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Orphazyme Clinical Trial

This page is dedicated to news and information about the Orphazyme's upcoming clinical trial for use in Niemann-Pick Disease Type C (NPC). We will release more information as it becomes available.

Glossary of Terms:

EMA                        European Medicines Agency (European equivalent of FDA)
FDA
                        United States Food and Drug Administration
HDAC                      Histone Deacetylase Inhibitors
ICV                          Intracerebroventricular
IND                          Investigational New Drug
i-IND                        Individual Investiational New Drug
IRB                          Institutional Review Board
NICHD                     National Institute of Child Health and Human Development
NIH                          National Institutes of Health
NPC                        Niemann-Pick Disease Type C Disease
TRND                      Therapeutics for Rare and Neglected Diseases


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Clinical update: Orphazyme announces update to its Niemann-Pick disease type C clinical programme — AIDNPC
Dateline: September 10th, 2015

Orphazyme's clinical program AIDNPC that investigates the orally available small molecule arimoclomal in Niemann-Pick disease type C is initiating in Europe, with recruitment commencing in September.  If proven effective, the program will be moved as quickly as possible to registration to become available for all patients suffering from Niemann-Pick disease type C.

Click here to read the fill Press Release from Orphazyme

Families should follow enrollment criteria as provided on the www.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02435030

The title of the trial is listed as: "A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C."

Patients are encouraged to contact their physicians regarding this information and their physicians will be able to further contact the trial transparency team at Vtesse provided in the www.clinicaltrials.gov clinical trial information page.

[Sept 16th, 2015 ~ blg]

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Orphazyme Announcement:
Orphazyme Clinical Trial Development Update on Arimoclomol in NPC
Dateline: 05/11/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation has been advised by Orphazyme that an “Observational” clinical study for NP-C, tied to their clinical development program of arimoclomol in NPC, has been registered and made available on www.clinicaltrials.gov.  The link to the information pertaining to just this trial is at: https://clinicaltrials.gov/ct2/show/NCT02435030?term=02435030&rank=1

This information was updated as of April 30, 2015. 

At this time, no site in the United States has been announced and currently the study is not open for participant recruitment.  Orphazyme has indicated that they are targeting a clinical trial to start date in the United States toward the end of first quarter 2016.  As we receive further announcements and updates, it will be posted to our NPD family community! 

You can read either the full text view or the tabular view depending upon your preference.  The text allows you to see the inclusion and exclusion information along with the primary outcome measures they will be looking at. 

Sincerely,

Nadine M. Hill
Executive Director
National Niemann-Pick Disease Foundation

[May 11th, 2015 ~ blg]

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Orphazyme Announcement:
Orphazyme Kicks off Clinical Program of Arimoclomol
in Niemann-Pick Disease Type C
Dateline: 04/15/2015

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

Below is the official announcement as provided to the NNPDF, which is being shared globally today by Orphazyme. 


Orphazyme Kicks off Clinical Program of Arimoclomol in Niemann-Pick Disease Type C

Orphazyme has interacted with key opinion leaders and experts from around the world to design a clinical programme that appropriately assesses the therapeutic response of Arimoclomol in Niemann-Pick disease type C.  Arimoclomol is a small-molecule inducer of heat shock proteins. It is an orally available experimental drug that readily crosses the blood-brain barrier.

Arimoclomol’s safety profile has been established through extensive pre-clinical studies and multiple Phase I clinical trials. After the meaningful feedback from EU and US regulators, the Orphan Drug Designations (EMA and FDA), and successful fundraising, Orphazyme has entered into the phase of clinical trial applications.

We are pleased to inform that submissions to various countries have been undertaken, and that the French competent authority (ANSM) has already approved the initiation of the “Observational” study of the clinical programme of Arimoclomol in Niemann-Pick disease type C.  Submission to the relevant authorities is part of this undertaking. Upon the respective approvals being obtained from the corresponding regulatory agencies and ethics committees, enrolment of patients will take place.

The programme is composed of two studies: a) an “Observational” study will start a staggered enrolment in the second quarter of 2015; b) after the 6-month follow-up, all the patients that complete the “Observational” study will be offered to enter into the “Interventional” study, a Phase II/III clinical trial with Arimoclomol. This will be a multi-centre clinical programme involving clinical centres throughout Europe. The clinical development programme also includes a clinical trial in the USA.

Carlos R. Camozzi 
Chief Medical Officer of Orphazyme
E-mail: crc@orphazyme.com


As more information becomes available we will continue to update this page, as well as, our social media sites.

[April 15th, 2015 ~ blg]

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FDA Grants Orphan Drug Designation for Arimoclomol
Dateline: 01/27/2015

Dear NNPDF Families and Friends,

Orphazyme today announced that the Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted an orphan drug designation for Orphazyme’s Arimoclomol as a new treatment for Niemann-Pick disease type C.

The US orphan drug designation complements the corresponding EMA orphan drug designation that was granted for the program in 2014.

