Welcome New Members

• 4MTherapeutics
• NemaGen Discoveries, Inc.
• Ubuntu Research Inc.
• Syzent Partners LLC
• TCG GreenChem Inc.
• And to the NJEDA for moving to an Executive Level Membership

 Learn more at www.BioNJ.org/Membership.

State Policymakers Can Protect American Innovation and Help Americans Live Longer, Healthier Lives

For the second year in a row, life expectancy is on the decline in America. The leading causes of death across the United States include cancer and chronic diseases. Even when patients are able to manage these conditions — e.g., overall cancer survivability has increased 33% since 1991 — their quality of life may still be diminished. To turn that tide, we need new treatments and cures that both extend life and improve it. Fortunately, America’s biopharmaceutical research companies are working to find innovative solutions that can help patients across the United States. We need this type of innovation to adequately address these leading causes of death. But we also need to address the leading reasons why patients can’t access and afford these innovative treatments. 

What are PBMs, Anyway?

Everyone’s talking about pharmacy benefit managers (PBMs) — including the Federal Trade Commission (FTC) — but what exactly are PBMs and why do they matter in the context of drug pricing? In February, the Federal Trade Commission (FTC) announced it is soliciting public input on how PBMs are affecting drug affordability and access, with the comment period closing on May 25. The term “PBM” has flown under the radar until recently — with patients, politicians and health advocacy groups starting to pay attention. These three little letters have a profound effect on patient costs and speak to an insidious insurance system that targets the sickest Americans, requiring them to pay more for the drugs they need to live and live well, while challenging the very validity of the title “health insurance.”

The State of Drug Development in America is Strong, but the IRA Unintentionally Weakens It

Americans today are benefiting from significant innovation across multiple therapeutic areas. We are on the cusp of highly effective treatments in obesity and important progress is being made in the fight against notoriously challenging diseases like Alzheimer’s. The state of drug development in America is strong. But we cannot take it for granted and we must protect it against short-sighted policies not in the best interest of patients. In President Biden’s State of the Union address, he touted the Inflation Reduction Act (IRA) as a major legislative accomplishment. However, within this sweeping legislation, the IRA fails by placing a drastic penalty on small molecule drug discovery, penalizing patient care along with it. Perhaps this was done out of legislative accident, but the fact remains that bad public policy is a great threat to biomedical innovation.

While Net Drug Prices Drop, Patients Pay More Thanks to Health Plans and PBMs

In 2022 drug manufacturers actually charged less for their products and patients paid slightly more than the previous year — but the cost of prescription medicines still rose less than inflation. While the media and policymakers continue to claim that drug prices are “skyrocketing,” the average change in list prices of brand name drugs in the U.S., which is what patients are paying for their medication when they are in their health plan’s deductible, has slowed sharply and has been less than 5% over that last four years, Drug Channels Institute finds. Meanwhile, the change in net prices (revenue after rebates and discounts) drug developers receive for brand-name drugs dropped over the last seven years and was negative over the last five.

Price Controls Put US Biopharma at Risk

Vital Transformation’s Grumpy Old Men discuss their recently published research which found that 60% of FDA approved medicines from 2011- 2020 originated in the United States. Their study, “The US Ecosystem for Medicines - How new drug innovations get to patients”, shows a profound shift in global innovation and highlights the success of U.S. biopharma in creating new medicines. VT’s Grumpy Old Men also highlight the many unintended consequences of the legislative proposals being made in Washington, DC to control the price of drugs, and the slippery slope that Europe is already traveling down due to similar policies. 

BioNJ President & CEO Debbie Hart Honored in the NJBIZ Power 100

The power lists are compiled by the NJBIZ editorial staff based on our reporting throughout the past year with input from experts in a variety of fields and recommendations from our readers. The staff looks for people who have gained public attention – and perhaps acclaim – for their professional accomplishments and public service. Congratulations to BioNJ President and CEO, Debbie Hart, for being honored on this list. "Hart is recognized as a respected thought leader and influential advocate collaborating with business, legislative, academic leaders and more to advance the life science industry, foster medical innovation and patient access while ensuring health equity and health care affordability. BioNJ’s motto is 'Because Patients Can’t Wait,' and patients are at the center of all of Hart’s efforts. In September, she was appointed by Gov. Phil Murphy as chair of the Commission on Science, Innovation and Technology." “Debbie has a long history of advocating for young, innovative companies in the Garden State and her decades of involvement has helped shape New Jersey innovation ecosystem and make it the ideal place for startups to grow and flourish,” said Governor Murphy.

Spotlight On: Debbie Hart, President & CEO, BioNJ

In an interview with Invest:, Debbie Hart, President & CEO of life sciences association BioNJ, talked about the current state of the industry specifically within NJ and efforts to ensure entrepreneurs are able to innovate and collaborate for the benefit of the public. Hart explained that one of her main goals is to increase health equity in clinical trials and what the organization has been doing to accomplish that.

