Welcome to the latest edition of the BioLines Weekender...
On Monday, BioNJ hosted a Patient Advocate/Legislator Roundtable discussion with policymakers focused on opportunities for New Jersey to support increased Patient access to new and emerging medications — as well as opportunities to support the early-stage innovation that produces novel therapeutics. Held at BioNJ Member PsychoGenics' facility in Paramus, which is led by BioNJ Chair Dr. Emer Leahy who kicked off the event, representatives from a variety of mental health and rare disease Patient advocacy organizations joined the discussion, including several members of the recently-appointed New Jersey Rare Disease Advisory Council.
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Photo from left to right: Dr. Ian McLaughlin, VP, Government Affairs, BioNJ; Patricia Geurds, Rare Action Network, NORD; Dr. Debra Wentz, President & CEO, NJAMHAA; Dr. Howard Fillit, CEO, Alzheimer’s Drug Discovery Foundation; Julie Raskin, CEO, Congenital Hyperinsulinism International; Dr. Emer Leahy, President & CEO, PsychoGenics & BioNJ Chair; Asw. Shama Haider (LD37); Asw. Lisa Swain (LD38); Asm. Chris Tully (LD38); Debbie Hart, President & CEO, BioNJ; Allan Malievsky, Sr. Director, External Affairs, atai Life Sciences; and Dr. Martina Flammer, Chair, NJ Rare Disease Advisory Council & Chief Medical Officer, Insmed. Missing from the photo are Assemblyman Chris DePhillips (D-40) and Dr. Kenneth Sonnenfeld, Board Member, Brain and Behavior Research Foundation who also participated. | |
Then, on Tuesday, we celebrated the highly anticipated ribbon-cutting for BioNJ Member BeiGene's new flagship U.S. biologics manufacturing and clinical research and development facility at the Princeton West Innovation Campus in Hopewell. This important occasion was marked by the presence of Governor Murphy highlighting the potential impact of this facility on advancing scientific innovation and showcasing New Jersey’s leadership as a leading life sciences hub. We extend our sincere congratulations and heartfelt gratitude to BeiGene for its commitment to the State and its dedication to Patients worldwide.
Meanwhile, the Pharmaceutical Research and Manufacturers of America released an important new campaign focused on highlighting the ways that pharmacy benefit managers (PBMs) play in driving up the costs that Patients face in accessing many of their medications. For a summary of the campaign, click here. For a video from the campaign, click here.
Please visit www.BioNJ.org for more information on PBMs and other important legislative efforts aimed at ensuring future innovation and providing Patients with access to the medicines they need.
Because Patients Can't Wait®,
Debbie Hart
President and CEO
BioNJ
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Putting Patients First:
The Value of Medical Innovation -
Recent Stories in the News
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Senator Vin Gopal Announces Joint Statement from District 11 Legislators, Senate President Scutari, Speaker Coughlin, Senate Health Committee Chair Vitale, Assembly Health Committee Chair Conaway and longtime Senate PBM Transparency Advocates Senators Greenstein, Mukherji and Singleton
In the light of recent findings from a New York Times investigation on Pharmacy Benefit Managers (PBMs), a group of New Jersey lawmakers and independent pharmacists are coming together to address the impact of PBMs and the crisis of rising drug costs on New Jersey. Senator Vin Gopal, Assemblywoman Margie Donlon, M.D., and Assemblywoman Luanne Peterpaul join Senate President Scutari, Assembly Speaker Craig Coughlin, Senate Health Chairman Joseph Vitale, Senators Raj Mukherji, Troy Singleton, Linda Greenstein, and Assembly Health Chairman Herb Conaway, M.D., in affirming their commitment to affordable prescription costs and regulating PBMs.
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FDA Rare Disease Innovation Hub to Enhance and Advance Outcomes for Patients
An estimated 10,000+ rare diseases affect more than 30 million people – approximately one out of every 10 people – in the U.S., and about half of these people are children. Many rare conditions are life threatening, and most do not have approved treatments. Fundamental to the mission of the U.S. Food and Drug Administration is to engage patients and caregivers – to understand their unique perspectives and experiences and keep these front of mind as we review medical products for rare disease patients. Recent rapid advances in the identification of promising drug targets and development of gene therapies offer momentum and potential to meet the needs of patients with rare diseases.
