Welcome New Members

• Government Systems Technologies, Inc. (GSTi) 
• Kathera Bioscience
• Lura Health
• Surplus Solutions, LLC

Learn more at www.BioNJ.org/Membership.

Organon, Sempre Health Collaborate on Innovative Cost-Savings Program

Jersey City-based Organon recently announced a partnership with Sempre Health to motivate asthma patients to refill their prescriptions on time and improve adherence through an innovative cost-savings program. The new program will enable eligible patients who refill their prescriptions on time and as prescribed for Dulera (mometasone furoate, formoterol fumarate dihydrate) and Asmanex (mometasone furoate) to receive discounts on their medications throughout the course of their therapy, with the aim of improving adherence to their medication.  

The Pill Penalty, Patients and Policymakers

The Centers for Medicare and Medicaid Services is continuing implementation of the Inflation Reduction Act (IRA) by holding “listening sessions” to gather perspectives on the first 10 drugs subject to price setting. While these sessions are hugely important – and we sincerely hope CMS pays close attention to concerns raised by the patients participating – we also urge lawmakers to consider an immediate action Congress can take to address the unintended consequences created by IRA. One such action is fixing the IRA’s small molecule “pill penalty.”  

The Medicare Budget Implications of the Inflation Reduction Act

The Inflation Reduction Act (IRA) has two primary policy initiatives: reforms to Medicare prescription drug policies and large-scale subsidies to clean (non-fossil) energy. In discussing the IRA, advocates typically keep the two separate, holding that the Medicare drug policy will yield savings for the Medicare program while clean energy subsidies will speed the transition to a new energy portfolio. Yet the two are linked by the unified federal budget. This reviews the budget impact of the IRA and finds that of the $266 billion in Medicare savings the IRA’s provisions will yield, none will benefit program participants and will instead be spent to subsidize the law’s clean energy tax credits and other related spending – and leave a residual to be borrowed.

The Problem With Drug Price Controls

President Biden’s war on drug prices is a prescription for shortages and fewer new and innovative drugs. To make matters worse, he keeps bragging about his populist reforms as if they are going to solve the problems with our healthcare system, the economy, the itch, the stitch, palsy and the gout. But he isn’t very convincing when he delivers the message, because his reasoning for passing bills changes depending on which audience he is talking to. His goal is merely to bask in the limelight of his populist agenda. But his policies are going to have long-term consequences.   

How the IRA Could Delay Pharmaceutical Launches, Reduce Indications and Chill Evidence Generation

Clinical development programs for new drugs include an array of research conducted both before and after a drug’s initial approval by the Food and Drug Administration (FDA). Post-approval clinical trials may be conducted in new patient populations to support additional indications and to demonstrate long-term safety and efficacy, including impacts on overall survival or major health events. Research beyond the trials required for initial FDA approval is a critical component of expanding accessible treatment options for patients. New indications, obtained by approximately a third of drugs, can reduce insurance-related barriers that patients face in accessing drugs for evidence-based off-label use. Furthermore, long-term outcomes data inform prescribing practices and clinical guidelines. The Inflation Reduction Act (IRA) of 2022 significantly changes the economic incentives surrounding clinical development in the United States.

Where the FDA is on AI

The Biden administration hasn’t decided how to handle emerging tools like chatbots that interact with patients and answer doctors’ questions – even though some are already in use. Biden is taking a step toward addressing that with an executive order that calls on the Department of Health and Human Services to create a task force to develop a strategic plan within a year on the responsible use of AI. The Food and Drug Administration has approved about 520 AI-enabled devices — mostly for radiology, where the technology has shown promise in reading X-rays. FDA Commissioner Robert Califf said he believed the agency has done well with predictive AI systems, which take data and conjecture an outcome.

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Video: ICER’s Value Assessments Harm Access for Protected Populations – What You Need to Know

(Alliance for Aging Research) “The Institute for Clinical and Economic Review promotes drug pricing metrics – including the quality adjusted life year (QALY) and equal value of life years gained (evLYG) – that discriminate and harm access for people with disabilities and older adults. Patients deserve better.”  

