Curis Reports Fourth Quarter and Year-End 2016 Financial Results

On March 9, 2017 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective drug candidates for the treatment of human cancers, reported its financial results for the fourth quarter and year ended December 31, 2016 (Press release, Curis, MAR 9, 2017, View Source [SID1234518052]).

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"We are pleased with the progress we’ve made in advancing our pipeline in 2016. For CUDC-907 and CA-170, we remain focused on patient enrollment in their respective trials to assess their benefit in cancer patients. The Phase 1 trial for CA-170 continues to progress on track through the dose escalation stage, and based on the early data, we have recently received regulatory approval to begin extension of the CA-170 Phase 1 study and enroll immunotherapy-naïve patients in Korea and Spain, with additional trial centers in other European countries projected to open in the second quarter. The Phase 2 trial of CUDC-907 in patients with relapsed/refractory MYC-altered diffuse large B cell lymphoma, or DLBCL is nearing completion of patient enrollment, and our goal is to assess CUDC-907’s efficacy in this patient population, as measured by the overall response rate in up to 60 patients. Depending on the Phase 2 trial results, we expect to hold discussions with the FDA regarding potential for accelerated approval of CUDC-907 as a monotherapy in this setting. In addition, we are completing IND-enabling studies of CA-327 and CA-4948," said Ali Fattaey, Ph.D., Curis’s CEO."

Dr. Fattaey continued, "Our collaboration with Aurigene continues to progress well. In September 2016, we completed a $24.5M financing with Aurigene. In October 2016, we licensed a second immuno-oncology program, and designated CA-327 as an oral small molecule development candidate targeting PDL1 and TIM3. We expect to file an IND for CA-327 in 2017. Our IRAK4 kinase inhibitor development candidate, CA-4948, has progressed well in preclinical characterization and IND-enabling studies, and we expect to file an IND for CA-4948 in mid-2017."

Royalty Financing Transaction

On March 6, 2017, Curis entered into an agreement with HealthCare Royalty Partners (HCR), for a $45 million debt transaction secured with future Erivedge royalties. As part of this transaction, Curis’s wholly-owned subsidiary, Curis Royalty LLC, borrowed $45 million at an annual interest rate of 9.95% interest to be repaid solely with Erivedge royalty payments received from Genentech. The transaction is expected to close later this month, at which time Curis would pay off the $18.4 million remaining balance on the existing loan from BioPharma-II.

"Erivedge represents an effective and medically important product for patients suffering from advanced forms of basal cell carcinoma, and we are pleased to partner with Curis in providing this non-dilutive financing as the company funds its oncology pipeline," said John Urquhart, Principal at HealthCare Royalty Partners.

"We continue to focus our capital and resources in developing our pipeline of innovative cancer therapies. We are especially pleased to work with HCR on this financing, which provides non-dilutive capital for our near-term development needs, while allowing us to retain the considerable upside potential in Erivedge. In its fifth year on the market, Erivedge revenue grew 21% to CHF 203 million in 2016. Genentech and Roche currently commercialize Erivedge in advanced basal cell carcinoma and are conducting clinical trials for its potential use in treating idiopathic pulmonary fibrosis and myelofibrosis," said James Dentzer, Curis’s Chief Financial and Chief Administrative Officer.

Full Year and Fourth Quarter 2016 Financial Results

For the year ended December 31, 2016, Curis reported a net loss of $60.4 million, or $(0.45) per share on both a basic and diluted basis, as compared to a net loss of $59.0 million, or $(0.48) per share on both a basic and diluted basis in 2015. For the fourth quarter of 2016, Curis reported a net loss of $11.3 million or $(0.08) per share on both basic and diluted basis, as compared to a net loss of $13.5 million, or $(0.10) per share on both basic and diluted basis for the same period in 2015. The net loss for the year ended December 31, 2016 includes a non-cash in-process research and development charge of $18.0 million related to the amendment of Curis’s license agreement with Aurigene. The net loss for the year ended December 31, 2015, includes a non-cash in-process research and development charge of $24.3 million related to Curis’s license agreement with Aurigene.

Revenues for the year ended December 31, 2016 were $7.5 million, as compared to $7.9 million for the same period in 2015. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche’s net sales of Erivedge. Revenues for the fourth quarters of 2016 and 2015 were $2.4 million and $2.1 million, respectively.

