Welcome to the latest edition of the BioLines Weekender...
 
As the world continues to navigate the "new" norm, it has never been more important for the life sciences community to come together and support and learn from one another. 

BioNJ is dedicated to working hard on behalf of our Members -- while you are hard at work for Patients. Make sure to take advantage of all the value-driven opportunities available to your organization through your BioNJ Membership. BioNJ...
  • Acts as a conduit for idea sharing, learning, collaboration and mentorship through its many networking and educational programs
  • Provides an extended marketing arm, offering a platform to showcase your science, services and organization to a broad network
  • Advocates in both Trenton and Washington DC to ensure that government fosters medical innovation and Patient access (there's never been a more important time than today to be involved in policy)
  • Works with policymakers to secure incentives and programs that allow New Jersey's innovation ecosystem to flourish
  • Provides deep savings through our Purchasing Consortium so Member companies can preserve their working capital
  • Offers numerous business-building tools, such as BioNJ's Talent and Career Portal
Not a Member? Not a problem. Contact Kim Minton, Director of Member Engagement, at [email protected] today to learn more about Membership. 

Drug development is difficult. Let BioNJ help to make it easier. 

Because Patients Can't Wait®,
The BioNJ Team

One of our favorite Patient advocates, the 2019 Heart of BioNJ Honoree and renowned industry artist, 12-year old "Magic" Max Schill, is Riding for RASopathy. Click here for additional information on "Miles for Max". 

Max, who has grown up before our very eyes, walked the halls of the Capitol on numerous occasions delivering hand-drawn pictures to Senators while advocating for the passage of the 21st Century Cures which he played a key role in getting passed in 2019. Keep up the great work and advocacy, Max! And all the best on your latest challenge!
BioNJ Calendar

IWSTEM Virtual Networking Event
Mark your calendar!
July 15, 2021
Putting Patients First: The Value of Medical Innovation

A recently released report by IQVIA -- an independent organization specializing in health care research -- provides new data showing prices for brand medicines went down last year. The report offers important facts to keep in mind as policymakers look to make health care more affordable. According to the report:
  • Net prices for brand medicines declined by 2.9%, on average, in 2020, and net prices are expected to remain flat or decline over the next five years.
  • Net spending on all prescription medicines increased by less than 1% in 2020.
  • On a per person basis, spending on medicines has increased just 0.5% per year over the past decade, even as many new medicines entered the market.  
  • In 2020, 74% of patients spent less than $20 out of pocket to pick up their brand medicine at the pharmacy. 
  • Just 8% of patients spent more than $500 over the course of the year on prescriptions in 2020.

BioNJ News
As featured in New Jersey Business Magazine

The life sciences industry has had a long and robust history in the Garden State, and thanks to recent advancements in the fight against COVID-19 by homegrown companies like Johnson & Johnson, the industry has never been more relevant. 
 
"We have a tremendous representation of biopharmaceutical companies in New Jersey. Those early companies establishing themselves here truly became the basis for what we have today," adds Debbie Hart, President and CEO of BioNJ. "The industry has grown to 3,200 facilities, which doesn't even include all of those who support the growth of the life sciences in our State, from clinical research organizations to attorneys and accountants -- not to mention the ongoing support of academic institutions and our state government."
As featured in Global Business Reports: U.S. Pharmaceuticals & Biopharmaceuticals 2021

How has BioNJ evolved and adapted to continue offering support and high-level discourse among its Member companies?

At our very core nothing has changed and BioNJ remains passionate about helping our members help patients. We strive to ensure that New Jersey has a robust life sciences ecosystem where innovation is supported and patients can access that innovation. However, if you drill down a bit, a lot has changed because of COVID. We have reinvented ourselves to adapt to the new "norm" to ensure that our members have the tools they need during this unprecedented time and that they are able to continue to do the work that is transforming the lives of patients around the world.

NJ Company News

Jersey City-based BioNJ Member SCYNEXIS, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved BREXAFEMME® (ibrexafungerp tablets), for oral use in patients with vulvovaginal candidiasis (VVC), also known as vaginal yeast infection. BREXAFEMME, which represents the first approved drug in a novel antifungal class in more than 20 years, was approved based on positive results from two Phase 3 studies in which oral ibrexafungerp demonstrated efficacy and a favorable tolerability profile in women with VVC. SCYNEXIS has partnered with Amplity Health, a leading global contract commercialization organization, to support U.S. commercialization of BREXAFEMME, with commercial launch scheduled in the second half of this year.

Jersey City-based BioNJ Member SCYNEXIS, Inc. announced that the U.S. Food and Drug Administration (FDA) granted BREXAFEMME (ibrexafungerp tablets), five years of exclusivity extension under the Generating Antibiotic Incentives Now (GAIN) Act. This exclusivity will be added to any other applicable exclusivity periods, such as the five years of new chemical entity (NCE) exclusivity, for a combined ten-year period of regulatory exclusivity. BREXAFEMME is also protected by multiple patents, including a composition-of-matter patent covering the ibrexafungerp molecule. With patent term extension, this patent is expected to expire in 2035, providing 14 years of protection from generic competitors in the U.S. 

Plainsboro-based BioNJ Member Novo Nordisk announced that the U.S. Food and Drug Administration (FDA) has approved Wegovy™ (the brand name for once-weekly semaglutide 2.4 mg injection in the US) for chronic weight management. Wegovy™ is indicated as an adjunct to diet and exercise for chronic weight management in adults with obesity (initial BMI≥30 kg/m2) or overweight (initial BMI≥27 kg/m) with at least one weight-related comorbidity. Wegovy™ is the first and only once-weekly glucagon-like peptide-1 (GLP-1) receptor agonist therapy approved for weight management for people living with obesity.

