Sangamo's Hemophilia A Results Light A Spark And Push Program Into Pfizer's Hands


  • Sangamo Therapeutics reports positive results from its phase 1/2 open-label study using SB-525 to treat patients with Hemophilia A.
  • The global hemophilia market is expected to reach $17 billion by 2026.
  • Sangamo had $351.6 million in cash as of March 31, 2019, with funds expected to last for at least the next 12 months.
  • The FDA gave SB-525 regenerative medicine advanced therapy (RMAT) designation, which has many added benefits, like more interactions with the FDA and potential for Fast Track and Breakthrough Therapy designation.
  • Pfizer will take over development of SB-525, and the next step is likely for a registration study to be initiated upon discussions with the FDA.
  • This idea was discussed in more depth with members of my private investing community, Biotech Analysis Central. Get started today »

Sangamo Therapeutics (NASDAQ:SGMO) reported positive results over this past weekend dealing with its phase 1/2 study using its gene therapy to treat patients with Hemophilia A. With this positive data on hand, the program can be given to Pfizer (PFE) to carry it forward as it progresses it in the clinic and towards potential regulatory approvals. It is highly likely that the next advancement for SB-525 is to be explored in a registration trial, which will be done by its partner, Pfizer. This will be a key catalyst event for Sangamo and its shareholders.

Gene Therapy Data Possibly Enough For Pivotal Trial Push

The phase 1/2 study was quite successful. It was noted that Hemophilia A patients treated with SB-525 had a sustained increase in Factor VIII levels. Before diving into the data itself and why I think it's a game changer for the biotech, it's important to get an understanding of Hemophilia A. Hemophilia A is a rare bleeding disorder that is caused by a deficiency of a functional plasma known as clotting factor VIII. Without this protein in place, patients tend to bleed often with no cause. The main form of treatment is patients getting infusion treatments, which must be done on a routine basis. That's because these patients need to keep enough factor VIII to keep bleeding episodes/symptoms to a minimum.

This is where SB-525 from Sangamo Therapeutics was able to shine in the most recently reported data. The open-label phase 1/2 study is known as Alta, and it recruited patients with severe Hemophilia A. Already you can see a hardship. If you look at the link above for Hemophilia A, you would see that severe patients have <1% of factor VIII in the blood. That means this population is the hardest to treat, because the factor levels are so low to begin with. They require the most constant type of treatment with intravenous infusion of recombinant products (prophylaxis). The study included about 10 patients and were spread out across 4 dose cohorts, which are:

  • 9e11 vg/kg - 2 patients
  • 2e12 vg/kg - 2 patients
  • 1e13 vg/kg - 2 patients
  • 3e13 vg/kg - 4 patients

The results were presented at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH) on July 6, 2019. If you look above, the best-performing cohort out of all of them was the 3e13 vg/kg dose of SB-525.

That's because these 4 patients benefited the most out of all the doses. Don't get me wrong. All patients in the study achieved dose-dependent increase in factor VIII levels, but the highest dose is what will get Sangamo to move forward towards registration studies. Patients 7 and 8 that were treated with the 3e13 vg/kg dose of SB-525 were able to have normal range of factor VIII levels on week 24 and 19 respectively. Patient 9 and 10, with the same higher dose, had rapid factor VIII activity that was in line with the other prior patients (7 and 8) at weeks 6 and 4 respectively. This is all solid news, but what can possibly make it superior over every other treatment option? It all boils down to a potential game changer in the Hemophilia A space for severe patients. The study noted that all patients given the 3e13 vg/kg dose of SB-525 had no bleeding events at the time of cut-off to evaluate the data. On top of that, no patient has needed factor replacement therapy through an intravenous infusion during that time. These improvements for Factor VIII in severe Hemophilia A patients was seen on average over a 24-week period. What this means is that so far, after 24 weeks of being given SB-525, these patients have been able to avoid routine therapy. There is still a risk. It is key to see if this solid batch of data continues with a durable response longer than 24 weeks. That still remains to be seen. It means the next set of data out many months from now will show if the response lasts longer than 24 weeks on average.

Then, it's important to highlight how the FDA feels. The FDA was happy to give SB-525 gene therapy the regenerative medicine advanced therapy (RMAT) designation for this patient population. The reason why is because this gene therapy proved to treat/cure a disease with a huge unmet medical need. What's the big deal about this designation? For starters, it gets early interactions with the FDA to discuss regulatory matters. It also provides automatic benefits toward Fast Track designation and Breakthrough Therapy designation. The global hemophilia market is expected to reach $17 billion by 2026. That's including Hemophilia A, Hemophilia B and Hemophilia C. However, even just including Hemophilia A alone, it is a multi-billion dollar market opportunity.

With this phase 1/2 study reporting positive results, what are the catalysts that could carry the stock higher after this? I believe investors should watch out for a milestone payment from Pfizer. That's because this program has already been partnered out with Pfizer. As a reminder, Sangamo received an upfront payment of $70 million upon establishing this partnership. It can also potentially receive up to $300 million in milestone payments for development and commercialization of SB-525. The company could also receive $175 million for any other product dealing with Hemophilia A. This is key, because now that Sangamo is just about done with the phase 1/2 study, Pfizer will take over everything else involved with this program. This includes the rest of the clinical trials, regulatory filings and commercialization. This is where another catalyst opportunity comes into play. Once Sangamo and Pfizer sit down and discuss the next steps with the FDA in detail, it is highly likely that they will start a registration study for SB-525.


According to the 10-Q SEC Filing, Sangamo Therapeutics has cash, cash equivalents, and investments of $351.6 million as of March 31, 2019. The company expects that its current cash on hand will be enough to fund its operations for at least the next 12 months. This is including any potential milestone payments, along with the April 2019 offering that was made to sell shares of common stock. In my opinion, it's quite possible that the company may do a cash raise. It's not guaranteed to, but it might do so to make sure it has a good cushion of cash. In either case, the stock should start to stabilize now that it has been able to get solid clinical data for its Hemophilia A study.


The positive results from the phase 1/2 study using SB-525 to treat patients with Hemophilia A is good news. Especially since those given the highest dose of 3e13 vg/kg achieved the best outcome possible. That is, they had gone through a 24-week period without any bleeding events. What's the risk that remains? The biggest risk is that while this data was good news, it's important to see whether this carries forward for a longer period of time. In other words, if the activity of the gene therapy continues to work past the 24-week period, that will prove long and durable responses for SB-525. Another item to note is that the trial was done with a very small group of patients. With Pfizer taking over the program, it will likely have to run a registration study with a larger group of patients. There is no guarantee that similar or superior results will be achieved for this next study. Still, Sangamo is in good shape, as it has achieved its part of the deal. Now, Pfizer must move to push the program forward the rest of the way.

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.

This article was written by

Terry Chrisomalis profile picture
Actionable ideas on small-large cap biotech stocks through deep analysis.

I am the Founder of Biotech Analysis Central, A subscription service on Seeking Alpha's Marketplace. If you want to learn more about biotech investing or you want to check out my biotech analysis you can do so with a free 2-week trial to my service. Just hit the "Learn More" button on the bottom of the Marketplace Research Tab. I have a Bachelors of Applied Science Degree In Technology Management, Industrial and Business Services Management from St. Petersburg College Florida. I have been investing in biotech stocks for many years, and I prefer to invest as a long term investor. With that In mind I seek stocks that have long term value! I primarily Like to Invest In biotechnology stocks and I accept the risks. I Write for the Healthcare Sector and Stock market in general. I contribute to Seeking Alpha.

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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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