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December 9, 2022

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Welcome to the latest edition of the BioLines Weekender...


BioNJ hosted its Inaugural Health Equity in Clinical Trials MBA Business Plan Case Competition this past Saturday, December 3, at Rutgers University Business School. Part of a broader BioNJ strategic initiative of Health Equity in Clinical Trials, the Business Plan Case Competition was designed to promote the next generation of diverse clinical trial innovators and identify innovative approaches and successful models that can be used nationally to strengthen diversity in clinical trials and expand health equity.


Eight teams, competing for more than $20,000 in prize money, were tasked with developing a business plan defining a new solution, application or technology to help address this important and challenging problem. During the Competition on Saturday, live pitch presentations were made to a Panel of Judges comprised of industry experts.

 

The three winning teams were:

  • First Place: Johns Hopkins University ($10,000) who focused their plan on increasing representation in clinical trials for the treatment of Alzheimer's Disease through community engagement strategies and the use of digital tools.
  • Second Place: Rutgers University ($7,000) who focused their plan on using population health and real-world data analytics to calculate health equity targets specific to clinical trial sites in prostate cancer.
  • Third Place: Baylor College of Medicine, Northwestern University & Rice University ($3,500) who focused their plan on increasing Hispanic/Latinx involvement in clinical trials by training community healthcare workers from refugee and immigrant populations.

 

A white paper with all of the presentations will be published in April and rolled out during BioNJ’s acclaimed BioPartnering Conference – which brings together more than 500 industry professionals from around the globe. The three winning teams will present their plans during the April 18 BioPartnering Conference and all of the students are invited to attend to meet the community.

 

Please click here for the official press release.

Click here for a photo gallery from the competition.


BioNJ is inspired by and privileged to work with those committed to improving the lives of Patients and their families around the world. For more information, visit BioNJ.org/Health-Equity-Clinical-Trials.


Because Patients Can't Wait®,

The BioNJ Team

Welcome New Members


 Learn more at www.BioNJ.org/Membership.

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Upcoming BioNJ Events

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BioNJ's Patient Advocacy Summit

Attendance is exclusive to Patient Advocacy Groups and R&D company Patient advocacy and government affairs professionals.

Register Today!

December 13, 2022

BioNJ's 30th Annual Dinner Meeting & Innovation

Celebration

Register Today!

February 2, 2023

Putting Patients First:

The Value of Medical Innovation

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New Data Show How Insurers and Middlemen Shift Rx Costs Onto Patients

There is only one place in the health care system where middlemen refuse to share negotiated prices with patients, and that’s at the pharmacy counter. A new report reveals how this harmful tactic leads to higher costs for patients. Health insurers and pharmacy benefit managers (PBMs) negotiate rebates and discounts that lower the price they pay for prescription medicines. The problem is they don’t always share these lower prices with patients who rely on prescription medicines. Instead, a patient in their deductible shows up at the pharmacy and is required to pay the full list price of the medicine. Insurers and PBMs also require patients with coinsurance to pay based on the full price of the treatment by charging them a percentage of the medicine’s full list price. A new report, based on an IQVIA analysis of health care spending data of commercially insured patients taking brand medicines, shows how these tactics are increasingly shifting the cost of medicines to patients.

Putting Delaware Patients First: Protecting Patient Assistance Programs

Patients with chronic health conditions in Delaware are seeing a new wave of relief thanks to Governor John Carney, members of the Delaware legislature and more than 40 patient groups. The recently passed SB267 will make it easier for vulnerable patients to be able to afford their brand medications. Insurers and middlemen, like pharmacy benefit managers (PBMs), are increasingly shifting more costs to patients through high deductibles and coinsurance. Patients arrive at the pharmacy counter to fill their prescriptions only to find out that their insurance coverage isn’t what they expected it to be. To help fill the gap, biopharmaceutical companies offer cost-sharing assistance (also known as copay coupons) and other forms of patient assistance.

IRA's Impact on Biopharma Innovation With Amitabh Chandra

With the U.S. Inflation Reduction Act now law, many in Washington DC are saying that it’s provisions reducing pricing by $80 billion dollars annually in Medicare for drugs near the end of their patent life will not have any negative impacts on the U.S. biopharma ecosystem. Enter Amitabh Chandra, the Director of Health Policy Research at the Harvard Kennedy School of Government, and a professor of business administration at Harvard Business School. Dr Chandra has been sounding the alarm of the unintended consequences of the many pricing bills that have been emerging from both the Trump and Biden Administrations, as well as the U.S. Congress. 

60% of FDA Approved Medicines from 2011- 2020 Originated in the United States

New research from the consultancy Vital Transformation (VT) has found that 60% of FDA approved medicines from 2011- 2020 originated in the United States. The FDA approved 363 new medicines over that time period, and the U.S. was solely responsible for the origination of 223 of those medicines. The study by Vital Transformation was an update of the often-cited 2010 Nature Reviews Drug Discovery publication by Robert Kneller. Broadly, since Kneller’s publication, the U.S. biopharma sector increased the total number of approved therapies that it originated from 118 to 223. Germany, the UK and the EU broadly have seen declines in the number of therapies it originated during the same period.

