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

Galectin Therapeutics has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Galectin Therapeutics, MAY 11, 2015, View Source [SID1234504181]).

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Provenge – Withdrawal of the marketing authorisation in the European Union

On 6 May 2015, the European Commission withdrew the marketing authorization for Provenge in the European Union (EU) (European Medicines Agency, MAY 11, 2015, View Source [SID:1234511747]). The withdrawal was at the request of Dendreon, which notified the European Commission of its decision to permanently discontinue the marketing of the product for commercial reason.

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Prima BioMed announces Japanese Collaboration

On May 11, 2015 Prima BioMed reported a collaboration with NEC Corporation and Yamaguchi University for Immutep’s IMP321 in combination with a peptide vaccine developed by NEC and Yamaguchi University (Press release, Prima Biomed, MAY 11, 2015, View Source [SID:1234508542]).
The preclinical study, to be conducted at Yamaguchi University and supported by NEC, will investigate the use of antigen presenting cell activator IMP321 as an adjuvant, together with peptide antigens believed to be involved in hepatocellular cancer.
Marc Voigt, Chief Executive of Prima, said: "We are very pleased to be able to assist NEC with this preclinical study. The use of IMP321 as an adjuvant to a vaccine is different to the other combination approaches being used for our planned clinical trials. The opportunity to broaden the application of IMP321 for other uses is exciting."
Yamaguchi University President Dr Masaaki Oka, MD, commented: "We look forward to working on this program and exploring the use of IMP321 together with our vaccine. We believe that this combination could offer a unique treatment opportunity. "
Osamu Fujikawa, General Manager, Corporate Business Development Division, NEC Corporation said: "It is exciting for our company to be able to support and oversee the study of this new combination. We hope to have good results in this study which would allow us to advance to the next step."

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About Prima BioMed
Prima BioMed is a globally active biotech company developing immunotherapeutic products for the treatment of cancer. Prima is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximize value for its shareholders. Prima owns a number of different products in preclinical and clinical development. www.primabiomed.com.au

About Immutep
Immutep is a 100% subsidiary of Prima BioMed Ltd. Founded in 2001 by Prof. Frédéric Triebel and John Hawken. Immutep is a French immunotherapy company developing a suite of products based on its Lymphocyte Activation Gene 3 (LAG–‐3) technology. Immutep’s lead product IMP321 works by binding to a receptor on antigen presenting cells (APCs) such as dendritic cells to activate them. Prima BioMed Ltd, Level 7, 151 Macquarie Street, Sydney NSW 2000 Phone: +61 2 9276 1224 Fax: +61 2 9276 1284 www.primabiomed.com.au ABN: 90 009 237 889 Immutep’s other products include IMP701, an antagonist antibody that acts to stimulate T cell proliferation in cancer patients, licensed to CoStim (Novartis) and IMP731, a depleting antibody that removes T cells involved in autoimmunity, licensed to GSK. It also has a dedicated R&D laboratory outside Paris.

About NEC
NEC Corporation is a leader in the integration of IT and network technologies that benefit businesses and people around the world. By providing a combination of products and solutions that cross utilize the company’s experience and global resources, NEC’s advanced technologies meet the complex and ever–‐changing needs of its customers. NEC brings more than 100 years of expertise in technological innovation to empower people, businesses and society. For more information, visit NEC at View Source

About Yamaguchi University
Founded in 1949 and located in Yamaguchi Prefecture in southern Japan, Yamaguchi University is a regional center of academic research as well as higher education and in the Japanese national university system. The University is known for basic and applied research in a wide variety of fields.

Ligand Reports First Quarter 2015 Financial Results

On May 11, 2015 Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) reported financial results for the three months ended March 31, 2015, and provided an operating forecast and program updates (Press release, Ligand, MAY 11, 2015, View Source [SID:1234506621]).

