Welcome to the latest edition of the  BioLines Weekender

As we move toward the end of 2019, we would like to wish all our Members Happy Holidays and a prosperous New Year. 
2019 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 more than 20 novel FDA drug approvals from companies with a footprint here.
Entrepreneurship is key to the growth of the ecosystem. BioNJ recently announced our Third Entrepreneurship Fellowship program and is looking for companies to host a Fellow. Please click here to learn more. 

Because Patients Can't Wait®, please join us as we celebrate the innovation coming from New Jersey's life sciences community during   BioNJ's 27th Annual Dinner Meeting & Innovation Celebration , taking place February 6, 2020 at the Hilton East Brunswick. Bringing together 700 biopharma professionals, you'll enjoy an evening of networking, business discussions and award presentations -- including the presentation of the Dr. Sol J. Barer Award to Chairman of the Board and Chief Executive Officer of Merck & Co. Kenneth C. Frazier by Dr. Sol Barer himself. Click here for details and to register. 
Finally, if you aren't a BioNJ Member, contact us today at [email protected] to learn about the many benefits of Membership. Please join us in our mission to help our Members help Patients. Thank you. 

Wishing you a very Happy Holiday Season, and a peaceful and prosperous New Year. All the best to you and yours and to Patients the World over.
Because Patients Can't Wait®,
The BioNJ Team
BioNJ Calendar

WEBINAR: A Demo of BioNJ's Talent Services Career Portal
January 14, 2020
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 [email protected]. Plus, click here for valuable tools and resources on the Value of Medical Innovation.

The  House of Representatives passed H.R. 3 in a nearly straight party-line vote last week. While prescription drug pricing reforms are needed, the legislation would unravel many of the market incentives that have made America the undisputed leader bringing new medicines -- and new hope -- to people facing devastating diagnoses.  Bipartisan legislation crafted by the  Senate Finance Committee , and supported by the White House, is the last opportunity Congress has to strike a sensible bargain that lowers drug costs for patients while sustaining America's leadership in this vital field.

The Senate confirmed Dr. Stephen Hahn as the next Food and Drug (FDA) Commissioner yesterday in a 72-18 vote.  Dr. Hahn is leaving his post as Chief Medical Executive at Houston's MD Anderson Medical Center. He is taking over from Dr. Ned Sharpless, who has served as acting commissioner after Dr. Scott Gottlieb resigned earlier this year. Dr. Sharpless will return to his job leading the National Cancer Institute. 

Improving affordability for prescription drugs, especially for seniors, has remained a key priority in Washington for the past year, both on Capitol Hill and with the administration. High out-of-pocket (OOP) exposure for Patients, particularly for those with chronic conditions, can threaten access and adherence and may lead to worsened Patient outcomes. PCSK9 inhibitors, a relatively new class of drugs designed to lower cholesterol levels and reduce the risk of heart attacks and strokes, have encountered challenges with uptake. 

BioNJ in the News

The U.S. House Ways and Means Committee, which oversees the Medicare program, voted in favor of  House Resolution 3, the Elijah E. Cummings Lower Drug Costs Now Act.  This bill establishes several programs and requirements relating to the prices of prescription drugs. In particular, the bill requires the Centers for Medicare & Medicaid Services (CMS) to negotiate prices for certain drugs -- current law prohibits the CMS from doing so. BioNJ President and CEO Debbie Hart said: "Today marks a very disappointing step in the fight to ensure that American patients have the greatest access in the world to lifesaving prescription drugs and biologics. H.R. 3 would decimate investment in the biopharmaceutical industry, reduce Patient access to lifesaving cures and disrupt the wildly successful Medicare Part D program."

For Debbie Hart, President and CEO of  BioNJ, the issues around including more women in STEM can be simplified.  "If one subscribes to the 'We can't be what we can't see' theory, then the opposite is true when BioNJ can help show women at all points in their professional development the pathways and people who can help them discover their potential," she said. Hart was speaking in reference to BioNJ's fifth annual Inspiring Women in STEM Conference, held at Sanofi U.S. headquarters in Bridgewater. More than 100 women with careers in all aspects of science, technology, engineering and mathematics attended.

Dr. Gaurang Daftary, Senior Director of Medical Affairs, Reproductive Medicine and Maternal Health at  Ferring Pharmaceuticals , said it was important for all the women attending  BioNJ 's Inspiring Women in STEM conference to understand how they themselves have been affected by systemic limitations in women's health care. "Women respond differently to diseases and diseases are differently manifested in women -- yet much of the research for available treatments have been based on male models," he said. "We still need more female models in all sorts of clinical trials."

