The March-In Drug Price Control Narrative Crumbles While Its Damage to American Innovation Grows

It was little more than a month ago when the Biden Administration unleashed its draft guidelines for applying the March-In provisions of the Bayh-Dole Act. For more than 43 years, the law was implemented as written. Every Administration — including the Biden Administration — rejected repeated attempts to misuse the law so the government could license copiers when critics felt that a product based on a federally-funded invention was too expensive. This was mainly sought under the guise of lowering drug prices. Even though the Administration issued a stinging denial of the most recent attempt last March, in December it reversed course. Although Congress hadn’t amended Bayh-Dole, the guidelines say agencies can use march in rights to impose “reasonable pricing” on federally funded inventions.

The Biden-Harris Administration Announces New Guidance to Enable Expanded Access to All FDA-Approved Contraceptives Without Cost

Agile Therapeutics, Inc., a women's healthcare company, commends the Biden-Harris Administration for their continued commitment to reproductive health including increasing access to no-cost contraception. Following several months of focus by the Biden Administration and lawmakers on contraception and impermissible barriers to reproductive healthcare, the Departments of Labor, Health and Human Services and the Treasury (the “Tri-Agencies”) jointly released new guidance in the form of Frequently Asked Questions. The FAQ outlines a “new pathway for plans and issuers to meet existing obligations under federal law by covering, at no cost, a broader range of FDA-approved contraceptive drugs and certain devices.”

Proposed IRA Expansion Would Cripple Biopharma, Lead to Fewer Drugs: Report

The proposed expansion to the Inflation Reduction Act would substantially harm U.S. biopharma, triggering hundreds of thousands of job cuts across the industry and leading to fewer innovative treatments in critical therapeutic areas, according to a report by healthcare consultancy Vital Transformation. The 34-page report makes the case that the proposed Inflation Reduction Act (IRA) expansion would eliminate around 136,000 to 216,000 jobs in the biopharma industry and lead to an additional 678,000 to 1,076,000 indirect layoffs across the U.S. economy. The expansion would also result in around 134 fewer FDA approvals over a 10-year period, which would most heavily affect “many areas of unmet need,” such as oncology, neurology, rare diseases and infectious diseases in the elderly population, the report contends.

Biden’s Got Pharma’s Back in Global Pandemic Treaty Negotiations

The Biden administration and the pharmaceutical industry are battling over drug prices in Washington and courthouses around the country. But in the international arena, they’re united in an effort to block developing countries from access to U.S. firms’ patented information on vaccines and drugs when the next pandemic hits. U.S. negotiators working on a global treaty that aims to guide the world’s response whenever a new deadly pathogen emerges have rejected proposals to loosen patent protections, a step that could enable developing countries to quickly make their own versions of the vaccines and drugs.

AstraZeneca Case Leads Off Busy Year of Challenges to Medicare Drug Pricing Law

A federal court in Delaware heard arguments in a key lawsuit challenging Medicare drug-negotiation provisions of the Inflation Reduction Act (IRA). Reports from inside the courtroom suggested that the judge was skeptical of the arguments raised by plaintiff AstraZeneca, though experts told BioSpace this is just an early step in what could be a protracted series of legal battles. Next week, the Centers for Medicare and Medicaid Services (CMS) is due to release its proposed maximum fair price for the initial set of 10 single-source drugs chosen for negotiation, after which time, drug companies will have 30 days to respond...Other plaintiffs include Merck, Novartis, Johnson & Johnson, Bristol Myers Squibb, Boehringer Ingelheim, Novo Nordisk and the lobbying group Pharmaceutical Research and Manufacturers of America (PhRMA)...

“Whether or not they would ultimately succeed I think largely will depend on whether or not the industry can get some more traction on its argument that compliance with this regime and program really is not voluntary,” said John Bennett, a litigator at Allen & Overy who works with biopharma clients.

Debbie Hart, President and CEO of BioNJ, a life sciences trade association in New Jersey, said in an email that she agrees with the claim that the IRA is essentially forcing drugmakers to bend to the will of the government rather than engage in true negotiation. “As a result, life sciences companies are in a position to ultimately accept a stipulated price or withdraw from the Medicare and Medicaid programs entirely, effectively barring all of the patients under those programs from access to those medications.” 

