The Unintended Consequences of the Inflation Reduction Act Threaten Patient Health and America’s Leadership in the Life Sciences

As policymakers continue to evaluate the law’s implementation, it is important to consider the real-world impact this law is having on patient access, innovation and American jobs. Protecting America’s leadership in life sciences innovation is essential, and today that must start with understanding the current policy environment, threats, and opportunities. Read what industry experts have to say about IRA’s current impact.

Revolutionizing Clinical Trial Enrollment: The Vital Role of Community Pharmacies

Clinical trial enrollment poses a significant hurdle for researchers and sponsors, grappling with the challenge of reaching diverse and representative patient populations. The traditional model, with participants often traveling considerable distances to testing sites, results in a staggering 80% of trials failing to meet enrollment targets and timelines, causing substantial financial losses and delays in critical drug development. Enter pharmacies, uniquely positioned to revolutionize clinical trial enrollment. With nearly 90% of Americans residing within five miles of a pharmacy, these accessible hubs provide a strategic solution to engage a broader population. Pharmacies could sensibly serve as enrollment sites, offering varied patient demographics and capitalizing on the trust patients place in pharmacy staff.

NJ Life Sciences Report Reveals Thriving Ecosystem

Choose New Jersey, in partnership with JLL, BioNJ and the New Jersey Economic Development Authority, has released the 2024 New Jersey Life Sciences Report, showcasing New Jersey’s thriving life sciences ecosystem and highlighting its place as a national leader for companies seeking market access, affordable real estate and world-class talent. The report showcases the state’s top-ranked research universities, highly skilled and highly trained workforce, the country’s highest concentration of engineers and scientists per square mile, multi-stage incubators, and flexible and creative economic incentive programs. It also reveals that New Jersey offers top-tier R&D and manufacturing spaces, including repurposed legacy sites that are business-ready and cost-effective.

“New Jersey continues to lead the way in life sciences innovation with more than 50% of all new FDA approvals in 2023 coming from companies within the Garden State, thanks to its robust and collaborative life sciences ecosystem,” said Debbie Hart, President and CEO of BioNJ. “Life sciences companies achieve greater value for their investment in New Jersey, partly due to its strong research institutions, supportive government policies, deep talent pool and affordable lab space.”

Driving Biotech Innovation in New Jersey

This series features key stakeholders from the global biotech ecosystem engaging in discussions on various topics focused on fostering and advancing biotech innovation on a global scale. This episode features Debbie Hart, sharing her insights about the position of New Jersey as a key cluster of innovation for Life Sciences.

NJ Excels in Life Sciences and Med-Tech Industry Support

With the work of trade associations, such as BioNJ, the State's life sciences and medical technology sectors are flourishing and delivering the breakthrough innovations patients around the world depend on. BioNJ is a value-centric organization dedicated to ensuring a vibrant ecosystem where science is supported, companies are created, drugs are developed and patients are paramount. We are committed to promoting policies that facilitate tomorrow's miracles and ensure that patients can access them. Recognized as a respected thought leader, an influential advocate and a sought-after convener of the life sciences industry, BioNJ works closely with policymakers in Trenton and Washington, D.C. We champion the advancement of life sciences and advocate for health equity and affordable healthcare.

Insmed Announces Positive Topline Results from Landmark ASPEN Study of Brensocatib in Patients with Bronchiectasis

Bridgewater-based BioNJ Member Insmed Incorporated (announced positive topline results from the ASPEN study, a global, randomized, double-blind, placebo-controlled Phase 3 study to assess the efficacy, safety and tolerability of brensocatib in patients with non-cystic fibrosis bronchiectasis. The study met its primary endpoint, with both dosage strengths of brensocatib demonstrating statistically significant reductions in the annualized rate of pulmonary exacerbations (PEs) versus placebo. The study also met several of its prespecified secondary endpoints with statistical significance. Based on these results, Insmed plans to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for brensocatib in patients with bronchiectasis in the fourth quarter of 2024. Pending regulatory approvals, Insmed anticipates a U.S. launch for brensocatib in mid-2025 followed by launches in Europe and Japan in the first half of 2026.

Insmed Announces Pricing of $650 Million Public Offering of Common Stock

Bridgewater-based BioNJ Member Insmed Incorporated announced that it priced an underwritten public offering of 12,621,359 shares of its common stock at a price to the public of $51.50 per share. The gross proceeds to Insmed from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Insmed, are expected to be approximately $650 million. Insmed intends to use the net proceeds from this offering to fund continued research and development of brensocatib as well as pre-commercial and, if approved, commercialization activities related to brensocatib, activities related to the further commercialization and development of ARIKAYCE® (amikacin liposome inhalation suspension), further research and development of treprostinil palmitil inhalation powder (TPIP) and any of the company's other research product candidates, and for other general corporate purposes, including business expansion activities.

PTC Therapeutics Announces Validation of Sepiapterin European MAA

Warren-based BioNJ Member PTC Therapeutics, Inc. announced that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. In addition, submissions are planned in a number of additional countries in 2024 including Brazil and Japan. The sepiapterin MAA includes data from the Phase 3 APHENITY trial which demonstrated a mean reduction in Phe levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU. The vast majority of subjects (84%) achieved Phe control in accordance with treatment guidelines of <360 µmol/L, and 22% of subjects had normalization of Phe levels.

Celldex Therapeutics Presents Data Demonstrating Profound Improvements in Angioedema in Barzolvolimab Phase 2 Study in Chronic Spontaneous Urticaria at EAACI 2024

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced data demonstrating that barzolvolimab profoundly improves angioedema at 12 weeks in the Company’s Phase 2 clinical trial in chronic spontaneous urticaria (CSU). Celldex previously announced that the Phase 2 study in CSU met its primary and secondary endpoints at 12 weeks with clinically meaningful and statistically significant decreases in UAS7 (weekly urticaria activity score) compared to placebo across multiple dose groups and demonstrated a favorable safety profile. The data presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress further support these results by demonstrating improvements in AAS7 (weekly angioedema activity score) and additional measures of angioedema control.

Cyclacel’s Fadraciclib Demonstrates Efficacy in Patient-Derived Colorectal Cancer Models at the 2024 ASCO Annual Meeting

Berkeley Heights-based BioNJ Member Cyclacel Pharmaceuticals, Inc. announced a presentation by independent investigators of preclinical data demonstrating therapeutic potential of fadraciclib, the Company’s cyclin-dependent kinase (CDK) 2/9 inhibitor, as a novel treatment for metastatic colorectal cancer (CRC). The data show that fadraciclib substantially inhibited growth, triggered apoptosis, and induced anaphase catastrophe in CRC patient-derived organoids (PDOs) and xenografts (PDX). Eighteen CRC PDOs generated from patients undergoing biopsy or resection for their primary or metastatic CRC were treated with standard of care chemotherapy (oxaliplatin, irinotecan (SN38) and 5-Fluorouracil), palbociclib (CDK4/6 inhibitor), or fadraciclib, to determine sensitivity to each drug. Subsequently, three matching, patient-derived xenografts (PDXs) were generated for in vivo validation and treated with fadraciclib via oral gavage at a dose of 25 mg/kg twice daily, five days a week for two weeks. 

Y-mAbs Announces Publication of Preclinical GD2-SADA Data at 2024 ASCO Annual Meeting

BioNJ Member Y-mAbs Therapeutics, Inc., with a site in Nutley, announced the publication of preclinical GD2-SADA data. The published abstract titled “Preclinical characterization of pretargeted radioimmunotherapy with GD2-SADA, a self-assembling and disassembling bispecific fusion protein” characterizes the binding properties of GD2-SADA across several GD2-expressing cell lines and lanthanide metal-DOTA complexes, while also demonstrating its anti-tumor efficacy when used with Lutetium 177 (Lu177)-DOTA in a two-step approach to pretargeted radioimmunotherapy (“PRIT”). In this analysis, GD2-SADA showed tight binding to cell lines expressing GD2, a glycolipid implicated in the malignant transformation of multiple solid tumors. GD2-SADA binding was significantly improved by a p53-derived domain that drives the self-assembly and disassembly (“SADA”) of GD2-SADA tetramers, which possess four distinct GD2-binding domains that cumulatively enhance binding. Previous studies have shown that the unbound GD2-SADA protein disassembles over time, facilitating clearance by the kidneys.

