Cannabis is firm’s ‘gateway drug’

August 2, 2010

Cannabis is firm’s ‘gateway drug’

Medical pot Has also designed a plant to produce a therapeutic enzyme

Postmedia News August 2, 2010

A reputation of any kind, even for a business, is hard to shake.

And when your company is the only federally licensed medical marijuana producer in Canada, that’s the first thing people think of when they hear the company’s name, says Brent Zettl, Prairie Plant Systems Inc.’s president and CEO.

But providing cannabis to patients authorized by Health Canada isn’t the Saskatoonbased company’s only focus, even if sales of the CanniMed herbal treatment account for between 60 and 65 per cent of its revenue, Zettl says.

“It’s kind of like our gateway drug, if I can use that term,” he says in an interview. “It’s our gateway drug to these other compounds that we’re planning to have produced in plants.”

For nearly 10 years, PPS has produced medical marijuana on a contract basis for the federal government. Originally grown in the deep depths of a decommissioned mine in Flin Flon, Man. -known unofficially as the Ganja Mine -PPS moved its hydroponic operation out of the town on the Saskatchewan border when the contract with the mine’s owner ended last summer.

PPS is still growing the marijuana for the government, but the location of the operation must remain confidential under federal regulations,

Casandra Kyle

Zettl says.

“North of the 49th and in between the Atlantic and the Pacific and Arctic oceans, that’s where it is,” he says.

Although the high-profile, legal and still-controversial practice of growing medical marijuana is what PPS is best known for, Zettl hopes the distinction will change over time.

“I think a lot of people forget this is a contract we bid on,” he says. “But we had a bigger purpose in mind. … Although it’s our reputation at this point, we’re trying to change that.”

The company, along with the Plant Biotechnology Institute, has designed a plant to produce a therapeutic enzyme known as adenosine deaminase, or ADA. The enzyme, Zettl explains, is part of the body’s immune system and is deficient in people with severe combined immunodeficiency disease, a condition often referred to as bubble-boy syndrome.

People with the disease must undergo enzyme-replacement therapy, Zettl says, and at the moment, most of the ADA used in the treatment is purified from cow spleens.

PPS’s ADA takes the animal out of the equation.

The cannabis side of the business, he adds, has helped PPS move forward with its therapeutic enzyme studies, with growing conditions, industry standards and pharmaceutical credibility supporting its scientific work.

linkback url: http://www.montrealgazette.com/health/Cannabis+firm+gateway+drug/3350160/story.html

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Gene Therapy Plus Chemotherapy Ups Odds for Success in Treating ‘Bubble Boy’ Disease

May 24, 2010

Gene Therapy Plus Chemotherapy Ups Odds for Success in Treating ‘Bubble Boy’ Disease

ScienceDaily (May 24, 2010) — Gene therapy combined with chemotherapy may lead to immune system improvement in patients with bubble boy disease, according to a study to be presented at the American Society of Gene & Cell Therapy 13th Annual Meeting.

Beginning in 2001, researchers from the University of California Los Angeles and the National Human Genome Research Institute, a division of the National Institutes of Health, treated 10 patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID), commonly referred to as bubble boy disease.

Four patients received ongoing enzyme replacement while six patients had their enzyme replacement therapy stopped and received low doses of chemotherapy, which reduces bone marrow density to create room for the transplanted cells to multiply. Fifty percent of the latter group demonstrated a restored immune response that parallels normal levels. The remaining patients show some independent production of the adenosine deaminase enzyme, but not enough to elicit independent immune responses.

“This trial extends prior findings and shows that ADA gene transfer can lead to restoration of a functionally protective immune system,” said Donald Kohn, MD, the clinical trial sponsor and principal investigator at UCLA. “Because our trial was done in two phases, we can directly compare the effects of these approaches and show that withholding the enzyme and giving pre-transplant chemotherapy is markedly more effective.”

Patients with ADA-SCID lack the enzyme adenosine deaminase and, as a result, have little or no immune system, leaving them vulnerable to infection and illness. ADA-SCID is one of the most promising conditions for treatment with gene therapy and has been the source of early successes in the field. Earlier trials in London and Milan have also showed restored immunity in patients treated with the gene therapy protocol.

