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

Thank you to the many companies that have submitted applications to present at this year's BioNJ's Virtual BioPartnering Conference, developed and presented in concert with J.P. Morgan and Johnson & Johnson InnovationThank you to our partners at JPM and J&JI for their active collaboration, engagement and counsel. It is extraordinary!
Taking place May 18 and 19, BioNJ's BioPartnering Conference is a unique opportunity to connect with life sciences executives, investors, institutional collaborators and business development professionals; schedule 1:1 meetings with potential partners; present and watch Company and Start-up Pitch Presentations; hear from industry experts; discuss business and research opportunities with world-renowned institutions and network directly with other conference attendees. Click here for a list of topics and speakers. 
The deadline to apply to present your own life sciences company is MONDAY, MARCH 22. Click here to learn more and take advantage of this great opportunity to move your science (including diagnostics, digital medicine/medtech technologies & health ITforward. 
Contact Samantha Bamberger at SBamberger@BioNJ.org with any questions or Kim Minton at KMinton@BioNJ.org to learn about Sponsorship opportunities. 

Meanwhile lots happening on the policy front. Please see this week's Public Policy Update here. Thank you!

Because Patients Can't Wait®,
The BioNJ Team

Medical Supply Chain Eligible for Vaccine Starting March 29

Many have questions on accessing the vaccine once the biopharmaceutical sector becomes eligible on March 29. In particular, individuals are seeking more information on how to update an existing registration in the New Jersey Vaccine Scheduling System. There are two ways to update a NJVSS registration: 
  1. Complete an online form at covid19.nj.gov/vaccinehelp
  2. Call 855-568-0545 for phone support
Additional information will be forthcoming on how employers will be able to play a role in employee COVID-19 vaccinations. More information on vaccine eligibility and prioritization as well as approved vaccine site locations is available at the NJ COVID-19 Information Hub.   

BioNJ Calendar

BioNJ's Diversity, Equity and Inclusion Conference...Because It Matters
March 24-25, 2021
BioNJ BioPartnering Virtual Conference With J.P. Morgan and Johnson & Johnson Innovation
May 18-19, 2021
BioNJ Membership Webinar: Learn About the Benefits of Membership
April 15, 2021
Putting Patients First: The Value of Medical Innovation

A commonly-used analysis to determine a medicine's value is based on flawed methodologies that would diminish innovation and access, finds a new report released by the nonpartisan Center for Medical Economics and Innovation at the Pacific Research Institute. "Cost effectiveness reports may provide precise estimates, but there is no reason to believe that these estimates accurately reflect the value of medicines," said Dr. Wayne Winegarden, the brief's author.  "The documented biases in their value assessments should raise serious concerns that ICER's work is inaccurate and biased toward undervaluing medicines."

NJ Pride

Governor Phil Murphy signed SJR82, which designates March 9th of each year as "COVID-19 Heroes Day" in New Jersey. "From nurses and doctors to grocery store workers and law enforcement, countless essential workers have been working day and night to serve New Jerseyans during these trying times," said Governor Murphy. "It is my honor to sign this bill recognizing the heroic efforts of those who have been serving on the front lines. These heroes have brought us to this moment where we can finally see the light at the end of the tunnel."

NJ Company News

Florham Park-based BioNJ Member Celularity Inc. announced that the company has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for its non-genetically modified cryopreserved human placental hematopoietic stem cell-derived natural killer (NK) cell therapy, CYNK-001, for the treatment of adults with recurrent glioblastoma multiforme (GBM). CYNK-001 is currently being investigated in multiple clinical trials, including a Phase 1 clinical trial for GBM. "We believe the unique attributes of placental-derived cell therapies will allow us to not only overcome the challenges of NK cell proliferation and persistence, but also improve therapeutic availability as compared to adult bone marrow or peripheral blood approaches," said Robert J. Hariri, M.D., Ph.D., Founder, Chairperson and Chief Executive Officer of Celularity.

Morristown-based BioNJ Member BioAegis Therapeutics Inc. announced that it has completed enrollment for its Phase 2 study of hospitalized patients with severe COVID-19 pneumonia. This study assesses BioAegis' therapeutic, recombinant human plasma gelsolin (rhu-pGSN), and its unique ability to regulate the overexuberant inflammatory response which can cause organ failure and death without suppressing the immune system. Low levels of gelsolin are associated with severe illness and organ failure in COVID-19 patients. According to Susan Levinson, Ph.D., CEO of BioAegis, "Our team is grateful for the efforts of the extended clinical study teams at each site who worked tirelessly to complete recruitment during this challenging pandemic. We look forward to demonstrating the value to patient care in this and other inflammatory conditions."

