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Tetraphase Pharmaceuticals Reports Second Quarter 2016 Financial Results and Highlights Recent Progress

On August 4, 2016 Tetraphase Pharmaceuticals, Inc. (NASDAQ:TTPH), a clinical stage biopharmaceutical company developing novel antibiotics to treat life-threatening multidrug-resistant (MDR) infections, reported financial results for the second quarter ended June 30, 2016, and provided an overview of certain corporate achievements and plans (Press release, Tetraphase, AUG 4, 2016, View Source [SID:1234514266]).

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"During the second quarter, we finalized the protocol for IGNITE4, our upcoming phase 3 clinical trial evaluating IV eravacycline in patients with complicated intra-abdominal infections (cIAI), and remain on track to initiate this trial early in the fourth quarter of 2016," said Guy Macdonald, Tetraphase’s President and Chief Executive Officer. "Based on discussions with the FDA, we have designed IGNITE4 using a 12.5% non-inferiority margin and a trial population of approximately 450 patients. We currently anticipate reporting top-line results from IGNITE4 as early as the fourth quarter of 2017. In parallel, we are also working to finalize the trial design for IGNITE3, our phase 3 clinical trial evaluating once-daily IV eravacycline in complicated urinary tract infections (cUTI), and we look forward to providing details regarding the design and timing of IGNITE3 once the protocol is completed."

Dr. Patrick Horn, Tetraphase’s Chief Medical Officer, commented, "Separately, we continue to advance development work on the oral formulation of eravacycline. Early data from this phase 1 program indicate that the oral dosing regimen used in IGNITE2 leads to lower systemic levels of eravacycline than expected. These data also suggest that administration of oral eravacycline in a fasted state results in increased drug exposure. With this information now in hand, we have commenced further clinical testing designed to evaluate several additional variables associated with optimizing the oral eravacycline dosing regimen."

"Along with the advancement of our clinical programs, we continue to build the profile of eravacycline by testing it against multidrug-resistant bacterial strains in vitro and, at ASM Microbe and ECCMID we presented data showing eravacycline’s potent activity against these difficult-to-treat pathogens, including carbapenem-resistant enterobacteriaceae and Acinetobacter baumannii," added Dr. Horn. "We also recently confirmed that in in vitro studies eravacycline retained its potent activity against colistin-resistant clinical isolates expressing the mcr-1 gene, which was just found in bacteria in the U.S. for the first time, and we look forward to presenting these data at an upcoming medical meeting."

Second Quarter 2016 Financial Results

As of June 30, 2016, Tetraphase had cash and cash equivalents of $178.3 million and 36.7 million shares outstanding. The company expects that its cash and cash equivalents, as well as expected revenue from its U.S. government awards, will be sufficient to fund operations into the middle of 2018.

Revenues during the second quarter of 2016 were $1.2 million compared to $3.3 million for the same period in 2015. Revenues for each period consisted of contract and grant revenue under the Company’s U.S. government awards for the development of Tetraphase compounds for the treatment of diseases caused by bacterial biothreat pathogens and for certain infections caused by life-threatening multidrug-resistant bacteria. The decrease in revenues was primarily due to the scope and timing of activities related to our BARDA Contract conducted during the quarter ended June 30, 2016.

Research and development (R&D) expenses for the second quarter of 2016 were $13.7 million compared to $22.9 million for the same period in 2015. The decrease in R&D expenses was primarily due to lower costs related to our Phase 3 clinical program for eravacycline.

General and administrative (G&A) expenses for the second quarter of 2016 were $4.8 million compared to $6.5 million for the same period in 2015. The decrease in G&A expenses was primarily due to a decrease in stock-based compensation expense, as well as a decrease in pre-commercialization activities for eravacycline.

For the second quarter of 2016, Tetraphase reported a net loss of $17.2 million, or $0.47 per share, compared to a net loss of $26.0 million, or $0.72 per share, for the same period in 2015.

Second Quarter and Recent Corporate Highlights

Presented data at the 26th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID), including preclinical data for eravacycline and TP-6076.

