Soligenix Announces Topline Results from its Phase 3 Clinical Trial of SGX942 for the Treatment of Oral Mucositis in Head and Neck Cancer Patients -…

PRINCETON, N.J., Dec. 22, 2020 /PRNewswire/ --Soligenix, Inc. (NASDAQ: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need,announced today preliminary top-line results for its pivotal Phase 3 DOM-INNATE (Dusquetide treatment in Oral Mucositis by modulating INNATE Immunity) trial evaluating SGX942 (dusquetide) in the treatment of severe oral mucositis (SOM) in patients with head and neck cancer (HNC) receiving chemoradiation. The study enrolled 268 patients randomized 1:1 to receive either SGX942 or placebo. The primary endpoint of median duration of SOM did not achieve the pre-specified criterion for statistical significance (p0.05); although biological activity was observed with a 56% reduction in the median duration of SOM from 18 days in the placebo group to 8 days in the SGX942 treatment group. Despite this clinically meaningful improvement, the variability in the distribution of the data yielded a p-value that was not statistically significant.

Other secondary endpoints supported the biological activity of dusquetide, including a statistically significant 50% reduction in the duration of SOM in the per-protocol population, which decreased from 18 days in the placebo group to 9 days in the SGX942 treatment group (p=0.049), consistent with the findings in the Phase 2 trial. Similarly, incidence of SOM also followed this biological trend as seen in the Phase 2 study, decreasing by 16% in the SGX942 treatment group relative to the placebo group in the per-protocol population. The per-protocol population was defined as the population receiving a minimum of 55 Gy radiation and at least 10 doses of study drug (placebo or SGX942) throughout the intended treatment period, with no major protocol deviations (e.g. breaks in study drug administration longer than 8 days between successive doses).

"We are obviously very disappointed with the unanticipated outcome of the study," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix."Despite the fact that SGX942 demonstrated clinically meaningful reductions in oral mucositis consistent with the Phase 2 study, the Phase 3 trial did not achieve the statistically significant benefit we expected. Over the coming weeks, we will be analyzing the data to better determine why the study did not meet expectations. If there is any clarity gained from further analysis of the dataset, especially with respect to specific subsets of patients that may benefit from SGX942 therapy, we will certainly communicate our findings and explore follow-up discussions with the FDA and the EMA."

Dr. Schaber continued, "With approximately $20 million of cash and our non-dilutive government funding, we will evaluate strategic options as we continue to execute on the multiple development programs across our rare disease pipeline. Most importantly, this will include the preparation of a New Drug Application for SGX301 in the treatment of cutaneous T-cell lymphoma, which demonstrated statistical significance in its pivotal Phase 3 clinical trial earlier this year, as well as continuing activities towards SGX301 U.S. commercialization where we expect peak annual sales to exceed $75 million."

The Company will host a webcast and conference call today at 8:30 AM EST to review the top-line findings.

Conference Call, December 22, 2020 at8:30 AM Eastern Time

The Company will share information on its Phase 3 top-line results for its SGX942 program in oral mucositis. A question and answer (Q&A) session with management will follow the presentations. If you would like to ask a question during the Q&A, please submit your request via emailtoir@soligenix.comat least 15 minutes prior to the scheduled start of the call.

U.S. toll free:1-866-652-5200 International:1-412-317-6060 Please request to be entered into the Soligenix call.

A transcript of the presentation will be archived for 30 days following the event.

About Oral Mucositis

Mucositis is the clinical term for damage done to the mucosa by anticancer therapies. It can occur in any mucosal region, but is most commonly associated with the mouth, followed by the small intestine. It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of mucositis, that mucositis affects approximately 500,000 people in the U.S. per year and occurs in 40% of patients receiving chemotherapy. Mucositis can be severely debilitating and can lead to infection, sepsis, the need for parenteral nutrition and narcotic analgesia. The gastrointestinal damage causes severe diarrhea. These symptoms can limit the doses and duration of cancer treatment, leading to sub-optimal treatment outcomes.

