July 2020

Source: www.uab.edu

Source: www.marketwatch.com

Source: www.richmond.com

APEIRON to utilize MaxCyte’s ExPERT® platform for siRNA-based cell therapy APN401 targeting solid tumors and secures manufacturing for upcoming clinical trials

VIENNA, AUSTRIA and GAITHERSBURG, MD, July 8, 2020 APEIRON Biologics AG (“APEIRON”), a private biotechnology company specializing in the discovery, development and commercialization of novel immunotherapies for cancer and respiratory diseases, and MaxCyte, Inc., a global cell-based therapies and life sciences company, today announces the signing of a clinical and commercial licensing agreement.

APEIRON Biologics will obtain non-exclusive clinical and commercial rights to use MaxCyte’s Flow Electroporation® technology and ExPERT™ platform for the advancement of APN401, a siRNA-based cell therapy currently in clinical development for various solid tumors. In return, MaxCyte will receive undisclosed development and approval milestones and sales-based payments in addition to other licensing fees.

“Securing access to MaxCyte’s ExPERT platform and unique electroporation technology is a crucial next step in the clinical advancement of our lead checkpoint inhibition Cbl-b candidate APN401,” said Peter Llewellyn-Davies, CEO of APEIRON Biologics.

Doug Doerfler, President & CEO of MaxCyte, said: “We are proud to support APEIRON in the development of a siRNA-based treatment that could help patients facing cancers with various forms of tumors. This agreement represents an important achievement for MaxCyte, and highlights the value of our next-generation technology platform to companies across the globe seeking to unlock the potential of their engineered cell therapy programs.”

MaxCyte’s ExPERT instrument portfolio represents the next generation of leading, clinically validated, electroporation technology for complex and scalable cell engineering. By delivering high transfection efficiency, seamless scalability and enhanced functionality, the ExPERT platform delivers the high-end performance essential to enable the next wave of biological and cellular therapeutics.

About APEIRON Biologics AG

APEIRON Biologics AG is a European private biotechnology company based in Vienna that specializes in the discovery, development and commercialization of novel immunotherapies for cancer and respiratory diseases. APEIRON’s approved cancer drug Qarziba® is being marketed by EUSA Pharma Ltd.. With APN01, APEIRON is conducting a clinical trial in Europe for the treatment of COVID-19, for which market approval is being sought. APEIRON’s APN401 clinical program is a “first-in-class” autologous cell therapy for strengthening immunoreactivity via the intra-cellular master checkpoint Cbl-b. APEIRON’s products and technologies are based on a strong patent portfolio and partnerships with leading pharmaceutical companies and academic institutions. For more information, visit www.apeiron-biologics.com

About MaxCyte

MaxCyte, the clinical-stage global cell-based therapies and life sciences company, uses its proprietary next-generation cell and gene therapies to revolutionise medical treatments and ultimately save lives. The Company’s premier cell engineering enabling technology is currently being deployed by leading drug developers worldwide, including all of the top ten global biopharmaceutical companies. MaxCyte licenses have been granted to more than 100 cell therapy programs, with more than 70 licensed for clinical use, and the Company has now entered into eleven clinical/commercial license partnerships with leading cell therapy and gene editing developers. MaxCyte was founded in 1998 and is headquartered in Gaithersburg, Maryland, US. For more information, visit www.maxcyte.com

APEIRON Contacts:

APEIRON Biologics AG
Peter Llewellyn-Davies, CEO
Email: investors@apeiron-biologics.com

Media and Investor Relations                           +49 89 210 228 0

MC Services AG
Julia Hofmann
Email: apeiron@mc-services.eu
 

MaxCyte Contacts:

US Investor Relations

Michael Levitan
Solebury Trout
+1 646-378-2920
mlevitan@soleburytrout.com

US Media Relations

Jamie Lacey-Moreira
PressComm PR, LLC
+1 410-299-3310
jamielacey@presscommpr.com

• Mount Sinai and Emergent to conduct clinical trials to evaluate COVID-HIG for post-exposure prophylaxis of COVID-19 in front-line health care workers and to support a potential Expanded Access Program for military personnel with funding from the U.S. Department of Defense
• ImmunoTek to extend operating license and provide training to Mount Sinai to establish onsite plasma collection to support production of COVID-HIG

