PODCAST: The Faceless Middleman Between You and Your Doctor

A troubling disconnect between medical expertise and insurance mandates is putting patients' health at risk. Policies like step therapy and prior authorization are forcing individuals into treatments not recommended by their doctors — often with devastating consequences. In this episode, we hear from a patient who endured months of suffering due to her insurer's 'fail-first' requirements. We also talk with a doctor navigating this impossible system and a biotech leader fighting to ensure innovative treatments reach the patients who need them most. 

Sanofi Tackles 340B Abuse with Innovative Credit Model

Sanofi’s commitment to chase the miracles of science to improve people’s lives only matters if people can access and afford the medicines they need. That is why we are proud to support critical safety net programs and deliver our own patient support programs to help lower costs for patients. One of the largest safety net programs designed to help some of the most vulnerable people in our communities is the 340B Drug Pricing Program. Created in 1992, 340B was created by Congress to provide better and affordable healthcare to low-income and uninsured patients at hospitals and health clinics that serve a disproportionate number of these vulnerable patients. In exchange for delivering better and affordable care, those 340B covered entities were given the ability to purchase outpatient medicines at significantly reduced prices with the expectation that they use the savings to help those most in need.

Unleashing America’s Entrepreneurs: The Bayh-Dole Formula for Economic Prosperity

The Bayh-Dole Coalition hosted a webinar to discuss how the Bayh-Dole Act plays a crucial role in bolstering the U.S. economy and our international competitiveness. Prior to the Bayh-Dole Act, the federal government retained all patents resulting from taxpayer-funded research — and it licensed less than 5% of those inventions. The Bayh-Dole Act, in turn, empowered universities and other research institutions to license and commercialize their innovations originating from federal funding. Now, about 30% of federally-funded patents are licensed for additional development. In the last two decades alone, Bayh-Dole-generated innovation has contributed $1 trillion to the U.S. GDP, supported 6.5 million jobs, and helped launch over 17,000 start-ups. International competitors have adopted legislation modeled after Bayh-Dole in attempts to rival the United States’ position as an innovation and economic leader.

PODAST: Impact of the Inflation Reduction Act on Venture Capital in Life Sciences

In this Vital Health Podcast, John Stanford, Executive Director of Incubate, discusses the impact of the Inflation Reduction Act (IRA) on venture capital investments in life sciences. Stanford emphasizes that the IRA's price control mechanisms have led to significant shifts in funding, particularly away from small-molecule drug development, creating what is described as the "small molecule penalty.

New Jersey’s Deadliest Bug Isn’t An Insect

Op-Ed Featured in NJ Spotlight by Debbie Hart, President & CEO, BioNJ

A record 384 New Jersey residents contracted a deadly fungal pathogen last year. The fungus, Candida auris, kills about a third of its victims. And it’s just one of many drug-resistant superbugs wreaking havoc across the Garden State — and across the entire world, for that matter. Antibiotic resistance contributes to roughly 170,000 U.S. deaths annually, and the toll grows each year. According to a new analysis, antibiotic resistance is forecast to cause more than 39 million global deaths between 2025 and 2050. That’s more than four times the entire population of New Jersey. Alarmingly, these pathogens are evolving to resist even our strongest antimicrobials. Soon, we may not have any way of combating these infections — unless biopharmaceutical companies are able to develop new medicines. To do so, those companies will need some help from Congress.

Biotechs Give Back With BioBears

As Featured in New Jersey Business Magazine

BioNJ, a trade association that represents the life sciences industry in the state, held its inaugural “BioNJ Gives Back” event this past Tuesday, in which some 200 employees from various biotechnology firms in the State gathered at two separate locations to build BioBears. The colorful animals, complete with superhero capes, masks, birth certificates and personalized notes from builders, will be distributed to pediatric patients at Children’s Specialized Hospital facilities throughout New Jersey this holiday season. The idea for the event emanated from last February’s BioNJ Annual Dinner Meeting and Innovation Celebration where Will Lewis, Chair and CEO of Bridgewater-based Insmed, former BioNJ chair and the recipient of the 2024 Dr. Sol J. Barer Award, delivered a call to action for the biotech industry to set aside a community day, similar to what Insmed itself has been doing for a number of years, and give back through volunteering.

Health Care Sector Gears Up for the Second Trump Administration

As Featured in NJBIZ

Following Donald Trump’s 2024 election win to secure a second term in office, sectors such as health care are gauging what his victory will mean for their industry over the next four years — and beyond. BioNJ is New Jersey’s life sciences trade association, dedicated to discovering, developing and delivering life-changing medicines that bring hope to patients and their families worldwide. “During the last Trump administration, BioNJ worked closely with federal partners to advance policies that foster innovation, support patients and sustain our industry’s global leadership,” said Debbie Hart, BioNJ President and CEO. “We are committed to continuing this collaboration with the incoming administration and Congress to address the challenges facing our ecosystem, drive breakthrough innovation and ensure that every patient has timely access to effective treatments and that U.S. economic competitiveness and national security are bolstered. Because Patients Can’t Wait.”

Rare Disease Specialist Insmed Continues Rapid Growth In NJ

As Featured in NJBIZ

Insmed is a biopharmaceutical company that develops and commercializes drugs for serious and rare diseases – advancing a diverse portfolio of approved and mid- to late-stage investigational medicines as well as cutting-edge drug discovery focused on serving the most pressing patient needs. Its most advanced programs are in pulmonary and inflammatory conditions, including a therapy approved in the U.S., Europe and Japan to treat a chronic, debilitating lung disease. The company is led by Will Lewis, who joined Insmed in 2012. Under his watch, the company has grown from a 30-person biotech company to a more than 1,200-person emerging global biopharmaceutical company with a nearly $13 billion market cap. BioNJ President and CEO Debbie Hart told NJBIZ that Lewis has built Insmed into one of the most dynamic and inspiring biopharmaceutical companies of our time. “An organization that is truly transforming the lives of people with serious and rare diseases. His unwavering commitment to putting Patients first extends beyond Insmed, influencing the broader biopharma landscape. Whether driving Patient-focused innovation, mentoring future leaders or enhancing trust and transparency across the industry, Will’s vision and passion are felt far and wide."

PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington's Disease Program

Warren-based BioNJ Member PTC Therapeutics announced the signing of an exclusive global license and collaboration agreement with BioNJ Member Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG, for its PTC518 Huntington's disease program, which includes related molecules. Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales. "PTC518 is the leading oral disease-modifying therapy in development for Huntington's disease and the economics of this agreement are consistent with the promise of this treatment," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "This collaboration combines PTC's expertise in developing small molecule splicing therapies with Novartis's expertise in global development and commercialization of neuroscience therapies."

Longer-term Data for Novartis Scemblix® Reinforce Superior Efficacy With Favorable Safety and Tolerability Profile in Adults With Newly Diagnosed CML

East Hanover-based BioNJ Member Novartis announced positive, longer-term results from the pivotal Phase III ASC4FIRST trial with Scemblix® (asciminib) showing superior major molecular response (MMR) rates at week 96. The study compared the MMR rate of Scemblix to investigator-selected standard-of-care (SoC) tyrosine kinase inhibitors (TKIs) (imatinib, nilotinib, dasatinib and bosutinib) and to imatinib alone in adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) at the week 96 evaluation, the study’s key secondary endpoints. The longer-term results showed an increasing difference in Scemblix MMR rate vs. SoC, vs. imatinib and vs. 2G TKIs (nilotinib, dasatinib and bosutinib). The median follow-up was 2.2 years for Scemblix and investigator-selected SoC TKIs. 

