Montgomery Co. biotech company receives FDA approval for human trials of HIV Cure program

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A Montgomery County-based biotechnology company has announced it received approval from the FDA to begin human trials for its HIV cure program.

American Gene Technologies has been developing its program for 12 years. The company submitted their request to the FDA in October to begin human trials which are slated to start at the end of September or the beginning of October.

“This is momentous news that we have FDA approval to launch Phase 1 and conduct our first human trials. We are beyond excited to reach this milestone,” Chief Science Officer David Pauza said in a news release. “Based on our successful commercial-scale product manufacturing runs and features of the product observed in our labs, this therapy has a high potential to be effective.”

The company’s work builds off of a previous study by Sangamo Therapeutics which clipped part of the protein that plays a major part in HIV infection.

 

Click here to read more.

5 Questions with Judy Costello, Managing Director of Economic Development for BioHealth Innovation, Inc. (BHI)

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“5 Questions With……” is a weekly BioBuzz series where we reach out to interesting people in the BioHealth Capital Region to share a little about themselves, their work, and maybe something completely unrelated. This edition features 5 Questions with Judy Costello who is Managing Director of Economic Development for BioHealth Innovation, Inc. (BHI).

Judy Costello is Managing Director of Economic Development for BioHealth Innovation, Inc. (BHI). Prior to joining BHI, she served as Director of the Maryland Department of Commerce’s Office of BioHealth and Life Sciences and as Deputy Director of the department’s BioMaryland Center. In these positions, she has worked to grow the region’s biohealth cluster by supporting industry recruitment and retention, commercialization, workforce, non-dilutive and dilutive fundraising, international soft landing, partnership, and promotion activities. She previously served as Executive Director of the Business Alliance for Innovation and Entrepreneurship.

At the Business Alliance, she managed the organization’s Bootcamp, angel investor forum, tech transfer showcases, educational seminars, and other programs connecting entrepreneurs, faculty innovators, students, and industry leaders in Maryland, DC, and Virginia with each other and with those providing funding and other resources to young companies.

Before joining the Business Alliance, Costello held positions in economic development, financial services marketing, and university public relations. She is a graduate of Georgetown University and holds an MBA from Loyola University in Maryland.

1. What was your first job/role in the biotech/pharma/life-sciences industry? Tell us about it. (How you ended up there. About the role and company).

Though I interacted with a few early-stage biotech companies while running the Business Alliance Entrepreneur Bootcamp and Grubstake Angel Investor programs, I knew little about the unique life cycle of these companies and the industry until I joined the Maryland Biotechnology Center (later known as the BioMaryland Center) as its first Deputy Director in 2011. The Center was the part of the Maryland Department of Business and Economic Development tasked with guiding the implementation of the state’s “Bio2020 plan” involving a $1.3 billion to growing the biotechnology industry in Maryland.  While serving in that role, I had the opportunity to provide economic development (i.e. facilities, financial incentives, workforce, and marketing) support to a wide range of biotech companies.  We worked with industry, academic and government leaders to help build a sense of community, connect the state’s biotechnology assets and business growth resources with each other; and promote them in and outside Maryland.

The Department was rebranded to the Maryland Department of Commerce in 2015 and still works, with TEDCO and other agencies, to support the growth of the bio industry in the state. Maryland has so much to offer in terms of its biotechnology industry that it was a very exciting place to be.   Every day, I learned about new technologies and more about resources provided to innovators by the state in which I have lived since 1986.

2. Tell us about your current role and company/org? (highlight what the company is working on, maybe career opportunities there, location, history)

Since 2017, I have worked as the Managing Director for Economic Development at BioHealth Innovation.  The mission is similar to what I was doing at Maryland –supporting biotech industry growth through initiatives focused on expanding capital, talent, commercialization assistance, and other resources to companies; connecting innovators with resources, and promoting the industry cluster. However, BHI’s geographic footprint is broader—focused primarily on the BioHealth Capital Region (Maryland, DC, and Virginia)—and also assisting a few companies outside the area.

Our Entrepreneurs-in-Residence, analysts, and consultants provide direct commercialization support to a number of companies including several as part of an international soft-landing program.  This varies from non-dilutive funding application development assistance to market research, business strategy, portfolio vetting and packaging, and business development services. We still support queries about space, workforce, capital raising, economic development incentives, and other growth resources. What I most enjoy is continuing to learn while helping others advance their technologies, career paths, and businesses.

 Click here to read more via BioBuzz

FDA Approves American Gene Technologies (AGT) to Move Forward with Phase 1 Clinical Trial of HIV Cure Program

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ROCKVILLE, Md., Aug. 11, 2020 (GLOBE NEWSWIRE) — American Gene Technologies (AGT) announced today approval by the Food and Drug Administration (FDA) to begin Phase 1, the first human clinical trial for AGT’s lead HIV program. AGT will conduct its Phase 1 study at clinical sites in the Baltimore/D.C. area, and has named Washington Health Institute, University of Maryland, Institute of Human Virology and Georgetown University as its initial trial sites. These sites are expected to begin enrollment in September 2020.  AGT hopes to report initial data before the end of the year.

