On May 9, 2016 Kite Pharma, Inc. (Nasdaq: KITE), a clinical-stage biopharmaceutical company focused on developing engineered autologous cell therapy (eACT) products for the treatment of cancer, reported a corporate update and reported first quarter 2016 financial results for the period ended March 31, 2016 (Press release, Kite Pharma, MAY 9, 2016, View Source [SID:1234512151]).

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"Our ZUMA-1 update at AACR (Free AACR Whitepaper) last month highlighted the potential of KTE-C19, our breakthrough immunotherapy candidate, to put patients with chemorefractory aggressive non-Hodgkin lymphoma (NHL), a patient population burdened with a short life expectancy and limited treatment options, into a durable complete remission," noted Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer. "We remain on track to provide interim data from the pivotal phase 2 portion of ZUMA-1 later this year and plan to submit the KTE-C19 registration filing to the U.S. Food and Drug Administration (FDA) by the end of 2016."

"Our commitment to delivering a breakthrough personalized cell therapy to cancer patients now extends fully to our manufacturing and market planning areas. Validation and qualification activities at our state-of-the-art commercial manufacturing facility are underway. The commercial team is actively evaluating a broad range of market and payor strategies for making KTE-C19 available to patients with a significant unmet need."

First Quarter 2016 and Recent Highlights

At AACR (Free AACR Whitepaper), reported rapid and durable responses in patients with chemorefractory diffuse large B cell lymphoma treated with KTE-C19 in the phase 1 portion of ZUMA-1.
Ongoing complete responses were observed in 3 of 7 patients at 9-month study follow-up (1 patient) and 6-month study follow-up (2 patients).
KTE-C19-related adverse events consisted predominantly of cytokine release syndrome and neurotoxicity, which were generally reversible.
Partnered with Genentech to study KTE-C19 in combination with the checkpoint inhibitor atezolizumab. Kite expects to initiate a Phase 1b/2 combination study in patients with chemorefractory diffuse large B cell lymphoma in the second half of 2016.
Expanded our clinical and research partnership with the National Cancer Institute (NCI) by entering into a new Cooperative Research and Development Agreement (CRADA) with James (Jim) N. Kochenderfer, M.D., and the NCI’s Experimental Transplantation and Immunology Branch.
Phase 1 study of human anti-CD19 chimeric antigen receptor for treating B-cell malignancies currently ongoing.
Also at AACR (Free AACR Whitepaper), William Lu, Ph.D., a collaborator of Kite’s at the NCI, reported data from a Phase 1 study of a T cell receptor (TCR) product candidate targeting MAGE-A3 that was advanced under Kite’s CRADA with the Surgery Branch at the NCI.
Data reported at AACR (Free AACR Whitepaper) support Kite’s plan to file later this year an investigational new drug (IND) application for a TCR product candidate that targets a MAGE-A3 antigen expressed on solid tumors.
Entered into a research and license agreement with Leiden University Medical Center in the Netherlands to identify and develop additional TCR product candidates targeting solid tumors that are associated with the human papillomavirus (HPV) type 16 infection.
Appointed Tim Moore, a biopharma executive with more than 30 years of global operations experience, as Executive Vice President, Technical Operations, to lead product development, manufacturing, supply chain, quality assurance, and end-to-end process optimization for all aspects of Kite’s engineered T cell product candidates.
Augmented Kite’s commercial function, under the leadership of Chief Commercial Officer Shawn Tomasello, with the appointment of an integrated executive team responsible for all aspects of commercial and medical affairs strategy, planning, and analysis for the potential launch of KTE-C19.
First Quarter 2016 Financial Results

Revenue was $5.1 million for the first quarter of 2016.
Research and development expenses were $34.4 million for the first quarter of 2016, and include $8.5 million of non-cash stock-based compensation expense.
General and administrative expenses were $16.5 million for the first quarter of 2016, and include $6.4 million of non-cash stock-based compensation expense.
Net loss was $43.9 million, or $0.90 per share, for the first quarter of 2016.
Non-GAAP net loss for the first quarter of 2016 was $29.1 million, or $0.60 per share, which excludes non-cash stock-based compensation of $14.9 million.
As of March 31, 2016, Kite had $577.4 million in cash, cash equivalents, and marketable securities.
Kite continues to expect the full year 2016 net cash burn to be $235 to $250 million dollars, which includes approximately $20 million in capital expenditures, but excludes any inflows or outflows from business development activities. The estimated full year 2016 net cash burn is primarily driven by an estimated net loss of $295 to $310 million, which includes an estimated $80 million of non-cash stock-based compensation expense.

