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ICYMI: Government Watchdog Report Finds Patients in Medicare Part D Paid Billions More for Medicines than Insurers and PBMs

Medicare Part D beneficiaries paid four times more than insurance companies and their pharmacy benefit managers (PBMs) for 79 of the top 100 highly rebated medicines, according to a report by the nonpartisan Government Accountability Office (GAO). This shocking revelation sheds new light on how PBMs’ and insurers’ failure to share rebate savings directly with patients can lead to higher out-of-pocket costs at the pharmacy counter and why strong PBM reforms are needed.

A Brief Look at Current Debates About Pharmacy Benefit Managers

Pharmacy benefit managers (PBMs) are entities that administer prescription drug insurance benefits. Their key functions include negotiating prices with drug manufacturers and pharmacies, establishing drug formularies and pharmacy networks, and processing drug claims. PBMs are currently attracting considerable critical attention from policymakers. Multiple congressional committees have recently reported out legislation related to PBMs, and there will likely be efforts to reconcile these bills this fall.

Incomplete Evaluations Undermine Patient Access to Transformational Medicine

The Institute for Clinical and Economic Review (ICER) bills itself as the arbiter of “clinical and economic value” in health care. The problem: Its assessments continue to lack transparency and ignore critical factors that contribute to the value of a medicine. The result is misleading evaluations that threaten access to, and the development of, innovative therapies. Thanks to the many cell and gene therapies currently available — with many more still in development — patients can experience long-lasting or even curative effects from just one administration. These therapies can bring tremendous value to patients, their families and society. Unfortunately, patients also could face restricted access to these treatments if payers rely on value assessment methodologies that undervalue these life-changing therapies.

4 Truths About State Government Price Setting

Groups motivated by special interests and backed by insurance companies are pushing states to adopt flawed price-setting schemes, often in the form of prescription drug affordability boards (PDABs). Establishing these boards wedges government-appointed bureaucrats between doctors and their patients while shortsightedly focusing on only one component of health care — all too curiously avoiding any review of or actions on abusive health insurance practices. This is not good news for patients.

PODCAST: Cancer Drug Developer Dr. Steve Potts' Congressional Testimony on the IRA

In this Vital Health Podcast, we’re speaking with Steve Potts before his congressional testimony for the U.S. House Committee on Energy and Commerce. Steve presented evidence regarding the negative impacts of the Inflation Reduction Act on U.S. venture capital investors, the development of small molecules and the needed therapies that will most likely be lost to patients over the next ten years.

PODCAST: Dan Leonard, Executive Director, We Work For Health

Dan is the newly appointed Executive Director of We Work For Health, bringing more than two decades of experience in advocacy, policy, and public affairs to this role. He previously headed the Association for Accessible Medicines (AAM) and the National Pharmaceutical Council. In this podcast, we discuss the ten drugs selected for negotiation under the IRA and their unintended consequences on the availability and access of generic and biosimilar medicines. We also highlight the desire for many regulators to broaden government negotiations within Medicare under the Smart Pricing Act and the recently introduced bill by Congressman Frank Pallone, Jr. (NJ-06). 

2023 ROI Influencers: Health Care: Debbie Hart and Pavita Howe

Some say that health care represents one-sixth of our economy. That may be a low estimate. From young to old, hospital stays to outpatient services, life sciences to pharma — to the care of our body, mind and soul — New Jerseyans seemingly interact with the health care industry on a weekly, if not daily, basis. That’s why the annual ROI Influencers: Health Care list is so important and impactful. We’re offering an in-depth look at who is helping to keep you healthy and happy in all facets of life. Congratulations to Debbie Hart, President & CEO of BioNJ, and Pavita Howe, Vice President of Innovation and Entrepreneurship, for their well-deserved recognition on the 2023 ROI-NJ Influencers: Health Care List. Click here for the full list including numerous BioNJ Members.

Amicus Therapeutics Announces FDA Approval and Launch of New Treatment for Pompe Disease

Princeton-based BioNJ Member Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved Pombiliti™ (cipaglucosidase alfa-atga) + Opfolda™ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). Pombiliti + Opfolda is a unique two-component therapy. Pombiliti is a recombinant human GAA enzyme (rhGAA) naturally expressed with high levels of bis-M6P (Mannose 6-Phosphate), designed for increased uptake into muscle cells. Once in the cell, Pombiliti can be properly processed into its most active and mature form to break down glycogen. Opfolda is an enzyme stabilizer designed to stabilize the enzyme in the blood.

Phathom Pharmaceuticals Announces Vonoprazan NDA Submission for Non-Erosive GERD

Florham Park-based BioNJ Member Phathom Pharmaceuticals, Inc. announced the submission of an NDA to the FDA for vonoprazan as a daily treatment for Non-Erosive gastroesophageal reflux disease (GERD) in adults. The regulatory submission is supported by the positive data from the PHALCON-NERD-301 study, a Phase 3 study evaluating the efficacy and safety of vonoprazan for the daily treatment of adults with Non-Erosive GERD (NERD). Vonoprazan is an investigational first-in-class potassium-competitive acid blocker (PCAB) from a novel class of medicines that block acid secretion in the stomach. The effectiveness and safety of vonoprazan 10 mg and 20 mg given once daily in NERD was evaluated in a randomized, placebo-controlled, double-blind, 4-week trial with a 20-week blinded extension conducted in the United States in 772 adult patients. 

Soligenix Announces Achievement of Two-Year Stability With Bivalent and Trivalent Thermostabilized Filovirus Vaccines when Stored at High Temperatures

Princeton-based BioNJ Member Soligenix, Inc. announced two-year stability of its thermostabilized bivalent and trivalent filovirus vaccine candidates at temperatures of 40 degrees Celsius (104 degrees Fahrenheit) when formulated in a single vial, needing reconstitution only with sterile water immediately prior to use. This follows the previous successful demonstration of 100% protection of non-human primates (NHPs) against lethal Sudan ebolavirus and Marburg marburgvirus challenge with the bivalent vaccine. This important milestone is part of an ongoing collaboration with the University of Hawaiʻi at Mānoa (UHM), demonstrating the successful presentation of one or more antigen(s) within the same formulation while maintaining full potency and thermostability, including vaccines for Sudan ebolavirus (SuVax™) and Marburg marburgvirus (MarVax™). 

EPKINLY (epcoritamab) Approved by Japan Ministry of Health, Labour and Welfare as the First Bispecific Antibody to Treat Adults Patients With Certain Types of Relapsed or Refractory (R/R) Large B-Cell Lymphoma (LBCL)

Princeton-based BioNJ Member Genmab announced that the Japan Ministry of Health, Labour and Welfare has approved EPKINLY™ (epcoritamab) as the first and only T-cell engaging bispecific antibody treatment in Japan of adult patients with certain types of relapsed or refractory large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), after two or more lines of systemic therapy. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies' oncology collaboration. The approval of EPKINLY in Japan is based on the results from two open-label, multi-center studies designed to evaluate the safety and preliminary efficacy of EPKINLY monotherapy in patients with R/R LBCL. 

Genmab Announces European Commission Approval of TEPKINLY® (epcoritamab) for Adults With Relapsed or Refractory (R/R) Diffuse Large B-cell Lymphoma (DLBCL)

Princeton-based BioNJ Member Genmab announced that the European Commission (EC) has granted conditional marketing authorization for TEPKINLY® (epcoritamab) as a monotherapy for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. TEPKINLY is the first and only subcutaneous T-cell engaging bispecific antibody approved for the treatment of this patient population in the European Union (EU), as well as Liechtenstein, Norway, and Iceland. DLBCL is the most common type of B-cell non-Hodgkin’s lymphoma worldwide. While patients may have access to chemoimmunotherapy regimens to treat their disease, they face limited treatment options, with few readily available, off-the-shelf medicines, especially for those whose disease has relapsed or become refractory to prior treatments.

