Eltoprazine, LymPro and MANF: The Path Forward to Value Creation
Posted on Monday, October 27th, 2014 at 8:00 am
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The last several weeks have been a particularly exciting time for the team at Amarantus. Months of effort are beginning to bear fruit, as we’ve begun to reach milestones in all three of our key programs. As the company continues to make systematic progress in executing on our business plan, we are encouraged by the reception we are receiving from patients, physicians and shareholders who understand the importance of what we are doing.
I’ve begun to receive many inquiries recently about our most advanced therapeutic program, Eltoprazine, following the announcement of our first interaction with the FDA. Given the imminent Phase 2b Parkinson’s disease clinical trial initiation, I’d like to take this opportunity to put some context to each of our programs so that there is a better understanding of their current status and what is before us in the weeks and months ahead, as many new prospective shareholders have been asking what our strategy is for each program going forward.
Eltoprazine
Eltoprazine is the company’s most classical asset from a pharmaceutical industry perspective. It is an orally administered, small molecule drug candidate, which selectively activates specific serotonin receptors in the brain. We are currently developing it for Parkinson’s disease Levadopa-induced dyskinesia (PD LID) and adult attention deficit hyperactivity disorder (Adult ADHD). Eltoprazine was originally developed by Solvay Pharmaceuticals for the treatment of aggression in multiple psychiatric conditions. Throughout its clinical development history, Eltoprazine has been administered to nearly 700 healthy subjects and patients, hence providing a sound body of safety data to support further development.
Amarantus holds all filed regulatory dossiers in the United States and Europe, and has an exclusive license to the key intellectual property covering Eltoprazine’s use in PD LID and Adult ADHD. Our license covers method of use patents issued for Eltoprazine in North America and Europe giving coverage for Eltoprazine through the middle of 2022, as well as method of use patent applications for PD LID filed in 2012 potentially giving coverage through 2027. Eltoprazine is eligible to receive post-approval marketing exclusivity of 5 years in the United States and 10 years in Europe under the New Chemical Entity (NCE) regulatory approval pathway.
Parkinson’s disease Levodopa-induced Dyskinesia (PD LID)
Initial proof-of-concept data for Eltoprazine in PD LID were obtained in a Phase 2a study supported by the Michael J Fox Foundation (MJFF). These data were presented at a MJFF meeting in 2012 and at the OneMedForum 2014 conference in January of this year and a manuscript of the study has been submitted for publication to a major a peer-reviewed Neurology journal. In the meantime, our therapeutics division clinical development team has been diligently preparing for the initiation of a Phase 2b study in PD LID in the United States and in Europe. We are looking forward to receiving pre-IND feedback from the FDA later in the fourth quarter and being able to open a new IND in the Neurology Division of FDA shortly thereafter. The Phase 2b trial is on schedule to start in early 2015.
There is a big unmet medical need for PD LID drugs. So far, there are no products specifically approved for this indication. There is a strong desire among clinicians, patient advocacy groups and regulators to see drugs approved for the treatment of LID, which would markedly improve the quality of life for patients with PD.
Apart from its potential for treating PD LID, we see substantial opportunities for Eltoprazine in other aspects of PD that would enhance the product lifecycle management, increase its market share and therefore potentially substantially increase its value. Non-motor symptoms that include mood disorders, cognitive deficits and impulse control disorders, are an important part of advanced PD that are poorly addressed by current treatments. The clinical data we already have in more than 350 psychiatric patients support a potential utility of Eltoprazine in the treatment of non-motor symptoms of PD, thereby creating an exciting opportunity to expand the clinical development into additional indications in PD patients.
We have seen recent successes by other companies in the PD space provide tremendous value for shareholders when treating PD symptoms, and we believe Eltoprazine positions Amarantus to follow on a similar path.
