Decentralized Clinical Trials and the Future of Drug Development

The U.S. Food and Drug Administration (FDA) recently released its first-ever guidance on the design and implementation of decentralized clinical trials (DCTs). These trials are noteworthy because they allow some or all clinical trial-related activities to occur at locations other than a traditional hospital or clinic. These in effect may help increase the number of patients participating in clinical trials who would not otherwise be able to take part. With thoughtful implementation, DCTs can have positive and lasting implications on how medicines are developed to the benefit of patients by: 1) reducing the burden to patients, caregivers and medical providers involved in clinical trials; 2) expanding access to populations that have traditionally been underrepresented in studies; and 3) leading to new ways of studying diseases and fostering innovation.

Gene Therapy Eyedrops Restored a Boy’s Sight. Similar Treatments Could Help Millions

Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye. Antonio, who’s been legally blind for much of his 14 years, can see again. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio’s eyes? This insight not only helped Antonio, it also opened the door to similar therapies that could potentially treat millions of people with other eye diseases, including common ones.

Drug Pricing and the ‘Impossible Trinity’ for Patients

You don’t have to be an economist to understand the law of demand: The higher the price, the less likely a customer is to buy a product. This, of course, limits how much companies can charge. The law of demand applies to pharmaceuticals, but with one big difference. It’s not always the customer who decides that the price is too high. Often, it’s the insurer. Insurers who regard a drug price as too high have only two ways to respond. First, they can force patients who want the medicine to pay high out-of-pocket costs in the form of high copayments. Because that reduces demand, drug companies are willing to lower prices in response. Second, insurers can limit access to new drugs. They can exclude the drugs from their formularies and refuse to cover them at all, or they can limit access via prior authorization, step therapy, and similar policies.

FDA Approves LONSURF® (TRIFLURIDINE/TIPIRACIL) in Combination With BEVACIZUMAB for Adult Patients With Metastatic Colorectal Cancer (MCRC)

Princeton-based BioNJ Member Taiho Oncology, Inc. and Taiho Pharmaceutical Co., Ltd. announced that the U.S. Food and Drug Administration (FDA) has approved LONSURF® (trifluridine/tipiracil) as a single agent or in combination with bevacizumab for the treatment of adult patients with metastatic colorectal cancer (mCRC) previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy. The results from the Phase 3 SUNLIGHT trial, which were published in the New England Journal of Medicine in May 2023, demonstrated that the combination of LONSURF plus bevacizumab provided statistically significant and clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) for patients with mCRC following disease progression or intolerance on two prior chemotherapy regimens compared to LONSURF alone.

Novo Nordisk A/S: Semaglutide 2.4 mg Reduces the Risk of Major Adverse Cardiovascular Events by 20% in Adults With Overweight or Obesity in the SELECT Trial

Plainsboro-based BioNJ Member Novo Nordisk announced the headline results from the SELECT cardiovascular outcomes trial. The double-blinded trial compared subcutaneous once-weekly semaglutide 2.4 mg with placebo as an adjunct to standard of care for prevention of major adverse cardiovascular events (MACEs) over a period of up to five years. The trial enrolled 17,604 adults aged 45 years or older with overweight or obesity and established cardiovascular disease (CVD) with no prior history of diabetes.The trial achieved its primary objective by demonstrating a statistically significant and superior reduction in MACE of 20% for people treated with semaglutide 2.4 mg compared to placebo. The primary endpoint of the study was defined as the composite outcome of the first occurrence of MACE defined as cardiovascular death, non-fatal myocardial infarction or non-fatal stroke. 

Novo Nordisk to Acquire Inversago Pharma and its Obesity Drug Development for $1.07B

Plainsboro-based BioNJ Member Novo Nordisk announced it will acquire Inversago Pharma in a $1.07 billion deal including the development of an obesity drug. Inversago is a privately owned company that develops therapies pitched at people with obesity, diabetes and metabolic disorders. The acquisition of Inversago includes the company’s lead development asset, INV-202, a cannabinoid receptor blocker, which has shown weight loss in a phase 1b trial and is now in phase 2 development. “The acquisition of Inversago Pharma will further strengthen our clinical development pipeline in obesity and related disorders,” Martin Holst Lange, Executive Vice President for Development at Novo Nordisk, said. “This promising class of medicine pioneered by the Inversago team could lead to life-changing new treatment options for those living with a serious chronic disease and, in particular, may offer alternative or complementary solutions for people living with obesity.”

