oN aPRIL 25, 2017 Biogen Inc. (NASDAQ: BIIB) reported first quarter 2017 financial results (Filing, Q1, Biogen, 2017, APR 25, 2017, View Source [SID1234518688]).

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Total revenues of $2.8 billion, a 3% increase versus the prior year and an 8% increase excluding hemophilia revenues*.
TYSABRI revenue grew 14% versus the prior year. Outside the U.S., TYSABRI revenues benefited by approximately $45 million due to reaching an agreement with the Price and Reimbursement Committee of the Italian National Medicines Agency (AIFA) related to TYSABRI sales in prior periods.
Versus Q4 2016, Biogen estimates TECFIDERA U.S. revenues were negatively impacted by approximately $50 million to $60 million due to lower inventory levels in the channel.
Worldwide SPINRAZA revenues were $47 million.
GAAP net income and diluted earnings per share (EPS) attributable to Biogen Inc. of $748 million and $3.46, respectively.
GAAP net income and diluted EPS were negatively impacted by $263 million and $1.22, net of tax, respectively, related to the settlement and license agreement with Forward Pharma including consideration of the U.S. Patent and Trademark Office (USPTO) ruling in favor of Biogen in the interference proceeding.
Non-GAAP net income and diluted EPS attributable to Biogen Inc. of $1.1 billion and $5.20, respectively.
* Total Q1 2017 revenues include hemophilia revenues only for the month of January. The 8% increase in total revenues excludes all hemophilia revenues from Q1 2016 and January 2017. Hemophilia revenues include ELOCTATE and ALPROLIX product revenues as well as royalty and contract manufacturing revenue related to Sobi.


(In millions, except per share amounts) Q1 ’17 Q4 ’16 Q1 ’16
Q1 ’17 v.
Q4 ’16
Q1 ’17 v.
Q1 ’16
Total revenues** $ 2,811 $ 2,872 $ 2,727 (2%)** 3%**

GAAP net income*** $ 748 $ 649 $ 971 15 % (23 %)
GAAP diluted EPS $ 3.46 $ 2.99 $ 4.43 16 % (22 %)

Non-GAAP net income*** $ 1,123 $ 1,093 $ 1,049 3 % 7 %
Non-GAAP diluted EPS $ 5.20 $ 5.04 $ 4.79 3 % 9 %

** Total revenues grew 4% versus Q4 2016 and 8% versus Q1 2016 excluding hemophilia.
***Net income attributable to Biogen Inc.

A reconciliation of GAAP to Non-GAAP quarterly financial results can be found in Table 3 at the end of this press release.

"I am very pleased with the results of the first quarter. We saw continued stability in our MS business, executed a strong launch of SPINRAZA, grew market share for our biosimilars business across Europe, and reinforced the intellectual property for TECFIDERA," said Chief Executive Officer Michel Vounatsos. "Furthermore, we continued to build our neurology pipeline with the anticipated addition of our new Phase 2-ready anti-tau antibody."

"We are encouraged by the progress we made launching SPINRAZA in the U.S., and, following the positive CHMP opinion, we are ramping up pre-launch activities in Europe. The value this therapy provides to patients is compelling, and we are working to accelerate patient access globally," Vounatsos continued. "Overall, I believe we’re building positive momentum at the company, and I look forward to leading Biogen into a new and exciting era."


Revenue Highlights

(In millions) Q1 ’17 Q4 ’16 Q1 ’16
Q1 ’17 v.
Q4 ’16
Q1 ’17 v.
Q1 ’16
Multiple Sclerosis:
TECFIDERA $ 958 $ 1,002 $ 946 (4 %) 1 %
Total Interferon $ 648 $ 688 $ 670 (6 %) (3 %)
AVONEX $ 537 $ 564 $ 564 (5 %) (5 %)
PLEGRIDY $ 112 $ 125 $ 106 (10 %) 5 %
TYSABRI $ 545 $ 474 $ 477 15 % 14 %
FAMPYRATM $ 20 $ 22 $ 20 (7 %) 1 %
ZINBRYTA $ 11 $ 6 $ — 81 % NMF

