New Research Shows Alarming Drop in Neurology Innovation Due to IRA’s Small Molecule ‘Pill Penalty’

During an event on Capitol Hill, California Rep. Scott Peters highlighted a graphic that clearly illustrates the distorted impact the Inflation Reduction Act (IRA) has levied on small molecule drugs and biologics. Peters explained the IRA currently allows nine years for small molecule drugs to earn market exclusivity before government price setting takes effect. Biologics, meanwhile, are granted 13 years. According to Vital Transformation's analysis of companies with a market capitalization of more than $5 billion, small molecule research candidates comprised about 80% of neurology clinical trials in 2023, but activity crashed to 0% from January to May 2024. In the same span, biologics have risen from just over 20% of new trials to 100%.

A White House Proposal That Could Harm the VC-Dependent

The White House could soon finalize a proposal that would shift the venture capital investment landscape in fields like renewable energy, quantum computing, national security and life sciences. If the plan moves ahead, high-tech entrepreneurs may not be able to raise the capital needed to develop the cutting-edge technologies of the future. The proposal centers on the 1980 Bayh-Dole Act, which allows universities, federal labs and businesses to file patents for any discoveries they make with the help of federal funding. Prior to the law, any patents resulting from taxpayer funding automatically belonged to the government, and billions of dollars worth of research simply gathered dust.

FDA Drug Application Costs Set to Rise to $4.3m from October

The U.S. Food and Drug Administration (FDA) has announced the cost to file a drug using clinical data will jump by around $300,000 in 2025 to more than $4.3M. On 30 July, the agency released its federal docket, Prescription Drug User Fee Rates for Fiscal Year 2025, which outlined the new fees that pharmaceutical companies will face following clinical trials if they hope to get market access. Filing for approval with clinical data will cost a sponsor $4.3M while an application that does not require clinical data will cost half of that, just under $2.2M. These costs have increased by around $300,000, from $4M for a clinical data application in 2024 and by approximately $150,000 for a drug, which does not require clinical data and cost $2M in 2024.

Citius Pharmaceuticals Receives FDA Approval for LYMPHIR™ (denileukin diftitox-cxdl) Immunotherapy for the Treatment of Adults with Relapsed or Refractory Cutaneous T-Cell Lymphoma

Cranford-based BioNJ Member Citius Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved LYMPHIR™ (denileukin diftitox-cxdl), a novel immunotherapy for the treatment of r/r cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. LYMPHIR is the only CTCL therapy that targets the interleukin-2 (IL-2) receptor found on malignant T-cells and Tregs. This is the first indication for LYMPHIR and the first FDA-approved product for Citius Pharma. CTCL is a rare and often debilitating chronic non-Hodgkin lymphoma that primarily affects the skin. Approximately 2,500-3,000 patients are diagnosed each year with an estimated 40,000 living with the disease. Patients with r/r CTCL have limited treatment options. No universally defined single treatment is used to treat these patients with incurable cancer. Patients typically cycle through several skin-directed therapies before the cancer becomes resistant and/or progressive at which point systemic agents are needed to achieve effective disease control. 

PTC Therapeutics Announces Sepiapterin NDA Submission to FDA

South Plainfield-based BioNJ Member PTC Therapeutics announced the submission of the sepiapterin NDA to the U.S. FDA. The NDA submission is for the treatment of pediatric and adult patients with phenylketonuria (PKU), including the full spectrum of ages and disease subtypes. "This NDA submission is an important step in bringing this therapy to children and adults living with PKU in the U.S.," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The robust sepiapterin clinical trial data, including Phe tolerance results, support the potential of sepiapterin to meet the persistent unmet medical need for the large number of children and adults with PKU." The sepiapterin NDA is based on the highly statistically significant and clinically meaningful results from the phase 3 APHENITY trial. The results demonstrate a mean reduction in Phe levels of 63% in the overall treated population and 69% in the subgroup of subjects with classical PKU.

Celldex Therapeutics Presents Positive Topline Results from Barzolvolimab Phase 2 Study in Chronic Inducible Urticaria

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced positive topline results from the company’s Phase 2 clinical trial of barzolvolimab in two of the most common forms of chronic inducible urticaria (CIndU) — cold urticaria (ColdU) and symptomatic dermographism (SD). The study includes patients who remain symptomatic despite treatment with antihistamines. Barzolvolimab is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity, which is required for mast cell function and survival. CIndU is characterized by the occurrence of hives or wheals that have an attributable trigger associated with them—exposure to cold temperatures in ColdU and scratching/rubbing of the skin in SD. Mast cell activation is known to be a critical driver in ColdU and SD.

