Category Archives: Biotech Companies France

Coave Therapeutics Launches coAAV-CSF-01, a Novel CNS-Targeted Gene Therapy Vector for Neurodegenerative and other CNS Diseases

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New breakthrough data from studies with Coave’s ALIGATER™-engineered AAV2-based capsid coAAV-CSF-01 (S01coAAV2) demonstrates improved brain tissue transduction and safety following intra-cerebrospinal fluid administration in non-human primates

Data selected for Late-Breaking Abstract at ASGCT 2025

Paris, France – May 9, 2025 – Coave Therapeutics (“Coave”), a company pioneering the future of genetic medicines, today announced the launch of coAAV-CSF-01, a novel CNS-targeted gene therapy capsid developed using its proprietary ALIGATER™ platform. New breakthrough data from studies evaluating the vector (research code S01coAAV2) have been selected for presentation as a Late-Breaking Abstract at the 2025 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, to be held May 13-17, in New Orleans, LA, USA.

The data highlight the therapeutic potential of coAAV-CSF-01 (S01coAAV2) to transform central nervous system (CNS) gene therapy delivery. In non-human primate (NHP) studies, coAAV-CSF-01 was administered via intra-cerebrospinal fluid (intra-CSF) routes, including intracisternal magna (ICM) and intracerebroventricular (ICV). The results demonstrated higher transgene expression in targeted brain regions and a better safety profile using coAAV-CSF-01 compared to AAV9.

Key findings with coAAV-CSF-01 include:

  • 100-fold higher transgene expression in the cortex and 10,000-fold increase in the hippocampus compared to AAV9 at the same dose
  • Comparable CNS biodistribution and expression at one-fifth the dose of AAV9
  • Significantly reduced peripheral transduction (including strong liver de-targeting) and peripheral nerve safety, addressing key safety concerns in CNS gene therapy

“These exciting new data from coAAV-CSF-01 represent a major step forward in CNS gene therapy,” said Lolita Petit, CSO of Coave Therapeutics. “In non-human primates, the vector achieved robust brain transduction via intra-CSF delivery – a route long viewed as promising but historically constrained by limited efficacy and off-target effects. The enhanced biodistribution and improved safety demonstrated in these studies support its potential in developing new genetic medicines for neurodegenerative and neurodevelopmental CNS disorders.

“Furthermore, the vector was developed using our proprietary ALIGATER™ platform, enabling modular modification of AAV2 and other capsids to optimize tissue targeting and therapeutic performance – potentially opening the door to broader therapeutic applications. Together, the data position coAAV-CSF-01 as a potentially transformative advance in gene therapy delivery.”

coAAV-CSF-01 is part of Coave’s growing portfolio of proprietary AAV capsids engineered to optimize gene delivery in challenging therapeutic areas. The ALIGATER™ platform enables chemical conjugation-based modification of AAV vectors (coAAVs), offering a modular and scalable approach to enhance tissue targeting and transduction efficiency.

Late-Breaking Poster Presentation Details:

  • Title: Enhanced CNS transduction and safety of S01coAAV2 (coAAV-CSF-01) following intra-cerebrospinal fluid (CSF) administration in cynomolgus macaques
  • Session: Late-Breaking Abstracts
  • Date/Time: Thursday May 15, 2025, 4:30 – 6pm CDT
  • Location: New Orleans
  • Presenter: Julien Spatazza, Senior Director, Discovery & Preclinical Research at Coave Therapeutics

Coave’s abstract (#LBA78) can be viewed on ASGCT’s website (https://annualmeeting.asgct.org/).

***

About ALIGATER™
Coave’s proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform is a breakthrough technology addressing key limitations in the delivery of genetic payloads to extra-hepatic tissues, including limited tissue specificity, delivery efficiency and safety. ALIGATER™ enables conjugation of targeting ligands, such as small molecules, peptides, or antibody fragments, on AAV or non-viral vectors, offering superior delivery efficiency, tissue specificity and safety profile for a broad range of diseases. Importantly, the platform streamlines the manufacturing process by avoiding prior AAV capsid modifications. These capabilities will enable Coave to develop best-in-class gene therapies designed for specific indications.

About Coave Therapeutics
Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases.

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit coavetx.com and follow us on LinkedIn.

CONTACTS

Coave Therapeutics
Rodolphe Clerval, CEO
contact@coavetx.com

MEDiSTRAVA
Sylvie Berrebi, Mark Swallow
coavetx@medistrava.com

4Moving Biotech Secures €7.6M France 2030 Grant for Pioneering First-In-Class Disease-Modifying Osteoarthritis Drug

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  • France 2030 validates 4Moving Biotech’s scientific pioneering approach
  • This strategic support positions the 4P004 drug candidate at the forefront of knee osteoarthritis innovation
  • This additional funding accelerates 4P004’s development toward market launch by 2030

LILLE, France & PARIS–(BUSINESS WIRE)–4Moving Biotech (4MB), a spin-off of 4P-Pharma developing the first potential disease-modifying drug (DMOAD) for knee osteoarthritis, announces that it has been awarded €7.6 million in funding under the France 2030 plan to accelerate the clinical development of its lead candidate, 4P004, currently in a Phase 2a trial.

France 2030 is a highly selective and strategic initiative by the French government designed to fast-track the next generation of breakthrough innovations. 4MB’s selection is a significant endorsement, granted through a rigorous, multi-stage evaluation process by leading scientific, clinical, and technological experts.

