BioNJ's 13th Annual BioPartnering Conference

What You Should Know About Real-World Evidence and Drug Development

Recently, industry leaders, regulators, academics, data partners and patient advocates gathered in Washington, D.C., to discuss using real-world data and real-world evidence for regulatory decision-making. The discussion explored emerging analytical methodologies to address the challenge of mitigating sources of bias and confounding variables that come up in nearly all sources of real-word data.

Branded Drug Report 2023: Lisa Joldersma, PhRMA

In recent decades, pharmacy benefit managers have grown into shadowy and hugely influential players in the prescription drug supply chain. For the most part, they have managed to avoid public scrutiny and government regulation. Fortunately, that’s starting to change, as policy makers find it difficult to overlook rampant conflicts of interest at the core of the typical PBM’s business model — conflicts that too often increase drug costs for patients, employers and the broader health care system. Insurers contract with PBMs to help determine which medicines will — and will not — be covered by a health plan. The PBM often establishes a list of covered drugs (known as a formulary) for the health plan, which governs what patients are charged at the pharmacy and may create hurdles, like prior authorization, for patients to overcome before receiving a prescribed medicine.

Study Finds Hepatitis C Cure Saved Medicaid $15 Billion

New research published in the American Journal of Managed Care (AJMC) underscores the critical role that medicines play in improving health and reducing disease complications and other, often costly, medical care and services. The study finds that since curative direct-acting antiviral medications for hepatitis C were approved in 2013, even after factoring in the cost of treatment, Medicaid has saved an estimated $15 billion in avoided health care costs and nearly 285,000 Medicaid enrollees are estimated to have been cured. The hepatitis C virus, or HCV, is a devastating disease that progresses slowly over time and can cause severe damage to the liver and other complications. 2013 marked a paradigm shift in the treatment of HCV, ushering in a new era of highly effective treatments thanks to innovation from the biopharmaceutical industry. Over the past decade, subsequent treatment regimens have offered continued improvements. Today, more than 95% of HCV infected persons can be cured of the disease.

Private Equity Acquires a Taste for Drug Development

Private equity firms that deemed drug development too risky for their liking in the past are increasingly investing in the sector, raising dedicated funds and coming up with deals that compensate them for the uncertainty involved. These firms are seeking to capitalize on the growing gap between the supply of capital for clinical research and the number of drugs competing for it. These deals are not structured as the leveraged buyouts that private equity firms are mostly known for. Instead, the buyout firms invest in the development of the drugs, typically when they are in so-called Phase 3 clinical trials, one step away from regulatory clearance. They negotiate with pharmaceutical companies the returns they will receive in advance.

Perlin to Receive Dr. Sol J. Barer Award from BioNJ

BioNJ has announced that Dr. David Perlin, Chief Scientific Officer and Executive Vice President for the Hackensack Meridian Center for Discovery and Innovation, will be the recipient of the 2023 Dr. Sol J. Barer Award for Vision, Innovation and Leadership. “We are thrilled to honor Dr. Perlin with the 2023 Dr. Sol J. Barer Award for Vision, Innovation and Leadership for his tireless work on behalf of patients, medical innovation and New Jersey’s ever-growing life sciences ecosystem,” said BioNJ President and CEO Debbie Hart. “Well recognized for his innovative and entrepreneurial leadership in biomedical sciences, Dr. Perlin is a highly accomplished researcher and visionary in the development of novel therapies to detect, combat and prevent deadly infections.”

Interview With Debbie Hart, President and CEO BioNJ

Investment Reports by Newsweek

In what way is New Jersey defending its position as leading hub for innovation and development in life sciences? The industry has grown by 9% from 2018 to 2021 in New Jersey, providing 105,000 direct jobs coming from nearly 4,500 establishments. We have a high degree of industry specialization - BIO just released its latest economic impact study in New Jersey, showing a 79% greater concentration of bioscience industry-to-employment relative to the national average. New Jersey is one of the top states when it comes to patent activity, and venture capital investment has increased consistently in recent years. Meanwhile all that productivity and investment are paying off. In fact, nearly 40% of novel FDA drug approvals in 2020 and 2021 came from companies with a footprint in New Jersey, including the first Covid-19 vaccines and therapies.

FDA Approves Once-Weekly Wegovy® Injection for the Treatment of Obesity in Teens Aged 12 Years and Older

Plainsboro-based BioNJ Member Novo Nordisk announced that the U.S. Food and Drug Administration (FDA) has approved a new indication for Wegovy® (semaglutide) injection 2.4 mg, making it the first-and-only prescription anti-obesity medicine for teens with once-weekly dosing. Wegovy® is now also indicated for use in adolescents (≥ 12 years old) with an initial BMI at or above the 95th percentile for age and sex (based on CDC growth charts) along with a reduced calorie meal plan and increased physical activity to lose weight and keep it off. Obesity is a disease that can start in childhood or adolescence, and up to 90 percent of teenagers with obesity may continue to live with obesity into adulthood. Teens with obesity are at a greater risk of developing serious weight-related health problems.

