Tesaro Announces Second-Quarter 2016 Operating Results

On August 4, 2016 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for second-quarter 2016 and provided an update on the Company’s marketed product and development programs (Press release, TESARO, AUG 4, 2016, View Source [SID:1234514313]).

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"The second quarter of 2016 was an exciting time for TESARO, marked by the availability of positive clinical results from our NOVA trial of niraparib. These results are the first and only Phase 3 data for a PARP inhibitor in a population of patients with platinum-sensitive, recurrent ovarian cancer," said Lonnie Moulder, CEO of TESARO. "Looking ahead, we intend to leverage these landmark data to enhance our ongoing ovarian cancer trials and expand the overall development strategy for niraparib. We are globalizing our mission of delivering transformative cancer therapies to patients, and are now expanding our commercial footprint in Europe to support planned launches of oral rolapitant and niraparib next year. Finally, our immuno-oncology portfolio continues to rapidly advance, and our antibody candidates targeting TIM-3 and PD-1 are now in Phase 1 studies."

Recent Business Highlights

The U.S. launch of VARUBI continues, and unit volume increased by 30% for the second quarter compared to the first quarter. For the month of June, VARUBI achieved a 27% market share in the oral NK-1 market in the U.S.
The New Drug Application (NDA) for an intravenous (IV) formulation of rolapitant was accepted for review by the Food and Drug Administration (FDA), and a U.S. launch is planned for the first half of 2017.
The Marketing Authorisation Application (MAA) for oral rolapitant is under review by the European Medicines Agency (EMA). In order to support a launch of niraparib and oral rolapitant in 2017, TESARO is now expanding its commercial operations in key European countries.
In June, TESARO and the European Network for Gynaecological Oncological Trial groups (ENGOT) announced that the Phase 3 NOVA trial of niraparib successfully achieved its primary endpoint of progression-free survival (PFS). This trial demonstrated that niraparib significantly prolonged PFS compared to control for patients in both the germline BRCA mutation (gBRCAmut) cohort and the non-germline BRCA mutation (non-gBRCAmut) cohort. These data will support NDA and MAA submissions during the fourth quarter of 2016.
Ongoing trials of niraparib in combination with other agents include the TOPACIO trial (formerly KEYNOTE-162) with KEYTRUDA (pembrolizumab) in patients with ovarian cancer or with triple negative breast cancer, and the AVANOVA trial with bevacizumab in patients with ovarian cancer.
The Phase 1 dose escalation study of TSR-022, our anti-TIM-3 antibody candidate, has been initiated, and the Phase 1 study of TSR-042, our anti-PD-1 antibody candidate, continues to enroll patients.
TESARO completed a public offering of common stock in July 2016, resulting in approximately $409 million in net proceeds.
Second Quarter 2016 Financial Results

TESARO reported a net loss of $58.4 million, or ($1.28) per share, for the second quarter of 2016, compared to a net loss of $60.6 million, or ($1.51) per share, for the second quarter of 2015.

Revenue recognition for certain sales of VARUBI to specialty distributors continues to be deferred at this point in the commercial launch. Net product revenue for the second quarter of 2016 totaled $1.4 million and included sales of VARUBI from specialty pharmacy customers to patients that were made during the second quarter, as well as sales from specialty distributors to providers that occurred in the first quarter of 2016 and the fourth quarter of 2015. The specialty pharmacy sales made during the second quarter represented a small portion of total product shipments. License revenue for the second quarter of 2016 totaled $35.1 million and included an up-front payment of $35 million from Janssen related to initiation of the global niraparib prostate cancer collaboration.

Research and development expenses increased to $50.1 million for the second quarter of 2016, compared to $38.9 million for the second quarter of 2015, driven primarily by higher costs related to the ongoing registration trials of niraparib, manufacturing costs associated with IV rolapitant and niraparib, and the advancement of our immuno-oncology portfolio, in addition to increased headcount.

Selling, general and administrative expenses increased to $36.2 million for the second quarter of 2016, compared to $16.8 million for the second quarter of 2015, primarily due to increased commercial headcount, including the establishment of a U.S. field sales organization in the fall of 2015, commercial activities in support of the launch of VARUBI, and higher professional service fees.

Acquired in-process research and development expenses totaled $4.0 million for the second quarter of 2016 and included a milestone payment related to our immuno-oncology portfolio, compared to $1.0 million for the second quarter of 2015, which also related to a development milestone achieved within our immuno-oncology programs. Operating expenses, as described above, include total non-cash, stock-based compensation expense of $11.7 million for the second quarter of 2016, compared to $5.5 million for the second quarter of 2015.

As of June 30, 2016, TESARO had approximately $320 million in cash and cash equivalents. This cash and cash equivalents total excludes the $409 million in net proceeds resulting from a follow-on offering of 5.3 million shares of common stock that was completed in July 2016.

For the quarter ended June 30, 2016, TESARO had approximately 45.8 million shares outstanding on a weighted average basis. As of July 7, following completion of the follow-on offering on that date, TESARO had approximately 51.4 million outstanding shares of common stock.

In anticipation of four product launches in 2017, TESARO will invest in pre-launch inventory manufacturing, development of supply chain capabilities and capacity, and expansion of European and targeted U.S. commercial operations, in addition to making milestone payments for regulatory submissions. As a result of these investments, the Company expects its cash and cash equivalents balance to decline by approximately $100 million on average, per quarter, during the third and fourth quarters of 2016.