This recognition highlights the robust scientific foundations of the 4P004 program and its potential to deliver a truly transformative therapy for OA’s unmet medical need and significant socio-economic impact with over 600 million people worldwide, including 10 million in France.

“We are honored by the renewed confidence through the France 2030 program,” said Revital Rattenbach, Co-founder and President of 4MB. “This highly competitive grant is a powerful validation of the scientific strengths of our 4P004 program and its potential to address one of the most pressing unmet medical needs.”

This additional funding will accelerate the 4P004 INFLAM MOTION Phase 2a clinical study, a GLP-1 analog designed to harness anti-inflammatory, analgesic, anti-catabolic, and anabolic effects across all joint tissues. The trial’s primary objective is to evaluate the efficacy of a single intra-articular injection of 4P004 in reducing knee pain while also assessing structural improvements via contrast-enhanced MRI and exploring novel biomarkers for disease progression and delayed need for total knee replacement.

“4MB gains extended momentum through The France 2030 grant marks an important milestone in the 4MB roadmap,” said Luc Boblet, CEO of 4MB. “With our phase 2a already initiated, this funding strengthens the value of our work, from early-stage discovery to clinical stage, and highlights the need to bring transformative therapies for patients suffering from OA”.

With the support of the France 2030 initiative, 4MB is fully equipped to deliver clinical proof-of-concept on time, as a critical inflection point that will unlock the next stage of development and reveal the full potential of 4P004 as a first-in-class DMOAD, creating significant attractiveness for future partnerships.

About 4MB

Incorporated in mid-2020 as a spin-off of 4P-Pharma, 4MB is a clinical stage biotechnology company dedicated to the development of the Disease-Modifying Osteoarthritis Drug (DMOAD). Its mission is to provide a sustainable therapeutic solution to the significant unmet medical need of osteoarthritis. The company is headquartered at the Pasteur Institute in Lille, France.

Website: https://www.4movingbiotech.com/
LinkedIn: https://fr.linkedin.com/company/4moving-biotech

The France 2030 investment plan

France 2030 is unprecedented in its scale: €54 billion is being invested so that our businesses, our schools, our universities and our research organizations fully succeed with their transitions in these strategic sectors. The challenge is to enable them to respond in a competitive way to the world’s future ecological and attractiveness challenges, and to showcase the future champions of our sectors of excellence. France 2030 is defined by two overarching objectives, which will see 50% of expenses given over to the decarbonization of the economy, and 50% to emerging key players, those at the forefront of innovation, without neglecting the environment (in the sense of the “Do No Significant Harm” principle).

France 2030 is being implemented collectively: designed and deployed in consultation with economic, academic, local and European key players to establish strategic guidelines and decisive action. Project holders are invited to submit their entries via the current application process, which is a demanding and selective one, to benefit from state support.

France 2030 is being led by the French General Secretariat for Investment, in charge of France 2030, on behalf of the Prime Minister, and in partnership with the relevant government ministries.

France 2030 is being implemented by the French National Research Agency (ANR), the French Agency for Ecological Transition (ADEME), Bpifrance and the Caisse des Dépôts et consignation (CDC).

More information : france2030.gouv.fr | @SGPI_avenir

About Bpifrance

Bpifrance is the French national investment bank: it finances businesses – at every stage of their development – through loans, guarantees, equity investments and export insurances. Bpifrance also provides extra financial services (training, consultancy) to help entrepreneurs meet their challenges (innovation, export…).

For more information, please visit: https://www.bpifrance.com/ Follow us on Twitter: @Bpifrance – @BpifrancePresse and LinkedIn

The only version of the 4Moving Biotech press release that is legally binding is the one in its original language. Translations must always be compared to the source text, which will establish precedence. The press release text resulting from a translation should not be considered official in any way.

Contacts

Press
Emmanuel Dadjé

Communication Manager

+33 6 30 06 12 13

4Moving Biotech welcomes Luc Boblet as Chief Executive Officer to lead next phase of growth

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LILLE & PARIS, France–(BUSINESS WIRE)–4Moving Biotech (4MB), a clinical-stage subsidiary of 4P-Pharma specializing in immuno-inflammation and osteoarthritis, is pleased to announce the appointment of Luc Boblet as Chief Executive Officer. The company, having successfully completed its Phase I clinical trial and a preparatory in silico Phase 2 analysis, looks to Luc Boblet’s seasoned expertise in biotech entrepreneurship and strategic development to guide the forthcoming stages of clinical proof of concept and early market access.

Luc Boblet, a biotech entrepreneur with two decades of experience, joins 4MB following his tenure at Egle Therapeutics, where his leadership was essential in advancing novel immunotherapies and securing a robust Series A funding backed by a big pharma strategic alliance. His previous roles included founding and leading companies such as H-IMMUNE – acquired by HiFiBIO – and Pathoquest, where he excelled in driving innovation, closing fundraising and forging strategic partnerships as instrumental catalyst of growth.

“Luc’s appointment comes at a turning point for 4Moving Biotech as we have concluded a milestone Phase I trial for our leading drug candidate and are advancing strategic discussions with the FDA, toward potential accelerated approval” said Revital Rattenbach, founder and CEO of 4P-Pharma and co-founder of 4Moving. “His track record in steering biotech ventures and his strategic approach to partnerships will be invaluable as we advance our clinical programs and explore opportunities for pharma co-development and partnership.”

As CEO of 4Moving Biotech, Luc Boblet’s immediate focus will be on overseeing the design and implementation of the development strategy for the upcoming Phase II clinical trials, as well as cultivating partnerships that align with the company’s vision for growth and patient access to innovative treatments.