Received guidance from the U.S. FDA confirming that one additional positive phase 3 clinical trial will be required to support a New Drug Application (NDA) submission for IV eravacycline. Based on the FDA’s guidance, Tetraphase plans to conduct two additional pivotal phase 3 clinical trials: one in cIAI (IGNITE4) to support the NDA filing and one in cUTI (IGNITE3) that will form the basis of a supplemental NDA for IV eravacycline.

Finalized the clinical trial design for IGNITE4 to support an NDA filing for IV eravacycline for cIAI. IGNITE4, the Company’s planned phase 3 clinical trial evaluating the efficacy and safety of twice-daily IV eravacycline compared to meropenem in patients with cIAI, is expected to enroll approximately 450 patients and the primary analysis will be conducted using a 12.5% non-inferiority margin. Tetraphase expects to initiate this clinical trial early in the fourth quarter of 2016, with top-line results expected as early as the fourth quarter of 2017.

Presented data at the American Society of Microbiology (ASM) Microbe 2016 Conference, including clinical and preclinical data for eravacycline and preclinical data for TP-271, a novel, broad-spectrum antibiotic candidate which is being developed to combat respiratory disease caused by bacterial biothreats and antibiotic-resistant public health pathogens, as well as bacterial pathogens associated with community-acquired bacterial pneumonia.

Commenced patient dosing in a phase 1 clinical trial for TP-6076, the lead candidate from the Company’s second-generation antibiotic program which has demonstrated potent preclinical activity against multidrug-resistant Gram-negative pathogens.
Continued clinical testing designed to advance the development of an oral dose formulation of eravacycline. During the second quarter, Tetraphase completed preliminary clinical testing which indicates that the overall efficacy results in IGNITE2 were likely driven by underperformance of the oral formulation due to a food effect. Preliminary clinical testing also suggests that administration of oral eravacycline in a fasted state results in increased drug exposure. Further clinical testing is now underway to evaluate several additional variables associated with increasing drug exposure and optimizing the oral eravacycline dosing regimen.
Added to the Russell Microcap Index when the Russell Investment Group reconstituted its family of U.S. indexes in late June 2016.

OncoGenex Pharmaceuticals, Inc. Reports Financial Results for Second Quarter 2016

On August 4, 2016 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported its second quarter 2016 financial results and provided a summary of anticipated milestones (Press release, OncoGenex Pharmaceuticals, AUG 4, 2016, View Source [SID:1234514258]).

Financial Results and Anticipated Near-term Milestones

As of June 30, 2016, the company’s cash, cash equivalents, and short-term investments were $39.7 million compared with $55.2 million as of December 31, 2015.

Based on current expectations, OncoGenex believes that its cash, cash equivalents, and short-term investments will be sufficient to fund its currently planned operations into the third quarter of 2017. Depending on timing of enrollment or event-driven final analyses, the expected key milestones and activities are as follows:

Custirsen
Announcing results from the AFFINITY trial, the phase 3 trial evaluating a survival benefit for custirsen in combination with cabazitaxel as second-line chemotherapy in approximately 630 patients with castrate-resistant prostate cancer. The final analysis for the intent-to-treat population is expected in the third quarter of 2016.
Announcing results from the ENSPIRIT trial, the phase 3 trial evaluating a survival benefit for custirsen in combination with docetaxel as second-line chemotherapy in approximately 700 patients with non-small cell lung cancer. The final survival analysis is expected by the first half of 2017.
Apatorsen
Announcing results from the Borealis-2 trial, an investigator-sponsored, randomized phase 2 trial evaluating apatorsen in combination with docetaxel treatment compared to docetaxel treatment alone in patients with advanced or metastatic bladder cancer. Final results are expected in the fourth quarter of 2016.
Completing a submission-ready investigational new drug application regarding apatorsen via intravesical administration in combination with Bacillus Calmette-Guerin (BCG) treatment in patients with non-muscle invasive bladder cancer.
Revenue for the three and six months ended June 30, 2016 was $2.1 million and $5.1 million, respectively, compared to $4.0 million and $5.4 million for the three and six months ended June 30, 2015, respectively. Revenue consists of recognition of deferred collaboration revenue representing our efforts in the development of custirsen. As of June 30, 2016, the full amount of the deferred collaboration revenue has been fully recognized.

