Acceleron Pharma Reports Fourth Quarter and Year Ended 2015 Financial and Operational Results and Recent Highlights

On February 25, 2016 Acceleron Pharma Inc. (NASDAQ:XLRN), a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutic candidates that regulate cellular growth and repair, reported a corporate update and reported financial results for the fourth quarter and year ended December 31, 2015 (Press release, Acceleron Pharma, FEB 25, 2016, View Source [SID:1234509189]).

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"We made significant progress over the past year in advancing our pipeline, as marked by successful clinical results across our programs and the recent launch of Phase 3 programs in MDS and beta-thalassemia with our partner Celgene," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "At the start of 2016, we raised $150 million in gross proceeds from an equity financing which will be used to fund our wholly owned programs, including our compounds for muscle diseases. Our locally acting muscle agent, ACE-083, showed unprecedented results in increasing muscle volume, and we plan to present new Phase 1 data later this year and initiate a Phase 2 trial in FSHD, a form of muscular dystrophy."

Added Dr. Knopf, "In late 2015, we introduced ACE-2494, a systemic muscle therapeutic created from our new IntelliTrap discovery platform. IntelliTrap is a powerful discovery engine, and we expect it to enable us to introduce a novel therapeutic into the clinic every 12 to 18 months."

2015 HIGHLIGHTS AND CURRENT UPDATES

DEVELOPMENT PROGRAMS

Hematology

Luspatercept in myelodysplastic syndromes (MDS)

Luspatercept is a protein therapeutic that increases hemoglobin levels and is being developed to help patients reduce or eliminate their need for red blood cell (RBC) transfusions

Initiated Phase 3 MEDALIST study in MDS with partner Celgene. MEDALIST is a 210-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in subjects with anemia due to very low, low, or intermediate-risk MDS with ring sideroblasts (≥ 15%) who require red blood cell transfusions.

Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 extension study showing that 50% of MDS patients achieved transfusion independence and 69% of patients achieved sustained increases in hemoglobin levels.

Expanded the Phase 2 MDS study with additional cohorts in lower risk MDS patients that are either erythropoietin-stimulating agent (ESA) treatment naïve or ring sideroblast negative.

Received FDA Fast Track Designation.

Luspatercept in beta-thalassemia

Initiated Phase 3 BELIEVE study in beta-thalassemia with partner Celgene. BELIEVE is a 300-patient, global, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept versus placebo in adults who require regular red blood cell transfusions due to beta-thalassemia.

Presented data at ASH (Free ASH Whitepaper) 2015 annual meeting from ongoing Phase 2 studies showing luspatercept reduced transfusion burden, improved health-related quality of life measures, had beneficial effects on liver iron concentration and demonstrated a favorable safety profile.

Received FDA Fast Track Designation.

Muscle Diseases

ACE-083

Protein therapeutic designed to increase muscle mass and strength in the muscles in which it is administered

First-in-human Phase 1 study results showed an unprecedented 14.5% mean increase in muscle volume in the injected rectus femoris muscle of the quadriceps. The data were presented in the Late Breaking Clinical Trials Session of the 8th International Conference on Cachexia, Sarcopenia, and Muscle Wasting in December 2015.

Preclinical data in mice showed that ACE-083 produced significant increases in muscle mass in the injected muscle with no observed effect on either the uninjected contralateral muscle or on whole body mass. Increases in muscle mass were associated with a significant increase in muscle force and power. The data were presented at the 20th International Annual Congress of the World Muscle Society in October 2015.

ACE-2494

Systemic muscle therapeutic designed to increase muscle mass and strength in a range of muscle diseases

Introduced ACE-2494, Acceleron’s first IntelliTrap molecule. Preclinical data in mice presented at the 2015 World Muscle Society Congress showed that after 4 weeks of treatment, ACE-2494 generated substantial dose-dependent mean increases in muscle mass: 41% in rectus femoris, 53% in gastrocnemius, and 87% in pectoralis.
Oncology

Dalantercept in renal cell carcinoma (RCC)

Protein therapeutic that inhibits angiogenesis and is being developed in combination with approved VEGF-based anti-angiogenesis compounds to improve patient outcomes.

