On May 04, 2016 MacroGenics, Inc. (NASDAQ:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported a corporate progress update and reported financial results for the quarter ended March 31, 2016 (Press release, MacroGenics, MAY 4, 2016, View Source [SID:1234511934]).

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"MacroGenics made steady progress across its pipeline of clinical and research-stage compounds during the first quarter of 2016," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "The SOPHIA study, a Phase 3 trial of margetuximab, our Fc-optimized anti-HER2 monoclonal antibody, continues to enroll patients with metastatic breast cancer. Our immuno-oncology efforts, highlighted by the B7-H3 franchise, also progressed nicely. We anticipate sharing additional enoblituzumab monotherapy study data later this year. Further, our portfolio of innovative molecules was the subject of five poster presentations at the recent American Association of Cancer Research annual meeting."

"As we look forward in 2016 and beyond, we expect to continue our pace of generating promising clinical development candidates based on MacroGenics’ technology platforms," commented Dr. Koenig. "In particular, we expect to submit one IND later this year and two additional INDs in 2017."

Pipeline Update

Margetuximab. Recent highlights related to our Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2, or HER2, include:

SOPHIA Study: MacroGenics’ Phase 3 pivotal study in patients with HER2-positive metastatic breast cancer is ongoing, as the Company continues to initiate sites and enroll patients. This study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 patients following progression after at least two lines of previous therapy. The Company is targeting completion of this study in 2018.
Phase 1b/2 Gastric Cancer Study: During the first quarter of 2016, MacroGenics dosed the first patient in a Phase 1b/2 clinical trial of margetuximab in combination with pembrolizumab, an anti-PD-1 therapy, in patients with advanced HER2-positive gastric cancer. Treatment options for these patients are limited and this proposed combination regimen being studied would avoid chemotherapy while exploiting the expected enhanced immune-mediated killing properties of both margetuximab and pembrolizumab. This trial is being conducted in collaboration with Merck and is currently recruiting patients in the United States, with plans to expand into Asian sites later this year.
B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action and take advantage of this antigen’s broad expression across multiple solid tumor types. Current ongoing development programs include:

Enoblituzumab (MGA271): The Company continues to recruit patients in three ongoing studies of enoblituzumab, an Fc-optimized monoclonal antibody that targets B7-H3. These studies include one monotherapy study and two combination studies with each of ipilimumab and pembrolizumab.
MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types. The results of preclinical studies of MGD009 were presented in an oral presentation at Keystone Symposia’s Antibodies as Drugs (X2) conference in March. These studies demonstrated that MGD009 redirected T cells to kill B7-H3-expressing human cancer cell lines from a range of tumor types in multiple in vitro and in vivo models.
B7-H3 Antibody-drug Conjugate: At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April, MacroGenics presented a poster that evaluated the therapeutic potential of anti-B7-H3 ADCs in multiple in vitro and in vivo models representing human cancer types that overexpress B7-H3.
DART Product Candidates. There are currently six DART molecules in Phase 1 clinical development, including MGD006 (CD123 x CD3, also known as S80880), MGD007 (gpA33 x CD3), MGD011 (CD19 x CD3, also known as JNJ-64052781), MGD010 (CD32B x CD79B), MGD009 (B7-H3 x CD3) and PF-06671008 (P-cadherin x CD3). The Company expects to submit IND applications for two additional DART molecules in 2017. These two product candidates are:

MGD013: MacroGenics is developing MGD013 to simultaneously block two immune checkpoint molecules, PD-1 and LAG-3. At the recent AACR (Free AACR Whitepaper) meeting, MacroGenics demonstrated that MGD013 has the potential to enhance T-cell immunomodulatory activity as compared to its individual components.
MGD014: MGD014 is a DART molecule that is being developed to eliminate latent HIV infection. MGD014 is being developed under a contract awarded to MacroGenics by the National Institute of Allergy and Infectious Diseases for up to $24.5 million. This is the first infectious disease DART program planned for clinical testing.
Beyond MGD013 and MGD014, MacroGenics continues to generate and evaluate multiple other candidates that target a range of immune regulatory and other molecules using its proprietary platforms. In addition to the B7-H3 ADC and MGD013 posters presented at the recent AACR (Free AACR Whitepaper) meeting, the Company also presented posters on the following three preclinical DART molecules at the meeting: EphA2 x CD3, IL13Rα2 x CD3 and ROR1 x CD3.

