On November 8, 2017 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the third quarter of 2017 and provided a business update (Press release, Regeneron, NOV 8, 2017, View Source [SID1234521761]).

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!

Financial Highlights

($ in millions, except per share data)

Three Months Ended
September 30,

2017

2016

% Change
EYLEA U.S. net product sales

$
953

$
854

12%
Total revenues

$
1,501

$
1,220

23%
GAAP net income

$
388

$
265

46%
GAAP net income per share – diluted

$
3.32

$
2.27

46%
Non-GAAP net income(2)

$
470

$
365

29%
Non-GAAP net income per share – diluted(2)

$
3.99

$
3.13

27%
"In the third quarter, Regeneron made significant progress with our commercialized medicines, including continued strong global sales for our retinal therapy EYLEA and the completion of enrollment in our Phase 3 PANORAMA study in diabetic retinopathy, which represents an important new potential indication for EYLEA. We also saw robust U.S. launch progress with Dupixent in moderate-to-severe atopic dermatitis, and a favorable U.S. appellate court ruling for Praluent in our ongoing PCSK9 antibody litigation," said Leonard S. Schleifer, M.D., Ph.D., President and Chief Executive Officer of Regeneron. "Looking forward, we anticipate a U.S. regulatory submission for dupilumab in uncontrolled asthma later this year and continue to advance a broad dupilumab development program in other Type 2 allergic diseases. In addition, we are making important strides in our immuno-oncology program and expect to submit our first U.S. regulatory application for cemiplimab, our PD-1 antibody, in advanced cutaneous squamous cell carcinoma in early 2018."

Business Highlights

Marketed Product Update

EYLEA (aflibercept) Injection for Intravitreal Injection

In the third quarter of 2017, net sales of EYLEA in the United States increased 12% to $953 million from $854 million in the third quarter of 2016. Overall distributor inventory levels remained within the Company’s one- to two-week targeted range.
In the third quarter of 2017, enrollment was completed in the Phase 3 PANORAMA study in patients with non-proliferative diabetic retinopathy without diabetic macular edema (DME).
Bayer commercializes EYLEA outside the United States. In the third quarter of 2017, net sales of EYLEA outside of the United States(1) were $564 million, compared to $471 million in the third quarter of 2016. In the third quarter of 2017, Regeneron recognized $205 million from its share of net profit from EYLEA sales outside the United States, compared to $171 million in the third quarter of 2016.
Dupixent (dupilumab) Injection

Dupilumab, an antibody that blocks signaling of IL-4 and IL-13, is currently being studied in asthma, pediatric atopic dermatitis, nasal polyps, and eosinophilic esophagitis (EoE).
In the third quarter of 2017, global net sales of Dupixent were $89 million, which were almost exclusively in the United States. Product sales for Dupixent are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Dupixent.
In September 2017, the European Commission granted marketing authorization for Dupixent for use in adults with moderate-to-severe atopic dermatitis who are candidates for systemic therapy.
In September 2017, the Company and Sanofi presented positive results from the Phase 3 LIBERTY AD CAFÉ study in atopic dermatitis at the annual European Academy of Dermatology and Venereology (EADV) Congress.
In September 2017, the Company and Sanofi announced that the Phase 3 LIBERTY ASTHMA QUEST study of dupilumab in a broad population of adults and adolescents with uncontrolled, persistent asthma met its two primary endpoints.
In October 2017, the Company and Sanofi announced that the Phase 3 LIBERTY ASTHMA VENTURE study evaluating dupilumab in adults and adolescents with severe, steroid-dependent asthma met its primary endpoint and key secondary endpoints.
In September 2017, the FDA granted orphan drug designation for the treatment of EoE.
In October 2017, the Company and Sanofi presented positive results from the Phase 2 study in adults with active moderate-to-severe EoE at the World Congress of Gastroenterology.
Praluent (alirocumab) Injection for the Treatment of Elevated Low-Density Lipoprotein (LDL) Cholesterol

In the third quarter of 2017, global net sales of Praluent were $49 million, compared to $38 million in the third quarter of 2016. Product sales for Praluent are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Praluent.
In October 2017, the U.S. Court of Appeals for the Federal Circuit ordered a new trial on the issues of written description and enablement and vacated the permanent injunction in the ongoing PCSK9 litigation.
A Phase 3 study in homozygous familial hypercholesterolemia (HoFH) was initiated in the fourth quarter of 2017.
Kevzara (sarilumab) Injection

