Welcome to the latest edition of the  BioLines Weekender

With the holiday approaching, we'd like to wish our industry colleagues and friends a Happy Thanksgiving! 

We hope to see you at a future BioNJ event, including  BioNJ's  Fifth Annual Inspiring Women in STEM Conference , entitled "Current and Future Progression of Women in the Workforce: Advancing Women in Life Sciences," on December 6 at Sanofi and BioNJ's Cybersecurity Awareness Breakfast Briefing on December 12 at Princeton Innovation Center BioLabs, where you'll hear directly from the FBI on cyber risk.

Visit www.BioNJ.org for more information. If you are not yet a BioNJ Member, contact Kim Minton today at KMinton@BioNJ.org  to learn about the many benefits of Membership. Thank you. 
Because Patients Can't Wait®,
The BioNJ Team

P.S. We'd like to share these two new guides designed by the New Jersey Action Center to help New Jersey businesses navigate State government. 
BioNJ Calendar

Inspiring Women in STEM Conference
Sanofi, Bridgewater, NJ
December 6, 2019
Cybersecurity Awareness Breakfast Briefing
Princeton Innovation Center BioLabs, Princeton, NJ
December 12, 2019
2020 Annual Dinner Meeting & Innovation Celebration
Hilton East Brunswick, NJ
February 6, 2020
BioNJ's 10th Annual BioPartnering Conference
Somerset, NJ
Save the date!
May 13, 2020
Putting Patients First: The Value of Medical Innovation

Medical innovation enables longer and better quality lives and, in turn, reduces the costs of health care. Share with our community how your medical innovation is improving lives and impacting health care 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.

As the national debate over prescription drug pricing reaches a fever pitch, drug manufacturers bringing new products to market face unique challenges. From the threats of political oversight and media scrutiny to academic guesstimates on the value of these products, the current environment for bringing breakthrough treatments to market is marked by unprecedented scrutiny. Perhaps nowhere is this more apparent than in the burgeoning world of cost-effectiveness analysis.

NJ Company News

East Hanover-based BioNJ Member  Novartis announced that the U.S. Food and Drug Administration (FDA) approved Adakveo® (crizanlizumab), previously known as SEG101, to reduce the frequency of vaso-occlusive crises (VOCs), or pain crises, in adult and pediatric patients aged 16 years and older with sickle cell disease. Adakveo represents the first FDA-approved medicine in sickle cell disease that binds to P-selectin -a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.

East Hanover-based BioNJ Member  Novartis announced new subgroup analyses from its global Phase III PARAGON-HF study of patients with heart failure with preserved ejection fraction (HFpEF), also known as diastolic heart failure. The data suggest that, in specific subgroups, treatment with Entresto may result in greater reductions in heart failure hospitalizations and cardiovascular death, as compared to valsartan. This greater benefit was seen in women with HFpEF and in HFpEF patients recently hospitalized for heart failure.

BioNJ Member Pfizer Inc., with offices in Madison, announced the United States (U.S.) Food and Drug Administration (FDA) has approved ABRILADA™ (adalimumab-afzb), as a biosimilar to Humira® (adalimumab), for the treatment of certain patients with rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn's disease, ulcerative colitis and plaque psoriasis. 

Princeton-based BioNJ Member  Agile Therapeutics, Inc.  announced that the  U.S. Food and Drug Administration  ( FDA ) has extended the Prescription Drug User Fee Act (PDUFA) goal date for its review of the New Drug Application (NDA) of Twirla® (levonorgestrel/ethinyl estradiol) transdermal system, an investigational combined hormonal contraceptive patch, from  November 16, 2019  to  February 16, 2020 .

South Plainfield-based BioNJ Member   PTC Therapeutics, Inc.  announced the launch of a no-cost testing program for aromatic L-amino acid decarboxylase (AADC) deficiency, a rare genetic condition. Children with AADC deficiency fail to thrive and commonly don't reach developmental milestones that include being able to hold up their head, sit unassisted, and stand. These patients also have muscular hypotonia, severe seizure-like episodes that include oculogyric crises, and the need for intensive life-long care.

