Welcome to the latest edition of the BioLines Weekender...
We at BioNJ are profoundly saddened by the passing of Representative Bill Pascrell, a devoted public servant who dedicated his life to championing the needs of the people of New Jersey. Congressman Pascrell's distinguished career spanned decades, beginning with his service in the State General Assembly in 1988 and continuing through his tenure in the U.S. House of Representatives since 1997. As Chair of the Subcommittee on Oversight for the Committee on Ways and Means, he remained open to the views and needs of the life sciences community throughout the years.
BioNJ is deeply appreciative of Congressman Pascrell’s unwavering support for both the life sciences industry and, most importantly, Patients. We extend our heartfelt condolences to his family and loved ones during this difficult time.
Meanwhile, last week, the Centers for Medicare and Medicaid Services announced the prices that they have set for the first 10 medications as a byproduct of the Inflation Reduction Act’s (IRA) drug provisions. Given the effect that the IRA has been projected to have on investment in innovation across the life sciences — as well as the failure of this policy to address central drivers of inhibited Patient access — BioNJ will continue to advocate for the adoption of efforts to mitigate some of the most fundamentally damaging provisions of the Inflation Reduction Act, including the EPIC Act and ORPHAN Cures Act, to ensure that this legislation does not further diminish the development of therapeutics on which a wide variety of patient communities depends.
On a different note, Focus NJ has unveiled an exciting expansion of its online Workforce Development Initiatives and Opportunities Research Page, a dynamic public resource designed to bridge the gap between workers, employers and training providers across New Jersey. This enhanced platform serves as a comprehensive hub for discovering workforce development programs statewide. Thanks to recent efforts, the database has doubled in size, offering an impressive collection of over 400 training programs and providers — complete with detailed programmatic and contact information. Click here to learn more.
BioNJ remains steadfast in supporting professionals on their journey toward their next career milestone. Through BioNJ's Career Portal, individuals can access an extensive Job Board showcasing hundreds of life sciences opportunities within the thriving New Jersey region. From IT, marketing and HR roles to scientific, manufacturing and more, the portal provides a diverse range of openings. Explore your next opportunity by visiting www.BioNJTalentNetwork.org.
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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Medicare Announces Drug Prices for Historic First Round of Negotiations
The day drug makers dreaded has finally arrived. Medicare officials unveiled the results of the program’s first 10 drug price negotiations, despite the industry’s two-decade, multimillion-dollar lobbying campaign and barrage of lawsuits to stop them. The drugs that received negotiated prices are Bristol Myers Squibb’s blood thinner Eliquis, Boehringer Ingelheim’s diabetes drug Jardiance, Johnson & Johnson’s blood thinner Xarelto, Merck’s diabetes drug Januvia, AstraZeneca’s diabetes drug Farxiga, Novartis’ heart failure treatment Entresto, Amgen’s rheumatoid arthritis drug Enbrel, J&J and AbbVie’s blood cancer treatment Imbruvica, J&J’s anti-inflammatory medicine Stelara and Novo Nordisk insulins that go by names including Fiasp and NovoLog.
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Understanding the IRA’s Real-World Impacts Starts With Understanding the Innovation Ecosystem
The Inflation Reduction Act (IRA) has already begun to shift the way that innovative companies are investing in future breakthroughs. Over the past few months, CEOs have been explicit about the incentives their businesses are facing, including the law’s inducements to favor large-molecule medicines over pills and limit orphan drug development. Despite the law’s already emerging dangers to drug development, anti-innovation skeptics have tried to suggest, using questionable assumptions, that the law will have no impact on how biotechnology companies develop medicines.
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The Impact of Proposed Government-Mandated Drug Pricing Policies
A new study estimates that there would be 237 fewer FDA approvals of new medicines for patients battling cancer, neurological, rare and infectious and other diseases over the next decade – as well as 1.1 million lost jobs – if proposals to expand government-mandated drug pricing policies are implemented. Watch this video to learn more and better understand the implications of expanded government-mandated drug pricing policies, and how they will erode the existing biopharmaceutical industry and related infrastructure and undercut innovations that patients desperately need.
