10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Incyte, NOV 3, 2015, View Source [SID:1234507927])

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Endocyte Reports Third Quarter 2015 Financial Results and Provides Update on Clinical Progress

On November 3, 2015 Endocyte, Inc. (NASDAQ:ECYT), a leader in developing targeted small molecule drug conjugates (SMDCs) and companion imaging agents for personalized therapy, reported financial results for the third quarter ending Sept. 30, 2015, and provided a clinical update (Press release, Endocyte, NOV 3, 2015, View Source [SID:1234507923]).

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"Our Phase 1 dose escalation trials of the folate receptor (FR)- and prostate-specific membrane antigen (PSMA)-targeted tubulysin SMDCs, EC1456 and EC1169, continue to progress well. Both SMDCs are being dose escalated on two different schedules," said Ron Ellis, Endocyte’s president and chief executive officer. "Once we have determined the maximum tolerated dose and optimal schedule for each SMDC, we look forward to moving into expansion cohorts where we will evaluate these SMDCs specifically in receptor-positive patients among different tumor types, both as single agents and in combination with standard of care drugs."

Earlier in the third quarter, Endocyte announced the final results from the Phase 2b TARGET trial evaluating its SMDC vintafolide in combination with docetaxel in patients with FR positive recurrent non-small cell lung cancer (NSCLC) at the World Conference on Lung Cancer in Denver, Colorado. Vintafolide plus docetaxel improved overall survival (OS) by 2.7 months in NSCLC regardless of histology (Median OS 11.5 vs. 8.8 months, OS HR=0.86, 95% CI [0.58, 1.26]). In the predefined subset analysis of patients with adenocarcinoma, which expresses higher levels of FR, vintafolide plus docetaxel improved OS by 5.9 months (12.5 vs. 6.6 months, HR=0.72, 95% CI [0.44, 1.16]). This data is in line with earlier findings from the study of improved progression free survival and higher overall response rates. Final safety results were also consistent with those previously reported.

Third Quarter 2015 Financial Results

Endocyte reported a net loss of $10.0 million, or $0.24 per basic and diluted share, for the third quarter of 2015, compared to a net loss of $5.7 million, or $0.14 per basic share and diluted share, for the same period in 2014. The third quarter of 2014 included the recognition of $3.9 million of revenue related to the former collaboration with Merck.

Research and development expenses were $6.6 million for the third quarter of 2015, compared to $5.7 million for the same period in 2014. The increase in research and development expense was primarily attributable to an increase in expenses for the EC1456 and EC1169 dose escalation trials, the drug manufacturing expense of EC1456, and an increase in compensation expense, including noncash stock compensation, partially offset by the decrease in manufacturing expenses of etarfolatide, our folate-targeted companion molecular imaging agent to vintafolide and EC1456.

General and administrative expenses were $3.8 million for the third quarter of 2015, compared to $4.0 million for the same period in 2014. The decrease in expenses was primarily attributable to a reduction in legal and patent fees, slightly offset by an increase in compensation expense, including noncash stock compensation.

Cash, cash equivalents and investments were $180.3 million at September 30, 2015, compared to $211.1 million at September 30, 2014, and $206.8 million at December 31, 2014.

Financial Expectations

The Company updated its 2015 financial expectations and now anticipates that its 2015 year-end cash balance will be approximately $170 million.

10-Q – Quarterly report [Sections 13 or 15(d)]

(Filing, 10-Q, Rigel, NOV 3, 2015, View Source [SID:1234507933])

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ARIAD Reports Third Quarter 2015 Financial Results and Progress on Strategic Objectives

On November 3, 2015 ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) reported financial results for the third quarter of 2015, including revenue from sales of Iclusig (ponatinib) (Press release, Ariad, NOV 3, 2015, View Source;p=RssLanding&cat=news&id=2105752 [SID:1234507898]). The Company also provided an update on key corporate initiatives and clinical-trial plans.

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"We are on track to achieve our product revenue guidance of $130 to $140 million for 2015, with important contributions coming from European and U.S. sales and anticipated successful resolution of pricing negotiations for Iclusig in France," stated Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. "Each of the major clinical initiatives – outlined at the beginning of 2015 as part of our three-year strategic operating plan – is progressing as projected. These include full patient enrollment in the ALTA pivotal trial of brigatinib, ongoing enrollment of patients in the dose-ranging OPTIC trial of Iclusig and anticipated initiation before year-end of the second-line trial of Iclusig. We expect that results from these randomized trials, if positive, will form the basis for expanded utilization of Iclusig and initial approval of brigatinib. We are also moving ahead with another randomized trial – a comparison of brigatinib and crizotinib in front-line non-small cell lung cancer – which we anticipate beginning in the first half of next year."

