On March 16, 2017 Omeros Corporation (NASDAQ: OMER), a biopharmaceutical company committed to discovering, developing and commercializing both small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, coagulopathies and disorders of the central nervous system, reported recent highlights and developments as well as financial results for the fourth quarter and year ended December 31, 2016 (Press release, Omeros, MAR 16, 2017, View Source [SID1234518173]).

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4Q 2016 total and OMIDRIA revenues were $12.9 million. Revenues from OMIDRIA sales rose 94% from the prior year’s fourth quarter and 14% from 3Q 2016. OMIDRIA units sold in the fourth quarter increased by 22% over the third quarter.
Total year 2016 revenues were $41.6 million, a 208% increase over 2015, the year in which OMIDRIA was launched.
Net loss in 4Q 2016 was $19.6 million, or $0.45 per share, and, for the full year of 2016, was $66.7 million, or $1.65 per share. Both periods included a $5.6 million ($0.13 per share in 4Q) charge for early extinguishment of previously existing debt. Non-cash expenses for 4Q and the full year of 2016 were $5.2 million, or $0.12 per share, and $16.1 million, or $0.40 per share, respectively.
Successful outcome of post-marketing clinical trial of OMIDRIA in pediatric patients undergoing cataract surgery, which is expected to result in an additional six months of regulatory exclusivity.
Opened enrollment in Phase 3 clinical trial of OMS721 in patients with atypical hemolytic uremic syndrome (aHUS).
Positive data from Phase 2 clinical trials of OMS721 in both renal disorders and hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).
"2016 was a year of significant achievements across a wide range of our programs," said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "OMIDRIA sales grew more than two hundred percent over the previous year and our pipeline made equally impressive progress. Our Phase 3 trial in aHUS with our MASP-2 inhibitor OMS721 is underway, and positive OMS721 Phase 2 data in both IgA nephropathy and HSCT-TMA have set the stage for additional Phase 3 registration trials this year. MASP-3, targeted by our OMS906 program, was shown to be the key activator of the alternative pathway. Both OMS906 and our PDE7 inhibitor OMS527 are advancing quickly toward the clinic, and our GPCR program continues to generate exciting data over broad therapeutic areas, including immuno-oncology. We are pleased with Omeros’ accomplishments in 2016, and we expect that 2017 will build on those successes."

Fourth Quarter and Recent Highlights and Developments

Omeros reported in November 2016 the successful outcome of its recently completed post-marketing clinical trial of the effect of OMIDRIA in pediatric patients undergoing cataract surgery. In the trial, OMIDRIA was well tolerated with adverse event rates consistent with those seen in pediatric cataract surgery and with the control group. The company plans to submit a supplemental NDA this year, requesting expanded label language to cover patients of any age. Omeros expects that this submission will result in an additional six months of regulatory exclusivity for OMIDRIA.
Highlights and developments regarding OMS721, Omeros’ lead human monoclonal antibody in its mannan-binding lectin-associated serine protease-2 (MASP-2) program for the treatment of thrombotic microangiopathies (TMAs), including aHUS and HSCT-TMA, and for the treatment of complement-related renal diseases, include:
Enrollment has opened in Omeros’ Phase 3 clinical trial in patients with aHUS.
Omeros reported positive data (p = 0.017) from its Phase 2 clinical trial of OMS721 for the treatment of renal disorders, including IgA nephropathy and membranous nephropathy, in October 2016. In this trial, OMS721 significantly improved key endpoints of renal function, and patients achieved partial remission with only 12 weeks of dosing. Also in October 2016, Omeros announced positive results in patients with HSCT-TMA from the company’s Phase 2 clinical trial of OMS721 in TMAs, with clinically meaningful improvement in measures of red blood cell destruction, specifically lactate dehydrogenase (LDH) and haptoglobin levels (p < 0.01 and p < 0.06, respectively).
Following discussion with the FDA, the company plans to pursue breakthrough therapy designation for OMS721 in IgA nephropathy and in HSCT-TMA. Omeros also has been pursuing accelerated approval for OMS721 in both of these indications as well as in aHUS. The FDA has granted fast track designation for OMS721 in patients with aHUS and orphan designation for OMS721 in patients with TMAs, including aHUS and HSCT-TMA.
Highlights and developments regarding OMS906, Omeros’ lead antibody targeting mannan-binding lectin-associated serine protease-3 (MASP-3), a protein essential for the activation of the alternative pathway of complement (APC), include:
In November 2016, the company announced data from the evaluation of OMS906 in non-human primates. Single-dose administration of OMS906 to cynomolgus monkeys resulted in sustained ablation of systemic APC activity for approximately 16 days. The APC is involved in a wide range of diseases, including paroxysmal nocturnal hemoglobinuria (PNH), aHUS, age-related macular degeneration, arthritis, asthma and traumatic brain injury. No safety concerns were identified.
Omeros is finalizing selection of lead and back-up molecules and preparing to initiate scale-up for clinical trials. The company is evaluating PNH as the first clinical indication for OMS906.
In October 2016, Omeros entered into a $125.0 million senior secured credit facility with CRG Servicing LLC, under which the company borrowed $80.0 million in November 2016. The credit facility has a six-year term with four years (through December 31, 2020) of interest-only payments after which quarterly principal and interest payments will be due through the September 30, 2022 maturity date; in addition, there is the potential to extend the interest-only period through maturity if certain milestones are satisfied. The company may borrow additional tranches of up to $25.0 million and $20.0 million, subject to the satisfaction of certain milestones on or before June 30, 2017 and December 31, 2017, respectively.
Financial Results

