Progressive MS Pipeline

A board to discuss future MS therapies in early stage (Phase I or II) trials.

Postby shah » Mon Sep 01, 2008 5:00 pm

ladystewart wrote:I am SPMS and sent information to go to Johns hopkins for Cyclophomide. They sent me information that showed a lot of SPMS individuals but--JH told me that they were accepting RRMS. What?? They could not explain.

I am a female and I have 2 sons-my mom had MS--but I don't want my sons to go through what I did. I don't want them to wipe my butt etc. HELP :!:

Sharon


I did 6 months of Cyclophosphamide at a monthly rate of 1000mg, while dignosed as a RRMS case that failed TY. Since I hardly showed any improvement after the 6 month treatment, I was upgraded to SPMS.
I'm currently taking Copaxone+4-AP+Rilutek+LDN+IVSM (monthly) and seem to have been stabilized. The next big gun for me will be novantrone,
which has shown to be more effective in progressive forms of MS.

what a ride..

Regards,
Shah
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Postby notasperfectasyou » Mon Jun 08, 2009 7:13 am

You might want to check with Sarah, but I think that ABX is working for folks with SPMS. At least it is for Kim.
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Postby SarahLonglands » Mon Jun 08, 2009 7:41 am

There are lots of Sarahs, but only one Anecdote. :wink:

Ken is correct, though: many people are using CAP antibiotics for SPMS, like his wife, Kim and also like me, except I have now finished treatment because it was so successful. Have a look at the Antibiotics forum and also send a PM to Katman, who found this treatment very successful for her PPMS.

Sarah
An Itinerary in Light and Shadow Completed Dr Charles Stratton / Dr David Wheldon abx regime for aggressive secondary progressive MS in June 2007, after four years. Still improving with no relapses since starting. Can't run but can paint all day.
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Postby dignan » Fri Aug 28, 2009 5:47 pm

I added PI-2301 to the phase 2 list based on THIS post from scorpion.
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Husband is on the FTY 720 trial phase III PPMS

Postby Astrid » Sat Oct 03, 2009 9:14 am

Hi,

My name is Astrid and I am from The Netherlands.
My husband (Rob) is diagnosed with PPMS in August 2006.
Since January 2009 het is on the new FTY 720 trial for PPMS.
We are 41 en 45 years old, no kids, own business wich I run on my own now.
Rob is getting worse everyday and we think he has side-effects from the FTY (thinking because in this trail you are not sure if you are on the drugs or not).
His EDSS score is 5.0, he doesn't feel his feet or legs, arm and hands but stil can walk about 200-300 meters.
He cann't coordinate his right hand anymore and his left hand is getting worse. He always has tintling (is that the right word?) sensations all over his body and his legs and arms feel very heavy and painfull.
Het had to quit working 2 years ago and is very tired.
But... we still try to enjoy life and when it is possible we go to parties, out for dinner or to friends.

I'd like to know if there is anyone out ther who is on this new trial for PPMS.
How are you feeling?

Thanks and I hope to hear from you!
We try to continue oure life
My husband, Rob, has PPMS, diagnosed august 2006, EDSS score 5.0. He is on the new FTY 720 trial, phase III since January 2009. We are from The Netherlands
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Don't mean to sound so dim but...

Postby jam » Sun Apr 03, 2011 5:32 am

marcstck wrote:I've heard their will be a FTY720-PPMS trial starting later this summer, based on the supposed neuroprotective properties of the drug...


This is an old message I know, but I don't know is has there been any further progress for pp ms? Any hope would be welcomed. I try not to get too caught up in all this but praying it will just happen, when is the probelm!

I am a Kiwi so progress is a little slow here.

The other issue I guess will be the expense once it is converted to NZ dollars.

Thanks for listening.
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PPMS Trials

Postby Smilingface » Sun Apr 03, 2011 6:24 am

Greetings from NC Jam! I have been in the fingoliomod trial for a year with
positive results. My energy level has greatly improved and my neurological
testing is showing increasing strength. I feel like my progression has halted.

Fortunately, my side effects have been temporary and minimal.

