On May 3, 2018 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2018 and provided a business update (Press release, Regeneron, MAY 3, 2018, View Source [SID1234526003]).

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"Regeneron’s commercial business continues to advance with positive sales growth for EYLEA and strong underlying demand for Dupixent," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "This year, we have reported positive Phase 3 results for Praluent in cardiovascular risk reduction and for EYLEA in diabetic retinopathy – and look forward to continued progress with Dupixent, including a U.S. regulatory decision in uncontrolled asthma and Phase 3 results in both adolescents with atopic dermatitis and adults with nasal polyps. Our immuno-oncology portfolio is advancing rapidly, with a potential first approval for cemiplimab in advanced cutaneous squamous cell carcinoma, and a broad pivotal program in lung cancer."

First Quarter 2018 Business Highlights

Key Pipeline Progress

Regeneron has seventeen product candidates in clinical development, which consist of EYLEA and fully human antibodies generated using the Company’s VelocImmune technology, including six in collaboration with Sanofi. Updates from the clinical pipeline include:

EYLEA (aflibercept) Injection

In the first quarter of 2018, the Company announced positive top-line results from the Phase 3 PANORAMA study of EYLEA in moderately severe to severe non-proliferative diabetic retinopathy (NPDR). PANORAMA will form the basis of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) by the end of the year.
Dupixent (dupilumab) Injection

Dupixent, an antibody that blocks signaling of IL-4 and IL-13, is currently approved in atopic dermatitis for adults in the United States, European Union, and certain other countries outside the United States.
Dupilumab is being studied in asthma, adolescent and pediatric atopic dermatitis, nasal polyps, and eosinophilic esophagitis (EoE), with additional studies planned in 2018. Data are expected to be reported from Phase 3 studies in patients with nasal polyps and adolescent patients with atopic dermatitis during 2018.
In March 2018, the sBLA for Dupixent as an add-on maintenance treatment in certain adults and adolescents (12 years of age and older) with moderate-to-severe asthma was filed with the FDA, with a target action date of October 20, 2018. In the first quarter of 2018, regulatory applications were also accepted for review by the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan for Dupixent in asthma.
Dupixent for the treatment of atopic dermatitis in adults not adequately controlled with existing therapies was approved by the Ministry of Health, Labor and Welfare (MHLW) in Japan in the first quarter of 2018, and has recently been launched.
In the first quarter of 2018, a Phase 2/3 study in younger pediatric patients (from six months to five years of age) with severe atopic dermatitis was initiated.
Praluent (alirocumab) Injection

In the first quarter of 2018, the Company and Sanofi announced that the ODYSSEY OUTCOMES trial met its primary endpoint, demonstrating that high-risk patients who added Praluent to maximally-tolerated statins experienced significantly fewer major adverse cardiovascular events compared to those on maximally-tolerated statins alone. In addition, in this study, adding Praluent to maximally-tolerated statins was associated with reduced death from any cause.
In May 2018, the Company and Sanofi announced they will lower the net price of Praluent in exchange for straightforward, more affordable patient access from Express Scripts. Praluent will become the exclusive PCSK9 inhibitor therapy on the Express Scripts national formulary. The agreement takes effect on July 1, 2018 for commercial patients covered by the Express Scripts National Preferred Formulary (approximately 25 million individuals in total).
Cemiplimab, an antibody to programmed cell death protein 1 (PD-1), is being studied in patients with cancer.

In April 2018, the FDA accepted for priority review the BLA for cemiplimab for the treatment of patients with metastatic CSCC or patients with locally advanced CSCC who are not candidates for surgery. The target action date for the FDA decision is October 28, 2018.
In April 2018, the EMA also accepted for review the Marketing Authorization Application (MAA) for cemiplimab in patients with metastatic CSCC or patients with locally advanced CSCC who are not candidates for surgery.
Fasinumab, an antibody targeting Nerve Growth Factor (NGF), is being studied in patients with osteoarthritis of the knee or hip and chronic low back pain in patients with concomitant osteoarthritis of the knee or hip.