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced two-year data from Part 2 of the SUNFISH trial evaluating Evrysdi™ (risdiplam) in children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA). These results demonstrated that Evrysdi patients sustained or improved in motor function after 24 months of treatment. Furthermore, the patients and caregivers reported improvements in their ability to function independently as well as their ability to complete daily tasks. "These data further support the strong U.S. launch and the need for an at home oral treatment option for the SMA community," said  Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. announced results from a real-world study of patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) who switched from prednisone to EMFLAZA® (deflazacort) following the FDA approval. The primary reason the majority of patients chose to switch to EMFLAZA was a desire to delay disease progression to improve the benefit and tolerability. During the 6-months average follow-up after switching, physician-reported outcomes were consistent with EMFLAZA addressing the primary reasons for switching. The real-world chart review was conducted between February 2017 and December 2018 by 55 neurologists who contributed data for 92 male patients with DMD or BMD that switched from prednisone to EMFLAZA.

South Plainfield-based BioNJ Member PTC Therapeutics, Inc. and The Spinal Muscular Atrophy (SMA) Foundation have entered into a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders with the goal of developing new treatments. SMA is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. The availability of several disease-modifying therapies for SMA has made regenerative interventions the next frontier in drug discovery and development. The SMA Foundation-PTC partnership will provide funding, managed by the SMA Foundation, to academic institutions and other collaborators to advance foundational research in the area of regenerative medicine.

Basking Ridge-based BioNJ Member Caladrius Biosciences, Inc. announced that the U.S. Food and Drug Administration ("FDA") has granted orphan drug designation to the Company's CD34+ cell therapy product, CLBS12, for the treatment of Buerger's disease -- also known as thromboangiitis obliterans -- a condition related to critical limb ischemia ("CLI") with no approved treatments to date in the U.S. "We are very pleased that the FDA has granted orphan drug designation to CLBS12 in Buerger's disease. Without a currently approved or effective treatment for this condition in the U.S., a significant unmet need remains for therapies that slow, stop or, ideally, reverse this debilitating disease," stated David J. Mazzo, Ph.D., President and Chief Executive Officer of Caladrius. 

Princeton-based BioNJ Member Soligenix, Inc. announced that it has received preliminary approval for a tax credit from the New Jersey Economic Development Authority's (NJEDA) New Jersey Technology Business Tax Certificate Transfer program. As a result, the company anticipates being able to transfer this credit and receive approximately $865,000 in net proceeds. "This is our eleventh year receiving NOL funding.  Over this time period, we have received approximately $6.5 million in non-dilutive NOL funding that has allowed us to advance our rare disease pipeline to where we are now preparing to begin submission of a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) in 2Q 2021 for our first-in-class therapy, SGX301 in the treatment of cutaneous T-cell lymphoma (CTCL)," stated Christopher J. Schaber, Ph.D., President and Chief Executive Officer of Soligenix.   

Princeton-based BioNJ Member Photocure announced a recently published systematic review and network meta-analysis of randomized controlled trials investigated the impact of enhanced optical techniques at time of TURBT with or without single immediate intravesical chemotherapy (SIIC) on recurrence rate of non-muscle-invasive bladder cancer (NMIBC). The study is a meta-analysis of optical enhancement techniques with or without single immediate intravesical chemotherapy, assessing the effect on 12-month recurrence. Its objective was to determine optimal initial bladder tumor management based on a combination of different optical techniques during TURBT and SIIC. 

Berkeley Heights-based Cyclacel Pharmaceuticals, Inc. announced the closing of its previously announced underwritten public offering of 2,078,214 shares of its common stock, offered at a price of $7.00 to the public, which includes the full exercise of the underwriter's over-allotment option to purchase additional shares. The gross proceeds to the company from this offering are approximately $14.5 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by the company. Existing and new institutional investors participated in the offering.

Jersey City-based BioNJ Member Mitsubishi Tanabe Pharma America, Inc. (MTPA) announced a post-hoc analysis of its Phase 3 edaravone study reviewing the results of intravenous (IV) edaravone treatment on disease progression milestones and events among people with amyotrophic lateral sclerosis (ALS). In the analysis, a risk reduction was observed for the exploratory composite estimate of time to death, tracheostomy, permanent assisted ventilation (PAV) and hospitalization. "I'm encouraged by the findings of this post-hoc analysis," said Benjamin Rix Brooks, M.D., an ALS specialist and lead author of the study. "We were able to uncover data that reinforces the need to further our research that can show an important association between edaravone use and the potential decrease in functional decline."