Nature Publishes Discovery and Preclinical Results for ABBV-CLS-484, a Potential First-in-Class PTPN2/N1 Inhibitor in Cancer Immunotherapy

BioNJ Member AbbVie, with a site in Branchburg, the Broad Institute of MIT and Harvard, and Calico Life Sciences announced the publication in Nature of the discovery and preclinical data that demonstrate investigational ABBV-CLS-484 is a potential first-in-class, orally bioavailable, PTPN2/N1 phosphatase inhibitor that enhances anti-tumor immunity. The findings, based on research conducted by AbbVie in collaboration with the Broad Institute and Calico, support the development of ABBV-CLS-484 as a promising new strategy for cancer immunotherapy. Titled "The PTPN2/PTPN1 inhibitor ABBV-CLS-484 unleashes potent anti-tumour immunity," the paper highlights the novel structural insights and design that led to the discovery of ABBV-CLS-484 and its dual mechanism of action that targets tumor cells and suppresses their growth, as well as promotes the activation of several immune cell types to increase their anti-tumor activity.

AbbVie Exercises Exclusive Right to Acquire Mitokinin, Further Strengthening Neuroscience Pipeline

BioNJ Member AbbVie, with a site in Branchburg, announced that it has exercised its exclusive right and completed the acquisition of Mitokinin, a discovery-stage biotechnology company developing a potentially first-in-class disease-modifying treatment for Parkinson's Disease (PD). Mitokinin's lead compound, a selective PINK1 activator, is designed to address mitochondrial dysfunction that is believed to be a major contributing factor to Parkinson's disease pathogenesis and progression. PINK1 plays a key role in maintaining a healthy mitochondrial system by facilitating the turnover of dysfunctional mitochondria. Mutations in PINK1 are associated with a loss of PINK1 function and cause familial forms of PD. Activation of PINK1 offers a potentially disease-modifying treatment approach beyond familial PD, by addressing mitochondrial dysfunction and improving mitochondrial health in sporadic PD.

Chugai Obtains Regulatory Approval for Phesgo, the Fixed-Dose Subcutaneous Combination of Perjeta and Herceptin for HER2-Positive Breast and Colorectal Cancer

BioNJ Member Chugai Pharmaceutical Co., with a site in Berkeley Heights, announced that it has obtained regulatory approval today from the Ministry of Health, Labour and Welfare (MHLW) for Phesgo® combination for Subcutaneous Injection MA, IN [generic name: pertuzumab (genetical recombination), trastuzumab (genetical recombination) and vorhyaluronidase alfa (genetical recombination) ] (hereafter, Phesgo), antineoplastic agent / anti-HER2 humanized monoclonal antibody for the treatment of “HER2-positive breast cancer” and “Advanced or recurrent HER2-positive colorectal cancer that has progressed following cancer chemotherapy and is not amenable to curative resection.” This subcutaneous fixed-dose combination without preparation contains the same monoclonal antibodies as Perjeta and Herceptin, and also a vorhyaluronidase alfa (genetical recombination) combined in a single vial.

Chugai Obtains Approval for Additional Indication of Actemra for Cytokine Release Syndrome Induced by Cancer Therapy in Japan

BioNJ Member Chugai Pharmaceutical Co., with a site in Berkeley Heights, announced that it obtained regulatory approval from the Ministry of Health, Labour and Welfare for the humanized anti-human IL-6 receptor monoclonal antibody, “Actemra® Intravenous Infusion 80 mg, 200 mg, and 400 mg” [generic name: tocilizumab (genetical recombination)] for an additional indication of treatment of cytokine release syndrome induced by cancer therapy. Actemra was approved for the indication of cytokine release syndrome induced by tumor-specific T-cell infusion therapy in March 2019. This additional indication will make it possible to administer the drug as an anti-cytokine therapy for cytokine release syndrome (CRS) in the cancer treatment other than tumor-specific T cell infusion therapy.

Chugai’s Enspryng Launched in Taiwan as First Indication for Neuromyelitis Optica Spectrum Disorder (NMOSD)

BioNJ Member Chugai Pharmaceutical Co., with a site in Berkeley Heights, announced that Chugai Pharma Taiwan Ltd., a wholly owned subsidiary of Chugai, has launched Enspryng®, a drug created by Chugai for “the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult and adolescent over 12 years old patients who are anti-aquaporin-4 (AQP4) antibody positive” as the first indication in Taiwan. Enspryng was designated as an orphan drug by the Ministry of Health and Welfare (MoHW) on September 14, 2022, and approved orphan drug license by Taiwan Food and Drug Administration (TFDA) on July 28, 2023. Enspryng, designed by Chugai, is a pH-dependent binding humanized anti-IL-6 receptor antibody, which was the first product developed by applying our proprietary recycling antibody® technology. 

Cellares, Bristol Myers Squibb Expand Manufacturing Agreement

Bridgewater-based BioNJ Member Cellares will provide proof-of-concept manufacturing for a second Bristol Myers Squibb CAR-T cell therapy as part of an expanded agreement between the companies. The announcement comes weeks after Cellares first indicated that Princeton-based BioNJ Member Bristol Myers Squibb had entered the Cellares Technology Adoption Partnership (TAP) program — a fast and low-risk opportunity for cell therapy developers to adopt the company’s automated manufacturing technology for products in their pipeline. Under the expanded terms, Cellares will optimize, automate and tech-transfer the additional CAR-T cell therapy process onto its automated manufacturing platform, the Cell Shuttle. Bristol Myers Squibb is using the program to evaluate the Cell Shuttle as an automated manufacturing process and produce comparability data to confirm whether it is a viable, cost-efficient, and scalable manufacturing solution for cell therapies.

Sotyktu (deucravacitinib) Long-Term Data Demonstrate Durable Efficacy and Consistent Safety for Up to Three Years in Moderate-to-Severe Plaque Psoriasis

Princeton-based BioNJ Member Bristol Myers Squibb announced new three-year results from the POETYK PSO long-term extension (LTE) trial of Sotyktu (deucravacitinib) treatment in adult patients with moderate-to-severe plaque psoriasis. At Week 148, clinical response rates were maintained with continuous treatment with modified nonresponder imputation (mNRI) responses of 73.2% for Psoriasis Area and Severity Index (PASI) 75, 48.1% for PASI 90 and 54.1% for static Physician’s Global Assessment (sPGA) 0/1. Sotyktu demonstrated a consistent safety profile with no increases in the rates of adverse events (AEs) or serious AEs over time, and no emergence of any new safety signals. The safety analysis assessed 1,519 patients who received at least one dose of Sotyktu across POETYK PSO-1, POETYK PSO-2 and POETYK PSO-LTE. 

Bristol Myers Squibb Presents New Zeposia (ozanimod) Data on Long-Term Disease Progression and Cognition in Patients With Relapsing Forms of Multiple Sclerosis

Princeton-based BioNJ Member Bristol Myers Squibb announced new data showing that after eight years of follow-up, 76% of patients treated with Zeposia for relapsing multiple sclerosis (RMS) were free of six-month confirmed disability progression (CDP). Findings also demonstrated treatment with Zeposia resulted in low rates of progression independent relapse activity (PIRA) and relapse-associated worsening (RAW), key drivers of disease progression and permanent disability in multiple sclerosis. In all participants, PIRA and RAW were observed in 13.2% and 10.7% of participants treated with continuous Zeposia, respectively, after eight years in the open-label extension study. Higher baseline expanded disability status scale (EDSS) scores to assess CDP and lower baseline whole brain, cortical grey matter and thalamic volumes were predictive of RAW but not PIRA. 