The NJBIZ Manufacturing Power 50 represent  the men and women who have been, and will continue to be, responsible for maintaining and expanding the skilled workforce necessary for a strong Garden State manufacturing sector. In addition to BioNJ President and CEO Debbie Hart, these BioNJ Members made the list:
  • Giovanni Caforio, Bristol-Myers Squibb
  • Frank Danieli, Choose New Jersey
  • Kenneth Frazier, Merck & Co.
  • Julia Lopez, Reed Smith LLP
  • Richard McCardle, Rutgers Food Innovation Center 
  • Brian Sabina, NJEDA
  • Marco Taglietti, SCYNEXIS, Inc.
  • Michael Tardugno, Celsion Corp. 

NJ Company News

Madison-based BioNJ Member Amarin Corporation plc announced that the U.S. Food and Drug Administration (FDA) has approved a new indication and label expansion for VASCEPA® (icosapent ethyl) capsules. After more than a decade of development and testing, VASCEPA is now the first and only drug approved by the FDA "as an adjunct to maximally tolerated statin therapy to reduce the risk of myocardial infarction, stroke, coronary revascularization, and unstable angina requiring hospitalization in adult patients with elevated triglyceride (TG) levels (≥150 mg/dL) and established cardiovascular disease or diabetes mellitus and two or more additional risk factors for cardiovascular disease."

Basking Ridge-based BioNJ Member  Amgen  announced that the  U.S. Food and Drug Administration  ( FDA ) has approved AVSOLA™ (infliximab-axxq) for all approved indications of the reference product, Remicade® (infliximab): for the treatment of moderate-to-severe rheumatoid arthritis (RA), moderate-to-severe Crohn's Disease (CD) in the adult and pediatric population, moderate-to-severe ulcerative colitis (UC) in the adult and pediatric population, chronic severe plaque psoriasis (PsO), psoriatic arthritis (PsA) and ankylosing spondylitis (AS).

Basking Ridge-based BioNJ Member  Amgen  announced additional results from the primary analysis of the Phase 3 CANDOR study evaluating KYPROLIS ®  (carfilzomib) in combination with dexamethasone and DARZALEX ®  (daratumumab) (KdD) compared to KYPROLIS and dexamethasone alone (Kd) in patients with relapsed or refractory multiple myeloma. At a median follow up of 17 months, the study met its primary endpoint of progression-free survival (PFS), resulting in a 37% reduction in the risk of disease progression or death in patients receiving KdD (HR=0.63; 95% CI: 0.464, 0.854; p=0.0014). Median PFS was not reached for the KdD arm versus 15.8 months for the Kd arm.

Pennington-based BioNJ Member OncoSec Medical Incorporated announced interim results from KEYNOTE-890, an ongoing Phase 2 study of TAVO™ (intratumoral IL-12) in combination with KEYTRUDA® (pembrolizumab), an anti-PD-1 checkpoint inhibitor, in patients with metastatic, chemotherapy-refractory triple negative breast cancer (mTNBC). Patients who previously had progressed after an average of three prior lines of chemotherapy were enrolled in the KEYNOTE-890 study to evaluate if the addition of TAVO could provide meaningful clinical activity when combined with KEYTRUDA®.  

Princeton-based BioNJ Member  Soligenix, Inc. announced that it has opened the study titled "A Phase 1C, Double-Blind, Placebo-Controlled, Randomized Study to Evaluate the Safety of RiVax®, a Lyophilized Ricin Toxin A-Chain Subunit Vaccine with Alum-Adjuvant, in Healthy, Normal Adults." Preliminary safety results from the trial are expected in the second quarter of 2020 with longer-term safety and immunogenicity results from throughout the 6-month follow-up period expected in the fourth quarter of 2020.

Madison-based BioNJ Member LEO Pharma A/S  announced that tralokinumab  met all primary and secondary endpoints in its three pivotal Phase 3 studies (ECZTRA 1-3) for the treatment of moderate-to-severe atopic dermatitis (AD) in adults. During the studies, the overall adverse event rate was comparable between tralokinumab and placebo. IL-13 is a key driver of the type 2 inflammation that plays a major role in AD, which is the most common inflammatory skin disease in the developed world, affecting up to five percent of adults across the United States, Canada, Europe and Japan.