In the third quarter of 2017, global net sales of Kevzara were $3 million. Product sales for Kevzara are recorded by Sanofi, and the Company shares in any profits or losses from the commercialization of Kevzara.
In September 2017, the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan approved Kevzara for the treatment of adult patients with rheumatoid arthritis who have had an inadequate response to conventional treatments.
Pipeline Progress

Regeneron has sixteen product candidates in clinical development, which consist of EYLEA and fully human monoclonal antibodies generated using the Company’s VelocImmune technology, including six in collaboration with Sanofi. In addition to EYLEA, Dupixent, Praluent, and Kevzara discussed above, updates from the clinical pipeline include:

Cemiplimab (REGN2810), an antibody to programmed cell death protein 1 (PD-1), is being studied in patients with cancer.

In the third quarter of 2017, the FDA granted Breakthrough Therapy designation for the treatment of adults with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC.
A pivotal Phase 2 study in metastatic or locally advanced and unresectable CSCC is ongoing.
A Phase 3 study in cervical cancer was initiated in the third quarter of 2017.
Fasinumab is an antibody targeting Nerve Growth Factor (NGF). A Phase 3 efficacy study of fasinumab compared to placebo or naproxen in patients with pain due to osteoarthritis of the knee or hip was initiated in the third quarter of 2017.

Suptavumab is an antibody to the Respiratory Syncytial Virus-F (RSV-F). In August 2017, the Company reported that a Phase 3 study evaluating suptavumab did not meet its primary endpoint of preventing medically-attended RSV infections in infants. Further clinical development of suptavumab has been discontinued.

Select Upcoming 2017 Milestones

Programs

Milestones
EYLEA
•
File sBLA with FDA for every 12-week dosing interval in
neovascular age-related macular degeneration (wet AMD)
Dupixent
•
Submit sBLA for asthma in adult/adolescent patients
•
Initiate Phase 3 studies in younger pediatric patients in
atopic dermatitis
Praluent
•
Complete ODYSSEY OUTCOMES study (with data
expected in early 2018)

•
File sBLA with FDA for use with apheresis
Cemiplimab (PD-1 Antibody)
•
Report interim data from pivotal Phase 2 CSCC study
Fasinumab (NGF Antibody)
•
Initiate Phase 3 study in patients with both chronic low back
pain and osteoarthritis
Nesvacumab/aflibercept
(Ang2 Antibody co-formulated
with aflibercept)
•
Report top-line data from Phase 2 studies in DME (RUBY)
and wet AMD (ONYX)
REGN2477 (Activin A
Antibody)
•
Initiate Phase 2 study in patients with Fibrodysplasia
Ossificans Progressiva (FOP)
Third Quarter 2017 Financial Results

Product Revenues: Net product sales were $957 million in the third quarter of 2017, compared to $857 million in the third quarter of 2016. EYLEA net product sales in the United States were $953 million in the third quarter of 2017, compared to $854 million in the third quarter of 2016.

Total Revenues: Total revenues, which include product revenues described above, increased by 23% to $1.501 billion in the third quarter of 2017, compared to $1.220 billion in the third quarter of 2016. Total revenues include Sanofi and Bayer collaboration revenues of $482 million in the third quarter of 2017, compared to $336 million in the third quarter of 2016. Sanofi collaboration revenue in the third quarter of 2017 included higher reimbursements by Sanofi in connection with validating the Company’s commercial manufacturing facilities and the recognition of a higher amount of previously deferred revenue from up-front and other payments in connection with the Company’s Antibody Discovery Agreement which will end on December 31, 2017 without any extension.

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

Research and Development (R&D) Expenses: GAAP R&D expenses were $530 million in the third quarter of 2017, compared to $543 million in the third quarter of 2016. The lower R&D expenses in the third quarter of 2017 were principally due to a $25 million up-front payment made in connection with the license and collaboration agreement with Adicet Bio in the third quarter of 2016 and a decrease in clinical manufacturing activities, partly offset by an increase in cemiplimab clinical trial costs. In addition, in the third quarter of 2017, R&D-related non-cash share-based compensation expense was $70 million, compared to $81 million in the third quarter of 2016.

Selling, General, and Administrative (SG&A) Expenses: GAAP SG&A expenses were $307 million in the third quarter of 2017, compared to $270 million in the third quarter of 2016. The higher selling, general, and administrative expenses were primarily due to the launches of Dupixent and Kevzara as well as an increase in commercialization-related expenses associated with EYLEA. In the third quarter of 2017, SG&A-related non-cash share-based compensation expense was $48 million, compared to $49 million in the third quarter of 2016.

