Welcome to the latest edition of the  BioLines  Weekender. 

As we move towards the end of 2018, we would like to wish all of our Members happy holidays and a  prosperous New Year. 

Thank you to all who joined us for yesterday's Third Annual Patient Advocacy Summit and Legislative Holiday Reception at Celgene, as well as BioNJ's Fourth Annual Inspiring Women in STEM Conference at Sanofi and our Legal, Compliance & Regulatory Implications of Patient Engagement Forum at Amicus Therapeutics last week. 

2018 has been an exciting year for New Jersey's life sciences ecosystem. We've seen the opening of new incubators supporting our emerging companies, the announcement of promising initiatives to boost New Jersey's innovation economy by Governor Murphy, the ribbon cuttings of new headquarters and facilities of biopharma companies expanding or moving to the Garden State and the approval of numerous novel FDA drug approvals.

Congratulations to the New Jersey Economic Development Authority (EDA) on the 20th anniversary of the  Technology Business Tax Certificate Transfer Program, more commonly known as the Net Operating Loss (NOL) Program. Since its inception, the EDA has approved more than $1 billion in funding for more than 540 life sciences and technology companies.

Entrepreneurship is key to the growth of the ecosystem. BioNJ recently announced our Second Entrepreneurship Fellowship program and is looking for companies to host a Fellow. Please click here to learn more. Congratulations to BioNJ Board Member and President and CEO of Soligenix, Dr. Christopher Schaber, for being appointed to the  Rowan University Entrepreneurship Advisory Council.  
Finally, if you aren't a BioNJ Member, contact us today at BioNJ@BioNJ.org to learn about the many benefits of Membership. Please join us in our mission to help our Members help Patients. Thank you. 

Because Patients Can't Wait®,
The BioNJ Team
BioNJ Calendar

BioNJ's Annual Dinner Meeting & Innovation Celebration
Hilton East Brunswick, East Brunswick, NJ

February 7, 2019

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

Medical innovation enables longer and better quality lives and, in turn, reduces the costs of healthcare. Share with our community how your medical innovation is improving lives and impacting healthcare costs. Send us your Patient and related stories to BioNJ@BioNJ.org. Plus, click here for valuable tools and resources on the Value of Medical Innovation.

The New Jersey Economic Development Authority (EDA) announced that it has approved more than $1 billion in funding for more than 540 technology and life sciences companies through the Technology Business Tax Certificate Transfer Program, more commonly known as the Net Operating Loss (NOL) Program, since the program's inception 20 years ago. Heralded as a lifeline for companies going through unprofitable times, the NOL Program enables eligible technology and life sciences companies to sell New Jersey net operating losses and unused research and development tax credits to unrelated profitable corporations for cash. The cash can then be used for working capital or to fund research. 

Technology and life sciences start-ups considering a move into incubators, accelerators or coworking spaces are now able to apply for rent support grants at nine collaborative workspaces throughout the state. These nine workspaces are the first to be approved by the New Jersey Economic Development Authority (NJEDA) to participate in  NJ Ignite In support of Governor Phil Murphy's strategy to create a thriving innovation ecosystem, NJ Ignite benefits both emerging technology and life science companies and participating collaborative workspaces. The program provides up to nine months of rent support for startup businesses moving to collaborative workspaces.

In patients with  myelodysplastic syndromes  (MDS), a cancer in which the bone marrow does not make enough healthy blood cells, red blood cells may not mature and function properly. As a result, about 85 percent of patients with MDS develop serious  anemia . Since roughly half of patients do not respond to current therapies aimed at increasing red blood cell production, many end up relying on frequent transfusions to treat the symptoms associated with anemia.

Beta-thalassemia  is a blood disorder, with more than 60,000 infants born worldwide with the disease each year. However, unless you live in Asia, India, the Middle East or the Mediterranean where beta-thalassemia is most prevalent, you may never have heard of this inherited blood disorder that disrupts the body's ability to make hemoglobin. But that may be changing as doctors in Europe and North America are increasingly encountering patients with beta-thalassemia. 

More people are being diagnosed with Crohn's disease and ulcerative colitis than ever before, but researchers aren't exactly sure why. A variety of factors including genetics, weakened immune systems and the environment may be at play.

BioNJ in the News

Despite New Jersey's historic reputation as the "nation's medicine chest" - a hub for pharmaceutical research and manufacture - clinical trials on drugs developed here have traditionally been done out of State. Last Monday, the Assembly health committee approved legislation to create a working group with public and private members to study what barriers prevent these entities -- drug makers, higher education, and clinical providers -- from collaborating effectively, recommend ways they can better work together, and identify opportunities for encouraging more clinical trials.