The mechanisms of mucositis have been extensively studied and have been recently linked to the interaction of chemotherapy and/or radiation therapy with the innate defense system. Bacterial infection of the ulcerative lesions is now regarded as a secondary consequence of dysregulated local inflammation triggered by therapy-induced cell death, rather than as the primary cause of the lesions.

It is estimated, based upon review of historic published studies and reports and an interpolation of data on the incidence of oral mucositis, that oral mucositis in HNC is a subpopulation of approximately 90,000 patients in the U.S., with a comparable number in Europe. Oral mucositis almost always occurs in patients with HNC treated with CRT and is severe, causing inability to eat and/or drink, in >80% of patients. It is common (40-100% incidence) in patients undergoing high dose chemotherapy and hematopoietic cell transplantation, where the incidence and severity of oral mucositis depends greatly on the nature of the conditioning regimen used for myeloablation.

In the pediatric population, head and neck cancer is a rarer occurrence and is caused by different underlying pathologies. The major types of HNC in children are lymphoma, sarcomas (including rhabdomyosarcomas), and neuroblastoma rather than squamous cell carcinoma, the major type of adult HNC cancers. Hematopoietic stem cell transplantation (HSCT), especially allogeneic transplantation with higher risk of oral mucositis, is more frequently used in the pediatric population than in adults when treating a number of primary tumor types, as seen in leukemia and lymphoma. Both treatment of HNC and HSCT are associated with high risk of oral mucositis in the pediatric population.

Oral mucositis remains an area of unmet medical need where there are currently no approved drug therapies in the context of any solid tissue tumors.

About the Phase 3 DOM-INNATE Study

This multinational, placebo-controlled, randomized study enrolled 268 subjects with squamous cell carcinoma of the oral cavity and oropharynx, scheduled to receive a minimum total cumulative radiation dose of 55 Gy fractionated as 2.0-2.2 Gy per day with concomitant cisplatin chemotherapy given as a dose of 80-100 mg/m2 every third week.Subjects were randomized to receive either 1.5 mg/kg SGX942 or placebo given twice a week during and for two weeks following completion of CRT. The primary endpoint for the study is the median duration of SOM, assessed by oral examination at each treatment visit and then through six weeks following completion of CRT.Oral mucositis is evaluated using the WHO (World Health Organization) Grading system.SOM is defined as a WHO Grade of 3. A positive interim analysis was conducted in August 2019, resulting in the recommended addition of 35 subjects / group to the study to maintain 90% power.Subjects are being followed for an additional 12 months after the completion of treatment.Soligenix has been working with leading oncology centers internationally, a number of which participated in the Phase 2 study.

About Dusquetide

Dusquetide (the active ingredient in SGX942) is an innate defense regulator (IDR), a new class of short, synthetic peptides. It has a novel mechanism of action whereby it modulates the body's reaction to both injury and infection towards an anti-inflammatory, anti-infective and tissue healing response. IDRs have no direct antibiotic activity but, by modulating the host's innate immune system responses, increase survival after infections caused by a broad range of bacterial Gram-negative and Gram-positive pathogens. It also accelerates resolution of tissue damage following exposure to a variety of agents including bacterial pathogens, trauma and chemo- and/or radiation therapy. Preclinical efficacy and safety has been demonstrated in numerous animal disease models including mucositis, colitis, macrophage activation syndrome (MAS) as well as bacterial infections, including melioidosis.

SGX942 has demonstrated safety in a Phase 1 clinical study in 84 healthy human volunteers. Positive efficacy results were demonstrated in an exploratory Phase 2 clinical study in 111 patients with oral mucositis due to CRT for HNC.

SGX942 has received Fast Track Designation from the FDA for the treatment of oral mucositis as a result of radiation and/or chemotherapy treatment in HNC patients, as well as Promising Innovative Medicine designation in the United Kingdom by the Medicines and Healthcare Products Regulatory Agency for the treatment of SOM in HNC patients receiving CRT. In addition, products containing the same active ingredient, dusquetide, have been granted Fast Track Designation as an adjunctive therapy with other antibacterial drugs, for the treatment of melioidosis and Orphan Drug Designations in the treatment of MAS and the treatment of acute radiation syndrome.