Cost of Collaboration and Contract Manufacturing (COCM): GAAP COCM was $58 million in the third quarter of 2017, compared to $14 million in the third quarter of 2016. The higher COCM costs were primarily due to validation activities at the Company’s Limerick commercial manufacturing facility related to products that are in collaboration with Sanofi.

Income Tax Expense: In the third quarter of 2017, GAAP income tax expense was $177 million and the effective tax rate was 31.3%, compared to $101 million and 27.6% in the third quarter of 2016. The effective tax rate for the third quarter of 2017 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 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.

GAAP and Non-GAAP Net Income(2): The Company reported GAAP net income of $388 million, or $3.64 per basic share and $3.32 per diluted share, in the third quarter of 2017, compared to GAAP net income of $265 million, or $2.53 per basic share and $2.27 per diluted share, in the third quarter of 2016.

The Company reported non-GAAP net income of $470 million, or $4.41 per basic share and $3.99 per diluted share, in the third quarter of 2017, compared to non-GAAP net income of $365 million, or $3.48 per basic share and $3.13 per diluted share, in the third quarter of 2016.

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

On November 8, 2017 RXi Pharmaceuticals Corporation (NASDAQ: RXII), a clinical-stage RNAi company developing innovative therapeutics that address significant unmet medical needs, reported its financial results for the third quarter ended September 30, 2017, and provided a business update (Press release, RXi Pharmaceuticals, NOV 8, 2017, View Source [SID1234521762]).

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!

"RXi maintained a conservative spend rate in the third quarter in line with its projected budget as it continues to execute on its strategy with anticipated readouts this quarter from our dermatology and ophthalmology clinical programs. Data collection and analysis is ongoing, and the release of data is on track as previously reported," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He further added that, "Our team has made notable progress with research efforts and data generation using our proprietary self-delivering RNAi technology platform (sd-rxRNA) for use in cancer therapeutics. sd-rxRNA compounds demonstrate high transfection efficiency with high cell viability in a number of immune cells. We believe that these assets have the potential to become a foundation for future growth opportunities, including several therapeutic approaches in the rapidly growing field of cell therapy for oncology. We look forward to successful and meaningful growth through our work as well as partnerships in this exciting and highly valuable medical field."

A live audio webcast will begin today at 4:30 p.m. ET. The webcast link is available under the "Investors – Events and Presentations" section of the Company’s website, www.rxipharma.com. The event may also be accessed by dialing toll-free in the United States: +1 (844) 376-4678. International participants may access the event by dialing: +1 (209) 905-5958. An archive of the webcast will be available on the company’s website approximately two hours after the presentation.

Select Third Quarter 2017 Financial Highlights

Cash Position

At September 30, 2017, the Company had cash of $5.4 million, compared with $12.9 million at December 31, 2016.

On August 8, 2017, the Company entered into a purchase agreement with Lincoln Park Capital Fund, LLC ("LPC"), pursuant to which the Company has the right to sell to LPC up to $15 million in shares of the Company’s common stock over the 30-month term of the agreement. We expect to use proceeds from the purchase agreement for general corporate purposes, including but not limited to the advancement of our immunotherapy program, our clinical trials, and general and administrative expenses. As of September 30, 2017, there have been no purchases under the agreement with LPC.

Research and Development Expenses

Research and development expenses for the quarter ended September 30, 2017 were $1.5 million, as compared with $1.5 million for the quarter ended September 30, 2016. Research and development expenses were consistent quarter over quarter, with slight increases due to subject fees for the second cohort in the Samcyprone Phase 2 clinical trial and preclinical work in the Company’s new immunotherapy program that was integrated into the Company with the acquisition of MirImmune in the first quarter of 2017, which were offset by a decrease in stock-based compensation expense.

General and Administrative Expenses

General and administrative expenses for the quarter ended September 30, 2017 were $1.0 million, as compared with $0.8 million for the quarter ended September 30, 2016. The increase in general and administrative expenses was primarily due to payroll-related expenses, including severance benefits, with the hire of the Company’s former chief business officer in connection with the acquisition of MirImmune, resulting in a higher employee headcount as compared to the same period of the prior year, offset by a decrease in stock-based compensation expense.

Net Loss

Net loss for the three months ended September 30, 2017 was $2.5 million, compared with $2.2 million for the three months ended September 30, 2016. The increase in net loss was primarily driven by the changes in general and administrative expenses, as discussed above.