AGT’s Phase 1 trial will investigate the safety of AGT103-T, measure key biomarkers and explore surrogate markers of efficacy. AGT103-T is a single-dose, lentiviral vector-based gene therapy developed to eliminate HIV from the millions of people globally infected with the disease.

HIV has affected society for more than four decades. Globally, nearly 38 million people have been infected with HIV and over one million people in the United States are living with HIV. So far, efforts to cure the disease have been unsuccessful. AGT developed a new treatment to repair immune system damage done by HIV and allow natural responses to control the virus. From its research, AGT believes a cure is attainable and is now taking the significant step of testing in humans.

The AGT103-T cell product is made from blood cells using an 11-day process that increases the HIV-fighting T cells and uses a gene therapy to help these cells survive in the body. The product demonstrates the ability to clear itself of HIV when challenged with the virus and HIV-infected human cells.

AGT’s characterization of AGT103-T was done in collaboration with government researchers from the National Institute of Allergy and Infectious Disease (NIAID), part of the National Institutes of Health.  Under a Collaborative Research Agreement, NIAID provided detailed characterization of CD4 T cells in the company’s AGT103-T cell product and insight into mechanisms of action for this biological drug that may benefit people living with HIV.

A peer-reviewed article co-authored by AGT and NIAID scientists was published in Molecular Therapy in June 2020 (read published article). The publication describes the evolution of the AGT103-T manufacturing process as researchers developed the large-scale production of modified HIV-specific CD4 T cells that resist infection and depletion by HIV. The upcoming Phase 1 study will allow AGT to examine the effect of AGT103-T in a clinical setting which was previously demonstrated in preclinical human cell models.

The levels of potency in the AGT103-T cell product that have been repeated and validated by NIAID have given AGT further confidence that antiretroviral therapy-free HIV remission could be achieved soon. According to AGT founder and CEO Jeff Galvin, “I am confident AGT103-T will be an important step towards an eventual cure for HIV.”

“This is momentous news that we have FDA approval to launch Phase 1 and conduct our first human trials. We are beyond excited to reach this milestone. This brings us closer to our goal of transforming lives with genetic medicines,” said Chief Science Officer David Pauza, Ph.D. “Based on our successful commercial-scale product manufacturing runs and features of the product observed in our labs, this therapy has a high potential to be effective.”

Phase 1 in Maryland

Maryland ranked fifth of all U.S. states and territories in HIV diagnosis rates, with nearly 1,000 new cases in 2018 alone according to the Maryland State Department of Health. The Washington, D.C. metropolitan area, including Baltimore, is a hotspot for HIV/AIDS.  Our clinical trial sites in the metro area will test our new treatment in populations hardest hit by HIV.

FOR MEDIA: Click Here to Register for the Virtual Press Conference.

About HIV

Today, approximately 37.9 million people worldwide and 1.2 million people in the United States are living with HIV/AIDS. The U.S. government has estimated that 38,900 Americans were newly infected with HIV in 2018 and 1.7 million individuals globally were infected with HIV in 2018. Locally, the Washington D.C./Baltimore area is often cited as a ‘hot spot’ for HIV with Washington, D.C. having the highest rate of infection at nearly 48 infections per 100,000 population and Baltimore City with similarly high infection rates of 36 per 100,000.

Since the late 1980s, antiretroviral drugs have restored quality of life to persons living with HIV and, in some cases, have even been used to prevent new infections. However, no approved treatments can cure HIV. This is an unmet medical need that AGT is closer to addressing.

About AGT 103-T

AGT103-T is a genetically-modified cell product made from a person’s own cells. AGT’s approach is unique in that it focuses on repairing the key immune system damage caused by HIV. When HIV infection causes this specific damage, killing of T helper cells required for immunity to HIV, the infected person becomes unable to eliminate the virus and thus, becomes chronically infected. AGT’s approach is designed to repair the T helper cell defect and provide durable virus control that is not compromised by HIV strains that vary in sequence or use alternate ways to enter and infect T cells. AGT’s AGT103-T HIV therapeutic drug should work to remove infected cells from the body and decrease or eliminate the need for lifelong antiretroviral treatment.

About American Gene Technologies (AGT)

American Gene Technologies (AGT) is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT’s mission is to transform people’s lives through genetic medicines that rid the body of disease. AGT has received 3 patents for the technology used to make the AGT103-T cell product and ten patents for its unique immuno-oncology approach to stimulate gamma-delta (γδ) T cells to destroy a variety of solid tumors. The company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT’s treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2020.

FOR MEDIA: Register Now for AGT’s Virtual Press Conference
AGT will be hosting a virtual press conference to provide the media details regarding the AGT103-T autologous cell therapy, pre-clinical results, clinical sites, enrollment plan, and timelines. If you are a member of the media and would like to attend this 1-hour press conference, please register here: (click link)

American Gene Technologies Contacts:

C. Neil Lyons, Chief Financial Officer
Phone: (301) 337-2269
Email: info@americangene.com
www.americangene.com

Sasha Whitaker, Digital Marketing and Communications
Phone: (301) 337-2100
Email: swhitaker@americangene.com
www.americangene.com

Videos are available at the following links:

https://www.globenewswire.com/NewsRoom/AttachmentNg/87674dba-67e6-47ea-b0d7-c7382ed01d90

https://www.globenewswire.com/NewsRoom/AttachmentNg/3ac30894-796a-4444-87f7-533b1172bec6

https://www.globenewswire.com/NewsRoom/AttachmentNg/c2344580-d880-4cb3-a5c4-5c66c3267262

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USM Maryland Momentum Fund Invests $250,000 in $3.5M Seed Round of miRecule Inc.

