Welcome to the latest edition of the BioLines Weekender...
Last week, a bill that would increase the investment incentives under New Jersey’s Angel Investor Tax Credit from 20% to 35%, along with an increase to 40% for investments in companies located in Opportunity Zones throughout the State, was reported by the Assembly Science, Innovation and Technology Committee (A2365). BioNJ is thrilled to see this prudent policy make progress and will continue to support its approval by the Legislature and ultimate signature by the Governor.
Meanwhile, heartfelt congratulations to BioNJ Member PTC Therapeutics on the recent decision by the European Commission (EC) to overturn the CHMP's negative opinion of January 24, 2024, regarding the annual renewal of the conditional marketing authorization for Translarna™ (ataluren). The EC has sent the opinion back to the CHMP for re-evaluation. Consequently, Translarna remains on the market and available for Patients in Europe under its current authorization. This development marks a significant victory for boys and young men with Duchenne muscular dystrophy and exemplifies an unprecedented partnership between Patients and the industry. Their collective efforts have demonstrated the formidable power of the Patient's voice in influencing regulatory decisions.
Attending the BIO Convention? Make sure to visit us in the New Jersey Pavilion and join us at New Jersey's BIO International Reception taking place Monday, June 3, from 5:00-7:00 p.m. at the Nason’s Beer Hall, located in the Gaslamp Quarter, just a few blocks from the San Diego Convention Center. Click here for details and to RSVP. Hope to see you there!
Because Patients Can't Wait®,
Debbie Hart
President and CEO
BioNJ
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Enjoy this recorded webinar brought to you
by the BioNJ Finance & Business Operations Committee, "Capital Markets Update /
State of the Economy".
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Putting Patients First:
The Value of Medical Innovation -
Recent Stories in the News
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Inflation Reduction Act's Dirty Little Secret: Largest Premium Increase Ever for Medicare Drug Benefit
When President Biden signed the Inflation Reduction Act in 2022, he promised it would lower Medicare costs for America's seniors. Loaded with the all-too-familiar political rhetoric of "taking it to drug companies"— what is missing from this talking point is how the President "took it to seniors." Thus far, however, seniors' costs have skyrocketed. A new report reveals that for those with a Medicare Part D drug plan, this year's premiums have increased by a crushing 21%, on average. Next year's prices are expected to be even higher. The IRA is unraveling the "Part D" drug benefit — and hitting seniors with bills that few can afford. It's high time to review what went wrong.
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Making Licensing Harder Doesn’t Boost U.S. Manufacturing
While it’s appropriate to lament the lack of bipartisan cooperation in Washington, just because something’s bipartisan doesn’t mean it’s a good idea. Exhibit A could be Senator Tammy Baldwin (D-WI) and Senator J.D. Vance’s (R-OH) “Invent It Here, Make It Here” bill. Despite the name and its good intentions, it condemns promising federally funded inventions to waste away without doing a thing to build our domestic manufacturing base. The Senators seek to address a serious national problem — too many times it’s not possible to find a domestic manufacturer to make a product incorporating a federally funded invention. Unfortunately, the Baldwin/ Vance proposal makes the problem worse by defining domestic manufacturing in a way that makes it even harder while introducing more bureaucracy into the process.
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The Bayh-Dole Coalition Explained
For 44 years, the Bayh-Dole Act has been a cornerstone of America's innovation economy - which is why our coalition of universities, industry groups and more is dedicated to protecting and preserving it. Watch their new video to learn more about their mission.
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Why BioNJ’s BioPartnering Event Was Master Class in Networking — and Sector-Building
It was easy to count the number of floors at the perfectly suited Liberty Science Center that were used during the 14th annual BioPartnering Conference: three. The same is true for counting the number of attendees at the BioNJ-run event — and where they were from: approximately 630 from 22 states and nine countries. And the number of prefunding and early stage companies making pitches: 90 — or about 20% higher than the year before. Here’s what is impossible to quantify: the number of substantial conversations between founders, investors, top life science companies and academic leaders that could lead to breakthrough partnerships that help enhance the already-strong sector in the state. That’s what made the BioPartnering Conference a master class in networking.
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At BioPartnering Event, it’s Easy to Find Cheerleaders for (and Critics of) State
Dean Petkanas is the CEO of pre-revenue Neuropathix, a Doylestown, Pennsylvania-based company, one of 90 that came to BioNJ’s BioPartnering Conference looking to make connections. And, if he’s looking for another job, he appears to be a prime candidate to be a spokesperson/cheerleader for the life sciences sector in New Jersey. “New Jersey is the center and capital of pharmaceutical biotech in the United States,” he said. “It always has been. I know that Silicon Valley and Cambridge have gotten a lot of attention, but, if you’re going anywhere as a startup in the pharmaceutical space, New Jersey’s the state to go to.” It’s where Neuropathix, a small molecule drug discovery company focused on inflammation, which currently is based in the Pennsylvania Biotechnology Center, may end up, he said.
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PTC Therapeutics Announces European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
South Plainfield-based BioNJ Member PTC Therapeutics announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the annual renewal of the conditional marketing authorization of Translarna™ (ataluren) and has returned the opinion to the CHMP for re-evaluation. As a result, Translarna remains on the market and available for patients in Europe consistent with its current marketing authorization. The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion. "The efficacy and safety demonstrated in clinical trials and the long-term STRIDE registry support that Translarna fills an otherwise unmet need for nmDMD patients. We look forward to working collaboratively with CHMP on next steps once they are defined," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics.
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PTC Therapeutics Announces FDA Acceptance and Priority Review of the BLA for Upstaza™
South Plainfield-based BioNJ Member PTC Therapeutics announced that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. The application has been granted Priority Review with a target regulatory action date of November 13, 2024. Upstaza is a one-time gene replacement therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L–amino acid decarboxylase (AADC) deficiency with a severe phenotype. It is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene. It is designed to correct the underlying genetic defect, by delivering a functioning DDC gene directly into the putamen, increasing the AADC enzyme and restoring dopamine production.
