Welcome to the latest edition of the BioLines Weekender...

It's been an exciting week with the announcement of promising results from both Pfizer's and Moderna's COVID-19 vaccine candidates. As the number of cases and deaths due to the Coronavirus continue to climb at record rates, this news couldn't come at a better time and amplifies the value, importance and impact of medical innovation. We thank the researchers, biopharmaceutical companies, government organizations and Patients who are committed to bringing effective and safe vaccines to market.

Meanwhile, yesterday, Governor Murphy announced the advancement, as well as the first four tenants, of The Hub -- a new collaborative site in downtown New Brunswick designed to foster research, entrepreneurship, innovation and start-up incubation. Scheduled to open in 2024, The Hub will bring the public and private sectors, along with academia and investors, together under one roof to foster the growth of start-ups and early stage companies. Princeton University, Rutgers University, Hackensack Meridian Health and RWJBarnabas Health will be the first tenants of The Hub.
 
Please join us for BioNJ's Sixth Annual Inspiring Women in STEM Conference taking place virtually on December 3 and 4, 2020 from 9:00 a.m.-12:45 p.m. The perfect opportunity to learn from the experiences of successful women who have traveled remarkable journeys, please click here to peruse the full program agenda. 

Until then, we wish you and yours a safe, healthy and happy Thanksgiving. 

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

BioNJ's Virtual Inspiring Women in STEM Conference: 
Inspiration, Learning & Professional Growth
Register today!
December 3 & December 4, 2020
BioNJ's Virtual Patient Advocacy Summit: 
*Registration is open to Patient Advocacy Professionals
December 10, 2020
COVID-19 Vaccine News
BioNJ Member Pfizer, with offices in Peapack, and BioNTech SE announced they will submit a request to the U.S. Food and Drug Administration (FDA) for Emergency Use Authorization (EUA) of their mRNA vaccine candidate, BNT162b2 against SARS-CoV-2, which will potentially enable use of the vaccine in high-risk populations in the U.S. by the middle to end of December 2020. The submission is based on a vaccine efficacy rate of 95% (p<0.0001) demonstrated in the companies' Phase 3 clinical study in participants without prior SARS-CoV-2 infection (first primary objective) and also in participants with and without prior SARS-CoV-2 infection (second primary objective), in each case measured from 7 days after the second dose. To date, the Data Monitoring Committee (DMC) for the study has not reported any serious safety concerns related to the vaccine.

BioNJ in the News
(As featured in BioWorld)

Although the global impact of a novel virus like SARS-CoV-2 has so far reached devastating proportions, it has spurred science at an unprecedented rate with potential vaccines developed under a year now nearing U.S. authorization. But while some may say the end to this pandemic is in sight, an actual date is blurry at best, as manufacturing and supply capabilities muddy the outlook. "This is something that will go on for a long time because 600 million vaccinations have never been done before," said Thomas Warf, an expert consultant with the U.S. Department of Health and Human Services' Biomedical Advanced Research and Development Authority (HHS; BARDA).
(As featured in New Jersey Business Magazine)

Against the backdrop of Pfizer's announcement this week of a promising COVID-19 vaccine, BioNJ, the trade group for the biotech life science industry in New Jersey, held a virtual event to discuss a "new normal" in bringing vaccines to market, including how the public and private sectors can work together to make the process faster and more efficient without sacrificing safety and effectiveness of vaccines and therapeutics. Mireli Fino, Sanofi's site head, Protein Sciences, spoke about what she called a "crazy nine months" with COVID-19, which has underscored a need to speed up vaccine development and its exploratory, pre-clinical, clinical and regulatory phases. "There's been a fundamental paradigm shift; we cannot take 10 years to develop a vaccine for a disease creating such problems for the whole world," she said. "The question was how to compress the timelines while managing risk."

