Biotech Strategy Blog

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

Posts from the ‘GvHD’ category

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…

In case you missed it, do listen to Dr Marcel van den Brink talking about GvHD on the Novel Targets Podcast. (Episode 9: Targeting the Microbiome)

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

Cellectis LogoLast 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.

Here’s an excerpt of the interview Cellectis CEO André Choulika, PhD gave Biotech Strategy Blog last year – it was the No1 post in 2014: Can Cellectis Revolutionize CAR-T cell Immunotherapy?

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?

Dr Krishna KomanduriLast 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 logoSeveral 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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