Arcellx Closes $115 Million Series C Financing to Advance its Pipeline of Adaptive and Controllable Cell Therapies

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GAITHERSBURG, Md., April 13, 2021 (GLOBE NEWSWIRE) – – Arcellx, a privately held clinical-stage biopharmaceutical company, today announced that it raised $115 million in a Series C financing to advance its pipeline of adaptive and controllable cell therapies. The proceeds will support the company’s development of CART-ddBCMA, a BCMA-specific CAR-modified T-cell therapy currently in Phase 1 and anticipated to begin a pivotal trial in 2022. In addition, the funding will support initiation of clinical trials evaluating ACLX-001 and ACLX-002, cell therapies derived from Arcellx’s uniquely controllable ARC-SparX platform, in multiple myeloma (MM) and acute myelogenous leukemia (AML), respectively.

This financing follows FDA clearance of Arcellx’s IND for ACLX-001, the first ARC-SparX program to enter clinical trials, and Arcellx’s initial release of clinical results at the 2020 American Society of Hematology (ASH) meeting. In the ASH release, the CART-ddBCMA data showed all six multiple myeloma patients responded per IMWG criteria, with four of those patients achieving stringent complete response. The therapy was also well-tolerated, and CAR-T related toxicities resolved rapidly.

Participants in the Series C financing include both existing and new investors to Arcellx. The financing was co-led by Samsara BioCapital and CAM Capital, joined by new investors Adage, Asymmetry, CaaSCapital, Cambrian Bio, Sixty Degree, Soleus Capital, Surveyor Capital (a Citadel company), Suvretta, and Terra Magnum Capital Partners, and existing investors NEA, Novo Holdings, SR One, Takeda Ventures, LG Tech, and Clough Capital.

“With support from this high caliber syndicate, Arcellx is poised to elevate the field of cell therapy by advancing our treatments for a range of cancers,” said Rami Elghandour, Chairman and Chief Executive Officer of Arcellx. “Our platform of both single infusion and controllable CAR-Ts based on our novel synthetic binding domain is built to address the limitations of cell therapy with the opportunity to improve efficacy, reduce toxicity, and shorten the time to intervention while expanding into new indications. This financing positions us to advance to a registrational study in multiple myeloma and to initiate a Phase 1 study in AML in 2022 as well as progress our solid tumor targets toward the clinic. It’s alsoa reflection of our incredibly talented and diverse team that is powering Arcellx forward. We appreciate the support of our new and existing investors as we advance our novel therapies to the benefit of cancer patients most in need.”

“Based on the early clinical data, we believe that CART-ddBCMA represents a potential best-in class therapy for multiple myeloma and with the support of this financing will be positioned to move into pivotal trials next year. We’re also excited about the opportunity for CART-ddBCMA to move into earlier lines of treatment for multiple myeloma based on the safety profile in this early data set. In addition, the ARC-SparX platform will be the first adaptive and controllable CAR-T system to enter the clinic and provides a unique approach to building next generation cell

therapies. We look forward to partnering with the Arcellx team to help bring these important therapies to patients,” said Mike Dybbs, Ph.D., Partner, Samsara BioCapital.

About Arcellx, Inc.

Arcellx is a clinical-stage biopharmaceutical company developing adaptive and controllable cell therapies for the treatment of patients with cancer and autoimmune diseases. The Arcellx vision is to utilize our novel proprietary platform to bring superior cell therapies to more patients through the care of academic and community practices worldwide. More information can be found at www.arcellx.com.

Media:

Zara Lockshin Solebury Trout 646-378-2960

zlockshin@soleburytrout.com

Investors:

Alan Lada Solebury Trout 646-378-2927

alada@soleburytrout.com

John Graziano Solebury Trout 646-378-2942

jgraziano@soleburytrout.com

The Pentagon projects exploring ways to end pandemics

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The military wants to stop pandemics. Many of the innovations and breakthroughs deployed to combat COVID-19 began years ago as Pentagon-funded research programs to protect soldiers, some spearheaded by the Defense Advanced Research Projects Agency. Bill Whitaker and 60 Minutes cameras were allowed in to report on some of the technologies the Department of Defense is developing to defend against future pandemics — including a single vaccine to ward off all coronaviruses. Whitaker’s report will be broadcast on 60 Minutes, Sunday, April 11 at 7 p.m. ET/PT on CBS.

 

https://www-cbsnews-com.cdn.ampproject.org/c/s/www.cbsnews.com/amp/news/last-pandemic-science-military-60-minutes-2021-04-09/

Novavax Initiates COVID-19 Vaccine Clinical Trial Crossover

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GAITHERSBURG, Md., April 5, 2021 /PRNewswire/ — Novavax, Inc. (Nasdaq: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, today announced the initiation of crossover arms in two ongoing clinical trials of NVX-CoV2373, the company’s COVID-19 vaccine candidate. Crossover ensures the administration of active vaccine to all participants in the trials and has begun for Novavax’ Phase 2b trial in South Africa and its pivotal Phase 3 trial in the United Kingdom.

