Pill Penalty Hurts Us All — Including Rare Disease Families

The “Inflation Reduction Act” continues to do more harm than good — from its government-dictated price controls to raiding Medicare for green energy subsidies to crushing innovation in developing new cures for cancer and other diseases. The falsely named IRA included anti-competitive provisions that make it harder for patients to get the medicines they need. The IRA gives small molecule drugs, like oral medications, nine years of protection from price controls, while biologics get 13 years. This disparity is called the “pill penalty.” As Jim Glassman wrote in The Hill last year, “For no good reason, the act discriminates against a class of drugs that are easier to take, don’t require trips to the doctor or hospital, and provide benefits to disadvantaged patients. You’re certainly familiar with this kind of drug. It’s called a pill.”

Rotten to the Core: Inflation Reduction Act Undermines Medicare Part D, Pill Penalty Drug Costs Increase Up to 76%

As a result of a flawed Inflation Reduction Act (IRA), CMS is authorized to set a maximum fair price (MFP) that takes effect as early as nine years post-approval for certain small molecule drugs with high Medicare expenditures. This price-setting timeline is four years earlier than when certain biologics can be subject to MFP. This difference is referred to as the small molecule “pill penalty”. The pill penalty has far-reaching effects; if the IRA had established equivalent timelines for price-setting small molecule drugs and biologics at the same timeframe —13 years — half of the drugs chosen for price controls in 2026 would not have been selected. Policy changes are needed to grant small molecule drugs the same timelines as biologics, allowing beneficiaries to maintain more affordable, consistent access to necessary treatments for longer. 

The Inflation Reduction Act and Implications for Intellectual Property Incentives to Develop Drugs for Select Populations

The pharmaceutical industry has been an engine for innovation for decades, developing and commercializing thousands of novel, lifesaving drugs and also vaccines. The industry requires significant at-risk investment in research and development (R&D) and has flourished in an environment underscored by strong intellectual property (IP) incentives set forth by Congress through a series of laws over the past 4 decades. The continued development of new medicines by the pharmaceutical industry has yielded enormous benefits to patients and society, including extending life expectancy, introducing cures, diminishing the impact of many crippling diseases, reducing pain and suffering, decreasing hospitalizations, employing millions of people and boosting the global economy. As a result of IP incentives and other factors, the United States (US) is not only the largest pharmaceutical market in the world but also is the most productive.

The Inflation Reduction Act Is Negotiating the United States Out of Drug Innovation

The IRA’s unnecessary and counterproductive distinction between small- and large-molecule drugs disproportionately discourages small-molecule drug innovation, as drug developers and investors become incentivized to shift resources toward potentially more-profitable biologics. Ultimately, this will come at a cost—not just for Medicare patients, but all citizens—in the form of fewer drug choices and reduced health care outcomes. Access to both large- and small-molecule medicines is what makes treatments for a plethora of conditions so effective, as diseases including cancer and neurodegenerative disorders can be targeted based on the most-recent scientific findings. Maintaining access to the most-innovative treatments is essential to ensuring that patients are given the best chance at combating their illnesses.

The Impact of the Small Molecule “Pill Penalty” Two Years Into the IRA

Research firm Vital Transformation is preparing a new analysis about the impacts of the Inflation Reduction Act on biopharma investment, research and development in the two years since the law’s passage. Early returns show significant harms to innovation and patients’ treatment options, particularly because of the IRA’s small molecule “pill penalty.” The IRA set small molecules on a shorter timeline than biologics (9 vs. 13 years) before they can be subjected to government price setting. Experts have warned this will have many negative consequences by disincentivizing the development of small molecules. This is a particular concern given the unique benefits of small molecules, including their ease of access and their ability to reach therapeutic targets inside cells and inside the brain.

The Time is Now: Addressing Health Inequities in the Workforce

As a major provider of health insurance for working-age Americans, employers can play a significant role in improving the health equity of their employees and family members. In this commentary, we describe how different stakeholders, including employers, their employees, clinicians, and health systems and health plans, each contribute to the observed inequities. Other systems-level factors, including racism, implicit bias, medical mistrust, health literacy limitations and health care access and affordability concerns have been also shown to contribute to inequitable outcomes. Opportunities exist for employers to improve health equity among their benefits-enrolled employees and family members using data-driven approaches to ensure that benefits are more equitable in scope, access and affordability.

Lawmakers Need to Act to Fuel the Engine Driving Research and Improved Outcomes for Cancer Patients

As featured in ROI-NJ

New Jersey has long served as the breeding ground for critical medical innovation in cancer care. The accomplishments driven by our state, its institutions and its residents have made a profound global impact. From local scientists developing innovative strategies to treat prostate cancer, to researchers leading cutting-edge clinical trials for challenging cancers like metastatic brain and HPV-related cancers, our contributions are shaping the future of medicine. New Jersey’s ongoing contributions to the cancer community are exemplified by the vital work being done by the National Cancer Institute (NCI)-designated Cancer Center at Rutgers and the New Jersey Commission on Cancer Research. Unfortunately, despite its leadership, New Jersey continues to face significant gaps in access to care across the cancer continuum, particularly in the availability of biomarker testing for its residents.

NJ Business Groups Dig Into Deficit, Warehouse Tax from $58B Budget

As featured in NJBIZ

The reaction continues to flow in from around New Jersey, following Gov. Phil Murphy’s $58 billion Fiscal Year 2026 budget proposal. BioNJ said it supports ensuring that New Jersey remains a “bedrock of innovation.”...“BioNJ looks forward to continuing to work with the Governor’s office and the Legislature to accomplish our shared goals of attracting even more world class life sciences companies to New Jersey,” the life sciences trade association said in a statement. “To ensure that New Jersey remains nationally competitive, offering the types of credits and incentives outlined by the Governor can effectively attract companies from across the globe."

Interview With BioNJ President and CEO Debbie Hart

As featured in Global Business Reports

New Jersey has firmly established itself as a powerhouse in the life sciences industry, fueled by substantial investments from both the state government and the private sector. With world-renowned universities and research institutions producing a highly skilled workforce, the State offers an unmatched ecosystem for innovation and growth. Boasting one of the world’s most skilled workforces in biopharmaceutical manufacturing, R&D, medical devices and cell and gene therapy, New Jersey is home to a thriving and diverse life sciences sector. The State’s investment in ResearchWithNJ.com connects companies with cutting-edge research at New Jersey universities, fostering partnerships and driving innovation to accelerate scientific and technological progress. The creation of cutting-edge innovation hubs, such as HELIX, NEST, SciTech Scity and ON3, underscore the State's unwavering commitment to advancing the life sciences. These strategic investments are building a strong foundation for continued success and positioning New Jersey as a hub of scientific and technological breakthroughs.

EPKINLY® (epcoritamab) Approved by Japan Ministry of Health, Labour and Welfare for Additional Indication as a Treatment for Relapsed or Refractory Follicular Lymphoma

Princeton-based BioNJ Member Genmab announced that the Japan Ministry of Health, Labour and Welfare has approved EPKINLY® (epcoritamab) for the treatment of patients with relapsed or refractory (R/R) follicular lymphoma (FL; Grades 1 to 3A) who have received two or more prior lines of therapy. With this additional indication, EPKINLY is now the first and only T-cell engaging bispecific antibody administered subcutaneously to be approved in Japan to treat both R/R FL and R/R large B-cell lymphomas, including diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma and primary mediastinal large B-cell lymphoma, after two or more prior lines of therapy. FL is typically an indolent (or slow growing) form of non-Hodgkin’s lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL, accounting for 20-30 percent of all cases.

Insmed Announces that FDA Does Not Currently Plan to Hold Advisory Committee Meeting to Discuss New Drug Application for Brensocatib in Patients With Bronchiectasis

Bridgewater-based BioNJ Member Insmed Incorporated announced that the U.S. Food and Drug Administration (FDA) has informed the Company that it does not currently plan to hold an advisory committee meeting to discuss the New Drug Application (NDA) for brensocatib for patients with non-cystic fibrosis bronchiectasis. The FDA provided this update in its Day 74 communication to Insmed. "We are very pleased with our ongoing communications with the FDA about the NDA for brensocatib," said Martina Flammer, M.D., MBA, Chief Medical Officer of Insmed. "We are committed to working closely with the agency to successfully complete the review and potentially bring forward this much-needed treatment for patients with bronchiectasis." The FDA had previously granted Priority Review to Insmed's NDA for brensocatib and set a target action date of August 12, 2025, under the Prescription Drug User Fee Act (PDUFA). 