A Phase II trial on the treatment is underway; three patients have been treated thus far. Research also continues into more efficient vectors for gene transfer, which scientists hope will result in a quicker, more robust immune response.

linkback url: http://www.sciencedaily.com/releases/2010/05/100524105403.htm


Last Chance for a Cure

February 26, 2010

Last Chance for a Cure

Up to 8 months of isolation await a young boy suffering from “bubble boy” disease.Reporter: Chris Woodard
Email Address: cwoodard@nbc15.com

POSTED: Friday, February 26, 2010 — 6:15 p.m.

Tonight 10-year-old Joshua Osborn is about to go through something many of us could never imagine.

Eight months of isolation, closed into the same room, at the same hospital await him.

His parents hope it can bring an end to his 10-year struggle with a rare disease.

After years of waiting and hoping the Osborn’s are getting their shot.

Since birth life has been a little different for Osborn.

His mother Julie says, “You think you’re coming home from the hospital with a healthy baby boy and to find out something different was a shock.”

Joshua was born without an immune system. He suffers from Severe Combined Immune Deficiency, or SCID-ADA, a one in a million disease.

Julie says, “Did I want to go down this road? Not necessarily. But when I put it in perspective of what other families deal with I do feel blessed that it hasn’t been worse.”

SCID is more commonly known as bubble boy disease, named after David Vetter a boy from Texas who spent all but the last few days of his 12 year life in a germ free bubble while doctors looked for a cure.

Things have changed a lot since then.

Joshua’s dad Clark says, “We’ve always probably been a little more cautious than the average parent but not a lot.”

Julie says, “Almost everybody that asks me says does he live in a bubble. If they don’t know him they still wonder.”

Now 10-years-old Joshua has been through a lot, including a failed bone marrow transplant and a week in intensive care at 3-years-old that almost ended his life.

Clark says, “No contest that was definitely the hardest time with him.”

Since then he’s had regular trips to Duke Children’s Hospital in North Carolina, monthly IVs and weekly shots in his legs all to basically give him a temporary immune system.

Joshua says, “It’s sort of painful.”

But for this little fighter there is new hope.

Julie says, “It was a relief to know we were going but it’s going to be a busy week to get it all done.”

Earlier this week Joshua’s parents got word he’ll be the 7th child selected to participate in a new gene therapy trial at Duke Medical Center.

The family will relocate to the hospital where Joshua will be forced to isolate himself in a hospital room for anywhere between 3 and 8 months. They leave Sunday.

Clark says, “It was an intimidating thought… with him not being able to leave that room.”

Julie says, “To know that he could possibly be completely healed and not have to have medicines anymore, it’s worth it.”

The most simplified explanation is that his own marrow is taken out, the defect with the immune system is fixed and they put it back in.

Josh says, “I know it’s not going to be dangerous but there are risks I heard. I’m not really afraid of that.”

For mom and dad it means picking up and leaving all while Julie is pregnant again.

They’re expecting a son who they just found out will be born with the same disease.

Clark says, ” When I told him that his brother was going to have the same condition he did, he just smiled and said, well I’m OK. I’m fine.”

Julie says, “I think god gives us what we can handle and there is purpose.”

It’s a life filled with question that may finally have an answer, but it will take a few months of isolation to find out.

Joshua says, ” I’m not really afraid of that no.”

If you want to follow along on Josh’s journey his dad has created a web page that Josh himself will be updating as often as he can.

To check it out just head to http://www.JoshuaOsborn.com

Clark says the best thing that’s come out of Josh’s sickness is newborn testing.

In January of 2008 Wisconsin became the first state to begin testing newborns for the disease which Clark says increases the survival rate from 50 -percent to close to 100-percent.

The Osborns were very active in lobbying for that testing and still hope all states will jump on board.

linkback url http://www.nbc15.com/news/headlines/85584522.html


Enzon Stockholders Approve Sale of Specialty Pharmaceutical Business

January 27, 2010

Enzon Stockholders Approve Sale of Specialty Pharmaceutical Business

BRIDGEWATER, NJ – January 27, 2010 – Enzon Pharmaceuticals, Inc. (Nasdaq: ENZN) (“Enzon”) announced that at a special meeting held this morning, its stockholders approved the sale of Enzon’s specialty pharmaceutical business to the sigma-tau Group (“sigma-tau”).