Operating expenses were $65.6 million for the year ended December 31, 2016, as compared to $64.4 million for the same period in 2015. Operating expenses for the fourth quarter of 2016 were $13.1 million, as compared to $15.3 million for the same period in 2015, and comprised the following:

Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche’s Erivedge net sales, were $0.4 million for both the years ended December 31, 2016 and 2015. Cost of royalty revenues for the fourth quarter of 2016 and 2015 were $0.1 million for both periods.

Research and Development Expenses. Research and development expenses were $31.6 million for the year ended December 31, 2016, as compared to $26.7 million for the same period in 2015. The increase was primarily due to increased direct spending related to clinical development activities of CUDC-907 and programs under the Aurigene collaboration over the prior year period. Employee-related expenses increased over the prior year period primarily due to additional headcount to support the multiple programs. Research and development expenses were $9.2 million for the fourth quarter of 2016 as compared to $12.0 million for the same period in 2015.

In-Process Research and Development Expense. In-process research and development expense was $18.0 million for the year ended December 31, 2016, as compared to $24.3 million for the same period in 2015. These charges are associated with the stock issuances of 10,208,333 and 17,120,131 shares of Curis common stock to Aurigene, in 2016 and 2015 respectively. These shares were issued as consideration for the rights granted under the terms of the September 2016 amendment to the collaboration agreement and partial consideration for the rights granted under the terms of the January 2015 collaboration agreement, respectively.

General and Administrative Expenses. General and administrative expenses were $15.6 million for the year ended December 31, 2016 as compared to $12.9 million for the same period in 2015. The increase in general and administrative expenses was driven primarily by higher personnel costs and stock-based compensation expense due to increased headcount, an increase in legal service costs and professional and consulting services. General and administrative expenses were $3.8 million for the fourth quarter of 2016, as compared to $3.2 million for the same period in prior 2015.

Other expense, net was $2.4 million for the year ended December 31, 2016, as compared to $2.5 million for the same period in 2015. Other expense, net was $0.6 million and $0.2 million for the fourth quarter of 2016 and 2015, respectively. Other expense, net primarily consisted of interest expense related to the loan made by BioPharma-II (an investment fund managed by Pharmakon Advisors) to Curis Royalty (a wholly owned subsidiary of Curis).

As of December 31, 2016, Curis’s cash, cash equivalents and investments totaled $44.5 million and there were approximately 141.1 million shares of common stock outstanding.

Recent Operational Highlights

Curis – Aurigene collaboration:

In January 2017, Curis exercised its option to extend the exclusivity period with Aurigene under the collaboration, license and option agreement established in January 2015. The extension of exclusivity is associated with a payment of $7.5 million to Aurigene, payable in two equal installments. The first installment was paid in January 2017 and the second installment is estimated to be paid in the third quarter of 2017.
CA-170 (PDL1/VISTA antagonist):

In November 2016, Curis presented preliminary clinical pharmacokinetic (PK) and early biomarker data from the ongoing dose escalation stage of its Phase 1 trial of CA-170 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting. Clinical data from a limited number of patients demonstrated that, similar to the preclinical findings, orally-administered CA-170 has a dose proportional and predictable PK profile in patients treated at various escalating doses. Additionally, analysis of patient blood samples showed that CA-170 is biologically active in modulating the immune system, with a several-fold increase in the percentage of circulating CD8+ T cells expressing activation markers within 24 hours of oral dosing.
CA-327 (PDL1/TIM3 antagonist):

In November 2016, Curis collaborator, Aurigene presented pre-clinical data at the SITC (Free SITC Whitepaper) Annual Meeting and EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) Molecular Targets and Cancer Therapeutics Symposium for CA-327, the development candidate targeting PDL1 and TIM3 immune checkpoints. The data from various in vitro and in vivo studies with CA-327 demonstrated potency and selectivity, oral bioavailability, desirable safety profile, and anti-tumor activity in multiple mouse syngeneic tumor models.
CUDC-907 (HDAC/PI3K inhibitor):

In December 2016, Curis presented non-clinical data from combination studies of CUDC-907 and venetoclax, a BH3 mimetic and BCL2 antagonist at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s annual meeting. In multiple DLBCL cell lines, the CUDC-907 and venetoclax combination exhibited synergistic activity resulting in reduced cell viability. The additive/ synergistic anti-tumor effects were also observed in vivo in mice bearing various DLBCL xenograft tumor models.
Erivedge:

In November 2016, the European Commission granted full approval to Erivedge (vismodegib) for the treatment of adult patients with symptomatic metastatic basal cell carcinoma (BCC) or locally advanced BCC inappropriate for surgery or radiotherapy. Erivedge was originally granted ‘conditional approval’ in July, 2013 in the European Union. Erivedge was approved in the U.S. in 2012 for the treatment of adults with metastatic basal cell carcinoma, or with locally advanced basal cell carcinoma that has recurred following surgery or who are not candidates for surgery or radiation. Erivedge was developed and is marketed by Roche and Genentech, a member of the Roche Group, under a collaboration agreement between Curis and Genentech.
Other:

In November 2016, Curis announced the appointment of Lori A. Kunkel, M.D. to its Board of Directors. Dr. Kunkel currently serves on the Board of Directors at Loxo Oncology, Amphivera Therapeutics Inc., Tocagen Inc., and Harpoon Therapeutics. In the past, Dr. Kunkel has served as CMO at Pharmacyclics, Inc., Proteolix Inc. (acquired by Onyx), Syndax, and ACT Biotech. She has also spent a number of years in academic/clinical medicine. She trained in internal medicine at Baylor College of Medicine, hematology at USC and oncology at UCLA, earning board certifications in these specialties.
Upcoming Activities
Curis expects that it will make presentations at the following conferences through April 2017:

Presentation of preclinical results from CA-4948 at the annual meeting of The American Association of Cancer Research in April, 2017

Progenics Pharmaceuticals Announces Fourth Quarter and Full-Year 2016 Financial Results and Business Update

On March 9, 2017 Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX) reported financial results and provided a business update for the fourth quarter and full-year 2016 (Press release, Progenics Pharmaceuticals, MAR 9, 2017, View Source [SID1234518050]).

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"Since the beginning of 2016, we have made tremendous progress in advancing our robust pipeline of innovative cancer treatments and executing against our strategic corporate goals," said Mark Baker, CEO of Progenics. "2017 has the potential to be a transformational year for Progenics, with AZEDRA topline data from our registrational trial in pheochromocytoma and paraganglioma expected in the coming weeks. Should we report positive data and meet the requirements of the Special Protocol Assessment, we will move quickly toward an NDA as we strive to introduce a promising treatment to patients with these rare and difficult-to-treat cancers."

Mr. Baker continued, "We also continued to advance our novel portfolio of prostate cancer imaging agents and therapeutics in 2016, including 1404, PyL and 1095, which could transform how physicians and patients find, fight and followTM prostate cancer. We are encouraged by the significant investigator interest in our PSMA-targeted candidates, and with our strong cash position, we look forward to progressing these programs through key trials this year."

Fourth Quarter and Recent Key Business Highlights

AZEDRA, Ultra-orphan radiotherapeutic candidate

AZEDRA Topline Results Expected First Quarter 2017. Progenics expects to report topline results from its ongoing registrational trial of AZEDRA by the end of March 2017. With positive data (AZEDRA trial meets the primary endpoint of the Special Protocol Assessment (SPA)), the Company expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in mid-2017.
PSMA-Targeted Prostate Cancer Pipeline

Positive DMC Recommendation for Continuation of Phase 3 Study of 1404. In December 2016, an independent Data Monitoring Committee (DMC) completed its review of an interim analysis of the Company’s ongoing Phase 3 clinical trial of its PSMA-targeted SPECT/CT imaging agent candidate 1404, and recommended that the trial continue. The study is designed to evaluate the specificity of 1404 imaging to identify patients without clinically significant prostate cancer and sensitivity to identify patients with clinically significant disease.

Initiated Phase 2/3 Trial of PSMA-Targeted PET/CT Imaging Agent PyL. Also in December 2016, Progenics announced that the first patient had been dosed in a Phase 2/3 trial of PyL. The study is designed to evaluate the diagnostic accuracy of PyL PET/CT imaging in patients with metastatic prostate cancer.

Launched PyL Research Access ProgramTM. In November 2016, Progenics announced a research access program making limited doses of PyL available to researchers. Progenics plans to use data generated from the access program to support its registration efforts for PyL and advance the development of algorithms designed to analyze and interpret the scans.