Under Novavax’ updated clinical trial protocols[1], all participants in the UK and US Phase 3 trials will be offered the opportunity to receive an additional round of injections. Participants who elect to do so will receive an additional two-dose regimen of either vaccine (for those who originally received placebo) or placebo (for those who originally received vaccine). Participants in the South Africa Phase 2b trial will receive either active vaccine for those who initially received placebo, or a booster dose of active vaccine for those who initially received active vaccine. Participants across all three trials will remain blinded to their courses of treatment to preserve the ability to assess efficacy in each trial, and all will be followed for up to two years to monitor the safety and durability of protection the vaccine. In the trials taking place in South Africa and the United Kingdom, half of the participants initially received the active vaccine while two-thirds of participants in PREVENT-19, the trial being conducted in the US and Mexico, initially received active vaccine.

“The crossover arms ensure that all participants have access to an active vaccine candidate while allowing Novavax to continue to monitor the safety and efficacy of our vaccine over the long term,” said Filip Dubovsky, M.D., Chief Medical Officer, Novavax. “We are grateful to the volunteers who stepped forward to take part in our clinical trials, without whom we would be unable to develop, study and ultimately deliver what we hope will be a significant tool in the fight against COVID-19.”

The company is also planning a crossover in the PREVENT-19 study, for which the company expects to read out initial clinical data during the second quarter. In addition, the company is planning to expand the trial to include pediatric and adolescent arms, which are also expected to begin in the second quarter.

About NVX-CoV2373
NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax’ recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is adjuvanted with Novavax’ patented saponin-based Matrix-M™ to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that blocked the binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response in Phase 1/2 clinical testing.

NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials, a trial in the U.K that demonstrated efficacy of 96.4% against the original virus strain and 89.7% overall, and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. It is also being tested in two ongoing Phase 2 studies that began in August 2020: A Phase 2b trial in South Africa that demonstrated 48.6% efficacy against a newly emerging escape variant, and a Phase 1/2 continuation in the U.S. and Australia.

NVX-CoV2373 is stored and stable at 2°- 8°C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.

About Matrix-M™
Novavax’ patented saponin-based Matrix-M™ adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About Novavax
Novavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company’s proprietary recombinant technology platform combines the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. Novavax is conducting late-stage clinical trials for NVX-CoV2373, its vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. NanoFlu™, its quadrivalent influenza nanoparticle vaccine, met all primary objectives in its pivotal Phase 3 clinical trial in older adults and will be advanced for regulatory submission. Both vaccine candidates incorporate Novavax’ proprietary saponin-based Matrix-M™ adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visit www.novavax.com and connect with us on Twitter and LinkedIn.

Novavax Forward Looking Statements
Statements herein relating to the future of Novavax and the ongoing development of its vaccine and adjuvant products are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include those identified under the heading “Risk Factors” in the Novavax Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release s0peak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

____________________
1 Clinical trial protocols may be found in the Resources section of the Novavax website and will be updated as appropriate.

Contacts:
Investors
Novavax, Inc.
Erika Schultz | 240-268-2022
ir@novavax.com

Solebury Trout
Jennifer Porcelli | 617-974-8659
jporcelli@soleburytrout.com

Media
Laura Keenan | 410-419-5755
Amy Speak | 617-420-2461
media@novavax.com

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BioIT Solutions Develops Mobile Application for COVID-19 Testing for Cambridge Consortium

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April 5, 2021 – Silver Spring, MD. BioIT Solutions announced that it has developed and launched a mobile application to support the COVID-19 testing efforts of the Cambridge Consortium for Rapid COVID-19 Tests (CCRCT), a partnership of LabCentralBioInnovation Labs (BioLabs), and E25Bio.

“BioIT Solutions is pleased to have been selected by the CCRCT to develop the application to support data collection in an IRB-approved human research study to compare an at-home COVID-19 antigen test against nasal samples processed by qRT-PCR in a laboratory,” said BioIT Solutions’ President, Mike Fannon. The longitudinal study was rolled out to determine if antigen testing at home allows employees to safely report for in-person work. Users are able to scan a QR code label on a test kit, enter relevant health information, and submit a camera image of the test strip from their homes. The antigen test was developed by E25Bio as a rapid home test for COVID-19, with easily readable results in 15 minutes. Test results are confirmed by a laboratory-based PCR test, facilitated by a laboratory management system also built by BioIT Solutions.

Celina Chang, LabCentral’s Vice President of Science Operations and Strategic Relationships, notes that “BioIT Solutions has been a valuable partner through the planning, implementation and deployment of a critical customized LIM system. They adapt quickly to changing needs and deliver high-quality systems in a remarkably short time. Users of BioIT’s mobile application consistently report that it is reliable, easy to use, and convenient, resulting in a positive user experience and reliable data to help us reduce disease transmission and provide a safe work environment.”

