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Welcome to the latest edition of the BioLines Weekender...
The Senate Parliamentarian will be reviewing a new version of the Build Back Better package, now called the Inflation Reduction Act of 2022, following an agreement between Senators Manchin and Schumer on Wednesday, July 27. The new legislation would be more expansive than the preceding version, and continues to include provisions associated with drug pricing. The Senate is expected to consider the legislation sometime next week, contingent on the findings of the Parliamentarian regarding whether it would be viable for the reconciliation process, which requires only a simple majority vote but must have some direct budgetary relevance.
This new legislation notably does not include any meaningful provisions associated with the business practices of Pharmacy Benefits Managers (PBMs), would eliminate tens of thousands of jobs across the many critical life sciences companies throughout the State — and, most importantly, would fail to accomplish the goal of making treatments more accessible and affordable to Patients. Put simply, these provisions would make it more difficult for Patients to receive the treatments they need while damaging the industry that creates them.
A report published yesterday by Vital Transformation shows that the Build Back Better Act (BBBA), designed to allow the U.S. Government to ‘negotiate’ pricing under a set framework of reductions based upon the amount of time a drug has spent on the market, would have significant, negative effects on Patient access to new therapies as funding would be severely curtailed for research and development.
In fact, the analysis shows that if price controls had been in place between 2012-2021, only 6 of 110 currently approved therapies would have made it to the market because companies would no longer have the necessary funds to invest in research and development and innovative partnerships with small biotech. Further, the proposed legislation will likely lead to the loss of over 590,000 U.S. biopharma jobs — including nearly 45,000 jobs right here in New Jersey.
BioNJ continues to engage in outreach campaigns to our Members of Congress to convey concerns regarding the ramifications that this legislation would have on innovation, Patient access to medications and the broader biopharmaceutical landscape. With the COVID pandemic and other diseases, such as cancer and diabetes impacting the health of Patients, now is the time to strengthen the innovative biopharmaceutical ecosystem and combat health inequity, not harm it through price controls.
To learn more and work with BioNJ in our efforts to protect the future of medical innovation and ensure that Patients have access to the medicines they need, contact us at BioNJ@BioNJ.org or visit www.BioNJ.org. Click here for a copy of Vital Transformation’s report, “Build Back Better Act Total market impact of price controls in Medicare parts D and B.”
Because Patients Can't Wait®,
The BioNJ Team
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Putting Patients First:
The Value of Medical Innovation
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Oncology is the leading therapy area for innovation — in terms of the level of clinical trial activity, number of companies investing in therapeutics, size of the pipeline of therapies in clinical development, novel active substances being launched and the level of expenditure on these drugs. In 2021, during a global pandemic, cancer care continued to be delivered although a backlog in treatment and screenings raised worrying questions. In a record-setting year, more novel cancer medicines became available for the first time than in any year in history, and many of them employ immunology or precision biomarkers to transform the way patients are treated. Adoption of breakthrough medicines and diagnostics is improving outcomes for millions around the world, though broad and equitable access remains a significant challenge to health care stakeholders — including patients.
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John Michael O'Brien was recently appointed as the President and Chief Executive Officer of the National Pharmaceutical Council. In this podcast, John discusses the multiple and varied assaults currently being lobbed at the U.S. innovative biopharmaceutical sector. It outlines how the ecosystem, which successfully created highly effective and innovative medicines for patients, could be rendered inert by the many and various ill-conceived pricing proposals currently emanating from Washington, DC.
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The average age of ALS onset is 55, and people affected experience a progressive loss of motor control, contributing to a median life expectancy of 3-5 years post-symptom onset. While there is currently no cure for ALS, multi-disciplinary care can be employed to help address symptoms, psychological and mobility challenges and decision-making needs related to ALS. The multi-disciplinary care approach to ALS may include a variety of medications, physical and other therapy and special equipment that can cost tens of thousands of dollars. Given the severe nature of the disease, it is essential for people with ALS to have affordable access to care regardless of demographic, economic or geographic differences. Health equity is one of the top priorities across the health care industry. So much so, that the Centers for Medicare & Medicaid Services (CMS) published health equity as the “first pillar” of its 2022 Strategic Plan.
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PhRMA member companies’ continued investment in research and development (R&D) has led to new medicines transforming human health. Innovative biopharmaceutical approaches to treating patients for a broad range of diseases, including chronic and rare conditions, as well as meeting urgent new health challenges like COVID-19 are making a positive impact. From new data released, in 2021 alone, PhRMA member companies invested $102.3 billion in R&D, the highest level of investment on record, according to the 2022 PhRMA member annual survey. The survey also found that in 2021, about one out of every five dollars of revenue was devoted to R&D. Additionally, PhRMA members’ R&D spending accounts for most of the estimated $122.2 billion spent by the entire U.S. biopharmaceutical industry on R&D in 2020.
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Democrats have sent the legislative text of a sweeping proposal for drug price controls to the U.S. Senate parliamentarian, who will report on whether the provisions qualify for the budget reconciliation process that allows lawmakers to evade a filibuster and pass a bill with a simple majority. Democrats expect the various drug-related provisions to reduce federal spending by roughly $300 billion over a decade. That’s a hard number to resist. But the proposal would have other monumental and decidedly harmful implications for American society. There is simply no way for government fiat to force down costs without harming innovation, especially at the start-ups and small biotechs fueled by venture capital (VC).
