Congratulations to BioNJ Member Cellares on announcing that it will be building a 118,000-square-foot manufacturing facility in Bridgewater thanks to $255 million in Series C funding. The commercial-scale smart factory has the potential to be a game-changer for cancer patients in the State, in the country and around the world. “New Jersey is a hub in the U.S. for biopharmaceutical manufacturing. We found a great talent pool, great infrastructure, plenty of large facilities available and proximity to a great number of customers," said co-founder and CEO Fabian Gerlinghau.

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• ApiJect Systems, Corp.
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Learn more at www.BioNJ.org/Membership.

Without Clinical Trial Diversity, We’re ‘Losing a Lot of Opportunity’

In 2022, Congress enacted the Food and Drug Omnibus Reform Act, also known as FDORA, which requires drug sponsors to submit diversity action plans to the U.S. Food and Drug Administration (FDA). A recent BlackDoctor.org Facebook Live panel discussion sponsored by the Biotechnology Innovation Organization (BIO) explored the progress since then, and what more needs to be achieved. “We are losing a lot of opportunity by having clinical trials underrepresent certain groups,” said Dr. Ted W. Love, Chair of BIO and former President and CEO of Global Blood Therapeutics. “We don’t get the science from studying those populations—and sometimes the science could be different, and we don’t know until we study it.”

With the First List of Medicines Coming, Let’s Not Forget Why Government Price Setting is Bad Policy

CMS will soon release the first list of medicines that will be subject to government price setting in Medicare — an arbitrary and politicized process being launched under an extremely compressed timetable. Just a little over a year after the IRA passed, the government is ushering in mandated prices for medicines that will focus on short-term political objectives rather than the real-world needs of current and future Medicare beneficiaries. Here are three key reasons why this is just bad policy: 1) Seniors may not benefit from government price setting and could see their access restricted, 2) It ignores the existing savings from private sector negotiation and competition in Part D, and 3) The government’s arbitrary process for deciding the price of medicines will drive research and investment away from potentially lifesaving treatment options for Medicare patients. 

Enzene Biosciences Expanding to New Jersey With $50M Investment in Bio Manufacturing Plant

Enzene Biosciences, Ltd., a global biotech company based in Pune, India, will open its state-of-the-art biopharmaceutical manufacturing plant on the Princeton West Innovation Campus in Hopewell. Operations in the new facility, the company’s first location outside of India, are expected to begin in June 2024. As a Contract Development and Manufacturing Organization (CDMO), Enzene Biosciences provides services to global pharmaceutical companies, partnering with them in their drug development and manufacturing processes. As part of Enzene’s expansion plans, the company will initially hire 50 employees and grow to 300 employees at its New Jersey facility.  “We are thrilled that Enzene Biosciences has chosen Princeton as the location for its new biomanufacturing facility – Enzene’s only U.S. site,” said Debbie Hart, BioNJ President and CEO. “With its extensive drug development know-how and experience, Enzene will be a valuable addition to New Jersey’s robust life sciences ecosystem – bringing innovative medicines to Patients around the world. A leading biomedical hub, the Garden State boasts a full complement of large and small biopharmaceutical companies, universities, research institutions, clinical research organizations and service providers working together to make meaningful advances in healthcare.”  

Lactiga Receives FDA 'Rare Pediatric Disease' Designation for Groundbreaking Mucosal Immunoglobulin Therapy for Common Variable Immunodeficiency Disease

New Brunswick-based BioNJ Member Lactiga US, Inc. announced that the U.S. Food and Drug Administration has granted "Rare Pediatric Disease" (RPD) designation for its biologic, LCTG-001, a mucosal antibody replacement therapy comprised of polyclonal Immunoglobulin A (IgA) for nasal administration in patients with Common Variable Immunodeficiency (CVID). This significant milestone represents a major step forward in Lactiga's mission to transform the management of rare and life-threatening immunodeficiencies, especially in pediatric patients. “This is the start of Lactiga's ambitious mission to shift the paradigm of therapeutic antibody development from systemic to mucosal delivery, where many pathogens initially enter the body. By targeting the mucosal immune system, we can create more effective therapies that prevent infection at the point of entry, leading to a much better quality of life, especially in our most vulnerable populations," said Dr. Rikin Mehta, Lactiga Co-founder and CEO.

