On May 3, 2016 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG), a specialty pharmaceutical company with a diverse portfolio of products in the areas of maternal health, anemia management and cancer supportive care, reported unaudited consolidated financial results for the first quarter ended March 31, 2016 (Filing, Q1, AMAG Pharmaceuticals, 2016, MAY 3, 2016, View Source [SID:1234511834]).

Most notably, the first quarter of 2016 included the approval of a single-dose, preservative-free formulation of Makena (hydroxyprogesterone caproate injection). Prior to the commercial availability of this new Makena formulation, healthcare providers and patients who wanted a preservative-free formulation of hydroxyprogesterone caproate had to use non-FDA-approved, compounded versions. The company believes the commercial launch of the single-dose, preservative-free formulation of Makena will enable AMAG to capture significant additional share from the compounded segment of the market. The company estimates that compounding pharmacies currently hold approximately 37% of the Makena-eligible market. Additionally, AMAG entered into an agreement with a leading provider of home nursing services, which will exclusively administer Makena to all indicated patients who are eligible for their home health services.

"We are off to a strong start in 2016, achieving goals on our next-generation program for Makena, including the commercial launch of our new single-dose, preservative-free formulation, as well as solidifying an important new strategic relationship to further expand access to FDA-approved Makena," said William Heiden, AMAG’s chief executive officer. "First quarter 2016 sales of Makena increased 17% over the prior year, and early data from the recent launch of the new formulation of Makena, including April enrollment data at the Makena Care Connection that is up approximately 50% over March, suggests an acceleration in sales growth. We expect these trends to continue through the remainder of the year and are confirming our annual net product sales guidance for Makena."

"CBR, our newborn stem cell preservation offering, as well as our anemia management and cancer supportive care products, performed in-line with our expectations and remain on track with our 2016 sales guidance," added Mr. Heiden.

1 See summaries of non-GAAP adjustments for the three months ended March 31, 2016 and 2015 at the conclusion of this press release.

First Quarter 2016 and Recent Business Highlights:

· Increased net product sales of Makena to $65 million, compared with $55.5 million in the first quarter of 2015. This growth in sales was driven by a 16% increase in volume as more at-risk pregnant women were treated with Makena. Net revenue per injection was up 1% versus the first quarter of 2015.

· Received approval from the Food and Drug Administration (FDA) in February 2016 for a single-dose, preservative-free formulation of Makena and began commercial promotion in April 2016.

· Entered into a new agreement with a leading provider of home nursing services, which had previously utilized compounded hydroxyprogesterone caproate and now will exclusively provide at-home administration of Makena.

· Continued development of the next-generation program to deliver Makena subcutaneously via an auto-injector, with a range of activities underway, such as CMC work and pilot pharmacokinetic studies. The company currently anticipates filing the supplemental New Drug Application (sNDA) in the second quarter of 2017 and recently received confirmation from the FDA that the review time will be six months from submission.

· Generated a record $24.2 million of Feraheme (ferumoxytol) sales in the first quarter of 2016, or 13% growth over the same period in the prior year. Strong execution of the Feraheme business strategies by the commercial team enabled the company to realize a 7% increase in volume.

· Began enrolling patients in a head-to-head, Phase 3 clinical trial evaluating the safety of Feraheme compared to Injectafer (ferric carboxymaltose injection) in adults with iron deficiency anemia (IDA). This study is intended to support an sNDA filing to broaden the use of Feraheme beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment.

· Increased cash, cash equivalents and investments by $13.9 million to $480 million, net of $12 million utilized to purchase the company’s common stock and to repay debt.

· Strengthened the executive management team with the additions of Nik Grund as chief commercial officer and Ted Myles as chief financial officer.

First Quarter Ended March 31, 2016 (unaudited)

Financial Results (GAAP Basis)

Total revenues for the first quarter of 2016 were $109.3 million, compared with $89.5 million in the first quarter of 2015. Net product sales of Makena were $65.0 million in the first quarter of 2016, compared with $55.5 million in the same period last year. Sales of Feraheme and MuGard totaled $24.5 million in the first quarter of 2016, compared with $21.9 million in the first quarter of 2015. Service revenue from Cord Blood Registry (CBR), which AMAG purchased in August 2015, totaled $19.5 million in the first quarter of 2016.

