Roche wins landmark approval for targeted cancer drug vismodegib

Posted under basal cell carcinoma, Blog, Companies, Curis, Diagnostics, Funding, Genentech, Medical Devices, Medical Supply, Pharmaceuticals, Roche, Startups, T-DM1, Universities, Videos by John Carroll

Roche ($RHHBY) and Genentech won a landmark approval for the cancer drug vismodegib today. After gaining significant efficacy data from a Phase II study on basal cell carcinoma, the drug giant not only went straight to the regulators for a decision, they won the approval well ahead of the agency's decision deadline. And top regulators signaled that they would look kindly on other developers who take the same approach to targeted therapeutics.

"Our understanding of molecular pathways involved in cancer, such as the Hedgehog pathway, has enabled the development of targeted drugs for specific diseases," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "This approach is becoming more common and will potentially allow cancer drugs to be developed more quickly. This is important for patients who will have access to more effective therapies with potentially fewer side effects."

The treatment, which inhibits signaling in the Hedgehog pathway, was tested in one cohort suffering from locally advanced basal cell carcinoma and a separate cohort of metastatic patients. In the mid-stage study, which involved 104 patients, vismodegib demonstrated an ability to shrink tumors or heal visible lesions in 43% of the patients with locally advanced BCC and 30% of patients with metastatic BCC. The median progression-free survival rate for both groups was 9.5 months.

Vismodegib, which will be marketed as Erivedge, was developed in collaboration with Curis ($CRIS).

The approval may well help spur other developers to consider taking the same approach. But it won't be easy. Roche tried to get an approval for the impressive T-DM1 on mid-stage data, but the regulators wouldn't even consider that application. A big plus this time is the lack of any other treatments for these patients.

- here's the press release from the FDA

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4SC surges on positive midstage data for advanced liver cancer

Posted under 4SC AG, Acetylon Pharmaceuticals, ASCO, Blog, Companies, Curis, Diagnostics, Funding, Medical Devices, Medical Supply, Nexavar, Pharmaceuticals, Startups, Universities, Videos by John Carroll

Shares of Germany's 4SC ($VSC) surged 35% after investigators reported promising mid-stage data for the cancer drug resminostat, an HDAC inhibitor, in difficult-to-treat patients with advanced liver cancer.

4SC will now head to ASCO with data from a small Phase II trial indicating that their experimental treatment combined with Nexavar halted disease progression for at least 12 weeks among two thirds of 15 patients in the study, with some individuals going more than a year without further progression. Three of the 9 patients in the monotherapy arm went progression-free for at least 12 weeks.

"The now presented data of our SHELTER study validate impressively the growing applicability of the new epigenetic mechanism of action offered by our lead anti-cancer compound resminostat," says 4SC CEO Ulrich Dauer. "Tumor cell resensitization, which is mediated by resminostat through the inhibition of HDAC enzymes, is highly relevant for clinical practice, since the supplementary administration of resminostat can permit the continued and effective treatment of patients with a cancer drug to which patient response is no longer adequate."

4SC experienced a bitter setback last summer when its oral RA treatment flunked a midstage study. At the time Dauer vowed to push ahead with the program for other autoimmune disease. A number of biotechs are working on HDAC inhibitors, including Curis and Acetylon.

- here's the press release

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German contender chases Pfizer in oral RA drug pursuit

Curis gets $4M for blood cancer therapy

Posted under Blog, Companies, Curis, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Maureen Martino

The Leukemia & Lymphoma Society (LLS) has handed Lexington, MA-based Curis $4 million to support early development of two of the company's drugs for blood cancers. Curis' drug, CUDC-907, is an oral small molecule dual Pi3K and HDAC inhibitor for patients with B-cell lymphoma and multiple myeloma, the company said in a release. LLS said it would pay for half of the drug's development costs, up to $4 million, and in return will receive milestone payments from Curis if the developer commercializes the drug. "LLS's capital commitment to this program, along with their extensive knowledge of hematological malignancies, will be important resources as we seek to advance this promising first-in-class molecule," Curis CEO Dan Passeri said in a statement. Curis release | Article