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.
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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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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T cell activation has been very much to the fore over the last couple of years with many companies looking at different ways to use them against cancer cells, with chimeric antigen receptor (CAR) T cell therapy, vaccines or monoclonal antibodies. There are situations though, where T cells are not necessarily a good thing.
Graft versus Host disease (GvHD) is an area of tremendous unmet medical need that is triggering the interest of a number of biotech and rare disease companies such as Alexion Pharmaceuticals (ALXN).
Houston based Bellicum Pharmaceuticals (BLCM), whose IPO raised around $140M last month, have said they plan to spend most of the funds on bringing to market a new cell therapy that could make stem cell transplants more effective and reduce GvHD. They also have a CAR-T therapy in early development.
Indeed, at last month’s ASH 2014 annual meeting in San Francisco, GvHD was very much a hot topic, with data presented in the plenary session by Dr Wei Li (pictured below) on a novel biomarker for GI GvHD.
This post discusses one of the GvHD oral sessions at ASH 2014, and includes post-presentation commentary from Dr Marcel van den Brink, who is an expert in the area. The related interview Dr Brink kindly gave BSB at the SITC annual meeting is well worth reading if you missed it.
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