Plexxikon Announces Promising Preliminary PLX3397 Phase 1 Extension Data in Patients with Pigmented Villonodular Synovitis (PVNS)

Plexxikon Announces Promising Preliminary PLX3397 Phase 1 Extension Data in Patients with Pigmented Villonodular Synovitis (PVNS)

79 Percent of Patients Achieved Partial Response; 21 Percent Had Stable Disease with Targeted Therapy PLX3397

BERKELEY, Calif., May 14, 2014 (BUSINESS WIRE) -- Plexxikon, a member of the Daiichi Sankyo Group, announced today promising, proof-of-concept Phase 1 extension clinical data with PLX3397 in pigmented villonodular synovitis (PVNS), a type of rare, often locally aggressive, musculoskeletal neoplasm that arises from the soft tissues of joints and tendons. Interim data from this ongoing trial show that all evaluable patients treated with PLX3397 achieved either partial responses or stable disease. PLX3397 is a novel, oral small molecule that potently and selectively inhibits CSF1R, KIT and oncogenic FLT3 kinases, which play important roles in cancer. CSF1R, in particular, has been shown to be a primary driver in PVNS. These data are being released today as part of the American Society of Clinical Oncology (ASCO) 50th Annual Meeting Press Program. More detailed data will be presented at the ASCO 50th Annual Meeting, being held May 31-June 3 in Chicago.

Plexxikon receives European approval for Zelboraf

February 21, 2012

Plexxikon, a member of Daiichi Sankyo Group, has received European Commission approval for Zelboraf (vemurafenib) for the monotherapy treatment of adult patients with BRAF mutation-positive unresectable or metastatic melanoma.

Zelboraf is designed to selectively inhibit the BRAF mutation that occurs in about half of all cases of melanoma.

Zelboraf and its companion diagnostic, the cobas 4800 BRAF V600 mutation test, have been approved in the US, Switzerland, Israel, Brazil, New Zealand and Canada.

Plexxikon CEO Peter Hirth said the approval of Zelboraf by the European Commission marks a significant advancement for European patients with metastatic melanoma who historically have had very limited treatment options.

"We are very pleased that our strategy to co-develop Zelboraf along with a companion diagnostic helped accelerate the availability of this personalized medicine for these patients," Hirth added.

First and Only Personalized Treatment for Deadliest Form of Skin Cancer Approved by Health Canada


~ Health Canada approves Zelboraf, a targeted medicine designed to inhibit cancer growth in patients known to have a common type of metastatic melanoma ~

MISSISSAUGA, ON, Feb. 16, 2012 /CNW/ - Roche announced today that Zelboraf (vemurafenib) was approved in Canada as monotherapy for the treatment of BRAF V600 mutation-positive unresectable or metastatic melanoma. A validated test is required to identify BRAF V600 mutation status.1

Melanoma is the deadliest and most aggressive form of skin cancer, killing 80 to 85 per cent of stage IV patients within five years.2 Approximately half of all patients with melanoma carry a genetic mutation in the BRAF gene.3 Zelboraf is the first and only medicine shown to improve survival in people with BRAF V600 mutation-positive metastatic melanoma. It works by targeting and inhibiting the mutated BRAF protein found in about half of all cases of melanoma.

Researchers uncover how new melanoma drug accelerates secondary skin cancers

January 19, 2012

By Kim Irwin - Patients with metastatic melanoma taking the recently approved drug vemurafenib (marketed as Zelboraf) responded well to the twice-daily pill, but some of them developed a different, secondary skin cancer.

Now, researchers at UCLA's Jonsson Comprehensive Cancer Center, working with investigators from the Institute of Cancer Research in London, Roche and Plexxikon, have elucidated the mechanism by which the drug excels at fighting melanoma but also allows for the development of skin squamous-cell carcinomas.

A look back, and forward

This past year has held some pleasant surprises for me. I was particularly intrigued by the Plexxikon buyout for $935 million, the bulk of it upfront. In a sane world, Plexxikon--which developed the remarkable new skin cancer drug Zelboraf (vemurafenib)--would have gone public. But as far as the IPO market for biotech companies is concerned, this isn't a sane world.

Plexxikon helped illustrate the potential biotechs have to distinguish themselves with great science and a sharp development focus. That kind of expertise is clearly worth a considerable sum; a maxim other companies like Pharmasset have clearly learned how to profit by. Plexxikon's experience also demonstrated the limitations developers face in a world where investors will typically turn a cold shoulder to all the risks involved in drug development.

I'm wrapping this year's run of FierceBiotech reports with a look back, and forward. The key trends of 2011--with a wide array of venture capitalists complaining loudly, the R&D revolution still at the midway point, deal-making more important than ever, and regulators feeling the heat from a disgruntled industry--will all play into 2012.

Fundamental changes can't occur quickly in biotech. It took years to get where we are today. Years more are required to see if new strategies can work or new biotechs can navigate the hazards of clinical development. Now that we've passed the 10th anniversary of FierceBiotech, we can say we've reached the second generation of the publication. And we're planning to take our coverage of the industry up a notch or two in 2012. Call it FierceBiotech 2.0.

We'll be taking our traditional holiday break next week, returning the Tuesday after New Year's Day. It's a chance for us to retool some aspects of our coverage and come back stronger than ever. We hope you enjoy your own break and will see you in the new year.