Costs of product sales and services totaled $23.8 million in the first quarter of 2016. In the first quarter of 2015, cost of product sales totaled $21.0 million and did not include CBR cost of services. Total operating expenses for the first quarter of 2016 were $78.0 million, compared with $39.7 million for the same period in 2015. The increase in operating expenses was primarily due to the acquisition of CBR in the third quarter of 2015, including the non-cash amortization of intangible assets, and higher research and development

costs. These R&D costs included the initiation of the company’s Phase 3 clinical trial to broaden the use of Feraheme to include all adult IDA patients, costs to support our Makena subcutaneous auto-injector and costs associated with preparing and filing with the FDA for a second source manufacturer of the single-dose, preservative-free formulation of Makena.

The company reported operating income of $7.4 million and a net loss of $7.5 million, or ($0.22) per basic and diluted share, for the first quarter of 2016, compared with operating income of $28.8 million and net income of $12.9 million, or $0.47 per basic share and $0.39 per diluted share, for the same period in 2015.

Financial Results (Non-GAAP Basis)1,2

Non-GAAP revenues totaled $117.9 million in the first quarter of 2016, up from $83.1 million in the first quarter of 2015. Non-GAAP CBR revenue totaled $28.1 million in the first quarter of 2016. The difference between GAAP and non-GAAP revenue for CBR represents purchase accounting adjustments related to deferred revenue. Non-GAAP revenue in 2015 excludes certain non-cash revenue related to the company’s ex-U.S. marketing agreement with its former partner, Takeda Pharmaceutical Company Limited.

Total costs and expenses on a non-GAAP basis totaled $70.4 million resulting in a gross margin of 92% and adjusted EBITDA margin of 40% for the first quarter of 2016. This compares to costs and expenses of $35.7 million in the same period of 2015, which resulted in a gross margin of 96% and adjusted EBITDA margin of 57%. The decline in gross margin resulted from the acquisition of CBR, which carries lower gross margins than the company’s pharmaceutical products. Investments in research and development to enhance the long-term revenue potential of Makena and Feraheme contributed to the lower adjusted EBITDA margin in the first quarter of 2016. Non-GAAP adjusted EBITDA for the first quarter of 2016 was $47.5 million, compared with $47.4 million for the same period in 2015.

After deducting cash interest expense, the company generated first quarter 2016 non-GAAP net income of $32.9 million, or $0.95 per non-GAAP basic share and $0.94 per non-GAAP diluted share. In the first quarter of 2015, non-GAAP net income totaled $40.0 million, or $1.47 per non-GAAP basic share and $1.17 per non-GAAP diluted share.

Balance Sheet Highlights

As of March 31, 2016, the company’s cash and investments totaled approximately $480 million and total debt (principal amount outstanding) was approximately $1.04 billion.

"We are reiterating our full year 2016 guidance for revenue, adjusted EBITDA and non-GAAP net income, including top-line revenue growth of approximately 40%, which underscores the strong recent trends for Makena and the overall underlying demand we are generating across our portfolio of products," said Frank Thomas, president and chief operating officer. "During the quarter and throughout 2016, we are investing in our products through R&D to potentially expand our label for Feraheme and provide more patient- and provider-friendly versions of Makena. We believe these investments will enhance the long-term revenue potential of these products."

2 See share count reconciliation at the conclusion of this press release.

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On May 3, 2016 Rigel Pharmaceuticals, Inc. (Nasdaq:RIGL) reported financial results for the first quarter ended March 31, 2016 (Press release, Rigel, MAY 3, 2016, View Source;p=RssLanding&cat=news&id=2164508 [SID:1234511894]).

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"We look forward to our upcoming Phase 3 data as we continue with our planning for the potential commercial launch of fostamatinib in the United States," said Raul Rodriguez, president and chief executive officer of Rigel. "Also, we initiated the Phase 2 proof-of-concept study with fostamatinib in autoimmune hemolytic anemia (AIHA). We anticipate that the results of these studies as well as the IgA nephropathy study will be forthcoming later this year," he added.

During the first quarter, Rigel announced that patient enrollment was completed for the two studies in the FIT Phase 3 clinical program of fostamatinib in immune thrombocytopenic purpura (ITP). The results from the first study are expected in the middle of 2016, with the results for the second study expected shortly thereafter. Rigel plans to submit a New Drug Application to the Food and Drug Administration in the first quarter of 2017, subject to the results of the program. In addition, Rigel is in the early stages of establishing its sales and marketing infrastructure for the commercial launch of fostamatinib.

For the first quarter of 2016, Rigel reported a net loss of $17.5 million, or $0.19 per share, compared to a net loss of $18.2 million, or $0.21 per share, in the first quarter of 2015.

