StemCells, Inc. CEO Discusses Interim 6-Month Data From Spinal Cord Injury Trial - Call Transcript

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StemCells, Inc. (STEM) Interim 6-Month Data from Spinal Cord Injury Trial Call September 4, 2012 11:30 AM ET


Martin M. McGlynn – President, Chief Executive Officer & Director

Armin Curt, M.D. – Professor & Chairman of the Spinal Cord Injury Center University of Zurich

Stephen Huhn, M.D., FACS, FAAP – Vice President & Head of CNS Program


Stephen M. Dunn – LifeTech Capital

[Jason Colbert – Max M Group]


Welcome to the StemCells, Inc. webcast on interim six month data from spinal cord injury trial. At this time all participants are in a listen only mode. We will be facilitating a question and answer session towards the end of today’s conference. (Operator Instructions) As a reminder, the conference is being recorded for replay purposes. I would now like to turn the presentation over to the host for today’s conference Mr. Martin McGlynn, Chief Executive Officer.

Martin M. McGlynn

Welcome to StemCells, Inc.’s webcast during which we will present a summary of the Phase I/II data in our chronic spinal cord injury trial which is being conducted in Balgrist Switzerland. Before we go any further I would like to introduce you to Dr. Armin Curt who is on the line on the call who is professor and chairman of the Spinal Cord Injury Center at the University of Zurich and is the medial director of the Paraplegic Center at Balgrist University Hospital and Dr. Curt is the principle investigator on this study.

Dr. Curt will make the majority of the remarks and conduct most of the presentation. He will be preceded by Dr. Stephen Huhn who is a neurosurgeon and heads our CNS Program and is the vice president of StemCells, Inc. Stephen will start off with a few introductory slides and then when Dr. Curt is finished with his presentation Stephen will wrap up on the presentation and then thereafter we will open it up to Q&A. Without any further adieu I’d like to hand you over to Dr. Stephen Huhn.

Stephen Huhn, M.D., FACS, FAAP

This morning we have about 20 or 21 Slides to go through and as Martin said I’ll have some brief remarks in the first few slides and then I’m going to have Dr. Curt go through the interim data in more detail and then we’ll finish up with a Q&A at the end. The Phase I/II study design, just to remind everyone is an open label clinical investigation dealing with a single dose and a fixed dose of the human central nervous system stem cell which is delivered directly into the spinal cord at a dose of 20 million cells.

Patients are immuno suppressed because this is an alginate transplant for a period of nine months post operatively. The trial is designed to enroll 12 subjects who have a thoracic level injury from the second thoracic segment down to the 11th segment. Patients are eligible to enroll in this trial who are between three and 12 months after injury and this corresponds to what we believe is generally considered the sub acute or late sub acute to early chronic phase of spinal cord injury.

In addition to the timeframe after injury patients who both have a complete and incomplete type of injury are eligible for the trial and will be enrolled in a sequential fashion. The first cohort of the trial, the subject of our discussion today, are those patients who have complete injury and by that I mean patients who have lost sensory function and motor function below the level of spinal cord trauma. This of course corresponds with what we call the AIS cohort A.

The trial will then continue after we dose this first cohort into patients with incomplete injury AISA B and ASIA C patients. With each cohort we have a data monitoring committee that will review the status of not only the recent safety data but the equivalent safety data from the prior dosed patients as we continue in the trial. The trial is first and foremost designed to examine safety, tolerability, and feasibility of our interventions but we’re also obviously expanding a lot of effort to determine whether we have any signals of an effectiveness.

In that regard we examine the patients clinically for a number of sensory modalities in the thoracic area which include light touch, pinprick pain, and temperature sensation. We’re also doing tests to examine electrical impulse transmission in the spinal cord across the level of injury and this refers to what is known as somatosensory evoked potentials or SSEPs as well as electrical heat potentials, different sensory stimuli if you will that are then recorded to determine whether any electrical current across the level of injury. And then of course, MRI imaging as well.

The summary today that we’ll be providing you with in more detail that Dr. Curt will go through has given us so far a very favorable safety profile. At this point with the six month interim data we have no adverse events that are related to the cells and there’s no evidence of any change in the pain threshold or sensation for the patients in terms of adverse concerns. Also, the surgery immunosuppression have been well tolerated in all three patients.

