UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 


 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of

The Securities Exchange Act of 1934

 


 

Date of Report (Date of earliest event reported): August 8, 2018

 

STRONGBRIDGE BIOPHARMA plc

(Exact name of registrant as specified in its charter)

 

Ireland
(State or other
jurisdiction of incorporation)

 

001-37569
(Commission
File Number)

 

98-1275166
(I.R.S. Employer
Identification No.)

 

900 Northbrook Drive
Suite 200
Trevose, PA
(Address of principal executive offices)

 

19053
(Zip Code)

 

Registrant’s telephone number, including area code: (610) 254-9200

 


 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

 

o  Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

o  Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

o  Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

o  Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

 

Emerging growth company                    x

 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. x

 

 

 



 

Item 2.02              Results of Operations and Financial Condition.

 

On August 8, 2018, Strongbridge Biopharma plc (the “Company”) issued a press release reporting second quarter financial results and providing a corporate update. A copy of the press release is attached to this Current Report on Form 8-K as Exhibit 99.1.

 

The information contained in Item 2.02 of this Current Report on Form 8-K and Exhibit 99.1 is being furnished to the Commission and shall not be deemed “filed” for the purpose of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section. The information in Exhibit 99.1 shall not be incorporated by reference into any registration statement or other document pursuant to the Securities Act of 1933, as amended (the “Securities Act”), except as expressly set forth by specific reference in such filing. The information set forth herein will not be deemed an admission as to the materiality of any information required to be disclosed solely to satisfy the requirements of Regulation FD.

 

Item 7.01              Regulation FD.

 

The response to Item 2.02 is incorporated herein by reference to this Item 7.01.

 

Item 8.01              Other Information.

 

On August 8, 2018, the Company issued a press release announcing top-line results from the multinational, pivotal Phase 3 SONICS study evaluating Recorlev (levoketoconazole) for the treatment of endogenous Cushing’s syndrome.  A copy of the press release announcing the results is attached as Exhibit 99.2 and is incorporated herein by reference.

 

The Company intends to discuss the top-line data results and earnings results on a call to be held today at 8:30 a.m. Eastern Time, as described in the press release.

 

A copy of the Company’s investor presentation is attached as Exhibit 99.3.  Other than with respect to slides number 6 through 16, which are incorporated herein by reference, the investor presentation is being furnished and shall not be deemed “filed” for the purpose of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section. The information in Exhibit 99.3 (other than with respect to slides number 6 through 16) shall not be incorporated by reference into any registration statement or other document pursuant to the Securities Act, except as expressly set forth by specific reference in such filing. A copy of the investor presentation attached as Exhibit 99.3 to this Current Report on Form 8-K will also be made available on the Company’s website.

 

Item 9.01              Financial Statements and Exhibits.

 

(d)   Exhibits

 

Exhibit
Number

 

Exhibit Table

 

 

 

99.1

 

Press Release issued by Strongbridge Biopharma plc, dated August 8, 2018.

 

 

 

99.2

 

Press Release issued by Strongbridge Biopharma plc, dated August 8, 2018.

 

 

 

99.3

 

Presentation of Strongbridge Biopharma plc, dated August 8, 2018.

 

2



 

SIGNATURES

 

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

 

STRONGBRIDGE BIOPHARMA PLC

 

 

 

By:

/s/ A. Brian Davis

 

Name:

A. Brian Davis

 

Title:

Chief Financial Officer

 

 

 

Date: August 8, 2018

 

 

 

3


 

Exhibit 99.1

 

 

Strongbridge Biopharma plc Reports Second Quarter 2018 Financial Results and Provides Corporate Update

 

~ Statistically Significant Positive Top-line Clinical Results Reported from SONICS Phase 3 Study of RECORLEV™ (levoketoconazole) in Endogenous Cushing’s Syndrome ~

 

~ Rare Endocrine Franchise Expands with the U.S. Launch of MACRILENÔ (macimorelin) for the Diagnosis of Adult Growth Hormone Deficiency (AGHD) ~

 

~ Patent Issued in United States for Extended-release veldoreotide Formulation, Providing Patent Protection Until 2037 ~

 

~ KEVEYIS® (dichlorphenamide) Achieves $4.3 Million in Second Quarter 2018 Revenue, a 187 Percent Increase from Second Quarter 2017 ~

 

Dublin, Ireland and Trevose, Pa., August 8, 2018 — 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 reported second quarter 2018 financial results.

 

Second Quarter 2018 And Recent Highlights:

 

Rare Endocrine Franchise:

 

·                            Earlier today, Strongbridge reported statistically significant positive top-line clinical results from the SONICS Phase 3 study of RECORLEV™ (levoketoconazole) in endogenous Cushing’s syndrome.

·                            Launched MACRILENÔ (macimorelin) in the United States at the end of July. MACRILEN is the Company’s second rare disease commercial product and the first and only FDA-approved oral drug indicated for the diagnosis of adult growth hormone deficiency (AGHD), a condition often treated by the same endocrinologists who diagnose and treat Cushing’s syndrome.

·                           Patent issued for veldoreotide, a next-generation somatostatin analog (SSA), with claims covering an extended-release formulation and a method of manufacturing the formulation, strengthening the Company’s intellectual property portfolio. The patent is scheduled to expire in February 2037.

 

Rare Neuromuscular Franchise:

 

·                            Achieved KEVEYIS net product sales of $4.3 million in the second quarter of 2018, a 187 percent increase compared to $1.5 million in the second quarter of 2017.

·                            Full-year 2018 KEVEYIS revenue guidance remains $18 — $20 million.

 



 

“We have achieved several value-creating milestones across our rare disease portfolio over the last several months, including the compelling RECORLEV Phase 3 clinical trial results announced earlier today,” said Matthew Pauls, president and chief executive officer of Strongbridge Biopharma. “In addition to the continued strong performance of KEVEYIS, we are particularly excited about the launch of MACRILEN, which provides Strongbridge with an efficient, synergistic presence in the rare endocrine community ahead of the potential regulatory approval of RECORLEV. Early launch indicators are encouraging, signaling pent-up demand for this novel solution to diagnose adult growth hormone deficiency (AGHD), a serious, rare, under-diagnosed disease.”