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Baltimore, MD. (Aug. 10, 2020) — The University System of Maryland (USM) Momentum Fund has invested $250K in miRecule, an emerging biotech company with a cutting-edge discovery platform to create RNA therapeutics alongside theFSHD Society as a co-investor. This investment is complemented by recent funding raised over the past year, which includes lead investor Alexandria Venture Investments as well as Pathway Bioventures, Alumni Ventures Group, and angel investors. In total, miRecule’s seed round raised more than $3.5M in private seed funding. In addition to its private investments miRecule has also been awarded a non-dilutive $2M Phase II Small Business Innovation Research (SBIR) grant from the National Cancer Institute.

miRecule’s DREAmiRTM discovery platform was developed through more than a decade of research in collaboration with the National Institutes of Health in Bethesda, Md. DREAmiR analyzes genomic and outcome data from thousands of patients to identify the underlying genetic abnormalities causing a disease in an individual patient. The company then creates an RNA therapeutic to directly target and fix the abnormality. miRecule is applying this platform to a variety of diseases, developing lifesaving RNA therapeutics for cancer, muscular dystrophy, and even COVID-19.

“We are excited to add another therapeutic company to our investment portfolio. miRecule’s technology has the potential to be incredibly impactful for the health care industry and certainly for individual patients,” said Claire Broido Johnson, managing director of the Momentum Fund. “In addition to an innovative product, miRecule has a strong management team and we have a lot of confidence in their ability.”

miRecule was founded in Biohealth Innovation’s incubator in Montgomery County (Md.) by Chief Executive Officer (CEO) Anthony D. Saleh, PhD, and Chief Operating Officer Ashwin Kulkarni, MS, an alum of University of Maryland, College Park. The majority of miRecule’s seed round funding will be used to advance their lead program, MC-30, which addresses head and neck cancer, towards clinical development. Head and neck cancer is the sixth most common form of cancer worldwide, and for many patients, survival is measured in months not years.

“We are hopeful that MC-30 will be a real game changer,” said CEO Dr. Saleh. “By correcting the underlying mutations that make a patient resistant to treatment, we think we can triple response rates and give years of quality life back to many patients.”

About the USM Momentum Fund 
The Maryland Momentum Fund is an initiative of the University System of Maryland (USM) to provide late seed investment funding for promising technology ventures that come out of any of the 12 constituent USM institutions, its research parks, and its students, faculty, or graduates. With a $10M commitment from the USM already in place, the Fund co-invests with venture capitalists, foundations, and angel investors.

The Maryland Momentum Fund, which was established by the USM Board of Regents to support promising commercial opportunities arising from advances in research and intellectual property at USM campuses, has invested in 15 startups to date:  MF FireNextStep Roboticsthe North American Wave Engine CorporationZest TeaPaverGuideRetriumVeralox TherapeuticsGemstone BiotherapeuticsNeoprogenMinnowtechARMR SystemsInfercabuaryDatakwippathOtrakKaloCyte, and miRecule. The Fund is designed to create returns and support USM’s most innovative ideas as they enter the marketplace. Learn more at www.momentum.usmd.edu.

About miRecule

miRecule, Inc. is a pre-clinical biotechnology company focused on the development of RNA therapeutics to treat a variety of diseases. miRecule has developed DREAmiR, a genomics-based discovery platform that identifies critical RNA targets in specific subsets of patients that would benefit from treatment. The company then creates proprietary chemically modified RNA therapeutics with improved pharmacology and utilizes antibodies for targeted delivery to the diseased tissue. miRecule’s business model is to develop a pipeline of RNA therapeutics through human proof-of-concept and then out-license the candidate drug to larger biopharma companies. miRecule’s lead candidate, MC-30 for head and neck cancer, addresses a market of greater than $1B with high unmet need. This therapeutic replaces the potent tumor suppressor activity of microRNA-30, which is lost in half of head and neck cancer patients. MC-30 has over 50x improved half-life and 10x improved efficacy over the natural microRNA, and has demonstrated curative potential with low toxicity in animal models. miRecule’s second program, MC-DX4 for the treatment of Facioscapulohumeral muscular dystrophy (FSHD), addresses a market worth over $1B in the U.S. alone with over 40,000 patients and no approved treatment. MC-DX4 eliminates expression of the DUX4 gene, the cause of the disease in 95% of FSHD patients.

Contact-miRecule
Anthony Saleh
anthony@mirecule.com

Contact: Mike Lurie
Phone: 301.445.2719
Email: mlurie@usmd.edu

BioForward Wisconsin’s Lisa Johnson Virtually joins Rich Bendis on BioTalk

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The BioHealth Capital Region and BioForward Wisconsin come together as Lisa Johnson joins Rich Bendis on BioTalk to discuss her career, the industry, and growing biohealth in the Midwest.

