- 3 years ago
Longeveron is a clinical-stage biotechnology company developing cellular therapies for aging-related and life-threatening conditions. Its lead investigational product is Lomecel-B™, which is derived from the bone marrow of young healthy adult donors.
The company believes that by using the same cells that promote tissue repair, organ maintenance and immune system function, it can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging.
The company believes that by using the same cells that promote tissue repair, organ maintenance and immune system function, it can develop safe and effective therapies for some of the most difficult diseases and conditions associated with aging.
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NewsTranscript
00:00 Well, how are you doing this Thursday?
00:06 Thanks again for joining us on Benzinga's All Access.
00:08 Oh, thank you for having me, Aaron.
00:11 Of course.
00:12 So let's just hop right into it.
00:14 We've got a lot of people in the audience today, people that I think might be interested
00:19 in this.
00:20 So give us an overview of your company, of Longevron.
00:23 All right.
00:25 Well, Longevron was established in 2014.
00:29 Its focus on cell therapy is specifically producing a product which is under the name
00:35 of Lomacell-B. So remember that name.
00:39 The product is MSC or mesenchymal stem cells.
00:44 And our body naturally produced those cells.
00:47 But after the age of 45, our production of these cells declined.
00:53 And they play a very important role in regenerating our body.
00:59 Got it.
01:00 So tell us a little bit about the science underpinning the Lomacell-B.
01:03 Yes.
01:05 So as I said, Lomacell-B is an MSC.
01:10 And it is believed that this MSC play a very important role in regenerating our body, including
01:17 our brain and a lot of other functions.
01:21 But basically, there are four potential mechanisms for these MSCs.
01:28 One of the most important one of them is the anti-inflammatory effect.
01:32 So that is going to play a big role for many of the diseases that we'll be talking about
01:36 for our company.
01:39 But that basically a very important part.
01:43 Got it.
01:44 So I understand that you just announced top line results from your phase 2a clear mind
01:50 trial for treatment of Alzheimer's.
01:53 Before we get into results, can you tell us about the details, the design and goals of
01:57 the study?
01:58 Sure, Aaron.
02:00 So the way we designed this trial, so this study was a phase 2a.
02:05 So it's a mid-stage.
02:06 We already had one study before, which is called phase 1.
02:10 And this was the second study in the Alzheimer.
02:13 And we designed this trial to compare placebo, which is basically no product whatsoever,
02:22 to three different dosing regimen of our Lomacell B. One single low dose, one multiple low dose,
02:32 and one high dose.
02:33 And when we talk about these doses, we're talking about 25 million, that's the low dose
02:38 cells, and 100 million cells is the high dose.
02:44 Got it.
02:45 So essentially, it's just to try to kind of see the effectiveness of these doses compared
02:49 to the placebo?
02:52 Yes.
02:54 And each one of those group had 12 patients.
02:56 So the placebo had 12, and each one of those group had 12 patients.
03:01 We also evaluated these patients over a period of nine months.
03:05 And we had nine visits that we followed up these patients, including the administration
03:10 of the product in these visits as well.
03:14 Got it.
03:15 OK, so how about the results?
03:16 How did the study go?
03:18 Well, you can imagine, I mean, if you follow Alzheimer, you will see that many of the trials,
03:24 especially the late stage trials that is done in the area of the Alzheimer, have a significant
03:30 larger number of patients.
03:33 So definitely, we approach this study with the goal to look and evaluate the safety,
03:38 which has been a really a big challenge for many of the product, even those who have been
03:42 approved in the market.
03:45 And of course, when you evaluate safety, you want to also evaluate the efficacy of the
03:50 product.
03:51 So we had several secondary endpoints to evaluate the efficacy of the product.
03:57 And we were absolutely pleased, very pleased with the results, actually.
04:02 One, it reaffirmed our safety profile.
04:06 Our safety has been-- so no significant serious adverse events that has been reported with
04:11 the trial.
04:13 And specifically, there was no area which is a specific adverse events common with a
04:21 lot of the drugs that has been approved for Alzheimer.
04:25 So we have not seen one single events of the area.
04:29 And in addition to that, we were very pleased to also see a secondary endpoints on the efficacy.
04:37 So we used several scales to look at this.
04:43 So we looked at the CADS score, which is a composite Alzheimer disease score.
04:48 We looked at activity of daily livings.
04:51 We looked at ADAS-CoG.