 

Second Quarter 2018 Financial Results

 

For the three months ended June 30, 2018, basic net loss attributable to ordinary shareholders on a GAAP basis was $2.9 million, or $0.06 per share, compared to a basic net loss attributable to ordinary shareholders of $30.2 million, or $0.86 per share, for the same period in 2017. Net loss for the three months ended June 30, 2018 was lower than the same period in 2017 primarily due to an unrealized gain of $19.0 million on the fair value of warrants recorded in 2018, compared to an unrealized loss of $15.2 million on the fair value of warrants recorded in the same period of 2017, as well as increased net revenues recorded in 2018 from sales of KEVEYIS, offset in part by increased operating expenses associated with the commercialization of KEVEYIS and MACRILEN, higher research and development expenses primarily associated with the continued development of RECORLEV, and higher interest expense.

 

For the three months ended June 30, 2018, non-GAAP basic net loss attributable to ordinary shareholders was $16.7 million, or $0.36 per share, compared to a non-GAAP basic net loss attributable to ordinary shareholders of $12.2 million, or $0.34 per share, for the same period in 2017. The increase in non-GAAP net loss was primarily due to increased operating expenses associated with the commercialization of KEVEYIS and MACRILEN, higher research and development expenses primarily associated with the continued development of RECORLEV, and higher interest expense, offset in part by net revenues recorded from KEVEYIS product sales.

 

The Company recorded net revenues from sales of KEVEYIS of $4.3 million and cost of goods sold of $0.8 million for the three months ended June 30, 2018, compared to net revenues of $1.5 million and cost of goods sold of $0.4 million for the same period in 2017.

 

Research and development expenses were $5.5 million for the three months ended June 30, 2018, compared to $4.1 million for the same period in 2017. The increase during the 2018 period was primarily due to expenses related to the RECORLEV LOGICS and OPTICS clinical trials.

 

Selling, general and administrative expenses were $15.2 million for the three months ended June 30, 2018, compared to $10.1 million for the same period in 2017. The increase during the 2018 period was primarily due to costs incurred to establish the commercial and corporate infrastructure necessary to support the commercialization of KEVEYIS and MACRILEN.

 

Year-to-Date June 2018 Financial Results

 

For the six months ended June 30, 2018, basic net loss attributable to ordinary shareholders on a GAAP basis was $31.6 million, or $0.69 per share, compared to a basic net loss attributable to

 



 

ordinary shareholders of $59.7 million, or $1.69 per share, for the same period in 2017.  Net loss for the six months ended June 30, 2018 was lower than the same period in 2017 primarily due to an unrealized gain of $9.3 million on the fair value of warrants recorded in 2018, compared to an unrealized loss of $30.1 million on the fair value of warrants recorded in the same period of 2017, as well as increased net revenues recorded in 2018 from sales of KEVEYIS, offset in part by increased operating expenses associated with the commercialization of KEVEYIS and MACRILEN, higher research and development expenses primarily associated with the continued development of RECORLEV, and higher interest expense.

 

For the six months ended June 30, 2018, non-GAAP basic net loss attributable to ordinary shareholders was $31.0 million, or $0.67 per share, compared to a non-GAAP basic net loss attributable to ordinary shareholders of $22.5 million, or $0.64 per share, for the same period in 2017. The increase in non-GAAP net loss was primarily due to increased operating expenses associated with the commercialization of KEVEYIS and MACRILEN, higher research and development expenses primarily associated with the continued development of RECORLEV, and higher interest expense, offset in part by net revenues recorded from KEVEYIS product sales.

 

The Company recorded net revenues from sales of KEVEYIS, which was launched in April 2017, of $8.2 million and cost of goods sold of $1.4 million for the six months ended June 30, 2018, compared to net revenues of $1.5 million and cost of goods sold of $0.4 million for the same period in 2017.

 

Research and development expenses were $10.3 million for the three months ended June 30, 2018, compared to $7.6 million for the same period in 2017. The increase during the 2018 period was primarily due to expenses related to the RECORLEV LOGICS and OPTICS clinical trials.

 

Selling, general and administrative expenses were $27.6 million for the six months ended June 30, 2018, compared to $17.6 million for the same period in 2017. The increase during the 2018 period was primarily due to costs incurred to establish the commercial and corporate infrastructure necessary to support the commercialization of KEVEYIS and MACRILEN.

 

Strongbridge had $85.5 million of cash and cash equivalents and $87.4 million in outstanding debt as of June 30, 2018, compared to $57.5 million of cash and cash equivalents and $40.8 million in outstanding debt as of December 31, 2017. The Company believes the combination of existing cash resources and potential additional borrowings available under its credit facility will provide sufficient cash resources under its current operating plan, which includes the potential U.S. regulatory approval and launch of RECORLEV, to achieve consistent positive cash flows from operating activities.

 

Conference Call Details

 

Strongbridge will host a conference call on Wednesday, August 8 at 8:30 a.m. EDT. To access the live call, dial 844-285-7153 (domestic) or 478-219-0180 (international) with conference ID 2971518. The conference call will also be audio webcast from the Company’s website at www.strongbridgebio.com under the “Investor/Webcasts and Presentations” section. A replay of the call will be made available for one week following the conference call. To hear a replay of the call, dial 855-859-2056 (domestic) or 404-537-3406 (international) with conference ID 2971518.

 



 

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 MACRILEN™ (macimorelin), the first and only FDA-approved oral drug indicated for the diagnosis of adult growth hormone deficiency, RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies 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. MACRILEN has orphan drug exclusivity in the United States, and 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.