U.S. FDA Approves TALVEY™ (talquetamab-tgvs), a First-in-Class Bispecific Therapy for the Treatment of Patients With Heavily Pretreated Multiple Myeloma

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of TALVEY™ (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. This indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication is contingent upon verification and description of clinical benefit in confirmatory trial(s).

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

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking to expand the indication of EDURANT® (rilpivirine) to include the treatment of HIV-1 infection in children weighing 10 kg or more. A parallel Marketing Authorization application has also been submitted to the European Medicines Agency (EMA) in support of a type II variation and line extension for expanded pediatric use in Europe. These applications reflect Janssen’s longstanding commitment to address the needs of adults, adolescents and children affected by the global HIV epidemic. 

HyBryte™ Expanded Treatment Trial in Cutaneous T-Cell Lymphoma Opens Enrollment

Princeton-based BioNJ Member Soligenix, Inc. announced that patient enrollment has been opened for the investigator-initiated study (IIS) at the University of Pennsylvania, supported by a $2.6 million Orphan Products Development grant award by the U.S. Food and Drug Administration (FDA). The IIS will evaluate the expanded treatment, including up to 12 months of treatment, with HyBryte™ (synthetic hypericin) in patients with early-stage cutaneous T-cell lymphoma (CTCL). The trial is sponsored by Ellen Kim, M.D., Director, Penn Cutaneous Lymphoma Program, Vice Chair of Clinical Operations, Dermatology Department, and Professor of Dermatology at the Hospital of the University of Pennsylvania who was a leading enroller in the recently published positive Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study in the treatment of early-stage CTCL.

Bristol Myers Squibb Inducted into the Billion Dollar Roundtable, Expands Supplier Diversity Efforts

Princeton-based BioNJ Member Bristol Myers Squibb induction into the Billion Dollar Roundtable, joining other Fortune 100 companies that have invested $1 billion with diverse-owned suppliers. This milestone highlights BMS' longstanding history of taking purposeful action to advance health equity, foster a diverse and inclusive workplace, improve and increase the diversity in its clinical trials and expand the diversity among its supplier base. BMS established aspirational inclusion and diversity goals and health equity commitments in 2020 to address health disparities by 2025. In connection to these goals, BMS aspired to spend $1 billion with diverse and minority-owned businesses and has delivered on this important milestone ahead of its 2025 timeline. Sustaining and building on this achievement will remain a priority.

Sanofi to Acquire Qunol®, a Fast-Growing U.S. Brand in the Healthy Aging Segment

Bridgewater-based BioNJ Member Sanofi announces it has entered into a definitive agreement to acquire ownership of Qunol®, a U.S.-based, market-leading brand in health & wellness. This transaction will strengthen Sanofi’s Consumer Healthcare’s (CHC) Vitamin, Mineral and Supplements (VMS) category, one of the largest and fastest-growing consumer health categories in the U.S., focusing on the active ‘healthy aging’ segment. With Qunol’s CoQ10 in heart health and Turmeric in joint health, CHC at Sanofi adds a trusted, profitable double-digit growth brand to its U.S. portfolio, focused on chronic conditions with growing consumer demand. With this acquisition, Sanofi continues to pursue growth opportunities and value creation for its consumer healthcare business.

Kyowa Kirin International and Grünenthal Announce Successful Completion of Deal to Enter Joint Venture Collaboration

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, and Grünenthal GmbH announced the successful completion of a deal to enter into a Joint Venture Collaboration for Kyowa Kirin International's established medicines portfolio. The portfolio comprises 13 brands across six therapeutic areas primarily focused on pain management, including Abstral® and PecFent® for breakthrough cancer pain, Moventig® for opioid-induced constipation, and Adcal-D3® for osteoporosis. All products will continue to be marketed through affiliates in seven major European countries and through a network of partners in various additional territories worldwide. "Grünenthal is a global leader in pain management. With the addition of this strong portfolio, we will be able to help more patients who suffer from various forms of pain," said Gabriel Baertschi, Chief Executive Officer, Grünenthal.

