Biotech Strategy Blog

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

Posts tagged ‘Allogeneic CAR-T therapy’

Readers don’t need Biotech Strategy Blog to tell them that Chimeric Antigen Receptor (CAR) T cell therapy (CAR-T), along with Checkpoint blockade, is one of the hottest areas of cancer drug development.

The last two days have seen pre #JPM15 deal activity with Kite Pharmaceuticals ($KITE) announcing a commercial collaboration with Amgen ($AMGN), which is not surprising given several of the Kite senior management team previously worked at the company.

Meanwhile, both Seattle based Juno Therapeutics ($JUNO) and Houston based Bellicum Pharmaceuticals ($BLCM) had successful IPO’s at the end of 2014. Interestingly, Bellicum are initially focusing most of their IPO funds, not on bringing their CAR-T to market, but on a novel cell therapy post stem cell transplant that aims to lower graft versus host disease (GvHD). GvHD is something we’ve been writing about regularly here!

Just this morning we’ve seen yet more CAR-T activity, with European Cardio3Biosciences (Euronext Brussels and Paris: CARD) acquiring the CAR-T technology of Oncyte (the oncology division of privately-held U.S. biotechnology company Celdara Medical).

There’s certainly a lot of activity in the CAR-T space and I expect we will hear more at next week’s JP Morgan Healthcare conference in San Francisco (#JPM15). One player in the CAR-T space who has not been gaining as much attention, and one that I think should not be dismissed, is Paris based Cellectis (Alternext: ALCLS.PA), who struck deals with both Servier and Pfizer last year. In June, BSB went to Paris and interviewed Chairman and CEO André Choulika, PhD and CSO Philippe Duchateau, PhD. At the recent American Society of Hematology (ASH) annual meeting in San Francisco, Julianne Smith, PhD (pictured below), Vice President CART Development at Cellectis, gave an in-depth interview to BSB. Dr Julianne Smith Cellectis ASH 2014 Interview Some key questions to address here are what are some of the important milestones for Cellectis in 2015 and and what makes the Cellectis CAR-T approach different from other companies in this space? Update Nov 7: This post now has two updates relating to the important news that came out after this post was published concerning the issuance by the USPTO of a gene editing patent that covers Cellectis’ intellectual property.  Subscribers can login to read more or you can purchase access by clicking on the blue icon below.

Marcel R.M. van den Brink, M.D., Ph.D.At the 2014 Society for Immunotherapy of Cancer (SITC) annual meeting at National Harbor, MD, one of the presentations that caught my attention was by Marcel van den Brink MD PhD (@DrMvandenBrink), pictured right, from Memorial Sloan-Kettering Cancer Center in New York. (picture courtesy of MSKCC with permission).

Dr van den Brink, who is Head of the Division of Hematologic Oncology and Alan.N. Houghton Chair, gave a fascinating talk entitled, “The role of the Intestinal Microbiome on GvHD” (Graft versus Host Disease).

Think of the yogurt pots and probiotic drinks we see in the supermarket with “beneficial” bacteria. We’re familiar with the idea that the make up of the millions of bacteria in our gut can make a difference to our digestion and health.

Well, it turns out it can make a difference to our immune system too. Research presented at the recent SITC meeting by Dr van den Brink showed that manipulating the bacteria in the gut could potentially help the thousands of patients around the world who receive a bone marrow transplant (BMT).  BMT is a gruelling procedure that many leukemia, myeloma and lymphoma patients have to face as part of their treatment protocol.

Sadly, about 20%* of people who receive an allogeneic bone marrow transplant from an unmatched donor die from Graft versus host disease (GvHD)

* According to 2010-2011 data from the Center for International Blood and Marrow Transport (CIBMTR), GvHD was the cause of death in 19-23% of people who received an allogeneic hematopoietic stem cell transplant.

After his informative and interesting presentation at SITC, Dr van den Brink spoke with BSB about his findings and what’s on the horizon for the treatment of GvHD.

Over the course of a half hour conversation, he covered some of the new treatment options, one of which may change the standard of care, and the rational some companies are pursuing by targeting the innate immune system.

For those interested in CAR-T cell therapy and the promise of allogeneic CAR-T cells, Dr van den Brink kindly talked about some unpublished data from MSKCC about why we have not seen GvHD in patients who receive allogeneic CAR-T cells.

GvHD is an important topic, and one that deserves more attention. I expect we will here a lot more about it at ASH this year so reading this interview will, hopefully, help you better put that data in context.

Subscribers can login below or you can sign up to read more about what Dr van den Brink had to say at SITC about GvHD.

National Harbor MarylandThe Society for Immunotherapy of Cancer (SITC) annual meeting promises to be a most interesting one, if the first day is anything to go by. It’s being held this week at National Harbor, Maryland on the banks of the Potomac River just south of Washington DC.

As the meeting started with some intensive workshops yesterday, the American Society for Hematology (ASH) annual meeting abstracts were released at 9am, giving up a choice between writing up SITC in situ or switching gears and analysing the initial hematology abstracts. In the interests of sanity, we have decided to focus on SITC for the next week, then move onto the AACR-NCI-EORTC conference, before reviewing the ASH data in detailed previews.

SITC is mostly a translational science meeting with a little bit of relevant clinical data through in here and there. It’s also not for the faint hearted, especially given the sheer intensity and pace of some of the talks – keeping up with pen and paper to hastily scribble notes is surprisingly quite hard!