Otsuka and Lundbeck Announce FDA Acceptance and Priority Review of sNDA for Brexpiprazole for the Treatment of Agitation Associated With Alzheimer’s Dementia

BioNJ Member Otsuka Pharmaceutical Co., Ltd., with a site in Princeton, and H. Lundbeck A/S announce the U.S. Food and Drug Administration (FDA) has determined that the supplemental New Drug Application (sNDA) for brexpiprazole for use in the treatment of agitation associated with Alzheimer’s dementia (AAD) is sufficiently complete to permit a substantive review. The FDA has assigned the application priority review and a Prescription Drug User Fee Act (PDUFA) target action date of May 10, 2023. The FDA also indicated that they are currently planning to hold a Psychopharmacologic Drug Advisory Committee meeting to discuss the application. The sNDA submission is based on data from two positive clinical phase III studies that investigated the treatment of brexpiprazole in patients with AAD.

Hepion Pharmaceuticals Receives $3.2 Million in Aggregate Non-Dilutive Funding

Edison-based BioNJ Member Hepion Pharmaceuticals, Inc. announced the receipt of $2.9 million in net proceeds from the sale of tax benefits pursuant to the Company’s participation in the New Jersey Economic Development Authority (“NJEDA”) NOL program under the New Jersey Economic Recovery Act of 2020, and receipt of a C$416,415 (US$309,000) Alberta Innovation Employment Grant. NJEDA’s NOL program enables qualified, New Jersey-based technology or biotechnology companies to sell net operating losses to unrelated profitable corporations. This allows qualifying technology and biotechnology companies with NOLs to turn their tax losses and credits into cash proceeds to fund growth and operations, including research and development (“R&D”) or other allowable expenditures.

AbbVie and Immunome Announce Strategic Collaboration to Discover Multiple Novel Oncology Targets

BioNJ Member AbbVie, with a site in Madison, and Immunome, Inc. announced a worldwide collaboration and option agreement directed to the discovery of up to 10 novel antibody-target pairs arising from three specified tumor types using Immunome's Discovery Engine. "Partnering with Immunome represents AbbVie's commitment to developing and commercializing novel treatment approaches for solid tumors," said Steve Davidsen, Ph.D., Vice President, Oncology Discovery Research, AbbVie. "Immunome's approach has the potential to unlock novel cancer biology and yield multiple therapeutic candidates. We look forward to utilizing their Discovery Engine to enhance our existing oncology pipeline." Under the terms of the agreement, Immunome will grant AbbVie the option to purchase worldwide rights for up to 10 novel target-antibody pairs arising from the selected tumors.

Helsinn Publishes Results from a Secondary Analysis of a Phase 3 trial in Patients With CINV in The Oncologist

BioNJ Member Helsinn Grou,p with a site in Iselin, announced results from a secondary analysis of a Phase 3 multicenter pivotal trial in patients with breakthrough chemotherapy-induced nausea and vomiting (CINV) receiving antiemetic prophylaxis (oral or IV NEPA) have been published in The Oncologist. Using data from a previously reported Phase 3 trial, the analysis classified patients into 3 groups based on their response during the first chemotherapy cycle: CR, short-term CINV (1-2 days), and extended CINV (3-5 days). The analysis indicated that patients who experienced CR in their first cycle had a 93% or higher CR rates in subsequent cycles. Comparing patients with short-term CINV and extended CINV in cycle 1, recurrent CINV occurred in 30.6% and 69.5% of subsequent cycles, respectively (p<0.001). With close monitoring during the first chemotherapy cycle, clinicians can use the patient’s 

Oyster Point Pharma and Famy Life Sciences Acquired by Viatris Establishing New Viatris Eye Care Division

Viatris Inc. announced that it has closed its acquisitions of Princeton-based BioNJ Member Oyster Point Pharma and Famy Life Sciences to establish a new Viatris Eye Care Division. The transaction was first announced as part of Viatris’ strategic update. Former Oyster Point Pharma CEO, Jeffrey Nau, Ph.D., MMS, will lead the new division. Viatris CEO Michael Goettler said, “We are starting the year out strong with the closings of our acquisitions of Oyster Point Pharma and Famy Life Sciences, bringing together our collective commercial expertise, scientific capabilities and global infrastructure to create the Viatris Eye Care Division, which has the potential to deliver even more access to patients and showcases the power of our Global Healthcare Gateway.” 

