The Food and Drug Administration has granted marketing approval ahead of time for Incyte’s (INCY) drug Jakafi (ruxolitinib). The approval is for intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF - indications that represent 80% to 90% of MF patients. The drug Jakafi is an oral JAK1 and JAK2 inhibitor. It is the first in a new class of drugs, known as JAK inhibitors, and the first and only product to be approved by the FDA for MF. The drug is also in Phase III trials for polycythemia vera, in Phase II for essential thrombocythemia, pancreatic cancer and solid hematologic tumors.
There is no doubt in our mind that the approval is, indeed, a tremendous achievement for Incyte. First because the drug is the outcome of the efforts made by the firm’s assembled dream team of scientists in the firm’s laboratory. Second, because MF - a progressive, life-threatening blood cancer - has limited treatment options and patients with this disease suffer bone marrow failure, enlarged spleen (splenomegaly) and other debilitating symptoms, including fatigue, severe itching (pruritus), night sweats, bone pain, and early satiety (a feeling of fullness), leading to impaired quality of life. All these symptoms, including the enlarged spleen, were improved in a high percentage of patients taking the drug. Third, because the enlarged spleen and debilitating symptoms of MF are linked to deregulated signaling in the Janus kinase (JAK) pathway, which is the target of Incyte’s drug Jakafi.
Here is what Srdan Verstovsek, M.D., Ph.D., Associate Professor, Department of Leukemia, Division of Cancer Medicine, the University of Texas MD Anderson Cancer Center and the principal investigator of the COMFORT-I pivotal trial had to say: “Today’s FDA approval of Jakafi has the potential to transform the way we treat myelofibrosis. In this Phase III clinical trial, we observed significant reductions in spleen size and significant improvements in symptoms. Importantly, these benefits were achieved early on, most within a month, and tended to be durable during treatment. In contrast, most of the patients who received placebo saw their spleens increase and their symptoms worsen.”
The FDA approval was granted based on the results from two randomized Phase III trials (COMFORT-I and COMFORT-II), which demonstrated that patients treated with Jakafi experienced significant reductions in splenomegaly (enlarged spleen). COMFORT-I also demonstrated improvements in pain under the left ribs, early feeling of fullness, night sweats, bone and muscle pain and itching. Most patients taking placebo experienced worsening of these same parameters. The COMFORT-I trial compared Jakafi to placebo in patients with primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF. The trial has met the primary endpoint.
The COMFORT-II trial, conducted by Novartis (NVS), Incyte’s collaboration partner outside of the U.S., also met its primary endpoint. This study, which compared the results of Incyte’s drug Jakafi with the best available therapy, demonstrated that 28.5% of patients treated with Jakafi experienced a 35% or greater reduction in spleen volume at 48 weeks, compared with 0% of patients in the best available therapy arm (p<0.0001).
Click here to read the press release, or go to Incyte’s website.
Incyte represents the biotech firms that we believe would provide the cutting edge technologies that would distance the U.S. from the rest of the world. We have no doubt whatsoever that a great deal of America’s future prosperity resides in biotechnology firms like Incyte. Since its inception, Incyte has made sure to provide all the scientific disciplines that can take drug discovery and development to a higher level. Its scientists come from a broad range of backgrounds, bringing with them a tremendous expertise in genomics, bioinformatics, chemogenomics, high-throughput screening, combinatorial chemistry, computational chemistry, structural biology, as well as all aspects of molecular biology, biochemistry, in vitro and in vivo pharmacology, and pharmacokinetics. Over 70 professionals with broad experience in all aspects of clinical development comprise Incyte’s development team.
Incyte’s scientific team and its development strategy and plans have enabled it to select compelling targets and design drug candidates that it rapidly and efficiently moved into preclinical and human clinical trials. This capability has also enabled it to carefully select its partners and conclude partnerships that are fair and lucrative. One can obviously confirm this reality as per the relatively large amount of payments the partners had to pay Incyte for collaborating on its products. It is also evident from the post approval conditions, which are definitely fair to Incyte. The firm has agreements with top pharmaceutical firms, including Novartis, Eli Lilly (LLY) and Pfizer (PFE). We will tackle these agreements with more details in future articles about Incyte.
Nothing will stop this firm from becoming a top-tier biotech firm contributing to the advancement in the treatment of cancer, inflammatory disease and other diseases. Incyte has been capable of progressing 13 internally developed proprietary compounds into clinical development. Medicinal chemistry and the firm’s medicinal chemists have contributed a great deal in this productivity. Additionally, to conduct clinical trials as efficiently and effectively as possible, Incyte has built a clinical development and regulatory team whose task is to deal with clinical research organizations (CROs), expert scientific advisory boards and leading consultants.
What About The Stock Now Following Approval?
We will see a number of analysts announce they are neutral on the stock at this time. We really don’t believe anybody who knows this firm can have no opinion on it at this stage. Our opinion is that these analysts are afraid of the current market status and are yielding to the negative trend taking place right now with regard to the value of drug approvals. Buy on rumors and sell on news, regardless of whether it is good or bad is not serving the biotechnology companies’ purposes, the country’s future, and the majority of investors who usually wait for years for the drugs to be approved so they can reap the fruits of their patience.
We are already reading articles posted on the Internet making general statements that FDA approval of drugs is not important and that what is important is the launch. No credit for good news that is a fact, but a lot of questioning based on unsubstantiated skepticism, which has no place anywhere in the world of reality.
We vote for Incyte against all baseless skepticism.
Disclosure: No positions.