On May 9, 2016 Dicerna Pharmaceuticals, Inc. (Nasdaq:DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, reported financial and operational results for the first quarter ended March 31, 2016 (Press release, Dicerna, MAY 9, 2016, View Source [SID:1234512150]).

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"We continue to achieve milestones across our portfolio of opportunities, including program launches with our DsiRNA-EX Conjugate technology, as well as our DCR-PH1 and DCR-MYC clinical programs," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "We look forward to sharing new preclinical data for our conjugate programs as well as further updates on our clinical programs at our upcoming Investor Day."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: Dicerna’s Dicer substrate short interfering RNA extended (DsiRNA-EX) Conjugates are proprietary RNAi therapeutic candidates rationally designed to enable convenient subcutaneous delivery for Dicerna’s emerging pipeline of liver-targeted RNAi investigational therapies. These conjugates do not involve lipid nanoparticles and are built on the DsiRNA-EX platform, using an extension to one end of the double-stranded DsiRNA molecule. These extensions are unique to Dicerna and utilize proprietary RNA structures that enable a differentiated and independent approach to subcutaneous delivery of RNAi-inducing therapeutics.

Dicerna is enhancing and extending the potency and reach of its DsiRNA-EX Conjugate technology, and has now achieved an IC50 level as low as 0.1 mg/kg in mice, generating greater than 90% gene knockdown at a dose of 1 mg/kg. Dicerna believes that potency in this range will translate into simple, infrequent single-shot dosing regimens in patients.
Dicerna will present extensive preclinical data for its DsiRNA-EX Conjugate programs, including non-human primate data against multiple gene targets, at the Company’s first Investor Day on June 29, 2016 in New York City.
Dicerna is on track to launch three DsiRNA-EX Conjugate programs in 2016, including an ongoing subcutaneous program for the treatment of primary hyperoxaluria that is advancing into IND-enabling studies.
Dicerna’s emerging library of DsiRNA-EX Conjugate molecules encompasses subcutaneously administered inhibitors for more than a dozen liver disease gene targets at various levels of optimization. These gene targets are in the fields of rare diseases as well as chronic liver disease, cardiovascular disease, and viral infectious disease. We expect these emerging programs will enable Dicerna to build a deep and broad pipeline of liver-targeted therapies, and create extensive opportunity for partnership and collaboration leading to additional program launches.
Rare Disease Program Update

DCR-PH1: DCR-PH1 is an intravenously infused DsiRNA-EX-based therapeutic candidate for primary hyperoxaluria type 1 (PH1), a severe, rare genetic disease of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, DCR-PH1 knocked down the activity of the HAO1 gene transcript that encodes for the enzyme glycolate oxidase, thereby significantly reducing the production of oxalate, the key mediator of disease pathology in PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit. In non-human primate studies, a single dose of DCR-PH1 led to an average of 84% knockdown of the HAO1 gene transcript. The DCR-PH1 clinical program consists of the following studies:

PHYOS: During the fourth quarter of 2015, Dicerna initiated PHYOS (Primary Hyperoxaluria Observational Study), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is designed to measure biomarkers implicated in PH1 and to identify patients who may be eligible for the Phase 1 trial of DCR-PH1.
Healthy Volunteer Study: Dicerna continues to enroll in DCR-PH1-102, a Phase 1 dose escalation trial of DCR-PH1 in healthy volunteers. The primary objective of this study is to determine the safety profile of DCR-PH1 in healthy volunteers in order to support dosing of PH1 patients in the United States. This trial was initiated in the fourth quarter of 2015.
PH1 Patient Study: During the second quarter of 2016, Dicerna initiated DCR-PH1-101, a Phase 1 dose escalation trial in PH1 patients, in Germany. Dicerna anticipates initiating additional sites in Europe and the United States during the second-half of 2016. The primary objective of this study is to determine the safety and tolerability of single ascending doses of DCR-PH1, and secondary objectives include measurement of the drug’s PK properties, and corresponding PD measurements of oxalate (associated with disease progression) and glycolate (a marker of DCR-PH1 activity). Dicerna expects to complete this trial in the first half of 2017.
Oncology Program Update