Boehringer Ingelheim and 3T Biosciences Join Forces to Develop Next-Generation Cancer Immunotherapies

BioNJ Member Boehringer Ingelheim, with a site in North Brunswick, and 3T Biosciences, announced they have entered into a strategic collaboration and licensing agreement to discover and develop next-generation cancer therapies to address high unmet patient needs. The partnership will bring together 3T Biosciences’ best-in-class 3T-TRACE (T-Cell Receptor Antigen and Cross-Reactivity Engine) discovery platform with Boehringer Ingelheim’s two-pronged research strategy combining cancer cell-directed and immune cell-targeting compounds, further strengthening the company’s pipeline. “At Boehringer Ingelheim, we are committed to transforming patients’ lives. Through our new partnership with 3T Biosciences, we aim at accelerating and expanding our pipeline of first-in-class T-cell based therapies for patients affected by cancer,” said Lamine Mbow, Ph.D., Global Head of Cancer Immunology + Immune Modulation, Boehringer Ingelheim.  

Sanofi Ventures Announces Multi-Year Capital Commitment from Sanofi, Increasing Evergreen Fund to $750M

Sanofi Ventures has announced an additional multi-year commitment from Bridgewater-based BioNJ Member Sanofi, with an increase in capital to more than $750 million to the evergreen venture fund. In addition to serving as a financial partner to top-tier early-to-mid-stage portfolio companies, the fund supports future efforts for business development and M&A opportunities within Sanofi. The additional capital, confirmed by the executive committee, will also fuel the expansion and investment capacity of the Sanofi Ventures investment team on a global scale. Sanofi Ventures invests in top innovators working in areas including immunology and inflammation, rare diseases, oncology, cell and gene therapy, vaccines, and digital health and data science. The team partners across all stages of the private company lifecycle, from Seed to Series B and beyond, leading financings, serving on boards, and taking pride in working alongside portfolio companies to drive long-term value. 

FDA Accepts Nirsevimab Application as First Protective Option Against RSV Disease for All Infants

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) has accepted the Biologics License Application (BLA) for nirsevimab for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants entering or during their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. Nirsevimab is being developed jointly by Sanofi and AstraZeneca and, if approved, would be the first protective option for the broad infant population, including those born healthy, at term or preterm, or with specific health conditions. The FDA has indicated they will work to expedite their review. The Prescription Drug User Fee Act date, the FDA target action date for their decision, is in the third quarter of 2023. 

Gilead to Acquire All Remaining Rights to Potential First-in-Class Immunotherapy GS-1811 from Jounce Therapeutics

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, and Jounce Therapeutics, Inc. amended their existing license agreement for GS-1811 (formerly JTX-1811), enabling Gilead to buy out remaining contingent payments potentially due under the license agreement executed in August 2020. As part of the transaction, certain operational obligations of the parties related to GS-1811, an anti-CCR8 antibody, set forth in the license agreement have also been terminated. Gilead will acquire certain related intellectual property, including all outstanding rights of Jounce to GS-1811, pursuant to the transaction agreement. GS-1811, a potentially first-in-class immunotherapy, is designed to selectively deplete immunosuppressive tumor-infiltrating T regulatory cells in the tumor microenvironment and is currently in Phase 1 clinical development as a possible treatment for patients with solid tumors. 

Gilead and EVOQ Therapeutics Announce Collaboration to Advance Immunotherapies

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, and EVOQ Therapeutics, Inc. (EVOQ) announced a collaboration and licensing agreement to advance EVOQ’s proprietary technology for the treatment of rheumatoid arthritis (RA) and lupus. EVOQ’s NanoDisc technology is designed to enable lymph-targeted delivery of disease-specific antigens and has the potential to change the paradigm for the treatment of autoimmune diseases. Under the agreement, Gilead and EVOQ will collaborate on preclinical development. Gilead has the option to exclusively license rights to EVOQ’s NanoDisc technology to pursue product candidates for RA and lupus indications and will be responsible for clinical development and commercialization.

European Medicines Agency Validates Marketing Authorization Application for Trodelvy® (sacituzumab govitecan-hziy) for Pre-treated HR+/HER2- Metastatic Breast Cancer

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced that the European Medicines Agency (EMA) has validated a Type II variation Marketing Authorization Application (MAA) for Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. This Marketing Authorization Application is based on data from the registrational Phase 3 TROPiCS-02 study, which met its primary endpoint of progression-free survival (PFS) and key secondary endpoint of overall survival (OS) versus comparator chemotherapies (treatment of physician’s choice (TPC) of chemotherapy). 

BeiGene Announces Acceptance of 12th Regulatory Submission in China for PD-1 Inhibitor Tislelizumab

BioNJ Member BeiGene, with a site under development in Hopewell, announced it has accepted a supplemental biologics license application (sBLA) for tislelizumab in patients with first-line unresectable or metastatic hepatocellular carcinoma (HCC). Hepatocellular carcinoma is the most common type of primary liver cancer worldwide and is associated with a very poor prognosis. New cases and deaths due to HCC in China account for half of the global numbers and the 5-year survival rate for patients with HCC in China is only 14%. The sBLA is supported by data from the RATIONALE 301 clinical trial (NCT03412773) that enrolled 674 patients from research centers across Asia, Europe and the United States. 

