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New Analysis Shows PBMs Use Fees as a Profit Center

Pharmacy benefit managers (PBMs) demand double the amount of fees today than they did five years ago, according to a new report from Nephron Research. While there’s rightfully been more of a focus on the significant rebates PBMs negotiate, this report shows how the PBM business model keeps shifting to maximize profits and avoid policymakers’ efforts to enhance transparency and accountability. Here are four key findings from the report, which includes both Nephron’s existing research and new data on PBM compensation from biopharmaceutical manufacturers from 2018 to 2022.

It’s Time to Put Patients Before Middlemen and Reform the PBM Industry

When it comes to the alphabet soup of acronyms in health care, it can be hard to keep up. Whether it is the difference between an HMO and a PPO, the ACA versus the AMA, or a litany of other examples, acronyms have become so commonplace that there is an entire Wikipedia article dedicated to them. Recently, three buzzworthy letters have taken the health care industry and its regulators in Congress by storm: PBM. Also known as pharmacy benefit managers, PBMs were created in the 1960s around the same time health insurers began covering prescription drugs. Over the years, as the scope and cost of prescription drugs have grown, PBMs have consolidated their power and entangled themselves in our health care supply chain.

Correcting the Record: IRA’s Government Price Setting Provisions Are Unconstitutional

Let’s start by making one thing clear: The price setting program is not voluntary. Manufacturers are forced between a rock and a hard place under the price setting scheme. Once a manufacturer’s medicine is selected for price setting, there are only two options if the manufacturer does not capitulate to the government and its unilaterally determined price.  

The Reality of Drug Pricing Negotiations Isn't a Slam Dunk for Patients

Roughly two-thirds of non-dual eligible Medicare beneficiaries live with multiple chronic conditions. For these patients, many of whom rely on prescription drugs to manage their health, the impacts of the new program could be considerable. These unprecedented negotiations between the federal government and drug makers, authorized in last year's Inflation Reduction Act, will fundamentally alter Medicare's "Part D" prescription drug benefit – in obvious and not-so-obvious ways.

Biden’s Price Control Scheme Harms Patients, Compromises Free Market

The Biden administration named the first 10 prescription drugs that will be subject to new government price controls. The novel power is derived from the Inflation Reduction Act (IRA), which permits the federal government to price-fix certain medications that are accessed through Medicare. Democrats will cheerlead the heavy-handed move as a win while ignoring the unintended consequences. But, to the detriment of patients and the free market, the fallout will far outweigh the benefits. 

It’s PBMs, Not Patents, Blocking Competition

The Biden administration named the first 10 prescription drugs that will be subject to new government price controls. The novel power is derived from the Inflation Reduction Act (IRA), which permits the federal government to price-fix certain medications that are accessed through Medicare. Democrats will cheerlead the heavy-handed move as a win while ignoring the unintended consequences. But, to the detriment of patients and the free market, the fallout will far outweigh the benefits. 

VT’s Grumpy Old Men on Price Controls and IP Attacks

The U.S. Congress and Biden Administration’s attacks on IP and Drug Pricing continue unabated as the 2024 election approaches. In this Vital Health Podcast, VT’s Grumpy Old Men (Duane Schulthess, Joe Hammang and Harry P. Bowen) are particularly grumpy about the current state of affairs and dig into their research findings on the Inflation Reduction Act. They also ruminate on what the continued ratcheting-up of rhetoric and legislation against the industry means for tough-to-treat neurological disorders, late-stage cancers and orphan diseases.

ROI Influencers: People of Color 2023 — Top 25

Pavita Howe, Vice President, Innovation & Entrepreneurship, BioNJ

One of the sharpest minds in the entrepreneurial space, she is an expert at helping start-ups commercialize new technologies, launch new products, formulate business plans, pitch to investors and drive business development.

Pfizer and BioNTech Receive U.S. FDA Approval for 2023-2024 COVID-19 Vaccine | Business Wire

BioNJ Member Pfizer, with a site in Peapack, and BioNTech SE announced that the U.S. Food and Drug Administration (FDA) approved the supplemental Biologics License Application (COMIRNATY 2023-2024 Formulation) for individuals 12 years and older and granted emergency use authorization for individuals 6 months through 11 years of age for the companies’ Omicron XBB.1.5-adapted monovalent COVID-19 vaccine. This season’s vaccine is indicated as a single dose for most individuals 5 years of age and older. Children under the age of 5 may be eligible to receive additional doses of this season’s vaccine if they have not already completed a three-dose series with previous formulations of a COVID-19 vaccine.