Soligenix Announces Recent Accomplishments and Second Quarter 2024 Financial Results

Princeton-based BioNJ Members Soligenix, Inc. announced its recent accomplishments and financial results for the quarter ended June 30, 2024. "This is a pivotal time for Soligenix with a great deal of clinical activity and upcoming milestones," stated Christopher J. Schaber, PhD, President and CEO of Soligenix. "While we prepare for the upcoming initiation of our confirmatory Phase 3 placebo-controlled study evaluating the safety and efficacy of HyBryte™ (synthetic hypericin) in the treatment of cutaneous T-cell lymphoma (CTCL) patients with early-stage disease, we are incredibly encouraged by the recent positive clinical results from our comparability study evaluating HyBryte™ against Valchlor® (mechlorethamine gel), which demonstrated a three-fold higher response rate over a 12-week treatment period and more favorable safety profile for HyBryte™ in the treatment of CTCL. These findings, coupled with the promising interim data from the ongoing open-label, investigator-initiated study evaluating extended HyBryte™ treatment, reinforce our excitement about the future of HyBryte™ as a potential front-line treatment option for patients with early-stage CTCL."

Novartis Scemblix® Granted FDA Priority Review for the Treatment of Adults With Newly Diagnosed CML

East Hanover-based BioNJ Member Novartis announced today that Scemblix® (asciminib) has been granted Priority Review status by the U.S. Food and Drug Administration (FDA) for treatment of newly diagnosed adult patients with Philadelphia chromosome-positive CML in chronic phase (Ph+ CML-CP). The FDA grants Priority Review to medicines that address serious or life-threatening diseases or conditions and, if approved, would provide significant improvements in treatment safety or efficacy. Scemblix previously received Breakthrough Therapy designation for the treatment of newly diagnosed adult patients and is currently being reviewed under the FDA’s Real-Time Oncology Review (RTOR) program. Scemblix received Priority Review and Breakthrough Therapy designations at the time of the original new drug application for the treatment of adult patients with Ph+ CML-CP who have previously been treated with two or more TKI.

Lilly's Tirzepatide Successful in Phase 3 Study Showing Benefit in Adults With Heart Failure With Preserved Ejection Fraction and Obesity

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced positive topline results from the SUMMIT Phase 3 clinical trial evaluating the safety and efficacy of tirzepatide injection (5 mg, 10 mg or 15 mg) in adults with heart failure with preserved ejection fraction (HFpEF) and obesity. Tirzepatide demonstrated statistically significant improvements in both primary endpoints with a reduction in the risk of heart failure outcomes, assessed as a composite endpoint, and improvements in heart failure symptoms and physical limitations, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ) Clinical Summary Score (CSS), compared with placebo.

Genmab Takes Full Control of Acasunlimab Development Program

Princeton-based BioNJ Member Genmab announced that it will assume sole responsibility for the continued development and potential commercialization of acasunlimab. BioNTech SE has opted not to participate in the further development of the acasunlimab program under the parties’ existing collaboration agreement. The program will be subject to payment of certain milestones and a tiered single-digit royalty on net sales by Genmab to BioNTech. Genmab plans to initiate the Phase 3 study in the second half of this year. While the emerging clinical profile of acasunlimab is encouraging, BioNTech informed the company that it has taken this decision for reasons relating to its portfolio strategy. The companies’ long-standing collaboration in antibody science remains in place, and both parties will continue with the existing programs under development under their existing agreements, which were expanded in 2022.

Sarclisa Induction Treatment Demonstrated Significantly Improved Progression-Free Survival in Patients With Newly Diagnosed Multiple Myeloma Eligible for Transplant

Bridgewater-based BioNJ Member Sanofi announced new results from the two-part, double-randomized, German-speaking Myeloma Multicenter Group (GMMG)-HD7 Phase 3 study show that Sarclisa (isatuximab) in combination with lenalidomide, bortezomib and dexamethasone (RVd) during induction therapy in transplant-eligible, newly diagnosed multiple myeloma (NDMM) significantly prolonged progression-free survival (PFS) from first randomization, resulting in a statistically significant and clinically meaningful reduction in disease progression or death, compared to RVd induction regardless of the maintenance regimen. GMMG-HD7 is one of six Phase 3 studies to report positive results for Sarclisa in patients with multiple myeloma, which includes four positive readouts of a Sarclisa-based quadruplet in the frontline setting.