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U.S. Cancer Survivor Population Projected to Reach 26 Million by 2040
The prevalence of cancer survivors in the United States has risen in recent years and is projected to increase more in years to come, according to research published in the Journal of the National Cancer Institute. To estimate the current and projected prevalence of cancer survivors in the US, researchers used the Prevalence Incidence Approach Model and data from the Surveillance, Epidemiology, and End Results (SEER) Program, the National Center for Health Statistics, and the US Census Bureau. The researchers estimated that, as of January 1, 2022, the prevalence of cancer survivors in the US was 18.1 million. The team noted that this is a nearly 4-fold increase in cancer survivors since the mid-1970s. Of the 18.1 million cancer survivors, 70% had survived at least 5 years from diagnosis, 48% had survived at least 10 years, 19% had survived at least 20 years and 11% had survived at least 25 years.
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BeiGene Ribbon-Cutting: Big Get for Life Science Industry — and Bigger Vote of Confidence for State
As featured in ROI-NJ
BeiGene co-founder and CEO John Oyler cut the ribbon on the company’s $800 million, 42-acre state-of-the-art clinical research and manufacturing facility at the Princeton West Innovation Center in Hopewell on Tuesday. Oyler shared the usual reasons that the next-generation oncology company is eager to dramatically increase its presence in the State, such as access to top talent, closeness to other global life sciences companies and opportunities for richly diverse clinical trials. In addition, he offered one reason that may surprise some: New Jersey is a great place to do business.
Debbie Hart, President and CEO of BioNJ shared, “This momentous event marks the official addition of a vital new member of the ecosystem. The collaboration, innovation and jobs that BeiGene is bringing to New Jersey will contribute to the community and provide hope and promise for patients.”
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BeiGene Cuts Ribbon on $800M Hopewell Hub
As featured in NJBIZ
Tuesday marked another major notch in the great Garden State’s reputation as a life sciences and innovation leader. BeiGene opened its new $800 million flagship U.S. facility in Hopewell July 23. The manufacturing and clinical R&D center is located at the Princeton West Innovation Campus. It provides the global oncology company 400,000 square feet of dedicated clinical- and commercial-stage biologics manufacturing space. BeiGene also has the ability to add 700,000 square feet over time. The company notes that the 42-acre facility offers flexibility to scale production of its innovative medicines today – as well as in the future – to meet the needs of cancer patients.
“Today, we celebrated the highly anticipated ribbon cutting for BioNJ Member BeiGene’s new flagship U.S. biologics manufacturing and clinical research and development facility,” said BioNJ President and CEO Debbie Hart, who attended the ceremony. “This important occasion was marked by the presence of Gov. Murphy, highlighting the potential impact of this facility on advancing scientific innovation and showcasing New Jersey’s leadership as a leading life sciences hub. We extend our sincere congratulations and heartfelt gratitude to BeiGene for its commitment to the state and its dedication to patients worldwide."
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Phathom Pharmaceuticals Announces FDA Approval of VOQUEZNA® (vonoprazan) Tablets for the Relief of Heartburn Associated With Non-Erosive GERD in Adults
BioNJ Member Phathom Pharmaceuticals, Inc. headquartered in Florham Park, announced the U.S. Food and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan) 10 mg tablets for the relief of heartburn associated with Non-Erosive Gastroesophageal Reflux Disease (Non-Erosive GERD) in adults. Non-Erosive GERD represents a substantial segment of the U.S. GERD population, affecting millions of individuals suffering from frequent heartburn. This is the third FDA approval for VOQUEZNA, which is also approved to treat all severities of Erosive Esophagitis (EE), also referred to as Erosive GERD, and in combination with antibiotics for the eradication of Helicobacter pylori (H. pylori) infection. Non-Erosive GERD is the largest category of GERD and is characterized by reflux-related symptoms in the absence of esophageal mucosal erosions. An estimated 45 million U.S. adults living with Non-Erosive GERD, and approximately 15 million are treated with a prescription medicine annually.
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Celldex Therapeutics Initiates Global Phase 3 Program for Barzolvolimab in Patients With Chronic Spontaneous Urticaria
Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced the initiation of its global Phase 3 program, consisting of two Phase 3 trials (EMBARQ-CSU1 and EMBARQ-CSU2) designed to establish the efficacy and safety of barzolvolimab in adult patients with CSU who remain symptomatic despite H1 antihistamine treatment. The studies will also include patients who remain symptomatic after treatment with biologics. For patients with CSU, the activation of mast cells in the skin results in episodes of itchy hives, swelling and inflammation of the skin that can severely impact their daily lives for years or even decades. Treatment options are limited, particularly for patients not adequately controlled by omalizumab. Barzolvolimab, a novel monoclonal antibody, works upstream of other treatment approaches for CSU, targeting the root driver of the disease—mast cells—by blocking the receptor tyrosine kinase KIT, which is required for mast cell function and survival.
