Replies to post #67271 on Biotech Values

[DewDiligence]

Good article, but readers should examine #msg-30789679 for the counterpoint argument.

Also, there are a couple of factual inaccuracies in the article: i) I don’t think it’s correct to say that King has “removed” bovine factor V from Thrombin-JMI; and ii) GTCB is no longer planning to develop recombinant albumin, although the technology to make it in transgenic cows has been shown to work.

Thanks for posting the article. Regards, Dew

[Preciouslife1]

The 13th Congress of the European Hematology Association (EHA) in Copenhagen (June 12-15, 2008) will be remembered as yet another successful event in the annals of EHA congress.
The meeting attracted over 6600 delegates from 103 countries, who enjoyed a wide ranging scientific program of the highest quality.

http://www.ehaweb.org/eha/congress/previous_congresses/13th_congress_copenhagen/abstracts_2008

Dr M Greaves, from the University of Aberdeen, addressed both
the possible tensions and opportunities for collaboration at the EHA annual meeting in June 2008.
He began by quoting David
Blumenthal’s statement from the New England Journal of
Medicine: ‘when a great profession and the forces of capitalism interact, drama is the likely result’. He pointed out that research and development is declining in the industry due to a combination of patent expiration and generic competition, together with the huge costs involved in bringing new drugs to the market. It is predicted that between 2007 and 2012, $ 67 billion will be lost from the top pharmaceutical companies in the USA…
Drug costs are now a huge issue for society, especially when
linked to efficacy. It is quite amazing when a number of drugs
with similar modes of action, but different development histories, are similarly priced without any regard to their research and development costs!
Tyrosine Kinase inhibitors (TKI’s) are examples of drugs which have totally changed the outlook for patients with Chronic Myeloid Leukemia (CML). However this disease has been transformed into a chronic illness and thus patient numbers are accumulating.
It is estimated that in the USA there soon will be
250,000 patients alive with CML, representing an annual cost
somewhere in the region of € 7,5 billion for TKI’s. While the
FDA does not take cost into account, countries – for example
the UK - restrict new agents on the basis of cost-effectiveness, often resulting in a public outcry!
With other expensive drugs, efficacy is difficult to demonstrate.
The FDA approved a cholesterol-lowering agent in 2002 after a
small number of clinical trials, which included 3,900 patients!
In the absence of proven efficacy against heart attacks the drug earned billions of dollars for the company, presumably due to the robust advertising campaign. We would all agree (as mentioned in a previous newsletter) that all clinical trials should be registered and the results, whether favourable or not, be made available to the public.
Finally I am always intrigued by serendipity, especially in medicine.
Although we live in an age of ‘certainty’ and people speak
about focused research, it remains that many great medical
developments still happen by chance and hopefully will continue
to do so in the future.
Shaun Mc Cann
Editor in Chief

List of abstracts from the 13th Congress of the EHA
Poster session I
17:45-19:00 Novel therapies, drug resistance and pharmacology I
17:45-19:00 IN VITRO AND IN VIVO PROPERTIES OF PT-401, A NOVEL ERYTHROPOIETIN FUSION PROTEIN 0288 J. Sytkowski, J.Y. Jeong, K.L. Davis, A.L. Socha, H.J. Gomez (Boston, MA, United States of America)
http://www.ehaweb.org/eha/congress/previous_congresses/13th_congress_copenhagen/abstracts_2008

[DewDiligence]

Baxter Offers Relief for Ailing Investors

[Maybe there’s something in the water supply in the stretch of Illinois prairie between Chicago and the Wisconsin border because the two big healthcare companies based there—ABT and BAX—have been putting up some great numbers.]

http://online.barrons.com/article/SB123578816781099005.html

›February 28, 2009
By SANDRA WARD

Investors suffering from anemic returns might find that shares of Baxter International offer just the right therapy.

The giant health-care company, which specializes in blood-protein treatments and medical-delivery systems for hemophilia, infectious diseases and kidney failure, among other ailments, is one of the few large-cap companies that delivered double-digit earnings growth last year. Even better, it is expected to give a repeat performance this year.

Baxter (ticker: BAX) has forecast 2009 sales growth of 7%, excluding any foreign-exchange impact, and fully diluted earnings of between $3.70 and $3.78 a share, before any special items, up from $3.38 last year. Cash flow for the full year is expected to exceed $2.6 billion.

