UroMems Reaches Significant Milestone: Successful Results in Clinical Feasibility Study of UroActive™ Smart Implant for Stress Urinary Incontinence Treatment
UroMems Reaches Significant Milestone: Successful Results in Clinical Feasibility Study of UroActive™ Smart Implant for Stress Urinary Incontinence Treatment
Successful primary and secondary results demonstrate proof of feasibility in male patients, paving way for launch of large-scale pivotal clinical study in the U.S. and Europe
GRENOBLE, France and MINNEAPOLIS, Dec. 13, 2023 /PRNewswire/ — UroMems, a global company developing innovative, mechatronics technology to treat stress urinary incontinence (SUI), announced today it has reached a significant milestone: the complete treatment cohort in the first-of-its-kind clinical feasibility study has successfully reached the six-month primary endpoints.
The feasibility assessment of the UroActive System was completed through a prospective multicenter clinical study. UroActive is the first smart automated artificial urinary sphincter (AUS) to treat SUI, and the only one to reach this critical milestone. The results of this initial clinical study support design and implementation of UroMems’ pivotal SUI trial in Europe and the U.S. All six men are now implanted for at least seven months and up to fifteen months, with their devices operating as expected and no need for revision nor explant. In addition, extremely positive follow-up was received on secondary outcomes measures, including leak rate values and patient quality of life questionnaires.
UroMems received very positive feedback from all patients participating in the study cohort. “You have changed my life,” stated one of the study participants. “I can do all activities again, without stress, without anxiety!”
“We’re so pleased to see that our expectations about our device’s performance were met or even exceeded and delighted to successfully treat and receive such high praise from patients who had been suffering from SUI for years,” said Professor Pierre Mozer, UroMems chief medical officer and co-founder. “The results of this study will allow us to prepare our pivotal study which will be a major step in the development of UroActive.”
UroActive is the first smart active implant that treats SUI, powered by a MyoElectroMechanical System (MEMS). This innovative system is placed around the urethral duct and is controlled based on the patient’s activity, without the need for manual adjustments, intending to provide patients with ease of use and a better quality of life than current options.
“The very compelling results of this first-in-man clinical study demonstrate the high potential of our technology and pave the way for larger clinical trials that will allow us to demonstrate all the benefits we are expecting to offer patients suffering from debilitating SUI,” said Hamid Lamraoui, UroMems chief executive officer and co-founder. “We could not have reached this important milestone without the enthusiastic participation of the men in this initial study cohort. We’re extremely grateful to them for being a vital part in bringing this potentially revolutionary treatment to market.”
SUI, or involuntary urinary leakage, affects an estimated 40 million Americans and 90 million Europeans, and occurs when the pressure in the bladder exceeds that of the muscle (the sphincter) around the urethra, caused by activities involving high intra-abdominal pressure, like coughing, laughing and exercising. SUI significantly impacts quality of life, as it can be debilitating, and often leads to depression, low self-esteem and social stigma.
About UroActive
UroActive is an active implantable electronic artificial urinary sphincter that is being developed to compensate for sphincter insufficiency in patients, both men and women, with SUI. It is based on a unique bionic platform using embedded smart, digital and robotic systems which, based on data collected from a patient, create a treatment algorithm that is specific for each patient’s needs. The UroMems technology platform is protected by more than 120 patents and is designed to overcome the limitations of current solutions by optimizing safety and performance, patient experience and surgeon convenience. STeP participation does not imply product authorization. UroActive has not received marketing authorization from the FDA and is not available for sale in the United States or in the EU.
For more information, please visit www.uromems.com.
Media Contact:
Shelli Lissick
651-276-6922
SOURCE UroMems
Nature Medicine Publication Highlights Potential for ONWARD® ARC Therapy™ to Improve Mobility After Parkinson’s Disease
Nature Medicine Publication Highlights Potential for ONWARD® ARC Therapy™ to Improve Mobility After Parkinson’s Disease
Therapy to be supported by newly awarded $1M grant from Michael J. Fox Foundation
EINDHOVEN, the Netherlands — November 06, 2023 — ONWARD Medical N.V. (Euronext: ONWD), the medical technology company creating innovative spinal cord stimulation therapies to restore movement, function, and independence in people with spinal cord injury (SCI), today announces a publication in Nature Medicine using investigational ONWARD ARC Therapy to address gait challenges related to Parkinson’s disease.
The study participant described in the Nature Medicine publication has been living with Parkinson’s disease for nearly three decades. He has a severe gait disorder that has not responded to conventional therapies.
“I could hardly walk without frequent falls, several times a day. In certain situations, like getting into an elevator, I would stomp, freeze as they say,” said the study participant in a press release issued by Switzerland’s Lausanne University Hospital (CHUV).
In 2021, researchers from the Swiss Federal Institute of Technology (EPFL) and CHUV investigated the possibility that ONWARD ARC Therapy (precise, targeted electrical stimulation of the spinal cord) could address common side effects of Parkinson’s disease that negatively impact mobility. The team collaborated with Dr. Erwan Bezard, a renowned neuroscientist from France’s National Institute of Health and Medical Research (INSERM).
After the introduction of ARC Therapy and benefitting from a few weeks of rehabilitation, the study participant was able to walk without previously noticeable gait interruptions. Today, he uses ARC Therapy eight hours per day.
“I turn on the stimulation in the morning and turn it off in the evening. It allows me to walk better, to stabilize myself. Even stairs don’t scare me anymore. Every Sunday I go to the lake, and I walk about six kilometers. It’s awesome,” said the participant in the CHUV release.
“The breakthrough reported in Nature Medicine shows the remarkable potential to use the same technology platform and therapy we are developing for spinal cord injury to also address mobilitychallenges stemming from Parkinson’s disease,” says Dave Marver, CEO of ONWARD.
“It is impressive to see that by electrically stimulating the spinal cord the same way we have done in paraplegic patients, we may be able to address gait disorders due to Parkinson’s disease,” adds neurosurgeon Jocelyne Bloch, co-director with Professor G. Courtine of ONWARD research partner .NeuroRestore.
.NeuroRestore was awarded a $1 million grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to implant the ARC-IM System and investigate the effect of ARC Therapy in six additional participants with Parkinson’s disease. This study will assist ONWARD in determining whether to conduct additional clinical trials and potentially commercialize ARC Therapy in the future for those living with Parkinson’s disease.
“MJFF is committed to fulfilling the unmet needs of people living with Parkinson’s disease by ensuring the development of improved therapies,” said Katharina Klapper, Director of Clinical Research, MJFF. “The Foundation is pleased to award a grant for scientists at EPFL and ONWARD Medical to investigate the effects of ARC Therapy in people with experiencing gait challenges.”
All ONWARD devices and therapies, including but not limited to ARC-IM, ARC-EX, and ARC Therapy, are investigational and not available for commercial use.
Note: ONWARD has previously disclosed achievement of human proof-of-concept for Parkinson’s disease in its Company Presentation.
About ONWARD Medical
ONWARD is a medical technology company creating therapies to restore movement, function, and independence in people with spinal cord injury (SCI) and movement disabilities. Building on more than a decade of science and preclinical research conducted at leading neuroscience laboratories, the Company has received nine Breakthrough Device Designations from the US Food and Drug Administration for its ARC Therapy™ platform.