Teva Announces FDA Approval of AUSTEDO® XR (deutetrabenazine) Extended-Release Tablets, a New Once-Daily Formulation of AUSTEDO® (deutetrabenazine) Tablets

Parsippany-based BioNJ Member Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., announced that the U.S. Food and Drug Administration (FDA) has approved AUSTEDOXR (deutetrabenazine) extended-release tablets, a new once-daily formulation indicated in adults for tardive dyskinesia (TD) and chorea associated with Huntington’s disease (HD). AUSTEDO XR is an additional formulation of the currently marketed twice-daily AUSTEDO.Once-daily AUSTEDO XR has been shown to be therapeutically equivalent to the twice-daily formulation, providing another effective treatment choice. It will be available in three extended-release tablet strengths – 6 mg, 12 mg, and 24 mg – and can be taken with or without food. The new tablet strengths provide an updated regimen which may result in a decreased pill count for patients compared to the twice-daily formulation.

Teva Announces Publication of New Clinician Scale in Journal of Clinical Psychiatry to Assess the Impact of Tardive Dyskinesia on Patients’ Lives

Parsippany-based BioNJ Member Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., announced the print publication of the IMPACT-TD Scale, an easy-to-use, standardized and clinician-rated assessment. The scale, developed by a consensus panel, will assist healthcare providers in determining the impact of tardive dyskinesia (TD) on different aspects of a patient’s daily functioning. IMPACT-TD categorizes the consequences of TD symptoms into multiple functional domains: social, psychological/psychiatric, physical and vocational/educational/recreational. The scale was developed to collect insights from not only patients but also family members, caregivers and providers, to help foster more productive dialogue and provide the most comprehensive understanding of the impact of TD.

EYLEA® (aflibercept) Injection Approved as the First Pharmacologic Treatment for Preterm Infants With Retinopathy of Prematurity (ROP) by the FDA

BioNJ Member Regeneron Pharmaceuticals, with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has approved EYLEA® (aflibercept) Injection to treat preterm infants with retinopathy of prematurity (ROP). Following this first pediatric approval, EYLEA is now indicated to treat five retinal conditions caused by ocular angiogenesis. “Retinopathy of prematurity is a leading cause of childhood blindness worldwide. Until now, the only FDA-approved treatment in common use was laser photocoagulation, a complex and lengthy procedure that permanently ablates retina tissue and is stressful not only for infant patients but also the family navigating a delicate time after a preterm birth,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron, and a principal inventor of EYLEA.

In a KRAS G12C-Mutated NSCLC Cell Line, Calu-1, Hillstream BioPharma Demonstrated Significantly Greater Tumor Inhibition Combining Pembrolizumab, an anti-PD-1 Antibody, and HSB-1216, a Ferroptosis Inducer

Bridgewater-based BioNJ Member Hillstream BioPharma, Inc. announced initial results evaluating the synergy of HSB-1216 with immune checkpoint inhibitors in KRAS G12C-mutated non-small cell lung cancer (NSCLC) cells, Calu-1, with Pembrolizumab, an anti-PD-1 antibody. The study demonstrated significantly greater tumor inhibition of Calu-1 cells, when grown with human PBMCs (SEB-activated) and treated with HSB-1216 and Pembrolizumab. “We are greatly encouraged by the initial results showing crosstalk and synergy between an established and validated immuno-oncology pathway, anti-PD-1, and Ferroptosis, a new emerging iron-dependent cell death pathway and mechanism of action for HSB-1216,” said Randy Milby, Hillstream’s CEO. Lung cancer remains one of the leading causes of cancer-related deaths worldwide with NSCLC accounting for approximately 80–85% of all cases. 

Hillstream BioPharma Signs Exclusive Option Agreement With Applied Biomedical Science Institute (ABSI) to License Technology for HER2 and HER3 to be Developed for Potential Treatments Against Drug Resistant Cancers Including HER2-Positive Metastatic Breast Cancer, Gastric Cancer, Lung Cancer and Ovarian Cancer

Bridgewater-based BioNJ Member Hillstream BioPharma, Inc. announced signing an exclusive option agreement with Applied Biomedical Science Institute (ABSI) to license technology for HER2 and HER3 Conformational Domain Bridging Epitopes in human monoclonal antibodies to develop proprietary multi-format biologics (bi- and tri-specific antibodies, ADCs (antibody drug conjugates), CAR-T and CAR-NKs, in Quatramers and Quatrabodies) against drug resistant cancers including HER2-positive metastatic breast cancer, gastric cancer, lung cancer and ovarian cancer . “We look forward to this unique opportunity to work with Dr. Smider and the Applied Biomedical Science Institute,” said Randy Milby, CEO of Hillstream. “This agreement allows Hillstream to build our antibody platform to advance our immune-oncology biologic pipeline with HER2 and HER3 agents targeting highly aggressive drug resistant metastatic tumors, representing a major unmet need for patients.” 

Opdivo® (nivolumab) in Combination With CABOMETYX® (cabozantinib) Shows Durable Survival With Over Three Years of Follow-Up in the CheckMate -9ER Trial in First-Line Advanced Renal Cell Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb and Exelixis, Inc. announced three-year (36.5 months minimum; 44.0 months median) follow-up results from the Phase 3 CheckMate -9ER trial, demonstrating sustained survival and response rate benefits with the combination of Opdivo® (nivolumab) and CABOMETYX® (cabozantinib) versus sunitinib in the first-line treatment of advanced renal cell carcinoma (RCC). Additionally, a biomarker analysis showed that improvements in median progression-free survival (PFS) and overall survival (OS) were sustained with the combination of Opdivo and CABOMETYX regardless of PD-L1 status. Mauricio Burotto, M.D., Medical Director, Bradford Hill Clinical Research Center, Santiago, Chile, said “With these updated results from CheckMate -9ER, we’ve now seen nivolumab in combination with cabozantinib durably extend survival and sustain response benefits compared to sunitinib for over three years, regardless of patients’ risk classification.” 