Plainsboro-based BioNJ Member Novo Nordisk A/S and Heartseed Inc. announced that they have entered into an exclusive worldwide collaboration and license agreement for development, manufacturing and commercialization of Heartseed's lead asset HS-001. HS-001 is an investigational cell therapy using purified cardiomyocytes derived from induced pluripotent stem cells (iPSC), which is currently under development by Heartseed for the treatment of heart failure. Heartseed is planning to initiate a phase 1/2 clinical trial in Japan (LAPiS Study) in the second half of 2021 evaluating the safety and efficacy of HS-001 for the treatment of heart failure caused by ischemic heart disease.

Princeton-based BioNJ Member Oyster Point Pharma, Inc. announced a newly formed research collaboration with Adaptive Phage Therapeutics (APT) to leverage APT's PhageBank™ technology for the development of potential treatments for multiple ophthalmic diseases. APT's PhageBank™ technology is engineered as an ever-expanding library of bactericidal agents, called bacteriophages ("phages"), to provide broad spectrum coverage. Phages, also are viruses found in the natural environment that infect and replicate specifically in bacteria. Once a bacteriophage attaches to a susceptible bacterium it causes the host cell to die, releasing new bacteriophage to infect other bacteria. APT's approach provides expanded bacterial coverage where prior, traditional, antibiotic approaches have diminished coverage or have become obsolete due to emerging antimicrobial resistance (AMR).

Princeton-based BioNJ Member Oyster Point Pharma, Inc. announced the expansion of its pipeline with the introduction of Enriched Tear Film (ETF™) Gene Therapy and proof-of-concept in vivo study results from its first gene therapy candidate, OC-101. ETF™ Gene Therapy is a proprietary adeno-associated virus (AAV) based gene therapy approach where a target gene is delivered to human lacrimal gland cells via intralacrimal gland injection. Rather than replacing a gene that is defective or missing, a new target gene is delivered that may potentially produce a selected naturally occurring protein, enzyme, or other therapeutic gene product. The goal for this target gene is to produce a selected gene product to change cell behavior and function on the ocular surface. 

Hampton-based BioNJ Member Celldex Therapeutics, Inc. announced preliminary data from the company's Phase 1 dose escalation study of PD-L1xCD27 bispecific antibody CDX-527 in patients with advanced malignancies. These are the first-in-human data of CDX-527, the first candidate developed from Celldex's bispecific platform which utilizes the Company's proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway. "We are encouraged by this emerging initial data where we have observed a good safety profile along with promising pharmacodynamic and pharmacokinetic activity, which are important key hurdles for the development of bispecific antibodies," said Tibor Keler, Ph.D., Executive Vice President and Chief Scientific Officer, Celldex Therapeutics. 

Iselin-based BioNJ Member Outlook Therapeutics, Inc. reported that it has administered the final dose to the last patient enrolled in its pivotal NORSE TWO safety and efficacy study evaluating ONS-5010 (bevacizumab-vikg) for treatment of wet age-related macular degeneration (wet AMD). Topline data are expected to be reported for NORSE TWO in the third calendar quarter. The NORSE TWO pivotal Phase 3 clinical trial enrolled a total of 228 wet AMD patients at 39 clinical trial sites in the United States. Patients in the trial are being treated for 12 months.

Princeton-based BioNJ Member Soligenix, Inc. announced that it has received approximately $865,000, net of transaction costs, in non-dilutive financing via the State of New Jersey's Technology Business Tax Certificate Transfer Program. "This is our eleventh year receiving Net Operating Loss (NOL) funding. Over this time period, we have received approximately $6.5 million in non-dilutive NOL funding that has allowed us to advance our rare disease pipeline to where we are today. We are, again, very grateful for New Jersey Economic Development Authority's (NJEDA) continued support of its biotechnology industry," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix.

Princeton-based BioNJ Member Soligenix, Inc. announced that it has received a Pediatric Investigation Plan (PIP) waiver from the European Medicines Agency for HyBryte™ (SGX301 or hypericin), which has recently and successfully concluded a Phase 3, pivotal clinical study for the treatment of early stage cutaneous T-cell lymphoma (CTCL). "This achievement is an important regulatory milestone as we move forward with marketing applications worldwide," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix. "The PIP waiver allows us to work towards advancing a marketing authorization application (MAA) with EMA in a more cost-effective manner since we will not need to expend significant time and resource to conduct a pediatric clinical study in the European Union.

Bridgewater-based BioNJ Members Sanofi is partnering with leading groups delivering practice-changing breast cancer research, the Breast International Group (BIG), the European Organization for Research and Treatment of Cancer (EORTC) and the Alliance Foundation Trials (AFT), to initiate a pivotal trial of an oral selective estrogen receptor degrader (SERD) in the adjuvant setting. The Phase 3 AMEERA-6 study will evaluate the efficacy and safety of Sanofi's amcenestrant vs. tamoxifen for women with estrogen receptor-positive (ER+) breast cancer who were unable to continue their adjuvant aromatase inhibitor (AI) therapy.

Vaxelis™ (Diphtheria and Tetanus Toxoids and Acellular Pertussis, Inactivated Poliovirus, Haemophilus b Conjugate and Hepatitis B Vaccine), developed as part of a partnership between BioNJ Members Sanofi, based in Bridgewater and Merck & Co., based in Kenilworth, is now available in the U.S. Vaxelis is the first and only hexavalent (six-in-one) combination vaccine available in the U.S. Vaxelis is a vaccine given to protect your child from getting diphtheria, tetanus (lockjaw), pertussis (whooping cough), polio, Hib (Haemophilus influenzae type b), and hepatitis B. Your child cannot get any of these diseases from Vaxelis. 