Fourth Quarter 2016

For the quarter ended December 31, 2016, total revenues were $12.9 million, all relating to sales of OMIDRIA. This compares to OMIDRIA revenues of $6.7 million for the same period in 2015. On a sequential quarter-over-quarter basis, OMIDRIA revenue grew $1.6 million, or 14%. The increase in units sold from 3Q to 4Q 2016 was 22%. The quarter-over-quarter increases in OMIDRIA revenue and units sold are due to continued acceptance of and increased demand for OMIDRIA in the ophthalmic surgery community.

Total operating costs and expenses for the three months ended December 31, 2016 were $24.8 million compared to $24.7 million for the same period in 2015. The change in the current year quarter was primarily due to increased legal costs associated with the company’s patent infringement lawsuit against Par Pharmaceutical and additional headcount-related costs, partially offset by a decrease in research and development costs due to the timing of clinical and manufacturing activities.

In November 2016, the company incurred a $5.6 million loss ($0.13 per share) on early extinguishment of debt associated with the initiation of a new secured credit facility and the prepayment of Omeros’ prior secured credit facility.

For the three months ended December 31, 2016, Omeros reported a net loss of $19.6 million, or $0.45 per share, which included noncash expenses of $5.2 million ($0.12 per share). This compares to the prior year’s fourth quarter when Omeros reported a net loss of $19.8 million, or $0.52 per share, which included non-cash expenses of $2.8 million ($0.07 per share).

At December 31, 2016, the company had cash, cash equivalents and short-term investments of $45.3 million. In addition, the company had $5.8 million of restricted cash on hand to satisfy its credit facility covenant and lease obligations.

Full Year 2016

Revenues for the full year 2016 were $41.6 million, compared to $13.5 million for the full year 2015.

Total operating costs and expenses for the year ended December 31, 2016 were $95.9 million, an increase of $11.1 million compared to 2015. The 2016 increase related primarily to the Par lawsuit and to increased employee costs, including stock compensation costs.

For the full year 2016, Omeros reported a net loss of $66.7 million, or $1.65 per share, including non-cash expenses of $16.1 million, or $0.40 per share. This compares to a net loss of $75.1 million, or $2.00 per share in 2015, including non-cash expenses of $11.0 million, or $0.29 per share.

On March 15, 2017 Cellectar Biosciences, Inc. (Nasdaq: CLRB), (the "company"), an oncology-focused, clinical stage biotechnology company, reported financial results for the year ending December 31, 2016 (Filing, 8-K, Cellectar Biosciences, MAR 16, 2017, View Source [SID1234518164]). Management will host a teleconference and live webcast to review these financial results, followed by a review of corporate performance and 2017 objectives at 6:00 PM EDT today.

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Summary of Q1 2017 Accomplishments:

· Positive safety, tolerability and activity data through Cohort 3 of Phase I study of CLR 131 in multiple myeloma

· Initiation of fourth cohort of Phase I study of CLR 131 in multiple myeloma

· Additional IP protection for CLR 131 in solid tumors

· Japanese Composition of Matter Patent for CLR 1501 and CLR 1502

· Consolidation of IP portfolio for CLR 131 in multiple myeloma following license agreement with Wisconsin Alumni Research Foundation

Summary of 2016 Financial Results:

Research and development expenses for 2016 were $4.8 million, a reduction of $0.4 million from 2015. This reflects the company’s continued focus on its therapeutic lead product candidate, CLR 131 and the implementation of operating improvements, which have reduced its cost structure.

General and administrative expenses for the year totaled $4.7 million, compared to $3.4 million in 2015. The increase was largely a result of increased consulting and personnel costs, of which approximately $0.5 million are not expected to recur.