European Commission Approves Pfizer’s LITFULO™ for Adolescents and Adults With Severe Alopecia Areata

BioNJ Member Pfizer Inc., with a site in Peapack, announced that the European Commission (EC) has granted marketing authorization for LITFULO™ (ritlecitinib) to treat adults and adolescents 12 years of age and older with severe alopecia areata. LITFULO, a once-daily oral capsule, is the first medicine authorized by the EC to treat individuals as young as 12 years of age with severe alopecia areata. LITFULO is also the first and only treatment to selectively inhibit Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases. The marketing authorization for LITFULO is valid in all 27 EU member states, and in Iceland, Liechtenstein, and Norway. This authorization follows the recommendation for approval by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2023.

Cellares and Lyell to Evaluate Automated Manufacturing of Lyell's CAR T-Cell Therapy on Cellares' Cell Shuttle Platform

Bridgewater-based BioNJ Member Cellares, the first Integrated Development and Manufacturing Organization (IDMO) dedicated to clinical and industrial-scale cell therapy manufacturing, and Lyell Immunopharma, a clinical stage T-cell reprogramming company advancing a diverse pipeline of cell therapies for patients with solid tumors, today announced Lyell will evaluate Cellares' automated manufacturing platform, the Cell Shuttle™, through Cellares' Technology Adoption Partnership (TAP) program. As part of the collaboration, the companies have agreed on a proof-of-concept technology transfer process for the manufacture of Lyell's LYL797 CAR T-cell therapy, using the Cell Shuttle. LYL797 is an investigational CAR T-cell therapy in development for the treatment of solid tumors that express ROR1, a protein present on the surface of various solid tumors. LYL797 is enhanced with Lyell's novel genetic and epigenetic reprogramming technologies designed to generate highly tumor-reactive, longer-lasting functional T cells.

Palatin Completes Enrollment in Phase 3 Melody-1 Study of PL9643 for the Treatment of Patients With Dry Eye Disease

Cranbury-based BioNJ Member Palatin Technologies, Inc. announced that enrollment in the PL9643 MELODY-1 Phase 3 study in dry eye disease (DED) is complete, with 570 patients enrolled. The Company is on track to release topline results by the end of the fourth quarter of calendar year 2023. “We are committed to addressing unmet needs of DED patients. We believe that PL9643, with its novel mechanism of action and differentiated product profile for efficacy, safety and ocular tolerability, if approved, will be a first-in-class treatment option for the millions of patients who suffer from DED,” said Carl Spana, Ph.D., President and CEO of Palatin. “We are optimistic about results of the Phase 3 PL9643 data based on the positive interim analysis and are pleased to have completed enrollment so promptly.”

BeiGene Strengthens Global Portfolio and Regains Full Rights from Novartis for Anti-PD-1 Antibody TEVIMBRA® (tislelizumab)

BioNJ Member BeiGene, Ltd. with a site in Hopewell, announced that it has entered into an agreement with Novartis to regain worldwide rights to develop, manufacture and commercialize TEVIMBRA (tislelizumab). “We are excited to regain the global rights to TEVIMBRA, which enables us to build out our in-house solid tumor commercial capabilities and complements our deep pipeline presented at our recent R&D Day. With more than 12,000 patients enrolled in our TEVIMBRA global clinical trial program, we plan to rapidly accelerate our regulatory and development plans across a wider range of tumor types,” said John V. Oyler, Co-Founder, Chairman and CEO of BeiGene. “BeiGene will continue to work with Novartis on development, regulatory and manufacturing priorities. Novartis will manufacture TEVIMBRA for many markets worldwide and explore its potential in combination with their oncology assets.”

BeiGene Announces Positive Regulatory Updates in Europe and the U.S. After Recently Regaining Global Rights for TEVIMBRA®

BioNJ Member BeiGene, Ltd. with a site in Hopewell announced that the European Commission (EC) has approved TEVIMBRA® (tislelizumab) as monotherapy for the treatment of adult patients with unresectable, locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior platinum-based chemotherapy. Additionally, the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for tislelizumab as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic ESCC. BeiGene has launched more than 20 potentially registration-enabling trials with TEVIMBRA, of which 10 Phase 3 randomized trials and four Phase 2 trials have already had positive readouts.

First Wave Biopharma Announces Exclusive Global License Agreement for Capeserod from Sanofi

BioNJ Member First Wave BioPharma, Inc., announced an agreement with Bridgewater-based BioNJ Member Sanofi to license Capeserod, a selective 5-HT4 receptor partial agonist, which First Wave will repurpose and develop for gastrointestinal (GI) indications. Under the terms of the agreement, First Wave will receive from Sanofi an exclusive, global license for Capeserod and will assume responsibility for all future clinical development. The licensing agreement, which includes a modest upfront payment, backend milestone payments and single digit royalties on net sales, provides a right of first refusal for Sanofi to reacquire Capeserod following certain stages of clinical development and to commercialize the product.

