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Ignyta Announces First Quarter 2016 Company Highlights and Financial Results

On May 10, 2016 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported company highlights and financial results for the first quarter ended March 31, 2016 (Press release, Ignyta, MAY 10, 2016, View Source [SID:1234512213]).

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"During 2016, we have continued to make significant progress towards becoming a leading precision medicine company focused on the development of first-in-class and best-in-class therapies for the benefit of cancer patients, and we have steadily advanced each of our three clinical stage assets," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "For our lead program, entrectinib, we have continued to successfully execute our potentially registration-enabling Phase 2 clinical trial, STARTRK-2. We also announced at the AACR (Free AACR Whitepaper) Annual Meeting compelling results from our two Phase 1 clinical trials, highlighting entrectinib’s emerging safety and efficacy profile. For the RXDX-105 program, we selected a recommended Phase 2 dose and initiated the Phase 1b portion of our ongoing clinical trial. For the taladegib program, we reported a complete response in a patient with medulloblastoma – providing the first clinical proof of concept for taladegib in a solid tumor type outside of advanced basal cell carcinoma. Finally, we strengthened our balance sheet, enabling us to continue to move rapidly to develop meaningful new therapies for the benefit of cancer patients."

Company Highlights

Updated Entrectinib Data Presented at AACR (Free AACR Whitepaper) Annual Meeting

In April 2016, updated results of the two Phase 1 clinical trials of entrectinib, the company’s proprietary oral tyrosine kinase inhibitor targeting solid tumors harboring activating alterations to NTRK1, NTRK2, NTRK3, ROS1, or ALK, were presented in an oral plenary session at the 2016 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) in New Orleans, Louisiana.

The data cut-off for the AACR (Free AACR Whitepaper) presentation was March 7, 2016. Highlights of the data included:

Safety

A total of 119 patients with a range of solid tumors had been dosed across the two Phase 1 clinical trials, with 45 patients treated at the recommended Phase 2 dose (RP2D) of 600 mg, taken orally once per day (QD).

Entrectinib was well tolerated:

Across both studies, the most frequent (>10% incidence) treatment-related adverse events were fatigue (44%), dysgeusia (41%), paresthesia (28%), nausea (24%), and myalgia (22%).
The vast majority of treatment-related adverse events were Grade 1 or 2 in severity.
The most frequent (>2% incidence) Grade 3 treatment-related adverse events were fatigue (4%) and anemia (3%).
Adverse events were reversible with dose modification.
There was no evidence of cumulative toxicity, hepatic or renal toxicity, or QTc prolongation.
Efficacy

Across the two Phase 1 clinical studies, there were 25 patients treated who met the company’s Phase 2 clinical trial eligibility criteria, which include:

Presence of NTRK1/2/3, ROS1, or ALK gene rearrangements, as opposed to other types of molecular alterations (e.g., SNPs, amplifications, deletions);
Naïve to inhibitors of the relevant target (Trk, ROS1, or ALK, respectively); and
Treatment at or above the RP2D.
Among the 25 patients treated who met the company’s Phase 2 clinical trial eligibility criteria, tumor regression was seen in 80% (20 out of 25 treated patients):