Corporate Objectives

The following is a summary of TESARO’s key objectives:

VARUBI / Rolapitant:

Achieve #1 market share position within the oral NK-1 receptor antagonist market by year-end 2016 in the U.S.;
Launch IV rolapitant into the U.S. market in 1H 2017, pending FDA approval;
Establish a European commercial organization; and
Launch oral rolapitant in Europe in 1H 2017, pending EMA approval.
Niraparib:

Present NOVA data at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) annual congress in Copenhagen in October;
Submit the niraparib NDA and MAA in Q4 2016;
Launch niraparib in the U.S. in 1H 2017 and in Europe in 2H 2017, pending regulatory approval;
Submit sNDA and MAA based upon QUADRA data in mid-2017;
Report Phase 3 BRAVO data in 2H 2017;
Finalize a potential lung cancer registration strategy and initiate development program in 1H 2017; and
Determine the potential registration strategy for niraparib plus an anti-PD-1 antibody in ovarian cancer and triple-negative breast cancer in mid-2017.
Immuno-Oncology Portfolio:

Identify a dose and schedule for TSR-042 by the end of 2016;
Select at least one bispecific antibody clinical candidate by the end of 2016;
Identify the first clinical candidate within the MD Anderson collaboration in 1H 2017;
Initiate a Phase 1 study of TSR-033 (anti-LAG-3 antibody) in 1H 2017;
Finalize the TSR-042 registration strategy and initiate a registration program in 1H 2017; and
Initiate a Phase 1 clinical trial of TSR-022 in combination with an anti-PD-1 antibody in mid-2017.

Stemline Therapeutics Reports Second Quarter 2016 Financial Results and Highlights Recent Clinical Progress

On August 4, 2016 Stemline Therapeutics, Inc. (Nasdaq:STML) reported financial results for the quarter ended June 30, 2016 (Press release, Stemline Therapeutics, AUG 4, 2016, View Source [SID:1234514305]). Clinical highlights and milestones include:

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SL-401 in blastic plasmacytoid dendritic cell neoplasm (BPDCN)

Oral presentations of Phase 2 clinical trial results of SL-401 in patients with BPDCN delivered at annual meetings of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper).
SL-401 efficacy and safety data continue to be positive with increasing treatment duration, drug exposure, and patient enrollment.
As of July 2016, 29 BPDCN patients have received SL-401, including 26 adults (16 first-line and 10 relapsed/refractory) and 3 pediatric compassionate use patients.
SL-401 continues to maintain high overall response rates (ORR) in both first-line (>90% ORR) and relapsed/refractory adult BPDCN (>50% ORR). Notably, a relapsed/refractory BPDCN patient was recently bridged to stem cell transplant (SCT) following a major response to SL-401.
Response duration, progression free and overall survival data continue to mature and trend favorably. Multiple patients remain progression free after receiving ongoing SL-401 treatment (up to 12+ months [16+ cycles], ongoing) or experiencing a major response on SL-401 and proceeding to SCT (up to 16+ months, ongoing).
Further clinical and regulatory updates expected this year.
SL-401 in additional malignancies

Completed lead-in, dose escalation stage of Phase 2 trial in patients with acute myeloid leukemia (AML) in remission with high relapse risk including minimal residual disease (MRD). There were no unexpected side effects and no dose limiting toxicities. The expansion stage is currently enrolling MRD+ AML patients at 12 ug/kg.
Patients are currently enrolling in the lead-in, dose escalation stage of a Phase 2 trial in advanced, high risk myeloproliferative neoplasms (MPN). The first two dosing cohorts have been cleared with no unexpected side effects and no dose limiting toxicities. Patients are currently enrolling in the third dosing cohort, 12 ug/kg.
Patients are currently enrolling in a Phase 1/2 trial in relapsed or refractory multiple myeloma with SL-401 in combination with pomalidomide and dexamethasone (POM+DEX); the first combination trial of SL-401 with another agent. No unexpected side effects and no dose limiting toxicities have been observed with the combination to date. Enrollment continues.
Further clinical updates expected later this year from these trials.
SL-701

Phase 2 trial of SL-701 in combination with bevacizumab in adult patients with second-line GBM currently enrolling patients.
Trial updates expected later this year.
SL-801

Phase 1 trial of SL-801 in patients with advanced solid tumors currently enrolling. The first and second dosing cohorts have cleared, and the third cohort is currently enrolling.
Trial updates expected later this year and on into next year.
Ivan Bergstein, M.D., Stemline’s Chief Executive Officer, commented, "We are making substantial progress across our entire clinical pipeline this year. A key highlight of the quarter was the high profile oral presentations of positive results from our ongoing Phase 2 potentially pivotal trial of SL-401 in BPDCN at both the ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) annual meetings." Dr. Bergstein continued, "The efficacy and safety data from this trial continue to be strong with increasing treatment duration and patient exposure. We look forward to additional clinical and regulatory updates this year, including more clarity around our registration pathway and timeline."

Dr. Bergstein concluded, "As we continue to build upon our compelling data in BPDCN, we also look forward to further updates later this year regarding our multiple enrolling trials with SL-401 in additional hematologic cancers, SL-701 in brain cancer, and SL-801 in advanced solid tumors. Importantly, our strong cash position provides us with the resources to reach important milestones across all of our programs this year and beyond."

Second Quarter 2016 Financial Results Review
Stemline ended the second quarter of 2016 with $81.2 million in cash, cash equivalents and investments, as compared to $87.8 million as of March 31, 2016, which reflects a cash burn of $6.6 million for the quarter.