TEVIMBRA Approved in U.S. for First-line Treatment of Advanced Esophageal Squamous Cell Carcinoma in Combination With Chemotherapy

Hopewell-based BioNJ Member BeiGene, Ltd., a global oncology company that intends to change its name to BeOne Medicines Ltd., announced the U.S. Food and Drug Administration (FDA) has approved TEVIMBRA® (tislelizumab-jsgr), in combination with platinum-containing chemotherapy, for the first-line treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1 (≥1). The additional indication is based on results from BeiGene’s RATIONALE-306 (NCT03783442), a randomized, placebo-controlled, double-blind, global Phase 3 study to evaluate the efficacy and safety of TEVIMBRA in combination with platinum-containing chemotherapy as a first-line treatment in adult patients (n=649) with unresectable, locally advanced recurrent or metastatic ESCC. The study met its primary endpoint and demonstrated a statistically significant improvement in overall survival (OS) for adult patients randomized to TEVIMBRA in combination with chemotherapy compared to placebo in combination with chemotherapy.

Tris Pharma Announces Positive Results from ALLEVIATE-2 Phase 3 Pivotal Trial for Cebranopadol, an Investigational First-in-Class Oral Dual-NMR Agonist, for the Treatment of Moderate-to-Severe Acute Pain

Monmouth Junction-based BioNJ Member Tris Pharma, Inc. announced positive topline results from its ALLEVIATE-2 Phase 3 pivotal clinical trial evaluating cebranopadol, an investigational therapy, for the treatment of moderate-to-severe acute pain in patients following bunionectomy surgery. The company also detailed additional positive data from the ALLEVIATE-1 Phase 3 pivotal clinical trial in patients following abdominoplasty surgery from which topline data were recently announced. These results add to the growing body of data underscoring the promising efficacy and safety profile of cebranopadol, a first-in-class pain therapy involving dual-nociceptin/orphanin FQ peptide (NOP) receptor and µ-opioid peptide (MOP) receptor (dual-NMR) agonism. This dual-NMR agonist has the potential to deliver significant pain relief comparable to opioids with minimized risk of significant side effects, dependence and addiction by leveraging the body’s pain biology modulation processes, synergizing the analgesic and safety characteristics of the NOP receptor with the analgesic advantages of the MOP receptor.

Celldex Therapeutics Presents Positive Results from Barzolvolimab Phase 2 Studies in Patients With Chronic Urticaria Demonstrating Improved Disease Control and Quality of Life at AAAAI 2025

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced positive data on measurements of disease control and quality of life from the company’s Phase 2 barzolvolimab studies in patients with chronic urticaria. Barzolvolimab is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity, which is required for mast cell function and survival. In a Phase 2 chronic spontaneous urticaria (CSU) study (52 week analysis) and a Phase 2 chronic inducible urticaria (CIndU) study (12 week analysis), barzolvolimab demonstrated rapid and sustained improvement in urticaria control and greatly reduced disease impact on quality of life, as measured by the Urticaria Control Test (UCT) and Dermatology Life Quality Index (DLQI). 

Boehringer Ingelheim and Partners Start Clinical Development of a First-in-Class, Inhaled Gene Therapy for People With Cystic Fibrosis

BioNJ Member Boehringer Ingelheim, with a site in North Brunswick,IP Group, the UK Respiratory Gene Therapy Consortium (GTC) and OXB announce the start of LENTICLAIR^TM 1, a Phase I/II trial of BI 3720931, a novel, first-in-class, gene therapy aiming to improve disease outcomes in people with cystic fibrosis (CF), regardless of gene mutations that are causing the disease. The trial specifically focuses on adults with CF who genetically cannot benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators. BI 3720931 is a first-in-class, inhaled lentiviral vector-based gene therapy that could potentially address unmet needs by inserting a functional copy of the CFTR gene in the DNA of airway epithelial cells. The aim is to improve lung function and reduce exacerbations for people with CF irrespective of their mutations, including those who genetically cannot benefit from CFTR modulators.

Chugai Obtains Regulatory Approval for Tecentriq for the Additional Indication of Alveolar Soft Part Sarcoma, an Ultra-rare Disease

BioNJ Member Chugai Pharmaceutical, with a site in Berkeley Heights, announced that it obtained regulatory approval today from the Ministry of Health, Labour and Welfare for the anti-cancer agent/humanized anti-PD-L1 monoclonal antibody Tecentriq® Intravenous Infusion [generic name: atezolizumab (genetical recombination)] for an additional indication of unresectable alveolar soft part sarcoma. Tecentriq is the first immune checkpoint inhibitor in Japan for the treatment of this disease. This approval is based on the results from a Phase II ALBERT study initiated by investigators in Japan including National Cancer Center Hospital and an overseas Phase II clinical study conducted by the National Cancer Institute (NCI), which evaluated the efficacy and safety of Tecentriq in patients with unresectable alveolar soft part sarcoma.

Y-mAbs Announces Publication of Phase 2 Interim Results in Nature Communications

BioNJ Member Y-mAbs Therapeutics, Inc., with a site in Nutley, announced the publication of interim data from a Phase 2 clinical trial evaluating naxitamab with granulocyte-macrophage colony-stimulating factor (GM-CSF) in patients with relapsed/refractory high-risk neuroblastoma in the journal Nature Communications. The article, titled “The anti-GD2 monoclonal antibody naxitamab plus GM-CSF for relapsed or refractory high-risk neuroblastoma: a phase 2 clinical trial,” details the results of a single-arm, global Phase 2 trial (Trial 201, NCT03363373) of patients with relapsed/refractory high-risk neuroblastoma and residual disease in the bone/bone marrow who received naxitamab on days 1, 3 and 5 (3 mg/kg/day) with GM-CSF (days -4 to 5) every 4 weeks, until a complete response (CR) or partial response (PR) was achieved, followed by 5 additional cycles every 4 weeks. Overall, naxitamab demonstrated statistically significant efficacy with a manageable safety profile.

Lexicon Pharmaceuticals Announces Topline Results from Phase 2b PROGRESS Study Evaluating Pilavapadin (LX9211) in Adults With Diabetic Peripheral Neuropathic Pain

BioNJ Member Lexicon Pharmaceuticals, with a site in Bridgewater, announced topline results from the PROGRESS Phase 2b study evaluating pilavapadin (LX9211), an oral, non-opioid investigational adaptor-associated kinase 1 (AAK1) inhibitor in adult patients with moderate to severe diabetic peripheral neuropathic pain (DPNP). Lexicon’s objective for the PROGRESS study was to identify a dose exhibiting meaningful pain reduction and improved tolerability compared to that observed in the previous Phase 2 RELIEF-DPN-1 study. All objectives for the PROGRESS study were achieved with respect to the 10 mg dose, which Lexicon has identified as the appropriate dose to advance into Phase 3 development based both on these PROGRESS results and the previous results from RELIEF DPN-1. In RELIEF-DPN-1, dosing regimens utilized a ten-fold loading dose on Day 1 and the 100 mg/10 mg dose arm, but not the 200 mg/20 mg dose arm, achieved significantly reduced pain scores as compared to both baseline and placebo, with the loading dose affecting tolerability in both treatment arms.

Bristol Myers Squibb Presents Late-Breaking Data from Phase 3 POETYK PsA-2 Trial Demonstrating Superiority of Sotyktu (deucravacitinib) Compared With Placebo in Adults With Psoriatic Arthritis

Princeton-based BioNJ Member Bristol Myers Squibb announced positive data from the pivotal Phase 3 POETYK PsA-2 trial (IM011-055) evaluating the efficacy and safety of Sotyktu (deucravacitinib) in adults with active psoriatic arthritis (PsA). The POETYK PsA-2 trial met its primary endpoint, with a significantly greater proportion of Sotyktu -treated patients achieving ACR20 response (at least a 20 percent improvement in signs and symptoms of disease) compared with placebo at Week 16 (54.2% versus 39.4%, respectively; p=0.0002). The overall safety profile of Sotyktu through 16 weeks of treatment was consistent with that established in a Phase 2 PsA clinical trial and Phase 3 moderate-to-severe plaque psoriasis clinical trials. Additionally, treatment with Sotyktu met important secondary endpoints across PsA disease activity at Week 16, demonstrating improvement across clinical signs and symptoms, extra-articular manifestations and patient-reported outcomes. 

Bristol Myers Squibb Receives European Commission Approval for Opdivo® (nivolumab) Plus Yervoy® (ipilimumab) for the First-Line Treatment of Adult Patients With Unresectable or Advanced Hepatocellular Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Commission (EC) has approved Opdivo^® (nivolumab) plus Yervoy^® (ipilimumab) for the first-line treatment of adult patients with unresectable or advanced hepatocellular carcinoma (HCC). The decision is based on results from the CheckMate -9DW study. Results showed that dual immunotherapy treatment with Opdivo plus Yervoy led to a statistically significant and clinically meaningful improvement in overall survival (OS), the clinical trial’s primary endpoint. In the trial, 85% of patients in the comparator arm were treated with lenvatinib and 15% were treated with sorafenib. The median OS was 23.7 months (95% CI: 18.8–29.4) for Opdivo plus Yervoy compared to 20.6 months (95% CI: 17.5–22.5) with the investigator’s choice of lenvatinib or sorafenib (HR: 0.79 (0.65–0.96); p=0.018). The OS benefit was observed across clinically relevant patient subgroups. The trial also showed an overall response rate (ORR) of 36.1% (95% CI: 31-41.5) compared to 13.2% (95% CI: 9.8-17.3; P<0.0001) of patients treated with lenvatinib or sorafenib and a deeper response with Opdivo plus Yervoy .