The Health Insurer Will See You Now: How UnitedHealth is Keeping More Profits, As Your Doctor

Next year, UnitedHealth Group — one of the largest health care companies on the planet — expects to make a lot more money in a relatively simple way: by funneling more of the insurance premiums it collects from workers and taxpayers toward itself. That’s possible because UnitedHealth, known for its sprawling insurance presence, has pivoted to become one of the largest providers of outpatient care by acquiring numerous physician practices, surgery centers, urgent care facilities, and pharmacy benefit managers. And whenever possible, UnitedHealth is directing more of its insurance members to get care and prescription drugs through those entities that are owned by its Optum division — essentially allowing its left hand to pay its right.

BioNJ in the News

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Inaugural Health Equity in Clinical Trials Business Plan Competition Held

BioNJ recently hosted an Inaugural Health Equity in Clinical Trials MBA Business Plan Case Competition on Saturday, Dec. 3, at Rutgers Business School. BioNJ’s Business Plan Case Competition, which is part of a broader BioNJ strategic initiative of Health Equity in Clinical Trials, is designed to promote the next generation of diverse clinical trial innovators and identify innovative approaches and successful models that can be used to strengthen diversity in clinical trials and expand health equity. BioNJ believes that health equity in clinical trials is critical to deepening the understanding of the safety and efficacy of medicines in under-represented populations, expanding access to medical innovation for these populations, and increasing dialogue and trust among the biopharma industry, communities and healthcare systems.

New CSIT Clean Tech Demonstration Program to Support Technology Start-ups Testing Their Prototypes

The New Jersey Commission on Science, Innovation and Technology (CSIT) announced plans to launch a competitive $2.5 million program to support New Jersey companies accelerating the commercialization and deployment of innovative clean energy technologies. The Pilot Clean Tech Demonstration Program will provide grants of up to $250,000 each to help startups develop their technologies intended to create a cleaner and greener future for New Jersey. “In addition to their innovations in the life sciences and technology sectors, New Jersey innovators are creating technologies that are intended to combat climate change and reduce carbon footprints worldwide,” said CSIT Chair Debbie Hart. “By providing resources necessary to support entrepreneurs as they bring their products to the marketplace, we are not only helping early stage New Jersey companies succeed, but also helping to build the green economy that Governor Phil Murphy champions.”

NJ Company News

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Ferring Receives U.S. FDA Approval for REBYOTA® (fecal microbiota, live-jslm) – A Novel First-in-Class Microbiota-Based Live Biotherapeutic

BioNJ Member Ferring Pharmaceuticals, with offices in Parsippany, announced the U.S. Food and Drug Administration (FDA) approved REBYOTA® (fecal microbiota, live-jslm), a novel first-in-class microbiota-based live biotherapeutic indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI. The FDA approval of REBYOTA is based on the results from the clinical program including the randomized, double-blind, placebo-controlled Phase 3 PUNCH™ CD3 trial in which a single dose of REBYOTA demonstrated superiority to placebo as a treatment to reduce recurrence of CDI after standard-of-care antibiotic treatment. 

Tevogen Bio to Develop T Cell Therapies for Treatment of Epstein-Barr Virus Related Cancers and Multiple Sclerosis

Warren-based BioNJ Member Tevogen Bio announced its intention to study potential therapeutic use of its allogeneic genetically unmodified precision T cell technology in multiple sclerosis (MS) by developing Epstein-Barr virus (EBV) specific CD8+ cytotoxic T lymphocytes (CTL). These CTLs will also be studied for the potential use in EBV-related cancers, such as nasopharyngeal carcinoma and certain lymphomas. Tevogen’s next generation precision T cell platform is designed to provide increased immunologic specificity to eliminate malignant and virally infected cells, while allowing healthy cells to remain intact. Multiple targets are selected in advance with the goal of overcoming the mutational capacity of cancer cells and viruses which can otherwise allow for escape from immunologic targeting.

Celldex Therapeutics Presents Positive Data from Barzolvolimab Phase 1b Study in Chronic Inducible Urticaria at Global Urticaria Forum 2022 Dec 6, 2022

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced new data from the company’s open label Phase 1b clinical trial of barzolvolimab in patients with antihistamine refractory chronic inducible urticarias are being presented at the GA²LEN Global Urticaria Forum. These diseases, which are often severe and debilitating, can significantly impact patients’ lives. Barzolvolimab is a humanized monoclonal antibody that binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity. All 9 of 9 (100%) cold urticaria patients who received a 1.5 mg/kg single full dose of barzolvolimab experienced a complete response as assessed by provocation testing, including 4 of 4 patients with omalizumab refractory disease. 

Brazilian Regulatory Authority Grants Approval for Waylivra™ for Familial Partial Lipodystrophy

South Plainfield-based BioNJ Member PTC Therapeutics announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This is the first approval globally for Waylivra for the FPL indication. Waylivra is also approved in Brazil for the treatment of Familial Chylomicronemia Syndrome (FCS). "Waylivra's approval as the first treatment for FPL is an important milestone for FPL patients in Brazil," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "This approval is another example of PTC leveraging its strong capabilities in Latin America to bring first of its kind transformational therapies to patients with high unmet needs."

Genmab Announces U.S. Food and Drug Administration Accepts for Priority Review Biologics License Application (BLA) for Epcoritamab (DuoBody®-CD3xCD20) for the Treatment of Relapsed/Refractory Large B-Cell Lymphoma (LBCL)

Princeton-based BioNJ Member Genmab announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for subcutaneous epcoritamab (DuoBody®-CD3xCD20), an investigational bispecific antibody, for the treatment of patients with relapsed/refractory large B-cell lymphoma (LBCL) after two or more lines of systemic therapy. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 21, 2023. The BLA submission is based on safety and preliminary efficacy data from the LBCL cohort of the pivotal EPCORE™ NHL-1 open-label, multi-center phase 2 clinical trial evaluating epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin lymphoma (B-NHL). 