First Quarter 2016 Financial Results

Cash Position: Cash, cash equivalents and investments as of March 31, 2016 were $304.4 million, compared to $339.0 million as of December 31, 2015.
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $2.8 million for the quarter ended March 31, 2016, compared to $71.3 million for the quarter ended March 31, 2015. This decrease is primarily due to the $62.3 million in revenue recognized under the Janssen agreement in the first quarter of 2015.
R&D Expenses: Research and development expenses were $27.3 million for the quarter ended March 31, 2016, compared to $21.5 million for the quarter ended March 31, 2015. This increase was due primarily to increased activity in MacroGenics’ preclinical immune checkpoint programs, including MGD013, and the initiation of two Phase 1 clinical trials combining enoblituzumab with other compounds. This increase was partially offset by a decrease in margetuximab expense as a result of start-up costs in 2015 for the SOPHIA trial.
G&A Expenses: General and administrative expenses were $6.1 million for the quarter ended March 31, 2016, compared to $4.7 million for the quarter ended March 31, 2015. This increase was primarily due to higher labor-related costs, including stock-based compensation expense.
Net Loss: Net loss was $30.3 million for the quarter ended March 31, 2016, compared to net income of $45.1 million for the quarter ended March 31, 2015.
Shares Outstanding: Shares outstanding as of March 31, 2016 were 34,536,621.

On May 4, 2016 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its first quarter 2016 financial results and ongoing progress with its pipeline, including duvelisib, an investigational, oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, and IPI-549, an immuno-oncology development candidate that selectively inhibits PI3K-gamma (Press release, Infinity Pharmaceuticals, MAY 4, 2016, View Source;p=RssLanding&cat=news&id=2165087 [SID:1234511932]).

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Infinity expects to report topline data from DYNAMO, a registration-focused Phase 2 monotherapy study of duvelisib in patients with refractory indolent non-Hodgkin lymphoma (iNHL), early in the third quarter of 2016. Infinity also anticipates completing an interim analysis of DUO, a registration-focused Phase 3 monotherapy study of duvelisib in patients with relapsed/refractory chronic lymphocytic leukemia (CLL), early in the second half of 2016. The company expects marketing applications, if supported by these data, to be submitted to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in the fourth quarter of 2016.

"We believe the next few months will be fundamental to advancing Infinity’s goal of bringing important new medicines to patients. If the duvelisib data that we anticipate in the coming months are favorable, these data could enable regulatory filings to be submitted in the U.S. and Europe in the fourth quarter of 2016. Achieving these milestones positions us to potentially bring duvelisib to patients next year," stated Adelene Perkins, president and chief executive officer. "Infinity is also advancing several clinical studies designed to evaluate the safety and activity of duvelisib in broader patient populations. Additionally, our collaborator, AbbVie, has initiated a Phase 1b/2 clinical study of duvelisib in combination with Venclexta," Ms. Perkins continued.

Recent developments:

Preliminary data from CONTEMPO to be presented in June: Infinity reported that preliminary data from CONTEMPO, a Phase 1b/2 study evaluating duvelisib in combination with rituximab or obinutuzmab in treatment-naïve follicular lymphoma patients, has been accepted for presentation in a poster session at the 21st Congress of European Hematology Association (EHA) (Free EHA Whitepaper), which will take place June 9 – 12, 2016, in Copenhagen.

Phase 1b/2 study of duvelisib in combination with Venclexta initiated: AbbVie has initiated a Phase 1b/2 clinical study of duvelisib in combination with Venclexta, AbbVie’s B-cell lymphoma-2 (BCL-2) selective inhibitor. This study is designed to evaluate the safety and efficacy of duvelisib in combination with venetoclax in approximately 174 patients with relapsed or refractory iNHL, aggressive NHL, small lymphocytic lymphoma or CLL.

Preclinical data for IPI-549 presented at the 2016 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting: In April, Infinity researchers in collaboration with researchers at Memorial Sloan Kettering Cancer Center presented preclinical data for IPI-549 at the 2016 AACR (Free AACR Whitepaper) Annual Meeting. Preclinical data in multiple solid tumor models demonstrated that IPI-549 targets immune cells and alters the immune-suppressive microenvironment, promoting an anti-tumor immune response that leads to tumor growth inhibition. Data also demonstrated that IPI-549 enhances the effects of checkpoint inhibitors, resulting in improved survival in murine models.
A Phase 1 study of IPI-549 is ongoing to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with an anti-PD-1 antibody, a checkpoint inhibitor, in approximately 150 patients with advanced solid tumors, including non-small cell lung cancer and melanoma. IPI-549 is the only investigational PI3K-gamma inhibitor in clinical development.

