Shire HGT’s #RareDisease Developments

Posted Saturday March 23, 2013 by Melissa

For those of us intimately involved in the future of Shire and its division Shire HGT, either because we or our kids already use a Shire product (us), because we’re in a Shire clinical trial (also us), or because we are hoping that Shire will develop a product to treat a disease we or our children have, I decided to compile Shire’s rare disease division events from the last year that might be of interest to our community.

Photo courtesy of Shire HGT

Photo courtesy of Shire HGT

The events speak of both the importance of the HGT division to Shire, I believe, but also to what I perceive as some reorganization as to its direction. I request your thoughts below.

New Lead in MPS Franchise: In July 2012, Shire named Soren Tulstrup to lead the company’s global MPS Franchise (see press release). He reports to the president of Shire HGT, Sylvie Grégoire at the time, and was set to be based out of the company’s Nyon, Switzerland office.

New Lead in Angioedema Franchise: In September 2012, Shire named Frédéric Chereau to lead their Hereditary Angioedema franchise. (see press release). He reports to the President of Shire HGT, Sylvie Grégoire at the time, and was set to be based out of the company’s Lexington, MA office.

TIGEM: In October 2012, Shire entered into a “a long-term, broad based, multi-indication research collaboration in rare diseases with Fondazione Telethon, a major Italian biomedical charitable foundation, for research carried out at the Telethon Institute of Genetics and Medicine (TIGEM). This alliance will facilitate research on 13 undisclosed rare disease indications and has the potential to add multiple, novel therapeutic candidates into Shire’s early stage pipeline.” (see press release).

Boston Children’s Hospital: In November 2012, Shire entered into a ” three-year, broad research collaboration in rare diseases.  The goal of the collaboration is to develop novel therapies to treat a number of rare pediatric diseases with high unmet medical need, thereby leveraging Boston Children’s research expertise and Shire’s development and commercialization capabilities.” (see press release). The hospital boasts The Manton Center for Orphan Disease Research which seems to offer some specialty in gene therapy and stem cell research.

ArmaGen Technologies: Of utmost interest to those of us participating in this clinical trial is that also in November 2012, Shire participated, along with Boehringer Ingelheim Venture Fund (lead), Takeda Ventures and Mitsui & Co., in a $17M backing of ArmaGen and its brain-penetrating technologies (see press release). Someone from BI and Shire also will also join the board. ArmaGen has a product, AGT-182, that is specifically for the brain treatment in Hunter Syndrome. It is a  genetically engineered IgG fusion protein along with a HIRMAb molecular Trojan horse that crosses the human blood-brain barrier via a specific receptor-mediated transport system. More information about the specific research was recently published. ArmaGen also has a pipeline product for MPS I, Hurler Syndrome.

Total BD Transactions: Shire reported in its FYE 2012 materials that it completed 15 total business development transactions in its HGT business. So there were a number of other transactions in addition to those above that I don’t discuss.

Shire HGT President to Step Down: Also in that FYE 2012 earnings call (registration required for transcript), Shire reported that Sylvie Gregoire, president of Shire HGT, was going to step down effective end of March 2013. Flemming Ørnskov, who would just be taking over the role of CEO of Shire globally as of May 1, 2013, having apprenticed with Angus Russell, the current CEO, would also serve as the interim President of Shire HGT.



First, I find the ArmaGen investment incredibly interesting given that from our MPS II perspective, we see the ArmaGen AGT-182 product as directly competitive to the Shire Idursulfase-IT product in development. With the board seat given to Shire’s Arthur Tzianabos, a former Harvard medical school professor and current VP and Head of Shire’s Research and Early Development, I would assume a type of chinese wall (don’t ask, don’t tell) would surround his acquisition of any competitive data and I would hope that such a relationship would only serve to bring additional insight into the market rather than hinder or delay ArmaGen’s development of AGT-182. Is there a potential acquisition strategy in play? I don’t know. It might be a case of keep your friends close and your enemies/competitors closer. I wish I could be at the Partnerships in Drug Delivery conference (day two agenda) in October 2013 to hear Dr. Tzianabos potentially discuss the issue and ask questions.

Second, I also find the TIGEM collaboration quite interesting. I have followed TIGEM’s research for several years and while they have published interesting findings regarding MPS II specifically, they historically have had little ability to translate this research forward into the clinical setting. I specifically exchanged correspondence with them regarding the high-dose Elaprase research that our community was very interested in given the finding of some parents (myself included) of a perceived cognitive bump upon initiation of Elaprase therapy. They have performed research on several of the MPS conditions (specifically MPS II, IIIA, IIIC, and VI). Some specific TIGEM research on MPS II includes:

I hope that this collaboration allows much of this research to move forward. I am interested to know more specifics regarding the relationship, specifically, what are the parameters surrounding disclosure and publishing of the research? Is this research that is solely available to Shire, embargoed for a specific period, or freely publishable? Is there a chance the research may never see the light of day if it shows results unfavorable to Shire’s current products or competitive thereto? These are important questions regarding any academic and for-profit pharma collaboration.

Third, I hope to see a relatively quick appointment of a new President of Shire HGT. While I wish the position were located out of the Lexington, MA location, given the HGT franchise’s locus there, I would expect that the candidate would continue to be based out of the Nyon, Switzerland offices.

Overall, I am encouraged by Shire’s actions that clearly show a long-term dedication to the rare disease space as well as a diversification among the MPS conditions and other genetic and non-genetic conditions. Adding the ArmaGen investment and TIGEM collaboration to current Shire products/pipeline, Shire would then have a potential interest in MPS I, II, IIIA, IIIB, IIIC and VI. I hope that these collaborations move the research forward in a timely manner as opposed to stifling any research that might be competitive to Shire’s current product or pipelines.

What are your thoughts about these developments in the Shire HGT franchise?

Update 3/25/2013: Some additional discussion of the TIGEM relationship can be found at numeraled page 15 (right-hand side) of this program for the Telethon XVII Scientific Convention held in mid-March 2013:

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One Response to “Shire HGT’s #RareDisease Developments”

  1. Hi Melissa … I think you might be pleasantly surprised to learn about the new president and where his home office will be.

    We are guardedly optimistic about all of the external activity being additive to HGT’s work, but I worry that in the short term this will be a distraction as they digest new cultures, technologies, and opportunities. We’d love to see HGT more responsive in a number of non-scientific areas.

    MLD is involved with TIGET, which I believe has some science relationship to TIGEM. TIGET’s MLD phase I/II gene therapy clinical trial has reached the 2 year point and the patient reported results are looking quite good. The PI’s are planning to publish interim data on the first three patients – we understand that data is looking very good as well. However, GlaxoSmithKline has the first in line clinical option on the core technology if they choose to exercise it.