On August 10, 2015 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported financial results for the quarter ended June 30, 2015 and provided an update on its development programs, including ThermoDox, its proprietary heat-activated liposomal encapsulation of doxorubicin, and GEN-1, an IL-12 DNA-based immunotherapy encased in a synthetic nanoparticle delivery system, which is currently under development for the localized treatment of ovarian and brain cancers (Press release, Celsion, AUG 10, 2015, View Source [SID:1234507134]).

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"Over the past few months, we reported positive data highlighting the multiple development opportunities for our portfolio, launched our European Early Access Program for ThermoDox in recurrent chest wall breast cancer and strengthened our balance sheet, providing a strong foundation as we advance our pipeline," said Michael H. Tardugno, Celsion’s chairman, president and CEO. "We remain on track to initiate key clinical studies this year, including the Euro-DIGNITY study evaluating ThermoDox in breast cancer, a Phase 1b trial for GEN-1 in first-line ovarian cancer, and a trial evaluating GEN-1 with Avastin in platinum-resistant ovarian cancer patients. In parallel, we continue enroll patients from North America, Europe and Asia Pacific in our global Phase III OPTIMA Study evaluating ThermoDox in primary liver cancer. Finally, we continue to evaluate ways to leverage our TheraSilence technology platform to advance the development of RNAi therapeutics that can be delivered directly to the lung."

Recent Developments

ThermoDox

Reported Positive Interim Data from the Phase II US DIGNITY Study in RCW Breast Cancer.

In July 2015, Celsion announced continuing positive interim data from its Phase II DIGNITY trial of ThermoDox in recurrent chest wall (RCW) breast cancer. Of the 17 patients enrolled and treated in the DIGNITY Study, 13 were eligible for evaluation of efficacy. Based on available data, every patient experienced a clinical benefit of their highly refractory disease with a local response rate of 69% observed in the 13 evaluable patients, notably 5 complete responses, 4 partial responses and 4 patients with stable disease.

Announced Updated Overall Survival Data from Phase III HEAT Study, Providing Strong Support for the Clinical Protocol for the Phase III OPTIMA Study.

As of July 15, 2015, the latest Overall Survival (OS) analysis demonstrated that in a large, well bounded, subgroup of patients (n=285, 41% of the study patients), the combination of ThermoDox and optimized RFA provided a 58% improvement in OS compared to optimized RFA alone. The Hazard Ratio at this analysis is 0.63 (95% CI 0.43 – 0.93) with a p-value of 0.0198. Median overall survival for the ThermoDox group has been reached which translates into a 25.4 month (2.1 year) survival benefit over the optimized RFA only group (79 months for the ThermoDox plus optimized RFA group versus 53.6 months for the optimized RFA only group). These data continue to support the protocol for the Phase III OPTIMA Study, which is evaluating ThermoDox in combination with optimized RFA, which will be standardized to a minimum of 45 minutes across all investigators and clinical sites for treating lesions 3 to 7 centimeters, versus standardized RFA alone. The study is expected to enroll up to 550 patients globally in up to 75 clinical sites in the United States, Europe, China and Asia Pacific.

Launched the ThermoDox Early Access Program (EAP) in Europe.

The Company and myTomorrows launched the ThermoDox Early Access Program in the second quarter, making ThermoDox available for sales to physicians who are treating patients with limited therapeutic options. The EAP provides physicians with access to products in later stage development demonstrating evidence of clinical benefit, with an acceptable safety profile and a quality manufacturing process in place. Celsion will be allowed to price ThermoDox at commercial rates.

GEN-1 IL-12 DNA-Based Immunotherapy

Presented Phase Ib Data for GEN-1 in Platinum-Resistant Ovarian Cancer at ASCO (Free ASCO Whitepaper).

In May 2015, Celsion presented clinical results from the Phase Ib trial for GEN-1in combination with pegylated doxorubicin in 16 patients with platinum-resistant ovarian cancer in a poster session at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting in Chicago. The clinical findings demonstrated an overall clinical benefit of 57% for all treatment arms, with a partial response (PR) rate of 21% and a stable disease (SD) rate of 36%. The overall clinical benefit observed at the highest dose cohort in this difficult-to-treat patient population was 100% (PR=33% and SD=67%) in all six evaluable patients. GEN-1 was well tolerated, with no dose limiting toxicities and no overlapping toxicities between GEN-1 and pegylated doxorubicin.

TheraSilence

Demonstrated Potent, Durable Preclinical Lung Expression Data for Its TheraSilence.

In May 2015, Celsion reported data from a preclinical study confirming that its TheraSilence technology platform can safely and effectively deliver RNA to the lungs in non-human primates, enabling the development of RNA therapeutics for lung diseases. In the study, TheraSilence-formulated signaling RNA resulted in preferential expression in the lungs, with expression in the liver at less than 15% of expression levels observed in the lungs, and expression levels in tissues other than the lung, spleen and liver at very low or background levels. A liver-directed delivery system, used as a positive control for the study, yielded preferential expression in liver and spleen, with only background expression levels observed in the lung.