Inducement Grant Under NASDAQ Listing Rule 5635(c)(4): In connection with the appointment of Mr. Christopher Lindblom as vice president, finance and treasurer at Infinity, the Compensation Committee of Infinity’s Board of Directors approved the grant of an option to Mr. Lindblom to purchase 40,000 shares of Infinity’s common stock. The option was granted outside of the company’s equity incentive plans and was made as an inducement material to Mr. Lindblom’s acceptance of employment. The option has an exercise price of $5.87 per share, which is equal to the closing price of Infinity’s common stock on May 2, 2016. One-fourth of the shares underlying Mr. Lindblom’s option will vest on the one year anniversary of his date of hire and thereafter 1/48th of the shares underlying Mr. Lindblom’s option will vest monthly, such that the shares underlying the option will be fully vested on the fourth anniversary of his date of hire, subject to his continued employment with Infinity on each such vesting date.
First Quarter 2016 Financial Results

At March 31, 2016, Infinity had total cash, cash equivalents and available-for-sale securities of $193.0 million, compared to $245.2 million at December 31, 2015.

Revenue during the first quarter of 2016 was $9.3 million for research and development (R&D) services associated with the strategic collaboration with AbbVie for duvelisib in oncology, compared to $4.4 million for R&D services for the first quarter of 2015.

R&D expense for the first quarter of 2016 was $39.2 million, compared to $88.4 million for the first quarter of 2015. R&D expense for the first quarter of 2015 included a $52.5 million payment related to the exercise of an option to buy out the company’s royalty obligations to Takeda Pharmaceutical Company Limited for duvelisib worldwide oncology sales. Excluding the $52.5 million payment in 2015, the increase in R&D expense was primarily due to higher clinical development expenses for duvelisib as well as an increase in staffing.

General and administrative (G&A) expense was $10.8 million for the first quarter of 2016, compared to $8.6 million for the same period in 2015. The increase in G&A expense was primarily related to an increase in staffing as well as external commercial expenses in preparation for the potential launch of duvelisib in 2017.

Net loss for the first quarter of 2016 was $40.7 million, or a basic and diluted loss per common share of $0.82, compared to $93.3 million, or a basic and diluted loss per common share of $1.91, for the same period in 2015.
Conference Call Information
Infinity will host a conference call today, May 4, 2016, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 89736154. An archived version of the webcast will be available on Infinity’s website for 30 days.

About Duvelisib
Duvelisib is an investigational, oral, dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, two proteins with predominantly non-overlapping roles known to support the growth and survival of malignant B-cells.[i] Preclinical data suggest that PI3K-delta signaling can lead to the proliferation of malignant B-cells, and both PI3K-gamma and PI3K-delta play a role in the formation and maintenance of the supportive tumor microenvironment.[ii] Duvelisib is the only investigational PI3K-delta,gamma inhibitor in Phase 3 clinical development and has the potential to be a first-in-class treatment for certain types of hematologic malignancies, or blood cancers. AbbVie and Infinity Pharmaceuticals, Inc. are jointly developing duvelisib in oncology.

Infinity and AbbVie are conducting a broad clinical development program evaluating duvelisib in patients with hematologic malignancies. In addition to DYNAMO and DUO, ongoing studies include BRAVURA, a Phase 3, double-blind, placebo-controlled study in patients with relapsed iNHL; FRESCO, a Phase 2 study in patients with relapsed/refractory follicular lymphoma; CONTEMPO, a Phase 1b/2 study in treatment-naïve patients with follicular lymphoma, and SYNCHRONY, a Phase 1b study in CLL patients previously treated with a Bruton’s tyrosine kinase (BTK) inhibitor. AbbVie has also initiated a clinical study in duvelisib in combination with Venclexta (venetoclax) in patients with relapsed or refractory CLL, small lymphocytic lymphoma, iNHL or aggressive NHL, as well as a Phase 1 study of duvelisib in Japanese subjects with relapsed or refractory lymphoma. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.

