Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Immunotherapy

Posts tagged ‘ASH 2013 annual meeting’

Understanding the Breakthrough Therapy Designation – Part 1 of an interview with FDA New Drugs Director John Jenkins

The FDA approval earlier this week of ibrutinib (Imbruvica) for the treatment of mantle cell lymphoma (MCL), and the recent approval of GA101 / obinutuzumab (Gazyva), for previously untreated chronic lymphocytic leukemia (CLL) is good news for patients.

The forthcoming annual meeting of the American Society of Hematology (ASH) in New Orleans (Dec 7 – 10, 2013) is set to be an exciting event with the launch of new products to treat blood cancers.

Both ibrutinib in MCL and obinutuzumab were granted “breakthrough therapy” designation (BTD) from the FDA. Over the past several months I have been researching what a BTD may mean for cancer drug development.

The catchy “breakthrough” title has given companies and the FDA a noticeable bonanza of good PR, but there’s been a paucity of critical analysis by the media. I have yet to see a convincing argument that that there was a compelling need for a new approval pathway for cancer drugs, or that innovative and breakthrough cancer drugs such as imatinib (Glivec/Gleevec) and crizotinib (Xalkori) could have got to market any faster.

One of the key FDA decision makers is John K. Jenkins, MD, Director, Office of New Drugs in the Center for Drug Evaluation and Research (CDER); he’s Richard Pazdur’s boss. I had the privilege to conduct a phone interview with him over the summer.

In my first post from this interview, subscribers to Premium Content will obtain Dr Jenkins’ perspective on what constitutes a breakthrough? If you are an investor you want to try and predict what may be a “breakthrough” before it becomes one…

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ASH 2013 Myelofibrosis Preview

This year, the 2013 annual meeting of the American Society of Hematology (ASH) in New Orleans is potentially the meeting of the year with much new data emerging, not only on a multitude of products, but also many biotechs – big and small – not just Pharma.

Last week a chance event unexpectedly found me on the road for two days instead of feverishly browsing the ASH abstracts in real time for the first time in years. I’ve been attending the meeting since the mid 1990’s in various guises, so was quite sad not to be ‘present’ for moment they went live.

Nonetheless, I must pay tribute to the Twitter Bio community who merrily shared and discussed their favourite abstracts on various topics, which made finding and sorting relevant ones much easier and quicker than it might have been. In particular, Patrick Crutcher (@chasingthealpha) of Chimera Research Group did a sterling job of highlighting many key sessions on Twitter, despite being on the west coast for a 9am open time. If you’re not following him on Twitter, check him out!

While reading through all the links and notes I collated, it made sense to write a series of preview posts on different topics. This year, I will be writing five preview posts, with a different topic highlighted in each one:

  • Myelofibrosis
  • Multiple Myeloma
  • Chronic Lymphocytic Leukemia (CLL)
  • Acute Leukemias
  • Lymphomas

For today’s review of Myelofibrosis, the following companies and compounds will be covered:

Companies: Incyte, Novartis, Gilead, Sanofi, BMS, Geron, CTIC, Lilly, Celgene
Compounds: ruxolitinib, INCB039110, momelotinib, fedratinib, BMS-91143, imetelstat, pacritinib, LY2784544, pomalidomide, sonidegib

Note that not all of these agents are JAK inhibitors – others have a different target and can potentially be used in combination with a JAK inhibitor, some are pan JAK inhibitors, some selective and others dual inhibitors. This previews are really mini reviews of the abstracts, meaning it may be easier to print them out and read or contemplate over a cup of coffee.

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Ibrutinib leads the race to CLL 17p deletion market

Earlier this month, Janssen/Pharmacyclics announced they had submitted a New Drug Application (NDA) for Food & Drug Administration (FDA) approval of ibrutinib, an oral Bruton’s tyrosine kinase inhibitor (BTK) in chronic lymphocytic leukemia (CLL) for the treatment of patients with a deletion of the short arm of chromosome 17 (del17p). Here’s a link to July 10 press release.

The company have requested Priority review; approval later this year or in early 2014 is highly likely given that the agent has also been designated a Breakthrough Therapy by the FDA.

This is great news for CLL patients!

CLL is an incurable disease. It is the most common leukemia in the United States with 15,500 new diagnoses a year.

Chromosomal abnormalities are fairly common in CLL and predict both time to first treatment and overall survival i.e. how long someone will live. Sadly, those with a 17p deletion have the worst outcome and a poor prognosis.

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