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

Now that we are one year in and vaccines are a reality, there is so much to think about and plan for! Join us next Thursday, February 25, for BioNJ's Virtual Briefing where we'll learn the latest on the vaccine and its variants and hear from those directly in the trenches developing plans for returning to the office and engaging with their Teams through the pandemic. Click here for details.

Then, mark your calendars and register your Team for BioNJ's Inaugural Diversity, Equity & Inclusion Conference, taking place virtually on March 24 and 25, 2021. The Mission of the conference is to "Engage, Empower and Inspire All to Enable and Impact Diversity, Equity and Inclusion" -- Because it Matters. In this important endeavor, we will explore solutions, best practices and new ideas for addressing this very real problem. We hope to inspire the community by sharing case studies, vignettes, workshops and tools that we can all use every day to impact change in the critical domains of Culture, Self, Talent and Business. Click here to learn more. 

Not yet a Member? Not a problem! Contact Kim Minton at KMinton@BioNJ.org to discuss how your organization can benefit from BioNJ Membership. 

Because Patients Can't Wait®,
The BioNJ Team

BioNJ Calendar

BioNJ Briefing: Vaccines are Here... Now What? 
Planning for the Next 6 Months
February 25, 2021
March 4, 2021
BioNJ's Diversity, Equity and Inclusion Conference...Because It Matters
March 24-25, 2021
BioNJ BioPartnering Virtual Conference 
May 18 & 19, 2021
Welcome New BioNJ Members

Putting Patients First: The Value of Medical Innovation

As breakthrough drugs stream out of biopharmaceutical laboratories, how much they should cost and who will get access to them remain thorny issues. During his campaign for president, Joe Biden indicated that he plans to set reimbursement for specialty biologic drugs through value assessments conducted by a single independent review board, which could prove to be a problem for patients and vulnerable citizens. Review boards conducting similar value assessments can be rife with conflicts of interests and lean on methodologies that disadvantage certain drugs and the patients who may desperately need them.

A recent Institute for Clinical and Economic Review (ICER) "Report on Unsupported Price Increases," concluded that: "Among the top drugs with price increases in 2019...ICER determined that seven of 10 lacked adequate new evidence to demonstrate a substantial clinical benefit that was not yet previously known."  The impression left by the report is that drug companies arbitrarily raise prices without good reason.  As with so many ICER products, the study is misleading and demonstrates a profound lack of business acumen.

NJ Pride

New Jersey prides itself in having one of the most diverse populations in the country. It also has the most diverse city. Jersey City was selected as the most ethnically diverse city in the country in a recent study from the personal finance outlet WalletHub.com. It's a distinction that city has been given many times by many different organizations. "Being named the most diverse city in the nation once again is more than just a ranking to us here in Jersey City, it is a fact that highlights all we have to offer as one of the state's largest cities, as a sanctuary city and as a place where we support all of our residents, regardless of ethnicity, race or gender as we see our diversity as an asset that sets us apart as the best place to live and work," said Mayor Steve Fulop.

BioNJ in the News

Nasdaq Congratulates

BioNJ Chair Will Lewis
on the Nasdaq Tower in
Times Square, New York.

BioNJ, the life sciences trade association for New Jersey, announced the election of its 2021 officers and board members. Newly elected officers include Chair Will Lewis, JD, MBA, Chair and CEO, Insmed, Inc.; Vice Chair Monika A. Vnuk, M.D., Vice President, R&D Business Development, Pfizer, Inc.; Treasurer Greg Oakes, MBA, President, North America, Vifor Pharma and re-elected as Secretary Michele Korfin, R.Ph., MBA, Chief Operating Officer & Chief Commercial Officer, Gamida Cell. Mr. Lewis succeeds Christopher J. Schaber, Ph.D., President and CEO, Soligenix, Inc., who concluded a two-year term moving into the Immediate Past Chair's role.

Globally, the industry has stepped up with lightening speed. At this point, there are over 800 different programs that are addressing COVID - whether therapies, vaccines, testing, etc. More than 70 of these companies are based in New Jersey. Many are working on more than one COVID program. The first FDA approved saliva test was developed early on by New Jersey's own Rutgers University, and, BioNJ member Pfizer, that has a large presence in New Jersey, delivered the first vaccine - with additional vaccines on the immediate horizon coming from legendary New Jersey companies, including J&J, Sanofi and GSK, not to mention the scores of smaller companies.

At a discussion titled "Keeping the Innovation State Growing" recently, the panelists agreed on one point: Work at tech and life sciences companies will look dramatically different in the years to come. This trend presents opportunities for New Jersey and its real estate sector, and the State should be ready to capitalize on them. The panel discussion took place at a conference called "Opportunities for NJ's Innovation Economy during COVID-19".