Twenty-four patients had tumors that were evaluable by RECIST criteria. The overall response rate by RECIST was 79% (19 responses, including two complete responses, out of 24 treated patients, as assessed and confirmed by the clinical sites).
One patient had an astrocytoma. Assessment by RECIST criteria demonstrated stable disease. However, since RECIST criteria are not validated for primary brain tumors, the clinical site performed three-dimensional volumetric analysis of this patient’s tumor to determine changes in tumor size, which resulted in an estimated 45% decrease in tumor size from baseline.
The responses included:
with respect to patients with NTRK1/2/3 gene rearrangements who met the company’s Phase 2 eligibility criteria, three confirmed responses out of three patients evaluable by RECIST criteria, one of whom had metastases to the central nervous system that resulted in a complete response. In addition, a patient with an astrocytoma had a response as determined by volumetric analysis. Two of these Trk patients remained on study, one of whom had been on study for longer than 12 months;
with respect to patients with ROS1 gene rearrangements who met the company’s Phase 2 eligibility criteria, 12 confirmed responses out of 14 patients, including 11 confirmed responses out of 13 patients with non-small cell lung cancer (NSCLC). Eleven of the ROS1 responders remained on study in response, with the longest at 27 months. One ROS1 NSCLC patient has met the criteria for RECIST progression but has remained on study due to clinical benefit; and
with respect to patients with ALK gene rearrangements who met the company’s Phase 2 eligibility criteria, four confirmed responses out of seven patients, with another patient having stable disease. Two of the responders remained on study in response, as did the patient with stable disease.
Many of these responses occurred rapidly, within the first four weeks of entrectinib treatment. Seventeen of the patients remained on study treatment, having received up to 27 months of treatment. Of note, three of four patients with primary or metastatic central nervous system (CNS) disease responded.

In addition, the AACR (Free AACR Whitepaper) presentation included a late-breaking case study of a 20-month-old baby boy with NTRK3-rearranged infantile fibrosarcoma that had metastasized to the brain, who had exhausted all available therapies and was enrolled under a compassionate use protocol and therefore was not included in the Phase 1 cohort. The patient was first dosed in February 2016, and after five weeks of treatment experienced a decrease in his brain lesions of approximately 58%, as estimated from radiology assessment, with accompanying clinical improvement.

Expansion of RXDX-105 Clinical Trial

In March 2016, the company announced the selection of an RP2D and the initiation of the Phase 1b portion of its Phase 1/1b clinical trial of RXDX-105, the company’s orally available, small molecule multikinase inhibitor with potent activity against such targets as RET and BRAF. The Phase 1b portion of the study utilizes a basket design focusing on patients with solid tumors that contain molecular alterations of RET or BRAF. In addition, based on clinical data seen to date, the company intends to build into the Phase 1b portion of the study one or more baskets to evaluate RXDX-105 in both squamous non-small cell lung cancer and non-small cell lung adenocarcinoma.

Taladegib Complete Response Outside of Basal Cell Carcinoma

In April 2016 at the AACR (Free AACR Whitepaper) Annual Meeting, the company reported the first clinical proof of concept for taladegib in a solid tumor type outside of advanced basal cell carcinoma, in a patient with medulloblastoma who had a complete response. The company further announced that it would be exploring medulloblastoma and other solid tumor types driven by hedgehog pathway alterations in a Phase 1b basket study, which is expected to be initiated in the third quarter of 2016.

Financing Transaction

In May 2016, the company issued an aggregate of 9.2 million shares of its common stock in an underwritten public offering at a purchase price of $6.25 per share, which resulted in aggregate gross proceeds of $57.5 million.

First Quarter 2016 Financial Results

For the first quarter of 2016, net loss was $25.5 million, or $0.79 per share, compared with $23.5 million, or $1.15 per share, for the first quarter of 2015.

Ignyta did not record any revenue for the three months ended March 31, 2016, or for the three months ended March 31, 2015.

Research and development expenses for the first quarter of 2016 were $19.8 million, compared with $20.2 million for the first quarter of 2015. During the first quarter of 2015, the company recorded an in-process research and development charge representing the net value of the assets exchanged for the intellectual property assets acquired from Teva, as well as related transaction and drug product costs. Excluding these costs, research and development costs would have increased in 2016 as compared to 2015 by $12.7 million, primarily due to an $8.0 million increase in the development costs associated with the company’s entrectinib, taladegib, and other product candidates. The remaining increase in costs between periods was due to personnel expenses related to hiring and engaging additional employees and consultants to help advance the company’s product candidates.

General and administrative expenses were $5.2 million for first quarter of 2016, compared with $2.8 million for first quarter of 2015. The increase was primarily caused by increases in personnel costs, as well as higher facilities-related expenses resulting from the expansion of the company’s leased facilities space. The increase was also attributable to increases in legal and intellectual property costs, consulting fees, and depreciation expenses.