BeiGene to Present Final PFS Results from ALPINE Trial Demonstrating Superior PFS for BRUKINSA® Versus IMBRUVICA® in Late-Breaking Oral Session at ASH 2022

BioNJ Member BeiGene, with a site under development in Hopewell, announced that the results of the final progression free survival (PFS) analysis of the ALPINE trial will be presented at the 64th American Society of Hematology Annual Meeting. ALPINE is a global Phase 3 trial comparing BRUKINSA (zanubrutinib) with IMBRUVICA® (ibrutinib) in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL). In this final PFS analysis, BRUKINSA achieved superior PFS compared with ibrutinib, as assessed by both Independent Review Committee (IRC) and investigator (HR: 0.65 [95% CI, 0.49-0.86] p =.0024, for both investigator and IRC). The PFS results favored zanubrutinib consistently across major pre-defined subgroups including IGHV status and patients with del(17p)/TP53, regardless of IRC or investigator assessment. 

Kyowa Kirin and MEI Pharma Announce Topline Data from the Phase 2 MIRAGE Study Evaluating Zandelisib in Patients With Indolent B-cell Non-Hodgkin’s Lymphoma in Japan

BioNJ Member Kyowa Kirin Co., with a site in Bedminster, announced topline data from the Phase 2 MIRAGE study evaluating zandelisib, an orally administered investigational phosphatidylinositol 3-kinase delta ("PI3Kδ") inhibitor, in patients with indolent B-cell Non-

Hodgkin’s Lymphoma (iB-NHL) without small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL), and Waldenström's macroglobulinemia (WM) in Japan. The data demonstrated a 75.4% objective response rate (ORR) and 24.6% of patients achieved a complete response (CR) as determined by Independent Review Committee (IRRC) assessment (n=61). The data is currently insufficiently mature to accurately estimate duration of response (DOR). With 9.5 months median duration of follow-up, a discontinuation rate due to any treatment emergent adverse event was 14.8%.

AbbVie Announces European Commission Approval of SKYRIZI® (risankizumab) for the Treatment of Moderate to Severe Active Crohn's Disease

BioNJ Member AbbVie, with a site in Madison, announced the European Commission (EC) approved SKYRIZI® (risankizumab, 600 mg intravenous [IV] induction and 360 mg subcutaneous [SC] maintenance therapy) as the first specific interleukin-23 (IL-23) inhibitor for the treatment of adults with moderately to severely active Crohn's disease who have had inadequate response, lost response or were intolerant to conventional or biologic therapy. The EC approval for SKYRIZI in Crohn's disease is supported by results from the global Phase 3 program, which included three studies: ADVANCE induction, MOTIVATE induction and FORTIFY maintenance. The three Phase 3 studies are multicenter, randomized, double-blind, placebo-controlled studies and include assessments of efficacy, safety and tolerability of SKYRIZI.

AbbVie Launches Strategic Collaboration with HotSpot Therapeutics to Further Expand Immunology Pipeline

BioNJ Member AbbVie, with a site in Madison, and HotSpot Therapeutics, Inc. announced an exclusive worldwide collaboration and option to license agreement for HotSpot's discovery-stage IRF5 program for the treatment of autoimmune diseases. IRF5 is a transcription factor that acts as a key regulator of certain types of immune responses, and its dysregulation is strongly implicated in several poorly treated autoimmune disorders. Efforts to modulate IRF5 using conventional small molecule approaches have been unsuccessful because IRF5 lacks a traditional active site. Leveraging its proprietary Smart Allostery™ platform, HotSpot discovered what it believes to be the first and only disclosed small molecule IRF5 inhibitor that targets a previously unknown allosteric pocket on the protein that is critical for its endogenous regulation – a "natural hotspot".

Gilead and Arcus Biosciences Announce Positive Update on Joint TIGIT Program from Interim Analysis of ARC-7 Study in Non-Small Cell Lung Cancer

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, and Arcus Biosciences announced a positive update from the fourth interim analysis of the randomized, open-label Phase 2 ARC-7 study in patients with first-line metastatic non-small cell lung cancer (NSCLC) with PD-L1 tumor proportion score (TPS) ≥50% without epidermal growth factor receptor or anaplastic lymphoma kinase (EGFR/ALK) mutations. ARC-7 is evaluating the combinations of anti-TIGIT antibody domvanalimab plus anti-PD-1 antibody zimberelimab (doublet), and domvanalimab plus zimberelimab and etrumadenant, an A2a/b adenosine receptor antagonist (triplet), versus zimberelimab alone, and represents the first randomized Phase 2 study of an Fc-silent anti-TIGIT/anti-PD-1 combination. The protocol-specified fourth interim analysis was conducted when the trial reached full enrollment, with a clinical data cutoff date of August 31, 2022. 

European Commission Grants Expanded Marketing Authorization for Gilead’s Biktarvy® for the Treatment of HIV in Pediatric Populations

BioNJ Member Gilead Sciences, Inc. with a site in Morris Plains, announced that the European Commission (EC) has authorized a new low-dose tablet dosage form of Biktarvy® (bictegravir 30 mg/emtricitabine 120 mg/tenofovir alafenamide 15 mg tablets) and an extension of the indication for Biktarvy to treat HIV infection in virologically suppressed children who are at least two years of age and weigh at least 14 kg. The European Marketing Authorization is the first pediatric approval for Biktarvy in the European Union (EU) and applies to all 27 member states of the EU, as well as Norway, Iceland and Liechtenstein. While there have been many advances in the treatment of HIV in children and adolescents, there remains a need to prioritize, evaluate and develop options for the millions of children worldwide.