U.S. Food and Drug Administration Accepts Bristol Myers Squibb’s Supplemental Biologics License Application for Opdivo® Plus Yervoy® for Patients With Unresectable or Metastatic Microsatellite Instability-High or Mismatch Repair Deficient

Princeton-based BioNJ Member Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental biologics license application (sBLA) for Opdivo® (nivolumab) plus Yervoy® (ipilimumab) as a potential first-line treatment option for adult and pediatric patients (12 years and older) with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The FDA granted the application Breakthrough Therapy Designation and Priority Review status and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 23, 2025. The application was based on results from the three-arm Phase 3 CheckMate -8HW study demonstrating that Opdivo plus Yervoy met the dual primary endpoints of progression free survival (PFS) compared to investigator’s choice of chemotherapy in the first-line setting and compared to Opdivo monotherapy across all lines of therapy, as assessed by Blinded Independent Central Review.

Teva and Medincell Announce FDA Acceptance of Supplemental New Drug Application for UZEDY(R) (risperidone) Extended-Release Injectable Suspension as a Treatment for Patients With Bipolar I Disorder

Parsippany-based BioNJ Member Teva Pharmaceuticals and Medincell announced that the supplemental New Drug Application (sNDA) for UZEDY extended-release injectable suspension for the maintenance treatment of BP-I in adults has been accepted for filing by the U.S. Food and Drug Administration (FDA). The sNDA is based on leveraging the existing clinical data for UZEDY coupled with the Agency’s previous findings of safety and efficacy of past risperidone formulations approved for the treatment of BP-I. Teva will lead the regulatory process and be responsible for potential commercialization of UZEDY for BP-I, with Medincell eligible for royalties on net sales. UZEDY was approved in the U.S. for the treatment of schizophrenia in adults in 2023. The efficacy and long-term safety and tolerability of UZEDY for the treatment of schizophrenia have been evaluated in two Phase 3 pivotal studies.

Teva and Sanofi Present New Positive Phase 2b Study Results at ECCO 2025 Reinforcing Best-in-Class Potential of Duvakitug (Anti-TL1A) in Ulcerative Colitis and Crohn’s Disease

Parsippany-based BioNJ Member Teva Pharmaceuticals and Bridgewater-based BioNJ Member Sanofi presented new, detailed results from the RELIEVE UCCD Phase 2b study of duvakitug (TEV’574/SAR447189), a human IgG1-λ2 monoclonal antibody targeting TL1A, for the treatment of moderate-to-severe ulcerative colitis (UC) and Crohn’s disease (CD), the two most common forms of inflammatory bowel disease (IBD). In the UC cohort of the RELIEVE UCCD study, 36% (450 mg dose) and 48% (900 mg dose) of patients treated with duvakitug achieved the primary endpoint of clinical remission (mMS) at week 14 compared to 20% treated with placebo; placebo-adjusted rates were 16% (450 mg) and 27% (900 mg) (p=0.050 and 0.003, respectively).. In addition, higher clinical remission rates were observed for both doses of duvakitug versus placebo in both advanced therapy (AT)-experienced and AT-naïve subgroups of patients.

Dupixent Late-Breaking Positive Pivotal Data in Bullous Pemphigoid Presented at AAD

Bridgewater-based BioNJ Member Sanofi announced positive results from the pivotal ADEPT Phase 2/3 study evaluating the investigational use of Dupixent (dupilumab) in adults with moderate-to-severe bullous pemphigoid (BP). BP is a chronic, debilitating, and relapsing skin disease with underlying type 2 inflammation and characterized by intense itch and blisters, reddening of the skin and painful lesions. The ADEPT study met all primary and key secondary endpoints, enrolling 106 adults with moderate-to-severe BP who were randomized to receive Dupixent 300 mg (n=53) every two weeks after an initial loading dose or placebo (n=53) added to standard-of-care oral corticosteroids (OCS). During treatment, all patients underwent a protocol-defined OCS tapering regimen if control of disease activity was maintained. Sustained disease remission was defined as complete clinical remission with completion of OCS taper by week 16 without relapse and no rescue therapy use during the 36-week treatment period.

Sarclisa Approved in Japan for Patients With Newly Diagnosed Multiple Myeloma

The Ministry of Health, Labour and Welfare (MHLW) in Japan has approved Bridgewater-based BioNJ Member Sanofi’s Sarclisa, in combination with bortezomib, lenalidomide, and dexamethasone (VRd), for the treatment of adult patients with newly diagnosed multiple myeloma (NDMM) based on data from the IMROZ phase 3 study. In Japan, Sarclisa was launched in August 2020 and has been approved for four different treatment regimens (in combination with pomalidomide and dexamethasone, as monotherapy, in combination with carfilzomib and dexamethasone, or in combination with dexamethasone for the treatment of patients with relapsed or refractory multiple myeloma). In addition, Sarclisa has front-line approvals in the EU and the US. In the Asia Pacific region, Sarclisa combination regimens were also recently approved by the National Medical Products Administration in China, specifically Sarclisa-VRd in NDMM patients who are not eligible for autologous stem cell transplant.

New Analysis of Organon’s VTAMA® (tapinarof) cream, 1% Phase 3 Data Shows Atopic Dermatitis Disease Activity Remained Low After Treatment-Free Interval in Adults and Children 2 Years of Age and Older

Jersey City-based BioNJ Member Organon announced results from an analysis of the Phase 3 ADORING 3 open-label, long-term extension study evaluating VTAMA® (tapinarof) cream, 1% once daily in adults and children 2 years of age and older with atopic dermatitis (AD), also known as eczema. The findings demonstrate that AD disease activity remained mild in patients who had achieved treatment success and subsequently entered a treatment-free interval lasting on average 80 days.  The new results demonstrate that, at the end of the first treatment-free interval, which lasted an average of 79.8 consecutive days, 84% of patients had a vIGA-AD score of 2 (mild). Itch, which was assessed with the patient reported Peak Pruritus Numerical Rating Scale (PP-NRS) score ranging from 0 to 10, remained low with a mean weekly PP-NRS score of 2.9. The mean Eczema Area and Severity Index (EASI) score was 3.4, indicating mild disease.

Lilly's Baricitinib Delivered High Rates of Hair Regrowth for Adolescents With Severe Alopecia Areata in Phase 3 BRAVE-AA-PEDS Study

BioNJ Member Eli Lilly and Company, with a site in Branchburg, and Incyte (NASDAQ: INCY) found adolescent patients (ages 12 to under 18) with severe alopecia areata (AA) treated with once-daily, oral baricitinib 4 mg and 2 mg saw clinically meaningful improvements in hair regrowth on the scalp, eyebrows and eyelashes at Week 36. AA is an immune system condition that causes patchy hair loss on the scalp, face and sometimes on other areas of the body that can progress over time. Approximately 40% of patients with AA experience first onset by 20 years of age. In the BRAVE-AA-PEDS study, 257 patients were randomized to receive once-daily baricitinib 4 mg, baricitinib 2 mg or placebo. The primary endpoint of this study was a Severity of Alopecia Tool (SALT) score ≤20 (i.e., 80% or more scalp hair coverage) at Week 36.

Lilly's EBGLYSS® (lebrikizumab-lbkz) Single Monthly Maintenance Injection Achieved Completely Clear Skin at Three Years in Half of Patients With Moderate-to-Severe Atopic Dermatitis

With a site in Branchburg, BioNJ Member Eli Lilly and Company's EBGLYSS achieved deep and sustained response for patients with moderate-to-severe atopic dermatitis (eczema) at three years. EBGLYSS is an interleukin-13 (IL-13) inhibitor that selectively blocks IL-13 signaling with high binding affinity. The cytokine IL-13 is a primary cytokine in atopic dermatitis, driving the type-2 inflammatory cycle in the skin, leading to skin barrier dysfunction, itch, skin thickening and infection. Three-year depth of response data being presented are part of ADjoin, the long-term extension study of the EBGLYSS trials, and include participants who responded to EBGLYSS treatment at Week 16 from ADvocate 1 and ADvocate 2 monotherapy trials. Patients received a maintenance dose of 250 mg EBGLYSS either every two weeks or once monthly (every four weeks) and were assessed for depth of response using IGA 0, EASI 90 and EASI 100. 

Lilly's Jaypirca (pirtobrutinib) Recommended by CHMP for Approval in the European Union for Adults With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) Previously Treated With a BTK Inhibitor

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Jaypirca (pirtobrutinib), a non-covalent (reversible) Bruton's tyrosine kinase (BTK) inhibitor, for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) who have been previously treated with a BTK inhibitor. Following this positive opinion, the application for the use of Jaypirca in patients with relapsed or refractory CLL who have been previously treated with a BTK inhibitor is now referred to the European Commission for final action. Jaypirca has also previously received a conditional marketing authorization by the EMA for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a BTK inhibitor.