For the second quarter of 2016, Stemline had a net loss of $9.3 million, or $0.52 per share, compared with a net loss of $10.2 million, or $0.58 per share, for the same period in 2015.

Research and development expenses were $6.9 million for the second quarter of 2016, which reflects a decrease of $1.3 million, or 16%, compared with $8.2 million for the second quarter of 2015. The lower expenses year over year were primarily attributable to an increase in costs in the prior year relating to clinical site startup expenses and initiation of clinical trial activities.

General and administrative expenses were $2.9 million for the second quarter of 2016, which reflects an increase of $0.7 million, or 33%, compared with $2.2 million for the second quarter of 2015. The increase in expense year over year was primarily attributable to higher non-cash stock based compensation and payroll costs relating to employees.

Regeneron Reports Second Quarter 2016 Financial and Operating Results

On August 4, 2016 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the second quarter of 2016 and provided a business update (Press release, Regeneron, AUG 4, 2016, View Source [SID:1234514303]).

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Financial Highlights

($ in millions, except per share data)

Three Months Ended
June 30,

2016

2015*

% Change
EYLEA U.S. net product sales

$
831

$
655

27
%
Total revenues

$
1,213

$
999

21
%
GAAP net income

$
196

$
195

1
%
GAAP net income per share – diluted

$
1.69

$
1.69


%
Non-GAAP net income(2)

$
329

$
265

24
%
Non-GAAP net income per share – diluted(2)

$
2.82

$
2.27

24
%

* See Table 3 of this press release for an explanation of revisions made to 2015 non-GAAP amounts previously reported.

"In the first half of this year, EYLEA continued to demonstrate strong sales growth, and Praluent sales made steady progress as healthcare providers become more familiar with this new therapeutic class and learn to navigate payer utilization management criteria," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "In the second half of the year, for sarilumab in rheumatoid arthritis, we look forward to the upcoming U.S. regulatory decision and potential launch. We also recently completed a U.S. regulatory submission for dupilumab for the treatment of atopic dermatitis and are working to bring this breakthrough therapy to patients as soon as possible."

Business Highlights

Marketed Product Update

EYLEA (aflibercept) Injection for Intravitreal Injection

In the second quarter of 2016, net sales of EYLEA in the United States increased 27% to $831 million from $655 million in the second quarter of 2015. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.
Bayer commercializes EYLEA outside the United States. In the second quarter of 2016, net sales of EYLEA outside of the United States(1) were $486 million, compared to $338 million in the second quarter of 2015. In the second quarter of 2016, Regeneron recognized $167 million from its share of net profit from EYLEA sales outside the United States, compared to $107 million in the second quarter of 2015.
Praluent (alirocumab) Injection for the Treatment of Elevated Low-Density Lipoprotein (LDL) Cholesterol

In the second quarter of 2016, global net sales of Praluent were $24 million. Product sales for Praluent are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Praluent. Praluent was launched in the United States in the third quarter of 2015 and in certain countries in the European Union commencing in the fourth quarter of 2015.
In the second quarter of 2016, the U.S. Food and Drug Administration (FDA) accepted for review a supplemental Biologics License Application (sBLA) for a monthly dosing regimen of Praluent, with a target action date of January 24, 2017.
In July 2016, the Japanese Ministry of Health, Labour and Welfare granted marketing and manufacturing authorization for Praluent for the treatment of uncontrolled LDL cholesterol, in certain adult patients with hypercholesterolemia at high cardiovascular risk.
The ODYSSEY OUTCOMES trial remains ongoing, and is assessing the potential of Praluent to demonstrate cardiovascular benefit.
Pipeline Progress
Regeneron has fifteen product candidates in clinical development. These consist of EYLEA and fourteen fully human monoclonal antibodies generated using the Company’s VelocImmune technology, including four in collaboration with Sanofi. In addition to EYLEA and Praluent, highlights from the antibody pipeline include:

Sarilumab, the Company’s antibody targeting IL-6R for rheumatoid arthritis, is currently being studied in the global Phase 3 SARIL-RA program.

In December 2015, the FDA accepted for review a Biologics License Application (BLA) for sarilumab, with a target action date of October 30, 2016.
In July 2016, the European Medicines Agency (EMA) accepted for review the Marketing Authorization Application (MAA) for sarilumab.
Dupilumab, the Company’s antibody that blocks signaling of IL-4 and IL-13, is currently being studied in atopic dermatitis, asthma, nasal polyps, and eosinophilic esophagitis.

A BLA for atopic dermatitis was recently submitted to the FDA.
In April 2016, the Company and Sanofi reported that the Phase 3 LIBERTY AD SOLO 1 and SOLO 2 trials evaluating dupilumab in adult patients with inadequately controlled moderate-to-severe atopic dermatitis met their primary endpoints.
In June 2016, the Company and Sanofi reported that the Phase 3 LIBERTY AD CHRONOS trial evaluating dupilumab with topical corticosteroids in adult patients with inadequately controlled moderate-to-severe atopic dermatitis met its primary and key secondary endpoints.
Fasinumab, the Company’s antibody targeting Nerve Growth Factor (NGF), is currently being studied in patients with pain due to osteoarthritis (Phase 3) and chronic low back pain (Phase 2b/3).