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Insmed Presents Positive Patient-Reported Outcomes and Microbiologic Data from Phase 3 ARISE Study Of ARIKAYCE® (Amikacin Liposome Inhalation Suspension) in Patients With NTM Lung Disease Caused by MAC at American Thoracic Society 2024 International Conference Plenary Session
Bridgewater-based BioNJ Member Insmed Incorporated announced that late-breaking data from the ARISE study of ARIKAYCE® (amikacin liposome inhalation suspension) were presented at the American Thoracic Society (ATS) 2024 International Conference. As previously announced, data from ARISE demonstrated that the Quality of Life-Bronchiectasis (QOL-B) respiratory domain may be an effective patient-reported outcome (PRO) tool in patients with MAC lung disease. The ARISE study was designed to help support the validation of a PRO tool to be used in ENCORE, the ongoing Phase 3b registrational study evaluating the efficacy and safety of an ARIKAYCE-based regimen in patients with newly diagnosed or recurrent MAC lung disease who have not received antibiotics for their current infection. In ARISE, ARIKAYCE-treated patients performed better than those in the comparator arm (a macrolide-based multi-drug regimen) as measured by the QOL-B instrument, with 43.8% of patients achieving an improvement in QOL-B respiratory score above or equal to the estimated meaningful within-subject score difference of 14.8, compared with 33.3% of patients in the comparator arm.
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Ascendis Pharma Announces Extension of U.S. Food and Drug Administration Review Period for TransCon™ PTH for Adults With Hypoparathyroidism
BioNJ Member Ascendis Pharma, with a site in Princeton, announced that the U.S. Food and Drug Administration (FDA) notified the Company that information submitted in response to the FDA’s ongoing review of the New Drug Application (NDA) for TransCon PTH (palopegteriparatide) for adults with hypoparathyroidism constituted a major amendment to the NDA. Accordingly, the FDA has extended the PDUFA target action date by three months, to August 14, 2024, to provide time for a full review of the submission. “Adults with hypoparathyroidism in the United States, who are receiving TransCon PTH in our clinical trials and our Expanded Access Program (EAP) will continue to receive their medication, and the EAP remains open for enrollment for eligible patients,” said Jan Mikkelsen, Ascendis Pharma’s President and CEO.
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Significant and Sustained Improvements in Renal Function Reported for Adults With Chronic Hypoparathyroidism Treated With TransCon™ PTH (Palopegteriparatide): 2-year Results from Phase 3 PaTHway Trial
BioNJ Member Ascendis Pharma, with a site in Princeton, announced 2-year results from a post-hoc analysis of the Company’s Phase 3 PaTHway Trial demonstrating significant and sustained improvements in renal function in adults with chronic hypoparathyroidism treated with TransCon PTH (palopegteriparatide). The post-hoc analysis examined the impact of treatment with TransCon PTH on renal function using estimated glomerular filtration rate (eGFR) through Week 104 (n=76) of PaTHway, a Phase 3 double-blind, placebo-controlled trial of 82 dosed adults with chronic hypoparathyroidism randomized 3:1 (TransCon PTH:placebo; both arms initially co-administered with conventional therapy of active vitamin D and oral calcium), with a 26-week blinded period followed by an ongoing 156-week open-label extension period.
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Positive Clinical Results from HyBryte™ Compatibility Study in the Treatment of Cutaneous T-Cell Lymphoma Published in JEADV Clinical Practice
Princeton-based BioNJ Member Soligenix announced that the results of its compatibility study evaluating HyBryte™ (synthetic hypericin) for the treatment of cutaneous T-cell lymphoma (CTCL) have been published in the Journal of the European Academy of Dermatology & Venereology (JEADV) Clinical Practice. The publication highlights the positive clinical results from study, HPN-CTCL-02, evaluating HyBryte™ in the treatment of CTCL. The open-label study enrolled 9 patients to receive 8 weeks of HyBryte™ treatment of their cancerous lesions, with an assessment of treatment response conducted at week 10 using the modified Composite Assessment of Index Lesion Severity (mCAILS) score. The treatment response results of 22% following 8 weeks of twice weekly HyBryte™ therapy reinforces and confirms the results of the Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) trial, despite the fact that patients in Study HPN-CTCL-02 were specifically selected to have more extensive disease consistent with its potential commercial use.
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Celldex Announces First Patient Dosed in Phase 2 Study of Barzolvolimab in Prurigo Nodularis
Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced that the first patient has been dosed in the company’s Phase 2 subcutaneous study of barzolvolimab in prurigo nodularis (PN). 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 the function and survival of the mast cell. Mast cells are believed to play an important role in amplifying chronic itch and neuroinflammation, including in PN where mast cells are associated with pruritic sensory neurons in PN lesions. Celldex presented first in class data from the Company’s Phase 1b study in PN demonstrating that barzolvolimab and its novel mast cell depleting mechanism could play a meaningful role in breaking the stubborn scratch/itch cycle of this disease, resulting in lesion healing.
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Palatin Announces FDA Clearance of IND Application for the Co-Administration of Bremelanotide With Tirzepatide (GLP-1) for the Treatment of Obesity
Cranbury-based BioNJ member, Palatin Technologies, Inc. announced that the U.S. Food and Drug Administration (FDA) has completed its 30-day review of the investigational new drug (IND) application for the use of bremelanotide, a melanocortin receptor 4 agonist (MCR4), for the treatment of obesity. The company is cleared to begin enrollment in a Phase 2 clinical study evaluating the safety and efficacy of bremelanotide, co-administered with tirzepatide (GLP1/GIP) in obese patients. The Phase 2 clinical study is expected to start mid-calendar year 2024, with topline data results by the end of calendar year 2024. The clinical study, “A Phase II, Randomized, Double-Blind, Placebo-Controlled, Clinical Study Investigating the Safety, Tolerability, and Effectiveness of the Co-Administration of Bremelanotide with Tirzepatide (GLP-1/GIP) for the Treatment of Obesity” has been reviewed by FDA with an approval to proceed under Palatin’s IND.
