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ChemoCentryx Reports Third Quarter 2015 Financial Results and Provides Corporate Update

On November 9, 2015 ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders and cancer, reported financial results for the third quarter ended September 30, 2015 and provided an update on the Company’s corporate and clinical development activities (Press release, ChemoCentryx, NOV 9, 2015, View Source [SID:1234508151]).

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"We continue to achieve important milestones according to plan in the development of the CCXI pipeline," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Our novel drug candidates for orphan diseases, cancer, and renal pathologies are gaining increased visibility within the global medical community following presentations at high-profile medical meetings. We have several key milestones in the near-term, especially in our complement inhibitor CCX168 program, from which we expect to report top-line data from the ANCA Vasculitis Phase II CLEAR trial by the end of this year or in early January. We look forward to the CLEAR trial results which may provide promise for patients suffering from this devastating disease."

Recent Pipeline Developments Across Key Therapeutic Areas

Orphan and Rare Diseases: CCX168 is an orally-administered complement inhibitor targeting the C5a receptor (C5aR), and is being developed for several rare disease indications, including ANCA-associated vasculitis (AAV) and atypical Hemolytic Uremic Syndrome (aHUS). These are severe and often fatal autoimmune diseases that are characterized by inflammation that can destroy different organ systems. AAV is the lead indication in the Company’s orphan and rare disease program.

By damaging the body’s small blood vessels, AAV affects many organ systems, mostly the kidneys, eyes, lungs, sinuses and nerves. This damage is caused by the destructive activity of inflammatory leukocytes in the body, with neutrophils considered to be the terminal effector cell. In AAV, neutrophils are attracted to sites of vascular destruction as well as activated at those sites by the activity of the complement system product known as C5a and its receptor, C5aR, which is the target of CCX168.

AAV affects approximately 40,000 people in the US (with approximately 4,000 new cases each year) and greater than 75,000 people in Europe, with at least 7,500 new cases each year, and is currently treated with courses of immuno-suppressants (cyclophosphamide or rituximab) combined with high dose steroid administration. Following initial treatment, up to 30 percent of patients relapse within 6 to 18 months, and approximately 50% of all patients will relapse within 3 to 5 years.

Current standard of care for AAV is associated with significant safety issues. First year mortality is approximately 11 to 18 percent. The single major cause of premature mortality is not disease related adverse events, but rather infection that is thought largely to be a consequence of steroid administration. Indeed, the multiple adverse effects of courses of steroid treatment (both initial courses and those that are repeated as a consequence of relapse) are major causes of both short-term and long-term disease and death. Such therapy related adverse events contribute significantly to patient care costs, as well as to the diminution of quality of life for patients.

The Company’s clinical development program in AAV aims to reduce or eliminate steroids in the standard of care regimen, essentially replacing high dose steroids with the complement inhibitor CCX168. The Phase II CLEAR clinical trial has been conducted in a three step fashion. The Company previously reported data from Steps 1 and 2 of the CLEAR Phase II trial, which provided evidence that steroids could be significantly reduced or even eliminated in AAV patients who were dosed with CCX168, with no diminished therapeutic efficacy compared to that seen with full dose steroid standard of care. Cumulative data from all three steps of the trial is expected in late December or early January.

In addition to AAV, the Company is also exploring the expansion of the use of the complement inhibitor CCX168 in other diseases, particularly in rare and orphan diseases.

Progress in the complement inhibitor CCX168 program includes:

All patients in the AAV CLEAR Phase II trial have completed treatment. Centralized Birmingham Vasculitis Activity Score (BVAS) assessments, which serves as the primary endpoint, and database finalizations are now underway;

Clinical study sites for the CLASSIC Phase II trial in AAV in North America continue to successfully enroll patients; and
Data showing a strong anti-thrombogenic effect of C5a receptor inhibition by CCX168 in aHUS patients’ sera was presented on November 6th at the American Society of Nephrology (ASN) Kidney Week 2015.