Published Preclinical Data Demonstrating Lung Specific Delivery of microRNA-145 Inhibitor Using the TheraSilence Platform.

In May 2015, an abstract published in the Journal of Controlled Release summarized findings from a preclinical study confirming effective delivery of RNA to lung cells. In the study, the Company’s TheraSilence technology platform safely and effectively delivered an inhibitor of microRNA-145 (miR-145) in a well-established model of severe occlusive pulmonary arterial hypertension (PAH). Treatment was associated with significant delivery of miR-145 inhibitor in the lung, inhibition of miR-145 levels and reversal of the pulmonary hypertension associated with the advanced stages of the disease leading to a normalization of cardiovascular function.

Corporate Developments

Raised $8 Million Through Registered Direct Equity Offering priced "at the market".

During the second quarter of 2015, the Company completed an $8 million at-the-market registered direct equity offering and a concurrent private placement of warrants to purchase common stock with two institutional healthcare investors.

Financial Results

For the quarter ended June 30, 2015, Celsion reported a net loss of $5.7 million, or $(0.27) per share, compared to a net loss of $6.7 million, or $(0.38) per share, in the same period of 2014. Operating expenses were $5.4 million in the second quarter of 2015 compared to $6.5 million in the same period of 2014. For the six month period ended June 30, 2015, the Company reported a net loss of $12.7 million, or $(0.62) per share, compared to $12.1 million, or $(0.71) per share, in the same period of 2014. Operating expenses were $11.9 million in the first half of 2015 compared to $11.9 million in the same period of 2014. Net loss and operating expenses for the three-month and six-month periods ended June 30, 2014 included $1.1 million of one-time costs associated with the acquisition of EGEN, Inc. Net cash used in operations was $11.6 million in the first half of 2015 compared to $9.0 million in the same period last year. The Company ended the second quarter of 2015 with $30.8 million of total cash, investments and accrued interest on these investments, which included the proceeds of an $8 million registered direct offering that was completed during the quarter.

Research and development costs were $3.6 million in the second quarter of 2015 compared to $3.2 million the same period last year. Research and development costs were $8.1 million in the first half of 2015 compared to $6.1 million the same period last year. The increases in 2015 is primarily due to costs associated with the operations of EGEN, Inc., which the Company acquired in June 2014, and the costs associated with the initiation of the Phase III OPTIMA Study in 2014 and the production of clinical supplies in the first half of 2015 for the three GEN-1 Phase I studies. General and administrative expenses were $1.8 million in the second quarter of 2015 compared to $2.3 million the same period of 2014. General and administrative expenses were $3.8 million in the first half of 2015 compared to $4.7 million the same period of 2014. These decreases were primarily the result of lower insurance premiums and lower personnel costs.

On August 10, 2015 Celsion Corporation (NASDAQ: CLSN), an oncology drug development company, reported that it has expanded its Early Access Program (EAP) for lyso-thermosensitive liposomal doxorubicin (LTLD), referred to by Celsion as ThermoDox, with myTomorrows to include patients with primary liver cancer, also known as hepatocellular carcinoma (HCC) and liver cancer metastases, in all countries of the European Union (EU) territory, Switzerland, Turkey and Israel (Press release, Celsion, AUG 10, 2015, View Source [SID:1234507133]). The Company’s original European EAP with myTomorrows, formed in January 2015, provides eligible patients with access to LTLD for the treatment of recurrent chest wall (RCW) breast cancer. The EAP for LTLD was launched in the second quarter. LTLD is Celsion’s proprietary heat-activated liposomal encapsulation of doxorubicin, known as ThermoDox.

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The expanded EAP is based, in part, on data from Celsion’s Phase III HEAT study of ThermoDox in HCC. As of July 15, 2015, the latest overall survival (OS) analysis demonstrated that in a large, well bounded, subgroup of patients (n=285, 41% of the HEAT study patients), the combination of ThermoDox and optimized RFA provided a 58% improvement in OS compared to optimized RFA alone. The Hazard Ratio at this analysis is 0.63 (95% CI 0.43 – 0.93) with a p-value of 0.0198. Median Overall Survival for the ThermoDox group has been reached which translates into a 25.4 month (2.1 year) survival benefit over the optimized RFA only group (79 months for the ThermoDox plus optimized RFA group versus 53.6 months for the optimized RFA only group).

"The survival data from the large subgroup of patients in our HEAT study provides strong support for the expansion of the LTLD EAP into this patient population that has limited treatment options," stated Dr. Nicolas Borys, Celsion’s senior vice president and chief medical officer. "We will be working closely with myTomorrows to set up the EAP in this indication."

EAPs allow biopharmaceutical companies to provide eligible patients with ethical access to investigational medicines for unmet medical needs within the scope of the existing early access legislation. Access is provided in response to physician requests in a fully compliant manner, where no alternative treatment options are available to these patients. Celsion will provide LTLD in the EU, Switzerland, Turkey and Israel through myTomorrows. The Company expects to have LTLD available for HCC in the second half of 2015.