Listen now via Google https://bit.ly/2DtF6Y1, Apple, https://apple.co/3fJU0q3, Spotify https://spoti.fi/3fFpb5H, TuneIn https://bit.ly/3iqNAh9, and YouTube Audio https://bit.ly/2PERPcH

Lisa Johnson is CEO of BioForward Wisconsin which serves as the collective voice of the state’s robust and comprehensive biohealth cluster. BioForward Wisconsin represents more than 220 member organizations and focuses on initiatives to strengthen the talent pipeline, enable collaborations and enhance professional development.

Appointed as CEO of BioForward Wisconsin in May 2015, Johnson brought entrepreneurial, international and government experience to the organization building its statewide focus on integrated health solutions. Her expertise in general management, operations, business development, and leading results-oriented teams powers a positive, action-oriented ecosystem that significantly contributes to the biohealth industry and the state’s economy.

Johnson started her bioscience career in 1989 by partnering to launch Novagen, a successful life science reagents company focused on protein expression and purification. As Finance and Business Development Manager responsible for administration, sales, and business development functions, she led teams in managing licensing and OEM agreements, distributor relations, and the integration process during the 1998 buyout with CN Biosciences & Merck KGaA.

At Merck KGaA, Johnson held a variety of positions as General Manager for EMD Biosciences, managing teams to achieve global sales of $80M. She was promoted to Vice President of Operations for EMD Biosciences and later to Vice President of Corporate Development for EMD Chemicals North America.

In June 2009, she joined Semba Biosciences, Inc. as Chief Business Officer. In 2011, Johnson left the bioscience world to join state government as Vice President, Entrepreneurship and Innovation for the Wisconsin Economic Development Corporation.

Altimmune Completes Enrollment In Phase 1b Clinical Trial Of NasoShield™, A Single Dose Intranasal Anthrax Vaccine Candidate

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GAITHERSBURG, Md., Aug. 07, 2020 (GLOBE NEWSWIRE) — Altimmune, Inc. (Nasdaq: ALT), a clinical-stage biopharmaceutical company, today announced that it has completed enrollment in its Phase 1b clinical trial of NasoShield, a single dose intranasal anthrax vaccine candidate. With this milestone, the data readout for the Day 28 immunogenicity endpoint remains on target for Q4 2020.

The clinical trial has enrolled 42 evaluable healthy subjects to receive intranasally administered NasoShield or placebo and be followed for 6 months following dosing. The primary immunogenicity readouts are the serum antibody response to protective antigen and toxin-neutralizing antibody titer 28 and 56 days after dosing. As with Altimmune’s other vaccine programs, stimulation of a mucosal IgA immune response in the nasal cavity will also be assessed as a potential additional benefit to other immunologic responses. Nasal mucosal immunity, which can play an important role in the body’s defense against respiratory diseases, is best stimulated by the nasal route of vaccine administration.

The NasoShield program is being developed under a contract with the Biomedical Advanced Research and Development Authority (BARDA), with a total potential value of $133.7 million if all options in the contract (HHSO100201600008C) are exercised. At the conclusion of the Phase 1b NasoShield trial, BARDA will have the option of exercising the remaining contract options valued at approximately $105 million to enable Phase 2 development.

NasoShield has the potential to be a convenient and simple alternative to the current approved multi-dose anthrax vaccine, as it is intended to confer protection after a single intranasal dose instead of the three-injection regimen required by the only licensed vaccine. Intranasal dosing provides the potential to be administered rapidly and without the need for needles, syringes or trained healthcare personnel in the event of an anthrax incident. In addition, NasoShield’s expected room temperature stability profile may allow for broad distribution of the vaccine without the need for expensive cold-chain logistics, such as refrigeration or freezing.

Earlier this quarter, the Company reported positive preclinical data on AdCOVID, a single dose intranasal COVID-19 vaccine candidate which is derived from the same replication-deficient adenovirus 5 vaccine platform technology as NasoShield. In these preclinical studies performed in collaboration with the University of Alabama at Birmingham (UAB), AdCOVID elicited a strong systemic antibody response against the receptor-binding domain (RBD) in mice, stimulated serum viral neutralization titers, and activated a 29-fold induction of mucosal IgA against SARS-CoV-2 in bronchoalveolar fluid of vaccinated mice. The Company plans to initiate a Phase 1 clinical trial of AdCOVID during the fourth quarter of 2020.

About Altimmune

Altimmune is a clinical stage biopharmaceutical company focused on developing intranasal vaccines, immune modulating therapies and treatments for liver disease. Our diverse pipeline includes proprietary intranasal vaccines for COVID-19 (AdCOVID™), anthrax (NasoShield™) and influenza (NasoVAX™); an intranasal immune modulating treatment for COVID-19 (T-COVID™); and next generation peptide therapeutics for NASH (ALT-801) and chronic hepatitis B (HepTcell™). For more information on Altimmune, please visit www.altimmune.com.