Debbie Hart, P resident and CEO of BioNJ, said  "BioNJ strongly supports efforts to further advance New Jersey's status as a global hub for biopharmaceutical research, development, manufacturing, clinical research and clinical trials." She also welcomed the chance to work with lawmakers to build on existing collaborations. 

It has been proven, Debbie Hart said, that companies with women throughout their organizations -- including in higher-ranking positions -- are more successful.  "That is why today speaks directly to our commitment at  BioNJ ," Hart, President and CEO of the organization, said. "Our mission is to make sure our members have what they need to help patients, which includes being able to employ strong and successful women in the board rooms and throughout the ranks at their companies."

NJ Company News

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

Newark-based BioNJ Member   Rafael Pharmaceuticals, Inc. announced the initiation of a phase 3 multicenter, open-label, randomized pivotal trial (AVENGER 500) to evaluate the efficacy and safety of its lead compound CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX) as first-line therapy in patients with metastatic adenocarcinoma of the pancreas. AVENGER 500 will compare the efficacy and safety of FOLFIRINOX (FFX, control arm) with CPI-613 in combination with mFFX.

Newark-based BioNJ Member   Rafael Pharmaceuticals, Inc. announced the initiation of a Phase 3, multicenter, open-label, randomized, pivotal trial (ARMADA 2000) to evaluate the efficacy and safety of its lead compound CPI-613 (devimistat) in combination with high dose cytarabine and mitoxantrone (CHAM) in older patients with relapsed or refractory acute myeloid leukemia (AML). ARMADA 2000 will compare the efficacy and safety of high dose cytarabine and mitoxantrone (HAM, control arm) with CHAM. 

Eatontown-based BioNJ Member American CryoStem Corporation announced receipt from the USPTO of the Issue Notification for our Patent titled ''Systems and Methods for the Digestion of Adipose Tissue Samples from a Client for Cryopreservation'' for Application No. 13/646,647 (the ''Digestion Patent''). With the granting of this Intellectual Property, the Company's third major patent covering our methods of Collection, Processing, Expansion, Cell Culture Medium, Cryogenic Storage and Distribution of adipose tissue and adipose-derived stem cells, 

Princeton-based BioNJ Member Soligenix, Inc. announced that it has received preliminary approval for a tax credit from the New Jersey Economic Development Authority's (NJEDA) New Jersey Technology Business Tax Certificate Transfer program. As a result, the company anticipates being able to transfer this credit and receive approximately $611,000 in net proceeds by year end.

Princeton-based BioNJ Member Soligenix, Inc. announced publication of a review article discussing the therapeutic applications of its innate immune modulator technology, including dusquetide (the active ingredient in SGX942), its lead clinical Innate Defense Regulator (IDR). The article entitled, "Targeting Innate Immunity to Treat Disease: Potential Therapeutic Applications", is published in the journal Drug Target Review onlineInnate immune modulation as a therapeutic intervention reflects our enhanced understanding of an ancient aspect of the immune system. 

Summit-based BioNJ Member Celgene Corporation announced initial data from the dose-escalation part of an ongoing, open-label multicenter Phase 1/2 study of investigational lisocabtagene maraleucel (liso-cel; JCAR017) in patients with relapsed/refractory chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL), including patients with cytogenetic features of high-risk disease, who were previously treated with ibrutinib. Data presented from TRANSCEND CLL-004 include 16 patients from the ongoing Phase 1 monotherapy dose-escalation part of the study. 

Summit-based BioNJ Member Celgene Corporation announced initial safety data from its ongoing proof-of-concept trial of JCARH125 in patients with relapsed/refractory multiple myeloma. JCARH125 is an investigational BCMA-targeting CAR T cell therapy being developed by Juno Therapeutics, a Celgene company. The data reported from the multicenter, phase 1/2 EVOLVE trial includes patients who have been treated with JCARH125 in the dose escalation study. The primary objectives of the Phase 1 portion of the trial are safety and identification of a recommended Phase 2 dose. 

Summit-based BioNJ Member Celgene Corporation announced results of the Phase 3 AUGMENT study, which showed that REVLIMID® (lenalidomide) in combination with rituximab (R2) demonstrated superior progression-free survival (PFS) in patients with relapsed/refractory indolent lymphoma compared to patients who received rituximab plus placebo (R-placebo). The Phase 3 randomized, double-blind, international clinical study evaluated the efficacy and safety of the investigational combination of R2 versus rituximab plus placebo in patients (n=358) with relapsed/refractory follicular (n=295) and marginal zone (n=63) lymphoma.

