Arqule (ARQL) had a quiet 2011 with only two meaningful events, both occurring in January: Initiation of phase III in lung cancer and a public offering. With respect to clinical data, the last meaningful data set was announced in March 2010 in the form of positive phase II results in lung cancer for Arqule’s lead agent, tivantinib (ARQ197). Almost two years later, Arqule returns to investors with more good news, this time in liver cancer. Actual data will be announced only at ASCO in June, however, the limited information provided by the company already positions tivantinib as one of the most promising agents in development for liver cancer. (more…)

Earlier this week, AVEO (AVEO) announced topline results from a phase III trial for tivozanib in renal cancer. Although technically the trial met its primary endpoint and could probably lead to approval, results were far from satisfactory. (more…)

The annual meeting of the American Society of Hematology (ASH) was concluded last week and provided investors a peek into the future of blood cancer treatment. Below are 5 companies that presented promising data that could change the therapeutic landscape in the coming years. (more…)

As far as personalized medicine goes, it doesn’t get any sweeter than Pfizer’s (PFE) Xalkori.  As the industry is moving away from a “one size fits all” model to personalized medicine, this drug sets an enviable example of how to get fast approval for a lucrative niche indication by selecting the right patients. Hailed as the poster child of personalized medicine,  Xalkori is currently approved for lung cancer patients whose tumors have rare mutations in the protein ALK, which occurs in ~4% of non-small cell lung cancer cases. (more…)

Exelixis (EXEL) saw its share price cut in half last week, due to a regulatory setback. Based on what others have published and questions I received there appears to be some confusion with respect to  the implications for the company. I decided to address this issue using a questions and answers format, based on the many inquiries I received. Hope this format sheds some light on the situation.

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Developing oncology drugs is getting harder and harder. The rising regulatory hurdles, the constant flow of new agents and competition for trial participants all make getting a drug to market a formidable challenge. This is particularly true in drugs for blood cancers, a field that saw tremendous progress in the past decade and is becoming very crowded. As a result, even highly effective drugs require long and expensive studies with active regimens in the control arm and survival as an endpoint. (more…)

Last week BMS (BMY) increased its stake in BMS-936558 (formerly MDX-1106) by regaining worldwide marketing rights for the drug except in Japan, Korea and Taiwan. This was the result of a deal with Ono Pharmaceutical, who originally held ex-US rights for the drug. In return, Ono received marketing rights for Orencia, a BMS drug  for Rheumatoid arthritis which is already in the market. BMS’ decision to exchange its stake in a product with real sales in return for a candidate in mid stage clinical development might seem odd at first glance, but a quick look at BMS-936558’s data is enough to understand the deal was a brilliant move.  

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It is hard to be neutral on Exelixis’ lead drug - people either love it or hate it. The drug, cabozantinib, got investors and researchers scratching their heads after showing mind boggling activity against bone metastases. The drug led to partial or complete resolution of bone scans in a substantial portion of patients with cancer, including prostate, breast and lung cancer. To my knowledge, this extent of activity has never been observed with approved anti-cancer drugs, all of which rarely affect bone mets. (more…)

Dendreon’s (DNDN) recent meltdown already sent shockwaves throughout the entire biotech arena but one segment which could suffer the most is cancer vaccine companies. Dendreon’s Provenge, the first and only cancer vaccine approved by the FDA, made cancer immunotherapy very popular in the eyes of investors, who were looking for the next Dendreon among the tens of clinical stage cancer vaccine companies.

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Immunogen becomes a $1B company

Immunogen (IMGN) concluded the week with a market cap of ~$1B, up 200% in less than a year. This valuation is quite unusual for a company that ascribes the vast majority of its value from a 3-5% royalty stake in a single drug - Roche’s T-DM1. T-DM1, which utilizes Immunogen’s antibody-drug conjugate (ADC) technology, comprises of Herceptin conjugated to a drug payload. It is in two phase III trials and multiple phase II studies in breast cancer. If proven effective, many believe T-DM1 will eventually replace Herceptin, at least in certain treatment lines.

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This is the second part of an article I posted back in April. The final list includes only 9 candidates, as one of the candidates (Seattle Genetics’ SGN-75) was taken off the list after generating fairly disappointing results at ASCO. Enjoy.

Micromet

Micromet (MITI), who is developing antibodies for cancer, definitely has a potential game changer in its hands. The company’s lead agent, blinatumomab (Bmab), belongs to a new class of antibodies called BiTE (Bispecific T cell Engagers). These antibodies can harness the patient’s immune system to attack tumors by redirecting T cells (the most potent immune cells in the body) against cancer cells. BiTE antibodies achieve this by simultaneously binding a cancer cell on one side and an immune cell on the other. This unleashes a potent anti-tumor immune response. (more…)

More melanoma breakthroughs

This year’s meeting will probably be remembered as a historical event with regards to melanoma. Last year, it was a phase III trial for BMS’ (BMY) Yervoy (ipilimumab), which was the first in history to show a survival benefit in advanced melanoma patients (discussed in my ASCO 2010 write up). This trial led to Yervoy’s historical approval 3 months ago.