NEW YORK and GAITHERSBURG, Md. and NEW ORLEANS and LAFAYETTE, La., July 08, 2020 (GLOBE NEWSWIRE) — The Mount Sinai Health System, Emergent BioSolutions (NYSE: EBS), and ImmunoTek Bio Centers today announced that they will collaborate to develop, manufacture, and conduct clinical trials to evaluate Emergent’s COVID-19 hyperimmune globulin product, COVID-HIG, including a post-exposure prophylaxis (PEP) study on health care providers at high risk of COVID-19 infection and other high-risk populations, with $34.6 million in funding from the U.S. Department of Defense’s (DOD) Joint Program Executive Office for Chemical, Biological, Radiological, and Nuclear Defense (JPEO-CBRND).

Located in New York City, one of the early epicenters of the outbreak, Mount Sinai has experience treating more than 10,000 COVID-19 cases and was among the very first in the United States to initiate a convalescent plasma program in late March. A leader in COVID-19 research and clinical care, Mount Sinai developed a serological assay to detect SARS-CoV-2 antibodies, one of the first to receive emergency use authorization from the U.S. Food and Drug Administration (FDA).

“There is emerging evidence that convalescent plasma is an effective treatment for COVID-19 patients,” said David L. Reich, MD, President and Chief Operating Officer of The Mount Sinai Hospital. “Therefore, hyperimmune globulin may become an effective option in the prevention and treatment of COVID-19 currently, in the absence of a vaccine, as well as in the future, particularly for patients who do not develop immunity from a vaccine. It is imperative that we have more options to prevent this terrible disease in front-line workers and other high-risk populations and to potentially decrease the severity of illness in those infected. We are eager to collaborate with Emergent and ImmunoTek to advance the science and identify effective therapeutics in the fight against COVID-19.”

The collaborators will establish plasma collection capabilities at Mount Sinai through an extension of ImmunoTek’s FDA-approved establishment license and the transfer of technical know-how to Mount Sinai. Plasma from recovered donors will support the development and manufacture of COVID-HIG for evaluation of the product candidate in clinical trials, and for potential emergency use or broader patient use as allowed by the FDA.

“Our collaboration with Mount Sinai, ImmunoTek, and the Department of Defense enhances the response to COVID-19 and broadens our efforts to have a meaningful impact,” said Dr. Laura Saward, SVP and Therapeutics Business Unit Head at Emergent BioSolutions. “The front-line health care workers and others who protect us are a top priority for reducing the impact of COVID-19. Emergent is drawing from decades of experience with our human hyperimmune platform, on which several products have been FDA-licensed, to develop COVID-HIG. Our mission – to protect and enhance life – is at the forefront of everything we do.”

Evaluating COVID-HIG in Clinical Trials
Mount Sinai and Emergent will evaluate COVID-HIG in a post-exposure prophylaxis study in individuals at high risk of exposure to COVID-19, such as front-line health care workers and military personnel.

The clinical research program is designed to assess whether prophylaxis with COVID-HIG could help protect individuals at high risk of exposure and limit the spread of disease. Under the agreement with the JPEO-CBRND, Emergent will collaborate to collect convalescent plasma to manufacture COVID-HIG for use in a clinical study under a potential Expanded Access Program to support military personnel.

Establishing Sustainable Plasma Collection Capabilities
ImmunoTek will provide technical, scientific, and industry expertise in plasma collection and will extend its FDA license as an approved source plasma collection establishment to enable plasma collection onsite at Mount Sinai. ImmunoTek will also provide staff training and compliance information to assist in establishing standard operating procedures and plasma criteria. Mount Sinai intends to collect convalescent plasma from its broad pool of eligible donors to support Emergent’s manufacture of COVID-HIG and the evaluation of the product candidate in clinical trials, and for potential emergency use or broader patient use as allowed by the FDA.

“This unprecedented public health crisis is a critical moment for Americans to donate plasma,” said Jerome Parnell III, CEO and President, ImmunoTek Bio Centers. “Specifically, blood plasma donors from New York impacted by the pandemic could unlock the potential of a viable hyperimmune globulin product to protect our health care providers, military, and first responders, and to treat patients with severe complications from COVID-19. Our unique collaboration honors our hero donors while highlighting the importance of expanding plasma collection capabilities across mainstream communities and diverse municipalities in the fight against COVID-19 and other rare diseases.”