- John Carroll

Plexxikon Inc. Advances Novel Targeted Treatment PLX3397 in Blood Cancer

Berkeley, CA, December 12, 2011 -- Plexxikon Inc., a member of Daiichi Sankyo Group, today announced scientific findings from preclinical studies showing that treatment with a novel oral agent, PLX3397, selectively inhibited key cancer-driving Flt3 mutations that occur in 20-30 percent of acute myeloid leukemia (AML) patients. In a preclinical model of AML, PLX3397 showed significant tumor regression. This preclinical work also showed that PLX3397 retained activity against certain drug-resistant forms of mutated Flt3 that can occur with other treatments. These scientific findings were presented during the American Society of Hematology (ASH) Conference, taking place December 10-13, 2011 in San Diego (Abstracts #764 and #3632). Plexxikon recently initiated a Phase 1/2 study in AML patients, further described at

FierceBiotech's 2011 Women in Biotech - Plexxikon President and Tesaro Co-founder Honored

November 29, 2011

Female biotech executives have been key players in many of the biggest events in the industry this year--Takeda Pharmaceutical's buyout of Nycomed, the merger of Alkermes ($ALKS) and Elan Drug Technologies and the sale of Plexxikon to Daiichi Sankyo. Should we be surprised? No, women in this industry defy the odds when they rise to key positions in the male-dominated biotech game. Of course we're seeing them accomplish big things. But they deserve recognition.

So, we're excited to bring our readers FierceBiotech's much-awaited-and belated-Women in Biotech feature. We had an overwhelming response to our call for nominations this year, with more than 130 great submissions and an amazing slate of candidates. True to our mission of providing readers the top news in biotech, many of the honorees here are women who drove some of the big stories we covered this year. We also wanted the women featured this year to represent the best of the global biotech industry, and you'll find women here who are making an impact for organizations based in Asia, Europe and here in the U.S.These women are inspiring, not just to women, but (at least speaking for the males on our team) men involved in the industry, too. Our profiles will bring you up to date with what each of these female movers in biotech are working on these days. Some are rallying scientists at young startups, gearing up for important late-stage trials or leading research of serious health concerns such as HIV. For each of the honorees, there are unique stories about how impressive women have gotten ahead in the competitive biotech field."

I think that the potential interesting little extra that you get from speaking to some of the women in biotech is we've probably been challenged with thinking a little bit more about how to cultivate our careers," said Abbie Celniker, chief executive of Eleven Biotherapeutics and one of this year's Women in Biotech. "As a result, we can be a tiny bit more self-reflective because we've had to do lots of course correction to make sure we could compete in the days when it was more predominately male."

Kathleen Sereda Glaub
President, Plexxikon

In the biotech business and at her home garden, Kathy Glaub likes to plant seeds and watch them grow. Of course, the seeds of biotech involve investments in R&D and carefully crafted financing strategies. In those regards, there's been a bumper crop this year at the drug discovery firm Plexxikon, and Glaub has been able to savor the fruits of her 10 years spent helping to shape the business strategy and firming up lucrative partnership deals.

Mary Lynne Hedley
President, CSO, Co-founder, Tesaro

Mary Lynne Hedley's latest biotech adventure has her exploring multiple paths to find and develop new therapies and supportive care drugs for cancer patients. Hedley, the president and chief scientist at Lexington, MA-based Tesaro, is now leading the company's excursion to gain FDA approval of its lead compound, rolapitant, which aims to prevent nausea and vomiting from chemotherapy and is ready for late-stage trials. At the same time, she's responsible for hunting for other drugs for cancer patients that fit the company's in-licensing strategy.

FDA Approves Plexxikon Drug

October 18, 2011

Zelboraf, the most-recent Pappas Ventures-backed product to be approved by the FDA, has attracted worldwide media interest. Click here to see the ABC News report on how the melanoma drug, pioneered by Plexxikon, is providing new hope for patients who have this deadly disease.

Plexxikon’s Glaub: personalized medicine is ‘the wave of the future’

October 7, 2011

They came from all parts of North Carolina’s Research Triangle, and several of them from far beyond, to hear the story of how a small biotechnology company developed a breakthrough cancer treatment, blazed a path for future development of personalized medicine treatments and was purchased in a deal valued at $935 million — the biggest venture-backed acquisition this year.

The company is California biotech Plexxikon. And one of its investors was Durham, North Carolina-based Pappas Ventures, which saw a return greater than 10 times its original investment. Plexxikon President Kathy Glaub was the guest speaker at a packed house for Pappas Ventures’ annual life sciences symposium. Pappas was one of Plexxikon’s early investors, pumping money into the company shortly after its 2001 launch. Art Pappas, founder and managing partner of the firm, said the investment was as much in founder and CEO Peter Hirth as it was in the science. Pappas said he thought Hirth could do with Plexxikon what he had done with previous company Sugen, whose cancer drug Sutent is now a blockbuster drug for Pfizer (NYSE:PFE).

Plexxikon, Daiichi Sankyo complete potential $935M deal

April 4, 2011

Daiichi Sankyo Co. Ltd. completed its acquisition of Plexxikon Inc., giving shareholders $805 million upfront with the possibility of near-term milestone payments of another $130 million on Plexxikon’s potential melanoma drug.

Plexxikon will retain its name, employees and Berkeley facilities, and it will continue research and development operations as an independent unit of Tokyo-based Daiichi Sankyo.

The deal was announced in late February.