ONWARD® ARC Therapy, which can be delivered by external ARC-EX™ or implantable ARCIM™ systems, is designed to deliver targeted, programmed spinal cord stimulation. Positive results were presented in 2023 from the Company’s pivotal study, called Up-LIFT, evaluating the ability for transcutaneous ARC Therapy to improve upper extremity strength and function. The Company is now preparing regulatory approval submissions for ARC-EX for the US and Europe. In parallel, the Company is conducting studies with its implantable ARC-IM platform, which demonstrated positive interim clinical outcomes for improved blood pressure regulation, a component of hemodynamic stability following SCI. Other ongoing studies include combination use of ARC-IM with a brain-computer interface (BCI).
Headquartered in Eindhoven, the Netherlands, ONWARD has a Science and Engineering Center in Lausanne, Switzerland and a US office in Boston, Massachusetts. The Company also has an academic partnership with .NeuroRestore, a collaboration between the Swiss Federal Institute of Technology (EPFL), and Lausanne University Hospital (CHUV).
For more information, visit ONWD.com, and connect with us on LinkedIn and YouTube.
For Media Enquiries:
Aditi Roy, VP Communications
For Investor Enquiries:
Khaled Bahi, CFO
Disclaimer
Certain statements, beliefs, and opinions in this press release are forward-looking, which reflect the Company’s or, as appropriate, the Company directors’ current expectations and projections about future events. By their nature, forward-looking statements involve several risks, uncertainties, and assumptions
that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties, and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition, and technology, can cause actual events, performance, or results to differ significantly from any anticipated development. Forward-looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions, or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person’s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release. All ONWARD devices and therapies referenced here, including but not limited to ARC-IM, ARC-EX, and ARC Therapy, are investigational and not available for commercial use.
Carisma Presents Pre-Clinical Proof of Concept for in vivo CAR-M using mRNA platform in collaboration with Moderna at SITC
Carisma Presents Pre-Clinical Proof of Concept for in vivo CAR-M using mRNA platform in collaboration with Moderna at SITC
- Data demonstrate feasibility, tolerability, and early efficacy of mRNA/LNP in vivo CAR-M therapy in pre-clinical models of metastatic solid tumors
- Proof of concept achieved for platform under the Moderna collaboration
- "In vivo CAR-M: Redirecting endogenous myeloid cells with mRNA for cancer immunotherapy" to be presented Friday, November 3, 2023, at 11:30 am PT
PHILADELPHIA, Oct. 31, 2023 /PRNewswire/ — Carisma Therapeutics Inc. (Nasdaq: CARM) (“Carisma” or the “Company”), a clinical stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced that it will present new findings at the Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting regarding its first-of-its-kind collaboration with Moderna. The collaboration aims to bring together Carisma’s chimeric antigen receptor macrophage (CAR-M) platform with Moderna’s mRNA and lipid nanoparticle (LNP) technologies to generate and develop in vivo CAR-M therapeutics.
Accepted as a late-breaking presentation, “In vivo CAR-M: Redirecting endogenous myeloid cells with mRNA for cancer immunotherapy,” showcases data that demonstrate Carisma’s CAR-M therapy can be directly produced in vivo, or within the body, successfully redirecting endogenous myeloid cells against tumor-associated antigens using mRNA/LNP. The pre-clinical data demonstrate feasibility, tolerability, and efficacy against metastatic solid tumors. This novel approach to cancer immunotherapy offers an off-the-shelf solution that has the potential to increase access to CAR-based therapies and will be the basis of CAR-M programs to be developed under the Carisma and Moderna collaboration.
“The data presented at SITC is incredibly exciting as it demonstrates that we have the ability to make CAR-M directly in vivo with mRNA/LNP technology, leading to robust and targeted anti-tumor activity,” said Michael Klichinsky, PharmD, PhD, Co-Founder and Chief Scientific Officer at Carisma. “This off-the-shelf approach to treat cancer with engineered myeloid cells, developed in collaboration with Moderna, has the potential to transform the CAR field and be applied to numerous cancer targets and indications.”
“We are pleased to share data about the successful application of our mRNA platform to advance in vivo cell therapy,” said Lin Guey, PhD, Chief Scientific Officer of External Research Ventures at Moderna. “We look forward to the continuation of our pre-clinical work with Carisma and are optimistic that the joint scientific knowledge of both companies will accelerate the development of novel in vivo CAR-M therapies for patients.”
Details of Carisma data accepted for presentation at the SITC 38th Annual Meeting are as follows:
- “In vivo CAR-M: Redirecting endogenous myeloid cells with mRNA for cancer immunotherapy” presented on Friday, November 3, 2023, at 11:30 am PT
- “CAR-Macrophages with custom intronic shRNA exhibit enhanced efficacy against solid tumors” presented on Friday, November 3, 2023, 9:00 am – 7:00 pm PT
- “Engineered Microenvironment Converters (EM-C): Macrophages expressing synthetic cytokine receptors reverse immunosuppressive signals in solid tumors” presented on Friday, November 3, 2023, 9:00 am – 7:00 pm PT
- “A Phase 1, First in Human (FIH) study of autologous macrophages engineered to express an anti-HER2 chimeric antigen receptor (CAR) in participants (pts) with HER2 overexpressing solid tumors” presented on Friday, November 3, 2023, 9:00 am – 7:00 pm PT
Presentation and posters will be available on the SITC 38th Annual Meeting portal for registered attendees.
About Carisma
Carisma Therapeutics Inc. is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA. For more information, please visit www.carismatx.com.
About Moderna
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio and integrated manufacturing facilities that allow for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years. To learn more, visit www.modernatx.com.
Cautionary Note on Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Carisma’s business, strategy, future operations, cash runway, the advancement of Carisma’s product candidates and product pipeline, and pre-clinical and clinical development of Carisma’s product candidates, including expectations regarding timing of initiation and results of pre-clinical and clinical trials. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “may,” “might,” “outlook,” “plan,” “project,” “potential,” “predict,” “target,” “possible,” “will,” “would,” “could,” “should,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of these risks and uncertainties, and other important factors, any of which could cause Carisma’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” set forth in the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 10, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Carisma’s other recent filings with the Securities and Exchange Commission. Any forward-looking statements that are made in this press release speak as of the date of this press release. Carisma undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.
Media Inquiries:
Carisma to Present First Results From in vivo CAR-M Collaboration with Moderna at SITC 2023
Carisma to Present First Results From in vivo CAR-M Collaboration with Moderna at SITC 2023
-Late-breaking abstract to be presented on November 3rd at 11:30 am PT highlighting pre-clinical data with Moderna
-Two additional pre-clinical data abstracts highlighting next-generation enhancements to Carisma's cell therapy platform to be shared
PHILADELPHIA, Oct. 25, 2023 /PRNewswire/ — Carisma Therapeutics Inc. (Nasdaq: CARM) (“Carisma” or the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing innovative immunotherapies, today announced that new pre-clinical data leveraging an mRNA platform to develop in-vivo chimeric antigen receptor macrophage (“CAR-M”) will be presented as a late-breaking abstract (#LBA1514) at the upcoming Society for Immunotherapy of Cancer (SITC) 38th Annual Meeting held from Wednesday, November 1, to Sunday, November 5, 2023, in San Diego, California. Two posters highlighting next-generation enhancements to Carisma’s CAR-M platform, including a custom intronic shRNA approach and data on Engineered Microenvironment Converters (EM-C), will also be presented. Additionally, Carisma will share a trial-in-progress poster overviewing its Phase 1 first-in-human (FIH) study design of its lead program, CT-0508, sharing objectives and eligibility criteria.