Emergent will support ImmunoTek and Mount Sinai in other regulatory and compliance efforts related to plasma collection and supply, as well as activities leading up to an Investigational New Drug application submission to the FDA.

Advancing COVID-HIG With U.S. Government Support
In addition to receiving DOD funding to advance a post-exposure prophylaxis indication for COVID-HIG, Emergent was awarded $14.5 million in April by the U.S. Department of Health and Human Services (HHS) to develop COVID-HIG as a potential treatment for COVID-19. The product candidate will be evaluated in clinical studies by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH), as a potential treatment in hospitalized patients and patients at high risk of progression to severe disease. Emergent will produce investigational COVID-HIG material from the plasma collected at Mount Sinai to support the post-exposure prophylaxis and treatment clinical trials, for which Mount Sinai will serve as a study site.

“COVID-19 outbreaks in the military cause a significant risk to readiness and the ability to conduct training and perform our mission. Military training is often conducted in close contact as a unit or team, which makes social distancing nearly impossible. Our goal is to deliver medical solutions to enable military readiness. Knowing that HIG has been used in other disease outbreaks successfully as a prophylaxis, we are excited to partner with Emergent to develop this potential solution for the military and the nation,” said Army Col. Ryan Eckmeier, the JPEO-CBRND’s Joint Project Manager for Chemical, Biological, Radiological, and Nuclear Medical (JPM CBRN Medical).

About Hyperimmune Globulin
Hyperimmune globulin, sometimes referred to as polyclonal antibodies, is a concentrated antibody product derived from the antibody-rich plasma of people who were previously infected with and recovered from an illness; in this case, COVID-19 caused by the virus SARS-CoV-2. In order to produce plasma-derived therapeutics that can be administered to patients in need, plasma must be collected from a pool of human donors and then manufactured, or fractioned, into specialized therapeutic products. Hyperimmune globulin treatments have been used successfully to treat other viruses.

About the Mount Sinai Health System
The Mount Sinai Health System is New York City’s largest academic medical system, encompassing eight hospitals, a leading medical school, and a vast network of ambulatory practices throughout the greater New York region. Mount Sinai is a national and international source of unrivaled education, translational research and discovery, and collaborative clinical leadership ensuring that we deliver the highest quality care—from prevention to treatment of the most serious and complex human diseases. The Health System includes more than 7,200 physicians and features a robust and continually expanding network of multispecialty services, including more than 400 ambulatory practice locations throughout the five boroughs of New York City, Westchester, and Long Island. The Mount Sinai Hospital is ranked No. 14 on U.S. News & World Report’s “Honor Roll” of the Top 20 Best Hospitals in the country and the Icahn School of Medicine as one of the Top 20 Best Medical Schools in country. Mount Sinai Health System hospitals are consistently ranked regionally by specialty and our physicians in the top 1% of all physicians nationally by U.S. News & World Report.

For more information, visit https://www.mountsinai.org or find Mount Sinai on Facebook, Twitter and YouTube.

About Emergent BioSolutions
Emergent BioSolutions is a global life sciences company whose mission is to protect and enhance life. Through our specialty products and contract development and manufacturing services, we are dedicated to providing solutions that address public health threats. Through social responsibility, we aim to build healthier and safer communities. We aspire to deliver peace of mind to our patients and customers so they can focus on what’s most important in their lives. In working together, we envision protecting or enhancing 1 billion lives by 2030. For more information visit www.emergentbiosolutions.com. Find us on LinkedIn and follow us on Twitter @emergentbiosolu and Instagram @life_at_emergent.

About ImmunoTek Bio Centers LLC
ImmunoTek Bio Centers is an emerging bio-tech company committed to the safe collection and procurement of human blood plasma from the public. The management team has extensive experience in the blood, plasma, and biopharma industries. Through contracts and strategic agreements with pharmaceutical companies, IMMUNOTEK is fully capable of constructing, opening, FDA/EU licensing, and managing multiple plasma collection sites and plasma supply contracts in order to meet on going demand in the plasma proteins therapeutics market. Growth of current therapeutic drugs and vaccines as well as additional new therapeutic indications expected to put considerable strain on global blood plasma supply. IMMUNOTEK currently owns and operates plasma collection centers in the USA and has over 40 collection centers in development through 2020. For more information, please visit www.immunotek.com.