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Members of Congress continue to push a misguided drug pricing proposal under the guise of negotiation as part of the reconciliation package. This proposal is nothing short of government price setting and, if implemented, would require the government to dictate prices and choose winners and losers when it comes to which diseases are likely to see new treatments. Instead of addressing a broken insurance system, the most recent proposal doubles down on bad policies that will threaten patient access to medicines and future innovations.
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Large, well-funded pharmaceuticals and biotechs get most of the headlines when it comes to cancer research. But smaller companies that toil in the shadows are also making some big advances. Their budgets may not rival those of the heavyweights, but agility and focus enable startups and other growing companies to make significant contributions to the fight against cancer. “With more than 100 types — including rare ones — cancer can be complex and challenging to treat,” said Debbie Hart, founding President and CEO of BioNJ, which advocates for the life sciences industry. “From large pharma to small biotech companies, New Jersey is the No. 2 state for oncology drug development — with an estimated 4,500 current oncology clinical trials and eight of the 15 novel FDA oncology drug approvals in 2021 coming from companies with a footprint in New Jersey.”
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Princeton-based BioNJ Member Soligenix announced that it has received agreement from the U.S. Food & Drug Administration (FDA) on an initial pediatric study plan (iPSP) for HyBryte™ (synthetic hypericin) for the treatment of cutaneous T-cell lymphoma (CTCL). The agreed iPSP stipulates that Soligenix intends on requesting a full waiver of pediatric studies upon submission of a new drug application (NDA). HyBryte™ (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. “This is consistent with decisions by the European Medicines Agency (EMA) and Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom which have previously granted product-specific waivers from the requirement for pediatric studies in applications for marketing authorization of HyBryte™ in the UK and Europe," said Christopher J. Schaber, Ph.D., President & Chief Executive Officer of Soligenix.
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Princeton-based BioNJ Member Soligenix announced that it has signed a worldwide exclusive license to supply its ricin antigen to SERB Pharmaceuticals (SERB), for development of a novel therapeutic treatment against ricin toxin poisoning. There is an unmet need for protection against this highly potent toxin for which there is no vaccine or therapeutic intervention available. In pursuit of a ricin antidote, SERB will leverage its unique broad-spectrum polyclonal antibody platform, gained in its acquisition of BTG Specialty Pharmaceuticals. This specialized manufacturing process generates binding fragments from antibodies that are specific to a given antigen, helping to ensure potency and purity.
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Bedminster-based BioNJ Member Matinas BioPharma announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending MAT2203 for designation as an orphan medicinal product for the treatment of cryptococcosis. The U.S. Food and Drug Administration (FDA) previously granted Orphan, Qualified Infectious Disease Product (QIDP) and Fast Track Designations to MAT2203 for the treatment of cryptococcosis. Matinas is currently preparing to evaluate MAT2203 in a pivotal Phase 3 trial in patients with cryptococcal meningitis. The FDA has previously designated MAT2203 as a QIDP with Fast Track Status and Orphan Drug Disease designation for three additional indications: the treatment of invasive candidiasis and treatment of invasive aspergillosis and the prevention of invasive fungal infections due to immunosuppressive therapy.
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Jersey City-based BioNJ Member Scynexis announced the presentation of positive outcomes from the CANDLE 304s nested sub-study investigating oral ibrexafungerp in patients with recurrent vulvovaginal candidiasis (RVVC) who failed fluconazole treatment. The CANDLE open-label sub-study enrolled 24 patients who failed to respond to an initial three-doses of fluconazole given over seven days. Fluconazole failure was defined as persistent vaginal signs and symptoms (VSS) score equal to or greater than three after therapy. Participants received a one-day treatment of ibrexafungerp (300 mg BID). In this population, 71% of patients (17 of 24) achieved a significant reduction or elimination of signs and symptoms after treatment with ibrexafungerp.
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Hampton-Based BioNJ Member Celldex announced that the first patient has been dosed in a Phase 2 clinical study of barzolvolimab for the treatment of the two most common forms of chronic inducible urticaria (CIndU) — cold urticaria (ColdU) and symptomatic dermographism (SD). Barzolvolimab is a humanized monoclonal antibody that specifically binds the receptor tyrosine kinase KIT with high specificity and potently inhibits its activity. CIndU is characterized by the occurrence of hives or wheals that have an attributable trigger associated with them. ColdU symptoms include itching, burning wheals and angioedema when skin is exposed to temperatures below skin temperature. SD symptoms include the development of wheals and a flare reaction in response to stroking, scratching or rubbing of the skin.
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Warren-based BioNJ Member GlaxoSmithKline announced it had signed a framework contract with the European Commission’s (EC) Health Emergency Preparedness and Response Authority (HERA) for the reservation of future production and supply of 85 million doses of its pandemic influenza vaccine Adjupanrix [pandemic influenza vaccine (split virion, inactivated, adjuvanted)]. This is one of the first contracts signed by HERA since it was established in September 2021. Its core mission is to prevent, detect, and rapidly respond to health emergencies through working closely with other EC and national health agencies, industry and international partners to improve Europe’s readiness for health emergencies.