Jersey City-based BioNJ Member Mitsubishi Tanabe Pharma America, Inc. (MTPA) announced findings from a new analysis evaluating the impact of symptom progression on the ability of people with amyotrophic lateral sclerosis (ALS) to independently conduct activities of daily living (ADLs). For the analysis, 21 neurologists examined de-identified medical record data for 166 ALS patients in the U.S. to retroactively assess demographics, symptoms, caregiver status, hospitalizations, ability to perform ADLs, emotional well-being, and number of patient-reported falls. Of the patients whose records were reviewed, 54 percent (90 patients) were dependent on a caregiver to assist with at least one ADL. 

Bridgewater-based BioNJ Member Eli Lilly and Company announced patient-reported outcomes (PRO) for the investigational use of Verzenio® (abemaciclib) in combination with standard adjuvant endocrine therapy (ET) for the treatment of hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) high risk early breast cancer (EBC). The PRO analysis included patients in both arms of the study and measured their experiences with side effects, symptoms, and health-related quality of life, in those receiving Verzenio plus ET versus ET alone. In one analysis, the PRO data indicated that most patients (approximately 70-75%) in both arms reported being bothered "a little bit" or "not at all" by treatment-related side effects. 

Bridgewater-based BioNJ Member Eli Lilly and Company announced that mirikizumab met the primary and all key secondary endpoints in LUCENT-1, a 12-week Phase 3 induction study evaluating the efficacy and safety of mirikizumab for the treatment of patients with moderate to severe ulcerative colitis (UC). LUCENT-2, a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week LUCENT-1 induction study is ongoing. UC is a chronic inflammatory disease of the large intestine, also referred to as the colon, that affects the lining of the colon and may cause small sores, or ulcers, to form.

Bridgewater-based BioNJ Member Eli Lilly and Company and Welldoc, Inc. announced a collaboration and licensing agreement to integrate Welldoc's software into Lilly's connected insulin solutions, currently in development. Under the terms of the agreement, Lilly and Welldoc will collaborate to create a new version of the BlueStar® insulin management solution that integrates insulin dosing data for several Lilly insulins. Lilly will commercialize the pen platform, which will include the new app and Lilly's connected insulin pen solutions. The app has both prescription and non-prescription features such as insulin titration support, a bolus calculator and personalized health coaching, and integrates blood glucose monitoring and continuous glucose monitoring for people who use insulin.

Bridgewater-based BioNJ Member Eli Lilly and Company presented Phase 2 TRAILBLAZER-ALZ results published in the New England Journal of Medicine expand on previously reported top-line data that found donanemab met its primary endpoint and showed significant slowing of decline on the integrated Alzheimer's Disease Rating Scale (iADRS), a composite measure of cognition and daily function, in patients with early symptomatic Alzheimer's disease compared to placebo. "This is the first late-stage study in Alzheimer's disease to meet its primary endpoint at the primary analysis. Donanemab has the potential to become a very important treatment for Alzheimer's disease," said Daniel Skovronsky, M.D., Ph.D., Lilly's Chief Scientific Officer and President of Lilly Research Laboratories.

Bridgewater-based BioNJ Member Eli Lilly and Company and Biolojic Design Ltd. announced a research collaboration and license agreement that will leverage Biolojic's AI-based multibody platform to discover and develop a potential novel antibody-based therapy for the treatment of diabetes. A multi-specific antibody, or multibody, is a human antibody that is computationally engineered to bind two or more targets at each of its arms. Fine tuning the affinity and respective competition of the two targets can produce therapies with differential activities, which vary depending on tissue localization and metabolic parameters. The specific targets to be studied in the collaboration were not disclosed.

BioNJ Member Pfizer, with offices in Peapack, and Valneva SE announced initiation of study VLA15-221. The VLA15-221 study builds on previous positive Phase 2 studies, incorporates new dose regimens and is anticipated to be the final Phase 2 study readout before a decision to progress into pivotal Phase 3 studies. As announced in December 2020, VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. It will be the first VLA15 study to include a pediatric population (aged 5-17 years). Overall, the study will enroll approximately 600 healthy participants (aged 5-65 years) who will receive VLA15 or placebo. It will compare the three-dose vaccination schedule (Month 0-2-6) with a two-dose schedule (Month 0-6).

