CSL Behring

So Long! Extended Haf-Life Factor Sets the Stage

You’ve probably heard about Biogen Idec’s clinical studies of factor with extended half-lives. They aren’t the only ones investigating. CSL Behring just announced that they are starting a global phase II/III, multi-center study to assess the safety, efficacy and pharmacokinetics of recombinant fusion protein.

This recombinant fusion protein links coagulation factor IX with recombinant albumin, called “rIX-FP.” rIX-FP is indicated for the prophylaxis and treatment of bleeding episodes in patients with factor IX deficiency.

Of course, extended half-life factor means potentially fewer injections for patients, and may enable or enhance prophylactic treatment, improving quality of life for patients.

So many patients worldwide have been hoping for gene therapy–a “cure”–but it seems that the next great thing that will come along just might be extended half-life factor. We’re not sure yet which company will deliver first, but keep watching. These are exciting times and the long acting factor race is on!

Book I Just Read
The Hunger Games by Suzanne Collins (Kindle edition)

This best selling novel takes place in post-apocalyptic America, now called–for no reason disclosed– Panem. In fact, there are a lot of things that happen for no apparent reason in this book. Quick synopsis: the powers that be dictate that one teen from every district must compete to the death in the Hunger Games, in retribution for an uprising that happened a long time ago. To the victor goes fame and food to their district. The whole event is televised–reality TV where kids get killed. The heroine narrates the story, and has a pretty flat tone throughout. Think “The Truman Show” meets “Mad Max Beyond Thunderdome” meets The Olympics meets “Rollerball” and you get a great idea what the book is about. It’s not terribly original; the writing is mediocre and the story pretty predictable, characters shallow with no depth of thought or emotion. After killing her first kid, arrow through the throat, the 16-year-old narrator with the flat affect seems more concerned about whether the boy she is with loves her. Someone recommended this to me, but neglected to tell me it’s teen romance fiction, with a lot of killing. Read it if you want to kill time; it’s a few steps up from a comic book. If you want fantasy with substance and depth, try Tolkein. The book seems to be meant for a movie, and reads like a screenplay; I somehow think the movie will be better as a result, coming in March. Two stars/five.

Namaste from New York!


Last Tuesday I dusted off my salwar kameez outfit from Pakistan, hopped on a plane with executive director Martha Hopewell of Save One Life, our child sponsorship nonprofit, and went to New York City for a wonderful fundraiser called “Bollywood Chic,” hosted in the art studio of Hunt Slonem.

The studio takes your breath away: cavernous at 15,000 sf, and filled from ceiling to floor with framed art, statues, and even a museum quality collection of butterflies that was stunning. In the center of the studio: bird cages filled with colorful macaws and doves. Hunt’s specialty is painting birds; you can see his famed parrots on a new Tiffany set of serving ware. The birds are raucous when not being fawned over; they demand attention by screeching. This added an exotic flavor to the evening!

How did we snag the goodwill of an artist of Hunt’s calibre? Through board member Tara Reddi, who is vice president of the Marlborough Gallery in New York, and has a nephew with hemophilia in India.

Guests arrived around 6:30 and stayed till 9, sampling some wonderful Indian-style food, and wine at our bar, and listening to Bollywood soundtrack. Guests could wander freely throughout the studio, which is subdivided into different rooms, each with a theme. The Lincoln room is filled with Hunt’s portraits of Lincoln in many colors. Another room is filled top to bottom with framed paintings of rabbits. Another room is filled with Greek or Italian statues. Another room contained a massive round table and filled with candles, looking like an invitation to a seance! Guests were really intrigued by the decor!

We were pleased to have with us Dr. Ranjan Kulkarni from the Nasik Chapter of the Indian Federation of Hemophilia. She is our program partner there, and has enrolled many children with hemophilia in Save One Life. She just happened to be visiting in New Jersey, which worked out perfectly! The fundraiser gave her a chance to meet two other doctors and sponsors: Dr. Lucy and Dr. Shipra, both attired in lovely Indian dresses!