In May 2016, the Company reported top-line results from a Phase 2/3 study evaluating fasinumab in patients with moderate-to-severe osteoarthritis pain of the hip or knee who have a history of inadequate pain relief or intolerance to current analgesic therapies. The study met its primary endpoint at 16 weeks.
REGN2810, an antibody to programmed cell death protein 1 (PD-1), entered a potentially pivotal clinical study for the treatment of advanced cutaneous squamous cell carcinoma in the second quarter of 2016.

Evinacumab is an antibody to Angptl-3. In May 2016, the Company reported positive interim results from an ongoing proof-of-concept study in patients with homozygous familial hypercholesterolemia (HoFH).

REGN3470-3471-3479 is a combination of antibodies to Ebola virus. A Phase 1 clinical study in healthy volunteers was initiated in the second quarter of 2016. In addition, in the second quarter of 2016, the FDA granted orphan-drug designation to REGN3470-3471-3479 for the treatment of Ebola virus infection.

REGN2477 is an antibody to Activin A being developed for Fibrodysplasia Ossificans Progressiva (FOP). A Phase 1 clinical study was initiated in the second quarter of 2016 in healthy volunteers.

Select Upcoming 2016 Milestones

Clinical Programs
Milestones
REGN2176-3 (PDGFR-beta Antibody co-formulated with aflibercept)
Report results from Phase 2 study
Praluent
Data Monitoring Committee interim analysis of ODYSSEY OUTCOMES trial
Ongoing launch in additional countries
Sarilumab (IL-6R Antibody)
FDA target action date of October 30, 2016
File for additional regulatory approvals outside the United States
Dupilumab (IL-4R Antibody)
FDA to provide target action date related to BLA submission for atopic dermatitis in the United States
Complete patient enrollment in Phase 3 asthma trial
Initiate Phase 3 study in pediatric patients in atopic dermatitis

Business Development Update

In April 2016, the Company and Intellia Therapeutics, Inc. entered into a license and collaboration agreement to advance CRISPR/Cas gene-editing technology for in vivo therapeutic development. In addition to the discovery, development and commercialization of new therapies, the companies will focus on technology development of the CRISPR/Cas platform. In May 2016, Intellia completed an initial public offering of its common stock and the Company purchased $50.0 million of Intellia common stock in a concurrent private placement.
In July 2016, the Company and Adicet Bio, Inc. entered into a license and collaboration agreement to develop next-generation engineered immune-cell therapeutics with fully human chimeric antigen receptors and T-cell receptors directed to disease-specific cell surface antigens in order to enable the precise engagement and killing of tumor cells.
Second Quarter 2016 Financial Results

Product Revenues: Net product sales were $834 million in the second quarter of 2016, compared to $658 million in the second quarter of 2015. EYLEA net product sales in the United States were $831 million in the second quarter of 2016, compared to $655 million in the second quarter of 2015.

Total Revenues: Total revenues, which include product revenues described above, increased by 21% to $1,213 million in the second quarter of 2016, compared to $999 million in the second quarter of 2015. Total revenues also include Sanofi and Bayer collaboration revenues of $355 million in the second quarter of 2016, compared to $329 million in the second quarter of 2015. Collaboration revenues in the second quarter of 2016 increased primarily due to an increase in the Company’s net profit from commercialization of EYLEA outside the United States and reimbursement of the Company’s research and development expenses and amortization of up-front payments received in connection with the Company’s July 2015 immuno-oncology collaboration with Sanofi, partly offset by lower reimbursement of the Company’s research and development expenses and an increase in the Company’s share of losses primarily from the commercialization of Praluent under the Company’s antibody collaboration with Sanofi.

Refer to Table 4 for a summary of collaboration revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $560 million in the second quarter of 2016, compared to $390 million in the second quarter of 2015. The higher R&D expenses in the second quarter of 2016 were principally due to the $75 million up-front payment made in connection with the April 2016 license and collaboration agreement with Intellia, higher development costs primarily related to fasinumab and REGN2810, and higher headcount to support the Company’s increased R&D activities, partly offset by lower development costs primarily related to dupilumab. In addition, in the second quarter of 2016, R&D-related non-cash share-based compensation expense was $79 million, compared to $60 million in the second quarter of 2015.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $292 million in the second quarter of 2016, compared to $175 million in the second quarter of 2015. The increase was primarily due to higher commercialization-related expenses in connection with EYLEA and Praluent, and higher headcount. In addition, in the second quarter of 2016, SG&A-related non-cash share-based compensation expense was $48 million, compared to $32 million in the second quarter of 2015.

Cost of Goods Sold (COGS): GAAP COGS was $41 million in the second quarter of 2016, compared to $61 million in the second quarter of 2015. COGS primarily consists of royalties as well as costs in connection with producing U.S. EYLEA commercial supplies, and various start-up costs in connection with the Company’s Limerick, Ireland commercial manufacturing facility. COGS decreased principally due to a decrease in royalties since the Company’s obligation to pay Genentech based on sales of EYLEA ended in May 2016.

Income Tax Expense: In the second quarter of 2016, GAAP income tax expense was $96 million and the effective tax rate was 32.9%, compared to $133 million and 40.7% in the second quarter of 2015. The effective tax rate for the second quarter of 2016 was positively impacted, compared to the U.S. federal statutory rate, by the tax benefit associated with stock-based compensation, the domestic manufacturing deduction, and the federal tax credit for increased research activities, partly offset by the negative impact of losses incurred in foreign jurisdictions with rates lower than the federal statutory rate and the non-tax deductible Branded Prescription Drug Fee. As described in Table 3 of this press release, the Company adopted Accounting Standards Update 2016-09 (ASU 2016-09), Compensation – Stock Compensation, Improvements to Employee Share-Based Payment Accounting, during the second quarter of 2016. ASU 2016-09 requires companies to recognize all excess tax benefits and tax deficiencies in connection with stock-based compensation as income tax expense or benefit in the income statement (previously, excess tax benefits were recognized in additional paid-in capital on the balance sheet).