Initiated Phase 1 Trial of 1095 for the Treatment of Metastatic Prostate Cancer. The trial, which is being conducted at Memorial Sloan Kettering Cancer Center, is expected to include approximately 30 patients with mCRPC who have demonstrated tumor avidity to 1095. The objectives of this trial are to determine the maximum tolerated dose, safety and tolerability, biodistribution, and efficacy — results that will guide the decision of an optimal dose for a potential Phase 2 study.
RELISTOR, treatment for OIC (partnered with Valeant Pharmaceuticals International, Inc.)


RELISTOR (SC and Oral) Net Sales for the Fourth Quarter of 2016 Totaled $16 million. Full-year 2016 net sales totaled $70.6 million as reported by our partner, Valeant.

Announced $50 Million RELISTOR Royalty-Backed Non-Dilutive Debt Financing with HealthCare Royalty Partners. In November 2016, Progenics announced a $50 million non-recourse, term loan agreement at a per annum interest rate of 9.50 percent, to be secured by and repaid from royalties on future sales of RELISTOR. Any future sales milestones received under Valeant agreement are excluded from the transaction and would not be used to repay interest or principal on the loan.
Fourth Quarter and Full-Year 2016 Financial Results

Total revenue decreased $0.4 million for the fourth quarter and increased $60.8 million for the full-year, over the fourth quarter and full-year of 2015, respectively. The full-year increase was due primarily to milestone revenue of $50 million for the July 19 FDA approval of RELISTOR Tablets, higher RELISTOR royalty income, and the recognition of $7 million in upfront and development milestone payments from Bayer for the collaboration of our PSMA antibody technology in combination with Bayer’s alpha-emitting radionuclides.

Fourth quarter and full-year research and development expenses increased by $2.7 million and $9.4 million, respectively, compared to the corresponding periods in 2015, resulting primarily from higher clinical trial expenses for 1404 and PyL and higher contract manufacturing expenses for 1095, PyL and AZEDRA. Fourth quarter and full-year general and administrative expenses increased by $1 million and $5.2 million, respectively, compared to the corresponding prior year periods, primarily attributable to higher depreciation expense as a result of a reduction in the remaining useful lives of our leasehold improvements at our Tarrytown, NY former location and higher compensation and market research expenses. Progenics also recorded non-cash adjustments of $6 million and $4.6 million in the fourth quarter and full-year 2016, respectively, related to a decrease in the fair value estimate of the contingent consideration liability.

Net loss attributable to Progenics for the quarter was $7.2 million or $0.10 per diluted share, compared to a net loss of $7.1 million or $0.10 per diluted share in the corresponding 2015 period. Net income attributable to Progenics for the full-year 2016 was $10.8 million or $0.15 per basic and diluted share, compared to net loss of $39.1 million or $0.56 per diluted share for the full-year 2015.

Progenics ended the year with cash and cash equivalents of $138.9 million, reflecting increases of $40 million in the quarter and $64.8 million from 2015 year-end.

ImmunoCellular Therapeutics Announces Fourth Quarter and Full Year 2016 Financial Results and Provides Corporate Update

On March 9, 2017 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT: IMUC) reported financial results for the fourth quarter and full year 2016 and provided a corporate update (Press release, ImmunoCellular Therapeutics, MAR 9, 2017, View Source [SID1234518049]).

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Anthony Gringeri, PhD, ImmunoCellular’s President and Chief Executive Officer, commented: "During 2016, we continued to implement our ICT-107 registration trial in patients with newly diagnosed glioblastoma, and conduct our phase 1 trial of ICT-121 in patients with recurrent glioblastoma.Today, in our ICT-107 phase 3 trial, 376 patients have been screened, 21 have been randomized, and clinical site activation is continuing, with a total of 68 sites activated to date. We are pleased to report that the protocol amendment we submitted to the US FDA for the ICT-107 trial, designed to modify some elements of the patient screening process and accelerate the pace and efficiency of randomization, is currently being implemented at US clinical sites, and amended protocol submissions are underway in Europe and Canada. As a result of the protocol change, the Special Protocol Assessment (SPA) is no longer applicable. We plan to engage in further discussions with the FDA concerning a SPA for the amended protocol in the future. We do not expect the change in the status of the SPA to materially impact the execution of the phase 3 trial, and data generated from a successful phase 3 study can still be used as primary evidence to support a marketing application."

Continued Dr. Gringeri: "The phase 1 open-label trial of ICT-121 being conducted at six sites in the US, completed enrollment of 20 patients. We are now in the process of analyzing the results, and have submitted an abstract to present the data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June. As previously noted, the preliminary results, while unvalidated, were encouraging. We were also pleased to announce the achievement of a significant milestone in our Stem-to-T-cell program: the successful sequencing of a target T cell receptor gene, which is a key step toward identification of a potential clinical candidate. We are excited that our stem cell program is advancing, and look forward to working with our collaborators to develop antigen-specific killer T cell therapies."