Hampton-based  BioNJ Member   Celldex Therapeutics, Inc.  announced that a Phase 1a study of CDX-0159 has initiated in healthy volunteers. CDX-0159 is a humanized monoclonal antibody that specifically binds the KIT receptor and potently inhibits its activity. The KIT receptor tyrosine kinase is expressed in a variety of cells, including mast cells. In certain inflammatory diseases, such as chronic idiopathic urticaria (CIU), also known as chronic spontaneous urticaria (CSU), mast cell degranulation plays a central role in the onset and progression of the disease.

Plainsboro-based BioNJ Member   Novo Nordisk A/S and Dicerna™ Pharmaceuticals, Inc. announced an agreement to discover and develop novel therapies for the treatment of liver-related cardio-metabolic diseases using Dicerna's proprietary GalXC™ RNAi platform technology. The collaboration plans to explore more than 30 liver cell targets and may deliver multiple clinical candidates for disorders including chronic liver disease, non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity, and rare diseases. 

Princeton-based BioNJ Member Bristol-Myers Squibb announced the initiation of a new randomized, controlled study, GUARD-AF (ReducinG stroke by screening for UndiAgnosed atRial fibrillation in elderly inDividuals). The study seeks to determine if earlier detection of atrial fibrillation (AFib) through screening in previously undiagnosed men and women at least 70 years of age in the U.S. ultimately impacts the rate of stroke, compared to usual standard medical care. 

Kenilworth-based BioNJ Member  Merck & Co. announced that the European Commission has approved KEYTRUDA, Merck's anti-PD-1 therapy, as monotherapy or in combination with platinum and 5-fluorouracil (5-FU) chemotherapy, for the first-line treatment of patients with metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 (combined positive score [CPS] ≥1). This approval allows marketing of the KEYTRUDA monotherapy and combination regimen in all 28 EU member states plus Iceland, Lichtenstein and Norway.

Kenilworth-based BioNJ Member  Merck & Co. announced that the Phase 3 VICTORIA study evaluating the efficacy and safety of Vericiguat, a soluble guanylate cyclase (sGC) stimulator being developed to treat patients with worsening chronic heart failure, has met the primary efficacy endpoint. Vericiguat reduced the risk of the composite endpoint of heart failure hospitalization or cardiovascular death in patients with worsening chronic heart failure with reduced ejection fraction compared to placebo when given in combination with available heart failure therapies.

Kenilworth-based BioNJ Member  Merck & Co. and AstraZeneca announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) and granted priority review for the MEK 1/2 inhibitor selumetinib as a potential new medicine for pediatric patients aged three years and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PNs). This is the first acceptance of a regulatory submission for an oral MEK 1/2 monotherapy for patients with NF1, a rare and incurable genetic condition. 

Bridgewater-based  BioNJ Member  Sanofi's new indication for Dupixent® (dupilumab) in chronic rhinosinusitis with nasal polyposis (CRSwNP) was approved by The European Commission (EC). Dupixent is indicated as an add-on therapy with intranasal corticosteroids for the treatment of adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control. CRSwNP is a chronic disease of the upper airway that obstructs the sinuses and nasal passages.

Bridgewater-based BioNJ Member Allergan plc announced the U.S. Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) Designation and Fast Track Designation for ATM-AVI (aztreonam and avibactam), for the treatment of complicated intra-abdominal infections (cIAI), complicated urinary tract infections (cUTI), and hospital-acquired bacterial pneumonia (HABP)/ventilator-associated bacterial pneumonia (VABP). ATM-AVI is an investigational, fixed-dose, intravenous combination antibiotic under development globally.

A new interim analysis of three-year data from the EMPagliflozin compaRative effectIveness and SafEty (EMPRISE) real-world study on effectiveness shows that Bridgewater-based BioNJ Member   Eli Lilly and Company's  Jardiance® (empagliflozin) was associated with a decreased risk of hospitalization for heart failure and a similar risk of non-fatal atherosclerotic cardiovascular events compared with DPP-4 inhibitors and GLP-1 receptor agonists. The interim analysis included 190,000 adults in the U.S. with type 2 diabetes with and without cardiovascular disease. 