Otsuka Pharmaceutical and Lundbeck Announce Topline Results from Two Phase 3 Trials of Brexpiprazole as Combination Therapy With Sertraline for the Treatment of Post-Traumatic Stress Disorder in Adults

Princeton-based BioNJ Member Otsuka Pharmaceutical Co., Ltd. and H. Lundbeck A/S announce results from two Phase 3 clinical trials of Otsuka’s brexpiprazole as combination therapy with sertraline for the treatment of post-traumatic stress disorder (PTSD). The first trial was a Phase 3, randomized, double-blind, 2-arm, flexible dose trial to evaluate the efficacy, safety, and tolerability of brexpiprazole (2 - 3 mg/day) as combination therapy with sertraline in 416 randomized adult subjects with PTSD. The second trial was a Phase 3, randomized, double-blind, 3-arm, fixed dose trial to evaluate the efficacy, safety and tolerability of brexpiprazole (2 or 3 mg/day) as combination therapy with sertraline in 553 randomized adult subjects with PTSD. 

Bristol Myers Squibb’s Investigational LPA1 Antagonist Reduces Rate of Lung Function Decline in Progressive Pulmonary Fibrosis Cohort of Phase 2 Study

Princeton-based BioNJ Member Bristol Myers Squibb announced results from a Phase 2 study evaluating BMS-986278, a potential first-in-class, oral, lysophosphatidic acid receptor 1 (LPA1) antagonist in patients with progressive pulmonary fibrosis (PPF). The study showed that twice-daily administration of 60 mg of BMS-986278 over 26 weeks reduced the rate of decline in percent predicted forced vital capacity (ppFVC) by 69% compared to placebo (overall, 38% of patients in the study received background antifibrotic therapy). This Phase 2 study was a global, randomized trial in which parallel cohorts of patients with idiopathic pulmonary fibrosis (IPF) and PPF received 30 mg or 60 mg of BMS-986278 or matched placebo orally twice-daily for 26 weeks. Stable background use of antifibrotics in the IPF cohort and/or select immunosuppressives in the PPF cohort were allowed. 

GSK and Save the Children Renew Award-Winning Partnership, With Focus on ‘Zero Dose’ Children Who Have Never Received a Vaccine

Warren-based BioNJ Member GSK and Save the Children have announced that they are renewing their award-winning partnership for a further five years. GSK will invest £15 million in the partnership with Save the Children and they will work together to increase child vaccination rates, with a focus on reducing the number of ‘zero dose’ children in Ethiopia and Nigeria. Featured prominently in Gavi, the Vaccine Alliance's Phase 5 strategy and World Health Organization's Immunization Agenda 2030, the 'zero dose' concept refers to children who have never received any routine vaccinations. Using their combined expertise and 10 years of experience working together, GSK and Save the Children will develop, pilot and implement tailored approaches to reach zero dose children in diverse settings.

GSK Regulatory Submission for Momelotinib for the Treatment of Myelofibrosis Accepted for Review by Japanese Regulator

Warren-based BioNJ Member GSK announced that the Ministry of Health, Labour and Welfare (MHLW), Japan, has accepted for review a new drug application (NDA) for momelotinib, a potential new medicine with a differentiated mechanism of action that may address the significant medical needs of myelofibrosis patients, especially those with anaemia. The NDA is based on data from the pivotal phase III trials SIMPLIFY-1 and MOMENTUM. Myelofibrosis is a blood cancer that can lead to splenomegaly (enlarged spleen); constitutional symptoms such as fatigue, night sweats, and bone pain; and severely low blood counts, including anaemia and thrombocytopenia. About 70% of patients diagnosed with primary myelofibrosis and about half of patients diagnosed with secondary myelofibrosis in Japan have moderate to severe anaemia at the time of diagnosis, and nearly all patients are estimated to develop anaemia over the course of the disease. 

Ojjaara (momelotinib) Approved in the U.S. as the First and Only Treatment Indicated for Myelofibrosis Patients With Anaemia

Warren-based BioNJ Member GSK announced that the U.S. Food and Drug Administration (FDA) has approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis, including primary myelofibrosis or secondary myelofibrosis (post-polycythaemia vera and post-essential thrombocythaemia), in adults with anaemia. Ojjaara is a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. To date, it is the only approved medicine for both newly diagnosed and previously treated myelofibrosis patients with anaemia that addresses the key manifestations of the disease, namely anaemia, constitutional symptoms, and splenomegaly (enlarged spleen). Myelofibrosis is a blood cancer affecting approximately 25,000 patients in the US. Myelofibrosis can lead to severely low blood counts, including anaemia and thrombocytopaenia; constitutional symptoms such as fatigue, night sweats and bone pain; and splenomegaly. 

Johnson & Johnson Marks New Era as Global Healthcare Company With Updated Visual Identity

For more than 135 years, New Brunswick-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson has provided health care products and solutions to people worldwide. Now, with its exclusive focus on healthcare innovation and tackling the toughest health challenges, the company is updating its brand and uniting both its MedTech and pharmaceutical segments under the Johnson & Johnson brand name to demonstrate its collective power in healthcare. The announcement marks the next era for Johnson & Johnson, which is leveraging its expertise in innovative medicine and medical technology to prevent, treat and cure complex diseases and introduce solutions that are smarter, less invasive and more personalized.