An independent Data Monitoring Committee monitoring the ongoing safety and efficacy of the fasinumab clinical trials recommended that the higher dose-regimens be discontinued based on the risk benefit assessment and that the program may continue with the lower dose-regimens of fasinumab. The trials are being modified accordingly.
Evinacumab is an antibody to angiopoietin-like protein 3 (ANGPTL3). A Phase 3 study in homozygous familial hypercholesterolemia (HoFH) was initiated in the first quarter of 2018.

REGN3500 is an antibody to interleukin-33 (IL-33). In the first quarter of 2018, a Phase 2 study in asthma was initiated.

Select Upcoming 2018 Milestones

Programs

Milestones

EYLEA

•

FDA decision on sBLA for every 12-week dosing interval in wet AMD (target action date of August 11, 2018)

•

Submit sBLA for the treatment of NPDR in patients without DME

•

Submit sBLA for pre-filled syringe

Dupixent (dupilumab)

•

FDA decision on sBLA for asthma in adult/adolescent patients (target action date of October 20, 2018)

•

Additional regulatory agency decisions on applications for atopic dermatitis in adults outside the United States

•

Report data from Phase 3 study in adolescent patients (12-17 years of age) with atopic dermatitis and submit sBLA and MAA for expanded indication

•

Report data from Phase 3 studies in nasal polyps

•

Initiate Phase 3 study in EoE

•

Initiate Phase 3 program in chronic obstructive pulmonary disease (COPD)

•

Initiate clinical program in co-morbid allergic conditions

•

Initiate Phase 2 studies in peanut allergy and grass allergy

Praluent (alirocumab)

•

Submit for regulatory approval for cardiovascular risk reduction in the United States and EU and for first-line treatment of hyperlipidemia in the United States

•

FDA decision on sBLA for use with apheresis (target action date of August 24, 2018)

•

Initiate Phase 3 pediatric studies in HoFH and HeFH

Kevzara (sarilumab)

•

Initiate Phase 3 study in giant cell arteritis

•

Initiate Phase 3 study in polymyalgia rheumatica

Cemiplimab (PD-1 Antibody)

•

FDA decision on BLA for advanced CSCC (target action date of October 28, 2018)

•

Continue patient enrollment in Phase 3 study for first-line treatment of non-small cell lung cancer, as well as various other studies

•

Initiate additional studies in non-small cell lung cancer

Fasinumab (NGF Antibody)

•

Report data from first Phase 3 efficacy study in osteoarthritis pain

Evinacumab (Angptl-3 Antibody)

•

Initiate Phase 2 study in severe hypertriglyceridemia

REGN3500 (IL-33 Antibody)

•

Initiate Phase 2 studies in COPD and atopic dermatitis

Bispecific Antibodies

•

Initiate Phase 2 study for REGN1979 (CD20xCD3 Antibody) in Follicular Lymphoma

•

Initiate clinical study in REGN4018 (MUC16xCD3 antibody)

•

Submit Investigational New Drug Application (IND) for BCMAxCD3 antibody

Financial Results

Product Revenues: Net product sales were $988 million in the first quarter of 2018, compared to $858 million in the first quarter of 2017. EYLEA net product sales in the United States were $984 million in the first quarter of 2018, compared to $854 million in the first quarter of 2017. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.

Total Revenues: Total revenues, which include product revenues described above, increased by 15% to $1.511 billion in the first quarter of 2018, compared to $1.319 billion in the first quarter of 2017. Total revenues include Sanofi and Bayer collaboration revenues of $437 million in the first quarter of 2018, compared to $404 million in the first quarter of 2017. Sanofi collaboration revenue in the first quarter of 2018 decreased primarily due to the Company’s Discovery and Preclinical Development Agreement with Sanofi ending on December 31, 2017, lower reimbursement for Dupixent (dupilumab) development activities, and an increase in the Company’s share of the collaborations’ Dupixent commercialization expenses. These decreases were partly offset by the Company’s share of higher net sales of Dupixent (as the product was launched at the end of March 2017), and an increase in reimbursement revenues in connection with late-stage clinical development activities for cemiplimab. Bayer collaboration revenue increased in the first quarter of 2018 primarily due to an increase in the Company’s share of net profits in connection with higher sales of EYLEA outside the United States.

Other revenue in the first quarter of 2018 increased primarily due to higher reimbursements of the Company’s fasinumab research and development expenses in connection with the Company’s collaboration agreement with Teva.