BioNJ Member Pfizer, with offices in Peapack, The Israel Ministry of Health (MoH) and BioNTech SE announced real-world evidence demonstrating dramatically lower incidence rates of COVID-19 disease in individuals fully vaccinated with the Pfizer-BioNTech COVID-19 Vaccine (BNT162b2), underscoring the observed substantial public health impact of Israel's nationwide immunization program. These new data build upon and confirm previously released data from the MoH demonstrating the vaccine's effectiveness in preventing symptomatic SARS-CoV-2 infections, COVID-19 cases, hospitalizations, severe and critical hospitalizations and deaths. The latest analysis from the MoH proves that two weeks after the second vaccine dose protection is even stronger -- vaccine effectiveness was at least 97% in preventing symptomatic disease, severe/critical disease and death. 

East Hanover-based BioNJ Member Novartis announced new data that reinforce the transformational benefit of Zolgensma® (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy (SMA). The overall safety profile remains favorable following presymptomatic treatment, in the long-term follow-up period from clinical studies and in the real-world setting. New data underscore the critical importance of identifying and treating SMA as early as possible. In contrast to the natural history of this devastating disease, which leads to progressive and irreversible loss of motor function, children treated with Zolgensma presymptomatically in the Phase 3 SPR1NT trial achieved age-appropriate motor milestones within the World Health Organization (WHO) window of normal development.

Morris Plains-based BioNJ Member Gilead Sciences, Inc. and Plainsboro-based BioNJ Member Novo Nordisk announced that the companies have expanded their clinical collaboration in non-alcoholic steatohepatitis (NASH). The companies will conduct a phase 2b double-blind, placebo-controlled study to investigate the safety and efficacy of Novo Nordisk's semaglutide, a GLP-1 receptor agonist, and a fixed-dose combination of Gilead's investigational FXR agonist cilofexor and investigational ACC inhibitor firsocostat, alone and in combination in people with compensated cirrhosis (F4) due to NASH. The four-arm study in approximately 440 patients will evaluate the treatments' impact on liver fibrosis improvement and NASH resolution and will begin recruitment in the second half of 2021.

Morris Plains-based BioNJ Member Gilead Sciences, Inc. presented additional results from the Phase 2/3 CAPELLA trial evaluating the company's investigational, long-acting HIV-1 capsid inhibitor, lenacapavir, in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. The data build on the positive primary endpoint resutls announced previously. The new interim efficacy results demonstrate that lenacapavir administered subcutaneously every six months maintained high rates of virologic suppression through 26 weeks in a difficult-to-treat patient population with limited therapy options and high unmet medical need. In this analysis of the ongoing maintenance period of CAPELLA, which evaluated lenacapavir in combination with an optimized background regimen, 73% (n=19/26) of participants who reached Week 26 since the first dose of subcutaneous lenacapavir achieved undetectable viral load (<50 copies/mL). 

Kenilworth-based BioNJ Member Merck & Co. and Gilead Sciences, Inc. announced that they have entered into an agreement to co-develop and co-commercialize long-acting treatments in HIV that combine Gilead's investigational capsid inhibitor, lenacapavir and Merck's investigational nucleoside reverse transcriptase translocation inhibitor, islatravir, into a two-drug regimen with the potential to provide new, meaningful treatment options for people living with HIV. Islatravir and lenacapavir are both potentially first-in-class medicines in late-stage clinical trials, with significant clinical data generated to date.

Kenilworth-based BioNJ Member Merck & Co. announced that the European Commission (EC) has approved an expanded label for KEYTRUDA, Merck's anti-PD-1 therapy, as monotherapy for the treatment of adult and pediatric patients aged 3 years and older with relapsed or refractory classical Hodgkin lymphoma (cHL) who have failed autologous stem cell transplant (ASCT) or following at least two prior therapies when ASCT is not a treatment option. This approval is based on results from the pivotal Phase 3 KEYNOTE-204 trial, in which KEYTRUDA monotherapy demonstrated a significant improvement in progression-free survival (PFS) compared with brentuximab vedotin (BV), a commonly used treatment.

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 Drug Application (NDA) for the hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor belzutifan (pronounced bell-ZOO-ti-fan), a novel investigational candidate in Merck's oncology pipeline, for the potential treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), not requiring immediate surgery. This NDA is based on data from the Phase 2 Study-004 trial, in which belzutifan showed a confirmed overall response rate of 36.1% (n=22/61) (95% CI: 24.2-49.4) in patients with VHL disease-associated RCC. 