GAAP and Non-GAAP Net Income: The Company reported GAAP net income of $196 million, or $1.88 per basic share and $1.69 per diluted share, in the second quarter of 2016, compared to GAAP net income of $195 million, or $1.89 per basic share and $1.69 per diluted share, in the second quarter of 2015.

The Company reported non-GAAP net income of $329 million, or $3.15 per basic share and $2.82 per diluted share, in the second quarter of 2016, compared to non-GAAP net income of $265 million, or $2.58 per basic share and $2.27 per diluted share, in the second quarter of 2015.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

2016 Financial Guidance(3)

The Company’s updated full year 2016 financial guidance consists of the following components:

EYLEA U.S. net product sales
20% – 25% growth over 2015 (reaffirmed)
Sanofi reimbursement of Regeneron commercialization-related expenses
$310 million – $340 million
(previously $320 million – $370 million)
Non-GAAP unreimbursed R&D(2) (4)
$970 million – $1.01 billion
(previously $875 million – $950 million)
Non-GAAP SG&A(2) (4)
$980 million – $1.02 billion
(previously $925 million – $1.0 billion)
Effective tax rate
33% – 41%
Capital expenditures
$480 million – $530 million
(previously $550 million – $625 million)

(1)
Regeneron records net product sales of EYLEA in the United States. Outside the United States, EYLEA net product sales comprise sales by Bayer in countries other than Japan and sales by Santen Pharmaceutical Co., Ltd. in Japan under a co-promotion agreement with an affiliate of Bayer. The Company recognizes its share of the profits (including a percentage on sales in Japan) from EYLEA sales outside the United States within "Bayer collaboration revenue" in its Statements of Operations.

(2)
This press release uses non-GAAP net income, non-GAAP net income per share, non-GAAP unreimbursed R&D, and non-GAAP SG&A, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles ("GAAP"). These non-GAAP financial measures are computed by excluding certain non-cash and other items from the related GAAP financial measure. Non-GAAP adjustments also include the income tax effect of reconciling items.

The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company’s control, such as the Company’s stock price on the dates share-based grants are issued. Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. Additionally, such non-GAAP measures provide investors with an enhanced understanding of the financial performance of the Company’s core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company’s non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by Regeneron should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP. A reconciliation of the Company’s historical GAAP to non-GAAP results is included in Table 3 of this press release.

(3)
The Company’s 2016 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

(4)
A reconciliation of full year 2016 non-GAAP to GAAP financial guidance is included below:

Projected Range
(In millions)

Low

High
GAAP unreimbursed R&D (5)

$
1,390

$
1,450

R&D: Non-cash share-based compensation expense

(320)

(340)

R&D: Upfront payments related to license and
collaboration agreements

(100)

(100)

Non-GAAP unreimbursed R&D

$
970

$
1,010

GAAP SG&A

$
1,205

$
1,275

SG&A: Non-cash share-based compensation expense

(225)

(255)

Non-GAAP SG&A

$
980

$
1,020

(5)
Unreimbursed R&D represents R&D expenses reduced by R&D expense reimbursements from the Company’s collaborators and/or customers.

Raptor Pharmaceutical Corp. Reports Second Quarter 2016 Financial Results

On August 4, 2016 Raptor Pharmaceutical Corp. (NASDAQ:RPTP), a biopharmaceutical company developing and commercializing transformative treatments for rare diseases, reported its financial results for the second quarter of 2016 and provided an update on recent corporate developments (Press release, Raptor Pharmaceutical, AUG 4, 2016, View Source [SID:1234514301]).

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Summary

Global net product revenue was $32.0 million for the second quarter ended June 30, 2016, a 37.3% increase compared to $23.3 million for the same period in 2015.

Net loss on a GAAP basis was $14.0 million, or $0.16 per share, for the second quarter of 2016 compared to a GAAP net loss of $13.9 million, or $0.17 per share, for the same period in 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, was $11.3 million, or $0.13 per share, for the second quarter of 2016 compared to a non-GAAP net loss of $9.6 million, or $0.12 per share, for the same period in 2015.

Cash and cash equivalents were $124.2 million as of June 30, 2016.

Raptor is raising its 2016 global net revenue guidance to $125 to 135 million from $115 to $125 million and affirming its guidance for non-GAAP operating expenses, excluding non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, of between $125 and $135 million.

"I’m delighted that we delivered another record quarter for sales, driven primarily by growing patient demand for PROCYSBI ," said Julie Anne Smith, President and CEO of Raptor. "For the first time, PROCYSBI revenue was augmented by sales of QUINSAIR, which is off to a terrific launch in Europe. Based on our outstanding commercial performance, we are pleased to raise our 2016 revenue guidance. We look forward to continued growth from both products and supporting patients living with rare diseases and with limited options."

Second Quarter 2016 Business Highlights

In April, Raptor commenced the first commercial sales of QUINSAIR (levofloxacin inhalation solution) in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis. To date, approval for reimbursement for QUINSAIR has been achieved in numerous European countries.

