On May 2, 2016 Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, reported its first quarter 2016 financial results and accomplishments (Press release, Sangamo BioSciences, MAY 2, 2016, View Source [SID:1234511807]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Sangamo BioSciences, Inc. (PRNewsFoto/Sangamo BioSciences, Inc.)
"During the first quarter of 2016 we focused on activities to enable initiation of the first clinical trials of our IVPRP approach in hemophilia B and MPS I, and I am pleased to report that we will meet our stated timelines for the opening of both trials," said Edward Lanphier, Sangamo’s president and chief executive officer. "We look forward to generating and presenting clinical data that support the application of this highly leverageable platform while continuing to progress our programs in hemophilia A and several lysosomal storage disorders toward clinical development."

Recent Highlights

Presentation of new data from Sangamo’s proprietary In Vivo Protein Replacement Platform (IVPRP) programs for MPS I and MPS II at the 12TH Annual WORLDSymposium Meeting. Sangamo scientists and academic collaborators from the University of Minnesota presented new preclinical data in disease models of the Company’s IVPRP-based MPS I (Hurler syndrome) and MPS II (Hunter syndrome) programs at the 2016 WORLDSymposium Annual Meeting. In animal models of disease, the data demonstrated the production of stable, therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease, and notably, statistically significant improvements in cognitive function in treated animals.
Publication in the New England Journal of Medicine (NEJM) highlighting Sangamo’s IVPRP approach for hemophilia B. In March, NEJM published a review authored by Dr. Amit Nathwani, a key opinion leader in the field of gene therapy approaches to hemophilia, highlighting Sangamo’s IVPRP-based SB-FIX program for the one-time, permanent treatment of hemophilia B. The article details the significant advantages of Sangamo’s ZFN-mediated, targeted integration of the Factor IX (FIX) gene at the albumin locus over conventional non-integrating gene therapy approaches that use adeno-associated virus (AAV) to deliver the FIX gene and other therapeutic genes to liver cells.
Sangamo augments clinical expertise with the appointment of Matthew Spear, M.D. as Vice President and Head of Clinical Development. Dr. Spear joins Sangamo with more than 20 years of experience in all stages of biopharmaceutical research and development and has led the development of over 15 therapeutic products. Most recently, Dr. Spear served as a Vice President in Clinical Development at Incyte Corp.
Presentation of immunological data from Sangamo’s SB-728-T Phase 2 clinical program in HIV/AIDS at the 2016 Annual Conference on Retroviral and Opportunistic Infections (CROI 2016). Sangamo’s collaborators from Case Western Reserve University presented analyses of immunological data from the Company’s clinical trials of SB-728-T, a ZFP Therapeutic designed to provide functional control of HIV/AIDS. The presentation outlined potentially interrelated mechanisms for viral load (VL) control in SB-728-T-treated subjects during a treatment interruption (TI) from their antiretroviral therapy. This suggests a model mechanism of action for SB-728-T and enables identification of patients who will most benefit from Sangamo’s ZFN-mediated CCR5 knock-out approach.
Upcoming Events in 2016

Initiation of IVPRP-based Phase 1/2 clinical trials SB-FIX-1501 (hemophilia B) and SB-318-1502 (MPS I / Hurler syndrome) by the end of the second quarter and mid-2016, respectively.
Presentation of data from several ZFP Therapeutic programs by Sangamo scientists and collaborators at the upcoming 19TH Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), which will be held in Washington D.C. from May 4-7, 2016.
Submission of investigational new drug (IND) applications in the first half of 2016 for Sangamo’s IVPRP-based SB-913 program for MPS II (Hunter syndrome), and beta-thalassemia program in collaboration with Biogen Inc. (Biogen).
Presentation of new clinical data from Cohort 3* of Sangamo’s SB-728-1101 Phase 1/2 trial in T-cells for the functional control of HIV/AIDS in the second half of 2016.
First Quarter 2016 Results
For the first quarter ended March 31, 2016, Sangamo reported a consolidated net loss of $16.5 million, or $0.23 per share, compared to a net loss of $5.3 million, or $0.08 per share, for the same period in 2015. As of March 31, 2016, the Company had cash, cash equivalents, marketable securities and interest receivable of $189.0 million.