Kenilworth-based BioNJ Member Merck & Co. announced that it has filed a Form 10 registration statement with the United States Securities and Exchange Commission ("SEC") in connection with the intended spinoff of its women's health, biosimilars and established brands businesses into a standalone, publicly-traded company, Organon. Organon will register its common stock under Section 12 of the Securities Exchange Act of 1934. "Merck is confident that the spinoff will deliver significant benefits for both companies, better meet patient and customer needs and create value for Merck shareholders," said Rob Davis, Executive Vice President, Global Services and Chief Financial Officer, Merck.

New Brunswick-based BioNJ Member Johnson & Johnson announced that the World Health Organization (WHO) has issued Emergency Use Listing (EUL) for its single-shot COVID-19 vaccine, developed by the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), to prevent COVID-19 in individuals 18 years of age and older. Data from the Phase 3 ENSEMBLE study showed that the Johnson & Johnson COVID-19 vaccine was well tolerated and demonstrated a 67 percent reduction in symptomatic COVID-19 disease in participants who received the vaccine in comparison to participants given the placebo. 

New Brunswick-based BioNJ Member Johnson & Johnson announced that the European Commission (EC) has granted a Conditional Marketing Authorization (CMA) for its single-dose COVID-19 vaccine, developed by the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen), to prevent COVID-19 in individuals 18 years of age and older. The data demonstrated the vaccine was 85 percent effective in preventing severe disease across all regions studied, and showed protection against COVID-19 related hospitalization and death, beginning 28 days after vaccination.

Raritan-based BioNJ Member The Janssen Pharmaceutical Companies of Johnson & Johnson announced long-term data from the Phase 3 DISCOVER-2a study showing that the skin clearance, joint symptom relief, and safety of TREMFYA® (guselkumab) previously demonstrated through 24 weeks and one year (Week 52) in adults with active psoriatic arthritis (PsA) continued through two years (Week 112). These findings also confirmed that the robust efficacy TREMFYA demonstrated in patients at Week 24 on physical function, physical aspects of health-related quality of life, and resolution of enthesitisb and dactylitisc was also seen through Week 100.1-8 In addition, the extent of radiographic progression was studied through two years.

Bridgewater-based BioNJ Member Sanofi and Translate Bio announced the start of the Phase 1/2 clinical trial for MRT5500, an mRNA vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. The companies expect interim results from this trial in the third quarter of 2021. Preclinical studies are ongoing and will continue over the next several months to evaluate whether MRT5500, as well as additional mRNA vaccine candidates, will induce neutralizing antibodies against the emerging SARS-CoV-2 variants, with the potential to inform current and future clinical development.

Bridgewater-based BioNJ Member Sanofi and Basking Ridge-based Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) as an add-on treatment for children aged 6 to 11 years with uncontrolled moderate-to-severe asthma. Dupixent is currently approved as an add-on treatment for patients with uncontrolled moderate-to-severe asthma aged 12 and older with elevated eosinophils or oral corticosteroid dependent asthma. The target action date for the FDA decision is October 21, 2021 and the European Union (EU) regulatory submission for children aged 6 to 11 years with asthma is planned for Q1 2021.

Bridgewater-based BioNJ Member Sanofi announced positive results demonstrating an overall survival (OS) benefit from the Phase 3 trial investigating Sanofi and Regeneron's PD-1 inhibitor Libtayo® (cemiplimab) monotherapy compared to chemotherapy in patients previously treated with chemotherapy whose cervical cancer is recurrent or metastatic. The trial will be stopped early based on a unanimous recommendation by the Independent Data Monitoring Committee (IDMC), and the data will form the basis of regulatory submissions in 2021. This is the largest Phase 3 randomized clinical trial in advanced cervical cancer and included women (median age: 51 years) with either squamous cell carcinoma or adenocarcinoma. 

Warren-based BioNJ Member GlaxoSmithKline plc announced that the first patient has been dosed in the SWIFT-2 trial as part of the phase 3 clinical programme investigating the safety and efficacy of GSK3511294 ("GSK'294") in severe eosinophilic asthma (SEA). GSK'294 is an investigational medicine with potential to be the first biologic to deliver long-acting suppression of IL-5 in patients with SEA from one subcutaneous injection every six months. GSK'294 is an anti-IL-5 monoclonal antibody currently in development for the treatment of severe eosinophilic asthma. It is a distinct, new biologic entity and has been engineered for high affinity and long-acting suppression of IL-5 function.

Warren-based BioNJ Member GlaxoSmithKline plc and Vir Biotechnology, Inc. announced that an Independent Data Monitoring Committee (IDMC) recommended that the Phase 3 COMET-ICE (COVID-19 Monoclonal antibody Efficacy Trial - Intent to Care Early) trial evaluating VIR-7831 (GSK4182136) as monotherapy for the early treatment of COVID-19 in adults at high risk of hospitalization be stopped for enrolment due to evidence of profound efficacy. Based on these results, Vir and GSK plan to submit an Emergency Use Authorization (EUA) application to the U.S. Food and Drug Administration (FDA) and for authorisations in other countries.