Iselin-based BioNJ Member Helsinn Group and MEI Pharma, Inc. announced interim data from a Phase 2 study evaluating pracinostat, a histone deacetylase inhibitor, in combination with azacitadine for the treatment of patients with IPSS-R high/very high-risk of Myelodysplastic Syndrome (MDS). The data demonstrate a 9% discontinuation rate due to adverse events, a substantially lower rate than observed in an earlier study, as well as an encouraging 36% complete response rate among patients receiving at least 6 cycles of treatment. 

Iselin-based BioNJ Member Helsinn Group and Mundipharma China Pharmaceutical jointly announce that ALOXI® IV has received approval from the National Medical Products Administration (NMPA). This is the first Helsinn cancer supportive care product to be approved for marketing in China. Mundipharma China Pharmaceutical has exclusive marketing, promotion and sale rights for ALOXI® in China.

Pennington-based BioNJ Member   OncoSec Medical Incorporated announced an update regarding tumor responses in its KEYNOTE-695 global, multicenter Phase 2b, open-label trial of intratumoral delivery of TAVO (tavokinogene telseplasmid / IL-12) with intravenous KEYTRUDA® (pembrolizumab) in patients with unresectable, advanced melanoma. The Company previously reported at the Society for Immunotherapy of Cancer's 33rd Annual Meeting (SITC) that, of the first nine patients to complete 12 weeks of treatment and reach initial tumor evaluation (by RECIST v1.1), two patients had a partial response.  

Pennington-based BioNJ Member   OncoSec Medical Incorporated announced the presentation of new data from the company's Phase 2 study of TAVO (tavokinogene telseplasmid) for the treatment of metastatic melanoma. The newly presented data reports abscopal responses with TAVO™; monotherapy, finding that treatment with TAVO™ monotherapy resulted in 47 percent of patients experiencing tumor regression in at least one untreated lesion. 

Berkeley Heights-based BioNJ Member Chugai Pharmaceutical Co., Ltd. announced that humanized anti-PD-L1 monoclonal antibody TECENTRIQ® Intravenous Infusion 1200 mg [generic name: atezolizumab (genetical recombination)], and ROS1/TRK inhibitor entrectinib which is under development, received orphan drug designation by the Ministry of Health, Labour and Welfare for the treatment of small cell lung cancer (SCLC) and NTRK fusion-positive locally advanced or metastatic solid tumors, respectively.

Berkeley Heights-based BioNJ Member Chugai Pharmaceutical Co., Ltd. announced that the interim analysis data of the Phase I/II study COMPOSER study (NCT03157635) of anti-C5 antibody SKY59, an investigational drug administrated subcutaneously for Paroxysmal Nocturnal Hemoglobinuria (PNH) in patient subjects, was presented at the 60th American Society of Hematology (ASH) Annual Meeting. A Phase I/II global, multicenter, open-label study to assess the safety and efficacy, pharmacokinetics, and pharmacodynamics of SKY59 in healthy volunteers and in patients with PNH.

Paramus-based BioNJ Member PsychoGenics Inc. and Sunovion Pharmaceuticals Inc. announced positive results from SEP 361-201, a pivotal Phase 2 study that evaluated the efficacy and safety of SEP-363856, a novel psychotropic agent for the treatment of patients with schizophreniaThe study met its primary endpoint, demonstrating that hospitalized patients with acute exacerbation (worsening) of schizophrenia treated with SEP-363856 showed statistically significant and clinically meaningful improvement in the Positive and Negative Syndrome Scale (PANSS) total score compared to placebo after four weeks of treatment (-17.2 vs. -9.7, respectively; p=0.001).

BioNJ Member Pfizer Inc., with offices in Madison, announced at the American Society of Hematology Annual Meeting that the REFLECTIONS B328-06 study, a comparative safety and efficacy study of PF-05280586 versus Rituxan®/MabThera® (rituximab-EU)i, met its primary endpoint of overall response rate (ORR) at Week 26 of the 52-week study. "It is encouraging to see new data supporting a potential rituximab Biosimilar. If approved this may help provide a more cost-effective treatment option and expand access for patients and physicians," said Dr. Jeff Sharman, Medical Drector, US Oncology Hematology Research.

Kenilworth-based BioNJ Member   Merck& Co. announced a collaboration agreement to develop vaccines to protect against dengue virus disease, the mosquito-borne infection. Instituto Butantan and Merck have licensed certain rights from National Institute of Allergy and Infectious Diseases (NIAID), part of the United States National Institutes of Health (NIH), for the development of live attenuated tetravalent vaccines (LATV). Instituto Butantan's dengue vaccine candidate, TV003, is currently being evaluated in a large Phase 3 study in Brazil.