Save One Life now sponsors over 550 children in nine countries, giving them funding that they would normally never receive. Funding is used for transportation to clinic, to buy medicine, and to pay school fees. The nonprofits that administer the program–our program partners–check in regularly with the children, to ensure they are doing well. So more than just a charity, we are guaranteeing these kids get seen, are known and are helped.

Our deepest thanks to Hunt Slonem for generously allowing us the privilege and pleasure to spend an evening at his studio. Thanks to Tara Reddi for organizing this marvelous event, which required a lot of hard work. Thanks to all the guests who showed up despite the dreary weather, many of whom were friends and patrons of Tara’s and Hunt, and who supported the fundraiser. And thanks to my friends at NHF, whom I was so happy to see present!

Many thanks to two companies who helped underwrite the event: CSL Behring and Inspriration Biopharmaceuticals. All proceeds of the event will go towards our India program, where the bulk of our beneficiaries are located. I’ll be traveling to India in September to check on our program and will be sure to keep everyone posted. Namaste!

For more information or to sponsor a child in need: www.SaveOneLife.net
Also see: www.cslbehring.com, www.inspirationbio.com, www.marlboroughgallery.com

Do Not Pass Go; Do Not Collect $3.1 Billion

The game of “Monopoly” is over– at least that’s what the Federal Trade Commission would have called it. Worried that CSL Behring would become too big after acquiring Talecris, and concerned that there was some antitrust issues, the FTC slapped the wrists of these two billion dollar plus companies and announced it would oppose the merger through the US courts, if necessary. Talecris management decided to withdraw from the $3.1 billion takeover deal by CSL Behring.



The US FTC opposed the proposed merger by alleging that the US plasma market was a tightly-controlled oligopoly, and that the merger would violate antitrust laws.
 CSL Behring has rejected these claims. The merger would have left CSL Behring the biggest plasma manufacturer in the world.

I read that CSL, Talecris and market leader Baxter International of Illinois have 83% of the US market for blood-based drugs; hemophilia plasma-derived products are included in this.

Just a little history: Talecris is the company formed in 2005 when Bayer spun off its plasma division. Bayer produced a plasma-derived factor VIII product called Koate DVI. Talecris inherited it and still produces it. It’s a big company, with 2008 sales of over $1.4 billion. It’s not big in hemophilia in the US, but it is a major player in the immune defificent disorders community.

The proposed sale to CSL Behring is no surprise. I’ve heard from the get-go that management at Talecris would expand the company, and then sell it and reap the profits. Private equity companies Cerberus Capital Management and Ampersand Ventures together bought the plasma division of Bayer for more than $300 million. And now it’s up for $3.1 billion.

So what happens now?

Well, we still have seven licensed manufacturers of factor on the US: Baxter, Bayer, CSL Behring, Grifols, Novo Nordisk, Wyeth and of course, Talecris. I wouldn’t be surprised if more mergers are proposed down the road but the FTC ruling might make it impossible to accomplish. Our community, our industry, is small and carefully watched. And the Obama climate seems to be very antitust-conscious.

Neal R. Stoll and Shepard Goldfein, in the New York Law Journal (November 19, 2008) note, “In his September 2007 statement to the American Antitrust Institute (AAI), President-elect Obama noted that ‘[a]ntitrust is the American way to make capitalism work for consumers.’ President-elect Obama emphasized that, ‘the [Bush] administration has what may be the weakest record of antitrust enforcement of any administration in the last half century,’ and he specifically cited lax merger enforcement for the rising cost of health insurance.” The authors then go on to say that antitrust laws are not good tools to effect economic engineering.

Read more about plasma derived products–their role in hemophilia history and today, in the next issue of PEN.

Do Not Pass Go


Big news from the corporate hemophilia world: CSL, maker of blood-plasma products, including Monoclate-P, Mononine, and Humate-P, has been foiled in its courtship of Talecris, also a maker of blood-plasma products, most notably Koate DVI. “The US Federal Trade Commission (FTC) recommended legal action to block the proposed $3.1 billion purchase of Talecris Bio- therapeutics Holdings,” writes Bloomberg news.

Why can’t CSL buy Talecris? What has lawmakers ruffled up about this? Gobbling up Talecris would place CSL at the top of the blood plasma food chain. This includes hemophilia, as well as autoimmune disorders and multiple sclerosis. Something about this screams violation of antitrust to Washington.