Nasdaq Compliance

On August 2, 2017, the NASDAQ Stock Market provided written notice and granted the Company an additional 180 calendar days to regain compliance with the minimum bid price requirements set forth in the NASDAQ listing rules. As a result of this extension, the Company has until January 29, 2018 to regain compliance by maintaining a closing bid price of at least $1.00 for 10 consecutive business days. The NASDAQ written notice has no effect on the listing of the Company’s common stock at this time.

Select Third Quarter 2017 and Recent Corporate Highlights

Select Business and Corporate Highlights

sd-rxRNA: Broadly applicable to numerous development areas providing continued and expanded growth

Grant Award – Development of self-delivering RNAi targeted to PTEN for treatment of spinal cord injury

The Company’s proprietary self-delivering platform (sd-rxRNA) is broadly applicable to numerous therapeutic areas. For example, BioAxone Biosciences was recently awarded a grant from the National Institute of Neurological Disorders and Stroke (NINDS), part of the agency’s SBIR Phase II funding program, to fund further development of BioAxone’s preclinical candidate BA-434 in collaboration with RXi Pharmaceuticals. This two-year grant provides funding for further development of BA-434, a novel sd-rxRNA compound that targets PTEN for the treatment of spinal cord injury.

BioAxone has been awarded a total of $1,794,895 to fund the collaborative project over 24 months. For their contribution, RXi will receive approximately $129,000 in the first year with the potential to receive an additional $118,800 in the second year after achieving certain milestones.

Cell Therapy for Oncology

The Company’s ongoing research programs with its sd-rxRNA platform have demonstrated robust cellular uptake in a number of immune cell types including, human T-cells, meso CAR-T, human NK, and dendritic cells. Our internal preclinical programs are focused on development of sd-rxRNA compounds for optimizing existing cell-based therapy treatment paradigms in oncology. In addition, the Company is actively seeking partnerships and collaborations with industry and academia to develop new technologies using engineered cells and our sd-rxRNA compounds. Our scientific team and advisors provide a strong foundation for the development of novel therapeutic treatment approaches using sd-rxRNA. This support positions RXi well with opportunities to provide meaningful growth for the Company.

Direct Therapeutic Use

RXI-109-1402 – Hypertrophic Scarring

The Company’s ongoing Phase 2 clinical trial, RXI-109-1402, is being conducted to evaluate RXI-109, a sd-rxRNA compound targeting connective tissue growth factor (CTGF), a key regulator of scar formation. This open-label, multi-center study is designed to evaluate the effectiveness and safety of RXI-109 to reduce scar formation in healthy volunteers post scar revision surgery. The Company expects to share final study outcomes before the end of this year.

RXI-109-1501 – Retinal Scarring in Advanced Age-related Macular Degeneration (AMD)

Enrollment is complete in this Phase 1/2 study evaluating the safety and clinical activity of RXI-109 to prevent the progression of retinal scarring, a harmful component of numerous retinal diseases. This study is a multi-dose, dose escalation trial conducted in patients with advanced neovascular or wet age-related macular degeneration (AMD) where retinal scarring can result in continued vision loss. The primary endpoint for RXI-109-1501 is to evaluate the safety and tolerability of RXI-109. Additional endpoints will assess RXI-109’s potential for clinical activity using numerous assessments to monitor ocular health and visual acuity. The Company expects to complete subject participation in the study by the end of 2017 and share top-line data in early 2018.

RXI-SCP-1502 – Treatment of Cutaneous Warts

Samcyprone, a proprietary topical formulation of the small molecule diphenylcyclopropenone (DPCP), is being evaluated in a Phase 2a clinical trial. RXI-SCP-1502 is a multi-center, multi-dose trial conducted in subjects with at least one cutaneous, plantar or periungual wart. The Company expects to share early read-outs before the end of this year.

Direct Consumer Care Use

RXI-231 – Consumer Care Products

The Company initiated a consumer testing program with RXI-231, a cosmetic ingredient based on sd-rxRNA that targets tyrosinase (TYR). The cosmetic product is a gel formulation designed to aid in the reduction of pigmentation and thereby improving skin appearance. The consumer testing program will evaluate the use and consumer acceptability of RXI-231.

There are three studies under this program. The first two studies in volunteers are performed to determine irritation and sensitization potential of the gel product containing RXI-231 when applied to the skin. A third study investigates the potential of the product to improve the appearance of skin pigmentation induced by UV exposure. The Company projects to report results before the end of this year.