Mental Health America (MHA) with support from Otsuka America Pharmaceutical, Launches Equity Impact Zones (EIZ) Program

Mental Health America (MHA) with support from Otsuka America Pharmaceutical, Inc. launched a new program called Equity Impact Zones (EIZ). The EIZ program will help New Jersey communities implement and sustain solutions to address mental health equity gaps. Through Otsuka’s support of MHA, the first EIZ program will offer multi-year grant funding to non-profit organizations in Burlington County and the City of Trenton that are actively working to improve mental health equity in local communities. Each community eligible for funding through the EIZ program will have a community-based and community-led strategy for addressing their specific mental health equity need. The goal of this investment is to enable recipients to quickly deliver a visible improvement soon after a project has begun, as well as to demonstrate plausibility, scalability, and the possibility of change. 

Merck & Moderna Announce 3-Year Data For mRNA-4157 (V940) in Combination With KEYTRUDA® (pembrolizumab) Demonstrated Sustained Improvement in Recurrence-Free Survival & Distant Metastasis-Free Survival Versus KEYTRUDA in Patients With High-Risk Stage III/IV Melanoma Following Complete Resection

Rahway-based BioNJ Member Merck & Co. and Moderna announced the first presentation of results from a planned analysis from the Phase 2b randomized KEYNOTE-942/mRNA-4157-P201 study, a clinical trial evaluating mRNA-4157 (V940), an investigational individualized neoantigen therapy (INT), in combination with KEYTRUDA, Merck’s anti-PD-1 therapy, in patients with resected high-risk melanoma (stage III/IV) following complete resection (n=157). With a median follow-up of approximately three years (34.9 months), adjuvant treatment with mRNA-4157 (V940) in combination with KEYTRUDA continued to demonstrate a clinically meaningful and durable improvement in recurrence-free survival (RFS), the primary endpoint of the study, reducing the risk of recurrence or death by 49% (HR [95% CI], 0.510 [0.288–0.906]; two-sided nominal p-value 0.019) compared with KEYTRUDA alone.

Merck to Acquire EyeBio

Rahway-based BioNJ Member Merck & Co. and Eyebiotech Limited, a privately held ophthalmology-focused biotechnology company, today announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire EyeBio. “We continue to execute on our science-led business development strategy to expand and diversify our pipeline,” said Dr. Dean Y. Li, President, Merck Research Laboratories. “The EyeBio team, under the leadership of Dr. David Guyer and Dr. Tony Adamis, has a strong track record of developing groundbreaking ophthalmology therapies. By combining our strengths, we aim to advance with rigor and speed the development of their promising pipeline of candidates targeting retinal diseases.”

FDA Grants Priority Review to Merck’s Application for KEYTRUDA® (pembrolizumab) Plus Chemotherapy as First-Line Treatment of Patients With Unresectable Advanced or Metastatic Malignant Pleural Mesothelioma

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 chemotherapy, for the first-line treatment of patients with unresectable advanced or metastatic malignant pleural mesothelioma. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 25, 2024. The sBLA is based on data from the pivotal Phase 2/3 IND.227/KEYNOTE-483 trial. Results from the final analysis of the study showed KEYTRUDA in combination with chemotherapy demonstrated a statistically significant improvement in overall survival (OS), reducing the risk of death by 21% (HR=0.79 [95% CI, 0.64-0.98]; two-sided p value=0.0324), with a median OS of 17.3 months (95% CI, 14.4-21.3) versus 16.1 months (95% CI, 13.1-18.2) for chemotherapy alone.

Merck Announces Phase 3 KEYNOTE-522 Trial Met its Overall Survival (OS) Endpoint in Patients With High-Risk Early Stage Triple Negative Breast Cancer (TNBC)

Rahway-based BioNJ Member Merck & Co. announced that the Phase 3 KEYNOTE-522 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, met its overall survival (OS) endpoint, in combination with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent after surgery (adjuvant) for the treatment of patients with high-risk early stage triple-negative breast cancer (TNBC). At a pre-specified interim analysis conducted by an independent Data Monitoring Committee, KEYTRUDA demonstrated a statistically significant and clinically meaningful improvement in OS compared to pre-operative chemotherapy. The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were observed. Results will be presented at an upcoming medical meeting and shared with regulatory authorities.

Novartis Presents Latest Phase III Fabhalta (iptacopan) Data in C3 Glomerulopathy (C3G) Showing Clinically Meaningful and Statistically Significant 35.1% Proteinuria Reduction vs. Placebo

East Hanover-based BioNJ Member Novartis presented results from the 6-month, double-blind period of the Phase III APPEAR-C3G study of Fabhalta (iptacopan). Patients treated with Fabhalta in addition to supportive care achieved a 35.1% (p=0.0014) reduction in proteinuria (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 6 months when compared to placebo on top of supportive care. In many kidney diseases, proteinuria reduction is an increasingly recognized surrogate marker correlating with delaying progression to kidney failure. Fabhalta is an oral Factor B inhibitor of the alternative complement pathway being investigated in adult patients with C3 glomerulopathy (C3G). Regulatory submissions, including to the FDA and EMA, for the adult C3G indication are planned for the second half of 2024.  

Novartis Atrasentan Phase III Data Show Clinically Meaningful Proteinuria Reduction Further Advancing Company's IgA Nephropathy (IgAN) Portfolio

East Hanover-based BioNJ Member Novartis presented results from a pre-specified interim analysis of the Phase III ALIGN study of atrasentan, an investigational oral selective endothelin A (ETA) receptor antagonist, in patients with IgA nephropathy (IgAN)1. Patients treated with atrasentan, in addition to supportive care (maximally tolerated and stable dose of a renin-angiotensin system [RAS] inhibitor), achieved a 36.1% (p<0.0001) reduction in proteinuria (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 36 weeks when compared to placebo on top of supportive care. The study also showed atrasentan has a favorable safety profile consistent with previously reported data. Proteinuria reduction is a recognized surrogate marker correlating with delaying progression to kidney failure and has been used as an endpoint in IgAN clinical trials to support accelerated regulatory approvals.

Novartis Scemblix® Phase III Data First to Show superior Efficacy With a Favorable Safety and Tolerability Profile vs. Standard-of-Care TKIs in Adults With Newly Diagnosed CML

East Hanover-based BioNJ Member Novartis presents positive results from the pivotal Phase III ASC4FIRST trial g. Scemblix® (asciminib) demonstrated superior major molecular response (MMR) rates at week 48 compared to investigator-selected standard-of-care (SoC) tyrosine kinase inhibitors (TKIs) imatinib, nilotinib, dasatinib and bosutinib, and compared to imatinib alone in patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP). Scemblix also showed a numerical improvement in MMR at week 48 vs. second generation (2G) TKIs (nilotinib, dasatinib and bosutinib). Additionally, Scemblix demonstrated a favorable safety and tolerability profile, with fewer adverse events (AEs) and treatment discontinuations vs. both imatinib and 2G TKIs.

Latest Analysis of Novartis NATALEE Study Shows Kisqali® Reduces Risk of Cancer Recurrence for Early Breast Cander Patients With High-Risk Node-Negative Disease

East Hanover-based BioNJ Member Novartis is announcing results from a subgroup analysis of patients with high-risk, node-negative (N0) hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer (EBC) from the Phase III NATALEE trial. The latest analysis demonstrated that Kisqali® (ribociclib) plus endocrine therapy (ET), compared to ET alone, showed an improvement in rates of invasive disease-free survival (iDFS), distant recurrence-free survival (DRFS), and distant disease-free survival (DDFS) in high-risk EBC patients with N0 disease. The safety profile of Kisqali at the 400 mg dose in the high-risk, N0 subgroup remains consistent with the well-tolerated profile previously demonstrated in the intent-to-treat population with generally low-grade adverse events (AEs), other than laboratory findings.