NJ Company News

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced the initiation of the second stage of the FITE19 clinical trial to assess PTC299 in COVID-19 patients. PTC299 is an oral investigational drug with a novel dual mechanism of action that has the potential to inhibit viral replication and attenuate the uncontrolled inflammatory response that ensues after infection. PTC299 functions by targeting a cellular enzyme, dihydroorotate dehydrogenase (DHODH). By targeting this enzyme instead of a viral protein, it makes it less likely to elicit drug-resistance. Enrollment for the second stage of the trial has been initiated in multiple centers outside of the US.

Bedminster-based BioNJ Member Kyowa Kirin Co., Ltd. announced that the Phase 2 study of investigational agent KHK4083 in patients with atopic dermatitis has met the primary endpoint. KHK4083 is a potential first-in-class, anti-OX40 fully human monoclonal antibody for the treatment of autoimmune diseases, including atopic dermatitis. KHK4083 was first discovered by Kyowa Kirin, and is produced with the company's patented POTELLIGENT® defucosylation technology to enhance its antibody dependent cellular cytotoxicity (ADCC) activity. The combination of ADCC and antagonist activity against OX40 may suppress inflammatory responses found to be the cause of atopic dermatitis.

Cranbury-based BioNJ Member Amicus Therapeutics announced positive initial results from its first in-human study of its CLN3 Batten disease gene therapy program, AT-GTX-502. The Abigail Wexner Research Institute (AWRI) at Nationwide Children's Hospital is conducting the ongoing Phase 1/2 clinical study of a single one-time administration of AT-GTX-502 gene therapy for classic juvenile neuronal ceroid lipofuscinosis (JNCL), also known as CLN3 Batten disease. With no approved treatments, CLN3 Batten disease is a fatal neurologic disease that leads to blindness, motor impairment, learning difficulties, epilepsy and, ultimately, premature death.

Princeton-based BioNJ Member Photocure ASA announced a new study in Germany based on real life claims data compares costs of initial TURBT in bladder cancer treatment using standard white light- vs. blue light-TURBT in 4500+ patients. Costs were calculated from the perspective of the statutory health insurances (GKV), including costs such as: outpatient, inpatient, medicinal products, appliances, remedies and sick pay. Cystectomy was identified as a major contributor to costs, masking TURBT-mediated effects. Excluding patients undergoing cystectomy, mean total costs associated with an initial blue light TURBT were comparable to that of a white light TURBT.

Princeton-based BioNJ Member Genmab A/S and Seagen Inc. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for tisotumab vedotin. This BLA requests FDA approval of tisotumab vedotin for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy. The submission is based on the results of the innovaTV 204 pivotal phase 2 single-arm clinical trial evaluating tisotumab vedotin as monotherapy in this setting. 

Parsippany-based BioNJ Member Ferring Pharmaceuticals, Rebiotix Inc., a Ferring Company, and MyBiotics Pharma Ltd. announced a multi-year strategic collaboration to develop live microbiota-based biotherapeutics to address bacterial vaginosis, a common vaginal infection among women of reproductive age linked to increased risk of miscarriage and complications for pregnancy and fertility. The collaboration is an important step forward in harnessing the power of the human microbiome in this area of women's health. The multi-year agreement combines MyBiotics' unique culturing, delivery and colonisation technologies aimed at restoring microbiome equilibrium with Rebiotix's expertise in developing clinical-stage live microbiota-based biotherapeutic products and Ferring's therapeutic development and commercial expertise

Berkeley Heights-based BioNJ Member Amgen announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review for sotorasib for the treatment of patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), following at least one prior systemic therapy. The FDA grants Priority Review to applications for medicines that offer significant improvements over available options by demonstrating safety or efficacy improvements, preventing serious conditions, or enhancing patient compliance. Based on the Priority Review designation, the Prescription Drug User  Fee Action (PDUFA) date for sotorasib is Aug. 16, 2021.

Jersey City-based BioNJ Member SCYNEXIS, Inc. announced it has entered into a licensing agreement and strategic partnership with Hansoh Pharmaceutical Group Company Limited, one of the leading biopharmaceutical companies in China. Under the terms of the agreement, Hansoh will obtain an exclusive license from SCYNEXIS to research, develop and commercialize ibrexafungerp in the Greater China region. Ibrexafungerp is a first-in-class, broad-spectrum triterpenoid antifungal agent providing the therapeutic advantages of both intravenous and oral formulations.