2015 Third Quarter Financial Results

Revenues

Net product revenues from sales of Iclusig were $27.5 million for the third quarter, compared to $14.5 million in the third quarter of 2014 and $27.8 million in the second quarter of 2015.

U.S. sales of Iclusig were $20.3 million for the third quarter, compared to $21.6 million in the second quarter of 2015, the difference primarily due to flat quarter-over-quarter demand and an increase in gross-to-net adjustments.

European sales of Iclusig were $7.2 million for the third quarter, compared to $6.2 million in the second quarter of 2015, the difference primarily due to increased quarter-over-quarter demand.

Shipments of Iclusig to patients in France were $2.2 million for the third quarter of 2015. Cumulative total shipments in France totaled $23.3 million through September 30, 2015. We will record revenue related to cumulative shipments in France upon completion of pricing and reimbursement negotiations in France, net of any amounts that will be refunded to the French health authorities as a result of these negotiations, which we anticipate will be completed by year-end 2015.

Operating Expenses

Research and development (R&D) expenses were $48.2 million for the third quarter of 2015, an increase of 75% compared to the third quarter of 2014. This reflects an increase in costs for our ongoing Phase 2 ALTA trial of brigatinib, and NDA-enabling pharmacology and manufacturing activities, costs related to the initiation of the Iclusig Phase 2 dose-ranging OPTIC trial, as well as an increase in personnel and other costs in support of our continuing R&D activities.
Selling, general and administrative (SG&A) expenses were $36.7 million for the third quarter of 2015, an increase of 9% compared to the third quarter of 2014. This reflects an increase in personnel costs, including the impact of severance costs associated with the pending retirement of our chief executive officer and an increase in costs in support of expanding distribution and sales of Iclusig.

Net Loss

Net loss for the quarter ended September 30, 2015 was $55.5 million, or $0.29 per share, compared to a net loss of $50.1 million, or $0.27 per share, for the same period in 2014.
During the quarter ended September 30, 2015, we recorded in marketable securities on our balance sheet the value of shares of common stock we own in REGENXBIO, Inc., which completed an initial public offering during the quarter; these shares are not saleable until the first quarter of 2016. The value of the shares at September 30, 2015 was $15.1 million. The value net of tax, $9.1 million, is recorded in other comprehensive income. An intra-period tax benefit of $6.0 million related to the tax consequences of this item included in other comprehensive income is recorded as a tax benefit in our statement of operations.

Cash Position

As of September 30, 2015, cash and cash equivalents totaled $282.2 million, compared to $352.7 million at December 31, 2014.
During the quarter ended September 30, 2015, we entered into a royalty financing agreement with PDL BioPharma, Inc. (PDL) under which we received $50 million from PDL in exchange for payments to PDL based on a percentage of global sales of Iclusig over time until PDL receives a 10% internal rate of return. In July 2016, we will receive an additional $50 million from PDL. Under the agreement, we also have an option, in our sole discretion, to receive up to an additional $100 million through July 2016.
Recent Progress and Key Objectives

Commercialization of Iclusig

Approximately 125 new patients were treated with Iclusig in the U.S. during the third quarter of 2015. Importantly, approximately 55% of these new patients receiving Iclusig are patients with chronic-phase chronic myeloid leukemia (CP-CML).
Through the third quarter, there have been approximately 980 unique prescribers of Iclusig in the U.S., an increase in the cumulative prescriber base of approximately 13% from the second quarter of 2015.
In Europe, we are now promoting Iclusig in 12 countries: the United Kingdom, France, Germany, Italy, Austria, Switzerland, The Netherlands, Luxembourg, Denmark, Norway, Sweden, and Finland. In addition, Iclusig is available for purchase and is being supplied through named-patient programs and prior authorizations in Spain, Portugal, Ireland, Turkey and in several markets in Eastern Europe.

Iclusig is now reimbursed in Canada and Australia, and commercial launches continue in both countries through our regional distribution partners.

Iclusig Clinical Development

In August, we initiated patient enrollment in the OPTIC (Optimizing Ponatinib Treatment In CML) trial of Iclusig. This randomized, dose-ranging trial is designed to evaluate three different starting doses of ponatinib in patients with refractory CP-CML and is expected to inform the optimal use of Iclusig in these patients. Approximately 450 patients will be enrolled at clinical sites around the world.