Hemophilia:
ELOCTATE**** $ 48 $ 149 $ 108 (68 %) (55 %)
ALPROLIX**** $ 26 $ 93 $ 75 (72 %) (65 %)

Spinal Muscular Atrophy
SPINRAZA $ 47 $ 5 $ — NMF NMF

Other Product Revenues:
Biosimilars $ 66 $ 53 $ 2 25 % NMF
FUMADERMTM $ 10 $ 11 $ 11 (15 %) (15 %)

Total Product Revenues: $ 2,380 $ 2,503 $ 2,309 (5 %) 3 %

Anti-CD20 Revenues $ 341 $ 318 $ 329 7 % 3 %
Other Revenues**** $ 90 $ 51 $ 88 77 % 2 %

Total Revenues** $ 2,811 $ 2,872 $ 2,727 (2%)** 3%**

Note: Numbers may not foot due to rounding; percent changes represented as favorable & (unfavorable)
**** Q1 2017 ELOCTATE and ALPROLIX revenues reflect only the month of January, prior to the spin-off of our hemophilia business. Other revenues include royalty and contract manufacturing revenue related to Sobi only for the month of January.


Expense Highlights

(In millions) Q1 ’17 Q4 ’16 Q1 ’16
Q1 ’17 v.
Q4 ’16
Q1 ’17 v.
Q1 ’16
GAAP cost of sales $ 385 $ 378 $ 313 (2 %) (23 %)
Non-GAAP cost of sales $ 385 $ 363 $ 313 (6 %) (23 %)

GAAP R&D $ 423 $ 534 $ 437 21 % 3 %
Non-GAAP R&D $ 421 $ 531 $ 437 21 % 4 %

GAAP SG&A $ 499 $ 496 $ 497 (1 %) (0 %)
Non-GAAP SG&A $ 483 $ 484 $ 497 0 % 3 %
Note: Percent changes represented as favorable & (unfavorable)

Biogen also recorded GAAP-only pre-tax charges in Q1 2017 of $354 million related to the settlement and license agreement with Forward Pharma including consideration of the USPTO ruling in favor of Biogen in the interference proceeding. These charges are included in amortization of acquired intangible assets and exceed the amounts anticipated in Biogen’s previously announced 2017 full year GAAP financial guidance related to this agreement.
Other Financial Highlights

As of March 31, 2017, Biogen had cash, cash equivalents and marketable securities totaling approximately $5.7 billion, and approximately $6.5 billion in notes payable and other financing arrangements.
For the first quarter of 2017, the Company’s weighted average diluted shares were approximately 216 million. The Company ended the quarter with approximately 214 million basic shares outstanding.
During the first quarter of 2017, Biogen repurchased approximately 2 million shares of the Company’s common stock for a total value of $584 million. Since the end of the quarter, the Company has repurchased an additional approximately 2 million shares for a total value of $543 million.
Business Development Highlights

In April 2017, Biogen announced an agreement with Bristol-Myers Squibb to exclusively license BMS-986168, an experimental medicine with potential in Alzheimer’s disease and progressive supranuclear palsy (PSP), a rare condition that affects movement, speech, vision, and cognitive function. Biogen plans to initiate Phase 2 studies for BMS-986168 in both of these indications. Biogen anticipates making an upfront payment of $300 million to Bristol-Myers Squibb in the second quarter of 2017 as well as a near-term $60 million milestone payment to the former stockholders of iPierian, Inc. upon initiation of a Phase 2 trial for BMS-986168. These amounts exceed the estimated $100 million in business development expense assumed in Biogen’s previously announced 2017 full year financial guidance. This agreement is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 in the United States, and is expected to close in the second quarter of 2017.
Other Recent Events