European Commission Approves BeiGene’s TEVIMBRA for First-Line Treatment of Advanced/Metastatic Esophageal Squamous Cell Carcinoma and Gastric or Gastroesophageal Junction Cancer

Hopewell-based BioNJ Member BeiGene, a global oncology company that intends to change its name to BeOne Medicines, announced that the European Commission has approved TEVIMBRA® (tislelizumab) in combination with chemotherapy for the first-line treatment of esophageal squamous cell carcinoma (ESCC) and gastric or gastroesophageal junction (G/GEJ) adenocarcinoma. “Patients diagnosed with advanced gastric and esophageal cancers confront median survival times measured in months, not years—highlighting the urgent need for more effective treatment options,” said Prof. Florian Lordick, Director and Professor of Oncology of the University Cancer Center Leipzig, Germany. “The compelling data from the RATIONALE-305 and 306 trials underscore the unique clinical profile of tislelizumab and its potential to deliver meaningful improvements in outcomes for eligible patients, offering new hope where it’s needed most.” 

BeiGene and CLL Society Partner to Advance Test Before Treat™ Campaign, Promoting Biomarker Testing to Improve Patient Outcomes in CLL

Hopewell-based BioNJ Member BeiGene, a global oncology company that intends to change its name to BeOne Medicines Ltd., announced its new partnership with CLL Society, the world’s leading authority for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) cancer patients. Together, they are advancing Test Before Treat™, an awareness campaign that aims to connect with healthcare providers (HCPs) and educate patients with CLL/SLL about the critical importance of biomarker testing before initial treatment and retesting at each subsequent treatment to help ensure each patient receives the most effective, personalized therapy. Test Before Treat empowers CLL patients and HCPs to make informed, personalized treatment decisions through a comprehensive awareness initiative. 

Acadia Pharmaceuticals Announces Exclusive License Agreement With Saniona for SAN711

BioNJ Member Acadia Pharmaceuticals Inc., with a site in Princeton, announced it has entered into an exclusive worldwide license agreement with Saniona for the development and commercialization of SAN711, a first-in-class, highly selective GABAA-α3 positive allosteric modulator. The first indication the company plans to pursue is the development of SAN711 for essential tremor, a neurological condition that includes shaking or trembling movements in one or more parts of the body. Acadia is planning to initiate a Phase 2 study of SAN711 in essential tremor in 2026. “Licensing SAN711 to expand our pipeline underscores our unwavering commitment to delivering innovative therapies for patients with central nervous system disorders,” said Catherine Owen Adams, CEO. 

HyBryte™ Clinical Results Demonstrate Continued Improvement Post-Treatment

Princeton-based BioNJ Member Soligenix announced that analysis of the post-treatment data from the open-label study (protocol HPN-CTCL-04) comparing HyBryte™ (synthetic hypericin) to Valchlor® (mechlorethamine) has demonstrated continued improvement in HyBryte™ treated patients and their individual lesions even after stopping treatment. The study, which enrolled 10 patients randomized 1:1 with 12 weeks of treatment and 4 weeks of follow-up post-treatment, was previously reported to demonstrate a positive difference in the overall per patient treatment response rate (60% in the HyBryte™ group vs. 20% in the Valchlor® group) at the end of treatment. After the 4-week follow-up period (Week 16), the majority (3 of 5) of HyBryte™ patients continued to demonstrate improvement with at least a further 10% improvement (absolute difference) at Week 16 relative to the primary outcome measure at Week 12, including one of the HyBryte™ patients achieving a "complete response". 

Kura Oncology and Kyowa Kirin Announce Global Strategic Collaboration to Develop and Commercialize Ziftomenib in Acute Leukemias

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, and Kura Oncology, Inc. announced they have entered into a global strategic collaboration to develop and commercialize ziftomenib, Kura’s selective oral menin inhibitor, being investigated for the treatment of patients with acute myeloid leukemia (AML) and other hematologic malignancies. Under the terms of the agreement, Kura will receive an upfront payment of $330 million and expects to receive up to $420 million in near-term milestone payments, including a payment upon the launch of ziftomenib in the monotherapy relapsed/refractory (R/R) setting. In addition, Kura is eligible to receive additional development, regulatory and commercial milestone payments of $741 million, totaling up to $1.161 billion in payments for milestones and the opt-in for solid tumor indications.

Kyowa Kirin and Kura Oncology Report Positive Combination Data for Ziftomenib at American Society of Hematology Annual Meeting

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, and Kura Oncology, Inc. provided encouraging clinical data from KOMET-007, a Phase 1 dose-escalation trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care, including cytarabine/daunorubicin (7+3) and venetoclax/azacitidine (ven/aza), in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). Ziftomenib was generally well tolerated in combination at all dose levels evaluated across all cohorts in the Phase 1a dose-escalation portion of the study. No dose-limiting toxicities, evidence of ziftomenib-associated QTc prolongation, drug-drug interactions or additive myelosuppression were observed. In the 7+3 combination cohorts, on-target differentiation syndrome (DS) occurred in 2% (1/51) of patients.

Otsuka Provides Update on Sibeprenlimab, an Investigational Monoclonal Antibody for the Treatment of IgA Nephropathy in Adults

BioNJ Member Otsuka, with a site in Princeton, announce the decision to submit a Biologics License Application (BLA) in the U.S. for sibeprenlimab, an investigational drug for the treatment of immunoglobulin A nephropathy (IgA nephropathy) in adults, in the first half of 2025. The update follows a recent meeting with the U.S. Food & Drug Administration to discuss the positive interim analysis results from the Phase 3 VISIONARY study. Sibeprenlimab is an investigational, anti-APRIL monoclonal antibody (A PRoliferation-Inducing Ligand) that blocks a key initiating step in the immune pathogenic cascade of IgA nephropathy by limiting aberrant IgA1 (Gd-IgA1) production and immune complex formation. IgA nephropathy is a progressive, autoimmune, chronic kidney disease that can lead to end-stage kidney disease (ESKD) over the lifetime of most patients.

Zenyaku Kogyo Co., Ltd. Chugai Pharmaceutical Co., Ltd. Anti-CD20 Monoclonal Antibody Rituxan® Approved for Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children

BioNJ Member Chugai Pharmaceutical Co, with a site in Berkeley Heights, and Zenyaku Kogyo Co., Ltd. announced that Zenyaku obtained regulatory approval from the Ministry of Health, Labour and Welfare (MHLW), for an additional dosage and administration of an anti-CD20 monoclonal antibody Rituxan ® intravenous injection 100 mg and 500 mg [generic name: rituximab (genetical recombination)] (hereafter, “Rituxan”), which is co-marketed by both companies, for “chronic idiopathic thrombocytopenic purpura in children.” Chronic ITP had previously only been approved for use in adults with specified dosage and administration, and its use in children had not been approved. The Japanese Society of Pediatric Hematology/Oncology requested the addition of dosage and administration for Rituxan for “chronic ITP in children.”

European Commission Approves Pfizer’s HYMPAVZI™ (marstacimab) for the Treatment of Adults and Adolescents With Severe Hemophilia A or B Without Inhibitors

BioNJ Member Pfizer, with a site in Peapack, announced that the European Commission (EC) has granted marketing authorization for HYMPAVZI™ (marstacimab) for the routine prophylaxis of bleeding episodes in patients 12 years of age and older weighing at least 35 kg with severe hemophilia A (congenital factor VIII [FVIII] deficiency, FVIII <1%) without FVIII inhibitors or severe hemophilia B (congenital factor IX [FIX] deficiency, FIX <1%) without FIX inhibitors. HYMPAVZI is the first and only anti-tissue factor pathway inhibitor (anti-TFPI) approved in the European Union (EU) for the treatment of hemophilia A or B and the first hemophilia medicine approved in the EU to be administered via a pre-filled, auto-injector pen. HYMPAVZI offers a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.