Bedminster-based BioNJ Member Tyme Technologies, Inc. announced that it has received notification that the United States Patent and Trademark Office has granted additional patent claims related to the Company's metabolomic technology platform. The patent, U.S. Patent No. 10,905,698, is directed to methods for treating COVID-19. Unlike immune therapies that depend upon the structure of the external virus coat of COVID-19 where the therapy directs its attack, we believe TYME-19 is agnostic to this structure and any mutations to the viral coat. Like other TYME agents, TYME-19 affects cellular metabolism. It constrains viral replication after a virus has inserted its genetic blueprint into an infected cell by inhibiting the ability of the virus to use the cells synthetic apparatus to make viral proteins and lipids.

Bedminster-based BioNJ Member Tyme Technologies, Inc. announced that it closed its previously announced registered direct offering of 40,000,000 shares of its common stock, par value $0.0001 per share, at a purchase price of $2.50 per share, priced at-the-market under Nasdaq rules. The gross proceeds of the offering were $100 million, prior to deducting placement agent's fees and other offering expenses payable by TYME. TYME intends to use the net proceeds from the offering for the development of the company's clinical and preclinical assets and for general corporate purposes, capital expenditures, working capital and general and administrative expenses.

Basking Ridge-based BioNJ Member Caladrius Biosciences, Inc. announced that it has closed on its previously announced sale to institutional investors of an aggregate of 24,906,134 shares of its common stock and warrants to purchase up to an aggregate of 12,453,067 shares of common stock at a purchase price of $2.45 per share of common stock and associated warrant in a registered direct offering priced at-the-market under Nasdaq rules. The warrants have an exercise price of $2.90 per share, are immediately exercisable, and will expire five years from the issuance date.

Edison-based BioNJ Member Hepion Pharmaceuticals, Inc. announced the pricing of its underwritten public offering of 44,200,000 shares of its common stock at a public offering price of $2.00 per share, for gross proceeds of $88.4 million before deducting underwriting discounts, commissions and other offering expenses. All of the shares of common stock are being offered by the company. Hepion intends to use the net proceeds from the offering to fund research and development activities, as well as for working capital and other general corporate purposes.

Madison-based BioNJ Member AbbVie and Caribou Biosciences, Inc., a leading clinical-stage CRISPR genome editing biotechnology company, announced that they have entered into a collaboration and license agreement for the research and development of chimeric antigen receptor (CAR)-T cell therapeutics. Although allogeneic, "off-the-shelf" CAR-T cell therapies have shown early promise in some cancer patients, the need for overcoming the rejection of allogeneic CAR-T cells by the host immune system remains a key challenge to their broader development. Employing Caribou's CRISPR genome editing platform to engineer CAR-T cells to withstand host immune attack would enable the development of the next-generation of "off-the-shelf" cellular therapies to benefit a broader patient population.

New Brunswick-based BioNJ Member Johnson & Johnson announced that Janssen-Cilag International N.V., has submitted a conditional Marketing Authorisation Application (cMAA) to the European Medicines Agency (EMA) seeking authorisation for its investigational single-dose Janssen COVID-19 vaccine candidate. The submission is based on topline efficacy and safety data from the Phase 3 ENSEMBLE clinical trial. The company announced in December it had initiated a rolling submission with the EMA for its investigational Janssen COVID-19 vaccine, enabling the EMA to review data as they become available.

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the final analysis of the Phase 3 TITAN study, which demonstrated the continued statistically significant benefit of the addition of ERLEADA® (apalutamide) to androgen deprivation therapy (ADT) in overall survival (OS) in patients with metastatic castration-sensitive prostate cancer (mCSPC), regardless of extent of disease, when compared to placebo plus ADT. With nearly four years of median follow-up, data from the final analysis of the Phase 3 TITAN study confirmed that ERLEADA® plus ADT provided a statistically significant improvement in OS with a 35 percent reduction in risk of death versus ADT alone (HR 0.65; p<0.0001).

Raritan-based BioNJ Member the Janssen Pharmaceutical Companies of Johnson & Johnson announced results from the randomized, double-blind, placebo-controlled Phase 3 ACIS study, which met the primary endpoint of radiographic progression-free survival (rPFS) with a 31 percent reduction in the risk of radiographic progression or death in patients with chemotherapy-naïve metastatic castration-resistant prostate cancer (mCRPC) receiving androgen deprivation therapy (ADT). Patients in the trial received either a combination of ERLEADA® and ZYTIGA® plus prednisone (combination arm) or placebo and ZYTIGA® plus prednisone (control arm).

Bridgewater-based BioNJ Member Eli Lilly and Company and Rigel Pharmaceuticals, Inc. announced a global exclusive license agreement and strategic collaboration to co-develop and commercialize Rigel's R552, a receptor-interacting serine/threonine-protein kinase 1 (RIPK1) inhibitor, for all indications including autoimmune and inflammatory diseases. Pursuant to the collaboration, Lilly will also lead all clinical development of brain penetrating RIPK1 inhibitors in central nervous system (CNS) diseases.