Immuno-Oncology: CCX872 is a potent and selective inhibitor of the chemokine receptor known as CCR2 which is being evaluated in patients with non-resectable pancreatic cancer. Pancreatic cancer is the fourth leading cause of cancer death in the US. Even with the most recent advances in the treatment of pancreatic cancer, median overall survival is 6-8 months. As such, more effective and better tolerated treatments are needed in both first-line and second-line therapies. In an ongoing, multi-center clinical trial with CCX872, up to 54 patients with non-resectable pancreatic cancer will be enrolled. The primary efficacy measurement of this study is progression-free survival after at least 24 weeks of treatment.

Enrollment in the Phase Ib clinical trial of CCX872 in patients with non-resectable pancreatic cancer continues to advance; patient enrollment has surpassed 20 percent of target;

At the 2015 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), human pharmacokinetic data from the oral dosing of CCX872 in non-resectable pancreatic patients shows very good CCR2 blockade in the first step of the trial, and supports expectation of greater than 90 percent receptor coverage will be achieved throughout the day in the ongoing trial; and

At the same meeting as noted above, as well as at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, results from the combination therapy of chemokine receptor and check point inhibitors in triple negative breast cancer models were presented. These data suggest that a CCR1 inhibitor combined with an anti-PD-L1 antibody potentiates anti-tumor effects in triple negative breast cancer in vivo.

Chronic Kidney Disease: CCX140 is an inhibitor of the chemokine receptor known as CCR2 (distinct from CCX872 above) and is being developed as an orally-administered therapy for the treatment of diabetic nephropathy (DN), one of the most common forms of chronic kidney disease affecting greater than 10 million individuals in the US alone. The Company previously reported that a Phase II clinical trial in DN patients taking CCX140 achieved its primary endpoint of significantly lowering protein in the urine, an important marker of improved kidney function, over 52 weeks of therapy. CCR2 inhibition therapy represents a therapeutic approach entirely distinct from current standard of care treatment, which comprises common blood pressure medications that slow but do not halt the progression of disease.

Study results from the Phase II trial in patients with diabetic nephropathy treated with CCX140 were presented in an oral presentation at ASN Kidney Week 2015 in a session entitled, "Clinical Trials in CKD: Pursuing a New Horizon"and at the European Association for the Study of Diabetes (EASD) Annual Meeting.

Post treatment follow-up data indicate persistence of therapeutic effect after CCX140 was withdrawn, consistent with a disease modifying effect; and

Phase II clinical trial results of CCX140 in patients with diabetic nephropathy were published in the Lancet Diabetes & Endocrinology illustrating CCX140 to be safe and well tolerated while demonstrating statistically significant improvements in kidney function in patients with diabetic nephropathy.

Anticipated Milestones

Orphan and Rare Diseases:

Report top-line results from AAV CLEAR Phase II trial in Europe with CCX168 in late December or early January 2016;
Complete enrollment in the AAV CLASSIC Phase II trial in North America with CCX168 by the end of 2015, and report top-line data in the second quarter of 2016;
Prepare for End of Phase II meetings with regulatory agencies in 2016 to review CLEAR and CLASSIC Phase II clinical results; plan to initiate a Phase III clinical program with CCX168 in patients with AAV by the end of 2016; and
Report early results from the Phase II proof-of-concept clinical trial of CCX168 in aHUS patients who are on dialysis in the first half of 2016.

Immuno-Oncology:

Advance the Phase Ib pancreatic cancer trial of CCX872 in combination with FOLFIRINOX, and report early overall response data in the first half of 2016 and initial progression free survival in the second half of 2016.

Chronic Kidney Disease:

Continue preparations for an End of Phase II meeting with the FDA including the design of the Phase III clinical development program for CCX140 in diabetic nephropathy, as well as continued business development efforts.

Third Quarter 2015 Financial Results

Research and development expenses were $7.9 million for the three months ended September 30, 2015 compared to $7.5 million reported for the same period in 2014. The increase from 2014 to 2015 was primarily attributable to higher expenses associated with CCX168, our C5aR inhibitor, due to ongoing Phase II clinical trials for the treatment of AAV in Europe, the CLEAR trial, and in North America, the CLASSIC trial, and our Phase II pilot clinical trials in patients with aHUS and IgAN. Further, costs associated with CCX140, our CCR2 inhibitor, in preparation to conduct an end-of-Phase II meeting with the FDA and the ongoing clinical trial with CCX872, our second CCR2 inhibitor, in patients with pancreatic cancer also contributed to the increases. These increases were partially offset by lower expenses associated with CCX507, our second generation CCR9 inhibitor, due to the completion of Phase I clinical development in the third quarter of 2014.