Abecma (idecabtagene vicleucel) Reduced the Risk of Disease Progression or Death by 51% Versus Standard Regimens in Earlier Lines of Therapy for Relapsed and Refractory Multiple Myeloma Based on Results from Phase 3 KarMMa-3 Study

Princeton-based BioNJ Member Bristol Myers Squibb and 2seventy bio, Inc. announced the first publication and presentation of positive results from KarMMa-3, a pivotal Phase 3, open-label, global, randomized, controlled study evaluating Abecma (idecabtagene vicleucel) compared with standard combination regimens in adults with relapsed and refractory multiple myeloma after two to four prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody, and who were refractory to their last regimen. Data from KarMMa-3 are being published in The New England Journal of Medicine. At a median follow up of 18.6 months, treatment with Abecma (n=254) demonstrated a clinically meaningful and statistically significant improvement in the primary endpoint of progression-free survival (PFS) compared with standard regimens (n=132), with a median PFS of 13.3 months (95% CI: 11.8-16.1) vs. 4.4 months (95% CI: 3.4-5.9), respectively (HR:0.49; p<0.0001). 

Jubilant Therapeutics Inc. Receives Orphan Drug Designation for the PRMT5 inhibitor - JBI-778 for the Treatment of Glioblastoma Multiforme (GBM)

Bedminster-based BioNJ Member Jubilant Therapeutics Inc. announced that the United States Food and Drug Administration (US FDA) has granted Orphan Drug Designation (ODD) for JBI-778 for the treatment of Glioblastoma Multiforme (GBM). JBI-778 is an oral, brain penetrant and substrate-competitive protein arginine methyl transferase 5 (PRMT5) inhibitor for the treatment of tumors with brain metastases and primary brain tumors including high-grade glioma. JBI-778 is the company's second drug candidate to receive ODD along with JBI-802, which received ODD for the treatment of small cell lung cancer (SCLC) and acute myeloid leukemia (AML). "JBI-778 is our highly differentiated, substrate competitive PRMT5 inhibitor in development for both systemic and brain tumors with certain genetic signatures. It is the second clinical stage oral drug candidate that has emerged from the TIBEO discovery engine," said Syed Kazmi, CEO, Jubilant Therapeutics Inc. 

First Wave BioPharma Announces Phase 2 Adrulipase Combination Trial Data to be Presented at the 2023 Digestive Disease Week (DDW) Conference

BioNJ Member First Wave BioPharma, Inc. announced that an abstract has been accepted for a presentation at the 2023 Digestive Disease Week Conference. The presentation will share data from the Phase 2 adrulipase Combination Therapy Trial which evaluated the safety and efficacy of adrulipase in combination with the current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT), for the treatment of severe EPI in patients with CF. “The research presented at DDW 2023 is intended to highlight the potential benefits that adrulipase may offer in the treatment of EPI above and beyond PERT, the current standard of care,” stated James Sapirstein, President and CEO of First Wave BioPharma.

FDA Approves Once-Weekly ALTUVIIIO™ , a New Class of Factor VIII Therapy for Hemophilia A that Offers Significant Bleed Protection 

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Proteinehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. ALTUVIIIO is the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing, and significantly reduces bleeds compared to prior factor VIII prophylaxis.

New Data for Tolebrutinib, Sanofi’s Investigational Brainpenetrant BTK Inhibitor, Showed Significant Effect on CNS Immune Mediators that Drive MS Disease Progression

Bridgewater-based BioNJ Member Sanofi announced new data for tolebrutinib, Sanofi’s investigational oral Bruton’s tyrosine kinase (BTK) inhibitor for the treatment of multiple sclerosis (MS), showed a significant effect on key immune mediators that may drive disease progression within the CNS. In the study, researchers measured proteomic changes in the cerebrospinal fluid of MS patients treated with tolebrutinib, compared to patients receiving either no treatment or the B-cell depleting therapy, ocrelizumab. These findings stem from a collaborative research and development partnership with the National Institute of Neurological Disorders and Stroke (NINDS).

Lilly and IABL Collaborate to Ensure Patient Access to High-Quality Affordable Insulin in Bangladesh

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced it will supply its active pharmaceutical ingredient (API) for human insulin at a reduced price to International Agencies (Bangladesh) Ltd. (IABL) in an effort to increase patient access and improve affordability for high-quality insulin for nearly one million people living with diabetes in Bangladesh by 2030. IABL will formulate, fill and finish human insulin vials and cartridges under its own trademark and brand name by 2025. The IABL-produced insulin will be exclusively for the Bangladesh market. "Lilly is committed to addressing the critical gaps in access to essential medicines for people living with diabetes in low- and middle-income countries," said Ilya Yuffa, President of Lilly International. 