Cellares Cuts the Ribbon on Bridgewater Smart Factory Facility

During a ribbon cutting for the BioNJ Member Cellares' new Somerset County location, Cellares CEO and co-founder Fabian Gerlinghaus described the facility’s magnitude. “Bridgewater is such an important site for us,” he said. “It’s our flagship IDMO (Integrated Development and Manufacturing Organization) Smart Factory.” Cellares previously secured a $255 million Series C funding round, which is going toward this new state-of-the-art Smart Factory at 95 Corporate Drive in Bridgewater. Formerly occupied by Pfizer, the 118,000-square-foot facility represents the world’s first IDMO dedicated to clinical and industrial-scale cell therapy manufacturing. The company expects to create 350 new jobs at the facility. The site will be powered by robotics and software and capable of producing more than 40,000 cell therapy batches a year. The location utilizes state-of-the-art Cell Shuttles, a modular platform for industrial scale cell therapy manufacturing.

Ferring Announces Full Availability of ADSTILADRIN® (nadofaragene firadenovec-vncg) in the U.S.

Parsippany-based BioNJ Member Ferring Pharmaceuticals announced that ADSTILADRIN® (nadofaragene firadenovec-vncg) is now fully available across the U.S. for healthcare providers to prescribe for their adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Approved by the U.S. Food & Drug Administration (FDA) in December 2022, ADSTILADRIN is the first and only FDA-approved intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy. Ferring also initiated a non-interventional study, known as the “ADSTILADRIN in Bladder CancEr” (ABLE-41) U.S. Real World Evidence (RWE) Study (NCT06026332). This ongoing study is exploring early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting.

Novartis Lutathera® Significantly Reduced Risk of Disease Progression or Death by 72% as First-Line Treatment for Patients With Advanced Gastroenteropancreatic Neuroendocrine Tumors

East Hanover-based BioNJ Member Novartis presented data from the Phase III NETTER-2 trial showing that Lutathera® (lutetium Lu 177 dotatate) plus long-acting release (LAR) octreotide reduced the risk of disease progression or death by 72% as first-line therapy in patients with somatostatin receptor-positive (SSTR+) well-differentiated grade 2/3 advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs) versus high-dose octreotide LAR alone. No new or unexpected safety findings were observed in the study and data are consistent with the already well-established safety profile of Lutathera. Most patients (88%) in the Lutathera arm received all four cycles of Lutathera treatment. The most common all-grade AEs (≥20%) for the Lutathera arm vs. control arm were nausea (27.2% vs 17.8%), diarrhea (25.9% vs 34.2%) and abdominal pain (17.7% vs 27.4%), and the most common grade ≥3 AE (>5%) was lymphocyte count decreased (5.4% vs 0%).

European Medicines Agency Validates Type II Variation Application for PADCEV® (enfortumab vedotin) With KEYTRUDA® (pembrolizumab) for First-Line Treatment of Advanced Bladder Cancer

BioNJ Member Pfizer, with a site in Peapack, and Astellas Pharma Inc. announced that the European Medicines Agency (EMA) validated for review a Type II variation application for PADCEV® (enfortumab vedotin) with KEYTRUDA® (pembrolizumab) as a combination therapy for the first-line treatment of adult patients with previously untreated locally advanced or metastatic urothelial cancer (la/mUC). If approved, PADCEV with KEYTRUDA has the potential to change the treatment paradigm, becoming the first combination treatment to offer an alternative to platinum-containing chemotherapy, the current standard of care in first-line la/mUC. Globally, approximately 573,000 new cases of bladder cancer and 212,000 deaths are reported annually. It is estimated that approximately 200,000 people in Europe are diagnosed with bladder cancer each year.

European Commission Authorizes GSK’s Omjjara (momelotinib)

Warren-based BioNJ Member GSK announced the European Commission granted marketing authorization for Omjjara (momelotinib), a once-a-day, oral JAK1/JAK2 and activin A receptor type 1 (ACVR1) inhibitor. Omjjara is the first authorized medicine in the EU for disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. Nina Mojas, Senior Vice President, Oncology Global Product Strategy, GSK, said, “Until now, there have been no options specifically indicated to treat these symptoms in patients who also experience anaemia. The authorization of Omjjara brings a new treatment option with a differentiated mechanism of action to these patients in the EU.”