The operating loss was $9.4 million for 2016, compared to $8.8 million in 2015. Other income was $3.3 million for fiscal 2016 and 2015, and is primarily due to changes in the valuation of certain warrants that are classified as liabilities on the company’s balance sheet. These amounts are almost exclusively non-cash in nature. As a result, the company’s net loss for the year ended December 31, 2016 was $6.2 million, or $1.36 per share, compared to a 2015 net loss of $5.5 million, or $7.03 per share.




As of December 31, 2016, the company had $11.4 million in cash and cash equivalents on hand, compared to $3.9 million in cash and cash equivalents at December 31, 2015. Management anticipates its available cash and cash equivalents will fund its planned operations into the first quarter of 2018, and believes additional capital will be required to complete its research and development plans.

"Over the last year Cellectar Biosciences, has made tremendous progress in the development of our lead product candidate, CLR 131, which is in the fourth cohort of a Phase I trial for multiple myeloma, and will enter an NCI-supported Phase II study in hematological malignancies in the first quarter of this year," said Jim Caruso, president and CEO of Cellectar Biosciences. "The numerous additions to our intellectual property portfolio provide further protection for our product assets and we are encouraged by the progress with our conjugate program and Pierre Fabre partnership. We look forward to reviewing our 2016 successes and 2017 plans on today’s conference call."

On March 16, 2017 Celsion Corporation (NASDAQ:CLSN), an oncology drug development company, reported financial results for the year ended December 31, 2016 and provided an update on its development programs for ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin and GEN-1, an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein (Press release, Celsion, MAR 16, 2017, View Source [SID1234518159]). The Company’s lead program is ThermoDox which is currently in Phase III development for the treatment of primary liver cancer and in Phase II development for the treatment of recurrent chest wall breast cancer. The Company’s immunotherapy program consists of GEN-1 and is currently in Phase I development for the localized treatment of ovarian cancer.

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"Celsion had an exceedingly productive 2016 as our accomplishments, one after another, established meaningful progress with respect to our two leading-edge technology platforms designed to enhance clinically powerful therapies. With sound fundamentals, the superb execution of our ongoing global, pivotal Phase III OPTIMA Study in primary liver cancer, continues to attract interest and support from the medical community, international regulatory agencies, and research organizations like the National Institutes of Health for this ground-breaking study," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "Our efforts in immuno-oncology are equally important. Over the past year, we have demonstrated the potential of our GEN-1 program to be an effective adjuvant, in both first and second-line ovarian cancer. Recruiting the immune system to work in combination with the standard of care in this patient population has been the goal of medical researchers worldwide. With GEN-1, we believe there is the potential for a break-through and we look forward to reporting comprehensive clinical findings and translational data from our nearly complete OVATION Study in the first half of 2017."

Recent Developments
ThermoDox
Announced the Independent NIH Analysis Showing Treatment with ThermoDox Plus RFA may Significantly Improve Overall Survival of Patients with Primary Liver Cancer. In November 2016, the Company announced the presentation of results from an independent retrospective analysis conducted by the National Institutes of Health (NIH) on the intent-to-treat population of the Company’s HEAT Study during The Interventional Oncology Series: Hepatocellular Carcinoma and Cholangiocarcinoma at the 102nd Scientific Assembly and Annual Meeting of the Radiological Society of North America (RSNA). The NIH analysis, which sought to evaluate the correlation between RFA burn time per tumor volume (min/ml) and clinical outcome, concluded that increased burn time per tumor volume significantly improved overall survival (OS) in patients with solitary lesions treated with RFA + ThermoDox compared to patients treated with RFA alone. More specifically, the analysis showed that a one unit increase in RFA duration per tumor volume improved OS by 20% in patients treated with optimized RFA + ThermoDox compared to RFA alone.These findings are consistent with Celsion’s analysis of the HEAT Study data showing that in patients treated with RFA for more than 45 minutes, standardized RFA plus ThermoDox resulted in a statistically significant improvement in overall survival of over two years when compared to standardized RFA alone.