Iveric Bio Receives U.S. FDA Approval for IZERVAY™ (avacincaptad pegol intravitreal solution), a New Treatment for Geographic Atrophy

Cranbury-based BioNJ Member Iveric Bio/Astellas Pharma announced the U.S. Food and Drug Administration (FDA) approved IZERVAY™ (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) on August 4, 2023. IZERVAY, a new complement C5 inhibitor, is the only approved GA treatment with a statistically significant reduction (p<0.01) in the rate of GA progression at the 12-month primary endpoint across two Phase 3 clinical trials. The FDA approval was based on the GATHER1 and GATHER2 Phase 3 clinical trials, which evaluated the safety and efficacy of monthly 2 mg intravitreal administration of IZERVAY in patients with GA secondary to AMD. The rate of GA growth was evaluated at baseline, 6 months, and 12 months. In each registrational trial, over a 12-month period, the primary analysis showed a statistically significant reduction in the rate of GA growth in patients treated with IZERVAY compared to sham.

European Medicines Agency Accepts Iveric Bio’s Marketing Authorization Application for Avacincaptad Pegol for Geographic Atrophy

Cranbury-based BioNJ Member Iveric Bio/Astellas Pharma announced the European Medicines Agency (EMA) has accepted for regulatory review the marketing authorization application (MAA) for avacincaptad pegol (ACP), an investigational complement C5 inhibitor for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The Committee for Medicinal Products for Human Use (CHMP) of the EMA will start its review of the MAA under the centralized licensing procedure for all 27 member states of the European Union (EU). GA is a form of AMD that can cause irreversible vision loss. Without timely treatment, an estimated 66% of people with GA may become blind or severely visually impaired. Globally, approximately 5 million people are estimated to have GA at least in one eye.

Phathom Pharmaceuticals Announces Submission of Six-Month Stability Data in Support of Erosive GERD New Drug Application

Florham Park-based BioNJ Member Phathom Pharmaceuticals, Inc. announced it has submitted to the FDA six-month stability data from its long-term and accelerated stability program for its reformulated vonoprazan tablets. The additional stability was required for the FDA to complete its NDA review for vonoprazan, a novel first-in-class potassium-competitive acid blocker (PCAB), for the treatment of Erosive GERD (gastroesophageal reflux disease), also referred to as erosive esophagitis. The long-term and accelerated six-month data from Phathom’s stability program have demonstrated that the minor drug product tablet reformulation is controlling NVP growth through six months and keeping levels greater than tenfold below the acceptable daily intake limit of 96 ng/day or 2.4 ppm based on the maximum approved daily dose of 40 mg/day.

Amicus Therapeutics Announces Approval and Launch of New Pompe Disease Therapy in the United Kingdom

BioNJ Member Amicus Therapeutics, with a site in Cranbury, announced that the Medicines and Healthcare products Regulatory Agency (MHRA) of the United Kingdom (U.K.) has granted marketing authorizations for Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) for adults living with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency). The indication for Pombiliti is a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). The indication for Opfolda is an enzyme stabilizer of cipaglucosidase alfa long-term enzyme replacement therapy for adults with LOPD. In addition, the National Institute for Health and Care Excellence (NICE) issued final guidance recommending reimbursement of Pombiliti + Opfolda for use within the National Health Service (NHS) in England and Wales.

Mitsubishi Tanabe Pharma America Announces Post-Hoc Analysis Examining Effect of Early Intervention With RADICAVA (edaravone) on Survival and Disease Progression Milestones in People With ALS

Jersey City-based BioNJ Member Mitsubishi Tanabe Pharma America, Inc., announced the publication of a post-hoc analysis of the pivotal, Phase 3 study (MCI186-19) reviewing the effect of intravenous (IV) RADICAVA® (edaravone) on survival and additional disease progression milestone events in people with amyotrophic lateral sclerosis (ALS). Results from the analysis, which were published in Muscle & Nerve, suggest that timely and continued treatment with RADICAVA when initiated first was associated with a lower risk of death, tracheostomy, permanent assisted ventilation (PAV) or hospitalization, compared to people with ALS who received placebo first followed by RADICAVA. The post-hoc analysis assessed the double-blind, placebo-controlled 24-week Phase 3 MCI186-19 study of RADICAVA, including an additional 24-week open-label period with all study participants receiving RADICAVA first (edaravone-edaravone [EE]) vs. placebo first (placebo-edaravone [PE]).