In an investment sector that is, by its nature, defensive, Baxter could be a real winner, with growth prospects that look unusually alluring at a time when more investors are seeking stocks perceived to be safe.

Compared with the erosion in the broader market, the shares have been a bastion of strength the past 52 weeks, trading until days ago at roughly the same level they did a year earlier, and representing a market value of about $32 billion. At about 52 Friday, the stock was changing hands at 14 times estimated earnings for '09 and about 12.3 times the forecast for '10.

There's a strong case for the shares to reach 65 by year's end, or nearly 20% above current levels, based on strong earnings growth, margin expansion and continuing share buybacks. Bulls note that management has a habit of providing conservative guidance, and that Baxter could well exceed its current expectations of 10% to 12% earnings growth.

Followers of the stock point out that, historically, it has been a good time to buy the stock when it trades around 13 to 15 times earnings; conversely, it's made sense to sell when the multiple reaches 18 to 20.

Baxter has managed to increase earnings even while making a deep commitment to research and development: R&D spending has roughly doubled since 2004, to an estimated $1 billion this year. Indeed, out of that program came Gammagard Liquid, a solution of intravenous immunoglobulin for the treatment of immunodeficiency disorders. Early studies have shown that Gammagard might be useful in slowing cognitive deterioration in Alzheimer's patients, a potential multibillion market.

Baxter trades at a slight premium to medical-technology companies such as Becton Dickinson and Covidien, but its diversified line of businesses, fortress-like balance sheet and solid free cash flow justify the higher price, say its fans.

Much credit for the boom that Baxter is enjoying goes to Robert Parkinson, chairman and chief executive, who came on board in 2004 as the company was reeling from a mismanaged expansionary push. Parkinson had spent much of his career at Abbott Laboratories, rising to president and chief operating officer before retiring in 2001.

Under his leadership, Baxter's biosciences business has become a powerhouse, producing $5.3 billion in sales in 2008, or 44% of the company's total sales. Baxter is the leader in providing plasma-based therapies for immune disorders, as well as recombinant proteins to treat hemophilia. Baxter also has a leading share of the intravenous, or IV, solutions market: In 2008, its medication-delivery group raked in $4.6 billion in sales, or 37% of the total. And through its renal division, a $2.3 billion business, Baxter dominates the peritoneal-dialysis market, providing portable home therapy for people with irreversible kidney disease.

But by no means is Baxter immune to the economic pressures bearing down on industries around the world. Cutbacks and tighter inventory controls at hospitals as admissions drop could hurt Baxter; however, many view its products, such as IV solutions and bags as well as anesthesia products, as nondiscretionary.

Changes in government-reimbursement programs are always a threat for health-care companies. Last week, the sector fell sharply in response to President Obama's budget plan that would cut Medicare payments to private insurers. For a company in which 60% of sales come from abroad, foreign exchange is an issue as well. Although much of Baxter's European exposure has been hedged, the strengthening dollar has dented results from Latin America. Some analysts estimate a 10-to-15-cent shortfall in 2009 earnings as a result of foreign-exchange translations.

Wall Street is also watching the merger of two Baxter competitors, betting that consolidation will remove a weaker hand and result in better pricing. Privately held Talecris Biotherapeutics has agreed to be acquired by biotherapy giant CSL in a deal valued at $3.1 billion [#msg-31416788]. The transaction awaits U.S. antitrust approval.

But these are small issues compared with the huge potential for biotherapy breakthroughs and higher returns that Baxter's shares represent. Indeed, they could be just the shot in the arm investors need.‹

[DewDiligence]

FTC to Block CSL-Talecris Deal

[This article needs clarification on several counts. First, the article switches back and forth between talking about US sales and worldwide sales of plasma proteins; the former is a $6M/yr market while the latter is $15B/yr. Second, the article switches back and forth between US dollars and Australian dollars, which are worth about 20% less at current exchange rates. Third, the article does not make it clear that protein drugs derived from plasma are only a fraction of the market for plasma proteins because the market includes such drugs as BAX’s Advate, which is manufactured recombinantly.

