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.

On March 16, 2017 Oncolytics Biotech Inc. (Oncolytics or the Company) (TSX:ONC) (OTCQX:ONCYF) reported that cancer charity Myeloma UK launched MUK eleven, a first-of-its-kind immunotherapy trial that aims to modulate the immune system to target myeloma (Press release, Oncolytics Biotech, MAR 16, 2017, View Source [SID1234518154]). The Phase 1b trial will study immuno-viral therapy, REOLYSIN (pelareorep), in combination with Celgene Corporation’s immunomodulatory drugs (IMiDs), Imnovid (pomalidomide) or Revlimid (lenalidomide), as a rescue treatment in relapsing myeloma patients.

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"This trial, the first of its kind, is a novel way of activating a patient’s own immune system to target their myeloma (immunotherapy) using a natural virus and lenalidomide or pomalidomide, which could offer a new treatment strategy in myeloma," said MUK eleven Chief Investigator Gordon Cook, Consultant Haematologist at Leeds Teaching Hospitals Trust.

In MUK eleven, REOLYSIN will be combined with Celgene’s Imnovid or Revlimid in patients whose myeloma is progressing while on these IMiD treatments. The dose escalation trial will look at the safety and tolerability of these combinations, and will investigate whether the addition of REOLYSIN extends disease control in this patient group.

Dr. Adrian Kilcoyne, Celgene UK and Ireland Medical Director adds, "Celgene is delighted to be involved in this research with the Institute of Cancer Research, the University of Leeds, Myeloma UK and Oncolytics Biotech Inc. We are committed to bold pursuits in science and this is evidence of how strong collaboration between academia, patient groups and industry can drive important scientific advances for patients."

This clinical study expands on earlier pre-clinical work by Professor Cook that demonstrates that REOLYSIN has dual modes of action against multiple myeloma; being both directly cytotoxic and also activating immune effector cells to target and destroy cancer cells. Further, this immune-mediated activity can be enhanced by immunomodulatory agents to eliminate disease.

"This is an important collaboration for Oncolytics, as testing IMiD combinations with REOLYSIN is one of the three pillars of our clinical development plan," said Dr. Matt Coffey, President and CEO of Oncolytics. "We are eager to find out if REOLYSIN can enhance the benefits of these immune modulators and we are excited to start working with Myeloma UK and Celgene."

The trial will aim to recruit 44 patients across up to six Myeloma UK Clinical Trial Network centres in the UK. MUK eleven is part of the Myeloma UK Clinical Trial Network, a portfolio of early stage trials coordinated by the Clinical Trials Research Unit at the University of Leeds, focused on testing and speeding up access to promising new treatments for patients.

"We focus on strategic, collaborative and innovative approaches to delivering trials and treatments to patients," said Myeloma UK Director of Research Dr. Simon Ridley. "Our Clinical Trial Network is focused on ensuring that patients now, and in the future, are getting access to the exciting new treatment combinations we are seeing coming down the line, and so we are pleased to launch this novel trial which focuses on a pivotal area of myeloma research."

Oncolytics and Celgene UK & Ireland are providing their respective products for MUK eleven: Oncolytics Biotech Inc. is providing REOLYSIN and Celgene UK & Ireland is providing Imnovid and Revlimid.