Phathom Pharmaceuticals Announces FDA Approval of VOQUEZNA® (vonoprazan) Tablets for the Treatment of Erosive GERD and Relief of Heartburn Associated With Erosive GERD in Adults

Florham Park-based BioNJ Member Phathom announced the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) tablets 10 mg and 20 mg, a novel potassium-competitive acid blocker (PCAB), as a new treatment for adults for the healing of all grades of Erosive Esophagitis, also known as Erosive GERD (gastroesophageal reflux disease), maintenance of healing of all grades of Erosive GERD, and relief of heartburn associated with Erosive GERD. This approval is based on positive results from the Phase 3 PHALCON-EE study (NCT04124926). The pivotal trial was a randomized, double-blind, multicenter study that enrolled 1,024 patients with Erosive GERD in the U.S. and Europe and compared VOQUEZNA to the PPI lansoprazole in the healing and maintenance of healing of Erosive GERD and associated heartburn symptom relief.  

Phathom Pharmaceuticals Announces FDA Approval of Reformulated Vonoprazan Tablets for VOQUEZNA® TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin) and VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) for the Treatment of H. pylori Infection in Adults

Florham Park-based BioNJ Member Phathom announced that the U.S. Food and Drug Administration (FDA) has approved the Prior Approval Supplement (PAS) for the reformulation of vonoprazan tablets for both VOQUEZNA TRIPLE PAK (vonoprazan tablets, amoxicillin capsules, clarithromycin tablets) and VOQUEZNA DUAL PAK (vonoprazan tablets, amoxicillin capsules), for the treatment of Helicobacter pylori (H. pylori) infection in adults. VOQUEZNA treatment regimens contain antibiotics conveniently packaged with vonoprazan, a novel potassium-competitive acid blocker (PCAB) and the first innovative acid suppressant from a new drug class approved in the U.S. in over 30 years. These initial product approvals were based on safety and efficacy data from the Phase 3 PHALCON-HP trial, the largest U.S. registrational trial ever conducted in H. pylori, randomizing 1,046 patients.  

Celldex Therapeutics Announces Positive Topline Results from Barzolvolimab Phase 2 Study in Chronic Spontaneous Urticaria

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced positive topline results from the Company’s Phase 2 clinical trial of barzolvolimab in patients with moderate to severe chronic spontaneous urticaria (CSU) refractory to antihistamines, including patients who received prior biologics. Barzolvolimab is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity, which is required for mast cell function and survival. CSU is characterized by the occurrence of hives or wheals for 6 weeks or longer without identifiable specific triggers or causes. Treatment options for patients with CSU are limited and there are no approved therapies for patients who do not respond to omalizumab.

Celldex Therapeutics Presents Positive Data from Prurigo Nodularis Phase 1b Study Demonstrating Meaningful Reduction in Itch and Skin Clearing With Single Dose 3.0 mg/kg Barzolvolimab

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced data from the company’s Phase 1b study of barzolvolimab in prurigo nodularis (PN). Barzolvolimab is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity, which is required for the function and survival of the mast cell. Mast cells are believed to play an important role in amplifying chronic itch and neuroinflammation, including in PN. This study is the first to demonstrate that barzolvolimab, a mast cell depleting agent, can potentially be used to treat PN and other chronic itch indications. PN is a chronic skin disease that causes hard, intensely itchy lumps/nodules to form on the skin.  

EmphyCorp Inc. Non-Steroidal N115 Nasal Spray Completed Phase III Clinical Trial for COVID-19, Long COVID-19 (Long Haulers), and Pulmonary Fibrosis

Flemington-based BioNJ Member EmphyCorp Inc. has announced their flagship product, N115, has demonstrated efficacy, with no known side effects, for all lung and sinus diseases tested including COPD, Pulmonary Fibrosis, CF, Allergic Rhinitis, Chronic Rhinitis, Sinusitis, and Flu. In 17 human clinicals (Phase I, II, III including animal safety data) submitted to the FDA, N115 produced statistically significant increases in all lung functions with the reduction in nasal and respiratory inflammation, a reduction in oxygen radicals, congestion, coughing, fatigue, and a reduction of inflammatory cytokines including IL-6, a cause of the cytokine storm in COVID-19 patients. N115 also increases the synthesis of nasal nitric oxide, a natural defense molecule that kills invading bacteria, fungi, and viruses to prevent and reduce the severity of infections.  

