Welcome to the latest edition of the  BioLines Weekender

The BioNJ Team has been busy putting finishing touches on next week's Annual Dinner Meeting and Innovation Celebration, taking place Thursday, February 7, at the Hilton East Brunswick. We hope you will join us for what promises to be an exciting evening, including an opening address by New Jersey Governor Phil Murphy and the presentation of the Dr. Sol J. Barer Award for Vision, Innovation and Leadership to Dr. William N. Hait by Dr. Barer himself. Click here to register.

Additionally, since the last edition of the Weekender, we...
  • Attended a briefing meeting with the Governor's office on the Evergreen Innovation Fund
  • Participated in Higher Education Task Force Marketing Branding sub-committee meetings
  • Provided an overview of the New Jersey life sciences ecosystem, as well as BioNJ, to a Japanese delegation representing Japan's National Institute in Basic Biology at Princeton University
  • Hosted BioNJ's Public Policy Committee where Asm. Chris DePhillips (LD40) spoke on his perspective for the life sciences sector in 2019
  • Attended and co-supported a Biotech Fireside Chat with Genewiz's Founder Dr. Steve Sun at Princeton BioLabs
  • Supported Amicus Therapeutics' Senior Vice President of Global Regulatory Affairs Dr. Andrew Mulberg who testified before the NJ Assembly Science, Innovation and Technology Committee on the status of the innovation economy
  • Announced a free Webinar on February 20 about BioNJ's Member-only Health Benefits Plan, a cost-effective health benefits solution to the New Jersey life sciences ecosystem (Click here to register)
We look forward to seeing you next Thursday, February 7! Because Patients Can't Wait ®!!

Because Patients Can't Wait®,
The BioNJ Team
The Value of Medical Innovation Featured in Times Square

Last week BioNJ featured digital billboards in high-traffic areas of New York City featuring messaging on the value of medical innovation and thanking the biopharmaceutical industry for bringing new therapies, cures and hope to Patients and their loved ones around the world. Take a look by clicking on each image. 

BioNJ Calendar

BioNJ's Annual Dinner Meeting & Innovation Celebration
Hilton East Brunswick, East Brunswick, NJ
February 7, 2019
BioNJ Health Benefits Plan
February 20, 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.

Celgene announced it will combine with Bristol-Myers Squibb, creating an innovative global biopharma leader.  This combination will enable Celgene to create greater impact for the patients who rely on our therapies, opportunities for our people, and value for our shareholders. Together with Bristol-Myers Squibb, Celgene will have leading franchises and a deep and broad pipeline-driving sustainable growth and delivering new options for patients with cancer, inflammatory and immunologic disease and cardiovascular disease.

As states continue to grapple with prescription drug costs, a new Pioneer Institute study lays out the key ethical, methodological and disease-specific questions policy makers should address before deciding whether to contract with the Institute for Clinical and Economic Review (ICER) to conduct cost effectiveness reviews used to make decisions about the purchase of medicines and other medical innovations.

The IQVIA Institute for Human Data Science released a new report that reveals important realities about U.S. drug pricing trendsToo many politicians and journalists remain committed to the false narrative of "skyrocketing" and "soaring" drug prices. By contrast, IQVIA data reveal that list prices for brand-name drugs grew by less than 6% in 2018. 

In Medicare Part D, the six protected classes policy protects vulnerable seniors and low-income beneficiaries with serious and complex health conditions, while also allowing Part D insurance plans to use the tools they need to control costs. Medicines for some of the sickest patients in Part D are covered within the six protected classes, including those for cancer, epilepsy, HIV/AIDS and mental illness.

Showing Our New Jersey Pride

A Newsletter by Choose New Jersey
We have our sights set across the Atlantic and our passports in tow to spread the message: New Jersey is open for international business. The Choose New Jersey team is hard at work planning to attend over 25 events both in the States and abroad in 2019, including the potential for another business attraction mission to Europe in the Spring with Governor Murphy.

BioNJ in the News

I was privileged to participate in a recent Patient Advocacy Summit, a remarkable event organized by BioNJ. We heard from many impassioned patients, parents, advocates and supporters who shared their individual stories -- each touching and powerful -- with an unmistakable common thread of optimism and determination. Their commitment to change, progress, and success is inspirational, and for all of us who are lucky to work in patient engagement -- or patient involvement -- these testimonies make it very clear what matters.