Total operating expenses for the three and six months ended June 30, 2016 were $8.5 million and $15.9 million, respectively, compared to $9.6 million and $16.0 million for the three and six months ended June 30, 2015, respectively. Net loss for the three and six months ended June 30, 2016 was $6.9 million and $10.6 million, respectively, compared to $6.0 million and $10.5 million for the three and six months ended June 30, 2015, respectively.

As of Aug. 4, 2016 OncoGenex had 30,009,730 shares outstanding.

Navidea Reports Second Quarter 2016 Financial Results

On August 4, 2016 Navidea Biopharmaceuticals, Inc. (NYSE MKT:NAVB), reported financial results for the second quarter of 2016. Navidea reported total revenue for the second quarter of 2016 of $5.4 million, including Lymphoseek (technetium Tc 99m tilmanocept) injection sales revenue of $4.2 million (Press release, Navidea Biopharmaceuticals, AUG 4, 2016, View Source;p=RssLanding&cat=news&id=2192878 [SID:1234514255]). The net loss from operations was $580,000 and the net loss attributable to common stockholders was $6.7 million.

"Despite the significant disruption in our organization during the first half of 2016 caused by legal and financial challenges, we remain committed to advancing our Lymphoseek commercial efforts, expanding our Manocept platform to other larger immunodiagnostic and immunotherapeutic indications, and controlling our operating expenses" said Jed Latkin, interim Chief Operating Officer and Chief Financial Officer at Navidea. "Despite the disruptions caused by CRG’s (Capital Royalty Partners II L.P.) actions we are confident that the technology we are developing and our lead commercial product, Lymphoseek, provide Navidea and its shareholders with significant unrealized value. Given the advanced state of our technology, continued growth of Lymphoseek in the U.S. and the impending launch in Europe, we believe we will be successful in seeking a replacement financing arrangement for the CRG debt. We believe we have significant claims for damages against CRG that we intend to pursue. Finally our Macrophage Therapeutics subsidiary has made great strides towards demonstrating the breadth of the technology’s potential to develop innovative immunotherapies."

Specific events and milestones achieved since the beginning of the second quarter include the following:

Commercial

Achieved sequential quarter-on-quarter Lymphoseek revenue growth of 12% and continued improvement in key performance indicators;
Reported investigator-initiated study results demonstrating beneficial performance characteristics of Lymphoseek and positive comparative results versus commonly-used, non-receptor-targeted imaging agents in breast cancer presented by Emory University School of Medicine and University of California San Diego (UCSD) at the 2016 Society of Nuclear Medicine and Molecular Imaging annual meeting; and
Continued to progress our development efforts to meet the projected Q4 launch of Lymphoseek in Europe by our partner, Norgine BV.
Lymphoseek Lifecycle Management

Continued market development clinical activities with Navidea’s and investigator-initiated studies in cervical cancer, pediatric solid tumors, anal-rectal cancer, endometrial cancer, and for further confirmation of workflow efficiency compared to sulfur colloid, which are supported in large part by National Institutes of Health (NIH) grant funding; and
Received Western Institutional Review Board (WIRB) approval of several Lymphoseek investigational protocols including anal-rectal cancer sentinel lymph node detection, imaging in Kaposi sarcoma (KS) and intravenous (IV) administration in rheumatoid arthritis (RA).
Immunodiagnostic & Immunotherapeutic Development Pipeline

Rheumatoid Arthritis Immunodiagnostic Indication
Received IRB approval at University of California, San Francisco and from WIRB for the RA subcutaneous administration clinical trial protocol;
Expect patient enrollment in the subcutaneous injection trial to begin shortly;
Awarded Part 2 grant funding of $1.1 million from our previously announced RA grant;
Completed cardiovascular disease imaging study with Massachusetts General Hospital, manuscripts being prepared for publication;
Expect to begin grant-funded Phase 1/2 evaluation of Lymphoseek – IV in KS patients in the second half of 2016;
Received $1.8 million grant to support the development of Manocept immunotherapeutic program in KS; and
Successfully completed a number of preclinical studies of Manocept in animal models of nonalcoholic steatohepatitis (NASH), arthritis, asthma, neuro-inflammation and tumor-associated macrophage (TAM) depletion in two cancer models. Both MT1000 class and MT2000 class molecules demonstrated their predicted activity in animal testing.
Operational & Financial

