Ozanimod

Ozanimod


The Science

Ozanimod is an immunomodulatory drug developed by Celgene that is currently in Phase III clinical trials for the conditions of relapsing Multiple Sclerosis (RMS) and Ulcerative Colitis (UC).

Multiple Sclerosis is a condition in which the bodies immune system attacks myelin, a substance designed to protect neurons and help nerve signals travel quickly around the nervous system. This strips the myelin from the neurons and leaves scars known as lesions or plaques. This damage disrupts messages travelling along nerve fibres – they can slow down, become distorted, or not get through at all.

Immune cells (Lymphocytes) contain a receptor called a sphingosine-1-phosphate (S1P) receptor. When S1P interacts with this receptor, it causes the egress of Lymphocytes from the thymus and Lymph nodes into lymphatic vessels, from there it can reach the CNS.

The drug acts as a S1P receptor agonist, meaning it binds to the receptor and produces a biological response. In this case, the response causes Lymphocytes (White Blood Cells) to become trapped in the bodies Thymus and Lymph nodes. In turn, this means the WBCs cannot enter the brain and spinal cord and cause damage to myelin.

Ozanimod is taken as tablet, once daily. This is a lot less intrusive than other rrMS drugs which are mainly given intravenously.

The Upside

In a phase II study, 258 people with relapsing remitting MS took one of two doses of ozanimod or placebo for 24 weeks.  The main measure was the number of new active lesions seen on MRI.  Ozanimod significantly reduced the number of lesions compared to placebo.


  • SUNBEAM - ozanimod compared to  interferon beta 1a (Avonex)

1346 people with relapsing MS were recruited to this phase III study.  Participants took one of two doses of ozanimod or interferon beta 1a (Avonex) for one year.  For the group taking 0.5mg ozanimod, the annual relapse rate was 0.24, a 31% reduction compared to Avonex. For the group taking 1.0mg ozanimod, the relapse rate was 0.18, representing a 48% reduction compared to Avonex.

Compared to Avonex, both doses of ozanimod also significantly reduced the number of new active lesions seen on MRI.  Changes in brain volume were not significantly different between the two doses of ozanimod and Avonex.


  • RADIANCE - ozanimod compared to interferon beta 1a (Avonex)

This phase III study recruited 1320 participants with relapsing MS who took one of two doses of ozanimod or interferon beta 1a (Avonex) for two years.  The main aim of the study was to compare relapse rates for ozanimod and Avonex.   For the group taking 0.5mg ozanimod, the annual relapse rate was 0.22, a 21% reduction compared to Avonex.  For the group taking 1.0mg ozanimod, the relapse rate was 0.17, a 38% reduction compared to Avonex.   Compared to Avonex, both doses of ozanimod also significantly reduced the number of new active lesions seen on MRI.  Both doses also reduced brain volume loss compared to Avonex.


Multiple sclerosis affects approximately 400,000 people in the U.S. and approximately 2.5 million people worldwide. Approximately 85 percent of patients are initially diagnosed with RMS, compared with 10-15 percent with progressive forms of the disease.

A quick search for the price of Ozanimod found that 25mg could be bought for $656.00. According to the data above, the 1.0mg dose was more effective so I will use that for my calculations.

Ozanimod needs to be taken daily. Thus at $656.00 / 25 mg it would cost $26.24 for 1mg if we were buying in bulk and therefore getting the cheapest price possible (this rises to $31.60 if not bought in bulk).

$26.24 x 365 is a cost of $9577.60 per patient per year.  Now in the US there are 400,000 people with Multiple Sclerosis and 340,000 with Relapsing Multiple Sclerosis meaning in the US there is an approximate total market value of $3,256,384,000 a year.  In the entire world this figure would be approximately $20,352,400,000. Realistically Ozanimod would not have 100% market share of the RMS market. Lets say it gains a conservative 10%, the total sales could be approximately $325,638,400 a year in the US and $2,035,240,000 a year across the world. Other analysts have predicted even more, some around $3 Billion mark and others even up to $5 Billion.

The Downside

The launch of Celgene’s multiple sclerosis drug, ozanimod, was delayed after the company failed to submit key information in its application to the Food and Drug Administration (FDA).

The notice was based on Celgene’s omission of preclinical and clinical pharmacology information on the oral pill, which meant a complete review of ozanimod could not take place. To me this seems like a silly mistake, however it was a large setback for Celgene as it allowed competitor drugs valuable time to launch.

U.S. NDA and EU Marketing Authorization Application (MAA) submissions in relapsing multiple sclerosis (RMS) for Ozanimod are now on-track for Q1:2019.

Ozanimod does belong to the same class of S1P inhibitors as Gilenya, a competing drug, it has been thought these drugs will soon lose exclusivity. Gilenya, for example, will soon have generic forms flood the market.


The Conclusion

To me I see no reason why the drug will not receive approval in 2019. I believe the clinical data is extremely promising and offers significant benefit over existing treatments. The drug has had two successful Phase III trials for RMS, and it is a well known fact that 85% of drugs that have successful phase III trials receive FDA approval. 

The refusal to file notice back in early 2018 was an obvious setback but can be attributed to the drugs previous owner Receptos. Celgene initiated a small study that duplicates the work Receptos did to perhaps overwrite the results obtained by Receptos. This study was not finished before Celgene originally submitted the application for Ozanimod, so the FDA decided to surprise the company by demanding Receptos' data be added to the application. 

Now Celgene's study results will be used and the application can be submitted properly.


-OC


Disclaimer:
I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company who is mentioned in this article. This article is not investment advice, and can't be relied upon by anyone for any reason except, arguably, entertainment purposes. I am not an investment advisor.





https://www.mssociety.org.uk/about-ms/what-is-ms
https://en.wikipedia.org/wiki/Sphingosine-1-phosphate
https://www.mstrust.org.uk/a-z/ozanimod
https://ir.celgene.com/press-releases/press-release-details/2018/New-Analyses-from-Pivotal-Phase-3-Trials-of-Oral-Ozanimod-to-Be-Presented-at-ECTRIMS-2018/default.aspx
https://pharmaphorum.com/news/ozanimod-launch-delayed-by-failures-in-fda-application/
https://www.investors.com/news/technology/how-celgene-gambled-on-ozanimod-and-why-it-didnt-pay-off/
https://www.businesswire.com/news/home/20190107005376/en/Celgene-Corporation-Announces-2019-Financial-Guidance-Key
https://multiplesclerosisnewstoday.com/ozanimod-former-rpc1063

Comments

Popular posts from this blog

Liso-Cel

VK2809 - Viking Therapeutics

Ruxolitinib - Incyte Corporation