GSK’s RSV Vaccine, Arexvy, Accepted for Regulatory Review by the European Medicines Agency for the Prevention of RSV disease in Adults Aged 50-59 at Increased Risk

Warren-based BioNJ Member GSK announced that the European Medicines Agency (EMA) has accepted the company’s regulatory application to expand the use of its adjuvanted recombinant respiratory syncytial virus (RSV) vaccine to adults aged 50-59 who are at increased risk for RSV disease. If accepted, GSK’s RSV vaccine would be the first vaccine available to help protect this population. Arexvy is currently approved in Europe in adults aged 60 and over for the prevention of lower respiratory tract disease (LRTD) caused by RSV. This regulatory submission follows the positive results from a phase III trial [NCT05590403] ⁴ evaluating the immune response and safety of GSK’s RSV vaccine in adults aged 50-59, including those at increased risk for RSV-LRTD due to underlying medical conditions. 

Dupixent® FDA Approved as First and Only Treatment Indicated for Children Aged 1 year and Older With Eosinophilic Esophagitis (EoE)

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) has approved Dupixent® (dupilumab) for the treatment of pediatric patients aged 1 to 11 years, weighing at least 15 kg, with eosinophilic esophagitis (EoE). Dupixent is now the first and only medicine approved in the U.S. specifically indicated to treat these patients. This approval expands the initial FDA approval for EoE in May 2022 for patients aged 12 years and older, weighing at least 40 kg. The FDA evaluated Dupixent for this expanded indication under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions.

Sanofi to Acquire Inhibrx, Inc., Adding Potential Best-in-Class Rare Disease Asset for Alpha-1 Antitrypsin Deficiency to Pipeline

Bridgewater-based BioNJ Member Sanofi and Inhibrx, Inc., have entered into a definitive agreement under which Sanofi has agreed to acquire Inhibrx following the spin-off of non-INBRX-101 assets into New Inhibrx. INBRX-101 is a human recombinant protein that holds the promise of allowing Alpha-1 Antitrypsin Deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent (monthly vs. weekly) dosing. AATD is an inherited rare disease characterized by low levels of AAT protein, predominantly affecting the lung with progressive deterioration of the tissue. INBRX-101 may help to reduce inflammation and prevent further deterioration of lung function in affected individuals.

Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases

BioNJ Member Kyowa Kirin Co., Ltd., with a site in Princeton, and Orchard Therapeutics announced they have successfully completed the acquisition of Orchard Therapeutics. Orchard Therapeutics’ portfolio comprises Libmeldy® (atidarsagene autotemcel), which is intended for the treatment of eligible patients with early onset metachromatic leukodystrophy (MLD), a rare and life-threatening inherited disease of the body’s metabolic system. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. Libmeldy is approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA). Libmeldy is known as OTL-200 in the U.S., where it is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA).

Sandoz Announces Agreement to Acquire CIMERLI® Business from Coherus, Strengthening Position in US Market

Princeton-based BioNJ Member Sandoz announced they have signed an agreement to acquire the US biosimilar ranibizumab CIMERLI®* (ranibizumab-eqrn) from Coherus BioSciences, Inc. for an upfront cash purchase payment of USD 170 million. This is inclusive of a biologics license application, product inventory, ophthalmology sales and field reimbursement talent, as well as access to proprietary commercial software. Keren Haruvi, President Sandoz North America, said, “I am pleased that we can add another high-value product to the growing Sandoz biosimilar portfolio, further strengthening our existing ophthalmology franchise. The addition of CIMERLI® reinforces our commitment to biosimilars and represents a huge step towards our goal of pioneering patient access to more affordable and much-needed medicines in the US.”

Sandoz Launches First and Only Biosimilar for Multiple Sclerosis, Tyruko® (natalizumab), in Germany

Princeton-based BioNJ Member Sandoz announced announces the launch of Tyruko® (natalizumab) in Germany. Developed by Polpharma Biologics, Tyruko® is the first and only biosimilar to treat RRMS. Tyruko® is indicated as a single disease-modifying therapy (DMT) in adults with highly active RRMS This is the same indication as approved by the European Commission for reference medicine Tysabri. Access to novel high efficacy DMTs remains restricted with only roughly 20% of people living with MS in Europe able to make use of these innovative treatments. This number is significantly lower in Eastern European countries, roughly 3% to 4%. This highlights that more must be done to ensure early and unrestricted access to these crucial medicines so that irreversible neurological damage and disease progression can be delayed.

