Welcome to the latest edition of  BioLines Weekender,  highlighting the innovation taking place in our great State, including progress in clinical trials, drug development, corporate partnering and research collaborations.  

This week we celebrated the signing of the  21st Century Cures Act into law by President Barack Obama. Our 8-year old Patient advocate  "Magic" Max Schill was on hand with the President and Vice President for the signing. 

The 21st Century Cures Act is a triumph for innovation and for bipartisanship in Congress. Most importantly, it is a victory for Patients everywhere who will have access to clinical trials and to treatments and cures that we can only imagine today. Given the prominence and prevalence of the biopharmaceutical industry in New Jersey, our State is particularly well situated to benefit from this legislation. This accomplishment is the result of years of work by many people, but it would not be a reality without the efforts of New Jersey's own Congressmen Frank Pallone and Leonard Lance, who contributed their leadership and their determination to this effort. We thank the entire New Jersey Delegation for their support for 21st Century Cures. 

As 2016 comes to an end, we thank our Members who spend their days focused on developing new therapies and cures for Patients around the world. Please join us on February 2, 2017 at
BioNJ's 24th Annual Gateway Gala & Innovation Celebration as we celebrate the medical innovation coming from New Jersey's vibrant life sciences ecosystem -- bringing hope to Patients everywhere.

Because Patients Can't Wait SM ,
The BioNJ Team
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Thank you to all who participated in BioNJ's three last events of 2016...   
From a moving fireside chat with former U.S. Country Head and President of Novartis Christi Shaw to the excitement of attendees waiting to meet one of the world's leading positive experts on grit  Caroline Adams Miller , all left BioNJ's 2nd Annual Inspiring Women in STEM Conference  on December 2 with actionable ideas and valuable insight on how to advance their "Path to Power".   
With the prominence of pricing and medical innovation in the news, BioNJ's Beyond Value Frameworks Workshop on December 8 attracted Patient advocates, industry leaders and legislators for an "outstanding discussion on key issues and impactful ideas to help advance improving the 'value' of value frameworks." (Quote by Gary Binder, Helsinn Therapeutics) Click here for a list of resources on the Value of Medical Innovation. 

Breakfast with BioNJ on December 13 at Princeton University featured a fireside chat with two two successful industry leaders: Scott Jackson, former CEO of Celator Pharmaceuticals recently acquired by Jazz Pharmaceuticals, and Jeff Hatfield, former CEO of Vitae Pharma acquired by Allergan. Attendees heard their respective paths to success, including insights into the strategic decisions they made and the challenges they faced along the way to their respective successful exits. Two inspiring success stories!
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BioNJ is launching two new website tools listing BioNJ Members that support New Jersey's life sciences research & development companies. The Real Estate Corner and Clinical Research Center will each include respective supplier organizations and links to their company websites. If you are a BioNJ Member with offerings aligned with either specialty, and would like to be included in our new resource centers, please submit your organization name and brief description of your service offering. Click here to participate. 
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BioNJ Calendar

BioNJ & Friends Wine Reception 
During JPMorgan Conf.
The Marker, San Francisco, CA

Jan. 8, 2016
BioNJ's 24th Annual Gateway Gala & 
Innovation Celebration
The Hilton, East Brunswick, NJ

Feb. 2, 2017

Putting Patients First: The Value of Medical Innovation
Brought to You by Celgene Corporation



In reflecting on the three-year journey of #CuresNow becoming law, there was one young advocate who was there every step of the way -- our friend Max. The tiniest of advocates first joined the #Path2Cures in the late spring of 2015 at the tender age of six. Max has been a critical voice and played an important role in helping us get this game-changing innovation package across the finish line. His story is a familiar one. Max has a RASopathy called Noonan syndrome, which is a rare genetic condition that prevents normal development in various parts of the body. Max has helped lead the charge for #CuresNow -- visiting 83 Senate offices over the course of two days and taking over the House GOP blog to explain the importance of 21st Century Cures.

An Op-Ed Featured in Forbes by Brent Saunders, Chairman and Chief Executive, Allergan

With the election of Donald Trump as our next President, is the focus off drug prices? Will things go back to business as usual? Will changes to the Affordable Care Act, the economy, jobs and immigration consume all of the political oxygen in the room, leaving no room for anything else? My answer to those questions: No. In September, Allergan drafted our Social Contract with Patients. It outlines four principles that guide us to do what is right for physicians and patients. One principle is limiting price increases and ensuring access to life-enhancing treatments.



BioNJ in the News


Article Featured in  NJSpotlight

A new federal program pledges to invest more than $6 billion to promote cutting-edge healthcare initiatives, including research on cancer and brain disorders, and improve access to treatments for mental health and substance use disorders. And New Jersey, with its established biopharmaceutical industry, is well positioned to benefit from the new law, observers suggested.

Debbie Hart, President and CEO of BioNJ, an alliance of hundreds of life-sciences companies, called it a "groundbreaking" and "revolutionary" law that will help patients get therapies and cures more quickly, while reducing healthcare costs overall. In addition to new funding, the law calls for reforms to the drug approval process, greater collaboration among diverse researchers and more racial diversity and patient engagement in federally funded health studies." 


Article by BioNJ President and CEO Debbie Hart in New Jersey Business Magazine

While cancer remains the second most common cause of death in the U.S., as cited by the Centers for Disease Control and Prevention, major investments in cancer research have led to dramatic improvements of our understanding and treatment of cancer.