As we have examined these patients over time at both the three and six month intervals, Dr. Curt and his team have observed considerable gains in sensory function in two of the three subjects. The third subject remains stable. What we have seen is changes in sensory modalities over more than one time of sensation and we see these in multiple segments below the level of injury. We’ve also been able to look at these sensory changes in a quantitative way as well as looking at electrophysiology and this is again, details that Dr. Curt will go into. But both the quantitative and electrophysiology are helping confirm the clinical examination changes that we are seeing for sensations in these two patients. So in general what we have seen no win this interim analysis is progressive and consistent changes in sensory function over more than one level in both of these patients and give what we understand to be the history of spinal cord injury recovery we think that these changes are unanticipated and we’ll show you some data from the European network that speaks to this in detail.

I’d like now to turn the presentation over to Dr. Curt who will take you through this data and the background nature of the trials and tests that we’re doing.

Armin Curt, M.D.

I’m going to explain to you a little bit the clinical field because trails in patients with spinal cord injury and complete paraplegia is pretty much difficult and challenging and you need to manage your expectations. Coming to this point it is very much important to have the right expectation what to achieve in complete patients and eventually when the study progresses what to achieve in incomplete patients.

Therefore, in the patients with the complete spinal cord injury we addressed our focus necessarily on two aspects of safety at the segmental level so is there a harmful change but also with the same token looking for efficacy. So is there a true change in each segment close to the level of lesion and as you can see in the schematic patient here at the trunk there are many segments to address.

The assessments we do are clinical assessments which are routine clinical assessments on the left hand side such as pinprick and light touch sensation and in addition to the [semi] quantitative measurements and electrophysiology to have some more objective data because one of the concerns is that patients might give a different interpretation of changes and we want to have cooperating findings to actually ensure the changes a patient are reporting can be considered to be true changes. So this is a challenge and very much important and the given cohort we’ll very closely look to the segmental changes close to the level of lesion and in patients eventually with an incomplete injury also longitudinal projections will be addressed.

To understand what is a complete spinal cord injury in the trunk means it is changes in the segments and paraplegic patients are very aware. You see here a graph that shows what is the extent of changes of level of lesions so this is a set of almost 160 patients followed at least three to four times over a year period in the European network. What you see is actually the majority of patients don’t change the level of lesion. So looking to the level of lesion is a main to see as a relevant change.

The main change in his population of patient resembled our cohort with the level of lesion and completeness is actually very minor. So you see the mean changes point too. This is actually true for both qualities pinprick sensation, kind of pain sensation and also light touch sensation. In our cohort, just to give a reminder, we dealt with three patients, all patients having had a traumatic severe spinal cord injury with a mid thoracic complete spinal cord injury so no movement and no sensation below the level of lesion and you can see that the patient has been dosed, the surgery has been done between four and nine months after injury.

This is also an important note. We observe the patient, we do several baseline measurements until the patient plateaus and intervention is done in a condition where we have confirmed the patient is actually not any more changing. So the individual patient can be taken at the time of the baseline control and this has been actually helpful understanding changes that might be introduced due to the intervention and actually to distinguish this from any kind of spontaneous recovery.

In the next slide I just want to show you that the intervention we do is a serious intervention so you do see an MRI scan of the patient with a severe spinal cord injury. The slide in the middle shows you an ultrasound during surgery and the black big hole you can see, this is actually the lesion area and this area you have to consider that the neuro tissue has actually been completely damaged and completely deteriorated. The cell implantation is actually going above and below the level of lesion so it’s not going into this traumatic [cist] but actually it kind of visits spinal cord tissue above and below the level of lesion. This is a very important concept.

On the right slide you’ll actually see how that looks like during surgery. You open the spinal canal, you open the dura, you look at the cord, you take an ultrasound to identify where the [cist] is and then with ultrasound guided the cells can be implanted exactly above and below those lesions. This is something very important as a procedure.