Bristol Myers Squibb Announces Perioperative Regimen of Neoadjuvant Opdivo (nivolumab) and Chemotherapy Followed by Adjuvant Opdivo Significantly Improves Event-Free Survival in Patients With Resectable Non-Small Cell Lung Cancer

Princeton-based BioNJ Member Bristol Myers Squibb announced the Phase 3 CheckMate -77T trial met its primary endpoint of improved event-free survival (EFS) as assessed by Blinded Independent Central Review (BICR) in patients with resectable stage IIA to IIIB non-small cell lung cancer (NSCLC). In a prespecified interim analysis, the perioperative regimen of neoadjuvant Opdivo (nivolumab) with chemotherapy followed by surgery and adjuvant Opdivo showed a statistically significant and clinically meaningful improvement in EFS compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo. The safety profile of this Opdivo-based regimen was consistent with previously reported studies in NSCLC.

Bristol Myers Squibb Strengthens and Diversifies Oncology Portfolio With Acquisition of Mirati Therapeutics

Princeton-based BioNJ Member Bristol Myers Squibb and Mirati Therapeutics, Inc.® announced that they have entered into a definitive merger agreement under which Bristol Myers Squibb has agreed to acquire Mirati for $58.00 per share in cash, for a total equity value of $4.8 billion. Mirati’s assets are a strong fit with Bristol Myers Squibb’s portfolio and innovative pipeline and represent an attractive opportunity to grow Bristol Myers Squibb’s oncology franchise. Through this acquisition, Bristol Myers Squibb will add KRAZATI, an important lung cancer medicine, to its commercial portfolio. The company gains access to several promising clinical assets that complement its oncology pipeline and are strong candidates for single agent development and combination strategies.

Novo Nordisk and Valo Health to Collaborate to Discover and Develop Novel Treatments for Cardiometabolic Diseases using Human Data and Artificial Intelligence

Plainsboro-based BioNJ Member Novo Nordisk and Valo Health, Inc. announced they have entered into an agreement to discover and develop novel treatments for cardiometabolic diseases based on Valo’s large human dataset and computation powered by artificial intelligence (AI). The collaboration between the two organizations will leverage the capabilities of Valo’s Opal Computational Platform™ including access to real-world patient data, AI-enabled small molecule discovery and Biowire® human tissue modelling platform designed to speed up the discovery and development process. Valo’s preclinical capabilities enable identification and validation of novel druggable targets, as well as development of drug candidates against these targets, and can help predict compound safety and efficacy.

Pfizer Broadens Portfolio of Respiratory Vaccines Recommended by CDC Advisory Committee With ABRYSVO™ for RSV

BioNJ Member Pfizer, with a site in Peapack, announced it has broadened its portfolio of respiratory vaccines recommended by the U.S. Centers for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) following a favorable vote for ABRYSVO™ [Respiratory Syncytial Virus Vaccine], the company’s bivalent RSV prefusion F (RSVpreF) vaccine, as a maternal immunization. This is the first-ever fall in which eligible individuals can receive Pfizer vaccines to help protect against RSV, COVID-19 and pneumococcal pneumonia. The ACIP recommendation follows the U.S. Food and Drug Administration’s (FDA) approval of ABRYSVO in August as the first and only maternal vaccine for the prevention of lower respiratory tract disease (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.

U.S. FDA Approves Pfizer’s BRAFTOVI® + MEKTOVI® for BRAF V600E-Mutant Metastatic Non-Small Cell Lung Cancer

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) has approved BRAFTOVI® (encorafenib) + MEKTOVI® (binimetinib) for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, as detected by an FDA-approved test. BRAF V600E mutations can be assessed from either plasma or tumor tissue using the FoundationOne Liquid CDx or the FoundationOne CDx FDA-approved companion diagnostic tests, respectively. The FDA’s approval is based on data from the ongoing Phase 2 PHAROS clinical trial (NCT03915951), an open-label, multicenter, single‑arm study examining BRAFTOVI + MEKTOVI combination therapy in both treatment-naïve and previously treated patients with BRAF V600E-mutant metastatic NSCLC.

Once-Weekly ALTUVIIIO® Approved in Japan as a New Class of Factor VIII Therapy for Hemophilia A

Bridgewater-based BioNJ Member Sanofi announced the Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO® [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for control of bleeding tendency in patients with hemophilia A (factor VIII deficiency). ALTUVIIIO was also recently approved by the Taiwan Food and Drug Administration for treatment of adults and children with hemophilia A on August 31, 2023. Also referred to as efanesoctocog alfa, 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 in adults and adolescents, and significantly reduces bleeds compared to prior factor VIII prophylaxis in adults and adolescents with severe hemophilia A. 

Sanofi Announces Agreement for Potential First-in-Class Vaccine Against Extraintestinal Pathogenic E. Coli

Bridgewater-based BioNJ Member Sanofi announced that it has entered into an agreement with BioNJ Member Janssen Pharmaceuticals, a Johnson & Johnson company, to develop and commercialize the vaccine candidate for extraintestinal pathogenic E. coli (9-valent) developed by Janssen, currently in Phase 3. The agreement brings together Janssen’s robust science behind this potential first-in-class product and Sanofi’s worldwide manufacturing footprint and recognized world-class expertise in launching innovative vaccines. Under the terms of the agreement, both parties will co-fund current and future research and development costs. Sanofi will pay USD 175M upfront to Janssen, followed by development and commercial milestones. There will be a profit-share arrangement in the U.S., EU4 (France, Germany, Italy, Spain) and the UK. In the rest of the world (ROW), Janssen will receive tiered royalties and sales milestones. Closing is subject to customary regulatory clearance.

Sanofi and Teva Announce Exclusive Collaboration to Deliver Inflammatory Bowel Disease Treatment

Bridgewater-based BioNJ Member Sanofi and Parsippany-based BioNJ Member Teva Pharmaceutical announced a collaboration to co-develop and co-commercialize asset TEV’574, currently in Phase 2b clinical trials for the treatment of Ulcerative Colitis and Crohn's Disease, two types of inflammatory bowel disease. Paul Hudson, Chief Executive Officer, Sanofi said “Anti-TL1As are a promising class of therapies, and we believe that TEV’574 could emerge as a best-in-class option for people living with serious gastrointestinal diseases. This collaboration strengthens our commitment to advancing innovative treatment options for inflammatory conditions with a high unmet need and bolsters our goal to be an industry leader in immunology.”

New Data at WMS 2023 Reaffirm Long-Term Efficacy of Nexviazyme® (avalglucosidase alfa) for the Treatment of Pompe Disease

New data shared by Bridgewater-based BioNJ Member Sanofi build upon the considerable body of evidence supporting the use of Nexviazyme® (avalglucosidase alfa) to treat a wide range of patients living with Pompe disease across various clinical circumstances. Nexviazyme is a monotherapy approved in the United States and other markets for the treatment of late-onset Pompe disease and is approved for infantile-onset Pompe disease in Europe and other countries. In the U.S., Nexviazyme is currently being evaluated in Phase 3 clinical trials for infantile-onset Pompe disease. Data include research across people living with late-onset or infantile-onset Pompe disease, people who are treatment naïve (never before treated) or have switched from previous treatment with the long-time standard of care, alglucosidase alfa, which is marketed under the brand name Myozyme® or in the U.S. as Lumizyme®, and patients with varying baseline characteristics.