Kenilworth-based BioNJ Member Merck & Co. and AstraZeneca announced the first presentation of data from the Phase 3 OlympiA trial, in which LYNPARZA demonstrated a statistically significant improvement in its primary endpoint of invasive disease-free survival (iDFS) versus placebo in the adjuvant treatment of patients with germline BRCA1/2 mutations and high-risk human epidermal growth factor receptor 2 (HER2)-negative early breast cancer following definitive local treatment and neoadjuvant or adjuvant chemotherapy. Results were also published in the New England Journal of Medicine. OlympiA is a Phase 3, multicenter, randomized, double-blind, placebo-controlled trial. 

Kenilworth-based BioNJ Member Merck & Co. announced first-time results from the pivotal Phase 3 KEYNOTE-564 trial evaluating KEYTRUDA, Merck's anti-PD-1 therapy, for the potential adjuvant treatment of patients with renal cell carcinoma (RCC) at intermediate-high or high risk of recurrence following nephrectomy (surgical removal of a kidney) or following nephrectomy and resection of metastatic lesions. After a median follow-up of 24.1 months (14.9-41.5), KEYTRUDA demonstrated a statistically significant and clinically meaningful reduction in the risk of disease recurrence or death by 32% compared to placebo (HR=0.68 [95% CI, 0.53-0.87]; p=0.0010). 

Kenilworth-based BioNJ Member Merck & Co. announced it has completed the spinoff of Organon & Co. (Organon). "Today marks a significant milestone for both Merck and Organon. Organon is now an independent, publicly traded company with a broad portfolio of important medicines and products, and is fully prepared to deliver sustainable growth and value," said Rob Davis, president, Merck. "With leadership in our growth pillars and our robust pipeline, Merck is well positioned for strong long-term growth, with scientific innovation remaining the source of our value creation." Merck's goal with the spinoff was to create two patient-focused companies with enhanced strategic and operational focus, improved agility, simplified operating models, optimized capital structures and improved financial profiles. 

Kenilworth-based BioNJ Member Merck & Co. announced new investigational data from the pivotal Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, which evaluated the combinations of KEYTRUDA, Merck's anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). Results from a new analysis evaluating health-related quality of life (HRQoL) based on patient-reported outcomes. Data from this trial are currently under review with the U.S. Food and Drug Administration (FDA).

Kenilworth-based BioNJ Member Merck & Co. announced it has entered into a procurement agreement with the United States government for molnupiravir (MK-4482). Molnupiravir is currently being evaluated in a Phase 3 clinical trial, the MOVe-OUT study, for the treatment of non-hospitalized patients with laboratory-confirmed COVID-19 and at least one risk factor associated with poor disease outcomes. Merck is developing molnupiravir in collaboration with Ridgeback Biotherapeutics. Through the agreement, if molnupiravir receives Emergency Use Authorization (EUA) or approval by the U.S. Food and Drug Administration (FDA), Merck will receive approximately $1.2 billion to supply approximately 1.7 million courses of molnupiravir to the United States government. 

Morris Plains-based BioNJ Member Gilead Sciences, Inc. announced new data from the Phase 3 ASCENT study evaluating Trodelvy® (sacituzumab govitecan-hziy) in relapsed or refractory metastatic triple-negative breast cancer (TNBC). In this subgroup analysis of brain metastases-negative patients who received only one line of prior systemic therapy in the metastatic setting in addition to having disease recurrence or progression within 12 months of (neo) adjuvant chemotherapy, Trodelvy improved progression-free survival (PFS), with a 59% reduction in the risk of disease worsening or death (HR: 0.41; 95% CI: 0.22-0.76) and a median PFS of 5.7 months (n=33) versus 1.5 months with chemotherapy (n=32). 

Princeton-based BioNJ Member Genmab A/S and Bolt Biotherapeutics, Inc. announced that the companies have entered into an oncology research and development collaboration. Together, the companies will evaluate Genmab antibodies and bispecific antibody engineering technologies in combination with Bolt's proprietary Boltbody™ immune-stimulating antibody conjugate (ISAC) technology platform, with the goal of discovering and developing next-generation, immune-stimulatory, antibody-based conjugate therapeutics for the treatment of cancer. This research collaboration will evaluate multiple bispecific ISAC concepts to identify up to three clinical candidates for development. Genmab will fund the research, along with the preclinical and clinical development of these candidates through clinical proof of concept.

East Hanover-based BioNJ Member Novartis announced the U.S. Food and Drug Administration (FDA) has approved Cosentyx® (secukinumab) for the treatment of moderate to severe plaque psoriasis in pediatric patients six years and older who are candidates for systemic therapy or phototherapy. This is the first approval for Cosentyx in a pediatric population in the U.S. The Cosentyx clinical profile is supported by five years of adult data showing long-lasting efficacy and a consistent safety profile across moderate to severe plaque psoriasis, psoriatic arthritis and ankylosing spondylitis.

East Hanover-based BioNJ Member Novartis announced 2-year positive results from the Phase III JUNIPERA study, demonstrating that Cosentyx® (secukinumab) significantly delayed time to flare vs. placebo (P<.001) in pediatric patients with juvenile psoriatic arthritis (JPsA) and enthesitis-related arthritis (ERA) -- two subtypes of juvenile idiopathic arthritis (JIA). The JUNIPERA study also demonstrated sustained efficacy for Cosentyx with more patients achieving and maintaining the JIA American College of Rheumatology (ACR) 30 and JIA ACR 70 responses from Week 12 to Week 104 vs placebo. Cosentyx demonstrated a favorable safety profile with no new safety signals reported in pediatric patients (age 2 to 17 years) with two years of treatment.