At the 69th annual meeting of the American Academy of Neurology (AAN) being held in Boston from April 22 to April 28, 2017, Biogen is presenting data from its portfolio of treatments and investigational therapies for people with serious neurological and neurodegenerative diseases. Platform and poster presentations include new real-world evidence supporting TECFIDERA and TYSABRI, underscoring the importance of early and appropriate treatment for multiple sclerosis (MS); new data demonstrating the clinically meaningful efficacy and favorable benefit-risk profile of SPINRAZA for spinal muscular atrophy; and previously presented results from the Phase 1b studies of aducanumab, an investigational treatment for early Alzheimer’s disease.
In April 2017, Biogen announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion recommending the granting of a marketing authorization in the European Union (EU) for SPINRAZA (nusinersen) to treat patients with spinal muscular atrophy. The CHMP reviewed SPINRAZA under an accelerated assessment procedure, which is a regulatory mechanism to facilitate earlier access to patients for medicines that fulfill unmet medical needs. SPINRAZA is the first treatment for spinal muscular atrophy to be recommended by the CHMP for approval in the EU.
In March 2017, the USPTO ruled against the Coalition for Affordable Drugs V LLC, an entity associated with a hedge fund, in the inter partes review of Biogen’s U.S. Patent No. 8,399,514 (the ‘514 patent). The ‘514 patent includes claims covering the treatment of MS with 480 mg of dimethyl fumarate as provided for in Biogen’s TECFIDERA label.
In March 2017, the USPTO ruled against Forward Pharma in the interference proceeding between Forward Pharma’s pending U.S. Patent Application No. 11/576,871 and the ‘514 patent.
In March 2017, Biogen presented data from its Alzheimer’s and Parkinson’s disease programs at the 13th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in Vienna, Austria. The Biogen presentations included data from research of Alzheimer’s and Parkinson’s disease biomarkers; the investigational treatment for Alzheimer’s disease, aducanumab; and the investigational treatment for Parkinson’s disease, BIIB054.
In March 2017, Roche announced that the U.S. Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) voted unanimously (11 to 0) that the benefit-risk of rituximab/hyaluronidase for subcutaneous (under the skin) injection was favorable for the treatment of certain blood cancers. This new co-formulation includes the same monoclonal antibody as intravenous RITUXAN (rituximab) and hyaluronidase, a molecule that helps to deliver medicine under the skin. The FDA is expected to make a decision on approval by June 26, 2017. Roche and Biogen collaborate on RITUXAN in the U.S.
In March 2017, Biogen appointed Anirvan Ghosh, Ph.D. as Senior Vice President, Research and Early Development (RED). Dr. Ghosh will lead Biogen’s RED organization in the discovery and development of drug candidates from idea through proof of concept.
In February 2017, Biogen announced the completion of the separation of its global hemophilia business. The new company, known as Bioverativ, is an independent, publicly traded global biotechnology company focused on hemophilia and other rare blood disorders. Bioverativ trades under the symbol "BIVV" on the NASDAQ Global Select Market.
In January 2017, Siemens Healthineers and Biogen announced plans to jointly develop magnetic resonance imaging (MRI) applications with the intent of quantifying key markers of MS disease activity and progression.
In January 2017, Biogen initiated a Phase 1 trial of an anti-tau monoclonal antibody, BIIB076, in healthy volunteers and participants with Alzheimer’s disease. BIIB076 was derived from Neurimmune’s reverse translational medicine platform.

On April 25, 2017 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene edited CAR T-cells, reported that data on its gene-edited allogeneic off-the-shelf CAR T-cell immunotherapies (UCART) will be presented at the ASGCT (Free ASGCT Whitepaper) 20th Annual Meeting (Press release, Cellectis, APR 25, 2017, View Source [SID1234518685]) . The meeting will be held from May 10th to 13th, 2017 in Washington, D.C., USA.