Achievements, Upcoming Goals and Milestones:

ICT-107:
Continue to bring US, Canadian and European clinical sites online. A total of 68 sites have been activated to date.
Implementation of the protocol amendment, which will modify some elements of how patients qualify for the trial. We plan to randomize a total of 542 patients, and anticipate randomization of all patients to be completed by mid-2019, and an additional 2-3 years from then to achieve the number of required events.
Plan to conduct a futility interim analysis at 30% of events, or at about the time of full randomization, and an efficacy interim analysis at 67% of events, about six months later.
ICT-121:
Complete analysis of the data from the phase 1 trial. An abstract to ASCO (Free ASCO Whitepaper) has been submitted, with the goal of presenting preliminary results at the annual conference in June 2017.
Research:
Having achieved identification of a T cell receptor identified for a Stem-to-T-cell clinical candidate, the next research phase will focus on packaging the T cell receptor sequence into a lentivirus vector, the vehicle that will be used to transfect human hematopoietic cells.
Continue to progress in collaboration with University of Maryland on projects that have application to existing dendritic cell immunotherapy and Stem-to-T-cell technology platforms.
Fourth Quarter and Full Year 2016 Financial Results

For the year ended December 31, 2016, the Company incurred a net loss of $22.1 million, or $7.89 per basic and diluted share, compared to a net loss of $12.8 million, or $5.87 per basic and diluted share, for the year ended December 31, 2015. During 2016, the Company incurred $19.1 million of research and development expenses compared to $10.9 million in 2015. The $8.2 million increase reflects the additional expenses associated with the implementation of the phase 3 trial of ICT-107. During 2016, the Company accrued $1.3 million of interest expense related to the California Institute of Regenerative Medicine (CIRM) award compared to $134,000 in 2015. For the quarter ended December 31, 2016, the Company incurred a net loss of $6.3 million, or $1.36 per basic and diluted share, compared to a net loss of $4.8 million, or $2.13 per basic and diluted share, for the quarter ended December 31, 2015.

The Company also reported that cash used in operations in 2016 was $19.9 million compared to $19.0 million in 2015. During 2015, the Company purchased $2.2 million in supplies and incurred $3.7 million in vendor deposits. During 2016, the Company utilized $2.2 million of vendor deposits, and the amount of supplies on-hand remained relatively constant with the prior year. As of December 31, 2016, the Company had $11.4 million in cash and cash equivalents.

In August 2016, the Company entered into an underwriting agreement with Maxim Group LLC, pursuant to which the Company received net proceeds of approximately $6.6 million (after deducting the underwriting discount and offering expenses) from the initial sale of 863,750 shares of the Company’s common stock, base warrants to purchase 881,250 shares of common stock at an exercise price of $7.68 per share, and pre-funded warrants to purchase 311,250 shares of common stock at an exercise price of $0.40 per share. The aforementioned securities were sold at an offering price of $6.40 per unit. The underwriters partially exercised their option to purchase additional shares and base warrants and purchased an additional 37,500 shares of the Company’s common stock at a public offering price of $6.00 per share and base warrants to purchase 111,965 shares. The pre-funded warrants were substantially paid for at the time of the offering and have an exercise price of $0.40 per share. As of December 31, 2016, the Company had 3,444,859 shares of common stock issued and outstanding.

Additionally, the CIRM award was modified such that ImmunoCellular received an additional $1.5 million in August 2016 as part of the initial award received from CIRM. The total amount of the award and other award conditions remain unchanged.

MIRATI THERAPEUTICS REPORTS FINANCIAL RESULTS AND PROVIDES BUSINESS UPDATE FOR THE FOURTH QUARTER AND FULL YEAR 2016

On March 9, 2017 Mirati Therapeutics, Inc. (NASDAQ: MRTX) ("the Company," "we," "our," "us," or "Mirati") reported financial results for the fourth quarter and year ended December 31, 2016 and provided an update on its product development programs (Press release, Mirati, MAR 9, 2017, View Source [SID1234518044]).