Enrollment is ongoing in Part 2 of the Phase 2 DART study, a randomized, double-blind study of dalantercept plus axitinib compared to placebo plus axitinib in patients with advanced renal cell carcinoma.

Results from Part 1 of the DART study with dalantercept plus axitinib demonstrated a median progression free survival of 8.3 months across all dose levels tested versus the historic control of 4.8 months for axitinib alone. The data were presented at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and ASCO (Free ASCO Whitepaper) 2015 Genitourinary Cancers Symposium.

Received FDA Fast Track Designation for dalantercept in combination with axitinib for the treatment of patients with advanced RCC following treatment with one anti-angiogenic agent.

Nephrology

Sotatercept in chronic kidney disease

Protein therapeutic that has effects on fibrosis, vascular calcification, bone mineral density and red blood cell (RBC) levels

Presented preliminary data from ongoing Phase 2a clinical studies of sotatercept in End-Stage Kidney Disease patients, including effects on hemoglobin, vascular calcification, bone mineral density and safety and tolerability at the American Society of Nephrology Kidney Week in October 2015.

Acceleron and Celgene assessing the opportunity for the development of sotatercept in the pre-dialysis chronic kidney disease (CKD) setting.

RESEARCH AND DEVELOPMENT

Introduced the Company’s IntelliTrap platform for discovery of selective and novel compounds targeting the transforming growth factor-beta superfamily of proteins.

Identified ACE-2494 as the first compound to be developed from the platform and is expected to be Acceleron’s fifth internally discovered therapeutic to enter the clinic.

Acceleron plans to have a new internally discovered compound enter the clinic every 12 to 18 months.

CORPORATE UPDATES

Raised $150 million in gross proceeds in an underwritten public offering of common stock in January 2016.

Hosted first Research & Development Day in October 2015. Company executives and a panel of outside experts briefed the investment community on Acceleron’s clinical programs in MDS, beta-thalassemia, cancer and muscular dystrophies.

UPCOMING 2016 PROGRAM MILESTONES AND EVENTS

We anticipate the following milestones and events in 2016:

Hematology

Luspatercept in MDS

Will update long-term treatment results at major medical conferences.

Initial data on ring sideroblast negative and ESA treatment naïve (front-line) patients (YE 2016).
Luspatercept in beta-thalassemia

Update long-term treatment results at major medical conferences.

Muscle Diseases

ACE-083

Present Phase 1 data from two new cohorts (Mid 2016).

Initiate Phase 2 trial in facioscapulohumeral muscular dystrophy (FSHD) patients (H2 2016).
ACE-2494

Submit IND for first-in-human study for ACE-2494 (YE 2016).
Oncology

Dalantercept in RCC

Present preliminary Part 2 DART study results (dalantercept in combination with axitinib) (YE 2016).
Nephrology

Sotatercept in CKD

Provide an update on development strategy (H2 2016).
Financial Results

Cash Position – Cash, cash equivalents and investments were $136.0 million as of December 31, 2015. Net cash used in operating activities in 2015 was $44.2 million. We believe that our existing cash, cash equivalents and investments, including the net proceeds of $140.4 million from our January 2016 offering, will be sufficient to fund our projected operating requirements into the second half of 2019.

Revenue – Collaboration revenue for the year was $18.1 million. This includes license and milestone amortization of $1.2 million and cost sharing reimbursement revenue from our Celgene partnership of $16.9 million related to expenses incurred by the Company in support of our partnered programs.

Costs and expenses – Total costs and expenses for the year were $79.0 million. This includes R&D expenses of $58.4 million and G&A expenses of $20.6 million.

Net Loss – The Company’s net loss for the year ended December 31, 2015 was $63.9 million.

