MedDay Raises More Funds For Experimental MD1003 Multiple Sclerosis Drug.


Source: Fierce Biotech

MedDay MD1003 Experimental Medication:

MD1003 is an experimental drug that has pro-myelinotic properties and is thought to improve the supply of energy for nerve impulse transmission. It acts by activating acetyl-CoA carboxylases (ACC1 and ACC2), which are the rate-limiting enzymes in the fatty acids synthesis required for myelin formation, and by activating the Krebs cycle (a series of chemical reactions used by all aerobic organisms to generate energy through the oxidation of  derived from carbohydrates, fats and proteins into carbon dioxide and chemical energy in the form of adenosine) in demyelinated axons to increase energy production.

The MS-SPI study is a multicenter, randomized, double-blind, placebo-controlled trial assessing MD1003 (300 mg/day) in patients with progressive MS in 16 different medical centers in France. In total, 154 MS patients who had a baseline Expanded Disability Status Scale (EDSS) score of 4.5 to 7 were evaluated. The study was carried out for 12 months.

The News:

MedDay has raised $38.5 million in a Series B funding round as it looks toward finishing up late-stage trials for its lead multiple sclerosis candidate–which the company is touting as a “breakthrough drug.”

The French biotech told FierceBiotech the money was “additional security” and comes as it prepares to seek European approval for its multiple sclerosis drug candidate MD1003, while also helping MedDay to conduct a confirmatory Phase III study in the U.S. This builds on the $8.5 million it raised back in 2013.

Dr. Frédéric Sedel, MedDay’s CEO, said that while there is a big and growing market for MS, MD1003 will not be entering into a crowded market as it targets progressive forms of MS, where no drugs are currently approved. Most new treatments are licensed for relapsing-remitting forms of the disease.

“The big players aren’t providing drugs for this market [progressive forms of MS]–but it’s not a small market; we think it makes up around half of all cases. So we are looking to move into a market with no rivals and utilize the drug’s unique action that targets the nervous system, but not the immune system, as other treatments do.

“It can not only slow down inflammation but also reverse it in a significant number of patients–so I think it’s a real breakthrough drug in this field.”

The near-term focus will be on Europe and filing with the EMA, Sedel said, where the drug is currently being supplied by MedDay on a named patient basis to around 1,000 patients–something which is bringing in early revenue for the company, and what Sedel describes as a “validation of our approach with the drug.”

He added that the company aims to file with the EMA “within the next month.” The drug hit a setback last year after missing an endpoint in a Phase III vision loss trial. But the company did find efficacy in another arm of the study, leading it to re-focus on progressive forms of MS.

An IPO “would be the logical next step”, Sedel said, and would help pay toward a future launch–but given the current environment for public offerings, which has seen a number of companies sell their shares at a much lower rate than they want, Sedel said he would wait until the headwinds died down before going public.

In terms of partnering, Sedel said the company is independent but adds that it is in discussions with other drugmakers about its lead candidate. “But as this time, I would say we don’t need them. Once we have approval and need to sell the drug, we may look for a commercial partner to help us in certain regions; however I think we can move forward as a standalone company.”

The Data:

Data for the drug is based from a Phase III trial, which looked at 144 participants with primary or secondary progressive MS. The patients were split into two groups, receiving either 300 mg of MD1003 daily for 24 months or a placebo for a year, followed by 300 mg of MD1003 daily for 12 months.

Researchers were measuring whether participants had any improvement in disability at 9 months of treatment that was still apparent at 12 months. The trial data, released last April, showed 13% of the treatment group compared to none of the placebo group met this criteria demonstrating some improvement in the treatment group.

MedDay has also carried out a Phase III trial testing the effectiveness of MD1003 as a treatment for optic neuritis. In all, 93 participants with MS who experience optic neuritis (progressive and nonprogressive forms) were again split into two groups.

The aim was to test the effect of MD1003 on the clearness of vision and the thickness of the retina (the light sensitive nerve fiber layer at the back of the eye). The results did show that MD1003 had some improvement compared to placebo–but this was not significant, and the overall study did not show positive results.

The group that did see the most benefit were those with progressive forms of optic neuritis, and was why the company decided to refocus its future Phase III efforts confirming the drug’s efficacy in patients with progressive forms of MS.

MedDay has also released a small, early-stage pilot study involving 23 participants with primary or secondary progressive MS that demonstrated that high doses of MD1003 may lead to improvements in disability.

This was an open-label study, in which patients were given 100-300 mg of MD1003 per day for 2-36 months. In total, 21 out of the 23 taking part showed evidence of some kind of improvement, including improved vision, cognition or walking.

Edmond de Rothschild Investment Partners (EDRIP) led the new investment round alongside existing investors Sofinnova Partners and InnoBio (Bpifrance). Large Venture (Bpifrance) also participated in the operation.

The company is also developing a number of other early-stage pipeline candidates as well as the advancement of a research platform aimed at identifying new metabolic targets in neurological diseases.

MedDay Phara LogoMedDay is a biotechnology company developing new drugs for nervous system disorders. The company was founded in 2011 by Frédéric Sedel, MD, PhD (Chief Executive Officer), a leading neurologist and neuroscientist; and Guillaume Brion, MD (Chief Operating Officer), who has 25 years of experience in drug development and clinical research in the pharmaceutical industry. In April 2013, InnoBio, a biotechnology fund managed by BPIFrance, and Sofinnova Partners together invested a total of €8 million in MedDay.

MedDay addresses areas of unmet need in neurological and psychiatric diseases through the brain metabolism. This approach relies on unique clinical and pharmaceutical development expertise in the field of rare inborn errors of metabolism.

Our vision is to:

  • Treat neurological diseases and manifestations by treating metabolic pathway modifications.
  • Develop compounds that have a known activity in neurological metabolic pathways.
  • Focus on fast and meaningful clinical proof of concept in a small number of patients. We believe that, if a drug has an effect on a neurodegenerative disease, we should be able to demonstrate the effect a small group of patients. Once the proof of concept is established, the results have to be confirmed in properly designed phase 2/3 placebo-controlled studies involving a larger number of patients.
  • Build Intellectual Property based on formulation/use patents.
  • Develop strong academic collaborations to decipher the mechanism of action of the selected drugs in adequate models.

Our ethics

  • MedDay prioritizes diseases of the central nervous system with high unmet medical need.
  • We place patients and their families at the heart of our mission: our approach is designed to bring safe and efficacious drugs to patients in a short period of time.
  • To achieve this goal, MedDay mitigates the risks of early development by:
    1. Investigating diseases directly in patients’ cerebrospinal fluid (through the SPECMET platform);
    2. Using molecules of the pharmacopeia with an acceptable safety profile;
    3. Focusing on the clinical proof of efficacy in a small number of patients before moving to full pharmaceutical development.