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…)
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.
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…)
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.
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.
Yesterday Pfizer (PFE) announced that Sutent failed to prolong survival of lung cancer patients when given in combination with Tarceva. The drug led to an increase in progression free survival (PFS), which was the secondary endpoint of the study. The results are not published and there are several open questions such as the extent of PFS improvement, benefit across different subtypes and use of Sutent in patients from the placebo cohort after progressing on Tarceva. Nevertheless, chances to see Sutent+Tarceva as a standard of care for 2nd/3rd line NSCLC are slim.
Pfizer’s failure provided some clarity for Arqule (ARQL) and its partner Daiichi Sankyo, who plan to initiate a phase III study for ARQ-197 in combination with Tarceva. The indication Arqule is pursuing is very similar to that pursued by Pfizer, so had the Sutent trial been successful, it would have adversely affect ARQ-197’s prospects in general and potentially the required clinical route. (more…)
For part I click here
On top its JAK programs, Incyte has been developing two additional programs it intends to out-license. The first program is INCB13739 for diabetes, which already reached clinical proof of concept and could be licensed imminently. The second program, INCB7839 for breast cancer, is less advanced but could become very interesting later this year depending on data from an ongoing trial.
Last week, Incyte (INCY) sold over $130M worth of stock and $400M worth of convertible debt in an effort to solve its balance sheet issues. Thanks to the stronger cash position, the company can finally be evaluated based on its promising pipeline rather than its capital structure. More importantly, it will be able to complete a series of business development deals and focus on becoming a commercial stage company.
A drug with an almost certain approval and immediate sales potential of hundreds of millions of dollars is an asset very few biotech companies possess. In that sense, Incyte (INCY), which is developing a breakthrough drug for blood disorders, represents a unique opportunity in an industry plagued by risk and uncertainty. Incyte is also unique in its problematic capital structure, which makes an otherwise simple investment decision into a tricky one.
Morphosys (MOR.DE) is one of the most unusual biotech companies, as it breaks three basic rules that apply to drug development companies:
Rule No. 1: Development-stage companies burn cash and therefore must constantly raise capital and dilute existing shareholders.
Rule No. 2: Development-stage companies are risky and volatile because they rely on a limited number of binary events.
Rule No. 3: Investing in cutting edge, growing segments of the pharmaceutical industry is associated with a high level of risk.
Morphosys is the only company I am familiar with that systematically breaks each and every one of these rules. It does not have any drugs on the market and is not expected to have any in the foreseeable future, yet it is profitable. It is involved in drug discovery which is associated with a high attrition rate, yet statistically, there is a very high chance that it will have commercial revenues at some point in the future. It is involved in one the fastest growing segments in the industry, but can be regarded as a conservative holding since it will never be dependent on a limited number of binary events. And finally, it has no need to raise cash in the coming decade in order to support its activities, as its costs are covered by other companies.
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