Any word from anyone in Europe? The trial started there earlier by 2 years.

I am hopeful that this drug will help all of us who progress and are without
Disease modifying treatment. There are other PP MS trials such as idebenone going on but I have no knowledge of anyone participating.

Keep your hopes up. :)
Primary Progressive, Onset 10 years ago at age 42, diagnosis 6 years ago, Vit D, Chinese Herbs, Exercise, yoga. So far tried antibiotics, fumaric acid and 4AP. Currently participant in the FTY720/PPMS Trial.<br />
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Postby patientx » Tue Apr 05, 2011 7:37 am

From a quick check of the clinicaltrials.gov site, it looks like the fingolimod trial for PPMS is still ongoing, and they are still recruiting:
http://www.clinicaltrials.gov/ct2/show/ ... %22&rank=2

Also, they are recruiting for the Idebenone trial:
http://www.nationalmssociety.org/news/n ... x?nid=4502

This says they may cover travel expenses, so if nothing else, someone could get a trip to the Washington DC area out of it.
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Re: Progressive MS Pipeline

Postby xpsychiatricmd » Sun Feb 10, 2013 4:21 pm

I would add Tcelna (old Tovaxin ) to the list with the Abili-T trial. I see that it hasn't been updated in a while.
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Efficacy and safety of Siponimod (BAF312) in SPMS patients

Postby seeva » Sun Jan 19, 2014 5:11 am

http://www.mstrials.org.au/multicenter- ... uration-st
HI FRIENDS This new drug SIPONIMOD for SPMS Patients the trial due to recruiting people with SPMS until may 2014. trial in sydney difference hospitals details in the website
regards:
seeva
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Re: Progressive MS Pipeline

Postby 1eye » Sat Jan 25, 2014 11:12 am

This is a pretty recent one (2014?):
Abstract
BACKGROUND:

Human endogenous retrovirus (HERV) genes represent about 8% of the human genome. A member of the HERV family W, the Multiple Sclerosis-Associated Retrovirus (MSRV) gene, encodes an envelope protein (Env), which can activate a proinflammatory and autoimmune cascade through its interaction with Toll-like receptor 4. Due to its proinflammatory property and an inhibitory effect on oligodendrocyte precursor cell differentiation, the MSRV-Env protein could play a crucial role in the pathogeny of multiple sclerosis. GNbAC1 is a humanized monoclonal antibody of the immunoglobulin G4 type, which is directed against MSRV-Env. After validation of the MSRV-Env as a therapeutic target in preclinical experimental models, a clinical development program was initiated.
OBJECTIVE:

This study evaluated the safety profile, pharmacokinetic parameters, and immunogenicity of GNbAC1 in healthy male volunteers.
METHODS:

In this first-in-humans, Phase I, randomized, double-blind, placebo-controlled, dose-escalation study, each subject received a single dose of IV GNbAC1 0.0025, 0.025, 0.15, 0.6, 2, or 6 mg/kg or inactive vehicle (placebo), infused over 1 hour. Tolerability and other laboratory parameters were observed, and regular blood sampling was performed, to study the pharmacokinetic properties and immunogenicity of this monoclonal antibody.
RESULTS:

A total of 33 male subjects (mean age, 44 years) completed the study. GNbAC1 was well tolerated after dosing in all subjects and in each dose cohort. Only minor and nonspecific adverse events (AEs) were recorded; no serious AEs were reported. Pharmacokinetic data show a dose-linear pharmacokinetic profile. The mean elimination half-life ranged between 19 and 26 days, with therapeutically efficient concentrations maintained over a 4-week periods at doses of 2 and 6 mg/kg. No emergence of anti-GNbAC1 antibodies were detected after dosing in any subject over the entire observation period of 64 days.
CONCLUSIONS:

In these healthy male subjects, the safety and pharmacokinetic profiles of GNbAC1 appeared favorable. These findings are expected to allow for the launch of a Phase II development program for this innovative therapeutic approach in patients with multiple sclerosis. ClinicalTrials.gov identifier: NCT01699555.
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Re: Progressive MS Pipeline