Reduced cash used in operations by over 87% for the first half of 2016 compared to the first half of 2015; and
Continued partnering/divestiture efforts for the Company’s investigational imaging agent, NAV4694, for the detection of amyloid plaques in Alzheimer’s disease.
CRG litigation update

As previously reported, on April 7, 2016, Navidea received a notice from CRG pursuant to the Term Loan Agreement, dated May 8, 2015 which claimed that certain Events of Default, unrelated to repayment terms, had occurred under the Loan Agreement. CRG commenced a state court action in Harris County, Texas District Court against the Company on that same date. By letter dated May 31, 2016, CRG declared all of the Company’s obligations under the Loan Agreement and all other loan documents to be immediately due and payable in the amount of $56,157,240.69. The Company disputes the total amount claimed to be due and owing, and contends CRG’s acceleration of the maturity of the loan was improper. On July 13, 2016, a hearing was held in the District Court of Harris County, Texas with respect to CRG’s application for a temporary injunction seeking to restrain Navidea from operating or using new accounts without having first entered into a blocked account control and/or pledge collateral account control agreement with CRG for any such new account. At the conclusion of the temporary injunction hearing, the Court ordered the parties to mediation and stayed any ruling on CRG’s request for injunctive relief until after mediation has been completed. The parties participated in a mediation on July 20, 2016, but did not reach a settlement. The district judge in the Texas case has since recused herself from the case, and the case has been reassigned to a different Harris County District Court. The district court did not issue a ruling on the application for temporary injunction prior to the judge’s recusal from the case, and no hearing or other matter is currently set in the Texas court case.

Concurrently with the Texas court case, CRG previously sent a notice to Cardinal Health demanding that all monies owing to Navidea be sent directly to CRG. In response, Cardinal filed an interpleader action in Ohio, pursuant to which the Ohio court initially ruled that 50% of the monies should be sent to Navidea, and 50% should be placed into the registry of the Court pending a determination of the parties’ rights to the funds. The court has since ruled that 75% of the Cardinal payments should be sent to Navidea and 25% should be deposited into the registry of the court up until the deposit in the registry of the court equals $1 million, which will serve as a bond pending a determination on the merits of the case, and then Navidea will receive 100% of the Cardinal payments pending further order by the Court in the Ohio case.

The Company reiterates its firmly-held position that the alleged claims by CRG do not constitute Events of Default under the Loan Agreement and will vigorously defend against such claims. The Company also contends CRG’s wrongful conduct has caused harm to the Company and it will pursue its counterclaims against CRG seeking all remedies and other relief it may be entitled to under the law.

The Company is also continuing to explore alternative financing arrangements in order to refinance the CRG debt. The Company believes that the actions of CRG are a violation of the Loan Agreement and, as a result, CRG is in breach of the Loan Agreement, not the Company. The Company believes that its best course of action is to refinance the CRG debt and pursue its claims for damages.

Financials

Total revenues for the quarter ended June 30, 2016 were $5.4 million compared to $2.9 million in the second quarter of last year. Second quarter 2016 product revenues recognized from the sale of Lymphoseek were $4.2 million, compared to $3.8 million in the first quarter of 2016 and $2.0 million in the second quarter of 2015. During the second quarter of 2016, the Company also reported $1.2 million in grant, licensing and other revenue. For the six months ended June 30, 2016, Navidea’s total revenue was $10.1 million compared to $5.0 million for the same period in 2015, an increase of 103%. The primary driver of this increase was revenues recognized from the sale of Lymphoseek which exceeded $8.0 million for the six months ended June 30, 2016 compared to $3.8 million for the same period last year.

Gross margins on Lymphoseek product sales grew to 87% for the second quarter of 2016 compared to 83% for the second quarter of 2015, primarily due to inventory written off in 2015 related to a production issue.

