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

As 2020 comes to an end, we would like to wish all our Members a Happy and Healthy Holiday Season and a Prosperous New Year
2020 has been challenging to say the least. But, with the unwavering commitment and tireless work being done by the life sciences industry here in New Jersey and beyond, there is a light at the end of the tunnel. We thank the researchers, biopharmaceutical companies, government organizations and Patients who are committed to bringing effective and safe vaccines and therapies to market.
To that end, upon the historic approval of Pfizer's COVID-19 vaccine for emergency use authorization by the FDA and the subsequent approval by the Centers for Disease Control and Prevention for mass national distribution, BioNJ has launched a COVID-19 Vaccine Toolbox. Updated daily, our Members and the Community can rely on this portal for the latest information on vaccine approvals and distribution as well as useful educational tools on vaccine development and safety. 
Looking ahead to 2021, Governor Murphy announced that an agreement has been reached oncomprehensive economic recovery package that will support small businesses, drive sustainable economic growth and reform our tax incentives systemThe plan will help to attract more high-growth businesses to the Garden State while retaining and supporting the companies and talent that presently call New Jersey home. 

BioNJ President and CEO Debbie Hart is testifying in support of the measure in both the Senate and Assembly hearings today.

We'd would like to thank Governor Murphy, Speaker Coughlin and Senate President Sweeney for their work in creating a progressive incentives program that will ensure New Jersey remains the "State of Innovation" for years to come. Such a program will help to lay the foundation for a stronger, more resilient post-COVID economy in New Jersey. We look forward to continuing our work together in support of the Garden State's biopharmaceutical innovators and the Patients we serve.    

Finally, we thank our invaluable BioNJ Members for your continued support. Please stay safe and well!

Because Patients Can't Wait®,
The BioNJ Team
BioNJ Calendar

Webinar: China and U.S. Collaboration: Innovation on the Horizon (Co-hosted by BioNJ & SAPA)
Save the Date
January 14, 2021
BioNJ Virtual IT/Cybersecurity Briefing
January 28, 2021
BioNJ Board & Member Virtual Meet Up

Save the Date
February 4, 2021
Welcome New Members

Atux Iskay Group LLC
Click here for BioNJ's COVID-19 Vaccine Toolkit.

Governor Phil Murphy, joined by Health Commissioner Judith Persichilli, University Hospital President and CEO Dr. Shereef Elnahal, and Rutgers New Jersey Medical School Dean Dr. Robert Johnson, visited University Hospital's Vaccine Clinic at Rutgers New Jersey Medical School to mark the historic beginning of New Jersey's vaccination effort and witness the administration of the first COVID-19 vaccinations to the state's frontline health care workers.

BioNJ Member Pfizer Inc., with offices in Peapack, and BioNTech SE announced that the U.S. Food and Drug Administration (FDA) has authorized the emergency use of the mRNA vaccine, BNT162b2, against COVID-19 in individuals 16 years of age or older. The vaccine is now authorized under an Emergency Use Authorization (EUA) while Pfizer and BioNTech gather additional data and prepare to file a planned Biologics License Application (BLA) with the FDA for a possible full regulatory approval in 2021.

BioNJ Member Pfizer, with offices in Peapackand BioNTech SE announced that the U.S. Centers for Disease Control and Prevention's (CDC) Advisory Committee on Immunization Practices (ACIP) recommend the use of the Pfizer-BioNTech COVID-19 vaccine in people 16 years of age and older under the Emergency Use Authorization (EUA) issued by the U.S. Food and Drug Administration (FDA). This ACIP vote follows the December 1, 2020, ACIP recommendation for a Phase 1a rollout where first priority of COVID-19 vaccines is given to health care personnel treating patients, and residents in nursing homes and other long-term care facilities.

A second coronavirus vaccine is one step closer to the arms of Americans. An advisory panel voted Thursday to recommend the Food and Drug Administration authorize Moderna's vaccine for emergency use, as they did last week for Pfizer's vaccine. Members of the Vaccines and Related Biological Products Advisory Committee voted 20-0 with one abstention in favor of the vaccine. The FDA is expected to quickly authorize emergency use of the vaccine in the fight against COVID-19, which means it could start being administered as early as next week.

BioNJ in the News

"Incentive programs can play a meaningful role in ensuring New Jersey remains the 'State of Innovation.' BioNJ has long supported reenacting incentive programs to help our State keep pace with others in the region," said Debbie Hart, President and CEO of BioNJ. "Thank you to Governor Murphy, Speaker Coughlin and Senate President Sweeney for your work toward making these programs a future reality. We look forward to continuing our work together in support of the Garden State's biopharmaceutical innovators and the patients we serve."    