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Financial highlights for the first quarter of 2015 include:

Total revenues were $14.6 million and royalty revenues were $10.3 million, an increase of 31% compared with the first quarter of 2014
Adjusted EPS was $0.33 and GAAP EPS was $0.04
The company generated operating cash of $7.6 million, a significant increase over $2.6 million of operating cash generated in the first quarter of 2014
A description of adjusted calculations and reconciliation to comparable GAAP financial measures is provided in the accompanying table titled "Adjusted Financial Measures."

"First quarter financial results feature continued strong growth in royalty revenues from both of our lead commercial assets, Promacta and Kyprolis, and strong growth in operating cash flow," said John Higgins, President and Chief Executive Officer of Ligand. "Our growing roster of partners made important clinical and regulatory progress with many of our programs, both in the U.S. and in Europe and including orphan and large-market indications. Notably, Promacta has now transitioned from GSK to the highly capable stewardship of Novartis, and Amgen reported favorable clinical trial results with Kyprolis in a head-to-head comparison versus Velcade in patients with relapsed multiple myeloma. We also marked the successful IPO of a key partner and acquired a portfolio of royalty-bearing programs. The team at Ligand is executing extremely well and we are very pleased with our performance as we move into mid-2015."

First Quarter 2015 Financial Results

Total revenues for the first quarter of 2015 were $14.6 million, compared with $16.0 million for the same period in 2014. Royalty revenues increased 31% to $10.3 million from $7.9 million for the same period in 2014 primarily due to higher royalties from Promacta and Kyprolis. Material sales were $3.7 million compared with $5.7 million for the same period in 2014 due to the timing of Captisol purchases for use in clinical trials. Collaborative research and development and other revenues were $0.6 million compared with $2.4 million for the same period in 2014 due primarily to significant milestones earned in the first quarter of 2014 from Merck for approval of Noxafil and from Amgen for a Kyprolis sales milestone.

Cost of goods sold decreased to $1.1 million for the first quarter of 2015 from $2.5 million for the same period in 2014, as a result of lower material sales. Research and development expenses were $4.0 million compared with $3.1 million for the same period of 2014. The increase was primarily due to costs associated with funding internal development programs. General and administrative expenses for the first quarter of 2015 were $6.0 million, compared with $5.1 million for the same period in 2014.

Net income for the first quarter of 2015 was $0.8 million, or $0.04 per diluted share, compared with net income for the first quarter of 2014 of $2.1 million, or $0.10 per diluted share. Adjusted net income from continuing operations for the first quarter of 2015 was $6.9 million, or $0.33 per diluted share, compared with adjusted net income from continuing operations for the first quarter of 2014 of $7.3 million, or $0.35 per diluted share.

As of March 31, 2015, Ligand had cash, cash equivalents, short-term investments and restricted investments of $177.8 million.

Full-Year and Second Quarter 2015 Financial Forecast

Ligand will provide a summary of the accounting impact of the recently-completed initial public offering of Viking Therapeutics by June 30, 2015 and will also update its 2015 financial forecast at that time.

Currently, the Company affirms expectations for full-year 2015 total revenues to be between $81.0 million and $83.0 million, and adjusted earnings per diluted share to be between $2.14 and $2.18.

For the second quarter of 2015, Ligand expects total revenues to be between $17.0 million and $17.5 million, and adjusted earnings per diluted share to be between $0.37 and $0.40. Adjusted earnings per diluted share guidance excludes changes in contingent liabilities, mark-to-market adjustment for amounts owed to licensors, non-cash stock-based compensation expense and non-cash debt related costs.

First Quarter and Recent Business Highlights

Partnered Program Progress

Promacta/Revolade

Novartis completed the acquisition of Promacta and related assets from GSK on March 1, 2015. Compared with GSK, Novartis has significantly larger oncology R&D and commercial capabilities.

