Month: May 2023

Planegg-Martinsried, Germany, 31 May 2023 – 4SC AG (4SC, FSE Prime Standard: VSC) has now filed with EMA its letter of intent to file for Marketing Authorization for resminostat in cutaneous T cell lymphoma (CTCL) and request the appointment of rapporteurs.  Given the clear benefit provided by resminostat with respect to the lengthening of progression free survival in CTCL patients 4SC is moving forward with its Marketing Authorisation Application (MAA) process for resminostat and to initiate pre-submission interactions with EMA.  4SC remains on track to submit the MAA in Q1, 2024.

RESMAIN is a pivotal study, conducted as a multi-center, double blind, randomized, placebo-controlled study, evaluating resminostat for maintenance treatment of patients with advanced-stage cutaneous T-cell lymphoma (CTCL) who have achieved disease control with prior systemic therapy.

Jason Loveridge, CEO of 4SC commented: “Our assessment of the initial data from RESMAIN, and in particular the highly significant outcome for the primary endpoint, justifies us moving rapidly forward with the first steps towards the submission of our MAA for resminostat. We continue to actively analyse the data from RESMAIN and we expect a more detailed dataset will be published at a scientific meeting probably in Q3, 2023”.

EINDHOVEN, the Netherlands — May 24, 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 showing that a wireless brain-computer interface (BCI) can use thought to modulate ARC Therapy. Researchers reported that when paired with ARC Therapy, an implanted BCI allowed an individual to gain augmented control over when and how he moved his paralyzed legs.

“This publication shows the remarkable potential of ARC Therapy to be enhanced with the introduction of a BCI, facilitating more natural movement based on the thoughts of a person living with paralysis,” said Dave Marver, CEO of ONWARD. “We have positioned ONWARD as a leader in the BCI field with our unique understanding of spinal cord stimulation for people with SCI.”

“The BCI establishes a continuous link between movement intentions and spinal cord stimulation, allowing for more natural restoration of mobility,” said neuroscientist Grégoire Courtine, professor at EPFL, and co-author of the Nature paper. “I look forward to working with the ONWARD team to advance this important new technology.”

The data published today are part of an ongoing clinical feasibility study investigating the safety and preliminary effectiveness of brain-controlled spinal cord stimulation after SCI. The study is being coordinated by .NeuroRestore co-Directors – Grégoire Courtine and Jocelyne Bloch, a neurosurgeon at Lausanne University Hospital (CHUV) – as well as Guillaume Charvet, Head of the Medical Device Development Lab at CEA-Leti / Clinatec.

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.

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 U.S. 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 following SCI in 2022. These 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 U.S. office in Boston, Massachusetts. The Company also has an academic partnership with .NeuroRestore, a collaboration between EPFL, the Swiss Federal Institute of Technology in Lausanne, 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:
Lara Smith Weber, CFO

• Resminostat met the primary endpoint in the RESMAIN study, demonstrating a statistically significant improvement in progression free survival in CTCL patients by ninety seven point six percent (97.6%) with a risk reduction of thirty eight percent (38%) compared to placebo
• Resminostat is the first and only drug to show statistically proven PFS improvement in the maintenance setting in CTCL
• Resminostat did not meet the key secondary endpoint – time to symptom (pruritus) worsening
• 4SC intends to approach the European Medicines Agency in order to evaluate the preparation of its Marketing Authorization Application
• 4SC also intends to file for Orphan Drug Designation for Resminostat in CTCL in both Europe and the United States

Waghaeusel (Germany), May 4, 2023 – Advanced Medical Balloons GmbH (formerly Creative Balloons GmbH), a specialist in medical technology from Waghaeusel near Heidelberg, today announced that the company is expanding its intensive care business to the United States. This is based on the approval which has recently been granted by the U.S. Food and Drug Administration (FDA) for their innovative catheter system hygh-tec®.1, 2 The novel fecal management system hygh-tec provides maximum seal and prevention of fecal leakage in patients receiving intensive care.