Janssen Submits Marketing Authorization Application to the European Medicines Agency Seeking Approval of Erdafitinib for the Treatment of Patients With Locally Advanced or Metastatic Urothelial Cancer With Susceptible FGFR Alterations

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval of erdafitinib for the treatment of adult patients with locally advanced unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations, with disease progression during or following at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-[L]1) inhibitor. Europe has one of the highest rates of bladder cancer in the world, with more than 203,000 patients diagnosed in 2020 alone. UC is the most common form and up to 20 percent of patients with metastatic UC (mUC) have FGFR alterations.

Treatment With RYBREVANT® (amivantamab-vmjw) and Lazertinib Plus Chemotherapy Showed Durable Progression-Free Survival in Patients With Previously Treated EGFR-Mutated Advanced Non-Small Cell Lung Cancer

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced follow-up results from the Phase 1b/2 CHRYSALIS-2 study cohort evaluating the safety and tolerability of the combination of RYBREVANT® (amivantamab-vmjw), a bispecific antibody targeting epidermal growth factor receptor (EGFR) and mesenchymal-epithelial transition (MET), with lazertinib, an oral third-generation EGFR tyrosine kinase inhibitor (TKI), plus platinum-based chemotherapy (carboplatin and pemetrexed) in patients with relapsed/refractory non-small cell lung cancer (NSCLC) and EGFR mutations. CHRYSALIS-2 (NCT04077463) is an ongoing, multicohort, clinical study evaluating RYBREVANT® in combination with lazertinib in patients with advanced NSCLC with EGFR exon 19 deletion mutations (ex19del) or L858R activating mutations.

U.S. FDA Approves Jardiance® for the Treatment of Adults With Chronic Kidney Disease

The U.S. Food and Drug Administration (FDA) has approved Jardiance® (empagliflozin) 10 mg tablets to reduce the risk of sustained decline in estimated glomerular filtration rate (eGFR), end-stage kidney disease, cardiovascular death and hospitalization in adults with chronic kidney disease (CKD) at risk of progression, BioNJ Member Eli Lilly and Company, with a site in Branchburg and Boehringer Ingelheim announced. "This approval provides healthcare professionals in the U.S. with another treatment option for adults with CKD that can reduce the risk of kidney function decline, kidney failure, cardiovascular death and hospitalizations," said Katherine Tuttle, M.D., Executive Director for Research, Providence Inland Northwest Health, Regional Principal investigator for the Institute of Translational Health Sciences and Professor of Medicine at the University of Washington, and EMPA-KIDNEY steering committee member.

Gilead’s Phase 2 EVOKE-02 Study of Trodelvy® (sacituzumab govitecan-hziy) in Combination With KEYTRUDA® (pembrolizumab) Demonstrates Promising Clinical Activity in First-Line Metastatic Non-Small Cell Lung Cancer

Parsippany-Troy Hills-based BioNJ Member Gilead Sciences, Inc. announced promising early data from the global, open-label, Phase 2 EVOKE-02 study evaluating Trodelvy® (sacituzumab govitecan-hziy) in combination with Merck’s anti-PD-1 therapy KEYTRUDA® (pembrolizumab) with or without platinum agents in patients with previously untreated advanced or metastatic non-small cell lung cancer (NSCLC) without actionable genomic alterations. The data from the EVOKE-02 study gives us confidence in the clinical activity of sacituzumab govitecan in combination with pembrolizumab in first-line metastatic NSCLC patients,” said Byoung Chul Cho, MD, PhD, Professor in the Division of Medical Oncology at Yonsei Cancer Center, Yonsei University College of Medicine.

CHMP Adopts Positive Opinion to Extend the Use of Veklury® (Remdesivir) to Treat COVID-19 in People With Hepatic Impairment

Parsippany-Troy Hills-based BioNJ Member Gilead Sciences, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) granted a positive opinion for the use of Veklury® (remdesivir) to treat people with COVID-19 with mild to severe hepatic impairment. The European Commission (EC) will review the CHMP recommendation and, if adopted, Veklury will become the first and only authorized antiviral COVID-19 treatment that can be used across all stages of liver disease. Europe has the highest burden of liver disease in the world and cases are expected to grow across many countries. Furthermore, people with liver disease represent a population that is highly vulnerable to COVID-19 and are at increased risk of morbidity and mortality.

Merck and Eisai Provide Update on Two Phase 3 Trials Evaluating KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) in Patients With Certain Types of Metastatic Non-Small Cell Lung Cancer

Rahway-based BioNJ Member Merck & Co. and Eisai provided updates on two Phase 3 trials, LEAP-006 and LEAP-008, evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, in patients with certain types of metastatic non-small cell lung cancer. In both the LEAP-006 and LEAP-008 trials, the safety profiles of the KEYTRUDA plus LENVIMA-based treatment regimens were consistent with that observed in previously reported studies evaluating the combination. A full evaluation of the data from these studies is ongoing. The companies will work with investigators to share the results with the scientific community.