A submission was made to the EMA seeking an additional indication for the treatment of pediatric patients with chronic immune thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids or immunoglobulins. The submission was based on the results from the Phase 3 PETIT2 study and the Phase 2 PETIT study in pediatric chronic ITP.
Kyprolis

Interim analysis showed the Phase 3 ENDEAVOR clinical trial evaluating Kyprolis (carfilzomib) versus Velcade (bortezomib) met the primary endpoint of progression-free survival (PFS).

Patients with relapsed multiple myeloma treated with Kyprolis lived twice as long without their disease worsening, 18.7 months for Kyprolis versus 9.4 months for Velcade.

Kyprolis demonstrated superiority over Velcade for overall response rate and lower neuropathy events.
The FDA accepted the sNDA of Kyprolis for the treatment of patients with relapsed multiple myeloma who have received at least one prior therapy. The FDA granted Kyprolis priority review with a PDUFA date of July 26, 2015.
The EMA accepted the MAA of Kyprolis for the treatment of patients with relapsed multiple myeloma who have received at least one prior therapy. The MAA was granted accelerated assessment by the EMA.

Additional Pipeline and Partner Developments

Spectrum Pharmaceuticals announced that its NDA for Captisol-Enabled Melphalan was accepted by the FDA and assigned a PDUFA date of October 23, 2015. Spectrum is now referring to CE-Melphalan as EVOMELA.

Melinta Therapeutics announced positive top-line results from the first of two Phase 3 PROCEED studies to evaluate delafloxacin against vancomycin + aztreonam for the treatment of patients with acute bacterial skin and skin structure infections (ABSSSI).
Delafloxacin met the study’s primary endpoint of a reduction in the measurement of lesion erythema at the primary infection site at 48-to-72 hours post-treatment, the endpoint required by the FDA.

Delafloxacin was comparable to vancomycin in the study’s secondary endpoints, including investigator assessment of signs and symptoms of infection at the follow-up visit, a metric required by the EMA.

SAGE Therapeutics announced completion of treatment for the first patient enrolled in its Phase 3 open-label expanded access protocol. Study 302 is designed to evaluate the safety of SAGE-547 in patients with SRSE. In conjunction with this announcement, Ligand earned a $500,000 milestone payment from SAGE.

Retrophin announced the FDA granted orphan drug designation to sparsentan (RE-021) for the treatment of Focal Segmental Glomerulosclerosis (FSGS). Orphan designation will provide sparsentan with seven years of market exclusivity for FSGS upon approval. Prior to FDA approval, orphan designation provides incentives for sponsors including tax credits for clinical research expenses, the opportunity to obtain government grant funding to support clinical research and an exemption from FDA user fees.
TG Therapeutics announced the first presentation of preclinical data demonstrating single-agent and combination activity with two of the Company’s IRAK4 inhibitor compounds under development. The data demonstrated that:
Both IRAK4 inhibitors not only inhibited cell proliferation but also induced apoptosis as single agents in various B-cell lymphoma cell lines, potentially elucidating the mechanism of action of and clinical rationale for IRAK4 inhibition.

In addition, both compounds demonstrated marked synergy when combined with the novel targeted kinase inhibitors TGR-1202, the Company’s proprietary PI3K delta inhibitor, and with the BTK inhibitor ibrutinib.

Viking Therapeutics closed its initial public offering on May 5, 2015. In connection with the offering, Ligand received an equity milestone payment of 3.4 million shares. Ligand invested an additional $9.0 million in the offering. Combining the value of the equity milestone and additional direct investment, Ligand owned 49.8% of Viking’s outstanding common stock at closing. Key programs licensed to Viking include:
VK5211 (SARM) – Phase 2 program for hip fracture.
VK2809 and VK0214 (TRß) – Phase 2 program for dyslipidemia and nonalcoholic steatohepatitis (NASH). VK2809 is also in a preclinical program for adrenoleukodystrophy (ALD).
VK0612 (FBPase) – Phase 2 program for type 2 diabetes.
EPOR – Preclinical program for anemia.
DGAT-1 – Preclinical program for obesity and dyslipidemia.