Cellares Raises $255M Series C to Launch First Integrated Development and Manufacturing Organization (IDMO) and Pioneering Smart Factory to Meet Global Demand for Life-Saving Cell Therapies

BioNJ Member Cellares has secured $255 million in Series C funding led by new investor Koch Disruptive Technologies. In connection with the financing, David Mauney, Managing Director at Koch Disruptive Technologies, will join the company’s board of directors. Global biopharmaceutical company and cell therapy leader Bristol Myers Squibb also participated in the round, alongside DFJ Growth, Willett Advisors and existing investors Eclipse, Decheng Capital, and 8VC. Cellares will use the new funding to launch the world’s first commercial-scale IDMO Smart Factory, which seamlessly integrates advanced robotics, purpose-built technology, and interconnected software. The 118,000 sq. ft. IDMO Smart Factory, located in Bridgewater, New Jersey, will be capable of producing 40,000 cell therapy batches per year. 

Odronextamab Receives EMA Filing Acceptance for the Treatment of Relapsed/Refractory Follicular Lymphoma and Diffuse Large B-Cell Lymphoma

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for odronextamab to treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies. The EMA previously granted odronextamab Orphan Drug Designation for FL and DLBCL. Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing. FL and DLBCL are the two most common subtypes of B-cell non-Hodgkin lymphoma (B-NHL). FL is a slow-growing subtype, with patients often relapsing within five years.

EYLEA HD (AFLIBERCEPT) Injection 8 MG Approved by the FDA for Treatment of Wet Age-related Macular Degeneration (WAMD), Diabetic Macular Edema (DME) and Diabetic retinopathy (DR)

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has approved EYLEA HD (aflibercept) Injection 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). The recommended dose for EYLEA HD is 8 mg (0.07 mL of 114.3 mg/mL solution) every 4 weeks (monthly) for the first 3 months across all indications, followed by 8 mg every 8 to 16 weeks (2 to 4 months) in wAMD and DME and every 8 to 12 weeks (2 to 3 months) for DR. The FDA approval is based on the 48-week results of PULSAR and PHOTON – two double-masked, active-controlled pivotal trials evaluating EYLEA HD compared to EYLEA (aflibercept) Injection 2 mg.

VEOPOZ™ (POZELIMAB-BBFG) Receives FDA Approval as the First Treatment for Children and Adults With Chaple Disease

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced the U.S. Food and Drug Administration (FDA) has approved Veopoz™ (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed. FDA action on the aflibercept 8 mg BLA is expected in the next few weeks. Veopoz, a fully human monoclonal antibody, is designed to target complement factor C5, a protein involved in complement system activation.

Pfizer’s ELREXFIO™ Receives U.S. FDA Accelerated Approval for Relapsed or Refractory Multiple Myeloma

BioNJ Member Pfizer, with a site in Peapack, announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval to ELREXFIO™ (elranatamab-bcmm) for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. Approval was based on the results of the single-arm Phase 2 MagnetisMM-3 trial, and continued approval for this indication is contingent upon verification of clinical benefit in a confirmatory trial(s). ELREXFIO is a subcutaneously delivered B-cell maturation antigen (BCMA)-CD3-directed bispecific antibody (BsAb) immunotherapy that binds to BCMA on myeloma cells and CD3 on T-cells, bringing them together and activating the T-cells to kill myeloma cells.

European Commission Approves Pfizer’s ABRYSVO™ to Help Protect Infants through Maternal Immunization and Older Adults from RSV

BioNJ Member Pfizer, with a site in Peapack, announced that the European Commission (EC) has granted marketing authorization for ABRYSVO™, the company’s bivalent respiratory syncytial virus (RSV) prefusion F (RSVpreF) vaccine, to help protect both infants through maternal immunization and older adults. This marketing authorization follows the recent positive opinion from the Committee for Medicinal Products for Human Use (CHMP). The authorization is valid in all 27 EU member states plus Iceland, Liechtenstein, and Norway. ABRYSVO is the first licensed vaccine designed and studied explicitly for maternal immunization and now a single dose of the vaccine could be administered in the EU between weeks 24 and 36 of gestation. In addition, ABRYSVO has been studied in adults 60 years of age and older. The marketing authorization includes single-dose use in this population as well.

