~ Strongbridge Commercial Introduction of KEVEYIS® (dichlorphenamide) in Mid-April Shows Encouraging Initial Demand ~
~ RECORLEV™ (levoketoconazole) SONICS Study Remains on Track to Complete Enrollment in the Second Quarter of 2017; Data Safety Monitoring Board Recommends Study Continue as Planned ~
DUBLIN, Ireland and TREVOSE, Pa., May 16, 2017 (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 provided a corporate update and reported first quarter 2017 financial results.
“We recently initiated our commercial introduction of KEVEYIS® (dichlorphenamide), the first and only FDA approved treatment for Primary Periodic Paralysis (PPP), an ultra-rare, genetic, debilitating neuromuscular disease. We are focused on meeting the needs of patients and the broader PPP community by enabling access to treatment and providing comprehensive patient and physician support services. Although it is still early in our launch rollout, it is clear that the unmet need in the PPP market is significant, and we are pleased with the interest in, and demand for, KEVEYIS,” said Matthew Pauls, president and chief executive officer of Strongbridge Biopharma. “Simultaneously, we have maintained our strong focus on driving the clinical development of RECORLEV™ (levoketoconazole), our Phase 3 product candidate for the treatment of endogenous Cushing’s syndrome (CS). Enrollment in the Phase 3 SONICS study is on track for completion in the second quarter, and enrollment in the LOGICS study will begin on schedule by mid-year. The need for a safe and effective, next-generation cortisol inhibitor in the treatment of CS is substantial, and we are excited about the potential to become a leader in the growing CS market in the near term,” Pauls added.
“Primary Periodic Paralysis (PPP) is an ultra-rare and complex neuromuscular condition, which is often misunderstood and can take a significant toll on a patient's ability to participate in everyday life activities. For the five-to-six thousand people in the U.S. living with this physically debilitating genetic condition, the time from disease onset to confirmed diagnosis can sometimes take up to 20 years,” said Jacob Levitt, M.D., F.A.A.D., president and medical director of the Periodic Paralysis Association. “The PPP community is pleased by Strongbridge’s commitment and early efforts to further educate physicians and improve upon the time it takes to diagnose this treatable condition.”
Recent Corporate Highlights
- Strongbridge Commercial Introduction of KEVEYIS in Mid-April Shows Encouraging Initial Demand, Successful Transition of Existing Patients to Commercial Supply. Strongbridge launched KEVEYIS (www.keveyis.com) in the U.S. market in mid-April after acquiring the U.S. marketing rights to it from Taro Pharmaceutical Industries Ltd. in December 2016. KEVEYIS is the first and only FDA-approved treatment indicated for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis (PPP), which is a group of rare hereditary disorders that causes potentially severe episodes of muscle weakness and/or paralysis.
The Company has partnered with PANTHERx® Specialty Pharmacy to build a customized support and product distribution program, Strongbridge CareConnection, for members of the KEVEYIS ecosystem, including patients, caregivers, physicians and payers. During the month of April, a phased reimbursement transition process was implemented, resulting in the Company successfully transitioning, thus far, more than 70 percent of the 80 existing KEVEYIS patients to Strongbridge’s commercial supply, and continues to make progress transitioning remaining patients. The Company’s highly specialized 12 person rare disease sales team, with nearly 80 years of collective rare disease sales experience and a combined 20 orphan product launches, has generated a number of new patient start forms just three weeks into the recent KEVEYIS launch. The Company has also built out key functions across the enterprise to support the KEVEYIS efforts, including patient support services, medical science liaisons, market access, and marketing personnel.
- RECORLEV Phase 3 clinical development program timeline on track. The Phase 3 SONICS study evaluating RECORLEV for the treatment of endogenous Cushing’s syndrome has a sufficient number of patients dosed and potential subjects in the enrollment queue to achieve full enrollment by the end of the second quarter of 2017, with top-line data supporting the primary efficacy analysis available in the first quarter of 2018. The SONICS Data and Safety Monitoring Board (DSMB) recently completed its semi-annual comprehensive data review, and determined that the study was progressing well and that there were no safety concerns warranting changes to the study of any kind. Patient enrollment in the Phase 3 LOGICS study, which will supplement the long-term efficacy and safety data from SONICS, is anticipated to begin enrolling in mid-2017 with top-line data anticipated in the third quarter of 2018.
- Financial position consistent with prior guidance. Strongbridge had cash and cash equivalents of $49.9 million and $20.0 million in outstanding debt as of March 31, 2017, compared to cash and cash equivalents of $66.8 million and $20.0 million in outstanding debt as of December 31, 2016. Net cash used in operating activities was $9.3 million for both the three months ended March 31, 2017 and 2016. In addition, during the three months ended March 31, 2017, the Company paid the remaining $7.5 million of the upfront payment owed in connection with the acquisition of the U.S. marketing rights of KEVEYIS. The Company continues to believe its existing financial resources are sufficient to fund planned operations into 2019.
