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Status report on the potential market of NASH and its actors in Q3 2016

The market remains important and globally estimated at about $ 40 billion from 2022.

The market  is segmented by the severity of the disease and also geographically, Europe and USA are the first targets

The severity of the disease is also an important factor as it impacts both the ease of diagnosis, the number of patients and the cost of treatment.

The cost of treatment will mainly depend on the targeted patients.

The cost recently proposed by Intercept Pharmaceutical for its Ocaliva in the treatment of PBC (about $ 70,000 annually) provides an indication of their pricing strategy without it being really significant.

This price is arguably a waiting strategy in anticipation of hypothetical market access of NASH. A high price leaves them plenty of time to refine their future pricing strategy on the NASH market that will most likely be lower.

The small size of the PBC market is a good excuse to do so because the weak results obtained by Ocaliva on a ad hoc surrogate endpoint in the clinical study that allowed them AMM does not call for such a high price.

There is indeed a dilemma for laboratories having a molecule with a broad spectrum activity in NASH.

Either they limit their target to cirrhotic and pre-cirrhotic patients market with a higher sale price but few patients, and, because of this high cost, can’t  target the rest of the numerous least affected patients.

Either they target all patients but then they plan a much lower price and bet on the development of widely available diagnostic tools.

Diagnostic tools are becoming more advanced and we can imagine that they will be marketed by 2019 to allow the diagnosis of moderately ill patients, but their use by the medical world will not happen in a day and will take probably take several years.

Meanwhile, it is easier to identify the most affected with cirrhosis and whose symptoms become apparent patients. CONATUS in its presentation estimated that to date, between 70% and 95% of cirrhotic patients are diagnosed when only 15% to 20% fibrotic patients (F2 F3) are diagnosed!

According to published studies, the number of patients who reached the stage of cirrhosis is between 4 and 9% of patients with NASH.

These patients will probably require a dual therapy treating NASH and specifically liver fibrosis. These patients usually have dyslipidemia and type 2 diabetes, wich must not be worsened.

Since the prognosis can be quickly engaged to patients with cirrhosis and that the cost of a liver transplant, only existing proposed treatment is very high (currently $ 530,000 in the United States of America), the cost treatment may be high too if its effectiveness is proven.

Many analysts agree on an average annual cost of NASH treatment around $ 15,000. 

In my view, this cost will be bearable for the most affected patients with a vital prognosis committed in the short or medium term.

I think it is a cost that can be applied to only the first few years of marketing, or for a specific combo targeting NASH and fibrosis and reserved for the most advanced patients in their illness.

This ability to Combo often mentioned by the industry is not neutral, because besides the possibility of increasing the effects of a treatment combining several molecules, its  also allows labs to segment markets and to charge very different prices  for these drugs combo depending on the size and severity of market segments.

I am convinced that this is the strategy pursued by GILEAD and justifies its many acquisitions of molecules targeting NASH very early in their development.

Meanwhile, labs believe in short-term market for the sickest patients. This is why, at first, it seems logical that their strategy is to give priority to this market, even to change their pricing strategy as and as the number of diagnosed patients increases.

Regarding the distribution of market it should be noted that recent studies show a prevalence of NASH in Asia much higher than originally planned. We will closely monitor the license agreements for this area of the world.

The laboratories in the race are always the same, some labs like Novartis have also joined the race but their studies are at the very beginning and we will focus on laboratories with Phase 3 and 2b ongoing.

The two laboratories in phase 3 are INTERCEPT Pharmaceutical (USA) and GENFIT (France)

In terms of timing, they are neck and neck, neither one nor the other will give the number of patients already enrolled in their study but the two announced an end to forecasting recruitment in the first half of 2017.

Sign that recruitment is a sensitive point and not so easy , INTERCEPT  has launched a website dedicated to the recruitment of the REGENERATE study.

TOBIRA announced its Phase 2b results in NASH with his cenicriviroc, they are bad regarding their NASH criterion (down 2 pts of NASH score without worsening of fibrosis) but interesting on improvement of liver fibrosis, it opens up a door to negotiate with one of the other players in the NASH to develop a combo targeting the very advanced patients as explained above. 

They announced preparing a Phase 3 on liver fibrosis, it remains to estimate their ability to find funding for this phase 3. We will therefore monitor their news announcements in the coming months.

GILEAD should end its phase 2b for simtuzumab targeting liver fibrosis in a few weeks, few leaks on the net spoke of bad results but it is difficult to trust rumors, if that were the case it is quite possible that GILEAD resume then its acquisitions in NASH, and cenicriviroc (Tobira) with positive results in fibrosis would be a perfect target to replace simtuzumab in the portfolio of Gilead.

GALMED seems to have great difficulty in recruiting for their ARREST study on Aramchol in NASH with almost a year behind the original schedule, they now announce a end of recruitment at the end  of  2016 and results of Phase 2b third quarter 2018.

They spent distribution agreements for Asia with upfront payments' to fund in part the ongoing study.

CONATUS hope the results of the ENCORE study evaluating the NF-action emricasan on induced NASH cirrhotic patients in 2018, 

It should be noted that their target is only cirrhosis, they also target alcohol or hepatitis C induced cirrhosis 

Their phase 2 study showed efficacy only on the patient with a MELD score high (> = 15)

They explain in their presentation that the market is more  in cirrhosis than in fibrosis but their study STILL NC (NCT02686762) target fibrosis F1 to F3 (with restrictions for F1 and excluded patients cirrhosis?

GENFIT announced they intent to launch a study in pediatric NASH and their Phase 3 RESOLV'IT in the NASH will include 200 patients with F1 fibrosis to explore this segment of patients ignored by competitors.

Finally, a phase 3 study is supposed to be launched shortly for sarogiltazar in NASH but its design has not yet been published.

To be continue

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