On November 8, 2017 Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx, reported its financial results for the nine months ended September 30, 2017 and provided a corporate update (Press release, Protalix, NOV 8, 2017, View Source;p=RssLanding&cat=news&id=2315313 [SID1234521760]).

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"I am excited about the progress we made recently," said Moshe Manor, Protalix’s President and Chief Executive Officer. "We are no longer facing any debt issue or financing overhang. Moreover, with the recent strategic collaboration with Chiesi, not only did we secure a strong, experienced clinical and commercial partner, but we also meaningfully increased our capital resources, providing us with sufficient cash into 2020, irrespective of any milestone payments we are entitled to from Chiesi, increased revenues or from future partnerships. In addition, on the strategic front, we now have three novel and clinically differentiated product candidates in the clinic with potentially superior efficacy which provides us multiple shots at goal to realize significant value for our stockholders."

Third Quarter and Recent Clinical Highlights

Pegunigalsidase alfa (PRX-102) for Fabry Disease

Protalix entered into an EX-US collaboration agreement with Chiesi Farmaceutici S.p.A., or Chiesi, for pegunigalsidase alfa, or PRX-102. Under the terms of the agreement, Protalix is entitled to an upfront payment of $25 million from Chiesi, up to $25 million to cover development costs for pegunigalsidase alfa, subject to a maximum of $10 million per year, and up to an additional $320 million in regulatory and commercial milestone payments. Additionally, Protalix is entitled to tiered payments of 15% to 35% of Chiesi’s net sales.

Patient enrollment in the Company’s phase III clinical trials, referred to as the Balance, Bridge and Bright studies, is on-going.
Alidornase alfa (PRX-110) for Cystic Fibrosis

Protalix applied for a financial grant from the Cystic Fibrosis Foundation to support the clinical development of alidornase alfa.

A poster titled "Development of Novel Actin Inhibition Resistant DNase I Enzyme – alidornase alfa – for the Treatment of Cystic Fibrosis" was presented at the North American Cystic Fibrosis Conference held in November 2017.
Oral antiTNF (OPRX-106) for Ulcerative Colitis

The final patients needed to complete enrollment in the Company’s phase II clinical trial of OPRX-106 for the treatment of ulcerative colitis are in the screening process. The Company remains on track to complete enrollment during the fourth quarter and report top-line results in early 2018.
Alfataliglicerase for Gaucher Disease

Shipment of approximately $3.0 million of alfataliglicerase was completed this quarter for a total of $6.6 million for the nine months ended September 30, 2017.

According to the purchase order received by the Company, additional shipments are scheduled to be made during fourth quarter of 2017, and into 2018.
Financial Results for Nine Months ended September 30, 2017

The Company reported a net loss of $32.1 million, or $0.25 per share, basic and diluted, for the nine-month period ended September 30, 2017, excluding a one-time, non-cash net charge of $38.1 million in connection with the remeasurement of a derivative, compared to a net loss of $26.7 million, or $0.27 per share, basic and diluted, for the same period of 2016.

The Company recorded total revenues of $16.8 million for the nine-month period ended September 30, 2017, compared to $7.1 million during the same period of 2016. The increase is primarily the result of increased sales of drug product to Brazil of $6.6 million in the nine months ended September 30, 2017, compared to $2.7 million in the same period of 2016, and the sale of drug substance to Pfizer Inc.

Research and development expenses, net were $19.8 million for the nine-month period ended September 30, 2017, compared to $18.9 million for the same period of 2016. Selling, general and administrative expenses were $8.2 million for the nine-month period ended September 30, 2017, compared to $6.2 million incurred during the same period of 2016. The increases are primarily attributed to increased activities in three ongoing clinical trials and selling in Brazil.

During the nine-month period ended September 30, 2017 and during October, note holders converted the entire $8.55 million in aggregate principal amount of the Company’s 4.50% convertible notes due 2022.

As of today, the Company’s outstanding convertible notes include 4.50% convertible notes due September 2018 with an aggregate principal amount of $5.9 million and senior secured 7.50% convertible notes due November 2021 with an aggregate principal amount of $61.9 million.

On September 30, 2017, the Company had $33.4 million of cash and cash equivalents. With the Company’s current cash, plus the additional $25 million upfront payment due from Chiesi, and without giving effect to any milestone payment we are entitled to from Chiesi, anticipated increase in revenue run rate or any additional potential partnerships, the Company has sufficient resources to fund operations into 2020.
Conference Call and Webcast Information

The Company will host a conference call on Wednesday, November 8, 2017, at 8:30 am ET to review the clinical, corporate and financial highlights.

To participate in the conference call, please dial the following numbers prior to the start of the call: United States: (844) 358-6760; International: (478) 219-0004. Conference ID number 6767278.

The conference call will also be broadcast live and available for replay for two weeks on the Company’s website, www.protalix.com, in the Events Calendar of the Investors section. Please access the Company’s website at least 15 minutes ahead of the conference to register, download, and install any necessary audio software.

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]).

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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]).

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"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]).

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"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]).

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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.