Bridgewater-based BioNJ Member   Eli Lilly and Company  announced that the  U.S. Food and Drug Administration  ( FDA ) has granted Fast Track designation to baricitinib, which is being studied for the treatment of systemic lupus erythematosus (SLE). "There has been only one new treatment for SLE approved in the U.S. in the past 50 years, and Lilly is excited to be at the forefront of potentially bringing a new treatment option to patients with this chronic, multi-organ autoimmune disease that can cause widespread tissue damage," said Lotus Mallbris, M.D., Ph.D., Vice President of Immunology.

Bridgewater-based BioNJ Member   Eli Lilly and Company  and  AC Immune SA  announced that the two companies have signed a license and collaboration agreement to research and develop tau aggregation inhibitor small molecules for the potential treatment of Alzheimer's disease (AD) and other neurodegenerative diseases. The collaboration combines  AC Immune's  proprietary Morphomer platform technology with Lilly's clinical development expertise and commercial capabilities in central nervous system disorders. The collaboration will focus primarily on  AC Immune's  lead molecule, ACI-3024, which has demonstrated tau aggregation inhibition in preclinical models.

Princeton -based Sandoz Inc., a division of BioNJ Member  Novartis, and Pear Therapeutics, Inc., the leader in prescription digital therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted clearance for reSET-OTMreSET-O is intended to increase retention of patients with Opioid Use Disorder (OUD) in outpatient treatment by providing cognitive behavioral therapy (CBT), as an adjunct to outpatient treatment that includes transmucosal buprenorphine (medication-assisted-treatment, or MAT) and contingency management, for patients 18 years or older who are currently under the supervision of a clinician. 

East Hanover -based BioNJ Member  Novartis announced data from subgroup analyses of the three pivotal Phase III MONALEESA trials showing that Kisqali® (ribociclib) plus endocrine therapy extended progression-free survival (PFS) compared to endocrine therapy alone, regardless of the presence of visceral metastases in pre-, peri- and postmenopausal women with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced breast cancer.

East Hanover-based BioNJ Member Novartis announced the initiation of Phase III trials for ligelizumab (QGE031) -- a high-affinity monoclonal anti-IgE antibody -- in chronic spontaneous urticaria (CSU) patients whose symptoms are inadequately controlled by H1-antihistamines. Phase III studies PEARL1 and PEARL 2 are planned to include more than 2,000 CSU patients. "CSU has a big impact on patients' lives," said Marcus Maurer, M.D., Professor of Dermatology and Allergy and Director of Research at the Department of Dermatology and Allergy, Allergie-Centrum-Charité of the Charité-Universitätsmedizin in Berlin, Germany.

East Hanover-based BioNJ Member Novartis announced additional analysis from the global Phase III SOLAR-1 trial investigating the alpha-specific PI3K inhibitor BYL719 (alpelisib) in combination with fulvestrant in men and postmenopausal women with PIK3CA mutated hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) advanced or metastatic breast cancer. In SOLAR-1, the addition of BYL719 to fulvestrant nearly doubled median progression-free survival (PFS) in patients with PIK3CA mutated HR+/HER2- advanced breast cancer who progressed on or after an aromatase inhibitor (AI) compared to fulvestrant alone. 

East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited treatment options. ZOLGENSMA previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019. 

Titusville-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the ECLIPSE study demonstrating that TREMFYA® (guselkumab) was superior to Cosentyx®(secukinumab) in treating adults with moderate to severe plaque psoriasis for the primary endpoint assessed at week 48. Data from the multi-center, randomized, double-blind head-to-head Phase 3 study demonstrated that 84.5 percent of patients treated with TREMFYA achieved at least 90 percent improvement in their baseline Psoriasis Area Severity Index (PASI) score at week 48, compared with 70.0 percent of patients treated with Cosentyx (p<0.001).

Titusville-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson reported updated results from Legend Biotech Inc.'s LEGEND-2 Phase 1/2 open-label study, which evaluated the investigational chimeric antigen receptor T-cell (CAR-T) therapy LCAR-B38M in the treatment of patients with advanced relapsed or refractory (R/R) multiple myeloma. The findings build upon the data from one of four independent institutional studies, the Second Affiliated Hospital of Xi'an Jiaotong University. 

