TROPHOGEN INC — Department of Health and Human Services SBIR Phase I: NICHD

Description

PROJECT SUMMARY ABSTRACT It is estimated that over of all American couples suffer from infertility a topic of great importance to NICHD and cited frequently by this Institute Professional American women andamp those from all developed nations increasingly delay childbearing for several years compared to the past because of career advancement and increasing social acceptability This problem is not solely a woman s health issue but equally affects men as only of infertility is attributable solely to women while an estimated equal is attributable to the male partner who also contributes to another of combined female andamp male causes Currently recombinant r human h follicle stimulating hormone FSH is the single largest therapeutic component of the infertility market with sales considerably in excess of $ billion per year of which of that total over $ Million per year in the much higher priced and growing US market and another in the European market Several infertility consultants to the Trophogen PIs have confirmed that rhFSH therapy for infertility is much too expensive for most American women as the cost is not covered by insurance in many states and even if covered there are often prohibitive out of pocket costs In addition the results with current rhFSH treatments are far from optimal with only an average response rate of per cycle and much worse for women over especially for greatly under reported poor responders The latter include both to conventional first line in vivo FSH stimulation performed with intrauterine insemination IUI as well as to more effective but costly and invasive in vitro fertilization IVF Poor responders often produce fewer than oocytes per superovulation cycle of variable quality and may require repeated cycles up to or more to achieve a full term pregnancy if they are successful at all with devastating emotional and financial cost to infertile couples Moreover rhFSH stimulation is an important component of many rapidly growing additional markets limited only by cost elective and medical egg banking ethical preferred sexing of IVF babies andamp especially important to the PIs future disease prevention using cutting edge entire genome screening of IVF embryos for even more than currently screened chromosomic aberrations all known monogenic diseases and even proclivity to complex genetic and epigenetic diseases the latter using newest metabolomics techniques To be most effective in disease prevention all such methods also require an improved rhFSH analog to produce the largest possible number of high quality IVF embryos of which only and in the future only will be transferred to the uterus Trophogen has recently designed a rd generation neoglycosylated long acting bovine FSH analog already licensed to Zoetis formerly Pfizer Animal Health and the world s largest animal pharma which has performed extensive due diligence on the safety and all aspects of the superiority of this analog compared to all currently used wild type animal FSH products This new bovine FSH analog has recently inspired the homologous design of the hFSH analog on an hFSH template amino acids different from the bovine Using the expedited FDA b NDA regulatory track for Biosuperiors and with all these attributes potency maximal efficacy long action much lower production costs than big pharma using only inexpensive internet marketing with no need for a sales force Trophogen is confident it will still be highly profitable pricing its vastly superior new FSH analog in the US well below that of both currently branded FSH as well as coming biosimilars to quickly assume a dominant position within the infertility treatment market in the US andamp globally In the current studies the PIs propose in Phase to establish unequivocally the superiority of the lead TR molecule based on extended in vivo testing andamp stimulation of ovarian weight size andamp number of antral follicles to establish a master Chinese Hamster Ovary CHO cell bank for future good manufacturing practices GMP production providing high level expression of the lead FSH analog produce andamp purify additional larger of the final FSH analog candidate in roller bottles or bioreactors sufficient to submit an IND using methods of production and purification optimized in preliminary data research studies Phase transfer Trophogen s previously developed smaller scale GLP production methods and purification methods developed in Phase to the GMP facility of its about to be selected foreign pharma partner or alternatively to an outstanding commercial GMP facility tentatively selected with previous methods now adapted to major up scaling for large commercial disposable bioreactors plus addition of required new viral clearance steps obtain larger amounts of GMP material sufficient for an FDA agreed upon Phase combined clinical trial as well as only one more final Phase B clinical pathway for Biosuperiors perform all FDA required efficacy specificity stability metabolic pharmacokinetic pharmacodynamic and analytic studies Perform FDA IND required toxicology studies without repro tox studies with supplemental support from NCATS BrIDGs preparation andamp submission of IND Advanced development of this vastly superior FSH analog on a low cost Fast track NDA for Biosuperiors using internet marketing will make rhFSH treatment for infertility andamp many other growing markets for the first time much more effective andamp affordable for US andamp women throughout the world!It is estimated that over of all American couples suffer from infertility a topic of great importance to NICHD and cited frequently by this Institute Professional American women andamp those from all developed nations increasingly delay childbearing for several years compared to the past because of career advancement and increasing social acceptability This problem is not solely a woman s health issue but equally affects men as only of infertility is attributable solely to women while an estimated equal is attributable to the male partner who also contributes to another of combined female andamp male causes Currently recombinant r human h follicle stimulating hormone rhFSH is the single largest therapeutic component of the infertility market with sales considerably in excess of $ billion per year of which of that total or over $ Million per year in the much higher priced and growing US market Trophogen has recently designed a new long acting and more effective FSH analog for cattle and other livestock reproduction which is already licensed to Zoetis formerly Pfizer Animal Health and the world s largest animal pharma which has performed extensive due diligence confirming the safety andamp all aspects of the superiority of this analog This new bovine FSH analog has recently inspired the analogous design by the principal investigators inventors at Trophogen of a similar long acting requiring many fewer injections much more potent and effective hFSH for infertility showing similar great superiority to all current FSH products With its very low production costs for this much more potent drug and its vastly smaller overhead costs than big pharma Trophogen is confident it will still be highly profitable pricing its vastly superior new FSH analog in the US well below that of both currently branded FSH as well as coming generics called biosimilars which will quickly assume a dominant FSH market position in the US and globally The advanced development of this vastly superior FSH analog for infertility on a low cost fast track FDA pathway for superior biotech drugs using novel internet marketing will make rhFSH treatment for infertility and many other growing markets for the first time much more effective and affordable for U S and women throughout the world!