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South Plainfield-based BioNJ Member PTC Therapeutics announced that Upstaza™ (eladocagene exuparvovec) was granted marketing authorization by the European Commission. Upstaza is the first approved disease-modifying treatment for aromatic L-amino acid decarboxylase (AADC) deficiency and the first marketed gene therapy directly infused into the brain. It is approved for patients 18 months and older. During Upstaza clinical studies, patients went from not achieving any developmental motor milestones to demonstrating clinically meaningful motor skills from as early as three months following treatment, with transformational improvements shown to continue up to ten years after treatment. In addition, cognitive skills improved in all treated patients. Upstaza also reduced symptoms that cause potentially life-threatening and morbid complications.
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Morristown-based BioNJ Member Melinta Therapeutics, LLC and Cidara Therapeutics announced that they have entered into a License Agreement to facilitate a strategic partnership to commercialize rezafungin, a novel, once-weekly echinocandin antifungal in the United States (U.S.). Cidara is developing rezafungin for the treatment of candidemia and invasive candidiasis in adults, as well as for the prophylaxis of invasive fungal infections in adult patients undergoing allogeneic blood and marrow transplantation. The partnership follows Cidara’s submission of a New Drug Application (NDA) for rezafungin for the treatment of candidemia and invasive candidiasis, for which no new therapies have been approved in over a decade. The U.S. Food and Drug Administration (FDA) has previously granted Qualified Infectious Disease Product (QIDP) designation to rezafungin for injection which confers priority review of the NDA.
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Berkeley Heights-based BioNJ Member Chugai Pharmaceutical Co. announced that the primary analysis of a Japanese Phase III clinical trial (AGEHA Study) for Chugai’s anti-coagulation factor IXa/X humanized bispecific monoclonal antibody/coagulation factor VIII substitute Hemlibra® [generic name: emicizumab (genetical recombination)] in acquired hemophilia was approved in Japan for the additional indication of “routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with acquired hemophilia A”. AGEHA study is a multicenter, single-arm, Japanese Phase III clinical trial with several cohorts to investigate the safety, efficacy, pharmacokinetics and pharmacodynamics of subcutaneous administration of Hemlibra in acquired hemophilia A. The primary analysis was conducted in 12 adults with acquired hemophilia A, who were undergoing or scheduled to start immunosuppressive therapy at the time of enrollment (cohort 1).
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Berkeley Heights-based BioNJ Member Chugai Pharmaceutical Co. announced results from the primary analysis of the Phase III HAVEN 6 study, which show that Hemlibra® (emicizumab) continued to demonstrate a favorable safety profile and effective bleed control in people with moderate or mild hemophilia A, without factor VIII inhibitors. Hemlibra is approved to treat people with hemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 95 countries worldwide, including the U.S. and Japan for all severities of hemophilia A and the EU for only severe hemophilia A. It has been studied in one of the largest clinical trial programs in people with hemophilia A with and without factor VIII inhibitors, including eight Phase III studies.
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BioNJ Member Kyowa Kirin, with a site in Princeton, announced publication of data from the Phase 1B clinical study of zandelisib, an orally administered investigational phosphatidylinositol 3-kinase delta ("PI3Kδ") inhibitor, in patients with relapsed or refractory(R/R) B-cell malignancy. The published data demonstrate that an intermittent dosing regimen of zandelisib resulted in lower cumulative risk of Grade 3 or worse adverse events of special interest compared to a continuous daily dosing regimen without loss of efficacy. “The results observed in our Phase 1b study reinforce the potential of zandelisib, an investigational clinical candidate, to provide a well-tolerated clinical profile either as a single-agent or in combination with other therapies without a loss of efficacy via a unique intermittent schedule compared to continuous daily dosing,” said Richard Ghalie, M.D., Chief Medical Officer.
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BioNJ Member Legend Biotech Corporation, with a site in Piscataway, announced that it intends to offer and sell $250 million of American Depositary Shares (“ADSs”), each representing two ordinary shares, in an underwritten public offering. All ADSs to be sold in the proposed offering will be offered by Legend Biotech. Legend Biotech also intends to grant the underwriters a 30-day option to purchase up to an additional $37.5 million of ADSs sold in the public offering at the public offering price, less underwriting discounts and commissions. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed or the actual size or terms of the offering.
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Princeton-based BioNJ Member Sandoz, a Novartis division, announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Supplemental Biologics License Application (sBLA) for a high concentration formulation of 100 mg/mL (HCF) of its biosimilar Hyrimoz® (adalimumab-adaz). The application includes the indications of the reference medicine Humira® (adalimumab)* not protected by orphan exclusivity, including rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, and plaque psoriasis. The potential U.S. FDA approval of the HCF for Hyrimoz builds on Sandoz’s global biosimilar portfolio in immunology. Sandoz has more than 65 million days of patient experience with Hyrimoz 50 mg/mL worldwide and if approved, Hyrimoz 100 mg/mL citrate-free HCF would represent the first launch of a Sandoz biosimilar in the U.S. market in this specific disease space.
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Princeton-based BioNJ Member Sandoz, a Novartis division, announced that the U.S. Food and Drug Administration (FDA) has accepted its biologics license application (BLA) for a proposed first-of-a-kind biosimilar natalizumab, developed by Polpharma Biologics. The application includes all indications covered by the reference medicine Tysabri® (natalizumab) for relapsing forms of multiple sclerosis (MS) including clinically isolated syndrome (CIS), relapsing-remitting MS (RRMS), active secondary progressive disease in adults and Crohn´s Disease. The European Medicines Agency (EMA) also accepted the marketing authorization application (MAA) for this proposed biosimilar natalizumab, covering treatment as a single disease-modifying therapy (DMT) in adults with highly active RRMS, the same indication as approved by the EMA for reference medicine Tysabri®.