Otsuka and Sunovion Initiate Clinical Development of Ulotaront for the Adjunctive Treatment of Major Depressive Disorder

BioNJ Member Otsuka Pharmaceutical Development & Commercialization, Inc., with a site in Princeton, and Sunovion Pharmaceuticals Inc. announced that the first patient has been enrolled in a Phase 2/3 clinical study to evaluate ulotaront (SEP-363856), a trace amine-associated receptor 1 (TAAR1) agonist with 5-HT1A agonist activity, as an adjunctive therapy in the treatment of adults living with major depressive disorder (MDD). Ulotaront, which is also being evaluated in Phase 3 clinical development for the treatment of schizophrenia, is the first TAAR1 agonist to be studied as an adjunctive therapy in the treatment of MDD. Patients with an inadequate response to current antidepressant treatment will be randomized to receive ulotaront or placebo in addition to their antidepressant therapy.

Otsuka Pharmaceutical and Lundbeck Present Positive Data from Multiple Phase 3 Studies Showing Brexpiprazole Significantly Improved Symptoms of Agitation in Patients With Alzheimer's Dementia at the 2022 Clinical Trials for Alzheimer’s

BioNJ Member Otsuka America Pharmaceutical, Inc., with a site in Princeton, and Lundbeck US presented positive efficacy and safety data from multiple Phase 3 studies of brexpiprazole in the treatment of agitation in patients with Alzheimer's dementia at the Clinical Trials for Alzheimer’s Disease (CTAD) Congress. Data from two Phase 3 studies showed that brexpiprazole 2 mg or 3 mg doses significantly (p<0.05) improved symptoms of agitation in patients with Alzheimer’s dementia compared to placebo. The primary endpoint across the studies was improvement from baseline in the Cohen-Mansfield Agitation Inventory (CMAI) total score while the key secondary endpoint was an improvement from baseline to week 12 in the Clinical Global Impression-Severity (CGI-S) score related to agitation. Brexpiprazole also demonstrated a favorable safety and tolerability profile in all three studies. 

European Commission Approves Enjaymo® (sutimlimab) for Treatment of Hemolytic Anemia in Adult Patients With Cold Agglutinin Disease

Bridgewater-based BioNJ Member Sanofi announced the European Commission (EC) has granted marketing authorization for Enjaymo® (sutimlimab) for the treatment of hemolytic anemia in adult patients with cold agglutinin disease (CAD), a rare, serious and chronic autoimmune hemolytic anemia, where the body’s immune system mistakenly attacks healthy red blood cells and causes their rupture, known as hemolysis. Enjaymo is currently the only approved treatment for CAD and is a first-in-class humanized monoclonal antibody that is designed to selectively target and inhibit the classical complement pathway specific serine protease, C1s. It will be available as a 50mg/mL solution for infusion.

PERLA Phase II Trial of Jemperli (dostarlimab) Plus Chemotherapy Shows Positive Results in First-line Metastatic Non-squamous Non-small Cell Lung Cancer

Warren-based BioNJ Member GSK announced results from the PERLA Phase II clinical trial investigating dostarlimab in combination with chemotherapy versus pembrolizumab in combination with chemotherapy as a first-line treatment for patients with metastatic non-squamous non-small cell lung cancer (NSCLC). Dostarlimab plus chemotherapy achieved very promising results for the primary endpoint of confirmed objective response rate (ORR) as well as for the key secondary endpoint of median progression-free survival (mPFS). The PERLA Phase II trial is a randomised, double-blind trial of 243 patients and is the largest global head-to-head trial of programmed death receptor-1 (PD-1) inhibitors in this patient population. 

U.S. FDA Accepts for Priority Review the Biologics License Application for Pfizer’s Respiratory Syncytial Virus Vaccine Candidate for the Prevention of RSV Disease in Older Adults

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) accepted for priority review a Biologics License Application (BLA) for its respiratory syncytial virus (RSV) vaccine candidate, PF-06928316 or RSVpreF, as submitted for the prevention of lower respiratory tract disease caused by RSV in individuals 60 years of age and older. Priority Review designation by the FDA reduces the standard BLA review period by four months. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA on the RSVpreF application is in May 2023. This decision follows the FDA’s Breakthrough Therapy Designation of RSVpreF in older adults in March 2022.

Pfizer and Clear Creek Bio to Collaborate on a Research Program Targeting SARS-CoV-2 Papain-Like Protease

BioNJ Member Pfizer, with a site in Peapack, and Clear Creek Bio, Inc. announced a research collaboration and exclusive license agreement to advance the discovery and development of potential inhibitors of the SARS-CoV-2 papain-like protease (PLpro) for the oral treatment of COVID-19. PLpro is an essential enzyme, which, along with the main protease (Mpro), plays an important role in viral replication. This program will expand Pfizer’s innovative anti-infective pipeline and, if successful, will complement Pfizer’s existing portfolio of COVID-19 products with direct-acting antiviral agents against different SARS-CoV-2 targets.