Carisma will participate in the virtual SITC 2023 Annual Meeting Press Conference on Wednesday, November 1, 2023, from 12:00–1:30 pm PT.
“We are excited to present this pre-clinical data from our collaboration with Moderna for the first time,” said Steven Kelly, President and Chief Executive Officer of Carisma. “Over the past year, we have leveraged the expertise of both companies to pioneer the development of in-vivo mRNA/LNP-based cell therapy. The study is exploring the potential for an off-the-shelf treatment approach to enhance therapeutic benefit for patients living with cancer.”
Oral & Poster Presentations:
- In vivo CAR-M: Redirecting endogenous myeloid cells with mRNA for cancer immunotherapy
- Primary Author: Bindu Varghese, PhD
- Presentation Type/#: Oral – 1514
- Session Date/Time (PT): Friday, November 3, 2023, 11:30 am, Session 104: Late Breaking Abstract Session
- CAR-Macrophages with custom intronic shRNA exhibit enhanced efficacy against solid tumors
- Primary Author: Chris Sloas, PhD
- Presentation Type/#: Poster – 307
- Session Date/Time: Friday, November 3, 2023, 9:00 am – 7:00 pm
- Engineered Microenvironment Converters (EM-C): Macrophages expressing synthetic cytokine receptors reverse immunosuppressive signals in solid tumors
- Primary Author: Chris Sloas, PhD
- Presentation Type/#: Poster – 389
- Session Date/Time: Friday, November 3, 2023, 9:00 am – 7:00 pm
- A Phase 1, First in Human (FIH) study of autologous macrophages engineered to express an anti-HER2 chimeric antigen receptor (CAR) in participants (pts) with HER2 overexpressing solid tumors
- Primary Author: Kim Reiss, MD
- Presentation Type/#: Poster – 635
- Session Date/Time: Friday, November 3, 2023, 9:00 am – 7:00 pm
Presentation and posters will be available in the Journal for ImmunoTherapy of Cancer (JITC) supplement once published on Tuesday, October 31, 2023, at 9:00 am ET.
About CT-0508
CT-0508 is a human epidermal growth factor receptor 2 (HER2) targeted chimeric antigen receptor macrophage (CAR-M). It is being evaluated in a landmark Phase 1 multi-center clinical trial that focuses on patients with recurrent or metastatic HER2-overexpressing solid tumors whose cancers do not have approved HER2-targeted therapies or who do not respond to treatment. Carisma is selecting participants who have tumors of any anatomical origin, but with the commonality of overexpressing the HER2 receptor on the cell surface, which is the target for our CAR-M. The Phase 1 clinical trial marks the first time that engineered macrophages are being studied in humans. The trial continues to enroll patients at seven clinical sites in the U.S., including (i) Penn Medicine’s Abramson Cancer Center, (ii) the University of North Carolina Lineberger Comprehensive Cancer Center, (iii) the City of Hope National Medical Center, (iv) the MD Anderson Cancer Center, (v) the Sarah Cannon Cancer Research Institute, (vi) Oregon Health & Science University and (vii) Fred Hutchinson Cancer Center.
About Carisma
Carisma Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response. Carisma is headquartered in Philadelphia, PA. For more information, please visit www.carismatx.com.
About Moderna
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio and integrated manufacturing facilities that allow for rapid clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Moderna has been named a top biopharmaceutical employer by Science for the past eight years. To learn more, visit www.modernatx.com.
Cautionary Note on Forward-Looking Statements
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to Carisma’s business, strategy, future operations, cash runway, the advancement of Carisma’s product candidates and product pipeline, and clinical development of Carisma’s product candidates, including expectations regarding timing of initiation and results of pre-clinical and clinical trials. The words “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “goals,” “intend,” “may,” “might,” “outlook,” “plan,” “project,” “potential,” “predict,” “target,” “possible,” “will,” “would,” “could,” “should,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.
Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. For a discussion of these risks and uncertainties, and other important factors, any of which could cause Carisma’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” set forth in the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 10, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Carisma’s other recent filings with the Securities and Exchange Commission. Any forward-looking statements that are made in this press release speak as of the date of this press release. Carisma undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.
Media Inquiries:
Immatics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ACTengine® IMA203 TCR-T Monotherapy
Immatics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ACTengine® IMA203 TCR-T Monotherapy
- RMAT designation granted by FDA CBER for IMA203 cell therapy in multiple PRAME-expressing tumors including cutaneous and uveal melanoma, ovarian cancer and other cancer types
- Regulatory activities underway with an initial focus on a registration-directed trial in melanoma as step one to leverage the full breadth of PRAME
Houston, Texas and Tuebingen, Germany, October 24, 2023 – Immatics N.V. (NASDAQ: IMTX, “Immatics”), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, today announced that its IMA203 TCR-T program has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA Center for Biologics Evaluation and Research (CBER) in multiple relapsed and/or refractory HLA-A*02:01-positive and PRAME-expressing cancers, including cutaneous melanoma, uveal melanoma, endometrial carcinoma, synovial sarcoma, and ovarian cancer. IMA203 is a TCR-T cell therapy targeting PRAME, a protein frequently expressed in a large variety of solid tumors.
“The FDA RMAT designation for multiple indications underscores the broad potential of IMA203 and the benefits it may provide for advanced-stage solid tumor patients. This is an important regulatory milestone and a recognition of our clinical development progress for this program,” said Cedrik Britten, Chief Medical Officer of Immatics. “The close support from the FDA resulting from the RMAT status enhances our efforts to accelerate bringing IMA203 to cancer patients by enabling real-time discussions on patient populations, trial design and CMC.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising pipeline products, including cell therapies, that includes all the benefits of Fast Track and Breakthrough designation programs. An investigational cell therapy is eligible for RMAT designation if it meets the definition of regenerative medicine therapy, it is intended to treat, modify, reverse, or cure a serious or life-threatening disease; and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. Advantages of the RMAT designation include early interactions with the FDA that may be used to discuss potential surrogate or intermediate endpoints for accelerated approval and potential ways to satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.
Based on publicly available information1, it is the Company’s understanding that this is the first time that FDA has granted a RMAT designation for an oncology drug candidate for more than two solid tumor indications. As of Sep 30, 2023, the U.S. FDA has received at least 238 requests for RMAT designations and granted 922.
About IMA203 and target PRAME
ACTengine® IMA203 T cells are directed against an HLA-A*02:01-presented peptide derived from preferentially expressed antigen in melanoma (PRAME), a protein frequently expressed in a large variety of solid cancers, thereby supporting the program’s potential to address a broad cancer patient population. Immatics’ PRAME peptide is present at a high copy number per tumor cell and is homogeneously and specifically expressed in tumor tissue. The peptide has been identified and characterized by Immatics’ proprietary mass spectrometry-based target discovery platform, XPRESIDENT®. Through its proprietary TCR discovery and engineering platform XCEPTOR®, Immatics has generated a highly specific T cell receptor (TCR) against this target for its TCR-based cell therapy approach, ACTengine® IMA203.