Evotec and CHDI Foundation Extend Strategic Drug Discovery Collaboration in Huntington’s Disease

Princeton-based BioNJ Member Evotec SE has announced the extension of its 20-year collaboration with CHDI Foundation, Inc. CHDI is a privately funded nonprofit biomedical research organization exclusively dedicated to collaboratively developing therapeutics that will substantially improve the lives of those affected by Huntington’s disease. The collaboration will further our fundamental scientific understanding of the disease and thereby aid the development of new effective treatments. The collaboration utilizes Evotec’s integrated neuroscience platform and broad expertise in CNS drug discovery and development. It covers a wide range of biology and chemistry services supported by compound and library management, target validation, stem-cell research, high-content screening, computational chemistry, pharmacology, protein production and biomarker discovery and validation across multiple sites. Initiated in 2006, the long-standing collaboration is one of the largest strategic drug discovery alliances within Evotec.

Evotec, Inserm, Lille University Hospital and Inserm Transfert Enter Collaboration to Identify Novel Therapeutic Targets in Obesity and Metabolic Diseases

Princeton-based BioNJ Member Evotec SE announced that the company entered a partnership with Inserm, the French National Institute of Health and Medical Research, Lille University Hospital and Inserm Transfert to identify novel therapeutic targets and diagnostic and prognostic markers in obesity and metabolic diseases. Lille University Hospital is the sponsor of the clinical study entitled ABOS / DIABOMICS (Biological Atlas of Severe Obesity). In the framework of this study, Lille University Hospital develops and maintains the ABOS Biobank composed of a biobank and different associated databases dedicated to the study of obesity and its comorbidities. The strategic partnership between Evotec, Inserm, Lille University Hospital and Inserm Transfert aims at identifying novel multi-modality therapeutic targets. Evotec will gain access to a large patient cohort with severe obesity for the longitudinal assessment of metabolic outcomes after bariatric surgery-induced weight loss.

“LEQEMBI” (Lecanemab) Approved for the Treatment of Alzheimer’s Disease in South Korea

Nutley-based BioNJ Member Eisai Co., Ltd. and BioNJ Member Biogen Inc. announced that the Ministry of Food and Drug Safety (MFDS) in South Korea has approved humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody “LEQEMBI” (lecanemab) for treatment in adult patients with mild cognitive impairment due to Alzheimer’s disease (AD) or mild AD (early AD). LEQEMBI selectively binds to soluble Aβ aggregates (protofibrils*), as well as insoluble Aβ aggregates (fibrils) which are a major component of Aβ plaques in AD, thereby reducing both Aβ protofibrils and Aβ plaques in the brain. LEQEMBI is the first and only approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline through this mechanism. South Korea is the fourth country to grant approval, following approvals in the U.S., Japan and China.

Eisai Named to List of the TIME 100 Most Influential Companies

Eisai Co. Ltd announced that TIME has selected Nutley-based BioNJ Member Eisai as one of the TIME100 Most Influential Companies of 2024. TIME reveals the TIME100 Most Influential Companies list, highlighting companies making an extraordinary impact around the world. Then TIME editors evaluated each company based on key factors, including impact, innovation, ambition and success. Eisai is recognized in the “Pioneers” category for its human health care concept of prioritizing patient needs and the development of our Alzheimer’s disease treatment LEQEMBI® (lecanemab). LEQEMBI is the first and only treatment approved in Japan, the United States, China, and South Korea shown to reduce the rate of disease progression and to slow cognitive and functional decline, that acts on the underlying pathology of AD.

Johnson & Johnson to Obtain Rights to a Clinical-Stage Bispecific Antibody to Address Distinct Patient Needs in Atopic Dermatitis

New Brunswick-based BioNJ Member Johnson & Johnson announced it has entered into a definitive agreement with Numab Therapeutics, a clinical-stage biotechnology company advancing a pipeline of immunology and oncology therapeutics, to acquire from Numab’s shareholders its wholly-owned subsidiary for the global rights to a novel, investigational first-in-class bispecific antibody, NM26, in an all-cash transaction of approximately $1.25 billion. NM26, which is ready to enter Phase 2 studies, targets two clinically proven pathways, IL-4R alpha subunit (IL-4Rα) and IL-31, in atopic dermatitis (AD). Atopic dermatitis, the most common inflammatory skin disease, is highly heterogeneous with different disease-driving mechanisms in distinct patient subpopulations. NM26 targets IL-4Rα, which triggers Th2-mediated skin inflammation4, and IL-31, which impacts skin itch and subsequent scratching that worsen the disease.

Johnson & Johnson Pivotal Study of Seltorexant Shows Statistically Significant and Clinically Meaningful Improvement in Depressive Symptoms and Sleep Disturbance Outcomes

Titusville-based BioNJ Member Johnson & Johnson announced today positive topline results from the pivotal Phase 3 MDD3001 clinical trial evaluating the efficacy and safety of seltorexant as an adjunctive treatment to baseline antidepressants in adult and elderly patients with major depressive disorder (MDD) with insomnia symptoms. Seltorexant is an investigational first-in-class selective antagonist of the human orexin-2 receptor being studied for the adjunctive treatment of MDD with insomnia symptoms. The Phase 3 randomized, double-blind, multicenter, placebo-controlled study achieved all primary and secondary endpoints, with seltorexant demonstrating both a statistically significant and clinically meaningful improvement in depressive symptoms based on the Montgomery-Asberg Depression Rating Scale (MADRS) total score at day 43, and improved sleep disturbance outcomes, in patients who had a prior inadequate response to SSRI/SNRI antidepressants alone.

First Results from Late-Breaking Phase 3 PALOMA-3 Study Show Five-Fold Reduction in Infusion-Related Reactions With Five-Minute Subcutaneous Amivantamab Administration

New Brunswick-based BioNJ MemberJohnson & Johnson announced first data from the Phase 3 PALOMA-3 study evaluating subcutaneous (SC) amivantamab combined with lazertinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion (ex19del) or L858R mutations. Study results showed non-inferior efficacy and pharmacokinetics for SC amivantamab combined with lazertinib compared to intravenous (IV) administration, the currently approved formulation of RYBREVANT® (amivantamab-vmjw). Administration time for SC amivantamab was reduced to approximately five minutes from five hours (across two days) and showed a five-fold reduction in infusion-related reactions (IRRs). Results showed SC amivantamab was non-inferior to IV amivantamab, meeting both co-primary pharmacokinetic (PK) efficacy endpoints as measured by amivantamab levels in the blood (Ctrough and area under the serum concentration time curve from day 1 to 15).

RYBREVANT® (amivantamab-vmjw) Plus Lazertinib is the Only Chemotherapy-Free Regimen Showing Longer Progression-Free Survival Versus Osimertinib in First-Line Treatment of Patients With High-Risk EGFR-Mutated Non-Small Cell Lung Cancer

New Brunswick-based BioNJ Member Johnson & Johnson announced new data from the Phase 3 MARIPOSA study demonstrating the benefit of first-line treatment with RYBREVANT® (amivantamab-vmjw) in combination with lazertinib in patients with high-risk disease or clinical features, which occur in nearly 85 percent of patients with non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) mutations. Results from the new analysis show the RYBREVANT® combination consistently and significantly improved progression-free survival (PFS) compared to osimertinib in patients with NSCLC with EGFR exon 19 deletion (ex19del) or L858R mutations. The MARIPOSA study enrolled treatment-naïve patients with EGFR-mutant (ex19del or L858R) advanced NSCLC. Overall, results showed RYBREVANT® plus lazertinib resulted in a significant reduction in the risk of disease progression or death compared to osimertinib as previously reported.