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First Half of 2024: Boehringer Ingelheim Progresses Pipeline at Pace and Reaches Major Milestones
BioNJ Member Boehringer Ingelheim, with a site in North Brunswick, reported significant progress in its pipeline across key therapeutic areas as it reached major milestones in the first half of the year. “We are stepping up our investments in R&D beyond our plans announced in April,” said Hubertus von Baumbach, Chairman of the Board of Managing Directors. “The data read-outs that we received in MASH and will receive for oncology, mental health, and pulmonary fibrosis, give us reason to accelerate our launch preparedness for these late-stage assets. We are pleased to see our pipeline developing at such pace.” Boehringer advanced its pipeline across clinical phases with five new phase I, II, or III initiations in cardiometabolic diseases, mental health, and oncology, and achieved two additional fast track designations for programs in inflammation.
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Chugai Transfers and Future Marketing of Physiological Intestinal Function Improving and Hyperammonemia Drug "MONILAC ® Syrup 65%" in Japan
BioNJ Member Chugai Pharmaceutical Co., Ltd., with a site in Berkeley Heights, and Maruishi Pharmaceutical Co., Ltd. announced that Chugai will transfer to Maruishi, the business in Japan concerning the physiological intestinal function improving and hyperammonemia drug "MONILAC® Syrup 65%" (hereafter, MONILAC) for which Chugai holds the manufacturing and marketing authorization in Japan. Chugai has been marketing MONILAC and providing information in Japan since 1975. In consideration of the maturity of its product lifecycle, Chugai has been considering its future domestic marketing. As a result of discussions between Chugai and Maruishi, it was agreed that Chugai would transfer all assets relating to MONILAC in Japan, including manufacturing and marketing authorization and intellectual property rights such as trademarks, to Maruishi, and Maruishi would thereafter be responsible for sales of MONILAC.
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Teva Announces First Phase 3 Evidence of Efficacy and Safety of an Anti-CGRP Monoclonal Antibody in Children in AJOVY® (fremanezumab) SPACE Trial for Prevention of Episodic Migraine
Parsippany-based BioNJ Member Teva Pharmaceutical announced positive topline results from its Phase 3 SPACE study evaluating the efficacy of AJOVY (fremanezumab) for the prevention of episodic migraine in children and adolescent patients aged 6-17 years. The trial met its primary end point with AJOVY (fremanezumab) achieving a statistically significant superior efficacy over 12 weeks of treatment compared to placebo. Safety data was consistent with that observed in adult populations, with no emergent safety signals. Full data from the SPACE study will be presented at a medical meeting later this year. The SPACE data are the first Phase 3 trial evidence of safety and efficacy of an anti-CGRP monoclonal antibody for the treatment of migraine in a pediatric population. The results have particular significance given the lack of preventive migraine treatments approved for use in children and adolescent populations.
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Merck Announces Topline Results from Phase 2b/3 Trial of Clesrovimab (MK-1654), an Investigational Respiratory Syncytial Virus Preventative Monoclonal Antibody for Infants
Rahway-based BioNJ Member Merck & Co. announced positive topline results from its Phase 2b/3 clinical trial (MK-1654-004) evaluating clesrovimab (MK-1654), the company’s investigational prophylactic monoclonal antibody designed to protect infants from respiratory syncytial virus (RSV) disease. In the trial, clesrovimab met its primary safety and efficacy endpoints, including reducing medically attended lower respiratory infections (MALRI) caused by RSV through Day 150. Detailed findings of the study will be presented at an upcoming scientific congress and Merck plans to file these data with global regulatory authorities. “RSV is highly contagious and can cause inflammation in the airways of infants leading to difficulty breathing. As a widespread illness globally, RSV is the leading cause of hospitalization for healthy infants,” said Dr. Paula Annunziato, Senior Vice President, Infectious Diseases and Vaccines, Global Clinical Development, Merck Research Laboratories.
