Clinical update: Orphazyme announces update to its Niemann-Pick disease type C clinical programme — AIDNPC

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


After several interactions with regulatory authorities in the US and Europe it has become clear that registration of arimoclomol will require the performance of a double-blinded clinical trial with a control group.

The control group will comprise a number of patients that will receive placebo for a period of 12 months during the conduct of the trial. However, patients participating in the trial will not be required to discontinue any registered medication that they are receiving when entering the trial. The trial design will allow two patients to receive arimoclomol for every patient receiving placebo.

After 12 months, all patients will be offered to receive arimoclomol.

Patients who experience abrupt and rapid deterioration during their participation in the trial will be offered a transfer to a “rescue arm” of the study where they will be offered open label arimoclomol.

“We have now reached a conclusion on the overall clinical design. We are aware that every day counts in the lives of NP-C patients, and we put our full effort into the design of a study that meets the requirements of pace and robustness to be able to provide all NP-C patients with an effective new therapy option within the shortest possible time frame,” says Dr. Anders Hinsby, Chief Executive Officer, Orphazyme ApS.

“We realize that using a control group for a period requires extra dedication from the patients and their families, but this trial design improves the robustness of the study and it allows us to reduce the duration of the trial to 12 months. To reach our common goal of making a meaningful difference in the lives of NP-C patients, we will match the dedication and commitment of the patients and their families,” says Dr. Carlos Camozzi, Chief Medical Officer.

The AIDNPC programme

AIDNPC comprises two stages:

Stage 1: An observational study, which is a 6 month study used to map the individual patient’s baseline disease progression rate. The patients stay on current standard of care (including Zavesca and/or symptom management). The stage 1 will provide a baseline of each individual patient that will increase the likelihood of documenting a robust effect of arimoclomol within the following period of 12 months.


Every patient completing the observational study is offered to enter Stage 2.

Stage 2: The interventional study, starting immediately after completing stage 1, is a double-blind, controlled study, where patients will receive either arimoclomol or placebo in addition to their ongoing standard of care treatment for a 12-month period. Patients will be randomized 2:1, which means that two patients will receive arimoclomol for every one patient receiving placebo. Neither the patients nor the treating physicians will know during the 12 months whether they are receiving placebo or arimoclomol (i.e., ‘double-blind’).

After completing the 12-month intervention study every patient will be offered arimoclomol.

The primary endpoint of the study is disease progression rate, which, supported by biochemical readouts, will provide the basis for an application for market authorization at the completion of the study.

Inclusion criteria

The participants in the AIDNPC trial programme must be between 2 and 18 years of age and diagnosed with Niemann-Pick disease type C. Moreover, they must be presenting with at least one neurological symptom. For information of further criteria for inclusion/exclusion in the observational baseline study, please refer to (identifier NCT02435030).

About arimoclomol

Arimoclomol is a new chemical entity with a proven safety record in man: Seven Phase I clinical studies have been conducted in healthy volunteers. Arimoclomol is administered orally three times daily (t.i.d.) and it is presented in a hard-gelatin capsule, from which the content is easily dissolved in liquids or food stuff for best possible patient comfort and compliance.

For more information, visit the dedicated AIDNPC program web site

Meet Orphazyme, represented by Dr. Anders Hinsby (CEO), Dr. Thomas Kirkegaard Jensen (CSO), Dr. Carlos R. Camozzi (CMO) and Karen Møller (Clinical Trial Manager), at the Niemann-Pick UK Annual Family Conference the 18th–20th of September and at the bi-annual INPDA Face-to-Face Meeting in Mainz, Germany, the 24th of October.

Further information: 
Carlos Camozzi, CMO of Orphazyme

About Orphazyme

Orphazyme ApS is a Danish biopharmaceutical company, which develops paradigm-changing medicines for the treatment of genetic diseases. The lead programme is entering clinical development as treatment for lysosomal diseases. This family of genetic disorders includes NP-C and consists of more than 45 diseases, often affecting children, most of which are currently untreatable and often fatal. Orphazyme has raised more than €37M in seed, Series A and Series B financing from a strong investor base, which includes Novo A/S, Sunstone Capital, Aescap Venture, Kurma Partners and Idinvest Partners. For more information, please visit