DCR-MYC: DCR-MYC is a potent and specific inhibitor of MYC, an oncogene frequently amplified or overexpressed in a wide variety of tumor types, including hepatocellular carcinoma (HCC). MYC has long been considered "undruggable" with small molecule and antibody technologies. DCR-MYC is a Dicer substrate short interfering RNA (DsiRNA)-based therapeutic formulated in Dicerna’s EnCore lipid nanoparticle for delivery to solid tumors. In preclinical studies, DCR-MYC knocked down MYC transcript levels and significantly reduced tumor volume in multiple mouse tumor models, including models of HCC. DCR-MYC is currently being tested in two ongoing clinical trials.

Phase 1 DCR-MYC Trial in Solid Tumors

Having established safety at 1.0 mg/kg, Dicerna has initiated two expansion cohorts of this Phase 1 clinical trial. The first expansion cohort is enrolling patients with low-to-intermediate grade pancreatic neuroendocrine tumors (PNETs) having failed one or two lines of prior therapy. The second expansion cohort will enroll patients who will undergo pre- and post-treatment biopsies in order to directly assess molecular markers of RNAi activity against the MYC transcript. Direct observation of RNAi activity of DCR-MYC, combined with observations both of anti-tumor activity and inhibition of FDG uptake in tumors, will establish proof-of-concept for the RNAi-based mechanism of action of DCR-MYC. The Company expects to have these proof-of-concept data in 2016.
Phase 1 DCR-MYC Trial in Hepatocellular Carcinoma (HCC)

In December 2014 Dicerna initiated a Phase 1b/2 clinical trial of DCR-MYC in patients with advanced HCC. As of April 26, 2016, 19 patients have been treated with DCR-MYC in six cohorts. The current dosing level is 0.85 mg/kg. While an objective response based on modified Response Evaluation Criteria in Solid Tumors (mRECIST) has not been observed, a reduction in circulating alpha-fetoprotein level, a marker associated with anti-tumor activity, has been observed. The trial continues with dose escalation. Dicerna expects to have preliminary data from the HCC trial by the end of 2016.
Corporate Update

Dicerna announces today that it plans to host an Investor Day on June 29, 2016 in New York City to present new preclinical data for its emerging pipeline of DsiRNA-EX Conjugate programs, as well as an update on its PH1 and oncology clinical programs.

Financial Results

Cash Position – As of March 31, 2016, the Company had $80.6 million in cash and cash equivalents and held-to-maturity investments as compared to $94.6 million in cash and cash equivalents and held-to-maturity investments as of December 31, 2015. In addition, the Company had $1.1 million of restricted cash, which reflects collateral securing its lease obligations.
R&D Expenses – Research and development (R&D) expenses for the first quarter were $11.3 million, compared to $8.7 million for the same period in 2015. The increase in R&D expenses was due primarily to increased expenses related to discovery and early development of future programs including development of the DsiRNA-EX Conjugate delivery platform, increases in clinical activities from initiating additional studies in PH1, increased employee-related expenses primarily due to additional hiring during the period, along with stock-based compensation, and increased occupancy costs.
G&A Expenses – General and administrative (G&A) expenses for the first quarter were $4.5 million, compared to $5.4 million for the same period in 2015. The decrease in these costs was primarily due to stock-based compensation and payroll related cost savings, and a decrease in costs related to the facility move incurred in the first quarter of 2015.
Net Loss – Net loss for the first quarter was $15.7 million compared to a net loss of $14.1 million for the same period in 2015.
For more detailed information and analysis see the Company’s Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on May 9, 2016.

Guidance

Based on Dicerna’s current cash position and operating plan, the Company reiterates its expectation that it has sufficient cash to fund operations for at least the next 12 months. This estimate assumes no additional funding from new partnership agreements or debt or equity financing events.

On May 9, 2016 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer, reported financial results for the first quarter ended March 31, 2016, and also provided an overview of certain corporate developments (Press release, Verastem, MAY 9, 2016, View Source;p=irol-newsArticle&ID=2166539 [SID:1234512149]).