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Innovative Payment Arrangements Bring Transformative Therapies from the Lab to Patients
In recent years, the number of disruptive, cutting-edge therapies approved or under review by the Food and Drug Administration (FDA) has grown substantially. So-called transformative therapies, including cell and gene therapies, offer new treatment options for patients, often for conditions that currently have limited or no treatment options available. However, these therapies also present challenges related to payment and access that vary by payer market and setting of care. Because they are characterized by having a small patient population; often no existing highly effective treatment; a substantial “cure” rate; and high cost; many transformative therapies do not fit neatly into legacy payment systems that often discourage the use of new technologies and may not pay accurately for low patient populations.
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FDA Approves YORVIPATH® (Palopegteriparatide) as the First and Only Treatment for Hypoparathyroidism in Adults
Princeton-based BioNJ Member Ascendis Pharma announced that the U.S. Food & Drug Administration (FDA) has approved YORVIPATH® (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults. YORVIPATH is a prodrug of parathyroid hormone (PTH[1-34]), administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the United States. “FDA approval of our second TransCon product, YORVIPATH, reflects our values and dedication to following the science to help patients, as well as our unwavering commitment these past years to addressing the significant unmet medical needs of the hypoparathyroidism community in the United States,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.
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TEPKINLY® (epcoritamab) Receives Second European Commission Approval for the Treatment of Adults With Relapsed/Refractory Follicular Lymphoma
Princeton-based BioNJ Member Genmab A/S announced that the European Commission (EC) has granted conditional marketing authorization for TEPKINLY® (epcoritamab) as a monotherapy for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy. TEPKINLY is the first and only subcutaneous T-cell engaging bispecific antibody approved for the treatment of this patient population in the European Union (EU), as well as the European Economic Area (EEA) countries (Iceland, Liechtenstein, Norway) and Northern Ireland. FL is typically a slow-growing form of Hodgkin’s lymphoma (NHL) that arises from B-cell lymphocytes. FL is the second most common form of NHL overall, accounting for 20-30 percent of all NHL cases, and represents 10-20 percent of all lymphomas in the western world.
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Novartis Receives FDA Accelerated Approval for Fabhalta® (iptacopan), the First and Only Complement Inhibitor for the Reduction of Proteinuria in Primary IgA Nephropathy (IgAN)
East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Fabhalta® (iptacopan), a first-in-class complement inhibitor for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. Fabhalta specifically targets the alternative complement pathway of the immune system. When overly activated in the kidneys, the complement system is thought to contribute to the pathogenesis of IgAN. This indication is granted under accelerated approval based on the pre-specified interim analysis of the Phase III APPLAUSE-IgAN study measuring reduction in proteinuria at 9 months compared to placebo. It has not been established whether Fabhalta slows kidney function decline in patients with IgAN.
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Sandoz Receives FDA Approval for Enzeevu™ (aflibercept-abzv), Further Strengthening U.S. Biosimilar Position
Princeton-based BioNJ Member Sandoz announced that the U.S. Food and Drug Administration (FDA) has approved Enzeevu™ (aflibercept-abzv) 2 mg vial kit and pre-filled syringe for intravitreal injection. Enzeevu™ is indicated to improve and maintain visual acuity in patients with neovascular age-related macular degeneration (nAMD). In addition, the FDA provisionally determined Enzeevu™ would be interchangeable with the reference medicine as it is currently subject to an unexpired exclusivity for the first interchangeable biosimilar products. Enzeevu™ is a key biosimilar value driver for the company and this approval is a major step in advancing the Sandoz growth strategy by further extending its leading U.S. ophthalmology portfolio. Launch timing will be dependent on several factors, including the progress and outcome of pending or potential future related litigations or any potential settlements.
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Pfizer Announces Top-Line Results of ABRYSVO® for RSV in Immunocompromised Adults
BioNJ Member Pfizer, with a site in Gladstone, announced positive top-line safety and immunogenicity results from substudy B of the ongoing pivotal Phase 3 clinical trial (NCT05842967) MONeT (RSV I Mmunizati ONStudy for Adul Ts at Higher Risk of Severe Illness), evaluating two doses of ABRYSVO vaccine in immunocompromised adults aged 18 and older at risk of developing severe respiratory syncytial virus (RSV)-associated lower respiratory tract disease (LRTD). Adults with immunocompromising conditions have an increased risk of developing RSV-LRTD. Substudy B of the MONeT trial was conducted to assess the safety and immunogenicity of two doses of ABRYSVO, administered one month apart, in four groups of immunocompromised adults: those with non-small cell lung cancer, those on hemodialysis due to end-stage renal disease, those with autoimmune inflammatory disorder receiving active immunomodulator therapy, and solid organ transplant recipients.