Corporate Update

Management Team

On September 15, 2017, the Company announced the departure of Dr. Alexey Eliseev, former CEO of MirImmune Inc. Dr. Eliseev joined RXi on January 6, 2017 as Chief Business Officer of the Company in connection with its acquisition of MirImmune. MirImmune was a privately-held company developing cell-based therapeutics for cancer treatments based on a license to RXi’s proprietary self-delivering RNAi technology. In his role as Chief Business Officer of RXi, Dr. Eliseev was responsible for the integration of this new therapeutic approach into the activities of RXi Pharmaceuticals, and for introducing the Company to several key industry and academic groups active in this emerging field.

Dr. James Cardia, RXi’s current Director of Business Development and Intellectual Property now leads the management of the various ongoing activities in partnering and business development. During his tenure at RXi, Dr. Cardia’s group was responsible for the discovery and optimization of "self-delivering" rxRNAs (sd-rxRNAs) as well as the development and characterization of RXI-109, a promising anti-fibrotic agent currently in clinical trials for the treatment of both dermal and retinal scarring.

On November 8, 2017 Sierra Oncology, Inc. (NASDAQ: SRRA), a clinical stage drug development company focused on advancing next generation DNA Damage Response (DDR) therapeutics for the treatment of patients with cancer, reported its financial and operational results for the third quarter ended September 30, 2017 (Press release, Sierra Oncology, NOV 8, 2017, View Source [SID1234521764]).

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!

"During the quarter, we continued to aggressively drive the clinical program for SRA737, expanding the number of sites recruiting patients into our Monotherapy trial from three to eight leading centers across the United Kingdom. With growing investigator interest for evaluating our Chk1 inhibitor, we anticipate further expanding this trial into additional sites over the coming months. We plan to provide an update on the SRA737 development program in February 2018 and remain on track to report interim clinical results at a medical conference in the second half of 2018," said Dr. Nick Glover, President and CEO of Sierra Oncology.

"Subsequent to the end of the quarter, we presented a poster at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) reporting on preclinical data demonstrating that sub-therapeutic, non-cytotoxic doses of gemcitabine induce replication stress and hence potentiate the anti-tumor activity of SRA737. These data support the design of our second ongoing Phase 1 clinical trial which is specifically evaluating this combination and is also proceeding as planned," added Dr. Glover. "As we advance our two trials, we are also preparing for a potential clinical study of SRA737 in combination with a PARP inhibitor, where we see a strong biological rationale to expect synergy with Chk1 inhibition. We are also excited about the emerging evidence of biological synergy between immune checkpoint blockade and Chk1 inhibition, and we are currently designing a potential clinical study for this combination as well."

"We continue to advance SRA141, our potent, selective, orally bioavailable small molecule inhibitor of Cell division cycle 7 kinase (Cdc7), an emerging DDR target with exciting potential in cancer. SRA141 is currently undergoing preclinical research in preparation for an Investigational New Drug (IND) filing. Given our near-term focus on broadening the development program for our lead asset SRA737, we are evaluating when best to advance SRA141 into the clinic within the context of our current operational and capital resources," added Dr. Glover.

"Our development program for SRA737 continues to be strongly supported by our collaboration with Cancer Research UK and the Institute of Cancer Research, the originators of SRA737. During the quarter, we also announced the formation of our new DDR Advisory Committee, consisting of leading experts in the DDR field. We recently held two Key Opinion Leader (KOL) events which featured members from our Advisory Committee who shared their insights on emerging DDR biology and the potential of Chk1 inhibition in oncology, providing further support for our programs," concluded Dr. Glover. Recordings of the Sierra’s recent KOL events are accessible at www.sierraoncology.com/events-and-webcasts.

Third Quarter 2017 Financial Results (all amounts reported in U.S. currency)

Research and development expenses were $7.4 million for the three months ended September 30, 2017, compared to $12.3 million for the three months ended September 30, 2016. The decrease is primarily due to fees incurred in 2016 in accordance with the license agreement for SRA737: a $7.0 million upfront license fee for the exclusive license of SRA737, and a $2.0 million fee related to the successful transfer to the company of the two ongoing clinical trials of SRA737. These decreased costs were partially offset by a $3.5 million increase in clinical trial costs, third-party manufacturing costs and research and other costs related to SRA737 and SRA141. Research and development expenses included non-cash stock-based compensation of $1.0 million and $0.9 million for the three months ended September 30, 2017 and 2016.