In the US plasma-protein market, CSL and Talecris combined would have attained roughly a 40% share, about the same as BAX’s, and the FTC evidently thinks an 80% share in the hands of two companies is an excessive concentration. Worldwide, the plasma-protein market is much more fragmented: BAX and CSL combined (even with Talecris added) have less than a 50% share.

BAX recently told investors that pricing for plasma proteins has been firm, and hence they did not see a potential pricing benefit from the elimination of the #3 player in the US market (Talecris). A corollary is that the Talecris deal’s being nixed by the FTC should not be materially negative for pricing and profitability in this business segment.]

http://online.wsj.com/article/SB124323949988451123.html

›MAY 26, 2009
By Andrew Harrison

MELBOURNE -- CSL Ltd., the world's second-largest plasma products maker, said the U.S. Federal Trade Commission is likely to block its US$3.1 billion takeover of U.S. rival Talecris Biotherapeutics Inc.

CSL Managing Director Brian McNamee met with officials Friday to argue for the transaction and present potential remedies to address antitrust concerns, the Australia-based company said.

CSL said it was informed during the meeting that FTC staff have recommended its commissioners start legal action to block the deal. The commissioners are likely to vote on the proposed acquisition by Thursday.

A CSL spokeswoman said if the deal isn't forthcoming it would reconsider returning surplus funds to investors.

The blood products company raised 1.75 billion Australian dollars (US$1.36 billion) in October, selling new stock at A$36.75 each, to help fund the Talecris transaction.

The spokeswoman said that if the FTC opposes the deal, CSL would need to challenge the decision in the courts if it were to pursue the matter further.

CSL proposed in August to buy North Carolina-based Talecris to boost its share of the US$15 billion-a-year global plasma therapeutics market. At the time, CSL said garnering the requirement approvals from U.S. regulators would likely take a year.

Talecris is the third-largest producer of plasma medicines in the U.S., behind CSL and market leader Baxter International Inc. The three companies control 83% of the U.S. market.

CSL had said that with Talecris, its annual sales of plasma products would rise to about A$3.8 billion, compared with Baxter's yearly revenue of about A$5 billion. Talecris is highly complementary to CSL's business and would give it economic scale, broaden its range of plasma products and boost its geographic presence.

CSL said in August that the purchase would add 10% to earnings per share in its first year of acquisition and deliver synergies worth about US$220 million over three years.

Dan Hurren, a health-care analyst at UBS in Sydney, said the deal is less likely as "the commission is unlikely to recommend against its legal advice."

If CSL fails to secure approval to complete the deal, the company will be liable to pay a US$75 million break fee to Talecris's private equity owners, Cerberus Partners and Tribeca Investment Partners [ouch], the company spokeswoman said.

CSL shares fell 1.8% to A$30.35 Monday in Sydney, partly recovering from an early low of A$29.94. The benchmark index dropped 0.6%.‹

[DewDiligence]

Talecris Seeks to IPO—Again

[Talecris was formed by VC’s in 2005 to buy Bayer’s plasma-protein business (#msg-5925270); it has been owned ever since by these VC’s, who were unsuccessful attempting to IPO in 2007 (#msg-21625850). In 2008, Talecris agreed to be bought by CSL Behring, but the FTC rejected the deal as anticompetitive (#msg-38506825). Now its back to Plan ‘A.’

Aside from CSL and Talecris, the main player in plasma proteins is BAX; all three companies have seen their plasma-protein business hold up remarkably well in the face of competition from recombinant alternatives (#msg-32783294).]

http://www.reuters.com/article/marketsNews/idINN2816419020090728

›Tue Jul 28, 2009 2:20pm EDT

NEW YORK, July 28 (Reuters) - Talecris Biotherapeutics Holdings Corp, whose acquisition by CSL Ltd was blocked by regulators, may be moving toward an initial public offering, according to recent filings with the U.S. Securities and Exchange Commission.

Last month, the biotechnology company and Australian blood products group CSL (CSL.AX) terminated their $3.1 billion merger agreement under pressure from U.S. antitrust regulators.

Since then, Talecris has filed an updated registration statements with the SEC, laying the groundwork for a potential IPO. The company had previously aimed to launch an IPO, but dropped the plan last year when it agreed to the deal with CSL.