Titusville-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the Phase 3 MAIA study demonstrating that the addition of DARZALEX® (daratumumab) to lenalidomide and dexamethasone (Rd) significantly reduced the risk of disease progression or death in patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT)

Titusville-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the National Cancer Institute (NCI)-sponsored Phase 3 study (E1912) led by the ECOG-ACRIN Research Group (ECOG-ACRIN) evaluating IMBRUVICA® (ibrutinib) plus rituximab compared to a chemotherapy regimen of fludarabine, cyclophosphamide, and rituximab (FCR) in previously untreated patients aged 70 years or younger with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). 

Morris Plains-based BioNJ Member  Immunomedics, Inc.  presents updated Phase 2 results at the 2018 SABCS, confirming that monotherapy with sacituzumab govitecan achieved an objective response rate (ORR) of over 30 percent among heavily pre-treated patients with metastatic triple-negative breast cancer (mTNBC), with a manageable safety profile. With an additional 5 months of follow-up for the previously reported mTNBC patient cohort, sacituzumab govitecan monotherapy continued to demonstrate robust clinical activity with an ORR of 33 percent and 34 percent based on local assessment and blinded independent central review (BICR), respectively. 

Princeton-based BioNJ Member   Bristol-Myers Squibb and Vedanta Biosciences announced a clinical trial collaboration to evaluate Bristol-Myers Squibb's programmed death-1 (PD-1) immune checkpoint inhibitor Opdivo in combination with Vedanta Biosciences' VE800, a rationally-defined human bacterial consortium, in patients with advanced or metastatic cancers. In a range of preclinical models of cancer, including those sensitive and resistant to checkpoint inhibition, VE800 was shown to induce CD8+ T cells, potentiate the immune system's attack of tumor cells, and significantly amplify the effects of anti-PD-1 therapy.

Cranbury-based BioNJ Member  Oncobiologics, Inc.  announced several key corporate events that enhance the company's ability to advance the development of its lead product candidate, ONS-5010, a proprietary ophthalmic bevacizumab product candidate for the treatment of wet age-related macular degeneration (wet AMD). The company has changed its name from  Oncobiologics, Inc.  to  Outlook Therapeutics, Inc. , effective immediately.  The company will continue to be listed on the  Nasdaq Capital Market  and its common stock and Series A warrants will begin trading under the ticker symbols "OTLK" and "OTLKW." 

Princeton-based BioNJ Member  Agile Therapeutics, Inc.  announced that on  December 11, 2018 , it met with the U.S. Food and Drug Administration's  Division of Bone , Reproductive, and Urologic Products ("DBRUP") to discuss the design of a comparative wear study between Twirla® and Xulane® as suggested by FDA's  Office of New Drugs  ("OND") in its decision on the company's formal dispute resolution request, which was announced in October 2018.

Bridgewater-based BioNJ Member Nevakar Inc., a specialty pharmaceutical company developing multiple differentiated products in the ophthalmic and injectable areas, has entered into a $20 million senior secured term loan with Oxford Finance LLCAccording to a statement, as part of the financing agreement,Nevakar may also access an additional $10 million of funding provided that certain pre-defined milestone events are achieved. Proceeds from the financings will be used to accelerate the company's pipeline projects and for general corporate services.

Lawrenceville-based Asana BioSciences announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Asana's investigational oral Janus Kinase (JAK) and Spleen Tyrosine Kinase (SYK) dual inhibitor ASN002 for the treatment of moderate-to-severe atopic dermatitis. "We are pleased that the FDA has granted Fast Track designation to ASN002. ASN002 is currently being evaluated in moderate-to-severe atopic dermatitis in the Phase 2b RADIANT Study (Relief from Atopic DermatitIs with JAK and SYK INhibiTion - NCT03654755). It is also being evaluated in a Phase 2 trial in patients with severe chronic hand eczema (NCT03728504).

Woodcliff Lake-based  Eisai Co., Ltd. and University College London (UCL) announced that they have commenced preparations for Phase I clinical studies on E2814, the first clinical candidate from their drug discovery collaboration, in Alzheimer's disease patients within fiscal 2018. E2814 is an anti-tau monoclonal antibody which is designed to slow the progression of Alzheimer's disease and other tauopathies. In addition, the research collaboration, agreed in 2012 for an initial period of six years, has been extended for a further 5 years to 2023.

Basking Ridge-based  Daiichi Sankyo Co. and Nashville, Tennessee-based  Sarah Cannon Research Institute announced a strategic collaboration to develop novel cancer therapies.  The partnership will combine Daiichi Sankyo's ability to develop novel cancer agents with Sarah Cannon's ability to design and optimize clinical trials. Under the agreement, Sarah Cannon will provide clinical development services to several of Daiichi Sankyo's translational development programs. 