CARVYKTI® (ciltacabtagene autoleucel) Significantly Improved Progression-Free Survival and Deepened Responses Versus Two Standard Therapies for Patients With Functional High-Risk Multiple Myeloma

New Brunswick-based BioNJ Member Johnson & Johnson announced results from a subgroup analysis of the Phase 3 CARTITUDE-4 study. The data show CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) significantly improved progression-free survival (PFS) compared to standard therapies of pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) for patients with lenalidomide-refractory multiple myeloma after one prior line of therapy (LOT), including patients with functional high-risk (FHR) multiple myeloma. FHR was defined as progressive disease within 18 months after receiving autologous stem cell transplant (ASCT) or the start of initial frontline therapy in patients with no ASCT. Data from the CARTITUDE-4 study supported the recent U.S. FDA approval of CARVYKTI®, the first and only B-cell maturation antigen (BCMA)-targeted therapy approved for the treatment of patients with relapsed/refractory multiple myeloma as early as after first relapse.

Johnson & Johnson Submits Supplemental New Drug Application to U.S. FDA Seeking Expanded Pediatric Indication for HIV-1 Therapy PREZCOBIX®

Titusville-based BioNJ Member Johnson & Johnson announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking to expand the indication of PREZCOBIX® (darunavir/cobicistat) to include the treatment of HIV-1 infection in younger children at least 6 years of age weighing at least 25 kg. A parallel line extension application and type 2 variation application have also been submitted to the European Medicines Agency (EMA) for expanded pediatric use in Europe, where the product is marketed as REZOLSTA®. If the applications are approved, PREZCOBIX®/REZOLSTA® could be administered to adults and pediatric patients at least 6 years of age, weighing at least 25kg. A new co-formulated tablet containing a weight-adjusted pediatric dose (darunavir 675 mg/cobicistat 150 mg) has been developed to aid administration for younger children.

DARZALEX® (daratumumab)-Based Regimens Significantly Improve Clinical Outcomes in Both Transplant-Eligible and -Ineligible Patients Who are Newly Diagnosed With Multiple Myeloma

New Brunswick-based BioNJ Member Johnson & Johnson announced data from the Phase 3 PERSEUS study showing deepening of responses and sustained minimal residual disease (MRD) negativity at both 10-5 and 10-6 levels with an induction regimen of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone (D-VRd) followed by a maintenance regimen of DARZALEX FASPRO® plus lenalidomide (D-R) for the treatment of patients with transplant-eligible (TE) newly diagnosed multiple myeloma (NDMM). The rates of deep and sustained MRD negativity were associated with improved progression-free survival (PFS) with DARZALEX FASPRO®-based quadruplet induction, consolidation and doublet maintenance regimen in these patients versus VRd. 

Bristol Myers Squibb Receives European Commission Approval for Opdivo® (nivolumab) in Combination With Cisplatin and Gemcitabine for the First-Line Treatment of Adult Patients With Unresectable or Metastatic Urothelial Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Commission (EC) has approved Opdivo® (nivolumab) in combination with cisplatin and gemcitabine for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC). With this approval, Opdivo in combination with cisplatin and gemcitabine becomes the first concurrent immunotherapy-chemotherapy approved for the treatment of adult patients with unresectable or metastatic UC in the first-line setting in the European Union. The EC’s decision is based on results from the CheckMate -901 trial studying Opdivo in combination with cisplatin and gemcitabine. In CheckMate -901, Opdivo in combination with cisplatin and gemcitabine followed by Opdivo monotherapy demonstrated statistically significant and clinically meaningful improvements in the primary efficacy endpoints of overall survival (OS) and progression-free survival (PFS) compared to chemotherapy alone, as assessed by Blinded Independent Central Review (BICR).

KRAZATI (adagrasib) Demonstrated Statistically Significant Improvement in Progression-Free Survival in Patients With Pretreated Locally Advanced or Metastatic KRASG12C-Mutated Non-Small Cell Lung Cancer

Princeton-based BioNJ Member Bristol Myers Squibb (announced results from the Phase 3 KRYSTAL-12 study evaluating KRAZATI ® (adagrasib) compared to standard of care chemotherapy in patients with locally advanced or metastatic KRASG12C -mutated non-small cell lung cancer (NSCLC) who had previously received platinum-based chemotherapy, concurrently or sequentially with anti-PD-(L)1 therapy. At a median follow-up of 9.4 months, KRAZATI demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS), the study’s primary endpoint, as assessed by Blinded Independent Central Review (BICR) compared to docetaxel (HR: 0.58; [95% CI, 0.45-0.76]; P <0.0001). Median PFS was 5.5 months for KRAZATI compared to 3.8 months for docetaxel. Overall response rate (ORR) as assessed by BICR was also significantly higher with KRAZATI compared to docetaxel (32% vs 9%; odds ratio, 4.68; P < 0.0001).

Bristol Myers Squibb’s Breyanzi Demonstrates Clinically Meaningful Outcomes Across Broad Range of B-Cell Malignancies in New Data Presented at 2024 ASCO® Annual Meeting

Princeton-based BioNJ Member Bristol Myers Squibb announced data from three studies evaluating Breyanzi® (lisocabtagene maraleucel; liso-cel), including long-term data with three-year follow-up from the Phase 3 TRANSFORM trial of Breyanzi as a second-line treatment in patients with relapsed or refractory large B-cell lymphoma (LBCL), results from a subgroup analysis evaluating the efficacy and safety of Breyanzi by number of prior lines of therapy in the mantle cell lymphoma (MCL) cohort of the TRANSCEND NHL 001 trial, and results from a subgroup analysis assessing the efficacy and safety of Breyanzi based on use of bridging therapy in the TRANSCEND FL trial in relapsed or refractory follicular lymphoma (FL).

Bristol Myers Squibb Announces Opdivo (nivolumab) Plus Yervoy (ipilimumab) Significantly Improved Overall Survival Compared to Lenvatinib or Sorafenib as First-Line Treatment for Patients With Advanced Hepatocellular Carcinoma in CheckMate -9DW Trial

Princeton-based BioNJ Member Bristol Myers Squibb announced the first presentation of results from the Phase 3 CheckMate -9DW trial evaluating the dual immunotherapy combination of Opdivo® (nivolumab) plus Yervoy® (ipilimumab) compared to investigator’s choice of lenvatinib or sorafenib as a first-line treatment for patients with unresectable hepatocellular carcinoma (HCC). The safety profile for the combination of Opdivo plus Yervoy remained consistent with previously reported data and was manageable with established protocols. Treatment-related adverse events (TRAEs) of any grade were reported in 84% of patients with Opdivo plus Yervoy and 91% in patients with lenvatinib or sorafenib. Grade 3/4 TRAEs occurred in 41% and 42% of patients, respectively.

Sarclisa Accepted for FDA Priority Review for the Treatment of Transplant-Ineligible Newly Diagnosed Multiple Myeloma

The U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for the investigational use of Bridgewater-based BioNJ Member Sanofi’s Sarclisa (isatuximab) in combination with bortezomib, lenalidomide and dexamethasone (VRd) for the treatment of patients with transplant-ineligible newly diagnosed multiple myeloma (NDMM). If approved, Sarclisa would be the first anti-CD38 therapy in combination with standard-of-care VRd in newly diagnosed patients not eligible for transplant, which would be the third indication for Sarclisa in multiple myeloma. The target action date for the FDA decision is September 27, 2024. A regulatory submission is also under review in the European Union (EU).

Sarclisa is First Anti-CD38 to Significantly Improve Progression-Free Survival in Combination With VRd for Newly Diagnosed Transplant-Ineligible Multiple Myeloma in Phase 3

Data from Bridgewater-based BioNJ Member Sanofi’s IMROZ Phase 3 study demonstrated Sarclisa (isatuximab) in combination with standard-of-care bortezomib, lenalidomide and dexamethasone (VRd) followed by Sarclisa-Rd (the IMROZ regimen) significantly reduced the risk of disease progression or death by 40%, compared to VRd followed by Rd in patients with newly diagnosed multiple myeloma (NDMM) not eligible for transplant. IMROZ is the first global phase 3 study of an anti-CD38 monoclonal antibody in combination with standard-of-care VRd to significantly improve PFS and show deep responses in this patient population who often have poor prognoses. The use of Sarclisa in combination with VRd in transplant-ineligible NDMM is investigational and has not been fully evaluated by any regulatory authority.