Pfizer and BioNTech Report New Data on Omicron BA.4/BA.5-Adapted Bivalent Booster Demonstrating Improved Immune Response Against Emerging Omicron Sublineages

BioNJ Member Pfizer, with a site in Peapack, and BioNTech announced results from an analysis examining the immune response induced by their Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine [Pfizer-BioNTech COVID-19 Vaccine, Bivalent (Original and Omicron BA.4/BA.5)] against newer Omicron sublineages, including BA.4.6, BA.2.75.2, BQ.1.1 and XBB.1. These data indicate that the companies’ bivalent vaccine elicits a greater increase in neutralizing antibody titers than the companies’ original COVID-19 vaccine against these emerging Omicron sublineages. Based on these findings, the Omicron BA.4/BA.5-adapted bivalent booster may help to provide improved protection against COVID-19 due to Omicron BA.4 and BA.5 sublineages as well as new sublineages that continue to increase in prevalence.

Novartis and Medicines for Malaria Venture Announce Decision to Move to Phase 3 Study for Novel Ganaplacide/Lumefantrine-SDF Combination in Adults and Children With Malaria

As the threat of resistance to current malaria treatments grows, East Hanover-based BioNJ Member Novartis and MMV announce the decision to progress ganaplacide/lumefantrine- solid dispersion formulation (SDF) into Phase 3 development for the treatment of patients with acute uncomplicated malaria due to Plasmodium falciparum. Ganaplacide is a novel agent with a new mechanism of action, which is combined with a new formulation of lumefantrine optimized for once-daily dosing. This combination has the potential not only to clear malaria infection, including artemisinin-resistant strains, but also to block the transmission of the malaria parasite. The medicine is being developed with scientific and financial support from MMV and their partners.

Novartis Pluvicto™ Shows Statistically Significant and Clinically Meaningful Radiographic Progression-Free Survival Benefit in Patients With PSMA–Positive Metastatic Castration-Resistant Prostate Cancer

East Hanover-based BioNJ Member Novartis announced the pivotal Phase III PSMAfore study with Pluvicto (INN: lutetium (177Lu) vipivotide tetraxetan), a prostate-specific membrane antigen (PSMA)-targeted radioligand therapy, met its primary endpoint. Pluvicto demonstrated a statistically significant and clinically meaningful improvement in radiographic progression-free survival (rPFS) in patients with PSMA–positive metastatic castration-resistant prostate cancer (mCRPC) after treatment with androgen-receptor pathway inhibitor (ARPI) therapy, compared to a change in ARPI1. No unexpected safety findings were observed in PSMAfore; data are consistent with the already-well established safety profile of Pluvicto.

Phase III Trial Demonstrated Jardiance® is the First SGLT2 Inhibitor to Show Statistically Significant Reduction in Blood Sugar Levels in Children and Adolescents With Type 2 Diabetes

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced The DINAMO Phase III clinical trial met its primary endpoint by demonstrating a statistically significant reduction in HbA1c (a marker of average blood sugar) with Jardiance® (empagliflozin) compared with placebo for children and adolescents aged 10-17 years living with type 2 diabetes. When Jardiance was added to other baseline treatments (diet, exercise, metformin and/or insulin) HbA1c was reduced by 0.84% compared with placebo at week 26 (95% CI –1.50 to –0.19; P=0.012). The DINAMO (DIabetes study of liNAgliptin and eMpagliflozin in children and adOlescents) trial included youth aged 10-17 years with type 2 diabetes and HbA1c ≥6.5% and ≤10.5%. Participants were randomly assigned treatment with Jardiance (10 or 25 mg) (n=52), Tradjenta® (linagliptin) (5 mg) (n=53) or placebo (n=53) once daily. 

Lilly Announces Updated Data from the Verzenio® (abemaciclib) Phase 3 monarchE Trial Presented at SABCS and Simultaneously Published in The Lancet Oncology

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced updated results from the pivotal Phase 3 monarchE trial of adjuvant Verzenio® (abemaciclib) in combination with standard endocrine therapy (ET) for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-), node-positive, high risk early breast cancer (EBC). These data, which include results for investigational uses in the intent-to-treat (ITT) and Cohort 1 populations, were published in The Lancet Oncology. Data include updated results from a prespecified analysis reflecting a median follow-up of 3.5 years, with all patients having now discontinued or completed the two-year Verzenio treatment period.

Lilly Completes Acquisition of Akouos Expanding Efforts to Help People With Genetic Diseases

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced the successful completion of its acquisition of Akouos, Inc. The acquisition expands Lilly's efforts in genetic medicines to include Akouos's portfolio of potential first-in-class adeno-associated viral gene therapies for the treatment of inner ear conditions, including sensorineural hearing loss. "Akouos brings more top-tier talent and an important pipeline to Lilly's Institute for Genetic Medicine that will further accelerate our work to advance genetic medicines for people living with difficult-to-treat diseases," said Andrew C. Adams, Ph.D., Senior Vice President of Genetic Medicine at Lilly and Co-Director of the Institute for Genetic Medicine. 

Lilly Shares Positive Donanemab Data in First Active Comparator Study in Early Symptomatic Alzheimer's Disease

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that donanemab met all primary and secondary endpoints for the 6-month primary outcome analysis in the Phase 3 TRAILBLAZER-ALZ 4 study, providing the first active comparator data on amyloid plaque clearance in patients with early symptomatic Alzheimer's disease treated with amyloid-targeting therapies. This data comparing donanemab to Aduhelm® * (aducanumab-avwa) to assess superiority on amyloid plaque reduction were shared at the 15th Clinical Trials on Alzheimer's Disease (CTAD) Conference. Donanemab is an investigational antibody that targets a modified form of beta amyloid plaque called N3pG. Through the FDA's accelerated approval pathway, the FDA has recognized that the reduction of amyloid beta plaque is a biomarker reasonably likely to predict clinical benefit in the treatment of early Alzheimer's disease.