Enzon’s specialty pharmaceutical business includes four marketed products, Oncaspar®, Adagen®, DepoCyt®, and Abelcet®, as well as a manufacturing facility in Indianapolis, Indiana.

As previously announced, Enzon has entered into a definitive agreement to sell its specialty pharmaceutical business to sigma-tau for $300 million plus an additional amount of up to $27 million based on success milestones. Enzon will also receive royalties of 5 to 10 percent on incremental net sales above a 2009 baseline amount from Enzon’s four marketed specialty pharmaceutical products through 2014.

The asset sale is expected to be completed later this week.

linkback url: http://www.enzon.com/index.php?id=208


Enzon sells specialty drug business for $300M

November 9, 2009

Enzon sells specialty drug business for $300M

(AP) – Nov 9, 2009

BRIDGEWATER, N.J. — Enzon Pharmaceuticals Inc. said Monday it is selling most of its business to Italian drugmaker sigma-tau Group and will focus on its experimental cancer drugs and technologies.

Enzon will get $300 million upfront, along with milestone payments of as much as $27 million. Sigma-tau could also pay Enzon royalties on Enzon’s four approved drugs through 2014. Sigma-tau is also acquiring a manufacturing facility in Indianapolis in the deal.

Enzon stock reached an annual high of $9.60 on the news. That was its highest price since December 2007.

The specialty drugs sigma-tau is buying are cancer drug Oncaspar, fungal infection treatment Abelcet, meningitis drug DepoCyt, and Adagen, which is used for severe combined immunodeficiency disease, or “Bubble Boy Disease.”

In 2008, Enzon announced plans to spin off its biotechnology business into an independent company so it could focus on developing treatments for cancer. It canceled those plans in December due to the weak state of the financial markets. With the specialty drug unit sold, the company said it will focus on potential cancer treatments including the drug candidate Peg SN38.

The transaction, which is subject to financing and approval by Enzon shareholders, is expected to close in the first quarter next year.

In morning trading, Enzon shares rose 43 cents, or 4.7 percent, to $9.41.

linkback url; http://www.google.com/hostednews/ap/article/ALeqM5jV-rR103S0X1M8TGto7sRi86EyLgD9BS41Q00


DellaCamera Claims Enzon CEO Can (Almost) Never Be Fired

June 10, 2009

DellaCamera Claims Enzon CEO Can (Almost) Never Be Fired

DellaCamera Capital gathered what it claimed was more than 50 percent of Enzon’s shareholders into a hotel conference room in Midtown New York today to make the case for staging a coup against CEO Jeff Buchalter.

The presenter, DellaCamera research chief Richard Mansouri, dropped some interesting new nuggets into the debate over whether Buchalter should be removed by his shareholders (the stock is down by more than 50 percent over the last five years). Among them:

* Buchalter’s contract requires an unusual 12 months’ notice if the company wants to fire him — and as he’s also contractually required to be Enzon’s board chairman, it is thus virtually impossible for the board to remove him. “We’ve never seen anything like this,” Mansouri said. While dual CEO/chairman roles often create unaccountable “imperial” bosses with built-in conflicts of interest, “The fact that it is contractually mandated is even worse,” he said. Buchalter gets about $5 million a year in compensation even though his company only makes $197 million in revenues.
* Buchalter announced he was exploring the sale of his specialty pharma division in August 2008, and then concluded that effort with no sale in November. No explanation or detail has been offered as to exactly why that effort failed, Mansouri said. “We are not at all satisfied with this lack of information.”
* Enzon has gone through $55 million in “other” R&D costs on “discontinued” products since 2005, and that “other” expenses are the company’s largest R&D cost.

“Something is wrong. Something is very wrong here,” Mansouri concluded.

The audience was subjected to this PowerPoint slideshow, much of which rehashed the case for change that Ralph DellaCamera’s (pictured above) group previously made in a June 1 SEC filing. Get BNET’s take on the back story here.