Luc Boblet, CEO of 4Moving Biotech said: “The opportunity to lead 4Moving Biotech at this juncture is one that is aligned with my commitment to bringing pioneering therapies for untreated severe diseases. The company’s progress in drug development and the potential to significantly improve patient outcomes in osteoarthritis is a strong foundation for future success. I am looking forward to applying my experience in corporate strategy business development to deliver our value proposal and establish meaningful partnerships.”

About 4Moving Biotech

4Moving Biotech, a subsidiary of 4P-Pharma, is dedicated to developing advanced first in class therapies in the field of immuno-inflammation and osteoarthritis. With a commitment to improving patient care, 4MB is actively engaged in the clinical development of novel treatments poised to enhance quality of life for patients worldwide.

Biography of Luc Boblet, CEO of 4Moving Biotech.

Luc Boblet is a serial biotech entrepreneur with 20 years of experience in entrepreneurship, business development, corporate finance and early drug development. Luc is coming from Egle Therapeutics, spin out of Institut Curie dedicated to developing novel Regulatory T-cells based immunotherapies for oncology and autoimmune diseases, he has co-founded in early 2020. As a founding CEO, he built Egle Tx with a vision to become a game changer in the field of Tregs immunomodulation catalyzed by strategic alliance with Takeda Pharmaceutical and backed by 45M€ Series A from a high-quality syndicate of renowned international investors. Before creating Egle Tx, Luc co-founded and ran H-IMMUNE, start-up spun out of French Nuclear Agency (CEA), developing novel anti-check-point immunotherapies against T-effector and Tregs for oncology. He paved H-IMMUNE corporate path through partnerships with Pierre Fabre Laboratories and Norther Biologics and have exited H-IMMUNE through an acquisition by HiFiBIO in 2018. Before co-founding H-IMMUNE, Luc co-founded and ran Pathoquest, a start-up company spun out of Institute Pasteur, which develops disruptive unbiased NGS-based diagnostics in the field of infectious diseases. Under his guidance, Pathoquest raised two VC-backed rounds of financing, closed strategic collaborations and corporate partnerships with leading pharma companies (CERBA Laboratories and Covance), and launched several clinicals trials in France and the U.S.

From 2011 to 2015, he served on the board of directors of the BioAster Technological Research Institute.

He started his career in technology transfer, consulting in finance and business development for toxicogenomics R&D. Luc Boblet holds a biotech engineering degree, master’s degree in Virology and has been trained as a PhD in Virology from University Paris VII.

About 4Moving Biotech

Incorporated in mid 2020 as a spin-off of 4P-Pharma, 4Moving Biotech is a clinical stage biotechnology company dedicated to the development of the Disease-Modifying Osteoarthritis Drug (DMOAD). Its mission is to provide a sustainable therapeutic solution to the significant unmet medical need of osteoarthritis. The company is headquartered at the Pasteur Institute in Lille, France.

Website: https://www.4movingbiotech.com/
LinkedIn: https://fr.linkedin.com/company/4moving-biotech
X: https://twitter.com/4Moving_Biotech

The only version of the 4Moving Biotech press release that is legally binding is the one in its original language. Translations must always be compared to the source text, which will establish precedence. The press release text resulting from a translation should not be considered official in any way.

Contacts

Press:
Emmanuel Dadje

Communication Manager – emmanuel.dadje@4P-Pharma.com
+33 6 30 06 12 13

Median Technologies, a World Leading Oncology Clinical Trial Imaging Services Provider, Becomes Preferred Vendor to Another Top 3 Pharmaceutical Company

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  • Median Technologies has been selected as a preferred clinical trial imaging services provider for another leading global pharmaceutical company.
  • Median Technologies is now preferred provider for two of the Top 3 global pharma companies, based on oncology sales1.
  • The pharmaceutical company has one of the largest clinical trial pipelines in oncology and is a new client for Median.
  • The agreement covers late phase oncology trials for which central image readouts are required.
  • The new agreement highlights Median’s strength as a leading clinical trial imaging provider in the United States.

SOPHIA ANTIPOLIS, France–(BUSINESS WIRE)–Regulatory News:

Median Technologies (FR0011049824, ALMDT, PEA/PME scheme eligible, “Median” or “The Company”) (Paris:ALMDT) announced today that the Company has been selected as preferred vendor by a Top 3 pharmaceutical company, adding another one of the largest global pharmaceutical companies to its client portfolio. Median Technologies is now preferred provider for two of the Top 3 pharma companies, based on oncology sales2.

The Master Service Agreement (MSA) came after an in-depth selection process. The new client has one of the largest oncology pipelines in the world and presents one of the highest R&D to revenue ratios in the pharmaceutical industry3. Median will manage central image reads for late phase clinical trials in the client’s oncology pipeline. The MSA is a recognition of Median’s performance, quality, and competitiveness for imaging services worldwide.

Fredrik Brag, CEO and Founder of Median Technologies said: “Median’s’ iCRO services are best-in-class and recognized as such worldwide. The agreement will ensure stable future bookings and revenues growth for Median, while the Top 3 pharmaceutical company will benefit from improved operational execution using the best technology available in the market. Our recognized expertise in AI applied to medical images, has been a strong technology differentiator during the diverse phases of the negotiation; it represents a unique asset compared to the standard offering of our competitors as well as a major trigger for the acceleration of growth opportunities for our Company, in the era of precision medicine.”