Groundbreaking Nipocalimab Study of Pregnant Individuals at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn Published in The New England Journal of Medicine

New Brunswick-based BioNJ Member Johnson & Johnson announced the results from the Phase 2 open-label UNITY study of nipocalimab for the treatment of alloimmunized pregnant individuals at risk of early onset severe (EOS) HDFN have been published in The New England Journal of Medicine (NEJM). The UNITY study met its primary endpoint with 54 percent of individuals receiving nipocalimab achieving a live birth at or after 32 weeks gestational age (GA) without the need for IUT. Nipocalimab is currently the only therapy reported to be in clinical development for HDFN, a serious and rare condition that occurs when the blood types of a pregnant individual and the developing fetus are incompatible, potentially causing life-threatening anemia in the fetus or infant. These results showed that nipocalimab delayed or prevented severe fetal anemia requiring treatment prenatally and reduced the need for IUTs in pregnancies at high risk for recurrent EOS HDFN.

DARZALEX FASPRO® (Daratumumab and Hyaluronidase-fihj)-Based Quadruplet Regimen Approved in the U.S. for Patients With Newly Diagnosed Multiple Myeloma Who are Transplant-Eligible

New Brunswick-based BioNJ Member Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) approved DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone (D-VRd) for induction and consolidation in patients with newly diagnosed multiple myeloma (NDMM) who are eligible for an autologous stem cell transplant (ASCT). Patients will have the opportunity to receive this DARZALEX FASPRO®-based quadruplet therapy at initial diagnosis, providing them with a new treatment that may significantly improve outcomes. This approval is supported by data from the Phase 3 PERSEUS study evaluating DARZALEX FASPRO^® in a regimen that included D-VRd induction and consolidation therapy compared to bortezomib, lenalidomide and dexamethasone (VRd) during induction and consolidation in patients with NDMM eligible for ASCT.

European Regulatory Authority Adopts a Positive Opinion for an Update of the Wegovy® Label to Reflect Risk Reduction of Major Adverse Cardiovascular Events

Plainsboro-based BioNJ Member Novo Nordisk announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion for an update of the Wegovy® (semaglutide 2.4 mg) label to reflect data from the SELECT cardiovascular outcomes trial, demonstrating a risk reduction of major adverse cardiovascular events (MACE) including cardiovascular death, non-fatal heart attack (myocardial infarction) or non-fatal stroke in adults with established cardiovascular disease and either overweight or obesity (initial BMI ≥27 kg/m2) without diabetes. The SELECT trial demonstrated that Wegovy® statistically significantly reduced the risk of MACE by 20% compared to placebo when added to standard of care. In addition, the findings from SELECT showed that over a period of up to five years, risk reductions in MACE were achieved regardless of baseline age, sex, race, ethnicity, body mass index (BMI) and level of renal function impairment.

Arexvy, GSK’s Respiratory Syncytial Virus (RSV) Vaccine, Receives Positive European Medicines Agency CHMP Opinion for Adults Aged 50-59 at Increased Risk for RSV Disease

Warren-based BioNJ Member GSK announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the approval of GSK’s respiratory syncytial virus (RSV) vaccine for the prevention of lower respiratory tract disease (LRTD) caused by RSV from adults aged 60 and above to include adults aged 50-59 years at increased risk for RSV disease. This positive opinion is the first time that an indication for adults aged 50-59 has been recommended by CHMP for a RSV vaccine, one of the final steps prior to the extension of the marketing authorization by the European Commission. The European Commission’s final decision is expected by September 2024. Since June 2023, Arexvy (respiratory syncytial virus vaccine, recombinant adjuvanted) has been approved in Europe for adults aged 60 and over for the prevention of RSV-LRTD.  