At March 31, 2016, the company had cash, cash equivalents, and available-for-sale securities totaling $151.2 million and current and long-term debt of approximately $31.0 million. This balance does not include the gross proceeds of $57.5 million from the May 2016 underwritten public offering. At December 31, 2015, the company had cash, cash equivalents, and available-for-sale securities totaling $172.1 million and current and long-term debt of $31.0 million.

ChemoCentryx Reports First Quarter 2016 Financial Results and Provides Corporate Update

On May 10, 2016 ChemoCentryx, Inc., (Nasdaq:CCXI), a clinical-stage biopharmaceutical company developing orally-administered therapeutics to treat autoimmune diseases, inflammatory disorders and cancer, reported financial results for the first quarter ended March 31, 2016 and provided an update on the Company’s corporate and clinical development activities (Press release, ChemoCentryx, MAY 10, 2016, View Source [SID:1234512206]).

"2016 is proving to be a transformational year for ChemoCentryx," said Thomas J. Schall, Ph.D., President and Chief Executive Officer. "Following the positive results from the CLEAR trial with CCX168 in AAV that we reported in January, we continue to build considerable momentum. Indeed, today I am extremely pleased to announce that we have entered into a strategic regional license agreement with Vifor Pharma to commercialize CCX168 outside the U.S. and most of Asia. This partnership with Vifor Pharma, who will also work together with its nephrology partner company Vifor Fresenius Medical Care Renal Pharma, is an ideal alliance for the CCX168 program. Given Vifor Pharma’s deep experience and focus in the kidney care space, the formation of this partnership validates our approach, and the clinical data that we’ve obtained to date, in blocking chemoattractant receptors to treat patients with rare and other significant renal diseases."

Dr. Schall continued, "Important developments in the CCX168 program are ahead: we look forward to reporting top line data from our Phase II CLASSIC trial of CCX168 in patients with AAV this quarter, and we are preparing for regulatory discussions that we expect will pave the way for Phase III development of CCX168 by the end of this year. In our other clinical development programs, such as our clinical trial in pancreatic cancer with CCX872, we continue to chart excellent progress as well. We anticipate data on initial response rates, as well as progression-free survival data in the CCX872 treated patients as 2016 progresses. It’s truly an exciting time for the Company."

Pipeline Developments Across Key Therapeutic Areas

Orphan and Rare Diseases: CCX168 is an orally-administered complement inhibitor targeting the C5a receptor (C5aR), and is being developed for several rare disease indications, including ANCA-associated vasculitis (AAV) and atypical hemolytic uremic syndrome (aHUS). CCX168 acts by blocking the destructive action of neutrophils that are activated as a consequence of the complement protein known as C5a binding to C5aR on neutrophils during autoimmune inflammatory events including the destruction of blood vessels in AAV.

ChemoCentryx entered into an exclusive collaboration and license agreement with Vifor Pharma to commercialize CCX168 in certain licensed territories outside of the United States and Asia (except South Korea). Under the terms of the agreement, ChemoCentryx will:
Receive a non-refundable upfront payment of $85 million, comprising $60 million in cash in addition to $25 million in an equity investment to purchase ChemoCentryx common stock at a price of $7.50 per share;
Retain control of all ongoing and future development of CCX168, other than country-specific development in the licensed territories;
Retain all commercialization rights to CCX168 in the United States and other countries not licensed to Vifor Pharma;
Receive additional payments upon the achievement of certain regulatory and sales based milestones with CCX168; and
Receive tiered royalties with rates ranging from the teens to mid-twenties on potential net sales of CCX168 by Vifor Pharma in the licensed territories.
The agreement is the first step of a potentially broader kidney health alliance, beyond orphan and rare diseases, as it also provides Vifor Pharma with an exclusive option to negotiate during 2016, a worldwide license agreement for an additional ChemoCentryx drug candidate, CCX140, an orally administered inhibitor of the chemokine receptor known as CCR2 for use in diabetic nephropathy.
ChemoCentryx announced positive top-line data from the Phase II CLEAR trial with CCX168 in patients with AAV. Chronic high dose steroid administration in the current standard of care (SOC) is associated with premature death and a spectrum of other harmful side effects in AAV therapy. The objective of the CLEAR trial was to eliminate chronic high dose steroids and their associated significant safety issues including death, from the SOC regimen in AAV and replace steroids with CCX168.
The CLEAR trial met its primary endpoint based on the BVAS response at week 12 in patients receiving CCX168, compared to those patients receiving the high dose steroid-containing SOC. Specifically, all treatment groups receiving CCX168 demonstrated a numerically superior, statistically significant (P=0.002) non-inferior clinical efficacy outcome when compared to SOC.

