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October 8, 2021

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Welcome to the latest edition of the BioLines Weekender...


Last week, BioNJ released a new white paper, “Prescription Drug Affordability Boards: Myths vs. Facts on Drug Supply Chain,” that corrects the record on several common myths about Prescription Drug Affordability Boards (PDAB) and their harmful impact on Patients and the medical innovation ecosystem. The BioNJ white paper concludes that policymakers should avoid the creation of a PDAB as a means to control drug costs and should instead adopt solutions that will improve Patient access and deliver savings to the health care system, such as capping Patient out-of-pocket costs and using negotiated rebates to provide savings to Patients.

 

BioNJ remains committed to working with New Jersey policymakers to implement solutions that ensure Patients have meaningful access to their medicines, reduce health care costs, and support innovation necessary for future cures and treatments.


Download a copy of the white paper here.

Download an infographic featuring the Myths vs. Facts here.


Meanwhile, BioNJ is excited to share a new digital media campaign just released yesterday. The digital ad, prominently featured on NJ.com’s home page, highlights Dr. Ted Love, CEO of Global Blood Therapeutics, addressing the issue of price controls disproportionally impacting the BIPOC (Black, Indigenous and People of Color) Community. Dr. Love’s company is focused on addressing Sickle Cell Disease. The ad takes viewers to a landing page explaining the negative impact of H.R. 3 in greater detail and calls for viewers to take action by providing the tools needed to “Tell Congress to Take Real Action for Patients.”

 

Please click here and take action.

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To learn more about BioNJ’s work on H.R. 3 and Prescription Drug Affordability Boards, including podcasts, webinars and more, reach out to John Slotman, Vice President of Government Affairs, at JSlotman@BioNJ.org. We hope to see you next Friday, October 15, at BioNJ’s Ninth Annual C-Suite Summit tackling the issues keeping you and your Team up at night. Click here for details.

Upcoming BioNJ Events

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C-Suite Summit: Transitioning to the "Next Norm" 

(In-Person Kick-Off Reception the Evening Before!

Register Today!

October 15, 2021

IT/Cyber/Finance Briefing: Topics in ESG and Beyond: Cyber, IT, Tax and More

Mark Your Calendar!

November 17, 2021

Membership Webinar

Mark Your Calendar!

November 18, 2021

Inspiring Women in STEM Conference 

Mark Your Calendar!

December 3, 2021

Patient Advocacy Summit

Mark Your Calendar!

December 9, 2021

Putting Patients First:

The Value of Medical Innovation

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New Report Shows 3 in 10 Americans That Have Insurance Still Face a Financial Barrier to Care

A report, titled “Barriers to Health Care Access in the Patient Experience,” surveyed over 4,700 Americans to explore the barriers they face in access to health care and prescription medicines. The survey revealed that 3 in 10 Americans that have insurance still face a financial barrier to care, like having trouble paying medical bills or having out-of-pocket costs that are more than they can afford. Health insurance just isn’t working like it should because insurers and pharmacy benefit managers are increasingly shifting more health care costs onto patients through high deductibles and coinsurance.

Reference Pricing Isn’t Worth the Risk to Innovation and Patient Health

As policymakers in Washington, D.C. debate ways to stem rising health care spending, international reference pricing is one of many ideas being considered that could have damaging effects on the future of biopharmaceutical innovation and patient health. International reference pricing policies would link the price that Medicare pays for certain drugs to prices paid in other countries, with these negotiated prices also extending to private health plans. A new "Health Affairs Blog" explores the tradeoffs and risks associated with international reference pricing and suggests alternative policy approaches that would reduce drug costs, while still maintaining incentives for innovation.

BioNJ in the News

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2 N.J. Organizations Sign MOUs With Leading Indian Biotech Organization

The Association of Biotechnology Led Enterprises, a not-for-profit organization representing the Indian biotechnology sector, has signed two separate memoranda of understanding that will strengthen New Jersey’s ties with cutting-edge biotechnology companies in India, Choose New Jersey announced Wednesday. The MOUs are with two organizations: the Institute for Life Science Entrepreneurship, a New Jersey-based not-for-profit focused on supporting life science entrepreneurship based out of Kean University, and BioNJ, the largest life sciences association in New Jersey. ABLE was first introduced to New Jersey leaders during Gov. Phil Murphy’s business attraction trip to India with Choose New Jersey in 2019.

BioNJ Releases White Paper: "Prescription Drug Affordability Boards: Myths vs. Facts on Drug Supply Chain"

BioNJ released a new white paper, "Prescription Drug Affordability Boards: Myths vs. Facts on Drug Supply Chain," that corrects the record on several common myths about Prescription Drug Affordability Boards (PDAB) and their harmful impact on Patients and the medical innovation ecosystem. The BioNJ white paper concludes that policymakers should avoid the creation of a PDAB as a means to control drug costs and should instead adopt solutions that will improve Patient access and deliver savings to the health care system, such as capping Patient out-of-pocket costs and using negotiated rebates to provide savings to Patients.


NJ Company News

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Outlook Therapeutics Reports New Positive 12-Month Safety Data from Pivotal Phase 3 NORSE TWO Trial

Iselin-based BioNJ Member Outlook Therapeutics announced new 12-month safety data from the pivotal Phase 3 NORSE TWO trial that further confirm the strong safety profile in this study of ONS-5010 / LYTENAVA™ (bevacizumab-vikg) for treatment of neovascular age-related macular degeneration (wet AMD). In August 2021, Outlook Therapeutics announced the topline readout of pivotal data from its NORSE TWO trial, which included 11 months of safety data. The topline data previously reported from NORSE TWO demonstrated clinically relevant and highly statistically significant results as well as a robust safety profile indicating that in the trial ONS-5010 was well tolerated and showed no unanticipated safety signals.

Researchers Discover Previously Unknown Role of BioAegis’ Therapeutic Gelsolin as Key Signaling Protein Vital for Regulating Immune Cell Inflammatory Response

North Brunswick-based BioNJ Member BioAegis Therapeutics announced that a previously unknown role of gelsolin in a signaling system in macrophages was published in the Journal of Immunology. Researchers have detected a biological phenomenon called quorum sensing in macrophages. The paper, “Quorum Sensing by Gelsolin Regulates Programmed Cell Death 4 Expression and a Density-Dependent Phenotype in Macrophages.” Quorum-sensing, a property associated with cell density and well-known in bacteria for decades has now been discovered in macrophages, innate immune cells responsible for addressing infectious and non-infectious threats. These cells are able to detect their population density and respond by either increasing or decreasing their activity.

Celldex Presents Positive Data on Symptom Control and Quality of Life Measurements that Further Support CDX-0159 Clinical Benefit in Phase 1b Study in Chronic Inducible Urticaria at EADV 2021

Hampton-based BioNJ Member Celldex Therapeutics announced positive data on measurements of symptom control and quality of life from the company’s ongoing, open label Phase 1b clinical trial of CDX-0159 in patients with antihistamine refractory cold urticaria and symptomatic dermographism, the two most common forms of chronic inducible urticaria. These diseases, which are often severe and debilitating, can significantly impact patients’ lives. CDX-0159 is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity.

Amicus Therapeutics and ARYA Sciences Acquisition Corp IV Announce Formation of a Next-Generation Genetic Medicine Company: “Caritas Therapeutics”

Cranbury-based BioNJ Member Amicus Therapeutics announced its intent to launch a next-generation genetic medicine company, Caritas Therapeutics, Inc., through a definitive business combination agreement pursuant to which the Amicus gene therapy business will be acquired by ARYA Sciences Acquisition Corp, a special purpose acquisition company or SPAC, sponsored by Perceptive Advisors. The transaction will result in two independent publicly traded companies with attractive stand-alone investment profiles. Amicus will become the largest shareholder in Caritas with a ~36% ownership stake (assuming no redemptions by ARYA’s shareholders) and retain co-development and commercialization rights to the Fabry and Pompe gene therapy programs as well as negotiation rights on select future muscular dystrophy programs.