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"We have made significant progress which positions us to report key data from all of our programs in 2017," said Charles M. Baum, M.D., Ph.D., President and CEO of Mirati. "Earlier this year, we presented glesatinib data demonstrating clinical responses in non-small cell lung cancer patients with MET driver alterations. In addition, preliminary data from our Phase 1b trial of sitravatinib has shown clinical benefit in patients with RET mutations. We plan to provide updates for both glesatinib and sitravatinib in the second half of this year.

We are excited about our immuno-oncology programs which combine sitravatinib or mocetinostat with checkpoint inhibitors. There is strong scientific rationale that sitravatinib or mocetinostat can significantly enhance the efficacy of checkpoint inhibitors. Following our successful public offering in January, we are well positioned to achieve the 2017 milestones in each of our programs with funding into late 2018."

Single Agent Programs

Glesatinib (MGCD265)
The Company is enrolling patients in its registration-enabling Phase 2 non-small cell lung cancer (NSCLC) AMETHYST clinical trial, which is evaluating single agent glesatinib for the treatment of NSCLC patients with MET driver alterations. In January 2017, the Company reported early data that demonstrated promising activity in these molecularly selected patients. In NSCLC patients with MET Exon 14 deletion mutations treated with glesatinib, across both the Phase 1b and Phase 2 trials, tumor reduction was observed in 11 of 13 patients, with confirmed and unconfirmed partial responses in 6 of 13 evaluable patients. Glesatinib also demonstrated clinical benefit in NSCLC patients with MET gene amplification, including tumor reduction in six of eight patients as well as two unconfirmed partial responses out of eight evaluable patients. The Company expects to provide an update on efficacy data from the AMETHYST trial in the second half of 2017.

Sitravatinib (MGCD516)
The Company is enrolling patients in its Phase 1b expansion clinical trial, which is evaluating single agent sitravatinib for the treatment of NSCLC patients with RET, CHR4q12 and CBL genetic alterations. In January 2017, the Company reported that six NSCLC patients with RET fusion mutations had been enrolled and all four evaluable patients showed tumor reductions with one confirmed and one unconfirmed response. The Company expects to provide an update on efficacy data in the third quarter of 2017.


Immuno-oncology Combination Programs

Sitravatinib plus nivolumab
Sitravatinib is a potent inhibitor of the TAM (Tyro, Axl, Mer) and split (KDR, KIT) tyrosine kinase families which have been shown in preclinical studies to enhance anti-tumor immunity and the effects of checkpoint inhibitors. In November 2016, the Company initiated enrollment in a multicenter Phase 2 NSCLC clinical trial evaluating sitravatinib in combination with nivolumab, a PD-1 checkpoint inhibitor approved for the treatment of patients with NSCLC. The trial is enrolling patients who have relapsed after treatment with a checkpoint inhibitor. The Company expects to provide initial results from this combination trial in the second half of 2017.

Mocetinostat (MGCD103) plus durvalumab
The Company is collaborating with MedImmune/Astra Zeneca on a Phase 1b/2 clinical trial combining mocetinostat, an orally administered spectrum-selective Class 1 HDAC inhibitor, and durvalumab, MedImmune’s monoclonal antibody inhibiting PD-L1. Preclinical data have shown that mocetinostat significantly enhances the efficacy of the checkpoint inhibitor through its effects on the tumor microenvironment. The combination trial is exploring the potential of mocetinostat to enhance the effectiveness of durvalumab in NSCLC patients and the Company expects to provide an update in mid 2017.

Preclinical Programs
The Company’s lead preclinical programs to develop inhibitors of LSD1 and KRAS are expected to have meaningful milestones in 2017. The Company’s LSD1 inhibitor is highly-potent and potentially best-in-class with potential for rapid clinical proof-of-concept in small cell lung cancer and/or acute myeloid leukemia. An investigational new drug (IND) submission is planned for this compound in the fourth quarter of 2017. The Company has identified a mutant-selective KRAS inhibitor which is advancing to the candidate selection phase. Prototype inhibitors have demonstrated marked tumor regression in KRAS mutant tumor models and the Company anticipates selecting an IND candidate in the second half of 2017.

Financing
In January 2017, the Company strengthened its balance sheet by completing a public offering of common stock and pre-funded common stock warrants generating net proceeds of $66.8 million. Following this offering, the Company expects that its available cash, cash equivalents and short-term investments are sufficient to fund operations into late 2018.