Immuno-Oncology: CCX872 is a potent and selective inhibitor of the chemokine receptor known as CCR2, which is being evaluated in patients with non-resectable pancreatic cancer. In an ongoing, multi-center clinical trial with CCX872, 50 patients with non-resectable pancreatic cancer have been enrolled. In addition to evaluating objective response rate data after 12 weeks of treatment, the primary outcome measurement of this study is progression-free survival after at least 24 weeks of treatment. In addition, ChemoCentryx is conducting preclinical research with various chemokine receptor inhibitors, such as CCX9588, an inhibitor of the chemokine receptor known as CCR1, in combination with checkpoint inhibitors.

Presented combination data with check point (PD-L1) and chemokine receptor (CCX9588) inhibitors at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting showing synergistic effect with combination treatment in triple negative breast cancer models.
Combination treatment was shown to significantly decrease circulating and tumor infiltrating granulocytic myeloid-derived suppressor cells which are known to be responsible for the induction of a metastatic phenotype in primary tumors, leading to the early dissemination of cancer cells.
Overall tumor size and progression was also significantly reduced by the combination treatment.

Anticipated Milestones

Orphan and Rare Diseases:

Oral presentation of results from the Phase II CLEAR trial in patients with AAV treated with CCX168 at the 53rd ERA-EDTA Congress;
Report top-line results from the Phase II CLASSIC trial in patients with AAV in North America with CCX168 in June 2016;
Conduct End of Phase II meetings with regulatory agencies to review CLEAR and CLASSIC AAV Phase II clinical results and discuss the potential Phase III plan in mid-2016;
Initiate Phase III development program with CCX168 for the treatment of AAV by the end of 2016;
Report early results from the Phase II pilot study of CCX168 in aHUS patients who are on dialysis in 2016; and
Report results from preclinical model using CRISPR-CAS9 designed to assess the contribution of C5a on two aspects of complement over-activation which can manifest as renal damage in aHUS and complement factor 3 glomerulopathy at the 53rd ERA-EDTA Congress.

Immuno-Oncology:

Advance pancreatic cancer trial of CCX872 in combination with FOLFIRINOX; report initial overall response data in mid-2016 and initial progression free survival data in the second half of 2016.

Chronic Kidney Disease:

The recently announced Vifor Pharma agreement for CCX168 also provides Vifor Pharma with an exclusive option to negotiate in 2016, a worldwide license agreement for CCX140; and
Conduct End of Phase II meeting with the FDA in 2016 to review the Phase II data and discuss the potential Phase III clinical development program for CCX140 in diabetic nephropathy.

First Quarter 2016 Financial Results and Outlook

Cash, cash equivalents and investments totaled $65.3 million at March 31, 2016, excluding the $85.0 million upfront payment in connection with the partnership with Vifor Pharma announced earlier today.

Research and development expenses were $11.2 million for the three months ended March 31, 2016 compared to $8.4 million reported for the same period in 2015. The increase in research and development expense from 2015 to 2016 was primarily attributable to higher costs associated with CCX168, the Company’s C5aR inhibitor, due to the completion of ancillary Phase I studies to support anticipated end of Phase II meetings with regulatory agencies and higher expenses associated with CCX872, the Company’s second CCR2 inhibitor, for the ongoing pancreatic cancer trial.