GSK and Flagship Pioneering Partner to Discover Novel Medicines and Vaccines

Warren-based BioNJ Member GSK and Flagship Pioneering announced they have entered a collaboration with the goal of discovering and developing a portfolio of future transformational medicines and vaccines, starting in respiratory and immunology. This alliance brings together GSK’s disease area expertise and development capability with Flagship’s ecosystem of bioplatform companies, inclusive of its novel modalities and technologies, to make major advances in healthcare. GSK and Flagship will initially fund up to $150 million upfront to support an exploration phase to identify the most promising concepts for further research and development with Flagship’s bioplatform companies. From these explorations, the collaboration aims to identify a portfolio of up to 10 novel medicines and vaccines which will each be subject to an exclusive option by GSK for further clinical development.

U.S. FDA Expands Jemperli (Dostarlimab) Plus Chemotherapy Approval to All Adult Patients With Primary Advanced or Recurrent Endometrial Cancer as the First and Only Immuno-oncology-based Treatment to Show an Overall Survival Benefit

Warren-based BioNJ Member GSK announced the U.S. Food and Drug Administration (FDA) has approved Jemperli (dostarlimab) in combination with carboplatin and paclitaxel (chemotherapy) followed by Jemperli as a single agent for the treatment of adult patients with primary advanced or recurrent endometrial cancer. This approval broadens the previous indication for Jemperli plus chemotherapy to include patients with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumours who represent 70-75% of patients diagnosed with endometrial cancer and who have limited treatment options. The supplemental Biologics License Application (sBLA) supporting this expanded indication received Priority Review and was approved ahead of the Prescription Drug User Fee Act action date.

Otsuka Pharmaceutical to Acquire Jnana Therapeutics Inc.

Princeton-based BioNJ Member Otsuka Pharmaceutical and Jnana Therapeutics Inc. announced that they have entered into a definitive merger agreement pursuant to which Otsuka will acquire Jnana, making it a wholly owned subsidiary through Otsuka's 100-percent owned subsidiary, Otsuka America, Inc. (OAI). The acquisition is expected to be completed in the third quarter of fiscal 2024, subject to customary closing conditions. Based on the terms of the agreement, Otsuka will pay USD $800 million to the shareholders of Jnana upon completion of the acquisition, as well as up to an additional USD $325 million in development and regulatory milestones. Jnana's novel approach to drug discovery is enabled by RAPID - the company's next-generation chemoproteomics platform designed to discover medicines for highly validated but challenging-to-drug targets.

New Clinical Data Demonstrates Three Years of Continuous Treatment With Dual-Acting LEQEMBI® (lecanemab-irmb) Continues to Significantly Benefit Early Alzheimer’s Disease Patients Presented at AAIC 2024

Nutley-based BioNJ Member Eisai and BioNJ Member Biogen Inc. announced that the latest findings for lecanemab-irmb (U.S. brand name: LEQEMBI®), an anti-amyloid beta (Aβ) protofibril antibody for the treatment of early Alzheimer’s disease (AD), were presented at the Alzheimer’s Association International Conference. Dual-acting lecanemab is the only early AD treatment widely available to support neuronal function by clearing the highly toxic protofibrils that continue to cause neuronal injury and death even after plaques have been cleared from the brain. Clarity AD was a global Phase 3 placebo-controlled, double-blind, parallel-group, randomized study in 1,795 people with early AD (Lecanemab group: 10 mg/kg bi-weekly IV treatment: 898, placebo group: 897). 95% of patients who completed the core study (18 months) chose to continue in the open-label extension study (OLE). 

Citius Pharma’s $675M Merger Deal Advances

Cranford-based BioNJ Member Citius Pharmaceuticals Inc. and TenX Keane Acquisition announced they entered into a definitive agreement to merge TenX and Citius Pharma’s wholly owned oncology subsidiary. If approved, the newly combined public company will be called Citius Oncology Inc. Under the terms, Citius Pharma would receive 67.5 million shares in Citius Oncology at $10 per share and retain majority ownership of approximately 90%. Both companies’ boards of directors have approved the approximately $675 million deal, which is expected to close in the first half of 2024, pending approval by TenX stockholders and other customary closing conditions. Citius Oncology would focus on developing and commercializing novel targeted oncology therapies. Currently, Citius is seeking U.S. Food and Drug Administration approval of LYMPHIR for the treatment of persistent or recurrent cutaneous T-cell lymphoma, a rare form of non-Hodgkin lymphoma.