Adult Attention Deficit and Hyperactivity Disorder (Adult ADHD)
Recent increased interest in the market’s need for new, non-scheduled ADHD drugs has highlighted the importance of Eltoprazine’s positive clinical data in the area of Adult ADHD, where the market seems to be growing rapidly in comparison to pediatric ADHD as many pediatric ADHD patients mature into adults. We previously outlined our intentions to initiate a Phase 2b trial for Eltoprazine in Adult ADHD following the initiation of the Phase 2b PD LID clinical trial, and we remain on track to achieve this as much of the groundwork for the Adult ADHD trial is being completed for the Phase 2b PD LID trial. Of note, the clinical data obtained in the Phase 2a Adult ADHD suggest that Eltoprazine has clinically significant activity across a broad spectrum of Adult ADHD patients including both the Primarily Inattentive (“PI”) subgroup, as well as the Hyperactive subgroup.
A copy of the summary of the clinical data for Adult ADHD data is available online at: http://www.sec.gov/Archives/edgar/data/142481...9-1.htm.We believe Eltoprazine could eventually be used to treat Pediatric ADHD in addition to Adult ADHD. With its novel mechanism of action in this area, and potential applicability to a variety of patient subtypes, we see an important market potential for Eltoprazine in ADHD. This condition affects over 58 million people worldwide, and the key unmet medical need in this indication is for non-scheduled, non-stimulant drugs, such as Eltoprazine.
We firmly believe Eltoprazine will generate significant value for the company as we continue its further development, especially given its extensive drug profiling while at Solvay. Combined with data emerging from our planned studies, we believe there will be significant interest from the pharmaceutical industry as our trials advance in 2015. The company intends to be active in business development for this asset.
LymPro
LymPro is our most advanced program, given that it is set to be commercialized in the very near future. We are on track to bring LymPro to market for the Research Use Only market in the fourth quarter, primarily targeting pharmaceutical clinical trials, and will be submitting to CLIA shortly thereafter for widespread commercial use. LymPro demonstrated very high specificity and sensitivity for Alzheimer’s disease detection in severe and moderate patients in our recently announced LP-002 bridging study, and we are looking forward to additional data in early-stage Alzheimer’s this quarter from an extension of the LP-002 study to support additional intended-use statements under CLIA for LymPro. The company is currently preparing marketing materials to support the launch to both the pharmaceutical industry and the general practitioner community to ensure they understand the value that LymPro will bring to their diagnostic paradigms. In addition, the company is continuing the development of its reimbursement strategy to support commercial sales, in addition to putting in place its LymPro general marketing strategy. We believe putting all of the pieces in place for a successful launch of LymPro is crucial to its initial launch and establishment as the market leading blood diagnostic for Alzheimer’s disease. We intend to publish the LymPro data in a peer-reviewed journal following study completion.
As the launch of LymPro nears, the company is also looking to maximize the value of the investment we have made in the asset since acquiring it in late 2012. As a holding company, Amarantus is continuing to evaluate methods of returning value to shareholders, including a potential spinoff of the company’s diagnostic division. In addition to LymPro and our NuroPro Parkinson’s diagnostic, we are actively evaluating additional complementary late-stage neurology-focused diagnostic assets that would bolster our diagnostic division and make for a successful ‘spin-out’ while concurrently returning value to our shareholders. We believe we will be able to execute on this strategy in the near-term.
As a holding company, our strategy is to acquire undervalued assets, incubate them through critical de-risking milestones and thereafter seek to deliver value to our shareholders through strategic initiatives. We believe we are well positioned to deliver on that promise in the near-term with our diagnostic division, making for the first data point of success of the overall business model and positioning Amarantus to continue this model as we move forward. We believe wholeheartedly in LymPro, and want to ensure that it receives the necessary management focus and commercialization expertise for it to become the gold standard for Alzheimer’s diagnosis. We expect to have updates on this initiative in the near-term.
MANF
MANF continues to deliver. In the last year, we have strategically shifted the development focus of MANF towards ophthalmology, where we see significant opportunity to generate value in the near-term. MANF recently was shown to be efficacious in two additional Retinitis Pigmentosa models, as well as delivering positive data in a model of ocular ischemia which supports development in a number of indications, including Cerebral Retinal Ventricular Occlusion (CRVO), Central Retinal Arterial Occlusion (CRAO) and Glaucoma. We also have experiments ongoing evaluating MANF’s efficacy in Wolfram’s Syndrome with Washington University in Saint Louis. We recently applied to the FDA for orphan drug designation for MANF in Retinitis Pigmentosa and expect additional orphan filings in the near future in the area of ophthalmology. We expect to initiate GMP manufacturing for MANF this quarter to support multiple IND filings for MANF in various indications.