Sanofi Completes Acquisition of Inhibrx, Inc.

Bridgewater-based BioNJ Member Sanofi announced the completion of its acquisition of Inhibrx, Inc. The acquisition adds SAR447537 (formerly INBRX-101) to Sanofi’s rare disease pipeline, underscoring the company’s commitment to pursuing differentiated and potential best-in-class medicines that build upon our existing strengths and capabilities. SAR447537 is a human recombinant protein that holds the promise of allowing alpha-1 antitrypsin deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent (monthly vs. weekly) dosing. AATD is an inherited rare disease characterized by low levels of AAT protein, predominantly affecting the lung with progressive deterioration of the tissue. SAR447537 may help to reduce inflammation and prevent further deterioration of lung function in affected individuals.

Dupixent Recommended for EU Approval by the CHMP to Treat Patients With COPD

Bridgewater-based BioNJ Member Sanofi announced the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the approval of Dupixent (dupilumab) in the European Union (EU) as an add-on maintenance treatment in adults with uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood eosinophils. The European Commission is expected to announce a final decision on the Dupixent application in the coming months. COPD is a respiratory disease that damages the lungs and causes progressive lung function decline and is the fourth leading cause of death worldwide. Symptoms include persistent cough, excessive mucus production and shortness of breath that may impair the ability to perform routine daily activities, which may lead to sleep disturbances, anxiety and depression. 

Update on FDA Priority Review of Dupixent for the Treatment of COPD Patients With Type 2 Inflammation

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) has extended by three months the target action date of its priority review of the supplemental Biologics License Application (sBLA) for Dupixent (dupilumab) as an add-on maintenance treatment in certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD). The revised target action date is September 27, 2024. The FDA did not raise any concerns regarding the approvability of Dupixent for this indication. The FDA had requested additional efficacy analyses on the efficacy of Dupixent in the BOREAS and NOTUS pivotal trials. Based on the submission of these analyses earlier in May, the agency has now determined that this additional information constituted a major amendment to the sBLA and extended the target action date accordingly.

Lilly, UNICEF expand support to help millions of young people at risk of noncommunicable diseases

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced it will donate $6.5 million to the United States Fund for UNICEF to expand UNICEF's work to improve the health outcomes of millions of children and youth at risk of noncommunicable diseases (NCDs) living in resource-limited settings in India. This will bring the company's total commitment since 2022 to more than $20 million. With this additional funding, UNICEF will seek to improve efforts to address NCD risk factors, strengthen health systems, enhance the ability of health workers to care for people and support millions of children and their families challenged by diseases like type 1 diabetes, respiratory illnesses, rheumatic and congenital heart disease and sickle cell disease in resource-limited areas in India. 

Updated Data from the Phase 1/2 Study of Olomorasib in KRAS G12C-Mutant Advanced Solid Tumors Presented at the 2024 ASCO® Annual Meeting

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced updated data from the Phase 1/2 clinical trial evaluating olomorasib as a monotherapy in patients with KRAS G12C-mutant advanced solid tumors and in combination with Merck's anti-PD-1 therapy KEYTRUDA® (pembrolizumab) in patients with KRAS G12C-mutant advanced non-small cell lung cancer (NSCLC). Olomorasib is an investigational, oral, potent and highly selective second-generation inhibitor of the KRAS G12C protein. "These data show efficacy with olomorasib across tumor types and, importantly, tolerability that suggests it can be combined with immunotherapy, the backbone of first-line treatment for KRAS-mutant NSCLC," said Timothy Burns, M.D., Ph.D., Associate Professor of Medicine, University of Pittsburgh Medical Center Hillman Cancer Center.

Pfizer’s LORBRENA® CROWN Study Shows Majority of Patients With ALK-Positive Advanced Lung Cancer Living Beyond Five Years Without Disease Progression

BioNJ Member Pfizer, with a site in Peapack, announced longer-term follow-up results from the Phase 3 CROWN trial evaluating LORBRENA® (lorlatinib, a third-generation ALK inhibitor, available in Europe under the brand name LORVIQUA®) versus XALKORI® (crizotinib) in people with previously untreated, anaplastic lymphoma kinase (ALK)-positive advanced non-small cell lung cancer (NSCLC). After five years of median follow-up, median progression-free survival (PFS) based on investigator assessment was not reached with LORBRENA, with an observed Hazard Ratio (HR) of 0.19 (95% Confidence Interval [CI], 0.13-0.27), representing an 81% reduction in the rate of disease progression or death compared to XALKORI. Further, 60% of patients treated with LORBRENA (95% CI, 51-68) were alive without disease progression after five years compared to 8% (3-14) on the XALKORI treatment arm.

Pfizer and Takeda Announce Four-Year Results from Positive Phase 3 HD21 Trial of Additional ADCETRIS® (brentuximab vedotin) Combination in Frontline Hodgkin Lymphoma

BioNJ Member Pfizer, with a site in Peapack, and Takeda announced that the German Hodgkin Study Group (GHSG) will present positive results from the Phase 3 HD21 trial evaluating ADCETRIS® (brentuximab vedotin) in combination with chemotherapy. The four-year analysis presented by the GHSG showed superior progression-free survival (PFS) and improved tolerability for patients compared to a current standard of care regimen used in Europe in this setting. The HD21 study is a Phase 3, randomized, multi-country, prospective, open-label study, sponsored by the GHSG and supported by Takeda, designed to evaluate ADCETRIS in combination with etoposide, cyclophosphamide, doxorubicin, dacarbazine and dexamethasone (BrECADD) in comparison to a standard of care treatment – escalated doses of bleomycin, etoposide, doxorubicin, cyclophosphamide, vincristine, procarbazine, prednisone (eBEACOPP) – in patients with newly diagnosed Stage IIb/III/IV classical Hodgkin lymphoma. 

Pfizer’s ADCETRIS® Regimen Produces Clinically Meaningful Improvement in Overall Survival in Patients With Relapsed/​Refractory Diffuse Large B-cell Lymphoma (DLBCL)

BioNJ Member Pfizer, with a site in Peapack, announced detailed overall survival (OS) results from the Phase 3 ECHELON-3 study of ADCETRIS® (brentuximab vedotin) in combination with lenalidomide and rituximab for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). The study showed that the ADCETRIS combination reduced patients’ risk of death by 37% compared to placebo in combination with lenalidomide and rituximab (HR 0.63 [95% CI: 0.445-0.891] p=0.0085). The overall survival benefit was consistent across levels of CD30 expression. “ECHELON-3 is one of the first randomized, placebo-controlled Phase 3 studies to demonstrate an overall survival benefit in patients with relapsed/refractory DLBCL after two or more prior lines of systemic therapy,” said principal investigator Dr. Jeung-A Kim, College of Medicine, The Catholic University of Korea.

Gilead Announces New England Journal of Medicine Publication of Data that Demonstrate Bulevirtide With PegIFN Achieved Post-Treatment Undetectable HDV RNA

BioNJ Member Gilead Sciences, with a site in Morris Plains, announced data from the Phase 2b MYR204 open-label study assessing the efficacy and safety of the first-in-class entry inhibitor bulevirtide as monotherapy and in combination with pegylated interferon alfa-2a (PegIFN), in adults living with compensated chronic hepatitis delta virus (HDV) infection. Published in the New England Journal of Medicine (NEJM), the data demonstrate that the investigational combination of bulevirtide 10 mg with PegIFN was superior to investigational bulevirtide 10 mg monotherapy in achieving undetectable HDV RNA (lower limit of quantification (LLOQ), target not detected) at Week 24 after the end of treatment (EOT). The end of study data demonstrate that treatment with bulevirtide 10 mg in combination with PegIFN maintained a 46% rate of undetectable HDV RNA at Week 48 after EOT, confirming its potential as a finite therapy for adults living with chronic HDV. HDV affects an estimated 5% of people living with chronic hepatitis B (HBV), with a global prevalence of more than 12 million people.