Berkeley Heights-based BioNJ-Member  Cyclacel Pharmaceuticals, Inc.  announced study design and preliminary data from two of the company's Phase 1 studies evaluating a combination of CYC065, a CDK2/9 inhibitor, with venetoclax, a BCL2 inhibitor, to treat patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) and chronic lymphocytic leukemia (CLL) respectively. The CYC065-venetoclax combination was well tolerated and no dose-limiting toxicities have been reported. No tumor lysis syndrome was observed. 

Warren -based BioNJ-Member  Celularity, Inc. announced data introducing an extensive platform in genetically-modified NK- and T cell-based programs, and highlighting long-term safety and efficacy results from the Phase 1 study of PNK-007 in patients with multiple myeloma (MM). In pre-clinical studies, Celularity's allogeneic, placental-derived CD19 CAR T cell-based product candidate demonstrated in vivo anti-tumor efficacy in a lymphoma tumor model. 

Parsippany-based BioNJ Member Ferring Pharmaceuticals announced positive results from the pivotal Phase 3 clinical trial evaluating nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy, for the treatment of high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). FKD Therapies Oy (FKD) has led the development and regulatory filing of nadofaragene firadenovec, which has been studied in 33 centers across the U.S. in collaboration with the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). 

Edison-based BioNJ Member  Hepion Pharmaceuticals, Inc. announced the successful advancement to the next higher dose in its ongoing clinical trial of CRV431, a Phase 1 multiple ascending dose ("MAD") study.  The open-label MAD study was initiated in the third quarter of 2019 to assess safety, tolerability and pharmacokinetics of CRV431 in healthy volunteers. The study is designed to examine doses of 75 mg, 150 mg, 225 mg and 300 mg, with the potential to assess higher doses, where CRV431 is administered orally, once daily for 28 days.

Princeton-based BioNJ Member Photocure ASA  is pleased to announce that the results from a new study with Blue Light Cystoscopy (BLC™) with Hexvix and flexible cystoscopes are now published in Urology 699 patients at 3 hospitals in Denmark were enrolled and randomized 1:1 to either BLC with Hexvix with flexible cystoscopes or white light (WL) only flexible cystoscopy at the time of first follow up after TURBT. Primary endpoint was tumor recurrence within eight months from the randomization. Secondary outcomes were numbers of procedures (TURBTs) in general anesthesia, time to the first recurrence, differences in tumor size, risk of tumor progression and identification of carcinoma in situ.

Princeton-based BioNJ-Member  Genmab A/S announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending broadening the existing marketing authorization for DARZALEX® (daratumumab) in the European Union. The recommendation is for the use of DARZALEX in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant (ASCT).

Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced overall survival (OS) results from the Phase 3 ALCYONE study, which showed the addition of DARZALEX® (daratumumab) to bortezomib, melphalan and prednisone (D-VMP) improved OS in patients with newly diagnosed, transplant-ineligible multiple myeloma, with a 40 percent reduction in the risk of death compared to VMP alone. These updated data from the ALCYONE study also demonstrated that the addition of DARZALEX® to VMP resulted in higher rates of minimal residual disease (MRD) negativity.

Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the Phase 3 CANDOR study showing the addition of DARZALEX® (daratumumab) to carfilzomib (Kyprolis®) and dexamethasone (DKd), compared to carfilzomib and dexamethasone (Kd) alone, significantly improved progression-free survival (PFS) in patients with relapsed/refractory multiple myeloma, resulting in a 37 percent reduction in the risk of progression or death (HR, 0.63; 95 percent CI, 0.46-0.85; P=0.0014).

Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for JNJ-68284528 (JNJ-4528), an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy in previously treated patients with multiple myeloma. Breakthrough Therapy Designation is granted to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition.

Titusville-based BioNJ Member Janssen Biotech, Inc., a Janssen Pharmaceutical Company of Johnson & Johnson, announced that it has entered a definitive agreement to acquire all rights to the investigational compound bermekimab from XBiotech Inc. Bermekimab is an anti-IL-1alpha monoclonal antibody (mAb) in Phase 2 development for the treatment of atopic dermatitis and hidradenitis suppurativa. It is the only antibody targeting IL-1a currently in clinical development and has the potential for superior efficacy and safety compared to the current standard of care.

New Brunswick-based BioNJ Member Johnson & Johnson announced that its Janssen Pharmaceutical Companies will provide up to 200,000 Ebola vaccine regimens to the Republic of Rwanda. This commitment will support a new immunization program led by the Rwanda Government that aims to help protect the country's citizens from the Ebola outbreak in neighboring Democratic Republic of the Congo (DRC). The first batches of Janssen vaccine have been delivered to the country, and further shipments are being organized.