Announced Support for the OPTIMA Study from the China FDA and Vietnam Ministry of Health. The Company discussed ThermoDox and the OPTIMA Study with regulatory agencies in two key markets, China and Vietnam. The Company met with the China Food and Drug Administration (CFDA) to review the ongoing Phase III OPTIMA Study and regulatory pathway for ThermoDox in China. CFDA was presented with the final overall survival data from the Chinese patient cohort of the HEAT study, which demonstrated a survival benefit in patients treated with ThermoDox plus optimized RFA versus optimized RFA alone. The CFDA informed the Company that if the ongoing Phase III OPTIMA Study is successful, the trial could serve as the basis for a direct regulatory filing in China without the need to file for prior approval in the U.S. or European Union which is currently required for foreign company application. This would allow the Company to accelerate its plans for a regulatory filing in China and, if approved, provide for a significantly earlier launch date in China than originally expected. In addition, the Company’s management team met with the Ministry of Health in Vietnam and based on that meeting, it will move forward with launching additional trial sites for the OPTIMA Study in that country. The Company expects to have approximately 5 additional clinical trial sites in Vietnam activated in early 2017. Vietnam represents a significant market for
ThermoDox where HCC incidence rates are among the highest in the world.

Announced the Issuance of Two New Patents for ThermoDox. In January 2017, the Company announced the issuance of two patents which are directly applicable to the method of treating cancer using our current ThermoDox formulation. These new patents further strengthen the Company’s global patent portfolio around novel heat-sensitive liposome engineered to address a broad range of difficult-to-treat cancers.

Announced Collaboration with the Children’s Research Institute to Evaluate the Use of ThermoDox and High Intensity Focused Ultrasound in the Treatment of Solid Tumors in Children and Young Adults. In October 2016, the Company announced a collaboration with the Children’s Research Institute to conduct a clinical study of ThermoDox in combination with magnetic resonance-guided high intensity focused ultrasound to treat relapsed or refractory solid tumors in children and young adults. This investigator-sponsored Phase I clinical study is being partially funded by the National Institutes of Health and commenced in the fourth quarter of 2016.
GEN-1 Immunotherapy

Announced Continuing Positive Data from the OVATION Study in Newly Diagnosed Advanced Ovarian Cancer Patients. In January 2017, the Company announced data from the first four cohorts of patients in its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1 with the standard of care for the treatment of newly-diagnosed patients with advanced ovarian cancer who will undergo neoadjuvant chemotherapy followed by interval debulking surgery. In the first twelve patients dosed in the OVATION Study, GEN-1 plus standard chemotherapy produced impressive results, with no dose limiting toxicities and highly promising efficacy signals in this difficult to treat cancer. The efficacy data included highly encouraging tumor response rates – 100% disease control rate (DCR) and 75% objective response rate (ORR), successful surgical resections of the eligible patients’ tumors, impressive pathological responses and dramatic, clinically meaningful drops in CA-125 protein levels.

Announced the Issuance of Two New U.S. Patents for GEN-1 Immuno-Oncology Product. In November 2016, the Company announced the issuance of two patents which expand the use of GEN-1 into additional cancer treatment modalities in combination with other chemotherapeutics and extends previous patent claims on the making of and composition of formulations consisting of our PPC delivery polymer and nucleic acids. These new patents further strengthen coverage of GEN-1 for the localized treatment of ovarian cancer and glioblastoma multiforme (GBM), which is already covered by a composition of matter patent in the United States.

Corporate Development
Raised $6.8 Million Through Two Equity Offerings in December 2016 and February 2017. The Company completed two equity offerings of shares of common stock, or pre-funded warrants in lieu thereof, to purchase common stock with institutional healthcare and retail investors totaling $6.8 million in gross proceeds.

Financial Results
For the year ended December 31, 2016, Celsion reported a net loss of $22.1 million, or $0.85 per share, compared to a net loss of $22.5 million, or $1.03 per share, in 2015. Operating expenses were $21.1 million in 2016 compared to $21.3 million in 2015. This decrease was primarily due to lower general and administrative expenses and clinical supply costs offset by higher clinical development costs for the Phase III OPTIMA Study.

Research and development (R&D) costs were constant at $14.6 million in 2016 and 2015. Clinical development costs for the OPTIMA Study were $5.6 million in 2016 compared to $3.6 million in 2015 due to higher patient enrollment, investigator grants and site initiation expenses in the trial. R&D costs for other development programs were lower as a result of the Company’s tighter clinical development focus around the pivotal Phase III OPTIMA Study for the treatment of primary liver cancer and the clinical development program for GEN-1 IL-12 immunotherapy for the localized treatment of ovarian cancer coupled with lower costs in 2016 associated with the production of ThermoDox to support the OPTIMA Study. General and administrative expenses decreased $0.2 million, from $6.7 million in 2015 to $6.5 million in the current year. This decrease in general and administrative expenses in 2016 is primarily the result of reductions in personnel costs and professional fees offset by $0.6 million of non-cash amortization expense related to the covenant not to compete from the June 2014 EGEN acquisition.