General and administrative expenses were $3.8 million for the three months ended September 30, 2015 compared to $3.5 million for the comparable period in 2014. The increase from 2014 to 2015 was primarily due to increases in intellectual property related expenses, travel expenses and professional service fees, partially offset by lower employment related expenses and legal fees.

Total shares outstanding at September 30, 2015 were approximately 44.1 million shares.

Cash, cash equivalents and investments totaled $85.2 million at September 30, 2015.

Asterias Biotherapeutics Reports Third Quarter Results

On November 9, 2015 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company focused on the emerging field of regenerative medicine, reported financial and operating results for the third quarter ended September 30, 2015 (Press release, BioTime, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110632 [SID:1234508149]).

"Asterias continued to achieve solid progress in the third quarter toward advancing the clinical development of our key therapeutic programs," stated Pedro Lichtinger, President and CEO of Asterias. "We completed recruitment for the initial, low-dose safety cohort of the AST-OPC1 Phase 1/2a clinical trial for spinal cord injury. To date, no serious adverse events have been observed in any of the three treated patients. Following recommendation by the Data Monitoring Committee, we started patient enrollment for the second cohort in which patients will be treated with a higher dose of AST-OPC1 that is in the range that we believe to be efficacious and that has the potential to bring benefit to patients. Importantly, the first patient in the initial, low-dose safety cohort has shown steady neurological improvement at both 2-month and 3-month post-injection assessments. Also during the quarter, we announced a strategic collaboration to advance development of large scale manufacturing processes for AST-VAC2, our allogeneic dendritic cell immunotherapy. This manufacturing agreement will streamline and scale manufacturing processes for AST-VAC2 to support advanced clinical trials, including the planned Phase 1/2a clinical trial in lung cancer, and eventual commercialization of AST-VAC2. We anticipate achieving significant additional clinical milestones during the next 12 months."

Recent Corporate Developments

On November 2, 2015, Asterias announced the appointment of Georgia Erbez as Chief Financial Officer. Ms. Erbez brings to Asterias an impressive track record of achievement and a broad range of financial management and strategic planning experience, especially in the areas of capital resource development and business development.
Research and Development Highlights

AST-VAC1 (antigen-presenting autologous dendritic cells)

Following the presentation of promising Phase 2 results at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting during the second quarter, the Company has continued to advance its planning for further clinical development of AST-VAC1, including holding a clinical advisory panel to design a pivotal trial, and initiating the selection process for a contract manufacturing organization for that trial.

AST-OPC1 (oligodendrocyte progenitor cells)

In August, Asterias concluded recruitment of the initial safety cohort of the SCiStar Phase 1/2a dose-escalation clinical trial of AST-OPC1 (oligodendrocyte progenitor cells) for complete cervical spinal cord injury (SCI), in which three patients were administered a low dose of 2 million AST-OPC1 cells. The results of the study continue to support a robust safety profile for AST-OPC1, with no serious adverse events observed in any of the three treated patients to date.

In October, following review of the 30-day post-injection safety data from the initial safety cohort, the Data Monitoring Committee recommended dose escalation to the second cohort in the SCiStar Phase 1/2a clinical trial. Recruitment for the second cohort has commenced, with a planned enrollment of five patients who will each receive 10 million cells of AST-OPC1. Asterias expects to complete enrollment of the 10 million cell cohort, and to receive safety data, in the first half of 2016. Initial efficacy data readouts from the trial are expected in the second half of 2016. The first patient in the safety cohort, at the 2-month post-injection assessment, had progressed from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS B injury. At 3-months post-injection, this patient demonstrated additional neurological improvement to an AIS C injury.

Asterias announced the publication of preclinical data in Regenerative Medicine that supports the safety and use of AST-OPC1 as a treatment for SCI. The preclinical results showed that AST-OPC1 cells did not cause any adverse clinical observations, toxicities, allodynia or tumors. AST-OPC1 exhibited robust persistence and limited migration within the thoracic and cervical spinal cord. In addition, AST-OPC1 demonstrated nerve growth stimulating properties and remyelinating properties that supported restoration of function in animal models.