Gilead’s Seladelpar Demonstrated a Sustained and Consistent Long-Term Efficacy and Safety Profile in Primary Biliary Cholangitis

BioNJ Member Gilead Sciences, with a site in Morris Plains, following the recent acquisition of CymaBay Therapeutics, Inc., announced two-year interim results from the ongoing ASSURE study of investigational seladelpar for the treatment of primary biliary cholangitis (PBC), a rare, chronic inflammatory liver disease. The two-year interim analysis includes people living with PBC who participated in any prior clinical studies of seladelpar (legacy studies) and participants from the pivotal Phase 3 RESPONSE study. Results demonstrated rapid and sustained improvements in markers of cholestasis, including high rates of normalization of liver biomarkers and a clinically meaningful reduction in pruritus (itch).

Gilead and Arcus Announce Anti-TIGIT Domvanalimab Plus Zimberelimab and Chemotherapy Exceeded One Year of Median Progression-Free Survival as a First-Line Treatment for Upper GI Cancers

BioNJ Member Gilead Sciences, with a site in Morris Plains, and Arcus Biosciences, Inc. announced longer-term efficacy and safety results from Arm A1 of the Phase 2 EDGE-Gastric study. These updated data show consistent objective response rate (ORR) and provide mature progression-free survival (PFS) in patients with locally advanced unresectable or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma (upper GI cancers). The ongoing, multi-arm, global Phase 2 EDGE-Gastric study is evaluating the safety and efficacy of various combinations of the Fc-silent anti-TIGIT antibody domvanalimab plus the anti-PD-1 monoclonal antibody zimberelimab and chemotherapy in this patient population. At data cutoff (DCO, March 12, 2024), safety and efficacy were evaluated in all patients enrolled and treated (n=41).

Gilead and Arcus Announce Etrumadenant Plus Zimberelimab Regimen Significantly Reduced the Risk of Death in Third-line Metastatic Colorectal Cancer

BioNJ Member Gilead Sciences, with a site in Morris Plains, and Arcus Biosciences, Inc. announced new data from Cohort B of ARC-9, a Phase 1b/2 study evaluating the safety and efficacy of etrumadenant, a dual A2a/b adenosine receptor antagonist, plus anti-PD-1 monoclonal antibody zimberelimab, FOLFOX chemotherapy and bevacizumab (EZFB) in third-line metastatic colorectal cancer (mCRC). Cohort B of ARC-9 randomized 112 patients with comparable baseline characteristics between two arms: EZFB or regorafenib. At the time of data cut-off (November 13, 2023) median follow-up was 20.4 months. Patient baseline characteristics were similar to those of third-line patients who have progressed on oxaliplatin- and irinotecan-based regimens in mCRC.

Gilead Sciences Advances Enrollment in Collaborative Studies to Assess Twice-Yearly HIV Prevention Option for Both Cisgender Women in the U.S. and People Who Inject Drugs in the U.S.

BioNJ Member Gilead Sciences, with a site in Morris Plains, announced the company is advancing enrollment in two groundbreaking collaborative studies to evaluate the tolerability of an investigational long-acting HIV prevention option in groups of people in the United States who are disproportionately affected by HIV but who are often underrepresented in HIV clinical trials. The Phase 2 studies, PURPOSE 3 (HPTN-102, NCT06101329) and PURPOSE 4 (HPTN-103, NCT06101342), are part of Gilead’s PURPOSE program. The PURPOSE program, which also includes two ongoing Phase 3 clinical trials, is assessing the potential of twice-yearly subcutaneous injections of lenacapavir to help a diverse range of people around the world who could benefit from HIV.

Boehringer Ingelheim’s COPD and Asthma Inhalers are Now Available for $35 a Month for Eligible Patients

BioNJ Member Boehringer Ingelheim, with a site in North Brunswick, Boehringer Ingelheim’s new program to cap out-of-pocket costs at $35 per month for eligible patients for the company’s inhalers is now available. With this program, patients who have had difficulty navigating the current healthcare system will now be able to afford the Boehringer inhalers they need. Starting today, the reduced out-of-pocket cost will be automatically applied at participating retail pharmacies for eligible patients with commercial insurance. More than 90% of pharmacies across the country are participating in the program, making the change seamless and automatic for most patients. There are no forms to fill out or websites to go to—the discount happens electronically with no action required.

Teva Announces AUSTEDO® XR (deutetrabenazine) Extended-Release Tablets Now U.S. FDA Approved as a One Pill, Once-Daily Treatment Option for Clinically Therapeutic Doses (24–48 mg/day)

Parsippany-based BioNJ Member Teva Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved AUSTEDO XR as a one pill, once-daily treatment option, now with four new tablet strengths (30, 36, 42, 48 mg) indicated in adults for TD and HD chorea. Currently more than 57 million Americans are living with a mental illness, 14 million of whom are living with a serious mental health condition. For those taking certain mental health medications, one in four may experience the onset of TD, an often-overlooked chronic movement disorder that can have a physical, emotional and psychological impact on patients. HD is a fatal neurodegenerative disease characterized by cognitive deterioration, behavior and/or psychological problems and uncoordinated and uncontrollable movements known as chorea – a symptom that affects 90% of patients.

Teva Presents First Real-World Data from the IMPACT-TD Registry Study at Psych Congress Elevate 2024

Parsippany-based BioNJ Member Teva Pharmaceuticals announced new patient- and physician-reported interim results from the Phase 4 IMPACT-TD Registry study, reinforcing that TD has a wide-reaching, multidimensional impact on patients’ quality of life. The two-part IMPACT-TD study is a 3-year longitudinal observational study evaluating how TD progresses over time and impacts a patient’s quality of life (Part A), as well as outcomes related to treatment with once-daily AUSTEDO XR and twice-daily AUSTEDO (Part B). Clinician-reported TD severity and impact is assessed using the IMPACT-TD scale, the AIMS (Abnormal Involuntary Movement Scale) and the CGIS-TD (Clinical Global Impression of Severity of TD) scale. The interim analysis includes 286 patients with varying levels of TD severity and highlights, for the first time, that clinicians reported that TD has a multidimensional impact, even on patients with mild TD severity.

New Data Provide Schizophrenia Treatment Insights into Switching to UZEDY® (risperidone) Extended-Release Injectable Suspension from Invega Sustenna® (paliperidone palmitate)

Parsippany-based BioNJ Member Teva Pharmaceuticals announced the presentation of seven studies from its long-acting injectable (LAI) schizophrenia research program. Presentations include data informing clinical strategies for switching patients to UZEDY, an extended-release injectable suspension of risperidone for subcutaneous use every one or two months for the treatment of schizophrenia in adults, from a once-monthly intramuscular injection of Invega Sustenna. In a population pharmacokinetic (PopPK) analysis, simulations were performed to predict PK exposures when switching to UZEDY 4 - 6 weeks at steady state after the last injection of once-monthly paliperidone palmitate. Model simulations showed that switching to UZEDY at four weeks after the last dose of once-monthly paliperidone palmitate yielded generally higher PK parameters, both within the total active moiety range for oral risperidone.

Blenrep Combination Reduced the Risk of Disease Progression or Death by Nearly 50% Versus Standard of Care Combination in Relapsed/Refractory Multiple Myeloma

Warren-based BioNJ Member GSK plc announced positive results from an interim analysis of the DREAMM-8 Phase III head-to-head trial evaluating Blenrep (belantamab mafodotin), in combination with pomalidomide plus dexamethasone (PomDex), versus a standard of care, bortezomib plus PomDex, as a second line and later treatment for relapsed or refractory multiple myeloma. On the primary endpoint of progression-free survival (PFS), a statistically significant and clinically meaningful improvement (hazard ratio [HR]: 0.52 [95% confidence interval (CI): 0.37-0.73], p-value<0.001) was observed with the belantamab mafodotin combination (n=155) compared to the bortezomib combination (n=147). At a median follow-up of 21.8 months, the median PFS was not yet reached (95% CI: 20.6-not yet reached [NR]) with the belantamab mafodotin combination compared to 12.7 months (95% CI: 9.1-18.5) in the bortezomib combination. 