Pfizer’s IBRANCE® in Combination with Standard-of-Care Therapies Extends Median Progression-Free Survival by Over 15 Months in Phase 3 PATINA Study in Patients with HR+, HER2+ Metastatic Breast Cancer

BioNJ Member Pfizer Inc., with a site in Peapack, and Alliance Foundation Trials, LLC (AFT) announced results from the Phase 3 PATINA trial demonstrating that the addition of IBRANCE® (palbociclib) to current standard-of-care first-line maintenance therapy (following induction chemotherapy) resulted in statistically significant and clinically meaningful improvement in progression-free survival (PFS) by investigator assessment in patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-positive (HER2+) metastatic breast cancer (MBC). In the study, which is sponsored by AFT, median PFS was 44.3 months (95% CI: 32.4-60.9) for patients treated with IBRANCE in combination with anti-HER2 therapy (trastuzumab or trastuzumab plus pertuzumab) and endocrine therapy, and 29.1 months (95% CI: 23.3-38.6) for patients treated with anti-HER2 therapy and endocrine therapy alone [HR: 0.74 (95% CI, 0.58-0.94); unstratified 1-sided p= 0.0074].

Tolebrutinib Designated Breakthrough Therapy by the FDA for Non-relapsing Secondary Progressive Multiple Sclerosis

Bridgewater-based BioNJ Member Sanofi announced the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to tolebrutinib for the treatment of adults with non-relapsing secondary progressive multiple sclerosis (nrSPMS). This is based on positive results from the HERCULES Phase 3 study, demonstrating that tolebrutinib delayed the time to onset of 6-month confirmed disability progression (CDP), by 31% compared to placebo (HR 0.69; 95% CI 0.55-0.88; p=0.0026), with further analysis of secondary endpoints demonstrating that the number of participants who experienced confirmed disability improvement was nearly double with tolebrutinib (10%) compared to those on placebo (5%) (HR 1.88; 95% CI 1.10 to 3.21; nominal p=0.021).

ASH: Rilzabrutinib Demonstrated Significant Patient Benefit in the First Positive Phase 3 Study of a BTK Inhibitor in ITP

Bridgewater-based BioNJ Member Sanofi announced positive results from the pivotal LUNA 3 Phase 3 study of rilzabrutinib in adults with persistent or chronic immune thrombocytopenia (ITP), a rare immune-mediated disease, reinforce the efficacy and safety of rilzabrutinib, an oral, reversible, covalent Bruton’s tyrosine kinase (BTK) inhibitor, and further support its potential as a first-in-class treatment for ITP. Platelet response was achieved in 65% (n=86) of patients receiving rilzabrutinib compared to 33% (n=23) of patients on placebo. The primary endpoint was met, with rilzabrutinib demonstrating durable platelet response in 23% of ITP adult patients compared to 0% on the placebo arm (p<0.0001), as well as key secondary endpoints including reduced bleeding, number of weeks with platelet response, the need for rescue therapy use, and improved physical fatigue and quality of life measures.

Two Combination Vaccine Candidates for Prevention of Influenza and COVID-19 Granted Fast Track Designation in the U.S.

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration has granted Fast Track designation to two Sanofi combination vaccine candidates to prevent influenza and COVID-19 infections in individuals 50 years of age and older. Both candidates combine two already licensed and authorized vaccines with proven efficacy through randomized controlled studies, and with favorable tolerability. The first combination vaccine candidate (NCT06695117) consists of the influenza protein-based trivalent vaccine Fluzone High-Dose combined with the adjuvanted recombinant Novavax COVID-19 vaccine. The second candidate (NCT06695130) combines the influenza recombinant protein-based trivalent vaccine Flublok with the Novavax COVID-19 vaccine. Both Fluzone High-Dose and Flublok have been proven to prevent more influenza infections in older adults than standard-dose influenza vaccines in pivotal randomized clinical studies.

Gilead and Tubulis Enter Into Exclusive Option and License Agreement to Develop ADC Candidate for Select Solid Tumor Target

BioNJ Member Gilead Sciences, with a site in Morris Plains, and Tubulis announced that they have entered into an exclusive option and license agreement to discover and develop an antibody-drug conjugate (ADC) against a solid tumor target. Through this agreement, Gilead will gain access to Tubulis’ proprietary Tubutecan and Alco5 platforms. The companies will collaborate to select the best technology to utilize, with Tubulis leading discovery and development efforts to design a Topoisomerase I inhibitor-based ADC candidate with superior biophysical properties and stability to address current treatment challenges such as durability and off-target toxicity. “With Gilead’s ongoing focus on innovating with next-generation therapies and combinations, we are excited to partner with Tubulis to explore a range of solutions that may help increase the therapeutic value of the ADC modality,” said Flavius Martin, MD, Executive Vice President, Research, Gilead Sciences.

Lilly's Omvoh® (mirikizumab) Recommended by CHMP for Approval in the European Union for Adults With Moderately to Severely Active Crohn's Disease

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 Omvoh® (mirikizumab), an interleukin-23p19 (IL-23p19) antagonist, for the treatment of adults with moderately to severely active Crohn's disease who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic treatment. Omvoh was previously approved in the European Union, U.S. and Japan in 2023 as a first-in-class treatment for adults with moderately to severely active ulcerative colitis (UC) and is approved in 44 countries around the world. This positive opinion marks the next step toward European regulatory approval of Omvoh for patients with moderately to severely active Crohn's disease, and it is now referred to the European Commission for final action.

Lilly's Zepbound® (tirzepatide) Superior to Wegovy® (semaglutide) in HHHHead-to-Head Trial Showing an Average Weight Loss of 20.2% vs. 13.7%

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced topline results from the SURMOUNT-5 Phase 3b open-label randomized clinical trial. Zepbound® (tirzepatide) provided a 47% greater relative weight loss compared to Wegovy® (semaglutide). On average, Zepbound led to a superior weight loss of 20.2% compared to 13.7% with Wegovy.i At 72 weeks, Zepbound beat Wegovy on both the primary endpoint and all five key secondary endpoints in this trial of adults living with obesity or overweight with at least one weight-related medical problem and without diabetes. In addition, in a key secondary endpoint, 31.6% of people taking Zepbound achieved at least 25% body weight loss compared to 16.1% of those taking Wegovy.

Phase 3 Results for Lilly's Jaypirca® (pirtobrutinib) in Covalent BTK Inhibitor Pre-Treated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma to be Presented at the 2024 ASH Annual Meeting

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced results from the Phase 3 BRUIN CLL-321 trial evaluating pirtobrutinib, a non-covalent (reversible) Bruton's tyrosine kinase (BTK) inhibitor in adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) previously treated with a covalent BTK inhibitor. The study's primary endpoint of progression-free survival (PFS) was met at primary analysis, demonstrating pirtobrutinib was superior to investigator's choice of idelalisib plus rituximab (IdelaR) or bendamustine plus rituximab (BR), based on independent review committee (IRC) assessment. The updated results corresponding to the final prespecified analysis, demonstrate consistent improvement in PFS for patients treated with pirtobrutinib, with a reduction in risk of relapse, disease or death by 46% compared to IdelaR or BR. 

Lilly's Imlunestrant, an Oral SERD, Significantly Improved Progression-Free Survival as Monotherapy and in Combination with Verzenio® (abemaciclib) in Patients with ER+, HER2- Advanced Breast Cancer

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced results from the Phase 3 EMBER-3 study of imlunestrant, an investigational, oral selective estrogen receptor degrader (SERD), in patients with estrogen receptor positive (ER+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer (ABC), whose disease progressed on a prior aromatase inhibitor (AI), with or without a CDK4/6 inhibitor. Imlunestrant demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) as monotherapy in patients with an ESR1 mutation versus standard of care endocrine therapy (SOC ET), reducing the risk of disease progression or death by 38%. Imlunestrant in combination with Verzenio (abemaciclib; CDK4/6 inhibitor) reduced the risk of progression or death by 43% versus imlunestrant alone, in all patients.

