Tackling Glioblastoma: Five Companies Meeting Brain Cancer Head On

Glioblastoma is a complex, aggressive brain cancer that has no cure. The median length of survival following diagnosis is between 15 and 18 months, and the five-year survival rate sits at around 10%. More than 14,000 Americans receive a glioblastoma diagnosis each year.

Glioblastoma tumors are notoriously difficult to treat, due to their location and the fact that a single tumor will contain many different types of cancer cells. These tumors form microscopic branches that spread throughout the brain and that are impossible to remove via surgery. The blood-brain barrier prevents many chemotherapy drugs from reaching their target, and tumors are hard to treat using radiation or other treatments, meaning that all glioblastomas recur.

Against these odds, researchers are striving to develop new therapeutics and treatment options. There are 203 clinical trials currently recruiting patients in the US, with areas of focus including intracranial infusions of Avastin, a tumor-starving therapy normally used in conjunction with chemotherapy, CAR T cell therapies and intraoperative radiotherapy.

PRISM MarketView takes a look at five biotech companies having an impact in the treatment of glioblastoma and glioma.

Human brain with tumor – 3D illustration

Day One Biopharmaceuticals (DAWN)

Last week, Day One Biopharmaceuticals announced that the FDA had approved OJEMDA (tovorafenib) for the treatment of pediatric patients with relapsed or refractory BRAF-altered Pediatric low-grade glioma. Day One also received a rare pediatric disease priority review voucher from the FDA. Dr. Samuel Blackman, co-founder and Head of R&D at Day One, said, “As a pediatric neuro-oncologist, the approval of OJEMDA is a dream realized.” Following the news, the company’s stock jumped 12% to reach $17.07, before settling down at $14.92 at the market’s close on Friday.

Biodexa (BDRX)

In March 2024, biopharmaceutical company, Biodexa, announced its candidate for the treatment of Diffuse Midline Glioma (DMG) in pediatric patients and for Recurrent Glioblastoma (rGBM) in adults, MTX110, had shown promise in extending survival rates in early studies, including median overall survival of 26 months, compared to a historical overall survival rate of 10 months. Biodexa says the drug achieves high drug concentrations within the tumor while limiting systemic exposure. The company’s share price soared nearly 90% in intraday trading following the update, and its market cap currently stands at $5.7 million.

Candel Therapeutics (CADL)

In February, Candle Therapeutics announced it had been granted FDA Fast Track designation for CAN-3110, a first-in-class herpes simplex virus-1 (HSV-1) immunotherapy candidate for the treatment of patients with recurrent high-grade glioma (HGG) to improve overall survival. The company demonstrated that CAN-3110 was well tolerated with no dose-limiting toxicity in an ongoing Phase 1b clinical trial in October 2023. Candel expects to announce initial results from its Phase 1b study in the second half of 2024.

Telix Pharmaceuticals (TLPPF)

Australian biopharmaceutical company, Telix, announced in April that it had been granted FDA Fast Track designation for its investigational glioma imaging product, TLX101-CDx (Pixclara™[2], 18F-floretyrosine or 18F-FET). The company stated that it is in the final stages of preparing its New Drug Application (NDA) for TLX101-CDx in progressive or recurrent glioma in both adult and pediatric patients. There is currently no FDA-approved targeted positron emission tomography (PET) agent for brain cancer imaging in the US; Telix’s goal is to make this product commercially available in the US.

Helmholtz Munich

Privately-held biotechnology company, Helmholtz Munich, announced this week that the first patient had been dosedin an ongoing phase 1 clinical trial targeting malignant glioblastoma. The company’s innovative compound is dosed in conjunction with a radionuclide and seeks to eradicate residual cancer cells that have proven resistant to conventional therapies, offering potential for complete elimination. Prof. Reinhard Zeidler, who is providing scientific oversight to the clinical trial said, “The Phase 1 study builds on previous preclinical data that point to radiopharmaceuticals as a potential new method with which to circumvent previous challenges posed by glioblastoma.”

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Tackling Glioblastoma: Five Companies Meeting Brain Cancer Head On

Catie Corcoran

Biotech Editor