More people are surviving cancer than ever before, and most of this progress comes from the discovery and development of highly tailored, targeted therapies that provide better control of cancer with fewer side effects. And the bulk of this oncology R&D is occurring right here in New Jersey, with the vast majority of clinical trials in New Jersey being in Oncology, with 58% in New Jersey vs. 41% in the rest of the country, as reported in BioNJ's Economic Impact of Clinical Trials Activity in New Jersey, released in partnership with Rutgers Business School and Rutgers Bloustein School of Planning and Public Policy.


Article Featured in NJBIZ

BioNJ, the State's pre-eminent advocate organization for the biotech and life sciences industries, hosted its second annual "Inspiring Women in STEM" conference earlier this month at the Sanofi U.S. headquarters in Bridgewater.

"Today is about bringing together professionals of all levels for an empowering and memorable program designed to encourage and support women involved in all aspects of (science, technology, engineering and mathematics) -- from biotech, pharmaceutical, chemistry, medical device and technology companies, as well as universities, medical and research institutions," Debbie Hart, CEO and President of BioNJ, said.



BioNJ is pleased to shine The Spotlight on one of our Innovation Members each month featuring their progress and work on behalf of Patients and their impact on the New Jersey life sciences ecosystem. This month's Emerging Company in The Spotlight is TAXIS Pharmaceuticals.

TAXIS Pharmaceuticals  is a privately held biopharmaceutical company headquartered in Monmouth Junction, NJ. Dedicated to the antibiotic resistance crisis, its mission is to discover drugs that will change the treatment landscape. Currently, TAXIS' lead product is TXA709, a second-generation prodrug that is in the last stage of clinical development.

NJ Company News


Ever proud of our ecosystem, we are pleased to share our Company News. Be sure to click on the article titles below to read the full release.


BioNJ Member Pfizer Inc., with offices in Madison, announced today that the U.S. Food and Drug Administration (FDA) approved EUCRISATM (crisaborole) ointment 2%, a novel non-steroidal topical phosphodieterase-4 (PDE-4) inhibitor for the treatment of mild to moderate atopic dermatitis (AD) in patients two years of age and older. AD, often called eczema, is a chronic condition impacting nearly 18 million children and adults in the United States. Approximately 90 percent of people living with AD have the mild to moderate form of the condition.



Princeton-based BioNJ Member  Genmab A/S announced that the European Commission (EC) has granted a marketing authorization for the use of ofatumumab (Arzerra®) in combination with fludarabine and cyclophosphamide (FC) for the treatment of adult patients with relapsed chronic lymphocytic leukemia (CLL) in the European Union. The variation to the Marketing Authorization for this indication was submitted to the European Medicines Agency (EMA) in March 2016 by Novartis under the ofatumumab collaboration between Novartis and Genmab.  



Hackensack-based BioNJ Member BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell technologies for neurodegenerative diseases, announced that new data from the company's Phase 2 study of NurOwn® in ALS were presented by lead investigator Dr. James Berry at the 27th International Symposium.  In the Phase 2 trial, levels of neurotrophic factors and inflammatory markers were measured in cerebral-spinal fluid (CSF) samples collected from patients. In the samples of those patients treated with NurOwn, a statistically significant increase in levels of neurotrophic factors VEGF, HGF and LIF was observed from pre- to post-transplantation. 



Iselin-based BioNJ Member Helsinn, an oncology company focused on the clinical development of novel therapies for cancer, announced final results from a Phase 2 clinical study of the investigational drug candidate Pracinostat and azacitidine in elderly patients with acute myeloid leukemia (AML) who were not eligible for induction chemotherapy, including evidence of prolongation of survival in the overall population and across a number of patient subgroups.



Hampton-based BioNJ Member Celldex Therapeutics, Inc. presented data on new product candidate CDX-1140, a fully human antibody targeted to CD40 that has demonstrated potent agonist and anti-lymphoma activity. Found on antigen presenting cells, such as dendritic cells, macrophages and B cells, CD40 is a key activator of the immune response and is expressed on many cancer cells, in particular B cell lymphomas. CDX-1140 is expected to be ready to enter clinical studies in patients with advanced cancers, including lymphoma, in 2017.



Cranbury-based BioNJ Member Cornerstone Pharmaceuticals, Inc., a privately held, clinical-stage, oncology-focused pharmaceutical company, presented data from two Phase I trials evaluating its lead compound, CPI-613. The two data sets show encouraging efficacy and safety results in patients with acute myeloid leukemia (AML) and T-cell non-Hodgkin's lymphoma (T-cell NHL), respectively, supporting the clinical rationale for combining CPI-613 with standard chemotherapy in two hematologic malignancies that have historically been difficult to treat. 



Parsippany-based BioNJ Member Ferring Pharmaceuticals and MetaboGen AB announced a multi-year research collaboration aimed at developing a microbiome-based product to prevent and treat intrahepatic cholestasis of pregnancy (ICP), a potentially serious liver disorder which affects around 400,000 pregnant women annually.  The genes and gene products produced by the trillions of microorganisms that live in the human body are collectively known as the microbiome. Current research into the microbiome is changing perspectives on health and disease, but there are still many factors that are not yet properly understood.