Nicolas Dano, COO & CCO iCRO, added: “The new preferred vendor agreement significantly strengthens our position in the United States. It is the result of our investments to deliver top-tier best-in-class imaging services, our commitment to achieving customer satisfaction, and the wealth of experience acquired in supporting sponsors with their regulatory filings. With the combination of our two offerings, iSee® for central image reads, and Imaging Lab, iCRO’s unique AI-powered imaging offering, we cover a very wide and unique spectrum of imaging services for the pharma industry, from standard image reads to advanced imaging services using cutting edge AI-based technologies. We are proud to support the pharma industry in bringing the next generation of life-saving oncology drugs to market”.

About Median Technologies: Pioneering in innovative imaging solutions and services, Median Technologies harnesses cutting-edge AI to elevate the accuracy of early cancer diagnoses and cancer treatments. Median’s offerings, including iCRO for medical image analysis and management in oncology trials and eyonis™, AI/ML tech-based suite of software as medical devices (SaMD), empower biopharmaceutical entities and clinicians to advance patient care and expedite novel therapies. The French-based company, with a presence in the U.S. and China, is listed on the Euronext Growth stock exchange (ISIN: FR0011049824, ticker: ALMDT). Median is eligible for the French SME equity savings plan scheme (PEA-PME). For more information: www.mediantechnologies.com

______________________________

1 Arjun Murthy – Top 15 biopharma companies by Oncology Sales in 2023

2 Arjun Murthy – Top 15 biopharma companies by Oncology Sales in 2023

3 Update: April 30, 2024: https://www.drugdiscoverytrends.com/top-pharma-companies-2023-rd-spend/

Contacts

Median Technologies
Emmanuelle Leygues

Head of Corporate Marketing & Financial Communications

+33 6 10 93 58 88

emmanuelle.leygues@mediantechnologies.com

Press – ALIZE RP
Caroline Carmagnol

+33 6 64 18 99 59

median@alizerp.com

Investors – ACTIFIN
Ghislaine Gasparetto

+33 6 21 10 49 24

ghislaine.gasparetto@seitosei-actifin.com

EGLE Therapeutics Strengthens its Leadership Team with the Appointment of Pejvack Motlagh, MD, MSc, as Chief Medical Officer and its Board of Directors with Appointment of John Celebi, MBA, as Independent Board Member

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EGLE Therapeutics, a clinical-stage biotechnology company uniquely positioned to advance the next generation of regulatory T cell-focused therapies for oncology and auto-immunity, announced today the appointment of Pejvack Motlagh MD, MSc, as Chief Medical Officer and John Celebi, as Independent Board Member.

“We are excited to welcome Pejvack and John at Egle and to benefit from their strong experience.

The appointment of Dr Motlagh is critical for Egle, as the company is moving to clinical development of assets in oncology and auto-immunity. Pejvack will impact the future development of the rich pipeline at Egle, based on the modulation of regulatory T cells.

As we advance toward the clinical development of EGL-001, John’s expertise in business development in the field of oncology is instrumental for us to successfully execute on our strategic priorities.” said Vincent Brichard, M.D., EGLE’s Interim CEO and Board member.

“I am excited to join Egle Therapeutics to translate into the clinic the company’s groundbreaking and unique science, whose innovative and versatile regulatory T-cell focused platform, a key component of the immune system, has a tremendous potential to change the treatment paradigm for the many patients with high unmet medical needs in oncology and auto-immunity” said Pejvack Motlagh, MD, MSc, CMO at Egle Therapeutics. As CMO, Pejvack will drive the clinical and medical strategy and development of our Company in the years to come.

“This is a transformational period for Egle Therapeutics as the company further strengthens its team and advances toward the clinic a series of highly novel candidates aimed at arming or disarming Treg function to achieve the desired therapeutic outcome. I look forward to collaborating with this talented Board and management team as we advance Egle’s compelling pipeline for patients with unmet need,” said John Celebi, Independent Board Member at Egle Therapeutics.

Pejvack Motlagh, MD, MSc, a seasoned clinical development executive who has previously held overall responsibility for candidate development from first in human through registrational studies, brings to Egle Therapeutics the appropriate expertise and successful drug development experience. As Chief Medical Officer, he will be responsible for advancing Egle’s pipeline towards the clinic and notably EGL-001 (a Treg-selective anti-CTLA4-IL-2m) in oncology and EGL-003 (a non-targeted IL-2 Treg engager) in auto-immune diseases. Prior to Egle Therapeutics, Dr. Motlagh was Chief Medical Officer at Mablink, an ADC biotech company, where through the establishment of a differentiating clinical development plan he contributed to the value creation leading to an acquisition by Eli Lilly. Previously, he held global positions at leading oncology companies such as GSK, Bristol-Myers Squibb and AstraZeneca, where he was instrumental in the transition of several compounds towards the late clinical development phases of now approved drugs. At Boehringer Ingelheim, he oversaw, managed and prioritized their whole IO portfolio.

John Celebi, MBA, brings over 25 years of experience building innovative entrepreneurial biotechnology companies. He currently serves as President and CEO of Sensei Biotherapeutics (NASDAQ: SNSE), a biotechnology company focused on the discovery and development of novel immunotherapies for cancer patients with unmet needs. Previously, Mr. Celebi served as the Chief Operating Officer of X4 Pharmaceuticals where he established and oversaw the company’s oncology business strategy. He also served as Chief Business Officer of Igenica Biotherapeutics, Inc., an immunotherapy company formed by The Column Group, 5AM Ventures, Orbimed, and Third Rock Ventures, where he established key academic and industry relationships, including with MedImmune. Mr. Celebi has extensive transactional and alliance management experience, having served as Vice President of Business Development, New Product Planning and Alliance Management at ArQule, Inc., where he played a central role in the formation of alliances with Roche, Daiichi-Sankyo, and Kyowa Hakko Kirin. Mr. Celebi was an early employee at Tularik, Inc., where he conducted drug discovery and basic research. Mr. Celebi received an MBA from Carnegie Mellon University and a B.S. in biophysics from the University of California, San Diego.