NJ Pride

Gov. Phil Murphy's dream of returning New Jersey to its place as the nation's leader in innovative companies and ideas got a boost Thursday when he announced the State's two leading health systems and two leading universities will be anchor tenants at The Hub in New Brunswick. Princeton University, Rutgers University, Hackensack Meridian Health and RWJBarnabas Health will be the first tenants of The Hub, which is viewed as a new collaborative site in downtown New Brunswick designed to foster research, innovation, entrepreneurship and start-up incubation in New Jersey.

NJ Company News
Bridgewater-based BioNJ Member Insmed Incorporated announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to brensocatib for the treatment of non-cystic fibrosis bronchiectasis (NCFBE). Brensocatib is a novel, first-in-class, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) being developed by Insmed for the treatment of NCFBE and other inflammatory diseases. "We are building on the strength of the Phase 2 WILLOW study with the initiation of a pivotal Phase 3 program for brensocatib that we hope will bring forth this urgently needed solution," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. 

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced that the United States (U.S.) Food and Drug Administration (FDA) has granted PTC596 both Orphan Drug Designation and Fast Track designation for the potential treatment of leiomyosarcoma (LMS), a rare type of cancer that affects smooth muscle tissue. Furthermore, the FDA has also granted PTC596 a Rare Pediatric Disease designation and Orphan Drug Designation for the potential treatment of Diffuse Intrinsic Pontine Glioma (DIPG), an ultra-rare childhood glioma. PTC596 is currently being studied in clinical trials in LMS and DIPG.  

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced the initiation of a Phase 1 clinical trial to evaluate PTC518 in healthy volunteers. PTC518 was identified from PTC's splicing platform and is being developed for the treatment of Huntington's disease (HD). PTC518 is an orally bioavailable molecule with broad central nervous system and systemic distribution and has been designed to target Huntingtin protein expression with high selectivity and specificity. PTC518 has favorable pharmaceutical properties and has demonstrated uniform lowering of the Huntingtin protein throughout the brain in animal models. Currently there are no approved disease modifying therapies for HD.

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced the presentation of data on two rare diseases, providing new insights on the burden of Aromatic L-Amino acid Decarboxylase deficiency (AADC-d) and Duchenne muscular dystrophy caused by nonsense mutation (nmDMD). The data, presented at the Virtual ISPOR Europe 2020 Conference, further advance the understanding of these rare and devastating childhood genetic disorders, and the need and value of effective treatments. 
 