East Hanover-based BioNJ Member Novartis announced updated median overall survival (OS) results for Kisqali® (ribociclib) in combination with fulvestrant in postmenopausal women with hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer. The exploratory analysis of OS after an additional 16.9 months of follow-up of the Phase III MONALEESA-3 trial evaluated Kisqali plus fulvestrant as first- or second-line treatment compared to fulvestrant alone in postmenopausal women with HR+/HER2- metastatic breast cancer. The analysis found that with extended follow-up of more than four years, Kisqali in combination with fulvestrant continued to demonstrate a clinically relevant OS benefit of more than a year compared with fulvestrant alone.

East Hanover-based BioNJ Member Novartis announced robust data from the primary analysis of the pivotal Phase II ELARA trial of Kymriah® (tisagenlecleucel) in patients with relapsed or refractory (r/r) follicular lymphoma (FL). In the primary ELARA analysis, 97 patients were infused and evaluated for safety, 94 patients were evaluable for efficacy with a median follow-up of 11 months. Importantly, no patients experienced grade 3/4 cytokine release syndrome (CRS), the most common side effect associated with CAR-T therapy. Kymriah led to responses for the majority of patients treated, with 66% achieving a complete response (CR) (95% CI, 56-75). The overall response rate was 86% (95% CI, 78-92). Response rates were consistent across high-risk patient subgroups. 

East Hanover-based BioNJ Member Novartis announced that results of the Phase III VISION study evaluating Lu-PSMA-617, a targeted radioligand therapy, plus best standard of care (SOC) demonstrated significant improvement in overall survival (OS) compared to SOC alone, in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). The difference in OS between study arms was statistically significant (one-sided p<0.001), with an estimated 38% reduction in risk of death in the Lu-PSMA-617 arm (n=551) compared to the best standard of care only arm (n=280) (hazard ratio: 0.62 with 95% confidence interval (CI): (0.52, 0.74)).

East Hanover-based BioNJ Member Novartis reported the final analysis from the NETTER-1 phase III study comparing treatment using Lutathera® (INN: lutetium (177Lu) oxodotreotide / USAN: lutetium Lu 177 dotatate) plus 30 mg octreotide LAR to 60 mg of octreotide LAR in patients with midgut neuroendocrine tumors. The previously reported primary analysis of the trial demonstrated a statistically significant improvement in progression free survival (PFS) (HR: 0.18*, p < 0.0001). In the final analysis of overall survival, a secondary objective of the trial, treatment with Lutathera resulted in a clinically relevant prolongation in median overall survival of 11.7 months [48.0 months (95%CI: 37.4-55.2) compared to the control arm (36.3 months (95%CI: 25.9-51.7)]. 

East Hanover-based BioNJ Member Novartis announced results from the pivotal Phase III RATIONALE 302 trial showing the investigational anti-PD-1 immune checkpoint inhibitor tislelizumab improved overall survival (OS) versus chemotherapy (median 8.6 months vs. 6.3 months, p=0.0001).The study evaluated tislelizumab in patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who had received prior systemic therapy. Results from RATIONALE 302 in ESCC showed tislelizumab extended median OS by 2.3 months compared to chemotherapy with a 30% reduction in the risk of death (HR=0.70, 95% CI: 0.57-0.85, p=0.0001). In PD-L1 positive patients, tislelizumab extended median OS by 3.5 months with a 46% reduction in the risk of death (HR=0.54, 95% CI: 0.36-0.79, p=0.0006).

East Hanover-based BioNJ Member Novartis announced Phase II primary endpoint data showing investigational iptacopan (LNP023) - a first-in-class, oral, targeted factor B inhibitor -- reduced protein in the urine (proteinuria), an increasingly recognized surrogate marker correlating with progression to kidney failure, and showed promise in stabilizing kidney function in patients with IgA nephropathy (IgAN). In the Phase II study (NCT03373461), patients (n=112) with IgAN were randomized to placebo or different doses of iptacopan. The primary endpoint was met with a statistically significant (p=0.038) dose response effect on reduction in proteinuria (as measured by 24-hour urinary protein to creatine ratio [UPCR 24h]) with iptacopan vs. placebo, at 90 days. 

East Hanover-based BioNJ Member Novartis announced positive new interim Phase II data showing investigational iptacopan (LNP023) -- a first-in-class, oral, targeted factor B inhibitor -- improved estimated glomerular filtration rate (eGFR) slope and stabilized kidney function in patients with C3 glomerulopathy (C3G) treated with iptacopan. The new interim analysis data from the open-label Phase II study (NCT03832114) in patients with C3G showed twice-daily 200mg iptacopan stabilized kidney function, as measured by change in eGFR slope: a key measure of kidney clearance function that estimates the rate of blood passing through and being filtered by the kidneys.

Madison-based BioNJ Member AbbVie announced results from a four-year follow-up analysis of the Phase 3 CLL14 trial, which showed that previously untreated patients with chronic lymphocytic leukemia (CLL) with coexisting conditions who were treated with VENCLYXTO®/VENCLEXTA® (venetoclax) plus obinutuzumab continued to show longer progression-free survival (PFS) and higher rates of undetectable minimal residual disease (MRD) compared to patients receiving a standard of care chemoimmunotherapy regimen of obinutuzumab and chlorambucil.