Talecris aims to trade its stock on Nasdaq under the symbol "TLCR," according to the SEC filings. It plans to use the proceeds to repay debt, pay dividends on convertible preferred stock, and fund working capital, capital expenditures and other corporate purposes, according to the filings, the latest of which was on July 21.

The SEC filings did not include an expected price range for an offering.

Talecris, which makes plasma-derived protein therapies for people suffering from chronic and acute conditions, could not be reached immediately for comment.

In 2008, Talecris generated net income of $65.8 million on revenues of $1.4 billion. In the first three months of 2009, it earned $33.4 million on revenues of $371.8 million, according the SEC filings.

The North Carolina company employs more than 4,500 workers worldwide.‹

[DewDiligence]

Talecris IPO/SPO Expected This Week

[Insiders want to cash out under the cover of an IPO and, consequently, this IPO includes a large concurrent secondary offering in which insiders are selling their own shares (wallstarb please take note :- )).

Please see the prologue of #msg-39979601 for background on the company and its competitors.]

http://finance.yahoo.com/news/Talecris-Biotherapeutics-apf-2826935900.html?x=0&.v=2

›Tuesday September 29, 2009, 2:47 pm EDT

NEW YORK (AP) -- Talecris Biotherapeutics Inc. is expected to go public this week after a two-year pause, potentially giving the two private equity firms behind the biotechnology company a payday while allowing Talecris to pay debt.

The Research Triangle Park, N.C.-based maker of protein therapies is offering 28.9 million shares for $18 to $20 apiece, while stockholders are offering 15.8 million shares, according to a Securities and Exchange Commission filing. The company said it would use about $515 million in proceeds to pay loans, and shareholders Cerberus Capital Management and Ampersand Ventures will raise $300 million by selling their own stock.

The offering is expected to be the largest this week, as a growing number of companies are once again turning to the equity market for capital now that the financial crisis seems to have abated. Talecris, which originally planned to go public in 2007, pulled the IPO in December that year due to uncertainties regarding future plasma supplies.

Morningstar analyst Damien Conover said the "window" for biotech IPOs seems to be opening again as the market improves and companies search for capital. The downturn in the market was especially hard on biotech IPOs which are already considered high-risk stocks.

Talecris, though, is already an established biotech operation, with about 4,800 employees and $1.4 billion in revenue last year. Founded in 2005, Talecris makes a range of plasma-derived protein therapies, including intravenous treatments for immune system conditions and lung conditions. In its SEC filing, the company said it ranks third in the world, with about 12 percent of the market, behind leader Baxter International Inc., Deerfield, Ill., and CSL Behring of King of Prussia, Pa.

In June, CSL Behring parent CSL Ltd. of Australia pulled out of its plan to buy Talecris for $3.1 billion, less debt, following a challenge by the Federal Trade Commission.

A key revenue driver for Talecris is Gamunex IGIV, which is used to boost the antibodies of patients with compromised immune systems because of issues ranging from genetic conditions to chemotherapy treatment side effects. Gamunex had about 21 percent of the U.S. market in 2008.

The market itself for plasma-derived protein therapies was worth about $9.7 billion in 2007 and has been growing at a compound annual rate of about 8 percent, the company said. For Talecris, expansion into international markets is a key strategy. Last year, 80 percent of its sales came from North America, while the region represented only about 40 percent of the global market for plasma product sales.

Baxter, CSL Behring, and Talecris also compete in the A1PI market with products aimed at boosting a protein that protects the lungs from inflammation. [A1PI is another name for alpha-1 antitrypsin.] Talecris said its A1PI Prolastin product had a 67 percent share of sales in the U.S. in 2008 and 90 percent in the European Union.

Lawrence D. Stern, 52, has been the CEO of Talecris since 2007 and a director since 2005. He is also chairman. Chief Financial Officer John M. Hanson, 57, has held the position since 2005.

Risks the company faces include shifts in government and insurance company reimbursement policies, as Talecris' products are primarily used in hospital or physician settings. If payors reduce reimbursement, that could prompt customers to stop administering the product, cut the doses, or to substitute lower cost products, the company said.

Underwriters for the IPO are Morgan Stanley, Goldman Sachs, Citi, and JPMorgan Securities.