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Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced new data from the Phase 1b/2 CHRYSALIS-2 study cohort evaluating the safety and tolerability of the combination of RYBREVANT® (amivantamab-vmjw) with the third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) lazertinib and platinum-based chemotherapy (carboplatin and pemetrexed) in patients with relapsed/refractory non-small cell lung cancer (NSCLC) and EGFR mutations. CHRYSALIS-2 (NCT04077463) is an ongoing clinical trial evaluating RYBREVANT® in combination with lazertinib in patients with advanced NSCLC with EGFR exon 19 deletion mutations or L858R activating mutations. One cohort of CHRYSALIS-2 evaluates the combination of RYBREVANT® and lazertinib with carboplatin and pemetrexed.
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Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended conditional marketing authorization (CMA) for TECVAYLI® (teclistamab) as monotherapy for adult patients with relapsed and refractory multiple myeloma (RRMM), who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. Teclistamab is an off-the-shelf, T-cell redirecting bispecific antibody. It targets both B-cell maturation antigen (BCMA), a marker found on multiple myeloma cells, and CD3, on T-cells.
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BioNJ Member Gilead Sciences, with a site in Morris Plains, and the European Commission signed a new joint procurement agreement (JPA) that will ensure continued rapid and equitable access to Veklury® (remdesivir) for participating Member States across the European Union (EU) and European Economic Area (EEA). In the EU and EEA Veklury is the only antiviral indicated for the treatment of COVID-19 in adult and adolescent patients with pneumonia requiring supplemental oxygen (low- or high-flow oxygen or other non-invasive ventilation). The European Commission expanded the indication for Veklury to include adults who do not require supplemental oxygen and are at an increased risk of progressing to severe COVID-19.
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BioNJ Member Gilead Sciences, with a site in Morris Plains, announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) adopted a positive opinion on the fulfillment of the last specific obligation and recommended the granting of Marketing Authorization (MA) for Veklury® (remdesivir) that is no longer subject to specific obligations. Veklury was initially granted a conditional marketing authorization for the treatment of COVID-19 in adults and adolescents with pneumonia requiring supplemental oxygen (low- or high-flow oxygen or other non-invasive ventilation at the start of treatment). The conditional authorization was expanded to include adults who do not require supplemental oxygen and are at increased risk of developing severe COVID-19.
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Plainsboro-based BioNJ Members Genmab announced that BioNJ Member AbbVie will submit a conditional marketing authorization application (MAA) with the European Medicines Agency (EMA) for subcutaneous epcoritamab (DuoBody®-CD3xCD20), an investigational bispecific antibody, for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL). Genmab recently announced that the company will submit a biologics license application (BLA) for epcoritamab with the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory large B-cell lymphoma (LBCL). The MAA submission is supported by results from the large b-cell lymphoma (LBCL) cohort of the pivotal EPCORE™ NHL-1 open-label, multi-center trial evaluating the safety and preliminary efficacy of epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin lymphoma (B-NHL), including DLBCL.
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RINVOQ® (upadacitinib) Approved by European Commission as an Oral Treatment for Adults With Active Non-Radiographic Axial Spondyloarthritis
BioNJ Member AbbVie, with a site in Madison, announced that the European Commission (EC) has approved RINVOQ® (upadacitinib 15 mg, once daily), an oral therapy, for the treatment of active non-radiographic axial spondyloarthritis (nr-axSpA) in adult patients with objective signs of inflammation, as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI), who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs). Thomas Hudson, M.D., Senior Vice President of Research and Development, Chief Scientific Officer, said, "AbbVie is proud to offer RINVOQ as a first-in-class treatment option now approved in the European Union for adults living with nr-axSpA with objective signs of inflammation and inadequate response to NSAIDs. RINVOQ is the first and only JAK inhibitor approved to treat patients across the spectrum of axial spondyloarthritis, which includes nr-axSpA and ankylosing spondylitis."
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BioNJ Member AbbVie, with a site in Madison, announced that it has submitted applications for a new indication to the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for upadacitinib (RINVOQ®, 45 mg [induction dose] and 15 mg and 30 mg [maintenance dose]) for the treatment of adult patients with moderately to severely active Crohn's disease. The applications to the FDA and EMA are supported by data from three Phase 3 clinical trials, including two induction studies (U-EXCEED & U-EXCEL) and one maintenance study (U-ENDURE). Across all three studies, significantly more patients treated with upadacitinib achieved the co-primary endpoints of clinical remission and endoscopic response, with clinical remission measured by the Crohn's Disease Activity Index (CDAI) or by the patient-reported symptoms of stool frequency/abdominal pain (SF/AP).
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BioNJ Member AbbVie, with a site in Madison, announced it has submitted a marketing authorization application (MAA) for atogepant for the prophylaxis of migraine in adult patients who have at least four migraine days per month. The application is supported by the pivotal Phase 3 ADVANCE and PROGRESS studies evaluating the safety, efficacy and tolerability of atogepant in adult patients with episodic migraine and chronic migraine, respectively. Migraine is a complex neurological disease and one of the leading causes of disability worldwide. It is highly prevalent, affecting more than 1 billion people worldwide, including an estimated 11.4 percent of the population in Europe. If approved, atogepant would be the first daily oral CGRP receptor antagonist for the prophylaxis of migraine for adult patients in Europe.