The chief weakness in the DellaCamera case is that the group won’t say who it thinks should replace Buchalter (pictured below) and nor will it reveal what it believes the company’s strategy should be. “We don’t have any other agenda,” Mansouri said.

This hole will become more important as it gathers shareholder support, because despite its flaws Enzon isn’t a complete disaster: It made $6 million in profit on $48 million in revenue in Q1, both small increases from the year before. Shareholders faced with a stay-the-course plan from Buchalter and no plan at all from DellaCamera might be forgiven for sticking with the devil they know.

In the wings is Carl Icahn, who owns 7.8 percent of Enzon. Icahn just succeeded in putting two supporters on Biogen’s board and two on Amylin’s board. He already has one guy on Enzon’s board.

The event set the stage for an exciting battle, but don’t get too excited: When pressed by BNET, Mansouri all but admitted that the historical success rate of shareholder votes to remove CEOs is zero.

linkback url: http://industry.bnet.com/pharma/10002625/dellacamera-claims-enzon-ceos-contract-says-he-can-almost-never-be-fired/


Biotech Pioneer Life Science Leader, May 2009

May 29, 2009

Biotech Pioneer

Life Science Leader, May 2009
Written by: Cliff Mintz

Dr. Abraham Abuchowski, COO and president of Prolong Pharmaceuticals, grew up on a chicken farm in rural Vineland, NJ. Realizing that chicken farming wasn’t in his future, Abe attended Rutgers University, receiving a B.S. degree in 1970 and Ph.D. in biochemistry in 1975. During his thesis work, Abe developed a novel protein delivery system called PEGylation, which involved chemically linking the polymer polyethylene glycol (PEG) to proteins. He continued his work on protein PEGylation at Rutgers, first as a postdoctoral fellow, then as research assistant professor and Leukemia Society scholar until 1983, when he left to form Enzon, one of New Jersey’s first publicly traded and profitable biotechnology companies.

During his 13-year tenure as president and CEO of Enzon, Abe and his team optimized protein PEGylation, which led to the development of the first three FDA-approved PEGylated products. Recognizing the need for biotechnology advocacy, Abe formed and served as the first chairman of the Biotechnology Council of New Jersey in 1994, which still exists today and has helped grow the New Jersey biotechnology industry. Abe left Enzon in 1996 and spent the next nine years in semiretirement — playing with his hobbies (woodworking, aviation, auto mechanics, and feeding chickens) on his farm in northwestern New Jersey and helping would-be entrepreneurs start up new biotechnology companies. In 2002, realizing that protein PEGylation still had enormous potential for the biopharmaceutical industry, he formed Prolong Pharmaceuticals, a company that focuses on developing novel PEGylated protein-based products.

Abe, who attributes his entrepreneurial success to martial arts training and participation in the McDonald Corporation management trainee program, is a biotechnology industry pioneer and is often referred to as the “Father of PEGnology.” Over the past 25 years, he has received numerous scientific and business achievement awards — most recently, the Dong Sung Pharmaceutical Award in Seoul, Korea for the development of PEGylation technology and the USC Marshall School of Business Outstanding Achievement Award.

I had an opportunity to catch up with Abe, who has two new PEGylated protein products in clinical development, to chat about the events that led to the development of protein PEGylation, his views on entrepreneurship, and the current state of the biotechnology industry.

How did you come up with the idea for protein PEGylation?

Abuchowski: My thesis advisor, Dr. Frank Davis, long believed that chemical conjugation of polymers to proteins may help alleviate some of the deleterious and potentially life-threatening side effects commonly associated with nonhuman proteins like insulin (isolated from animal sources) that were routinely used in the 1970s. Frank reasoned that the addition of an inert polymer to a protein might dampen host immune responses and reduce the side effects following the parenteral administration of nonhuman proteins. Because this was Frank’s pet project, he required all graduate students who wanted to work with him to “take a crack at protein conjugation.” Despite knowing that many graduated students had failed before me, I enthusiastically agreed to work on the protein conjugation project — I was young, brash, and naively overconfident at the time!