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Johnson & Johnson to Acquire Proteologix, Inc. to Lead in Atopic Dermatitis Treatment
New Brunswick-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that it has entered into a definitive agreement to acquire Proteologix, Inc., a privately held biotechnology company focused on bispecific antibodies for immune-mediated diseases, for $850 million in cash, with potential for an additional milestone payment. Proteologix’s portfolio includes PX128, a bispecific antibody targeting IL-13 plus TSLP, which is ready to enter phase 1 development for moderate to severe atopic dermatitis (AD) and moderate to severe asthma, and PX130, a bispecific antibody targeting IL-13 plus IL-22, which is in preclinical development for moderate to severe AD. Since AD and asthma are both heterogeneous diseases with different disease-driving pathways in distinct patient subpopulations, targeting multiple pathways offers the potential to deliver high-bar efficacy and remission.
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TAR-210 Results Show 90% Recurrence-Free Survival and 90% Complete Response in Patients With High-Risk and Intermediate-Risk Non–Muscle-Invasive Bladder Cancer, Respectively
New Brunswick-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced updated results from an open-label, multicenter, multi-cohort Phase 1 study of the safety and efficacy of TAR-210, an intravesical targeted releasing system designed to provide sustained, local release of erdafitinib into the bladder, in patients with non–muscle-invasive bladder cancer (NMIBC) with select FGFR alterations. Results featured updated data from Cohort 1 (C1), patients with recurrent, Bacillus Calmette-Guérin (BCG)–unresponsive high-risk (HR) NMIBC (high-grade Ta/T1; papillary only) who refused or were ineligible for radical cystectomy and Cohort 3 (C3), patients with recurrent, intermediate-risk NMIBC (Ta/T1) low-grade papillary disease left in situ as tumor marker lesions. At the data cutoff of March 22, 2024, 64 patients had been treated with TAR-210 across the 2 cohorts. Of the 21 patients in C1 with HR-NMIBC, the 12-month recurrence-free (RF) survival rate was 90%. In C3, 31 patients were efficacy evaluable with a complete response (CR) rate of 90%.
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TREMFYA® (guselkumab) QUASAR Maintenance Study in UC Met its Primary Endpoint and All Major Secondary Endpoints, Including Highly Statistically Significant Rates of Endoscopic Remission
New Brunswick-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced the first data from the Phase 3 QUASAR Maintenance Study that showed 50.0 percent (p<0.001) of patients with moderately to severely active ulcerative colitis (UC) receiving subcutaneous (SC) TREMFYA® (guselkumab) 200 mg every four weeks (q4w) and 45.2 percent (p<0.001) of patients receiving SC TREMFYA® 100 mg every eight weeks (q8w) achieved the primary endpoint of clinical remission at Week 44 compared to placebo (18.9 percent). In additional analyses of patients who were in clinical remission, 67 percent, and 71 percent, respectively, were also in endoscopic remission at Week 44 (Mayo endoscopic subscore [MES]= 0), indicating they had normal appearance of intestinal mucosa.
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TREMFYA® (guselkumab) Demonstrates Superiority Versus STELARA® (ustekinumab) in Phase 3 Crohn’s Disease Program
New Brunswick-based BioNJ Member Johnson & Johnson today announced the first Phase 3 results for TREMFYA® (guselkumab) in adult patients with moderately to severely active Crohn’s disease (CD), which demonstrated superiority of both subcutaneous (SC) maintenance doses (200 mg every 4 weeks [q4w] and 100 mg every 8 weeks [q8w]) versus placebo and ustekinumab.1 Data showed that both maintenance doses of TREMFYA® met the composite co-primary endpoints compared to placebo in each individual study. In results versus ustekinumab, both doses of TREMFYA® demonstrated statistically significant and clinically meaningful differences on all prespecified pooled endoscopic endpoints. The GALAXI 2 (n=508) and GALAXI 3 (n=513) studies were the first-ever double-blind registrational head-to-head clinical trials to demonstrate superiority versus ustekinumab in CD.
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With Once-a-Week Dosing, Insulin Efsitora Alfa Delivers A1C Reduction and Safety Profile Consistent With Daily Insulin
BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced positive topline results from the QWINT-2 and QWINT-4 phase 3 clinical trials evaluating once-weekly insulin efsitora alfa (efsitora) in adults with type 2 diabetes using insulin for the first time (insulin naïve) and those who require multiple daily insulin injections. In the treat-to-target clinical trials, efsitora showed non-inferior A1C reduction compared to the most commonly used daily basal insulins globally. QWINT-2 evaluated the efficacy and safety of once-weekly efsitora compared to once-daily insulin degludec for 52 weeks. The trial randomized insulin-naïve adults with type 2 diabetes to receive efsitora once weekly or insulin degludec once daily and was also designed to assess efficacy in patients using and not using GLP-1 receptor agonists.
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More than One-half of Patients With Crohn's Disease Treated With Lilly's Mirikizumab Achieved Clinical Remission at One Year, Including Patients With Previous Biologic Failure
BioNJ Member Eli Lilly and Company's pivotal Phase 3 VIVID-1 study, patients with moderately to severely active Crohn's disease, with or without previous biologic failure, achieved statistically significant and clinically meaningful improvements across multiple clinical and endoscopic endpoints at one year with mirikizumab compared to placebo. "Crohn's disease is a complex condition that, if untreated, may result in irreversible damage to the digestive tract. Mirikizumab patients achieved high rates of combined clinical remission and endoscopic response, two important treatment targets that are difficult to achieve in the same patient, at one year. This is particularly impressive for patients with previous biologic failure who are generally considered hard-to-treat," said Bruce Sands, M.D., M.S., Dr. Burrill B. Crohn Professor of Medicine and Chief of the Dr. Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai.