ACTengine® IMA203 TCR-T is currently being evaluated in three ongoing Phase 1b dose expansion cohorts in last-line patients: Cohort A IMA203 GEN1 monotherapy, Cohort B IMA203 in combination with an immune checkpoint inhibitor (deprioritized) and Cohort C IMA203CD8 GEN2 monotherapy, where IMA203 engineered T cells are co-transduced with a CD8αβ co-receptor.
About ACTengine®
ACTengine® is a personalized cell therapy approach for patients with advanced solid tumors. The patient’s own T cells are genetically engineered to express a novel, proprietary TCR directed against a defined cancer target. The modified T cells are then reinfused into the patient to attack the tumor. The approach is also known as TCR-engineered cell therapy (TCR-T). All Immatics’ ACTengine® product candidates can be rapidly manufactured utilizing a proprietary manufacturing process designed to enhance T cell engraftment and persistence in vivo.
The ACTengine® T cell products are manufactured at the Evelyn H. Griffin Stem Cell Therapeutics Research Laboratory in collaboration with UTHealth.
– END –
About Immatics
Immatics combines the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors with the goal of enabling a robust and specific T cell response against these targets. This deep know-how is the foundation for our pipeline of Adoptive Cell Therapies and TCR Bispecifics as well as our partnerships with global leaders in the pharmaceutical industry. We are committed to delivering the power of T cells and to unlocking new avenues for patients in their fight against cancer.
Immatics intends to use its website www.immatics.com as a means of disclosing material non-public information. For regular updates, you can also follow us on Twitter, Instagram and LinkedIn.
Forward-Looking Statements:
Certain statements in this press release may be considered forward-looking statements. Forward-looking statements generally relate to future events or Immatics’ future financial or operating performance. For example, statements concerning the timing of product candidates and Immatics’ focus on partnerships to advance its strategy are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may”, “should”, “expect”, “intend”, “will”, “estimate”, “anticipate”, “believe”, “predict”, “potential” or “continue”, or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward looking statements. These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Immatics and its management, are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including general economic conditions and other risks, uncertainties and factors set forth in filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. Immatics undertakes no duty to update these forward-looking statements. All the scientific and clinical data presented within this press release are – by definition prior to completion of the clinical trial and a clinical study report – preliminary in nature and subject to further quality checks including customary source data verification.
For more information, please contact:
Media | |
Eva Mulder or Charlotte Spitz | |
Trophic Communications | |
Phone: +31 65 2331 579 | |
Investor Relations | |
Sabrina Schecher, Ph.D. | |
Senior Director, Investor Relations | |
Phone: +49 89 262002433 | |
1 Source: https://bioinformant.com/rmat/
2 Source: FDA – https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/cumulative-cber-regenerative-medicine-advanced-therapy-rmat-designation-requests-received-fiscal
MinervaX Raises EUR 54M in Upsized Financing to Advance the Development of its Maternal Vaccine Against Group B Streptococcus
MinervaX Raises EUR 54M in Upsized Financing to Advance the Development of its Maternal Vaccine Against Group B Streptococcus
- New investment from EQT Life Sciences and OrbiMed with participation from existing investors
- MinervaX will hold financial reserves of more than €125 million, following the financing
- The financing supports the Company’s efforts to commence a Phase III clinical trial of its Maternal Vaccine against Group B Streptococcus
Copenhagen, Denmark, 11 October 2023 – MinervaX ApS, a privately held Danish biotechnology company developing a novel, prophylactic vaccine against Group B Streptococcus (GBS), has today announced the completion of a EUR 54 million upsized financing. The financing includes investment from new investors EQT Life Sciences and OrbiMed, with participation from existing investors Novo Holdings, Pureos Ventures, Sanofi Ventures, Trill Impact Ventures, Adjuvant Capital, Wellington Partners, Industrifonden, Sunstone LifeScience Ventures, and LF Invest. Vincent Brichard of EQT Life Sciences and Tal Zaks of OrbiMed will join the MinervaX Board of Directors.
GBS is a leading cause of life-threatening infections in newborns as well as adverse pregnancy outcomes such as preterm delivery and stillbirths. Current prophylactic measures provide insufficient protection, meaning there is an urgent need to accelerate the development of a GBS vaccine. A video describing the unmet medical need for a GBS vaccine and details of MinervaX’s novel GBS maternal vaccine can be found on the Company’s website, here: https://youtu.be/HvkRJBqsjjY.
The Company is currently progressing two Phase II clinical trials in 470 pregnant persons across Denmark, United Kingdom, Uganda and South Africa. Initial data from these clinical trials are highly positive and demonstrate that the vaccine has an acceptable safety profile, is highly immunogenic and gives rise to functionally active antibodies. Details of MinervaX’s clinical trials can be found at clinicaltrials.gov under the identifiers NCT04596878 and NCT05154578. In addition to pregnant persons, MinervaX is also pursuing Phase I development of its novel GBS vaccine in Older Adults, under identifier NCT05782179.
This financing will enable MinervaX to progress its novel GBS vaccine towards Phase III clinical trials in 2024.
Per Fischer, CEO of MinervaX, said: “The addition of EQT Life Sciences and OrbiMed to our existing investor consortium further strengthens the Company’s resolve to advance our novel GBS vaccine towards Phase III clinical trials in pregnant persons. It also provides additional validation and recognition of the acceptable safety profile and strong data demonstrated in the Phase II clinical trials. We are delighted to welcome Vincent Brichard and Tal Zaks to the board of directors, who will bring invaluable vaccine expertise as we continue to address the pressing need for the development of a novel vaccine to address the unmet medical burden of Group B Streptococcus.”
Vincent Brichard, Venture Partner EQT Life Sciences, commented: “EQT Life Sciences is thrilled to take an active part in the MinervaX prophylactic vaccine against GBS with the hope to save newborns’ lives. We are impressed by the clinical data achieved so far, the quality of the team and the near-term milestones enabling MinervaX to start a registration trial.”
Tal Zaks, Partner at OrbiMed, added: “We recognize the unmet need for better protection against GBS disease for vulnerable populations and the potential for MinervaX’s vaccine to provide best-in-class efficacy. I look forward to working with the MinervaX team to support the full development of this program.”
ENDS
For further information please contact:
MinervaX
Per Fischer | Chief Executive Officer
Email:
Optimum Strategic Communications
Mary Clark / Stephen Adams/ Zoe Bolt
Email:
Tel: +44 (0) 203 882 9621
Notes to Editors:
About MinervaX
MinervaX is a Danish biotechnology company, established in 2010 to develop a prophylactic vaccine against Group B Streptococcus (GBS), based on research from Lund University. MinervaX is developing a GBS vaccine for maternal immunization, and now also for vaccination of older adults, with Phase II data suggesting superior efficacy compared with other GBS vaccine candidates in development. The latter are based on traditional capsular polysaccharide (CPS) conjugate technology. By contrast, MinervaX’s vaccine is a protein-only vaccine based on fusions of highly immunogenic and protective protein domains from selected surface proteins of GBS (the Alpha-like protein family). Given the broad distribution of proteins contained in the vaccine on GBS strains globally, it is expected that MinervaX’s vaccine will confer protection against virtually 100% of all GBS isolates. www.minervax.com
About Group B Streptococcus (GBS)
GBS is responsible for nearly 50% of all life-threatening infections in newborns. At any given time, some 15-25% of women are spontaneously colonized with GBS, and they run the risk of transmitting the bacteria to their child in the womb, during birth and/or during the first months of life. GBS colonization may lead to late abortions, premature delivery, or stillbirth and, in the newborn child, may result in sepsis, pneumonia or meningitis, all of which carry a significant risk of severe morbidity, long- term disability or death.
