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• SciMar ONE, Inc. 

 Learn more at www.BioNJ.org/Membership.

Two Years of U.S. COVID-19 Vaccines Have Prevented Millions of Hospitalizations and Deaths

It has been two years since the first COVID-19 vaccine was given to a patient in the United States. Since then, the U.S. has administered more than 655 million doses – 80 percent of the population has received at least one dose – with the cumulative effect of preventing more than 18 million additional hospitalizations and more than 3 million additional deaths. The swift development of the vaccine, emergency authorization to distribute widely and rapid rollout have been instrumental in curbing hospitalization and death, while mitigating socioeconomic repercussions of the pandemic.

Three Things to Know About the Accelerated Approval Pathway

If you or a loved one were to receive a diagnosis of a serious or life-threatening disease, you would likely want access to a U.S. Food and Drug Administration (FDA) approved safe and effective medicine as soon as possible. That’s precisely why the accelerated approval pathway was created back in 1992 at the urging of patient advocates. Since then, it has been a lifeline for millions of patients. Primarily utilized for cancers, rare diseases and HIV/AIDS, the pathway allows patients earlier access to potentially lifesaving medicines – often when no other treatment is available and when patients are facing difficult odds.

How the New Congress can Improve Drug Pricing Reform

Few aspects of U.S. health care arouse passions more than out-of-pocket prices for prescription drugs. Americans pay more for the same drugs than do patients in other countries, and we pay a higher share of GDP on prescription drugs. As the federal government moves forward with reforms set in motion in recent years, the new Congress should refocus on a more specific problem: too few incentives to sell generic or other lower-cost drugs and the role of pharmacy benefit managers (PBMs). While a few drugs are very expensive, the truth is that through a combination of effective policies, rules and more reliance on insurance, Americans’ average out-of-pocket expenditure for drugs has been falling.

ICER Misses…Again

The Institute for Clinical and Economic Review released another edition of their deeply flawed Unsupported Price Increase (UPI) Reports in a strained narrative to keep drug prices front and center for policymakers and the press. The incomplete data and arbitrary methodology behind this report should give anyone pause before using this report for decisions on patient access. ICER describes itself as an independent source that transparently reviews all available evidence to help align a treatment’s price with how well it improves the lives of patients and families. The UPI reports do none of these things. Using ICER’s brand to suggest independence and credibility for a misleading report with a subjective and ever-shifting methodology is problematic. Collaborating with those who prioritize price over value to “minimize administrative burden” and “generate adverse publicity” exposes the motivation of these reports.

Traditional HTAs Keep Diverse Patients Out of the Conversation

Concerns about the cost of health care have led to calls for the use of health technology assessments or HTAs by the federal government. This would allow government agencies to make important decisions about patient access to health care based on their determination of the value of new tests and treatments. As we work toward a more value-driven health care system, health technology assessments offer one tool that can be used by patients, physicians and health insurance companies to support these efforts. Yet it’s critical value assessments are more than just a simple budgetary exercise, especially in light of the recently passed Inflation Reduction Act that gives CMS unprecedented new power to make judgments about the relative clinical value of new treatments.

In Their Own Words: Insured Americans Support Policy Reforms That Address Access and Affordability Concerns

A survey of more than 5,000 Americans conducted with Ipsos found that insured Americans want better health care coverage. These insights come from the third installment of the Patient Experience Survey and show how Americans favor policy solutions that improve their ability to navigate and access their care by seeking to remove barriers imposed by insurers and middlemen, such as pharmacy benefit managers (PBMs). Here are three key findings, along with patients’ direct perspectives, from the latest PES report: 1) To insured Americans, better coverage means lower out-of-pocket costs, increased transparency and more predictability, 2) Patients want to eliminate inefficiencies introduced by insurance companies and PBMs and 3) Insured Americans support reforms that pass along the savings and make health care coverage more affordable.

Drug Pricing Reform in the Inflation Reduction Act: What Are The Implications?

The Inflation Reduction Act (IRA), signed by President Biden on August 16, 2022 is the most substantial drug payment and coverage legislation enacted since the Medicare Modernization Act of 2003. The IRA promises to have important consequences, both intended and unintended, across the health care system. Further, the new law gives the Centers for Medicare and Medicaid Services (CMS) considerable discretion to implement a multidimensional “maximum fair price” (“MFP”) for affected drugs at or below the regulated MFP ceiling, putting a substantial burden on CMS to implement a sustainable and predictable price determination framework.

New Appointment and Team Promotions at BioNJ

BioNJ, the trade association for New Jersey’s life sciences industry, announced that Pavita Howe, MBA, joined the organization as Vice President, Innovation and Entrepreneurship. In addition, a number of BioNJ Team members have been promoted. “As we enter our 30th year, BioNJ continues to expand its role within New Jersey’s vibrant life sciences ecosystem,” stated BioNJ President and CEO, Debbie Hart. “These promotions of our talented team members, along with the addition of the new position of Vice President, Innovation and Entrepreneurship, will further support BioNJ’s mission of helping our members help Patients. BioNJ is inspired by and privileged to work with those committed to improving the lives of patients and their families around the world.”

Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

BioNJ Member Ferring Pharmaceuticals, with a site in Parsippany, announced the U.S. Food and Drug Administration (FDA) approved Adstiladrin® (nadofaragene firadenovec-vncg), a novel adenovirus vector-based gene therapy, for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Adstiladrin, an intravesical therapy administered every three months, targets the patient’s own bladder wall cells to enhance the body’s natural defenses to fight cancer. The FDA approval was based on results of the Phase 3 clinical trial, which met its primary endpoint with more than half (51%, n=50 of 98; 95% CI 41 to 61) of patients with carcinoma in situ with or without concomitant high-grade Ta or T1 disease (CIS ± Ta/T1) achieving a complete response (CR) by three months. Of the patients who achieved an initial CR, 46% (n=23 of 50) continued to remain free of high-grade recurrence at 12 months.

ADMA Biologics Announces FDA Approval for Room Temperature Storage of ASCENIV™ & BIVIGAM®

Ramsey-based BioNJ Member ADMA Biologics, Inc. announced the United States Food and Drug Administration’s (“FDA”) approval for its supplemental Biologics License Applications (BLAs) for both ASCENIV and BIVIGAM to now include room temperature (25°C) storage conditions for up to 4 weeks during the first 24 months of the 36-month approved shelf life. The room temperature approval applies to all existing ASCENIV and BIVIGAM lots currently in the commercial supply chain as well as to future production of ASCENIV and BIVIGAM. The newly approved room temperature storage labeling for ASCENIV and BIVIGAM is immediately effective, and product is commercially available to U.S. healthcare providers and patients.

ADMA Biologics Announces Closing of $69 Million Public Offering Including Full Exercise of Underwriters’ Option to Purchase Additional Shares

Ramsey-based BioNJ Member ADMA Biologics, Inc. announced the closing of its previously announced underwritten public offering of 20,979,020 shares of its common stock at a public offering price of $2.86 per share, in addition to the exercise in full of the underwriters’ option to purchase an additional 3,146,853 shares of common stock. The gross proceeds from the exercise of the overallotment option were $9 million, bringing the total gross proceeds to ADMA from the offering to $69 million, before deducting underwriting discounts and commissions and other estimated offering expenses.