U.S. FDA Accepts Filings for Amicus’ AT-GAA for the Treatment of Pompe Disease

Cranbury-based BioNJ Member Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA, the company’s investigational two-component therapy for the treatment of Pompe disease. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. The BLA and NDA are based on the evaluation of the effects of AT-GAA in Pompe disease patients and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies as well as data from the open-label extension study.

Results Show Long-Lasting and Holistic Improvements in Children With AADC Deficiency Treated With PTC-AADC Gene Therapy

South Plainfield-based BioNJ Member PTC Therapeutics presented a new analysis of five-year results that shows its novel gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, a previously intractable, fatal and devastating rare disorder of the central nervous system. Children treated with PTC-AADC developed motor function and cognitive skills not previously seen, such as holding up their head, sitting or standing with support and communicating, and these persisted for up to 10 years. The new, five-year results show that motor function improvements were sustained, demonstrating that the treatment effect of PTC-AADC is durable, which is highly important for a single administration therapy for a chronic, fatal disease.

Phathom Pharmaceuticals Announces Results from VONO-103, a Phase 1 Study Evaluating Gastric Acid Inhibition of Vonoprazan and Lansoprazole (PREVACID®)

Florham Park-based BioNJ Member Phathom Pharmaceuticals reported results from VONO-103, a Phase 1 trial evaluating the effects of vonoprazan 20 mg once daily (“QD”) and lansoprazole 30 mg QD in healthy U.S. subjects. In the study, vonoprazan demonstrated significantly greater acid inhibition as compared to lansoprazole. The study treatments were generally well tolerated with no serious adverse events reported. The primary pharmacodynamic endpoints of VONO-103 were mean gastric pH over twenty-four hours (“mean 24-hour pH value”) and the percentage of time with gastric pH above 4 (pH>4 holding time ratio or “pH>4 HTR”) on Days 1 and 7. Gastric pH levels are measured on a logarithmic scale from 0.0 to 14.0, in which each point represents a 10-fold change in acidity and higher pH values represent lower acidity.

Rafael Pharmaceuticals Announces APOLLO613 Phase I/II Clinical Trial of CPI-613® (devimistat) in Combination With Hydroxychloroquine in Patients With Relapsed Clear Cell Sarcoma Begins Enrollment at City of Hope in Duarte, California

Cranbury-based BioNJ Member Rafael Pharmaceuticals, Inc. announced that the Phase 1 clinical trial of CPI-613® (devimistat) in combination with hydroxychloroquine in patients with clear cell sarcoma is open for enrollment. The clinical trial will begin enrolling patients at City of Hope in Duarte, California, with other sites across the country to quickly follow. “Clear cell sarcoma is truly one of the most challenging sarcomas to treat, as it often spreads quickly to other parts of the body and prognosis is generally poor,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “The opening of this trial at City of Hope and several other sites across the country has the potential to grant a population with significant unmet medical needs a promising new treatment option.”

Agile Therapeutics Partners With Carli Lloyd, U.S. Women’s Soccer Star and Four-Time Olympian, to Bring Awareness to Women’s Health Topics

Princeton-based BioNJ Member Agile Therapeutics announced a partnership with U.S. Women’s Soccer legend, Carli Lloyd. The partnership with Agile will focus on bringing awareness to women’s health topics, and Agile’s commitment to elevating women’s healthcare. Agile has built its brand around a commitment to addressing women’s health needs. It received approval from the U.S. Food and Drug Administration (FDA) in February 2020 for its first product, Twirla® (levonorgestrel and ethinyl estradiol) transdermal system, a weekly contraceptive patch. It currently has four additional contraceptive products in its pipeline.

All Endpoints Met in Hepion Pharmaceuticals’ Drug-Drug Interaction Study With CRV431

Edison-based BioNJ Member Hepion Pharmaceuticals announced results from a Drug-Drug Interaction (‘DDI’) study with its lead drug candidate, CRV431. CRV431 targets several isoforms of cyclophilins which comprise a family of enzymes involved in processes including collagen production, inflammation, cell injury, cell death, and protein folding. Attenuation of the activities of this multifunctional enzyme family represents a novel approach to the treatment of NASH. The randomized, open label DDI study was conducted in healthy volunteers (n = 24) to determine if single or multiple oral doses of ketoconazole (400 mg) influence the pharmacokinetics (‘PK’) of CRV431 (75 mg) and its major metabolites. The study also examined if single or multiple once daily oral doses of CRV431 effected the PK of midazolam (2 mg IV) and its metabolite, 1-hydroxymethyl midazolam.

Soligenix Announces Accelerated Publication Demonstrating Efficacy and Broad Neutralizing Activity of its COVID-19 Vaccine in Non-Human Primates

Princeton-based BioNJ Member Soligenix, Inc. announced publication of pre-clinical immunogenicity studies for CiVax™ (heat stable COVID-19 vaccine program) demonstrating durable broad-spectrum neutralizing antibody responses, including against the Beta, Gamma and Delta variants of concern. The article, titled "Protein Vaccine Induces a Durable, More Broadly Neutralizing Antibody Response in Macaques than Natural Infection with SARS-CoV-2 P.1," is part of the ongoing collaboration with Axel Lehrer, Ph.D., Associate Professor at the Department of Tropical Medicine, Medical Microbiology and Pharmacology, John A. Burns School of Medicine (JABSOM), University of Hawaiʻi at Mānoa (UHM). Development continues under a non-dilutive $1.5M Small Business Innovation Research (SBIR) grant from the National Institute of Allergy and Infectious Diseases (NIAID) awarded to Soligenix in December 2020.

Otsuka and Sumitomo Dainippon Pharma Announce a Worldwide Collaboration and License Agreement for Four Psychiatry and Neurology Compounds

Princeton-based BioNJ Member Otsuka and Sumitomo Dainippon Pharma announced that they have executed a collaboration and license agreement for worldwide joint development and commercialization of the following four novel candidate compounds currently under development in psychiatry and neurology area by Sumitomo Dainippon Pharma and Sunovion. Under the terms and conditions of this agreement, Sunovion grants Otsuka rights to jointly develop and commercialize the four compounds worldwide. The Sumitomo Dainippon Pharma Group (Sumitomo Dainippon Pharma, Sunovion, Sumitomo Pharmaceuticals (Suzhou) Co., Ltd., and Sumitomo Pharmaceuticals Asia Pacific Pte. Ltd.) and Otsuka will pursue joint development of these compounds.

Helsinn Group and BridgeBio Pharma’s Affiliate QED Therapeutics Announce Health Canada Conditional Approval of TRUSELTIQ™ (infigratinib) for Patients With Cholangiocarcinoma

Iselin-based BioNJ Member Helsinn and BridgeBio Pharma announced that Health Canada has approved TRUSELTIQ™ (infigratinib), a small molecule kinase inhibitor that targets fibroblast growth factor receptor (FGFR), under the Notice of Compliance with Conditions (NOC/c) policy, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma (CCA) with a FGFR2 fusion or other rearrangement. An NOC/c is a form of market approval granted to a product on the basis of promising evidence of clinical effectiveness following review of the submission by Health Canada.

FDA Expands Lilly's ERBITUX® (cetuximab) Label With Combination of BRAFTOVI® (encorafenib) for the Treatment of BRAF V600E Mutation-Positive Metastatic Colorectal Cancer (CRC) After Prior Therapy

Bridgewater-based BioNJ Member Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has granted approval of a new indication for ERBITUX® (cetuximab injection) in combination with BRAFTOVI® (encorafenib), marketed by BioNJ Member Pfizer, Inc., for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy. ERBITUX is the first and only anti-EGFR antibody approved, in combination with encorafenib, for this indication and is based on results from Pfizer's BEACON CRC trial, the only Phase 3 trial to specifically study patients with previously treated metastatic CRC with a BRAF V600E mutation.