New Jersey's life sciences and medical technology firms continue to make significant inroads in the fight against cancer. In fact, to date in 2020, "The U.S. Food & Drug Administration reports more than 10 new novel oncology drug approvals, with half coming from companies with a footprint in New Jersey," says Debbie Hart, President and CEO of BioNJ. "The medical innovation has led to lower death rates from cancer in many cases, making cancer a chronic condition to be managed, instead of a death sentence." Hart says that the state's vibrant life sciences ecosystem has transformed New Jersey into a hub for cell and gene therapy, especially when it comes to oncology. 

Hart worked with the State's biotechnology industry leaders to establish BioNJ in 1994 and has been dedicated to building the State's life sciences ecosystem ever since. Those efforts have been even more critical during the COVID-19 pandemic, with New Jersey situated squarely in the middle of both the outbreak and the fight to end it. With New Jersey being the top biomanufacturing state in the country, BioNJ is taking an active role in assessing and promoting this growing sector, especially in the area of cell and gene therapy where New Jersey is leading the way from discovery through manufacturing.

NJ Company News

Princeton-based BioNJ Member Agile Therapeutics, Inc. announced the U.S. commercial launch of Twirla® (levonorgestrel and ethinyl estradiol) transdermal system, a new non-daily, non-invasive contraceptive patch. Twirla is now available in the United States by prescription for women of reproductive potential with a body mass index (BMI) <30 kg/m2 for whom a combined hormonal contraceptive is appropriate to prevent pregnancy. Twirla is less effective in women with a BMI ≥ 25 kg/m2 to < 30 kg/m2 and should not be used in women with a BMI ≥ 30 kg/m2.

Pennington-based OncoSec Immunotherapies announced that the European Patent Office has issued notices of intention to grant for two patent applications. The applications are directed to the use of OncoSec's interleukin-12 (IL-12)-based immunotherapy platform, including its lead product candidate TAVO™ (tavokinogene telseplasmid) delivered with the company's proprietary intratumoral gene electrotransfer system for the treatment of cancer. "Pursuing these patents is important to strengthen the intellectual property protections around TAVO and our electroporation gene delivery system," said Daniel O'Connor, Chief Executive Officer of OncoSec.

Cranbury-based BioNJ Member Rafael Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company's lead compound, CPI-613® (devimistat), for the treatment of acute myeloid leukemia (AML). "Receiving Fast Track designation, especially during a pandemic that has created significant challenges for many trials across the globe, is a testament to the dedicated work of the Rafael team," said Sanjeev Luther, President and CEO of Rafael. "We would not be here without the support of the FDA, our doctors, our patients, and all who are invested in the hope of finding a successful treatment for this hard-to-treat cancer."

Bedminster-based BioNJ Member Matinas BioPharma Holdings, Inc. announced that they plan to collaborate with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), to test oral formulations of remdesivir in preclinical models. Remdesivir is owned by Gilead Sciences, Inc. (Nasdaq: GILD) and the lipid nanocrystal (LNC) platform is owned by Matinas. Any product generated as a part of efforts by Matinas and NIAID would require a license from Gilead for the use of remdesivir and a license from Matinas for the use of the LNC formulation.

BioNJ Member Pfizer, with offices in Peapackand BioNTech SE announced additional data on neutralizing antibody and T cell responses from the Phase 1/2 trial with BNT162b2 conducted in Germany. The study results demonstrate that BNT162b2 elicits a combined adaptive humoral and cellular immune response against SARS-CoV-2 and provide insights into the composite nature of BNT162b2-induced T cell immunity. The results were published on the preprint server MedRxiv and are available here. BNT162b2 is an investigational COVID-19 vaccine developed by Pfizer-BioNTech. It has been authorized for emergency use for individuals 16 years of age and older in several countries around the world.

Princeton-based BioNJ Member Soligenix, Inc. announced that it has demonstrated extended protection with its heat stable ricin toxin vaccine, RiVax®. Mice, vaccinated twice on Days 1 and 21 were protected for at least 365 days against subsequent ricin challenge. These results demonstrate that the thermostabilized vaccine formulation is capable of eliciting enduring protection in mice. Coupled with previous demonstration of efficacy in mice and non-human primates (NHPs) as well as long-term thermostability (at least 1 year at 40°C or 104°F), these results reinforce the practicality of stockpiling and potentially utilizing the RiVax® vaccine in warfighters and civilian first responders without the complexities that arise for vaccines that require cold chain handling. 

Princeton-based BioNJ Member Soligenix, Inc. announced that it has entered into a $20 million convertible debt financing agreement with Pontifax Medison Debt Financing (Pontifax), the health care-dedicated venture and debt fund of the Pontifax life sciences funds. "The access to less dilutive capital provided by this facility is designed to increase our financial flexibility as we continue to advance our rare disease pipeline, and build toward commercialization in the United States with SGX301 for the treatment of cutaneous T-cell lymphoma (CTCL) and SGX942 for the treatment of oral mucositis in head and neck cancer patients," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix.

Somerset-based BioNJ Member Legend Biotech Corporation announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application to evaluate LB1901, the company's investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of adults with relapsed or refractory T-cell lymphoma (TCL). Under this IND, Legend will initiate a Phase 1 clinical study for LB1901 in the United States. LB1901 is an investigational CAR-T product targeting CD4, which is a surface membrane glycoprotein uniformly expressed in a majority of TCL subtypes. 

