are forward-looking statements, including statements related to Strongbridge’s strategy, plans, results of clinical trials, outcomes of product development efforts and objectives of management for future operations. Forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those expressed in such statement, including risks and uncertainties associated with clinical development and the regulatory approval process, the reproducibility of any reported results showing the benefits of RECORLEV, the adoption of RECORLEV by physicians, if approved, as treatment for any disease and the emergence of unexpected adverse events following regulatory approval and use of the product by patients.  Additional risks and uncertainties relating to Strongbridge and its business can be found under the heading “Risk Factors” in Strongbridge’s Annual Report on Form 10-K for the year ended December 31, 2020 and its subsequent Quarterly Reports on Form 10-Q, as well as its other filings with the SEC. These forward-looking statements are based on current expectations, estimates, forecasts and projections and are not guarantees of future performance or development and involve known and unknown risks, uncertainties and other factors. The forward-looking statements contained in this press release are made as of the date of this press release, and Strongbridge Biopharma does not assume any obligation to update any forward-looking statements except as required by applicable law.

Contacts:

Corporate and Media Relations
Elixir Health Public Relations
Lindsay Rocco
+1 862-596-1304
lrocco@elixirhealthpr.com

Investor Relations
Solebury Trout
Mike Biega
+1 617-221-9660
mbiega@soleburytrout.com

FAQ

What does the recent Strongbridge Biopharma press release say about KEVEYIS?

The press release states that long-term treatment with KEVEYIS is confirmed to be safe and effective for Chronic use in treating Primary Periodic Paralysis, as evidenced by recent study analyses.

What were the findings of the KEVEYIS study published in Muscle & Nerve?

The study found that KEVEYIS maintained its efficacy over a 61-week period, with significant reduction in muscle weakness attacks among users.

What safety information was reported about KEVEYIS in the press release?

The press release indicated that no new safety signals were detected during the final 52 weeks of the study, with common adverse events occurring less frequently in the latter stages.

Which conditions is KEVEYIS approved to treat?

KEVEYIS is approved for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.

How did patients respond to KEVEYIS regarding adverse events?

Patients managed with dosage reductions for adverse events like paresthesia often experienced symptom resolution, as noted in the study's findings.