About the JPEO-CBRND
The Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) protects the Joint Force by providing medical countermeasures and defense equipment against chemical, biological, radiological and nuclear threats. As an effective DoD acquisition program, the JPEO-CBRND’s vision is a resilient Joint Force enabled to fight and win unencumbered by a CBRN environment; championed by innovative, agile, results-oriented acquisition professionals. Its Joint Project Manager for Chemical, Biological, Radiological, and Nuclear Medical (JPM CBRN Medical) facilitates the advanced development and acquisition of medical solutions to combat CBRN and emerging threats. JPM CBRN Medical works with JPEO-CBRND’s Joint Project Lead for Chemical, Biological, Radiological and Nuclear Defense – Enabling Biotechnologies to provide new and improved medical countermeasures to enable a single treatment for many threats, rapid medical countermeasure responses, genomic sequencing and the capability to diagnose CBRN threats before the onset of symptoms. To learn more about JPEO-CBRND’s COVID-19 response, visit https://www.jpeocbrnd.osd.mil/coronavirus.

Emergent BioSolutions Safe Harbor Statement
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, including statements regarding our ability to collect convalescent plasma, develop COVID-HIG for prophylaxis or treatment, obtain FDA approval or authorization for emergency or broader patient use are forward-looking statements. These forward-looking statements are based on our current intentions, beliefs and expectations regarding future events. We cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from our expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, we do not undertake to update any forward-looking statement to reflect new information, events or circumstances.

There are a number of important factors that could cause the company’s actual results to differ materially from those indicated by such forward-looking statements, including the success of the collaboration and planned development programs; the timing of and our ability to obtain and maintain regulatory authorizations or approvals; and our commercialization, marketing and manufacturing capabilities. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our periodic reports filed with the SEC, when evaluating our forward-looking statements.

Contacts:

Mount Sinai Health System
Elizabeth Dowling
Director, Media Relations
347-541-0212
elizabeth.dowling@mountsinai.org

Emergent BioSolutions
Media:
Miko B. Neri
Senior Director, Corporate Communications
240-631-3392
nerim@ebsi.com

Investor:
Robert G. Burrows
Vice President, Investor Relations
240-631-3280
burrowsr@ebsi.com

ImmunoTek Bio Centers
Pam Farris
Executive Administrative Assistant
337-500-1294
pfarris@immunotek.com

his content is provided by Clark A. Kendall of Kendall Capital in Rockville, MD.

While COVID-19 has thrown the world into an uproar, there’s been a slim silver lining for Maryland. Thanks to a robust group of biohealth and life science organizations, Montgomery County has a unique opportunity to help people nationwide — and worldwide — in the fight against the disease.

As a resident, you may not realize just how much Montgomery County does to help in biohealth across the country.

To start, there is the National Institutes of Health, which hardly needs an introduction, what with its many contributions to medicine throughout the world for more than a century. Just in April, the NIH launched the Accelerating COVID-19 Therapeutic Interventions and Vaccines partnership to coordinate — and hopefully accelerate — research on a vaccine.

Montgomery County is also just down the street from Johns Hopkins, which has been the backbone of COVID-19 data tracking since January.

Among the biggest and most hopeful recent news surrounds Novavax, a vaccine development company headquartered in Gaithersburg, Maryland, with additional facilities in Rockville, Maryland and Uppsala, Sweden. The Maryland-based company, which has never brought a product to market before, just made the biggest deal to date with the Trump administration’s Operation Warp Speed. The federal government will pay the vaccine maker Novavax $1.6 billion to expedite the development of 100 million doses of a coronavirus vaccine by the beginning of next year.