Other expenses included a non-cash charge of $1.4 million related to the impairment of in process research and development for the Company’s RNA delivery system (TheraSilence) offset by a $0.7 million reduction in the earn-out liability related to potential milestone payments for the TheraSilence asset. Interest expense decreased by $0.7 million in 2016 due to lower principal balances outstanding under the Company’s current debt facility.

Net cash used in operations was $18.4 million in 2016 compared to $20.8 million in the prior year. The Company ended 2016 with $4.5 million of total cash, investments and accrued interest on these investments. In February 2017, the Company raised an additional $5 million in gross proceeds under a secondary public offering.

On March 16, 2017 Calithera Biosciences, Inc. (Nasdaq:CALA), a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, reported its financial results for the fourth quarter and year ended December 31, 2016 (Press release, Calithera Biosciences, MAR 16, 2017, View Source [SID1234518158]). As of December 31, 2016, cash, cash equivalents and investments totaled $51.8 million. Subsequent to the end of the year, Calithera received an upfront payment of $45.0 million from its global collaboration and license agreement with Incyte Corporation and gross proceeds of $46.0 million from the sale of common stock to Incyte and through its at-the-market program with Cowen and Company LLC.

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"2016 was a transformative year for Calithera as our lead product candidate CB-839 entered into multiple novel combination trials and progressed towards Phase II, and CB-1158 advanced into clinical development leading to a partnership with Incyte Pharmaceuticals for a strategic development and commercialization collaboration in January 2017," said Susan Molineaux, PhD, President and Chief Executive Officer of Calithera. "Looking forward to 2017, we expect to highlight new clinical data from each of our clinical programs at scientific meetings, with clinical data expected from CB-1158 in the first half of the year, and multiple clinical updates on CB-839 combination trials in the second half of 2017. This includes the first clinical data presentation of CB-839 dosed in combination with Bristol Myers Squibb’s Opdivo."

Fourth Quarter 2016 and Recent Highlights

CB-1158: Global Research, Development and Commercialization Collaboration with Incyte. In January 2017, Incyte and Calithera announced the global collaboration and license agreement for the research, development and commercialization of the first in class, small molecule arginase inhibitor CB-1158. Under the terms of the collaboration and license agreement, Calithera has received an up-front payment of $45 million from Incyte in addition to an $8 million equity investment. CB-1158 entered clinical trials in September 2016, and pharmacodynamic data on the first three patients was presented at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) meeting in November 2016.

CB-839: Phase I Triple Negative Breast Cancer Combination Data at the 2016 San Antonio Breast Cancer Symposium (SABCS). New data was presented at the 2016 SABCS in December 2016 on 28 triple negative breast cancer patients treated with CB-839 in combination with paclitaxel; 23 patients were evaluable for efficacy. Among evaluable patients treated with CB-839 doses of at least 600 mg bid (n=16), there were 5 partial responses (31%) and disease control (response or stable disease) in 11 patients (69%). In addition, the combination overcame resistance to paclitaxel in heavily pretreated TNBC patients. There was a 38% response rate and 50% disease control rate in patients who received prior taxanes in the metastatic setting. There was a 50% response rate among taxane-refractory African American patients.

CB-839: Phase I Renal Cell Carcinoma Combination Data at the 28th EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium on Molecular Targets and Cancer Therapeutics. In a plenary session at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Symposium in November 2016, new data was presented with CB-839 in combination with everolimus. Fifteen renal cell carcinoma patients were treated and evaluable for response, including 12 clear cell patients, and three papillary patients. Ninety-three percent (93%) had disease control; one patient had a partial response, one patient had progressive disease, and 13 patients had stable disease. The median progression free survival was 8.5 months and for the majority of patients, their time on therapy was longer than their time on treatment in their prior therapy. In the clear cell patient population the disease control rate was 100%.

At-the-Market Program. In 2017, Calithera received approximately $38.0 million in gross proceeds, $36.9 million in net proceeds, from the sale of common stock pursuant to its at-the-market offering program with Cowen.

Key Management Appointments. Curtis Hecht was named Senior Vice President of Business and Corporate Development, Frank Parlati was named Vice President of Research, and Jennifer McNealey was named Vice President of Investor Relations and Strategy.
Selected Fourth Quarter and Year-end 2016 Financial Results

Cash, cash equivalents and investments totaled $51.8 million at December 31, 2016, compared with $71.9 million at December 31, 2015. Subsequent to the end of the year, Calithera received a $45.0 million upfront payment from Incyte and an additional $44.8 million in net proceeds from sales of common stock in 2017.

Research and development expenses for the full year 2016 were $27.7 million, compared with $23.7 million in the prior year. The increase of $4.0 million was due to an increase of $2.3 million due to higher employment related expenses and an increase of $1.7 million primarily related to the advancement of CB-1158 to a phase 1 clinical trial. Research and development expenses for the fourth quarter of 2016 were $6.6 million, compared to $5.8 million for the same period last year.