Lilly and Susan G. Komen® Partner to Address Disparities in Breast Cancer Outcomes Experienced by Black Women

Bridgewater-based BioNJ Member Eli Lilly and Company and Susan G. Komen®, the world's leading breast cancer organization, launched a new multiyear program to address breast cancer health disparities experienced by Black women in the U.S. Through the partnership, Lilly and Komen will expand existing resources that provide direct support to Black women facing breast cancer to meet their psychosocial needs, provide guidance to credible health information, and access to local services and resources. The partnership will also provide culturally competent patient navigators who will help guide Black women living with breast cancer through the complex health care system to help ensure timely diagnosis and treatment.

OLUMIANT® Significantly Improved Hair Regrowth to at Least 80% Scalp Coverage as Early as 24 Weeks Across First Completed Phase 3 Studies for Alopecia Areata

Bridgewater-based BioNJ Member Eli Lilly and Company announced detailed results from two pivotal Phase 3 trials (BRAVE-AA1 and BRAVE-AA2), which found once-daily OLUMIANT® (baricitinib) 4-mg was superior to placebo in achieving significant scalp hair regrowth as early as 24 weeks in adults with severe alopecia areata (AA) as defined by ≥50% scalp hair loss at baseline. Significant improvements in scalp hair regrowth compared to placebo were achieved at 36 weeks for patients taking OLUMIANT 2-mg and 4-mg oral doses, as previously disclosed in topline findings from BRAVE-AA1 and BRAVE-AA2 earlier this year. In both studies, a greater proportion of patients taking OLUMIANT 4-mg compared to placebo also achieved full regrowth or regrowth with minimal gaps in coverage of eyebrow and eyelash hair at 36 weeks, which were key secondary endpoints of the studies.

At Week 104, 75% of Patients With Ulcerative Colitis Taking Mirikizumab Maintained Symptomatic Remission in Phase 2 Study

Patients taking mirikizumab for their moderately to severely active ulcerative colitis (UC), were in symptomatic remission for up to two years as demonstrated in new results from Bridgewater-based BioNJ Member Eli Lilly and Company's Phase 2 study. Symptomatic remission is defined as no more than two bowel movements more than an individual's normal bowel frequency and no blood in their stool. In a separate, post-hoc analysis of patients treated with mirikizumab in this Phase 2 study, an absence of bowel urgency (the sudden or immediate need for a bowel movement) in patients with moderately to severely active UC at 12 weeks was strongly associated with reduced levels of inflammatory biomarkers, an indicator of disease activity.

Ready for the Challenge? Boehringer Ingelheim and Eli Lilly and Company Launch First Ever Online Game on Cardio-Renal-Metabolic Conditions for Healthcare Professionals

Bridgewater-based BioNJ Member Eli Lilly and Company announced the launch of CRMSynced™, an educational initiative that uses gamification to encourage healthcare professionals (HCPs) to prioritize a holistic approach to care for cardio-renal-metabolic (C-R-M) conditions. Accounting for up to 20 million deaths in the U.S. annually, C-R-M conditions, including diabetes, heart disease and chronic kidney disease, are the leading causes of death worldwide. The cardiovascular, renal (kidney) and metabolic systems are closely interconnected and share many of the same disease-related pathways. Dysfunction in one system may accelerate the onset of dysfunction in others, resulting in the progression of linked diseases such as type 2 diabetes, heart failure and chronic kidney disease.

Johnson & Johnson Announces Submission of Emergency Use Authorization Amendment to the U.S. FDA to Support Booster of its Single-Shot COVID-19 Vaccine

New Brunswick-based BioNJ Member Johnson & Johnson announced it has submitted data to the U.S. Food and Drug Administration (FDA) to support use of a booster shot of the Johnson & Johnson COVID-19 vaccine in individuals 18 years of age and older. The submission includes recent results from the Phase 3 ENSEMBLE 2 study that found a booster of the Johnson & Johnson COVID-19 vaccine given 56 days after the primary dose provided 94 percent protection against symptomatic (moderate to severe/critical) COVID-19 in the U.S. (CI, 58%-100%) and 100 percent protection (CI, 33%-100%) against severe/critical COVID-19, at least 14 days post-booster vaccination. Also part of the submission is Phase 1/2a data showing that when a booster of the Johnson & Johnson COVID-19 vaccine was given six months after the single shot, antibody levels increased nine-fold one week after the booster and continued to climb to 12-fold higher four weeks after the booster.

New Analysis Presented by Janssen at United European Gastroenterology Week (UEGW) Demonstrates Long-Term Safety Profile for STELARA®(ustekinumab) in Older Patients Across Approved Indications

Titusville-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced a new analysis of STELARA® (ustekinumab) pooled safety data from 13 clinical studies across approved indications, showing rates of key safety events among adults 60 years and older treated with STELARA for up to five years were similar to rates observed with placebo during the control phase of these trials. Approved indications included adults with moderately to severely active Crohn’s disease (CD), moderately to severely active ulcerative colitis (UC), moderate to severe plaque psoriasis (PsO) and active psoriatic arthritis (PsA). These data represent an important patient population as patients 60 years old and older are at a higher risk of disease and therapy associated morbidity, which can result in disease management challenges.

New Phase 3 Data Suggest Positive Effect and Show Similar Safety With XARELTO® (rivaroxaban) Compared to Aspirin in Pediatric Fontan Procedure Patients at Risk for Blood Clots and Blood Clot-Related Events

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced new data from the Phase 3 UNIVERSE study showing treatment with XARELTO® (rivaroxaban) in an oral suspension formulation, compared to treatment with aspirin, was associated with numerically fewer blood clots and clinical events strongly associated with blood clots in pediatric patients (aged 2-8 years) who have undergone the Fontan procedure. These findings, which were published in the Journal of the American Heart Association and included in a recent New Drug Application submitted to the U.S. Food and Drug Administration, also found treatment with XARELTO® was associated with a similar safety profile compared to aspirin.

Janssen Announces Start of Phase 3 Trial for Investigational Respiratory Syncytial Virus (RSV) Vaccine in Older Adults

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced the initiation of its Phase 3 EVERGREEN study. The study will evaluate the efficacy, safety and immunogenicity of Janssen’s investigational adult vaccine against lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV), when compared with placebo in approximately 23,000 adults aged 60 years and older throughout North America and a selection of countries across Europe, Asia and the Southern Hemisphere. The EVERGREEN study was initiated based on positive results from the Phase 2b CYPRESS study, the first large study evaluating the efficacy and safety of Janssen’s investigational RSV vaccine against RSV-associated LRTD in vaccinated adults aged 65 and older in the United States.

Janssen Announces Phase 2b Data Demonstrating its Investigational RSV Adult Vaccine Provided 80% Protection Against Lower Respiratory Infections in Older Adults

Raritan-based BioNJ Member Janssen Pharmaceutical Companies of Johnson & Johnson announced that its investigational respiratory syncytial virus (RSV) vaccine candidate was highly effective in protecting against lower respiratory tract disease (LRTD) caused by RSV, demonstrating vaccine efficacy of 80 percent (CI, 52.2-92.9%) in adults aged 65 and older. The CYPRESS study met its primary and secondary endpoints, with the investigational RSV adult vaccine demonstrating efficacy of 80 percent (CI, 52.2-92.9%) against confirmed RSV-associated LRTD. The investigational RSV adult vaccine also demonstrated efficacy of 70 percent (CI, 42.7-85.1%) against any symptomatic RSV-associated acute respiratory infection (ARI).

FDA Approves Repatha® (evolocumab) in Pediatric Patients Age 10 and Older With Heterozygous Familial Hypercholesterolemia

Berkeley Heights-based BioNJ Member Amgen announced that the U.S. Food and Drug Administration (FDA) has approved Repatha® (evolocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C. HeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide. High levels of LDL-C starting at birth accelerate the development of atherosclerotic cardiovascular disease, leading to an overall increased risk of cardiovascular events, including heart attack and other vascular conditions, at an earlier age.