8-K – Current report

On February 25, 2016 Radius Health, Inc. ("Radius" or the "Company") (Nasdaq:RDUS), a science-driven biopharmaceutical company that is committed to developing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, reported its financial results for the fourth quarter and full year ended December 31, 2015, and provided recent corporate highlights (Filing, 8-K, Radius, FEB 25, 2016, View Source [SID:1234509187]). As of December 31, 2015, Radius had $473.3 million in cash, cash equivalents and marketable securities.

"Radius continues to make significant progress in advancing its pipeline, including the submission of our MAA in Europe for our investigational drug abaloparatide-SC for the treatment of women with postmenopausal osteoporosis who are at risk for a fracture, which is under regulatory review. We are on track to submit an NDA in the U.S. at the end of the first quarter of 2016," said Robert Ward, President and Chief Executive Officer of Radius. "We are continuing our productive partnering discussions and anticipate entering into an abaloparatide collaboration prior to a potential first commercial launch. We have continued to make progress across the portfolio with the abaloparatide transdermal patch program and RAD1901 trials in breast cancer and vasomotor symptoms."

Pipeline Updates

Abaloparatide-SC

In November 2015, Radius submitted a marketing authorization application ("MAA") to the European Medicines Agency ("EMA"), which subsequently was validated and is currently undergoing regulatory review. Radius plans to submit a new drug application ("NDA") in the United States at the end of the first quarter of 2016. Subject to regulatory review and a favorable regulatory outcome, Radius anticipates the first commercial sales of abaloparatide-SC will take place in 2016.

Abaloparatide-TD

Radius also is developing abaloparatide-transdermal, which it refers to as abaloparatide-TD, based on 3M’s patented Microstructured Transdermal System technology for potential use as a short wear-time transdermal patch. During 2014, Radius reported progress towards the development of an optimized transdermal patch that may be capable of demonstrating comparability to abaloparatide-SC. In preliminary, nonhuman primate pharmacokinetic studies, Radius achieved a desirable pharmacokinetic profile, with comparable AUC, Cmax, Tmax and T1/2 relative to abaloparatide-SC. Radius believes that these results support continued clinical development of abaloparatide-TD toward future global regulatory submissions as a potential post-approval line extension of the investigational drug abaloparatide-SC. Radius commenced a human replicative clinical evaluation of the optimized abaloparatide-TD patch in December 2015 with the goal of achieving comparability to abaloparatide-SC.

RAD1901

Radius continues to enroll and dose patients in the United States in its Phase 1 multicenter, open-label, two-part, dose-escalation study of RAD1901 in postmenopausal women with advanced estrogen receptor positive and HER2-negative breast cancer. The study is designed to determine the recommended dose for a Phase 2 clinical trial and includes a preliminary evaluation of the potential anti-tumor effect of RAD1901. In December 2015, Radius reported on the progress of this study at the San Antonio Breast Cancer Symposium in San Antonio, TX. In addition, in December 2015, Radius commenced a Phase 1 FES-PET study in patients with metastatic breast cancer in the European Union, which includes the use of FES-PET imaging to assess estrogen receptor occupancy in tumor lesions following RAD1901 treatment.

In January 2016, Radius entered into a worldwide clinical collaboration with Novartis Pharmaceuticals to evaluate the safety and efficacy of combining RAD1901 with Novartis’ investigational agent LEE011 (ribociclib), a cyclin-dependent kinase 4/6 inhibitor, and BYL719 (alpelisib), an investigational phosphoinositide 3-kinase inhibitor.

RAD1901 also is being evaluated at low doses as an estrogen receptor ligand for the potential relief of the frequency and severity of moderate to severe hot flashes in postmenopausal women with vasomotor symptoms. Radius commenced a Phase 2b clinical study of RAD1901 for the potential treatment of postmenopausal vasomotor symptoms in December 2015.