The Company adopted Accounting Standard Codification (ASC) 606, Revenue from Contracts with Customers, as of January 1, 2018. The Company adopted the standard using the modified retrospective method; prior period amounts have not been adjusted and the Company recognized a cumulative-effect adjustment to reduce Retained earnings and increase Deferred revenue on January 1, 2018 by $143 million, net of tax. The adoption of the new standard did not have a material impact of the Company’s total revenues in the first quarter of 2018.

Refer to Table 4 for a summary of collaboration and other revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $499 million in the first quarter of 2018, compared to $507 million in the first quarter of 2017. The lower R&D expenses in the first quarter of 2018 were principally due to a decrease in clinical manufacturing costs and a decrease in dupilumab development costs. These decreases were partly offset by an increase in cemiplimab and fasinumab clinical trial costs. In the first quarter of 2018, R&D-related non-cash share-based compensation expense was $41 million, compared to $74 million in the first quarter of 2017. The decrease in total non-cash share-based compensation expense in the first quarter of 2018 was primarily attributable to a revision in the Company’s estimate of the number of stock options that are expected to be forfeited.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $331 million in the first quarter of 2018, compared to $297 million in the first quarter of 2017. The higher SG&A expenses in the first quarter of 2018 were primarily due to higher headcount and headcount-related costs and an increase in commercialization-related expenses to support the launch of Dupixent. In the first quarter of 2018, SG&A-related non-cash share-based compensation expense decreased to $35 million, compared to $54 million in the first quarter of 2017, primarily due to the revision in the Company’s estimate of stock option forfeitures as described under "R&D Expenses" above.

Income Tax Expense: In the first quarter of 2018, GAAP income tax expense was $107 million and the effective tax rate was 18.3%, compared to $183 million and 42.4% in the first quarter of 2017. The Company’s effective tax rate for the first quarter of 2018 was significantly impacted by the bill known as the Tax Cuts and Jobs Act (the "U.S. Tax Reform Act"), which reduced the U.S. federal corporate income tax rate from 35% to 21% effective January 1, 2018. The effective tax rate for the first quarter of 2018 was positively impacted, compared to the U.S. federal statutory rate, primarily by the foreign-derived intangible income deduction and the federal tax credit for research activities.

Other income, net: GAAP other income in the first quarter of 2018 included the recognition of unrealized gains on equity securities. In the first quarter of 2018, the Company adopted Accounting Standards Update ("ASU") 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities, as of January 1, 2018, which requires the Company to measure equity investments at fair value with changes in fair value recognized in net income; previously, such changes in fair value were recognized in Other comprehensive income (loss). Refer to Table 3 for the non-GAAP adjustment related to these gains.

GAAP and Non-GAAP Net Income(2): GAAP net income was $478 million, or $4.44 per basic share and $4.16 per diluted share, in the first quarter of 2018, compared to GAAP net income of $249 million, or $2.36 per basic share and $2.16 per diluted share, in the first quarter of 2017.

2018 Financial Guidance(3)

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.

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 estimated 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 or changes in the fair value of the Company’s equity investments) or items that are not associated with normal, recurring operations (such as changes in applicable laws and regulations). 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.

Conference Call Information

Regeneron will host a conference call and simultaneous webcast to discuss its first quarter 2018 financial and operating results on Thursday, May 3, 2018, at 8:30 AM. To access this call, dial (800) 708-4540 (U.S.) or (847) 619-6397 (International). A link to the webcast may be accessed from the "Investors & Media" page of Regeneron’s website at View Source A replay of the conference call and webcast will be archived on the Company’s website and will be available for 30 days.

(3)

The Company’s 2018 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

On May 3, 2018 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2018 and provided a business update (Press release, Regeneron, MAY 3, 2018, View Source [SID1234526025]).

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"Regeneron’s commercial business continues to advance with positive sales growth for EYLEA and strong underlying demand for Dupixent," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "This year, we have reported positive Phase 3 results for Praluent in cardiovascular risk reduction and for EYLEA in diabetic retinopathy – and look forward to continued progress with Dupixent, including a U.S. regulatory decision in uncontrolled asthma and Phase 3 results in both adolescents with atopic dermatitis and adults with nasal polyps. Our immuno-oncology portfolio is advancing rapidly, with a potential first approval for cemiplimab in advanced cutaneous squamous cell carcinoma, and a broad pivotal program in lung cancer."