This procedure per say would do some harm to the patient because you inject something and this is one of the primary questions regarding safety and so if you can understand we did a lot of effort to look to the safety aspect and the segment close where this injection has been done. The result of the first patient is shown here and on the left hand you’ll see the condition of the patient before surgery, and this is just one picture of several measurements before sugary, the condition on the left side has been repeated several times and what you do see as you examine the light touch sensation of the patients the green area is where the patients report there is normal sensation. The yellow one indicates where the patient has some changes so he is still sensing if you stroke the skin but not to a normal extent and below where it becomes red actually in this area the patient has no sensation, not any sensation for stroking the skin.

On the right hand side you do see how that looks like on the patient at six months and actually this picture again summarizes five or six repeated measurements confirming that the patient has no changes in these segments. The examination showcased here with the light touch sensation was also true for the other modalities and also for the electrophysiology. So from this perspective it’s a very safe finding but no change. This is very much different than what we observed in the second patient.

In the second patient again, the same procedure, left side procedure pre surgical measurements and on the right side you do see a change and this change occurred with a delay after surgery. The delay was about three months, so not an immediate change, a change that evolved over time. What you see is that he has more kind of yellow segments close to the level of lesion. Interestingly, the patient reported that actually the sensation was more brisk, the sensation was more intense although at no time kind of painful or [inaudible] but the sensation was more clear. So to understand what that could mean we applied also the other testing and the first one here you can see is the electrical perception threshold measurements.

So that sounds difficult but actually it’s not that difficult. What you do is you apply a very controlled electrical stimulation to these skin areas where you see the yellow mark and on the left hand side again you’ll see an area that is quite normal at [inaudible] level so high level and at the belly area you see this reddish line that the patient has no sensation to the electrical stimulation or just a very minor one.

Then over time we do see that this sensation to stimulation reappeared so the color is becoming greenish or yellow and this reappearance was actually in accordance to the increased sensation to light touch. It’s important in a sense that those kinds of modalities electrical stimulation and ultra light touch are considered to be conveyed by very similar pathways within the spinal cord so this is kind of a confirmation of the clinical find of light touch.

Going to the next modality in the segment which does mean pain sensation, the concept here is actually to do a heat perception. Heat perception is one of the modalities associated with pain and you can control this very nicely so you put a [thermode] on very defined skin areas, you measure when the patient is actually able to first sense heat stimulation and then you can also measure when this heat sensation becomes painful.

The advantage is that this technique in contrast to pinprick sensation is that the patient is not actually knowing when stimulation is coming and what you are doing with the stimulation and this gives you a very reliable kind of thresholds. Again, you do see here that the [inaudible] area to 10 level the sensation was very much impaired at the baseline measurements and after six months actually these areas actually also became normal. So again in this area where the patient reported to us such an increased sensation actually the heat perception became normal.

We wondered of course how reliable our measurements over time are. Obviously, we have many baseline measurements and control studies but still in these patients looking to the changes over time having such an intervention you will ask yourself, “Okay, how reliable are these measurements? How useful are these measurements?” And for this question we illustrate two dermatomes at level T7, so it’s bluish kind of start and the level T10 with the reddish.

But in the level T7 the patient never reported a change of his sensation so you would think, “Okay, nothing occurred at this level and your measurements should be on one hand normal and should not necessarily change over time.” This is actually illustrated in the upper diagram where you do see the pain perception levels and the pain thresholds which throughout this measurement and you see the testing six measurements, two measurements before surgery, four measurements after surgery actually remained normal and didn’t change over time.

However, in the segment T10 where we saw and the patient reported this reappearance of sensation you do see that until measurement four both the perception of heat, so first time to sense heat at all it was very much increased and even the painful sensation, pain heat, which was completely abolished actually started to reappear at the sixth measurement time. This is again a confirmation, this is a very different technique that what the patient reported as a change in his pinprick sensation actually holds true also with such a kind of a semi quantitative measurement applying a very different kind of stimulation but the finding actually holds true.

Going to the next patient, this is a patient also with a thoracic injury T4 level. You can see the presurgical MRI on the left hand side. You see in the middle the implementation of the broken spine and on the right hand several of the follow up MRIs with interest to see if certain major change [inaudible] in these areas. Actually, this has not been observed in any of the patients. In none of the patients we saw so far any change to the post traumatic cist. We did not see actually an area of the [inaudible] forming or compressing the area.

