On May 10, 2016 CTI BioPharma Corp. (NASDAQ and MTA:CTIC) reported financial results for the first quarter ended March 31, 2016 (Press release, CTI BioPharma, MAY 10, 2016, View Source [SID:1234512209]).

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"We continue to believe in the potential of pacritnib to help patients in need and are working to address the clinical hold on the pacritinib program," said James A. Bianco, M.D., President and Chief Executive Officer of CTI BioPharma. "While we work with the FDA to seek to address their recommendations for getting pacritinib off hold, we have made progress in our efforts to support patients who were deriving benefit from pacritinib at the time of the clinical hold by providing pacritinib under a single patient IND program and, separately, to individual patients for "compassionate use" under the FDA’s emergency Expanded Access program. We are pleased that certain investigator-sponsored trials can resume as the agency has removed the clinical hold at their sites."

"We are also preparing for the release of top-line results from the PERSIST-2 Phase 3 clinical trial, which we expect to report in the third quarter of 2016," added Dr. Bianco.

First Quarter and Recent Events

In the first quarter, patient enrollment was completed in the PERSIST-2 Phase 3 clinical trial of pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis. PERSIST-2 is evaluating pacritinib for patients with myelofibrosis whose platelet counts are less than or equal to 100,000 per microliter (≤100,000/μL). In February, the U.S. Food and Drug Administration (FDA) placed a full clinical hold on pacritinib clinical studies, and as such, all patients participating in the PERSIST-2 clinical trial discontinued pacritinib treatment. Although not all patients enrolled were able to reach the primary endpoint evaluation at 24 weeks from randomization prior to the full clinical hold on pacritinib, approximately two thirds of the enrolled patients reached or exceeded the 24-week endpoint evaluation. The Company believes there is sufficient power to reach statistical significance of the primary objectives. Top-line results from the PERSIST-2 Phase 3 trial of pacritinib are expected in the third quarter of 2016.
In March 2016, the FDA expressed interest in allowing patients who were receiving benefit from pacritinib treatment at the time the clinical hold was imposed to submit requests to the FDA to resume pacritinib treatment under a Single Patient IND (SPI) program on a case-by-case basis. The Company has been working with investigators in submitting SPI requests to the FDA. Separately, the FDA has informed clinical investigators that emergency requests may be submitted to the FDA for individual patient Expanded Access to pacritinib. Expanded Access, sometimes called "compassionate use," is the use outside of a clinical trial of an investigational medical product. The Company recently learned that the FDA has released the clinical hold on certain investigator-sponsored trials that were evaluating pacritinib.
In April 2016, researchers presented findings from an investigator-sponsored preclinical study indicating that pacritinib, an inhibitor of JAK2, FLT3, IRAK1 and CSF1R may potentially be effective in reducing survival of myelofibrosis and acute myeloid leukemia (AML) repopulating cells. Further, this study also demonstrated that the combination of pacritinib at low nanomolar concentrations with dasatinib may eliminate self-renewing leukemia stem cells in blast crisis of chronic myeloid leukemia (CML) with minimal toxicity toward normal progenitors. These and other findings were presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.
First Quarter Financial Results

Total revenues for the first quarter ended March 31, 2016, were $36.5 million compared to $2.7 million for the same period in 2015. The increase in total revenue for the first quarter of 2016 is primarily due to recognition of $32 million in milestone payments related to pacritinib. The Company had previously received a cash advance for these milestone payments in the second quarter of 2015 that was accounted for as long-term debt until the achievement of the associated milestones in the first quarter of 2016. Additionally, net product revenues of PIXUVRI for the first quarter ended March 31, 2016 increased to $1.2 million compared to $0.8 million for the same period in 2015.

GAAP operating income for the first quarter ended March 31, 2016, was $4.1 million compared to GAAP operating loss of $27.5 million for the same period in 2015. Non-GAAP operating income, which excludes non-cash share-based compensation expense, for the first quarter ended March 31, 2016, was $8.0 million compared to the non-GAAP operating loss of $23.1 million for the same period in 2015. The Company’s operating income for the first quarter ended March 31, 2016, as compared an operating loss for the same period in 2015, is primarily due to recognition of $32 million in milestone payments related to pacritinib as mentioned above. Non-cash share-based compensation expense for the first quarter ended March 31, 2016, was $3.8 million compared to $4.3 million for the same period in 2015. For information on CTI BioPharma’s use of this non-GAAP measure and a reconciliation of such measure to GAAP operating loss, see the section below entitled "Non-GAAP Financial Measures."