Regulatory Applications Accepted in the U.S. and Japan for Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) in Relapsed or Refractory Follicular Lymphoma (FL) and Relapsed or Refractory Mantle Cell Lymphoma (MCL)

Princeton-based BioNJ Member Bristol Myers Squibb announced three regulatory acceptances from the U.S. Food and Drug Administration (FDA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for Breyanzi^® (lisocabtagene maraleucel). In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for Breyanzito expand into new indications to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) and relapsed or refractory mantle cell lymphoma (MCL) after a Bruton tyrosine kinase inhibitor (BTKi). The FDA has granted both applications Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 23, 2024 for Breyanzi in relapsed or refractory FL and May 31, 2024 for Breyanzi in relapsed or refractory MCL. Japan's MHLW has also accepted Bristol Myers Squibb’s supplemental New Drug Application (sNDA) for Breyanzi for the treatment of relapsed or refractory FL.

Opdivo (nivolumab) Plus Yervoy (ipilimumab) Reduced the Risk of Disease Progression or Death by 79% Versus Chemotherapy in Patients With Microsatellite Instability-High or Mismatch Repair Deficient Metastatic Colorectal Cancer in CheckMate -8HW Trial

Princeton-based BioNJ Member Bristol Myers Squibb announced results from the Phase 3 CheckMate -8HW trial evaluating Opdivo (nivolumab) plus Yervoy (ipilimumab) compared to investigator’s choice of chemotherapy (mFOLFOX-6 or FOLFIRI with or without bevacizumab or cetuximab) as a first-line treatment for patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC). The dual immunotherapy combination of Opdivo and Yervoy demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of progression-free survival (PFS) as assessed by Blinded Independent Central Review (BICR), with a reduction in the risk of disease progression or death by 79% (Hazard Ratio [HR]: 0.21; 95% Confidence Interval [CI]: 0.14-0.32; p<0.0001) compared to chemotherapy in patients with centrally confirmed MSI-H/dMMR mCRC.

Opdivo® (nivolumab) in Combination With CABOMETYX® (cabozantinib) Demonstrates Long-Term Survival Benefits After Four Years of Follow-Up in the CheckMate -9ER Trial in First-Line Advanced Renal Cell Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb and Exelixis, Inc. announced four-year follow-up results from the CheckMate -9ER trial evaluating Opdivo® (nivolumab) in combination with CABOMETYX® (cabozantinib) vs. sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). Results continued to show superior progression-free survival (PFS) and objective response rates (ORR) in patients treated with Opdivoplus CABOMETYX over sunitinib, regardless of risk classification based on International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) scores. Superior overall survival (OS) was also observed in patients treated with the combination. At a median follow-up of 55.6 months (48.1 months minimum), all patients randomized to the Opdivo plus CABOMETYX treatment arm (n=323) continued to experience benefits over those who received sunitinib (n=328) across efficacy endpoints.

Eight-Year Data for Opdivo (nivolumab) Plus Yervoy (ipilimumab) Continue to Demonstrate Longest Survival Benefit vs. Sunitinib Reported in Patients With Previously Untreated Advanced or Metastatic Renal Cell Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that Opdivo (nivolumab) plus Yervoy (ipilimumab) continued to demonstrate long-term survival results in the Phase 3 CheckMate -214 trial, reducing the risk of death by 28% in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC) vs. sunitinib after eight years, regardless of International Metastatic RCC Database Consortium (IMDC) risk group. Patients treated with Opdivo plus Yervoy maintained superior survival and more durable response benefits compared to those who received sunitinib in both patients with intermediate- and poor-risk prognostic factors and across all randomized patients. 

Bristol Myers Squibb Completes Acquisition of Mirati Therapeutics, Strengthening and Diversifying Oncology Portfolio

Princeton-based BioNJ Member Bristol Myers Squibb announced that it has successfully completed its acquisition of Mirati Therapeutics, Inc.®. With the completion of the acquisition, Mirati shares have ceased trading on the NASDAQ Global Select Market and Mirati is now a wholly owned subsidiary of Bristol Myers Squibb. “The closing of the Mirati transaction is a significant milestone in our efforts to further diversify our oncology portfolio and strengthen our pipeline in the latter half of the decade and beyond,” said Chris Boerner, Ph.D., Chief Executive Officer, Bristol Myers Squibb. “Mirati’s incredibly talented employees have built a strong portfolio of assets and capabilities that are highly complementary with BMS’.”