Evotec Announces Progress in Strategic Neuroscience Partnership With Bristol Myers Squibb

Princeton-based BioNJ Member Evotec SE announced progress within the company’s strategic partnership with BioNJ Member Bristol Myers Squibb, further bolstering the joint pipeline of advanced neuroscience programs. Evotec receives a US$ 25 m payment to progress further research. Evotec and Bristol Myers Squibb entered their strategic neuroscience collaboration in December 2016 to identify disease-modifying treatments for a broad range of neurodegenerative diseases where there remains a significant unmet medical need for therapies that slow down or reverse disease progression. The progress announced here leverages Evotec’s PanOmics platform in conjunction with patient-derived disease models, which is one of the largest and most sophisticated platforms in the industry. The partnership has generated a promising pipeline of discovery to clinical-stage programs.

AbbVie Announces European Commission Approval of SKYRIZI® (risankizumab) for the Treatment of Adults With Moderately to Severely Active Ulcerative Colitis

BioNJ Member AbbVie, with a site in Madison, announced that the European Commission has approved SKYRIZI® (risankizumab) for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy. "Ulcerative colitis is a chronic, unpredictable and sometimes debilitating disease, and people living with the condition need sustained symptom relief," said Edouard Louis, M.D., Ph.D., Professor and Head of Gastroenterology, Liège University Hospital, Dean of Faculty, Liège University, and INSPIRE trial investigator. "Patients treated with SKYRIZI in the INSPIRE and COMMAND clinical trials experienced significant improvements in clinical remission and mucosal healing. These are important findings as mucosal healing goes beyond symptom management to restoration of the intestinal lining and is associated with improved long-term outcomes.”

AbbVie Completes Acquisition of Cerevel Therapeutics

BioNJ Member AbbVie, with a site in Madison, announced that it has completed its acquisition of Cerevel Therapeutics. With the completion of the acquisition, Cerevel is now part of AbbVie. "AbbVie's acquisition of Cerevel strengthens our foundation in neuroscience and positions us to deliver sustainable long-term performance into the next decade and beyond," said Robert A. Michael, chief executive officer, AbbVie. "Our new Cerevel colleagues share our commitment to deliver meaningful change for patients living with neurological and psychiatric conditions. We are excited to welcome the talented Cerevel team to AbbVie." There are multiple programs in Cerevel's pipeline across several neurological and psychiatric conditions such as schizophrenia, Parkinson's disease and mood disorders, where there continues to be significant unmet need for patients. 

Chugai In-Licenses PI3K Inhibitor Inavolisib for Breast Cancer With a PIK3CA Mutation

BioNJ Member Chugai Pharmaceutical Co., with offices in Berkeley Heights, announced that it has concluded a license agreement with F. Hoffmann-La Roche Ltd for inavolisib, a PI3K alpha inhibitor, currently in development for advanced hormone receptor-positive, HER2-negative breast cancer with a PIK3CA mutation in combination with palbociclib and fulvestrant. Under the license agreement between Roche and Chugai, Chugai obtained exclusive rights for the development and marketing of inavolisib in Japan. Inavolisib was discovered by Genentech, a member of the Roche Group, and is currently under development for two global Phase III clinical studies in patients with locally advanced or metastatic HR-positive/HER2-negative breast cancer with PIK3CA mutations (INAVO120 and INAVO121) and one global Phase III clinical study in patients with PIK3CA mutated HER2-positive breast cancer (INAVO122).

CN201 is a Next Generation CD3xCD19 Bispecific Antibody that Augments and Diversifies Merck’s Pipeline, With Potential Applications in B-cell Malignancies and Autoimmune Diseases

Rahway-based BioNJ Member Merck & Co. and Curon Biopharmaceutical (Curon), a privately held biotechnology company, today announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, has agreed to acquire CN201, a novel investigational clinical-stage bispecific antibody for the treatment of B-cell associated diseases.

“We continue to identify opportunities to expand and diversify our pipeline,” said Dr. Dean Y. Li, president, Merck Research Laboratories. “Early clinical data have provided robust evidence for the potential of CN201 to target and deplete circulating and tissue B cells with the potential to treat a range of malignant and autoimmune diseases.” Under the terms of the agreement, Merck through a subsidiary will acquire full global rights to CN201 for an upfront payment of $700 million in cash. Curon is also eligible to receive up to $600 million in milestone payments associated with the development and regulatory approval of CN201.

Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) Plus Padcev® (enfortumab vedotin-ejfv) as First-Line Treatment for Patients With Unresectable or Metastatic Urothelial Carcinoma

Rahway-based BioNJ Member Merck & Co. announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with Padcev (enfortumab vedotin-ejfv), an antibody-drug conjugate, for the first-line treatment of adult patients with unresectable or metastatic urothelial carcinoma. The recommendation is based on results from the first interim analysis of the Phase 3 KEYNOTE-A39 trial (also known as EV-302), which was conducted in a research collaboration with Pfizer (previously Seagen) and Astellas, in which KEYTRUDA plus enfortumab vedotin demonstrated statistically significant and clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) versus platinum-based chemotherapy (gemcitabine plus cisplatin or carboplatin).

Daiichi Sankyo and Merck & Co., Inc., Rahway, NJ, USA Enter into Global Development and Commercialization Agreement for MK-6070

Basking Ridge-based Daiichi Sankyo and Rahway-based BioNJ Member Merck & Co. have expanded their existing global co-development and co-commercialization agreement for three investigational DXd antibody drug conjugates to include Merck’s MK-6070, an investigational delta-like ligand 3 (DLL3) targeting T-cell engager. The companies will jointly develop and commercialize MK-6070 worldwide, except in Japan where Merck will maintain exclusive rights. Merck will be solely responsible for manufacturing and supply for MK-6070. MK-6070 is a T-cell engager targeting DLL3, an inhibitory canonical Notch ligand that is expressed at high levels in small cell lung cancer (SCLC) and neuroendocrine tumors, currently being evaluated in a phase 1/2 clinical trial. The companies are planning to evaluate MK-6070 in combination with ifinatamab deruxtecan in certain patients with SCLC, as well as other potential combinations.

IDeate-Lung02 Phase 3 Trial of Ifinatamab Deruxtecan Initiated in Patients With Relapsed Small Cell Lung Cancer

Basking Ridge-based Daiichi Sankyo and Rahway-based BioNJ Member Merck & Co. announced that the first patient has been dosed in the IDeate-Lung02 Phase 3 trial evaluating the efficacy and safety of investigational ifinatamab deruxtecan (I-DXd) in patients with relapsed small cell lung cancer (SCLC) versus treatment of physician’s choice of chemotherapy. Ifinatamab deruxtecan is a specifically engineered potential first-in-class B7-H3 directed DXd antibody drug conjugate (ADC). Small cell lung cancer is the second most common type of lung cancer, accounting for about 15% of cases. SCLC is aggressive and progresses rapidly to the metastatic stage, which has a five-year survival rate of only 3%. Approximately 65% of all SCLC tumors have a moderate-to-high expression of the protein B7-H3, which is associated with disease progression and poor prognosis.

Calliditas Announces Positive TRANSFORM Phase 2b Topline Data in Primary Biliary Cholangitis

Hoboken-based Calliditas Therapeutics announced that the Phase 2b TRANSFORM trial met its primary endpoint, showing statistically significant improvement in ALP (Alkaline Phosphatase) for both doses tested versus placebo. The trial evaluated setanaxib, a NOX enzyme inhibitor, in patients with primary biliary cholangitis (PBC) and elevated liver stiffness. The TRANSFORM trial is a double-blind, randomized, placebo-controlled Phase 2b study investigating the effect of setanaxib 800 mg AM + 400 mg PM, (“1200 mg arm”) and 800 mg BID (“1600 mg arm”) over 24 weeks of treatment. The basis for the analysis consisted of a dataset of 76 patients with primary biliary cholangitis (PBC) and elevated liver stiffness. The treatment groups were relatively well-balanced with no clinically relevant differences between the groups observed at baseline. 