Later this quarter, we expect to begin a Phase 3 trial of Iclusig in patients with CP-CML, who have experienced treatment failure after imatinib therapy. This second-line study of Iclusig is expected to enroll approximately 600 patients and is aimed at expanding the indication for Iclusig in patients with resistant and intolerant CML.

Brigatinib Clinical Development

In September, we achieved full patient enrollment in the pivotal Phase 2 ALTA trial of brigatinib. This registration study enrolled approximately 220 patients at 71 sites in North America, Europe and Asia. We are on track to file in the third quarter of 2016 for approval of brigatinib in the U.S.

The randomized front-line clinical trial of brigatinib is expected to begin in the first half of 2016. This Phase 3 trial will compare brigatinib and crizotinib in approximately 300 patients with ALK+ non-small cell lung cancer (NSCLC), who have not received prior ALK inhibitors.

Advancing the Pipeline

We are on track to file an investigational new drug (IND) application for AP32788 by year-end 2015 and to begin a Phase 1/2 proof-of-concept clinical trial in 2016. AP32788 is an orally active tyrosine-kinase inhibitor (TKI), which has a unique profile against a validated class of mutated targets in NSCLC and certain other solid tumors and may address an important unmet medical need.
Today’s Conference Call at 8:30 a.m. ET

We will hold a live webcast and conference call of our third quarter 2015 financial results this morning at 8:30 a.m. ET. The live webcast can be accessed by visiting the investor relations section of the Company’s website at View Source The call can be accessed by dialing 888-311-8173 (domestic) or 330-863-3376 (international) five minutes prior to the start time and providing the pass code 55634805. A replay of the call will be available on the ARIAD website approximately two hours after completion of the call and will be archived for three weeks.

About Iclusig (ponatinib) tablets

Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug-design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

Iclusig is approved in the U.S., EU, Australia, Switzerland, Israel and Canada.

In the U.S., Iclusig is a kinase inhibitor indicated for the:

Treatment of adult patients with T315I-positive chronic myeloid leukemia (chronic phase, accelerated phase, or blast phase) or T315I-positive Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL).
Treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia or Ph+ ALL for whom no other tyrosine kinase inhibitor (TKI) therapy is indicated.
These indications are based upon response rate. There are no trials verifying an improvement in disease-related symptoms or increased survival with Iclusig.

IMPORTANT SAFETY INFORMATION, INCLUDING THE BOXED WARNING

WARNING: VASCULAR OCCLUSION, HEART FAILURE, and HEPATOTOXICITY

See full prescribing information for complete boxed warning

Vascular Occlusion: Arterial and venous thrombosis and occlusions have occurred in at least 27% of Iclusig treated patients, including fatal myocardial infarction, stroke, stenosis of large arterial vessels of the brain, severe peripheral vascular disease, and the need for urgent revascularization procedures. Patients with and without cardiovascular risk factors, including patients less than 50 years old, experienced these events. Monitor for evidence of thromboembolism and vascular occlusion. Interrupt or stop Iclusig immediately for vascular occlusion. A benefit risk consideration should guide a decision to restart Iclusig therapy.

Heart Failure, including fatalities, occurred in 8% of Iclusig-treated patients. Monitor cardiac function. Interrupt or stop Iclusig for new or worsening heart failure.

Hepatotoxicity, liver failure and death have occurred in Iclusig-treated patients. Monitor hepatic function. Interrupt Iclusig if hepatotoxicity is suspected.

Please see the full U.S. Prescribing Information for Iclusig, including the Boxed Warning, for additional important safety information.

8-K – Current report

On November 3, 2015 Fate Therapeutics, Inc. (NASDAQ: FATE), a biopharmaceutical company dedicated to the development of programmed cellular immunotherapeutics for the treatment of cancer and immune disorders, reported business highlights and financial results for the third quarter ended September 30, 2015 (Filing, 8-K, Fate Therapeutics, NOV 3, 2015, View Source [SID:1234507925]).

"We have firmly established a leadership position in a unique and broadly applicable strategy for cancer immunotherapy — the production of T cells and NK cells from pluripotent cells, bringing an off-the-shelf approach to the field of cell-based immunotherapies," said Scott Wolchko, Chief Operating and Financial Officer of Fate Therapeutics. "Additionally, our clinical experience with PROHEMA, preclinical studies with PROTMUNE and research collaborations with Juno Therapeutics and the University of Minnesota all provide compelling support that the administration of programmed immune cells to patients fighting cancer will serve as a cornerstone treatment paradigm."

