On February 29, 2016 Incyte Corporation (Nasdaq: INCY) announced that they have joined the European Organization for Rare Diseases (EURODIS), the National Organization for Rare Disorders (NORD) and other rare disease health organizations and advocates in recognizing the ninth annual Rare Disease Day 2016 (Press release, Incyte, FEB 29, 2016, View Source;p=RssLanding&cat=news&id=2144192 [SID:1234509267]).

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"Rare Disease Day offers a time for patients, physicians, caregivers and advocates to join together to honor those with rare diseases across the globe, among them an estimated 200,000 individuals in the U.S. living with a group of rare blood cancers known as myeloproliferative neoplasms (MPNs)," said Steven Stein, Chief Medical Officer, Incyte Corporation.
MPNs are a closely related group of progressive blood cancers that can strike anyone at any age. MPNs occur when the bone marrow cells that produce the body’s blood cells develop and function abnormally. The three main MPNs are myelofibrosis (MF), polycythemia vera (PV) and essential thrombocythemia (ET).

This year’s Rare Disease Day theme, Patient Voice, highlights the impact patient voices have on quality care and treatment and the importance of patient and caregiver voices in the broader community to raise awareness for rare diseases. In keeping with this theme, Incyte shares story highlights from three patients from our "Voices of MPN" program.
"One of the first things I would tell someone who is newly diagnosed with a rare disease, is to be your own advocate," shares Aimee Akins, a PV patient and advocate. "I was diagnosed in October 2013 with polycythemia vera (PV). It was a long journey as I had been going to doctors since 2008. It wasn’t until I became my own advocate and looked into finding my own specialist that things really started to come together for me."

Rare Disease Day also provides an opportunity to honor patients, doctors, caregivers, advocates and organizations that contribute to bringing understanding, compassion and strength to those living with MPNs through the MPN Heroes Recognition Program 2016. Now in its fifth year, the MPN Heroes program, sponsored by Incyte in collaboration with CURE Media Group, publishers of CURE magazine, recognizes individuals and organizations who have contributed to the MPN community by going above and beyond to make a real difference. Our goal is to create as many opportunities as possible for people with MPNs to be heard and to be supported, so more solutions and treatment options will emerge. Nominations for the 2016 MPN Heroes program are now being accepted online and via email until September 8, 2016. Visit www.MPNHeroes.com for more details.

Share Your Voice and Find More Resources at Voices of MPN:
The Voices of MPN website features patients and caregivers sharing their stories; links to disease information, educational programs and patient support groups; and a "Raise Your Voice" Toolkit which provides tips on how to help spread awareness within the community.

Stay connected and informed with all of the MPN awareness activities happening throughout September by "Liking" the "Voices of MPN" Facebook page or following "Voices of MPN" on Pinterest.

On February 29, 2016 Curis, Inc. (NASDAQ:CRIS), a biotechnology company focused on the development and commercialization of innovative drug candidates for the treatment of human cancers, reported its financial results for the fourth quarter and year ended December 31, 2015 (Press release, Curis, FEB 29, 2016, View Source [SID:1234509266]).

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"2015 has been a year of significant milestones for Curis," said Ali Fattaey, Ph.D., Curis’ President and CEO. "During the fourth quarter, we presented data from the Phase 1 trial of CUDC-907 at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s annual meeting, including notable results from heavily pre-treated patients with DLBCL who experienced complete or partial responses, particularly in those with alterations of the MYC oncogene in their cancers. Based on these promising early results, in January we initiated a Phase 2 study to evaluate the efficacy of CUDC-907 in patients with relapsed/refractory DLBCL that harbor MYC alterations. There is a high unmet need for effective therapies for these patients due to their poor prognosis and a lack of optimal therapeutic options."

Dr. Fattaey continued, "We are making considerable progress with our pipeline and are on-track to file an IND application and initiate a Phase 1 clinical trial in cancer patients for CA-170, our first oral immuno-oncology drug candidate within the first half of 2016. We also expect to file an IND application for CA-4948, our IRAK4 inhibitor drug candidate, during the second half of 2016."

Full Year and Fourth Quarter 2015 Financial Results

For the year ended December 31, 2015, Curis reported a net loss of $59.0 million, or ($0.48) per basic and fully diluted share, as compared to a net loss of $18.7 million or ($0.22) per basic and fully diluted share in 2014. The 2015 net loss includes a one-time charge for in-process research and development expense of $24.3 million associated with the issuance of 17,120,131 shares of Curis common stock to Aurigene as partial consideration for the rights granted under the terms of the parties’ January 2015 collaboration agreement. For the fourth quarter of 2015, Curis reported a net loss of $13.5 million, or ($0.10) per basic and fully diluted share, as compared to a net loss of $5.7 million or ($0.07) per basic and fully diluted share for the same period in 2014.

