MedDay and DSM announce a partnership to produce pharmaceutical grade D-Biotin to treat progressive multiple sclerosis
The partnership covers the manufacturing and exclusive supply by DSM for MedDay’s lead product MD1003, a pharmaceutical grade D-Biotin, which is currently being investigated in a series of Phase III clinical trials.
This partnership follows the announcement by MedDay in April 2015 of positive results from the pivotal Phase III clinical trial, MS-SPI in patients suffering from progressive multiple sclerosis (MS). The Phase III study demonstrated evidence of the efficacy and safety of MD1003, a highly-concentrated pharmaceutical-grade biotin administered at a dose of 300 mg per day in the treatment of primary and secondary progressive MS, a major area of unmet medical need.
MedDay’s MD1003 will require a chronic administration of very high dose of biotin to treat patients with progressive MS, corresponding to 10,000 times the recommended daily intake, and therefore a pharmaceutical grade extra-pure source of biotin is required. Different sources of D-Biotin currently exist for low-dose food supplements or veterinary use. DSM is currently producing the only pharmaceutical grade biotin having a CEP (Certificate of suitability of Monographs of the European Pharmacopoeia) suitable for chronic administration of high doses in patients.
The partnership will also cover the full support from DSM for regulatory filings in Europe and USA as well as an investment in a dedicated production facility at a DSM manufacturing site in Europe for the industrial scale production of pharmaceutical grade D-Biotin.
Frédéric Sedel, Chief Executive Officer of MedDay, said: “Our partnership with DSM to secure the manufacturing of large amounts of a pharmaceutical grade D-Biotin represents a significant milestone for MedDay. With more than 50 years of experience in the synthesis of biotin, we believe DSM is an excellent partner to supply us with a high-quality active pharmaceutical ingredient (API), and to support us through a regulatory filing.”
Christoph Goppelsroeder, President & CEO, DSM Nutritional Products, commented: “As a science-based company, DSM is delighted to partner with MedDay in bringing this novel treatment to patients worldwide. We see an increasing number of vitamin applications emerging as pharmaceutical products for so-far difficult to treat illnesses. This is a very promising field in which we are proud to take the lead.”
MedDay is a privately held biotechnology company developing new drugs for nervous system disorders. The company was founded in 2011 by Frédéric Sedel, MD, PhD (Chief Executive Officer); and Guillaume Brion, MD (Chief Operating Officer). In April 2013, InnoBio, a biotechnology fund managed by BPIFrance, and Sofinnova Partners together invested in MedDay. The Company’s most advanced pipeline candidate is MD1003 for the treatment of primary and secondary progressive multiple sclerosis. For more information, please see: www.medday-pharma.com.
MD1003 is an investigational medicine thought to have both pro-myelinogenic effects and to enhance the supply of energy for nerve impulse transmission. MD1003 is an active pharmaceutical ingredient administered at a dose of 300 mg /day has patent protection in EU and US for dose and use in multiple sclerosis. MD1003 has a mode of action which potentially influences two targets related to progressive MS: (1) it activates acetyl-CoA carboxylases (ACC1 and ACC2), the rate-limiting enzymes in the synthesis of fatty acids required for myelin synthesis, and (2) it activates the Krebs cycle in demyelinated axons to increase energy production.
The MS-SPI study is a randomized 2:1, double-blinded, placebo-controlled study conducted in 16 MS reference centres in France. Treatment duration was one year. The patient population was defined as patients suffering from primary progressive Multiple Sclerosis (PPMS) or secondary progressive Multiple Sclerosis (SPMS) with EDSS (Exepended disability scale status) progression in the two years prior to inclusion and with EDSS ranging from 4.5 to 7. 154 patients were randomized (103 in the MD1003 arm and 51 in the placebo arm). The primary endpoint was met (p=0.0051) in the intent to treat population with 12.6% of patients in the MD1003 arm showing an improvement of EDSS (Expanded Disability Status Scale) or TW25 (a timed 25-foot walk) at Month 9, confirmed at Month 12, compared to none of the patients (0%) in the placebo arm. The primary endpoint was supported by secondary analyses showing evidence for a decrease in the risk of disease progression. The mean change of EDSS between M0 and M12 decreased in the MD1003 group (-0.03) compared to progression in the placebo group (+0.13, p=0.014). In the MD1003 arm, only 4% of patients treated with MD1003 exhibited EDSS progression at M9 confirmed at M12 vs 13% in the placebo group (p=0.07), which equates to a 67% decreased risk of progression in the active arm within the studied period. Results have been presented during the clinical trials plenary session this year at the American Academy of Neurology congress.
About progressive MS
MS is the most common disabling neurological disease among young adults, with first symptoms typically manifesting between 20 and 40 years of age. In the majority (85%) of cases, patients experience an initial phase of relapsing-remitting neurological dysfunction (RRMS), which typically evolves into a secondary progressive disease at a later point in the clinical course (SPMS). Once MS is in the progressive phase, individuals experience a gradual worsening of neurological disability leading to problems with vision, walking, incontinence, cognitive changes, fatigue, and pain. Primary progressive MS (PPMS) characterized by disease progression from onset is less common, affecting 10–15% of patients. The overall prevalence of patients with progressive disease is estimated to be at least 40% of all MS patients i.e. around 350,000 patients in Europe and USA.