 

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 MACRILEN

 

MACRILEN® (macimorelin) is a prescription oral solution that is used to test for adult growth hormone deficiency (AGHD).  In clinical studies, the most common side effects of MACRILEN were changed sense of taste, dizziness, headache, fatigue, nausea, hunger, diarrhea, upper respiratory tract infection, feeling hot, excessive sweating, sore nose and throat, and decreased heart rate.  These are not all of the possible side effects that you may experience with MACRILEN. Call your healthcare provider for medical advice about side effects. You are encouraged to report side effects to Strongbridge at 1-855-324-8912, or to the FDA at 1-800-FDA-1088 or visit www.strongbridgebio.com/products/macrilen/.  Please see Full Prescribing Information for additional important MACRILEN safety information.

 

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 the anticipated demand for MACRILEN, our ability to achieve positive cash flows from operating activities, Strongbridge’s strategy, plans, future financial position, anticipated investments, costs and results, outcomes of

 



 

product development efforts, status and results of clinical trials, intellectual property portfolio 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, the emergence of unexpected adverse events following regulatory approval and use of the product by patients, and risks related to our ability to access funds under our credit facility.  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, 2017 and subsequent 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
[email protected]

 

Investor Relations

U.S.:

The Trout Group
Marcy Nanus
+1 646-378-2927
[email protected]

 

Europe:

First House

Geir Arne Drangeid
+47 913 10 458
[email protected]

 

USA
900 Northbrook Drive
Suite 200
Trevose, PA 19053
Tel. +1 610-254-9200
Fax. +1 215-355-7389

 



 

STRONGBRIDGE BIOPHARMA plc

Select Consolidated Balance Sheet Information (unaudited)

(in thousands, except share and per share data)

 

 

 

June 30,

 

December 31,

 

 

 

2018

 

2017

 

 

 

( in thousands)

 

Consolidated Balance Sheet Data:

 

 

 

 

 

Cash and cash equivalents

 

$

85,514

 

$

57,510

 

Total assets

 

156,212

 

103,925

 

Long-term debt, net

 

77,572

 

37,794

 

Total liabilities

 

147,349

 

115,839

 

Total stockholders’ equity (deficit)

 

8,863

 

(11,914

)

 



 

STRONGBRIDGE BIOPHARMA plc

Consolidated Statement of Operations and Comprehensive Loss (unaudited)

(in thousands, except share and per share data)

 

 

 

Three Months Ended June 30,

 

Six Months Ended June 30,

 

 

 

2018

 

2017

 

2018

 

2017

 

 

 

 

 

 

 

 

 

 

 

Consolidated Statement of Operations Data:

 

 

 

 

 

 

 

 

 

Revenues:

 

 

 

 

 

 

 

 

 

Net product sales

 

$

4,296

 

$

1,529

 

$

8,166

 

$

1,529

 

Total revenues

 

4,296

 

1,529

 

8,166

 

1,529

 

 

 

 

 

 

 

 

 

 

 

Cost and expenses:

 

 

 

 

 

 

 

 

 

Cost of sales (excluding amortization of intangible assets)

 

$

753

 

$

377

 

$

1,420

 

$

377

 

Selling, general and administrative

 

15,210

 

10,142

 

27,572

 

17,584

 

Research and development

 

5,453

 

4,128

 

10,334

 

7,609

 

Amortization of intangible assets

 

1,872

 

1,255

 

3,641

 

2,511

 

Total cost and expenses

 

23,288

 

15,902

 

42,967

 

28,081

 

Operating loss

 

(18,992

)

(14,373

)

(34,801

)

(26,552

)

Other income (expense), net:

 

 

 

 

 

 

 

 

 

Unrealized gain (loss) on fair value of warrants

 

19,017

 

(15,219

)

9,317

 

(30,147

)

Interest expense

 

(3,289

)

(737

)

(6,163

)

(1,474

)

Foreign exchange gain (loss)

 

13

 

(14

)

(7

)

(25

)

Loss on early extinguishment of debt

 

 

 

(500

)

 

Other income, net

 

329

 

60

 

509

 

25

 

Total other income (expense), net

 

16,070

 

(15,910

)

3,156

 

(31,621

)

Loss before income taxes

 

(2,922

)

(30,283

)

(31,645

)

(58,173

)

Income tax (expense) benefit

 

(1

)

92

 

(1

)

(1,502

)

Net loss

 

(2,923

)

(30,191

)

(31,646

)

(59,675

)

 

 

 

 

 

 

 

 

 

 

Net loss attributable to ordinary shareholders:

 

 

 

 

 

 

 

 

 

Basic

 

$

(2,923

)

$

(30,191

)

$

(31,646

)

$

(59,675

)

Diluted

 

$

(21,940

)

$

(30,191

)

$

(40,963

)

$

(59,675

)

Net loss per share attributable to ordinary shareholders:

 

 

 

 

 

 

 

 

 

Basic

 

$

(0.06

)

$

(0.86

)

$

(0.69

)

$

(1.69

)

Diluted

 

$

(0.43

)

$

(0.86

)

$

(0.81

)

$

(1.69

)

Weighted-average shares used in computing net loss per share attributable to ordinary shareholders:

 

 

 

 

 

 

 

 

 

Basic

 

45,829,600

 

35,335,026

 

45,728,793

 

35,335,026

 

Diluted

 

50,437,716

 

35,335,026

 

50,363,801

 

35,335,026

 

 



 

STRONGBRIDGE BIOPHARMA plc

Reconciliation of Non-GAAP Financial Measures (unaudited)

(in thousands, except share and per share data)

 

 

 

 

Three Months Ended June 30, 2018

 

 

 

Operating loss

 

Loss before
income taxes

 

Net loss
attributable to
ordinary
shareholders

 

Net loss per share
attributable to
ordinary
shareholders

 

 

 

 

 

 

 

 

 

 

 

GAAP

 

$

(18,992

)

$

(2,922

)

$

(2,923

)

$

(0.06

)

 

 

 

 

 

 

 

 

 

 

Non-GAAP Adjustments:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Amortization of intangible assets (a)

 

$

1,872

 

$

1,872

 

$

1,872

 

$

0.04

 

Stock-based compensation - Research & Development (b)

 

$

463

 

$

463

 