Bridgewater-based BioNJ Member Nevakar Inc. announced that the first patient has completed three year enrollment in its Phase III CHAMP (Childhood Atropine for Myopia Progression) study, evaluating the ability of its lead compound, NVK-002, to slow the progression of myopia in children. NVK-002 is a proprietary, investigational, preservative-free eye drop administered nightly and intended for patients ages three to seventeen. The CHAMP trial, which is being conducted in the U.S. and Europe, follows ground-breaking studies conducted in Asia that concluded that low doses of atropine could be used to slow the progression of myopia in children.
Monmouth Junction-based OncoSec Immunotherapies has developed experimental cancer treatments that deliver genes into tumors. There, the injected genes produce a natural signalling molecule called IL-12, which attracts the attention of immune cells that attack the cancer. In the spring, OncoSec began adapting their technology to make a vaccine for the coronavirus. The vaccine, called CORVax12, consists of a loop of DNA that encodes both the spike protein and IL-12. Causing the body to make extra IL-12 could potentially enhance the immune system's ability to make antibodies to the spike protein. On Nov. 13, the company registered a Phase 1 trial to test the safety of the CORVax12.
Cranbury-based BioNJ Member Rafael Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company's lead compound, CPI-613® (devimistat), for the treatment of metastatic pancreatic cancer. Rafael has continued to reach milestones throughout the year, including achieving its target enrollment of 500 patients for its Phase 3 trial for metastatic pancreatic cancer ahead of schedule. The company also recently announced that the FDA granted devimistat Orphan Drug Designation for the treatment of soft tissue sarcoma.  
Morristown-based BioNJ Member BioAegis Therapeutics Inc. announced that it has expanded its clinical trial in severe COVID-19 to additional sites in Europe. The study has recruited more than one third of the expected enrollment and the independent Data Safety Monitoring Board (DSMB) has recently recommended continuation of recruitment based on a review of the safety data. BioAegis' Phase 2 clinical trial of recombinant human plasma gelsolin (rhu-pGSN) for the treatment of severe COVID-19 was initiated in August in Spain where COVID-19 cases were spiking. The study assesses rhu-pGSN, and its ability to regulate the overactive inflammatory response often responsible for causing lung damage and death in COVID-19 patients. 
BioNJ Member Pfizer, with offices in Peapack, announced results from the Phase 3 CROWN trial of LORBRENA® (lorlatinib, available in Europe under the brand name LORVIQUA®) versus XALKORI® (crizotinib) in people with previously untreated anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC) were published online ahead of print in the New England Journal of Medicine. At a planned interim analysis, LORBRENA treatment resulted in statistically significant and clinically meaningful improvement in progression-free survival (PFS) according to blinded independent central review (BICR), the primary endpoint, compared to XALKORI (HR 0.28: 95% CI, 0.19 to 0.41; p<0.001), corresponding to a 72
BioNJ Member Pfizer, with offices in Peapack, announced positive top-line results from the Phase 3 JADE REGIMEN study. This 52-week study investigated abrocitinib, an investigational oral once-daily Janus kinase 1 (JAK1) inhibitor, in patients 12 and older with moderate to severe atopic dermatitis (AD) following response to initial open label induction treatment with abrocitinib 200mg. Patients were randomized into one of three arms: 200mg, 100mg, or placebo. Both doses of abrocitinib met the primary endpoint, resulting in significantly fewer patients experiencing a loss of response requiring rescue treatment, or "flaring," compared to those randomized to placebo. 
BioNJ Member Pfizer, with offices in Peapack, announced the publication of an analysis showing that VYNDAQEL® (tafamidis meglumine) 80 mg/VYNDAMAX® (tafamidis) 61 mg were associated with a statistically significant improvement in long-term survival in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) compared to VYNDAQEL 20 mg, reaffirming optimal dosing for these patients. Results from the analysis of the Phase 3 Tafamidis in Transthyretin Amyloid Cardiomyopathy Clinical Trial (ATTR-ACT) and its long-term extension study, which were published in the European Journal of Heart Failure, demonstrated a 30% relative reduction in the risk of death (p=0.0374) among patients with ATTR-CM initially treated with VYNDAQEL 80 mg and subsequently transitioned to VYNDAMAX 61 mg, versus patients initially treated with VYNDAQEL 20 mg and transitioned to VYNDAMAX 61 mg.









Jersey City -based BioNJ Member Mitsubishi Tanabe Pharma America, Inc. announced that patient enrollment has been completed for the global Phase 3 open-label study evaluating the long-term safety and tolerability of an investigational oral suspension formulation of edaravone (MT-1186) in patients with amyotrophic lateral sclerosis (ALS). The study is sponsored by Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) The global multi-center, open-label study (MT-1186-A01), is evaluating approximately 185 ALS patients across approximately 50 sites in the U.S., Canada, Europe and Japan over the course of 48 weeks of treatment. 

New Brunswick-based BioNJ Member Johnson & Johnson announced the Phase 3 ENSEMBLE study of the single-dose regimen of JNJ-78436735, the investigational vaccine candidate for the prevention of COVID-19 being developed by the Janssen Pharmaceutical Companies of Johnson & Johnson, continues to enroll and vaccinate study participants. ENSEMBLE is proceeding to enroll up to 60,000 participants worldwide. In addition to the single-dose regimen ENSEMBLE study, Janssen has now initiated the two-dose regimen ENSEMBLE 2 trial. ENSEMBLE 2 is a complementary, planned, pivotal, large-scale, multi-country Phase 3 trial that will study the safety and efficacy of a two-dose regimen of the investigational Janssen vaccine candidate for the prevention of COVID-19 in up to 30,000 participants worldwide. 

