3 Things In Biotech You Should Learn Today: April 17, 2017

Includes: BMY, CASI, NVCR
by: Zach Hartman

Boehringer entering into collaboration with Bristol-Myers to ramp immunotherapy to new heights.

CASI Pharmaceuticals stops enrollment in its phase 2 breast cancer trial.

NovoCure achieves significant landmark by tuning tumors out of your brain.

Welcome, everyone! After taking an Easter Sunday off, I'm back to delve into some more recent biotech news and views. There are some interesting things to discuss, but we'll be taking a few step backwards in time to continue covering some straggling news from AACR, which continues to ripple. Still, you may not have heard the news on these companies!

Let's get to it.

Boehringer entering into collaboration with Bristol-Myers to ramp immunotherapy to new heights

Apexigen is a clinical-stage biotech partnered with Boehringer Ingelheim to develop APX005M, an antibody designed to activate the CD40 molecule on antigen-presenting cell macrophage and B cells, to stimulate enhanced innate and adaptive immune responses. It is currently in early stage clinical studies as a monotherapy for several tumor types.

On April 11, the company announced that it is initiating a clinical trial in conjunction with Bristol-Myers Squibb (NYSE:BMY) to investigate the potential of adding APX005M to Opdivo therapy in the settings of second-line lung cancer (after failure of chemotherapy) and melanoma (after failure of immunotherapy).

Looking forward: Activation of CD40 has been hypothesized as an avenue for therapy for a variety of tumor types, including very difficult to treat malignancies like pancreatic cancer. So this collaboration does not come as a shock. The continued exploration of targeting the tumor microenvironment has led us to many promising innovations, like the IDO inhibitors. This feels intuitively like a high-risk, high-reward strategy, as further indiscriminate activation of the immune system intuitively would see to carry a greater risk of immunologic toxicity, which is already a rare but serious issue with Opdivo. Still, this collaboration continues to point where BMY's head is with respect to forward momentum in immunotherapy.

CASI Pharmaceuticals stops enrollment in its phase 2 breast cancer trial

CASI Pharmaceuticals (NASDAQ:CASI) is a microcap biotech currently developing a multitargeted kinase inhibitor, ENMD-2076, for several forms of cancer. This agent is designed to block Aurora kinase A, along with several growth factor receptors, including Flt-3 and VEGFR.

On April 14, CASI announced that they had reached an agreement with their investigators to terminate enrollment in a phase 2 study of ENMD-2076 in triple-negative breast cancer (triple-negative meaning it lacks expression of the two hormone receptors and HER2). Enrollment was stopped at 41 patients due to factors relating to recruitment, as triple-negative breast cancer is an uncommon form of the disease, representing between 10% and 15% of tumors. CASI will continue to monitor patients in this study, and they feel they have accrued enough patients to assess the preliminary efficacy of ENMD-2076.

Looking forward: At its face, I can buy the explanation that recruitment was difficult, and that CASI would want to lower costs as much as possible. Triple-negative breast cancer remains a substantial unmet need, but indeed most cases are not triple-negative. Moreover, finding patients to undergo testing with a multitargeted kinase inhibitor is probably another challenge in this era where immunotherapy "miracle cures" are being studied aggressively in triple-negative disease. Personally, I wouldn't feel so confident that CASI will be able to move this agent too far.

NovoCure achieves significant landmark by tuning tumors out of your brain

A few weeks ago at AACR 2017, NovoCure Limited (NASDAQ:NVCR) presented findings from its EB-14 study, which assessed the addition of its tumor-treating fields (TTF) technique to standard chemotherapy for glioblastoma.

After over three years follow-up, it has become clear that adding TTF to temozolomide increased survival in patients with a hazard ratio of 0.63 (so a 37% improvement). Roger Stupp, a lead investigator on the EB-14 study, provides some fantastic perspective on the grim prognosis of glioblastoma in the era before TTF technology.

When I started treating patients with GBM 20 years ago, the majority of patients died within less than one year and long-term survival was nearly absent. Now, we see a meaningful improvement in survival at two years and beyond. With the combination of Optune and temozolomide, one out of seven patients is living longer than five years."

Looking forward: Since around 2014 (for me), NVCR has been producing some seriously enticing data in the realm of glioblastoma. These are tough tumors to treat, and the idea that you could target the brain and get increased activity with tumor-treating fields is very interesting. And NVCR is rapidly improving the technology to increase its wearability (early models were heavy, required you to carefully shave your head, etc), which can lead to better compliance and efficacy. These EB-14 results further confirm that NVCR has something serious on its hands with this, as we knew it was able to give similar efficacy to chemotherapy but without systemic side effects.

Back when I started paying attention to Optune, it was unclear whether the benefit seen in the early days of the late-stage studies was a bit of a fluke, or whether the technology was worth it. EB-14 is pretty much unambiguous, with a clinically meaningful improvement in survival for a very, very high-risk subset of patients.


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