Radius Expects the Following Upcoming Milestones

· Abaloparatide-SC
· Submit an NDA in the United States for abaloparatide-SC at the end of the first quarter of 2016.
· Receive opinion from the Committee for Medicinal Products for Human Use regarding the EMA’s review of the abaloparatide-SC MAA.
· Enter into a collaboration for the potential commercialization of abaloparatide-SC prior to a commercial launch.
· Abaloparatide-TD
· Complete the clinical evaluation of the optimized abaloparatide-TD patch during 2016.
· RAD1901
· Complete the dose-escalation study for RAD1901 in metastatic breast cancer patients by the middle of 2016.
· Initiate the expansion cohorts in breast cancer during 2016.

Radius Expects To Make Presentations at the Following Upcoming Conferences

· Abstract Presentations at the Endocrine Society Annual Meeting, April 1-4, 2016, in Boston, MA. The titles of the presentations are as follows:

"Abaloparatide Significantly Reduces Vertebral and Non-vertebral Fractures and Increases BMD Regardless of Baseline Risk"

"RAD1901 a Novel Estrogen Receptor Ligand with a Unique Pharmacologic Profile for Potential Use in the Treatment of Postmenopausal Vasomotor Symptoms"

· Abstract Presentations at the World Congress of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases, April 14-17, 2016, in Spain. The titles of the presentations are as follows:

"Effects of Abaloparatide on Vertebral, Non-vertebral, Major Osteoporotic and Clinical Fracture Incidence in Postmenopausal Women with Osteoporosis: Results of the Phase 3 Active Trial"

"Eighteen Months of Treatment with Abaloparatide Followed by Six Months of Treatment with Alendronate in Postmenopausal Women with Osteoporosis- Results of the ACTIVExtend Trial"

"Effect of Investigational Treatment Abaloparatide for Prevention of Major Osteoporotic Fracture or any Fracture is Not Altered by Baseline Fracture Probability"

· Abstract presentation at the American Association of Cancer Research Annual Meeting 2016, April 16-20, 2016, in New Orleans, LA.

"RAD1901, an orally available SERD, as an effective combination partner in ER+ breast cancer"

· IMPAKT 2016 Conference, May 12-14, 2016, in Brussels, Belgium.

"RAD1901, a novel oral, selective estrogen receptor degrader (SERD), for the treatment of advanced estrogen receptor (ER)+ breast cancer (BC)"

· Cowen and Company 36th Annual Healthcare Conference, March 7-9, 2016, in Boston, MA.
· Deutsche Bank 41st Annual Healthcare Conference, May 4-5, 2016, in Boston, MA.
· Bank of America Merrill Lynch 2016 Healthcare Conference, May 10-12, 2016, in Las Vegas, NV.

Recent Corporate Highlight

· On December 7, 2015, Radius announced the appointment of Jean-Pierre (JP) Garnier to its Board of Directors, and as Chair of the Compensation Committee. Mr. Garnier is currently Chairman of the Board of Actelion Ltd., and was previously Chief Executive Officer of GlaxoSmithKline plc.

Fourth Quarter 2015 Financial Results

For the three months ended December 31, 2015, Radius reported a net loss of $33.2 million, or $0.77 per share, as compared to a net loss of $18.0 million, or $0.55 per share for the three months ended December 31, 2014. The increase in net loss for the three months ended December 31, 2015 as compared to the three months ended December 31, 2014 was primarily due to an increase in research and development and general and administrative expenses, partially offset by a decrease in interest expense.

Research and development expenses for the three months ended December 31, 2015 were $22.2 million, compared to $11.6 million for the same period in 2014. The increase for the 2015 period as compared to the 2014 period was primarily attributable to an increase in contract service costs associated with the development of RAD1901, consulting costs incurred to support Radius’ MAA and planned NDA submissions for abaloparatide-SC and an increase in compensation expense, including an increase of $0.6 million of non-cash stock-based compensation expense, due to an increase in research and development headcount from December 31, 2014 to December 31, 2015.