Merck Announces Phase 3 KEYNOTE-A39/EV-302 Trial Met Dual Primary Endpoints of Overall Survival (OS) and Progression-Free Survival (PFS) in Certain Patients With Previously Untreated Locally Advanced or Metastatic Urothelial Cancer

Rahway-based BioNJ Member Merck & Co. announced positive topline results from the Phase 3 KEYNOTE-A39 trial (also known as EV-302), which was conducted in collaboration with Seagen and Astellas, evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with Padcev (enfortumab vedotin-ejfv) versus chemotherapy (gemcitabine plus cisplatin or carboplatin) in patients with previously untreated locally advanced or metastatic urothelial carcinoma (la/mUC). The trial enrolled patients who may or may not be eligible for treatment with cisplatin-based chemotherapy, regardless of PD-L1 status. In the trial, KEYTRUDA in combination with enfortumab vedotin met its dual primary endpoints of overall survival (OS) and progression-free survival (PFS), demonstrating a statistically significant and clinically meaningful improvement versus chemotherapy in patients with previously untreated la/mUC.

Merck Presents New Analyses Supporting the Promising Potential of Sotatercept, its Investigational Medicine for Adults With Pulmonary Arterial Hypertension (PAH)

Rahway-based BioNJ Member Merck & Co. announced new analyses from studies of sotatercept, Merck’s novel investigational activin signaling inhibitor biologic, for adults with pulmonary arterial hypertension (PAH) (WHO Group 1) at the European Respiratory Society (ERS) International Congress 2023. A new exploratory post-hoc analysis of right heart catheterization and echocardiography data from patients in the Phase 3 STELLAR study showed treatment with sotatercept for 24 weeks on top of background therapy reduced right heart size and improved right-ventricular (RV) function and hemodynamic status. This analysis was featured in an oral presentation, with simultaneous publication in the European Respiratory Journal. An interim analysis of the Phase 3 SOTERIA open-label extension study was also presented, representing the longest safety and efficacy analysis of sotatercept to date.

Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) as Adjuvant Treatment for Adults with Non-Small Cell Lung Cancer at High Risk of Recurrence Following Complete Resection and Platinum-Based Chemotherapy

Rahway-based BioNJ Member Merck & Co. announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, for the adjuvant treatment of adults with non-small cell lung cancer (NSCLC), who are at high risk of recurrence following complete resection and platinum-based chemotherapy. The recommendation is based on results from the Phase 3 KEYNOTE-091 trial, in which KEYTRUDA demonstrated a statistically significant improvement in disease-free survival (DFS) in patients with NSCLC who are at high risk of recurrence (stage IB [T2a ≥4 centimeters], II or IIIA), and clinically meaningful results in the patients who received adjuvant chemotherapy. 

FDA Grants Priority Review to Merck’s Application for KEYTRUDA® (pembrolizumab) Plus Concurrent Chemoradiotherapy as Treatment for Patients With Newly Diagnosed High-Risk Locally Advanced Cervical Cancer

Rahway-based BioNJ Member Merck & Co. announced the U.S. Food and Drug Administration (FDA) has accepted for priority review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with external beam radiotherapy (EBRT) plus concurrent chemotherapy, followed by brachytherapy (also known as concurrent chemoradiotherapy) as treatment with definitive intent for newly diagnosed patients with high-risk locally advanced cervical cancer. The sBLA is based on data from the KEYNOTE-A18 trial, also known as ENGOT-cx11/GOG-3047, in which KEYTRUDA plus concurrent chemoradiotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to concurrent chemoradiotherapy alone. If approved, this would be Merck’s third approved indication in cervical cancer and first in an earlier stage of the disease. 

FDA Accepts for Priority Review Merck’s Supplemental New Drug Application for WELIREG® (belzutifan) in Certain Previously Treated Patients With Advanced Renal Cell Carcinoma (RCC)

Rahway-based BioNJ Member Merck & Co. announced the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a supplemental new drug application (sNDA) seeking approval for WELIREG, Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, for the treatment of adult patients with advanced renal cell carcinoma (RCC) following immune checkpoint and anti-angiogenic therapies. The sNDA is based on data from the LITESPARK-005 trial, in which WELIREG demonstrated 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. 