Net income for the first quarter of 2016 was $3.3 million, or $0.01 per share, compared to a net loss of $28.6 million, or ($0.16) per share, for the same period in 2015.

As of March 31, 2016, cash and cash equivalents totaled $104.6 million, compared to $128.2 million as of December 31, 2015.

On May 10, 2016 ARIAD Pharmaceuticals, Inc. (NASDAQ: ARIA) reported financial results for the first quarter of 2016, including revenue from sales of Iclusig (ponatinib) (Press release, Ariad, MAY 10, 2016, View Source [SID:1234512197]).

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The Company also provided an update on corporate developments and its ongoing strategic review and provided financial guidance following its announced transaction with Incyte Corporation.

"Iclusig demonstrated strong performance in both the U.S. and European markets during the first quarter of 2016 compared to the prior year period, primarily driven by increasing demand and new patient growth," said Paris Panayiotopoulos, president and chief executive officer of ARIAD. "Following our major announcement yesterday with Incyte regarding our agreement to divest our European operations and license the commercial rights to Iclusig in Europe, we are on track to complete our strategic review this quarter aimed at delivering patient and shareholder value. We also look forward to the presentation of pivotal, registration data on brigatinib at ASCO (Free ASCO Whitepaper), along with our planned filing for marketing approval of brigatinib in the U.S. in the third quarter of this year."

2016 First Quarter Financial Results

Revenues

Net product revenues from sales of Iclusig were $33.6 million for the first quarter of 2016, compared to $23.9 million in the first quarter of 2015, representing growth of 41%. Excluding the impact of one-time revenue of $1.2 million recognized in Q1 2015 in connection with our transition to the sell-in method in the U.S., worldwide growth would have been 48%.
U.S. sales of Iclusig were $24.9 million for the first quarter of 2016, compared to $18.7 million in the first quarter of 2015, representing growth of 33%. Excluding the impact of one-time revenue of $1.2 million recognized in Q1 2015 in connection with our transition to the sell-in method, U.S. growth would have been 42%.
European sales of Iclusig were $8.7 million for the first quarter of 2016, compared to $5.2 million in the first quarter of 2015, representing growth of 67%. This difference is primarily due to increased demand and the launch of Iclusig in new countries.
GAAP and Non-GAAP Net Loss

GAAP net loss for the quarter ended March 31, 2016 was $53.8 million, or $0.28 loss per share, compared to GAAP net loss of $52.7 million, or $0.28 loss per share, for the quarter ended March 31, 2015.

Non-GAAP net loss for the quarter ended March 31, 2016 was $44.1 million, or $0.23 loss per share, compared to non-GAAP net loss of $44.2 million, or $0.24 per share for the quarter ended March 31, 2015.

Non-GAAP net loss excludes stock-based compensation and restructuring charges. See "Use of Non-GAAP Financial Measures" below for a description of non-GAAP financial measures and the reconciliation between GAAP and non-GAAP measures at the end of this press release.

Operating Expenses

GAAP R&D expenses were $44.1 million for the first quarter of 2016, an increase of 12% compared to the first quarter of 2015, reflecting an increase in costs for our investigational ALK+ inhibitor, brigatinib, related to the ongoing Phase 2 ALTA trial and NDA-enabling pre-clinical studies, as well as increase in personnel and other costs in support of our R&D activities.
GAAP selling, general and administrative expenses were $36.0 million for the first quarter of 2016, an increase of 7% compared to the first quarter of 2015, reflecting an increase in professional fees and other expenses related to the commercialization of Iclusig.
Restructuring charge expenses were $2.9 million for the first quarter of 2016, associated with employee workforce reductions of approximately 90 positions.
Cash Position

As of March 31, 2016, cash, cash equivalents and marketable securities totaled $168.3 million, compared to $242.3 million at December 31, 2015.
2016 Financial Guidance

Following the anticipated closing of the transaction announced yesterday in which ARIAD has agreed to sell its European operations and license commercial rights to Iclusig in Europe to Incyte, we are revising our product revenue guidance for 2016 and providing expense and year-end cash guidance for 2016, as follows:
Global Iclusig net product and royalty revenues are expected to be in the range of $170 million to $180 million, compared to previous guidance of $190 million to $200 million, reflecting a reduction in European product revenue and the addition of royalty revenue following the Incyte transaction. Of the $170 million to $180 million, approximately $162 million to $170 million consists of expected product revenue, and $8 million to $10 million consists of expected royalty revenue.
Research and development and sales, general and administration expense are expected to be as follows:
$175 million to $180 million for research and development expense
$120 million to $125 million for sales, general and administration expense
This new 2016 expense guidance reflects an expected reduction in expenses of approximately $30 million from the planned divesture of our European business to Incyte.