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"To date in 2016, Verastem has announced two new clinical collaborations with world-class organizations, including Merck KGaA and Pfizer, and Washington University in St. Louis and Merck & Co., to further elucidate the potential of FAK inhibition to enhance the efficacy of PD-(L)1 inhibitors in patients with pancreatic and ovarian cancer," said Robert Forrester, President and Chief Executive Officer of Verastem. "The data generated from these trials will continue to inform the ongoing development of our anti-cancer therapeutics which reduce cancer stem cells and modulate the local tumor microenvironment to allow both cancer treatments and the immune system to do their job more efficiently. We’ve had a strong start to 2016 with the announcement of these clinical collaborations in addition to attracting key strategic hires on the development team, including Dr. Greg Berk as Chief Medical Officer and Dr. Toyin Shonukan as Vice President of Clinical Development, to oversee and execute on our ongoing and future studies. We are well financed with approximately $100 million in available capital and we look forward to keeping you updated in the coming quarters on our progress."

First Quarter 2016 and Recent Highlights:
Focal Adhesion Kinase Inhibition Program
Clinical Collaboration with Pfizer and Merck KGaA to Evaluate Combination of VS-6063 and Avelumab in Ovarian Cancer – In March 2016, the companies announced a clinical trial collaboration agreement to evaluate the combination of Verastem’s focal adhesion kinase (FAK) inhibitor VS-6063 and Pfizer and Merck KGaA’s anti-PD-L1 immunotherapy avelumab. Verastem has previously reported initial signs of clinical activity in patients with ovarian cancer when VS-6063 is used in combination with paclitaxel. Under the terms of the agreement, the parties will conduct a planned Phase 1/1b clinical trial evaluating escalating doses of the combination of VS-6063 and avelumab as a potential treatment option for patients with advanced ovarian cancer.

Washington University in St. Louis Initiated a Clinical Study of VS-6063 in Combination with Merck & Co.’s Pembrolizumab and Gemcitabine in Pancreatic Cancer – In January 2016, Verastem announced the initiation of a Phase 1 dose-escalation study at Washington University to evaluate its FAK inhibitor VS-6063 in combination with Merck & Co.’s anti-PD-1 immunotherapy pembrolizumab and gemcitabine in patients with pancreatic cancer. The trial builds upon preclinical research conducted by Dr. David Denardo, presented at several conferences in late 2015 and early 2016, demonstrating the ability of FAK inhibition to increase the efficacy of checkpoint inhibition in the reduction of tumor volume and overall survival in models of pancreatic cancer. This Phase 1 clinical trial is currently enrolling and is anticipated to enroll approximately 50 patients with advanced pancreatic cancer.

Presented Scientific Data Supporting FAK Inhibition in Combination with Immunotherapy at Key Medical Meetings – During the first quarter of 2016, Verastem presented data in support of its new development programs focused on advancing its FAK inhibitors in combination with immune-oncology agents and other current and emerging standard of care cancer treatments. Data were presented at several medical and scientific meetings, including the 2016 American Academy of Cancer Research (AACR) (Free AACR Whitepaper), the Society for Gynecologic Oncology’s 2016 Annual Meeting on Women’s Cancer, the Keystone Symposium on Cancer Pathology, the Keystone Symposium on Stem Cells and Cancer, and Immunotherapy World 2016.

Presented Clinical Data from the Window of Opportunity Study at iMig 2016 – In May 2016, the Company announced results from the ongoing open-label, single-center, neoadjuvant Window of Opportunity study evaluating tolerability, along with biomarker and tumor volume response to VS-6063 (400mg BID) following either 12 days (Cohort 1) or 35 days (Cohort 2) of treatment in surgically-eligible patients with malignant pleural mesothelioma. Data analysis from Cohort 1 and Cohort 2 showed that VS-6063 was generally well tolerated with early signs of tumor reduction observed, with six of the twenty patients demonstrating an encouraging tumor reduction after brief treatment with VS-6063.

Development of VS-4718 Continues in Solid Tumors – Dosing continues in a Phase 1 dose escalation trial evaluating single-agent VS-4718 and a Phase 1 clinical trial evaluating VS-4718 in combination with gemcitabine and Abraxane is currently ongoing. Following results from the dose escalation trial, an expansion cohort of VS-4718 + Gemcitabine/Abraxane vs Gemcitabine/Abraxane alone in patients with pancreatic cancer is planned.