DUPIXENT® (DUPILUMAB) SBLA for Treatment of Eosinophilic Esophagitis (EOE) in Children Aged 1 to 11 Accepted for FDA Priority Review

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, and BioNJ Member Sanofi, with a site in Bridgewater, announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) to treat children aged 1 to 11 years with eosinophilic esophagitis (EoE). The target action date for the FDA decision is January 31, 2024. Dupixent is the first and only treatment in the U.S. approved for children and adults aged 12 years and older with EoE, weighing at least 40kg. Priority review is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. 

Odronextamab BLA for Treatment of Relapsed/Refractory Follicular Lymphoma (FL) and Diffuse Large B-Cell Lymphoma (DLBCL) Accepted for FDA Priority Review

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) 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 target action date for the FDA decision is March 31, 2024. 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. The BLA was supported by data from a Phase 1 and pivotal Phase 2 trial (ELM-1 and ELM-2). 

EYLEA® HD (aflibercept) Injection 8MG Two-Year Results from Pivotal Pulsar Trial in Wet Age-Related Macular Degeneration Presented at Euretina

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced the first presentation of positive two-year (96 weeks) results from the pivotal PULSAR trial investigating EYLEA® HD (aflibercept) Injection 8 mg with 12- and 16-week dosing regimens, compared to EYLEA® (aflibercept) Injection, in patients with wet age-related macular degeneration (wAMD). PULSAR (N=1,009) is a double-masked, active-controlled pivotal trial evaluating non-inferiority of EYLEA HD 12-week (n=335) and 16-week (n=338) dosing regimens compared to an 8-week dosing regimen for EYLEA (n=336). All patients received three initial monthly doses. Patients receiving EYLEA HD could have their dosing intervals shortened to an every 8-week interval if protocol-defined criteria for disease progression were observed. 

FDA Approves Novartis Cosentyx® as First Intravenous (IV) Formulation Interleukin-17A Antagonist for Rheumatic Diseases

East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) has approved an intravenous (IV) formulation of Cosentyx® (secukinumab) for the treatment of adults with psoriatic arthritis (PsA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA). Cosentyx is the only treatment approved in an IV formulation that specifically targets and blocks interleukin-17A (IL-17A), and the only non-tumor necrosis factor alpha (TNF-α) IV option available in all these indications. The IV formulation of Cosentyx offers patients a monthly 30-minute, weight-based dosing option, requiring no pre-medication and no lab monitoring. The new IV administration option will be available in Q4 of 2023.

Sandoz Receives European Commission Approval for Tyruko® (natalizumab), First and Only Biosimilar for Multiple Sclerosis in Europe

Princeton-based BioNJ Member Sandoz announced that the European Commission (EC) granted marketing authorization for the first and only biosimilar Tyruko® (natalizumab), developed by Polpharma Biologics. The authorization covers treatment as a single disease-modifying therapy (DMT) in adults with highly active RRMS, the same indication as approved by the EC for the reference medicine Tysabri® (natalizumab). Rebecca Guntern, President Europe, Sandoz, said: “Multiple sclerosis is a chronic condition with no cure at present and timely access to affordable, high-quality healthcare is therefore even more essential. Today’s approval brings us one step closer to reducing the burden of this disease for those living with multiple sclerosis in Europe by making the life-enhancing treatments they need more accessible.”

Sandoz Receives Positive CHMP Opinion for Breast and Gastric Cancer Biosimilar Trastuzumab

Princeton-based BioNJ Member Sandoz announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), has adopted a positive opinion recommending marketing authorization for their biosimilar trastuzumab (150 mg, for intravenous use), developed by EirGenix, Inc. The positive opinion for Sandoz trastuzumab, a monoclonal antibody, covers treatment of human epidermal growth factor receptor 2 positive (HER2-positive) breast cancer and metastatic gastric cancers, the same indications as approved by EMA for the reference biologic. Sandoz and EirGenix signed a license agreement in April 2019. Under this agreement, EirGenix will remain responsible for the development and manufacturing of trastuzumab, while Sandoz will hold the rights to commercialize the medicine upon approval in respective markets.

Sandoz Enters New Era as Standalone Global Leader and European Champion in Generic and Biosimilar Medicines

Princeton-based BioNJ Member Sandoz celebrated the start of share trading on the SIX Swiss Exchange as an independent company. The new company is included in key SIX market indices and has an investment-grade credit rating that gives it a strong competitive position. It is well placed for continued profitable growth as a standalone global leader and European champion in generic and biosimilar medicines. Sandoz Board Chairman Gilbert Ghostine says: “Today marks the dawn of a new era for Sandoz as an independent company, but our Purpose is unchanged: pioneering access for patients. It’s what we do best, have always done best, and will always do best: making quality medicines available to more people, in more places, in more and novel ways.”

Kyowa Kirin to Acquire Orchard Therapeutics

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, and Orchard Therapeutics plc announced the companies have entered into a definitive agreement under which Kyowa Kirin will acquire Orchard Therapeutics for $16.00 per American Depositary Share (ADS) in cash (approximately $387.4 million, or ¥57.3 billion), under which Orchard shareholders will hold an additional contingent value right (CVR) of $1.00 per ADS. An additional $1.00 CVR will be paid for a total of $17.00 per ADS, or approximately $477.6 million (¥70.7 billion) if the conditions are met. Orchard Therapeutics’ portfolio comprises Libmeldy® (atidarsagene autotemcel), also known as OTL-200, intended for eligible patients with early onset metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system.

Evotec SE: Launch of Partnering Agreement to Strengthen Biotech Innovators in Shared R&D Economy

Princeton-based BioNJ Member Evotec SE announced an agreement to boost early stage innovations through access to Evotec's leading shared end-to-end R&D platform. The agreement will empower LabCentral / BioLabs / MBC BioLabs's network of promising early stage life sciences and biotech companies by enhanced access to Evotec's industry-leading, innovative end-to-end platform and latest technologies for successful drug discovery and development to translate innovative life science research into new, improved ways to treat patients. Through this partnership, Evotec's team of scientists, business builders and experts will have regular presence on-site across the LabCentral / BioLabs / MBC BioLabs network in the form of hosted educational events and office hours – parts of the efforts towards the joint goal of enhancing success rates of the early stage companies in the ecosystem. 

Merck’s KEYTRUDA® (pembrolizumab) Met Primary Endpoint of Disease-Free Survival (DFS) in Certain Patients With Muscle-Invasive Urothelial Carcinoma (MIUC) After Surgery

Rahway-based BioNJ Member Merck & Co. announced that the Phase 3 AMBASSADOR (A031501) trial (KEYNOTE-123) evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, met one of its dual primary endpoints of disease-free survival (DFS) for the adjuvant treatment of patients with localized muscle-invasive urothelial carcinoma (MIUC) and locally advanced urothelial carcinoma versus observation. At a pre-specified interim analysis review conducted by an independent Data Monitoring Committee, KEYTRUDA demonstrated a statistically significant and clinically meaningful improvement in DFS versus observation in these patients after surgery. The trial will continue to evaluate its other dual primary endpoint of overall survival (OS). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified. 

Merck Receives Priority Review from FDA for New Biologics License Application for Sotatercept, an Activin Signaling Inhibitor to Treat Adults With Pulmonary Arterial Hypertension (PAH)

Rahway-based BioNJ Member Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review a new Biologics License Application (BLA) for sotatercept, Merck’s novel investigational activin signaling inhibitor, for the treatment of adult patients with pulmonary arterial hypertension (PAH) (WHO Group 1). The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of March 26, 2024. The application for sotatercept is based on data from the Phase 3 STELLAR trial, in which sotatercept on top of background therapy demonstrated a statistically significant and clinically meaningful improvement in 6-minute walk distance (6MWD) and eight of nine secondary outcome measures. 