Pfizer and BioNTech Submit Supplemental Biologics License Application for U.S. FDA Approval of Omicron BA.4/BA.5-Adapted Bivalent COVID-19 Vaccine for Ages 12 Years and Older as Primary Series or Booster

BioNJ Member Pfizer, with a site in Peapack, and BioNTech SE announced they have submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for approval of their Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine as a primary series and booster dose(s) for individuals 12 years of age and older. On January 26, 2023, the U.S. FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted to harmonize the composition of COVID-19 vaccines across booster and primary series doses. If this sBLA is approved, people 12 years or older would be able to receive the companies’ Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine for their primary series, rather than completing their primary series with the original vaccine (COMIRNATY® (COVID-19 Vaccine, mRNA)) before having access to the bivalent vaccine.

Pfizer Announces Positive TALZENNA® and XTANDI® Combination Data from Phase 3 TALAPRO-2 Study

BioNJ Member Pfizer, with a site in Peapack, announced positive results from the Phase 3 TALAPRO-2 study of TALZENNA® (talazoparib), an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with XTANDI® (enzalutamide), demonstrating a statistically significant and clinically meaningful improvement in radiographic progression-free survival (rPFS) compared to placebo plus XTANDI in men with metastatic castration-resistant prostate cancer (mCRPC), with or without homologous recombination repair (HRR) gene mutations. In addition, the U.S. Food and Drug Administration (FDA) has granted Priority Review for Pfizer’s supplemental new drug application (sNDA) for TALZENNA in combination with XTANDI for the treatment of men with mCRPC. The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists. 

FDA Approves Pfizer's Supplemental New Drug Application for CIBINQO® (abrocitinib)

BioNJ Member Pfizer, with a site in Peapack, announced that the United States (U.S.) Food and Drug Administration (FDA) approved its supplemental New Drug Application (sNDA) for CIBINQO® (abrocitinib), expanding its indication to include adolescents (12 to <18 years) with refractory, moderate-to-severe atopic dermatitis (AD) whose disease is not adequately controlled with other systemic drug products, including biologics, or when use of those therapies is inadvisable. CIBINQO was previously approved only for the treatment of adults 18 years and older. Data from the robust clinical trial program included in the prescribing information now stems from five randomized, placebo-controlled clinical trials and a long-term extension study with more than 1,600 patients treated with CIBINQO. 

Pfizer Announces The Lancet Neurology has Published Phase 3 Data for Zavegepant for the Acute Treatment of Migraine in Adults

BioNJ Member Pfizer, with a site in Peapack, announced publication of results in The Lancet Neurology from the Phase 3 pivotal clinical trial of zavegepant, an investigational calcitonin gene-related peptide (CGRP) receptor antagonist nasal spray for the acute treatment of migraine. The study met its co-primary endpoints, showing that a single 10 mg dose of zavegepant was more effective than placebo for both pain freedom and freedom from the most bothersome symptom (MBS) at two hours post-dose. Additionally, zavegepant demonstrated relief from migraine pain in 15 minutes, with relief lasting up to 48 hours for many patients. Zavegepant was well tolerated, and there were no serious adverse events reported in treated participants.

Pfizer’s Elranatamab Receives FDA and EMA Filing Acceptance

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for the company’s Biologics License Application (BLA) for elranatamab, an investigational B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb), for the treatment of patients with relapsed or refractory multiple myeloma (RRMM). Priority Review is intended to direct attention and resources from regulatory authorities toward drugs that, if approved, could offer significant improvements over existing options for serious conditions in order to make these drugs available to patients faster. The FDA’s decision on the application is expected in 2023. The European Medicines Agency (EMA) has also accepted elranatamab’s marketing authorization application (MAA). The company is working closely with the EMA to facilitate their review and will provide updates on timing as appropriate. 

U.S. FDA Accepts Biologics License Application for Pfizer’s Respiratory Syncytial Virus Maternal Vaccine Candidate for Priority Review

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for its respiratory syncytial virus (RSV) vaccine candidate PF-06928316 or RSVpreF for the prevention of medically attended lower respiratory tract illness (MA-LRTI) and severe MA-LRTI caused by RSV in infants from birth up to six months of age by active immunization of pregnant individuals. This decision follows the FDA’s Breakthrough Therapy Designation for RSVpreF in March 2022. The FDA has accepted the BLA for priority review and has set a Prescription Drug User Fee Act (PDUFA) action date of August 2023. In addition, the European Medicines Agency (EMA) has accepted Pfizer’s marketing authorization application (MAA) under accelerated assessment for its RSV vaccine candidate for both older adults and maternal immunization to help protect infants. 

Gilead Presents Positive Proof-of-Concept Data for Investigational Combination Regimen of Lenacapavir With Broadly Neutralizing Antibodies as a Potential Twice-Yearly Approach for the Treatment of HIV

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced data evaluating lenacapavir in combination with broadly neutralizing antibodies (bNAbs) teropavimab and zinlirvimab as a potential long-acting treatment regimen with twice-yearly dosing. Results from the Phase 1b clinical trial demonstrated the investigational combination was generally well tolerated with high efficacy in select virologically suppressed participants living with HIV. The study evaluated the safety and efficacy profile of lenacapavir + teropavimab + zinlirvimab in selected adults living with HIV who were virologically suppressed (HIV-1 RNA <50 copies/mL) for ≥2 years while taking antiretroviral therapy (ART). Study participants (n=20) were sensitive to both bNAbs by HIV proviral DNA phenotype and had a CD4 cell count of ≥500 at study entry. 