Merck Announces Phase 3 KEYNOTE-859 Trial Met Primary Endpoint of Overall Survival in Patients With HER2-Negative Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma

Kenilworth-based BioNJ Member Merck & Co. announced positive topline results from the pivotal Phase 3 KEYNOTE-859 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy for the first-line treatment of patients with human epidermal growth factor receptor 2 (HER2)-negative locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma. KEYTRUDA in combination with chemotherapy showed a statistically significant and clinically meaningful improvement in the trial’s primary endpoint of overall survival (OS) versus chemotherapy alone in the all-randomized patient population at a pre-specified interim analysis conducted by an independent Data Monitoring Committee. Statistically significant and clinically meaningful improvements in progression-free survival (PFS) and overall response rate (ORR) were also observed in the all-randomized patient population. 

Merck to Acquire Imago BioSciences, Inc.

Kenilworth-based BioNJ Member Merck & Co. and Imago BioSciences, Inc. announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Imago for $36.00 per share in cash for an approximate total equity value of $1.35 billion. “We continue to invest in our pipeline with a focus on applying our unique capabilities to unlock the value of breakthrough science for the patients we serve,” said Robert M. Davis, President and Chief Executive Officer, Merck. “This acquisition of Imago augments our pipeline and strengthens our presence in the growing field of hematology.” Imago is a clinical stage biopharmaceutical company developing new medicines for the treatment of myeloproliferative neoplasms (MPNs) and other bone marrow diseases. 

SEZABY™ (phenobarbital sodium powder for injection) Approved by U.S. FDA for the Treatment of Neonatal Seizures

Sun Pharmaceutical Industries Limited, with a site in Princeton, announced that the U.S. Food and Drug Administration has approved SEZABY™ (phenobarbital sodium powder for injection) for the treatment of neonatal seizures. With this approval, SEZABY becomes the first and only product specifically indicated in the U.S. for the treatment of neonatal seizures in term and preterm infants. SEZABY is expected to be available in the U.S. in Q4FY23. SEZABY is a benzyl alcohol-free and propylene glycol-free formulation of phenobarbital sodium powder for injection. It was granted orphan drug designation by the U.S. FDA for the treatment of neonatal seizures. SEZABY was approved based on the results of NEOLEV2, a Phase 2 study that evaluated levetiracetam compared to phenobarbital in the first-line treatment of neonatal seizures.

LEO Pharma Announces Positive Phase 3 Topline Results from DELTA 1 Trial With Delgocitinib Cream in Adults With Moderate to Severe Chronic Hand Eczema (CHE)

LEO Pharma, with a site in Madison, announced positive results of the DELTA 1 trial. DELTA 1 is the first of two pivotal phase 3 clinical trials with delgocitinib cream, an investigational topical pan-Janus kinase (JAK)-inhibitor, for the potential treatment of adults with moderate to severe chronic hand eczema (CHE). The trial met its primary endpoint with a statistically significant improvement in CHE after 16 weeks of treatment compared to vehicle and the treatment was generally well-tolerated. All or most of the signs and symptoms of CHE were cleared early in the treatment period in a significantly larger proportion of subjects treated with delgocitinib cream compared to subjects treated with vehicle.

ENHERTU® Approved in Japan for Patients With Previously Treated HER2 Positive Metastatic Breast Cancer

Basking Ridge-based Daiichi Sankyo announced that ENHERTU® (trastuzumab deruxtecan) has been approved in Japan for the treatment of adult patients with HER2 positive unresectable or recurrent breast cancer after prior chemotherapy, which includes trastuzumab and a taxane. The approval by Japan’s Ministry of Health, Labour and Welfare (MHLW) was based on the results of the DESTINY-Breast03 trial where ENHERTU demonstrated a 72% reduction in the risk of disease progression or death compared to trastuzumab emtansine (T-DM1) (hazard ratio [HR] = 0.28; 95% confidence interval [CI]: 0.22-0.37; p<0.000001) in patients with HER2 positive unresectable and/or metastatic breast cancer previously treated with trastuzumab and a taxane. 

SJP Completes Eisai’s 300K sq. ft. HQ at ON3 in Nutley

SJP Project Solutions on Wednesday announced the buildout of Eisai’s new U.S. headquarters at ON3 in Nutley is now complete. The pharmaceutical company relocated from Woodcliff Lake, taking the entirety of 200 Metro Blvd. at ON3 and moving the entirety of its staff of approximately 1,200 corporate and research & development workers to the new headquarters in the first quarter of 2022. “New Jersey has a long history of serving as an incubator for some of the most innovative companies in the pharmaceutical and life sciences spaces, and Eisai’s new headquarters at ON3 will set the standard for the future of the industry’s workplaces,” Andrew Natale, Chief Operating Officer at SJP Project Solutions, stated. “We are proud to have helped deliver a best-in-class headquarters that will support Eisai’s peerless standard of research and development, as well as its relentless focus on promoting teamwork, safety and innovation in the office.”