Lilly Launches Additional Zepbound Vial Doses and Offers New Savings for Self-Pay Patients

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced the launch of 7.5 mg and 10 mg Zepbound (tirzepatide) single-dose vials, available for $499 with the new Zepbound Self Pay Journey Program. Lilly also reduced the price of the 2.5 mg and 5 mg vials. These new offerings are available exclusively through LillyDirect Self Pay Pharmacy Solutions, which enables a transparent price by removing third-party supply chain entities and allowing patients to access savings directly outside of insurance. Zepbound is also available in 2.5 mg, 5 mg, 7.5 mg, 10 mg, 12.5 mg, or 15 mg per 0.5 mL doses in a single-dose pen (autoinjector). The recommended maintenance dosages are 5 mg, 10 mg or 15 mg injected subcutaneously once weekly.

Novo Nordisk A/S: CagriSema Demonstrates Superior Weight Loss in Adults With Obesity or Overweight and Type 2 Diabetes in the REDEFINE 2 Trial

Plainsboro-based BioNJ Member Novo Nordisk announced headline results from REDEFINE 2, a Phase 3 trial in the global REDEFINE program. REDEFINE 2 is a 68-week efficacy and safety trial investigating once-weekly subcutaneous CagriSema (a fixed dose combination of cagrilintide 2.4 mg and semaglutide 2.4 mg) compared to placebo. The trial included 1,206 randomized people with obesity or overweight and type 2 diabetes and a mean baseline body weight of 102 kg. The trial achieved its primary endpoint by demonstrating a statistically significant and superior weight loss at week 68 with CagriSema versus placebo. The REDEFINE 2 trial was based on a flexible protocol, allowing patients to modify their dosing throughout the trial. After 68 weeks, 61.9% of patients treated with CagriSema were on the highest dose.

Odronextamab BLA Accepted for FDA Review for the Treatment of Relapsed/Refractory Follicular Lymphoma

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of the Biologics License Application (BLA) for odronextamab in relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. The target action date for the FDA decision is July 30, 2025. Acceptance of the BLA resubmission follows the achievement of an FDA-mandated enrollment target for the Phase 3 confirmatory trial in R/R FL ( OLYMPIA -1). This was the sole approvability issue identified by the FDA in the complete response letter associated with the previous submission. The BLA resubmission is supported by data from the Phase 1 and pivotal Phase 2 trials (ELM-1 and ELM-2), which demonstrated an overall response rate of 80% (n=103), with 74% (n=95) achieving a complete response. 

Latest DB-OTO Results Demonstrate Clinically Meaningful Hearing Improvements in Nearly All Children With Profound Genetic Hearing Loss in CHORD Trial

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced updated data for the investigational gene therapy DB-OTO from the Phase 1/2 CHORD trial in 12 children who have profound genetic hearing loss due to variants of the otoferlin (OTOF) gene. These include 72-week results showing speech and development progress in the first child dosed at 10 months of age, as well as initial results in 11 children (aged 10 months to 16 years old) – three of whom received DB-OTO bilaterally (in both ears). In the trial, 12 participants have received DB-OTO to date – of whom nine were administered an intracochlear injection in one ear and three received it bilaterally. The surgical procedure to administer DB-OTO leverages an approach similar to cochlear implantation, which enables use in young infants.

First Clinical Data for Gilead's Investigational Once-Yearly Lenacapavir for HIV Prevention Presented at CROI 2025 and Published in The Lancet

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, presented the first data from its ongoing Phase 1 study investigating two novel, once-yearly formulations of lenacapavir, the company’s injectable HIV-1 capsid inhibitor, for the investigational use of HIV prevention as pre-exposure prophylaxis (PrEP). Data will support the future development of once-yearly lenacapavir for PrEP, for which Gilead plans to launch a Phase 3 study in the second half of 2025. The Phase 1 study data showed that the two different formulations of once-yearly lenacapavir administered via intramuscular injection achieved and maintained plasma concentrations exceeding those associated with HIV prevention efficacy observed in the Phase 3 PURPOSE 1 trial (NCT04994509) and PURPOSE 2 trial (NCT04925752). The previously reported PURPOSE 1 and PURPOSE 2 data showed that twice-yearly subcutaneous lenacapavir demonstrated superiority at reducing HIV infections when compared to background HIV incidence (bHIV) and once-daily oral Truvada® (emtricitabine 200mg and tenofovir disoproxil fumarate 300mg; F/TDF) in a broad and geographically diverse range of people.

European Medicines Agency Validates Gilead’s Marketing Authorization Application and EU-Medicines for All Application for Twice-Yearly Lenacapavir for HIV Prevention

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced that the European Medicines Agency (EMA) has validated for parallel accelerated review the company’s Marketing Authorization Application (MAA) and EU-Medicines for all (EU-M4all) application for lenacapavir—the company’s twice-yearly injectable HIV-1 capsid inhibitor—for the prevention of HIV as pre-exposure prophylaxis (PrEP). Based on the assessment of EMA’s Committee for Medicinal Products for Human Use (CHMP) that twice-yearly lenacapavir for prevention is a product of major interest for public health and therapeutic innovation, both applications will be assessed under an accelerated assessment review timeline. The EU MAA will be reviewed by the CHMP under the centralized licensing procedure for all 27 Member States of the European Union, as well as Norway, Iceland and Liechtenstein.

Kyowa Kirin and Amgen Provide Top-Line Results from Rocatinlimab Phase 3 Ignite Study in Adults With Moderate to Severe Atopic Dermatitis

BioNJ Members Kyowa Kirin Co., Ltd., with a site in Princeton, and Basking Ridge-based Amgen announced new results from the ongoing ROCKET Phase 3 clinical trial program evaluating rocatinlimab, an investigational T-cell rebalancing therapy targeting the OX40 receptor, in moderate to severe atopic dermatitis (AD). The IGNITE study, which evaluated two dose strengths of rocatinlimab, met its co-primary endpoints and all key secondary endpoints, achieving statistical significance for both rocatinlimab dose strengths versus placebo. IGNITE was a 24-week, randomized, placebo-controlled, double-blind study to assess the efficacy, safety and tolerability of rocatinlimab monotherapy every 4 weeks in 769 adults with moderate to severe AD, including patients previously treated with a biologic or systemic Janus kinase (JAK) inhibitor medication. 

Kyowa Kirin Announces Approval for Partial Change of Rituximab Biosimilar Received by Sandoz in Japan 

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, announced that BioNJ Member Sandoz KK, its strategic partner of the anti-CD20 monoclonal antibody Rituximab biosimilar which Kyowa Kirin markets in Japan, has received approval for the partial change of Rituximab biosimilar for the treatment of refractory nephrotic syndrome (frequently relapsing or steroid-dependent). This product is a biosimilar to the rituximab (genetic recombination) with a MOA (mechanism of action) of antibody-dependent cytotoxicity (ADCC), complement-dependent cytotoxicity (CDC) and B-cell depletion caused by apoptosis. Under the agreement with Sandoz announced on January 26, 2016, Kyowa Kirin is responsible for sales and marketing of the product in Japan. 

Sandoz Launches Biosimilar Pyzchiva® (ustekinumab-ttwe) in the U.S., Offering New Treatment for Around 12 Million Patients

Princeton-based BioNJ Member Sandoz announces the launch of Pyzchiva®* (ustekinumab-ttwe) in the US. From today, the medicine is commercially available to patients across the U.S. Developed by Samsung Bioepis Co., Ltd., and commercialized by Sandoz, Pyzchiva® has been approved by the US Food and Drug Administration (FDA) for the treatment of certain chronic inflammatory diseases, with the same indications as reference medicine Stelara®. Pyzchiva® is a key biosimilar value driver for Sandoz, contributing to the company’s overall growth strategy. The company ranks number one in biosimilars globally and across key markets in Europe. The commercial availability of Pyzchiva®, which builds on the U.S. launch of Hyrimoz® in July 2023, marks an important step in the Sandoz strategic ambition to become number one in biosimilars in the U.S.

Icotrokinra Meets Primary Endpoint of Clinical Response in Ulcerative Colitis Study and Shows Potential to Transform the Treatment Paradigm for Patients

New Brunswick-based BioNJ Member Johnson & Johnson announced positive topline results from ANTHEM-UC, a Phase 2b study of icotrokinra (JNJ-2113), the first investigational targeted oral peptide that selectively blocks the IL-23 receptor, in adults with moderately to severely active ulcerative colitis (UC). The study met its primary endpoint of clinical response in all icotrokinra dose groups evaluated and demonstrated clinically meaningful differences versus placebo in key secondary endpoints of clinical remission, symptomatic remission and endoscopic improvement at Week 12. In the ANTHEM-UC study (n=252), three doses of once daily icotrokinra were tested with all meeting the primary endpoint of clinical response at Week 12. A response rate of 63.5% for patients treated with the highest dose of icotrokinra was achieved at Week 12 versus 27% for placebo (p<0.001). 