Postby vw35qrt » Sat Jul 19, 2014 7:19 pm

They are testing an over-the-counter antihistamine, Tavist, to promote remyelination. It is being tested on relapsing remitting patients, but I can't think of a reason why they don't try it out on people with PPMS. The generic of this product at any drugstore is called Clemastine. I'm thinking about trying a dosage of Tavist out for 3 months taking two 4mg tablets daily. Has anyone tried it and had any improvement in symptoms?

http://clinicaltrials.gov/show/NCT02040298
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Re: Progressive MS Pipeline

Postby CureOrBust » Sun Jul 20, 2014 1:29 am

vw35qrt wrote:They are testing an over-the-counter antihistamine, Tavist, to promote remyelination. It is being tested on relapsing remitting patients, but I can't think of a reason why they don't try it out on people with PPMS.
I am guessing, but they would wish it to show its best results (if any) and RRMS would be mostly Myelin damage, where PPMS has more axon damage, so coating with Myelin wouldn't help. Just a guess.
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Re: Progressive MS Pipeline

Postby Fred1208 » Tue Apr 14, 2015 2:01 pm

Tuesday, April 07, 2015
MedDay provides update on pioneering pivotal Phase III study design in Progressive Multiple Sclerosis


~ Phase III data to be disclosed at AAN Annual Meeting in Clinical Trials Plenary Session on April 24th 2015~

Paris, France, April 7 2015 - MedDay, a biotechnology company focused on the treatment of nervous system disorders, today 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 multiple sclerosis, a major area of unmet medical need. Data from the MS-SPI study will be presented at the Clinical Trials Plenary Session at The American Academy of Neurology (AAN) Annual Meeting, Washington DC, on Friday April 24th at 1200 EST.

MS-SPI is a randomized, double-blind, multicenter, placebo-controlled (2:1) trial of MD1003, 300 mg/day, in patients with progressive MS who have demonstrated progression in the 2 years prior to enrolment.

A total of 154 patients with a baseline EDSS (Expanded Disability Status Scale) score of between 4.5 and 7 were enrolled from 16 MS reference centers across France. Treatment duration was one year.

The primary endpoint for the study was defined as the proportion of patients 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.

The main secondary endpoints evaluate the effect of MD1003 in stabilizing or slowing down the rate of progression. These endpoints include the change in EDSS between M0 and M12, the proportion of patients with progression at M9 confirmed at M12 and the change in TW25.

Frédéric Sedel, MD, Chief Executive Officer of MedDay, commented: “This trial was particularly ambitious. This is the first time that a study in progressive MS has evaluated the proportion of patients improved at M9 and confirmed at M12. This challenging clinical endpoint was designed during discussions with European and US regulators. We look forward to presenting the results of the trial at AAN later this month.”

Another important objective of the trial is to evaluate the safety of long-term treatment with MD1003 300 mg/day. Serious and non-serious adverse events recorded during the trial will also be presented.

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.

Despite these different initial clinical phenotypes, the time to reach certain disability milestones and the ages at which the milestones are reached are similar for patients with PPMS and SPMS. Recent guidelines have therefore proposed to group PPMS and SPMS within a single entity called “progressive disease”. The overall prevalence of patients with progressive disease is estimated to be at least 40% of all MS patients.

Full session details and data presentation listings for the 2015 Annual Meeting can be found through the AAN website: http://www.aan.com/conferences/2015-annual-meeting.

-Ends-



About MD1003

MD1003 is an investigational medicine thought to have both pro-myelinotic 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. It has patent protection in the 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 long chain fatty acids required for myelin synthesis; and (2) it activates the Krebs cycle in demyelinated axons to increase energy production.

MD1003’s proof of concept has been obtained in a pilot open label study involving 23 subjects with primary and secondary progressive MS. Results were positive with up to 90% of subjects exhibiting clinical improvement over time. Treatment efficacy was also assessed using electrophysiology studies and magnetic resonance spectroscopy. Results were published this year in the Journal of Multiple Sclerosis and Related Disorders. For more information on the study, please see: http://www.msard-journal.com/article/S2 ... 1/abstract.