Bridgewater-based BioNJ Member Eli Lilly and Company announced the U.S. Food and Drug Administration (FDA) granted Emergency Use Authorization (EUA) for investigational bamlanivimab (LY-CoV555) 700 mg and etesevimab (LY-CoV016) 1400 mg together. This therapy is authorized for the treatment of mild to moderate COVID-19 in patients aged 12 and older who are at high risk for progressing to severe COVID-19 and/or hospitalization. Bamlanivimab and etesevimab should be administered together via a single intravenous infusion as soon as possible after a positive COVID-19 test and within 10 days of symptom onset.

Bridgewater-based BioNJ Member Eli Lilly and Company announced Tirzepatide led to significant A1C and body weight reductions from baseline in adults with type 2 diabetes in SURPASS-3 and SURPASS-5 phase 3 clinical trials after 52 weeks and 40 weeks, respectively. In topline results, the primary and all key secondary endpoints were met for both estimands in SURPASS-3, which compared tirzepatide to titrated insulin degludec, and in SURPASS-5, which compared tirzepatide to placebo, both as an add-on to titrated insulin glargine.

BioNJ Member Pfizer, with offices in Peapackand BioNTech SE announced an agreement with the European Commission (EC) to supply an additional 200 million doses of COMIRNATY®, the companies' COVID-19 Vaccine, to the 27 European Union (EU) member states. The EC has the option to request supply of an additional 100 million doses. 
This new agreement is in addition to the 300 million doses that have already been committed to the EU through 2021 under the first supply agreement signed last year. The additional 200 million doses are expected to be delivered in 2021, with an estimated 75 million to be supplied in the second quarter.

BioNJ Member Pfizer, with offices in Peapackannounced that the first participant has been dosed in the registration-enabling Phase 2 MagnetisMM-3 study (NCT04649359) of elranatamab (PF-06863135), an investigational B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody, in patients with relapsed/refractory multiple myeloma. The study evaluates the efficacy and safety of elranatamab, administered subcutaneously, in patients with disease that is refractory to at least one agent in each of three major classes of medications approved for multiple myeloma. The study's estimated primary completion date is June 2022.

BioNJ Member Pfizer, with offices in Peapackannounced that the U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for PANZYGA® (Immune Globulin Intravenous [Human] - ifas 10% Liquid Preparation) to treat adult patients with a rare neurological disease of the peripheral nerves called chronic inflammatory demyelinating polyneuropathy (CIDP). PANZYGA is the only intravenous immunoglobulin (IVIg) with two FDA-approved maintenance dosing options for CIDP, helping to meet the clinical needs of patients. PANZYGA can also be administered at infusion rates up to 12 mg/kg/min.

Bridgewater-based BioNJ Member Sanofi announced the U.S. Food and Drug Administration (FDA) has approved the PD-1 inhibitor Libtayo® (cemiplimab-rwlc) as the first immunotherapy indicated for patients with advanced basal cell carcinoma (BCC) previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate. Full approval was granted for patients with locally advanced BCC and accelerated approval was granted for patients with metastatic BCC. Libtayo is the first treatment to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial.

Bridgewater-based BioNJ Member Sanofi announced the European Commission has approved an additional indication for Plavix® (clopidogrel) to include its use in combination with aspirin in adult patients with moderate to high-risk Transient Ischemic Attack (TIA) (ABCD2 score ≥4) or minor Ischemic Stroke (IS) (NIHSS1 ≤3) within 24 hours of either the TIA or IS event. Usage under this new indication can continue for 21 days, followed by long-term single anti-platelet therapy. The additional indication is based on the results of two double-blind, randomized, placebo-controlled investigator-initiated Phase 3 trials involving more than 10,000 patients. 

East Hanover-based BioNJ Member Novartis announced that the U.S. Food and Drug Administration (FDA) has approved the following expanded indication for Entresto® (sacubitril/valsartan): to reduce the risk of cardiovascular death and hospitalization for heart failure in adult patients with chronic heart failure. Benefits are most clearly evident in patients with left ventricular ejection fraction (LVEF) below normal. The label also states LVEF is a variable measure and clinical judgment should be used in deciding whom to treatFor the first time, there is a treatment with benefit for patients diagnosed with guideline-defined heart failure that includes both those with heart failure with reduced ejection fraction (HFrEF) and many with heart failure with preserved ejection fraction (HFpEF).