Soligenix has a strong intellectual property position in the IDR technology platform, including composition of matter for dusquetide and related analogs. Dusquetide was developed pursuant to discoveries made by Professors B. Brett Finlay, PhD and Robert Hancock, PhD of the University of British Columbia, Canada. Soligenix has received partial funding from NIH for its oral mucositis clinical studies. The Phase 2 study was supported with a Phase I SBIR grant (#R43DE024032) award, with the Phase 3 study supported by a Phase II SBIR grant (#R44DE024032) award.

In addition, a high level review of the IDR technology platform is available here.

About Soligenix, Inc.

Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing SGX301 as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma, our first-in-class IDR technology, dusquetide (SGX942) for the treatment of oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203) and acute radiation enteritis (SGX201).

Our Public Health Solutions business segment includes active development programs for RiVax, our ricin toxin vaccine candidate, SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease, and our research programs to identify and develop novel vaccine candidates targeting viral infection including Ebola, Marburg and SARS-CoV-2 (the cause of COVID-19). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agents (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA).

For further information regarding Soligenix, Inc., please visit the Company's website at http://www.soligenix.com.

This press release may contain forward-looking statements that reflect Soligenix, Inc.'s current expectations about its future results, performance, prospects and opportunities, including but not limited to, potential market sizes, patient populations and clinical trial enrollment. Statements that are not historical facts, such as "anticipates," "estimates," "believes," "hopes," "intends," "plans," "expects," "goal," "may," "suggest," "will," "potential," or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, such as experienced with the COVID-19 outbreak. Soligenix cannot assure you that it will be able to successfully develop, achieve regulatory approval for or commercialize products based on its technologies, particularly in light of the significant uncertainty inherent in developing therapeutics and vaccines against bioterror threats, conducting preclinical and clinical trials of therapeutics and vaccines, obtaining regulatory approvals and manufacturing therapeutics and vaccines, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that it will be able to successfully obtain any further funding to support product development and commercialization efforts, including grants and awards, maintain its existing grants which are subject to performance requirements, enter into any biodefense procurement contracts with the US Government or other countries, that it will be able to compete with larger and better financed competitors in the biotechnology industry, that changes in health care practice, third party reimbursement limitations and Federal and/or state health care reform initiatives will not negatively affect its business, or that the US Congress may not pass any legislation that would provide additional funding for the Project BioShield program. Despite the statistically significant result achieved in the SGX301 Phase 3 clinical trial for the treatment of cutaneous T-cell lymphoma, there can be no assurance that a marketing authorization from the FDA or EMA will be successful. Further, there can be no assurance that RiVax will qualify for a biodefense Priority Review Voucher (PRV) or that the prior sales of PRVs will be indicative of any potential sales price for a PRV for RiVax. Also, no assurance can be provided that the Company will receive or continue to receive non-dilutive government funding from grants and contracts that have been or may be awarded or for which the Company will apply in the future. These and other risk factors are described from time to time in filings with the Securities and Exchange Commission, including, but not limited to, Soligenix's reports on Forms 10-Q and 10-K. Unless required by law, Soligenix assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.

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SOURCE Soligenix, Inc.

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Soligenix Announces Topline Results from its Phase 3 Clinical Trial of SGX942 for the Treatment of Oral Mucositis in Head and Neck Cancer Patients -...

The global cell isolation market size is projected to reach USD 15.0 billion by 2025 from USD 6.9billion in 2020, at a CAGR of 16.8% – GlobeNewswire

December 21, 2020 05:04 ET | Source: ReportLinker

New York, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Cell Isolation/Cell Separation Market by Product, Cell Type, Cell Source, Technique, Application, End-User - Global Forecast to 2025" - https://www.reportlinker.com/p04315097/?utm_source=GNW With the rising focus on the development of personalized medicine, the number of personalized medications available in the market has steadily increased over the last decade, and this trend is expected to continue in the coming years.