U.S. FDA Approves ABRYSVO™, Pfizer’s Vaccine for the Prevention of Respiratory Syncytial Virus (RSV) in Infants Through Active Immunization of Pregnant Individuals 32-36 Weeks of Gestational Age

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) has approved ABRYSVO™ (Respiratory Syncytial Virus Vaccine), the company’s bivalent RSV prefusion F (RSVpreF) vaccine, for the prevention of LRTD and severe LRTD caused by RSV in infants from birth up to six months of age by active immunization of pregnant individuals at 32 through 36 weeks gestational age. ABRYSVO is unadjuvanted and composed of two preF proteins selected to optimize protection against RSV A and B strains and was observed to be safe and effective. The FDA’s decision is based on the data from the pivotal Phase 3 clinical trial (NCT04424316) MATISSE (MATernal Immunization Study for Safety and Efficacy), a randomized, double-blinded, placebo-controlled Phase 3 study designed to evaluate the efficacy, safety, and immunogenicity of the vaccine against LRTD and severe LRTD due to RSV in infants born to healthy individuals vaccinated during pregnancy. 

Merck Initiates Phase 3 Clinical Program for Oral PCSK9 Inhibitor Candidate MK-0616

Rahway-based BioNJ Member Merck & Co. announced the initiation of the company’s Phase 3 clinical program, CORALreef, for MK-0616, an investigational, oral proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, being evaluated for the treatment of adults with hypercholesterolemia. This is the first Phase 3 clinical program for an oral PCSK9 inhibitor. The first participants are now enrolling in two registrational Phase 3 studies evaluating low-density lipoprotein (LDL) cholesterol reduction: CORALreef Lipids and CORALreef HeFH. Merck also plans to initiate a Phase 3 cardiovascular outcomes study, CORALreef Outcomes by the end of 2023. PCSK9 is a validated target for lowering LDL cholesterol. However, there are no oral PCSK9 inhibitors available to physicians and patients.

LYNPARZA® (olaparib)Plus Abiraterone and Prednisone or Prednisolone Approved in Japan for the Treatment of BRCA-Mutated Metastatic Castration-Resistant Prostate Cancer

Rahway-based BioNJ Member Merck & Co. and AstraZeneca announced that LYNPARZA in combination with abiraterone and prednisone or prednisolone (abi/pred) has been approved in Japan for the treatment of adult patients with BRCA-mutated (BRCAm) castration-resistant prostate cancer with distant metastasis (mCRPC). This approval by the Japanese Ministry of Health, Labor and Welfare was based on an exploratory subgroup analysis of the Phase 3 PROpel trial which showed that LYNPARZA plus abi/pred demonstrated clinically meaningful improvements in both radiographic progression-free survival (rPFS) (HR=0.23 [95% CI, 0.12-0.43]) and overall survival (OS) (HR=0.39 [95% CI, 0.16-0.86]) versus abi/pred alone in patients with BRCAm mCRPC.

Merck Announces WELIREG® (belzutifan) Phase 3 LITESPARK-005 Trial Met Primary Endpoint of Progression-Free Survival in Certain Previously Treated Patients With Advanced Renal Cell Carcinoma

Rahway-based BioNJ Member Merck & Co. announced topline results from LITESPARK-005, the first positive Phase 3 trial investigating WELIREG, Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor. LITESPARK-005 is evaluating WELIREG for the treatment of adult patients with advanced renal cell carcinoma (RCC) that has progressed following PD-1/L1 checkpoint inhibitor and vascular endothelial growth factor-tyrosine kinase inhibitor (VEGF-TKI) therapies. In the trial, WELIREG showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to everolimus, based on a pre-specified interim analysis conducted by an independent Data Monitoring Committee. A statistically significant improvement in the trial’s key secondary endpoint of objective response rate (ORR) was also demonstrated.

Bristol Myers Squibb Receives European Commission Approval for Opdivo (nivolumab) as an Adjuvant Treatment for Patients With Completely Resected Stage IIB or IIC Melanoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Commission (EC) has approved Opdivo (nivolumab) as a monotherapy for the adjuvant treatment of adults and adolescents 12 years of age and older with stage IIB or IIC melanoma who have undergone complete resection, based upon results from the CheckMate -76K trial. This approval builds upon the 2018 EC approval based on results from the CheckMate -238 trial. Opdivo is now indicated for use in the European Union as a monotherapy for the adjuvant treatment of adults and adolescents 12 years of age and older with stage IIB or IIC melanoma, or melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection. 