"This expansion of our EAP in liver cancer reflects our commitment to provide access to patients in desperate need of new treatment options," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "We have been pleased with establishing our Early Access Program in RCW breast cancer, and look forward to being able to provide expanding access to patients with HCC and liver metastases consistent with our goal of providing effective therapies which may significantly prolong and improve the quality of life. In parallel with this EAP, we are also continuing to focus on enrolling patients in our global Phase III OPTIMA study in HCC designed to support registration in key markets worldwide. We have expanded our HCC clinical development footprint with the addition of 17 clinical sites in Spain, Germany and Italy."

About LTLD

Lyso-Thermosensitive Liposomal Doxorubicin (LTLD) is a proprietary heat-activated liposomal encapsulation of doxorubicin, an approved and frequently used oncology drug for the treatment of a wide range of cancers. LTLD is being evaluated in a Phase III clinical trial for primary liver cancer and a Phase II clinical trial for recurrent chest wall breast cancer. Localized mild hyperthermia (39.5 – 42 degrees Celsius) releases the entrapped doxorubicin from the liposome. This delivery technology enables high concentrations of doxorubicin to be deposited preferentially in a targeted tumor.

About myTomorrows

myTomorrows is an online patient platform that is creating freedom of choice for patients with unmet medical needs by offering earlier access to medicines that show promising results during clinical trials, but are not officially registered yet. With the support of their doctors, patients who suffer from cancer, a neurological disorder, a rare disease or a severe depression, can have earlier access to such medicines. For more information about myTomorrows, please visit the website www.mytomorrows.com.

On August 10, 2015 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the second quarter ended June 30, 2015 (Press release, Celldex Therapeutics, AUG 10, 2015, View Source [SID:1234507132]).

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"Celldex is acutely aware of the significant unmet need for patients with glioblastoma, and we continue to focus considerable efforts on advancing the RINTEGA program," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "At ASCO (Free ASCO Whitepaper) in June, we presented data from the Phase 2 ReACT study, which achieved the primary endpoint of progression-free survival at six months and, most importantly, demonstrated a survival benefit for patients on the RINTEGA arm. We continue to follow these patients and look forward to presenting data on the emerging long-term survival benefit later this year. The Phase 3 ACT IV study continues to progress as planned, and we look forward to the second interim analysis in late 2015/early 2016."

"Our momentum is further supported by ongoing progress in our pipeline. Varlilumab is now in four combination studies, and we anticipate that a fifth study with Roche’s anti-PDL1 antibody will start later this year. We look forward to presenting data from a number of these studies in 2016 and believe these data will define an important role for varlilumab in cancer immunotherapy treatment. Glembatumumab vedotin also continues to actively enroll patients in two trials—the METRIC study in triple negative breast cancer and a study in metastatic melanoma. We are completing preparations that support the opening of METRIC study sites in the EU early next year and remain on track to complete enrollment in 2016. As we continue to advance Celldex towards becoming a fully integrated, commercial-stage biotechnology company, we were pleased to announce the promotion of Dr. Rick Wright to the position of Chief Commercial Officer. In this role, Rick will be responsible for developing a global business strategy and building the infrastructure required to support commercialization of RINTEGA and our cancer immunotherapy pipeline," concluded Marucci.

Program Updates:

RINTEGA ("rindopepimut"; "rindo"; CDX-110), an EGFRvIII(v3)-specific therapeutic vaccine for glioblastoma (GBM)

In June, the independent Data Safety and Monitoring Board (DSMB) recommended continuation of the Phase 3 ACT IV study of RINTEGA (rindopepimut) in patients with newly diagnosed glioblastoma as a result of a prespecified interim analysis assessing safety, futility and efficacy at 50% of events (deaths). The ACT IV study is a randomized, double-blind, placebo controlled study of RINTEGA plus GM-CSF added to standard of care temozolomide in patients with newly diagnosed, surgically resected, EGFRvIII-positive glioblastoma. 745 patients were enrolled into ACT IV to reach the required 374 patients with minimal residual disease (assessed by central review) needed for analysis of the primary overall survival endpoint. All patients, including those with disease that exceed this threshold, will be included in a secondary analysis of overall survival as well as analyses of progression-free survival, safety and tolerability, and quality of life. The second interim analysis is expected to occur in late 2015/early 2016.
Data from the Phase 2 ReACT study in patients with recurrent glioblastoma were presented in an oral session at the 2015 ASCO (Free ASCO Whitepaper) Annual Meeting by David A. Reardon, M.D., Clinical Director, Center for Neuro-Oncology, Dana-Farber Cancer Institute and Associate Professor of Medicine, Harvard Medical School, and the lead investigator of the ReACT study. Patients on the RINTEGA arm experienced a statistically significant overall survival (OS) benefit, and an impressive long-term survival benefit is emerging. The primary endpoint of the study, progression-free survival at six months (PFS6), was met, and a clear advantage was demonstrated across multiple, clinically important endpoints including long-term progression-free survival, objective response rate (ORR) and need for steroids. The Company anticipates that mature overall survival and long-term survival will be presented by year-end at an upcoming medical meeting.