Research and development expenses were $22.6 million for the nine months ended September 30, 2017, compared to $28.1 million for the nine months ended September 30, 2016. The decrease is primarily due to expenses incurred in 2016, including fees related to the SRA737 license agreement, a $0.9 million upfront payment for the exclusive license of SRA141, and a $2.4 million restructuring charge related to close-out expenses for PNT2258. These decreases were partially offset by increases of $4.4 million in third-party manufacturing costs and $2.4 million in research and other costs related to SRA737 and SRA141. Research and development expenses included non-cash stock-based compensation of $3.0 million and $2.6 million for the nine months ended September 30, 2017 and 2016.

General and administrative expenses were $2.8 million for the three months ended September 30, 2017, compared to $3.0 million for the three months ended September 30, 2016. General and administrative expenses were $9.2 million for the nine months ended September 30, 2017, compared to $10.8 million for the nine months ended September 30, 2016. The decrease for the nine months ended September 30, 2017 compared to the prior period was primarily due to a $1.0 million decrease in business development expenses and a $0.4 million decrease in restructuring costs as compared to 2016. General and administrative expenses included non-cash stock-based compensation of $0.5 million and $1.5 million for the three and nine months ended September 30, 2017, and $0.4 million and $1.4 million for the three and nine months ended September 30, 2016.

For the three months ended September 30, 2017, Sierra incurred a net loss of $10.0 million compared to a net loss of $15.2 million for the three months ended September 30, 2016. For the nine months ended September 30, 2017, Sierra incurred a net loss of $31.4 million compared to a net loss of $38.6 million for the nine months ended September 30, 2016.

Cash and cash equivalents totaled $107.8 million as of September 30, 2017, compared to $116.7 million as of June 30, 2017, and $109.0 million as of December 31, 2016. The company believes that its existing cash and cash equivalents will be sufficient to fund current operating plans through approximately mid-2019.

At September 30, 2017, there were 52,268,443 shares of common stock issued and outstanding, and stock options to purchase 7,685,449 shares of common stock issued and outstanding.

On November 8, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2017, and recent company developments (Press release, TG Therapeutics, NOV 8, 2017, View Source [SID1234521767]).

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!

Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "The third quarter was an extremely productive and exciting time for the Company highlighted by the completion of enrollment into our UNITY-CLL Phase 3 study, the commencement of our global Phase 3 trials in multiple sclerosis, and the additional clarity we received from the FDA regarding the GENUINE study. We look forward, over the next 6-12 months, to what we believe will be a number of value creating milestones, including overall response data from UNITY-CLL and additional data supporting our strategy in NHL." Mr. Weiss continued, "From a financial perspective, we remain well positioned through these important milestones."

Third Quarter and Recent Highlights

ASH 2017: The Company looks forward to the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting where data presentations will include three clinical poster presentations and three pre-clinical poster presentations.
TG-1101 Data at ECTRIMS: Updated results from the ongoing Phase 2 Study of TG-1101 in patients with Multiple Sclerosis were presented at the 7th Joint ECTRIMS-ACTRIMS Meeting demonstrating robust activity on B-cell depletion, reduction of T1 Gd enhancing lesions, and positive effects on disability measurements.
UNITY-CLL Enrollment: Full enrollment in the UNITY-CLL Phase 3 Trial was completed in October, which should allow for top-line data on Overall Response Rate (ORR) in Q2 2018.
GENUINE Update: The Company met with the FDA and confirmed that accelerated approval based on the ORR results from GENUINE would be a review issue and that the potential may exist for full approval based on the PFS results from the GENUINE study.
TGR-1202 Grant: TGR-1202 (umbralisib) was selected for a grant by the National Multiple Sclerosis Society to support the development of TGR-1202 as an oral B-Cell targeted treatment option in progressive Multiple Sclerosis (PMS).
Anti-PD-L1 Entered the Clinic: The Company’s anti-PD-L1 monoclonal antibody commenced clinical development, with the first patient being dosed in a Phase I clinical trial.
ULTIMATE Phase 3 Trials in MS: Received a Special Protocol Assessment (SPA) for the Phase 3 ULTIMATE I and II studies in relapsing forms of multiple sclerosis and commenced enrollment into the global studies.
UNITY-NHL: Announced successful outcome from the first pre-planned interim analysis by independent DSMB of the DLBCL cohort in the UNITY-NHL Phase 2b trial, where based on pre-set hurdles of ORR, the DSMB recommended continued enrollment in the TG-1101 plus TGR-1202 combination arm (also referred to as the U2 combination) and replacement of the single agent TGR-1202 arm with U2 plus bendamustine.
Financial Results for the Third Quarter 2017