New Brunswick-based BioNJ Member Johnson & Johnson announced $100 million in commitments and collaborations over the next five years to invest in and promote health equity solutions for Black people and other communities of color in the United States. Society has been significantly impacted by systemic racism, the COVID-19 pandemic, and the economic decline throughout this year, which have all spotlighted health care inequities. The company's commitment prioritizes three key areas: Healthier Communities -- investing in programs that help provide equitable health care for underserved communities; Enduring Alliances -- forging partnerships and alliances that combat racial and social health determinants; and Diverse & Inclusive Corporate Culture -- ensuring a diverse and inclusive workforce.
Princeton-based BioNJ Member Bristol Myers Squibb announced results from an ongoing Phase 2 study evaluating the safety and efficacy of deucravacitinib (BMS-986165) 6 mg or 12 mg once daily, compared with placebo in adults with active psoriatic arthritis. The study met the primary endpoint of at least a 20 percent improvement in signs and symptoms of disease (ACR 20) at Week 16. Patients treated with deucravacitinib 6 mg (n=70) or 12 mg (n=67) demonstrated significantly greater ACR 20 responses versus placebo (n=66) at Week 16 (52.9% and 62.7% versus 31.8%, respectively) (p=0.0134 and p=0.0004, respectively). 
Princeton-based BioNJ Member Bristol Myers Squibb announced that it has successfully completed its acquisition of MyoKardia, Inc. in an all cash transaction for approximately $13.1 billion. With the completion of the acquisition, MyoKardia shares have ceased trading on the NASDAQ Global Select Market and MyoKardia is now a wholly-owned subsidiary of Bristol Myers Squibb. Through the transaction with MyoKardia, Bristol Myers Squibb gains mavacamten, a potential first-in-class cardiovascular medicine for the treatment of obstructive hypertrophic cardiomyopathy ("HCM"), a chronic heart disease with high morbidity and patient impact.
East Hanover-based BioNJ Member Novartis announced that it entered into an exclusive worldwide license and collaboration agreement with Mesoblast to develop, commercialize and manufacture remestemcel-L for the treatment of acute respiratory distress syndrome (ARDS), including that associated with COVID-19. ARDS is an area of significant unmet need, with an approximate 40% mortality rate with current standard of care, which includes prolonged ICU treatment and mechanical ventilation.1,2 As the potential first ARDS therapy, remestemcel-L will use mesenchymal stromal cells (MSCs), a cell-based platform technology, to treat this deadly condition and improve outcomes. 
East Hanover-based BioNJ Member Novartis announced results from two pooled post-hoc analyses of Phase III ORION-9, -10 and -11 trials, evaluating the impact of age and gender on the efficacy and safety of inclisiran, an investigational and potential first-in-class small interfering RNA (siRNA) for hyperlipidemia in adults with atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH). The data showed that at month 17, inclisiran was well-tolerated and provided effective and sustained reduction in low-density lipoprotein cholesterol (LDL-C) when used in addition to other lipid lowering therapies regardless of patients' age and gender.

Bridgewater-based BioNJ Member Eli Lilly and Incyte announced that the U.S. Food and Drug Administration (FDA) issued an Emergency Use Authorization (EUA) for the distribution and emergency use of baricitinib to be used in combination with remdesivir in hospitalized adult and pediatric patients two years of age or older with suspected or laboratory confirmed COVID-19 who require supplemental oxygen, invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).

Bridgewater-based BioNJ Member Eli Lilly and Company and Precision BioSciences, Inc. announced a research collaboration and exclusive license agreement to utilize Precision's proprietary ARCUS® genome editing platform for the research and development of potential in vivo therapies for genetic disorders, with an initial focus on Duchenne muscular dystrophy (DMD) and two other undisclosed gene targets. ARCUS is a unique, proprietary, and versatile genome editing platform with attributes including specificity, ability to make a variety of efficient edits (knock-in, knock-out, and repair), and small size, thereby enabling a range of therapeutic editing. 

