On June 22, 2021 AstraZeneca and MSD reported that its Koselugo (selumetinib) has been granted conditional approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above (Press release, AstraZeneca, JUN 22, 2021, View Source [SID1234584201]).

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NF1 is a debilitating genetic condition affecting one in 3,000 individuals worldwide.1,2 In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.3-7

The approval by the European Commission was based on positive results from the SPRINT Stratum 1 Phase II trial sponsored by the National Institute of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). This trial showed Koselugo reduced the size of inoperable tumours in children, reducing pain and improving quality of life.7,8 This is the first approval of a medicine for NF1 PN in the EU and follows the positive recommendation by the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2021. Safety and efficacy data from the SPRINT Phase II trial with longer follow up will be provided as one of the conditions of approval.

Brigitte C. Widemann, MD, Principal Investigator of the SPRINT trial and Chief, NCI Pediatric Oncology Branch, said: "For children with neurofibromatosis type 1, plexiform neurofibromas can grow and develop so significantly that, in some cases, it becomes debilitating. In the SPRINT trial, selumetinib shrank NF1-associated PNs in 66% of patients and showed clinically meaningful improvements in PN-related symptoms."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "As the first medicine approved in the EU for patients with neurofibromatosis type 1, Koselugo has the potential to transform the way plexiform neurofibromas are managed and treated. The SPRINT data showed that Koselugo not only shrank tumours in some children, but also reduced pain and improved their quality of life. This significant milestone was made possible thanks to our research partners, the National Cancer Institute, the Neurofibromatosis Therapeutic Acceleration Program, the Children’s Tumor Foundation, the patient community and every child, parent and doctor involved in the clinical trial."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Before this approval, surgery was the only treatment option for children in the EU with neurofibromatosis type 1 plexiform neurofibromas. This approval marks a significant step forward in addressing the debilitating impact of these tumours."

The SPRINT Stratum 1 Phase II trial showed Koselugo demonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated with Koselugo as twice-daily oral monotherapy.8 ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20% reduction in tumour volume).8 Results were published in The New England Journal of Medicine.7

Koselugo is approved in the US and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PN. Further regulatory submissions are underway. Clinical trials of Koselugo in adult patients with NF1 PN, including an alternative age-appropriate formulation for paediatric patients, are scheduled to begin this year.

NF1
NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called ‘café au lait’ spots). In 30-50% of people, tumours develop on the nerve sheaths.1,3,9,10 These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bladder/bowel dysfunction and disfigurement, as well as having the potential to transform into malignant peripheral nerve sheath tumours.4-7,10 PN begin developing during early childhood, with varying degrees of severity, and can reduce life expectancy by eight to 15 years.3,6,11,12

SPRINT
The SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy.7 This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

Koselugo
Koselugo (selumetinib) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).8 MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.13

Koselugo received US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019 and US Orphan Drug Designation in February 2018. Further orphan designations have been granted in the EU, Japan, Russia, Switzerland, South Korea, Taiwan and Australia.

AstraZeneca and MSD strategic oncology collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor, and Koselugo (selumetinib), a mitogen-activated protein kinase (MEK) inhibitor, for multiple cancer types. Working together, the companies will develop Lynparza and Koselugo in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and Koselugo in combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

On June 21, 2021Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, ("Transgene" or the "Company") reported the launch of a capital increase through the issuance of ordinary shares, with a waiver of preferential subscription rights, via a private placement in the context of an accelerated bookbuilding for an amount of approximately 32 million euros (the "Capital Increase") (Press release, Transgene, JUN 21, 2021, View Source [SID1234621818]).

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The 32-million euros Capital Increase could be increased to approximately 20% of Transgene’s share capital resulting in the issuance of a maximum of 16,768,266 new ordinary shares. The Offering is exclusively directed at certain qualified and institutional investors located in and outside of France. This transaction will benefit from the support of the Company’s historical shareholders. It will extend the Company’s financial visibility until the fourth quarter of 2023.

Funds will be directly used by descending order of strategic priority:
• circa €25m for the finalisation of clinical studies and the obtention of data with TG4001, TG4050, BT-001 and TG6002;
• circa €4m to launch the clinical development of new oncolytic viruses based on the Invir.IO platform and currently undergoing preclinical evaluation;
• for the remainder, to finance, together with operational products of the Company, R&D and operational costs, as well as current cash consumption.

MAIN TERMS OF THE CAPITAL INCREASE The Capital Increase consists of a private placement pursuant to Articles L. 225-136 of the French Commercial Code and L. 411-2 1° of the French Monetary and Financial Code. The transaction will be carried out with a waiver of preferential subscription rights, pursuant to the delegation of authority granted to the Board of Directors under the 22nd resolution of the Combined General Shareholders’ Meeting of May 27, 2020. The offering is reserved in the European Economic Area (including in France), to "qualified investors", as that term is defined in Article 2(e) of Regulation (EU) 2017/1129 of the European Parliament and of the Council of June 14, 2017 as amended (the "Prospectus Regulation"), in the United States of America to "Qualified Institutional Buyers" in accordance with Rule 144A under the U.S. Securities Act of 1933 (the "Securities Act") and outside the United States of America to "institutional investors", in accordance with Regulation S of the Securities Act.