Berkeley Heights-based BioNJ Member Amgen and Five Prime Therapeutics announced an agreement under which Amgen will acquire Five Prime Therapeutics for $38.00 per share in cash, representing an equity value of approximately $1.9 billion. This acquisition adds Five Prime's innovative pipeline to Amgen's leading oncology portfolio. "The acquisition of Five Prime offers a compelling opportunity for Amgen to strengthen our oncology portfolio with a promising late-stage, first-in-class global asset to treat gastric cancer," said  Robert A. Bradway, Chairman and Chief Executive Officer at Amgen.

Ridgefield Park-based BeiGene, Ltd. announced that a supplemental Biologics License Application (sBLA) for anti-PD1 antibody tislelizumab was accepted by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for treatment in the second- or third-line setting of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have progressed on prior platinum-based chemotherapy.

Ridgefield Park-based BeiGene, Ltd. announced that the first patient has been dosed in a Phase 1 clinical trial of BGB-15025, its investigational hematopoietic progenitor kinase 1 (HPK1) inhibitor. BGB-15025 is designed to be a potent and highly selective small molecule oral inhibitor of HPK1, a kinase downstream of the T cell receptor (TCR) signaling pathway that is believed to play a key role in T cell activation. This first-in-human Phase 1 trial (NCT04649385) will assess the safety, tolerability, pharmacokinetics, and preliminary antitumor activity of BGB-15025 alone and in combination with tislelizumab in patients with advanced solid tumors. This trial will be conducted in multiple countries globally.

Cranbury-based Rocket Pharmaceuticals, Inc. announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). RMAT designation was granted based on encouraging preliminary safety and efficacy data from the ongoing Phase 1/2 clinical trial of RP-L201. Additionally, patient enrollment has been fully completed for the Phase 1/2 trial. The study is being conducted at the University of California Los AngelesUniversity College London (UCL)/Great Ormond Street Children's Hospital and Hospital Infantil Universitario Niño Jesús.

Florham Park-based PDS Biotechnology Corporation announced that its COVID-19 vaccine consortium consisting of PDS Biotech, Farmacore Biotechnology and Blanver Farmoquímica, has received a commitment from the Secretary for Research and Scientific Training of the MCTI to fund up to approximately US $60 million to support the clinical development and commercialization of a novel, Versamune®-based, second generation COVID-19 vaccine in Brazil. MCTI intends to start making the funds available to prepare to perform a combined Phase 1/2 clinical trial, upon authorization by the Brazilian regulatory agency, Agência Nacional de Vigilância Sanitária (Anvisa) to initiate the proposed Versamune®-based COVID-19 vaccine clinical program in Brazil.

Woodcliff Lake-based Eisai Co., Ltd. announced that it has received orphan drug designation for LENVIMA® (generic name: lenvatinib mesylate), the orally available multiple receptor kinase inhibitor discovered by Eisai, with a prospective indication for uterine body cancer, by the Ministry of Health, Labour and Welfare (MHLW). In Japan, the estimated number of patients with uterine body cancer is approximately 30,000. It is estimated that in 2020, there were more than 17,000 new cases of uterine body cancer and more than 3,000 deaths from the disease.

Chatham-based Tonix Pharmaceuticals Holding Corp. announced preliminary results following vaccination of non-human primates with TNX-1800 (modified horsepox virus, live vaccine), a live attenuated COVID-19 vaccine candidate engineered to express the SARS-CoV-2 (CoV-2) spike protein. Immunogenicity and protective efficacy of single-dose TNX-1800 were assessed at two dose levels (n=4 per group). At Day 41 after the vaccination, animals were challenged with live SARS-CoV-2 through intra-nasal and intra-tracheal routes. Protection was assessed at Day 47, six days after challenge. The research is part of an ongoing collaboration between Southern Research, the University of Alberta and Tonix.

A biotechnology company has signed a lease at a Bedminster office park, according to real estate firm JLLTessa Therapeutics signed for 12,828 square feet at 1 Crossroads Drive, part of Eden Wood Realty Crossroads Business Center. The center is a 136,000-square-foot Class A complex at 1 & 2 Crossroads DriveSingapore-based Tessa is a clinical-stage biotech developing cell therapies for cancer.