The U.S. Food and Drug Administration (FDA) granted Emergency Use Authorization (EUA) for Bridgewater-based BioNJ Member Eli Lilly and Company's investigational neutralizing antibody bamlanivimab (LY-CoV555) 700 mg. Bamlanivimab is authorized for the treatment of mild to moderate COVID-19 in adults and pediatric patients 12 years and older with a positive COVID-19 test, who are at high risk for progressing to severe COVID-19 and/or hospitalization. Bamlanivimab should be administered as soon as possible after a positive COVID-19 test and within 10 days of symptom onset. The authorization allows for the distribution and emergency use of bamlanivimab, which is administered via a single intravenous infusion.

Bridgwater-based BioNJ Member Eli Lilly and Company and Ypsomed announced a non-exclusive, global agreement to advance an automated insulin delivery system as part of Lilly's connected diabetes solutions. Under the terms of the agreement, Lilly will commercialize the system, which is currently in development and will include an insulin pump developed and manufactured by Ypsomed. Ypsomed's insulin pump has been marketed in Europe since 2016. Lilly will commercialize a version of this insulin pump, along with continuous glucose monitoring (CGM) and automated insulin delivery technology, in the United States and Europe. This pump will use pre-filled insulin cartridges for Lilly's rapid-acting insulins.

Bridgewater-based BioNJ Member Eli Lilly and Company announced Jardiance® (empagliflozin) reduced the risk of total (first plus recurrent) cardiovascular events compared with placebo, when both were given on top of standard of care, in adults with type 2 diabetes and established cardiovascular disease over the three years of the EMPA-REG OUTCOME® trial, according to results of a new post-hoc analysis. Total cardiovascular events included 3P-MACE (a composite of non-fatal heart attack, non-fatal stroke and cardiovascular death), hospitalization for heart failure and all-cause hospitalization.

Princeton-based BioNJ Member Evotec SE announced that, as part of its ongoing collaboration with STORM Therapeutics ("STORM"), the leading biotechnology company focused on the discovery and development of small molecule therapies modulating RNA epigenetics, STORM has selected STC-15 as a first-in-class development candidate. STORM will now use INDiGO, Evotec's unique integrated, accelerated IND-enabling platform, to progress STC-15 towards an IND application in 2021.
 
Plainsboro-based BioNJ Member Novo Nordisk announced headline results from the SUSTAIN FORTE trial, a Phase 3b 40-week, efficacy and safety trial with once-weekly semaglutide 2.0 mg vs once-weekly semaglutide 1.0 mg as add-on to metformin and/or sulfonylureas in 961 people with type 2 diabetes in need for treatment intensification. The trial achieved its primary endpoint by demonstrating a statistically significant and superior reduction in HbA1c at week 40 with semaglutide 2.0 mg compared to semaglutide 1.0 mg.
 
Plainsboro-based BioNJ Member Novo Nordisk A/S and Gilead Sciences, Inc. announced results from a Phase 2 proof-of-concept trial. The five-arm trial evaluated combinations of Novo Nordisk's semaglutide, a GLP-1 receptor agonist, with Gilead's investigational FXR agonist cilofexor and/or Gilead's investigational ACC inhibitor firsocostat over 24 weeks in 108 people with non-alcoholic steatohepatitis (NASH). The trial met its primary endpoint by demonstrating that in people with NASH and mild to moderate fibrosis all regimens were well tolerated. The most common adverse events (AEs) were gastrointestinal. Minimal pruritus (itching) was observed in people treated with cilofexor.
 