In June, Raptor participated in the 39th European Cystic Fibrosis Conference and presented new analyses of data from its Phase 3 clinical study (MPEX-209) comparing QUINSAIR to inhaled tobramycin solution in patients with cystic fibrosis and chronic Pseudomonas aeruginosa infections. The data suggest that subjects with three or more pulmonary exacerbations in the prior year who were randomized to receive QUINSAIR had a significantly lower incidence of pulmonary exacerbations when compared with their peers who were randomized to receive the active comparator, tobramycin inhalation solution (p=0.026).

Second Quarter 2016 Financial Results

Raptor provides non-GAAP financial measures, which it believes can enhance an overall understanding of its financial performance when considered together with GAAP figures. Refer to the section of this press release below entitled "Non-GAAP Financial Information and Other Disclosures" for further discussion on this subject.

Net Product Revenue

Global net revenue for the second quarter of 2016 was $32.0 million, compared to $23.3 million for the second quarter of 2015. The 37.3% revenue growth was driven by further market penetration in the U.S. and in Europe and an increase in named patient sales in other international territories for PROCYSBI, and by the introduction of QUINSAIR during the quarter.

Cost of Sales

Cost of sales was $4.9 million for the second quarter of 2016, compared to $2.6 million for the second quarter of 2015. The increase was due to higher quarterly sales year-over-year, leading to higher direct costs and royalty expenses in 2016.

Research & Development (R&D)

R&D expenses for the second quarter of 2016 were $15.7 million, compared to $11.9 million for the second quarter of 2015. The increased expense for the three month period ended June 30, 2016 was primarily due to increased activities associated with our product portfolio, support for the European QUINSAIR launch and related to preparation for a potential NDA filing for QUINSAIR in a cystic fibrosis indication in the US.

Selling, General and Administrative (SG&A)

SG&A expenses were $20.9 million for the second quarter of 2016 compared to $17.8 million for the second quarter of 2015. The increase in SG&A expenses was primarily a result of increased personnel costs and promotional support for the worldwide commercial operations of PROCYSBI and QUINSAIR in Europe.

Interest Expense

Interest expense for the second quarter of 2016 was $4.3 million, compared to $4.8 million for the second quarter of 2015. The decrease in interest expense was primarily due to a decrease in principal from which Raptor’s interest expense is calculated.

Net Loss

GAAP net loss in the second quarter of 2016 was $14.0 million, or $0.16 per share, compared to a net loss of $13.9 million, or $0.17 per share for the second quarter of 2015.

Non-GAAP net loss, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, for the second quarter of 2016 was $11.3 million, or $0.13 per share, compared to a non-GAAP net loss of $9.6 million, or $0.12 per share, in the second quarter of 2015.

Cash and Cash Equivalents

As of June 30, 2016, Raptor had $124.2 million in cash and cash equivalents compared to $132.0 million in cash and cash equivalents at March 31, 2016.

2016 Full-Year Financial Guidance

Raising 2016 global net revenue guidance to $125 to $135 million from $115 to $125 million.
Refining PROCYSBI revenue growth to at least 30% over 2015 revenues.
Reiterating non-GAAP operating expense guidance, which excludes non-cash expenditures such as stock compensation, amortization and impairment of IPR&D, and change in the fair value of the contingent consideration liability related to acquisitions, of $125 to $135 million. Raptor is unable to reconcile non-GAAP operating expense guidance to GAAP operating expense guidance without unreasonable efforts and believes any such reconciliation would imply a degree of precision that could be confusing to investors. Raptor is unable to predict the probable significance of any of the above-listed adjustments.
Product and Pipeline Updates

PROCYSBI for Nephropathic Cystinosis

In the second quarter, Raptor recorded the first PROCYSBI sales in several additional European countries.
A New Drug Submission (NDS) for PROCYSBI for the treatment of nephropathic cystinosis is currently under review by Health Canada. Recently, Raptor received notice from Health Canada that it is seeking additional information to complete its review. Raptor intends to submit a response to Health Canada in a timely fashion to preserve Priority Review status and expects that Health Canada’s timeline for review of the NDS will re-commence when it accepts Raptor’s response. As a result, Raptor anticipates a delay in Health Canada’s decision on the potential marketing approval of PROCYSBI.
QUINSAIR for Cystic Fibrosis

In April, Raptor launched QUINSAIR in Germany and Denmark for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adults with cystic fibrosis, and to date, QUINSAIR has become available for reimbursement in several territories in the EU and Raptor is actively expanding into new countries.
Raptor anticipates launching QUINSAIR in Canada for the same indication, for which it has received marketing approval, in the second half of 2016.
In August, the European Patent Office issued EP Pat. No. 1 901 749 with claims covering Raptor’s QUINSAIR formulation. This patent will expire in May 2026, supplementing Raptor’s 10 years of data exclusivity.
MP-376 (Investigational Form of QUINSAIR)

Raptor had a meeting with the FDA in the second quarter concerning MP-376 for the management of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis in the U.S. The FDA requested additional information pertinent to Raptor’s existing trials prior to the company’s potential submission of an NDA. Raptor submitted a response to the FDA’s request and expects to have additional discussions with the FDA.
The company intends to initiate a clinical study for MP-376 in bronchiectasis (BE) in 2016. Raptor plans to provide additional clarity on the timing of key activities once the study design has been finalized.
RP103 for Huntington’s Disease

With current efforts focused on prioritizing clinical programs that represent Raptor’s best use of current capital, the company continues to explore non-dilutive funding and partnering options for RP103 in Huntington’s disease.
RP103 for Mitochondrial Diseases

The second interim analysis, which occurred after 12 patients completed 24 weeks of treatment, indicated the ongoing trial should continue as planned, which is currently ongoing.
Anticipated Upcoming Milestones

2H 2016 — Potential QUINSAIR launch in Canada
2016 — Advancement towards an NDA submission for MP-376 in cystic fibrosis in the U.S.
2016 — Initiation of a clinical study for MP-376 in BE
1H 2017 — Phase 2 RP103 data in mitochondrial diseases

Ironwood Pharmaceuticals Provides Second Quarter 2016 Investor Update

On Auguest 4, 2016 Ironwood Pharmaceuticals, Inc. (NASDAQ: IRWD), a commercial biotechnology company, reported an update on its second quarter 2016 results and recent business activities (Press release, Ironwood Pharmaceuticals, AUG 4, 2016, View Source [SID:1234514298]).