About IPI-549
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 inhibits immune suppressive macrophages within the tumor microenvironment, whereas other immunotherapies such as checkpoint modulators more directly target immune effector cell function. As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

Duvelisib and IPI-549 are investigational compounds and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On May 04, 2016 CombiMatrix Corporation (NASDAQ:CBMX), a molecular diagnostics company specializing in DNA-based testing services for pre-implantation genetic diagnostics and screening, miscarriage analysis, prenatal and pediatric diagnostics, reported financial results for the three months ended March 31, 2016 (Press release, CombiMatrix, MAY 4, 2016, View Source [SID:1234511927]).

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"We are reporting another exceptional quarter of financial progress, with 28% revenue growth, expanded gross margin, increased cash collections and well managed operating expenses," said Mark McDonough, CombiMatrix President and CEO. "Our growth was driven by a 39% increase in reproductive health revenue on an 18% increase in test volume, reflecting higher average revenue per test. We are particularly pleased with our performance in miscarriage analysis testing, with revenues up 43% on 13% test volume growth. We also benefited from better productivity from our newer sales representatives, which contributed to an 18% increase in our customer base.

"We anticipate that various industry dynamics will favorably impact our business," he added. "In March, the two leading associations in women’s healthcare, the American College of Obstetricians and Gynecologists (ACOG) and the Society for Maternal-Fetal Medicine (SMFM), issued revised practice bulletins recommending that all women regardless of age or other risk factors are offered prenatal genetic testing. These bulletins reiterated the need to perform confirmatory testing when screening tests reveal positive results for fetal abnormalities. Additionally, a number of health plans, including Cigna and several in the Blue Cross Blue Shield network, have recently revised their medical policies to cover chromosomal microarray testing for recurrent pregnancy loss. We believe that more health plans will follow suit based on the growing clinical support for this valuable patient information.

"We are firmly focused on growth and a path toward profitability, supported by tight execution of our business strategy," said Mr. McDonough. "This year we plan to expand our IVF testing portfolio and expect to increase physician adoption through more clinical validation, improved marketing developed from the insights from our newly appointed Scientific Advisory Board, and greater productivity from our sales organization. We also are seeking opportunities to build upon our leadership position in the growing reproductive health diagnostics market and enhance shareholder value through partnerships and other business development alternatives. With the completion of an $8 million financing in late March, we are well positioned to execute on our plans."

First Quarter Financial and Operational Highlights (all comparisons are with the first quarter of 2015)

Total revenues of $3.0 million, up 28%
Reproductive health revenues of $2.2 million, up 39%
Reproductive health test volume of 1,426, up 18%
Number of billable customers reaches 264, up 18%
Cash collections of $2.5 million, up 15%
Closed $8.0 million underwritten public offering in March of 2016
Launched CombiPGD – Preimplantation Genetic Diagnosis for Single Gene Disorders and Chromosomal Translocations
Formed Scientific Advisory Board

Volumes
Revenues (in 000’s)
Q1 ’16 Q1 ’15 # Δ % Δ Q1 ’16 Q1 ’15 $ Δ % Δ
Prenatal 264 324 (60 ) (19 %) $ 321 $ 422 $ (101 ) (24 %)
Miscarriage analysis 995 882 113 13 % 1,622 1,132 490 43 %
PGS 167 - 167 - 222 - 222 -
Subtotal – reproductive health 1,426 1,206 220 18 % 2,165 1,554 611 39 %
Pediatric 452 467 (15 ) (3 %) 500 497 3 1 %
Subtotal – all arrays 1,878 1,673 205 12 % 2,665 2,051 614 30 %
Non-array tests 770 672 98 15 % 265 236 29 12 %
Total – all tests 2,648 2,345 303 13 % 2,930 2,287 643 28 %
Royalties 42 42 - 0 %
Total revenues $ 2,972 $ 2,329 $ 643 28 %

Financial Results

Total revenues for the first quarter of 2016 increased 28% to $3.0 million from $2.3 million for the first quarter of 2015. Revenues for the first quarter of 2016 were comprised of $2.9 million of diagnostic services revenue and $42,000 in royalties. Reproductive health diagnostic test revenue, which includes prenatal microarrays, miscarriage analysis and PGS, increased 39% to $2.2 million and related testing volumes increased 18% to 1,426. The first quarter 2016 revenue increase was driven partially by higher test volumes, particularly from miscarriage analysis and PGS testing, but primarily by higher average revenue per test in miscarriage analysis testing.