General and administrative expenses for the three months ended December 31, 2015 were $11.6 million, compared to $5.6 million for the same period in 2014. The increase for the 2015 period as compared to the 2014 period was primarily attributable to an
increase in legal fees and professional support costs, including the costs associated with growing Radius’ headcount and preparing for the potential commercialization of abaloparatide-SC.

There was no interest expense for the three months ended December 31, 2015, compared to $0.8 million for the same period in 2014. The decrease was a result of the prepayment of all amounts owed under Radius’ loan and security agreement on August 4, 2015.

Full Year 2015 Financial Results

For the twelve months ended December 31, 2015, Radius reported a net loss of $101.5 million, or $2.56 per share, as compared to a net loss of $62.5 million, or $4.04 per share, for the twelve months ended December 31, 2014. The increase in net loss for 2015 was primarily due to an increase in research and development expenses, general and administrative expenses, and loss on retirement of note payable.

Research and development expenses for the twelve months ended December 31, 2015 were $68.3 million, compared to $45.7 million for 2014. The increase for 2015 was primarily attributable to an increase in compensation expense, including an increase of $5.9 million of non-cash stock-based compensation expense, due to an increase in research and development headcount from December 31, 2014 to December 31, 2015, an increase in consulting costs incurred to support Radius’ MAA and planned NDA submissions for abaloparatide-SC and an increase in contract service costs associated with the development of RAD1901. These increases were partially offset by a decrease in the costs associated with the abaloparatide-SC Phase 3 ACTIVE and ACTIVExtend clinical trials.

General and administrative expenses for the twelve months ended December 31, 2015 were $30.8 million, compared to $13.7 million for 2014. The increase for 2015 as compared to 2014 was primarily attributable to an increase in legal fees and professional support costs, including the costs associated with growing Radius’ headcount and preparing for the potential commercialization of abaloparatide-SC, subject to favorable regulatory review. This increase also was driven by an increase in compensation expense, including an increase of $1.8 million of non-cash stock-based compensation expense, due to an increase in general and administrative headcount from December 31, 2014 to December 31, 2015.

For the twelve months ended December 31, 2015, loss on retirement of note payable was $1.6 million, compared to $0.2 million for 2014. The loss on retirement of note payable for 2015 was a result of the prepayment of all amounts owed under Radius’ loan and security agreement on August 4, 2015.

As of December 31, 2015, Radius had $473.3 million in cash, cash equivalents and marketable securities. Based upon Radius’ cash, cash equivalents and marketable securities balance, it believes that, prior to the consideration of revenue from the

potential future sales, subject to favorable regulatory review, of any of its investigational products, it has sufficient capital to fund its development plans, U.S. commercial scale-up and other operational activities into 2018.

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About Abaloparatide

Abaloparatide is an investigational therapy for the potential treatment of women with postmenopausal osteoporosis who are at an increased risk for a fracture. Abaloparatide is a novel synthetic peptide that engages the parathyroid hormone receptor (PTH1 receptor) and was selected for clinical development based on its favorable bone building activity.

Abaloparatide has completed Phase 3 development for potential use as a daily self-administered injection (abaloparatide-SC). In the fourth quarter of 2015, Radius’ Marketing Authorisation Application (MAA) for abaloparatide-SC for the treatment of patients with postmenopausal osteoporosis was validated and is currently undergoing regulatory review by the European Medicines Agency (EMA). Radius plans to submit a New Drug Application (NDA) for abaloparatide-SC to the US Food and Drug Administration (FDA) at the end of the first quarter of 2016.

Radius also is developing abaloparatide-transdermal (abaloparatide-TD) based on 3M’s patented Microstructured Transdermal System technology for potential use as a short wear-time transdermal patch.

About RAD1901

RAD1901 is a selective estrogen receptor down-regulator/degrader (SERD), which at high doses is being evaluated for potential use as an oral non-steroidal treatment for hormone-driven, or hormone-resistant, breast cancer. RAD1901 is currently being investigated for potential use in postmenopausal women with advanced estrogen receptor positive (ER+), HER2-negative breast cancer, the most common form of the disease. Studies completed to date indicate that the compound has the potential for use as a single agent or in combination with other therapies to overcome endocrine resistance in breast cancer.