Teva Presents Positive Efficacy and Safety Data of AJOVY® (fremanezumab) for the Prevention of Episodic Migraine in Children and Adolescents from Phase 3 SPACE Trial

Parsippany-based BioNJ Member Teva Pharmaceutical presented positive data from its Phase 3 SPACE study evaluating the efficacy and safety of AJOVY® (fremanezumab) for the prevention of episodic migraine in children and adolescent patients aged 6-17 years. The trial showed statistically significant superior efficacy compared to placebo over 12 weeks with a favourable safety profile consistent with that observed in the adult population. Migraine is common among children, with an overall estimated prevalence of 7.7%. The prevalence increases from 5% among children aged 5 to 10 years-old to approximately 15% among adolescents. Migraine can cause significant disability in children and adolescents, leading to absence from school, impaired educational performance and missed social activities. SPACE is a multicentre, double-blind study evaluating the efficacy and safety of fremanezumab in 237 children and adolescents with episodic migraine aged 6-17 years. 

Teva Prolia® (Denosumab) Biosimilar Candidate is Accepted for Review by U.S. FDA and EU EMA

Parsippany-based BioNJ Member Teva Pharmaceutical announced that the United States (U.S.) Food and Drug Administration (FDA) has accepted, and the European Medicines Agency (EMA) has validated, applications for TVB-009P, a biosimilar candidate to Prolia® (denosumab). Both applications, a Biologics License Application (BLA) in the U.S., seeking interchangeability, and a Marketing Authorization Application (MAA) in the European Union (EU), include all indications approved for the reference product, Prolia, including conditions with a high risk for fracture, such as osteoporosis in postmenopausal women. The FDA’s anticipated decision and EMA’s expected opinion are anticipated in the second half of 2025. TVB-009P, Teva’s proposed biosimilar to Prolia, is the first of Teva’s internally developed biosimilars to be submitted to the U.S. FDA. 

Merck Receives Positive EU CHMP Opinion for WELIREG® (belzutifan) as Treatment for Adult Patients With Certain Types of Von Hippel-Lindau Disease-Associated Tumors and for Certain Previously Treated Adult Patients With Advanced Renal Cell Carcinoma

Rahway-based BioNJ Member Merck & Co. announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the conditional approval of WELIREG® (belzutifan), Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, as monotherapy for: the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated, localized renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), and for whom localized procedures are unsuitable; and the treatment of adult patients with advanced clear cell renal cell carcinoma (RCC) that progressed following two or more lines of therapy that included a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and at least two vascular endothelial growth factor (VEGF) targeted therapies.

Merck’s WELIREG® (belzutifan) Approved in China for the Treatment of Adult Patients With Certain Types of Von Hippel-Lindau (VHL) Disease-Associated Tumors

Rahway-based BioNJ Member Merck & Co. announced that the National Medical Products Administration (NMPA) in China has approved WELIREG ® (belzutifan), for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery. WELIREG is a first-in-class oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor and is the first and only approved HIF-2α inhibitor in China. This approval is based on objective response rate (ORR) and median duration of response (DOR) results from the Phase 2 LITESPARK-004 trial and is the 17th approval of WELIREG for these patients globally.

Merck and Ridgeback Biotherapeutics Announce Initiation of Phase 3 Study (MOVe-NOW) Evaluating LAGEVRIO™ (molnupiravir) for the Treatment of COVID-19 in High-Risk Adults

Rahway-based BioNJ Member Merck & Co. announced the initiation of the Phase 3 MOVe-NOW clinical trial to evaluate LAGEVRIO TM (molnupiravir), an investigational oral antiviral COVID-19 medicine, for the treatment of adults with COVID-19 at high risk for disease progression. This double-blind, placebo-controlled, global study is enrolling individuals who are at least 18 years of age, tested positive for SARS-CoV-2 infection, have had COVID-19 symptoms for four days or less, and are not hospitalized. Additionally, the study will only enroll adults who cannot receive nirmatrelvir/ritonavir (NMV/r) due to drug-drug interactions, allergy, previous adverse effects, or inaccessibility. The MOVe-NOW study will use a different formulation of LAGEVRIO that includes two smaller 400-mg tablets per dose (four daily tablets) instead of the currently available four 200-mg capsules per dose (eight daily capsules). 

Merck Announces Pivotal Phase 3 ZENITH Trial Evaluating WINREVAIR™ (sotatercept-csrk) Met Primary Endpoint at Interim Analysis

Rahway-based BioNJ Member Merck & Co. announced positive topline results from the Phase 3 ZENITH study evaluating WINREVAIR (sotatercept-csrk) in adults with pulmonary arterial hypertension (PAH, WHO* Group 1) functional class (FC) III or IV at high risk of mortality. ZENITH met its primary endpoint of time to first morbidity or mortality event (all-cause death, lung transplantation, or PAH worsening related hospitalization of ≥ 24 hours). In the study, WINREVAIR demonstrated a statistically significant and clinically meaningful reduction in the risk of morbidity or mortality events compared to placebo, both on top of background PAH therapy. Based on the strength of these results, an independent data monitoring committee has recommended ZENITH be stopped early and all participants be offered the opportunity to receive WINREVAIR through the SOTERIA open-label extension study. 

Merck Announces Phase 3 KEYLYNK-001 Trial Met Primary Endpoint of Progression-Free Survival (PFS) in Patients With Advanced Epithelial Ovarian Cancer

Rahway-based BioNJ Member Merck & Co. announced that the Phase 3 KEYLYNK-001 trial evaluating KEYTRUDA ® (pembrolizumab) plus chemotherapy followed by maintenance with LYNPARZA ® (olaparib), with or without bevacizumab, as a first-line treatment for people with BRCA non-mutated advanced epithelial ovarian cancer met its primary endpoint of progression-free survival (PFS). At the final analysis conducted by an independent Data Monitoring Committee, the KEYTRUDA plus LYNPARZA regimen demonstrated a statistically significant and clinically meaningful improvement in PFS for these patients compared to chemotherapy alone. “KEYLYNK-001 is the first positive Phase 3 trial for KEYTRUDA plus LYNPARZA, highlighting our commitment to research that may help address the global impact of women’s cancers,” said Dr. Gursel Aktan, Vice President, Global Clinical Development, Merck Research Laboratories.

FDA Grants Breakthrough Therapy Designation to Sacituzumab Tirumotecan (sac-TMT) for the Treatment of Certain Patients With Previously Treated Advanced or Metastatic Nonsquamous Non-Small Cell Lung Cancer With EGFR Mutations

Rahway-based BioNJ Member Merck & Co. announced that the FDA has granted Breakthrough Therapy designation to sacituzumab tirumotecan (sac-TMT) for the treatment of patients with advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) mutations (exon 19 deletion [19del] or exon 21 L858R) whose disease progressed on or after tyrosine kinase inhibitor (TKI) and platinum-based chemotherapy. Sac-TMT is an investigational trophoblast cell-surface antigen 2 (TROP2)-directed antibody drug conjugate (ADC) being developed in collaboration with Kelun-Biotech. This designation is based on data from the Phase 2 expansion cohort of a Phase 1/2 study evaluating sac-TMT in patients with EGFR-mutated NSCLC, as well as data from two parts of a Phase 2 study evaluating sac-TMT in patients with EGFR-mutated NSCLC who have been treated with at least two lines of prior therapy.

LYNPARZA® (olaparib) Demonstrated Clinically Meaningful Prolonged Survival Benefit in Early Breast Cancer in OlympiA Phase 3 Trial

Rahway-based BioNJ Member Merck & Co. and AstraZeneca announced long-term results from the OlympiA Phase 3 trial which showed LYNPARZA (olaparib) demonstrated sustained, clinically meaningful improvements in overall survival (OS), invasive disease-free survival (IDFS) and distant disease-free survival (DDFS) for people with germline BRCA-mutated (gBRCAm) HER2-negative high-risk early breast cancer. These results were published in The New England Journal of Medicine. Breast cancer is the second most diagnosed cancer worldwide, with an estimated 2.3 million patients diagnosed in 2022. About 63% of all breast cancer patients are diagnosed at an early stage of disease and BRCA mutations are found in approximately 5-10% of patients.