Revenues for the first quarter of 2016 were $3.9 million, compared to $13.5 million for the same period in 2015. First quarter 2016 revenues were generated from the Company’s collaboration agreements with Biogen and Shire International GmbH (Shire), enabling technology agreements and research grants. The revenues recognized for the first quarter of 2016 consisted of $3.7 million from collaboration agreements and $0.2 million from research grants, compared to $12.7 million and $0.8 million, respectively, for the same period in 2015.

The decrease in collaboration agreement revenues was primarily a result of an amendment to the Company’s collaboration and license agreement with Shire in the third quarter of 2015, which returned the rights to the hemophilia programs to Sangamo, and a decrease in revenue under the Company’s collaboration agreement with Sigma.

In the first quarter of 2016, Sangamo recognized $2.0 million of revenues related to research services performed under the collaboration agreement with Biogen, and $0.4 million of revenues related to research services performed under the collaboration agreement with Shire. In addition, Sangamo received upfront payments of $13.0 million and $20.0 million pursuant to the agreements entered into with Shire in 2012 and Biogen in 2014, respectively. The Shire payment is being recognized as revenue on a straight-line basis over the initial six-year research term. Beginning in January 2016, the Biogen payment will be recognized over approximately 42 months which reflects the revised service period related to Sangamo’s deliverables under the Biogen agreement. The Company recognized $0.5 million of the Shire upfront payment and $0.6 million of the Biogen upfront payment as revenue for the first quarter of 2016.

Research and development expenses were $15.3 million for the first quarter of 2016, compared to $15.0 million for the same period in 2015. Research and development expenses were primarily comprised of manufacturing expenses, research expenses associated with Sangamo’s clinical and preclinical programs, and personnel-related expenses, including stock-based compensation.

General and administrative expenses were $5.4 million for the first quarter of 2016, compared to $4.7 million for the same period in 2015.

Total operating expenses for the first quarter of 2016 were $20.6 million, compared to $19.7 million for the same period in 2015.

Financial Guidance for 2016
The Company reiterates its earlier guidance as follows:

Cash and Investments: Sangamo expects that its cash, cash equivalents and marketable securities will be at least $150 million at the end of 2016, inclusive of research funding from existing collaborators but exclusive of funds arising from any additional new collaborations or partnerships, equity financings or other new sources.
Revenues: Sangamo expects that revenues will be in the range of $20 million to $25 million in 2016, inclusive of research funding from existing collaborations.
Operating Expenses: Sangamo expects that operating expenses will be in the range of $85 million to $95 million for 2016

On May 02, 2016 Pacira Pharmaceuticals, Inc. (NASDAQ:PCRX) provided updates on EXPAREL (bupivacaine liposome injectable suspension) for postsurgical pain in the United States and announced consolidated financial results for the first quarter ended March 31, 2016 (Press release, Pacira Pharmaceuticals, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163649 [SID:1234511733]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With immense leverage across our organization from manufacturing to financials, we began our aggressive investment in the future growth of Pacira in the first quarter of 2016," said Dave Stack, Chief Executive Officer and Chairman. "In this short amount of time, we were able to enhance our executive team with two important hires, initiate two Phase 3 studies for the EXPAREL nerve block indication and initiate a randomized controlled trial in total knee arthroplasty to establish best practice for best outcomes with EXPAREL. As we prepare for our oral surgery launch later this year, we look forward to further investments in the expanded use of EXPAREL and development of pipeline opportunities."

Recent Highlights

Key Executive Appointments to Support Company’s Future Growth: Pacira recently announced the appointment of two key executives to the Pacira management team. As Chief Financial Officer, Charles A. Reinhart III will be responsible for all financial and capital market activities, including accounting, financial reporting, financial planning and analysis and investor relations. He will succeed former Chief Financial Officer Jim Scibetta, who will continue to serve as President, and will report to Dave Stack. As Chief Commercial Officer, Robert Weiland will oversee commercial activities for EXPAREL, which include marketing, sales, national accounts, training and commercial operations and analytics. He will report to Jim Scibetta.