Currently, the only preventative strategy available involves the use of intravenously delivered prophylactic antibiotics which cannot comprehensively prevent GBS infection in utero and do not protect against late-onset infection in newborns. Not only is this approach expensive and logistically challenging, it fails to cover all, including the most severe cases in the US and Europe, and is rarely available in resource- limited settings. Finally, it carries the risk for increasing antibiotics resistance, a recognized worldwide health threat.
The development of a GBS vaccine is also endorsed by Group B Strep Support and Group B Strep International, and GBS has been prioritized by several public health organizations including the WHO. Both increased uptake of immunization among pregnant women and greater awareness of the implications of GBS suggest that a safe and effective vaccine targeting GBS would be well suited to address this unmet need.
About EQT Life Sciences
EQT Life Sciences was formed in 2022 following an integration of LSP, a leading European life sciences and healthcare venture capital firm, into the EQT platform. As LSP, the firm raised over EUR 3.0 billion (USD 3.5 billion) and supported the growth of more than 150 companies since it started to invest over 30 years ago. With a dedicated team of highly experienced investment professionals, coming from backgrounds in medicine, science, business, and finance, EQT Life Sciences backs the smartest inventors who have ideas that could truly make a difference for patients.
More info: www.eqtgroup.com
Follow EQT on LinkedIn, Twitter, YouTube and Instagram
About OrbiMed
OrbiMed is a healthcare investment firm, with approximately $17 billion in assets under management. OrbiMed invests globally across the healthcare industry through a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed’s team of over 100 professionals is based in New York City, San Francisco, Shanghai, Hong Kong, Mumbai, Herzliya, London and other key global markets.
More info: www.orbimed.com
Sidekick Health announces landmark acquisition, expanding its portfolio to offer regulated Prescription Digital Therapeutics
Sidekick Health announces landmark acquisition, expanding its portfolio to offer regulated Prescription Digital Therapeutics
The acquisition of aidhere strengthens Sidekick Health’s digital health offering to patients and partners with the world’s leading PDT company
Las Vegas, October 10, 2023 – Sidekick Health, a global leader in digital health innovation, is poised to transform the landscape of digital therapeutics with the strategic acquisition of aidhere, a leading developer of prescription digital therapeutics (PDT) based in Germany.
In a significant move that underscores Sidekick Health’s commitment to furthering the impact of digital healthcare, the company is thrilled to announce its expansion into the direct prescribing space working collaboratively with healthcare providers.
This acquisition brings with it zanadio, one of the most successful PDTs launched to date. zanadio has fulfilled over 50,000 prescriptions and is recognized within Germany’s regulated nationwide DiGA (Digitale Gesundheitsanwendungen) formulary for prescribable digital apps. Achieving long-term behavioral change and significant weight loss of 8% in patients at the end of 12 months[1], and with over 10,000 doctors prescribing it, zanadio is fully reimbursed for patients with a BMI from 30 to 40 kg/m². As a permanently approved DiGA, zanadio is reimbursed by all statutory health insurers in Germany.
Sidekick Health’s CEO, Dr. Tryggvi Thorgeirsson, shared that the decision to acquire aidhere was fueled by the shared PDT vision and cultural alignment of the two organizations. “Developing and distributing prescribed therapeutics has always been core to our long-term vision, and when we were introduced to aidhere’s remarkable products and exceptional team, we knew that joining forces was the right step to take,” said Thorgeirsson.
This strategic acquisition places Sidekick Health at the forefront of the industry, while presenting exciting opportunities for the introduction of a new offering to Payer partners in the US, and the co-development of prescription digital therapeutic products with global pharmaceutical partners.
By integrating aidhere’s expertise and products into its portfolio, Sidekick Health strengthens its position as a trusted partner for global health insurers and pharmaceutical giants as it pioneers the introduction of PDTs.
In addition to the newly formed PDT business, Sidekick Health’s business is primarily centered on two areas: payers in the US and global pharma. Within the US payer landscape, the company has notably collaborated with the largest US health insurer, and is live with US insurance partners in Medicare, Medicaid and commercial populations. In the pharmaceutical sector, it has cultivated strategic partnerships with industry giants like Pfizer and Eli Lilly, with multiple live programs in both the US and Europe, and expanding to more markets in 2024.
The acquisition brings together the passion, knowledge, and experience of two industry leaders. Sidekick Health’s robust digital health ecosystem, a patient-centric platform with multi-chronic capabilities that supports over 20 disease areas, coupled with aidhere’s expertise in highly-regulated PDTs, creates a synergy poised to redefine patient-centric care. With its platform approach, Sidekick Health is positioned to emerge as a natural consolidator in the digital therapeutics space.
“aidhere has a strong and respected track record within the digital health industry, with one of the most successful PDT products in the world. This acquisition brings market-leading products and a depth of expertise in prescription digital therapeutics to Sidekick, as we enhance and build on our patient care offerings in the digital health and therapeutics space with our global partners,” said Thorgeirsson.
Sidekick Health’s recent success in closing its Series B fundraising round in May 2022 laid the foundations for the company to accelerate innovation and drive even greater impact in the digital health and therapeutics sector. Notable investors in Sidekick Health include Novator Partners, Asabys Partners, Frumtak Ventures, and Wellington Partners.
– ENDS –
About Sidekick Health
Sidekick Health is a digital health and therapeutics innovator founded by two passionate medical doctors on a mission to improve the health of humanity. Sidekick provides a uniquely wide range of digital health programs (including oncology, cardiovascular, metabolic, inflammatory, and other chronic conditions), engaging and empowering people to make positive changes, and improve their health outcomes and quality of life. Sidekick’s Adaptive CarePaths combine multiple programs into one, enabling multi-chronic care to support the growing number of people living with multiple chronic conditions.
Sidekick works with health insurers, including the largest U.S. health insurer, and pharmaceutical companies, including Eli Lilly and Pfizer, to improve patient outcomes, drive clinical efficiency, and lower the cost of care. Sidekick currently operates in multiple global markets and across the U.S., including patients in all insurance classes (Medicare, Medicaid, and Commercial).
Sidekick has offices in Boston, Berlin, Hamburg, Reykjavik, and Stockholm. To learn more, visit www.sidekickhealth.com.