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Merck Completes Acquisition of EyeBio
Rahway-based BioNJ Member Merck & Co. announced the completion of the acquisition of Eyebiotech Limited. EyeBio is now a wholly-owned subsidiary of Merck. EyeBio’s lead candidate, Restoret™ (EYE103), is an investigational, potentially first-in-class tetravalent, tri-specific antibody that acts as an agonist of the Wingless-related integration site (Wnt) signaling pathway. Based on positive results from the open-label Phase 1b/2a AMARONE study in patients with diabetic macular edema (DME) and neovascular age-related macular degeneration (NVAMD), Restoret is scheduled to advance into a pivotal Phase 2b/3 trial to evaluate its potential for the treatment of patients with DME in the second half of 2024. Additional pipeline candidates include clinical and preclinical assets being developed for the prevention and treatment of vision loss associated with retinal vascular leakage, a known risk factor for retinal diseases.
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Bristol Myers Squibb Receives European Medicines Agency Validation of Application for Opdivo (nivolumab) Plus Yervoy (ipilimumab) for First-Line Treatment of Unresectable or Advanced Hepatocellular Carcinoma
Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Medicines Agency (EMA) validated its Type II variation application for Opdivo® (nivolumab) plus Yervoy® (ipilimumab) as a potential first-line treatment option for adult patients with unresectable or advanced hepatocellular carcinoma (HCC) who have not received prior systemic therapy. The application was based on results from the Phase 3 CheckMate -9DW trial and validation of the application confirms the submission is complete and begins the EMA’s centralized procedure review. In the Phase 3 CheckMate -9DW trial, Opdivo plus Yervoy demonstrated statistically significant and clinically meaningful improvement in overall survival (OS) compared to investigator’s choice of lenvatinib or sorafenib, which showed the clinical benefit of the combination treatment option when provided in the first-line setting.
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NEJM Publishes ALTUVIIIO XTEND-Kids Phase 3 Data Supporting its Potential to Transform the Treatment Landscape for Children With Severe Hemophilia A
Bridgewater-based BioNJ Member Sanofi announced full results from the XTEND-Kids Phase 3 study published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety and pharmacokinetic profile of ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein]. ALTUVIIIO (efanesoctocog alfa), a first-in-class, high-sustained factor VIII replacement therapy, is approved for adults and children with hemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes as well as for perioperative management (surgery). The pivotal XTEND-Kids study published in NEJM shows ALTUVIIIO met primary and secondary endpoints, which included occurrence of factor VIII inhibitors and annualized bleed rates (ABRs). The results show no inhibitor development to factor VIII was detected with ALTUVIIIO (0% [95% confidence interval (CI)] 0–5]).
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Blenrep (belantamab mafodotin) Combinations in Multiple Myeloma Application Accepted for Review by the European Medicines Agency
Warren-based BioNJ Member GSK announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for Blenrep (belantamab mafodotin) in combination with bortezomib plus dexamethasone (BorDex) or pomalidomide plus dexamethasone (PomDex) as a treatment for relapsed or refractory multiple myeloma. The EMA’s Committee for Medicinal Products for Human Use (CHMP) will begin the formal review process to make a recommendation to the European Commission regarding this potential authorization. Hesham Abdullah, Senior Vice President, Global Head Oncology, R&D, GSK, said: “Today’s milestone reinforces the potential for Blenrep to redefine outcomes for patients with multiple myeloma at or after first relapse. We are working to bring Blenrep to patients as quickly as possible given the high unmet need and the clinically robust effects of the Blenrep combinations in the DREAMM-7 and DREAMM-8 phase III head-to-head trials.”
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Johnson & Johnson Seeks U.S. FDA Approval of SPRAVATO® (esketamine) as the First and Only Monotherapy for Adults With Treatment-Resistant Depression
Titusville-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking approval of SPRAVATO® (esketamine) CIII nasal spray as a monotherapy for adults living with treatment-resistant depression (TRD). Nearly 30 percent of the estimated 280 million people worldwide living with major depressive disorder (MDD) have TRD, which occurs when there is an inadequate response to two or more oral antidepressants during the same depressive episode. SPRAVATO® is approved by the FDA, in combination with an oral antidepressant, to treat adults with TRD and depressive symptoms in adults with MDD with acute suicidal ideation or behavior. To date, SPRAVATO® has been approved in 77 countries and administered to more than 100,000 people worldwide.