Research and development (R&D) expenses for the second quarter of 2016 were $2.5 million, compared to $2.3 million in the second quarter of last year. R&D expenses were $5.2 million for the six months ended June 30, 2016 compared to $6.3 million in the same period of 2015. The net decreases in year-to-date R&D expenses were primarily a result of decreased headcount costs coupled with decreased project costs related to the Company’s neuro assets, offset by increased project costs related to the Company’s Manocept and Lymphoseek programs. Selling, general and administrative (SG&A) expenses for the second quarter of 2016 were $2.9 million, compared to $4.0 million in the second quarter of last year. SG&A expenses were $7.0 million for the six months ended June 30, 2016, compared to $9.5 million for the same period in 2015. The net decrease in year-to-date SG&A expenses was due primarily to decreased headcount coupled with decreased costs related to contracted medical science liaisons, commercialization costs for Lymphoseek and NAV4694 and license fees, offset by increases in commercial headcount costs related to the addition of our internal sales force coupled with increased legal and professional services. Total operating expenses were $5.4 million for the second quarter of 2016, compared to $6.3 million in the second quarter of last year. Operating expenses were $12.2 million for the six months ended June 30, 2016, compared to $15.8 million for the same period in 2015.

Navidea’s net loss from operations for the quarter ended June 30, 2016 was $580,000 compared to $3.8 million for the same period in 2015. For the six months ended June 30, 2016, Navidea’s net loss from operations was $3.1 million compared to a net loss from operations of $11.6 million for the same period in 2015. Navidea’s net loss attributable to common stockholders for the quarter ended June 30, 2016 was $6.7 million, or $0.04 per share, compared to $9.7 million, or $0.06 per share, for the same period in 2015. For the six months ended June 30, 2016, Navidea’s net loss attributable to common stockholders was $10.4 million, or $0.07 per share, compared to a net loss attributable to common stockholders of $17.0 million, or $0.11 per share, for the same period in 2015. Net losses attributable to common stockholders include fees paid to CRG (which the Company is disputing in court), the interest expense on our outstanding debt, as well as significant non-cash charges. For the six-month periods ended June 30, 2016 and June 30, 2015, net loss attributable to common stockholders included $7.2 million and $5.4 million, respectively, in interest, debt-related fees, losses on extinguishment of debt, and changes in the fair value of financial instruments.

Navidea ended the quarter with $1.7 million in cash, $501,000 of which was restricted related to the CRG debt.

Intellia Therapeutics Reports Financial Results for Second Quarter 2016

On August 4, 2016 Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, reported financial results and recent company highlights for the quarter ended June 30, 2016 (Press release, Intellia Therapeutics, AUG 4, 2016, View Source;p=RssLanding&cat=news&id=2193255 [SID:1234514254]).

"Intellia has made substantial progress with our science, financing and operations in the first half of 2016," said Nessan Bermingham, Ph.D., Chief Executive Officer and Founder, Intellia Therapeutics. "Our product focus, therapeutic discovery and development strength, delivery expertise and intellectual property portfolio make Intellia well positioned to advance CRISPR/Cas9 into clinically meaningful genome editing therapeutics for patients with severe and life-threatening diseases."

Recent Highlights

On April 11, 2016, Intellia signed a multi-year research and development collaboration and licensing agreement with Regeneron Pharmaceuticals to advance CRISPR/Cas9 genome editing technology for in vivo therapeutic development. Regeneron has the exclusive rights to discover and develop CRISPR-based products against up to 10 targets, focused primarily on therapies for a broad range of diseases that may be treated by editing genes in the liver. Transthyretin amyloidosis (TTR) is the first target to be jointly developed and potentially commercialized by the companies.
The Company also strengthened its leadership team with the addition of Perry Karsen as the Chairman of Intellia’s Board of Directors. Mr. Karsen brings decades of biopharmaceutical leadership experience to his role as Chairman. He most recently held senior leadership positions at Celgene Corporation, including Chief Operations Officer and Executive Vice President as well as Chief Executive Officer of Celgene’s cellular therapeutics division.
The Company, since its inception, has raised an aggregate of $350.5 million, of which $170.5 million is from the initial public offering and concurrent private placements in May 2016, $95 million is through collaboration agreements, and $85 million is from the sale of convertible preferred stock.
Second Quarter 2016 Financial Results

As of June 30, 2016, Intellia had $300.7 million in cash and cash equivalents, which includes net proceeds from its initial public offering. Net loss for the second quarter 2016 was $6.9 million, compared to $3.0 million in the same period in 2015.