For those of you who are new to the MANF story, MANF has demonstrated efficacy in various animal models of human disease, including:
Retinitis Pigmentosa
Ocular Ischemia (CRVO, CRAO and Glaucoma)
Parkinson’s disease
Diabetes
Cardiovascular Ischemia (Myocardial Infarction)
Cerebral Ischemia (Stroke, Epilepsy)
A select list of peer-reviewed publications for MANF is available on our website at: http://ir.amarantus.com/scientific-publications
This is an impressive list of potential indications. We are currently focusing the majority of our efforts in the ophthalmological areas, however we see significant value in strategically advancing proof of concept animal work in these and other areas as MANF approaches first-in-man studies in orphan ocular indications. We continue to believe that MANF could be one of the big successes in biotechnology due to its unique mechanism and will diligently shepherd the MANF Program through de-risking milestones on its path to treating patients. We are very, very excited about MANF and what lies ahead.
In Closing
We are continuing to evaluate new opportunities for the company that we believe will add significant value to our pipeline outside of our current areas of focus. As we expect to soon derive value from our diagnostics division, we believe it will be important to replenish our pipeline with strategically positioned programs that have the potential to deliver significant return on investment for the company, as LymPro has. We believe Eltoprazine is also well positioned to replicate that model, and believe the value created by both of these assets will further support MANF’s development. As a biotechnology holding company, we have the luxury of being strategically diversified in highly attractive areas of growth across our pipeline. With this strategy we are insulated from enterprise risk from any one program, thereby giving us leverage as we look for additional product opportunities and allowing us to return value to our long-term shareholders either through equity gains or dividends.
Alzheimer’s disease, Parkinson’s disease and blindness are devastating disorders that can leave families, caregivers and patients reeling from damage left in their path. Being able to impact each of these conditions delivers a sense of great pride to our employees that we hope you share as shareholders.
I thank you for taking the time to read this blog, and look forward to communicating with you regarding our progress on many of the points discussed above later this quarter.
Warmest regards,
Gerald E. Commissiong,
President & CEO
Posted on Monday, October 27th, 2014 at 8:00 am
0 0 0 Google +0
The last several weeks have been a particularly exciting time for the team at Amarantus. Months of effort are beginning to bear fruit, as we’ve begun to reach milestones in all three of our key programs. As the company continues to make systematic progress in executing on our business plan, we are encouraged by the reception we are receiving from patients, physicians and shareholders who understand the importance of what we are doing.
I’ve begun to receive many inquiries recently about our most advanced therapeutic program, Eltoprazine, following the announcement of our first interaction with the FDA. Given the imminent Phase 2b Parkinson’s disease clinical trial initiation, I’d like to take this opportunity to put some context to each of our programs so that there is a better understanding of their current status and what is before us in the weeks and months ahead, as many new prospective shareholders have been asking what our strategy is for each program going forward.
Eltoprazine
Eltoprazine is the company’s most classical asset from a pharmaceutical industry perspective. It is an orally administered, small molecule drug candidate, which selectively activates specific serotonin receptors in the brain. We are currently developing it for Parkinson’s disease Levadopa-induced dyskinesia (PD LID) and adult attention deficit hyperactivity disorder (Adult ADHD). Eltoprazine was originally developed by Solvay Pharmaceuticals for the treatment of aggression in multiple psychiatric conditions. Throughout its clinical development history, Eltoprazine has been administered to nearly 700 healthy subjects and patients, hence providing a sound body of safety data to support further development.
Amarantus holds all filed regulatory dossiers in the United States and Europe, and has an exclusive license to the key intellectual property covering Eltoprazine’s use in PD LID and Adult ADHD. Our license covers method of use patents issued for Eltoprazine in North America and Europe giving coverage for Eltoprazine through the middle of 2022, as well as method of use patent applications for PD LID filed in 2012 potentially giving coverage through 2027. Eltoprazine is eligible to receive post-approval marketing exclusivity of 5 years in the United States and 10 years in Europe under the New Chemical Entity (NCE) regulatory approval pathway.