Y-mAbs’ DANYELZA® (naxitamab-gqgk) for the Treatment of High-Risk Neuroblastoma Approved in China

BioNJ Member Y-mAbs Therapeutics, Inc., with a site in Nutley, announced that the National Medical Products Administration in China has granted DANYELZA (naxitamab-gqgk) 40mg/10ml conditional approval. DANYELZA will be marketed in China by Y-mAbs’ partner SciClone Pharmaceuticals (Holdings) Limited. DANYELZA is a humanized, monoclonal antibody that targets the ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumors and sarcomas. DANYELZA is administered to patients three times a week in an outpatient setting and the treatment is repeated every four weeks. DANYELZA is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. 

Soligenix Announces Submission of New Drug Application to the FDA for HyBryte™ in the Treatment of Cutaneous T-cell Lymphoma

Princeton-based BioNJ Member Soligenix announced that it submitted a new drug application (NDA) to the United States (U.S.) Food and Drug Administration (FDA) for HyBryte™ (synthetic hypericin) in the treatment of early stage cutaneous T-cell lymphoma (CTCL), a rare cancer and area of unmet medical need affecting over 25,000 patients in the U.S. The company estimates the potential worldwide market for HyBryte™ to be in excess of $250 million for the treatment of CTCL. "The submission of our NDA is a significant milestone for Soligenix, positioning us well to fulfill our promise to offer an innovative first-in-class therapy to the CTCL community of patients, families and healthcare professionals," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix.

Soligenix Initiates Phase 2 Clinical Trial of SGX302 (synthetic hypericin) for the Treatment of Mild-to-Moderate Psoriasis

Princeton-based BioNJ Member Soligenix announced that patient enrollment has been opened for its Phase 2a study (protocol number HPN-PSR-01) evaluating SGX302 (synthetic hypericin) in the treatment of mild-to-moderate psoriasis. "We are excited to expand synthetic hypericin's development into different cutaneous T-cell diseases such as psoriasis, as a component of our long-term strategy to enhance the value of this unique compound," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix. "Given our promising published results with hypericin to date, including a small Phase 1/2 proof of concept clinical trial in mild-to-moderate psoriasis, and the Phase 3 FLASH study in cutaneous T-cell lymphoma, where we filed a New Drug Application (NDA) this month, we are hopeful synthetic hypericin will have a role to play in helping patients suffering from this difficult to treat and chronic disease." 

Soligenix Announces Publication Demonstrating Positive Correlates of Protection With RiVax® in Non-Human Primate Survival after Ricin Challenge

Princeton-based BioNJ Member Soligenix announced publication of preclinical immunogenicity challenge studies for RiVax® (heat stable ricin toxin vaccine) demonstrating statistically significant correlates of protection predicting survival after lethal aerosolized ricin challenge in non-human primates (NHPs). The article titled "Serum antibody profiling identifies vaccine-induced correlates of protection against aerosolized ricin toxin in rhesus macaques" has been accepted for publication in the journal npj Vaccines. The article results from collaborative work lead by the Wadsworth Center of the New York State Department of Health. This same thermostabilization approach is also being advanced in the development of Soligenix's CiVax™ vaccine for COVID-19 and SuVax™ and MarVax™ vaccines for ebola type filovirus infections.

Tevogen Bio to Study its Investigational COVID-19 T Cell Therapy in Immunocompromised Patients Who are Unable to Benefit from Currently Available Prevention or Treatment Options

Warren-based BioNJ Member Tevogen Bio announced its intention to study the potential therapeutic use of its investigational COVID-19 T cell therapy, TVGN-489, in immunocompromised patients. There exists a significant unmet need for the treatment of COVID-19 in immunocompromised patients. Currently, there are very few treatment options that have not been impacted by variants or are suitable for this patient population due to possible medication interactions. A comprehensive review, completed by the company’s Research and Development team, led by Dr. Neal Flomenberg, Tevogen’s Chief Scientific Officer, found that TVGN-489 is expected to retain activity against recent variants, including XBB and BQ and its subtypes, which appear to have the ability to evade some existing prophylactic options and immunity. 

Amicus Therapeutics Receives Positive CHMP Opinion for Pombiliti™ (cipaglucosidase alfa) for Late-Onset Pompe Disease

BioNJ Member Amicus Therapeutics, with a site in Cranbury, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization of cipaglucosidase alfa, a long-term enzyme replacement therapy (ERT) used in combination with miglustat for adults with late-onset Pompe disease (LOPD). A decision from the European Commission (EC) on cipaglucosidase alfa, the enzyme replacement therapy component of AT-GAA, is expected in the first quarter of 2023. Cipaglucosidase alfa will be commercialized under the brand name POMBILITI™. The company expects a CHMP opinion of miglustat, the enzyme stabilizer component of AT-GAA, in the second quarter of 2023.

Amarin Announces VAZKEPA® (icosapent ethyl) Receives Regulatory Approval in Switzerland

Bridgewater-based BioNJ Member Amarin Corporation plc announced that Switzerland’s Swissmedic has granted approval to VAZKEPA (icosapent ethyl). Swissmedic approved VAZKEPA to reduce risk of cardiovascular events in adult statin-treated patients at high cardiovascular risk with elevated triglycerides (≥ 150 mg/dL [≥ 1.7 mmol/L]) and established cardiovascular disease, or diabetes and at least one other cardiovascular risk factor. In parallel with the regulatory submission, Amarin had already initiated the process for obtaining national pricing and reimbursement in Switzerland, which is expected to conclude in the course of 2023. According to the European Heart Network, the burden of cardiovascular disease (CVD) is greater than that of any other disease and the leading cause of death in Europe and in the world. 60 million people are living with CVD in the EU, with 13 million new cases of CVD each year. 

Hovione and GEA Announce a Strategic Collaboration to Advance Continuous Tableting

BioNJ Member Hovione, with a site in East Windsor, and GEA announced a strategic collaboration to advance Continuous Tableting. This partnership stemmed from a successful customer-supplier relationship spanning several years. It combines GEA´s engineering expertise with Hovione´s development and manufacturing experience and both parties commit to partner to accelerate the adoption of Continuous Tableting technology. The advantages of continuous over batch manufacturing are well known. Continuous production systems allow for leaner and risk-reduced development paths, leaner supply chains, increased built-in quality, and in general, manufacturing processes of greater flexibility and reduced complexity. In the context of this strategic collaboration, Hovione and GEA will combine their strengths to further advance the technology and contribute to the establishment of new standards and new levels of market acceptance. 

Tactical Therapeutics New Drug Carboxyamidotriazole Orotate (CTO) is Getting Notoriety for its Success in Treating Brain Cancer Patients Who Went Through a Phase 1 trial at the Memorial Sloan Kettering Cancer Center

Rashida Karmali, Ph. D, CEO at BioNJ Member Tactical Therapeutics is very excited that high-profile organizations and healthcare partners are recognizing her company after the results of their Phase 1 trial for its drug called Carboxyamidotriazole Orotate, or CTO. The drug has been tested on patients with different types of advanced solid cancers as well as on patients of Glioblastoma, recurrent (rGBM) and newly diagnosed (nGBM). Speaking on the success of CTO and its potential for more impact if funded for future trials, Dr. Antonio Omuro, Fellow of the American Academy of Neurology, had this to say: “We are excited about CTO because it has shown highly promising results in patients with glioblastoma and other malignant gliomas, which are devastating diseases.” 