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“LEQEMBI®” (Lecanemab) Approved for the Treatment of Alzheimer’s Disease in Israel
Nutley-based BioNJ Member Eisai and Princeton-based BioNJ Member Biogen Inc. announced that humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody “LEQEMBI®” (generic name: lecanemab) has been approved in Israel as a treatment of Alzheimer’s disease (AD). Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment (MCI) or mild dementia stage of disease (collectively referred to as early AD), the population in which treatment was initiated in clinical trials. LEQEMBI selectively binds to soluble Aβ aggregates (protofibrils), as well as insoluble Aβ aggregates (fibrils) which are a major component of Aβ plaques, thereby reducing both Aβ protofibrils and Aβ plaques in the brain. LEQEMBI is the first approved treatment shown to reduce the rate of disease progression and to slow cognitive and functional decline through this mechanism.
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Eisai Enters into License Agreement for Fosravuconazole in Asia/Oceania With Sato Pharm
Nutley-based BioNJ Member Eisai announced that the company has entered into a license agreement regarding the development and commercialization rights for the antifungal agent fosravuconazole in the Asia/Oceania region with Sato Pharmaceutical Co., Ltd. Under this agreement, Eisai exclusively licenses the intellectual property rights of fosravuconazole in the given countries/regions to Sato Pharma. Ravuconazole, the active ingredient of fosravuconazole, is an antifungal agent discovered and developed by Eisai. Fosravuconazole is a prodrug of ravuconazole, which improves its solubility and bioavailability. In Japan, Seren Pharma and Sato Pharma have developed fosravuconazole based on the exclusive rights to develop, commercialize and sublicense granted by Eisai. The treatment has been marketed by Sato Pharma as the oral antifungal agent Nailin® for the indication of “onychomycosis” since July 2018, which Eisai is co-promoting.
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AbbVie Submits Regulatory Applications to FDA and EMA for Upadacitinib (RINVOQ®) in Giant Cell Arteritis
BioNJ Member AbbVie, with a site in Madison, announced that it has submitted applications for a new indication to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for upadacitinib (RINVOQ®; 15 mg, once daily) for the treatment of adult patients with giant cell arteritis (GCA). GCA is an autoimmune disease of medium and large arteries and can cause headache, jaw pain, and changes in vision, including sudden and permanent loss of vision. "Currently, there are few approved treatment options for patients with GCA. Most patients are managed with glucocorticoids, and many are unable to discontinue them without the recurrence of GCA symptoms," stated Roopal Thakkar, M.D., Executive Vice President, Research and Development, and Chief Scientific Officer, AbbVie. "We recognize the importance of maintaining remission and limiting the use of glucocorticoids in GCA."
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Lexicon Pharmaceuticals Receives December 20, 2024 PDUFA Goal Date for Sotagliflozin Type 1 Diabetes NDA Resubmission
BioNJ Member Lexicon Pharmaceuticals, Inc., with a site in Bridgewater, announced that the U.S. Food and Drug Administration (FDA) has acknowledged the resubmission of its New Drug Application (NDA) for Zynquista™ (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease (CKD). The FDA indicated that it considers the resubmission to be a complete response to the FDA’s 2019 action letter regarding the sotagliflozin NDA for type 1 diabetes, providing a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2024. “Lexicon has continued confidence in the favorable benefit/risk profile of sotagliflozin for adults with type 1 diabetes and CKD. With a PDUFA date now in hand, we can begin the next phase of launch preparation for Zynquista,” said Dr. Mike Exton, Chief Executive Officer and Director.
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Sandoz Launches Biosimilar Pyzchiva® (ustekinumab) Across Europe, to Treat Chronic Inflammatory Diseases
Princeton-based BioNJ Member Sandoz announces the launch of Pyzchiva®* (ustekinumab) across Europe, starting today. Pyzchiva®, developed and registered by Samsung Bioepis, is the first ustekinumab biosimilar to launch in Europe with all reference medicine strengths, including the 130mg vial initiation dose for Crohn’s disease. The launch strengthens our well-established immunology portfolio in Europe and leverages our existing footprint, with five biosimilars now marketed in this therapeutic area. Pyzchiva® is a key biosimilar driver, adding value and contributing to the company’s mid-term growth strategy. Pyzchiva® is approved for treatment of adults with plaque psoriasis, psoriatic arthritis, Crohn’s disease and pediatric plaque psoriasis for patients six years and older weighing over 60 kg.
