Pivotal study showed vismodegib helped shrink tumours or heal lesions in people with rare form of advanced skin cancer
Category: BioValleyRoche (SIX: RO, ROG; OTCQX: RHHBY) announced that a pivotal Phase II study with vismodegib showed positive results in people with advanced basal cell carcinoma (BCC) for whom surgery is considered inappropriate.
Basal Cell Carcinoma is a form of skin cancer that can cause disfiguring and debilitating effects and can ultimately be life-threatening. Vismodegib is an investigational, oral medicine designed to selectively inhibit signalling in the Hedgehog pathway, which is implicated in more than 90 percent of BCC cases.1
The trial showed vismodegib substantially shrank tumours or healed visible lesions (overall response rate) in 43 percent of patients with locally advanced BCC (laBCC) and 30 percent of patients with metastatic BCC (mBCC), as assessed by independent review, the primary endpoint. The most common drug related adverse events were muscle spasms, hair loss, altered taste sensation, weight loss, fatigue, nausea, decreased appetite and diarrhoea.
“Vismodegib is an example of our commitment to understanding and developing medicines that target the biologic cause of a particular disease,” said Hal Barron, M.D., Chief Medical Officer and Head, Global Product Development. “Our goal is to provide a medicine to people with this rare and disfiguring form of advanced skin cancer as soon as possible, and we are discussing these results with global regulatory authorities.”
Full results of the study will be presented tomorrow at the Seventh European Association of Dermato-Oncology (EADO) Congress taking place in Nantes, France (Abstract C014,14:50. CET).
In order to provide people with advanced BCC (who are appropriate candidates) access to vismodegib while Roche discusses next steps with the European Medicines Agency, the company is conducting a phase II safety study in the EU and other countries.
About the Phase II Trial (ERIVANCE BCC/SHH4476g)
ERIVANCE BCC is an international, single-arm, multi-centre, two-cohort, open-label Phase II study that enrolled 104 patients with advanced BCC, including laBCC (71) and mBCC (33). laBCC patients had lesions that were inappropriate for surgery (inoperable, or for whom surgery would result in substantial deformity) and for which radiotherapy was unsuccessful or contraindicated. mBCC was defined as BCC that had spread to other parts of the body, including the lymph nodes, lung, bones and/or internal organs. The 31 study sites were located in the US, Australia and Europe. Study participants received 150mg vismodegib orally, once daily until disease progression or intolerable toxicity.
The primary endpoint of the trial showed an overall response rate of 43 percent in the laBCC cohort, and 30 percent in mBCC, as assessed by independent review. Study investigators assessed the overall response rate for laBCC and mBCC at 60 percent and 46 percent, respectively (secondary endpoint). The median duration of progression-free survival (PFS) by independent review for both metastatic and locally advanced BCC patients was 9.5 months.
In addition, the clinical benefit rate (defined as patients who experienced response as well as those who experienced prolonged stable disease for more than 24 weeks) showed vismodegib shrank tumours or healed visible lesions, or prevented them from growing any further in 75 percent of patients, as assessed by independent review.
The most common adverse events included muscle spasms, hair loss, altered taste sensation, weight loss, fatigue, nausea, decreased appetite and diarrhoea. Serious adverse events (SAEs) were observed in 26 patients (25 percent), however of these only four (4 percent) patients had SAEs that were considered to be related to treatment with vismodegib. Fatal events were reported in seven patients (7 percent); none were considered by investigators to be related to treatment with vismodegib. In all cases, patients had other pre-existing diseases or symptoms that were related to their presumed cause of death.
About Basal Cell Carcinoma</strong