About BioIT Solutions
BioIT Solutions delivers highly effective data management and workflow systems ready-made for today’s fast-paced life science laboratories. We apply our decades of experience in the biopharmaceutical industry with our flexible 1PLATFORM4® Software Suite to engineer rapidly deployed and customized solutions in a cost-effective manner. With BioIT Solutions, your scientific data are safely managed by a logical, consistent and responsive system.

Our customers benefit from tailored and adaptable solutions while avoiding rigidity, expense and operational inefficiencies. We have the tools and expertise to translate your procedures into practical and adaptable workflow applications with modern and easy-to-use interfaces.

Supernus Announces FDA Approval of Qelbree™ (SPN-812) for the Treatment of ADHD

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ROCKVILLE, Md., April 02, 2021 (GLOBE NEWSWIRE) — Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today announced that the U.S. Food and Drug Administration (FDA) approved Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in pediatric patients 6 to 17 years of age.

“Based on the efficacy demonstrated in the clinical program, we believe Qelbree offers a unique new alternative for the treatment of ADHD,” said Jack A. Khattar, President and Chief Executive Officer of Supernus Pharmaceuticals. “Qelbree provides prescribing physicians and patients living with ADHD a therapy that is not a controlled substance with proven efficacy and a tolerable safety profile. We are grateful to the patients, families and their care givers who participated in and supported our research.”

“ADHD is one of the most common mental health issues in the U.S.,” said Andrew J. Cutler, M.D., Clinical Associate Professor of Psychiatry at SUNY Upstate Medical University, and Chief Medical Officer, Neuroscience Education Institute. “The right treatment is key for children and adolescents, as they grow and navigate school and social relationships. This approval offers a novel once a day sprinkleable non-stimulant that can be a great option for children and adolescents with ADHD.”

The approval of Qelbree is supported by data from an extensive development program consisting of four Phase III clinical trials that studied more than 1000 pediatric patients from the age of 6 to 17 years. In December 2020, the Company announced positive results from a Phase III trial in adult patients with ADHD and plans to submit a supplemental New Drug Application to the FDA for Qelbree in adults in the second half of 2021.

IMPORTANT SAFETY INFORMATION

Qelbree may increase suicidal thoughts and actions in some children with ADHD, especially within the first few months of treatment or when the dose is changed. Pay close attention to any new or sudden changes in mood, behavior, thoughts, and feelings. Call your child’s doctor right away if there are any new or sudden changes, or if there is development of suicidal thoughts or actions.  Qelbree should not be taken by patients that also take certain anti-depression medicines, especially those called a monoamine oxidase inhibitor or MAOI, or certain asthma medicines.

Please see full Prescribing Information, including Boxed Warning, for Qelbree.

About Supernus Pharmaceuticals, Inc. 

Supernus Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases.

Our diverse neuroscience portfolio includes approved treatments for epilepsy, migraine, attention-deficit hyperactivity disorder (ADHD), hypomobility in Parkinson’s disease, cervical dystonia and chronic sialorrhea. We are developing a broad range of novel CNS product candidates including new potential treatments for hypomobility in Parkinson’s disease, epilepsy, depression, and rare CNS disorders.

For more information, please visit www.supernus.com.

Forward-Looking Statements:

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements do not convey historical information but relate to predicted or potential future events that are based upon management’s current expectations. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. In addition to the factors mentioned in this press release, such risks and uncertainties include, but are not limited to, the Company’s ability to sustain and increase its profitability; the Company’s ability to raise sufficient capital to fully implement its corporate strategy; the implementation of the Company’s corporate strategy; the Company’s future financial performance and projected expenditures; the Company’s ability to increase the number of prescriptions written for each of its products; the Company’s ability to increase its net revenue; the Company’s ability to commercialize its products including Qelbree; the Company’s ability to enter into future collaborations with pharmaceutical companies and academic institutions or to obtain funding from government agencies; the Company’s product research and development activities, including the timing and progress of the Company’s clinical trials, and projected expenditures; the Company’s ability to receive, and the timing of any receipt of, regulatory approvals to develop and commercialize the Company’s product candidates; the Company’s ability to protect its intellectual property and operate its business without infringing upon the intellectual property rights of others; the Company’s expectations regarding federal, state and foreign regulatory requirements; the therapeutic benefits, effectiveness and safety of the Company’s product candidates; the accuracy of the Company’s estimates of the size and characteristics of the markets that may be addressed by its product candidates; the Company’s ability to increase its manufacturing capabilities for its products and product candidates; the Company’s projected markets and growth in markets; the Company’s product formulations and patient needs and potential funding sources; the Company’s staffing needs; and other risk factors set forth from time to time in the Company’s filings with the Securities and Exchange Commission made pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. The Company undertakes no obligation to update the information in this press release to reflect events or circumstances after the date hereof or to reflect the occurrence of anticipated or unanticipated events.