The consumablesaccounted for the highest growth rate in thecell isolationmarket, by productduring the forecast period Based on product, the cell isolation market is segmented into consumables and instruments.The consumables segment accounted for the largest share in the cell isolation market in the forecasted period.

The increasing investments by companies to develop technologically advanced products as well as the repetitive use of consumables as compared to instruments are the major factors driving the growth of this segment.

Human cells segment accounted for the highest CAGR Based on cell type, the cell isolation market is segmented into human cells and animal cells.The human cells segment accounted for the largest share of the global cell isolation market in the forecasted period.

The increasing investments by public and private organizations for research on human cells, growing application areas of human stem cells, and the high and growing incidence of diseases such as cancer are the major factors driving this segments growth.

Biotechnology and biopharmaceutical companiessegment accounted for the highest CAGR The cell isolation market is segmented into hospitals and diagnostic laboratories, biotechnology and biopharmaceutical companies, research laboratories and institutes, and other end users based on end users.In 2019, the biotechnology and biopharmaceutical companies segment accounted for the largest share.

The widespread adoption of advanced instruments in cell-based experiments and cancer research in biotechnology and biopharmaceutical companies, as well as the increasing number of R&D facilities globally are the major factors driving this segments growth.

Asia Pacific: The fastest-growing regioncell isolation market The global cell isolation market is segmented into North America, Europe, Asia Pacific, and Rest of the world.The Asia Pacific region is projected to register the highest CAGR during the forecast period.

Growth in this region is expected to be centered on China and Japan. Factors such as the expansion by key market players in emerging Asian countries and the increasing trend of pharmaceutical outsourcing to Asian countries like India and China are driving the growth of the cell isolation market in this region.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 20%, Tier 2 - 45%,and Tier 3 -35% By Designation: C-level - 30%, D-level - 20%, and Others - 50% By Region: North America -35%, Europe - 24%, Asia Pacific - 25%, Rest of the world 16%

Lits of Companies Profiled in the Report: Thermo Fisher Scientific, Inc. (US) Becton, Dickinson and Company Limited (US) Beckman Coulter Inc. (US).Merck KGaA (Germany) Terumo BCT (Japan), GE Healthcare (US) Bio- Rad Laboratories Inc. (US) Corning Inc. (US) Roche Diagnostics (Switzerland) Alfa Laval (Sweden) Miltenyl Biotech (Germany) pluriSelect Life Science (Germany) STEMCELL Technologies Inc. (Canada) Akadeum Life Sciences, Inc (US) Bio- Techne (US), Bio Legend (US) Invent Biotechnologies (US)

Research Coverage: This report provides a detailed picture of the global cell isolation market.It aims at estimating the size and future growth potential of the market across different segments, such as product, celltype, cell source, technique, application, end user, and region.

The report also includes an in-depth competitive analysis ofthe key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report: The report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall cell isolation market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to better position their business and make suitable go-to-market strategies. This report will enable stakeholders to understand the markets pulse and provide them with information on the key market drivers, restraints, trends, and opportunities.

Read the full report: https://www.reportlinker.com/p04315097/?utm_source=GNW

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The global cell isolation market size is projected to reach USD 15.0 billion by 2025 from USD 6.9billion in 2020, at a CAGR of 16.8% - GlobeNewswire

Robert Gallo of the UM School of Medicine Institute of Human Virology and Global Virus Network Awarded Top Life Sciences and Medicine Prize from China…

BALTIMORE, Dec. 21, 2020 /PRNewswire/ --Robert C. Gallo, MD, The Homer & Martha Gudelsky Distinguished Professor in Medicine, co-founder and director of the Institute of Human Virology at the University of Maryland School of Medicine and co-founder and international scientific advisor of the Global Virus Network, was awarded the "VCANBIO Award for Biosciences and Medicine," a significant and authoritative award in the life sciences and medicine field of China. The elite Prize is jointly presented by the University of Chinese Academy of Sciences and the VCANBIO CELL & GENE ENGINEERING CORP, LTD to push forward scientific research, technological innovation and continuous development in the life sciences and medicine field of China.