GSK and Zhifei Revise and Extend Strategic Vaccine Collaboration in China

Warren-based BioNJ Member GSK announced that it has entered into an agreement with Chongqing Zhifei Biological Products, Ltd. to revise the terms on which Zhifei will commercialize GSK’s shingles vaccine, Shingrix, in mainland China. The revised agreement extends the original 3-year period (2024-2026) during which Zhifei has exclusive rights to import, distribute and co-promote the vaccine in mainland China for an additional 8 years through to 2034, with revised expected volumes. Under the revised agreement, Zhifei also agrees to engage exclusively with GSK to explore a potential collaboration, with an initial term of 10 years, on the commercialization of a respiratory syncytial virus (RSV) vaccine in mainland China, subject to regulatory approval of the vaccine.

Blenrep Shows Significant Overall Survival Benefit, Reducing the Risk of Death by 42% in Multiple Myeloma at or After First Relapse

Warren-based BioNJ Member GSK announced statistically significant and clinically meaningful overall survival (OS) results from a planned interim analysis of the DREAMM-7 trial evaluating Blenrep (belantamab mafodotin) in combination with bortezomib plus dexamethasone (BVd) versus daratumumab in combination with bortezomib plus dexamethasone (DVd) as a second line or later treatment for relapsed or refractory multiple myeloma. The OS findings from DREAMM-7 build on previous data from the DREAMM-7 and DREAMM-8 trials, which showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) for both belantamab mafodotin-based combinations versus standard of care comparators. With a median follow up of 39.4 months, the analysis presented today shows a statistically significant 42% reduction in the risk of death among patients receiving the belantamab mafodotin combination (n=243) versus the daratumumab-based comparator (n=251) (HR 0.58; 95% CI: 0.43-0.79; p=0.00023).

Blenrep Combinations Accepted for Review by the U.S. FDA for the Treatment of Relapsed/Refractory Multiple Myeloma

Warren-based BioNJ Member GSK announced the U.S. Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for Blenrep (belantamab mafodotin) in combinations with bortezomib plus dexamethasone (BorDex [BVd]) and pomalidomide plus dexamethasone (PomDex [BPd]) for the treatment of patients with multiple myeloma who have received at least one prior line of therapy. Hesham Abdullah, Senior Vice President, Global Head Oncology, R&D, GSK, said, “Relapsed/refractory multiple myeloma treatment could be transformed by additional, efficacious treatment options with manageable side effects and community-based administration. The evidence from DREAMM-7 and DREAMM-8 supporting our Blenrep combinations submission has been further strengthened by the statistically significant overall survival results from the DREAMM-7 trial. We look forward to working with the FDA on this review.”

Blenrep (belantamab mafodotin) Combination Accepted for Priority Review in China in Relapsed/Refractory Multiple Myeloma

Warren-based BioNJ Member GSK announced that the National Medical Products Administration (NMPA) of China has accepted for review a new drug application (NDA) for Blenrep (belantamab mafodotin) in combination with bortezomib plus dexamethasone (BVd) as a treatment for relapsed or refractory multiple myeloma. Earlier this year, the NMPA granted priority review for this application as well as Breakthrough Therapy Designation for the BVd combination, which is intended to expedite development of investigational drugs with potential for substantial improvement over available therapies. This is the seventh major regulatory filing acceptance this year for belantamab mafodotin in combination for the treatment of relapsed or refractory multiple myeloma based on the results of the DREAMM-7 and DREAMM-8 trials. 

Arexvy Approval Expanded to Adults Aged 50-59 at Increased Risk of Severe RSV Disease in Japan

Warren-based BioNJ Member GSK announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved a regulatory application to extend the indication of Arexvy (respiratory syncytial virus vaccine, recombinant adjuvanted) for the prevention of RSV disease to include adults aged 50-59 at increased risk. Since September 2023, GSK’s RSV vaccine has been approved in Japan for adults aged 60 and over for the prevention of RSV disease.² Tony Wood, Chief Scientific Officer at GSK, said, "This approval reflects our ambition to protect people at increased risk from the severe consequences of RSV infection. Adults aged 50-59 with certain underlying medical conditions can face debilitating consequences from RSV, so we are pleased to offer those in Japan a vaccine for the first time.” 

U.S. FDA Accepts GSK’s Submission for the use of Nucala (mepolizumab) in COPD

Warren-based BioNJ Member GSK announced the FDA has accepted for review, data from the MATINEE study to support the regulatory review process to obtain a new indication for the use of Nucala (mepolizumab), as an add-on maintenance treatment for patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The submission is based on data from the MATINEE study, which evaluated the efficacy and safety of mepolizumab in 804 patients with COPD who have evidence of type 2 inflammation characterized by blood eosinophil count. The trial recruited COPD patients with broad clinical presentations including hard to treat patients with emphysema-only, chronic bronchitis only, or a mix of both. The MATINEE study met its primary endpoint with the addition of mepolizumab to inhaled maintenance therapy, achieving a statistically significant and clinically meaningful reduction in the annualized rate of moderate/severe exacerbations versus placebo with patients treated for 52-104 weeks.

GSK’s Fully Liquid Menveo Meningococcal Vaccine Approved by European Commission

Warren-based BioNJ Member GSK announced that the European Commission (EC) has approved a single-vial, fully liquid presentation of Menveo (Meningococcal Group A, C, W-135 and Y conjugate vaccine, MenACWY vaccine) to help protect against invasive meningococcal disease (IMD) caused by bacterial serogroups A, C, W and Y. This single-vial presentation is now licensed for active immunization of children from 2 years of age, adolescents and adults, offering healthcare providers an option that does not require reconstitution before its use. GSK’s submission to the EC was based on two positive Phase IIb trials. The primary and secondary outcomes of these trials, supported by post-hoc pooled analyses, show that the fully liquid formulation of this vaccine has comparable immunogenicity, tolerability and a comparable safety profile to the existing lyophilised/liquid formulation.

First Single-Dose Medicine for P. Vivax Malaria Prequalified by WHO and Included in WHO Guidelines

Warren-based BioNJ Member GSK and Medicines for Malaria Venture announced that the World Health Organization has awarded prequalification to tafenoquine, the first single-dose medicine for the prevention of relapse of Plasmodium vivax (P. vivax) malaria. Tafenoquine, co-administered with chloroquine, is now also included in WHO’s updated Guidelines for malaria, in South America, marking the first time the medicine has been recommended by WHO. This milestone is a significant step toward closing the treatment gap for P. vivax malaria. The WHO prequalification and updated guidelines include both adults and children aged 2 years and older, weighing at least 10 kg. A single-dose medicine provides an opportunity to overcome challenges with adherence to the existing longer, one-two week regimen of the standard of care, which can be a challenge for patients with relapsing malaria whose symptoms improve shortly after treatment initiation.

Two Data Analyses from Clinical Trials Show Epcoritamab (DuoBody® CD3xCD20) Induces Durable Complete Reponses As Monotherapy and Combination Treatment in Patients With Diffuse Large B-Cell Lymphoma

BioNJ Member AbbVie, with a site in Madison, announced new results from two ongoing clinical trials evaluating epcoritamab, a CD3xCD20 bispecific T-cell-engaging antibody administered subcutaneously, in adult patients with diffuse large B-cell lymphoma (DLBCL). Arm 1 of the Phase 1b/2 EPCORE® NHL-2 multi-arm trial evaluates fixed-duration investigational epcoritamab in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in untreated high-risk DLBCL patients (n=46) with International Prognostic Index (IPI) scores of 3 to 5. Results from this arm of the study showed an overall response rate (ORR) of 100% and a complete response (CR) rate of 87%. Among complete responders, an estimated 83% remained in remission after two years. 

Johnson & Johnson Seeks U.S. FDA Approval for Subcutaneous Induction Regimen of TREMFYA® (guselkumab) in Ulcerative Colitis, a First for an IL-23 Inhibitor

New Brunswick-based BioNJ Member Johnson & Johnson announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of a subcutaneous (SC) induction regimen of TREMFYA® (guselkumab) for the treatment of adults with moderately to severely active UC. The filing is supported by data from the Phase 3 ASTRO study of TREMFYA® SC induction therapy in adults with UC and builds upon the recent U.S. approval of TREMFYA® in this indication. The Phase 3 ASTRO study met its primary endpoint, achieving a statistically significant and clinically meaningful results for clinical remission at Week 12 with a 400 mg SC induction dose of TREMFYA® administered at Weeks 0, 4, and 8. All secondary endpoints, including endoscopic improvement and histologic-endoscopic mucosal improvement (HEMI), were also met.