Media Contact:
Serene Touma
Senior Director of Marketing & Communications
+49 1522 6788040
eGenesis Announces Publication in Nature of Landmark Preclinical Data Demonstrating Long-Term Survival with Genetically Engineered Porcine Kidneys
eGenesis Announces Publication in Nature of Landmark Preclinical Data Demonstrating Long-Term Survival with Genetically Engineered Porcine Kidneys
- Proof of concept study resulted in life-supporting organ function and recipient survival of over two years
- Donor kidneys carrying human transgenes resulted in longer survival time
Cambridge, Mass. (Oct 11, 2023) – eGenesis, a biotechnology company developing human-compatible (HuCo™) organs for the treatment of organ failure, today announced publication in the journal Nature of long-term survival data from a proof-of-concept study evaluating engineered porcine donor kidneys transplanted into a cynomolgus macaque model. This dataset will support advancement of the company’s lead candidate for kidney transplant,
EGEN-2784, toward clinical development.
These results represent the largest and most comprehensive preclinical dataset published in the field to date. Recipient survival in the preclinical setting has historically been measured in weeks or months. The publication titled: “Design and Testing of a Humanized Porcine Donor for
Xenotransplantation,” reported long-term survival of NHP recipients of the company’s genetically engineered porcine kidneys. In the case of one recipient, survival of over two years (758 days) was achieved.
“At eGenesis, we are focused on transformational progress for the field – improving long-term survival for transplant recipients from months to years,” said Michael Curtis, Ph.D., Chief Executive Officer of eGenesis. “Our HuCoTM organs offer the hope of a new donor source for the hundreds of thousands of individuals in need of lifesaving organ transplants. The data published in Nature illustrate our rapid advancement in engineering porcine donor organs to enhance recipient compatibility and long-term survival, a critical step toward successful translation in human clinical trials.”
The donor kidneys evaluated in this study carried three types of edits: (1) knock out of three genes involved in the synthesis of glycan antigens implicated in hyperacute rejection, (2) insertion of seven human transgenes involved in the regulation of several pathways that modulate rejection: inflammation, innate immunity, coagulation, and complement, and (3) inactivation of the endogenous retroviruses in the porcine genome.
Donor kidneys carrying human transgenes resulted in longer survival time when transplanted into NHPs. Donor kidneys containing only knock out of the three-glycan antigens experienced poor graft survival, whereas those harboring the knock outs and human transgenes resulted in more than seven times longer duration – a median of 24 days versus 176 days, respectively. The results indicate the benefit of human transgene expression in porcine kidney grafts onlong-term survival.
In vitro functional analysis showed that edited porcine kidney endothelial cells modulated inflammation in a manner that mirrored human endothelial cells, suggesting the edited cells acquired a high level of human immune compatibility. Furthermore, the evaluation of renal function biomarkers in recipients with stable grafts revealed that a single transplanted porcine kidney provided sufficient filtration of metabolites to compensate for the lack of two native kidneys.
“This is a major step forward for the field of transplantation,” said Tatsuo Kawai, M.D., Ph.D., Professor of Surgery at Harvard Medical School and A. Benedict Cosimi Chair in Transplant Surgery at Massachusetts General Hospital. “One of the biggest hurdles has been long-term survival of the genetically engineered organ in the NHP recipient, and this dataset demonstrates remarkable progress in editing the porcine genome to minimize hyperacute rejection, improve recipient compatibility and address the risk of viral transmission from donor to host. We anticipate that transplant outcomes in humans will be even more favorable, as these gene edited organs are a better match for humans, as compared with NHPs.”
The data generated in this study will support the advancement of the company’s lead candidate for kidney transplant, EGEN-2784, toward clinical development. eGenesis is also progressing programs for extracorporeal liver perfusion as well as cardiac transplant.
Organ failure is a life-threatening condition for which transplantation is considered the gold standard treatment. However, the demand for organs far outstrips supply – of the more than 100,000 individuals on the organ transplantation waitlist in the U.S., less than 40% will receive a potentially life-saving organ. In addition, the existing organ failure treatment paradigm is suboptimal for patients and the healthcare system due to organ incompatibility and variable donor organ quality.
About eGenesis
eGenesis is pioneering a genome engineering-based approach in the development of safe and effective transplantable organs. The eGenesis Genome Engineering and Production (EGEN™) Platform is the only technology of its kind to comprehensively address cross-species molecular incompatibilities and viral risk via genetic engineering. eGenesis has demonstrated durable preclinical success to date and is advancing development programs for acute liver failure, kidney transplant, and pediatric as well as adult heart transplant. Learn more at
www.egenesisbio.com.
Media Contact
Kimberly Ha
eGenesis
4SC: Landmark RESMAIN study data presented at the EORTC Cutaneous Lymphoma Tumour Group Annual Meeting
Maintenance therapy is now clinically proven to postpone disease progression in advanced CTCL which could significantly change current clinical practice
- RESMAIN is one of the largest clinical trials in cutaneous T-cell lymphoma (CTCL) to-date
- Resminostat (Kinselby) as a maintenance treatment for advanced CTCL, is clinically proven to postpone disease progression in patients – a new treatment paradigm in CTCL
- Resminostat (Kinselby) treatment has shown a statistically significant improvement in progression free survival of 97.6% compared to placebo, with a risk reduction of 38% in recently announced headline trial results (median PFS: 8.3 months versus 4.2 months; p=0.015; HR: 0.623 (95%CI: 0.424, 0.916)
- Resminostat (Kinselby)’s median time to next treatment (median TTNT) versus placebo showed a significant improvement, more than doubling to 8.8 months compared to 4.2 months; p= 0.002; HR: 594 (95% CI: 0.424, 0.916)
- The side effects of resminostat were mainly mild to moderate, manageable and reversible
- Additional analyses showed
- A clinically meaningful improvement in median “total” PFS (defined from start of last prior therapy to disease progression) of 24.3 months for patients treated with resminostat, compared to 14.9 months for those in the placebo group
- That resminostat (Kinselby) significantly delayed the development of new, or worsening of existing, skin tumours
- 4SC is actively preparing to file for its Marketing Authorization approval in the European Union, Switzerland and UK
- The Company will host a live webinar on Wednesday 4th October to discuss the findings presented at the EORTC. Dr. Susanne Danhauser-Riedl, 4SC’s Chief Medical Officer will be joined by medical experts, Professor Dr. Rudolf Stadler and Professor Julia Scarisbrick.
Planegg-Martinsried, Germany, 25 September 2023 – 4SC AG (4SC, FSE Prime Standard: VSC), a biotech company improving the lives of patients suffering with advanced-stage CTCL, today announces that renowned dermato-oncology expert and study investigator, Professor Dr. Rudolf Stadler, University Hospital Johannes Wesling, Minden, Germany, presented positive new data from the pivotal RESMAN study of resminostat (Kinselby) at the EORTC Cutaneous Lymphoma Tumour Group Annual Meeting, at the Leiden University Medical Center Amsterdam, The Netherlands, 21-23 September.
The presented findings show that maintenance therapy is now clinically proven to postpone disease progression in advanced CTCL which could significantly change clinical practice. In RESMAIN, one of the largest randomized, controlled clinical trials in advanced CTCL, resminostat (Kinselby) treatment has shown a statistically significant improvement in progression free survival of 97.6% compared to placebo, with a risk reduction of 38% in recently announced headline trial results (median PFS: 8.3 months versus 4.2 months; p=0.015; HR: 0.623 (95%CI: 0.424, 0.916).