AbbVie Submits Supplemental New Drug Application to U.S. FDA to Support New Indication of LINZESS® (linaclotide) for Functional Constipation in Children and Adolescents 6 to 17 Years of Age

BioNJ Member AbbVie, with a site in Princeton, announced that it has submitted a supplemental New Drug Application (sNDA) for linaclotide (LINZESS®) to the U.S. Food and Drug Administration (FDA) for the treatment of children and adolescents 6 to 17 years of age with functional constipation (FC). The sNDA submission is based on results from a Phase 3 clinical trial, which met the primary and secondary endpoints, evaluating linaclotide (72 mcg) for increased frequency of spontaneous bowel movements (SBM) and improvement in stool consistency in patients aged 6 to17 years. LINZESS is developed and marketed by AbbVie and Ironwood Pharmaceuticals in the United States and is currently indicated for the treatment of adults with chronic idiopathic constipation (CIC) or irritable bowel syndrome with constipation (IBS-C).

U.S. FDA Approves VRAYLAR® (cariprazine) as an Adjunctive Treatment for Major Depressive Disorder

BioNJ Member AbbVie, with a site in Princeton, announced that the U.S. Food and Drug Administration (FDA) has approved VRAYLAR® (cariprazine) as an adjunctive therapy to antidepressants for the treatment of major depressive disorder (MDD) in adults. Supported by clinical data demonstrating efficacy and well-established tolerability, this additional indication provides a new option for adults who have a partial response to the treatment of an antidepressant. MDD is one of the most common mental disorders in the U.S.; approximately one in five adults will experience this disorder during their lifetime. In a large U.S. study of adults with MDD, approximately 50 percent still had depressive symptoms with their first antidepressant. 

BeiGene’s BRUKINSA® (zanubrutinib) Demonstrated Superior Progression-Free Survival Over IMBRUVICA® (ibrutinib) in Chronic Lymphocytic Leukemia in Late-Breaker at ASH

BioNJ Member BeiGene with a site in Pennington, presented the final progression-free survival (PFS) analysis of the ALPINE trial demonstrating superior efficacy and a favorable cardiac safety profile for patients receiving BRUKINSA® as compared to IMBRUVICA® in a global phase 3 trial in patients with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL). In this final analysis, BRUKINSA achieved superior PFS over ibrutinib (HR: 0.65 [95% CI, 0.49-0.86] p=0.0024, for both Independent Review Committee [IRC] and investigator). At 24 months, the investigator-assessed PFS rates were 78.4% for BRUKINSA compared to 65.9% with ibrutinib. The PFS benefit was observed across all major subgroups, including high-risk del(17p)/TP53 (HR: 0.52; [95% CI, 0.30-0.88]), as assessed by IRC. BRUKINSA also demonstrated higher overall response rate (ORR), with a response rate of 80.4% versus 72.9% (two-sided p=0.0264), as assessed by IRC.

Taiho Oncology and Astex Pharmaceuticals Present Overall Survival Data for Oral Decitabine and Cedazuridine (INQOVI®, ASTX727) in Patients With MDS and CMML Harboring TP53 Mutations at 64th ASH Annual Meeting

BioNJ Member Taiho Oncology, Inc., with a site in Princeton, and Astex Pharmaceuticals, Inc. announced preliminary data from the Phase 3 ASCERTAIN trial assessing overall and leukemia-free survival in adults with intermediate and high-risk myelodysplastic syndromes (MDS) including chronic myelomonocytic leukemia (CMML) harboring biallelic TP53 mutations following treatment with oral decitabine and cedazuridine (ASTX727). In the study, the population of patients harboring a TP53 mutation (44 of 125 patients) was characterized by allelic status: 14 patients had biallelic mutations and 30 patients had monoallelic mutations without other chromosomal deletions. The median OS (mOS) in patients treated with ASTX727 with biallelic vs. monoallelic mutations was 13.0 months (95% Confidence Interval [CI]: 5.3, 29.1) vs. 29.2 months (95% CI: 19.8, NE).

Evotec Achieves Further Progress in Neuroscience Collaboration With Bristol Myers Squibb

Princeton-based BioNJ Member Evotec announced that the company has achieved further significant progress within its neuroscience collaboration with BioNJ Member Bristol Myers Squibb. The collaboration expands the portfolio by two additional drug discovery projects and has designated a target-based program for further development, triggering payments in total of US$ 26 m to Evotec. The collaboration was initiated in December 2016 with the goal of identifying disease-modifying treatments for a broad range of neurodegenerative diseases. Currently approved drugs only offer short-term management of the patients' symptoms and there is a huge unmet medical need for treatments that slow down or reverse disease progression. The collaboration leverages Evotec's precision medicine technologies for modality-agnostic drug discovery and development. 

GSK and Wave Life Sciences Announce Collaboration to Drive Discovery and Development of Oligonucleotide Therapeutics Focusing on Novel Genetic Targets

Warren-based BioNJ Member GSK and Wave Life Sciences announced a strategic collaboration to advance oligonucleotide therapeutics, including Wave’s preclinical RNA editing program targeting alpha-1 antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial four-year research term. It combines GSK’s unique insights from human genetics, as well as its global development and commercial capabilities, with Wave’s proprietary discovery and drug development platform, PRISMTM. Wave’s PRISM platform is the only oligonucleotide platform offering three RNA-targeting modalities (editing, splicing, and silencing, including siRNA and antisense). Importantly, these modalities incorporate novel chemistry, including PN backbone chemistry and control of stereochemistry, to optimize the pharmacological properties of therapeutic oligonucleotides.

Dupixent® (dupilumab) Approved by European Commission as the First and Only Targeted Medicine Indicated for Prurigo Nodularis

Bridgewater-based BioNJ Member Sanofi announced the European Commission (EC) has expanded the marketing authorization for Dupixent® (dupilumab) in the European Union to treat adults with moderate-to-severe prurigo nodularis who are candidates for systemic therapy. The EC decision is based on data from two Phase 3 trials, PRIME and PRIME2, evaluating the efficacy and safety of Dupixent (PRIME n=75; PRIME2 n=78) in adults with uncontrolled prurigo nodularis compared to placebo (PRIME n=76; PRIME2 n=82). In these trials, respectively, 44% and 37% of Dupixent patients experienced a clinically meaningful reduction in itch at 12 weeks, compared to 16% and 22% for placebo. The improvement further increased at 24 weeks, with approximately three times as many Dupixent patients (60% and 58%) experiencing a clinically meaningful reduction in itch from baseline, compared to placebo (18% and 20%).  

Sanofi and Innate Pharma Expand Collaboration for Natural Killer Cell Therapeutics in Oncology

Bridgewater-based BioNJ Member Sanofi and Innate Pharma announced an expansion of their collaboration, with Sanofi licensing a natural killer (NK) cell engager program targeting B7H3 from Innate’s ANKET^TM (Antibody-based NK Cell Engager Therapeutics) platform. Sanofi will also have the option to add up to two additional ANKET^TM targets. Upon candidate selection, Sanofi will be responsible for all development, manufacturing and commercialization. Innate and Sanofi signed a first NK cell engagers collaboration in 2016 for the generation and evaluation of up to two bispecific NK cell engagers, which are currently being evaluated by Sanofi’s R&D team, with one of these molecules already in clinical studies.

Merck and Kelun-Biotech Announce Exclusive License and Collaboration Agreement for Seven Investigational Antibody-drug Conjugate Candidates for the Treatment of Cancer

Kenilworth-based BioNJ Member Merck & Co and and Kelun-Biotech, a clinical-stage biotech company focused on biologic and small molecule discovery and development, announced that the companies have entered into an exclusive license and collaboration agreement to develop seven investigational preclinical antibody-drug conjugates (ADC) for the treatment of cancer. Under the agreement, Kelun-Biotech has granted Merck exclusive global licenses to research, develop, manufacture and commercialize multiple investigational preclinical ADC therapies and exclusive options to obtain additional licenses to ADC candidates. Kelun-Biotech retains the right to research, develop, manufacture and commercialize certain licensed and option ADCs for mainland China, Hong Kong and Macau.