Tracy Fink asked the business professionals attending  BioNJ 's Inspiring Women in STEM Conference in December to speak with a partner on what they love and are challenged by in their careers. "When 'listening,' were you also ruminating about the past? Or worrying about the future?"   As founder and CEO of  The Tortoise Institute  in East Brunswick, Fink said she employs science-based practices, one-on-one coaching and leadership development techniques to increase businesses' focus on the relationship between leadership and well-being.  

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.

Princeton-based BioNJ Member Soligenix, Inc. announced that the European Patent Office has granted the patent entitled "Novel Peptides for Treating and Preventing Immune-Related Disorders, including Treating and Preventing Infection by Modulating Innate Immunity."  The newly issued patent claims composition of matter of innate defense regulator (IDR) analogs, expanding patent protection around the company's lead IDR, dusquetide.

BioNJ Member OrthogenRx, Inc. announced the launch of TriVisc® (sodium hyaluronate) for the treatment of patients with osteoarthritic (OA) knee pain who have failed to get adequate relief from simple painkillers or from exercise and physical therapy. TriVisc is a solution of highly purified hyaluronic acid (sodium hyaluronate; HA) and helps supplement the viscous properties of the fluid in the knee joint.

Jersey City-based BioNJ Member  SCYNEXIS, Inc.  announced positive results from the first interim efficacy analysis of the ongoing FURI study. FURI is a Phase 3 open-label study evaluating oral ibrexafungerp as a salvage treatment in patients with difficult-to-treat mucocutaneous and invasive fungal infections that are refractory to or intolerant of currently available standards of care.

When Dr. Marco Taglietti became Chief Executive Officer of Jersey City-based BioNJ Member SCYNEXIS, Inc. in April 2015, he knew that he wanted to move the publicly-traded biopharmaceutical company from North Carolina to a hub of biotechnology activity along the Northeast Corridor. He selected Jersey City because of the Garden State's connectivity, deep talent pool, and collaborative ecosystem.

Newark-based BioNJ Member Rafael Pharmaceuticals, Inc. announced the initiation of a Phase II open-label trial in collaboration with University Hospitals Cleveland Medical Center to evaluate the efficacy and safety of investigational drug CPI-613 in combination with modified FOLFIRINOX (mFFX) in patients with localized borderline and unresectable pancreatic cancer. The primary objective of this study is to determine the potential increase in overall survival (OS) when CPI-613 is administered in combination with mFFX and to further assess the safety of the CPI-613+ mFFX combination. 

Newark-based BioNJ Member Rafael Pharmaceuticals, Inc.announced that the European Medicines Agency (EMA) has granted orphan drug designation to devimistat (CPI-613®), the Company's lead Altered Metabolism Directed (AMD) platform drug candidate, for the treatment of Metastatic Pancreatic Cancer. Devimistat is a first-in-class drug developed based on Rafael's Altered Metabolism Directed (AMD) platform. 

Newark-based BioNJ Member Rafael Pharmaceuticals, Inc. announced the initiation of patient enrollment for a phase II clinical trial of CPI-613 for patients with relapsed or refractory Burkitt Lymphoma/Leukemia. In June 2018, CPI-613 received orphan drug designation for treatment of Burkitt Lymphoma from the US FDA.

Kenilworth-based BioNJ Member  Merck & Co. announced that V114, the company's investigational 15-valent pneumococcal conjugate vaccine, has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of invasive pneumococcal disease (IPD) caused by the vaccine serotypes in pediatric patients 6 weeks to 18 years of age. V114 is also under development for the prevention of IPD in adults. Both indications are currently being studied in Phase 3 clinical trials.

Kenilworth-based BioNJ Member  Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has accepted for review supplemental New Drug Applications (sNDAs) for PIFELTRO™ and DELSTRIGO™. The applications seek approval for PIFELTRO (in combination with other antiretroviral medicines) and DELSTRIGO for use in people living with HIV-1 who are switching from a stable antiretroviral regimen and whose virus is suppressed (HIV-1 RNA <50 copies/mL).