U.S. FDA Approves Merck’s ERVEBO® (Ebola Zaire Vaccine, Live) for Use in Children 12 Months of Age and Older

Rahway-based BioNJ Member Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for ERVEBO, which is now indicated for the prevention of disease caused by Zaire ebolavirus in individuals 12 months of age and older. The vaccine was previously approved for use in individuals 18 years of age and older. ERVEBO does not protect against other species of Ebolavirus or Marburgvirus and the duration of protection conferred by ERVEBO is unknown. The effectiveness of the vaccine when administered concurrently with antiviral medication, immune globulin (IG), and/or blood or plasma transfusions is unknown. ERVEBO includes a contraindication for individuals with a history of a severe allergic reaction (e.g., anaphylaxis) to any component of the vaccine, including rice protein.

Lilly's Retevmo® (selpercatinib) is the First Targeted Therapy to Demonstrate Superior Progression-Free Survival Compared to a PD-1 Inhibitor Plus Chemotherapy for Adults With Newly-Diagnosed Advanced or Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced topline results from the LIBRETTO-431 study evaluating Retevmo versus platinum-based chemotherapy plus pemetrexed – with or without pembrolizumab – as an initial treatment for patients with rearranged during transfection (RET) fusion-positive advanced or metastatic non-small cell lung cancer (NSCLC). The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS). This result was based on a pre-specified interim efficacy analysis conducted by an independent data monitoring committee (IDMC). Adverse events observed on Retevmo were generally consistent with those identified across the previously reported Retevmo development program (LIBRETTO-001, LIBRETTO-121, LIBRETTO-321).

Lilly Completes Acquisition of DICE Therapeutics

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced the successful completion of its acquisition of DICE Therapeutics, Inc. The acquisition expands Lilly's immunology portfolio to include DICE's novel oral therapeutic candidates, including oral IL-17 inhibitors currently in clinical development, to treat chronic diseases in immunology. "Since our founding nearly 150 years ago, we've strived to make life better for people around the world – but we know that to achieve this goal, we have to bring the brightest minds to Lilly," said Ajay Nirula, Ph.D., Senior Vice President of Immunology at Lilly. "With the passion and expertise of our new colleagues from DICE, we look forward to continuing our pursuit of discovering and delivering life-changing medicines for patients living around the world with chronic immunologic diseases."

Two-Year Pulsar Trial Results for Aflibercept 8 mg Demonstrate Durable Vision Gains at Extended Dosing Intervals in Wet Age-Related Macular Degeneration

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced positive, two-year (96 weeks), topline data from the pivotal PULSAR trial investigating aflibercept 8 mg in patients with wet age-related macular degeneration (wAMD). During the trial, aflibercept 8 mg patients were initially randomized to either 12- or 16-week dosing intervals (after three initial monthly doses) and were able to shorten or extend dosing intervals if pre-specified criteria were met. The longer-term data follow the positive two-year results for PHOTON with diabetic macular edema (DME), with PULSAR similarly demonstrating that the vast majority of aflibercept 8 mg patients with wAMD were able to maintain or further extend their dosing intervals. 

Aflibercept 8 mg Two-Year Results from Pivotal Photon Trial in Diabetic Macular Edema Presented at ASRS

BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced the first presentation of positive two-year (96 weeks) results from the pivotal PHOTON trial investigating aflibercept 8 mg with 12- and 16-week dosing regimens, compared to EYLEA® (aflibercept) Injection, in patients with diabetic macular edema (DME). PHOTON (N=658) is a double-masked, active-controlled pivotal trial evaluating non-inferiority of aflibercept 8 mg extended dosing intervals compared to EYLEA. Patients receiving aflibercept 8 mg were initially randomized to either 12- (n=328) or 16-week (n=163) dosing intervals (after three initial monthly doses) compared to an 8-week dosing regimen for EYLEA (n=167) after five initial monthly doses. The PHOTON trial met its primary endpoint last year with aflibercept 8 mg patients achieving clinically equivalent vision gains to EYLEA, with approximately 90% maintaining 12- and 16-week dosing regimens through the first year. 