"The Prize also serves to facilitate the industrial development and application of innovative life science achievements,"said George F. Gao, DVM, DPHIL (OXON), Director General of the Chinese Center for Disease Control and Prevention (China CDC), Director, CAS Key Laboratory of Pathogenic Microbiology and Immunology, Professor, Institute of Microbiology, Dean of the Medical School of the Chinese Academy of Sciences and Director of China's Global Virus Network Center of Excellence. "Dr. Gallo is a pioneer in virus research and most worthy of this Prize. We are pleased to see him recognized by many members of the Chinese Academy of Sciences."

"Hosted by the Medical School of the University of Chinese Academy of Sciences, this award commends outstanding and innovative Chinese and foreign scientists, who have accomplished innovation achievements and breakthroughs in the life sciences and medicine field,"said Yiming Shao, MD, the Chief Expert on AIDS, China CDC, Director of the Division of Research on Virology and Immunology, National Center for AIDS/STD Control and Prevention, China and Member of the GVN SARS-CoV-2 Task Force and China GVN. "I have worked with Dr. Gallo through the decades and admire his intellect and leadership, which have led to discoveries that have broad implications in protecting mankind from viral threats. I am delighted that my Chinese colleagues are recognizing him with this significant honor."

"Prof. Gallo has made a great deal of contribution to promote the Sino-American friendship and collaboration, especially for medical talent training and public health in China,"said Prof. Guanhua Xu, Chairman of the selection committee of the VCANBIO Award for Biosciences and Medicine.

"This is a tremendous and well-deserved honor for Dr. Gallo,"said E. Albert Reece, MD, PhD, MBA, Executive Vice President for Medical Affairs, UM Baltimore, the John Z. and Akiko K. Bowers Distinguished Professor, and Dean, University of Maryland School of Medicine. "Dr. Gallo has dedicated his career to building international collaborations that have produced major scientific discoveries in human virology, including with leading scientists and academic colleagues in China. As a result, the Institute of Human Virology continues to be recognized as the global leader in the fight against chronic viral diseases."

"I am humbled and honored to receive this esteemed Prize from my colleagues in China,"said Dr. Robert Gallo. "I take this opportunity to stress that it is imperative that government and politics not interfere with science, and that my Chinese and American colleagues, who have a long history of collaborating together and contributing scientific breakthroughs to protect humanity from global health threats, continue to have the freedom to do so and to grow these collaborations."

A Distinguished Scientific Career Advancing Global Health

Dr. Robert Gallo has long believed in the necessity of international cooperation and collaboration in medical sciences in general, and infectious diseases in particular, in part to build global friendships and advance humanitarian principles. Though entertainment and sports facilitate such connections he believes the "knots" are tied best through medical sciences. Throughout his 30 years at the National Institutes of Health (NIH) and more recently his nearly 25 years at Baltimore's Institute of Human Virology (IHV) at the University of Maryland School of Medicine, he has fostered these connections. First, through his pioneering scientific discoveries including his discovery in 1976 of Interleukin-2, the first cytokine, a growth regulating substance now used as immune therapy in some cancers and in autoimmune diseases when suppressive T cells are needed. Then in 1980, the first human retrovirus, HTLV-1, a cause of human leukemia and paralytic neurological diseases as well as severe inflammatory disorders, which is endemic in some regions such as parts of Africa, the Caribbean Islands, Japan, Aboriginal Australians, Iran and South America. Dr. Gallo and his team developed a blood test for HTLV-1 applicable to all countries which protects people receiving blood transfusions that would be contaminated with this virus. Similarly, in 1984, when he and his team co-discovered HIV as the cause of AIDS they also developed the HIV blood test for the world and made their reagents available to all. Dr. Gallo and his team established collaborations in HIV/AIDS research, education, therapy and care for many African countries, particularly in Nigeria and some Caribbean nations. During the current pandemic he quickly became involved in initiating preventive measures against SARS-CoV-2 and COVID-19 disease through the idea of stimulating innate immunity with "live" virus vaccines such as the oral polio vaccine. Dr. Gallo, abetted by his collaborator, Konstantin Chumakov, PhD, Associate Director for Research for the U.S. Food and Drug Administration's (FDA) Office of Vaccines Research and Review and a GVN Center Director and his clinical colleague, Shyam Kottilil, MBBS, PhD, professor of medicine and director of the Clinical Care and Research Division of the Institute of Human Virology at the University of Maryland School of Medicine and senior advisor at the GVN, are advising on trials in India and in discussion about trials in Mexico, Brazil, Uzbekistan and China. He has also fostered the international nature of his research by hosting students beyond the U.S., including post-doctoral senior scientists from Asia, Middle East, Europe, the Americas and many African nations.