Furthermore, resminostat (Kinselby)’s median time to next treatment (median TTNT) versus placebo showed a significant improvement of 8.8 months compared to 4.2 months; p= 0.002; HR: 0.594 (95% CI: 0.424, 0.916).
The side effects of resminostat were mainly mild to moderate, manageable and reversible and the known safety profile of resminostat (Kinselby) was confirmed in the RESMAIN study.
Additional analyses established that those treated showed a clinically meaningful improvement in median “total” PFS (defined from start of last prior therapy to disease progression) of 24.3 months, compared to 14.9 months for those in the placebo group. It was also noted that there was a significant delay in the development of new, or worsening of existing, skin tumours.
Jason Loveridge, Ph.D., CEO of 4SC, commented: “Positive data from the RESMAIN study demonstrate that resminostat (Kinselby) is effective in significantly slowing disease progression in CTCL patients. This unique treatment, which is the only proven maintenance therapy for CTCL, means that it is well placed to offer significant benefits for patients who would otherwise have no other similar treatment options available to them.
Our focus in the near term is on the registration, approval and commercialization of Kinselby in the European Union, Switzerland and the UK and we are on track to file for European Marketing Approval of Kinselby in Q1 2024, to rapidly bring this therapy into clinical use. 4SC is well positioned for realization of resminostat (Kinselby)’s considerable value through either a sale, licensing, or partnership agreement.”
The Company will host a live webinar on Wednesday 4th October to discuss the findings presented at EORTC. Dr. Susanne Danhauser-Riedl, 4SC’s Chief Medical Officer, will be joined by medical experts, Professor Dr. Rudolf Stadler and Professor Julia Scarisbrick, who will provide further detail on these data, with the presentation being followed by the opportunity to ask questions during a Q&A session moderated by Dr. Jason Loveridge, 4SC’s Chief Executive Officer.
Title: Presentation by Professor Rudolf Stadler and Professor Julia Scarisbrick on recent new positive data from the RESMAIN study
Date and Time: Wednesday 4th October, 3.00pm CET
Register and submit questions: https://stream.brrmedia.co.uk/broadcast/64f5f2dac6e9d7476c27f154
– END –
About 4SC
4SC AG is a clinical-stage biopharmaceutical company developing small-molecule drugs that target key indications in cancer with high unmet medical needs. 4SC’s pipeline is protected by a comprehensive portfolio of patents and currently comprises one drug candidate in clinical development: Kinselby – resminostat.
4SC aims to generate future growth and enhance its enterprise value by entering into partnerships with pharmaceutical and biotech companies and/or the eventual marketing and sales of approved drugs in select territories by 4SC itself.
4SC is headquartered in Planegg-Martinsried near Munich, Germany. The Company had 16 employees as of 30 June 2023 and is listed on the Prime Standard of the Frankfurt Stock Exchange (FSE Prime Standard: VSC; ISIN: DE000A3E5C40).
About Kinselby (resminostat)
Resminostat is an orally administered class I, IIb and IV histone deacetylase (HDAC) inhibitor that potentially offers an approach to treating different kinds of cancer. Resminostat demonstrated that it is well tolerated and can inhibit tumor growth and proliferation, cause tumor regression, and strengthen the body’s immune response to cancer. Resminostat is currently being investigated in a pivotal study in cutaneous T-cell lymphoma (CTCL) as maintenance treatment by 4SC in Europe and by Yakult Honsha in Japan.
About cutaneous T-cell lymphoma (CTCL)
CTCL is a rare disease with approximately 5,000 patients being newly diagnosed in Europe each year. The disease arises from malignant transformation of T-cells, a specialized subgroup of immune cells, and primarily affects the skin, but may ultimately involve lymph nodes, blood and visceral organs.
Currently, CTCL is incurable and treatment options for advanced-stage CTCL are limited. Although patients respond to the available treatment options, the duration of response is often short-lived and declines as the severity of the disease increases. The key therapeutic challenge in advanced-stage CTCL is therefore to make remissions more durable by halting disease progression and improving patient’s quality of life.
About the RESMAIN study – resminostat for maintenance treatment of CTCL
The pivotal RESMAIN study was conducted at more than 50 clinical centers in 11 European countries and Japan. It included 201 patients who suffer from advanced-stage cutaneous T-cell lymphoma (CTCL) that have achieved disease control with systemic therapy. The patients were randomized 1:1 to receive either resminostat or placebo. Patients who experienced disease progression – while being on placebo – were offered resminostat in an open label treatment arm.
The primary goal of the study was to determine whether maintenance treatment with resminostat prolongs progression-free survival and other secondary objectives. Data demonstrating that resminostat met the primary endpoint of the RESMAIN study was published in May 2023.
About the concept of maintenance therapy
The pivotal RESMAIN study is focused on patients with advanced-stage, incurable, cutaneous T-cell lymphoma (CTCL). Such patients suffer from painful and itchy skin lesions resulting in disfigurement and a severely impaired quality of life. Furthermore, lymph nodes, blood or visceral organs can be involved. The current therapeutic options rarely provide long-lasting responses or stabilization of disease for meaningful periods, with most patients progressing within a few months.
Resminostat is being evaluated as maintenance treatment – a unique innovative treatment approach in CTCL (Stadler et al., 2021) - intended to prolong the period patients are stable and not progressing.
Forward-looking information
Information set forth in this press release contains forward-looking statements, which involve risks and uncertainties. The forward-looking statements contained herein represent the judgement of 4SC as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond 4SC’s control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. 4SC expressly disclaims any obligation or undertaking to release any updates or revisions to any such statements to reflect any change in its expectations or any change in events, conditions or circumstances on which any such statement is based.
Contacts:
4SC AG | |
Optimum Strategic Communications | |
Mary Clark, Jonathan Edwards, Eleanor Cooper | |
Moderna and Immatics Announce Strategic Multi-Platform Collaboration to Develop Innovative Oncology Therapeutics
Moderna and Immatics Announce Strategic Multi-Platform Collaboration to Develop Innovative Oncology Therapeutics
- Collaboration combines leading technologies to develop breakthrough, mRNA-enabled in vivo expressed TCER® molecules
- Companies to leverage Immatics’ XPRESIDENT® target discovery platform and Moderna’s mRNA technology for the development of novel cancer vaccines
- Collaboration to include evaluation of Immatics’ investigational IMA203 PRAME TCR-T in combination with Moderna’s investigational PRAME mRNA cancer vaccine
- Immatics to receive $120 million upfront cash payment plus research funding with the potential for additional milestone and royalty payments
Cambridge, Massachusetts and Tuebingen, Germany, September 11, 2023 – Moderna, Inc. (NASDAQ: MRNA, “Moderna”) and Immatics N.V. (NASDAQ: IMTX, “Immatics”), a clinical-stage biopharmaceutical company active in the discovery and development of T cell-redirecting cancer immunotherapies, today announced a strategic research and development collaboration to pioneer novel and transformative therapies for cancer patients with high unmet medical need. This broad multi-platform collaboration will leverage the deep scientific expertise and core operational capabilities of both companies, combining Immatics’ TCR platform with Moderna’s cutting-edge mRNA technology, and span various therapeutic modalities including bispecifics, cell therapy and cancer vaccines.