Contract revenues from collaborations of $5.0 million in the first quarter of 2016, compared to $2.2 million in the first quarter of 2015, were comprised of the amortization of the $30.0 million upfront payment and FTE fees earned pursuant to Rigel’s collaboration and license agreement with Bristol-Myers Squibb.

Rigel reported total costs and expenses of $22.6 million in the first quarter of 2016, compared to $20.4 million in the first quarter of 2015. The increase in costs and expenses was primarily due to the increase in research and development costs related to Rigel’s clinical research programs with fostamatinib in ITP and AIHA.

As of March 31, 2016, Rigel had cash, cash equivalents and short-term investments of $103.6 million, compared to $126.3 million as of December 31, 2015. Rigel expects this amount to be sufficient to fund operations into the third quarter of 2017.

On May 03, 2016 Supernus Pharmaceuticals, Inc. (NASDAQ:SUPN), a specialty pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, reported financial results for first quarter 2016 and associated company developments (Press release, Supernus, MAY 3, 2016, View Source [SID:1234511895]).

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Commercial Update

First quarter 2016 product prescriptions for Trokendi XR and Oxtellar XR, as reported by IMS, totaled 114,773, a 49.7% increase over the first quarter of 2015.

Prescriptions
Q1 2016 Q1 2015 Change %

Trokendi XR 85,987 55,227 55.7 %
Oxtellar XR 28,786 21,463 34.1 %

Total 114,773 76,690 49.7 %

Source Data: Product prescriptions as reported by IMS

Total revenue for the first quarter of 2016 was $43.1 million, a 53.1% increase over $28.1 million in the same period last year. Total revenue for both periods consisted almost exclusively of net product sales.

Net Product Sales ($mil.)
Q1 2016 Q1 2015 Change %

Trokendi XR $ 32.3 $ 20.9 54.5 %
Oxtellar XR $ 10.7 $ 7.2 49.3 %

Total $ 43.0 $ 28.1 53.1 %

Consistent with industry trends and the Company’s experience in the first quarter last year, product prescriptions and net product sales for Trokendi XR and Oxtellar XR were unfavorably impacted by the reset of insurance coverage at the beginning of the year, with high deductible and high co-pay programs. Additionally, changes in wholesaler inventory levels and ordering patterns during the first quarter of 2016 had the effect of reducing net product sales by approximately $3 million in the first quarter of 2016.

"Our business continues to post strong growth compared to last year. Total first quarter IMS prescriptions, and net product sales grew by 50% or more compared to the same period last year. We continue to build on the momentum we finished with last year promoting the benefits of our products to patients with epilepsy," said Jack Khattar, President and CEO of Supernus Pharmaceuticals.

Regarding our supplemental new drug application requesting approval to expand the Trokendi XR label to include treatment of migraine in adults, we are ready to launch the new migraine indication once we receive full approval from the FDA.

Progress of Product Pipeline

Enrollment continues for both Phase III trials for SPN-810, which is currently in development for Impulsive Aggression in patients who have ADHD, and for the Phase IIb trial for SPN-812, currently in development for ADHD. The Company continues to expect Phase III data for SPN-810 to be available by mid 2017, and data from the SPN-812 Phase IIb trial to be available by early 2017.

"In addition to our recent findings on the favorable emerging clinical profile of SPN-812 as it relates to adverse events, we completed the evaluation of the cardiac effects portion of the single ascending and multiple ascending dose study. We are pleased to report that there was no clinically significant change in QT interval and other ECG parameters. We believe these additional safety data in adult healthy volunteers, which show a lack of cardiac effects, are very encouraging and further strengthen the differentiation of SPN-812," said Jack Khattar.

Collaboration Update

Shire recently announced positive results of SHP465 in a safety and efficacy study in children and adolescents with ADHD. The study addresses a key U.S. Food and Drug Administration (FDA) requirement, keeping SHP465 on track for resubmission in the fourth quarter of 2016 and a potential launch in the second half of 2017, if it is approved by the FDA. SHP465 was originally developed by Shire Laboratories, the former division of Shire that subsequently became Supernus Pharmaceuticals. Based on the agreement between Supernus and Shire, Shire will pay to Supernus a single-digit percentage royalty on net sales of the product.

Operating Expenses

Research and development expenses in the first quarter of 2016 were $10.6 million, as compared to $3.7 million in the same quarter the prior year. This increase is primarily due to the ongoing Phase III testing of SPN-810 and Phase IIb testing of SPN-812.