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European Commission Approves Pfizer’s DURVEQTIX® (fidanacogene elaparvovec), a One-Time Gene Therapy for Adults With Hemophilia B
BioNJ Member Pfizer Inc., with a site in Peapack, announced that the European Commission (EC) has granted conditional marketing authorization for DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for the treatment of severe and moderately severe hemophilia B (congenital factor IX deficiency) in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74. DURVEQTIX is designed to enable people living with hemophilia B to produce factor IX (FIX) themselves via a one-time dose, rather than multiple intravenous FIX infusions weekly or biweekly with the current standard of care. Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others.
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Pfizer Announces Positive Topline Results From Phase 3 Study of Hemophilia A Gene Therapy Candidate
BioNJ Member Pfizer Inc., with a site in Peapack, announced positive topline results from the Phase 3 AFFINE study (NCT04370054) evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A. The AFFINE study achieved its primary objective of non-inferiority, as well as superiority, of total annualized bleeding rate (ABR) from Week 12 through at least 15 months of follow up post-infusion compared with routine Factor VIII (FVIII) replacement prophylaxis treatment. Following a single 3e13 vg/kg dose, giroctocogene fitelparvovec demonstrated a statistically significant reduction in mean total ABR compared to the pre-infusion period (1.24 vs 4.73; one-sided p-value=0.0040). Key secondary endpoints as defined by the trial protocol were met and also demonstrated superiority compared to prophylaxis.
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Pharma Two B Announces Plans to Go Public via Merger With Hepion Pharmaceuticals, Inc. and Concurrent $11.5 Million Private Placement
Edison-based BioNJ Member Hepion Pharmaceuticals, Inc. announced it has entered into a definitive merger agreement with Pharma Two B Ltd., a late-clinical stage private Israeli company that is developing P2B001, an innovative combination product candidate in development for the treatment of Parkinson’s Disease. Under the Merger Agreement, Hepion will merge into and become an indirectly wholly-owned subsidiary of Pharma Two B (the “Merger”). The combined company will continue to operate under the “Pharma Two B” name and Pharma Two B has agreed to file a registration statement on Form F-4 (the “Form F-4”) with the U.S. Securities and Exchange Commission to register the ordinary shares proposed to be issued (or reserved for issuance) to Hepion’s equity-holders in the acquisition, and will also apply to list its ordinary shares on Nasdaq under the ticker symbol “PHTB”.
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Shionogi Provides Updates on Zatolmilast, an Investigational Drug for Fragile X Syndrome, Including Recent Changes to Study Protocol to Increase Access for Participants and Families
Shionogi & Co., Ltd., with a site in Florham Park, announced progress on the development program for zatolmilast (BPN14770), an investigational drug for Fragile X syndrome (FXS), the leading cause of inherited intellectual disabilities, like autism spectrum disorder. Zatolmilast was discovered by Tetra Therapeutics Inc., which was acquired by Shionogi in 2020. Zatolmilast is the first and only selective PDE4D inhibitor being investigated for FXS. There are currently no pharmacological agents approved by the U.S. Food and Drug Administration (FDA) to treat FXS. The FDA recently granted Fast Track designation to zatolmilast, reflecting the significant unmet medical need for FXS. The FDA previously granted zatolmilast Rare Pediatric Disease Designation and Orphan Drug Designation. The European Commission granted Orphan Medicinal Product designation for zatolmilast in FXS earlier this year.
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Biocon Biologics Establishes North American HQ in Bridgewater
Biocon Biologics established its first North American headquarters right here in the great Garden State, selecting Bridgewater as the site. The global biosimilars company announced the location this week. Its work is dedicated to using cutting-edge science to lower costs of biological therapeutics while improving health care outcomes for millions of patients globally. Biocon Biologics boasts eight commercialized biosimilars in the country. It also has one of the largest pipelines in the industry. The company has 20 biosimilar assets across diabetology, oncology, immunology, ophthalmology and other non-communicable diseases. The 9,624-square-foot headquarters is located at 685 Route 202/206 in Bridgewater at the Bedminster 78 Corp Center. Company leaders say the new office will house both commercial and administrative functions for the organization. It will also position it for future growth in North America.