Dual PI3K/mTORC1/2 Inhibition Program
Confirmatory Recommended Phase 2 Dose and Expansion Cohorts – The maximum tolerated dose of single-agent VS-5584 has been reached in a Phase 1 study, and the recommended Phase 2 dose (RP2D) is being confirmed. Reductions in pharmacodynamic markers of PI3K and mTOR activity and clinical activity has been observed in some tumor types.

Corporate
Gregory I. Berk, MD Named Chief Medical Officer – In April 2016, the Company announced the appointment of Gregory I. Berk, MD as Chief Medical Officer. Dr. Berk, a highly accomplished physician and a well-regarded oncology veteran with more than 25 years of both industry and academic experience, will be responsible for leading the Company’s global clinical development strategy and clinical operations.

Announced Key Executive Management Appointments and Changes – In April 2016, the Company strengthened its management team through the appointment and promotion of several key individuals. Jonathan Pachter, PhD was promoted to Chief Scientific Officer, David Weaver, PhD was appointed Vice President, Translational Medicine, Joe Chiapponi, Vice President, Finance, was named Treasurer, Principal Accounting and Financial Officer and Oluwatoyin (Toyin) Shonukan, MD, has been appointed Vice President, Clinical Development. Dr. Shonukan most recently served as Senior Medical Director, Oncology Clinical Development at Vertex Pharmaceuticals and has held previous senior appointments at Millennium: The Takeda Oncology Company, Novartis Oncology and Eli Lilly.

First Quarter 2016 Financial Results
Net loss for the first quarter ended March 31, 2016 (2016 Quarter) was $8.3 million, or $0.22 per share, as compared to a net loss of $15.2 million, or $0.46 per share, for the first quarter ended March 31, 2015 (2015 Quarter). Net loss for the 2016 Quarter and 2015 Quarter, excluding non-cash stock-based compensation expense of $1.7 million and $2.9 million, was $6.6 million and $12.3 million, respectively.

Research and development expense for the 2016 Quarter was $4.2 million compared to $10.5 million for the 2015 Quarter. The $6.3 million decrease from the 2015 Quarter to the 2016 Quarter was primarily related to a decrease of $4.2 million in contract research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, a decrease in personnel related costs of $1.4 million, a decrease of approximately $550,000 in stock-based compensation, and a decrease of approximately $441,000 in travel, facilities and other research and development costs. These decreases were partially offset by an increase of approximately $276,000 in consulting fees.

General and administrative expense for the 2016 Quarter was $4.3 million compared to $4.7 million for the 2015 Quarter. The decrease of approximately $400,000 from the 2015 Quarter to the 2016 Quarter primarily resulted from approximate decreases in stock-based compensation expense of $734,000 and $148,000 in personnel related costs. These decreases were offset by an increase of approximately $411,000 in consulting and professional fees.

As of March 31, 2016, Verastem had cash, cash equivalents and investments of $99.5 million compared to $110.3 million as of December 31, 2015. Verastem used $10.8 million for operating activities during the 2016 Quarter settling one-time compensation payments, severance payments and paying down accounts payable and accruals.
The number of outstanding common shares as of March 31, 2016, was 36,992,418.

Financial Guidance
Based on current operating plans, we expect to have sufficient cash, cash equivalents and short-term investments to fund our research and development programs and operations into 2018.

About Focal Adhesion Kinase
Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for their ability to improve patient survival.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors

On May 9, 2016 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a clinical stage biopharmaceutical company focused on discovering and developing novel cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the first quarter of 2016 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, MAY 9, 2016, View Source;p=irol-newsArticle&ID=2166540 [SID:1234512148]).

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"We continue to make good progress advancing our stem cell transplant, CAR T and TCR programs," said Tom Farrell, President and Chief Executive Officer of Bellicum. "Our study of lead product candidate BPX-501, an adjunct T-cell therapy in the haploidentical transplant setting, continued to yield impressive preliminary results. As of the end of the first quarter, with a median follow-up of approximately seven months, we have seen no transplant-related mortality in 49 evaluable patients at our lead European site, including 24 of 24 children with life-long genetic blood diseases who remain alive and disease-free, and 16 of 17 leukemia patients who remain in remission. We were also excited to see that two of three compassionate use relapsed/refractory AML patients treated with multiple doses of BPX-501 remain in remission 13 and 4 months post-transplant respectively."