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BioNJ Member AbbVie, with a site in Madison, announced a strategic transaction to further develop and commercialize iSTAR Medical's MINIject® device, a minimally invasive glaucoma surgical (MIGS) device for patients with glaucoma. This complementary alliance will support iSTAR Medical's development and commercial efforts for MINIject®, as well as provide an opportunity to expand AbbVie's eye care business, building on its glaucoma portfolio which includes drops, sustained release implants and stent offerings. MINIject® received Conformité Européenne (CE) marking approval to commercialize in European countries in the last quarter of 2021 and launched commercially in select European countries in early 2022. iSTAR Medical is currently enrolling a U.S. Premarket Approval study (STAR-V) to enable commercialization in the U.S.
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Princeton-based BioNJ Member Bristol Myers Squibb announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency has recommended approval of the fixed-dose combination of nivolumab and relatlimab for the first-line treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents 12 years of age and older with tumor cell PD-L1 expression < 1%. The European Commission, which has the authority to approve medicines for the European Union, will now review the CHMP opinion. The positive opinion is based upon efficacy and safety results from the Phase 2/3 RELATIVITY-047 trial. The trial showed that treatment with the fixed-dose combination of nivolumab and relatlimab more than doubled the median progression-free survival (PFS), including in patients with tumor cell PD-L1 expression < 1%, when compared to nivolumab monotherapy – an established standard of care.
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Princeton-based BioNJ Member Bristol Myers Squibb announced, in collaboration with Disability Solutions, a U.S.-based non-profit organization that supports companies globally to achieve true disability inclusion, the launch of the Disability Diversity in Clinical Trials (DDiCT) initiative. This new initiative aligns with Bristol Myers Squibb’s broader inclusion and diversity health equity commitments to address health disparities, clinical trial diversity, supplier diversity, employee giving and workforce representation between 2020 and 2025. The DDiCT initiative initially aims to make recommendations on how to effectively improve access, engagement, speed of enrollment, and participation of people with disabilities in clinical trials, to ensure all patient groups are reflective of the real-world population and aligned with the epidemiology of the disease studies.
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BioNJ Member Pfizer, with a site in Peapack, announced they have completed a submission to the European Medicines Agency (EMA) for an Omicron-adapted bivalent COVID-19 vaccine candidate, based on the BA.1 sub-lineage, for individuals 12 years of age and older. This application follows guidance from the EMA and International Coalition of Medicines Regulatory Authorities (ICMRA) to work towards introducing an Omicron-adapted bivalent vaccine candidate to address the continued evolution of SARS-CoV-2. They previously announced safety, tolerability and immunogenicity data from a Phase 2/3 trial that found a 30 µg booster dose of the Omicron-adapted bivalent vaccine candidate elicited a superior immune response against Omicron BA.1 as compared to the companies’ current COVID-19 vaccine. The bivalent vaccine candidate was well-tolerated with a favorable safety profile.
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BioNJ Member Pfizer, with a site in Peapack, and BioNTech SE announced that the companies have initiated a randomized, active-controlled, observer-blind, Phase 2 study to evaluate the safety, tolerability, and immune response of an enhanced COVID-19 mRNA-based vaccine candidate at a 30 µg dose level. This next-generation bivalent COVID-19 vaccine candidate, BNT162b5, consists of RNAs encoding enhanced prefusion spike proteins for the SARS-CoV-2 ancestral strain (wild-type) and an Omicron variant. The enhanced spike protein encoded from the mRNAs in BNT162b5 has been modified with the aim of increasing the magnitude and breadth of the immune response that could better protect against COVID-19. This is the first of multiple vaccine candidates with an enhanced design which the companies plan to evaluate as part of a long-term scientific COVID-19 vaccine strategy to potentially generate more robust, longer-lasting, and broader immune responses against SARS-CoV-2 infections and associated COVID-19.
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Lexicon Pharmaceuticals, with a site in Basking Ridge, announced that the U.S. Food and Drug Administration (FDA) has accepted for review and filed its New Drug Application (NDA) for sotagliflozin, an investigational dual SGLT1 and SGLT2 inhibitor, for the treatment of heart failure. The FDA assigned a standard review for the NDA filing with a Prescription Drug User Fee Act (PDUFA) target action date in May 2023. The NDA is supported by the results from the Phase 3 SOLOIST-WHF clinical study in patients with type 2 diabetes who had recently been hospitalized for worsening heart failure and the Phase 3 SCORED clinical study in patients with type 2 diabetes, chronic kidney disease and risks for cardiovascular disease.
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Lexicon Pharmaceuticals, with a site in Basking Ridge, announced the pricing of its previously announced underwritten public offering of 16,843,600 shares of its common stock, par value $0.001. The shares of Common Stock are being offered at a public offering price of $2.50 per share. All of the shares are being offered by Lexicon. The gross proceeds from the public offering are expected to be approximately $42.1 million, before deducting underwriting discounts and commissions and other offering expenses. Lexicon currently intends to use the net proceeds that it will receive from the public offering and the concurrent private placement, together with its existing cash and cash equivalents and short-term investments, for (i) funding pre-commercial and commercial launch activities for sotagliflozin in heart failure; (ii) funding continued development of sotagliflozin in heart failure and LX9211 in neuropathic pain; and (iii) working capital and other general corporate purposes.