SHAREHOLDERS’ SUBSCRIPTION AND LOCK-UP AGREEMENTS Institut Mérieux, which currently holds 99.5% through its subsidiary TSGH, 60% of the company’s share capital indicated that they intend to place an order of 25 million euros. Furthermore, SITAM Belgium (previously Dassault Belgique Aviation), which hold 4.9% of the company’s share capital, have indicated that they intend to subscribe for new ordinary shares in the Capital Increase in order to maintain their current level of participation in the Company. The total amount of these two orders represent up to approximately 80% of the Offering. Orders of TSGH and SITAM Belgium will be placed "at any price" in order not to interfere with the pricing of the Capital Increase. The press release announcing the results of the Capital Increase will set out the shareholding resulting from the transaction.

This renewed support is a sign of Transgene’s historical shareholders continued confidence in the Company’s strategy and cutting-edge technologies, against a background of the strong interest shown by clinicians in the Company’s highly innovative drug candidates. Independent board members of the Company were called upon to vote on the transaction, and unanimously approved both the principle of the Capital Increase and its terms. Ms Flory, for TSGH, et Mr Habert, for SITAM Belgique, did not take part to the board meeting. TGSH and SITAM Belgique have agreed with the Global Coordinators for the contemplated transaction that they will not sell or transfer its shares of Transgene for a period ending 90 days after the settlement and delivery of the Capital Increase, subject to the usual exceptions. In connection with the Capital Increase, the Company will enter into a lock-up agreement with the Global Coordinators for a period ending 90 days after the settlement and delivery of the Capital Increase, subject to customary exceptions. The Capital increase is the subject of a placing agreement entered into by the Company and the Global Coordinators on June 21, 2021.

The placing agreement may be terminated by the Global Coordinators at any time up to (and including) the settlement date of the Capital Increase which is expected to take place on June 24, 2021, subject to certain customary conditions. Not for release, publication or distribution, directly or indirectly, in or into the United States of America, Canada, Australia or Japan Page 3/5 C1-Public Natixis INDICATIVE TIMETABLE The private placement will be carried out via an accelerated bookbuilding that will determine the number of ordinary shares to be issued and their subscription price. The accelerated bookbuilding process will begin immediately and should be concluded before the opening of the markets on June 22, 2021, subject to acceleration. Transgene will announce the subscription price as well as the number of ordinary shares to be issued in a further press release at the latest on June 22, 2021 and as soon as practicable after the closure of the bookbuilding process. Settlement of the new ordinary shares to be issued in the context of the Capital Increase as well as their admission of the new shares to trading on Euronext Paris are expected to occur on June 24, 2021. The new shares will bear current dividend rights and will be admitted for trading on the regulated market on Euronext Paris under ISIN code: FR0005175080 – TNG. INFORMATION AVAILABLE TO THE PUBLIC The Capital Increase will not be subject to a prospectus approved by the French Financial Market Authority (the "AMF").

Detailed information on Transgene, including its business, results, prospects and related risk factors are described in the Company’s universal registration document filed with the AMF on April 1st, 2021 under number D.21-0248 (the "Universal Registration Document"), which is available together with other regulated information and all press releases of the Company, on Transgene’s website (www.transgene.fr).

Transgene draws the attention of the public to:
• The risk factors presented in the Universal Registration Document ; the materialization of any or all of these risks is likely to have a detrimental effect on the activity, financial situation, or the results of Transgene or on its ability to achieve its objectives.
• The main risks associated with the Capital Increase are the following: o the market price of the Company shares may fluctuate and fall below the subscription price of the new shares
; o due to stock market fluctuations, the volatility and liquidity of the Company shares may vary significantly; o the sale of Company shares may occur on the secondary market, after the capital increase, and have a negative impact on the Company share price
; o regarding the use of the expected proceeds of the issuance within the context of the Capital Increase, the Company has room for maneuver as to the use of the funds raised and could use them in a way that the shareholders may not adhere to or that would not increase the value of their investment in the short term; and o a new market-based call by the Company, after the Capital Increase, could result in further dilution for the investors.

On June 21, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported that CEO Robert Cobuzzi, Jr., Ph.D., will present via webcast at the Life Sciences Investor Forum being held on June 24, 2021 (Press release, Diffusion Pharmaceuticals, JUN 21, 2021, View Source [SID1234586811]).