FDA Approves Lilly's Zepbound™ (tirzepatide) for Chronic Weight Management, a Powerful New Option for the Treatment of Obesity or Overweight with Weight-Related Medical Problems

The U.S. Food and Drug Administration (FDA) approved BioNJ Member Eli Lilly and Company's Zepbound™ (tirzepatide) injection, the first and only obesity treatment of its kind that activates both GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) hormone receptors. Zepbound is indicated for adults with obesity (with a BMI of 30 kg/m2 or greater), or those who are overweight (with a BMI of 27 kg/m2 or greater) and also have weight-related medical problems such as hypertension, dyslipidemia, type 2 diabetes mellitus, obstructive sleep apnea or cardiovascular disease, to lose weight and keep it off. It should be used with a reduced-calorie diet and increased physical activity.  

FDA Approves Novartis Cosentyx® as the First New Biologic Treatment Option for Hidradenitis Suppurativa Patients in Nearly a Decade

East Hanover-based BioNJ Member Novartis announced that the US Food and Drug Administration (FDA) has approved Cosentyx® (secukinumab) to treat moderate to severe hidradenitis suppurativa (HS) in adults. Cosentyx is the only FDA-approved fully human biologic that directly inhibits interleukin-17A (IL-17A), a cytokine believed to be involved in the inflammation of HS. The FDA approval was based on analyses from the largest Phase III program in HS to date, SUNSHINE and SUNRISE, in which a higher proportion of patients given Cosentyx 300 mg either every two weeks or every four weeks achieved a Hidradenitis Suppurativa Clinical Response (HiSCR50) compared to placebo. Cosentyx for HS is approved as a 300 mg dose, administered every four weeks, with the option to increase to every two weeks if the patient has an inadequate response.  

FDA Approves Merck’s KEYTRUDA® (pembrolizumab) Plus Gemcitabine and Cisplatin as Treatment for Patients With Locally Advanced Unresectable or Metastatic Biliary Tract Cancer

Rahway-based BioNJ Member Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with gemcitabine and cisplatin, for the treatment of patients with locally advanced unresectable or metastatic biliary tract cancer (BTC). The approval was based on results from the Phase 3 KEYNOTE-966 trial, in which KEYTRUDA plus chemotherapy demonstrated a statistically significant improvement in the study’s primary endpoint of overall survival (OS), reducing the risk of death by 17% (HR=0.83 [95% CI, 0.72-0.95]; one-sided p=0.0034) compared to chemotherapy alone at the trial’s pre-specified final analysis for OS. Median OS was 12.7 months (95% CI, 11.5-13.6) for KEYTRUDA plus chemotherapy versus 10.9 months (95% CI, 9.9-11.6) for chemotherapy alone.  

KEYTRUDA® (pembrolizumab) Significantly Improved Overall Survival (OS) Versus Placebo as Adjuvant Therapy for Certain Patients With Renal Cell Carcinoma (RCC) Following Nephrectomy

Rahway-based BioNJ Member Merck & Co. announced that the Phase 3 KEYNOTE-564 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, met its key secondary endpoint of overall survival (OS), for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions. At a pre-specified interim analysis review conducted by an independent Data Monitoring Committee, KEYTRUDA demonstrated a statistically significant and clinically meaningful improvement in OS compared to placebo. The safety profile of KEYTRUDA was consistent with that observed in previously reported studies; no new safety signals were observed.  

Pfizer and BioNTech Announce Positive Topline Data for mRNA-based Combination Vaccine Program Against Influenza and COVID-19

BioNJ Member Pfizer, with a site in Peapack, and BioNTech announced positive topline results from a Phase 1/2 study (NCT05596734) evaluating the safety, tolerability and immunogenicity of mRNA-based combination vaccine candidates for influenza and COVID-19 among healthy adults 18 to 64 years of age. In the clinical trial, the vaccine candidates were compared to a licensed influenza vaccine and the companies’ Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine given at the same visit. The data from the trial showed that the companies' lead formulations demonstrated robust immune responses to influenza A, influenza B, and SARS-CoV-2 strains. The topline results of the ongoing trial demonstrated that the combination formulations evaluated had a safety profile consistent with the safety profile of the companies’ COVID-19 vaccine.  