Florham Park-based  Shionogi & Co., Ltd. announced the U.S. Food and Drug Administration (FDA) has approved FETROJA ®  (cefiderocol) for patients 18 years of age or older who have limited or no alternative treatment options, for the treatment of complicated urinary tract infections (cUTI), including pyelonephritis, caused by the following: susceptible Gram-negative microorganisms: Escherichia coli Klebsiella pneumoniae , Proteus mirabilis , Pseudomonas aeruginosa  and Enterobacter cloacae  complex.

Parsippany-based  The Medicines Company announced detailed results from ORION-9, the last of three pivotal 18-month low-density lipoprotein cholesterol (LDL-C) lowering Phase 3 clinical studies of inclisiran, an investigational twice-yearly therapy to reduce LDL-C and the first and only cholesterol-lowering treatment in the siRNA (small-interfering RNA) class. In ORION-9, twice-yearly dosing (following initial and three-month doses) with inclisiran sodium 300 mg met all primary and secondary efficacy endpoints, was well-tolerated and demonstrated an excellent safety profile. 

Woodcliff Lake-based  Eisai Co., Ltd. announced that it has launched the Equfina ®  50mg TABLETS (safinamide mesilate, "Equfina") for the indication of improvement of wearing-off phenomenon in patients with Parkinson's disease under treatment with a drug containing levodopa in Japan. Manufacturing and marketing approval of Equfina were obtained on September 20, 2019, and Equfina was included to Japan's National Health Insurance Drug Price List on November 19, 2019.

Bridgewater-based  Foamix Pharmaceuticals Ltd.  announced the completion of Phase 2 clinical trial enrollment of FCD105 for the treatment of moderate-to-severe acne vulgaris. The trial will evaluate the efficacy and safety of the investigational topical combination foam, which comprises minocycline 3% and adapalene 0.3%. "We are truly grateful to participants, caregivers and clinical centers for their commitment and diligence in enrolling this trial so rapidly. We look forward to completing the remainder of this study and we now expect to report topline data in the second quarter of next year," said  David Domzalski , Chief Executive Officer of Foamix."

Monmouth Junction-based Kadmon Holdings, Inc. announced positive topline results from the planned interim analysis of ROCKstar (KD025-213), the fully enrolled pivotal trial evaluating KD025 in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. The trial met the primary endpoint of Overall Response Rate (ORR) at the interim analysis, which was conducted as scheduled two months after completion of enrollment.

Bedminster-based  Aerie Pharmaceuticals, Inc.  announced the signing of an agreement for the acquisition of Avizorex Pharma, S.L., a Spanish ophthalmic pharmaceutical company developing therapeutics for the treatment of dry eye disease. AVX completed a Phase 2a study in dry eye subjects earlier this year with its lead product candidate, AVX-012. The active ingredient in AVX-012 is a potent and selective agonist of the TRPM8 ion channel, a cold sensor and osmolarity sensor that regulates ocular surface wetness and blink rate. 

Warren-based Aquestive Therapeutics, Inc. announced that it granted a license to Zambon S.p.A. for the development and commercialization of Riluzole Oral Film (ROF) in the European Union for the treatment of amyotrophic lateral sclerosis (ALS). Zambon is a multinational pharmaceutical company strongly committed to innovating cure and care to make patients' lives better, with a focus on the central nervous system (CNS) therapeutic area.

Warren-based  Bellerophon Therapeutics, Inc.  announced positive initial data from an acute, dose escalation, clinical study (PHPF-002) of INOpulse® for the treatment of Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD). PHPF-002 is an ancillary study to the Company's ongoing double-blind, placebo-controlled, randomized, Phase 2/3 iNO-PF study of INOpulse for the treatment of PH-ILD, for which the company expects to report top line results for Cohort 2 by the end of the year.     

Paramus-based SK Life Science, Inc. announced The Lancet Neurology has published results from a multicenter, double-blind, randomized, placebo-controlled, dose-response study of the safety and efficacy of cenobamate, an investigational anti-epileptic drug (AED), in adults with uncontrolled focal (partial-onset) seizures. The results of the study demonstrated that adjunctive treatment with cenobamate produced significant reductions in seizures compared to placebo. 

BrainStorm Cell Therapeutics, Inc.,  with offices in Hackensack,  announced that the company has received a  $495,330  grant from the  National Multiple Sclerosis Society , through its Fast Forward program, to advance BrainStorm's Phase 2 open-label, multicenter clinical trial of repeated intrathecal administration of NurOwn® (autologous MSC-NTF cells) in participants with progressive Multiple Sclerosis ( NCT03799718 ).  