Cash Position: Cash, cash equivalents, investment securities, and interest receivable were $91.8 million as of September 30, 2017, as compared to $86.5 million at June 30, 2017.
R&D Expenses: Research and development (R&D) expenses were $27.1 million and $76.5 million for the three and nine months ended September 30, 2017, respectively, compared to $21.8 million and $46.9 million for the three and nine months ended September 30, 2016. Included in research and development expense for the three and nine months ended September 30, 2017 was $7.1 million and $20.4 million, respectively, of manufacturing and CMC expenses for Phase 3 clinical trials and potential commercialization. The increase in R&D expenses for both the three and nine months ended September 30, 2017, is primarily due to the ongoing clinical development programs and related manufacturing costs for TG-1101 and TGR-1202.
G&A Expenses: General and administrative (G&A) expenses were $4.5 million and $11.3 million for the three and nine months ended September 30, 2017, respectively, as compared to $3.2 million and $8.1 million for the three and nine months ended September 30, 2016. The increase in G&A expenses for the nine months ended September 30, 2017 relates primarily to non-cash compensation expenses related to equity incentive grants recognized during 2017. We expect G&A expenses to remain relatively constant through the remainder of 2017.
Net Loss: Net loss was $31.5 million and $87.6 million for the three and nine months ended September 30, 2017, respectively, compared to a net loss of $24.8 million and $54.6 million for the three and nine months ended September 30, 2016, respectively.
Financial Guidance: The Company believes its cash and cash equivalents will be sufficient to fund the Company’s planned operations through 2018.
Conference Call Information

The Company will host an investor conference call today, November 8, 2017, at 8:30am ET, to discuss the Company’s third quarter 2017 financial results and provide a business outlook for the remainder of 2017.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Third Quarter 2017 Earnings Call. A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

On November 8, 2017 Pacira Pharmaceuticals, Inc. (NASDAQ: PCRX) reported consolidated financial results for the third quarter ended September 30, 2017 (Press release, Pacira Pharmaceuticals, NOV 8, 2017, View Source;p=RssLanding&cat=news&id=2315327 [SID1234521756]).

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!

"We continue to make important progress during 2017 as we advance our strategy to expand the role of EXPAREL as the only long-acting local analgesic capable of providing non-opioid pain control during the most intense period of postsurgical pain," said Dave Stack, chairman and chief executive officer of Pacira. "Recent highlights include the FDA acceptance of our sNDA for nerve block; the publication of new research quantifying the devastating impact of opioids for postsurgical pain; a unique collaboration with Aetna to reduce opioid use associated with impacted wisdom teeth extractions; and the advancement of our important partnership with J&J."

"During the quarter we generated year over year EXPAREL growth despite fewer selling days and the impact of weather in areas of the southern United States representing about 20 percent of our business. From our commercial initiatives and current trends however; we remain confident in the growth potential of EXPAREL and our steadfast commitment to reducing the use of opioids for postsurgical pain."

Recent Highlights

U.S. Food and Drug Administration (FDA) Acceptance of Supplementary New Drug Application for Nerve Block. In October, the FDA accepted the company’s resubmission of its supplemental new drug application (sNDA) seeking expansion of the EXPAREL (bupivacaine liposome injectable suspension) label to include administration via nerve block for prolonged regional analgesia. If approved, EXPAREL could help eliminate the need for cumbersome devices like pumps and catheters and shift numerous procedures to an outpatient setting. The expected action date for the FDA under the Prescription Drug User Fee Act (PDUFA) is April 6, 2018.
Provided Invited Testimony at White House Opioid Crisis Commission Meeting. In September, Pacira Chief Executive Officer Dave Stack testified before President Trump’s Commission on Combating Drug Addiction and the Opioid Crisis. The Commission, led by New Jersey Governor Chris Christie, was created to study ways to combat and treat drug abuse, addiction and the opioid crisis. Stack provided insights into the critical nature of clinician and patient access to non-opioid medications that can effectively manage postsurgical pain while reducing opioid requirements.
Published New Research Highlighting Serious Threats Associated with Overprescribing Postsurgical Opioids. Newly published research, conducted by the QuintilesIMS Institute, shows individuals undergoing surgery are at particular risk for long-term opioid use. An overwhelming majority of patients (nine in 10) are exposed to opioids to manage postsurgical pain, and those given prescriptions received an average of 85 pills each. In addition, nearly 3 million individuals who had surgery in 2016 became persistent opioid users, according to the research. This report, The United States for Non-Dependence, represents the most current analysis of national trends in opioid prescribing.
Collaboration with Aetna and the American Association of Oral and Maxillofacial Surgeons to Reduce Opioid Use. In September, Pacira announced a nationwide collaboration with Aetna and the American Association of Oral and Maxillofacial Surgeons (AAOMS) aimed at reducing the number of opioid tablets prescribed to patients undergoing impacted third molar extraction by at least 50 percent through the utilization of EXPAREL to provide prolonged non-opioid postsurgical pain control.
Multiple EXPAREL Data Presentations at the New York School of Regional Anesthesia Annual Fall Symposium. In September, the company presented three posters evaluating the use of EXPAREL administered as a regional nerve block to manage postsurgical pain at the New York School of Regional Anesthesia’s (NYSORA) 16th Annual Symposium on Regional Anesthesia, Pain and Perioperative Medicine.
Third Quarter 2017 Financial Results