Summit-based BioNJ Member  Celgene Corporation  announced interim results from the ABOUND clinical trial program evaluating the use of ABRAXANE® (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) in patients with advanced non-small cell lung cancer (NSCLC). Interim data being presented from the ABOUND trials during the IASLC 17th World Conference on Lung Cancer (WCLC) reinforces the benefit of ABRAXANE/carboplatin doublet therapy in first-line NSCLC. 



Summit-based BioNJ Member  Celgene Corporation  announced results from two studies (Abstracts #1143, LBA-1) evaluating the investigational use of REVLIMID® (lenalidomide) maintenance therapy in patients with multiple myeloma.  The Myeloma XI trial is a  UK -based, large, randomized, open-label Phase III study that included a comparison of investigational lenalidomide maintenance treatment versus no maintenance for patients with newly diagnosed multiple myeloma. In the Phase III BMT CTN 0702 StaMINA trial, TE patients were randomized following transplant between three arms to receive either 4 cycles of lenalidomide-bortezomib-dexamethasone (RVD) consolidation (ACM) (n=254), tandem melphalan 200mg/m2 autologous stem cell transplant (TAM) consolidation (n=247), or no consolidation (AM) (n=257). 



Summit-based BioNJ Member  Celgene Corporation  announced that the results of its randomized phase II tnAcity trial of ABRAXANE® for injectable suspension (paclitaxel protein-bound particles for injectable suspension) (albumin-bound) found that an investigational weekly combination regimen of ABRAXANE + carboplatin had significantly longer progression-free survival (PFS) (7.4 months) compared to weekly regimens of either ABRAXANE + gemcitabine (5.4 months) or of carboplatin + gemcitabine (6.0 months) as first-line treatment of patients with metastatic triple-negative breast cancer (mTNBC).



Princeton-based BioNJ Member Otsuka Pharmaceutical Development & Commercialization, Inc. and Lundbeck presented data on aripiprazole for extended-release injectable suspension for intramuscular use (hereinafter ABILIFY MAINTENA®) in the maintenance treatment of bipolar I disorder (BP-I).  Study data demonstrate that ABILIFY MAINTENA delayed time to recurrence of mood episodes in adult patients . Treatment was compared with placebo and demonstrated a safety profile consistent with that observed with ABILIFY MAINTENA in the treatment of patients with schizophrenia.



Bedminster-based BioNJ Member  Aerie Pharmaceuticals, Inc. , a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies, announced the completion of patient enrollment in the company's second Phase 3 registration trial ("Mercury 2") of Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, a novel once-daily eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. 



Princeton-based BioNJ Member  Advaxis, Inc., a clinical stage biotechnology company developing cancer immunotherapies, announced that preclinical data on the potential use of detoxified Listeriolysin O (dtLLO) as an effective adjuvant for the development of infectious disease vaccines was presented at the 11th annual ImVacS-Immunization and Vaccine Summit.  Dr. Hector Vivanco Cid, of the Institute of Medical Biological Research at the University of Veracruz in Mexico, presented data that supports the use of dtLLO as a potential immunologic adjuvant or carrier for vaccinations. 



Edison-based BioNJ Member ContraVir Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of targeted antiviral therapies, provided new insights into the mechanism of action of its hepatitis B (HBV)-optimized cyclophilin inhibitor CRV431, showing that it effectively blocks a critical interaction between HBV X protein (HBx) and host cyclophilin A, with nanomolar potency. CRV431 is believed to be the first antiviral drug with the potential to selectively block the HBx pathway, which is known to play several significant roles in HBV infection including protecting infected liver cells from immune destruction, and promoting the development of liver cancer.



Cranbury-based BioNJ Member  Amicus Therapeutics , a global biotechnology company at the forefront of rare and orphan diseases, announced positive preliminary data from a global Phase 1/2 study ( ATB200-02) to investigate ATB200/AT2221. ATB200/AT2221 is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone.



Princeton-based BioNJ Member Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced the long-term follow-up data from its Phase 2 clinical trial with SGX942 (dusquetide), a first-in-class Innate Defense Regulator (IDR), in the treatment of oral mucositis (OM) in head and neck cancer patients undergoing chemoradiation therapy (CRT).


Princeton-based BioNJ Member Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced SGX942 (dusquetide) has been granted Promising Innovative Medicine (PIM) designation in the United Kingdom (UK) by the Medicines and Healthcare Products Regulatory Agency (MHRA) for the treatment of severe oral mucositis in head and neck cancer patients receiving chemoradiation therapy.


Bedminster-based BioNJ Member  Matinas BioPharma Holdings, Inc., a clinical-stage biopharmaceutical company focused on identifying and developing safe and effective broad spectrum therapeutics for the treatment of serious and life-threatening infections, announced that it has commenced dosing in the Phase 1 clinical study of its lead antibiotic product candidate MAT2501, under development for the treatment of nontuberculous mycobacterium infections (NTM) as an initial indication.



Bedminster-based BioNJ Member  Matinas BioPharma Holdings, Inc., a clinical-stage biopharmaceutical company focused on identifying and developing safe and effective broad spectrum therapeutics for the treatment of serious and life-threatening infections, announced that it has received a research contract award from Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, to study its lead antibiotic product candidate MAT2501, for the treatment of pre-clinical nontuberculous mycobacterium infection (NTM) in models of cystic fibrosis (CF). 