Princeton -based BioNJ Member  Bristol-Myers Squibb Company  announced clinical results from the QUAZAR AML-001 study, evaluating investigational agent CC-486 as maintenance therapy in a broad population of patients with front-line, newly diagnosed acute myeloid leukemia (AML) who have achieved remission with intensive induction chemotherapy. In the QUAZAR AML-001 study, treatment with CC-486 in the maintenance setting provided patients a statistically significant and clinically meaningful improvement in overall survival (OS) and relapse-free survival (RFS), as compared to those patients treated with placebo.

Princeton -based BioNJ Member  Bristol-Myers Squibb Company  and Acceleron Pharma announced data evaluating the erythroid maturation agent (EMA) Reblozyl ® (luspatercept-aamt) in patients with anemia associated with a range of serious and rare blood diseases. "The data presented at ASH this year underscore the role that Reblozyl, the first and only erythroid maturation agent, plays for patients with beta thalassemia-associated anemia and may serve in further investigational uses in myelofibrosis and MDS," said Samit Hirawat, M.D., Chief Medical Officer of Bristol-Myers Squibb.

Kenilworth-based BioNJ Member  Merck & Co. announced that the U.S. Food and Drug Administration has approved ERVEBO® (Ebola Zaire Vaccine, Live) for the prevention of disease caused by Zaire ebolavirus in individuals 18 years of age and older. The duration of protection conferred by ERVEBO is unknown. ERVEBO does not protect against other species of Ebolavirus or Marburgvirus. Effectiveness of the vaccine when administered concurrently with antiviral medication, immune globulin (IG) and/or blood or plasma transfusions is unknown. 

Kenilworth-based BioNJ Member  Merck & Co. announced that KEYTRUDA, Merck's anti-PD-1 therapy, received new approvals from the Japan Pharmaceuticals and Medical Devices Agency (PMDA) in advanced renal cell carcinoma (RCC) and head and neck cancer for additional indications in Japan. "Today's approval of three new first-line KEYTRUDA regimens represents a significant milestone for patients diagnosed with these aggressive forms of cancer and will provide patients in Japan with important alternatives to standard therapies," said Dr. Jonathan Cheng, Vice President, Oncology Clinical Research, Merck Research Laboratories. 

Kenilworth-based BioNJ Member  Merck & Co. announced that the Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) voted 9-4 in favor of recommending KEYTRUDA, Merck's anti-PD-1 therapy, for the treatment of certain patients with high-risk, non-muscle invasive bladder cancer (NMIBC). The ODAC discussions were based on the supplemental Biologics License Application (sBLA), currently under priority review at the FDA, seeking approval of KEYTRUDA monotherapy for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, NMIBC with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder). 

Kenilworth-based BioNJ Member  Merck & Co. announced that KEYTRUDA, Merck's anti-PD-1 therapy, showed improvements in overall survival (OS), progression-free survival (PFS) and objective response rate (ORR) as monotherapy for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC) whose tumors expressed PD-L1 (tumor proportion score [TPS] ≥1%), regardless of KRAS mutational status. These findings, which are based on an exploratory analysis of the pivotal Phase 3 KEYNOTE-042 trial.

Kenilworth-based BioNJ Member  Merck & Co. and ArQule, Inc. announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire ArQule for $20 per share in cash for an approximate total equity value of $2.7 billion. ArQule is a publicly traded biopharmaceutical company focused on kinase inhibitor discovery and development for the treatment of patients with cancer and other diseases.

Princeton -based BioNJ-Member  PDS Biotechnology Corporation   announced that Dr. Jared Weiss , an internationally recognized expert in the field of thoracic and head and neck cancers and faculty at the  University of North Carolina at Chapel Hill  School of Medicine and  Lineberger Comprehensive Cancer Center , will be the Principal Investigator for the Company's Phase 2 VERSATILE-002 study in collaboration with BioNJ Member  Merck & Co The combination study will evaluate the efficacy and safety of PDS Biotech's lead Versamune®-based immunotherapy, PDS0101, in combination with  Merck's  anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) in the first-line treatment of patients with recurrent or metastatic human papillomavirus-16 (HPV16) positive head and neck cancer. 

Bridgewater-based BioNJ-Member  Eli Lilly and Company  announced the launch of TRIUMPH, a long-term, real-world evidence study of Emgality ®  (galcanezumab-gnlm). The objective of the TRIUMPH study (Preventive Tr eatment of Mi graine: Ou tcom es for P atients in Real-World H ealthcare Systems) is to evaluate the real-world effectiveness of Emgality, in comparison to other preventive treatments for migraine, among people receiving routine medical care who are switching or beginning a new prescription treatment for migraine prevention.