Subcutaneous Nivolumab (nivolumab and hyaluronidase) Shows Noninferiority Compared to Intravenous Opdivo (nivolumab) in Advanced or Metastatic Clear Cell Renal Cell Carcinoma in CheckMate -67T Trial

Princeton-based BioNJ Member Bristol Myers Squibb announced the first disclosure of data from the Phase 3 CheckMate -67T trial, evaluating the subcutaneous formulation of Opdivo (nivolumab) co-formulated with Halozyme’s proprietary recombinant human hyaluronidase (rHuPH20) (herein referred to as “subcutaneous nivolumab”) compared to intravenous (IV) Opdivo in patients with advanced or metastatic clear cell renal cell carcinoma (ccRCC) who have received prior systemic therapy, demonstrating noninferiority for the co-primary endpoints of Cavgd28 (time-averaged Opdivo serum concentration over 28 days) and Cminss (trough serum concentration at steady state) compared to IV Opdivo. In addition, subcutaneous nivolumab displayed noninferior objective response rate (ORR) as assessed by Blinded Independent Central Review (BICR) vs. IV Opdivo. 

Bristol Myers Squibb Receives Positive CHMP Opinion for CAR T Cell Therapy Abecma (idecabtagene vicleucel)in Earlier Lines of Therapy for Triple-Class Exposed Relapsed and Refractory Multiple Myeloma

Princeton-based BioNJ Member Bristol Myers Squibb announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization approval of Abecma® (idecabtagene vicleucel; ide-cel) for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an anti-CD38 monoclonal antibody. The CHMP adopted a positive opinion based on the final progression-free survival (PFS) analysis from the pivotal, Phase 3, open-label, global, randomized, controlled KarMMa-3 study evaluating Abecma compared with standard combination regimens in adults with relapsed and refractory multiple myeloma after two to four prior lines of therapy, including an IMiD, a PI and an anti-CD38 monoclonal antibody, which are the three main classes of therapy (triple-class exposed) in multiple myeloma, and who were refractory to their last regimen. 

Johnson & Johnson Submits Supplemental Biologics License Application to U.S. FDA Seeking Approval of DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj)-Based Regimen for the Treatment of Patients With Transplant-Eligible, Newly Diagnosed Multiple Myeloma

Raritan-based BioNJ Member Johnson & Johnson announced today the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) seeking approval of a new indication for DARZALEX FASPRO® (daratumumab and hyaluronidase-fihj) in combination with bortezomib, lenalidomide and dexamethasone (D-VRd) for induction and consolidation treatment and with lenalidomide (D-R) for maintenance treatment of adult patients who are newly diagnosed with multiple myeloma (NDMM) and are eligible for autologous stem cell transplant (ASCT). This submission is supported by data from the Phase 3 PERSEUS (NCT03710603) study evaluating D-VRd induction and consolidation therapy, ASCT, and D-R maintenance therapy, compared to bortezomib, lenalidomide and dexamethasone (VRd), ASCT and lenalidomide (R) maintenance. 

TREMFYA® (guselkumab) Demonstrates Significant and Rapid Scalp Psoriasis Clearance in People of Color in New Large Phase 3b Study

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that TREMFYA® (guselkumab) demonstrated rapid and significant clearance in moderate to severe scalp psoriasis (PsO) and significant improvement in scalp itch, as well as patient-reported health-related quality of life outcomes, including post-inflammatory pigmentation at 16 weeks. VISIBLE is the first prospective, large-scale, randomized-controlled trial dedicated to people of color across all skin tones with moderate to severe plaque PsO and scalp PsO to objectively measure clearance and other treatment outcomes with TREMFYA. The VISIBLE clinical program will produce an expansive longitudinal collection of more than 20,000 clinical images across all skin tones to support patients and providers with disease recognition and education. 

U.S. Food and Drug Administration Grants Full Approval for BALVERSA® to Treat Locally Advanced or Metastatic Bladder Cancer With Select Genetic Alterations

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) approved a supplemental New Drug Application (sNDA) for BALVERSA® (erdafitinib) for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC) with susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations whose disease has progressed on or after at least one line of prior systemic therapy. BALVERSA® is not recommended for the treatment of patients who are eligible for and have not received prior PD-1 or PD-L1 inhibitor therapy. This FDA action converts the April 2019 accelerated approval of BALVERSA® to a full approval based on the clinical and overall survival benefit observed in the Phase 3 THOR study. 