Revenues for the year ended December 31, 2015 were $7.9 million as compared to $9.8 million for the same period in 2014. Substantially all of the Company’s revenues in 2015 and 2014 were recorded under Curis’ collaboration with Genentech. The decrease in revenues for the year ended December 31, 2015 was primarily due to a decrease in license fee revenues associated with a $3 million milestone payment earned during the year ending 2014. This decrease was partially offset by an increase in royalty revenues, which were $8.0 million and $6.8 million for the years ending 2015 and 2014, respectively.

Revenues for the fourth quarters of 2015 and 2014 were $2.1 million and $2.0 million, respectively, and were comprised almost entirely of Erivedge royalty revenues.

Operating expenses were $64.4 million for the year ended December 31, 2015, as compared to $25.7 million for the same period in 2014. Operating expenses for the fourth quarter of 2015 were $15.3 million, as compared to $6.8 million for the same period in 2014.

Costs of royalty revenues. Cost of royalty revenues, which are comprised of amounts due to third-party university patent licensors in connection with Genentech/Roche’s Erivedge net sales, were $406,000 for the year ended December 31, 2015, as compared to $340,000 for 2014. Cost of royalty revenues were $103,000 and $93,000 for the fourth quarters of 2015 and 2014, respectively.

In-process research and development expenses. The Company recorded a one-time charge for in-process research and development expense of $24.3 million during the year ended December 31, 2015 associated with the issuance of common stock to Aurigene.

Research and development expenses. Research and development expenses were $26.7 million for the year ended December 31, 2015 as compared to $13.7 million for 2014. The increase was primarily due to increases in spending on the Company’s CUDC-907 clinical development program, and preclinical programs under its collaboration with Aurigene. These increases were partially offset by decreases in spending on the Company’s other programs, including CUDC-427.

Research and development expenses were $12.0 million for the fourth quarter of 2015, as compared to $3.5 million for the same period in 2014. The Company incurred $6 million in milestone payments under the Aurigene collaboration during the fourth quarter of 2015.

General and administrative expenses. General and administrative expenses were $12.9 million for the year ended December 31, 2015 as compared to $11.7 million in 2014, and $3.2 million for each of the fourth quarters of 2015 and 2014. The increase in annual expense was primarily due to increased spending on legal costs, consulting and professional services and stock-based compensation.

Other expense was $2.5 million for the year ended 2015 and $2.9 million for the year ended 2014, and is primarily comprised of interest expense associated with the loan made by BioPharma-II to Curis Royalty, a wholly-owned subsidiary of Curis. Other expense was $215,000 for the fourth quarter of 2015, as compared to $878,000 for the fourth quarter of 2014. Interest expense was $788,000 and $914,000 for the fourth quarters of 2015 and 2014, respectively.

As of December 31, 2015, Curis’ cash, cash equivalents, marketable securities and investments totaled $82.2 million and there were approximately 129.0 million shares of common stock outstanding.

Financial Guidance

Curis expects to end 2016 with cash, cash equivalents and investments of $27 to 34 million, excluding any potential future payments from existing or new collaborators.

Curis expects that 2016 research and development expenses will be $40 to $45 million and that general and administrative expenses will be $12 to $14 million. These expense expectations include approximately $1 million and $2.5 million of estimated 2016 stock-based compensation expense in research and development and general and administrative expense, respectively, based on stock awards that are currently outstanding.

Recent Operational Highlights

CUDC-907 (oral, dual inhibitor of HDAC and PI3K enzymes):

In December 2015, Curis presented data from the Phase 1 trial of CUDC-907 at the American Society of Hematology (ASH) (Free ASH Whitepaper)’s Annual Meeting in Orlando, FL. The data demonstrated that monotherapy treatment with CUDC-907 resulted in complete (n=3) and partial responses (n=5) in heavily pretreated patients with relapsed/ refractory diffuse large B cell lymphoma (DLBCL), including those with cancers harboring alterations of the MYC oncogene, a poor performing sub-group of lymphomas for which there are no approved targeted therapies.

In January 2016, Curis initiated a Phase 2 study to evaluate the efficacy and safety of CUDC-907 with and without rituximab in patients with relapsed/ refractory MYC-altered DLBCL.

Aurigene Collaboration (Immuno-Oncology):

In October 2015, Curis exercised its option to exclusively license a first-in-class oral, small molecule antagonist of immune checkpoint proteins and designated it CA-170. The molecule targets PD-L1 and VISTA, two negative regulators of immune activation. The toxicology studies required to support the investigational new drug (IND) application for CA-170 have been completed and Curis expects to file the IND application and initiate its Phase 1 clinical development in the first half of 2016.