Amgen and Kyowa Kirin Present Positive Late-Breaking Data from Phase 2 Study of AMG 451/KHK4083 in Adult Patients With Moderate-to-Severe Atopic Dermatitis at EADV Congress

Berkeley Heights-based BioNJ Member Amgen and BioNJ Member Kyowa Kirin announced positive data from a Phase 2 study of AMG 451/KHK4083. AMG 451/KHK4083 is a potential first-in-class anti-OX40 fully human monoclonal antibody in development for the treatment of moderate-to-severe atopic dermatitis. The Phase 2, multicenter, randomized, double-blind, placebo-controlled trial investigated the efficacy and safety of AMG 451/KHK4083 in adults with moderate-to-severe atopic dermatitis who were not adequately controlled with topical agents. The Phase 2, multicenter, randomized, double-blind, placebo-controlled trial investigated the efficacy and safety of AMG 451/KHK4083 in adults with moderate-to-severe atopic dermatitis who were not adequately controlled with topical agents.

New, Late-Breaking Data at EADV Highlights Emerging Clinical Profile of Amlitelimab (Formerly KY1005) in Adults With Inadequately Controlled Moderate-to-Severe Atopic Dermatitis

Bridgewater-based BioNJ Member Sanofi announced positive results from a Phase 2a study evaluating the safety and efficacy of amlitelimab, a human monoclonal antibody targeting key immune system regulator OX40-Ligand. In the study, amlitelimab showed significant improvements in signs and symptoms of moderate-to-severe atopic dermatitis with a well-tolerated safety profile in adults whose disease cannot be adequately controlled with topical medications or for whom topical medications are not a recommended treatment approach.

Sanofi Announces Positive Phase 1/2 Study Interim Results for its First Mrna-Based Vaccine Candidate

Bridgewater-based BioNJ Member Sanofi announced positive interim results from a Phase 1/2 study mRNA-based COVID-19 vaccine candidate confirm the potential of recently acquired Translate Bio’s messenger RNA (mRNA) and lipid nanoparticle (LNP) platform and support Sanofi’s mRNA strategy. The initial data from Phase 1/2 showed neutralizing antibody seroconversion (defined as 4-fold increase vs baseline) in 91% to 100% of study participants, two weeks after a second injection, across all 3 dosages tested. No safety concern has been observed and the tolerability profile is comparable to that of other unmodified mRNA COVID-19 vaccines.

Evotec Expands Neuroscience Collaboration With Bristol Myers Squibb to Include Novel Cell Type

Princeton-based BioNJ Member Evotec SE announced that its collaboration with BioNJ Member Bristol Myers Squibb has been expanded to include a new cell type, triggering a payment of $ 9.0 m to Evotec. The collaboration was initiated in December 2016 with the goal of identifying disease-modifying treatments for a broad range of neurodegenerative diseases. Currently approved drugs only offer short-term management of the patients’ symptoms and there is tremendous unmet medical need for therapeutic options that slow down or reverse disease progression. The collaboration pursues an innovative approach to the discovery and development of novel medicines by leveraging Evotec’s iPSC platform using patient-derived disease models, which is one of the largest and most sophisticated platforms in the industry.

U.S. Food and Drug Administration Accepts Bristol Myers Squibb’s Applications for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + Chemotherapy for Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Applications (sBLA) for both Opdivo®(nivolumab) in combination with Yervoy (ipilimumab) and Opdivo in combination with fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC), based on results from the CheckMate -648 trial. The FDA assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 28, 2022.

European Medicines Agency Validates Bristol Myers Squibb’s Application for Mavacamten for the Treatment of Obstructive Hypertrophic Cardiomyopathy

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for mavacamten, an investigational, first-in-class cardiac myosin inhibitor, for the treatment of patients with obstructive hypertrophic cardiomyopathy (obstructive HCM). The application is based on the results of the pivotal Phase 3 EXPLORER-HCM trial, which evaluated mavacamten in patients with symptomatic obstructive HCM versus placebo. Results from the trial showed mavacamten demonstrated a clear treatment effect, with clinically meaningful improvements in symptoms, functional status and quality of life, as well as the ability to relieve left ventricular outflow tract obstruction.

European Medicines Agency Validates Bristol Myers Squibb’s Application for LAG-3-Blocking Antibody Relatlimab and Nivolumab Fixed-Dose Combination as First-Line Treatment for Patients With Unresectable or Metastatic Melanoma

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for the LAG-3-blocking antibody relatlimab and nivolumab fixed-dose combination for first-line treatment of adult and pediatric patients (12 years and older and weighing at least 40 kg) with advanced (unresectable or metastatic) melanoma. The filing was based on the efficacy and safety results from the Phase 2/3 RELATIVITY-047 trial, which is the first trial to demonstrate a statistically significant and clinically meaningful progression-free survival benefit of a combination therapy over standard of care anti-PD-1 monotherapy in metastatic melanoma.

Novartis Data Show Achieving Complete Control of Chronic Spontaneous Urticaria (CSU) Improves Overall Quality of Life, as Reported by Patients

East Hanover-based BioNJ Member Novartis announced new analysis from a Phase IIb study demonstrating the importance of achieving complete control of chronic spontaneous urticaria (CSU) symptoms in improving overall health-related quality of life (HRQoL) for patients. Complete control of symptoms brings enormous benefit to people with CSU and is associated with improvements in key HRQoL measures such as overall quality of life, sleep interference, activity interference and work impairment. There is a marked decline in these improvements to quality of life even when patients are living with only a low level of CSU symptoms. Complete control of CSU symptoms was assessed by a composite of patient reported outcomes (PROs) and was shown to be more likely achieved and sustained with ligelizumab than Xolair® (omalizumab) or placebo.

New Data Reinforce Efficacy and Convenience of Novartis Cosentyx® (Secukinumab) 300 Mg Autoinjector in Adults With Psoriasis

East Hanover-based BioNJ Member Novartis announced data from an international Phase IIIb study, which showed treatment with Cosentyx® (secukinumab) 300 mg in a 2 mL autoinjector (UnoReady® pen) resulted in high efficacy and convenient administration in adults with moderate to severe plaque psoriasis. The MATURE study assessed the use of a Cosentyx 300 mg autoinjector, versus two 150 mg pre-filled syringes or placebo. Patients using the 300 mg autoinjector reported significantly improved skin clearance measured by Psoriasis Area and Severity Index (PASI) 75 and 90 versus placebo.

Novartis and Medicines for Malaria Venture Report Positive Results for Phase 2b Study of Novel Ganaplacide/Lumefantrine Combination in Children With Malaria

East Hanover-based BioNJ Member Novartis and MMV have reported positive results of a new non-artemisinin combination in a Phase 2b study. The study tested ganaplacide, a novel agent with an entirely new mechanism of action, in combination with a new formulation of lumefantrine that is optimised for once daily dosing. This combination has the potential not only to clear malaria infection, including artemisinin resistant strains, but also to block the transmission of the malaria parasite. This Phase 2b study was an open label randomized controlled study conducted in two parts and which enrolled more than 500 patients with acute uncomplicated malaria due to Plasmodium falciparum infection.

Novartis Data Show Rapid and Effective Disease Activity Control With Remibrutinib (LOU064) in Patients With Chronic Spontaneous Urticaria

East Hanover-based BioNJ Member Novartis announced positive Phase IIb data showing remibrutinib (LOU064), a potentially best-in-class oral BTK inhibitor, demonstrated rapid and effective disease control in patients with inadequately controlled chronic spontaneous urticaria (CSU). This randomized, double-blind, placebo-controlled study (NCT03926611) evaluated the efficacy and safety of remibrutinib over 12 weeks in patients inadequately controlled with antihistamines. Patients (n=311) were randomized to placebo or different doses of remibrutinib, taken orally. The primary endpoint was achieved with remibrutinib showing a statistically significant dose-response compared to placebo with respect to change from baseline in UAS7 score at Week 4.