Icotrokinra Results Show Potential to Set a New Standard of Treatment in Plaque Psoriasis

New Brunswick-based BioNJ Member Johnson & Johnson announced new icotrokinra (JNJ-2113) data from its comprehensive Phase 3 clinical program and the start of the first-ever head-to-head study in plaque psoriasis (PsO) seeking to demonstrate the superiority of an oral pill, icotrokinra, compared to an injectable biologic, ustekinumab. Icotrokinra is a first-in-class investigational targeted oral peptide that selectively blocks the IL-23 receptor and is being studied in adults and adolescents 12 years of age and older with moderate-to-severe plaque PsO. Data from the Phase 3 ICONIC-LEAD study show once daily icotrokinra demonstrated significant skin clearance and a favorable safety profile in adults and adolescents 12 years of age and older with moderate-to-severe plaque PsO.

TREMFYA® (guselkumab) Subcutaneous (SC) Induction Data Support Potential to be the First and Only in its Class to Offer the Option of Both Intravenous and SC Induction Therapy in Ulcerative Colitis

New Brunswick-based BioNJ Member Johnson & Johnson announced data from the Phase 3 ASTRO study of TREMFYA® (guselkumab) subcutaneous (SC) induction therapy in adults with moderately to severely active ulcerative colitis (UC) at the 20th Congress of the European Crohn’s and Colitis Organization (ECCO). Study findings through Week 12 showed statistically significant and clinically meaningful improvements compared to placebo across all clinical and endoscopic measures consistent with the U.S. Food and Drug Administration (FDA)-approved intravenous (IV) induction regimen in this population. In prespecified analyses of subpopulations defined by prior advanced therapy treatment status, TREMFYA® demonstrated statistically significant results across endpoints in both biologic and JAK inhibitor-naïve and biologic and JAK inhibitor-refractory patients.

Merck Announces Positive Data from Phase 3 Trials that Show the Investigational, Once-Daily, Oral, Two-Drug Regimen of Doravirine/Islatravir (DOR/ISL) Maintained HIV-1 Viral Suppression at Week 48

Rahway-based BioNJ Member Merck & Co. announced the presentation of positive results from two pivotal Phase 3 trials of the investigational, once-daily, oral, two-drug regimen of doravirine/islatravir [DOR/ISL (100mg/0.25mg)] in adults with HIV-1 infection that is virologically suppressed on bictegravir/emtricitabine/tenofovir alafenamidei [BIC/FTC/TAF (50mg/200mg/25mg)] in trial MK-8591A-052 ) or antiretroviral therapy [baseline antiretroviral therapy (bART)] in trial MK-8591A-051. In both trials, DOR/ISL met the primary efficacy success criterion for non-inferiority to comparator antiretroviral therapies and primary safety objectives at Week 48. Merck plans to begin submitting applications for marketing authorization to regulatory agencies by mid-2025. In the double-blind trial MK-8591A-052 (Abstract #204A), results for the primary endpoint (HIV-1 RNA ≥50 copies/mL) showed that 1.5% of participants who switched to DOR/ISL had a viral load of ≥50 copies/mL at Week 48, compared to 0.6% on BIC/FTC/TAF (treatment difference 0.9%, 95% CI -1.9, 2.9).

FDA Grants Priority Review to Merck’s Application for KEYTRUDA® (pembrolizumab) Plus Standard of Care as Perioperative Treatment for Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma

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® (pembrolizumab), Merck’s anti-PD-1 therapy, for the treatment of patients with resectable locally advanced head and neck squamous cell carcinoma (LA-HNSCC) as neoadjuvant treatment, then continued as adjuvant treatment in combination with standard of care radiotherapy with or without cisplatin and then as a single agent. The sBLA is based on data from the Phase 3 KEYNOTE-689 trial. Results from a pre-specified first interim analysis, which will be presented at an upcoming medical meeting, showed that in patients with resectable LA-HNSCC, the KEYTRUDA perioperative treatment regimen demonstrated a statistically significant and clinically meaningful improvement in event-free survival (EFS) compared to adjuvant radiotherapy (with or without cisplatin) alone.

Depemokimab Applications Accepted for Review by the U.S. FDA for Asthma With Type 2 Inflammation and for Chronic Rhinosinusitis With Nasal Polyps (CRSwNP)

Warren-based BioNJ Member GSK plc announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application for the use of depemokimab in two indications. The proposed indications are as add-on maintenance treatment of asthma in adult and pediatric patients aged 12 years and older with type 2 inflammation characterised by an eosinophilic phenotype on medium- to high-dose inhaled corticosteroids (ICS) plus another asthma controller and, as add-on maintenance treatment in adult patients with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP). The Prescription Drug User Fee Act (PDUFA) date is 16 December 2025. Depemokimab, a monoclonal antibody that targets IL-5, is the first ultra-long-acting biologic to be evaluated in Phase III trials and be accepted for regulatory review for use in these conditions. 

Depemokimab Delivers Clinically Meaningful and Statistically Significant Improvements for Patients With Chronic Rhinosinusitis With Nasal Polyps (CRSwNP)

Warren-based BioNJ Member GSK plc announced full results from the positive ANCHOR-1 and ANCHOR-2 Phase III clinical trials assessing the efficacy and safety of depemokimab versus placebo (both with standard of care [SOC]) in adults with CRSwNP. Depemokimab is an investigational, ultra-long-acting monoclonal antibody targeting interleukin-5 (IL-5), a key cytokine (protein) in type 2 inflammation that presents in up to 85% of people with CRSwNP.ANCHOR-1 (N=271) and ANCHOR-2 (N=257) met their co-primary endpoints, with twice-yearly administration of depemokimab showing clinically meaningful and statistically significant improvements in nasal polyp size and nasal obstruction, two key clinical measures of disease severity, versus placebo. ANCHOR-1 and ANCHOR-2 recruited a broad patient population with heterogenous symptom severity, reflective of real-world clinical practice. The treatment benefits were observed by the first assessment and sustained to week 52.

Eisai Enters into License Agreement for the Development and Distribution of Fibroblast Growth Factor (FGF) Receptor Selective Tyrosine Kinase Inhibitor Tasurgratinib in Greater China Region (Mainland China, Hong Kong, Macau, and Taiwan) With SciClone

Nutley-based BioNJ Member Eisai Co., Ltd. announced that the company has entered into a license agreement granting the exclusive development and distribution rights for the fibroblast growth factor (FGF) receptor selective tyrosine kinase inhibitor tasurgratinib succinate (generic name, development code: E7090, Japan brand name: TASFYGO®, “tasurgratinib”) in the Greater China region (mainland China, Hong Kong, Macau and Taiwan,“licensed regions”) to a subsidiary of SciClone Pharmaceuticals Limited. Eisai will maintain the rights to tasurgratinib worldwide outside of the licensed regions and continue to manufacture and market it in Japan, where it has been already launched. Tasurgratinib is an orally available novel tyrosine kinase inhibitor that demonstrates selective inhibitory activity against FGFR1, FGFR2 and FGFR3. Tasurgratinib was approved in Japan for the treatment of patients with unresectable biliary tract cancer with FGFR2 gene fusions or rearrangements that progressed after cancer chemotherapy.

AbbVie Receives Positive CHMP Opinion for Upadacitinib (RINVOQ®) for the Treatment of Adults With Giant Cell Arteritis

BioNJ Member AbbVie, with a site in Madison, announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of upadacitinib (RINVOQ®; 15 mg, once daily) for the treatment of adult patients with GCA. The final European Commission decision is expected in the first half of 2025. If approved, upadacitinib would be the first and only oral advanced therapy for adults living with GCA. The CHMP positive opinion is supported by data from the pivotal Phase 3 SELECT-GCA clinical trial evaluating the efficacy and safety of upadacitinib in adults 50 years and older with GCA. In the first study period, patients were randomized to receive upadacitinib 7.5 mg or 15 mg in combination with a 26-week corticosteroid taper regimen or placebo in combination with a 52-week corticosteroid taper regimen.

Evotec Announces Key Progress in Neuroscience Collaboration With Bristol Myers Squibb

Princeton-based Evotec SE announced another major accomplishment in its long-standing neuroscience collaboration with BioNJ Member Bristol Myers Squibb. The latest scientific achievement in the partnership has triggered a research payment of US$20 m to Evotec, enabling further development of a promising pre-clinical program in neurodegeneration. Since the initiation of their strategic neuroscience collaboration in December 2016, Evotec and Bristol Myers Squibb have been working together to identify disease-modifying treatments for a broad range of neurodegenerative diseases. While currently approved therapies primarily focus on symptom management, this partnership aims to develop transformative treatments that can slow or halt disease progression ― addressing a significant unmet medical need. The collaboration has already yielded strong results, including the in-licensing of EVT8683 (now BMS-986419) by Bristol Myers Squibb in September 2021.