However, nothing demonstrates his concern for medical science cooperation more than when he established the idea for the Global Virus Network (GVN), which he co-founded in 2011 with the late Prof. Reinhard Kurth, MD, formerly Director of the Robert Koch Institute in Berlin, and Prof. William Hall, BSc, PhD, MD, DTMH, of University College Dublin. Now, GVN is headed by its President Christian Brchot, MD, PhD. The GVN was formed to advance medical and zoological science without any government influence, giving members of the GVN maximum freedom to speak freely while encouraging all nations to be involved. China, among several dozen other countries, has an active Center of Excellence within the GVN and was the site of the 7th meeting of the GVN in May 2015 held in Beijing and hosted by the late GVN Center Director, Zeng Yi of Beijing University of Technology. Experts shared information on varying viral threats, including those causing hemorrhagic fevers, hepatitis, HIV, measles, influenza, dengue and chikungunya, to name a few. GVN members also reviewed strategies at the center of the organization including the creation of specialized task forces and the launch of training programs to address growing viral threats.

A History with Chinese-American Collaborations

Dr. Gallo had a deep friendship with Dr. Robert Ting who came to the U.S. as a child refugee from Shanghai during the Japanese invasion. As a student, Dr. Ting worked with the famous Italian molecular biologist, Dr. Salvatore Luria at MIT, who won a Nobel Prize. Dr. Ting then went to Caltech to work with another Italian Nobel Prize winner, Dr. Renato Dulbecco and they were joined by two others who were soon to be Nobel Prize winners, Drs. Howard Temin and David Baltimore. Dr. Ting was not just Dr. Gallo's friend but also his teacher by introducing him to Chinese culture and food, tennis, and the field of virology. Soon after meeting Dr. Ting, another Chinese-born and educated young man, Dr. Alan Wu, came to Dr. Gallo's lab from Toronto bringing with him the knowledge and skills of blood stem cells. There were then several other Chinese post-doctoral fellows culminating with Dr. Flossie Wong-Staal from Canton Province and who played a very major role in advancing molecular biology on Dr. Gallo's team for about 15 years. Dr. Nancy Chang, also Chinese, came as a visiting scientist on a few occasions. On one such time she was key to the development of the second-generation HIV blood test used around the world.

In 2009, with the help of a University of Maryland School of Medicine colleague, Dr. Richard Zhao, born in China and educated in the U.S., the Shandong Academy of Medical Sciences (SAMS) announced the establishment of the Shandong Gallo Institute of Virology (SGIV). The announcement was made simultaneously with a ceremony to establish China's first Molecular Diagnostic Center for Personalized Healthcare (MDCPH), which was a joint venture among the University of Maryland, Baltimore, Roche Diagnostics Asia Pacific and SGIV at the Shandong Academy of Medical Sciences. The mission of the SGIV is to promote the basic science of virology especially in the area of HIV/AIDS and other important and emerging viral diseases and to facilitate translational research and clinical trials for related diseases. SGIV also aims to provide molecular-based testing for disease diagnosis, prognosis and treatment in the area of individualized molecular testing for personalized medicine.