First Quarter 2018 Business Highlights

Key Pipeline Progress
Regeneron has seventeen product candidates in clinical development, which consist of EYLEA and fully human antibodies generated using the Company’s VelocImmune technology, including six in collaboration with Sanofi. Updates from the clinical pipeline include:
EYLEA (aflibercept) Injection

In the first quarter of 2018, the Company announced positive top-line results from the Phase 3 PANORAMA study of EYLEA in moderately severe to severe non-proliferative diabetic retinopathy (NPDR). PANORAMA will form the basis of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) by the end of the year.

Dupixent (dupilumab) Injection

Dupixent, an antibody that blocks signaling of IL-4 and IL-13, is currently approved in atopic dermatitis for adults in the United States, European Union, and certain other countries outside the United States.

Dupilumab is being studied in asthma, adolescent and pediatric atopic dermatitis, nasal polyps, and eosinophilic esophagitis (EoE), with additional studies planned in 2018. Data are expected to be reported from Phase 3 studies in patients with nasal polyps and adolescent patients with atopic dermatitis during 2018.

In March 2018, the sBLA for Dupixent as an add-on maintenance treatment in certain adults and adolescents (12 years of age and older) with moderate-to-severe asthma was filed with the FDA, with a target action date of October 20, 2018. In the first quarter of 2018, regulatory applications were also accepted for review by the European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan for Dupixent in asthma.

Dupixent for the treatment of atopic dermatitis in adults not adequately controlled with existing therapies was approved by the Ministry of Health, Labor and Welfare (MHLW) in Japan in the first quarter of 2018, and has recently been launched.

In the first quarter of 2018, a Phase 2/3 study in younger pediatric patients (from six months to five years of age) with severe atopic dermatitis was initiated.

Praluent (alirocumab) Injection

In the first quarter of 2018, the Company and Sanofi announced that the ODYSSEY OUTCOMES trial met its primary endpoint, demonstrating that high-risk patients who added Praluent to maximally-tolerated statins experienced significantly fewer major adverse cardiovascular events compared to those on maximally-tolerated statins alone. In addition, in this study, adding Praluent to maximally-tolerated statins was associated with reduced death from any cause.

In May 2018, the Company and Sanofi announced they will lower the net price of Praluent in exchange for straightforward, more affordable patient access from Express Scripts. Praluent will become the exclusive PCSK9 inhibitor therapy on the Express Scripts national formulary. The agreement takes effect on July 1, 2018 for commercial patients covered by the Express Scripts National Preferred Formulary (approximately 25 million individuals in total).

Cemiplimab, an antibody to programmed cell death protein 1 (PD-1), is being studied in patients with cancer.

In April 2018, the FDA accepted for priority review the BLA for cemiplimab for the treatment of patients with metastatic CSCC or patients with locally advanced CSCC who are not candidates for surgery. The target action date for the FDA decision is October 28, 2018.

In April 2018, the EMA also accepted for review the Marketing Authorization Application (MAA) for cemiplimab in patients with metastatic CSCC or patients with locally advanced CSCC who are not candidates for surgery.

Fasinumab, an antibody targeting Nerve Growth Factor (NGF), is being studied in patients with osteoarthritis of the knee or hip and chronic low back pain in patients with concomitant osteoarthritis of the knee or hip.

An independent Data Monitoring Committee monitoring the ongoing safety and efficacy of the fasinumab clinical trials recommended that the higher dose-regimens be discontinued based on the risk benefit assessment and that the program may continue with the lower dose-regimens of fasinumab. The trials are being modified accordingly.

Evinacumab is an antibody to angiopoietin-like protein 3 (ANGPTL3). A Phase 3 study in homozygous familial hypercholesterolemia (HoFH) was initiated in the first quarter of 2018.

Financial Results

Product Revenues: Net product sales were $988 million in the first quarter of 2018, compared to $858 million in the first quarter of 2017. EYLEA net product sales in the United States were $984 million in the first quarter of 2018, compared to $854 million in the first quarter of 2017. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.