Calliditas Partner STADA Receives European Commission Decision for Full Approval of Kinpeygo® for the Treatment of IgA Nephropathy

Hoboken-based Calliditas Therapeutics announced that the European Commission has granted a full marketing authorization for Kinpeygo for the treatment of adults with primary immunoglobulin A nephropathy (IgAN). The European Commission has granted a full marketing authorization of Kinpeygo®. The granting of the full approval results in a significantly broader label for patients with primary IgAN, moving from a urine protein excretion (UPCR) limitation of > 1.5g/g to encompassing the entire study population, defined as UPCR of ≥ 0.8g/g, or proteinuria of ≥1.0 g/g over 24 hours. This expanded label is based on full two-year data set from the Phase 3 NefIgArd clinical trial, published in leading medical journal The Lancet..

Ipsen and Day One Enter Into Exclusive ex-U.S. Licensing Agreement to Commercialize Tovorafenib for the Most Common Childhood Brain Tumor

Ipsen, with a site in Basking Ridge, and Day One Biopharmaceuticals announced a new global partnership outside the U.S. for tovorafenib, an oral, once-weekly, type II RAF inhibitor for pediatric low-grade glioma (pLGG), the most common form of childhood brain cancer, and any future indications developed by Day One. Tovorafenib was granted Orphan Drug Designation and received U.S. FDA approval in April 2024 as a monotherapy treatment for patients six months and older with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. These BRAF alterations account for more than half of pLGG cases worldwide and there are no approved targeted treatments for people with pLGG harboring BRAF fusions outside the U.S. Day One will maintain exclusive global development and U.S. commercial rights for tovorafenib.

Up to $500M in AI Tax Credits Available Via Next NJ Program

The Garden State took its latest step in establishing itself as a leader in the artificial intelligence space, as Gov. Phil Murphy signed legislation into law establishing the Next New Jersey Program. The initiative aims to attract new investment into the State’s AI industry, providing up to $500 million in tax credits for AI businesses. The incentives were originally allocated for the Aspire and Emerge programs. Assembly Bill 4558/Senate Bill 3432 was passed alongside the Fiscal Year 2025 state budget as that process came down the home stretch. “We’re here today to sign a piece of legislation that will empower our State’s top minds to shape the future of generative artificial intelligence – both ambitiously and responsibly,” said Gov. Murphy. “Because in a moment when the future of this realm has yet to be written, we here in New Jersey can be one of its prime authors."

DePuy Synthes Launches its First Active Spine Robotics and Navigation Platform

Johnson & Johnson MedTech* announced that DePuy Synthes, The Orthopaedics Company of Johnson & Johnson, is launching a proprietary dual-use robotics and standalone navigation platform developed in collaboration with eCential Robotics. The system, called the VELYS™ Active Robotic-Assisted System (VELYS™ SPINE), received 510(k) clearance from the U.S. Food and Drug Administration (FDA) and is intended for use in planning and instrumenting spinal fusion procedures in the cervical, thoracolumbar and sacroiliac spine. This new spine technology was designed to help surgeons tackle their most complex challenges. The VELYS™ SPINE system is dual use, meaning it features both a standalone navigation and an active robotics platform that can enable surgeon flexibility in their approach and plans. Active robotics allows for surgical guidance tailored to surgeon preference.

BD, Quest Diagnostics Join Forces to Develop Companion Diagnostics for Cancer, Other Diseases

Franklin Lakes-based BioNJ Member BD and Secaucus-based Quest Diagnostics said they have collaborated to develop, manufacture and commercialize flow cytometry-based companion diagnostics intended to help select the best treatment for patients with cancer and other diseases. Together, BD and Quest will aim to provide the pharmaceutical industry with an end-to-end solution for CDx development. The joint offerings will range from exploratory panel development to the manufacturing and distribution of Food and Drug Administration-approved diagnostic kits. Using companion diagnostics to help select a first-line therapy for cancer patients can be critical to ensuring the best outcomes and cost savings. Today’s companion diagnostic tests commonly involve technologies such as immunohistochemistry, fluorescence in situ hybridization, polymerase chain reaction, next-generation sequencing and imaging.