AbbVie's SKYRIZI® (risankizumab) Met All Primary and Secondary Endpoints Versus Stelara® (ustekinumab) in Head-to-Head Study in Crohn's Disease

BioNJ Member AbbVie, with a site in Branchburg, announced top-line results from the Phase 3 SEQUENCE clinical trial evaluating risankizumab (SKYRIZI®, 600 mg intravenous [IV] induction at week 0, 4 and 8 and 360 mg subcutaneous injection [SC] starting at week 12 and every 8 weeks thereafter) versus ustekinumab (STELARA®, IV dose at week 0 and 90 mg SC every 8 weeks thereafter) through week 48 in patients with moderately to severely active Crohn's disease who have failed one or more anti-TNFs. "We are encouraged by these results, which demonstrate the impact SKYRIZI can have in helping patients achieve both clinical and endoscopic remission," said Roopal Thakkar, Senior Vice President, Development and Regulatory Affairs and Chief Medical Officer, AbbVie. "These head-to-head data reinforce SKYRIZI is an effective treatment option for patients living with Crohn's disease."

Novo Nordisk and UNICEF to Extend Joint Initiative to Prevent Global Childhood Obesity

Plainsboro-based BioNJ Member Novo Nordisk and UNICEF announced an extension of their long-term partnership to help prevent global childhood overweight and obesity – a public health crisis that affects millions of children worldwide. Over the next three years Novo Nordisk will commit 8 million U.S. dollars to scale and accelerate efforts to create healthier environments for children through policy implementation and innovations that enable children to eat well, play and be physically active. The aim is to positively impact at least 10 million children.With the intention to share best practices, knowledge and tools globally, the partnership is intensifying its efforts in Latin America and the Caribbean and expanding to the East Asia and the Pacific region with deeper work in Mexico, Colombia, Brazil and Indonesia. 

Cellectar Biosciences Receives European Medicines Agency Priority Medicines (PRIME) Designation for Iopofosine for Waldenstrom’s Macroglobulinemia

Florham Park-based Cellectar Biosciences, Inc. announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to iopofosine I 131, the company’s lead small-molecule drug candidate, for Waldenstrom’s macroglobulinemia (WM) in patients who have received two or more prior treatment regimens. The PRIME program aims to optimize development plans and speed up evaluation of medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options. These medicines are considered priority medicines by the EMA and are intended to reach patients earlier. To be accepted for PRIME, new therapies must demonstrate the potential to significantly address an unmet medical need in clinical trials.

Quizartinib Recommended for Approval in EU by CHMP for Patients With Newly Diagnosed FLT3-ITD Positive AML

Daiichi Sankyo, with a site in Basking Ridge, announced that quizartinib has been recommended for approval in the European Union (EU) in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by quizartinib single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from the Phase 3 QuANTUM-First trial, which were published in The Lancet. The recommendation will now be reviewed by the European Commission, which has the authority to grant marketing authorizations for medicines in the EU.

DS-3939 Enters Clinical Development in Patients Across Several Types of Advanced Solid Cancers

Daiichi Sankyo, with a site in Basking Ridge, announced that the first patient has been dosed in a first-in-human Phase 1/2 trial evaluating DS-3939 in patients with several types of advanced solid tumors including non-small cell lung, breast, urothelial, ovarian, biliary tract and pancreatic cancer. DS-3939 is a specifically engineered potential first-in-class tumor-associated mucin-1 (TA-MUC1) directed antibody drug conjugate (ADC) designed using Daiichi Sankyo’s proprietary DXd ADC technology. TA-MUC1 is a tumor-specific transmembrane glycoprotein and is overexpressed in most human epithelial cancers, making it a promising target for cancer therapy. Currently, there are no TA-MUC1 directed therapies approved for any type of cancer.

Omicron XBB.1.5-adapted COVID-19 Vaccine (DS-5670) Supplemental New Drug Application Submitted in Japan

Daiichi Sankyo, with a site in Basking Ridge, announced that it has submitted a supplemental New Drug Application (sNDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for an omicron XBB.1.5-adapted mRNA vaccine (DS-5670) against the novel coronavirus infectious disease (COVID-19) for booster vaccination for individuals aged 12 years or older. The monovalent vaccine containing omicron XBB.1 lineage will be used in the current special temporary vaccination program against COVID-19 in Japan that begins in September 2023, and Daiichi Sankyo is advancing production preparations to start the supply of DS-5670 within this year. DS-5670 is an mRNA vaccine against COVID-19 designed to produce antibodies against the receptor binding domain (RBD) of the spike protein of the novel coronavirus, utilizing a novel nucleic acid drug delivery system discovered by Daiichi Sankyo.

Daiichi Sankyo Announces Results of Booster Vaccination Trial of mRNA COVID-19 Vaccine DS-5670 (Bivalent: Original Strain and Omicron Strain) in Japan

Daiichi Sankyo, with a site in Basking Ridge, announced that the primary endpoint was achieved in a Phase 3 trial of a booster vaccination with DS-5670, an mRNA vaccine against the novel coronavirus infectious disease (COVID-19) being developed in Japan (in a booster vaccination trial) by Daiichi Sankyo. The booster vaccination trial was conducted as a phase 3 clinical study to evaluate the immunogenicity and safety of a booster vaccination with DS-5670 (bivalent, the original strain and omicron BA.4-5 subvariant) in individuals who were aged 12 years or older and had completed the primary and booster series of SARSCoV-2 vaccines. The primary endpoints of the booster vaccination trial were the geometric mean blood neutralizing antibody titer and immune response against SARS-CoV-2 (omicron BA.5 subvariant) four weeks after the investigational vaccination. 