Phase III RUBY Trial of Jemperli (dostarlimab) Plus Chemotherapy Meets Endpoint of Overall Survival in Patients With Primary Advanced or Recurrent Endometrial Cancer

Warren-based BioNJ Member GSK announced positive headline results from a planned analysis of Part 1 of the RUBY/ENGOT-EN6/GOG3031/NSGO Phase III trial investigating Jemperli (dostarlimab) plus standard-of-care chemotherapy (carboplatin and paclitaxel), followed by dostarlimab as a single agent, compared to placebo plus chemotherapy followed by placebo in adult patients with primary advanced or recurrent endometrial cancer. The trial met its primary endpoint of overall survival (OS), demonstrating a statistically significant and clinically meaningful benefit in the overall patient population. A clinically meaningful OS benefit was observed in both prespecified subpopulations in the trial: mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) and mismatch repair proficient (MMRp)/microsatellite stable (MSS) patient subgroups. OS is one of two primary endpoints in the RUBY Part 1 trial.  

UZEDY™ (risperidone) Extended-Release Injectable Suspension Significantly Prolongs Time to Schizophrenia Relapse in RISE Study Results Published in The Lancet Psychiatry

Parsippany-based BioNJ Member Teva Pharmaceutical announced the publication of efficacy and safety findings from the Phase 3 Risperidone Subcutaneous Extended-Release (RISE) study in The Lancet Psychiatry. The data supported the FDA approval for UZEDY, which was approved in April 2023 for the treatment of schizophrenia in adults as a subcutaneous injection every one or two months using a pre-filled syringe. In RISE, UZEDY significantly prolonged time to impending relapse by 5.0 times with once-monthly dosing (HR 0.200, 95% CI 0.109–0.367; log rank p<0.0001) and 2.7 times with once-every-two-months dosing (0.375, 0.227–0.618; log rank p<0.0001) versus placebo in patients with schizophrenia, corresponding to a decreased risk of relapse by 80% and 62.5%, respectively. Study findings also show UZEDY provided clinically relevant plasma concentrations within 24 hours of administration and maintained them over the flexible dosing intervals.   

GSK Enters Agreement to Obtain Exclusive License for JNJ-3989 to Expand the Development of Bepirovirsen

Warren-based BioNJ Member GSK and Arrowhead Pharmaceuticals Inc. announced that they have reached an agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company, to transfer exclusive worldwide rights to further develop and commercialize JNJ-3989 to GSK. JNJ-3989 (formerly ARO-HBV) was initially in-licensed by Janssen from Arrowhead in 2018. JNJ-3989 is an investigational hepatitis B virus-targeted small interfering ribonucleic acid (siRNA) therapeutic that GSK intends to evaluate in a sequential regimen with bepirovirsen, GSK’s investigational antisense oligonucleotide, for the treatment of adult non-cirrhotic patients with chronic hepatitis B (CHB) on nucleos(t)ide analogue (NA) therapy. There is high unmet need in CHB with an estimated 300 million people living with the disease and a less than 3-7% functional cure rate with current treatment options.   

JAMA Neurology Publishes Complete Results of Positive Phase 3 Study of REXULTI® (brexpiprazole) for Agitation Associated With Dementia Due to Alzheimer’s Disease

BioNJ Member Otsuka Pharmaceutical Development & Commercialization, Inc., with a site in Princeton, and Lundbeck Pharmaceuticals LLC announce that treatment with REXULTI® (brexpiprazole) resulted in statistically significant and clinically meaningful improvements in adult patients with agitation associated with dementia due to Alzheimer’s disease, according to the complete results of the placebo-controlled pivotal Phase 3 Study 213 (NCT03548584), published in JAMA Neurology. In May 2023, brexpiprazole became the first and only drug to receive approval from the Food and Drug Administration for this indication. REXULTI is not indicated as an as needed (“prn”) treatment for agitation associated with dementia due to Alzheimer’s disease.   

Phase 2 Nipocalimab Data Establish Proof of Mechanism in Adults Living With Moderate to Severe Rheumatoid Arthritis, Supporting its Progression into a Combination Study

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced data from the Phase 2a IRIS-RA clinical study for the treatment of adults living with moderate to severe active rheumatoid arthritis (RA) who have tested positive for anti-citrullinated protein antibodies (ACPAs) and/or rheumatoid factor (RF), and who have had an inadequate response or intolerance to at least one anti-tumor necrosis factor (anti-TNF) therapy. The data from this study establish proof of mechanism for nipocalimab in RA and support its progression into a combination study. IRIS-RA is the first clinical study to assess the efficacy and safety of the anti-neonatal Fc (FcRn)-driven MOA for the treatment of RA. The study findings, along with an acceptable benefit-risk profile, support the further investigation of nipocalimab in this refractory population.   