General and administrative expenses were $4.1 million for the three months ended March 31, 2016 compared to $3.7 million for the comparable period in 2015. The increase from 2015 to 2016 was primarily due to increases in intellectual property related expenses and travel and professional fees associated with our business development efforts.

Net loss was $15.2 million for the first quarter ended March 31, 2016 compared to $12.0 million in the same period in 2015.

Total shares outstanding at March 31, 2016 were approximately 44.3 million shares.

About ANCA-Associated Vasculitis and Other Rare Renal Diseases

Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, or AAV, is a type of rare autoimmune inflammation caused by auto-antibodies. AAV encompasses granulomatosis with polyangiitis (GPA, formerly known as Wegener’s granulomatosis), microscopic polyangiitis (MPA), eosinophilic polyangiitis (formerly Churg-Strauss syndrome) and renal limited vasculitis.

AAV represents a severe and often fatal autoimmune disease that is characterized by inflammation that can destroy different organ systems. AAV is the lead indication in the Company’s orphan and rare disease program which has the objective of eliminating chronic high dose steroids, which are associated with significant safety issues including death, from the standard of care (SOC) regimen in AAV and replace steroids with CCX168.

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

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

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

Atypical hemolytic uremic syndrome, or aHUS, an ultra-rare, life threatening disease that causes chronic blood vessel damage, thrombosis or clotting within blood vessels, hemolysis or red blood cell rupture, and sudden, progressive organ failure, such as kidney failure. The disease is caused by genetic defects in factors that control the activation of the complement system. Current treatment options are still quite limited and prognosis and quality of life are extremely poor.

About Pancreatic Cancer

It is estimated that over 337,000 cases of pancreatic cancer are diagnosed worldwide every year, accounting for 2.4 percent of all cancers. The incidence of pancreatic cancer in the U.S. is about 45,000, with prevalence being only negligibly higher owing to the poor survival rates on current therapy. Current standards of care include surgical resection and chemotherapeutic regimens such as gemcitabine and FOLFIRINOX. These regimens are limited by marked toxicities. Almost 67 percent of cases are diagnosed in people aged 65 and over. In the U.S., pancreatic cancer is the fourth most common cause of deaths due to cancer. Pancreatic cancer has a low survival rate regardless of stage of disease, with 93 percent of patients dying from their disease within five years.

Celator® Pharmaceuticals Announces First Quarter 2016 Financial Results and Business Update

On May 10, 2016 Celator Pharmaceuticals, Inc. (Nasdaq: CPXX) reported business highlights and financial results for the first quarter ended March 31, 2016 (Press release, Celator Pharmaceuticals, MAY 10, 2016, View Source [SID:1234512203]).

"The first quarter of 2016 was transformative for Celator," said Scott Jackson, chief executive officer of Celator. "Our Phase 3 trial of VYXEOS demonstrated a statistically significant improvement in overall survival, among other benefits, in patients with high-risk acute myeloid leukemia (AML) and we plan to submit a New Drug Application (NDA) to the Food and Drug Administration (FDA) by the end of the third quarter of this year. We look forward to presenting additional data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting next month. In addition, following the positive Phase 3 results, we raised capital that we believe is sufficient to fund planned operations into 2018."

Celator anticipates achieving the following key objectives:

Continue to execute on the VYXEOS commercial readiness in the United States;
Submit the New Drug Application (NDA) for VYXEOS by the end of Q3 2016;
Continue to expand the clinical development of VYXEOS via investigator-initiated studies, oncology cooperative groups and company-sponsored studies;
Prescription Drug User Fee Act (PDUFA) date (assuming FDA grants priority review) by mid-2017; and
United States commercial launch (assuming FDA approval in mid-2017) in Q3 2017.
Recent Business and First Quarter 2016 Highlights