While there were many potential conjugation candidates to choose from, Frank decided on PEG mostly because it was a ubiquitous substance found in a wide variety of cosmetics, ointments, suppositories, and even food products. Further, and perhaps more importantly, PEG was generally regarded as safe by the FDA, which meant if we were successful, regulatory approval of PEG-protein conjugates would be less challenging. After developing some rudimentary protein conjugation chemistries, I began PEGylating any proteins that I could get my hands on. I PEGylated more proteins than I can remember. After creating the PEG-protein conjugates, I tested them (and their un-PEGylated counterparts) for immunogenicity, turnover rates, and circulating half–life times in a wide variety of standardized animal models that were being used at the time. And, as predicted, the PEGylated proteins were virtually non-immunogenic, had lower turnover rates, and exhibited vastly improved circulation times as compared with un-PEGylated controls.

Because at the time that we developed protein PEGylation, the use of proteins as drugs was very limited — the biotechnology industry was just coming to fruition — there wasn’t much excitement or commercial interest in our technology. Most scientists generally regarded PEGylation as an anachronistic technology. Nobody realized at the time that protein PEGylation would help to improve the characteristic and delivery of virtually any protein-based biotechnology product.

Why did it take over 25 years for PEGylation to be accepted as a bona fide protein delivery technology?

Abuchowski: Before I began working on protein PEGylation, many high-powered academic laboratories had diligently tried (and failed) to chemically modify proteins to reduce their immunogenicity — a vexing problem that severely limited the use of nonhuman therapeutic proteins in the 1970s. Also, at the time, many prominent scientists thought that the newly emerging field of genetic engineering could be used to overcome the immunogenicity problems that plagued nonhuman, protein-based therapies. Consequently, many of my peers mistakenly viewed protein PEGylation as a competing (rather than an adjunctive) technology and refused to acknowledge our work.

Despite several publications and reams of scientifically sound data, most scientists in the late 1970s refused to believe that protein PEGylation really worked! In retrospect, I suspect many of them refused to believe that a young chicken farmer from New Jersey could accomplish something that many of them failed to do! Nevertheless, I was convinced that protein PEGylation had the potential to revolutionize the emerging biotechnology industry. Further, I realized, unlike my colleagues, that I didn’t have to discover new proteins to make drugs — I could simply PEGylate existing, well-characterized ones that seemed like good drug candidates — a strategy that took others over 20 years to recognize.

After leaving Rutgers in 1983, I was determined to commercialize protein PEGylation. However, because pharma wasn’t interested in the technology, I had to form my own company (Enzon) to confirm PEGylation as a reliable protein delivery system. It was a huge gamble because there were many naysayers and nonbelievers in the scientific community who, I think, were hoping that I would fail. To overcome persistent doubts about the technology, we decided to PEGylate a protein that could be used to treat an otherwise fatal disease — thinking, that if we were successful, FDA would have no choice but to approve the drug. This resulted in development of Adagen, the first and only approved treatment for severe combined immunodeficiency disease (SCID). Interestingly, Adagen was approved in 1990 on the strength of data we collected from eight patients with SCID.

Because Adagen was approved as an orphan drug, the pharmaceutical industry continued to doubt the commercial and financial potential of protein PEGylation. To overcome these lingering doubts, we developed Oncospar (pegaspargase), an anticancer treatment for acute lymphocytic leukemia (ALL). ALL was an obvious choice to showcase the utility of protein PEGylation because patients with ALL frequently developed immune hypersensitivity to asparaginase, the only recognized ALL treatment at the time. PEGylating asparaginase rendered the enzyme nonimmunogenic, eliminated the hypersensitivity reaction, and brought asparaginase-refractory patients with ALL into remission. Oncospar received FDA approval in 1994.

While Enzon was able to gain approval for two PEGylated products in 11 years — a remarkable feat for a biotechnology startup — the commercial potential of protein PEGylation wasn’t fully realized until 2001, when Schering-Plough and Enzon introduced PEG-Intron as a treatment for hepatitis C infections. Within a year, PEG-Intron had captured 65% of the hepatitis C market and reached about a billion dollars in sales. Protein PEGylation had finally hit the big time!

What advantages does protein PEGylation offer over other potential protein delivery technologies?