Janssen Submits Phase 3 Study Data to the European Medicines Agency and U.S. Food and Drug Administration for SIRTURO® (bedaquiline)

New Brunswick-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Type II Variation application to the European Medicines Agency (EMA) for the company’s medicine SIRTURO® (bedaquiline). A supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) was also submitted in August 2023. SIRTURO® is indicated in adults and pediatric patients (5 years to less than 18 years of age and weighing at least 15 kg) as part of combination therapy of pulmonary tuberculosis (TB) due to multi-drug resistant Mycobacterium tuberculosis. The approved indication may vary per country. These submissions reflect the company’s longstanding commitment to patients affected by multidrug-resistant TB (MDR-TB), one of the world’s most significant global health challenges.  

New Real-World Data Show TREMFYA® (guselkumab) Was Associated With Clinically Meaningful Improvements in Patient-Reported Outcomes for Adults Living With Active Psoriatic Arthritis

Raritan-based BioNJ Member the Janssen Pharmaceuticals, Inc., a Johnson & Johnson company, announced new data from the CorEvitas Psoriatic Arthritis (PsA) and Spondyloarthritis Registry that showed a substantial proportion of people living with treatment-resistant active PsA and using TREMFYA® (guselkumab) in real-world settings reported meaningful improvements in pain, physical function and fatigue through six months. Additionally, across the DISCOVER-1, DISCOVER-2 and COSMOS clinical trials, treatment with TREMFYA was associated with higher rates of clinically meaningful improvements in a composite assessment of patient-reported pain, fatigue, physical function, skin problems and PsA-related depression than placebo in the first assessment of the PsA 5-Thermometer Scale Domains (PsA-5T-Ds). TREMFYA is the first and only fully human selective interleukin (IL)-23p19-subunit inhibitor therapy approved for the treatment of adults living with active PsA.  

New England Journal of Medicine Publishes Complete Results of Positive Phase 2 Trial of Sibeprenlimab in Treatment of Immunoglobulin A Nephropathy (IgAN)

Princeton-based BioNJ Member Otsuka Pharmaceutical Development & Commercialization, Inc., and Visterra Inc., announced that the full results of a Phase 2 trial of sibeprenlimab (VIS649) for treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease) have been published in the New England Journal of Medicine. Sibeprenlimab is an investigational humanized monoclonal antibody that blocks the action of the cytokine A Proliferation Inducing Ligand (APRIL), an immune cell growth factor believed to play a key role in the development and progression of IgAN. The study results demonstrated that 12 months of sibeprenlimab treatment in patients with IgAN resulted in significant reductions in proteinuria compared to placebo. At 12 months, geometric mean ratio reduction in 24-hour uPCR from baseline was 47.2%, 58.8%, 62.0% and 20.0% with sibeprenlimab 2, 4, and 8 mg/kg, and placebo, respectively.  

Evotec and Dewpoint Therapeutics Enter Strategic Partnership in Oncology

Princeton-based BioNJ Member Evotec and Dewpoint Therapeutics announced a strategic R&D collaboration to advance Dewpoint’s leading oncology pipeline programmes of condensate modifying therapeutics (“c-mods”) to Investigational New Drug Applications (“INDs”) using Evotec’s industry-leading fully integrated data-driven platform (“INDiGO”). The partnership brings together Dewpoint’s advanced oncology pipeline programmes discovered using its groundbreaking condensate biology and A.I. technology platform for identifying modulators of biomolecular condensates with Evotec’s leading capabilities to de-risk and accelerate pre-clinical development candidates (“PDCs”) to first-in-human (“FIH”) clinical studies. Under the collaboration, Evotec will facilitate the rapid development of Dewpoint’s oncology assets using Evotec’s fully integrated clinical-enabling INDiGO platform to de-risk and accelerate the path to clinical testing. The partnership also enables Dewpoint to maximize capital efficiency while reducing risk.  