Bridgewater-based BioNJ-Member  Eli Lilly and Company  has announced a global commercialization agreement to integrate  DexCom, Inc.  products into Lilly's personalized diabetes management system, currently in development to advance the treatment of diabetes. Under the terms of the non-exclusive agreement, Lilly will use Dexcom's continuous glucose  monitoring (CGM) devices in both the pen- and pump-based platforms of the system being designed to help improve diabetes management.

Bridgewater-based BioNJ-Member  Eli Lilly and Company  announced that the  European Medicines Agency's  (EMA)  Committee for Medicinal Products for Human Use  (CHMP) issued a positive opinion recommending a new indication and associated label update for CYRAMZA ®  (ramucirumab). The Committee agreed that the label should include an indication for CYRAMZA in combination with erlotinib for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) mutations. 

Bridgewater-based BioNJ-Member  Eli Lilly and Company  announced the opening of the LIBRETTO-431 clinical trial [NCT04194944] for selpercatinib, also known as LOXO-292, for treatment-naïve RET  fusion-positive non-small cell lung cancer (NSCLC) patients. Enrolled trial participants will be randomized to receive either selpercatinib or platinum-based (carboplatin or cisplatin) and pemetrexed therapy with or without pembrolizumab as initial treatment of their advanced or metastatic RET  fusion-positive NSCLC.

Bridgewater-based BioNJ-Member  Eli Lilly and Company  announced interim clinical data from the LOXO-305 global Phase 1/2 BRUIN dose escalation trial. LOXO-305 is an investigational, highly selective, non-covalent Bruton's tyrosine kinase (BTK) inhibitor. At all doses studied, LOXO-305 delivered objective responses in patients who had received diverse prior therapies and had exhibited varied molecular mechanisms of acquired resistance.

Madison-based BioNJ-Member  Allergan plc  announced that positive results from ACHIEVE I (UBR-MD-01), a robust Phase 3 clinical trial evaluating the efficacy, safety and tolerability of ubrogepant, have been published in The New England Journal of Medicine. The data from this second published pivotal trial reinforced that the acute treatment of migraine with ubrogepant, compared with placebo, led to significantly greater rates of pain freedom and freedom from the most bothersome migraine-associated symptom at two hours with both the 50 mg and 100 mg doses.

Morristown-based BioNJ-Member  BioAegis Therapeutics, Inc. announces publication of new research findings with recombinant human plasma gelsolin in influenza. The research demonstrates that recombinant human plasma gelsolin therapy dramatically improves survival in a highly lethal influenza animal model. Seasonal influenza continues to be a cause of substantial morbidity and mortality. The U.S. Center for Disease Control and Prevention (CDC) documented that seasonal influenza was responsible for close to 57,000 deaths during the 2018-2019 season.

A pivotal Phase 3 open-label, single-arm trial evaluating the safety and efficacy of Bridgewater-based BioNJ Member Sanofi's sutimlimab in people with primary cold agglutinin disease (CAD) met its primary and secondary endpoints. Sutimlimab is the first investigational treatment designed to selectively target and inhibit C1s in the classical complement pathway, a part of the immune system that is responsible for activating the mechanism of hemolysis in CAD. It has the potential to be the first approved therapy for this rare autoimmune hemolytic anemia. 

Bridgewater-based BioNJ Member  Sanofi and Synthorx, Inc. entered into a definitive agreement under which Sanofi will acquire all of the outstanding shares of Synthorx for $68 per share in cash, which represents an aggregate equity value of approximately $2.5 billion (on a fully diluted basis). The transaction was unanimously approved by both the Sanofi and Synthorx Boards of Directors. "This acquisition fits perfectly with our strategy to build a portfolio of high-quality assets and to lead with innovation," said Paul Hudson, Chief Executive Officer, Sanofi.

Bridgewater-based BioNJ Member Sanofi  and Regeneron Pharmaceuticals, Inc. announced their intent to simplify their antibody collaboration for Kevzara ®  (sarilumab) and Praluent ®  (alirocumab) by restructuring into a royalty-based agreement. Under the proposed restructuring, Sanofi is expected to gain sole global rights to Kevzara and sole ex-U.S. rights to Praluent. Regeneron is expected to gain sole U.S. rights to Praluent. Under the proposed terms of the agreement, each party will be solely responsible for funding development and commercialization expenses in their respective territories. These changes are expected to increase efficiency and streamline operations for the products. 

