An oral testosterone treatment for Constitutional Delay of Growth in Puberty (CDGP) in boys

TSX-002 has been developed using TesoRx’s novel drug delivery platform, enabling the otherwise challenging oral delivery of pure, unmodified testosterone.

TSX-002 is highly differentiated from injected testosterone (used off-label) as well as oral testosterone drugs in development in that its formulation, route of administration and PK profile are ideal for use in the adolescent CDGP population.

CDGP is characterized by delayed growth and a transient state of hypogonadism. Although there are no drugs currently approved for CDGP, it is often treated off-label with monthly testosterone depot injections to induce puberty. These injections are effective but suffer from significant limitations:

  • Not approved or sized for use in adolescents
  • Large peak levels; has been an FDA safety concern in adults
  • Low trough levels preceding next injection
  • Unknown safety profile in adolescent population
  • Poor patient acceptance of injections
  • Delayed puberty and reduced growth in adolescent boys

In contrast, TSX-002 has demonstrated an ideal profile for this population in that it does not generate supraphysioligical peaks, delivers testosterone in a pattern mimicking natural physiology and is given as an oral dose.


Market

Since CDGP affects about 112,000 boys in the US, it qualifies as an orphan indication. TesoRx has a pending application for orphan designation for TSX-002, and expects to receive confirmation of this status from the FDA in Q1 2016.

Given the significant advantages and patient preference of a safe oral formulation and minimal number of patients, TesoRx is projecting a strong market for this treatment.


Development

TSX-002 has been dosed in adult men and exhibits a profile ideal for treatment of CDGP

  • Induced T levels and PK profile appropriate for pediatric population
  • No supraphysiolgical peaks in T levels
  • Convenience of daily oral dosing

Given the well-established safety and kinetic profile in human Phase 2 studies, TSX-002 is ready for testing in adolescent CDGP patients. Given its ideal oral formulation for use in this population, TSX-002 has the potential to be a meaningful treatment option for CDGP, an orphan indication with no currently approved treatments.