Jemperli (dostarlimab) Trial Continues to Show Unprecedented Results With No Evidence of Disease in 100% of Patients With Locally Advanced Mismatch Repair Deficient (dMMR) Rectal Cancer

Warren-based BioNJ Member GSK plc announced updated, longer-term results from the Phase II supported collaborative study with Memorial Sloan Kettering Cancer Center (MSK) evaluating Jemperli (dostarlimab) as a first-line treatment—as an alternative to surgery—for mismatch repair deficient (dMMR) locally advanced rectal cancer. The trial showed an unprecedented 100% clinical complete response rate (cCR) in 42 patients who completed treatment with dostarlimab, defined as complete pathologic response or no evidence of tumours as assessed by magnetic resonance imaging, endoscopy and digital rectal exam. In the first 24 patients evaluated, a sustained clinical complete response with a median follow-up of 26.3 months (95% CI: 12.4-50.5) was observed.

AbbVie Advances Oncology Pipeline With Start of Multiple Myeloma Phase 3 Clinical Trial for Investigational Asset ABBV-383

BioNJ Member AbbVie, with a site in Madison, announced that the first patient has been treated with investigational ABBV-383 in the CERVINO Phase 3 study. ABBV-383 is a distinctive B-cell maturation antigen (BCMA) and CD3 bispecific antibody T-cell engager composed of bivalent BCMA-binding domains allowing for high BCMA-avidity and a low-affinity CD3 binding domain. ABBV-383 is being evaluated in a Phase 3, multicenter, randomized, open-label study compared with standard available therapies in patients with r/r MM who received at least two lines of prior therapy. Multiple myeloma is a blood cancer characterized by abnormal proliferation of plasma cells, which can cause end-organ damage and is the second most commonly occurring blood cancer in the world. An estimated 176,000 people globally were diagnosed with multiple myeloma in 2020, and 117,000 people died from the disease.

RINVOQ® (upadacitinib) Now Available for Pediatric Patients Two Years and Older With Polyarticular Juvenile Idiopathic Arthritis and Psoriatic Arthritis

BioNJ Member AbbVie, with a site in Madison, announced that RINVOQ® (upadacitinib) is indicated in the U.S. for the treatment of pediatric patients two years of age and older with active polyarticular juvenile idiopathic arthritis (pJIA) as well as psoriatic arthritis (PsA), provided they have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers. Additionally, a new weight-based oral solution, RINVOQ® LQ (upadacitinib), is now available as an option for these pediatric populations. Nearly 300,000 children and adolescents in the U.S. have a form of juvenile idiopathic arthritis, which includes pJIA and PsA. The polyarticular form of juvenile idiopathic arthritis is characterized by inflammation in five or more joints that persists for at least six weeks in children and adolescents before 16 years of age. 

Vyluma Announces Marketing Authorization Application Validation for the European Union

Bridgewater-based Vyluma, Inc. announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for its lead compound, NVK002. The application is confirmed to be eligible for a Pediatric Use Marketing Authorization (PUMA), providing 10 years of data exclusivity and marketing protection upon product approval. The regulatory application of NVK002 is supported by previously reported safety and efficacy data from the multi-center, placebo-controlled, Phase III CHAMP (Childhood Atropine Myopia Progression) clinical study which evaluated the safety and efficacy of the product in children aged three years and older.

Datopotamab Deruxtecan Showed Clinically Meaningful Overall Survival Improvement Versus Chemotherapy in Patients With Advanced Nonsquamous Non-Small Cell Lung Cancer in TROPION-Lung01 Phase 3 Trial

Basking Ridge-based Daiichi Sankyo announced topline overall survival (OS) results from the TROPION-Lung01 Phase 3 trial, which previously met the dual primary endpoint of progression-free survival (PFS), numerically favored datopotamab deruxtecan (Dato-DXd) compared to docetaxel in the overall trial population of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) treated with at least one prior line of therapy. Survival results did not reach statistical significance in the overall trial population. In the pre-specified subgroup of patients with nonsquamous NSCLC, datopotamab deruxtecan showed a clinically meaningful improvement in OS compared to docetaxel, the current standard of care chemotherapy. 

ENHERTU® Demonstrated a Median Progression-Free Survival of 13.2 Months in HR Positive, HER2 Low and HER2 Ultralow Metastatic Breast Cancer Following One or More Lines of Endocrine Therapy

Basking Ridge-based Daiichi Sankyo announced detailed positive results from the DESTINY-Breast06 Phase 3 trial showed that ENHERTU® (trastuzumab deruxtecan) demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to standard of care chemotherapy in patients with HR positive, HER2 low (IHC 1+ or IHC 2+/ISH-) metastatic breast cancer and the overall trial population (patients with HR positive, HER2 low and HER2 ultralow [defined as IHC 0 with membrane staining] expression) following one or more lines of endocrine therapy. 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.

NS Pharma Shares Preliminary Results of Viltolarsen (NS-065 / NCNP-01) Phase 3 Clinical Trial (RACER53 Study)

Paramus-based NS Pharma, Inc. announced that it has received preliminary analysis results from the global Phase 3 clinical trial (RACER53 study, NCT04060199) of NS-065/NCNP-01 (generic name: viltolarsen). Viltolarsen was approved by the United States (US) Food and Drug Administration (FDA) in 2020 under the brand name VILTEPSO® – for the treatment of Duchenne muscular dystrophy (Duchenne) in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping – under the FDA accelerated approval pathway based on an increase in dystrophin production in skeletal muscle observed in treated patients. In the US, continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Ipsen Presents Long-erm Elafibranor Efficacy and Itch-Related Quality of Life Data in Patients With Primary Biliary Cholangitis 

Ipsen, with a site in Basking Ridge, announced new data demonstrating the enduring efficacy of elafibranor in managing disease progression after 78 weeks of treatment. In the variable double-blind period of the ELATIVE® Phase III trial in primary biliary cholangitis (PBC), the potential for elafibranor to improve itch-related quality of life (QoL) as measured by the itch domain of the PBC-40 and the 5-D Itch questionnaire was also demonstrated. Elafibranor is a novel, potential first-in-class, PPAR agonist. It is currently under review by the U.S. Food and Drug Administration, the European Medicines Agency and the UK Medicines and Healthcare Products Regulatory Authority. 

Johnson & Johnson Completes Acquisition of Shockwave Medical

BioNJ Member Johnson & Johnson said it completed its acquisition of Shockwave Medical. Shockwave is now part of the New Brunswick-based health care giant and will operate as a business unit within Johnson & Johnson MedTech. Shockwave’s differentiated solutions and robust pipeline represent an opportunity for Johnson & Johnson MedTech to bring more innovations to patients in one of the largest areas of unmet medical need, as it further extends Johnson & Johnson MedTech’s leadership in cardiovascular intervention. Shockwave offers the first and only commercially available intravascular lithotripsy platform for coronary artery disease and peripheral artery disease and complements Johnson & Johnson’s leading positions in heart recovery and electrophysiology to make it a category leader in four of the largest and highest-growth medtech markets within cardiovascular intervention.

BD to Acquire Edwards Lifesciences’ Critical Care Business for $4.2B

As part of a move to extend its reach in patient monitoring, Franklin Lakes-headquartered medtech Becton, Dickinson and Co. plans to acquire Edwards Lifesciences’ smart connected care business. BD announced the $4.2 billion all-cash deal. Under the transaction, it will take over the critical care unit. The segment develops and sells advanced blood and heart monitoring systems used in surgical and intensive care settings. It will operate as a separate unit within BD’s medical segment, led by Katie Szyman. BD said Szyman has served as corporate vice president of critical care since 2015. BD Chairman, CEO and President Tom Polen commented, “Critical care expands BD’s portfolio of smart connected care solutions with its growing set of leading monitoring technologies, advanced AI-enabled clinical decision tools and robust innovation pipeline that complement BD’s existing technologies serving operating rooms and intensive care units.”