Jemperli (dostarlimab) Plus Chemotherapy Approved in the U.S. as the First New Frontline Treatment Option in Decades for dMMR/MSI-H Primary Advanced or Recurrent Endometrial Cancer

Warren-based BioNJ Member GSK announced that the U.S. Food and Drug Administration (FDA) has approved Jemperli (dostarlimab) in combination with carboplatin and paclitaxel, followed by Jemperli as a single agent for the treatment of adult patients with primary advanced or recurrent endometrial cancer that is mismatch repair deficient (dMMR), as determined by an FDA-approved test, or microsatellite instability-high (MSI-H). The supplemental Biologics License Application (sBLA) supporting this new indication received Priority Review and was approved ahead of the Prescription Drug User Fee Act action date. With this approval, Jemperli is now indicated earlier in treatment in combination with chemotherapy for patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer. 

New Research Published in Science Highlights Potential of a Naturally Occurring Bacterium to Help Eradicate Malaria

Research published in Science shows that a naturally occurring bacterium discovered by Warren-based BioNJ Member GSK scientists – Delftia tsuruhatensis Tres Cantos 1 (TC1) – could be the basis for new anti-malarial interventions. Studies done in collaboration at the Johns Hopkins Malaria Research Institute at Johns Hopkins Bloomberg School of Public Health show that the bacterium can significantly reduce the malaria parasite load in both the mosquito midgut and salivary glands indicating the potential of TC1 to inhibit transmission of the parasite to humans via the mosquito. The potentially ground-breaking research, which also includes data from preliminary semi-field studies conducted with Institut de Recherche en Sciences de la Santé (IRSS) in a contained ‘MosquitoSphere’ facility in Burkina Faso suggest that laboratory findings could be successfully translated to the field for malaria control. 

European Commission Approves Gilead’s Trodelvy® for Pre-Treated HR+/HER2- Metastatic Breast Cancer

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced that the European Commission (EC) approved Trodelvy® (sacituzumab govitecan) as a monotherapy for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer who have received endocrine-based therapy, and at least two additional systemic therapies in the advanced setting. The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products and is based on the Phase 3 TROPiCS-02 study, in which Trodelvy demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit of 3.2 months versus comparator single-agent chemotherapy (treatment of physician’s choice; TPC) (median OS: 14.4 months vs. 11.2 months; hazard ratio [HR]=0.79; 95% CI: 0.65-0.96; p=0.02). 

Palatin Initiates Clinical Program for Bremelanotide Co-Formulated With a PDE5I for the Treatment of ED in Patients Non-Responsive to PDED51 Treatment

Cranbury-based BioNJ Member Palatin Technologies, Inc. announced it has initiated a clinical development program evaluating bremelanotide, a melanocortin receptor 4 (MCR4) agonist, co-formulated with a PDE5 inhibitor (PDE5i), for the treatment of erectile dysfunction (ED) in patients that do not respond to PDE5i monotherapy. Published clinical data of the use of bremelanotide and PDE5i drugs in ED patients, provides support that the combination of bremelanotide with a PDE5i, two active ingredients that act by different and distinct mechanisms of action, into a single drug product, has the potential to be synergistic, at lower doses, thereby reducing the potential for side effects. Working with a collaborator, Palatin has developed a co-formulation of bremelanotide and a PDE5i which can be administered as a single injection.

Palatin's Vyleesi Licensee Fosun Pharma Reports First Commercial Sale in China and South Korean Licensee Kwangdong Pharmaceuticals Completes Enrollment in Phase 3 Clinical Study

Cranbury-based BioNJ Member Palatin Technologies, Inc. announced that Fosun Pharma, the licensee of Vyleesi in China, reported its first sale in the Hainan Province of China. Palatin also announced that their South Korean licensee, Kwangdong Pharmaceuticals, completed enrollment in its Phase 3 clinical trial evaluating the efficacy and safety of Vyleesi in premenopausal women with HSDD. Data from this trial is anticipated by year end with a potential regulatory submission in the first half of 2024. Vyleesi is the first and only as-needed treatment approved by the U.S. Food and Drug Administration (FDA) for premenopausal women with acquired, generalized hypoactive sexual desire disorder (HSDD).