BioNJ Member Lantern Pharma, with offices in Kearny, announced new scientific data that substantiates blood brain barrier permeability (BBB) for its drug candidate LP-184. LP-184 is being targeted for treating Glioblastoma Multiforme (GBM), an aggressive malignant form of brain cancer that comprises about 52% of all primary malignant brain tumors according to the American Association of Neurological Surgeons. GBM has a median survival rate of only 15 months and ranks among the most aggressive of human cancers. It is considered an orphan disease for which there is no cure. The ability of a drug candidate to cross the blood brain barrier is of critical importance in treatment outcomes for CNS and brain cancers. 

Jersey City-based BioNJ Member Mitsubishi Tanabe Pharma America, Inc. announced the initiation of a global clinical study exploring daily dosing of an investigational oral formulation of edaravone (MT-1186) in patients with amyotrophic lateral sclerosis (ALS). The study, sponsored by Mitsubishi Tanabe Pharma Development America, Inc. is the postmarketing commitment following the U.S. Food and Drug Administration (FDA) approval of RADICAVA® (edaravone) IV. The global, multicenter, randomized, double-blind study, MT-1186-A02, will compare two dosing regimens for oral edaravone in approximately 380 people with ALS.

Cranbury-based BioNJ Member Palatin Technologies, Inc. announced positive results in its Phase 2 study of PL9643 for the treatment of dry eye disease (DED). Statistically significant improvement in multiple signs and symptoms was achieved in the moderate to severe patient population after 2 weeks of dosing and at the 12-week visit. There were no safety signals identified and PL9643 had excellent ocular tolerability. However, statistical significance for the primary endpoints was not reached in the overall enrolled population that included mild, moderate, and severe patients, as measured at the 12-week primary evaluation visit.

Bridgewater-based BioNJ Member Eli Lilly and Company and Incyte announced that the New England Journal of Medicine has published peer-reviewed results from the Adaptive COVID-19 Treatment Trial (ACTT-2) sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). The Phase 3 study included 1,033 patients from 67 trial sites in eight countries. These results support the emergency use authorization (EUA) issued by the U.S. Food and Drug Administration (FDA) for baricitinib in combination with remdesivir in hospitalized patients with COVID-19 requiring supplemental oxygen.

Bridgewater-based BioNJ Member Eli Lilly and Company and Prevail Therapeutics Inc. announced a definitive agreement for Lilly to acquire Prevail for $22.50 per share in cash (or an aggregate of approximately $880 million) payable at closing plus one non-tradable contingent value right ("CVR") worth up to $4.00 per share in cash (or an aggregate of approximately $160 million), for a total consideration of up to $26.50 per share in cash (or an aggregate of approximately $1.040 billion). The CVR is payable (subject to certain terms and conditions) upon the first regulatory approval of a product from Prevail's pipeline as set forth in more detail below. 

Bridgewater-based BioNJ Member Eli Lilly and Company announced that qualified physicians may now enroll patients in the study New IDEAS: Imaging Dementia-Evidence for Amyloid Scanning at New IDEAS-Study.org. New IDEAS builds off the historic IDEAS Study, the largest Alzheimer's disease study ever conducted with over 18,000 participants. The new study will enroll 7,000 participants and investigate the impact of amyloid positron emission tomography (PET) scans on patient management and associated health outcomes in a more diverse population, with a goal of over 50% of study participants identifying as Black/African American or Hispanic/Latino.

Bridgewater-based BioNJ Member Eli Lilly and Company announced Tirzepatide led to superior A1C and body weight reductions from baseline in adults with type 2 diabetes after 40 weeks of treatment in topline results from the Company's SURPASS-1 monotherapy clinical trial evaluating the efficacy and safety of tirzepatide compared to placebo. Using the efficacy estimandi, the highest dose of tirzepatide led to an A1C reduction of 2.07 percent and reduced body weight by 9.5 kg (11.0 percent). More than half (51.7 percent) of participants in this arm achieved an A1C less than 5.7 percent -- the level seen in people without diabetes.

BioNJ Member Evotec SE announced that the company has achieved key milestones in its partnership with BioNJ Member Bristol Myers Squibb in the field of targeted protein degradation with the first two targets transitioning into drug discovery after completing a comprehensive target validation process. Evotec and Bristol Myers Squibb (the successor in interest to Celgene) initiated their long-term strategic drug discovery and development partnership in the field of targeted protein degradation in 2018 with the goal to identify novel drug targets. The partnership leverages Evotec's proprietary PanOmics platform, which combines enhanced throughput proteomics, high throughput transcriptomics and cell imaging with an integrated data analysis platform PanHunter. 