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Pfizer and BioNTech Receive U.S. FDA Approval & Authorization for Omicron KP.2-adapted COVID-19 Vaccine
BioNJ Member Pfizer, with a site in Gladstone, and BioNTech SE announced that the U.S. Food and Drug Administration (“FDA”) has approved the supplemental Biologics License Application for individuals 12 years of age and older (COMIRNATY ® (COVID-19 Vaccine, mRNA), and granted emergency use authorization for individuals 6 months through 11 years of age (Pfizer-BioNTech COVID-19 Vaccine) of the companies’ Omicron KP.2-adapted 2024-2025 Formula COVID-19 vaccine. This season’s vaccine is for use as a single dose for most individuals 5 years of age and older. Individuals 5 years of age and older with certain kinds of immunocompromise previously vaccinated with Pfizer and BioNTech COVID-19 vaccines or children under the age of 5 who have not already completed a three-dose series with previous formulas of a COVID-19 vaccine may be eligible to receive additional doses.
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Pfizer and BioNTech Provide Update on mRNA-based Combination Vaccine Program Against Influenza and COVID-19 in Individuals 18-64 Years of Age
BioNJ Member Pfizer, with a site in Gladstone, and BioNTech SE announced top-line results from their Phase 3 clinical trial to evaluate the companies’ combined mRNA vaccine candidate against influenza and COVID-19 in healthy individuals 18-64 years of age. The combination candidate consists of Pfizer’s mRNA-based influenza vaccine candidate with the companies’ licensed COVID-19 vaccine. The Phase 3 trial measured two primary immunogenicity objectives (immunogenicity against SARS-CoV-2 as well as immunogenicity against influenza A and B), of which one was met. In a separate Phase 2 trial, Pfizer evaluated trivalent (“tIRV”) influenza mRNA standalone vaccine candidates which demonstrated robust immunogenicity in individuals 18-64 years of age. The companies are evaluating adjustments to the combination vaccine candidate aimed at improving immune responses against influenza B and will discuss next steps with health authorities.
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Tirzepatide Reduced the Risk of Developing Type 2 Diabetes by 94% in Adults With Pre-Diabetes and Obesity or Overweight
BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced positive topline results from the SURMOUNT-1 three-year study (176-week treatment period) evaluating the efficacy and safety of tirzepatide (Zepbound® and Mounjaro®) once weekly for long-term weight management and delay in progression to diabetes in adults with pre-diabetes and obesity or overweight. Weekly tirzepatide injections (5 mgi, 10 mg, 15 mg) significantly reduced the risk of progression to type 2 diabetes by 94% among adults with pre-diabetes and obesity or overweight compared to placebo. Additionally, treatment with tirzepatide resulted in sustained weight loss through the treatment period, with adults on the 15 mg dose experiencing a 22.9% average decrease in body weight compared to 2.1% for placebo in adults with pre-diabetes and obesity or overweight at the end of the treatment period.
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Lilly Completes Acquisition of Morphic to Improve Outcomes and Expand Options for People Living With Inflammatory Bowel Disease
BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced the successful completion of its acquisition of Morphic Holding, Inc. Morphic is a biopharmaceutical company developing oral integrin therapies for treatment of serious chronic diseases, including a selective oral small molecule inhibitor of α4β7 integrin (known as MORF-057) for inflammatory bowel disease (IBD). "Acquiring Morphic reinforces our growing capabilities in gastroenterology, building on the strong foundation of Omvoh, our first-in-class molecule already approved and launched around the world for ulcerative colitis and under regulatory review for Crohn's disease,” said Daniel Skovronsky, M.D., Ph.D., Chief Scientific Officer of Lilly and President, Lilly Research Laboratories and Lilly Immunology.
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Princeton-based BioNJ Member Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Opdivo ® (nivolumab) plus Yervoy ® (ipilimumab) as potential first-line treatment for adult patients with unresectable hepatocellular carcinoma (HCC), based on results from the Phase 3 CheckMate -9DW trial. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of April 21, 2025. The filing was based on the results from the Phase 3 CheckMate -9DW study in which the combination demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) compared to investigator’s choice of lenvatinib or sorafenib. The combination of Opdivo plus Yervoy has been an established second-line treatment for patients with advanced HCC, and these results support the combination becoming a potential new treatment option in the first-line setting.