About Egle Therapeutics SAS (Egle)

Egle Therapeutics is a biotechnology company focused on developing immunotherapies targeting suppressive regulatory T cells. Egle is leveraging a proprietary discovery platform to unveil novel Treg specific targets and to develop innovative Treg-focused drug candidates for oncology and autoimmune diseases. Egle aspires to advance toward the clinic its most advanced drug candidates, EGL-001 (a Treg-selective anti-CTLA4-IL-2m) and EGL-003 (non-targeted IL-2 Treg engager), which are currently developed in IND-enabling studies.

Find out more at www.egle-tx.com.

Contacts

contact@egle-tx.com / 0033 (0)1 86 64 08 57

investor.relations@egle-tx.com / 0033 (0)1 86 64 08 57

Sanofi and Novavax announce co-exclusive licensing agreement to co-commercialize COVID-19 vaccine and develop novel flu-COVID-19 combination vaccines

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Sanofi and Novavax announce co-exclusive licensing agreement to co-commercialize COVID-19 vaccine and develop novel flu-COVID-19 combination vaccines

  • Agreement provides patients with broader access to a protein-based non-mRNA adjuvanted COVID-19 vaccine through combined commercial strength, from 2025 onwards
  • Accelerates potential for development of a novel flu-COVID-19 combination product based on authorized vaccines with demonstrated efficacy and tolerability, potentially offering patients enhanced convenience and protection

Paris and Gaithersburg, Md., United States. May 10, 2024. As part of Sanofi’s commitment to developing a diverse portfolio of best-in-class vaccines, the company has entered into a co-exclusive licensing agreement with Novavax, a biotechnology company headquartered in Maryland, US.

The terms of the agreement include: a co-exclusive license to co-commercialize Novavax’s current stand-alone adjuvanted COVID-19 vaccine worldwide (except in countries with existing Advance Purchase Agreements and in India, Japan, and South Korea where Novavax has existing partnership agreements); a sole license to Novavax’s adjuvanted COVID-19 vaccine for use in combination with Sanofi’s flu vaccines; and a non-exclusive license to use the Matrix-M adjuvant in vaccine products. In addition, Sanofi will take a minority (<5%) equity investment in Novavax.

Jean-Francois Toussaint
Global Head of Vaccines R&D
“With flu and COVID-19 hospital admission rates now closely mirroring each other, we have an opportunity to develop non-mRNA flu-COVID-19 combination vaccines offering patients both enhanced convenience and protection against two serious respiratory viruses. We’re excited by the prospect of combining Novavax’s adjuvanted COVID-19 vaccine that has shown high efficacy and favorable tolerability, with our rich portfolio of differentiated flu vaccines that have demonstrated superior protection against flu and its serious complications. Improved tolerability and thermostability, without compromise on efficacy, are what regulators, recommending bodies, and patients will demand.”

John Jacobs
CEO, Novavax

“This collaboration is important for Novavax and for global public health. Our new partnership combines Novavax’s proprietary recombinant protein and nanoparticle technologies, Matrix™ adjuvant, and R&D expertise with Sanofi’s world-class leadership in launching and commercializing innovative vaccines. Together, we can broaden access to both our COVID-19 vaccine and our adjuvant to ensure more individuals can benefit from the protection vaccines can provide. Novavax is now in a stronger position to refocus our efforts on leveraging our technology platform and novel adjuvant in research and development and pipeline expansion to help advance our mission of developing life-saving vaccines to fight infectious diseases.” 

Under the terms of the licensing agreement:

  • Novavax will receive an upfront payment of $500 million and up to $700 million in development, regulatory and launch milestones, up to $1.2 billion in total.
  • Starting in 2025, Sanofi will book sales of Novavax’s adjuvanted COVID-19 vaccine and will support certain R&D, regulatory, and commercial expenses.
  • Novavax will receive tiered double-digit percentage royalty payments on sales by Sanofi of COVID-19 vaccines and flu-COVID-19 combination vaccines.
  • Sanofi will be solely responsible for development and commercialization of any novel flu-COVID-19 combination vaccine containing a Sanofi flu vaccine.
  • Outside of the collaboration, each party may develop and commercialize their own flu-COVID-19 vaccines and adjuvanted products at their own cost.
  • Novavax is entitled to additional launch and sales milestones opportunities of up to $200 million plus mid-single digit royalties for each additional Sanofi vaccine product developed under a non-exclusive license with Novavax’s Matrix-M adjuvant technology.
  • In addition, Sanofi will take a minority (<5%) equity investment in Novavax.

About Novavax
Novavax, Inc. (Nasdaq: NVAX) promotes improved health by discovering, developing and commercializing innovative vaccines to help protect against serious infectious diseases. Novavax, a global company based in Gaithersburg, Md., U.S., offers a differentiated vaccine platform that combines a recombinant protein approach, innovative nanoparticle technology and Novavax’s patented Matrix-M adjuvant to enhance the immune response. The Company’s portfolio includes its COVID-19 vaccine, and its pipeline includes CIC and stand-alone influenza vaccine candidates. In addition, Novavax’s adjuvant is included in the University of Oxford and Serum Institute of India’s R21/Matrix-M malaria vaccine. Please visit novavax.com and LinkedIn for more.