LYNPARZA® (olaparib) in Combination With Abiraterone and Prednisone or Prednisolone Approved in the EU as Treatment for Certain Patients With Metastatic Castration-Resistant Prostate Cancer

Kenilworth-based BioNJ Member Merck & Co and AstraZeneca announced that LYNPARZA has been approved in the European Union (EU) in combination with abiraterone and prednisone or prednisolone for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated. This approval by the European Commission follows the positive recommendation from the Committee for Medicinal Products for Human Use received in November this year and was based on the Phase 3 PROpel trial, results of which were published in NEJM Evidence in June 2022. In PROpel, LYNPARZA in combination with abiraterone and prednisone or prednisolone reduced the risk of disease progression or death by 34% (HR=0.66 [95% CI, 0.54-0.81]; p<0.0001) versus placebo plus abiraterone and prednisone or prednisolone, based on investigator assessment.

Merck and AstraZeneca Provide Update on U.S. Regulatory Review of LYNPARZA® (olaparib) in Combination With Abiraterone and Prednisone or Prednisolone as Treatment of Metastatic Castration-Resistant Prostate Cancer

Kenilworth-based BioNJ Member Merck & Co and AstraZeneca announced that the U.S. Food and Drug Administration (FDA) has informed AstraZeneca that the agency will extend by three months the Prescription Drug User Fee Act (PDUFA) date for the pending supplemental new drug application (sNDA) for LYNPARZA in combination with abiraterone and prednisone or prednisolone for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC). The purpose of the extension is to provide further time for the full review of the submission. The companies will continue to work with the FDA to facilitate the completion of the agency’s review. 

Merck and Moderna Announce mRNA-4157/V940, an Investigational Personalized mRNA Cancer Vaccine, in Combination With KEYTRUDA® (pembrolizumab), Met Primary Efficacy Endpoint in Phase 2b KEYNOTE-942 Trial

Kenilworth-based BioNJ Member Merck & Co. and Moderna, Inc. announced that the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial of mRNA-4157/V940, an investigational personalized mRNA cancer vaccine, in combination with KEYTRUDA®, Merck’s anti-PD-1 therapy, demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of recurrence-free survival (RFS) versus KEYTRUDA alone for the adjuvant treatment of patients with stage III/IV melanoma following complete resection. Adjuvant treatment with mRNA-4157/V940 in combination with KEYTRUDA reduced the risk of recurrence or death by 44% (HR=0.56 [95% CI, 0.31-1.08]; one-sided p value=0.0266) compared with KEYTRUDA alone. The companies plan to discuss the results with regulatory authorities and initiate a Phase 3 study in melanoma patients in 2023.

Astellas, Seagen and Merck Announce FDA Acceptance of Supplemental Biologics License Applications for PADCEV® (enfortumab vedotin-ejfv) With KEYTRUDA® (pembrolizumab) for the First-Line Treatment of Certain Patients With Locally Advanced or Metastatic Urothelial Cancer

Kenilworth-based BioNJ Member Merck & Co., Astellas Pharma Inc. and Seagen Inc. announced the U.S. Food and Drug Administration (FDA) has accepted for priority review supplemental Biologics License Applications (sBLAs) for PADCEV® (enfortumab vedotin-ejfv) and KEYTRUDA® (pembrolizumab) for use of these two agents in combination for the treatment of patients with locally advanced or metastatic urothelial cancer (la/mUC) who are not eligible to receive cisplatin-containing chemotherapy. The respective applications are intended to expand both labels for PADCEV and KEYTRUDA. The agency set a Prescription Drug User Fee Act (PDUFA) goal date for each application of April 21, 2023.

Merck Begins Tender Offer to Acquire Imago BioSciences, Inc.

Kenilworth-based BioNJ Member Merck & Co. is commencing, through a subsidiary, a cash tender offer to purchase all outstanding shares of common stock of Imago BioSciences, Inc. On Nov. 21, 2022, Merck announced that it had entered into a definitive agreement to acquire Imago. Upon the successful closing of the tender offer, stockholders of Imago will receive $36 in cash for each share of Imago common stock validly tendered and not validly withdrawn in the offer, without interest and subject to deduction for any required tax withholding. Following the purchase of shares in the tender offer, Imago will merge with and into a subsidiary of Merck, with Imago surviving the merger. As a result, Imago will become a subsidiary of Merck. 

Pfizer Presents Updated Favorable Elranatamab Data from Pivotal Phase 2 MagnetisMM-3 Trial

BioNJ Member Pfizer, with a site in Peapack, announced 10.4 month follow-up data from the pivotal Phase 2 MagnetisMM-3 clinical trial suggesting elranatamab, a B-cell maturation antigen (BCMA)-CD3-targeted bispecific antibody (BsAb), is efficacious and has a manageable safety profile in patients with relapsed or refractory multiple myeloma (RRMM) in a heavily pretreated population, who have received at least three classes of prior therapies including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody (i.e. triple-class refractory or exposed). Elranatamab is an investigational humanized BsAb that targets both BCMA-expressing multiple myeloma cells and CD3-expressing T-cells, bridging them together and activating the T-cells to kill the myeloma cells. 

Pfizer and BioNTech Receive U.S. FDA Emergency Use Authorization for Omicron BA.4/BA.5-Adapted Bivalent COVID-19 Vaccine in Children Under 5 Years

BioNJ Member Pfizer, with a site in Peapack, and BioNTech announced the U.S. Food and Drug Administration (FDA) granted Emergency Use Authorization (EUA) of their Omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine as the third 3-µg dose in the three-dose primary series for children 6 months through 4 years of age. Children in this age group can receive a primary series consisting of two 3-µg doses of the original Pfizer-BioNTech COVID-19 Vaccine followed by a third 3-µg dose of the bivalent vaccine to complete the primary series. Vaccine doses can be delivered immediately upon receipt of a recommendation from the U.S. Centers for Disease Control and Prevention ("CDC"). 

First-in-Class, Twice-Yearly Treatment Option for People Living With Multi-Drug Resistant HIV

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, announced that Sunlenca® (lenacapavir), in combination with other antiretroviral(s) (ARV), has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of HIV-1 infection in heavily treatment-experienced (HTE) adults with multi-drug resistant (MDR) HIV-1 infection. Sunlenca has a multi-stage mechanism of action distinguishable from other currently approved classes of antiviral agents and no known cross resistance exhibited in vitro to other existing drug classes. Sunlenca offers a new, twice-yearly treatment option for adults with HIV that is not adequately controlled by their current treatment regimen.

Anti-TIGIT Domvanalimab-Containing Study Arms Improve Progression-Free Survival Compared to Anti-PD1 Alone in Phase 2 Non-Small Cell Lung Cancer Study

BioNJ Member Gilead Sciences, Inc., with a site in Morris Plains, and Arcus Biosciences, Inc. announced positive results from the fourth interim analysis of the 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 a Phase 2, multicenter, three-arm, randomized, open-label study evaluating the combinations of Fc-silent anti-TIGIT monoclonal antibody domvanalimab plus anti-PD1 monoclonal antibody zimberelimab (doublet) and domvanalimab plus zimberelimab and etrumadenant, an A2a/b adenosine receptor antagonist (triplet), versus zimberelimab monotherapy. 