Total operating expenses were $4.4 million for the first quarter of 2016, compared with $4.1 million for the first quarter of 2015. The increase was due primarily to an increase in cost of services related to higher test volumes and from an increase in sales and marketing expenses related to sales force expansion. Gross margins for the first quarter of 2016 improved to 51.6% from 46.2% for the first quarter of 2015 due primarily to improved average reimbursement on miscarriage analysis testing.

The net loss for the first quarter of 2016 was $1.5 million, or $1.73 per share, compared with a net loss for the first quarter of 2015 of $1.8 million, or $2.24 per share. Net loss attributable to common stockholders for the first quarter of 2016 was $3.1 million, or $3.63 per share, compared with a net loss attributable to common stockholders for the first quarter of 2015 of $2.7 million, or $3.37 per share. The higher net loss attributable to common stockholders in 2016 reflected one-time, non-cash charges of $1.9 million related to deemed dividends from the issuance of Series F convertible preferred stock and warrants in the $8.0 million public offering that closed on March 24, 2016. This increase was partially offset by the reversal of the $890,000 Series E deemed dividend recognized in 2015 from the repurchase of those securities upon closing of our public offering, partially reduced by the $656,000 deemed dividend paid to the Series E investors in February of 2016.

The Company reported $6.6 million in cash, cash equivalents and short-term investments as of March 31, 2016, compared with $3.9 million as of December 31, 2015. The Company used $1.7 million in cash to fund operating activities during the first quarter of 2016, compared with $1.2 million to fund operating activities during the comparable period in 2015. The increase in net cash used to fund operating activities in 2016 resulted primarily from the timing of the purchase of inventory supplies, coupled with higher overall headcount in early 2016 compared with 2015.

On May 4, 2016 bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the first quarter ended March 31, 2016 (Press release, bluebird bio, MAY 4, 2016, View Source;p=RssLanding&cat=news&id=2165083 [SID:1234511925]).

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"In early 2016 we achieved two crucial clinical milestones: treating the first patient in the Phase 1 study of our anti-BMCA CAR T therapy bb2121, and presenting the first clinical data from our Starbeam study of Lenti-D in boys with CALD. We are very pleased with this significant progress as we continue to build our T cell immunotherapy and HSC gene therapy platforms," said Nick Leschly, chief bluebird. "On the LentiGlobinTM program, we are on track to achieve our remaining milestones this year, which include initiation of the HGB-207 study in non-ß0/ß0 transfusion-dependent thalassemia (TDT) as well as integration of manufacturing process improvements into our LentiGlobin clinical trials."

Recent Highlights

PRESENTED INTERIM DATA FROM STARBEAM STUDY AT AAN – In April, Dr. Florian Eichler of Massachusetts General Hospital for Children presented interim clinical data from the Starbeam study of Lenti-D in CALD at AAN. Initial Starbeam results suggest Lenti-D gene therapy may have similar efficacy to allogeneic hematopoietic stem cell transplant (HCT), the current standard of care, with a more favorable safety profile. As of March 31, 2016, three of the 17 patients enrolled in the study have reached two years of follow-up and remain free of major functional disabilities (MFDs), the primary endpoint of the study. Sixteen of the 17 patients had stabilization of their neurological function score (NFS), and 14 of 17 had a stable Loes score. The safety profile of Lenti-D treatment appeared consistent with myeloablative conditioning.

TEN ABSTRACTS ACCEPTED FOR PRESENTATION AT ASGCT (Free ASGCT Whitepaper) 19th ANNUAL MEETING – Two oral presentations given by bluebird’s academic collaborators will highlight previously presented data from bluebird bio’s ongoing gene therapy clinical trials, including interim data from the Starbeam Study of Lenti-D in cerebral adrenoleukodystrophy, and interim data from the HGB-205 study of LentiGlobin in severe sickle cell disease and TDT. Eight additional presentations will be featured at the meeting, highlighting progress across the company’s preclinical, research and process development activities in both HSC gene therapy and T cell immunotherapy.