Berkeley Heights-based BioNJ Member Amgen and AstraZeneca announced positive topline results from the Phase 3 NAVIGATOR trial in which the investigational medicine tezepelumab demonstrated a statistically significant reduction in exacerbations compared to placebo in patients with severe asthma. The NAVIGATOR trial met the primary endpoint with tezepelumab added to standard of care (SoC) demonstrating a statistically significant and clinically meaningful reduction compared to placebo plus SoC in the annualized asthma exacerbation rate (AAER) over 52 weeks in the overall patient population. SoC was medium- or high-dose inhaled corticosteroids (ICS) plus at least one additional controller medication with or without oral corticosteroids (OCS).
Kenilworth-based BioNJ Member Merck & Co. announced a collaboration with the Bill & Melinda Gates Foundation (the foundation) where the foundation is committing to provide funding to support a pivotal Phase 3 study investigating a once-monthly oral pre-exposure prophylaxis (PrEP)1 option in women and adolescent girls at high risk for acquiring HIV-1 infection in sub-Saharan Africa. The study, IMPOWER 22, will evaluate the efficacy and safety of islatravir -- Merck's novel investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) under evaluation for both treatment and prevention -- and is anticipated to begin by early 2021. 
Kenilworth-based BioNJ Member Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck's anti-PD-1 therapy, in combination with chemotherapy for the treatment of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) as determined by an FDA-approved test. The approval is based on results from the Phase 3 KEYNOTE-355 trial, where KEYTRUDA in combination with chemotherapy -- paclitaxel (pac), paclitaxel protein-bound (commonly known as nab-paclitaxel) or gemcitabine (gem) and carboplatin (carbo) -- significantly reduced the risk of disease progression or death by 35% for patients whose tumors express PD-L1 (CPS ≥10) versus the same chemotherapy regimens alone (HR=0.65 [95% CI, 0.49, 0.86]; p=0.0012). 
Kenilworth-based BioNJ Member Merck & Co. announced new investigational data demonstrating positive top-line results from the pivotal Phase 3 KEYNOTE-581/CLEAR trial (Study 307). In the trial, the combinations of KEYTRUDA, Merck's anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus were evaluated versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). KEYTRUDA plus LENVIMA met the trial's primary endpoint of progression-free survival (PFS) and its key secondary endpoints of overall survival (OS) and objective response rate (ORR), demonstrating a statistically significant and clinically meaningful improvement in PFS, OS and ORR versus sunitinib in the intention-to-treat (ITT) study population.
 
Florham Park-based PDS Biotechnology Corporation announced that its VERSATILE-002 Phase 2 clinical trial evaluating the combination of PDS0101 and KEYTRUDA® (pembrolizumab) for the first-line treatment of head and neck cancer that has returned or spread is now open. The trial is being conducted in collaboration with Merck. The VERSATILE-002 Phase 2 trial is a multi-center, open label, single arm, non-randomized trial that plans to enroll approximately 100 patients across 25 sites in the U.S. The University of Tennessee Medical Center is the first site to open.
 
Florham Park-based PDS Biotechnology Corporation announced progress on its co-development program with Farmacore Biotechnology for a Versamune®-based vaccine aimed at preventing COVID-19 infection. Based on promising and robust preclinical data with PDS0203, and in the interest of rapid development, PDS Biotech and Farmacore have jointly prioritized advancement of PDS0203 to human clinical trials. PDS Biotech had previously announced the parallel development of two Versamune®-based COVID-19 vaccines PDS0203 and PDS0204 each containing different SARS-CoV-2 proteins. 

Basking Ridge-based Mallinckrodt plc announced initiation of a retrospective chart review study, titled "Nitric Oxide Treatment In COVID-19 Evaluation (NOTICE)" to collect real-world data on the use of INOmax® (nitric oxide) gas, for inhalation therapy in patients with respiratory complications associated with the novel coronavirus SARS-CoV-2 (COVID-19). INOmax is indicated for the treatment of term and near-term neonates with hypoxic respiratory failure associated with pulmonary hypertension. The safety and efficacy of INOmax to treat lung complications associated with COVID-19 has not been evaluated or established by the U.S. Food and Drug Administration.
 
Woodcliff Lake-based Eisai Co., Ltd. and Biogen announced that it has received approval from the European Commission for the use of its in-house discovered and developed anti-epileptic agent (AED) Fycompa® (generic name: perampanel) in the treatment of pediatric patients. This approval extends the use of Fycompa as an adjunctive therapy for partial-onset seizures (POS) (with or without secondary generalization) by expanding the approved age range from 12 years and above to 4 years and above, and for primary generalized tonic-clonic seizures (PGTCS) from 12 years and above to 7 years and above.
 