East Hanover-based BioNJ Member Novartis announced that it has entered into a grant agreement with the Bill & Melinda Gates Foundation. As part of the agreement, the foundation will provide funding support for the discovery and development of a single-administration, in vivo gene therapy to cure sickle cell disease (SCD). The project brings together Novartis drug discovery and gene therapy expertise with the Gates Foundation's charitable objectives to expand access to health care in low-resource settings in an effort to address this potentially life-threatening genetic disease.

East Hanover-based BioNJ Member Novartis announced new multicenter survey results showing that people living with multiple sclerosis (MS) and nurses prefer the Sensoready® autoinjector pen for self-administration of Kesimpta® (ofatumumab) over other autoinjectors used for other disease-modifying therapies in MS. These data continue to show that Kesimpta has the potential to become a first-choice treatment option, as ease of administration plays an important role in patient satisfaction and treatment adherence.

East Hanover-based BioNJ Member Novartis announced that asciminib -- a novel investigational treatment specifically targeting the ABL myristoyl pocket (STAMP) -- has been granted Breakthrough Therapy designation (BTD) by the US Food and Drug Administration (FDA) for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). Asciminib was also granted BTD for the treatment of adult patients with Ph+ CML in CP harboring the T315I mutation.

East Hanover-based BioNJ Member Novartis announced that Sandoz, a Novartis division, has signed an agreement to acquire GSK's cephalosporin antibiotics business, reinforcing its leading global position in antibiotics. The agreement includes the global rights to three established brands (Zinnat®, Zinacef® and Fortum®)) in more than 100 markets. It excludes the rights in the US, Australia and Germany to certain of those brands, which were previously divested by GSK, and in India, Pakistan, Egypt, Japan (to certain of the brands) and China, which will be retained by GSK.

Warren-based BioNJ Member GlaxoSmithKline plc announced that patient dosing has begun in a Phase III clinical programme investigating the immunogenicity, safety, reactogenicity and persistence of its Respiratory Syncytial Virus (RSV) candidate vaccine for older adults (GSK3844766A), following  the release of positive Phase I/II results on safety, reactogenicity and immunogenicityRSV represents a significant health threat for older adults (>60 years of age), with 360,000 hospitalisations and 24,000 deaths associated with RSV infections estimated annually in developed countries.

Princeton-based BioNJ Member Bristol Myers Squibb announced that the European Commission (EC) has granted full Marketing Authorization for Inrebic® (fedratinib) for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adult patients with primary myelofibrosis, post-polycythaemia vera myelofibrosis or post-essential thrombocythaemia myelofibrosis, who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. Inrebic is the first, once-daily oral therapy to significantly reduce spleen volume and symptom burden for patients with myelofibrosis where treatment with ruxolitinib has failed, who are intolerant to ruxolitinib or who are JAK inhibitor naïve. 

Princeton-based BioNJ Member Bristol Myers Squibb and Exelixis, Inc. announced results from new analyses from the pivotal Phase 3 CheckMate -9ER trial, demonstrating clinically meaningful, sustained efficacy benefits as well as quality of life improvements with the combination of OPDIVO® (nivolumab) and CABOMETYX® (cabozantinib) compared to sunitinib in the first-line treatment of advanced renal cell carcinoma (RCC). With a median follow-up of two years (23.5 months), OPDIVO in combination with CABOMETYX continued to show superior progression-free survival (PFS), objective response rate (ORR) and overall survival (OS) versus sunitinib, with a low rate of treatment-related adverse events (TRAEs) leading to discontinuation.

Princeton-based BioNJ Member Bristol Myers Squibb announced results from the Phase 3 CheckMate -274 trial, which showed that Opdivo (nivolumab) significantly improved disease-free survival (DFS) as an adjuvant treatment across all randomized patients with surgically resected, high-risk muscle-invasive urothelial carcinoma and in the subgroup of patients whose tumors express PD-L1 ≥1%, meeting both of the study's primary endpoints. CheckMate -274 is the first positive Phase 3 trial evaluating an immunotherapy in the adjuvant setting of muscle-invasive urothelial carcinoma

Kenilworth-based BioNJ Member Merck & Co. and AstraZeneca announced the Phase 3 OlympiA trial for LYNPARZA will move to early primary analysis and reporting following a recommendation from the Independent Data Monitoring Committee (IDMC). Based on the planned interim analysis, the IDMC concluded that the trial crossed the superiority boundary for its primary endpoint of invasive disease-free survival (iDFS) versus placebo in the adjuvant treatment of germline BRCA-mutated (gBRCAm), high-risk human epidermal growth factor receptor 2 (HER2)-negative early stage breast cancer following definitive local treatment and neoadjuvant or adjuvant chemotherapy.