ENHERTU® Demonstrated Statistically Significant and Clinically Meaningful Improvement in Overall Survival in Patients With HER2 Positive Metastatic Gastric Cancer at Interim Analysis of DESTINYGastric04 Phase 3 Trial

Basking Ridge-based Daiichi Sankyo announced positive topline results from the DESTINY-Gastric04 Phase 3 trial showed ENHERTU® (trastuzumab deruxtecan) demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of overall survival (OS) compared to ramucirumab and paclitaxel in patients with second-line HER2 positive (IHC 3+ or IHC 2+/ISH+) unresectable and/or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma. At a planned interim analysis, the Independent Data Monitoring Committee recommended unblinding the trial based on the superior efficacy of ENHERTU. ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC). ENHERTU is currently approved in more than 65 countries based on DESTINY-Gastric01, a randomized Phase 2 trial, and DESTINY-Gastric02 and DESTINY-Gastric06, two single-arm phase 2 trials. DESTINYGastric04 is the first phase 3 trial of ENHERTU in HER2 positive advanced gastric cancer.

ENHERTU® Recommended for Approval in the EU by CHMP for Patients With HER2 Low or HER2 Ultralow Metastatic Breast Cancer Following at Least One Endocrine Therapy

Basking Ridge-based Daiichi Sankyo and AstraZeneca’s ENHERTU® (trastuzumab deruxtecan) has been recommended for approval in the European Union (EU) as a monotherapy for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (IHC 0 with membrane staining) breast cancer who have received at least one endocrine therapy in the metastatic setting and who are not considered suitable for endocrine therapy as the next line of treatment. ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from the DESTINY-Breast06 Phase 3 trial.

Amneal’s BLA Submissions for Two Denosumab Biosimilars Accepted for Review by U.S. FDA

Bridgewater-based Amneal Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics Licensing Application (BLA) for two proposed denosumab biosimilars referencing Prolia® and XGEVA® . mAbxience is a Fresenius Kabi majority-owned group with partial ownership from Insud Pharma. Fresenius Kabi is an operating company of Fresenius. The FDA has assigned a target action date in the fourth quarter of 2025. Currently, Amneal commercializes three biosimilars in the U.S., with the two denosumab biosimilar candidates representing its next potential biosimilar launches. Additionally, three more biosimilars are in development, positioning Amneal to have a portfolio of six biosimilars across eight product presentations by 2027. Denosumab is a monoclonal antibody drug that inhibits bone reabsorption.

IMUNON Announces Translational Data from Phase 1/2 OVATION 2 Study of IMNN-001 in Advanced Ovarian Cancer

Lawrenceville-based IMUNON announced new translational data from ongoing analyses of results from the company’s Phase 2 OVATION 2 Study of IMNN-001, its investigational interleukin-12 (IL-12) immunotherapy based on the company’s proprietary TheraPlas® technology, for the treatment of newly diagnosed advanced ovarian cancer. Results demonstrated a 20% increase in IL-12 levels in women treated with IMNN-001 (100 mg/m2 administered intraperitoneally weekly) plus standard-of-care (SoC) neoadjuvant and adjuvant chemotherapy (NACT) compared to IL-12 levels in women treated with IMNN-001 (79 mg/m2). In this analysis increases in IL-12 levels were sampled in the peritoneal fluid cavity, which is the primary tumor microenvironment. Little to no changes were observed in the systemic blood stream of treated patients.

LEO Pharma Meets Primary Endpoint in Phase 3 DELTA China Trial With Anzupgo® (delgocitinib) Cream in Chinese Patients with Moderate to Severe Chronic Hand Eczema (CHE)

Madison-based LEO Pharma announced the positive results for the primary endpoint from the double-blind treatment period of the DELTA China trial. DELTA China is a phase 3 clinical trial with Anzupgo® (delgocitinib) 20mg/g cream, a topical pan-Janus kinase (JAK) inhibitor, for the potential treatment of Chinese adults and adolescents (aged 12 and above) with moderate to severe Chronic Hand Eczema (CHE) for whom topical corticosteroids are inadequate or inappropriate. The Phase 3 trial consists of a 16-week randomized, double-blind, vehicle-controlled, treatment period, followed by an open-label treatment period of 36 weeks. A total of 362 subjects were recruited and randomized in a 2:1 ratio to receive either Anzupgo or cream vehicle.

Cosette Pharmaceuticals Acquires Mayne Pharma for $672M

Bridgewater-based pharmaceutical giant Cosette Pharmaceuticals has made a $672 million cash takeover bid for women’s health and dermatology group Mayne Pharmaceuticals. The Boards of Directors of both companies have approved the transaction and Mayne Pharma’s Board of Directors has unanimously recommended that its shareholders vote in favor of the transaction. The combination of the two companies will create a leading women’s health and dermatology focused pharmaceuticals company in the U.S. with an established presence in international markets, utilizing the strengths of two industry players to drive innovation and expand access to women’s health therapies. “This acquisition marks a transformational step for Cosette, adding patented, high-growth products to solidify our leadership in women’s health in the U.S. and expanding our reach globally,” Apurva Saraf, president and CEO of Cosette Pharma said.

UroGen Advances Long-Term Growth Strategy by Acquiring a Next-Generation Investigational Oncolytic Virus (ICVB-1042) and Announces Multiple Strategic Research Collaboration

Princeton-based UroGen Pharma Ltd. announced expansion of its oncology pipeline portfolio through the acquisition of assets relating to a next-generation oncolytic virus ICVB-1042 from IconOVir Bio, Inc. (IconOVir). In addition, UroGen also announced that it has entered into multiple strategic research collaborations to explore the potential of its proprietary RTGel technology to enhance clinical effectiveness of multiple immunotherapies, including optimizing dwell time to improve treatment outcomes. The use of biological agents to treat bladder cancer has its roots in the development of Bacillus Calmette-Guérin (BCG) therapy. Much like BCG therapy, ICVB-1042 is being developed to activate the immune system within the tumor microenvironment, but unlike BCG therapy, ICVB-1042 has the potential to selectively destroy cancer cells while retaining potency and trigger a robust immune response.

Rx Bandz and Zeyco Sign MOU for Filling Epinephrine Auto-Injector and Distribution in Latin America

Fairfield’s Rx Bandz has signed a memorandum of understanding (MOU) with Zeyco to fill and distribute its MiniJect® epinephrine auto-injector across Mexico, Central and South America. This partnership aims to improve access to affordable, life-saving treatment for individuals with severe allergies. “Zeyco is a world-class provider with extensive experience in epinephrine. We are honored to collaborate with them to expand access to this critical medication,” said Jessica Walsh, CEO of Rx Bandz. “Our MiniJect is a compact, patient-centric auto-injector with an extended shelf life and reduced environmental impact, addressing key barriers to use.” Zeyco’s Pharmaceutical Plant Director, Alejandro Zepeda Sahagun, emphasized the urgent need for emergency epinephrine auto-injectors in Latin America. “Currently, there are no publicly available options in Mexico and many neighboring countries. Making MiniJect accessible will help prevent life-threatening anaphylactic reactions.”

Mallinckrodt and Endo to Combine to Create a Global, Scaled, Diversified Pharmaceuticals Leader

Mallinckrodt plc, with a site in Bedminster, and Endo, Inc. announced that they have entered into a definitive agreement to combine in a stock and cash transaction to create a global, scaled, diversified pharmaceuticals leader. "The combination of Mallinckrodt and Endo brings together two essential pharmaceuticals organizations to accelerate value creation for our shareholders, customers, employees, the patients we serve and our other stakeholders," said Siggi Olafsson, President and CEO of Mallinckrodt. "Our businesses are highly complementary, with durable, on-market products in our branded portfolios and extensive capabilities across the value chain in our generics businesses. This exciting combination will create a larger and more diversified entity with the scale and resources needed to unlock the full potential of both companies. Additionally, with a strong pro forma balance sheet and compelling synergy opportunities, we will have greater flexibility to invest in innovation and pursue growth opportunities."

CoreWeave Secures $11.9 Billion Deal With OpenAI

BioNJ Member CoreWeave has announced a landmark agreement with OpenAI to expand the AI leader’s computing capacity, supporting the training and deployment of its latest models at scale. The contract is valued at up to $11.9 billion, further cementing CoreWeave’s role as a key infrastructure provider in the AI industry. As part of the agreement, OpenAI will become an investor in CoreWeave, acquiring $350 million in CoreWeave stock. This partnership underscores CoreWeave’s ability to deliver high-performance cloud solutions tailored for AI workloads. “Partnering with OpenAI on this new contract highlights CoreWeave’s proven capability to provide reliable and scalable infrastructure for world-leading AI labs,” said Michael Intrator, co-founder and CEO of CoreWeave. “We remain a partner of choice for organizations pioneering AI’s potential to transform the world.”

