BYM338-Novartis Breakthrough Therapy for IBM

Is the effectiveness of the drug related to the advancement of one’s IBM?

BYM338will there be any compassionate use available to those patients who can’t get in to the trial (study), and whose disease is progressing rapidly?

Hi Dr Greenburg,Can you please answer the questions below:Please clarify what stage the drug is at- i believe stage 3 – can you please confirm?What requirements are there to enter into a clinical trial and will Novartis approach specialist consultants for suitable patients? We are currently waiting to hear whether a trial will run in the UK ( we are based in Ireland.) Is there any update on final list of trial locations?Do we know any more on the stage two results and the potential health risks involved in taking BYM338?Any more info on the success/ failure rates of the drug and benefits/ adverse effects patients have experienced?many thanks

As of Thursday, December 12 the clinical trials.gov website shows this study having 12 participating investigative sites in the United States. It appears that only two are currently recruiting while the other 10 are listed as “not yet recruiting”. As I understand it, the active treatment part of the study will run for 52 weeks with an additional year of follow-up on each study patient. The start date is listed as September 2013 and estimated completion date November 2015. It seems as though the all-site completion date will be well beyond that. Could you comment please.

If the BYM338 trial shows positive results, how long will it be until it is available for patient treatment? How is the drug administered?

What are the anticipated side-effects of this medication?

I have had 2 muscle biopsies looking to clinically confirm IBM. Unfortunately neither of those confirmed the diagnosis. My doctor has diagnosed me with “suspicion of IBM”. Am I a candidate for BYM338 trial?

Assuming the best scenario, with no unexpected delays, and also assuming approval by the FDA, what is the earliest likely time that BYM338 will be generally available?

Is there any likelihood that BYM338 will be useful in treating the effects of severe polymyositis and dermatomyositis, cases in which the disease was very active for many years and has resulted in long-term, possibly permanent, muscle damage?

Why was testing never done on using anabolic steroids on IBM patients the way the anabolic steriods are used on patients with AIDS and heart disease? Sure there are serious risks to them, but the risks are less than with drugs like the cyclophosphamide used to treat DM/PM — as with the heavy duty drugs used to treat DM/PM, why did physicians not consider that the risk was justified by the benefit? Also these are older drugs with track records and the long term risks are relatively well known compared with brand new drugs.

Presuming that BYM 338 becomes approved for IBM treatment, will it become available to all doctors, or will travel to a limited number specialists throughout the country be necessary?

Do clinical trials, specifically BYM 338, accept patients from Canada?

Can a family doctor submit documents for a clinical trial if IBM doctor is not interested?

Patients with IBM have weakness in different muscle groups, some in legs and arms, some in hands or feet. Does the BYM338 target specific muscle groups or does it impact all muscles?

Will there be a trial site at Stanford Medical Center in Palo Alto, California? If so who is the contact?

If the trial is successful, is there any chance the drug will be available before 2017?

I have had ibm for 20yr. I am Reduced to a wheelchair. would bym 338be a approrate medication for me.

Do you have any specific examples to illustrate the extent of increased muscle strength in Phase II patients; for example, amount of increased distance walked in a specific time; number difference in QMT.

Does receiving IVIG infusions eliminate a patient from participating in upcoming Phase III studies?

In the Novartis press release, it stated results of the Phase II study were to be presented at the American Neurological Assn. meeting on Oct. 14 and was expected to be published in a major medical journal late this year. Did this occur? Could you update us on the content of the study results, or is there a website you could refer us to?

At the 2012 TMA conference in Orlando, you stated there was a possibility of a blood test becoming available that would aid in diagnosing IBM. Did this occur?

How does BYM338 work? Does it improve strength of existing muscles only? Does it grow new muscles? Does it prevent future muscle cell/strength loss?

If I am chosen for the clinical trial and receive the placebo, will they switch everyone over to BYM338 if they are observing positive results?

Hi Dr Greenberg, It was nice to meet you in Kentucky as you were leaving your last session at the conference. I am wondering if Nucleic Acid Metabolism maybe some sort of link to IBM and if that has anything to do with BYM338. I am looking to understand the theory of what BYM338 does for our awful disease.

Dr Greenburg Will this drug also be effective for other IBM issues such a swallowing and arm and hand weekness?

How can we sign up for the therapy?

Dr. Greenberg,Thank you for undertaking research into this rare disease, and taking the time today to provide us information. I have two questions:1. Dr. would you speculate to the cause of the cramping reported in this study, could it be an insufficient blood supply? 2. Additionally, if this therapy is approved could you speculate on the efficacy for someone who has had IBM for 20 years and has been in a wheelchair for 10.

Is there a probability that individual whose IBM is more advanced will be excluded from the trials?

Can a patient expect to get better with this medicine or does it just make the progress of the disease slower?

Dr. Greenberg. Will additional trial sites be established beyond those already identified?

For several months, the clinicaltrials.gov website has listed only 2 sites enrolling into the trial. Do you know when the other sites will begin enrolling?

Are there any very early indications from stage 3 trials that have been ongoing for few months?

Will BYM338, if it becomes available, be available at different times in different countries? Trial is international and perhaps different countries have different legislation and procedures.

Will it be necessary to be at the clinical study more than once a month for the injection?

What is the name of the new blood test being used to assist with IBM diagnosis?

I am a wheelchair-bound IBM patient. If the drug is successful, an increase in my hand and arm strength would greatly increase my quality of life. Might the test protocol be changed so people in my situation can be included in the clinical trial? I don’t expect to ever be able to walk again.

Dr. Greenberg. Thank you for your answers. I wanted still to clarify my question – I was meaning that if BYM388 becomes available in drugstores, will that happen different time in different countries? FDA does not regulate Denmark, for example – as far as I know.

If an individual is enrolled in the study in Phoenix or Houston can they transfer to another site if they move?

Does this new med interfere with heart issues such as hypertension or cause any arrythmias?

How reliable is the blood test in accurately diagnosing IBM?

Can you explain the mechanism of action for this new drug and a how it works on damaged muscles?

I would guess that most of us here saw your presentation on BM338 at the TMA conference in October. Is there anything new that you can tell us, perhaps something that was not included in your presentation?

Do all of the sights need to be enrolled before the trial in Boston can begin

Can you speak to what positive findings were seen in earlier parts of the study that prompted the FDA to move further investigation to a “breakthrough” status? Specifically, what clinical changes were seen that justified the acceleration of these studies?

Dr. Greenberg, the clinical trials website has changed at http://www.clinicaltrials.gov/ct2/show/NCT01925209?term=IBM&rank=11There is no longer list of recruiting sites. How can we understand now, which site is enrolling and we should immediately contact them? Is there some other website we should check? Thank you for answers.

A couple of sites have been listed as “recruiting”. Have either of those sites actually administered any BYM338 to any patients yet?

Has it been studied if the nerve activation signal improves with this drug through emgs or just muscle size. If i am remembering correctly you need a good nerve impulses traveling along mylin sheaths to conduct nerve impulses to make the muscles work, so improving amount of muscle might not help the signal.

Is it possible that the Boston site may still be enrolling participants in April?

Is there a risk the drug trial could see positive outcomes for people with IBM but be denied authorization to be produced for fear of improper use by athletes to “bulk” up?

Thank you Dr. Greenberg for taking the time to answer our questions. Approximately how often will it be necessary to go to the trial site? If one traveled out of state, would there be any compensation to assist with travel expenses?

What is the chemical or biological name for BYM338?