Eisai Announces Approval of Partial Change to Label for Dosage and Administration of Aricept® for Treatment of Dementia With Lewy Bodies

Nutley-based Eisai Co., Ltd. announced that its application for a partial change to label for Aricept® (donepezil hydrochloride), a treatment for Alzheimer’s disease and dementia with Lewy bodies (DLB) that was discovered and developed in-house, regarding dosage and administration for the treatment of DLB in Japan, has been approved. This partial change to label is based on the results of a reexamination for the indication of "suppression of progression of dementia symptoms in dementia with Lewy bodies" of this drug, which was categorized as Category 2. Eisai will continue to place the highest priority on the provision of proper use and safety information for this drug, and will make continued contributions to address the diversified needs of, and increase the benefits provided to people living with DLB and their families.

New Evidence: Positive Impact of Blue Light Cystoscopy on Recurrence in Bladder Cancer Presented at the Annual SUO Meeting

Photocure ASA, The Bladder Cancer Company, with a site in Princeton, announced that new evidence demonstrating that Blue Light Cystoscopy (BLC®) decreases the risk and prolongs time to recurrence based on Real World Evidence from the U.S. multi-institutional Blue Light Cystoscopy with Cysview® Registry database was presented. This large, prospective, longitudinal, multisite registry collects data on NMIBC patients who have undergone transurethral resection of bladder tumor (TURBT) using Blue Light as an adjunct to white light cystoscopy (WLC). The new data represented an interim analysis of the study, which is projected to enroll 4,400 patients in total. In this study, all registry patients analyzed (n=2055) received Blue Light. They were directly compared to a historical White-Light (WL)-only control group (n=261) of similar age, stage, grade, and clinical management (i.e., intravesical therapy). 

UroGen Announces New Data from the OLYMPUS Trial that Shows Median Durability of Response of 28.9 Months for JELMYTO®, the Only Non-Surgical, Chemoablative Treatment for Adults with Low-Grade Upper Tract Urothelial Cancer

UroGen Pharma Ltd., with a site in Princeton, announced new data from the OLYMPUS registration trial designed to obtain long-term follow-up data on JELMYTO® (mitomycin) for pyelocalyceal solution that shows median durability of response (DOR) of 28.9 months. The study was presented at SUO. “The clinical benefit of JELMYTO was demonstrated in the Phase 3 OLYMPUS study and data presented highlighted the long-term durability of that benefit,” said Dr. Phillip Pierorazio, M.D., Chief, Section of Urology at Penn Presbyterian Medical Center. “JELMYTO provides an effective and durable kidney-sparing treatment option and should be considered as primary therapy for adult patients with LG-UTUC.” Outcomes of interest include DOR in patients who remain in complete response (CR) at the end of OLYMPUS, events of disease recurrence and progression, post-study treatments and death.

UroGen Announces New Data from the OPTIMA II Study that Show Median Durability of Response of 24.4 Months for UGN-102, an Investigational Non-Surgical Chemoablative Treatment for Low-Grade, Intermediate-Risk Non-Muscle Invasive Bladder Cancer

UroGen Pharma Ltd. with a site in Princeton, announced new data from the OPTIMA II study designed to obtain long-term follow-up data on UGN-102 that shows median duration of response (DOR) of 24.4 months for UroGen’s investigational drug UGN-102 currently in Phase 3 development for low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The study (Abstract #193) was presented at SUO. “LG-IR-NMIBC is a challenging disease to treat because of the high recurrence rate managed by repetitive surgeries,” says William C. Huang, M.D., FACS, Professor and Vice Chair of Urology at NYU Langone Health and Principal Investigator of the OPTIMA II trial. “The OPTIMA II Phase 2b trial showed significant tumor response benefit for patients using the novel chemoablative therapy UGN-102 for LG-IR-NMIBC and this latest analysis shows that the treatment benefit lasted for more than two years. I look forward to additional data on UGN-102, including evidence from the ENVISION Phase 3 study.” 

People in the News

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Princeton-Based Otsuka, Mental Health Leader, Names Kraus Chief Medical Officer

Dr. John Kraus has been named Executive Vice President and Chief Medical Officer of Otsuka, the Princeton-based company that is a leader in the mental health space. Since joining Otsuka in July 2021 as Vice President of Global Clinical Development, Central Nervous System, Dr. Kraus has played an instrumental role in leading the company’s CNS research and development efforts. With nearly 20 years of leadership experience in the pharmaceutical industry, Dr. Kraus leverages his scientific and medical expertise to lead drug development programs, offer strategic oversight and collaborate cross-functionally to optimize clinical trials.

Teva Announces Appointment of Richard Francis as President and CEO

Parsippany-based BioNJ Member Teva Pharmaceutical announced that the company’s Board of Directors has appointed Richard Francis as President and Chief Executive Officer. Mr. Francis is a seasoned pharmaceutical executive, bringing more than two and a half decades of experience to Teva, including five years as Chief Executive Officer of Sandoz and a member of the Novartis Executive Team. Prior to his role at Sandoz, Mr. Francis was a senior executive at Biogen for 13 years where he held a number of senior roles, including leading the U.S. business. Mr. Francis is currently the CEO of Purespring Therapeutics, a pioneering gene therapy company focused on transforming the treatment of kidney diseases, and CEO of Forcefield Therapeutics, a pioneer of best-in-class therapeutics to protect heart function. 

Funding

New Jersey Manufacturing Voucher Program Launched

The New Jersey Economic Development Authority (NJEDA) has announced its new pilot grant program that awards New Jersey Manufacturers up to $20,000,000. This grant program will assist manufacturers with acquiring the equipment they need to be able to be more effective, productive and profitable. Each manufacturer can be awarded a maximum of $250,000 for eligible equipment costs. It's important to note that NJ manufacturers planning to apply for this program must be requesting assistance for a project they are currently considering but have not yet committed to. Projects not eligible for this grant include: projects where contracts have been signed, a Purchase Order placed, a deposit made in advance or previously purchased equipment.  