Since the founding of the Institute of Human Virology (IHV), Dr. Gallo notes that several of his key science leaders at the Institute of Human Virology came from China, including: Dr. Wuyuan Lu (recent Director of the Division of Infectious Agents and Cancer), Dr. Yang Liu (recent Director of the Division of Immunotherapy), Dr. Pan Zheng (Division of Immunotherapy), Dr. Lishan Su (current Director of the Division of Virology, Pathogenesis and Cancer), Dr. Man Charurat (current Director of the Division of Epidemiology and Prevention and Ciheb) and Dr. Lai-Xi Wang (formerly at IHV and now at University of Maryland, College Park). With each of these leaders also came labs full of Chinese colleagues, who Dr. Gallo states contributed greatly to advancing America's biomedical research. Further, over the past six decades, Dr. Gallo visited China countless times to discuss potential collaborations with public and private sector entities, mentored rising Chinese scientists and facilitated open scientific discussions to advance the field of human virology, among other important things.

About the Institute of Human Virology

Formed in 1996 as a partnership between the State of Maryland, the City of Baltimore, the University System of Maryland, and the University of Maryland Medical System, the IHV is an institute of the University of Maryland School of Medicine and is home to some of the most globally-recognized and world-renowned experts in all of virology. The IHV combines the disciplines of basic research, epidemiology, and clinical research in a concerted effort to speed the discovery of diagnostics and therapeutics for a wide variety of chronic and deadly viral and immune disorders - most notably, HIV the virus that causes AIDS. For more information, visit http://www.ihv.org and follow us on Twitter @IHVmaryland.

About the Global Virus Network (GVN)

The Global Virus Network (GVN) is essential and critical in the preparedness, defense and first research response to emerging, exiting and unidentified viruses that pose a clear and present threat to public health, working in close coordination with established national and international institutions. It is a coalition comprised of eminent human and animal virologists from 59 Centers of Excellence and 11 Affiliates in 33 countries worldwide, working collaboratively to train the next generation, advance knowledge about how to identify and diagnose pandemic viruses, mitigate and control how such viruses spread and make us sick, as well as develop drugs, vaccines and treatments to combat them. No single institution in the world has expertise in all viral areas other than the GVN, which brings together the finest medical virologists to leverage their individual expertise and coalesce global teams of specialists on the scientific challenges, issues and problems posed by pandemic viruses. The GVN is a non-profit 501(c)(3) organization. For more information, please visit http://www.gvn.org. Follow us on Twitter @GlobalVirusNews

SOURCE Global Virus Network

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) – GlobeNewswire

December 21, 2020 07:00 ET | Source: Orchard Therapeutics (Europe) Limited

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please visitwww.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Christine Harrison Vice President, Corporate Affairs +1 202-415-0137 media@orchard-tx.com

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

Excerpt from:
Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire

3D Printing Industry review of the year: February 2020 – 3D Printing Industry

The month of February 2020 saw industry leaders come together for TV SDs 2nd Additive Manufacturing Conference, alongside 3D printing-enabled medical breakthroughs and a landmark delivery of 3D printed parts within the maritime sector.

Read on for the standout developments of February 2020, which involved the likes of Renishaw, Wilhelmsen, Formlabs, COBOD, and Evonik.

TV SDs Additive Manufacturing Conference

February got underway with the 2nd TV SD Additive Manufacturing Conference, held in the heart of Bavaria, which brought together 3D printing experts, industry leaders, and academics to share their thoughts on the implementation of industrial additive manufacturing.

During the conference, standardization and digitization were singled out as the gateways to a fully automated additive manufacturing system in years to come in regards to material testing, quality assurance, part certification, and process chains.

Space-time research expands for AM

Meanwhile, researchers from the Delft University of Technology (TU Delft) developed a method to concurrently optimize 3D printed structures and the fabrication sequence that creates them, specifically in the wire arc additive manufacturing process (WAAM).

Recently awarded the ISSMO/Springer prize from the International Society of Structural and Multidisciplinary Optimization (ISSMO), the research can be explained using the example of a robot building a bridge in a futuristic scenario, where the bridge must maintain its mechanical strength as it is being built in order to support the weight of the robot.

