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Monday October 12, 2009

Embassy Suites, Chesterbrook, Pennsylvania (directions)


QR Pharma

A specialty pharmaceutical company

Presenter: Maria Maccecchini, President & CEO of QR Pharma



QR Pharma, Inc. (QR) is a specialty pharmaceutical company founded in 2008 to develop novel drugs for the treatment of Alzheimer’s disease (AD). The company has two compounds in clinical development, Posiphen® and bisnorcymserine (BNC). Both exhibit a dual mechanism of action, with disease modifying and symptomatic activity. Posiphen and BNC exhibit amyloid inhibitory activity that promises to prevent progression of the disease as well as two different symptomatic activities, so that they can be targeted towards different AD populations.


Current AD therapies provide symptomatic relief with limited levels of success; they fail to prevent the progression of the underlying disease. Today the greatest unmet need is the availability of disease-modifying drugs (DMD). Evidence in the literature suggests that targeting the accumulation of A, a hydrophobic, neurotoxic self-aggregating 40 to 42 amino acid peptide that accumulates preferentially within senile plaques (SP) in the brain, could change the course of AD. Other data from Genentech show that APP in the absence of trophic factors is shed from the surface of neuronal cells and processed into an amino terminal fragment (N-APP) that binds to DR6 receptors and induces nerve cell death. The Bredesen Lab identified another factor that is cleaved from the C-terminal end of APP (C31) and causes nerve cell degeneration and death in tissue culture cells and in transgenic mice.


QR’s lead compound, Posiphen®, inhibits amyloid precursor protein (APP) synthesis and reduces A as well as the N- and C-terminal toxic peptides derived from the precursor. Posiphen has a high brain to plasma ratio (10:1), good toxicology data in 3 animal species, an active Investigational New Drug (IND) application, sufficient clinical supplies to continue clinical development and it has completed two phase I clinical studies in more than 100 healthy volunteers in single and multiple dose escalating studies without evidence of adverse effects.


The second, earlier stage preclinical compound is bisnorcymserine (BNC). BNC also has a dual mechanism of action: it inhibits amyloid precursor protein (APP) synthesis which reduces A as well as the N- and C-terminal toxic peptides derived from the precursor and it provides symptomatic results by inhibiting selectively butyrylcholinesterase. Since BuChE is elevated in advanced AD patients, its inhibition suggests that the compound may work in advanced AD. All preclinical work for BNC, such as efficacy in tissue culture cells, normal mice and old rats as well as tox, pharmacokinetics (pK) and pharmacodynamics (pD) in dogs, rats and mice, has been completed and we are planning to file the IND before the end of the year.


In summary, due to their dual mechanism we expect the efficacy of both compounds to be better in patients than would be expected with one action only. We also expect reduction in APP levels to result in better neuroprotection than inhibition of A only, because new research has shown that the precursor is cleaved into a number of toxic peptides that all result in nerve cell death.



3 Major Issues


  1. Where best to look for funding in today's unfriendly economic investment climate?
  2. How best to differentiate our product from the competiton?
  3. What are the chief risks, and what steps should we take to reduce them?





6:30 - Cocktails & Dinner (Cash bar and special 2-entree buffet menu)

8:00 - Maria Maccecchini, President & CEO of QR Pharma will deliver the Company's "Elevator" Pitch to the Group

8:20 - A Panel will address 3 Major Issues for the Company

9:00 - Open discussion: members and guests



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Contact Info:

Maria Maccecchini, PhD
President & CEO


QR Pharma
259 N. Radnor Chester Road
Radnor, PA 19087




QR Pharma


List of Attendees

Abrams, Todd
Alaedini, Pedram
Becker, Michael (panelist)
Behrens, Carl
Bell, Caroline
Bergey, Jim (panelist)
Blaxland, Chris
Derrico, Ellen
Dytko, George
Fetterman, Jeff (panelist)
Gavin, Brenda
Glen, Jeffrey
Goldberg, Paula
Gonen, Boas
Hand, Jim
Hay, Douglas
Hesson, David
Hughes, Glenn
Kindt, Barbara
Laird, Jim
Lewis, Jim
Maccecchini, Maria (presenter)
Macphail, Bruce
Marcy, Alice
Martini, Jeff
Milby, Randy
Miller, Marjorie
Mitchell, Robin
Moore, Bill
Nash, Claude
Notvest, Ron
Owens, David
Patterson, Sara
Pischl, Mike
Rothman, Ronald
Schina, Michael

Shickel, Tracy

Skerrett, Don
Skoutelas, Peter
Temeles, Gretchen
Thompson, Tom
v/d Kam, Peter
Weber, John
Weinstein, Josh
Wells, Beth
Wierz, David
Zindulis, Joe
Zodda, Deni



Questions or Coments?

Contact: Peter van der Kam, | (610) 296-8086




Good Company! Good Drink! Good Food! Good Program! Good Fun!


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Pharmaceutical Consulting Consortium International, Inc., or (610) 296-8086

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