Recent Acquisition

Ligand acquired financial rights to potential future milestones and royalties for more than 15 development programs from Selexis SA for $4 million in cash. The acquired programs include a mix of novel biologics and biosimilars. The programs are in various stages of development ranging from preclinical through Phase 3. Each acquired program is fully funded by a development partner. The acquisition expands Ligand’s portfolio to more than 120 fully-funded assets and increases the number of partners to more than 70. Ligand previously acquired a portfolio of biologic development programs from Selexis in April, 2013.

New Licensing Deal

Ligand and Sermonix Pharmaceuticals announced the signing of a license agreement for the development and commercialization of oral lasofoxifene in the U.S. and additional territories. Under the terms of the agreement, Ligand has received an undisclosed initial payment, and is entitled to receive up to $45 million in potential regulatory and commercial milestone payments and tiered royalties of 6% to 10% on future net sales. Lasofoxifene is an estrogen partial agonist for the treatment of osteoporosis and other diseases.

Internal Program Progress

Ligand announced that enrollment was completed for the Phase 1b Multiple-Ascending Dose trial of the Glucagon receptor antagonist LGD-6972. Data from the trial will be presented in a poster presentation at American Diabetes Association 75th Scientific Sessions in Boston on June 7th at 12:00 p.m. ET. The Company will also host an investor presentation that evening beginning at 7:00 p.m. ET in Boston.

Adjusted Financial Measures

The adjusted financial measures discussed above and in the tables below for the three months ended March 31, 2015 and 2014 exclude changes in contingent liabilities, mark-to-market adjustment for amounts owed to licensors, non-cash stock-based compensation expense, and non-cash debt related costs.

Management has presented net income, net income per share, income from continuing operations and income from continuing operations per share in accordance with GAAP and on an adjusted basis. Ligand believes that the presentation of adjusted financial measures provides useful supplementary information to investors and reflects amounts that are more closely aligned with the cash profits for the period as the items that are excluded from adjusted net income are all non-cash items. Ligand uses these adjusted financial measures in connection with its own budgeting and financial planning. These adjusted financial measures are in addition to, and not a substitute for, or superior to, measures of financial performance prepared in conformity with GAAP.

Halozyme Reports First Quarter 2015 Financial Results

On May 11, 2015 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported financial results for the first quarter ended March 31, 2015 (Press release, Halozyme, MAY 11, 2015, View Source [SID:1234506620]). Financial highlights include revenues of $18.7 million and a net loss of $15.1 million, or $0.12 per share, compared to revenues of $12.0 million and a net loss of $26.5 million, or $0.22 per share, for the first quarter of 2014.

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"We continued to execute well in the first quarter against a focused strategy in oncology, ramping investments in our core program and achieving significant milestones toward the initiation of a Phase 3 study in pancreatic cancer patients early next year," said Dr. Helen Torley, president and chief executive officer. "In addition to this strategic progress, we exited the quarter in a much stronger financial position than last year due to a steady increase in royalty revenues from our rHuPH20 platform."

First Quarter 2015 Highlights and Subsequent Events

Announced plans to initiate a Phase 3 clinical study (Study 301) in metastatic pancreatic cancer patients with high-hyaluronan (HA) tumors: Based on feedback following a Type B meeting with the FDA, the company intends to initiate a Phase 3 study that will allow for a potential marketing application based on either progression free survival (PFS) or overall survival. Use of PFS as the basis for marketing approval will be subject to the overall benefit and risk associated with Halozyme’s investigational new drug, PEGPH20, combined with nab-paclitaxel (ABRAXANE) and gemcitabine therapy, including the:
Magnitude of the PFS treatment effect observed;
Toxicity profile; and
Interim overall survival.

The company plans to use a companion diagnostic test to prospectively identify and select patients with high levels of HA for its Phase 3 trial. The FDA provided feedback supporting the selection of high-HA patients and confirmed that an Investigational Device Exemption – or IDE – will be required prior to initiating the Phase 3 study. An IDE is a regulatory application that summarizes the methodology, validation and proposed cut-point for patient selection. The company plans to submit the IDE to support the late Q1 2016 Study 301 start.