General and administrative expenses for the full year 2016 were $10.6 million, compared with $9.1 million in the prior year. The increase of $1.5 million in 2016 was primarily due to higher employment related expenses, including stock based compensation expense. General and administrative expenses for the fourth quarter of 2016 were $3.0 million, compared to $2.3 million for the same period last year. Loss from operations for the three months and year ended December 31, 2016 was $9.5 million and $38.0 million, respectively.

Financial Guidance for 2017

Calithera expects that its cash, cash equivalents and investments will be between $95 and $105 million at the end of 2017, exclusive of any funds arising from new collaborations or partnerships, milestone payments, additional equity financings or other new sources.

On March 16, 2017 BioTime, Inc. (NYSE MKT and TASE: BTX), a clinical-stage biotechnology company developing and commercializing products addressing degenerative diseases, reported financial results for the fourth quarter and year ended December 31, 2016 (Press release, BioTime, MAR 16, 2017, View Source [SID1234518157]).

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"I’m happy with the tremendous progress we made last year in all three of our strategic objectives, clinical development, simplification and unlocking value of non-core programs," said Adi Mohanty, Co-Chief Executive Officer. "We still have more to do, and are well on our way to transforming the company in 2017."

"This year is shaping up to be the year of data with top line data from our registrational trial for Renevia in Europe planned for mid-year, data from OpRegen in Dry AMD to be reported in early May at ARVO and then again in the second half of the year, final validation data from our affiliate OncoCyte on its lung cancer diagnostic leading to a commercial launch in the second half of the year and additional data on OPC-1 for spinal cord injury from our affiliate Asterias and several other data reports from our other pipeline products. These data events and our planned actions to simplify the company while unlocking value will lead to continued positive transformation of BioTime," concluded Mr. Mohanty.

Highlights

Clinical Progress

Renevia (adipose cells + cell delivery matrix)

Positive early Renevia data presented at the International Federation for Adipose Therapeutics and Science meeting (IFATS) meeting in November. The data related to the treatment of the initial 9 run-in patients for BioTime’s ongoing pivotal clinical trial in Europe assessing the efficacy of Renevia for the treatment of HIV-associated lipoatrophy. In December, achieved the recruitment milestone of 50 patients enrolled in the trial. Data from the run-in portion of the study (N=9) indicated that adipose progenitor cells (fat cells), obtained from a liposuction aspirate, remained viable and were observed to proliferate when combined with Renevia hydrogel. Analysis suggests that the grafts retain volume over the assessment period, and the treating physician observed incremental volume was retained in patients who had progressed to the one-year follow-up evaluation. In addition, there were encouraging signs of new tissue regeneration observed. No serious adverse events were noted during the run-in portion of the study.
Additional positive data from the pivotal trial would allow the Company to launch a commercial product in about a year and provide support for future studies of Renevia in certain broader indications of fat tissue deficits in various medical aesthetics applications, such as age-related and trauma-related facial fat loss.
Top-line clinical trial results are expected to be read out mid-2017. If the data are positive, the Company plans to submit an application for CE Mark approval in Europe at the end of 2017.
OpRegen (retinal pigment epithelial cells)

Presented positive early OpRegen data at the International Symposium on Ocular Pharmacology and Therapeutics (ISOPT) in December. The data from the first cohort in the Phase I/IIa clinical trial of OpRegen in the advanced form of dry age-related macular degeneration (dry-AMD). The data suggest that OpRegen is safe and well tolerated. Imaging data from a patient who completed one-year of post-treatment clinical assessment suggests that the graft can survive for at least 12 months.
Enrollment in the second cohort, in which patients are receiving a higher and more clinically meaningful 200,000 cell dose started in 2016. The Company intends to approach the DSMB in the second quarter of this year for approval to begin administering the next higher 500,000 cell dose to the third cohort, and if approved, also begin the fourth cohort before year end.
An abstract with data from the first and second cohorts in the ongoing trial has been accepted for poster presentation at the annual meeting for the Association for Research in Vision and Ophthalmology (ARVO), which will take place in Baltimore, MD, May 7- 11.
The Company is expanding the trial to U.S. sites and recently announced that two of the top retinal surgeons will be enrolling patients at their centers of excellence.
AST-OPC1 (oligodendrocyte progenitor cells)