RAD1901 also is being evaluated in a Phase 2b study at low doses for potential relief of the frequency and severity of moderate to severe hot flashes in postmenopausal women with vasomotor symptoms. Additional information on the clinical trial program of RAD1901 is available on www.clinicaltrials.gov.

RAD140

RAD140 is a non-steroidal selective androgen receptor modulator (SARM) that is currently in preclinical development for potential use in multiple indications including cancer. RAD140 resulted from an internal drug discovery program focused on the androgen receptor pathway, which is highly expressed in many breast cancers. Due to its receptor and tissue selectivity, potent oral activity and long half-life, RAD140 could have clinical potential in the treatment of conditions where androgen modulation may play a role.

Seattle Genetics Initiates Phase 1 Trial of SGN-CD19B for Patients with B-cell Non-Hodgkin Lymphoma

On February 25, 2016 Seattle Genetics, Inc. (Nasdaq: SGEN) reported initiation of a phase 1 clinical trial of SGN-CD19B for relapsed or refractory patients with two subtypes of B-cell non-Hodgkin lymphoma (NHL): diffuse large B-cell lymphoma (DLBCL) and grade 3 follicular lymphoma (Press release, Seattle Genetics, FEB 25, 2016, View Source [SID:1234509210]).

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About 85 percent of NHL is of B-cell lineage, and CD19 is broadly expressed across all subtypes of B-cell malignancies. SGN-CD19B is a novel antibody-drug conjugate (ADC) targeted to CD19 utilizing Seattle Genetics’ newest ADC technology. SGN-CD19B is composed of an anti-CD19 antibody attached to a highly potent cytotoxic DNA-crosslinking agent called a pyrrolobenzodiazepine (PBD) dimer, via a proprietary site-specific conjugation technology to a monoclonal antibody with engineered cysteines (EC-mAb). The trial is designed to assess the safety and antitumor activity of SGN-CD19B.

"B-cell malignancies are the most common type of non-Hodgkin lymphoma, or NHL. In the relapsed or refractory disease setting, B-cell NHL is difficult to treat, and there is an urgent need to identify more effective treatment options for these patients," said Jonathan Drachman, M.D., Chief Medical Officer and Executive Vice President, Research and Development at Seattle Genetics. "With SGN-CD19A and SGN-CD19B, we are evaluating two clinical-stage ADCs directed against CD19 utilizing different ADC technologies with distinct mechanisms of action, which may result in differentiated clinical profiles and utility in NHL. Together with ADCETRIS, these programs are a part of our extensive efforts to improve outcomes for patients with lymphoma."

The study is a phase 1, open-label, multi-center, dose-escalation clinical trial. The primary endpoints are the estimation of the maximum tolerated dose and evaluation of the safety of SGN-CD19B. In addition, the trial will evaluate antitumor activity, pharmacokinetics, objective response rate and progression-free survival. The study is designed to evaluate SGN-CD19B administered every four or six weeks and will enroll up to approximately 100 relapsed or refractory patients at multiple centers in the United States.

Preclinical data presented at the 2015 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated that SGN-CD19B exhibits antitumor activity against a broad panel of CD19-expressing B-cell malignancies, inducing durable tumor regressions in multiple preclinical models of NHL and B-ALL.

With more than 15 years of experience and innovation, Seattle Genetics is the leader in developing ADCs. ADCs are monoclonal antibodies that are designed to selectively deliver cell-killing agents to tumor cells. This approach is intended to spare non-targeted cells and, thus, reduce many of the toxic effects of traditional chemotherapy while enhancing antitumor activity.

For more information about the trial, including enrolling centers, please visit www.clinicaltrials.gov.