Berkeley Heights-based BioNJ Member  Edge Therapeutics, Inc. , a clinical-stage biotechnology company developing novel hospital-based therapies in the management of acute, life-threatening conditions, announced that the results from its completed North American Phase 1/2 clinical study of EG-1962 are now available online through Stroke, a leading peer-reviewed journal from the American Heart Association.



Parsippany-based BioNJ Member Radius Health, Inc., a science-driven biopharmaceutical company focused on developing innovative therapeutics in the areas of osteoporosis, oncology and endocrine diseases, announced data from two ongoing Phase 1 studies of RAD1901, an oral selective estrogen receptor degrader (SERD), in patients with estrogen receptor positive (ER+) breast cancer.  As of the cut-off date of October 7, 2016, 20 patients have been treated in the RAD1901 Phase IB safety expansion cohort at the 400 mg dose. 



South Plainfield-based BioNJ Member  PTC Therapeutics Inc.  announced that the company received France's 2016 Prix Galien for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in the Medicines "Rare Disease" category. The award recognizes the scientific innovation represented by Translarna's ability to target the underlying cause of nmDMD and the impact the drug is having on patients. The award was presented at the annual gala and ceremony in Paris, France .


Rutherford-based BioNJ Member Immunomedics, Inc.  announced a novel immuno-oncology agent, (E1)-3s, that can direct a patient's T cells, a type of white blood cells, to kill cancer cells with the cancer marker, the Trop-2 antigen, which can be enhanced by combining with an immune checkpoint inhibitor. Trop-2 is a receptor found on many solid cancers and the target of the company's lead antibodydrug conjugate, sacituzumab govitecan (IMMU-132). In a single-arm Phase 2 study, IMMU-132 has produced promising results in patients with diverse late-stage metastatic disease, including TNBC, small-cell and non-small-cell lung, and urothelial cancers.


Rutherford-based BioNJ Member Immunomedics, Inc.  announced that  IMMU - 140 ,  the company ' s latest investigational product from its award - winning antibody - drug conjugate (ADC) program,  demonstrated a significant antitumor effect  without any undue toxicity  in four  hematopoietic tumors  in preclinical studies . IMMU-140 comprises the humanized antibody, IMMU-114, conjugated to the moderately-toxic drug, SN-38, through the company's proprietary linker, CL2A, the same toxic payload and antibody-drug linker that are in sacituzumab govitecan (IMMU-132). 



Rutherford-based BioNJ Member Cancer Genetics, Inc., an emerging leader in enabling precision medicine for oncology through the use of molecular markers and information, announced that it is on Merck's list of national reference laboratories offering the PD-L1 22C3 pharmDx immunohistochemistry (IHC) assay, the first U.S. Food and Drug Administration (FDA)-approved companion diagnostic test for pembrolizumab, KEYTRUDA®, for PD-L1 testing in non-small cell lung cancer (NSCLC). 



Rutherford-based BioNJ Member Cancer Genetics, Inc. announced a collaboration with BeiGene, Ltd., a global, clinical-stage, research-based biopharmaceutical company developing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer. The two companies will work together on BeiGene's immune marker and genomics initiatives to accelerate studies of tumor microenvironment in cancer patients, which will aid in drug target identification and biomarker discovery.



Berkeley Heights-based BioNJ Member Cyclacel Pharmaceuticals, Inc., a biopharmaceutical company developing oral therapies that target the various phases of cell cycle control for the treatment of cancer and other serious disorders, announced the presentation of preclinical data demonstrating the therapeutic potential of the company's novel polo-like kinase (PLK) 1 inhibitor, CYC140, as a targeted anti-cancer agent. The data demonstrates that CYC140 is a selective PLK1 inhibitor which preferentially induces growth inhibition and cell death in malignant versus non-malignant cells. 



Princeton-based BioNJ Member Photocure ASA (PHO-NO) announced new data from a prospective multicenter real world study that included 338 patients, 415 BLC procedures and 1060 lesions from nine study sites in the United States. BLCC as an adjunct to white light cystoscopy (WLC) resulted in the detection of an additional 151 lesions and 45 patients uniquely with the addition of blue light. This resulted in upgrading or upstaging of 52 (15%) of the patients. Correct staging and grading impacts the management of bladder cancer patients and may have an impact on patient outcomes.



Princeton-based BioNJ Member Bristol-Myers Squibb Company announced important changes to its U.S. geographic footprint that will strengthen capabilities of the company's R&D hubs of world-class science and innovation in Central New Jersey, the San Francisco Bay Area and Cambridge, Massachusetts while further integrating biologics development with clinical manufacturing at key sites to foster greater speed and collaboration.  Bristol-Myers Squibb will make investments in the construction of a new R&D building at the company's Lawrenceville, New Jersey campus that will co-locate lab-based Discovery and Translational Medicine activities and construction at its New Brunswick, New Jersey facility to support biologics development. 



Princeton-based BioNJ Member Bristol-Myers Squibb Company announced updated results for Opdivo monotherapy (3 mg/kg every two weeks [n=98]) and in combination with Yervoy (Opdivo 1 mg/kg plus Yervoy 3 mg/kg every three weeks [n=61]) in previously treated small cell lung cancer (SCLC) patients, a cohort of the Phase 1/2 open-label CheckMate -032 trial. The confirmed objective response rate (primary objective) was 25% (95% CI: 15, 37) in patients who received Opdivo plus Yervoy and was 11% (95% CI: 6, 19) with Opdivo alone with additional follow-up. Response was observed regardless of platinum sensitivity or prior lines of therapy.