Based on discussions to date with the regulatory authorities about the Phase 2 REACT data, the Company continues to believe the most likely scenario for potential RINTEGA approval filings will be upon receiving data from the ACT IV study, at which time the Company expects that it would file for full approval in both the front-line and recurrent setting. Under the RINTEGA program’s recently awarded Breakthrough Therapy Designation, the Company is actively engaged with the Center for Biologics Evaluation and Research (CBER) to complete all of the required activities associated with the ability to apply for licensure and considerable progress is being made, particularly in the areas of chemistry manufacturing and controls (CMC) and companion diagnostics readiness. The Company will continue to take all the necessary steps to prepare for filing so this process can be completed as expeditiously as possible when ACT IV data become available.

Glembatumumab vedotin ("glemba"; CDX-011), an antibody-drug conjugate targeting gpNMB in multiple cancers

Patient enrollment has accelerated in the Company’s Phase 2b randomized study (METRIC) of glembatumumab vedotin in patients with metastatic triple negative breast cancers that overexpress gpNMB, a molecule associated with poor outcomes for triple negative breast cancer patients and the target of glembatumumab vedotin. Approximately 100 sites are open to enrollment across the United States, Canada and Australia. Trial expansion into the European Union (EU) is underway, and the Company plans to open enrollment in up to 50 sites in the EU in early 2016. Based on current projections, enrollment will be completed in the second half of 2016.

Patient enrollment continues in the Phase 2 study of glembatumumab vedotin in metastatic melanoma. To date, 10 sites are open to enrollment in the United States.

Celldex continues to advance plans to expand the study of glembatumumab vedotin in other cancers in which gpNMB is expressed.
Study design is being finalized for a Phase 2 study in squamous cell lung cancer, and the study is expected to commence in 2H 2015.

Celldex and the National Cancer Institute have entered into a Cooperative Research and Development Agreement (CRADA) under which the NCI will sponsor two studies of glembatumumab vedotin—one in uveal melanoma and one in pediatric osteosarcoma. Protocols for the study are currently being developed.

Varlilumab ("varli"; CDX-1127), a fully human monoclonal agonist antibody that binds and activates CD27, a critical co-stimulatory molecule in the immune activation cascade

The Phase 1/2 study of varlilumab and nivolumab (Opdivo) in adult patients with advanced non-small cell lung cancer, metastatic melanoma, colorectal cancer, ovarian cancer, and head and neck squamous cell carcinoma is actively enrolling patients. This study is being conducted by Celldex under a clinical trial collaboration with Bristol-Myers Squibb Company. The companies are sharing development costs.

In April 2015, Celldex announced that it had entered into a clinical trial collaboration with Roche to evaluate the combination of varlilumab and atezolizumab (anti-PDL1) in a Phase 1/2 study in renal cell carcinoma. Under the terms of this agreement, Roche will provide study drug, and Celldex will be responsible for conducting and funding the study, which is expected to open to enrollment in 2H 2015.

In the second quarter, the Company announced the initiation of two combination studies of varlilumab, both of which are now enrolling patients. Efforts are underway for additional Phase 2 studies of varlilumab, and the Company will provide updates on these studies as they are initiated. Newly initiated studies in the second quarter include:

A Phase 1/2 safety and tolerability study examining the combination of varlilumab and sunitinib (Sutent) in patients with metastatic clear cell renal cell carcinoma (CC-RCC); and,

A Phase 1/2 safety and tolerability study examining the combination of varlilumab and ipilimumab (Yervoy) in patients with Stage III or IV metastatic melanoma. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive Celldex’s CDX-1401.

Celldex presented preclinical data that support varlilumab’s expansion into combination studies with PD-1 inhibitors in a poster session at the 2015 AACR (Free AACR Whitepaper) Annual Meeting in April. Data demonstrated that the combination of varlilumab and anti-PDL1 induces a potent immune-mediated effect that results in important changes in the tumor microenvironment. Most notably, the combination strategy improved the ratio of effector T cells to regulatory T cells, which was accompanied by a reduction in the expression of PD-1 on both effector and regulatory T cells.

The Phase 1b study of varlilumab and ONT-10, Oncothyreon’s therapeutic vaccine targeting the tumor-associated antigen MUC1, continues to actively enroll patients with advanced breast or ovarian cancer. Celldex is providing study drug, and Oncothyreon is conducting the study.

CDX-1401, an antibody-based NY-ESO-1-specific therapeutic vaccine for multiple solid tumors

In the second quarter, Celldex announced the initiation of a Phase 1/2 study examining the combination of varlilumab and ipilimumab (Yervoy) in patients with Stage III or IV metastatic melanoma. This study is currently open to enrollment. In the Phase 2 portion of the study, patients with tumors that express NY-ESO-1 will also receive CDX-1401, an off-the-shelf antibody-based dendritic cell targeted vaccine.

Celldex continues to support several external collaborations, including a National Cancer Institute sponsored Phase 2 study of CDX-1401 and CDX-301 for patients with metastatic melanoma, which is open to enrollment.