Fourth Quarter and Full Year Financial Results
Cash, cash equivalents, and short-term investments were $56.7 million on December 31, 2016, as compared to $122.3 million on December 31, 2015.
Research and development expenses for the fourth quarter of 2016 were $16.0 million, compared to $14.9 million for the same period in 2015. Research and development expenses for the year ended December 31, 2016 were $68.5 million, compared to $49.0 million for the same period in 2015. The increases in research and development expenses for both periods primarily reflect costs to advance the clinical development of the Company’s glesatinib and sitravatinib oncology development programs, an increase in salaries and related expense, which is due to an increase in research and development employees during 2016 compared to the same period of 2015, and an increase in early discovery expenses.

General and administrative expenses for the fourth quarter of 2016 were $3.9 million, compared to $3.6 million for the same period in 2015. General and administrative expenses for the year ended December 31, 2016 were $15.3 million, compared to $15.8 million for the same period in 2015. The comparable level of expenses for both periods reflect a consistent level of general and administrative activities during both periods.
Other income, net, was $0.1 million for both the fourth quarter of 2016 and 2015. Other income, net, for the year ended December 31, 2016 was $0.6 million compared to $0.2 million for the same period in 2015.
Net loss for the fourth quarter of 2016 was $19.7 million, or $0.99 per share basic and diluted, compared to net loss of $18.4 million, or $0.96 per share basic and diluted for the same period in 2015. Net loss for the year ended December 31, 2016 was $83.1 million, or $4.20 per share basic and diluted, compared to net loss of $64.5 million, or $3.82 per share basic and diluted for the same period in 2015.

Epizyme Reports 2016 Financial Results and Provides 2017 Pipeline Goals

On March 9, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported financial results for the fourth quarter and full year 2016 (Press release, Epizyme, MAR 9, 2017, View Source [SID1234518042]). In addition, the Company reviewed recent highlights and provided guidance on its clinical and research pipeline in 2017.

“2016 was a year of tremendous progress across all aspects of Epizyme, which set the stage for 2017 to be a transformational year for the company,” said Robert Bazemore, president and chief executive officer of Epizyme. “We have a broad clinical development program with tazemetostat in multiple tumor types and treatment settings. We plan to present data from both our ongoing, global Phase 2 studies in both molecularly defined solid tumors and non-Hodgkin lymphoma in June, and to initiate discussions with regulators to define registration pathways for tazemetostat. We are advancing our ongoing monotherapy and combination studies and plan to announce the next development candidate from our preclinical pipeline this year. As we look ahead, we are on our way to achieving our vision of rewriting cancer treatment through novel epigenetic medicines.”

Recent Achievements

Tazemetostat Combination with Prednisolone Initiated: Epizyme has initiated clinical investigation of tazemetostat in combination with prednisolone in relapsed/refractory patients with DLBCL, based on observed preclinical synergy of the agents. This combination regimen is being conducted as the sixth cohort to the ongoing Phase 2 NHL study. Epizyme also anticipates initiating a new combination study in patients with follicular lymphoma in 2017.
Enrollment Completed in Three of Five NHL Study Cohorts: Epizyme has completed enrollment in three of the five cohorts of its ongoing, global Phase 2 study of tazemetostat in patients with relapsed/refractory non-Hodgkin lymphoma (NHL): the two cohorts enrolling patients with diffuse large B-cell lymphoma (DLBCL) with wild-type EZH2 and a third enrolling patients with follicular lymphoma with wild-type EZH2.
Follicular Lymphoma Enrollment Initiated in U.S.: In January 2017, following a positive written response from the U.S. Food and Drug Administration (FDA) to the company’s written request, Epizyme opened enrollment to patients with follicular lymphoma in the United States as part of the Company’s Phase 2 study in NHL.
Fast Track Designation Granted for DLBCL with EZH2 Mutations: In December 2016, the U.S. FDA granted Fast Track designation to tazemetostat for the treatment of patients with DLBCL whose tumors carry an EZH2 activating mutation. DLBCL is the most common form of NHL. The Fast Track designation provides expedited processes that may reduce development time and costs associated with bringing a drug to market.
Epithelioid Sarcoma Cohort Expanded in Phase 2 Solid Tumor Study: In late 2016, Epizyme expanded the epithelioid sarcoma cohort of its ongoing Phase 2 trial of tazemetostat in adult patients with certain molecularly defined solid tumors, following review by the Independent Data Monitoring Committee. This expansion was based on encouraging early activity, including confirmed objective responses, and surpassing the pre-specified futility hurdle in the cohort. This cohort will enroll up to 60 patients, or double the initial cohort size of 30 patients.
Synovial Sarcoma Cohort Completed Enrollment in Phase 2 Solid Tumor Study: The synovial sarcoma cohort of the Phase 2 trial in patients with molecularly defined solid tumors was fully enrolled in November 2016. This arm surpassed its pre-specified futility hurdle; however, the Company concluded that the activity was insufficient to continue further investigation of tazemetostat as a monotherapy. Epizyme is focusing its efforts on the remaining four cohorts of INI1-negative tumors.
Key 2017 Milestones and Goals