Merck Announces Pivotal KEYNOTE-671 Trial Meets Dual Primary Endpoint of Overall Survival (OS) in Resectable Stage II, IIIA or IIIB Non-Small Cell Lung Cancer (NSCLC)

Rahway-based BioNJ Member Merck & Co. announced the Phase 3 KEYNOTE-671 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, as a perioperative treatment regimen for patients with resectable stage II, IIIA or IIIB (T3-4N2) non-small cell lung cancer (NSCLC) met its dual primary endpoint of overall survival (OS). At a pre-specified interim analysis, KEYTRUDA plus chemotherapy before surgery (neoadjuvant), followed by resection and KEYTRUDA as a single agent after surgery (adjuvant), demonstrated a statistically significant and clinically meaningful improvement in OS compared to neoadjuvant placebo plus chemotherapy followed by adjuvant placebo in these patients. The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were identified. 

Landmark Phase 3 MARIPOSA Study Meets Primary Endpoint Resulting in Statistically Significant and Clinically Meaningful Improvement in Progression-Free Survival for RYBREVANT® (amivantamab-vmjw) Plus Lazertinib Versus Osimertinib in Patients With EGFR-Mutated Non-Small Cell Lung Cancer

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced positive topline results from the Phase 3 MARIPOSA study evaluating RYBREVANT® (amivantamab-vmjw), a bispecific antibody targeting epidermal growth factor receptor (EGFR) and mesenchymal-epithelial transition (MET), in combination with lazertinib, an oral third-generation EGFR tyrosine kinase inhibitor (TKI), versus osimertinib as first-line treatment in patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC). The pivotal Phase 3 MARIPOSA study met its primary endpoint with a statistically significant and clinically meaningful improvement in progression-free survival (PFS) in patients receiving RYBREVANT® plus lazertinib compared to osimertinib. The combination of RYBREVANT® and lazertinib demonstrated a safety profile consistent with previously reported data on the combination. 

Johnson & Johnson Launches Health Equity Innovation Challenge

In a bid to address health disparities across the U.S., BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson is seeking changemakers to develop solutions to help close gaps in racial health and mortality in some of the country’s biggest cities. In announcing the launch of its second Health Equity Innovation Challenge, the New Brunswick-based health conglomerate said this year’s initiative will focus on five cities – Atlanta, Chicago, Los Angeles, New York and Philadelphia – which were selected due to their demonstrated need for health care education, access and care. Created in 2021, the Challenge aims to foster innovation, entrepreneurship and socioeconomic impact by supporting innovative solutions from local entrepreneurs, startups, innovators and community-based organizations that have the potential to advance health equity.

Janssen Submits Application to the European Medicines Agency for RYBREVANT®▼ (amivantamab) in Combination With Chemotherapy for the First-Line Treatment of Adult Patients with Advanced Non-Small Cell Lung Cancer With Activating EGFR Exon 20 Insertion Mutations

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Type II extension of indication application to the European Medicines Agency (EMA) seeking approval of RYBREVANT®▼ (amivantamab) in combination with chemotherapy (carboplatin and pemetrexed) for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) exon 20 insertion mutations. “People living with advanced or metastatic NSCLC with activating EGFR exon 20 insertion mutations generally face a poor survival probability, and new treatment options are urgently needed from the very first line of therapy,” said Martin Vogel, EMEA Therapeutic Area Lead Oncology, Janssen-Cilag GmbH.

Lilly's Mirikizumab Helped Patients With Crohn's Disease Achieve Long-Term Remission in Phase 3 Trial

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that mirikizumab (an investigational interleukin-23p19 antagonist) met the co-primary and all major secondary endpoints compared to placebo in VIVID-1, a Phase 3 study evaluating the safety and efficacy of mirikizumab for the treatment of adults with moderately to severely active Crohn's disease. The double-blind, treat-through trial included mirikizumab, placebo and active control (ustekinumab) arms. Crohn's disease is a form of inflammatory bowel disease (IBD) that can cause systemic inflammation manifested as abdominal pain, diarrhea, fever and weight loss. It can lead to intestinal obstruction, fibrosis and other complications.

Lilly to Acquire POINT Biopharma to Expand Oncology Capabilities into Next-Generation Radioligand Therapies

BioNJ Member Eli Lilly and Company, with a site in Branchburg, and POINT Biopharma Global, Inc. announced a definitive agreement for Lilly to acquire POINT, a radiopharmaceutical company with a pipeline of clinical and preclinical stage radioligand therapies in development for the treatment of cancer. POINT's lead programs are in late-phase development. PNT20021 is a prostate-specific membrane antigen (PSMA) targeted radioligand therapy in development for patients with metastatic castration-resistant prostate cancer (mCRPC) after progression on hormonal treatment. Topline data from this study are expected in the fourth quarter of 2023. PNT20031 is a somatostatin receptor (SSTR) targeted radioligand therapy in development for the treatment of patients with gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Beyond the late-stage clinical pipeline, POINT has several additional programs in earlier stages of clinical and preclinical development. 

GSK and Zhifei Announce Exclusive Strategic Vaccine Partnership in China

Warren-based BioNJ Member GSK announced that it has reached an exclusive agreement with Chongqing Zhifei Biological Products, Ltd. (Zhifei) to co-promote GSK’s shingles vaccine, Shingrix, in China for an initial three-year period, with the potential to extend the partnership should all parties agree. Zhifei, the largest Chinese vaccine company by revenue, has a track record of driving access to innovative vaccines in China. By bringing together the scale and expertise of the two companies, the strategic partnership will significantly extend the availability of Shingrix, supporting the rapid expansion of patient access to the vaccine and future potential indications. Set to start on 1 January 2024, Zhifei will have exclusive rights to import and distribute Shingrix in China – focusing on promoting the vaccine through its extensive service network, which covers more than 30,000 vaccination points across the country. 

“LEQEMBI® Intravenous Infusion” (Lecanemab) Approved for the Treatment of Alzheimer’s Disease in Japan

Nutley-based Eisai Co., Ltd. and BioNJ Member Biogen Inc. announced that humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody “LEQEMBI® Intravenous Infusion” (200 mg, 500mg, lecanemab) has been approved in Japan as a treatment for slowing progression of mild cognitive impairment (MCI) and mild dementia due to Alzheimer’s disease (AD). LEQEMBI is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril*) and insoluble forms of Aβ. LEQEMBI is the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline by selectively binding to and eliminating the most toxic Aβ aggregates (protofibrils) that contribute to neurotoxicity in AD.

European Medicines Agency Committee for Orphan Medicinal Products Provides Positive Opinion on Calliditas’ Application for Setanaxib in Alport Syndrome

Hoboken-based Calliditas Therapeutics announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the company’s application for orphan drug designation in the European Union (EU) for setanaxib in Alport syndrome. The COMP opinion will now go to the European Commission, which is responsible for adopting the decision in relation to the application for orphan designation and adding it to the Community register of orphan medicinal products for human use. “We are pleased that the COMP has issued a positive opinion for orphan drug designation for setanaxib and are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products,” said CEO Renée Aguiar-Lucander. 