CDX-301 (recombinant human Flt3L), a potent hematopoietic cytokine that uniquely expands dendritic cells and hematopoietic stem cells

CDX-301 is being developed as a combination product with other immuno-oncology agents in a number of investigator-sponsored studies.

A pilot study of CDX-301 alone and in combination with Mozobil in hematopoietic stem cell transplantation was initiated in September 2014 and is currently enrolling patients and sibling-matched donors.

Second Quarter and First Six Months 2015 Financial Highlights and 2015 Guidance

Cash position: Cash, cash equivalents and marketable securities as of June 30, 2015 were $334.0 million compared to $359.8 million as of March 31, 2015. The decrease was primarily driven by our second quarter net cash burn of $25.8 million. As of June 30, 2015 Celldex had 98.5 million shares outstanding.

Revenues: Total revenue was $2.2 million in the second quarter of 2015 and $2.7 million for the six months ended June 30, 2015, compared to $0.6 million and $1.0 million for the comparable periods in 2014. The increase in the second quarter of 2015 and the six months ended June 30, 2015 was primarily due to our clinical trial collaboration with Bristol-Myers Squibb and our research and development agreement with Rockefeller University.

R&D Expenses: Research and development (R&D) expenses were $26.5 million in the second quarter of 2015 and $51.6 million for the six months ended June 30, 2015, compared to $24.1 million and $51.2 million for the comparable periods in 2014.

G&A Expenses: General and administrative (G&A) expenses were $8.2 million in the second quarter of 2015 and $14.3 million for the six months ended June 30, 2015, compared to $4.8 million and $9.4 million for the comparable periods in 2014. The increase in G&A expenses was primarily attributable to higher commercial personnel-related expenses as we prepare for potential product launch and a $2.2 million increase year to date in RINTEGA and glembatumumab vedotin commercial planning costs over the $1.9 million spent in the comparable period in 2014.

Net loss: Net loss was $32.4 million, or ($0.33) per share, for the second quarter of 2015 and $62.5 million, or ($0.65) per share, for the six months ended June 30, 2015, compared to a net loss of $28.3 million, or ($0.32) per share and $58.2 million, or ($0.65) per share for the comparable periods in 2014.

Financial guidance: Celldex expects that its cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements through 2017; however, this could be impacted by our clinical data results from the RINTEGA program and their potential impact on our pace of commercial manufacturing and the rate of expansion of our commercial operations.

RINTEGA is a registered trademark of Celldex Therapeutics. Opdivo and Yervoy are registered trademarks of Bristol-Myers Squibb. Sutent is a registered trademark of Pfizer. Mozobil is a registered trademark of sanofi-aventis U.S. LLC.

On August 10, 2015 BioTime, Inc. (NYSE MKT:BTX) reported financial results for the second quarter ended June 30, 2015 and provided a corporate update (Filing, 8-K, BioTime, AUG 10, 2015, View Source [SID:1234507131]).

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"BioTime’s management team has sharpened its focus on our high priority programs," said Dr. Michael D. West, BioTime’s Chief Executive Officer. "Our strategy for achieving the leadership role in regenerative medicine includes: continuing to advance the ongoing clinical trials of our products that are expected to address large unmet patient needs, collaborating with high-quality corporate partners and leading academic medical institutions, financial de-risking by leveraging various sources of non-dilutive financing, adding experienced biopharma executives to our teams at both BioTime and our subsidiaries, and progressively unlocking shareholder value in our subsidiaries. We continue to make progress with our several clinical programs in cell therapies, cell delivery matrices, and cancer diagnostics."

2015 Highlights

Through the second quarter, BioTime and its subsidiaries have reported the following progress on key products and programs.

Cell Therapies

Cell Cure Neurosciences Ltd.

Cell Cure Neurosciences, Ltd. (Cell Cure Neurosciences) is currently enrolling patients at Hadassah University Medical Center in Jerusalem, Israel, in a clinical Phase I/IIa dose-escalation study evaluating the safety and efficacy of OpRegen for geographic atrophy (GA), the severe stage of the dry form of age-related macular degeneration (dry-AMD). Dry-AMD represents nearly 90% of AMD prevalence and currently has no FDA-approved therapy. The Phase I/IIa clinical trial is designed with four cohorts and allows for interim data readouts.

Cell Cure Neurosciences presented preclinical efficacy data for its lead product candidate, OpRegen at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in May. The findings demonstrated the product’s potential to preserve vision and retinal structure when transplanted into the leading animal model of retinal disease.
In May, Cell Cure Neurosciences was awarded a grant for 2015 of 6.24 million shekels (approximately $1.61 million) from Israel’s Office of the Chief Scientist (OCS) to help finance the development of OpRegen. The OCS has previously supported Cell Cure Neurosciences, providing grants totaling approximately $8.0 million to date in non-dilutive funding.
Asterias Biotherapeutics, Inc. (NYSE MKT: AST)

Asterias Biotherapeutics, Inc. (Asterias) promoted Edward Wirth, M.D., Ph.D. to Chief Medical Officer.
In June, Asterias announced that the first patient had been dosed at the Atlanta-based Shepherd Center in a Phase I/IIa clinical trial evaluating the activity of escalating amounts of AST-OPC1 (oligodendrocyte progenitor cells) in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI). The Phase I/IIa trial is part of the planned registration program for AST-OPC1, with neurologically complete cervical SCI as the first targeted indication.