The strategic R&D collaboration between Moderna and Immatics focuses on three pillars:
- Applying Moderna’s mRNA technology for in vivo expression of Immatics’ next-generation, half-life extended TCR bispecifics (TCER®) targeting cancer-specific HLA-presented peptides.
- Enabling the discovery and development of novel mRNA-based cancer vaccines by leveraging Moderna’s deep knowledge of mRNA science and customized information from Immatics’ wealth of tumor and normal tissue data included in the target discovery platform XPRESIDENT® and its bioinformatics and AI platform XCUBE™.
- Evaluating Immatics’ IMA203 TCR-T therapy targeting PRAME in combination with Moderna’s PRAME mRNA-based cancer vaccine. The collaboration contemplates conducting preclinical studies and a Phase 1 clinical trial evaluating the safety and efficacy of the combination with the objective of further enhancing IMA203 T cell responses.
“We are excited to embark on this strategic collaboration with Immatics, a pioneer in developing innovative cancer immunotherapies. This partnership presents a groundbreaking opportunity to leverage our mRNA technology alongside Immatics’ TCR platform, potentially diversifying and augmenting the way we approach cancer treatment. We believe this collaboration will accelerate the development of novel oncology therapies and bring us one step closer to providing significant benefits for patients with high unmet medical needs,” said Rose Loughlin, Ph.D., Moderna’s Senior Vice President for Research and Early Development.
“We are thrilled to join forces with Moderna in our quest to pioneer innovative and transformative therapies to combat cancer. We believe Immatics’ cancer target and TCR platforms, along with Moderna’s cutting-edge mRNA technology, represent a powerful combination that has the potential to deliver meaningful benefits to cancer patients,” said Toni Weinschenk, PhD, Chief Innovation Officer at Immatics. “The rapid advancement of our first 2 TCER® programs into the clinic, with additional TCER® compounds fueling our pre-clinical pipeline, underscores our commitment to develop innovative therapeutics. We are confident that we can explore the optimal delivery of TCER® molecules through this collaboration to maximize clinical benefit in a broad patient population,” added Carsten Reinhardt, MD, PhD, Chief Development Officer of Immatics.
About the Collaboration
Under the terms of the agreement, Immatics will receive an upfront payment of $120 million. Immatics will also receive research funding and is eligible to receive development, regulatory, and commercial milestone payments that could exceed $1.7 billion. Immatics is also eligible to receive tiered royalties on global net sales of TCER® products and certain vaccine products that are commercialized under the agreement. Under the agreement, Immatics has an option to enter into a global profit and loss share arrangement for the most advanced TCER®.
Moderna will lead the clinical development and commercialization of cancer vaccines and TCER® therapeutics resulting from the collaboration. Immatics will be responsible for conducting the preclinical studies and a potential Phase 1 clinical trial investigating IMA203 TCR-T in combination with the PRAME mRNA vaccine to further enhance IMA203 T cell responses. Each party will retain full ownership of its investigational PRAME compound, and the parties will fund the clinical study on a cost sharing basis.
Within the collaboration, preclinical activities conducted by Immatics will be managed by the Immatics Discovery Unit, a recently created internal division at Immatics integrating its technology platforms into one interdisciplinary team focused on all early-stage preclinical pipeline and collaboration programs.
The collaboration is subject to customary antitrust clearance in the United States.
– END –
About Moderna
In over 10 years since its inception, Moderna has transformed from a research-stage company advancing programs in the field of messenger RNA (mRNA), to an enterprise with a diverse clinical portfolio of vaccines and therapeutics across seven modalities, a broad intellectual property portfolio in areas including mRNA and lipid nanoparticle formulation, and an integrated manufacturing plant that allows for both clinical and commercial production at scale. Moderna maintains alliances with a broad range of domestic and overseas government and commercial collaborators, which has allowed for the pursuit of both groundbreaking science and rapid scaling of manufacturing. Most recently, Moderna’s capabilities have come together to allow the authorized use and approval of one of the earliest and most effective vaccines against the COVID-19 pandemic.
Moderna’s mRNA platform builds on continuous advances in basic and applied mRNA science, delivery technology, and manufacturing, and has allowed the development of therapeutics and vaccines for infectious diseases, immune-oncology, rare diseases, cardiovascular diseases, and autoimmune diseases. Moderna Genomics was created to leverage the recent advancement in both the RNA delivery platform and the genomic medicines to create the next generation of in vivo gene editing therapeutics. Moderna has been named a top biopharmaceutical employer by Science for the past eight years. To learn more, visit www.modernatx.com.
Moderna Forward-Looking Statements:
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including regarding: the agreement between Moderna and Immatics to develop innovative oncology therapeutics; the opportunity presented by the collaboration to leverage Moderna’s mRNA technology alongside Immatics’ TCR platform to potentially diversify and augment the way we approach cancer treatment; the potential for the collaboration to accelerate the development of novel oncology therapies; and the antitrust clearance process in the United States. The forward-looking statements in this press release are neither promises nor guarantees, and you should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Moderna’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements. These risks, uncertainties, and other factors include those other risks and uncertainties described under the heading “Risk Factors” in Moderna’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the U.S. Securities and Exchange Commission (SEC), and in subsequent filings made by Moderna with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Moderna disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Moderna’s current expectations and speak only as of the date of this press release.
About Immatics
Immatics combines the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors with the goal of enabling a robust and specific T cell response against these targets. This deep know-how is the foundation for our pipeline of Adoptive Cell Therapies and TCR Bispecifics as well as our partnerships with global leaders in the pharmaceutical industry. We are committed to delivering the power of T cells and to unlocking new avenues for patients in their fight against cancer.
Immatics intends to use its website www.immatics.com as a means of disclosing material non-public information. For regular updates you can also follow us on Twitter, Instagram and LinkedIn.
Immatics Forward-Looking Statements:
Certain statements in this press release may be considered forward-looking statements. Forward-looking statements generally relate to future events or Immatics’ future financial or operating performance. For example, statements concerning the timing of product candidates and Immatics’ focus on partnerships to advance its strategy are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may”, “should”, “expect”, “intend”, “will”, “estimate”, “anticipate”, “believe”, “predict”, “potential” or “continue”, or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward looking statements. These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by Immatics and its management, are inherently uncertain. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. Factors that may cause actual results to differ materially from current expectations include, but are not limited to, various factors beyond management’s control including general economic conditions and other risks, uncertainties and factors set forth in filings with the SEC. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. Immatics undertakes no duty to update these forward-looking statements.
Moderna Contacts:
Media Contact | |
Chris Ridley | |
Vice President, Communications | |
Phone: +1 617-800-3651 | |
Investor Relations Contact | |
Lavina Talukdar | |
Senior Vice President & Head of Investor Relation | |
Phone: +1 617-209-5834 | |
Immatics Contacts:
Media and Investor Relations Contact | |
Eva Mulder or Charlotte Spitz | |
Trophic Communications | |
Phone: +31 6 52 33 15 79 | |
Immatics N.V. | |
Anja Heuer | Sabrina Schecher, Ph.D. |
Senior Director, Corporate Communications | Senior Director, Investor Relations |
Phone: +49 89 540415-606 | Phone: +49 89 262002433 |