Calliditas and STADA Announce the Filing for Full Marketing Authorization of Kinpeygo® in the EU Partners

Hoboken-based Calliditas Therapeutics and STADA announced the submission of a request to the European Medicines Agency (EMA) for the Committee for Medicinal Products for Human Use (CHMP) to convert the conditional marketing authorization for Kinpeygo®, their treatment for primary IgA nephropathy (IgAN), to standard, or “full”, marketing authorization. Kinpeygo is currently approved under conditional approval to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram. This was granted in the interest of public health because the medicine addresses an unmet medical need, and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required.

Calliditas Therapeutics Granted Orphan Drug Designation by the FDA for the Treatment of Alport Syndrome With Setanaxib

Hoboken-based Calliditas Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib. Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled Phase 2 clinical study in Alport syndrome with around 20 patients in the fourth quarter of 2023. “We are excited to start another clinical program in the renal space targeting an orphan indication where today there are no approved products,” said CEO Renée Aguiar-Lucander. Alport syndrome is a genetic disorder arising from the mutations in the genes that code for type 4 collagen. The type 4 collagen alpha chains are primarily located in the kidneys, eyes and cochlea and thus the condition is characterized by kidney disease, loss of hearing and eye abnormalities. 

Calliditas Presents Data from the NefIgArd Phase 3 Trial at the 17th International Symposium on IgA Nephropathy (IIgANN) Tokyo

Hoboken-based Calliditas Therapeutics announced the presentations of new biomarker and subgroup analyses from the Phase 3 NefIgArd study with Nefecon (TARPEYO® (budesonide) delayed release capsules/Kinpeygo®) in adults with Primary IgA nephropathy (IgAN). “Taken together, the positive findings from our biomarker and patient subgroup analyses from our Phase 3 NefIgArd study indicate that Nefecon treatment results in a coordinated immunological response with the potential to modulate the intestinal immune network responsible for IgA production. These additional findings further reinforce the potential of TARPEYO in enabling physicians to proactively manage and potentially mitigate the impact of the disease by targeting the source and slowing kidney function decline, said Richard Phillipson, Chief Medical Officer at Calliditas.

Vyluma Announces Positive Results from Phase III CHAMP Study of NVK002 for Treatment of Myopia in Children

Bridgewater-based Vyluma Inc. announced positive top-line results from the second stage of its Phase III CHAMP (Childhood Atropine for Myopia Progression) clinical study of its lead compound, NVK002 (low dose atropine 0.01%), a proprietary, investigational, preservative-free eye drop administered nightly and intended for patients ages 3 to 17. An analysis of the results of this multi-center, international study conducted after 4 years of treatment and follow up, demonstrated strong safety, the absence of rebound upon washout of the study drug and continued efficacy for NVK002 as a potential treatment for myopia in children. Navneet Puri, Ph.D., the company’s Founder and Chairman, noted NVK002 currently is under FDA review with a Prescription Drug User Fee Act (PDUFA) date of January 31, 2024 set.

FDA Grants Transcenta Clearance to Proceed With Global Phase III Trial of Osemitamab (TST001) as First-Line Treatment for Gastric/Gastroesophageal Cancer Patients

Princeton-based Transcenta Holding Limited announced that FDA has granted it clearance to proceed with TranStar 301 global Phase III pivotal trial of Osemitamab (TST001) in combination with Nivolumab and chemotherapy as first-line treatment in patients with HER2-negative, CLDN18.2 expressing locally advanced or metastatic gastric or gastroesophageal (G/GEJ) adenocarcinoma. This clearance marks a major step forward in the global development of Osemitamab (TST001) and another important milestone following the approvals by the Center for Drug Evaluation (CDE) in China and MFDS in South Korea for the Phase III pivotal trial of Osemitamab (TST001) in July 2023. This milestone marks a crucial advancement in the progression of Osemitamab (TST001) toward becoming a global therapy that elevates the current standard of care for HER2-negative metastatic gastric or gastroesophageal (G/GEJ) adenocarcinoma. 

Tris Pharma Secures First Ex-US Authorizations for Quillivant ER Oral Suspension and Quillivant ER Chewable Tablet for the Treatment of Attention Deficit Hyperactivity Disorder

Monmouth Junction-based Tris Pharma, Inc. announced that Health Canada has authorized the use of Quillivant® ER (extended release) for the treatment of attention deficit hyperactivity disorder (ADHD) in children ages 6 to 12. Quillivant ER will be available as both an oral suspension and chewable tablet (known in the United States as Quillivant XR® and QuilliChew ER®, respectively). Quillivant ER is the first once-daily, long-acting, chewable and oral suspension formulations of methylphenidate to be approved for the treatment of ADHD in Canada. Ontario-based KYE Pharmaceuticals, Inc. will make Quillivant ER Oral Suspension and Quillivant ER Chewable Tablets available to patients in Canada beginning in Q1 2024. Tris will manufacture Quillivant ER in the United States and export to KYE Pharmaceuticals for Canadian patients.

Datopotamab Deruxtecan Demonstrated Statistically Significant and Clinically Meaningful Progression-Free Survival Benefit in Patients With HR Positive, HER2 Low or Negative Breast Cancer in TROPION-Breast01 Phase 3 Trial

Daiichi Sankyo, with a site in Basking Ridge, announced positive topline results from the TROPION-Breast01 Phase 3 trial showed datopotamab deruxtecan (Dato-DXd) demonstrated a statistically significant and clinically meaningful improvement for the primary endpoint of progression free survival (PFS) compared to investigator’s choice of chemotherapy in patients with inoperable or metastatic hormone receptor (HR) positive, HER2 low or negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer previously treated with endocrine-based therapy and at least one systemic therapy. Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo. Data for the dual primary endpoint of overall survival (OS) were not mature at this interim analysis and the trial will continue as planned to assess OS.

Ascendis Pharma Presents Results from Long-Term enliGHten Trial of TransCon™ hGH in Pediatric Growth Hormone Deficiency

Ascendis Pharma, with a site in Princeton, announced results from enliGHten, the company’s open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH as a once-weekly treatment for children and adolescents with growth hormone deficiency (GHD). The enliGHten trial enrolled 298 participants (mean age 10.3 years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients switching from daily somatropin treatment. Patients in these trials received a total of up to 6 years of treatment with TransCon hGH. At the time of the enliGHten Trial conclusion, 81 participants were designated as treatment completers, based on their physician’s determination that treatment for pediatric GHD was no longer required.

Ascendis Pharma Launches SKYTROFA® (lonapegsomatropin) in Germany for the Once-Weekly Treatment of Children and Adolescents With Growth Hormone Deficiency

Ascendis Pharma, with a site in Princeton, announced the launch in Germany of SKYTROFA® (lonapegsomatropin), its growth hormone approved in the European Union for the once-weekly treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient endogenous growth hormone secretion (growth hormone deficiency, or GHD). “SKYTROFA is designed to deliver unmodified somatropin, the same growth hormone used in daily pediatric GHD treatments for more than 30 years, and we are pleased now to make it available in Germany,” said Camilla Harder Hartvig, Executive Vice President and Global Chief Commercial Officer at Ascendis Pharma. “This first European Union launch of SKYTROFA begins acceleration of its commercialization beyond the U.S. as we work to expand our leadership to new geographies.”

Zatolmilast, an Investigational Treatment for Fragile X Syndrome, Receives Rare Pediatric Disease Designation from the U.S. FDA

Shionogi & Co., Ltd., with a site in Florham Park, announced the U.S. Food and Drug Administration (FDA) has granted Tetra Therapeutics Inc. (hereafter "Tetra"), a Shionogi Group Company, Rare Pediatric Disease Designation for zatolmilast (BPN14770), an investigational treatment being studied for Fragile X syndrome (FXS), a leading cause of inherited intellectual disability and autism. FDA grants Rare Pediatric Disease Designation (RPD) for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The RPD designation allows Tetra to request a priority review voucher from FDA which, if granted, may be used for a subsequent human drug application. Zatolmilast was also awarded Orphan Drug Designation by the FDA in 2018.