Initiation of Important Clinical Studies for EXPAREL: Pacira initiated two Phase 3 clinical trials that will serve as the basis of the supplemental New Drug Application (sNDA) to be filed for EXPAREL use in nerve block to provide postsurgical analgesia. One study will assess brachial plexus block with EXPAREL in patients undergoing total shoulder arthroplasty or rotator cuff repair. The second study will evaluate femoral nerve block with EXPAREL for patients undergoing total knee arthroplasty. Pacira also initiated a randomized controlled trial comparing local infiltration analgesia with EXPAREL to local infiltration analgesia without EXPAREL in total knee arthroplasty. This study is intended to provide a procedure-specific protocol on appropriate technique and administration for producing optimal outcomes with EXPAREL.

First Quarter 2016 Financial Results

EXPAREL net product revenues were $63.8 million in the first quarter of 2016, compared to $56.0 million in the first quarter of 2015.

Total revenues were $65.5 million in the first quarter of 2016, compared to $58.3 million in the first quarter of 2015.

Total operating expenses were $67.7 million in the first quarter of 2016, compared to $55.0 million in the first quarter of 2015.

GAAP net loss was $3.9 million, or $(0.10) per share (basic and diluted), in the first quarter of 2016, compared to GAAP net income of $1.3 million, or $0.03 per share (basic and diluted), in the first quarter of 2015.

Non-GAAP net income was $5.7 million, or $0.15 per share (basic) and $0.14 per share (diluted), in the first quarter of 2016, compared to non-GAAP net income of $9.8 million, or $0.27 per share (basic) and $0.23 per share (diluted), in the first quarter of 2015.

Pacira ended the first quarter of 2016 with cash and cash equivalents, short-term investments and long-term investments ("cash") of $163.5 million.

Pacira had 37.0 million basic and 41.1 million diluted weighted average shares of common stock outstanding in the first quarter of 2016.

2016 Outlook

Pacira affirms that it expects the following operating expenses for 2016:

Non-GAAP research and development (R&D) expense of $60 million to $70 million.

Non-GAAP selling, general and administrative (SG&A) expense of $125 million to $135 million.

Stock-based compensation expense of $35 million to $40 million.

Today’s Conference Call and Webcast Reminder

The Pacira management team will host a conference call to discuss the company’s financial results and recent developments today, Monday, May 2, 2016, at 9 a.m. ET. The call can be accessed by dialing 1-877-845-0779 (domestic) or 1-720-545-0035 (international) ten minutes prior to the start of the call and providing the Conference ID 54803652.

A replay of the call will be available approximately two hours after the completion of the call and can be accessed by dialing 1-855-859-2056 (domestic) or 1-404-537-3406 (international) and providing the Conference ID 54803652. The replay of the call will be available for two weeks from the date of the live call.

The live, listen-only webcast of the conference call can also be accessed by visiting the "Investors & Media" section of the company’s website at investor.pacira.com. A replay of the webcast will be archived on the Pacira website for two weeks following the call.

Non-GAAP Financial Information

This press release contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), non-GAAP net income, because such measures exclude stock-based compensation and amortization of debt discount. These measures supplement our financial results prepared in accordance with GAAP. Pacira management uses these measures to better analyze its financial results, estimate its future R&D, SG&A and stock-based compensation outlook for 2016 and to help make managerial decisions. In management’s opinion, these non-GAAP measures are useful to investors and other users of our financial statements by providing greater transparency into the operating performance at Pacira and the company’s future outlook. Such measures should not be deemed to be an alternative to GAAP requirements or a measure of liquidity for Pacira. Non-GAAP net income measures are also unlikely to be comparable with non-GAAP disclosures released by other companies. See a reconciliation of non-GAAP net income to GAAP net income (loss) below.

On May 2, 2016 AVEO Oncology (NASDAQ:AVEO) reported that it has filed provisional patent applications with the United States Patent and Trademark Office (USPTO) covering composition of matter claims for AV-353, the Company’s potent inhibitory antibody specific to Notch 3 (Press release, AVEO, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163617 [SID:1234511738]). These patent applications are the second set of applications related to AV-353 and the Company’s Notch 3 antibody program.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Notch 3 signaling pathway is important in cell-to-cell communication involving gene regulation mechanisms that control multiple cell differentiation processes during the entire life cycle. Scientific literature has implicated the Notch 3 receptor pathway in multiple diseases, including cancer, cardiovascular diseases and neurodegenerative conditions. Recent publications, including Nature Medicine (2009), have implicated the Notch 3 pathway in Pulmonary Arterial Hypertension (PAH). PAH is a rare and life-threatening disorder that affects approximately 250,000 people worldwide and is caused by enlargement of the arterial walls in small arteries between the heart and the lungs, resulting in restricted blood flow.

