dm+d

13813003

New Medicines

QizendayPrimary progressive multiple sclerosis (MS)

Information

Qizenday
New molecular entity
MedDay
Not Known

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Jun 20 Following failure of PIII SP12 trial to meet it´s endpoints development of MD1003 has been discontinued [13].
Mar 20Following data from PIII SP12 trial, the company will will review the findings in detail to understand these outcomes and confer with regulators.[11]
Nov 18MedDay Pharmaceuticals announces it intends to submit NDA and MAA to the US FDA and European Commission in H2 20 [9].
Dec 17MedDay have withdrawn the EU Marketing Authorisation Application for biotin in progressive MS. The CHMP had notified the company that it did not consider the data presented so far were adequate to prove efficacy and safety, and therefore that their opinion was that at that point the benefits were not shown to outweigh the risks. The company has reserved the right to reapply in future [8].
Sep 16Filed in EU via centralised procedure [7].
Apr 16EU filing planned in May 2016 [6].
Nov 14Market launch is expected by 2017 [2].

Category

A water-soluble vitamin acting as a coenzyme for carboxylases involved in key steps of energy metabolism and fatty acid synthesis. Also known as vitamin H, this formulation contains 10,000 times the recommended daily food intake.
The prevalence of MS in England is approximately 0.16% , equivalent to approximately 85,600 affected people. Primary progressive multiple sclerosis (PPMS) affects about 10-15% of people diagnosed with MS , equating to between 8,560 and 12,840 people in England.
Primary progressive multiple sclerosis (MS)
Oral

Further information

Yes

Trial or other data

Mar 20: The PIII SP12 trial did not meet its primary endpoints (reversal of functional disability measured by improvement in EDSS scale and time needed to walk 25 feet (TW25) over 12-15 months). There were no treatment emergent safety signals.[12]


Oct 18: Enrolment completes in PIII SPI2 trial [9].


Nov 16: PIII SPI2 confirmatory trial to evaluate the safety and efficacy of biotin 100mg capsule tid in patients with progressive multiple sclerosis starts (NCT02936037). 660 patients will be recruited in the US, Canada, UK, Germany, Sweden, Spain, Italy, Australia, Belgium, Hungary, Poland, Tukey, and Czech Republic [9].


Apr 15: MedDay announce positive results from pivotal PIII MS-SPI trial. The study demonstrated evidence of the efficacy and safety of MD1003 at a dose of 300mg per day for the treatment of primary and secondary progressive MS. The primary endpoint was met (p=0.0051) with 12.6% of patients in the intention to treat population showing improvement in EDSS or TW25 at month 9, confirmed at month 10, compared to 0% in placebo arm. Secondary analysis showed a decrease in risk of disease progression. 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=0.015). Only 4% of patients treated with MD1003 exhibited EDSS progression at M9 vs. 13% in placebo group (p=0.07). The study was not prospectively powered to reach significance for this secondary endpoint. MD1003 was well tolerated; the overall incidence of events was similar across the two groups [5].


Apr 15: Meday provided further information about the design of its pivotal clinical trial (MS-SPI) to investigate the efficacy and safety of MD1003 in the treatment of primary and secondary progressive MS. MS-SPI is a randomized, double-blind, multicentre, placebo-controlled (2:1) trial of MD1003, 300 mg/day, in 154 patients with progressive MS who have demonstrated progression in the 2 years prior to enrolment. Patients were enroled with a baseline EDSS (Expanded Disability Status Scale) score of between 4.5 and 7 and were treated for one year. The primary endpoint was the proportion of pts who improved at nine months (M9), with confirmation at 12 months (M12). Improvement was defined as either a decrease in EDSS (by at least 1 point for baseline EDSS =5.5 and 0.5 points for EDSS =6) or an improvement in TW25 (a timed 25-foot walk) of at least 20%. The comparison for each outcome was the best EDSS and TW25 scores obtained at the screening and randomisation visits [4].


Mar 15. PIII randomised, double-blind, placebo-controlled trial (NCT02220933) completed. [3]


Oct 13: PIII (NCT02220933; MS-SPI) study begins. The purpose of this study is to demonstrate the superiority of MD1003 over placebo in the disability of patients suffering from progressive MS (primary or secondary) and especially those with gait impairment. Primary outcome is the proportion of patients improved on either Expanded Disability Status Scale (EDSS) or time to walk 25 feet. 144 pts will be recruited in France. Collection of primary outcome data should complete in Jan 15 [1].