Forward-Looking Statement

Any statements made in this press release relating to future financial or business performance, conditions, plans, prospects, trends, or strategies and other financial and business matters, including without limitation, the timing of key milestones for our clinical assets, the initiation and timing of the NasoShield clinical trial and receipt of data from the clinical trial in 2020, the potential for additional funding from BARDA, the potential immunization effects of NasoShield, and the prospects for regulatory approval, commercializing or selling any product or drug candidates, are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In addition, when or if used in this press release, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Altimmune, Inc. (the “Company”) may identify forward-looking statements. The Company cautions that these forward-looking statements are subject to numerous assumptions, risks, and uncertainties, which change over time. Important factors that may cause actual results to differ materially from the results discussed in the forward looking statements or historical experience include risks and uncertainties, including risks relating to: potential impacts due to the COVID-19 pandemic such as delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy the reliability of the results of the studies relating to human safety and possible adverse effects resulting from the administration of the Company’s product candidates; funding delays, reductions in or elimination of U.S. government funding and/or non-renewal of expiring funding under the Company’s agreement with BARDA; the Company’s ability to satisfy certain technical milestones under the Company’s contracts with BARDA that would entitle the Company to receive additional funding over the period of the agreement; the Company’s ability to obtain potential regulatory approvals on the timelines anticipated, or at all; and the Company’s ability to expand its pipeline of products and the success of future product advancements, including the success of future clinical trials, and the Company’s ability to commercialize its products. Further information on the factors and risks that could affect the Company’s business, financial conditions and results of operations are contained in the Company’s filings with the U.S. Securities and Exchange Commission, including under the heading “Risk Factors” in the Company’s annual report on Form 10-K for the fiscal year ended December 31, 2019 and quarterly report on Form 10-Q for the quarter ended March 31, 2020 filed with the SEC, which are available at www.sec.gov.

Investor Contacts:
Will Brown Ashley R. Robinson
Chief Financial Officer LifeSci Advisors, LLC
Phone: 240-654-1450 617-430-7577
wbrown@altimmune.com arr@lifesciadvisors.com
Media Contacts:
Warren Rizzi
Sard Verbinnen & Co.
Phone: 212-687-8080
altimmune-svc@sardverb.com

GEN: NIH Invests $248.7 Million to Ramp up COVID-19 Testing

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Six months into the COVID-19 pandemic, the question of why tests that detect the presence of the SARS-CoV-2 virus are not faster, better, and cheaper in this country still remains. Despite an incredibly complicated answer, the National Institutes of Health (NIH) has announced that they are going to help some companies work to develop their technologies, with the hope of getting over this roadblock. The program will give $248.7 million to seven biomedical diagnostic companies. The money is intended to support a range of new lab-based and point-of-care tests that, they hope, could significantly increase the number, type, and availability of tests by millions per week as early as September 2020.

The initiative is called NIH’s Rapid Acceleration of Diagnostics (RADx). “The RADx initiative has enabled some of the nation’s most creative biomedical device inventors to ramp up development of their testing technologies at unprecedented speed,” said NIH director Francis S. Collins, MD, PhD. “The innovations selected to date represent the diverse types of promising technologies that will serve the nation’s testing needs.”

The seven companies range in scope from small start-ups to large publicly-held organizations and their corresponding technologies use different methods and formats and can be performed in a variety of settings to meet diverse needs.

Click here to read the entire article from GEN

Adaptive Phage Therapeutics Announces Mayo Clinic as Lead Investor in Convertible Note Financing

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GAITHERSBURG, Md.–(BUSINESS WIRE)–Adaptive Phage Therapeutics (APT), a clinical-stage biotechnology company founded to provide an effective therapeutic response to the global rise of multi-drug resistant (MDR) pathogenic bacteria, today announced Mayo Clinic has committed $1.75M as a lead investor in a $7 million convertible note financing round.

Mayo Clinic is collaborating with APT to conduct a clinical trial using PhageBankTM investigational therapy to address the significant unmet need for alternative treatments for chronic infections following joint replacement (referred to as “periprosthetic joint infections,” or “PJIs”). Proceeds from the convertible note are designated to initiate the trial.

“Prosthetic joint infection is a potential complication of joint replacement implantation that can be very serious and lead to loss of life or limb. As we face a growing public health threat from drug-resistant bacterial infections, we need more research to assess phage therapy’s potential to help people with these infections and save lives,” said Gina Suh, M.D., infectious diseases specialist and one of the experts at Mayo Clinic leading the research collaboration with APT.

APT has supported Mayo Clinic physicians in the treatment of multiple patients with end-stage chronic PJIs under emergency use approvals by the US Food and Drug Administration (FDA). In each case, the patient faced serious challenges due to chronic and refractory infections unresponsive to commercially approved therapies following knee replacement implantations. Use of APT’s PhageBank in lieu of surgery is often referred to as the “Haverty Protocol,” in recognition of John Haverty, the first PJI patient treated with investigational PhageBank therapy at Mayo Clinic. The research is published in the July issue of Clinical Infectious Diseases (CID).

APT anticipates initiating the PJI trial at Mayo Clinic in the fall of 2020. If successful, the company anticipates potential commercial availability in 2023. Based on an international survey of hospital payers and doctors, conducted by Triangle Insights Group, it is estimated that APT’s PhageBank treatment of PJI represents a commercial market of more than $900 million per year.