Raritan-based BioNJ Member Janssen Research & Development, LLC announced results from a Phase 2 study demonstrating a 48% overall response rate (ORR) as assessed by an Independent Review Committee (IRC) with single-agent ibrutinib (IMBRUVICA®) in patients with relapsed/refractory (r/r) marginal zone lymphoma (MZL). At a median follow-up of 19.4 months in 60 patients, 3% (n=2) treated with ibrutinib achieved complete responses (CR) and 45% (n=27) achieved partial responses (PR). Overall, 78% of patients experienced tumor reduction. 



Raritan-based BioNJ Member Janssen Research & Development, LLC announced Phase 2 ibrutinib (IMBRUVICA®) data showing encouraging results in patients with chronic graft-versus-host-disease (cGVHD), a life-threatening consequence of stem cell or bone marrow transplant, who failed prior systemic therapy. Study findings indicate ibrutinib demonstrated clinically meaningful and durable responses and reduced symptom severity, with an overall response rate (ORR) of 67% in patients with cGVHD.



Kenilworth-based BioNJ Member Merck and Eisai Inc. announced new interim data investigating Merck's anti-PD-1 therapy, KEYTRUDA® (pembrolizumab), in combination with Eisai's microtubule dynamics inhibitor, HALAVEN® (eribulin), in patients with metastatic triple-negative breast cancer (TNBC). Findings were based on interim data from 39 evaluable patients and showed an overall response rate (ORR) of 33.3% (n=13/39; 95% CI, 19.5-48.1), with one complete response and 12 partial responses (Abstract #: P5-15-02).      



Kenilworth-based BioNJ Member Merck announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for KEYTRUDA® (pembrolizumab), the company's anti-PD-1 therapy, for the treatment of patients with refractory classical Hodgkin lymphoma (cHL) or for patients who have relapsed after three or more prior lines of therapy. The FDA granted Priority Review with a PDUFA, or target action, date of March 15, 2017. 



Plainsboro-based BioNJ Member Novo Nordisk has announced the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for semaglutide, for the treatment of adults with type 2 diabetes.  Semaglutide is a once-weekly administered analog of human glucagon-like peptide-1 (GLP-1) that stimulates insulin and suppresses glucagon secretion in a glucose-dependent manner. The submission is based on results from the SUSTAIN clinical trial program, which included over 8,000 patients with type 2 diabetes and was comprised of eight Phase 3a studies. 



Plainsboro-based BioNJ Member Novo Nordisk announced new phase 3b trial (DUAL VII) results with Xultophy ® (IDegLira). Xultophy ® is a once-daily, single injection fixed combination of a long-acting basal insulin (insulin degludec) and a glucagon-like peptide-1 (GLP-1) receptor agonist (liraglutide). The open-label trial investigated the efficacy and safety of Xultophy ® compared with insulin glargine U100 in combination with insulin aspart at all main meals, after 26 weeks of treatment in 506 adults with type 2 diabetes. 



Sandoz, a division of East Hanover-based BioNJ Member Novartis, and the pioneer and global leader in biosimilars, announced data from the ASSIST-FL trial. The confirmatory safety and efficacy study shows GP2013 met its primary endpoint of overall response rate (ORR), demonstrating equivalence with the reference product, MabThera®*, in 629 patients. The combination treatment phase of the ASSIST-FL study -- the first of a three-phase protocol -- confirms that, for patients with previously untreated advanced follicular lymphoma, the ORR of GP2013 (87.1%) and the reference product (87.5%) were equivalent.


East Hanover-based BioNJ Member Novartis announced new data from the Tasigna® (nilotinib) ENESTop Treatment-free Remission (TFR) study, which demonstrate that TFR rates are consistent among Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) patients who switched from Glivec® (imatinib) due to intolerance, resistance or physician preference. ENESTop evaluated stopping Tasigna treatment in eligible Ph+ CML adults with chronic phase disease after they achieved and sustained deep molecular response (MR) for at least one year with Tasigna but had not achieved and sustained this response previously with Glivec.



East Hanover-based BioNJ Member Novartis announced results from its Phase III open-label, randomized, active-controlled, multi-center ASCEND-4 study, which found that patients with anaplastic lymphoma kinase-positive (ALK+) advanced non-small cell lung cancer (NSCLC) treated with first-line Zykadia® (ceritinib) had a median progression-free survival (PFS) of 16.6 months (95% confidence interval [CI]: 12.6, 27.2), compared to 8.1 months (95% CI: 5.8, 11.1) in patients treated with standard first-line chemotherapy with maintenance. 



East Hanover-based BioNJ Member Novartis announced that the European Commission (EC) has granted an additional indication for Lucentis® (ranibizumab) to treat patients with visual impairment due to choroidal neovascularization (CNV) associated with causes other than neovascular age-related macular degeneration (nAMD), or secondary to pathologic myopia (PM). With this approval, Lucentis is the first retinal treatment approved for these conditions, addressing an important unmet medical need.