CONTACT:

Jack A. Khattar, President and CEO
Jim Kelly, Executive Vice President and CFO
Supernus Pharmaceuticals, Inc.
Tel: (301) 838-2591

Or

Investor Contact:
Peter Vozzo
Westwicke/ICR
Office: (443) 213-0505
Mobile: (443) 377-4767
Email: peter.vozzo@westwicke.com

United Therapeutics Announces FDA Approval and Launch of Tyvaso® for the Treatment of Pulmonary Hypertension Associated with Interstitial Lung Disease

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First and only approved therapy in the United States for patients with PH-ILD, a serious, life-threatening disease with potentially more than 30,000 patients in need

FDA approval based on data from the INCREASE clinical trial

PH-ILD is the second FDA-approved indication for Tyvaso, which was initially approved for the treatment of pulmonary arterial hypertension


SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C., April 1, 2021 /PRNewswire/ — United Therapeutics Corporation (Nasdaq: UTHR) today announced that the U.S. Food and Drug Administration (FDA) has approved Tyvaso® (treprostinil) Inhalation Solution for the treatment of patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability. This is the second FDA-approved indication for Tyvaso, which was first approved in July 2009 for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise ability.

“Adults living with both interstitial lung disease and pulmonary hypertension typically have a poor quality of life because of increased shortness of breath, poor exercise tolerance, and increased mortality. Until now, clinicians treating these patients did not have any approved treatment options,” said Aaron Waxman, M.D., Ph.D., Director of the Pulmonary Vascular Disease Program at Brigham and Women’s Hospital and the chair of the INCREASE Study Steering Committee. “The regulatory approval of Tyvaso, an inhaled treatment, is exciting news both for patients with PH-ILD and the physicians who treat adults living with this serious, life-threatening disease. This will change the way we manage these patients.”

Interstitial lung disease (ILD) is a group of lung diseases in which marked scarring occurs within the lungs. It is often complicated by pulmonary hypertension (PH; high blood pressure in the lungs), which further symptoms and decreases survival. PH is estimated to affect at least 15% of patients with early-stage ILD (approximately 30,000 PH-ILD patients in the United States) and may affect up to 86% of patients with more severe ILD.

“The FDA approval of Tyvaso for patients with PH-ILD is a landmark treatment advancement for this vulnerable patient population,” said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics. “It also underscores our commitment to driving innovation in the field of pulmonary hypertension and expanding the number of patients who can achieve a clinical benefit from Tyvaso. We plan to tap into our experience and expanded infrastructure to bring this safe and effective inhaled therapy to the many patients living with PH-ILD in the United States.”

“With this approval representing such a breakthrough for PH-ILD patients, we’re treating this indication launch with a sense of urgency,” said Michael Benkowitz, President and Chief Operating Officer of United Therapeutics. “We’ve already expanded our field-based teams by 40% to educate the ILD community on the benefits of Tyvaso and how to properly diagnose PH-ILD. We expect rapid uptake of Tyvaso in this indication and expect to double the number of patients on Tyvaso therapy by the end of 2022, barring any COVID-related delays.”

The FDA approval of the supplemental New Drug Application (sNDA) for Tyvaso for PH-ILD is supported by data from INCREASE, the largest and most comprehensive completed study of adult patients with PH-ILD. The multicenter, randomized, double-blind, placebo-controlled, 16-week, parallel-group study of 326 patients met its primary endpoint, demonstrating a significant improvement in six-minute walk distance (6MWD). Results, published in the New England Journal of Medicine, and discussed at a recent United Therapeutics investor meeting, also showed benefits across several key subgroups, including etiology of PH-ILD, disease severity, age, gender, baseline hemodynamics, and dose. Significant improvements were also observed in each of the secondary endpoints, including reduction in the cardiac biomarker NT-proBNP, time to first clinical worsening event, change in peak 6MWD at week 12, and change in trough 6MWD at week 15. Additional observations included placebo-corrected improvements in forced vital capacity (FVC) and significantly fewer exacerbations of underlying lung disease in patients receiving Tyvaso. Treatment with Tyvaso of up to 12 breaths per session, four times daily, in the INCREASE study was well tolerated and the safety profile was consistent with previous Tyvaso studies and known prostacyclin-related adverse events (see the Important Safety Information below under “About TYVASO® (treprostinil) Inhalation Solution”).

About PH-ILD
Interstitial lung disease (ILD) is a group of lung diseases that are characterized by marked scarring or fibrosis of the bronchioles and alveolar sacs within the lungs. Increased fibrotic tissue in ILD prevents oxygenation and free gas exchange between the pulmonary capillaries and alveolar sacs, and the condition can present with a wide range of symptoms, including shortness of breath with activity, labored breathing, and fatigue.