Kenilworth-based BioNJ Member Merck & Co. and Eisai announced the first presentation of new investigational data from the pivotal Phase 3 CLEAR study (KEYNOTE-581/Study 307). The trial evaluated the combinations of KEYTRUDA, Merck's anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, and LENVIMA plus everolimus versus sunitinib for the first-line treatment of patients with advanced renal cell carcinoma (RCC). KEYTRUDA plus LENVIMA demonstrated statistically significant and clinically meaningful improvements in progression-free survival (PFS; HR=0.39 [95% CI: 0.32-0.49]; p<0.001), overall survival (OS; HR=0.66 [95% CI: 0.49-0.88]; p=0.005) and objective response rate (ORR; relative risk=1.97 [95% CI: 1.69-2.29]; p<0.001) versus sunitinib. 

Basking Ridge-based Regeneron Pharmaceuticals, Inc. announced The Lancet published results from a pivotal trial designed to evaluate the investigational use of the PD-1 inhibitor Libtayo® (cemiplimab) compared to platinum-doublet chemotherapy in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with ≥50% PD-L1 expression in tumor cells. The U.S. Food and Drug Administration (FDA) granted a Priority Review with a target action date of February 28, 2021The safety of Libtayo in the trial was generally consistent with previous Libtayo pivotal trials, and according to the publication, consistent with the safety profiles of other PD-1 or PD-L1 inhibitors in NSCLC and other tumor types.

Basking Ridge-based Daiichi Sankyo Company, Limited announced that it recently entered into an outsourcing agreement with AstraZeneca K.K. to manufacture the AstraZeneca-developed COVID-19 vaccine, AZD1222, in Japan. In order to play a part in the COVID-19 vaccine manufacturing and supply scheme agreed upon between AstraZeneca and the government of Japan, Daiichi Sankyo will use undiluted solutions provided by AstraZeneca to manufacture COVID-19 vaccines in the country, including vial filling and packaging, in accordance with the outsourcing agreement. 

Woodcliff Lake-based PaxMedica, Inc. announced positive topline data from its Phase 2 dose-ranging clinical trial evaluating PAX-101 (IV suramin), an investigational drug with a novel mechanism that the Company is developing as a potential treatment for the core symptoms of Autism Spectrum Disorder. With no FDA approved treatments available currently, PAX 101 has the potential to be the first drug approved for treatment of the core symptoms of ASD. The Phase 2 study was a dose-ranging, randomized, double-blind, placebo-controlled, multidose trial evaluating the safety and efficacy of PAX-101 in patients diagnosed with moderate to severe autism spectrum disorder.

Lawrenceville-based Ellodi Pharmaceuticals announced that APT-1011 (fluticasone propionate oral disintegrating tablet) has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of Eosinophilic Esophagitis (EoE), a rare, chronic allergic inflammatory disease characterized by eosinophilic infiltration of the esophagus. The Fast Track designation was granted based on data from the Phase 2b study (FLUTE) evaluating histological remission and symptomatic improvement after 12 weeks of treatment (induction), and the long-term maintenance of these improvements. 

Jazz Pharmaceuticals plc, with offices in Ewing, announced the completion of the rolling submission for the supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking marketing approval for Xywav™ (calcium, magnesium, potassium, and sodium oxybates) oral solution for the treatment of adult patients with idiopathic hypersomnia. If approved, Xywav will be the first and only approved treatment in the U.S. for adults with idiopathic hypersomnia. 

Paramus-based BrainStorm Cell Therapeutics Inc. announced that it recently concluded a Type C meeting with the U.S. Food and Drug Administration (FDA) to review specific aspects of the company's planned manufacturing modifications to support the development of a semi-automated commercial manufacturing process for NurOwn® (MSC-NTF cell). The meeting included a detailed review of the requirements for comparability testing to support future modifications along with geographic considerations in the sourcing of starter materials and future manufacturing production.

Somerset-based Catalent announced it has signed an agreement with a German biotech company to support the manufacturing of a therapy product. Catalent will support Trizell GmbH's Phase 1 cell therapy for the treatment of micro- and microangiopathies, it said in a news release. Manufacturing of the cell therapy will take place at Catalent's facility in Gosselies, Belgium. "Our relationship with Trizell will build on Catalent's deep knowledge in immunotherapies, from the diagnosis and de-risk stage all the way through to commercial-ready processes," Manja Boerman, President, Catalent Cell & Gene Therapy.

Thor Sciences has started on the base building infrastructure for 95G, a 350,000-square-foot property at 95 Greene St. The eight-story building is being repositioned for research & development use by SGA Architects, and JLL has been chosen as exclusive leasing agent. "The property stands at the center of a diverse and highly educated talent pool and provides easy access to the region's medical centers, higher education institutions and accelerators that are at the source of the remarkable life sciences growth in the region," said Joseph Sitt, Chairman of Thor Equities.