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Antiepileptic Drug FYCOMPA® Approved in China for Adjunctive Treatment of Primary Generalaized Tonic-Clonic Seizures
Nutley-based BioNJ Member Eisai announced that it has received approval in China for the additional indication of its in-house discovered antiepileptic drug (AED) Fycompa® (generic name: perampanel hydrate) for adjunctive treatment of primary generalized tonic-clonic seizures in patients with epilepsy aged 12 years and older. Fycompa is a first-in-class AED discovered at Eisai’s Tsukuba Research Laboratories. The agent is a selective, noncompetitive AMPA receptor antagonist that is postulated to reduce neuronal hyper-excitation associated with seizures by targeting glutamate activity at AMPA receptors on postsynaptic membranes. In China, Fycompa was approved for the adjunctive treatment of partial-onset seizures (with or without secondarily generalized seizures) in patients with epilepsy aged 12 years and older in September 2019.
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Novo Nordisk A/S: Once-weekly and Once-Monthly Mim8 Demonstrate Superior Reduction of Treated Bleeding Episodes Compared to On-Demand and Prior Prophylaxis Treatment in People With Haemophilia A in the Frontier 2 Trial
Plainsboro-based BioNJ Member Novo Nordisk announced the headline results from the FRONTIER 2 trial, a pivotal phase 3a, 26-week open-label, randomized, controlled, multi-arm trial in 254 people. The trial investigated the efficacy and safety of once-weekly and once-monthly subcutaneous Mim8 versus no prophylaxis and versus prior coagulation factor prophylaxis treatment in people aged 12 years or older with haemophilia A with or without inhibitors. The trial achieved its co-primary endpoints by demonstrating a statistically significant and superior reduction of treated bleeding episodes with both once-weekly and once-monthly Mim8 versus no prophylaxis treatment and prior coagulation factor prophylaxis treatment. In people with no prior prophylaxis treatment, once-weekly and once-monthly Mim8 demonstrated superior reductions of 97% and 99% in treated bleeds, respectively, compared to those who received no prophylaxis treatment.
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Novo Nordisk Announce Research Collaboration With Flagship Pioneering and Metaphore Biotechnologies to Develop Next-Generation Therapeutics for Obesity Management
Plainsboro-based BioNJ Member Novo Nordisk announced a research collaboration with Metaphore Biotechnologies and Flagship Pioneering to develop up to two next-generation therapeutics for obesity management. The collaboration is signed under the broader strategic partnership between Novo Nordisk and Flagship Pioneering to develop a portfolio of novel treatment approaches for cardiometabolic and rare diseases. Pioneering Medicines, Flagship's in-house drug development and partnerships unit, is responsible for leading the broader partnership together with Novo Nordisk's Bio Innovation Hub, an R&D unit designed to accelerate the development of therapeutics via co-creation partnerships. Metaphore will work jointly with Pioneering Medicines and Novo Nordisk to advance the programs through foundational activities and preclinical development, after which point Novo Nordisk could advance the programs into clinical studies.
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Dupixent® (dupilumab) sBLA Accepted for FDA Priority Review for Treatment of Adolescents With Chronic Rhinosinusitis With Nasal Polyposis (CRSwNP)
Bridgewater-based BioNJ Member Sanofi and BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) as an add-on maintenance treatment for adolescents aged 12 to 17 years with inadequately controlled chronic rhinosinusitis with nasal polyposis (CRSwNP). The target action date for the FDA decision is September 15, 2024. Dupixent is currently approved as an add-on maintenance treatment for adults with CRSwNP whose disease is not adequately controlled. The sBLA in adolescents is supported by an extrapolation of efficacy data from two positive pivotal trials (SINUS-24 and SINUS-52) in adults with CRSwNP.
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Dupixent® Late-Breaking Data from NOTUS Confirmatory Phase 3 COPD Study Presented at ATS and Published in NEJM
Bridgewater-based BioNJ Member Sanofi announced late-breaking data from the NOTUS Phase 3 study evaluating the investigational use of Dupixent® (dupilumab) as an add-on maintenance treatment in adults with uncontrolled COPD on maximal standard-of-care inhaled therapy (nearly all on triple therapy) and evidence of type 2 inflammation (i.e., blood eosinophils ≥300 cells per μL). The NOTUS study confirmed the positive results demonstrated in the landmark Phase 3 BOREAS study. The NOTUS study met its primary and key secondary endpoints. All patients were on background maximal standard-of-care inhaled therapy (nearly all on triple therapy). The safety results were generally consistent with the known safety profile of Dupixent in its approved indications.
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Beyfortus Real-World Evidence Published in The Lancet Shows 82% Reduction in Infant RSV Hospitalizations
Bridgewater-based BioNJ Member Sanofi announced Beyfortus reduced respiratory syncytial virus (RSV) hospitalizations by 82% (95% CI: 65.6 to 90.2) in infants under 6 months of age, compared to infants who received no RSV intervention, according to the interim results of an ongoing study published in The Lancet. These results, from the first RSV season after Beyfortus’ introduction, are part of the three-year NIRSE-GAL study conducted in Galicia, Spain under a collaborative framework with the Galician Directorate of Public Health of the Xunta de Galicia (Galician government) and Sanofi. The results echo real-world evidence (RWE) reported from several broad infant immunization programs across the US, Spain and France during the 2023-2024 RSV season, which add to the consistent and high efficacy seen in pivotal clinical studies with Beyfortus.