WHO Group 3 Pulmonary hypertension (PH) frequently complicates the course of patients with interstitial lung disease and is associated with worse functional status measured by exercise capacity, greater supplemental oxygen needs, decreased quality of life, and worse outcomes.  PH is estimated to affect at least 15% of patients with early-stage ILD (approximately 30,000 PH-ILD patients in the United States) and may affect up to 86% of patients with more severe ILD.

About TYVASO® (treprostinil) Inhalation Solution

INDICATION
TYVASO (treprostinil) is a prostacyclin mimetic indicated for the treatment of:

  • Pulmonary arterial hypertension (PAH; WHO Group 1) to improve exercise ability. Studies establishing effectiveness predominately included patients with NYHA Functional Class III symptoms and etiologies of idiopathic or heritable PAH (56%) or PAH associated with connective tissue diseases (33%).

The effects diminish over the minimum recommended dosing interval of 4 hours; treatment timing can be adjusted for planned activities.

While there are long-term data on use of treprostinil by other routes of administration, nearly all controlled clinical experience with inhaled treprostinil has been on a background of bosentan (an endothelin receptor antagonist) or sildenafil (a phosphodiesterase type 5 inhibitor). The controlled clinical experience was limited to 12 weeks in duration.

  • Pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve exercise ability. The study establishing effectiveness predominately included patients with etiologies of idiopathic interstitial pneumonia (IIP) (45%) inclusive of idiopathic pulmonary fibrosis (IPF), combined pulmonary fibrosis and emphysema (CPFE) (25%), and WHO Group 3 connective tissue disease (22%).

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

  • TYVASO is a pulmonary and systemic vasodilator. In patients with low systemic arterial pressure, TYVASO may produce symptomatic hypotension.
  • TYVASO inhibits platelet aggregation and increases the risk of bleeding.
  • Co-administration of a cytochrome P450 (CYP) 2C8 enzyme inhibitor (e.g., gemfibrozil) may increase exposure (both Cmax and AUC) to treprostinil. Co-administration of a CYP2C8 enzyme inducer (e.g., rifampin) may decrease exposure to treprostinil. Increased exposure is likely to increase adverse events associated with treprostinil administration, whereas decreased exposure is likely to reduce clinical effectiveness.

DRUG INTERACTIONS/SPECIFIC POPULATIONS

  • The concomitant use of TYVASO with diuretics, antihypertensives, or other vasodilators may increase the risk of symptomatic hypotension.
  • Human pharmacokinetic studies with an oral formulation of treprostinil (treprostinil diolamine) indicated that co-administration of the cytochrome P450 (CYP) 2C8 enzyme inhibitor, gemfibrozil, increases exposure (both Cmax and AUC) to treprostinil. Co-administration of the CYP2C8 enzyme inducer, rifampin, decreases exposure to treprostinil. It is unclear if the safety and efficacy of treprostinil by the inhalation route are altered by inhibitors or inducers of CYP2C8.
  • Limited case reports of treprostinil use in pregnant women are insufficient to inform a drug-associated risk of adverse developmental outcomes. However, pulmonary arterial hypertension is associated with an increased risk of maternal and fetal mortality. There are no data on the presence of treprostinil in human milk, the effects on the breastfed infant, or the effects on milk production.
  • Safety and effectiveness in pediatric patients have not been established.
  • Across clinical studies used to establish the effectiveness of TYVASO in patients with PAH and PH–ILD, 268 (47.8%) patients aged 65 years and over were enrolled. The treatment effects and safety profile observed in geriatric patients were similar to younger patients. In general, dose selection for an elderly patient should be cautious, reflecting the greater frequency of hepatic, renal, or cardiac dysfunction, and of concomitant diseases or other drug therapy.

ADVERSE REACTIONS

  • Pulmonary Arterial Hypertension (WHO Group 1)
    In a 12-week, placebo-controlled study (TRIUMPH I) of 235 patients with PAH (WHO Group 1 and nearly all NYHA Functional Class III), the most common adverse reactions seen with TYVASO in ≥4% of PAH patients and more than 3% greater than placebo in the placebo-controlled study were cough (54% vs 29%), headache (41% vs 23%), throat irritation/pharyngolaryngeal pain (25% vs 14%), nausea (19% vs 11%), flushing (15% vs <1%), and syncope (6% vs <1%). In addition, adverse reactions occurring in ≥4% of patients were dizziness and diarrhea.
  • Pulmonary Hypertension Associated with ILD (WHO Group 3)
    In a 16-week, placebo-controlled study (INCREASE) of 326 patients with PH-ILD (WHO Group 3), adverse reactions were similar to the experience in studies of PAH.

Please see Full Prescribing Information, the TD-100 and TD-300 TYVASO® Inhalation System Instructions for Use manuals, and other additional information at www.tyvaso.com or call 1–877–UNITHER (1-877-864-8437).