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"During the second quarter of 2016, Ironwood continued to deliver strong operational performance, resulting in a near-doubling of Ironwood revenue, year-over-year. We are well-positioned to advance our four priority franchises, including the launch of two new products in the next nine months, and we remain on track to become cash flow positive in 2018," said Peter Hecht, chief executive officer of Ironwood. "Our flagship product LINZESS demonstrated solid growth as the branded prescription market leader in adult patients with IBS-C or CIC and is on track to exceed $1 billion in net sales by 2020, with increased commercial margins driven by strong demand."

Second Quarter 2016 and Recent Highlights

Irritable Bowel Syndrome with Constipation (IBS-C) / Chronic Idiopathic Constipation (CIC) Franchise

LINZESS. U.S. net sales, as provided by Ironwood’s U.S. collaboration partner Allergan, were $150.5 million in the second quarter of 2016, a 34% increase compared to the second quarter of 2015. Ironwood and Allergan share equally in brand collaboration profits or losses.
More than 650,000 total LINZESS prescriptions were filled in the second quarter of 2016, a 29% increase compared to the second quarter of 2015, according to IMS Health. Since launch, more than 5 million prescriptions for LINZESS have been filled by more than 1 million unique patients, making LINZESS the branded prescription market leader in IBS-C and CIC.
Net profit for the LINZESS U.S. brand collaboration, including commercial and research and development (R&D) expenses, was $58.3 million in the second quarter of 2016, a 289% increase compared to the second quarter of 2015. LINZESS commercial margin was 52% in the second quarter of 2016, compared to 31% in the second quarter of 2015.
Ironwood and Allergan expect to launch a 72 mcg dose of linaclotide in early 2017 that, if approved, can increase physician prescribing of LINZESS within the large, heterogeneous adult CIC patient population. The companies announced during the second quarter of 2016 that the U.S. Food & Drug Administration (FDA) accepted the supplemental new drug application filing for this dose.
Linaclotide Colonic Release. Ironwood and Allergan completed enrollment in a Phase IIb clinical trial and expect data later this year; if positive, the companies anticipate initiating a Phase III trial in 2017. This second-generation guanylate cyclase-C (GC-C) agonist product candidate has the potential, if approved, to provide greater and faster abdominal pain relief in adult IBS-C patients, expand the IBS-C and CIC markets, and extend patent protection to the mid-2030s.
Uncontrolled Gout Franchise

ZURAMPIC. Ironwood expects to launch FDA-approved ZURAMPIC in October 2016 for use in combination with a xanthine oxidase inhibitor (XOI) for the treatment of hyperuricemia associated with uncontrolled gout. Gout is a form of inflammatory arthritis, and an estimated two million patients in the U.S. suffer from uncontrolled gout in which traditional first-line XOI treatment alone is not sufficient to achieve target serum uric acid (sUA) levels. Many of these patients experience painful flares due to hyperuricemia despite treatment with an XOI. In two Phase III clinical trials, the combination of the XOI allopurinol, which decreases production of uric acid, and ZURAMPIC, which increases excretion of uric acid, demonstrated nearly a two-fold increase in the percentage of patients reaching target serum uric acid levels under 6 mg/dL over allopurinol alone at month six. ZURAMPIC is not recommended for the treatment of asymptomatic hyperuricemia and should not be used as monotherapy. Ironwood closed the transaction with AstraZeneca for the exclusive U.S. rights to all products containing lesinurad during the second quarter of 2016.
Lesinurad-allopurinol fixed-dose combination product. The fixed-dose combination of lesinurad and allopurinol is expected to be submitted for FDA review during the second half of 2016.
Refractory Gastroesophageal Reflux Disease (rGERD) Franchise

IW-3718. Ironwood continues to enroll patients in a dose-ranging Phase IIb clinical trial of IW-3718, a wholly-owned asset for the potential treatment of rGERD. Data are expected in 2017. IW-3718 is a novel, investigational gastric retentive formulation of a bile acid sequestrant designed to work with a proton pump inhibitor (PPI) to reduce the detrimental effects of bile and acid on the esophagus. An estimated 10 million people in the U.S. suffer from rGERD and continue to experience heartburn symptoms despite treatment with PPIs.
Vascular and Fibrotic Franchise

IW-1973. Ironwood generated positive topline data from its Phase Ib multiple ascending dose study of IW-1973. The data were consistent with previous preclinical and Phase Ia findings and support advancement of IW-1973 into Phase II clinical trials, expected to begin later this year. Specifically, in the Phase Ib study, IW-1973 demonstrated characteristics that Ironwood believes could be important pharmacokinetic differentiators in the field of soluble guanylate cyclase (sGC) stimulators, including a narrow peak-to-trough ratio indicative of the potential to maintain a durable and consistent therapeutic effect; a profile that indicates suitability for once-daily dosing; and a volume of distribution consistent with penetration beyond the vasculature and into the tissues.
IW-1701. Ironwood completed enrollment in a Phase Ib multiple ascending dose study, with data expected later this year and a Phase II trial expected to begin before year-end.
Global Collaborations and Partnerships