Updated Data from TRIDENT-1 Trial Show Durable Efficacy Benefits With Repotrectinib for Patients With Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer

Princeton-based BioNJ Member Bristol Myers Squibb announced updated results from the registrational TRIDENT-1 study, demonstrating that repotrectinib, a next-generation ROS1/TRK tyrosine kinase inhibitor (TKI), continued to show high response rates and durable responses in patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC). In this updated analysis, repotrectinib continued to demonstrate durable efficacy in patients with ROS1-positive NSCLC, including intracranial activity, in patients who were TKI-naïve or previously treated with one TKI and no chemotherapy. The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive advanced solid tumors. Bristol Myers Squibb thanks the patients and investigators involved with the TRIDENT-1 clinical trial.

Sandoz Announces Positive Results from Mylight Phase lll Study for Biosimilar Aflibercept

Princeton-based BioNJ Member Sandoz, a Novartis division, announced positive results from the MYLIGHT Phase III confirmatory efficacy and safety study for its biosimilar aflibercept, for patients living with wet macular degeneration – a key development in its efforts to address this area of unmet medical need. Mylight (ClinicalTrials.gov NCT04864834) is part of a comprehensive biosimilar development program that encompasses analytical, preclinical, and a clinical study. The Mylight Phase III confirmatory efficacy and safety study met its primary efficacy endpoint, showing therapeutic equivalence in mean change of best corrected visual acuity (BCVA) from baseline to week 8 between the biosimilar aflibercept and the reference biologic, Eylea®. Safety, immunogenicity, and pharmacokinetics results further confirm that there is no clinically meaningful difference between the products.

U.S. FDA Approves AKEEGA™ (Niraparib and Abiraterone Acetate), the First-And-Only Dual Action Tablet for the Treatment of Patients With BRCA-Positive Metastatic Castration-Resistant Prostate Cancer

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food & Drug Administration (FDA) has approved AKEEGA™ (niraparib and abiraterone acetate), the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC, as detected by an FDA-approved test. “As a physician, identifying patients with a worse prognosis is a priority, especially those whose cancers have a BRCA mutation,” said Kim Chi, M.D., Medical Oncologist at BC Cancer – Vancouver and principal investigator of the Phase 3 MAGNITUDE study. “We prospectively designed the MAGNITUDE study to identify the subset of patients most likely to benefit from targeted treatment with AKEEGA and to help us understand how we can potentially achieve better health outcomes for patients.”

U.S. FDA Approves TALVEY™ (talquetamab-tgvs), a First-in-Class Bispecific Therapy for the Treatment of Patients With Heavily Pretreated Multiple Myeloma

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of TALVEY™ (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. This indication is approved under accelerated approval based on response rate and durability of response. TALVEY™ is a bispecific T-cell engaging antibody that binds to the CD3 receptor on the surface of T cells and G protein-coupled receptor class C group 5 member D (GPRC5D) expressed on the surface of multiple myeloma cells, non-malignant plasma cells and healthy tissue such as epithelial cells in keratinized tissues of the skin and tongue.

European Commission Approves Reduced Dosing Frequency for Janssen’s Bispecific Antibody TECVAYLI®▼ (teclistamab)

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the European Commission (EC) has granted the approval of a Type II variation application for TECVAYLI®▼ (teclistamab), providing the option for a reduced dosing frequency of 1.5mg/kg every two weeks in patients who have achieved a complete response (CR) or better for a minimum of six months. Teclistamab was the first bispecific antibody targeting B-cell maturation antigen (BCMA) on multiple myeloma cells, and CD3 on T-cells, to receive approval in Europe for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

Lilly's Retevmo® (selpercatinib) Demonstrates Superior Progression-Free Survival Compared to Approved Multikinase Inhibitors in RET-Mutant Medullary Thyroid Cancer

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced topline results from the LIBRETTO-531 study evaluating Retevmo versus physician's choice of the multikinase inhibitors (MKIs) cabozantinib or vandetanib as an initial treatment for patients with advanced or metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC). The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS). This result was based on a pre-specified interim efficacy analysis conducted by an independent data monitoring committee (IDMC). Adverse events observed on Retevmo were generally consistent with those identified across the previously reported Retevmo development program (LIBRETTO-001, LIBRETTO-121, LIBRETTO-321).

