Pfizer’s Push For Antisense Licensing Is Closely Aligned With Current Target Indications – Pfizer Inc. (NYSE:PFE)

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Pfizer (PFE) announced that it had licensed an antisense drug from Akcea Therapeutics (AKCA), which is a majority-owned affiliate of Ionis Pharmaceuticals (IONS) and Pfizer. This push to license the antisense drug AKCEA-ANGPTL3-LRx is a good move because of the possible target indications that Pfizer can go after with it. It also makes sense that Pfizer already has several other drugs in its pipeline that are targeting NASH and other metabolic diseases. This antisense drug is currently being evaluated in a mid-stage study, with data expected in the first half of 2020. It won’t be long before Pfizer knows where or not this drug works in treating certain metabolic disorders.

Massive Deal, With Massive Market Potential

As I highlighted above, this is a massive deal for Pfizer. However, the opportunity to obtain AKCEA-ANGPTL3-LRx is highly ideal. That’s because it can be used to treat a range of metabolic diseases such as:

  • Non-alcoholic fatty liver disease (NAFLD)
  • Type 2 Diabetes (T2D)
  • hypertriglyceridemia (Elevated fat triglycerides in the blood)

AKCEA-ANGPTL3-LRx is currently being explored in a phase 2 study for all three indications I listed above. Pfizer gets the opportunity to use this drug to target all these large market indications. For instance, the NAFLD market is expected to reach billions of dollars in the coming years. NAFLD may ultimately lead to patients with NASH. The NASH market is expected to reach $21.5 billion by 2025. This is just one of the market opportunities I highlighted above. This is great, but why is Akcea moving forward in licensing this drug out? It’s because Akcea is committed to maintaining itself in treating rare diseases (smaller markets). It feels that Pfizer has a stronger marketing team and commercial approach in going after larger market indications. It feels that Pfizer is more capable of handling any potential commercialization for AKCEA-ANGPTL3-LRx. This idea is backed up by a quote from the interim CEO of Akcea Damien McDevitt, Ph.D. :

AKCEA-ANGPTL3-LRx has the potential to treat people suffering from certain cardiovascular and metabolic diseases. Given the unmet medical need for this patient population and the broad market potential, we believe Pfizer’s expertise and breadth of experience in cardiovascular and metabolic diseases makes it well suited to accelerate clinical development of AKCEA-ANGPTL3-LRx, and to deliver it to patients in need of additional therapies for these life threatening diseases”

This clinical product was developed by the use of Ionis’ Ligand Conjugated Antisense (LICA) technology platform. The rational reason for using this product involves the gene known as ANGPTL3. It is said that reduced levels of ANGPTL3 can contribute to a decreased risk of diabetes and cardiovascular disease. A preliminary phase 1/2 study was important in determining whether or not this clinical antisense product could be advanced forward. It was shown that patients were able to achieve a dose-dependent reduction in several key measures in the study that proves the drug has an effect on ANGPTL3. These key measures were reductions in:

  • LDL-Cholesterol
  • Total cholesterol
  • Triglycerides in the blood

It is expected that results from the currently ongoing phase 2 study will have results readout by first half of 2020. If such results are positive for all 3 indications, that would be highly positive for Pfizer. Under the terms of the agreement, Akcea and Ionis will be eligible to receive $250 million as an upfront license fee. Akcea has decided that it will give its part of the upfront fee it will receive (the $125 million) as Akcea common stock to Ionis. That’s alright anyways because both companies will be eligible for the possible milestone payments totaling $1.3 billion. This includes tiered royalties on net sales should this product reach the market. I believe this is a good deal for both Akcea and Ionis. That’s because they would no longer be responsible for development of the drug past the phase 2 study. Once the phase 2 study is completed, Pfizer is going to be responsible for the development, regulatory aspect, and sales costs associated with the antisense drug. That makes sense considering that Pfizer has a lot of cash on hand to handle the rest of the costs associated with AKCEA-ANGPTL3-LRx.

NASH Focus Has Always Existed For A Decade Of Development

This latest deal with Akcea for an antisense drug to be developed for NASH and T2D is definitely not out of the ordinary for Pfizer. That’s because it has already been working on a NASH pipeline for quite some time. It currently has a pipeline dealing with three phase 2 studies and one phase 1 study for the treatment of NASH. The clinical products in question that are being explored in these studies are:

  • PF-05221304 – An Acetyl CoA-Carboxylase (ACC) Inhibitor
  • PF-06835919 – Ketohexokinase (KHK) Inhibitor
  • Combo of PF-05221304 and PF-06865571 – ACCi and DGAT2 Combination
  • PF-06865571 – Diacylglycerol O-Acyltransferase 2 (DGAT2) Inhibitor

As you can see above, Pfizer already has an extensive NASH program being explored with several drugs already. Most of these NASH drugs are currently being explored in phase 2 studies. The addition of AKCEA-ANGPTL3-LRx as an antisense for NASH only works to add another shot on goal. That’s not all either because last year Pfizer even went as far as to team up with another big pharmaceutical company by the name of Novartis (NVS) for a potential NASH combination treatment being developed. The goal of the partnership is to use Pfizer’s NASH drugs above in combination with Novartis’ Farnesoid X receptor (FXR) agonist tropifexor. The ability to explore combinations is another key positive for Pfizer’s NASH program.


The ability of Pfizer to license AKCEA-ANGPTL3-LRx from Akcea is very good news. Especially, when you consider that this antisense drug can be used not only for NASH but also for other indications like Type 2 Diabetes (T2D) and hypertriglyceridemia. A phase 2 study is currently underway for AKCEA-ANGPTL3-LRx and data is expected to be released by the first half of 2020. The risk here is that there is no guarantee that the phase 2 study will yield positive data for all 3 indications noted above. That’s always a risk when running clinical studies. However, as I have highlighted above, Pfizer was already working on developing several drugs for NASH. There are 4 ongoing studies that Pfizer has with its drug alone. On top of that, Pfizer doesn’t want to leave its program hanging on just its NASH drugs alone. That’s why last year, it had developed an agreement to combine its NASH drugs together with Novartis’ tropifexor to be explored in some studies also. This adds many more shots on goal for NASH. With all the programs Pfizer has ongoing for NASH, it has a good chance to succeed in this large market indication.

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I’m currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33.50% discount price of $399 per year.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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