U.S. FDA Accepts for Review the Biologics License Application for Pfizer’s Investigational Pentavalent Meningococcal Vaccine Candidate (MenABCWY) in Adolescents

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for its investigational pentavalent meningococcal vaccine candidate (MenABCWY). Pfizer submitted MenABCWY for the prevention of meningococcal disease caused by the most common serogroups in individuals 10 through 25 years of age. Pfizer’s MenABCWY vaccine candidate combines the components of two vaccines into one, helping protect against the meningococcal serogroups that cause the majority of invasive meningococcal disease (IMD) globally. In the U.S., approximately 55 million adolescents and young adults are in the age range for meningococcal vaccination (11-23 years old), according to ACIP recommendations.

Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate

BioNJ Member Pfizer, with a site in Peapack, announced positive top-line results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. “Pfizer has more than 30 years of experience in developing and commercializing therapies for hematological disorders, and a deep understanding of the significant challenges that people living with hemophilia continually face. We are proud to advance the latest innovation for people living with hemophilia B and are encouraged by the potential of this investigational gene therapy,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. 

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Pfizer’s 20-Valent Pneumococcal Conjugate Vaccine in Infants and Children

BioNJ Member Pfizer, with a site in Peapack, announced that the U.S. Food and Drug Administration (FDA) accepted for priority review a supplemental Biologics License Application (sBLA) for its 20-valent pneumococcal conjugate vaccine candidate (20vPnC) for the prevention of invasive pneumococcal disease (IPD) caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes contained in the vaccine in infants and children 6 weeks through 17 years of age, and for the prevention of otitis media caused by seven of the 20 Streptococcus pneumoniae serotypes contained in the vaccine. Priority Review designation by the FDA reduces the standard sBLA review period by four months. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA on the 20vPnC vaccine application is anticipated in April 2023. 

Organon Pursues New Solutions in High-Need Areas of Women’s Health With Investment in Claria Medical

Jersey City-based BioNJ Member Organon announced it has invested in Claria Medical Inc., a privately held company developing an investigational medical device being studied for use during minimally invasive laparoscopic hysterectomy. The agreement also grants Organon the option to acquire Claria Medical. Under the terms of the agreement, Organon will pay $8 million upfront and have the option to acquire Claria Medical for predefined terms. The $8 million upfront payment will be classified as an in-process research & development expense in 2023. Claria’s initial investigational device, the Claria System, uses an intelligent uterine containment and extraction system that aims to improve the hysterectomy procedure for both patients and physicians.

Alvotech and Teva Announce Acceptance of U.S. Biologics License Application for AVT04, a Proposed Biosimilar to Stelara® (ustekinumab)

Alvotech and Parsippany-based BioNJ Member Teva Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for review a Biologics License Application (BLA) for AVT04, Alvotech’s proposed biosimilar to Stelara® (ustekinumab), which is prescribed to treat a variety of inflammatory conditions. The companies anticipate that the FDA’s review will be completed in second half of 2023. “We are pleased to be one step closer to making AVT04 available to patients and providers as a biosimilar treatment option to Stelara® for indicated inflammatory conditions,” said Christine Baeder, SVP, Chief Operating Officer US Generics, Teva. “Teva continues to remain focused on our commitment to lower healthcare costs and expand the availability and access of biosimilars.” 

Libtayo® (cemiplimab) Approved in Japan for Advanced or Recurrent Cervical Cancer

Regeneron Pharmaceuticals, with a site in Basking Ridge, announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing authorization for Libtayo® (cemiplimab) as monotherapy to treat patients with advanced or recurrent cervical cancer whose disease progressed after chemotherapy. The MHLW approval is based on positive data from the international, multicenter Phase 3 EMPOWER-Cervical 1 trial, conducted in collaboration with NRG Oncology-Japan, the GOG Foundation, Inc., and the European Network for Gynecological Oncological Trial (ENGOT) groups. The trial evaluated Libtayo compared to an investigator's choice of chemotherapy and enrolled 608 patients across 14 countries, including Japan, irrespective of PD-L1 expression status or histology. 

FDA Approves LEQEMBI™ (lecanemab-irmb) Under the Accelerated Approval Pathway for the Treatment of Alzheimer’s Disease

Nutley-based Eisai Co., Ltd. and BioNJ Member Biogen Inc. announced that under the Accelerated Approval Pathway the U.S. Food and Drug Administration (FDA) has approved lecanemab-irmb (Brand Name in the U.S.: LEQEMBI™) 100 mg/mL injection for intravenous use, a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (“protofibril”) and insoluble forms of amyloid beta (Aβ) for the treatment of Alzheimer’s disease (AD). The approval is based on Phase 2 data that demonstrated that LEQEMBI reduced the accumulation of Aβ plaque in the brain, a defining feature of AD. Using the recently published data from the large global confirmatory Phase 3 clinical trial, Clarity AD, Eisai will work quickly to file a Supplemental Biologics License Application (sBLA) to the FDA for approval under the traditional pathway.