Novartis Investigational Iptacopan Provides Clinically Meaningful Increases in Hemoglobin Levels in Complement-Inhibitor-Naïve Patients With PNH

East Hanover-based BioNJ Member Novartis announced the Phase III APPOINT-PNH study (NCT04820530) of investigational oral monotherapy iptacopan in complement-inhibitor-naïve (including anti-C5 therapies) adults with PNH met its primary endpoint. Topline results showed a significant proportion of patients treated with iptacopan (200 mg twice daily) achieved clinically meaningful hemoglobin-level increases of 2 g/dL or more from baseline without the need for blood transfusions at 24 weeks. In the study, the safety profile of iptacopan monotherapy was consistent with previously reported data. “This second iptacopan readout for PNH underscores the robust potential for this therapy, enabling us to submit a broad regulatory package with the goal of iptacopan potentially becoming the first oral monotherapy for PNH,” said David Soergel, M.D., Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis.

Novartis Receives European Commission Approval for Pluvicto® as the First Targeted Radioligand Therapy for Treatment of Progressive PSMA–Positive Metastatic Castration-Resistant Prostate Cancer

East Hanover-based BioNJ Member Novartis announced the European Commission (EC) approved Pluvicto® (INN: lutetium (177Lu) vipivotide tetraxetan), a targeted radioligand therapy. Pluvicto® is approved in combination with androgen deprivation therapy (ADT) with or without androgen receptor (AR) pathway inhibition, for the treatment of adult patients with prostate-specific membrane antigen (PSMA)–positive metastatic castration-resistant prostate cancer (mCRPC). These patients have been treated with AR pathway inhibition and taxane-based chemotherapy. The approval follows a positive opinion issued in October by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and is applicable to all 27 European Union member states plus Iceland, Norway, Northern Ireland and Liechtenstein.

Novartis Presents Pivotal Phase III APPLY-PNH Data at ASH Demonstrating Investigational Oral Monotherapy Iptacopan Superiority Over Anti-C5

East Hanover-based BioNJ Member Novartis announced detailed results from the pivotal Phase III APPLY-PNH trial. The results showed a vast majority of patients with paroxysmal nocturnal hemoglobinuria (PNH) who received the investigational oral monotherapy iptacopan achieved clinically meaningful increases in hemoglobin levels compared to anti-C5 therapy. The study met both primary endpoints and most secondary endpoints, with iptacopan demonstrating superiority over anti-C5 therapy in adult patients with PNH experiencing residual anemia despite prior treatment with anti-C5 therapy. In the study, the safety profile of iptacopan monotherapy was consistent with previously reported data, with no serious infections caused by encapsulated bacteria. 

Lilly and ProQR to Expand RNA Editing Collaboration

BioNJ Member Eli Lilly and Company, with a site in Branchburg, and  ProQR Therapeutics N.V. announced the expansion of their licensing and collaboration agreement focused on the discovery, development and commercialization of new genetic medicines. The collaboration, originally announced in September 2021, applied ProQR’s proprietary Axiomer® RNA editing platform to target disorders of the liver and nervous system. Through the course of work to date, advances in the platform have significantly increased editing efficiency and refined biodistribution in both the liver and nervous system, opening up new potential applications to not only correct known mutations, but also introduce protective variants in specific transcripts. Through this expanded collaboration, Lilly and ProQR will explore further applications of the Axiomer platform to unlock new innovative treatments for people living with diseases with high unmet medical need.

Lilly and EVA Pharma Announce Collaboration to Enhance Sustainable Access to Affordable Insulin in Africa

BioNJ Member Eli Lilly and Company, with a site in Branchburg, and EVA Pharma announced a collaboration to deliver a sustainable supply of high-quality, affordable human and analogue insulin to at least one million people living with type 1 and type 2 diabetes in low- to middle-income countries (LMICs), most of which are in Africa. In a first for Lilly, the company will supply its active pharmaceutical ingredient (API) for insulin at a significantly reduced price to EVA Pharma. Lilly will also provide a pro-bono technology transfer to enable EVA Pharma to formulate, fill and finish insulin vials and cartridges – establishing the company as a trusted manufacturer of these lifesaving products in Africa.

Loxo@Lilly Presents Updated Pirtobrutinib Data from the Phase 1/2 BRUIN Clinical Trial at the 2022 American Society of Hematology Annual Meeting

Loxo@Lilly, the oncology unit of BioNJ Member Eli Lilly and Company, announced updated clinical data from the pirtobrutinib global Phase 1/2 BRUIN trial in patients with chronic lymphocytic leukemia (CLL), small lymphocytic lymphoma (SLL), mantle cell lymphoma (MCL), Richter transformation (RT), and Waldenström macroglobulinemia (WM). Pirtobrutinib is an investigational, highly selective, reversible (non-covalent) inhibitor of Bruton's tyrosine kinase (BTK). "These data build on previous results with significantly longer follow-up and continue to expand on the body of evidence supporting pirtobrutinib as a potential treatment option for patients previously treated with a covalent BTK inhibitor across a range of B-cell malignancies," said Susan O'Brien, M.D., UCI Health hematology oncologist, Chao Family Comprehensive Cancer Center and professor of hematology/oncology at University of California, Irvine. 

Real-World Study Shows Patients Treated With IMBRUVICA® (ibrutinib) Were Less Likely to Initiate a Next-Line Treatment than Patients on Acalabrutinib in First-line Chronic Lymphocytic Leukemia

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced results of a real-world study showing that patients with chronic lymphocytic leukemia (CLL) treated with first-line acalabrutinib monotherapy were 89 percent more likely to start a next-line treatment than those treated with IMBRUVICA® (ibrutinib). These data suggest the potential that first-line treatment with IMBRUVICA® in routine practice may provide patients with the ability to use once-daily, all-oral IMBRUVICA® as a monotherapy treatment for a longer period without the need to start the next line of therapy. The study used Acentrus, de-identified academic electronic medical records (EMR) to identify patients initiating first-line treatment with IMBRUVICA® or acalabrutinib between November 21, 2019 and April 30, 2022 and examined time to next treatment (TTNT) as a clinically meaningful surrogate measure for disease progression.

Janssen Presents Efficacy and Subgroup Analyses from MAIA Study Showing Long-Term Results of DARZALEX® (daratumumab)-based Regimen in Newly Diagnosed, Transplant-Ineligible Multiple Myeloma

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced new analyses from the Phase 3 MAIA study of DARZALEX® (daratumumab) in combination with lenalidomide and dexamethasone (D-Rd), evaluating progression-free survival (PFS), minimal residual disease (MRD) negativity and overall response rate (ORR) at a median follow-up of 64.5 months, and overall survival (OS) at a median follow-up of 73.6 months in newly diagnosed, transplant-ineligible (TIE) patients with multiple myeloma, regardless of patients’ age and across clinically important subgroups, as well as health-related quality of life (HRQoL) among frail TIE patients. “Initial data from the MAIA study were instrumental in establishing the D-Rd regimen as a standard of care for the treatment of patients with newly diagnosed, transplant-ineligible multiple myeloma,” said study author, Shaji Kumar, M.D., Consultant, Division of Hematology, Department of Internal Medicine, Mayo Clinic.”

