Biophytis

Biophytis is a clinical-stage biotechnology company focused on the development of transformative therapies for diseases of aging, particularly sarcopenia (the age-related loss of muscle mass and strength, even in people who perform resistance training) and its interface with obesity. Its CEO is Stanislas Veillet; its CSO is Pierre J. Dilda; its CMO is Samuel Agus, M.D.

Biophytis’ longevity platform page delineates the Hallmarks of Aging, but goes on to say “We explore and identify the fundamental mechanisms of aging by targeting pathways like Mas-related G protein-coupled receptor (MasR), which is part of the renin angiotensin system [RAS], Peroxisome proliferator-activated receptor pathway and key inflammatory and angiogenic signaling routes, including NF-κB and AP-1 transcription factors.” Elsewhere, they say they have “a unique platform that combines synergistic technology modalities systematically accelerated by AI through our partnership with Lynx Analytics. This enables us to discover therapeutic solutions across the aging disease spectrum.”

BIO101 and possibly Biophytis itself originated with SARCOB, a consortium of French companies and academic laboratories formed to develop drug and dietary supplement candidates for sarcopenic obesity. Members of the consortium tested various candidates in murine and human muscle cell lines as well as myocye-adipocyte co-cultures and in different rodent models, and the receptor for phytoecdysteroids in muscle was identified.

From this emerged Biophytis’ most advanced candidate: 20-hydroxyecdysone, a phytoecdysteroid that plays a key role in insect development and activates the MAS receptor in mammals. The MAS receptor is a node in the protective alternative RAS pathway that counteracts the effects of the classical RAS pathway centered on AT1R. MasR’s principal ligand is Ang 1-7, a bioactive metabolite produced by ACE2.

Labeled 20-hydroxyecdysone dietary supplements have been marketed as anabolic agents since the 1980s, but independent testing found 10 out of 12 tested supplements contained single-digit percentages of the labeled amount, and a followup study using one of the supplements from that study that approximated its label claim found that a later lot of the same product again contained a fraction of the labeled amount.

Under the name BIO101 (also known as Sarconeos or Ruvembri for some indications and time periods), Biophytis is developing 20-hydroxyecdysone for indications related to sarcopenia and obesity, as well as for COVID-19 and Duchenne muscular dystrophy (DMD).

In June 2024, Biophytis announced that it had transferred production of BIO101 to Seqens, an integrated global producer of active pharmaceutical ingredients and intermediates and of specialty products. Its first batch of GMP-compliant BIO101 was to be used in Biophytis’ clinical development program for DMD.

BIO101 (AKA Sarconeos and Ruvembri) for Sarcopenia
In April 2022, Biophytis-affiliated scientists presented the results of the Phase II SARA-INT trial of BIO101 for sarcopenia (NCT03452488). 233 participants were randomized into one of three arms: 175 mg BIO101 twice daily, 350 mg twice daily, or placebo. For most subjects, the trial lasted 6 months, but for 50 subjects it was extended to a total of 9 months. The primary endpoint was the 400-meter walking test (400MWT), with additional physical activity assessments as secondary endpoints.

BIO101 failed in the primary analysis, perhaps in part because approximately half of the subjects’ final assessments were missing due to the COVID pandemic, thereby lowering the trial’s statistical power. There was a nonsignificant trend toward an improvement of 0.07 m/s in the 400MWT of the 350 mg twice daily group at 6 months, and a nominally significant improvement of 0.09 m/s in this group in the per-protocol population (p=0.008); the investigators noted that this result, if not an artifact, approaches the Minimal Clinically Important Difference (MCID) in sarcopenia of 0.1 m/s. This result also held in predefined high-risk subpopulations, and the abstract stated that the investigators had observed trends in unspecified additional endpoints and subgroups.

In May 2023, Biophytis announced that it had submitted an application to the European Medicines Agency (EMA) for Clinical Trial Authorization (CTA) to launch the Phase III SARA-31 trial, the first Phase III trial in sarcopenia. In September 2023, Biophytis announced that FDA had granted it approval to initiate the SARA-31 trial in the US.