Palatin Announces Direct-to-Consumer Telehealth Partnership With UpScriptHealth™ for Vyleesi®

Cranbury-based BioNJ Member Palatin Technologies, Inc. announced that it has entered a strategic partnership with UpScriptHealth, a leading direct-to-consumer telemedicine company providing telemedicine services to pharmaceutical and medical technology companies. Under terms of the agreement, UpScriptHealth will make Vyleesi available to the hundreds of thousands of patients in its proprietary Women's Telehealth Platform. Vyleesi is the first and only as-needed treatment approved by the U.S. Food and Drug Administration (FDA) for premenopausal women with acquired, generalized hypoactive sexual desire disorder (HSDD). "We are excited to partner with UpScriptHealth, a pioneer in telehealth," stated Carl Spana, Ph.D., President and CEO of Palatin. "This partnership fortifies our existing telehealth programs and provides a platform to create greater awareness of HSDD and access to Vyleesi through UpScriptHealth's patient base of hundreds of thousands of women."

Huidagene Therapeutics Receives FDA Rare Pediatric Disease Designation for HG004 to Treat Inherited Blindness

Clinton-based HuidaGene Therapeutics announced the U.S. Food and Drug Administration (FDA) had granted Rare Pediatric Disease Designation (RPDD) to HG004 for the treatment of inherited retinal disease caused by RPE65 mutations (RPE65-IRDs). The FDA grants RPDD to incentive development of new treatments for serious or life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people affected in the U.S. The RPDD program allows for a sponsor who receives an approval to qualify for a priority review voucher (PRV) that can be deemed to receive an expedited six-month priority review for any subsequent marketing application or may be sold or transferred. 

DAICHIRONA® for Intramuscular Injection (Monovalent: Original Strain), mRNA COVID-19 Vaccine, Approved for Manufacturing and Marketing as Booster Vaccination in Japan

Daiichi Sankyo, with a site in Basking Ridge, announced that it has received approval for the manufacturing and marketing of DAICHIRONA® for Intramuscular Injection (DS-5670), an original monovalent mRNA vaccine against the novel coronavirus infectious disease (hereafter, COVID-19), in Japan for the prevention of infectious disease caused by SARS-CoV-2 (booster vaccination). The application for approval of DAICHIRONA® for its manufacturing and marketing in Japan was submitted in January 2023, and it is now approved as the first Japan-made mRNA vaccine against COVID-19. DAICHIRONA® can be distributed and stored under refrigeration (2°C–8°C), offering improved convenience to healthcare professionals. In the current special temporary vaccination program against COVID-19, Omicron-adapted bivalent mRNA vaccines are used for the booster series campaign that began in May 2023.

Amarin and Neopharm Announce Exclusive Commercialization Agreement for VAZKEPA® (icosapent ethyl) in Israel

Bridgewater-based Amarin Corporation plc and Neopharm Israel announced that the two companies have entered into an exclusive marketing and commercialization agreement for VAZKEPA® (icosapent ethyl) in the State of Israel, Gaza, West Bank and the territories of the Palestinian Authority. VAZKEPA capsules are the first prescription treatment comprised solely of the active ingredient, icosapent ethyl, a highly purified form of eicosapentaenoic acid. The agreement follows Israel’s Ministry of Health (MOH) Pharmaceuticals Division granting regulatory approval to VAZKEPA (icosapent ethyl). The MOH issued its approval of VAZKEPA to reduce the risk of cardiovascular events in adult statin-treated patients at high cardiovascular risk with elevated triglycerides (≥150 mg/dL [≥ 1.7 mmol/L]) and either established cardiovascular disease or diabetes and at least one other cardiovascular risk factor.