Scientific Advisory Board

Prof. Alan Thompson (Chair, UCL, UK); Prof. Jack Antel (McGill, Canada); Dr Robert Fox (Cleveland, USA); Prof. Reinhard Hohlfeld (Munich, Germany); Prof. Jean Pelletier (Marseille, France); Prof. Per Soelberg Sorensen (Denmark); and Prof. Ayman Tourbah (Reims, France, principal investigator in the study).

About MedDay

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: http://www.medday-pharma.com.

For more information, please contact:

MedDay Pharmaceuticals

Email: contact@medday-pharma.com

Consilium Strategic Communications

Mary-Jane Elliott, Jonathan Birt, Ivar Milligan, Laura Thornton

Tel: +44 (0)20 3709 5700

Email: medday@consilium-comms.com
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Re: Progressive MS Pipeline

Postby Fred1208 » Tue Apr 14, 2015 2:03 pm

GeNeuro and Servier sign partnership to develop first medicine addressing a causal factor of Multiple Sclerosis

GeNeuro responsible for development of GNbAC1 till completion of Phase IIb in Multiple Sclerosis, receiving $47M from Servier, after which Servier can exercise licensing option in all markets excluding the USA and Japan.
GeNeuro retains independence & full control of the U.S. & Japan and for applications of its technology in other diseases.
After exercising option, Servier will cover Phase III global development costs and pay GeNeuro up to US$408M in future development and sales milestones, as well as royalties on future sales.
Servier will also have the option to take an equity stake in GeNeuro as a minority shareholder in 2015.

