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This morning, Actinium Pharmaceuticals (ATNM) announced that the company has received positive Scientific Advice from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) related to the EU approval pathway for Iomab-B. According to the press release, correspondence from the EMA notes that the trial design, primary endpoint and planned statistical analysis of the U.S. pivotal Phase 3 SIERRA trial are acceptable and can serve as the basis for submission of a Marketing Authorization Application (MAA) in Europe.

In addition, the EMA commented that it does not anticipate the need for further standalone preclinical toxicology or safety studies on Iomab-B prior to reviewing the MAA. The EMA did request some supporting data; but, management noted that this information is already being collected as part of the U.S. pivotal Phase 3 SIERRA trial.

The Update We Were Hoping For!

The full acceptance of SIERRA as a registration quality study by the EMA is fantastic news for Actinium. Recall, this process started last fall when management announced they would seek advice from the EMA on the regulatory path for Iomab-B in Europe. Talking to the EMA made perfect sense for Actinium. Iomab-B had been designated as an Orphan Drug in October 2016 and in August 2016, Actinium qualified for "small and medium-sized" (SME) status. Companies granted SME status are eligible for administrative and regulatory support including scientific advice, scientific services, pre-authorization inspection and post-authorization procedures. In addition, financial support is provided with fee reductions up to 100% in certain instances.

In February 2017, I laid out some near-term catalysts for Actinium investors. With respect to Iomab-B, I noted that the Phase 3 SIERRA trial initiated last July 2016 continued enrollment with the first planned Data Monitoring Committee (DMC) update expected around the middle of the year once 25% of the patients are treated. A second DMC update at 50% completion is expected late 2017 / early 2018. There will be a third update once 75% of the target population has completed, and top-line safety and efficacy data are expected during the second half of 2018.

The second thing I noted was that investors could expect an update on the Scientific Advice from the EMA shortly. Below is a snap-shot of what I said in February 2017. Based on today's press release from Actinium, it looks like the company received the most bullish response we were hoping for.
 

Time To Partner?

Now that Actinium has a confirmed path forward for Iomab-B in the EU, it likely makes sense for management to seek out a commercialization partner for the drug in Europe. In February 2017, Executive Chairman, Sandesh Seth, noted in this presentation to investors at BioCEO that the company's options in Europe include licensing Iomab-B for distribution following receipt of Scientific Advice from the EMA. With that advice now in hand, I think Actinium moves forward with talks to partner Iomab-B in Europe. This sets up the potential for a nice deal announcement later in the year.

A Big Opportunity

The market opportunity with Iomab-B is significant. According to the American Cancer Society, there will be an estimated 21,380 new cases of AML in the U.S. in 2017. Another 27,500 new cases arise in Europe each year. The U.S. NCI estimated 73% of newly diagnosed AML patients are over the age of 55 and there are no currently approved treatment options for these elderly patients with relapsed or refractory disease. It's an estimated patient population of approximately 15,000 individuals, the majority of which cannot tolerate standard conditioning regimens that allow them to progress to a BMT. By offering a more effective and more tolerable solution to these elderly r/r AML patients, Actinium is sitting on a sizable market opportunity with Iomab-B. Standard conditioning regimens cost between $50,000 and $200,000. They can also take as long as 28 to 42 days. At $75,000 for a course of treatment over only six days, Iomab-B targets a $1.15 billion opportunity.

As noted above, it makes sense for the company to partner Iomab-B in Europe. This will allow for expansion of the market opportunity and likely an upfront cash payment that will fund the U.S. commercialization plans. In the U.S., the bone marrow transplant market is highly concentrated and very conducive to specialty promotion by Actinium alone. There are only ~150 BMT centers in the U.S. and >50% of all procedures take place at the top 30 centers. The top 10 centers do ~30% of all procedures and the majority are participating in SIERRA today. The centers include the Fred Hutchinson, Memorial Sloan Kettering, Baylor, and MD Anderson. 
 

These are all organizations which Actinium has a solid collaborative relationship from previous clinical studies and the company's scientific advisory board (SAB). Recall that Iomab-B was invented by researchers at the Fred Hutchinson Cancer Research Center and licensed for development to Actinium Pharmaceuticals.

Conclusion

Today's news is very positive for Actinium. Enrollment in SIERRA continues on plan and there are DMC updates expected around the middle of the year and again potentially at year end. SIERRA was obviously already incredibly important to Actinium, but today's announcement that the trial will serve as the basis for regulatory submissions in the EU is fantastic news. It saves the company an enormous amount of time and money and potentially paves the way to a commercial partnership for Iomab-B in Europe later this year.