Glioblastoma multiforme (GBM)

An urgent, unmet medical need

Glioblastoma multiforme (GBM) is the most aggressive form of primary brain tumor and represents a critical area of unmet medical need. Despite advances, the prognosis for glioblastoma patients remains dismal, with a median survival rate of just 15-18 months post-diagnosis. This stark reality underscores the urgent need for innovative therapeutic approaches and a deeper understanding of the disease’s underlying mechanisms.

The greatest barrier to treating CNS disorders has been the inability of therapeutic molecules to bypass the blood brain barrier (BBB). That’s because the blood brain barrier actively works to prevent foreign substances from entering the central nervous system (CNS).

Most molecules can’t pass through the blood brain barrier in sufficient quantities:

0%
of small molecules
0%
of large molecules

Current treatment limitations

The standard treatment for GBM often involves a combination of surgery, radiation, and chemotherapy. However, these approaches have limitations:

  • Surgery: Complete removal is rarely possible due to the tumor’s invasive nature.
  • Radiation: Brain tissue is sensitive to radiation, leading to potential side effects.
  • Chemotherapy: The blood-brain barrier (BBB) restricts drug delivery to the tumor site, hindering the effectiveness of many chemotherapeutic agents.
  • Limited treatment options for recurrent GBM: GBM has a high rate of recurrence, and treatment options for recurrent tumors are even more limited.

Our initial indication for GBM is well positioned to help address these limitations

The limitations of current treatments highlight the urgent need for novel approaches in GBM therapy. Great clinical (and commercial) opportunities exist in these two areas:

Improved Drug Delivery: Developing strategies to bypass the BBB or utilizing targeted drug delivery systems could significantly improve treatment efficacy.

Immunotherapy: Harnessing the immune system to fight GBM holds immense promise, with ongoing research exploring immune therapies.

Multi-billion $ commercial opportunity

Several therapeutics are currently approved to treat glioblastoma representing a total market size of nearly $2 billion

Market size and current therapies (total size = $1.9 billion)

Characteristics

Temador

  • Oral and IV
  • Approved in 2005
  • Generic launch 8/23

Avastin

  • IV
  • Approved for 2nd line glioblastoma in 2009
  • Evaluation as addition to 1st line

Optune

  • Device
  • Originally approved in 2011
  • Approved for newly diagnosed and recurrent GBM

Glidel Wafer

  • Implant
  • Approved in 1996

More than just glioblastoma

The BRiTE technology can also address other $ billion markets, such as CNS, autoimmune, and cardiovascular