Similar to SARA-INT, SARA-31 will evaluate BIO101 in approximately 900 people over the age of 65 years with severe sarcopenia who are at risk of mobility disability. Treatment will continue for at least a year and up to three. The primary endpoint will be a version of the 400MWT, with secondary endpoints including the 4 minute walking speed from the Short Physical Performance Battery (SPPB) test, grip strength, and patient-reported quality of life (on the sarcopenia-specific Patient Reported Outcome SarQol questionnaire.

In March 2025, Biophytis announced the publication of SARA-INT. The full publication added some information on subgroup analyses and highlighted BIO101’s good safety profile.

In July 2025, Biophytis announced a strategic partnership with Lynx Analytics, a company working in artificial intelligence solutions for life sciences, “to accelerate the discovery and development of novel small molecule drugs for the treatment of sarcopenia”.

In October 2025, Biophytis announced that it had inked a Memorandum of Understanding (MoU) with a consortium of investors that included Ronghui Renhe Life Technology to finance and launch the SARA-31 trial. The company stated that it anticipates commencement early in 2026. Under the terms, the investors are to invest up to US$20 million over the following two years to fund the trial and have the right to develop and exclusively commercialize BIO101 in China, Japan, and Korea. Under the MoU, Biophytis will recruit up to 932 subjects with sarcopenia and will include subjects in Asia in addition to Europe.

BIO101 for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is caused by mutations in the gene for dystrophin, a key part of a protein complex that anchors muscle fibers to the extracellular matrix. These mutations typically introduce a premature stop codon into the dystrophin mRNA, resulting in a truncated, dysfunctional protein. Without a functional dystrophin protein, muscle fibers necrotize, leading to progressive muscle weakness and high fatigue, leaving most children with DMD wheelchair-bound and often also with respiratory impairment and cardiac hypertrophy.

In May 2018,  Biophytis announced that FDA had granted BIO101 orphan drug designation for DMD. Shortly thereafter, they announced that the European Medicines Agency (EMA) had followed suit.

At the 2019 Myology conference, Biophytis-affiliated scientists presented preclinical studies on BIO101 in the mdx mouse, an X-linked muscular dystrophy mouse model of DMD. Relative to cyclodextrin vehicle controls, BIO101-treated mice exhibited reduced loss of running endurance, a slight increase in isometric strength, reduced muscle fiber atrophy and necrosis, sustained lung mechanical function, and reduced respiratory pausing (Penh).

In October 2019, Biophytis-affiliated researchers presented a proposal for a clinical trial of BIO101 in DMD that would combine Phases I through III “in one operational seamless study with adaptive elements.” DMD patients would be “recruited” (the proposal did not say “randomized”) into one of the four ascending multiple-dose cohorts in Part 1, where they would remain for an additional 24 weeks in Part 2 (serving as Phase II), from which researchers would select the dose for the pivotal Part 3. All patients from Parts 1 and 2, plus additional patients, would then enter a 48-week Phase III-equivalent stage (Part 3) to establish long-term safety and efficacy.

In December 2019, Biophytis announced that FDA had granted it Investigational New Drug (IND) approval to initiate a Phase I/II clinical proof-of-concept trial (MYODA-INT) of an oral pediatric formulation of BIO10 for DMD1, using a ‘seamless’ clinical trial design with composite clinical endpoints. It is not clear how this correlates with the design proposed in the October meeting presentation. This trial was expected to begin early in 2023.

In March 2024, Biophytis stated its intention to initiate a Phase I/II clinical trial in non-ambulatory DMD patients suffering from respiratory failure by the end of that year.

As of November 2025, the status of this trial is unclear to us. Biophytis’ dedicated DMD page is no longer maintained, but MYODA DMD is present and described on its “other projects” pipeline page, which indicates it is still preclinical.