Antitrust law prohibits any activity that restricts free trade and competition between businesses, especially those that would lead a company to a dominant position–a monopoly. So the government is always sniffing out predatory pricing, price gouging, and mergers and acquisitions of large corporations. The FTC website says, “Courts have applied the antitrust laws to changing markets, from a time of horse and buggies to the present digital age. Yet for over 100 years, the antitrust laws have had the same basic objective: to protect the process of competition for the benefit of consumers, making sure there are strong incentives for businesses to operate efficiently, keep prices down, and keep quality up.”

And antitrust laws have been around longer than 100 years. Wikipedia says: “The history of competition law reaches back to the Roman Empire, with the Lex Julia de Annona, enacted during the Roman Republic around 50 BC. To protect the grain trade, heavy fines were imposed on anyone directly, deliberately and insidiously stopping supply ships.”

The term “antitrust” is American, and arose when large American corporations used trusts to conceal business arrangements. Big trusts meant big monopolies, and when you have a monopoly, you can easily control supply and demand, and prices. You have no competition. Have you played the game Monopoly and landed on your friend’s Park Place, the one with a red hotel? Ouch. Consumers have suffered at the hands of those with monopolies.

In America, monopoly is perceived even as a threat to democracy. The FTC website writes: “Free and open markets are the foundation of a vibrant economy. Aggressive competition among sellers in an open marketplace gives consumers — both individuals and businesses — the benefits of lower prices, higher quality products and services, more choices, and greater innovation. The FTC’s competition mission is to enforce the rules of the competitive marketplace — the antitrust laws. These laws promote vigorous competition and protect consumers from anticompetitive mergers and business practices.” http://www.ftc.gov/bc/antitrust/index.shtm

Perhaps the FTC thinks that a CSL acquisition of Talecris would make the company too big for its britches, too powerful? Talecris, owned by the private equity groups Cerberus Partners and Ampersand Ventures, had revenue of about $1.4 billion in 2008. CSL had $2.97 billion in revenue in the year ending June 2008. Together they would dominate the blood plasma field, surpassing Baxter, which is now top dog. Indeed, Talecris, Baxter and CSL already control 83% of the US market. Other producers include Grifols, which makes blood products, and Octapharma, which also makes blood products although its factor products are not yet registered in the US.

What’s next? After the FTC publishes its reasons for preventing the acquisition, CSL will then decide what to do. Wooing a prospective partner when the parents don’t approve is expensive. But not so bad as in Roman times: Under Diocletian in 301 AD businessmen faced the death penalty for violating a tariff system, for example by buying up, concealing or contriving the scarcity of everyday goods. Death penalty? Thankfully we don’t do things gladiator style anymore. If CSL cannot complete the deal, the company will be liable to pay a $75 million break fee to Talecris’s owners, Cerberus Partners and Tribeca Investment Partners, a company spokeswoman said. That should soften any break up. Stay tuned!

Interesting Book I Just Read
The Blue Sweater by Jacqueline Novogratz

Novogratz started her career as a young, starry-eyed do-gooder out to change the world, and got an eye-opener working in Africa in the 1980s for various nonprofits. She had valuable experiences about what works and what doesn’t in helping the poor. Eventually, she returned to the US and founded the Acumen Fund, which finances businesses in the developing world that give jobs to the poor, tackle issues of poverty and offer microloans. From a one-woman show to heading a huge NGO now, her story is very interesting. In many ways it’s an homage to the African women who struggled alongside her and taught her, and learned from her.