Basking Ridge-based Daiichi Sankyo Company, Limited announced updated efficacy and safety results from the ongoing Phase 1/2 study of U3-1402, an investigational and potential first-in-class HER3-targeting antibody drug conjugate (ADC), in 42 heavily pretreated patients with HER3-positive metastatic breast cancer. Updated efficacy data for 42 evaluable patients who received U3-1402 in dose levels between 1.6 mg/kg to 8.0 mg/kg in the dose escalation and dose finding parts of the study showed a confirmed overall response rate of 42.9 percent (18/42 patients) and a disease control rate of 90.5 percent (38/42 patients) at a median follow-up time of 10.5 months.

East Windsor-based Lynkogen Inc. and Alteogen Inc. announced that they have entered into an exclusive option agreement for the development of Alteogen's novel GLP-1 mimetic and Alpha-1 Antitrypsin (A1AT) fusion proteins based on their proprietary NexP TM  fusion technology to tackle NASH and metabolic diseases. Under the terms of the agreement Lynkogen will have exclusive worldwide rights, excluding Korea and emerging markets, to develop and commercialize therapeutic GLP-1/A1AT fusion proteins.

Jersey City-based Mitsubishi Tanabe Pharma America, Inc. announced it will conduct a study to identify and measure specific biomarkers in people with amyotrophic lateral sclerosis (ALS). The findings may assist researchers and clinicians in starting the process of validation and the use of biomarkers as quantifiable, biological, non-clinical measures for disease progression and treatment effect of edaravone in people with ALS. Interim data are expected in 2019.

Florham Park-based Cellectar Biosciences announces the initiation of Cohort 6 of its ongoing Phase 1b trial evaluating CLR 131 for the treatment of relapsed/refractory (R/R) multiple myeloma (MM). Cohort 6 will evaluate up to four patients with each receiving two doses of 18.75 mCi/mof CLR 131 administered one week apart. This fractionated dosing regimen will result in each patient being treated with a total of approximately 75.0 mCi of CLR 131, representing an increase in average total exposure of greater than 15% over Cohort 5.

Warren-based  Aquestive Therapeutics, Inc.  announced findings from two clinical studies, including the Adult Epilepsy Monitoring Unit (EMU) study, showing that its investigational diazepam buccal film (DBF) was successfully used and had similar bioavailability whether administered between seizures (interictal) or during or shortly after seizures (ictal/peri-ictal) in adults with poorly controlled tonic-clonic seizures or focal seizures with impaired awareness. Investigational diazepam buccal film (DBF), tentatively named Libervant, is a novel formulation of diazepam as a small, thin film strip for placement inside the cheek.

Lexicon Pharmaceuticals, Inc., with offices in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) plans to review Lexicon collaborator BioNJ Member Sanofi's New Drug Application (NDA) for sotagliflozin, an investigational oral treatment for adults with type 1 diabetes, on January 17, 2019. The NDA included data from the inTandem clinical trial program, which includes three Phase 3 clinical trials assessing the safety and efficacy of sotagliflozin in approximately 3,000 adults with inadequately controlled type 1 diabetes.

Fair Lawn-based SK Life Science, Inc. announced results of a large, Phase 3, multicenter, open-label safety study of its lead investigational antiepileptic drug, cenobamate (YKP3089). Two previous pivotal, randomized, double-blind, placebo-controlled studies evaluated the efficacy and safety of cenobamate in adult patients with partial-onset seizures. During this clinical development program, a serious, delayed hypersensitivity adverse event, DRESS (drug reaction with eosinophilia and systemic symptoms) syndrome, emerged.

Takeda Pharmaceutical Company Limited, with offices in East Windsor, presented data from the Phase 3 randomized, TOURMALINE-MM3 study evaluating the effect of single-agent oral NINLARO™ (ixazomib) as a maintenance therapy in adult patients diagnosed with multiple myeloma who previously responded to high-dose therapy (HDT) and autologous stem cell transplant (ASCT)The trial achieved its primary endpoint with NINLARO resulting in a statistically significant improvement in progression-free survival (PFS) versus placebo in adult patients diagnosed with multiple myeloma who responded to HDT and ASCT as assessed by an Independent Review Committee (IRC) (HR 0.72; p-value=0.002). 