Presented in January the interim results of Study 202 evaluating PEGPH20 with gemcitabine and ABRAXANE (nab-paclitaxel) in metastatic pancreatic cancer patients: In a retrospectively defined sub-population of patients, the data showed a statistically significant doubling in median PFS in metastatic pancreatic cancer patients with high levels of HA who were treated with PEGPH20 combined with nab-paclitaxel (ABRAXANE) and gemcitabine (9.2 months vs. 4.3 months in patients treated with nab-paclitaxel ABRAXANE and gemcitabine alone). The potential risk profile, including rate of thromboembolic events, was also evaluated.

In April, the company also announced plans to present the interim results of the study during an oral presentation by Dr. Sunil Hingorani, a principal investigator, at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper).

Initiated enrollment in a Phase 1b/2 clinical trial of investigational PEGPH20 in non-small cell lung cancer (NSCLC): The company began enrollment in the Phase 1b/2 randomized clinical trial (PRIMAL) of PEGPH20 in combination with docetaxel as a second-line therapy for patients with locally advanced and metastatic NSCLC.

Published preclinical study results of PEGPH20 in Molecular Cancer Therapeutics (Singha et al., February 2015 14:523-532): Data highlights the use and potential of PEGPH20 to enhance the anti-cancer activity of the accompanying immunotherapy (trastuzumab and immune cells) in high-HA breast and ovarian cancer preclinical models. The paper expands on previously reported data in a poster presentation at the ASCO (Free ASCO Whitepaper) 2015 Gastrointestinal Cancers Symposium in San Francisco. The manuscript was written and funded by Halozyme.

Daratumumab selected as the first product candidate under Janssen collaboration: In March, Genmab A/S announced plans for a Phase 1 clinical trial of a subcutaneous formulation of the anti-CD38 antibody daratumumab using the ENHANZE technology. Daratumumab is being developed under a collaboration between Janssen and Genmab A/S since August 2012 when Genmab granted Janssen an exclusive worldwide license to develop, manufacture, and commercialize daratumumab. Daratumumab, a human monoclonal antibody that targets CD38, is in clinical development as a single agent and in combination with standard of care therapies in several settings of multiple myeloma.

First Quarter 2015 Financial Highlights

Revenues for the first quarter of 2015 were $18.7 million, compared to $12.0 million for the first quarter of 2014. Revenues in the first quarter included $6.8 million in royalty revenue from sales of products under collaboration agreements, $6.1 million in product sales of bulk rHuPH20 for use in manufacturing collaboration products for Roche, $3.8 million in Hylenex recombinant (hyaluronidase human injection) product sales, and $2.0 million in collaboration revenues. Royalty revenues represent October to December 2014 sales as a result of the one quarter lag in royalty reports.

Research and development expenses for the first quarter of 2015 were $16.7 million, compared to $21.4 million for the first quarter of 2014. The decrease was primarily due to a planned decrease in expenses associated with the diabetes program.

Selling, general and administrative expenses for the first quarter of 2015 were $9.4 million, compared to $10.3 million for the first quarter of 2014. The decrease was primarily due to a decrease in compensation expenses.

The net loss for the first quarter of 2015 was $15.1 million, or $0.12 per share, compared to a net loss for the first quarter of 2014 of $26.5 million, or $0.22 per share.

Cash, cash equivalents and marketable securities were $128.5 million at March 31, 2015, compared to $135.6 million at December 31, 2014. Net cash used in the first quarter of 2015 was approximately $7.1 million.
Financial Outlook for 2015

For the full year 2015, the company reiterated its previously disclosed guidance of:

Net revenues to be in the range of $85 million to $95 million.
Operating expenses to be in the range of $145 million to $155 million.
Net cash burn to be between $35 million to $45 million.