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Calliditas Therapeutics, with a site in Hoboken, announced that the European Commission (EC) has granted conditional marketing authorization for Kinpeygo® for the treatment of primary immunoglobulin A (IgA) nephropathy (IgAN) in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/gram. Kinpeygo is an orphan medicinal product and the first and only approved treatment for IgAN, a rare, progressive autoimmune disease of the kidney with a high unmet need, with more than 50% of patients potentially progressing to end-stage renal disease (ESRD). Kinpeygo will be marketed in the European Economic Area (EEA) exclusively by STADA Arzneimittel AG. The conditional marketing authorization applies in all 27 European Union Member States as well as Iceland, Norway and Liechtenstein.
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Daiichi Sankyo, with a site in Basking Ridge, has been approved in the European Union (EU) as a monotherapy 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 specifically engineered HER2 directed antibody drug conjugate (ADC) being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca. In Europe, more than 530,000 patients are diagnosed with breast cancer each year. Approximately one in five patients with breast cancer are considered HER2 positive. Despite initial treatment with trastuzumab, pertuzumab and a taxane, patients with HER2 positive metastatic breast cancer will often experience disease progression.
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Daiichi Sankyo, with a site in Basking Ridge, announced that the first patient has been dosed in a global Phase 2 trial evaluating the efficacy and safety of DS-7300 in patients with pretreated extensive-stage small cell lung cancer (SCLC). DS-7300 is a specifically designed potential first-in-class B7-H3 directed antibody drug conjugate (ADC). Lung cancer is the second most common cancer and the leading cause of cancer-related deaths worldwide, with up to 14% being classified as SCLC in the U.S. Most patients with SCLC are diagnosed with extensive-stage disease and have a 5-year survival rate as low as 3%, highlighting the significant unmet need for more effective treatment options.
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Florham Park-based Shionogi & Co. announced that S-217622, Shionogi’s orally administered antiviral drug for COVID-19 (caused by infection with the novel coronavirus (SARS-CoV-2)), shows high in vitro antiviral activity against the Omicron subvariants (BA.4 and BA.5), with antiviral potency in preclinical testing similar to its potency against other existing variants. The SARS-CoV-2 Omicron BA.4 and BA.5 subvariants, which have been suggested to be a major factor in the recent increases in the number of infected people compared to prior Omicron strains, have been detected in Japan1. In some regions, the prevalence of BA.4 and BA.5 subvariants is increasing and the number of infected people is also increasing.
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Monmouth Junction-based Tris Pharma, Inc. announced that it has successfully developed an oxybate formulation for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy that has the potential to be significantly advantageous to existing approved therapies, such as Jazz Pharmaceuticals’ blockbuster XYWAV® and XYREM® franchise. The Tris oxybate formulation is believed to provide an advantage over approved therapies by utilizing Tris’s proprietary LiquiXR technology platform and our newly developed RaftWorks design to provide a unique oxybate formulation offering both once-nightly dosing, and very low sodium content. The Tris oxybate formulation is investigational and not yet approved by any regulatory agency.
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Monmouth Junction-based Tris Pharma, Inc. announced the publication of a Phase 3 study in the Journal of Clinical Psychiatry that demonstrated that adult patients with ADHD who took DYANAVEL XR (amphetamine) tablets CII had statistically significant improvement in symptoms over patients who took placebo. The study demonstrated that DYANAVEL XR tablets are an effective and well-tolerated treatment for the symptoms of ADHD in adults. The efficacy assessment included the administration of serial Permanent Product Measure of Performance (PERMP) tests pre-dose and at 0.5, 1, 2, 4, 8, 10, 12, 13, and 14 hours post dose. DYANAVEL XR employs Tris’s unique LiquiXR® platform technology, which allows immediate-release products to be transformed into products with both immediate-release and extended-release characteristics and permits once-daily dosing.
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Franklin Lakes-based BD (Becton, Dickinson and Company) has completed its $1.5 billion acquisition of Parata Systems from Frazier Healthcare Partners. The move provides BD with a portfolio of innovative pharmacy automation solutions that will power a growing network of pharmacies to reduce costs, enhance patient safety and improve the patient experience for retail, hospital and long-term care pharmacies. As a combined company, BD is positioned to offer a comprehensive set of technologies to the $600 million pharmacy automation market segment that is expected to grow approximately 10% annually to $1.5 billion in the U.S. alone over 10 years.
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Franklin Lakes-based BD (Becton, Dickinson and Company) is targeting monkeypox through a new collaboration with CerTest BioTec. The two will develop a molecular diagnostic test for the monkeypox virus. As part of the collaboration, the assay will use the BD MAX open system reagent suite to validate the CerTest Viasure Monkeypox CE/IVD molecular test on the BD MAX System. The assay will be available for BD MAX users and may help advance understanding of the global spread of the disease. The BD MAX System is an automated platform that performs nucleic acid extraction and real-time PCR providing results for up to 24 samples across multiple syndromes in less than three hours.