Total Revenues: Total revenues, which include product revenues described above, increased by 15% to $1.511 billion in the first quarter of 2018, compared to $1.319 billion in the first quarter of 2017. Total revenues include Sanofi and Bayer collaboration revenues of $437 million in the first quarter of 2018, compared to $404 million in the first quarter of 2017. Sanofi collaboration revenue in the first quarter of 2018 decreased primarily due to the Company’s Discovery and Preclinical Development Agreement with Sanofi ending on December 31, 2017, lower reimbursement for Dupixent (dupilumab) development activities, and an increase in the Company’s share of the collaborations’ Dupixent commercialization expenses. These decreases were partly offset by the Company’s share of higher net sales of Dupixent (as the product was launched at the end of March 2017), and an increase in reimbursement revenues in connection with late-stage clinical development activities for cemiplimab. Bayer collaboration revenue increased in the first quarter of 2018 primarily due to an increase in the Company’s share of net profits in connection with higher sales of EYLEA outside the United States.

Other revenue in the first quarter of 2018 increased primarily due to higher reimbursements of the Company’s fasinumab research and development expenses in connection with the Company’s collaboration agreement with Teva.

The Company adopted Accounting Standard Codification (ASC) 606, Revenue from Contracts with Customers, as of January 1, 2018. The Company adopted the standard using the modified retrospective method; prior period amounts have not been adjusted and the Company recognized a cumulative-effect adjustment to reduce Retained earnings and increase Deferred revenue on January 1, 2018 by $143 million, net of tax. The adoption of the new standard did not have a material impact of the Company’s total revenues in the first quarter of 2018.

Refer to Table 4 for a summary of collaboration and other revenue.

Research and Development (R&D) Expenses: GAAP R&D expenses were $499 million in the first quarter of 2018, compared to $507 million in the first quarter of 2017. The lower R&D expenses in the first quarter of 2018 were principally due to a decrease in clinical manufacturing costs and a decrease in dupilumab development costs. These decreases were partly offset by an increase in cemiplimab and fasinumab clinical trial costs. In the first quarter of 2018, R&D-related non-cash share-based compensation expense was $41 million, compared to $74 million in the first quarter of 2017. The decrease in total non-cash share-based compensation expense in the first quarter of 2018 was primarily attributable to a revision in the Company’s estimate of the number of stock options that are expected to be forfeited.

5

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $331 million in the first quarter of 2018, compared to $297 million in the first quarter of 2017. The higher SG&A expenses in the first quarter of 2018 were primarily due to higher headcount and headcount-related costs and an increase in commercialization-related expenses to support the launch of Dupixent. In the first quarter of 2018, SG&A-related non-cash share-based compensation expense decreased to $35 million, compared to $54 million in the first quarter of 2017, primarily due to the revision in the Company’s estimate of stock option forfeitures as described under "R&D Expenses" above.

Income Tax Expense: In the first quarter of 2018, GAAP income tax expense was $107 million and the effective tax rate was 18.3%, compared to $183 million and 42.4% in the first quarter of 2017. The Company’s effective tax rate for the first quarter of 2018 was significantly impacted by the bill known as the Tax Cuts and Jobs Act (the "U.S. Tax Reform Act"), which reduced the U.S. federal corporate income tax rate from 35% to 21% effective January 1, 2018. The effective tax rate for the first quarter of 2018 was positively impacted, compared to the U.S. federal statutory rate, primarily by the foreign-derived intangible income deduction and the federal tax credit for research activities.

Other income, net: GAAP other income in the first quarter of 2018 included the recognition of unrealized gains on equity securities. In the first quarter of 2018, the Company adopted Accounting Standards Update ("ASU") 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities, as of January 1, 2018, which requires the Company to measure equity investments at fair value with changes in fair value recognized in net income; previously, such changes in fair value were recognized in Other comprehensive income (loss). Refer to Table 3 for the non-GAAP adjustment related to these gains.

GAAP and Non-GAAP Net Income(2): GAAP net income was $478 million, or $4.44 per basic share and $4.16 per diluted share, in the first quarter of 2018, compared to GAAP net income of $249 million, or $2.36 per basic share and $2.16 per diluted share, in the first quarter of 2017.

Non-GAAP net income was $537 million, or $4.99 per basic share and $4.67 per diluted share, in the first quarter of 2018, compared to non-GAAP net income of $337 million, or $3.19 per basic share and $2.92 per diluted share, in the first quarter of 2017

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.