The recent successful completion of a €20m Series B financing round, together with the FDA and EMA orphan drug designations, establish a solid foundation for initiating our clinical studies in NP‑C with the purpose of providing arimoclomol as a safe and effective therapeutic option to patients suffering from this devastating disease as well as relief to their families,” says Dr. Carlos R. Camozzi, Chief Medical Officer of Orphazyme.
 
Orphazyme plans to start a clinical trial in NP-C in 2015.
 
See the FDA log of its decision here and the EMA log here.
 
For further information:
Carlos Camozzi, CMO at Orphazyme ApS
E-mail: crc@orphazyme.com

Click here to view the full press release

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions(http://www.nnpdf.org/CyberCautions.html) about medical information provided on the Internet."

[Jan 27th, 2015 ~ blg]

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Dear NNPDF Families and Friends,

RE:  Orphazyme ~ "Orphazyme ApS (www.orphazyme.com) was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins."

The NNPDF is making press release announcements from Orphazyme, pertaining to their advances towards a clinical trial for Niemann-Pick Disease type C, available to the wider NPC Community.

Listed below are the links back to the Orphazyme APS (LLC) website to read the press releases.

Orphazyme Raised 20 Million Euros in a Series B Financing Round
01/09/2015
COMP Issues Prositive Opinion on Orphan Drug Application for Arimoclomol
10/20/2014
Orphazyme in Final Preparation Stages for a Phase II/III Clinical Trial  
10/06/2014

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet."

[Jan 15th, 2015 ~ blg]


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Orphazyme ApS (LLC)*  
Clinical Trial Updates for Niemann-Pick Type C Disease
September 8th, 2014

Update from: Anders M. Hinsby
Orphazyme Chief Executive Officer

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is pleased to share with you the following announcement received from Orphazyme ApS of an upcoming clinical trial for Niemann-Pick Disease Type C.  Orphazyme ApS (Copenhagen, Denmark) develops new therapies for the treatment of rare and genetic diseases.

Below is the official announcement as provided to the NNPDF, which is being shared globally today by Orphazyme.  As more information becomes available we will continue to update this page as well as our social media sites.

Orphazyme moves towards clinical development in Niemann-Pick type C
(Click the above link for the announcement in a PDF)

Orphazyme has identified and evaluated two candidate products through preclinical development for Niemann-Pick type C. One is the recombinant human protein HSP70 and the other is an orally available small molecule, Arimoclomol that works as an inducer of heat shock proteins, including HSP70**.

Both product candidates have produced encouraging biochemical and disease-modifying effects in studies in the Niemann-Pick type C mouse model performed in collaboration with Prof. Frances Platt (University of Oxford). Both compounds have successfully completed toxicity studies and are now enabled for clinical trials. In the third quarter of 2014 Orphazyme will select the product candidate for a clinical Niemann-Pick type C trial. We expect to start the multi-centre clinical trial in patients in the first quarter of 2015. Final inclusion criteria and participating clinical sites are still to be confirmed.

It is a priority for Orphazyme to ensure the clinical and regulatory feasibility of our program in continuous dialogue with patient organizations and national regulatory agencies including EMA and the FDA.

Orphazyme's Chief Scientific Officer and Founder, Dr. Thomas Kirkegaard Jensen, and Chief Medical Officer Dr. Carlos R. Camozzi, MD, will present the preclinical results and supply further information about the planned clinical trial at the Niemann-Pick Disease Group (NPDG) UK meeting, 26-28. September 2014.

We look forward to sharing the exciting data and plans with the patient community.

Kindest regards,
Anders M. Hinsby, Orphazyme Chief Executive Officer

*Orphazyme ApS is founded on the pioneering research from the laboratory of Professor Marja Jäättela, and with the aim of converting human chaperone proteins into effective therapies for degenerative diseases. Orphazyme's novel technology also has the potential to address the neurodegenerative aspects of these devastating diseases.

Orphazyme’s core program is developing heat shock protein based therapies for the treatment of diseases caused by defects in the function and/or metabolism of proteins. The company focuses on severe and often fatal diseases with a very high unmet need.

What is HSP70?

Heat shock proteins (HSP) are a class of functionally related proteins involved in the folding and unfolding of other proteins and are named according to their molecular weight. Proteins with similar structure exist in virtually all living organisms.

Production of high levels of HSPs can be triggered by exposure to different kinds of environmental stress conditions, such as infection, inflammation or exercise. They also occur under non-stressful conditions, acting as “monitors” for the cell’s proteins, by carrying old proteins to the cell’s “recycling bin” (proteasome) and helping newly synthesised proteins to fold properly. These activities are part of a cell’s own repair system, called the “cellular stress response” or the “heat-shock response”.