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European Medicines Agency Validates Bristol Myers Squibb’s Application for CAR T Cell Therapy Breyanzi for Relapsed or Refractory Follicular Lymphoma
Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Medicines Agency (EMA) has validated its Type II variation application to expand the indication for Breyanzi ® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. Validation of the application confirms the submission is complete and allows the scientific review to begin under the EMA’s centralized review procedure. The application is supported by data from the Phase 2 TRANSCEND FL study, the largest clinical trial to date to evaluate a CAR T cell therapy in patients with relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including FL.
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GSK Receives U.S. FDA Breakthrough Therapy Designation for its B7-H3-Targeted Antibody-Drug Conjugate in Relapsed or Refractory Extensive-Stage Small-Cell Lung Cancer
Warren-based BioNJ Member GSK announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for GSK5764227 (GSK’227), the Company’s investigational B7-H3-targeted antibody drug conjugate (ADC) being evaluated for the treatment of patients with extensive-stage small-cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy (relapsed or refractory). Lung cancer is one of the most common cancers worldwide. In the US, approximately 15% of all lung cancers are small-cell. Of patients with small-cell lung cancer, 70% have extensive-stage disease meaning the cancer has spread throughout one or both lungs and/or to other parts of the body. ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options. The 5-year survival rate is approximately 3%.
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RYBREVANT® (amivantamab-vmjw) Plus LAZCLUZE™ (lazertinib) Approved in the U.S. as a First-Line Chemotherapy-Free Treatment for Patients With EGFR-Mutated Advanced Lung Cancer
Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) approved RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE™ (lazertinib) for the first-line treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test. With this milestone, RYBREVANT® plus LAZCLUZE™ becomes the first and only multitargeted, chemotherapy-free combination regimen with demonstrated superiority versus osimertinib approved for the first-line treatment of patients with EGFR-mutated NSCLC. RYBREVANT® is an EGFR- and MET*-directed bispecific antibody that engages the immune system, and LAZCLUZE™ is a highly selective, brain-penetrant, third-generation oral EGFR TKI. RYBREVANT® plus LAZCLUZE™ is the only multitargeted regimen targeting both the common EGFR mutations directly.
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Johnson & Johnson to Acquire V-Wave
New Brunswick-based BioNJ Member Johnson & Johnson announced that it has entered into a definitive agreement to acquire V-Wave Ltd., a privately held company focused on developing innovative treatment options for patients with heart failure. Under the terms of the agreement, Johnson & Johnson will acquire V-Wave for an upfront payment of $600 million, subject to customary adjustments, with the potential for additional regulatory and commercial milestone payments up to approximately $1.1 billion. V-Wave will join Johnson & Johnson as part of Johnson & Johnson MedTech. The planned acquisition of V-Wave will extend Johnson & Johnson MedTech’s position as an innovation leader in addressing cardiovascular disease. It will further accelerate its shift into high-growth and high-opportunity markets and will deepen its relationships with structural interventional cardiologists and heart failure specialists.
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Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by U.S. FDA
BioNJ Member Gilead Sciences, with a site in Morris Plains, announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Livdelzi® (seladelpar) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. The use of Livdelzi is not recommended for people who have or develop decompensated cirrhosis. The FDA approved Livdelzi under accelerated approval based on a reduction of ALP. Improvement in survival or prevention of liver decompensation events have not been demonstrated. Continued approval of Livdelzi for the approved indication may be contingent on verification and description of clinical benefit in confirmatory trial(s).
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Chugai Files Elevidys (SRP-9001) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan
BioNJ Member Chugai Pharmaceutical Co., with a site in Berkeley Heights, announced that it filed a regulatory application with the Ministry of Health, Labour and Welfare (MHLW) for delandistrogene moxeparvovec, a gene therapy product under development (development code: SRP-9001, overseas product name: Elevidys), for the treatment of Duchenne muscular dystrophy (DMD). Its target population aims to include ambulatory boys aged 3-7 years with DMD who do not have any deletion in exons 8 and/or 9 in the DMD gene and do not have a pre-existing immunity against AAVrh74. Delandistrogene moxeparvovec received orphan regenerative medical product designation from the MHLW, and the applications will be reviewed under priority review.