“APT is pleased that Mayo Clinic recognizes the value of phage therapy and its role in treating chronic PJI infection. Current standard of care can cost up to $400k per patient and often results in five-year mortality rates comparable to those of colon cancer,” said Greg Merril, co-founder and chief executive officer, Adaptive Phage Therapeutics.

Last month, APT announced a collaboration with Mayo Clinic to advance and commercialize a phage susceptibility test (PST) to be offered as a laboratory developed test for worldwide commercial availability by Mayo Clinic Laboratories. PST will enable rapid identification of patient-specific precision therapy for challenging bacterial infections.

Mayo Clinic and Dr. Suh have a financial interest in the technology referenced in this news release. Mayo Clinic will use any revenue it receives to support its not-for-profit mission in patient care, education and research.

About Antimicrobial Resistance (AMR)

According to the Centers for Disease Control, more than 2.8 million antibiotic-resistant infections occur in the United States each year, and more than 35,000 people die as a result.1 The World Health Organization estimates that superbugs will kill up to 10 million people globally each year by 2050.2 Thirty-five percent of common human bacterial infections are already resistant to currently available medicines in some high-income countries.3

According to the World Health Organization, there is a US $100 trillion potential cost in terms of lost global production between now and 2050.4 The indirect costs of drug-resistant infections to the individual and society from morbidity, disability, premature deaths and reduced effective labor supply are estimated to cause a decrease in the global economic output of 1–3% by 2030, with estimated losses ranging from US $1 trillion to US $3.4 trillion annually if no action is taken.5

About Periprosthetic Joint Infections (PJI)

Periprosthetic joint infections (PJIs), are infections involving a joint prosthesis and adjacent tissue, which develop following a joint replacement implantation. PJIs have been projected to occur in approximately two percent of joint replacement surgeries performed. Risk factors for PJI include co-morbidities of obesity, diabetes or rheumatoid arthritis, among others. PJI is known to occur more frequently in revision surgeries, and the majority of PJIs occur within one year of surgery. The economic impact to the healthcare industry is projected to reach more than $1.62 billion in 2020. PJIs are painful and create a tremendous burden for individual patients.9

About Phage

Bacteriophage (“phage”) are viruses that host on bacteria. Phage have evolved to become the most prolific killers of bacteria on earth. Phage have been used for clinical applications since their initial discovery at the beginning of the twentieth century. The ability of phage to replicate exponentially and kill bacteria suggests that they could play a vital role in our armamentarium for the treatment of infectious diseases.8 There are an estimated 10 phage for each bacteria on earth, and each phage strain has evolved to eradicate a narrow range of bacteria. The introduction of penicillin in the 1940s displaced interest in commercial development of phage. With the growing crisis of bacterial resistance to antibiotics, there has been renewed interest in phage therapy.

About PhageBank

PhageBank is an ever-expanding collection of genetically diverse, carefully screened, and purified phage that are collectively broad spectrum, negating the effects of bacterial resistance that increasingly diminish the effectiveness of antibiotics. In 2017, APT acquired world-wide exclusive commercial rights to PhageBank from the biodefense program of the U.S. Department of Defense. APT is advancing the technology through innovation in bioinformatics, rapid phage-bacteria matching and phage purification methods in an approach capable of providing a precision-matched therapy based upon the diagnosis of a specific bacterial pathogen.

About Adaptive Phage Therapeutics (APT)

APT is a clinical-stage company founded to provide an effective therapeutic response to the global rise of multi-drug resistant (MDR) pathogenic bacteria. APT’s core asset is a large and continually expanding phage library (PhageBank™) deployed with a companion diagnostic to achieve rapid response and cost-effective therapy for otherwise recalcitrant bacterial infections. The technology was developed by the biodefense program of U.S. Department of Defense. APT acquired the world-wide exclusive commercial rights in 2017. Under the FDA emergency Investigational New Drug (eIND) allowance, APT has provided investigational PhageBank therapy to treat more than 23 critically ill patients in which standard-of-care antibiotics had failed. For more information, visit http://www.aphage.com.

REGENXBIO Announces Additional Positive Interim Phase I/IIa Trial Update and Program Updates for RGX-314 for the Treatment of Wet AMD

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ROCKVILLE, Md., Aug. 4, 2020 /PRNewswire/ —

  • Company on track to initiate RGX-314 subretinal delivery pivotal program by the end of 2020
  • RGX-314 was generally well-tolerated in 42 patients at all dose levels in Phase I/IIa trial
  • Positive interim update from Cohorts 4 and 5 at one year informs pivotal program
    • Durable treatment effect observed with stable to improved visual acuity and retinal thickness
    • Demonstrated meaningful reductions in anti-VEGF treatment burden over one year
      • 61% and 85% reduction of anti-VEGF injections in Cohorts 4 and 5, respectively
      • 73% of patients (8/11) in Cohort 5 remain anti-VEGF injection-free
    • Intraocular RGX-314 protein expression levels are dose-dependent at one year
  • Phase II trial for RGX-314 for the treatment of wet AMD using suprachoroidal delivery (AAVIATE) is active
    • Enrollment expected to begin in Q3 2020; interim data update from first cohort expected by end of 2020
  • Company to host conference call and webcast on Tuesday, August 4 at 8:30a.m. ET, featuring wet AMD Key Opinion Leaders, Robert Avery, M.D., Dante Pieramici, M.D., and Peter Kaiser, M.D.