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Franklin Lakes-based BD (Becton, Dickinson and Company) acquired MedKeeper, a provider of cloud-based pharmacy management applications. BD currently offers solutions in compounding, logistics workflow, controlled substance management and inventory optimization. By adding MedKeeper‘s platform, “the companies will work to optimize the preparation of compounded medications — a critical need in the pharmacy today,” BD stated. “Preparing the right dose for patients requires a combination of factors — including personnel, training, and compliance. Pharmacy leaders require IV workflow management technology that is personalized to their needs and works within their environment,” Mike Garrison, BD’s Worldwide President of Medication Management Solutions.
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Patch Work: Princeton Start-Up Aims to Change the Nature of Birth Control
Al Altomari’s place as head of a local start-up that’s focused on women’s health care and birth control options has thrust him into the debate on national issues. And he’s willing to wade in. “If you look at, I hate to say, the political environment for women’s health and reproductive choices more broadly across the country, it has been a rough couple years,” he said. “What’s gone on in some states has been truly challenging. … And, so, it’s as important as ever to have more birth control options available for women.” Altomari’s small Princeton company has made its foray into large issues and a large market: the several-billion-dollar hormonal contraceptive sector. He’s chairman and CEO of Agile Therapeutics, which in the last year commercially launched a form of birth control called Twirla. It’s a weekly patch worn by women — and not a pill, which is still very much the dominant form of birth control in the country.
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Presenting the 2022 NJBIZ Law Power 50
As most readers recognize, the legal landscape is shifting quickly and dramatically around the country. Rights and obligations that were once taken for granted are now called into question almost daily. And that process will likely only gain momentum after the congressional midterm elections this fall. Here in New Jersey, the legal order has been relatively stable. So far. But even if that doesn’t change, it’s only a matter of time before the jurisprudential waves generated in Washington and state capitals wash over the Garden State’s economy and court system. When that happens, the professionals listed the 2022 NJBIZ Law Power 50 will, in large measure, determine how the State’s business, political and cultural climates are altered. Congratulation to these BioNJ Members for making the list: Joseph Boccassini, McCarter & English; Vito Gagliardi, Jr., Porzio, Bromberg & Newman; Elie Honig, Rutgers Institute for Secure Communities; Chris Porrino, Lowenstein Sandler; Audrey Murphy, Hackensack Meridian Health; William Palatucci, McCarter & English; and Michael Ullmann, Johnson & Johnson.
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Lawrenceville-based immunotherapy and vaccine company BioNJ Member Celsion Corp. has named a new CEO and President. Dr. Corinne Le Goff, an experienced biopharmaceutical leader, has been appointed CEO, President and a Director. The former CEO and President, Michael H. Tardugno, will remain Executive Chairman of the Board of Directors. Dr. Le Goff most recently served as Chief Commercial Officer of Moderna, where she was responsible for developing the global presence and capabilities necessary to ensure the global distribution of that company’s COVID-19 vaccine. She has also worked in executive roles with Amgen and Roche France. “I am thrilled and honored to join as President and CEO of Celsion at this pivotal point for the company,” she said.
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BioNJ Member Gilead Sciences, with a site in Morris Plains, announced that Deborah Telman will join the company as Executive Vice President, Corporate Affairs and General Counsel, and will become a member of the company’s senior leadership team. Ms. Telman will assume responsibility for Gilead’s legal and corporate affairs function, which includes government and policy, and public affairs. She will also become Corporate Secretary of Gilead. Ms. Telman will succeed Brett Pletcher, who is retiring from Gilead after 17 years with the company. She joins Gilead from Organon, where she most recently served as the company’s legal adviser and was responsible for legal affairs, ethics and compliance and global environmental health and safety.
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New Brunswick-based BioNJ Member Johnson & Johnson announced that Michael (Mike) Ullmann will retire from his position as Executive Vice President, General Counsel at the end of this year, following 33 years of leadership and service to the company. Throughout his distinguished career, Mr. Ullmann has worked with the organization’s Board of Directors and partnered with four company CEOs to shape and grow Johnson & Johnson. He will be succeeded in this role by Elizabeth (Liz) Forminard, a strong Credo-based leader who has had an exceptional career with depth and breadth across the company’s Pharmaceutical, MedTech and Consumer Health sectors.
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Woodcliff Lake-based Eagle Pharmaceuticals Inc. announced it completed its $103.9 million acquisition of Acacia Pharma Group Plc. Eagle announced it retained about 20 of Acacia’s approximately 70 employees. One of those employees is Debra Hussain, who was appointed Senior Vice President, Head of Commercial. Ms. Hussain – who joined Acacia in 2021 as Chief Commercial Officer, has nearly 25 years of pharmaceutical industry experience leading commercial launches in the hospital and critical care space. The acquisition added Acacia’s two U.S. Food & Drug Administration-approved products to its portfolio. Barhemsys is used for the treatment of postoperative nausea and vomiting, and Byfavo, for the induction and maintenance of procedural sedation in adults undergoing procedures lasting 30 minutes or less.
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East Brunswick-based Avet Pharmaceuticals has named George Svokos as its CEO and President after serving as a strategic business adviser to numerous global health care companies. Mr. Svokos’ most recent role was with Emcure. Prior to that, he served as Chief Operations Officer and Chief Commercial Officer at Albany Molecular Research Inc. Mr. Svokos also spent nearly 35 years at Teva Pharmaceutical Industries Ltd. in a number of senior leadership roles. “The Board of Directors is confident that George’s leadership, vision and track record for operational excellence will drive forward Avet’s business and our aspiration to serve as a leader in bringing generic medications to patients in the U.S. market,” said Marvin Samson, Chairman of the Board of Directors at Avet.