It was an honour to attend as one of the few members of the media here. The excitement is palpable, with speakers reminding us of how only a few years ago, few people attended immunotherapy sessions at ASCO. SITC is rapidly becoming a major meeting with a record-breaking 1500 expected for the first time! It is the immuno-oncology meeting to attend for those interested in understanding the emerging trends, landscape and direction that research is taking us.

Yesterday SITC fielded two workshops with impressive line-ups from the immuno-oncology space that included Drs Carl June, James Allison, Tom Gajewski, Susan Topalian, Stephen Hodi and Mario Sznol, to name a few. The workshops focused on different topics:

  • A basic one on understanding the immune system
  • A more advanced one on combination strategies in immunotherapy

Rather than summarise all the talks from both sessions that ran a full day each, we’ve decided to focus on some themes, ideas and concepts that catch our attention each day. Here’s the first of our daily reviews from the SITC 2014 annual meeting. Thanks to all our subscribers whose support enabled us to attend this meeting for the first time.

To learn more about our impressions from the SITC immunotherapy workshops yesterday, you can sign in or sign up below.

Directions to Cellectis in ParisCellectis is a Paris based biotechnology company, (NYSE alternext: ALCLS.PA) with an aspiring “blue ocean” strategy that, if successful, could revolutionize cancer immunotherapy.

The potential of using engineered T-cells (known as chimeric antigen receptors) to fight cancer was highlighted by the impressive data presented at last year’s annual meeting of the American Society of Hematology (ASH 2013).

To many, the data for the U Penn/Novartis engineered T-Cell therapy (CTL019) in pediatric acute lymphoblastic leukemia (pALL) was worthy of presentation in the plenary session at the meeting.

Over the past year, investors have poured money into companies active in the field: we’ve written about the launch of Juno Therapeutics and their intellectual property (IP) dispute with Novartis. More recently Kite Pharma had a successful IPO.

Why was Biotech Strategy Blog keen to interview Cellectis Chief Scientific Officer (CSO) Philippe Duchateau, PhD and Chief Executive Officer (CEO) André Choulika, PhD (picture left and right respectively)?

ellectis CSO CEO

The answer is they have a completely new and innovative approach to CAR-T cell therapy that in the long run could be a “game changer.” Their lead product (UCART19) is an allogeneic CAR T cell for ALL and CLL. Allogeneic means the T cells that are modified come from a donor. This is in contrast to the autologous approaches that Kite, Novartis and Juno are developing where the engineered CAR-T cells come from the patient themselves.

All credit to Pfizer for seeing the potential in a company that has been on our radar for a while. They recently announced a major collaboration with Cellectis that could turn both Cellectis and Pfizer into major players in the cancer immunotherapy space.

In this fast moving R&D space there are already signs of where competition to Cellectis may come from, and it’s not Novartis, Juno or Kite.

Subscribers and those with an interest in CAR-T cell immunotherapy can login or sign-up below to read more, including excerpts of the interview at Cellectis HQ in Paris:

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At ASCO 2014, one of the posters that attracted a lot of attention was the one from Kite Pharma ($KITE) on their rapid cell expansion (RACE) technology for the production of engineered autologous T cell therapy.

Dr Renier Brentjens at 2012 Chemotherapy Foundation Symposium

As regular blog readers will know, we’ve been following the development of CD19-targeted T cells for the treatment of B cell malignancies such as CLL, ALL and aggressive NHL for some time.

Back in Autumn 2012, at the Chemotherapy Foundation Symposium (CFS) in NYC, more commonly known as the “Greenspan” meeting after the late Dr Ezra Greenspan, we heard presentations on early CAR-T cell data from Dr Renier Brentjens (pictured right) of Memorial-Sloan Kettering Cancer Center (MSKCC) and Dr Michael Kalos from the University of Pennsylvania (Penn).

At ASH 2012, we wrote about the data presented by Dr Carl June in the Ernest Beutler Prize Lecture. Dr Blazar, who jointly received the award, gave this quote from Dr Beutler, which is a reminder of why basic science is worthy of funding, and how important it is to innovation:

“The tendency to merely elaborate on what many others are doing arises, at least in part, from the almost universal misconception that our understanding of nature is profound, that most or all of the basic concepts have already been discovered, and that success in science consists of filling in the blanks with large teams of collaborators.”

What started off as pure academic research, has within a short period of time, become a hot (if not the hottest) area of immunotherapy drug development as inspired by the potential of early data, companies and investors pour money into commercializing CAR-T cell therapy.

Novartis had obtained the exclusive rights to Penn’s CTL019 CAR-T cell therapy in August 2012 at what now seems a bargain price.

Juno Therapeutics was subsequently created with $176M in Series A private equity funding at the end of 2013 to commercialize the CAR-T cell research of Memorial Sloan Kettering in New York and Seattle’s Fred Hutchinson Cancer Research Center.

Last week brought further development with an Initial Public Offering (IPO) from Kite Pharma who have a collaboration with the National Cancer Institute (NCI). Kite said they expected to raise $106M at a share price of $17, which was at the high end of the range. The shares soared 70% in initial trading, and closed at $29 on Friday.

To throw more fuel into the competitive fire, Pfizer have announced the signing of an agreement with French company, Cellectis, to collaborate on the development of their CAR-T cell technology.

In this first of a series of blog posts on gems from the ASCO poster hall, we take a look at the data presented by Kite Pharma and some of the challenges and opportunities the company faces.

Please note this post offers no investment advice and makes no recommendation on whether you should buy or sell shares in $KITE.

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