Sparta-based PLx Pharma Inc. announced that two chemistry and manufacturing control ("CMC") supplemental New Drug Applications ("sNDAs"), one for VAZALORE 325 mg and one for VAZALORE 81 mg dose (referred to together as "VAZALORE"), were submitted to the U.S. Food and Drug Administration ("FDA") in October for regulatory approval. The 325 mg sNDA provided information on a change in formulation and a new manufacturing site for the currently approved VAZALORE and also contains a bioequivalence ("BE") clinical study report with the required data and analyses from the recently completed BE study. 
 
Ridgefield Park-based BeiGene, Ltd. announced that the RATIONALE 303 trial of its anti-PD-1 antibody tislelizumab versus docetaxel in the second- or third-line setting in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who progressed on prior platinum-based chemotherapy, met its primary endpoint of overall survival (OS) in the intention-to-treat (ITT) patient population at the planned interim analysis, as recommended by the independent Data Monitoring Committee (DMC). The safety profile of tislelizumab was consistent with the known risks of tislelizumab, with no new safety signals identified.
 
Paramus-based BrainStorm Cell Therapeutics Inc. announced topline results from the company's randomized, double-blind placebo-controlled Phase 3 trial evaluating NurOwn® (MSC-NTF cells) as a treatment for Amyotrophic lateral sclerosis (ALS). Results from the trial showed that NurOwn® was generally well tolerated in this population of rapidly progressing ALS patients. While showing a numerical improvement in the treated group compared to placebo across the primary and key secondary efficacy endpoints, the trial did not reach statistically significant results.
 
Basking Ridge-based Lexicon Pharmaceuticals, Inc. announced that both the SOLOIST and SCORED Phase 3 studies achieved their primary endpoints by demonstrating statistically significant reductions in total cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits in patients treated with sotagliflozin as compared with placebo. In the SOLOIST study, the primary endpoint was achieved with a hazard ratio (HR) of 0.67 (p<0.001) in people with type 2 diabetes and a recent hospitalization for worsening heart failure.  

Basking Ridge-based Daiichi Sankyo Company Limited announce that it will create a new Oncology Business Unit, effective April 1, 2021, aligning U.S and European oncology businesses, global oncology functions of marketing, market access and pricing, medical affairs as well as alliance management under one team singularly devoted to people with cancer. Ken Keller, currently leading the U.S. Business and CEO of the company's U.S. affiliates Daiichi Sankyo, Inc. and American Regent, Inc., will lead this new team as Head of the Oncology Business Unit, effective, April 1, 2021, and will remain President and CEO of Daiichi Sankyo, Inc.
 
Bedminster-based Aerie Pharmaceuticals, Inc. announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending approval of the marketing authorisation application (MAA) for Roclanda® (netarsudil and latanoprost ophthalmic solution) 0.02%/0.005%. The recommended indication is the reduction of elevated intraocular pressure in adult patients with primary open-angle glaucoma or ocular hypertension for whom monotherapy with a prostaglandin or netarsudil provides insufficient IOP reduction.
 
Paramus-based Epygenix Therapeutics, Inc. announced that the first patient has been successfully dosed in a Phase 2, 20-week multicenter, randomized, double-blind, placebo-controlled trial of EPX-100 as adjunctive therapy in children with Dravet Syndrome. This study will evaluate the efficacy and safety of EPX-100 in children suffering from Dravet Syndrome. The company is in the middle of recruiting 24 patients to complete the study. Dravet Syndrome is a rare, catastrophic, lifelong form of epilepsy which begins in the first year of life with frequent or prolonged seizures.
 
Franklin Lakes-based BD announced it has acquired cloud-based software company Cubex LLC's Medical Business assets. The acquisition will grow the company's medication management offerings into the care continuum space and help provide better integration with electronic health records, BD - also known as Becton, Dickinson & Co. "This strategic acquisition brings together our industry-leading BD Pyxis Automated Dispensing Cabinets with the Medical Business of Cubex's MedBank cloud-based software and analytics platform to help enable patient-centered care beyond the acute care setting," Mike Garrison, BD's worldwide President of medication management solutions, said in a prepared statement.