In November, BioTime’s affiliate, Asterias (NYSE MKT: AST) reported the successful administration of the highest dose of 20 million cells of AST-OPC1 to a patient with complete cervical spinal cord injury (SCI) as part of the SCiStar Phase I/IIa clinical trial. In January 2017, they announced positive efficacy results that showed additional motor function improvement at 6-months and 9-months following administration of 10 million AST-OPC1 cells in 6 AIS-A patients with complete cervical SCIAsterias plans to initiate discussions with the FDA in mid-2017 to determine the most appropriate clinical and regulatory path forward for AST-OPC1.
Liquid Biopsy (lung cancer confirmatory test)

In early March, BioTime’s affiliate, OncoCyte (NYSE MKT: OCX) reported successful completion of a critical step in the development of its lung cancer diagnostic test by locking the prediction algorithm. The cancer diagnostic, has been selected for presentation in a poster discussion session at the 2017 American Thoracic Society (ATS) International Conference that will take place May 19-24 in Washington, D.C.
The lung cancer diagnostic test targets a multi-billion dollar market opportunity. On March 6, 2017, OncoCyte announced the successful completion of the study and that it has locked the prediction algorithm of the test. Based on the study results, OncoCyte announced that it will begin ramping-up its commercial capabilities in anticipation of the potential commercial launch of the test. OncoCyte will initiate a clinical validation phase for the diagnostic. During this phase, OncoCyte will also continue to carry out analytical validation studies to refine its operational stage laboratory processes, and will apply for certification of its CLIA diagnostic testing lab. Upon CLIA certification, OncoCyte will conduct a small CLIA lab validation study to demonstrate that the full assay system utilized in the CLIA lab provides the same results on clinical samples as those obtained in the R&D lab. OncoCyte will then begin a clinical validation study on a new set of at least 300 blinded prospectively collected blood samples to confirm whether the sensitivity and specificity of the test remain within commercial parameters in a CLIA operational setting. Assuming successful completion of these steps, OncoCyte anticipates launching the test commercially in the second half of 2017.
Simplification and Unlocking Value

Subsidiary Deconsolidation

In February, OncoCyte financials were deconsolidated from BioTime’s consolidated financial statements. This will be reflected in BioTime’s future quarterly and annual consolidated financial statements, beginning February 18, 2017.
Business Development

In February, BioTime announced that it expanded its ophthalmology portfolio with the acquisition of a world-wide license to ophthalmology-related intellectual (IP) property assets from University of Pittsburgh. The technology was developed in part in collaboration with BioTime scientists. The IP includes composition and methodologies to develop 3-D retinal tissue constructs from pluripotent cells for their implantation in patients with advanced stages of retinal degeneration.
BioTime continues to leverage and develop its delivery technology platforms. The Company’s Hystem technology is being used to deliver Renevia. ReGylde is in preclinical development as a device for viscosupplementation and a combination product for drug delivery in osteoarthritis. Delivery is a third area of focus for the Company, in addition to aesthetics and ophthalmology.
Management

BioTime expanded its senior management team with the appointments of Jim Knight as Senior Vice President of Corporate Development, in October, and Stephana Patton, Ph.D., J.D., as General Counsel, in February. These industry veterans further strengthen the BioTime management team, complementing other management team additions, such as Oscar Cuzzani, M.D., Ph.D., Vice President of Clinical Development. Dr. Cuzzani joined in February 2016.
Operations

In January, the Company announced the opening of a new, state-of-the art, cGMP manufacturing facility in Jerusalem Bio Park on the campus of Hadassah University Hospital in Jerusalem. This facility has the capabilities to produce multiple cell therapy products.
Financial

In February, the Company successfully completed a public equity offering raising net proceeds of approximately $18.7 million. The raise was completed on attractive terms involving modest discounts and no warrants. It followed a similarly successful raise in June of 2016.
Cash Position and Marketable Securities:

Cash and cash equivalents totaled $22.1 million as of December 31, 2016, compared to $42.2 million as of December 31, 2015, which included Asterias’ cash and cash equivalents of $11.2 million. Based on the March 15, 2017 closing prices of Asterias and OncoCyte common stock owned by BioTime, the combined market value of these securities was $152 million on that date.

Revenues: Total revenues were $1.1 million for the fourth quarter, compared to $1.5 million in the fourth quarter of 2015. Asterias’ total revenues included in the fourth quarter of 2015 were $0.6 million as compared to none in 2016 due to the deconsolidation in May 2016. Total revenues for 2016 were $5.9 million compared to $7.0 million in 2015, which included revenues of Asterias of $2.4 million and $3.6 million, respectively. BioTime’s operating revenues are currently generated primarily from research grants, advertising from the marketing of online database products, sales of research products and royalties and licensing fees.