Additionally, many businesses along the I-270 biotech corridor are working on testing and vaccine development. Here are just a few that the Montgomery County Economic Development Corporation has highlighted:

• AdvaGenix is producing self-administered oral swab tests with a 48-hour turnaround, which is a game-changer for essential frontline workers in hospitals and nursing homes around the country.
• Sanaria is partnering with the University of Maryland and Germany’s University of Tübingen to work on antibody testing.
• GlaxoSmithKline and its research and development vaccine center has “offered to share its vaccine adjuvant with others developing vaccines,” according to Rockville Economic Development, Inc.

There are many other organizations doing the critical work of research and development in the fight against COVID-19 which bring profound economic opportunity to the area.

Read more via WTOP

ROCKVILLE, Md., July 8, 2020 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV^® Technology Platform, today announced that it has completed dosing of three patients in Cohort 2 of the Company’s Phase I/II study of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and reported encouraging data under a single-patient investigator-initiated Investigational New Drug (IND) application for RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) conducted at CHOC Children’s.

“We are pleased to have completed the dosing of three patients with MPS II at the second dose level of our RGX-121 Phase I/II study, which included involvement from new leading centers at UCSF Benioff Children’s Hospital Oakland and Hospital de Clínicas de Porto Alegre in Brazil. We expect to provide an additional interim data update from the RGX-121 program later this year,” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. “Further, we are encouraged by the initial data from the first patient dosed with RGX-111, and we look forward to advancing our RGX-111 Phase I/II study.”

Enrollment in Cohort 2 of the Phase I/II study of RGX-121 is now complete, with three patients with MPS II dosed intracisternally with 6.5×10¹⁰ genome copies per gram (GC/g) of brain mass. As of June 24, 2020, RGX-121 is reported to be well-tolerated in patients across two dose levels, with no drug-related serious adverse events (SAEs). Additional data from both cohorts in this study and a program update will be available in the second half of 2020.

Under a single-patient investigator-initiated IND for RGX-111, Raymond Wang, M.D., a biochemical genetics specialist at CHOC Children’s, dosed a patient with severe MPS I intracisternally with 1×10¹⁰ GC/g of brain mass at the age of 21 months. MPS I is caused by a deficiency of the enzyme α-l-iduronidase (IDUA) and subsequent accumulation of heparan sulfate (HS) within the central nervous system (CNS). High levels of HS in the CSF closely correlate with neurocognitive decline and are a key biomarker of enzyme activity. As of June 9, 2020, RGX-111 is reported to be well-tolerated in this patient, with no drug-related SAEs.

The IDUA enzyme activity in the patient’s cerebrospinal fluid (CSF) was below the limit of quantification prior to the administration of RGX-111. An increase in IDUA enzyme activity was detected at Week 12, the latest timepoint available following administration of RGX-111. High levels of HS in the CSF were measured in this patient at baseline, and initial data demonstrated sustained decreases in total HS in the CSF over time, with a 50% reduction from baseline at Week 12 and a 45% reduction from baseline at Week 33, the latest timepoint available.

Neurocognitive development in untreated MPS I patients is characterized by a plateau followed by a significant decline between 1 and 3 years of age. At Week 32 post-administration of RGX-111, neurocognitive evaluations indicated that the patient, who was then 29 months of age, continued to acquire cognitive developmental skills at a normal rate.

Dr. Wang commented, “I thank the research team at CHOC Children’s and the child’s family for working together to make this study possible. I am extremely encouraged by the clinical and biochemical indicators eight months post-treatment in this first person with MPS I dosed with RGX-111. The promising signals of IDUA expression and reduced HS levels in CSF, and an indication of ongoing neurocognitive development, provide additional evidence of the potential of RGX-111 for individuals who have MPS I.”

Recruitment, screening and additional site activations are ongoing in a Phase I/II clinical trial of RGX–111. REGENXBIO expects to provide a program update in the second half of 2020. Additional information about REGENXBIO’s Phase I study of RGX-111 may be found at ClinicalTrials.gov, using Identifier NCT: NCT03580083.

About RGX-111

RGX-111 is a product candidate for the treatment of Mucopolysaccharidosis Type I (MPS I), also known as Hurler syndrome. RGX-111 is designed to use the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene. Delivery of the enzyme that is deficient within cells in the central nervous system (CNS) could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration.