Artificial Turf: Princeton University AI Hub Aims to Position N.J. as Sector Leader

Artificial Intelligence has been around since the 1950s. According to IBM, GenAI is artificial intelligence that can create original content — such as text, images, video, audio or software code — in response to a user’s prompt or request. New Jersey is responding to the opportunities and possibilities that GenAI technologies represent in the state. Last April, Gov. Phil Murphy and Princeton University President Chris Eisgruber announced plans to formally create a hub for AI activity and research on the Princeton campus. Operated in conjunction with the N.J. Economic Development Authority, the hub will aim to leverage state funding and private-sector partnerships to advance the state’s leadership in AI innovation in AI technologies, drive job growth and economic development across the region, promote rapid and responsible development of the field and guide governments and public entities on AI implementation.

Iktos Secures Prestigious EIC Accelerator Grant to Advance its Automated AI and Robotics Platform for Drug Discovery

BioNJ Member Iktos, with a site in Neshanic Station, has secured a prestigious €2.5 million grant from the European Innovation Council (EIC) Accelerator to advance its AI and robotics synthesis automation technology. This funding, with the potential for an additional €5 million from the EIC, will propel Iktos' mission to revolutionize drug design by fully integrating AI-driven molecular generation, automated synthesis, and high-precision biological testing — paving the way for faster, more efficient drug discovery. Over the next two years, the company will expand its drug discovery platform following the 2024 acquisition of Synsight and its proprietary technology, MTBench, which enables high-precision biological screening of challenging targets in biologically relevant environments. The investment will boost Ikto's capabilities by integrating AI-driven molecular design, robotic synthesis, and biological testing. 

Palisades Therapeutics Secures USPTO Patent for AI-Supported Portable Modular Artificial Liver

Cliffside Park-based Pop Test Oncology LLC dba Palisades Therapeutics proudly announces that the United States Patent and Trademark Office has awarded a patent for its Portable Modular Artificial Liver—a groundbreaking advancement in drug discovery, toxicity testing, and regenerative medicine. This AI-supported system is set to transform liver disease research and therapeutic development by providing an unprecedented human-relevant liver model. Metabolic Dysfunction-Associated Steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), is a progressive liver disease with limited treatment options. Traditional drug testing relies on animal models that often fail to predict human outcomes. The Portable Modular Artificial Liver overcomes these challenges by accurately replicating human liver function in a dynamic, AI-supported system.

Biomedical-Focused Strategic Innovation Center Coming to Camden

The New Jersey Economic Development Authority and the Coriell Institute for Medical Research are bringing the Garden State’s latest Strategic Innovation Center (SIC) to Camden. Officials say the innovation center will leverage the world-class assets available at Coriell, such as its international reputation in biobanking and cutting-edge biomedical research, to support innovative startup companies. Coriell is a leader in biomedical innovation. It is also home to some of the most famous biobank repositories, including the National Institutes of Health premier collections representing thousands of human diseases. “Through the NJEDA’s Strategic Innovation Center initiative, Camden will serve as a pivotal hub for cutting edge biomedical research, advancing lifesaving technologies in cancer treatment, stem cell applications, epigenetics, and drug development,” said NJEDA Chief Executive Officer Tim Sullivan.

New NJBioFutures Partnership Aims to Promote Workforce Development in Vital Industry

“The NJBioFutures collaboration between Raritan Valley, Middlesex and Mercer County community colleges and industry partners represents the very best in public-private partnerships,” said Raritan Valley Community College President Michael McDonough during the recent launch of the program at the Branchburg-based institution. Johnson & Johnson is serving as a Founding Sponsor, contributing $1 million to NJBioFutures, which will be housed at three leading community colleges – RVCC, Middlesex and Mercer ― with a goal of advancing workforce development in the Garden State’s rapidly expanding cell and gene therapy sector. That support from Johnson & Johnson will support the development of curricula and modernization of training and lab facilities on each college campus, including a more than 22,000-square-foot expansion of the Workforce Training Center on the RVCC campus.

Institute for Life Science Entrepreneurship Launches Unique Venture Studio Training Program

The Institute for Life Science Entrepreneurship (ILSE) at Kean University launched the Entrepreneur Training Program to build the next generation of life sciences companies in New Jersey. Funded by the U.S. Small Business Administration, the ETP combines hands-on, mentor-guided learning with leadership development in a unique way, developing the entrepreneur along with the new start-up. The Program is open to prospective and current entrepreneurs and begins in August 2025. Applications are now open for participants, and ILSE is actively recruiting Mentors and Instructors. To apply, visit the ILSE website or use one of the following links:

• Program participants apply here. Applications are due May 23.
• Mentors and Instructors register here. The early registration deadline is April 15.

Legacy Moment: Murphy Proposes to Add $20 Million to Fund Additional Strategic Innovation Centers

Gov. Phil Murphy has made creating an innovation economy the cornerstone of his administration. During his last budget address, he said he wants to provide an additional $20 million in funding to support one of his signature innovation programs: Strategic Innovation Centers. SICs are facilities that support research and development, innovation and entrepreneurship through mentorship, networking opportunities, hands-on training, business support services and education opportunities. SICs can be accelerators, incubators or research centers — and often are connected to institutions of higher education and come with support from the N.J. Economic Development Authority. Having a physical location where entrepreneurs can collaborate will help support new, diverse innovators and help drive long-term economic growth. The additional $20 million in funding could provide resources to open two or three more centers.

Kean University’s Merger Proposal Accepted by NJCU

New Jersey City University’s (NJCU) Board of Trustees have voted to accept Kean University’s merger proposal, a move aimed at addressing NJCU’s financial challenges and ensuring its long-term sustainability. This decision marks the beginning of negotiations to formalize a letter of intent, integrating NJCU into the Kean University system under the new name “Kean Jersey City.” The merger carries significant implications for faculty unions at both institutions, which are affiliated with AFT New Jersey and the Council of New Jersey State College Locals. Union leaders have pledged to advocate for fair and equitable treatment of faculty and staff throughout the transition. AFT New Jersey President Jennifer S. Higgins emphasized the importance of collaboration with both administrations to protect union members’ interests.

Two Irish Health Start-ups Selected for HMH, Choose NJ Pitch Competition

A pitch competition jointly run by Hackensack Meridian Health and Choose New Jersey announced the selection of two winning Irish health start-ups. The HMH Emerge Program launched to provide Ireland-based health startups with the opportunity to bring their ideas to fruition through piloting, partnering, mentorship and/or investment. Selected by the HMH Emerge board of directors, the winners are: 1) Spiorad – aimed at simplifying the closure of femoral vascular access, making complex cardiovascular procedures safer for patients and easier for physicians. 2) NeuroBell – offers a pocket-sized (electroencephalogram) monitor with automated neonatal seizure detection with minimal setup and maintenance. Choose New Jersey Chief Business Development Officer Bill Noonan noted that the innovation coming out of Ireland is “truly remarkable.”

Hackensack Meridian Health Receives Grant from Genentech

Hackensack Meridian Health will receive a two-year, $750,000 Genentech Health Equity Innovation Grant to increase lung cancer screenings. The grant was awarded to Lisa Carter-Bawa, Ph.D., MPH, APRN, ANP-C, FAAN, the director of the Center for Discovery and Innovation (CDI)’s Cancer Prevention Precision Control Institute (CPPCI) and her team, in partnership with Greater Bergen Community Action (GBCA). The partnership between Hackensack Meridian Health and GBCA is called Community-2-Care+Environment: Lung, or C2C+E. It will result in hundreds, and perhaps thousands, of at-risk patients being screened for lung cancer in Bergen and Passaic counties. Many of these patients will be underserved and from minority groups, particularly African-Americans and Latinos. The main goal: to get full screening for the disease for those most at-risk.

HMH Research Projects Awarded $1.1M by NJ Health Foundation

Hackensack Meridian Health (HMH) has been awarded 25 grants totaling more than $1.1 million from the New Jersey Health Foundation (NJHF) for important research and community health projects. The grants support work across the entire HMH health network, spanning not only the Hackensack Meridian Health Research Institute (HMHRI) and clinical and basic research, but also the Hackensack Meridian School of Medicine (HMSOM), and a multitude of sites across the Garden State. “This remarkable level of funding from the New Jersey Health Foundation underscores the innovative and impactful research being conducted across our network,” said Robert C. Garrett, FACHE, CEO of Hackensack Meridian Health. “These grants will fuel critical advancements in patient care and contribute to a healthier future for the communities we serve.”

Middlesex County Breaks Ground on the Middlesex County Innovation Magnet School at Middlesex College

Middlesex County, in partnership with Middlesex County Magnet Schools, celebrated a major milestone with the groundbreaking of the new Middlesex County Innovation Magnet School on the Middlesex College campus. The event brought together County officials, state leaders, and community partners to advance the County’s Community, Innovation and Opportunity (CIO) Strategic Investment Plan — underscoring a commitment to education, workforce development, and economic growth. Located on the newly named “Innovation Way” at Middlesex College, the Innovation Magnet School will be the sixth high school within the Middlesex County Magnet School district and is slated to open in fall 2026. The 75,550-square-foot facility will introduce advanced academic programs that cover engineering principles supporting robotics and energy-efficient systems, applications of advanced technology in building design, data networking systems, and agricultural practices to enhance environmental quality.