People in the News




Somerset-based BioNJ Member Legend Biotech Corp has named a new CEO and Chairwoman. The company's Chief Financial Officer and interim CEO, Ying Huang, was named to the permanent CEO job. He will continue to serve as CFO until a successor is found. In addition, Ye "Sally" Wang was named Chairwoman of the board of directors. 
"The entire board is very pleased to have Ms. Wang as Legend Biotech's Chairwoman," Philip Yau, Chair of the Audit Committee, said in a prepared statement. "Sally's extensive experience and expertise in strategic planning and operational management will be instrumental as she leads our board and supports Legend Biotech's management team in continuing to develop therapies for patients in need."

Ken Keller, the CEO and President of Basking Ridge-based Daiichi Sankyo Inc. -- the U.S. unit of the Japanese company Daiichi Sankyo Co. Ltd. -- is changing some of his responsibilities within the parent group. Keller also serves as CEO and President of Daiichi Sankyo Group's American Regent Inc. business, a maker of injectables for health care providers. Between now and April 2021, he will transition to global head of the oncology business at Daiichi Sankyo Inc., to go along with his CEO and president duties with that company.
In a virtual setting, hundreds of representatives from the largest research organizations in the world, including some of New Jersey's best academic minds, gathered for the 41st annual Thomas Edison Patent Awards hosted by the Research & Development Council of New Jersey. Fourteen companies and institutions of higher education representing nearly 60 researchers received the prestigious award. Additionally, three researchers were honored with special individual recognitions. Congratulations to these BioNJ Members for being recognized: Kean University; Merck & Co., Inc.; New Jersey Institute of Technology; Princeton University; Rowan University and Stevens Institute of Technology

Institution and Education News
After undergoing quadruple bypass surgery more than two decades ago, telehealth pioneer James Tuchi took an interest in the research his doctors were doing. Tuchi, an early innovator in the burgeoning field of online medical care as CEO and founder of Millennium Healthcare Solutions, resolved to champion their work whenever possible. He recently committed $8 million in support of major initiatives at Rutgers Biomedical and Health Sciences (RBHS) and the Rutgers School of Communication and Information.


Funding News

The NIH Office of Science Policy (OSP) within the Office of the Director (OD) announced the availability of administrative supplements to support 1) research on bioethical issues to develop or support the development of an evidence base that may inform future policy directions, and/or 2) certain efforts to develop or augment bioethics research capacity. Applicants may propose to supplement parent awards focused on bioethics or to add a component related to bioethics to a parent award in which bioethics was not the focus. Note that applications must be within the general scope of the parent award.

Special Programs from Our Partners for BioNJ Members
December 2, 2020

Hear from founders and their investors who will share their lessons learned and successful steps in taking an idea from conceptual development through commercial launch, growth and eventual exit. Gain valuable insight on how to maximize resources and avoid common mistakes from our seasoned panelists who have experience in numerous business segments and stages of company development.
January 11-15, 2021

With a blend of on-demand and live sessions, the virtual 2021 program is designed to provide you with the latest industry updates and flexibility to view content according to your schedule. BioNJ Members: Contact Randi Bromberg at RBromberg@BioNJ.org for a special discount code.
January 11-15, 2021

Affordable access to BIO One-on-One Partnering, with an integrated video conferencing system, to provide a seamless partnering experience for all attendees, regardless of whether you are registered for a specific event during JPM week. BIO Members partner for free/ Non-Member companies partner for $150.

Thought Leadership from BioNJ Members
Watch this insightful video featuring life sciences industry experts from J.P. Morgan.

At the dawn of this year, few could have predicted the unprecedented market dislocation and rapid rebound. While the business environment was dramatically changed, innovative life sciences companies were part and parcel of the recovery and investors were at the ready. Join this conversation to hear two industry experts discuss this year's wild ride and what might be next.

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.