Princeton-based BioNJ Member Bristol Myers Squibb announced new results from the QUAZAR® AML-001 study evaluating Onureg®(azacitidine tablets; CC-486), an oral hypomethylating agent, as a treatment for adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy. Results demonstrated treatment with Onureg improved overall survival (OS), the primary endpoint of the study, as well as showed clinical benefit across other key secondary endpoints, compared to placebo, in patients with AML in first remission.

Plainsboro-based BioNJ Member Novo Nordisk announced the decision to enter Phase 3 development in Alzheimer's disease with 14 mg oral semaglutide, a once-daily oral formulation of the long-acting GLP-1 analogue semaglutide. The decision follows evaluation of GLP-1 data from preclinical models, real-world evidence studies, post-hoc analysis of data from large cardiovascular outcomes trials, as well as discussions with regulatory authorities. Novo Nordisk intends to initiate a pivotal phase 3a program with approximately 3,700 people with early Alzheimer's disease. The program is planned for initiation in the first half of 2021 and will investigate the efficacy and safety of once-daily oral semaglutide, compared to placebo.

Plainsboro-based BioNJ Member Novo Nordisk announced that the U.S. Food and Drug Administration (FDA) approved an updated label for Saxenda® (liraglutide) injection 3 mg for use in the treatment of obesity in adolescents (12-17 years) with a body weight above 60 kg and an initial body mass index (BMI) corresponding to 30 kg/m2 or greater for adults, as an adjunct to a reduced-calorie diet and increased physical activity. Saxenda® was approved by the FDA in 2014 for chronic weight management in adults with a BMI ≥30 kg/m2, or ≥27 kg/m2 with at least one weight-related comorbidity, as an adjunct to a reduced calorie diet and increased physical activity.

Plainsboro-based BioNJ Member Novo Nordisk announced the submission of a new drug application (NDA) to the US Food and Drug Administration (FDA) for subcutaneous semaglutide 2.4 mg, a once-weekly glucagon-like peptide-1 (GLP-1) analogue for chronic weight management. A priority review voucher has been applied to the NDA, leading to an anticipated review time of six months from the submission date, according to standard FDA review timelines. The submission is based on the results from the STEP phase 3a clinical trial programme, which included more than 4,500 adults with obesity or overweight.

Plainsboro-based BioNJ Member Novo Nordisk A/S has completed its $1.35 billion acquisition of Roseland-based drug delivery company Emisphere Technologies, Emisphere. Novo Nordisk also acquired the related royalty stream obligations owed to affiliates of MHR Fund Management LLC for $450 million, bringing its total price to $1.8 billion. "We are very pleased to reach this milestone and know Novo Nordisk will guide Emisphere into a promising new era," Chairman Timothy Rothwell said. "The Emisphere board of directors and its Special Committee are confident that this transaction has delivered substantial value to our stockholders."

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced initial data for the Phase 1 first-in-human dose escalation study of talquetamab (JNJ-64407564) for the treatment of relapsed or refractory multiple myeloma (NCT03399799). Talquetamab is a first-in-classand the only investigational bispecific antibody that targets both GPRC5D, a novel multiple myeloma target, and CD3 on T-cells. Initial results for both the subcutaneous (SC) and intravenous (IV) formulations show encouraging clinical activity against the GPRC5D target, which is highly expressed on multiple myeloma cells and associated with poor prognostic factors.

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced updated results from an ongoing Phase 1 first-in-human dose escalation study (NCT03145181) of teclistamab (JNJ-64007957) for the treatment of relapsed or refractory multiple myeloma. In heavily pretreated patients, the overall response rate (ORR) with teclistamab was 73 percent (16/22) at the recommended subcutaneous (SC) Phase 2 dose (RP2D). Those results for the SC formulation support the recommended dose for the pivotal Phase 2 registration trial, which has started. In addition, updated results for the intravenous (IV) formulation demonstrate the durability of responses. 

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced new data from the Phase 2 CAPTIVATE study evaluated the efficacy and safety of IMBRUVICA® (ibrutinib) plus venetoclax in the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) and showed that, after achieving undetectable minimal residual disease (uMRD) in both the blood and bone marrow with the IMBRUVICA® combination regimen, the one-year disease-free survival (DFS) of patients randomized to discontinue active treatment was comparable to that of patients randomized to continue IMBRUVICA® monotherapy (95.3 percent vs. 100 percent, respectively [p=0.1475]).

Kenilworth-based BioNJ Member Merck & Co. announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for KEYTRUDA, Merck's anti-PD-1 therapy, in combination with platinum and fluoropyrimidine based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus and gastroesophageal junction (GEJ). This sBLA is based on data from the pivotal Phase 3 KEYNOTE-590 trial, in which KEYTRUDA plus chemotherapy demonstrated significant improvements in the primary endpoints versus chemotherapy in these patients regardless of PD-L1 expression status and tumor histology. 