Wilhelmsens landmark maritime delivery

Global maritime industry group Wilhelmsen delivered what it claimed to be a landmark commercial delivery of 3D printed parts to dry bulk shipping company Berge Bulk. The delivery was part of Wilhelmsens Early Adopter Program with additive manufacturing service bureau Ivaldi Group, which was launched in late 2019 and involved the supply of 3D printed spare parts on-demand to ships and other vessels.

This is just the beginning of the journey, and we are quickly expanding our offering, together with our key development partners, enabling our customers to benefit from the savings provided by 3D printing, digital inventory and on-demand localized manufacturing, said Hakon Ellekjaer, head of venture, 3D printing, at Wilhelmsen Ships Services.

Research roadmaps and material exploration

Midway through February, a worldwide collective of researchers from various universities and institutions came together to produce a roadmap for 3D bioprinting, which was published in Biofabrication.

Summarising the current state of bioprinting, including recent advances of the technology, present developments, and challenges, the paper envisioned how the technology could improve in the future. Topics covered in the paper ranged from cell expansion and novel bio-ink development to stem cell printing and organoid-based tissue development, in addition to bioprinting human-scale tissue structures and multicellular engineered living systems.

Elsewhere, research coming out of the Tokyo University of Science explored how water molecules react when they come into contact with the surface of graphene, revealing there is much to learn about the interaction between water and 3D printed materials.

Surface water on carbon nanomaterials such as graphene has attracted much attention because the properties of these materials make them ideal for studying the microscopic structure of surface water, saidProfessor Takahiro Yamamoto, leader of the study.

Advancing dental restorations

SLA 3D printer manufacturer Formlabs announced new materials from German dental 3D printing pioneer BEGO were to be used in Formlabs Form 3B and Form 2 resin printers. Using these systems, dental professionals are now able to 3D print temporary and permanent crowns and bridges directly for patients.

Widely regarded as a specialist in the sector, BEGO has been developing dental-specific 3D printing materials for over two decades. Now, Formlabs printing systems will use BEGOs materials to print permanent single crowns, inlay, onlays, and veneers, and temporary crowns and bridges.

Construction printing: Live

Danish 3D printing construction firm COBOD demonstrated its construction technology by 3D printing the walls of small four houses during the international Bautec construction exhibition in Berlin, between 18-21 February. The demo was completed using the companys BOD2 3D construction printer, and aimed to provide an un-cut example of its technology in action to Bautec visitors.

When you print live, it is not possible to hide anything, explained Henrik Lund-Nielson, COBOD CEO. With this live printing, we are documenting that our technology has the quality, robustness and stability to perform hour after hour, day after day.

Evoniks first 3D printing software tool

Towards the latter stages of February, German specialty chemicals company Evonik launched its first software tool for 3D printing, designed to help manufacturers select the appropriate additive manufacturing process for their desired part.

Developed by Israeli start-up Castor, in which Evonik invested in late 2019, the software aids users in their choices by taking into account the designs geometry, material, and financial analysis.

Renishaw lands Parkinsons study milestone

Rounding off the month on a positive note, UK-based engineering technologies firm Renishaw announced a milestone in a medical study seeking to help sufferers of Parkinsons disease, for which it developed a patented intraparenchymal drug delivery device using 3D printing.

Initial results of the study were promising and indicated predictable and accurate placement of the neuroinfuse device, as well as significant efficacy and safety of both the device and the drug, CDNF, which was developed by pharmaceuticals experts Herantis Pharma.

Renishaw has since concluded an extension to the study, which is currently the only platform to facilitate repeated, intermittent infusions into the parenchyma, and enables patients to receive the infusions in an out-patient setting.

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Featured image showsFabian Meyer-Brtz, head of 3D printing at Peri, showcasing the end result for the printing on February 19, 2020. Photo via COBOD.

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3D Printing Industry review of the year: February 2020 - 3D Printing Industry