Also in June, Asterias announced positive long-term follow-up data from a Phase II clinical trial of AST-VAC1 in patients with acute myelogenous leukemia (AML). The results showed that more than 50% of those who received AST-VAC1 had prolonged relapse-free survival, even patients with high-risk AML, including those over 60 years of age and patients in second remission. The data were presented during an oral presentation at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in May.
Asterias was added to the Russell 3000, Russell Global, and Russell Microcap Indexes on June 26, 2015 as part of Russell Investments’ annual reconstitution of its comprehensive set of U.S. and global equity indexes. The Russell indexes are widely used by investment managers and institutional investors for index funds and they serve as benchmarks for passive and active investment strategies.

During the second quarter, Asterias raised a total of $14.5 million in aggregate gross proceeds through various private and public offerings, as well as receiving $1.1 million from the California Institute of Regenerative Medicine (CIRM) in accordance with a quarterly disbursement schedule under the $14.3 million grant award related to the AST-OPC1 development program. Year-to-date payments from CIRM total $3.3 million in non-dilutive funding.
Cell Delivery Matrices

Earlier this year, BioTime announced the successful treatment of the first patient in the Company’s pivotal clinical trial in Europe of Renevia for HIV-associated lipoatrophy, which was chosen as the clearest regulatory pathway as the first indication. Patient enrollment is ongoing with completion of enrollment in the trial expected by early next year. Renevia, BioTime’s proprietary cell delivery matrix, is specifically designed to facilitate the stable engraftment and proliferation of transplanted cells.
The results of the Renevia trial could ultimately lead to a submission in 2016 for CE Mark approval in Europe for the treatment of HIV-associated facial lipoatrophy. Positive outcomes of this trial could greatly accelerate the potential development of future therapeutics for other lipoatrophy-related conditions, as well as the potential to expand the use of BioTime’s cell delivery matrices for a number of additional cell types.
Cancer Diagnostics Platform

OncoCyte Corporation

William Annett was named Chief Executive Officer of OncoCyte Corporation (OncoCyte) on June 16, 2015. Bill has extensive experience as a CEO of diagnostics companies and as an executive with Genentech and Accenture, among other companies. His deep experience with product commercialization at leading companies is of particular importance as OncoCyte prepares to launch its first liquid biopsy cancer diagnostic test, currently scheduled for 2016.

OncoCyte also reported positive results from its proprietary, non-invasive, liquid biopsies diagnostics at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) for bladder and breast cancer and the American Thoracic Society (ATS) for lung cancer diagnostics.
OncoCyte announced the appointment of Andrew Arno to its Board of Directors on July 15, 2015. Mr. Arno’s depth of experience in the capital markets, as well as advising emerging companies is expected to greatly benefit the company.
Additional Updates

LifeMap Solutions, Inc.

LifeMap Solutions, Inc. (LifeMap Solutions) continues to extend its lead as the premier commercial entity building on the new ResearchKit software framework developed by Apple, Inc. As previously announced in the first quarter, LifeMap Solutions launched the Asthma Health app. Asthma Health serves as the interface for participants in a large-scale medical asthma research study with the Icahn School of Medicine at Mount Sinai. In the second quarter, the Company posted initial user-behavior findings to the official ResearchKit blog; these initial findings showed the app’s user-retention numbers to be comparable to those of top-charting apps like social networks.

In collaboration with the Mount Sinai – National Jewish Health Respiratory Institute, the Company has also developed a clinical-care app that empowers Chronic Obstructive Pulmonary Disease (COPD) patients to manage their condition under the oversight of a physician. This app, COPD Navigator, continues in its pilot program at Mount Sinai. The company has announced that it will build additional clinical-care apps to manage different chronic conditions.

Patents

In the first half of 2015, BioTime was notified of the issuance of 27 new patents that add to over 700 patents and patent applications filed world wide and licensed or owned by the BioTime family of companies in the field of regenerative medicine. The new patents, either issued or licensed to BioTime or certain of its subsidiaries, includes seven U.S. patents, as well as twenty additional patents issued in Europe, Japan, Canada and Singapore.
Financial Results

Revenue

BioTime’s operating revenues are currently generated from research grants, licensing fees and royalties from the sale of Hextend, and advertising from the marketing of the LifeMap Sciences, Inc.’s (LifeMap Sciences) online database products, and from the sale of hydrogels and stem cell products for research.

Total consolidated revenues for the six months ended June 30, 2015, on a consolidated basis, total revenues were $3.3 million, up $1.1 million or 50% from $2.2 million for the same period one year ago. The increase in revenues is primarily attributable to a $0.9 million increase in grant income primarily from Israel’s Office of the Chief Scientist and CIRM.

Expenses

Consolidated operating expenses for the second quarter were $15.2 million, compared to $13.9 million for the same period in 2014. General and administrative (G&A) expenses for the second quarter were $6.2 million, compared to $4.8 million in the second quarter a year ago. The $1.4 million increase is in part a result of increased staffing at Asterias and at LifeMap Solutions.