April – Celator announced that a late-breaking abstract was submitted to ASCO (Free ASCO Whitepaper) and accepted for an oral presentation on June 4, 2016 at 3:00 pm CT and will be presented by Dr. Jeffrey Lancet from H. Lee Moffitt Cancer Center & Research Institute.
March – Celator announced that Phase 3 trial data for VYXEOS in patients with high-risk AML demonstrated a statistically significant improvement in overall survival and induction response rate and meaningfully lower 60-day mortality. There was no substantial difference in Grade 3-5 adverse events between VYXEOS and the control arm (7+3 regimen). The company plans to submit an NDA for VYXEOS by the end of the third quarter.
March – Celator completed an underwritten public offering of 4,600,000 shares of the company’s common stock, resulting in net proceeds of approximately $40.6 million.
Financial Highlights

Cash Position: Cash and cash equivalents as of March 31, 2016 were $67.5 million, compared to $23.3 million as of December 31, 2015. The increase was primarily due to the underwritten public offering that netted approximately $40.6 million during the first quarter of 2016 and $9.8 million in net proceeds from the issuance of common stock through an at-the-market equity offering program. Management believes that the cash and cash equivalents at March 31, 2016 will be sufficient to meet estimated working capital requirements and fund planned operations into 2018.
R&D Expenses: Research and development expenses were $2.7 million for the three months ended March 31, 2016 consistent with the same period in 2015.
G&A Expenses: General and administrative expenses were $2.5 million for the three months ended March 31, 2016, as compared to $1.8 million for the same period of 2015. The increase was primarily attributable to increases in compensation, pre-launch commercial activities, public company expenses and professional fees.
Net Loss: Net loss increased to $5.5 million for the three months ended March 31, 2016, from $4.7 million for the same period in 2015.

AVEO Oncology Reports First Quarter 2016 Financial Results and Provides Business Update

On May 10, 2016 AVEO Oncology (NASDAQ:AVEO) reported financial results for the first quarter ended March 31, 2016 (Press release, AVEO, MAY 10, 2016, View Source;p=RssLanding&cat=news&id=2167104 [SID:1234512200]).

"We achieved several important goals in the first quarter critical to advancing the corporate strategy we outlined early in 2015. This includes the submission and validation of an MAA filing in Europe for tivozanib in front-line RCC by our partner EUSA Pharma and the licensing of AV-203 outside of North America by CANbridge Life Sciences. In addition, we also announced the filing of provisional patent applications and the initiation of partnership discussions for AV-353, a legacy discovery program which we will look to develop and commercialize in PAH through a global partnership." said Michael Bailey, president and chief executive officer. "These accomplishments, coupled with our noteworthy progress in 2015, have allowed us to retain significant North American rights to develop our three oncology-focused clinical programs, while positioning five programs to be advanced by partners. We believe these initiatives have the potential to unlock significant future value for patients as well as our shareholders."

Mr. Bailey concluded: "Our next steps in the development of tivozanib in North America include the potential initiation of TIVO-3, a Phase 3 U.S. pivotal study of tivozanib designed to support a first- and third-line indication in renal cell cancer (RCC), and a tivozanib-PD1 combination study in RCC. The Company continues to work toward the potential initiation of patient enrollment in the TIVO-3 Study in the second quarter of 2016."