Year-to-Date March 2017 Results
For the three months ended March 31, 2017, basic and diluted net loss attributable to ordinary shareholders was $29.5 million, or $0.83 per share. Basic and diluted net loss during the three months ended March 31, 2017 included a $14.9 million non-cash charge related to an increase in the fair value of the Company’s warrant liability resulting from the increase in the Company’s stock price during the quarter, and non-cash charges of $1.6 million for income taxes and $1.3 million for intangible asset amortization. The basic and diluted net loss attributable to ordinary shareholders for the three months ended March 31, 2016 was $12.2 million, or $0.57 per share.
Research and development expenses were $3.5 million for the three months ended March 31, 2017, compared to $6.8 million for the same period in the prior year. The decrease during the 2017 period was primarily due to decreased development spend related to programs discontinued during 2016, a planned decrease in development activity for veldoreotide, and a timing-related decrease in expenses relating to the ongoing clinical trials for RECORLEV.
Selling, general and administrative expenses were $7.4 million for the three months ended March 31, 2017, compared to $4.1 million for the same period in the prior year. The increase during the 2017 period was primarily due to costs incurred in preparing for the launch of KEVEYIS.
|STRONGBRIDGE BIOPHARMA plc|
|Select Consolidated Balance Sheet Information|
|(Unaudited, in thousands, except share and per share data)|
|March 31,||December 31,|
|( in thousands)|
|Consolidated Balance Sheet Data:|
|Cash and cash equivalents||$||49,898||$||66,837|
|Total shareholders’ equity||38,656||66,972|
|STRONGBRIDGE BIOPHARMA plc|
|Consolidated Statement of Operations and Comprehensive Loss|
|(Unaudited, in thousands, except share and per share data)|
|Three Months Ended March 31,|
|Consolidated Statement of Operations Data:|
|Research and development||$||3,481||$||6,794|
|Selling, general and administrative||7,442||4,129|
|Amortization of intangible asset||1,256||—|
|Total operating expenses||12,179||10,923|
|Other expense, net:|
|Unrealized loss on fair value of warrants||(14,928||)||—|
|Foreign exchange loss||(12||)||(47||)|
|Other expense, net||(35||)||(1,290||)|
|Total other expense, net||(15,712||)||(1,337||)|
|Loss before income taxes||(27,891||)||(12,260||)|
|Income tax (expense) benefit||(1,594||)||55|
|Net loss attributable to non‑controlling interest||—||51|
|Net loss attributable to Strongbridge Biopharma||$||(29,485||)||$||(12,154||)|
|Net loss attributable to ordinary shareholders:|
|Basic and diluted||$||(29,485||)||$||(12,154||)|
|Net loss per share attributable to ordinary shareholders:|
|Basic and diluted||$||(0.83||)||$||(0.57||)|
|Weighted-average shares used in computing net loss per share attributable to ordinary shareholders:|
|Basic and diluted||35,335,026||21,205,382|
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 first commercial product is KEVEYIS® (dichlorphenamide), the first and only FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of Primary Periodic Paralysis. KEVEYIS has orphan drug exclusivity status in the U.S. through August 7, 2022. In addition to establishing this neuromuscular disease franchise, the Company has a clinical-stage pipeline of therapies for rare endocrine diseases. Strongbridge's lead compounds include RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing's syndrome, and veldoreotide, a next-generation somatostatin analog being investigated for the treatment of acromegaly, with potential additional applications in Cushing's syndrome and neuroendocrine tumors. Both RECORLEV and veldoreotide have received orphan designation from the U.S. Food and Drug Administration and the European Medicines Agency. For more information, visit www.strongbridgebio.com.
KEVEYIS® (dichlorphenamide) is indicated for the treatment of primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants.
KEVEYIS Important Safety Information
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.
KEVEYIS is not for everyone. Do not take KEVEYIS if you:
- Are on a high-dose aspirin regimen
- Are allergic to sulfa-based drugs
- Have liver, kidney, or certain lung conditions
- Are pregnant, planning to become pregnant, or nursing
- Are under 18 years old
Taking KEVEYIS may cause a drop in the amount of potassium (an electrolyte) in your body, which can lead to heart problems. Ask your doctor if you need to eat foods that contain high amounts of potassium while taking KEVEYIS.
Your body may produce too much acid or may not be able to remove enough acid from body fluids while taking KEVEYIS. Your doctor will run tests on a regular basis to check for signs of acid buildup and may reduce your dose or stop your treatment with KEVEYIS.
KEVEYIS may also increase the risk of falls, especially in elderly patients and patients taking high doses of KEVEYIS. Use caution when driving, operating machinery, or performing any other hazardous activities while taking KEVEYIS, as this medication may cause drowsiness.
For additional KEVEYIS important safety information, please see full prescribing information at www.keveyis.com.
STRONGBRIDGE BIOPHARMA™ is a trademark of Strongbridge Biopharma plc.
KEVEYIS® is a registered trademark licensed exclusively in the U.S. to Strongbridge Biopharma plc.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this press release, are forward-looking statements. These statements relate to future events and involve known and unknown risks, including, without limitation, uncertainties regarding Strongbridge's strategy, plans, future financial position, anticipated investments, costs and results, outcomes of product development efforts, status and results of clinical trials and objectives of management for future operations. 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. 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.
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Source:Strongbridge Biopharma plc