Biotechs record clinical trial successes

Noema Pharma, a clinical-stage biotech targeting debilitating neuroscience disorders, has treated the first patient in an open-label Phase 2a study (NOE-PPM-201) evaluating the safety and efficacy of NOE-115, a broad-spectrum monoamine modulator, in women with vasomotor symptoms (VMS, also known as "hot flashes") and other symptoms such as weight gain, daytime sleepiness and cognitive difficulties due to menopause. The study will initially evaluate 30 women with moderate to severe VMS at 6 centers in the U.S.

"The initiation of this study marks our fifth Phase 2 program to enter the clinic as we seek to develop transformative medicines for underserved conditions which are rooted in neuroscience,” said Ilise Lombardo, CEO of Noema. “With over 2 million women in the U.S. entering menopause annually, there is significant medical need for a treatment option capable of addressing a broad constellation of symptoms that accompany the menopause transition, so that women are able to maintain their health and quality of life. These include vasomotor symptoms as well as weight gain, significant daytime fatigue and cognitive difficulties.”

Phase 3 for MoonLake’s inflammatory disease treatment
MoonLake, a clinical-stage biotechnology company developing therapies for inflammatory diseases, announced that the first patients have been enrolled in a U.S. study center as part of its global Phase 3 VELA clinical program evaluating sonelokimab, a Nanobody® for the treatment of inflammatory diseases, in patients with moderate to severe hidradenitis suppurativa (HS). HS is a severely debilitating chronic skin disease that affects up to 4.1% of the world's population. The initiation of this Phase 3 program follows the announcement of the successful outcome of MoonLake's Phase 2 interactions with the US Food and Drug Administration (FDA) in February 2024 and positive feedback from interactions with the European Medicines Agency (EMA), with both regulatory authorities unanimously supporting MoonLake's proposed approach to advancing its Phase 3 program for HS.

Kristian Reich, Founder and CSO at MoonLake commented: “The launch of our Phase 3 VELA program with our Nanobody® sonelokimab, using the higher clinical measure of HiSCR75 as the primary endpoint and a straightforward, proven study design is a landmark moment in our efforts to develop novel treatment options for patients suffering with this under-diagnosed and under-treated condition. We are making significant progress in establishing clinical trial sites to enroll 800 patients, and we eagerly anticipate reporting the week 16 primary endpoint readout around mid-2025.”

Timeline completed its MitoImmune clinical trial
Longevity startup Timeline, developing solutions for healthy aging and longevity, has successfully completed its immune aging clinical trial, referred to as MitoImmune, using Mitopure (proprietary urolithin A), a postbiotic nutrient shown to trigger a crucial recycling process within cells (mitophagy), targeting age-related cellular decline. Mitopure can be applied in the form of softgels, skin cremes or powders.

The MitoImmune study is a randomized, placebo-controlled trial conducted in collaboration with the Georg Speyer House Institute for Tumor and Experimental Therapy and the Buck Institute of Aging in California. Fifty middle-aged adults were enrolled for a four-week intervention with Mitopure. The co-primary endpoints of the study were an increase in youth naïve T cells and an improvement in their mitochondrial activity, which were successfully achieved with the Mitopure intervention compared to placebo. Other important secondary endpoints (including reduction in inflammatory response) were also achieved.

In addition to MitoImmune, Timeline has partnered on two new clinical trials - one in collaboration with the National Institute of Aging and one with the National Institute of Cancer - to expand the applications of Mitopure for healthy aging.

Nature Medicine published ONWARD’s pivotal trial results
ONWARD Medical is developing innovative spinal cord stimulation therapies to restore movement, function and independence in people with spinal cord injuries and movement disorders. The company has now announced that the results of its Up-LIFT pivotal trial have been published in Nature Medicine, one of the world's leading scientific journals. The global Up-LIFT study was conducted until 2022 to evaluate the safety and efficacy of ONWARD's ARC-EX® therapy for improving upper limb mobility in people with chronic incomplete quadriplegia. It was conducted with 65 participants at 14 leading SCI centers in the U.S., Europe and Canada.

This publication represents a developmental milestone that supports the company's goal of addressing the unmet needs of patients with spinal cord injury (SCI). Key findings indicated that ARC-EX therapy was safe and effective, 90% of participants improved either strength or function of their upper limbs, 87% of participants reported an improvement in quality of life, and improvement was detectable in participants up to 34 years post-injury. Study participants also reported reduced frequency of spasms, improved sleep and improved sensation in the upper body, including the sense of touch. According to Marco Baptista, chief scientific officer of the Christopher & Dana Reeve Foundation, the Up-LIFT study results represent a turning point in the field of SCI and paralysis science. They believe that functional recovery which was once deemed impossible may now be accessible.

The company has now applied to the FDA for U.S. regulatory clearance of the ARC-EX System with hopes to offer ARC-EX Therapy to people with SCI in the U.S. later this year and in Europe in mid-2025.

(Press releases / SR)