AbbVie and Capsida Biotherapeutics Expand Strategic Collaboration to Develop Targeted Genetic Medicines for Eye Diseases With High Unmet Need

BioNJ Member AbbVie, with a site in Princeton, and Capsida Biotherapeutics Inc. announced an expanded strategic collaboration to develop genetic medicines for eye diseases with high unmet need. AbbVie's extensive capabilities will be paired with Capsida's novel adeno-associated virus (AAV) engineering platform and manufacturing capability to identify and advance three programs. The collaboration builds upon the neurodegenerative disease partnership announced in 2021. "This expanded collaboration with Capsida has the potential to develop transformative therapies for patients with serious eye diseases," said Jonathon Sedgwick, Ph.D., Vice President and Global Head of Discovery Research, AbbVie. "In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner."

Merck and Moderna Announce mRNA-4157/V940, an Investigational Personalized mRNA Cancer Vaccine, in Combination With KEYTRUDA® (pembrolizumab), was Granted Breakthrough Therapy Designation by the FDA for Adjuvant Treatment of Patients With High-Risk Melanoma Following Complete Resection

Rahway-based BioNJ Member Merck & Co. and Moderna, Inc. announced that mRNA-4157/V940, an investigational personalized mRNA cancer vaccine, in combination with KEYTRUDA, Merck’s anti-PD-1 therapy, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of patients with high-risk melanoma following complete resection. The FDA granted Breakthrough Therapy Designation based on positive data from the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial. “This is an important milestone in the development of mRNA-4157/V940 in combination with KEYTRUDA,” said Dr. Eric H. Rubin, Senior Vice President, Global Clinical Development, Merck Research Laboratories. “We look forward to working with the FDA, in collaboration with Moderna, to conduct a rigorous and rapid clinical development program with a focus on addressing the needs of this important patient population.”

Merck’s KEYTRUDA® (pembrolizumab) Plus Chemotherapy Significantly Improved Overall Survival Versus Chemotherapy Alone in Patients With HER2-Negative Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma Regardless of PD-L1 Expression

Rahway-based BioNJ Member Merck & Co. announced results from the pivotal Phase 3 KEYNOTE-859 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of patients with human epidermal growth factor receptor 2 (HER2)-negative locally advanced unresectable or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma. After a median follow-up of 31.0 months (range, 15.3-46.3 months), KEYTRUDA in combination with chemotherapy significantly improved overall survival (OS), reducing the risk of death by 22% (HR=0.78 [95% CI, 0.70-0.87]; p<0.0001) compared to chemotherapy alone for these patients, regardless of PD-L1 expression. Median OS was 12.9 months (95% CI, 11.9-14.0) for KEYTRUDA plus chemotherapy versus 11.5 months (95% CI, 10.6-12.1) for chemotherapy alone.  

U.S. FDA Accepts for Priority Review the Supplemental New Drug Application for Merck’s PREVYMIS™ for Prophylaxis of Cytomegalovirus Disease in Kidney Transplant Recipients at High Risk

Rahway-based BioNJ Member Merck & Co. announced the U.S. Food and Drug Administration (FDA) has accepted for review two supplemental new drug applications (sNDA) for PREVYMIS™ (letermovir). The FDA granted priority review for the sNDA for PREVYMIS for prophylaxis of cytomegalovirus (CMV) disease in adult kidney transplant recipients at high risk (D+/R-); the Prescription Drug User Fee Act (PDUFA), or target action date, is June 5, 2023. The FDA grants priority review to medicines and vaccines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment or prevention of a serious condition. A second sNDA to extend use of PREVYMIS from 100 days to 200 days in adults receiving an allogeneic hematopoietic stem cell transplant (HSCT) who are at risk for late CMV infection and disease was also accepted for review. 

Merck Opens Enrollment in New Phase 3 Clinical Trials With Investigational Once-Daily Islatravir in Combination with Doravirine for Treatment of HIV-1 Infection

Rahway-based BioNJ Member Merck & Co. announced that the company has opened enrollment in its new Phase 3 clinical program with investigational once-daily islatravir 0.25 mg in combination with doravirine 100 mg (DOR/ISL) for the treatment of HIV-1 infection. “Our extensive efficacy and safety analyses from the islatravir clinical program over the past year provided critical information to shape our research going forward,” said Todd Correll, Executive Director, DOR/ISL Product Development Team Lead, Merck Research Laboratories. “We believe islatravir in combination with doravirine has the potential to help make a difference for people living with HIV and are excited to open enrollment in our new Phase 3 clinical trials.” 

Janssen Presents Updated Data Demonstrating Improved Outcomes from the Use of Niraparib in Combination With Abiraterone Acetate Plus Prednisone as a First-Line Therapy in Patients With BRCA-Positive Metastatic Castration-Resistant Prostate Cancer

New Brunswick-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced updated results from the Phase 3 MAGNITUDE study evaluating the investigational use of niraparib, a highly selective poly (ADP-ribose) polymerase (PARP) inhibitor, in combination with abiraterone acetate plus prednisone (AAP) in patients with metastatic castration-resistant prostate cancer (mCRPC) with or without specific homologous recombination repair (HRR) gene alterations, including BRCA mutations. “Patients with HRR-positive mCRPC, especially those with BRCA mutations, are more likely to experience poor outcomes. Although additional follow-up for overall survival continues, it is encouraging to see a trend toward improvement in overall survival among patients with BRCA-positive mCRPC who received niraparib and AAP as compared to placebo and AAP,” said Kim Chi, M.D., Medical Oncologist at BC Cancer - Vancouver and principal investigator of the MAGNITUDE study. 