About Non-Hodgkin Lymphoma

Lymphoma is a general term for a group of cancers that originate in the lymphatic system. There are two major categories of lymphoma: Hodgkin lymphoma and non-Hodgkin lymphoma (NHL). NHL is further categorized into indolent (low-grade) or aggressive lymphomas, including DLBCL and grade 3 follicular lymphoma. DLBCL is the most common type of NHL. According to the American Cancer Society, more than 72,000 cases of NHL will be diagnosed in the United States during 2016 and more than 20,000 people will die from the disease.

About SGN-CD19B

SGN-CD19B is a novel ADC targeted to CD19 utilizing Seattle Genetics’ newest ADC technology. CD19 is a protein expressed broadly on normal and malignant B-cells. SGN-CD19B is an antibody-drug conjugate (ADC) composed of an anti-CD19 antibody attached to two molecules of a highly potent DNA crosslinking agent called a pyrrolobenzodiazepine (PBD) dimer, using Seattle Genetics’ proprietary technology. PBD dimers are significantly more potent than systemic chemotherapeutic drugs, and site-specific conjugation technology (EC-mAb) allows uniform drug-loading of the cell-killing PBD dimer to the anti-CD19 antibody. The ADC is designed to be stable in the bloodstream and to release its potent DNA binding agent upon internalization into CD19-expressing cells. The ADC technology used in SGN-CD19B is the same as vadastuximab talirine (SGN-CD33A; 33A), an ADC in clinical development for myeloid malignancies.

Peregrine Pharmaceuticals Provides Update on Phase III SUNRISE Trial of Bavituximab

On February 25, 2016 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a biopharmaceutical company focused on developing therapeutics to stimulate the body’s immune system to fight cancer, reported that it is discontinuing the company’s Phase III SUNRISE trial of bavituximab in patients with previously treated locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) (Press release, Peregrine Pharmaceuticals, FEB 25, 2016, View Source [SID:1234509207]).

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The decision to stop the trial was based on the recommendation of the study’s Independent Data Monitoring Committee (IDMC) following a pre-specified interim analysis performed after 33% of targeted overall events (patient deaths) in the study were reached. Results of the analysis demonstrated that the bavituximab plus docetaxel group did not show a sufficient improvement in overall survival as compared to the docetaxel group to warrant continuation of the study. The interim analysis showed that the bavituximab combination group is performing as expected according to the original trial assumptions in terms of overall survival, while the docetaxel group is dramatically outperforming overall survival expectations based on the original trial assumptions and as compared to recently published studies.

"Let me start by taking this opportunity to thank all of the patients, their families, and the physicians who participated in the SUNRISE trial. While we are deeply disappointed by this early outcome from the SUNRISE trial, we plan to take a deliberate and detailed approach in reviewing and verifying all available data from the trial in order to understand what subgroups or other patient characteristics may have impacted the performance of the study. While we perform this analysis, we plan to put our other chemotherapy combination studies on hold until we have a clear understanding of the SUNRISE study results," said Steven W. King, president and chief executive officer of Peregrine. "While this is an unexpected and disappointing setback for the bavituximab chemotherapy combination clinical program, we have not seen anything in this trial result that diminishes our enthusiasm for advancing our immuno-oncology (I-O) combination trials. The I-O combination studies are based on different mechanistic synergies that are clearly separate from the chemotherapy combination being evaluated in the SUNRISE study. In addition, it is important to note that in no way do these results have any impact on our contract manufacturing business conducted through our wholly owned subsidiary, Avid Bioservices. This business has shown consistent revenue growth and has been instrumental in maintaining a strong cash position and our plan is to continue growing this business."

As of February 1, 2016, Avid Bioservices had a revenue backlog in excess of $58 million under committed contracts from existing clients. In addition, Peregrine had $67.5 million in cash and equivalents as of January 31, 2016.