Madison-based BioNJ Member AbbVie announced new data from the Phase 2 CAPTIVATE (PCYC-1142) study investigating IMBRUVICA® (ibrutinib) in combination with VENCLEXTA®/VENCLYXTO® (venetoclax), an all-oral, once-daily, chemotherapy-free, fixed-duration investigational combination, for patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The ibrutinib and venetoclax cohort met its primary endpoint of complete response (CR) rate of 56% (95% CI 48-64) among patients without del(17p), 70 years old or younger and with 27.9 months of follow up. This rate was higher than the 37% minimum meaningful rate study assumption (P<0.0001). The CR rate was consistent across all patients in the study including high-risk CLL patient groups. 

Madison-based BioNJ Member AbbVie announced extended long-term data from the Phase 3 RESONATE-2 study (PCYC-1115/1116) evaluating single-agent IMBRUVICA (ibrutinib) versus chlorambucil with up to seven years of follow-up in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The RESONATE-2 study evaluated 269 patients 65 years or older with previously untreated CLL/SLL, without 17p deletion, who received continuous single-agent IMBRUVICA until progression or chlorambucil up to 12 cycles. With up to seven years of follow-up, progression-free survival (PFS) benefit with single-agent IMBRUVICA was sustained (Hazard Ratio [HR] 0.160 [95 percent Confidence Interval (CI): 0.111-0.230]).

Berkeley Heights-based BioNJ Member Amgen announced updated results for investigational bemarituzumab in combination with chemotherapy from the Phase 2 FIGHT trial. The trial evaluated bemarituzumab plus chemotherapy (mFOLFOX6) versus chemotherapy alone in patients with FGFR2b-positive, HER2-negative frontline advanced gastric or gastroesophageal junction cancers (GEJ). New data includes median overall survival (OS), a secondary endpoint that was reached with longer follow-up, as well as additional analyses of patient subgroups. With a median follow-up of 12.5 months, the addition of bemarituzumab to chemotherapy resulted in a median OS of 19.2 months versus 13.5 months for chemotherapy alone in all randomized patients (n=155, HR: 0.6; 95% CI: 0.38, 0.94). 

Berkeley Heights-based BioNJ Member Amgen and Bedminster-based Kyowa Kirin Co. Ltd. announced an agreement to jointly develop and commercialize KHK4083, which is Kyowa Kirin's potential first-in-class, Phase 3-ready anti-OX40 fully human monoclonal antibody in development for the treatment of atopic dermatitis, with potential in other autoimmune diseases. In February, Kyowa Kirin announced positive results from a Phase 2 study of KHK4083 in patients with moderate-to-severe atopic dermatitis, which affects nearly 30 million people in major global markets. Under terms of the agreement, Amgen will lead the development, manufacturing, and commercialization for KHK4083 for all markets globally, except Japan, where Kyowa Kirin will retain all rights. 

Berkeley Heights-based BioNJ Member Amgen presented data on overall survival, a secondary endpoint, from the Phase 2 results of the CodeBreaK 100 clinical study for LUMAKRASTM (sotorasib) in previously treated patients with non-small cell lung cancer (NSCLC). These data were published in the New England Journal of Medicine. The publication includes mature overall survival and duration of response data, and results from subgroup and exploratory biomarker analyses. LUMAKRAS shows a median overall survival (OS) of 12.5 months among 124 evaluable patients, the majority of which were previously treated with both platinum-based chemotherapy and immunotherapy (81%) (data cutoff of March 15, 2021).

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced new data for ciltacabtagene autoleucel (cilta-cel), an investigational B-cell maturation antigen (BCMA)-directed CAR-T therapy, demonstrated sustained efficacy and durable responses in heavily pretreated patients with relapsed/refractory multiple myeloma (RRMM). Updated results from the Phase 1b/2 CARTITUDE-1 study (n=97) with a longer-term follow-up at a median of 18 months showed an overall response rate (ORR) of 98 percent, with 80 percent of patients achieving a stringent complete response (sCR), highlighting a deepening response over time (increasing from 67 percent presented at ASH 2020). These data also showed 66 percent of patients were progression free and alive at 18 months (95 percent Confidence Interval [CI], range, 54.9-75.0.

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for teclistamab in the treatment of relapsed or refractory multiple myeloma. This distinction for teclistamab, an off-the-shelf, T-cell redirecting, bispecific antibody targeting both B-cell maturation antigen (BCMA) and CD3 receptors, follows a PRIME (PRIority MEdicines) designation from the European Medicines Agency (EMA) received earlier this year. This BTD marks the 11th received by Janssen's Oncology Therapeutic Area.

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced new efficacy and safety data for first-in-class TREMFYA® (guselkumab), including data from the first study evaluating a selective IL-23 inhibitor in adult patients with active PsA, all of whom had demonstrated inadequate response or intolerance to TNFi. In the COSMOS Phase 3b study, significantly higher proportions of patients treated with TREMFYA showed joint symptom improvement and complete skin clearance versus placebo at week 24 in this true TNFi-IRa patient population, which is often more difficult to treat.

Princeton-based BioNJ Member Bristol Myers Squibb announced the first data from the Phase 2 BEYOND study evaluating Reblozyl®(luspatercept-aamt), a first-in-class erythroid maturation agent, plus best supportive care in adult patients with non-transfusion dependent (NTD) beta thalassemia. Results demonstrated that 77.7% of patients treated with Reblozyl achieved a hemoglobin increase (≥1.0 gram/deciliter) compared to 0% of patients in the placebo arm. Changes in patient-reported outcomes also correlated with increases in hemoglobin. NTD beta thalassemia is a term used to describe patients who do not require lifelong regular red blood cell (RBC) transfusions for survival, although they may require occasional or even frequent transfusions, usually for defined periods of time. 