Parkinson’s disease Levodopa-induced Dyskinesia (PD LID)
Initial proof-of-concept data for Eltoprazine in PD LID were obtained in a Phase 2a study supported by the Michael J Fox Foundation (MJFF). These data were presented at a MJFF meeting in 2012 and at the OneMedForum 2014 conference in January of this year and a manuscript of the study has been submitted for publication to a major a peer-reviewed Neurology journal. In the meantime, our therapeutics division clinical development team has been diligently preparing for the initiation of a Phase 2b study in PD LID in the United States and in Europe. We are looking forward to receiving pre-IND feedback from the FDA later in the fourth quarter and being able to open a new IND in the Neurology Division of FDA shortly thereafter. The Phase 2b trial is on schedule to start in early 2015.
There is a big unmet medical need for PD LID drugs. So far, there are no products specifically approved for this indication. There is a strong desire among clinicians, patient advocacy groups and regulators to see drugs approved for the treatment of LID, which would markedly improve the quality of life for patients with PD.
Apart from its potential for treating PD LID, we see substantial opportunities for Eltoprazine in other aspects of PD that would enhance the product lifecycle management, increase its market share and therefore potentially substantially increase its value. Non-motor symptoms that include mood disorders, cognitive deficits and impulse control disorders, are an important part of advanced PD that are poorly addressed by current treatments. The clinical data we already have in more than 350 psychiatric patients support a potential utility of Eltoprazine in the treatment of non-motor symptoms of PD, thereby creating an exciting opportunity to expand the clinical development into additional indications in PD patients.
We have seen recent successes by other companies in the PD space provide tremendous value for shareholders when treating PD symptoms, and we believe Eltoprazine positions Amarantus to follow on a similar path.
Adult Attention Deficit and Hyperactivity Disorder (Adult ADHD)
Recent increased interest in the market’s need for new, non-scheduled ADHD drugs has highlighted the importance of Eltoprazine’s positive clinical data in the area of Adult ADHD, where the market seems to be growing rapidly in comparison to pediatric ADHD as many pediatric ADHD patients mature into adults. We previously outlined our intentions to initiate a Phase 2b trial for Eltoprazine in Adult ADHD following the initiation of the Phase 2b PD LID clinical trial, and we remain on track to achieve this as much of the groundwork for the Adult ADHD trial is being completed for the Phase 2b PD LID trial. Of note, the clinical data obtained in the Phase 2a Adult ADHD suggest that Eltoprazine has clinically significant activity across a broad spectrum of Adult ADHD patients including both the Primarily Inattentive (“PI”) subgroup, as well as the Hyperactive subgroup.
A copy of the summary of the clinical data for Adult ADHD data is available online at: http://www.sec.gov/Archives/edgar/data/142481...9-1.htm.We believe Eltoprazine could eventually be used to treat Pediatric ADHD in addition to Adult ADHD. With its novel mechanism of action in this area, and potential applicability to a variety of patient subtypes, we see an important market potential for Eltoprazine in ADHD. This condition affects over 58 million people worldwide, and the key unmet medical need in this indication is for non-scheduled, non-stimulant drugs, such as Eltoprazine.
We firmly believe Eltoprazine will generate significant value for the company as we continue its further development, especially given its extensive drug profiling while at Solvay. Combined with data emerging from our planned studies, we believe there will be significant interest from the pharmaceutical industry as our trials advance in 2015. The company intends to be active in business development for this asset.
LymPro
LymPro is our most advanced program, given that it is set to be commercialized in the very near future. We are on track to bring LymPro to market for the Research Use Only market in the fourth quarter, primarily targeting pharmaceutical clinical trials, and will be submitting to CLIA shortly thereafter for widespread commercial use. LymPro demonstrated very high specificity and sensitivity for Alzheimer’s disease detection in severe and moderate patients in our recently announced LP-002 bridging study, and we are looking forward to additional data in early-stage Alzheimer’s this quarter from an extension of the LP-002 study to support additional intended-use statements under CLIA for LymPro. The company is currently preparing marketing materials to support the launch to both the pharmaceutical industry and the general practitioner community to ensure they understand the value that LymPro will bring to their diagnostic paradigms. In addition, the company is continuing the development of its reimbursement strategy to support commercial sales, in addition to putting in place its LymPro general marketing strategy. We believe putting all of the pieces in place for a successful launch of LymPro is crucial to its initial launch and establishment as the market leading blood diagnostic for Alzheimer’s disease. We intend to publish the LymPro data in a peer-reviewed journal following study completion.