Hamilton-based Genesis Drug Discovery & Development announced it has acquired New England Discovery Partners, a contract research organization specializing in synthetic and medicinal chemistry. The acquisition of NEDP's chemistry service portfolio will provide GD3 the ability to expand on its pre-clinical drug research and discovery services. "We're committed to building an industry-leading, fully integrated drug discovery CRO," Dr. Eli Mordechai, CEO of GBG, said. "Adding NEDP is another way to differentiate our capabilities in the early drug discovery space."

People in the News

RenovaCare, Inc., with offices in Hackensack,  announced the appointment of Alan L. Rubino as President and Chief Executive Officer. Mr. Rubino will lead the company through its next stages of clinical and corporate development and financial growth. Mr. Rubino is recognized for his many accomplishments in major pharmaceutical and small public specialty companies, and industry board positions, that include therapeutics across a broad range of disease areas, including medical devices to accelerate wound healing and tissue regeneration therapies for burns using innovative stem cell technologies.

Hackensack Meridian Health Hackensack University Medical Center announced that  Andrew L. Pecora, M.D., FACP, CPE, a nationally recognized hematologist/oncologist, presented two abstracts at the prestigious  Connective Tissue Oncology Society in  Tokyo, Japan"It is an honor to present our cutting-edge work to the world's leading sarcoma specialists," said Dr.  Pecora.  "We believe our findings, if confirmed in large trials, could lead to a transformation in clinical practice. The John Theurer Cancer Center at Hackensack University Medical Center is among the first to demonstrate that immunotherapy can result in durable remissions and potentially prolong life for patients with sarcoma who are resistant to all standard therapy."

Princeton-based  Otsuka Pharmaceutical Development & Commercialization Inc. (OPDC) announced that effective Jan. 1, 2020, President and Chief Executive Officer Dr. William Carson will retire from the company's operational leadership. Dr.  Carson will transition to Chairman of the OPDC Board of Directors, as well as spearhead Otsuka philanthropic initiatives to be announced in 2020. "Bill is a beloved leader at Otsuka and has been a driving catalyst behind the company's rapid growth in the years following Otsuka's establishment of a development organization in the U.S. more than 30 years ago," said Kabir Nath, President and CEO, North America Pharmaceutical Business.

Bridgewater-based BioNJ Member Insmed Inc. announced it has appointed Roger Adsett as its Chief Operating Officer, effective immediately. Mr.  Adsett, who most recently served as the company's Chief Commercial Officer, will be responsible for those duties as well as Insmed's overall business operations. He will report to Will Lewis, CEO and Chairman of the board. The company also promoted Drayton Wise to Senior Vice President, Head of United States.

Ernst & Young  announced it has named John F. Thero, CEO and President of  Amarin Corp. , its 2019 Entrepreneur of the Year for Life Sciences. The awards program, founded in 1986, recognizes entrepreneurs "who create the products and services that keep our worldwide economy moving forward and redefine the way we live, work and play," EY said in a news release. "This award is a great honor, especially in light of the many outstanding people, and the achievements of their companies, that were considered," Mr. Thero said. "The award is a tribute to the significant pioneering progress made by Amarin's dedicated employees in advancing cost-effective preventative care solutions for reducing the risk of cardiovascular disease, which is on the rise in the United States."

Somerset-based  Catalent announced it has appointed Mike Grippo as Senior Vice President, strategy and corporate development. Mr.  Grippo will be tasked with identifying shareholder growth opportunities and various corporate development functions. He will report to John Chiminsky, Chair and CEO of Catalent. Prior to his appointment, Mr. Grippo joined Catalent as Vice President of Corporate Affairs in 2016. Before that, he was Vice President of Corporate Development for CR Bard. Prior to CR Bard, he was in similar roles at Hill-Rom Holdings. 

Institution and Education News

Governor Phil Murphy announced the creation of the New Jersey STEM Strategic Advisory Board that was developed by the New Jersey STEM Pathways Network (NJSPN). This inaugural Board was launched by the New Jersey STEM Pathways Network, an initiative of the Research & Development Council of New Jersey, and will provide leadership and expertise for a statewide STEM ("Science, Technology, Engineering and Math") vision in education and workforce. It also plans to fund innovative STEM programs with data-driven results. The Board includes business and philanthropic leaders and will seek to continue to grow over the course of the year.