TREATED FIRST PATIENT IN PHASE 1 STUDY OF BB2121 IN MULTIPLE MYELOMA – In February, the first patient was infused in the CRB-401 study of anti-BCMA CAR T therapy bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene exercised its option to exclusively license bb2121. Under the terms of the collaboration agreement between the two companies, bluebird bio received a $10.0 million option exercise payment from Celgene and may now elect to co-develop and co-promote the product candidate in the United States with Celgene. We are also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.

FULLY ENROLLED EXPANDED NORTHSTAR STUDY – Achievement of 18 patient enrollment target in Northstar Study of LentiGlobin in patients with transfusion-dependent thalassemia, including three additional adolescent patients.
Upcoming Anticipated Milestones

Update on LentiGlobin process improvements in the second half of 2016
Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016
Presentation of updated clinical data for LentiGlobin at the ASH (Free ASH Whitepaper) annual meeting in December 2016
First Quarter 2016 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2016 were $826.9 million, compared to $865.8 million as of December 31, 2015, a decrease of $38.9 million.
Revenues: Collaboration revenue was $1.5 million for the first quarter of 2016 compared to $6.3 million for first quarter of 2015. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.
R&D Expenses: Research and development expenses were $41.9 million for the first quarter of 2016 compared to $23.7 million for the first quarter of 2015. The increase in research and development expenses was primarily attributable to increased employee compensation and facilities costs due to increased headcount, and increased manufacturing, clinical, research, and information technology costs to support the advancement of our clinical and pre-clinical programs.
G&A Expenses: General and administrative expenses were $16.0 million for the first quarter of 2016 compared to $7.3 million for the first quarter of 2015. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting costs to support our overall growth.
Net Loss: Net loss was $56.3 million for the first quarter of 2016 compared to $24.8 million for the first quarter of 2015.
Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $826.9 million as of March 31, 2016 will be sufficient to fund its current operations through 2018.

On May 4, 2016 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported its financial results for the first quarter were in line with the company’s expectations (Press release, Ionis Pharmaceuticals, MAY 4, 2016, View Source;p=RssLanding&cat=news&id=2164982 [SID:1234511917]).

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"2016 is off to a strong start. Target enrollment is complete in four Phase 3 studies across nusinersen, IONIS-TTRRx and volanesorsen. These important new drugs are now one step closer to potentially reaching the market and being available to patients. We are actively engaged in NDA preparations for all three of these drugs in anticipation of Phase 3 data from each in the first half of next year," said B. Lynne Parshall, chief operating officer of Ionis Pharmaceuticals. "We are pleased to have chosen a new commercial partner for Kynamro. We believe Kastle Therapeutics has the rare disease expertise, financial resources and initiative to expand the market potential for Kynamro. Already Kastle has begun to identify new patients to bring onto Kynamro therapy in the United States, and it is initiating activities to pursue marketing approval in other countries."

"Just last month at the AAN meeting, we provided an update on our Phase 2 open-label study with a data cut-off of January 26, 2016 in infants with SMA treated with nusinersen. The infants in this study are continuing to improve. At the AAN meeting, we reported that we had no deaths or events of permanent ventilation since late 2014. All of the continuing infants are over two years old and several have passed their third birthday. We are especially encouraged by continued improvements in motor function in these infants, as evidenced by both continued increases in muscle function scores and achievement of developmental milestones that infants with Type 1 SMA are never expected to achieve, including sitting, standing and even walking. These data give us further confidence in our two Phase 3 studies in infants and children with SMA, from which we expect to have data in the first half of 2017. We continue to work closely with our partner, Biogen, who is preparing to bring this drug to the market for infants and children with SMA," continued Ms. Parshall.

"Also at the AAN meeting, we and our collaborators presented more than a dozen presentations and posters highlighting our broad neurological disease pipeline, which is focused on treating diseases that have been largely untreatable using other therapeutic modalities. Over the last several years, we have substantially expanded the reach of our technology to address a large number of disease targets in the central nervous system," said Ms. Parshall. "We continue to increase the value of our pipeline and technology by expanding the use of our antisense technology into new disease areas, new targets, new tissues and new mechanisms. We hope that you will join us in July for our R&D day during which we will be discussing our broad, innovative pipeline and the groundbreaking work we are doing to continue to broaden the application of our technology," concluded Ms. Parshall.