Kenilworth-based BioNJ Member Merck & Co. announced that the pivotal Phase 3 KEYNOTE-775/Study 309 trial evaluating the investigational use of KEYTRUDA, Merck's anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, met its dual primary endpoints of overall survival (OS) and progression-free survival (PFS) and its secondary efficacy endpoint of objective response rate (ORR) in patients with advanced endometrial cancer following at least one prior platinum-based regimen. 

Kenilworth-based BioNJ Member Merck & Co. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending approval of KEYTRUDA, Merck's anti-PD-1 therapy, as monotherapy for the first-line treatment of adult patients with metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer. This recommendation is based on results from the pivotal Phase 3 KEYNOTE-177 trial, in which KEYTRUDA, as a monotherapy, demonstrated a significant improvement in progression-free survival compared to chemotherapy (investigator's choice: mFOLFOX6 with or without bevacizumab or cetuximab; or FOLFIRI with or without bevacizumab or cetuximab), a current standard of care.

East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) granted iptacopan (LNP023) Breakthrough Therapy Designation (BTD) in paroxysmal nocturnal hemoglobinuria (PNH) and Rare Pediatric Disease (RPD) Designation in C3 glomerulopathy (C3G). The breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. 

East Hanover-based BioNJ Member Novartis announced that the European Commission (EC) has approved Leqvio® (inclisiran) for the treatment of adults with hypercholesterolemia or mixed dyslipidemia. This approval is based on the results of the robust ORION clinical development program, where Leqvio provided an effective and sustained low-density lipoprotein cholesterol (LDL-C) reduction of up to 52% in patients with elevated LDL-C, despite maximally tolerated statin therapy. With two doses a year, after an initial dose and one at 3 months, Leqvio is expected to support long-term adherence.

East Hanover-based BioNJ Member Novartis announced positive findings from the first interpretable results of the Phase III KESTREL study, assessing the efficacy and safety of Beovu® (brolucizumab) 3 mg and 6 mg in diabetic macular edema (DME). Following KITE, KESTREL is the second pivotal Phase III study of Beovu in DME. The trial met its primary endpoint of non-inferiority in change in best corrected visual acuity (BCVA) from baseline of Beovu 6 mg to aflibercept 2 mg at year one. The trial also met its key secondary endpoint of non-inferiority in average change in BCVA of Beovu 6 mg to aflibercept 2 mg over week 40 through week 521. (Beovu 6 mg is the marketed dose for wet AMD.)

East Hanover-based BioNJ Member Novartis announced detailed results from the pivotal Phase III REACH3 study demonstrate Jakavi® (ruxolitinib) significantly improved outcomes across a range of efficacy measures in patients with steroid-refractory/dependent chronic graft-versus-host disease (GvHD) compared to best available therapy (BAT)In REACH3, patients treated with Jakavi achieved significantly greater overall response rate (ORR) compared to BAT (49.7% vs. 25.6%; p<0.0001i) at Week 24, the primary endpoint of the study. Jakavi also demonstrated statistically significant and clinically meaningful improvements in key secondary endpoints:

East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 12 to 1 that the data presented support the use of Entresto® (sacubitril/valsartan) in treatment of patients with heart failure with preserved ejection fraction (HFpEF). This was based on data supporting the benefit of Entresto in reducing worsening heart failure (total heart failure [HF] hospitalizations and urgent HF visits) in patients studied in PARAGON-HF. If approved by the FDA, Entresto could become the first therapy indicated for use in treatment of patients with HFpEF, as well as the first medication approved for both major types of chronic heart failure, HFpEF and heart failure with reduced ejection fraction (HFrEF), both based on trials that included active comparators (valsartan and enalapril, respectively).

East Hanover-based BioNJ Member Novartis announced new Kisqali® (ribociclib) data demonstrating consistent efficacy benefit with Kisqali plus endocrine therapy across the main intrinsic subtypes of hormone receptor positive, human epidermal growth factor receptor-2 negative (HR+/HER2-) metastatic breast cancer. The largest biomarker analysis of efficacy in intrinsic subtypes evaluated whether there was a correlation between these subtypes and efficacy outcomes in patients treated with Kisqali across the three Phase III MONALEESA trials.

East Hanover-based BioNJ Member Novartis announced analyses from two separate trials with Kymriah® (tisagenlecleucel) in patients with certain advanced lymphomas. In the interim analysis of the investigational Phase II ELARA study, Kymriah led to a complete response (CR) in 65% of patients with relapsed or refractory (r/r) follicular lymphoma (FL) and an overall response rate (ORR) of 83% after at least three months of follow-up. These patients continued to relapse or have refractory disease despite exposure to numerous lines of therapy (median four prior lines of therapy [range 2-13]) prior to Kymriah infusion

Morris Plains-based BioNJ Member Gilead Sciences, Inc. presented new data from the Phase 3 ASCENT trial of Trodelvy® (sacituzumab govitecan-hziy) in metastatic triple-negative breast cancer (mTNBC) at the 2020 San Antonio Breast Cancer Symposium. The new data and analyses from the ASCENT trial continue to demonstrate the high clinical activity of Trodelvy in this patient population with traditionally poor outcomes. Trodelvy is a Trop-2-directed antibody and topoisomerase inhibitor conjugate that is indicated in the U.S. for the treatment of adult patients with mTNBC who have received at least two prior therapies for metastatic disease. 