$

463

 

$

0.01

 

Stock-based compensation - Selling, General & Admin. (b)

 

$

1,521

 

$

1,521

 

$

1,521

 

$

0.03

 

Unrealized gain on fair value of warrants (c)

 

 

$

(19,017

)

$

(19,017

)

$

(0.41

)

Non-cash interest and debt extinguishment expenses (d)

 

 

$

1,430

 

$

1,430

 

$

0.03

 

Adjusted

 

$

(15,136

)

$

(16,653

)

$

(16,654

)

$

(0.36

)

 

 

 

Three Months Ended June 30, 2017

 

 

 

Operating loss

 

Loss before
income taxes

 

Net loss
attributable to
ordinary
shareholders

 

Net loss per share
attributable to
ordinary
shareholders

 

 

 

 

 

 

 

 

 

 

 

GAAP

 

$

(14,373

)

$

(30,283

)

$

(30,191

)

$

(0.86

)

 

 

 

 

 

 

 

 

 

 

Non-GAAP Adjustments:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Amortization of intangible asset (a)

 

$

1,255

 

$

1,255

 

$

1,255

 

$

0.04

 

Stock-based compensation - Research & Development (b)

 

$

281

 

$

281

 

$

281

 

$

0.01

 

Stock-based compensation - Selling, General & Admin. (b)

 

$

1,082

 

$

1,082

 

$

1,082

 

$

0.03

 

Unrealized loss on fair value of warrants (c)

 

 

$

15,219

 

$

15,219

 

$

0.43

 

Non-cash interest expense (d)

 

 

$

270

 

$

270

 

$

0.01

 

Non-cash income tax benefit (e)

 

 

 

$

(92

)

$

0.00

 

Adjusted

 

$

(11,755

)

$

(12,176

)

$

(12,176

)

(0.34

)

 



 

STRONGBRIDGE BIOPHARMA plc

Reconciliation of Non-GAAP Financial Measures

(Unaudited, in thousands, except share and per share data)

 

 

 

Six Months Ended June 30, 2018

 

 

 

Operating loss

 

Loss before
income taxes

 

Net loss
attributable to
ordinary
shareholders

 

Net loss per
share attributable
to ordinary
shareholders

 

 

 

 

 

 

 

 

 

 

 

GAAP

 

$

(34,801

)

$

(31,645

)

$

(31,646

)

$

(0.69

)

 

 

 

 

 

 

 

 

 

 

Non-GAAP Adjustments:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Amortization of intangible assets (a)

 

$

3,641

 

$

3,641

 

$

3,641

 

$

0.08

 

Stock-based compensation - Research & Development (b)

 

$

871

 

$

871

 

$

871

 

$

0.02

 

Stock-based compensation - Selling, General & Admin. (b)

 

$

2,801

 

$

2,801

 

$

2,801

 

$

0.06

 

Unrealized gain on fair value of warrants (c)

 

 

$

(9,317

)

$

(9,317

)

$

(0.20

)

Non-cash interest and debt extinguishment expenses (d)

 

 

$

2,662

 

$

2,662

 

$

0.06

 

Adjusted

 

$

(27,488

)

$

(30,987

)

$

(30,988

)

$

(0.67

)

 

 

 

Six Months Ended June 30, 2017

 

 

 

Operating loss

 

Loss before
income taxes

 

Net loss
attributable to
ordinary
shareholders

 

Net loss per
share attributable
to ordinary
shareholders

 

 

 

 

 

 

 

 

 

 

 

GAAP

 

$

(26,552

)

$

(58,173

)

$

(59,675

)

$

(1.69

)

 

 

 

 

 

 

 

 

 

 

Non-GAAP Adjustments:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Amortization of intangible asset (a)

 

$

2,511

 

$

2,511

 

$

2,511

 

$

0.07

 

Stock-based compensation - Research & Development (b)

 

$

498

 

$

498

 

$

498

 

$

0.01

 

Stock-based compensation - Selling, General & Admin. (b)

 

$

2,035

 

$

2,035

 

$

2,035

 

$

0.06

 

Unrealized loss on fair value of warrants (c)

 

 

$

30,147

 

$

30,147

 

$

0.85

 

Non-cash interest expense (d)

 

 

$

712

 

$

712

 

$

0.02

 

Non-cash income tax expense (e)

 

 

 

$

1,247

 

$

0.04

 

Adjusted

 

$

(21,508

)

$

(22,270

)

$

(22,525

)

$

(0.64

)

 


(a)   The effects of amortization of the intangible assets and charges related to the impairment of the intangible assets are excluded because these charges are non-cash, and we believe such exclusion facilitates investors’ ability to more accurately compare our operating results to those of our peer companies.

 

(b)   The effects of non-cash employee stock-based compensation are excluded because of varying available valuation methodologies and subjective assumptions. We believe this is a useful measure for investors because such exclusion facilitates comparison to peer companies who also provide similar non-GAAP disclosures and is reflective of how management internally manages the business.

 

(c)   The unrealized gain or loss on fair value of warrants are excluded due to the nature of this charge, which is non-cash and related primarily to the effect of changes in the company’s stock price at a point in time. We believe such exclusion facilitates investors’ ability to more accurately compare our operating results to those of our peer companies.

 

(d)   The effects of non-cash interest and debt extinguishment charges are excluded. We believe such exclusion facilitates an understanding of the effects of the debt service obligations on the Company’s liquidity and comparisons to peer group companies, and is reflective of how management internally manages the business.

 

(e)   The effect of non-cash tax expense or benefit related to valuation allowance adjustments of the deferred income tax asset is excluded because of its non-recurring nature. We believe such exclusion facilitates investor’s ability to more accurately compare our operating results to those of our peer companies.

