Replies to post #805 on GTC Biotherapeutics (fka GTCB)

[urche]

Atryn and Rhucin

Mblimon,
You bring up an interesting idea---I like the way you think. To repeat:



Interesting to see that GTCB's Atryn (rh anti-thrombin) and Pharming's Rhucin (rh c1-inhibitor) have both potentially an important role to play in reducing the negative effects of strokes, ischemia and sepsis. According to several studies both Thrombin and C1 apparently can do a lot of damage during these events and any other traumatic events, where the body defense system (over)reacts. Atryn and Rhucin can mitigate the cascade of overreactions triggered by the body and the damage done by the substances (proteines, peptides, toxicines?) released by the body during those moments.

But, to throw some cold water on your ideas, these systems don't work like switches that should just be turned off when they are having adverse effects. Both complement and thrombin are parts of complicated cascades involving proteins, enzymes, catalysts among other thingies I've forgotten. While it may be possible, say, to improve disseminated intravascular coagulation in sepsis by administering anti-thrombin, it is inconceivable to me that there won't be a myriad of effects both good and bad in other body systems. For instance, ATryn in the setting of burns or sepsis may help the coagulapathy, but at the same time, that same person may be having a small coronary event (where Atryn would presumably have a beneficial effect) or have a latent tumor or aneurysm (where Atryn may be expected to cause pathologic bleeding). I would expect interfering with complement or thrombin systems to have far flung effects anywhere inflammation occurs---which is to say everywhere the immune system and blood reach. In other words, the studies on these drugs need not focus excessively on toe nails and hair for adverse effects.

I am not negative on the prospects for Atryn in sepsis in burns. But, particularly in sepsis, as I've said before, these are complicated patients who have a lot of confounding illnesses and it isn't going to be easy to demonstrate pure favorable effects, IMO. Pharming is going to see the same thing. Using Rhucin for hereditary angioedema is very different than using it to shut down complement effects that occur in the cascade of healing a heart attack or stroke.

Urche

[DewDiligence]

>This apart from the obvious synergies there are by bringing all transgenics IP under one roof.<

The combination does make sense from a business standpoint, but there is the nagging matter of valuation. Would GTC would accept an equity stake in a combined Pharming-GTC that merely mirrored the relative valuations of the two companies in the stock market? I highly doubt it!

[DewDiligence]

Pharming Receives FDA Orphan Grant for Rhucin

[The grant is only $344,861, but it’s essentially free money because there are no strings attached.]

http://www.pharming.com/index.php?act=show&pg=304

>>
Leiden, The Netherlands, October 6, 2006. Biotech company Pharming Group NV (“Pharming”) (Euronext: PHARM) (PHARM.AS) announced today that it has received a grant from the US FDA’s Office of Orphan Products Development (OPD) for the clinical development of recombinant human C1 inhibitor (rhC1INH or Rhucin®) for treatment of attacks of hereditary angioedema (HAE).

The OPD grant program provides funding for clinical development of products for rare diseases with limited or no treatment options. The maximum one-year grant to be received by Pharming for clinical development of Rhucin® amounts to US$344,861. Pharming is eligible to receive additional funding for Rhucin® development from the OPD after this period if appropriate.

“Pharming is committed to developing products for unmet medical needs. We appreciate the FDA’s funding for Rhucin® clinical development in the US and the acknowledgement of Rhucin®’s promise as a treatment for HAE,” said Dr. Francis Pinto, CEO of Pharming.

Rhucin® has Orphan Drug designation for the treatment of attacks of HAE. The FDA Orphan Drug designation is reserved for promising new therapies being developed to treat diseases that affect fewer than 200,000 people in the United States. This designation provides an accelerated review process, certain tax advantages, eligibility for grants for clinical studies and a seven-year period of market exclusivity in the US upon product approval.

In the United States, the rhC1INH product is being studied in a randomized, placebo-controlled, double blind clinical trial for the treatment of patients with acute attacks of HAE. There is currently no approved therapy available in the US for the treatment of acute HAE attacks.

Background on Hereditary Angioedema

Hereditary angioedema (HAE) is a human genetic disorder caused by a shortage of C1 inhibitor activity. The disease is characterized by acute attacks of painful and in some cases fatal swelling of several soft tissues (edema), which usually last up to five days when untreated. In the Western world, approximately 1 in 30,000 individuals suffers from HAE, having an average of seven acute attacks per year.

Background on Pharming Group NV

Pharming Group NV is developing innovative protein products for the treatment of genetic disorders, specialty products for surgical indications, intermediates for various applications and food products. Pharming has two products in late stage development - Rhucin® (recombinant human C1 inhibitor) for hereditary angioedema (under review by EMEA) and human lactoferrin for use in functional foods (GRAS notification under review by US FDA). The advanced technologies of the Company include innovative platforms for the production of protein therapeutics, as well as technology and processes for the purification and formulation of these products. Additional information is available on the Pharming website, http://www.pharming.com.
<<