Morris Plains-based BioNJ Member Gilead Sciences, Inc., announced updated results from the magrolimab Phase 1b trial. Magrolimab is an investigational, potential first-in-class, anti-CD47 monoclonal antibody being studied in previously untreated acute myeloid leukemia (AML) patients who are ineligible for intensive chemotherapy, including patients with TP53-mutant AML. The study continues to demonstrate high response rates with magrolimab in combination with azacitidine, with an overall response rate of 63% (n=27/43) among the total patient population and 69% (n=20/29) in TP53-mutant patients. 

Cranford-based Citius Pharmaceuticals, Inc. announced interim data from a proof-of-concept ("POC") large animal study of its proprietary induced mesenchymal stem cell ("i-MSC") therapy for acute inflammatory respiratory conditions including COVID-19 related Acute Respiratory Distress Syndrome ("ARDS"). The available results of i-MSC therapy in the study show improvement in critical parameters, such as improved oxygenation, less systemic shock, and reduced lung injury, compared to the control group. The study was conducted in a widely accepted large animal model.

Lexicon Pharmaceuticals, Inc., with offices in Basking Ridge, announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for the development of LX9211 in diabetic peripheral neuropathic pain. "The FDA's Fast Track designation of LX9211 reflects the serious unmet medical need of people suffering from diabetic peripheral neuropathic pain," said Praveen Tyle, Ph.D., Executive Vice President of Research and Development. "We look forward to working closely with the FDA throughout the clinical development process to bring this potential new innovative treatment to patients as quickly as possible."

Basking Ridge-based Daiichi Sankyo Company, Limited and AstraZeneca's trastuzumab deruxtecan has been recommended for conditional marketing authorization in the European Union (EU) as monotherapy for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who have received two or more prior anti-HER2 based regimens. Following review of the application under its accelerated assessment procedure, the Committee for Medicinal Products for Human Use (CHMP) based its positive opinion on results from the pivotal phase 2 DESTINY-Breast01 trial.

Basking Ridge-based Daiichi Sankyo Company, Limited and AstraZeneca announced the initiation of TROPION-Lung01, a global pivotal Phase 3 head-to-head study of datopotamab deruxtecan (Dato-DXd; DS-1062), a TROP2 directed antibody drug conjugate (ADC), versus docetaxel in patients with advanced or metastatic non-small cell lung cancer (NSCLC) without actionable genomic alterations who have previously received platinum-based chemotherapy and immunotherapy. Patients with advanced or metastatic NSCLC without actionable genomic alterations (such as EGFR, ALK, ROS1, NTRK, BRAF, RET, or MET exon 14 skipping) have demonstrated an unmet need after currently approved front-line and second-line therapies are exhausted. 

Basking Ridge-based Daiichi Sankyo Company, Limited announced that a supplemental application for the antiplatelet agent, prasugrel hydrochloride (hereafter, prasugrel), was filed in Japan to expand the indication for prevention of recurrence of ischemic stroke as a partial change in items of the pharmaceutical manufacturing and marketing approval for prasugrel. This application is based on the results of a Phase 3 study in thrombotic stroke patients in Japan (PRASTRO-III study), as well as other Phase 3 studies in Japan targeting patients with ischemic cerebrovascular disease (PRASTRO-I and PRASTRO-II studies).

Red Bank-based Provention Bio, Inc. announced the initiation of PROVENT (PROtocol for coxsackievirus VaccinE in healthy voluNTeers), a first-in-human study of its polyvalent inactivated coxsackievirus B (CVB) vaccine candidate, PRV-101. Provention is developing PRV-101 for the prevention of acute CVB infection and the potential delay or prevention of type 1 diabetes (T1D) and celiac disease. PROVENT is a placebo-controlled, double-blind, randomized first-in-human study being conducted at the Clinical Research Services Turku - CRST Oy, a clinical trial unit in Turku, Finland

Passage Bio, Inc. announced that it has entered into a long-term lease to support Chemistry, Manufacturing and Controls (CMC) laboratory operations for the company's gene therapy programs. The new laboratory, slated to open in the second quarter of 2021 at the Princeton West Innovation Campus in Hopewell, New Jersey, will initially focus on state-of-the-art analytical capabilities, clinical assay development and validation, biomarker assay validation and clinical product testing to support both viral vector manufacturing and clinical development.

Princeton-based Fosun Pharmaceutical and BioNTech SE announced an agreement to supply Mainland China with 100 million doses initially of their BNT162 mRNA-based vaccine candidate against COVID-19 in 2021, subject to regulatory approval. Initial supply will be delivered from BioNTech's production facilities in Germany.  We would like to thank the Chinese government and National Medical Products Administration for their commitment and trust in our vaccine development efforts to help address this global pandemic threat," said Ugur Sahin, M.D., CEO and Co-founder of BioNTech. 