Interim Phase 2 Data Presentations Anticipated in the Second Quarter: Epizyme plans to report efficacy, safety and biomarker data from all five arms of the ongoing Phase 2 study in NHL, and from the study cohorts that have reached their futility analysis in the ongoing Phase 2 study in molecularly defined solid tumors. Pending abstract approval, the Company plans to present interim solid tumor data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Pending abstract submission and approval, the Company anticipates presenting interim data from all five arms of the ongoing Phase 2 NHL trial at The International Conference on Malignant Lymphoma (ICML).
Preparing for Regulatory Engagement beginning Mid-Year: Epizyme is preparing for regulatory engagement around its Phase 2 studies, beginning with the United States Food and Drug Administration (FDA) in mid-2017 on its molecularly defined solid tumor program. The Company is also preparing for FDA engagement on its NHL program in the second half of the year. These interactions are intended to determine registration paths for tazemetostat in each disease area.
Combination Studies Progressing: The combination studies of tazemetostat in front-line DLBCL with R-CHOP and in relapsed/refractory DLBCL with atezolizumab (Tecentriq) are continuing to enroll patients globally. Epizyme expects the optimal dose of tazemetostat in these combinations will be established this year, and for enrollment to continue to evaluate the safety and efficacy profile of both combinations.
Mesothelioma Study Progressing: Epizyme is continuing to enroll patients in its global Phase 2 study of tazemetostat in patients with mesothelioma characterized by BAP1 loss-of-function. The Company anticipates completing enrollment in this trial in 2017.
Next Development Candidate to be Named: Epizyme is progressing a pipeline of next-generation small molecules against novel epigenetic targets, and plans to name the next epigenetic development candidate from its pipeline in 2017. This is part of the Company’s goal of bringing three new product candidates into the clinic by 2020.
Fourth Quarter and Full-Year 2016 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $242.2 million as of December 31, 2016, as compared to $208.3 million as of December 31, 2015.
Revenue: Collaboration revenue was $0.5 million for the fourth quarter of 2016 and $8.0 million for the full year ended December 31, 2016, compared to $0.6 million for the fourth quarter of 2015 and $2.6 million for the full year ended December 31, 2015. The year-over-year increase was driven predominantly by the achievement of a $6.0 million milestone under the Company’s agreement with GSK during 2016.
R&D Expenses: Research and development (R&D) expenses were $28.4 million for the fourth quarter of 2016 and $91.5 million for the full year ended December 31, 2016, compared to $16.8 million for the fourth quarter of 2015 and $111.2 million for the full year ended December 31, 2015. R&D spending on discovery research programs and tazemetostat clinical development increased in both the quarter ended December 31, 2016 and, excluding the impact of the $40.0 million upfront payment to Eisai in the first quarter of 2015 to acquire worldwide rights, excluding Japan, to tazemetostat, in the year ended December 31, 2016.
G&A Expenses: General and administrative (G&A) expenses were $7.6 million for the fourth quarter of 2016 and $28.4 million for the full year ended December 31, 2016, compared to $6.0 million for the fourth quarter of 2015 and $23.9 million for the full year ended December 31, 2015. The increase was primarily due to higher compensation-related expenses associated with expansion of the senior leadership team in the first half of 2016 to support the Company’s operational growth, as well as increased investment in business development activities and tazemetostat-related pre-commercial activities.
Net Loss: Net loss was $35.0 million for the fourth quarter of 2016 and $110.2 million for the full year ended December 31, 2016, compared to $22.2 million for the fourth quarter of 2015 and $132.4 million for the full year ended December 31, 2015.
2017 Guidance
Epizyme believes that its cash, cash equivalents and marketable securities of $242.2 million as of December 31, 2016 will be sufficient to fund the Company’s planned operations into at least the third quarter of 2018.