Abuchowski: PEGylated proteins are generally nonimmunogenic, exhibit increased circulating half-lives (i.e. are longer acting), require fewer injections for therapeutic efficacy, have vastly improve tolerability and safety profiles, are resistant to proteolytic digestion, and are readily excreted in urine and feces. Sometimes, when the wrong chemistry is used to conjugate PEG to a protein, PEGylation may interfere with the biological or therapeutic activity of the molecule. Aside from that, I can’t easily think of any negative attributes associated with the technology. Believe me — I have tried for over 30 years to find problems with the technology, but I haven’t been able to!

As far as I know, there aren’t any competing FDA-approved protein delivery technologies on the market right now. There are several new delivery systems being developed, including several that use human serum albumin and other human endogenous proteins as delivery vehicles. To date, the FDA has approved eight PEGylated therapeutic proteins — most of which are multibillion-dollar blockbuster products. Further, there may be as many as 30 PEGylated proteins in clinical development. I think it is safe to say that protein PEGylation has become the gold standard of the protein delivery industry right now.

There are currently only a handful of companies like Amgen, Roche, Nektar, Schering-Plough, and Enzon that have taken advantage of PEGylation to deliver protein-based drugs. Why haven’t more biotech companies leveraged the power of protein PEGylation?

Abuchowski: As most scientists will tell you, there is a substantial amount of art — not just technical prowess — when it comes to optimizing scientific methods. While many of the numerous PEGylation chemistries out there are straightforward and easy to use, the key to creating a successful PEGylated product is choosing the best chemistry and the right-sized PEG that will impart the desired biological characteristics on a product. At present, there are numerous PEGylation chemistries and multiple different molecular species of PEGs that can be used to PEGylate proteins. The learning curve is steep for protein PEGylation, and unless somebody on your development team has prior experience with it, PEGylation can be a real hit-or-miss exercise.

In my opinion, many companies that try to PEGylate proteins spend too much time studying PEGylation chemistry and not enough time thinking about ways to optimize the PEGylation process for scale-up and manufacturing purposes. The days of charging 10 times the development costs of a product are gone — it is all about manufacturing efficiencies and pharmacoeconomics (the scientific discipline that compares the value of one pharmaceutical drug or drug therapy to another) now. It is no longer economically feasible for companies operating on slim margins to use a reaction that consumes 90% of your starting materials and yields only 10% product. Further, the manufacturing process has to be facile and lean. This is because the pharmaceutical operators who are charged with manufacturing a product are typically not Ph.D.s but people with high school and associate degrees. Processes need to be uncomplicated, robust, and scalable to be effective.

My almost 30 years in the biotechnology industry has taught me that the most critical step in creating a product is not science but the manufacturing and formulation processes. This is because patents that originally protect the scientific aspects of a product typically expire early in a product’s life cycle. Patents that are developed around manufacturing, formulation, and delivery are the ones that typically extend the life of a product and help to protect lucrative blockbuster drug franchises. A great example of this is Amgen’s EPO franchise. The composition of matter patents for EPO expired in 2004, but additional formulation and manufacturing patents will protect the franchise through 2017. I believe that the companies that understand manufacturing and commercialization will always trump companies that may have scientifically superior products.

What other products may benefit from protein PEGylation?

Abuchowski: Any protein-based product could possibly benefit from PEGylation. However, before PEGylating a protein, it is important to understand its biology and intended use. For example, early on, I PEGylated insulin and learned that increasing its circulation time was not such a good idea because it can easily lead to hypoglycemic shock. I think that you may see an increased use of PEGylation in single-chain antibodies, RNAi and oligonucleotide products, and even in nonviral-based gene delivery technologies.

While I believe some proteins can be delivered via the nasal or oral routes, these routes of administration are typically less efficient and not as cost-effective as parenteral delivery. That said, I think parenteral administration of protein-based drugs is here to stay.

What is the future of PEGylation in the biopharmaceutical industry? Are there any competing protein delivery technologies that may supplant it?