Johnson & Johnson Seeks U.S. FDA Approval for First Pediatric Indications for TREMFYA® (guselkumab)

New Brunswick-based BioNJ Member Johnson & Johnson announced the submission of two supplemental Biologics License Applications (sBLAs) to the U.S. Food and Drug Administration (FDA) seeking approval of TREMFYA® (guselkumab) for the treatment of children 6 years and older with moderate-to-severe plaque psoriasis (PsO) and children 5 years of age and older with active juvenile psoriatic arthritis (jPsA). The PsO submission is based on data from the Phase 3 PROTOSTAR study in pediatric patients with moderate to severe plaque PsO and bridging pharmacokinetic (PK) data from the Phase 3 VOYAGE 1 and 2 studies in adult patients with moderate to severe plaque PsO. The jPsA submission is based on PK extrapolation analyses from adult PsA studies (DISCOVER 1 and 2) and TREMFYA efficacy and safety data from the PROTOSTAR study.

CARVYKTI® (ciltacabtagene autoleucel) Demonstrated Significantly Higher Rates of Minimal Residual Disease (MRD) Negativity Compared to Standard Therapies in the CARTITUDE-4 Study

New Brunswick-based BioNJ Member Johnson & Johnson announced new results from the Phase 3 CARTITUDE-4 study that show a single infusion of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) significantly increased minimal residual disease (MRD) negativity rates (10-5) in patients with relapsed or refractory multiple myeloma (RRMM) who were lenalidomide-refractory and had received one to three prior lines of therapy, including a proteasome inhibitor (PI), compared to standard therapies of pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd).[i] MRD is a prognostic marker of prolonged survival outcomes for patients with multiple myeloma. These results add to the overall survival (OS) benefits recently presented at the International Myeloma Society meeting earlier this year, as the first and only cell therapy to significantly extend OS versus standard therapies for patients with multiple myeloma.

DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj)-Based Regimens Demonstrate Improved Rates of Minimal Residual Disease (MRD) Negativity and Progression-Free Survival in Patients With Newly Diagnosed Multiple Myeloma

New Brunswick-based BioNJ Member Johnson & Johnson announced data highlighting that DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj)-based regimens improve overall and sustained minimal residual disease (MRD) negativity rates and progression-free survival (PFS) in patients with newly diagnosed multiple myeloma (NDMM), regardless of transplant status. These findings were demonstrated in an expanded MRD analysis of the Phase 3 CEPHEUS study and a post hoc analysis of clinically relevant subgroups in the Phase 3 AURIGA study. Data from the expanded MRD analysis of the Phase 3 CEPHEUS study show the addition of DARZALEX FASPRO® to bortezomib, lenalidomide and dexamethasone (D-VRd) leads to improved and deepened rates of overall and sustained MRD negativity (both 10-5 and 10-6 sensitivity thresholds in patients who achieved a complete response or better) versus VRd alone, and shows significantly improved progression-free survival.

DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) Shows 51 Percent Reduction in Risk of Progression to Active Multiple Myeloma for Patients With High-Risk Smoldering Multiple Myeloma

New Brunswick-based BioNJ Member Johnson & Johnson announced data from the Phase 3 AQUILA study showing that DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) significantly delayed progression from high-risk smoldering multiple myeloma (SMM) to active multiple myeloma (MM) and extended overall survival compared to the current standard of care of active monitoring. In the AQUILA study, 194 patients received DARZALEX FASPRO® and 196 patients were actively monitored per current standard of care treatment for high-risk SMM. At a median follow-up of 65.2 (range, 0-76.6) months, patients who received DARZALEX FASPRO® showed statistically significant improved progression-free survival (PFS; defined as progression to active MM as assessed according to International Myeloma working Group (IMWG) diagnostic criteria for MM [SLiM-CRAB] or death) than the active monitoring group; 63.1 percent versus 40.8 percent of patients remained progression-free at 60 months (hazard ratio [HR], 0.49; 95 percent confidence interval [CI], 0.36-0.67; P<0.001). 

TECVAYLI® (teclistamab-cqyv) Demonstrates Potential as Frontline Combination Therapy for Patients With Newly Diagnosed Multiple Myeloma

New Brunswick-based BioNJ Member Johnson & Johnson announced new frontline data featuring TECVAYLI® (teclistamab-cqyv) from two investigational studies in patients with newly diagnosed multiple myeloma (NDMM) in induction and maintenance settings. The MajesTEC-5 (Abstract #493) and MajesTEC-4 (Abstract #494) studies establish the potential of TECVAYLI® for use in newly diagnosed patients, with promising efficacy and a tolerable safety profile. These data were highlighted as oral presentations at the 2024 American Society of Hematology (ASH) Annual Meeting. Forty-nine patients with transplant-eligible NDMM were treated with TECVAYLI® in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj), lenalidomide and dexamethasone (Tec-DRd) or DARZALEX FASPRO®, bortezomib, lenalidomide and dexamethasone (Tec-DVRd) as induction therapy in the MajesTEC-5 study. 

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

Nutley-based BioNJ Member Eisai and Biogen Inc. announced that the humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody “LEQEMBI®” has been launched in South Korea. LEQEMBI received the Ministry of Food and Drug Safety (MFDS) approval in May 2024 for treatment in adult patients with mild cognitive impairment due to Alzheimer’s disease (AD) or mild AD dementia (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 approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline through this mechanism.

Evotec SE Announces First Projects for LAB eN² Drug Discovery Accelerator With Novo Nordisk

Princeton-based Evotec announced that its translational drug discovery accelerator with BioNJ Member Novo Nordisk, LAB eN², which aims to nurture early research from academic institutions into novel therapeutics, has selected its first three projects to move forward in the program from Boston University, Harvard University in collaboration with Mass General Brigham and Joslin Diabetes Center. LAB eN² is also expanding to include five additional academic institutions: Boston Children’s Hospital, Boston University, Johns Hopkins University, Joslin Diabetes Center, and the Icahn School of Medicine at Mount Sinai. “We are thrilled to be able to start work on these first projects, leveraging Evotec’s integrated drug discovery and translational platforms and Novo Nordisk’s deep disease understanding,” said Dr Thomas Hanke, EVP & Head of Academic Partnerships at Evotec.

Ipsen and Biomunex Announce Exclusive Global Licensing Agreement for First-in-Class MAIT Cell Engager in Immuno-Oncology

Ipsen, with a site in Basking Ridge, and Biomunex Pharmaceuticals announced an exclusive global licensing agreement for BMX-502. BMX-502 is a bispecific antibody that engages and activates a subset of cytotoxic T cells called Mucosal-Associated Invariant T cells (MAIT cells) and targets the GPC3 tumor antigen, to kill cancer cells. GPC3 is a clinically validated target, highly expressed across several cancer types. MAIT cells are present throughout the body, highly enriched in many specific tissues of the body, particularly in mucosal and barrier tissues. BMX-502 offers a promising approach to treating tumors in these tissues. Developed using Biomunex’s proprietary BiXAb technology, BMX-502 selectively engages MAIT cells and leverages their unique properties to maximize anti-tumor activity.

