In Q1, the gene therapy space saw one big acquisition (Roche/ Spark (ONCE)) and several smaller deals including Biogen/Nightstar (NITE), Pfizer/Vivet and J&J/MeiraGTx (MGTX). These deals demonstrate the industry’s appetite for gene therapies with an emphasis on liver and ophthalmology as validated domains. CNS (primarily AAV9) and muscle (primarily AAVrh74) are the two other popular domains
What I find interesting in these deals is the fact they weren’t done from a position of strength (as opposed to the Novartis/Avexis deal, for example). Spark was struggling with its HemA program and did not have near term catalysts with other programs. Nightstar was trading around its IPO price with initial XLRP data that were hard to interpret at higher doses. MeiraGTx’s stock also hasn’t performed well and the company was facing an imminent fundraising. Continue reading →
This year’s ASCO marks a second year in a row of relatively uneventful meetings, with very few groundbreaking or practice-changing data. Just like last year’s meeting, there were too many “me too” drugs targeting the same validated targets while results for truly novel MOAs were mostly underwhelming or immature. This stagnation is particularly troubling in light of the huge budgets the industry is pouring into oncology drug development, which used to be a highly capital-efficient sector.
Looking at the different vertical segments, stagnation is apparent across the board with some exception with few kinase inhibitors and BCMA CARs. Continue reading →
Despite bouncing off a 2-year low, biotech is still an unpopular sector and investors are rightfully concerned about its near-term prospects. Recent drug failures, growing pricing pressure and the potential impact of biosimilars all contribute to the negative sentiment, but the main problem is the lack of growth drivers for the remainder of 2016 (and potentially 2017). Continue reading →
Although this year’s ASCO contained a limited amount of groundbreaking data, it provided some interesting take-aways and signaled important trends in oncology drug development. Below is my take on a quiet but important meeting.
Immuno-oncology – PD-1 combinations at their infancy
As in previous years, the meeting was dominated by PD-1/PD-L1 antibodies. Now that PD-1 blockers have been tested on every tumor known to mankind (see below a great figure from Merck), focus is shifting to combination regimens with PD-1 as a backbone. Combination partners range from other immune checkpoints to chemotherapy, targeted therapy and radiation. Continue reading →
Clovis (CLVS) lost 75% of its market cap last week after disclosing a disappointingly low response rate for rociletinib in T790M+ NSCLC patients. Updated response rates were 28%-34%, dramatically lower than the 54-60% response rate reported at ASCO 2015. According to the company, the dramatic difference stems from analyzing the same data set based on more stringent criteria (confirmed response rate). Continue reading →
A new lucrative indication for Sage (and potentially Marinus)
Last week, Sage (SAGE) surprised the market by announcing preliminary but impressive results for SAGE-547 in patients with postpartum depression (PPD, also known as postnatal depression). Four patients with severe PPD experienced a dramatic improvement in their depression score from an average of 26.5 to 1.8. In other words, these patients entered the study with a severe debilitating depression and became symptom-free within 2-3 days. Despite the preliminary nature of the results, they generated a clear efficacy signal that merits evaluating SAGE-547 in a randomized trial, to be started later this year. Continue reading →
As in previous years, the upcoming ASCO meeting will be dominated by immuno-oncology with a particular focus on PD-1 antibodies. The market’s primary focus is expected to be on non-small cell lung cancer (NSCLC) with data from three large randomized trials from BMS (BMY) and Roche. Beyond lung cancer, investors will look for additional indications where PD-1 agents may have clinical utility as monotherapy or in combination with other agents.
Overall, PD-1 programs continue to generate positive data across many indications but to date clinical experience has been sobering. PD-1 antibodies may lead to durable responses in some cases but the vast majority of patients derive limited benefit or don’t respond at all. This is true in most indications with the exception of melanoma where PD-1 antibodies have a dramatic impact and combination with Yervoy appears to lead to further improvement. Below is a recap of some of the data which will be presented over the weekend. Abstract numbers and links are also provided. Continue reading →
Back in 2010, ADCs were the hottest theme in oncology with the potential to revolutionize cancer treatment. The excitement was based on promising results for Adcetris (Seattle Genetics [SGEN]) and Kadcyla (Immunogen [IMGN]), both of which utilized new and improved ADC technologies. Adcteris and Kadcyla were perceived as the ultimate cancer drugs: They had strong single agent activity and provided meaningful benefit to patients with limited toxicity. All that was left to do was replicating their success with additional antibodies to cover every tumor type. Continue reading →
Below is my traditional end of the year summary and a recap of catalysts for 2015. As always, I did my best to cover the most important events, let me know if I missed anything… I would like to use this opportunity and wish the readers of this blog a happy and prosperous new year.
AML (Acute myeloid leukemia) remains one of the few blood cancers in which no progress has been made for decades. This is in contrast to other blood cancers where novel treatments have turned fatal conditions into chronic diseases with long term remissions. Prominent examples are CML, which today has a low mortality rate and multiple myeloma where median survival is approaching 8 years and counting. The recent launch of Imbruvica and Gazyva followed by the anticipated approval of ABT-199 is expected to have a similar impact on CLL as well as on certain subtypes of B cell lymphoma. Continue reading →