“We are delighted that the FDA cleared hygh-tec, which is a major milestone for AMB in accessing the U.S. market. Offering the first product from our unique portfolio in the U.S. for medical supply of ICU patients makes us very proud,” said Frank Gehres, CEO of Advanced Medical Balloons. “hygh-tec’s reliably high leak tightness means a significant reduction in workload and saving time for caregivers. The trans-anal sealing mechanism could also support early mobilization of cardiac patients or contribute to preventing infection in severely burnt patients. For physicians, this smart fecal management opens up novel therapeutic possibilities.”

Along with the start of U.S. sales, the company operates globally as Advanced Medical Balloons (AMB) since April 1, 2023. Advanced Medical Balloons Inc. is based in Atlanta, GA, and is currently establishing its own U.S. sales structure under the leadership of experienced General Manager Kent Johnson. “While building out our commercial team, we have initiated clinical use in ICU patients in the U.S. and are rolling out hygh-tec with intensive care clinicians throughout the country,” said Kent Johnson. “We have already seen very encouraging feedback from healthcare professionals confirming their need for a next-generation fecal management system, and I am extremely positive regarding our commercial success.”

Frank Gehres confirms: “While hygh-tec is already established in its lead market Germany, where we currently have almost a quarter of market share in ICUs and are continuously growing, the USA is the most important market for AMB, globally. With approximately 110,000 intensive care beds, we see a market potential in the mid three-digit million range. I am therefore very pleased that we have been able to win Dr. Ramona Koenig as Chief Operating Officer to support the roll-out of our products and the expansion of our business activities.” Ramona Koenig, a trained physicist with a PhD in physical chemistry, joined AMB on March 1, 2023, from Rottendorf Pharma.

The technical basis for hygh-tec is its innovative design of the elastic and deformable polyurethane catheter tube, which allows an unmatched trans-anal sealing performance. It clings to the rectal mucosa free from tension and spontaneously adapts to the sphincter dynamics and movements of the patient.

By adapting to the current pressure, intelligent synchronization with the sphincter is possible. When the tone decreases and the sphincter muscle opens, the sheath can spontaneously straighten in the anal canal and allows unobstructed defecation.

Company to host conference call today, May 2, at 8:30 am EDT / 2:30 pm CEST

• Update covers data from 11 heavily pre-treated, last-line patients in Phase 1b dose expansion Cohort A treated with IMA203 TCR-T monotherapy against PRAME
• Objective response rate (ORR): 64% (7/11) initial ORR at week 6 and 67% (6/9) confirmed ORR at month 3
• Median duration of response not reached at median follow-up time of 8.5 months at data cut-off
• Objective responses independent of solid tumor type at low, medium and high PRAME expression levels in checkpoint-refractory cutaneous melanoma, platinum-resistant ovarian cancer, uveal melanoma, head and neck cancer and synovial sarcoma
• Cohort A IMA203 monotherapy TCR-T treatment continues to show manageable tolerability with no high-grade CRS and no ICANS; no dose dependent increase of CRS observed
• Proprietary rapid manufacturing process with 7 days of manufacturing time; manufacturing success rate of 94% to reach current recommended Phase 2 dose
• Next data update and pathway towards registration-directed trials planned to be set out in 4Q 2023
• Company well capitalized with cash position¹ of $386m at YE 2022 and reach into 2025 to leverage multi-cancer PRAME opportunity

Houston, Texas and Tuebingen, Germany, May 2, 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 an interim clinical data update for 11 patients with recurrent and/or refractory solid cancers treated with ACTengine® IMA203 TCR-T monotherapy in the ongoing Phase 1b dose expansion Cohort A. IMA203 TCR-T cells are directed against an HLA-A*02-presented peptide derived from PRAME, a broadly expressed solid cancer target with clinical proof-of-concept for IMA203 demonstrated by Immatics in 2022. Overall, IMA203 showed a high rate of deep and durable objective responses, with a confirmed objective response rate of 67% (6/9), across multiple tumor types, including two confirmed partial responses (cPR) ongoing at more than 9 months after treatment and three additional partial responses ongoing at data cut-off. IMA203 monotherapy continues to be well tolerated in heavily pre-treated patients at doses of up to approximately 9 billion CD8+ TCR-T cells. No high-grade cytokine release syndrome (CRS) and no immune effector cell associated neurotoxicity syndrome (ICANS) were observed in Cohort A at data cut-off.