SPEVIGO® (spesolimab) Wins 2023 Prix Galien USA Award

BioNJ Member Boehringer Ingelheim, with a site in North Brunswick, announced that SPEVIGO® (spesolimab) has been recognized as Best Orphan/Rare Diseases Solution of 2023 by the Galien Foundation. In 2022 the U.S. Food and Drug Administration approved spesolimab as a treatment option for GPP flares in adults and the European Medicine Agency granted a conditional market authorization as first-in-class treatment option for GPP flares in adults. “GPP flares may appear suddenly, intensify quickly and can be life-threatening if left untreated, leaving those affected and their loved ones feeling anxious and uncertain about their future. We are humbled by the positive impact SPEVIGO® has had on GPP patients across the world and take this recognition as a driver to continue our pursuit for their continued benefit,” said Carinne Brouillon, Board Member, Managing Directors & Head, Human Pharma at Boehringer Ingelheim.  

Gilead and Arcus Announce New Data Showing Encouraging Clinical Activity of Anti-TIGIT Domvanalimab-Containing Regimen as First-line Treatment for Upper GI Cancers

BioNJ Member Gilead Sciences, with a site in Morris Plains, snd Arcus Biosciences, Inc. announced that domvanalimab plus zimberelimab and chemotherapy showed encouraging overall response rate (ORR) and six-month progression-free survival (PFS) rate results in a preliminary analysis from Arm A1 of the EDGE-Gastric study. This ongoing Phase 2, multi-arm, global study is evaluating the safety and efficacy of various combinations of the Fc-silent anti-TIGIT antibody domvanalimab plus the anti-PD-1 antibody zimberelimab and chemotherapy in patients with locally advanced unresectable or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma. 

Gilead Named Number One Overall Philanthropic Funder of HIV-Related Programs for Second Year in a Row by Funders Concerned About AIDS

BioNJ Member Gilead Sciences, with a site in Morris Plains, was recognized as the number one philanthropic funder of HIV-related programs for the second year in a row, in a new report released by Funders Concerned About AIDS (FCAA). FCAA’s annual Philanthropic Support to Address HIV and AIDS report is widely regarded as the most comprehensive study of its kind, and analyzed data from more than 5,000 grants, disbursed by 187 funders and awarded to 2,800 grantee organizations. The 2023 report analyzed 2021 funding data – the most recent yearly data available – and found that Gilead distributed more than 1,000 HIV-related grants totaling almost $240 million in HIV-related projects, ranking the company once again as the number one overall funder of HIV-related programs both globally and in the U.S.  

LEQEMBI® (LECANEMAB-IRMB) Named One of TIME’s Best Inventions of 2023

Nutley-based Eisai announced that TIME has selected Alzheimer’s disease (AD) treatment LEQEMBI® (lecanemab-irmb) as one of THE BEST INVENTIONS of 2023 in the Medical Care category. TIME’s annual list of THE BEST INVENTIONS features “200 extraordinary innovations changing lives.” To compile the list, TIME solicited nominations from its editors and correspondents around the world, and through an open online application process, paying special attention to growing fields, such as AI, green energy and sustainability. TIME then evaluated each contender on a number of key factors, including originality, efficacy, ambition and impact. LEQEMBI is the first and only treatment approved in Japan and the United States shown to reduce the rate of disease progression and to slow cognitive and functional decline, that acts on the underlying pathology of AD.  

Postpartum Depression Digital Therapeutic Shows Positive Results in Clinical Trial

Rocky Hill-based Curio Digital Therapeutics has announced positive clinical trial data for its postpartum depression digital therapeutic. MamaLift Predict is a validated algorithm that has demonstrated success in identifying and stratifying women at risk for postpartum depression. SuMMER, a study executed by HITLAB, assessed MamaLift Plus for eight weeks in 141 patients using the Edinburgh Postnatal Depression Scale (EPDS). The EPDS is a clinically validated tool that healthcare providers use to evaluate, diagnose, and monitor depression in pregnant and postpartum women. Scores 13 and above indicate depressive illness or a high risk of developing a depressive disorder. Patients involved in the MamaLift Predict trial had baseline EPDS scores 13 and above but not exceeding 19, and a confirmed diagnosis of postpartum depression prior to enrollment.  