Linaclotide is currently under review by the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan for potential approval for the treatment of adult patients with IBS-C. Additionally, Ironwood’s partner Astellas Pharma Inc. initiated a Phase III clinical trial of linaclotide in Japan for adults with chronic constipation.
Ironwood continued co-promoting Allergan’s VIBERZI (eluxadoline) for adults suffering from IBS with diarrhea (IBS-D) and Exact Sciences’ Cologuard noninvasive stool DNA screening test for colorectal cancer, in the U.S.
Corporate and Financials

Collaborative Arrangements Revenue
Collaborative arrangements revenue was $54.4 million in the second quarter of 2016, compared to $27.7 million in the second quarter of 2015. Revenue primarily consisted of $48.3 million in revenue associated with Ironwood’s share of the net profits from the sales of LINZESS in the U.S., compared to $24.3 million in the second quarter of 2015.
Operating Expenses
Operating expenses were $69.7 million in the second quarter of 2016, compared to $61.6 million in the second quarter of 2015. Operating expenses in the second quarter of 2016 consisted of $31.7 million in R&D expenses; $36.9 million in selling, general and administrative (SG&A) expenses; and $1.1 million in acquired intangible asset amortization expenses resulting from Ironwood’s U.S. licensing agreement with AstraZeneca for the exclusive rights to all products containing lesinurad.
Other Expense
Interest Expense. Net interest expense was $9.5 million in the second quarter of 2016, in connection with the $175 million debt financing executed in January 2013 and the approximately $336 million convertible debt financing executed in June 2015. Interest expense recorded in the second quarter of 2016 includes $6.2 million in cash expense and $3.6 million in non-cash expense. Both the cash and non-cash components of the 2022 convertible notes are recorded quarterly.
Gain/Loss on Derivatives. Ironwood records a gain/loss on derivatives related to the change in fair value of the convertible note hedges and note hedge warrants issued in connection with the convertible debt financing in June 2015. A gain on derivatives of $3.1 million was recorded in the second quarter of 2016.
Net Loss
GAAP net loss was $21.7 million, or $0.15 per share, in the second quarter of 2016, compared to $48.0 million, or $0.34 per share, in the second quarter of 2015.
Non-GAAP net loss was $23.8 million, or $0.16 per share, in the second quarter of 2016, compared to $47.8 million, or $0.34 per share, in the second quarter of 2015. Non-GAAP net loss excludes the impact of mark-to-market adjustments on the derivatives related to Ironwood’s convertible debt and the amortization of acquired intangible assets related to Ironwood’s U.S. lesinurad license. See Non-GAAP Financial Measures below.
Cash Position
Ironwood ended the second quarter of 2016 with $325 million of cash, cash equivalents and available-for-sale securities, a decrease of $109 million from the end of the first quarter of 2016. This figure includes the $100 million upfront payment to AstraZeneca from cash on hand under the lesinurad licensing agreement. Cash used in operations was $6 million, a 77% decline from the $26 million used during the same period a year ago.
2016 Financial Guidance
With the completion of its U.S. licensing transaction for all products containing lesinurad and support of the anticipated launch of FDA-approved ZURAMPIC in October 2016, Ironwood is updating its guidance for 2016. Ironwood expects:

R&D expenses to fall within a range of $140 million to $150 million, (previously $130 million to $145 million),
SG&A expenses to fall within a range of $170 million to $180 million, (previously $125 million to $140 million), and
Amortization of intangible assets to be $8 million (not applicable prior to the U.S. lesinurad license).
Consistent with its guidance following the announcement of the U.S. lesinurad license, Ironwood continues to expect to use less than $70 million in cash for operations in 2016.

Allergan and Ironwood continue to expect total 2016 marketing and sales expenses for LINZESS to be in the range of $230 million to $260 million, and the companies now expect these expenses to be in the mid to higher end of this range.

Non-GAAP Financial Measures

The company presents non-GAAP net loss and non-GAAP net loss per share to exclude the impact of net gains and losses on the derivatives related to our convertible notes that are required to be marked-to-market, and the amortization of acquired intangible assets. The derivative gains and losses may be highly variable, difficult to predict and of a size that could have a substantial impact on the company’s reported results of operations in any given period. The acquired intangible assets are valued at the time of acquisition and are amortized over their estimated economic useful life, and management believes excluding the amortization of acquired intangible assets provides more consistency with internally developed intangible assets for which research and development costs were previously expensed. The company has presented non-GAAP net loss and non-GAAP net loss per share in prior calendar quarters, and this is the first calendar quarter in which the company has amortization of acquired intangible assets that can be excluded from such non-GAAP financial measures. Management believes this non-GAAP information is useful for investors, taken in conjunction with Ironwood’s GAAP financial statements, because it provides greater transparency and period-over-period comparability with respect to Ironwood’s operating performance. These measures are also used by management to assess the performance of the business. Investors should consider these non-GAAP measures only as a supplement to, not as a substitute for or as superior to, measures of financial performance prepared in accordance with GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. For a reconciliation of these non-GAAP financial measures to the most comparable GAAP measures, please refer to the table at the end of this press release.