04:53 We looked at CDRS-B. We looked at, most importantly, also the left hippocampal volume.
04:59 So we looked at multiple things.
05:02 And yeah, we were very pleased with the results.
05:05 Well, that's good to hear.
05:08 So when will you guys have further results from either that study or other studies?
05:15 All right.
05:16 Well, first, let me just highlight some of the key findings, Aaron.
05:21 And then I will tell you also one additional thing.
05:24 So first--
05:25 Perfect.
05:26 Sounds good to me.
05:27 We demonstrated significant difference on the composite pool data from the three doses
05:32 together in all the CADS score.
05:36 We also have demonstrated significant difference and improvement in the low single dose for
05:43 also Lomacell-B. In addition, in the left hippocampal volume, we have seen a very nice
05:50 dose response and a preservation of the brain.
05:54 Actually, there was some improvement, actually, in the left hippocampal volume, which is something
06:00 very unexpected and in a very positive way.
06:04 We also saw improvement in activity of daily living.
06:07 And that scale is specifically-- it's actually observed and rated by the family and the caregivers
06:13 of these patients.
06:14 So that was very strong movement as well.
06:18 We plan to announce additional cognitive improvement scales like MMSE and MOCA, Montreal Cognitive
06:25 Assessment Scale, in addition to inflammatory biomarkers and other biomarkers later at the
06:34 end of this month.
06:35 And of course, this data is really good.
06:37 So we're going to plan to present it in scientific conferences and definitely have publications
06:42 around it as well.
06:44 Awesome.
06:45 And if the trials continue to be positive, how will your therapy change how Alzheimer's
06:50 is treated?
06:51 Yeah.
06:52 I think that Alzheimer's, despite the fact that there are many companies that are pursuing
06:57 that disease, we have not had our chance of success.
07:00 And some of the success has also been limited by some of the safety profile.
07:05 I think with the safety that we have demonstrated in our trial and the potential benefit that
07:10 this product will bring to these patients, I think it would be a fantastic addition to
07:15 the market.
07:16 And I really believe that many, many patients who, at once, hopefully, we successfully complete
07:21 the phase three and reaffirm our findings, that this product will provide a huge benefit
07:28 to many of the patients who need it for Alzheimer's dementia.
07:31 Yeah.
07:32 I mean, that's great to hear, especially for anyone out there who has anyone in their family
07:38 suffering from Alzheimer's.
07:39 Can you update us briefly on other Lama cell B indications, such as HLHS?
07:46 Sure.
07:47 So the HLHS is a congenital heart disease that affects about 1,000 plus babies born
07:54 every year in the United States, and many similar numbers outside of the US as well.
08:03 The condition is characterized by patients not having a fully developed left ventricle.
08:10 And basically, the current standard of care is re-plumbing the heart to allow the right
08:16 ventricle.
08:17 And we, as a rule, come here by injecting our cells directly in the wall of the heart
08:23 to improve the functionality of that heart.
08:26 This is a very exciting one.
08:28 We have orphan drug designation from the FDA.
08:31 We have a fast-track drug designation from the FDA.
08:34 We have a rare pediatric disease, which actually come with a nice priority voucher as well
08:39 from the FDA.
08:41 So all of those designation came because of the data that we have demonstrated.
08:46 We plan to present some follow-up data from our phase one study at the upcoming American
08:53 Heart Association meeting next month in Philadelphia.
08:57 In addition, we continue to marsh in our phase two program.
09:01 We're doing a head-to-head.
09:03 And we enrolled more than 50% of the patients.
09:05 So hopefully, we'll wrap up our enrollment plan sometime next year.
09:12 And we'll be in the waiting period of one year to measure the final assessment and hopefully
09:17 get this product to these babies who definitely need it.
09:20 Yeah, that's something, again, that's awesome to hear.
09:23 We'll be looking out for any big announcements next month in Philadelphia.
09:27 Well, Wael, Hachad, thank you so much for joining us on Benzinga's All Access today.
09:34 Again, the CEO of Longever on the ticker is up on the screen right there, LGVN.
09:39 Even if, I guess, you're not going to go invest in this stock, we're all rooting for Longever
09:44 on because millions of Americans out there are suffering from Alzheimer's, still without
09:49 really great treatment out there for those.
09:52 And everyone out there knows, I'm sure, how devastating that life is.
09:56 So again, thank you to everything you guys are doing.
09:59 And I look forward to catching up and speaking next time.
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