Pfizer and BioNTech Submit Initial Data to U.S. FDA from Pivotal Trial of COVID-19 Vaccine in Children 5 to <12 Years of Age

BioNJ Member Pfizer, with offices in Peapack, announced positive topline results from the pivotal trial on September 20, 2021. In the trial, which included 2,268 participants 5 to <12 years of age, the vaccine demonstrated a favorable safety profile and elicited robust neutralizing antibody responses using a two-dose regimen of 10 μg doses. These results – the first from a pivotal trial of any COVID-19 vaccine in this age group – were comparable to those recorded in a previous Pfizer-BioNTech study in people 16 to 25 years of age, who were immunized with 30 μg doses. The 10 μg dose was carefully selected as the preferred dose for safety, tolerability and immunogenicity in children 5 to <12 years of age.

Pfizer Starts Global Phase 2/3 Epic-Pep Study of Novel COVID-19 Oral Antiviral Candidate for Post-Exposure Prophylaxis in Adults

BioNJ Member Pfizer, with offices in Peapack, announced the start of the Phase 2/3 EPIC-PEP (Evaluation of Protease Inhibition for COVID-19 in Post-Exposure Prophylaxis) study to evaluate the investigational novel oral antiviral candidate PF-07321332, co-administered with a low dose of ritonavir, for the prevention of COVID-19 infection. This Phase 2/3 trial is part of a global clinical research program and is enrolling individuals who are at least 18 years old and live in the same household as an individual with a confirmed symptomatic SARS-CoV-2 infection. The Phase 2/3 EPIC-PEP trial is a randomized, double-blind, placebo-controlled study and will enroll up to 2,660 healthy adult participants aged 18 and older. 

Pfizer Starts Study of Mrna-Based Next Generation Flu Vaccine Program

BioNJ Member Pfizer, with offices in Peapack, announced that the first participants have been dosed in a Phase 1 clinical trial to evaluate the safety, tolerability, and immunogenicity of a single dose quadrivalent mRNA vaccine against influenza in healthy adults. Pfizer’s mRNA influenza vaccine program is the first in a planned wave of programs leveraging mRNA technology for influenza. Beyond influenza, the company plans to explore mRNA in other respiratory viruses, including medically appropriate vaccines combinations that could provide protection against more than one respiratory virus, as well as expand to develop mRNA technology in oncology, and genetic diseases.

Valneva and Pfizer Report Further Positive Phase 2 Results, Including Booster Response, for Lyme Disease Vaccine Candidate

BioNJ Member Pfizer, with offices in Peapack, and Valneva SE announced further positive Phase 2 results, including booster response, for Lyme disease vaccine candidate VLA15. The Phase 2 study, VLA15-202, is evaluating the immunogenicity and safety of VLA15 in a Month 0-2-6 vaccination schedule. The study enrolled 246 healthy adults 18 to 65 years of age in the U.S. As announced in October 2020, the study met its primary endpoint of demonstrating that VLA15 was immunogenic across all dose groups tested and elicited high antibody responses across all serotypes (ST1 - ST6) at one month after completion of the primary vaccination series. Continued evaluation at Month 18 showed that antibody titers declined thereafter across all groups, remaining above baseline but confirming the need for a booster strategy.

Positive Top-Line Results of Pfizer’s Phase 3 Study Exploring Coadministration of Prevnar 20™ With Seasonal Flu Vaccine in Older Adults Released

BioNJ Member Pfizer, with offices in Peapack, announced positive top-line results from a Phase 3 study (B7471004) evaluating the safety and immunogenicity of PREVNAR 20™ (Pneumococcal 20-valent Conjugate Vaccine) in adults 65 years of age or older when administered at the same time as the seasonal influenza vaccine (SIIV, Fluad Quadrivalent [adjuvanted], 2020/2021 strains). Responses elicited by PREVNAR 20 for all 20 serotypes and by seasonal influenza vaccine when given together were noninferior (the study’s primary immunogenicity objectives) to those elicited by the vaccines when administered one month apart. Across 66 investigator sites in the United States, a total of 1,796 participants were enrolled and randomized, with 1,727 of participants completing the study.

Japan’s Mhlw Approves Pfizer’s Cibinqo® (Abrocitinib) for Adults and Adolescents With Moderate to Severe Atopic Dermatitis

BioNJ Member Pfizer, with offices in Peapack, announced that the Japanese Ministry of Health, Labour and Welfare (MHLW) has approved CIBINQO®(abrocitinib), an oral, once-daily, Janus kinase 1 (JAK1) inhibitor, for the treatment of moderate to severe atopic dermatitis (AD) in adults and adolescents aged 12 years and older with inadequate response to existing therapies. CIBINQO will be available in Japan in doses of 100mg and 200mg. The approval of CIBINQO in Japan was based on the results from 1,513 patients across four Phase 3 studies, ranging from 12 to 16 weeks of treatment, and a long-term extension study from a robust clinical trial program.Regulatory applications for abrocitinib have been submitted to countries around the world for review, including the United States, Australia and the European Union.

FDA Approves QULIPTA™ (atogepant), the First and Only Oral CGRP Receptor Antagonist Specifically Developed for the Preventive Treatment of Migraine

Madison-based BioNJ Member AbbVie announced that the U.S. Food and Drug Administration (FDA) approved QULIPTA™ (atogepant) for the preventive treatment of episodic migraine in adults. QULIPTA is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) specifically developed for the preventive treatment of migraine. The approval is supported by data from a robust clinical program evaluating the efficacy, safety and tolerability of QULIPTA in nearly 2,000 patients who experienced 4 to 14 migraine days per month, including the pivotal Phase 3 ADVANCE study, the pivotal Phase 2b/3 study and the Phase 3 long-term safety study. Migraine is a complex disease with recurrent attacks that are often incapacitating and characterized by severe, throbbing headache pain as well as compounding associated symptoms like extreme sensitivity to light, sound or nausea.

AbbVie Presents New Analyses Evaluating RINVOQ® (upadacitinib) in Atopic Dermatitis Across Patient Characteristics and Body Regions at EADV Virtual Congress

Madison-based BioNJ Member AbbVie announced new analyses from the Phase 3 RINVOQ® (upadacitinib) atopic dermatitis clinical trial program. One analysis showed a greater proportion of patients treated with RINVOQ (15 mg or 30 mg; once daily) with or without topical corticosteroids achieved 75 percent improvement in the Eczema Area Severity Index (EASI 75) at week 16 compared to placebo, regardless of age, sex, race, weight, disease severity and previous exposure to systemic therapy. An additional analysis showed more patients treated with RINVOQ 30 mg achieved EASI 75 at week 16 compared to dupilumab when measured in four body regions.

AbbVie's RINVOQ® (upadacitinib) Met Primary and All Ranked Secondary Endpoints in Phase 3 Study in Ankylosing Spondylitis

Madison-based BioNJ Member AbbVie announced positive top-line results from the first of two studies of the Phase 3 SELECT-AXIS 2 clinical trial evaluating the efficacy and safety of RINVOQ® (upadacitinib; 15 mg, once daily) in patients with active ankylosing spondylitis (AS) who had an inadequate response to biologic DMARD therapy. In this study, RINVOQ met its primary endpoint of Assessment in SpondyloArthritis International Society (ASAS) 40 response and all ranked secondary endpoints at week 14. Significantly more RINVOQ-treated patients achieved ASAS40 response at week 14 compared to placebo (45 percent versus 18 percent; p<0.0001).

AbbVie Presents Late-Breaking Data on Risankizumab (SKYRIZI®) in Psoriatic Arthritis at the 30th European Academy of Dermatology and Venereology (EADV) Congress

Madison-based BioNJ Member AbbVie presented results from new Phase 3 data analyses of KEEPsAKE-1 and KEEPsAKE-2, evaluating risankizumab (SKYRIZI®, 150 mg) in adults with active psoriatic arthritis for one year (52 weeks). KEEPsAKE-1 included adult patients with active psoriatic arthritis who responded inadequately to non-biologic disease-modifying anti-rheumatic drugs (DMARDs). KEEPsAKE-2 included adult patients with active psoriatic arthritis who had responded inadequately or were intolerant to biologic therapy and/or non-biologic disease-modifying anti-rheumatic drugs (DMARDs). In the first phase of the studies (Period 1), patients were randomized to risankizumab or placebo until week 24.