About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

Media Relations
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com
Sally Bain | + 1 617 834 6026 | sally.bain@sanofi.com
Evan Berland | + 1 215 432 0234 | evan.berland@sanofi.com

Nicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.com

Investor Relations
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com
Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com
Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com
Tarik Elgoutni| + 1 617 710 3587 | tarik.elgoutni@sanofi.com
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com

Sanofi Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2023. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

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EyeDNA Therapeutics Announces Positive 24-month Data from Ongoing Phase I/II Trial of HORA-PDE6b Gene Therapy in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

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Clinically meaningful benefit in visual functions and good safety profile of HORA-PDE6b is confirmed at 24-month follow-up

Phase I/II results will be discussed with US and European health authorities to define optimal path to making HORA-PDE6b available to patients with PDE6b Retinitis Pigmentosa

Paris, France, May 7, 2024 – eyeDNA Therapeutics (‘eyeDNA’), a newly created subsidiary of Coave Therapeutics (‘Coave’), a genetic medicine company focused on developing life-changing therapies, today announces positive 24-month follow-up results from its Phase I/II study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b, its investigational gene therapy for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6b gene (PDE6b RP). These data were reported during an oral presentation* on May 6, at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, US.

The positive 24-month follow-up data presented confirm results from the previous interim analysis of the trial conducted at the 12-month follow-up point and support preparation for a registrational trial for HORA-PDE6b in PDE6b RP patients. Further discussions with health authorities in the US and Europe are planned to define the optimal path to making HORA-PDE6b available to PDE6b RP patients.

To date, HORA-PDE6b has been administered in 17 evaluable patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses in four consecutive cohorts. The treatment was administered in the more affected eye while the other eye served as an untreated control.

In a subgroup of clinical interest of six patients receiving the high dose, with less advanced disease (Best Corrected Visual Acuity (BCVA) score ≤75 ETDRS letters [ ≤ 20/32 Snellen equivalent], Goldmann Visual Field (GVF) ≥ 10 degrees), positive efficacy results were reported at 24 months on the BCVA and the GVF outcomes. The BCVA mean change from baseline increased by +0.09 LogMar in the untreated eye, while acuity in the treated eye was stabilized (+0.02 LogMar). From baseline, the mean reduction of the GVF was over 300 deg² superior in the untreated eyes compared to the treated eye.

Interestingly, long-term data available from patients from the low dose group (n=7) followed up over a five-year period found that the BCVA of the untreated eyes consistently declined (increase of 0.05 to 0.08 LogMAR/year from the second year), which is in line with the natural history of the disease. Meanwhile, the mean change from baseline of BCVA of the treated eyes in the same group is stabilized (between +0.03 and +0.06 LogMAR over the same follow-up period). The difference of BCVA mean change from baseline between the treated eyes and the untreated eyes at five years is 0.25 LogMAR (12 Letters).

Furthermore, the full-field stimulation test (FFST) in blue light assessing rod function continues to show an improvement of the light perception threshold in favor of the treated eyes, which is considered clinically meaningful (improvement of almost six decibels). The interesting positive trend on the retinal anatomical evaluation by Optical Coherence Tomography (OCT; Ellipsoid Zone horizontal length) observed at 12-month follow-up on the subgroup of clinical interest is maintained after 24 months.

Following the 24-month study period, both doses were well tolerated (n=17). Five ocular Serious Adverse Events (SAEs) occurred including two resolved SAEs possibly related to HORA-PDE6b (one chorioretinitis and one reduced visual acuity). Patients did not receive preventive oral corticosteroids.

An additional cohort of four to six younger patients aged 13-17 years old with a GVF at baseline ≥ 20 degrees in each meridian and at an earlier disease stage is ongoing with three patients enrolled.

“The highly encouraging safety and efficacy data observed in patients two years after treatment with HORA-PDE6b continue to support our view that this novel gene therapy could provide an important clinical benefit for PDE6b RP patients. These data will support our discussions with regulators to determine the optimal route for getting HORA-PDE6b to patients,” said Rodolphe Clerval, Chief Executive Officer. “At the same time, we continue to evaluate HORA-PDE6 in an expansion cohort of younger patients with less advanced disease, for whom treatment with HORA-PDE6b could have an even greater therapeutic impact.”

“PDE6b retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness. These two-year safety and promising efficacy results are of great medical interest and could represent a significant step towards providing an effective treatment for patients with this devastating disease,” commented Dr. Jean-Baptiste Ducloyer, MD, Nantes University Department of Ophthalmology.

*Abstract 2134:

JB Ducloyer, et al. 12-month Safety and Efficacy Evaluation of HORA-PDE6b, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation

***

About eyeDNA Therapeutics and HORA-PDE6b

eyeDNA Therapeutics, a wholly owned subsidiary of Coave Therapeutics, is a clinical-stage gene therapy company, focused on developing life-changing therapeutics for inherited retinal disorders. Our lead program HORA-PDE6b, an AAV5-based gene replacement therapy, is being evaluated in a Phase I/II trial for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene (PDE6b RP) (NCT03328130).

eyeDNA and Théa Open Innovation (‘TOI’) are partners for the development and commercialization of HORA-PDE6b. eyeDNA is responsible for the global development of HORA-PDE6b and retains commercial rights to the product in the US, Japan, South Korea, China and other territories outside Europe. In Europe and certain other countries, HORA-PDE6b is being co-developed by Coave and TOI under a license and development agreement with exclusive rights granted to TOI to commercialize HORA-PDE6b in these territories.