This year, investigators will present studies evaluating Yervoy as well as Plexxikon/Roche’s vemurafenib in pretreated melanoma patients. Yervoy was evaluated in combination with chemotherapy while vemurafenib was compared with chemotherapy. According to BMS’ and Roche’s press releases, both studies were successful and each drug led to a survival benefit.  The extent of this benefit is still unknown and will be revealed only at the conference. (more…)

One of the questions I am frequently asked is whether there are any good oncology drugs out there which are still available for partnering. The past years saw a surge in licensing and M&A deals, however, there are still several high quality assets out there being developed independently by small or mid cap biotechs. Below are ten companies with promising wholly-owned development stage programs, in alphabetical order.  

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2011 is shaping up as a transformational year for Synta (SNTA), who is making progress on multiple fronts with its lead agent ganetespib (formerly known as STA-9090). Ganetespib is an Hsp90 inhibitor, a protein with a well recognized role in cancer. The concept of inhibiting Hsp90 to fight cancer goes back two decades, but all attempts have been beset by failures so far. As Synta’s ganetespib appears to be the first active and safe Hsp90 inhibitor, it is poised to make a big dent in the multibillion dollar oncology market. I discussed the history of Hsp90 inhibitors and Synta’s unique positioning in a previous write up.)

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Last month, Morphosys (MOR.DE) reported its 2010 earnings, which seems like a good point of revisiting the stock. Last time I wrote about Morphosys (see article) was almost two years ago. Since then, the company has made a lot of progress but still without reaching a real value creation event. Nevertheless, Morphosys’ value proposition is now greater than ever, as it still offers a rare opportunity to invest in the fastest growing segment of the pharma industry with limited downside.

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Last week, Exelixis (EXEL) presented updated results for its lead agent cabozantinib (also known as XL184 or “cabo”). Cabo had become a closely watched agent last year after demonstrating unique and unprecedented activity in prostate cancer patients, leading to either partial or complete resolution of bone scans in 19 out of 20 patients. This effect had never been seen with any of the approved or investigational agents for prostate cancer, leading to widespread enthusiasm but also some skepticism.

Following last week’s data, it is now safe to say cabo has a real effect on bone metastases. The data set included 100 response evaluable patients, 62 of whom were evaluable for bone response. 85% of these patients experienced partial or complete resolution of their bone scans (the bone mets shrank or disappeared), 13% had stabilization and 2% (1 patient) had progression. Cabo had a profound effect on bone pain as well as markers for bone metabolism, implying the drug also has potential utility for treating bone related complications.

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In its earnings release last week, Seattle Genetics (SGEN) did not surprise anyone with the financial guidance and expected timelines for approval of its lead agent, SGN-35. However, on the business development front, the release did include an intriguing announcement that did not receive the attention it deserved. The company announced that Genentech recently advanced 3 new antibody drug conjugates (ADC) based on Seattle Genetics’ technology to phase I, this is in addition to the CD22 ADC already in clinical testing.

The announcement has several important implications for Seattle Genetics. First, the number of clinical programs in its partnered pipeline instantly jumped 50% from 6 to 9. By definition, this provides Seattle Genetics with more shots on goal and increases chances of substantial milestones and royalties down the road. More importantly, it establishes Seattle Genetics’ technology as Genentech’s preferred ADC platform, an attractive position given Genentech’s dominance in oncology and ADCs in particular. (more…)

Last week, Sanofi-Aventis (SNY) announced disappointing results from a phase III trial evaluating iniparib in breast cancer. The drug failed to improve survival and progression-free survival (PFS) in breast cancer patients and although actual data were not published, approval is unlikely even for a subset of patients. Failed phase III trials are quite common in oncology, a field with one of the highest attrition rates in the pharmaceutical industry. Nevertheless, iniparib’s failure is particularly disturbing, as the phase III was supported by compelling results from a randomized controlled phase II trial as well as strong scientific rationale. Importantly, this trial could have broader implications as it raises questions regarding the role of randomized phase II trials as a go/no go decision point for pivotal trials. 

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Earlier this week, Incyte (INCY) announced positive phase III results for its lead agent, INCB424, in myelofibrosis (MF). Although the full data set was not published, it will almost certainly lead to FDA approval, opening up a $200-$300 market in the US alone. Another similar phase III trial which will be reported in the coming months should support approval in Europe as well.   

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As expected, earlier this month at the annual American Society of Hematology (ASH) meeting, Seattle Genetics (SGEN) reported positive results that will likely lead to the company’s first ever regulatory approval for Brentuximab vedotin (SGN-35). The data will transform Seattle Genetics into a commercial stage company, with an initial market opportunity of ~$250M in the US alone. In addition, the results further validate the company’s ADC (antibody drug conjugate) technology, which has broad utility and huge commercial potential. In particular, Seattle Genetics could become a market leader in hematology by next year’s meeting, with results for two additional ADCs.

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