Biotech Strategy Blog

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

Posts tagged ‘CAR-T cell therapy market’

What’s driving the GvHD Landscape? #BMTTandem16

Aloha! If you have plans to be in Honolulu next week for #BMTTandem16, then one of the “must do” sights is the walk to the top of Diamond Head. It towers over Waikiki and is a major landmark for anyone travelling in an easterly direction.

Waikiki from Diamond Head

One way to get there is to start early and take the #23 The Bus ($2.50 fare, exact change) from Ala Moana & Waikiki to the state park entrance. The path starts off deceptively easy, kicks in with some uneven surfaces and hits you with a pile of steps near the top. Sensible shoes, water and sunscreen recommended.

The spectacular views at the end are well worth it, especially if you catch the sunrise/early morning light and manage to avoid arriving at the summit at the same time as busloads of Japanese tourists with the same idea. It’s a popular attraction!

One of the reasons for going to the top of hills and mountains is to see the “big picture” of the landscape below. In writing and reporting on the latest innovations in biomedicine, we often focus on the results of one trial or news from one company; yet it’s also important to see how the pieces of the jigsaw fit together.

That’s why pharmaceutical marketers and new produce development specialists often commission custom reports that review the strategic landscape in a therapeutic area.

As part of our coverage of #BMTTandem16, we’re taking a look at some of the strategic trends and drivers in the Graft versus Host Disease (GvHD) space.

It’s an area of unmet medical need and market opportunity.

Anyone with an interest in allogeneic “off the shelf” CAR-T cell therapy should already appreciate the significance of GvHD as a potential complication. If you don’t, then you’ve not been reading BSB…

This post is not intended to be a substitute for a landscape report, but offers some top line thoughts on some of the strategic drivers to look out for.

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Do Cellectis CAR T Cells Really Work?

That’s the $64K question we all want to know, and what’s more is gene editing necessary when it comes to creating an “off-the-shelf” T cell therapy, which instead of modifying a patient’s own T cells (autologous), uses cells from a healthy donor (allogeneic)?

We were really curious too, and sought out one of the world’s leading experts for their opinion on this very issue.

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Will CAR-T cells make an impact in solid tumours?

As we’re coming to the end of our European Association Urology (EAU) coverage for 2015, I wanted to discuss at a rather more quirky, off-the-wall topic and look at one of the gems from the poster halls at this conference.

This year, it’s the turn of urothelial bladder cancer (UBC), a topic that doesn’t usually get much coverage or respect when it comes to new product development. Part of the challenge is the need for new targets to aim at because the particular patient population doesn’t tolerate high dose chemotherapy very well.

At ASCO last year, perhaps the surprise (and most stunning) data of the meeting was the anti-PDL1 checkpoint data (Genentech’s MPDL3280A) in refractory UBC, a disease where there are a lot of elderly and frail patients who are challenging to treat in many ways. This certainly put more attention on the disease and raised awareness to the potential opportunities for new, targeted and altogether more benign approaches to treatment. Subsequently at ESMO last fall, we also saw early data for an anti-PD1 antibody (Merck’s pembrolizumab) in advanced urothelial cancer.

Checkpoint blockade is not the only potential way to treat UBC though, so what other novel therapeutics are in development in this space?

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CAR-T and Gene Therapy excite BMT Tandem Meeting

It remains exciting times in cancer immunotherapy with breakthrough new cell therapies and checkpoint inhibitors that enhance the effectiveness of T cells.

Last Friday, Paris based Cellectis filed their IPO registration statement with the Securities and Exchange Commission (Link to F-1).

They plan to raise $115M through an offering of American Depository Shares. You can read more about their allogeneic Chimeric Antigen Receptor (CAR) T cell approach in the two interviews we did with senior management last year.

As multiple companies seek to move CAR-T cell therapies forward in clinical trials, what will be interesting to see is how this novel treatment fits in with existing therapies such as bone marrow transplants. Will it replace them, or be a bridge to a transplant that enables relapsed or refractory patients to have a second chance?

In addition, where are the potential opportunities beyond B-cell malignancies such as acute lymphoid leukemia (ALL) where there’s been dramatic success, particularly in children?

Last week Biotech Strategy Blog had the privilege to interview Dr Krishna Komanduri who is Director of the Adult Stem Cell Transplant Program at the University of Miami Sylvester Cancer Center and holds the Kalish Family Chair in Stem Cell Transplantation.

A physician scientist, he exudes a sense of calm professionalism – I am sure this must reassure many of his patients. Having a bone marrow transplant has been likened to jumping off a cliff in terms of what it does to one’s immune system.

In the last 2-3 years, he has dramatically increased the number of transplants at the University of Miami Sylvester Cancer Center.

Dr Komanduri (@DrKomanduri) was co-chair of the 2015 BMT Tandem meeting that took place earlier this month in San Diego. It’s the combined annual meeting of the American Society of Blood and Marrow Transplantation (ASMBT) and the Center for International Blood and Marrow Transplant Research (CIBMTR).

In a half hour interview he shared his thoughts on what was exciting at Tandem, where the field is going and some of the best abstracts at the meeting which included data on CAR-T cell therapy, GVHD and gene therapy.

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Bellicum moves BPX-501 T Cell Therapy forward $BLCM

Several subscribers have written to ask what we think of Houston based Bellicum Pharmaceuticals?

Bellicum is a company that along with Novartis, Kite, Juno and Cellectis has a Chimeric Antigen Receptor (CAR) T cell therapy in development, amongst other things.

Readers already know the company had a successful IPO in December (NASDAQ: BLCM) and were reported to have raised $140M to fund future development.

This morning, the company announced enrollment of the first cohort of pediatric patients in a phase 1/2 dose escalation trial of BPX-501 (link to press release). This T cell therapy aims to mitigate the risk of graft versus host disease (GvHD) after an allogeneic haploid hematopoietic stem cell transplant.

BSB spoke with Bellicum CEO Tom Farrell and COO Dr Annemarie Moseley to answer some of the questions we think subscribers would like to know more about such as:

  • Market opportunity for BPX-501
  • Mechanism of action of BPX-501
  • Strategic direction the company is taking
  • Vision with regards to its CAR-T development
  • Milestones expected in 2015

We’ve provided some additional commentary on the challenges and opportunities Bellicum may face in the GvHD market and how we think the company stacks up against the competition in the CAR-T space. Be warned this piece is a long read: 6,000+ words!

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Cellectis ramps up CAR-T development in 2015

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.

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. 

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