Amarin and Lotus Pharmaceuticals Announce Exclusive Partnership Agreement to Commercialize Vazkepa (Icosapent Ethyl) in Southeast Asia and South Korea

Bridgewater-based Amarin Corporation plc and Lotus Pharmaceuticals announced that the two companies have entered into a long-term exclusive partnership to distribute and commercialize VAZKEPA® (icosapent ethyl) across 10 countries, including nine in Southeast Asia (the Association of Southeast Asian Nations – ASEAN) and South Korea. VAZKEPA capsules have been approved and commercialized in a number of global markets, including the U.S. and European markets, as the first prescription treatment comprised solely of the active ingredient, icosapent ethyl, a highly purified form of eicosapentaenoic acid (EPA). Patrick Holt, President & CEO, Amarin, said, “Lotus’ strong commercialization capabilities and footprint across ASEAN markets and South Korea make them the ideal partner to help us expand patient access to VAZKEPA – an innovative treatment option to reduce cardiovascular risk – across the region and fuel our international growth.”

Onkos Surgical Expands Pediatric Musculoskeletal Oncology Capability, Unveils New Manufacturing and Innovation Center

Onkos Surgical announced the launch of the JTS Extendible Prosthesis, a novel growing implant designed for pediatric patients with bone cancer. At the same time, the firm also unveiled its new, state-of-the-art manufacturing and research & development innovation center at its Parsippany headquarters to support the integration and growth of the technology. Patrick Treacy, Onkos’ founder and CEO, said, “Pediatric sarcoma patients, who are candidates for the JTS growing prosthesis, often have complex treatment cycles in which we need to deliver precision designs in a very specific treatment window. Our investment in manufacturing innovation and design expertise will make a big difference in the future for these surgeons, patients and families, and we couldn’t be more proud to serve their needs.”

Hikma and Rakuten Medical Sign Exclusive Licensing Agreement for Alluminox™ Platform Cancer Treatment for the Middle East and North Africa

Berkeley Heights-based Hikma Pharmaceuticals PLC announces an exclusive licensing and commercialisation agreement with Rakuten Medical, Inc. (Rakuten Medical), a global biotechnology company developing and commercialising precision, cell targeting therapies based on its proprietary Alluminox™ platform. Under the terms of the agreement, Hikma has an exclusive license to commercialize products in Rakuten Medical’s pipeline using its photoimmunotherapy technology platform, Alluminox™, in all its MENA markets. The Alluminox™ platform is an investigational technology platform based on a cancer therapy called photoimmunotherapy, which was developed by Dr. Hisataka Kobayashi and team from the National Cancer Institute in the United States. 

Photocure Partner Asieris Confirms Hexvix Phase III Clinical Trial in China Meets Endpoint

Princeton based Photocure announced  that its partner Asieris Pharmaceuticals has communicated that the Hexvix^® Phase III bridging trial in China has successfully met its primary research endpoint. The objective of this multicenter Phase III trial is to support regulatory approval of Hexvix in Mainland China. The bridging trial is intended to confirm results from prior clinical trials regarding the safety and detection benefits of blue light cystoscopy (BLC®) with Hexvix compared to white light cystoscopy (WLC) in the diagnosis of non-muscle invasive bladder cancer (NMIBC) in a Chinese population. The clinical trial follows the same protocols for registration studies in countries where Hexvix already has market authorization. 

ENHERTU® Demonstrated Clinically Meaningful Progression-Free Survival and Overall Survival Across Multiple HER2 Expressing Advanced Solid Tumors in DESTINY-PanTumor02 Phase 2 Trial

Topline results from the primary analysis of the ongoing DESTINY-PanTumor02 Phase 2 trial showed ENHERTU® (trastuzumab deruxtecan) demonstrated clinically meaningful progression-free survival (PFS) and overall survival (OS) in previously treated patients across multiple HER2 expressing advanced solid tumors, two secondary endpoints of the trial. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Basking Ridge-based Daiichi Sankyo and AstraZeneca. In the primary analysis, ENHERTU also continued to show durable responses based on investigator-assessed confirmed objective response rate (ORR), the primary endpoint of the trial, and duration of response (DoR), a secondary endpoint, reinforcing the results from an interim analysis of the trial.