About the Phase I/II Clinical Trial of RGX-111

RGX-111 is being evaluated in a Phase I/II, first-in-human, multi-center, open-label, dose escalation study in patients with Mucopolysaccharidosis Type I (MPS I). The Phase I/II trial is designed to evaluate the safety of RGX-111 and secondary endpoints include the effect of RGX–111 on biomarkers of α-l-iduronidase (IDUA) enzyme activity in the CSF, serum and urine, neurocognitive development and other outcome measures.

About Mucopolysaccharidosis Type I (MPS I)

MPS I is a rare autosomal recessive genetic disease caused by deficiency of IDUA, an enzyme required for the breakdown of the polysaccharides in lysosomes. These polysaccharides, called glycosaminoglycans (GAGs), accumulate in tissues of MPS I patients, resulting in characteristic storage lesions and diverse clinical signs and symptoms including in the central nervous system (CNS), which can include excessive accumulation of fluid in the brain, spinal cord compression, and cognitive impairment. MPS I is estimated to occur in 1 in 100,000 births. Current disease modifying therapies for MPS I include hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy with a recombinant form of human IDUA administered intravenously. However, intravenous enzyme therapy does not treat the CNS manifestations of MPS I, and HSCT can be associated with clinically significant morbidity and mortality.

About RGX-121

RGX-121 is a product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase (IDS) gene which encodes the iduronate-2-sulfatase (I2S) enzyme to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration.

About the Phase I/II Clinical Trial of RGX-121

RGX–121 is being evaluated in a Phase I/II, multi-center, open-label, multiple-cohort, dose–escalation study in patients with Mucopolysaccharidosis Type II (MPS II) in the United States and Brazil. The Phase I/II trial is designed to evaluate the safety of RGX-121 in up to 6 patients less than five years of age who have or are at high risk of developing neurocognitive effects. In addition, the study will evaluate the effect of RGX-121 on biomarkers of iduronate-2-sulfatase (I2S) enzyme activity as well as neurocognitive deficits and other clinical measures.

About Mucopolysaccharidosis Type II (MPS II)

MPS II is a rare, X-linked recessive disease caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (I2S) leading to an accumulation of glycosaminoglycans, including heparan sulfate (HS) in tissues which ultimately results in cell, tissue, and organ dysfunction. In severe forms of the disease, early developmental milestones may be met, but developmental delay is readily apparent by 18 to 24 months. Specific treatment to address the neurological manifestations of MPS II and prevent or stabilize cognitive decline remains a significant unmet medical need. Key biomarkers of I2S enzymatic activity in MPS II patients include its substrate heparan sulfate (HS), which has been shown to correlate with neurocognitive manifestations of the disorder.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Forward-Looking Statements

This press release includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would” or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO’s future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO’s expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO’s business, and other factors, many of which are beyond the control of REGENXBIO. Refer to the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2019, and comparable “risk factors” sections of REGENXBIO’s Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC’s website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com

Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com

Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com

SOURCE REGENXBIO Inc.

Related Links

http://www.regenxbio.com

Source: www.prnewswire.com

• Collaboration combines innovative plant-based and adjuvant technologies to develop and produce a COVID-19 candidate vaccine.

• Phase 1 clinical testing scheduled to begin mid-July

• Collaboration to explore vaccine development opportunities for other infectious diseases

GSK and Medicago today announced a collaboration to develop and evaluate a COVID-19 candidate vaccine combining Medicago’s recombinant Coronavirus Virus-Like Particles (CoVLP) with GSK’s pandemic adjuvant system. CoVLPs mimic the structure of the virus responsible for COVID-19 disease, allowing them to be recognised by the immune system. Use of an adjuvant can be of particular importance in a pandemic situation as it may boost the immune response and reduce the amount of antigen required per dose, allowing more vaccine doses to be produced and therefore contributing to protect more people.

Pre-clinical results with Medicago’s CoVLP vaccine candidate demonstrated a high level of neutralizing antibodies following a single dose when administered with adjuvant.

Phase 1 clinical testing is planned to start in mid-July and will evaluate the safety and immunogenicity of three different dose levels of antigen combined with GSKs pandemic adjuvant and in parallel with an adjuvant from another company, administered on a one- and two-dose vaccination schedule, given 21 days apart.

