Pharmacyclics, Inc. (PCYC): Will This Be the Breakthrough of All Breakthrough Therapy Drugs?

Page 1 of 2

In July 2012, the Food and Drug Administration Safety and Innovation Act was signed into law; and little did we know at the time, but the drug development process was about to be altered forever. This bill expanded many of the existing governing powers of the FDA, but also added one intriguing new provision — the breakthrough therapy designation.

Recognizing unique therapies
This new designation, as described on the FDA’s website, is to “assist drug developers to expedite the development and review of new drugs with preliminary clinical evidence that indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases.”

Pharmacyclics, Inc. (NASDAQ:PCYC)

Put even more simply, the breakthrough therapy designation is a monumental step in potentially bringing clinically superior drugs to market years ahead of the schedule they’d normally have to follow. Previously, drug developers had to run three separate clinical stage trials. Now, for drugs with the breakthrough therapy designation, preliminary data, (i.e., phase 1/2) can be used as the precursor for a new drug filing, shaving anywhere from one or maybe multiple years off the process of bringing a revolutionary drug to market.

According to the FDA, since Oct. 1, 2012, the agency has received 61 separate breakthrough therapy designation requests. Through June 28, 2013, it had granted 23, denied 15, and had an additional 23 pending.

What’s perhaps more remarkable is the fact that Pharmacyclics, Inc. (NASDAQ:PCYC) has three of those 23 approved breakthrough therapy designations for its lead experimental drug, ibrutinib. Ibrutinib, which is also licensed to Johnson & Johnson (NYSE:JNJ) subsidiary Janssen Pharmaceuticals, was designated as a breakthrough therapy for patients with chronic lymphocytic leukemia, mantle cell lymphoma, and Waldenstrom’s macroglobulinemia. The big potential indication here is CLL, which is the most common adulthood leukemia and occurs in 113,000 people in the U.S. By comparison, MCL diagnoses number about 5,000 each year.

Is this the breakthrough of all breakthrough therapies?
But, if you thought all of the fun stopped at the actual breakthrough therapy designation, then you have another thing coming. Yesterday, Pharmacyclics, Inc. (NASDAQ:PCYC) and Johnson & Johnson (NYSE:JNJ) took the first step toward proving whether this new designation pathway is going to have some merit by filing for a new drug application for ibrutinib.

Source: ppdigital, Deviantart.com.

The trial results, published in The New England Journal of Medicine last month appear to be unmistakably in favor of an eventual approval for ibrutinib. In the MCL trial across both cohorts (those who had taken Takeda Pharmaceuticals‘ Velcade and those who were Velcade-naive), ibrutinib delivered a 68% overall response rate with a median response duration of a whopping 17.5 months. The results in the CLL and small lymphocytic leukemia, or SLL, trial were even more impressive, with ibrutinib delivering an overall response rate across the two doses being tested of 71%. Furthermore, estimated progression-free survival at 26 months was estimated at an incredible 75%!

The results sort of speak for themselves, but even more than that it speaks to the breakthrough in the NDA process via the new breakthrough therapy designation. Ibrutinib, whether it likes it or not, will be the guinea pig of the breakthrough therapy drug approval process, thus making it the breakthrough candidate of breakthrough-designated drugs.

In addition, by moving ibrutinib along via this expedited pathway, it gives a previous small fry in the biotech sector, Pharmacyclics, Inc. (NASDAQ:PCYC), a chance to kick around the big boys like AbbVie Inc (NYSE:ABBV) and Celgene Corporation (NASDAQ:CELG). AbbVie’s experimental late-stage CLL and SLL drug, ABT-199, was placed on clinical hold in February following the death of two patients from a condition known as tumor lysis syndrome. This hold will delay any additional trials until the proper dosing is worked out and gives ibrutinib a chance to run away in treating CLL/SLL. For Celgene, which just received the added indication for Revlimid to treat MCL, it’s a kick in the pants. Revlimid’s MCL trial overall response rate was 26% compared to the 68% ORR in ibrutinib’s mid-stage trial.

Not so fast, optimists…
As exciting of a time as this might be for biotechnology and pharmaceutical companies, there are a lot of factors still to play out here. In trials, ibrutinib showed what I feel is reasonable safety, with most adverse events being reported as grade 1/2 (i.e., not serious). But, with such a small subset of patients being tested in some of these trials, the potential concerns over drug safety and their effect on the body over time cannot be discounted.

Another factor to consider is whether there will be sufficient data from an early stage or mid-stage trial to merit confidence in physicians and insurers to prescribe a drug. Again, I’m not trying to pick on ibrutinib at all here, but simply point out that with a small subset of patients, even the stamp of approval from the FDA may not be enough to get insurers or physicians to go along with prescribing the drug.

Page 1 of 2
Comments
Insider Monkey Small Cap Strategy
Insider Monkey Small Cap Strategy

Insider Monkey beat the market by 52 percentage points in 24 months. Our beta is only 1.2 (don't click this link if beating the market isn't important to you).

Lists

The 10 Largest Pharmaceutical Companies In the World

The 10 Most Expensive Android Apps

The 9 Most Expensive Designer Bags in the World

The 7 Most Expensive Real Estate in the World

The 10 Most Expensive eBay Items Ever Sold

The 10 Most Expensive iPhone Apps

The 9 Most Expensive Designer Shoes in the World

The 10 Most Expensive Cigarette Brands

The 10 Most Expensive Law Schools in the US

The 10 Best Wall Street Movies

The 10 Most Expensive Golf Clubs Ever Sold

The 10 Most Expensive Golf Memberships

The 10 Best Disney Characters Ever Created

The 8 Best Foods for Gaining Weight

The 10 Most Expensive Colleges in the World

The 7 Most Memorable Ad Campaigns of All Time

The 7 Most Expensive High Schools in the World

The 10 Electric Vehicles with the Longest Range

The 10 Cities with the Worst Drivers in the World

The 10 Most Expensive Dresses Ever Created

10 Islands to Visit Before You Die

10 Famous Celebrities Who Needed Rehab

The 15 Countries with the Largest Oil Reserves

The 10 Most Overused Excuses in the World

The 5 Best iOS Apps You Can’t Get on Android

5 Companies Damaged By Social Media Blunders

The 10 Most Legendary Blues Songs

The 10 Most Lawless Places in the World

4 Reasons China is a Threat to the US

The 17 Most Sugary Drinks in the World

The 10 Most Ruthless Rulers in History

The 10 Greatest Generals in History

Top 8 Travel Destinations for 2015

The 10 Safest Dog Breeds for Children

The 10 Most Stolen Vehicles in the US

The 7 Most Expensive Celebrity Weddings

The 10 Best LoL Teams in the World

Top 10 Worst Marketing Campaigns Ever Produced

Top 5 Diets that Help You Lose Weight

The 10 Best Ways to Stay Awake

7 Artists That Switched Musical Genres

The 10 Most Expensive Cities to Live in New Jersey

The 10 Best High Schools in New York

The 10 Countries With the Least Gender Inequality

The 6 Biggest Musician-Manager Feuds

The 10 Countries with the Cheapest Gas Prices

The 7 Most Theatrical Bands of All Time

The 8 Worst Band Breakups of All Time

The 10 Most Important South American Leaders

The 7 Most Successful Casting Show Winners

Subscribe

Enter your email:

Delivered by FeedBurner

X

Thanks! An email with instructions is sent to !

Your email already exists in our database. Click here to go to your subscriptions

Insider Monkey returned 47.6% in its first year! Wondering How?

Download a complete edition of our newsletter for free!