 


Exhibit 99.2

 

Strongbridge Biopharma plc Announces Positive Top-Line Results from the Pivotal Phase 3 SONICS Study of RECORLEVÔ (levoketoconazole) for the Treatment of Endogenous Cushing’s Syndrome

 

~ SONICS Study Met Primary Endpoint with RECORLEV Demonstrating a Statistically Significant Normalization Rate of Urinary Free Cortisol at Six Months ~

 

~ Key Secondary Endpoints Evaluating Cardiovascular Risk Markers Showed Statistically Significant and Clinically Meaningful Improvements from Baseline ~

 

~ RECORLEV was Generally Well Tolerated with Only 3.2 Percent of Patients Demonstrating an Increase in Alanine Aminotransferase Measurement of Greater Than Five Times the Upper Limit of Normal ~

 

~ Strongbridge Plans to Discuss Potential for Accelerated Approval of RECORLEV with FDA ~

 

~ Company to Host Conference Call Today at 8:30 AM EDT ~

 

DUBLIN, Ireland and TREVOSE, Pa., August 8, 2018 — 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 top-line results from the multinational, pivotal Phase 3 SONICS study evaluating RECORLEVÔ (levoketoconazole) for the treatment of endogenous Cushing’s syndrome.

 

The open-label, single-arm SONICS study achieved statistical significance of its pre-specified primary endpoint, with 30 percent of patients achieving normalization of mean urinary free cortisol (UFC) following six months of maintenance treatment with RECORLEV without a dose increase (p<.025). Sensitivity analyses as well as secondary and exploratory endpoints of UFC response were supportive of the primary endpoint.

 

For key secondary endpoints of cardiovascular risk, including fasting blood glucose, hemoglobin A1C, total cholesterol, low density lipoprotein (LDL)-cholesterol, body weight and body mass index (BMI), RECORLEV demonstrated statistically significant and clinically meaningful improvements from baseline (p<.0001 for each).

 

Safety and tolerability findings based upon data collected through the six-month maintenance phase indicate that RECORLEV was generally well tolerated.

 



 

·                  12 patients (12.8 percent) discontinued treatment with RECORLEV due to adverse events (AEs). For the two most commonly reported AEs, no patients discontinued treatment due to nausea and one patient discontinued treatment due to headache.

·                  14 patients (14.9 percent) reported one or more serious adverse events (SAEs), of whom four had SAEs deemed drug related by investigators.

·                  One patient death was reported and not considered drug related (colon cancer).

 

The following tables represent the SONICS liver-related findings and the most commonly reported treatment-emergent AEs:

 

SONICS Liver-Related Findings

 

Liver-Related Findings

 

N=94

 

Liver-related AEs defined in protocol as AE of special interest

 

7.4

%*

>3X Upper Limit of Normal (ULN) (includes those > 5x ULN)

 

10.6

%

>5x ULN

 

3.2

%

Bilirubin values > 1.5x ULN

 

0

%

 


*No severe drug-induced liver injury; no Hy’s law; no transaminases >20x ULN; no obvious dose relationship (exposure relationships analyses pending).

 

SONICS Treatment-Emergent Adverse Events (15%)

 

Treatment-Emergent AE

 

Subjects > 1 AE

 

% of Enrolled (N=94)

 

Nausea

 

30

 

32

%

Headache

 

26

 

28

%

Peripheral edema

 

18

 

19

%

Hypertension

 

16

 

17

%

Fatigue

 

15

 

16

%

Diarrhea

 

14

 

15

%

Alanine Aminotransferase (ALT) increased**

 

14

 

15

%

 


**Includes all ALT increases reported as an adverse event regardless of level or relationship to drug. A subset of these ALT increased events was also reported as AEs of special interest.

 

“Given the prospective design and rigorous nature of the SONICS primary endpoint, which required patients to maintain normalized mean 24-hour UFC (2 to 4 samples) without a dose increase for six months, a 30 percent intent-to-treat response rate has significant clinical relevance. The robust effect of RECORLEV in normalizing or decreasing UFC levels was complemented by key secondary

 



 

endpoint data showing significant improvements in multiple markers for cardiovascular risk,” said Maria Fleseriu, M.D., FACE, professor of Neurological Surgery and Medicine, and director of the Oregon Health Sciences University Northwest Pituitary Center. “SONICS efficacy and safety results, including a low frequency (3.2 percent) of significant ALT elevations (more than five times above the upper limit of normal) without clinical sequalae and fully reversible, positions RECORLEV, if approved, to play an important role in individualized medical therapy for Cushing’s syndrome, including consideration as a first-line treatment.”

 

“Strongbridge would like to thank the many patients and investigators who have and are participating in the SONICS trial. The data generated from this study has and will continue to provide important insights into the understanding of Cushing’s syndrome and the use of RECORLEV. Strongbridge looks forward to working with healthcare professionals and patients to raise awareness of Cushing’s syndrome and to potentially provide a new treatment option for this disease,” said Matthew Pauls, president and chief executive officer of Strongbridge Biopharma.

 

Fred Cohen, M.D., chief medical officer of Strongbridge Biopharma said, “The impressive top-line results of the SONICS study suggest that RECORLEV, the 2S,4R enantiomer of ketoconazole, is an effective and well tolerated cortisol synthesis inhibitor in Cushing’s syndrome. Based on these compelling data, we look forward to discussing the potential for accelerated approval of RECORLEV with the FDA and to continuing our discussions with regulators around the world.”

 

About the SONICS Study

 

SONICS is an open-label, Phase 3 study of RECORLEV as a treatment for endogenous Cushing’s syndrome that enrolled 94 patients at centers in North America, Europe and the Middle East. Following a screening phase, SONICS has three treatment phases:

 

(1)         Dose Titration Phase: Patients started RECORLEV at 150 mg twice daily (300 mg total daily dose) and titrated in 150 mg increments with the goal of achieving a therapeutic dose — a dose resulting in UFC normalization — at which point titration was stopped;

 

(2)         Maintenance Phase: The dose was fixed and should not have been changed other than for safety reasons or loss of efficacy. At the end of the six-month maintenance phase, the UFC response rate was measured; and

 

(3)         Extended Evaluation Phase: Patients continue for another six months.