Looking to the patient to his finding, you will see a very prominent, a very strong change. Again, on the left hand side the presurgical repeated measurements and you do see that at the segment T5 and below the patient does not have any sensation. This graph illustrates the finding both for light touch sensation and pinprick. On the right hand side you’ll see this massive change. So now from the dermatome before down to T11 the patient reports sensation. This sensation again actually occurred with a delay of about three months and the sensation here are demonstrated at the six month follow up but also at follow ups in between what had been done, these changes were made in this way.

Again, we double checked in a way this change in sensation and that’s what you will see in this next slide. It’s again the same electrical perception threshold measurement and you do see on the left hand in the segment T7 and T10 the patient had no sensation even to high electrical stimulation with more than 50 million. You can classify such a stimulation intensity as unpleasant so the patient did not sense this stimulation. But in the follow up stimulation and then again in accordance to the light touch findings there was a reappearance of this sensation. So he suddenly could sense the electrical stimulation very reliably.

The same with the EPT, we followed this as well up with the evoke potential and that is reported in this slide. So underlying this you will see the same finding with changes in the EPT and for the segment T4, though you can see the improvement from the right side, during the course we do see that the patient is actually for the first time showing evoke potential so this subjectively reported change but can also be confirmed not only in this EPT variable but also we see electrophysiology. That is for the level T4.

The same we did actually and be finding in the level T7. Same condition presurgically, no responses in this segment and after this observation period we got these changes. In the appreciation of this findings there’s always a question of okay what do these changes mean? Are these changes just in the noise of variability or are these changes indicating a trend and a true change? We again plotted here the three such in contrast to the control groups, the historical control groups consisting of 160 patients.

This is I think a meaningful kind of comparison and you do see that in the three subjects, two of the subjects actually had been very much to the side where changes could be observed while subject one actually behaved [inaudible] majority of patients in a comparable cohort of patients.

In summary, we do think that the six month observation with this very elaborated tests close to the segment where the surgery has been done truly does show the safety and tolerability over time. Most important, we could not identify so far any kind of loss of function. However, on the additional positive side, we actually saw changes which we rather did not expect to see and for us it’s very much important in this findings that the changes a patient objectively reports could be actually confirmed or supported by additional measurements like the semi quantitative assessments and also evoke potentials.

Always one of the big debates in clinical science and trial designs with patients with spinal cord injuries is that you come across this placebo effect and these findings here really support that the changes we have been seeing are not related to the [inaudible]. So the sensory changes at this level it is sensory and you need to manage the expectations, finding actually changes in sensory in such patients is a huge finding. But this sensory gains are actually indeed not anticipated at this level and obviously these patients will be followed further on.

We appreciate these changes as a biological effect although this of course has to be confirmed and more investigated in the next core of patients. With this I’ll hand it over to Dr. Huhn.

Stephen Huhn, M.D., FACS, FAAP

That was a great presentation of the date we have to date. We’ll just wrap up a now and reemphasize the points that you’ve heard. We’ve now dosed the first cohort in this trial, those patients with complete injury and you’ve just been taken through the six month time point for all three of those patients and obviously the ongoing follow up will continue. As Armin indicated as the medical monitor I think we have a very favorable safety profile for this intervention and we don’t have any adverse events that we can contribute to the cells at this point.

We are obviously very encouraged by the sensory observations that Dr. Curt and his team are seeing in the patients and from a standpoint as neurosurgeon this could not be more exciting for me. Our DMC, the data monitoring committee continues to recommend that we continue with the trial and in fact, now we’ll be going on to the next cohort of patients which will be those patients with incomplete injury. Obviously, we’re very interested to look at the outcomes in patients who have less severe spinal cord trauma.

The trial is now open to patients not only in Europe but from North America as well and as I said, we’re looking forward to continuing with our trial and proceeding to the next steps. With that I’ll turn it over to Mr. McGlynn and I think we’ll be opened to questions.

Question-and-Answer Session

Martin M. McGlynn

Our first question comes from Stephen M. Dunn – LifeTech Capital.