NJIT Renames College of Science and Liberal Arts, Honoring Historic Gift from Alumnus Jordan Hu

New Jersey Institute of Technology has announced the renaming of its College of Science and Liberal Arts to the Jordan Hu College of Science and Liberal Arts (JHCSLA) in recognition of a historic gift from Jordan Hu ’89, founder and chief executive officer of RiskVal Financial Solutions, LLC and a former member of the university’s Board of Trustees. Mr. Hu’s contribution will enhance STEM education and research across campus, and open a range of scholarship opportunities for students, both domestic and international. “This renaming celebrates his remarkable and exemplary investment in the foundation of this university, which will create new avenues for expanded education and research opportunities, profoundly impacting the lives and careers of NJIT students for many years to come,” said NJIT President Teik C. Lim

NJIT Appoints William Brady as Vice President of Human Resources

New Jersey Institute of Technology (NJIT) has named William Brady as the new Vice President of Human Resources, effective Oct. 1, 2024. Mr. Brady brings more than two decades of extensive experience in HR leadership, strategic planning and workforce development to NJIT. Mr. Brady has had a distinguished career in human resources, previously serving as the Chief Human Resources Officer at both Boise State University and the University of Massachusetts Amherst. In these roles, he has modernized the Human Resources offices to position them as strategic, data-informed partners to campus. He has experience making significant improvements in talent acquisition, employee engagement, training and development, compensation and data analytics.

Cooper Innovation Center Researchers Collaborate to Create Revolutionary New Technology to Study Heart and Vascular Function

Dr. Lawrence Mulligan, Director of Research in the Department of Anesthesiology at Cooper and Associate Professor at Cooper Medical School of Rowan University, in collaboration with Jeffrey Hill, of the Massachusetts College of Pharmacy and Health Sciences, have developed a patent-pending technology called Myocardial Work Quantification. Their creation has the potential to usher in a new era in cardiovascular research. This noninvasive technology merges cardiac ultrasound images and blood pressure in a way that could revolutionize the field. Using a special blood pressure device that is worn on the arm, Myocardial Work Quantification simultaneously captures blood pressure and two-dimensional cardiac ultrasound images to create a pressure-strain loop. The technology provides insights into the heart’s ability to contract against the aorta before a patient becomes symptomatic and their disease advances.

ROI Influencers: People of Color 2024 — A to Z

The 2024 ROI-NJ Influencers: People of Color list celebrates the diversity of our State and our world-class workforce. More than anything, it demonstrably shows that we are better when we come together — an ideal that’s never been more important to promote and highlight.

Congratulations to this year’s honorees, BioNJ, Pavita Howe; Hackensack Meridian Health, Dr. Noelle Aikman, Dr. Will Carroll, Donald Ferrell, Srikanth Jaikumar, David Kountz, Liz Paskas, Avonia Richardson Miller, and Dr. Lisa Tank; Inspira Health, Robin Walton; Johnson & Johnson, Natalya Johnson; Kean University, Felice Vazquez; N.J. Business & Industry Association, Althea D. Ford; NJEDA, Ram Akella, Michelle Bodden, Tai Cooper; New Jersey Institute of Technology, Angela Garretson, Teik Lim; Rowan University, Joe Cardona; Rutgers University, Lyneir Richardson; Withum, Marcus Dyer.

Pawlus, Perry to Join Kenvue Board

Kenvue, the Johnson & Johnson spinoff that is the world’s largest pure-play consumer health company by revenue, announced this week that it has appointed Kathleen Pawlus and Kirk Perry as new independent directors. Ms. Pawlus brings more than 40 years of audit, finance, strategy, mergers & acquisitions, quality, information technology and administration experience to Kenvue, having previously served as a partner at Ernst & Young, and chief financial officer and chief operating officer of its global assurance group. Mr. Perry currently serves as CEO of Circana, a global provider of technology, data and predictive analytics for the consumer, retail and media sectors. He is a seasoned consumer products and brand strategist, with more than 30 years of consumer-packaged goods and technology experience at two of the world’s largest and most respected companies, Procter & Gamble and Google.

NJEDA to Open Applications for Green Workforce Training Grant Challenge

The Green Workforce Training Grant Challenge is a competitive funding opportunity that will award grants to implement workforce development training programs that bolster the green economy talent pipeline in the state with a particular focus on NJ’s Overburdened Communities. A total of $7 million is available for programs under this Grant Challenge, including $2.65 million from the Wind Institute for Innovation and Training and $4.35 million through the Governor’s Council on the Green Economy. The minimum and maximum amounts for individual awards are set at $250,000 and $1.5 million respectively. NJEDA anticipates making multiple awards in this grant challenge.

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 fill out this registration form for each person attending. We look forward to seeing you there!

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.