As the launch of LymPro nears, the company is also looking to maximize the value of the investment we have made in the asset since acquiring it in late 2012. As a holding company, Amarantus is continuing to evaluate methods of returning value to shareholders, including a potential spinoff of the company’s diagnostic division. In addition to LymPro and our NuroPro Parkinson’s diagnostic, we are actively evaluating additional complementary late-stage neurology-focused diagnostic assets that would bolster our diagnostic division and make for a successful ‘spin-out’ while concurrently returning value to our shareholders. We believe we will be able to execute on this strategy in the near-term.
As a holding company, our strategy is to acquire undervalued assets, incubate them through critical de-risking milestones and thereafter seek to deliver value to our shareholders through strategic initiatives. We believe we are well positioned to deliver on that promise in the near-term with our diagnostic division, making for the first data point of success of the overall business model and positioning Amarantus to continue this model as we move forward. We believe wholeheartedly in LymPro, and want to ensure that it receives the necessary management focus and commercialization expertise for it to become the gold standard for Alzheimer’s diagnosis. We expect to have updates on this initiative in the near-term.
MANF
MANF continues to deliver. In the last year, we have strategically shifted the development focus of MANF towards ophthalmology, where we see significant opportunity to generate value in the near-term. MANF recently was shown to be efficacious in two additional Retinitis Pigmentosa models, as well as delivering positive data in a model of ocular ischemia which supports development in a number of indications, including Cerebral Retinal Ventricular Occlusion (CRVO), Central Retinal Arterial Occlusion (CRAO) and Glaucoma. We also have experiments ongoing evaluating MANF’s efficacy in Wolfram’s Syndrome with Washington University in Saint Louis. We recently applied to the FDA for orphan drug designation for MANF in Retinitis Pigmentosa and expect additional orphan filings in the near future in the area of ophthalmology. We expect to initiate GMP manufacturing for MANF this quarter to support multiple IND filings for MANF in various indications.
For those of you who are new to the MANF story, MANF has demonstrated efficacy in various animal models of human disease, including:
Retinitis Pigmentosa
Ocular Ischemia (CRVO, CRAO and Glaucoma)
Parkinson’s disease
Diabetes
Cardiovascular Ischemia (Myocardial Infarction)
Cerebral Ischemia (Stroke, Epilepsy)
A select list of peer-reviewed publications for MANF is available on our website at: http://ir.amarantus.com/scientific-publications
This is an impressive list of potential indications. We are currently focusing the majority of our efforts in the ophthalmological areas, however we see significant value in strategically advancing proof of concept animal work in these and other areas as MANF approaches first-in-man studies in orphan ocular indications. We continue to believe that MANF could be one of the big successes in biotechnology due to its unique mechanism and will diligently shepherd the MANF Program through de-risking milestones on its path to treating patients. We are very, very excited about MANF and what lies ahead.
In Closing
We are continuing to evaluate new opportunities for the company that we believe will add significant value to our pipeline outside of our current areas of focus. As we expect to soon derive value from our diagnostics division, we believe it will be important to replenish our pipeline with strategically positioned programs that have the potential to deliver significant return on investment for the company, as LymPro has. We believe Eltoprazine is also well positioned to replicate that model, and believe the value created by both of these assets will further support MANF’s development. As a biotechnology holding company, we have the luxury of being strategically diversified in highly attractive areas of growth across our pipeline. With this strategy we are insulated from enterprise risk from any one program, thereby giving us leverage as we look for additional product opportunities and allowing us to return value to our long-term shareholders either through equity gains or dividends.
Alzheimer’s disease, Parkinson’s disease and blindness are devastating disorders that can leave families, caregivers and patients reeling from damage left in their path. Being able to impact each of these conditions delivers a sense of great pride to our employees that we hope you share as shareholders.
I thank you for taking the time to read this blog, and look forward to communicating with you regarding our progress on many of the points discussed above later this quarter.
Warmest regards,
Gerald E. Commissiong,
President & CEO