QuANTUM-Wild Phase 3 Trial of VANFLYTA® Initiated in Patients With Newly Diagnosed FLT3-ITD Negative AML

The first patient has been dosed in the QuANTUM-Wild Phase 3 trial evaluating Basking Ridge-based Daiichi Sankyo’s VANFLYTA® (quizartinib) in combination with standard intensive induction and consolidation chemotherapy followed by single-agent maintenance in adults with newly diagnosed FLT3-ITD negative acute myeloid leukemia (AML). AML is an aggressive blood cancer with a five-year overall survival rate of approximately 32%. Targeted therapy with FLT3 inhibitors has improved survival for some patients with FLT3 gene mutations, which most commonly occur as FLT3-ITD. However, about 90% of patients with AML overexpress FLT3 regardless of mutational status. No FLT3 inhibitors are currently approved for patients without FLT3 mutations. The QuANTUM-Wild trial was initiated based on results of the QUIWI phase 2 trial evaluating VANFLYTA in combination with standard intensive chemotherapy and as subsequent maintenance monotherapy in adult patients with newly diagnosed FLT3-ITD negative AML.

Datopotamab Deruxtecan Demonstrated Meaningful Clinical Activity in Patients With Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer in TROPION-Lung05 and TROPION-Lung01 Pooled Analysis

Basking Ridge-based Daiichi Sankyo announced a pooled analysis of the TROPION-Lung05

Phase 2 and the TROPION-Lung01 Phase 3 trials showed datopotamab deruxtecan (Dato-DXd) demonstrated clinically meaningful tumor response in patients with previously treated advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC). Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca. Datopotamab deruxtecan demonstrated a confirmed objective response rate (ORR) of 42.7% (95% confidence interval [CI]: 33.6-52.2) in a pooled analysis of 117 patients with EGFR-mutated NSCLC from the TROPION-Lung05 (n=78) and TROPION-Lung01 (n=39) trials, as assessed by blinded independent central review (BICR).

Datopotamab Deruxtecan Granted Breakthrough Therapy Designation in U.S. for Patients With Previously Treated Advanced EGFR-Mutated Non-Small Cell Lung Cancer

Basking Ridge-based Daiichi Sankyo announced Datopotamab deruxtecan (Dato-DXd) has been granted Breakthrough Therapy Designation in the U.S. for the treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFR-mutated) non-small cell lung cancer (NSCLC) with disease progression on or after treatment with an EGFR tyrosine kinase inhibitor (TKI) and platinum-based chemotherapy. Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed by Daiichi Sankyo and AstraZeneca. The U.S. Food and Drug Administration BTD is designed to accelerate the development and regulatory review of potential new medicines that are intended to treat serious conditions and address significant unmet medical needs. The medicine needs to have shown encouraging preliminary clinical results that demonstrate substantial improvement on a clinically significant endpoint over available medicines.

New Data from a Long-Term Follow-up Study to the OLYMPUS Trial Show Median Duration of Response of Four Years in Patients Who Achieved a Complete Response with JELMYTO®

Princeton-based UroGen Pharma Ltd. announced results from a long-term follow-up study with JELMYTO (mitomycin) for pyelocalyceal solution, which is FDA approved for the treatment of low-grade, upper tract urothelial cancer (LG-UTUC) in adult patients. Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41, 20 of whom entered the long-term follow-up study), the median duration of response was 47.8 months (median follow-up 28.1 months [95% CI 13.1, 57.5]). The study results are published online in the Journal of Urology. Of the 71 patients enrolled in OLYMPUS, 41 achieved a complete response after treatment with JELMYTO and had a median duration of response of 47.8 months (95% CI 13.0, not estimable), with median follow-up of 28.1 months (95% CI 13.1, 57.5).

Galderma’s Phase III OLYMPIA 1 Data Published in JAMA Dermatology Demonstrate that Nemolizumab Improves Core Signs and Symptoms of Prurigo Nodularis

Galderma, with a site in Cranbury, announced that full results from the Phase III OLYMPIA 1 trial, a 24-week study which evaluated the efficacy and safety of nemolizumab monotherapy in adults with moderate-to-severe prurigo nodularis, were published in JAMA Dermatology. The trial met both primary and all key secondary endpoints, showing that nemolizumab-treated patients had significantly higher improvements in itch and skin lesions when compared to those receiving placebo at Week 16, with a rapid and clinically meaningful response on itch and sleep disturbance observed as early as Week 4. Nemolizumab was well-tolerated, and its safety profile was generally consistent with previous studies. The phase III OLYMPIA 1 trial enrolled 286 adult patients with moderate-to-severe prurigo nodularis.

Idorsia Enters into Exclusive Negotiations for Global Rights to Aprocitentan

Idorsia Ltd, with a site in Cherry Hill, announced that it has entered into exclusive negotiations with an undisclosed party for the global rights to aprocitentan. Idorsia will receive an exclusivity fee of USD 35 million, which extends Idorsia’s cash runway into 2025. The potential agreement under discussion could include an upfront payment, additional milestone payments, and tiered royalties on sales in return for the transfer of global rights to aprocitentan and certain team members from Idorsia. André C. Muller, CEO of Idorsia, commented, “I am pleased to announce that we made an important step to reaching a potential agreement for the global rights to aprocitentan. This is a first and crucial step in our plan to put Idorsia in a financially sustainable position and on the road towards profitability.”

Dr. Reddy's Launches Toripalimab in India, the First and Only Immuno-Oncology Drug Approved for the Treatment of Nasopharyngeal Carcinoma

Princeton-based Dr. Reddy’s Laboratories announced the launch of Toripalimab in India. Toripalimab is a New Biological Entity. It is the only immuno-oncology drug approved by various regulatory authorities around the world such as the United States Food and Drug Administration, European Medicines Agency, Medicines and Healthcare products Regulatory Agency and others for the treatment of adults with recurrent or metastatic nasopharyngeal carcinoma (RM-NPC). In 2023, Dr. Reddy’s entered into a license and commercialization agreement with Shanghai Junshi Biosciences Co. Ltd for Toripalimab. Under this agreement, Dr. Reddy’s obtained exclusive rights to develop and commercialize Toripalimab in 21 countries including India, South Africa, Brazil and various countries in Latin America. Additionally, the agreement allows Dr. Reddy’s to expand the scope of the license to cover Australia, New Zealand and nine other countries.

U.S. FDA Expands Indication for Impella Heart Pumps to Treat Pediatric Patients

Physicians have a new treatment option for many of the sickest pediatric patients with heart failure and cardiogenic shock. BioNJ Member Johnson & Johnson MedTech announced that the U.S. Food and Drug Administration (FDA) has expanded the indications for the Impella 5.5 with SmartAssist and Impella CP with SmartAssist heart pumps, granting premarket approval (PMA) for use in specific pediatric patients with symptomatic acute decompensated heart failure (ADHF) and cardiogenic shock. A PMA is the highest level of approval granted by the FDA for the safety and efficacy of medical devices. Impella 5.5 and Impella CP enable heart recovery as part of the world’s smallest heart pump platform.

GE HealthCare Expands Its Effortless Recon DL Portfolio, Bringing Advanced Deep Learning Image Reconstruction to Clinicians Worldwide

GE HealthCare unveiled three new advanced deep learning image processing and reconstruction solutions as a part of its Effortless Recon DL portfolio. Understanding the need to improve operational efficiencies within radiology departments and the ongoing challenge of burnout experienced by clinicians, GE HealthCare developed the technologies to deliver exceptional image quality, reduce scan times and support improved patient outcomes. The quality of clinical images plays a crucial role in providing accurate diagnoses and optimizing patient care. As technology continues to evolve – deep learning – a subset of artificial intelligence (AI), has emerged as a powerful tool for clinicians. It can positively impact diagnostic accuracy and improve disease detection, which can help improve patient outcomes across clinical specialties.

Sheft, A Legendary Figure In Entrepreneurial Ecosystem, Earns Inaugural Catalyst Award from R&D Council

It’s easy to see why Judith Sheft, a living legend in the STEM entrepreneurial ecosystem in this state, was presented with the first Catalyst Award last week at Bell Works at the 45th annual Edison Patent Awards, sponsored by the R&D Council of New Jersey. After all, as the Executive Director of the New Jersey Commission on Science, Innovation and Technology (which was reestablished six years ago), Sheft has led an organization that has given out 435 awards totaling $27.2 million, helping create 568 jobs while helping companies leverage CSIT funding 15x in terms of additional funding, grants, loans and other support. This, after a nearly two-decade career at NJIT, which followed a stellar career in the private sector.