Basking Ridge-based  Regeneron Pharmaceuticals, Inc.  announced initial clinical data for REGN5458, a BCMAxCD3 bispecific antibody, in patients with relapsed or refractory (R/R) multiple myeloma. BCMA (B-cell maturation antigen) is a protein that is typically over-expressed on multiple myeloma cells. REGN5458 is designed to bind to BCMA on multiple myeloma cells and the CD3 receptor on T-cells, bridging them together and activating T-cell killing of the cancer cell.

Basking Ridge-based  Daiichi Sankyo and AstraZeneca presented positive detailed data from the global pivotal pPhase 2 single-arm DESTINY-Breast01 trial of DS-8201 ([fam]-trastuzumab deruxtecan), an investigational HER2 targeting antibody drug conjugate (ADC), in patients with HER2 positive metastatic breast cancer. The primary endpoint of objective response rate (ORR), confirmed by independent central review, was 60.9% with DS-8201 monotherapy (5.4mg/kg) in patients with HER2 positive metastatic breast cancer who received two or more prior HER2 targeted regimens. 

Ridgefield Park-based  BeiGene, Ltd.  announced results from the Phase 3 ASPEN trial comparing its BTK inhibitor BRUKINSA™ (zanubrutinib) to ibrutinib for the treatment of Waldenström's macroglobulinemia (WM). While the trial did not achieve statistical significance on its primary endpoint of superiority in complete response (CR) and very good partial response (VGPR) rates for zanubrutinib compared to ibrutinib, zanubrutinib demonstrated a higher VGPR rate as well as improvements in safety and tolerability in this first randomized comparative trial to read out within the BTK inhibitor class.

Warren-based  Bellerophon Therapeutics, Inc.  announced positive top-line results from Cohort 2 of its ongoing Phase 2/3 randomized, double-blind, placebo-controlled clinical study (iNO-PF) of INOpulse® for the treatment of Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD).  Subjects in Cohort 2 of iNO-PF treated with iNO45 (45 mcg/kg IBW/hr) demonstrated statistically significant improvement in moderate to vigorous physical activity (MVPA), defined as walking, climbing stairs, yard work, and similar activities, versus placebo.

Basking Ridge-based  Mallinckrodt plc  confirmed enrollment of the first patient in the company's Phase 4, multi-center, multiple-dose, open-label study to assess the effects of Acthar Gel as a therapy option in patients with severe keratitis. Acthar Gel is approved by the  U.S. Food and Drug Administration  ( FDA ) for the treatment of severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa - such as keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation -- one of the product's 19 indications.

Woodcliff Lake-based  Eagle Pharmaceuticals, Inc. announced that the Company has commenced dosing in a pilot clinical study to assess the unique characteristics of its fulvestrant product candidate, which has the potential to enhance estrogen receptor ("ER") inhibition and improve patient outcomes. The results of the pilot study will inform the design of the Company's pivotal trial, which Eagle expects to commence in 2020.

Woodcliff Lake-based  Eagle Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) for RYANODEX ®  (dantrolene sodium) for the treatment of organophosphate exposure. Eagle is currently evaluating RYANODEX for the treatment of brain damage secondary to nerve agent exposure. If approved, RYANODEX would represent the first product available for this indication.

GlaxoSmithKline plc, with offices in Warren, announced  positive headline results for intravenous (IV) Benlysta (belimumab) in the largest controlled phase 3 study in active lupus nephritis (LN), an inflammation of the kidneys caused by systemic lupus erythematosus (SLE) which can lead to end-stage kidney disease.  Dr Hal Barron, Chief Scientific Officer and President R&D, GSK said: "The results of the BLISS-LN study show that Benlysta could make a clinically meaningful improvement to the lives of these patients who currently have limited treatment options."

GlaxoSmithKline plc, with offices in Warren, announced treatment with the investigational single-agent belantamab mafodotin resulted in a clinically meaningful 31% overall response rate (ORR) with the 2.5 mg/kg regimen in patients with heavily pre-treated multiple myeloma. Patients in the trial received a median of seven prior lines of treatment, were refractory to an immunomodulatory drug and a proteasome inhibitor and were refractory and/or intolerant to an anti-CD38 antibody. The median duration of response has not been reached at six months of follow-up.