FDA Approves Veklury® (Remdesivir) to Treat COVID-19 in People With Mild to Severe Hepatic Impairment With No Dose Adjustment

Parsippany-based BioNJ Member Gilead Sciences, Inc. announced that the U.S. Food and Drug Administration (FDA) approved a supplemental new drug application (sNDA) for the use of Veklury® (remdesivir) with no dose adjustments to treat COVID-19 in people with mild, moderate and severe hepatic impairment. This approval further supports the safety profile of Veklury as the first and only approved antiviral COVID-19 treatment that can be used across all stages of liver disease. The clinical benefit of Veklury in hospitalized populations with COVID-19 is supported by randomized controlled trials, real-world evidence and meta-analyses, but the pharmacokinetics of Veklury had not previously been evaluated in patients with hepatic impairment.

Gilead and Tentarix to Discover and Develop Novel Therapies to Address Unmet Medical Needs Across Cancer and Inflammation

Parsippany-based BioNJ Member Gilead Sciences, Inc. and Tentarix Biotherapeutics announced that the companies established three multi-year collaborations leveraging Tentarix’s proprietary Tentacles™ platform to discover and develop multi-functional, conditional protein therapeutics for oncology and inflammatory diseases. Designed to enhance both therapeutic benefit and safety, these molecules have the potential to conditionally target immune cells related to disease pathways without activating other immune cells that may create adverse events. “This early stage collaboration with Tentarix will be highly synergistic to our ongoing efforts, building upon our growing strength in protein therapeutics, and may provide access to next-generation, multi-specific biologics,” said Flavius Martin, M.D., Executive Vice President, Research, Gilead Sciences.

Kyowa Kirin Received Partial Change Approval of LUMICEF® for Palmoplantar Pustulosis in Japan  

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, announced that the company has received approval from the Ministry of Health, Labour and Welfare (“MHLW”) for partial change of approved indication of LUMICEF® [KHK4827, generic name: brodalumab (genetical recombination)] for the treatment of palmoplantar pustulosis*2 (PPP) that respond inadequately to existing therapies. LUMICEF® is a fully human anti-interleukin-17 (IL-17) receptor A antibody that inhibits biological activity of inflammatory cytokines such as IL-17A, IL-17A/F, IL-17F, IL-17C by binding to IL-17A receptor selectively. LUMICEF® is the first IL-17 pathway inhibitor approved in PPP. This approval is based on the results of a Phase 3 clinical trial of brodalumab in patients with PPP who have had an inadequate response to existing therapies in Japan. LUMICEF® met the primary endpoint, and efficacy and safety in patients with PPP were confirmed in this study. 

AbbVie Announces European Commission Approval of AQUIPTA® (atogepant) for the Preventive Treatment of Migraine in Adults

BioNJ Member AbbVie, with a site in Madison, announced that the European Commission has approved AQUIPTA® (atogepant) for the prophylaxis of migraine in adults who have four or more migraine days per month. The approval makes AQUIPTA the first and only once-daily oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) treatment in the European Union for the preventive treatment of both chronic and episodic migraine. Chronic migraine is characterized by 15 or more headache days per month and at least eight migraine days, while episodic migraine refers to people with migraine who have less than 15 headache days per month. People living with migraine may experience frequent disabling attacks that prevent them from performing daily activities and can significantly affect their quality of life. 

FDA Grants Priority Review for Full Approval of TARPEYO for the Treatment of IgA Nephropathy

Hoboken-based Calliditas Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted the submission for the supplemental New Drug Application (sNDA) for TARPEYO® (budesonide) delayed release capsules and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date is 20 December 2023. TARPEYO is currently approved under accelerated approval to reduce proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g. “The combination of the significant eGFR benefit and the reduction in proteinuria lasting for the entire 15-month follow-up period in the full results of our Phase 3 study provide strong rationale for establishing TARPEYO as the standard of care for IgAN patients,” commented Richard Phillipson, Chief Medical Officer at Calliditas.