About Coave Therapeutics

At Coave Therapeutics, we are leading the transition of genetic medicine from rare to prevalent conditions, starting with neurodegenerative and eye diseases. Our proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform introduces chemical modifications onto AAV capsids or Lipid Nanoparticles (LNPs) to overcome the limitations of current vectors on efficacy, safety, and manufacturability.

With low doses and optimized routes of administration, our conjugated vectors have demonstrated markedly improved transduction and biodistribution in the central nervous system and the eye across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent neurodegenerative and ocular diseases – including genetically and non-genetically defined indications.

Coave recently created its subsidiary eyeDNA Therapeutics to focus on the development – up to the marketing authorization application – of its unique gene therapy HORA-PDE6b for the treatment of inherited retinal diseases caused by mutations in the human PDE6b gene.

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit https://coavetx.com/ and follow us on LinkedIn.

CONTACTS
eyeDNA Therapeutics and Coave Therapeutics
Rodolphe Clerval, CEO
contact@coavetx.com

MEDiSTRAVA
Sylvie Berrebi, Leila Adlam, Mark Swallow
coavetx@medistrava.com

Medincell Enters Into Strategic Co-development and Licensing Agreement with AbbVie to Develop Next-generation Long-acting Injectable Therapies

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Medincell will receive an upfront payment of $35 million and is eligible for up to $1.9 billion in potential development and commercial milestones, plus royalties on worldwide sales.

Medincell and AbbVie will co-develop, and AbbVie will commercialize up to six cutting-edge long-acting injectables (LAI).

The first LAI program candidate has been selected and formulation activities are underway.

This pivotal alliance leverages Medincell’s commercial-stage LAI technology and development know-how, and AbbVie’s extensive clinical development and commercialization expertise, to deliver innovative therapeutic solutions to patients globally.

MONTPELLIER, France–(BUSINESS WIRE)–Medincell (Paris:MEDCL) today announced a collaboration with AbbVie to co-develop and commercialize up to six therapeutic products across multiple therapeutic areas and indications. Medincell will use its commercial-stage long-acting injectable technology platform to formulate innovative therapies. Medincell will conduct formulation activities and preclinical studies, including supportive CMC work to advance candidates into clinical trials. AbbVie will finance and conduct the clinical development for each program and will be responsible for regulatory approval, manufacturing, and commercialization.

Christophe Douat, Medincell’s CEO said: “This will be an exciting partnership with one of the most innovative and successful pharmaceutical companies. Medincell has entered a new period of growth following the FDA approval of the first product using our technology in April 2023. The full potential of long-acting injectable therapies is getting increasingly recognized.”

Sébastien Enault, Medincell’s Chief Business Officer added: “Our business development is accelerating following FDA approval of our first product. Our technology can help harvest the full potential of many known or yet untapped drugs. We are ready to make this exciting partnership very successful.”

Under the terms of the co-development and licensing agreement covering up to 6 programs, Medincell will receive a $35 million upfront payment and is eligible to receive up to $1.9 billion in development and commercial milestones ($315 million for each program). Medincell is also eligible to receive mid-single to low-double-digit royalties on net sales.

About Medincell

Medincell is a clinical- and commercial-stage biopharmaceutical licensing company developing long-acting injectable drugs in many therapeutic areas. Our innovative treatments aim to guarantee compliance with medical prescriptions, to improve the effectiveness and accessibility of medicines, and to reduce their environmental footprint. They combine active pharmaceutical ingredients with our proprietary BEPO® technology which controls the delivery of a drug at a therapeutic level for several days, weeks or months from the subcutaneous or local injection of a simple deposit of a few millimeters, entirely bioresorbable. The first treatment based on BEPO® technology, intended for the treatment of schizophrenia, was approved by the FDA in April 2023, and is now distributed in the United States by Teva under the name UZEDY® (BEPO® technology is licensed to Teva under the name SteadyTeq™). We collaborate with leading pharmaceutical companies and foundations to improve global health through new treatment options. Based in Montpellier, Medincell currently employs more than 140 people representing more than 25 different nationalities.

UZED® and SteadyTeq™ are trademarks of Teva Pharmaceuticals

www.medincell.com

Contacts

David Heuzé
Head of Corporate and Financial Communications, and ESG

david.heuze@medincell.com / +33 (0)6 83 25 21 86

Grace Kim
Head of US Financial Strategy & IR

grace.kim@medincell.com / +1 (646) 991-4023

Nicolas Mérigeau
Media Relations

medincell@newcap.eu / +33 (0)1 44 71 94 94

Louis-Victor Delouvrier/Alban Dufumier
Investor Relations France

medincell@newcap.eu / +33 (0)1 44 71 94 94

MaaT Pharma Announces Positive Review from the DSMB on the Ongoing Phase 1 Clinical Trial Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS)

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  • Independent Data Safety and Monitoring Board (DSMB) recommended that the trial proceeds as planned without modifications.
  • MaaT033 has shown a good safety profile and was generally well tolerated in the first 8 patients with ALS treated with MaaT033 used in a chronic setting.

    • MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival of patients with cancer, today announced that the DSMB reviewed safety data in the first 8 patients with Amyotrophic Lateral Sclerosis (ALS) treated with MaaT033 in the IASO clinical trial. The DSMB recommended that the trial continue without modifications.