EISAI Submits Marketing Authorization Application for LECANEMAB as Treatment for Early Alzheimer’s Disease in Europe

Nutley-based Eisai Co., Ltd. and BioNJ Member Biogen Inc. with a site in Princeton announced that Eisai has submitted a marketing authorization application (MAA) for lecanemab (Brand Name in the U.S.: LEQEMBI™), an investigational anti-amyloid beta (Aβ) protofibril antibody, for the treatment of early Alzheimer’s disease (mild cognitive impairment due to Alzheimer’s disease (AD) and mild AD dementia) to the European Medicines Agency (EMA). This application is based on the results of the Phase III Clarity AD study and the Phase IIb clinical study (Study 201), which demonstrated that lecanemab treatment showed a reduction of clinical decline in early AD and is subject to a validation to determine whether the EMA accepts the application for review.

EISAI Initiates BLA Submission of Data for LECANEMAB in China

Nutley-based Eisai Co., Ltd. and BioNJ Member Biogen Inc. announced that Eisai has initiated submission of data for Biologics License Application (BLA) to the National Medical Products Administration (NMPA) of China for lecanemab an investigational anti-amyloid beta (Aβ) protofibril antibody. The registration category of lecanemab was designated as a Category 1 drug (innovative biologics not approved in China or any other countries). The data submitted in this package includes data from the Phase II clinical trial (Study 201) in mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD) with confirmed Aβ accumulation in the brain and the top-line data of the large global Phase III Clarity AD study. Eisai will submit additional data including full data of the Clarity AD study, as directed by the NMPA.

Advaxis Updates on the Phase 1 Clinical Trial of ADXS-504 for the Treatment of Early Prostate Cancer

Monmouth Junction-based BioNJ Member Advaxis, Inc. announced an update on the Phase 1 clinical study evaluating ADXS-504, the company’s off-the-shelf neoantigen drug candidate, in patients with biochemically recurrent (early) prostate cancer that is being conducted at Columbia University Irving Medical Center. The study design of this Phase 1 open-label dose escalation study for patients in both dose cohorts. In this study, ADXS-504 is being administered via infusion every four weeks for a total of six doses, followed by four additional maintenance doses every twelve weeks, in patients with biochemically recurrent prostate cancer, i.e., those with elevation of prostate-specific antigen (PSA) in the blood after radical prostatectomy or radical radiotherapy (external beam or brachytherapy) and who are not currently receiving androgen ablation therapy. 

TG Therapeutics Announces FDA Approval of BRIUMVI™ (ublituximab-xiiy)

TG Therapeutics, Inc., with a site in Edison, announced the U.S. Food and Drug Administration (FDA) has approved BRIUMVI™ (ublituximab-xiiy), for the treatment of relapsing forms of multiple sclerosis (RMS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. Approval was granted for this indication based on data from the ULTIMATE I & II Phase 3 trials, which demonstrated superiority over teriflunomide in significantly reducing the annualized relapse rate (ARR, the primary endpoint), the number of T1 Gd-enhancing lesions and the number of new or enlarging T2 lesions. Results from the ULTIMATE I & II trials were recently published in August 2022 in The New England Journal of Medicine. 

American CryoStem Announces FDA Approval of IND to Commence Phase I Clinical Trial for Long COVID/Post-Acute Sequelae of SARS-CoV-2 (PASC)

Eatontown-based American CryoStem Corporation announced that it has received U.S. Food and Drug Administration (FDA) approval to proceed with a Phase I Clinical Trial for the treatment of Long COVID/PASC. The IND titled "A Pilot Study on Research Treatment of Long COVID/Post-Acute Sequelae of SARS-CoV-2 (PASC) using ATCell™", will assess CryoStem's proprietary ATCell™ therapy for safety and tolerability with the goal to rapidly develop a safe and effective therapeutic for the expansive Long COVID/PASC population afflicted with this life-altering disease. The Study is designed to enroll active-duty military members or their families who are suffering with a confirmed diagnosis of Long COVID. They will be treated with ATCELL™, the company's autologous mesenchymal cell therapy delivered through intravenous infusion.  

WuXi Biologics and GSK Enter into License Agreement on Multiple Novel Bi- & Multi-specific T Cell Engagers

WuXi Biologics, with a site in Cranbury, announced a license agreement with BioNJ Member GSK plc under which GSK will have exclusive licenses for up to four bi- & multi-specific TCE antibodies developed using WuXi Biologics’ proprietary technology platforms. Under the terms of the agreement, GSK will be granted an exclusive global license for the research, development, manufacturing and commercialization of a pre-clinical bispecific antibody that crosslinks tumor cells and T cells by targeting a tumor-associated antigen (TAA) on tumor cells and CD3 expression on T cells and up to three additional pre-clinical TCE antibodies currently at an earlier discovery stage. WuXi Biologics will receive a $40 million upfront payment and up to $1.46 billion in additional payments for research, development, regulatory and commercial milestones across the four TCE antibodies. 