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Sanofi and Novavax Announce Co-Exclusive Licensing Agreement to Co-Commercialize COVID-19 Vaccine and Develop Novel Flu-COVID-19 Combination Vaccines
As part of Bridgewater-based BioNJ Member Sanofi’s commitment to developing a diverse portfolio of best-in-class vaccines, the company has entered into a co-exclusive licensing agreement with Novavax. The terms of the agreement include: a co-exclusive license to co-commercialize Novavax’s current stand-alone adjuvanted COVID-19 vaccine worldwide (except in countries with existing Advance Purchase Agreements and in India, Japan and South Korea where Novavax has existing partnership agreements); a sole license to Novavax’s adjuvanted COVID-19 vaccine for use in combination with Sanofi’s flu vaccines; and a non-exclusive license to use the Matrix-M adjuvant in vaccine products. In addition, Sanofi will take a minority (<5%) equity investment in Novavax.
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European Medicines Agency Validates Bristol Myers Squibb’s Application for Opdivo (nivolumab) Plus Yervoy (ipilimumab) for the First-Line Treatment of Adult Patients With Microsatellite
Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Medicines Agency (EMA) has validated its Type II variation application for Opdivo® (nivolumab) plus Yervoy ® (ipilimumab) for the first-line treatment of adult patients with microsatellite instability–high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC). The EMA’s validation of the application confirms the submission is complete and begins the EMA’s centralized review process. The submission is based on results from the CheckMate -8HW study, in which Opdivo plus Yervoy demonstrated a statistically significant and clinically meaningful improvement in the dual primary endpoint of progression-free survival (PFS) compared to investigator’s choice of chemotherapy as assessed by Blinded Independent Clinical Review (BICR) for the first-line treatment of patients with MSI-H/dMMR mCRC.
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U.S. Food and Drug Administration Accepts Bristol Myers Squibb’s Application for Subcutaneous Nivolumab (nivolumab and hyaluronidase)
Princeton-based BioNJ Member Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for the subcutaneous formulation of Opdivo® (nivolumab) co-formulated with Halozyme’s proprietary recombinant human hyaluronidase (rHuPH20) (herein referred to as “ subcutaneous nivolumab”) across all previously approved adult, solid tumor Opdivo indications as monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of February 28, 2025. “We believe subcutaneous nivolumab has the potential to make a significant difference in the lives of patients, which is reinforced by the FDA’s acceptance of our application,” said Gina Fusaro, Ph.D., Vice President, Global Program Lead, Bristol Myers Squibb.
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New Four-Year Sotyktu (deucravacitinib) Data Demonstrate Durable Response Rates and Consistent Safety in Moderate-to-Severe Plaque Psoriasis
Princeton-based BioNJ Member Bristol Myers Squibb announced new four-year results from the POETYK PSO long-term extension (LTE) trial of Sotyktu (deucravacitinib) treatment in adult patients with moderate-to-severe plaque psoriasis. After four years of continuous treatment, Week 208 responses for Psoriasis Area and Severity Index (PASI) 75 and 90 were 71.7% and 47.5%, respectively, and 57.2% for static Physician’s Global Assessment (sPGA) 0/1 (clear/almost clear), using modified nonresponder imputation (mNRI). The safety profile of Sotyktu at Year 4 remained consistent with the established safety profile, with no new safety signals identified. The efficacy analysis included 513 patients who received continuous Sotyktu treatment from Day 1 in the pivotal POETYK PSO-1 and POETYK PSO-2 trials and transitioned to the POETYK PSO-LTE trial.
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Bristol Myers Squibb’s CAR T Cell Therapy Breyanzi Approved by the U.S. Food and Drug Administration for Relapsed or Refractory Follicular Lymphoma
Princeton-based BioNJ Member Bristol Myers Squibb announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Breyanzi ® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). Breyanzi is also now included in the National Comprehensive Cancer Network (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for B-cell Lymphomas as a Category 2A recommendation for third line and subsequent therapy for relapsed or refractory FL.
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Merck Provides Update on Phase 3 KeyVibe-010 Trial Evaluating an Investigational Coformulation of Vibostolimab and Pembrolizumab as Adjuvant Treatment for Patients With Resected High-Risk Melanoma
Rahway-based BioNJ Member Merck & Co. announced the discontinuation of the vibostolimab and pembrolizumab coformulation arm of the Phase 3 KeyVibe-010 trial. The trial is evaluating the investigational coformulation of vibostolimab, an anti-TIGIT antibody, and pembrolizumab (KEYTRUDA®), Merck’s anti-PD-1 therapy, compared to KEYTRUDA alone, as adjuvant treatment for patients with resected high-risk melanoma (Stage IIB-IV). At a pre-planned analysis, data showed that the primary endpoint of recurrence-free survival (RFS) met the pre-specified futility criteria. A higher rate of discontinuation of all adjuvant therapy by patients in the coformulation arm versus the KEYTRUDA-only arm, primarily due to immune-mediated adverse experiences, rendered it highly unlikely that the trial could achieve a statistically significant improvement in RFS.
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Ferring Announces Results Demonstrating the Efficacy and Safety of Follitropin Delta for Women Undergoing In Vitro Fertilization Using an Agonist Protocol
Parsippany-based BioNJ Member Ferring Pharmaceuticals announced that Human Reproduction published results from the BEYOND trial. This is the first trial to compare individualized follitropin delta dosing using a GnRH agonist versus a GnRH antagonist protocol in women aged 18-40 years, with anti-Müllerian hormone (AMH) levels below 35 pmol/L who are undergoing their first ovarian stimulation cycle for IVF/ICSI. Results from this randomized, controlled, open-label, multicentre trial showed that a statistically significant mean difference of 1.3 extra oocytes (95% CI: 0.22; 2.40, p=0.0185) were retrieved for women using the GnRH agonist protocol versus women using antagonist protocol. Use of both GnRH agonist and antagonist protocols yielded similar results in safety. The results supported a European Medicines Agency (EMA) SmPC label update to include information on the use of follitropin delta using the GnRH agonist protocol.
