Today, patients with CML (chronic myeloid leukemia) who take Novartis' (NYSE:NVS) drug Gleevec and follow it up with the newer medications, are projected to survive an average of 30 years, says Dr Brian Druker, the genius inventor behind both Gleevec and Iclusig.
This is a far cry from the three- to five-year prognosis that was standard when Druker began practicing medicine in the 1980s.
Last December the FDA approved Ariad's (NASDAQ:ARIA) Iclusig to treat adults with CML and Ph+ ALL (Philadelphia chromosome positive acute lymphoblastic leukemia), two rare blood and bone marrow diseases.
It is the only drug that can treat patients with the dreaded T315I mutation, something no other approved drug in the field is able to do.
The drug is also effective "across the board" for patients with CML, regardless of whether they have resistant mutations or what form those mutations take, said Michael J. Mauro, MD, who manages CML clinical trials at the Knight Cancer Institute at the Oregon Health & Science University (OHSU) and partners with Dr Druker.
On the negative side, Iclusig carries a Boxed Warning alerting patients and doctors that the drug can cause blood clots and liver toxicity.
Iclusig is off to a great start in the U.S. The first quarter sales of $6.4 million represents pure demand for Iclusig as it does not include any inventory stocking.
The company's goal is to make Iclusig the most widely prescribed drug for the CML in treatment resistant or intolerant patient population and leave no patient behind.
The company estimates that through April over 400 patients in the U.S. were treated with Iclusig obtained through physician prescriptions of the commercially supplied drug. This does not include any patient using the initial 30-day Quick Start supply, which is free of charge, or any clinical trial participation. Discontinuation rates appear to be low at less than 10 percent.
As expected, approximately 1 out of 4 prescriptions are being written for patients who have failed only one existing drug, which makes Iclusig a true second line treatment.
It turns out that less than 10 percent of the patients receiving Iclusig by prescription have the T315I mutation, once again, supporting the view that the drug applies to all resistant and intolerant patients.
Approximately 60 percent of the patients are in the chronic phase. These patients will have the opportunity to benefit from Iclusig therapy over more prolonged periods of treatment.
At the end of April, about 300 unique physicians prescribed Iclusig. Approximately 70% of the Iclusig prescribers are community-based physicians. The fact that community physicians have a 2 to 1 majority over academic physicians shows that Iclusig is being prescribed broadly among hematologists, oncologists in diverse clinical settings, not just investigators who participated in the clinical trials.
The payer coverage for Iclusig is widespread, and Iclusig has been added to the NCCN (National Comprehensive Cancer Network) Guidelines.
In the first 3 months of Iclusig launch, according to the IMS National Prescription Audit, the total prescriptions for Iclusig were tracking nearly 40 percent higher than Novartis' Tasigna did at the same point in its launch. And Tasigna is now doing over $1 billion in annual global sales.
Ariad management also predicts that Iclusig's total prescription volume will soon surpass that of bosulif from Pfizer (NYSE:PFE), which was approved 4 months before Iclusig.
Even doctors are unhappy with the soaring prices of cancer drugs.
100 cancer doctors and researchers specializing in treating the blood cancer CML argued the pressing need to lower cancer drug prices in a paper published recently in the journal Blood. "Unaffordable drug prices in CML may be preventing many patients from accessing these life-saving drugs," say the doctors.
The paper highlighted the jaw-dropping prices of three cancer drugs approved for treating CML by FDA in 2012: Iclusig at $138,000 per year; Synribo from Teva (NYSE:TEVA), at $28,000 for induction and $14,000 per maintenance course; and Bosulif, at about $118,000 per year. The authors also note that of the 12 drugs approved by FDA last year for various cancer indications, "11 were priced above $100,000 per year."
Ariad's policy of "no patient should be left behind regardless of their ability to pay" means that the company goes out of its way to help the financially strapped.
In addition to a free drug program for the uninsured, Ariad is offering co-pay assistance programs to bring out-of-pocket costs down to $20 per month for commercially insured patients. The company also donates to charitable foundations that provide funding for Medicare patient co-pays.
Clinical trials are continuing in order to expand Iclusig's indications.
The global EPIC trial, comparing Iclusig to Gleevec, is enrolling patients with newly-diagnosed CML. Interim analysis on efficacy and safety is expected by the third quarter of 2014.
In Japan, Ariad has now transitioned to the Phase 2 portion of the Phase 1&2 trial in patients with resistant and intolerant CML, and applying the recommended dose of 45 mgs given once daily. The company is planning to file for approval in Japan next year. Japan is the third-largest CML market in the world.
In the U.K. enrollment in the Spirit 3 trial will begin next quarter, in collaboration with the U.K. National Cancer Research Institute with about 1,000 patients at 172 research sites.
A Phase 2 trial of Iclusig in patients with gastrointestinal stromal tumors (GIST), will begin in the second quarter of 2013. Iclusig is a potent inhibitor of c-kit, which is the target in most cases of GIST, and can overcome many of the mutations which cause resistance to Gleevec and sutent from Pfizer, the current standards of care.
Each year, about 5,000 Americans are diagnosed with CML, a cancer of the white blood cells.
CML is caused by the abnormal gene BCR-ABL, which occurs when two chromosomes swap portions of their DNA from the BCR and ABL genes during cell division. This abnormality is called the Philadelphia chromosome and causes an excessive and unregulated production of white blood cells by the bone marrow.
Iclusig U.S. net sales in the first quarter of 2013 were $6.4 million using the sell-through method of revenue recognition.
Additionally, during the first quarter, the company shipped $2.1 million of Iclusig to patients in France through the ATU, or Temporary Authorization for Use program. Revenue related to these shipments will be recorded when the list price of Iclusig is determined, which will be later this year.
In March 2013, Ariad received a positive opinion from the European advisory board. Approval in Europe is expected by June 2013.
Ariad's net loss for the first quarter of 2013 was $64.7 million, larger than the net loss of $55.9 million for the same period 2012.
Expenses increased by $18.2 million from the first quarter of 2012 to the first quarter of 2013, reflecting the investment in commercial launch of Iclusig in the U.S. and the preparations for the anticipated commercial launch in Europe.
On March 31, 2013, the company had cash and cash equivalents of $398.3 million, compared to $164.4 million at December 31, 2012.
Ariad's other drug, AP26113, has shown that it can inhibit clinically relevant ALK mutants resistant to Pfizer's Xalkori in patients suffering from non-small cell lung cancer.
The stock price in the past 52 weeks ranged from $15.13 to 25.40, and since March it is trending down. It is currently below both the 50 and 200 day simple moving averages.
Analysts are largely positive on the stock: 18 votes for Strong buy and Buy, 3 votes for Hold and none for Sell according to the current Thomson/First Call poll.
Ariad has high hopes for Iclusig. Martin J. Duvall, Vice President of Commercial Operations, said:
"As with any new product launch, broadening the prescribing base to build physician experience will take time. Armed with the medicine that we believe to be best-in-class and programs driven by talented motivated teams, we look forward to sharing future results and successes in the U.S., Europe and around the world."