In the third quarter, Asterias received $1.1 million from the California Institute of Regenerative Medicine (CIRM) under the previously announced $14.3 million CIRM grant award for clinical development of AST-OPC1. CIRM disburses the grant funds in accordance with a quarterly disbursement schedule, subject to Asterias’ achievement of certain progress and safety milestones.
AST-VAC2 (antigen-presenting allogeneic dendritic cells)

Asterias announced a collaboration with the UK-based Cell Therapy Catapult to advance development of large scale manufacturing processes for AST-VAC2, Asterias’ allogeneic dendritic cell immunotherapy. Under the agreement, the Cell Therapy Catapult will streamline and scale manufacturing processes for AST-VAC2 to support advanced clinical trials and eventual commercialization of AST-VAC2. Asterias has an ongoing partnership with Cancer Research UK to execute the first stage of AST-VAC2 clinical development, under which Cancer Research UK will sponsor and manage a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung carcinoma.

Third Quarter [Unaudited] Financial Results

Total revenues in the third quarter 2015 were $1.4 million, which were primarily comprised of grant income and royalty revenues on product sales by licensees. Total revenues in the comparable quarter in the prior year were $85,000. Operating expenses in the third quarter were $6.2 million, compared to $4.0 million in the prior year period. Research and development (R&D) expenses in the third quarter were $4.6 million, compared to $2.6 million in the comparable quarter of the prior year. General and administrative (G&A) expenses in the third quarter were $1.6 million, compared to $1.5 million in the comparable quarter of the prior year.

Net loss for the third quarter 2015 was $3.5 million, including a deferred income tax benefit of approximately $1.6 million. Net loss in the third quarter 2014 was $1.7 million, including a deferred income tax benefit of approximately $2.3 million. On a per share basis, net loss for the third quarter was $0.09 per share, compared to a loss of $0.05 per share for the comparable quarter of the prior year.

Cash and cash equivalents were $12.6 million as of September 30, 2015, compared to $3.1 million as of December 31, 2014. At September 30, 2015, the Company held 3.9 million BioTime common shares, with a market value of approximately $11.6 million on that date.

For the third quarter, net cash used in operating activities was $3.6 million. The Company continues to expect net cash burn in 2015 to be in the range of $15 million to $17 million.

Bellicum Pharmaceuticals Reports Third Quarter 2015 Financial Results and Recent Program Updates

On November 9, 2015 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the third quarter of 2015 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, NOV 9, 2015, View Source;p=RssLanding&cat=news&id=2110660 [SID:1234508146]).

"During the third quarter we continued to make good progress in the advancement of our stem cell transplant, CAR-T and TCR programs," said Tom Farrell, President and Chief Executive Officer of Bellicum. "We are particularly excited about the interim data that will be presented at ASH (Free ASH Whitepaper) 2015 that underscore the potential of our lead product candidate BPX-501 to improve outcomes for patients with blood cancers and inherited blood diseases undergoing haplo transplantation. BPX-501 could be used in the treatment of over 60 rare diseases, enabling a potential curative transplant for many of those disorders, including sickle cell disease, where treatment-related mortality has severely limited the adoption of allogeneic transplantation."

Added Mr. Farrell, "We are also looking forward to multiple presentations at ASH (Free ASH Whitepaper) highlighting our CAR-T and TCR adoptive cell therapy programs and the power of our cellular control technologies. We continue to anticipate that three new product candidates, BPX-601, BPX-701, and BPX-401 will be initiating clinical development in the first half of 2016."

Program Updates:

BPX-501 at ASH (Free ASH Whitepaper) 2015: Company to present interim data from the BP-004 ongoing Phase 1/2 clinical trial. Bellicum will present initial data in malignant and non-malignant blood diseases at the upcoming 26th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in early December in Orlando, Florida. The open label dose escalation trial in pediatric patients is evaluating whether BPX-501 T cells from a haploidentical donor, administered following a T-depleted hematopoietic stem cell transplant (HSCT), are safe and can enhance immune reconstitution.

Enrollment in the BP-004 trial continues at a strong pace at sites in Europe and in the U.S., with 53 pediatric patients enrolled as of October 31st, including one patient with sickle cell disease.