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Oral presentation:

Development of Gene Edited Allogeneic CAR T-Cell Therapy
Philippe Duchateau, PhD. Chief Executive Officer, Cellectis

Session: 300 – Clinical Advancement of Gene Editing-Moving New Science to the Clinic – Organized by the Clinical Trials and Regulatory Affairs Committee
Friday, May 12, 2017 from 8:35 AM to 9:10 AM EST
Lincoln 2, 3, 4

Poster presentations:

176 – Genome-Wide Analysis of TALEN Activity in Primary Cells
Brian Busser, Sonal Temburni, Aymeric Duclert, Philippe Duchateau and Laurent Poirot

Session: Gene Targeting and Gene Correction I
Wednesday May 10, 2017 at 5:30 PM EST
Exhibit Hall A & B South

114 – UCART22: An Allogeneic Adoptive Immunotherapy for Leukemia Targeting CD22 with CAR T-cells
Anne-Sophie Gautron, Cécile Schiffer-Mannioui, Alan Marechal, Severine Thomas, Agnes Gouble, Laurent Poirot, Julianne Smith

Session: Cancer-Immunotherapy, Cancer Vaccines I
Wednesday May 10, 2017 from 5:30 PM to 7:30pm EST
Exhibit Hall A & B South

372 – Manufacturing of Gene-Modified Mouse CAR T-Cells
Laurent Poirot, Brian Busser, Sonal Temburni, Philippe Duchateau

Session: Gene Targeting and Gene Correction II
Thursday May 11, 2017 from 5:15 PM to 7:15 PM EST
Exhibit Hall A & B South

On April 25, 2017 VBL Therapeutics (NASDAQ:VBLT), reported that new data on VB-111 will be presented at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Annual Meeting, to be held June 2-6 in Chicago, Illinois, as well as in the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper), to be held May 10-13 in Washington, DC (Press release, VBL Therapeutics, APR 25, 2017, View Source [SID1234518683]).

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Data from the Phase 2 clinical trial that investigated VB-111 in recurrent glioblastoma (rGBM) will be presented at ASCO (Free ASCO Whitepaper), as detailed below:

Title:

Treatment Through Progression with Ofranogene Obadenovec
(VB-111), an Anti-Cancer Viral Therapy, Significantly
Attenuates Tumor Growth in Recurrent GBM: Individual Phase
2 Patient Data
Abstract: 2055
Session Type: Poster Session
Session Title: Central Nervous System Tumors
Date: June 5, 2017
Time: 1:15 – 4:45 Central Time
Location: Hall A
Poster Board: #297
Presenter:

Andrew Jacob Brenner, MD, Ph.D., The University of Texas
Health Science Center at San Antonio, et al.
Previously announced data from this Phase 2 clinical trial showed prolongation of overall survival in rGBM patients treated with a combination of VB-111 and Avastin (bevacizumab) compared to limited exposure to VB-111 and to historical controls. This poster at ASCO (Free ASCO Whitepaper) will feature data on individual patients who were enrolled in this trial.

In addition, preclinical data demonstrating that VB-111 augments the anti-tumor activity of PD-L1 blockade will be presented at the ASGCT (Free ASGCT Whitepaper), as detailed below:

Title:

Vascular targeting viral therapy augments PD-L1 checkpoint
blockade anti-tumor activity
Session Date/Time: Friday, May 12, 2017 5:45 PM – 7:45 PM
Session title: Cancer-Immunotherapy, Cancer Vaccines III
Room: Exhibit Hall A & B South
Abstract number: 585
About Ofranergene Obadenovec (VB-111)
Ofranergene obadenovec is a unique biologic agent that uses a dual mechanism to target solid tumors. Based on a non-integrating, non-replicating, Adeno 5 vector, ofranergene obadenovec utilizes VBL’s proprietary Vascular Targeting System (VTS) to target the tumor vasculature for cancer therapy. Unlike anti-VEGF or TKIs, ofranergene obadenovec does not aim to block a specific pro-angiogenic pathway; instead, it uses an angiogenesis-specific sensor (VBL’s PPE-1-3x proprietary promoter) to specifically induce cell death in angiogenic endothelial cells in the tumor milieu. This mechanism retains activity regardless of baseline tumor mutations or the identity of the pro-angiogenic factors secreted by the tumor and shows activity even after failure of prior treatment with other anti-angiogenics. Moreover, ofranergene obadenovec induces specific anti-tumor immune response, which is accompanied by recruitment of CD8 T-cells and apoptosis of tumor cells.