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These are New Jersey’s Best Places to Work for 2024
How do you determine New Jersey’s Best Places to Work? You ask the employees, of course. The 2024 honorees across small (15 – 49 employees), medium (50 – 249 employees) and large (more than 250 employees) employers are based upon the results of a survey conducted by BridgeTower Media’s Best Companies Group. The program confidentially collects data, allowing workers to share feedback about their employers, and for those companies to learn from and act on that knowledge. Congratulations to these BioNJ Members for making the list: Eisai, EisnerAmper, Genmab US and RSM US.
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Numerous N.J. Companies Named Best Places to Work for Those With Disabilities
More than a dozen New Jersey-based companies recorded a 100 score on the Disability Equality Index Best Places to Work list created by Disability:IN. The Disability Equality Index is the world’s most comprehensive benchmarking tool for Fortune 1000 companies to measure disability workplace inclusion inside their organizations and to assess performance across industry sectors. In simplest terms: New Jersey companies are leaders when it comes to cultivating a diverse workplace that includes those with disabilities. Congratulations to these BioNJ Members for making the list: Bristol Myers Squibb, Johnson & Johnson, Merck & Co., Novartis, Sanofi and Withum.
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JOGO Health Announces Collaboration With Mayo Clinic to Study Tremors
Bridgewater-based JOGO Health announced a collaboration with Mayo Clinic to study tremors. Entering a "know-how" agreement, the two companies will combine capabilities in medical analysis and device development to research Parkinson's tremors, functional tremors (FT) and essential tremors (ET). Both teams hope this initiative will lead to advancements in treating these conditions with AI-driven digital therapeutics. The study will include people who have functional tremors that are challenging to treat because they require specialized therapy instead of medication. At present, there are very few established treatment programs, with most requiring patients to take significant time off work or school.
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Rutgers–New Brunswick Awarded ‘Innovation and Economic Prosperity’ Designation
The Association of Public and Land-grant Universities (APLU) has recently designated Rutgers University–New Brunswick as an Innovation and Economic Prosperity University in recognition of its substantial and sustained commitment to economic engagement in the region. The national designation highlights Rutgers’ role as an economic catalyst through transformative innovations in academic initiatives, pioneering research and impactful community outreach programs. This includes business incubators that have launched thousands of startups, technologies addressing agricultural challenges and the university’s involvement in the New Jersey Health and Life Science Exchange (HELIX) in downtown New Brunswick, which is intended to become a leading hub for research, commercialization and education.
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Hackensack University Ranked No. 1 Hospital in State by Prestigious U.S. News Rankings
Hackensack Meridian Hackensack University Medical Center has been named the top-rated hospital in New Jersey for the second consecutive year, according to the prestigious U.S. News & World Report Best Hospital rankings for 2024. This year, HUMC also was ranked among the Top 5 in the New York metro area. The state rankings, however, annually draw the most attention. For the second consecutive year, HUMC edged out Morristown Medical Center in their annual battle for the top spot in New Jersey. The two are the only hospitals to earn the No. 1 ranking in the U.S. News state rankings in the past 11 years. Hackensack has earned the honor six times. Morristown has been so named four times. And once (in 2021), the two shared the top spot.
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NJSBDC Launches Program to Help NJ Innovators Write Strong Grant Proposals
The New Jersey Small Business Development Centers (NJSBDC) Tech Team has launched a new Proposal Writing Assistance program, funded by a $50,000 grant from the New Jersey Commission on Science, Innovation and Technology (CSIT). This program aims to support New Jersey innovators in developing strong proposals for federal Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) grants. Judith Sheft, Executive Director of CSIT, expressed enthusiasm about the collaboration: “The CSIT is thrilled that NJSBDC is expanding their FAST (Federal and State Technology Partnership Program) services to include grant development support for New Jersey entrepreneurs pursuing federal SBIR/STTR grant opportunities. Innovation-based startups face unique hurdles as they work toward commercialization, and partnering with the NJSBDC ensures that New Jersey entrepreneurs have access to additional technical assistance resources to position themselves for success.
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NJEDA Board Approves Investment of Up to $6M into Newark Venture Partners
The New Jersey Economic Development Authority (NJEDA) approved an investment into Newark Venture Partners, III, L.P. (NVP III) of up to $6 million under the NJEDA’s Venture Fund Investment Program. As a requirement of the program, NVP III will work to match every dollar of the NJEDA’s commitment to the Fund with an additional $2 into New Jersey-based companies. Additionally, up to $1 million of the NJEDA’s aggregate commitment will be invested through a fund to exclusively invest in Newark-based start-ups. NVP III is the third fund for Newark Venture Partners (NVP), a Newark-based early-stage venture capital firm. NVP leverages its corporate limited partner relationships, which include top corporate investors such as Audible (an Amazon company), Prudential, RWJBarnabas, Bank of America, and Horizon Blue Cross Blue Shield, to source potential investments, evaluate the commercial viability of opportunities, and add value to portfolio companies.