Temtokibart Achieves First Subject First Treatment (FSFT) Milestone in Phase 2b Trial

LEO Pharma, with a site in Madison, announced the First Subject First Treatment (FSFT) milestone for the Phase 2b clinical trial for temtokibart, LEO Pharma's IL-22RA1 antibody. The first patient dose was successfully administered on the 4th of October at the DermEdge Research Center in Ontario, Canada. Temtokibart is an investigational monoclonal antibody currently in Phase 2 development for the treatment of moderate-to-severe atopic dermatitis (AD). The drug targets the IL-22RA1 receptor subunit thereby inhibiting the effect of the interleukin-22 (IL-22) cytokine – known to be elevated in patients with AD. As IL-22RA1 is also involved in IL-20 and IL-24 signalling, temtokibart potentially also inhibits the effects of those two cytokines; however, this is not yet fully understood. Following the completion of the Phase 2a trial, LEO Pharma has now initiated the LP0145-2240 trial.

Bausch + Lomb Closes $2.5B XIIDRA Acquisition

Bausch + Lomb, with a site in Bridgewater, unveiled plans to acquire a dry eye disease treatment from Novartis for up to $2.5 billion. Now, the eye health company has closed the deal. The purchase of XIIDRA, a 5% a non-steroid eye drop, adds to Bausch + Lomb’s dry eye treatments, which includes MIEBO (perfluorohexyloctane ophthalmic solution), a medication that was cleared in May by the U.S Food and Drug Administration as the only approved eye drop for the condition that directly targets tear evaporation. “We expect to quickly take a leading position in the growing prescription dry eye category with the XIIDRA acquisition and MIEBO launch and, importantly, help the millions of patients not currently receiving adequate treatment for dry eye disease,” Bausch + Lomb Chairman and CEO Brent Saunders said. 

Couragene Co-Founders Awarded $40M NIH Grant

The team behind North Brunswick-based genetic medicines company Couragene is getting a big boost from the National Institute of Health’s Somatic Cell Genome Editing program. The company announced that co-founders Dr. Yong-Hui Jiang and Jianbing Zhou were awarded a competitive UG3/UH3 grant totaling approximately $40 million, contingent on milestones. According to Couragene, the funding is a “momentous” step toward leveraging genome editing to combat neurogenetic diseases. The grant was awarded to the Yale School of Medicine to support the development of gene-editing platform technology that can reach the human brain.

Bayer Fund Awards $7.4M to Nonprofit Organizations Throughout U.S. in 1st Half of 2023

Bayer Fund, a philanthropic arm of Whippany-based Bayer, awarded a total of $7.4 million in grant funding, spread across 1,800 organizations, during the first half of 2023. This latest round of grants raised Bayer Fund’s total giving to over $95 million since 2017 to nonprofit organizations across the U.S. Each Bayer Fund grant awarded goes toward stellar programs that are tackling some of the country’s most pressing challenges — combating malnutrition, enhancing science, technology, engineering and math education in schools and providing support services for patients and families managing cardiovascular disease and cancer. “With so many Americans facing challenges, Bayer Fund is proud to continue to support organizations to help strengthen communities across the U.S.,” Al Mitchell, President of Bayer Fund, said. 

NJ’s Best Places to Work 2023, Ranked

Earlier this year, NJBIZ released the finalists for the 2023 Best Places to Work in New Jersey awards. Now, we’re unveiling the complete list — ranked. The results are determined through employees’ participation with the Best Places to Work survey from BridgeTower Media firm Best Companies Group. The program had an unprecedented number of companies complete the process this year, so NJBIZ increased the number of winners to 150, up from 125 in 2022. To be eligible for consideration, organizations must meet several criteria, including having a facility in New Jersey. Congratulations to BioNJ Members Genmab US Inc., RSM US LLP, JLL (Jones Lang LaSalle) and EisnerAmper for making the list.

NJEDA Angel Tax Credit Approvals Drive Over $63 Million in Private Sector Investment into New Jersey Companies in 2023

The New Jersey Economic Development Authority (NJEDA) announced that 28 emerging New Jersey technology businesses benefitted from $63.2 million in private investment as supported in 2023 by the New Jersey Angel Investor Tax Credit Program (ATC). The NJEDA approved 207 investor applications in 2023, for a total of more than $8.2 million in tax credits, based on a percentage of eligible investment. Approved investments support emerging New Jersey-based companies in the life sciences, technology and clean technology sectors. Since the ATC program’s inception in 2013, 2,900 investments totaling $872 million have been approved to support 141 New Jersey companies.

The NJSBDC - Small Business Development Center - Offers Coaching and Guidance to NJ Companies Looking to Apply to the SBIR/STTR Program

The New Jersey Small Business Development Centers (NJSBDC) network provides comprehensive business advisory services and training to small and medium businesses (SMBs) and high-impact, growth-oriented businesses to maximize opportunities for growth and generate economic impact statewide. Services through the NJSBDC Technology Commercialization Program include guidance and assistance in pursuing SBIR/STTR programs, assistance in drafting proposals and written critiques of proposals with specific suggestions as to how to strengthen in order to be competitive in winning such grant awards. Contact Christian Pichardo at Christian.Pichardo@njsbdc.com for assistance.

Singer Bill that Promotes Innovation Through Expanded Grant Opportunities Signed into Law

Legislation sponsored by Senator Robert Singer that will expand certain grant opportunities for individuals employed at State colleges and universities was signed into law by Governor Murphy. “I’m proud this bill was signed into law. Lifting the prohibition on State grants for certain researchers will promote innovation and help integrate more advanced technologies into New Jersey industries,” said Singer (R-30). “This legislation will allow certain researchers employed at our public colleges and universities to obtain State grants issued by the Commission on Science, Innovation and Technology.”

Rutgers Gets $4.2M Grant to Study Alzheimer’s/Dementia in AAPI Populations

Rutgers Institute for Health, Health Care Policy and Aging Research, working in collaboration with New York University, has received more than $4.2 million in funding from the National Institute on Aging to develop the Resource Center for Alzheimer’s and Dementia Research in Asian and Pacific Americans. William Hu, Interim Director of the Center for Healthy Aging at the Rutgers Institute for Health, said the center’s goal is to use the five-year grant to advance behavioral, social and economic research related to Alzheimer’s disease and other dementias, especially in older Asian and Pacific Americans. RCASIA is the first NIH-funded center focused on Alzheimer’s and dementia research in New Jersey and the first NIA Resource Center for Minority Aging Research focused on Alzheimer’s and dementia in Asian and Pacific Americans.

Rutgers Research and Innovation Annual Impact Report

Senior Vice President for Research, Michel E. Zwick, Ph.D., of Rutgers University reported in Rutgers Research and Innovation Annual Impact Report, “Rutgers faculty's research, scholarship and creative endeavors pave the way for innovative solutions to global issues. Our faculty, students and staff continue to make us proud as they strive for excellence in their fields of study. The continued growth of the Rutgers research and innovation enterprise results from our dedication to New Jersey and the global community. Whether we are tackling global climate change, reducing Alzheimer's risk, or discovering new treatments for diseases, they pave the way for innovation and seek to improve our world. I am proud of the continued achievements of our faculty, staff and students. The Office for Research is committed to supporting and being a part of each researcher’s team.” 