Ofranergene obadenovec completed a Phase 2 study in rGBM, which showed a statistically significant improvement in overall survival in patients treated with ofranergene obadenovec through progression, compared to either patients treated with ofranergene obadenovec followed by bevacizumab alone, or to historical bevacizumab data. In a Phase 2 trial for recurrent platinum-resistant ovarian cancer, ofranergene obadenovec demonstrated a statistically significant increase in overall survival and 60% durable response rate (as measured by reduction in CA-125), approximately twice the historical response with bevacizumab plus chemotherapy in ovarian cancer. In a Phase 2 study in recurrent, iodine-resistant differentiated thyroid cancer, ofranergene obadenovec met the primary endpoint demonstrating disease stabilization with a positive safety profile, along with a dose-response and evidence of an overall survival benefit. Ofranergene obadenovec has received Fast Track Designation for recurrent glioblastoma in the U.S. and orphan drug status for glioblastoma in both the U.S. and EU.

On April 25, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to tazemetostat, the Company’s first-in-class EZH2 inhibitor, for the treatment of patients with relapsed or refractory follicular lymphoma, either wild type EZH2 or with EZH2 activating mutations (Press release, Epizyme, APR 25, 2017, View Source [SID1234518681]). Fast Track designation is intended to provide expedited processes for the development and FDA review of drugs that may reduce development time and costs associated with bringing a drug to market.

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Epizyme also announced that interim efficacy and safety data from all five study cohorts in its ongoing Phase 2 study of tazemetostat in patients with relapsed or refractory follicular lymphoma and diffuse large B-cell lymphoma (DLBCL) has been selected for a plenary session on Wednesday, June 14, 2017 at 2:00 p.m. CET at the International Conference on Malignant Lymphoma (ICML) in Lugano, Switzerland. In addition, results from a biomarker study of tazemetostat in patients with NHL will be presented in a poster session during ICML. The Company plans to hold a conference call to discuss these clinical findings on Wednesday, June 14 at 10:30 a.m. ET.

"This is an important milestone for our NHL program, with tazemetostat now having FDA Fast Track designation for relapsed or refractory diffuse large B-cell lymphoma with EZH2 activating mutations and for relapsed or refractory follicular lymphoma, regardless of EZH2 mutation," said Robert Bazemore, president and chief executive officer, Epizyme. "In addition to this regulatory recognition of tazemetostat’s therapeutic potential, the selection of interim Phase 2 data for the opening plenary session underscores the lymphoma community’s enthusiasm for our lead product candidate. Our development goal is to bring tazemetostat to patients as quickly as possible and we look forward to advancing this study throughout 2017."

The FDA Fast Track program is designed to facilitate the development of important new drugs and to provide patients access to those drugs more quickly. The designation enables early and frequent communication between FDA and a product sponsor throughout the drug development and review process. Through the Fast Track program, a product may be eligible for priority review at the time of a new drug application (NDA) filing and may also be eligible to submit completed sections of the NDA on a rolling basis before the complete application is submitted.

About the Tazemetostat Clinical Trial Program
Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied in ongoing Phase 2 programs in both follicular lymphoma and diffuse large B-cell lymphoma (DLBCL) forms of non-Hodgkin lymphoma; certain genetically defined solid tumors, including INI1-negative and SMARCA4-negative tumors and synovial sarcoma; and mesothelioma, as well as in combination studies in DLBCL. Tazemetostat has been granted Fast Track designation by the U.S. Food and Drug Administration for both relapsed/refractory follicular lymphoma with or without an EZH2 activating mutation and DLBCL with EZH2 activating mutations, as well as Orphan Drug designation for malignant rhabdoid tumors.