    The DSMB, composed of 4 independent experts, including an ALS patient association representative, concluded that safety was good. More precisely, it should be noted that no serious or severe adverse events were observed, and no infectious events could be related to MaaT033. The preliminary results reinforce confidence in the safety of MaaT033, a drug candidate produced by combining the microbiota from multiple donors using a “pooling” process.

    MaaT033 is currently evaluated, in a chronic setting, in a Phase 1b pilot study (NCT05889572) in ALS (also known as Lou Gehrig’s disease in the US and Charcot’s disease in French-speaking countries). The Company has developed the clinical trial with the French academic experts FILSLAN/ ACT4ALS-MND and in collaboration with the French patients’ association Tous en Selles contre la SLA. Data readout is now expected in early H2 2024. MaaT033 is also being evaluated in the Phase 2b trial PHOEBUS (NCT05762211), the largest one to date in Europe for a microbiome therapy in oncology, dedicated to improving survival of patients with blood cancers receiving allogeneic hematopoietic stem cell transplantation (HSCT).

    About MaaT033

    MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem TherapyTM containing anti-inflammatory ButycoreTM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA).

    About MaaT Pharma

    MaaT Pharma, a clinical stage biotechnology company, has established a complete approach to restoring patient-microbiome symbiosis in oncology. Committed to treating cancer and graft-versus-host disease (GvHD), a serious complication of allogeneic stem cell transplantation, MaaT Pharma launched, in March 2022, an open-label, single arm, phase 3 clinical trial in patients with acute GvHD (aGvHD), following the achievement of its proof of concept in a phase 2 trial. Its powerful discovery and analysis platform, gutPrint®, enables the identification of novel disease targets, evaluation of drug candidates, and identification of biomarkers for microbiome-related conditions. The company’s Microbiome Ecosystem Therapies are produced through a standardized cGMP manufacturing and quality control process to safely deliver the full diversity of the microbiome, in liquid and oral formulations. MaaT Pharma benefits from the commitment of world-leading scientists and established relationships with regulators to support the integration of the use of microbiome therapies in clinical practice. MaaT Pharma is listed on Euronext Paris (ticker: MAAT).

    Forward-looking Statements

    All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim”, “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.

    Contacts

    MaaT Pharma – Investor Relations
    Guilhaume DEBROAS, Ph.D.

    Head of Investor Relations

    +33 6 16 48 92 50

    invest@maat-pharma.com

    MaaT Pharma – Media Relations
    Pauline RICHAUD

    Senior PR & Corporate Communications Manager

    +33 6 14 06 45 92

    media@maat-pharma.com

    Trophic Communications
    Stephanie MAY or

    Priscillia PERRIN

    +49 171 185 56 82

    maat@trophic.eu

    Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

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    Autophagy is a catabolic process which degrades a cellular own component through the lysosomal machinery. The lysosome, at the heart of the autophagy sytem is important in various processes (cancer, infection…).

    Genoscience Pharma - new approaches disrupting cancer cell lysosomal functions

    In Cancer, they are required in tumor cells for cellular adhesion, motility and signaling, exocytosis, angiogenesis and overall survival, growth, aggressiveness, metastatic potential and drug resistance. Because of their high metabolic rates, rapidly dividing and invasive cancer cells require increased new biomass production to survive. The lysosome is also important for adaptation to nutrient stress as it contains hydrolytic enzymes that play a major role in the degradation of intracellular macromolecules and catabolic (such as autophagy) and anabolic growth. This busy lysosomal behavior leads to alterations in lysosomal structure and function, which, paradoxically, renders cancer cells more sensitive to lysosomal destabilization. In addition, lysosomal enzyme activity is elevated in many tumors compared to adjacent normal tissue, and several reports suggest that lysosomes in tumor cells are more fragile than normal lysosomes. Therefore, lysosome seems to be a target of interest in the fight against cancers. Targeting lysosomes triggers apoptotic and lysosomal cell death pathways.

    Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

    In virology, It has been shown that autophagy is activated during virus and bacterial infection and that some viruses can use the autophagy system to facilitate their own replication . Some viruses, such as Coronaviruses are single stranded, positive sense RNA viruses, which induce the rearrangement of cellular membranes upon infection of a host cell. This provides the virus with a platform for the assembly of viral replication complexes, improving efficiency of RNA synthesis. Genoscience Pharma focuses on new approaches disrupting cancer cell lysosomal functions.

    Genoscience Pharma was founded in 2001 by Pr Philippe Halfon, a world renowned medical expert on viral diseases, especially on Human Immunodeficient Virus (HIV) and Hepatitis C Virus (HCV). The company was initially focused on the development of anti-HCV agents. Two protease inhibitors were thus developed and out-licensed to BioLineRX. A new scientific direction was taken in 2012 after the discovery of a new chemical family: autophagy inhibitors. Following promising preliminary in vitro and in vivo results from these small molecules, including against cancer stem cells, Genoscience Pharma has decided to take the opportunity to make the difference in Oncology, especially in cancers where medical needs are still unmet. Now, Genoscience Pharma is a clinical stage biopharmaceutical company focused on translating novel scientific insights into medicines for patients with cancer.

    Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

    More info: https://www.genosciencepharma.com/

    Keywords : lysosomal functions , cancer, oncology , Genoscience Pharma , lysosomes , infectious diseases  , virus , Covid19, leukemia , pancreatic cancer , hepatocarcinoma , small molecules