Subject to successful clinical development and regulatory considerations, the companies aim to complete development and make the vaccine available, in the first half of 2021. Both companies will also evaluate expanding their collaboration to develop a post-pandemic vaccine COVID-19 candidate, should the need arise based on the further development of COVID-19 after the pandemic, and other infectious diseases.

The companies will use Medicago’s plant-based production platform to manufacture the COVID-19 vaccine antigen. This innovative technology uses the leaves of a plant as bioreactors to produce the S-spike protein which self-assemble into VLPs for use in the CoVLP vaccine candidate. It is highly scalable and can support the production of large amounts of vaccine in a significantly shortened timeline. Using this technology combined with GSKs proprietary adjuvant system, the companies expect to be able to manufacture approximately 100m doses by the end of 2021. By the end of 2023, a large-scale facility under construction in Quebec City, Canada, is expected to deliver up to 1 billion doses annually. The manufacturing platform has been used to produce a seasonal VLP flu vaccine and the license application is under review with the Canadian regulatory authority.

Dr Thomas Breuer, Chief Medical Officer, GSK Vaccines, said: “This agreement paves the way for an innovative vaccine option combining a scalable plant-based antigen technology with an adjuvant which has pandemic dose sparing capability. If successful, it will be a meaningful contributor in the fight against COVID-19. We strongly believe that multiple vaccines are needed, including post-pandemic vaccines. This plant-based technology also shows promise beyond COVID-19 and has the potential to help prevent other infectious diseases.”

Dr Bruce Clark, President and CEO of Medicago, said: “We are about to begin clinical trials with our CoVLP vaccine candidate harnessing GSKs pandemic adjuvant technology against the virus that causes COVID-19. This collaboration with GSK gives us access to a proven adjuvant which could enhance the effectiveness of our candidate vaccine, and also to a depth of scientific experience to support our development efforts.”

GSK and Medicago have entered into a binding agreement to develop and manufacture an adjuvanted COVID-19 vaccine. Medicago is a privately held company jointly owned by Mitsubishi Tanabe Pharma (MTPC) and Philip Morris International (PMI), with a shareholding ratio of 67:33, respectively. PMI has signalled that it is willing to evaluate offers for its shareholding from parties that may be better suited to help Medicago on the next phase of its journey and has initiated preliminary review to determine the optimal shareholding and governance structure for Medicago’s future success.

About Medicago

Medicago is a biopharmaceutical company headquartered in Quebec City, Canada. Medicago’s mission is to improve global health outcomes by leveraging innovative plant-based technologies for rapid responses to emerging global health challenges. Medicago is committed to advancing therapeutics against life-threatening diseases worldwide. For more information please visit www.medicago.com

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.

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 under Item 3.D “Risk Factors” in the company’s Annual Report on Form 20-F for 2019 and any impacts of the COVID-19 pandemic.

• Emergent will provide contract development and manufacturing services beginning in 2021 to produce drug substance at large scale for commercial manufacturing with first two years valued at approximately $480 million
• For the remaining three years beginning in 2023, Emergent will provide a flexible capacity deployment model to support annual dose requirements

^® technology. Emergent will provide contract development and manufacturing (CDMO) services to produce drug substance at large scale over five years, valued at approximately $480 million for the first two years.

“We are proud to deploy our manufacturing strength to address the COVID-19 pandemic,” said Robert G. Kramer Sr., president and chief executive officer of Emergent BioSolutions. “Advancing this collaboration is one of the ways we live our mission – to protect and enhance life.”

Under the agreement, Emergent will begin providing large-scale drug substance manufacturing for Johnson & Johnson’s adenovirus-based COVID-19 vaccine in 2021, upon successful completion of the activities under the previously executed Technology Transfer Agreement. For the subsequent years beginning 2023, Emergent will provide a flexible capacity deployment model to support additional drug substance batches annually.

“Over the next five years, we are committing our leading CDMO services to advance this important vaccine candidate,” said Syed T. Husain, senior vice president and CDMO business unit head at Emergent. “We have the expertise and capabilities to meet the long-term needs of our customers and provide ongoing commercial manufacturing to benefit patients.”

This long-term large-scale manufacturing agreement follows and is incremental to the contract announced in April for drug substance manufacturing technology transfer services and for reserving certain large-scale manufacturing capacity to pave the way for commercial drug substance manufacturing for the COVID-19 vaccine candidate.