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GSK Announces Positive Results from Phase III Severe Asthma Trials of Depemokimab
Warren-based BioNJ Member GSK announced positive headline results from the phase III clinical trials SWIFT-1 and SWIFT-2, which assessed the efficacy and safety of depemokimab versus placebo in adults and adolescents with severe asthma with type 2 inflammation characterised by blood eosinophil count. Both SWIFT-1 and SWIFT-2 met their primary endpoints of a reduction in the annualised rate of clinically significant exacerbations (asthma attacks) over 52 weeks. Across both trials the overall incidence and severity of treatment-emergent adverse events were similar in patients treated with either depemokimab or placebo. Further analysis of these data is ongoing. Depemokimab is the first ultra-long-acting biologic to be evaluated in phase III trials with a binding affinity and high potency for interleukin-5 (IL-5), enabling six-month dosing intervals for patients with severe asthma. IL-5 is known to be a key cytokine (protein) in type 2 inflammation.
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GSK to Become a Founding Partner of Fleming Initiative to Fight Antimicrobial Resistance (AMR)
Warren-based BioNJ Member GSK announced it will become the first founding partner of the Fleming Initiative, an innovative and collaborative approach led by Imperial College Healthcare NHS Trust and Imperial College London to tackle antimicrobial resistance (AMR) around the world. AMR is widely recognized, including by the World Health Organization, as an urgent global public health threat. In 2019 there were an estimated 1.2 million deaths directly attributable to drug-resistant bacterial infections and as many as 10 million people could die annually from AMR by 2050, resulting in global economic damage of up to $100 trillion by 2050 without effective action. The Fleming Initiative’s unique and transformative approach will bring together world-class scientific, technology, policy and behavioural science expertise with clinical experience in a global network of centres to find, test and scale solutions to AMR.
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Investigational Seladelpar Demonstrates Significant Improvements in Liver Disease Progression and Reduced Itching in Primary Biliary Cholangitis
BioNJ Member Gilead Sciences, with a site in Morris Plains, following the recent acquisition of CymaBay Therapeutics, Inc., announced interim results from the ongoing ASSURE study demonstrating treatment with seladelpar, an investigational PPAR delta agonist, led to improvements in markers of cholestasis and reduced inflammation. Additional findings demonstrate that seladelpar can help reduce pruritus (itch) in people living with primary biliary cholangitis (PBC). There are currently no treatments indicated to treat PBC-related pruritis. ASSURE is an open-label study evaluating the long-term safety and efficacy of seladelpar, a once daily potent and selective peroxisome proliferator-activated receptor (PPAR) delta agonist, or delpar. ASSURE enrolled adult patients with PBC who previously participated in a study of seladelpar where a key eligibility criterion included having an inadequate response or intolerance to ursodeoxycholic acid (UDCA).
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AbbVie and Gilgamesh Pharmaceuticals Announce Collaboration and Option-to-License Agreement to Develop Next-Generation Therapies for Psychiatric Disorders
BioNJ Member AbbVie, with a site in Madison, and Gilgamesh Pharmaceuticals announced a collaboration and option-to-license agreement to develop next-generation therapies for psychiatric disorders. This collaboration will leverage AbbVie's expertise in psychiatry and Gilgamesh's innovative research platform to discover novel neuroplastogens. Classic psychedelic compounds provide novel mechanisms to address mental health disorders, and some have shown promising clinical efficacy where other treatments have been ineffective. However, these first-generation compounds may induce profound psychoactive effects, such as hallucinations, necessitating in-office administration and concomitant supportive care. Next-generation versions known as neuroplastogens target mechanisms that have shown potential to provide significant clinical benefits and are designed to minimize the challenging effects seen with first-generation compounds.
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Regeneron Pharmaceuticals Takes 126K Square Feet at Warren Corporate Center
BioNJ Member Regeneron Pharmaceuticals, Inc., with a site in Basking Ridge, announced it has signed a lease to take 126,678 square feet at Warren Corporate Center. The deal means the campus is more than 95% leased. The Warren Corporate Center is surrounded by walking trails, a landscaped pond, four parking structures and a lifestyle center called Warren Hill. The Warren Hill facility is over 20,000 square feet. Additionally, PTC Therapeutics leased 360,000 square feet at Warren Corporate Center in 2022.
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Sandoz Receives European Commission Approval for Wyost® and Jubbonti®, the First and Only Biosimilars of Denosumab in Europe
Princeton-based BioNJ Member Sandoz announced that the European Commission (EC) has granted marketing authorization for Wyost® (denosumab) and Jubbonti® (denosumab), the first and only biosimilar versions of reference medicines Xgeva® and Prolia® in Europe. Wyost® is approved for the treatment of cancer-related bone disease. Jubbonti® is approved to treat osteoporosis. These are key biosimilar value drivers for the company over the mid-term and their approval is a major step in advancing the Sandoz growth strategy. We expect to launch from November 2025 onwards. Close to one quarter (4.2 million) of all newly reported cancer cases globally occur in Europe and cancer is ranked as a leading cause of premature death among those 30-69 years of age in most European countries
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Teva and Medincell Announce Positive Phase 3 Efficacy Results from SOLARIS Trial Evaluating TEV-‘749 (olanzapine) as a Once-Monthly Subcutaneous Long-Acting Injectable in Adults With Schizophrenia
Parsippany-based BioNJ Member Teva Pharmaceutical and Medincell announced results from the efficacy portion of the Phase 3 Subcutaneous OLAnzapine extended-Release Injection Study (SOLARIS) trial evaluating TEV-‘749 in adult patients with schizophrenia compared to placebo. Results demonstrated that TEV-‘749 met its primary endpoint as measured by a change in the PANSS total score from baseline after 8 weeks compared to placebo. TEV-‘749 utilizes SteadyTeq™, a copolymer technology proprietary to Medincell that provides a controlled steady release of olanzapine, the most prescribed 2nd generation antipsychotic for schizophrenia in the U.S. TEV-‘749 met its primary endpoint across all three dosing groups, with mean difference in change in the Positive and Negative Syndrome Scale (PANSS) total score from baseline to week 8 of -9.71 points, -11.27 points, and -9.71 points versus placebo for the high, medium, and low dose groups, respectively.