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Chugai In-Licenses Anti-TL1A Antibody RG6631 for the Intractable Diseases Ulcerative Colitis and Crohn’s Disease
BioNJ Member Chugai Pharmaceutical Co., with a site in Berkeley Heights, announced that it has concluded a license agreement with F. Hoffmann-La Roche Ltd. for RG6631, the anti-TL1A antibody, currently in development for ulcerative colitis and Crohn’s disease. Under the license agreement between Roche and Chugai, Chugai obtained exclusive rights for the development and marketing of RG6631 in Japan. Roche will receive an upfront fee and milestone payments. RG6631 is a potential first-in-class therapy targeting TL1A, is expected to demonstrate high efficacy by simultaneously suppressing inflammation and fibrosis and has the potential to be applied in multiple other diseases. In the global Phase IIb study (TUSCANY-2)2 in patients with moderate to severe ulcerative colitis, treatment with RG6631 showed improvement in multiple endpoints including clinical remission in both the induction and maintenance periods.
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“LEQEMBI®” (Lecanemab) Approved for the Treatment of Alzheimer’s Disease in the United Arab Emirates
Nutley-based BioNJ Member Eisai Co., Ltd. and Biogen Inc. announced that the Ministry of Health and Prevention in the United Arab Emirates (UAE) has approved humanized anti-soluble aggregated amyloid-beta (Aβ) monoclonal antibody “LEQEMBI®” (lecanemab) for the 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’s approval is based on the large global Phase 3 Clarity AD study. In the Clarity AD study, LEQEMBI met its primary endpoint and all key secondary endpoints with statistically significant results. In the UAE, it is reported that 4.09% of those over 60 years old have dementia.
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Lexicon Pharmaceuticals Announces FDA Advisory Committee Meeting for Zynquista
BioNJ Member Lexicon Pharmaceuticals, Inc., with a site in Bridgewater, announced that the U.S. Food and Drug Administration (FDA) expects to convene a meeting of the Endocrinologic and Metabolic Drugs Advisory Committee for Zynquista™ (sotagliflozin) as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease (CKD) on October 31, 2024, with no change to the PDUFA goal date of December 20, 2024. According to the notice provided, the FDA intends to make the background materials available to the public no later than two business days before the meeting. “Lexicon looks forward to the opportunity to share the breadth of evidence that has been generated to support the favorable benefit/risk profile of sotagliflozin for glycemic control in people with type 1 diabetes and CKD,” said Dr. Mike Exton, CEO and Director.
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Phathom Pharmaceuticals Announces Pricing of $130 Million Offering of Common Stock and Pre-Funded Warrants
Florham Park-Based BioNJ Member Phathom Pharmaceuticals, Inc. announced the pricing of its underwritten offering of 8,695,652 shares of its common stock and pre-funded warrants to purchase 2,608,922 shares of common stock. The shares of common stock are being sold at a price of $11.50 per share and the pre-funded warrants are being sold at a price of $11.499 per pre-funded warrant, which represents the per share price for the common stock less the $0.001 per share exercise price for each such pre-funded warrant. The gross proceeds to Phathom from the offering, before deducting the underwriting discounts and commissions and other offering expenses, are expected to be approximately $130 million. Phathom intends to use the net proceeds from the offering to fund commercialization expenses and further clinical development of vonoprazan and for working capital and general corporate purposes.
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JLL Q2 N.J. Life Science Report: Flight to Quality Migration Continues to Drive Workspace Requirements
JLL recently released its Q2 New Jersey Life Science update, noting that flight-to-quality migration will continue to drive life sciences workspace requirements. The data suggests that, while life sciences companies generated nearly 30% of New Jersey’s leasing velocity during the first half of 2023, demand had since decelerated one year later. Approximately 22% of leases involved life sciences companies during the first six months of this year, which lagged the 28% of activity registered by the banking/financial services sector. Among the notable deals includes BeiGene opening its new $800 million, 470,000-square-foot flagship biologic pharmaceutical and manufacturing facility at the Princeton West Innovation Campus @ Hopewell. In New Brunswick, construction continues on the first phase of the HELIX (New Jersey Health + Life Sciences Exchange) project.
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Organon, Eli Lilly Expand Partnership for Migraine Medication
Jersey City-based Organon is expanding its distribution and promotion agreement with BioNJ Member Eli Lilly and Co. for migraine medicine Emgality. Emgality, a monoclonal antibody that selectively binds to calcitonin gene-related peptide (CGRP), is used for the treatment of migraines in adults. The new agreement builds on a previous partnership between the two organizations: Organon has served as the sole distributor and promoter of Emgality in Europe since February 2024. Following this deal, the company will become the sole distributor and promoter for Emgality in: Canada, Colombia, Israel, South Korea, Kuwait, Mexico, Qatar, Saudia Arabia, Taiwan, Turkey and United Arab Emirates. Under the terms of the agreement, Eli Lilly will receive an upfront payment of $22.5 million as well as sales-based milestone payments. It will also remain the marketing authorization holder as well as manufacture the product for sale.