Recent Highlights

Filing of Provisional Patent Applications for AV-353, a Notch 3-Specific Inhibitor Antibody for PAH. In May 2016, AVEO announced that it had filed provisional patent applications with the United States Patent and Trademark Office covering composition of matter claims for AV-353, the Company’s potent inhibitory antibody specific to Notch 3 for development in Pulmonary Arterial Hypertension (PAH). These patent applications are the second set of applications related to AV-353 and the Company’s Notch 3 antibody program. Current treatments in PAH focus only on controlling symptoms by avoiding vasoconstriction and increasing vasodilation of vessels and do not reverse the underlying cause of the disease. In contrast, with the results of a recently concluded research study supported by AVEO, AV-353 has generated a growing body of preclinical data that supports AV-353’s ability to potentially reverse the disease phenotype, which would represent a potential disease-modifying approach to treatment. Consistent with the Company’s focus on developing oncology therapeutics, AVEO is currently seeking an appropriate partner to develop and commercialize AV-353 worldwide in PAH.
Exclusive Licensing Agreement for AV-203 Outside of North America with CANbridge Life Sciences. In March 2016 AVEO and CANbridge Life Sciences announced an exclusive collaboration and license agreement in which AVEO has granted CANbridge worldwide rights, excluding the United States, Canada, and Mexico, to AV-203, AVEO’s clinical-stage ErbB3 (HER3) inhibitory antibody candidate. CANbridge plans to develop AV-203 first in esophageal squamous cell cancer (ESCC). Under the terms of the agreement, CANbridge is obligated to pay AVEO an upfront payment of $1 million plus up to $133 million in potential reimbursement and milestone payments, assuming the successful achievement of specified development, regulatory and commercialization objectives. AVEO is also eligible for a tiered royalty, with a percentage range in the low double digits, on net sales of AV-203 in CANbridge’s territories. CANbridge will be responsible for costs associated with the execution of a development plan that includes additional manufacturing requirements as well as pre-clinical and clinical studies necessary to demonstrate proof-of-concept for AV-203 as a treatment for ESCC, including a Phase IIa proof-of-concept study meeting mutually agreed upon criteria. Following completion of the proof-of-concept studies, AVEO and CANbridge will negotiate a possible agreement under which the parties may co-develop AV-203, with each party bearing a percentage of the cost of global development activities based on respective geographic rights.
Submission and validation of a European Marketing Authorization Application for Tivozanib in Renal Cell Carcinoma. In February 2016, AVEO and its European partner, EUSA Pharma announced that EUSA Pharma submitted and received a validation notice for the Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for tivozanib as a first line treatment for renal cell carcinoma (RCC).
Acceptance of Registration Dossier for Tivozanib in RCC by the Ministry of Health of the Russian Federation. In February 2016, AVEO announced that a registration dossier seeking to obtain marketing authorization of tivozanib as a first line treatment of advanced RCC has been accepted by the Ministry of Health of the Russian Federation. The dossier was submitted in December 2015 by Pharmstandard Group, AVEO’s licensing partner in Russia, Ukraine and CIS.
Receipt of $3.5 Million AV-380 Inventory Reimbursement Payment from Novartis. AVEO previously announced that Novartis exercised its right under its license agreement for AV-380, AVEO’s first-in-class, potent, humanized inhibitory antibody targeting growth differentiation factor 15 (GDF15), to acquire AVEO’s inventory of clinical quality drug substance. This reimbursement payment of approximately $3.5 million was received in the first quarter of 2016.
First Quarter 2016 Financial Highlights

AVEO ended Q1 2016 with $23.8 million in cash, cash equivalents and marketable securities. The reduction in cash over base operations was primarily attributable to clinical trial startup costs related to the TIVO-3 study and a significant pay down in accounts payable quarter over quarter.
Total collaboration revenue in Q1 2016 was approximately $1.2 million compared with $0.1 million for Q1 2015. The increase was primarily due to an additional $1.0 million in revenue recognized in the first quarter of 2016 in connection with our out-licensing agreement with CANbridge, which was executed in March 2016.
Research and development (R&D) expense was $6.0 million in Q1 2016 compared with $2.7 million for Q1 2015. The increase was primarily attributable to an increase in tivozanib clinical trial costs associated with our preparation for a planned Phase 3 trial.
General and administrative (G&A) expense was $2.5 million in Q1 2016 compared with $3.3 million for Q1 2015. The decrease was primarily the result of a decrease in external legal costs associated with various ongoing legal matters, and a decrease in employee compensation, consulting, facilities and IT costs as a result of our decreased headcount and the reduction of our utilized facility space following our January 2015 restructuring.
There was no restructuring and lease exit expense in Q1 2016, compared with $4.3 million for Q1 2015. The expenses incurred during the three months ended March 31, 2015 related to the January 2015 restructuring, which was substantially completed in March 2015.
Net loss for Q1 2016 was $7.7 million, or a loss of $0.13 per basic and diluted share, compared with net loss of $10.9 million, or a loss of $0.21 per basic and diluted share for Q1 2015.
Financial Guidance

AVEO believe that its cash resources would allow the Company to fund its current operations into the fourth quarter of 2017. This estimate does not include the payment of potential licensing milestones to third parties or the uncommitted costs of conducting any contemplated clinical trials (such as a second phase 3 trial and PD-1 combination trial for tivozanib in RCC), and assumes no milestone payments from our partners, no additional funding from new partnership agreements, no equity financings, no debt financings, no accelerated repayment thereof and no further sales of equity under our ATM.