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UroGen Announces Results from ATLAS Showing Robust UGN-102 Durability of Response in New and Recurrent Low-Grade Intermediate-Risk Non-Muscle Invasive Bladder Cancer at AUA 2024
Princeton-based UroGen Pharma Ltd. announced results from a new analysis of the ATLAS trial, which estimates using Kaplan Meier methods the probabilities of remaining in complete response for both new and recurrent low-grade intermediate-risk non- muscle invasive bladder cancer (LG-IR-NMIBC) patients following treatment with investigational drug UGN-102 as primary therapy, with or without subsequent transurethral resection of the bladder tumor (TURBT) at 3 months. "These compelling findings shed light on the potential of UGN-102 as a nonsurgical primary treatment for low-grade intermediate-risk bladder cancer," said William Huang, M.D., Urologic Oncologist, Professor and Vice Chair of Urology, NYU Grossman School of Medicine. "These data are an encouraging step forward in addressing the broad spectrum of LG-IR-NMIBC and potentially curbing the high rates of disease recurrence associated with it."
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Shionogi & Co., Ltd. and Maze Therapeutics, Inc. Announce Exclusive Worldwide License Agreement for MZE001, a Novel Therapeutic Candidate for the Treatment of Pompe Disease
Shionogi & Co., Ltd., with a site in Florham Park, and Maze Therapeutics, Inc. announced the companies have completed an exclusive worldwide license agreement for the rights to MZE001, an investigational oral glycogen synthase 1 (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup. Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which can lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise. Under the terms of the agreement, Shionogi has acquired exclusive worldwide rights for MZE001 as well as related programs and intellectual property. MZE001 is a small molecule and specific inhibitor of GYS1, an enzyme involved in glycogen synthesis.
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42 Small Business Loans Totaling Over $3M Provided Under NJEDA’s NJ Capital Access Fund
The New Jersey Economic Development Authority (NJEDA) announced that more than $3.6 million in working capital loans have been approved under the NJ Capital Access Fund, since the program launched in November 2023. Administered by designated fund manager Calvert Impact, the $100 million fund, supported by the State Small Business Credit Initiative (SSBCI) program and a private capital match, leverages Community Development Financial Institutions (CDFIs) and Minority Deposit Institutions (MDIs) to offer technical assistance and an affordable and flexible working capital loan product to assist small businesses and nonprofits with payroll, rent, utilities and other operational business expenses.
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TAXIS Pharmaceuticals Awarded $2.67M NIH Grant for R&D on Combating Antibiotic-Resistant Pneumonia
North Brunswick-based TAXIS Pharmaceuticals received a $2.67 million grant, to be awarded over three years, from National Institute of Allergy and Infectious Disease, one of the institutes of the National Institutes of Health. The funding will allow TAXIS Pharmaceuticals to complete preclinical research on the first of its investigational efflux pump inhibitors — compounds that are intended to inhibit the bacterial cellular processes that cause antibiotic resistance, enabling the antibiotics to be effective in fighting off the bacteria they target. The focus will combine the company’s EPI compound with the FDA-approved antibiotic levofloxacin, commonly used to treat bronchitis and sinus infections. This approach aims to bolster the antibiotic’s efficacy against resistant bacterial strains.
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Sanofi, Formation Bio and OpenAI Announce First-in-Class AI Collaboration
Bridgewater-based BioNJ Member Sanofi, Formation Bio and OpenAI are collaborating to build AI-powered software to accelerate drug development and bring new medicines to patients more efficiently. The three teams will bring together data, software and tuned models to develop custom, purpose-built solutions across the drug development lifecycle. This represents a first collaboration of its kind within the pharma and life sciences industries. Sanofi will leverage this partnership to provide access to proprietary data to develop AI models as it continues on its path to becoming the first biopharma company powered by AI at scale. “This unique collaboration is the next significant step in our journey to becoming a pharmaceutical company substantially powered by AI. Next generation, first-of-its kind AI model customizations will be an important foundation in our efforts to shape the future of drug development for pharma and for the many patients waiting for innovative treatments,” said Paul Hudson, CEO, Sanofi.
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FDA Clears Curio’s Postpartum Depression PDT
Curio Digital Therapeutics Inc.’s MamaLife Plus offers a nonpharmaceutical alternative to treating postpartum depression. Last month, it became the first prescription digital therapeutic (PDT) cleared by the U.S. Food and Drug Administration for PPD. Princeton-based Curio announced the milestone April 23. MamaLift is intended for neurobehavioral interventions to patients 22 years or older. Curio said it is an adjunct to clinician-managed outpatient care. The eight-week tool treats mild to moderate postpartum depression by improving a patient’s symptoms, according to the company.
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Academia/Institutions/Incubators | |
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NJEDA to Open Applications for the Next Cohort of the First-in-Nation Innovation Fellows Program
The New Jersey Economic Development Authority (NJEDA) opened applications for the next cohort of the New Jersey Innovation Fellows (NJIF) Program, and is currently accepting questions from potential applicants. The NJIF Program supports entrepreneurs with income replacement grants, mentorship and training to provide security in the early stages of start-up business creation. Through the NJIF Program, the NJEDA supports teams of at least three entrepreneurs with mentorship, training and income replacement capital of up to $400,000 over a two-year period, attracting innovative ideas and entrepreneurs who would otherwise be unable to pursue the launch of a new venture due to income needs or socio-economic constraints.