Collaboration revenue was $4.2 million in the second quarter 2016, compared to $1.4 million in the same period of 2015. The increase in collaboration revenue is primarily attributable to the inclusion of amounts recognized under the Regeneron collaboration in 2016.

Research and development expenses in the second quarter 2016 were $7.4 million, compared to $2.0 million in the same period in 2015. This increase in expenses is primarily attributable to the growth of the Company’s research and development organization to accelerate the development of the CRISPR/Cas9 platform and Intellia’s proprietary and partnered pipeline candidates.

General and administrative expenses were $3.7 million in the second quarter of 2016, compared to $2.8 million for the same period in 2015. The increase in general and administrative expenses is primarily driven by incremental expenses to support the Company’s operations as a new public company, as well as increased headcount-based expenses to support the Company’s overall growth.

Research & Development Highlights

Intellia is advancing its pipeline through a risk-mitigated approach focused on sentinel indication development, platform delivery expansion, and preclinical and clinical scale up. The Company is focused on developing the following programs:

Programs Partnerships Type of
Edit Delivery Upcoming Milestones
In Vivo
Transthyretin Amyloidosis (ATTR) Co-developing with
Regeneron Knockout LNP to Liver Select 1 to 2 development candidates and advance to IND enabling
studies in 2H2017/1H2018
Alpha-1 Antitrypsin
Deficiency (AATD) Proprietary Knockout LNP to Liver
Repair
Hepatitis B Virus (HBV) Proprietary Knockout LNP to Liver
Inborn Errors of
Metabolism (IEMs) Proprietary Knockout LNP to Liver
Repair
Insertion
Ex Vivo
Hematopoietic
Stem Cells (HSCs) Selectively partnered
with Novartis;
proprietary Knockout Electroporation First Novartis IND expected to be submitted in 2018
Repair
Insertion
CAR T Cells Partnered with
Novartis Knockout Electroporation Advance preclinical development
Insertion

Upcoming Events

Intellia will present delivery data utilizing CRISPR/Cas9 at the Cold Spring Harbor Laboratory Meeting, taking place from August 17-20, 2016. The oral presentation, Robust In Vivo Gene Editing in Mouse Hepatocytes with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 Components, will be presented by Intellia’s Chief Technology Officer, David Morrissey, Ph.D.
Intellia’s CEO & Founder Nessan Bermingham, Ph.D., will be presenting at the Wedbush PacGrow Healthcare Conference in New York on August 17, 2016, the Wells Fargo Healthcare Conference in Boston, September 7-8, 2016, the Morgan Stanley Global Healthcare Conference in New York on September 12, 2016, and the Leerink Partners Rare Disease Roundtable Series in New York on September 28-29, 2016.

Endocyte Reports Second Quarter 2016 Financial Results

On August 4, 2016 Endocyte, Inc. (NASDAQ:ECYT), a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported financial results for the second quarter ending June 30, 2016, and provided a clinical update (Press release, Endocyte, AUG 4, 2016, View Source [SID:1234514253]).

"Excitement is building around our lead assets, EC1456 and EC1169, as data to date has demonstrated attractive safety profiles and signs of anti-tumor activity for both agents. Later this month, we will advance EC1456 into targeted patients with non-small cell lung cancer (NSCLC) expressing the folate receptor, who are most likely to respond," said Mike Sherman, Endocyte’s president and chief executive officer. "We look forward to our first visibility into efficacy data for both compounds during the second half of the year."

EC1456 (Folate-tubulysin)

The EC1456 phase 1 dose escalation data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June highlighted a 45 percent rate of stable disease as best study response across a patient population that included more than a dozen cancer types. Patients are enrolled regardless of their folate receptor (FR) status during this first part of the study.

Endocyte announced today that the maximum tolerated dose (MTD) has been determined in the twice weekly (BIW) dosing schedule for EC1456 at 6.0 mg/m2. Enrollment of FR-positive NSCLC patients in the BIW expansion cohort will begin in August. In this expansion cohort the company plans to evaluate efficacy endpoints, including tumor response, in addition to ongoing assessment of safety. Endocyte’s companion imaging agent, EC20 (etarfolatide), will be utilized to select these patients. The company is continuing to evaluate the MTD in the once weekly dosing regimen.