Continued Mr. Farrell, "We are also preparing to advance three of our next-generation CAR T and TCR product candidates into the clinic in 2016. We believe the inclusion of our proprietary cellular control switches and our novel MC co-stimulatory domains may improve the function of T-cell therapies for attacking both solid and hematologic cancers."

PROGRAM HIGHLIGHTS

BPX-501

Reported new interim data from BP-004 trial, showing disease-free outcomes in pediatric patients, including those with blood cancers who had undergone T-depleted, haploidentical hematopoietic stem cell transplantation (HSCT) followed by BPX-501 donor T-cell replacement. At the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), preliminary outcomes of 17 pediatric leukemia patients were reviewed in an oral presentation, showing that BPX-501 cells expand in vivo and persist over time, contributing to adaptive immunity. Additionally, the relapse rate compared favorably with that of historical controls, with 16 of 17 patients in the trial showing disease-free outcomes. The median follow-up period for these patients was approximately seven months. Initial outcomes for nonmalignant patients at the same site were also reviewed, which showed that all 24 children treated remain disease-free (median follow-up period of approximately seven months), consistent with earlier results presented at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2015. Transplant-related mortality (TRM) was 0% (0 of 49) across all patients reported.
Received orphan drug designation from FDA for the combination of BPX-501 genetically modified T cells and activator agent rimiducid as "replacement T-cell therapy for the treatment of immunodeficiency and Graft versus Host Disease after allogeneic hematopoietic stem cell transplant."
Preparing to meet with the European Medicines Agency and U.S. FDA, with the goal of defining the path to regulatory filing and approval.
BPX-601: Preparing to initiate a Phase 1 clinical trial with BPX-601 GoCAR-T product candidate in mid-2016 in the initial indication of non-resectable pancreatic cancer. GoCAR-T contains Bellicum’s proprietary iMC (inducible MyD88/CD40) activation switch and is designed to treat solid tumors expressing prostate stem cell antigen (PSCA).

BPX-701: Preparing to initiate a Phase 1 clinical trial with BPX-701 high affinity T cell receptor (TCR) product candidate in mid-2016. BPX-701 incorporates the CaspaCIDe safety switch and is designed to target malignant cells expressing the preferentially-expressed antigen in melanoma, or PRAME. Initial planned indications include Refractory or Relapsed Acute Myeloid Leukemia and Myelodysplastic Syndromes, with an additional clinical trial planned for metastatic uveal melanoma.

BPX-401: Continued to advance CIDeCAR CAR T therapy, with plans to initiate clinical development in the second half of 2016.

First Quarter 2016 Financial Results:

Bellicum reported a net loss of $15.1 million for the first quarter of 2016, compared to a net loss of $7.8 million for the first quarter of 2015. The results included non-cash, share-based compensation charges of $3.1 million and $1.5 million for the first quarter of 2016 and 2015, respectively. As of March 31, 2016, cash and investments totaled $151.8 million, compared to $150.4 million at December 31, 2015. In March 2016, we closed on a debt financing agreement that allows for borrowings of up to $30.0 million which we intend to use for the build-out of our manufacturing facilities and for general corporate purposes. We received initial net proceeds of $14.8 million on the closing date.

Research and development expenses were $11.0 million and $5.7 million for the three months ended March 31, 2016 and March 31, 2015, respectively. The $5.3 million increase in R&D expenses for the 2016 period was due to an increase in BPX-501 clinical and manufacturing costs of $2.3 million, primarily due to increased patient enrollment in our clinical trials. The higher R&D expenses were also due to an increase of $1.0 million for IND enabling activities on our product candidates, BPX-601, BPX-701 and BPX-401, plus an increase of $2.0 million of general research and development costs, which includes an increase of $1.6 million in research and development personnel costs, $0.6 million in allocated overhead costs and a decrease of $0.2 million in other costs.

General and administrative expenses were $4.3 million for the three months ended March 31, 2016 and $2.2 million for the three months ended March 31, 2015. The $2.1 million increase in G&A expenses for the 2016 period was principally due to our overall growth, including an increase of $1.4 million in costs related to personnel, of which $0.8 million was attributable to share based compensation expense, higher facility costs and increased legal, accounting and travel expenses.