Merck to Acquire Acceleron Pharma Inc.

Kenilworth-based BioNJ Member Merck & Co. and Acceleron Pharma Inc. announced that the companies have entered into a definitive agreement under which Merck, through a subsidiary, will acquire Acceleron for $180 per share in cash for an approximate total equity value of $11.5 billion. Acceleron is focused on harnessing the power of the transforming growth factor (TGF)-beta superfamily of proteins that is known to play a central role in the regulation of cell growth, differentiation and repair. Acceleron’s lead therapeutic candidate, sotatercept, has a novel mechanism of action with the potential to improve short-term and/or long-term clinical outcomes in patients with pulmonary arterial hypertension (PAH), a progressive and life-threatening blood vessel disorder.

Merck and Ridgeback’s Investigational Oral Antiviral Molnupiravir Reduced the Risk of Hospitalization or Death by Approximately 50 Percent Compared to Placebo for Patients With Mild or Moderate COVID-19 in Positive Interim Analysis of Phase 3 Study

Kenilworth-based BioNJ Member Merck & Co. announced that molnupiravir (MK-4482, EIDD-2801), an investigational oral antiviral medicine, significantly reduced the risk of hospitalization or death at a planned interim analysis of the Phase 3 MOVe-OUT trial in at risk, non-hospitalized adult patients with mild-to-moderate COVID-19. At the interim analysis, molnupiravir reduced the risk of hospitalization or death by approximately 50%; 7.3% of patients who received molnupiravir were either hospitalized or died through Day 29 following randomization (28/385), compared with 14.1% of placebo-treated patients (53/377); p=0.0012. Through Day 29, no deaths were reported in patients who received molnupiravir, as compared to 8 deaths in patients who received placebo.

Merck Announces KEYTRUDA® (pembrolizumab) Met Primary Endpoint of Overall Survival (OS) in Patients With Advanced Hepatocellular Carcinoma Previously Treated With Sorafenib

Kenilworth-based BioNJ Member Merck & Co. announced that the Phase 3 KEYNOTE-394 trial investigating KEYTRUDA, Merck’s anti-PD-1 therapy, in Asian patients with advanced hepatocellular carcinoma (HCC) previously treated with sorafenib met its primary endpoint of overall survival (OS). The study found that treatment with KEYTRUDA plus best supportive care resulted in a statistically significant improvement in OS compared with placebo plus best supportive care. KEYNOTE-394 also met its key secondary endpoints of progression-free survival (PFS) and objective response rate (ORR), with statistically significant improvements for KEYTRUDA compared with placebo. No new safety signals were observed.

LYNPARZA® (olaparib) in Combination With Abiraterone Significantly Delayed Disease Progression in Patients Regardless of Biomarker Status in PROpel Phase 3 Trial in First-Line Metastatic Castration-Resistant Prostate Cancer (mCRPC)

Kenilworth-based BioNJ Member Merck & Co. announced positive results from the Phase 3 PROpel trial, in which LYNPARZA in combination with abiraterone and prednisone demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of radiographic progression-free survival (rPFS) versus abiraterone plus prednisone as a first-line treatment for men with metastatic castration-resistant prostate cancer (mCRPC) with or without homologous recombination repair (HRR) gene mutations. At a planned interim analysis, the Independent Data Monitoring Committee concluded that the trial met the primary endpoint of rPFS in men with mCRPC who had not received treatment in the first-line setting, including new hormonal agents (NHAs) or chemotherapy.

FDA Grants Priority Review to ViiV Healthcare’s New Drug Application for Cabotegravir Long-Acting for Prevention of HIV

ViiV Healthcare, the global specialist HIV company majority owned by Warren-based BioNJ Member GlaxoSmithKline announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for a New Drug Application (NDA) for investigational, injectable cabotegravir long-acting for pre-exposure prophylaxis, or PrEP. The Priority Review designation of cabotegravir long-acting for PrEP builds upon its prior identification as a Breakthrough Therapy by the FDA. If approved, cabotegravir would be the first, long-acting therapy for the prevention of HIV for individuals at risk of sexually acquired HIV-1 infection, who have a negative HIV-1 test prior to initiation. The FDA has set a target approval date of 24 January 2022.

Viiv Healthcare Presents Three-Year Switch Data for Dovato (Dolutegravir/Lamivudine) Confirming Long-Term, Non-Inferior Efficacy With No Virologic Failure Versus Continuation of TAF-Based Regimens of at Least Three Drugs

ViiV Healthcare, the global specialist HIV company majority owned by Warren-based BioNJ Member GlaxoSmithKline presented three-year results from the TANGO study. Findings showed that the 2-drug regimen (2DR) Dovato (dolutegravir/lamivudine) continued to demonstrate non-inferior efficacy and a high barrier to resistance compared to continuation of tenofovir alafenamide fumarate (TAF)-based regimens of at least three drugs in virologically suppressed adults living with HIV-1 who had not experienced prior virologic failure. At three years, no participants on dolutegravir/lamivudine (0% [0/369]) met confirmed protocol-defined virologic failure, versus three participants (<1% [3/372]) on the TAF-based regimen. No resistance mutations were reported in either arm.

Antares Pharma Initiates Phase I Study for ATRS-1902 for Adrenal Crisis Rescue

Ewing-based Antares Pharma announced that it has initiated a Phase 1 study for ATRS- 1902 for adrenal crisis rescue. The development program supports a proposed indication for the treatment of acute adrenal insufficiency, known as adrenal crisis, in adults and adolescents, using a novel proprietary auto-injector platform to deliver a liquid stable formulation of hydrocortisone. The Phase I study is designed to evaluate the safety, tolerability and pharmacokinetics (PK) of a liquid stable formulation of hydrocortisone. “We are pleased to be able to dose the first subjects for this study with a liquid stable dosage that eliminates the need for reconstitution. The opportunity to provide an essential treatment that can be easily administered for a potentially life-threatening situation remains a core focus of our research and development efforts,” said Dr. Peter Richardson, MRCP (UK), EVP, Research and Development and Chief Medical Officer.

New Regen-Cov™ (Casirivimab and Imdevimab) Data Show Supportive Results in Patients Hospitalized With COVID-19

Basking Ridge-based Regeneron Pharmaceuticals announced that a trial assessing investigational REGEN-COV™ (casirivimab and imdevimab) in patients hospitalized with COVID-19 met its primary endpoint. Trial results show that REGEN-COV significantly reduced viral load in patients hospitalized with COVID-19 who entered the trial without having mounted their own antibody response (seronegative) and required low-flow or no supplemental oxygen (p=0.0172). The trial also had clinical results supportive of the much larger UK RECOVERY trial in hospitalized patients, with numeric improvements observed across all clinical endpoints assessed.

FDA Accepts Libtayo® (Cemiplimab-Rwlc) for Priority Review for Advanced Cervical Cancer

Basking Ridge-based Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the supplemental Biologics License Application (sBLA) for PD-1 inhibitor Libtayo® (cemiplimab-rwlc) to treat patients with recurrent or metastatic cervical cancer whose disease progressed on or after chemotherapy. The target action date for the FDA decision is January 30, 2022. The sBLA is also being reviewed under the FDA's Project Orbis initiative, which will allow for concurrent review by participating health authorities in Australia, Brazil, Canada and Switzerland. Additional global regulatory submissions are planned, including in the European Union (EU) by the end of 2021.