Cash position at December 31, 2016 is expected to be in the range of $280 million to $290 million, including the $140 million upfront payment from Incyte and $50 million to be received under our royalty financing agreement with PDL.
The foregoing 2016 financial guidance reflects the anticipated completion of the transaction with Incyte, which is expected to close on or about June 1, 2016, subject to customary closing conditions. In addition, the revenue guidance assumes that pricing and reimbursement approval in France (and the corresponding recognition of revenue) will occur prior to the closing of the Incyte transaction.
Recent Progress and Key Objectives

Iclusig Clinical Development

Patient enrollment is ongoing in the OPTIC and OPTIC-2L clinical trials in patients with resistant chronic-phase chronic myeloid leukemia (CP-CML)
Otsuka Pharmaceutical Co., Ltd. (Otsuka) submitted a new drug application (NDA) to the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) seeking approval for Iclusig for the treatment of resistant or intolerant CML and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ALL). This marketing application was submitted in early 2016 and is expected to lead to an initial approval of Iclusig in Japan by the end of this year.
Brigatinib Clinical Development

The ALTA 1L randomized, front-line clinical trial of brigatinib opened to patient enrollment in early April and is now underway. This global, Phase 3 trial is designed to compare brigatinib and crizotinib in patients with ALK+ non-small cell lung cancer (NSCLC), who have not received prior ALK inhibitors.
Clinical data from the Phase 2 ALTA trial of brigatinib has been accepted for oral presentation at this year’s annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June, 2016. We are on track to file for approval of brigatinib in the U.S. in the third quarter of this year.
Advancing the Pipeline

Preclinical data on AP32788 were presented at last month’s American Association of Clinical Research meeting and a Phase 1/2 proof-of-concept clinical trial is now open to patient enrollment.
AP32788 targets tumors driven by EGFR or HER2 kinases and was designed to achieve selective inhibition of exon 20 mutations in these kinases. ARIAD estimates that there are approximately 6,000 patients in the United States living with EGFR exon 20 or HER2 point mutations.

Upcoming Meetings

American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting, Chicago, June 3 to June 7, 2016
Jefferies Healthcare Conference, New York City, June 7-10, 2016
European Hematology Association (EHA) (Free EHA Whitepaper) 20th Congress, Austria, Vienna June 9 to 12, 2016
ARIAD Analyst and Investor Day, New York City, June 17, 2016
Today’s Conference Call at 8:30 a.m. ET

We will hold a live webcast and conference call of our first quarter 2016 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 84030796. 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.
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.

On May 10, 2016 MacroGenics, Inc. (Nasdaq: MGNX) reported that its collaboration partner, Pfizer Inc. (NYSE: PFE), has advanced a bispecific antibody therapeutic candidate generated by MacroGenics’ Dual-Affinity Re-Targeting, or DART, platform (Press release, MacroGenics, MAY 10, 2016, View Source [SID:1234512178]).

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Pfizer recently dosed a first patient in the Phase 1 clinical study of PF-06671008, which targets P-cadherin and CD3. Increased levels of the protein P-cadherin have been reported in various tumors, including breast, ovarian, endometrial, colorectal and pancreatic cancers, and is correlated with poor survival of patients. The commencement of the Phase 1 study triggers a $2 million milestone payment to MacroGenics under the companies’ October 2010 agreement.

PF-06671008 is the first partner-developed DART molecule to enter clinical development and represents the sixth DART molecule in clinical testing. At present, MacroGenics’ clinical pipeline includes multiple DART candidates for the treatment of cancer and one DART candidate for the treatment of autoimmune disorders.

Background on DART Platform

MacroGenics’ DART platform enables the targeting of multiple antigens or cells by using a single molecule with an antibody-like structure. DART molecules can be configured for the potential treatment of cancer, autoimmune disorders and infectious diseases. These DART molecules can be tailored for either short or prolonged pharmacokinetics and have demonstrated good stability and attractive manufacturability. Six DART molecules, including programs being developed by MacroGenics and its collaborators, are currently being evaluated in clinical studies.