On May 9, 2016 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company"), a specialty biopharmaceutical company engaged in developing and commercializing novel treatments in oncology, endocrinology and women’s health, reported financial and operating results for the first quarter ended March 31, 2016 (Press release, AEterna Zentaris, MAY 9, 2016, View Source [SID:1234512147]).

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Commenting on first quarter results, David A. Dodd, Chairman, President and Chief Executive Officer of the Company, stated, "During the first quarter, we made steady progress in the areas of product development, commercial performance and financial operations. I am pleased to report that we remain on track to attain our 2016 corporate objectives. We still expect to complete the pivotal Phase 3 Zoptrex trial in Q3 of 2016 and to report top-line results by year-end."

Mr. Dodd continued his commentary with a discussion of the development of Macrilen, which, if approved, will be the only FDA-approved means of evaluating adult growth hormone deficiency (AGHD), "Since announcing patient recruitment in the fourth quarter of last year, our CRO has opened approximately 20 of our anticipated 30 sites, enrolling approximately 30 patients. The pace of recruitment is accelerating, confirming our expectation that the confirmatory Phase 3 clinical study of Macrilen will be concluded in Q3 of 2016, also with top-line results being reported as soon as possible following trial conclusion. We feel very confident that the confirmatory study, which will consist of approximately 30 sites and a minimum of 110 patients, is on schedule."

Concluding, Mr. Dodd addressed the Company’s commercial operations, stating, "We are pleased with the building of success from our promotion of Saizen during the first quarter. Exceeding our baseline by 66% during the quarter was a great accomplishment by our sales team. Now that they have a number of targets that more closely matches our promotional capacity, we believe they should be in a position to produce meaningful commission revenue this year. I am hopeful that we will be able to add even more targets as EMD Serono recognizes the success of our efforts. Our full sales force sold APIFINY for only half of the first quarter but managed to generate commission revenue nevertheless. We are hopeful about the commercial potential of APIFINY because it is the only cancer-specific, non-PSA blood test for the evaluation of the risk of prostate cancer. Physicians have long needed such an adjunct to the PSA test. With APIFINY, we believe we can satisfy that need. As a result of our promotional efforts, we are realizing an increasing penetration and acceptance of APIFINY. More recently, we announced the expansion of our APIFINY agreement to reflect our exclusive rights to promote this product throughout the U.S. We believe that such an expanded agreement opens significant revenue opportunities in support of this exciting product."

First Quarter 2016 Financial Highlights

R&D costs were $3.7 million for the three-month period ended March 31, 2016, compared to $4.5 million for the same period in 2015. The decrease for the three-month period ended March 31, 2016, as compared to the same period in 2015, is mainly attributable to lower comparative third-party costs. The decrease in third-party costs is mainly attributable to the fact that the number of patients in active treatment in the clinical trial for Zoptrex was lower in the first quarter of 2016, as compared to the same period in 2015. The overall decrease in R&D costs is also explained by lower employee compensation and benefits costs, lower facilities rent and maintenance as well as lower other costs. A substantial portion of this decrease is due to the realization of cost savings in connection with our effort to streamline our R&D activities and to increase our commercial operations and flexibility by reducing our R&D staff, which was started in 2014 (the "Resource Optimization Program"), for which a provision was recorded in the third quarter of 2014.

G&A expenses were $1.9 million for the three-month period ended March 31, 2016, as compared to $3.4 million for the same period in 2015. The decrease is mainly attributable to the recording during three-month period ended March 31, 2015 of certain transaction costs associated with the completion of a public, registered offering of shares and warrants in March 2015.

Selling expenses were $1.7 million for the three-month period ended March 31, 2016, essentially unchanged as compared to the same period in 2015. The selling expenses for the three-month periods ended March 31, 2016 and 2015 represent the costs of our contracted sales force related to the co-promotion activities as well as our internal sales management team. Those activities were launched during the fourth quarter of 2014.

Net loss for the three-month period ended March 31, 2016 was $3.7 million, or $0.37 per basic and diluted share, compared to a net loss of $9.7 million, or $13.59 per basic and diluted share for the same period in 2015. The decrease in net loss for the three-month period ended March 31, 2016, as compared to the same period in 2015, is due largely to lower operating expenses and higher comparative net finance income.

Cash and cash equivalents were approximately $33.0 million as at March 31, 2016, compared to $41.5 million as at December 31, 2015.