December 2, 2014, Geneva, Switzerland, and Suresnes, France - GeNeuro SA, a pioneer of new therapies for neurology and autoimmune disorders, announced today that it has entered into a strategic partnership with Servier, the leading independent French pharmaceutical company, to develop and market GNbAC1 in Multiple Sclerosis (MS). As the first drug addressing a causal factor of the disease, GNbAC1 has the potential to radically change the way MS patients are treated.
GNbAC1, a humanized monoclonal antibody, targets MSRV-Env, the envelope protein of MS associated retrovirus, a member of the HERV-W family, the expression of which is usually silent but reactivated and expressed in MS lesions from an early stage in the disease. This protein has been shown to be both pro-inflammatory and an inhibitor of remyelination, the two major drivers of MS pathophysiology.
By targeting MSRV-Env, GeNeuro expects to bring to patients a safe, effective treatment that can halt progression of both relapsing-remitting and progressive forms of the disease without hampering the patient’s immune system. GNbAC1 has successfully completed Phase IIa, demonstrating an optimal safety profile and encouraging signs of efficacy on a first small cohort of patients. “GNbAC1’s original mode of action proposes a true innovation in the field of MS” says Prof Hans-Peter Hartung, chairman of the Department of Neurology of the University Hospital Düsseldorf and chairman of GeNeuro’s Advisory Board.
Under the terms of the agreement, GeNeuro will be responsible for the development of GNbAC1 until completion of Phase IIb, after which Servier can exercise the option to license the product for all markets excluding the USA and Japan. Financial considerations include the payment by Servier to GeNeuro of US$47 million to finance the completion of Phase IIb. Subsequent to exercising the option agreement, Servier will cover the costs of the Phase III global development program and pay GeNeuro up to US$408 million in future development and sales milestones, as well as royalties on future sales. Servier will also have the option to take an equity stake in GeNeuro as a minority shareholder in the next 12 months.
For François Curtin, CEO of GeNeuro stated: “This strategic agreement with Servier is a recognition of the innovative nature and huge potential of GeNeuro’s technology. Combining GeNeuro’s technical expertise with Servier’s scientific, medical and financial resources will create an exciting new alliance to fuel the development of our unique approach, ultimately benefitting MS patients around the world.”
Jesús Martin-Garcia, Chairman of GeNeuro declared: “This agreement is an ideal way to develop GeNeuro’s technology and deliver its full value for patients and stakeholders. With all further development costs in MS funded by our partner, GeNeuro has a clear path forward with a manageable geographic focus on two of the world’s major markets.”
Emmanuel Canet, VP Research & Development at Servier underlined that: “The importance of this agreement demonstrates Servier’s willingness to dedicate its research to serious diseases with major unmet medical needs. This new partnership should allow Servier to provide patients with a new treatment against a particularly disabling disease”.
Christian de Bodinat, Director of the Neuro-psychiatry Therapeutic Innovation Centre mentioned: “MS – and especially its progressive forms – is still today a major source of handicap in the world with no satisfactory therapeutic options. We are also confident that the strong expertise of GeNeuro in MS combined with Servier’s clinical experience in neurology will result in a perfect match for driving GNbAC1 to success.”
Olivier Laureau, President of Servier added: “Not only will this new strategic alliance allow Servier to enrich its portfolio in a disease with a huge unmet medical need, but we are especially proud to count as partner a company that was spun-off from Institut Mérieux, a French institution internationally recognized for its excellence in research.”
About Multiple Sclerosis
Multiple Sclerosis is an autoimmune disease affecting the brain and spinal cord, driven by inflammatory and neuro-degenerative processes. It damages the myelin sheath, the material that surrounds and protects nerve cells, resulting in axonal damage. This slows down or blocks nervous conduction between the brain and the body, which leads to the symptoms of MS. The causes of MS are still unclear.
This disease takes three main forms:
Primary Progressive Multiple Sclerosis (PPMS, about 10% of patients at onset), where symptoms continually worsen from the time of diagnosis.
Relapsing-Remitting Multiple Sclerosis (RRMS, about 90% of patients at onset), characterized by unpredictable attacks of neurological symptoms followed by partial or complete recovery.
Secondary Progressive MS (SPMS), developed after several years of relapsing-remitting MS, in which symptoms worsen without relapses.
Present MS treatments address RRMS forms, targeting the patient’s immune system to lower the frequency of relapses, with not always a clear impact on overall disease progression, and at the cost of an increased risk of opportunistic infections and cancers. The situation is even less satisfactory in progressive forms of the disease (about 40% of patients), with a high medical need and no approved treatment for PPMS forms.
About Servier
Servier is an independent French pharmaceutical research company. Its development is based on the continuous pursuit of innovation in the therapeutic areas of cardiovascular-, metabolic-neurologic-, psychiatric-, bone- and joint diseases as well as cancer.
In 2013, the company recorded a turnover of 4.2 billion euros.
91 percent of Servier drugs are consumed outside France.
27 percent of turnover from Servier drugs were reinvested in Research and Development in 2013.
With a strong international presence in 140 countries, Servier employs more than 21,000 people worldwide.
More information: www.servier.com
About GeNeuro and its technology
GeNeuro was created in 2006 at Eclosion, the Geneva life sciences accelerator, as a spin-off of Institut Mérieux where the technology was originally discovered. It develops first-in-class therapies against diseases associated with the expression of pathogenic proteins of human endogenous retroviral origin (HERV). Its lead product GNbAC1 targets MSRV-Env, a protein expressed in MS lesions from an early stage, which has been shown to be both pro-inflammatory and an inhibitor of remyelination, the two major drivers of MS progression.
The Multiple Sclerosis associated retrovirus (MSRV) is a member of the HERV-W family and was initially isolated in cell cultures from patients affected with Multiple Sclerosis. MSRV is normally latent in the genome of individuals, but it can be re-activated by viral infections and other co-factors to express a pathogenic protein, MSRV-Env. MSRV-Env provides the missing link between the observation that viral infections are associated with the onset of the disease and expression of the pathogenic factor (the MSRV-Env protein), which can then explain the inflammatory and demyelinating characteristics of MS.
By targeting MSRV-Env, GeNeuro expects to bring to patients a safe, long-term treatment that can halt progression of the disease, addressing both the inflammatory and demyelinating mechanisms relevant for all forms of MS. As the first drug addressing a causal factor of the disease, it will radically change the way MS patients are treated.

Further information can be found at: www.geneuro.com.
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