Janssen Presents First Data from MajesTEC-2 Trial of TECVAYLI™ (teclistamab-cqyv) in Combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) and Lenalidomide in Relapsed or Refractory Multiple Myeloma

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced new results from a cohort of the Phase 1b MajesTEC-2 study of TECVAYLI™ (teclistamab-cqyv), a first-in-class, BCMAxCD3 bispecific T-cell engager antibody, in combination with DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) and lenalidomide. According to the results, the immune-based triplet therapy regimen had a manageable safety profile with no unexpected safety signals observed. A very good partial response (VGPR) or better was achieved by 90.3 percent of patients with relapsed or refractory multiple myeloma who had received one to three prior lines of therapy, including a proteasome inhibitor and immunomodulatory drug, with responses deepening over time. 

Janssen Presents New Data for Talquetamab, a First-in-Class GPRC5DxCD3 Bispecific Antibody, Suggesting Durable Responses in Patients With Heavily Pretreated Multiple Myeloma

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced updated results from the Phase 1/2 MonumenTAL-1 study of talquetamab, an investigational, off-the-shelf (ready to use), bispecific T-cell engager antibody. Talquetamab targets both GPRC5D, a novel target on multiple myeloma cells, and CD3 on T cells, activating the body’s immune system to fight this blood cancer. Results from the study suggest patients with relapsed or refractory multiple myeloma who received a median of five prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody, achieved overall response rates (ORR) of 74.1 percent and 73.1 percent with subcutaneously (SC) administered recommended Phase 2 doses (RP2Ds) of 0.4 mg/kg weekly and 0.8 mg/kg every two weeks, respectively, with a median duration of response (DOR) of nine months or longer.

New Results from the Phase 3 GLOW Study of Fixed-Duration Treatment With IMBRUVICA® (ibrutinib) Plus Venetoclax Demonstrate Robust Efficacy and Sustained Response in Older, Unfit Patients With Previously Untreated Chronic Lymphocytic Leukemia

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced new four-year follow-up results from the Phase 3 GLOW study, which showed investigational, fixed-duration treatment with IMBRUVICA® + venetoclax (I+V) reduced the risk of progression or death by 79 percent among older and/or unfit patients with previously untreated chronic lymphocytic leukemia (CLL) compared to patients treated with chemoimmunotherapy. CLL is the most common form of leukemia in adults in the U.S. and currently has no cure. While the treatment landscape has evolved significantly since the emergence of targeted agents, there is still significant unmet need for novel treatment options, including fixed-duration regimens.

Janssen Submits Biologics License Application to U.S. FDA for Talquetamab for the Treatment of Patients With Relapsed or Refractory Multiple Myeloma

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for talquetamab for the treatment of patients with relapsed or refractory multiple myeloma. Talquetamab is an investigational, off-the-shelf (ready to use), bispecific T-cell engager antibody targeting both GPRC5D, a novel drug target that is on some normal cells but overexpressed on myeloma cells, and separately targets CD3 on T cells. This BLA is supported by data from the Phase 1/2, first-in-human MonumenTAL-1 study of talquetamab in patients with relapsed or refractory multiple myeloma who have received more than three prior lines of therapy.

Real-World Study Confirms Benefit of XARELTO® (rivaroxaban) for Secondary Prevention of Venous Thromboembolism in Cancer Patients

Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced observational data from eight years of clinical practice showing that the oral Factor Xa inhibitor XARELTO® (rivaroxaban) is associated with comparable effectiveness and safety to the Factor Xa inhibitor apixaban for the treatment of cancer-associated thromboembolism (CAT) in a broad cohort of patients with various cancer types. Patients with CAT are at a higher risk of venous thromboembolism (VTE), which is the second-leading cause of death in people with cancer.¹ Data from the Observational Study in Cancer-Associated Thrombosis for Rivaroxaban (OSCAR) found XARELTO® showed non-inferiority for the composite outcome of recurrent VTE or any bleeding resulting in hospitalization for treatment of patients with CAT. 

Odronextamab (CD20xCD3) Demonstrates High and Durable Complete Response Rate Among Patients With Relapsed/Refractory Follicular Lymphoma in Pivotal Phase 2 Trial

Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced positive first data from a cohort of a pivotal Phase 2 trial evaluating investigational odronextamab in patients with heavily pre-treated, relapsed/refractory (R/R) follicular lymphoma (FL) grades 1 to 3a. The results will form the basis of planned submissions to regulatory authorities in 2023, including the U.S. Food and Drug Administration (FDA). Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing. "These positive pivotal Phase 2 results investigating odronextamab in heavily pre-treated, relapsed/refractory follicular lymphoma patients showed deep and durable response – confirming earlier findings in this program – with the highest complete response rates seen in this patient population to date,” said Tae Min Kim, M.D., Ph.D., Internal Medicine, Seoul National University Hospital.

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

Boehringer Ingelheim, with a site in North Brunswick, 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 empagliflozin compared with placebo for children and adolescents aged 10-17 years living with type 2 diabetes. When empagliflozin 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%.  

European Commission Approves SPEVIGO® (spesolimab) for Generalized Pustular Psoriasis Flares

Boehringer Ingelheim, with a site in North Brunswick, announced the European Commission (EC) granted a conditional marketing authorization for Boehringer Ingelheim’s spesolimab as first in class treatment for GPP flares in adults. Spesolimab is a novel, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in the pathogenesis of GPP. The EC’s conditional approval of spesolimab is based on results from the pivotal EFFISAYIL® Phase II clinical trial. In the 12-week trial, patients experiencing a GPP flare were treated with spesolimab or placebo. Most patients at the outset of the trial had a moderate or severe pustulation of the skin. 

Boehringer Ingelheim and Click Therapeutics Expand Their Existing Collaboration to Develop Prescription Digital Therapeutics for Schizophrenia

Boehringer Ingelheim, with a site in North Brunswick, and Click Therapeutics announced the launch of an expanded collaboration for the development and commercialization of a second prescription-based digital therapeutic (PDT). The companies will collaborate to develop and commercialize a novel mobile application, which combines multiple clinically validated therapeutic interventions for use alone and in combination with pharmaceutical therapy to help people with schizophrenia achieve positive clinical outcomes. The partnership aims to provide additional treatment options to those living with schizophrenia, where there remains a significant unmet need due to lack of access to psychosocial intervention therapies.

Photocure: Partner Asieris Announces First Patient Treated in Hexvix Real-World Evidence Study in Hainan, China

Photocure, with a site in Princeton, announced that its partner Asieris Pharmaceuticals has communicated on the first patient treatment which took place in a Hexvix® real-world evidence clinical study at Hainan General Hospital Lecheng Branch. Real-world evidence (RWE) plays an increasingly important role in clinical development and regulatory application. The present RWE study will provide complementary data in addition to the Hexvix Phase III randomized controlled study for which patient enrollment also began last month in China. In January 2021, Asieris entered into a license agreement with Photocure ASA to obtain the exclusive registration and commercialization rights of Hexvix in mainland China and Taiwan.

Bladder Cancer: New Meta-Analysis

Photocure, with a site in Princeton, announced that a new meta-analysis was published earlier this month in “Photodiagnosis and Photodynamic Therapy”. The Analysis entitled “Comparison of hexaminolevulinate (HAL)-guided versus white light transurethral resection for NMIBC: A systematic review and meta-analysis of randomized controlled trial” evaluated recurrence, recurrence-free survival and progression. Several Meta-analyses prior to the present one included studies and trials on HAL and 5-ALA. (See Cochrane Analysis from December 2021. The new meta-analysis features HAL studies only, includes 2,775 patients and looks at both short and long-term outcomes. The analysis shows favorable outcomes on recurrence, recurrence free survival and progression for blue light (HAL-guided) cystoscopy compared to white light cystoscopy.