Abuchowski: I think that protein PEGylation has finally hit its stride, and that we will see more applications of the technology to biotechnology products in the future. There are many prospective therapeutic proteins out there that can’t be transformed into drugs because they may be too toxic, have poor circulating half-lives, or are turned over too rapidly following injection. PEGylation affords biopharmaceutical manufacturers an opportunity to increase a protein’s circulating half-life, improve its safety and tolerability profiles, and lower the cost per patient without sacrificing therapeutic efficacy. I think in today’s fast-paced and highly competitive market, the question is no longer whether or not to PEGylate, but when!

There are several companies working on new parenteral delivery technologies for biotechnology drugs. Most of these involve creation of fusion proteins, which genetically link a human carrier protein like serum albumin to a protein of interest. While these technologies are promising, they are more costly than PEGylation, and to my knowledge, none has been successfully scaled to commercial manufacturing levels. It is noteworthy that PEGylation became the de facto drug delivery system for protein-based drugs only after the introduction of PEG-Intron in 2001. This means it took the pharmaceutical industry about 19 years from the time I discovered the technology to recognize protein PEGylation’s utility and commercial potential. I suspect it may take as long or longer for a competing technology to supplant PEGylation as the gold standard for parenteral protein-based drug delivery.

You formed Enzon in the mid-1980s before there was a biotechnology industry to speak of and then formed Prolong Pharmaceuticals 20 years later when biotech had finally hit its stride. Many new entrants into the biotechnology industry contend that the drug commercialization paradigm has changed, and that it is much harder to get drugs approved today than in the past. What are your thoughts on these assertions?

Abuchowski: When I started Enzon there were no CROs, CMOs, or any other type of third-party vendors to help support operations. Consequently, we had to become a vertically integrated biopharmaceutical company so we could develop our products, manufacture them, and then sell them. While it was a lot of grueling, hard work, I was fortunate to have personally experienced each stage of the biopharmaceutical drug development paradigm, ranging from drug discovery through clinical development and marketing and sales. These experiences provided me with skill sets that have served me well over my 30 years in the biotechnology industry and enabled me to form Prolong in 2003 with much less money than it took to start Enzon. That said, the biotechnology industry landscape is much different than it was 25 years ago.

Almost every aspect of drug development can now be outsourced to competent and reputable third-party vendors. Consequently, there is absolutely no need for biotechnology companies to become vertically integrated today. CEOs who are intent on vertical integration are, in my opinion, wasting shareholders’ money and stroking their own egos. This is why so many biotechnology companies have failed and will continue to fail. In today’s world, companies are now expected to do a lot more with much less.

I don’t think gaining regulatory approval and commercializing biotechnology drugs is more difficult than it was in the 1980s. There is certainly more competition, but I think that is good for the industry as whole. While I was at Enzon, I worked very closely with FDA regulators and managed to get two drugs approved in 11 years. However, there is no question that, over the past decade or so, the relationship between the biotechnology industry and the agency has become very strained. Many biotechnology industry CEOs are angry and frustrated because the FDA isn’t approving new drugs as quickly as it has in the past. This has resulted in an intensive lobbying effort to relax the drug approval process. While this may lead to quicker drug approvals (and more money for drug company CEOs and their shareholders), I don’t think it would be in the best interest of the American public.

There is no question that the FDA could be improved and operated more efficiently and effectively. The agency has suffered from poor leadership, inadequate funding, and personnel shortages for the past eight years or so. However, I do believe the people who work at the FDA have the best interests of the public in mind.

Will the biotechnology industry be able to weather the current economic downturn?

Abuchowski: Yes, but it will be painful for inefficient companies and those who have no products in clinical development or on the market. Like others, I believe that about 60% of existing biotechnology companies will fail or be bought by big pharma over the next few years. This is because most of these companies are operating on venture capital money (which is getting harder to find) and haven’t been able to develop any products despite “being in business” for five or more years. Ironically, the venture capitalists may turn out to be their own worst enemies because they funded too many ill-conceived startups hoping to get quick returns on their investments without fully understanding the industry. The most important thing I learned about biotechnology companies is that while science and patents may be important, the success or failure of a biotechnology company invariably hinges on solid regulatory, commercialization, and manufacturing strategies. Companies that lack these are doomed to fail. I don’t expect this paradigm to change anytime soon as the biotechnology industry continues to evolve in the 21st century.
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