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Presenting the 2024 NJBIZ Law Power 50
The NJBIZ Power List is compiled by the NJBIZ editorial staff based on our reporting throughout the past year with input from experts in a variety of fields and recommendations from our readers. The staff looks for people who have gained public attention – and perhaps acclaim – for their professional accomplishments and public service. The list identifies individuals who, through their efforts, are helping to make New Jersey a better place to live, work and do business. Congratulations to these BioNJ Members for making the list: Audrey Murphy from Hackensack Meridian Health; Eric Hass from Johnson & Johnson; and Guillermo Artiles, Joseph Boccassini and Scott Kobler from McCarter & English.
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AbbVie Announces Appointment of Roopal Thakkar, M.D. as Executive Vice President, Research & Development and Chief Scientific Officer
BioNJ Member AbbVie, with a site in Madison, announced that Roopal Thakkar, M.D. who currently serves as Senior Vice President, Chief Medical Officer, Global Therapeutics has been appointed to the position of Executive Vice President, Research & Development and Chief Scientific Officer. In this position, Dr. Thakkar will lead the company's global R&D organization of more than 14,000 team members across all phases of discovery and development, including therapeutics and aesthetics. Thakkar is a physician by training with a deep commitment to innovation and patient care," said Rob Michael, Chief Executive Officer, AbbVie. "He has an excellent track record in building new capabilities, forging strategic partnerships and advancing our clinical programs to bring medicines and solutions to patients as quickly as possible.”
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Paladino, Sheft, Lowman, Loudig and Kerninghan to be Honored With Prestigious 'Eddy' Awards
Chris Paladino of New Brunswick Development Corp., Tony Lowman of Rowan University, Judith Sheft of the Commission on Science, Innovation and Technology, Dr. Olivier Loudig of the Hackensack Meridian Health Center for Discovery and Innovation and computer science pioneer Brian Kernighan will head the list of honorees at the 45th annual Edison Patent Awards.
The event, created and run by the Research & Development Council of New Jersey, honors the State’s top inventors and innovators. In addition to the five individual awards, 66 inventors representing 14 patents will be recognized. The event — themed “Jersey Innovates, the World Takes” — will be held Nov. 21 at Bell Works in Holmdel.
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ESG Best Practices: Tools to Learn From | |
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Bristol Myers Squibb Reaches Major Milestone in Environmental Sustainability
Bristol Myers Squibb announced validation for its near-term and net-zero science-based targets from the Science Based Targets initiative (SBTi), as part of a mid-year update on its environmental initiatives, highlighting progress in reducing emissions across its operations and supply chain. SBTi validation is considered the gold-standard for corporate greenhouse gas (GHG) emissions target setting and reduction strategies. BMS is committed to reaching net-zero GHG emissions across its value chain by 2050 from a 2022 baseline year. In addition, BMS has committed to reduce Scope 1 and 2 GHG emissions and Scope 3 GHG emissions (from fuel and energy related activities) by 54.6% by 2033 and engage 75% of its suppliers, by emissions, in their development of science-based targets by 2028.
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Job Fair Hosted by Assemblywoman Tennille McCoy
August 21, 2024 | 10:00AM-3:00PM
If you are interested in attending the Job Fair hosted by Assemblywoman Tennille R. McCoy, please complete this registration form for each person attending. We look forward to seeing you there!
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Bayer's Expert Mondays 2024
Select Mondays in March – December 2024
Calling pre-seed and seed innovators and entrepreneurs in Oncology, Cell & Gene Therapy, Cardiovascular, Immunology, Radiology and Digital Health! Do you have disruptive science but struggle to bring it to life in the entrepreneurial world? Bayer's passion is to enable and support standout healthcare innovation! Take your innovation to new heights and join Bayer´s Expert Mondays 2024, a series of knowledge exchange sessions to supercharge your start-up’s growth.
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For additional information, please reach out to Save@BIO.org. | |
This e-newsletter was sent by BioNJ. | | | | |