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 estimated 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 or changes in the fair value of the Company’s equity investments) or items that are not associated with normal, recurring operations (such as changes in applicable laws and regulations). 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.

The Company’s 2018 financial guidance does not assume the completion of any significant business development transactions not completed as of the date of this press release.

Conference Call Information

Regeneron will host a conference call and simultaneous webcast to discuss its first quarter 2018 financial and operating results on Thursday, May 3, 2018, at 8:30 AM. To access this call, dial (800) 708-4540 (U.S.) or (847) 619-6397 (International). A link to the webcast may be accessed from the "Investors & Media" page of Regeneron’s website at View Source A replay of the conference call and webcast will be archived on the Company’s website and will be available for 30 days.

On May 3, 2018 NANOBIOTIX (Euronext: NANO – ISIN: FR0011341205), a late clinical-stage nanomedicine company pioneering new approaches to the treatment of cancer, reported that it is launching a research collaboration with Weill Cornell Medicine to begin nonclinical studies of NBTXR3’s mechanism of action (Press release, Nanobiotix, MAY 3, 2018 View Source [SID1234526080]). NBTXR3 is a first-in-class product designed to destroy, when activated by radiotherapy, tumors and metastasis through physical cell death and to induce immunogenic cell death leading to specific activation of the immune system.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The research collaboration between Weill Cornell Medicine, based in New York City, and Nanobiotix will be conducted
over the course of one year, with the goal of continuing the exploration of the role of NBTXR3 in Immuno-Oncology.
The main objective of this collaboration is to study the impact of NBTXR3 activated by radiotherapy on the cGAS-STING
pathway using different in vitro and in vivo murine models (mammary). Along with immunogenic cell death, the cGASSTING
pathway has emerged as the key component of the anti-tumor immune response. Data generated from this
collaboration could support current evidence indicating that NBTXR3 activated by radiotherapy can increase the antitumor
immune response, compared with radiotherapy alone, and transform an irradiated tumor into an efficient in
situ vaccine.
Dr. Sandra Demaria, M.D., Professor of Radiation Oncology and Chief of the Division of Experimental Radiotherapy in
the Department of Radiation Oncology at Weill Cornell Medicine, and Principal Investigator for the study, said: "We
have learned that radiotherapy has the potential to convert a tumor into an in-situ vaccine, and enhance systemic
tumor responses to immunotherapy. But there is room for improvement: NBTXR3 nanoparticles enhance the proimmunogenic
effects of radiotherapy, and we want to understand how they work. This knowledge will further the
development of this innovative approach for the treatment of cancer patients who are resistant to immune checkpoint
inhibitors."
The Company received the FDA’s approval to launch a clinical study of NBTXR3 activated by radiotherapy in
combination with anti-PD1 antibody in lung, and head and neck cancer patients (head and neck squamous cell
carcinoma and nonsmall cell lung cancer). This trial that shall start in Q2 2018, aims to expand the potential of NBTXR3,
including using it to treat recurrent or metastatic disease.
NBTXR3 positioning in IO
Many IO combination strategies focus on ‘priming’ the tumor, which is now becoming a prerequisite of turning a "cold"
tumor into a "hot" tumor.
Compared to other modalities that could be used for priming the tumor, NBTXR3 could have a number of advantages:
the physical and universal mode of action that could be used widely across oncology, a one-time local injection and
good fit within existing medical practice already used as a basis for cancer treatment, as well as a very good chronic
safety profile and well-established manufacturing process.
Published preclinical and clinical data indicate that NBTXR3 could play a key role in oncology and could become a
backbone in immuno-oncology.
Nanobiotix’s immuno-oncology combination program opens the door to new developments, potential new
indications, and important value creation opportunities.
-ends-
2
About NBTXR3
NBTXR3 is a first-in-class product designed to destroy, when activated by radiotherapy, tumors and metastasis through physical
cell death and to immunogenic cell death leading to specific activation of the immune system.
NBTXR3 has a high degree of biocompatibility, requires one single administration before the whole radiotherapy treatment and
has the ability to fit into current worldwide standards of radiation care.
NBTXR3 is being evaluated in head and neck cancer (locally advanced squamous cell carcinoma of the oral cavity or oropharynx),
and the trial targets frail and elderly patients who have advanced cancer with very limited therapeutic options. The Phase I/II trial
has already delivered very promising results regarding the local control of the tumors and a potential metastatic control through
in situ vaccination.
Nanobiotix is running an Immuno-Oncology program with NBTXR3 that includes several studies. In the U.S., the Company received
the FDA’s approval to launch a clinical study of NBTXR3 activated by radiotherapy in combination with anti-PD1 antibodies in lung,
and head and neck cancer patients (head and neck squamous cell carcinoma and non-small cell lung cancer). This trial aims to
expand the potential of NBTXR3, including using it to treat recurrent or metastatic disease.
The first market authorization process (CE Marking) is ongoing in Europe in the soft tissue sarcoma indication.
The other ongoing studies are treating patients with liver cancers (hepatocellular carcinoma and liver metastasis), locally advanced
or unresectable rectal cancer in combination with chemotherapy, head and neck cancer in combination with concurrent
chemotherapy, and prostate adenocarcinoma.