New Jersey Innovation Fellows Program Will Provide Grant Funding and Mentorship to First-Time Entrepreneurs

The New Jersey Economic Development Authority (NJEDA) announced plans to create a competitive program – the New Jersey Innovation Fellows (NJIF), which will offer mentorship and grants of up to $400,000 to teams of first-time entrepreneurs. The program will provide income replacement so that entrepreneurs can devote their full-time attention to building out their startups. Through the NJIF Program, the NJEDA will support teams of at least three entrepreneurs with mentorship, training, and income replacement capital over a two-year period. The grant funding is designed to replace a stream of income an entrepreneur might forgo to launch an early stage business. The NJEDA anticipates that support from the NJIF Program will attract innovative ideas and entrepreneurs who would otherwise be unable to pursue the launch of a new venture due to the needs for income or, in the case of a recent graduate, who would choose to accept employment in lieu of entrepreneurship due to socio-economic constraints.

Academia/Institutions/Incubators

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Research With NJ Portal Gives Entrepreneurs and Investors Access to Research at New Jersey’s World-Class Universities

The New Jersey Commission on Science, Innovation and Technology (CSIT) announced the expansion of ResearchwithNJ.com to provide local, national and international innovation communities with insight into groundbreaking research being conducted at five New Jersey universities – Montclair State University, New Jersey Institute of Technology (NJIT), Princeton University, Rowan University and Rutgers, the State University of New Jersey. The Commission also announced that it has taken over management of the portal from the New Jersey Economic Development Authority (NJEDA) and the New Jersey Office of the Secretary of Higher Education (OSHE) due to its close ties to both the startup and academic communities.

BioCentriq Welcomes New Chief Quality and Compliance Officer

Newark-based BioCentriq, a cell and gene therapy manufacturing company, said that Jackie Veivia-Panter has been named its new Chief Quality and Compliance Officer. Ms. Veivia-Panter has 30 years of experience in the pharmaceutical and cell therapy industry with specialized expertise in quality management. Her skills include designing and delivering effective and compliant solutions, processes and organizational models. Throughout her career, she has managed site quality and research & development quality and has held global roles in training, regulatory intelligence and matrix organization management. “BioCentriq is at the forefront of developing and manufacturing cell and gene therapies, and I’m grateful for the opportunity to help usher it into its next era as a leading CDMO,” Ms. Veivia-Panter said.

Top Cities for Careers in Tech: Newark, Jersey City Among Top 5 Nationally

Newark and Jersey City both ranked among the Top 5 cities nationally for those seeking careers in technology, according to research done by the educational resource website edwize.org. Newark came in at No. 3 overall. Jersey City is at No. 5. Newark and Jersey City dominated their larger counterparts: Boston (No. 7), Seattle (No. 10), Austin, Texas (No. 27), and New York City (No. 36). San Jose, California, was No. 28; San Francisco came in at No. 48.

Hackensack Meridian School of Medicine Gets Full Accreditation by MSCHE

The Hackensack Meridian School of Medicine has been granted full accreditation by the Middle States Commission on Higher Education — a milestone moment that brings the school closer to full accreditation from all regulatory agencies. The MSCHE, which is responsible for more than 515 accredited and candidate degree-granting institutions, conferred its accreditation after a visit this past summer. The next milestone for the school is final accreditation from the Liaison Committee on Medical Education, the major national regulatory body. Hackensack Meridian Health CEO Robert Garrett said, “This is further validation of how the Hackensack Meridian School of Medicine continues to meet and surpass all expectations, while training incredible doctors of the future.” 

Events

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New Jersey Academic Drug Discovery Consortium on Infectious Diseases

December 16, 2022

The New Jersey Academic Drug Discovery Consortium is delighted to host the 2022 annual meeting at the Institute for Life Science Entrepreneurship taking place on Friday, December 16 from 8:00 a.m. - 3:00 p.m. There will be presentations, networking opportunities, lunch and poster sessions. Speakers include a keynote from David Perlin, CSO and SVP, from the Center for Discovery and Innovation and professor of medical sciences at the Hackensack Meridian School of Medicine; a scientific program featuring Jun Wang, Ph.D., and Purnima Bhanot, Ph.D., from Rutgers University, Thomas Dick from CDI-Hackensack Meridian, David Olsen from Merck, and a special presentation from Erin Duffy, Ph.D., CSO of CARBX.

Biotech Showcase: The Investor Conference for Innovators

January 9-11, 2023

The Biotech Showcase, now in its 15th year, will be delivered in-person with a virtual component and this year's event provides valuable opportunities to engage with key players from the life science ecosystem. You will discover groundbreaking therapies in innovative biotech presentations -- delivered during Biotech Showcase, have access to partneringONE starting 6 weeks prior to the conference and 3 months after the conference and submission of up to 150 meeting requests per company. BioNJ members will receive a $200 discount off their registration fees. Contact RBromberg@BioNJ.org for the discount code. 

Partner Spotlight

Email DSacco@BioNJ.org to connect you to this partner. 

For more information reach out to Save@BIO.org.

Please contact BioNJ with any questions.

BioNJ@BioNJ.org  | 609-890-3185 | www.BioNJ.org

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