Tokai Pharmaceuticals Reports First Quarter 2016 Financial Results

On May 10, 2016 Tokai Pharmaceuticals, Inc. (NASDAQ: TKAI), a biopharmaceutical company focused on developing and commercializing innovative therapies for prostate cancer and other hormonally driven diseases, reported company highlights and financial results for the quarter ended March 31, 2016 (Press release, Tokai Pharmaceuticals, MAY 10, 2016, View Source;p=RssLanding&cat=news&id=2166824 [SID:1234512193]).

"Over the past quarter we have made significant progress in our clinical development program for galeterone, as we continued to accelerate screening and enrollment in our ARMOR3-SV trial and expanded our Phase 2 trial to include additional mCRPC patients who have acquired resistance to enzalutamide," said Jodie Morrison, President and Chief Executive Officer of Tokai. "We have also strengthened our development team to sharpen focus on execution of our clinical trials, and we look forward to continued progress in the months ahead as we work with great urgency to meet the needs of patients who currently have limited therapeutic options."

Recent business highlights include:

Progress in ARMOR3-SV, a Phase 3 registration trial of galeterone in AR-V7+ mCRPC. Patient enrollment is ongoing in ARMOR3-SV, Tokai’s pivotal Phase 3 clinical trial evaluating whether administration of galeterone results in a statistically significant and clinically meaningful improvement in radiographic progression-free survival as compared to Xtandi (enzalutamide) in treatment-naïve metastatic castration-resistant prostate cancer (mCRPC) patients whose prostate tumor cells express the AR-V7 splice variant. AR-V7 is a truncated form of the androgen receptor that has been associated with poor response to commonly-used oral therapies for mCRPC. Over 100 clinical sites in eight countries are actively screening patients for potential eligibility to participate in ARMOR3-SV. Enrollment in ARMOR3-SV is expected to be completed by the end of 2016, and top-line data from the trial are anticipated by mid-2017.
Expansion of galeterone clinical development program. In March, patient dosing began in an expansion of the ongoing Phase 2 clinical trial of galeterone (ARMOR2) in mCRPC patients who have developed acquired resistance to enzalutamide. This expansion follows results observed in a patient who, following an initial response to enzalutamide, experienced a PSA drop of over 90 percent when treated with galeterone. This patient’s PSA response has remained at less than 0.1µg/L for over a year.
Strengthening of development team. Tokai enhanced its development capabilities with the addition of Kelly A. Lindert, M.D., as Executive Vice President and Head of Development responsible for the company’s clinical development, medical affairs, pharmacovigilance, regulatory affairs and quality assurance activities.
Financial Results

Cash and investments at March 31, 2016 were $54.3 million, as compared to $64.0 million at December 31, 2015.
Research and development expense for the quarter ended March 31, 2016 was $7.9 million, as compared to $10.6 million for the quarter ended March 31, 2015. Research and development expense in the first quarter of 2015 included a one-time fee paid to Qiagen Manchester Limited to access rights to its proprietary circulating tumor cell technology for use in the AR-V7 clinical trial assay. The decrease in research and development expense was also attributable to decreased manufacturing costs during the first quarter of 2016, partially offset by an increase in clinical trial costs associated with ARMOR3-SV.
General and administrative expense for the quarter ended March 31, 2016 was $3.5 million, as compared to $2.7 million for the quarter ended March 31, 2015. The increase in general and administrative expense was primarily attributable to increased headcount associated with operating as a public company, including related stock-based compensation expense.
Net loss was $11.4 million for the quarter ended March 31, 2016, or $0.51 per share, as compared to $13.3 million for the quarter ended March 31, 2015, or $0.59 per share.