ENHERTU® Demonstrated Strong and Durable Tumor Responses in Previously Treated HER2 Mutant Advanced Lung Cancer in DESTINY-Lung02 Phase 2 Trial

Daiichi Sankyo, with a site in Basking Ridge, announced results from the primary analysis of the DESTINY-Lung02 Phase 2 trial showed ENHERTU® (trastuzumab deruxtecan) continued to demonstrate strong and durable tumor responses in previously treated patients with HER2 mutant unresectable and/or metastatic non-squamous non-small cell lung cancer (NSCLC). ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. At the primary analysis, a confirmed objective response rate (ORR) of 49.0% (95% confidence interval [CI]: 39.0-59.1) in the 5.4 mg/kg arm and 56.0% (95% CI: 41.3-70.0) in the 6.4 mg/kg arm was observed as assessed by blinded independent central review (BICR).

Patritumab Deruxtecan Demonstrated Clinically Meaningful and Durable Responses in Patients With EGFR-Mutated Metastatic Non-Small Cell Lung Cancer in HERTHENA-Lung01 Phase 2 Trial

Daiichi Sankyo, with a site in Basking Ridge, announced results from the HERTHENA-Lung01 Phase 2 trial showed that patritumab deruxtecan (HER3-DXd) demonstrated clinically meaningful and durable responses in patients with EGFR-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC) following disease progression with an EGFR TKI and platinum-based chemotherapy. Patritumab deruxtecan is a specifically engineered potential first-in-class HER3 directed antibody drug conjugate (ADC) designed using Daiichi Sankyo’s proprietary DXd ADC technology. NSCLC accounts for approximately 85% of all lung cancers – 55% having distant spread at diagnosis – with EGFR-activating mutations occurring in 14% to 38% of all NSCLC tumors worldwide. After disease progression following treatment with an EGFR TKI and platinum-based chemotherapy, currently available therapies offer limited efficacy, highlighting the need for new approaches to improve outcomes.

Datopotamab Deruxtecan Plus Durvalumab Showed Promising Clinical Activity in the First-Line Advanced Non-Small Cell Lung Cancer Setting in TROPION-Lung04 Phase 1b Trial

Daiichi Sankyo, with a site in Basking Ridge, announced initial results from the TROPIONLung04 phase 1b trial showed that datopotamab deruxtecan (Dato-DXd) in combination with durvalumab, an anti-PD-L1 therapy, with or without carboplatin demonstrated encouraging responses and no new safety signals in patients with previously untreated advanced or metastatic non-small cell lung cancer (NSCLC) without actionable genomic alterations. Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo and AstraZeneca. More than one million people worldwide are diagnosed with advanced NSCLC each year. While first-line treatment with immune checkpoint inhibitors with or without chemotherapy has improved outcomes for patients with NSCLC without actionable genomic alterations, like EGFR or ALK, most patients eventually experience disease progression.

Ifinatamab Deruxtecan Continues to Demonstrate Durable Responses in Patients With Advanced Small Cell Lung Cancer in Early Trial

Daiichi Sankyo, with a site in Basking Ridge, announced updated results from a subgroup analysis of a Phase 1/2 trial showed that ifinatamab deruxtecan (I-DXd) continues to demonstrate durable responses in patients with heavily pretreated advanced small cell lung cancer (SCLC). Ifinatamab deruxtecan is a specifically engineered potential first-in-class B7-H3 directed antibody drug conjugate (ADC) designed using Daiichi Sankyo’s proprietary DXd ADC technology. A confirmed objective response rate (ORR) of 52.4% (95% CI: 29.8-74.3) was observed in 21 patients with advanced SCLC receiving ifinatamab deruxtecan (6.4 to 16.0 mg/kg) in the dose escalation part of the Phase 1/2 trial. One complete response (CR) and 10 partial responses (PRs) were seen. A median duration of response (DOR) of 5.9 months (95% CI: 2.8-7.5) was observed. 

Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease

Cranbury-based Rocket Pharmaceuticals, Inc. announced that alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease. Danon Disease is a uniformly fatal inherited cardiomyopathy that leads to mortality in the majority of male patients at age ~20 and females at age ~40, and for which there are no approved curative or disease-modifying therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe. The global, single-arm, multi-center Phase 2 pivotal trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon Disease, including a pediatric safety run-in (n=2), with a natural history comparator and a dose level of 6.7 x 1013 GC/kg.

Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock

Cranbury-based Rocket Pharmaceuticals, Inc. announced the pricing of an underwritten public offering of (i) 7,812,500 shares of its common stock at a public offering price of $16.00 per share and (ii) to certain investors, pre-funded warrants to purchase 3,126,955 shares of common stock at a price of $15.99 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.01 per share exercise price for each such pre-funded warrant. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be approximately $175 million. 

NJEDA Board Approvals Expand List of New Jersey Innovation Evergreen Fund Qualified Venture Firms

The New Jersey Economic Development Authority (NJEDA) approved three additional venture capital firms as Qualified Venture Firms (QVFs) in the New Jersey Innovation Evergreen Fund (NJIEF). With these latest approvals, a total of 10 QVFs can each access up to $12.5 million annually per investor from the NJIEF to co-invest in innovative, high-growth New Jersey-based businesses. Established under the New Jersey Economic Recovery Act (ERA) of 2020, signed into law by Governor Phil Murphy in 2021, the NJIEF allows the State to become an equity investor in innovative early stage businesses based in New Jersey, investing up to $300 million in New Jersey companies alongside approved QVFs for a total of $600 million.

Governor Murphy Announces Additional $20M in Funding for NJ Manufacturing Voucher Program

Governor Phil Murphy highlighted the additional $20 million included in the Fiscal Year 2024 budget to boost the New Jersey Economic Development Authority’s (NJEDA) New Jersey Manufacturing Voucher Program (MVP) – bringing the program’s total funding to $53 million. MVP, developed in 2022 as a pilot program, provides manufacturers vouchers to purchase equipment they need to improve their operations and upgrade their businesses. Since the program’s launch, the NJEDA has awarded 189 vouchers to manufacturers across New Jersey totaling $23.4 million and continues to process and approve applications daily.

N.J. Universities Place Amazingly Well in Latest U.S. News Survey

Record 6 schools make Top 100, including Rutgers-New Brunswick at record-high No. 40 (yes, Princeton was No. 1 again)

New Jersey’s institutions of higher education are making their move in the national rankings. A record six schools placed in the Top 100 of the U.S. News & World Report Best Colleges ranking of national universities, which was released Monday and long has been considered the gold standard of college rankings. Four additional schools were at No. 163 or better.

NJIT Rises to No. 86 Among National Universities in U.S. News Rankings – Also in Top 100 for Social Mobility, Best Value, Public Schools

New Jersey Institute of Technology’s rise in national rankings continues, with U.S. News & World Report placing NJIT No. 86 among national universities for 2024 — a jump of 11 rungs from 2023. The latest ranking is indicative of a consistent trend over the past four years — as NJIT climbed from 118 to 103 to 97 and now 86 — and supports the findings of other publications that rate U.S. universities, including Money and The Wall Street Journal, which placed NJIT as the second-highest ranked public institution nationally.

All 3 Rutgers Campuses Place in Top 100 Nationally in U.S. News Rankings

Many have stumbled when trying to describe Rutgers University’s campuses in New Brunswick, Camden and Newark: Are they three schools or one? Now, there’s an easier way: They are all Top 100 universities in the country. All three campuses made huge jumps in the influential U.S. News & World Report Best Colleges ranking. Rutgers-Newark came in at a tie for No. 82 overall (a jump of 33 spots from a year ago). Rutgers-Camden came in at a tie for No. 98 (a jump of 29 spots). Rutgers President Jonathan Holloway obviously was thrilled by the news.

ROI Influencers: People of Color 2023

ROI Influencers: People of Color list honors that spirit with recognition of some (of the many) people of color who have great impact in business, academia, nonprofit and governmental sectors. Congratulations to these BioNJ Members for being honored honored Guillermo Artiles, McCarter & English; Alexis Bailey, NJBIA; William Best, PNC Bank; Michelle Bodden, NJEDA; Vanessa Broadhurst, Johnson & Johnson; Joe Cardona, Rowan University; Will Carroll, Hackensack Meridian Health; Francine Conway, Rutgers University; Tai Cooper, NJEDA; Jose Cruz, JLL; Nariman Farvardin, Stevens Institute of Technology; Donald Ferrell, Hackensack University Medical Center; Althea D. Ford, NJBIA; Angela Garretson, NJ Institute of Technology; Barbara George Johnson, Kean University; Jonathan Holloway, Rutgers University; Ali Houshmand, Ph.D., Rowan University; Pavita Howe, BioNJ; Dan Jennings, NJEDA; Natayla Johnson, Johnson & Johnson; Robert Johnson, Rutgers NJ Medical School; Kellie LeDet, Kean University; Lei Lei, Rutgers Business School; Teik Lim, NJ Institute of Technology; Jose Linares, McCarter & English; Jose Lozano, Hackensack Meridian Health; Reshema Kemps-Polanco, Novartis; Liz Paskas, Hackensack Meridian Health; Rodney Priestly, Princeton University; Lamont Repollet, Kean University; Lisa Tank, Hackensack University Medical Center; Jackie Taylor, EY.

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