BD Onclarity™ HPV Assay Receives FDA Approval for Use With Both BD SurePath™ Liquid-based Pap Test and Hologic ThinPrep® Pap Test

Franklin Lakes-based BD (Becton, Dickinson and Company) announced U.S. Food and Drug Administration (FDA) market approval for the BD Onclarity™ HPV Assay to be used with the ThinPrep® Pap Test. The BD SurePath™ Liquid-based Pap Test vial and the Hologic ThinPrep® Pap Test PreservCyt® Solution vial are the two most common Pap vials used by laboratories in the United States. The inclusion of the ThinPrep® Pap Test improves access to the benefits of the BD human papillomavirus virus (HPV) assay, which is the only FDA-approved assay that tests for an extended set of HPV types individually, and particularly for HPV31, a specific type of HPV that poses a high-risk for causing cervical cancer. 

BD Receives FDA Emergency Use Authorization for COVID-19, Influenza A/B, RSV Combination Test

Franklin Lakes-based BD (Becton, Dickinson and Company) announced that it has received Emergency Use Authorization from the U.S. Food and Drug Administration (FDA) for a new molecular diagnostic combination test for SARS-CoV-2, Influenza A + B and Respiratory Syncytial Virus (RSV) to help combat illness in the current and future respiratory virus seasons.

The test, for use on the BD MAX™ Molecular Diagnostic System, uses a single nasal swab or a single nasopharyngeal swab sample to identify and distinguish if a patient has COVID-19, the flu, RSV or some combination of the three, with results available in as little as two hours. The test helps eliminate the need for multiple tests or doctor visits and can help clinicians implement the right treatment plan quickly. 

LEO Pharma Announces Positive Phase 3 Topline Results from DELTA 2 Trial With Delgocitinib Cream in Adults With Moderate to Severe Chronic Hand Eczema (CHE), Confirming the Positive Results of the Recent DELTA 1 Trial

LEO Pharma, with a site in Madison, announced positive results of the DELTA 2 trial. DELTA 2 is the second of two pivotal Phase 3 clinical trials with delgocitinib cream, an investigational topical pan-Janus kinase (JAK)-inhibitor, for the potential treatment of adults with moderate to severe chronic hand eczema (CHE). The trial met its primary endpoint with a statistically significant improvement in CHE after 16 weeks of treatment with delgocitinib cream compared to cream vehicle, and the treatment was well-tolerated. All or most of the signs and symptoms of CHE were cleared early in the treatment period for a significantly larger proportion of patients treated with delgocitinib cream compared to subjects treated with cream vehicle. 

Shionogi Advances Ensitrelvir Fumaric Acid COVID-19 Antiviral Clinical Program

Shionogi & Co., Ltd., with a site in Florham Park, announced progress on its comprehensive clinical development program for the novel COVID-19 oral antiviral ensitrelvir (Generic name: ensitrelvir fumaric acid, Code No.: S-217622, hereafter “ensitrelvir”). The program includes several Phase 3 clinical studies evaluating ensitrelvir’s safety and effectiveness across a wide range of COVID-19 patient populations. Ensitrelvir, known as Xocova® in Japan, recently received emergency regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for SARS-CoV-2 infection based on the results obtained through the Phase 3 part of the Phase 2/3 study in non-hospitalized patients conducted in Asia. It remains an investigational drug in the rest of the world.

Clinical Data Presented at ASCO GU Demonstrates Reduced Risk of Recurrence in Non-Muscle Invasive Bladder Cancer With the Use of BLC

Photocure ASA, The Bladder Cancer Company, with a site in Princeton, announced the presentation of clinical data from the Veterans Affairs (VA) BRAVO study at the ASCO Genitourinary Cancers Symposium. The clinical data demonstrated a significant decrease in the risk of recurrence & longer time to recurrence following Blue Light cystoscopy (BLC®) with Cysview® compared to White Light cystoscopy (WLC) alone. Dr. Steven Williams, University of Texas-Medical Branch, Galveston, presented the study abstract “The Impact of Blue Light Cystoscopy Use Among Non-Muscle Invasive Bladder Cancer Patients in an Equal Access Setting: Implications on Recurrence and Time to Recurrence Stratified by Race”. 

Octapharma USA Requests FDA Approval for wilate® VWD Prophylaxis Supplement

Paramus-based Octapharma USA has submitted a Biologics License Application Supplement (sBLA) to the U.S. Food and Drug Administration (FDA) to expand the approval of wilate®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, to include routine prophylaxis to reduce the frequency of bleeding episodes in children and adults with any type of von Willebrand disease (VWD). The application is supported by the results of Octapharma's WIL-31 study, a prospective, non-controlled, international, multicenter phase 3 trial that investigated the efficacy and safety of wilate® prophylaxis over 12 months in people of age 6 and older with severe VWD of any type except type 2N. VWD is the most common bleeding disorder, found in up to 1% of the U.S. population.