PharmaKinnex, Data360 Team Up to Extend Client Reach and Improve Patient Access

East Brunswick-based PharmaKinnex and Data360, a data and engagement solutions provider for health care marketers, recently announced they partnered to further enhance client reach and improve patient outcomes. Integration of Data360’s deep insights with PK’s marketing expertise will improve PK’s call plans, allowing PK to message to new audiences and reach prescribers ahead of the competition. The partnership positions PK as the only inside sales organization with access to such a high level of data, offering a unique and powerful industry advantage. From rare disease to oncology and beyond, this unique partnership is designed to deliver increased profits to clients, enable more advanced and secure data analysis and, most importantly, connect patients with life-saving treatments. 

Rutgers Cancer Institute of New Jersey Receives Grants to Support Ongoing Oncology Research

Grants totaling $1.5 million have been awarded by the New Jersey Commission on Cancer Research (NJCCR) to several investigators at Rutgers Cancer Institute of New Jersey, the State’s only National Cancer Institute-designated Comprehensive Cancer Center together with RWJBarnabas Health. NJCCR is the State’s only Commission that promotes significant and original research in New Jersey into the causes, prevention, treatment and palliation of cancer and serves as a resource to providers and consumers of cancer services. “As New Jersey’s only National Cancer Institute-designated Comprehensive Cancer Center, Rutgers Cancer Institute is committed to mentoring the next generation of investigators so that we may learn more about the underlying mechanisms of cancer,” said Rutgers Cancer Institute Deputy Director and Chief Scientific Officer Eileen White, Ph.D.

New Chief of Pediatric Bone Marrow Transplantation and Cellular Therapies Named to New Jersey’s Only NCI-designated Comprehensive Cancer Center

Rutgers Cancer Institute of New Jersey and RWJBarnabas Health have appointed Niketa C. Shah, M.D., as Chief of Pediatric Bone Marrow Transplantation and Cellular Therapies at New Jersey’s leading cancer program and the only one in the state designated as a Comprehensive Cancer Center by the National Cancer Institute. The selection comes following an extensive national search. Dr. Shah is also an associate professor of pediatrics at Rutgers Robert Wood Johnson Medical School. Dr. Shah will enhance and oversee the Pediatric Stem Cell Transplantation and Cellular Therapies Program at Rutgers Cancer Institute, which in collaboration with The Bristol Myers Squibb Children’s Hospital at Robert Wood Johnson University Hospital, offers children, adolescents and young adults both allogeneic and autologous transplants for cancers including leukemia, lymphoma and solid tumor cancers. 

Hackensack University Medical Center Named Top Hospital in State by U.S. News

Hackensack University Medical Center is once again the top-rated hospital in New Jersey, according to the prestigious U.S. News & World Report Best Hospitals ranking for 2023. It marks the eighth time HUMC has been first on the list in the past 12 years. This year, however, it can be argued that HUMC is better than ever. HUMC not only improved from No. 10 to No. 6 overall in the New York City metro area rankings, but it had six specialty areas ranked in the Top 50 nationally — or two more than a year ago. HUMC also had three specialty areas ranked as “high performing.” Jersey Shore University Medical Center was ranked No. 6 overall in the State and among the Top 25 in the metro area. 

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Insmed 2022 Responsibility Report

At Insmed, our core values of collaboration, accountability, passion, respect and integrity unite our people and drive our purpose to transform the lives of patients with serious and rare diseases. Patients are our North Star, and our business is built around serving their needs. To us, this means that being responsible isn’t something we do, it’s simply something we are and always have been. Insmed’s inaugural Responsibility Report reflects the culture and purpose we live by every day. The report describes the significant strides we have made in deepening our responsibility, as well as the commitments we have undertaken to continue these pursuits as we grow.

Winds of Change: Dealmaking Trends in the Evolving Innovation Economy

Hosted by LES USA & Canada

October 15-18, 2023

Under the theme of Winds of Change: Dealmaking Trends in the Evolving Innovation Economy, our dynamic international IP community will meet in Chicago for two action-packed days of education, outstanding programming and networking, with global deal making opportunities.