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

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%). 

Datopotamab Deruxtecan Showed Encouraging and Durable Efficacy in Patients With Heavily Pretreated HR Positive, HER2 Low or Negative Metastatic Breast Cancer

Basking Ridge-based Daiichi Sankyo announced initial results from the TROPION-PanTumor01 Phase 1 trial of datopotamab deruxtecan (Dato-DXd) showed encouraging and durable efficacy in patients with heavily pretreated hormone receptor (HR) positive, HER2 low (immunohistochemistry [IHC] 1+ or IHC 2+/in-situ hybridization [ISH]-) or HER2 negative (IHC 0) unresectable or metastatic breast cancer. Safety data were consistent with previous trials of datopotamab deruxtecan. Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo and AstraZeneca. In this cohort of TROPION-PanTumor01 (n=41) where patients previously received a median of five lines of treatment for metastatic disease, datopotamab deruxtecan demonstrated an objective response rate (ORR) of 27% as assessed by blinded independent central review (BICR). 

Datopotamab Deruxtecan Showed Promising Responses as Monotherapy and in Combination With Durvalumab in Patients With Metastatic Triple Negative Breast Cancer in Two Early Trials

Basking Ridge-based Daiichi Sankyo announced updated results from the TROPION-PanTumor01 Phase 1 trial showed datopotamab deruxtecan (Dato-DXd) continued to demonstrate encouraging responses in patients with heavily pretreated metastatic triple negative breast cancer (TNBC) and disease progression following standard treatment. Datopotamab deruxtecan is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) being jointly developed by Daiichi Sankyo and AstraZeneca. Approximately 15% of breast cancers are considered triple negative and are associated with higher disease recurrence and worse prognosis compared to other breast cancer subtypes. It is estimated that only 12% of patients with metastatic TNBC survive five years after diagnosis and median overall survival is between 12 to 18 months.

ENHERTU ® Supplemental New Drug Application Submitted in Japan for Patients With HER2 Mutant Metastatic Non-Small Cell Lung Cancer

Basking Ridge-based Daiichi Sankyo announced that it has submitted a

supplemental New Drug Application (sNDA) to Japan’s Ministry of Health, Labor and Welfare (MHLW) for ENHERTU® (trastuzumab deruxtecan) for the treatment of adult patients with previously treated HER2 mutant unresectable advanced or recurrent non-small cell lung cancer (NSCLC). Prognosis is particularly poor for patients with metastatic NSCLC as only approximately 8% will live beyond five years after diagnosis. In Japan, only 18.2% of patients with metastatic NSCLC will live beyond three years after diagnosis. There are currently no HER2 directed therapies specifically for the treatment of HER2 mutant NSCLC approved in Japan, which occurs in approximately 2% to 4% of patients with NSCLC and non-squamous histology.

Lexicon Announces Topline Results from Phase 2 Proof-of-Concept Study of LX9211 in Postherpetic Neuralgia

Lexicon Pharmaceuticals, Inc., with a site in Basking Ridge, announced topline results of RELIEF-PHN-1, its Phase 2 proof-of-concept study of LX9211 in postherpetic neuralgia. LX9211 achieved a reduction in average daily pain score (ADPS) of 2.42 from baseline at week 6 compared to a reduction of 1.62 in the placebo arm, with a placebo adjusted difference of 0.80 (p=0.12). Although these results did not reach statistical significance on the primary endpoint of the study, overall study results demonstrated clear evidence of effect. Separation of LX9211 from placebo on ADPS was seen at week 1 and maintained consistently thereafter, with an average placebo adjusted reduction over the 6 Week dosing period of 0.80 (p=0.03).

Yaral Pharma Launches in Parsippany

Yaral Pharma Inc., the U.S. generics subsidiary of IBSA, a long-standing, multinational pharmaceutical company, launched in Parsippany with the mission of providing access to high quality, affordable, authorized generics (AG) and complex generic medicines to enhance healthcare outcomes. Led by a team of industry veterans, Yaral Pharma will begin commercializing the first product from IBSA’s corporate pipeline of authorized generics in January 2023. Yaral Pharma’s current portfolio includes products across a range of therapeutic areas, including pain and endocrinology. It plans to rapidly expand its product line and therapeutic areas of focus through outside opportunities and external partnerships. IBSA is known for its manufacturing expertise and track record of product quality and supply continuity. 

Calliditas Therapeutics Announces License Agreement With Viatris to Register and Commercialize Specialty Therapy for IgA Nephropathy in Japan

Calliditas Therapeutics, with a site in Hoboken, announced that it has entered into an exclusive license agreement with Viatris Pharmaceuticals Japan Inc. to register and commercialize Nefecon, a specialty drug recently approved in Europe and the U.S. for the treatment of the chronic autoimmune kidney disease Immunoglobulin A Nephropathy (IgAN) in Japan. Under the terms of the agreement, Calliditas is entitled to receive an initial upfront payment of US$20M upon signing and up to an additional US$80M in pre-defined development and commercialization milestones. Viatris will also pay mid-teens percentage royalties on net sales. IgAN, also known as Berger´s disease, is a rare and serious progressive autoimmune disease in which up to 50% of patients end up at risk of developing end stage renal disease and thus requiring dialysis or a kidney transplant.

Notice Regarding the Signing of an Additional Purchasing Contract With the Ministry of Health, Labor and Welfare for Domestic Supply of Xocova® (Ensitrelvir Fumaric Acid) Tablets 125mg, a Novel Anti-SARS-CoV-2 Drug for COVID-19

Shionogi & Co., Ltd., with a site in Florham Park, announced that Shionogi has entered into an additional purchasing contract of Xocova® (Generic name: ensitrelvir fumaric acid), recently obtained emergency regulatory approval in Japan for the indication of SARS-CoV-2 infection, with the Ministry of Health, Labour and Welfare (MHLW). This contract is for the Japanese government to purchase additional Xocova® tablets for 1 million courses. Xocova® is an oral antiviral agent administered once daily for five days that suppresses the replication of SARS-CoV-2 by selectively inhibiting the viral 3CL protease. Shionogi plans to deliver this additional quantity by the end of the year so that it can contribute a steady supply of this new option for the treatment of SARS-CoV-2 infected patients. 

BD Releases 2022 Global Inclusion, Diversity & Equity Annual Report

Franklin Lakes-based BD released its second annual Global Inclusion, Diversity and Equity Report for 2022 — “We Are the Makers of Possible.” The report is an example of how BD is advancing its environmental, social & governance strategy — “Together We Advance.” It also showcases the power of diversity of thought through its 77,000 global associates. The report details the progress the company is making towards its 2030+ ESG goals for promoting a healthy workforce and communities. Most specifically, highlighting the improvements made to increase diverse representation by 1% year-over-year at the management and executive levels. The report also features spotlights of its global associates at various levels of the organization and Associate Resource Groups. The company continues to exceed health care industry benchmarks in gender and ethnic diversity representation.

Jackie Veivia-Panter Joins BioCentriq as Chief Quality and Compliance Officer

BioNJ Member BioCentriq is thrilled to announce Jackie Veivia-Panter as 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, Ms. Veivia-Panter has managed site quality and R&D 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,” said Ms. Veivia-Panter.