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Academia/Institutions/Incubators | |
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The New Jersey Department of Labor and Workforce Development (NJDOL) has awarded a total of $2.17 million in funding to seven businesses and organizations under the Growing Apprenticeship in Nontraditional Sectors (GAINS) and Pre-Apprenticeship in Career Education (PACE) grant programs. The initiatives support the creation and expansion of apprenticeships throughout the State and are part of the New Jersey Apprenticeship Network – which seeks to help all New Jerseyans cultivate careers across a wide range of employers. The program seeks to develop new and existing apprenticeship programs, create Registered Apprenticeship programs in high-growth industries and bolster workers and businesses that have been adversely affected by the COVID-19 pandemic – such as the health care industry. A particular focus of GAINS grants is supporting underrepresented populations.
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Coral Omene, M.D., Ph.D., medical oncologist in the Stacy Goldstein Breast Cancer Center at Rutgers Cancer Institute of New Jersey, together with RWJBarnabas Health, has been awarded a $50,000 grant from the V Foundation for Cancer Research in partnership with ESPN to increase clinical trial awareness and enrollment of Black women with breast cancer. The efforts in this funded proposal to increase clinical trial participation among Black breast cancer patients will include tailored patient education, advocacy and outreach, patient navigation and physician engagement and outreach. Importantly, notes Dr. Omene, this project will serve as a guide for increasing clinical trial participation among Black patients with other cancers that disproportionately affect this population, including colorectal, lung and prostate cancers.
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Rutgers University Board of Trustees announced the elected officers for 2022-2023. Nimesh Jhaveri has been elected to a one-year term as Chair of the Trustees. Mr. Jhaveri has served on Rutgers’ audit committee, the trustees executive committee and the Rutgers board of governors’ Committee on Health Affairs for the past eight years. Alan Crosta Jr., a Rutgers graduate and alumnus of the former University of Medicine and Dentistry of New Jersey, will serve a second term as Co-Vice Chair. Amy Mansue, a Charter Trustee through 2025, will serve as Co-Vice Chair of the Trustees. The Immediate Past Chair of the Trustees, Tilak Lal will serve on the Board of Governors as a Trustee Governor through June 30, 2028.
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Rutgers University’s Vincent Smeraglia, at the New Jersey Research Cores Partnering Conference, discussed how research core facilities at New Jersey universities affect the growth of private enterprise. Mr. Smeraglia said the long-existing research & development equipment and experts universities offer haven’t been at the forefront of public discourse about how innovative local companies take leaps forward. Rutgers owns a plethora of high-end research equipment. And it has people on staff that are the foremost experts on using these modern tools. These academic centers offer their specialized laboratories and all their equipment and instrumentation up for use on a fee-for-service basis to New Jersey’s companies, primarily early stage biotech firms. They can do the research & development work for the firms or invite them in to perform with some technical experts from the university there to assist.
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Rutgers Cancer Institute of New Jersey, the only program in the State designated as a Comprehensive Cancer Center by the National Cancer Institute (NCI), received the highest score in New Jersey in the Adult Specialty category for cancer, earning a High Performing rating. Rutgers Cancer Institute together with RWJBarnabas Health is at the forefront of cancer research and care and will soon be home to the Jack and Sheryl Morris Cancer Center, New Jersey’s only freestanding cancer hospital, slated to open in 2024 in New Brunswick. Only 92 cancer programs nationwide achieved this rating in the 2022-23 US News & World Report Best Hospitals survey.
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Stockton University announced that Harvey Kesselman will be retiring as President of the school at the end of the next fiscal year, June 30, 2023. Mr. Kesselman was a member of the first class at then-Stockton State College in 1971 and continued on to become a faculty member before holding several mid- and senior-level administrative positions, including Provost and Executive Vice President. He began serving as acting President in April 2015 before being named President in December 2015. Stockton has thrived under Mr. Kesselman’s leadership. He led the university through its expansion to Atlantic City and successfully championed equitable funding for public colleges and universities in New Jersey.
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Funding/Economic Development | |
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The New Jersey Innovation Evergreen Fund (NJIEF) corporate tax credit auction opens next Monday, August 1, 2022. The auction is an unprecedented opportunity for established corporate citizens in New Jersey to access a discount of up to 25% on their corporate business tax liability in the State and promote their strategic engagement and innovation objectives. Auction bids will be evaluated according to price and strategic commitment.
Join us on Thursday, August 18, for a FREE webinar led by our colleagues at the New Jersey Economic Development Authority and learn how you can take advantage of the New Jersey Innovation Evergreen Fund. You’ll leave with everything you need to know to take advantage of this game-changing opportunity to build New Jersey’s innovation economy! Click here to register.
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MDS-PAS 7th Annual Movement Disorders School for Neurology Residents
October 1, 2022
The Michael J. Fox Foundation, in partnership with the Edmond J. Safra Foundation, provides a free, two-day educational opportunity for neurology residents interested in learning about care, research and careers in Parkinson’s and other movement disorders. The course will take place October 1 and 2 in Northern New Jersey. The deadline to apply is September 5, 2022. Learn more and apply.
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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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