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Rutgers Institute Awarded $39.7M NIH Grant to Continue Clinical and Translational Science Program
The National Institutes of Health recently awarded New Brunswick-based BioNJ Member Rutgers Institute for Translational Medicine and Science more than $39.67 million over seven years to build and improve upon infrastructure that promotes clinical and translational science through the New Jersey Alliance for Clinical and Translational Science. The funding will promote translational science, which uses discoveries made in the laboratory, clinic and community to create interventions that improve the health of individuals and populations — from diagnostics and therapeutics to medical procedures and behavioral health interventions. The funding will enable NJ ACTS to expand and improve its statewide program that was created in 2019 to quickly translate research discoveries into patient care.
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New Jersey Innovation Institute Signs On to SciTech Scity
The highly anticipated SciTech Scity project at Liberty Science Center in Jersey City marked another major development, as the New Jersey Innovation Institute (NJII) joined the Healthcare Innovation Engine as a Lead Innovation Partner. NJII, a New Jersey Institute of Technology (NJIT) corporation and nonprofit headquartered in Newark, joins other notable partners signed onto the project touted as the “Science City of Tomorrow.” Those participants include RWJ Barnabas Health, Bristol Myers Squibb, EY, Nokia Bell Labs and Sheba Medical Center in Israel.
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PPPL Breaks Ground on $100M Innovative Fusion Research Hub
During a groundbreaking ceremony, officials put golden shovels in the ground to mark the forthcoming Princeton Plasma Innovation Center (PPIC) project. The more than $100 million effort will rise at the Princeton Plasma Physics Laboratory (PPPL) headquarters, located on Princeton University’s Forrestal Campus in Plainsboro. PPPL uses plasma to solve some of the world’s toughest science and technology challenges. It is a Department of Energy (DOE) National Laboratory that Princeton University manages.
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Tevogen Bio Appoints William Keane as VP, Strategic Operations
Warren-based BioNJ Member Tevogen Bio announced Warren’s former Chief of Police, William Keane, as Vice President of Strategic Operations. Mr. Keane brings 35 years of leadership experience in law enforcement and has held various management positions throughout his career. Mr. Keane’s accolades include being the recipient of the Department Medal of Honor, the Somerset County 200 Club Valor Award, and numerous departmental commendations during his distinguished career. He is a graduate of the FBI Law Enforcement Executive Development Seminar in Leadership held at Princeton University and the prestigious FBI National Academy in Quantico, Virginia, where he received advanced specialized training in executive management.
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Pfizer Announces New Chief Strategy and Innovation Officer
BioNJ Member Pfizer, with a site in Peapack, announced that Andrew Baum, M.D., will join the company as Chief Strategy and Innovation Officer, Executive Vice President. Dr. Baum will be a member of Pfizer’s Executive Leadership Team reporting to Chairman and Chief Executive Officer, Dr. Albert Bourla. He joins Pfizer from Citi, where he served as Head of Global Healthcare, Managing Director Equity Research. Prior to joining Citi in 2011, Dr. Baum covered European Pharmaceuticals at Morgan Stanley for 14 years and earlier in his career was a practicing physician at the Royal National Orthopaedic Radcliffe Hospital in Oxford where he completed his residency.
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PDS Biotech Appoints Stephan Toutain as Chief Operating Officer
Princeton-based PDS Biotechnology Corporation announced the appointment of Stephan Toutain, MS, MBA, as Chief Operating Officer. Mr. Toutain brings more than 30 years of operational experience in the pharmaceutical industry from drug development, general management, operations, commercial development, market access and sales and marketing leadership with prior global expertise in the oncology and orphan drugs markets. Before joining PDS Biotech, he served as COO at Anavex Life Sciences from May 2018 to April 2024 and Chief Commercial Officer at Interleukin Genetics from July 2016 to August 2017. Mr. Toutain also worked with Alnylam Pharmaceuticals to build its early access program.
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Octapharma USA Appoints Former Pfizer Exec as VP of Commercial Marketing & Development
Paramus-based Octapharma USA announced the appointment of pharmaceutical industry veteran Kelly Hearn as Vice President of Commercial Development & Marketing. She assumes immediate leadership of the product marketing team as the company continues to grow its presence in the U.S. market. During more than 20 years with Pfizer, Ms. Hearn has demonstrated impressive abilities to drive strategic expansion. Ms. Hearn most recently served as Pfizer’s Senior Director of Commercial Development, where she drove pipeline strategy for infectious and rare diseases. During more than two decades with Pfizer, Ms. Hearn has led a variety of franchises both in the U.S. and globally, and most notably played an integral role in building the first channel and customer-centric hospital business unit for Pfizer.
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ESG Best Practices: Tools to Learn From | |
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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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Propelus I-Corps Regional Program
May 31 & June 14 – Princeton University
The NSF I-Corps Hub Northeast Hub is accepting applications for a two-day, in-person educational program for tech-based entrepreneurs. The two-day intensive educational program, to be held May 31 and June 14 at Princeton University, is aimed at helping entrepreneurs gain a better understanding of the startup process. It is intended to help researchers develop early stage innovations into real-world products and services through training, funding, resources and follow-on opportunities.
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Deal Making and Structuring Early Stage Technology and Life Sciences Deals
June 12 – LES NY/NJ Chapter
A panel discussion on the challenges and opportunities associated with technology transfer and licensing from universities to corporates and between emerging companies and more established companies. This program is for University tech transfer professionals, early stage life sciences and technology companies, late-stage companies that recently acquired or in licensed IP originated at early stage companies.
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