Operating expenses for the six months ended June 30, 2015 were $29.7 million, compared to expenses of $26.0 million for the same period of 2014. Excluding Asterias’ operating expense of $10.8 million, BioTime’s expenses alone total $18.9 million. The increase in operating expenses is primarily attributable to increase in staffing and increased expenditures in the Asterias, OncoCyte, and LifeMap Solutions product development programs offset in part by a reduction in development expenses in BioTime’s HyStem hydrogel and the OrthoCyte and ReCyte Therapeutics product development programs.

Net Loss

Net loss attributable to BioTime for the three months ended June 30, 2015 was $9.7 million, including deferred income tax benefits of $1.3 million. For the same period in 2014, net loss was $9.5 million, including deferred income tax benefits of $1.5 million. On a per share basis, net loss for the second quarter in 2015 was $0.12 per share, compared to a net loss of $0.16 per share for the same period in 2014.

Net loss attributable to BioTime common shareholders for the six months ended June 30, 2015 was $19.9 million or $0.25 per share, compared to a net loss of $17.6 million or $0.29 per share per share for the same period in 2014. The increase in net loss is primarily attributed to increased expenditures in the Asterias, OncoCyte, and LifeMap Solutions product development programs offset in part by a reduction in development expenses in BioTime’s HyStem hydrogel and the OrthoCyte and ReCyte Therapeutics product development programs. This increase is to some extent offset by the $2.4 million income tax benefit recorded as of June 30, 2015 and $2.9 million in the same period in 2014.

Net losses attributable to BioTime include losses from BioTime majority owned subsidiaries based upon BioTime’s percentage ownership of those subsidiaries.

Balance Sheet and Subsequent Financing Events

Cash and cash equivalents totaled $31.5 million as of June 30, 2015, compared to $29.5 million as of December 31, 2014. The cash on hand as of June 30, 2015 includes $21.2 million held by Asterias and other subsidiaries.

During the six months ended June 30, 2015, BioTime and certain of its subsidiaries raised approximately $24.0 million of additional equity capital and $5.2 million in non-dilutive funding as follows:

Asterias

$2.8 million gross proceeds from the sale of Asterias AST common stock in "at-the-market" transactions;
$5.5 million in aggregate gross proceeds from the public offering and concurrent private placement of Asterias’ common stock;
$11.7 million from the exercise of 5,000,000 outstanding Asterias common share purchase warrants originally issued in June 2014;
$3.3 million in non-dilutive funding from CIRM.
BioTime

$621,000 from the exercise of BioTime options by employees.
Cell Cure Neurosciences

$1.9 million in non-dilutive funding from the OCS.
OncoCyte

$3.3 million from the sale of 3,000,000 of OncoCyte common stock to long-term OncoCyte investors.

On August 10, 2015 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a leading biotechnology company in the emerging field of regenerative medicine, reported financial and operating results for the second quarter ended June 30, 2015 (Press release, BioTime, AUG 10, 2015, View Source;p=RssLanding&cat=news&id=2078493 [SID:1234507130]).

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"In the second quarter, we continued to make progress on multiple fronts toward advancing the clinical development of our key therapeutic programs," stated Pedro Lichtinger, President and CEO of Asterias. "We presented new, long-term follow-up Phase 2 clinical data of AST-VAC1 in acute myelogenous leukemia, the most common form of acute leukemia in adults, which showed potential for prolonged relapse-free survival in the overall study population, as well as in the sub-population of adults 60 years and older for which there remains a major unmet need. Based on the promising clinical data observed with AST-VAC1, we are pursuing opportunities to fund a focused clinical development plan for potential commercialization of AST-VAC1 through partnerships or non-dilutive funding."

Mr. Lichtinger continued, "In addition, we dosed the first patient in our Phase 1/2a clinical trial of AST-OPC1 for complete cervical spinal cord injury, a trial which is designed to evaluate the product at the doses and in the population where it has the maximum potential to bring benefit to patients. Importantly, we were successful in our efforts to further strengthen our balance sheet during the quarter by increasing our cash position by approximately $8 million, which has enhanced our financial flexibility to continue to execute our strategic plan through mid-2016. At the same time, during the quarter we completed some administrative tasks, namely the registration of all unregistered Asterias outstanding shares, including those shares held by our parent company BioTime. BioTime remains our largest shareholder and it is important to note that BioTime has not sold any of its position in Asterias since the June 10, 2015 effective date of the registration statement."

Recent Research and Development Highlights:

AST-VAC1 (antigen-presenting autologous dendritic cells)

Positive, new, long-term follow-up data from a Phase 2 clinical trial of AST-VAC1, the Company’s autologous telomerase-based dendritic cell cancer vaccine, in patients with intermediate and high risk acute myelogenous leukemia (AML) was presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June. The long-term follow-up showed that 57% of patients who received AST-VAC1 had prolonged relapse-free survival, even patients with high-risk AML including those over 60 years old and patients in second remission.