Among non-malignant patients in the trial treated to date are four children with beta thalassemia major (in its most severe form, the β0/β0 type) who have successfully undergone the HSCT transplant procedure with the add-back of BPX-501 gene-modified T cells. Within two weeks of the transplant procedure all patients became blood transfusion-independent. All four patients are alive, disease-free and remain transfusion-independent.

At a medical symposium in Parma, Italy in early September, principal investigator Dr. Franco Locatelli shared initial outcomes from this ongoing trial. His presentation included the first 15 children enrolled in the clinical study with non-malignant inherited disorders (four with severe combined immunodeficiency, or SCID; three with Wiskott-Aldrich Syndrome; four with Fanconi anemia; three with beta thalassemia, and one with hemophagocytic lymphohistiocytosis) who received the BPX-501 T cell add-back following HLA-partially matched family donor HSCT. In all cases, the BPX-501 T cells engrafted and expanded with no secondary graft failures. Grade II skin-only graft versus host disease (GvHD) was observed in one patient, and promptly resolved with topical steroids. None of the patients so far have developed chronic GvHD, and all are alive and disease-free.

BPX-601 preclinical data to be presented at ASH (Free ASH Whitepaper) 2015. Bellicum continues to advance its first GoCAR-T product candidate, containing our proprietary iMC (inducible MyD88/CD40) activation switch, designed to treat solid tumors expressing prostate stem cell antigen (PSCA). The Company expects to file an IND for the initial indication of pancreatic cancer by the end of 2015. In addition to pancreatic cancer, PSCA is also expressed in prostate, ovarian, bladder, esophageal and gastric cancers. BPX-601 is differentiated from traditional CAR-T therapies with an MC co-stimulatory domain that is activated by administration of rimiducid.
BPX-401 CIDeCAR preclinical data to be highlighted in an oral presentation at ASH (Free ASH Whitepaper) 2015. BPX-401, a CIDeCAR product candidate incorporating Bellicum’s proprietary MC co-stimulatory domain and the CaspaCIDe safety switch, is designed to target blood cancers expressing CD19. BPX-401 is expected to enter the clinic in the first half of 2016.

BPX-701 progressing toward the clinic. Bellicum continues to advance its proprietary T cell receptor (TCR) product candidate designed to target solid tumors expressing the preferentially-expressed antigen in melanoma, or PRAME. The Company has identified clinical sites for its BPX-701 CaspaCIDe-enabled TCR product candidate and expects to file an IND by the end of 2015, initially for the indications of PRAME-expressing sarcomas and uveal melanoma.

Third Quarter and Nine Months Ended September 30, 2015 Financial Results:

Bellicum reported a net loss of $13.4 million for the third quarter of 2015 and $31.7 million for the nine months ended September 30, 2015, compared to a net loss of $4.1 million and $9.7 million for the comparable periods in 2014. The results included non-cash, share-based compensation charges of $2.3 million and $5.9 million for the third quarter and nine months ended September 30, 2015, respectively, and $0.1 million and $0.2 million for the comparable periods in 2014. As of September 30, 2015, cash and investments totaled $163.2 million.

Grant revenues were $0.1 million and $0.2 million for the three and nine months ended September 30, 2015, respectively, and $0.7 million and $1.8 million during the comparable periods in 2014. The decrease in grant revenues was primarily due to the June 2014 expiration of the Company’s grant award from the Cancer Prevention and Research Institute of Texas.

Research and development expenses were $9.8 million and $23.5 million, respectively, for the three and nine months ended September 30, 2015, compared to $2.3 million and $7.9 million during the comparable periods in 2014. The higher expenses in the 2015 periods were primarily due to an increase in manufacturing and clinical expenses as a result of increased patient enrollment in our BPX-501 clinical trials, increased expenses for the IND-enabling activities on the Company’s CAR-T and TCR product candidates and increased personnel and infrastructure costs.

General and administrative expenses were $3.9 million and $8.9 million, respectively, for the three and nine months ended September 30, 2015, compared to $1.3 million and $2.3 million during the comparable periods in 2014. The increased G&A expenses in 2015 were due to the growth of the organization and the costs associated with operating as a public company.