On May 3, 2018 Affimed N.V. (Nasdaq: AFMD), a clinical stage biopharmaceutical company focused on discovering and developing highly targeted cancer immunotherapies, reported that Dr. Adi Hoess, CEO, will present at Deutsche Bank’s 43rd Annual Health Care Conference on Tuesday, May 8, 2018 at 10:40 am ET (Press release, Affimed, MAY 3, 2018, View Source [SID1234526099]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

A live webcast of the conference presentation can be accessed through the "Events" section on the "Investors & Media" page of the Affimed website at www.affimed.com/events.php. A replay of the presentation will be available from Affimed’s website for 30 days following the conference.

On May 2, 2018 Blueprint Medicines Corporation (Nasdaq: BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported financial results and provided a business update for the quarter ended March 31, 2018 (Press release, Blueprint Medicines, MAY 2, 2018, View Source;p=RssLanding&cat=news&id=2346251 [SID1234525940]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"In the first quarter, we continued to make significant progress across our portfolio toward our vision of rapidly delivering potentially transformative kinase medicines to patients with genomically defined diseases," said Jeff Albers, Chief Executive Officer of Blueprint Medicines. "In particular, we were excited to present initial clinical proof-of-concept data for our highly selective RET inhibitor BLU-667, which showed consistent clinical activity in patients with multiple tumor types, RET alterations and prior therapies, along with a favorable safety and tolerability profile. In addition, we received positive feedback from the FDA supporting our registration plan in systemic mastocytosis, including support for a single-arm registration-enabling Phase 2 trial in patients with advanced systemic mastocytosis representing a potential expedited path to registration."

Clinical Programs:

Avapritinib: Gastrointestinal Stromal Tumors (GIST)

In March 2018, Blueprint Medicines completed enrollment of the PDGFRα D842V expansion cohort of its ongoing Phase 1 NAVIGATOR clinical trial. Based on feedback from the U.S. Food and Drug Administration (FDA) at an End-of-Phase 1 meeting in 2017, Blueprint Medicines believes that data from the PDGFRα D842V expansion cohort may be sufficient to support a New Drug Application (NDA) for avapritinib for the treatment of patients with PDGFRα D842V-driven GIST. Based on the expected timeline for the collection of data, Blueprint Medicines now anticipates it will submit an initial NDA to the FDA for avapritinib in the first half of 2019. In the first quarter, Blueprint Medicines announced it had completed enrollment of the third-line or later (KIT-driven) GIST cohort and initiated enrollment of the second-line GIST cohort in the Phase 1 NAVIGATOR trial. Blueprint Medicines anticipates presenting updated data from the NAVIGATOR trial in the second half of 2018.
Avapritinib: Advanced Systemic Mastocytosis (SM)

Blueprint Medicines recently received positive feedback from the FDA supporting its proposed registration plan for avapritinib in patients with advanced, smoldering and indolent SM. Consistent with feedback from the FDA, Blueprint Medicines plans to initiate a registration-enabling open-label, single-arm Phase 2 clinical trial in patients with advanced SM, called the PATHFINDER trial, by the middle of 2018. In addition, Blueprint Medicines plans to initiate a registration-enabling Phase 2 clinical trial in patients with indolent SM and smoldering SM, called the PIONEER trial, by the end of 2018.
Enrollment in the expansion portion of the Phase 1 EXPLORER clinical trial for advanced SM is ongoing, and Blueprint Medicines anticipates presenting updated data from this trial in the second half of 2018.
BLU-667: RET-Altered Solid Tumors