United Therapeutics: Enabling Inspiration
United Therapeutics Corporation focuses on the strength of a balanced, value-creating biotechnology model. We are confident in our future thanks to our fundamental attributes, namely our obsession with quality and innovation, the power of our brands, our entrepreneurial culture, and our bioinformatics leadership. We also believe that our determination to be responsible citizens – having a positive impact on patients, the environment, and society – will sustain our success in the long term.

Through our wholly owned subsidiary, Lung Biotechnology PBC, we are focused on addressing the acute national shortage of transplantable lungs and other organs with a variety of technologies that either delay the need for such organs or expand the supply. Lung Biotechnology is the first public benefit corporation subsidiary of a public biotechnology or pharmaceutical company.

Please visit unither.com to learn more.

Forward-looking Statements
Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, statements relating to the potential for the approval of Tyvaso in PH-ILD to change the way PH-ILD patients are treated, our expectation of a rapid uptake of Tyvaso in PH-ILD patients, and our expectation that we will double the number of patients on Tyvaso therapy by the end of 2022, our ability to create value and sustain our success in the long-term, as well as our efforts to develop technologies that either delay the need for transplantable organs or expand the supply of transplantable organs. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of April 1, 2021, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason.

TYVASO is a registered trademark of United Therapeutics Corporation.

For Further Information Contact:
Dewey Steadman at (202) 919-4097
Email: ir@unither.com  

SOURCE United Therapeutics Corporation

Related Links

http://www.unither.com

Maryland Biotech IES Life Sciences Developing Test to Detect How Severe a COVID Case Could Become

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IES Life Sciences is currently applying for an Emergency Use Authorization for the test from the FDA.

MARYLAND, USA — A Maryland biotech company is hoping to help doctors save lives by developing a new test that would indicate how severe a COVID case could become.

IES Life Science has partnered with the University of Maryland to develop the patented test, which will be administered via nasal swabs, like the majority of the standard COVID tests, once a patient has tested positive.

When the pandemic hit in February, IES Life Sciences pivoted the cancer detection technology it had been working on to measure the immune response to COVID-19 in patients. With that knowledge, they said physicians should be able to better focus their resources on the patients who need them most.

Director of Research and Development, Dr. Robert Figliozzi, said so far the team has tested a few hundred samples to measure the success of their technology. They’re working to procure thousands more.

“We’re looking at individuals’ … total immune health, and how they’re responding to this infection,” Dr. Figliozzi said. “And we’re grouping them into little groups. And different groups have different severity levels.”

Click here to read more via WUSA9

BioBuzz: BioHealth Capital Region Passes San Francisco in NIH funding

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The National Institutes of Health provides significant funding for drug development research to universities, companies, and organizations. Over the course of 2020, the NIH provided a significant amount of funding to the BioHealth Capital Region.

The top recipients of NIH funding in the BHCR were research universities, with Johns Hopkins University topping the list with $807,432,003 in financing for different research programs. The top 10 recipients of NIH funding in the BioHealth Capital Region secured more than $2.18 billion in financing. Life sciences organizations within the state of Maryland secured $2.3 billion in NIH funding, an increase from the $1.9 billion granted in 2019.

Since the year 2000 when life sciences companies within Maryland received $863 million, the state has seen significant growth in NIH funding, with Johns Hopkins alone surpassing that this year. In fact, the BHCR surpassed California’s Bay Area in NIH funding. Organizations across that important biotech region garnered $1.83 billion in NIH grant monies.

Click here to read more via BioBuzz.

BioBuzz: The BioHealth Capital Region, Epicenter of U.S. Regulatory Infrastructure

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As one of the world’s top bio hubs, the BioHealth Capital Region (BHCR) is rife with life science assets.

The region has been at the forefront of the SARS-CoV-2 vaccine race, and it is rapidly becoming a center for advanced manufacturing. The BHCR cell and gene therapy ecosystem is thriving, and the area boasts one of the richest and most diverse tapestries of life science talent and workforce development programming. What’s more, Maryland, Washington, DC, and Virginia are home to several dynamic university systems that keep the talent and tech transfer pipeline humming and full.

That said, one major BHCR asset tends to fly under the radar, yet just might be the source from which all else flows—the region’s expansive regulatory ecosystem.

The BHCR is home to a wide array of government and private sector regulatory organizations, including but not limited to the National Institutes of Health (NIH), the National Institute of Standards and Technology (NIST), the U.S. Food and Drug Administration (FDA), U.S. Health and Human Services’ Biomedical Advanced Research and Development Authority (BARDA), and private-sector regulatory stalwarts like United States Pharmacopeia (USP).

These critical regulatory bodies, and the non-dilutive funding some can provide to promising companies, have acted as a magnet, drawing (and keeping) scores of entrepreneurs, international companies, established biopharma organizations, and emerging life science entities to the region.