OPKO Announces Third Quarter Financial and Operating Results

On November 9, 2015 OPKO Health, Inc. (NYSE:OPK), a multinational biopharmaceutical and diagnostics company, reported financial and operating results for the three and nine months ended September 30, 2015 (Press release, Opko Health, NOV 9, 2015, View Source [SID:1234508144]).

Business Highlights

Completed the Acquisition of Bio-Reference Laboratories on August 20, 2015: Bio-Reference Laboratories is the third largest full-service clinical laboratory in the United States and is known for its innovative technological solutions and pioneering leadership in the areas of genomics and genetic sequencing. Through GeneDx, Bio-Reference Laboratories’ genetic sequencing laboratory, and GenPath Diagnostics, its Oncology and Women’s Health business units, Bio-Reference Laboratories has accumulated a vast array of genetic and genomic data that OPKO will make available to industry and academic scientists to enhance their drug discovery and clinical trial programs. Since closing, OPKO has begun to leverage the national marketing, sales and distribution resources of Bio-Reference Laboratories to enhance sales of OPKO’s 4Kscore test, a blood test that provides a personalized risk score for aggressive prostate cancer, and plans to further leverage the Bio-Reference capabilities with OPKO’s other diagnostic products under development.

4Kscore Recommended in National Comprehensive Cancer Network Guidelines for Prostate Cancer Early Detection: The National Comprehensive Cancer Network (NCCN) included 4Kscore as a recommended test in their 2015 Guidelines for Prostate Cancer Early Detection. The panel making this recommendation concluded that the 4Kscore, as a blood test with greater specificity over the PSA test, is indicated for use prior to a first prostate biopsy, or after a negative biopsy, to assist patients and physicians in further defining the probability of high-grade cancer.

Rayaldee PDUFA Date is March 29, 2016: In late 2014, OPKO announced successful top-line results from both pivotal Phase 3 trials with Rayaldee. These trials were identical randomized, double-blind, placebo-controlled, multisite studies intended to establish the safety and efficacy of Rayaldee as a new treatment for secondary hyperparathyroidism (SHPT) in patients with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency.

Completed Enrollment in Ongoing Phase 3 Trial in Growth Hormone Deficient Adults: The trial is designed to evaluate the safety and efficacy of hGH-CTP with a primary endpoint of superiority compared with placebo in decreasing fat mass in adults with GHD. The trial is a randomized, double-blind, placebo-controlled, multicenter, global study in adults with GHD. The study is divided into two treatment periods: a 26-week, double-blind, placebo-controlled period, followed by a 26-week, open-label extension period. The study is expected to conclude in the second half of 2016; with positive results, a regulatory submission to the FDA will follow study completion.

IND for Long-Acting Factor VIIa-CTP for Hemophilia Filed and Accepted: In March 2015, the FDA accepted OPKO’s IND application to initiate a Phase 2a trial for its long-acting intravenous coagulation Factor VIIa-CTP to treat hemophilia. Clinical trials are expected to commence during Q4 2015.

Clinical Studies for Long-Acting Oxyntomodulin for Obesity and Diabetes Expected to Begin During 2016: OPKO expects to commence studies for its long-acting subcutaneous oxyntomodulin for diabetes and obesity in Q1 2016.

VARUBITM (Rolapitant) was Approved by the FDA on September 2, 2015 and Commercial Launch is Expected to Commence this Month: OPKO’s partner, Tesaro received FDA approval of oral VARUBI, a neurokinin-1 (NK-1) receptor antagonist, in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic chemotherapy. Tesaro expects to commence commercial sales in the U.S. this month. VARUBI has been included in the NCCN Guidelines as a recommended option in combination with other antiemetic agents for patients receiving both high emetic risk intravenous chemotherapy (HEC) and moderate emetic risk intravenous chemotherapy (MEC). Category 1, the highest level category of evidence and consensus, was granted to VARUBI for both HEC and MEC chemotherapy. Following commercialization, OPKO is eligible to receive up to $110 million in additional milestones and tiered, double-digit royalties.