Japan Ministry of Health, Labor and Welfare Approves Manufacturing and Marketing of Adtralza® (tralokinumab) in Japan for Adults With Atopic Dermatitis

LEO Pharma, with a site in Madison, announced that the Japan Ministry of Health, Labor and Welfare (MHLW) has granted approval for the manufacturing and marketing of Adtralza® (tralokinumab) subcutaneous injection (S.C.) for adults with atopic dermatitis, which has inadequately responded to conventional therapies. Adtralza is the first and only approved human, monoclonal antibody developed to specifically target and neutralize the IL-13 cytokine, which plays a key role in the immune and inflammatory process which are the underlying causes of atopic dermatitis signs and symptoms. The MHLW has approved Adtralza to be administered as a subcutaneous injection in adults with an initial dose of 600mg, followed by a 300mg dose given every other week.

EZHARMIA® Launched in Japan as First Dual EZH1 and EZH2 Inhibitor Therapy for Patients With Adult T-Cell Leukemia-Lymphoma

Basking Ridge-based Daiichi Sankyo announced the launch of EZHARMIA® (valemetostat tosilate), a first-in-class dual inhibitor of EZH1 and EZH2, in Japan for the treatment of patients with relapsed or refractory adult T-cell leukemia-lymphoma (ATLL). Marketing approval of EZHARMIA was granted by Japan’s Ministry of Health, Labor and Welfare (MHLW) in September 2022 based on results of an open-label, single-arm pivotal phase 2 study in 25 patients with three aggressive subtypes of relapsed or refractory ATLL in Japan. Data from the trial demonstrated an objective response rate (ORR) of 48% (95% CI: 27.8%-68.7%). EZHARMIA was generally well-tolerated in the Phase 2 study. 

ENHERTU ® Type II Variation Application Validated by EMA for the Treatment of HER2 Mutant Metastatic Non-Small Cell Lung Cancer

Basking Ridge-based Daiichi Sankyo announced that the European Medicines Agency (EMA) has validated the Type II Variation application for ENHERTU® (trastuzumab deruxtecan) as a monotherapy for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations and who have received a prior systemic therapy. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use. This application is based on data from the DESTINY-Lung02 Phase 2 trial and the DESTINY-Lung01 Phase 2 trial published in The New England Journal of Medicine.

New Study Publication Reinforces the Clinical Benefits of Blue Light Cystoscopy in the ASC Setting; Supports Ongoing Initiatives to Further Improve Reimbursement

Photocure ASA, The Bladder Cancer Company, with a site in Princeton, announced the publication of the study “Clinical and Economic Impact of Blue Light Cystoscopy in the Management of NMIBC at U.S. Ambulatory Surgical Centers: What is the Site-of-Service Disparity?” in Urologic Oncology. The research objective was to quantify the clinical and economic impact of the incorporation of BLC in the management of NMIBC in ambulatory surgical centers (ASCs) considering 2022 Center for Medicare Services (CMS) patient-physician coverage and reimbursement. In the U.S., BLC for NMIBC surveillance in the ASC setting involves a flexible cystoscopy, an outpatient procedure without need of general anesthesia, allowing additional OR time for other hospital procedures. In the published clinical and health economic model, use of BLC resulted in the identification of 5 additional NMIBC recurrences compared to white light cystoscopy alone. 

Amneal Announces Collaboration With Orion Corp., Bringing its Products to Europe, Australia and New Zealand

Bridgewater-based Amneal Pharmaceuticals signed a long-term license agreement with Orion Corp. to bring its portfolio of complex generic programs to most parts of Europe as well as Australia and New Zealand. The financial terms of the agreement were not disclosed. The initial portfolio will include a mix of generic products commercially available in the U.S., as well as select high-value pipeline products currently under development. With its portfolio of essential medicines, Amneal experienced a very productive year in 2022. The company successfully launched 26 new generic products. In the fourth quarter of 2022 alone, it launched eight new generic products, including clindamycin phosphate gel 1%, ipratropium bromide nasal sprays and prednisolone sodium phosphate oral solution.

BrainStorm Cell Therapeutics Partners With NEALS, The ALS Association, and I AM ALS to Provide Public Access to Biospecimens from NurOwn's Phase 3 ALS Study

BrainStorm Cell Therapeutics Inc., with a site in Hackensack, announced that BCLI will donate biospecimens from NurOwn's placebo-controlled Phase 3 ALS trial to the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) biorepository for use by the research community. The specimens are being submitted to the biorepository in connection with a $500,000 grant previously awarded to BrainStorm by The ALS Association and I AM ALS, to support biomarker research. The specimens submitted to the NEALS biorepository include serum and cerebrospinal fluid (CSF) samples collected from trial participants treated with placebo. Samples of both biofluids were collected in a matched fashion and longitudinally at multiple (up to seven) time points over a 20-week period, allowing for the evaluation of intra-patient biomarker changes over time as well as comparisons of biomarker levels between serum and CSF.