Whippany-based Bayer announced the publication of an analysis of the PROTECT VIII extension study in which a subset of 33 patients with hemophilia A who received five years of prophylactic treatment with Jivi® (antihemophilic factor [recombinant] PEGylated-aucl) maintained a safety and efficacy profile that was consistent with a prior analysis of the same subset, as well as with an analysis of the pivotal Phase III PROTECT VIII main study.

Bridgewater-based Amneal Pharmaceuticals Inc. announced they have entered into a definitive agreement under which Amneal will acquire a 65.1% majority interest in AvKARE and its related affiliate doing business as R&S Northeast (collectively, "AvKARE") for an implied enterprise value of $340 million. Following the close of the transaction, AvKARE will operate on a standalone basis as an independent subsidiary of Amneal.

Medical technology company Integra LifeSciences held a ribbon-cutting ceremony for its new 167,000-square-foot global headquarters at 1100 Campus Road in Princeton (technically Plainsboro Township), where approximately 600 on-site employees now have access to a cutting-edge collaborative work space environment.  Integra's Vice President of Global Marketing Ruth Fleming told New Jersey Business that the site "is in a beautiful beltway of other medical technology companies."  She added, "Being in the Princeton and Plainsboro area [gives us access to significant labor market] talent to potentially recruit into our organization."

People in the News

The New Jersey  Commission on Science, Innovation and Technology (CSIT)  announced that it has named Judith A. Sheft as its Executive Director. Sheft, who has an extensive background in fostering opportunities for early-stage New Jersey companies and for the next generation of New Jersey entrepreneurs, will officially take the helm of the CSIT beginning January 13, 2020. In August 2018, Governor Murphy signed legislation re-establishing the former New Jersey Commission on Science and Technology as the CSIT. Comprised of representatives from the public and private sectors, as well as academia, the Commission is tasked with leading the way in promoting the State as a home for academic and technological research, development, and commercialization.

Princeton -based BioNJ Member SCYNEXIS, Inc. announced the appointment of accomplished business development executive Philippe Tinmouth to its Board of Directors. Mr. Tinmouth brings over 20 years of experience across multiple business development and alliance management roles, and is retiring as Vice President and Head of Business Development at Vertex Pharmaceuticals Incorporated, a role he has held since 2013.

Princeton-based BioNJ Member Bristol-Myers Squibb Company announced that John Elicker, Executive Vice President, Corporate Affairs and Investor Relations, will retire from the company March 31, 2020. Kathryn Metcalfe, currently Chief Communications Officer of CVS Health, has been appointed Executive Vice President, Corporate Affairs, effective January 6, 2020. Tim Power has been appointed Vice President and Head of Investor Relations, effective March 31, 2020 upon Mr. Elicker's retirement.

Cranbury-based PMV Pharmaceuticals Inc.  announced it has appointed Dr. Leila Alland as its new Chief Medical Officer.  "Dr. Alland's depth of experience and proven leadership in shepherding innovative products from pre-clinical development through approval will be critical as we advance toward the clinic," David Mack, CEO and President, PMV Pharma, said. "Her rich background in leading oncology clinical trials and drug development will help define the development strategies for our multiple programs."

Paramus -based Ichnos Sciences Inc. is pleased to announce two important additions to its leadership team. Patrick E. Flanigan III has assumed the role of Chief Financial Officer and Martin L. Wilson will start as General Counsel (GC) effective January 7, 2020Mr. Flanigan is a finance leader with over 20 years of experience in the biotechnology industry. Prior to joining Ichnos Sciences, he served as Senior Vice President of Investor Relations & Treasury for Celgene Corporation. Mr. Wilson is joining Ichnos Sciences from Teligent, Inc., where he served as GC from 2017-2019. He has more than 15 years of experience in the pharmaceutical industry.

Institution and Education News

New Jersey Institute of Technology (NJIT) in collaboration with New Jersey Innovation Institute (NJII), an NJIT corporation, has announced it will soon offer both a Professional Science Masters (PSM) degree program and professional graduate certificate in the rapidly expanding field of cell and gene therapy. The four-course certificate program, also available next year for industry professionals and students seeking training in cutting-edge technologies required for processing and commercializing of new therapies, may be applied to the 30-credit M.Sc. option recognized by the Professional Science Master's National Office.

Rutgers University, Princeton University and The Science Coalition (TSC) presented U.S. Representative Frank Pallone (D-NJ) with the Champion of Science Award, recognizing his support for the fundamental scientific research conducted by universities in New Jersey and across the United States. Rutgers and Princeton officials presented the award at a conference hosted by Rutgers' Center for Discrete Mathematics and Theoretical Computer Science (DIMACS).