 

In order to be considered a UFC responder for the primary efficacy analysis, a patient must have met either three or four criteria, as applicable: (1) have completed the six-month maintenance phase; (2) have normal (at or below the ULN) mean 24-hour UFC on the basis of at least two (and up to four) adequate 24-hour urine samples; (3) must not have increased the dose of RECORLEV used during the entire maintenance phase and; (4) for patients with a recent history of pituitary radiation must have exhibited a rebound increase in mean UFC above the ULN following a two-week minimum withdrawal of RECORLEV at the end of maintenance phase.

 

Conference Call Details

 

Strongbridge will host a conference call on Wednesday, August 8 at 8:30 a.m. EDT. To access the live call, dial 844-285-7153 (domestic) or 478-219-0180 (international) with conference ID 2971518. The conference call will also be audio webcast from the Company’s website at

 



 

www.strongbridgebio.com under the “Investor/Webcasts and Presentations” section. A replay of the call will be made available for one week following the conference call. To hear a replay of the call, dial 855-859-2056 (domestic) or 404-537-3406 (international) with conference ID 2971518.

 

About Endogenous Cushing’s Syndrome

 

Endogenous Cushing’s syndrome (CS) is a rare but serious and potentially lethal endocrine disease caused by chronic elevated cortisol exposure. Most people with CS have a variety of signs and symptoms — many of which, when they occur by themselves, are common and do not necessarily point to an underlying disease; this makes recognition of CS difficult. Common presenting symptoms include weight gain or obesity, fatigue, muscle weakness, headaches, mood or sleep disturbances, facial rounding or redness, excess body hair growth in women or baldness in men, thinned skin with stretch marks, easy bruising and other skin changes including acne, mood or sleep disturbances and irregular periods or loss of libido. Patients are often found by their doctors to have new-onset or worsening of high blood pressure, abnormal levels of blood lipids, such as cholesterol, polycystic ovaries and abnormal blood glucose or diabetes. People with uncontrolled disease are seriously ill and have a 2- to 4-fold higher mortality rate than age- and gender-matched controls, mainly due to metabolic and cardiovascular complications. Treatment options for CS include surgery, radiation therapy, and medical treatment. CS most commonly affects adults ages 20-50 and is more prevalent in females, accounting for about 70 percent of all cases.

 

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 MACRILEN™ (macimorelin), the first and only FDA-approved oral drug indicated for the diagnosis of adult growth hormone deficiency, RECORLEV™ (levoketoconazole), a cortisol synthesis inhibitor currently being studied in Phase 3 clinical studies 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. MACRILEN has orphan drug exclusivity in the United States, and 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 the potential advantages of RECORLEV, the potential for RECORLEV as a first line treatment, and discussions with regulators regarding the accelerated approval of RECORLEV, Strongbridge’s strategy, plans, status and results of other 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, 2017 and subsequent 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
[email protected]

 

Investor Relations

U.S.:

The Trout Group
Marcy Nanus
+1 646-378-2927
[email protected]

 

Europe:

First House

Geir Arne Drangeid
+47 913 10 458
[email protected]

 

USA
900 Northbrook Drive
Suite 200
Trevose, PA 19053
Tel. +1 610-254-9200
Fax. +1 215-355-7389

 


Exhibit 99.3

 Strongbridge Biopharma plc August 2018

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Forward-looking statements This document contains forward-looking statements relating to the Company’s strategy, objectives, business development plans and financial position. All statements other than statements of historical facts included in this document, including, without limitation, statements regarding the Company’s future financial position, strategy, anticipated investments, costs and results, status and results of clinical trials, size of patient population, plans, outcomes of product development efforts, intellectual property portfolio and objectives of management for future operations, may be deemed to be forward-looking statements. You can identify forward-looking statements by words such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of those terms, and similar expressions that convey uncertainty or future events or outcomes. These forward-looking statements involve known and unknown risks, uncertainties, and other factors that may cause the Company’s actual results, performance, or achievements or industry results to be materially different from those contemplated, projected, forecasted, estimated or budgeted, whether expressed or implied, by these forward-looking statements. Given these risks and uncertainties, investors should not place undue reliance on forward-looking statements as a prediction of actual results. A discussion of certain of these risks may be found in the filings the Company makes with the U.S. Securities and Exchange Commission. None of these forward-looking statements constitutes a guarantee of the future occurrence of such events or of actual results. These statements are based on data, assumptions, and estimates that the Company believes are reasonable. The forward-looking statements contained in this document are made only as of the date hereof. Except as otherwise required by law, the Company expressly disclaims any obligation or undertaking to release publicly any updates of any forward-looking statements contained in this document to reflect any change in its actual results, assumptions, expectations or any change in events, factors, conditions, or circumstances on which any forward-looking statement contained in this document is based. 2

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Strongbridge Biopharma plc: Building a portfolio of therapeutically-aligned vertical franchises in rare diseases Rare Endocrine Rare Neuromuscular Rare Disease Franchise #3 MACRILEN (macimorelin) The 1st and only FDA-approved oral drug for diagnosing adult growth hormone deficiency (Orphan Drug Exclusivity) The 1st and only FDA-approved drug for Primary Periodic Paralysis, an ultra-rare genetic neuromuscular condition (Orphan Drug Exclusivity) RECORLEV Next-generation cortisol inhibitor for Cushing’s Syndrome PHASE 3 (Orphan Drug Designation) VELDOREOTIDE Extended Release Potential next-generation somatostatin analog for Acromegaly PRECLINICAL (Orphan Drug Designation) Business development opportunities Business development opportunities 3

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Recent and anticipated milestones 2H - 20181H -2019 Macrilen Commercial launch (July 2018) Quarterly sales updates RecorlevSONICS Top-line data (August 2018) SONICS Additional data (Q4 2018) SONICS Top-line one-year data (Q1 2019) LOGICS Top-line data (Q1 2019) Keveyis Quarterly sales updates Quarterly sales updates Corporate Q2 2018 & Q3 2018 earningsQ4 2018 & Q1 2019 earnings 4

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 Recorlev (levoketoconazole)