Bridgewater-based BioNJ Member Eli Lilly and Company announced that the  U.S. Food and Drug Administration  has granted approval for a new indication for ALIMTA ® (pemetrexed for injection) in combination with KEYTRUDA ® (pembrolizumab), developed and marketed by BioNJ Member Merck & Co. , and platinum chemotherapy for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

Bridgewater-based BioNJ Member Eli Lilly and Company  announced that results from the global Phase 3 REACH-2 study of CYRAMZA ®  (ramucirumab) as a single agent in the second-line treatment of people with AFP-High (alpha-fetoprotein ≥400 ng/mL) hepatocellular carcinoma (HCC) were published in  The Lancet OncologyREACH-2 showed a statistically significant improvement in the primary endpoint of overall survival (OS) as well as in the secondary endpoint of progression-free survival (PFS).

Bridgewater-based BioNJ Member Eli Lilly and Company and BioNJ Member Pfizer, with offices in Madison,  announced positive top-line results from a Phase 3 study evaluating tanezumab 2.5 mg or 5 mg in patients with moderate-to-severe osteoarthritis (OA) pain. The tanezumab 5 mg treatment arm met all three co-primary endpoints at 24 weeks, demonstrating a statistically significant improvement in pain, physical function and the patients' overall assessment of their OA compared to those receiving placebo. 

The U.S. Food and Drug Administration has approved the use of the 0.5 mL dose of Fluzone® Quadrivalent (influenza vaccine) to include children age 6 through 35 months. Sanofi Pasteur, the vaccine division of Bridgewater-based BioNJ Member  Sanofi, will have the 0.5 mL dose, in addition to the 0.25 mL dose, available for the upcoming 2019-20 season for this expanded age range.

Parsippany-based BioNJ Member Ferring Pharmaceuticals and Karolinska Institutet announced a five-year extension of their collaboration to explore the potential of the human microbiome in reproductive medicine and women's health and gastroenterology. The collaboration brings together specialist expertise from Karolinska Institutet in early stage research, Rebiotix Inc. (acquired by Ferring in 2018), a late-stage clinical microbiome company and Ferring's therapeutic area and commercialization capabilities.

Summit-based BioNJ Member Celgene has signed on to a deal worth about $1 billion for a Canadian preclinical asset: a first-in-class small molecule that targets protein-protein interactions and epigenetic regulation in leukemia and lymphoma. The Big Biotech will pay $40 million upfront for the option to license TRPH-395, while committing to an additional $940 million or more in future R&D and sales milestone payments to Triphase Accelerator, a drug development company that looks to bring the compound through Phase 2 proof of concept.

Princeton-based BioNJ Member Evotec AG  announced that it has earned a $2M milestone payment from Second Genome, Inc., a leading microbiome science-based discovery & development company, for the initiation of a Phase II clinical study of SGM 1019, a first-in-class oral therapeutic for the treatment of nonalcoholic steatohepatitis (NASH). The Phase II trial of SGM-1019 has been initiated following the completion of two successful Phase I trials.

South Plainfield-based BioNJ Member  PTC Therapeutics, Inc.  launched PRIORITY, an annual funding program for innovative research. In its inaugural year, PRIORITY will fund programs designed to improve Duchenne screening in infants and support earlier diagnosis of this progressive, muscle-wasting disease. The 2019 PRIORITY program invites funding proposals from clinicians in the fields of neurology, genetics and pediatrics for submission between now and  March 31 , 2019. 

South Plainfield-based BioNJ Member  PTC Therapeutics, Inc.  announced that it approved non-statutory stock options to purchase an aggregate of 170,050 shares of its common stock to 24 new employees. The awards were made pursuant to the NASDAQ inducement grant exception as a component of the new hires' employment compensation. The inducement grants were approved by PTC's Compensation Committee and are being made as an inducement material to each employee's acceptance of employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4).

Bedminster-based  Aerie Pharmaceuticals, Inc.  announced that the  U.S. Food and Drug Administration   has reviewed the Investigational New Drug Application (IND) for AR-1105 (dexamethasone intravitreal implant) and it is now in effect, allowing Aerie to initiate human studies in the treatment of macular edema due to retinal vein occlusion (RVO). The IND was submitted in  December 2018 . Aerie expects to initiate a Phase 2 clinical study later in the first quarter of 2019.