EC1169 (PSMA-tubulysin)

The EC1169 phase 1 dose escalation study, as presented at ASCO (Free ASCO Whitepaper), highlighted that all patients in the study have some level of prostate specific membrane antigen (PSMA) positivity, and the drug has been well tolerated.

"EC1169 has the potential to be a truly differentiated therapy, and it has shown signs of anti-tumor activity, even at low doses, including reductions of prostate-specific antigen levels greater than 50 percent in some patients," commented Alison Armour, M.D., Endocyte’s chief medical officer. "We have worked with key opinion leaders in prostate cancer to define the expansion phase of this trial, and once we determine the MTD, we plan to begin enrolling second-line chemotherapy metastatic castrate resistant prostate cancer (mCRPC) patients, with a primary study endpoint of radiological progression free survival. We also plan to include an exploratory assessment of taxane-naïve patients, which could allow the possibility of an earlier line therapy."

Upcoming Expected Milestones

Phase 1 updates on EC1456 and EC1169 at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) conference in October 2016
Complete enrollment of first 15 patient cohort in EC1456 expansion trial; single agent efficacy data (tumor response) in NSCLC at a medical meeting in late 2016 or early 2017
EC1169 single agent efficacy data in prostate cancer in late 2016 or early 2017
Updates on plans for earlier stage programs
Second Quarter 2016 Financial Results

Endocyte reported a net loss of $14.0 million, or $0.33 per basic and diluted share, for the second quarter of 2016, compared to a net loss of $10.6 million, or $0.25 per basic and diluted share, for the same period in 2015.

Research and development expenses were $6.8 million for the second quarter of 2016, compared to $6.7 million for the same period in 2015. The slight increase was primarily attributable to an increase in expenses related to the EC1456 and EC1169 dose escalation trials, which was partially offset by a decrease in expenses related to the TARGET trial, which is now complete, and a decrease in compensation expenses, primarily related to noncash stock compensation.

General and administrative expenses were $7.4 million for the second quarter of 2016, compared to $4.1 million for the same period in 2015. The increase in expenses was primarily attributable to an increase in compensation expense related to the resignation of the company’s former Chief Executive Officer, P. Ron Ellis. The company executed a separation agreement with Mr. Ellis during the three months ended June 30, 2016, and under this agreement, the company incurred additional compensation expense of $2.8 million for noncash stock compensation and $0.8 million of expense for a cash payment. The increase in general and administrative expenses was partially offset by a decrease in legal fees.

Cash, cash equivalents and investments were $154.6 million at June 30, 2016, compared to $188.6 million at June 30, 2015, and $173.6 million at December 31, 2015.

Financial Expectations

The company revised guidance for its expected cash balance at the end of 2016 to be above $130 million. Previous guidance was between $125 and $130 million cash balance at the end of 2016.

About the EC1456 Phase 1 Trial

This open-label, multicenter, non-randomized, dose-escalation study is divided into two parts. The first part of the study was designed to evaluate safety and tolerability and identify the MTD of EC1456 in patients with metastatic or locally advanced solid tumors.

The second part of the study will determine the efficacy of EC1456 in patients with FR-positive NSCLC treated with the MTD. The BIW dosing schedule at 6.0 mg/m2 will be evaluated first. Upon the completion of this dosing schedule, additional patients will be enrolled in a once per week dosing schedule cohort. Single agent tumor response will be evaluated, which will inform and may trigger additional work in combination therapies and indications such as triple-negative breast cancer, ovarian cancer and endometrial cancer. Patient FR-status will be determined using the investigational companion imaging agent, EC20 (etarfolatide). EC1456 is currently being evaluated in a phase 1 study in patients with advanced solid tumors (ClinicalTrials.gov Identifier: NCT01999738).

About the EC1169 Phase 1 Trial

The second part of the study will determine the efficacy of the MTD of EC1169 in mCRPC patients who have been previously treated with a taxane-based chemotherapy. The primary study endpoint will be radiological progression free survival in patients selected as PSMA-positive. A second cohort will include an exploratory assessment of taxane-naïve patients. Patient PSMA status will be determined using the investigational companion imaging agent, EC0652. EC1169 is currently being evaluated in a phase 1 study in mCRPC patients (ClinicalTrials.gov Identifier: NCT02202447).