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Details of Diffusion’s presentation are as follows:

Event: Life Sciences Investor Forum
Date: Thursday, June 24, 2021
Time: 11:30 a.m., EDT
Link: View Source
During the presentation, Dr. Cobuzzi will discuss Diffusion Pharmaceuticals’ business strategy, which focuses on developing Trans Sodium Crocetinate to treat a variety of diseases driven by hypoxemia.

Company Highlights:

Completed enrollment and dosing in the TCOM 200-301 study in March 2021
Announced data from the COVID-19 100-303 study in February 2021
Raised $34.5 million in financing in February 2021.
This will be a live presentation. If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available after the event.

About Life Sciences Investor Forum:

Life Sciences Investor Forum is the leading proprietary investor conference series that provides an interactive forum for Life Sciences companies to meet with and present directly to investors.

A real-time solution for investor engagement, Life Sciences Investor Forum is powered by Intrado Digital Media and specifically designed for more efficient investor access. Replicating the look and feel of on-site investor conferences, Life Sciences Investor Forum combines leading-edge conferencing and investor communications capabilities with a comprehensive global investor audience network.

On June 21, 2021 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported that CEO Robert Cobuzzi, Jr., Ph.D., will present via webcast at the Life Sciences Investor Forum being held on June 24, 2021 (Press release, Diffusion Pharmaceuticals, JUN 21, 2021, View Source [SID1234584504]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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Details of Diffusion’s presentation are as follows:

Event: Life Sciences Investor Forum
Date: Thursday, June 24, 2021
Time: 11:30 a.m., EDT
Link: View Source
During the presentation, Dr. Cobuzzi will discuss Diffusion Pharmaceuticals’ business strategy, which focuses on developing Trans Sodium Crocetinate to treat a variety of diseases driven by hypoxemia.

Company Highlights:

Completed enrollment and dosing in the TCOM 200-301 study in March 2021
Announced data from the COVID-19 100-303 study in February 2021
Raised $34.5 million in financing in February 2021.
This will be a live presentation. If attendees are not able to join the event live on the day of the conference, an archived webcast will also be made available after the event.

About Life Sciences Investor Forum:

Life Sciences Investor Forum is the leading proprietary investor conference series that provides an interactive forum for Life Sciences companies to meet with and present directly to investors.

A real-time solution for investor engagement, Life Sciences Investor Forum is powered by Intrado Digital Media and specifically designed for more efficient investor access. Replicating the look and feel of on-site investor conferences, Life Sciences Investor Forum combines leading-edge conferencing and investor communications capabilities with a comprehensive global investor audience network.

On June 21, 2021 EVERSANA , the pioneer of next generation commercial services to the global life sciences industry, reported a broad expansion of its integrated compliance services across Europe, including leading medical information (MI) operations across 18 European countries with support in 10 local languages (Press release, EVERSANA, JUN 21, 2021, View Source [SID1234584267]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The new European hub joins integrated compliance services across Asia Pacific, North America and Latin America providing global support across time zones and languages. EVERSANA’s expanded compliance services include MI, pharmacovigilance (PV), quality assurance and quality systems, and regulatory affairs, connected by a fully integrated, automated, cloud-based technology platform enabled with artificial intelligence to ensure connectivity, analysis and reporting. Specific MI in-language contact center services include adverse experience reporting, product complaint intake and processing, clinical trials information call centers and medical affairs services.

"We’ve transformed the traditional, transactional approach to medical information into a dynamic, digitally driven operation that navigates local regulatory needs while meeting our clients’ global strategic needs," said Jim Lang, CEO, EVERSANA. "We are uniquely positioned to not only provide MI services but also compliantly activate the power of EVERSANA’s hub, clinical and sales deployment solutions and various outsourced services to propel the commercial success of better, safer products."

The tools and technology are complemented by highly skilled professionals, including PharmDs with extensive medical information experience supporting a wide range of life sciences companies across multiple therapeutic categories, including pharmaceuticals, biologics, medical devices, OTC products and digital therapeutics. The team’s disease category expertise includes but is not limited to oncology, hematology, rare orphan diseases, immunology, dermatology, cardiology, urology, gastroenterology, infectious diseases, nephrology, neurology, pain, respiratory, ophthalmology, endocrinology, pulmonology, hepatology, consumer products and women’s health.

Already serving more than 40 European pharmaceutical and biotechnology clients, EVERSANA globally supports more than 500 life sciences companies; and its rapid international expansion now includes more than 25 global offices and European-based employees working in the United Kingdom, France, Germany, Switzerland, Spain, Ireland, Bulgaria, Poland, Italy and Croatia.

"We are aggressively expanding our European presence to activate niche engagements or full-scale commercial services in and outside the region. The full scale of EVERSANA’s global commercialization services stands ready to support the rich pipeline of products in development in Europe and beyond," said Lang.