Biotech Strategy Blog

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

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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One Response to “Bellicum moves BPX-501 T Cell Therapy forward $BLCM”

  1. kirkhartley

    Thank you – that’s a very helpful summary. To follow up on part of your comments, pasted below are key excerpts from an article that indicates improvement in finding biomarkers to track developing GVHD. It feels as if steps of that sort will make it more feasible to use slow acting but less expensive drugs.
    ____________________________________________
    “A new test can guide treatment for patients with graft versus host disease (GVHD), an often life-threatening complication of bone marrow and stem cell transplants, according to research from the University of Michigan published in Lancet Haematology this month. ****

    Most GVHD starts out as mild, but in two-thirds it eventually becomes severe. The treatment for severe GVHD is high doses of medications that knock out the immune system. But doctors have to be careful with drugs that further weaken a newly transplanted immune system, because they increase the risk for serious and life-threatening infections. Until now there has been no test to determine which cases of GVHD will become severe, so treatment is often delayed until the GVHD worsens.

    The study’s lead author, John Levine, M.D., of the University of Michigan’s Blood and Marrow Transplant Program and his colleagues studied almost 800 patients from the US and Germany to develop and validate a new scoring system. The Ann Arbor GVHD score uses the levels of three proteins in the blood (TNFR1, ST2, and REG3a) to determine whether the patient should be treated right away or not and how intense the treatment should be. Patients with Ann Arbor 1 GVHD usually don’t need treatment while patients with Ann Arbor 3 GVHD often don’t respond to standard treatment and should be considered for clinical trials.”

    http://www.sciencedaily.com/releases/2015/01/150113184046.htm?utm_source=feedburner&utm_medium=email&utm_campaign=Feed%3A+sciencedaily%2Fhealth_medicine%2Fleukemia+%28Leukemia+News+–+ScienceDaily%29

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