But it’s not without problems: the book seems to have been written by two people, or at least in two voices. The first half is all anecdotal, about Novogratz’s efforts in Africa to help local women start a baking business, in great amounts of detail, without stats about poverty, its root causes. I care less about what color dresses the women wore (this seems to come up a lot!) than about how her efforts compare to other NGOs at the time. Her story paints a very accurate picture of what you still can see in Africa and what the challenges are. I would have found it more interesting and useful had she interspersed history, poverty statistics and analysis in it. The second half shifts suddenly to the Acumen Fund and there is almost nothing but statistics, and all success stories. Sounds like a promotional piece in many ways. Gone is the author’s personal style, which was full of feeling and self-reflection. The Acumen Fund is portrayed as a little too perfect, which I suppose happens when you write about the nonprofit you head. I would like to have seen more objectivity. Some of the stories made me cringe, not due to the nature of them, but they seem a bit self-serving. For example, the author shares a story of being in rural Pakistan, and hearing gun shots. Is it the Taliban? Warlords? Momentum builds. You might think she may be kidnapped (a la Daniel Pearl) but turns out it’s only the townspeople warning a local thief to give himself up. Big yawn. Nothing to do with her. Compare to Greg Mortgensen who actually was held by the Taliban for a week! In trying to spice things up, Novogratz comes out looking a bit needy for attention.

What I loved best are her vivid and accurate portrayals about the lives the people lead in developing countries, the daily struggles they face, the hardships they bear and overcome. Death is a constant, woven into their fabric of daily living, she writes and I have often thought that too. Only people who visit or live in these places truly understand the needs of the poor. Norogratz has definitely walked the walk; she has lived as a poor person, in subhuman dwellings.

I do wonder about Acumen’s not-so-successful stories, which are not mentioned and must exist: loans gone bad? I mean, Acumen gives a $600,000 loan to a local businessperson with diamond rings and expensive watches… what happens when they skip town, pad their books? I’ve seen nonprofits report how everything is successful– I mean, everything– and it raises eyebrows. Having been to developing countries, knowing their complexities, and the corruption and temptation, I am sure that Acumen has had some bad moments.

It’s an interesting story for sure, but for seasoned humanitarians, the book offers little meat. I applaud her efforts and devotion but the book would have been better written by a more objective third party. For those new to humanitarian aid, you’ll enjoy this, but also please read Three Cups of Tea and/or Mountains Beyond Mountains, both much better written, and better at both analyzing roots of poverty and solutions, and at providing a political/historic perspective. Two and a half stars.

Exciting News From Wash DC!!!

Tremendously great news from our nation’s capital. Life is about to change for a lot of people–but it’s nothing to do with a new president. Finally, a product has been approved by the US FDA for those with factor I deficiency!

It’s called RiaSTAP (Fibrinogen Concentrate (Human)), and it’s indicated for the treatment of acute bleeding episodes in patients with congenital fibrinogen deficiency (factor I deficiency), including afibrinogenemia and hypofibrinogenemia. RiaSTAP is a heat-treated, lyophilized fibrinogen powder made from pooled human plasma.

From www.businessspectator.com.au: “The approval by the FDA requires further study after marketing to confirm the product provides meaningful benefits to patients. The safety of Riastap, derived from human blood plasma, ‘appears to be acceptable,’ FDA reviewers said. The drug is already sold in parts of Europe as Haemocomplettan P.”

For more info, check out this site:

http://www.fda.gov/cber/products/riastap.htm

From the NHF website:

“Factor I deficiency is actually a collective term for several rare inherited fibrinogen deficiencies. Fibrinogen may be absent from the blood altogether (afibrinogenemia), present in only very low levels in the blood (hypofibrinogenemia), or measurable in normal quantities but defective (dysfibrinogenemia).

“The incidence of Factor I deficiency is estimated at 1 to 2 per million. It is inherited in an autosomal recessive fashion, which means it affects men and women equally.

“Fibrinogen helps platelets to glue together to form the initial ‘plug’ in response to an injury. Therefore, people with factor I deficiency, have a combined bleeding disorder because both platelets and clotting are abnormal. The severity of the disorder is directly related to the amount of fibrinogen present.

“Afibrinogenemia and hypofibrogenemia are usually diagnosed in newborns who can present with head bleeds, bleeding after circumcision and from the site of the umbilical cord. Easy bruising, nose and mouth bleeds, and soft tissue bleeds are also common. Joint bleeding is relatively uncommon. Women with afibinogenemia have an increased risk of spontaneous abortion. Persons with dysfibrinogenemia may have a disposition to thrombosis.”

It’s great to know there is now somethign commercially avaiable for patients. This is another great from CSL Behring.
Congratulations to all!

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