The data will be presented by Martin Wermke, MD, Professor at the University Hospital Dresden and Coordinating Investigator of the ACTengine® IMA203 TCR-T trial during a conference call today, May 2, at 8:30 am EDT / 2:30 pm CEST.

“The treatment of solid cancer patients who have exhausted all available standard of care options remains a significant challenge. These patients typically show fast progressing disease with very poor prognosis,” said Martin Wermke, MD, Coordinating Investigator of the ACTengine® IMA203 TCR-T trial. “It is therefore very encouraging to see that IMA203 is able to provide durable, clinically relevant responses in a variety of solid cancer patients.”

“Today marks a significant step in our efforts towards bringing our ACTengine® IMA203 monotherapy to patients with solid tumors, as we present for the first time longer-term clinical data demonstrating deep and durable responses, some of them ongoing beyond 9 months after treatment,” commented Cedrik Britten, MD, Chief Medical Officer at Immatics. “Furthermore, we show that these responses are agnostic of tumor type and that ACTengine® IMA203 achieved objective responses at widely differing PRAME expression levels. These data further increase our confidence in the success and broad potential of targeting PRAME, and our product candidate IMA203. We continue executing and anticipate announcing a potential fast-to-market pathway for the first 1-2 indications by the end of the year.”

Safety data for IMA203 TCR-T monotherapy in Phase 1b Cohort A: Treatment with IMA203 monotherapy continues to show manageable tolerability at doses as high as ~9×10⁹ TCR-T cells.

• At data cut-off on April 4, 2023, 11 PRAME-positive patients were infused with IMA203 TCR-T cells at dose level (DL) 4 or DL5 with a mean total infused dose of 3.67×10⁹ TCR-T cells (range 1.30-8.84×10⁹ TCR-T cells).
• Based on data review of 6 patients in the exploratory highest DL5, this DL was cleared by the DSMB (Data and Safety Monitoring Board) for safety, and the updated provisional recommended Phase 2 dose (RP2D) now includes DL4 and DL5. The final RP2D will be defined prior to starting Phase 2.
• Most frequent treatment-emergent adverse events (TEAEs) were as expected for cell therapies.
• All 11 patients experienced expected cytopenia (Grade 1-4) associated with lymphodepletion. 10 patients (91%) had a low to moderate (Grade 1-2) cytokine release syndrome (CRS), of which 5 patients (45%) had Grade 1, and 5 patients (45%) had Grade 2 CRS. No high-grade (Grade 3 or higher) CRS and no immune effector cell associated neurotoxicity syndrome (ICANS) were observed in any of these 11 patients. No dose-dependent increase of CRS was observed across Phase 1a and Phase 1b Cohort A (N=38 patients infused with IMA203 in total).
• No additional dose limiting toxicities (DLT) were observed in Cohort A since the initial Phase 1a dose escalation.

Clinical activity for IMA203 TCR-T monotherapy in Phase 1b Cohort A: IMA203 monotherapy demonstrates a high rate of deep objective responses with ongoing durability of more than 9 months after treatment in some patients.

• At data cut-off on April 4, 2023, 11 patients were infused with IMA203 TCR-T cells and evaluable for at least one tumor response assessment post treatment.
• Objective responses were observed in last-line solid cancer patients including cutaneous melanoma, ovarian cancer, uveal melanoma, head and neck cancer, synovial sarcoma.
• Patients were heavily pre-treated with a mean of ~4 lines of prior systemic treatments and had exhausted all available standard of care treatments.
• All cutaneous melanoma patients were checkpoint inhibitor-refractory, all ovarian cancer patients were platinum-resistant.
• Initial objective response rate (ORR) of 64% (7/11) was observed at ~week 6 (partial responses, PR, according to RECIST 1.1).
• Confirmed ORR of 67% (6/9) was observed at ~month 3; initial responses at week 6 were confirmed for all 6 responders with available subsequent 3-month scan.
• Median duration of response² was not reached (min 1.3+ months, max 8.8+ months) at a median follow-up³ of 8.5 months.
• At data cut-off, 5 of 7 responses remain ongoing:
  • 2 cPRs (cut. & uveal melanoma) ongoing at 9+ months
  • 1 cPR (cut. melanoma) ongoing at 6+ months
  • 1 cPR (ovarian cancer) ongoing at ~3 months
  • 1 PR (synovial sarcoma) ongoing at 6+ weeks
• Objective responses were observed in patients independent of tumor type at all PRAME expression levels above Immatics’ mass spectrometry-guided RNA threshold including expression levels at or just above this threshold.
• IMA203 T cells were found in all evaluable tumor tissues and the level of tumor infiltration was associated with objective responses.