Indeed, COVID-19 has temporarily disrupted the value of proximity, as companies in all sectors were forced to transition to nearly 100% remote work environments. However, as more vaccinations get into arms and the pandemic recedes, the intrinsic value of proximity and access will recover. That’s not to say the 9 to 5 in the office, road warriors of the I-270 corridor will return full force; remote work is here to stay. But the importance of face-to-face interactions and connecting in person at lunch or via conferences will eventually return. That is to say that the BHCR’s robust regulatory ecosystem will continue to be a significant biopharma industry asset even in the COVID-19 new normal.

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GSK to support manufacture of Novavax’ COVID-19 vaccine

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LONDON and GAITHERSBURG, Md., March 29, 2021 /PRNewswire/ — Novavax, Inc. (Nasdaq: NVAX), a biotechnology company developing next-generation vaccines for serious infectious diseases, GSK and the UK Government Vaccines Taskforce announced that they have reached an agreement in principle to support manufacturing of up to 60 million doses of Novavax’ COVID-19 vaccine candidate (NVX-CoV2373) for use in the UK. GSK will provide ‘fill and finish’ manufacturing capacity at its Barnard Castle facility in the North East of England beginning as early as May 2021, with a rapid technology transfer between the two companies beginning immediately. GSK and Novavax will negotiate a final agreement to include additional terms and conditions.

The UK Government has secured 60 million doses of the vaccine under an advance purchase agreement with Novavax. The protein antigen component of NVX-CoV2373 is also produced in the North East of England by Novavax’ manufacturing partner, FUJIFILM Diosynth Biotechnologies, at their site in Billingham, Stockton-on-Tees.

Fill and finish, to be provided by GSK, is the completion stage of vaccine manufacturing, preparing vials of the final vaccine and packaging them for distribution and use. The GSK site at Barnard Castle, which will deliver the vaccine doses under this collaboration, is a specialised facility in GSK’s global manufacturing network, which supports production of GSK pharmaceutical and vaccine products.

Roger Connor, President, GSK vaccines, said: “GSK is delighted to support Novavax and the UK Vaccines Taskforce with this manufacturing arrangement for the UK and our Barnard Castle facility is now undertaking the rapid preparation work required to manufacture up to 60m doses of this vaccine. We have ensured that we can deliver these volumes without impacting supply of our other vital medicines and vaccines, and without disruption to the other COVID-19 collaborations GSK is engaged in globally.”

“This partnership with GSK continues the expansion of our global supply network, which we expect to increase overall production capacity and, if approved by regulatory agencies, support access to a potentially important new vaccine against COVID-19,” said Rick Crowley, Executive Vice President and Chief Operations Officer, Novavax. “We thank the UK government’s Vaccine Taskforce for its instrumental role in ensuring the progress of our COVID-19 vaccine, from both a clinical and now manufacturing perspective, as well as GSK for making their facilities available to help fight the pandemic.”

Prime Minister Boris Johnson said: “I’m delighted by GSK’s investment, which shows the strength of UK manufacturing, and will further boost our vaccine rollout. The Vaccines Taskforce has worked hand in glove with business to successfully deliver vaccines to the whole of the UK, and this agreement will continue to support our approach. We remain on track to offer a first jab to all over 50s by 15 April, and all adults by the end of July, and I want to once again encourage everyone to come forward for a vaccine when you’re called.”

Health and Social Care Secretary Matt Hancock said: “We’ve all seen just how important onshore vaccine manufacturing capabilities are, and this fantastic deal will ensure more of these vital products can be produced here in the UK. The UK’s vaccination programme has been a national success, with over 30 million people now having received a first dose of a COVID-19 vaccine. Should the Novavax vaccine meet our medicines regulator’s high standards of safety and effectiveness, the agreement reached today will boost these efforts over the coming months.”

The Novavax vaccine candidate has demonstrated strong potential efficacy in Phase 3 clinical trials, including against the B.1.1.7 variant circulating in the UK. Submission of the vaccine for review by regulatory authorities in the UK is expected during the second quarter.

GSK commitment to tackling COVID-19
GSK’s response to COVID-19 has been one of the broadest in the industry, with two potential treatments in addition to our vaccine candidates in development.

GSK is collaborating with several organisations around the world on COVID-19 vaccines by providing access to our adjuvant technology. Our collaboration with Canada’s Medicago, combining our pandemic adjuvant with its plant-derived vaccine candidate is in Phase 3 clinical trials, and a collaboration with French company Sanofi on an adjuvanted, protein-based vaccine candidate is in Phase 2. An earlier stage collaboration with SK Bioscience of South Korea, with funding from CEPI and the Bill and Melinda Gates Foundation, aims to develop differentiated, affordable COVID-19 vaccines for supply globally through the COVAX facility. The use of an adjuvant can be of particular importance in a pandemic since it may reduce the amount of vaccine protein required per dose, allowing more vaccine doses to be produced and contributing to protecting more people.