The company will be listed on the Nasdaq Global Market under the symbol "TLCR."‹

[DewDiligence]

New Flu Victim: The Blood Supply

http://online.wsj.com/article/SB20001424052748703808904574525570410593800.html

›NOVEMBER 10, 200
By LAURA LANDRO

The swine-flu pandemic is creating unexpected problems for the nation's blood supply—shrinking the number of blood donors and raising questions about whether the flu can be spread through a transfusion.

A number of blood centers are reporting an unusual drop in collections because too many potential donors are sick with the H1N1 virus, or swine flu. Some blood drives in high schools and corporate offices have had to be scaled back or canceled because of high levels of absenteeism.

Another problem: Some centers say a growing number of donors are calling a day or two after giving blood to say they've come down with flu-like symptoms, forcing the centers to dispose of the blood as part of government regulations. Researchers in a government-funded study are testing samples of these donors' blood for viremia, the medical term for virus in the blood.

Unlike infectious diseases such as HIV/AIDS, hepatitis C and West Nile virus, colds and flu aren't believed to be transmitted by blood. But some studies indicate that more virulent strains can be present in the blood before flu symptoms show up. "When you get into strains that are pandemic, the rules don't necessarily apply," says Matthew Kuehnert, director of the Office of Blood, Organ and other Tissue Safety at the Centers for Disease Control and Prevention.

So far, medical officials say there is enough blood to comfortably supply the 15 million units used annually in trauma units, operating rooms and elsewhere. But blood centers have begun working with the federal government and hospitals to put emergency planning measures in place, such as allocating blood only to the sickest patients, should the pandemic worsen or return in a more virulent form next spring.

"We are very worried about the impact of swine flu," says Marsha Springel, donor resources manager at the Miller-Keystone Blood Center in Bethlehem, Pa., which serves eight counties in Pennsylvania and New Jersey. "Right now we have an ample supply, but if this hits as big as they are predicting, we will lose the schools, and they are a large proportion of our donor base," she says.

At one high-school drive last week, high absenteeism meant Miller-Keystone collected just 33 units of blood, instead of the 70 to 80 units they had expected. And over Halloween weekend, a local ski club had to cancel a drive because so many members were ill.

As of last week, 48 states reported widespread influenza activity, with almost all the illnesses identified so far as swine flu, according to the CDC. While visits to doctors for flu-like illnesses decreased slightly last week after four consecutive weeks of sharp increases, they are at much higher levels than what is expected at this time of year. Parts of the country are still seeing sharp increases in activity and it is possible that illness rates could rise again.

At donor centers, blood is separated into red blood cells, platelets and plasma, then undergoes 14 tests, including 11 for infectious agents such as HIV to ensure its safety. Blood is then labeled by type, stored at appropriate temperatures, and distributed to hospitals as needed. Centers are concerned that the supply they keep on hand—typically three to five days' worth of blood—could quickly become stretched if more donors fall ill.

Declining Turnout

America's Blood Centers, an association of independent facilities that combined collect about half the U.S. blood supply, says that 27% of its member centers are reporting a decrease in overall collections due to swine flu. Nearly a quarter of the centers reported that collections have dropped at high schools, which have been one of the fastest-growing sources of blood donations in recent years as more states allow students as young as 16 to donate. And more than 15% of centers reported declines in blood collections from colleges and from corporations, where layoffs and plant closings have hurt blood drives. Association members reported that the declines are manageable.

At Lane Memorial Blood Bank in Eugene, Ore., which supplies three hospitals in the area, Executive Director Doug Engel says absenteeism rates of up to 35% at local high schools have put a dent in collections. The blood bank is now calling schools and companies that sponsor drives a few days ahead to check on absentee rates, and may send a mobile van instead of setting up drives in high-school gymnasiums to save costs.

Partly offsetting the reduced collections is lower blood demand as more people put off costly elective surgeries due to the tough economy, says Richard Benjamin, medical director of the Red Cross, which supplies about 43% of the nation's blood. But a looming worry is the upcoming holiday season, when it is traditionally harder to recruit donors. "If swine flu continues on its path, disrupting blood collections during that period, we are really going to need more donors," Dr. Benjamin says. The Red Cross and its local chapters are turning to social-networking sites such as Facebook and Twitter to get the word out about the need for blood.

Medical experts generally don't believe colds and flu can be transmitted by blood, and no case of seasonal influenza transmitted by blood has ever been reported in the U.S. But there also has been little active surveillance for flu in the blood supply.