34 Innovative Companies Awarded Benefits Through NJEDA’s Technology Business Tax Certificate Transfer (NOL) Program in 2023

The New Jersey Economic Development Authority (NJEDA) announced that 34 technology and life sciences companies were awarded more than $68 million through the 2023 Technology Business Tax Certificate Transfer Program, commonly known as the Net Operating Loss (NOL) program. The NOL program allows early stage technology and life sciences companies in New Jersey to sell a percentage of their net operating losses and unused research and development (R&D) tax credits to unrelated profitable corporations for cash. The NJEDA opened applications for the 2024 NOL program on May 1, and the application will close at 11:59 p.m. on Sunday, June 30. 

Parkinson's Disease Therapeutics Pipeline Program

This program seeks to advance therapeutic development through pre-clinical and/or clinical testing of approaches addressing unmet needs of people with Parkinson’s disease (PD). The program is set up to benefit therapeutics with clear potential to prevent, stop, or delay disease progression or to reduce the burden of daily symptoms. The goal of this program is to increase the chances of moving therapies toward the clinic. To expedite the process, only industry and academia-industry partnerships in and outside the United States are welcome to apply. Applicants are permitted to submit multiple unique submissions but should NOT submit multiple pre-proposals supporting different stages or aspects of the same therapeutic development program. Applicants are also permitted and encouraged to re-submit a revised pre-proposal that addresses prior feedback provided by MJFF, if applicable. 

Melissa Seymour to Join Lilly as Executive Vice President of Global Quality

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that Melissa Seymour will join the company as executive vice president of Global Quality and member of the company's Executive Committee, effective July 22, 2024. Ms. Seymour currently serves as the Chief Quality Officer for Bristol Myers Squibb. Ms. Seymour is recognized as one of the foremost quality leaders in the pharmaceutical industry. She has held senior leadership roles at global pharmaceutical companies, including Bristol Myers Squibb and Biogen, with extended experience at Novo Nordisk and Glaxo Smith Kline. She has led the development of quality compliance strategies, implemented quality processes and systems and developed talent to ensure the highest level of quality and compliance in the pharmaceutical industry.

Y-mAbs Deepens Radiopharmaceutical Leadership with Appointment of Norman LaFrance, M.D. as Chief Development Officer

BioNJ Member Y-mAbs Therapeutics, Inc., with a site in Nutley, announced the appointment of Norman LaFrance, M.D. as Chief Development Officer. Dr. LaFrance brings over 40 years of deep radiopharmaceutical experience as both a nuclear medicine physician and pharmaceutical executive. He previously served as the Chief Medical Officer, Senior Vice President of PLUS Therapeutics, Inc., a U.S. clinical-stage pharmaceutical company where he led the development of targeted radiotherapeutics with advanced platform technologies for central nervous system (CNS) cancers. Before Plus Therapeutics, Dr. LaFrance served as Chief Medical Officer at Jubilant Pharma Ltd., where he was responsible for all Pharmaceutical & Medical Regulatory Affairs activities. Prior to Jubilant, Dr. LaFrance served as Global Chief Medical Officer at IBA Molecular and earlier, as Senior Vice President and Chief Medical Officer of Molecular Insight Pharmaceuticals, Inc.

Dr. Reddy’s Laboratories Appoints Milan Kalawadia as CEO, North America

Princeton-based Dr. Reddy’s Laboratories Ltd. said it appointed Milan Kalawadia as CEO, North America, and member of its Management Council. Mr. Kalawadia will be responsible for the company’s North America business and will be based out of the Princeton office. “I am excited to start this next chapter in my journey at Dr. Reddy’s,” Mr. Kalawadia said. “I have been fortunate to be a part of a remarkable team that has achieved significant milestones over the last 18 years. I now look forward to leveraging the relationships I have built over my years within the organization, as well as the industry connections I have made, to continue to lead the North America division to new heights.”

UroGen Pharma Appoints David Lin as New Chief Commercial Officer

Princeton-based UroGen Pharma Ltd. announced that David Lin will join UroGen as its Chief Commercial Officer and member of the Executive Leadership Team. In this role, Mr. Lin will be spearheading UroGen’s commercial strategy, including preparation for the potential launch of our lead pipeline candidate UGN-102, if approved, and driving the continued growth and commercialization of JELMYTO® (mitomycin) for pyelocalyceal solution. Mr. Lin brings to UroGen a wealth of experience garnered from a distinguished career in the pharmaceutical industry. Prior to joining UroGen, he held several key leadership positions at Bristol Myers Squibb, where he played pivotal roles in driving the success of various initiatives. Notably, as Head of U.S. Cell Therapy, Mr. Lin led the successful launch of two CAR T therapies, showcasing his expertise in bringing innovative therapies to market. 

Otsuka Pharmaceutical and Taiho Pharmaceutical Commence Material Recycling of Waste PTP Used in Pharmaceutical Packaging

Princeton-based BioNJ Members Otsuka Pharmaceutical and Taiho Pharmaceutical announced that they have started a material recycling initiative for press-through packaging (PTP) waste generated during pharmaceutical packaging. The initiative commenced in May 2024.This material recycling initiative seeks to reduce environmental impact by recycling PTP waste generated by Otsuka and Taiho. It utilizes technology to separate PTP sheets for packaging tablets and capsules into plastic and aluminum foil and includes a modal shift to rail transport, thereby helping to reduce CO2 emissions. Based on the 2050 Environmental Vision "Net Zero", the Otsuka group aims to reduce the total environmental impact of its business activities to zero. In line with this vision, the group has identified carbon neutrality, circular economy and water neutrality as material issues and is implementing initiatives to achieve these goals.

View Otsuka's Pharmaceutical's Environmental Initiatives

View Taiho Pharmaceutical's Environmental Initiatives

Bayer's Expert Mondays 2024

Select Mondays in March – December 2024

Calling pre-seed and seed innovators and entrepreneurs in Oncology, Cell & Gene Therapy, Cardiovascular, Immunology, Radiology and Digital Health! Do you have disruptive science but struggle to bring it to life in the entrepreneurial world? Bayer's passion is to enable and support standout healthcare innovation! Take your innovation to new heights and join Bayer´s Expert Mondays 2024, a series of knowledge exchange sessions to supercharge your start-up’s growth. 

Propelus I-Corps Regional Program

May 31 & June 14 – Princeton University

The NSF I-Corps Hub Northeast Hub is accepting applications for a two-day, in-person educational program for tech-based entrepreneurs. The two-day intensive educational program, to be held May 31 and June 14 at Princeton University, is aimed at helping entrepreneurs gain a better understanding of the startup process. It is intended to help researchers develop early stage innovations into real-world products and services through training, funding, resources and follow-on opportunities.

Deal Making and Structuring Early Stage Technology and Life Sciences Deals

June 12 – LES NY/NJ Chapter

A panel discussion on the challenges and opportunities associated with technology transfer and licensing from universities to corporates and between emerging companies and more established companies. This program is for University tech transfer professionals, early stage life sciences and technology companies, late-stage companies that recently acquired or in licensed IP originated at early stage companies.

Decentralization = Innovation: The Case Against Reinstating Government Management of Federally-Funded Tech

June 27 – Webinar

The Bayh-Dole Coalition will host a webinar to discuss how the Bayh-Dole Act’s decentralization of patent licensing facilitated a technological and economic revolution — and how a recent Biden administration proposal imperils this system. Prior to the Bayh-Dole Act, the government retained any patents resulting from taxpayer-funded research. And it licensed fewer than 5% of them for further research and development. The Bayh-Dole Act gave universities, non-profit research institutions, and startups the ability to retain and license their own patents. Thanks to this decentralization of the licensing process, and the incentives created by the law, roughly 60% of federally funded patents are now licensed for additional development.