Warren-based GlaxoSmithKline plc announced that it has successfully completed the acquisition of TESARO, Inc. for an aggregate cash consideration of approximately $5.1 billion (£4.0 billion). The transaction significantly strengthens GSK's pharmaceutical business, accelerating the build of GSK's pipeline and commercial capability in oncology. TESARO is a commercial-stage biopharmaceutical company, with a major marketed product, Zejula (niraparib), an oral poly ADP ribose polymerase (PARP) inhibitor currently approved for use in ovarian cancer.

Freehold-based Avalon GloboCare Corp. announced the discovery and development of the world's first saliva-based exosomal microRNA biomarker, miR-185, as a dual diagnostic and therapeutic target for oral cancer. Oral leukoplakia, with a prevalence of 2% affecting the worldwide population, is a pre-cancerous lesion that confers increased risk for the development of oral cancer.

Hackensack-based BrainStorm Cell Therapeutics Inc.  announced that the Israel Patent Office has granted an additional patent titled "Methods of Generating Mesenchymal Stem Cells which Secrete Neurotrophic Factors." The allowed claims cover the method of manufacturing the cells, the cells generated according to the method of manufacturing and the use of the cells for preparation of a therapy for treating a disease (including a neurodegenerative disease, a neurological and an immune disease).

Jersey City-based  TrialScope  announced a strategic partnership with Washington D.C-based  Vivli , a nonprofit focused on sharing of clinical trial data. The collaboration would offer sponsors and researchers access to the most complete solution for disclosure and data sharing. "There is a growing trend towards increased transparency within the clinical trial industry, not just to meet regulatory requirements, but as an ethical obligation to advance science and promote patient safety," said TrialScope CEO Jeff Kozloff.

Warren-based  Bellerophon Therapeutics, Inc.  announced that data from the company's ongoing INOpulse® Phase 2b clinical trial (iNO-PF) for the treatment of Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD) that supports actigraphy as a clinically meaningful endpoint will be the subject of a poster presentation at the 13th Annual  Pulmonary Vascular Research Institute World Congress on Pulmonary Vascular Disease .

Warren-based Bellerophon Therapeutics, Inc.  announced the closing and funding of its previously announced underwritten public offering of 10,000,000 shares of its common stock at a public offering price of  $0.70  per share, less underwriting discounts and commissions. The company has also granted the underwriter a 45-day option to purchase up to an additional 1,500,000 shares of its common stock at the public offering price per share, less underwriting discounts and commissions.

People in the News

Jersey City-based BioNJ Member SCYNEXIS, Inc. announced the appointment of Armando Anido to its Board of Directors. Mr. Anido has served as Chairman and Chief Executive Officer of Zynerba Pharmaceuticals since October 2014. He has more than 30 years of executive, operational and commercial leadership experience in the biopharmaceutical industry.

Kenilworth-based BioNJ Member Merck & Co. announced that Mary Ellen Coe, President, Google Customer Solutions, at Google Inc. will join the company's Board of Directors effective March 18, 2019. Ms. Coe will stand for election with the company's other directors in connection with Merck's Annual Meeting of Shareholders on May 28, 2019. With the addition of Ms. Coe, the Merck board will include 13 members.

Bridgewater-based Foamix Pharmaceuticals Ltd.  announced the appointment of  Sharon Barbari  to its Board of Directors. "We welcome Sharon to Foamix and are confident that she will add significant value as a board member. She brings extensive executive leadership and diverse board level experience in the pharma and biotech industries," said Dr.  Stanley Hirsch , Chairman of the Board of Foamix. 

Bridgewater-based  Amneal Pharmaceuticals Inc. announced it has appointed Todd P. Branning as Senior Vice President and Chief Financial Officer. In this role, Mr. Branning will be responsible for leading financial strategies, including overseeing accounting, internal audit, investor relations and corporate communications and information technology.