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 reported positive one year data from patients in Cohorts 4 and 5 of the Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). The Company plans to initiate a pivotal program for subretinal delivery of RGX-314 in patients with wet AMD by the end of 2020. In addition, REGENXBIO today announced that a Phase II trial of RGX-314 for the treatment of wet AMD delivered to the suprachoroidal space (AAVIATE) is active and expected to enroll patients in the third quarter of 2020.

“Today’s results provide further evidence of the clinical profile of RGX-314 as a promising one-time gene therapy treatment paradigm for patients with wet AMD,” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. “The data demonstrated stable to improved visual acuity and retinal thickness, as well as a meaningful reduction in anti-VEGF injection burden, in these higher dose levels at one year. Results from the Phase I/IIa trial will inform the design of the pivotal program of RGX-314 in patients with wet AMD, which we look forward to initiating by the end of this year.”

Dr. Pakola continued: “I am also pleased to announce that our AAVIATE trial, a Phase II trial of RGX-314 for the treatment of wet AMD utilizing the SCS Microinjector, is active and we expect to dose the first patient this quarter. The targeted, in-office suprachoroidal delivery approach may provide additional options for patients in all settings of patient care. We anticipate providing an interim data update from the first cohort in late 2020.”

“Based on the overall results to date from the Phase I/IIa trial, I believe that RGX-314 has the potential to profoundly impact all aspects of clinical management for patients with wet AMD,” said Robert Avery, M.D., Founder of California Retina Consultants and Research Foundation and investigator surgeon in the Phase I/IIa RGX-314 trial. “Wet AMD affects a large number of adults, and often results in loss of vision over time due to non-compliance with the current standard of care of frequent anti-VEGF injections. I am encouraged that RGX-314 has the potential to become a one-time gene therapy treatment option for a broad range of patients.”

Study Design and Safety Update from Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD using Subretinal Delivery

In the Phase I/IIa trial of RGX-314, 42 patients with long-standing severe wet AMD requiring frequent anti-vascular endothelial growth factor (anti-VEGF) injections were treated across five dose cohorts, with doses ranging from 3×109 GC/eye to 2.5×1011 GC/eye. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic or supplemental immune suppressive corticosteroid therapy for RGX-314.

Patients in the study are being assessed each month for two years and will receive safety follow-up for five years after RGX-314 administration. Efficacy assessments for the study include number of anti-VEGF intravitreal injections, change in vision as measured by Best Corrected Visual Acuity (BCVA), change in central retinal thickness (CRT) as measured by spectral domain optical coherence tomography (SD-OCT), and RGX-314 protein expression levels as measured from aqueous samples by electrochemiluminescence immunoassay (ECL).

As of July 13, 2020, RGX-314 was generally well-tolerated across all cohorts. Eighteen serious adverse events (SAEs) were reported in 11 patients, including 17 that were not related to RGX-314. One possibly drug-related SAE of significant decrease in vision was reported in Cohort 5 at Month 11 in a patient who had retinal pigmentary changes that involved the macula. The most common nonserious adverse events in the eye were generally assessed as mild (77%). These included post-operative conjunctival hemorrhage (69% of patients), post-operative inflammation (36% of patients), eye irritation (17% of patients), eye pain (17% of patients), and post-operative visual acuity reduction (17% of patients). In 67% of patients across all cohorts, and in 83% of patients in Cohorts 3 through 5, retinal pigmentary changes were observed on imaging, the majority of which were in the peripheral inferior retina. Retinal hemorrhage was observed in 24% of patients and is an anticipated event in patients with severe wet AMD. There have been no reports of clinically determined immune responses, drug-related ocular inflammation, or post-surgical inflammation beyond what is expected following routine vitrectomy.

Summary of Data from Cohorts 4 and 5 of Phase I/IIa Trial of RGX-314 for the Treatment of Wet AMD using Subretinal Delivery

Today’s update includes data from Cohorts 4 and 5 as of July 13, 2020. Each cohort enrolled 12 patients each at doses of 1.6×1011 GC/eye and 2.5×1011 GC/eye, respectively.

Patients in Cohort 4 and Cohort 5 at one year after administration of RGX-314 demonstrated stable visual acuity with a mean BCVA change of +4 letters and -2 letters from baseline, respectively, as well as decreased retinal thickness, with a mean change in CRT of -61 µm and -79 µm, respectively.

There was a clinically significant and meaningful reduction in anti-VEGF treatment burden in both Cohorts 4 and 5 compared to the 12 months prior to RGX-314 administration. Patients in Cohort 4 received a mean of 4.1 injections over one year following administration of RGX-314, a 61% reduction in treatment burden. Patients in Cohort 5 received a mean of 1.4 injections over one year following administration of RGX-314, a reduction in treatment burden of 85%.