Takeda Pharmaceutical Company Limited, with offices in East Windsor, announced that data from the ECHELON-2 phase 3 clinical trial will be presented in an oral session at the 60th American Society of Hematology (ASH) Annual Meeting. The data demonstrated that frontline treatment with ADCETRIS (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) is effective in extending progression-free survival (PFS) and overall survival (OS) with a safety profile comparable to CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone), a current standard of care in patients with CD30-expressing peripheral T-cell lymphomas (PTCL).

GlaxoSmithKline plc, with offices in Warren, and TESARO Inc. announced that the companies have entered into a definitive agreement pursuant to which GSK will acquire TESARO, an oncology-focused company, for an aggregate cash consideration of approximately $5.1 billion (£4.0 billion). The proposed transaction will significantly strengthen GSK's pharmaceutical business, accelerating the build of GSK's pipeline and commercial capability in oncology.

Montclair-based Endomedix, Inc. announced that it has submitted a comprehensive patent application to protect both its latest chemistry technology and also the design and function of the spray-on hydrogel hemostat that it is developing for brain and spine surgery indications. This patent application further expands and deepens the protection of its intellectual property provided by the eight issued patents that the Company has previously obtained.

East Rutherford-based  Cambrex Corp.  entered into a definitive agreement to acquire  Avista Pharma Solutions , a contract development, manufacturing and testing organization, for about $252 million. I n a statement, Cambrex said that with the acquisition, Cambrex will enter the large and growing market for early stage small-molecule development and testing services.  Avista, for its part, offers a broad suite of scientifically differentiated services ranging from API and drug product development to standalone analytical, microbiology testing and solid-state sciences. 

Quest Diagnostics announced that it will build a flagship laboratory in Clifton.  The provider of diagnostic information services said that the 250,000-square-foot laboratory will become the largest in the Quest network of more than 20 laboratory facilities across the nation, and will employ more than 1,100 people. The new laboratory is expected to be operational in early 2021.  

Franklin Lakes-based medical technology company  BD  unveiled a new software application designed to help hospitals and health systems identify drug diversion . In a press release, BD said that as part of the BD HealthSight platform that is designed to support enterprise-wide medication management, the new BD HealthSight diversion management application is "the next step in the company's efforts to address drug diversion through integrated solutions and analytics."

People in the News

New Jersey, one of the biggest melting pots in the country, is on pace to become a majority minority state in the next 10 years.  The influence people of color have in the State is growing quickly, as well. Within "ROI Influencers," you'll find the ranking of the 10 most influential people of color in the State, as well as 50 other influencers.  The lists were determined by interviews with key people in all sectors of the economy, as well as the judgment of the ROI-NJ Editorial Board. BioNJ congratulates its Members including, Jose Lozano, Choose New Jersey; Wes Mathews, New Jersey EDA; Kennith Frazier, Merck & Co.; Sonia Delgado, Princeton Public Affairs; Ali Houshmand, Rowan University and Nariman Farvardin, Stevens Institute of Technology for making the list.

East Hanover-based BioNJ Member Novartis announced the appointment of Dr. Klaus Moosmayer as Chief Ethics, Risk and Compliance Officer. He will report to Vas Narasimhan, M.D., CEO of Novartis and  become a member of the Executive Committee of Novartis (ECN). Dr. Moosmayer has been the Chief Compliance Officer of Siemens since January 1, 2014. Prior to that he served as the Chief Counsel Compliance at Siemens for four years. During this time he played a key role in building up Siemens' new, globally recognized compliance system.

Parsippany-based BioNJ Member  The Medicines Co.  announced that its has appointed Mark Timney its new CEO and member of the board of directors. He  will succeed Clive Meanwell, who has been named Chief Innovation Officer. Mr. Timney, who has more than 25 years of biopharmaceutical industry experience, previously served as President U.S., President Japan and President of global primary care at Merck & Co. After Merck, he was CEO of Purdue Pharmaceuticals. 

Adam Schechter, President of Global Human Health at  Merck, will step down from his current position as of Dec. 31 and shift to an advisory capacity to the CEO.  Concurrently, the Kenilworth-based company is creating two new executive committee roles, with Frank Clyburn being promoted to Chief Commercial Officer and Michael Nally Chief Marketing Officer.

Foamix Pharmaceuticals Ltd., with its U.S. headquarters in Bridgewater, announced it has appointed Matt Wiley its new Chief Commercial Officer.  In this newly-created role, Mr. Wiley will be responsible for developing and executing commercial strategies for the company's product portfolio of dermatological topical foams. Mr. Wiley, who has more than 20 years of commercial experience in the pharmaceutical industry, was previously at Jazz Pharmaceuticals for six years.