More than $2.3 million in Research Scholar Grants from the American Cancer Society have been awarded to three Rutgers Cancer Institute of New Jersey researchers to examine cell metabolism and tumor formation in forms of breast cancer, lung cancer and leukemia. Grants of of $792,000 each were awarded to Michael Gatza, Yanxiang Jessie Guo and Daniel HerranzBenito, who also all serves as faculty members at Rutgers Robert Wood Johnson Medical School. 

A Rutgers-led team has created better biosensor technology that may help lead to safe stem cell therapies for treating Alzheimer's and Parkinson's diseases and other neurological disorders.  The technology, which features a unique graphene and gold-based platform and high-tech imaging, monitors the fate of stem cells by detecting genetic material (RNA) involved in turning such cells into brain cells (neurons) and characterizes different kinds of stem cells with greater reliability, selectivity and sensitivity, according to a  study  published in the journal Nano Letters.

Deborah Heart and Lung Center announced it has received a $5 million donation from Gregory Olsen, the largest single gift donation in its 97-year history. The funds will go to Deborah100: The Campaign, a multiyear initiate to add new patient floors all with private inpatient suits to the Browns Mills-based specialty cardiac, pulmonary and vascular hospital. "Deborah has long stood at the forefront of ground-breaking medical care," Joseph Chirichella, CEO and President of Deborah Heart and Lung Center, said. "But we have also stood for compassion and kindness through our philosophy that 'there's no price on life'. We are thankful for Dr. Olsen's gift that will further elevate our care in the region."

Special Programs from Our Partners for BioNJ Members

December 3-4, 2019 | Boston

Meet, interact and network with more than 800 VCs, corporate VCs, angel investors, investment bankers and founders of venture backed, emerging and early stage companies at the New England Venture Summit being held on December 4 at The Hilton, Dedham, MA. BioNJ Members enjoy a registration discount. Contact Randi Bromberg at RBromberg@BioNJ.org to learn more.  Register now at http://bit.ly/2MutkwR.

December 5, 2019 |  Rutgers University,  Piscataway

Save the date to join Reynold Panettieri, Vice Chancellor for Translational Medicine and Science, for an event from the New Jersey Alliance for Clinical and Translational Science (NJ ACTS), a clinical and translational science consortium led by Rutgers University, with partnership from Princeton University and New Jersey Institute of Technology. 

December 10, 2019  | Webinar

Join this free webinar to learn more and understand how the favourable regulatory pathway works in Australia. The featured speakers will draw upon their experience with U.S. biopharmaceutical companies and will discuss the strategies being followed by U.S. biopharmaceutical companies in planning their first in human studies, which are ultimately accelerating their drug development pathway.

December 19, 2019  | Galloping Hill Golf Course, Kenilworth

This is the 22nd year for the New Jersey Chapter of the Licensing Executives Society to host his holiday event. Click here to register. The New Jersey Chapter uses online registration for members and non-members. Members should login to the registration page with their LES online account.

January 14, 2020 | Allentown High School

Showcase your college/university, technical program, military service branch and more! Set up a table or demonstration!  This year's event will be held during the school day to enable all students to explore the variety of careers offered to them within Science, Technology, Engineering & Math (STEM).  Please RSVP to Dale Cruzan at cruzand@ufrsd.net or 609-259-7292 ext. 1443 by December 16, 2019. 

BioNJ Member Services Provider Directory

BioNJ recognizes the vital role that our Service Provider Members play in the growth of New Jersey's robust life sciences ecosystem. For this reason, we are delighted to remind our community about BioNJ's Member Service Provider Directory, a categorical listing of BioNJ Members by service sector. By working with fellow BioNJ Member organizations, you are not only supporting our community, but benefiting from a wide variety of high quality organizations.  To learn how you can become a Member and be featured on this resource tool, please contact Kim Minton at KMinton@BioNJ.org, or 609-890-3185. Find providers in these categories:

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 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.

Please contact BioNJ at BioNJ@BioNJ.org or call 609-890-3185 
with any questions.