East Hanover-based BioNJ Member Novartis announced positive results from the first large-scale study exploring the effects of directly switching symptomatic, non-frequently exacerbating patients with moderate COPD from their current treatments, including steroid-containing combinations and long-acting bronchodilators, to the dual bronchodilator Ultibro® Breezhaler® (indacaterol/glycopyrronium) 110/50 mcg.



East Hanover-based BioNJ Member Novartis announced additional analyses from the Phase III MONALEESA-2 study that show LEE011 (ribociclib) plus letrozole significantly prolonged progression-free survival (PFS) across pre-planned patient subgroups with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer, including post-menopausal women diagnosed de novo, those with visceral liver and lung metastases, and those with bone-only disease.



Findings from East Hanover-based BioNJ Member Novartis' clinical trial (ELIANA) evaluating efficacy and safety of CTL019, an investigational chimeric antigen receptor T cell (CAR T) therapy, in relapsed/refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) found that 82% (41 of 50) of infused patients achieved complete remission or complete remission with incomplete blood count recovery at three months post CTL019 infusion. 



East Hanover -based BioNJ Member Novartis announced that results from the Phase II SUSTAIN study showed that SEG101 (crizanlizumab, formerly SelG1), an anti-P-selectin antibody, reduced the median annual rate of sickle cell-related pain crises (SCPC) by 45.3% compared to placebo (1.63 vs 2.98, p=0.010) in patients with or without hydroxyurea therapy.  In the SUSTAIN study, patients were assigned to high-dose (5.0 mg/kg), low-dose (2.5 mg/kg) and placebo arms. 



The U.S. Food and Drug Administration (FDA) approved Synjardy® XR (empagliflozin and metformin hydrochloride extended-release) tablets for adults with type 2 diabetes. When used along with diet and exercise, Synjardy® XR is indicated to improve blood sugar in adults with type 2 diabetes when both empagliflozin and metformin can be taken. It is marketed by  Bridgewater-based BioNJ Member  Eli Lilly and Company and Boehringer Ingelheim .



Bridgewater-based BioNJ Member Eli Lilly and Company announced that the neoMONARCH study of abemaciclib, a cyclin-dependent kinase (CDK) 4 and CDK 6 inhibitor, met its primary endpoint of reducing expression of Ki67, a biomarker of cell proliferation, after two weeks of treatment. Final data from the Phase 2 trial evaluated abemaciclib, both alone or in combination with the non-steroidal aromatase-inhibitor anastrozole, in postmenopausal women with previously untreated early stage hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer.



The U.S. Food and Drug Administration (FDA) approved a new indication for Jardiance® (empagliflozin) tablets to reduce the risk of cardiovascular death in adults with type 2 diabetes and established cardiovascular disease. JARDIANCE, marketed by  Bridgewater-based BioNJ Member  Eli Lilly & Company and  Boehringer Ingelheim,  is the first type 2 diabetes treatment approved with this additional indication and the only oral type 2 diabetes medicine shown in a clinical trial to provide a life-saving cardiovascular benefit. 



Bridgewater-based BioNJ Member Eli Lilly and Company and AstraZeneca announced a worldwide agreement to co-develop MEDI1814, an antibody selective for amyloid-beta 42 (Aβ42), which is currently in Phase 1 trials as a potential disease-modifying treatment for Alzheimer's disease (AD). This agreement builds on the existing collaboration related to AZD3293, a BACE inhibitor in two pivotal Phase 3 trials.



Cranbury-based BioNJ Member Palatin Technologies, Inc., whose primary project, Bremelanotide, treats female sexual dysfunction, said it has completed phase 3 trials and has begun to seek commercialization options, according to Chief Operating Officer Stephen Wills.  "We're a small biotech company; we just finished our phase 3 trials and released our data in early November," he said. "Post that data, we have process where we engaged an outside bank and they are assisting us with running the process with potential collaboration partners."



Cranbury-based BioNJ Member Palatin Technologies, Inc. announced that it has closed on a previously disclosed underwritten public offering of units with gross proceeds of $16,500,000. "This financing provides us with approximately  $15.4 million  in net proceeds, allowing us to continue advancing bremelanotide for hypoactive sexual desire disorder," said  Stephen T. Wills , Executive Vice President and Chief Financial Officer of Palatin Technologies. "We look forward to submitting a new drug application on bremelanotide in the second half of next year."



Roseland-based  Paratek Pharmaceuticals, Inc. , a biopharmaceutical company focused on the development and commercialization of innovative therapies based upon tetracycline chemistry, announced that it projects its ongoing pivotal Phase 3 clinical study evaluating omadacycline for the treatment of community acquired bacterial pneumonia (CABP) will report top-line data during the second quarter of 2017. The revised estimate reflects faster-than-expected patient enrollment in the study.


Woodcliff Lake-based Eagle Pharmaceuticals announced positive results from its pivotal animal study conducted under the Animal Rule1, for Ryanodex® for the treatment of Exertional Heat Stroke ("EHS"), an investigational new indication for the product. Due to the rare and unpredictable nature of EHS, the animal work is an essential part of the hybrid development program in support of the efficacy of Ryanodex for the treatment of EHS. The hybrid program is comprised of safety and efficacy data from a human study in EHS patients, and efficacy data from an established animal model. Both components are necessary to fulfill the NDA submission requirements.