Calliditas Therapeutics Announces Full Results from the NefIgArd Phase 3 Trial Published in The Lancet

Hoboken-based Calliditas Therapeutics announced publication in The Lancet of the full data from the Phase 3 NefIgArd Study with Nefecon® (TARPEYO® (budesonide) delayed release capsules/Kinpeygo®) in adults with Primary IgA nephropathy (IgAN). The Phase 3 trial met the primary endpoint, estimated glomerular filtration rate (eGFR), with Nefecon demonstrating significant kidney protective effect over placebo. The analysis published in The Lancet shows that Nefecon demonstrated a highly statistically significant and clinically relevant benefit compared to placebo in eGFR over the two-year period of 9-months of treatment with Nefecon and 15-months of follow-up off drug. After the two-year period, there was a 6.11 mL/min/1.73 m2 decline in eGFR in the Nefecon arm compared with a 12.0 mL/min/1.73 m2 decline in the placebo arm, corresponding to a difference in two-year eGFR total slope of 2.95 mL/min/1.73m2 per year (p<0·0001).

EMA Accepts LEO Pharma’s Marketing Authorization Application (MAA) for Delgocitinib Cream in Chronic Hand Eczema (CHE)

LEO Pharma, with a site in Madison, announced that the European Medicines Agency (EMA) has validated the marketing authorization application (MAA) for delgocitinib cream, an investigational product and topical pan-Janus kinase (JAK) inhibitor for the treatment of adult patients with moderate to severe chronic hand eczema (CHE). The acceptance of this MAA marks the beginning of the formal review procedure for this potential new treatment by the EMA’s Committee for Medicinal Products for Human Use (CHMP). “We are delighted that delgocitinib cream has entered this first stage of the European regulatory process. If approved, it will be the first topical treatment specifically indicated and developed for moderate to severe CHE,” says Kreesten Meldgaard Madsen, Chief Development Officer, LEO Pharma.

Daiichi Sankyo Highlights Progress in Creating New Standards of Care for Patients With Lung Cancer With Data Across DXd ADC Portfolio at WCLC

Daiichi Sankyo, with a site in Basking Ridge, will present new clinical research across its DXd antibody drug conjugate (ADC) portfolio in non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC) at the IASLC 2023 World Conference on Lung Cancer (WCLC). Data at WCLC showcasing the company’s progress in creating new standards of care for patients with lung cancer will include primary results of the HERTHENA-Lung01 pivotal phase 2 trial evaluating patritumab deruxtecan (HER3-DXd) in patients with EGFR-mutated NSCLC and late-breaking interim results of the TROPION-Lung04 phase 1b trial evaluating datopotamab deruxtecan (Dato-DXd) in combination with durvalumab with or without carboplatin in patients with metastatic NSCLC without actionable genomic alterations.

Kenvue Completes Separation from J&J

Kenvue, the largest pure-play consumer health company by revenue, is officially on its own following its separation from New Brunswick-based Johnson & Johnson. The maker of Aveeno, BAND-AID Brand Adhesive Bandages, Johnson’s, Listerine, Neutrogena and Tylenol products announced its first day as a fully independent company Aug. 23 following its May spinoff. “This is a historic moment for Kenvue and with a singular focus on delivering innovative care solutions to customers and consumers around the world, we’re excited to write the next chapter of consumer health,” said CEO and director Thibaut Mongon in a statement celebrating the milestone. Temporarily based in Skillman, Kenvue will ultimately establish its headquarters in Summit.

New Director of Cell Therapy and Bone Marrow Transplantation Northern Regions Named to New Jersey’s Only NCI-designated Comprehensive Cancer Center

Rutgers Cancer Institute of New Jersey and RWJBarnabas Health have appointed Adrienne Phillips, M.D., MPH, as Director of Cell Therapy and Bone Marrow Transplantation Northern Regions at New Jersey’s leading cancer program and only National Cancer Institute (NCI)- Designated Comprehensive Cancer Center. Dr. Phillips is also an associate professor of medicine at Rutgers Robert Wood Johnson Medical School. She was most recently an associate professor of Clinical Medicine at Weill Cornell Medical College and associate attending physician at the New York-Presbyterian Hospital. Dr. Phillips joins the Blood and Marrow Transplant and Cellular Therapies Program at the Rutgers Cancer Institute and will lead the cell therapy program in the northern regions which includes Cooperman Barnabas Medical Center, Newark Beth Israel Medical Center, Clara Maass Medical Center, Trinitas Regional Medical Center, RWJUH Rahway and Jersey City Medical Center