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Amneal Receives U.S. FDA Approval for IPX203 for Treatment of Parkinson’s Disease to Be Launched as CREXONT® (Carbidopa and Levodopa) Extended-Release Capsules
Bridgewater-based Amneal Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved CREXONT ® (carbidopa and levodopa) extended-release capsules for the treatment of Parkinson’s disease (PD). CREXONT is a novel, oral formulation of carbidopa/levodopa (CD/LD) that combines both immediate-release (IR) granules and extended-release (ER) pellets. “The approval of CREXONT is a seminal moment in the treatment paradigm for Parkinson’s disease. The burden of this incurable neurodegenerative disease increases with time. Some PD patients on IR CD/LD take up to 10 daily doses and still experience motor fluctuations. CREXONT’s innovative formulation provides a longer duration of “Good On” time with less frequent dosing compared to IR CD/LD,” said Chirag and Chintu Patel, Co-CEOs of Amneal.
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ENHERTU ® Approved in China for Patients With Previously Treated HER2 Positive Advanced or Metastatic Gastric Cancer
Basking Ridge-based Daiichi Sankyo and AstraZeneca’s ENHERTU® (trastuzumab deruxtecan) has received conditional approval in China as a monotherapy for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received two or more prior treatment regimens. This indication was granted conditional approval based on results from the DESTINY-Gastric06 Phase 2 trial. Full approval for this indication will depend on whether a randomized controlled confirmatory clinical trial can demonstrate clinical benefit in this population. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca.
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ENHERTU® Granted Breakthrough Therapy Designation in U.S. for Certain Patients With HER2 Low or HER2 Ultralow Metastatic Breast Cancer
Basking Ridge-based Daiichi Sankyo announced that ENHERTU® (fam-trastuzumab deruxtecan-nxki) has been granted Breakthrough Therapy Designation in the U.S. for the treatment of unresectable or metastatic hormone receptor positive HER2 low (IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (IHC >0 <1+) breast cancer patients who have received either two lines of endocrine therapy in the metastatic setting, or one line of endocrine therapy if they had demonstrated disease progression within six months of starting first-line treatment with endocrine therapy in combination with a CDK4/6 inhibitor or within 24 months of the start of adjuvant endocrine therapy. The FDA granted this BTD based on data from the DESTINY-Breast06 Phase 3 trial.
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ENHERTU ® Type II Variation Application Validated by EMA for Patients With HER2 Low or HER2 Ultralow Metastatic Breast Cancer Following at Least One Endocrine Therapy
Basking Ridge-based Daiichi Sankyo announced that the European Medicines Agency (EMA) has validated the Type II Variation application for ENHERTU® (trastuzumab deruxtecan) as a monotherapy for the treatment of adult patients with unresectable or metastatic HER2 low (defined as IHC 1+ or IHC 2+/ISH-) or HER2 ultralow (defined as IHC 0 with membrane staining) breast cancer who have received at least one endocrine therapy in the metastatic setting. ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use.
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NextGen Class of 2024
BioSpace is proud to present its NextGen Bio Class of 2024, a list of the hottest new life sciences companies in the United States. To come up with this list, BioSpace identified companies that launched between September 2022 and September 2023 with Series A funding. They were then assessed using several different criteria including finance, partnerships, pipeline, growth potential and innovation. After awarding points for each category, the BioSpace editorial team ranked its top 30. Congratulations to New Jersey's LinusBio for making the list. LinusBio’s platform introduces precision exposome biomarkers to identify a person’s various environmental exposures and potentially help diagnose diseases and monitor progression and serve as objective endpoints in clinical trials.
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Stevens, Green Room Partner on AI Health Care Initiative
Aiming to advance understanding and communication of artificial intelligence (AI) within the health care sector, Stevens Institute of Technology and Green Room Communications unveiled a new strategic partnership. The collaboration leverages the health care communications expertise of the Parsippany-headquartered company with the cutting-edge research capabilities and AI knowledge of the Hoboken-based research university. In particular, the school’s Stevens Institute of Artificial Intelligence (SIAI). Through the partnership, Stevens and Green Room will explore how AI is revolutionizing drug discovery, clinical trials and health care delivery. The partnership will also explore how these innovations are reshaping communication strategies within the biopharma industry.