People in the News

Pennington-based BioNJ Member OncoSec Medical Incorporated announced the appointment of Bridget O'Keeffe, Ph.D., a biotech industry veteran with more than 15 years of experience, as Vice President of Clinical Development. Dr. O'Keeffe most recently served as Senior Director of Clinical Development for Nektar Therapeutics, where she built and trained an oncology clinical science team and provided clinical development leadership in the development of company processes and policy through interdepartmental, company-wide committees, working groups and initiatives. 

Bridgewater-based BioNJ Member Eli Lilly and Company announced the board of directors of Eli Lilly and Company has elected  Kimberly H. Johnson as a new member. As a member of Lilly's board, she will serve on both the Compensation Committee and the Ethics and Compliance CommitteeMs. Johnson currently serves as executive vice president and chief operating officer of the Federal National Mortgage Association and is a member of Fannie Mae's Management Committee. Prior to serving as Executive Vice President and Chief Operating Officer,  Ms. Johnson held various leadership positions with Fannie Mae, including Chief Risk Officer. 

Princeton-based BioNJ Member Evotec announced that Dr. Cord Dohrmann has been appointed to the German Council of Science and Humanities for a term of three years. He is appointed by the Federal President Frank-Walter Steinmeier on the joint recommendation of the German Federal Government and the governments of the German states. Dr. Dohrmann joins the German Council of Science and Humanities as one of eight public figures and with more than 25 years of experience in biomedical research at leading academic institutions and in the biotech industry. 

Princeton-based Slayback Pharma LLC announced the appointment of Dave Picard as the company's Chief Commercial Officer. Mr. Picard will be based out of Slayback's Princeton office. He is a senior executive with an established track record of successfully leading sales teams and growing businesses. Throughout his career, he has held commercial positions of increasing responsibility at companies like AmerisourceBergen, Sandoz and Bristol Myers Squibb. His experience spans across various sales channels including hospital, clinic, retail and specialty.

Institution and Education News


Passaic County Technical-Vocational Schools (PCTVS), together with Passaic County Community College (PCCC) are seeking employer input to ensure that the Academy's initiatives directly address the workforce needs of the State's life sciences industry. Specifically, they are requesting information on your anticipated employment needs; skills and competencies required of entry level workers; and educational and work-based learning opportunities available within your company. Please take a few minutes to complete the brief Employer Survey. To further discuss the project, please contact Mr. Todd Sorber, Vice President of Institutional Advancement at Passaic County Community College (PCCC) at (973) 684-5656 or by email at tsorber@pccc.edu.

Researchers from Rutgers Cancer Institute of New Jersey, the state's only National Cancer Institute-designated Comprehensive Cancer Center, evaluated the frequency of SARS-CoV-2, the virus that causes COVID-19, on various environmental surfaces in outpatient and inpatient hematology/oncology settings located within Rutgers Cancer Institute and Robert Wood Johnson University Hospital, an RWJBarnabas Health facility. The study revealed extremely low detection of SARS-CoV-2 on environmental surfaces across multiple outpatient and inpatient oncology areas, including an active COVID-19 floor.

Anita Kinney, Ph.D., Director of the Center for Cancer Health Equity at Rutgers School of Public Health and Rutgers Cancer Institute of New Jersey, has been elected as the  2021 President-Elect of the American Society of Preventive Oncology (ASPO), a multi-disciplinary society that is committed to serving as an advocate for cancer prevention and control research. In this role, she will serve a two-year term prior to serving as President of the society. Dr. Kinney, who is also Associate Director for Population Science and Community Outreach at Rutgers Cancer Institute of New Jersey, Director of ScreenNJ and a professor of Biostatistics and Epidemiology at the Rutgers School of Public Health, has been an actively funded investigator in the area of cancer prevention and control for more than 25 years.

World Cancer Day is an international initiative uniting people around the world in an effort to raise awareness and take action. This year's theme -- "Together, all our actions matter" -- calls for individuals to reflect on the idea that collaboration and collective action make us stronger in the fight against cancer and spreads the powerful message that we all have a  role to play in reducing the global impact of the disease. At Rutgers Cancer Institute of New Jersey in partnership with RWJBaranabas Health, experts are always working toward the goal of helping individuals fight cancer through onsite research, clinical trials and collaborations with teams locally and across the globe.