Merck’s KEYTRUDA® (pembrolizumab) Significantly Improved Disease-Free Survival (DFS) as Adjuvant Therapy Versus Observation in High-Risk Patients With Localized Muscle-Invasive and Locally Advanced Urothelial Carcinoma After Surgery

Rahway-based BioNJ Member Merck & Co. announced results from the Phase 3 AMBASSADOR (A031501)/KEYNOTE-123 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, for the adjuvant treatment of high-risk patients with localized muscle-invasive urothelial carcinoma (MIUC) and locally advanced resectable urothelial carcinoma. “In this trial, adjuvant pembrolizumab [KEYTRUDA] reduced the risk of disease recurrence or death from any cause by 31% versus observation, demonstrating the potential of using pembrolizumab [KEYTRUDA] after surgery for high-risk patients with persistent muscle-invasive or locally advanced urothelial carcinoma who have high tumor stage, lymph node involvement or positive margins at surgery to help prevent their cancer from returning,” said Dr. Andrea Apolo, principal investigator and chief of the Bladder Cancer Section, Genitourinary Malignancies Branch, National Cancer Institute (NCI).

Merck’s KEYTRUDA® (pembrolizumab) Reduced the Risk of Death by 38% Versus Placebo as Adjuvant Therapy for Patients With Renal Cell Carcinoma (RCC) at an Increased Risk of Recurrence Following Nephrectomy

Rahway-based BioNJ Member Merck & Co. announced results from the Phase 3 KEYNOTE-564 trial evaluating KEYTRUDA, Merck’s anti-PD-1 therapy, for the adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy or following nephrectomy and resection of metastatic lesions “For patients with renal cell carcinoma, up to 40% may experience recurrence following surgery, at which point there is a significantly lower chance of survival,” said Dr. Toni K. Choueiri, Director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute and Jerome and Nancy Kohlberg Professor of Medicine, Harvard Medical School. “Results from KEYNOTE-564 show that pembrolizumab as adjuvant therapy significantly improved overall survival by 38% compared to placebo, becoming the first ever Phase 3 adjuvant trial to show improved survival for renal cancer patients at risk of recurrence after surgery.”

Antiepileptic Drug FYCOMPA® Injection Formulation Approved in Japan

Nutley-based Eisai announced that it has obtained marketing authorization approval from the Japanese Ministry of Health, Labour and Welfare for the injection formulation of its in-house discovered antiepileptic drug (AED) Fycompa® (perampanel) in Japan as an alternative therapy when oral administration is temporarily not possible. Fycompa is a first-in-class AED discovered at Eisai’s Tsukuba Research Laboratories. The agent is a highly 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. Two oral formulations of Fycompa are available in Japan: a tablet and a fine granule formulation. 

Eisai Submits New Drug Application for Mecobalamin Ultra High-Dose Formulation in Japan for the Indication of Amyotrophic LateralSclerosis

Nutley-based Eisai announced that it has submitted a New Drug Application (NDA) for ultrahigh-dose mecobalamin (development code: E0302) for the indication of amyotrophic lateral sclerosis (ALS) to the Pharmaceuticals and Medical Devices Agency (PDMA) in Japan. In May 2022, ultrahigh-dose mecobalamin received orphan drug designation by the Ministry of Health, Labour and Welfare (MHLW). This application is based on the results of JETALS (The Japan Early Stage Trial of Ultrahigh-Dose Methylcobalamin for ALS), a Phase III trial to evaluate efficacy and safety of ultrahigh-dose methylcobalamin (mecobalamin) in early onset ALS patients. ALS is an intractable, progressive, neurodegenerative disease that results in severe muscle atrophy and weakness in the muscles due to motor neuron dysfunction.

Latest Phase III Trial Data Investigating Cabometyx® in Combination With Immunotherapy to be Presented at ASCO GU 2024

Basking Ridge-based Ipsen announced new data on Cabometyx® (cabozantinib) in combination with immunotherapy across indications. With a median follow-up of 14.3 months, data from the primary analysis of progression-free survival (PFS) from the CONTACT-02 trial demonstrated a statistically significant PFS benefit for the combination of Cabometyx and atezolizumab of 6.3 months versus 4.2 months for a second NHT (hazard ratio [HR]: 0.65, 95% confidence interval [CI]: 0.50-0.84; p=0.0007). At an interim analysis for the other primary endpoint of overall survival (OS), the data demonstrated a trend toward improvement for the combination, however, these data were immature, and the trial will continue to the next planned analysis, anticipated in 2024. 