New England Journal of Medicine Publishes Positive Phase 3 Trial Results for Regen-Cov™ (Casirivimab and Imdevimab) to Treat Covid-19

Basking Ridge-based Regeneron Pharmaceuticals announced that the New England Journal of Medicine published positive detailed results from a Phase 3 trial that assessed the ability of REGEN-COV™ (casirivimab and imdevimab) to treat COVID-19 in infected high-risk non-hospitalized patients (outpatients). The trial met its primary and all secondary endpoints and showed treatment with REGEN-COV significantly reduced the risk of hospitalization or death, with a safety profile consistent with previously reported data. REGEN-COV is an investigational medicine authorized by the U.S. Food and Drug Administration (FDA) under an emergency use authorization to treat people who are at high risk of serious consequences from COVID-19, including those who are already infected (non-hospitalized) or those in certain post-exposure prophylaxis settings. 

LEO Pharma Presents New Adtralza® (Tralokinumab) Data Following Long-Term Continuous Treatment at EADV 30th Congress

Madison-based LEO Pharma announced results showing that after two years of continuous treatment with Adtralza, adult patients with moderate-to-severe atopic dermatitis maintained improvements in signs and symptoms, itch severity and sleep interference. The interim analysis investigated continued treatment with Adtralza as well as the ability to regain response after pausing and reinitiating Adtralza. Long-term efficacy outcomes were assessed in patients (n=345) who had received two years of treatment with Adtralza, including the full 52 weeks in the pivotal Phase 3 parent trials (ECZTRA 1 and 2) and 56 weeks in the ECZTEND trial.

Fully Human Anti-TNFα Monoclonal Antibody HUMIRA® Obtains Additional Approval for High-Dose Regimen of Ulcerative Colitis in Adult Patients and for New Regimen in Pediatric Patients

Woodcliff Lake-based Eisai Co., Ltd, BioNJ Member AbbVie GK and EA Pharma Co., Ltd. announced the additional approval for a high-dose regimen in adult patients with ulcerative colitis and for a new regimen in pediatric patients regarding fully Human Anti-TNFα Monoclonal Antibody HUMIRA® (generic name, adalimumab [recombinant]; “HUMIRA”). This approval allows for 40 mg weekly treatment or 80 mg biweekly treatment in addition to conventional 40 mg biweekly treatment as a remission maintenance therapy, which is expected to maintain remission in many patients. Furthermore, by having been added as a treatment option for ulcerative colitis in pediatric patients, it is expected that as the first at-home/self-injectable drug in Japan for pediatric patients with ulcerative colitis, HUMIRA will improve convenience for pediatric patients and their guardians in addition to reducing burdens due to hospital visits.

Eisai Initiates Rolling Submission to the U.S. FDA for Biologics License Application of Lecanemab (Ban2401) for Early Alzheimer’s Disease Under the Accelerated Approval Pathway

Woodcliff Lake-based Eisai Co., Ltd and Biogen Inc. announced that Eisai has initiated a rolling submission to the U.S. Food and Drug Administration (FDA) of a Biologics License Application (BLA) for lecanemab (BAN2401), the company’s investigational anti-amyloid beta (Aβ) protofibril antibody, for the treatment of early Alzheimer’s disease (early AD). The BLA is being submitted under the accelerated approval pathway and is primarily based on clinical, biomarker and safety data from the Phase 2b clinical trial (Study 201) in people with early AD and confirmed amyloid pathology. The lecanemab Phase 2b trial results demonstrated a high degree of Aβ plaque lowering and consistent reduction of clinical decline across several clinical endpoints.

ENHERTU® Granted Breakthrough Therapy Designation in U.S. for Patients With HER2 Positive Metastatic Breast Cancer Treated With One or More Prior Anti-HER2-Based Regimens

Basking Ridge-based Daiichi Sankyo announced the U.S. Food and Drug Administration (FDA) has granted ENHERTU® (fam-trastuzumab deruxtecan-nxki) Breakthrough Therapy Designation (BTD) in the U.S. for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received one or more prior anti-HER2-based regimens. ENHERTU is a HER2 directed antibody drug conjugate (ADC) jointly developed by Daiichi Sankyo Company, Limited, and AstraZeneca. “With the unprecedented data recently reported from the DESTINY Breast03 trial, we look forward to working closely with the FDA to bring ENHERTU to patients who have been previously treated for HER2 positive metastatic breast cancer as soon as possible,” said Ken Takeshita, M.D., Global Head, R&D, Daiichi Sankyo.

Certara Completes Acquisition of SaaS Software Company

Princeton-based Certara has completed its acquisition of Pinnacle 21, a provider of Software-as-a-Service solutions for clinical data, regulatory compliance and related sectors. “We are pleased to welcome Pinnacle 21’s talented team to Certara. With Pinnacle 21’s software for high-quality clinical data standardization, we can together provide a broader portfolio of industry-leading software and technology-driven services to our customers worldwide. This transaction amplifies our capabilities to inform critical decisions that further de-risk and expedite the drug development process.”

People in the News

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PsychoGenics Chief Innovation Officer Honored for Scientific Excellence

Paramus-based BioNJ Member PsychoGenics announces that Daniela Brunner Ph.D., Chief Innovation Officer, was named the recipient of Great Minds in STEM (GMiS) Scientist of the Year Award (GMiS 2021 Award Winners). Dr. Brunner was recognized for her contribution to the development of PsychoGenics’ AI-based, phenotypic platforms, including its transformative SmartCube® technology that predicts the therapeutic potential of compounds for neuropsychiatric disorders. Dr. Brunner joined PsychoGenics in 1999 where she developed preclinical tests for cognition, neurodegeneration, autism, among others. She conceived of the SmartCube discovery system and worked on the development of PsychoGenics’ AI technologies.

Tris Pharma® Hires Seasoned Business Development Executive Anthony Amato Joins as Executive Vice President Business Development – Generics Division

Monmouth Junction-based Tris Pharma, Inc. announced that Anthony Amato has joined the company as Executive Vice President Business Development – Generics Division. Mr. Amato's expertise includes sourcing and executing valuable transactions, assessing market trends, cultivating successful partnerships, and training and overseeing global cross-functional teams. His 20+ years of Business Development experience includes leadership roles in companies such as Hudson Healthcare Partners, Alvogen, Lupin Pharmaceuticals, Quadrant Capital Advisors, LLC and Barr Laboratories. 

NJBIZ Conversations: Dr. Abdul Gaffar and Peter Cho

NJBIZ Editor Jeff Kanige speaks with Dr. Abdul Gaffar and Peter Cho, co-founders of Salvacion-USA, an Englewood Cliffs company that is developing a nasal spray used to prevent COVID-19 infections, Covixyl-V. Dr. Gaffar, who was inducted into the New Jersey Inventors Hall of Fame in 2001 for his work in producing the first antibacterial toothpaste, and Mr. Cho discussed the product, what it would mean for the fight against the pandemic and how quickly Salvacion would be able to scale up production at its manufacturing facility if an EUA is issued.

Bayer Names New External Communications Exec

Whippany-based Bayer has named a new Director of U.S. External Communications. Nicole Hayes brings extensive agency and corporate communications experience to Bayer, where she will be primarily responsible for media relations. She will report to Christopher Loder, Vice President and Head of External Communications for Bayer U.S. “We are fortunate to have someone of Nicole’s knowledge and experience join our Bayer team,” Mr. Loder said. “Nicole has done stellar work overseeing high-profile consumer brands, and she will bring innovation and creativity to Bayer.” Before joining the company, Ms. Hayes helped lead external communications for the Bountiful Co.

Bayer Appoints New General Managers for Nutritionals and Digestive Health Businesses

Whippany-based Bayer has appointed new General Managers to lead its Nutritionals and Digestive Health businesses, two of its fastest growing categories. After 16 years at Bayer, Lisa Perez is being named General Manager and Vice President Marketing – Nutritionals. In addition, David Ball has been appointed General Manager and Vice President Marketing – Digestive Health. Ms. Perez currently serves as Senior Director Marketing, Pain/Cardio and has been instrumental in delivering some of the boldest marketing campaigns for Consumer Health. Mr. Ball currently serves as Care/of Retail Go-to-Market Lead responsible for driving the launch of Care/of into retail, developing the go-to market strategies for the retail class of trade, as well as broader Marketing and Strategy for the company.