10 from New Jersey Make Princeton Review Best Colleges 2024 List

The Princeton Review — one of the nation’s leading education services companies — released its Best 389 Colleges list, with 10 institutions from New Jersey making the grade. The surveys were conducted in 2022–23 and/or the previous two academic years. The 85-question survey asked students to rate their professors, administrators, financial aid, campus amenities, school services and other aspects of life at their colleges on a five-point scale and to report on their experiences at them. Listed alphabetically, the 10 colleges from the Garden State include:

• The College of New Jersey
• Drew University
• Monmouth University
• New Jersey Institute of Technology
• Princeton University
• Rider University
• Rowan University
• Rutgers University
• Seton Hall University
• Stevens Institute of Technology

Cooper Health Unveils Advances in Tech, Programming

Cooper University Health Care announced two significant developments – one regarding innovative technology to improve lung care and the other unveiling a program for patients with sensory disorders. The health care provider said its pulmonary experts were the first anywhere to use Boston Scientific’s iNod Ultrasound Guided Biopsy Needle in conjunction with robotic bronchoscopy. This technology is used to examine suspicious peripheral pulmonary lesions in the lungs, allowing earlier diagnosis and treatment of lung cancer. Following that milestone, Cooper Health announced that it is developing a program to enhance care for patients with intellectual and development disabilities. Disorders such as cerebral palsy, Down syndrome and autism spectrum affect the senses or how the brain processes or interprets information from the senses, according to Cooper Health.

HMH’s HUMC 1st in Nation to Earn Health Care Equity Certification

Hackensack Meridian Health announced that Hackensack University Medical Center has achieved what no other hospital in the nation has to date — it is the first hospital in the country to earn a Health Care Equity Certification from the Joint Commission. The certification indicates Hackensack Meridian Health’s extensive efforts to address and improve health care equity across its network. “Our team members at Hackensack Meridian Health are dedicated to providing safe, equitable care to all of our communities. Achieving health equity is one of our network’s top priorities,” Robert Garrett, CEO, Hackensack Meridian Health, said. “We are honored to have Hackensack University Medical Center be recognized as the first and only site in the nation to complete this important certification from the Joint Commission, and we look forward to continuing to advance health equity for our patients.”

Hackensack Meridian CEO to Chair Global Health Organization

Hackensack Meridian Health CEO Robert Garrett has been named Chair of the World Economic Forum’s Health and Healthcare Governor’s Community, the health system announced August 15. During his two-year term, Mr. Garrett’s mission will be to help define the industry agenda and drive change to improve access to health care and outcomes and to drive more efficiency in the industry. Dr. Shyam Bishen, Head of the Centre for Health & Healthcare at the World Economic Forum, said the group was “delighted” to have Mr. Garrett as Chair, adding, “We believe with him at the helm, during this period, the community will make significant strides in solving the pressing challenges of the health care industry and drive much needed impact especially in areas of health outcomes and access.” 

R&D Council of NJ to Honor Top Inventors at 44th Annual Edison Patent Awards

The Research & Development Council of New Jersey announced the honorees and winners of the 2023 Edison Patent Awards, the State’s highest recognition for inventors and innovation. The R&D Council will honor retired Bell Laboratories inventor and communications trailblazer Victor Lawrence, Ph.D. with its highest honor: the Science and Technology Medal; Robert C. Garrett, CEO of Hackensack Meridian Health will receive the Chairman’s Award; New Jersey Senator Andrew Zwicker, Ph.D. of Princeton Plasma Physics Laboratory will be honored with the Educator of the Year Award. BioNJ Members honored as 2023 Edison Patent Award winners include Bristol Myers Squibb, Merck Research Laboratories, New Jersey Institute of Technology, Princeton University, Rowan University, and Rutgers, The State University of New Jersey.

The TiE Women Global Pitch Competition

Hosted by TiE-NJ

November 2023

Calling all women entrepreneurs! Apply now for TIE Women Global Pitch Competition 2023.

The TiE Women Global Pitch Competition is a prestigious event that provides a platform for women entrepreneurs to pitch their innovative ideas and gain recognition and support. This is a great opportunity for women entrepreneurs in New Jersey to gain visibility and access to a Global network, in addition to mentorship and support from the local chapter. More about TiE Women here: www.tiewomen.org