NJEDA'S Emerge Program Secures Major Pharmaceutical Company's Presence in NJ

The New Jersey Economic Development Authority (NJEDA) Board approved tax credits under the Emerge Program to support Sun Pharmaceutical Industries Inc.’s (Sun Pharma) plan to open a new U.S. headquarters in Princeton. Sun Pharma engages in pharmaceutical and medicine manufacturing and is the fourth largest specialty generic pharmaceutical company in the world. The pharmaceutical company’s new location will create 220 new jobs and keep hundreds of jobs in the state, including the 230 that are in its current headquarters. Following several recent acquisitions, Sun Pharma is committed to the continued growth of its business in New Jersey. With more than 600 employees already working at locations across New Jersey, including its Princeton headquarters and facilities in Cranbury and New Brunswick, Sun Pharma has been scouting new locations large enough to house several hundred additional employees.

Nokia Bell Labs’ Future HELIX HQ Approved for $103M in Aspire Tax Credits

The New Jersey Economic Development Authority (NJEDA) board approved Aspire tax credits for H-2, the second building at the forthcoming HELIX in New Brunswick, which will house Nokia Bell Labs. The New Jersey Health + Life Sciences Exchange is one of the state’s most exciting and ambitious projects. The construction of H-1 continues to make progress, slated for completion this year. The groundbreaking for H-2 is also slated for 2025. That project anticipates completing the 10-story, 350,000-square-foot facility by the beginning of 2028. “Through the partnership of major institutions, like Nokia Bell Labs, the HELIX will support the creation of advanced technologies and allow startups to scale their business and commercialize lifechanging inventions,” said Governor Murphy.

Senate Passes Bill to Increase Tax Incentives for Angel Investors

The State Senate passed legislation, strongly supported by NJBIA, that would increase tax credits provided under the New Jersey Angel Investor law for qualified investments made in New Jersey emerging technology business ventures. The bill, S-3189, does not change the annual $35 million annual cap set by law for the Angel Investor program, but it increases the value of tax credits from 20% to 35% of a taxpayer’s qualified investment in an emerging technology start-up or venture fund. The additional 5% bonus under current law means that investments made in emerging technology businesses located in low-income communities, or state certified as a minority or women owned enterprises, could be eligible for a tax credit of up to 40%. The Senate voted 39-0 to pass the bill, which is sponsored by Senator Andrew Zwicker (D-16).  

NJ Start-ups With Maternal, Infant Health Focus Awarded $1.7M

The Commission on Science, Innovation, and Technology (CSIT) awarded $1.7 million in grants to 23 Garden State-based startups focused on maternal and infant health. The grants come via Round 2 of CSIT’s Maternal and Infant Health R&D Grant Program. The above startups will receive awards up to $75,000. “CSIT is thrilled to continue our partnership with First Lady Murphy, the Maternal and Infant Health Authority, and the NJEDA on this vital initiative that will support babies and their families by leveraging the innovative capabilities of New Jersey startups to tackle critical maternal and infant health care challenges,” said CSIT board Chair Debbie Hart. “In Round 2 of the program, 23 companies have received $1.7 million in funding to advance their projects – creating solutions where they are urgently needed.”

Pfizer Names Former FDA Director as Chief Medical Officer

BioNJ Member Pfizer, with a site in Peapack, named the former head of the U.S. health regulator's drug evaluation unit, Patrizia Cavazzoni, as its Chief Medical Officer, bringing back an industry veteran to lead its regulatory and drug safety operations. Dr. Cavazzoni, who stepped down from her role at the Food and Drug Administration in January, was among the top officials who left ahead of the new administration under President Donald Trump. She will succeed Aida Habtezion, who will depart the company to pursue other opportunities, and will report to Chris Boshoff, Pfizer's Chief Scientific Officer and R&D head. Dr. Cavazzoni headed clinical sciences and development operations across multiple senior roles in her previous stint at Pfizer. She has also held several senior executive positions in the pharmaceutical industry for nearly two decades.

CoreWeave Announces New Chief Information Security Officer Jim Higgins

BioNJ Member CoreWeave announced Jim Higgins has been appointed as the company’s new Chief Information Security Officer (CISO). With extensive experience in cybersecurity, regulatory compliance and enterprise risk management, Mr. Higgins will oversee the company’s security strategy, governance and infrastructure protection across the CoreWeave Cloud Platform. This strategic hire reinforces CoreWeave’s commitment to enterprise-grade security solutions that leading organizations can rely on to confidently scale their AI operations. Mr. Higgins was most recently CISO for Snap Inc., a global tech company most known for its social media platform Snapchat. Before Snap, Higgins was CISO at Block, Inc. and held senior leadership positions in information security and threat protection at Google and other Fortune 100 entities.

Johnson & Johnson Announces Darren Snellgrove as Vice President, Investor Relations

New Brunswick-based BioNJ Member Johnson & Johnson announced the appointment of Darren Snellgrove to Vice President, Investor Relations. Jessica Moore, who has served as Vice President, Investor Relations since 2021, will assume the role of Group Chief Financial Officer, Johnson & Johnson, Innovative Medicine. The new appointments are effective May 1, 2025, and Mr. Snellgrove as well as Ms. Moore, will continue to report to Johnson & Johnson’s Executive Vice President and Chief Financial Officer, Joseph Wolk. Mr. Snellgrove brings 25 years of strong and broad financial leadership spanning across Innovative Medicine, MedTech, R&D and Business Development positions. He most recently served as the Group Chief Financial Officer for Innovative Medicine where he has played a critical role in the financial leadership and analysis of Johnson & Johnson’s Innovative Medicine business.

GSK Announces Dr. Gavin Screaton to Join the Board as Non-Executive Director

Warren-based BioNJ Member GSK plc announced that Dr Gavin Screaton will join the Board of the company as a Non-Executive Director from 1 May 2025. Dr. Screaton’s appointment follows a search to replace the skills and experience of Dr Jesse Goodman who steps down from the Board after the company’s Annual General Meeting in May 2025, after nine years of service. Dr. Screaton is currently Head of the world-leading¹ Medical Sciences Division at the University of Oxford, and an expert in the field of immunology and infectious diseases, two areas of science critical to GSK. Dr. Screaton is a Fellow of the Academy of Medical Sciences, a Fellow of the Royal College of Physicians, and a former Senior Investigator at the National Institute for Health Research in the United Kingdom.

Cyclacel Pharmaceuticals Announces the Appointment of New Executive Leadership

Berkeley Heights-based BioNJ Member Cyclacel Pharmaceuticals, Inc. announced Dr. Doris Wong Sing Ee was elected as Chief Executive Officer and Executive Director of Cyclacel. Cyclacel also announced the appointment of Kiu Cu Seng as Executive Director, Secretary and Chief Financial Officer and, in such role, he will serve as the Company’s co-principal financial officer and co-principal accounting officer. “I am honored to serve as Cyclacel’s next CEO,” said Datuk Dr. Doris Wong Sing Ee. “I look forward to our exciting next chapter as I work with the rest of Cyclacel’s management team and board of directors to further build our biopharmaceutical business and deliver value for patients, shareholders and our other stakeholders.”

Catalent Announces New Board Appointments

Catalent added three new directors with deep expertise across biopharma and life sciences. The Somerset-based leader in enabling the development and supply of better treatments for patients worldwide appointed Susan Mahony, Marie-France Tschudin and Tim Walbert to its Board. “We are thrilled to welcome these biopharma leaders to our Board, whose expertise in navigating the complexities of the healthcare landscape will be invaluable,” John Greisch, chair of the board said. “We look forward to benefitting from their insights in support of Catalent’s mission to develop, manufacture and supply products that help people live better and healthier lives.” The Catalent Board also includes Jonathan Levy, senior partner at Novo Holdings, and Charles Patten, partner at Novo Holdings, both of whom joined the board upon Catalent becoming a private company.

Students 2 Science STEM Volunteer Opportunities

Inspire the next generation of STEM leaders – Volunteer at Students 2 Science (S2S), a recognized leader in providing rigorous, hands-on STEM experiences for students in 5th-12th grades. S2S is currently recruiting volunteers for its in-lab ISAAC program, which offers rigorous, hands-on STEM experiences for middle and high school students in state-of-the-art laboratories in East Hanover and Newark, NJ. For more information, click here. To register to volunteer with S2S or if you have any questions, please email info@students2science.org or call (973) 947-4880 ext. 545.

44th Annual Coriell Institute Science Fair

March 15, 2025 | Hosted by Camden County College, Blackwood, NJ

Join us as a sponsor of the 44th Annual Coriell Institute Science Fair! Support middle and high school students from Camden, Gloucester, and Burlington counties as they embark on their journey to the Delaware Valley Science Fair and the Regeneron International Science and Engineering Fair (ISEF), where they can compete for over $8 million in awards and scholarships. Samantha Piccolo at spiccolo@coriell.org can provide more details.