Asterias is in the process of establishing its clinical development plan for potential commercialization of AST-VAC1. The next step is planned to be a larger Phase 2/3 clinical trial designed to validate the positive findings from the completed Phase 2 study. Concurrently, the Company has commenced evaluation of strategic alternatives to further advance clinical development either independently or with a partner, and maximize the value of AST-VAC1.

AST-OPC1 (oligodendrocyte progenitor cells)

In June, the first patient was successfully dosed at the Atlanta-based Shepherd Center in a Phase 1/2a clinical trial evaluating activity of escalating doses of AST-OPC1 in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI). The Phase 1/2a trial, to be conducted in eight to ten centers in the United States, is part of the planned registration program for AST-OPC1, with neurologically complete cervical SCI as the first targeted indication. The open-label, single-arm study will test three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 13 patients with sub-acute, C-5 to C-7, neurologically complete cervical SCI. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product. The Company expects availability of safety data from the first patient cohort in the Phase 1/2a trial during the second half of 2015, and initial efficacy data readouts from the trial in the second half of 2016.

If initial safety data from the 10 million cell cohort is positive, Asterias plans to file an Investigational New Drug Application (IND) amendment with the U.S. Food and Drug Administration (FDA) to expand enrollment in the study to include up to 40 patients with an adaptive design. An adaptive design clinical study is a study that includes a prospectively planned opportunity for modification of one or more specified aspects of the study design and hypotheses based on analysis of data from subjects in the study. The Company believes this flexible methodology can increase the chance to demonstrate the superiority of AST-OPC1 by increasing the statistical confidence of the safety and efficacy readouts, and position the product for potential accelerated regulatory approvals.

In the second 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 is nearing completion of transfer of the cGMP-compatible AST-VAC2 process to development partner Cancer Research UK (CRUK). Confirmatory runs are in progress at both Asterias and CRUK, with completion of the full transfer expected in the fourth quarter of 2015. Following completion of the technology transfer, CRUK will, at its own cost, manufacture clinical grade AST-VAC2 and conduct the Phase 1/2a clinical trial in patients with non-small cell lung cancer in the UK, subject to regulatory approval. Asterias continues to expect potential regulatory clearance to begin treating patients as part of the Phase 1/2a trial in the second half of 2016.

Other Corporate Developments:

In May, Edward D. Wirth, III, M.D., Ph.D., was promoted to the newly created role of Chief Medical Officer. Dr. Wirth was Chief Translational Officer since joining Asterias in March 2013. In his new role, Dr. Wirth serves as an executive officer of the Company and provides strategic leadership for Asterias’ clinical development activities including its therapeutic programs, AST-OPC1 for spinal cord injuries, AST-VAC1 for AML and AST-VAC2 for lung cancer.

At the same time, Jane Lebkowski, President Research and Development was named as President of R&D and Chief Scientific Officer. Dr Lebkowski has been in the field and cell and gene therapy for 29 years and an employee of Asterias since March 2013. Dr Lebkowski is responsible for all research, product and regulatory development of Asterias products.

In May, Asterias received total proceeds of $11.7 million resulting from the exercise of all outstanding common share purchase warrants originally issued in June 2014. The proceeds will be used to further advance the Company’s development programs.
In June, Asterias was added to the Russell 2000, Russell 3000, Russell Global and Russell Microcap indexes following Russell Investments’ (Russell) reconstitution of its comprehensive set of U.S. and global equity indexes. Each June, Russell completely rebalances its indexes, known as a reconstitution, to reflect market changes in the past year. The Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for passive and active investment strategies.
Second Quarter [Unaudited] Financial Results

Total revenues in the second quarter 2015 were $772,000, which were primarily comprised of grant income and royalty revenues on product sales by licensees. Total revenues in the year ago quarter were $21,000. Operating expenses in the second quarter were $5.5 million, compared to $4.3 million in the prior year period. Research and development (R&D) expenses in the second quarter were $3.7 million, compared to $2.7 million in the year ago quarter. General and administrative (G&A) expenses in the second quarter were $1.8 million, compared to $1.5 million in the year ago quarter.

Net loss for the second quarter 2015 was $3.6 million, including a deferred income tax benefit of approximately $1.2 million. Net loss in the second quarter 2014 was $2.8 million, including a deferred income tax benefit of approximately $1.5 million. On a per share basis, net loss for the second quarter was $0.10 per share, compared to a loss of $0.09 per share for the year ago quarter.

Cash and cash equivalents were $15.6 million as of June 30, 2015, compared to $3.1 million as of December 31, 2014. In May 2015, Asterias received total proceeds of $11.7 million resulting from the exercise of all outstanding common share purchase warrants originally issued in June 2014. In addition, Asterias raised approximately $2.8 million in gross proceeds through the Company’s at-the-market (ATM) equity offering program during the second quarter of 2015. At June 30, 2015, the Company held 3.9 million BioTime common shares, with a market value of approximately $14.0 million on that date. For the second quarter, net cash used in operating activities was $4.7 million. The Company continues to expect net cash burn in 2015 to be in the range of $15 million to $17 million.