Princeton-based Integra LifeSciences Holdings Corp. has agreed to acquire ACell Inc., a regenerative medicine company, for $300 million in cash upfront, plus milestone payments that could reach another $100 million. The definitive agreement would give Integra a product portfolio that includes a proprietary urinary bladder matrix platform technology, known as MatriStem UBM. Integra is expanding its Orthopedics and Tissue Technologies segment, while selling off its orthopedics business and converting the unit to focus on tissue technologies. 

People in the News

Princeton-based BioNJ Member Oyster Point Pharma, Inc. announced the appointment of Marian Macsai, M.D., as Chief Medical Officer, and Eric Carlson, Ph.D., as Chief Scientific Officer. "Based on their industry expertise and unique experience in ocular surface diseases, Marian and Eric will add tremendous value to the organization and strategically grow their respective teams as we head into a pivotal year of expansion across our pipeline and with the potential for approval and launch of our lead investigational therapy, OC-01 (varenicline) nasal spray, for dry eye disease in 2021," said Jeffrey Nau, Ph.D., MMS, President and Chief Executive Officer at Oyster Point Pharma. 

Morris Plains-based BioNJ Member Gilead Sciences, Inc. announced that  Jeffrey A. Bluestone, Ph.D., has been appointed to the company's Board of Directors.  Dr. Bluestone is the President and Chief Executive Officer of Sonoma Biotherapeutics and the A.W. and Mary Margaret Clausen Distinguished Professor of Metabolism and Endocrinology at the University of California San FranciscoHis research has led to the development of multiple immunotherapies, including the first medicine approved by the U.S. Food and Drug Administration (FDA) targeting T-cell co-stimulation to treat autoimmunity and the first FDA-approved checkpoint inhibitor for the treatment of metastatic melanoma. 

New Providence-based Vertice Pharma announced that Sandra Miceli will join the Company as Chief Financial Officer, reporting to Scott Meyers, Chief Executive Officer. Ms. Miceli brings over 25 years of experience in successful pharmaceutical growth companies. Most recently, Ms. Miceli was a senior member of Amneal Pharmaceutical's finance team, where she served in senior leadership positions. She played a key role driving strategic initiatives including critical oversight of all treasury, debt, risk management and integration activities relating to the transition of the company from private to public. 

Bedminster-based Tessa Therapeutics announced the appointment of David McIntyre as its Chief Financial Officer. Mr. McIntyre brings over 25 years of global financial, operational and legal experience, including over 10 years as Chief Financial Officer of various publicly listed and privately held entities. He joins Tessa from AVITA Medical, Inc. where he served as the Chief Financial Officer. Before joining AVITA Medical, Mr. McIntyre served as a Partner with Apple Tree Partners, a multi-billion-dollar life sciences venture capital and growth equity fund. At ATP, Mr. McIntyre was responsible for ATP's medical device portfolio, together with various operating and Board functions, including acting as CFO and Head of Technical Operations at Braeburn, Inc.

Parsippany-based Zoetis CEO Kristin Peck said she had to double-check when she heard the news. "It was a surprise," she said. "It's not one of those things that you know about ahead of time. Someone actually emailed me that morning and I thought it was a joke." It's a big honor. Earlier this week, Peck was named one of Fortune magazine's 20 Top Businesspersons of the Year for 2020, landing at No. 14 on the list. Fortune said the rankings identify star executives and are based on 10 metrics, ranging from return on capital to total return to investors.

Like the others -- and like most of business and life in New Jersey -- the Manufacturing Power 50 is shaped by the COVID-19 pandemic. The top ten individuals are drawn mostly from the pharmaceutical industry, which will provide the treatments and tools that will eventually end the outbreak. No surprise there. Congratulations to the following BioNJ Members, including BioNJ President & CEO Debbie Hart, who made this year's list:
  • Johnson & Johnson - Alex Gorsky
  • Merck & Co. - Ken Frazier
  • Novartis - Thomas Kendris
  • BD - Tom Polen
  • NJBIA - Michele Siekerka 
  • Choose NJ - Frank Danieli
  • Rutgers Business School - Dr. Kevin Lyons
  • Rutgers Food Innovation Center - Nolan Lewin
  • Rutgers Food Innovation Center - Donna Schaffner
  • SCYNEXIS - Dr. Marco Taglietti
  • Celsion Corp. - Micharl Tardugno

Institution and Education News

S2S recently announced our new mission statement, vision and strategic goals, as well as a number of significant board and staff appointments -- all geared to position S2S for greater levels of success. These follow a comprehensive analysis of our organization and programs. The COVID-19 pandemic and its spotlight on educational inequities in our society have helped clarify the path forward for S2S and the steps we must take to achieve educational equity in the U.S. Our new mission statement is to inspire, motivate and educate students in underserved communities to pursue careers in science, technology, engineering and math (STEM) and foster a more diverse, equitable and representative workforce. 