R&D Expenses: Research and development expenses were $7.0 million for the fourth quarter, compared to $12.8 million for the comparable period in 2015. For 2016, research and development expenses were $36.1 million compared to $42.6 million in 2015. The year over year decrease in research and development expenses of $6.5 million was primarily attributable to the deconsolidation of Asterias which contributed to $8.6 million of the decrease as shown below. This decrease was offset by an increase of $1.2 million in BioTime research and development programs and $0.9 million in OncoCyte expenses related to cancer diagnostics.

The tables below show BioTime’s research and development expenses, by program and by company, for the three months ended December 31, 2016 and 2015, and for the years then ended.


Three Months Ended December 31
$000’s

Company Program 2016 2015
BioTime and ESI PureStem progenitor and pluripotent cell lines, and related research products $ 1,427 $ 1,609
BioTime
Hydrogel products, Renevia and other HyStem products and research

1,011 1,273
BioTime Hextend 12 18
Cell Cure OpRegen 1,653 1,357
OrthoCyte Orthopedic therapy 144 122
ReCyte Therapeutics Cardiovascular therapy 262 231
Subtotal therapeutic projects 4,509 4,610

Asterias Pluripotent cell therapy programs - 5,483

LifeMap Sciences Databases and mobile health products 1,099 1,459
OncoCyte Cancer diagnostics 1,405 1,236
Subtotal non-therapeutic projects 2,504 2,695

Total research and development expenses $ 7,013 $ 12,788

Year Ended December 31
$000’s

Company Program 2016 2015
BioTime and ESI PureStem progenitor and pluripotent cell lines, and related research products $ 6,060 $ 5,196
BioTime Renevia and other HyStem products and research 3,856 4,047
BioTime Hextend 54 59
Cell Cure OpRegen 4,803 4,086
OrthoCyte Orthopedic therapy 606 590
ReCyte Therapeutics Cardiovascular therapy 949 1,142
Subtotal therapeutic projects 16,328 15,120

Asterias Pluripotent cell therapy programs 8,684 17,322

LifeMap Sciences Databases and mobile health products 5,348 5,251
OncoCyte Cancer diagnostics 5,746 4,911
Subtotal non-therapeutic projects 11,094 10,162

Total research and development expenses $ 36,106 $ 42,604

G&A Expenses: The tables below show BioTime’s general and administrative expenses, by program and by company, for the three months ended December 31, 2016 and 2015, and for the years then ended. General and administrative expenses were $5.3 million for the fourth quarter, compared to $10.2 million for the fourth quarter of 2015. The decrease was primarily due to the May 2016 deconsolidation of Asterias financial results which contributed by $2.9 million of G&A recorded in the fourth quarter of 2015. The remaining decrease of approximately $2.0 million in the fourth quarter of 2016 was principally due to lower stock-based compensation. Year over year G&A was relatively unchanged at $28.4 million and $29.1 million, respectively.


Three Months Ended December 31
$000’s

Company 2016 2015
BioTime $ 2,331 $ 3,573
Cell Cure 266 211
OrthoCyte 322 422
ReCyte Therapeutics 106 413
Subtotal therapeutic entities 3,025 4,619

Asterias - 2,942

LifeMap Sciences 665 1,094
OncoCyte 1,653 1,568
Subtotal non-therapeutic entities 2,318 2,662
Total general and administrative expenses $ 5,343 $ 10,223

Year Ended December 31
$000’s

Company 2016 2015
BioTime $ 8,958 $ 9,761
Cell Cure 1,185 655
OrthoCyte 570 583
ReCyte Therapeutics 581 760
ESI 276 245
Subtotal therapeutic entities 11,570
12,003


Asterias 7,561 7,711

LifeMap Sciences 3,385 5,142
OncoCyte 5,910
4,278

Subtotal non-therapeutic entities 9,295
9,420

Total general and administrative expenses $ 28,426 $ 29,134

Net Income (loss) attributable to BioTime: Net loss attributable to BioTime was $5.1 million, or ($0.05) per basic and diluted common share for the three months ended December 31, 2016, compared to $13.4 million, or ($0.15) per basic and diluted common share due primarily to the deconsolidation of Asterias in May 2016. For 2016, net income attributable to BioTime was $33.6 million, or $0.35 per basic and $0.34 per diluted common share, compared to net loss attributable to BioTime of $47.4 million, or ($0.59) per basic and diluted common share. The 2016 net income attributable to BioTime was primarily due to the $49.0 million gain on deconsolidation of Asterias and the $34.4 million gain recognized from the increase in the market value of the Asterias shares owned by BioTime from May 13, 2016, the date of the deconsolidation, through the end of the year.