People in the News

Rutherford-based Cancer Genetics, Inc. announced the appointment of Roland J. Carrillo, Ph.D. as the Vice President of Business Development. Dr. Carrillo brings over a decade of experience with drug discovery and translational science with extensive knowledge of business development, marketing, and sales operations. His latest role was at Crown Bioscience, where he held positions as Senior Director for Sales Operations and Director of Business Development. He also led efforts in sales enablement, research service commercialization, and new business unit creation. Previously, Dr. Carrillo served in business development leadership roles at BellBrook Labs and The Jackson Laboratory.

NJBIZ announced and recognized leaders from the business community with the 2021 Digi-Tech Innovators Awards honorees. These executives represent pioneers from New Jersey who have and are, introducing significant advances in the technological and digital space at their organizations, thus enhancing the overall productivity and performance of their companies. Congratulations to these BioNJ Member honorees: Chris Colucci, Insmed; Pinkrose Hamilton, Hackensack Meridian Health; Peter Jin, Rutgers, The State University of New Jersey; and Rachel Weiss, Telehealth & New Business, Hackensack Meridian Health.

Bridgewater-based BioNJ Member Eli Lilly and Company announced the upcoming retirement of two executive committee members and the naming of their successors in the company's manufacturing operations and ethics and compliance organizations. M yles O'Neill, Senior Vice President and President of Lilly manufacturing operations, will retire May 2, 2021 after nearly 19 years of service with the company. Melissa Barnes, Senior Vice President and Chief Ethics and Compliance Officer, will retire June 27, 2021 after more than 26 years with the company.  Edgardo Hernandez, who currently serves as Senior Vice President, Global Parenteral Drug Product and Device Manufacturing, will succeed O'Neill as Senior Vice President and President of Lilly Manufacturing Operations. Alonzo Weems, presently Vice President and Deputy General Counsel, will succeed Barnes as Senior Vice President and Chief Ethics and Compliance Officer.

Kenilworth-based BioNJ Member Merck & Co. announced that Michael T. Nally, Chief Marketing Officer, Human Health, will leave the company at the end of March, and Frank Clyburn, Chief Commercial Officer, Human Health, will become President, Human Health and lead all Human Health commercial and marketing for the company. Starting April 1, 2021, Clyburn will report to Robert M. Davis, current Executive Vice President, Global Services and Chief Financial Officer; as previously announced, Davis will become President of Merck, effective April 1, 2021, at which time the company's operating divisions - Human Health, Animal Health, Manufacturing, and Merck Research Laboratories -- will begin reporting to him. Davis will become Chief Executive Officer on July 1, 2021.

Parsippany-based Diaceutics PLC announces the appointment of Nikita Lynn as Vice President of Finance. Ms. Lynn  has been promoted to this role following her tenure as Senior Director of Corporate Advancement. In her new role within Diaceutics, Ms. Lynn will be responsible for the financial operations and corporate development of the company, reporting to the CFO, Board of Directors and Non-Executive Directors. She brings extensive experience in financial reporting, taxation and auditing for public companies to the position.

Institution and Education News

Metuchen-based Tevogen Bio announces partnership with BioCentriq, a New Jersey-based cell and gene therapy development and manufacturing center, to support clinical manufacturing of Tevogen's investigational COVID-19 treatment. Under the partnership, experts from both entities will collaborate at BioCentriq's state-of-the art GMP facilities to produce Tevogen's proprietary COVID-19 targeted T cells. The teams will utilize the processes and technology required to ensure that all quality standards are met in the large scale manufacturing of Tevogen's clinical grade, allogeneic T-cell therapy.

New Jersey Governor Phil Murphy, President Daniel Chamovitz of Ben-Gurion University of the Negev (BGU) and President Joel S. Bloom of New Jersey Institute of Technology (NJIT) have unveiled a partnership that will create a world-class Institute for Future Technologies in New Jersey. Two powerhouse universities in the fields of cyber technologies and environmental engineering will come together to offer dual degrees and exciting new research opportunities. The Institute looks forward to receiving support and seed funding from the State of New Jersey.

Rutgers researchers have designed a new rapid test that can detect all three of the rapidly spreading variants of the coronavirus in a little over one hour -- much shorter than the three to five days required by current tests, which can also be more technically difficult and expensive to perform. Details and information on easily creating and running the rapid test -- which is not being patented by Rutgers because researchers believe it should be widely available to the public -- are published on the pre-print online server MedRxiv and available at no charge

Rutgers Global Health Institute has launched a program that offers crucial support to small businesses facing economic and operational challenges due to the COVID-19 pandemic. The program, Equitable Recovery for New Jersey's Small Businesses, is designed to help small businesses and non-profit organizations in low-income and minority communities. While the pandemic has adversely affected the majority of small businesses, the goal of the program is to help underserved communities in New Jersey for which the impact has been especially severe. 