Rutgers Cancer Institute of New Jersey said it received an anonymous philanthropic gift of $25 million. The donation will provide groundbreaking support for the Cancer Immunology and Metabolism Center of Excellence. This generous amount will support faculty recruitment, shared resource development and cancer research to help scientists better understand the human immune response to cancer and ultimately develop the foundation for new treatments or make existing therapies more effective.

County College of Morris President Tony Iacono is more than happy to talk about how the new Advanced Manufacturing and Engineering Center will help the school go from an 11,000-square-foot facility to one that is more than 31,000 square feet. And he's more than happy to talk about how capacity issues no longer will force the school to turn away students who are eager to train for careers in a sector that is desperate for a next generation of workers.

Saint Barnabas Medical CenterBayshore Medical Center and the Warren Campus of St. Luke's Hospital were among the 105 Top Hospitals named by the Leapfrog GroupThe Leapfrog Group, which bills itself as an independent national watchdog organization of employers and other purchasers focused on health care safety and quality, is best known for the semiannual grades it gives each hospital in the county. Leapfrog CEO Leah Binder said this year's awards are particularly impressive, considering the impact of the COVID-19 pandemic. "2020 put hospitals nationwide to the test in the most unforeseen and unprecedented ways, so we are particularly proud to recognize excellence in the hospital industry this year," she said. "All hospitals deserve our gratitude and these facilities, in particular, shine."

Funding News

The more than two-year battle on tax incentives for the State of New Jersey appears to be over as Gov. Phil Murphy, State Senate President Steve Sweeney and Assembly Speaker Craig Coughlin appeared to have agreed on a six-year, $11.5 billion program that will include annual caps on awards and is intended to do more than just reward companies for job creation. Tim Sullivan, the CEO of the State Economic Development Authority, tweeted many of details, saying the program represents a different approach to economic development -- one that invests in job creation, but also in innovation, Main Street and helping to solve longstanding economic inequality issues.

New Jersey Economic Development Authority CEO Tim Sullivan loves to talk about how the proposed tax incentive programs could have a major impact -- particularly in the State's most challenging areas. The New Jersey Innovation Evergreen Fund, a program in which the State becomes an equity investor in start-ups, has been a favorite of Gov. Phil Murphy's since it was first proposed -- more than two years ago. The idea is that the State's willingness to contribute significant dollars into start-ups that venture capital firms have identified could make more of those companies start here, grow here and stay here, the theory goes. And, since the fund will look to reinvest many of the potential gains that could be realized, there's a chance the fund could be self-sustaining.

The Institute for Entrepreneurial Leadership (IFEL) launched the Small Businesses Need Us initiative earlier this year in an effort to assist small businesses who are battling to stay alive due to COVID-19The program matches business professionals and experts with small business owners who are struggling to keep their businesses alive. The goal is to provide the life support that the businesses need to survive, and ultimately, to thrive despite the negative impact of the COVID-19 pandemic. 

Thought Leadership from BioNJ Members

By Lowenstein Sandler LLP

Early stage and small to midsize private and public companies in the life sciences sector began 2020 on a high note. Funding, particularly in the biotechnology sector, was pouring into the industry. Even several months into the pandemic, many biotech and other life sciences companies showed no signs of struggling to raise capital. But as COVID-19 continues to batter the world economy, life sciences leaders are likely to encounter new obstacles to funding growth.

Special Programs from Our Partners for BioNJ Members

January 11-15, 2021

With a blend of on-demand and live sessions, the virtual 2021 program is designed to provide you with the latest industry updates and flexibility to view content according to your schedule. BioNJ Members: Contact Randi Bromberg at RBromberg@BioNJ.org for a special discount code.

January 11-15, 2021

Affordable access to BIO One-on-One Partnering, with an integrated video conferencing system, to provide a seamless partnering experience for all attendees, regardless of whether you are registered for a specific event during JPM week. BIO Members partner for free/ Non-Member companies partner for $150.

February 16-19, 2021

This online virtual training course will provide a detailed understanding in upstream and downstream processing of viral vectors used in cell and gene therapy. The course will focus on viral vector theory, technical demonstrations of bioreactors, demonstration of chromatography, principles of tangential flow filtration (TFF) and scale-up topics. The course is intended for scientists and engineers from the biopharma industry interested in learning about upstream and downstream processing of viral vectors. Click here for more information.

BioNJ Member Services Provider Directory

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

The NJBAC is a state agency offering free and confidential assistance to help your business grow. As a "one-stop shop," NJBAC can help businesses of all sizes and types manage the state's regulatory processes, boost exports, gain access to financial resources where applicable and tackle other obstacles along the way. You can download the NJBAC guide by clicking here or contact their business advocates at 1-800 JERSEY 7.

Please contact BioNJ at BioNJ@BioNJ.org 
or call 609-890-3185 with any questions.