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Focus NJ Unveils Expanded Workforce Development Training Tool
Focus NJ announced the expansion of its online Workforce Development Initiatives and Opportunities Research Page. Offering a catalog of workforce development training programs throughout New Jersey, the public tool provides a resource to link workers, employers and training providers. It is maintained to assist workers with reskilling and upskilling – while also helping employers address workforce shortages. Focus NJ Executive Director, Kyle Sullender, noted that recent efforts have effectively doubled the size of Focus NJ’s online database and mapping. Now, it contains information for more than 400 training programs and providers throughout the State. Established in 2020, Focus NJ – the Center for Economic Research and Workforce Solutions – is a nonpartisan, nonprofit research foundation aimed at analyzing public policies, industry trends and economic indicators that impact the Garden State’s overall affordability, workforce and competitiveness.
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Students 2 Science Names Walker as New Chair, Appoints 7 New Trustees
Students 2 Science, a North Jersey 501(c)(3) nonprofit organization that aims to inspire and empower students from communities in need through experiential learning to pursue careers in STEM, recently announced a new chair and seven new trustees. Karnel Walker, an Executive Director at Thermo Fisher Scientific, was selected as the new board chair. Officials at Students 2 Science, which has centers in East Hanover and Newark, said each new trustee brings an extensive breadth of knowledge and experiences in a wide variety of fields, as well as a passion for and commitment to science, technology, engineering and math education. Walker said he is eager to begin his new role. “I am thrilled to take on the role of Chair for Students 2 Science, an organization deeply committed to fostering diversity & inclusion in STEM,” she said. “I’m truly inspired to embark on an ambitious path with an amazingly talented board that includes program expansion, more technology centers and curriculum development to meet future workforce needs in the state of New Jersey.
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Rutgers Health Awarded $4.8M Grant to Fund Cardiovascular Risks in Cancer Survivors
Researchers from Rutgers Robert Wood Johnson Medical School and Rutgers Cancer Institute in New Brunswick have been awarded a five-year, $4.83 million grant from the National Cancer Institute to support efforts in coordinating care for cancer survivors who are managing cardiovascular risks. Led by Denalee O’Malley, an assistant professor and social work researcher in the Department of Family Medicine and Community Health at Rutgers, the study builds on more than 15 years of extensive investigation into the challenges of integrating primary care in survivorship care. The team is one of four sites nationally to receive funding this year as part of the NCI’s initiative to address primary care needs of cancer survivors.
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Rutgers Cancer Institute Treats 200th CAR T-Cell Therapy Patient
Rutgers Cancer Institute, together with RWJBarnabas Health, said it recently treated its 200th patient with CAR T-cell therapy, a type of immunotherapy that uses a patient’s own immune cells to fight cancer. Harnessing the immune system to target cancer is the goal of a groundbreaking area of research called cell therapy. The process includes collecting T-cells from a patient’s blood through an infusion-like process called apheresis, and the cells are sent to a laboratory. In the lab, scientists modify the T-cells by adding a special receptor called chimeric antigen receptor, which enables the modified T-cells to seek out and kill cancer cells. The patient’s reengineered CAR T-cells are returned to them through an infusion, and the cells begin to multiply and attach to cancer cells to destroy them.
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Role of State Legislators With STEM Backgrounds in Advancing Evidence-Informed AI Legislation in U.S. States
Scientists must work together with policymakers and the public to shape sound AI policy that harnesses its potential to benefit individuals and society while placing checks on its potential to cause harms. There is already a great deal of insights from across scientific disciplines and professional practice fields that can productively inform policy discourse but does not effectively reach federal and state policymakers. There are many well documented barriers to efficient and productive flow and exchange of knowledge between science and policy, particularly when scientific complexity and politics get in the way, but research on the use of research evidence in policy generally highlights the important role of evidence intermediaries in bridging the gap.