Ascendis Presents Updated and New TransCon™ IL-2 β⁄γ Monotherapy and Combination Therapy Data Confirming Clinical Activity Across Tumor Types at ESMO 2023

Ascendis Pharma, with a site in Princeton, reported updated and new data from its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β⁄γ. The data included longer-term follow up of previously presented TransCon IL-2 β⁄γ monotherapy data from the IL-Believe Trial and was the first presentation of dose escalation data informing recommended Phase 2 dose (RP2D) for TransCon IL-2 β⁄γ in combination with a checkpoint inhibitor. “These updated data reinforce the promising Phase 1 monotherapy data for TransCon IL-2 β⁄γ reported earlier this year, further strengthening our confidence in its best-in-class potential,” said Stina Singel, M.D., Ph.D., Executive Vice President, Head of Clinical Development, Oncology at Ascendis Pharma. “In addition to previously reported monotherapy clinical activity, we are particularly encouraged to see anti-tumor responses in two of the three patients with SCLC in the combination portion of the trial who had previously progressed on checkpoint inhibitors.”  

New Drug Application Approval by the Pharmaceutical Administration Bureau of Macau for Nefecon® for the Treatment of Primary IgA Nephropathy

Calliditas Therapeutics, with a site in Hoboken, announced that its commercial partner Everest Medicines received approval from the Pharmaceutical Administration Bureau of the Macau Special Administrative Region, China. The approval for Nefecon is for the treatment of primary immunoglobulin A nephropathy (IgAN) in adults at risk of disease progression. Macau is the first region in Everest territories that received Nefecon approval. The NDA for Nefecon in mainland China is under Priority Review and Nefecon was the first non-oncology medicine to receive Breakthrough Therapy Designation in China. Nefecon has been available for clinical use in Shanghai Ruijin Hospital’s Hainan subsidiary through an early access program since April 2023.  

U.S. FDA Awards Both Rare Pediatric Disease and Orphan Drug Designations to HG204, a CRISPR RNA-Editing Therapy, for the Treatment of MECP2 Duplication Syndrome

Clinton-based HuidaGene Therapeutics announced the U.S. Food and Drug Administration (FDA) had granted both the Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) to HG204 for the treatment of MECP2 duplication syndrome (MDS), a rare and fatal childhood neurodevelopmental disorder for which there is no treatment. "We are delighted to have been awarded both RPDD and ODD for HG204, the world's first novel CRISPR RNA-editing therapy independently developed by HuidaGene for the treatment of the rare and fatal MDS genetic disease, by the U.S. FDA," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder and Chief Executive Officer of HuidaGene. "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.”  

Tris Partners With Perigon to Deliver ADHD Drug

Monmouth Junction-based Tris Pharma, Inc. announced a new partnership with Perigon Pharmacy 360, a full-service, specialty digital pharmacy, to enhance accessibility, affordability and adherence for patients treating symptoms of ADHD with Tris’ DYANAVEL® XR (amphetamine) extended-release tablets. Together, Tris Pharma and Perigon launched a direct-to-patient program, DYANAVEL Delivered, that allows patients and caregivers to access DYANAVEL XR extended-release tablets through Perigon’s digital pharmacy, enhance their medication management, and receive reimbursement assistance and support to help navigate their treatment journey while reducing administrative burden for both patients and providers. The program is available across the United States, enabling nationwide access to DYANAVEL XR extended-release tablets.  

NJ Department of Labor and Workforce Development Announces $10M Incumbent Worker Training Grant

The New Jersey Department of Labor and Workforce Development (NJDOL) has announced $10 million in available funding through the UPSKILL: NJ Incumbent Worker Training Grant to help employers further train existing workers. The FY24 funds will be competitively awarded to New Jersey employers to reimburse up to 50 percent of their cost for training frontline employees to meet current and future occupational skills needed for mid- and high-skill jobs. Applications are being accepted from individual employers; employer-, labor-, community- and faith-based organizations; and secondary or postsecondary school or training providers seeking to fulfill the training needs of an industry-specific consortium of employers. Individual employers may apply for a maximum of $200,000, while consortiums may apply for a maximum of $100,000. To apply, applicants must be registered and approved by NJDOL in the online IGX grant management system, submit a letter of intent and complete and upload required documents. 