Provention Bio Closes $35M Investment from Sanofi

Red Bank-based Provention Bio announced the closing of a $35 million equity investment from Bridgewater-based BioNJ Member Sanofi US. According to Jason Hoitt, Chief Commercial Officer of Provention Bio, the investment will support the commercial launch of TZIELD (teplizumab-mzwv), which he described as “the first immunomodulatory therapy approved to delay the onset of Stage 3 type 1 diabetes (T1D) in adults and pediatric patients aged 8 years and older with stage 2 T1D.” “TZIELD’s recent FDA approval advances our purpose to intercept autoimmunity in its earlier stages, rather than waiting to manage end-stage disease when symptoms, irreversible tissue damage and serious complications appear,” Hoitt continued. Olivier Bogillot, Sanofi’s Head of U.S. General Medicines, said the company was excited to support Provention Bio for the U.S. commercial launch of TZIELD.

Presenting the 2023 NJBIZ Power 100

Congratulations to all our members honored on this years NJBIZ Power 100 List! The power lists are compiled by the NJBIZ editorial staff based on our reporting throughout the past year with input from experts in a variety of fields and recommendations from our readers. The staff looks for people who have gained public attention – and perhaps acclaim – for their professional accomplishments and public service. In addition to BioNJ President & CEO Debbie Hart, congratulations to these BioNJ Members for making the list:

Celldex Therapeutics Announces Appointment of Rita Jain, M.D. to Board of Directors

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced that it has appointed Rita Jain, M.D., to the company’s Board of Directors. “We are pleased to welcome Dr. Jain to the Celldex Board of Directors at this important time in the company’s trajectory,” said Anthony Marucci, Co-founder, President and CEO of Celldex Therapeutics. “Dr. Jain’s deep background in drug development strongly complements our Board’s skills and experiences, and we look forward to her contributions as we continue to advance our programs into later stage development.” Dr. Jain is a board-certified rheumatologist. She previously served as Executive Vice President, Chief Medical Officer of ChemoCentryx, Inc., Chief Medical Officer of Immunovant, Inc. and prior to that, Senior Vice President and Chief Medical Officer of Akebia Therapeutics, Inc.

Novartis Announces the Appointment of the Chairman-Designate of Sandoz Board of Directors

East Hanover-based BioNJ Member Novartis announced the appointment of Gilbert Ghostine as Chairman-Designate of the new Board of Directors of Sandoz. Mr. Ghostine has served as CEO of Geneva-based Firmenich, the world's largest privately owned perfume and taste company, since 2014. Mr. Ghostine will become Chairman of the New Board of Directors of Sandoz, which will be formed following the spin-off from Novartis in the second half of 2023, subject to final Novartis Board of Directors and shareholder approvals. Mr. Ghostine is an experienced business leader with a track record of growing and transforming businesses in competitive industries. He held executive and senior leadership positions at Firmenich and Diageo in a career spanning three decades. 

Lauded Biotech Vet to Lead Johnson & Johnson Team

Biomedical industry veteran Dr. John Reed will join BioNJ Member Johnson & Johnson as Executive Vice President of Pharmaceuticals, R&D, the New Brunswick pharmaceutical giant announced. Bringing more than 35 years of biomedical research leadership to the role, Mr. Reed will also be a member of the company’s executive committee. Mr. Reed comes to Johnson & Johnson from Sanofi where he was Executive Vice President, Global Head of Research and Development since 2018. “Dr. Reed is an accomplished leader and highly regarded researcher with an extraordinary track record for discovering and developing life-saving innovations on a global scale,” said Johnson & Johnson Chairman and CEO Joaquin Duato. 

Johnson & Johnson Names Paula A. Johnson, President of Wellesley College, to its Board of Directors

New Brunswick-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced that Paula A. Johnson, M.D., MPH, President of Wellesley College, has been appointed to its Board of Directors. “I am excited to welcome Dr. Paula Johnson to Johnson & Johnson’s Board of Directors,” said Joaquin Duato, Chairman and CEO, Johnson & Johnson. “Paula is an impressive leader, physician-scientist and educator, with expertise in improving health outcomes for patients around the globe. Her extensive knowledge of medical research, public health and health policy, and her proven leadership across complex organizations will provide a unique skillset to the Johnson & Johnson Board of Directors.”

Former Bausch + Lomb CEO Returns to Lead Company

Bausch + Lomb Corp., with a site, in Bridgewater named Brent Saunders as Chief Executive Officer and Chair of the Board of Directors. Mr. Saunders succeeds Joseph Papa, who announced in July 2022 that he would step down. Additionally, Thomas Ross Sr., who was appointed Board Chair last summer, will become the Lead Independent Director of the Board for the eye health company. Having previously served as Bausch + Lomb CEO from 2010 to 2013, Mr. Saunders will rejoin the company in an advisory capacity to ensure a smooth transition. “Mr. Saunder’s strong inner-working knowledge of the company and unparalleled executive leadership experience in health care make him the ideal person to lead Bausch + Lomb at this pivotal time in our 170-year history,” Mr. Ross. 

CSIT Announces $2.4M+ Awarded Since Small Business Assistance Program Launched

The New Jersey Commission on Science, Innovation and Technology (CSIT) has awarded a total of $2.425 million in matching grants to more than 80 start-ups since 2020 through its NJ Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) Direct Financial Assistance Program. The program supports start-ups that are receiving funding from highly competitive federal research and development (R&D) grant programs as they work toward commercialization. The $2.425 million in funding includes $525,000 that was awarded to 17 start-ups through the program’s fourth funding round.