Parsippany-based Castle Creek Pharmaceuticals, LLC (CCP), a global company dedicated to delivering transformative therapies to patients with dermatologic and head/neck orphan diseases and underserved conditions, announced completion of an exclusive license agreement for all U.S. rights to Arlevert® from Hennig Arzneimittel GmbH &Co. KG. Arlevert is a fixed-dose oral combination of cinnarizine and dimenhydrinate. Arlevert is approved in multiple European Union countries where it is indicated for the treatment of patients with vertigo of various origins.


Northvale-based Elite Pharmaceuticals, Inc. announced that it has filed an Abbreviated New Drug Application (ANDA) with the US Food and Drug Administration (FDA) for a generic version of Norco (hydrocodone bitartrate and acetaminophen tablets, USP CII) 2.5mg/325mg, 5mg/325mg, 7.5mg/325mg and 10mg/325mg tablets. Norco is a combination medication and is used to help relieve moderate to moderately severe pain. The combination products of hydrocodone and acetaminophen have total annual U.S. sales of approximately $700 million according to IMS Health data.


East Windsor-based Takeda Pharmaceutical Company Limited announced that an analysis based on pre-specified and post-hoc exploratory outcomes of GEMINI II and GEMINI III data evaluating Entyvio (vedolizumab) therapy in patients with moderately to severely active Crohn's disease (CD) was published in Inflammatory Bowel Diseases. The data demonstrated higher rates of response in patients receiving vedolizumab as a first-line biologic after conventional therapy failure versus patients who had previously experienced tumor necrosis factor-alpha (TNF-α) antagonist therapy failure.


Bridgewater-based Valeant Pharmaceuticals International, Inc. announced positive results from a Phase 3, multicenter double-blind, randomized, vehicle-controlled clinical study to assess the safety and efficacy of IDP-118 (halobetasol propionate and tazarotene) lotion in the treatment of plaque psoriasis. Within the Phase 3 study of 215 adult subjects with moderate to severe psoriasis, IDP-118 showed statistical significance to vehicle with a treatment success rate of 45.33% and a p<0.001.


Basking Ridge-based Lexicon Pharmaceuticals, Inc. announced top-line results  from a Phase 2 clinical study of sotagliflozin, a dual SGLT1 and SGLT2 inhibitor, conducted by Lexicon in collaboration with JDRF, the leading global organization funding type 1 diabetes research. The purpose of this Phase 2 clinical trial was to assess the effects of a once-daily 400 mg dose of sotagliflozin compared to a placebo control in insulin-treated young adults with type 1 diabetes, aged 18-30 years, and particularly high baseline A1C levels of greater than or equal to 9.0%, representative of an at-risk population in which glycemic control and adherence to therapy are substantial challenges.


Cranford-based Citius Pharmaceuticals, Inc. provided details of its Phase 3 trial for Mino-Lok™, an antibiotic lock solution used to salvage infected central venous catheters (CVCs) and to treat catheter related bloodstream infections (CRBSIs). Mino-Lok is being developed as an adjunctive therapy for the treatment of catheter-related or central line associated bloodstream infection (CRBSI/CLABSI) in combination with appropriate systemic antibiotic(s), to salvage infected CVCs, to preserve central venous access, and to avoid the complications and morbidities associated with catheter removal and reinsertion.


New Brunswick-based Visikol Inc. has launched Visikol® HISTO™, the market's first portfolio of tissue clearing kits and reagents that can integrate 3D imaging into existing tissue processing workflows. The Visikol HISTO technology requires no special equipment to clear tissue and provides a rapid, non-destructive, easy-to-use process that allows any researcher to add a new dimension to his or her research with the power of tissue clearing.


Parsippany-based Daiichi Sankyo Company, Ltd. and DarwinHealth announced a strategic partnership to use quantitative systems biology-based algorithms and novel, validated approaches focused on tumor checkpoints -- a new class of cancer targets -- to help prioritize investigational compounds in the Daiichi Sankyo Cancer Enterprise pipeline for clinical development.


Parsippany-based Daiichi Sankyo Company, Ltd. and Maruishi Pharmaceutical Co., Ltd. announced that they have launched a generic version of the analgesic for general anaesthesia Remifentanil Intravenous Injection 2mg and 5mg "Daiichi Sankyo". Remifentanil injection is an ultra-short acting opioid analgesic characterized by its potent analgesic action together with superior ability to regulate pain, and is widely used for general anaesthesia.


Parsippany-based Daiichi Sankyo Company, Limited announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its investigational HER2-targeting antibody drug conjugate DS-8201 for the treatment of HER2-positive unresectable and/or metastatic breast cancer in patients who have progressed after prior treatment with HER2-targeted therapies including ado-trastuzumab emtansine  (T-DM1).



A Swiss supplier of products for the pharmaceutical, biotech and specialty ingredients markets is paying $5.5 billion in cash for a Morristown drug-delivery solutions business.  Lonza Group AG, based in Basel, has entered into a definitive agreement with global investment firm KKR to buy Capsugel S.A., which manufactures oral dosage delivery products such as hard capsule technologies.



