EDAP Announces Completion of Enrollment in Phase 3 Study Evaluating Focal One HIFU Therapy for the Treatment of Deep Infiltrating Rectal Endometriosis

Rhea-AI Summary

EDAP TMS SA has completed enrollment in its Phase 3 study evaluating Focal One High-Intensity Focused Ultrasound (HIFU) therapy for the treatment of deep infiltrating rectal endometriosis. The study, which included a sham-controlled arm, is expected to yield results in the second half of 2024. Positive results from the Phase 2 study showed a significant decrease in symptoms and improved quality of life for patients.

Positive

• Completion of enrollment in Phase 3 study is a significant clinical milestone
• Highly encouraging robust pace of enrollment in the study
• Positive results from Phase 2 study showed a significant decrease in symptoms and improved quality of life for patients

Negative

• None.

Insights

Medical Research Analyst

The completion of Phase 3 enrollment for EDAP's Focal One HIFU therapy presents a pivotal moment in the advancement of treatment options for rectal endometriosis. This condition, which is often managed through surgery, could potentially be addressed with a less invasive alternative, thus reducing the burden on patients. The previous Phase 2 results indicating a positive safety profile and consistent symptom improvement post-treatment suggest that the therapy could be a game-changer in women's health. The significance of this study lies not only in its potential to offer a new treatment but also in its design—a randomized, double-blind, sham-controlled trial—which strengthens the validity of the results. The focus on Quality of Life measures is particularly noteworthy, as it encompasses a holistic view of patient well-being beyond mere symptom management.

Healthcare Market Analyst

From a market perspective, the successful completion of Phase 3 enrollment within a relatively short timeframe underscores strong interest and demand for non-surgical alternatives for rectal endometriosis. This interest is likely driven by the chronic nature of the disease, the invasiveness of current treatments and the associated recovery times. EDAP's Focal One HIFU therapy positions the company at the forefront of a niche market with a significant unmet need, potentially leading to a competitive advantage. Should the Phase 3 results replicate the safety and efficacy observed in Phase 2, EDAP could see a substantial impact on its market share and valuation, given the size of the affected patient population. Investors will be closely monitoring the upcoming results for indications of the therapy's market viability and long-term revenue potential.

Financial Analyst

With the anticipated release of the Phase 3 study results in the second half of 2024, investors should consider the implications for EDAP's financials. Positive outcomes could lead to increased investor confidence, potentially driving up stock prices. However, it is essential to weigh the risks, as the development and approval process for medical treatments is inherently uncertain and can impact company expenses and revenue projections. The market's response to these results will depend on the data's strength and how it compares to existing treatments. If the therapy is approved, the company's growth trajectory could be significantly altered, with potential for increased R&D costs in the short-term balanced by long-term revenue through product commercialization.

• 60 Patients Completed Enrollment in Under 9 Months
• Study Results Expected in the Second Half of 2024

LYON, France, February 1, 2024 - EDAP TMS SA (Nasdaq: EDAP), the global leader in robotic energy-based therapies, announced today that it has completed enrollment in its Phase 3 study (Endo-HIFU-R2) evaluating Focal One High-Intensity Focused Ultrasound (HIFU) therapy for the treatment of deep infiltrating rectal endometriosis. The ongoing Phase 3 study (NCT05755958) is a comparative, randomized, double blind trial, with the primary objective of evaluating acute pelvic pain levels in 60 patients. Select treated patients are followed three months post-HIFU treatment in comparison to a sham group. The last patient was treated in January. Study results are expected in the second half of 2024.

“We are pleased to announce the completion of enrollment in our Phase 3 study, which represents a significant clinical milestone for our company,” said Ryan Rhodes, Chief Executive Officer of EDAP TMS. “Considering the fact that the Phase 3 study included a sham-controlled arm, we are highly encouraged by the robust pace of enrollment which we believe speaks to the strong interest from patients and physicians in exploring alternative treatment modalities beyond surgery. Rectal endometriosis is a painful and debilitating condition that impacts thousands of women each year with surgical intervention being the primary option beside medical treatment. By utilizing robotic HIFU, we hope to address this significant unmet need in women’s health by developing a safe and effective treatment option that is significantly less invasive than conventional surgical approaches. We would like to thank the patients and clinical investigators who participated in this study, and we look forward to reporting results from the study later this year.”   

Rectal endometriosis induces lesions associated with painful symptoms that can seriously alter quality of life for many women. Focal One HIFU is a non-invasive ablative procedure using a high-intensity ultrasound probe to deliver tissue devitalization through use of acoustic cavitation and thermal ablation.