$5M Growing Apprenticeship in Nontraditional Sectors (GAINS) Grant Announced by NJDOL

The New Jersey Department of Labor and Workforce Development (NJDOL) Office of Apprenticeship has revealed a $5 million fund. This comes from the Growing Apprenticeship in Nontraditional Sectors (GAINS) grant program. The grant aims to expand Registered Apprenticeship programs approved by the US Department of Labor. It also supports better-paying jobs and advanced credentials acquisition. The GAINS fund can cover up to 50 percent of new apprentices' wages. The maximum amount is $12,000 per apprentice over 52 work weeks. Proposals should offer new apprentices a starting wage of $17 per hour unless a lower rate is justified. The grant is open to various entities, including employers, labor organizations, educational agencies, non-profit organizations and business or trade organizations.

Princeton University Receives Major Gift that Will Name New Health Center

Princeton University announced a major gift from Dr. Thomas Frist Jr. that will name the University’s new health center. Dr. Thomas Frist Jr. gifted the school’s Venture Forward campaign which will be more than twice as large as the McCosh Health Center, the current home of University Health Services (UHS) and will incorporate a renovated Eno Hall with a significant new addition to the south. Scheduled to open in 2025, the Frist Health Center will help the University advance a culture of health and well-being on the Princeton campus. “I am delighted that the Frist family’s legacy in health care innovation will now be reflected in the spectacular Frist Health Center. The University is deeply grateful to Tommy and the Frist family for their visionary partnership on this project and, when considered along with the Frist Campus Center, for their incalculable and enduring impact on our campus community," said President Christopher Eisgruber.

Director of Center of Innovation at RWJUH Appointed to FDA Advisory Committee

Dr. Naveena Yanamala, Director of the Center of Innovation at Robert Wood Johnson University Hospital, has been appointed to the FDA’s Center for Devices and Radiological Health Patient Engagement Advisory Committee. Dr. Yanamala will serve as a special government employee on the PEAC until July 2027. The PEAC consists of nine voting members who are patients, caregivers, and health care advocates, along with one nonvoting industry representative and one voting consumer representative. The primary purpose of the committee is to ensure that patients’ needs and experiences are integral to the FDA’s discussions concerning intricate matters related to the regulation of medical device usage by patients.  

Tevogen Bio Appoints Mittul Mehta as CIO, Head of Its New AI Initiative

Warren-based BioNJ Member Tevogen Bio Inc. has appointed Mittul Mehta as Chief Information Officer. Mr. Mehta will also head Tevogen.ai, the newly launched initiative focused on harnessing the potential of artificial intelligence for the enhancement of drug discovery, development, manufacturing, distribution and patient access. With over 20 years of experience in information technology and almost a decade in senior management roles, Mr. Mehta brings with him a diversified background in IT. His expertise encompasses a wide range of responsibilities, from leading cross-functional teams to architecting solutions. Mr. Mehta most recently served as Senior Vice President, Global Head of Platforms Security, Mobility & Cloud Security at Jefferies LLC, where he played a pivotal role in enterprise strategy.  

Lynn Mason Appointed CEO for IVI RMA America

Basking Ridge-based IVI RMA America, the largest reproductive medicine group in the world, recently announced the appointment of Lynn Mason as CEO. She will oversee the North American operations as well as growth initiatives in support of bringing better fertility outcomes to more patients across the U.S. and Canada. “Lynn is a talented, proven leader who has made her mark on the ever-changing health care environment by working as a transformative executive in both large and small organizations, delivering quality care and a great patient experience,” Javier Sanchez Prieto, CEO of IVI RMA, said. “We’re excited for Lynn’s leadership as we continue to foster a patient-first approach while delivering industry-leading fertility solutions.”

Lexicon Strengthens Management Team With Two New Executives

Lexicon Pharmaceuticals, Inc., with a site in Princeton, announced that Matthew Cullen has joined the company as Vice President of Value and Access, and Lisa DeFrancesco will join as Head of Investor Relations and Corporate Strategy, effective November 13, 2023. Mr. Cullen brings Lexicon more than 35 years of industry experience in market access and sales leadership. Ms. DeFrancesco is an accomplished senior executive with nearly 25 years of experience in various investor relations, finance and corporate communications roles. “Matt and Lisa’s experience and knowledge will be instrumental in our execution of the launch of INPEFA® (sotagliflozin) in heart failure and the advancement of LX9211 into late-stage development for the long-term creation of value for our stakeholders,” said Lonnel Coats, Lexicon’s CEO.  

NJADDC Oncology Symposium

Hosted by New Jersey Academic Drug Discovery Consortium

November 16, 2023

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