The Family Resource Network said that effective Jan. 1, Chief Operating Officer Liza Gundell will assume the role of Chief Executive Officer when current CEO Eric Joice retires. Mr.  Joice has served as CEO for the nonprofit since 1983. Under his purview, FRN saw its annual revenue increase to nearly $10 million from under $100,000, the organization said. Prior to joining FRN, Ms. Gundell served as Director of Support Services for the Brain Injury Alliance of New Jersey.

The Rev. Dr. DeForest "Buster" Soaries Jr., has been appointed to  RWJBarnabas Health 's Board of Trustees and will serve a three-year term. "As a current member of the Board of Robert Wood Johnson University Hospital in New Brunswick, Rev. Soaries devotes his energies to improving the lives of the patients we serve," said Jack Morris, Chairman of the RWJBarnabas Health Board. Rev. Dr. Soaries served as New Jersey Secretary of State from 1999 to 2002.

Institution and Education News

Read the latest news on research, treatment, prevention and education from the State's National Cancer Institute-designated Comprehensive Cancer Center in Cancer Connection Monthly.

Rutgers recently became a partner in an innovative center -- funded with a $5.2 million National Science Foundation grant -- to translate the importance of scientific research to the general public. The ARIS Center will benefit researchers who drive discovery and "broader impact professionals" who collaborate with researchers and community stakeholders. It will engage members of the public who benefit from research and educational advancements. The center will stress support for traditionally underserved populations and inclusiveness to ensure a diverse science workforce.

American Water welcomed hundreds of people to a ceremony to mark the opening of its new national corporate headquarters on the Camden Waterfront.  The American Water Charitable Foundation presented a check of $200,000 to the  Camden School District  to develop an advanced STEM Lab at  Woodrow Wilson High School . Educators are prioritizing science, technology, engineering and mathematics education to prepare students for jobs that are in high demand.

Freehold-based CentraState has named Dr. John Pellegrino Medical Director of the hospital's Comprehensive Breast Cancer Program. Dr. Pellegrino specializes in the surgical treatment of breast cancer and other diseases of the breast. He has been on staff at CentraState Medical Center for more than 20 years.

The NJBAC is a state agency offering free and confidential assistance to help your business grow. As a "one-stop shop," NJBAC can help businesses of all sizes and types to manage the state's regulatory processes, boost exports, gain access to financial resources where applicable and tackle other obstacles along the way. You can download the NJBAC guide by clicking here or contact their business advocates at 1-800 JERSEY 7. 
Special Programs from Our Partners for BioNJ Members

December 17, 2018 | East Hanover

Students 2 Science (S2S) invites you to attend our  Pharmaceutical Day Open House, on December 17, at the S2S East Hanover Technology Center. Join us and learn about our programs, services and opportunities to volunteer. Come and experience one of these comprehensive high school programs where corporate mentors join students in our state-of-the-art commercial laboratory which is outfitted and equipped with $4 million of scientific instrumentation and equipment to provide authentic learning experiences. For more information and to learn how to register, click here.  

January 8, 2019 |  San Francisco

San Francisco's annual healthcare conference week starts in less than a month. Already, hundreds of investors have signed up to attend RESI San Francisco -- our largest event of the year, which serves as a dedicated venue for early stage investors to meet with start-ups in the biotech, medical device, diagnostic and digital health sectors. Would you like to book up to 16 face-to-face meetings with investors on January 8th? Click here to learn more. BioNJ Members enjoy a discount.

January 18-20, 2019 | The College of New Jersey

The College of New Jersey will be hosting the Conference for Undergraduate Women in Physics for the Mid-Atlantic states. The goal of the conference is to inspire, empower and enable young women scientists to pursue their career aspirations by equipping them with the skills and resources needed to achieve success in physics. The conference will host 200 undergraduate students and 50 high school students, and it will feature lectures by prominent women physicists, student research presentations, a networking fair featuring graduate schools, industries, and nonprofits, and workshops and panel discussions on professional development. For more information, please visit http://cuwip.tcnj.edu

June 3-6, 2019 | Philadelphia Convention Center

The BIO International Convention is hosted by the Biotechnology Innovation Organization (BIO), which represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. The key benefits of attending the 2019 BIO International Convention are access to global biotech and pharma leaders via BIO One-on-One Partnering, exposure to industry thought leaders with over 500 education sessions at your fingertips and unparalleled networking opportunities with 16,000+ attendees from 67 countries. 

BIO is offering BioNJ Members the opportunity to lock in a significant discount on two premier registration packages for the 2019 BIO International Convention and Convention Access & Partnering. Click here for details.