Evidence based evaluations

QizendaySecondary progressive multiple sclerosis (MS)

Information

Qizenday
New molecular entity
MedDay
Not Known

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Jun 20Following failure of PIII SP12 trial to meet it´s endpoints development of MD1003 has been discontinued [13].
Mar 20Following data from PIII SP12 trial, the company will will review the findings in detail to understand these outcomes and confer with regulators.[12]
Nov 18MedDay Pharmaceuticals announces it intends to submit NDA and MAA to the US FDA and European Commission in H2 20 [10].
Dec 17MedDay have withdrawn the EU Marketing Authorisation Application for biotin in progressive MS. The CHMP had notified the company that it did not consider the data presented so far were adequate to prove efficacy and safety, and therefore that their opinion was that at that point the benefits were not shown to outweigh the risks. The company has reserved the right to reapply in future [9].
Sep 16Filed in EU via centralised procedure [8].
Apr 16EU filing planned in May 2016 [6].
Nov 14Market launch is expected by 2017 [2].

Category

A water-soluble vitamin acting as a coenzyme for carboxylases involved in key steps of energy metabolism and fatty acid synthesis. Also known as vitamin H, this formulation contains 10,000 times the recommended daily food intake.
The prevalence of MS in England is approximately 0.16% , equivalent to approximately 85,600 affected people. Primary progressive multiple sclerosis (PPMS) affects about 10-15% of people diagnosed with MS , equating to between 8,560 and 12,840 people in England.
Secondary progressive multiple sclerosis (MS)
Oral

Further information

Yes

Trial or other data

Mar 20: The PIII SP12 trial did not meet its primary endpoints (reversal of functional disability measured by improvement in EDSS scale and time needed to walk 25 feet (TW25) over 12-15 months). There were no treatment emergent safety signals.[12]


Oct 18: Enrolment completes in PIII SPI2 trial [9].


Nov 16: PIII SPI2 confirmatory trial to evaluate the safety and efficacy of biotin 100mg capsule tid in patients with progressive multiple sclerosis starts (NCT02936037). 660 patients will be recruited in the US, Canada, UK, Germany, Sweden, Spain, Italy, Australia, Belgium, Hungary, Poland, Tukey, and Czech Republic [9].


Apr 15: MedDay announce positive results from pivotal PIII MS-SPI trial. The study demonstrated evidence of the efficacy and safety of MD1003 at a dose of 300mg per day for the treatment of primary and secondary progressive MS. The primary endpoint was met (p=0.0051) with 12.6% of patients in the intention to treat population showing improvement in EDSS or TW25 at month 9, confirmed at month 10, compared to 0% in placebo arm. Secondary analysis showed a decrease in risk of disease progression. 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=0.015). Only 4% of patients treated with MD1003 exhibited EDSS progression at M9 vs. 13% in placebo group (p=0.07). The study was not prospectively powered to reach significance for this secondary endpoint. MD1003 was well tolerated; the overall incidence of events was similar across the two groups [5].


Apr 15: Meday provided further information about the design of its pivotal clinical trial (MS-SPI) to investigate the efficacy and safety of MD1003 in the treatment of primary and secondary progressive MS. MS-SPI is a randomized, double-blind, multicentre, placebo-controlled (2:1) trial of MD1003, 300 mg/day, in 154 patients with progressive MS who have demonstrated progression in the 2 years prior to enrolment. Patients were enroled with a baseline EDSS (Expanded Disability Status Scale) score of between 4.5 and 7 and were treated for one year. The primary endpoint was the proportion of pts who improved at nine months (M9), with confirmation at 12 months (M12). Improvement was defined as either a decrease in EDSS (by at least 1 point for baseline EDSS =5.5 and 0.5 points for EDSS =6) or an improvement in TW25 (a timed 25-foot walk) of at least 20%. The comparison for each outcome was the best EDSS and TW25 scores obtained at the screening and randomisation visits [4].


Mar 15. PIII randomised, double-blind, placebo-controlled trial (NCT02220933) completed. [3]


Oct 13: PIII (NCT02220933; MS-SPI) study begins. The purpose of this study is to demonstrate the superiority of MD1003 over placebo in the disability of patients suffering from progressive MS (primary or secondary) and especially those with gait impairment. Primary outcome is the proportion of patients improved on either Expanded Disability Status Scale (EDSS) or time to walk 25 feet. 144 pts will be recruited in France. Collection of primary outcome data should complete in Jan 15 [1].

Evidence based evaluations