Selling, general and administrative expenses in the first quarter of 2016 were $25.2 million, as compared to $19.4 million in the same quarter the prior year. The increase is primarily due to the continued increase in our sales and marketing efforts for both Trokendi XR and Oxtellar XR and the efforts in preparing for the launch of the migraine indication for Trokendi XR.

Operating Income and Earnings Per Share

Operating income in the first quarter of 2016 was $5.3 million, an increase of 55.0% over operating income of $3.4 million in the same period last year. This improvement in operating income is primarily due to the increase in net product sales.

Diluted earnings per share were $0.08 in the first quarter ended March 31, 2016, compared to $0.02 in the same period last year.

Weighted-average diluted common shares outstanding were approximately 51.2 million in the first quarter of 2016, as compared to approximately 44.9 million in the same period the prior year.

Capital Resources

As of March 31, 2016, the Company had $114.0 million in cash, cash equivalents, marketable securities, and long term marketable securities, as compared to $117.2 million at December 31, 2015. As of March 31, 2016, approximately $6.6 million of the Company’s six year, $90 million notes, bearing interest at 7.5% per annum, remain outstanding.

Financial Guidance

For full year 2016, the Company reiterates its expectation that net product sales will range from $200 million to $210 million, R&D expenses to range from $55 million to $65 million, and operating income to range from $28 million to $35 million.

On May 2, 2016 Xencor, Inc. (NASDAQ: XNCR), a clinical-stage biopharmaceutical company developing engineered monoclonal antibodies for the treatment of autoimmune diseases, asthma and allergic diseases and cancer, reported financial results for the first quarter ended March 31, 2016 and provided a review of pipeline and corporate highlights (Filing, Q1, Xencor, 2016, MAY 2, 2016, View Source [SID:1234511837]).

"During the quarter we initiated Phase 2 trials with XmAb5871 in both IgG4-Related Disease (IgG4-RD) and systemic lupus erythematosus (SLE), two diseases with high unmet need and a strong rationale for B-cell inhibition," said Bassil Dahiyat, Ph.D., President and Chief Executive Officer of Xencor. "We also continued to advance our broad pipeline of wholly-owned and partnered programs. We remain on track to report the full results from our Phase 1a trial of XmAb7195 in the coming months and to initiate four additional clinical trials in 2016, including human trials evaluating our initial bispecific oncology product candidates, XmAb14045 for acute myeloid leukemia and XmAb13676 for B-cell malignances."

Pipeline Highlights:

XmAb5871: A first-in-class monoclonal antibody that targets CD19 with its variable domain and that uses Xencor’s proprietary XmAb immune inhibitor Fc domain to target FcgRIIb, a receptor that inhibits B-cell function. In March 2016, Xencor initiated a Phase 2 clinical study for the treatment of IgG4-Related Disease (IgG4-RD) and a Phase 2 clinical study for the treatment of systemic lupus erythematosus (SLE).

· Initiation of Phase 1 trial with a subcutaneous formulation expected in 2016
· Initial data from IgG4-RD Phase 2 trial expected in 1H 2017
· Initial data from subcutaneous formulation Phase 1 trial expected in 2017
· Initial data from SLE Phase 2 trial expected in 2018

The primary objective of the Phase 2, open-label, pilot study of XmAb5871 in patients with IgG4-RD is to evaluate the effect of every other week intravenous (IV) administration of XmAb5871 on the IgG4-RD Responder Index in patients with active IgG4-RD. Secondary and exploratory objectives are to determine the safety and tolerability profile, to characterize the pharmacokinetics and pharmacodynamics and to characterize immunogenicity of every other week IV administration of XmAb5871 in patients with IgG4-RD. The trial will enroll approximately 15 subjects for up to 24 weeks of treatment.

The primary endpoint for the Phase 2 randomized, double-blind, placebo-controlled study of XmAb5871 in patients with SLE is maintenance of disease activity improvement achieved by a brief course of disease-suppressing IM steroid therapy. Secondary and exploratory endpoints are to evaluate the time to loss of SLE disease activity improvement, to determine the safety and tolerability profile, to characterize the pharmacokinetics and pharmacodynamics, and to characterize immunogenicity of every other week IV administration of XmAb5871 in patients with SLE. The trial will enroll approximately 90 subjects for up to 24 weeks of treatment.

XmAb7195: A first in class monoclonal antibody that targets IgE with its variable domain and uses Xencor’s XmAb immune inhibitor Fc domain to target FcgRIIb, resulting in three distinct mechanisms of action for reducing IgE levels. XmAb7195 is being developed for the treatment of severe asthma and allergic diseases.