In April 2018, Blueprint Medicines presented proof-of-concept data from its ongoing Phase 1 ARROW clinical trial of BLU-667 in patients with RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced solid tumors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. The data showed broad and robust clinical activity across multiple tumor types and RET genotypes, including in patients whose disease had progressed on prior multi-kinase therapy. Radiographic tumor reductions were observed in 84 percent of patients with RET-altered solid tumors and measurable target lesions, and preliminary overall response rates were 50 percent and 40 percent in patients with NSCLC and MTC, respectively. The data also showed that BLU-667 was generally well-tolerated, and most adverse events reported by investigators were Grade 1. Read the full data here. The maximum tolerated dose (MTD) was determined to be 400 mg once daily, and global enrollment in the dose expansion portion of the Phase 1 ARROW clinical trial is ongoing.
In April 2018, Blueprint Medicines announced the publication of foundational preclinical data and clinical proof-of-concept results for BLU-667. The publication outlined preclinical data characterizing the potency and selectivity of BLU-667 against multiple oncogenic RET variants and resistant mutants and anti-tumor activity in multiple solid tumor models. It also included four patient vignettes from the ongoing Phase 1 ARROW clinical trial, showing clinical responses in patients with RET-KIF5B-altered NSCLC and MTC harboring multiple RET mutations. The paper, titled "Precision targeted therapy with BLU-667 for RET-driven cancers," was published online in Cancer Discovery.
Research Programs:

In the first quarter of 2018, Blueprint Medicines initiated investigational new drug application-enabling studies for BLU-782, its development candidate for the treatment of patients with fibrodysplasia ossificans progressiva. Blueprint Medicines plans to report preclinical data for the program in 2018.
Blueprint Medicines also recently nominated a new wholly-owned discovery program for an undisclosed kinase target.
First Quarter Financial Results:

Cash Position: As of March 31, 2018, cash, cash equivalents and investments were $621.1 million, as compared to $673.4 million as of December 31, 2017. This decrease was primarily related to cash used in operating activities.
Collaboration Revenues: Collaboration revenues were $0.9 million for the first quarter of 2018, as compared to $5.8 million for the first quarter of 2017. This decrease was primarily due to the termination of the Alexion agreement in 2017, which resulted in no revenue recognized under this agreement in the first quarter 2018, as well as the impact on revenue recognized under the Roche agreement as a result of the adoption of Accounting Standards Codification 606 effective January 1, 2018.
R&D Expenses: Research and development expenses were $50.0 million for the first quarter of 2018, as compared to $28.5 million for the first quarter of 2017. This increase was primarily attributable to increased clinical and manufacturing expenses associated with advancing avapritinib, BLU-554, and BLU-667 further through clinical trials and increased personnel-related expenses. Research and development expenses included $3.0 million in stock-based compensation expenses for the first quarter of 2018.
G&A Expenses: General and administrative expenses were $9.9 million for the first quarter of 2018, as compared to $5.7 million for the first quarter of 2017. This increase was primarily attributable to increased personnel-related expenses and increased professional fees, including pre-commercial planning activities. General and administrative expenses included $2.5 million in stock-based compensation expenses for the first quarter of 2018.
Net Loss: Net loss was $56.5 million for the first quarter of 2018, or a net loss per share of $1.29, as compared to a net loss of $28.0 million for the first quarter of 2017, or a net loss per share of $0.84.
Financial Guidance:

Based on its current plans, Blueprint Medicines expects that its existing cash, cash equivalents and investments, excluding any potential option fees and milestone payments under its existing collaboration with Roche, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the middle of 2020.

Conference Call Information:

Blueprint Medicines will host a live conference call and webcast today at 8:30 a.m. ET. The conference call may be accessed by dialing (855) 728-4793 (domestic) or (503) 343-6666 (international) and referring to conference ID 7572918. A webcast of the conference call will be available in the Investors section of the Blueprint Medicines’ website at View Source The archived webcast will be available on Blueprint Medicines’ website approximately two hours after the conference call and will be available for 30 days following the conference call.