Activities will be performed at Emergent’s Baltimore Bayview facility, a designated Center for Innovation in Advanced Development and Manufacturing (CIADM) by the U.S. Department of Health and Human Services (HHS), designed for rapid manufacturing of large quantities of vaccines and treatments during public health emergencies.

Emergent’s Bayview facility has unique capabilities across four independent suites to produce at clinical scale to get candidates rapidly into the clinic, while at the same time scaling up to enable large-scale manufacturing to up to 4000L to prepare for production of commercial volumes to meet customer demand. The CIADM has the capacity to produce tens to hundreds of millions of doses of vaccine on an annual basis, based upon the platform technology being used.

Financial Considerations
The company will provide an update to its 2020 financial outlook incorporating expectations related to this agreement and any other relevant information when it reports its second quarter financial results.

About Emergent BioSolutions
Emergent BioSolutions is a global life sciences company whose mission is to protect and enhance life. Through our specialty products and contract development and manufacturing services, we are dedicated to providing solutions that address public health threats. Through social responsibility, we aim to build healthier and safer communities. We aspire to deliver peace of mind to our patients and customers so they can focus on what’s most important in their lives. In working together, we envision protecting or enhancing 1 billion lives by 2030. For more information visit www.emergentbiosolutions.com. Find us on LinkedIn and follow us on Twitter @emergentbiosolu and Instagram @life_at_emergent.

Emergent’s Response to COVID-19
Emergent BioSolutions is deploying its decades of experience in vaccine and hyperimmune development and manufacturing, as well as its molecule-to-market contract development and manufacturing (CDMO) services to provide comprehensive medical countermeasure solutions in response to the COVID-19 pandemic.

Using its established hyperimmune platforms, Emergent is developing two investigational plasma-based treatments – COVID-Human Immune Globulin (COVID-HIG) and COVID-Equine Immune Globulin (COVID-EIG). COVID-HIG is being developed as a human plasma-derived therapy candidate with $14.5 million in HHS funding and will be evaluated in two studies of the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, for potential treatment of COVID-19 in severe hospitalized and high-risk patients. COVID-EIG is being developed as an equine plasma-derived therapy candidate for potential treatment of severe disease in humans. Both candidates are anticipated to be in Phase 2 clinical studies in 2020. These investigational products are not approved by the U.S. Food and Drug Administration and their safety and effectiveness have not been established.

Emergent is deploying its CDMO capabilities, capacities, and expertise to support the U.S. government’s Operation Warp Speed to pave the way for innovators to advance COVID-19 programs. The company is working with four innovators to develop and manufacture COVID-19 vaccine candidates. For the COVID-19 vaccine response, Emergent’s integrated CDMO network provides development services from its Gaithersburg facility, drug substance manufacturing at its Baltimore Bayview facility, and drug product manufacturing at its Baltimore Camden and Rockville facilities, all in Maryland.

For 22 years Emergent has focused on advancing public health, and its multi-pronged approach to tackling COVID-19 demonstrates its commitment to its mission – to protect and enhance life.

Safe Harbor Statement
This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, including statements regarding our ability to produce viable COVID-19 vaccine candidates at the prescribed scale, meet annual dosage requirements in the anticipated timeline and pave the potential pathway to licensure and commercial manufacturing of these candidates, are forward-looking statements. These forward-looking statements are based on our current intentions, beliefs and expectations regarding future events. We cannot guarantee that any forward-looking statement will be accurate. Investors should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from our expectations. Investors are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, we do not undertake to update any forward-looking statement to reflect new information, events or circumstances.

There are a number of important factors that could cause the company’s actual results to differ materially from those indicated by such forward-looking statements, including the success of the planned development programs; the timing of and ability to obtain and maintain regulatory approvals for the product candidates; and our commercialization, marketing and manufacturing capabilities. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our periodic reports filed with the SEC, when evaluating our forward-looking statements.

Media Contact:
Miko B. Neri
Senior Director, Corporate Communications
240-631-3392
NeriM@ebsi.com

Investor Contact:
Robert G. Burrows
Vice President, Investor Relations
240-631-3280
BurrowsR@ebsi.com