111 Students Selected for 2024-2025 Governor’s STEM Scholars Class

111 New Jersey students have entered the 2024-2025 Governor’s STEM scholars (GSS) class. Selected from among over 1,000 applicants across the State, these students represent some of the highest achievers in New Jersey. GSS is a public-private partnership between the Research & Development Council of New Jersey, the Office of the Governor, the New Jersey Department of Education, the New Jersey Secretary of Higher Education and public and private research institutions. New Jersey has the highest concentration of engineers and scientists per square mile in the U.S., according to the NJEDA. Recognizing New Jersey’s rich talent pool, GSS serves as a vital bridge for talented STEM students to connect with research and STEM professionals within the industry, academia and government sectors. 

MOU Signed to Increase Collaboration Between Biotechs in NJ and India

In Bengaluru, India, a Memorandum of Understanding (MOU) was signed between the New Jersey-India Commission, Choose New Jersey, the Institute for Life Science Entrepreneurship (ILSE) and the Association of Biotechnology Lead Enterprises (ABLE), an Indian biotechnology organization. Lt. Gov. Tahesha Way signed the MOU which is expected to create a strong connection between biotechnology companies in New Jersey and India. “Through this exciting partnership with Institute for Life Science Entrepreneurship and Association of Biotechnology Lead Enterprises, we will continue to strengthen the deep economic and cultural ties between New Jersey and India,” said Way. “We are eager to bolster opportunities in the biotechnology sector for both regions, and this latest agreement reaffirms our dedication to nurturing economic ties with one of the world’s fastest-growing economies.”

Zena Therapeutics: Rutgers Start-up Aims to Design Safer Prescription Opiates

Rutgers start-up Zena Therapeutics has a noble aim: To create narcotic medications that will minimize or even eliminate overdoses from prescription drugs. Co-founded by Dr. Eileen Carry and Dr. Ariane Vasilatis, the company is based on an innovation developed at Rutgers: A novel compound that does not increase the risk of overdose if taken with other central nervous system depressing substances such as opioids and alcohol. “What we want to do is design medication so that even if it is misused, death is not the consequence,” Dr. Carry said. “Right now, when it comes to narcotics drugs, the onus is on the patient to take the medication as prescribed, but that is not a guarantee. We hope to shift the paradigm to substantially reduce overdose risk without compromising efficacy.”

Rutgers Business School and Google Partner to Enhance Education With AI

Rutgers Business School is partnering with Google Public Sector and Slalom to provide its students and educators with the ability to experience the power of generative AI in the classroom. The collaboration will provide students and faculty access to Google Cloud AI, a single place to discover, customize and deploy more than 150 foundation models from Google and its partners such as Meta’s Llama, Anthropic’s Claude and Google’s Gemini. At the same time, Rutgers Business School is introducing a revamped curricula to ensure that students gain the knowledge necessary to thrive in a rapidly evolving job market shaped by AI. “The fast advancement of technologies has fundamentally shifted the landscape of the business world and is making a transformative impact across industries,” said Lei Lei, dean of Rutgers Business School.

Kean Launches New Degree Programs to Meet Demand for Advanced Technology Education and Innovation

Kean University is moving forward on expanding its academic portfolio with two groundbreaking programs – a Ph.D. in Computer Science and a Bachelor of Science in Artificial Intelligence – reinforcing its position at the forefront of technological education and innovation. “Kean continues to shape the future of higher education, reinforcing its role as both an anchor institution and a leading research university in New Jersey,” President Lamont Repollet, said. “These new programs embody our commitment to creating pathways of opportunity for our students while addressing the critical technological needs of our society and State”. The initiatives reflect Kean’s strategic focus on meeting global workforce demands and dedication to excellence in STEM education.

Hackensack Meridian CDI Researcher Earns NJ ‘Emerging Tech Award’

A Hackensack Meridian Center for Discovery and Innovation (CDI) researcher was recently honored with the Research & Development Council of New Jersey’s inaugural Emerging Tech Award – the first such distinction for a scientist from Hackensack Meridian Health. Olivier Loudig, Ph.D., an associate member of the CDI, was honored for his innovative efforts on biomarker discovery for early detection of metastasizing breast cancer and lung cancer. He received the award at the 45th annual Edison Patent Awards Ceremony and Reception on Nov. 21 at Bell Works in Holmdel. The awards, the highest recognition of innovation in the state, celebrated 14 patents created by 62 inventors and five individual award winners. 

Surendralal Karsanbhai Elected to Merck Board of Directors

Rahway-based BioNJ Member Merck & Co. announced that Surendralal L. “Lal” Karsanbhai, President and CEO, Emerson Electric Co. will join Merck’s Board of Directors effective January 1, 2025. “We are pleased to welcome Lal Karsanbhai to the Merck board. Mr. Karsanbhai brings extensive business and operational expertise with a strong global perspective, and we look forward to his contributions and insights,” said Robert M. Davis, Chairman CEO, Merck. Mr. Karsanbhai joined Emerson in 1995 and held various roles increasing in scope and responsibility before becoming Emerson’s President and CEO and an executive director on its board in 2021. Mr. Karsanbhai also serves as deputy chair of the Federal Reserve Bank of St. Louis and on the board of the U.S.-China Business Council.

NS Pharma Announces Change in Commercial Leadership

Paramus-based NS Pharma Inc. announced a change of leadership within its Commercial division. Donald Foy – who had previously served as National Sales Director – was appointed to the role of Vice President, Commercial. Jennifer Tamberino – who had been Regional Business Director, East, National Sales, was promoted to backfill Mr. Foy’s former position as National Sales Director. “It is with incredible excitement that we announce Don’s promotion to lead our sales, marketing, market access, operations and patient services departments as our new Head of Commercial at NS Pharma,” said NS Pharma President Yukiteru Sugiyama, Ph.D. “With Don and Jennifer at the helm, we are well- positioned to execute our plans for growth in the rare disease space in the United States. Our new leadership structure is designed to foster industry collaboration and company innovation from the top down.”

Merck Earns Top Spot on Newsweek’s List of America’s Most Responsible Companies 2025

This report of Merck & Co., Inc., Rahway, NJ, USA includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the Company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

LEO Pharma Commits to Net-Zero Greenhouse Gas Emissions by 2050

A net-zero target is a natural continuation of our current commitments to run a sustainable business, LEO Pharma says. LEO Pharma today announced its commitment to achieving a net-zero climate target by 2050. It involves developing an extensive decarbonization plan across the company’s operations, aligning with the Paris Agreement and climate science recommendations to limit global warming to 1.5⁰ C. This commitment builds on LEO Pharma’s earlier ambition to reduce carbon emissions by more than 50% by 2030 from a 2019 baseline.

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.

Tech Opportunity Webinar: Novel Signal Amplification By Reversible Exchange (SABRE) Catalyst for Improved MRI Metabolic Sensing

December 18, 2024 | Hosted by NCI Technology Transfer Center

The National Heart Lung & Blood Institute (NHLBI) and the National Cancer Institute (NCI) are seeking research co-development partners and/or licensees for a novel technology: a newly developed method of obtaining hyperpolarized imaging agents for real-time metabolic imaging using magnetic resonance imaging (MRI). This method, catalyst and the resulting imaging agents, can be used in MRI metabolic imaging to detect cancer, cardiovascular disease, stroke, and traumatic brain injury; and monitor therapies without the presence of metals, such as gadolinium or iron. Learn about this new technology by attending this free webinar.

NCATS SBIR/STTR Funding: Translating Your Research into Real-World

January 22, 2025 | Hosted by National Center for Translational Sciences

Are you a researcher or entrepreneur with a groundbreaking idea in translational science? Want to take your research from the lab to the market? The National Center for Advancing Translational Sciences (NCATS) Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs can help turn your vision into reality. These programs offer non-dilutive funding and other resources to support the development and commercialization of your innovative technologies.