About Bavituximab: A Targeted Investigational Immunotherapy
Bavituximab is an investigational chimeric monoclonal antibody that targets phosphatidylserine (PS). Signals from PS inhibit the ability of immune cells to recognize and fight tumors. Bavituximab is believed to override PS mediated immunosuppressive signaling by blocking the engagement of PS with its receptors as well as by sending an alternate immune activating signal. PS targeting antibodies have been shown to shift the functions of immune cells in tumors, resulting in multiple signs of immune activation and robust anti-tumor immune responses.

Foundation Medicine Announces First-Ever Public Release of Broad Pediatric Data From FoundationCORE™ to Accelerate the Development of Precision Medicine Strategies for Pediatric Cancers

On February 25, 2016 Foundation Medicine, Inc. (NASDAQ:FMI) reported that the company will release to the public molecular profiles of pediatric cancers currently maintained as part of FoundationCore, the company’s information knowledgebase (Press release, Foundation Medicine, FEB 25, 2016, View Source [SID:1234509199]). FoundationCORE is one of the largest and most robust collections of common and rare clinical tumor profiles globally. The release of this data is part of the company’s ongoing effort to stimulate and accelerate research and development of much needed precision oncology therapies to treat all cancers, including pediatric cancers. The announcement of the data release, which is the first-of-its-kind from FoundationCORE, was announced today at the Precision Medicine Initiative Summit held at the White House.

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"One of the biggest impediments to progress in the field of childhood cancer is the paucity of genomic data available from patients at the time of disease relapse. These data are critical to defining comprehensive strategies to precisely treat relapsed disease, and more importantly, to prevent disease recurrence in the first place," stated John M. Maris, M.D., Giulio D’Angio Endowed Professor of Pediatric Oncology at Children’s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania. "The data being made available from Foundation Medicine will serve as a tremendous resource for the cancer research community and should catalyze the process of bringing effective new therapies to children with cancer."

FoundationCORE is a molecular information knowledgebase that contains genomic information and insights from more than 68,000 adult and pediatric patients whose tumors were profiled by Foundation Medicine. FoundationCORE is continuously evolving over time, informed by every new tumor sequenced with the company’s clinical comprehensive genomic profiling assays, FoundationOne and FoundationOne Heme. Foundation Medicine is developing a web-based HIPPA-compliant portal for public access to the de-identified, molecular-level pediatric tumor information contained in FoundationCORE. The portal is expected to be accessible for public access by March 31st.

"The proposed release of data by Foundation Medicine is a major advance towards achieving our nation’s precision medicine initiatives, particularly in an area of significant unmet need for children," said Nancy Goodman, executive director, Kids v Cancer. "This type of out-of-the-box initiative gives great hope to the families and patients who need it most. We’re looking forward to the renaissance in targeted and immunotherapies for children that may come as a result of this data."

Pediatric cancers are a rare and diverse collection of diseases. While childhood and adolescent cancer is rare, it remains the leading cause of death by disease among children. The American Cancer Society predicted that about 10,380 children in the United States would be diagnosed with cancer in 2015 and childhood cancer rates have been rising slightly for the past few decades.1 The data set being released from FoundationCORE offers robust opportunities for researchers, and can be used to generate hypotheses, validate rare findings and investigate the genomic landscape of rare tumors for which only small studies exist. Data from thousands of tumors will be publicly available without cost to the research community for exploration and incorporation into future experiments that will ultimately improve the treatment and prognosis for children with cancer.

"There are few things in life as devastating as when a child is diagnosed with cancer. It’s critically important to the achievement of our corporate mission that the robust genomics information we have amassed is freely and easily accessible to researchers and utilized as an important tool to address the significant unmet medical need in pediatric cancers," said Michael Pellini, M.D., chief executive officer, Foundation Medicine. "We are extremely proud to be in a position that enables us to foster global collaborations among researchers by providing anywhere, anytime access to rich molecular information in an easy to use format. In doing so, we hope to accelerate the pace of innovation in pediatric cancer with the intent of bringing new, potentially lifesaving therapeutics to children."