TOXINS 2024: Results from Ipsen’s AboLiSh Study Demonstrate the Significant Clinical Benefit of Using Injection Guidance Techniques When Treating Spasticity With AbobotulinumtoxinA

Basking Ridge-based Ipsen announced top line results from its real-world AboLiSh study. The study evaluated utilization and effectiveness of Dysport® (abobotulinumtoxinA) in people living with lower-limb spasticity and found that injection guidance techniques significantly help to improve outcomes and goal attainment in patients. AboLiSh was a prospective 16-month observational study with a primary endpoint of goal attainment measured by subject centred Goal Attainment Scaling-Leg (LegA) T score. Topline results demonstrated statistically significant improvement in rehabilitation goal attainment in instances where physicians used guidance techniques, such as ultrasound, electrostimulation, electromyography or a combination of techniques, to deliver the first cycle of treatment to patients, compared to those receiving treatment without the use of guidance techniques. 

EZHARMIA® Supplemental New Drug Application Submitted in Japan for Patients With Peripheral T-Cell Lymphoma 

Basking Ridge-based Daiichi Sankyo has submitted a supplemental New Drug Application (sNDA) to Japan’s Ministry of Health, Labour and Welfare (MHLW) for EZHARMIA® (valemetostat tosilate), a first-in-class dual inhibitor of EZH1 and EZH2, for the treatment of adult patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). PTCL is a group of rare and aggressive blood cancers, which represent about 10 to 15% of all nonHodgkin lymphomas (NHL). A majority of patients with PTCL experience disease progression following initial treatment with a multi-drug chemotherapy-based regimen and median overall survival following relapse is approximately 5.8 months. The sNDA for EZHARMIA is based on VALENTINE-PTCL01 Phase 2 trial results. EZHARMIA had previously received SAKIGAKE designation from the Japan MHLW for treatment of patients with relapsed or refractory PTCL, and the sNDA represents the first submission for this potential indication globally.  

ENHERTU® Granted Priority Review in the U.S. for Patients with Metastatic HER2 Positive Solid Tumors

Basking Ridge-based Daiichi Sankyo and AstraZeneca's supplemental Biologics License Application for ENHERTU® (famtrastuzumab deruxtecan-nxki) has been accepted and granted Priority Review in the U.S. for the treatment of adult patients with unresectable or metastatic HER2 positive (immunohistochemistry [IHC] 3+) solid tumors who have received prior treatment or who have no satisfactory alternative treatment options. ENHERTU is a specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. The U.S. Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available treatment options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. 

Simtra BioPharma Solutions Moves HQ to Parsippany

Simtra BioPharma Solutions, a leading sterile injectable contract development and manufacturing organization, announced Wednesday that it is making Parsippany its new corporate home. The company has established its new headquarters at 400 Interpace Parkway. The expansion to Parsippany will serve as a communication and logistical hub, enabling day-to-day operations to run smoothly and efficiently. Centralizing its corporate operations in New Jersey will facilitate Simtra’s access to its growing global clientele and expand its recruiting reach. Simtra officials said the move strengthens Simtra’s global presence and solidifies its commitment to enhancing collaboration and service excellence within the pharmaceutical industry.

Rutgers Health Receives $3M Grant to Study Pregnancy and Children’s Health

A research team from Rutgers Health‘s multidisciplinary group of researchers recently received a $3.1 million grant from the National Institutes of Health to study the impact of environmental influences on pregnancy and children’s health. The research project is part of the NIH’s Environmental Influences on Child Health Outcomes, or ECHO, Program. The Rutgers team plans to recruit up to 500 pregnant participants who are less than 20 weeks from conception from RWJ University Hospital and St. Peter’s University Hospital, both in New Brunswick, into the ECHO cohort. The researchers will follow the participants, their partners and children for seven years, tracking factors such as obesity, neurodevelopment, exposure to environmental chemicals and positive health through biosamples and surveys. 

Cole Named Chief Innovation Officer for State

Dave Cole, a former senior adviser on innovation in President Barack Obama’s administration and a longtime member of the State’s Office of Innovation, was selected by Gov. Phil Murphy to be the State’s new chief innovation officer. Mr. Cole, who joined the Office of Innovation in 2020, will now lead it in its mission to drive innovation in the Garden State and improve the design and delivery of policies and services to the State’s residents, businesses and institutions. He succeeds Beth Noveck, who Murphy recently announced will become the State’s first chief artificial intelligence strategist. Gov. Murphy said Mr. Cole is the right person for the job. “Dave’s applied experience and expertise will be an integral addition to my team,” he said. “I am confident that he will successfully guide the Office of Innovation’s work to design and develop iterative, continuous improvements to state programs.”