Princeton-based BioNJ Member Bristol Myers Squibb announced that data from 28 company-sponsored and investigator-sponsored studies will be presented at the EULAR 2021 Virtual. The research highlights the depth and strength of the company's growing immunology pipeline and portfolio, commitment to the rheumatology research community and focus on delivering meaningful solutions that address unmet patient needs across immune-mediated diseases. "Rheumatic diseases can be debilitating for the tens of millions of people worldwide who live with these conditions. With the understanding that these diseases can behave very differently from person to person, Bristol Myers Squibb is pursuing pathbreaking science to tailor therapies to individual needs, improve outcomes and expand treatment options," said Mary Beth Harler, M.D., Head of Immunology and Fibrosis Development, Bristol Myers Squibb. 

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Commission (EC) has approved Opdivo (nivolumab) plus Yervoy (ipilimumab) for the first-line treatment of adults with unresectable malignant pleural mesothelioma (MPM). The EC's decision is based on results from the CheckMate -743 trial, the first and only positive Phase 3 study of an immunotherapy in first-line MPM. The trial met its primary endpoint, showing superior overall survival (OS) with Opdivo plus Yervoy versus chemotherapy (pemetrexed and cisplatin or carboplatin) in all randomized patients. The safety profile for Opdivo plus Yervoy in first-line MPMwas manageable using established adverse event management protocols and consistent with previous studies of the combination in other tumor types.

Princeton-based BioNJ Member Bristol Myers Squibb announced results from the Phase 3 CheckMate -648 trial, in which two Opdivo-based treatment combinations -- Opdivo (nivolumab)plus chemotherapy and Opdivo plus Yervoy (ipilimumab) -- demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to chemotherapy at the pre-specified interim analysis in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥1%, as well as in the all-randomized population. Opdivo plus Yervoy is the first dual immunotherapy combination to demonstrate a superior survival benefit versus chemotherapy in this setting.

Bridgewater-based BioNJ Member Eli Lilly and Company announced new data for the investigational use of Verzenio® (abemaciclib) in high risk early breast cancer, and for its oral selective estrogen receptor degrader (SERD) LY3484356 at the 57th Annual Meeting of the American Society of Clinical Oncology (ASCO). Lilly is presenting an exploratory analysis from the positive Phase 3 monarchE trial evaluating Verzenio, a CDK4/6 inhibitor, in a subgroup of patients with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) high risk early breast cancer (EBC) who had received neoadjuvant chemotherapy. Physicians often treat patients with HR+, HER2- breast cancer who they believe to be at the highest risk of recurrence with neoadjuvant chemotherapy prior to curative intent surgery.

To improve the understanding and advance the treatment of migraine, Bridgewater-based BioNJ Member Eli Lilly and Company presented data on unmet needs in migraine from the OVERCOME (U.S.) study and on its portfolio of migraine. New findings from OVERCOME (U.S.) revealed the top six reasons why people hesitate to seek migraine care. Real-world data insights revealed greater adherence and persistence for Emgality® (galcanezumab-gnlm), a calcitonin gene-related peptide monoclonal antibody (CGRP mAb), compared to oral standard of care (non-CGRP mAb) preventive migraine treatments in a health care claims study and greater probability of patient preference for the Emgality auto-injector profile compared to Aimovig® (erenumab) and AJOVY® (fremanezumab) device characteristics in a survey.

Bridgewater-based BioNJ Member Eli Lilly and Company and Incyte will present data from post-hoc analyses that suggested OLUMIANT® (baricitinib) 4 mg tablet reduced pain and duration of morning joint stiffness, and improved overall physical function at 12 weeks, among patients with moderate to severe rheumatoid arthritis (RA), compared to HUMIRA® (adalimumab) and placebo. In a post-hoc analysis of the Phase 3 RA-BEAM study, patients treated with OLUMIANT 4 mg saw greater improvements in pain relief and physical function, as well as reduced duration of morning joint stiffness, at 12 weeks compared to HUMIRA and placebo. These differences in pain relief were not influenced by disease activity during treatment. In this analysis, improvements in fatigue with OLUMIANT 4 mg were greater than with placebo and similar to HUMIRA after 12 weeks of treatment. Safety results were consistent with the established safety profile for OLUMIANT in patients with RA.

Bridgewater-based BioNJ Member Eli Lilly and Company will present new data from Phase 3 studies that further demonstrated the long-term efficacy and safety profile of Taltz® (ixekizumab) among patients with axial spondyloarthritis (axSpA). AxSpA is recognized as a single disease entity, with two subtypes which are defined depending on the presence (radiographic axSpA, or r-axSpA) or absence (non-radiographic axSpA, or nr-axSpA) of defined structural damage of the sacroiliac joints on plain x-ray films as per the modified New York (mNY) criteria. Lotus Mallbris, M.D., Ph.D., Vice President of Immunology Development at Lilly said, "We are excited to present a range of new data at EULAR that demonstrate treatment with Taltz provides consistent, long-term efficacy on common signs and symptoms over time in axial spondyloarthritis."

Ridgefield Park-based BeiGene, Ltd. announced that results from the interim analysis of the Phase 3 ALPINE trial comparing BRUKINSA® (zanubrutinib) to ibrutinib in adults with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) will be reported in an oral presentation as part of the Presidential Symposium at the 26th European Hematology Association. "The positive topline results from the Phase 3 ALPINE trial interim analysis suggest that BRUKINSA could improve clinical benefit for patients with CLL, compared to the first-generation BTK inhibitor," said  Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. Results from the interim analysis were based on 415 enrolled patients in the trial, including 207 on BRUKINSA treatment and 208 on ibrutinib treatment. 