Financial Results

"We finished the first quarter of 2016 with a pro forma net operating loss of $35 million and more than $700 million in cash. On a GAAP basis, our operating loss was $55 million. In the first quarter, we earned $37 million of revenue including more than $15 million in milestone payments, the majority of which were related to the progression of our Phase 3 program for nusinersen. As nusinersen and our other partnered programs advance, we have the opportunity to earn significant revenue this year. We are eligible to earn up to $95 million in upfront and milestone payments from Kastle, $15 million of which we will recognize in the second quarter. We will receive a ten percent common equity position in Kastle’s parent company. Starting in 2017, we are also entitled to royalties that average in the mid to low teens on global sales of Kynamro. Additionally, our financial projections include numerous significant milestone payments in the second half of this year, including a $55 million milestone payment from Bayer related to advancing IONIS-FXIRx, compared to 2015 when our revenue was more evenly spread throughout the year. We also have the opportunity to earn a $25 million milestone payment for advancing the first drug under our cardiometabolic collaboration with AstraZeneca and a $10 million milestone payment for advancing the first drug under our J&J collaboration. We also have numerous opportunities during the remainder of 2016 to earn meaningful milestone payments as we advance research programs and drugs under our Biogen collaborations," said Elizabeth L. Hougen, chief financial officer of Ionis Pharmaceuticals.

"Our pro forma operating expenses for the first quarter were $71 million, a significant portion of which were associated with the five Phase 3 studies and three open-label extension studies related to these Phase 3 studies, we are conducting. In addition, Akcea continues to build its infrastructure and conduct the pre-commercialization activities necessary to launch volanesorsen. We are doing all of this while managing our expenses prudently. On a GAAP basis, our operating expenses were $92 million," continued Ms. Hougen.

"Our first quarter financial results were in line with our expectations and we are on track to meet our 2016 guidance of a pro forma NOL in the low $60 million range and a year-end cash balance in excess of $600 million," concluded Ms. Hougen.

All pro forma amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of pro forma and GAAP measures, which is provided later in this release.

Revenue

Ionis’ revenue for the three months ended March 31, 2016 was $36.9 million, compared to $62.6 million for the same period in 2015. Ionis’ revenue in the first quarter of 2016 included the following:

$12.5 million from Biogen for advancing the Phase 3 program for nusinersen and advancing IONIS-BIIB4Rx;
$1.5 million from GSK for advancing IONIS-HBV-LRx; and
$22.9 million primarily from the amortization of upfront fees and manufacturing services Ionis performed for its partners.
Ionis’ revenue fluctuates based on the nature and timing of payments under agreements with its partners and consists primarily of revenue from the amortization of upfront fees, milestone payments and license fees. The Company’s financial projections include numerous significant milestone payments in the second half or this year, compared to 2015 when its revenue was more evenly spread throughout the year.

Operating Expenses
Ionis’ operating expenses included costs to support the Company’s ongoing Phase 3 studies for nusinersen, IONIS-TTRRx and volanesorsen. In addition, Akcea continued to build its operations in preparation for the commercial launch of volanesorsen. As such, Ionis’ pro forma operating expenses were $71.4 million for the three months ended March 31, 2016, and increased compared to $58.6 million for the same period in 2015. On a GAAP basis, Ionis’ operating expenses for the three months ended March 31, 2016 were $91.5 million, compared to $71.9 million for the same period in 2015. Ionis’ operating expenses on a GAAP basis included non-cash compensation expense related to equity awards, which increased because the average fair value of unvested stock options has risen due to the increase in the exercise price of the stock options the Company has granted over the past several years.

Net Loss
Ionis reported a net loss of $62.9 million for the three months ended March 31, 2016, compared to a net loss of $16.7 million for the same period in 2015. Basic and diluted net loss per share for the three months ended March 31, 2016 was $0.52 compared to $0.14 for the same period in 2015. Ionis’ net loss increased for the three months ended March 31, 2016 compared to the same period in 2015 primarily due to variations in the timing of revenue from milestone payments and to a lesser extent, an increase in operating expenses primarily associated with the Company’s Phase 3 studies.

Balance Sheet
As of March 31, 2016, Ionis had cash, cash equivalents and short-term investments of $703.8 million compared to $779.2 million at December 31, 2015. Ionis’ cash balance decreased in 2016 primarily due to spending to support the Company’s ongoing Phase 3 programs for nusinersen, IONIS-TTRRx and volanesorsen. Ionis’ working capital was $639.4 million at March 31, 2016 compared to $688.1 million at December 31, 2015.