"OPKO has already achieved numerous important milestones during 2015," said Phillip Frost, M.D., Chairman and CEO. "We believe that the Pfizer transaction for hGH-CTP and the acquisitions of EirGen and Bio-Reference Laboratories have had a positive impact on our financial operations and will provide significant revenue opportunities and an expanded commercial platform for us going forward. The addition of our 4Kscore Test to the NCCN guidelines was an important step toward obtaining reimbursement from healthcare payors, a key factor for obtaining broad access to men for the test. Our NDA filing for Rayaldee continues to advance through the FDA drug approval process and we have high expectations for our new treatment option for patients with stage 3 or 4 chronic kidney disease and secondary hyperparathyroidism. Our clinical development programs for Factor VIIa-CTP and oxyntomodulin, each with great commercial potential, are advancing on plan and we expect to initiate human trials for both products in the near future," continued Dr. Frost.

Financial Highlights

Consolidated revenues increased to $143.0 million from $19.8 million for the three months ended September 30, 2015 compared to three months ended September 30, 2014, and increased to $215.5 million from $65.6 million for the nine months ended September 30, 2015 as compared to the 2014 period. The 2015 periods include revenue from Bio-Reference and EirGen beginning with their acquisitions in August and May 2015, respectively. Revenue for the three and nine months ended September 30, 2015 also includes $17.7 million and $47.8 million, respectively, from OPKO’s collaboration with Pfizer.

Net income for the three months ended September 30, 2015 was $128.2 million compared with net loss of $48.7 for the 2014 period and net losses for the nine months ended September 30, 2015 decreased to $31.6 million compared and $118.7 million for the 2014 period. The 2015 three and nine month periods include significant non-recurring and/or non-cash activities, including:
$93.0 million and $87.2 million of income tax benefit reflecting the release of valuation allowances against all of OPKO’s U.S.-based deferred tax assets as a result of the Bio-Reference acquisition in the three and nine month periods of 2015, respectively;

17.3 million gain related to the deconsolidation of OPKO’s previously consolidated variable interest entity, SciVac, in the three month period of 2015 as SciVac completed an initial public offering by merger with Levon Resources Ltd. in July 2015;

$25.9 million of non-recurring operating expense related to the repayment of a grant to the Office of the Chief Scientist in Israel related to the Pfizer transaction in the nine month period of 2015; and
,
Other income and (expense) of $32.2 million and ($34.1) million related to the change in fair value of derivative instruments in the three and nine months of 2015, respectively, compared with $3.3 million and $3.8 million in the 2014 periods. The change in fair value is principally related to an embedded derivative in our January 2013 convertible senior notes due in 2033.

Cash, cash equivalents and marketable securities were $212.1 million as of September 30, 2015.

This reflects receipt of Pfizer upfront payments of $295.0 million, partially offset by a $94.7 million cash payment for the acquisition of EirGen (net of EirGen’s cash on hand) and a one-time $25.9 million payment to the Office of the Chief Scientist in Israel related to the Pfizer transaction.

TG Therapeutics, Inc. Announces Third Quarter 2015 Financial Results and Business Update

On November 09, 2015 TG Therapeutics, Inc. (NASDAQ:TGTX) reported its financial results for the third quarter ended September 30, 2015 and recent company developments (Press release, TG Therapeutics, NOV 9, 2015, View Source [SID:1234508143]).

Michael S. Weiss, the Company’s Executive Chairman and Interim Chief Executive Officer, stated, "During the third quarter, we achieved another major milestone for the Company in obtaining a Special Protocol Assessment for our UNITY-CLL trial, a study evaluating the safety and efficacy of our proprietary ‘1303′ combination regimen in patients with front-line as well as previously treated CLL. This is a very important and exciting clinical trial for the Company, as it represents our first pivotal trial for our proprietary combination and, if successful, should provide a broad approval in CLL offering patients in both first-line and relapsed/refractory setting, a novel, non-chemotherapy treatment option. Further, it would provide us a broad label for building additional three and, possibly, four drug proprietary combinations to further improve outcomes for patients with CLL. With Phase 3 programs in oncology now underway for both TG-1101 and TGR-1202, we’re excited to begin exploring the potential of our pipeline products for the treatment of autoimmune disease, an area where B-cell targeted therapies have proven highly effective, and anticipate commencing our first trial in Multiple Sclerosis in the near-term." Mr. Weiss continued, "We also remain focused on aggressively enrolling into our ongoing GENUINE Phase 3 clinical trial, and expect top-line data from this study in the second half of 2016. Finally, from a financial perspective, with more than $115 million in cash and investments we have enough cash to execute on our business plan."