In January 2022, EDAP reported positive results from the Phase 2 Endo-HIFU-1R study (N=60) evaluating Focal One HIFU for the treatment of deep infiltrating rectal endometriosis. Results from the study showed a positive safety profile with 96.7% of patients with no or non-significant adverse events (Clavien 1), 3.3% of treated patients presenting Clavien 2 complications and zero patients presenting Clavien 3 complications. The study also evaluated the effect of HIFU treatment on endometriosis symptoms and Quality of Life (QoL). Results showed a significant decrease of the evaluated symptoms (acute pelvic pain, dyspareunia, diarrhea, constipation, rectal bleeding, false urges, tenesmus, rectal spams, posterior pelvic pain and asthenia) from the first post-treatment evaluation (at one month), and the reduction of symptoms was maintained at three and six months following HIFU treatment. With respect to QoL measurement, a significant improvement was also observed from the first month after HIFU treatment and maintained at three and six months after treatment for almost all evaluated criteria: physical functioning, role limitation due to emotional problems, energy – fatigue, emotional well-being, social functioning, bodily pain, general health, and on physical and mental global score components. The study also blindly evaluated the evolution of nodule volume via MRI, noting a significant reduction of the volume of lesions observed at six months.

About Endometriosis

Endometriosis is a chronic, progressive disease affecting nearly 10-12% of women of reproductive age. The disease is characterized by tissue resembling the lining of the uterus growing outside the uterine cavity. This extraneous endometrial tissue may commonly occur in the peritoneum or in pelvic and extra-pelvic organs such as the bowels, appendix, bladder, diaphragm muscle and thoracic cavity. The space between the uterus and the rectum, known as the Douglas pouch, is one of the most frequent and symptomatic sites of endometriosis leading to rectal endometriosis.¹

¹ Source: https://drseckin.com/rectal-endometriosis/

About EDAP TMS SA

A recognized leader in the global therapeutic ultrasound market, EDAP TMS develops, manufactures, promotes and distributes worldwide minimally invasive medical devices for various pathologies using ultrasound technology. By combining the latest technologies in imaging and treatment modalities in its complete range of Robotic HIFU devices, EDAP TMS introduced the Focal One^® in Europe and in the U.S. as an answer to all requirements for ideal prostate tissue ablation. With the addition of the ExactVu™ Micro-Ultrasound device, EDAP TMS is now the only company offering a complete solution from diagnostics to focal treatment of Prostate Cancer. EDAP TMS also produces and distributes other medical equipment including the Sonolith^® i-move lithotripter and lasers for the treatment of urinary tract stones using extra-corporeal shockwave lithotripsy (ESWL). For more information on the Company, please visit http://www.edap-tms.com, us.hifu-prostate.com and www.focalone.com.

Forward-Looking Statements

In addition to historical information, this press release contains forward-looking statements within the meaning of applicable federal securities laws, including Section 27A of the U.S. Securities Act of 1933 (the “Securities Act”) or Section 21E of the U.S. Securities Exchange Act of 1934, which may be identified by words such as “believe,” “can,” “contemplate,” “could,” “plan,” “intend,” “is designed to,” “may,” “might,” “potential,” “objective,” “target,” “project,” “predict,” “forecast,” “ambition,” “guideline,” “should,” “will,” “estimate,” “expect” and “anticipate,” or the negative of these and similar expressions, which reflect our views about future events and financial performance. Such statements are based on management's current expectations and are subject to a number of risks and uncertainties, including matters not yet known to us or not currently considered material by us, and there can be no assurance that anticipated events will occur or that the objectives set out will actually be achieved. Important factors that could cause actual results to differ materially from the results anticipated in the forward-looking statements include, among others, the clinical status and market acceptance of our HIFU devices and the continued market potential for our lithotripsy and distribution divisions, as well as risks associated with the current worldwide inflationary environment, the uncertain worldwide economic, political and financial environment, geopolitical instability, climate change and pandemics like the COVID 19 pandemic, or other public health crises, and their related impact on our business operations, including their impacts across our businesses or demand for our devices and services.

Other factors that may cause such a difference may also include, but are not limited to, those described in the Company's filings with the Securities and Exchange Commission and in particular, in the sections "Cautionary Statement on Forward-Looking Information" and "Risk Factors" in the Company's Annual Report on Form 20-F.

Forward-looking statements speak only as of the date they are made. Other than required by law, we do not undertake any obligation to update them in light of new information or future developments. These forward-looking statements are based upon information, assumptions and estimates available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete.

Company Contact
Blandine Confort
Investor Relations / Legal Affairs
EDAP TMS SA
+33 4 72 15 31 50
bconfort@edap-tms.com

Investor Contact
John Fraunces
LifeSci Advisors, LLC
(917) 355-2395
jfraunces@lifesciadvisors.com

FAQ

What is the status of EDAP TMS SA's Phase 3 study enrollment?

EDAP TMS SA has completed enrollment in its Phase 3 study evaluating Focal One High-Intensity Focused Ultrasound (HIFU) therapy for the treatment of deep infiltrating rectal endometriosis.

When are the study results expected?

The study results are expected in the second half of 2024.

What were the results of the Phase 2 study for Focal One HIFU?

Positive results from the Phase 2 study showed a significant decrease in symptoms and improved quality of life for patients.

What is the primary objective of the Phase 3 study for EDAP TMS SA?

The primary objective of the Phase 3 study is to evaluate acute pelvic pain levels in 60 patients.

What is the ticker symbol for EDAP TMS SA?

The ticker symbol for EDAP TMS SA is EDAP.