Hikma and Junshi Biosciences Sign Exclusive Licensing Agreement for Cancer Treatment Drug Toripalimab for the Middle East and North Africa Region

Hikma Pharmaceuticals, with a site in Berkeley Heights, announced a new exclusive licensing and commercialization agreement with Junshi Biosciences. Under the terms of the agreement, Hikma has an exclusive license to develop and commercialize toripalimab injection in all its MENA markets. In addition, Hikma has the right of first negotiation for the future commercialization of three under development drugs in MENA. Toripalimab is an innovative anti-PD-1 monoclonal antibody approved for marketing in China for six indications to date. Over thirty toripalimab clinical studies covering more than fifteen indications have been conducted globally, including in China, the United States, Southeast Asia and Europe. Ongoing or completed pivotal clinical studies evaluating the safety and efficacy of toripalimab cover a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin, among others.

Celularity Appoints Paul Graves as Chief Communications Officer

Florham Park-based BioNJ Member Celularity Inc. announced that Paul Graves has been appointed Senior Vice President, Chief Communications Officer. Mr. Graves is a seasoned communications professional who brings more than 30 years of strategic communications experience in the biopharmaceutical industry. He has deep expertise in corporate reputation, global research and development, product launch and promotion, investor relations and public affairs. He will provide senior strategic communications leadership for Celularity as it advances its broad-based clinical pipeline toward U.S. Food and Drug Administration (FDA) submissions and approvals. Most recently, Mr. Graves served as vice president, global head of research and development (R&D) communication at the Janssen Pharmaceutical Companies of Johnson & Johnson. 

Tevogen Bio Appoints Cell Therapy Expert as Global Clinical Development Lead

Warren-based BioNJ Member Tevogen Bio announced it has appointed Delores Grosso as Global Clinical Development Lead to implement the company’s expanding clinical trial portfolio. Ms. Grosso has written over 20 investigator-initiated research protocols with multiple first and last author publications. She has served as a research mentor to numerous physician fellows, including several participating in a Master’s in Pharmacology Program at Jefferson. “After 40 years caring for patients, I highly value Tevogen Bio’s commitment to developing next-generation affordable immunotherapies,” Ms. Grosso stated. “As a researcher, I am excited to participate in the expansion of Tevogen’s precision T-cell platform for the novel treatment of disease.”

Helsinn Announces the Appointment of Dr. Melanie Rolli as Group Chief Executive Officer

BioNJ Member Helsinn Group, with a site in Iselin, announced the appointment of the Group’s current Chief Operating Officer, Dr. Melanie Rolli as Group Chief Executive Officer. Giorgio Calderari, former Group CEO, will transition to Vice Chairman of the Board. Dr. Rolli joined the enterprise in the summer of 2022 as Chief Operating Officer, with over 20 years’ experience across pharmaceutical and biotech companies, with exceptional experience in the development and commercialization of oncology products. Previous management roles include CEO of AELIX and CEO of Piqur Therapeutics in the areas of HIV vaccination, oncology and dermato-oncology respectively. Prior to this, Dr. Rolli spent 14 years at Novartis in a number of roles with increasing responsibilities.

ADC Therapeutics Appoints Mohamed Zaki as Chief Medical Officer

BioNJ Member ADC Therapeutics, with a site in New Providence, announced the appointment of industry veteran Mohamed Zaki, M.D., Ph.D., as Chief Medical Officer. He will serve on the company’s executive leadership team. Dr. Zaki succeeds Joseph Camardo, M.D., who is stepping down from full-time work to pursue other avenues to engage with the pharmaceutical industry and patient community. “ADC Therapeutics is a pioneer and leader in the evolving field of antibody drug conjugates,” said Dr. Zaki. “I look forward to working with the company’s experienced team of researchers to expand on their innovative science and bring more therapeutic options to those impacted by cancer worldwide.” 

BrainStorm Cell Therapeutics Announces Promotion of Dr. Stacy Lindborg to Co-Chief Executive Officer

BrainStorm Cell Therapeutics Inc., with a site in Hackensack, announced the promotion of Dr. Stacy Lindborg to the role of Co-Chief Executive Officer. Dr. Lindborg, who joined BrainStorm in 2020 and is based in the U.S., brings to her expanded role more than 25 years of pharmaceutical industry experience in R&D, strategy development, regulatory and analytics. She will work alongside Mr. Chaim Lebovits, who is retaining his position as President and CEO. In addition, Dr. Ralph Kern will retire from his current role as President and Chief Medical Officer later this month but will continue to support BrainStorm as part of the company's Scientific Advisory Board.