Ortho Clincal Diagnostics, a Raritan-based diagnostics company, announced it has named Robert Yates as Chairman and CEO, effective Feb. 12. Mr.  Yates will succeed Dr. Martin Madaus, who is retiring after being chairman and CEO since June 2014. Mr. Yates, who is the current President at Ortho and has served as Director of the company's board since June 2014, previously held several leadership roles at Ortho, including as its Chief Operating Officer, President and Executive Advisor on Strategy, Partnerships and M&A. 

Institution and Education News

Montclair State University  is partnering with  Princeton University Rowan University  and STEM education nonprofit  Math for America  to implement the New Jersey STEM Innovation Fellowship, giving New Jersey public school teachers the opportunity to improve STEM teaching.  "New Jersey has some of the best public school teachers in the nation, who are committed to helping students reach their greatest potential. This Math for America pilot will help teachers implement new, STEM-based teaching practices and will enable them to further provide our children with the skills they need to prosper in the innovation economy," Gov. Phil Murphy said in a statement.  

Rutgers Cancer Institute of New Jersey researcher Dr. Sharon Pine received a $200,000 Lung Cancer Discovery Award from the  American Lung Association to examine potential new treatment options for patients with a sub-type of non-small cell lung cancer. Dr.  Pine's goal is to test a novel way to treat squamous cell carcinomas that have a mutation in the gene known as PIK3CA. A mutation in PIK3CA is believed to be the "driver" of this type of non-small cell lung cancer tumor.

Scientists at  Rutgers University and universities in Russia, Poland and England have unraveled a 30-year mystery on how the molecular machinery works in an enzyme that makes a potent antibiotic.  The findings pr ovide the tools to design new antibiotics, anticancer drugs and other therapeutics. In a statement, Rutgers said that the potent natural antibiotic, microcin B17, kills harmful E. coli bacteria. Microbial resistance to antibiotics -- due to their overuse and misuse -- is one of the biggest threats facing humanity, and there is an urgent need to find new drugs. 

Researchers at  Rutgers University have discovered the reason why many people with joint replacements experience harmful inflammation and bone loss.  The findings could pave the way for new therapies to reduce pain and prevent the need for follow-up surgery. In a statement Rutgers reported that as many as 15 percent of joint replacements will fail, often requiring revision surgery to replace the implant. 

Hackensack, NJ John Theurer Cancer Center at Hackensack Meridian Health Hackensack University Medical Center announced that it is leading enrollment worldwide for WIZARD 201G, a global Phase 2 study in glioblastoma (GBM). The trial, sponsored by Boston Biomedical, is evaluating DSP-7888, an investigational cancer vaccine, in combination with bevacizumab for recurrent disease.

Seton Hall University  has been named a "High Research Activity" institution among Doctoral Universities in the latest Carnegie Classification. The new research level designation, also known as "R2," places Seton Hall among only 139 universities across the country designated as "High Research."  Prior to this designation as R2, Seton Hall was classified as a "Moderate Research Activity" or "R3" university, placing it among the top ten percent of all universities classified, but in the third tier.

Hackensack Meridian Health announced that it is funding the development of new technology that uses a form of ultraviolet light energy to keep hospitals clean and safe.  "Innovation is in our DNA at Hackensack Meridian Health and we are proud to fund this exciting technology which will ultimately add to our robust strategies to keep our patients safe and healthy'' said Robert C. Garrett, CEO of Hackensack Meridian Health.

Funding News

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

February 19, 26 2019 | Newark, East Hanover

The mission of Students 2 Science is to "inspire, motivate, and educate elementary, middle and high school students to pursue careers in science, technology, engineering and math (STEM subjects)." This social and open house will be held on Tuesday, Feb.19, from 3-6 p.m. in the Newark Technology Center, 765 Broad St., Newark, 07102. It is a "free, fun and casual event" open to anyone 21 and older who would like to learn more about the organization and how to get involved. RSVP to Cyndi Mayer, Director of Volunteer Recruitment and Corporate Relations, 862-777-4237.

February 20, 2019 | Newark, DE

The Science & Technology Career Fair connects employers with exemplary candidates in search of new career opportunities. Organizations looking to fill full-time, part-time and internship roles are encouraged to exhibit. This event traditionally attracts over 350 scientists and technologists with a broad range of educational and technical expertise. For more information, visit https://delawarebio.org/events/career-fair/.

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.