EXPAREL net product sales were $66.8 million in the third quarter of 2017, a 3% increase over the $64.9 million reported for the third quarter of 2016. There were two fewer selling days in the third quarter of 2017 compared to the third quarter of 2016.
Total revenues were $67.3 million in the third quarter of 2017, a 1% decrease versus the $68.4 million reported for the third quarter of 2016, primarily related to the discontinuation of DepoCyt(e) and lower collaborative licensing and milestone revenue.
Total operating expenses were $70.9 million in the third quarter of 2017, compared to $89.2 million in the third quarter of 2016.
GAAP net loss was $7.6 million, or $(0.19) per share (basic and diluted), in the third quarter of 2017, compared to a GAAP net loss of $22.2 million, or $(0.59) per share (basic and diluted), in the third quarter of 2016.
Non-GAAP net income was $4.4 million, or $0.11 per share (basic and diluted) in the third quarter of 2017, compared to non-GAAP net income of $8.0 million, or $0.22 per share (basic) and $0.20 per share (diluted), in the third quarter of 2016.
Pacira ended the third quarter of 2017 with cash, cash equivalents, short-term and long-term investments ("cash") of $374.9 million.
Pacira had 40.5 million basic weighted average shares of common stock outstanding in the third quarter of 2017.
For non-GAAP measures, Pacira had 41.4 million diluted weighted average shares of common stock outstanding in the third quarter of 2017.
2017 Outlook

Pacira is updating its full year 2017 sales guidance and reiterating its remaining financial guidance as follows:

EXPAREL net product sales of $280 million to $285 million from its previously guided range of $290 million to $310 million.
Non-GAAP gross margins of approximately 70%.
Non-GAAP research and development (R&D) expense of $50 million to $60 million.
Non-GAAP selling, general and administrative (SG&A) expense of $145 million to $155 million.
Stock-based compensation of $30 million to $35 million.
See "Non-GAAP Financial Information" and "Reconciliations of GAAP to Non-GAAP 2017 Financial Guidance" below.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Wednesday, November 8, 2017, at 8:30 a.m. ET. The call can be accessed by dialing 1-877-845-0779 (domestic) or 1-720-545-0035 (international) ten minutes prior to the start of the call and providing the Conference ID 96590192.

A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing 1-855-859-2056 (domestic) or 1-404-537-3406 (international) and providing the Conference ID 96590192. The replay of the call will be available for two weeks from the date of the live call.

The live, listen-only webcast of the conference call can also be accessed by visiting the "Investors & Media" section of the company’s website at investor.pacira.com. A replay of the webcast will be archived on the Pacira website for two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP net income (loss), non-GAAP cost of goods sold, non-GAAP gross margins, non-GAAP research and development (R&D), non-GAAP selling, general and administrative (SG&A) and non-GAAP product discontinuation expenses, because such measures exclude stock-based compensation, amortization of debt discount, loss on early extinguishment of debt, a contract termination fee with CrossLink BioScience, LLC, or CrossLink, exit costs related to the discontinuation of DepoCyt(e) production and inventory and related reserves from 2016.

These measures supplement the company’s financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future cost of goods sold, gross margins, R&D and SG&A outlook for 2017 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and the company’s future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See the tables below for a reconciliation of GAAP to non-GAAP measures, and a reconciliation of our GAAP to non-GAAP 2017 financial guidance for gross margins, R&D and SG&A.