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Cushing’s syndrome complications and comorbidities Psychosis, impaired memory, sleep disturbance, depression, anxiety Heart attacks, stroke, high blood pressure, high cholesterol, vein clots Overweight/obesity, facial, neck and abdominal fat accumulation, diabetes Muscle and skin atrophy Osteoporosis Compared to those without Cushing’s disease, young patients have:1* 2-5x higher incidence rates of comorbidities (eg, cardiovascular, endocrine, musculoskeletal, and mental health conditions) 7x higher medical costs 4x higher pharmacy costs Source: Company sponsored research and published research including Feelders RA, Hofland LJ. (J Clin Endoc Metab. 2013;98(2):425-438) and Daly et al. (J Clin Endoc Metab 2006) *According to a retrospective analysis of claims from a large US commercial health plan (885 selected Cushing’s disease cases and 2,655 matched controls without Cushing’s disease) from 2007 to 2011. Burton T, et al.6 Pituitary. 2016;19:167–174.

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Phase 3 Cushing’s syndrome clinical program 94 Patients EnrolledBoth are pivotal trials to support FDA regulatory DOSE TITRATION: 2 to 21 weeks Titrate in 150 mg increments up to max 600 mg 2x daily until UFC normalization is achieved MAINTENANCE: 6 months Maintain UFC normalization with fixed therapeutic dose EXTENDED EVALUATION: 6 months Primary endpoint UFC level measured submission for approval Target 35 Patients SONICS-completers enter at Randomized Withdrawal LEVOKETOCONAZOLE-NAÏVE-ONLY DOSE TITRATION: 14 to 19 weeks RANDOMIZED WITHDRAWAL: (Active) RANDOMIZED WITHDRAWAL: (Placebo) RESTORATION PHASE Open-label extension * * OPTICS is not intended as pivotal trial. Up to 19 weeks

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SONICS Study Design: Single Arm, Open-Label Phase 3 2 – 21 weeks 6 months 6 months Dose Titration Maintenance Extended Evaluation Increase dose in 150mg increments up to max of 600mg 2x daily until UFC normalization Maintain UFC normalization for 6 months with no dose increase Primary endpoint 94 patients enrolled 77 patients entered 61 patients completed Secondary endpoints

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SONICS Patient Demographics

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Full and Partial UFC Responder Analysis At End Of Maintenance Phase Analysis based on repeated measures model. Least squares mean estimate and associated 95% confidence interval CI: 95% Confidence interval around the least squares means estimate from repeated measures model *1-sided vs. null hypothesis of 20% or lower rate **Data based on 55 maintenance phase completers with both baseline and month 6 UFC data available

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SONICS Key Secondary Endpoints at Month 6 *Hochberg adjustment applied to p-values to control type 1 error (except Body Mass Index); reductions from baseline based on least squares mean changes from repeated measures model. No significant changes observed in blood pressure or c-reactive protein; small but statistically significant decrease observed in HDL-c

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SONICS Liver-Related Lab Values *No severe drug-induced liver injury; no Hy’s law; no transaminases >20x ULN; no obvious dose relationship (exposure relationships analyses pending)

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 Most common reported AEs (>=15%, both phases combined) *Includes all alanine aminotransferase (ALT) increases reported as an adverse event regardless of level or relationship to drug. A subset of these ALT increased events was also reported as adverse events of special interest.

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Recorlev Next Steps Strongbridge intends to discuss accelerated approval and the regulatory pathway with the FDA Report one year safety and efficacy data from SONICS (Q1 2019) Complete enrollment and report results (Q1 2019) from Phase 3 randomized withdrawal LOGICS study

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Recorlev U.S. Commercial Opportunity Patient Population Estimated US diagnosed prevalence ~25,000 •If approved, position Recorlev as first-line, first-choice therapy •Based on SONICS data, Recorlev launch strategy could also include targeting off-label Active disease: addressable ~7,000 Active disease: Rx-treated ~5,000 Controlled ~3,000 Remission (often relapse) ~18,000 Active disease: Not Rx-treated ~2,000 Not-controlled ~2,000 Initial target ketoconazole switches (~2,000-3,000 patients) •Targeted pricing corridor of ~$200K-$400K per patient per year •Recorlev time to peak sales could be pulled forward due to Macrilen strategic fit Source: Company-sponsored research15

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 Macrilen (macimorelin)

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Macrilen Macrilen is an oral ghrelin receptor agonist (secretagogue) administered to stimulate growth hormone production FDA approval in December 2017 for use in the diagnosis of patients with AGHD Strongbridge acquired U.S. and Canadian rights in January 2018 and launched Macrilen in the United States in July 2018 17

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Adult growth hormone deficiency (AGHD) Symptoms Causes • Body composition altered: • Increased fat mass • Decreased lean body mass • Reduced skeletal muscle strength • Cardiovascular (CV) issues: • Increased risk of CV death • Abnormal lipids, other risk markers • Bone mineral density: • Increased fracture rate • Overall: • Decrease quality of life • Low physical/mental energy • Tumors • Surgery • Cranial radiation • Infectious / inflammatory • Trauma / vascular injury • Childhood onset (idiopathic) Growth hormone treatment has been shown in clinical trials to improve health for adults with GHD

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 Many patients are not screened for AGHD due to the complexity, tolerability and safety issues of the current tests NOT FDA APPROVED 1. Yuen KCJ. South Dartmouth, MA; Endotext [Internet]. https://www.ncbi.nlm.nih.gov/books/NBK395585/. Accessed February 19, 2018. 2. Agrawal V, Garcia JM. The macimorelin-stimulated growth hormone test for adult growth hormone deficiency diagnosis. Expert Rev Mol Diag. 2014;14(6):647-654. 3. Leong KS, et al. Clin Endocrinol. 2001;54: 463-468. 4. Post-Traumatic Hypopituitarism – Who Should be Screened, When, and How, Mark Quinn and Amar Agha

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 Macrilen: The 1st and only FDA-approved oral drug for diagnosing adult growth hormone deficiency (AGHD)