· Full results from Phase 1a trial expected in 2Q 2016
· Initiation of Phase 1 trial with a subcutaneous formulation expected in 2016
· Initial data from subcutaneous formulation Phase 1 trial expected in 1H 2017

In 2015, Xencor announced interim data from Part 1 of this trial, which showed a rapid reduction of free IgE levels to below the limit of detection in 90% of treated subjects, including those treated at the lowest dose evaluated of 0.3 mg/kg, with parallel reductions in total IgE. A dose limiting toxicity of transient, asymptomatic thrombocytopenia was observed at the 3.0 mg/kg dose. Moderate urticaria was also reported in some treated subjects with an apparent correlation of dose with frequency of occurrence. The Phase 1 trial with a subcutaneous formulation in healthy volunteers will evaluate safety, tolerability and immunogenicity, and will measure IgE levels.

Internal Bispecific Oncology Pipeline: Xencor’s initial bispecific programs are tumor-targeted antibodies that contain both a tumor antigen binding domain and a cytotoxic T-cell binding domain (CD3). These bispecific antibodies activate T cells for highly potent and targeted killing of malignant cells. Their XmAb Fc domains confer long circulating half-lives, stability and ease of manufacture.

· Initiate clinical trial for XmAb14045 in 2016
· Initiate clinical trial for XmAb13676 in 2016
· Initial data for XmAb14045 expected in 2017
· Begin clinical trials for additional bispecific oncology candidates in 2017

Xencor plans to initiate clinical trials for its first two bispecific oncology candidates, XmAb14045, for the treatment of acute myeloid leukemia (AML), and XmAb13676, for the treatment of B-cell malignancies, in 2016. Xencor has multiple additional bispecific oncology candidates, both tumor targeting bispecifics and dual T-cell checkpoint inhibitor bispecifics in pre-clinical development.

Partnered XmAb Programs: Xencor’s partners currently have seven programs in clinical testing that were either discovered at Xencor or that rely on Xencor’s proprietary XmAb technology.

In January 2016, Xencor announced that the National Institutes of Health (NIH) initiated a Phase 1 clinical trial of VRC01LS, a therapeutic antibody for the treatment of HIV that uses Xencor’s Xtend antibody half-life extension technology.

MorphoSys announced that in 2017 it plans to begin a Phase 3 clinical trial of XmAb5574/MOR208 in diffuse large B-cell lymphoma (DLBCL).

First Quarter Ended March 31, 2016 Financial Results

Cash, cash equivalents and marketable securities totaled $178.7 million as of March 31, 2016, compared to $193.3 million on December 31, 2015. The decrease reflects net spending on operations in the first quarter of 2016.

Revenues for the first quarter ended March 31, 2016 were $7.3 million, compared to $1.5 million in the same period of 2015. Increased revenue for the first quarter of 2016 over revenue for the same period in 2015 is primarily the result of revenue recognized under our 2015 Amgen collaboration.

Research and development expenditures for the first quarter ended March 31, 2016 were $10.0 million, compared to $5.2 million for the same period in 2015. Increased research and development spending in the first quarter of 2016 over the same period in 2015 reflects additional spending on our XmAb5871 clinical programs and our initial bispecific development candidates, XmAb14045 and XmAb13676.

General and administrative expenses in the first quarter ended March 31, 2016 were $4.0 million compared to $2.8 million for the same period in 2015. Increased spending on general and administration in the first quarter of 2016 over the comparable period in 2015 reflects additional spending on professional fees including legal fees.

Non-cash, share based compensation expense for the first quarter ended March 31, 2016 was $2.0 million, compared to $1.1 million for same period in 2015.

Net loss for the first quarter ended March 31, 2016 was $6.40 million, or $(0.16) on a fully diluted per share basis, compared to a net loss of $6.44 million, or $(0.19) on a fully diluted per share basis, for the same period in 2015. Increased revenue in the first quarter of 2016 over the same period of 2015 was offset by increased expenditures of a similar amount such that the net loss for both periods is comparable. The lower loss per share amount in the first quarter of 2016 over the amount reported in the first quarter 2015 reflects the additional shares outstanding at the end of the first quarter of 2016.

The weighted-average shares outstanding used to compute loss per share was 40,626,729 for the quarter ended March 31, 2016, compared to 34,297,782 for the quarter ended March 31, 2015.

Financial Guidance

Based on current operating plans, Xencor expects to have cash to fund research and development programs and operations through 2019.

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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]).

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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