HSP70 chaperones are required for key cellular processes. A chaperone is a protein that assists folding/unfolding in molecular biology. The HSP70s are an important part of the cell’s machinery for protein folding, and help to protect cells from stress. HSP70 also aids in trans-membrane transport of proteins, by stabilizing them in a partially folded state.

Please refer to their website for more information: http://www.orphazyme.com/

[Sept 8th, 2013 ~ blg]


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Orphazyme Update - 10/31/2013
Orph001 (rhHSP70) Project Progress

Dear Families and Friends,

The NNPDF Central Office received an update pertaining to the current research and clinical trial work as it relates to Orphazyme and their proposed clinical trial titled: Orph001 (rhHSP70).

Dear Reader,

We would like to update you about the progress of the Orph001 clinical development programme.

With the aim of reaching the highest achievable quality in standards and procedures certain amendments have been introduced into the Orph001 development programme to ensure full dose definition, and high alignment to regulatory recommendations and requirements in Europe and US.
Please, let us give you a short review of our development program and the steps ahead.

Orphazyme History and Lead R&D Programme HSP70

Orphazyme ApS was founded in June 2009 with the aim of developing heat shock protein based therapy for the treatment of diseases caused by defects in the function and/or metabolism of proteins. The cornerstone in the company is the research of its scientific founders, Professor Marja Jäättela and Thomas Kirkegaard Jensen, on the cytoprotective properties of human heat shock proteins (HSPs), a class of functionally related proteins involved in the folding and metabolism of other proteins.

Orphazyme leading lysosomal storage disease (LSDs) programme, Orph001, a recombinant version of human HSP70 (rhHSP70), is being developed for the treatment of Niemann-Pick Disease type C.

rhHSP70 (OR0005) has shown to be effective in correcting conditions of lysosomal functional deficiency and concurrent cellular stress. The initial studies were published in Nature by Orphazyme’s scientific founders (Kirkegaard et al. Nature 2010, 463:549-53).

Non-clinical development

Orphazyme has elucidated OR0005 (rhHSP70) therapeutic potential by demonstrating in vitro a remarkable reversion of the cellular pathology in primary cells from NP-C patients as well as other LSDs, including all the major sphingolipidoses. The beneficial effect of OR0005 on primary cells was observed after a single exposure to rhHSP70.

Subsequently, in vivo studies in murine models of NP-C, performed at Oxford University, have provided pharmacological proof of concept that the treatment with OR0005 may modify the progressive clinical manifestations of the Niemann-Pick Disease type C. In addition to the marked disease modification, biochemical analyses of CNS lipid storage and analyses of the CNS penetration of HSP70 show that it enters into the brain and it reduces the pathological lipid storage burden in the selected neurodegenerative models. Meaningful improvements were observed in biological and pathological variables including the symptoms arising from compromised CNS functionality. A manuscript is being prepared for submission in Q4 2013.

Cynomolgus monkey (Macaca fascicularis) presents 100% interspecies (humans and non-human primates) homology of HSP70. The cynomolgus monkey is therefore considered to be the most suitable non-rodent species for the required toxicity studies. Results are expected before the initiation of the clinical development programme.

Clinical Development

NP-C is an extremely rare disease. The estimated prevalence in the EU is 0.1 cases in 10,000. Clinical symptoms of NP-C and natural disease progression show a broad inter-individual variability. This reality represents a significant challenge for clinical development design. In order to obtain an efficacy signal in the clinical development setting, the participating patient population must be as homogeneous as possible regarding metabolic and immunological parameters, and clinical and biological disease status.

The clinical programme is as follows:

  • A PK, safety and dose escalation Phase I study is under preparation to be started in 2014.
  • The initiation of enrolment of NP-C patients for the “observational study” has now been set up by second half of 2014,
  • The Phase II interventional Clinical trial in the already selected population will follow. This study will evaluate safety, efficacy and quality of life within 52 weeks follow-up upon biological, clinical and imaging markers. The objective of the study is to define the clinical benefit of OR0005 in the NP-C patients.

Regulatory Steps

Close and fruitful interactions with health authorities (national and EMA/FDA) are going on to review the progress in research and development outcome. Scientific advice meetings were already held from 2011 to 2013 with the Danish Agency (DMKA), Swedish Medicinal Products Agency (MPA), United Kingdom Medicines an Healthcare products Regulatory Agency (MHRA) and EMA (July 2013). The EMA’s scientific advice enhanced company’s focus in key areas like the selection of representative biomarkers based on expertise, knowledge and understanding of newer definitions and publications.

Plans for protocol optimization

Orphazyme concentrates efforts to increase the standards in protocol optimization and clinical operations procedures. We are confident that these improvements will bring even higher certainty in regulatory steps for the benefit of NP-C patients.

We will keep you informed about the latest development and timelines as more information is made available.

Click here for a summary of the information available from Orphazyme originally provided & developed by the NPD UK Group ~ Sept-Oct 2013

[Nov 5th, 2013 ~ blg]

 

“The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the Canadian Chapter of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the CCNNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”