Stephen M. Dunn – LifeTech Capital

This question is for Dr. Curt and I just want your conjecture here, statistically I don’t see any correlation between patient one, let’s say a non-responder, and patients two and three. There’s no pattern based on age, location of injury, or time of implant. Do you have any kind of a theory on why we saw such unusual improvements in patient two and three and not in one? Was it the severity of the damage?

Armin Curt, M.D.

I hear your question. Actually, with a number of three it might be difficult to try for correlation. We may choose that the patients all have the same kind of density of lesions so clinically the represent a truly completely paralyzed patient the only difference was between the level of the lesion being T4 and being T8 but otherwise the clinical representation was really a very severe complete spinal cord injury. Definitely we have to learn what makes a responder and a non-responder so this is not unusual to have non-responder in an intervention but this needs more numbers to learn about it.

Stephen M. Dunn – LifeTech Capital

Patient number two which had the lowest injury at T9, just subjectively did you see any improvement in breathing ability or trunk stability in that patient?

Armin Curt, M.D.

That indeed improved but that could be also related to his generally being more active. He was going back to work, reemployed and also very active and straightforward patient so that we could not detangle where it addresses to. But definitely the patient is a very active completely independent patient.

Stephen M. Dunn – LifeTech Capital

Last question and I’ll jump back in the queue, it was a little hard on the slides to see but the improvements that we saw in patients two and three did those hold steady or did those improvements decline over time?

Armin Curt, M.D.

They held steady. So they occurred within three months, so they had not been present in the first follow up region in one month after surgery and so they occurred at the three month time window and since then they didn’t change so they have been constant since then.

Stephen M. Dunn – LifeTech Capital

So if there’s going to be improvement we typically see it around the three month timeframe and then that’s as good as it gets? Is that what we can draw from these three patients?

Armin Curt, M.D.

I’m not sure because conceptually the cells can become integrated and they should remain active over a long period of time. So we don’t know to have an understanding of a time window when to see and how long to see changes. This is something we are very much interested and excited to learn because the advantage of this approach in contrast with any kind of drug intervention might be that the cells should remain active and can interact with the spinal cord for a much longer period of time.

Stephen M. Dunn – LifeTech Capital

Finally, on the next cohort of patients which have some degree of feeling are we also going to check for any signs of potential movement or do you think the next cohort we’re not really looking to see any movement?

Armin Curt, M.D.

No definitely we’ll have a very intense checkup for any kind of changes in the mood or behavior, definitely.

Martin M. McGlynn

Your next question comes from [Jason Colbert – Max M Group].

[Jason Colbert – Max M Group]

Are these patients continuing with immunosuppression and how long do you think that immunosuppression will be required?

Stephen Huhn, M.D., FACS, FAAP

Just briefly to answer your question, the protocol has a nine month course of immune suppression and thereafter the immunosuppression is stopped. We feel that a temporary course of immunosuppression is indicated when you’re transplanting alginatic cells but not long term and we’re obviously going to continue to monitor the patients as they reach that milestone in their trial.

[Jason Colbert – Max M Group]

Do you think then that these cells will continue to integrate – what’s the longevity of the cells in the implant?

Stephen Huhn, M.D., FACS, FAAP

If you look at alginatic data in general from the field you can see survival of alginatic cells put in the brain that are 16 years out. Obviously, those aren’t our studies but it’s proof of concept that an alginatic cell will survive in the brain. We consider the spinal cord the same time of immunoprivilege environment. If you look at our data from our Phase I trial in [NCL] we’ll also see evidence of the cells surviving at least two and a half years out and that particular patient who succumbed to complications related to the disease and that was a year and a half after stopping the immunosuppression.

So I believe that the field in general holds that the CNS, the brain and spinal cord are immunoprivilege to the point where you can get long term survival of the cells without the use of immunosuppression.

[Jason Colbert – Max M Group]

For Dr. Curt, one thing I didn’t understand on patient number two, it seemed like they had developed more yellow segments from green segments. Did I read that slide wrong from the November 3rd treatment date to the March 3rd reading?

Armin Curt, M.D.