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N.J. Ranked No. 3 in Country for VC Investment in 1st Half of 2024
Gov. Phil Murphy campaigned on the idea of recreating and reinvigorating an “innovation” economy in the State — an initiative he has pushed throughout his six years in office. The effort appears to be paying off. PitchBook ranked New Jersey third in the country for venture capital investment in the first half of 2024 — another milestone climb in VC investment that had dropped dramatically before he took office. In the first six months of the year, New Jersey had $9.8 billion in VC investment (on 103 deals). The $9.8 billion investment in VC money in the first six months roughly equals the total VC investment in the state for 2021-23 in its entirety. Administration officials said the increases can be attributed to a number of things. More specifically, they noted programs such as the unique Innovation Evergreen Fund, which was launched in 2022 by the New Jersey Economic Development Authority — giving the state a groundbreaking tool to increase access to strategic resources and venture capital.
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NJ Small Business Manual
The “New Jersey Small Business Manual” provides information on how to establish, maintain and expand a small business in New Jersey. Individuals may order, at no charge, one hard copy or download a copy print a digital version.
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BD Names Michael Feld as President of Life Sciences Segment
Franklin Lakes-based BD (Becton, Dickinson and Co.) announced that Michael Feld will serve as Executive Vice President and President of the Life Sciences Segment for BD. Mr. Feld succeeds Dave Hickey, who retired from BD last month after 10 years with the company. Coming to BD from Veralto, Mr. Feld most recently served at the Danaher spinoff as President of its Analytical Instrument Business, Hach. Prior to that, he held several senior roles at Danaher, including Senior Vice President and General Manager of Cepheid Europe, and served as President of several other Danaher businesses, including Mammotome, XOS and Dover Motion, all focused on strategic planning, superior quality, associate development and cultural progression.
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ESG Best Practices: Tools from Which to Learn | |
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2023 Insmed Responsibility Report
As a company committed to transforming the lives of patients facing serious and rare diseases, corporate responsibility is at the core of what we do. Our actions are guided by the needs of our patients, as well as the families and caregivers who support them. We take this responsibility very seriously, and have robust governance frameworks in place to uphold our commitment to patient support and stakeholder value creation. As we grow, we continue to prioritize integrating ESG principles across our enterprise, fostering ongoing transparency and accountability. By embedding an ESG mindset across all levels of Insmed, guided by our Board of Directors and highest levels of leadership, our dedication to responsible business is evident in every aspect of our operations.
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FDA | NIH: Regulatory Do’s and Don’ts: Tips from FDA
September 4, 2024 – Hosted by CDER SBIA
This September 4, 2024 webinar will provide an overview of resources and programs FDA has developed across CBER, CDER and CDRH to help academic life sciences accelerators and early stage, oncology-related companies. This is great chance for entrepreneurs new to the FDA to learn when to contact the Agency during preclinical development. The discussion will include Q&A sessions with FDA experts.
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Electronic Drug Registration and Listing (eDRLS) Using CDER Direct 2024 – Webinar
September 12, 2024 – Hosted by CDER SBIA
This webinar will feature a demonstration of how to submit establishment registration and drug listing data using CDER Direct, give an overview on registration and listing regulatory requirements and compliance framework and present case studies for an interactive learning experience.
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Advancing Generic Drug Development: Translating Science to Approval 2024 – Hybrid
September 24 - 25, 2024 – Hosted by CDER SBIA
At this event, FDA experts will demonstrate the FDA’s Generic Drug User Fee Amendments (GDUFA) Science and Research Program’s transformative impact on generic drug development, regulation, and approval. Attend to gain insights into GDUFA III progress, GDUFA science and research on complex products, and scientific issues related to product-specific guidance development, pre-ANDA discussions, and ANDA meetings. The workshop will also highlight innovative science and cutting-edge methodologies in generic drug development within the U.S. and globally. FDA Commissioner Robert M. Califf will deliver the keynote!
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2024 SAPA Annual Conference: Redefining Medicine: Navigating Resilience, Transforming Lives
September 27-28, 2024
Sino-American Pharmaceutical Professionals Association (SAPA) will host the 2024 Annual Conference on Friday & Saturday, September 27 & 28 at Hyatt Regency New Brunswick, NJ. We are thrilled to invite you to our upcoming event, packed with insightful sessions, networking opportunities, and engaging discussions with industry leaders! The conference theme this year is “Redefining Medicine: Navigating Resilience, Transforming Lives” and will feature 2 plenary sessions along with 6 parallel sessions, including Executive & Legal Summit; CMC & Outsourcing; Career Development; Investment & Business Development; Drug Discovery and Clinical Development & Regulatory Forums.
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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. | |
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