The National Institutes of Health since the late 1980s has funded studies of blood safety through a program known as the Retrovirus Epidemiology Donor Study, or Reds, to determine the prevalence of HIV among blood donors and the risks of transmitting HIV and other viruses via transfusions. As part of a second phase of that study, researchers in 2006 began examining whether influenza virus could be detected in blood. Philip Norris, associate director of the Blood Systems Research Institute in San Francisco, and a lead investigator on the project, says testing of hundreds of blood donors at risk for seasonal influenza hasn't revealed any evidence of influenza virus in the blood. He says his group is repeating the study with new blood samples "in case our assumption that H1N1 will behave like seasonal influenza is wrong."

Dumping Blood

Food and Drug Administration regulations require blood centers to turn away would-be donors who have any symptoms of illness. And any donor who falls ill shortly after giving blood is asked to notify the center where they donated so their blood can be removed from inventory. So far, the swine flu has turned out to be less infectious than medical experts at first feared. Still, because many patients who get blood transfusions have weak immune systems, a flu infection transmitted through the blood could be extremely dangerous, increasing their risk of death.

The FDA says the benefits of a transfusion where medically necessary far outweigh the risks, including any "theoretical" risk of swine-flu transmission through blood or blood products. But an FDA spokeswoman says the agency will continue to review any new scientific information about the potential risks and may revise its recommendations "as appropriate."

When a new threat is discovered, the FDA has shown it can act quickly. In 2003, a test for West Nile virus was developed and approved by the FDA that identified nearly 1,000 blood donors with the infection before their donations entered the blood supply.

"Assuming the swine flu virus doesn't begin to look more virulent, we hope we can look back at H1N1 in 2009 as an important planning exercise for the future to see where our vulnerabilities lie," says Louis Katz, executive vice president, medical affairs, of the Mississippi Valley Regional Blood Center in Davenport, Iowa, and chair of a national task force on pandemic flu and the blood supply.

Meanwhile, along with screening out obviously ill patients, blood centers are taking extra hand hygiene and infection-prevention precautions to "create as safe an environment for blood donors as possible," Dr. Katz says. "The most important message is that people still need to get out there and donate blood.‹

[DewDiligence]

FDA Approves Octapharma’s Wilate for von Willebrand Disease

[Octapharma is one of the world’s five-largest plasma-protein companies and the only of the five that is private.]

http://finance.yahoo.com/news/Octapharma-USA-Announces-FDA-prnews-629408821.html?x=0&.v=1

›Milestone Marks Swiss Company's Entrance into the U.S. Blood Coagulation Market

12:00 pm EST, Monday December 7, 2009

HOBOKEN, N.J., Dec. 7 /PRNewswire/ -- Octapharma USA today announced the U.S. Food and Drug Administration has approved wilate® for the treatment of spontaneous and trauma-induced bleeding episodes in patients with all types of von Willebrand disease (VWD). Wilate® is a newly developed, high-purity, double virus inactivated von Willebrand Factor/Coagulation Factor VIII Concentrate (Human) that demonstrated efficacy for all types of VWD, including pediatric patients, in four prospective clinical trials utilizing both objective and subjective criteria.

Wilate® is indicated for the treatment of spontaneous and trauma-induced bleeding episodes in patients with severe VWD as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated. Wilate® is the first double virus inactivated VWF/FVIII (von Willebrand Factor / Factor FVIII), high-purity concentrate, utilizing the solvent/detergent (S/D) process and a special terminal dry-heating (TDH) system. The selected purification processes isolates the VWF/FVIII complex under highly protein-protecting conditions, resulting in a 1:1 ratio of VWF:RCo (ristocetin cofactor) and FVIII activities that is similar to normal plasma. No albumin is added as a stabilizer. Wilate® is exclusively derived from large pools of human plasma collected in U.S. FDA approved plasma donation centers.

The FDA approval of wilate® marks the entrance of Octapharma USA into the U.S. blood coagulation market, with product availability scheduled for early 2010. Octapharma USA is the rapidly growing U.S. division of Octapharma AG, one of the largest plasma products manufacturers in the world.

VWD is the most common bleeding disorder, which is found in approximately 1% to 2% of the U.S. population, according to the Centers for Disease Control and Prevention. The illness is a result of the body's inability to make von Willebrand Factor, the human protein that helps clot blood.