Recent Highlights & Upcoming Milestones

· Off-the-Shelf Cancer Immunotherapy Strategy to be Presented at ASH (Free ASH Whitepaper) 2015 Annual Meeting. The Company’s patent-protected pluripotent cell platform combines genetic engineering of pluripotent cells with rapid and efficient generation of immune cells, enabling production of off-the-shelf engineered T- and NK-cell-based therapeutics without requiring patient-sourced cells. Fate plans to present its novel strategy for developing off-the-shelf cancer immunotherapies using its pluripotent cell platform during two poster sessions at the American Society of Hematology (ASH) (Free ASH Whitepaper) 2015 Annual Meeting.

· NK-Cell Cancer Immunotherapeutic Undergoing Preclinical Development. In July 2015, Fate entered into a collaboration with the University of Minnesota to enable clinical development of a novel population of "adaptive" NK cells, which exhibit prolonged persistence and enhanced anti-tumor activity mediated through CD16 signaling in preclinical studies. The Company’s development strategy seeks to use "adaptive" NK cells in combination with solid tumor-targeting antibodies to induce potent killing of cancer cells.

· PROTMUNE IND Filing Planned. The Company expects to initiate a first-in-human clinical trial in 2016 to investigate the potential of PROTMUNE to prevent the life-threatening complications of acute graft-versus-host disease (GvHD) and severe infections in patients undergoing mobilized peripheral blood (mPB) transplantation. During an ASH (Free ASH Whitepaper) 2015 Annual Meeting poster session, Fate plans to present scientific findings showing that a single administration of programmed peripheral blood cells resulted in a statistically-significant reduction in GvHD score and improvement in survival as compared to vehicle-treated peripheral blood cells in preclinical models.

· PUMA Study Reaches 70% of Target Enrollment. Fate is currently preparing a second interim data-cut from its ongoing Phase 2 PUMA study of PROHEMA in adult patients undergoing double umbilical cord blood transplantation for the treatment of hematologic malignancies. The Company expects to report additional data on neutrophil engraftment and severe infection-related adverse events from the PUMA study during the 2015 ASH (Free ASH Whitepaper) Annual Meeting.

· Leadership Transition. On October 12, 2015, the Company announced that Scott Wolchko, a Fate founder and the Company’s Chief Operating & Financial Officer, will succeed Christian Weyer, M.D., M.A.S., as President and Chief Executive Officer, effective December 1. The Company also announced that Stewart Abbot, Ph.D. has been named Chief Development Officer after joining Fate earlier this year from Celgene Cellular Therapeutics, where he was instrumental in developing the company’s hematopoietic cell-based immuno-oncology programs and partnerships. Fate also announced the promotions of Daniel Shoemaker, Ph.D., who joined the Company in 2009, to Chief Scientific Officer, and Cindy Tahl, J.D., who joined the Company in 2009, to General Counsel.

Financial Results

· Cash Position: Cash and cash equivalents as of September 30, 2015 were $72.9 million, compared to $49.1 million as of December 31, 2014. The increase is primarily driven by net proceeds from the Company’s public offering of common stock in May 2015 and cash generated from entering into a research collaboration and license agreement with Juno Therapeutics in May 2015, offset by cash used to fund operating activities.

· Total Revenue: Revenue was $1.0 million for the third quarter of 2015, which was derived from the Company’s collaboration with Juno.

· Total Operating Expenses: Total operating expenses were $7.4 million for the third quarter of 2015, compared to $6.0 million for the third quarter of 2014. Operating expenses for the third quarter of 2015 include $0.6 million of stock compensation expense, compared to $0.5 million for the third quarter of 2014.

· R&D Expenses: Research and development expenses were $5.0 million for the third quarter of 2015, compared to $4.1 million for the third quarter of 2014. The increase in R&D expenses is primarily related to an increase in third-party professional consultant and service provider expenses to support the clinical development of PROHEMA, and an increase in personnel expense, including stock-based compensation expense, resulting from additional headcount to support the conduct of research activities.

· G&A Expenses: General and administrative expenses were $2.4 million for the third quarter of 2015, compared to $1.9 million during the third quarter of 2014. The increase in G&A expenses is primarily related to an increase in personnel expense, including stock-based compensation expense.

· Common Shares Outstanding: Common shares outstanding as of September 30, 2015 were 28.7 million compared to 20.6 million as of December 31, 2014. Common shares outstanding increased primarily as a result of the 6.9 million shares of the Company’s common stock issued pursuant to the May 2015 financing, and the 1.0 million shares of the Company’s common stock issued and sold to Juno pursuant to the collaboration.

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