You read it perfectly and this was one of the very important points for us. If you apply formal criteria of the clinical testing using this ASIA protocol you would indicate this change as an impairment because it’s different from very normal and so then it’s rated as yellow impaired without necessarily indicating what it actually means. In this context the additional information based on the other testing has been utmost important to actually qualify what the changes are about. So it is rather an [altered] segment than an actually impaired segment.

This is also one of the challenges which is important in such kind of cutting edge studies that besides applying the typical standards testing you also need to add additional measurements which give you an additional and a complementary information. In our view we would call it altered and not necessarily impaired but based on the standards it’s the way you have to show it.

[Jason Colbert – Max M Group]

This becomes even more important as we look at the next cohorts ASIA B and ASIA C because we’ll get a better feeling for whether there’s any potential deleterious effect of the cells versus benefit. It should be even more visible in those cohorts, is that correct?

Armin Curt, M.D.

Absolutely and that was the reason I had been showing you this one example with the two segments to show that measurements are constant if nothing happens but that the measurements are sensitive to some changes and definitely in the incomplete patients we even have more concerns, if you wish so, for safety and for this aspect these measurements are even more important. But again with the same interest also for efficacy.

[Jason Colbert – Max M Group]

Two more question and that is at a microscopic level when you’re looking at these patients and they’re completely severed are there still some residual synaptic connections that are maybe demyelinated and is it possible to visualize that and then maybe what may be happening is these cells are then going in and remyelinating those connections? By the way whenever I need really good scientific information I always contact the CFO so Rodney was very helpful in talking about this with me but I’d like to understand your perception of what you think is happening here.

Armin Curt, M.D.

Obviously if this occurred to get the patient under the microscope so the way we could show and get an understanding if there is still some spared tissues is actually based on the MRI to some extent but with a much better resolution with the ultrasound during surgery. This ultrasound does show that there still is some tissue bridges left at the level of the cist formation. So definitely none of the patients have a disrupted cord or completely disconnected cord so the cord was still in continuity, however, with this big kind of lesion cist at the middle of the cord.

Therefore, the assumption based on preclinical studies is that there are still fibers left where a remyelination that could make a difference that is still in a way given but it’s obviously hard to quantify on a high level of resolution.

[Jason Colbert – Max M Group]

Dr. Huhn, when you look at this data and you look out at ASIA B and ASIA C coming can you help me understand what you think might be the clinical strategy going forward in terms of let’s assume that the ASIA B and the ASIA C cohorts, that the data continues to show that its safe and that there’s an improvement in some efficacy, then help me understand kind of what the vision is going forward clinically.

Stephen Huhn, M.D., FACS, FAAP

I think it’s a good question. One of the things that we would be entertaining would be moving into patients with cervical injury. As you well know most patients with spinal cord injury have that level of trauma at their neck in the cervical region not the thoracic. So we would take our data from our thoracic trial and obviously be interested in moving as quickly as possible into patients who have cervical injury because if you think about this in very broad terms, if the data that we’re seeing corresponds to segmental gains that if you take that same level of segmental changes in the cervical cord you’re going to be in a much better position to determine changes in motor function as well as potential enact those changes with the cells.

So one of the things from a big picture would be looking into the cervical patient population. I think we’d also be looking at ways to expand the indications in terms of perhaps time from injury would be another area that we would want to focus on and try to understand more. In other words, could we go to time points beyond one year would be another thing we’d look at carefully.


At this time we have no further questions. I’d like to turn the call over to Mr. Martin McGlynn for closing remarks.

Martin M. McGlynn

Again, I’d like to thank everybody for joining us this morning and in particular I’d like to thank Dr. Armin Curt and congratulate him for his groundbreaking work. This is very exciting and we look forward to continuing to observe these three patients through the next six months timeframe. It will be very interesting to see where they are at the 12 month checkpoint as it were. And of course, the next steps for us will be to start transplanting the next cohort which are the ASIA Bs. We look forward to keeping you appraised of our progress and once again, I’d like to thank everybody for joining us. We look forward to updating you again in the not too distant future.


Ladies and gentlemen thank you for your participation in today’s conference. This concludes the presentation you may now disconnect.

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