In Cohort 4, three out of twelve (25%) patients received no anti-VEGF injections over one year, and these patients demonstrated a mean BCVA improvement of +6 letters and a mean reduction in CRT of -62 µm at one year. In Cohort 5, eight out of the eleven (73%) patients observed through one year have received no anti-VEGF injections after administration of RGX-314 and these patients demonstrated a stable mean BCVA change of 0 letters and a mean reduction in CRT of -95 µm at one year.

Consistent with previous results, intraocular RGX-314 protein expression levels were observed in a dose-dependent manner across each cohort at one year after administration of RGX-314. The mean protein expression levels in Cohort 4 and Cohort 5 were 420.9 ng/ml and 457.5 ng/ml, respectively.

Study Design for Phase II Trial for RGX-314 for Treatment of Wet AMD using Suprachoroidal Delivery (AAVIATE)

REGENXBIO also announced that a Phase II trial, AAVIATE, to evaluate the suprachoroidal delivery of RGX-314 in patients with wet AMD, will begin dosing patients in the third quarter of 2020. AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation study that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector, a targeted, in-office route of administration.

AAVIATE will enroll 40 patients with severe wet AMD who are responsive to anti-VEGF treatment. Patients will be randomized to one-time RGX-314 SCS delivery versus monthly 0.5 mg ranibizumab intraocular injection at a 3:1 ratio and two dose levels of RGX-314 will be evaluated: 2.5×1011 GC/eye and 5×1011 GC/eye. Patients will not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.

The primary endpoint of the study is mean change in vision, as measured by BCVA, at 40 weeks from baseline compared to monthly ranibizumab. Other endpoints include mean change in CRT and number of anti-VEGF intravitreal injections.

The Company expects to report interim data from the first cohort of this trial by the end of 2020.

Conference Call

In connection with this announcement, REGENXBIO will host a webcast and conference call with accompanying slides today at 8:30 a.m. ET. This event will feature Robert Avery, M.D., Founder of California Retina Consultants and Research Foundation, Dante Pieramici, M.D., Director, California Retina Research Foundation and Partner, California Retina Consultants, and Peter Kaiser, M.D., Chaney Family Endowed Chair in Ophthalmology Research and Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine and Cole Eye Institute.

To access a live or recorded webcast of the call and accompanying slides, please visit the “Investors” section of the REGENXBIO website at www.regenxbio.com. To access the live call by phone, dial (888) 317-6003 (domestic) or (412) 317-6061 (international) and enter the passcode 6960652. The recorded webcast will be available for approximately 30 days following the call.

About RGX-314

RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other additional chronic retinal conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an antibody fragment which is designed to inhibit VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss.

About the Phase I/IIa Clinical Trial of RGX-314

RGX–314 is being evaluated in a Phase I/IIa, multi-center, open-label, multiple-cohort, dose–escalation study in adult patients with wet AMD in the United States. The study includes patients previously treated for wet AMD who are responsive to anti-VEGF therapy. The study is designed to evaluate five escalating doses of RGX-314, with six patients in the first three dose cohorts and 12 patients in the fourth and fifth dose cohorts. Patients were enrolled into all dose cohorts independent of their neutralizing antibody titers to AAV and did not receive prophylactic immune suppressive oral corticosteroid therapy before or after administration of RGX-314. The primary endpoint of the study is safety at 6 months following administration of RGX-314. Secondary endpoints include visual acuity, retinal thickness on SD–OCT, ocular RGX-314 protein expression, and the need for additional anti-VEGF therapy. Following completion of the primary study period, patients enter a follow-up period and will continue to be assessed until week 106 for long-term safety and durability of effect.

About Wet AMD

Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long intraocular injections, typically repeated every four to 12 weeks in frequency, to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

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

Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com

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

(PRNewsfoto/REGENXBIO Inc.)

Jennifer Butler, Executive Vice President and General Manager, Innate Pharma US Inc. sits down with Rich Bendis for BioTalk

By | BioTalk, News | No Comments

Jennifer Butler, Executive Vice President and General Manager, Innate Pharma US Inc. joins Rich Bendis on BioTalk to discuss her career, bringing Innate from France to the US, and goals.

Listen now on Apple https://apple.co/3i30Bxs, Google https://bit.ly/2PkUw2U, Spotify https://spoti.fi/31gDh8n, TuneIn https://bit.ly/3fqn7i3, and YouTube Audio https://bit.ly/3kgU9ou.

Jennifer Butler was appointed Executive Vice President and General Manager of Innate Pharma US Inc. in March 2019.

Prior to her appointment, Ms. Butler served as Chief Business Officer, Chief Commercial Officer and Head of US operations, responsible for global business development and commercial strategy at Tessa Therapeutics, a clinical-stage oncology company. She previously served for more than 10 years in various commercial and strategy roles with increasing responsibility at AstraZeneca/MedImmune across several therapeutic areas. Ms. Butler also previously worked in strategic healthcare consulting and as an analyst in Equity Capital Markets at Merrill Lynch.

Ms. Butler has more than 20 years of strategic marketing and commercial leadership expertise along with general management experience.

Click Here to Read the Transcript.

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