In November 2015, Curis’ collaborator Aurigene presented preclinical data from the CA-170 program at the 2015 AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference in Boston, MA. The presentation included data from in vitro functional studies as well as studies with isolated human T cells that demonstrated that short exposures to CA-170 are adequate to rescue and sustain activation of T cells functions in culture. In addition, daily oral administration of CA-170 resulted in anti-tumor activity in multiple syngeneic tumor models including melanoma and colon cancer, whereas no activity was observed in immune-deficient SCID-Beige mice, suggesting that the anti-cancer effects of CA-170 were mediated via activation of immune responses to these cancers.

In October 2015, Curis also selected a third program for potential further development under the collaboration, which is the second preclinical program within the immuno-oncology field and which is focused on orally available small molecules targeting PD-L1 and TIM-3 immune checkpoints. TIM-3 is an independent inhibitory checkpoint that is co-expressed with PD-1 on highly exhausted cytotoxic T cells in tumor tissues and is also expressed on certain regulatory T cells. The Company has not yet exercised its option to license this program.
Aurigene Collaboration (IRAK4 Inhibitor):

In October 2015, Curis exercised its option to exclusively license a program of orally available small molecule inhibitors of IRAK4 kinase, a serine/threonine kinase involved in innate immune responses as well as in certain hematologic cancers. The Company has since designated the development candidate as CA-4948 and expects to file an IND application for this molecule during 2016.

In November 2015, Curis’ collaborator Aurigene presented preclinical data from the IRAK4 program at the 2015 AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference in Boston, MA. This presentation included data from chemically distinct series of small molecule compounds with potent IRAK4 inhibitory activity in biochemical assays as well as in in vivo preclinical models, including MYD88 mutant DLBCL xenograft tumor models as well as a model of inflammatory disease.

Erivedge:

During the fourth quarter of 2015, Roche initiated a clinical study to evaluate the efficacy and safety of Erivedge in combination with ruxolitinib for the treatment of patients with intermediate- or high-risk myelofibrosis.

In January 2016, Roche initiated a study of Erivedge in combination with pirfenidone in patients with idiopathic pulmonary fibrosis (IPF). The study is designed as a single arm, multicenter Phase 1b study to evaluate the safety and tolerability of Erivedge in combination with pirfenidone in participants with IPF currently being treated with pirfenidone.
Upcoming Activities

Curis expects that it will make presentations at the following investor and scientific conferences through April 2016:

Cowen and Company 36th Annual Health Care Conference on March 7-9 in Boston, MA

ROTH Capital Growth Conference on March 13-16 in Los Angeles, CA

Jefferies 2016 Immuno-Oncology Summit on April 7-8 in Boston, MA

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting on April 16-20 in New Orleans, LA

On February 29, 2016 ArQule, Inc. (NASDAQ:ARQL) reported its financial results for the fourth quarter and full year of 2015 (Press release, ArQule, FEB 29, 2016, View Source [SID:1234509263]).

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For the quarter ended December 31, 2015, the Company reported a net loss of $2,852,000 or $0.05 per share, compared with net loss of $3,512,000 or $0.06 per share, for the quarter ended December 31, 2014. The Company reported a net loss of $13,774,000 or $0.22 per share, for the year ended December 31, 2015, compared with a net loss of $23,391,000 or $0.37 per share, for the year ended December 31, 2014.

At December 31, 2015, the Company had a total of approximately $38,772,000 in cash and marketable securities.

Key Highlights

Tivantinib pivotal phase 3 trial, METIV-HCC, completed enrollment and preliminary biomarker data analysis reported at ASCO (Free ASCO Whitepaper) GI: The biomarker-driven phase 3 trial for tivantinib, METIV-HCC, in second line hepatocellular carcinoma (HCC) completed patient enrollment of over 300 patients. The planned interim assessment is expected to occur by early in the second quarter of 2016. Additionally, preliminary biomarker data from the METIV-HCC trial presented at the Gastrointestinal Symposium Conference (ASCO GI) confirmed Phase 2 data demonstrating that MET status, as determined by immunohistochemistry, was more frequently high after first-line therapy.

ARQ 087 trial continues to enroll: Enrollment in the phase 2 portion of the company’s trial with its FGFR inhibitor, ARQ 087, in intrahepatic cholangiocarcinoma (iCCA) continues to enroll patients at centers in Italy and the U.S. where they are screening patients for FGFR2 fusions. As this is an open label study the company remains encouraged by the ongoing clinical benefit observed in recently enrolled patients.

ARQ 092 Phase 1 trial for Proteus syndrome completed enrollment of the first cohort: Our collaborator, the National Institutes of Health (NIH), has completed enrollment of the first cohort of the Phase 1 trial with ARQ 092 in Proteus syndrome. To date, the drug has been well tolerated. As a next step, the NIH expects to begin enrolling the second cohort including patients 12 years and older.