Many people saw the breaking news earlier this week that Aduhelm, a drug produced by Biogen to treat one aspect of the fight against Alzheimer's disease, was given accelerated FDA approval, becoming the first Alzheimer's drug approved in more than two decades. What you might have missed is this: The drug is being co-developed and co-manufactured by Eisai, the Tokyo-based pharmaceutical firm that has its North American headquarters in Woodcliff Lake. Eisai Chairman Ivan Cheung said the reason is simple: Biogen and Eisai agreed that Biogen would take the lead on the product in the U.S. while Eisai co-promotes the product.

New Brunswick-based Infinity BiologiX announced the launch of a new test meant to assess the severity of COVID-19 in individuals. The COVID-19 Risk Test is designed to predict disease severity in people ages 18 and older, using genetic and clinical information to provide a risk score-individuals can then use that score to understand their personal risk of contracting a serious case of COVID-19 and make more informed decisions. In addition, employers, governments and other public health entities may use the data to make informed decisions about disease risk, treatment options and vaccination priorities. This will assist in guiding proactive steps to minimize disease exposure and manage the pandemic in the weeks and months ahead

Ridgefield Park-based BeiGene, Ltd. announced clinical data from two pivotal trials of its anti-PD-1 antibody tislelizumab, including the Phase 3 RATIONALE 302 trial of tislelizumab compared to chemotherapy in previously treated patients with advanced or metastatic esophageal squamous carcinoma (ESCC) and the pivotal Phase 2 trial of tislelizumab in patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch-repair-deficient (dMMR) solid tumors. "We and our collaborator BioNJ Member Novartis are committed to advancing tislelizumab in a broad global clinical program both as a monotherapy and in combination with other cancer therapeutics," said Yong Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene.

Ridgefield Park-based BeiGene, Ltd. announced that its PARP inhibitor pamiparib showed efficacy in patients with HER2-negative breast cancer and demonstrated numerically higher but not statistically significant progression-free survival in gastric cancer. The results in gastric cancer may have been influenced by the fact that the study did not meet the planned enrollment target. These data included initial reporting from a Phase 2 trial evaluating pamiparib in locally advanced or metastatic HER2-negative (HER2[-]) breast cancer with germline BRCA1/2 mutation (gBRCA1/2m) and initial reporting of a randomized Phase 2 trial of pamiparib as maintenance therapy in patients with inoperable locally advanced or metastatic gastric cancer who responded to platinum-based first-line chemotherapy.

Basking Ridge-based Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) updated the Emergency Use Authorization (EUA) for REGEN-COV™, lowering the dose to 1,200 mg (600 mg casirivimab and 600 mg imdevimab), which is half the dose originally authorized. As part of the updated EUA, REGEN-COV should be administered by intravenous (IV) infusion; subcutaneous (SC) injections are an alternative when IV infusion is not feasible and would lead to a delay in treatment. REGEN-COV is authorized for use under an EUA to treat mild-to-moderate COVID-19 in adults and pediatric patients (12 years of age and older weighing ≥40 kg) with positive results of direct SARS-CoV-2 viral testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death.

Franklin Lakes-based BD (Becton, Dickinson and Co.) announced it received pandemic orders for needles and syringes totaling 2 billion injection devices to support global COVID-19 vaccination efforts. This new milestone reflects commitments to governments across the world, including the United States, Australia, Brazil, Canada, France, Germany, India, Philippines, Saudi Arabia, South Africa, Spain and the United Kingdom, among many others. In total, BD is supporting pandemic orders for more than 40 countries, non-governmental organizations and other partners.

Bayer agreed to acquire Noria Therapeutics Inc. (Noria) and PSMA Therapeutics Inc., through which the Whippany-based company will broaden its existing oncology portfolio of targeted alpha therapies. The deal, for which financial details were not disclosed, will see Bayer pick up exclusive rights to two investigational therapies for prostate cancer that will complement the already approved therapy Xofigo. Targeted alpha therapies, or TATs, treat difficult tumors by delivering alpha radiation inside the body using either a bone-seeking property or through the combination of alpha radionuclides with specific molecules.



People in the News

Finding educated, skilled employees can be difficult. Competition for the best and the brightest is often fierce, with the top candidates having many options. Creating early relationships with potential stars is one way to get a jump on rivals. Stuart Peltz is doing just that. Dr. Peltz is the CEO of BioNJ Member PTC Therapeutics, a biopharmaceutical company based in South Plainfield. The company created an internship program for recent college graduates -- 53 in its first year. For this edition of NJBIZ Conversations, Editor Jeff Kanige talked to Dr. Peltz about the company and the origins of the internship program.


On this edition of NJBIA's "Minding Your Business," Martin Rexroad, Chief Culture Officer of South Plainfield-based BioNJ Member PTC Therapeutics, discusses the company's mission and considerable efforts to get younger people interested and involved in life sciences careers. 

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced the election of  Mary L. Smith to the company's Board of Directors.  Ms. Smith has previously served as the Principal Deputy Director and CEO equivalent at the Indian Health Service, a $6 billion division of the U.S. Department of Health and Human Services (HHS) and system of hospitals providing healthcare to more than two million Native Americans in the United States.  Ms. Smith currently serves as Vice Chair at the VENG Group, a non-partisan government relations and public affairs firm where she consults clients on business development, strategy, health care and corporate governance.

Brunswick-based BioNJ Member BioAegis Therapeutics Inc. announces that Ernst & Young LLP (EY US) named CEO Susan L. Levinson, Ph.D., as an Entrepreneur of the Year® 2021 New Jersey Award finalist. Now in its 35th year, the Entrepreneur of the Year program honors unstoppable business leaders whose ambition, ingenuity and courage in the face of adversity help catapult us from the now to next and beyond. Dr. Levinson was selected by a panel of independent judges. Dr. Levinson has founded two New Jersey biotech companies in partnership with scientific and business founders and is passionately committed to developing unique products to meet patient needs. 