Cormedix CEO Retires; CFO to Serve in Interim Role During Search

Berkeley Heights-based CorMedix Inc. announced a number of leadership changes, including the retirement of its CEO. The company said CEO Khoso Baluch is retiring, and has also resigned from the board of directors. In addition, John Armstrong, Executive Vice President for Technical Operations, is retiring. Dr. Matt David, EVP and Chief Financial Officer, will serve as interim CEO, while remaining in his CFO post. Phoebe Mounts, General Counsel and Head of Regulatory, Compliance and Legal, will have direct authority over the Technical Operations Group. The board of the company is initiating a search process for a new CEO. Mr. Baluch will serve in an advisory capacity during the transition.

Certara Creates Chief Commercial Officer Position, Names Veteran Pharma Exec

Princeton-based Certara Inc. has named a new Chief Commercial Officer. Drayton T. Virkler has been named to the newly created position, in which he will drive the company’s global commercial strategy and expand its business development team, according to a news release. Mr. Virkler, who has more than 20 years of experience in the pharmaceutical industry, was most recently Vice President and Global Head of Sales for Iqvia’s Real World Solutions Global Business Unit. “Drayton’s extensive leadership experience and commercial knowledge will be invaluable as we continue increasing adoption of biosimulation and other technologies to transform the drug development process,” said CEO William F. Feehery.

Funding

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Launch Lane Approved to Participate in NJ Accelerate Program

The University City Science Center’s Launch Lane has been approved to participate in the NJ Accelerate program, the New Jersey Economic Development Authority announced. The University City Science Center’s Launch Lane is based in Philadelphia — but is still eligible for the EDA program, which matches investments made by an approved accelerator into start-ups that locate in New Jersey within six months after graduating from the participating accelerator’s program. The EDA’s match will be in the form of a direct loan up to $250,000. A 5% match bonus is available for companies that are certified as women- or minority-owned in New Jersey. The EDA will also provide rent support for up to six months if a company locates in an approved NJ Ignite collaborative workspace.

Academia/Institutions/Incubators

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Liberty Science Center to Break Ground On SciTech Scity Oct. 22, 2021

In addressing why America would go to the Moon, President John F. Kennedy said, “we do these things not because they are easy but because they are hard, because the goal will serve to organize and measure the best of our energies and skills…” The same creative vision and determination is driving Liberty Science Center in developing the 30-acre “City of Tomorrow” innovation campus called SciTech Scity that aims to launch and grow world-changing science and technology companies and reimagine public school science education. SciTech Scity will bring together scientists, community leaders, teachers, students and other thinkers and doers.

100 NJ Students Named to Prestige STEM Program

The Governor’s STEM Scholars announced their 2021-2022 class, made up of 100 high school and college STEM students from across the Garden State. The Governor’s STEM Scholars is a public-private partnership between the Research & Development Council of New Jersey, the Office of the Governor, the New Jersey Department of Education, the New Jersey Secretary of Higher Education and public and private research institutions based in New Jersey. The Governor’s STEM Scholars believes that maintaining a pipeline of talented individuals is critical to maintaining excellence in innovation. In recognition of this and to counter the State’s brain drain, the Governor’s STEM Scholars was created to engage the next generation of research and innovation leaders in the State’s vast STEM economy early.

NJII, Institute for Life Science Entrepreneurship Win $50K SBA Competition Awards

Two New Jersey organizations were among the winners of the U.S. Small Business Administration’s Growth Accelerator Fund Competition and Small Business Innovation Research Catalyst competition. New Jersey Innovation Institute and the Institute for Life Science Entrepreneurship each won $50,000 as part of the Growth Accelerator Fund Competition program track. That element of the contest includes entries from accelerators and incubators that target assistance to STEM and research & development entrepreneurs from underrepresented groups, including women, people of color, rural and veteran entrepreneurs. “With the wide range of startups and innovators who are working on cutting-edge STEM/R&D ideas here in the Garden State, these awards will help provide additional access for these small businesses to technical support and capital,” said New Jersey District Director Al Titone.

The Unstoppable Innovation Ventures Engine

It’s been a record year for innovation at Rutgers University, and for the Innovation Ventures team in the Office for Research, despite the challenges and new reality that came with the COVID-19 pandemic. Even limited access to laboratories, countless Zoom meetings and the burnout many experienced over the past year could not stop the Rutgers Research and Innovation engine. In Fiscal Year 2021, 64 U.S. patents and 128 global patents were issued for Rutgers innovations, 154 inventions were disclosed, and 400 agreements were negotiated and signed through the efforts of the Innovation Ventures team bringing a record $42.6 million in licensing revenue to Rutgers. Also, five new start-ups were formed, bringing the total number of active Rutgers start-ups to 88.

Rutgers Cancer Institute Research: Abdominal Fat Linked to Worse Outcomes for Black Breast Cancer Survivors

Findings from a recent population-based cohort study show that Black women diagnosed with breast cancer who also have central obesity, which means excess body fat in the abdominal area, were more likely to die from breast cancer or any other cause than similar women who didn’t have central obesity. Elisa V. Bandera, M.D., Ph.D., Chief of Cancer Epidemiology and Health Outcomes and Co-leader of the Cancer Prevention and Control Program at Rutgers Cancer Institute of New Jersey, Professor of Medicine at Rutgers Robert Wood Johnson Medical School, and Professor of Epidemiology at Rutgers School of Public Health, is lead author of the study.

Hackensack Meridian Names Chief Information, Digital Engagement Officer

Hackensack Meridian Health has named a new Chief Information and Digital Engagement Officer. Kash Patel, who will also serve as an Executive Vice President, has more than 25 years of experience in technology leadership in the health care industry, with start-ups and with multinational corporations. Mr. Patel joins HMH from Penn Medicine in Philadelphia, where he was Vice President and Chief Digital Technology Officer. Prior to Penn Medicine, he was with Mount Sinai Health System in New York, where he was Vice President, Population Health and Analytics.

RWJBH to Rename Flagship Facility Cooperman Barnabas Medical Center in Honor of Historic $100M Donation

Philanthropists Leon and Toby Cooperman announced that the Cooperman Family Foundation is making a historic $100 million donation to Saint Barnabas Medical Center in Livingston. In honor of the family’s gift, the largest ever given to a hospital in New Jersey, RWJBarnabas Health will rename the facility Cooperman Barnabas Medical Center. The Coopermans, who live and have raised their family in Essex County, have an extensive history of philanthropy in a number of areas, but to this Barnabas facility in particular. The couple has donated more than 100 times to the medical center previously, including a $25 million gift in 2014 that provided the catalyst for a vast expansion of the hospital.

Events

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Pathways to Success in Biopharma Virtual Graduate Summit and Career Expo October 27–28, 2021

PhRMA will be hosting a two-day, free virtual summit for graduate students, postdoctoral trainees, faculty, PhRMA member companies and other community partners to learn about career pathways and increasing diverse talent within the industry. Join us online to hear about the industry’s latest innovations, learn about career options and connect with the leading biopharma research companies.

Thought Leadership from BioNJ Members

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Navigating the Life Science and Health Care Funding Landscape

A White Paper by BioNJ Member J.P. Morgan

Life science and health care companies have many avenues available to raise capital at various stages, each with its own benefits and considerations. Savvy founders and start-up leaders should understand which asset class can offer the best opportunity to advance and support their company’s success. Across all sectors – therapeutics, diagnostics, tools, medical devices and digital health – the approach you take today to create your syndicate of investors will impact all future funding rounds.

BioNJ Member Services Provider Directory

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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 KMinton@BioNJ.org, or 609-890-3185. Find providers in these categories:




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Please contact BioNJ with any questions.

BioNJ@BioNJ.org  | 609-890-3185 | www.BioNJ.org

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