On April 25, 2017 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported that it has entered into a three-year collaboration with Duke University to evaluate VAL-083, the Company’s platform compound, as a front-line treatment for newly diagnosed patients with glioblastoma multiforme ("GBM") (Press release, DelMar Pharmaceuticals, APR 25, 2017, View Source [SID1234518680]).

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Under the terms of the collaboration, DelMar will fund a series of pre-clinical studies to be conducted by Duke University’s Glioblastoma Drug Discovery Group to evaluate VAL-083, either alone or in combination with other agents, for activity against a range of glioblastoma subtypes (personalized drug development). The goal of the research will be to identify molecular characteristics of GBM tumors that are more likely to respond to VAL-083, and not the standard of care, temozolomide, as a front-line treatment or through combination therapies.

"VAL-083 is a promising drug that has the potential to help many GBM patients, especially the over half of GBM patients whose tumors predominantly exhibit an unmethylated MGMT promoter and do not respond to temozolomide, the current standard of care," commented Dr. Madan Kwatra, Director of the Glioblastoma Drug Discovery Group at Duke University.

GBM is a deadly brain cancer in which the current standard of care consists of surgery followed by radiation and chemotherapy using temozolomide. However, temozolomide only works for about 40% of GBM patients that have a methylated MGMT promoter. In contrast, VAL-083 is a novel chemotherapeutic agent that has activity against GBM with both methylated and unmethylated MGMT promoters. As such, VAL-083 is a more versatile chemotherapeutic agent that may help a wider subset of GBM patients.

"We are delighted to be working with another top-tier research institute and in particular, Dr. Kwatra who is a world renowned expert in the GBM field," stated Jeffrey Bacha, chairman and chief executive officer of DelMar Pharmaceuticals. "The efforts of his department in the coming years will greatly contribute to our goal of optimizing VAL-083 as a next generation personalized solution for a broad range of cancers."

About VAL-083:

VAL-083 is a "first-in-class," small-molecule chemotherapeutic that demonstrated clinical activity against a range of cancers including GBM in historical clinical trials sponsored by the U.S. National Cancer Institutes. DelMar has demonstrated that VAL-083’s anti-tumor activity against GBM is unaffected by the expression of MGMT in vitro. Further details can be found at View Source

VAL-083 has received an orphan drug designation in Europe for the treatment of malignant gliomas and the U.S. FDA Office of Orphan Products has granted an orphan designation to VAL-083 for the treatment of glioma, medulloblastoma and ovarian cancer.

DelMar is currently conducting a Phase 2 clinical trial in patients with MGMT-unmethylated, Bevacizumab-naive, recurrent GBM in collaboration with the University of Texas MD Anderson Cancer Center. DelMar has also announced plans to advance VAL-083 into a pivotal randomized multi-center Phase 3 clinical trial for the treatment of bevacizumab-failed GBM and into a separate international Phase 2 trial for newly diagnosed MGMT-unmethylated GBM patients.

DelMar believes that data from its clinical trials, if successful, will form the basis of a new treatment paradigm for the vast majority of GBM patients whose tumors exhibit features that make them unlikely to respond to currently available therapies.

About Glioblastoma Multiforme (GBM)

GBM is the most common and the most lethal form of brain cancer. Approximately 15,000 new cases of GBM are expected to be diagnosed in the United States during 2017. GBM progresses quickly and patients deteriorate rapidly. Common symptoms include headaches, seizures, nausea, weakness, paralysis and personality or cognitive changes such as loss of speech or difficulty in thinking clearly. The majority of GBM patients do not survive for more than two years following diagnosis, and the median survival in newly diagnosed patients with best available treatments is less than 15 months.