Lawrenceville-based BioNJ Member Celsion Corporation announced the appointment of  Christine A. Pellizzari to the Celsion Board of Directors.  Ms. Pellizzari is Chief Legal Officer of BioNJ Member Insmed Incorporated, a global biopharmaceutical company dedicated to transforming the lives of patients with serious and rare diseases and brings to Celsion more than 20 years of leadership in the global pharmaceutical industry. Her appointment increases the size of Celsion's Board from seven to eight Directors.  Ms. Pellizzari joined Insmed in 2013 as General Counsel and Corporate Secretary and was named Chief Legal Officer in 2018. 


Funding

New Jersey Economic Development Authority (NJEDA) announced that VentureWell's ASPIRE Program has become the third accelerator approved to participate in its NJ Accelerate program. NJ Accelerate was created by the NJEDA last year to grow New Jersey's innovation ecosystem by promoting greater participation by Garden State entrepreneurs in qualified accelerator programs throughout the United States. It is also designed to incentivize graduate companies from approved accelerators to consider locating in NJ, as well as to showcase the talent in Garden State accelerators on a national stage. 

New Jersey Economic Development Authority (NJEDA) released a draft of the rules under which the new Personal Protective Equipment (PPE) Manufacturing Tax Credit program will be administered. Created under the New Jersey Economic Recovery Act of 2020 (ERA), the PPE Manufacturing Tax Credit program is a three-year program that provides tax credits to incentivize the development of PPE manufacturing within the State. Members of the public are encouraged to review the preliminary rules and submit written feedback through an online form available on the Economic Recovery Act website.

New Jersey Economic Development Authority (NJEDA) Board approved an update to Phase 2 of the Small Business Emergency Assistance Loan Program. The program will provide up to $100,000 in low-cost financing to New Jersey small businesses and nonprofits with priority given to entities that have taken on new space, transitioned from a home-based business or smaller space into a larger space, or acquired an owner-occupied commercial space. More information on the program is available at https://business.nj.gov/covid/emergency-assistance-loan-program. 

Institution and Education News

In the world of cancer therapies, nanoparticles hold great promise for delivering more effective and safer cancer treatment than the standard small molecule drugs that are commonly used today. New Brunswick-based Rutgers Cancer Institute of New Jersey Director Steven K. Libutti, M.D., FACS, whose work involves understanding the tumor microenvironment; William Zamboni, PharmD, Ph.D., an expert in the pharmacology of nanoparticles at the University of North Carolina Eshelman School of Pharmacy; and Sha Chang, Ph.D., Director of Medical Physics Research at UNC, are hoping to fight pancreatic cancer using the combination of a novel type of radiation and nanoparticles. The work is supported by a $2,792,913 grant (1R01CA247652-01A1) from the National Institutes of Health's National Cancer Institute.

This year marks a grim milestone for breast cancer seeing it overtake lung cancer to become the most commonly diagnosed cancer globally. Around one in eight U.S. women is expected to develop breast cancer over the course of her lifetime, and nearly one in 40 will die from it. These figures have renewed urgency within the scientific community to come up with better strategies for treating this disease, including researchers at New Brunswick-based Rutgers Cancer Institute of New Jersey and Rutgers New Jersey Medical School (NJMS). In a new study, they discovered a phage display-based approach to halt tumor growth that could be used to treat triple-negative breast cancer -- considered the most aggressive type of breast cancer.

Michael E. Zwick of Emory University will be Senior Vice President for Research at Rutgers University, effective Sept. 1 pending approval by the Board of Governors. Dr. Zwick will arrive at Rutgers after serving as Associate Vice President for Research of the Robert W. Woodruff Health Sciences Center at Emory University and the associate dean of research and professor of human genetics and pediatrics in the Emory University School of Medicine. He spent over 16 years at the Atlanta-based research institution, beginning as an assistant professor in 2005.

Jonathan G.S. Koppell has been selected as the 9th President of Montclair State University (MSU). As Vice-Provost for Public Service and Social Impact and Dean of the Watts College of Public Service & Community Solution at Arizona State University (ASU), Dr. Koppel is a nationally regarded scholar of policy, organization and management who transformed the public affairs college at ASU into one of the largest, best and most innovative in the nation. "At a pivotal moment in higher education, this university -- with its deep commitment to serving the public interest and advancing student success -- has an opportunity to define the future. I am so impressed by what the faculty and staff have accomplished with President Cole, and I am confident we can build on this foundation in bold, imaginative ways that contribute to the prosperity, health and well being of New Jersey and the nation," said Dr. Koppel.

Events

Join TechUnited for its first-ever BetterWellness Innovation Summit, powered by RWJBarnabas Health, dedicated to showcasing innovation, collaborating on solutions for our communities and providing insight into the HealthTech industry. Attendees can expect to hear pitches from cutting-edge start-ups in the hottest health care industries, gain insights from extremely accomplished doctors, executives and founders, and be the FIRST to get all of the details on the brand-new TechUnited:BetterWellness Challenge, announced at the Summit.

BioNJ Member Services Provider Directory

BioNJ recognizes the vital role that our Service Provider Members play in the growth of New Jersey's robust life sciences ecosystem. For this reason, we are delighted to remind our community about BioNJ's Member Service Provider Directory, a categorical listing of BioNJ Members by service sector. By working with fellow BioNJ Member organizations, you are not only supporting our community, but benefiting from a wide variety of high quality organizations. To learn how you can become a Member and be featured on this resource tool, please contact Kim Minton at [email protected], or 609-890-3185. Find providers in these categories:

Please contact BioNJ at [email protected] 
or call 609-890-3185 with any questions.