BD Announces Leadership Transitions

Franklin Lakes-based BD announced that Michelle Quinn has been named acting general counsel. Currently Senior Vice President, Deputy General Counsel, and Chief Ethics and Compliance Officer, Ms. Quinn succeeds Samrat “Sam” Khichi, who is leaving the company “for a new opportunity,” effective Feb. 3. Ms. Quinn joined the company in 2019. Now, she will be responsible for leading BD’s Law Group and will report to Chairman, CEO and President, Tom Polen. She will also become a member of BD’s executive leadership team. Before joining BD, Ms. Quinn was Vice President and General Counsel of North America for Sandoz Inc., a division of Novartis. She also served as Vice President and Associate General Counsel at Catalent Pharma Solutions.

Mallinckrodt Announces Appointment of Dr. Peter Richardson, MRCP, as Chief Scientific Officer

Mallinckrodt, with a site in Bedminster, announced that Dr. Peter Richardson, B.Med.Sci., BM, BS, MRCP (UK) has been appointed Executive Vice President and Chief Scientific Officer. With more than 30 years of research and development experience in the pharmaceutical industry, Dr. Richardson will have executive responsibility for Mallinckrodt's branded research and development (R&D), medical affairs, safety, and regulatory affairs functions. He will serve on the company's Executive Committee. Dr. Richardson previously served as Executive Vice President, Head of Research and Development and Chief Medical Officer at Antares Pharmaceuticals, overseeing all pharmaceutical research and development activities across the organization. Prior to this role, Dr. Richardson held senior leadership positions in research and development at several pharmaceutical companies.

Institute for Life Science Entrepreneurship Awarded $2.5 Million Appropriation in the FY2023 Federal Omnibus Bill

The Institute for Life Science Entrepreneurship (ILSE) at Kean University was awarded a $2.5 million appropriation in the recent federal omnibus spending bill to expand its life science incubator for biotech entrepreneurs in New Jersey. U.S. Senator Robert Menendez of New Jersey sponsored the appropriation, which supports ILSE’s $10 million capital campaign and will fund the creation of new offices and biological and chemical laboratories for start-up companies to establish operations at ILSE. The labs also will provide expanded opportunities for Kean faculty research and student experiential learning. “We are thrilled to receive this investment to build a larger critical mass of entrepreneurs, academic and industry scientists, investors and innovators on Kean’s campus,” said Thomas Richardson, Ph.D., ILSE President. “We anticipate the expanded facility will ultimately house up to 30 start-ups and employ 75 to 100 scientists performing pioneering research leading to transformative technologies for health care.”

Kean to Receive $4.25M in Federal Funding to Support 3 Key Initiatives

Kean University announced the school will receive more than $4 million for a series of new initiatives tied to its new designation as an urban research university under the federal spending bill signed into law by President Joe Biden. The $1.7 trillion spending plan includes a total of $4.25 million specifically for new Kean projects, including a higher education program for middle and high school students and their families; a neighborhood revitalization project in Trenton; and expanded research facilities for life science and biotech startups housed at Kean. “As the State’s urban research university, Kean is a leader in serving students from all backgrounds and conducting research that makes a difference in our communities,” Kean President Lamont Repollet said. “This funding will strengthen New Jersey’s technology economy, open more opportunities for students and revitalize one of our State’s great cities.”

BioCentriq Hires David Smith as Vice President of Development

As BioCentriq advances toward becoming a leading CDMO, it is proudly building a solid leadership team to carry out BioCentriq’s mission of bringing drug therapy products to patients worldwide. With combined decades of experience, BioCentriq’s strong leadership team is now welcoming David Smith, Ph.D., aboard as Vice President of Development. With 15+ years of experience in the field of regenerative medicine, Dr. Smith’s proven expertise and background in process development and engineering have led to a history of success in advancing cell therapy product manufacturing. His skills and experience include design of experiments, quality-by-design, R&D, data analysis, strong leadership and cross-functional oversight, among many others. In his new role at BioCentriq, Smith will lead MS&T, Process Development and Analytical Method Development. 

$1.14M in Funding Secured for Seton Hall’s Interprofessional Health Science Campus

Seton Hall University‘s Interprofessional Health Sciences campus will receive a major boost through federal funding secured by U.S. Rep. Bill Pascrell and U.S. Sens. Robert Menendez and Cory Booker. Home to its School of Health and Medical Sciences and College of Nursing, the funding will provide upgrades and new equipment that will allow the school to prepare future health care professionals to work in a patient-centered, integrated health care delivery system and will address health care workforce shortages and demand throughout New Jersey and beyond. Anticipating continued increases in demand for health care education, Seton Hall requested funds for: Enhancements to the simulation lab experience; construction of control rooms; simulation education equipment; and to mimic points of care.