GSK is working with German mRNA specialist, CureVac, to jointly develop next generation, multi-valent mRNA vaccines for COVID-19 with the potential to address multiple emerging variants in one vaccine. GSK will also support manufacturing of up to 100m doses of CureVac’s first generation COVID-19 vaccine at our facilities in Belgium.

GSK is also exploring potential therapeutic or treatment options for COVID-19 patients. We are collaborating with Vir Biotechnology to develop existing and identify new anti-viral antibodies that could be used as therapeutic or preventive options for COVID-19. We recently reported that an Independent Data Monitoring Committee recommended that the Phase 3 COMET-ICE trial evaluating VIR-7831 as monotherapy for the early treatment of COVID-19 in adults at high risk of hospitalisation be stopped for enrolment due to evidence of profound efficacy, based on an interim analysis of data from the trial. We are now seeking Emergency Use Authorization in the US and authorisations in other countries. We are also assessing whether an investigational monoclonal antibody, otilimab, can help severely ill COVID-19 patients aged over 70 who experience an overreaction of their immune system.

About GSK
GSK is a science-led global healthcare company with a special purpose: to help people do more, feel better, live longer. For further information please visit www.gsk.com/about-us.

About NVX-CoV2373
NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease. NVX-CoV2373 was created using Novavax’ recombinant nanoparticle technology to generate antigen derived from the coronavirus spike (S) protein and is adjuvanted with Novavax’ patented saponin-based Matrix-M™ to enhance the immune response and stimulate high levels of neutralizing antibodies. NVX-CoV2373 contains purified protein antigen and can neither replicate, nor can it cause COVID-19. In preclinical studies, NVX-CoV2373 induced antibodies that block binding of spike protein to cellular receptors and provided protection from infection and disease. It was generally well-tolerated and elicited robust antibody response numerically superior to that seen in human convalescent sera in Phase 1/2 clinical testing. NVX-CoV2373 is being evaluated in two pivotal Phase 3 trials, a trial in the U.K that demonstrated efficacy of 96.4% against the original virus strain and 89.7% overall, and the PREVENT-19 trial in the U.S. and Mexico that began in December 2020. It is also being tested in two ongoing Phase 2 studies that began in August: a Phase 2b trial in South Africa that demonstrated 48.65% efficacy against a newly emerging escape variant, and a Phase 1/2 continuation in the U.S. and Australia. NVX-CoV2373 is stored and stable at 2°- 8°C, allowing the use of existing vaccine supply chain channels for its distribution. It is packaged in a ready-to-use liquid formulation in 10-dose vials.

About Matrix-M™
Novavax’ patented saponin-based Matrix-M™ adjuvant has demonstrated a potent and well-tolerated effect by stimulating the entry of antigen presenting cells into the injection site and enhancing antigen presentation in local lymph nodes, boosting immune response.

About Novavax
Novavax, Inc. (Nasdaq: NVAX) is a biotechnology company that promotes improved health globally through the discovery, development and commercialization of innovative vaccines to prevent serious infectious diseases. The company’s proprietary recombinant technology platform combines the power and speed of genetic engineering to efficiently produce highly immunogenic nanoparticles designed to address urgent global health needs. Novavax is conducting late-stage clinical trials for NVX-CoV2373, its vaccine candidate against SARS-CoV-2, the virus that causes COVID-19. NanoFlu™, its quadrivalent influenza nanoparticle vaccine, met all primary objectives in its pivotal Phase 3 clinical trial in older adults and will be advanced for regulatory submission. Both vaccine candidates incorporate Novavax’ proprietary saponin-based Matrix-M™ adjuvant to enhance the immune response and stimulate high levels of neutralizing antibodies.

For more information, visit www.novavax.com and connect with us on Twitter and LinkedIn.

Novavax Forward Looking Statements
Statements herein relating to the future of Novavax and the ongoing development of its vaccine and adjuvant products are forward-looking statements. Novavax cautions that these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include those identified under the heading “Risk Factors” in the Novavax Annual Report on Form 10-K for the year ended December 31, 2020, as filed with the Securities and Exchange Commission (SEC). We caution investors not to place considerable reliance on forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC, available at sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press release speak only as of the date of this document, and we undertake no obligation to update or revise any of the statements. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

GSK Cautionary statement regarding forward-looking statements
GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described in the Company’s Annual Report on Form 20-F for 2020 and any impacts of the COVID-19 pandemic.

Novavax enquires:

Investor enquiries:
Novavax, Inc.
Erika Schultz | 240-268-2022
ir@novavax.com

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jporcelli@soleburytrout.com

Media enquiries:
Amy Speak | 617-420-2461
Laura Keenan | 410-419-5755
media@novavax.com

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