A Rutgers pediatrician specializing in critical care discusses how to differentiate among multisystem inflammatory syndrome, acute COVID-19 and Kawasaki disease in children. A study recently published in the Journal of the American Medical Association determined clinical criteria for diagnosing multisystem inflammatory syndrome (MIS-C) from severe COVID-19 and Kawasaki disease, which have similar symptoms. Steven Horwitz, Assistant Professor of Pediatrics at Rutgers Robert Wood Johnson Medical School, was an author of the study that looked at 1,116 people under 21 hospitalized from March through October 2020 with symptoms that could have been caused by any of these three disorders to determine how to accurately diagnose MIS-C. 

Hackensack Meridian Health announced the formation of Hackensack Meridian Health Foundation. The newly created foundation will serve as the parent organization for the network's 10 community-based hospital foundations. In addition to raising funds for the network's local medical centers, Hackensack Meridian Health Foundation will also focus on network-wide priorities such as behavioral health, children's health, the Hackensack Meridian Center for Discovery & Innovation and the Hackensack Meridian School of Medicine.

STEMKAM is showing kids that anyone can be a scientist and that scientists do many different things! We are seeking STEM professionals to participate in 3-5 minute interviews on their career in STEM. These videos will help add important, real-world context to STEMKAMP, a 5-day hands-on STEM summer camp for 1500 military connected kids in 10 states across the nation. We are specifically seeking professionals in environmental science, electrical and mechanical engineering, pharmaceutical formulation, bioinformatics, biosecurity and analytical chemistry but ALL STEM fields are welcome. Please contact avogel@ibio.org for more information. 

Independent Colleges and Universities for New Jersey, an organization that empowers deserving students to realize their goals through student scholarships and other programs, is seeking researchers, human resource specialists and STEM career professionals from among New Jersey's leading industries to serve as judges for its annual Undergraduate Research Symposium. Virtual research presentations will be held over three days (April 12-14) via Zoom. Judges will interact directly with students during 25-minute poster presentations of semester-long research projects. Your participation is needed to provide students with valuable insights on their projects and presentation skills. Judges will have the opportunity to view the student's poster and ask questions during the presentation. Abstracts will be provided in advance. Please volunteer to be a judge by contacting Yvette Panella at ypanella@njcolleges.org

Funding News

The New Jersey Economic Development Authority (NJEDA) announced that it sent final approval letters to the 49 life sciences and technology companies that are participating in the 2020 Net Operating Loss (NOL) ProgramHailed as a lifeline for New Jersey companies that have yet to reach profitability, the NOL Program enables participants to sell their New Jersey net operating losses and unused research and development (R&D) tax credits to unrelated profitable corporations for cash. The cash can then be used for working capital or to fund research. The NJEDA and the New Jersey Department of Treasury's Division of Taxation jointly administer the program.

The New Jersey Economic Development Authority (NJEDA) Board of Directors approved the creation of the Micro Lender Support Grant Program. This $2 million pilot program will make grant funding available to organizations that lend to New Jersey small and micro businesses. These funds will help eligible entities by supporting the costs associated with scaling up their operations and offering direct assistance to New Jersey-based businesses. This is critical now as the COVID-19 pandemic has created overwhelming need among micro businesses.

Thirteen months ago, Golden Seeds, a nationwide angel investor network dedicated to investing in women-led startups, launched a New Jersey chapter in partnership with First Lady Tammy Snyder Murphy and the New Jersey Economic Development Authority (NJEDA) to increase available capital for female-led businesses. Since then, Golden Seeds has connected more than 80 female-led companies with seasoned angel investors through a series of monthly office hours and follow-up conversations. In celebration of Women's History Month, the NJEDA takes a closer look at the chapter's important role in New Jersey's innovation economy and the resources available to female entrepreneurs.

Special Programs from Our Partners for BioNJ Members

March 22, 2021

Join the Governor for his next virtual town hall on Monday, March 22nd. The Governor will be presenting his priorities for the upcoming budget year as we continue our work to contain the pandemic together. As always, the Governor will be answering questions that can be submitted when you register for the town hall. Click here to register; registration is required to join.

April 6, 2021

Join leading experts from Rutgers and Tel Aviv Universities in the upcoming research symposium examining advances in gene therapy. Creating research opportunities today that will change the world tomorrow.

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.