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Macrilen accurately diagnoses AGHD: comparison to ITT Insulin Tolerance Test (ITT) (GH cutoff 5.1 ng/mL) Positive Negative Total Tests Macrilen (GH cutoff 2.8 ng/mL) Positive 55 4 59 Negative 19 62 81 Total 74 66 140 AGRE EMENT 74%* 94%* 84% •Positive agreement higher (89%) for high-risk AGHD category •Negative agreement 86-94% •Labeling reflective of overall and risk-stratified agreements •<1% of Macrilen tests were not evaluable vs. 17% of ITTs •Macrilen was highly reproducible (91%) in the same patient Negative Agreement: 93.94% (CI: 85.20%, 98.32%)-Met pre-defined performance criterion (i.e. excluded negative agreement with ITT of less than 75% with 95% confidence), Positive Agreement: 74.32% (CI: 62.84%, 83.78%)-Did not meet pre-defined performance criterion (i.e. could not exclude positive agreement with ITT of less than 70% with 95% confidence) * Co-Primary efficacy endpoints; a positive test is one that fails to reach the respective test’s GH cutoff 21

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Macrilen Safety Summary •Macrilen well tolerated as compared to ITT (see table) •Most common adverse reactions with Macrilen: •Dysgeusia (4.5%); dizziness, fatigue, and headache (3.9% each); nausea (3.2%) •Most adverse reactions were mild; none severe or led to failure to complete test •QTcF upper 90% confidence limit about 11 ms at doses 2x-4x higher than used for GH stimulation (NO hERG inhibition) •Concomitant QT-prolonging drugs should be avoided •NO need to perform ECG before or after use of Macrilen •DDI potential: Discontinue strong CYP3A4 inducers (avoids false-positive results) 22

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Macrilen Market Opportunity 40-60k Annual Tests* 33% increase in AGHD testing** TBI’s 2.8M per year*** (on-label: no further clinical studies needed) Pre-Macrilen Potential Post-Macrilen Growth Opportunity • Expected increase in testing for AGHD because of Macrilen WAC price per unit of $4,500 • All patients with moderate to severe traumatic brain injury (TBI) require evaluation of pituitary function. *Oppenheimer 2011—manufacturer-sponsored research; Navigant, 2009—manufacturer-sponsored research; Symphony, 2017—manufacturer-sponsored research; TVG Research—manufacturer-sponsored research, 2017; Lumelian, 2017—manufacturer-sponsored research. **Results of quantitative (n=40 endocrinologists) and qualitative (n=5) market research conducted by a 3rd party sponsored by Strongbridge (2017). ***https://www.cdc.gov/traumaticbraininjury/severe.html. 23

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Flexible and Scalable Distribution & Services Model Third-Party Logistics Specialty Distributors Specialty Wholesale Distribution HOPD*, Physician Clinic, VA Specialty Pharmacy Network Macrilen Service Center Reimbursement and Order Triage Specialty Pharmacy Distribution Inbound Service Requests Physician Practice *Hospital Outpatient Department 24

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 Veldoreotide Extended Release

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 Veldoreotide extended release: a novel, multi-receptor somatostatin analog BACKGROUND Acquired immediate-release formulation of veldoreotide in 2015 and focused initial R&D on long-acting reformulation 10/2016: Successfully formulated for convenient, at-home, subcutaneous administration using PLGA microspheres Formulation patent issued in U.S. for extended-release veldoreotide; patent protection to 2037 SSAs: Somatostatin Analogs Data through Phase IIa: potential differentiated benefits from currently approved somatostatin analogues Comparable maximal GH suppression to octreotide Reduced impact on gallbladder function, bile acid production, and GI motility in rodents Reduced impact on hormonal responses to mixed meals in healthy subjects 26

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 Keveyis (dichlorphenamide) The first and only FDA-approved therapy for primary periodic paralysis* * FDA-approved treatment for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis

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 Primary periodic paralysis: a spectrum of rare, chronic, genetic, neuromuscular disorders PPPSymptoms/triggersImpact of attacks Frequency Causes recurrent, progressive, and debilitating episodes of muscle weakness and temporary paralysis2-4 ~4-5k patients in the U.S. 5 Symptoms: clumsiness, extreme fatigue, weakness, palpitations, pain Triggers: may include potassium, carbohydrates, rest after exercise, cold exposure, stress Paralytic attacks are acute episodes that can be debilitating4 Attacks may last from one hour to several days1 As patients age, muscle weakness can become permanent3 59% have weekly attacks 28% have daily attacks Sources: 1. Charles G, Zheng C, Lehmann-Horn F, Jurkatt-Rott, Levitt J. Characterization of hyperkalemic periodic paralysis: a survey of genetically diagnosed individuals. J Neurol. 2013;260:2606-2613. 2. Cannon SC. Channelopathies of skeletal muscle excitability. Compr Physiol. 2015;5:761-790. 3. Cavel-Greant D, Lehmann-Horn F, Jurkat-Rott K. The impact of permanent muscle weakness on quality of life in periodic paralysis: a survey of 66 patients. Acta Myol. 2012;31:126-133. 4. Sansone V, Meola G, Links TP, Panzeri M, Rose MR. Treatment for periodic paralysis. Cochrane Database Syst Rev.28 2008; Jan 23;(1):CD005045. 5. Based on Strongbridge Biopharma analysis of medical claims database

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Keveyis revenue Q2 2018 actual FY 2018 guidance $4.3m $18-20m

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 About Strongbridge

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 The management team is highly experienced in managing orphan and ultra-rare disease assets

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Intellectual property and orphan exclusivity

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Strong Balance Sheet to Support Growth Objectives $86m Cash $87m Debt $205m Market Capitalization 46.7m ordinary shares 64.3m fully diluted shares Sufficiently capitalized with cash and potential additional borrowings to fund operations through Recorlev approval and launch to cash flow positive Cash, debt and shares as of June 30, 2018. Market capitalization as of August 7, 2018

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 Strongbridge Biopharma plc

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