Institution and Education News



Memorial Sloan Kettering Cancer Center and Hackensack Meridian Health announced the Memorial Sloan Kettering-Hackensack Meridian Health partnership, which brings together two world-class organizations with deep New Jersey roots and a single goal: Finding more cures for cancer faster while ensuring that patients have access to the highest-quality, most individualized cancer care when and where they need it. To achieve this goal, the new Memorial Sloan Kettering-Hackensack Meridian Health partnership will combine both organizations' unparalleled expertise in all areas of cancer care and research, as well as their commitment to excellence, to accelerate new discoveries and improve the lives of patients they jointly serve, while training a new generation of leaders in the field.


Childhood cancer patients depend on clinical trials to find better, safer cures that can prevent lifelong damage as a result of radiation and chemotherapy. BioNJ Member Rutgers Cancer Institute of New Jersey will receive an infrastructure grant from the St. Baldrick's Foundation to expand access to their clinical trials. D espite the increase of childhood cancer survival rates in the last two decades, adolescent and young adult (AYA) patients have had minimal improvement in cure rates. Contributing to this statistic is the AYA population's low numbers of participation in clinical trials.  Funding from St. Baldrick's, totaling $50,000, will ensure that more AYA patients can be treated by clinical trials with the most innovative drugs and therapies available at Rutgers Cancer Institute of New Jersey.


A genomic analysis study by BioNJ Member Rutgers Cancer Institute of New Jersey investigators and other colleagues has identified recurrent genomic alterations in a subset of breast cancer that are typically associated with a form of thyroid cancer and an intestinal birth defect known as Hirschsprung disease. The study was conducted in conjunction with the Avera Center for Precision Oncology in South Dakota and Foundation Medicine in Massachusetts.


A new discovery, out of BioNJ Member Rowan University, may unlock the answer to a vexing scientific question: How to conduct mitochondrial replacement therapy, a new gene-therapy technique, in such a way that safely prevents the transmission of harmful mitochondrial gene mutations from mothers to their children.  A study published in the journal  Nature  suggests that clinicians select egg donors whose mitochondrial DNA (mtDNA) is compatible to the mother's ancestral mitochondria. Similar groups of mitochondrial DNA are known as haplotypes, each of which represents major branching points on the human genetic family tree.
People in the News



Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced the appointment of Gerald McMahon, Ph.D., to the company's Board of Directors. Dr. McMahon was previously the President and Chief Executive Officer of Kolltan Pharmaceuticals.  Prior to joining Kolltan, Dr. McMahon served as Senior Vice President, R&D Oncology at MedImmune, where he oversaw the strategy and investment for the oncologic biologics pipeline.



Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced that the company has hired Theresa LaVallee, Ph.D., to the newly created position of Senior Vice President, Regulatory and Precision Medicine. Dr. LaVallee was previously Senior Vice President, Translational Medicine and Product Development at Kolltan Pharmaceuticals, which Celldex recently acquired. Dr. LaVallee brings more than 15 years of industry experience in discovery and development of drug candidates in oncology and inflammation, including extensive global regulatory experience and expertise in translational medicine to enable science-driven drug development.



Cranbury-based BioNJ Member Cornerstone Pharmaceuticals, Inc., a clinical stage, oncology-focused pharmaceutical company and an innovator in the field of cancer metabolism, announced the hiring of Brian Mullaney, M.D., Ph.D., as Chief Medical Officer responsible forclinical development of Cornerstone's platform technology. Dr. Mullaney's hiring strengthens Cornerstone's drug development and commercialization capabilities, while also continuing the company's recent momentum.



Morristown-based  Pernix Therapeutics Holdings, Inc. , a specialty pharmaceutical company with a focus on Pain and CNS conditions, announced the appointment of Gabriel Leung to its Board of Directors, effective immediately. The Board also determined that Mr. Leung is an independent director and appointed him as Chairperson of the Nominating Committee and as a Member of the Audit and Compensation Committees.



Woodcliff Lake-based  Eisai Inc., the U.S. pharmaceutical unit of Eisai Co. Ltd., has named a new Senior Vice President.  Rebecca Jolley, a former Novartis executive, will serve as Eisai's Senior Vice President, Americas Commercial, in the Oncology Business Group. In her new role, Ms. Jolley will lead the Americas Oncology Commercial Team in the creation and driving of commercial strategies for approved and pipeline products, as well as late-stage compounds.



Plainsboro-based Integra LifeSciences Holdings Corp. announced it has appointed three new key executives:  Robert T. Davis Jr. has been named Corporate Vice President and President, Orthopedics and Tissues Technologies;  Dan Reuvers has been named Corporate Vice President and President, Specialty Surgical Solutions; and  Glenn Coleman, Corporate Vice President, Chief Financial Officer and Principal Accounting Officer, will assume oversight of the international business.

Upcoming Programs from Our Partners for BioNJ Members


January 17 | 4:30 p.m.-6:30 p.m. | Commercialization Center for Innovative Technologies 

Join us at the CCIT Incubator, located at the Technology Centre of New Jersey, a 75 acre life sciences research park in the heart of N.J.'s Research Corridor, for an insightful conversation about how life sciences companies will be funded by venture capital firms in 2017.  Learn tips and trends in biotech venture investing from the top biotech venture capital firms in the country.  


February 13-14 | Waldorf Astoria, New York

Registration is now open for the  BIO CEO & Investor Conference, one of the largest investor conferences focused on established and emerging publicly traded and select private biotech companies. BioNJ Members receive an  additional $200 off registration fees! Click here for the special registration code.