Organon Makes Weaver’s Interim General Counsel and Corporate Secretary Position Official

Kirke Weaver, who in July 2022 was appointed General Counsel and Corporate Secretary, for Organon, will officially be named as such effective Jan. 1, 2023. Mr. Weaver brings nearly 20 years of pharmaceutical industry experience to the role. Prior to serving as Interim General Counsel and Corporate Secretary, he served as Senior Vice President, Commercial, Regulatory, Securities, Employment and Deputy Corporate Secretary at Organon. As a senior member of the legal leadership team, he was critical to launching Organon as a standalone company in June 2021. Prior to joining Organon, Mr. Weaver was a valuable member of the legal team at Merck & Co., most recently serving as vice president, Office of General Counsel. 

Major Milestones for NJ Innovation Evergreen Fund

The New Jersey Economic Development Authority (NJEDA) approved eight corporations to purchase $50 million in tax credits to fuel the New Jersey Innovation Evergreen Fund (NJIEF). The Authority also announced that it is accepting applications from investment firms to be considered Qualified Venture Firms, meaning they would be certified for participation in the program. This will enable them to access both additional capital to invest in qualifying high-growth New Jersey businesses and additional strategic resources available in partnership with the NJIEF tax credit purchasers. This marks a milestone in the creation of the NJIEF, a groundbreaking tool to increase access to strategic resources and venture capital in New Jersey. Under the NJIEF, the state will become an equity investor in startups deploying up to $600 million into companies alongside professional venture capital groups. The capital raised from the sale of these tax credits are expected to result in the funding of initial investments into six to ten high-growth businesses in New Jersey in 2023.

EDA Begins Prequalification Process for $20M Manufacturing Program This Week

The New Jersey Economic Development Authority is accepting prequalification applications for the $20 million New Jersey Manufacturing Voucher Program. The New Jersey MVP was created to provide grants to support the purchase of equipment to help manufacturers improve their operations. The MVP will offer grants valued at 30-50% of the cost of eligible equipment, including installation, up to a maximum award amount of $250,000. Prequalification applications will be reviewed on a rolling basis — not first-come, first-served. Interested manufacturers must prequalify to access the full application that will be made available at a later date. Applicants must complete the full application to be considered for grant funding. The prequalification application will remain open until 11:59 p.m. Dec. 22. https://www.njeda.com/njmvp/

NJEDA to Receive $255M in Federal Funds to Support New Jersey’s Small Business Community

The New Jersey Economic Development Authority (NJEDA) announced that the State of New Jersey is poised to receive more than $255 million in funding through the federal State Small Business Credit Initiative (SSBCI). Following the U.S. Department of Treasury’s approval yesterday of New Jersey’s SSBCI application, the NJEDA will utilize this funding to develop six programs that will provide financial and technical assistance to small and micro businesses, and early stage, innovation-focused companies located in New Jersey. Administered by the U.S. Department of the Treasury, SSBCI is a federal program designed to catalyze lending and investment of private capital into small businesses, particularly those that would not otherwise have access to funding.

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 start-ups. 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.

Merck Digital Sciences Studio Announces Launch of 1st Cohort

Merck Digital Sciences Studio, a collaboration between Kenilworth-based BioNJ Member Merck & Co, Microsoft for Start-ups and New Jersey Innovation Institute, has officially launched. The collaborative cohort will focus on important and disruptive biotechnical approaches in artificial intelligence and machine learning, with expertise and tools from clinical scientists to continue Merck’s mission to strengthen the life sciences ecosystem. The 10-month start-up accelerator program, with direct investments from Merck Global Health Innovation Fund, Northpond Ventures and McKesson Ventures, plans to empower members and enable the generation of innovative digital technologies for drug discovery and development through hands-on mentorship, connection opportunities with industry leading partners and training in an active entrepreneurial setting.

Boscamp Named Dean of the Hackensack Meridian School of Medicine

Hackensack Meridian School of Medicine announced that Jeffrey Boscamp, M.D., has been named Dean. He will also hold the distinguished Robert C. and Laura C. Garrett Endowed Chair for the School of Medicine Dean. Dr. Boscamp had been Interim Dean of the school since the sudden passing of his predecessor, Dr. Bonita Stanton, last January. Dr. Boscamp had previously been Vice Dean of the school. “Dr. Boscamp has performed terrifically in the interim role, and he’s proven to be a visionary educator,” said Robert C. Garrett, FACHE, Chief Executive Officer, Hackensack Meridian Health. “We are excited to see how he leads the school to an ever-brighter future.”

Rutgers’ Gennaro Named NAI Fellow for Work on Infectious Diseases, Including TB and COVID

Maria Laura Gennaro, a Professor of Medicine at Rutgers New Jersey Medical School and a Professor of Epidemiology at Rutgers School of Public Health in New Brunswick, has been named a National Academy of Inventors fellow, which is the highest professional distinction accorded solely to academic inventors. Ms. Gennaro has focused her research on infectious diseases, particularly tuberculosis and COVID-19. She and her team have pursued both fundamental and translational studies on the stress responses of host and pathogen in TB, on new methods and platforms for TB diagnostics and on antibody responses to SARS-CoV-2 infection. Ms. Gennaro said she was grateful for the recognition and thankful for the opportunities she has.

Rutgers Med School Researchers Report Breakthrough in Ultrasound Imaging Technique for Early Detection of Heart Disease

Researchers Rutgers Robert Wood Johnson Medical School and Robert Wood Johnson University Hospital have reported a new breakthrough in ultrasonic imaging methods that can detect microscopic changes in heart structure and function. The procedure, which uses miniaturized ultrasound devices that can be carried in the pocket, may be useful for screening early heart disease. Dr. Partho Sengupta and Dr. Naveena Yanamala revealed how they used artificial intelligence modeling techniques to compile and analyze pixel-based patterns in echocardiogram images of humans to develop expert-level interpretation of cardiac conditions that lead to heart failure. They then used a mouse model of heart failure and discovered that these patterns arise from microscopic changes in heart muscle geometry.

Junior Achievement of N.J. Receives $25,000 STEM Grant from Bayer Fund

Junior Achievement of New Jersey said it received a grant for $25,000 from Bayer Fund, a philanthropic arm of Bayer in the U.S. The funding will be used to inspire New Jersey’s students for careers in science, technology, engineering and mathematics through JA Crack the Code hands-on learning experiences, comprised of a series of free coding and STEM workshops. Given that careers in STEM fields are in high demand, yet diversity remains a challenge, JANJ has a unique opportunity to make STEM learning and awareness into fun, interactive educational experiences through JA Crack the Code. This creative space for 8-to-15-year-old students, encourages the discovery of careers in STEM, while learning from older peers and professionals with similar backgrounds. High school students serve as role models, together with corporate volunteers, and provide instruction and mentorship for hundreds of middle school-age beginners and experienced coders alike.

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 -- providing valuable opportunities to engage with key players from the life sciences ecosystem. You will discover groundbreaking therapies in innovative biotech presentations delivered during the Biotech Showcase and have access to partneringONE starting six weeks prior to the conference and three months after the conference, with submission of up to 150 meeting requests per company. BioNJ Members receive a $200 discount off their registration fees. Contact [email protected] for the discount code. 

State-of-the-Art Equipment from Rubius’ Cambridge, MA Lab

January 9, 2023

BioNJ Members, don’t miss an auction opportunity of late model lab instrumentation. Our BIO Business Solutions Partner, ALT is hosting an online auction of State-of-the-Art Equipment from Rubius Therapeutics Cambridge, MA Lab! Bids open January 9. Register now: https://lnkd.in/gr9v5T_Y