“This grant program is specifically designed to support the smallest of startups competing in the global marketplace,” said CSIT Chair Debbie Hart. “By helping grantees maximize their federal funds, we are enabling them to scale their operations and focus on commercializing their products. This, in turn, will lead to grantees expanding their presence and creating good paying jobs in the Garden State.”

N.J. Life Sciences Building Back Better: Massive HELIX, Merck Sites Aim to be Complementary

When the HELIX, a 12-story, 573,400-square-foot building in New Brunswick that is committed to doing translational research in life sciences, received a $271 million tax credit to get the building off the ground, State officials said the massive complex would be a game-changer. Less than two weeks later, Onyx Equities announced it was buying the 108-acre Merck campus in Kenilworth — one already filled with 1.4 million square feet of state-of-the-art research facilities. In response, New Brunswick Development Corp. President Chris Paladino, the master developer of the HELIX, said “I think it’s extraordinarily complementary. Since the earliest phases of the HELIX project and the New Jersey Innovation Hub, this has been the desire: To have a place that can help create and accelerate the commercialization of research by start-ups and early-stage companies. What we’re trying to do is make New Jersey a driver of the country’s innovation economy. So, I think these places, and others like them, are very compatible.”

Rutgers Signs Exclusive License to Market Innovative DNA Analysis Technology

Rutgers Office for Research has signed an exclusive license with SoftGenetics, LLC for the company to commercialize innovative software technology developed by Rutgers-Camden professors Catherine Grgicak, Ph.D. and Desmond Lun, Ph.D.. The technology, called NOCIt, is a computational tool that calculates the probability of the number of contributors in a DNA profile. “One of the challenges that forensic crime laboratories have is estimating the number of contributors, which is required before they take the next step: calculating the weight of evidence against the suspect,” said Grgicak. “Many labs are still estimating that number of contributors manually. What Desmond and I and our team developed is software that estimates the number of contributors quantitatively. We use as much of the information as is available to estimate that number of contributors.”

Six Innovation Strategies for Life Sciences Organizations in 2023

IBM Business Transformation Blog

Last year, as life sciences organizations were consumed by the recovery from COVID-19, their focus had to shift rapidly to mitigating supply chain constraints, labor and skill shortages, and by the end of the year, inflationary pressures — all of which were exacerbated by the Russia-Ukraine war. Along with these challenges, the ongoing drive to reduce costs, improve efficiency and productivity, drive better decision-making and reduce risk will continue to drive pharma investment in cloud, AI/ML, analytics and automation in 2023 despite higher interest rates.

Biopharma Finances: Bringing A New Drug To Market

By Kathryn McDonough, Co-Head of Healthcare for Middle Market Banking & Specialized Industries, J.P. Morgan Commercial Banking

Biopharma companies face a long journey with each drug they research, test and — if all goes right — bring to market. And just as new drug development moves forward in phases, so too should your organization’s financial strategy. American pharmaceutical companies spent more than $90 billion in R&D in 2020 according to the National Center for Science and Engineering Statistics’ Business Enterprise Research and Development Survey.

TiE New Jersey Gala

February 25, 2023

Annual Gala & Awards Night at The Mansion at Natirar

400 Natirar Drive, Peapack-Gladstone, NJ 07977

NORD New Jersey Rare Action Network Rare Disease Day

February 28, 2023

One of the greatest challenges individuals living with a rare disease, their families, and patient advocates have is finding and accessing information and resources to help them advocate for themselves and their loved ones, as well as to raise awareness of issues facing others in the community. To help alleviate these daily burdens and ensure individuals and advocates can navigate local and state-based resources, NORD New Jersey Rare Action Network will be hosting a State Resource Fair for the rare disease community to coincide with Rare Disease Day. There is no cost to participants to attend this event.

The Entrepreneur's Journey: How to Prepare for Fundraising

March 9, 2023

Panel Discussion — “The Entrepreneur’s Journey: How to Prepare for Fundraising”  

Hybrid, with in person portion at Princeton Innovation Center BioLabs. This event will be about the nuts and bolts of what you need to get ready to start raising funds for your startup. You will hear from both the founder and the funder side. Bring your questions. The panelists are from the Life Sciences, but many of the details are applicable to all industries. The event will be followed by a networking reception — all are welcome.

IP & Licensing Basics: A One Day Review

March 20, 2023

Whether you are an entrepreneur in the making, a principal investigator or student at any level who wants a better grasp of the basics, or an aspiring technology transfer professional, this course will give you the tools you need to understand and participate effectively in the process of protecting IP and facilitating its commercialization. Delivered over one day, this course consists of 4 modules: IP Basics; IP Commercialization Basics; Licensing Fees & Royalty Rates and Managing Risk.

ON Helix

July 6, 2023

This one-day conference will address key bio innovation trends, from developments in life sciences and technology research to their translation into new diagnostics, prevention tools or treatments. Delegates and Supporters will connect with the One Nucleus network to explore New Horizons for Bio Innovation. BioNJ Members enjoy a 10% discount. Contact RBromberg@BioNJ.org for the discount code.