Investigators at Rutgers Cancer Institute of New Jersey have found that a treatment based on a novel cellular product programmed to deliver an overabundance of chimeric antigen receptor causes cell death in non-Hodgkin lymphoma models that are sensitive or resistant to standard therapies. B-cell non-Hodgkin lymphomas are the most common form of lymphoma in the Western world. The disease is generally treatable, but a vast majority of patients with slow-growing disease is incurable and a significant minority of patients with an aggressive form of B-cell non-Hodgkin lymphoma die from it.

Investigators at Rutgers Cancer Institute of New Jersey have determined that an overabundance of a novel gene known as ADNP in an aggressive form of ovarian cancer may serve as a prognostic tool.  Rutgers Cancer Institute researcher Michael Gatza, an Assistant Professor of Radiation Oncology at Rutgers Robert Wood Johnson Medical School, is the senior and corresponding author of the work, and said the topic is important because ovarian cancer is the fifth leading cause of cancer-related deaths among women in the United States.

Rutgers  Robert Wood Johnson Medical School scientists and Microsoft Corp. volunteers announced the rollout of Baby be Well, a free mobile app to reduce the risk of sudden infant death syndrome.  In a press release, Rutgers said the app will help families keep infants safe throughout the first year of life through its proven guidance of safe sleep practices as part of its AI for Good initiative and Tata Consultancy Services.

The Stevens Institute of Technology said it opened a $68 million teaching and research facility -- the Gateway Academic Center.  Stevens described the Center as part of a broad campaign that began in 2014 to modernize its infrastructure into a modern campus for teaching, research, learning and living. The building features the Mary Jane and Frank Semcer Sr. '65 Center for Healthcare Innovation to address gaps and issues related to advancing biomedical technology and health care delivery, and to integrate external strategic partnerships. 

Funding News

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  $850,000  in net proceeds. "This is our ninth year receiving NOL funding. Over this time period, we have received approximately $5.7 million in non-dilutive NOL funding that has allowed us to advance our rare disease pipeline to where we are now approaching final data readout in two pivotal Phase 3 clinical trials," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. 

Pennington-based BioNJ Member OncoSec Medical Incorporated announced the Company has received preliminary approval for a tax credit from the New Jersey Technology Business Tax Certificate Transfer (NOL) Program for the year 2019. The company anticipates it will be able to transfer this credit and receive approximately  $885,000  in cash. "As a biotech company committed to being careful stewards of our shareholder's capital, we moved our corporate headquarters to the State of New Jersey for its terrific tax incentive program and dedication to fostering innovation in the New Jersey biotechnology industry," said Daniel J. O'Connor, CEO of OncoSec. "We expect proceeds from the NOL program to continue to grow annually and help fund further development of our TAVO™ programs."

Lawrenceville-based BioNJ Member  Celsion Corporation  announced it has received approval from the New Jersey Economic Development Authority's (NJEDA) Technology Business Tax Certificate Transfer (NOL) program to sell  $2.0 million  of its unused  New Jersey  net operating losses (NOLs) for the tax years 2017 through 2018. "This innovative funding mechanism offered by the NJEDA reinforces our belief in the State of New Jersey's commitment to biotechnology research," said  Michael H. Tardugno , Celsion Corporation's Chairman, President and Chief Executive Officer. "The proceeds from the NOL sale strengthen our cash position in a non-dilutive fashion and extends Celsion's operating runway into the first quarter of 2021."

The state's  Economic Development Authority approved 46 businesses to take part in New Jersey's 21-year old Net-Operating Loss credit program, an incentive that helps companies offset many of the financial losses that come with research and development.  Under the NOL Program, technology and life sciences businesses can acquire R&D tax breaks, which can be sold for at least 80 percent of their value, capped at $15 million per business.  The $60 million allocated to the program this year will be spread among all 46 companies, 12 of which are taking part in the NOL program for the first time.  This year marks the second in a row that the EDA maxed out the $60 million of tax credits available under the program.

Special Programs from Our Partners for BioNJ Members

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 [email protected] or 609-259-7292 ext. 1443 by December 16, 2019. 

Jun 8-11, 2020 | San Diego

The BIO International Convention is hosted by the  Biotechnology Innovation Organization  (BIO). BIO 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. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products. The BIO International Convention offers a range of opportunities to increase your brand exposure and connect with a global audience of 17,000+ attendees from 65+ countries. 

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 [email protected], 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 [email protected] or call 609-890-3185 
with any questions.