François-Xavier Roger appointed Chief Financial Officer, Member of Sanofi Executive Committee

François-Xavier Roger will be appointed Chief Financial Officer and a member of BioNJ Member Sanofi’s Executive Committee effective April 1, 2024. He will be based in Paris and will succeed Jean-Baptiste Chasseloup de Chatillon who will step down from his role to become Head of Apprentis d’Auteuil. Apprentis d’Auteuil is a foundation that supports adolescents who are struggling in life by providing education, training and integration programs, in France and internationally. Being active in charity work for many years, Jean-Baptise decided to devote the next stage of his career to giving back to society. François-Xavier will join from Nestle, where he has been the CFO for more than 8 years, providing strong financial leadership and driving sustainable value creation. He is a seasoned executive who has lived and worked in Europe, the U.S, Asia, Africa and Latin America. 

Cyclacel Pharmaceuticals Announces the Appointment of Brian Schwartz, M.D. as Chief Medical Officer

Berkeley Heights-based BioNJ Member Cyclacel announced that Brian Schwartz, M.D. will assume the role of interim Chief Medical Officer of the company effective immediately. “I am pleased to join the Cyclacel executive team to support the development of the company’s exciting pipeline,” said Dr. Schwartz. “As a member of the Board, I have been closely following emerging clinical data from Phase 1 studies for both fadra and plogo. I am particularly intrigued by potential biomarkers which may assist patient selection in future studies. I believe that these molecules have competitive product profiles and address oncology indications with large unmet medical needs. I look forward to working with the Cyclacel team to advance the programs to proof-of-concept studies.”

BeiGene Welcomes Experienced Life Sciences Executive Olivier Brandicourt to Board of Directors

Hopewell-based BioNJ Member BeiGene announced that Olivier Brandicourt, M.D. has been appointed to its Board of Directors. Dr. Brandicourt will join the audit committee of the Board. Dr. Brandicourt replaces Thomas Malley, who has served on the Board since 2016. “Dr. Brandicourt brings a wealth of global experience as we expand the perspective of BeiGene’s Board of Directors to include deep expertise in leading multinational growth companies, spearheading launches in a range of therapeutic areas and scaling into new geographies,” said John V. Oyler, Chairman, CEO and Co-Founder of BeiGene. “The Board looks forward to working with Dr. Brandicourt, who drove excellence as an outstanding leader for multiple global pharmaceutical brands, as we continue our evolution as a next-generation oncology innovator.”

Johna Norton to Retire as Lilly Executive Vice President of Global Quality

BioNJ Member Eli Lilly and Company, with a site in Branchburg, announced that Johna Norton, Executive Vice President of Global Quality, will be retiring after 34 years of service with the company, effective July 31, 2024. "Johna's career has been built on her commitment to ensuring that our medicines are produced with the highest quality standards. By ensuring our company, manufacturing sites, production lines, team members and collaborators share this commitment, she has had a profound and positive impact on patients and our company," said David A. Ricks, Lilly's Chair and CEO. "During her tenure as leader of Global Quality, Johna has overseen significant expansion, modernization of systems and improvements in our processes and team."

Tris Pharma Appoints Fowler as Chief Compliance Officer, General Counsel

Monmouth Junction-based Tris Pharma announced that Franchesca Fowler has been named Chief Compliance Officer and Vice President, General Counsel. Ms. Fowler will oversee all legal matters within the company, including legal compliance, intellectual property management, litigation and more. “I am pleased to welcome Franchesca to the executive team, and I am confident that she will be a tremendous asset to the organization as we continue to serve individuals with ADHD through our portfolio of marketed products and advance our robust pipeline of investigational therapies for the treatment of ADHD, pain, addiction and neurological disorders,” Ketan Mehta, Founder and CEO at Tris Pharma, said.

Introduction to Advanced Biopharmaceuticals Processing

March 4, 5, 6, 7 and 8 - Rutgers University (New Brunswick, NJ)

This course aims to give the participants an overview of the major biopharma unit operations. Key questions that will be addressed include – What are the underlying physicochemical processes for each unit operation? What are the key features of process design, process analytical technology (PAT), and process development for these unit operations? At the end of the course, the audience will gain a basic understanding of major biopharma unit operations. Case studies will be presented to gain a deeper understanding of these unit operations. Last but not the least, hands on demo of key process steps will be provided, along with review of major operations in a biomanufacturing pilot-plant and bioanalytical lab facility.