ARQ 092 Phase 1b trial in oncology has five partial responses (PRs), four of which are now confirmed to have AKT1/PI3K mutations: A phase 1b trial of ARQ 092 as a single agent completed enrollment in the lymphoma and endometrial cohorts. The final cohort in patients with cancers harboring the AKT1 or P13K mutations is still enrolling. We continue to monitor duration of response and safety and will provide an update on the study later in the year.

ARQ 751, next generation AKT inhibitor, received investigational new drug approval from the Food and Drug Administration (FDA): ARQ 751 expands the company’s AKT portfolio. ARQ 751 has demonstrated signal abrogation and efficacy in pre-clinical in vitro and in vivo models harboring AKT1 and PI3K mutations. A phase 1 trial in oncology is expected to begin during the first half of 2016 targeting AKT1 and PI3K mutations.

Company announced it has entered into definitive agreements with institutional and accredited investors to raise $15.3 million through a registered direct offering of common stock: Net proceeds from the offering will be used to advance ArQule’s proprietary pipeline and for general business purposes, including working capital. The proceeds are expected to fund company operations into 2018.

2016 Goals

ARQ 092 – AKT Inhibitor

Analyze phase 1 data in Proteus syndrome and make decisions on next steps
Analyze phase 1b data in oncology and make decisions on next steps
Explore opportunities in other rare over-growth diseases

ARQ 087 – FGFR Inhibitor

Complete phase 2 portion of trial in iCCA and make decisions on next steps
Complete phase 1b portion of trial in oncology and make decisions on next steps

ARQ 751 – next-generation AKT inhibitor

Initiate phase 1 trial in oncology in patients with AKT1 and PI3K mutations

Tivantinib

Complete METIV-HCC planned interim analysis by early Q2’16 and make decisions on next steps

"We are extremely pleased with the progress we made in 2015," said Paolo Pucci, Chief Executive Officer of ArQule. "From reducing our cash utilization, to progressing two proprietary compounds in three indications in the clinic, and ultimately completing enrollment of the METIV-HCC trial, it has been a productive year at ArQule. Our accomplishments in 2015 are moving us closer to being able to provide therapeutic options to patients with unmet needs through the use of precision medicine."

"2015 was a successful year for us in pipeline development," said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "We initiated two new trials within our proprietary pipeline, presented data at six scientific meetings and had two manuscripts published on ARQ 092 in peer reviewed medical journals. We are also beginning to see the value of our precision medicine approach through the clinical results we have accumulated in the ARQ 092 oncology trial and the ARQ 087 iCCA trial."

"2016 will be a year of clinical data at ArQule," said Mr. Pucci. "With the METIV-HCC interim assessment and final results on the horizon, we will have our first potential opportunity to move towards commercialization. Results from our earlier stage clinical trials, including ARQ 092 in oncology and Proteus syndrome, and ARQ 087 in iCCA, could provide us with our first opportunity to bring proprietary assets into the final stages of clinical development. We look forward to providing updates on our pipeline as the year progresses."

Revenues and Expenses

Revenues for the quarter ended December 31, 2015, were $2,797,000, compared with revenues of $3,015,000 for the quarter ended December 31, 2014. Revenues for the year ended December 31, 2015 were $11,239,000 compared with revenues of $11,254,000 for the year ended December 31, 2014. Research and development revenue in 2015 and 2014 includes revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement.

Research and development expenses in the fourth quarter of 2015 were $3,641,000, compared with $4,290,000 for the fourth quarter 2014. Fiscal 2015 research and development expenses were $15,561,000, compared with $22,271,000 for fiscal 2014.

The $0.6 million decrease in research and development expense in the fourth quarter of 2015 was primarily due to lower labor related costs of $0.2 million from reduced headcount and facility costs of $0.4 million. The $6.7 million decrease in research and development expense in fiscal 2015 was primarily due to lower labor related costs of $2.4 million from reduced headcount, outsourced clinical and product development costs of $1.7 million, facility costs of $1.9 million and lab expenses of?$0.7 million.

General and administrative expenses in the fourth quarter of 2015 were $2,028,000, compared with $2,836,000 for the fourth quarter of 2014. General and administrative expenses for fiscal 2015 were $9,830,000, compared to $12,154,000 for fiscal 2014.

General and administrative expense in the fourth quarter of 2015 decreased primarily due to lower facility costs of $0.8 million. General and administrative expense in 2015 decreased $2.3 million primarily due to lower labor related costs from reduced headcount of $0.7 million and facility costs of $1.5 million.

2016 Financial Guidance

For 2016, ArQule expects net use of cash to range between $23 and $25 million. Revenues are expected to range between $4 and $5 million. Net loss is expected to range between $24 and $27 million, and net loss per share to range between $(0.38) and $(0.43) for the year. ArQule expects to end 2016 with between $29 and $31 million in cash and marketable securities, inclusive of the $15.2 million of funds expected to be received upon the closing of the recent stock offering.