"The FDA approval of wilate® makes this the first and only replacement therapy developed and manufactured specifically for VWD. Its unique viral attenuation steps and it's close to 1:1 ratio of FVIII and VWF will provide a next generation treatment option for patients with von Willebrand disease," said Craig Kessler, M.D., Georgetown University Hospital, Professor of Medicine and Pathology and Director of the Division of Coagulation.

Four prospective clinical trials have demonstrated the safety, tolerability and hemostatic efficacy of wilate® in the treatment of acute bleeding episodes and prophylaxis in patients with various types of VWD. Using objective criteria, wilate® was observed in 1,068 bleeding episodes and determined to be successful between 84% and 93% of the time with results varying dependent on patient type.

"Octapharma's worldwide commitment to coagulation disorders dates back to Octapharma Group's formation 25 years ago," said Octapharma USA President Flemming Nielsen. "We are thrilled that U.S. patients will now have access to wilate® following its significant success in Europe as a next generation therapy. Octapharma is committed to providing the U.S. market with life-enhancing therapies."

Since the mid-1980s, the requirements for the viral safety of plasma preparations have constantly been made increasingly stringent, requiring demonstrated virus elimination/inactivation. Several viral inactivation steps have enhanced the safety of coagulation products, but S/D inactivation is the current gold standard for safety from highly infectious enveloped viruses. Octapharma was the first manufacturer to apply the S/D inactivation to a large-scale production of plasma derivatives. The wilate® manufacturing process provides two independent and effective virus inactivation procedures, namely S/D treatment in bulk and TDH treatment of the lyophilized product in final container. In addition, the ion-exchange chromatography step utilized during wilate® manufacturing contributes to the viral safety.

Plasma contains VWF and FVIII at very low concentration. The wilate® manufacturing process is designed to enrich the proportion of VWF/FVIII complex. Accompanying plasma proteins that may give rise to clinical side-effects, as well as proteases that could impair the stability of coagulation factors and degrade their natural structure and functionality, are efficiently removed during production.

Octapharma AG

Headquartered in Lachen, Switzerland, Octapharma is one of the largest plasma products manufacturers in the world and has been committed to patient care and medical innovation for over 25 years. Octapharma's core business is the development, production and sale of high quality human protein therapies from both human plasma and human cell lines, including immune globulin intravenous (IGIV). In the U.S., Octapharma's IGIV product, octagam® (immune globulin intravenous [human] 5%), is used to treat disorders of the immune system, and Octapharma's Albumin (Human) is indicated for the restoration and maintenance of circulating blood volume. Octapharma employs over 3,000 people and has biopharmaceutical experience in 80 countries worldwide, including the United States, where Octapharma USA is headquartered in Hoboken, N.J. Octapharma operates two state-of-the-art production sites licensed by the U.S. Food and Drug Administration, providing the highest level of production flexibility and minimizing product shortages. For more information, please visit www.octapharma.com.‹

[DewDiligence]

After a 1-year delay (#msg-50961841), the Grifols-TLCR merger closes today, creating one of the world’s largest plasma-protein companies:

http://finance.yahoo.com/news/Grifols-and-Talecris-prnews-3003366758.html?x=0&.v=1

TLCR was a 2005 spinoff from Bayer. It tried to be acquired by CSL (the world’s largest plasma-protein company), but this was blocked by the FTC (#msg-38107926). Instead, TLCR IPO’d in 2009, but it was always crystal clear that the goal was to be acquired.

[DewDiligence]

Plasma-protein companies are unhappy about having to pay the ObamaCare pharmaceutical fee:

http://finance.yahoo.com/news/IRS-Proposed-Regulations-on-prnews-4238685858.html?x=0&.v=1

The fee is waived for products with an FDA Orphan Drug designation, but most plasma-protein products don’t qualify for such a designation.

[DewDiligence]

BAX starts phase-3 trial of recombinant factor for von Willebrand disease:

http://finance.yahoo.com/news/Baxter-Initiates-Phase-III-bw-32737753.html?x=0&.v=1

Plasma-based products are already approved for VWD (e.g. #msg-44288894), but BAX 111 is the only recombinant factor from any company in clinical development for VWD.