Current treatments in PAH focus only on controlling symptoms by avoiding vasoconstriction and increasing vasodilation of vessels and do not reverse the underlying cause of the disease. In contrast, with the results of a recently concluded research study supported by AVEO, AV-353 has generated a growing body of preclinical data that supports AV-353’s ability to potentially reverse the disease phenotype, which would represent a potential disease-modifying approach to treatment. A manuscript of the results is being prepared for submission to a peer-reviewed journal.

"Like AV-380 in cachexia, which AVEO licensed to Novartis in 2015, AV-353 is an AVEO legacy discovery program that, with the appropriate development support from a partner, has the potential to transform the treatment paradigm for a debilitating disorder and underserved patient population," said Michael Bailey, president and chief executive officer of AVEO. "We believe that AV-353’s selectivity and high affinity to Notch 3, as demonstrated in preclinical studies, makes it unique relative to pan-notch inhibitory or ligand inhibitory approaches in PAH treatment, allowing us to potentially build a broad patent estate around this program. Consistent with our current focus on developing oncology therapeutics, AVEO is currently seeking an appropriate partner to develop and commercialize AV-353 worldwide in PAH."

On May 2, 2016 Prima BioMed Ltd (ASX: PRR; NASDAQ: PBMD) ("Prima", the "Company") reported the granting of patent number 5908210 entitled "Use of Recombinant LAG-3 or the Derivatives thereof for Eliciting Monocyte Immune Response" by the Japanese Patent Office (Filing, 6-K, Prima Biomed, MAY 2, 2016, View Source [SID:1234511818]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This patent relates to Prima’s IMP321 and its use either alone, or in combination, for immunotherapeutic or chemotherapeutic purposes to induce an increase in circulating monocyte numbers in order to protect against cancer. This patent will therefore support the use of IMP321 as it is being used in Prima’s AIPAC trial in metastatic breast cancer. Patent expiry is expected to be 3rd of October 2028.

About Prima BioMed
Prima BioMed is a globally active biotechnology company positioned to become a leader in the development of immunotherapeutic products for the treatment of cancer. Prima BioMed is dedicated to leveraging its technology and expertise to bring innovative treatment options to market for patients and to maximise value to shareholders.

Prima’s current lead product is IMP321, based on the LAG-3 immune control mechanism which plays a vital role in the regulation of the T cell immune response. IMP321, which is a soluble LAG-3Ig fusion protein, is an APC activator boosting T cell responses. IMP321 is currently in a Phase II clinical trial as a chemoimmunotherapy for metastatic breast cancer termed AIPAC (clinicaltrials.gov identifier NCT 02614833) and in a Phase I combination therapy trial in metastatic melanoma termed TACTI-mel (clinicaltrials.gov identifier NCT 02676869). A number of additional LAG-3 products including antibodies for immune response modulation in autoimmunity and cancer are being developed by large pharmaceutical partners.

On May 2, 2016 Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company with three clinical-stage development programs focused on the emerging field of regenerative medicine, reported that clinical data from its AST-VAC1 (antigen-presenting autologous dendritic cells) immunotherapy clinical program will be presented during an oral session at the upcoming American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 19th Annual Meeting, to be held on May 4-7, 2016 in Washington, D.C (Press release, BioTime, MAY 2, 2016, View Source;p=RssLanding&cat=news&id=2163621 [SID:1234511741]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Jane S. Lebkowski, Ph.D., Asterias’ President of R&D and Chief Scientific Officer, will present the data, which is from the Company’s AST-VAC1 Phase 2 clinical trial in acute myelogenous leukemia (AML) and was first presented at the 2015 annual meeting of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper). The abstract is now available online on the ASGCT (Free ASGCT Whitepaper) Annual Meeting website.

Presentation Details

Title: Long-Term Relapse-Free Survival of Patients with Acute Myeloid Leukemia (AML) Receiving a Telomerase-Engineered Dendritic Cell Immunotherapy
Abstract Number: 276
Session Title: Cancer-Immunotherapy, Cancer Vaccines I
Date: Thursday, May 5, 2016
Time: 5:00 – 5:15 PM
Room: Washington 4