A First for Rowan: Researcher Named an NIH New Innovator

A Rowan University psychologist has received the National Institutes of Health (NIH) Director’s New Innovator Award, the first researcher at the 100-year-old academic institution to do so. The award is part of the agency’s High-Risk, High-Reward Research program, which supports highly innovative scientists who propose visionary and broadly impactful behavioral and biomedical research projects. Dani Arigo, Ph.D., an associate professor of psychology in Rowan’s College of Science & Mathematics, will use the five-year, $1.5 million grant to pursue the next frontier in understanding the many ways people are influenced by social comparisons. The grant will support a full-time research coordinator; two Ph.D. students in the clinical psychology program; six undergraduate students and contributions from additional Ph.D. students and a postdoctoral research fellow. 

Hackensack Meridian Health Research Institute Creates 1st Spinoff Company, EValuate Diagnostics

Hackensack Meridian Health is spinning off its first company based on science from its research institute. The company, EValuate Diagnostics, will offer tests that identify disease biomarkers for the early detection of tumors and other diseases. The company is based upon the work of Olivier Loudig, an associate member of the Hackensack Meridian Center for Discovery and Innovation, who is directly involved with the development of EValuate Diagnostics. His laboratory at the CDI has developed research programs for biomarker discovery and early detection of breast, lung and prostate cancers using liquid biopsies. A core tenet of the mission of the CDI, founded in 2019, is translating cutting-edge science to rapidly impact patient health for New Jersey’s largest and most comprehensive health network. 

Lozano Promoted to EVP, Chief Growth Officer at HMH

Jose Lozano has been appointed Executive Vice President, Chief Growth Officer, Hackensack Meridian Health. In this role, Mr. Lozano will assume responsibility for facilities and nonclinical transformation and growth initiatives — in addition to his existing responsibilities, which include HMH’s real estate portfolio and strategic partnerships. He will report to CEO Bob Garrett for these responsibilities. “As the Chief Growth Officer, Mr. Lozano will be tasked with driving the expansion of HMH through identifying and pursuing key regional, national and international business opportunities as well as furthering development within its real estate and facilities footprint,” Mr. Garrett said.

RWJBarnabas Health Announces New Leaders to Executive Leadership Team

RWJBarnabas Health, announced that it has appointed Frank Pipas as Executive Vice President, Chief Financial Officer and Balpreet Grewal-Virk, Ph.D., as Senior Vice President, Community Health. In addition, several executives have transitioned to new roles, including Indu Lew, PharmD, as Chief of Staff to Mark E. Manigan, President of RWJBarnabas Health; Robert T. Adamson, PharmD, FASHP, as Executive Vice President and Chief Information Officer; and Robert Pellechio, RPh, MBA, as Senior Vice President and Chief Pharmacy Officer. “This is an exciting and pivotal time for RWJBarnabas Health, as each executive brings significant experience and fresh perspectives to their roles,” said Mr. Manigan. 

BioCentriq Expands Executive Leadership; Appoints Jennifer Manning as COO

BioCentriq announced it has appointed Jennifer Manning as Chief Commercial Officer. “We are thrilled to welcome Jennifer to the BioCentriq leadership team,” BioCentriq founder and CEO Haro Hartounian said. “Jennifer is a driven and energetic leader with a track record of building high-performing business development teams. She brings a wealth of expertise that will be instrumental in driving BioCentriq’s growth and success. Jennifer’s leadership, long-term vision and dedication to fostering strategic partnerships align perfectly with our mission to accelerate the development and manufacture of life-changing immunotherapies.” Ms. Manning’s 25 years of life sciences experience spans biotech and contract development and manufacturing organizations. Most recently, she served as the Senior Vice President of Global Strategic Partnerships at the Center for Breakthrough Medicines. 

NJIT Awarded $6M Grant to Accelerate Research in Health Care, Energy and Tech

New Jersey Institute of Technology said it secured a $6 million grant from the National Science Foundation to translate science and engineering discoveries into market-ready technologies that will improve quality of life in areas such as health care, sustainable energy and data privacy. Awarded by the NSF’s Directorate for Technology, Innovation and Partnerships, the grant will accelerate the development of promising prototypes and enable market validation and other commercialization activities. The funding also will strengthen the university’s entrepreneurial culture through training workshops in technology translation for undergraduates, Ph.D. students, post-doctoral researchers and faculty, through a new Center for Translational Research.

GSK Announces Wendy Becker to Join the Board as Non-Executive Director

Warren-based BioNJ Member GSK announced that Wendy Becker will join the Board of the Company as a Non-Executive Director. On joining the Board, Ms. Becker will become a member of the Company’s Remuneration Committee. She will succeed Urs Rohner as Chair of the Remuneration Committee after the Company’s 2024 Annual General Meeting when Mr. Rohner steps down on completion of his nine years’ service to the Board. Ms. Becker is currently Chair of Logitech International S.A., and a Board member and Chair of the Compensation Committee of Sony Group Corporation. She has strong executive management experience including senior roles at McKinsey & Company, TalkTalk, and Vodafone and as CEO at Jack Wills.  

Lilly Announces Leadership Transitions

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced changes to its executive leadership team. Mike Mason, Executive Vice President and President of Lilly Diabetes and Obesity, will retire from Lilly at the end of 2023. Patrik Jonsson will assume leadership of Lilly Diabetes and Obesity as Executive Vice President and President, effective January 1, 2024, in addition to his current responsibility as Executive Vice President and President of Lilly USA. Daniel Skovronsky, M.D., Ph.D., Lilly's Chief Scientific Officer and President of Lilly Research Laboratories, will take on the additional role of President of Lilly Immunology from Jonsson. In addition, Leigh Ann Pusey, Executive Vice President of Corporate Affairs and Communications, has decided to leave the company at the end of 2023. 

Globally Known Professor and Dermatologist Joins LEO Pharma

LEO Pharma, with a site in Madison, announced the appointment of Professor Alexander Egeberg, a globally recognized dermatologist and top key opinion leader as the company’s new Vice President, Head of Global Medical Affairs. Alexander Egeberg joins LEO Pharma from a role as Professor of Dermatology at the University of Copenhagen and head of one of the largest clinics in Northern Europe for biologic therapy at Bispebjerg Hospital's Department of Dermatology. He is a globally known professor and a board-certified dermatologist with an extensive research and clinical background. Having authored over 300 international scientific publications, he is an expert in inflammatory skin diseases with extensive experience in epidemiology, real-world evidence, translational medicine and clinical trials. 

Tom Garner Joins Lexicon as Senior Vice President and Chief Commercial Officer

Lexicon Pharmaceuticals, Inc., with a site in Bridgewater, announced that Tom Garner is joining the company as Senior Vice President and Chief Commercial Officer. Mr. Garner has over 25 years of pharmaceutical industry experience, having started his career in the United Kingdom with roles of increasing seniority at Boehringer Ingelheim and Eli Lilly before joining Bristol Myers Squibb (BMS) in 2002. During his time at BMS, Mr. Garner held a broad array of roles throughout BMS’ commercial organization including positions at local, country and global levels, and across a diverse range of brands and therapeutic areas. In his most recent role, Mr. Garner was the Senior Vice President and Head of the U.S. Cardiovascular and Established Brands business unit.

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

NJADDC Oncology Symposium

Hosted by New Jersey Academic Drug Discovery Consortium

November 16, 2023

The 2023 NJADDC symposium will bring academic, industry, and hospital system partners together for an engaging networking opportunity with the goal of developing regional partnerships, enhancing NJ as a collaborative nexus, and showcasing oncology research from across the state. This year's event will be hosted at Princeton University's Frick Chemistry Laboratory.