Funding News

The New Jersey Economic Development Authority (NJEDA) announced that it has launched a website to provide information on the recently signed Economic Recovery Act of 2020 and collect input from the public on the implementation of the package of tax incentive, financing and grant programs that will be created under the Act. Governor Phil Murphy signed the Economic Recovery Act into law on January 7, 2021 to address the ongoing economic impacts of the COVID-19 pandemic and build a stronger, fairer New Jersey economy. 

The New Jersey Economic Development Authority (NJEDA) introduced NJ Capital Connections, new quarterly newsletter to showcase the multitude of angel and venture capital (VC) funding activity taking place within the Garden State. Each issue highlights the top deals of the previous quarter and offer insight into current funding trends. The newsletter will complement the newly launched Angel/VC Funding website that also serves to showcase the Angel/VC activity within the Garden State. 

The New Jersey Commission on Science, Innovation and Technology (CSIT) opened the application for its Clean Tech Seed Grant Program. The program will help accelerate development and innovation of clean technologies by furthering research and development (R&D) within the Garden State's clean technology startup community. CSIT developed the program in coordination with the New Jersey Board of Public Utilities (NJBPU) and the New Jersey Economic Development Authority (NJEDA)The application is available at: https://application.njeda.com/csit. 

The New Jersey Economic Development Authority (NJEDA) announced that interest in its Angel Investor Tax Credit Program saw record levels in 2020, as investors submitted more than 700 applications, nearly three times the average of previous program years. Applications also hit the legislative cap of $25 million in late October, marking the first time the program cap had ever been reached. The Angel Investor Tax Credit Program offers investors refundable tax credits against qualified investments for New Jersey businesses. The program supports technology businesses with a physical presence in New Jersey that conduct research, manufacturing, or technology commercialization in the State. 

Thought Leadership from BioNJ Members

Written By J.P. Morgan

In 2020, constant change was the only norm, and the ability to adapt became a more essential leadership skill than ever. Three start-up leaders from different industries -- medical devices, fashion and telehealth -- share the invaluable lessons they learned during a year unlike any other.

Special Programs from Our Partners for BioNJ Members

February 23, 2021

As the industry moves to increase domestic production and capacity, NJ is uniquely positioned to welcome new life sciences businesses, from research and development to the manufacturing of vaccine components. NAIOP NJ's webinar will bring together experts to provide: a primer on the life sciences sector, emerging opportunities to retrofit existing space and what you must know about the specific needs of these atypical end users (zoning restrictions, high costs of space conversions, insurance considerations in multi-tenanted buildings, hazmat risks).

February 24-25, 2021

Penn's Orphan Disease Center (ODC) is the first of its kind, a program dedicated to identifying promising therapies for often overlooked disorders and providing access to those therapies for patients of all populations. Led by world-renowned gene therapy pioneer Jim Wilson, the ODC has a track record of accelerating progress across a number of arenas, including infectious diseases, blood disorders, metabolic diseases, and neurologic disorders like Rett syndrome, CDKL5 deficiency disorder, and Duchenne muscular dystrophy. Join us as we take a look at where we have been -- and where we are going. 

February 25, 2021

Get to know these entrepreneur-scientists, help them succeed and get an inside look at the newest cell and gene therapy breakthroughs coming out of university research. Investors, accelerators, fellow entrepreneurs, students and members of the community are all welcome to attend. After pitching, an interactive virtual expo will be available for guests to speak with founders and sponsors. The format is flexible so feel free to drop in whenever your schedule permits!

March 4, 2021

On March 4, rare disease patients, families, caregivers and advocates will come together for Rare Disease Day in Trenton. This annual event will be virtual in 2021 and help raise awareness with our policymakers on the impact of rare diseases on patients and their families. Click here for more information on 2021 Rare Disease Day in Trenton.

March 15-17, 2021

Take advantage of this unique opportunity for professionals to make connections, start collaborations and forge partnerships in North America's life sciences and health technology hub. The agenda co-created by the organizing committee is aimed at entrepreneurs, scientists, researchers, students, investors and life sciences and health technology professionals in Canada and abroad. 

March 16-18, 2021

Join over 150 leading VCs, Corp VCs, Angel Investors and over 100 Top Innovators at #VSVC Venture Summit Virtual Connect West. Dedicated Life sciences / Health care Track, One-on-One (zoom) investor meetings, two days of content, including timely panel discussions, workshops and an inspiring keynotes. Contact RBromberg@BioNJ.org for 20% discount code for BioNJ Members.

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:

We'd like to take this time to thank our Members for your continued support and assure you that we value our relationship. In doing so, please know that we do not share any Member contact information. Should you receive a solicitation to purchase BioNJ's Membership Contact List, please disregard it and know that it is spam and that we keep all contact information confidential. Should you have any questions or concerns, please reach out to us at BioNJ@BioNJ.org. Thank you, again.

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.