Best Overall Response – Phase 1b Cohort A

¹ Ovarian cancer patient A-DL5-04 erroneously received one dose of nivolumab and is part of intent-to-treat population (shown here) but not per-protocol population; NET: Neuroendocrine Tumor; PD: Progressive disease; SD: Stable disease; PR: Partial response; cPR: Confirmed partial response; BL: Baseline; BOR: Best Overall Response

Response over Time – Phase 1b Cohort A

Manufacturing of IMA203 TCR-T cells

• Immatics’ proprietary manufacturing process has a manufacturing time of 7 days (+7-day expedited release testing), with a success rate of 94% in achieving the provisional RP2D.
• Manufacturing improvements (including monocyte depletion) and higher applied cell doses implemented for the Phase 1b part of the trial led to significantly increased levels of IMA203 T cells in the blood of patients in Phase 1b Cohort A compared to patients in the Phase 1a dose escalation.
• Immatics is currently building a state-of-the-art facility designed to manufacture ACTengine® IMA203 TCR-T products, as well as other cell therapy candidates, for registration-directed trials and initial commercial supply. Built with flexibility and cost-efficiency in mind, the facility is designed to be scalable via a modular design to accommodate manufacturing demands. The facility is expected to be operational in 2024.

Development strategy to realize the multi-cancer opportunity PRAME

Immatics believes, the results presented today further validate PRAME as one of the most promising solid tumor targets for TCR-based therapies. Immatics’ IMA203 development strategy is based on two pillars aimed initially at a (1) fast-to-market approach and, later at a (2) broad development.

The first objective is to deliver the PRAME-targeted TCR-T cell therapy in 1-2 last-line solid cancer types as fast as possible with a focus on indications with PRAME prevalence above 80% and where clinical proof-of-concept has been demonstrated, such as cutaneous melanoma (potentially bundled with uveal melanoma) and/or ovarian cancer. The buildout of the manufacturing facility will support Immatics’ efforts to maximize speed to market. Immatics plans to start a first Phase 2 trial in 1H 2024, which is intended to be designed as a registration-directed trial.

As a second step, Immatics plans to also expand development to other cancer types, such as uterine cancer, lung cancer, breast cancer, head and neck cancer and other tumor types having a broad patient reach.

An update on all three IMA203 Phase 1b Cohorts and clinical development path for PRAME TCR-T monotherapy towards registration-directed trials and potential commercialization is planned for 4Q 2023.

In addition to ACTengine® TCR-T, Immatics is addressing PRAME-positive cancers with a second therapeutic modality, TCR Bispecifics (TCER®), to leverage the full potential of the multi-cancer opportunity PRAME. Immatics’ TCER® IMA402 is a next-generation, half-life extended TCR Bispecific for which Immatics submitted a clinical trial application (CTA⁴) to the Paul-Ehrlich-Institute (PEI) on April 14, 2023, to initiate the Phase 1/2 trial. The trial is expected to commence in 2H 2023 with first clinical data planned in 2024.

Both approaches, ACTengine® and TCER®, are distinct therapeutic modalities that have the potential to provide innovative treatment options for a variety of cancer patient populations with different medical needs. Immatics will continue to evaluate which of these therapeutic modalities (ACTengine® vs. TCER® or both) is best suited for each cancer type.

Immatics conference call
Immatics will host a conference call today, May 2^nd, 2023, at 8:30 am EDT / 2:30 pm CEST to discuss the clinical data. The webcast and presentation can be accessed directly through this link. Participants may also access the slides presented in the webcast on the Immatics website in the Investors section under “Presentations” at www.investors.immatics.com/events-presentations. A replay of the webcast will be made available shortly after the conclusion of the call and archived on Immatics website for at least 90 days.