Recent Developments and Highlights

In September 2015, we announced a Special Protocol Assessment (SPA) agreement with the FDA for the first Phase 3 clinical trial of our proprietary combination regimen of TG-1101 (ublituximab) with TGR-1202 ("1303") for patients with chronic lymphocytic leukemia, the UNITY-CLL study.
In September 2015, we announced the initiation of a Phase 1/2 clinical trial investigating the use of TG-1101 and TGR-1202 in combination with pembrolizumab, the anti-PD-1 immune checkpoint inhibitor, in patients with relapsed or refractory CLL, the first clinical trial evaluating the safety, tolerability and effectiveness of the triple combination of a PI3K delta inhibitor with an anti-CD20 mAb and an anti-PD-1 checkpoint inhibitor.

Goals/Objectives for the Remainder of 2015

Continue to aggressively recruit into the GENUINE Phase 3 Clinical Trial of TG-1101 in combination with ibrutinib
Enroll the first patient by year end in our UNITY-CLL Phase 3 clinical trial of TG-1101 plus TGR-1202 in front-line and relapsed/refractory CLL
Announce our next registration trial evaluating 1303 in patients with NHL
Continue to recruit into the triple combination cohort of 1303 plus ibrutinib as well as the triple combination study of 1303 plus pembrolizumab, as well as seek to evaluate additional novel triple combinations of interest
Expand into autoimmune diseases with the first Phase 2 trial in Multiple Sclerosis to commence in the near-term
Continue to advance our pre-clinical compounds, including IRAK4, anti PD-L1 and anti-GITR forward towards clinical development
Continue to seek additional compounds to further complement our current portfolio

Financial Results for the Third Quarter 2015

At September 30, 2015 the Company had cash, cash equivalents, investment securities, and interest receivable of $115.4 million, which includes approximately $67.0 million of net proceeds from the utilization of the Company’s at-the-market ("ATM") sales facility during the year (all of which was previously disclosed in connection with our last quarterly update), as compared to December 31, 2014 when we had $78.9 million.

Our consolidated net loss for the third quarter ended September 30, 2015, excluding non-cash items, was approximately $12.4 million, which included approximately $6.9 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the third quarter ended September 30, 2015, inclusive of non-cash items, was $13.7 million, or $0.28 per diluted share, compared to a consolidated net loss of $17.5 during the comparable quarter in 2014, representing a decrease in consolidated net loss of $3.8 million. The decrease in consolidated net loss during the third quarter ended September 30, 2015 was primarily the result of $8.1 million of expense ($4.1 million of which was non-cash stock expense) recorded during the quarter ended September 30, 2014 in conjunction with the Company’s licensing agreement for TGR-1202, and a $2.9 million decrease in non-cash compensation expense related to equity incentive grants over the comparable period in 2014. Partially offsetting the aforementioned decreases, other research and development expenses for TG-1101 and TGR-1202 increased $4.8 million and $2.1 million, respectively, over the comparable period in 2014.

Our consolidated net loss for the nine months ended September 30, 2015, excluding non-cash items, was approximately $32.5 million, which included approximately $16.0 million of manufacturing and CMC expenses in preparation for Phase 3 clinical trials and potential commercialization. The consolidated net loss for the nine months ended September 30, 2015, inclusive of non-cash items, was $45.3 million, or $1.01 per diluted share, compared to a consolidated net loss of $37.0 million during the comparable period in 2014, representing an increase in consolidated net loss of $8.3 million. The increase in consolidated net loss during the nine months ended September 30, 2015 was primarily the result of other research and development expenses for TG-1101 and TGR-1202 increasing approximately $14.6 million and $4.5 million, respectively, over the comparable period in 2014. This was offset by $9.3 million of expense ($5.3 million of which was non-cash stock expense) recorded in conjunction with the Company’s licensing agreements for TGR-1202 and the IRAK4 inhibitors program during the nine months ended September 30, 2014, and a decrease of $3.2 million in non-cash compensation expense related to equity incentive grants over the comparable period in 2014.